Stem Cell Clinic

LVMH’s head of research: ‘What matters for Dior is to invent and be a … – Glossy

Reverse aging research is all the rage, with medical companies and now beauty brands exploring how to tap into a newer and more advanced concept than anti-aging.

Most recently, Dior Beauty stepped into the spotlight with its late-March announcement at the 21st Aesthetic and Anti-Aging Medicine World Congress in Monaco that it had established an International Reverse Aging Scientific Advisory Board. Comprising the board are 600 researchers and 18 experts, including Dr. Nicola Neretti, a biologist at the Institute for Brain & Neural Systems at Brown University, and Dr. David Furman, the director of the 1000 Immunomes Project at Stanford University. Diors own research will add to the knowledge base on aging science around stem cells, inflammation and cell communication. The idea is to better understand the 12 signs of aging, including genomic instability, epigenetic changes, stem cell fatigue and chronic inflammation. It will then weave that knowledge into skin-care product innovations to reverse these signs, according to Bruno Bavouzet, evp of R&D at LMVH, who oversees all beauty brands including Dior, Givenchy, Fresh and Benefit Cosmetics.

Our No. 1 goal is understanding the different path [around aging], from a biological standpoint. With time, we will make discoveries on the real mechanisms that accelerate aging, he said. No. 2 is to identify the potential actions that we can have through cosmetic [products] and how can we can [reverse] the biological age.

Bavouzet spoke with Glossy further about the concept of reverse aging, including why Dior is exploring the topic, what can be gained with the new advisory board and what a collective intelligence approach means. This interview has been lightly edited and condensed for clarity.

What does reverse aging mean?When we talk about reverse aging, basically what we know is that chronological age is different from biological age. That means that if you are 40- or 50-years-old, that definitely is your chronological age, but your biological age and the age of your cells can be different. We know in medicine that you can reverse your biological age by adding good health practices. What we dont always understand is the mechanisms of these aging processes and how they are connected to each other. The first stage [of our research] is to better understand the underlying mechanisms, with all their biological complexity, and why we are aging differently.

Reverse aging starts with the 12 hallmarks of aging. Historically, anti-aging people used to work on one approach, perhaps it was stem cells or polymers, et cetera. But we believe the best approach [to reverse aging or anti-aging] is a holistic approach. But that can be complex. Because there has been progress in biological discoveries and big data, we are able to [process] complex information.

What is the boards purpose and what does it do?We believe [the field of] reverse aging is impacted by very different biological mechanisms. And we believe that we need to have all the people in different fields work together, exchange information and build bridges between topics. We are working on reverse aging internally, obviously, but we want to accelerate that [research], meaning that we need more insights, from a scientific standpoint, on discoveries, [as well as] more data, and more experience. The board is advising us on our research. It will regroup several times a year. But throughout the year, we might also have continuous exchanges on specific topics with around four people. Its about building what I would call a collective intelligence approach, meaning that we are grouping all these people who are experts on the human body, while we [Dior] work on the skin. Not all of them are skin experts, but they can help us translate some research on different [scientific] topics to the skin, as well.

Why is Dior Beauty the right LVMH brand for this research?Dior has always been science-focused. Dior was the first brand back in the 1980s to launch liposomes in the skin-care industry. Thats really something that was important at the time with big effects. Its true that when you think about Dior, you obviously think about fashion and makeup. But Dior has always been thinking about and integrating the idea of how to make women happier. At an early stage [in the brands history] it started to launch skin-care products that were acting on the transformation of the skin. What matters for Dior is to invent and be a pioneer in beauty, as it has in the fashion industry.

What has spurred this reverse aging focus?There is a continuity from what has historically been the focus of anti-aging. The topic is also led by medicine. There has been a lot of progress in medicine and many therapies, like stem cells which are considered the mother cell, and trying to apply the same science to cosmetics. Obviously, we cannot do exactly the same thing. But when we started to work about 10-15 years ago on the microbiome of the skin, we got inspiration from work that was focused on [gastrointestinal science]. It is a pretty similar approach we are conducting here with reverse aging.

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Exosome Therapy For Skin Care & Hair Loss Is A Promising … – The Zoe Report

Our skin is designed to heal itself, but like the need to down a shot of espresso to get going into the morning, sometimes the organ needs an extra kick. Thats where exosome therapy comes into play, the latest trend in the aesthetics space. Falling under the regenerative therapy umbrella, a category of treatments that use stem cells or platelets (like in PRP) to replace or regenerate the skin in order to improve texture and tone, exosomes are poised to become the next It girl in skin care. And like PRP, this treatment can also be used to treat hair loss by stimulating the hair follicles.

Until recently, exosomes were dismissed as mere byproducts of cellular activity, but recent research has revealed their incredible potential to transform how we approach skin care and hair care a modern-day Cinderella story, if you will. Especially because the science behind the treatment has a long-lasting, holistic effect rather than arguably functioning like a Bandaid for skin concerns like Botox and many fillers do. Exosomes are usually derived from stem cells and contain signaling molecules and growth factors designed to stimulate regeneration and healing, explains Dr. Julie Russak, M.D., board-certified dermatologist and founder of Russak Dermatology Clinic in New York City.

But, as with any trendy new beauty treatment, its important to get the facts before touting it as a game-changing innovation. Ahead, TZR gets the lowdown on exosome therapy with the help of a few dermatologists, including its benefits and whether or not it lives up to its promises of healthy skin and hair.

To put it simply, exosomes are tiny vesicles (bubbles) that help cells communicate with each other. When they are injected into tissue, they can reprogram surrounding cells to start growing and regenerating. These vesicles contain amino acids, proteins, lipids, peptides, and growth factors, which are all beneficial to the skin and hair. This manifests aesthetically as improved skin texture, increased hydration, reduced fine lines and wrinkles, and improved overall skin tone and brightness. They can also promote hair growth by stimulating and strengthening hair follicles, and promotes thicker strands.

It's important to note that exosomes need a little help to work their magic. When applied topically, they are unable to penetrate the skin's surface and end up just hanging out on the outermost layer. To be effective, they must be infused deep into the epidermis, which is why they are typically used in conjunction with other treatments that create disruptions in the skin like resurfacing lasers or microneedling (a technique that creates tiny channels on the skin's surface). The therapy can also be combined with other regenerative therapies, like PRP or stem cell therapy, to further enhance their effectiveness.

Another reason why you wont see the vesicles on the inkey lists of skin care products at Sephora? Exosomes are highly fragile and need to be refrigerated at cold temperatures; they cannot survive for long outside of these conditions. So, for example, a cream touting exosome technology could possibly possess [similar] benefits, but the end product is non-living, and more testing needs to be done to determine the biological effect, explains Dr. Sanjay Batra, Ph.D., a regenerative medicine expert.

Exosomes are an excellent solution for tackling wrinkles as these tiny vesicles can effectively stimulate cell growth and repair. Due to their unique ability to transport to directed sources, they are far more efficacious than simply applying peptides or growth factors directly on the skin, explains board-certified dermatologist and founder of Ocean Skin & Vein Institute, Dr. Divya Shokeen, M.D. Anyone looking to bring a bit of vibrancy back to their face can benefit from the therapy, as it increases collagen and elastin production and the dermatologist says, has been shown to have a significant visible reduction in fine lines, aging, and sunspots. What's more, exosomes have anti-inflammatory properties that can help calm skin. Research also shows they can minimize the symptoms of psoriasis and atopic dermatitis.

Within just one week of treatment, you may notice an improvement in texture, tone, firmness, the appearance of pore size, and the look of fine lines. Continued use of exosome therapy can lead to long-term improvements, resulting in healthier, stronger, and more youthful-looking skin over time, adds Dr. Russak.

Exosomes not only trigger healing and tissue regeneration on your face, but also in the cells of hair follicles on the scalp, which results in a stronger potential for regrowth in those experiencing hair loss. Russak explains, By delivering these molecules to the skin on the scalp and directly to the hair follicles, we can provide intensive hair rejuvenation benefits and signal the hair follicles to grow healthy hair. When hair follicles quit, we deliver both exosomes into the scalp to awaken the follicle and stimulate hair growth.

While many people turn to PRP injections for hair growth, Dr. Russak notes, exosomes are the superior option compared to PRP due to their increased potency and reliability. Unlike PRP, which is derived from platelets in our blood, the vesicles are extracted from stem cells found in sources such as fat, umbilical cords, or bone marrow. "The growth factors in our PRP may be weaker depending on our age and health status," she explains. "Exosomes contain growth factors and signaling molecules for regeneration and healing, which are extracted from inside the fresh cell. Many are also from embryonic stem cells, which are brand-new cells that have the most regenerative potential possible." Research also shows that they can help cells grow and heal wounds better than PRP can without having to use a patient's own blood.

Exosome therapy is paving the way for a new era in beauty treatments that prioritize skin and hair regeneration over simply masking common concerns like visible signs of aging. With the ability to promote natural healing and rejuvenation, this therapy offers a promising solution for those looking to keep their skin and hair in tip top shape for the long term.

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Live Event for April 21: Sleeping pill reduces levels of Alzheimers proteins – Newswise

What:Researcher at Washington University School of Medicine in St. Louis will discuss the studywhich involved a sleeping aid known as suvorexant that is already approved by the Food and Drug Administration (FDA) for insomnia, hints at the potential of sleep medications to slow or stop the progression of Alzheimers disease.

When:April 21st, 2PM EST

Where:Live Events Zoom Room (link will be given once you register)

Who: Dr. Brendan P. Lucey, MD -Associate Professor of Neurology, Section Head, Sleep Medicine

Researcher's info:

Brendan Lucey is associate professor of neurology and Sleep Medicine Section head. Born and raised in Burlington, Vermont, he received his undergraduate degree at the University of Vermont and his medical degree from the Johns Hopkins University School of Medicine. Following medical school, Lucey completed his neurology residency at Washington University and a clinical neurophysiology fellowship at Brigham and Womens Hospital. From 2008-2012, Lucey was on active duty in the U.S. Air Force and then joined the Department of Neurology at Washington University.

Luceys current research interests are in sleep, aging and Alzheimers disease. His lab focuses on studying the potential of sleep interventions to prevent or delay the onset of Alzheimers disease. Using lumbar catheters, he investigates how sleep affects different markers of Alzheimers disease changes in the brain such as amyloid-beta and tau. Lucey is also interested in whether or not sleep changes may be non-invasive markers for Alzheimers disease progression.

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Novel PSMA Targeted Therapy Induces Preliminary Responses … – Targeted Oncology

Jones T. Nauseef, MD, PhD

Since prostate-specific membrane antigen (PSMA) became a standard-of-care therapy for patients with metastatic castration-resistant prostate cancer (mCRPC) in 2022, multiple agents showing potential to offer higher potency have entered the pipeline.

225Ac-J591, an alpha-emitter antibody-drug conjugate is 1 PSMA targeted therapy making headway, according to Jones T. Nauseef, MD, PhD. A phase 1 dose-escalation study investigating the agent in patients with mCRPC recently completed and is advancing to phase 2.1

There are a couple different ways to target PSMA. One is with a small molecule ligand, and the other one would be with a monoclonal antibody. J591 is our monoclonal antibody that has terrific targeting on cells that are expressing PSMA, and it helps with internalization into the cell. This monoclonal antibody is going to go throughout the body and with it carry actinium 225, and that is a potent radionuclide. With the antibody going to the sites where you want it to go, it will bring with its payload, which is radionuclide, explained Nauseef, assistant professor of medicine, Division of Hematology and Medical Oncology, Weill Cornell Medicine, and assistant attending physician at NewYork-Presbyterian Hospital, in an interview with Targeted Oncology.

Results from the 24 patients with mCRPC included in the phase 1 study of 225Ac-J591 showed that the administration of a single fractionated cycle of 225Ac-J59l led to few high-grade adverse events. Moreover, preliminary efficacy was demonstrated with the drug in terms of prostate-specific antigen decline and changes in circulating-tumor (ct)DNA.

In the interview, Nauseef discussed how 225Ac-J591 achieves apoptosis, and findings from the phase 1 study, which he presented during the American Association (AACR) for Cancer Research Annual Meeting 2023.

TARGETED ONCOLOGY: Can you talk about the introduction of PSMA-targeted therapy in the prostate cancer space and the impact it has had?

Nauseef: The treatment of prostate cancers, specifically metastatic castrate-resistant prostate cancer, has changed so much in 25 years. [There has been] approval of drugs in many sectors, [including] AR pathway inhibitors, taxanes, targeted therapies, you name it. Most recently, with the approval of 177 Lu- PSAM-617, targeted radiotherapy is now reality in prostate cancer. This has come about because PSMA is just such a terrific target. Almost all of it is on prostate cells or prostate cancer cells. There are some in physiologic spaces, but otherwise, it makes for a nice target. The drugs that are now approved have had some real successes.

What can say about the agent 225Ac-J591 and the rationale for using it to treat patients with mCRPC?

There are a couple different ways to target PSMA. One is with a small molecule ligand, and the other one would be with a monoclonal antibody. J591 is our monoclonal antibody that has terrific targeting on cells that are expressing PSMA, and it helps with internalization into the cell. This monoclonal antibody is going to go throughout the body and with it carry actinium 225, and that is a potent radionuclide. With the antibody going to the sites where you want it to go, it will bring with its payload, which is radionuclide.

Image Credit: heitipaves [stock.adobe.com]

This is important. Patients with mCRPC can have cancer anywhere in their body, and you want to be able to target this. You can do that effectively with an antibody like J591 and bring with it a potent radionuclide. Thats one of the things that's so nice about the potency of this drug is that it has a short path length. Though you're delivering a high energy that even a single hit on a cell can result in double stranded DNA breaks and cell death, the path length is short, so your toxicity may be lower because the drug isn't going to get away from where you want it to be. This will be true anywhere that a patient has cancer. That is commonly in the bones and the lymph nodes, but also visceral sites of disease, such as the liver and lungs.

Can you explain about the study design and methods used in this study?

The data we presented was our phase 1 study of patients who did not have prior exposure to Lutetium-177-PSMA-617 in all patients with mCRPC. These patients were all the men had prior exposure to taxane-based chemotherapies, and half of them had received 2 or more prior androgen receptor signaling inhibitors, but everyone needed to have at least 1. This study design was modified 3 + 3 dose-escalation.

A key point to note about this study, in contrast to the VISION study [NCT03511664] that led to the approval of PSMA 617 is that PSMA PET imaging was done at baseline and was part of the screening criteria. But avidity or positivity on the scan was not required to be enrolled in the study. This is important because you could essentially take almost any patient with mCRPC and put them in this study and be able to generalize the results that we observed. In the dose-escalation, we had 3 cohorts, and after seeing what the toxicity was, we added a fourth cohort. That was how we established our recommended phase 2 dose.

What results did you present during AACR 2023?

Anytime we do a phase 1 study, everyone is interested in any evidence for preliminary efficacy. But the primary objective of these studies is to determine the dose-limiting toxicity, and the recommended phase 2 dose, and those things were established in our study.

Regarding the dose-limiting toxicity [DLT] period, we defined this period as the 8 weeks following the first dose of the drug, and the drug is given twice, once on day 1, once on day 15 in a dose intense fractionated regimen, during a single cycle. We looked specifically for DLTs related to neutropenia, thrombocytopenia, and then any-grade toxicity that would have delayed receipt of the second dose by greater than 2 weeks. In the dose-escalation, we observed at the highest dose, 2 DLTs. One patient had grade 4 thrombocytopenia, and that patient had prior exposure to PSMA 617 radioligand therapy. Then, a second DLT was in a patient with grade 2 thrombocytopenia that resulted in delay of the second dose by greater than 2 weeks. Following that, we did revise the protocol as mentioned and made an intermediate dose, a cohort 2.5 at 120 kg, delivered in 2 doses. In this group, 6 patients were treated and only 1 DLT was observed, which was grade 4 thrombocytopenia.

The grade 4 thrombocytopenia that I mentioned was transient and reversible. I saw this patient last month. His platelets are now back to normal, there were no high-grade non-hematologic toxicities, and fatigue and pain flare were common, but were all grade 1 or grade 2.

As far as efficacy goes, we observed very good efficacy regarding PSA change. Of the patients treated who were evaluable, we saw PSA declined in almost every patient with commonly PSA declines of 50% or 90%. Of those patients who had a PSA decline of 50%, 12 of the 16 were confirmed as PSA50, and 1 of those patients is still pending as confirmatory PSA.

We also saw good efficacy regarding circulating tumor cells. There were 14 patients that had CTCs at baseline, and at 12 weeks were available for change. Across the board we saw good responses.

Breaking that down into greater detail, most of the patients did have a CTC response. Almost 80% of patients had any CTC response of the patients who had variable CTCs at baseline. At 12 weeks, more than 50% had a 50% or greater decline in CTC count. There were also 5 patients who were unfavorable at baseline, and 2 of them converted to favorable. I think most excitingly, of the 11 patients who had detectable CTC as baseline, 6 of them became undetectable at 12 weeks, consistent with a good response.

What are the next steps with this research?

We've identified as the recommended phase 2 dose 120 KBq/kg given into fractions. We are proceeding with the phase 2 study with that dose.

There are important changes to highlight that will be occurring in the phase 2. One is the addition of a radioligand-specific patient-reported outcome. This is nice because we can observe whether the adverse event profile is different than it might be from chemotherapy or targeted agent or something on the AR signaling pathway. This patient-reported outcome was tailored specifically to radionuclide therapies. I think that's important to highlight and it makes us better able to take care of these patients as this becomes a more common modality. The second change is the introduction of dosimetry for alpha emitters. This will be done using multi-timepoint specification for Francium and Bismuth. Using dosimetry will allow us to measure and calculate the radioactivity dose received rather than just delivered, which I think is important as we move into using alpha emitters more commonly.

REFERENCE:

Nauseef JT, Sun M, Thomas C, et al. CT014 - Phase I dose-escalation study of fractionated dose 225Ac J591 for metastatic castration resistant prostate cancer. resented at: 2023 AACR Annual Meeting; April 14-19, 2023; Orlando, FL. Abstract CT-014.

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TIBI Director and CEO Ali Khademhosseini receives Technology … – EurekAlert

(LOS ANGELES) April 20, 2023 - Dr. Ali Khademhosseini has been awarded the 2023 Technology Innovation and Development Award from the Society of Biomaterials (SFB). The award honors those whose research, scientific innovations, and leadership are used to develop novel products or technologies to benefit patients.

Dr. Khademhosseini is the founding Director and CEO of the Terasaki Institute for Biomedical Innovation (TIBI), which incorporates a variety of interdisciplinary research platforms and uses patient-derived cells for micro- and nanoscale technologies to achieve 'personalized' approaches to medical problems.

Prior to coming to TIBI, he was the Founding Director of the Center for Minimally Invasive Therapeutics and the Levi Knight Professor of Bioengineering, Chemical Engineering, and Radiology at the University of California-Los Angeles (UCLA). Before UCLA, he was at Harvard University, a Professor at Harvard Medical School and faculty at the Harvard-MIT's Division of Health Sciences and Technology, Brigham and Women's Hospital, and associate faculty at the Wyss Institute for Biologically Inspired Engineering.

Over the course of his career, Dr. Khademhosseini has pioneered high-performance, personally tailored biomaterials and has been a leader in tissue engineering, vascular tissue biofabrication, cell-based therapies, and stem cell regulation.

His extensive work has produced several novel products and innovative technologies to benefit medical patients, which have come about through multi-national research collaborations, the generation of 700+ papers and 50+ patents, and the founding of three start-up companies.

Among these innovations, the engineering of shear-thinning materials termed Gel Embolic Materials (GEM) developed for the embolization of blood vessels is of particular significance. The GEM technology is an up-and-coming technology for replacing the current gold standard in embolization using metallic coils. Based on this technology, Dr. Khademhosseini co-founded Obsidio Medical (http://obsidiomed.com/) to pursue clinical applications of this invention. Due to its semi-solid nature, the GEM can be placed locally and customized for each person. Different than other embolic materials that can require time to form an obstruction to blood flow, the GEM technology quickly fills the targeted vasculature and instantly creates a barrier. The GEM technology was cleared by the U.S. Food and Drug Administration, and the Boston Scientific Corporation acquired Obsidio to enhance its interventional oncology and embolization portfolio.

Dr. Khademhosseini has a long history of achievements, starting with his early years as the recipient of the Presidential Early Career Award for Scientists and Engineers, the highest honor given by the US government for early-career investigators. Since then, he has received over 70 major national and international awards. This award is Khademhosseini's third from the Society, following his 2017 Clemson Award and the 2011 Young Investigator Award. It was formally presented to him during the Society for Biomaterials Annual Meeting and Exposition 2023.

The SFB is a multidisciplinary society of academic, healthcare, governmental, and business professionals dedicated to promoting advancements in all aspects of biomaterial science, education, and professional standards to enhance human health and quality of life.

About the Terasaki Institute for Biomedical Innovation

The Terasaki Institute for Biomedical Innovation is accelerating the pace of translational research by supporting the worlds leading scientists with an open, entrepreneurial environment for bioengineering new materials, biological models, and advanced technologies to address critical challenges to the health of the planet and its people. The Institutes worldwide collaborations with academic, clinical, and entrepreneurial partners provide a rich foundation for translating innovations to the real world.

Contact:Stewart HanPresidentTerasaki Institute for Biomedical Innovationshan@terasaki.org

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TIBI Director and CEO Ali Khademhosseini receives Technology ... - EurekAlert

Growing Prevalence and Recurrence of Rheumatoid Arthritis to … – Digital Journal

PRESS RELEASE

Published April 20, 2023

The global rheumatoid arthritis stem cell therapy market account for a share of USD 23.42 Billion in 2022. The market is anticipated to surpass the valuation of USD 33.30 Billion by end of the forecast period i.e. 2032. The rheumatoid arthritis stem cell market is expected to grow with a CAGR of 4.5 %.

Rheumatoid arthritis is a chronic inflammatory disorder in which the bodys immune system attacks its own joints. It is a progressive and disabling disease, which can cause a lot of pain and deformity of the joints if not managed properly. The symptoms of rheumatoid arthritis include joint stiffness, swelling, fatigue, and joint pain. Stem cell therapy is one of the most promising treatments for rheumatoid arthritis as it has the potential to repair and regenerate damaged tissue.

Download Sample Copy of This Report:https://www.factmr.com/connectus/sample?flag=S&rep_id=1001?AS

Market Trends

Eminent Players

Regional Insights

Geographically, the global rheumatoid arthritis stem cell therapy market can be segmented into viz. North America, Latin America, Europe, Asia-Pacific excluding Japan (APEJ), Japan, and the Middle East and Africa (MEA). North America is expected to be the dominant region in the global rheumatoid arthritis stem cell therapy market, owing to the presence of various key players.

The rheumatoid arthritis stem cell therapy market in Asia Pacific excluding Japan is expected to grow at a significant CAGR due to the expansion of product offerings by key players. Europe is expected to have the second large share in the global rheumatoid arthritis stem cell therapy market throughout the forecast period.

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In-Depth Assessment on Key Segments

Tentatively, the global rheumatoid arthritis stem cell therapy market can be segmented on the basis of treatment type, application, end-user, and geography.

Key Questions Covered in the Rheumatoid arthritis stem cell therapy Report

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Cancer survivor gives $10 million to speed translational research … – EurekAlert

image:Dr. Marie and Vijay Goradia view more

Credit: Courtesy of Vijay Goradia

HOUSTON Vijay Goradia, a Houston-based businessman, philanthropist, and cancer survivor, has donated $10 million to The University of Texas MD Anderson Cancer Center to speed translational research and clinical trials. An initial allocation of $3.5 million will fund the institutions clinical trial of a CD70-targeted chimeric antigen receptor (CAR) natural killer (NK) cell therapy for patients with renal cell carcinoma (RCC), developed by Katy Rezvani, M.D., Ph.D., professor of Stem Cell Transplantation and Cellular Therapy.

We are focused on supporting the innovation and commercialization of groundbreaking research, said Goradia, who also serves as a member of the MD Anderson Cancer Center Board of Visitors Executive Committee. There is so much impactful work happening at MD Anderson. Our first step was to fund Dr. Rezvanis incredible work in CAR NK cells. She has had some success in treating blood cancers, but now she is working on finding treatments for solid tumors, including renal cancer. We felt this was something worth supporting and we are very excited. We are very hopeful.

Goradia, a kidney cancer survivor, and his wife, Marie, established the Vijay and Marie Goradia Cancer Fund at MD Anderson through this transformational gift. Their hope is to not only expedite innovation coming from MD Anderson but to invest in the continued success of such research. The remainder of the gift will support additional therapeutics across the research enterprise.

The generosity of the Goradia family will be felt by cancer patients and their families for decades to come, said Peter WT Pisters, M.D., president of MD Anderson. Future translational and therapeutic discoveries that will be supported by this fund, including the CD70 CAR NK trial, will not only expand our efforts in targeting the disease, but also may generate financial benefits to support promising research in the years ahead.

A true game changerRenal cell carcinoma is the most common form of kidney cancer. Each year, RCC accounts for approximately 79,000 new cancer cases and 14,000 deaths in the U.S, according to the National Institutes of Health (NIH). While initial treatment with surgery for localized tumors can be curative, about 30% of patients develop metastatic disease, which currently is considered incurable because most of these tumors resist available chemotherapies.

Rezvani has led extensive translational research at MD Anderson to develop and expand the power of NK cells. These tumor-destroying immune cells serve as a first defense against malignancy, but cancer cells can make themselves invisible. By genetically modifying the NK cells with CARs, which are special receptors designed to bind to specific proteins on the surface of cancer cells, the NK cells can better find and eliminate target cancer cells. Rezvanis laboratory then multiplies the number of CAR NK cells, growing them in large numbers before infusing them into patients.

This research has the potential to become a viable treatment in a fairly short period of time, said Rezvani. I am grateful to Vijay and the entire Goradia family for their support and generosity as well as their belief in the science. This is the right place, the right time, and the right opportunity for such a transformational investment that may have such a grand impact in so many patients lives.

The clinical trial will be a Phase I/II study that will test the safety, feasibility, persistence, and antitumor activity of off-the-shelf cord blood-derived CAR NK cells targeting CD70 and armored with IL-15 for patients with RCC. While this study is directed specifically at RCC, CD70 also is present in other malignancies, including mesothelioma, lung, and bladder cancers, suggesting there may be potential applications in other cancer types.

MD Anderson has long been a leader in cancer research, diagnosis and treatment and we are inspired by the institutions efforts to bring cutting-edge research and innovation to patients in need, said Sapphira Goradia, executive director of the Vijay and Marie Goradia Charitable Foundation. The Marie and Vijay Goradia Charitable Foundation is dedicated to expanding access to quality health care and we hope this investment will support the acceleration of more effective and affordable cancer treatments.

Inspiring innovationPhilanthropy has been ingrained in Goradias DNA. From his humble beginnings in Mumbai, India, Goradia has made a concerted effort to help others. And he is not stopping anytime soon.

We are all hoping for the same end result, Making Cancer History, he said. And that cannot happen without meaningful research being done by very smart, talented, committed people. Our hope is that MD Anderson remains at the forefront by attracting very capable and talented people to come and join its already very capable research faculty.

Goradia said this cannot be accomplished by one, 10 or even 100 philanthropists. It is going to take more.

We have the format. My hope is that other donors who are interested in funding groundbreaking research and innovation may want to replicate this whole idea, he said. We can do this over and over again. Together, lets end cancer.

- 30 -

About MD AndersonThe University of Texas MD Anderson Cancer Center in Houston ranks as one of the world's most respected centers focused on cancer patient care, research, education and prevention. The institutions sole mission is to end cancer for patients and their families around the world. MD Anderson is one of only 53 comprehensive cancer centers designated by the National Cancer Institute (NCI). MDAnderson is No. 1 for cancer in U.S. News & World Reports Best Hospitals rankings and has been named one of the nations top two hospitals for cancer since the rankings began in 1990. MDAnderson receives a cancer center support grant from the NCI of the National Institutes of Health (P30 CA016672).

2023 The University of Texas MD Anderson Cancer Center

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Stem cell transplants found to extend survival by 4 years in ALS |… – ALS News Today

Transplants using mesenchymal stromal cells or MSCs a type of stem cell found in bone marrow delivered into the spinal canal can significantly extend survival in people with amyotrophic lateral sclerosis (ALS) compared with what would be expected based on their clinical characteristics, a new analysis of trials from the early 2000s found.

In two Phase 1 clinical trials that tested such stem cell transplants, the median time patients lived without requiring permanent ventilatory support was about 118 months, or nearly 10 years about four years longer than the roughly 70 months of predicted survival time pooled study data show.

At the time of the analysis, four patients (about 20% of participants) were alive, including one with a predicted survival of 91 months (about 7.5 years) who was alive at 303 months without needing a breathing or feeding tube. In other words, the patient was alive more than 20 years later.

The current study represents the first very long-term analysis of survival as an effect of MSC focal transplantation in the central nervous system [brain and spinal cord] of ALS patients, demonstrating that MSC transplantation could potentially slow down ALS progression and improve survival, the researchers wrote.

The study, Effect of mesenchymal stromal cell transplantation on long-term survival in amyotrophic lateral sclerosis, was published inCytotherapy.

ALS is a rare neurodegenerative disease marked by the progressive loss of motor neurons, the nerve cells that control voluntary movements. There is no cure to date, and few effective treatments are available to slow disease progression.

MSCs, also called mesenchymal stem cells, are a form of stem cells that can give rise to several connective tissue cells, such as fat cells and blood vessel cells.

These cells also can produce a range of signaling molecules that modulate immune responses and have tissue-protective and regenerative properties. As such, their use is considered a potentially promising approach for treating ALS.

The safety and feasibility of transplanting MSCs into the spinal canal has been established in ALS clinical trials. But few studies have reported the long-term effects of these cells in patients.

To address that, and eventually support a Phase 2/3 trial, researchers in Italy examined the long-term outcomes of ALS patients treated with MSCs in clinical trials conducted in the early 2000s. Specifically, the two Phase 1 clinical trials reviewed here were conducted by the team in 2002 and 2006.

Nineteen patients were treated in these studies, with nine involved in the first trial and 10 in the second.

For each patient, the team calculated survival defined as time to permanent non-invasive ventilation, breathing tube, or death using the European Network to Cure ALS (ENCALS) model. This tool predicts survival based on certain clinical characteristics, including age at disease onset, lung function, any delay in diagnosis, the rate of disease progression, and the presence of certain ALS-related mutations.

The expected survival for each patient based on this model was then compared with the individuals actual survival time.

The results showed MSC treatment significantly extended the patients lives compared with what would be expected based on their clinical characteristics. While the ENCALS model estimated a median survival of 70.8 months, patients actually lived for 118.8 months on average four more years than estimated.

The current study represents the first very long-term analysis of survival as an effect of MSC focal transplantation in the central nervous system [brain and spinal cord] of ALS patients, demonstrating that MSC transplantation could potentially slow down ALS progression and improve survival.

Of the total 19 patients, 13 (68%) had a longer survival time than expected. One patient had been classified as having a short survival and transitioned to intermediate, and three were intermediate and ended up having a long-to-very-long survival. The other nine were classified as very long survival, but still ended up living for more months than expected.

The survival extension was particularly evident in a group of patients with slow disease progression, 80% of whom lived longer than expected. Among those with fast progression, only 55% had longer survival than estimated; the other 45% lived less time than expected.

At the time of the analysis, four patients were still alive, including one who started noninvasive ventilation 35 months (nearly three years) after treatment and one who is still living without the need for ventilatory support. The other two patients have required a tracheostomy a procedure in which a tube is fitted through a hole in the neck to help with breathing.

Together, these results show a potential for stem cell transplants with MSCs to slow disease progression and improve survival in ALS patients, providing new insights for planning the next generation of efficacy MSCs clinical trials in ALS, the researchers wrote.

The team noted that stem cell transplantations role in ALS still needs further understanding. But the researchers hypothesized that the observed clinical effects could be primarily based on modifying the extra-motor neuron environment, such as through the release of trophic factor and the modulation of neuroinflammation.

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Identification of tunnels connecting neurons in the developing brain – Science Daily

Over a hundred years after the discovery of the neuron by neuroanatomist Santiago Ramn y Cajal, scientists continue to deepen their knowledge of the brain and its development. In a publication in Science Advances on April 5, a team from the Institut Pasteur and the CNRS, in collaboration with Harvard University, revealed novel insights into how cells in the outer layers of the brain interact immediately after birth during formation of the cerebellum, the brain region towards the back of the skull. The scientists demonstrated a novel type of connection between neural precursor cells via nanotubes, even before the formation of synapses, the conventional junctions between neurons.

In 2009, Chiara Zurzolo's team (Membrane Traffic and Pathogenesis Unit at the Institut Pasteur) identified a novel mechanism for direct communication between neuronal cells in culture via nanoscopic tunnels, known as tunneling nanotubes. These are involved in the spread of various toxic proteins that accumulate in the brain during neurodegenerative diseases. Nanotubes may therefore be a suitable target for the treatment of these diseases or cancers, where they are also present.

In this new study, the researchers discovered nanoscopic tunnels that connect precursor cells in the brain, more specifically the cerebellum -- an area that develops after birth and is important for making postural adjustments to maintain balance -- as they mature into neurons. These tunnels, although similar in size, vary in shape from one to another: some contain branches while others don't, some are enveloped by the cells they connect while others are exposed to their local environment. The authors believe these intercellular connections (ICs) may enable the exchange of molecules that help pre-neuronal cells physically migrate across various layers and reach their final destination as the brain develops.

Intriguingly, ICs share anatomical similarities with bridges formed when cells finish dividing. "ICs could derive from cellular division but persist during cell migration, so this study could shed light on the mechanisms allowing coordination between cell division and migration implicated in brain development. On the other hand, ICs established between cells post mitotically could allow direct exchange between cells beyond the usual synaptic connections, representing a revolution in our understanding of brain connectivity. We show that there are not only synapses allowing communication between cells in the brain, there are also nanotubes," says Dr. Zurzolo, senior author and head of the Membrane Traffic and Pathogenesis Unit (Institut Pasteur/CNRS).

To achieve these discoveries, the researchers used a three-dimensional (3D) electron microscopy method and brain cells from mouse models to study how the brain regions communicate between each other. Very high resolution neural network maps could thus be reconstructed. The 3D cerebellum volume produced and used for the study contains over 2,000 cells. "If you really want to understand how cells behave in a three-dimensional environment, and map the location and distribution of these tunnels, you have to reconstruct an entire ecosystem of the brain, which requires extraordinary effort with twenty or so people involved over 4 years," said the article's first author Diego Cordero.

To meet the challenges of working with the wide range of cell types the brain contains, the authors used an AI tool to automatically distinguish cortical layers. Furthermore, they developed an open-source program called CellWalker to characterize morphological features of 3D segments. The tissue block was reconstructed from brain section images. This program being made freely available will enable scientists to quickly and easily analyze the complex anatomical information embedded in these types of microscope images.

The next step will be to identify the biological function of these cellular tunnels to understand their role in the development of the central nervous system and in other brain regions, and their function in communication between brain cells in neurodegenerative diseases and cancers.The computational tools developed will be made available to other research teams around the globe.

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Identification of tunnels connecting neurons in the developing brain - Science Daily

Professor Neil Hanley to become new Head of the College of … – University of Birmingham

The University of Birmingham has appointed Professor Neil Hanley as Pro-Vice-Chancellor and Head of the College of Medical and Dental Sciences

The University of Birmingham has appointed Professor Neil Hanley as Pro-Vice-Chancellor and Head of the College of Medical and Dental Sciences.

Professor Hanley, who is currently Vice-Dean (Research and Innovation) in the Faculty of Biology, Medicine and Health at the University of Manchester and an Honorary Consultant Endocrinologist at Manchester Royal Infirmary, will take up his new role from 1 September 2023.

Professor Adam Tickell, Vice-Chancellor of the University of Birmingham, said:

I am delighted to announce that we have found the next leader of our College of Medical and Dental Sciences. In Neil, we have an excellent clinician, educator, and researcher with an enviable CV, who will bring energy and experience to this important role.

Professor Hanley holds a BSc in Pharmacology, an MBChB from the University of Edinburgh, and a PhD in Molecular Genetics from Newcastle University. He was first made full professor in 2006 and is a past president of the Association of Physicians of Great Britain and Ireland. He has been at The University of Manchester since 2008, becoming Director of Research and Innovation at Manchester University NHS Foundation Trust in 2016, and Vice-Dean (Research and Innovation) in 2020.

There is a real sense of ambition at the University, making it an honour and a very exciting time to be joining the mission to change lives for the better through education and research.

Professor Hanley said:

The University of Birmingham has a strong track record for improving the health of people locally and across the world through new discoveries, treatments, and training the next generation of healthcare practitioners. There is a real sense of ambition at the University, making it an honour and a very exciting time to be joining the mission to change lives for the better through education and research.

An expert in early human development and stem cell biology, Professor Hanley has held doctoral, intermediate, and senior clinical fellowships from NIHR or Wellcome, and additional funding as lead investigator from MRC, BBSRC and EPSRC, among others. Over the last decade, this has led to outputs in several Nature journals, eLife, and PNAS. His current partnership as chief investigator with Innovate UK and industry leaders has translated his collaborative discovery science into integrated novel diagnostics for the early detection of liver disease.

Professor Hanley has a passion for education and training, having established an academy in Manchester for those embarking on their own research careers. He also directs the Wellcome-funded PhD programme for healthcare professionals across the universities of Leeds, Manchester, Newcastle and Sheffield, and led the future vision of NIHRs 2016 national review of training.

The University of Birminghams College of Medical and Dental Sciences is shaping the future of health and medicine through the provision of innovative education and exceptional research, both delivered by world-leading academics. Earlier this year, the University of Birmingham rose 30 places in the QS World Subject Rankings for Life Sciences and Medicine and is now ranked at number 62.

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Professor Neil Hanley to become new Head of the College of ... - University of Birmingham