Category Archives: Stem Cell Medical Center

Multi-Hit TP53 Mutations Have Major Impact on Posttransplant … – AJMC.com Managed Markets Network

People with myelofibrosis (MF) who undergo hematopoietic stem cell transplantation (HSCT) may have a greater risk of adverse outcomes if they have multiple TP53 mutations, according to a new study.

Those risks include a significantly higher likelihood of leukemic transformation shortly after transplantation, according to the report, which was published in Blood.

The study authors explained that mutations of the TP53 gene have long been associated with inferior outcomes in patients with hematologic malignancies. However, they wrote that there is little scientific evidence regarding the prognostic effects of TP53 mutations in people with MF.

To help fill the gap, the investigators used a large, multicenter cohort of 349 patients with MF who underwent their first HSCT between 1997 and 2001. Of those, 49 patients (13%) had mutations of TP53. Thirty of those patients had a multi-hit mutational configuration, and the rest had single-hit configurations.

The investigators used the International Consensus Classifications to categorize patients as single- or multi-hit cases. Multi-hit constellations were defined as the presence of 2 or more distinct mutations of TP53 with variant allele frequency of equal to or greater than 10%, one mutation plus 1 deletion involving the TP53 locus at 17p, 1 mutation with a variant allele frequency equal to or greater than 50%, or 1 mutation plus complex karyotype. Single-hit cases were defined as the absence of multi-hit constellations and a variant allele frequency of less than 50%.

Most of the people in the study cohort had a favorable cytogenetic risk profile (71%), while 23% had unfavorable cytogenetic risk and 6% were classified as very high risk. Thirty-six patients (10%) had a complex karyotype.

The data showed patients with TP53 mutations had significantly shorter median survival compared with patients with wild-type TP53: 1.5 vs 13.5 years, respectively. However, that gap seemed to be largely based upon whether the patient was categorized as multi-hit or single-hit. Among patients with multi-hit TP53 mutations, the 6-year survival rate was 25%, but for patients with single-hit mutations, the survival rate was 56%. The latter figure was similar to that of people with wild-type TP53, who had a survival rate at 6 years of 64%.

People with multi-hit constellations also had a much higher cumulative incidence of relapse (52% vs 17% for single-hit mutations vs 21% for people with wild-type TP53). Likewise, of the 10 people with TP53 mutations who experienced leukemic transformation, 8 had mult-hit constellations. In the wild-type cohort, 7 patients had leukemic transformation.

Our data, in line with previous reports in the nontransplant setting investigating patients with myelodysplastic syndromes and acute myeloid leukemia, clearly show that multi-hit TP53MT significantly increases the risk of relapse and, more importantly, leukemic relapse, in patients with myelofibrosis receiving HSCT, the study investigators wrote.

Clinically, they noted that people with multi-hit TP53 mutations were more likely to have severe anemia and thrombocytopenia at baseline, although other disease-specific and patient-specific characteristics were generally balanced between the cohorts.

The investigators also noted that posttransplant relapse remains the major cause of treatment failure, and they said more research is needed to better understand the role next-generation sequencing might play in monitoring patients and making treatment decisions.

Our data are not only of immediate clinical relevance for the transplant and nontransplant community, but also highlight the need for translational and clinical studies focused on understanding the mechanism of TP53MT specifically in patients with myelofibrosis, they concluded.

Reference

Gagelmann N, Badbaran A, Salit RB, et al. Impact of TP53 on outcome of patients with myelofibrosis undergoing hematopoietic stem cell transplantation. Blood. Published online March 20, 2023. doi:10.1182/blood.2023019630

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Multi-Hit TP53 Mutations Have Major Impact on Posttransplant ... - AJMC.com Managed Markets Network

A young boys nightmare diagnosis, and the $3 million one-time … – The Boston Globe

Youre getting your stem cells back, baby, Conners mother, Richelle Brooks, said on the other side of the bed, wiping her tears with a tissue as excited hospital employees crowded the room.

To fix your boo-boo, Adam added, using the phrase doctors suggested his parents employ when talking with Conner about the ultra-rare genetic condition, cerebral adrenoleukodystrophy, or CALD.

The devastating disorder strikes boys, usually between the ages of 4 and 10, causing rapid loss of cognitive and physical abilities, including hearing, vision, and movement, often only months after symptoms first appear. Most boys die within a few years.

Conner, who had spent eight days undergoing a grueling drug regimen to make room in his bone marrow for the stem cells, stirred but stayed asleep as nurses whooped and clapped during the half-hour, one-time infusion on March 16.

A spirited boy from upstate New York who loves the online game Roblox and has no obvious symptoms, Conner is the first patient to receive the gene therapy since the Food and Drug Administration approved it in September.

Marketed as Skysona, it is one of six gene therapies cleared since 2017 that have transformed the outlook for disorders once considered hopeless. It has also renewed thorny questions about how the health care system can afford such breathtakingly expensive medicines.

Skysona was developed by Bluebird Bio, a Somerville biotech that specializes in gene therapies for rare diseases. Conners disorder afflicts only hundreds of patients in the United States according to experts. Few people had heard of it until it was depicted in the 1992 movie Lorenzos Oil.

In a clinical trial that followed CALD patients for two years, 91 percent of 32 children who received a single dose of Skysona survived without developing any major functional disabilities. Another 35 children underwent the treatment in a follow-up study, researchers say, and the results have held up so far. The first patient in the trials, a boy from Northern Ireland, received it at Boston Childrens Hospital almost a decade ago and is still doing well.

But Skysona poses the risk of potentially serious side effects. And because it treats a disorder that causes an irreversible buildup of toxic fatty acids and inflammation in the brain, the gene therapy must be given before symptoms appear. After that, the neurological damage is done. That means early detection of the faulty gene that causes the disease is crucial.

Over the past decade, 36 states Massachusetts not among them have mandated that newborns be screened for the genetic mutation through a blood test, partly as a result of prodding by a Brooklyn woman whose 7-year-old son died of CALD in 2012. New York became the first to enact the requirement a year later, enabling Conners parents to learn he had the defect days after his birth in 2017.

It was a nightmare to find out the news, but looking back, it was a true blessing, said Richelle Brooks, of Swain, N.Y., who works for a collection agency. (Her fiance, Adam Hess, Conners father, is a stay-at-home dad helping to raise him and three older siblings.)

Because of those test results, Conner had to undergo magnetic resonance imaging of his brain regularly to determine whether he might start developing the most devastating form of the disorder, as roughly a third of patients with the defective gene do. Unfortunately, a lesion that appeared on his MRI in September indicated he almost certainly would.

Theres clearly a flame thats about to start spreading like wildfire, Dr. Florian Eichler, Conners neurologist and a CALD expert at Massachusetts General Hospital, said of such lesions. Eichler helped run the trials that were led by researchers from MGH and Childrens Hospital, rival hospitals that acted as collaborators.

Skysonas arrival has elated patients families and doctors, validated decades of research by Harvard-affiliated hospitals and Bluebird, and underscored the potential for gene therapies to halt and, perhaps, even cure inherited diseases.

I hesitate to say cure because theyre still at risk for something from their disease, Dr. Christine Duncan, Conners transplant specialist at Boston Childrens, said of CALD patients. But the gene therapy appears to halt the relentless neurological decline that leads to a vegetative state or death in most patients.

Duncan helped oversee the Skysona trials and testified before a panel of advisers to the FDA that voted 15-0 last June to recommend the treatment be approved. She said she expects Conner to lead a relatively normal life, although he will still need to take a steroid to compensate for an adrenal gland deficiency related to the disease. And he may develop peripheral nerve damage as an adult that could affect his ability to walk and even necessitate a wheelchair, as well as other problems.

For more than 30 years, doctors have known that stem cells from the blood of healthy donors can halt CALD. That made those cells, which are the bodys raw materials, the only proven treatment before Skysona. Donated cells travel to the bone marrow, multiply, and drift into the brain. There they slowly turn into guard cells of the central nervous system called microglial cells. In CALD patients, those cells appear to stop the buildup of fatty acids that damage protective sheaths around nerve cells as a result of the defective gene.

But it can be hard to find a suitable donor. Stem cells work best when they come from siblings, whose genetic makeup is more likely to be compatible with that of the patient. Cells donated by someone other than a sibling often behave as though the patients own cells are a threat and attack them, a potentially life-threatening complication called graft-versus-host disease.

But because CALD runs in families, siblings are also more likely to have the same genetic defect, making them ineligible to donate the cells. Indeed, Conners 13-year-old brother has the same faulty gene, although it hasnt led to the cerebral form of the disease.

In the end, less than a quarter of patients have siblings who can become donors, said Duncan.

The researchers behind Skysona sidestepped the donor conundrum. They used stems cells removed from the blood of patients themselves, inserted a good gene into those cells in the laboratory, and infused them back into the bone marrow. The patients modified stem cells halted the disease as effectively as stem cells donated by healthy, compatible siblings and better than stem cells from unrelated donors.

Skysona doesnt come without a serious risk. Three of the 67 children who received it in the trials developed blood cancers believed to be related to the gene therapy. Although the cancers were treatable, Duncan said, the risk must be weighed by families considering the gene therapy.

As impressively as Skysona performs, critics of the pharmaceutical industry say it also illustrates an alarming trend: drug prices soaring into the stratosphere.

At $3 million, Skysona became the most expensive drug ever when the FDA approved it. That sum doesnt cover the cost to administer it or to provide related treatments at the hospital, which, in Conners case, included eight debilitating days of chemotherapy to make room in his bone marrow for his altered stem cells. Medicaid is covering his medical expenses, his mother said, because she cant afford the premiums for the health insurance offered by her employer.

Whenever a gene therapy wins approval, it seems to shatter the record set by the previous one for the most expensive drug ever. Skysonas price tag broke the record set a month earlier by another gene therapy from Bluebird called Zynteglo, which costs $2.8 million and treats a rare inherited blood disease called beta thalassemia. Barely two months after Skysona was approved, it lost its title as the priciest drug to Hemgenix, a $3.5 million gene therapy for hemophilia B, made by CSL Behring, of King of Prussia, Pa.

Of the six gene therapies cleared since 2017, the cheapest was Kymriah, a Novartis treatment for a rare form of leukemia that costs $475,000. The Alliance for Regenerative Medicine predicts another half-dozen gene therapies could be approved this year.

Dr. Ezekial Emanuel, a bioethicist and vice provost for global initiatives at the University of Pennsylvania decries such eye-popping prices. Skysona, he said, costs about 25 percent more than the lifetime median income for someone in the United States with a bachelors degree. Its definitely exorbitant, he said.

Daniel Ollendorf, a drug-pricing expert at Tufts Medical Center, said gene therapies like Skysona can save the health care system money in the long run because children with devastating disorders sometimes live for years with feeding tubes or respiratory equipment, piling up staggering medical expenses.

Nonetheless, Ollendorf, director of value measurement and global health initiatives at the Tufts Center for the Evaluation of Value and Risk in Health, said the Skysona trials were relatively small and that the gene therapys durability remains to be seen. He wishes Bluebird had offered to reimburse insurers for the cost of the treatment if it doesnt work, as the biotech did with Zynteglo, its recently approved gene therapy for beta thalassemia.

Bluebird spokeswoman Jess Rowlands said the company and insurers had little interest in arranging such agreements given CALDs rarity. Bluebird priced Skysona at $3 million, she added, because the treatment is urgently needed, will benefit patients and families, and could save money in the long run. Rowlands said the price had nothing to do with what it costs to make a medicine for a small number of patients, and declined to discuss Bluebirds profit on the gene therapy.

But Stoke Therapeutics chief executive Edward Kaye who has spent more than two decades developing drugs for rare diseases and treated boys with CALD as a pediatric neurologist said the startling prices of gene therapies undoubtedly reflect, in part, the massive costs to make medicines used by relatively few patients. I would be disingenuous to say anything else, he said.

For his part, Bluebirds chief executive, Andrew Obenshain, said Skysonas price tag should be balanced with the value that the treatment brings to patients and to the health care system. These boys lose the ability to talk, walk, go to the bathroom, and eat by themselves.

No one needs to explain that to Darcy Gray, who lives near Waterbury, Conn. Her son, Christopher, a precocious, good-humored boy, had been an excellent student, learned to ride a two-wheeler before his third birthday, and earned a blue belt in martial arts. But at the age of 7, in 2016, he began complaining of blurry vision and his hearing seemed impaired.

After a series of medical tests, his family learned that he had CALD and that the disease was too advanced for a stem cell transplant. In a matter of months, he could no longer see, hear, talk, sit up, and eat, and he needed a feeding tube.

Darcy Gray and her husband, Billy, sleep with Christopher, now 14, in the living room because they cant carry him upstairs to his bedroom. They move him from a hospital-style bed there to a recumbent couch and sometimes take him for a walk outside after strapping him into a wheelchair.

CaptionPHOTO GALLERY: Billy and Darcy Gray prayed over their dinner while their son Christopher sat nearby in his wheelchair at their home in Watertown, Conn. (Erin Clark/Globe Staff)1 of 6

Theres not a day that goes by that we dont have grief in our hearts, Darcy Gray said. I still hear my sons voice in my head.

Given such dire outcomes, Massachusetts insurers appear to be taking Skysonas cost in stride, partly because the disease is so rare.

Bluebird says that only 5 to 10 patients are expected to get the treatment this year at three US sites qualified to administer it: Boston Childrens, Childrens Hospital of Philadelphia, and the University of Minnesota Masonic Childrens Hospital.

The Massachusetts Association of Health Plans, which represents 14 insurers, recently contacted its members to see whether they will cover Skysona, and none that responded said they wouldnt, according to Lynda Jackson, a spokeswoman for the association.

Nonetheless, she said, the group continues to have concerns about the costs of pharmaceutical drugs, including these gene therapy drugs. While many of these therapies are lifesaving, they come with exorbitant price tags.

Point32Health, the states second-biggest health insurer, said it will cover Skysona, as it has other gene therapies for rare diseases. The worry, said its recently departed chief medical officer Dr. Michael Sherman, is what happens when a drug company wins approval for a gene therapy to treat a not-so-rare disorder like sickle cell disease. That blood disorder affects about 100,000 people in the United States.

As it happens, Bluebird is among several drug firms working on a gene therapy for sickle cell, and the company has a lot riding on it. The biotech has struggled financially in recent years and lost more than 97 percent of its value on the stock market since its shares peaked in 2018.

Neither Skysona nor Zynteglo will make the biotech profitable, Bluebirds chief executive, Obenshain said, but approval of a gene therapy for sickle cell which the firm hopes to launch early next year would. With all three therapies together, that is a sustainable company, he said.

People familiar with Skysonas backstory say an unsung hero is Elisa Seeger, of Brooklyn. Her son, Aidan, who loved playing soccer and chess, began having trouble seeing when he was in first grade.

After multiple doctors appointments, he underwent an MRI in 2011 that detected a lesion in his brain the telltale sign of CALD. Although Aidan already had significant brain damage, he received an unrelated donors stem cells at a Duke University hospital. But it was too late, and he died at the age of 7 the following year.

Seeger turned her grief into a crusade and began lobbying state houses to add the genetic defect to the conditions doctors screen for in newborns.

My son would be here right now if we had this information when he was born in 2004, and we didnt, said Seeger, who founded a patients advocacy group called the ALD Alliance, for adrenoleukodystrophy.

Although Massachusetts provides the screening test as part of a pilot program that began in 2018, parents have to opt in. The state Department of Public Health said that nearly 300,000 newborns were screened for the defective gene as of January and 22 were found to have it. An advisory committee to the health department, whose deliberations were delayed during the pandemic, plans to resume meeting this year and may consider making the screening automatic.

Thanks to Aidans legacy, Conners parents are optimistic that their son will be able to lead a relatively normal life.

Two weeks after the infusion, Conners white blood cell count indicated that his modified stem cells were multiplying and circulating in his body, enabling Skysona to stave off damage to his brain. He could be discharged as soon as Sunday.

A few days ago he happily played Roblox in bed and gave a thumbs up when asked about starting kindergarten in the fall. (He had to delay elementary school a year because of his illness.)

Richelle, his mother, said she had doubts about Skysona when she first heard of it. Those feelings vanished when she went to the Childrens Hospital website and read about the boy from Northern Ireland who received the gene therapy in the first trial 10 years ago. It said he plays soccer now, and featured a picture of him standing with his fists clenched and grinning.

At that moment, she said, I had no more doubts.

Jonathan Saltzman can be reached at jonathan.saltzman@globe.com.

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A young boys nightmare diagnosis, and the $3 million one-time ... - The Boston Globe

Lee C. Bollinger and the Revitalization of Columbia’s Medical Center – Columbia University

In his 2002 inaugural address, Columbia President Lee C. Bollinger made it clear that the Universitys medical center would be among his core leadership priorities. The discoveries in the combined areas of medicine and health care, biology, engineering, chemistry, physics, computers, and technologyknown today as the life sciencesare revolutionary in scale, he said. No great university can minimize the potential here for transforming our understanding of life and our capacity to preserve health. And a great university will figure out how to deal with one of the most important questions of higher education, namely how to bridge the intellectual strengths of the health sciences and professions and the fundamental science disciplines in Arts and Sciencesrepresented physically for us by the two campuses of Washington Heights and Morningside Heights.

In his first full year at Columbia, Bollinger took major steps to realize those goals, strengthening the medical centers leadership both within the institution and around the world. He worked with Mailman School Professor Wafaa El-Sadr and then-Dean Allan Rosenfeld on the establishment of ICAP, which went on to become one of the pioneering AIDS/HIV prevention, care, and research initiatives in sub-Saharan Africa. He also created auniversity-wide executive vice president for research, naming David Hirsh, the College of Physicians and Surgeons chair of biochemistry and molecular biophysics, to build an office designed to elevate faculty research though a more systemic, unified, and professionalized system for grant applications, compliance, and administration.While the office supports the academic and clinical research mission across every campus, its work has been especially important at the medical center, which typically generates at least two-thirds of Columbia's total research funding.

While Columbia University Irving Medical Center (CUIMC) had long been among the nations leading academic medical centers, it was essential for the University to address the growing financial and organizational challenges of managing the institutions resources and relationship with its clinical partner, NewYork-Presbyterian Hospital. In the spring of 2006, Bollinger named Lee Goldman of the University of California San Francisco as the executive vice president for health and biomedical sciences and dean of the Faculties of Health Sciences and Medicine. Over the years that followed, the two went on to appoint transformational deans at every CUIMC school: Linda Fried at the Mailman School, Christian Stohler at the College of Dental Medicine, and Bobbie Berkowitz and later Lorraine Frazier at the School of Nursing. Fried, for example, a distinguished geriatrician at Johns Hopkins, expanded Mailmans local and global leadership in establishing auniversity-wide, interdisciplinary aging center to explore and better understand the aging process and its societal implications. After Goldmans retirement in 2020, Bollinger asked Anil Rustgi, professor of medicine, director of the Herbert Irving Comprehensive Cancer Center, and associate dean of oncology at Vagelos College of Physicians and Surgeons, to serve as interim executive vice president and dean of the Faculties of Health Sciences and Medicine during what turned out to be a period of profound challenge and success in CUIMCs response to the COVID-19 pandemic. Last year, Bollinger appointedHarvard Medical School Professor Katrina Armstrong, chair of the Department of Medicine and physician in chief at Massachusetts General Hospital, to write the next chapter of CUIMC history as its chief executive and dean of the Vagelos College of Physicians and Surgeons.

Individually and collectively, these leaders have revitalized the medical center over the last two decades, expanding research, teaching, and clinical care; hiring a diverse range of accomplished faculty members; and deepeningpartnerships with and services for Upper Manhattan communities. They also helped transform the CUIMC campus through a massive investment in acclaimed new buildings for medical education at VP&S and the Nursing School, as well as capital renovations that enhanced some 1.8 million square feet of space for students, clinicians, researchers, and staff. Goldman oversaw the introduction of a modernized Faculty Practice Organization, the opening of a center for outpatient clinical care in Midtown Manhattan, a major expansion of ColumbiaDoctors into New Yorks Westchester County, as well as other locations across the city and region.

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Lee C. Bollinger and the Revitalization of Columbia's Medical Center - Columbia University

Gill Foundation honors researchers for excellence in cardiovascular … – UKNow

LEXINGTON, Ky. (April 3, 2023) The Gill Awards, given each year through the generous support of the Gill Foundation of Texas, honor individuals who have made significant contributions to the field of cardiovascular medicine, both through research and clinical care. The awards are based on nominations and assessments by a committee of prominent cardiovascular researchers from across the nation. The awards will be presented at the 25thannual University of Kentucky Gill Heart and Vascular Institute Cardiovascular Research Day on Oct. 20, 2023, at the Central Bank Center.

Joseph C. Wu, M.D., Ph.D.,the director of the Stanford Cardiovascular Institute, and the Simon H. Stertzer, M.D., Professor of Medicine and Radiology, will be awarded the Gill Heart and Vascular Institute Award for Outstanding Contributions to Cardiovascular Research.

Wu, who is president-elect of the American Heart Association, will be honored for his lifelong and notable achievements in research and the direct impact his work has had on cardiovascular biology, disease and the standard of cardiovascular clinical care.

Wus lab works on biological mechanisms of patient-specific and disease-specific induced pluripotent stem cells. The goals of his research are to better understand the disease and implement precision medicine for the prevention and treatment of cardiovascular patients. He has published hundreds of manuscripts and is a highly-cited researcher.

Wu will receive a plaque and a cash prize of $25,000.

Rebecca A. Haeusler, Ph.D.,an associate professor of pathology and cell biology and researcher at Columbia Universitys Naomi Berrie Diabetes Center, will be awarded the Gill Heart and Vascular Institute Early Career Gill Award.

The award is given to those who are typically within the first seven years of their faculty appointment. Awardees are recognized for innovation and creativity that has impacted cardiovascular research and/or advancements in clinical care.

The committee selected Haeusler for the award based on her research focused on lipoprotein and cholesterol metabolism and its potential link to insulin resistance and cardiovascular disease.

Haeusler will receive a plaque and a cash prize of $10,000.

The continued support of Linda and Jack Gill grants us a special opportunity to recognize two highly accomplished researchers in cardiovascular medicine, bring them to our campus and provide all the members of the University of Kentucky Cardiovascular Research Priority Area a chance to learn from the best, said Alan Daugherty, director of the Saha Cardiovascular Research Center and Gill Foundation Chair in Preventive Cardiology.

About the Gill Foundation

The Gill Foundation of Texasfocuses on providing scholarships for economically disadvantaged students; creating Centers of Excellence in technical fields (e.g., cardiovascular sciences, neurosciences and entrepreneurial programs for scientists, engineers & physicians); and creating novel courses, seminars and programs to promote interdisciplinary collaboration at universities.

Their $5 million donation in 1997 established theLinda and Jack Gill Heart and Vascular Institute(GHVI) atUK HealthCare, home to cutting-edge cardiovascular research, clinical care and education for cardiovascular professionals. GHVI focuses on advancing the knowledge of cardiovascular disease, diagnoses, clinical treatments and prevention. This donation also funded an endowed chair in preventive cardiology, five endowed professorships, endowed research and special programs and supports the annual Cardiovascular Research Day symposia.

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Gill Foundation honors researchers for excellence in cardiovascular ... - UKNow

Five health tech startups you should know – University of Wisconsin-Madison

Health-care startups with their roots at UWMadison have boosted the Wisconsin economy and helped improve patients lives. Photo: Bryce Richter

When it comes to helping Wisconsin residents and the states economy, youCant Stop a Badger. This March, see how UWMadison scientists conduct cutting-edge research that delivers tangible benefits for Wisconsinites and the world. Follow along using #CantStopABadger on social media.Your supportcan help us continue this work.

Five innovative University of WisconsinMadison startups are leveraging scientific research to provide new insights into medicine and treatments, which improves peoples lives and boosts Wisconsins economy.

Starting a research-based company is hard work and it can take years to get established. Here at UWMadison theres increasing support to help innovators on their journey from concept to commercialization, says Discovery to Product (D2P) director Andy Richards. These five startups combined their passion and persistence with D2P and other campus resources to further their ideas to benefit society.

As these early-stage companies continue to grow, theyre creating high-paying scientific and professional jobs in Wisconsin. Theyre also contributing to the states economy by attracting venture capital investment and other funding.

From new methods for drug development to personalizing tumor treatment, here are five startups changing how we experience healthcare:

The idea for Ayrflo was born out of the lab of co-founder Guelay Bilen-Rosas in the UWMadison Department of Anesthesiology. The team is developing a new way of monitoring breathing patterns in patients who are breathing spontaneously recovering from surgery or receiving ongoing sedation, both of which make them vulnerable for respiratory complications. This respiratory monitoring deviceprovides real-time breathing metricsby measuring airflow velocities across the windpipe, immediately detecting changes in breathing. Typical fingertip reading methods can have a devastating lag timein warning about the downstream onset of oxygen deprivation. Real-time monitoring provides critical extra time for medical professionals to address problems and save lives.

Founders: Guelay Bilen-Rosas, Irene Ong, Humberto RosasYear: 2020

UWMadison Connection: School of Medicine and Public Health

The AIQSolutions medical device software platform is built on technology invented at UWMadison by faculty at the Carbone Cancer Center, and the School of Medicine and Public Healths Oncology and Medical Physics departments. The company helps doctors better understand and predict a patients response to treatment for complex diseases such as cancer. The technology usesadvanced algorithms,includingartificial intelligenceto analyze radiology images to create a detailed map of how each area in the body responds to treatment. Physicians are using AIQs product to improve patient outcomes and decreasehealthcarecosts at hospitals, including UW Health.

Founders: Dona Alberti (COO), Robert Jeraj (CSO), Glenn Liu (CMO), Guy Starbuck (CTO)

Year: 2015

Employees: 28

UWMadison Connection: School of Medicine and Public Health

Su-Chun Zhang UW Health / John Maniaci

BrainXell is based on technology developed in the lab of co-founder Su-Chun Zhang, a professor of neuroscience and neurology at the Waisman Center on the UWMadison campus. The company creates drug discovery and toxicology testing tools using patient-derived or genetically modified stem cells. They provide large-scale production of specialized neural cells to the pharmaceutical and biotechnology industry. Neural cells produced with this same technology are used to treat devastating central nervous system diseases such as Parkinsons and Alzheimers as well as spinal cord injuries, through their subsidiary BrainXell Therapeutics.

Founders: Shouming Du (President & CEO), Su-Chun Zhang (Board member)

Year: 2015

Employees: 26

UWMadison Connection: Waisman Center; School of Medicine and Public Health

Mike Sussman

The core technology for Immuto Scientific was developed by co-founder Michael Sussman, UWMadison biochemistry professor, in collaboration with Leon Shohet, engineering professor emeritus. Other co-founders include former engineering postdocs and grad students Josh Blatz, Daniel Benjamin and Faraz Choudhury, as well as Dr. Sussmans lab scientist Benjamin Minkoff. The company develops solutions that accelerate the drug discovery process by automating complex protein structure analysis for protein therapeuticsa type of drug produced within living cells (such as Herceptin). These drugs are used in the treatment of difficult-to-cure diseases such as cancer. Their Plasma Induced Modification of Biomolecules technology helps companies understand how a drug attaches to its target molecule in the body.

Founders: Daniel Benjamin (CTO), Josh Blatz, Faraz A. Choudhury (President & CEO), Benjamin Minkoff, Leon Shohet, Mike Sussman (CSO)Year: 2018

Employees: 11

UWMadison Connection: College of Engineering; College of Agricultural & Life Sciences

Bryan Bednarz

Voximetry is a spin-off company out of the lab of Bryan Bednarz, associate professor of medical physics at UWMadison, who co-founded the company with two other lab members, Joseph Grudzinski and Paul Wickre. Using high-speed graphics processing, the technology helps deliver personalized radiopharmaceutical therapy (a radioactive drug)a safe, effective and targeted approach to tumor treatment. The software provides detailed information on how the drug circulates and how it interacts with both cancers and healthy tissues. This enables physicians and physicists to tailor treatment to each individual patient.

Founders: Bryan Bednarz (CSO), Joseph Grudzinski (CIO), Paul Wickre (CTO)

Year: 2016

Employees: 12

UWMadison Connection: School of Medicine and Public Health

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Five health tech startups you should know - University of Wisconsin-Madison

Annals of Rheumatic Diseases Publishes Results from Phase 2 Study of emapalumab in Patients with Secondary HLH/Macrophage Activation Syndrome – Yahoo…

Sobi, Inc.

Results showed that MAS remission was seen in 13 of 14 patients with sHLH/MAS receiving emapalumab who had an inadequate response to high-dose glucocorticosteroids

WALTHAM, Mass., April 04, 2023 (GLOBE NEWSWIRE) -- Sobi North America, the North American affiliate of Swedish Orphan Biovitrum AB (Sobi) (STO:SOBI), today announced that the Annals of Rheumatic Diseases has published results from an open-label, single-arm, multicenter phase 2 study evaluating the safety and efficacy of emapalumab, an anti-interferon-gamma monoclonal antibody, being investigated in patients with Systemic Juvenile Idiopathic Arthritis (sJIA) or Adult-onset Still's Disease (AOSD) who developed secondary hemophagocytic lymphohistiocytosis (sHLH)/Macrophage Activation Syndrome (MAS) following an inadequate response to high-dose glucocorticosteroids.

In the published study, 14 patients with sJIA or AOSD and sHLH/MAS who did not respond to high-dose glucocorticosteroids received emapalumab. MAS remission was seen in 13 of the 14 patients by week 8, at a median time of 25 days, based on clinical and laboratory criteria. All 14 patients completed the trial, entered long-term follow-up and were alive at the end of follow-up. Based on the results of this study, Sobi decided to continue to evaluate emapalumab in this patient population and initiated the EMERALD phase 3 study, which is ongoing.

"I believe that the results of this study represent an important milestone for the identification of a potential novel targeted treatment for patients with sJIA or AOSD with sHLH/MAS who have failed high-dose glucocorticoids," said Dr. Fabrizio De Benedetti, Head of the Division of Pediatric Rheumatology and Head of the Laboratory of ImmunoRheumatology at Bambino Ges Children's Hospital in Rome, Italy. "We are eager to collect more data so that emapalumab becomes part of the therapeutic armamentariumfor this rare disease and its high unmet medical need."

sHLH/MAS is a rare, life-threatening condition characterized by uncontrolled hyperinflammation which may develop on a background of rheumatologic diseases such as sJIA. It is classified as a secondary form of HLH and is caused by excessive activation and expansion of T cells and macrophages. A body of scientific evidence has been accumulated suggesting interferon gamma (IFN) is a major driver of hyperinflammation in diseases such as sHLH/MAS.

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The results published in the Annals of Rheumatic Diseases are encouraging for patients with sJIA or AOSD developing sHLH/MAS, who have had an inadequate response to high dose glucocorticoid treatment, said Dr. Alexei Grom, Research Director, Division of Rheumatology and Professor of Pediatrics at Cincinnati Children's Hospital Medical Center, in Cincinnati, Ohio. The ability of emapalumab to achieve MAS remission is meaningful as there is a high unmet need for these patients.

We are pleased that this newly published clinical data shows the potential benefit of emapalumab in patients with secondary HLH, said Tony Hoos, Head of Research & Development and Medical Affairs, Chief Medical Officer at Sobi. We remain committed to evaluating emapalumab as a potential new treatment option for patients affected by this severe condition. If our ongoing EMERALD phase 3 study in sHLH/MAS confirms the benefit, we are intending to file a supplemental Biologics License Application.

Emapalumab is marketed in the United States as Gamifant and is indicated for the treatment of adult and pediatric (newborn and older) patients with primary hemophagocytic lymphohistiocytosis (HLH) with refractory, recurrent or progressive disease or intolerance with conventional HLH therapy.Gamifant has not been reviewed or approved by the U.S. Food and Drug Administration for sHLH/MAS.

ContactsFor details on how to contact the Sobi Investor Relations Team, please click here. For Sobi Media contacts, click here.

About macrophage activation syndrome (MAS) Macrophage activation syndrome (MAS) is a severe complication of rheumatic diseases, most frequently systemic juvenile idiopathic arthritis (sJIA) a rare systemic disorder of auto-inflammatory nature with common clinical manifestations such as daily spiking fever, typical transient cutaneous rash, arthritis, lymphadenopathy, hepatosplenomegaly and serositis. MAS is characterized by fever, hepatosplenomegaly, liver dysfunction, cytopenias, coagulation abnormalities and hyperferritinaemia, possibly progressing to multiple organ failure and death. MAS is classified as a secondary form of haemophagocytic lymphohistiocytosis (HLH).

About emapalumab-lzsgEmapalumab-lzsg is a fully human, anti-IFN monoclonal antibody that binds free and receptor-bound IFN, neutralizing its biological activity. In the US, emapalumab-lzsg is indicated for pediatric (newborn and older) and adult primary hemophagocytic lymphohistiocytosis (HLH) patients with refractory, recurrent or progressive disease, or intolerance to conventional HLH therapy. Emapalumab-lzsg is the first and only medicine approved in the US for primary HLH, a rare syndrome of hyperinflammation that usually occurs within the first year of life and can rapidly become fatal unless diagnosed and treated. The FDA approval in 2018 was based on data from the phase 2/3 studies (NCT01818492 and NCT02069899). Emapalumab is indicated for administration through intravenous infusion over one hour twice per week until hematopoietic stem cell transplantation (HSCT). The efficacy and safety of emapalumab are currently being evaluated in a phase 3 study in patients with MAS in Stills disease or systemic lupus erythematosus (SLE) (EMERALD; NCT05001737).

U.S. Indication for Gamifant (emapalumab-lzsg)Gamifant (emapalumab-lzsg) is an interferon gamma (IFN)blocking antibody indicated for the treatment of adult and pediatric (newborn and older) patients with primary hemophagocytic lymphohistiocytosis (HLH) with refractory, recurrent, or progressive disease or intolerance with conventional HLH therapy.

Important Safety InformationInfectionsBefore initiating Gamifant, patients should be evaluated for infection, including latent tuberculosis (TB). Prophylaxis for TB should be administered to patients who are at risk for TB or known to have a positive purified protein derivative (PPD) test result or positive IFN release assay. During Gamifant treatment, patients should be monitored for TB, adenovirus, Epstein-Barr virus (EBV), and cytomegalovirus (CMV) every 2 weeks and as clinically indicated. Patients should be administered prophylaxis for herpes zoster, Pneumocystis jirovecii, and fungal infections prior to Gamifant administration.

Increased Risk of Infection with Use of Live VaccinesDo not administer live or live attenuated vaccines to patients receiving Gamifant and for at least 4 weeks after the last dose of Gamifant. The safety of immunization with live vaccines during or following Gamifant therapy has not been studied.

Infusion-Related ReactionsInfusion-related reactions, including drug eruption, pyrexia, rash, erythema, and hyperhidrosis, were reported with Gamifant treatment in 27% of patients. In one-third of these patients, the infusion-related reaction occurred during the first infusion.

Adverse ReactionsIn the pivotal trial, the most commonly reported adverse reactions (10%) for Gamifant included infection (56%), hypertension (41%), infusion-related reactions (27%), pyrexia (24%), hypokalemia (15%), constipation (15%), rash (12%), abdominal pain (12%), CMV infection (12%), diarrhea (12%), lymphocytosis (12%), cough (12%), irritability (12%), tachycardia (12%), and tachypnea (12%). Additional selected adverse reactions (all grades) that were reported in less than 10% of patients treated with Gamifant included vomiting, acute kidney injury, asthenia, bradycardia, dyspnea, gastrointestinal hemorrhage, epistaxis, and peripheral edema.

Please see full US prescribing information for Gamifant.

Sobi North AmericaAs the North American affiliate of international biopharmaceutical company Sobi, the Sobi North America team is committed to Sobis vision of providing access to innovative treatments that make a significant difference in the lives of individuals with rare diseases. Our product portfolio includes multiple approved treatments focused on immunology, hematology and specialty care. With U.S. headquarters in the Boston area, Canadian headquarters in the Toronto area, and field sales, medical and market access representatives spanning North America, our growing team has a proven track record of commercial excellence. More information is available atwww.sobi-northamerica.comor atwww.sobi.com.

SobiSobi is a specialized international biopharmaceutical company transforming the lives of people with rare and debilitating diseases. Providing reliable access to innovative medicines in the areas of hematology, immunology and specialty care, Sobi has approximately 1,600 employees across Europe, North America, the Middle East, Asia and Australia. In 2022, revenue amounted to SEK 18.8 billion. Sobis share (STO:SOBI) is listed on Nasdaq Stockholm. More about Sobi atsobi.com, LinkedIn and YouTube.

Media Contact:matt.tooth@sobi.com

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Annals of Rheumatic Diseases Publishes Results from Phase 2 Study of emapalumab in Patients with Secondary HLH/Macrophage Activation Syndrome - Yahoo...

Direct Biologics Announces FDA Authorization to Expand Ongoing Phase 3 Clinical Study of ExoFlo to All-Cause Moderate-to-Severe ARDS – Yahoo Finance

Ongoing pivotal Phase 3 EXTINGUISH ARDS trial expands to enroll hospitalized patients with moderate-to-severe ARDS, regardless of underlying etiology

ExoFlo is the first cell or cell-derived therapeutic candidate to be evaluated in a Phase 3 trial for all-cause moderate-to-severe ARDS

AUSTIN, Texas, April 04, 2023--(BUSINESS WIRE)--Direct Biologics, LLC, a late-stage biotechnology company leveraging its regenerative medicine platform using extracellular vesicles (EVs) secreted from bone marrow-derived mesenchymal stem cells to address multiple disease indications, announces that the U.S. Food and Drug Administration (FDA) has authorized the expansion of its pivotal Phase 3 EXTINGUISH ARDS trial to evaluate the safety and efficacy of ExoFlo in the treatment of moderate-to-severe acute respiratory distress syndrome (ARDS) from any underlying etiology.

"ARDS is a respiratory disease characterized by a rapid onset of inflammation and fluid in the lungs with unacceptably high mortality and unsustainable treatment costs," said Mark Adams, Chief Executive Officer of Direct Biologics. "We look forward to the results of our Phase 3 study given the significant survival benefit observed in our Phase 2 trial and the absence of any FDA-approved biologic for the treatment of moderate-to-severe ARDS."

The global multicenter randomized, double-blinded, placebo-controlled pivotal Phase 3 EXTINGUISH ARDS trial (NCT05354141) is designed to evaluate the safety and efficacy of ExoFlo for the treatment of all-cause moderate-to-severe ARDS. The trial is expected to enroll 320 patients ages 18-65. The trial will have two treatment arms with half of the enrolled patients receiving a placebo and half receiving up to three intravenous doses of 15 mL of ExoFlo. All patients in both arms will receive standard of care.

The primary endpoint of 60-day all-cause mortality was selected based on the significant survival benefit observed in the completed randomized Phase 2 clinical trial of ExoFlo. Secondary endpoints include ventilator-free days, oxygen-free days and ICU-free days, along with additional exploratory endpoints. In addition, the trial will evaluate the efficacy of ExoFlo in ARDS subtypes to better understand the disease process. In March 2022, ExoFlo received Regenerative Medicine Advanced Therapy, or RMAT, designation by the FDA, which provides opportunities to expedite ExoFlos clinical development for ARDS.

Story continues

"FDA authorization to expand our ongoing Phase 3 clinical trial to all-cause ARDS marks a defining moment for regenerative medicine. ExoFlo, designed to repair lung tissue while being a potent anti-inflammatory and immunomodulatory agent, is the first cell or cell-derived therapeutic candidate to be evaluated in a Phase 3 trial for all-cause moderate-to-severe ARDS. In fact, ExoFlo is one of a small handful of biologics that has demonstrated adequate tolerability and clinical activity to gain allowance by the FDA for Phase 3 evaluation in moderate-to-severe ARDS," said Amy Lightner, M.D., Chief Medical Officer of Direct Biologics.

About ARDS

Acute respiratory distress syndrome (ARDS) is a life-threatening condition characterized by acute and diffuse inflammatory lung injury resulting in increased fluid in the lungs, loss of ability to oxygenate and decreased lung compliance. Currently, 15% of all ICU patients and 23% of all ventilated patients in the United States are ARDS patients, which results in an annual intensive care expenditure exceeding $80 billion. The mortality rate of moderate-to-severe ARDS remains unacceptably high at 50%, despite improved ventilatory strategies such as protective ventilation and prone positioning. After decades of research, there is still no targeted or individualized therapy for the treatment of ARDS in the United States. The standard of care is still centered around optimizing mechanical ventilation and supportive care strategies without known mortality benefit.

About Direct Biologics

Direct Biologics is a late-stage biotechnology company leveraging a regenerative medicine platform which uses extracellular vesicles (EVs) secreted from mesenchymal stem cells to address multiple disease indications. Direct Biologics mission is to be the global leader in regenerative medicine through discovery, innovation, advancement of science, and treatment of patients in a safe and effective manner. Our therapeutic product candidate, ExoFlo, is designed to be a scalable, reproducible, and effective next-generation biologic that leverages our proprietary EV platform technology designed to reduce inflammation, modulate the immune system, and restore tissue through cellular regeneration. Direct Biologics is currently conducting the global Phase 3 EXTINGUISH ARDS clinical trial of ExoFlo for the treatment of hospitalized adults with moderate-to-severe acute respiratory distress syndrome (ARDS). In addition, the Company has initiated Phase 1 clinical trials with ExoFlo for the treatment of ulcerative colitis and Crohns disease, and expanded access trials in solid abdominal organ transplantation and severe ARDS patients. Direct Biologics intends to pursue additional clinical applications including perianal fistulizing Crohns disease and necrotizing enterocolitis. Headquartered in Austin, Texas, Direct Biologics also has an R&D facility at the Center for Novel Therapeutics on the campus of University of California at San Diego, and operations and order-fulfillment center in San Antonio, Texas. For more information, please visit http://www.directbiologics.com and follow us on Twitter @directbiologics.

View source version on businesswire.com: https://www.businesswire.com/news/home/20230404005769/en/

Contacts

Direct Biologics 800-791-1021info@directbiologics.com Twitter: @directbiologics

Investor Relations LHA Investor RelationsYvonne Briggs310-691-7100YBriggs@lhai.com

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Direct Biologics Announces FDA Authorization to Expand Ongoing Phase 3 Clinical Study of ExoFlo to All-Cause Moderate-to-Severe ARDS - Yahoo Finance

Multiple Myeloma in the Black Community – Everyday Health

For many Black Americans with multiple myeloma, socioeconomic status can stand in the way of even standard care, especially for those who live in rural areas, according to Calloway-Campbell.

Youre not close to an academic center, she says. And local doctors may not have even heard of multiple myeloma. Getting to a doctor can be near-impossible for people who dont have a car or other means of transportation, as well, and even gathering information can be challenging without easy access to Wi-Fi.

Treatment for myeloma is exorbitantly expensive too. According to a study published in JAMA Open Network in July 2021, the average lifetime cost of treating multiple myeloma was upwards of $185,000 an intimidating price tag for most people, much less for those who may be unemployed or underpaid, lacking health insurance, or inadequately covered.

The National Cancer Institute (NCI)reports that while 67 percent of white Americans under 65 and 44 percent over 65 have private health insurance, only 51 percent of African Americans under 65 and 28 percent over 65 are insured. And private health insurance is key to quality myeloma care.

According to the NCI, for instance, non-white recipients of Medicaid or Medicare are less likely to be prescribed new drugs or treated with stem cell transplants compared to non-white patients who have private insurance. This is significant, given a quarter of Medicare and Medicare Advantage recipients are African American.

Financial assistance for myeloma treatment is available though. On the IMF websites Financial Assistance page, for instance, are a list of options ranging from small stipends, which can be put directly toward treatment, to sources of help for non-medical expenses, such as transportation and childcare. The organization also has a page with information about drug reimbursement and help with copays.

Other sources for financial aid for people of all ethnicities living with multiple myeloma include:

Originally posted here:
Multiple Myeloma in the Black Community - Everyday Health

Diamyd Medical partners with JDRF to advance the DIAGNODE-3 … – PR Newswire

STOCKHOLM, April 4, 2023 /PRNewswire/ -- Diamyd Medical and JDRF, the leading global type 1 diabetes research and advocacy organization, have entered into a four-year research and development collaboration including a non-dilutive $5 million award to Diamyd Medical to support its ongoing Phase 3 trial with the precision medicine antigen-specific immunotherapy Diamyd. The grant will be funded under JDRF's Industry Discovery & Development Partnerships program that focuses on commercialization of therapeutics and devices for the treatment, cure, and prevention of type 1 diabetes and its complications.

"We could not have a better partner than JDRF as we are focusing on rapid advancement of our antigen-specific immunotherapy towards the market," said Ulf Hannelius, CEO of Diamyd Medical. "We expect this collaboration to significantly boost patient recruitment to this international study as well as our commercial preparations."

"JDRF is committed to supporting and advancing disease modifying therapies to delay and reverse type 1 diabetes," said Sanjoy Dutta, Ph.D., chief scientific officer at JDRF. "We are excited about Diamyd Medical's groundbreaking Phase 3 trial and its potential advancements in the preservation of insulin production for people recently diagnosed with type 1 diabetes."

"JDRF has played a crucial role in many of the most novel therapeutics and devices that have been approved in the field for those with type 1 diabetes," said Mark Atkinson, Ph.D., director of the Diabetes Institute at the University of Florida and Diamyd Medical Board Member. "JDRF's commitment to this Phase 3 program is a validation of the scientific and clinical value of the antigen-specific immunotherapy Diamyd. It also emphasizes the importance of making disease modifying therapies available to everyone affected by this disease."

About Diamyd MedicalDiamyd Medical develops precision medicine therapies for Type 1 Diabetes. Diamyd is an antigen-specific immunotherapy for the preservation of endogenous insulin production. DIAGNODE-3, a confirmatory Phase III trial is actively recruting patients with recent-onset Type 1 Diabetes in eight European countries and is being preparedto start recruiting patients in the US this summer. Significant results have previously been shown in a large genetically predefined patient group in a large-scale meta-analysis as well as in the Company's European Phase IIb trial DIAGNODE-2, where the Diamyd was administered directly into a lymph node in children and young adults with recently diagnosed Type 1 Diabetes. A biomanufacturing facility is being set up in Ume for the manufacture of recombinant GAD65, the active ingredient in the antigen-specific immunotherapy Diamyd. Diamyd Medical also develops the GABA-based investigational drug Remygen as a therapy for regeneration of endogenous insulin production and to improve hormonal response to hypoglycaemia. An investigator-initiated Remygen trial in individuals living with Type 1 Diabetes for more than five years is ongoing at Uppsala University Hospital. Diamyd Medical is one of the major shareholders in the stem cell company NextCell Pharma AB as well as in the artificial intelligence company MainlyAI AB.

Diamyd Medical's B-share is traded on Nasdaq First North Growth Market under the ticker DMYD B. FNCA Sweden AB is the Company's Certified Adviser.

About JDRF JDRF's mission is to accelerate life-changing breakthroughs to cure, prevent and treat T1D and its complications. To accomplish this, JDRF has invested more than $2.5 billion in research funding since our inception. We are an organization built on a grassroots model of people connecting in their local communities, collaborating regionally and globally for efficiency and broader fundraising impact, and uniting on a global stage to pool resources, passion, and energy. We collaborate with academic institutions, policymakers, and corporate and industry partners to develop and deliver a pipeline of innovative therapies to people living with T1D. Our staff and volunteers throughout the United States and our five international affiliates are dedicated to advocacy, community engagement, and our vision of a world without T1D. For more information, please visit jdrf.org or follow us on Twitter (@JDRF), Facebook (@myjdrf), and Instagram (@jdrfhq).

About Type 1 Diabetes Type 1 diabetes is an autoimmune condition that causes the pancreas to make very little insulin or none at all. This leads to dependence on insulin therapy and the risk of short or long-term complications, which can include highs and lows in blood sugar; damage to the kidneys, eyes, nerves, and heart; and even death if left untreated. Globally, it impacts nearly 9 million people. Many believe T1D is only diagnosed in childhood and adolescence, but diagnosis in adulthood is common and accounts for nearly 50% of all T1D diagnoses. The onset of T1D has nothing to do with diet or lifestyle. While its causes are not yet entirely understood, scientists believe that both genetic factors and environmental triggers are involved. There is currently no cure for T1D.

For further information, please contact:Ulf Hannelius, President and CEOPhone: +46 736 35 42 41E-mail: [emailprotected]

The following files are available for download:

SOURCE Diamyd Medical AB

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Diamyd Medical partners with JDRF to advance the DIAGNODE-3 ... - PR Newswire

Rejuvenation Roundup March 2023 – Lifespan.io News

March marks the beginning of spring and the time for renewal, and this month, labs around the world have looked into renewing older concepts of aging and discovered potential methods for renewing our bodies.

Partnership Opportunities for the 5th Annual Age-Related Disease Therapeutics Summit: This conference is seeking partners who want to showcase their companies to biotechnology and investment leaders, listing contract research, biomarkers of aging, epigenetics, and preclinical services as the preferred areas of interest.

Thank you to OneSkin, one of our philanthropic partners who has generously agreed to donate a percentage of their monthly revenue to Lifespan.io, to support our mission of longevity biotechnology advocacy and education.

OneSkin is a longevity company developing products designed to extend skin and body health by targeting aging at its source.

Team and activities

Rep. Bilirakis on the Longevity Science Caucus: We in the longevity field have received powerful allies on Capitol Hill with the creation of the bipartisan Congressional Caucus for Longevity Science. We had the opportunity to ask questions of one of its co-chairs.

Lifespan News

Human Brain Organoids in Rats: Emmett Short talks about how human neurons in rat brains can actually fulfill critical functions in these animals.

NAD+, Fat, and Muscle: On this episode, Emmett Short goes over a new study showing that nicotinamide riboside (NR) increases both fat and muscle.

Best Time to Exercise: This episode features some rather surprising results gleaned from examining the effects of regular morning exercise on UK Biobank participants.

Protein and Muscle: This Lifespan News is on a study showing that diets with less protein are connected to better muscle maintenance with aging.

Exercise and Supplements: Emmett Short talks about a recent meta-study of exercise and supplements, which showed inconclusive results despite its large dataset.

Young Blood for Brain Boosting: This episode discusses a study showing that the brains of old mice benefit by receiving the blood of young mice.

Prof. Tzipi Strauss on the Upcoming Longevity Center: In Sheba Medical Center in Israel, the first-of-its-kind Longevity Center will soon open its doors. We spoke with its future director, Prof. Tzipi Strauss, who is also leading the Department of Neonatology at Sheba.

Prof. George Church on Cellular Reprogramming and Longevity: Professor of Genetics at Harvard Medical School, a veteran geroscientist, and a serial entrepreneur, George Church hardly needs an introduction. While we are always happy to discuss the present and future of geroscience with him, this interview focuses on the two gene therapy papers that he recently co-authored.

Ashley Zehnder on Harnessing Animal Genes Against Aging: Many species have developed amazing mechanisms to cope with various drivers of aging. A handful of bold entrepreneurs are trying to go commercial, and one of them is Ashley Zehnder, DVM, PhD, co-founder and CEO of Fauna Bio, a biotech startup that looks for protective genotypes in animals in order to weaponize them against human diseases.

Ryan OShea of Future Grind hosts this months podcast, showcasing the events and research discussed here.

Human Fasting Modulates Macrophage Function: This month, Dr. Oliver Medvedik explored a recent study that looked at the effects of prolonged fasting on human macrophages and how metabolites from that fasting increased median lifespan in Caenorhabditis elegans.

Insider Insight: Meet the Organizers of LongHack: LongHack, the longevity hackathon hosted by DeSci organization VitaDAO, was held on January 20th-23rd, 2023. Gathering researchers, developers, and other interested parties to create new tools and solutions for longevity, ten teams competed to impress the judging panel and take home prizes.

A Lasting Rise in Investment in the Longevity Sector: 2021 marked one of the biggest years in longevity financing, with 2022 following closely behind. Over the past ten years, the industry has grown in financing from $500 million in 2013 to a peak of $6.2 billion in 2021.

Dr. Nir Barzilai on How to Age Later: In Age Later: Healthspan, Lifespan, and the New Science of Longevity, Dr. Nir Barzilai provides an insightful and comprehensive overview of the latest research on aging and longevity.

Research Roundup

Association Between BMI and Mortality Revisited: Ryan K. Masters, professor at CU Boulder, suggests that when adjusted for body shape and lifelong shifts, the relationship between BMI and mortality is more linear and robust than previously thought, with normal BMI being the healthiest.

Exercise and Supplements Against Age-Related Inflammation: In a new systematic review, researchers have shown that combining some dietary supplements and exercise might be beneficial for people over the age of 60.

How NAD+ Relates to Smell Loss with Age: Researchers publishing in Aging Cell have elucidated a relationship between aging, the loss of smell, and NAD+ in a mouse model. Roughly half of people over the age of 65 experience a decreased ability to smell, and research has shown that it is an early biomarker for neurodegenerative diseases.

Extracellular Vesicles as a Hallmark of Aging: A review paper published in Cells has described multiple ways in which the secretion of extracellular vesicles changes with aging, leading the authors to propose it as its own hallmark.

New Small Molecule Alleviates Alzheimers in Mouse Model: Scientists have developed a custom-made molecule that targets a post-translationally modified kinase linked to Alzheimers, improving symptoms in a murine model of the disease.

Metformin and Rapamycin Rejuvenate Stem Cells in Mice: In a new study published in Aging Cell, researchers have shown that two promising anti-aging agents, the antibiotic rapamycin and the anti-diabetic drug metformin, reverse aging in a population of intestinal stem cells.

Young Blood Alters Gene Expression in Old Brain Cells: Research published in Nature Aging has shown that heterochronic parabiosis, the circulatory joining of young and old organisms, has rejuvenative effects on the gene expression of multiple types of brain cells in mice.

New Treatment Alleviates Depression Symptoms in Mice: Scientists have shown that the protein GDF11 can reverse depression-like symptoms in naturally aged mice and in a mouse model of depression.

Mitochondria, DNA, and Oxidative Stress: A paper published in Experimental Gerontology has provided a fresh and detailed look at the effects of oxidative stress on longevity. The free radical theory of aging, which purports that reactive oxygen species (ROS) are the core driver of aging, was developed all the way back in 1956.

Epigenetic Biomarker for Measuring Aging Through Fitness: A new biomarker for measuring biological aging based on physical fitness has been published in Aging, and it has been found to be useful in predicting health issues.

Excessive Fat, Not Sugar, Leads to Obesity in Mice: In a new study published in Endocrinology and Metabolism, researchers have shown that fat rather than sugar is the macronutrient that drives obesity and other detrimental metabolic changes if it constitutes a large proportion of dietary calories.

Inflammation Strongly Predicts Mortality After Statin Use: Analyzing data from three clinical trials, scientists have shown that excessive inflammation is a much stronger predictor of cardiovascular and all-cause mortality in patients on statins than excessive LDL cholesterol levels.

Sleep Apnea and Inflammatory Biomarkers of Tooth Decay: A study published in Heliyon has explained a relationship between sleep apnea and an increase of inflammatory factors in the mouth that are connected to the gum disease peridontitis.

The Human Cost of Metabolic Diseases: A new paper published in Cell Metabolism has shown the growing influence of metabolic diseases in an aging population. Metabolic diseases include hypertension, type 2 diabetes, hyperlipidemia, obesity, and non-alcoholic fatty liver disease.

Mediterranean Diet Might Lower Risk of Dementia: In a large-scale observational study, British scientists have shown that high levels of adherence to the Mediterranean diet might substantially lower the risk of dementia. The Mediterranean diet, is based on ingredients such as olive oil, vegetables, legumes, fish, and whole grains.

Vital Muscle Enzyme Declines With Aging: Research published in Nature Metabolism has described PCYT2, an enzyme essential for muscle function, and how it declines with aging. A necessary lipid synthesizer in muscle Human beings need PCYT2 as part of the Kennedy pathway to synthesize two critical components of the phosopholipid membrane that surrounds cells.

Long-Term Resistance Exercise Increases Autophagy: In a new systemic review published in Autophagy Reports, researchers have demonstrated that exercise plays a role in regulating autophagy, depending on its type. Autophagy is the way cells break down misbehaving or nonfunctional organelles and proteins in the cell.

Promoting Muscle Regeneration With an Immune Factor: A paper published today in Nature Aging has shown that a macrophage-regulating factor has a significant impact on muscle regeneration.

Vesicles from Senescent Cells Encourage Young Stem Cells: Scientists have shown that extracellular vesicles derived from senescent stem cells can improve the proliferation, viability, and migration capacity of healthy stem cells.

Senescent Cells and Loose Teeth: A team of researchers has recently published a study on the effects of senescence on periodontal tissues, which hold teeth to bone, in Aging.

Inhibiting DREAM for Enhanced DNA Damage Repair: In a new study published in Nature Structural and Molecular Biology, researchers have demonstrated that by manipulating the DREAM protein complex, a major regulator of DNA damage response, it might be possible to alter the number of DNA mutations accumulated with age.

Using AI to Measure Age Through the Eyes: An accepted manuscript in eLife Sciences has described eyeAge, a new clock that uses deep learning to analyze the eye in detail in order to predict chronological age and age acceleration.

Daily Step Count, Less Mortality, Diminishing Returns: A new study using wearable accelerometers suggests that you dont have to clock extreme numbers of steps every day to stay healthy.

Associations of sleeping, sedentary and physical activity with phenotypic age acceleration: Sedentary behavior was positively associated with aging. Replacing sedentary behaviors with walking/bicycling or moderate to vigorous physical activity was adversely associated with aging among adults.

Coenzyme Q10 supplementation improves the motor function of middle-aged mice by restoring the neuronal activity of the motor cortex: This study shows that CoQ10 improves brain mitochondrial function and physical performance in mice.

Increased SIRT1 Concentration Following Four Years of Selenium and Q10 Intervention Associated with Reduced Cardiovascular Mortality at 10-Year Follow-Up: This study suggests that this combination increases SIRT1 in a way that helps to prevent vascular aging.

Dietary magnesium intake is related to larger brain volumes and lower white matter lesions with notable sex differences: Higher dietary magnesium intake has been found to be associated with better brain health in the general population, particularly women.

Potential reversal of biological age in women following an 8-week methylation-supportive diet and lifestyle program: There was a statistically significant (p=.039) difference in the participants mean biological age before (55.83 years) and after (51.23 years) the 8-week diet and lifestyle intervention, with an average decrease of 4.60 years.

Six-Month Synbio Administration Affects Nutritional and Inflammatory Parameters of Older Adults: The PROBIOSENIOR project demonstrated how SYNBIO supplementation may positively influence some nutritional and inflammatory parameters in the elderly.

Mendelian randomization analyses reveal causal relationships between the human microbiome and longevity: These findings strongly implicate that these commensal microbes play a role in human longevity and suggest that they should be monitored to promote longevity.

AAV1.NT-3 gene therapy prevents age-related sarcopenia: The researchers reported functional, in vivo muscle physiology improvements

Transcriptional activation of endogenous Oct4 via the CRISPR/dCas9 activator ameliorates Hutchinson-Gilford progeria syndrome in mice: These results suggest that partial rejuvenation by activating this Yamanaka factor can be used as a novel strategy in treating geriatric diseases.

Ageing as a software design flaw: Well-known aging researcher Joo Pedro de Magalhes expands on the idea that aging is the result of misguided genetic programming rather than entropic damage.

Necroptosis inhibition counteracts neurodegeneration, memory decline, and key hallmarks of aging, promoting brain rejuvenation: These results demonstrate that necroptosis, which mediates degeneration of injured axons, contributes to age-dependent brain degeneration.

Effect of peripheral cellular senescence on brain aging and cognitive decline: Preserved cognition was associated with the removal of peripheral senescent cells, which decreased the systemic inflammation that normally drives neuroinflammation, BBB breakdown, and impaired synaptic function.

Immunotherapeutic approach to reduce senescent cells and alleviate senescence-associated secretory phenotype in mice: These results show that HCW9218 represents a novel immunotherapeutic approach and a clinically promising new class of senotherapeutic agents targeting cellular senescence-associated diseases.

The Fifth Annual Age-Related Disease Therapeutics Summit: The Age-Related Disease Therapeutics Summit will once again be held in San Francisco this year, and Lifespan.io readers are encouraged to use the code 32026Lifespan when signing up.

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Rejuvenation Roundup March 2023 - Lifespan.io News