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Wearables and Healthcare: The 3 Companies Leading the Way – Nasdaq

November 15th, 2019 8:43 pm

Initially seen as a fashion accessory, smartwatches and fitness bands have now entered the healthcare ecosystem. By leveraging advanced technologies such as Artificial Intelligence (AI), Cloud, Machine Learning, Big Data and Analytics, these wrist-worn devices are becoming more sophisticated and relevant in the present day by providing actionable insights and prescriptive measures for end users. This has also ushered in technology giants such as Apple, Samsung and Google in the healthcare space.

Heres a look at the fast-growing wrist-wear market in the backdrop of the digital health space

The increasing focus on personalized care, early detection, and precision medicine along with rising health consciousness is driving the demand for healthcare-related wearables and applications. Asurveyby Accenture shows that the use of wearable devices by consumers has nearly quadrupled in the four year period (2014-2018), from just 9% in 2014 to 33% in 2018. Further, roughly three-fourths of health consumers view wearables such as those that monitor glucose, heart rate, physical activity and sleep as beneficial to understanding their health condition (75%), engaging with their health (73%), and monitoring the health of a loved one (73%).

The smartwatch market is growing at a fast pace and isexpectedto reach a market value of $130.55billion by 2024 from $48.14billion in 2018, registering a CAGR of 18.23% during the forecast period (2019-2024).

Together, Apple, Samsung and Fitbit capture 72% of the market.

In 2018, Apple (AAPL)receivedDe Novo classification for the ECG app and the irregular heart rhythm notification from the Food and Drug Administration (FDA). The AppleWatchis capable of generating an ECG similar to a single-lead electrocardiogram; it can check the heart rate and alert a person about any irregularities. The Apple Watch comes with a vital fall detection feature and can even provide insight to women about their menstrual cycle. Not just smartwatches, Apple is working across the healthcare technology segment, something which has been echoed by Tim Cook in one of his interviews where hesaid, If you zoom out into the future, and you look back, and you ask the question, What was Apples greatest contribution to mankind? It will be about health.

Apples arch rival in the smartphone space, Samsung (SSNLF) is one of its closest competitors in the wrist wearables space as well. Samsung smartwatches help track heart rate, stress levels, sleep, water intake and much more. In June this year, the Galaxy Watch Activerevealedits anytime, anywhere blood pressure-checking capability using My BP Lab 2.0.

The global smartwatch shipments grew an impressive 42% annually to reach 14 million units in the third quarter of 2019. Apple Watch maintained first position with 48% global smartwatch market share, while Samsung held second place, and Fitbit (now acquired by Google) ranks at third,accordingto Strategy Analytics.

Fitbit (FIT) once enjoyed being the uncrowned king of the wearable device market, controlling a major market share untilthe Apple Watch came on the scene, and the dynamics started to change. Fitbitsold22.3 million connected health and fitness devices in 2016, while the same dropped to 13.9 million in 2018. The limited functionality of a fitness band when compared to a smartwatch has been a major reason for Fitbits shrinking market share. A CCS reporthighlights, Western consumers now find less value in the simple fitness bands, even with the added convenience of long battery life, preferring to move on to smartwatches, which offer all the functionality of fitness trackers and so much more.

Earlier this month, Google (GOOG,GOOGL) entered into a definitive agreement to acquire Fitbit for $2.1 billion. Over the years, Fitbit has sold more than 100 million devices and utilizes data to deliver unique personalized guidance and coaching to its users. While, Google introducedWear OSby Google back in 2014, and has been makingadvances in medical technologybut not particularly in wearables. Fitbit gives Google the opportunity to expandfurtherinto the wearables segment as well as introduce Made by Google wearable devices into the market.Back in 2018, Fitbit and Google collaborated to explore the development of consumer and enterprise health solutions. Last month, Fitbitannouncedan alliance with Bristol-Myers Squibb and Pfizer to help drive timely diagnosis of atrial fibrillation (AFib) with the aim of improving earlier detection in individuals at increased risk of stroke.

Fitbit and Google together can become a significant competitor to the existing market leaders in the wrist-wearables space. Wearables will assume a more significant role in the healthcare ecosystem and become a significant revenue generator for the technology providers. WHO Director-General Dr. Tedros Adhanom Ghebreyesus summarizesthe role of digital health technologies: Harnessing the power of digital technologies is essential for achieving universal health coverage. Ultimately, digital technologies are not an end in themselves; they are vital tools to promote health, keep the world safe, and serve the vulnerable.

The views and opinions expressed herein are the views and opinions of the author and do not necessarily reflect those of Nasdaq, Inc.

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SLC22A1 And ATM Genes Polymorphisms Are Associated With The Risk Of Ty | TACG – Dove Medical Press

November 15th, 2019 8:43 pm

Rana M Altall,1 Safaa Y Qusti,1 Najlaa Filimban,2 Amani M Alhozali,3 Najat A Alotaibi,4 Ashraf Dallol,2 Adeel G Chaudhary,2 Sherin Bakhashab1,2

1Department of Biochemistry, Faculty of Science, King Abdulaziz University, Jeddah 21589, Kingdom of Saudi Arabia; 2KACST Technology Innovation Center in Personalized Medicine, King Abdulaziz University, Jeddah 21589, Kingdom of Saudi Arabia; 3Department of Internal Medicine, Faculty of Medicine, King Abdulaziz University, Jeddah 21589, Kingdom of Saudi Arabia; 4Department of Family and Community Medicine, Faculty of Medicine, King Abdulaziz University Hospital, Jeddah 21589, Kingdom of Saudi Arabia

Correspondence: Sherin BakhashabKing Abdulaziz University, P.O. Box 80218, Jeddah 21589, Kingdom of Saudi ArabiaTel +966 12 6400000Fax +966 12 6952076Email sbakhashab@kau.edu.sa

Introduction: Type 2 diabetes mellitus (T2DM) is a major global health problem that is progressively affected by genetic and environmental factors. The aim of this study is to determine the influence of solute carrier family 22 member 1 (SLC22A1) rs628031 and rs461473, and ataxia telangiectasia mutated (ATM) rs11212617 polymorphisms on the risk of T2DM in Saudi Arabia by considering many parameters associated with glycemic control of T2DM, such as body mass index (BMI), fasting blood glucose, glycated hemoglobin (HbA1c), and triglyceride.Methods: In a case-control study, genomic DNA from controls and diabetic groups was isolated and genotyped for each single-nucleotide polymorphism.Results: There were significant correlations between T2DM and both BMI and HbA1c. Significant associations between G/G and A/G genotypes of rs628031 and rs461473 variants of SLC22A1 and high levels of HbA1c were detected. Therefore, G was predicted to be the risk allele among the assessed SLC22A1 variants. A significant correlation was observed between A/A and A/C genotypes of the rs11212617 polymorphism of ATM and elevated HbA1c. Relative risk calculation confirmed A to be the risk allele in the T2DM population.Conclusion: Our study showed the risk of the assessed SLC22A1 and ATM variants on glycemic control parameters in diabetic patients.

Keywords: type 2 diabetes mellitus, single-nucleotide polymorphism, solute carrier family 22 member 1, ataxia telangiectasia mutated

This work is published and licensed by Dove Medical Press Limited. The full terms of this license are available at https://www.dovepress.com/terms.php and incorporate the Creative Commons Attribution - Non Commercial (unported, v3.0) License.By accessing the work you hereby accept the Terms. Non-commercial uses of the work are permitted without any further permission from Dove Medical Press Limited, provided the work is properly attributed. For permission for commercial use of this work, please see paragraphs 4.2 and 5 of our Terms.

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SLC22A1 And ATM Genes Polymorphisms Are Associated With The Risk Of Ty | TACG - Dove Medical Press

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Personalized Medicine Market Analysis by Top Companies, Driver, Existing Trends and Global Forecast by 2026 – WindStreetz

November 15th, 2019 8:43 pm

Personalized Medicine Market Overview:

The Personalized Medicine Market is expected to grow at a significant pace, reports Verified Market Research. Its latest research report, titled [Global Personalized Medicine Market Size and Forecast 2019-2026, Breakdown Data by Companies, Key Regions, Types and Application], offers a unique point of view about the global market. Analysts believe that the changing consumption patterns are expected to have a great influence on the overall market. For a brief overview of the global Personalized Medicine Market, the research report provides an executive summary. It explains the various factors that form an important element of the market. It includes the definition and the scope of the market with a detailed explanation of the market drivers, opportunities, restraints, and threats.

GlobalPersonalized Medicine Market: Segmentation

The chapters of segmentation allow the readers to understand the aspects of the market such as its products, available technologies, and applications of the same. These chapters are written in a manner to describe their development over the years and the course they are likely to take in the coming years. The research report also provides insightful information about the emerging trends that are likely to define progress of these segments in the coming years.

Request a Sample Copy of this report @https://www.verifiedmarketresearch.com/download-sample/?rid=7106&utm_source=WSN&utm_medium=AK

Key Players Mentioned in the Personalized Medicine Market Research Report:

Personalized Medicine Market: Regional Segmentation

For a deeper understanding, the research report includes geographical segmentation of the Personalized Medicine Market. It provides an evaluation of the volatility of the political scenarios and amends likely to be made to the regulatory structures. This assessment gives an accurate analysis of the regional-wise growth of the Personalized Medicine Market.

Personalized Medicine Market: Research Methodology

The research methodologies used by the analysts play an integral role in the way the publication has been collated. Analysts have used primary and secondary research methodologies to create a comprehensive analysis. For an accurate and precise analysis of the Personalized Medicine Market, analysts have bottom-up and top-down approaches.

Ask for Discount @https://www.verifiedmarketresearch.com/ask-for-discount/?rid=7106&utm_source=WSN&utm_medium=AK

Table of Content

1 Introduction of Personalized Medicine Market

1.1 Overview of the Market 1.2 Scope of Report 1.3 Assumptions

2 Executive Summary

3 Research Methodology of Verified Market Research

3.1 Data Mining3.2 Validation3.3 Primary Interviews3.4 List of Data Sources

4 Personalized Medicine Market Outlook

4.1 Overview4.2 Market Dynamics4.2.1 Drivers4.2.2 Restraints4.2.3 Opportunities4.3 Porters Five Force Model4.4 Value Chain Analysis

5 Personalized Medicine Market, By Deployment Model

5.1 Overview

6 Personalized Medicine Market, By Solution6.1 Overview

7 Personalized Medicine Market, By Vertical

7.1 Overview

8 Personalized Medicine Market, By Geography8.1 Overview8.2 North America8.2.1 U.S.8.2.2 Canada8.2.3 Mexico8.3 Europe8.3.1 Germany8.3.2 U.K.8.3.3 France 8.3.4 Rest of Europe 8.4 Asia Pacific 8.4.1 China 8.4.2 Japan 8.4.3 India 8.4.4 Rest of Asia Pacific 8.5 Rest of the World 8.5.1 Latin America 8.5.2 Middle East

9 Personalized Medicine Market Competitive Landscape

9.1 Overview 9.2 Company Market Ranking 9.3 Key Development Strategies

10 Company Profiles

10.1.1 Overview 10.1.2 Financial Performance 10.1.3 Product Outlook 10.1.4 Key Developments

11 Appendix

11.1 Related Research

Complete Report is Available @ https://www.verifiedmarketresearch.com/product/global-personalized-medicine-market-size-and-forecast-to-2026/?utm_source=WSN&utm_medium=AK

We also offer customization on reports based on specific client requirement:

1-Freecountry level analysis forany 5 countriesof your choice.

2-FreeCompetitive analysis of any market players.

3-Free 40 analyst hoursto cover any other data points

About Us:

Verified Market Research has been providing Research Reports, with up to date information, and in-depth analysis, for several years now, to individuals and companies alike that are looking for accurate Research Data. Our aim is to save your Time and Resources, providing you with the required Research Data, so you can only concentrate on Progress and Growth. Our Data includes research from various industries, along with all necessary statistics like Market Trends, or Forecasts from reliable sources.

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Tag: Personalized Medicine Market Size, Personalized Medicine Market Growth, Personalized Medicine Market Analysis, Personalized Medicine Market Forecast, Personalized Medicine Market Outlook, Personalized Medicine Market Trends, Personalized Medicine Market Research, Personalized Medicine Market Report

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Personalized Medicine Market Analysis by Top Companies, Driver, Existing Trends and Global Forecast by 2026 - WindStreetz

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Anatomic Pathology Market – Global Forecast to 2024: High Incidence of Cancer and Other Target Diseases Drives Growth – ResearchAndMarkets.com -…

November 15th, 2019 8:43 pm

DUBLIN--(BUSINESS WIRE)--The "Anatomic Pathology Market by Product & Service (Instruments (Tissue Processing Systems, Microtomes), Consumables (Antibodies), Histopathology), Application (Disease Diagnosis (Cancer (Gastrointestinal)), End User (Hospital, Lab) - Global Forecast to 2024" report has been added to ResearchAndMarkets.com's offering.

The anatomic pathology market is projected to reach a value of USD 44.4 billion by 2024 from USD 33.0 billion in 2019, at a CAGR of 6.1% during the forecast period.

The growth in this market is attributed to the high incidence of cancer and other target diseases, availability of reimbursement, and the growing focus on personalized medicine.

In addition, emerging economies such as China, India, and Brazil are expected to offer significant growth opportunities for players operating in the anatomic pathology market during the forecast period. However, the availability of refurbished products, the lack of skilled professionals, and product recalls are expected to hamper the market growth to a certain extent in the coming years.

Disease diagnosis segment accounted for the larger share of the anatomic pathology market, by application, in 2018

Based on application, the anatomic pathology market is segmented into disease diagnosis and medical research. In 2018, the disease diagnosis segment accounted for the larger market share, due to the rapid growth in the geriatric population and the increasing incidence of cancer and other chronic diseases.

Hospital laboratories to register the highest growth in the anatomic pathology market during the forecast period

Based on end-user, the anatomic pathology market is segmented into clinical laboratories, hospital laboratories, and other end users. The hospital laboratories segment accounted for the largest market share in 2018 and is projected to register the highest CAGR during the forecast period. The growth of this segment can be attributed to the increasing number of patient visits to hospitals, a growing number of in-house diagnostic procedures performed in hospitals, growing awareness regarding early disease diagnosis, and the availability of reimbursements in developed markets for clinical tests performed in hospitals.

North America will continue to dominate the anatomic pathology market during the forecast period

Geographically, the anatomic pathology market is segmented into North America, Europe, the Asia Pacific, and the Rest of the World. In 2018, North America accounted for the largest market share, followed by Europe. Factors such as the rising prevalence of chronic diseases, increasing healthcare expenditure, the high-quality infrastructure for hospitals and clinical laboratories, and the presence of major market players in the region are driving the growth of the anatomic pathology market in North America.

Market Dynamics

Drivers

Opportunities

Challenges

Trends

List of Companies Profiled in the Report

For more information about this report visit https://www.researchandmarkets.com/r/i0353n

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Anatomic Pathology Market - Global Forecast to 2024: High Incidence of Cancer and Other Target Diseases Drives Growth - ResearchAndMarkets.com -...

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Mosunetuzumab Induces Complete Remissions in Poor Prognosis Non-Hodgkin Lymphoma Patients, Including Those Who Are Resistant to or Relapsing After…

November 15th, 2019 8:43 pm

Introduction:Improved treatments are needed for relapsed or refractory (R/R) non-Hodgkin lymphoma (NHL) pts. Options are particularly limited for pts with B-cell NHLs who are R/R to CAR-T therapies or for whom a delay in effective therapy precludes this approach. Mosunetuzumab (M; RG7828) is a full-length, fully humanized immunoglobulin G1 (IgG1) bispecific antibody targeting both CD3 (on the surface of T cells) and CD20 (on the surface of B cells). In an ongoing Phase I/Ib study (GO29781; NCT02500407), promising efficacy and favorable tolerability were observed in R/R NHL pts (Budde et al. ASH 2018; Bartlett et al. ASCO 2019). We report complete remissions (CRs) with M in NHL pts who are R/R to CAR-T therapy, as well as activity with M re-treatment.

Methods:GO29781 is an open-label, multicenter, Phase I/Ib, dose escalation and expansion study of M in R/R B-cell NHL. Data is presented from Group B, in which M is administered with step-up dosing on Days 1, 8, and 15 of Cycle 1, and then as a fixed dose on Day 1 of each subsequent 21-day cycle (maximum 17 cycles). Outcome measures include best objective response rate (ORR) by revised International Working Group criteria, maximum tolerated dose (MTD), and tolerability.

Results:As of June 4, 2019, 218 pts in Group B had received any amount of M. Indolent NHL (iNHL) pts (n=72) were mainly follicular lymphoma (FL, n=69). Aggressive NHL (aNHL) pts (n=141) were mainly diffuse large B-cell lymphoma (DLBCL, n=87) or transformed FL (trFL, n=29). Median prior systemic therapies was 3 (range: 1-14). Twenty-three pts had prior CAR-T therapy (12 DLBCL, 6 trFL, 5 FL), and 16 were efficacy evaluable (7 DLBCL, 5 trFL, 4 FL). ORR and CR rates were 43.8% (7/16) and 25.0% (4/16, 2 DLBCL and 2 FL), respectively. Expansion of previously administered CAR-Ts after M administration was detected by quantitative PCR, in line with the mechanism of action of M.

Dose escalation is ongoing, supported by a positive exposure-response relationship for efficacy and broad therapeutic window with step-up dosing (Li et al. ASH 2019). Among efficacy-evaluable pts across all dose levels, ORR and CR rates were 64.1% (41/64) and 42.2% (27/64) in iNHL pts and 34.7% (41/119) and 18.6% (22/119) in aNHL pts, respectively.

CRs appeared durable, with 25/27 (92.6%) iNHL pts (median time from first CR: 5.8 months; range: 0.2-28.9) and 15/22 (68.2%) aNHL pts (median time from first CR: 8.8 months; range: 0.0-25.4) who achieved CR remaining in remission. Re-treatment with M was allowed in CR pts who relapsed. Four pts, including 1 in Group A who was initially treated with a fixed, non-step-up dosing schedule, received M re-treatment. One CR and 2 partial responses were observed. All three responses are ongoing, with the CR pt in second remission for 314 days.

The MTD of M has not been reached at doses up to 1/2/60mg (Cycle 1 Day 1, 8, and 15). Adverse events (AEs) leading to treatment withdrawal were uncommon (12/218, 5.5%). Cytokine release syndrome (CRS), graded by Lee criteria (Lee et al. Blood 2014;124:188-95), was observed in 28.4% of pts, and was mostly Grade (Gr) 1 (21.1%) or Gr 2 (6.0%); Gr 3 CRS occurred in 1.4% of pts. Most CRS events occurred in Cycle 1; 5 pts (2.7%) had CRS during or after Cycle 2. Three of 218 pts (1.4%) received tocilizumab for CRS management; all 3 events resolved without sequelae (for 1 pt, CRS resolved after the cutoff date). Neurological AEs (NAEs) were reported in 44% of pts (Gr 1, 28.0%; Gr 2, 12.8%; Gr 3, 3.2%). Common NAEs were headache (14.7%), insomnia (10.1%), and dizziness (9.2%). Potential immune effector cell-associated neurotoxicity syndrome (ICANS)-like NAEs of Gr 1 or Gr 2 confusional state occurred in 3 pts (1.4%) during cycles 1 and 2. The frequency of CRS and NAEs did not correlate with M exposure, likely due to step-up dosing, which effectively mitigates acute toxicities and allows administration of higher doses (Bartlett et al. ASCO 2019; Li et al. ASH 2019). Among the 4 pts who were re-treated with M, no CRS was observed and NAEs were reported in 1 pt (Gr 1 headache and insomnia). Among the 23 pts who were R/R to CAR-T therapy, CRS occurred in 5 pts (21.7%; Gr 1, 13.0%; Gr 2, 4.3%; Gr 3, 4.3%) and NAEs in 8 pts (34.8%; Gr 1, 17.4%; Gr 2, 13.0%; Gr 3, 4.3%), with no ICANS-like events.

Conclusions:M has favorable tolerability and durable efficacy in pts with heavily pre-treated R/R B-cell NHL, including CRs in pts with disease progression after CAR-T therapies. Preliminary data support the possibility for re-treatment with M.

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The world’s first Gattaca baby tests are finally here – MIT Technology Review

November 14th, 2019 4:51 pm

Anxious couples are approaching fertility doctors in the US with requests for a hotly debated new genetic test being called 23andMe, but on embryos.

The baby-picking test is being offered by a New Jersey startup company, Genomic Prediction, whose plans we first reported on two years ago.

The company says it can use DNA measurements to predict which embryos from an IVF procedure are least likely to end up with any of 11 different common diseases. In the next few weeks it's set to release case studies on its first clients.

Handed report cards on a batch of frozen embryos, parents can use the test results to try to choose the healthiest ones. The grades include risk estimates for diabetes, heart attacks, and five types of cancer.

According to flyers distributed by the company, it will also warn clients about any embryo predicted to become a person who is among the shortest 2% of the population, or who is in the lowest 2% in intelligence.

The test is straight out of the science fiction film Gattaca, a movie thats one of the inspirations of the startups CEO, Laurent Tellier. The companys other cofounders are testing expert Nathan Treff and Stephen Hsu, a Michigan State University administrator and media pundit.

So far, fertility centers have not leaped at the chance to offer the test, which is new and unproven. Instead, prospective parents are learning about the designer baby reports through word of mouth or news articles and taking the companys flyer to their doctors.

One such couple recently turned up at New York Universitys fertility center in Manhattan, says David Keefe, who is chairman of obstetrics and gynecology there. Right off the bat it raises all kind of questions about eugenics, he says.

Keefe, who has seven children, worries that couples who think they can choose kids from a menu could be disappointed. Its fraught with parenting issues, he says. So many couples just need to feel they have done enough.

Picking your baby

The companys project remains at a preliminary stage. While some embryos have been tested by the company, Tellier, the CEO, says he is unsure if any have yet been used to initiate a pregnancy.

The test is carried out on a few cells plucked from a days-old IVF embryo. Then Genomic Prediction measures its DNA at several hundred thousand genetic positions, from which it says it can create a statistical estimate, called a polygenic score, of the chance of disease later in life.

Genomic prediction

In October, the company pitched the test, which it calls LifeView, from a trade-show booth at the annual meeting of fertility doctors in Philadelphia. A promotional banner read: She has your partners ears and smile. Just not their risk of diabetes.

Criticism of the company from some genetics researchers has been intense.

It is irresponsible to suggest that the science is at the point where we could reliably predict which embryo to select to minimize the risk of disease. The science simply isnt there yet, says Graham Coop, a geneticist at the University of California, Davis, and a frequent critic of the company on Twitter.

The company has raised several million dollars in venture capital from investors including People Fund, Arab Angel, Passport Capital, and Sam Altman, the chairman of Y Combinator and CEO of OpenAI.

At an investor event last April, Genomic Prediction compared itself to 23andMe for IVF clinics and boasted it was preparing for a massive marketing push.

Our reporting suggests the company has struggled both to validate its predictions and to interest fertility centers in them. Its customers so far seem to be a scattering of individuals from around the world with specific family health worries. The company declined to name them, citing confidentiality.

The company is expected to soon present its first case reports, describing clients and their embryo test results. One case involves a married gay couple who have begun IVF using donor eggs and plan to employ a surrogate mother. That couple wants a child with a low risk for breast cancer.

How will it be used?

Genomic Prediction thinks it can piggyback on the most common type of preimplantation embryo test, which screens days-old embryos for major chromosome abnormalities, called aneuploidies. Such testing has become widespread in fertility centers for older mothers and is already employed in nearly a third of IVF attempts in the US. The new predictions could be added to it.

Fertility centers can also order tests for specific genetic diseases, such as cystic fibrosis, where a gene measurement will give a definite diagnosis of what embryo inherited the problem The new polygenic tests are more like forecasts, estimating risk for common diseases on the basis of variations in hundreds or thousands of genes, each with a small effect.

In a legal disclaimer, the company says it cant guarantee anything about the resulting child and that the assessment is NOT a diagnostic test.

Santiago Munne, an embryo testing expert and entrepreneur, thinks patients already undergoing aneuploidy testing would likely want the add-on test, but that doctors could object if it introduces uncertainty: For monogenic disease, if the embryo is abnormal, we will tell you, and it is. With a risk score, it may be affected. And some patients will only have embryos with higher risks. Then what?

As well, he says it wont be possible with a test to optimize a child for many features at once: My personal opinion is once you start looking, some embryos will be brighter, some will be taller, some will have longevity, and none will have those qualities all together. And in an IVF cycle, you produce maybe six embryos on average. You wont be able to get all the traits that you want.

Despite such inherent limits, theres a bigger plan afoot. Treff, the startup's chief scientist, believes even fertile couples might begin to undergo IVF just so they can select the best child. I do believe this is going to be the future we can start to ... reduce the incidence of disease in humans through IVF, Treff told an audience at a conference in China last month.

How many people will be willing to go through the trouble of IVF if they dont need it to have a baby? IVF involves weeks of hormone shots and two medical procedures (one to collect eggs, another to implant the embryos) and typically costs around $15,000. Add to that the companys fee to test embryos, which is $1,000, plus $400 for each embryo scored.

If someone is fertile, unless there is a family history of disease, I dont think that it is going to be popular, says Munne.

Can you get a smarter baby?

Genomic Prediction has so far won the most attention for the possibility of using genetic scores to pick the most intelligent children from a petri dish. It has tried to distance itself from the controversial concept, but thats been difficult because Hsu, a cofounder, is frequently in the media discussing the idea.

Hsu told The Guardian this year that accurate IQ predictors will be possible if not in the next five years, the next 10 years certainly. He says other countries, or the ultra-wealthy, might be the first to try to boost IQ in their kids this way.

During his talk in China, Treff called improving intelligence via embryo selection an application that many people think is unethical." In private, Treff tells other scientists he thinks it's doable, but wants to promote the technology for medical purposes only.

Ms Tech

For now, the company is limiting itself to alerting parents to embryos it predicts will be the least intelligent, with the highest chance of an IQ which qualifies as intellectual disability according to psychiatric manuals.

Some experts see a transparent maneuver to avoid controversy. They say theyre going to test for the medical condition of intellectual disability, not for the smartest embryos, because they know people are going to object to that, says Laura Hercher, who trains genetic counselors at Sarah Lawrence College. They are trying to slide, slide into traits without admitting as much.

A May report from the Hebrew University of Jerusalem found that trying to pick the tallest or smartest embryos might not work particularly well. Researchers there estimated that using polygenic scores to locate the tallest or smartest child from a batch of sibling embryos would result in an average gain of 2.5 centimeters in height, and less than three IQ points.

They modeled what everyone is scared of happening, Treff said of that study. Its not what we are doing.

The predictions, however, could be more effective at helping people avoid children with specific diseases. Treff, during his speech in China, said that a couple choosing between two embryos would see, on average, a 45% reduction in risk for type 1 diabetes. That is a serious disease from which Treff suffers and which runs in families, although it has complex causes. The more embryos there are to choose from, he says, the more the risk will go down.

Demand for the test

Patients and doctors are mostly on their own when it comes to deciding if the tests really work. While federal and state agencies do oversee laboratory accuracy, the oversight is limited to whether analytes like DNA are correctly measured, not what they mean. So Genomic Prediction doesnt need to prove that the test is useful before selling it. In fact, it could take decades to ascertain if tested kids fare better than others.

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And its not only whether the test works or not. Uptake will depend on demand from patients and the degree of pushback from doctors and genetic counselors. In the US, tests for genderthat is, picking a boy or a girl embryoare accepted and relatively routine. But thats never become the case for choosing eye color, which is also possible. In terms of eye color, the pressure not to do it, to not offer it, was met with a weak market demand. So it doesnt exist, says Hercher.

Genomic Prediction provided a map of 12 fertility clinics it says will order its test, including five in the US and others in Nigeria, Peru, Thailand, and Taiwan.

MIT Technology Review was able to independently locate two IVF clinics where customers have recently requested the embryo predictions. Michael Alper, founder of Boston IVF, one of the worlds largest fertility clinics, says his center was approached by a couple a few weeks ago but he decided the request needs to be weighed by the centers ethics committee before he would agree to order it.

This is the first case we have had, says Alper. To me its a 23andMe type of prediction: theres a propensity, but how strong? That is the problem. We dont have any problem testing for cystic fibrosisthat is a lethal disease, it strikes young. But we are not there yet with these other tests. Its soft; its not that predictive.

At NYU, Keefe says the test raises profound questions. His center is in Midtown Manhattan, just blocks from a hub of finance and legal offices. He says his clientele are typically well-off professionals, people who have programmed everything in life and feel they are in control. They sometimes even ask out loud if a mere doctor is smart enough to help them.

The case he is working on involves a family that has two children with autism. They now want a child without the condition, and they hope the intelligence feature of the test will help them. Treff says he counseled the family that the Genomic Prediction test wasnt likely to helpautism can have specific genetic causes that the intelligence prediction isnt designed to capture.

Yet the family remains interested. They want to do whatever they can to have a healthy kid. Keefe says hes so far supporting their choice, but he is concerned by all that it implies. There is potential psychological harm to the kid, he says. God forbid the kids ends up with autism after spending this money.

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The world's first Gattaca baby tests are finally here - MIT Technology Review

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A short guide to regulation for disruptive technologies – Lexology

November 14th, 2019 4:51 pm

Introduction

Regulation, by necessity, introduces rigidity to otherwise flexible processes. Done proportionately, this can be an efficient societal device for preventing harm. At the same time, inherent regulatory rigidity creates particular challenges when the nature of the regulatory target changes quickly or unexpectedly.

Disruptive technologies in life sciences - a very dynamic field of activity are a good example of this. Disruptive technologies challenge the way a sector operates, and it is self-evident that (in most cases) this will also have an impact on the relevant normative framework. This effect is most visible in areas which have a direct impact on human life and wellbeing, as these areas are tightly (and often, rather specifically) regulated, and a failure to control a technology appropriately may lead to undesirable outcomes.

The dual purposes of preventing harm through proportionate regulation and maintaining trust in innovation mean that it is all the more important to ensure that regulation is adequately responsive and flexible to react to a disruptive technology. This can be a difficult line to tread, particularly in fields where research and development is also morally or ethically contentious.

We will illustrate the context and challenge of regulating disruptive technologies by discussing two specific case studies: artificial intelligence, and cell and gene therapy. In both cases, we suggest that the current regulatory framework in the UK strikes an appropriate balance between precaution and freedom of research, allowing for innovation subject to strict controls and licensing frameworks. There are, however, numerous challenges which need to be considered and addressed as these technologies advance. Regulators, policy makers and innovators working in this sector must continue to work together to ensure that responsible science is allowed to flourish.

Artificial intelligence

The science of making machines do things that would require intelligence if done by people (Definition of artificial intelligence from the Proposal for the Dartmouth Summer Research Project on Artificial Intelligence, 1955.)

Artificial intelligence (AI) technologies hold the potential to significantly improve health and care, providing faster and more accurate diagnosis, speedier treatments, and facilitating medical breakthroughs through drug discovery.

This is particularly the case in contexts where the pattern-recognition strengths of AI can be deployed to their fullest potential. Tasks such as the correct identification of tumour cells, recognition of areas of concern in medical imaging, and the processing of large amounts of genomic data can be carried out with much greater speed and accuracy by algorithms that learn from previous datasets, and develop their own datasets from which to learn from in the future. The ability to check a patients image or test result against all other available and comparable datasets is, at first glance, far superior to a clinicians ability to make an assessment on the basis of his or her experience.

At the same time, this does give rise to risk. For example, there is an inherent (and proven) risk that an algorithm which learns on the basis of historic human-generated data also takes on the biases that human decision-making has inevitably introduced. So how does regulation play a part in addressing this risk?

The first point to make is that no one body is solely responsible for regulating the adoption of AI technologies in the UK healthcare sector. A number of different regulatory bodies have a remit to oversee aspects of AI, including the Medicines and Healthcare products Regulatory Agency (MHRA) and the Information Commissioners Office (ICO). In addition, there are nonregulatory bodies which also play an important role, including the National Institute for Health and Care Excellence (NICE) and NHSX. However, no one institution has overall responsibility for policing, for example, the prevention of bias in AI algorithms. The most effective way of addressing this risk at present is to avoid exclusively automated decision-making so that the use of AI technologies in the clinical setting will focus instead on assisted decision-making and triage. The application of this approach will come down to individual healthcare payors and providers: in the absence of any direct regulation, it is left to them to decide how best to mitigate risk, and whether and if so how to apply nonbinding codes of conduct, such as the Department of Health and Social Cares code of conduct for data-driven technologies which seek to address the risk.

Reliance on nonbinding codes of conduct as a substitute for regulation may not be ideal and can result in a lack of certainty. Equally, overlapping codes, rules and regulations also pose a risk, for example, as to how NICEs evidence standards framework for digital health technologies interacts with MHRA regulations concerning software as a medical device in relation to clinical evidence. The risk is lack of clarity; the mitigation is raising awareness.

Another challenge arises where regulation designed for a specific purpose is used for a new purpose, for example the application of MHRA regulations designed for traditional medical devices to software incorporating algorithms. A recent state of the nation survey on the use of AI in health and care revealed that half of all software developers were not intending to seek CE mark classification, with the most commonly cited reason being that they did not believe the medical device classification was applicable. It is essential that the sector raises awareness of these requirements, albeit that they are complex and sometimes impenetrable.

One significant area of concern is how existing laws relating to negligence, liability and insurance apply to the clinical use of AI whether in assisting decision-making about a patients treatment, or in the operation of medical devices. Currently, claims are almost always brought against the treating clinician or healthcare provider, but for a clinician using big data analysis as well as his or her own experience, where does the division of responsibility lie? If a patient is injured as a result of a malfunction in an AI-driven device, does liability lie with the manufacturer of the device, the programmer who wrote the code which operates the device, the clinical team, the hospital or all of the above? It remains to be seen whether this will give rise to novel constellations of liability, such as an increase in manufacturers liability or a change in statutory and wider insurance requirements.

One of the major areas of opportunity for AI-based technologies is biomedical research where the strengths of speed and range have huge potential. The extrapolation of the potential of certain compounds against huge databases of similar compounds is commercially powerful. The ability to quickly check clinical trial design against public registries of published results to avoid unnecessary duplication of human-based experimentation is ethically desirable. But as innovators seek to improve drug discovery using AI, it will be important to continue to keep under review laws relating to intellectual property and how they apply to AI-based technologies.

Cell and gene therapy

The area of cell and gene therapy is of particular significance, and great potential, in regenerative medicine. It has seen a decade-long genesis since its inception, and it does not immediately strike one as a field that meets the definition of a disruptive technology. At the same time, however, it provides a good illustration of how a technology may mature for a long time, or be repurposed in an unexpected way, before it becomes disruptive.

The field has come a long way since the first systematic trials in 1989, and by now, there are 17 FDA-approved cell and gene therapy products. Over and beyond technical questions of the safety of the vectors used for the manipulation of cells, there are few remaining ethical and legal issues in relation to somatic cell gene therapy for particularly debilitating conditions (i.e. where the manipulation does not lead to heritable genetic characteristics).

From a regulatory and ethical perspective, however, cell and gene therapy becomes more complex where germline gene therapy is used. The modification of the human germline is subject to significant debate and, in some jurisdictions, strongly prohibitive regulation. The advent of disruptive technologies such as CRISPR/Cas9 prime editing techniques, with their associated precision and purported safety, have already reignited the debate around the prohibition of germline manipulation, with some commentators calling for a relaxation of the regulation while others demand either a global ban or at least a moratorium.

Although the United Kingdom has a reputation of being a liberal jurisdiction for research, it is in fact very tightly regulated and only potentially permissive. UK law reects a compromise: we permit research (including research involving germline gene editing), but we subject such research to strict scrutiny, licensing and oversight, and we criminalise unlicensed research. That being said, the legislation is drafted in such a way as to facilitate a broad variety of research, including (again, potentially) the introduction of novel techniques, and few procedures are prohibited. Overall, this framework helps allay public and political concern about what is often controversial research and provides a degree of protection for researchers operating under a licence, facilitating innovation. Such a robust framework is particularly valuable when it comes to considering how best to address the clinical application of germline genome modication. In circumstances where UK law is comprehensive and clear in its application to gene editing, there is no merit or purpose in a moratorium or further restriction on the use of this technology as some have demanded.

Concluding remarks

The UK has a mature and robust regulatory framework governing research and development in life sciences. We have a successful history in regulating numerous disruptive and controversial new technologies, such as stem cell research, the creation of human-animal hybrids, the clinical use of preimplantation genetics, and mitochondrial donation all testaments to the strength of this framework and its capacity to adapt to accommodate new technologies. This success, however, has been built upon a vital foundation of open and accessible dialogue between innovators, parliamentarians, policy makers and the public, and it is to be hoped that a similar transparency will be maintained in the future. Such dialogue will also ensure that if there are gaps or restrictions in regulation that need to be addressed to avoid stifling innovation, these can be pre-empted.

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Mirza Dinnayi’s Aid to Terror Victims Recognized with Aurora Prize – The Armenian Mirror-Spectator

November 14th, 2019 4:51 pm

In Germany, he became engaged in many Yazidi cultural activities, advocated for the rights of non-Muslim minorities in Iraq, and worked for peace and coexistence. After the fall of the Saddam regime in 2003, when Kurdish leader Jalal Talabani became interim president of Iraq, he invited Dinnayi to become his advisor for non-Muslim minorities. Dinnayi said he stayed there from 2004 to 2005 but it was hard for him to reintegrate into Iraqi society after becoming used to a German lifestyle. More importantly, a civil war had engulfed Baghdad by 2006 and 2007 and death was always around the corner. His family would call every few days to ask what he was doing there.

After this, he became an advisor for some ten years to the Kurdish Regional Government (KRG) in Erbil concerning disputed regions, which include Yazidi, Christian and Turkmen populated areas, claimed by both the KRG and the central government.

Air Bridge Iraq

On August 14, 2007, Dinnayi was in Germany when the extremist Sunni Muslim terror organization al-Qaeda attacked two Yazidi villages near Mosul in Iraq, killing more than 300 people and injuring more than 850 with truck bombs inside the villages and markets. About 60 children were among the latter.

By this point, Dinnayi had studied both medicine and law, and though not a practicing doctor, knew what the needs were. He did fundraising in Germany for the victims and then went as a volunteer to distribute the money and aid. When he saw many children in the hospital who would die soon without help, he posted an appeal in a German newspaper, Neue Osnabrcker Zeitung, asking for hospitals to host some of these child victims. Two hospitals agreed.

The real challenges then began. Dinnayi said that as the children were from villages, they did not have any passports or identification papers, and the age of one child, who had no family left, was not clear. Dinnayi asked the Iraqi government to issue some kind of passport for them. He also spoke with the German ambassador in Iraq, and explained the emergency. As Dinnayi had dealt with him before when he was an advisor to the government, he trusted Dinnayis judgment and agreed to issue visas within days of receiving the passports. This was a big risk for the embassy because, explained Dinnayi, there was no German NGO in Iraq at the time because of the civil war, and no one believed that Dinnayi would return the children to Iraq after treatment.

Within three days, the passports arrived. The problem, Dinnayi said, was that all Baghdad was a ball of fire. A friend at the German embassy requested that Dinnayi stay in the hotel until going to the airport. As a German citizen, if Dinnayi went outside, he might be kidnapped and cause the government problems.

Yet the hotel happened to have been attacked by al-Qaeda three or four weeks prior and activists and politicians staying there had been killed. Half of the hotel had been destroyed and food was not available.

Dinnayi took the passports and visas of the six children in the first group and went to Erbil, from which he planned to take a charter flight to Germany. The German embassy did not allow family members to go.Some of the children receiving treatment in 2007 (photo courtesy Mirza Dinnayi)

He said, So you can imagine, I had a 6-year-old child who cannot speak, barely walking. I had a girl with broken legs in a wheelchair. I had two other boys, also in wheelchairs. So I was alone with those six children.

An ambulance took them to the airport, but after a wait of two hours, they were told that Turkey would not allow the charter flight to pass through its airspace.

Dinnayi said, The children were very upset. The families told me, you brought the children 150 kilometers from Dohuk to the airport. Now they cannot fly what are you doing? Everybody was blaming me. He took the children to a hotel and the next day got them tickets for a flight to Istanbul. There they booked an airport hotel to stay overnight, and a flight to Dsseldorf, Germany, the next day.

The next morning at the Istanbul airport, the person responsible for check-in refused to let the children on because the flight had no medical equipment. Dinnayi had no choice but to ask for the manager and play hardball. He recalled that he said, These six children are victims of the al-Qaeda terror organization. You have a choice. I can call BBC and CNN in Istanbul and tell them that al-Qaeda killed the innocent minorities of Iraq but the Turks are not allowing us go, or you will bring me a piece of paper and I will sign that all that happens to the children is my responsibility. I will not charge anything. I will not ask for any compensation if anything happens. I am responsible alone in person.

This was accepted, and after the children were treated for about two months in Germany, Dinnayi brought them back to Iraq successfully.

At this point, he thought that since there is no German humanitarian organization in Iraq he and his German friends might as well make one. He said, We called it Luftbrcke Irak because of the Luftbrcke Berlin after Second World War, which also provided humanitarian aid via the air. It was formalized as an organization in November 2008 (see https://luftbruecke-irak.de/?lang=en) and it helps terror victims of all creeds and backgrounds.

From 2007 to the present, all funding has been from private donors and friends. The host families in Germany, along with volunteer workers, do not receive any pay. Approximately 150 children from all over Iraq and from all religious communities (Muslim, Christian, Yazidi etc.) have received treatment in all. This does not include work to aid survivors of the 2014 genocide of Yazidis attempted by ISIS.. Mirza Dinnayi with Lamya Haji Bashar at the International Criminal Court, October 14, 2016. Haji Bashar was forced by ISIS into sexual slavery. She escaped in 2016 but was injured by a land mine. Dinnayis Air Bridge Iraq helped her obtain medical treatment and she became an activist for the Yazidis. In recognition of her human rights achievements, she was given the Sakharov Prize of the European Union. (photo courtesy Mirza Dinnayi)

Assistance to Yazidi Victims of ISIS

Dinnayi was in Erbil for his job as advisor to the KRG as Mosul fell under the control of ISIS on June 10, 2014. He was planning to return to Germany for a summer vacation with his family but he called his wife to cancel, declaring that he feared a huge catastrophe would soon occur. Indeed, two months later, the Yazidis in Sinjar were attacked and the entire community displaced. When 325,000 people went to the mountain it was a huge problem due to the lack of water and food.

Dinnayi was engaged in lobbying, meeting every day with diplomats to try to convince the international community to act. The whole mountain area was occupied by ISIS and the safe zone was 150 kilometers away. For this reason, the Iraqi government decided to initiate a humanitarian mission via helicopter from the Kurdish area to bring food and water there and extract vulnerable people to the safe area.

Dinnayi volunteered to fly with the helicopters, he said, because he knew all the areas where the refugees had collected and was in contact with them. Nearly every day he was with the flights, which were being shot at by ISIS. The helicopters were very old Russian Mi-17 models which were supposed to hold 20-25 people and yet each time they picked up 40-50 people.

One day, Dinnayi said, our helicopter crashed because of overload and I broke my leg. Unfortunately. I lost my friend who was the pilot and some of the refugees died. But we were very lucky, because the crash was over the mount.

Dinnayi was in a wheelchair for three months as his leg and broken ribs healed. He came to Germany and then to Geneva only one week after his return to speak at the UN Human Rights Council on the Yazidis, which led to an investigation about the Yazidi Genocide.Mirza Dinnayi receives a medal as Aurora Humanitarian from the 2011 Liberian Nobel Peace Laureate and Aurora Selection Committee member Leymah Gbowee (Aram Arkun photo)

Helping Yazidi Girls and Women

Only 1 weeks later he returned to Iraq and met the first group of girls who had been raped by ISIS. He said, I was ashamed to hear these stories of atrocities, as a man, to hear what happened with those innocent girls of 16, 17 years old. I decided, I said, well, the catastrophe of the Yazidis and the plight of the Yazidi people is so huge that maybe I cannot help them in all the issues, but maybe I can do something for those women and children. And this was the reason that I concentrated my work to help the survivors of ISIS, and the women and children especially, who were sexually abused.

He helped pressure the Yazidi Spiritual Council to accept these women, because in the beginning the Yazidi community itself would not accept them. Fortunately, the Yazidi religious leader or Baba Sheikh accepted these children and women. Yet, Dinnayi realized, there is no medical or psychological aid for these traumatized beings in Iraq. There is only one psychotherapist per every 250,000 people in Iraq, and generally that person has no experience in trauma.

One of the German states, Baden-Wrttemberg, decided to accept up to one thousand of the women and children victimized by ISIS. The Germans asked Dinnayi to lead this project. Dinnayi did this as a volunteer, and his NGO became a partner of the German project. A German team from the government ministry led and decided for the project, but Dinnayi led the receiving commission in Iraq.

Within almost 9 months, 1,100 women and children were resettled through this project. Dinnayi said it was a very, very hard job. He worked 18 hours a day and had to interview all the women and children. He said, I myself was traumatized, because you hear every day 20 stories of rape, and you ask yourself, every time, why did people do that to those innocent women. I saw 9-year-old or 11-year-old girls who were pregnant because they were raped 20 times or 30 times. In the summer of 2015, I was actually at the end. I couldnt sleep. I was crying every day. I came back to my family, to my wife and children living in Germany. And I told them, okay, lets go and take three days vacation.Mirza Dinnayi surrounded by the founders of the Aurora Prize at Yerevans Freedom Square after he was announced as the 2019 Aurora Prize Laureate (Aram Arkun photo)

During those three days, he debated with himself whether he should withdraw from the project and seek psychological treatment himself. At the end, he decided that if he withdrew, the project would collapse because no one could live under such stress. On the other hand, he said that if he continued, it is only helping me, because I see these atrocities and besides that I see that I can help those victims so maybe this will help to heal my trauma. And I was lucky, that I had overcome this trauma. Until now, I have these traumatic ideas.

The project also had various bureaucratic hurdles to be overcome. The Germans required detailed files on each woman, each between 11 and 32 pages, which Dinnayi had to translate. Then most of the husbands of the women had been killed or were missing, so they were not allowed to get passports for the children nor to fly without their husbands.

Dinnayi went to Iraqi civil courts and asked them to issue a temporary guardian certificate for the children, with which passports could be obtained. When children did not have family, Dinnayi ended up being the guardian.

He had no time for anything but work. Dinnayi related, The problem was that I even forgot day and night during the project, because I was sleeping one night in Duhok (my office was in Duhok), and then I went with the beneficiaries to Erbil, almost two hundred kilometers, sleeping one night there; flying in a special charter with 60 or 70 people to Stuttgart, Germany, sleeping one night in Stuttgart, then moving by train to my family, sleeping one night there, then coming back to Erbil because I had to prepare the next mission. So I was during four days in four different placesSo every time, it was like a joke, before I opened my eyes. Am I at home, I shouldnt do a mistake at least in front of my wifewhere is the bathroom?

Dinnayi said, I am so happy when I compare the situation of those women and children who we got to Germany and the children and women who are still in the camps. There is a big difference. The children are very well integrated. They still have this pain. There is an injury inside them that we are not able heal, unfortunately, because you cannot return back to them their pasts, and you cannot bring their relatives back to them, but their lives are secure, they are no longer living in tents.

The girls and women got visas for two years and there were 22 municipalities in the state of Baden-Wrttemberg state which placed those women in special houses. There they had 24-hour translators, a social worker would take care of them, and each family had its privacy. After 2 years they were moved to regular housing units, the children went to school and the women went to treatment. This was the first time in the history of Germany that a state undertook such a project, Dinnayi said, which is why the project is so unique.

After this, the Canadians started another project for resettlement and took a couple of hundred of the women and the Australians did the same. No Americans helped.

Yazidis and ArmeniansThe new Yazidi temple Quba Mere Diwane at Aknalich, Armenia (Aram Arkun photo)

Dinnayi speaks often about the special relationship between Armenians and Yazidis.

The Armenian parliament and now the Aurora Prize have provided special recognition to the Yazidi Genocide. He said, This is the first time that the Yazidi were accepted and we are so lucky that Armenia, especially the grandchildren of a previous genocide 100 years ago, recognized this genocide. Furthermore, he said, Through the establishment of this forum and this prize Armenia became one of the greatest nations, because they are in solidarity with the victims of genocide.

In general, he said, The Yazidi community in Armenia is a well-integrated community in Armenia. He noted the recent building of a new Yazidi temple in Armenia and contrasted that to the situation in Iraq. If a temple would be destroyed in Iraq, there would be no possibility of rebuilding it, as Yazidis are treated as infidels.

He said, I think we share a cultural heritage together, but unfortunately we also share a history of pain. Aside from culture, Dinnayi has found that there are even close genetic connections between Yazidis and Armenians. He said, I did a DNA investigation with the Family Tree DNA laboratory in the US on some 30 Yazidis from Iraq and the nearest population to the Yazidis was the Armenians. In my family tree, I have more than 100 matches of which many, many are Armenian; 75 percent of my matches were from Armenia and Asia Minor.

The Future

Prior to the Aurora Prize, Dinnayi had another small humanitarian project in Iraq, but at present he said he was mainly working for the recognition of the Yazidi Genocide in Europe, especially with the European Parliament. The latter has passed various resolutions, but what he wants, he said, is to have a special tribunal, either a hybrid tribunal for the crimes of ISIS concerning the Yazidi Genocide, or an internationalized Iraqi tribunal to bring those ISIS fighters to justice and try them according to the international conventions about genocide. While many ISIS fighters are in prisons, they are being tried according to the Iraqi anti-terror law or the Iraqi penal code and not in connection with genocide or international crimes in general. Dinnayi said that the Iraqi penal code is a jokeit is very easy for rapists, for example, to have impunity and overturn any punishment due to a provision allowing this if a certificate of marriage is presented afterwards.

Dinnayi noted that many countries, among them Armenia, symbolically recognized through their parliaments the Yazidi Genocide, which he said is very good and important. He added, We know that this challenge will take a long time. We know about the Armenian Genocide, that it took 100 years until some countries said yes, while the perpetrators until now say no, this was not a genocideSo you see how difficult a situation it is.

He is also working to persuade other countries to accept more women and more victims, though there are no new projects in this vein so far. In the past five years, Dinnayi said, little has changed. Around 80 percent of the Yazidis from Sinjar remain refugees or internally displaced persons in the camps in Kurdistan or outside of Iraq. The people refuse to return to their villages, he said, while those who remain seek an opportunity to leave.

Among the problems is the corruption of the current Iraqi government. It took no steps towards transitional justice and reconciliation. The future, not only for the Yazidis but also the Christians and Mandaeans, is bleak, he said, if there is no special zone or a kind of autonomy established in Sinjar, or the Nineveh plain for Christians. Furthermore, although ISIS is not in this area at present, militias and the Iranian-Turkish conflict create instability.

Meanwhile, Dinnayi is afraid that the Turkish invasion of Syria may lead to a big wave of refugees coming to Sinjar. He exclaimed, I hope that the international pressure on President Trump, on the Europeans, will put enough pressure on Mr. Erdogan to stop this invasion, because it is against humanity, it is against international law. All the Yazidis meanwhile have been deported or displaced from places like Afrin, Syria, over the last five years. There used to be around 35,000 Yazidis there. Some were forcibly converted to Islam.

Going forward, Dinnayi has no intention to slow down.

Dinnayi will continue his work, despite paying a heavy personal price, including health issues. He declared, If you start humanitarian work, you will be part of this humanitarian family and you cannot stop any more. Because you are in direct touch with the victims, with the people in need, with the vulnerable children women, men, and if you stop for one minute, you will feel guilty and you cannot stop more. This was the reason [I continue my work]. I was not expecting to get a prize from any people.

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Global Carrier Screening Market to reach $2.93 billion by 2029 according to a new research report – WhaTech – WhaTech

November 14th, 2019 4:51 pm

The global carrier screening market was valued at $846.9 million in 2018 and is estimated to grow over $2.93 billion by 2029.

According to a new market intelligence report by BIS Research, titled Global Carrier Screening Market- Analysis and Forecast, 2019-2029, the global carrier screening market was valued at $846.9 million in 2018, and is estimated to grow over $2.93 billion by 2029. The global carrier screening market is expected to grow at a compound annual growth rate (CAGR) of 11.59% during the forecast period from 2019 to 2029.

The development of the market is aided by the impressive growth in the field of non-invasive prenatal testing (NIPT), genetic testing, and precision medicine.

Browse 16 Market Data Tables and 142 Figures spread through 201 Pages and in-depth TOC on "Global Carrier Screening Market"

Genetic diseases are the leading cause of the infant death, accounting for approximately 20% of annual infant mortality in the U.S. Advancements in the technological platforms in the genomic medicine have made possible low cost, pan-ethnic expanded carrier screening, enabling obstetric care providers to offer screening for over 100 recessive genetic disorders.

However, the rapid integration of use of genomic medicine into routine obstetric practices has eventually raised concerns about the implementation of carrier testing.

Technological and other advancements over the past decade have led to the discovery of thousands of genes that are associated with autosomal and X-linked recessive Mendelian disorders. Recent improvements in assessing the individual variants in the human genome, generally offer the possibility of testing populations for all known severe recessive genetic disorders.

For decades, general population carrier screening was based on the clinical validity and the utility to direct services based on ethnicity, social factors or race that may lead to particular conditions being more common in a particular group. With advancement in genetic knowledge and technologies, carrier screening for disorders such as cystic fibrosis has now become a part of primary care.

BIS Research Report: bisresearch.com/industrarket.html

With genetic screening, arises several important legal issues such as insurance and employment discrimination, confidentiality, and informed consent for both testing and treatment. Approximately, seven states have laws that penalize providers for violating a patients privacy regarding the genetic information of patients and 27 states require consent for disclosure of genetic information to the third parties.

According to Wahid Khan, Principal Analyst at BIS Research North America is the leading contributor to the global carrier screening market and is noticed to be contributing more than 51.82% of the global market value. However, Asia-Pacific is expected to grow at an impressive CAGR of 15.97% during the forecast period from 2019 to 2029.

Currently, the Asia-Pacific market is estimated to contribute approximately 15.11% of total global market value.

Research Highlights:

Report: bisresearch.com/requeste=download

This market intelligence report provides a multidimensional view of the global carrier screening market in terms of market size and growth potential. This research report aims at answering various aspects of the global carrier screening market with the help of key factors driving the market, threats that can possibly inhibit the overall market growth, and the current growth opportunities that are going to shape the future trajectory of the market expansion.

Furthermore, the competitive landscape chapter in the report explicates the competitive nature of the global market and enables the reader to get acquainted with the recent market activities, such as product launches, regulatory clearance, and certifications, partnerships, collaborations, business expansions as well as mergers and acquisitions. The research report provides a comprehensive analysis of the product sales and manufacturers and trend analysis by segment and demand analysis by geographical region.

This report is a meticulous compilation of research on more than 30 players in the market ecosystem and draws upon insights from in-depth interviews with the key opinion leaders of more than 20 leading companies, market participants, and vendors. The report also profiles 15 key companies, namely, Illumina, Inc., Myriad Genetics, Inc., Thermo Fisher Scientific Inc., Laboratory Corporation of America Holdings, Quest Diagnostics, Natera, Inc., Invitae Corporation, Eurofins Scientific, GenMark Diagnostics, 23and Me, Inc., Sema4, BGI, Centogene AG, Pathway Genomics, and Gene By Gene.

Key Questions Answered in the Report:

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Global Carrier Screening Market to reach $2.93 billion by 2029 according to a new research report - WhaTech - WhaTech

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Treatment-free Remission at Heart of New CML Study – AJMC.com Managed Markets Network

November 14th, 2019 4:48 pm

Maggie L. Shaw

Researchers aim to eliminate leukemia stem cells safely.

Its like removing the tree, but leaving the roots that can sprout new shoots, they said in a statement.

Because stem cells are responsible for cell self-renewal and differentiation, Lacorazza and his team set out to better understand the LSC self-renewal process, hoping to pinpoint possible new options to target that activity, prevent relapses, and lead to treatment-free remission. Their results appeared recently in Blood, the official publication of the American Society of Hematology.

Knowing that Krppel-like factor 4 (KLF4) plays an essential part in carcinogenesisalthough it has also been shown to have antitumor activitythe study investigators removed KLF4 to see if it was necessary also for LSCs to survive. The result? Loss of LSC/progenitor cells and increased levels of tyrosine-(Y)-phosphorylation-regulated kinase 2 (DYRK2) protein.

According to the authors, A major change in the absence of KLF4 was an increase in the production of kinase DYRK2, an enzyme involved in protein stability, cell cycle control, and apoptosis.

Stabilizing levels of this protein, then, could be a possible solution. To do this, they inhibited ubiquitin E3 ligase SIAH2 by introducing menadione (vitamin K3), as this has been shown to stimulate cell death in human CML stem/progenitor cells and increase levels of DYRK2. The drawback to this approach is that vitamin K3 can be toxic. To remedy this, the authors suggest SIAH2 inhibitors with lower hematological toxicity, evaluating the safety of that inhibition, and developing alternatives to activating DYRK2 in CML LSCs. In other words, stabilize or increase DYRK2 levels to inhibit LSCs.

Having set out to better their understanding of LSC renewal for new inroads to treatment-free survival and relapse prevention, Lacarozza and colleagues did just that. They identified the DYRK2 checkpoint in LSC/progenitor cell survival and self-renewal, showing there are 2 ways to increase its levels: (1) remove the Klf4 gene or (2) inhibit the ubiquitin ligase SIAH2 pharmacologically.

At present, they continue to search for ways to accomplish this that will not harm patients with CML, who today must take TKIs for life. We envision that targeting the bulk of leukemia with tyrosine kinase inhibitors plus a new drug that targets the stem cells might be a future strategy for patients to reach drug-free remission.

Reference

Park CS, Lewis A, Chen T, et al. KLF4 represses DYRK2 inhibition of self-renewal and survival through c-Myc and p53 in leukemia stem/progenitor cells [published online September 12, 2019]. Blood. doi: 10.1182/blood.2018875922.

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INDIA Indian doctor: Medical innovation should not try to replace the Creator – AsiaNews

November 14th, 2019 4:48 pm

Dr Pascoal Carvalho addressed the 21st convention of Indias Catholic nurses in Mumbai. He spoke about the ethical aspects of genetic engineering, citing the doctrine of the Church towards human cloning and stem cells. Respect for human dignity must prevail from conception to natural death.

Mumbai (AsiaNews) Medical innovation, which increasingly uses modern technologies to improve life, should not attempt to artificially replicate creation, said Dr Pascoal Carvalho, a doctor from Mumbai and a member of the Pontifical Academy for Life, speaking at the 21st convention of Catholic nurses (8-10 November).

In his address on 9 November, he referred to therapeutic cloning, stem cells and modified human DNA before an audience of more than 200 Catholic health workers.

"[W]e can rest assured in the wisdom of the Church," he said, because for her, The dignity of a person must be recognized in every human being from conception to natural death.

Some areas of medical research that raise serious moral and ethical questions touch stem cells, embryos and DNA.

In his view, today There is a growing threat of overestimating genetic modification techniques and underestimating the repercussions of cloning and human gene therapy.

On the one hand, we have the positive results of therapeutic cloning aimed at organ and tissues reconstructed in laboratory for transplanting into patients to reduce the risk of rejection; on the other, reproductive cloning, like in the case of Dolly the sheep, seeks to reproduce living beings.

He warns against research that leads to alterations in an organisms DNA, like the famous case of the Chinese scientist who in 2018 said that he had created two twins in the laboratory immune to the HIV virus. This kind of experiment can reduce life expectancy and increase susceptibility to other, and perhaps more common, diseases.

The doctor cites the Dignitas Personae, which defines any attempt at human cloning as unacceptable, because it represents a serious offense to the dignity of the person and fundamental equality between men.

As for therapeutic cloning, To create embryos with the intention of destroying them, even with the intention of helping the sick, is completely incompatible with human dignity, because it makes the existence of a human being at the embryonic stage nothing more than a means to be used and destroyed. It is gravely immoral to sacrifice a human life for therapeutic ends.

Citing the doctrine of the Church, Dr Carvalho stresses the importance of the method with which stem cells are taken. In his view, Methods which do not cause serious harm to the subject from whom the stem cells are taken are to be considered licit.

This is generally the case when tissues are taken from: a) an adult organism; b) the blood of the umbilical cord at the time of birth; c) foetuses who have died of natural causes.

Overall, the doctor believes that modern gene technologies raise new moral questions, whilst attempts to create a new type of human being contains an ideological element in which man tries to take the place of his Creator.

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‘Dr. Death’ and ‘Bad Batch’ Host Laura Beil on the Future of Podcasts – ELLE.com

November 14th, 2019 4:48 pm

Laura Beil was skeptical when Wondery called her two years ago. The sensationalistic podcast hitmaker behind Dirty John needed a host for its new series about Christopher Duntsch, the infamous Dallas neurosurgeon accused of maiming his patients. Beil, a veteran Dallas Morning News medical reporter, hadn't listened to a true crime podcast in full, let alone reported one. She'd certainly never heard of Wondery. "I said, 'I'm a print journalist,'" she tells ELLE.com. "Why are you calling me?" With some hesitation, she agreed to do it. Today, she's grateful she did.

Since airing last September, Dr. Death has been downloaded more than 50 million times and ordered as a television series. On the heels of its massive success, Wondery greenlit a second Beil-led podcast, Bad Batch, now available on Apple Podcasts and Spotify. In the six-part investigative series, she takes listeners through the crazy, complicated world of stem cell medical treatment. Like Dr. Death, there's a narrative arc (corrupt system, suspicious CEO, unsuspecting victims); unlike Dr. Death, she says, it serves a real purpose. "The chances of you coming across a horrible neurosurgeon are pretty slim," she says, "but the chances of you or someone you love wanting to spend a bunch of money on stem cells because you're promised a miracle cure? That's much higher. This has a greater chance of having an impact on listeners."

Bad Batch has already garnered 3 million listeners since it debuted three weeks ago, and is now the fourth most popular show on Apple podcasts, ahead of rival My Favorite Murder.

On the phone, Beil and I discuss her transition to audio from print journalism, the future of true crime content in a frenetic digital age, and her secret sauce to producing a hit podcast.

Apparently a Dirty John listener had emailed Wondery saying, "Hey, have you heard of Christopher Duntsch?" They wanted a journalist who had knowledge of the healthcare system in Dallas, where Duntsch practiced, to look into him, and that's a pretty short list. When they called, I hadn't even heard of Wondery. But I decided to take a chance on it.

Journalism is journalism. There are some things I had to get used to, of course. For example, in print journalism, if you need something else, you can go back and get it from a source. You'll email or you'll text somebody to follow up as you find out you need more details. With audio, you just have one shot. It's a lot harder to go back and reinterview someone. You have to make the one interview really count, and that means asking the same question over and over again in a different way, to get details that draw people out. It's something that I'm still learning how to do, frankly.

The feedback about my voice has been all over the place. I didn't get so much with Dr. Death, but for Bad Batch I am. Listeners will say, "Oh, the narrator's too dramatic." And then someone else will say, "Oh, the narrator's too robotic." It's all conflicting. My favorite bit of feedback was from a listener who said they preferred the host of Dr. Death to Bad Batch.

I don't see true crime being dethroned anytime soon. It will always dominate, because people love it. That said, Bad Batch doesn't necessarily fit in the true crime box. There wasn't really a crime, and nobody died. What you need, just like in a print piece, is a good central narrative to hang your story off. The stem cell story is complicated, because you can't just say it's all a big con job. There's legitimate stem cell research going on. The business is growing so much and most of the information about it is coming from people trying to sell it. There's a lot to explore and explain.

In this business, so much is contracting, like newspapers, so it's nice to see one aspect of journalism that's expanding. To see more demand for audio journalism is heartening. It's reviving a lot of the long-form storytelling that's been cut in other places. Dr. Death had 50 million downloads. The same story was told in print on ProPublica, which is a hugely popular website, and yet the response from our audio was so much greater. A lot of things that we're told people want nowadaysshorter stories that are more clickable and scannablewell, you can't do that with a podcast. I can't explain it, but people can't get enough of podcasts.

I do enjoy doing the audio stuff, but I have to say, in my heart of hearts, I'm still a print writer. If I had to give up one or the other, I'd give up the audio.

[Laughs] With two number one podcasts out in a row, Wondery is like, "Do you have anything else?" After Dr. Death, I had so many emails from people saying, "Here's another horrible doctor to look into." It was depressing. I don't want to do another bad doctor story, I want to do something completely different. I want it to be the right story. It'll be something medical of course.

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The Heart of the Matter: Leveraging Advances in Cardiac Biology to Innovate Gene-Based Therapies for Heart Failure – Physician’s Weekly

November 14th, 2019 4:48 pm

Heart failure (HF) is the most frequent cardiovascular diagnosis and exacts significant health and financial costs around the globe. It is estimated that at least 26 million people worldwide are living with HF, including nearly 6 million in the United States.1, 2 One in nine U.S. deaths in 2009 included heart failure as a contributing cause and about 50 percent of people in the U.S. with HF die within five years of diagnosis.2 The annual cost of HF-related healthcare services, medication and missed days of work is estimated at $40 billion in the United States and $108 billion globally.3, 4 Quality of life in HF patients is frequently worse than many other chronic diseases and comorbidities are common.5-7 The challenges of HF are expected to grow, as it is estimated that more than 8 million people in the United States alone will have HF by 2030.2 Current therapies improve quality of life in the short-term and have improved long-term survival but a significant number of patients have Class 3 HF despite optimal medical and device therapy. These patients have limited treatment options beyond heart transplant and left ventricular assist devices (LVAD). New therapeutic approaches that address the underlying causes of HF are needed to improve patient outcomes.

Heart failure is a complex disease process and multiple pathways contribute to its development and progression. Myocardial ischemia is frequently an issue in both ischemic and non-ischemic cardiomyopathy as well as HF with preserved and/or reduced ejection fraction. Myocardial ischemia results in insufficient oxygen and nutrients and leads to hypoxia, cardiomyocyte and fibrosis, which all contribute to the progression of heart failure. More effective angiogenesis may prevent this progression. Cell homing also plays a critical role, as injured cardiac tissue secretes factors that lead to the recruitment, proliferation, migration and differentiation of progenitor cells that can help repair tissue damage. Stromal cell-derived factor (SDF)-1 has been shown to play an important role in cardiac repair by mediating cell homing.10 Mitochondrial energy generation is also impaired in HF, leading to decreased contractility and adverse changes to cardiac architecture.11 Scar tissue formed in response to cardiomyocyte injury or death can compromise the hearts mechanical strength or electrical signaling results in myocardial infarction. Inflammatory responses to cardiac tissue damage can promote inappropriate and chronic inflammation and the expression of pro-inflammatory molecules that lead to pathologic changes to cardiac architecture.12, 13

These pathways offer a variety of potential new targets for therapeutic intervention to prevent the development and progression of HF. This opens the door to the development of novel therapies that address the underlying molecular and cellular causes of disease rather than treating HF symptoms alone.

After decades of development, gene-based therapies are now validated therapeutic modalities for the treatment of inherited retinal disorders and cancer and are undergoing clinical evaluation in a variety of inherited, acute and chronic diseases. Nearly two dozen single gene-based therapies for HF have been evaluated in clinical trials.14 Genes evaluated as monogenic gene therapy for HF in clinical trials include vascular endothelial growth factor (VEGF) and fibroblast growth factor type 4 (FGF4) to promote angiogenesis; adenylyl cyclase type 6 (AC6) and sarco/endoplasmic reticulum Ca2+-ATPase type 2 (SERCA2) to improve cardiac calcium homeostasis, which plays a critical role in the contraction and relaxation of heart muscle; and stromal cell-derived factor-1 (SDF-1) to improve cell homing and promote cardiac tissue repair. Late-stage trials of single gene therapies have yielded conflicting results, raising the question as to whether positively impacting a single pathway can be sufficient to overcome detrimental activity of other pathways that contribute to the development and progression of HF. Other potential limitations to HF therapies evaluated in clinical trials to date include the method of delivery, dose and the potency of vectors and gene products.

Given the multiple molecular and cellular pathways active in HF, a multi-gene approach to HF gene therapy may be needed. Simultaneously delivering multiple genes that target diverse HF-related pathways has the potential to improve cardiac biology and function. A triple gene therapy approach (INXN-4001, Triple-Gene LLC, a majority-owned subsidiary of Intrexon Corporation) is currently in clinical development, with each of the genes targeting a specific HF-related pathway. The investigational drug candidate INXN4001 vector expresses: the S100A1 gene product, which regulates calcium-controlled networks and modulates contractility, excitability, maintenance of cellular metabolism and survival; SDF-1a which recruits stem cells, inhibits apoptosis and supports new blood vessel formation; and VEGF-165 which initiates new vessel formation, endothelial cell migration/activation, stem cell recruitment and tissue regeneration. The hypothesis is that the simultaneous delivery of multiple genes in a single vector would more effectively improve multiple aspects of cardiac function compared with single gene therapy. It is delivered by retrograde coronary sinus infusion of a triple effector plasmid designed with a self-cleaving linker to constitutively express human S100A1, SDF-1a and VEGF 165. This route is designed to allow for delivery of a dose to the ventricle which may help achieve improved therapeutic effect.

Several preclinical studies have set the foundation on which to advance a triple gene therapy for HF into the clinic.15-17 Using in vitro studies, transfecting cells derived from patients with dilated cardiomyopathy with a triple gene combination demonstrated improvement in contraction rate and duration, to the levels demonstrated by the control cells and did not result in increased cell death compared to controls.15 Studies in an Adriamycin-induced cardiomyopathy rodent model demonstrated triple gene therapy increased fractional shortening and myocardial wall thickness compared to controls.16 In addition, retrograde coronary sinus infusion of INXN-4001 in a porcine model of ischemic HF resulted in a cardiac-specific biodistribution profile.17

A Phase 1 clinical study has been initiated to evaluate the safety of a single dose of triple gene therapy in stable patients implanted with a LVAD for mechanical support of end-stage HF. An independent Data and Safety Monitoring Board agreed to proceeding to the second cohort following review of the data from the first cohort in the multi-site study.18 The study is ongoing and final results will help to inform our understanding of the potential that multi-gene therapy may play in the treatment of HF.

The recent FDA approvals of gene therapies for an inherited retinal disease and cancer are evidence that gene therapy is a valid therapeutic strategy. Realizing the potential of gene therapy in HF will require appropriately designed clinical trials, but several interesting approaches currently in development may prove to be effective. The results of the initial investigational drug INXN-4001 Phase 1 trial should provide insight into the safety of combining S100A1, SDF-1a and VEGF-165. Evaluation of additional multi-gene combinations will also be important for understanding which targeted pathways yield the greatest effects with respect to relevant clinical endpoints. Continued refinement and optimization of vector design and delivery methods will also be important for advancing further HF gene therapies from bench to bedside.

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The Heart of the Matter: Leveraging Advances in Cardiac Biology to Innovate Gene-Based Therapies for Heart Failure - Physician's Weekly

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These Israeli Companies Are Finding Innovative Ways To Improve Diabetes Care, Treatment | Health News – NoCamels – Israeli Innovation News

November 14th, 2019 4:48 pm

Diabetes is a major public health crisis that is approaching epidemic proportions around the globe and growing at an alarming rate. According to the International Diabetes Federation (IDF), over 425 million adults are currently living with diabetes. That number is expected to rise to 629 million by 2045.

The disorder, which occurs when the pancreas does not produce enough insulin (the hormone that regulates blood sugar) or when the body cannot use its produced insulin, has become so deadly, that the International Diabetes Federation said it was responsible for four million deaths in 2017 alone. The World Health Organization estimated diabetes to be the seventh leading cause of death globally in 2016.

Over 350 million people are at risk of developing Type 2 diabetes, when the body becomes to resistant to insulin due to lifestyle facts such as excess weight gain, while more than 1.1 million children are living with Type 1 diabetes, the disorder that occurs when the bodys immune system attacks cells of the pancreas that produce insulin, according to the International Diabetes Federation.

World Diabetes Day, marked annually on November 14 to honor the co-discoverer of insulin Canadian Dr. Frederick Banting, born on that day in 1891, aims to raise awareness of the impact of this disease while promoting its management, care, and prevention. For the past two years (including in 2019,) the theme of World Diabetes Day has been The Family and Diabetes promoting the familys role in awareness and education of the disorder.

To mark the day, NoCamels is taking a closer look at Israels role in advancing diabetes research and treatment as well as some of the companies with cutting-edge technology that stand out in the field.

Dr. Irit Yaniv, a general manager at Accelmed Ventures II, a new $100 million venture early-stage health tech fund for medical device and digital health startups and co-founder and chairperson of Type 2 diabetes medical device startup Digma Medical, calls diabetes the most dangerous global epidemic. Israel is no exception, she says, citing half a million people living with the disease in the country.

Additionally, about 300,000 are prediabetic, a condition manifesting as borderline high blood sugar levels and an increased risk to progress to diabetes Type 2 within a few years, she explains.

Dr. Yaniv says life science entrepreneurs address the challenges presented by the epidemic in a number of ways, including creating companies and projects aimed at reducing the risk of developing the diseases, development of novel drug delivery methods for diabetes drugs, and unique medical devices for managing the disorder.

SEE ALSO: 6 Israeli Companies At The Forefront Of Diabetes Care, Prevention, and Treatment

There are a few interesting examples such as oral insulin and nasal insulin delivery systems that were developed in Israel, Yaniv says. A recent success story is Nutrino, [an Israeli] software company for the management of diabetes that was acquired by Medtronic.

Treatment for Type 2 diabetes has been focused on lifestyle changes and pharmacologic solutions. Both have challenges including patient compliance and adverse effects such as weight gain, hypoglycemia, and other reactions, Yaniv says. More than 50 percent of the patients arent able to control the disease, even with combination therapeutics.

The medical device industry has made many efforts in recent years to address this need, Yaniv says, Companies such as GI Dynamics have paved the way for new therapeutic alternatives. Digma Medical has demonstrated initial positive clinical data with its unique duodenal ablation system.

Yaniv founded Digma Medical in 2013 with Ilan Ben Oren. Backed by leading venture capital firms such as Arkin Holdings and Peregrine Ventures, the company is dedicated to the development of its DiaGone device to treat insulin resistance. DiaGone is an endoscopic, disposable device, that uses innovative laser technology to treat the duodenum, a segment of the small intestine, without an implant. The Duodenal Glycemic Control procedure is a one-time 30 minute GI procedure, in which the gastroenterologist uses DiaGone to treat the duodenum for restoring the natural ability of the body to control glucose levels, said to provide long term remission from Type 2 diabetes and other metabolic syndrome-related diseases.

For Rami Epstein, who assumed the role of CEO at stem cell company Kadimastem in May 2019, a need exists to find a better solution for the management of diabetes in order to decrease the morbidity, mortality, and costs linked to it and its medical-related care, he tells NoCamels. This method is more than just controlling insulin levels in the body. It is controlling the dose of insulin administered to the body.

All Type 1 diabetes and 30 percent of Type 2 diabetes patients depend on the daily administration of insulin in order to control their glucose levels in the blood. This is not ideal since patients have to calculate insulin dosages and take into account meal times and portions, physical activity, and other parameters, he explains. Unfortunately, many patients do not manage to stabilize their blood glucose levels properly, thereby risking complications that arise from episodes of hypoglycemia or hyperglycemia,

Through Kadimastems groundbreaking stem-cell therapy technology, developed by the companys chief scientist Professor Michel Revel at the Weizmann Institute of Science and used as the basis for the companys founding in 2009, the Ness Ziona-based firm has developed and manufactured an off-the-shelf cell product for the treatment of insulin-dependent diabetes based on its proprietary tech platform. The tech platform has been used to treat multiple diseases, including ALS, through the expansion and differentiation of Human Embryonic Stem Cells (hESCs) into clinical-grade functional cells.

The product, called IsletRx, is currently in pre-clinical trials. Its goal is to free patients from continuous monitoring of blood sugar levels and repeated insulin injections. The drug contains an endless source of pancreatic functional islet cells, which produce and secrete insulin and glucagon in response to external glucose levels.

Meanwhile, Tel Aviv-based clinical-stage pharmaceutical company Oramed, which is focused on the development of oral drug delivery systems, announced this week that Phase IIb trial evaluating the efficacy and safety of its lead oral insulin candidate, ORMD-0801, has had positive results.

The study was a 90-day, double-blind, randomized, multi-center trial designed to evaluate the safety and efficacy of ORMD-0801 as a treatment for patients with type 2 diabetes, Oramed said in a statement. The primary efficacy endpoint was a reduction in Hemoglobin A1c (A1C, also known as HbA1c, is a key clinical measure of blood glucose control) at Week 12, with no weight gain.

Israeli scientists are taking diabetes management solutions one step further and adding machine learning and AI to the mix. DreaMed Diabetes, a medical tech startup founded in 2014 to develop these types of personalized solutions, announced in September that it had received clearance from the US Food and Drug Administration (FDA) as well as a CE Mark for its DreaMed Advisor Pro, an AI-based insulin dosing decision support software. The software is for patients with Type 1 diabetes using insulin pump therapy with continuous glucose sensors and blood glucose meters (BGMs)

The decision-support platform uses proprietary algorithms to process data from a range of connected devices, including insulin pumps and self-management glucometers. The data is then analyzed to provide an optimized insulin dosing treatment plan to maintain a balanced glucose level.

SEE ALSO: Israeli AI Startup Can Predict Which Diabetes Patients Will develop Kidney Disease

This clinical and technological advance leverages the power of artificial intelligence to optimize insulin administration in a streamlined and cost-effective manner, the company said in a statement.

This year I am more optimistic that a change will happen in how we manage diabetes. We see more technology adopted in all markets, more sharing of data between patients providers and industry all for the benefit of offering better care for patients, the companys CEO Eran Atlas tells NoCamels in an email. DreaMed is happy to be part of the leaders in this effort, by offering a unique artificial intelligence technology that can analyze data and recommend in only a few seconds how to optimize the technology. With such technology, the proliferation of expert care can be achieved even in emerging markets.

Another key player operating in artificial intelligence in the diabetes management market is Sweetch. Founded in 2013, Sweetch offers an AI-based platform that aims to identify those at high risk of developing Type 2 diabetes. The company calls itself the first AI-powered therapeutics solution to help people with the disorder and comes with a mobile app and a wireless Bluetooth-connected scale. The early prevention platform announced last year that it will partner with US-based integrated healthcare system WellSpan Health and provide its app to 15,000 employees, including 200 primary care and specialty physicians, as well as advanced practice clinicians in central Pennsylvania and northern Maryland.

In 2016, the startup raised $3.5 million in a Series A round led by equity crowdfunding platform OurCrowd and Philips.

Diabetes can cause circulation problems and related conditions such as peripheral arterial disease (PAD) which occurs when plaque builds up in the arteries and reduces blood flow to the limbs. For some patients, high levels of blood glucose can damage blood vessels and cause plaque build-up affecting healthy blood flow.

The northern Israel-based startupElastiMedhas developed a wearable medical device that doesnt treat diabetes patients directly but can help the patient improve his or her circulation.

It cant treat the disease directly, but it can treat some of its symptoms, says Elastimed CEO and founder Omer Zelka.

Compression socks improve circulation by squeezing the foot and calf muscles, which straightens out the vein walls to a better working state, says Advanced Tissue, the leading wound care supply provider. Compression therapy is particularly beneficial for diabetes patients because they improve circulation in a non-invasive manner, helping to maintain the right amount of pressure in feet and legs.

ElastiMeds sock uses battery-operated technology to activate a smart material that compresses and massage the legs to stimulate circulation. The pulses mimic contractions in the calf muscles that in turn increase blood flow.

The sock provides patients with a comfortable, easy-to-wear, highly effective, and cost-effective treatment option to prevent symptoms such as swelling, blood clots, leg ulcers and reduce athletes recovery time.

ElastiMed is currently finishing an ongoing clinical study to demonstrate the safety and the feasibility of this device and its ability to increase venous blood flow. The study is led by Dr. Vered Shuster Ben-Yosef, R&D Lab Manager of the company and is currently taking place at Hillel Yaffe Medical Center in Hadera. The company currently has a working prototype and aims to get the product on the market in early 2021.

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Heartbreaking moment dad meets the woman who saved his life – he feared his son would grow up without a father – Manchester Evening News

November 14th, 2019 4:48 pm

This is the tear-jerking moment a dad shares a hug with the woman who saved his life.

James O'Donnell, from Burnage, feared the worst after being diagnosed with a blood disorder similar to leukaemia in 2016.

Usual treatments were failing and James was undergoing a blood transfusion every week while battling constant infections, the Liverpool Echo reports.

James was running out of options and despaired at the pain his death could cause his eight-year-old son, Harrison.

But in a stunning stroke of fortune, his saviour was only the other side of the M62 - LiverpoolCouncil admin worker Leah McDougall.

The 29-year-old mum, from Bootle, had taken the time to sign up to the register of potential stem cell donors on her lunch break at a pop-up stall, organised by blood cancer charity DKSM, the previous year.

James, who despite his Manc heritage is an avid Liverpool FC fan, told staff at the charity that he would be up for meeting his donor, who could have been anyone from a number of European countries using the register.

James, along with his wife Andrea and young Harrison, got the chance to meet Leah for the first time at a DKSM charity gala in London on Wednesday last week (November 6).

James, who says he finally feels like himself after a long period of illness, told the ECHO: "I was just getting chest infections and water infections all the time.

"I am quite a healthy person, and I was in good shape and I knew I should not be getting ill all the time."

He said after a few weeks of tests his was invited to take a bone marrow biopsy and was told the devastating news on his 40th birthday.

The disease meant James' bone marrow was not producing enough white blood cells, but doctors told him a treatment called anti-thymocite globulin (ATG) had a "75% chance" of success.

However, when that failed, fear and doubt began to creep in.

He said: "We are always saying I would get through this, we were thinking I would get better. But I started to think it's not happening, it's not going to be for me, this.

"I thought, I have been good in life, I need some luck. We were having a really hard time.

"My son was four or five then, and it was hard for him having a dad going from playing football with him to being in hospital."

Eventually doctors revealed the only option was for James to have a bone marrow transplant.

The O'Donnell's went through further disappointment when tests on his three siblings revealed none were a match, so the waiting game to find a suitable donor began.

But on a March day in 2017, he got a call to say: "We have got a perfect match, a 10 out of 10."

The operation was a success and after four weeks doctors told James the new bone marrow cells were taking effect.

He said: "We were so lucky to find a donor only about 25 miles away. Some people never find one and we had one on our doorstep."

The powerful emotion of meeting Leah last week is summed up by James: "It was the second best moment of my life after my son being born.

"What she has done means that I can see my son growing up and that he has a father."

Leah did not hesitate to agree to help a total stranger when she was asked by DKSM.

Describing the moment she met James and his family, she told the ECHO: "We were both speechless. When I walked on stage we were just hugging each other for ages.

"It is weird, we felt like we had known each other for years, I felt like I had known him my whole life.

"It just takes five minutes out of your time to sign up to the register; that's like going to the kitchen to make a drink.

"You just think about the impact it is going to have on someone, it is saving someone's life. I feel lucky to have been able to give something back."

James says his family and Leah are planning to meet up again, possibly at a Liverpool FC game.

He said: "Without her, I wouldn't have a future."

DKSM has urged anyone aged 17-55, and in general good health, to sign up to the register here.

Dr Manos Niklolousis, Haematologist at University Hospital Birmingham NHS Foundation Trust, said:"Blood stem cells can be used to treat a wide range of blood cancers and blood disorders and we urgently need more people to come forward as donors.

"Currently, only 2% of the UK population are registered so matching donors with patients isnt easy within a growing multicultural population.

"Many of those in need are unable to find a sibling match and so rely on the generosity of strangers, and a blood stem cell transplant can be some patients only hope of survival.

"As a doctor who treats people with blood cancer or disorders, it is upsetting to know that some patients could have been saved if only more potential donors were registered and available to donate.

"I look forward to the day when there will be a donor for every patient in need."

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New blood test could lead to better treatment for sepsis – NHS Website

November 14th, 2019 4:48 pm

"Blood test for sepsis could save lives of thousands of children," reports the Mail Online.

Researchers in the US have developed a test for 5 proteins, which they say allows them to identify people with sepsis who have a high, medium or low risk of dying from the condition. They hope this will eventually help doctors identify the best treatments for individual patients.

Sepsis, sometimes called blood poisoning, is when the body's immune system over-reacts to an infection, causing inflammation and damage to the body's own tissues and organs. Anyone with an infection can get sepsis, although it is more common in babies and people over 75.

One of the challenges in treating sepsis is that symptoms can develop and worsen extremely quickly. So, a test that can confirm a diagnosis of sepsis before this happens should help reduce the risk of serious complications and, in some cases, deaths.

The test is still experimental. However, early experiments in mice suggest people at a high risk of dying from sepsis might benefit from higher doses of antibiotics than would normally be given.

Sepsis is a medical emergency. Find out more about the symptoms and what to do if you think someone may have sepsis.

The researchers who contributed to the study came from 16 institutions in the US, mainly children's hospitals. The research was led by Cincinnati Children's Hospital Medical Center. It was funded by grants from the US National Institutes of Health and Cincinnati Children's Hospital. The study was published in the peer-reviewed journal Science Translational Medicine.

The Mail Online did a good job of reporting a complicated study.

The researchers carried out a series of experiments, testing children with sepsis and then experimenting on mice to explore the results further. This is early-stage experimental research that may one day result in better treatment, but there is much more research to be done.

In earlier experiments, researchers had identified 5 proteins created by the body during sepsis, plus a measure of platelets (components in blood that make clots). They hoped the test, called Persevere II, would work to predict who was at highest risk of dying from sepsis.

They used the test on 461 children admitted to hospitals with sepsis, and divided them into 3 groups high, medium and low risk of death within 28 days. They then looked to see how accurately the test predicted what happened to the children. All children were treated as normal for sepsis in their admitting hospital.

The researchers then developed a mouse equivalent of the test, to see whether it would also predict survival in mice given sepsis in a laboratory experiment. They later investigated whether the higher-risk mice had more inflammation or more bacteria than low-risk mice. In 2 further experiments, they tested whether giving the high-risk mice steroids for inflammation, or higher-dose antibiotics to fight bacteria, changed their chances of survival. They also checked to see whether blocking 1 of the 5 proteins produced by the body and measured in the test affected the outcome.

Finally, researchers checked the results of the children with sepsis. They wanted to see if those classified as high risk by the Persevere II test were more likely to have had bacteria in their blood tests.

The researchers found the Persevere II test worked well to identify the risk level of children with sepsis. Almost all of the patients categorised as low risk survived, while just over half (55%) of those categorised as high risk survived. The test correctly identified as high risk 86% of those who did not survive (86% sensitivity) and correctly identified as low risk 69% of those who survived (69% specificity).

The test also worked well when predicting mice at high and low risk. Further experiments on mice showed high-risk mice had more signs of inflammation in the lungs and throughout the body as well as bigger colonies of bacteria from their infection.

Tests also showed that giving high-risk mice the anti-inflammatory drug dexamethasone or placebo made no difference to their survival, but they were more likely to survive if given high-dose antibiotics.

Mice given a drug to block 1 of the proteins used in the test were more likely to survive for 10 days, but the difference was small enough that the results could have come about by chance.

The researchers found that children who had been identified as high risk by the Persevere II test were more likely to have had bacteria cultured from their blood. While this is not a direct measure of the size of the bacteria colony, larger colony sizes are more likely to be able to be cultured after blood tests.

The researchers said: "On the basis of our findings, we suggest that Persevere II might identify a subgroup of children with septic shock [sepsis] who will benefit most from targeted therapeutic drug monitoring to ensure optimal dosing of antibiotics."

Because little is known about why sepsis happens after some infections, and how exactly it develops, it is hard to develop new treatments. Treatments have stayed the same for many years, and sepsis is a life-threatening condition.

This study is the first for many years to make some progress in our understanding of sepsis. It opens up possibilities for research into potential drugs that could be used in the future. This study also suggests ways in which the Persevere II test could be used to identify people at high risk of life-threatening sepsis, so they can be treated quickly and with the most appropriate dose of antibiotics.

However, this is very early-stage research. Just because a treatment works for mice does not mean it will work for humans. Translating results from a species to another, as the researchers do in this study, does not always work. Higher-dose antibiotics have not been tested for children identified as at high risk of life-threatening sepsis. Higher than usual doses could have damaging effects.

The Persevere II test is still being worked on, so is not yet generally available for doctors to use. In a media interview, a researcher said it is about 2 years away from being made available.

Sepsis can be hard to spot. There are lots of possible symptoms.

Find out more about spotting the signs of sepsis.

Analysis by BazianEdited by NHS Website

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Jury Hands Down $42.5M Total Verdict Against Philip Morris and RJR in Retrial Over Smoker’s Cancer Death – CVN News

November 14th, 2019 4:48 pm

Stock image.

Miami, FL R.J. Reynolds and Philip Morris were hit with a $42.5 million total verdict in a retrial over the 1996 cancer death of a Florida smoker. Gloger v. R.J. Reynolds and Philip Morris, 2011-CA-23377.

The award includes $15 million in compensatories awarded Friday, as well as an $11 million punitive award imposed against Philip Morris and $16.5 million in punitives imposed against Reynolds Wednesday for the responsibility jurors found the companies bore for Irene Glogers lung cancer.

Gloger, who had smoked for decades, died at 47, about a year after she quit smoking. Her family contends a tobacco industry-wide conspiracy to hide the dangers of smoking throughout much of the 20th century hooked Gloger to cigarettes and ultimately led to her fatal cancer.

The verdictmore than doubles the $17.5 million jurors awarded in a 2018, CVN-covered trial of the case. That verdict was thrown out in March by Floridas Third District Court of Appeal, which found the trial court had not not properly limited Kenneth Glogers testimony concerning conversations he had with his wifes doctors.

The Gloger case is among thousands that stem from Engle v. Liggett Group Inc., a 1994 Florida state court class-action lawsuit against Philip Morris and other tobacco companies. The state's supreme court ultimately decertified the class, but ruled that so-called Engle progeny cases may be tried individually. Plaintiffs are entitled to the benefit of the jury's findings in the original verdict, including the determination that tobacco companies placed a dangerous, addictive product on the market and conspired to hide the dangers of smoking.

However, in order to be entitled to those findings, plaintiffs must prove the smokers at the heart of their cases suffered from nicotine addiction that caused a smoking-related illness.

The origin of Glogers cancer, as well as what, if any responsibility she bore for her smoking, served as key battle lines in the 12-day trial.

During Fridays closings in the trials first phase, on Engle class membership, King & Spaldings Cory Hohnbaum, representing Reynolds, challenged the claim that Gloger had smoking-related lung cancer, and argued that a mass was never found in Glogers lung. He also contended doctors could not agree on the cell-type of Glogers cancer.

There is confusion, massive confusion among the pathologists about what this was, Hohnbaum said, noting Gloger saw several pathologists during her treatment. You dont need to go talk to multiple pathologists if the pathology is clear. It was never clear.

Arnold & Porters Keri Arnold, representing Philip Morris, added that, regardless of the cancers origin, Gloger knew the dangers of cigarettes, yet chose to continue smoking. Arnold noted that Gloger had been smoking for years by the time she began smoking Philip Morris brand cigarettes. She was an adult, she was married, she had a family, she was a medical professional. She had all the maturity and information she needed to make her own decisions about her own smoking and her own health, Arnold said.

But the Gloger familys attorney, The Ratzan Law Groups Stuart Ratzan, argued Gloger was heavily addicted to cigarettes and unable to stop in time to avoid her cancer. Ratzan walked jurors through Glogers smoking history, which included numerous failed quit attempts. Theres no reason no reason at all for a person to coat their lungs every 30 or 40 minutes, a pack-and-a-half a day for 30 years, if its not for nicotine, Ratzan said. Its the only reason.

And Ratzan argued medical records and pathology reports showed Glogers treating physicians concluded she had lung cancer. He noted one pathology report, on fluid taken from around Glogers lungs, found she had non-small cell carcinoma. It means lung cancer, Ratzan said. And I defy anybody to determine otherwise.

Email Arlin Crisco at acrisco@cvn.com.

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Can ‘smart toilets’ be the next health data wellspring? – University of Wisconsin-Madison

November 14th, 2019 4:47 pm

Wearable, smart technologies are transforming the ability to monitor and improve health, but a decidedly low-tech commodity the humble toilet may have potential to outperform them all.

Thats the conclusion of a team of metabolism scientists at the University of WisconsinMadison and the Morgridge Institute for Research, who are working to put the tremendous range of metabolic health information contained in urine to work for personalized medicine.

Urine contains a virtual liquid history of an individuals nutritional habits, exercise, medication use, sleep patterns and other lifestyle choices. Urine also contains metabolic links to more than 600 human conditions, including some of the major killers such as cancer, diabetes and kidney disease.

The toilet could someday outperform wearable, smart technologies in the ability to monitor and improve health. Photo: Pexels.com

The team has two essential questions. First, can frequent monitoring and testing of urine samples glean useful real-time information about an individuals health? And second, can a technology platform be adapted to toilets that can make the collection process simple, accurate and affordable?

They received some promising answers to the first question in a small pilot study conducted this year, the results of which were published in the Nov. 11 issue of the open access Nature journal npj Digital Medicine. Two research subjects consistently collected all urine samples over a 10-day period and submitted those samples for tests with both gas chromatography and mass spectrometry for a complete readout of their metabolic signatures.

The two subjects also happen to be lead authors on the paper: the Morgridge Institutes Joshua Coon and Ian Miller, data scientist with the Coon research group. Collectively, they provided 110 samples over the 10-day period, and also used wearable technology to track their heart rates, number of daily steps, calorie consumption and sleep patterns.

Joshua Coon believes the smart toilet concept could have major population health implications, possibly providing early warning of viral or bacterial outbreaks.

The results? The samples do indeed contain a remarkable health fingerprint that follows the ebbs and flows of daily life. For example, the subjects kept records of coffee and alcohol consumption, and the biomarkers with a known connection to both of those drinks were abundantly measured. One subject took acetaminophen, which was measured in urine by a spike in ion intensity. They were also able to measure with precision the metabolic outputs from exercise and sleep.

The next step: The research team is designing a smart toilet that will incorporate a portable mass spectrometer that can recognize the individual and process samples across a variety of subjects. They plan to install the toilet in their research building and expand the user group to a dozen or more subjects. Coon says the design is a bit Rube Goldberg-like but functional.

We know in the lab we can make these measurements, says Coon, a UWMadison professor of chemistry and biomolecular chemistry. And were pretty sure we can design a toilet that could sample urine. I think the real challenge is were going to have to invest in the engineering to make this instrument simple enough and cheap enough. Thats where this will either go far or not happen at all.

While the pilot experiment didnt examine health questions, the researchers say many possibilities exist. For example, testing could show how an individual metabolizes certain types of prescription drugs in ways that could be healthy or dangerous. Also, as the population ages and pursues more stay-at-home care, urine tests would indicate whether people are taking their medications properly and if those medications are having their intended effects.

This graphic illustrates how an integrated smart toilet system might work as a real-time method of monitoring health. While the application may be years away, proof of principle is being developed in the lab. Dasom (Somi) Hwang,Joshua Coon Lab

Coon also believes the smart toilet concept could have major population health implications, not unlike the National Institutes of Health All of Us human genome database. If you had tens of thousands of users and you could correlate that data with health and lifestyle, you could then start to have real diagnostic capabilities, he says, adding that it might provide early warning of viral or bacterial outbreaks.

Coon, who runs the National Center for Quantitative Biology of Complex Systems, says the idea of meta-scale urine testing has intrigued him for some time. Josh mentioned this at a group meeting one time and it was met with laughter, Miller recalls. I thought, you know, I kind of like the idea. I already track a lot this stuff in my everyday life.

Adds Coon: So we went out and bought a couple coolers and started collecting.

While the mass spectrometer small molecule analyses are being done on $300,000 machines, Coon says portable mass spectrometer technologies exist at a tenth of that cost. He says that with a market this massive, they could eventually hit a reasonable cost threshold.

Almost every automobile on the road is more complicated than that portable mass spectrometer, he says.

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CDMOs to Benefit From Rising BTDs, Orphan Drugs and Fast Track Status – Contract Pharma

November 14th, 2019 4:47 pm

CPhI Worldwide has released the fourth part of its 2019 Annual Report. In it experts Peter Soelkner, managing director, Vetter Pharma International; Stuart Needleman, chief commercial officer, Piramal Pharma Solutions; and Adam Bradbury, industry analyst, GlobalData, discuss the future of patient centric CDMOs.In his piece, Bradbury explores how the changing drugs pipeline and increasing diversity of innovators are creating opportunities for the CDMO sector. He asserts that the increase in drug approvals and priority reviews are likely to be beneficial to the CMO industry in the next few years, as manufacturing of these products is often outsourced.In 2018, 34 orphan-designed drug NMEs were approved, the highest between 2009 and 2018; this represented 53% of all NME approvals, said Bradbury. Of these approvals, 65% were outsourced, the strongest outsourcing propensity for orphan NMEs since 2014. Mega cap companies sponsored five Orphan NMEs and only one of these had commercial dose outsourced.Priority review therapies and other accelerated drugs are more likely to be contract manufactured than products that go through standard review. Both small and mid-size cap companies are more likely to outsource their dose manufacturing capabilities and/or expertise. Therefore, small and medium sized pharma companies with priority review therapies represent a likely strong stream of new business for the industry in the near term.According to Bradburys analysis, 23 NMEs were approved with Fast Track status in 2018. This was significantly more than in 2017. Of these products, 70% of them were outsourced, which represents a five-year increase average of 58%. Due to this, the rising number of priority review designations and small cap companies gaining FDA approvals can only be a positive sign for dose CMOs. Similarly, outsourcing percentages for CDMOs of Breakthrough Therapy Designation (BTD) has never been so high. Demand also remains for high potency APIs, of which around 60% are developed for oncology and we continue to see rising demand for CDMOs with containment facilities.In other new implications for the contract services sector, Vetters Peter Soelkner predicts that new drugs will have to be produced using a more patient-centric methodology. Soelkner emphasizes that the use of personalized medicines will mean increasingly individualized dosages and packaging. But he also forewarns that this will necessitate that contract providers and the industry adapt to help reduce the costs per patient.Soelkner says that personalized medicines will require new logistics and manufacturing systems, as each therapy is tailored specifically to an individuals own genetic profile. Looked at more broadly, nearly 40% of the new drug pipeline already involves injectables, and often for smaller patient cohorts. This will continue to present manufacturing challenges, as companies switch from systems designed for larger batches.Such systems will need to allow for the creation of APIs in a small scale and possibly, be directly integrated at the filling site, said Soelkner. However, we believe that for the main disease indications todays existing types of therapies will remain standard procedure. For many diseases individualized personalized medicine is still someway off from becoming clinical routine.Personalized medicine helps avoid unwanted costs for insurance payers, by only prescribing these expensive medications for people who are going to respond to it. But these smaller volume therapies are also simultaneously driving up manufacturing costs per unit for each affected patient. To counter these costs, Soelkner states that increased Digitalization and the use of Artificial Intelligence might help manufacturers and their partners to lower the total cost of production.Digitization and Artificial Intelligence innovations may help manufacturers and their partners enhancing overall productivity and improve cost effectiveness in the future, he said.Stuart Needleman, chief commercial officer, Piramal Pharma Solutions, explores how the patient-centric journey will operate through the pharmaceutical supply chain. He states that there will be a progressive trend across the pharmaceutical industry, with a shift towards lower volume targeted therapies. This has been driven by an increasing amount of research into more complex drugs and compounds.Needleman argues that one approach is to deliver new ways of motivating the workers so that they have a real understanding of the importance of their work and the effect on real world patients.At Piramal Pharma Solutions, Patient Awareness Councils are being introduced across global sites in order to deliver true patient centricity. According to Needleman, These new bodies comprise cross-functional executives and employees, and they act as the patients advocates and ambassadors for patient centricity though development and commercialization. Moving forward in the future, they will have an extremely important role to play in every project, and are tasked with creating, managing and monitoring the best practices for applying patient-centricity to the entire organization.In another emerging patient focused trend on the rise, Needleman also reports that patients are seeking a greater deal of information, calling them the globally informed patient. Consequently, to help improve transparency, we may even see license holders share and celebrate the manufacturing records of CDMO partners.Orhan Caglayan, CPhIs brand directory, added, These results mirror what we are seeing at ICSE the worlds largest contract services exhibition where our exhibitors are reporting robust growth in demand, especially for smaller and medium pharma customers who need specialist partners to help discover, develop and manufacture therapies. It is, of course, also why our event is crucial, as we bring together the contract services community so that attendees can meet multiple potential contract services partners, while also learning about the main trends in the industry. In the next few years, we anticipate extremely good growth for our contract service specialists and we will continue to adapt our content to showcase the newest technologies that help pharma customers advance products more quickly to patients.The complete findings of the CPhI Annual Report are available for free here.

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Halting the progression of multiple sclerosis by blocking harmful B cells – FierceBiotech

November 14th, 2019 4:47 pm

The blood-brain barrier in healthy people is a powerful shield that protects neurons from harmful invaders. But in people with multiple sclerosis (MS), that shield malfunctions, allowing B cells from the immune system to pass into the brain and destroy healthy tissues.

Scientists at the University of Montreal Hospital Research Centre (CRCHUM) have identified a new target that they suggest could be exploited to slow down the flow of B cells into the brains of people with MS. They reported the discovery in the journal Science Translational Medicine.

B cells produce a substance called activated leukocyte cell adhesion molecule (ALCAM) that allows them to migrate into the brain via blood vessels, the researchers found. Blocking ALCAM in mouse models of MS reduced the flow of B cells into the brain and slowed the progression of the disease, they reported.

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RELATED: How new gene discoveries could guide precision medicine in multiple sclerosis

B cells are a major culprit in progressive MS, the most severe form of the disease, and there are drugs on the market designed to deplete them, including Roches Ocrevus. Novartis is in phase 3 trials of Arzerra (ofatumumab), a drug that eliminates B cells by binding to the surface protein CD20. Arzerra is approved to treat chronic lymphocytic leukemia, but Novartis has been gunning for a bigger market opportunity. In September, it released new phase 3 data showing that Arzerra reduced MS relapse rates by more than 50% when compared to Sanofis Aubagio.

Meanwhile, several academic teams are looking to genetics as a path to personalized MS treatment strategies. In October, researchers at Johns Hopkins reported that newly discovered variants in the genes C1, CR1 and C1QA are associated with vision loss in progressive MS. They believe further research into these complement genes could lead to the development of new MS therapies.

Blocking ALCAM could also offer a promising strategy for thwarting B cells in MS, said University of Montreal Professor Alexandre Prat, Ph.D., in a statement. "The molecule ALCAM is expressed at higher levels on the B cells of people with multiple sclerosis, he said. By specifically targeting this molecule, we will now be able to explore other therapeutic avenues for the treatment of this disease."

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