Stem Cell Clinic

Discussing the Future Role of Stem Cell Transplant in Multiple … – Targeted Oncology

Surbhi Sidana, MD

Assistant Professor

Department of Medicine

Division of Blood and Marrow Transplantation & Cellular Therapy

Stanford University School of Medicine

Stanford, CA

DISCUSSION QUESTIONS

SURBHI SIDANA, MD: Im a transplanter, so of course Im biased. What do you think about ASCT based on the results of the DETERMINATION study? Progression-free survival [PFS] was better [but] we dont see an overall survival [OS] difference.1 Did that change how you practice? Do you think it favors doing ASCT early [or shows that since] theres no difference in OS, we should not do the transplant?

ASHKAN LASHKARI, MD: As we incorporate MRD [minimal residual disease] assessment or diagnostic evaluations into our management of our patients with multiple myeloma, we might have a better way of determining whether we need to [transplant] or not. Thats a question that may be answered with the incorporation of MRD, at least in more clinical trials.

SIDANA: You bring up a great point, which is if youre MRD negative after induction, do you need a transplant? I dont think any of these trials answer that question because they did not randomly assign patients who were MRD negative versus MRD positive to ASCT versus no ASCT. I think we need to do these trials in the future.

Some of these trials are starting to happen in small phase 2 trial format. Currently, at least in the IFM 2009 trial [NCT01191060], even if you were MRD negative post hocthey were never randomly assigned based on MRD negativitythe MRD negativity did appear to be somewhat deeper and more sustained with the transplant.2 Those patients had a slightly better PFS. Whether that will hold true for DETERMINATION remains to be seen. I think what the transplant did was make it more sustained, at least in the IFM 2009 trial with 1 year of lenalidomide maintenance, versus no [transplant].

ANDY JANG, MD: I think the DETERMINATION trial is probably dampening the enthusiasm for ASCT for a couple of reasons. If I have a brand-new patient today whom I put on quadruplet therapies, I probably can [avoid transplant] for at least 7 years. Then you have CAR [chimeric antigen receptor] T-cell therapy, which gives you a very high overall response rate [above] 90%.3 The complete response rate is very high. Then you have other targets that are coming and CELMoDS [cereblon E3 ligase modulators]. Based on what I see right now and how effective the quadruplet therapy is, the transplant is probably going to get less emphasized.

SIDANA: Some of our standard-risk patients can get to [7 years on first-line therapy], but a lot of our high-risk patients [will not]. Do you change your practice patterns based on whether they have standard risk versus high risk?

JANG: Yes, of course. Butyou buy them so much [time]. Five years from now, the standard of care for multiple myeloma is definitely going to change. There may be a whole slew of other therapies. Because frontline therapy nowadays is so effectiveIm looking at the transplant value in general and looking at whats coming and whats already here. The CAR T-cell therapy data and even the bispecific antibody data are excellent in triple- or even penta-refractory patients.

SIDANA: Even though I am a transplanter, I hope one day ASCT will go away because every few years you want newer therapies that are less toxic to take over for older therapies. I think of it with a standpoint [where] were still not curing anybody, so lets use all of our treatments, ASCT included. But I hope one day that there is a treatment that can give us similar PFS and hopefully its less toxic and ASCT does go away.

SAM YEH, MD: I like the ASCT arm [in DETERMINATION] because I feel like patients do much better on maintenance than going back on treatment. You get [almost] 20 months extra PFS on the transplant arm based on DETERMINATION.4 Those [nearly] 2 years can give patients a good quality of life versus [if] the disease comes back, and they have lytic bone fractures and quality-of-life issues. There is going to be more toxicity going back on triplets or quadruplets versus when they go on ASCT, then they have a longer PFS and their quality of life may be better.

JANG: If you look at CAR T-cell therapy, it is also one-and-done, the patient has fairly good quality of life, and youre not giving them melphalan. The question is, do you go CAR T-cell therapy or ASCT; they are both one-and-done therapies. But with the transplant, you have to put them on maintenance. So you can argue the other way, [that CAR T-cell therapy is] one-and-done and gives patients a good quality of life.

YEH: CAR T-cell therapy is not indicated in that setting. Its much later.

JANG: That is why I said there is going to be less emphasis on the transplant [in the future] because a CAR T-cell therapy is one-and-done and the patient doesnt have to be on lenalidomide, you dont have to be on dexamethasone, and thats a tremendous selling point for the patient.

SIDANA: Both of you have good points. CAR T-cell therapy is one-and-done, but the indications are completely different. Right now, its fifth line for CAR T-cell therapy, first or second line for ASCT.5

JANG: Thats [true in this] moment in time. We know its not going to [require] 4 lines of therapy [in the future]. Its just because the trial was done that way. When Im going to refer CAR T-cell therapy, Im not going to wait for fourth-line therapies. Im going to start referring them when they have triple or quadruple failures. But right now, I understand the label. Looking atthe future of myeloma therapies, its not going to [require] a fourth-line therapy [before] you refer to CAR T-cell therapy. Its going to change.

SIDANA: I hope its a one-and-done but in all the trials that were designed in early lines, were adding maintenance to the CAR T-cell therapy. Theres a trial coming comparing ASCT to CAR T-cell therapy [CARTITUDE-6; NCT05257083] and theres going to be [lenalidomide] maintenance in the CAR T-cell therapy arm. Im hoping with CAR T-cell therapy we can continue it because patients love the treatment-free interval. They tell me thats the best few months of their life, but because patients still relapse, we are [acting like] ASCT used to be, with no maintenance. But then we added maintenance.

I see whats coming down the pipeline and worry that were going to add maintenance to [CAR T-cell therapy], too, but perhaps not for everybody. We will find out in the future if we need to do that for everybody.

References:

1. Richardson PG, Jacobus SJ, Weller EA, et al. Triplet therapy, transplantation, and maintenance until progression in myeloma.N Engl J Med. 2022;387(2):132-147. doi:10.1056/NEJMoa2204925

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

3. Berdeja JG, Madduri D, Usmani SZ, et al. Ciltacabtagene autoleucel, a B-cell maturation antigen-directed chimeric antigen receptor T-cell therapy in patients with relapsed or refractory multiple myeloma (CARTITUDE-1): a phase 1b/2 open-label study. Lancet. 2021;398(10297):314-324. doi:10.1016/S0140-6736(21)00933-8

4. Richardson PG, Jacobus SJ, Weller EA, et al. Triplet therapy, transplantation, and maintenance until progression in myeloma.N Engl J Med. 2022;387(2):132-147. doi:10.1056/NEJMoa2204925

5. NCCN. Clinical practice guidelines in oncology. Multiple myeloma, version 3.2023. Accessed March 30, 2023. https://bit.ly/2T0mDYS

Continued here:
Discussing the Future Role of Stem Cell Transplant in Multiple ... - Targeted Oncology

Stem Cell Junk Yards Reveal a New Clue About Aging – WIRED

Robert Signer sees himself as an auto mechanic for human cells. The professor of regenerative medicine at UC San Diego is intrigued by the elusive secrets of the stem cells in our blood. These are a class of rejuvenating entities that replenish supplies of red and white blood cells and platelets. Their job is to help keep our bodies healthy, but as we age their performance dips. When they fail, it can lead to blood cancers, anemia, clotting issues, and immune problems. Signers job is to understand why, and he thinks the answer has to do with how they handle their garbage.

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Our cells assemble around 20,000 specific proteins that allow us to do everything from digesting dairy to killing tumors. But the process isnt perfect. When cells mess up, they wind up with whats essentially junk: proteins with missing, extra, or incorrect amino acids in their chains. These can settle into unexpected shapes and malfunctionor worse. They start to stick together, and they form these aggregates, Signer says. Aggregates gum up the machine. Misfolded proteins can actually be toxic. (Researchers have linked Alzheimers disease to gummed-up clumps of protein.)

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Most mature blood and immune cells live fast and die hard. They thrive by churning out protein after protein, and mistakes are part of the deal. But life moves slowly for a stem cell. Even modest increases in protein production can be very catastrophic, says Signer. If they make a mistake, waste leads to worse performance, which leads to more waste. So stem cells trying to survive for the long haul must manage their waste like pros.

A healthy stem cell keeps tight control over proteins production and destruction, and this ability to maintain what researchers call protein homeostasis is what fades with age. We think that if we can jump in and prevent this from happening, or improve the ability of stem cells to maintain this protein homeostasis, then we might be able to prevent the decline in stem cell function and the diseases that are associated with those changes, says Signer.

Biologists have long known that stem cells run a tight ship, but not how. So writing in the journal Cell Stem Cell in March, Signers team reported an up-close look at what happens inside the stem cells of young and old mice. (You can't be a good mechanic if you've never looked under the hood, Signer says.)

What they learned was surprising. Biologists had previously assumed that stem cells stay tidy by breaking down waste as fast as it arises, reducing junk proteins into amino acid fodder they can reuse immediately. But Signers group found that blood's stem cells actually squirrel away their misfolded waste and only recycle it when they need it. Scientists had seen this behavior before, but they thought that cells did it in rare cases, when under extreme stress. Signer now believes that healthy stem cells do this as a baselineits a way of pacing themselves in order to maintain control. The mouse data showed that this sophisticated process breaks down with age.

This revelation offers insight into why we age and what critical cellular machinery we must keep running to combat age-related diseases, according to Maria Carolina Florian, a stem cell biologist at the Catalan Institution for Research and Advanced Studies who was not involved in the work. To Florian, it suggests the possibility of creating drugs that can maintain this control for stem cells. It looks particularly important, she says, because of this possibility to be targetedto be able to reverse aging.

Signers lab studied blood stem cells taken from mouse bone marrow. Doctoral researcher Bernadette Chua first extracted marrow from young mice (ages 6 to 12 weeks) and isolated several types of cellsstem cells as well as blood and immune cellsto observe them during an early stage of development. Then, using fluorescent molecules that stick to specific components of the cell, she snooped on each to see how it was managing its trash.

Cells use proteasomes, protein complexes containing enzymes that immediately chew up their misfolded proteins. But Signers lab had previously found that, like neural stem cells, blood stem cells in young mice dont rely on proteasomes very much. In this new experiment, Chua and Signer found that instead of breaking down misfolded proteins right away, stem cells swept them out of the way, collecting them into piles, like mini junk yards. Later, they disintegrated them with a different protein complex called an aggresome. We believe that by storing these misfolded proteins in one place, they're basically holding onto those resources for when they need them, Signer says. Collecting piles of waste may let cells control the pace of their recycling and, as a result, avoid living too fast or too slow.

Yet when Chua next examined marrow from 2-year-old mice, she found a shocking breakdown in this waste management system. Older mice lost their ability to form aggresomes almost entirely at least 70 percent of the stem cells in young mice do it, but only 5 percent in old mice. Instead, old mice swapped to using more proteasomes, a move Signer likens to slapping a spare tire onto an aging car. That was definitely a surprise, Signer says.

This change in waste control machinery is bad news for stem cells. Mice that were genetically engineered to not cache their trash had four times fewer surviving stem cells in their bone marrow in old age. It suggests that those cells are aging, and expiring, faster than they were before.

This distinction between enzymes, wonky as it sounds, could prove crucial for efforts to harness stem cells as anti-aging therapies because it runs counter to previous assumptions. Let's say that you want to engineer a stem cell for regenerative medicine, says Dan Jarosz, a systems biologist from Stanford University who was not involved in the work. Before reading this, I might have thought that a really good thing to do would be to amp up the proteasome activity.

The idea that young, healthy stem cells control the pace of their lives by collecting debris into a storage center, instead of consuming it immediately, is very cool, he continues. This suggests that we need a much more nuanced understanding of how protein quality control functions in aging.

Why older stem cells change their behavior remains an open question. Florian suspects it has something to do with how cells change shape as they age. A healthy cell is typically lopsided, as its contents are sectioned into distinct compartmentsthis asymmetric shape is referred to as being polarized. But stem cells lose their polarity with age, and this affects their ability to shuttle waste to their storage center.

Florians lab is developing drugs that maintain cell polarization. Last year, she reported rejuvenating mouse stem cells with a treatment that tamps down the activity of an overactive enzyme that messes with cell polarity. When transplanted into immunocompromised mice, the stem cell treatment extended their median lifespans by over 12 weeks, or 10 percent. It has a very profound effect on the blood, she says. Basically, you rejuvenate the blood of the mice, and they leave healthier and longer. (Florian serves on the advisory board for rejuvenation start-up Mogling Bio.)

For his part, Signer imagines a drug that maintains the equipment that stem cells use to compost malformed proteinshe doesnt yet know what that would be, but the new experiment gives researchers an idea of where to look. Figuring out that stem cells trash collection system falls apart as the cells age is important, he says, because pinpointing what goes wrong with age gives us an idea of how to target future fixes.

Signer and Florian admit that any drug meant to keep cells young and active carries some cancer risk. Older cells activate genes that prevent tumors and suppress stem cells. Its possible that helping stem cells survive in old age will help cancer cells do the same.

But I also think that there is an alternative possibility happening in parallel, Signer says. Maybe helping stem cells clear their trash slowly and steadily prevents the cascade of effects that lead to problems like cancer, he says: If we can prevent some of those changes, we might be able to prevent multiple types of age-related diseases.

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Stem Cell Junk Yards Reveal a New Clue About Aging - WIRED

Novo’s latest deal targets cell therapy for diabetes, obesity – BioPharma Dive

Dive Brief:

Novo, while historically not big on buyouts, has turned to acquisitions over the past couple years to expand its slate of technologies and research programs. In late 2021, it agreed to spend north of $3 billion on Dicerna Pharmaceuticals, a company specialized in so-called RNA interference. And in 2022, it picked up Forma Therapeutics in a $1 billion deal that provided an experimental drug for sickle cell disease in late-stage clinical testing.

Yet, Novos core business still revolves around diabetes and, more recently, obesity. Aided by newer drugs like Rybelsus, Wegovy and, especially, Ozempic, the company last year recorded a 26% increase in net sales and a 34% increase in gross sales.

The new collaboration with Aspect indicates that Novo sees further room to grow in its core areas. Per deal terms, Aspect is eligible to receive as much as $650 million for each resulting product, provided it hits certain developmental, regulatory, commercial and sales goals. Additionally, the biotech would get tiered royalties on future sales of any products.

The deal also further entrenches Novo in the field of cellular medicine. The company established a California-based manufacturing site dedicated to stem cell therapies in 2018. And currently, its researching ways that cell therapies could be used to treat illnesses like Parkinsons disease, chronic heart failure and Type 1 diabetes.

Partnering with Aspect adds an important component to our strategy to develop comprehensive cell therapy products, Jacob Sten Petersen, Novos head of cell therapy research and development, said in a statement.

Novo isnt alone in its pursuits, however. Just last month, Vertex Pharmaceuticals announced that it would be licensing gene editing technology from CRISPR Therapeutics to develop therapies for Type 1 diabetes. The two companies had already been collaborating on a gene editing therapy for sickle cell and another blood disorder, and CRISPR had been working with the biotech Viacyte on its own Type 1 diabetes cell therapy program.

Vertex bought Viacyte last year for $320 million, hoping that the biotechs tools would help accelerate the development of VX-880, an experimental, stem-cell-derived therapy targeting Type 1 diabetes, which Vertex got through its $950 million acquisition of Semma Therapeutics in 2019.

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Novo's latest deal targets cell therapy for diabetes, obesity - BioPharma Dive

Modified stem cell therapy extends survival in infantile Batten… – Batten Disease News

A treatment using stem cells modified to produce PPT1 the missing enzyme in infantile Batten disease prevented symptoms from developing and substantially extended survival in a mouse model of the disease, a study reports.

It also prolonged survival and slowed disease progression when given to mice that had already developed symptoms, a remarkable result, the researchers wrote in An innovative hematopoietic stem cell gene therapy approach benefits CLN1 disease in the mouse model, which was published in EMBO Molecular Medicine.

The findings support further studies on this therapeutic strategy, they said.

Infantile Batten, also called CLN1 disease, is a severe type of Batten diseasecaused by mutations in the PPT1 gene,which provides instructions for making an enzyme that helps break down complex molecules in cells.

Without a working version of the PPT1 enzyme, molecules called lipofuscins build up to toxic levels and damage cells, especially those in the brain, promoting neuroinflammation.

Within the brain, immune cells called microglia are involved in neuroinflammation and in clearing molecular waste. They may therefore be invaluable targets for treating disorders like Batten disease that feature the toxic accumulation of molecular waste.

Hematopoietic stem and progenitor cells (HSPCs) are stem cells that normally live in the bone marrow and give rise to blood cells, including most immune cells. Under the right conditions, theyre able to grow into microglia-like cells, suggesting their transplantation could restore efficient waste clearance.

Scientists in the U.S. and Italy tested the effects of delivering HSPCs from healthy mice directly into the bloodstream of a mouse model of infantile Batten. Before treatment, the mice were given a myeloablative regime, consisting of chemotherapy to destroy their existing HSPCs.

Compared with untreated mice, those treated with healthy HSPCs showed markedly less severe disease, as measured with a new scale devised by the researchers, results showed. They also lived significantly longer relative to untreated animals (median, 248 vs. 228 days).

These data represent the first demonstration that [HSPC] transplantation could be beneficial in CLN1 disease, wrote the researchers, who then tested the effects of HSPCs they genetically modified to produce higher than normal levels of the PPT1 enzyme. The new treatment led to further improvements in survival and reductions in measures of disease severity.

By day 260 (about eight months), all the untreated mice had either died or reached conditions where it was considered inhumane to keep them alive. But 77% of mice treated with the modified HSPCs were still alive. Most of them (68%) were also still alive after a year.

Analyses of the treated animals that died suggested the cause of death was usually not related to Batten. Instead, most died due to complications associated with the myeloablative regime.

The team then showed that administering the modified stem cells directly into the brain had comparable effects to the intravenous route. The best results were obtained when both routes were combined, however. Animals given the genetically modified HSPCs via both forms of administration had lower disease severity scores than those treated through only one.

At about eight months after treatment, 86% of mice given both routes were still alive and more than three-quarters (76%) lived past a year without displaying any Batten-like symptoms.

The combinatorial transplant strategy could achieve greater clinical benefit than the individual approaches, the researchers wrote, noting that also tended to result in less variable outcomes.

Tissue analyses of the mices brain and spinal cord suggested an increase in PPT1 levels with the treatment, in many cases to levels higher than those seen in healthy mice. Again, the highest enzyme levels were achieved with the combination strategy.

Data also suggested the stem cell treatment reduced neuroinflammation.

In all these experiments, treatment was administered early in life, before the mice displayed Batten-like symptoms. In subsequent tests, the researchers gave the modified HSPCs only when the mice were about 4 months old, when symptoms start becoming apparent in this model.

We decided to treat with the [genetically engineered HSPCs] at onset of symptoms to verify whether we could achieve therapeutic benefit also in a stage of the disease when damage has already accumulated in the brain and spinal cord, the researchers wrote.

The treatment diminished disease severity and extended survival also in this context 80% of treated mice versus none of the untreated mice were alive at eight months.

The combined administration approach of modified HSPCs results in the most robust therapeutic benefit among the tested approaches on both presymptomatic [before symptoms] as well as symptomatic animals, since it resulted in complete abrogation of the disease in a clinically relevant mouse model and determined a long-lasting and thorough prevention of symptoms, the researchers wrote.

Notably, this same approach could uniquely benefit adult symptomatic animals, a finding of utmost importance for translation purposes, the researchers wrote, adding the clinical translatability of our strategy is further supported by the favorable safety profile we showed here.

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Modified stem cell therapy extends survival in infantile Batten... - Batten Disease News

Regenerative Medicine Market Investments, Share and Revenue Analysis | Latest InsightAce Report – Yahoo Finance

InsightAce Analytic Pvt. LTd.

The global Regenerative Medicine market is estimated to reach over USD 183.08 billion by 2031, exhibiting a CAGR of 15.02% during the forecast period

Jersey City, NJ, April 12, 2023 (GLOBE NEWSWIRE) -- InsightAce Analytic Pvt. Ltd. announces the release of a market assessment report on the "GlobalRegenerative Medicine Market Size, Share & Trends Analysis Report By Product (Therapeutics, Primary cell-based therapeutics, Stem Cell & Progenitor Cell-based therapeutics), By Therapeutic Category (Dermatology, Musculoskeletal, Immunology & Inflammation, and Oncology)- Market Outlook And Industry Analysis 2031"

The global Regenerative Medicine market is estimated to reach over USD 183.08 billion by 2031, exhibiting a CAGR of 15.02% during the forecast period.

In recent year, it has been determined that regenerative therapies can uniquely change the underlying pathological processes. Trial-stage regenerative medicines offer promising treatments for particular chronic diseases with unmet medical needs. Novartis announced the release of T-ChargeTM in December 2021, a next-generation CAR-T platform that would be used for cutting-edge investigational CAR-T cell treatments.

Free PDF Report Brochure @https://www.insightaceanalytic.com/request-sample/1687

The development of gene-based treatment, which uses targeted DNA delivery as a drug to combat numerous illnesses, results from significant effects in molecular therapeutics. With the restoration of gene function, gene therapy holds great promise for treating cancer and type 1 and type 2 diabetes. Gene-based medicines treat patients with conditions such as cancer, oncology, infectious diseases, cardiovascular disorders, monogenic diseases, genetic disorders, ophthalmological indications, and central nervous system illnesses. These elements have helped the market for regenerative medicine expand.

Recent Developments:

In April 2022, Obecabatagene autoleucel, a CD19-directed autologous chimeric antigen receptor T therapy being investigated in the ongoing FELIX Phase 2 study of leukaemia, has been given the Regenerative Medicine Advanced Therapy designation by the U.S. Food and Drug Administration (FDA). This was announced by Autolus Therapeutics plc.

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List of Prominent Players in the Regenerative Medicine Market:

AstraZeneca plc;

F. Hoffmann-La Roche Ltd.;

Integra Lifesciences Corp.;

Astellas Pharma, Inc.;

Cook Biotech, Inc.;

Bayer AG;

Pfizer, Inc.;

Merck KGaA;

Abbott;

Vericel Corp.;

Novartis AG;

GlaxoSmithKline (GSK);

Baxter International, Inc.;

Boehringer Ingelheim;

Amgen, Inc.;

Cesca Therapeutics, Inc.;

U.S. Stem Cell, Inc.;

Bristol-Myers Squibb;

Eli Lilly and Company;

NuVasive, Inc.;

Organogenesis, Inc.;

MiMedx Group, Inc.;

Takara Bio, Inc.;

Osiris Therapeutics, Inc.;

Corline Biomedical AB

Get Customized Report @https://www.insightaceanalytic.com/customisation/1687

Regenerative Medicine Market Report Scope:

Report Attribute

Specifications

Market size value in 2022

USD 52.66 Bn

Revenue forecast in 2031

USD 183.08 Bn

Growth rate CAGR

CAGR of 15.02 % from 2023 to 2031

Quantitative units

Representation of revenue in US$ Million, and CAGR from 2023 to 2031

Historic Year

2019 to 2022

Forecast Year

2023-2031

Report coverage

The forecast of revenue, the position of the company, the competitive market statistics, growth prospects, and trends

Segments covered

Product And Therapeutic Category

Regional scope

North America; Europe; Asia Pacific; Latin America; Middle East & Africa

Country scope

U.S.; Canada; U.K.; Germany; China; India; Japan; Brazil; Mexico; The UK; France; Italy; Spain; China; Japan; India; South Korea; Southeast Asia; South Korea; Southeast Asia

Market Dynamics:

Drivers- The ability of adult stem cells to proliferate or self-renew forever and to develop all the cell kinds of the organ from which they originate has propelled research into these cells, with the potential to regenerate the complete organ from a few cells. No embryo must be destroyed in order to produce adult stem cells. Furthermore, medical research on stem cells has been thoroughly examined and attracted much attention. ExCellThera Inc. and Ossium Health recently announced a partnership to explore and advance opportunities to use adult stem cells from deceased donors from Ossium Health's first-ever bone marrow bank in combination with ExCellThera's ECT-001 cell expansion and rejuvenation technology. This collaboration will take place in April 2021. These kinds of developments are anticipated to accelerate market expansion.

Challenges:The market for regenerative medicine is projected to be hampered by a lack of information and moral considerations surrounding the usage of embryonic stem cells for research and development. Since cell therapy is a crucial component of regenerative medicine, it has a significant impact on the market growth rate. One of the leading market inhibitors may be the high cost of investment, which might be followed by problems with assay sensitivity, robustness, and reproducibility; the challenge of culture/propagation; and finally, the challenge of handling.

Regional Trends:Due to the presence of big players, the rapid advancement of technology, significant investments in stem cell and oncology research, and the presence of major players, North America is predicted to have the largest revenue share. The largest market in North America is the United States. In the U.S., numerous stem cell therapies are increasingly being used to treat a growing number of ailments like cancer and diabetes. According to the Heart Disease & Stroke Statistics Fact Sheet 2020, congenital heart abnormalities are predicted to affect at least 40,000 infants annually in the United States.

Enquiry Before Buying @https://www.insightaceanalytic.com/enquiry-before-buying/1687

Segmentation of Regenerative Medicine Market-

By Product-

Therapeutics

Primary cell-based therapeutics

Dermatology

Musculoskeletal

Surgical

Dental

Others

Stem Cell & Progenitor Cell-based therapeutics

Autologous

Allogenic

Others

Cell-based Immunotherapies

Gene Therapies

Tools

Banks

Services

By Therapeutic Category-

By Region-

North America-

Europe-

Germany

The UK

France

Italy

Spain

Rest of Europe

Asia-Pacific-

China

Japan

India

South Korea

South East Asia

Rest of Asia Pacific

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Regenerative Medicine Market Investments, Share and Revenue Analysis | Latest InsightAce Report - Yahoo Finance

FDA Grants FTD to Off-the-Shelf CAR T in R/R Multiple Myeloma – Cancer Network

The FDA has granted fast track designation to the investigational allogenic CAR T-cell therapy product CB-011 for the treatment of patients with relapsed or refractory multiple myeloma, according to a press release from Caribou Biosciences, Inc.1

"Fast track designation for CB-011 allows us instrumental interactions with the FDA as we progress our clinical development and regulatory plans for CB-011, according to the manufacturers of CB-011.

The FDA originally cleared an investigational new drug application for CB-011 in this patient population in November 2022, permitting investigators to advance with their evaluation of the agent in the phase 1 CaMMouflage trial (NCT05722418).2

According to findings from a poster session presented at the 2023 Tandem Meeting, CB-011 demonstrated potent anti-tumor activity in vitro and enhanced survival in multiple myeloma xenograft models.3 Additionally, investigators observed no adverse safety signals associated with treatment in vitro.

Fast track designation for CB-011 allows us instrumental interactions with the FDA as we progress our clinical development and regulatory plans for CB-011, Syed Rizvi, MD, chief medical officer of Caribou, said in the press release. Our goal is to develop CB-011 as a readily available off-the-shelf treatment option for patients with relapsed or refractory multiple myeloma to overcome the need for apheresis or bridging therapy, variable quality and long manufacturing timelines, manufacturing failures, or the inability to bear the burden of treatments that require frequent dosing over several months.

CB-011 is an allogeneic CAR T-cell therapy targeting BCMA that was engineered with Cas12a chRDNA technology. Investigators believe its design allows it to enable anti-tumor activity through an immune cloaking strategy that removes the B2M protein and inserts a B2MHLA-E fusion protein.

Investigators of the open-label, multi-center phase 1 CaMMouflage trial are assessing CB-011 as treatment for adult patients with relapsed or refractory multiple myeloma. Part A will include a dose escalation of CB-011 in ascending doses based on a traditional 3+3 design. In part B, up to 30 patients will receive CB-011 at the maximum tolerated dose or recommended phase 2 dose determined in part A.

The primary end point of part A is the number of patients who experience dose limiting toxicities. In part B, the primary end point is the overall response rate based on International Myeloma Working Group (IMWG) criteria.

Patients 18 years and older with a documented diagnosis of relapsed or refractory multiple myeloma and measurable disease per IMWG criteria are eligible to enroll on the trial. Additional inclusion criteria include receipt of at least 3 prior lines of therapy including a proteasome inhibitor, an immunomodulatory drug, and an anti-CD38 monoclonal antibody; having an ECOG performance status of 0 or 1; and adequate hematologic, hepatic, renal, pulmonary, and cardiac function.

Patients who received prior treatment with a CAR T-cell therapy or autologous stem cell transplant within 6 weeks prior to undergoing lymphodepletion are not eligible for enrollment. Patients are also unsuitable for enrollment if they have received allogeneic stem cell transplant within 6 months prior to lymphodepletion, known active or prior central nervous system involvement, stroke or seizure within 6 months of study entry, or are seropositive or have a history of human immunodeficiency virus.

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FDA Grants FTD to Off-the-Shelf CAR T in R/R Multiple Myeloma - Cancer Network

NSF CAREER Award: Rowan researcher takes aim at critical barrier … – Rowan Today

In humans, mesenchymal stem cells (MSCs) live in bone marrow and body fat. Their identity can be defined by their ability to divide into more stem cells, adapt to new environments and differentiate into specialized cells that produce tissues like bone, ligament, cartilage and fat.

When studied in the lab, these unique properties decline over time. The inability to produce large quantities of MSCs with their identity intact is a critical barrier that limits the use of these cells for reproducible research and stem cell therapies.

Sebastin L. Vega, Ph.D., an assistant professor of biomedical engineering at Rowan University, has received a five-year, $512,890 CAREER Award from the National Science Foundation (NSF) to investigate why stem cells lose their identity outside the body. The Faculty Early Career Development (CAREER) Program offers the independent federal agencys most prestigious awards to early-career faculty. Vega has dual appointments in the Henry M. Rowan College of Engineering and the Rowan-Virtua School of Translational Biomedical Engineering & Sciences, where he is also a founding faculty member of the Rowan-Virtua Institute of Regenerative Medicine and Transplantation.

Using stem cells, scaffolds and environments to grow functional tissuea replacement of a persons own tissueis the future, with treatment strategies beginning to be available to patients now, said Mark Byrne, Ph.D., founding dean of the Rowan-Virtua School of Translational Biomedical Engineering & Sciences. Dr. Vegas research is critical to improving and scaling stem cell therapies, which lead to much better outcomes for patients. At Rowan, we actively recruit top researchers like Dr. Vega, who have high potential to lead next-generation treatment strategies.

This award recognizes Dr. Vegas exceptional research and the promise of future breakthroughs associated with stem cell manufacturing, said Giuseppe Palmese, Ph.D., dean of the Henry M. Rowan College of Engineering. We are delighted to have Dr. Vega represent our college in this way.

Funding from Vegas CAREER Award will be used to develop materials with independently controllable properties to understand the role of stiffness, adhesion and the interactions between cells on MSC identity.

MSCs are typically grown in plastic petri dishes, which are stiffer than any tissue in the human body, Vega said. MSCs remember their culture history and prolonged exposure to unnatural materials like plastic negatively impacts their ability to divide, adapt and differentiate.

Instead of relying on traditional petri dishes for culturing cells, Vega is designing a replacementsofter, bioactive materials meant to preserve the cells identities even after countless cell divisions in the lab. The goal is to create materials that can be used to culture large populations of high-quality stem cells for research and clinical use.

Another unwanted side effect of prolonged exposure of these cells to plastic is a loss of cell-material communication, which is necessary for taking on the properties of specialized cell types theyre meant to become. The team will develop new culture environments with prescribed properties to significantly improve MSC adaptation to biomaterials used in regenerative medicine.

Despite being discovered over half a century ago and having become one of the most-studied cell types in regenerative medicine, FDA-approved MSC therapies do not exist, Vega said. Our proposed research will help overcome cell manufacturing challenges seen in many MSC clinical trials and I am hopeful that our studies will enable the clinical use of MSCs in tissue engineering and to treat degenerative diseases.

This award will also integrate research with ongoing educational outreach activities. Vega is the founder of BEAM (BioEngineering and Me), a program that exposes high school students to biomedical engineering. Through this program, Glassboro High School students learn about biomedical engineering through research talks and interactive activities in research labs. To reach more students, Vega developed the annual BEAM Summer Program, a free, two-week summer program open to any high school student nationwide. To date, 74 high school students from nine states have participated.

Vega also runs the RISER (Research Immersion in biomedical Science & Engineering at Rowan) Summer Program, a six-week research experience for high school students to join biomedical engineering or biomedical science labs, which culminates with student presentations at the annual RISER Summer Symposium.

CAREER awards from the National Science Foundation support early career researcher educators in attaining their long-term goals, said NSF program director Wendy Crone. Dr. Vega's project on regulating stem cell proliferation, mechanical adaptation, and differentiation is poised to make advancements in the ability to preserve stem cell identity while also engaging high school and undergraduate students with biotechnology research.

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NSF CAREER Award: Rowan researcher takes aim at critical barrier ... - Rowan Today

Germany Cell and Gene Therapy Market Focused Insights Report 2023: A $3.44 Billion Market by 2028 – Competitive Landscape, Pipeline Analysis, Clinical…

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Dublin, April 12, 2023 (GLOBE NEWSWIRE) -- The "Germany Cell and Gene Therapy Market - Focused Insights 2023-2028" report has been added to ResearchAndMarkets.com's offering.

The Germany cell and gene therapy market was valued at $0.34 billion in 2022 and is projected to reach at $3.44 billion in 2028, with a compound annual growth rate (CAGR) of 40.15%, during the study period.

The study considers a detailed scenario of the present cell and gene therapy market and its market dynamics for 2023-2028 in Germany. It covers a detailed overview of several market growth enablers, restraints, and trends. The report offers both the demand and supply aspects of the market. It profiles and examines leading companies and other prominent players operating in the market.

This report offers market size & forecast data for cell & gene therapy market in Germany. The report covers commercial cell & gene therapy products, such as conventional cell therapies, CAR-T cell therapies, gene therapies, cell-based immunotherapies, and oncolytic virus therapies.

Cell therapies sourced from mesenchymal stem cells, cell-based immune modulation therapies, and cell/tissue-based products derived from patients' blood are also covered in the report. Tissue-engineered products and tissue grafts/scaffolds with synthetic mechanical structures are excluded from the scope of the report.

This report provides a comprehensive and current market scenario of the Germany cell & gene therapy market, including the Germany cell & gene therapy market size, anticipated market forecast, relevant market segmentations, and industry trends.

MARKET DEFINITION

Cell and gene therapies are part of advanced therapy medicinal products (ATMPs). ATMPs are medicines that are developed based on genes, tissues and cells.

The introduction of many novel ATMPs in recent years, especially cell and gene therapies changed the treatment dynamics of many complex and challenging diseases like cancer and genetic disorders.

Story continues

Government, as well as industrial and academic financing and collaboration among small and large biopharmaceutical companies, offer significant growth to the market in the coming years.

KEY HIGHLIGHTS

Among all commercial cell & gene therapies, Zolgensma is the first and only product to achieve the blockbuster drug tag. Zolgensma generated a revenue of $1.37 billion in 2022 and has the potential to reach around $5.00 billion by 2026.

Drug developers are prioritizing to develop and commercialize CAR-T cell-based gene therapies. Globally, more than 1,000 clinical trials are being conducted on CAR T-cell therapies, of which atleast 500 clinical trials are for cancer alone. Kymriah (Novartis), Yescarta (Gilead Sciences), Tecartus (Gilead Sciences), Breyanzi (Bristol Myers Squibb), Abecma (Bristol Myers Squibb) and Carvykti (Janssen Biotech / Legend Biotech) are the commercial CAR-T therapies available in the market.

In 2022, Germany was the first-largest market in Europe, followed by France and the UK. The market is majorly driven by an increase in target patient population, regulatory body support, and higher access to healthcare due to well-established healthcare infrastructure.

The European Medicines Agency (EMA) has approved more than nineteen cell and gene therapy drugs, but the new product pipeline has approximately 193 investigational therapies, with more than half of these in Phase 2 clinical trials. Oncology and rare diseases continue to be the top areas targeted by gene therapies from preclinical through pre-registration.

Approximately 261 clinical trials are under investigation for various cell and gene therapies in Germany. This increase could be because of an improvement in funding for cell and gene therapy. The industry sponsored trials are continued to dominate with a share of 88.51%.

Novartis, Gilead Sciences, Spark Therapeutics, Amgen, Orchard Therapeutics, Bristol-Myers Squibb, BioMarin, PTC Therapeutics, and Organogenesis are the leading players in the Europe cell and gene therapy market. The market offers tremendous growth opportunities for existing and future/emerging players because of the presence of a large pool of target patient populations with chronic diseases such as cancer, genetic and rare diseases, and other complex disorders.

PRODUCT SEGMENTATION & FORECAST

Product type

Application type

Oncology

Genetic Disorders

Dermatology

Musculoskeletal

Others

End-user type

Hospitals

Cancer Care Centers

Others

MARKET STRUCTURE

PRICING & REIMBURSEMENT SCENARIO

Pricing, Reimbursement and Market Access

Contract Manufacturing Organizations

Regulatory Landscape and Policies

For more information about this report visit https://www.researchandmarkets.com/r/ppr7fp

About ResearchAndMarkets.comResearchAndMarkets.com is the world's leading source for international market research reports and market data. We provide you with the latest data on international and regional markets, key industries, the top companies, new products and the latest trends.

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How can stem cells treat diabetes? The Upcoming – The Upcoming

How can stem cells treat diabetes?

Diabetes is a persistent medical condition that impairs the bodys capacity to regulate blood sugar levels. Although various treatments and medications are available to mitigate the symptoms and avoid complications, the disease has no known cure. It is caused by an autoimmune response that targets and destroys pancreatic (beta) cells, which are responsible for producing insulin.

Since the body fails to maintain blood sugar levels on its own, people with diabetes require insulin injections. This is currently the only option for patients suffering from type 1 diabetes.

According to research, stem cell therapy has been shown to restore the function of insulin-producing beta cells in the pancreas potentially, thus helping treat this problem. In this article, this will be discussed.

Stem cell therapy for diabetes

The cell-based approach is a promising treatment for diabetes as it can help in the regeneration of damaged pancreatic cells that produce insulin. This factor makes stem cell therapy particularly important for patients with type 1 diabetes, considering their permanent dependency on insulin injections to control blood glucose levels. Lets look at the types of the disorder and how stem cells are helpful in minimising their harmful effects.

What is type 1 diabetes?

This autoimmune disease is characterised by the immune system attacking and destroying insulin-producing beta cells in the pancreas. Insulin controls the blood glycaemic index by allowing glucose to enter the bodys cells and use it for energy and storage. Insufficient amounts of insulin result in a rise in glucose levels in the bloodstream, leading to high blood sugar levels, which can cause various symptoms and complications.

The symptoms of the disorder include:

Type 1 diabetes usually develops in childhood or young adulthood but can occur at any age. It is often diagnosed through a blood test that measures blood sugar levels and the presence of certain autoantibodies such as:

Treatment for type 1 diabetes typically involves lifelong insulin therapy, monitoring blood sugar levels, and lifestyle modifications with a proper workout plan and dietary alterations.

What is type 2 diabetes?

Non-insulin-dependent diabetes, also known as diabetes type 2, is a chronic condition that affects how the body processes blood sugar (glucose). In type 2 variation, the body either doesnt produce enough insulin or becomes resistant to insulin, a hormone that regulates the blood glycemic index. After food consumption, the body converts carbohydrates into glucose, which is then transported into cells by insulin. However, in people with type 2 variation, cells in fat, muscles, and liver cannot take in enough glucose, leading to a buildup of it in the bloodstream.

Over time, high blood sugar levels can cause serious health problems, including damage to the heart, blood vessels, nerves, eyes, and kidneys. Some risk factors that may lead to the disorder include:

Old age is also one of the factors which may lead to the onset of type 2 diabetes.

Is there a cure for diabetes?

There is currently no known cure for this disease. Diabetes type 2 occurs by a combination of lifestyle and genetic factors, such as being overweight or inactive. People with type 2 can often manage their condition through a healthy diet and exercising regularly but may also need medications, insulin, or other treatments to help manage blood sugar levels.

While there is no cure for this disorder, ongoing research is focused on developing new treatments and therapies that can improve management and prevent complications. People suffering from this condition are advised to work with their healthcare providers to develop a personalised treatment plan that fits their individual needs and goals.

The International Diabetes Federation predicts that by 2045, nearly 700 million adults will have diabetes. The cause, specifically of type 1 diabetes (T1D), is not fully understood, but research suggests a combination of genetic, environmental, and viral factors may contribute to its development. Currently, the most widely used treatment for T1D is administering insulin externally, but it does not provide a cure for the disease.

The Role of Mesenchymal Stem Cells in the Treatment of Type 1 Diabetes, doi: 10.7759/cureus.27337.

Can stem cells help with diabetes?

A potential treatment proposed to cure the disorder is stem cell therapy. However, its effectiveness and safety are still being researched and debated in the scientific community, i.e., can stem cells cure diabetes?

Much work is being done to use stem cell for diabetes type 2 and metabolically programming them to create glucose-sensing, insulin-producing beta cells in the body. Some researchers are exploring the possibility of using stem cells as replacements for these, which would allow the body to produce insulin again.

The bodys immune system resulting in the destruction of insulin-producing cells in the pancreas causes type 1 diabetes. Scientists working on stem cells observed that they could help people with T1D better control their blood sugar levels. Several clinical trials are ongoing to study the safety and effectiveness of stem cell therapy for diabetes, with varying degrees of success.

We now understand stem cell transplants can succeed in treating diabetes for some. We discovered the immune signature predicting these outcomes either favourable or not which is the first step toward personalised medicine in type 1 diabetes. Understanding why it sometimes fails will allow us to design new treatment strategies for those less fortunate patients. Also, it is the first definitive proof that T1D can be cured.

Bart Roep, PhD, Professor of Diabetology.

Stem cells can be derived from various sources, including induced pluripotent, embryonic, and adult versions. However, each source has its advantages and limitations regarding availability, differentiation potential, and ethical considerations.

Benefits and disadvantages of stem cell therapy

Like any medical treatment, cell therapy for diabetes has both pros and cons. The disorder can cause several complications, including cardiovascular disease, neuropathy, and retinopathy. Stem cell therapy may reduce the risk of these complications by repairing and regenerating damaged cells in the body. Transplanted stem cells cures diabetes to some extent by potentially recreating insulin-producing pancreatic islets. It has the potential to manage the disease by regenerating damaged cells of the pancreas and thus restoring the bodys ability to produce and regulate insulin. It is a natural way of healing from the inside of the body.

Disadvantages of stem cell therapy for the disorder include its safety. There is a risk of complications, such as infection, and immune system reactions, in case the cell-based product was not properly prepared. In addition, stem cell therapy is an expensive treatment, and it may not be covered by health insurance and thus can be a significant barrier for some patients. The use of embryonic stem cells for therapy raises ethical concerns for some people. In this regard, adult mesenchymal stem cells are a better option for therapy.

Final thoughts

In conclusion, cell therapy has the potential to revolutionise the treatment of diabetes. Still, more research is needed to fully understand its benefits and risks and develop safe and effective protocols for its clinical application. It is essential to weigh stem cell therapys potential benefits and disadvantages carefully and consult with a healthcare professional before making any decisions about treatment.

The editorial unit

The information contained in this article is for general informational purposes only and is not intended to be a substitute for professional medical advice, diagnosis, or treatment. Always seek the advice of a qualified healthcare provider with any questions you may have regarding a medical condition. Never disregard professional medical advice or delay in seeking it because of something you have read.

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How can stem cells treat diabetes? The Upcoming - The Upcoming

Brave girl, 4, rings cancer-free bell after ending three years of treatment – Yahoo News UK

Watch: Brave girl, four, rings bell after ending three years of cancer treatment

This is the moment a brave four-year-old girl rang the cancer-free bell after ending three years of treatment.

A video showed Phoebe Ashfield receiving a round of applause and cheers from nurses.

She was diagnosed with acute lymphoblastic leukaemia at seven months old in 2019 and relapsed three times during her treatment.

Her mother Emma Wyke, 30, from Dudley, West Midlands, recorded Phoebe ringing the bell following her ordeal.

Read more: Spot on womans finger turned out to be false widow spider bite

Mother Emma Wyke and Phoebe (centre) alongside family members. (Caters)

Wyke said: "At one stage of her treatment I was told that she isn't going to pull through and to prepare myself for the worst.

"It's devastating and heartbreaking to watch my baby girl go through all this.

Read more: Boy, 9, nearly dies after falling eight foot off scooter

"When Phoebe was first diagnosed, I thought they had the wrong child, and it simply cannot be my daughter, as all she had was a cold and chest infection and it can't be cancer.

"When she finally rang the bell it's a mixture of emotions you want to cry happy tears, but you still have that worry of wondering if it's going to come back. You don't truly know how to feel."

Phoebe as a baby during her treatment. (Caters)

Phoebe started her treatment with chemotherapy but it wasn't strong enough to keep the cancer away, Wyke said.

She relapsed in June 2019 and had to go for a Car-T therapy, which involved taking her stem cells and modifying them to fight the cancer.

She relapsed again in September of that year.

Her mother said further treatment was successful until January 2020 when she relapsed for the third time and needed a stem cell transplant, and at this moment "time was against us".

Wyke added: "If there is one thing to come of this is that to register to become a stem cell donor through (non-profit) DKMS and (charity) Anthony Nolan because without these selfless people, adults and children, my daughter wouldn't be here to tell this tale.

If you can please register you could save someone's life."

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Brave girl, 4, rings cancer-free bell after ending three years of treatment - Yahoo News UK