StemCell Therapy

Victoria Gray (second from left) with children Jamarius Wash, Jadasia Wash and Jaden Wash. Now that the gene-editing treatment has eased Gray's pain, she has been able be more active in her kids' lives and looks forward to the future. "This is really a life-changer for me," she says. Victoria Gray hide caption

Victoria Gray (second from left) with children Jamarius Wash, Jadasia Wash and Jaden Wash. Now that the gene-editing treatment has eased Gray's pain, she has been able be more active in her kids' lives and looks forward to the future. "This is really a life-changer for me," she says.

The last thing a lot of people want to do these days is get on a plane. But even a pandemic would not stop Victoria Gray. She jumped at the chance to head to the airport this summer.

"It was one of those things I was waiting to get a chance to do," says Gray.

She had never flown before because she was born with sickle cell disease. She feared the altitude change might trigger one of the worst complications of the devastating genetic disease a sudden attack of excruciating pain.

But Gray is the first person in the United States to be successfully treated for a genetic disorder with the help of CRISPR, a revolutionary gene-editing technique that makes it much easier to make very precise changes in DNA.

About a year after getting the treatment, it was working so well that Gray felt comfortable flying for the first time. She went to Washington, D.C., to visit her husband, who has been away for months on deployment with the National Guard.

"It was exciting. I had a window. And I got to look out the window and see the clouds and everything," says Gray, 35, of Forest, Miss.

Gray wore a mask the whole time to protect herself against the coronavirus, kept her distance from other people at the airport, and arrived happily in Washington, D.C., even though she's afraid of heights.

"I didn't hyperventilate like I thought I would," Gray says, laughing as she recounts the adventure in an interview with NPR.

NPR has had exclusive access to follow Gray through her experience since she underwent the landmark treatment on July 2, 2019. Since the last time NPR checked in with Gray in June, she has continued to improve. Researchers have become increasingly confident that the approach is safe, working for her and will continue to work. Moreover, they are becoming far more encouraged that her case is far from a fluke.

At a recent meeting of the American Society for Hematology, researchers reported the latest results from the first 10 patients treated via the technique in a research study, including Gray, two other sickle cell patients and seven patients with a related blood disorder, beta thalassemia. The patients now have been followed for between three and 18 months.

All the patients appear to have responded well. The only side effects have been from the intense chemotherapy they've had to undergo before getting the billions of edited cells infused into their bodies.

The New England Journal of Medicine published online this month the first peer-reviewed research paper from the study, focusing on Gray and the first beta thalassemia patient who was treated.

"I'm very excited to see these results," says Jennifer Doudna of the University of California, Berkeley, who shared the Nobel Prize this year for her role in the development of CRISPR. "Patients appear to be cured of their disease, which is simply remarkable."

Another nine patients have also been treated, according to CRISPR Therapeutics in Cambridge, Mass., and Vertex Pharmaceuticals in Boston, two companies sponsoring the research. Those individuals haven't been followed long enough to report any results, officials say.

But the results from the first 10 patients "represent an important scientific and medical milestone," says Dr. David Altshuler, Vertex's chief scientific officer.

The treatment boosted levels of a protein in the study subjects' blood known as fetal hemoglobin. The scientists believe that protein is compensating for defective adult hemoglobin that their bodies produce because of a genetic defect they were born with. Hemoglobin is necessary for red blood cells to carry oxygen.

Analyses of samples of bone marrow cells from Gray six months after getting the treatment, then again six months later, showed the gene-edited cells had persisted the full year a promising indication that the approach has permanently altered her DNA and could last a lifetime.

"This gives us great confidence that this can be a one-time therapy that can be a cure for life," says Samarth Kulkarni, the CEO of CRISPR Therapeutics.

Gray and the two other sickle cell patients haven't had any complications from their disease since getting the treatment, including any pain attacks or hospitalizations. Gray has also been able to wean off the powerful pain medications she'd needed most of her life.

Prior to the treatment, Gray experienced an average of seven such episodes every year. Similarly, the beta thalassemia patients haven't needed the regular blood transfusions that had been required to keep them alive.

"It is a big deal because we we able to prove that we can edit human cells and we can infuse them safely into patients and it totally changed their life," says Dr. Haydar Frangoul at the Sarah Cannon Research Institute in Nashville. Frangoul is Gray's doctor and is helping run the study.

For the treatment, doctors remove stem cells from the patients' bone marrow and use CRISPR to edit a gene in the cells, activating the production of fetal hemoglobin. That protein is produced by fetuses in the womb but usually shuts off shortly after birth.

The patients then undergo a grueling round of chemotherapy to destroy most of their bone marrow to make room for the gene-edited cells, billions of which are then infused into their bodies.

"It is opening the door for us to show that this therapy can not only be used in sickle cell and thalassemia but potentially can be used in other disorders," Frangoul says.

Doctors have already started trying to use CRISPR to treat cancer and to restore vision to people blinded by a genetic disease. They hope to try it for many other diseases as well, including heart disease and AIDS.

The researchers stress that they will have to follow Gray and many other patients for a lot longer to be sure the treatment is safe and that it keeps working. But they are optimistic it will.

Gray hopes so too.

"It's amazing," she says. "It's better than I could have imagined. I feel like I can do what I want now."

The last year hasn't always been easy for Gray, though. Like millions of other Americans, she has been sheltering at home with three of her children, worrying about keeping them safe and helping them learn from home much of the time.

"I'm trying to do the things I need to do while watch them at the same time to make sure they're doing the things they need to do," Gray says. "It's been a tough task."

But she has been able do other things she never got to do before, such as watch her oldest son's football games and see her daughter cheerleading.

"This is really a life-changer for me," she says. "It's magnificent."

She's now looking forward to going back to school herself, learning to swim, traveling more when the pandemic finally ends, and watching her children grow up without them worrying about their mother dying.

"I want to see them graduate high school and be able to take them to move into dorms in college. And I want to be there for their weddings just everything that the normal people get to do in life. I want to be able to do those things with my kids," she says. "I can look forward now to having grandkids one day being a grandmama."

Read more here:
1st Patients To Get CRISPR Gene-Editing Treatment Continue To Thrive - NPR

By Bio-IT World Staff

December 10, 2020 | The All of Us Research Program has begun to return genetic results to participants who have donated biosamples for research, NIH announced. The program prioritizes information return to its research volunteers. Initially, participants can choose to receive information about their genetic ancestry and traits, with health-related results available at a later date.

The All of Us Research Program is working to build a diverse community of 1 million or more participant partners across the U.S. to help researchers learn more about how genetics, environment and lifestyle factors affect health outcomes. So far, more than 270,000 people have contributed biosamples and more than 80% come from communities that are historically underrepresented in biomedical research. These include racial and ethnic minorities, sexual and gender minorities, and other groups.

Participants can also share data via surveys and their electronic health records. Data are stripped of personal identifiers and made available for research through the All of Us Research Hub.

As part of its core values, the program is committed to ensuring that participants have access to their own information, and many participants have expressed a strong desire to understand what their DNA can tell them.

Were changing the paradigm for research, said Josh Denny, M.D., All of Uss chief executive officer, in the announcement. Participants are our most important partners in this effort, and we know many of them are eager to get their genetic results and learn about the science theyre making possible. Were working to provide that valuable information in a responsible way.

The program's in-depth genetic analyses include both whole genome sequencing and genotyping. To return genetic information, the program has developed a robust informed consent process, giving participants information and choice about whether or not to receive results and which results they want to get back. The program also provides access to genetic counselors to help answer questions from participants and their health care providers.

All of Us teamed up with a network of awardees across the country to support this work, including the health technology company Color (Color received an initial $4.6 million in funding in August 2019) and a set of leading genome centers: Baylor College of Medicine, the Broad Institute and the Northwest Genomics Center at the University of Washington, with their partners.

With the All of Us Research Program, were beginning to return results for a genomics program that is of unprecedented scale, said Alicia Zhou, Ph.D., chief science officer at Color. For a long time, the research community has recruited participants into large-population genomics studies without returning any results back to them. With All of Us, weve provided the tools to do just thatin a convenient and accessible way. We now have a real opportunity to return value to participants.

All of Us is taking a phased approach to the return of genetic results and will offer additional results over time. In about a year, the program plans to begin offering participants the option to receive information about how their DNA may affect their bodys response to certain types of medicines (pharmacogenetics), and about genetic variants associated with the increased risk of certain diseases, based on guidelines of the American College of Medical Genetic and Genomics. Participants will receive information back as their DNA samples are processed, so not everyone will receive information immediately.

We need programs like All of Us to build diverse datasets so that research findings ultimately benefit everyone, said Brad Ozenberger, Ph.D., All of Uss genomics director. Too many groups have been left out of research in the past, so much of what we know about genomics is based mainly on people of European ancestry. And often, genomic data are explored without critical context like environment, economics and other social determinants of health. Were trying to help change that, enabling the entire research community to help fill in these knowledge gaps.

All of Us plans to begin making genetic data available to researchers in about a year, with strict privacy and security safeguards in place to protect participants information. The program seeks to engage researchers from diverse backgrounds to undertake a wide range of studies and learn more about how to tailor care to peoples different needs.

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All of Us Returns First Genetic Results To Participants - Bio-IT World

BURLINGTON, Mass. and NASHVILLE, Tenn., Dec. 10, 2020 /PRNewswire/ --Concert Genetics and Trapelo Health announced today that they have partnered to provide a streamlined, integrated solution that will expand access to evidence-based, genomic-informed oncology decision support.

The partnership is built on a shared mission to improve connectivity, transparency, and alignment among the healthcare stakeholders that support the delivery of oncology care. It combines the unique strengths of the two organizations: Concert's genetic testing data and digital infrastructure and Trapelo's expertise in molecular oncology decision support.

For an in-depth interview with leadership from both companies listen to this episode of the Precision Medicine Podcast.

For Concert's health plan customers, the joint solution will expand access to Trapelo's unique decision-support capabilities, which are built on a continuously updated knowledgebase of curated evidence that is used to inform molecular testing, treatment decisions and clinical trial recommendations.Offering this solution through Concert's ecosystem of data integrations, customers, and users will improve transparency and accountability in the most complex and dynamic aspects of molecular oncology,providing health plans the information they need to support evidence-based care decisions.

This partnership comes at a time when precision oncology care is rapidly advancing, bringing both new treatments and increased complexities to patient care. In the first six months of 2020, the U.S. Food and Drug Administration approved 21 precision oncology drugs, exceeding its total for all of 2019.1 A 2018 report from The Pharmaceutical Research and Manufacturers of America showed more than 1,100 oncology drugs in development with most requiring the use of molecular testing.2 These advances underscore the urgent need for clinical support solutions that can be deployed now.

"Achieving the promise of precision medicine requires bringing exactly the right data to bearat the moment of truth, when diagnosis is determined and treatment decisions are made," said Rob Metcalf, CEO of Concert Genetics. "Together, Concert and Trapelo can improve the quality and clarity of information available at that moment for the benefit of all stakeholders, especially the patient."

"This partnership enables a first-of-its-kind approach to managing the growing complexities precision oncology," said Clynt Taylor, CEO of Trapelo Health. "An increasing number of treatment decisions require both familial and somatic testing. Our collaboration gives stakeholders the resources and expertise to effectively manage genomic-informed decision making in the new age of cancer care."

Concert Genetics is a software and managed services company that promotes health by providing the digital infrastructure for reliable and efficient management of genetic testing and precision medicine. Concert's genetic testing management capabilities leverage a proprietary database of the U.S. clinical genetic testing market, market-leading expertise, and a technology platform that supports genetic test ordering, resulting, coding, coverage, and payment integrity.

Trapelo Healthis an information technology company on a mission to address the challenges that result from rapid changes in the science, technology and business of next-generation cancer care. Its product, Trapelo, is a win-win solution for doctors, labs and payers that need real-time, evidence-based information and full transparency to make patient-based decisions faster. As part of this commitment, Trapelo is the lead sponsor of The Precision Medicine Podcasta media platform that brings together industry leaders to help accelerate the effective use of precision medicine for cancer care. Tune in and follow us on Twitter @PMPbyTrapelo and on Linked-in.

Media Contacts:Nick Tazik, Vice President Growth, Concert Geneticsntazik@concertgenetics.com, (615) 861.2634

Karan Cushman, Chief Marketing Officer, Trapelo Healthkcushman@trapelohealth.com, (617) 250.7572

1Ray, T. (2020, July 11). FDA Approves Record Number of Precision Oncology Drugs in H1 2020. Precision Oncology News. https://www.precisiononcologynews.com/cancer/fda-approves-record-number-precision-oncology-drugs-h1-2020#.X8-T4BNKjly2 List of 2018 Medicines in Development for Cancer. (2020, May 30). PhRMA. https://www.phrma.org/report/list-of-2018-medicines-in-development-for-cancer

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Concert Genetics and Trapelo Health Partner to Advance Molecular Oncology Decision Support - BioSpace

SINGAPORE, Dec. 14, 2020 /PRNewswire/ -- Hummingbird Bioscience, an innovative clinical-stage biotech company focused on developing revolutionary therapies for hard-to-drug targets, today announced a collaboration with Tempus, a leader in artificial intelligence and precision medicine, to drive the development of Hummingbird's lead clinical program, HMBD-001, as it advances into clinical trials in HER3-driven cancers, including those that harbor neuregulin 1 (NRG1) fusions. As part of the collaboration, Hummingbird will be leveraging Tempus' AI-enabled platform and proprietary data, as well as joining its TIME Trial Network, for rapid identification, site activation and efficient enrollment of cancer patients who have NRG1 fusions and meet eligibility criteria for HMBD-001 clinical trials.

NRG1 fusions are a rare genetic mutation that are increasingly recognized as a driver of multiple tumor malignancies, and an actionable target for HER3 targeted therapy. NRG1 fusions cause the overproduction of NRG1 ligands, resulting in increased HER3 activation and tumor growth. Up to 1% of all solid tumors harbor NRG1 fusions, therefore, it is important to identify this patient population and develop therapies that can treat them.[1]

HMBD-001 is a uniquely differentiated anti-HER3 neutralizing antibody that was developed using Hummingbird's proprietary Rational Antibody Discovery platform. HMBD-001 has been immune-engineered to bind with high affinity to the HER3 dimerization interface and block HER3 growth signals to the cancer. Most importantly, HMBD-001 uniquely blocks HER3 in both open and closed conformations, and in the presence or absence of high concentrations of NRG1.[2] Pre-clinical studies have shown that these differentiated properties of HMBD-001 lead to robust and sustained tumor growth inhibition in multiple HER3 cancer models, including those with NRG1-fusions.

"We are excited to collaborate with Tempus to leverage their just-in-time clinical trial program and apply Hummingbird's deep knowledge of disease driving protein mechanisms in order to identify patients with actionable genetic abnormalities," said Dr Piers Ingram, Co-founder and CEO of Hummingbird.

"We look forward to Hummingbird joining our TIME Trial Network, providing patients across the country access to its HMBD-001 clinical trial," said Amy Franzen, Vice President of Operations, Therapies, Tempus. "This collaboration is an opportunity to identify those patients who could benefit from this investigational therapy, and if they are eligible, rapidly open the trial just for them."

About NRG1 fusions

A subset of patients with cancer have recently been identified who possess abnormal NRG1 gene fusions, that is the hybridization of their NRG1 gene with any one of a number of genes to produce NRG1 proteins that overexpress the HER3-binding domains.[3] This results in increased HER3 binding and dimerization, which consequently leads to increased tumor growth. Less than 1% of all solid tumors harbor NRG1 fusions, and there are currently no approved therapies to treat this patient population.[1] Moreover, studies suggest that NRG1 fusions are mutually exclusive with other known molecular drivers of cancer, such as ALK, ROS, and RET gene fusions, meaning that NRG1 fusions are likely to be a distinct orphan indication in a discrete patient population.[1]

About HMBD-001

HMBD-001 represents a unique, highly-specific, anti-HER3 neutralizing antibody with a novel mechanism of action that offers significant potential for broad clinical benefit. Previous attempts to block the HER3 receptor, a key player in the signaling pathway that promotes cell division and tumor growth in cancer, have not proven to be efficacious. HER3 is activated by the binding of NRG1, which stabilizes a transient open conformation to allow it to form heterodimers with HER2/EGFR. In the presence of abundant HER2/EGFR, heterodimers can form without NRG1.

Pre-clinical models have shown that HMBD-001 is able to effectively and uniquely bind to a difficult-to-target region on HER3, blocking the heterodimerization of HER3 with HER2/EGFR independent of NRG1 binding. This potently inhibits the activation of the signaling pathway and consequently, stops tumor growth. Cancer Research UK has partnered with Hummingbird Bioscience to advance this novel antibody drug into clinical trials for the treatment of HER3-driven cancer.

About Hummingbird Bioscience

Hummingbird Bioscience is an innovative clinical-stage biotech company focused on developing revolutionary therapies against hard-to-drug targets for improved treatment outcomes. We harness the latest advances in systems biology and data science to better understand and solve the underlying causes of disease and guide development of our therapeutics.

Enabled by our proprietary Rational Antibody Discovery platform, we discover antibodies against optimal yet elusive epitopes on important targets that have not been successfully drugged, unlocking novel mechanisms of action. We are advancing a rich pipeline of first- and best-in-class drug candidates in oncology, autoimmune and infectious diseases, in collaboration with global partners in academia and industry.

Our highly experienced teams in Singapore and the US span antibody discovery, pharmacology, production and clinical development. Together we aim to accelerate the journey of new drugs from concept to clinical care. For more information, please visit http://www.hummingbirdbioscience.com, and follow Hummingbird on LinkedIn and Twitter (@hummingbirdbio).

About Tempus

Tempus is a technology company advancing precision medicine through the practical application of artificial intelligence in healthcare. With one of the world's largest libraries of clinical and molecular data, and an operating system to make that data accessible and useful, Tempus enables physicians to make real-time, data-driven decisions to deliver personalized patient care and in parallel facilitates discovery, development and delivery of optimal therapeutics. The goal is for each patient to benefit from the treatment of others who came before by providing physicians with tools that learn as the company gathers more data. For more information, visit tempus.com.

[1] Jonna S, Feldman RA, Swensen J, Gatalica Z, Korn WM, Borghaei H, Ma PC, Nieva JJ, Spira AI, Vanderwalde AM, Wozniak AJ, Kim ES, Liu SV.. Detection of NRG1 Gene Fusions in Solid Tumors. Clin Cancer Res. 2019; 25: 49664972. https://doi.org/10.1158/1078-0432.CCR-19-0160

[2] Thakkar D, Sancenon V, Taguiam MM, Guan S, Wu Z, Ng E, Paszkiewicz KH, Ingram PJ, Boyd-Kirkup JD. 10D1F, an Anti-HER3 Antibody that Uniquely Blocks the Receptor Heterodimerization Interface, Potently Inhibits Tumor Growth Across a Broad Panel of Tumor Models. Mol Cancer Ther. 2020; 19: 490501.

[3] Ruiz-Saenz A, Dreyer C, Campbell MR, Steri V, Gulizia N, Moasser MM. HER2 Amplification in Tumors Activates PI3K/Akt Signaling Independent of HER3. Cancer Res. 2018; 78: 36453658.

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Hummingbird Bioscience Announces Collaboration with Tempus to Harness AI-driven Precision Medicine to Accelerate Clinical Development of HMBD-001 In...

The following are some highlights from the edited transcript.

Richard Kuhn: Traditionally, for viruses, we've either taken a virus and inactivated it, and used that as a vaccine, or we've taken a virus and made it less infectious that is, it's attentuated and made that a vaccine; or we've expressed proteins that are on the surface of a virus and used those proteins as stimulants for your immune system. These would be purified proteins that would be injected. The protein self-assembles into something that resembles the virus, but it doesn't have any of the components that allow the virus to replicate. So those are the standard, traditional vaccines.

The technology that Moderna and Pfizer are pushing right now is one in which you use the coding sequence, the information that codes for the viral protein that you're interested in. In the case of COVID-19, we're interested in a surface protein that we call the spike glycoprotein spike for short. This technology basically uses the genetic information that will make this spike protein when you put it into a cell. And that information is encoded in what we call messenger RNA mRNA. That's the vaccine, and it's packaged in a lipid nanoparticle for delivery purposes.

Serena Marshall: That's a ton of information, and I want to unpack it a little. Let's talk about the vaccines of days past, [in which we get] infected with a weakened version, an attenuated version, as you said. A lot of people think, Okay, so when I get this new COVID vaccine, am I going to be getting COVID? That's not the case here.

Richard Kuhn: That's absolutely correct. First of all, there's no infectious material being injected into an individual; you're only making a single protein, but it's the critical protein that your immune system will respond to.

What will happen is, that lipid nanoparticle will be able to enter cells in your body after you've been vaccinated. And that RNA, the messenger RNA, will make a protein, just like all the proteins your cells normally make. The only difference being that once it gets made, other cells are going to recognize it as foreign. And they're going to mount a response against it.

Serena Marshall: Why is it that this virus is able to have that protein and able to have that immune response?

Richard Kuhn: Well, this technology has been around for a few years. In fact, Moderna developed the technology initially against Zika virus. In the case of Zika virus, there was this massive expansion and infection of people in South and Central America, and everybody was very concerned, and then the virus died off. So Moderna had this technology but was never able to go to clinical trials because there was no Zika virus prevalent in the population.

Serena Marshall: So when we hear that this is a brand-new technology that's never been approved before, that's all true. But it's not new research; it actually, as you said, goes back to Zika. But also, [for] decades before they've been looking into this.

Richard Kuhn: The COVID-19 pandemic is the perfect situation for producing a messenger RNA vaccine, because it's very easy to produce in a large scale. Because it's synthetic, you don't have to grow anything in cells, which has been the traditional way that you produce vaccines. So it's very easy, it's very rapid. As soon as you have the genetic information of a virus or a pathogen, you can begin to develop a messenger RNA vaccine against it, which trims off years of very difficult work that we've previously had to do with the older vaccines.

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Track the Vax: What Do We Need to Know About the New Vaccines? - Everyday Health

Who do you think performs your medical laboratory tests for COVID-19 or any other test? If you answered my doctor or my nurse or a robot, you would be completely wrong.

To put it bluntly, your life is in the hands of medical laboratory professionals. We perform an estimated 13 billion laboratory tests in the United States each year. That means that laboratory testing is the single highest-volume medical activity in the lives of Americans.

Why should you care? Those 13 billion tests help drive approximately two-thirds of all medical decisions made by your doctor and other health care professionals from cradle to grave. There are only 337,800 practicing medical laboratory professionals for a population of just over 330 million people in the U.S.

Ive worked in public health and medical laboratories for three decades, specializing in the study of viruses and other microbes while also educating the next generation of medical laboratory scientists. In 2014, I coined the phrase the hidden profession that saves lives. With COVID-19, these unsung professionals are now in the limelight. Unfortunately, this pandemic has led to a nationwide burnout of these professionals, causing dangerous shortages in the U.S. health care infrastructure.

Medical laboratory testing is performed by highly skilled, rigorously educated, certified, licensed and dedicated medical laboratory professionals. You have probably never seen one of these medical scientists at work because they are rarely in public view unlike nurses, doctors and pharmacists.

In fact, without the test results we provide, your doctor or other health care professionals are flying blind. Dont get us wrong we have great respect and value for all health care professionals. We just want you to understand that we save lives every day even though you dont necessarily see us in the shadows of health care.

Since the beginning of the COVID-19 pandemic in the U.S., we have performed approximately 213 million tests and counting. And now we are tired. We hear the calls for more testing. Many of my former students, now colleagues in medical laboratories all over the country, are exhausted and dealing with burnout or thoughts of quitting.

The most common form of testing for COVID-19 and the gold standard is called a PCR test, which stands for polymerase chain reaction. Like most testing, PCR testing is largely invisible to patients once a nasal swab is taken. The purpose of this test is to detect the viruss genetic material called RNA in the cells collected on the swab.

For laboratory professionals the first step is to convert any RNA from the virus into DNA. Then, using a series of chemical reactions and specialized equipment, the DNA is replicated millions of times so that it is easier to detect. If genetic material from SARS-CoV-2 is detected, then the patient is infected with the coronavirus.

The demands of such a precise test and meticulous process are putting a massive burden on this workforce.

Recently the American Society for Clinical Pathology conducted a survey of laboratory professionals and reported that 85.3% felt burnt out; 36.5% reported inadequate staffing; 31.5% complained of a heavy workload and pressure to complete all testing; and 14.9% cite a lack of recognition and respect.

Part of the weariness stems from the fact that in addition to COVID-19, we are also running tests for people who are having babies, heart attacks, cancer, antibiotic resistant infections, strep throat and other illness or diseases. These 13 billion tests are performed by a workforce that has vacancy rates of 7%-11% in almost every region of the country.

A medical, or clinical, laboratory science degree often requires an average of five years of college education. Medical laboratory scientists all have bachelors degrees and have certification or a license to practice. I, and many of my colleagues, have a masters degree, and also a doctorate. These complex qualifications are reflected in our education and clinical background.

A degree in medical laboratory testing requires mastery of several areas of medicine including the study of hematology, molecular diagnostics, immunology, urine analysis, microbiology, chemistry, parasitology, toxicology, immunohematology (blood banking), coagulation and transfusion, and laboratory safety and operation. I often tell my students that this degree is basically like having to complete four majors.

Our profession can also start toward a laboratory science career at an entry level with a bit less education and clinical training even as a technician, which requires only a 2-year associates medical laboratory technician degree. These technicians often move up the career ladder by obtaining other degrees. Like any health care professional degree, ours is externally accredited through the National Accreditation Agency for Clinical Laboratory Sciences.

Currently there are an estimated 337,800 employed medical laboratory professionals in the U.S., according to the Bureau of Labor Statistics. This is an estimate, because without licenses in every state, an accurate number of practicing laboratory professionals is not available. But the demand for these professionals is expected to grow by 25,000 between 2019 and 2029, according to the Bureau of Labor Statistics. But that doesnt include the number of jobs that will become vacant when workers retire or leave the profession during the pandemic.

What is frightening to me is that while the demand for clinical laboratory personnel is growing, the number of training programs actually is declining. Currently, there are 235 medical laboratory scientist and 240 medical laboratory technician training programs in the U.S. This is a 7% decline from the year 2000. In some states, there are no programs.

Fewer training programs, coupled with greater demand for laboratory professionals, could impact patient care, notes Jim Flanigan, executive vice president of the American Society for Clinical Laboratory Science. He is concerned by the lack of federal programs supporting medical laboratory education as compared to all other health programs. Vacancy rates are exceeding the number of medical laboratory scientist and medical laboratory technician graduates.

A number of other factors help explain our low workforce numbers. Training laboratory personnel is expensive, and there are few scholarship or loan programs available for prospective students. Salaries are also problematic. Compared to nursing, physical therapists or pharmacists, our professionals are paid 40%-60% less on average for annual salaries.

The American Society for Clinical Laboratory Science is calling for expansion of the Title VII health professions program which provides education and training opportunities in high-demand disciplines to include medical (clinical) laboratory science. The organization also supports efforts to improve visibility of the profession by engaging in community outreach opportunities and by partnering with middle and high school STEM programs to show young people that laboratory medicine is a viable career path.

Lastly, with competition for laboratory personnel intensifying over the last year, turnover rates for some categories of laboratory personnel now exceed 20%. Because of the difficulty in finding qualified staff, medical laboratories are increasingly turning to temporary staff to handle the patient testing workload. In a sense, the pandemic has exacerbated a free-agent effect for traveling medical laboratory professionals that hurts continuity and quality in health care.

We hope that you see us and hear us. Your life or that of a loved one depends on it.

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Who is doing all those COVID-19 tests? Why you should care about medical laboratory professionals - The Conversation US

Dec. 15, 2020 11:00 UTC

MENLO PARK, Calif.--(BUSINESS WIRE)-- Avellino announced today the appointment of three new members to its Board of Directors. In addition, the company announced a new Chief Executive Officer and President, and Chief Financial Officer and Treasurer. All appointments are effective December 2020.

The Board of Directors has been further strengthened by the appointment of Aimee S. Weisner, Esq.; Richard Gannotta, NP, DHA, FACHE; and William Stasior, MS, PhD. Joining the executive team is current Board member Jim Mazzo as CEO and President, and Cyril Allouche as CFO and Treasurer.

Avellino Group Chairman Gene Lee said, All of our newest Board members embody the spirit and ingenuity of Avellino and bring such broad talent, expertise, and energy to the table. We are very fortunate to have them by our side to help us continue to grow as a company. And with Jim moving into a senior executive leadership position, his immeasurable business experience with scientific acumen will enhance our ability to continue to lead the way in delivering best-in-class personalized genetic and molecular diagnostics, data, and therapeutics. Also, we are excited to welcome Cyril to our leadership team. His depth of business experience across multiple industries and preparing companies for entering public markets will be a perfect complement to the scientific expertise we have fostered at Avellino.

Board of Directors Appointments

Aimee S. Weisner, Esq. is an experienced independent director in the medical device, pharmaceutical and biotech spaces, and she brings significant expertise as a corporate medtech executive and attorney. Most recently, from 2011 to 2019, Ms. Weisner served as Corporate Vice President, General Counsel of Edwards Lifesciences Corporation.

Richard Gannotta, NP, DHA, FACHE is a recognized leader in the health sector with service in CEO / President and executive roles in some of the nations most prominent academic and public health systems and a leading global medical technology company. In addition, Dr. Gannotta is Senior Lecturer at the NYU Wagner Graduate School of Public Service where his area of focus is on the management of healthcare organizations and health policy.

William Stasior, MS, PhD has established himself as a creative innovator with technical expertise at Silicon Valleys most recognizable technology companies. Dr. Stasior currently serves as Corporate Vice President, Technology, and a member of the Office of the Chief Technology Officer at Microsoft. Prior to joining Microsoft, he was for many years the Vice President, Artificial Intelligence, at Apple and head of Apples Siri division. Among many of his career accomplishments, Dr. Stasior also served as the Vice President of Amazon Search and was CEO of Amazon Silicon Valley subsidiary A9.com. Prior to joining the Board, Mr. Stasior provided guidance to Avellino as part of the Executive Advisory Committee.

Chief Executive Officer and President

Avellino appoints Jim Mazzo as the new CEO and President. Mr. Mazzo is one of the ophthalmic industrys best known and most respected business leaders with over 38 years of proven experience. His global reputation for building and running world-class organizations is based on 22 years leading Allergans North American and European eye care organizations. His many accomplishments and contributions to the healthcare, business and educational communities include serving as Board Chairman for AdvaMed as well as Vice-Chairman and Trustee for Chapman University and the University of San Diego.

Chief Financial Officer and Treasurer

Avellino also welcomes Cyril Allouche to the executive team as its new Chief Financial Officer and Treasurer. Mr. Allouche brings to Avellino over 20 years of experience in finance leadership in both public and pre-IPO companies, including diagnostics and biopharmaceutical. He most recently served as CFO at Dermavant Sciences and held finance leadership roles at Revance Therapeutics and CareDx. He also spent over a decade at PricewaterhouseCoopers in Audit and Transaction services.

The inclusion of Aimee, Richard, and William provides expanded leadership and broader operational, digital, marketing, and commercialization expertise that will surely complement our executive team. Along with Cyrils deep and extensive experience in leading the financial operations of healthcare businesses, Avellino will continue to grow our genetic and molecular diagnostic tests pipeline and flourish as a company, said Avellino Group Chairman Gene Lee.

Added newly appointed CEO Jim Mazzo: Its exciting times here at Avellino, all of the additions to the boardroom along with the changes taking place at the senior executive level shows that we are set up for success with unlimited potential for tremendous growth. Considering this, and the positive impact Avellino has had in providing testing during the pandemic and their efforts to fight blindness since their inception, and the advances they will bring to healthcare in the future, joining the senior management team was an easy decision for me to make.

About Avellino

Avellino Lab USA, Inc. is a global leader in gene therapy and molecular diagnostics and is at the forefront of precision medicine for eye care. The company is a proud member of the California State COVID-19 Testing Taskforce, which is focused on the expansion of CoV2 testing and the reduction of testing turn-around times (TAT). Beyond the AvellinoCoV2 test, Avellino recently launched AvaGen, the worlds first DNA test to confirm the presence of genetic indicators that are positively associated with corneal dystrophies and keratoconus genetic risk factors. The company will also soon launch an infectious disease panel of diagnostic tests. Beyond diagnostics, the company is also pioneering CRISPR gene editing to manage and potentially cure inherited diseases. Avellino is headquartered in Silicon Valley, California, with operations in Korea, Japan, China, and the UK.

To learn more about Avellino, please visit http://www.avellino.com.

View source version on businesswire.com: https://www.businesswire.com/news/home/20201215005070/en/

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Avellino Expands Its Board of Directors and Welcomes New Executive Management Members - BioSpace

The Fountain of Youth -- an enduring aspiration, particularly as the ravages of age reduce human faculties prior to leading inexorably to death. Reduction in sight is the human faculty that can have the greatest effect on quality of life in the aged -- a faculty that begins to decline in the 4th or 5th decade of life and doesn't get better (when it does) without medical intervention.

But what if there were a way to rejuvenate sight? That prospect is the tantalizing suggestion in a paper published on December 2nd entitled "Reprogramming to recover youthful epigenetic information and restore vision," Nature 588: 124-29*. The basis of the report is the recognition that many of the age-related effects on vision are an example of gene expression differences associated with epigenetic changes in chromosomal DNA. Epigenetics is a phenomenon of gene structure and expression involving small differences in nucleotide bases, typically methylation of cytosine residues at specific (CpG) sites. These changes have been studied in normal development, where gene expression changes arise as different cell types properly differentiate and act as a molecular "clock" reflecting age. The ability to turn back cellular time has been demonstrated by the development of induced pluripotent stem cells (iPSCs), wherein terminally differentiated somatic cells (typically fibroblasts) can be turned into pluripotent cells. Pluripotent cells are capable of differentiating into cells of each embryonic germinal layer (ectoderm, mesoderm, endoderm), and iPSCs can be produced by expressing four specific genes: OCT4, SOX2, KLF4 and MYC. All of these genes encode transcription factors capable of affecting (and effecting) developmentally relevant gene expression. Consequent to this "de-differentiation" occasioned by expression of these genes is a "resetting" of the epigenetic patterns associated with development. In this paper the researchers hypothesized that resetting these epigenetic patterns could also rejuvenate neuroretinal cells to reinvigorate and overcome the ocular nerve damages by glaucoma in an animal model.

Because one of these genes (MYC) is also associated with cancer development (i.e., it is an oncogene) the researchers developed an inducible expression construct that expressed only the OCT4, SOX2, and KLF4 members of the quartet (OSK). (This decision was also informed by the experience of other researchers that continuous expression of all four genes in animal models resulted in teratomas or was fatal within days of introduction.) Their system used a polycistronic (i.e., all the genes in one linear array) construct of all three genes regulated by a tetracycline response element (TRE) promoter in a adeno-associated viral vector. This construct was tested by introduction into fibroblasts from aged (20 month old) mice and gene expression related to aging (i.e., that showed differential expression with age) was evaluated. These studies showed that OSK expression for 5 days resulted in a "youthful" mRNA expression pattern in these genes (without any effect on the terminal differentiation state of the fibroblasts).

The TRE promoter enabled selection for or against expression of the OSK gene cassette; as the authors explain "[t]he TRE promoter can be activated either by reverse tetracycline-controlled transactivator (rtTA) in the presence of the tetracycline derivative doxycycline (DOX) ('Tet-On') or by tetracycline-controlled transactivator (tTA) in the absence of DOX ('Tet-Off')." Simply put, the presence of absence of DOX in the animal's drinking water determined whether the expression cassette is "on" or "off," as illustrated in this figure:

Long-term (10-18 months) expression of this cassette was achieved in both young (5 months-old) and aged mice with no tumorigenesis or other negative side effects being observed.

To test the ability of induced OSK expression to rejuvenate optical nerve cells the researchers examined retinal ganglion cells (RGC, which project axons away from the retina informing the optic nerve) in an optic nerve crush injury model (which mimics the effects of optic nerve injury due to inter alia glaucoma). The construct was delivered by injection into the vitreous humor and resulted in about 37% of the RGCs taking up and expressing the OSK genes in response to DOX administration. A separate cohort of mice were administered versions of the construct where DOX inhibited OSK expression. In these experiments, "the greatest extent of axon regeneration and RGC survival occurred when all three genes were delivered and expressed as a polycistron within the same AAV particle" according to the researchers. In contrast, inhibition of OSK expression in the "Tet-Off" mice showed no axonal growth. Moreover, delivery of the OSK genes individually in separate viral vectors or in pairs also did not show axonal growth, indicating the need for these genes to be expressed together in proper relative amounts provided by the polycistronic construct. The researchers also found OSK expression induced expression of Stat3, a gene know to encourage regeneration. These results were obtained in using 12-month-old mice as well as 1- and 3-month-old mice, which indicated, as the authors note, that "ageing does not greatly diminish the ability of OSK transcription factors to induce axon regeneration." Increased axonal growth from RGCs was found even after crush injury, an effect found with no other treatment modalities.

The researchers then determined whether these reinvigorated RGCs showed changes in DNA methylation patterns. In the absence of DOX-induced OSK expression injury in this model caused an "accelerated" aging pattern, whereas in the presence of DOX-induced OSK expression counteracted this effect according to the results reported in this paper. Interestingly, this preservation of a "youthful" pattern of DNA methylation was found to be enriched at genes "associated with light detection and synaptic transmission." Having shown this association the researchers then investigated whether axonal regeneration required youthful changes in DNA methylation. These experiments were performed by reducing expression of genes that caused DNA demethylation in RGCs (and whose expression was known to be increased in cells expressing OSK) and detecting that axonal regeneration did not occur in these mice even in the presence of DOX-induced OSK expression.

Whether these effects of OSK expression would also be seen in human neurons was investigated using differentiated human neurons in vitro. Neurons harboring an OSK-encoding construct were treated with vincristine (a drug that occasions axon injury) and DOX-induced OSK expression was shown to "counteract[] axonal loss and the advancement of DNA methylation age," showing a 15-fold greater area of proliferation in OSK-expressing cells than control vincristine-treated neural cells. These cells also showed the demethylation-dependent characteristics that were shown in RGCs in the mouse optic nerve crush injury model.

The most clinically significant result disclosed in this paper involved the effect of OSK expression in a glaucoma model in vivo. Intraocular pressure was increased to pathological levels by injecting microbeads unilaterally into the anterior chamber of mouse eye for 21 days. At 4 weeks, after these animals showed correspondingly unilateral decreases in axonal density and the number of RGCs present in the treated eye. The viral vector encoding inducible OSK expression thereafter was introduced by intravitreal injection followed by DOX-induced OSK expression for 4 weeks. Compared with control (introduction of saline or viral vectors not encoding OSK into the microbead-treated eyes) the OSK vector-treated eyes showed "restored axon density equivalent to that in the non-glaucomatous eyes, with no evidence of RGC proliferation." These mice also showed a reversal of vision loss caused by the glaucomatous injury. Together these results indicated that OSK expression could be a therapy for glaucoma in humans.

Finally, the paper reports efforts to determine whether OSK expression could improve age-related (as opposed to injury- or pathology-related) vision problems. In these experiments, 3-and 11-month-old mice were treated by intravitreal injection of DOX-inducible OSK encoding constructs and OSK expression induced for 4 weeks. Twelve-month-old mice showed age-related visual acuity and RGS electrical activity diminution which was reversed by DOX-induced OSK expression. However, these phenotypic changes were not observed to be associated with an increased number of RGCs or axon density, which prompted these researchers to hypothesize that the effect were dependent on changes in gene expression ("transcriptomic changes" as these were termed in the paper). RGCs from treated or untreated 12-month-old mice were isolated and compared with RGCs from 5-month-old mice and expression of 464 genes were found to be altered: expression of almost all (90%) of these genes were found to be restored to youthful levels in OSK-expressing RGCs. The participation of DNA methylation changes in aged RGCs in producing a youthful pattern of gene expression was further assessed and validated using artificial intelligence/machine learning approaches.

The results reported in this paper suggest therapeutic interventions that could improve vision in the aged human population even in the absence of vision-impairing pathologies such as glaucoma. Although cautious to mention that "we do not wish to imply that DNA methylation is the only epigenetic mark involved in this process" and "[i]t is likely to involve other transcription factors and epigenetic modifications," the authors are not blind to the implication that:

[W]e show that it is possible to safely reverse the age of a complex tissue and restore its biological function in vivo. Using the eye as a model system, we present evidence that the ectopic expression of OSK transcription factors safely induces in vivo epigenetic restoration of aged CNS neurons, without causing a loss of cell identity or pluripotency. Instead, OSK promotes a youthful epigenetic signature and gene-expression pattern that causes the neurons to function as though they were young again. The requirement for active demethylation in this process supports the idea that changes in DNA methylation patterns are involved in the ageing process and its functional reversal.

* By researchers from Harvard Medical School, Yale University School of Medicine, Massachusetts General Hospital, UCLA Geffen School of Medicine, and The University of New South Wales Medical School.

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Epigenetic Changes Implicated in Age-related Diminution in Vision and Its Possible Reversal - JD Supra

Israeli biotech Pluristem is canning its experimental phase 3 critical limb ischemia therapy after an outside review said it was no good.

Haifa, Israel-based Pluristems R&D operation is built upon placenta-derived adherent stromal cells, which the biotech has designed for use in patients of all human leukocyte antigen types. This approach is made possible by the low immunogenicity of the cells. Once inside the body, Pluristem hopes the cells will drive the healing of injured tissue.

But one of its leading contenders using this approach has been judged a failure in phase 3: An independent data monitoring committee (DMC) took a look at the ongoing data for its pivotal phase 3 in patients with critical limb ischemia (CLI), a severe obstruction of the arteries which markedly reduces blood flow to the extremities and can lead to amputation.

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The DMC said the test was unlikely to meet the primary endpoint, and that the CLI study population has experienced a substantial low number of events (major amputation of the index leg or death), different from what is known in clinical medicine for the rate of these events in this patient population. The lower than anticipated event rate in the placebo group reduced the statistical power of the study to meet its primary endpoint.

The biotech is now tossing out the therapy and will instead focus on other pipeline areas, including a long-shot stem cell attempt at treating COVID-19. The biotechs shares fell nearly 40% on the news.

We are deeply disappointed by the outcome of the CLI interim analysis. In light of the DMCs recommendation, we decided that it would be in the best interests of the company and its shareholders to terminate the CLI study and focus our resources and efforts on our other lead indications, said Pluristem CEO and President Yaky Yanay.

We expect to present topline clinical results during calendar year 2021, including our phase 3 study in muscle regeneration following hip fracture, phase 2 studies in Acute Respiratory Distress Syndrome associated with COVID-19 and our phase 1 study in incomplete hematopoietic recovery following hematopoietic cell transplantation. Pluristem is well positioned to advance and support future development of these indications.

Last year, Pluristem penned a deal with NASA to assess its cell therapies against the health problems caused by spending time in space, teaming up with NASAs Ames Research Center for the project, which focuses on using its PLX placenta-derived cell therapies to try to prevent or treat medical conditions that can occur during and after space missions, including conditions that affect the blood, bone, muscle, brain and heart.

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NASA-partnered Pluristem crashes to Earth as it axes leading therapy - FierceBiotech

High-dose melphalan (Evomela) followed by autologous stem cell transplant (ASCT) has demonstrated significant efficacy for the treatment of patients with multiple myeloma. The agent has improved progression-free survival (PFS) in patients when administered as upfront therapy to patients aged 66 years or younger.

The median PFS shown with melphalan plus ASCT in a phase IFM/DFCI 2009 study was 50 months versus 26 months when compared with the standard of care treatment regimen lenalidomide (Revlimib), bortezomib (Velcade), and dexamethasone (RVD) alone hazard ratio for disease progression or death, 0.65;P<.001). As the treatment strategy continues to be explored in patients with multiple myeloma, researchers have now begun to investigate an outstanding biological question of whether the alkylating agent causes an increased amount of DNA damage.

An analysis presented during the virtual 2020 American Society of Hematology (ASH) Annual Meeting showed that between the time of diagnosis and relapse, patients treated with high-dose melphalan had an increased number of point mutations. It was unclear from this research how this result translated to treatment selection and sequencing.

In an interview with Targeted Oncology, Mehmet Samur, PhD, senior research scientist, Dana-Farber Cancer Institute, discussed the investigation of high-dose melphalan following ASCT in patients with multiple myeloma and shared insights into how the ongoing questions can be explored in the future.

TARGETED ONCOLOGY: Can you explain what was demonstrated prior with high-dose melphalan followed by ASCT in patients with multiple myeloma?

Sumar: The clinical part of the phase 3 study was published previously. It showed that when you do RVD plus high-dose melphalan following by stem cell treatment, patients do significantly better than patients who get RVD alone. Adding high-dose melphalan increased the PFS benefit by around 12 months.

TARGETED ONCOLOGY: Can you provide background on your analysis of Melphalan for patients with multiple myeloma?

Sumar: Melphalan is an alkylating agent. Because of the way the agent works, we always think that it creates more DNA damage. The study that we presented at ASH was questioning whether this was true or not.

We collected DNA sequencing data from patients who were treated with RVD followed by high-dose Melphalan and a bone marrow transplant. We had a total of 25 patients, and we collected data at the time of diagnosis and the time of relapse. To compare this compilation, we also collected data from 43 patients from the IFM/DFCI 2009 study who only received RVD. We also collected data at diagnosis and relapse in the 43 patients. Genomic alterations were compared at diagnosis and relapse for patients who were injected with high-dose Melphalan and RVD versus patients who were only treated with RVD.

TARGETED ONCOLOGY: What were the findings from this study?

Sumar: We found that patients who got high-dose melphalan plus RVD followed by transplant accumulated more point mutations. To be precise, they accumulated around 10,000 new mutations between diagnosis and relapse at 5 years. For RVD patients, there were around 4500 new point mutations. The study showed that treating patients with high-dose Melphalan is increasing the mutational load by about 2.9-fold at the time of relapse.

TARGETED ONCOLOGY: What are the implications of these findings?

Sumar: There are a couple of things that we see from our study. One point is that we only saw point mutations. We didnt see any large-scale DNA alterations. This suggests that our patients who are treated with high-dose melphalan are more likely to experience changes.

In terms of the pathways that are mutated, the DNA damage repair pathway is more frequently mutated between diagnosis and relapse in patients treated with high-dose melphalan. We think that if we combine inhibitors that can overcome the selection of DNA damage repair pathway mutations, those patients may get additional benefit from the treatment.

We dont have clear data yet on whether this increased mutational load is something that is bad for patients. Even though these patients have more mutations, overall survival times are similar between the 2 arms. C outcomes are not impacted by the increased number of mutation so far.

TARGETED ONCOLOGY: What plan are underway to further this research?

Sumar: We are expanding our study in multiple ways. There is no clear data set we can get answers to our ongoing questions yet. We have reached out to our partners around the world to see if we can come up with a cohort to investigation. Also, we are looking at impact of these mutational load increase on other features like secondary cancer rate.

TARGETED ONCOLOGY: The understanding of gene mutations in myeloma is evolving. Can you discuss the current role of genomic testing?

Sumar: It has been shown in many studies that genetic testing at diagnosis can tell us which patients are high risk and which are low risk. Studies have also shown that patients who have loss of p53 or with deletion 17p will have bad outcomes.

There was a study published last year in the Journal of Clinical Oncology showing which patients with myeloma would have a lower risk. The study also shows that there are certain genomic features prolong survival time in patients.

We have different genomic tools that we can use to look at these different alterations and assess patient risk. Today, I think people are looking at these alterations from all different angles to plan stratification in upcoming clinical trials.

TARGETED ONCOLOGY: In your opinion, what change will we see in the myeloma treatment landscape in the next 5 years?

Sumar: There are a lot of studies looking at new treatment. Everyone is carefully watching out for data on new treatment options like chimeric antigen receptor T-cell therapy, bispecific antibodies, and monoclonal antibodies. It looks like these agents are providing benefit to patients, but they are at the very early stages of research.

Reference:Attal M, Lauwers-Cances V, Hulin C, et al. Lenalidomide, bortezomib, and dexamethasone with transplantation for myeloma. N Engl J Med. 2017; 376(14):1311-1320. doi: 10.1056/NEJMoa1611750

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Exploring Increase of Point Mutations Associated with High-Dose Melphalan in Multiple Myeloma - Targeted Oncology

Take a look at a month-by-month recap of the biggest stories at the University of Miami during the past year.

From a pandemic that forced the migration of spring semester classes to an online environment, to a new dean for the College of Engineering, to a Super Bowl halftime performance by the Band of the Hour, to a record-breaking gift for the Sylvester Comprehensive Cancer Center, 2020 proved to be a very unusual year for the University of Miami.

January

The Planet Kreyol student organization and the Office of Multicultural Student Affairs commemorate victims of the 2010 Haiti earthquake with dance, song, poetry, and more on January 15.

With evidence mounting that climate change is worsening everything from wildfires to hurricanes, the University of Miamis Rosenstiel School of Marine and Atmospheric Science hosts the three-day Miami Climate Symposium 2020: Predicting and Living with Extremes. The summitheld Jan. 22-24explored how sea level rise, saltwater intrusion, extreme heat waves, and other conditions exacerbate tropical cyclones, storm surge, and coastal flooding, as well as analyzed adaptation policies and strategies.

A professor of jazz trumpet at the Frost School of Music and four alumni of the University of Miami receive Grammy Awards during the 62nd annual ceremony held Jan. 26 in Los Angeles. Brian Lynch, jazz trumpet professor, is honored in the category of Best Large Jazz Ensemble. Cristian Macelaru, B.M. 03; Julio Reyes Copello, M.M. 00; Carlos Fernando Lopez, B.M. 12; and Natalia Ramirez, M.A. 17, also win Grammy Awards.

Miami baseball alumnus Tommy Adams makes the largest gift in support of the baseball program by a former University baseball student-athlete, donating $1 million toward the University of Miamis Baseball Facility Enhancement Campaign.

February

Five months after Hurricane Dorian devastated the Bahamas, students enrolled in the semester-long action project interdisciplinary class offered by the Miami Herbert Business School travel to Freeport for a three-day visit, meeting with Bahamian students and local businesses to share ideas and inspire hope.

Building on the immense resources and expanding the vision of the Center for Computational Science, the University establishes the Miami Institute for Data Science and Computing to catalyze data-intensive research that will solve real-world problems and enhance the understanding of data science among students and the public.

The Lancet, one of the worlds leading medical journals, announces that Felicia Marie Knaul, director of the University of Miami Institute for Advanced Study of the Americas, will lead a new Lancet Commission examining gender-based violence and maltreatment of young people, two areas with a dearth of study and understanding.

Students from the University of Miamis Frost Band of the Hour and the color guard, as well as the Hurricanettes dancers, perform in the Pepsi Super Bowl LIV Halftime Show at Hard Rock Stadium on Feb. 2.

Redshirt senior David Dinsmore wins his fourth straight gold medal in the mens platform on Feb. 21, capturing top honors at the 2020 ACC Swimming and Diving Championships.

At the Dolphins Cancer Challenge, Team Hurricaneswith 1,250 strongjoined thousands of others on Feb. 29 to run, walk, and ride to fight cancer and support the Sylvester Comprehensive Cancer Center.

March

For the health and well-being of the campus community, the University extends spring break for students through March 22, announcing that classes will resume on March 23 but strictly in online environments through at least April 4. Shortly thereafter, with COVID-19 cases surging across the nation, the University, in accordance with public health guidance to reduce density on campus, extends online instruction through the remainder of the spring semester and implements partial closing of on-campus housing.

April

An international team of scientists led by Dr. Camillo Ricordi, director of the Diabetes Research Institute and Cell Transplant Center at the University of Miami Miller School of Medicine, is granted immediate FDA authorization for a 24-patient clinical trial to test the safety and exploratory efficacy of umbilical cord-derived mesenchymal stem cells to block the life-threatening lung inflammation that accompanies severe cases of COVID-19.

Joined by the Rapid Defense Network in New York, the Southern Poverty Law Center, and others, the School of Laws Immigration Clinic files a lawsuit on April 13 accusing U.S. Immigration and Customs Enforcement authorities of ignoring COVID-19 guidelines in three Florida detention centers.

Debbie Ajagbe is named the 2020 ACC Indoor Track Scholar-Athlete of the Year on April 17, with five other Miami women joining her on the All-ACC Academic Team for Indoor Track and Field. Earlier in the year, Ajagbe, a mechanical engineering major, earned both ACC Womens Field Performer of the Year and ACC Championship Field MVP honors, winning both the weight throw and shot put at the conference championships.

May

The 180 graduates of the Miller School of Medicines Class of 2020 celebrate their newly minted degrees during a virtual commencement on May 9.

Arva Moore Parks, a prominent historian and preservationist, who served on the University of Miami Board of Trustees for 26 years and wrote several books on Greater Miami, Coral Gables, and University history, passes away on May 10.

Nine University of Miami Athletics programsmens basketball, mens and womens cross country, mens diving, golf, rowing, mens and womens tennis, and womens track and fieldare recognized for perfect single-year scores of 1,000 in the 2018-19 Academic Progress Report released May 19 by the NCAA.

UMTVs first Black show, The Culture, is nominated by the Suncoast Chapter of the National Academy of Television Arts & Sciences for a student production award in the magazine program category.

Brian Van Belle and Chris McMahon, two of the Miami Hurricanes best pitchers, are named All-Americans by Collegiate Baseball Newspaper on May 26. Both Van Belle and McMahon earned spots on the second team after posting brilliant performances in the abbreviated 2020 season that was halted due to the COVID-19 pandemic.

June

Pratim Biswas, the Lucy and Stanley Lopata Professor in the McKelvey School of Engineering at Washington University in St. Louis and a pioneer in his field recognized for applying aerosol science and engineering to multiple areas, is named dean of the University of Miami College of Engineering.

The University of Miami Board of Trustees elects six new members to its ranks with expertise in business, finance, law, technology, and strategic planning. Patricia Menendez-Cambo, Adam E. Carlin, Jose R. Mas, Alice S. Vilma, Carolyn B. Lamm, and Jordan Rhodes were elected to the Board on June 19.

Dr. Judy Schaechter, chair of the Miller School of Medicine Department of Pediatrics, is named a 2020-21 Health Policy Fellow by the Robert Wood Johnson Foundation and the National Academy of Medicine. The prestigious one-year fellowship in Washington, D.C., will enable her to expand her longtime involvement in health policy and child policy at the local and state levels to the federal level.

July

The University becomes one of 89 locations around the nation, and one of only six in Florida, to enroll volunteers for the first Phase 3 clinical trial of a COVID-19 vaccine. The trial, part of the National Institutes of Health COVID-19 Prevention Trials Network, tested a vaccine developed by scientists at the NIHs National Institute of Allergy and Infectious Diseases and collaborators at biotechnology company Moderna, Inc. Vice President Mike Pence visited the Miller School of Medicine along with Florida Gov. Ron DeSantis on July 27 to thank the University for its participation in the trial.

The National Oceanic and Atmospheric Administration selects the Rosenstiel School of Marine and Atmospheric Science to host the Cooperative Institute for Marine and Atmospheric Studies, which will bring together the research and educational resources of 11 partner universities to increase scientific understanding of the Earths oceans and atmosphere within the context of NOAAs mission. The selectionmade through an open, competitive evaluationcomes with an award of up to $310 million over the course of five years, with the potential for renewal for another five years based on successful performance.

Amid ongoing nationwide protests against police brutality sparked by the tragic death of Minneapolis resident George Floyd in May, President Julio Frenk reaffirms his commitment to racial and ethnic equality, outlining in a letter sent to all students, faculty, and staff a 15-point plan the University will implement to support racial equality, inclusion, and justice across the institution and in the greater South Florida community.

Patti Herberta longtime University of Miami alumna and benefactor who, along with her husband Allan, donated millions of dollars to the institution, helping to transform academics and student lifepassed away on Monday, July 27. She was 84.

University of Miami Libraries launches Documenting COVID-19: South Floridas Pandemic Experience. Through community-generated and community-contributed content that will be made available through digital collections and by visiting the library, the initiative will chronicle how local communities are dealing with the crisis.

August

In a move to support a safe learning and working environment for students, faculty, and employees, the Butler Center for Service and Leadership establishes a new team of public health ambassadors to support the Universitys COVID-19 reopening and operating plan. The 75 students who made up the new Public Health Ambassadors Program during the fall semester enforced guidelines on the Coral Gables Campus by offering support and utilizing peer-to-peer influence to encourage members of the campus community to engage in the healthy behaviors of physical distancing, wearing face coverings, and hand washing/sanitizing.

Lakeside Village, a 12-acre facility on the shores of Lake Osceola in the heart of the Coral Gables Campus, officially opens its doors on Aug. 13 to greet the inaugural class of students to the transformative housing complex.

Featuring a mix of virtual and in-person instruction, the first day of fall semester classes begins on Aug. 17. On the Coral Gables Campus, a number of safety protocolsfrom mandatory mask-wearing to social distancinghelp ensure the well-being of students, faculty, and staff.

The Miller School of Medicine Class of 2024 launches the innovative NextGenMD Curriculum, which focuses on health system science and features an enhanced emphasis on mentorships. The students will be significantly better prepared to respond to COVID-19 and to the public health challenges that will follow.

The Miller School of Medicine becomes one of a few medical schools across the nation selected by the National Institutes of Health to test the effectiveness of treating COVID-19 patients with convalescent plasma.

Following a limited opening in April 2020, Canes Central, a new student-centered, service-oriented department, fully opens. It offers both in-person and online undergraduate and graduate students assistance on matters relating to registration and records, billing and payment, financial aid, and Cane Cards.

Legendary Miami Hurricanes baseball coach Jim Morriswho in his 25 seasons at the University of Miami won 1,090 games, made the NCAA postseason 23 straight years, reached the College World Series 13 times, and won national championships in 1999 and 2001is voted into the 2020 induction class of the National College Baseball Hall of Fame.

September

Sylvester Comprehensive Cancer Center at the University of Miami Leonard M. Miller School of Medicine receives a landmark gift of $126 million. The groundbreaking donationthe single largest in the University of Miamis 95-year historywill accelerate breakthrough advances in finding cures for cancer and expand innovative treatment options for cancer patients.

The University of Miami jumps eight spots to No. 49 in U.S. News & World Reports 2021 Best Colleges issue, placing the institution back among the 50 top-tier colleges and universities. This jump in the rankings reflects our commitmenteven in these unprecedented timesto comprehensive excellence and selective preeminence, said President Julio Frenk.

Sylvester Comprehensive Cancer Center, part of the University of Miami Health System, opens the Dwoskin Proton Therapy Center on Sept. 15. The new state-of-the-art facility treats patients with proton therapy, an advanced type of low-dose radiation that is extremely precise and two-thirds the speed of light.

Physician-researchers with the Miller School of Medicine begin a new Phase 3 clinical trial to test another investigational vaccine for COVID-19. Part of a large-scale international trial in partnership with Janssen Pharmaceuticals, the clinical trial to test the Janssen vaccine is the Miller Schools second human study of its kind.

October

The University of Miami becomes the first college testing site for a quick, easy, and cost-effective Israeli-produced COVID-19 breath analyzer that could revolutionize coronavirus testing if approved by the FDA.

In a Miami Herbert Business School webinar held Oct. 8, U.S. Secretary of Health and Human Services Alex Azar highlights the administrations effort to restructure the health care system to combat the COVID-19 pandemic and support countries in Latin America and the Caribbean.

November

Taking their dedication to fighting cancer to a new level, the Miami Dolphins pledge a transformational $75 million gift to Sylvester Comprehensive Cancer Center at the University of Miami Leonard M. Miller School of Medicine, South Floridas only NCI-designated cancer center.

Musicians from the Frost School of Music join Dean Shelly Berg and celebrity musicians to participate in a benefit concert on Thanksgiving Day in support of nurses. The livestreamed Nurse Heroes Live! concert raises funds for the Nurse Heroes Foundation, an initiative working to support and honor nurses.

December

Four extraordinary University of Miami alumniJose R. Mas, Jackie Nespral, Hilarie Bass, and Jaret L. Davisshare their advice with more than 5,000 students at four virtual commencement ceremonies held Dec. 10 and 11.

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2020 at the U: The year in review - University of Miami

COVID-19 has many symptoms, including fever, coughing, and fatigue. But one of the more distinctive signs is the loss of the ability to smell. Were not talking about the usual stuffy nose that goes along with a cold, but an inability to process scent even when youre not congested. People have reported that not being able to smell their own perfume or finding no aroma in their cup of mint tea was their first clue that they might be infected.

James Schwob, a professor of developmental, molecular, and chemical biology at Tufts University School of Medicine, researches the olfactory system and the roughly 1,000 types of neurons that are involved in our ability to register odors both good and bad.

Right now, he is studying tissue from COVID-19 patients to better understand how the virus leads to anosmia, or loss of sense of smell. Tufts Now talked to Schwob about what we know about viruses and their effects on sense of smell.

Tufts Now: How does sense of smell work?

James Schwob: The sense of smell operates by chemicals wafting in on the air and reaching the upper and back parts of the nasal cavity. Those chemicals bind to receptors on sensory neurons in the epitheliumthe thin tissue that lines the nasal passages. Those neurons then send a signal up the olfactory nerve into the brain, where it registers as the delicious smell of coffee or fresh cut grass.

What causes you to lose it?

There are a number of pretty well-known causes for loss of sense of smell. One is via post-viral infection, and we think that has something to do with the immune system causing inflammation. It also happens with head injuryin that case, its likely the part of the brain that receives the smell signals that is damaged. Toxin exposurefrom cadmium, formaldehyde, or methyl bromide, for examplewill make you lose your sense of smell. Chronic sinus infections and simply the aging process can also cause anosmia.

How common is loss of sense of smell among COVID-19 patients?

We have long known that people can lose their sense of smell after other viral infections, such as the flu, but the percentage of people who have had this problem with COVID-19 is quite remarkable. One study from Iran reported 98 percent of hospitalized patients had an objective problem with their sense of smell.

How does COVID-19 cause people to lose their sense of smell?

We dont really know why this happens with COVID-19. It could be that the virus is harming the neurons that send smell signals to the brain, or that the bodys immune system, in trying to deactivate the virus, is killing other, supporting cells that are part of that pathway.

There is some evidence that certain cells in the lining of the nasal passages express a protein receptor, called ACE2, that the coronavirus uses it as a way to infiltrate the body. That is one of the things Im hoping to investigate.

Back in 2000, colleagues and I published two papers looking at a different coronavirus, called mouse hepatitis virus. We looked at what effect that had on the peripheral olfactory nerve in the central olfactory system. What we found was that this virus would pass up that nerve into the brain and cause problems in the brain.

One of the other things that has been described is that there have been some neurological symptoms due to the infection with the SARS-CoV2-virus. And one of the questions we have is whether the virus is crawling up the nerve in some fashion in these patients who have died of the infection.

The good news is that the olfactory epithelium contains stem cells that can give birth to new neurons throughout life as long as they remain intact. So the system has a capacity to repair itself. Some COVID-19 patients have recovered their sense of smell within a couple weeks. Thats actually quicker than you would expect new neurons to be created, so there could be some sort of functional disruptionrather than neuron deathgoing on.

With all the more serious symptoms associated with COVID-19, why is sense of smell worth investigating?

Sometimes loss of smell is a COVID-19 patients only symptom. Any symptom that can be tied directly to the disease becomes an important one to be aware of, so that it can be used to guide testing and keep people from unknowingly spreading the disease. That is part of the reason I think its important to figure this out.

An intact sense of smell is also critical to good nutrition. If smell is lost so is most of foods flavor. As a consequence, patients may overeat (to try to get the pleasure back), undereat (why bother?) or over-salt or -spice their food, because those aspects of food flavor can still be detected by nerves and taste buds in the oral cavity.

Loss of sense of smell can be very disturbing, because eating is of our great pleasures in life. We dont want to lose that when we have so few pleasures left to us now that were stuck at home.

How can a person know if their sense of smell is really hampered, and they arent just imagining it?

One of the things that can be done pretty easily, pretty objectively by someone at home would be to take some ground coffee and see how far away you can hold it and still smell it. Or do the same with rubbing alcohol or your shampoo. If your nose is not congested and you have trouble recognizing those or other scents that are familiar to you, you might want to call your doctor about getting tested.

Julie Flaherty can be reached at julie.flaherty@tufts.edu.

Link:
Something Wrong With Your Sniffer? It Could Be the Coronavirus - Tufts Now

I

m going to turn 40 this month, a milestone Im approaching with nine-tenths equanimity, one-tenth mild existential dread.

On the one hand, with a kid and a job, Im too busy to spend much time peering at my newly middle-aged navel. Plus, if youre lucky enough to be healthy, griping about simply growing older is churlish. On the other, there is an undeniable psychic wrench in the realisation that I now tick the aged 40-60 box. Somehow, I seem to have lurched from young and clueless to the age where people will make jokes about the number of candles on my cake, without an intervening period of solid competence. I still run for the bus. I can still never find the lids for the Tupperware. I am older than the prime minister of Finland, but I cant reliably pair my own socks. I dont feel fully grown up, and yet there is the creeping sense that, as Sue Townsend put it via her timeless mouthpiece Adrian Mole, Im on a pathetic slide towards gum disease, wheelchair ramps and death.

But is this slide inevitable? Thats the central question of Ageless: The New Science of Getting Older Without Getting Old, an ambitious and energetic new book by the scientist and writer Andrew Steele. While hes not peddling some holy grail of immortality, he does give a startling round-up of the biological factors that make us age and the emerging techniques to tackle them, offering the prospect of both longer and healthier lives.

To start with some perspective: human lifespans vary hugely based on time and place, with life expectancy under the age of 40 the norm everywhere at the start of the 19th century, largely due to high child mortality. Drawing examples from the animal kingdom, Steele points out that innings vary from five minutes for a mayfly to 400 years for a Greenland shark. We are, of course, neither flies nor sharks. But if we accept the idea that a human life isnt capped at 82 and pursue all the opportunities of frontier research, perhaps we could become more like tortoises. As Steele explains, the Galapagos tortoise, along with a handful of other blessed oddballs in the natural world, are negligibly senescent meaning they have no obvious impairments of movement or senses as they get older, and they experience no age-related declines in fertility.

They dont live forever. But referring to a giant tortoise discovered by Charles Darwin who lived until 2006, Steele writes: Harriet was likely pretty much as sprightly at 170 as she was at 30, at the height of Queen Victorias reign which is to say, not very; she was a giant tortoise, after all.

This liveliness of tone helps to carry the reader through an unsparing account of the toll ageing takes on the human body. The comic writer Nora Ephron may have called her mature memoir I Feel Bad About My Neck, but ageing emerges from this book as less aesthetic challenge, more devastating, systemic collapse. The gloomiest statistic of the lot? Your chance of death doubles every eight years, as a range of processes make us progressively more susceptible to big killers like cancer, heart disease, stroke, dementia and diabetes.

Steele gives a jauntily accessible account of the mechanisms behind our decline. One overarching driver is disposable soma theory: the evolutionary logic that prioritises the health of our reproductive cells over the soma cells which make up our own bodies. I had always suspected that having a child somehow drained me of my life force: here is the evidence. And as our neglected soma cells age, things go wrong. Our telomeres, protective caps on our chromosomes which are essential for healthy cell division, get shorter. Autophagy the process by which cells clear out their own junk declines. Proteins misfold into sticky clumps that risk forming the plaques behind Alzheimers disease. Frail old cells linger: They stick around, no longer dividing aged, zombie cells which refuse to commit cell suicide, known as senescent cells. Our mitochondria which give cells energy misfunction. Stem cells falter. Chronic inflammation ups the risk of everything from diabetes to cancer. Its a startling wake-up call that there are worse things bubbling away in your biology than a few grey hairs, or the frown lines that make every Zoom call of this pandemic a harrowing ordeal for the over-35s.

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But what if there was another way? Dont book your return trip to the moon for 2125 just yet. The science has a long way to go: as Steele points out, most of the research is in its early stages, bristling with the potential for pitfalls and unintended consequences. But there are a number of approaches that hold promise, with todays huge advances in computing spurring new therapies. Steele posits that senolytics, or drugs that destroy senescent cells, could be with us in the next few years, albeit to tackle age-related conditions rather than ageing itself. After that, more advanced treatments like gene and stem cell therapies could be available on timescales measured in decades. Ultimately, instead of tackling the individual symptoms of ageing a creaky knee here, a furred-up artery there we will move towards systems medicine that stops us falling to bits in the first place:

The first ageless generation probably wont realise their luck at first theyll grow up expecting to die at 100, or 150, or whatever old is for their society but, one after another, lifesaving medical breakthroughs will push their funerals further and further into the future.

Steele writes with the maverick confidence of the outside perspective. He took a PhD in physics, then moved into computational biology before pursuing his interest in ageing as a writer. In this career switch, he has something in common with Aubrey de Grey, the bearded high priest of the anti-ageing movement who began with a background in computer science. While Steele stops short of making outlandish predictions de Grey famously ruffled academic feathers with his claim people could live to be 1,000 the logic is the same.

While we would all merrily jettison our wrinkles and give bowel cancer a miss, are we really ready for an ageless society, presuming the science stacks up? Steele writes quite reasonably that no one would invent the suffering of old age as a solution to an over-crowded world, but this research nonetheless opens up a fascinating Pandoras box of challenges. Rather than a utopia of dewy-faced centenarians hover-boarding to work, we would probably first see a deepening of the grotesque inequalities that already exist in health today. Theres currently a nine-year gap in lifespans between the richest and poorest parts of the UK. Add some fancy preventive drugs to the mix and you can guess who would get them first. Moreover, with ageism rife in the workplace, especially in the tech industry which provides an increasing number of jobs, who could afford not to quaff the pills that keep you looking fresh in your hoodie?

Writing with the vim of a Bill Bryson and the technical knowledge of a scientist, Steele at least gives us a chance to grasp whats at stake in this dazzling, daunting age where big data meets human biology. Negligible senescence may remain a stretch for my New Years resolutions, but Id happily progress at a more tortoise-like pace towards the next milestones of decrepitude.

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Science can stop us ageing, according to a new book. But do we want it to? - The Independent

Three University of Pennsylvania researchers have been honored by The Sanford and Sue Greenberg Prize to End Blindness by 2020 for their research, which led to the first Food and Drug Administration-approved gene therapy for a genetic disease. Gustavo D. Aguirre of the School of Veterinary Medicine and Jean Bennett and Albert M. Maguire of the Perelman School of Medicine, together with William Hauswirth of the University of Florida, are recipients of the Outstanding Achievement Prize, to be awarded in a virtual ceremony today.

The four scientists share a $1 million prize, funds that will go to support further laboratory and clinical research that advances vision science. Together, their workgoing from an animal model of disease to human clinical trialsled to an FDA-approved gene therapy for Leber congenital amaurosis (LCA) caused by a mutation in the RPE65 gene, a retinal disease that causes visual impairments beginning in infancy. Now commercialized and used routinely, this treatment has reversed blindness and spared the vision of children and adults around the world.

The researchers are among 13 recipients of a total of $3 million from the End Blindness by 2020 initiative, originated by Sanford Greenberg, an

inventor, entrepreneur, and investor who serves as chairman of the board of the Johns Hopkins University Wilmer Eye Institute and who lost his sight at the age of 19 due to disease.

Gustavo D. Aguirre is professor of medical genetics and ophthalmology in the School of Veterinary Medicine. His internationally renowned research, generated during more than four decades, has investigated the genetic basis of a variety of inherited vision disorders, including LCA, Best disease, achromatopsia, and retinitis pigmentosa. His work on novel gene therapy approaches to treatment, which deliver to the eye a functional copy of a gene that is otherwise dysfunctional, has restored vision in animal models of X-linked retinitis pigmentosa and LCA. Aguirre, who earned his V.M.D. and Ph.D. from Penn, is a fellow of the Association for Research in Vision and Ophthalmology and a member of the National Academy of Medicine, College of Physicians of Philadelphia, and American Association for the Advancement of Science. He is also a recipient of the Louis Braille Award, Proctor Medal, and Foundation Fighting Blindess Board of Directors Award, among other honors.

Jean Bennett is the F.M. Kirby Professor of Ophthalmology in the Perelman School of Medicine. She is an internationally-recognized pioneer in gene therapy and has dedicated her career to restoring eyesight in the blind. Bennett earned a Ph.D. in zoology and cell and development biology from the University of California, Berkeley, and a medical degree from Harvard University, where she met her future husband and research collaborator, Albert M. Maguire. She has developed a number of strategies for gene therapy-mediated treatments for retinal disease. Her research is focused on the molecular genetics of inherited retinal degenerations in order to develop rational approaches for treating blindness. In addition to the eye, projects in Bennett's laboratory target other diseases/organs suffering from mutations in cilia proteins, including the ear and the kidney.

Albert M. Maguire is a professor of ophthalmology in the Perelman School of Medicine and an attending physician in the Division of Pediatric Ophthalmology at Children's Hospital of Philadelphia. He earned his medical degree from Harvard University, completed an internship in surgery at Yale-New Haven Hospital, a residency at Johns Hopkins Hospital, and a fellowship at the William Beaumont Hospital in Royal Oaks, Michigan. Maguire specializes in the diagnosis and treatment of pediatric retinal diseases. His research interests involve the development of treatments for incurable retinal degenerative disease, including LCA.

The hourlong streamed ceremony, freely accessible at http://www.EndBlindness2020.com, will feature Art Garfunkel, Margaret Atwood, Al Gore, Michael Bloomberg, U.S. Sen. Chris Coons, and musical performances. It will also feature a tribute to the late U.S. Supreme Court Justice Ruth Bader Ginsburg, a longtime supporter of the End Blindness movement, including exclusive footage of Ginsburg reading from Hello Darkness, My Old Friend, the memoir of Sanford Greenberg.

More here:
Vision researchers honored by End Blindness 2020 | Penn Today - Penn Today

"Kodi's inspirational tenacity and spirit is exactly the empowerment the Foundation wants to provide our community."

Kodi's Music to Our Eyes performance will feature a wide variety of covers from his musical repertoire, including You Are The Reason by Calum Scott and Don't Let The Sun Go Down On Me by Elton John. Kodi will also perform holiday favorites, Winter Wonderland by Johnny Mathis and Santa Claus Is Coming to Town by Michael Bubl.

In between sets, Jason Menzo, chief operating officer at the Foundation Fighting Blindness, will interview Kodi and his siblings, Derek and Kayla, about their experiences with Kodi's vision loss and discuss his determination to achieve his dreams no matter what obstacles come his way. Event registration is free, but attendees will have the opportunity to contribute, as all net proceeds will go towards the Foundation's mission.

"We're excited to be collaborating with Two Blind Brothers again for a livestream musical performance with Kodi Lee," says Jason Menzo, COO at the Foundation. "Kodi's inspirational tenacity and spirit is exactly the empowerment the Foundation wants to provide for the blind and low vision community."

For more information and to register for a reminder about this event, visit: https://bit.ly/3m84Wkr

About the Foundation Fighting BlindnessEstablished in 1971, the Foundation Fighting Blindness is the world's leading private funding source for retinal degenerative disease research. The Foundation has raised more than $800 million toward its mission of accelerating research for preventing, treating, and curing blindness caused by the entire spectrum of retinal degenerative diseases including: retinitis pigmentosa, age-related macular degeneration, Usher syndrome, and Stargardt disease. Visit FightingBlindness.org for more information.

Media Contacts:Chris AdamsVice President, Marketing & Communications[emailprotected]tingblindness.org (410) 423-0585

SOURCE Foundation Fighting Blindness

http://www.FightBlindness.org

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Kodi Lee Teams up with Foundation Fighting Blindness and Two Blind Brothers for Music to our Eyes Livestream Music Series - PRNewswire

Glaucoma refers to the group of conditions characterized by optic nerve damage, visual field loss, and secondary to retinal ganglion cell damage, which may lead to death. According to the International Agency for the Prevention of Blindness: 2016, glaucoma is second leading cause of blindness and leading cause of irreversible blindness worldwide. The most common types of glaucoma are Primary Open Angle Glaucoma (POAG) and Primary Angle Closure Glaucoma (PACG). Moreover, PACG is most common in South-East Asian population while POAG is most common in white Caucasians and individuals of African origin. PACG is associated with a high risk of blindness as compared to POAG.

Glaucoma is a group of eye diseases which result in damage to the optic nerve and cause vision loss. The most common type is open-angle (wide angle, chronic simple) glaucoma, in which the drainage angle for fluid within the eye remains open, with less common types including closed-angle (narrow angle, acute congestive) glaucoma and normal-tension glaucoma. Open-angle glaucoma develops slowly over time and there is no pain. Peripheral vision may begin to decrease, followed by central vision, resulting in blindness if not treated. Closed-angle glaucoma can present gradually or suddenly. The sudden presentation may involve severe eye pain, blurred vision, mid-dilated pupil, redness of the eye, and nausea. Vision loss from glaucoma, once it has occurred, is permanent. Eyes affected by glaucoma are referred to as being glaucomatous.

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Risk factors for glaucoma include increasing age, high pressure in the eye, a family history of glaucoma, and use of steroid medication. For eye pressures, a value of greater than 21 mmHg or 2.8 kPa is often used, with higher pressures leading to a greater risk. However, some may have high eye pressure for years and never develop damage. Conversely, optic nerve damage may occur with normal pressure, known as normal-tension glaucoma. The mechanism of open-angle glaucoma is believed to be slow exit of aqueous humor through the trabecular meshwork, while in closed-angle glaucoma the iris blocks the trabecular meshwork. Diagnosis is by a dilated eye examination.Often, the optic nerve shows an abnormal amount of cupping.

If treated early, it is possible to slow or stop the progression of disease with medication, laser treatment, or surgery. The goal of these treatments is to decrease eye pressure. A number of different classes of glaucoma medication are available. Laser treatments may be effective in both open-angle and closed-angle glaucoma. A number of types of glaucoma surgeries may be used in people who do not respond sufficiently to other measures. Treatment of closed-angle glaucoma is a medical emergency.

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ProstaglandinsAlpha AgonistBeta BlockersCarbonic Anhydrase InhibitorCholinergicCombined Medication

Application Coverage (Market Size & Forecast, Different Demand Market by Region, Main Consumer Profile etc.):

HospitalsOphthalmic ClinicsAmbulatory Surgical Centers

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Glaucoma Therapeutics The market is expected to see a surge in growth | Allergan , Merck , Novartis - The Courier

Night blindness, also known as nyctalopia, is a type of vision impairment that makes it difficult to read or see in dim light or at night. It is not a disease itself, but rather a symptom of an underlying medical condition. Although the name suggests blindness during the night, it usually means difficulty in reading or driving in a dusky environment, which can happen any time during the day.

Difficulty to see in a dark room is usually normal but in nyctalopia, it is more strenuous. It can even prevent you from pointing out the stars in the night sky. Night blindness may make it difficult to recognize faces or locate obstacles in a dimly-lit room. Your vision could also take longer than usual to adjust to the transition from darkness to bright light.

Causes of night blindness

Night blindness may be caused by a variety of underlying causes including the following:

Treatment for night blindness

It is important to know the exact cause of the condition for an appropriate intervention to be planned. The treatment for nyctalopia may vary from simply getting a new pair of prescription glasses or switching glaucoma medications to surgery if the cause is cataracts.

A proper balance diet may also be advised if the condition is caused due to a dietary imbalance. Vitamin and mineral requirements may be fulfilled by supplementation, depending on your doctors recommendation. If a retinal disease is discovered, the treatment will be carried out by an ophthalmologist depending on the type and severity of the condition.

Unfortunately, treatment of genetic conditions such as retinitis pigmentosa and Usher syndrome that cause nyctalopia are yet to be developed.

Prevention and precautions

There is no way to prevent night blindness when it is caused by genetic conditions or birth defects. However, in the case of other causes, you can take the following measures to reduce the risk of developing the condition:

For more information, read our article on Night Blindness.

Health articles on News18 are written by myUpchar.com, Indias first and biggest resource for verified medical information. At myUpchar, researchers and journalists work with doctors to bring you information on all things health.

Link:
Nyctalopia: All You Need to Know About Night Blindness - News18

PETALUMA, Calif., Dec. 15, 2020 /PRNewswire/ --RetinalGeniXTM Technologies, Inc. https://retinalgenix.com/ announced today the engagement of Amato and Partnersto provide Investor Relations services. RetinalGeniXTM Technologies is a private California-based medical technology company focused on prevention of blindness through mass medical retinal screening and patient home monitoring.

RetinalGeniX is moving forward with a strategic investor relations plan that will be supported by Amato and Partners. Amato and Partners is an independent Investor Relations firm headquartered in New York City. The firm has a strong and well-recognized brand in the U.S. capital markets. The Amato team has over 40 years of experience developing and executing investor relations programs and a proven track record of achieving results both for private and public companies.

"Amato and Partners provides experienced advice and established Wall Street relationships that will cultivate company visibility with market participants including equity research analysts, investment banks and appropriate potential investors," said Jerry Katzman, M.D., RetinalGeniX's chief executive officer. "We very much look forward to working together."

About RetinalGeniX Technologies, Inc. RetinalGeniX Technologies, Inc. is a privately held emerging medical device company focused on preventing blindness through a cost-effective Mass Retinal Screening Device and a Patient Real-Time Home Monitoring Imaging & Physician Alert System. (A) The Mass Retinal Screening device provides retinal imaging that captures a 200 FOV without pupil dilation and detects the earliest stages of diabetic retinopathy to prevent blindness. This device requires submission to the FDA's 510(k) approval process. (B) RetinalGeniX's second product is RetinalCam, the first in-home, self-monitoring device providing real-time ocular and retinal imaging and streams video to the physician. The technology enables high-risk individuals to send their physician digitized retinal images and streaming video easily from their home. This second device does not require submission to the FDA's 510(k) approval.

Safe Harbor Statement This press release includes "forward-looking statements" within the meaning of the Private Securities Litigation Reform Act of 1995. Forward-looking statements in this press release include, but are not limited to, statements that relate to the advancement and development of the Mass Retinal Screening Device and a Patient Real-Time Home Monitoring Imaging & Physician Alert Systemand other information that is not historical information. When used herein, words such as "anticipate", "being", "will", "plan", "may", "continue", and similar expressions are intended to identify forward-looking statements. In addition, any statements or information that refer to expectations, beliefs, plans, projections, objectives, performance or other characterizations of future events or circumstances, including any underlying assumptions, are forward-looking. All forward-looking statements are based upon RetinalGeniX's current expectations and various assumptions. Voltron believes there is a reasonable basis for its expectations and beliefs, but they are inherently uncertain. RetinalGenix may not realize its expectations, and its beliefs may not prove correct. Actual results could differ materially from those described or implied by such forward-looking statements as a result of various important factors, including, without limitation, market conditions. Consequently, forward-looking statements should be regarded solely as RetinalGeniX's current plans, estimates and beliefs. Investors should not place undue reliance on forward-looking statements. RetinalGenix cannot guarantee future results, events, levels of activity, performance or achievements. Voltron does not undertake and specifically declines any obligation to update, republish, or revise any forward-looking statements to reflect new information, future events or circumstances or to reflect the occurrences of unanticipated events, except as may be required by law.

Contact:Jerry Katzman, M.D.Chief Executive Officer(415) 578-9583[emailprotected]

SOURCE RetinalGeniX Technologies, Inc.

https://retinalgenix.com/

Link:
RetinalGeniX Has Engaged New York Investor Relations Firm Amato and Partners, LLC - PRNewswire

Many neurodegenerative conditions, from glaucoma to Alzheimers disease, are characterized by injury to axons the long, slender projections that conduct electrical impulses from one nerve cell to another, facilitating cellular communications. Injury to axons often leads to neuronal impairment and cell death.

Researchers know that inhibiting an enzyme called dual leucine zipper kinase (DLK) appears to robustly protect neurons in a wide range of neurodegenerative diseases models, but DLK also inhibits axonal regeneration. Until now, there have been no effective methods to modify genes to improve both the long-term survival of neurons and promote regeneration.

In a paper published December 14, 2020 in PNAS, a multi-university team led by researchers at University of California San Diego School of Medicine and Shiley Eye Institute at UC San Diego Health identified another family of enzymes called germinal cell kinase four kinases (GCK-IV kinases) whose inhibition is robustly neuroprotective, while also permitting axon regeneration, making it an attractive therapeutic approach for treating at some neurodegenerative diseases.

We basically figured out that there are a set of genes that, when inhibited, allow optic nerve cells to survive and regenerate, said senior author Derek Welsbie, MD, PhD, associate professor of ophthalmology in the Viterbi Family Department of Ophthalmology at Shiley Eye Institute.

Example of retinal ganglion cells with axons and dendrites in the retina of a healthy eye.

Prior to this work, the field knew how to get these cells to survive, but not regenerate. Conversely, there are ways to promote regeneration, but then the survival was rather modest. Of course, for a successful strategy of vision restoration, you need both and this is a step in that direction.

The researchers conducted a series of screens after first creating retinal ganglion cells (RGC) from human stem cells. RGCs are a type of neuron located near the inner surface of the retina of the eye. They receive visual information from photoreceptors and collectively help transmit that information to the brain.

The first screen involved testing a group of well-studied chemicals to assess their ability to increase the survival of RGCs; the second to measure the ability of chemicals to promote regeneration.

We then used a machine-learning technique to understand why certain compounds were active while others were not and it identified these key genes, said Welsbie.

The discovery that these genes improved RGC survival was not surprising, he said. However, you would have predicted that they (like DLK) would have blocked regeneration when inhibited, not promote regeneration. That was definitely a surprise. It highlights one of the advantages of discovery-based science using high-throughput screening: By testing many agents at once, we can find identify overlooked genes that might not have been thought to play a role.

Welsbie and colleagues focused their work on RGCs because they are interested in optic neuropathies, such as glaucoma. Most people think only about glaucoma in terms of eye pressure, Welsbie said. But eye pressure is only part of the problem. At its core, glaucoma is a neurodegenerative disease characterized by progressive loss of RGCs and their axons, leading to measurable structural and functional damage to the optic nerve, visual impairment and blindness.

The U.S. Centers for Disease Control and Prevention estimate 3 million Americans have glaucoma. It is the second leading cause of blindness worldwide.

Welsbie cautioned that its not yet known whether these findings extend to other neuron types, but he noted that the work suggests strong therapeutic possibilities.

Co-authors include: Amit K. Patel, Risa M. Broyer, Cassidy D. Lee, Tianlun Lu, Mai T. Vu, Karl J. Wahlin and Robert N. Weinreb, all at UC San Diego; Mikaela J. Louie, Anna La Torre and Yang Hu, UC Davis; Hassan Al-Ali, John L. Bixby and Vance P. Lemmon, University of Miami; Katherine L. Mitchell and Vinod Jaskula-Ranga and Donald J. Zack, Johns Hopkins University; Xin Duan, UC San Francisco; Santiago Vilar, Truvitech, Miami.

Funding for this research came, in part, from the National Institutes of Health (grant 1RO1EY029342), Research to Prevent Blindness, E. Matilda Ziegler Foundation, Brightfocus Foundation, Fight for Sight Foundation, the Glaucoma Research Foundation Catalyst for the Cure and the Tushinsky family.

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Researchers Discover Clue to How to Protect Neurons and Encourage Their Growth - UC San Diego Health

Jos-Alain Sahel was on a rare vacation in Portugal in the spring of 2018 when his phone rang with grim news: The gene therapy he had worked on for a decade, a potential cure for a rare form of blindness, had failed in a pivotal trial.

In the first minute, I was very disappointed, Sahel says. I said, well OK, its not working.

A failed trial in drug development is crushing but not unexpected, a tradeoff of doing business in biology. You examine the full data, go back to the drawing board and either abandon the effort or tweak and try again. Sahel, founder of four companies and the longtime head of the Vision Institute of Paris, was used to the process. But this time, when the full data came, he was bewildered.

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They thought their gene therapy failed. Instead, it spawned a medical mystery - Endpoints News