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Cord Blood Banks Sold Families on False Hope – myheraldreview.com

Posted: July 21, 2024 at 2:34 am

Millions of pregnant women get the pitch through their OB-GYN: Put a bit of your newborns umbilical cord on ice, as a biological insurance policy. If your child one day faces cancer, diabetes or even autism, the precious stem cells in the cord blood could become a tailor-made cure.

Many families are happy to pay for the assurance of a healthy future. More than 2 million umbilical cord samples sit in a handful of suburban warehouses across the country. Its a lucrative business, with companies charging several thousand dollars upfront plus hundreds more every year thereafter. The industry has grown rapidly, bolstered by investments from medical device companies, hospital partnerships and endorsements from celebrities such as Drew Barrymore and Chrissy Teigen.

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Cord Blood Registry, in Tucson, Ariz., on June 26, 2024. The company stores more than one million samples, double the number it had in 2014. (Rebecca Noble/The New York Times)

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Renee Johnson in Scottsdale, Ariz., on July 7, 2024. Johnson banked her sons cord blood with ViaCord in 2014 and learned years later the cells were infected with E. coli. (Rebecca Noble/The New York Times)

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Anna Lazos at her home in Egg Harbor Township, N.J., on June 27, 2024. After spending thousands of dollars on cord blood storage, Lazos asked to withdraw a sample to enroll her son in an autism clinical trial. The company told her that the cells were contaminated with E. coli. (Hannah Yoon/The New York Times)

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Jenna Edwards with her son at home in Parkland, Fla., on June 24, 2024. When Edwards tried in 2017 to withdraw her sons cord blood cells for a clinical trial to treat his cerebral palsy, she learned that the company had found bacteria in the sample, but still charged her for the next two years. (Eva Marie Uzcategui/The New York Times)

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Cord Blood Banks Sold Families on False Hope - myheraldreview.com

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A Seventh Person Has Most Likely Been Cured of HIV Following Stem Cell Transplant – Them

Posted: July 21, 2024 at 2:34 am

A German man is reportedly the seventh person in history to be cured of HIV, according to researchers who will present the information at the 2024 International AIDS Conference in Munich, Germany.

Multiple tests given to the German man were unable to detect any copies of the virus in his body following a stem cell transplant for acute myeloid leukemia that occurred in 2015, according to a report in NBC News. The man stopped taking antiretroviral medication for his HIV in 2018 and remains in remission, or a state of having no virus present in the body.

The man has chosen to remain anonymous, but did issue a statement about his medical condition. A healthy person has many wishes, he said, a sick person only one.

The longer we see these HIV remissions without any HIV therapy, the more confidence we can get that were probably seeing a case where we really have eradicated all competent HIV, Dr. Christian Gaebler, a physician and scientist at the Charit-Universittsmedizin Berlin, told NBC News.

While any cure news is always welcomed both in the HIV community and to those outside it, the German mans case is emblematic of a trend that is proving hard to replicate for the 39 million people living with HIV worldwide. In each of the seven cases, according to NBC News, the cure came as the result of stem cell treatments for people living with HIV who had developed blood cancer. Such stem cell transplants are toxic and can be fatal, as NBC News, making it unethical as a treatment for the overwhelming majority of people with HIV.

When HIV enters a persons body, it functionally turns a persons immune cells into factories that make more and more copies of the virus. However, existing alongside these copies of the virus that actively attack a persons immune system is the latent HIV reservoir, a group of immune cells that have been overtaken by HIV but are not currently reproducing new copies of the virus. These dormant cells can awaken at any time, and targeting these cells is a major problem for HIV cure researchers. Often, these reservoirs are located in hard-to-reach areas, including the lymph nodes, the gut, and the brain, according to The Body, an HIV/AIDS news and information site.

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Cell BioEngines Secures an Additional $2 Million in Funding – GlobeNewswire

Posted: July 21, 2024 at 2:34 am

NEW YORK, July 20, 2024 (GLOBE NEWSWIRE) -- Cell BioEngines, Inc., a New York, USA-based company researching stem cells in order to develop new cell therapies unlocked $1.75 million additional funds from SOSV and the Partnership Fund through the new therapeutics seed track, available for therapeutics start-ups. In addition, Cell BioEngines raised $0.25 million from Empire State Developments NY Ventures, the states venture capital arm through the Pre-Seed and Seed Matching Fund Program.

This reflects strong growth and investor confidence in our innovative approach to advancing cell therapies, particularly timely as we expand within New York State, said Dr. Ajay Vishwakarma, Founder and CEO.

The funds will support our first multicenter clinical trial, aimed at hematological cancer patients unable to find a donor and seeking an alternative to HLA-haploidentical blood stem cell transplants. CBE-101 represents a novel approach with expanded cord blood-derived hematopoietic cell therapy, aligned with Cell BioEngines' vision to deliver 'off-the-shelf' cell-based therapies to patients, he added.

About Cell BioEngines

Cell BioEngines, Inc., founded in Dec 2022, is a clinical-stage biotech company focused on developing allogeneic 'off-the-shelf' stem cell-derived therapies as drugs for human disease treatment. The company leverages its proprietary platform technology using universal 'non-gene-modified' donor blood stem cells obtained from umbilical cord to produce clinical grade cells at scale.

For more information about Cell BioEngines, and to learn more the latest Company developments, please visit http://www.cellbioengines.com

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Mark Joubert., J.D. Chief Legal Officer Cell BioEngines, Inc info@cellbioengines.com

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Penn Dental Medicine Researchers Coaxing Stem Cells from Gum Tissue to Repair Nerves – Dentistry Today

Posted: July 21, 2024 at 2:34 am

Faced with repairing a major nerve injury to the craniofacial region, surgeons can use a nerve from an arm or leg to restore movement or sensation. This approachknown as an autograftis the standard of care, but it can take a toll on a previously uninjured body part, and the procedure doesnt always result in complete and functional nerve regrowth. Dr. Anh Le, Chair and Norman Vine Endowed Professor of Oral Rehabilitation in the Department of Oral and Maxillofacial Surgery at Penn Dental Medicine, is pioneering a different approach.

Le and collaborators are coaxing gingival mesenchymal stem cells (GMSCs)stem cells from gum tissueto produce nerve-supportive cells that facilitate nerve regrowth.

We wanted to create a biological approach and use the regenerating ability of stem cells, said Le. To be able to recreate nerve-supportive cells in this way is really a new paradigm.

For more than a decade, Les lab has explored the use of GMSCs to regenerate different types of craniofacial tissues and to treat osteonecrosis of the jaw, which can occur when a patient takes bisphosphonate, a drug used to treat metastatic cancer or prevent bone loss in osteoporosis. Her lab team was able to apply their previous understanding of GMSCs to facilitate their conversion into Schwann-like cells, the pro-regenerative cells of the peripheral nervous system that make neural growth factors and myelin, the insulating layer around nerves.

To move the work forward, Le collaborated with bioengineer D. Kacy Cullen of Penn Medicine, an expert in creating and testing nerve scaffold materials. Together they showed that infusing a collagen scaffold with these cells and using them to guide the repair of facial nerve injuries in animals was just as effective as an autograft procedure. Although the repaired gap was small, the team is continuing to refine the method to repair larger ones that often result from trauma or tumor-removal surgeries.

Le notes that this approach would enable patients with oral cancer or facial trauma to use their own tissue to recover motor function and sensation following a repair.

While Les group focuses on the head and neck, further work on this model could translate to nerve repair in other areas of the body as well.

Im hopeful we can continue moving this forward toward clinical application, she said.

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Surgeon Turns to Stem Cells to Help Treat Autism and Immune Disorders – The Well News

Posted: July 21, 2024 at 2:34 am

ORANGE PARK, Fla. Dr. Eric Weiss, a certified plastic and reconstructive surgeon with a thriving practice on the outskirts of Jacksonville, Florida, still remembers the moment he realized the transformative effect umbilical cord stem cells could have on those with autism and other immune disorders.

First introduced to stem cell treatment by his wife Christine, who was searching for medical help for their autistic son, Marston, Weiss became intrigued by its possibilities.

Compelled by the research, and the subsequent positive impact stem cell treatment had on his son a journey chronicled in a book he and his wife subsequently co-authored Weiss soon incorporated it into his practices.

Studies show that people with autism have neuroinflammation, similar to other immune disorders, which utilize stem cells as a treatment.

With the advance of technology and a better understanding of autoimmune disorders and autism, regenerative medicine has been a proven solution to these seemingly unsolvable health concerns.

While many people are hesitant to pursue this treatment because of the excessive cost and lack of FDA approval, Weiss says, I understand these fears because not everyone gets better. But the role of neuro-diseases is changing and this treatment is proven to help patients recover on a cellular level.

When addressing the FDA concerns, Weiss stressed that the FDA regulates drug companies, not health care, and that stem cell blood has been used in treatment for over 60 years.

The stem cells in the umbilical cord hold abundant powerful cells that help the human bodys healing capabilities. Stem cells work by sending chemical signals to old, damaged or injured cells to restore them.

However, stem cell research has faced substantial opposition from various cultural and religious groups around the world. The controversies stem from differing beliefs regarding the beginnings of life, the moral status of the embryo and the ethical implications of manipulating human cells.

Most controversial of all has been human embryonic stem cell research, because it involves the destruction of human embryos.

Regarding its efficacy, there have been multiple studies demonstrating the positive results of stem cell treatments in both children and adults. These treatments include remedies for neuro-related health concerns such as:

Weiss ultimate goal is to restore wholeness to patients by using stem cells to treat multiple health conditions. He is one of the only physicians in the country to utilize this form of treatment.

As the human body ages, it works harder to keep you healthy. Those with conditions like autism or other ailments are constantly struggling to find solutions.

As we age, our stem cells begin to die off at an alarming rate:

While stem cells can be found in various places throughout the body, the cells in the umbilical cord are the most useful. It is the least invasive form of stem cell gathering and as a bonus, the umbilical cord is chock full of them according to Weiss.

Today, patients come from all over the world to receive this treatment.

Weiss now dedicates two days a week of his practice to administering stem cell treatment.

There is still much to study regarding stem cell treatment for autism and other neurological diseases, but Weiss points those who are wary back to the science and the literature.

There have been lab studies, animal studies and human studies that show positive results with this treatment, so I want to do everything I can to help these patients get better, he said.

With the hope that this treatment becomes a standard form of care, Weiss is learning more every day and helping families and patients who need it most.

I thought to myself, why wouldnt this work for autism? Luckily it has, and now Im able to help families who have been through the same struggles as my own, Weiss said.

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Valentina Greco takes on new position as President of the ISSCR – EurekAlert

Posted: July 21, 2024 at 2:34 am

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"My focus will be on people and scrutinizing processes so that they better support the diversity of needs of our members across identities including geographies and career stages," Dr. Greco said."In turn this will increase opportunities for professional growth of our members and augment our collective impact. As I commit to this vision, I equally commit to speak with openness about the struggles that we have and will experience in order to make this vision a reality.

Credit: Yale School of Medicine

The ISSCR is thrilled to announce Valentina Greco, Yale School of Medicine, Genetics Department andYale Stem Cell Center USA, as its President. Her term began at the ISSCR 2024 Annual Meeting held in Hamburg, Germany that concluded on Saturday, 13 July 2024.

I am honored to be taking on the role of ISSCR President for the coming year, Dr. Greco said. Building on Amander Clarks efforts, my focus will be on people and scrutinizing processes so that they better support the diversity of needs of our members across identities including geographies and career stages. In turn this will increase opportunities for professional growth of our members and augment our collective impact. As I commit to this vision, I equally commit to speak with openness about the struggles that we have and will experience in order to make this vision a reality, Dr. Greco added.

Valentina Greco was born in Palermo, Italy on 3 September 1972. She earned an undergraduate degree in Molecular Biology at the University of Palermo, Italy (1996) where she studied the role of tumor suppressors in cell cycle using in vitro system in the lab of Aldo Di Leonardo (1995-1998), her first academic research experience. She was accepted by Suzanne Eaton and the EMBL/MPI-CBG PhD program, Germany (1998-2002) and fell in love with microscopy and the power of developmental biology using fly wing imaginal disc as a model system to understand epithelial cell communication. Dr. Greco subsequentially completed a post-doc training in the Fuchs lab at the Rockefeller University (2003-2009) where she learned about the mammalian skin hair follicle as model system for stem cell driven regeneration. She was then hired as an Assistant Professor in the Genetics department at Yale School of Medicine by Richard Lifton and Haifan Lin (1 August 2009).

Dr. Greco is currently the Carolyn Walch Slayman Professor of Genetics as well as the Co-chair of Status of Women in Medicine (SWIM) at the Yale School of Medicine. She and members of her lab feel excited about visual driven research to study how cells behave in a living mouse. The team understands cell behaviors as an expression of the architectures and principles that govern the tissues thesecells inhabit, much like human behaviors are an expression of the systems and structuresin whichthey are embedded(e.g. a lab, anorganization). The Greco lab is passionate about identifying the mechanisms that govern communication and cooperation to sustain function over a lifetime.

Dr. Greco has served in numerous leadership roles in the scientific community including many within the ISSCR over the last decade. She also serves on numerous additional boards including President Elect for the Society of Investigative Dermatology (SID), SID Board member 2016-2020, Member of the National Arthritis and Musculoskeletal and Skin Diseases Advisory Council (NAMSAC (2022-2024)), Member of the Yale Ciencia Academy Advisory Committee, Member of the 2030STEM Leadership Team and Secretary of Board of Directors of the Life Science Editors Foundation (2020-2023).

Greco lab research has been recognized by numerous accolades awarded to both lab members and Dr. Greco. She in particular has received the 2014 Women in Cell Biology Junior Award (WICB) for Excellence in Research from the American Society of Cell Biology (ASCB), the 2014 ISSCR (International Society for Stem Cell Research) Outstanding Young Investigator Award, the 2015Robertson Stem Cell Investigator Award from the New York Stem Cell Foundation (NYSCF), the 2015 Mallinckrodt Scholar Award, the 2016 Early Career Award from the American Society of Cell Biology (ASCB), the 2016 HHMI Faculty Scholar Award, the 2017 Glenn Foundation Award, the 2017 Class of 61 Award by the Yale Cancer Center, the 2019 NIH DP1 Pioneer Award and the 2021 ISSCR Momentum Award. Dr. Greco finds it particularly meaningful to have received the 2018 Yale Mentoring Award in the Natural Sciences, the 2019 Yale Genetic Department Mentoring Award, the 2019 Yale Post-doc Mentoring Award.

The ISSCR is equally pleased to announce Hideyuki Okano, MD, PhD, Keio University, Japan is President-elect and will serve as president officially starting 1 July 2025. Lorenz Studer, MD, founding director of the Center for Stem Cell Biology and member of the Developmental Biology Program, Memorial Sloan Kettering Cancer Center, USA, is the new Vice President.

The following three members are newly elected to the ISSCR Board of Directors and beginning their three-year term: Jacqueline Barry, PhD, Cell and Gene Therapy Catapult, UK, Tenneille E. Ludwig, PhD, WiCell, USA, and Thomas A. Rando, MD, PhD, University of California, Los Angeles, USA.

The following members are starting their second, three-year term as a result of the 2024 election: Melissa Carpenter, PhD, Carpenter Consulting Corporation, USA, Malin Parmar, PhD, Lund University, Sweden, and Mitinori Saitou, MD, PhD, Kyoto University, Japan.

Learn more about the ISSCR Board of Directors.

About the International Society for Stem Cell Research With nearly 5,000 members from more than 80 countries, the International Society for Stem Cell Research is the preeminent global, cross-disciplinary, science-based organization dedicated to stem cell research and its translation to the clinic. The ISSCR mission is to promote excellence in stem cell science and applications to human health. Additional information about stem cell science is available at AboutStemCells.org, an initiative of the Society to inform the public about stem cell research and its potential to improve human health.

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New B-cell therapy eases disease severity, delays onset: Study – ALS News Today

Posted: July 21, 2024 at 2:34 am

Repeat infusions of naive immune B-cells were safe and delayed disease onset, reduced disease severity, and extended survival in animal models of amyotrophic lateral sclerosis (ALS), a study found.

The approach was also deemed safe in the treatments first in-human case study, in which it eased disease severity and reduced biomarkers of inflammation in a person with advanced ALS.

We were able to show safety and efficacy, as well as how the B cell therapy worked in mice, and we demonstrated that our approach was safe and feasible in a human, senior author Mark C. Poznansky, MD, PhD, an attending physician at Massachusetts General Hospital (MGH) and a professor of medicine at Harvard Medical School, said in a hospital press release.

Details of the potential new B-cell therapy were published in The FASEB Journal in the study, Allogeneic B cell immunomodulatory therapy in amyotrophic lateral sclerosis.

This is a first study of its kind to apply B cells to the treatment of ALS and sets us up for a trial of this new treatment approach to a currently incurable disease, said Poznansky, who is also the director of the Vaccine and Immunotherapy Center at MGH.

Elevated inflammation is known to play a role in the onset and progression of ALS, a condition marked by the loss of motor neurons, the nerve cells that control certain body movements.

Mitigating neuroinflammation via immune targeting may provide an effective therapeutic strategy for attenuating neuronal loss in ALS, the researchers wrote.

B-cells are best known as the immune cells that produce antibodies to protect the body from infections and other harmful substances. But recent research has demonstrated that these cells can also respond to tissue injury, help rebalance immune function, and coordinate tissue repair.

Previous work by lead author Ruxandra F. Srbulescu, PhD, assistant professor of neurology at Harvard Medical School, showed that naive B-cells reduced inflammation and promoted recovery in mice with skin or brain injuries.

These benefits were driven by a process the team dubbed pligodraxis, in which B-cells can adopt immunoregulatory and neuroprotective properties.

What we observed early on in preclinical studies has been a remarkable effect of B cells in the context of brain lesions both brain structure and function were protected by treatment with these cells, which made us consider applying them as a therapeutic in the context of neurodegenerative disease, Srbulescu said.

A team led by Srbulescu and Poznansky tested this type of B-cell therapy in an ALS mouse model and, for the first time, an individual with ALS.

The ALS mouse model carried mutations in the SOD1 gene, a known ALS-related gene. The mice received 10 weekly doses of mature naive B-cells isolated from the spleens of donor mice, administered directly into the bloodstream.

Results showed that the B-cell treatment was safe and significantly delayed disease onset compared with a control treatment. Treated animals also tended to have better neurological function, indicating less severe disease, and a longer survival time, though the differences between groups failed to reach statistical significance.

Tissue analyses showed significantly less abnormal or dying motor neurons, as well as reduced markers of neuroinflammation.

We were able to show that B cells, which can be readily obtained from the blood, could treat ALS in a well-established mouse model of the disease, and we obtained permission from the US Food and Drug Administration and our hospital to try this treatment approach in an individual with ALS, Poznansky said.

The participant was a man in his 50s who received a diagnosis of ALS following the onset of bulbar symptoms, which affect the muscles of the face, jaw, and throat, impairing speech and swallowing. He was being treated with the standard ALS treatment riluzole (sold as Rilutek, among other brand names).

B-cells were collected from the participants adult child, who was an immunological match, and infused into the patients bloodstream twice, 60 days apart. This was deemed was safe and did not trigger an abnormal inflammatory response.

After the first infusion, the patient had a modest improvement in scores on the revised ALS Functional Rating Scale (ALSFRS-R), an assessment of disease severity that measures the ability to perform everyday activities.

After the second infusion, the participants ALSFRS-R score again improved but gradually declined, as expected in ALS, thereafter. The participant withdrew from the study on day 136.

Blood tests showed marked treatment-related reductions in markers for body-wide inflammation and in pro-inflammatory immune cells.

Still, levels of neurofilament light chain, a marker of nerve cell damage, continued to rise, suggesting that the intervention at a relatively late/end stage of disease progression would be insufficient to halt neurodegenerative processes, the researchers wrote.

A Phase 1 trial will be needed to further evaluate the safety and efficacy of this novel approach to treating ALS, the researchers concluded. Such a trial is now in the planning stage, Poznansky said.

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Treating HCC with genetically modified NK-cell therapy – Drug Target Review

Posted: July 21, 2024 at 2:34 am

Blocking the TGF- signalling pathway produced effective antitumour activity against hepatocellular carcinoma.

Scientists at the University of California San Diego have discovered that genetically modified NK-cell therapy improves the targeting and treatment of hepatocellular carcinoma (HCC), the most treatment-resistant form of solid tumour.

Unlike chimeric antigen receptor (CAR)-expressing T-cell therapy, the NK treatment does not require personalisation, meaning it could be mass produced and shelf-ready for patients to begin therapy without delay. Dr Dan Kaufman, lead study author and director of the Sanford Advanced Therapy Center, explained: To some extent all tumour cells perhaps hepatocellular carcinoma more so inhibit immune cells that try to kill themThis is one key reason why some immunotherapies like CAR T cells have been less successful for solid tumours than for blood cancers the immunosuppressive tumour microenvironment.

The researchers produced stem cell-derived NK cells in which the receptor for transforming growth factor beta (TGF-) was disabled. The liver and HCC tumours contain a high amount of TGF-, which inhibits immune cell activity and enables cancer to grow. It was discovered that typical NK cells without the disabled receptor, like CAR T cells, were not effective in destroying the cancer. When they assessed the modified NK cells, Dr Kaufman stated: we got very good antitumour activity and significantly prolonged survival.

These studies demonstrate that it is crucial to block transforming growth factor beta at least for NK cells, but I also think its true for CAR T cells, Dr Kaufman added. If you unleash NK cells by blocking this inhibitory pathway, they should kill cancer quite nicely.

In the future, Dr Kaufman believes their findings will aid clinical trials of numerous research groups and companies, whether they study CAR T-cell or NK-cell therapies and tackle hepatocellular carcinoma or other difficult types of solid tumours. He concluded: Anyone developing such therapies for solid tumours should be working to inhibit transforming growth factor beta activity to improve cancer-killing and attain effective anti-tumour activity.

This study was published in Cell Stem Cell.

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My Decision to Receive CAR-T Cell Therapy for Multiple Myeloma – Curetoday.com

Posted: July 21, 2024 at 2:34 am

When Mindy's previous treatment stopped working for multiple myeloma, she opted for CAR-T cell therapy.

Following an infection that was resistant to treatment and a few extra blood tests, the doctor advised me to see a hematologist (that was OK!) or an oncologist (not so good to hear). In January 2007, the diagnosis of multiple myeloma was followed by several new normals for my family and me.

Let the journey begin. What is myeloma? How do my husband and I tell and support my daughters (one finishing high school and the other in college)? How do I support my husband? Will I survive this? Who will be there to support me? What treatments will I need? Can I trust my doctor to provide the best treatment for me? Who are all these strange people in the clinic? What about my friends? My co-workers? Would they treat me the same? Who will understand what I am going through? After letting the diagnosis set in, while I was ready to fight this battle, I did not realize what a challenge this would be. I was faced with so many factors out of my control.

Fast forward 16 years. It took lots of energy, questioning, researching and talking. Some of my questions and fears were addressed, but new ones were constantly popping up. I remained in the working world for more than 13 years, sometimes walking from my office building to Massey Cancer Center in Richmond, Virginia for treatment. One autologous (my stem cells) transplant. One allogeneic (donor) transplant. Yes, I am now my brother! And thank goodness for my brothers loving generosity. Seven years of partial remission after transplants. What happened during these 16 years? Too many times, I heard that my numbers were stable followed by the news that my numbers were slowly climbing. More treatment regimens. It was an emotional roller coaster as I moved forward.

I cherished amazing inspiration, support and laughs from my dear family, my health care team, friends and other myeloma colleagues. I had completed several half-marathons to support The Leukemia & Lymphoma Society; organized and co-led a local International Myeloma Foundation support group; and volunteered at Massey Cancer Center. These activities kept me connected with my community, others who were impacted and it raised my resiliency.

The best celebrations enriching my life that I never expected was witnessing my daughters become beautiful, caring, compassionate, strong professional women; becoming a mother-in-law; and then becoming a grandmother! Life goes on in beautiful ways. For this, I am grateful.

In 2023 after two years of a specific treatment regimen that kept my numbers stable that tricky myeloma figured out a way to stump us again. Now what? CAR-T cell therapy? Bispecific antibodies? How grateful that I have options, but still anxious moving forward. I chose CAR-T cell therapy for the one and done option. The beginning of my CAR-T cell therapy journey was a little scary and only a bit bumpy. But, it was well worth the effort to power through! Twelve months after CAR-T cell therapy, I am still in complete remission and living my best life!

My favorite sayings during my journey have been This too shall pass and It is what it is. If I could use two words to describe my journey, they are hope and resilience. How are hope and resilience part of your story?

This post was written and submitted byMindy C. Fast. The article reflects the views ofFastand not of CURE. This is also not supposed to be intended as medical advice.

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Cell therapy shows potential in multiple age-related diseases – Longevity.Technology

Posted: July 21, 2024 at 2:34 am

Hot on the heels of receiving the FDAs coveted Regenerative Medicine Advanced Therapy (RMAT) designation for its Lomecel-B product, Longeveron this week revealed its lead cell therapy has also been granted Fast Track designation for the treatment of mild Alzheimers. The Miami-based biotech has now received five different FDA designations for Lomecel-B, and this latest one should help streamline its development and expedite its review as a potential Alzheimers therapy.

The Fast Track designation comes on the back of compelling Phase 2 trial results, which demonstrated that Lomecel-B slowed or prevented the progression of Alzheimers in some groups of patients with the mild form of the disease. Longeverons aim is now to move the therapy into the next phase of trials in Alzheimers as soon as possible, potentially as early as next year. But the company is not putting all its eggs in one basket

Longevity.Technology: Derived from the bone marrow of young, healthy adult donors, Lomecel-B, a so-called living cell product, has demonstrated pro-vascular, pro-regenerative and anti-inflammatory effects. With multiple mechanisms of action, the drug is being explored for its potential in several indications, spanning neurodegeneration, frailty and heart conditions. Could we be looking at a longevity drug in the making? To learn more, we caught up with Longeveron CEO, Wael Hashad.

Longeveron was founded with a mission to target age-related diseases, which are often characterized by chronic inflammation, combined with a decline in immune system and blood vessel function. The companys approach involves building therapeutics, like Lomecel-B, from special living cells called medicinal signaling cells (MSCs) derived from donated bone marrow tissue.

One of the key mechanisms through which MSCs exert their therapeutic benefits is by releasing exosomes, growth factors and other proteins, such as anti-inflammatory cytokines, which have the potential to significantly reduce inflammation, while stimulating the promotion of regenerative and repair responses. This is why, rather than targeting amyloid plaques or neurofibrillary tangles, Longeveron is going after the inflammation of the central nervous system that is increasingly recognized as a driver of neurodegeneration in Alzheimers.

With preliminary clinical data showing that Lomecel-B simultaneously targets multiple aging-related processes, Hashad explains that an important part of the recent Phase 2 Alzheimers trial was demonstrating these effects in humans.

We wanted to see if there was any data supporting the hypothesis of the mechanism of action, which is the anti-inflammatory and pro-vascular effect of the drug, he says. So, in addition to other clinical scales that you typically use in Alzheimers trials, we also added MRI measurement of brain volumes, which is a very objective exploratory endpoint.

This means that, in addition to study results showing that Lomecel-B was safe, well-tolerated, and demonstrated statistically significant improvements in specified cognitive function measurements in certain groups, Longeveron also generated some compelling MRI data.

We got a big, positive surprise from the effect that we saw on the MRI, which indicated that there was less shrinkage in the brain compared to placebo, says Hashad. And that was significant, not just on the total volume of the brain, but also on the hippocampal volume, which is the memory formation center of the brain.

This data, Hashad believes, was instrumental in the company receiving the recent designations from the FDA, which should help accelerate the process to move Lomecel-B forward as a potential treatment for mild Alzheimers.

Recent FDA guidance for Alzheimers disease indicates that clinical trials showing an effect on a surrogate endpoint that is determined to be reasonably likely to predict clinical benefit can be the basis for accelerated approval. The MRI data from the Phase 2 trial, which can be considered a surrogate endpoint, may significantly expedite the development and approval process for Lomecel-B.

We can now have a much more active dialogue with the FDA, and potentially discuss the possibility of an accelerated approach to bring the product to the market using surrogate endpoints, for example, he says. The next step is to meet with the FDA before the end of the year to talk about the blueprint for the next phase of development.

With chronic inflammation also heavily implicated in many other age-related conditions, Longeveron believes that the anti-inflammatory, pro-vascular mechanism of Lomecel-B can also play a role beyond the brain.

We have an indication that Lomecel-B may work by reducing levels of inflammation, and also improving the health of the vascular system and therefore the blood supply and the blood flow to the brain and other areas of the body, says Hashad.

In addition to its program in Alzheimers, Lomecel-B is also being explored as a treatment for age-related frailty, as well as a congenital heart condition called hypoplastic left heart syndrome (HLHS).

Age-related frailty, or sarcopenia, is thought to affect around 15% of individuals aged 65 and older, and significantly heightens the risk of adverse clinical outcomes from disease and injury. Despite its prevalence, no medical treatments for the condition have been approved by the FDA or any other regulatory body.

Longeveron has commenced multiple clinical trials of Lomecel-B in frailty but has faced some challenges in terms of determining the endpoints needed to demonstrate efficacy to the FDA.

We have seen good data in our studies, but there is no product approved for aging related frailty, and were still trying to get an agreement with the FDA on what that indication is, says Hashad. So far, what we have used in our trials is the six-minute walk test, which evaluates the distance a patient can walk within six-minutes. We have seen a statistically significant improvement in our Phase 2 study with this metric at nine months. We will continue to work with the FDA which also wants to see a more objective assessment as well, so were trying to get alignment on that before we proceed forward on that program.

Longeverons more recent work in cardiology is of keen interest to the companys founder, the renowned cardiologist Dr Joshua Hare. While the program is currently focused on a rare, inherited condition, Hashad admits that the company is exploring the potential to target other heart-related indications.

There are certain things that were working on that I cannot disclose at this time, he says. But cardiology remains one of Joshs biggest passions, and we believe that Lomecel-B may have multiple potential applications within the heart disease area as well as other products that were also working on.

So, if Lomecel-B goes on to prove successful in multiple age-related diseases, would that make it a potential longevity drug? Hashad doesnt bite, although he does believe the therapy could have more applications in other diseases.

As a startup, we must focus on the work that we currently have at hand, but hopefully, as we continue to advance our work and grow, then we can embark on other areas of disease that can also benefit patients in the future, he says. There is no doubt that life expectancy is increasing around the world the question is how we ensure that people are not just living longer but living longer with a better quality of life, whether by improving their heart health, muscle health, or brain health. We believe that Lomecel-B, and the other products that were working on in our labs, can potentially help to improve that quality of life.

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Cell therapy shows potential in multiple age-related diseases - Longevity.Technology

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