StemCell Therapy

Three investigative therapies by Proteostasis Therapeutics PTI-801, PTI-808, and PTI-428 showed potential to treat cystic fibrosis (CF) patients ineligible for approved CFTR modulators after patient-specific lab models produced promising results that support a future clinical trial.

Funded by the EUs initiative HIT-CF (Human Individualized Therapy of CF), this early study was conducted on patient organoids, which are miniaturized organ models derived from patient cells.

Based on the positive results, a clinical trial in adult CF patients is expected to start in 2020. If successful, the trial results may serve as the basis for a marketing authorization applicationin Europe in 2021.

With the help of CF-Europe (a patient organization) and the European Cystic Fibrosis Society Clinical Trial Network (ECFS-CTN), HIT-CF has been recruiting adult CF patients to collect tissue samples and create organoids, three-dimensional organ models grown in the lab.

Organoids mimic several characteristics of the organ from which they are derived, and are genetically identical to the donor. As such, they allow researchers to study biological processes and response to treatments in an environment that closely resembles human organs. As organoids are basically human cells cultured outside the body, they are said to be ex-vivo models.

The idea is to screen treatment candidates in the lab using intestinal organoids, or mini-intestines, obtained from rectal tissue (biopsies) of patients, which are collected through a quick and painless procedure. Then, based on how the organoids respond to each therapeutic candidate, a group of patients will be selected to enroll in a clinical trial.

Because the organoids can help determine the therapeutic candidate most likely to give the best results, researchers can personalize treatments for each patient before starting the trial.

So far, intestinal organoids from more than 300 patients have been created, of which 65 have been used to test Proteostasis potential CFTR modulators.

Proteostasis is honored to have been invited to participate in the HIT-CF project, and is the only company in the group with a combination of novel CFTR modulators being testedex-vivo. We are very enthusiastic about the progress of the study, Geoffrey Gilmartin, MD, chief medical officer of Proteostasis, said in a press release.

The positive results obtained on organoids support the launch of a clinical trial called CHOICES Crossover trial based on HumanOrganoidIndividual response inCF EfficacyStudy that will test the potential treatments on patients whose organoids responded favorably to the agents.

The study will also evaluate if organoids are good models to identify promising CF therapies, that is, if the responses measured on organoids translate to potential clinical benefits in patients, as reflected by lung function tests (FEV1) or sweat tests.

CHOICES will be a placebo-controlled, double-blind study that includes an eight-week treatment period, plus six months of constant dosing.

The study includes Proteostasis three candidate CFTR modulators, specially intended for patients who carry rarer CFTR genetic defects. All three compounds have different modes of action. PTI-801 works as a CFTR corrector, PTI-808 is a CFTR potentiator, and PTI-428 is a CFTR amplifier.

The trial is planned to start in mid-2020, with its first data anticipated for the end of 2020.

If moving forward, CHOICES will become the first trial based on personalized medicine for CF patients.

The inequality in access to CFTR modulators is an acute problem across Europe where 1 in 5 individuals do not have a F508del mutation [the most common CF-causing mutation]. In addition, drug reimbursement policies are leading to an ever-growing gap between patients who do, and those who do not have effective treatment options, said Christiane De Boeck, Work Package Leader at HIT-CF.

At HIT-CF Europe, we believe that novel strategies such as personalized medicine and development of new treatment options are central to addressing the inequality of access across the continent. We are thrilled with these initial results and look forward to providing additional updates, De Boeck added.

Gilmartin said that in Europe more than 2,300 adult CF patients are ineligible for approved CFTR modulators, and therefore excluded from participating in clinical trials.

This projects proposed personalized medicine approach is paving a potential new way to develop and provide access to novel CFTR modulators for patients with the most dire need for treatment options that target the cause of the disease. Additionally, based on an individual patients disease phenotype, and not just the genetic designation, this approach could also create a new path towards more effective treatment for all people with CF, Gilmartin said.

Proteostasis is also running a clinical trial evaluating a combination of the three candidate therapies for the treatment of CF patients who have at least one copy of the F508del mutation in the CFTR gene.

Ana is a molecular biologist enthusiastic about innovation and communication. In her role as a science writer she wishes to bring the advances in medical science and technology closer to the public, particularly to those most in need of them. Ana holds a PhD in Biomedical Sciences from the University of Lisbon, Portugal, where she focused her research on molecular biology, epigenetics and infectious diseases.

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Patrcia holds her PhD in Medical Microbiology and Infectious Diseases from the Leiden University Medical Center in Leiden, The Netherlands. She has studied Applied Biology at Universidade do Minho and was a postdoctoral research fellow at Instituto de Medicina Molecular in Lisbon, Portugal. Her work has been focused on molecular genetic traits of infectious agents such as viruses and parasites.

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Personalized CF Medicine to be Tested for Rare Genetic Defects in Europe - Cystic Fibrosis News Today

Artificial Intelligence has come a long way since it was in its blooming stages in the 1940s. It was a time when the direct interaction between AI and humans seemed impossible and people were even scared by the thought. All sorts of theories started emerging that made artificial intelligence look deadly for humans. The idea of robots or droids were thought of as something that could hurt the human population and even drive them to extinction. In spite of all theories, researchers were able to predict one thing for sure. Artificial intelligence will be able to transform lives and the way humans interact with one another. It will reshape the societies and industries that we know today.

Welcome to 2019, an era where artificial intelligence is vividly present all around us. In other words, we are living in the age of AI. Take a look around you. Be it the smart assistant on our mobile phones, websites, e-commerce platforms, search engine results, home automation appliances, classrooms, and more. Every single element is trying to adopt AI and cause an impact for the good. This is in turn radically transformssocieties and pushespeople to develop emotional bonding with their gadgets and AI devices. Today, when we want to find directions to a place, we do not stop by strangers and ask them. Instead, we turn on Google maps and let technology guide us.

One of the biggest breakthroughs in artificial intelligence has been its implementation in the healthcare sector. One of the most fundamental industries in the world, the healthcare sector is far behind in todays world. Even though technological marvels are constantly fascinating in the world, there is a significant amount of work that needs to be done in the healthcare sector. From diagnosis to medicine and providing healthcare facilities in rural areas, artificial intelligence has a lot of scope for health care support.

However, there has been some significant progress in the healthcare industry. All thanks to artificial intelligence that was powered by the wave of digitization. As more and more organizations are turning digital, its providing an abundance of data for machines to work upon them. Now we have robots that are successfully performing surgery on patients, software programs that are better diagnosing diseases far better than traditional pathological practices, and wearable devices that can monitor our health and send updates about alarming situations in real-time.

Still, theres an emerging need for transformation in the field of medicine. We still have drugs that address the concern of a mass population, only to leave a few with side effects. Similarly, diseases like cancer do not have any permanent remedy or targeted course of medication that treats the patient well without leaving them with any side effects. Similarly, people over the age of 50 are one of the fastest-growing demographic groups around the world. Its creating a lot of challenges for the global economy and the healthcare sector. These people need to be provided with efficient care and medical support that is targeted towards the longevity of their lives.

The Longevity Industry that provides healthcare support to the population above 50 is turning into a multi-trillion dollar industry, with close to 260 companies in the UK alone. Any progress in the longevity distribution at scale will have a huge impact and multiplicative effects on economies and societies around the world. After all, it would be a huge loss if the experienced population who have been responsible for more than a few innovations in the world are facing sudden deterioration in health in a way that reduces their quality of life. Not only will it be a huge loss to the economies but also result in increased dependency on the healthcare system. Having said this, artificial intelligence has a lot of potentials to manifest in this field and come up with solutions that target the emerging needs of the population accurately.

Research indicates that increasing the lifespan of humans even by one year will decrease the suffering of tens of millions of people and improve the quality of life for billions of others. One such effort in this direction is personalized medicine that targets the individual needs of humans with precision. So, lets say that if a person has asthma, precision medicine will only focus and target the cells that are responsible for asthma and help in regenerating them. This would leave other portions or tissues of the human body untouched and treat only what was needed. Therefore, in place of treating generalized symptoms, AI can help shift medicine towards prevention, personalization and ultimately precision.

With the advent of digitization in the healthcare sector, more and more patent records are going digital. This practice is helping researchers and medical experts understand the history of a patient in detail with the past treatments and health support theyve received. The vast amount of data collected from thousands of such medical records can be studied and used by AI to understand how a particular treatment can impact a particular gene inside the human. Moreover, digitization is also enabling researchers from all walks of life to work together, thus, laying an excellent foundation for biotechnology and computer experts to work together towards personalized medicine.

For example, CRISPR is a gene-editing technology that is being used to target the DNA sequences precisely. With this, medical experts can deliberately activate or inhibit certain genes in human beings. This demonstrates that there is a strong ability to target an individuals distinctive molecular and genetic profile, thus opening up new opportunities for personalized medicine.The National Institute of Health describes precision medicine as an emerging approach for the treatment of diseases and their prevention, which takes a persons gene variability, environment and lifestyle into account. It requires the assistance of deep learning algorithms that can learn from the data at an unprecedented rate and combine the knowledge of medical experts to reach a decision.

However, precision medicine will not be the end of human touch. It will still require vast expertise of medical experts to analyze the results obtained from machines and understand its real-world implications for the patient. With wearable devices, electronic health records and information about the geographic and demographic history of the patient, deep learning algorithms in AI have a lot of potential to device personalized medicine for people that target precision issues with high accuracy.

This article was originally published on Towards Data Science byJames Warner, a business intelligence analyst with knowledge on Hadoop/Big data analysis at NexSoftSys.com

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Thanks to AI, medical treatments are becoming more personalized - The Next Web

With help from Rachana Pradhan and Dan Goldberg

House Speaker Nancy Pelosis drug bill passes the House, but the Senates another matter.

President Donald Trumps FDA pick sails through the Senate and into the soup of vaping and other hot issues.

FDA encourages a focus on pediatric cancer with a shift to molecular targets.

A message from the Partnership for Americas Health Care Future:

New polling shows that lowering costs is the top health care priority for American voters, but embracing a one-size-fits-all new government health insurance system be it Medicare for All, Medicare buy-in, or the public option would mean higher taxes for middle-class families. Get the facts: Learn more.

Happy Friday and welcome back to Prescription Pulse! Were in the home stretch of 2019, just need to get a little thing called government funding done. Know what pharma and device provisions could make it in? Send pharma news, tips and fun reads to Sarah Owermohle (sowermohle@politico.com or @owermohle) and Sarah Karlin-Smith (skarlin-smith@politico.com or @sarahkarlin).

PELOSI DRUG BILL PASSES AND ITS OFF TO THE SENATE GRAVEYARD House Democrats overcame infighting within their ranks Thursday, passing a bill that for the first time would require the government to negotiate prices for costly drugs. The sweeping measure from Pelosi, H.R. 3 (116), hands Democrats a legislative victory to tout in the 2020 electionson a Trump administration priority, no less.

The measure passed 230-192, with two Republicans breaking ranks to support the measure. Eight Democrats jumped party lines to vote for a GOP amendment to replace the bill with H.R. 19 (116), a bundle of bipartisan provisions like measures banning pay-for-delay deals or capping seniors out-of-pocket costs, while avoiding controversial moves like negotiation or price hike caps.

In the Senate, Mitch McConnell has already said the bill is dead on arrival. But the majority leader hasnt committed to taking up his chambers alternative, a bipartisan measure from Senate Finance leaders Chuck Grassley (R-Iowa) and Ron Wyden (D-Ore.).

Grassley jumped on the House bill Thursday afternoon, saying it would result in dozens fewer therapies coming to market and that his bill was the only way forward. The House has acted and now the Senate must act, Grassley said.

Trump made vague promises to cut drug prices, including allowing government negotiations, during his 2016 campaign. But several promises have since faltered: courts killed a rule to require prices in drug ads, and the administration discarded a rule eliminating rebates over concerns it would increase premiums. The White House has thrown its support behind Grassleys bill, but the senator has been unable to rally the GOP votes.

Democrats aim to seize control of the drug pricing narrative in 2020. I believe we need real, substantive reforms, said Rep. Katie Porter (D-Calif.), a freshman who flipped her Orange County district last year, "and for a while, so did our president.

HAHN SAILS THROUGH SENATE FDA has a new leader. The Senate voted 72-18 Thursday to confirm the nomination of Washington newcomer Stephen Hahn.

Unlike his predecessor Scott Gottlieb who ultimately won much praise from Democrats who hadn't supported his nomination Hahn has no track record of working and profiting from the drug industry. He also lacks the prolific bibliography Gottlieb accumulated over the years writing on FDA-related policy, making it harder for Democrats to oppose him. Plus, Democrats were eager to see the departure of acting FDA chief Brett Giroir, the HHS assistant secretary of health, a conservative who has backed reproductive health policies opposed by the left.

But will his popularity last? Hahns bipartisan support will be tested by challenging political and public health issues, most notably how to regulate the booming e-cigarette industry amid an outbreak of serious lung illnesses associated with some vaping products.

The Trump administration promised a ban of flavored vaping products in September but in November retreated from that position after an outcry from free-market advocates and the vaping industry.

Hahn offered few hints throughout his confirmation process on how he would deal with vaping or other controversial topics like whether FDA should permit the importation of drugs from overseas to lower medication costs.

NEW REQUIREMENTS FOR PEDIATRIC ASSESSMENTS OF CANCER MEDS The agency issued draft guidance Thursday outlining how it will implement a provision in a 2017 law designed to ensure more cancer drugs are studied in children. Earlier laws requiring pediatric studies of drugs developed for adults only applied if children suffered from the same disease. But most adult cancers rarely occur in children, leading to gaps in pediatric cancer drug development.

Molecular targets a game changer: Researchers have found that a molecular-targeted drug that works to treat one cancer in adults may treat a different cancer in children if the same molecular target is relevant to both.

Sponsors of new cancer drugs for adults will now need to submit a report on pediatric cancer investigations for the drug with their marketing application to FDA, if the agency determines the target to be substantially relevant to a pediatric cancer. The requirements apply to drug applications submitted on Aug. 18, 2020 or later.

The guidance described how FDA will determine whether a molecular target is relevant and explains what should be in an initial pediatric study plan. FDA says studies described in the plan should evaluate dosing, safety and preliminary efficacy.

PHRMA SUES OVER OREGON DRUG PRICE LAWS The drug lobby on Monday challenged as unconstitutional two Oregon laws aimed at boosting transparency around prescription drug prices and increases, Portland Business Journals Elizabeth Hayes reports.

A measure passed last year, OR HB4005 (18R), requires manufacturers to submit a report to the Oregon Department of Consumer and Business Services when a drugs price rises over a threshold, explaining the hike and listing costs for making and selling the medicine. A newer provision, OR HB2658 (19R), mandates that drugmakers give 60 days notice on price increases.

A coalition of hospitals, insurers and health care advocates backed the first bill, but PhRMA said both measures were misguided and unconstitutional. The state should focus on capping out-of-pocket costs or give patients more of the negotiated savings on drugs, it said in a release.

Colorado issues report on strategies to lower drug costs Potential quick win policies that the state may pursue include price transparency related to drug price increases or PBM payments; requiring drug rebates to be passed through to employers and patients; and aligning the states importation policy with a potential expansion of federal regulations that the Trump administration is pursuing. Colorado Gov. Jared Polis has made health cost containment a priority and is one of a handful of states pushing the Trump administration to allow importation of prescription drugs from Canada.

SUPREME COURT REJECTS ARIZONAS CASE AGAINST SACKLERS Mark Brnovich, Arizonas AG, had taken the unusual step of asking the nations top court to hear his lawsuit, which accuses the Sacklers, the family behind Oxycontin-maker Purdue Pharma, of funneling $4 billion from the drug company while it faced huge liability for its role in the opioid crisis. Arizona is one of thousands of jurisdictions suing Purdue, which has declared bankruptcy. A bankruptcy judge put a hold on pending lawsuits but Brnovich decided to try his luck with the justices anyway.

RESEARCHERS CRITICIZE FINANCIAL DEPENDENCE ON INDUSTRY A group of researchers has demanded that medical research, education and practice become independent of commercial influences, Politico Europes Judith Mischke reports. The international researchers, clinicians, regulators and citizen advocates warned in a British journal that while everyone wants to ground healthcare decisions in "trustworthy evidence," there are still "widespread financial conflicts of interest across medical research, education, and practice.

In the United States, about 60 percent of medical research is industry funded, they noted. In some cases, "published outcomes of industry-sponsored studies tend to favor sponsors products, creating a 'sponsorship bias' in the evidence base that overplays benefits and underplays harms, the group writes.

They offer two solutions: Governments should pass laws requiring public disclosure of company ties, or regulators should bar public health organizations from receiving industry funding.

A message from the Partnership for Americas Health Care Future:

More than 180 million Americans rely on employer-provided coverage and new polling shows a majority of voters would rather build on whats working in our current health care system than replace it with a one-size-fits-all new government-controlled health insurance system.

Whether its called Medicare for All, Medicare buy-in or the public option, American families would face unaffordable costs and lower quality care under a one-size-fits-all system the exact opposite of what they want. See the results from the tracking poll here:Learn more.

PRESCRIPTION DRUG FACTORIES POLLUTING WATERWAYS Wastewater treatment plants are not typically equipped to remove pharmaceuticals from the waterflow, and drug cocktails have shown up in rivers across the U.S., reports Stat News Natasha Gilbert. In one area downstream of a Morgantown, W. Va., plant, an anti-seizure medication was measured at nearly 90 times the amount considered safe for wildlife. Stat and Type Investigations identified Mylan, Pfizer, Teva and three others as drugmakers dumping substantial levels of pharmaceuticals into the watershed.

CAR-T MANUFACTURING PROBLEMS PLAGUE NOVARTIS Novartis has had to give away some of its CAR-T cell therapy, Kymriah, because the personalized blood cancer treatment, made from a patients immune cells, havent met FDAs specifications, Biopharma Dive reports. In those cases the company delivered the treatment as if were an unapproved drug, through an expanded access protocol. In other cases, the company hasnt been able to use the product its made at all. Novartis presented real-world data this week that showed that even when the product didnt meet specifications it could attack cancer with no increased safety risk. Manufacturing is just one of the hurdles for commercializing the personalized medicine, which costs $475,000 for leukemia and $373,000 for lymphoma.

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Pelosi drug bill passes ... and it's off to the Senate graveyard - Politico

The landscape of precision medicine has changed dramatically over the years. The completion of the Human Genome Project in 2003 has greatly accelerated our understanding of individual genomes, leading to the idea of precision medicine: medical care tailored to the individual.

Since then, science has progressed greatly. Today, we are able to look beyond identifying mutations and instead gain a very comprehensive view of the molecular biology taking place in tumor cells. We nowunderstand that each person has a unique genetic and molecular setup, and each cell in the human body is different. Based on this knowledge, single-cell analysis tools are being developed to analyze the genetic, transcriptomic, proteomic, and epigenetic features of each cell.

As a relatively new suite of technologies, single-cell analysis can greatly improve precision medicine, a feat that is especially important in cancer biology. By providing deeper insights into each individuals disease, single-cell analysis can address many of the current challenges in precision medicine.

One area in which single-cell analysis is starting to make a difference is personalized cancer treatment. Tumors often contain very heterogeneous cell populations, which makes selecting treatments extremely challenging. Conventional diagnostic techniques often rely on the bulk analysis of cells. By taking the average of all cells in a sample, it is easy to miss a cell subpopulation that can become resistant to treatment and cause a relapse.

Now one company has taken it upon itself to improve precision medicine using single-cell analysis: Proteona. Among its first targets are blood cancers, such as multiple myeloma. As one of the most aggressive blood cancers, the treatment of multiple myeloma comes with a number of challenges.

Myeloma is a very difficult disease to treat because it constantly relapses, explains Chng Wee Joo, Senior Consultant and Head of Department of Haematology-Oncology at the National University Cancer Institute Singapore. The tumor cells are highly heterogeneous, and the immune microenvironment also plays an important role, creating additional complexity. Typically, patients are treated with multiple drugs at the same time, which triggers side effects and is extremely costly. There is definitely a need for better precision medicine here.

While the traditional bulk analysis of a tumor biopsy only provides information on the average of all cells present in a sample, single-cell analysis allows clinicians to analyze different tumor cell subpopulations, which might be resistant to a specific treatment and could cause a recurrence of the disease. In the case of multiple myeloma, single-cell proteogenomics can also be used to analyze the malignant plasma cells gene and protein expression and can be used to decide what treatments are plausible for the individual patient.

By adding protein expression analysis to single-cell sequencing, clinicians can gain valuable information on protein and gene expression as well as a better resolution between different cell types compared to analyzing gene expression alone. This is important for understanding patient cell heterogeneity.

For example, single-cell proteogenomics can provide a detailed analysis of the immune cells involved in a disease, can help clinicians understand possible resistance, and can be used for patient stratification in precision medicine trials.

There is a huge value in combining total mRNA profiling and proteomics in single-cell sequencing, says Andreas Schmidt, CEO of Proteona. While single-cell proteogenomics can be applied in various fields, what we find most interesting is the ability to gain a good snapshot of the immune cells as well as of tumor cells in the same sample. And that is valid for solid tumors as well as for liquid tumors. The information we are dealing with is already directly relevant for immuno-oncology, immunotherapy, and cell therapy. But despite the huge clinical value that we can already see on the horizon, there are no commercial solutions that can move single-cell sequencing into the clinic. And that is where we see the sweet spot.

Proteona has developed a suite of technologies called the Enhanced Single-Cell Analysis with Protein Expression (ESCAPETM) platform. ESCAPE uses DNA-barcoded antibodies to obtain protein and gene expression information from individual cells. Available as an in-house service or a kit, the ESCAPE platform has been used extensively for peripheral blood mononuclear cells (PBMC) profiling, whole tumor analysis, and cell therapy characterization.

One of the key challenges of single-cell analysis is the enormous amount of data generated. Even the sequencing of a single tumor results in hundreds of millions of data points that have to be analyzed and interpreted.

Combining datasets from different sources is also challenging. Cross-experimental comparison is often impeded by batch-to-batch differences. Moreover, single-cell analysis often results in the detection of previously unknown cell populations. Manual intervention is often needed for cell clustering and cell annotation, which demands specialist expertise, takes time and is susceptible to human error and bias.

Proteona definitely evolved from being a wet lab company with a bit of IT to a company that is equal parts bio and tech, says Schmidt. With single-cell proteogenomics you end up with thousands of mRNAs and potentially hundreds of proteins. If you were to gate them all by yourself and annotate that data, it would take a long time and it would create a lot of bias. We try to avoid this by using machine learning and automatic algorithms. One key point of our work is that we started with the wet lab and have a very deep understanding of the techniques and biology. At the end of the day, the biology is what informs us, not the algorithms.

In a partnership with AI Singapore, Proteona is further developing its computational workflows to support the knowledge-driven analysis of single-cell proteogenomics data to create unbiased data sets. The collaboration aims to further the development of artificial intelligence (AI) tools for single-cell multi-omics data analysis.

In Autumn 2019, Proteona issued an oncology challenge, co-sponsored by NovogeneAIT, in which it called for proposals from scientists and clinicians working on major clinical problems in oncology. Participants were asked to send in an abstract describing how they would use the ESCAPE platform with the chance to win $50.000 worth of single-cell analysis services from Proteona and NovogeneAIT.

The grant was awarded to Cesar Rodriguez Valdes from the Wake Forest School of Medicine in Winston-Salem, US. The proposal aims to tackle the issue of treatment selection for multiple myeloma.

By applying single-cell proteogenomics in their patient-derived 3D organoid models, the team will compare cell populations in response to multiple drugs, identifying the difference in protein and gene expression patterns. These studies will help to elucidate the mechanism of chemo-sensitivity, and potentially help to choose the suitable chemotherapy combination for each patient.

We are excited about combining Proteonas single-cell proteogenomic analysis with our patient-derived organoid screening platform, says Rodriguez Valdes. Our ambition is to develop a predictive, validated test that will facilitate clinical decision-making and improve the outcome of multiple myeloma treatment. This grant will help us to move closer to that goal.

One major challenge in multiple myeloma management is how to select the most suitable treatment strategy for each patient, given the wide range of available therapies, adds Hartmut Goldschmidt, Head of Hemato-oncology in Heidelberg. Currently, the decision of which drug to use and when depends largely on the clinicians experience. There is an urgent need for tools that help clinicians make evidence-based choices. That is where single-cell multi-omics can be extremely valuable.

Other awardees of Proteonas challenge include the runner-up, Sanjay de Mel (National University Cancer Institute, Singapore), and the finalists Nicholas Gascoigne (National University of Singapore, Singapore), Steve Bilodeau (Universit Laval, Canada), and Aaron Tan (National Cancer Centre Singapore, Singapore).

With the growing complexity of drug development and the advancement of cell therapies and gene editing techniques, the need for single-cell proteogenomics in research and the clinic is increasing. A key application of single-cell proteogenomics will be the quality control of cell therapies.

Furthermore, although there is already genomic monitoring in place for many clinical trials, oncology trials would greatly benefit from single-cell proteogenomics monitoring.

Besides blood cancers, we see multiple potential areas where single-cell multi-omics will make a clinical impact, Schmidt says. By providing in-depth data on cell heterogeneity and combining that with powerful analysis tools, we are in the best position to help clinicians to decipher complex cases, from cell therapy to solid tumors. We are only starting to harness the power of single-cell analysis.

Are you interested in learning more about single-cell proteogenomics? Check out Proteonas website for more information or get in touch with its team of experts at [emailprotected]!

Images via Shutterstock.com and Proteona

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Is Single-Cell Sequencing the Solution to Treating Aggressive Blood... - Labiotech.eu

Graph 1

PRS Significantly Modifies Lifetime Breast Cancer Risk in Mutation Carriers

Myriad Genetics, Inc.

SALT LAKE CITY, Dec. 14, 2019 (GLOBE NEWSWIRE) -- Myriad Genetics, Inc. (NASDAQ: MYGN), a leader in molecular diagnostics and precision medicine, announced that results of a new validation study of the companys polygenic risk score (PRS) for breast cancer were presented at the 2019 San Antonio Breast Cancer Symposium (SABCS) in San Antonio, Tx. The key finding is that the PRS significantly improves the precision and accuracy of breast cancer risk estimates for women of European ancestry who have pathogenic variants (PV) in high- and moderate-penetrance breast cancer genes.

Our goal is to help women understand their risk of breast cancer so that they can take steps to live longer, healthier lives. Women who have a family history of breast cancer should consider hereditary cancer testing with the myRisk Hereditary Cancer test, said Jerry Lanchbury, Ph.D., chief scientific officer of Myriad Genetics. In this landmark study, we demonstrated that for women who test positive for a mutation in one of the five most common breast cancer genes, there are additional genetic factors called single nucleotide polymorphisms (SNPs) that can further influence their lifetime risk of breast cancer.

A summary of the study follows below. Follow Myriad on Twitter via @myriadgenetics and keep up to date with SABCS meeting news and updates by using the #SACBS19 hashtag.

Myriad Poster Presentation Title: Polygenic Breast Cancer Risk Modification in Carriers of High and Intermediate Risk Gene Mutations.Presenter: Elisha Hughes, Ph.D.Date: Saturday, Dec. 14, 2019, 7:009:00 a.m. Location: Poster P6-08-07

This validation study evaluated the 86-SNP PRS as a breast cancer risk factor for women who carry PV in the BRCA1, BRCA2, CHEK2, ATM and PALB2 genes and for PV-free women. The analysis included data from 152,012 women of European ancestry who received a myRisk Hereditary Cancer test as part of their clinical hereditary cancer risk assessment. The results demonstrated that the 86-SNP PRS significantly modified the breast cancer risk for women with pathogenic mutations in the five tested breast cancer genes (p-value <10-4). For some women, the PRS significantly increased the gene-based risk of breast cancer, while in others the gene-based risk was reduced (see Graph 1). Importantly, the greatest PRS risk-modification was observed in carriers of CHEK2, ATM and PALB2 mutations with some women reaching the risk levels associated with BRCA1 and BRCA2 mutations.

To viewGraph 1: PRS Significantly Modifies Lifetime Breast Cancer Risk in Mutation Carriers, please visit the following link:https://www.globenewswire.com/NewsRoom/AttachmentNg/d56c93ca-e00f-452d-b051-6325a578454c

These findings mean that we have the potential to significantly improve the precision of hereditary cancer risk assessment for women who test positive for mutations in the high and intermediate risk breast cancer genes, said Elisha Hughes, Ph.D., lead investigator and director of Bioinformatics at Myriad Genetics. We are optimistic that this additional genetic information can help clinicians more accurately predict the risk of breast cancer and provide the best care for their patients in the future.

Next StepsThe company plans to publish these new data in a peer reviewed medical journal and make the PRS available for U.S. women of European ancestry who test positive for mutations in breast cancer genes. The PRS currently is available as part of myRisk Hereditary Cancer enhanced with riskScore for women of European ancestry who test negative for pathogenic mutations in the breast cancer genes. Specifically, the riskScore test combines the PRS with the Tyrer-Cuzick model to estimate a womans 5-year and lifetime risk for developing breast cancer. The company is committed to making myRisk Hereditary Cancer enhanced with riskScore available to all ethnicities and is developing the test for women of Hispanic and African-American ancestry who test negative. The company is currently conducting the largest ever PRS study in African Americans and will present the data at a future meeting.

Please visit Myriad at booth #113 to learn more about our portfolio of genetic tests for breast cancer. Follow Myriad on Twitter via @myriadgenetics and keep up to date with Symposium news by using the hashtag #SABCS19.

AboutriskScoreriskScore is a new clinically validated personalized medicine tool that enhances Myriads myRisk Hereditary Cancer test. riskScore helps to further predict a womens lifetime risk of developing breast cancer using clinical risk factors and genetic-markers throughout the genome. The test incorporates data from more than 80 single nucleotide polymorphisms identified through 20 years of genome wide association studies in breast cancer and was validated in our laboratory to predict breast cancer risk in women of European descent. This data is then combined with a best-in-class family and personal history algorithm, the Tyrer-Cuzick model, to provide every patient with individualized breast cancer risk.

About Myriad myRisk Hereditary CancerThe Myriad myRisk Hereditary Cancer test uses an extensive number of sophisticated technologies and proprietary algorithms to evaluate 35 clinically significant genes associated with eight hereditary cancer sites including: breast, colon, ovarian, endometrial, pancreatic, prostate and gastric cancers and melanoma.

About Myriad GeneticsMyriad Genetics Inc. is a leading precision medicine company dedicated to being a trusted advisor transforming patient lives worldwide with pioneering molecular diagnostics. Myriad discovers and commercializes molecular diagnostic tests that: determine the risk of developing disease, accurately diagnose disease, assess the risk of disease progression, and guide treatment decisions across six major medical specialties where molecular diagnostics can significantly improve patient care and lower healthcare costs. Myriad is focused on five critical success factors: building upon a solid hereditary cancer foundation, growing new product volume, expanding reimbursement coverage for new products, increasing RNA kit revenue internationally and improving profitability with Elevate 2020. For more information on how Myriad is making a difference, please visit the Company's website: http://www.myriad.com.

Myriad, the Myriad logo, BART, BRACAnalysis, Colaris, Colaris AP, myPath, myRisk, Myriad myRisk, myRisk Hereditary Cancer, myChoice, myPlan, BRACAnalysis CDx, Tumor BRACAnalysis CDx, myChoice CDx, EndoPredict, Vectra, GeneSight, riskScore, Prolaris, Foresight and Prequel are trademarks or registered trademarks of Myriad Genetics, Inc. or its wholly owned subsidiaries in the United States and foreign countries. MYGN-F, MYGN-G.

Safe Harbor StatementThis press release contains "forward-looking statements" within the meaning of the Private Securities Litigation Reform Act of 1995, including statements related to the Companys polygenic risk score and data being featured at the 2019 San Antonio Breast Cancer Symposium being held Dec. 10-14, 2019 in San Antonio, Tx.; the potential to significantly improve the precision of hereditary cancer risk assessment for women who test positive for mutations in the high and intermediate risk breast cancer genes; this additional genetic information helping clinicians more accurately predict the risk of breast cancer and provide the best care for their patients in the future; publishing these new data in a peer reviewed medical journal and making the PRS available for U.S. women of European ancestry who test positive for mutations in breast cancer genes; making myRisk Hereditary Cancer enhanced with riskScore available to all ethnicities and developing the test for women of Hispanic and African-American ancestry who test negative; conducting the largest ever PRS study in African Americans and presenting the data at a future meeting; and the Company's strategic directives under the caption "About Myriad Genetics." These "forward-looking statements" are based on management's current expectations of future events and are subject to a number of risks and uncertainties that could cause actual results to differ materially and adversely from those set forth in or implied by forward-looking statements. These risks and uncertainties include, but are not limited to: the risk that sales and profit margins of our molecular diagnostic tests and pharmaceutical and clinical services may decline; risks related to our ability to transition from our existing product portfolio to our new tests, including unexpected costs and delays; risks related to decisions or changes in governmental or private insurers reimbursement levels for our tests or our ability to obtain reimbursement for our new tests at comparable levels to our existing tests; risks related to increased competition and the development of new competing tests and services; the risk that we may be unable to develop or achieve commercial success for additional molecular diagnostic tests and pharmaceutical and clinical services in a timely manner, or at all; the risk that we may not successfully develop new markets for our molecular diagnostic tests and pharmaceutical and clinical services, including our ability to successfully generate revenue outside the United States; the risk that licenses to the technology underlying our molecular diagnostic tests and pharmaceutical and clinical services and any future tests and services are terminated or cannot be maintained on satisfactory terms; risks related to delays or other problems with operating our laboratory testing facilities and our healthcare clinic; risks related to public concern over genetic testing in general or our tests in particular; risks related to regulatory requirements or enforcement in the United States and foreign countries and changes in the structure of the healthcare system or healthcare payment systems; risks related to our ability to obtain new corporate collaborations or licenses and acquire new technologies or businesses on satisfactory terms, if at all; risks related to our ability to successfully integrate and derive benefits from any technologies or businesses that we license or acquire; risks related to our projections about our business, results of operations and financial condition; risks related to the potential market opportunity for our products and services; the risk that we or our licensors may be unable to protect or that third parties will infringe the proprietary technologies underlying our tests; the risk of patent-infringement claims or challenges to the validity of our patents or other intellectual property; risks related to changes in intellectual property laws covering our molecular diagnostic tests and pharmaceutical and clinical services and patents or enforcement in the United States and foreign countries, such as the Supreme Court decision in the lawsuit brought against us by the Association for Molecular Pathology et al; risks of new, changing and competitive technologies and regulations in the United States and internationally; the risk that we may be unable to comply with financial operating covenants under our credit or lending agreements; the risk that we will be unable to pay, when due, amounts due under our credit or lending agreements; and other factors discussed under the heading "Risk Factors" contained in Item 1A of our most recent Annual Report on Form 10-K for the fiscal year ended June 30, 2019, which has been filed with the Securities and Exchange Commission, as well as any updates to those risk factors filed from time to time in our Quarterly Reports on Form 10-Q or Current Reports on Form 8-K. All information in this press release is as of the date of the release, and Myriad undertakes no duty to update this information unless required by law.

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Myriad's Polygenic Risk Score Personalizes Risk of Breast Cancer for Woman with a Genetic Mutation in Important Breast Cancer Genes - GlobeNewswire

Thuy Truong thought her aching back was just a pulled muscle from working out. But then came a high fever that wouldnt go away during a visit to Vietnam. When a friend insisted Truong, 30, go to an emergency room, doctors told her the last thing she expected to hear: She had lung cancer. Back in Los Angeles, Truong learned the cancer was at stage 4 and she had about eight months to live.

My whole world was flipped upside down, says Truong, who had been splitting her time between the San Francisco Bay Area and Asia for a new project after selling her startup. Ive been a successful entrepreneur, but Im not married. I dont have kids yet. [The diagnosis] was devastating.

Doctors at the University of Southern California took a blood sample for genetic testing. The liquid biopsy was able to detect tumor cells in her blood, sparing her the risky procedure of collecting cells in her lungs.

Genetic sequencing allowed the lab to isolate the mutation that caused her cancer to produce too much of the EGFR (epidermal growth factor receptor) protein, triggering cancer cells to grow and proliferate. Fortunately, her type of mutation responds to EGFR-targeting drugs, such as Tarceva or Iressa, slowing tumor growth.

Unlike chemotherapy, which blasts all fast-growing cells in its wake,go after specific molecules. That makes them more effective at fighting particular types of cancers, including breast, colorectal and lung cancers. Now the approach is being expanded to fight an even broader range of cancers. Its all part of a new wave in health care called .

This is the future of medicine, says Dr. Massimo Cristofanilli, associate director for translational research and precision medicine at Northwestern University. There is no turning back. The technology is available and there are already so many targeted therapies.

Most medical treatments have been designed for the average patient, leading to a one-size-fits-all approach. But with vast amounts of data at their disposal, researchers now can analyze, our family histories and other health conditions to better understand which types of treatments work best for which segments of the population.

This is a big deal. But it requires the know-how of geneticists, biologists, experts inand computer scientists who understand big-data analytics. Several startups have already begun this work.

, founded by researchers at the University of Toronto, uses AI to predict how genetic mutations will change our cells and the impact those changes will have on the human body., co-founded by scientists and physicians from Stanford University, is building a map of what turns our genes on and off, giving physicians a guide they could use to craft personalized therapies. And, a small San Francisco startup, provides personalized advice on nutrition and wellness based on your DNA.

Just like Facebook learns to automatically recognize Aunt Martha in your family photos, Deep Genomics finds and categorizes patterns in genetic data. Once its found those patterns, the companys deep learning system can infer if and how changes to your DNA affect your body.

Thats a big step forward compared with current genetic tests. Most can only give a probability of, say, getting breast cancer based on data from an entire population. Other tests cant even tell you if the genetic changes theyve detected mean anything.

The work is personal for Brendan Frey, CEO and co-founder of Deep Genomics and a professor at the University of Toronto. Fourteen years ago, he and his wife discovered their unborn baby had a genetic condition.

We knew there was a genetic problem, but our counselor couldnt tell us if it was serious or if it was going to turn out to be nothing, Frey says. We were plunged into this very difficult, emotional situation.

The experience made Frey want to bridge the divide between identifying and understanding what they mean.

Deep learning or machine learning when computers teach themselves as they see more data can also help doctors know which drugs will most effectively treat a patients illness and whether that person is more likely to experience side effects.

It can also help predict how cancer cells will mutate. And that can help drug companies come up with new treatments as tumor cells change and patients no longer respond to the drugs that worked.

That could help turn a disease like cancer into a manageable chronic ailment, says Cristofanilli.

Where Deep Genomics analyzes patterns in genetic data to predict when mutations will make you sick, Epinomics looks at epigenomics, or the study of what turns our genes on and off.

The company describes it like this: If your genome, which shows what genes we have, is the hardware of our bodies, then the epigenome is its software programming. Epinomics aims to decode that programming.

Every cell in the body carries the same genetic code. But cells in the heart, brain, bone and skin function differently based on this programming. It happens because chemical markers attach to DNA to activate or silence genes. These markers, known as the epigenome, vary from one cell type to another and are affected by both nature (inheritance) and nurture, which can include the air we breathe and the food we eat.

Researchers think a disruption to the epigenome can cause illnesses such as,or. Understanding it could give physicians a guide to the best options for each patient, like having a GPS for treatments at the molecular level.

We are focusing on what is happening at the programming level of each cell, says Epinomics co-founder Fergus Chan. Once we understand how genes are being turned on and off, well be able to better predict which treatments will work or whether changes to lifestyle will have an impact on health.

When Vitagene co-founder and CEO Mehdi Maghsoodnia asked a doctor what vitamins he should be taking, he was handed a bottle of pills and told to hope for the best.

That was the beginning of Vitagene, which uses genetic data and other health information culled from a detailed questionnaire to deliver a personalized nutritional supplement plan that lists which vitamins you need and in what doses, as well as what to avoid.

Maghsoodnia offers an alternative to the one-size-fits-all $27 billion US dietary supplement industry. Customers pay $99 to have their DNA tested and blood analyzed. And for $69 a month, Vitagene will package and ship supplements in dosages tailored to your individual needs.

The Food and Drug Administration estimates there are more than 85,000 dietary supplements on the US market, most of which are unregulated. Nearly all are promising everything from anti-aging to weight loss, and no science behind it to tell you what works for you, says Maghsoodnia. We help filter through the noise.

Vitagenes algorithm has been tested on patients whove had bariatric surgery for weight loss, which often leaves them deprived of key nutrients. Vitagene helped develop a supplement regimen to get these patients the nutrition they need after surgery.

Precision medicine is in its early days.

This is especially true for psychiatry and its exploration of how the brain responds to the environment, stress and genetic disorders. Now several companies are selling tests to help psychiatrists select drug treatments by looking at patients DNA mutations and their metabolizing rate.

But critics caution that these genetic tests may be overselling their capabilities.

Precision medicine has been very promising in oncology, says Jose de Leon, a professor of psychiatry at the University of Kentucky who specializes in psychopharmacology. But we know a lot more about cancer and how it works. In psychiatry, its much harder because we dont know enough about how the brain works.

Yes, precision medicine holds enormous promise.

Even so, Northwesterns Cristofanilli cautions clinicians to stay grounded in reality. It can be difficult to understand where reality becomes imagination, he says. We want to make sure we are protecting patients from claims that we may not deliver.

For her part, Truong is grateful to benefit from the work thats already been done. Im an engineer, she says.

I dont believe in miracles. I believe in science.

This story appears in the spring 2017 edition of CNET Magazine. For other magazine stories, click.

: The CNET team reminds us why tech stuff is cool.

: In Europe, millions of refugees are still searching for a safe place to settle. Tech should be part of the solution. But is it? CNET investigates.

This articleoriginallyappeared on.

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Gene therapy: What personalized medicine means for you - Stock Daily Dish

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ALBUQUERQUE, N.M. Decisions, decisions. What books to get as holiday gifts for family and friends?

Here are some suggested books published this year to help narrow your choices.

n New Mexicos Palace of the Governors: Highlights from the Collections edited by Daniel Kosharek and Alicia Romero. (Museum of New Mexico Press). From front to back, this book is source of enjoyment, admiration and enlightenment, containing materials of wildly diverse subjects and many periods in the museums collections. For example, theres the image on the front cover, and on page 157, of five men of the Santa Fe Fire Department bearing intense expressions and wearing smart deep red coats, dark pants and stylish hats. The photo, an ambrotype, was taken between 1861 and 1880, and helps the reader understand the collecting function of the Palace of the Governors in Santa Fe. Ambrotype and tintype, the book states, are cased image processes that followed the ground-breaking daguerreotype. The book is a companion to the exhibit A Past Rediscovered set to open March 6, 2020 at the New Mexico History Museum in Santa Fe. A Race Around the World: The True Story of Nellie Bly & Elizabeth Bisland written by Caroline Starr Rose, illustrated by Alexandra Bye. (Albert Whitman & Company). On Nov. 14, 1889, the two ladies in the title both New York journalists began their competition in a global race against each other and against time. Nellie headed east by steamer. Elizabeth westward by train. Nellie eventually won the race. But there was more at stake. The participants were making a statement about womens independence. As Rose, an Albuquerque resident, writes in conclusion, Both journeyed alone. Both took on the world and triumphed, each on her own terms. Byes illustrations are bright, attractive and draw the reader to the art and the text. The audience is ages 4-8. Our History is the Future: Standing Rock versus the Dakota Access Pipeline, and the Long Tradition of Indigenous Resistance by Nick Estes. (Verso). From his indigenous perspective, the author details how a small 2016 Indian protest of the planned Dakota Access oil pipeline blossomed into the largest indigenous protest movement of this century. Estes also thinks of the anti-pipeline movement as a statement of goals growing out of the tradition within the long history of native resistance in the United States. The context relates in part to the contentious white-indigenous relations stretching back into the 19th century.

Estes is a citizen of the Lower Brule Sioux tribe and an assistant professor of American Studies at the University of New Mexico.

Noel Street by Richard Paul Evans. (Gallery) Evans, the author of The Christmas Box and other seasonal bestsellers, is out with a new novel, Noel Street. Set in 1975, the novel is about love, faith, family and forgiveness. Elle is a single mom working as a waitress at the Noel Street Diner in Mistletoe, Utah. She meets William, a recently returned Vietnam POW suffering from war-related mental demons. In spite of his own issues, he may hold the key to unlock Elles secret pain. This is the third book in Evans Noel Christmas collection. The Rabbit Effect: Live Longer, Happier, Healthier with the Groundbreaking Science of Kindness by Dr. Kelli Harding. (Atria) Research in 1978 was the starting point for the authors reportage of investigations into the value of hidden, crucial factors in understanding health, factors such as love, friendship and community.

Theres a social dimension to health weve completely overlooked in our scramble to find the best and most cutting-edge personalized medical care, Dr. Harding writes in the introduction. She isnt the first person to probe medicines connection to kindness or compassion. But her book contributes to a greater understanding of the link. Harding is an assistant clinical professor of psychiatry at Columbia University Irving Medical Center.

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Seasons readings to end this year or start the next with - Albuquerque Journal

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Personalized Medicine Market Outlook, Growth by Top Company, Region, Application, Driver, Trends and Forecasts By 2026| Verified Market Research -...

A two-day workshop on healthy aging hosted by UCLA is kicking off a five-year partnership between the University of California system and Nanyang Technological University, Singapore.

The workshop is being held at Mong Auditorium in Engineering VI from Dec. 9-10 with several experts from NTU and the majority of UC campuses participating.

The first day focuses on technologies for healthy aging and includes talks on the role of brain-computer interfaces in brain health and on molecular mechanisms of aging and precision medicine. The neurobiology of aging and the impact of lifestyle, prevention and policy is the topic for the second day of the workshop.

UCLA Samueli participants include Jacob Rosen, a professor of mechanical and aerospace engineering, who is presenting on technology for rewiring the brain for stroke patients, and Sam Emaminejad, an assistant professor in electrical and computer engineering, whose main focus is wearable sensors and personalized medicine.

The joint effort between the two organizations fosters an exchange of faculty and students and expands academic and research opportunities in areas including health, technology, innovation and climate change.

NTU President Professor Subra Suresh and UC President Janet Napolitano signed the memorandum of understanding on behalf of their respective institutions earlier this year in February, and the health workshop is one of the initial outcomes of the partnership.

The workshop is organized by a team led by Hal Monbouquette, a UCLA Samueli professor of chemical and biomolecular engineering; NTU Graduate College Associate Dean Sierin Lim; and NTU Graduate College Dean K. Jimmy Hsia.

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Healthy Aging Workshop Launches UC Partnership with Nanyang Technological University, Singapore - UCLA Samueli School of Engineering Newsroom

REDWOOD CITY, Calif., Dec. 11, 2019 (GLOBE NEWSWIRE) -- Despite longstanding medical guidelines recommending biomarker testing for all patients with metastatic colon cancer, a new study published in JCO Precision Oncology shows that only 40 percent of patients are tested according to guidelines.1 These findings are consistent with the rates seen in late-stage lung cancer, where clinical adoption of genomic profiling remains below the recommended standard-of-care guidelines.2 The result is that many patients are potentially being treated with less effective drugs, some having serious side-effects; while others are not being offered highly effective personalized treatment options.

The multi-center retrospective study also found that certain subgroups of patients were less likely to be tested than others. Suboptimal genotyping occurred more often if the patient was male, older than 65, progressed from early stage disease, or treated in a community cancer center. Furthermore, only 28 percent of patients on anti-EGFR treatments received the necessary testing to determine eligibility. This study also showed that using a comprehensive panel, as opposed to single-gene testing, could possibly result in a 50 percent increase in guideline-recommended testing rates.

For over a decade, medical guidelines have recommended multigene mutation testing for metastatic colon and rectal cancer patients to ensure patients receive the optimal treatment available. While the study shows that testing has improved slightly, the disappointing reality is that the majority of patients, including those receiving targeted treatments, are not being comprehensively tested, said Stuart Goldberg, MD, a lead study investigator who served as chief scientific officer at COTA, Inc. during the project. Personalized medicine holds great promise for achieving better outcomes, but we will not see the benefits until genomic testing is routinely adopted into clinical practice.

Newly available therapies routinely help some people with metastatic colorectal cancer gain a year or two of life, but these novel treatments cannot be applied unless comprehensive genotyping is routinely performed, said Richard Lanman, MD, Guardant Health Global Chief Medical Officer. Just as concerning was that 28 percent of patients received targeted treatments without being tested, and for some we would have known in advance that the treatment was not going to work. At Guardant we are committed to helping reverse this trend by helping to educate oncologists and offering a non-invasive and comprehensive genomic testing solution that overcomes the limitations of tissue biopsies with our Guardant360 blood test.

The American Society of Clinical Oncology (ASCO) and National Comprehensive Cancer Network (NCCN) guidelines recommend biomarker testing for all patients with metastatic colon cancer to screen for genomic alterations in KRAS, NRAS, BRAF, ERBB2 (HER2), NTRK and microsatellite instability (MSI) to help guide more effective treatment decisions. Targeted therapies and immunotherapies have been shown to improve clinical outcomes. For example, anti-EGFR therapies are commonly used in treating metastatic colon cancer but are not efficacious in patients whose tumors harbor KRAS, NRAS, or BRAF V600E mutations.3 Additionally, immunotherapy is effective in patients with MSI and anti-HER2 treatment is effective in patients with ERBB2 (HER2) amplifications.4,5 Professional guidelines continue to be updated as new therapies come to market such as the recent recommendation to test patients for NTRK fusions because they may benefit from treatment with tumor-agnostic NTRK inhibitors.

Study details can be found here.

About Guardant HealthGuardant Health is a leading precision oncology company focused on helping conquer cancer globally through use of its proprietary blood tests, vast data sets and advanced analytics. The Guardant Health Oncology Platform leverages capabilities to drive commercial adoption, improve patient clinical outcomes and lower healthcare costs across all stages of the cancer care continuum. Guardant Health has launched liquid biopsy-based Guardant360 and GuardantOMNI tests for advanced stage cancer patients and LUNAR assay for research use and for use in prospective clinical trials. In parallel, Guardant Health is actively exploring the performance of the LUNAR assay in initial studies related to screening and early detection in asymptomatic individuals.

Forward Looking StatementsThis press release contains forward-looking statements within the meaning of federal securities laws, including statements regarding the adoption of Guardant Healths products, which involve risks and uncertainties that could cause Guardant Healths actual results to differ materially from the anticipated results and expectations expressed in these forward-looking statements. These statements are based on current expectations, forecasts and assumptions, and actual outcomes and results could differ materially from these statements due to a number of factors. These and additional risks and uncertainties that could affect Guardant Healths financial and operating results and cause actual results to differ materially from those indicated by the forward-looking statements made in this press release include those discussed under the caption Risk Factors in Guardant Healths Annual Report on Form 10-K for the year ended December 31, 2018, and in its other reports filed by Guardant Health with the Securities and Exchange Commission. The forward-looking statements in this press release are based on information available to Guardant Health as of the date hereof, and Guardant Health disclaims any obligation to update any forward-looking statements provided to reflect any change in its expectations or any change in events, conditions, or circumstances on which any such statement is based, except as required by law. These forward-looking statements should not be relied upon as representing Guardant Healths views as of any date subsequent to the date of this press release.

In light of the foregoing, investors are urged not to rely on any forward-looking statement in reaching any conclusion or making any investment decision about any securities of Guardant Health.

Investor Contact:Lynn Lewis or Carrie Mendivilinvestors@guardanthealth.com

Media Contact:Anna Czene or Ian Stonepress@guardanthealth.com

References1. Gutierrez ME, Price KS, Lanman RB, et al. Genomic Profiling for KRAS, NRAS, BRAF, Microsatellite Instability (MSI) and Mismatch Repair Deficiency (dMMR) among Patients with Metastatic Colon Cancer. JCO Precision Oncol. December 20192. Leighl NB, Page RD, Raymond VM, et al. Clinical Utility of Comprehensive Cell-Free DNA Analysis to Identify Genomic Biomarkers in Patients with Newly Diagnosed Metastatic Non-Small Cell Lung Cancer. Clin Cancer Res. April 20193. Van Cutsem E, Khne CH, Lng I, et al. Cetuximab plus irinotecan, fluorouracil, and leucovorin as first-line treatment for metastatic colorectal cancer: updated analysis of overall survival according to tumor KRAS and BRAF mutation status. J Clin Oncol. May 20114. Overman MJ, Lonardi S, Wong KYM, et al. Durable clinical benefit with nivolumab plus ipilimumab in DNA mismatch repair-deficient/microsatellite instability-high metastatic colorectal cancer. J Clin Oncol. March 20185. Sartore-Bianchi A, Trusolino L, Martino C, et al. Dual-targeted therapy with trastuzumab and lapatinib in treatment-refractory, KRAS codon 12/13 wild-type, HER2-positive metastatic colorectal cancer (HERACLES): a proof-of-concept, multicentre, open-label, phase 2 trial. Lancet Oncol. April 2016

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Study Shows Only 40 Percent of Patients with Metastatic Colon Cancer Receive Guideline-Recommended Biomarker Testing - GlobeNewswire

On March 8, 2019, the US Food and Drug Administration (FDA) approved the PD-L1 inhibitor atezolizumab for first-line treatment of metastatic triple-negative breast cancer in combination with nab-paclitaxel chemotherapy for patients who are ineligible for surgery and whose tumors express PD-L1. This marks the first time a regimen approved for breast cancer includes an immunomodulator, and represents the first targeted agent approval in triple-negative breast cancer a disease that, historically, has only had chemotherapy as a treatment option.

Triple-negative breast cancer is an especially aggressive subtype of breast cancer that is identified by the absence of expression of estrogen, progesterone, or HER2 protein receptors on the cancers surface. These proteins, when present in other subsets of breast cancer, allow us to target and take advantage of the protein to kill the cancer cell. For patients with triple-negative metastatic cancer, the average survival is only slightly more than a year, meaning that half of women diagnosed with this disease will not live beyond that benchmark.

This groundbreaking approval was based on results of the IMpassion130 trial (ClinicalTrials.gov Identifier: NCT02425891), for which Sarah Cannon, the Cancer Institute of HCA Healthcare in Nashville, Tennessee, participated. The IMpassion130 trial treated 902 women with either nab-paclitaxel alone or nab-paclitaxel in combination with atezolizumab.

A post-hoc update of long-term progression-free survival revealed that in the PD-L1 immune cell-positive population, the updated median progression-free survival was 7.5 months

(95% CI, 6.79.2) with atezolizumab plus nab-paclitaxel and 5.3 months (95% CI, 3.85.6) for nab-paclitaxel alone (stratified hazard ratio [HR], 0.63; 95% CI, 0.500.80; P <.0001).1

In patients with PD-L1positive tumours, median overall survival was 25 months (95% CI, 19.630.7) with atezolizumab and 18 months (13.620.1) with placebo. The stratified hazard ratio for overall survival was 0.71 (95% CI, 0.540.94).1

Immunotherapy treatments help a patients immune system recognize cancer as foreign and attack it. In contrast to chemotherapy, which can be thought of as poison for cancer cells, immunotherapy is essentially a way to boost ones own immune cells to fight cancer. Chemotherapy has known side effects, such as hair loss, nausea, and fatigue; however, the side-effect profile for immunotherapy treatment is different. Patients receiving immunotherapy treatment may experience low thyroid hormone levels or inflammation in the lungs or gastrointestinal system. When treating patients with immunotherapy, it is important to closely monitor patients for these rare side effects and treat them with steroids when appropriate.

The approval of atezolizumab not only represents a win for some patients with triple-negative breast cancer characterized by PD-L1 expression, it also more broadly represents a win for personalized medicine. In the past decade, we have gotten smarter about knowing that every persons cancer may be driven by individual mutations and alterations. We have had access to tests to examine the expression of estrogen receptors, progesterone receptors, and HER2 receptors for some time, but only now do we know to test for PD-L1, which allows us break this broad disease down into smaller subsets using the markers the cancer possesses, and, prescribe more targeted treatments. We have also started looking at a broader profile of the alterations in a cancer through molecular profiling, where hundreds of genes are examined for mistakes and abnormal copy numbers in an attempt to personalize care. Tailoring treatment to each person and their individual cancer characteristics at the cell level will become even more common in the coming years.

IMpassion130 also highlights the benefit of clinical trials not just for future patients, but also for those who are currently enrolled. I treated patients enrolled in this trial, and in fact, we still have patients who are receiving this therapy years after their diagnosis. This trial and this agent highlight the advances we make in cancer treatment every day through the power of clinical trials. Unfortunately, only about 2% to 5% of adults with cancer participate in clinical trials in the US and more than 50% say they are not even aware of the existence of trials.2,3 These studies show us that we need to continue to work toward making clinical trials a well-understood concept through patient education, dispelling misconceptions, and celebrating the big advances, such as the approval ofatezolizumab in this patient group.

Erika Hamilton, MD, is the Director of the Breast Cancer and Gynecologic Cancer Research Program at Sarah Cannon Research Institute in Nashville, Tennessee.

References

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Triple-Negative Breast Cancer Now Has a Targeted Agent - Cancer Therapy Advisor

FREMONT, CA: Diagnostics and treatment have undergone sea changes in recent years. The field which was dominated by traditional medical technologies is now on the cusp of artificial intelligence-driven changes. The area of radiology has also come under the influence of advancement, leading to the emergence of radiomics. The development has been helping the medical industry in dealing with the imminent shortage of qualified radiologists. Innovators have put their brains together and taken diagnostic scanning to the next level with radiomics. Radiomics is still in a developmental phase, but it has already contributed to the healthcare industry. Some ways in which the technology is helping medical fraternity is mentioned in the list below.

Immunohistochemical Evaluations

Patients can be checked for immunohistochemical (IHC) characteristics with the help of radiomics-based imaging. Medical researchers have successfully created unique machine learning models that can detect thyroid nodules in patients with high degrees of accuracy. The development makes the process non-invasive and enables the prediction of hormonal presence in patients. Computer topography imaging techniques cannot present ICH related information. According to experts, these capabilities make a timely diagnosis of thyroid cancers possible and feasible.

Brain Tumor Detection and Treatment

Current technologies for detection and classification of malignant brain tumors are good enough, but with radiomics, the amounts of data that can be extracted increases substantially. Subsequently, radiomic analysis can generate insightful data which are helpful for diagnosis, evaluation, and treatments. The advanced imaging also enables practitioners to adhere to recently announced protocols which vary significantly from the standard ones. One of the significant advantages of using radiomics in brain tumor assessment is that it allows the determination of molecular characteristics which help categorize molecular subtypes.

Diagnosis of Cancers in Breast, Lungs and Neck

Radiomics has been widely adopted by medical fraternity for the neck, lungs, and breast cancer diagnosis, treatment, and prognosis. Predicting the stage of cancer in cancer-afflicted patients with minimum or no invasive procedures is a significant step forward. Radiomics has shown excellent results in projecting the differences which help in classifying the stage of cancer. Even reoccurrence predictions have been benefitted with radiomic signature-based investigations. The ability of radiomics to take into consideration a higher number of factors and features by combining recorded clinical information makes it useful in early detection of breast, lungs, and neck cancers. In the case of breast cancer patients, radiomics is predicting response to therapy with high accuracy and thus optimizing treatment.

Precision Medicine

High sensitivity and high specificity are the remarkable features of radiomics. As the medical industry adopts precision medicine, radiomics is all set to play a very crucial role. Radiomics is powerful because it applies artificial intelligence and deep learning characteristics to radiology, thus helping define tissues with much more accuracy. Every patient can undergo a radiomics-based diagnosis and come up with personalized reports. These reports can relay detailed information which enables doctors to prescribe personalized medication or treatment models. Thus, radiomics is helping extract qualitative and quantitative data which make precision medicine possible.

Reading and interpreting medical data is challenging. However, radiomics is transforming decision-making by bettering identification and characterization. One can expect the diagnosis to become faster and error-free as computerization becomes central to the process. Subsequently, patient care will also improve as doctors gain access to deeper health insights.

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How is Radiomics the Next Wave in Medtech? - Medical Tech Outlook

GMI Research has recently published a report on Global Precision Medicine market, the report highlights the key ongoing market trend, market growth, drivers and restraints of Global Precision Medicine market. The report provides the forecast data for the period of 2019 to 2026. GMI Research analysts have a thorough understanding of the market as they are on top of any market trends and developments, which help them to understand current market dynamics, market competitive landscape and future trends of Global Precision Medicine in a couple of years.According to the report, the Global Precision Medicine Market is projected to grow at a CAGR growth of ~XX% through the forecast period from 2019 to 2026 owing to various key factors such as Precision medicine is being eyed upon as lucrative business investment opportunity by the major pharmaceutical companies owing to the rising patient preference for personalized treatment, advancements in wireless technologies and big data analytics, and tremendous increase in genetic diseases such as cancer. Our analyst knows the importance of data and are also aware of the significance of providing accurate data. The analyst deploys various approaches from primary research to credible secondary research sources and validates the data from multiple sources to make sure that our client gets the accurate picture of the Global Precision Medicine and the market size, market projections, competitive landscape etc.The reports also profile the major market players. The major players are Illumina, Inc., GE Healthcare, Roche Group, bioMerieux SA, IBM Corporation, Novartis AG, and Abbott Laboratories among others.Request Sample Report Now at Link https://www.gmiresearch.com/report/global-precision-medicine-market/sample-requestKey areas the report on Global Precision Medicine market addresses are: What are the major market drivers influence the growth of Global Precision Medicine? Which region provides the maximum growth opportunities? What makes one product more attractive over another and ranking of all the products and the market opportunity they provide? What are the key developments and how they are impacting the Global Precision Medicine market? What are the current key trends of Global Precision Medicine? How the competitive landscape looks like and what strategies companies are adopting to increase their market share?How the insights on Global Precision Medicine will help our clients in critical decision making? Help our client to know all the segments and sub-segments in detail Assessment of competitive landscape of Global Precision Medicine Details of new product launch trend, innovations, market expansions and other strategies adopted by major players Regional market trends and opportunities available in each region North America, Europe, Asia Pacific and RoWSpeak to Research Analyst to Understand more About Research at Link https://www.gmiresearch.com/report/global-precision-medicine-market/Competitive Landscape key players main strategies and their product offerings Major players products strength on each segment and vertical Product Benchmarking analysis of key players Key Players Recent Developments and strategic directionRequest for Customization at Link https://www.gmiresearch.com/report/global-precision-medicine-market/request-for-customizationAbout GMI ResearchGMI Research provides research and consulting solutions to our clients. We help our global clients through independent fact-based insights, ensuring their business achieve success by beating the competition. We provide syndicated research, customized market and competitive intelligence research, sales enablement support and data analytics services.Contact UsGMI ResearchLevel 1, The Chase Carmanhall Road, Sandyford Industrial Estate,Dublin D18 Y3X2, IrelandPh. No: +353 1 442 8820Email: [emailprotected]Web: https://www.gmiresearch.com

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Aspen Neuroscience, a new San Diego biotech company working on stem cell treatment for Parkinsons disease, has come out of stealth mode and raised $6.5 million to pursue clinical testing for its therapy.

Co-founded by well-known stem cell scientist Jeanne Loring, Aspen Neuroscience proposes creating stem cells from modified skin cells of Parkinsons patents via genetic engineering.

The stem cells, which can become any type of cell in the body, then would undergo a process that makes them specialize into dopamine-releasing neurons.

People with Parkinsons lose a large number up to 50 percent at diagnosis of specific brain cells that make the chemical dopamine.

Without dopamine, nerve cells cannot communicate with muscles and people are left with debilitating motor problems.

Once these modified skin cells have been engineered to specialize in producing dopamine, they can be transplanted into the Parkinsons patient to restore the types of neurons lost to the disease.

The reason we called it Aspen is because l was raised in the Rocky Mountain states, said Loring. When there is a forest fire in the Rockies, the evergreens are wiped out but the aspens are the fist that regenerate after the burn. So it is a metaphor for regeneration.

Aspen still has a long way to go before its proposed therapy would be available to Parkinsons patients. It has been meeting with the U.S. Food and Drug Administration to provide animal trial data and other information in hopes of getting permission to start human clinical trials.

But the company expects the earliest it would get the go-ahead from FDA to start human trials would be 2021.

Loring has been working on the therapy for eight years. She is professor emeritus and founding director of the Center for Regenerative Medicine at the Scripps Research Institute.

Loring co-founded the 20-employee company with Andres Bratt-Leal, a former post-doctoral researcher in Lorings lab at Scripps.

Joining them as Aspens Chief Executive is Dr. Howard Federoff, former vice chancellor for health affairs and chief executive of the University of California Irvine Health System.

Federoff said the company is the only one pursuing the use of Parkinsons patients own cells as part of neuron replacement therapy.

Aspens proprietary approach does not require the use of immuno-suppression drugs, which can be given when transplanted cells come from another person and perhaps limit the effectiveness of the treatment.

Aspens approach is a therapy that is likely to benefit from the fact that your own cells know how to make the best connections with their own target cells in the brain, even in the setting of Parkinsons disease, said Federoff. So when transplanted it is able to set back the clock on Parkinsons.

In addition to Aspens main therapy, it is researching a gene-editing treatment for forms of Parkinsons common in certain families.

Aspens research work up to now has been supported by Summit for Stem Cell, a non-profit on which provides a variety of services for people with Parkinsons disease.

The new seed funding round was led by Domain Associates and Axon Ventures, with additional participation from Alexandria Venture Investments, Arch Venture Partners, OrbiMed and Section 32.

Aspens financial backing, combined with its experienced and proven leadership team, positions it well for future success, said Kim Kamdar, a partner at Domain Associates. Domain prides itself on investing in companies that can translate scientific research into innovative medicines and therapies that make a difference in peoples lives. We clearly see Aspen as fitting into that category, as it is the only company using a patients own cells for replacement therapy in Parkinsons disease.

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Aspen Neuroscience gets funding to pursue personalized cell therapy for Parkinsons disease - The San Diego Union-Tribune

ViewRay has inked respective agreements with Elekta AB and Medtronic that aim to increase understanding into and practices of MR-guided radiation therapy.

The partnerships will each include the establishment of clinical studies to assess the impact and benefits provided by the treatment, as well as probes into additional therapeutic areas in which the technology could prove beneficial.

"We are pleased to announce these important collaborations and investments in ViewRay, said ViewRays president and CEO Scott Drake in a statement. Our goal is to concurrently prove the value of MR-guided radiation therapy and strengthen our balance sheet.

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Elekta will also invest in ViewRay, taking on a minority interest with the potential to rise to 9.9 percent.

Elekta believes that competition is crucial to drive the adoption of any new technology, and that is why we are committed to continuing to develop and offer customers our high-field Elekta Unity and promoting this technology to benefit patients worldwide, said Elekta's president and CEO Richard Hausmann in a statement. By investing in ViewRay, we ensure that the two inventors of MR-guided radiation therapy continue to drive the paradigm shift forward.

The deal between Medtronic and ViewRay is a similar clinical collaboration, that will make use of the MRIdian MR-guided radiation therapy system in studies. Medtronic has also committed to investing in a minority share of ViewRay as part of the deal.

"We are well-positioned to drive MRIdian to standard of care, noted Drake, adding that, the ability to see clearly during the procedure, track tumors and soft tissues, and auto-gate the beam is integral to delivering highly precise, personalized medicine."

The agreements with Elekta and Medtronic follow a rough period for ViewRay, which was,hit with an investor lawsuit in September, filed over allegedly false or misleading statements made regarding negative operational and financial issues reported in August.

Specific allegations included failure to disclose that demand for ViewRay systems dropped due, in part, to changes around Medicare reimbursement; overstating reported backlog; and damages suffered by investors because of materially false and misleading statements made by ViewRay at all relevant times. Upon disclosing these challenges, the companys stock price fell by more than 50 percent. It also slashed its previously issued full fiscal year 2019 financial guidance.

In addition to the deals with Elekta and Medtronic, ViewRay's biggest shareholder, Fosun International Limited will be putting in more money in order to keep its ownership percentage at present levels.

The investments from Elekta and Medtronic, however, will only take place if at least $75 million is raised. Elekta's investment will also require an aggregate cap of $36 million.

The deals are non-binding, subject to various terms and conditions.

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ViewRay teams with Elekta and Medtronic on MR-guided radiation therapy - DOTmed HealthCare Business News

Washington, DC (UroToday.com) On the final day of the Society of Urologic Oncology Meeting (SUO 2019), the six best abstracts submitted were selected for presentation. One of these highlighted studies from Memorial Sloan Kettering Cancer Center (MSKCC) evaluated the differential response of high grade non-muscle invasive bladder cancer to Bacillus Calmette-Guerin (BCG) on the basis of FGFR3 alterations. Dr. Timothy Clinton, a urologic oncology fellow at MSKCC, presented the groups findings.

Recently, the FDA approved Erdafitinib, a fibroblast growth factor receptor (FGFR)3 kinase inhibitor, for use in metastatic urothelial carcinoma on the basis of phase II results published this summer in NEJM. In that publication, Loriot et al. demonstrated a 59% response rate in patients with unresectable or metastatic urothelial carcinoma.1 These results have driven interest in expanding the use of FGFR3 kinase inhibitors and looking to FGFR3 alterations for prognostication. Specifically, FGFR3 mutations have been previously associated with lower response rates to immune checkpoint inhibition, are more commonly identified in non-muscle invasive bladder cancer which is specifically less likely to progress. For these reasons, Dr. Clinton postulated that FGFR3 alterations may be able to predict BCG response.

To that end, Dr. Clinton and colleagues identified treatment nave patients with high-grade nonmuscle invasive bladder cancer (NMIBC) enrolled in a prospective protocol that captured targeted exon sequencing on pretreatment tumor DNA and matched germline DNA. Within the MSKCC cohort there were over 1,400 bladder specimens with a quarter demonstrating genomic alterations in FGFR3. Of these patients, 119 patients with high-grade NMIBC underwent BCG treatment with 51containing aFGFR3alteration (43%); 39 patients (76%) were cTaHG and 12 (24%) were cT1HG at diagnosis. Interestingly, no patients with CIS had alterations in FGFR3.

Dr. Clinton proceeding by highlight the differential response to BCG between those who were FGFR3 wild-type and those with alterations. Overall, the median follow up was 60 months (IQR 32-75). At that time 39% of the entire cohort was free from high-grade recurrence with a significant difference between the two groups. 65% of wild-type patients were free from high-grade recurrence, were as only 39% of the FGFR3 alteration group was similarly recurrence-free(p<0.05). There was no difference in progression, however, the rate was very low in both groups. Looking more in depth into the FGFR3 altered tumors, the group looked for additional mutations that corresponded to BCG response, identifying significant co-occurrence ofFGFR3mutations with cell cycle genes includingCDKN2A(p=0.03) andMDM2(p=0.03) and noFGFR3mutated tumor contained anERBB2mutation.

Overall, Dr. Clinton concluded that while FGFR3 has been historically associated with favorable prognosis, FGFR3altered tumors are associated with high recurrence rates following BCG. Given these findings and the recent BCG shortages, patients with these alterations may benefit from alternative therapy with FGFR3 kinase inhibitors. The study represents a major step in predicting BCG response rates and FGFR3 profiling could represent a novel path for personalized medicine in the future. Clinical trials for FGFR3 in NMIBC are currently underway with results pending.

Presented by:TimothyClinton, MD, Urologic Oncology Fellow at Memorial Sloan Kettering Cancer Center

Written by: Adrien Bernstein, MD, Society of Urologic Oncology Fellow, Fox Chase Cancer Center, Fox Chase Cancer Center, Philadelphia, PAat the 20th Annual Meeting of the Society of Urologic Oncology (SUO), December 4 - 6, 2019, Washington, DC

References:1.Loriot, Y., Necchi, A., Park, S., Garcia-Donas, J., Huddart, R., & Burgess, E. et al. (2019). Erdafitinib in Locally Advanced or Metastatic Urothelial Carcinoma.New England Journal Of Medicine,381(4), 338-348. doi: 10.1056/nejmoa1817323

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SUO 2019: Response of FGFR3 Alterations In High-Grade Non-Muscle Invasive Bladder Cancer Treated with Intravesical BCG Therapy - UroToday

In this video from SC19, Jaan Mannik from One Stop Systems describes how the company delivers AI on the Fly.

AI on the Fly puts computing and storage resources for the entire AI workflow, not in the datacenter, but on the edge near the sources of data. Applications are emerging for this new AI paradigm in diverse areas including autonomous vehicles, predictive personalized medicine, battlefield command and control, and industrial automation. The common elements of these solutions are high data rate acquisition, high speed low latency storage and efficient high performance AI training and inference computing. All of these building block elements are connected seamlessly with memory mapped PCI Express system, interconnected and customized as appropriate, to meet the specific environmental requirements of in the field installations.

AI on the Fly solutions are different than traditional datacenter centric AI infrastructure, as they must deploy in harsh and rugged environments. The solutions must meet unique criteria for shock and vibration, redundancy, large operating temperature, altitude ranges and uninterrupted power sources. Few companies have the expertise across all the technologies required for designing and delivering tightly integrated AI on the Fly platforms.

OSS is a custom manufacturer of specialized high performance computing systems for industries that are showing the largest proliferation of use cases for AI on the Fly systems. These industries include:

These edge applications have unique requirements over traditional embedded computing. There is no compromise possible in delivering high performance while maintaining efficient space, weight and power. Delivering the high performance required in edge applications necessitates PCIe interconnectivity providing the fast data highway between high speed processors, NVMe storage and compute accelerators using GPUs or application specific FPGAs. AI on the Fly high performance applications will naturally demand this capability on the edge. Additionally, these solutions often require unique space and power saving form factors and specialized rugged enclosures.

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One Stop Systems does AI on the Fly at SC19 - insideHPC

BETHESDA, Md., Dec. 4, 2019 /PRNewswire/ --The American College of Medical Genetics and Genomics (ACMG) heads to a new destination in sunny San Antonio, Texas in 2020. Named one of the fastest growing meetings in the USA by Trade Show Executive Magazine, the ACMG Annual Clinical Genetics Meeting continues to provide groundbreaking research and news about the latest advances in genetics, genomics and personalized medicine. To be held March 17-21, the 2020 ACMG Annual Meeting will feature more than 40 scientific sessions, 3 Short Courses, workshops, TED-Style talks and satellite symposia, and over 800 poster presentations on emerging areas of genetic and genomic medicine.

Interview those at the forefront in medical genetics and genomics, connect in person with new sources and get story ideas on the clinical practice of genetics and genomics in healthcare today and for the future. Learn how genetics and genomics research is being integrated and applied into medical practice.

Topics include gene editing, cancer genetics, molecular genomics, exome sequencing, pre- and perinatal genetics, biochemical/metabolic genetics, genetic counseling, health services and implementation, legal and ethical issues, therapeutics and more.

Credentialed media representatives on assignment are invited to attend and cover the ACMG Annual Meeting on a complimentary basis. Contact Kathy Moran, MBA at kmoran@acmg.net for the Press Registration Invitation Code, which will be needed to register at http://www.acmgmeeting.net.

Abstracts of presentations will be available online in January 2020. A few 2020 ACMG Annual Meeting highlights include:

Program Highlights:

Cutting Edge Scientific Concurrent Sessions:

Three half-day Genetics Short Courses on Monday, March 16 and Tuesday, March 17:

Photo/TV Opportunity: The ACMG Foundation for Genetic and Genomic Medicine will present bicycles to local children with rare genetic diseases at the Annual ACMG Foundation Day of Caring on Friday, March 20 from 10:30 AM 11:00 AM at the Henry B. Gonzlez Convention Center.

Social Media for the 2020 ACMG Annual Meeting: As the ACMG Annual Meeting approaches, journalists can stay up to date on new sessions and information by following the ACMG social media pages on Facebook,Twitter and Instagram and by usingthe hashtag #ACMGMtg20 for meeting-related tweets and posts.

Note be sure to book your hotel reservations early.

The ACMG Annual Meeting website has extensive information at http://www.acmgmeeting.net.

About the American College of Medical Genetics and Genomics (ACMG) and the ACMG Foundation for Genetic and Genomic Medicine (ACMGF)

Founded in 1991, the American College of Medical Genetics and Genomics (ACMG) is the only nationally recognized medical society dedicated to improving health through the clinical practice of medical genetics and genomics and the only medical specialty society in the US that represents the full spectrum of medical genetics disciplines in a single organization. The ACMG is the largest membership organization specifically for medical geneticists, providing education, resources and a voice for more than 2,300 clinical and laboratory geneticists, genetic counselors and other healthcare professionals, nearly 80% of whom are board certified in the medical genetics specialties. ACMG's mission is to improve health through the clinical and laboratory practice of medical genetics as well as through advocacy, education and clinical research, and to guide the safe and effective integration of genetics and genomics into all of medicine and healthcare, resulting in improved personal and public health. Four overarching strategies guide ACMG's work: 1) to reinforce and expand ACMG's position as the leader and prominent authority in the field of medical genetics and genomics, including clinical research, while educating the medical community on the significant role that genetics and genomics will continue to play in understanding, preventing, treating and curing disease; 2) to secure and expand the professional workforce for medical genetics and genomics; 3) to advocate for the specialty; and 4) to provide best-in-class education to members and nonmembers. Genetics in Medicine, published monthly, is the official ACMG peer-reviewed journal. ACMG's website (www.acmg.net) offers resources including policy statements, practice guidelines, educational programs and a 'Find a Genetic Service' tool. The educational and public health programs of the ACMG are dependent upon charitable gifts from corporations, foundations and individuals through the ACMG Foundation for Genetic and Genomic Medicine.

Kathy Moran, MBAkmoran@acmg.net

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Press Registration for the 2020 ACMG Annual Clinical Genetics Meeting Is Now Open - Herald-Mail Media

An international team of scientists has developed artificial neurons on silicon chips that behave just like neurons in the body. The first-of-its-kind achievement could lead to the development of medical devices for treating chronic diseases, such as heart failure, Alzheimers disease, or other neurodegenerative disorders. Critically, the artificial neurons not only behave just like biological neurons, but require just one-billionth the power of a microprocessor, making them ideally suited for use in medical implants and other bio-electronic devices.

Until now neurons have been like black boxes, but we have managed to open the black box and peer inside, stated research lead Alain Nogaret, PhD, professor from the University of Bath department of physics. Our work is paradigm-changing because it provides a robust method to reproduce the electrical properties of real neurons in minute detail. But its wider than that because our neurons only need 140 nanoWatts of power. Thats a billionth of the power requirement of a microprocessor, which other attempts to make synthetic neurons have used. This makes the neurons well suited for bio-electronic implants to treat chronic diseases.

The researchers, led by the University of Bath team, and including collaborators at the Universities of Bristol, Zurich, and Auckland, describe the artificial neurons in a study published in Nature Communications, titled, Optimal solid-state neurons.

Designing artificial neurons that respond to electrical signals from the nervous system in the same way that real neurons would has been a major goal in medicine for decades, as it would open up the possibility of curing conditions in which neurons dont work properly, have had their processes severed, as in spinal cord injury, or have died. Artificial neurons could repair diseased biocircuits by replicating their healthy function and responding adequately to biological feedback to restore body functions.

However, developing artificial neurons comes with immense challenges associated with the complex biology and hard-to-predict neuronal responses. The difficulty of measuring microscopic parameters that control the dynamics of ionic currents and the nonlinearity of ionic conductances has hampered so far theoretical efforts to build quantitative computational models and subsequently neuromorphic devices replicating the exact response of a biological neuron, the authors wrote.

Silicon neurons, synapses, and brain-inspired networks have all been proposed, but these designs werent meant to recapitulate the behavior of biological cells in detail, rather, they were intended to help identify the organizing principles of biology that could be applied to practical devices. The increasing focus on implantable bioelectronics to treat chronic disease is however changing this paradigm and is instilling new urgency in the need for low-power analog solid-state devices that accurately mimic biocircuits.

For their research to design artificial neurons, the researchers successfully modeled and derived equations to explain how neurons respond to electrical stimuli from other nerves. This is a hugely complicated task, as responses are non-linear, such that a doubling of signal strength may not elicit a reaction doubling. The response may be more, or less than double.

The team then designed silicon chips that accurately modeled biological ion channels, and confirmed that their silicon neurons precisely mimicked real, living neurons responding to a range of stimulations. To demonstrate their models the researchers accurately replicated the complete dynamics of hippocampal neurons and respiratory neurons from rats, under a wide range of stimuli. we built six-channel silicon devicesthat faithfully model CA1 hippocampal and respiratory neurons, they stated. The completed models predict the membrane voltage of biological neurons in excellent agreement (9497%) with the membrane voltage oscillations observed in response to 60 different current protocols.

The potential applications are manifold, the researchers believe. For example, were developing smart pacemakers that wont just stimulate the heart to pump at a steady rate but use these neurons to respond in real-time to demands placed on the heartwhich is what happens naturally in a healthy heart, Nogaret stated.

In heart failure for example, neurons in the base of the brain do not respond properly to nervous system feedback, they in turn do not send the right signals to the heart, which then does not pump as hard as it should. For example, the respiratory neurons which we have modeled couple the respiratory and cardiac rhythms and are responsible for respiratory sinus arrhythmia, the authors commented. Loss of this coupling through age or disease is a prognosis for sleep apnoea and heart failure. Therefore, a device that adapts to biofeedback in the same way as respiratory neurons may offer a much needed therapy for heart failure. Our accurate description of the neurobiology within a model derived from silicon physics answers this need.

Other possible applications could be in the treatment of conditions like Alzheimers and neuronal degenerative diseases more generally, Nogaret suggested. Our approach combines several breakthroughs. We can very accurately estimate the precise parameters that control any neurons behavior with high certainty. We have created physical models of the hardware and demonstrated its ability to successfully mimic the behavior of real living neurons. Our third breakthrough is the versatility of our model which allows for the inclusion of different types and functions of a range of complex mammalian neurons.

Study co-author Giacomo Indiveri, PhD, from the University of Zurich and ETF Zurich, commented, This work opens new horizons for neuromorphic chip design thanks to its unique approach to identifying crucial analog circuit parameters.

Added co-author, Julian Paton, PhD, a physiologist at the University of Auckland and the University of Bristol, said, Replicating the response of respiratory neurons in bioelectronics that can be miniaturized and implanted is very exciting and opens up enormous opportunities for smarter medical devices that drive towards personalized medicine approaches to a range of diseases and disabilities.

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Artificial Neurons on a Chip Developed to Treat Chronic Diseases - Genetic Engineering & Biotechnology News

DUBLIN, Dec. 6, 2019 /PRNewswire/ -- The "The State of Personalized/ Precision Medicine" report has been added to ResearchAndMarkets.com's offering.

This report covers current and future challenges for the development and launch of these medicines, focusing on challenges with transitioning these therapies from bench to bedside and demonstrating efficacy in order to secure reimbursement.

Report ScopeDefinition of Personalized/Precision Medicine - Key attributes of personalized/precision medicine, examining and contrasting definitions by regulatory bodies and KOL respondents.

Personalized Medicine-Impact on Healthcare - Key factors to impacting healthcare for patients and stakeholders providing respondent mix by region and organization size.

Marketed Products - Detailed information is provided for key personalized/precision medicines, including case studies for marketed oncology and rare disease therapeutics.

Pipeline Products - The diverse Phase III pipeline is presented, including detailed information on key candidate's mechanism of action and clinical trial parameters.

Investment in Personalized Medicine - Investment in personalized medicine by indication and clinical trial design and approaches are presented, with case studies for three ongoing oncology trials being considered.

Market Opportunities and Challenges - Key areas of opportunity and roadblocks in developing, launching, and gaining reimbursement are analyzed by geography, company size, and seniority level.

Emerging Trends - Critical new trends, including the use of advanced digital technologies, are examined.Key Topics Covered

1. Preface

2. Executive Summary

3. Overview of Personalized/Precision Medicine3.1 Definition3.2 Definition of Personalized/Precision Medicine3.3 KOL and Payer Perspecive3.4 Key Attributes of Personalized Medicine Therapy3.5 Types of Biomarkers and Diagnostics

4. Impact of Personalized Medicine on Healthcare4.1 Personalized Medicine - Impact on Healthcare - Patients4.2 Advantages of Personalized Medicine - Patients4.3 Role of Personalized Medicine in Clinical Unmet Needs4.4 Personalized Medicine - Impact on Healthcare - Drug Developers' Perspective4.5 Advantages of Personalized Medicine to Drug Developers4.6 Personalized Medicine - Impact on Healthcare - Health System

5. Key Marketed Products

6. Pipeline Products

7. Investment in Personalized Medicine

8. Deal-Making Landscape

9. Market Opportunities and Challenges

10. Pricing, Reimbursement, and Regulatory Strategy

11. Personalized Medicine - Emerging Trends

12. Summary of Key Findings

13. Appendix

Companies Mentioned

For more information about this report visit https://www.researchandmarkets.com/r/v886fo

Research and Markets also offers Custom Research services providing focused, comprehensive and tailored research.

Media Contact:

Research and Markets Laura Wood, Senior Manager press@researchandmarkets.com

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The State of Personalized/Precision Medicine in 2019: Impact on Healthcare, Marketed Products, Pipeline Products, Investments, Market Opportunities &...