StemCell Therapy

Significant Clinical Progress Across Target Indications during 2019 and 2020 Year-to-Date

NEW YORK and LONDON, March 31, 2020 - Akari Therapeutics, Plc (Nasdaq: AKTX), a biopharmaceutical company focused on innovative therapeutics to treat orphan autoimmune and inflammatory diseases where complement (C5) and/or leukotriene (LTB4) systems are implicated, today announced financial results for the fourth quarter and full year ended December 31, 2019, as well as recent business highlights.

2019 was a very important year for the company as we generated positive clinical data across all four of our programs. For BP, AKC and HSCT-TMA, the rapid patient response we generally saw in our clinical studies validates these disease targets for nomcaopan where the specific dual action of the drug provides a potential significant differentiation with its inhibition of both the complement (C5) and leukotriene (LTB4) pathways, said Clive Richardson, Chief Executive Officer of Akari Therapeutics. In 2020, we look forward to expanding these programs further and plan on focusing on preparatory work for potential pivotal studies in anticipation of lessening the impact of the COVID-19 pandemic. At the same time we are working with our employees, partners and patients to help ensure their safety and maintain continuity where possible.

Full Year 2019 and Recent Business Highlights

Akaris strategy is to focus on orphan inflammatory diseases with significant unmet medical need, where the role of the complement and leukotriene systems are implicated. Akaris lead programs are in BP, AKC, and HSCT-TMA where clinical data with nomacopan has shown rapid and sustained clinical improvement in patients. These diseases have no approved treatments.

The Company is working with clinical sites and is following regulatory and health agency guidance related to the COVID-19 pandemic to help ensure the safety of its employees and patients. Our BP study has completed recruitment while our AKC study has halted recruitment with around two thirds of patients recruited. We expect delays in opening sites for our HSCT-TMA program. We expect our long-term safety program will shift to being managed on a country by country basis and some disruption is expected.

Phase II clinical trial in patients with BP

In the fourth quarter of 2019, interim Phase II trial results with nomacopan were presented at the 28th European Academy of Dermatology and Venereology (EADV) Congress. The data showed that four of the six patients were classified as at the upper limit of moderate BP. The four patients saw a rapid and significant improvement in symptoms, with a mean 63% decline in BPDAI score and mean 68% decline in blister score by Day 42, with either no or minimal early steroid treatment with one moderate patient having a flare up post Day-28. The data showed nomacopans potential as a possible treatment for BP with the additional and important benefit of reducing steroid use which has multiple adverse effects including a threefold increased risk of mortality. The Phase II trial has completed recruitment, with full data expected in the second quarter of 2020.

During the third quarter of 2019, the FDA granted orphan drug designation for nomacopan for the treatment of BP. The Company is now evaluating pivotal trial designs.

Phase III clinical trial in pediatric patients with HSCT-TMA

Initiated a pivotal Phase III trial for HSCT-TMA with nomacopan following the opening of an IND by the FDA. As a result of the COVID-19 pandemic, although we are looking to continue the process of site openings, we anticipate this will be delayed and hence any enrollment. This two-part Phase III study in pediatric patients with HSCT-TMA is based on guidance from the Companys end-of-Phase II meeting with the FDA. Part A of the trial is a dose confirmation study with the dosing agreed with FDA via their Model Informed Drug Development Program (MIDD). Part B of the trial is a single arm responder-based efficacy study that will follow an interim analysis of Part A and a meeting with the FDA. This devastating condition has an estimated 80% mortality rate in children, at elevated risk of dying who will be recruited to the trial and has no approved treatments. Akari has both FDA fast track pediatric patients and orphan drug designation status for this program.

Phase I/II clinical trial in patients with AKC

In 2019, the Company successfully completed Part A of the Phase I/II clinical trial in severe AKC patients who showed a rapid overall improvement of a mean 55% in the composite clinical score. The nomacopan eye drops were found to be comfortable and well tolerated with no reported drug related serious adverse events. Enrollment in the Part B placebo-controlled efficacy arm of the study has now stopped due to the COVID outbreak, but recruited patients continue to be treated. We anticipate that when the trial closes, we will have data on around two thirds of the target 19 patient study.

During the first quarter of 2020, the Company announced new preclinical data indicating that PAS-nomacopan, the long acting form of the drug, significantly reduced both retinal inflammation and intraocular VEGF. PAS-nomacopan was found to reduce intraocular VEGF levels by as much as the anti-VEGF antibody with 74% (p=0.04) and 68% (p=0.05) reductions respectively, compared to saline control. Furthermore, while clinically assessed inflammation increased in both the control and anti-VEGF groups by 49% and 33%, respectively, PAS-nomacopan treatment resulted in a 9% reduction in inflammation which represents a 58% difference compared to control assessed by retinal fundoscopy (p=0.02). This therapeutic activity across multiple pathogenic pathways (VEGF, inflammation and complement) supports the potential for nomacopan as a new mode of action for the treatment of back of the eye diseases.

PNH program

The Company continues to accumulate positive long-term treatment data, which includes more than 30 cumulative patient-years of data with 14 PNH patients across four clinical trialswith no reported drug related serious adverse events. Interim data from the Phase III CAPSTONE study on the first eight PNH patients who were all transfusion dependent at entry to the CAPSTONE trial show that all four patients randomized to nomacopan were transfusion independent for the first six months of treatment while all four patients on standard of care (SOC) remained transfusion dependent. Recruitment into the Phase III CAPSTONE study has been discontinued, although a PNH program may be re-initiated to potentially take advantage of the new high concentration formulation.

Akari has been granted orphan status from theFDA andthe European Medicines Agency(EMA) for nomacopan for treatment of PNH.

Fourth Quarter and Full Year 2019 Financial Results

A copy of the Companys Annual Report on Form 20-F for the year ended December 31, 2019 has been filed with the Securities and Exchange Commission and posted on the Companys website at http://investor.akaritx.com/financial-information/sec-filings. You may request a copy of the Companys Form 20-F, at no cost to you, by writing to the Financial Controller of the Company at 75/76 Wimpole Street, London W1G 9RT, United Kingdom or by calling the Company at +44 20 8004 0261.

Important Message Regarding COVID-19

Public health epidemics or outbreaks could adversely impact our business. In late 2019, a novel strain of COVID-19, also known as coronavirus, was reported in Wuhan, China. While initially the outbreak was largely concentrated in China, it has now spread to several other countries, including in the United Kingdom and the United States, and infections have been reported globally. In particular, our clinical trial sites are based in areas currently affected by coronavirus. Epidemics such as this can adversely impact our business as a result of disruptions, such as travel bans, quarantines, and interruptions to access the trial sites and supply chain, which could result in material delays and complications with respect to our research and development programs and clinical trials. Moreover, as a result of coronavirus, there is a general unease of conducting unnecessary activities in medical centers. As a consequence, our ongoing trials have been halted or disrupted. It is too early to assess the full impact of the coronavirus outbreak on trials for nomacopan, but coronavirus is expected to affect our ability to complete recruitment in our original timeframe. The extent to which the coronavirus impacts our operations will depend on future developments, which are highly uncertain and cannot be predicted with confidence, including the duration and severity of the outbreak, and the actions that may be required to contain the coronavirus or treat its impact. In particular, the continued spread of the coronavirus globally, could adversely impact our operations and workforce, including our research and clinical trials and our ability to raise capital, could affect the operations of key governmental agencies, such as the FDA, which may delay the development of our product candidates and could result in the inability of our suppliers to deliver components or raw materials on a timely basis or at all, each of which in turn could have an adverse impact on our business, financial condition and results of operation.

About Akari Therapeutics

Akari is a biopharmaceutical company focused on developing inhibitors of acute and chronic inflammation, specifically for the treatment of rare and orphan diseases, in particular those where the complement (C5) or leukotriene (LTB4) systems, or both complement and leukotrienes together, play a primary role in disease progression. Akari's lead drug candidate, nomacopan (formerly known as Coversin), is a C5 complement inhibitor that also independently and specifically inhibits leukotriene B4 (LTB4) activity. Nomacopan is currently being clinically evaluated in four indications: bullous pemphigoid (BP), atopic keratoconjunctivitis (AKC), thrombotic microangiopathy (TMA), and paroxysmal nocturnal hemoglobinuria (PNH). Akari believes that the dual action of nomacopan on both C5 and LTB4 may be beneficial in AKC and BP. Akari is also developing other tick derived proteins, including longer acting versions.

Original post:
Akari Therapeutics Reports Fourth Quarter and Full Year 2019 Financial Results and Business Highlights - PharmiWeb.com

VANCOUVER, Washington, March 31, 2020 (GLOBE NEWSWIRE) CytoDyn Inc. (OTC.QB: CYDY), (CytoDyn or the Company), a late-stage biotechnology company developing leronlimab (PRO 140), a CCR5 antagonist with the potential for multiple therapeutic indications, announced today that the U.S. Food and Drug Administration (FDA) just provided clearance for initiation of a Phase 2 trial with CytoDyns leronlimab to treat COVID-19 patients with mild to moderate indications. The Companys investigational new drug, leronlimab, has been administered to 10 severely ill patients with COVID-19 at a leading medical center in the New York City area under an emergency IND recently granted by the FDA. The treatment with leronlimab is intended to serve as a therapy for patients who experience mild-to-moderate respiratory complications as a result of contracting SARS-CoV-2 causing the Coronavirus Disease 2019 (COVID-19).

The Phase 2 clinical trial is a randomized, double blind, placebo-controlled study to evaluate the efficacy and safety of leronlimab in patients with mild to moderate documented COVID-19 illness and calls for 75 planned patients in up to 10 centers in the United States. Patients enrolled in the trial are expected to have a treatment window of approximately 6 weeks.

Bruce Patterson, M.D., Chief Executive Officer and founder of IncellDx, a diagnostic partner and advisor to CytoDyn, commented, We are very pleased with the FDAs responsiveness to facilitate the commencement of this important Phase 2 trial. In light of the test results of the various immunologic markers from the critically ill patients treated under the emergency IND, we remain hopeful that leronlimab may be therapeutically beneficial to those COVID-19 patients with mild to moderate indications.

Nader Pourhassan, Ph.D., President and Chief Executive Officer of CytoDyn said, The FDA has been very collaborative with our team to accelerate the opportunity to introduce a potentially beneficial treatment to so many patients affected by this horrific pandemic. We hope to complete enrollment of this trial very quickly.

About Coronavirus Disease 2019SARS-CoV-2 was identified as the cause of an outbreak of respiratory illness first detected in Wuhan, China. The origin of SARS-CoV-2 causing the COVID-19 disease is uncertain, and the virus is highly contagious. COVID-19 typically transmits person to person through respiratory droplets, commonly resulting from coughing, sneezing, and close personal contact. Coronaviruses are a large family of viruses, some causing illness in people and others that circulate among animals. For confirmed COVID-19 infections, symptoms have included fever, cough, and shortness of breath. The symptoms of COVID-19 may appear in as few as two days or as long as 14 days after exposure. Clinical manifestations in patients have ranged from non-existent to severe and fatal. At this time, there are minimal treatment options for COVID-19.

About Leronlimab (PRO 140)The FDA has granted a Fast Track designation to CytoDyn for two potential indications of leronlimab for deadly diseases. The first as a combination therapy with HAART for HIV-infected patients and the second is for metastatic triple-negative breast cancer.Leronlimab is an investigational humanized IgG4 mAb that blocks CCR5, a cellular receptor that is important in HIV infection, tumor metastases, and other diseases, including NASH.Leronlimab has completed nine clinical trials in over 800 people, including meeting its primary endpoints in a pivotal Phase 3 trial (leronlimab in combination with standard antiretroviral therapies in HIV-infected treatment-experienced patients).

In the setting of HIV/AIDS, leronlimab is a viral-entry inhibitor; it masks CCR5, thus protecting healthy T cells from viral infection by blocking the predominant HIV (R5) subtype from entering those cells. Leronlimab has been the subject of nine clinical trials, each of which demonstrated that leronlimab could significantly reduce or control HIV viral load in humans. The leronlimab antibody appears to be a powerful antiviral agent leading to potentially fewer side effects and less frequent dosing requirements compared with daily drug therapies currently in use.

In the setting of cancer, research has shown that CCR5 may play a role in tumor invasion, metastases, and tumor microenvironment control. Increased CCR5 expression is an indicator of disease status in several cancers. Published studies have shown that blocking CCR5 can reduce tumor metastases in laboratory and animal models of aggressive breast and prostate cancer. Leronlimab reduced human breast cancer metastasis by more than 98% in a murine xenograft model. CytoDyn is, therefore, conducting aPhase 1b/2 human clinical trial in metastatic triple-negative breast cancer and was granted Fast Track designation in May 2019.

The CCR5 receptor appears to play a central role in modulating immune cell trafficking to sites of inflammation. It may be crucial in the development of acute graft-versus-host disease (GvHD) and other inflammatory conditions. Clinical studies by others further support the concept that blocking CCR5 using a chemical inhibitor can reduce the clinical impact of acute GvHD without significantly affecting the engraftment of transplanted bone marrow stem cells. CytoDyn is currently conducting a Phase 2 clinical study with leronlimab to support further the concept that the CCR5 receptor on engrafted cells is critical for the development of acute GvHD, blocking the CCR5 receptor from recognizing specific immune signaling molecules is a viable approach to mitigating acute GvHD. The FDA has granted orphan drug designation to leronlimab for the prevention of GvHD.

About CytoDynCytoDyn is a late-stage biotechnology company developing innovative treatments for multiple therapeutic indications based on leronlimab, a novel humanized monoclonal antibody targeting the CCR5 receptor. CCR5 appears to play a critical role in the ability of HIV to enter and infect healthy T-cells.The CCR5 receptor also appears to be implicated in tumor metastasis and immune-mediated illnesses, such as GvHD and NASH. CytoDyn has successfully completed a Phase 3 pivotal trial with leronlimab in combination with standard antiretroviral therapies in HIV-infected treatment-experienced patients. CytoDyn plans to seek FDA approval for leronlimab in combination therapy and plans to complete the filing of a Biologics License Application (BLA) in April of 2020 for that indication. CytoDyn is also conducting a Phase 3 investigative trial with leronlimab as a once-weekly monotherapy for HIV-infected patients. CytoDyn plans to initiate a registration-directed study of leronlimab monotherapy indication. If successful, it could support a label extension. Clinical results to date from multiple trials have shown that leronlimab can significantly reduce viral burden in people infected with HIV with no reported drug-related serious adverse events (SAEs). Moreover, a Phase 2b clinical trial demonstrated that leronlimab monotherapy can prevent viral escape in HIV-infected patients; some patients on leronlimab monotherapy have remained virally suppressed for more than five years. CytoDyn is also conducting a Phase 2 trial to evaluate leronlimab for the prevention of GvHD and a Phase 1b/2 clinical trial with leronlimab in metastatic triple-negative breast cancer. More information is atwww.cytodyn.com.

Forward-Looking StatementsThis press releasecontains certain forward-looking statements that involve risks, uncertainties and assumptions that are difficult to predict. Words and expressions reflecting optimism, satisfaction or disappointment with current prospects, as well as words such as believes, hopes, intends, estimates, expects, projects, plans, anticipates and variations thereof, or the use of future tense, identify forward-looking statements, but their absence does not mean that a statement is not forward-looking. The Companys forward-looking statements are not guarantees of performance, and actual results could vary materially from those contained in or expressed by such statements due to risks and uncertainties including: (i)the sufficiency of the Companys cash position, (ii)the Companys ability to raise additional capital to fund its operations, (iii) the Companys ability to meet its debt obligations, if any, (iv)the Companys ability to enter into partnership or licensing arrangements with third parties, (v)the Companys ability to identify patients to enroll in its clinical trials in a timely fashion, (vi)the Companys ability to achieve approval of a marketable product, (vii)the design, implementation and conduct of the Companys clinical trials, (viii)the results of the Companys clinical trials, including the possibility of unfavorable clinical trial results, (ix)the market for, and marketability of, any product that is approved, (x)the existence or development of vaccines, drugs, or other treatments that are viewed by medical professionals or patients as superior to the Companys products, (xi)regulatory initiatives, compliance with governmental regulations and the regulatory approval process, (xii)general economic and business conditions, (xiii)changes in foreign, political, and social conditions, and (xiv)various other matters, many of which are beyond the Companys control. The Company urges investors to consider specifically the various risk factors identified in its most recent Form10-K, and any risk factors or cautionary statements included in any subsequent Form10-Q or Form8-K, filed with the Securities and Exchange Commission. Except as required by law, the Company does not undertake any responsibility to update any forward-looking statements to take into account events or circumstances that occur after the date of this press release.

CYTODYN CONTACTSInvestors:Dave Gentry, CEORedChip CompaniesOffice: 1.800.RED.CHIP (733.2447)Cell: 407.491.4498dave@redchip.com

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FDA Clears CytoDyn's Phase 2 Randomized Trial to Treat Mild-to-Moderately Ill Coronavirus Patients with Leronlimab; Enrollment to Begin Immediately -...

WALTHAM, MACoronavirus that causes COVID-19 disease is the worst pandemic the world has ever seen. If you are confused with the overwhelming information about the virus, you may want to watch this video interview with Dr. Sailaja Reddy, MD, IFMCP, ABoIM, ABOM, CCMS.

In an exclusive video interview with INDIA New England News, Dr. Reddy explains coronavirus, how it is different from other viruses, how we can prevent it from infection, how it infects and how to take care after recovery from COVID-19.

To watch the full interview, please click here or on the image below.

Dr. Reddy educates, supports, treats and transforms peoples lives by getting to the root cause of illness using the best of science and nature. Dr. Reddy is functional and integrative medicine specialist, amd is founder of RootHealthMD in Framingham, Mass, focusing on reversal of chronic diseases and supports the development of vibrant longevity.

Dr. Reddy did her residency at St. Elizabeths Hospital and received advanced training in obesity medicine, Integrative medicine and culinary medicine. She is a certified physician through Institute of Functional Medicine.

Immunity, says Dr. Reddy, is a defense system that has biological structure and process within humans that protects against diseases.

Our body builds immunity through healthy lifestyle. Diet, genetics and environment play a major role, says Dr. Reddy, adding that one should change lifestyle in order to build immunity.

A diet that is nutrient rich, with low sugar, good quality protein and less processed will strengthen your immune system, says Dr. Reddy. The latest science suggests that being fit boosts our immune systems, and that even a single workout can amplify and improve our ability to fight off germs.

PS: This information is for information purposes only. If you have any discomfort, please consult your physician immediately.

Originally posted here:
Everything You Need to Know About Coronavirus: Before, During, After and How to Build Immunity Against Future Viruses - India New England

As the coronavirus spreads around the globe, it has mutated in tiny, subtle ways. Those mutations arent cause for concern, and so far, dont appear to be making the virus any more or less dangerous. But scientists can use those slight changes to track the virus from person to person, and location to location.

If we identify a new outbreak cluster in one state, and theres a question of whether its related to a previous cluster or not, the small mutational changes can help you figure out if theyre connected, says Patrick Boyle, a synthetic biologist at Ginkgo Bioworks.

The coronavirus is made up of around 29,000 building blocks of genetic material called nucleotides. Like other biotechnology companies and labs, Ginkgo has the technology to take a sample of the virus and read out the full sequence of those nucleotides. For the most part, the sequence will be the same in each sample. But the virus makes copies of itself within a human host, and sometimes, it can make mistakes switching one or two nucleotides out for another. The version of the virus with those changes can then be passed on when that person infects someone else.

Ginkgo is repurposing its systems, which normally dont sequence viruses, to analyze as many samples of the coronavirus as possible. The goal is to help build out the maps that show how the virus jumped from one person to the next. Theyre hoping to scale up to be able to publish the full genetic sequence of 10,000 virus samples a day.

Despite the skyrocketing numbers of COVID-19 cases in the US, only a limited number of virus samples collected in the country have been sequenced in full. Scientists have more sequences from Washington state than other places. Consequently, they know more about the trajectory of the outbreak in Washington than they know about outbreaks in other states.

Some of that genetic data is how Trevor Bedford, a virologist at the Fred Hutchinson Cancer Research Center, was able to link a case of COVID-19 diagnosed on February 27 in Washington to a case that was diagnosed in late January in the state indicating that the virus had been circulating locally, and undetected, for that entire time. It also showed that the January case sparked a cluster of illnesses that spread through the community.

Other states are starting to do the same kind of detective work, using genetic sequences to help clarify their outbreaks. An analysis of nine virus samples collected in Connecticut showed that the some were related to viruses found in Washington state, which suggests that the coronavirus was spreading domestically, not being repeatedly brought in from other countries. The analysis has not yet been peer-reviewed or published. Other preliminary research examined virus samples from northern California, and found that the coronavirus was introduced to the area at multiple points.

One challenge in expanding the number of virus sequences available, Boyle says, is obtaining patient samples to analyze. Labs in the US and other countries that are running tests for the virus receive hundreds or thousands of patient samples each day. But the focus of those labs is checking a sample to see if the coronavirus is there and the patient has COVID-19 or if it isnt. The emphasis on testing and diagnosing patients is critical to track the pandemic, Boyle says.

The problem is, it only gives you a positive or negative answer, he says. Tests dont provide any extra information about the particular virus in each patient. Ginkgo plans to partner with testing labs, so that they can take a closer look at the virus in a patient sample after the testing is done. Other labs and groups worldwide are embarking on similar projects: a research consortium in the United Kingdom, for example, has over $20 million in funding to sequence samples. Boyle says that Gingko is coordinating with some other labs interested in this work.

Theyre also making sure that they can access the chemicals and other supplies they need to run the genetic analysis, Boyle says. We want to make sure that our supply stream is not competing with the supply stream that keeps the testing running.

Expanding the number of coronavirus sequences available will give scientists a picture of the outbreak, in the US and around the world. Along with testing, its one way scientists can keep track of the viruss movements and help to rein it in.

Read the original post:
Genetic analysis of the coronavirus gives scientists clues about how its spreading - The Verge

Humanity really has only two options to confront the coronavirus pandemic currently sweeping the planet. The first is to mount a rolling program of lockdowns and other drastic social distancing strategies to restrain the pace of the virus epidemic, with a view to gradually building up natural herd immunity among the human population.

That strategy, especially if combined with successful anti-viral drug treatments and a massively upscaled testing effort, should give some relief. But it would come at the likely cost of many millions of deaths and incalculable worldwide economic damage, hitting especially hard in countries with little resilience and limited healthcare infrastructure.

The second approach is to develop a vaccine, and to do so as rapidly as possible. A fully effective vaccine would not just tame COVID-19 but possibly eradicate it altogether as the world successfully did with smallpox and is on the verge of doing with polio (both also viral diseases).

These two approaches will most likely be concurrent: the first will buy us time, while the second provides an exit strategy from a constant pattern of repeating lockdowns and travel restrictions that could otherwise go on for years.

With the current total of confirmed cases rapidly closing in on one million worldwide, the true picture is most likely that many tens of millions of people have already caught COVID-19. Humanitys most desperate challenge, therefore, is to find an effective vaccine.

Fortunately, science is already stepping up. History was made on March 16, when the first clinical trial volunteer was injected with an investigational vaccine for coronavirus at the Kaiser Permanente Washington Health Research Institute in Seattle.

The volunteer was mother-of-two Jennifer Haller, a 43-year-old Seattle resident who told National Public Radio that she wanted to do something because theres so many Americans that dont have the same privileges that Ive been given.

The vaccination was produced by Moderna, with the first batch being delivered to the US National Institutes of Health a remarkable 42 days after the viral genome was first sequenced in China.

This Phase 1 trial does not yet test the efficacy of the vaccine against COVID-19. Carried out over six weeks among a group of 45 healthy adult volunteers aged between 18 and 55, it will test the basic safety of the proposed vaccine and its ability to stimulate an immune response in the human body.

Although the Phase 1 trial will continue with the Seattle-area recruits being monitored for a whole year, the urgency of the global situation means that the collaborators will likely rush to Phase 2 at the same time, testing the ability of the vaccine to prevent infection by the novel coronavirus SARS-CoV-2 that causes COVID-19.

The Moderna vaccine trial is a world first not just for the particular disease target but because it is one of a whole new potential class of vaccines that employ messenger RNA (mRNA) to program human cells to produce the viral proteins that trigger an immune response, rather than injecting proteins or viral particles directly, as have most previous vaccines.

This natural role of mRNA is why Modernas approach is so quick. Normal vaccines have to be produced from actual viruses, which are grown within chicken eggs and then refined into sufficient quantities to be directly injected once weakened or killed into the human body. This takes months, at a minimum, and is difficult to scale quickly.

For the mRNA approach, all that was needed was the correct viral genetic sequence, which in the case of SARS-CoV-2 encodes for the spike proteins that enable the virus to gain entry into human respiratory cells. This genetic sequence for the viral protein can then be encoded into mRNA synthetically generated in a lab a rapid process that is easy to scale.

Thats the good news. The bad news is that the mRNA approach, while undoubtedly quick and versatile, is so new that it has yet to be fully proven in any vaccine in either humans or animals. Some tests have shown efficacy against rabies, for example, but others have shown little lasting immune response.

The mRNA approach is therefore a moon-shot rather than a marathon. Even so, Moderna is optimistic enough to already be making plans to produce millions of doses intended for health workers initially as early as this fall.

Other companies and partnerships are also racing to develop a vaccine using the same mRNA approach. One of these, the German firm CureVac, generated so much interest that President Trump reportedly tried to acquire it in order to ensure any potential vaccine would be available to Americans first.

Like Moderna, CureVacs efforts are supported financially by CEPI the international Coalition for Epidemic Preparedness Innovations, which has raised over $700 million from governments around the world and philanthropic foundations like the Bill & Melinda Gates Foundation (which also supports the Cornell Alliance for Science) and Wellcome.

While Moderna has been able to restart vaccine projects originally intended for MERS and SARS, CureVac has already achieved some success with an mRNA vaccine against rabies virus in humans. In a Phase 1 trial doses as low as a millionth of a gram of mRNA vaccine were sufficient to fully protect humans against rabies, it reported in January.

Such small doses offer major promise for immunizing huge numbers of people if CureVac is able to achieve the same success with SARS-CoV-2 as it has with rabies and move rapidly into Phase 2 trials to further demonstrate real efficacy.

Also in Germany, BioNTech and Pfizer are racing to shift their mRNA vaccine work from influenza to SARS-CoV-2, and are aiming to start clinical trials as soon as April. As part of a broader collaboration, BioNTech has already demonstrated that an mRNA vaccine protected mice and non-human primates against Zika virus, raising hopes for similar effectiveness against COVID-19.

RNAs double-stranded cousin, DNA, is also being deployed in a novel but equally promising vaccine system against the coronavirus. The approach is related, but rather than injecting mRNA directly into cells so that it can produce viral proteins, DNA is inserted, which in turn produces mRNA inside cells to do the same job.

This DNA is not intended to integrate into the genome of the target cell in humans indeed if this happens, damaging mutations might occur. Instead, DNA is formed into circular plasmids which operate separately to the integral genetic material inside a cells nucleus. Like genomic DNA however, these plasmids are read and transcribed via mRNA into viral proteins which can then prime the bodys immune system against a later invasion by the real virus.

The US-based Inovio Pharmaceuticals announced on 12 March that it had received a grant of $5 million from the Bill & Melinda Gates Foundation to accelerate the testing of a DNA vaccine for COVID-19, with a view to starting Phase 1 clinical trials in April.

Inovio has another advantage: its DNA vaccine INO-4700 was the only vaccine candidate against MERS to progress to Phase 2 trials demonstrating, at least initially, the potential feasibility of the DNA approach. The US Department of Defense with an eye to protecting its military personnel all over the world against COVID-19 has pumped another $11.9 million into INO-4800. The company has also demonstrated protection in early trials using its DNA vaccine against Chikungunya, Zika and influenza viruses.

CEPI is not putting all its eggs in one basket, however. As well as DNA and RNA systems, another promising approach for a COVID-19 vaccine is to use a genetically engineered measles vaccine a strategy supported by a $5 million CEPI grant split between collaborating institutions Themis in Vienna, Institut Pasteur in France and the University of Pittsburghs Center for Vaccine Research.

This takes the live attenuated measles virus vaccine a vaccine with a long history of safe use, having been used to immunize billions of children over the last 40 years and uses reverse genetics technology to insert new genes coding for proteins expressed by other viruses. These then induce an immune response against the new virus whose genetic material has been introduced.

The research team aims to have a COVID-19 candidate vaccine ready for animal testing as soon as April, with wider tests in human volunteers by the end of the year.

Measles virus is not the only candidate for the vector approach. Chinese scientists have reported that they are about to proceed to Phase I human trials with a vaccine candidate starting at the pandemics epicenter in Wuhan. The scientists have genetically engineered a replication-defective adenovirus type 5 (Ad5) as a vector to express the SARS-CoV-2 spike protein, with the resulting vaccine candidate named Ad5-nCoV.

This is perhaps the easiest approach, as all that has to happen is for the engineered harmless adenovirus to infect patients in order to trigger the production of antibodies which should be effective against invading novel coronavirus too. The Chinese company CanSion Biologics has successfully demonstrated this approach with another fully completed vaccine against Ebola, Ad5-EBOV, which is already on the market in China.

A more tried-and-tested approach already widely used to produce flu vaccines is to grow viral proteins directly: these are then injected as a vaccine into human patients so that the immune system is already primed against the real pathogen when it attempts to infect the body. Usually chicken eggs are used, but to speed things up insect cell lines are becoming the preferred option for the coronavirus pandemic.

Here genetics is again an important component: the company Novavax uses a baculovirus vector to genetically engineer an insect cell line originally isolated decades ago from the ovaries of the fall armyworm. The baculovirus transports genes into the insect cells, which program them to manufacture viral proteins that are correctly folded and biologically active, more reliably enabling the human immune system to produce antibodies against them.

According to Novavax, its resulting recombinant protein nanoparticles then self-assemble into a structure that approximates the actual virus, helping enhance the immune response. It claims to have already tested this system in RSV virus, a recalcitrant pathogen that has so far resisted attempts at a vaccine. This approach looks promising enough that CEPI has pumped $4 million in so far with a view to launching Phase I trials by late spring 2020.

In a similar way, the company Sanofi is taking a snippet of genetic code from SARS-CoV-2 and splicing it also via baculovirus into insect cell lines. Its advantage, made in a pitch to the US government that resulted in a big cash injection, is that it already has an FDA-approved facility that could make 600 million doses a year of any resulting vaccine.

Plants can also be engineered to produce viral proteins. The company Medicago is working with genetically modified tobacco plants with this aim in mind. To speed things up, instead of adding new genes to the nucleus of cells and regenerating entire plants from these single cells (as happens with conventional plant genetic engineering), it uses the Agrobacterium vector in a vacuum to transfer recombinant DNA directly into the nucleus of fully-grown leaf cells. This DNA enables the production of the desired viral proteins without ever being integrated into the genome, enabling proteins to be harvested from transformed leaves within a matter of days.

Using this system, Medicago claims to have produced a virus-like particle of the coronavirus within just 20 daysof the SARS-CoV-2 genetic sequence becoming available. The government of Canada quickly put millions of dollars behind the effort as a result.

Astonishingly, given that the coronavirus pandemic is now threatening to devastate societies and economies around the planet on a scale second only to a world war, this effort is still short of cash. CEPI has issued an urgent call for funding, seeking to raise $2 billion: it says just $375 billion by the end of March would enable four-to-six vaccine candidates to move rapidly towards phase 2/3 trials.

Scientists are also hoping desperately that SARS-CoV-2 does not rapidly mutate as influenza viruses tend to do, which would likely reduce the effectiveness of any single vaccine. So far, according to researchers studying 1,000 samples of the virus from around the world, this seems not to be the case.

This means that the race to find a vaccine, and to do so in sufficient time to salvage the situation before the world tips into an economic depression and millions of people die, has a decent chance of success and that any successful vaccine would likely confer lasting immunity.

Meanwhile, all of humanity is waiting. And if the scientists do succeed in this urgent challenge, it will very likely be due to modern genetics. Though genetic engineering was once a dirty word, it now could literally help save the world.

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Science to the rescue? How modern genetics could help save the world from coronavirus - Alliance for Science - Alliance for Science

Demonstrates broad biodistribution, including across the blood-brain barrier into the central nervous system, and into skeletal muscle, in non-human primates (NHPs) after systemic administration

Durable and therapeutically relevant drug concentrations achieved in NHPs after single intravenous dose

Potent cell-based activity and allele-specific enrichment in patient-derived cell lines

Platform validation data supports expansion of the therapeutic pipeline into new organ systems previously unreachable with first-generation antisense oligonucleotide technology

Management to hold a conference call today at 8 a.m. ET

PITTSBURGH, March 31, 2020 (GLOBE NEWSWIRE) -- NeuBase Therapeutics, Inc. (Nasdaq:NBSE) (NeuBase or the Company), a biotechnology company developing next-generation antisense oligonucleotide (ASO) therapies to address genetic diseases, today announced positive preclinical data from its pharmacokinetics studies in non-human primates (NHPs) and in vitro pharmacodynamics data in patient-derived cell lines. NeuBase believes these data validate the key advantages of the proprietary NeuBase peptide-nucleic acid (PNA) antisense oligonucleotide (PATrOL) platform and support the Companys decision to advance the development of its Huntingtons disease (HD) and myotonic dystrophy type 1 (DM1) programs, as well as the potential expansion of its therapeutic pipeline into other indications.

Dr. George Church, professor of genetics at Harvard Medical School and member of the National Academy of Sciences, stated, Given the activity and broad biodistribution observed in these studies and the potential for easier target definition, I believe the PATrOL technology may have a potent impact on the future of drug development and treatment of genetic diseases.

Non-Human Primate Pharmacokinetic Study

Quantitative whole-body autoradiography was performed on NHPs.A PATrOL-enabled compound was radio-labeled, and theresulting material was injected into NHPs at 5 mg/kg via a bolus tail vein injection. At four hours, twelve hours, and seven days post-dosing, NHPs were sacrificed andsectioned into 40 m slices.Slices were exposed to autoradiography imaging plates alongside a dilution series of radioactive PNA in whole blood.Upon imaging, the dilution series enabled an analysis of the amount of compound in each of the tissues. In addition, prior to sacrifice, whole blood, urine, and feces were collected from the NHPs at specified timepoints.The major conclusions from this study include:

Rapid uptake of compound out of the bodys circulation after systemic intravenous administration, with a half-life in circulation of approximately 1.5 hours;

Compound penetrates every organ system studied, including the central nervous system and skeletal muscle;

Compound crosses the blood-brain barrier and into the key deep brain structures, including the caudate, supporting a key capability for the development of the Companys lead program in HD; Delivery of the compound to skeletal muscle, the primary organ system that is affected in DM1;Because both HD and DM1 have manifestations outside of the primary affected organ, the broad biodistribution of the compounds may enable a potential whole-body therapeutic solution in both indications.

96% of administered compound remained in vivo after a one-week period (latest timepoint tested);Redistribution over one week after administration between organ systems enriches concentrations in key brain regions up to two-fold, including in those deep brain structures most relevant for HD;Retention of ~90% of compound concentrations achieved in skeletal muscle over the course of one-week post-single-dose administration; and

Patient-Derived Huntingtons Cell Line Pharmacodynamic Studies

Multiple Huntingtons disease candidate compounds were incubated with HD-derived cells and assayed for their toxicity and their ability to selectively knock down mutant huntingtin protein (mHTT) expression by engaging with the CAG repeat expansion in the huntingtin (HTT) gene transcript. Multi-well plates were seeded with cells and candidates were added to the culture at various concentrations.Cells were grown for three days and thereafter assayed for cell death.Cell pellets were also collected, lysed, and run on gradient SDS-PAGE gels.Following the transfer of the proteins to a membrane, the membrane was probed with anti-huntingtin and anti-beta-actin antibodies.Secondary antibodies were used to image the immunoblots.The beta-actin bands were used to normalize the amount of protein across the wells.The amounts of mutant and wild type huntingtin protein in treated cells were compared to untreated cells to determine the level of knockdown.The major conclusions from this study include:

Story continues

Activity in engaging target disease-causing transcripts and knocking-down resultant malfunctioning mHTT protein levels preferentially over normal HTT protein knock-down; and

Dose limiting toxicities were not observed relative to a control either at or above the doses demonstrating activity in human cells in vitro.

In addition, PATrOL enabled compounds were generally well-tolerated in vivo after systemic administration, both after single dose administration in NHPs and multi dose administration in mice for over a month.

We believe the PATrOL platform has the potential to create drugs that are easy for patients to take at infrequent intervals after they have tested positive for a genetic disease but before symptoms emerge, said Dietrich Stephan, Ph.D., chief executive officer of NeuBase. We believe the best way to effectively manage degenerative genetic diseases is to get ahead of the disease process, and we believe that can only be achieved with early diagnosis coupled with well-tolerated, effective, and easily administered therapies.

Dr. Robert Friedlander, chief medical officer of NeuBase and member of the National Academy of Medicine, stated, An allele specific approach that can be systemically administered and cross the blood brain barrier would be an ideal drug profile for many untreatablegenetic diseases.I believe that NeuBase is moving towards realizing this goal.

The intersection of the NHP pharmacokinetic data and the in vitro patient-derived pharmacodynamic data provides a roadmap to create a pipeline of therapeutic candidates which can reach target tissues of interest after systemic administration and achieve the desired activity at that dose. NeuBase believes the data from these studies support the advancement of the Companys HD and DM1 programs into lead optimization and subsequent IND-enabling studies, as well as provide a roadmap for the future expansion of the Companys therapeutic pipeline into other indications, including oncology.

Dr. Sam Broder, former Director of the National Cancer Institute of the National Institutes of Health and member of the National Academy of Sciences, stated, I believe that the NeuBase strategy of targeting transcripts before they become dangerous mutant proteins has the potential to deliver a dramatic improvement in our collective capabilities to effectively treat a wide range of genetic diseases, including some of the most deadly cancers, by targeting driver mutations and accelerating immunotherapy capabilities.

Conference Call

NeuBase Therapeutics, Inc. will discuss these data and next steps for development during a webcasted conference call with slides today, March 31, 2020, at 8:00 a.m. ET. The live and archived webcast of this presentation can be accessed through the IR Calendar page on the Investors section of the Companys website, http://www.neubasetherapeutics.com. The dial-in details for the call are 877-451-6152 (domestic) or +1-201-389-0879 (international), and conference ID: 13701118. The archived webcasts will be available for approximately 30 days following the presentation date.

About NeuBase Therapeutics

NeuBase Therapeutics, Inc. is developing the next generation of gene silencing therapies with its flexible, highly specific synthetic antisense oligonucleotides. The proprietary NeuBase peptide-nucleic acid (PNA) antisense oligonucleotide (PATrOL) platform is designed to permit the rapid development of targeted drugs, thereby potentially increasing the treatment opportunities for the hundreds of millions of people affected by rare genetic diseases, including those that can only be treated through accessing of secondary RNA structures. Using PATrOL technology, NeuBase aims to first tackle rare, genetic neurological disorders.

Safe Harbor Statement under the Private Securities Litigation Reform Act of 1995

This press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act. These forward-looking statements include, among other things, statements regarding the Companys goals and plans and the Companys pharmacokinetics and pharmacodynamics studies. These forward-looking statements are distinguished by use of words such as will, would, anticipate, expect, believe, designed, plan, or intend, the negative of these terms, and similar references to future periods. These views involve risks and uncertainties that are difficult to predict and, accordingly, our actual results may differ materially from the results discussed in our forward-looking statements. Our forward-looking statements contained herein speak only as of the date of this press release. Factors or events that we cannot predict, including those described in the risk factors contained in our filings with the U.S. Securities and Exchange Commission, may cause our actual results to differ from those expressed in forward-looking statements. The Company may not actually achieve the plans, carry out the intentions or meet the expectations or projections disclosed in the forward-looking statements, and you should not place undue reliance on these forward-looking statements. Because such statements deal with future events and are based on the Companys current expectations, they are subject to various risks and uncertainties and actual results, performance or achievements of the Company could differ materially from those described in or implied by the statements in this press release, including: the Companys plans to develop and commercialize its product candidates; the Companys plans to commence clinical trials in Huntingtons disease and myotonic dystrophy type 1 and to potentially expand the pipeline into other indications; the utility of the preclinical data generated in existing studies performed by the Company in determining the results of potential future clinical trials and of the potential benefits of the PATrOL platform technology; the timing of initiation of the Companys planned clinical trials; the timing of the availability of data from the Companys clinical trials; the timing of any planned investigational new drug application or new drug application; the Companys plans to research, develop and commercialize its current and potential future product candidates; the clinical utility, potential benefits and market acceptance of the Companys current and potential future product candidates; the Companys commercialization, marketing and manufacturing capabilities and strategy; the Companys ability to protect its intellectual property position; and the requirement for additional capital to continue to advance these product candidates, which may not be available on favorable terms or at all, as well as those risk factors in our filings with the U.S. Securities and Exchange Commission. Except as otherwise required by law, the Company disclaims any intention or obligation to update or revise any forward-looking statements, which speak only as of the date hereof, whether as a result of new information, future events or circumstances or otherwise.

NeuBase Investor Contact:Dan FerryManaging DirectorLifeSci Advisors, LLCDaniel@lifesciadvisors.comOP: (617) 535-7746

NeuBase Media Contact:Travis Kruse, Ph.D.Russo Partners, LLCtravis.kruse@russopartnersllc.comOP: (212) 845-4272

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NeuBase Therapeutics Announces Positive, Preclinical Data Validating its Novel Genetic Therapy PATrOL Platform - Yahoo Finance

Sarah Cutshall | Visual Editor

Not leaving home leaves plenty of time to spend with your pets.

Were sure youre up to date with all the bad news thats been circulating. Stocks are down. Schools are closed. People are under orders to stay home, etc. With all the depressing news of the last month or two, were almost certain youve been missing out on some of the good things that have happened around the world. That being said, the editorial board presents you with 10 positive things to briefly distract you from all the negative headlines.

You heard us right, ladies and gents. Jack Black is on TikTok. Whether youre a fan of his or not, this is probably going to be a wild and entertaining ride.

Across the world, people are recovering from COVID-19. Wuhan, China the site of the first outbreak is largely on the mend. Meanwhile, in Italy, a 101-year-old man recovered from the virus and was released from the hospital he was being treated in. Elsewhere, people are making similar recoveries.

Thats right college students who previously barely had time to eat, let alone cook, are now standing in front of the stove with a recipe pulled up on their phone screens. What exactly are we cooking? Well, it seems like the top choice is bread. Our take on this: nothing is more comforting than bread thats fresh out of the oven.

Yeah. That Toxic. On March 20, the one and only Andrew Hozier Byrne covered Toxic by Britney Spears on an Instagram live concert. We dont need to explain why this is positive.

Turns out even a global pandemic cant stop love. A couple in New York City got married in the street while their friend officiated from a fourth-floor window. Similarly, in Pittsburgh, a couple got married on their front porch with their neighbors as witnesses standing six feet away, of course.

Things are looking up, especially when it comes to the weather! As spring comes in, the outside world promises to get warmer and unless youre living in Pittsburgh sunnier.

With many places under orders to stay home, pets are bound to be less lonely. Their owners are at home with them, which is basically what dogs have been dreaming about forever. Cats could probably care less about this news, though they probably dont mind having their owners around to sit on.

Animal shelters have seen a large increase in people interested in fostering pets. This is great for both the pets and the people fostering them as the pets get to live with a family rather than in a shelter, and the family gets to experience the joys of having a pet.

Two years after he received a stem cell treatment to cure HIV, Adam Castillejo is still free from the virus. While doctors say that the treatment he received was high-risk and not a widespread solution, this is still by and large good news hopefully, we are moving towards finding a less invasive and more widely accessible cure.

Since the Montreal Protocol was enacted in 1987, the ozone layer has been on the mend. The protocol phased out numerous ozone depleting substances such as chlorofluorocarbons, and its clear now that this made a positive change for the ozone layer. The atmospheric presence of these substances is declining, allowing the ozone layer to heal itself.

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Editorial: Top 10 positive things going on right now - University of Pittsburgh The Pitt News

NEW YORK, March 31, 2020 (GLOBE NEWSWIRE) -- Incysus Therapeutics, Inc. (Incysus), a clinical-stage biopharmaceutical company focused on delivering an innovative gamma-delta () T cell immunotherapy for the treatment of cancers, today announced that the Company will present at the 2020 NYC Spring Oncology Investor Conference being held on March 31st and April 1st. William Ho, President and Chief Executive Officer of Incysus Therapeutics, will present a company update. With the on-going COVID-19 concerns, the Companys presentation will be live broadcast over a premier, private, and secure Zoom conference account. Please see below for more information.

About The NYC Oncology Investor ConferenceThe NYC Oncology Investor Conference brings together leading life science and oncology venture capitalists, family offices, lawyers, pharma executives, startup public and private cancer companies and cancer foundations for a discussion on trends, opportunities and risks in oncology investing (www.oncologyinvestorconference.com).

About Incysus Therapeutics, Inc.Incysus is focused on delivering a novel off-the-shelf cell therapy for the treatment of cancer. By using genetically modified gamma-delta () T cells, the Companys technology addresses the challenges that immunotherapies face targeting cold, low mutation cancers. Incysus immuno-oncology programs include activated and gene-modified adoptive cellular therapies that protect cells from chemotherapy and allow novel combinations to disrupt the tumor microenvironment and more selectively target cancer cells. The Companys first clinical program delivering a genetically modified T cell for the treatment of newly diagnosed glioblastoma (GBM) can be found here: (NCT04165941) and its second clinical program with an allogeneic T cell therapy for leukemia and lymphoma patients undergoing allogeneic stem cell transplantation can be found here: (NCT03533816). For more information about the Company and its programs, visitwww.incysus.com.

Forward Looking StatementsCertain statements herein concerning the Companys future expectations, plans and prospects, including without limitation, the Companys current expectations regarding its business strategy, product candidates, and clinical development process and timing, constitute forward-looking statements. The use of words such as may, might, will, should, expect, plan, anticipate, believe, estimate, project, intend, future, potential, or continue, the negative of these and other similar expressions are intended to identify such forward looking statements. Such statements, based as they are on the current expectations of management, inherently involve numerous risks and uncertainties, known and unknown, many of which are beyond the Companys control. Consequently, actual future results may differ materially from the anticipated results expressed in such statements. In the case of forward-looking statements regarding investigational product candidates and continuing further development efforts, specific risks which could cause actual results to differ materially from the Companys current expectations include: scientific, regulatory and technical developments; failure to demonstrate safety, tolerability and efficacy; final and quality controlled verification of data and the related analyses; expense and uncertainty of obtaining regulatory approval, including from the U.S. Food and Drug Administration; and the Companys reliance on third parties, including licensors and clinical research organizations. Do not place undue reliance on any forward-looking statements included herein, which speak only as of the date hereof and which the Company is under no obligation to update or revise as a result of any event, circumstances or otherwise, unless required by applicable law.

Contact:William HoIncysus Therapeutics, Inc.+1 646.600.6GDTinfo@incysus.comwww.incysus.com

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INCYSUS THERAPEUTICS TO PRESENT AT THE NYC SPRING ONCOLOGY INVESTOR CONFERENCE - Yahoo Finance

Michelle Kuppersmith's doctor recommended a bone marrow biopsy after suspecting she had a rare blood disorder. Though the biopsy was done by an in-network provider at an in-network hospital, Kuppersmith learned she was on the hook for $2,400 for out-of-network genetic profiling. Shelby Knowles for KHN hide caption

Michelle Kuppersmith's doctor recommended a bone marrow biopsy after suspecting she had a rare blood disorder. Though the biopsy was done by an in-network provider at an in-network hospital, Kuppersmith learned she was on the hook for $2,400 for out-of-network genetic profiling.

Michelle Kuppersmith feels great, works full time and exercises three to four times a week. So she was surprised when a routine blood test found that her body was making too many platelets, which help control bleeding.

Kuppersmith's doctor suspected the 32-year-old Manhattanite had a rare blood disorder called essential thrombocythemia, which can lead to blood clots, strokes and, in rare cases, leukemia.

Her doctor suggested a bone marrow biopsy, in which a large needle is used to suck out a sample of the spongy tissue at the center of the patient's hip bone.

Doctors examine the bone marrow under a microscope and analyze the DNA. The procedure allows doctors to judge a patient's prognosis and select treatment, if needed. Kuppersmith had heard the procedure can be intensely painful, so she put it off for months.

The biopsy performed by a provider in her insurance network, at a hospital in her network lasted only a few minutes, and Kuppersmith received relatively good news.

While a genetic analysis of her bone marrow confirmed her doctor's suspicions, it showed that the only treatment she needs, for now, is a daily, low-dose aspirin. She will check in with her doctor every three to four months to make sure the disease isn't getting worse.

All in all, Kuppersmith felt relieved.

Then she got a notice saying her insurer refused to pay for the genetic analysis, leaving her responsible for a $2,400 payment.

The patient: New York resident Michelle Kuppersmith, 32, who is insured by Maryland-based CareFirst Blue Cross Blue Shield. She works as director of special projects at a Washington-based watchdog group. Because she was treated in New York, Empire Blue Cross Blue Shield which covers that region handled part of her claim.

Total amount owed: $2,400 for out-of-network genetic profiling

The providers: Kuppersmith had her bone marrow removed at the Mount Sinai Ruttenberg Treatment Center in New York City, which sent her biopsy sample to a California lab, Genoptix, for testing.

Medical services: Bone marrow biopsy and molecular profiling, which involves looking for genetic mutations

What gives: The field of molecular diagnostics, which includes a variety of gene-based testing, is undergoing explosive growth, said Gillian Hooker, president of the National Society of Genetic Counselors and vice president of clinical development for Concert Genetics, a health IT company in Nashville, Tennessee.

A report from Concert Genetics, a company that helps clients manage genetic testing, found there are more than 140,000 molecular diagnostic products on the market, with 10 to 15 added each day.

The field is growing so quickly that even doctors are struggling to develop a common vocabulary, Hooker said.

Kuppersmith underwent a type of testing known as molecular profiling, which looks for DNA biomarkers to predict whether patients will benefit from new, targeted therapies. These mutations aren't inherited; they develop over the course of a patient's life, Hooker said.

Medicare spending on molecular diagnostics more than doubled from 2016 to 2018, increasing from $493 million to $1.1 billion, according to Laboratory Economics, a lab industry newsletter.

Charges range from hundreds to thousands of dollars, depending on how many genes are involved and which billing codes insurers use, Hooker said.

Based on Medicare data, at least 1,500 independent labs perform molecular testing, along with more than 500 hospital-based labs, said Jondavid Klipp, the newsletter's publisher.

In a fast-evolving field with lots of money at stake, tests that a doctor or lab may regard as state-of-the-art an insurer might view as experimental.

Worse still, many of the commercial labs that perform the novel tests are out-of-network, as was Genoptix.

Stephanie Bywater, chief compliance officer at NeoGenomics Laboratories, which owns Genoptix, said that insurance policies governing approval have not kept up with the rapid pace of scientific advances. Kuppersmith's doctor ordered a test that has been available since 2014 and was updated in 2017, Bywater said.

Although experts agree that molecular diagnostics is an essential part of care for patients like Kuppersmith, doctors and insurance companies may not agree on which specific test is best, said Dr. Gwen Nichols, chief medical officer of the Leukemia & Lymphoma Society.

Tests "can be performed a number of different ways by a number of different laboratories who charge different amounts," Nichols said.

Insurance plans are much more likely to refuse to pay for molecular diagnostics than other lab tests. Laboratory Economics found Medicare contractors denied almost half of all molecular diagnostics claims over the past five years, compared with 5-10% of routine lab tests.

With so many insurance plans, so many new tests and so many new companies, it is difficult for a doctor to know which labs are in a patient's network and which specific tests are covered, Nichols said.

"Different providers have contracts with different diagnostic companies," which can affect a patient's out-of-pocket costs, Nichols said. "It is incredibly complex and really difficult to determine the best, least expensive path."

Kuppersmith said she has always been careful to check that her doctors accept her insurance. She made sure Mount Sinai was in her insurance network, too. But it never occurred to her that the biopsy would be sent to an outside lab or that it would undergo genetic analysis.

She added: "The looming threat of a $2,400 bill has caused me, in many ways, more anxiety than the illness ever has."

The resolution: Despite making dozens of phone calls, Kuppersmith got nothing but confusing and contradictory answers when she tried to sort out the unexpected charge.

An agent for her insurer told her that her doctor hadn't gotten preauthorization for the testing. But in an email to Kuppersmith, a Genoptix employee told her the insurance company had denied the claim because molecular profiling was viewed as experimental.

A spokesperson for New York-based Empire Blue Cross Blue Shield, which handled part of Kuppersmith's claim, said her health plan "covers medically necessary genetic testing."

New York, one of 28 states with laws against surprise billing, requires hospitals to inform patients in writing if their care may include out-of-network providers, said attorney Elisabeth Benjamin, vice president of health initiatives at the Community Service Society, which provides free help with insurance problems.

A spokesperson for Mount Sinai said the hospital complies with that law, noting that Kuppersmith was given such a document in 2018 nearly one year before her bone marrow biopsy and signed it.

Benjamin said that's not OK, explaining: "I think a one-year-old, vague form like the one she signed would not comply with the state law and certainly not the spirit of it."

Instead of sending Kuppersmith a bill, Genoptix offered to help her appeal the denied coverage to CareFirst. At first, Genoptix asked Kuppersmith to designate the company as her personal health care representative. She was uncomfortable signing over what sounded like sweeping legal rights to strangers. Instead, she wrote an email granting the company permission to negotiate on her behalf. It was sufficient.

A few days after being contacted by KHN, Kuppersmith's insurer said it would pay Genoptix at the in-network rate, covering $1,200 of the $2,400 charge. Genoptix said it has no plans to bill Kuppersmith for the other half of the charge.

The takeaway: Kuppersmith is relieved her insurer changed its mind about her bill. But, she said: "I'm a relatively young, savvy person with a college degree. There are a lot of people who don't have the time or wherewithal to do this kind of fighting."

Patients should ask their health care providers if any outside contractors will be involved in their care, including pathologists, anesthesiologists, clinical labs or radiologists, experts said. And check if those involved are in-network.

"Try your best to ask in advance," said Jack Hoadley, a research professor emeritus at Georgetown University. "Ask, 'Do I have a choice about where [a blood or tissue sample] is sent?'"

Ask, too, if the sample will undergo molecular diagnostics. Since the testing is still relatively new and expensive most insurers require patients to obtain "prior authorization," or special permission, said Dr. Debra Regier, a medical geneticist at Children's National Hospital in Washington and an associate with NORD, the National Organization of Rare Diseases. Getting this permission in advance can prevent many headaches.

Finally, be wary of signing blanket consent forms telling you that some components of your care may be out-of-network. Tell your provider that you want to be informed on a case-by-case basis when an out-of-network provider is involved and to consent to their participation.

Bill of the Month is a crowdsourced investigation by Kaiser Health News and NPR that dissects and explains medical bills. Do you have a perplexing medical bill you want to share with us? Tell us about it here.

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Bill Of The Month: Pricey Genetic Test For Essential Thrombocythemia : Shots - Health News - NPR

Universityof Toronto researcherSachdev Sidhuand his collaborators are engineering antibody molecules that can neutralize the novel coronavirus in the body before it invades cells.

Sidhu (left) already leads a differentteam that received supportin the first round of federal funding. The goal of that project is to design antiviral medicines that block viral replication.

With our two funded projects, we are working to develop molecules that can target the virus both inside human cells and on the outside to prevent it from getting in, says Sidhu, who is a professor of molecular genetics in the Faculty of Medicine.

Rini has previously helped to determine how antibodies bind to and inactivate the SARS virus, the coronavirus that caused the outbreak in Asia more than 15 years ago. Also on the team isAlan Cochrane, a professor in the department of molecular genetics and an HIV virologist with expertise in viral RNA processing.

The antibodies will be engineered to block the so-called S-protein that forms spikes on the virus's surface. The spikes lock on to a protein called ACE2 on the surface of human cells to gain entry. Coating viral particles with synthetic antibodies should prevent the spikes from binding to ACE2.

Sidhu and Rini will also engineer antibodies that bind ACE2 to make it inaccessible to the virus. This type of engineered immunity surpasses the capacity of the bodys natural immune system since antibodies that react against self-proteins have been filtered out. If successful, the approach may obviate worries about viral mutations that can render drugs ineffective to new emerging viral strains becausethe host protein ACE2 does not change over time.

Sidhus team has advanced a technology called phage display to rapidly create and select human antibodies with desired biological properties, including blocking the virussspike protein. Over the last decade, his team has created hundreds of antibodies with therapeutic potential some of which are in clinical development through spin-off companiesand large pharmaceutical firms.

The group has demonstrated success with both approaches for inhibiting viral entry, having developed neutralizing antibodies that target the Ebola virus as well as antibodies that target the human host receptor of hantavirus or hepatitis C. Moreover, other research has shown that antibodies targeting SARS, a related virus whose genetic material is over 80 per cent identical to the one causing COVID-19, can clear infection in cells and mice.

Using phage display, in which tiny bacterial viruses called phages are instructed to create vast libraries of diverse antibodies, the team will select the antibodies that can kill the virus in human cells before testing them on mice and, eventually, patients. Experiments on mice could start within three to six months, Sidhu says.

In addition to creating antibodies tailored to the new virus from scratch, the researchers will also modify existing SARS-blocking antibodies so that they attack COVID-19 and provide an additional route to the development of a therapeutic.

Given the global spread of the virus, its possible that it will become endemic and circulate in the population like seasonal flu. And, like the flu, it could mutate into new strains that will evade acquired immunity and the vaccines that are being developed. By generating a panel of different antibodies, the researchers aim to stay one step ahead of the virus.

Our advances in antibody engineering technologiesand access to the complete genomes of the COVID-19 virus and its relatives provides us with an opportunity to create tailored therapeutic antibodies at a scale and speed that was not possible even a few years ago, says Sidhu.

Ultimately, we aim to optimize methods to the point where the evolution of new drugs will keep pace with the evolution of the virus itself, providing new and effective drugs in response to new outbreaks.

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Researchers at U of T developing antibodies to 'neutralize' novel coronavirus before it invades cells - News@UofT

By mining a vast trove of genetic data,researchers at theUniversity of Virginia School of Medicineare enhancing doctors ability to treat cancer, predict patient outcomes and determine which treatments will work best for individual patients.

The researchers have identified inherited variations in our genes that affect how well a patient will do after diagnosis and during treatment. With that information in hand, doctors will be able to examine a patients genetic makeup to provide truly personalized medicine.

Oncologists can estimate how a patient will do based on the grade of the tumor, the stage, the age of the patient, the type of tumor, etc. We found [adding a single genetic predictor] can improve our predictive ability by 5% to 10%, said UVAs Anindya Dutta. Many of the cancers had multiple inherited genetic change that were predictive of outcome, so if we add those in, instead of a 10% increase we might get a 30% increase in our ability to predict accurately how patients will do with our current therapy. Thats amazing.

Dutta, the chair of UVAs Department of Biochemistry and Molecular Genetics, believes reviewing the inherited genetic makeup of a patient can provide similar benefits for predicting outcome and choosing therapy for many, many other conditions, from diabetes to cardiac problems. As such, the approach represents a major step forward in doctors efforts to tailor treatments specifically to the individuals needs and genetic makeup.

The research offers answers to questions that have long perplexed doctors.Every clinician has this experience: Two patients come in with exactly the same cancersame grade, same stage, received the same treatment. One of them does very well, and the other one doesnt, Dutta said.The assumption has always been that there is something about the two that we didnt understand, like maybe there are some tumor-specific mutations that one patient had but the other did not. But it occurred to us that with all this genomic data, there is another hypothesis that we could test.

Instead of a 10% increase we might get a 30% increase in our ability to predict accurately how patients will do with our current therapy. Thats amazing.

- Anindya Dutta

To determine if genetic differences in the patients could be the answer, Dutta and his colleagues did a deep dive into the Cancer Genome Atlas, an enormous repository of genetic information assembled by the National Institutes of Healths National Cancer Institute. The researchers sought to correlate inherited genetic variations with patient outcomes.

This incredibly smart M.D.-Ph.D. student in the lab, Mr.Ajay Chatrath,decided that this was a perfect time to explore this, Dutta recalled. With the help of cloud computing services at UVA, we managed to download all this genomic sequencing data and identify what are known as germline variants not just tumor-specific mutations, but the mutations that were inherited from the parents and are present in all cells of the patient.

The researchers started small, but soon realized how quickly the work could be done and how big the benefits could be. Once we realized this was a very easy thing to do, we went on to do all 33 cancers and all 10,000 patients, and that took another six months, Dutta said. All of this came together beautifully. It was very exciting that every single member in the lab contributed to the analysis.

Dutta is eager to share his findings in hopes of finding collaborators and inspiring researchers and private industry to begin mining the data for other conditions. This is very low-hanging fruit, he said. Germline variants predicting outcome can be applicable to all types of diseases and not just cancer, and [they can predict] responsiveness to all types of therapy, and thats why Im particularly excited.

The researchers have published their findings in the scientific journal Genome Medicine. The studys authors were Chatrath, Roza Przanowska, Shashi Kiran, Zhangli Su, Shekhar Saha, Briana Wilson, Takaaki Tsunematsu,Ji-Hye Ahn, Kyung Yong Lee, Teressa Paulsen, Ewelina Sobierajska, Manjari Kiran, Xiwei Tang, Tianxi Li, Pankaj Kumar, Aakrosh Ratan and Dutta.

The research was supported by the National Institutes of Health, grants R01 CA166054, R01 1094 CA60499, T32 GM007267, AHA 18PRE33990261; and a Cancer 1095 Genomics Cloud Collaborative Support grant. The Seven Bridges Cancer 1096 Genomics Cloud has been funded by the National Cancer Institute, National Institutes of Health.

To keep up with the latest medical research news from UVA, subscribe to theMaking of Medicineblog.

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UVA Finds Way to Improve Cancer Outcomes by Examining Patients' Genes - University of Virginia

NEW YORK, March 31, 2020 /PRNewswire/ -- 2020 World Molecular Diagnostic Analyzer and Reagent Forecasts for 100 Tests: Americas, EMEA, APAC--A 68-Country Analysis--Infectious and Genetic Diseases, Cancer, Forensic and Paternity Testing

Read the full report: https://www.reportlinker.com/p05876987/?utm_source=PRN

This new 68-country survey provides granular data and analysis not available from any other source. The report is designed to help current suppliers and potential market entrants identify and evaluate major business opportunities emerging in the molecular diagnostics market during the next five years.

Highlights

- Supplier sales and market shares in major countries

- Five-year test volume and sales forecasts

- Strategic profiles of market players and start-up firms developing innovative technologies and products

- Emerging technologies

- Review of molecular diagnostic analyzers

- Specific product and business opportunities for instrument and consumable suppliers

Rationale

The molecular diagnostics market is unquestionably the most rapidly growing segment of the in vitro diagnostics industry. The next five years will witness significant developments in reagent systems and automation, as well as introduction of a wide range of new products that will require innovative marketing approaches. The rate of market penetration into routine clinical laboratories, however, will depend on the introduction of cost-effective and automated systems with amplification methods.

In order to successfully capitalize on the opportunities presented by the molecular diagnostics market, many companies are already exploiting new molecular technologies as corporate strategic assets, managed in support of business and marketing strategies. Integrating new technology planning with business and corporate strategies will be one of the most challenging tasks for diagnostic companies during the next five years.

Geographic Regions

Asia-Pacific, Europe, Latin America, Middle East, North America

Country Analyses

Argentina, Australia, Austria, Bahrain, Bangladesh, Belgium, Brazil, Bulgaria, Canada, Chile, China, Colombia, Croatia, Cyprus, Czech Republic, Denmark, Estonia, Finland, France, Germany, Greece, Hong Kong, Hungary, Iceland, India, Indonesia, Iran, Iraq, Ireland, Israel, Italy, Japan, Jordan, Kuwait, Latvia, Lebanon, Lithuania, Malaysia, Malta, Mexico, Myanmar, Netherlands, New Zealand, Norway, Oman, Pakistan, Peru, Philippines, Poland, Portugal, Qatar, Romania, Saudi Arabia, Serbia, Singapore, Slovakia, Slovenia, South Korea, Spain, Sweden, Switzerland, Taiwan, Thailand, UK, United Arab Emirates, USA, Venezuela, Vietnam

Market Segmentation Analysis

- Sales and market shares of key suppliers of molecular diagnostic reagent kits and components in major markets.

Five-year test volume and sales forecasts for major applications, including:

- Infectious Diseases - Forensic Testing- Cancer - Paternity Testing/HLA Typing- Genetic Diseases - Others

- Five-year test volume and sales projections for over 30 NAT assays.

- A comprehensive analysis of the sequencing market, by country and laboratory segment,including:

- Industrial - Academic- Government- Commercial

- Market segmentation analysis, including review of the market dynamics, trends, structure, size, growth and suppliers in major countries.

Product/Technology Review

- Comparison of leading molecular diagnosticanalyzers marketed by Abbott, Beckman Coulter, BD, Bio-Rad, Gen-Probe, Roche, Tecan and other suppliers.

- Extensive review of molecular diagnostic technologies, test formats, detection methodologies, trends in testing automation and over 30 target/signal amplification methods, including:

- PCR - bDNA - SDA - NASBA - TMA - SSSR, and others - LCR

- Universities and research centers developing new molecular diagnostictechnologies and products.

Competitive Assessments

- Extensive strategic assessments of major suppliers and emerging market entrants, including their sales, product portfolios, marketing tactics, collaborative arrangements and new technologies/products in RandD.

- Companies developing and marketing molecular diagnostics products, by test and application.

Opportunities and Strategic Recommendations

Story continues

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2020 World Molecular Diagnostic Analyzer and Reagent Forecasts for 100 Tests: Americas, EMEA, APAC--A 68-Country Analysis--Infectious and Genetic...

Michelle Kuppersmith, 32, feels great, works full time and exercises three to four times a week. So she was surprised when a routine blood test found that her body was making too many platelets, which help control bleeding. Kuppersmiths doctor suspected she had a rare blood disorder called essential thrombocythemia, which can lead to blood clots, strokes and, in rare cases, leukemia.

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Her doctor suggested a bone marrow biopsy, in which a large needle is used to suck out a sample of the spongy tissue at the center of the patients hip bone. Doctors examine the bone marrow under a microscope and analyze the DNA. The procedure allows doctors to judge a patients prognosis and select treatment, if needed. Kuppersmith had heard the procedure can be intensely painful, so she put it off for months.

The biopsy performed by a provider in her insurance network, at a hospital in her network lasted only a few minutes, and Kuppersmith received relatively good news. While a genetic analysis of her bone marrow confirmed her doctors suspicions, it showed that the only treatment she needs, for now, is a daily, low-dose aspirin. She will check in with her doctor every three to four months to make sure the disease isnt getting worse.

All in all, Kuppersmith felt relieved.

Then she got a notice saying her insurer refused to pay for the genetic analysis, leaving her responsible for a $2,400 payment.

The Patient: New York resident Michelle Kuppersmith, 32, who is insured by Maryland-based CareFirst Blue Cross Blue Shield. She works as director of special projects at a Washington-based, nonpartisan watchdog group. Because she was treated in New York, Empire Blue Cross Blue Shield which covers that region handled part of her claim.

Total Amount Owed: $2,400 for out-of-network genetic profiling

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The Providers: Kuppersmith had her bone marrow removed at the Mount Sinai Ruttenberg Treatment Center in New York City, which sent her biopsy sample to a California lab, Genoptix, for testing.

Medical Services: Bone marrow biopsy and molecular profiling, which involves looking for genetic mutations

What Gives: The field of molecular diagnostics, which includes a variety of gene-based testing, is undergoing explosive growth, said Gillian Hooker, president of the National Society of Genetic Counselors and vice president of clinical development for Concert Genetics, a health IT company in Nashville, Tennessee.

A Concert Genetics report found there are more than 140,000 molecular diagnostic products on the market, with 10 to 15 added each day.

The field is growing so quickly that even doctors are struggling to develop a common vocabulary, Hooker said.

Kuppersmith underwent a type of testing known as molecular profiling, which looks for DNA biomarkers to predict whether patients will benefit from new, targeted therapies. These mutations arent inherited; they develop over the course of a patients life, Hooker said.

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Medicare spending on molecular diagnostics more than doubled from 2016 to 2018, increasing from $493 million to $1.1 billion, according to Laboratory Economics, a lab industry newsletter.

Charges range from hundreds to thousands of dollars, depending on how many genes are involved and which billing codes laboratories use, Hooker said.

Based on Medicare data, at least 1,500 independent labs perform molecular testing, along with more than 500 hospital-based labs, said Jondavid Klipp, the newsletters publisher.

In a fast-evolving field with lots of money at stake, tests that a doctor or lab may regard as state-of-the-art an insurer might view as experimental.

Worse still, many of the commercial labs that perform the novel tests are out-of-network, as was Genoptix.

After lining up an in-network provider at an in-network hospital, Kuppersmith pushed back when she got a $2,400 charge for an out-of-network lab. She appealed and won but says, There are a lot of people who dont have the time or wherewithal to do this kind of fighting.

Stephanie Bywater, chief compliance officer at NeoGenomics Laboratories, which owns Genoptix, said that insurance policies governing approval have not kept up with the rapid pace of scientific advances. Kuppersmiths doctor ordered a test that has been available since 2014 and was updated in 2017, Bywater said.

Although experts agree that molecular diagnostics is an essential part of care for patients like Kuppersmith, doctors and insurance companies may not agree on which specific test is best, said Dr. Gwen Nichols, chief medical officer of the Leukemia & Lymphoma Society.

Tests can be performed a number of different ways by a number of different laboratories who charge different amounts, Nichols said.

Insurance plans are much more likely to refuse to pay for molecular diagnostics than other lab tests. Laboratory Economics found Medicare contractors denied almost half of all molecular diagnostics claims over the past five years, compared with 5-10% of routine lab tests.

With so many insurance plans, so many new tests and so many new companies, it is difficult for a doctor to know which labs are in a patients network and which specific tests are covered, Nichols said.

Different providers have contracts with different diagnostic companies, which can affect a patients out-of-pocket costs, Nichols said. It is incredibly complex and really difficult to determine the best, least expensive path.

Kuppersmith said she has always been careful to check that her doctors accept her insurance. She made sure Mount Sinai was in her insurance network, too. But it never occurred to her that the biopsy would be sent to an outside lab or that it would undergo genetic analysis.

She added: The looming threat of a $2,400 bill has caused me, in many ways, more anxiety than the illness ever has.

Kuppersmiths doctor recommended a bone marrow biopsy after suspecting she had a rare blood disorder. Though the biopsy was done by an in-network provider at an in-network hospital, Kuppersmith learned she was on the hook for $2,400 for out-of-network genetic profiling.

The Resolution: Despite making dozens of phone calls, Kuppersmith got nothing but confusing and contradictory answers when she tried to sort out the unexpected charge.

An agent for her insurer told her that her doctor hadnt gotten preauthorization for the testing. But in an email to Kuppersmith, a Genoptix employee told her the insurance company had denied the claim because molecular profiling was viewed as experimental.

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A spokesperson for New York-based Empire Blue Cross Blue Shield, which handled part of Kuppersmiths claim, said her health plan covers medically necessary genetic testing.

New York, one of 28 states with laws against surprise billing, requires hospitals to inform patients in writing if their care may include out-of-network providers, said attorney Elisabeth Benjamin, vice president of health initiatives at the Community Service Society, which provides free help with insurance problems.

A spokesperson for Mount Sinai said the hospital complies with that law, noting that Kuppersmith was given such a document in 2018 nearly one year before her bone marrow biopsy and signed it.

Benjamin said thats not OK, explaining: I think a one-year-old, vague form like the one she signed would not comply with the state law and certainly not the spirit of it.

Instead of sending Kuppersmith a bill, Genoptix offered to help her appeal the denied coverage to CareFirst. At first, Genoptix asked Kuppersmith to designate the company as her personal health care representative. She was uncomfortable signing over what sounded like sweeping legal rights to strangers. Instead, she wrote an email granting the company permission to negotiate on her behalf. It was sufficient.

A few days after being contacted by KHN, Kuppersmiths insurer said it would pay Genoptix at the in-network rate, covering $1,200 of the $2,400 charge. Genoptix said it has no plans to bill Kuppersmith for the other half of the charge.

The Takeaway: Kuppersmith is relieved her insurer changed its mind about her bill. But, she said: Im a relatively young, savvy person with a college degree. There are a lot of people who dont have the time or wherewithal to do this kind of fighting.

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Patients should ask their health care providers if any outside contractors will be involved in their care, including pathologists, anesthesiologists, clinical labs or radiologists, experts said. And check if those involved are in-network.

Try your best to ask in advance, said Jack Hoadley, a research professor emeritus at Georgetown University. Ask, Do I have a choice about where [a blood or tissue sample] is sent?

Ask, too, if the sample will undergo molecular diagnostics. Since the testing is still relatively new and expensive most insurers require patients to obtain prior authorization, or special permission, said Dr. Debra Regier, a medical geneticist at Childrens National Hospital in Washington and an associate with NORD, the National Organization of Rare Diseases. Getting this permission in advance can prevent many headaches.

Finally, be wary of signing blanket consent forms telling you that some components of your care may be out-of-network. Tell your provider that you want to be informed on a case-by-case basis when an out-of-network provider is involved and to consent to their participation.

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Her Genetic Test Revealed A Microscopic Problem And A Jumbo Price Tag - Bryan-College Station Eagle

People wait in line to be tested for coronavirus disease (COVID-19) while wearing protective gear, outside Elmhurst Hospital Center in the Queens borough of New York, March 30, 2020.

Jeenah Moon | Reuters

People with diabetes, chronic lung disease, heart disease or those who smoke may be at increased risk of developing severe complications if they get infected with the coronavirus, the Centers for Disease Control and Prevention said Tuesday.

In its first report looking at underlying health conditions that could make COVID-19 worse, the CDC analyzed data from confirmed cases in all 50 states and four U.S. territories between Feb. 12 and March 28. The agency examined 7,162 cases where data was available on underlying health conditions or other potential risk factors.Confirmed cases among people repatriated to the United States from Wuhan, China, where the virus emerged, and the Diamond Princess cruise ship were excluded, the agency said.

Among the 7,162 U.S. cases, 37.6%, had one or more underlying health conditions or risk factors, and 62.4%, had none of these conditions reported, according to the CDC's preliminary findings. The most commonly reported conditions were diabetes, chronic lung disease and cardiovascular disease.

The CDC found that a higher percentage of patients with underlying conditions were admitted to the hospital or into intensive care than patients without underlying conditions. About 78% of ICU patients and 71% of hospitalized COVID-19 patients had one or more reported underlying health conditions, the CDC said. In contrast, 27% of the patients who were not hospitalized had at least one underlying health condition, the agency said.

"These preliminary findings suggest that in the United States, persons with underlying health conditions or other recognized risk factors for severe outcomes from respiratory infections appear to be at a higher risk for severe disease from COVID-19 than are persons without these conditions," the CDC wrote. It recommended that people with underlying health conditions keep at least a 30-day supply of medication, a 2-week supply of food and other necessities and to know the COVID-19 symptoms.

The new data comes as U.S. cases climb to more than 181,000 and deaths surpass 3,000, more than the number of people who died in the Sept. 11, 2001, terror attacks. The death toll is expected to rise over the next few weeks as more patients flood hospitals, U.S. officials say.

Public health officials have long said the virus appeared to be particularly severe in the elderly and those with underlying health conditions.Symptoms can include a sore throat, runny nose, fever, dry cough, diarrhea or pneumonia and can progress to multiple organ failure or even death in some cases, they said.

A recent study published journal Pediatrics showed that some children can develop severe or critical disease. More than 90% of the caseswere asymptomatic, mild or moderate cases. However, nearly 6% of the children's cases were severe or critical, compared with 18.5% for adults.

The CDC said Tuesday the findings were in line with data from researchers in China and Italy, where the number of confirmed cases has topped 105,000.

The CDC recommended people who are sick, especially those with underlying health conditions, should stay at home, except to get medical care.

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CDC says diabetes, lung disease, heart disease and smoking may increase risk of severe coronavirus illness - CNBC

Eli Lilly wants diabetes patients who use its drugs to know that help is available, and to get the word out, itbought full-page ads in more than a dozen newspapers in the U.S. The simple print ads, which ran Monday, spoke directly to people who recently lost jobs or health insurance and told themto contact the Lilly Diabetes Solution Center for help.

Driven by COVID-19 shutdowns and job losses in the millions, Lilly hadalready seen a 32% increase in call volumes to the diabetes help call center. After the ads ran Monday, call volumejumped by 91%over the previous Monday, March 23. The 340 incoming calls markedthe highest single-day call volumesince theprogram beganin August 2018.

We expect the numbers tocontinue to rise as different states take measures from a quarantine standpoint andasmore and more people are affected through loss of job or loss of insurance, or if they just weren't aware previously of the diabetes solution center, said Andy Vicari, senior director of Lillys insulin business in the U.S.

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RELATED: Media makeover: Eli Lilly media chief switches up marketing mix and overhauls go-to-market model

Lilly did a similar ad campaign in December to highlight the diabetes help center in an effort to reach people whose healthcare deductibles were about to reset, he said. The ad tells people they may be able to get free insulin andthat the calls are simple and average 10 minutes, and it also mentions that Spanish-speaking staff members are available. Lilly's insulin meds supported through the solutioncenter include its Humalog family of products. Other Lilly non-insulin diabetes treatments not in the programincludeGLP-1 receptoragonistTrulicity and SGLT2 inhibitor Jardiance in partnership with Boehringer Ingelheim.

While Lilly doesnt deliver meds directly to patients, it can help callers navigate pharmacies thatdo, as well as sign up those who qualify for Lilly Cares, a non-profit run under the Lilly Foundation. For people with diabetes who call but dont use Lilly meds, the call center staff redirects them to assistance programs from the drugmaker whose products they do use, Vicari said.

The campaign will run on social media along with the print ads that ran in cities including New York, Los Angeles, Chicago, San Francisco, Seattle, Newark, New Jersey, and Columbus, Ohio. Digital and radio ads that began in December with the previous diabetes solution center campaign are still running and continuing through April.

RELATED: Eli Lilly's new Trulicity TV ad counters Trump with first-ever pricing information

Lillys now staying-at-home field force is also helping the effort, doing outreach to healthcare professionals. The reps are not making sales calls but rather, as Vicari said, calling to ask, What do you need and how can we help? Lilly reps can give information on the patient diabetes programs or help provide other resources, such as samples for new patients, that doctors can use.

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Lilly ad campaign in U.S. newspapers offers diabetes med help for patients affected by COVID-19 shutdowns - FiercePharma

Editor's note: Find the latest COVID-19 news and guidance in Medscape's Coronavirus Resource Center.

The era of diabetes telemedicine has arrived.

In the midst of the current COVID-19 pandemic, clinicians of all specialties who were already incorporating telehealth visits into their practices are now ramping it up, while those who were not using the technologies before are now scrambling to put them in place.

A free access article, "Top Ten Tips for Successfully Implementing a Diabetes Telehealth Program," was published March 19 in Diabetes Technology & Therapeutics by pediatric endocrinologist Stephanie Crossen, MD, of the University of California, Davis, and colleagues.

Written before the COVID-19 crisis hit, the article offers detailed practical advice in 10 key areas (for more details, see further down the article):

Hardware

Video software

Diabetes software

Scheduling telehealth visits

Standardizing telehealth visit processes

Reimbursement (for the US only)

Electronic health record (EHR) integration

Patient expectations

Patient-centered care

Culture change among providers and institutions

The document focuses primarily on implementing patient-to-clinic video encounters, although it also discusses asynchronous data review of patient-generated data and clinic-to-clinic video encounters.

In an interview, senior author Aaron B. Neinstein, MD, an endocrinologist at the University of California, San Francisco (UCSF), told Medscape Medical News: "What people are realizing is that this is a lot easier and there are fewer barriers than they thought. I keep hearing this. It seems big and scary but once people start doing it they think it's great and not that hard."

"I hope these are pragmatic tips that help people get over the hump."

Even under normal circumstances, routine diabetes care, whether in an endocrinology or primary care setting, is particularly well-suited to the use of telehealth: much of it involves electronic downloading of data from devices and speaking with patients about their own self-management.

Greg Dodell, MD, an endocrinologist with Mount Sinai Hospital in New York City who moderates a Twitter chat about telemedicine for endocrinologists using the hashtag #endotwitter, spoke with Medscape Medical News.

"I think this is an excellent paper and really a 'wow!' It comprehensively covers the landscape of telehealth including the proper setup, reimbursement, scheduling, and most importantly, how it can potentially facilitate the clinical relationship by enhancing self-care and eliminating potential barriers to follow-up."

Although nearly all of the information in the article can be applied now during the COVID-19 pandemic, it doesn't address two emergency federal actions that affect reimbursement in the United States, Neinstein noted.

Effective March 6, the Centers for Medicare & Medicaid Services lifted Medicare restrictions on the use of telehealth services during the COVID-19 crisis so that physicians will be paid for telehealth services at the same rate as in-patient visits for all diagnoses, not just services related to COVID-19.

Patients can receive telehealth services in their homes, anywhere in the country from a physician anywhere in the country. And physicians can reduce or waive cost-sharing for telehealth visits.

In addition, the Office of Civil Rights of the US Department of Health & Human Services (HHS) has waived penalties for violation of the Health Insurance Portability and Accountability Act (HIPAA) so that during the COVID-19 pandemic healthcare providers can communicate with patients through technologies such as FaceTime or Skype.

Dodell said that in the current COVID-19 crisis, endocrinologists and other clinicians who see a large number of patients with diabetes are at a bit of an advantage because of how well suited the condition is to virtual care.

"As a small business owner, I'm stressed but would be a lot more stressed if I couldn't do what I do...I have a good friend who's a gynecologist and had to close," he told Medscape Medical News.

Right now Dodell is not having patients get routine lab work done, but normally that would happen prior to a telehealth visit.

And in usual practice, patients still need to come in once a year for a physical exam. Of course, those appointments are also on hold for now.

"There are tools like digital stethoscopes and the Apple watch, and home blood pressure reading is easy. I don't think any of that stuff should replace physical contact, but in a situation like we're going through with this pandemic they're great options," he notes.

Neinstein, who is director of clinical informatics at the UCSF Center for Digital Health Innovation, added that, as the situation evolves, different care models will need to be adopted.

"It will become clear that as the healthcare workforce is strained and there's less capacity [for] care for chronic disease, we need to be looking at...a lot more nonphysician visits coaches, mental health professionals, peer groups, group visits for education and a lot more...texting or chatting."

And in the non-COVID setting, Dodell points out that implementing telehealth could streamline office flow and even save money: "I can do a telemed visit in half the time [of a conventional office visit]."

There are actually far more than 10 tips in the article, but they are grouped under 10 headings.

Hardware: Basic requirements for video visits are a mobile device (smartphone or tablet), laptop, or desktop with audio and video capabilities, an internet connection, and software download capability. This section covers equipment including cameras, headphones, monitors, and room lighting.

Video software: Many options for video conferencing software are HIPAA-compliant (assuming the same rules return after the COVID-19 crisis). Patients need to download the software application or run a temporary application for the visit. Most platforms offer multiparty conferencing for calls with children, adolescents, or the elderly.

Diabetes software: Nearly all diabetes devices incorporate data-sharing platforms, although unfortunately at this time many aren't compatible with each other or with EHRs. This section lists several desirable features, including compatibility with the broadest array of devices including insulin pumps, continuous glucose monitors (CGMs), glucose meters, and smart pens, easy upload for patients, and "seamless and flexible" account administration.

Scheduling telehealth visits: The authors recommend setting aside a block of time for telehealth visits separate from in-person visits to avoid overlap.

Standardizing telehealth visit processes: Patients need to be trained in advance on how to upload their data prior to the visit, and instructed when and where to have lab work done. This section discusses the role of office support staff in these processes.

Reimbursement (United States only): In general, video visits should be coded using typical current procedural terminology (CPT) codes based on time, such as 99214 for an established patient visit lasting 25-39 minutes, with the modifier 95 and the point-of-service code 02 for telehealth. As with in-person visits, additional codes can also be added such as CPT 95251 for CGM review and interpretation. For the most recent regional and state policies on this, check the Center for Connected Health Policy's website.

EHR integration: Minimum requirements include having the correct billing codes built-in, the ability to designate a separate visit type in providers' schedules, and standardized documentation for video visits. "We're still a long way from integration," Neinstein noted. "There are still several device companies that will not let the patient move their data off the device into software that they want to use. When you're trying to run a virtual clinic that makes life really, really hard." But he also said that new HHS regulations aimed at lowering EHR burden on physicians and other clinicians should help, assuming that device manufacturers comply.

Patient expectations: Patients need to know when these visits are available, what they will cost (typically the copay is the same as an in-person visit, but not always), and when they will be expected to return in person.

Patient-centered care: Whereas traditional diabetes care is based on the provider's availability, "with telehealth, diabetes care can take place in the home at a frequency customized to the individual." This section discusses several other potential patient-centered benefits.

Culture change among providers and institutions: "Acknowledging concerns and building supportive practices will increase your likelihood of success. We have found it critical to engage all institutional stakeholders early in the process to allow for successful integration of telehealth practices into routine care," the authors write.

"Recent improvements in both diabetes technology and telehealth policy make this an ideal time for diabetes providers to begin integrating telehealth into their practices," they conclude.

Crossen has reported receiving research support from the National Center for Advancing Translational Sciences, National Institutes of Health. Neinstein has reported receiving research support from Cisco Systems; consulting fees from Nokia Growth Partners and Grand Rounds; serving as an advisor to Steady Health; receiving speaker honoraria from the Academy Health and Symposia Medicus; writing for WebMD; and being a medical advisor and cofounder of Tidepool.

Diabetes Technol Ther. Published online March 19, 2020. Full text

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Top 10 Tips for Diabetes Telehealth Prophetic in Face of COVID-19 - Medscape

The following article is part of conference coverage from the American College of Cardiology (ACC.20) /World Congress of Cardiology. The Cardiology Advisors staff is reporting on research conducted by leading experts in cardiology. Check back for the latest news from the ACC.20.

Thepresence of diabetes before liver transplantation may be an independentpredictor of sudden cardiac death after the procedure, according to studyresults intended to be presented at the American College of Cardiologys 69thAnnual Scientific Session.

Suddencardiac death has been recognized as a common type of cause-specific mortalityafter liver transplantation. To better understand the predictors of suddencardiac death, researchers prospectively collected data on clinical outcomesafter liver transplantation from 4538 adults who visited 6 centers in Australiaand New Zealand between 1985 and 2017 (median follow-up, 10.5 years).

Apanel of 2 cardiologists and a transplant physician assessed the cause of deathin this cohort, and cases of sudden cardiac death were defined as witnessedarrests or unwitnessed cases, in which patients were deemed to be healthy atthe last time of contact. In this cohort, there were 240 cardiovascular-relateddeaths (5.3%), of which 30.4% were categorized as sudden cardiac death. Suddencardiac death vs death with a different etiology occurred earlier after livertransplantation (7.5 vs 9.0 years, respectively; P =.03).

The presence of diabetes before liver transplantation was identified as an independent predictor of sudden cardiac death after adjusting for univariate predictors (ie, coronary artery disease, age, and steatohepatitis of nonalcoholic origin) in a multivariate regression analysis (hazard ratio, 2.5; 95% CI, 1.1-6.0; P <.001).

Studiesare needed to assess mechanisms of [sudden cardiac death] following [livertransplantation], and whether intensive risk factor modification in thepost-[liver transplantation] diabetic population improves survival,concluded the study authors.

Reference

Koshy AN, Gow PJ, Han HC, et al. Diabetes is an independent predictor of sudden cardiac death following liver transplantation: results from the Australian and New Zealand Liver Transplant Registry over 30 years. Intended to be presented at: American College of Cardiologys 69th Annual Scientific Session; March 28-30, 2020; Chicago, IL.

Visit The Cardiology Advisors conference section for coverage intended to be presented at the annual meeting of the American College of Cardiology (ACC.20)/World Congress of Cardiology

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Diabetes May Be an Independent Predictor of Sudden Cardiac Death After Liver Transplantation - The Cardiology Advisor

Dapagliflozin reduced cardiovascular morbidity and mortality in patients with heart failure with reduced ejection fraction (HErEF) regardless of diabetes status, a new study suggests.

Additional treatments are needed for heart failure with reduced ejection fraction (HFrEF), the authors, publishing in Journal of the American Medical Association (JAMA), wrote in their study. Sodium-glucose cotransporter 2 (SGLT2) inhibitors may be an effective treatment for patients with HFrEF, even those without diabetes.

To evaluate the effects of dapagliflozin in patients with heart failure with reduced ejection fraction both with and without diabetes, the exploratory analysis included patients with NYHA class II to IV with ejection fraction rates less than or equal to 40% and elevated plasma NT-proBNP. Patients (n=4,744) were given a once-daily dose of 10 mg dapagliflozin (or placebo) added to regular therapy. The primary study outcome was composite worsening heart failure or cardiovascular death, and outcomes were analyzed by diabetes status.

According to the results, 4,742 patients completed the trial. Among patients without diabetes, the primary study outcome occurred in 13.2% of patients in the dapagliflozin group and 17.7% in the placebo group (HR=0.73; 95% CI, 0.60 to 0.88). In diabetic patients, the primary study outcome occurred in 20.0% in the dapagliflozin group and 25.5% in the placebo group (HR=0.75; 95% CI, 0.63 to 0.90; P for interaction=0.80). In patients without diabetes and with a glycatred hemoglobin of at least 5.7%, the primary outcome occured in 13.7% of those in the dapagloflozin group and 18.0% in the placebo group (HR=0.74; 95% CI, 0.59 to 0.94; P for interaction=0.72).

In this exploratory analysis of a randomized trial of patients with HFrEF, dapagliflozin compared with placebo, when added to recommended therapy, significantly reduced the risk of worsening heart failure or cardiovascular death independently of diabetes status, the researcher wrote in their conclusion.

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Dapagliflozin Linked with Reduced Morbidity, Mortality in HFrEF Patients Irrespective of Diabetes - DocWire News

Diabetes is the No. 1 underlying condition in people who have died of COVID-19 in Louisiana, the state health department reported Monday.

Of the 185 people who have died so far, 40 percent of them were diabetic, according to a press release from the Louisiana Department of Health.

Only 3 percent of those who have died had no underlying health conditions.

Other conditions in those who died include:

Most of the 185 people 105 who died of COVID-19 in Louisiana were 70 or older. Thirty-two were ages 60 to 69, 28 were ages 50 to 59, 13 were ages 40 to 49, and six were ages 30 to 39. This count is according to the Louisiana Department of Health website and only adds up to 184 one short of the official count of those who have died.

Want to take a breather and catch up later?Sign up for our New Orleans Public Radio newsletterand we'll send you a news roundup at the end of each week.

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Diabetes Tops The List Of Underlying Conditions In People Dead From COVID-19 In Louisiana - WWNO

Treatment with ticagrelor, an anti-platelet medication, alone demonstrated cardiovascular benefit in patients with diabetes when compared with dual therapy of ticagrelor and aspirin, according to new research presented at the American College of Cardiologys Annual Scientific Session Together with World Congress of Cardiology (ACC.20/WCC).

The findings were also published in the Journal of the American College of Cardiology.

The study, known as the TWILIGHT trial, examined whether ticagrelor alone or ticagrelor plus aspirin more effectively reduced bleeding without increasing the risk for heart attacks, stroke, death, or other adverse events caused by arterial blockages in patients who had received at least 1 stent and were at high risk for adverse events.

In the trial, 9006 patients at 187 medical centers in 11 countries were enrolled. Patients had received at least 1 stent and were at high risk for bleeding or another arterial blockage.

For the current study, 2670 patients with diabetes were evaluated. Of these patients, those who received ticagrelor plus a placebo were less likely to have clinically significant bleeding compared with those who received ticagrelor plus aspirin, 4.5% versus 6.7%, respectively.

Additionally, 4.6% of patients treated with ticagrelor plus a placebo died or had a heart attack or stroke, compared with 5.9% of those who received ticagrelor plus aspirin, according to the study findings. The authors noted that, although not statistically significant, the findings suggest that eliminating aspirin does not have any negative effects on patients.

According to the authors, patients in the study were diagnosed with diabetes, but this was not confirmed by laboratory testing, citing one of the studys limitations. Additionally, patients with the most severe type of heart attack were excluded from the trial.

These findings were consistent with the overall results of the TWILIGHT trial and were seen across all types of diabetes patients, irrespective of their clinical presentation and the treatment they were receiving for their diabetes, study author Dominick J. Angiolillo, MD, PhD, professor of medicine at the University of Florida College of Medicine in Jacksonville, Florida, said in a press release.

He noted that the results concluded that eliminating aspirin reduced bleeding without increasing risk of death, heart attack, or strokes. However, further research is needed to identify the best treatment for patients after they have completed 1 year on ticagrelor monotherapy.

Excerpt from:
Ticagrelor Monotherapy Shows Cardiovascular Benefit in Patients with Diabetes - Drug Topics