Category Archives: Cell Medicine

Businesses, Hospitals, Municipal Buildings, Museums, Schools, Landmarks To Illuminate In Red On World Sickle Cell Awareness Day To Show Support of Sickle Cell Warriors, Raise Awareness of Sickle Cell Disease, Advocate For A Universal Cure

Fraternal Organization, Global Healthcare Company, NFL Team, Building Owners Management Association Join Campaign

PHILADELPHIA, June 13, 2024 /PRNewswire/ -- A collaborative of 46 nonprofit, community-based organizations and medical providers in the Northeast U.S. is set to Shine the Light on Sickle Cellon June 19, World Sickle Cell Awareness Day. This collaborative initiativewhich includes the illuminationin red of businesses, hospitals, municipal buildings, museums, schools, and other landmarksaims to unite individuals and communities in support of sickle cell warriors, raise awareness of Sickle Cell Disease (SCD) and advocate for a universal cure.Fraternal organization Phi Beta Sigma Fraternity, Inc., global healthcare company Novo Nordisk Inc., the Baltimore Ravens, and Building Owners Management Association-Philadelphia will also join the campaign that is supported by the federal Health Resources and Services Administration (HRSA) as part of the Sickle Cell Disease Treatment Demonstration Project.

"Now in its sixth year, Shine the Light on Sickle Cell is a powerful testament to what we can achieve when communities, healthcare providers, and advocates unite," said Johns Hopkins University School of Medicine Professor Dr. Rosalyn Stewart. "Our goal is to make Sickle Cell Disease as common a topic as COVID or HIV and to inspire action towards a future where Sickle Cell Disease is better understood, better treated, and ultimately cured."

SCD is a genetic blood disorder that disproportionately affects individuals of African descent yet remains widely misunderstood and underrepresented in public discourse. With over 100,000 people in the U.S. affected by SCD and millions more carrying the Sickle Cell Trait, the need for improved access to care and research advancements is critical. Individuals living with SCD often face numerous health complications, including stroke, acute chest syndrome, and chronic organ damage, leading to a significantly reduced life expectancy compared to the general population and earning them the name of sickle cell warrior. Despite these challenges, there is currently no universal cure for SCD.

"I am deeply committed to shining the light on sickle cell and advocating for better care of people with the disease, a disease that causes severe episodic and chronic pain. Those with sickle cell disease frequently report poor interpersonal treatment within health-care settings," said Dr. Sophie Lanzkron, Director of the Division of Hematology at Thomas Jefferson University. "Eliminating the discrimination in health-care settings is imperative as research has proven its correlation with greater pain severity, stress, depression, and sleep issues."

Shine the Light on Sickle Cell primarily operates in the Northeast region, encompassing New England, the mid-Atlantic, Virginia, West Virginia, the U.S. Virgin Islands, and Puerto Rico. Shine the Light on Sickle Cell's hallmark is the illumination of landmark structures and buildings in red the emblematic color of the blood cell associated with the genetic disorder and a symbol of the urgency, passion, and unwavering determination in the fight against this disease in the U.S. and around the world on and around June 19.

Major landmarks that will Shine the Light on Sickle Cell this year include the Franklin Institute, the Hospital of the University of Pennsylvania, and Boathouse Row (Philadelphia); Novo Nordisk headquarters (Plainsboro Township, New Jersey); the Baltimore Ravens' M&T Bank Stadium and the Johns Hopkins University Dome (Baltimore); the Rhode Island State House (Providence); and University of Maryland's Capital Region Medical Center, Bowie Health Center, and Laurel Medical Center (Washington, D.C. metropolitan area).

Zemoria Brandon is a longtime sickle cell advocate whose husband passed away from the disease in 1998. Now in her role as chair of the Shine the Light on Sickle Cell Steering Committee and administrator/social worker with Sickle Cell Disease Association of America, Philadelphia/Delaware Valley Chapter, Ms. Brandon said: "By illuminating our communities in red, we are symbolizing the urgency and passion needed to address the challenges of Sickle Cell Disease. Although Shine the Light on Sickle Cell began six years ago as an initiative in the Northeastern United States, it grew into a campaign, and now it has become a movement. It is uplifting to have seen participation from 29 states across the country and 21 countries around the world."

Organizations in the collaborative are set to engage their community in various activities, including sickle celebrations, candlelight vigils, blood drives, awareness walks, wearing red, posting photos and videos on social media, and more. Shine the Light on Sickle Cell events (click for details and registration information) include:

Join the conversation about Shine the Light on Sickle Cell using #shinethelightonsicklecell2024.

About Shine the Light on Sickle Cell

Shine the Light on Sickle Cell is an annual community awareness campaign to celebrate World Sickle Cell Awareness Day on June 19 as proclaimed by the United Nations in 2008 and to advocate for a universal cure. Shine the Light on Sickle Cell is led by a collaborative of 46 nonprofit, community-based organizations and medical providers in the Northeast United States, SiNERGe (Sickle Cell Improvement in the Northeast Region through education), whose aim is to increase awareness of Sickle Cell Disease and advocate for treatments and better outcomes for individuals with the disease. Learn more at Shine the Light on Sickle Cell.

Media ContactShinePR for Shine the Light on Sickle Cell[emailprotected]

Participating organizations and provider organizations include (by state):

Connecticut

Citizens for Quality Sickle Cell Care* University of Connecticut

Delaware

Christiana Care Sickle Cell Association of Delaware

District of Columbia

Faces of Our Children Sickle Cell Association of the National Capital Area Inc.

Maine

Maine Medical Center

Maryland

Adult and Pediatric to Adult Sickle Cell Clinic, Johns Hopkins University Armstead-Barnhill Foundation for Sickle Cell Anemia Association for the Prevention of Sickle Cell Anemia Inc., Harford and Cecil Counties and the Eastern Shore* Christopher Gipson Sickle Cell Moyamoya Foundation Eastern Shore of Maryland Sickle Cell Association Johns Hopkins University Maryland Sickle Cell Disease Association* Project Spirit Sickle Cell Sally's Sunshine Foundation Sickle Cell Coalition of Maryland William E. Proudford Sickle Cell Fund Inc.

Massachusetts

Boston University Massachusetts General Hospital Massachusetts Sickle Cell Disease Association*

New Hampshire

Dartmouth Hitchcock Medical Center

New Jersey

Donna T. Darrien Memorial Foundation for Sickle Cell Newark Beth Israel Medical Center Sickle Cell Association of New Jersey*@

New York

Candice's Sickle Cell Fund^ Children's Hospital of Monefiore New York Sickle Cell Advisory Network NYC Health + Hospitals New York Sickle Cell Advocacy Network (formerly Queens Sickle Cell Anemia Network)* Sickle Cell Advocates of Rochester^ Sickle Cell Awareness Foundation Corp International Sickle Cell Thalassemia Patients Network*@ Sickle Cell Warriors of Buffalo^ Westchester Sickle Cell Outreach

Pennsylvania

Children's Sickle Cell Foundation Inc.*#@ Crescent Foundation@ Sickle Cell Disease Association of America, Philadelphia/Delaware Valley Chapter*#@ South Central Pennsylvania Sickle Cell Council*# Hospital of the University of Pennsylvania

Puerto Rico

Anemia Falciforme Sickle Cell Disease en Puerto Rico

Rhode Island

Rhode Island Hospital

Vermont

University of Vermont

Virginia

Life and Family Foundation Richmond (formerly Living with Sickle Cell RVA) Sickle Cell Association Inc.*

West Virginia

CAMC Institute for Academic Medicine

*Chapters of the Sickle Cell Disease Association of America (SCDAA)#Members of the Pennsylvania Sickle Cell Disease Providers Network (PASCDPN)^Affiliates of Sickle Cell Thalassemia Patients Network (SCTPN)@HRSA Newborn Screening Grantees

SOURCE Shine the Light on Sickle Cell

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46 NONPROFIT, COMMUNITY-BASED ORGANIZATIONS AND MEDICAL PROVIDERS IN THE NORTHEAST U.S. ... - PR Newswire

This article originally appeared on Targeted Oncology.

Patients with advanced renal cell carcinoma (RCC) who received nivolumab (Opdivo; Bristol Myers Squibb) plus cabozantinib (Cabometyx; Exelixis) in the first line achieved a longer treatment-free survival (TFS) vs sunitinib (Sutent; Pfizer), according to data from an analysis of the phase 3 CheckMate 9ER trial (NCT03141177) presented duringASCO 2024.1

CheckMate 9ER was an open-label study that enrolled patients with histologically confirmed untreated advanced or metastatic clear cell RCC who did not receive prior systemic therapy for RCC.

Over the 48-month period following random assignment, patients in the intention-to-treat (ITT) population who received the combination (n = 323) experienced a mean TFS of 7.0 months compared with 4.6 months (95% CI, 0.8-3.9) in the sunitinib arm (n = 328), for a difference of 2.4 months (95% CI, 0.8-3.9). Patients without a grade 2 or higher treatment-related adverse effect (TRAE) experienced a mean TFS of 3.0 months vs 2.3 months, respectively; those who experienced a grade 2 or higher TRAE experienced a mean TFS of 3.9 months vs 2.3 months. The 48-month mean overall survival (OS) was 35.1 months in the doublet arm vs 30.7 months in the sunitinib arm. The mean times on protocol treatment were 22.6 and 14.1 months, respectively.

Additionally, the 48-month OS rates were 49.2% in the nivolumab plus cabozantinib arm vs 40.2% in the sunitinib arm, and the 48-month TFS rates were 33.3% compared with 12.9%, respectively.

There are multiple immunotherapy-based combination treatments with similar efficacies approved for first-line treatment of advanced RCC. Therefore, its important to characterize patient survival time after treatment initiation, Charlene Mantia, MD, a medical oncologist at Dana-Farber Cancer Institute and instructor in medicine at Harvard Medical School in Boston, Massachusetts, said during a presentation of the findings. Over the 4-year period since first-line protocol therapy initiation, [patients treated with] nivolumab plus cabozantinib achieved a 1.5-times longer mean TFS vs sunitinib.

CheckMate 9ER was an open-label study that enrolled patients with histologically confirmed untreated advanced or metastatic clear cell RCC who did not receive prior systemic therapy for RCC. However, treatment with 1 prior adjuvant or neoadjuvant therapy was permitted for patients with completely resectable disease if the agent was not VEGF targeted and if recurrence occurred at least 6 months following treatment.2,3

Patients were randomly assigned 1:1 to receive intravenous nivolumab 240 mg every 2 weeks in combination with oral cabozantinib 40 mg daily or sunitinib 50 mg daily for 4 weeks in 6-week cycles. Treatment in both arms continued until disease progression or unacceptable toxicity, and the maximum duration of nivolumab therapy was 2 years.

The primary end point was progression-free survival by blinded independent central review per RECIST 1.1 criteria. Secondary end points included OS, objective response rate, and safety.

In the ITT population, the baseline characteristics were well balanced between the combination and sunitinib arms; the median (IQR) age was 62 (29-90) years vs 61 (28-86) years, respectively. Most patients in both arms were not from North America or Europe (51.1% vs 50.9%), had a Karnofsky performance status (KPS) of 90 or 100 (79.6% vs 73.5%), underwent prior nephrectomy (68.7.% vs 71.0%), and had tumors without sarcomatoid features (89.1% vs 87.1%).2

Additional findings from the TFS analysis showed that patients with International Metastatic Renal Cell Carcinoma Database Consortium (IMDC) favorable-risk disease in the combination (n = 74) and control (n = 72) arms achieved a 48-month mean TFS of 6.1 months vs 4.3 months, respectively, for a difference of 1.8 months (95% CI, 1.1 to 4.6). The mean OS during this time was 38.2 months in the nivolumab plus cabozantinib arm vs 38.6 months in the sunitinib arm. Patients spent a mean time of 25.2 months vs 19.9 months on protocol treatment, respectively.1

Further, patients with IMDC intermediate- or poor-risk disease in the combination (n = 249) and sunitinib (n = 256) arms achieved a 48-month mean TFS of 7.2 months vs 4.7 months, respectively, for a difference of 2.5 months (95% CI, 0.8-4.3). The 48-month mean OS was 34.2 months vs 28.5 months, respectively, and patients spent a mean time of 21.8 months in the combination arm vs 12.5 months in the sunitinib arm on protocol treatment.

Results from a TFS subgroup analysis showed that the benefit with nivolumab plus cabozantinib vs sunitinib was observed across prespecified subgroups. The largest differences in 48-month mean TFS in favor of the combination were reported among patients with a KPS of 80 or less (5.3 months; 95% CI, 2.1-8.5), IMDC poor-risk disease (4.0 months; 95% CI, 0.5-7.5), and a PD-L1 expression of at least 1% (3.5 months; 95% CI, 0.8-6.1).

Mean TFS differences were similarly longer for nivolumab plus cabozantinib vs sunitinib across most baseline subgroups analyzed, Mantia said in conclusion. TFS was more frequently observed after patients stopped therapy due to adverse effects, study drug toxicity, or prespecified other reasons with nivolumab plus cabozantinib vs sunitinib. We believe that future studies should continue to investigate the impact of having a defined treatment duration for patients. This partitioned OS analysis highlights important aspects of patient survival time that can be considered in making decisions when choosing a first-line treatment for patients.

References

1. Mantia C, Jegede O, Viray H, et al. Partitioned overall survival: Comprehensive analysis of survival states over 4 years in CheckMate 9ER comparing first-line nivolumab plus cabozantinib versus sunitinib in advanced renal cell carcinoma (aRCC). J Clin Oncol.2024;42(suppl 16):4507. doi:10.1200/JCO.2024.42.16_suppl.4507

2. Powles T, Burotto M, Escudier B, et al. Nivolumab plus cabozantinib versus sunitinib for first-line treatment of advanced renal cell carcinoma: extended follow-up from the phase III randomised CheckMate 9ER trial.ESMO Open. 2024;9(5):102994. doi:10.1016/j.esmoop.2024.102994

3. A study of nivolumab combined with cabozantinib compared to sunitinib in previously untreated advanced or metastatic renal cell carcinoma (CheckMate 9ER). ClinicalTrials.gov. Updated January 17, 2024. Accessed June 13, 2024. https://clinicaltrials.gov/study/NCT03141177

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Treatment-Free Survival Extended With Nivolumab/Cabozantinib in Advanced Renal Cell Carcinoma - AJMC.com Managed Markets Network

LOS ANGELES--(BUSINESS WIRE)-- Treating prostate cancer with immunotherapy is currently difficult to do. But preliminary results from a first in-human phase 1 trial using a chimeric antigen receptor (CAR) T cell therapy developed by researchers from City of Hope, one of the largest cancer research and treatment organizations in the United States, showed that patients with advanced prostate cancer had minimal side effects with the cellular immunotherapy and had promising therapeutic activity, according to a study published today in Nature Medicine.

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Saul Priceman, Ph.D., City of Hope associate professor, Department of Hematology & Hematopoietic Cell Transplantation, and Tanya Dorff, M.D., City of Hope section chief, Genitourinary Disease Program, and their teams worked together on the phase 1 clinical trial using a City of Hope-developed CAR T cell therapy for prostate cancer. (Photo: Business Wire)

The trial treated 14 prostate stem cell antigen (PSCA)-positive patients who had metastatic castration resistant prostate cancer (mCRPC), which had spread beyond the prostate and no longer responded to hormone treatment, using CAR T cell therapy. More than 34,000 men with this type of prostate cancer die each year in the United States.

Saul Priceman, Ph.D., City of Hope associate professor, Department of Hematology & Hematopoietic Cell Transplantation, and team developed CAR T cells that target prostate stem cell antigen (PSCA) found to be highly expressed in prostate cancer. The treatment took a patients immune cells known as T cells from the bloodstream, and reprogrammed the cells in a laboratory with a CAR to recognize and attack the PSCA protein on the surface of cancer cells. CAR T cells were then infused back into the patients system to destroy cancer cells.

Prostate cancer has been called an immune desert the tumor microenvironment is difficult to treat with immunotherapies because you dont get a lot of T cells inside the tumor, said Tanya Dorff, M.D., City of Hope section chief, Genitourinary Disease Program, and professor, Department of Medical Oncology & Therapeutics Research. It takes something really powerful to overcome that. Our study showed that City of Hopes CAR T cell therapy for prostate cancer could be a step closer to doing that.

Our trials preliminary major finding is that PSCA-directed CAR T cells may be effective against mCRPC, Priceman added. This opens up the opportunity to continue to develop this type of cellular immunotherapy for these patients, who currently have no other effective treatment options.

The trials goals were to examine the therapys safety and dose-limiting toxicities, or side effects that limit the amount of treatment that can be administered, as well as preliminary data on the treatments efficacy in patients.

The studys findings were:

One patient who had received several prior therapies responded well to the CAR T cell therapy. His PSA level decreased by 95% and cancer in his bones and soft tissue also declined. He experienced this positive response for approximately eight months.

The patients results were very encouraging, and we are deeply grateful for his participation in our study as well as other patients and their families, Dorff said. We want to continue with this therapy and increase the amount of CAR T cells, and continue to carefully monitor for any health problems, as we think this can improve the therapys effectiveness.

The phase 1b trial using the PSCA-CAR T cell therapy in combination with radiation to enhance anti-tumor activity aims to enroll up to 24 patients.

City of Hope, a recognized leader in CAR T cell therapies, has treated nearly 1,500 patients since its CAR T program started in the late 1990s. The institution continues to have one of the most comprehensive CAR T cell clinical research programs in the world it currently has about 70 ongoing CAR T clinical trials, which include 13 different solid tumor types. The trials use City of Hope-developed therapies and industry-sponsored products. A recent study published in Nature Medicine featured City of Hopes CAR T cell therapy for brain tumors.

City of Hope manufactured the CAR T cells in its own facility, the Cell Therapy Production Center on its Los Angeles campus.

The Prostate Cancer Foundation helped fund the trial.

About City of Hope

City of Hope's mission is to make hope a reality for all touched by cancer and diabetes. Founded in 1913, City of Hope has grown into one of the largest cancer research and treatment organizations in the U.S. and one of the leading research centers for diabetes and other life-threatening illnesses. City of Hope research has been the basis for numerous breakthrough cancer medicines, as well as human synthetic insulin and monoclonal antibodies. With an independent, National Cancer Institute-designated comprehensive cancer center at its core, City of Hope brings a uniquely integrated model to patients spanning cancer care, research and development, academics and training, and innovation initiatives. City of Hopes growing national system includes its Los Angeles campus, a network of clinical care locations across Southern California, a new cancer center in Orange County, California, and cancer treatment centers and outpatient facilities in the Atlanta, Chicago and Phoenix areas. City of Hopes affiliated group of organizations includes Translational Genomics Research Institute and AccessHopeTM. For more information about City of Hope, follow us on Facebook, X, YouTube, Instagram and LinkedIn.

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Saul Priceman, Ph.D., City of Hope associate professor, Department of Hematology & Hematopoietic Cell Transplantation, and Tanya Dorff, M.D., City of Hope section chief, Genitourinary Disease Program, and their teams worked together on the phase 1 clinical trial using a City of Hope-developed CAR T cell therapy for prostate cancer. (Photo: Business Wire)

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City of Hope CAR T Cell Therapy for Advanced Prostate Cancer Demonstrates Positive Results in Phase 1 Clinical Trial - BioSpace

One clinical-stage biotech firm is making arthritis injectable drug treatments not for humans, but for pets.

Irvine-based Cell Therapy Tools (CTT) aims to develop cell-based therapies for pain and inflammation, starting with osteoarthritis in dogs.

The company last month said that it has begun enrolling dogs with the degenerative joint disease in a three-month clinical study using its experimental drug product Chondroshield.

During osteoarthritis, theres cartilage under destruction because of biochemical processes happening in the joint, Chief Executive Fari Izadyar told the Business Journal.

Chondroshield is going to reverse that process.

Izadyar said that this upcoming pilot study is the first step toward gaining approval from the Food and Drug Administrations Center for Veterinary Medicine.

CTT plans to enroll 20 dogs in the study, which will be overseen by Chief Veterinary Medical Officer Robert Jeff Mayo in Washington.

Izadyar said the company is currently self-funded but is open to fundraising once CTT has more clinical data.

We are hoping that the closer we get to the finish line, we can go to the fundraising phase, he said.

Canine Arthritis?

Chondroshield is intended to be an off-the-shelf product that can be stored in the fridge until needed for use by a veterinarian.

The drugs formulation utilizes platelets. Mainly known for blood clotting, platelets also have wound-healing properties that help stimulate regeneration of tissue.

Existing cell therapies for canine osteoarthritis typically involve some sort of surgery to extract cells from the patients body.

The issue, however, is that production for these therapies is labor-intensive and the quality can be inconsistent, according to Izadyar.

Clinicians cannot verify how many platelets are in there or what is the growth factor composition, Izadyar said.

Chondroshield, on the other hand, is an allogenic cell-based therapy, which means the cells come from donors instead of the patient and doesnt require any blood collection.

Izadyar said there is an unmet need in the market for such a treatment with there being close to 80 million dogs owned per household in the U.S. and 20% of them suffer from osteoarthritis.

As part of the pilot study, CTT is also partnering with New York-based pet tech startup Maven and will use its smart collars to monitor pet activity.

It will give us objective data as to how movement and mobility of the animal improved or not after this study, Izadyar said.

Stem Cell Research

Izadyar got his veterinary degree nearly four decades ago from the University of Tehran.

As a practitioner, Izadyar said he saw animal patients with debilitating chronic diseases that couldnt be treated with traditional medical therapy.

It was frustrating. Izadyar said.

The experience pushed him to return to school and pursue a doctorate in reproductive biology at Utrecht University in the Netherlands where he became more involved in stem cell research.

I was fascinated by the powerful healing ability of cells, Izadyar said.

He left the Netherlands for the U.S. in 2004 and went on to be chief scientific officer at local biotech companies developing cell-based products for regeneration purposes, including Santa Ana-based PrimeGen Biotech LLC and its subsidiary VetCell Therapeutics that was created in 2015.

After working at these companies, Izadyar realized he wanted to make his own product and joined CTT in 2022.

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Cell Therapy Tools Studies Medication for Dogs in Pain - Orange County Business Journal

Cartesian Therapeutics investigational cell therapy Descartes-08 has been granted regenerative medicine advanced therapy (RMAT) designation by the U.S. Food and Drug Administration (FDA) for the treatment of myasthenia gravis (MG).

RMAT status is intended for experimental regenerative medicines to treat, modify, reverse, or cure a serious or life-threatening disease and which have shown preliminary clinical evidence of being able to address an unmet need for treating that disease.

Developers of RMAT-designated treatments receive all the incentives of fast track and breakthrough therapy designations, including early and intensive interactions with the FDA that can help hasten treatment development.

Descartes-08 has also been granted orphan drug designation by the FDA for MG, another status intended to speed the therapys development by offering regulatory support and financial incentives.

Receipt of RMAT designation underscores our belief that Descartes-08 could serve as a meaningful addition to the MG treatment landscape, Carsten Brunn, PhD, president and CEO of Cartesian, said in a company press release.

We look forward to working closely with the FDA to efficiently advance the development of Descartes-08 for this underserved population, Brunn added.

Meanwhile, Descartes-08 is being tested in MG patients in a Phase 2b clinical trial, called MG-001 (NCT04146051). Cartesian remains on track to announce top-line results from the study by mid-year.

MG, an autoimmune disease, is caused by self-reactive antibodies that attack healthy proteins residing at the site of communication between nerve and muscle cells. These antibodies are produced by certain types of immune B-cells.

Descartes-08 is designed to deplete the levels of these B-cells, thereby lowering the production of disease-driving antibodies and easing MG symptoms.

Immune T-cells have the means to kill off these B-cells, but they dont naturally know they should target them. With Descartes-08, T-cells are removed from a patients body and engineered in the lab to be equipped with a chimeric antigen receptor, or CAR, that specifically binds to a protein found on immune B-cells, called BCMA. When the modified T-cells are infused back into the patient, they are now equipped to specifically bind and destroy the harmful B-cells.

While the concept of CAR T-cell therapy for treating autoimmune diseases isnt new, Descartes-08 takes a unique approach to help avoid toxicity-related safety issues that are common with this type of therapy.

With most CAR T-cell therapies, the CAR is introduced by providing T-cells with DNA that encodes its production. While having the advantage of being a one-time treatment, this also requires that patients undergo chemotherapy to kill off existing immune cells before the treatment, leading to toxicity-related side effects.

Instead of delivering DNA, Descartes-08 delivers RNA, an intermediate molecule thats produced when converting the information in DNA to a working protein. While this RNA-based version requires repeat dosing, it also avoids the need for chemotherapy and is thought to have a better safety profile. That also means it can be administered as an outpatient procedure without hospital admission.

The MG-001 trial included Phase 1b, Phase 2a, and Phase 2b parts, all involving adults with generalized MG (gMG). After an initial Phase 1b dose-finding portion, 11 gMG patients were enrolled in the Phase 2a study. There, they were assigned to receive six into-the-vein infusions of Descartes-08 in various dosing schedules.

Across both the Phase 1b and Phase 2a parts, the treatment was well tolerated and not associated with side effects common with DNA-based CAR T-cell therapies, including neurotoxicity and cytokine release syndrome, both of which are serious types of immune responses.

Evidence of clinically meaningful improvements in measures of MG disease severity were observed among Phase 2a participants that persisted for months after the six-week treatment period ended in most patients. Levels of disease-driving antibodies in the bloodstream were also reduced.

In the ongoing Phase 2b study, up to 30 adults with gMG will be randomly assigned to receive six weekly doses of Descartes-08 or a placebo. The groups will then be switched, with those initially given Descartes-08 receiving a placebo and vice versa.

The main goal of this part of the trial is to compare the effects of Descartes-08 versus a placebo on a standard measure of patient-reported disease severity.

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FDA grants CAR T-cell therapy Descartes-08 its RMAT designation - Myasthenia Gravis News

A new analysis has revealed detailed information about genetic variation in brain cells that could open new avenues for the targeted treatment of diseases such as schizophrenia and Alzheimers disease.

The findings, reported May 23 in Science, were the result of a multi-institutional collaboration known as PsychENCODE, founded in 2015 by the National Institutes of Health, which seeks new understandings of genomic influences on neuropsychiatric disease. The study was published alongside related studies in Science, Science Advances, and Science Translational Medicine.

Previous research has established a strong link between a persons genetics and their likelihood of developing neuropsychiatric disease, saysMark Gerstein, the Albert L. Williams Professor of Biomedical Informatics at Yale School of Medicine and senior author of the new study.

The correlations between genetics and your susceptibility to disease are much higher for brain diseases than for cancer or heart disease, said Gerstein. If your parents have schizophrenia, youre much more likely to get it than you are to get heart disease if your parents have the disease. There is a very large heritability for these brain-related conditions.

Whats less clear, however, is how this genetic variation leads to disease.

We want to understand the mechanism, said Gerstein. What is that gene variantdoingin the brain?

For the new study, researchers set out to better understand the genetic variation across individual cell types in the brain. To do so, they performed several types of single-cell experiments on more than 2.8 million cells taken from the brains of 388 people, including healthy individuals and others with schizophrenia, bipolar disorder, autism spectrum disorder, post-traumatic stress disorder, and Alzheimers disease.

From that pool of cells, the researchers identified 28 different cell types. Then they examined gene expression and regulation within those cell types.

In one analysis, the researchers were able to link gene expression to variants in upstream regulatory regions, bits of genetic code situated before the gene in question that can increase or decrease the genes expression.

Thats useful because if you have a variant of interest, you can now link it to a gene, said Gerstein. And thats really powerful because it helps you interpret the variants. It helps you understand what effect theyre having in the brain. And because we looked across cell types, our data also allow you to connect that variant to an individual cell type of action.

The researchers also assessed how particular genes, such as those associated with neurotransmitters, varied across individuals and cell types, finding variability was usually higher across cell types than across individuals. This pattern was even stronger for genes that code for proteins targeted for drug treatment.

And thats generally good for a drug, Gerstein said. It means that those drugs are homing in on particular cell types and not affecting your whole brain or body. It also means those drugs are more likely to be unaffected by genetic variants and work in many people.

Using the data generated by the analysis, the researchers were able to map out within-cell type genetic regulatory networks and between-cell communication networks, and then plug those networks into a machine learning model. Then, using an individuals genetic information, the model could predict whether they had a brain disease.

Because these networks were hard coded in the model, when the model made a prediction we could see which parts of the network contributed to it, said Gerstein. So we could identify which genes and cell types were important for that prediction. And that can suggest candidate drug targets.

In one example, the model predicted an individual with a particular genetic variant might have bipolar disorder, and the researchers could see that prediction was based on two genes in three cell types. In another, the researchers identified six genes in six cell types that contributed to a schizophrenia prediction.

The model also worked in the opposite direction. The researchers could introduce a genetic perturbation and see how that might affect the network and an individuals health. This, Gerstein says, is useful for drug design or previewing how well drugs or drug combinations might fare as treatments.

Together, the findings could help facilitate precision-medicine approaches for neuropsychiatric disease, said the researchers.

To further this work, the consortium hasmade its results and model availableto other researchers.

Our vision is that researchers interested in a particular gene or variant can use our resources to better understand what its doing in the brain or to perhaps identify new candidate drug targets to investigate more, said Gerstein.

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Tracking the cellular and genetic roots of neuropsychiatric disease - Yale News

Two Bristol academics, Professors Eugenia Piddini and Gene Feder OBE, have been elected to the Academy of Medical Sciences respected and influential Fellowship. They join 58 exceptional biomedical and health scientists selected for their exceptional contributions to the advancement of medical science.

The new Fellows, announced on Tuesday 21 May, have been recognised for their remarkable contributions to advancing biomedical and health sciences, groundbreaking research discoveries and translating developments into benefits for patients and wider society.

Awardees join an esteemed Fellowship of over 1,400 researchers who are at the heart of the Academy's work, which includes nurturing the next generation of researchers and shaping research and health policy in the UK and worldwide. The expertise of Fellows elected this year spans a wide range of clinical and non-clinical disciplines, from midwifery to cancer stem cell biology.

Eugenia Piddini, Professor of Cell Biology in the School of Cellular and Molecular Medicine, is conducting innovative work to identify cell competition-based strategies to gain control over tissue colonisation, its impact in tissue colonisation in regenerative medicine and to prevent tumour expansion in cancer.

A cell and developmental biologist,Eugenia is known for her seminal work in the field of cell competition the mechanism of tissue quality control that removes damaged cells from tissues. Eugenias discoveries have helped widen the scope of cell competition in terms of physiological relevance and potential therapeutic impact. Recently, Eugenias group demonstrated that cell competition acts in adult tissues. There it can potentially slow down the onset of disease/ageing by eliminating damaged cells.

Eugenias team has also shown that tumour cells kill surrounding normal cells via cell competition to free space for their own growth. Their work has identified many mechanisms and signals that cells use to compete. By explaining the mechanisms that cells use to compete the Piddini group aims to identify cell competition-based strategies to gain control over tissue colonisation.

In recognition of her work Eugenia, who is also School Research Director, was awarded the British Society for Cell Biology Hooke Medal in 2019 and in 2023, was elected as a Member of the European Molecular Biology Organisation.

Gene Feder, is a GP and Professor of Primary Care at Bristols Centre for Academic Primary Care, Bristol Medical School and Director of VISION, a UK Prevention Research Partnership (UKPRP) consortium.

Professor Feder leads ground-breaking national and international research on domestic violence and abuse (DVA) from epidemiology to health care response. He is the architect of IRIS, a national DVA programme for general practice, and co-founded IRISi, a social enterprise implementing IRIS nationally. He has extended his research globally through EU and Medical Research Council grants, and co-leadership of HERA, a National Institute for Health and Care Research (NIHR) Global Health Group in collaboration with researchers in Brazil, Nepal Sri Lanka, and the occupied Palestinian territories (oPT).

Committed to developing and evaluating effective and compassionate health care, Professor Feder has championed the use of randomised controlled trials to test improvements in general practice care of patients with heart and respiratory conditions, and robust methods to develop and implement clinical guidelines that make a difference to patients. He extended epidemiological, trial and meta-analytic methods to research on gender-based violence, combining quantitative and qualitive data to evaluate interventions, collaborating with statisticians, epidemiologists, economists, and social scientists. He has chaired four NICE guidelines and the World Health Organisation (WHO) intimate partner and sexual violence guideline development group.

In 2012, he co-founded the Foundation for Family Medicine in Palestine, which aims to support universal health coverage throughout the occupied Palestinian Territories based on effective, efficient and high-quality primary care. In 2016, Professor Feder was awarded an OBE for services to health care and survivors of domestic violence. In 2022, Gene was appointed Director of VISION, a five-year UKPRP inter-disciplinary consortium researching the intersection of violence and health to reduce and mitigate the effects of violence through better measurement and analysis of health care, police, criminal justice, and voluntary sector data. He is an expert advisor to UK Government and WHO.

Professor Andrew Morris PMedSci, President of the Academy of Medical Sciences, said: It is an honour to welcome these brilliant minds to our Fellowship. Our new Fellows lead pioneering work in biomedical research and are driving remarkable improvements in healthcare. We look forward to working with them, and learning from them, in our quest to foster an open and progressive research environment that improves the health of people everywhere through excellence in medical science.

This year's cohort marks a significant milestone in the Academy's efforts to promote equality, diversity and inclusion (EDI) within its Fellowship election. Among the new Fellows, 41 per cent are women, the highest percentage ever elected. Additionally, Black, Asian and minority ethnic representation is 29 per cent, an 11 per cent increase from the previous year. The new Fellows hold positions at institutions across the UK, including in Edinburgh, Birmingham, Liverpool, Manchester, Sheffield, Nottingham and York.

Professor Morris added: It is also welcoming to note that this year's cohort is our most diverse yet, in terms of gender, ethnicity and geography. While this progress is encouraging, we recognise that there is still much work to be done to truly diversify our Fellowship. We remain committed to our EDI goals and will continue to take meaningful steps to ensure our Fellowship reflects the rich diversity of the society we serve."

The new Fellows will be formally admitted to the Academy at a ceremony on Wednesday 18 September 2024.

The Academy of Medical Sciences is the independent, expert body representing the diversity of medical science in the UK. Its mission is to advance biomedical and health research and its translation into benefits for society. The Academy's elected Fellows are the most influential scientists in the UK and worldwide, drawn from the NHS, academia, industry and the public service.

About the Academy of Medical SciencesThe Academy of Medical Sciences is the independent, expert voice of biomedical and health research in the UK. Our Fellowship comprises the most influential scientists in the UK and worldwide, drawn from the NHS, academia, industry, and the public service. Our mission is to improve the health of people everywhere by creating an open and progressive research sector. We do this by working with patients and the public to influence policy and biomedical practice, strengthening UK biomedical and health research, supporting the next generation of researchers through funding and career development opportunities, and working with partners globally.

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May: academy-medical-sciences | News and features - University of Bristol

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Benefits of Stem Cell Therapy: Unlocking Regenerative Medicine's Potential - Intelligent Living

May 06, 2024 -Researchers from the University of Texas (UT) Southwestern Medical Center have developed a computational tool to predict breast cancer immunotherapy response by analyzing how cancer and immune cells interact.

The research team indicated that forecasting patients therapy response has significant potential to inform and improve treatment, as current immunotherapies are limited.

Immunotherapies have made incredible strides in extending survival for cancer patients, but they only work about 20% of the time. To make immunotherapies more beneficial, we need to have a much better understanding of the cellular composition of specific tumors and how those cells interact with each other, said Isaac Chan, MD, PhD, assistant professor of Internal Medicine and Molecular Biology and in the Harold C. Simmons Comprehensive Cancer Center at UT Southwestern, in the press release.

The researchers noted that existing immunotherapies rely on the knowledge that many types of cells including cancer and immune cells are present in tumors. These immune cells can either attack tumors or promote them.

Current immunotherapies often stimulate T cells, a type of immune cell, to fight cancer. However, other immune cells, such as natural killer (NK) cells, can also help attack cancer cells.

But the research team emphasized that in some cases, interactions between NK cells and cancer cells can reprogram the NK cells to promote tumor growth, rather than hinder it. In previous research, Chan and colleagues identified reprogrammed NK cells in breast cancer samples and found that patients with high levels of these cells faced worse outcomes than those with lower levels.

To investigate the potential for therapeutic targeting of reprogrammed NK cells, the research team used single cell RNA sequencing (scRNA-seq) to assess individual gene expression and cancer-immune cell interactions.

Combining eight existing scRNA-seq databases, the researchers developed a single-cell breast tumor atlas consisting of 119 tumor samples from 88 breast cancer patients. The atlas allowed the research team to analyze information from over 236,000 cells.

The analysis revealed 10 categories of cancer cells based on epithelial cell heterogeneity and gene expression.

Currently, three categories of cells triple negative, hormone positive and HER2 positive are used to guide therapy planning.

The researchers also determined that the distributions of the 10 cell categories varied among tumors, with most samples containing a mix of cell subtypes. The study further showed that patients with certain combinations of cell subtypes had worse outcomes than their peers.

Using these insights, the research team developed and tested InteractPrint, a precision medicine tool designed to predict breast cancer immunotherapy response. The tool demonstrated high performance when deployed to predict response to immune checkpoint inhibition (ICI) in breast cancer during two clinical trials testing neoadjuvant anti-PD-1 therapy.

The research team highlighted that InteractPrint could prove useful for helping clinicians evaluate the likelihood of immunotherapy response in patients across cancer types, enabling them to choose the treatment with the highest odds of success.

Others are also developing predictive analytics techniques to advance precision medicine.

In April, researchers from Arizona State University detailed the development of a machine learning model to predict how a patients immune system would respond to foreign cells.

The tool, a convolutional neural network called human leukocyte antigen (HLA)-Inception, utilizes individualized data on molecular interactions to assess how Major Histocompatibility Complex-1 (MHC-1) proteins affect immune response.

These proteins are critical to the immune systems ability to recognize and respond to foreign cells.

HLA-Inception sheds light on how MHC-1s interact with foreign peptides, allowing researchers to forecast immune response and potentially personalize treatment.

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Precision medicine tool predicts breast cancer immunotherapy response - HealthITAnalytics.com

Precedence Research Says, the global cell separation market size was valued at USD 10.92 billion in 2023 and it is increasing over USD 38.99 billion by 2033 with a CAGR of 13.54% from 2024 to 2033.

Cell separation, also known as cell sorting or cell isolation, is a process that separates specific types of cells from a heterogeneous mixture, such as blood or tissue samples. This technique is crucial in various fields, including biomedical research, diagnostics, and therapeutics. In research, cell separation allows scientists to study and analyze specific cell populations, aiding in understanding cell behavior, disease mechanisms, and drug development. In diagnostics, it enables the detection and identification of rare cells, such as circulating tumor cells, which can provide valuable insights into disease progression and treatment response.

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In therapeutics, cell separation plays a vital role in regenerative medicine and cell-based therapies by isolating and purifying desired cell types for transplantation or tissue engineering applications. Techniques for cell separation include centrifugation, magnetic-activated cell sorting (MACS), fluorescence-activated cell sorting (FACS), and microfluidic-based methods. The cell separation market encompasses a range of products and services, including reagents, instruments, and consumables, catering to diverse applications across research, diagnostics, and therapy development. This market continues to evolve with advancements in technology and increasing demand for personalized medicine and cell-based therapies.

Key Insights

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Regional Instances

Reports Highlights

By Product

In the cell separation market, the consumables segment emerges as the dominant force, commanding a significant share due to its essential role in the day-to-day operations of cell separation procedures. Consumables encompass a wide array of products such as reagents, antibodies, beads, and disposable kits, crucial for sample preparation, labeling, and isolation of target cells. With ongoing advancements in cell separation technologies and increasing demand for research and clinical applications, the consumables segment continues to experience steady growth, driven by the perpetual need for high-quality, reliable products to support cell isolation and analysis across diverse biomedical fields.

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By Technique

Among the various techniques employed in cell separation, centrifugation emerges as the dominant segment in the market. This method utilizes centrifugal force to separate cells based on differences in size, density, and sedimentation rate. Widely used across diverse applications, centrifugation offers simplicity, efficiency, and scalability, making it a preferred choice for many researchers and clinicians. Its versatility allows for the isolation of various cell types, ranging from blood components to cell cultures, contributing significantly to biomedical research, diagnostics, and therapeutic development. With ongoing advancements in centrifugation technology, this segment continues to maintain its leading position in the cell separation market.

By Application

In the cell separation market, the biomolecule isolation segment emerges as a dominant force, driven by its wide-ranging applications across biomedical research, diagnostics, and therapeutic development. Biomolecule isolation techniques enable the extraction and purification of specific molecules such as DNA, RNA, proteins, and antibodies from complex biological samples, facilitating critical analyses and downstream applications. With increasing demand for precise molecular profiling, biomolecule isolation remains a cornerstone in unlocking insights into cellular mechanisms, disease pathology, and therapeutic targets, thereby propelling its dominance in the cell separation market.

By End Use

The research laboratory and institutes segment significantly dominates the cell separation market, representing a cornerstone of demand for advanced technologies and solutions. Research laboratories and institutes are at the forefront of scientific exploration, driving the need for precise cell isolation techniques to investigate cellular behavior, disease mechanisms, and therapeutic targets. With a relentless pursuit of knowledge and innovation, these entities heavily rely on cell separation methodologies to unlock new discoveries and advancements in fields such as biomedicine, molecular biology, and drug development. Thus, they remain pivotal contributors to the sustained growth and evolution of the cell separation market.

Market Dynamics

Driver

Advancements in personalized medicine

With the rise of personalized medicine, characterized by tailored treatments based on individual patient characteristics, the demand for precise cell separation technologies has surged. Cell separation plays a crucial role in isolating specific cell types for diagnostic testing, therapy development, and regenerative medicine applications. As healthcare shifts towards more targeted and effective interventions, there is an increasing need for reliable and efficient cell sorting methods to obtain pure cell populations. This drive towards personalized healthcare fuels continuous innovation in cell separation techniques, bolstering the growth and expansion of the market.

Restraint

Regulatory challenges and compliance hurdles

One significant restraint facing the cell separation market is the complex regulatory landscape governing the development, manufacturing, and commercialization of cell separation technologies. Stringent regulations and compliance requirements, particularly in the healthcare and biotechnology sectors, impose considerable challenges and costs on market players. Navigating through diverse regulatory frameworks across different regions often entails lengthy approval processes, thereby delaying product launches and market entry. Moreover, ensuring adherence to evolving regulatory standards demands continuous investment in quality control, documentation, and compliance strategies. Consequently, regulatory hurdles pose a notable barrier to the growth and expansion of the cell separation market.

Opportunity

Expansion into point-of-care applications

One promising opportunity for the cell separation market lies in expanding into point-of-care applications. With the increasing demand for decentralized healthcare solutions and the rise of personalized medicine, there is a growing need for portable and user-friendly cell separation devices that can be utilized directly at the point of care, such as clinics, doctors offices, and even remote or resource-limited settings. Developing efficient and cost-effective point-of-care cell separation technologies would not only enhance accessibility to diagnostic and therapeutic procedures but also open new avenues for market growth and innovation in addressing global health challenges.

Recent advancements

Cell Separation Market Top Companies

Segments Covered in the Report

By Product

By Cell Type

By Technique

By Application

By End-Use

By Geography

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Cell Separation Market is Expanding at 13.54% CAGR by 2033 - BioSpace