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Category Archives: Cell Therapy

Photodynamic Therapy to Treat Cancer – NCI

Posted: October 29, 2022 at 2:16 am

Photodynamic therapy is a two-step process. First, you will first receive a photosensitizer. The drug may be taken by mouth, spread on the skin, or given through an IV, depending on where the tumor is in the body. After 24 to 72 hours, most of the drug will have left normal cells but remain in cancer or precancer cells. Then your tumor will be exposed to the light source.

How the light is applied depends on where the tumor is. For skin tumors, the light is aimed right at the cancer. For tumors in the throat, airways, and lungs, your doctor will insert an endoscope down your throat. An endoscope is a thin, lighted tube that can help the doctor see inside the body. Once the endoscope is in place, the doctor threads a fiber optic cable that transmits light through it to reach the treatment areas.

One type of photodynamic therapy called extracorporeal photopheresis (ECP) is used to treat abnormal white blood cells that can cause skin symptoms in people withcutaneous T-cell lymphoma. In ECP, a machine collects your blood cells, treats them with a photosensitizer, exposes them to light, and then returns them to your body through a needle in a vein.

Most often, you will have photodynamic therapy as anoutpatient, which means you go home after treatment and do not spend the night in the hospital. You may have photodynamic therapy by itself, or you may have it along with other cancer treatments.

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Photodynamic Therapy to Treat Cancer - NCI

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Researchers at University Hospitals create a breakthrough in CAR-T cell therapy for cancer patients – News 5 Cleveland WEWS

Posted: October 29, 2022 at 2:16 am

Researchers at University Hospitals create a breakthrough in CAR-T cell therapy for cancer patients  News 5 Cleveland WEWS

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Researchers at University Hospitals create a breakthrough in CAR-T cell therapy for cancer patients - News 5 Cleveland WEWS

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Mustang Bio Announces Phase 1/2 Clinical Trial Data of MB-106, a First-in-Class CD20-targeted, Autologous CAR T Cell Therapy, to be Presented at 11th…

Posted: October 29, 2022 at 2:15 am

Mustang Bio Announces Phase 1/2 Clinical Trial Data of MB-106, a First-in-Class CD20-targeted, Autologous CAR T Cell Therapy, to be Presented at 11th International Workshop for Waldenstrom's Macroglobulinemia  GlobeNewswire

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Mustang Bio Announces Phase 1/2 Clinical Trial Data of MB-106, a First-in-Class CD20-targeted, Autologous CAR T Cell Therapy, to be Presented at 11th...

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Gilead, working to improve cancer cell therapy, partners with California startup – BioPharma Dive

Posted: October 21, 2022 at 2:39 am

  1. Gilead, working to improve cancer cell therapy, partners with California startup  BioPharma Dive
  2. Kite and Refuge Biotechnologies Announce Exclusive License Agreement for Investigational Gene Expression Platform for Blood Cancers  Gilead Sciences
  3. Kite Pharma Licenses Refuge Biotechnologies Platform to Bolster CAR T-Cell Therapy Development  Precision Oncology News
  4. Gilead's Kite ties itself to Refuge's gene platform in blood cancer CAR-T deal  FierceBiotech
  5. View Full Coverage on Google News

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Gilead, working to improve cancer cell therapy, partners with California startup - BioPharma Dive

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Allogene starts first pivotal trials of an ‘off-the-shelf’ cell therapy for cancer – BioPharma Dive

Posted: October 13, 2022 at 2:21 am

Allogene Therapeutics has begun the first pivotal test of an off-the-shelf cell therapy for cancer.

The biotechnology company, which has been at the forefront of a push in recent years to develop such treatments, known as allogeneic therapies and derived from donor cells, announced the start of two trials on Thursday. One will test a blood cancer drug known as ALLO-501A, while the other will evaluate a regimen Allogene uses to prepare patients for treatment. Assuming positive results, Allogene expects the studies will support approval applications for both of them.

The studies represent milestones for allogeneic cell therapies, which are meant to be more convenient alternatives to the personalized CAR-T treatments that have come to market for a handful of blood cancers. Allogene, spun out of Pfizers cell therapy work in 2018, is the largest and most advanced among the companies advancing them. Its run by former executives of Kite Pharma, which successfully developed the cell therapies now sold by Gilead Sciences.

Allogene raised more than a half a billion dollars in private financing and an initial public offering to fund its work. The company has had a bumpy ride since then, however. Its cell therapies, including ALLO-501A, have shown promise, but also face lingering questions about their durability and effectiveness compared to CAR-T treatments. The field has also gotten more competitive, with an emerging group of companies advancing therapies using different types of cells and CAR-T moving into earlier lines of care.

Additionally, Allogenes research was delayed by the unexpected finding of a chromosomal abnormality in a treated patient. The companys treatment was exonerated, but the FDA froze Allogenes programs for months during the investigation. Longtime partner Servier cut ties with the company last month as well.

The company now has the chance to prove how its technology stacks up. ALLO-501A is being tested in a Phase 2 study in patients with relapsed or refractory large B cell lymphoma, a setting for which multiple CAR-T treatments are already available.

In a research note, RBC Capital Markets analyst Luca Issi noted that its trial, ALPHA2, mimics the design of the studies underlying approvals of those treatments for lymphoma. Its a single-arm study of about 100 patients whove previously received at least two prior treatments, but not a CAR-T therapy. The study will be judged by ALLO-501As ability to induce a response. Allogene didnt disclose a bar for success, but Issi, after speaking with management, said executives believe efficacy needs to be in the range of approved CAR-T therapies.

Notably, the company is testing a single dose of the treatment, not a repeat-dosing regimen Allogene has been experimenting with to strengthen the effects of ALLO-501A. Issi indicated the decision was made for ease and convenience.

Allogenes other study, EXPAND, is a registrational trial for ALLO-647, an antibody drug the company is using to prepare patients for treatment. That study will enroll about 70 patients and have a control arm that doesnt receive Allogenes drug. Updates are expected by the end of the year, Allogene said.

Allogene shares climbed 12% in pre-market trading Friday, though at about $11, shares still trade well below their highs of about $54 apiece in 2020.

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Allogene starts first pivotal trials of an 'off-the-shelf' cell therapy for cancer - BioPharma Dive

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Vita Therapeutics Closes $31 Million Series B Financing to Develop Cell Therapies for Neuromuscular Diseases and Cancers – Business Wire

Posted: October 13, 2022 at 2:21 am

BALTIMORE--(BUSINESS WIRE)--Vita Therapeutics, a cell engineering company harnessing the power of genetics to develop novel cellular therapies to treat muscular dystrophies and cancers, today announced the completion of a $31 million Series B financing. The fundraise was led by Cambrian BioPharma and new investor Solve FSHD. New investors included Riptide Ventures and Cedars Sinai, which participated alongside TEDCO and other existing investors. Proceeds from the financing will be used to advance Vitas lead pre-clinical program VTA-100 for limb-girdle muscular dystrophy (LGMD2A) to the clinic. It will also fund the development of Vitas newest program, VTA-120 for the treatment of patients with facioscapulohumeral muscular dystrophy (FSHD), and to further expand Vitas discovery pipeline. Since inception, Vita has raised a total of $66 million.

The support from this strategic group of quality investors further validates Vitas cell therapy platform and our mission to bring transformative therapies that target the root cause of disease to patients with muscle disorders and cancers, said Douglas Falk, MS, Chief Executive Officer at Vita Therapeutics. This syndicates confidence in our ability to further progress our programs is energizing and we are thrilled to have them as partners. We are making notable progress with our investigational IND-enabling studies for VTA-100 and are on track to reach the clinic with this important therapeutic candidate within 18 months. Additionally, we are excited to further expand our pipeline to include VTA-120 for the treatment of patients with FSHD. Im incredibly proud of our entire team and the steady momentum we continue to have.

Chip Wilson, Founder of lululemon athletica and of Solve FSHD noted, Living with FSHD for over 30 years, my upper body muscles are quite wasted. We are hopeful that Vitas cell therapy approach will stimulate muscle regeneration and help people like me to build up muscle faster than it breaks down.

Currently there are no treatments available for FSHD, and there is an urgent need to develop disease-modifying treatments that not only regenerate muscle but correct the genetic defect that otherwise leads to the muscles inability to repair itself, added Eva Chin, Executive Director for Solve FSHD. We are pleased to support Vita as they continue to expand their induced pluripotent stem cell (iPSC) technology towards FSHD and LGMD.

Vita Therapeutics aligns with Cambrians mission of building medicines that will redefine healthcare in the 21st century, said Cambrian BioPharma Chief Executive Officer, James Peyer, PhD. The team, as well as the scientific platform, continues to impress us as they aim to solve for treatments that go beyond symptom management to truly impact these diseases in a positive way.

Pipeline Overview

Vita Therapeutics current pipeline includes lead program, VTA-100 for the treatment of LGMD2A, VTA-120 for the treatment of FSHD, and VTA-300 targeting multiple cancers.

About Limb-Girdle Muscular Dystrophy

Limb-girdle muscular dystrophy (LGMD) is a group of disorders that cause weakness and wasting of muscles closest to the body (proximal muscles), specifically the muscles of the shoulders, upper arms, pelvic area, and thighs. The severity, age of onset, and disease progression of LGMD vary among the more than 30 known sub-types of this condition and may be inconsistent even within the same sub-type. As the atrophy and muscle weakness progresses, individuals with LGMD begin to have trouble lifting objects, walking, and climbing stairs, often requiring the use of assistive mobility devices. There is currently no cure for LGMD, with treatments limited to supportive therapies such as corticosteroids.

About Facioscapulohumeral Muscular Dystrophy

Facioscapulohumeral muscular dystrophy (FSHD) is an autosomal dominant muscular dystrophy, although thirty percent of new FSHD patients have no prior family history of the disease and result from a congenital spontaneous genetic mutation. FSHD typically first presents with weakness of the muscles of the facial muscles and scapular region, with proximal weakness of the pectoral and abductor muscles limiting upper extremity function at the shoulder girdle. Onset is typically in the teenage and early adult years, but it can present in infancy, which tends to be a more aggressive course. The disease is slowly progressive and approximately 20% of patients are wheelchair bound by age 50. Currently there are no treatments specifically indicated for use in FSHD, with no disease-modifying treatments available.

About Vita Therapeutics

Vita Therapeutics is a biotechnology company developing state-of-the-art cellular therapeutics for the treatment of debilitating neuromuscular diseases and cancers. Vita Therapeutics uses induced pluripotent stem cell (iPSC) technology to engineer specific cell types designed to replace those that are defective in patients. The Company is progressing its lead program VTA-100 for the treatment of limb-girdle muscular dystrophy (LGMD2A) with the goal of filing Investigational New Drug Applications with the US Food and Drug Administration in the next 18 months. Long term, the Company is developing its pipeline of cellular therapies following a dual development strategy beginning with autologous-derived cells before moving to a universal hypoimmunogenic cell line. Vita Therapeutics is currently working with numerous partners, including PanCella, Wyss Institute, and Johns Hopkins University, to advance their clinical programs. Learn more about the company at http://www.Vitatx.com.

About Cambrian BioPharma

Cambrian BioPharma is building the medicines that will redefine healthcare in the 21st century therapeutics to lengthen healthspan, the period of life spent in good health. As a Distributed Development Company, Cambrian is advancing multiple scientific breakthroughs each targeting a biological driver of aging. Its approach is to develop interventions that treat specific diseases first, then deploy them as preventative medicines to improve overall quality of life during aging. For more information, please visit http://www.cambrianbio.com or follow us on Twitter @CambrianBio and LinkedIn.

About SOLVE FSHD

SOLVE FSHD is a venture philanthropic organization established to catalyze innovation and accelerate key research in finding a cure for FSHD. Established by renowned Canadian entrepreneur and philanthropist Chip Wilson, the founder of technical apparel company lululemon athletica inc. Chip has committed $100 million to kick-start funding into projects that support the organizations mission to find a cure for FSHD by 2027. The goal of SOLVE FSHD is to find a solution that can stop muscle degeneration, increase muscle regeneration and strength, and improve the quality of life for those living with FSHD. For more information, please visit: http://www.solvefshd.com.

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Vita Therapeutics Closes $31 Million Series B Financing to Develop Cell Therapies for Neuromuscular Diseases and Cancers - Business Wire

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CAR T-cell Therapy Market Riding on the Wave of Growth and will Grow at a CAGR of 30.6% to 2031 | TMR Study – GlobeNewswire

Posted: October 13, 2022 at 2:21 am

Wilmington, Delaware, United States, Oct. 12, 2022 (GLOBE NEWSWIRE) -- Transparency Market Research Inc. The solutions in the global CAR T-cell therapy market are increasingly being hailed as cutting-edge innovations in the field of cancer treatments. Clinical trials conducted by players in the global CAR T-cell therapy market are displaying promising results, particularly in end stage patients suffering from acute lymphocytic leukemia. The success of these trials is translating into substantial revenue grab opportunities for market players. As per a recent professional survey report, the global CAR T-cell therapy market is estimated to grow at a CAGR of 30.6% over the forecast period of 2021 to 2031.

Players in the global CAR T-cell therapy market are working closely with research institutes and hospitals to develop cost-effective solutions, as the high cost of CAR T-cell therapies remains a major barrier for the adoption of these solutions. Chimeric Antigen Receptors or CAR are protein constructs that have been genetically engineered to be incorporated into patients cytotoxic T cells for aiding them in fighting cancer cells by efficiently recognizing them. Players in the global CAR T-cell therapy market offer various types of novel products, including tisagenlecleucel, lisocabtagene maraleucel, axicabtagene ciloleucel, brexucabtagene autoleucel, and idecabtagene vicleucel, among others.

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CAR T-cell Therapy Market Key Findings of the Report

CAR T-cell Therapy Market Growth Drivers

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CAR T-cell Therapy Market Key Players

Some of the key companies operating in the global CAR T-cell therapy market include Novartis AG, Amgen, Inc., Johnson & Johnson Services, Inc., Merck & Co., Inc., Pfizer, Inc., Bristol-Myers Squibb, Sorrento Therapeutics, Inc., Gilead Sciences, Inc., and bluebird bio, Inc., among others. Some of the noteworthy expansion strategies employed by these players include acquisitions, strategic alliances, geographical expansion, new product launches, and distribution agreements.

Furthermore, leading players in the global CAR T-cell therapy market are also focusing on research and development to design and develop new types of CAR T-cell therapy products intended for treating different types of cancer. Stakeholders in the global CAR T-cell therapy market are complying with various research and health policy frameworks in order to adhere to stringent regulations put forth by governments across the world.

CAR T-cell Therapy Market Regional Growth Assessment

In 2020, North America region held the dominant share of the global CAR T-cell therapy market. This leading industry positioning of the North America region can be attributed to the presence of a large number of leading international players, rising healthcare spending, and early adoption of new medical technologies and techniques. Furthermore, rising number of research activities in the region is also boosting growth within the North America CAR T-cell therapy market. In coming years, Asia Pacific is also expected to witness significant growth in the global market.

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CAR T-cell Therapy Market: Segmentation

CAR T-cell Therapy Market, by Product Type

CAR T-cell Therapy Market, by Indication

CAR T-cell Therapy Market, by End User

CAR T-cell Therapy Market, by Region

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CAR T-cell Therapy Market Riding on the Wave of Growth and will Grow at a CAGR of 30.6% to 2031 | TMR Study - GlobeNewswire

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Inceptor Bio and Avectas Announce Collaboration to Improve Manufacturing of CAR-T Cell Therapies for the Treatment of Solid Tumors – PR Newswire

Posted: October 13, 2022 at 2:21 am

Collaboration brings together Avectas' breakthrough cell engineering technology and Inceptor Bio's CAR-T platform

MORRISVILLE, N.C. and DUBLIN, Oct. 12, 2022 /PRNewswire/ --Inceptor Bio, acell therapy biotechnology company and Avectas, a cell engineering technology leader, announce their collaboration to improve the development and manufacturing of next-generation CAR-T cell therapies for the treatment of solid tumors.

Under the agreement, Inceptor will utilize Avectas' SOLUPORE technology as an alternative to electroporation for engineering T cells with the goal to yield a healthier T cell product. Avectas' SOLUPORE technology is well suited to Inceptor Bio's CAR-T cell process, which aims to improve the quality of the engineered T cells, and ultimately, enhance their durability in the tumor microenvironment. By combining Avectas' SOLUPORE delivery with Inceptor Bio's CAR-T cell therapy platform, the engineered cells have the potential for improved performance and efficacy.

Avectas has developed the non-viral SOLUPORE technology to overcome the limitations of current delivery modalities to enable cell engineering, including complex editing, by efficiently delivering cargoes into cells. SOLUPORE technology permeabilizes the target cell membrane so that molecular cargoes can be delivered while retaining high cell viability and functionality levels.

"Inceptor Bio is committed to building and advancing a pipeline of programs based on diversified cell therapies, including CAR-M, CAR-T, and CAR-NK. This collaboration with Avectas is part of our strategy of advancing Inceptor Bio's next-generation cell therapy platform focused on multiple novel mechanisms to address solid tumors," saidShailesh Maingi, Founder and CEO of Inceptor Bio. "The aim for our proprietary CAR-T platform is to transform how solid tumors are treated."

"Avectas is delighted to collaborate with Inceptor Bio to leverage the benefits of the SOLUPORE delivery platform for solid tumor CAR-T cell therapies," said Michael Maguire, PhD, CEO of Avectas. "Excellent cell health and functionality, after complex editing, are critical to the success of these next-generation therapies".

About Avectas

Avectas is a cell engineering technology business that has developed a unique delivery platform to enable theex vivomanufacture of gene-modified cell therapy products that retain highin vivofunctionality. Our vision is to position the non-viral SOLUPOREcell engineering technology to be integrated into manufacturing processes, including complex gene editing, for multiple autologous and allogeneic therapies and commercialized through development and license agreements. For more information, please visit the company's websiteatwww.avectas.com.

About Inceptor Bio

Inceptor Bio is a biotechnology company developing multiple next-generation cell therapy products to deliver cures for underserved and difficult-to-treat cancers. Inceptor Bio is developing CAR-T, CAR-M, and CAR-NK platforms to generate a diversified pipeline to strategically treat a broad range of solid tumors. Inceptor Bio is headquartered in the Research Triangle Park area in Morrisville, North Carolina. More information is available at http://www.inceptor.bio.

Media contacts:

For Inceptor BioAbe Maingi[emailprotected]

For Avectas

InternationalSue Charles, Charles Consultants+44 7968 726585 [emailprotected],com

IrelandRay Gordon, Gordon MRM+353 87 2417373 [emailprotected]

SOURCE Avectas

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Inceptor Bio and Avectas Announce Collaboration to Improve Manufacturing of CAR-T Cell Therapies for the Treatment of Solid Tumors - PR Newswire

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Cell Therapy Technologies Market Expected to Reach US ~$12.27 Billion and CAGR 14.5% by 2030 Polaris Market Research – Digital Journal

Posted: October 13, 2022 at 2:21 am

The recently published report entitled Cell Therapy Technologies Market 2022: By Types, Applications, Size, Share, Key Players & Regions Forecast Analysis till 2030 by Polaris Market Research covers unique insights into the Cell Therapy Technologies market that are contributing to the growth of the overall industry.

According to the research report, the global cell therapy technologies market was valued at USD 3.86 billion in 2021 and is expected to reach USD 12.27 billion by 2030, to grow at a CAGR of 14.5% during the forecast period.

The report can help stakeholders gain knowledge about the competitive environment and design profound strategies to help them increase their revenue. The report includes comprehensive information on the market size of Cell Therapy Technologies, its volume, share, recent trends and developments, demand, and growth rate. The report guides readers about the current status of the industry and its future prospects. It analyzes the market by type of component, end-user, company, and region. Also, market size (value and volume), share, and growth rate by types and applications in estimated and included in the report.

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Key Highlights of Report

The report focuses on the current status, competitor landscape, changing dynamics, detailed cost analysis, supply chain, marketing channels, and geographical overview. The study sheds light on crucial information on critical dynamics such as drivers, restraints, opportunities, and challenges. Recommendations and business advice are given that will help the readers ensure success in the market. It offers a qualitative and quantitative analysis of the overall industry. Facts and data are represented through graphs, pie charts, diagrams, and other graphical representations.

Product and Application Segment Analysis

Based on product segment, the report estimates sales, revenue, price, market share, and the growth rate by type. Then, it focuses on the application of Cell Therapy Technologies by evaluating the status and outlook for major applications/end users, consumption (sales), market share and growth rate for each application, consumption, and the growth rate of each application. Growth prospects are given in terms of sales and revenue forecast, and regional forecast. It also makes projections of the market by type and application. Overall, segment analysis helps in understanding the key factors that support the markets growth.

Top Market Players Profiles Covered in This Report:

Competitive Scenario

The report emphasizes leading players of the Cell Therapy Technologies market covering their information such as production, company profiles, revenue, product images, as well as specification, price, capacity, price, and contact information. Upstream raw materials, instruments as well as downstream demand survey is also mentioned in this report. This section also helps to analyze the strategies, developments, and collaborations made by key players.

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Overview of Markets Key Factors

Regional Scope of the Market

The report analyzes and examines various factors that determine the regional growth of a particular region. Analysts have studied the data on revenue, production, and manufacturers of each region. This section conducts an in-depth analysis and overview of the market development of the key countries on national, regional, and international levels. Market share assessments for the regional and country level segments are delivered in this report.

The report aims to assist the reader in developing business expansion plans. In this document, SWOT analysis and Porters five forces analysis are used, which help in providing the precise direction of the market. It further sheds light on the factors driving the Cell Therapy Technologies industry growth coupled with those expected to hamper the development of the market in the coming years. Analysts have evaluated the growing investments in manufacturing activities as well as product innovation that are likely to impact these segments. Further, the research explains the massive movements in this market for the predicted timeline.

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Cell Therapy Technologies Market Expected to Reach US ~$12.27 Billion and CAGR 14.5% by 2030 Polaris Market Research - Digital Journal

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Early Data on GPRC5D-Directed CAR T-Cell Therapy Open Doors for New Myeloma Treatment Strategies – Precision Oncology News

Posted: October 13, 2022 at 2:21 am

NEW YORK Bristol Myers Squibb is advancing a larger, multi-center trial of its investigational GPRC5D-targeting CAR T-cell therapy after a proof-of-principle trial in heavily treated multiple myeloma patients yielded encouraging response rates.

Meanwhile, based on the activity seen in this early-stage study, researchers are also exploring the potential of dual CAR T-cell therapy simultaneously targeting GPRC5D and BCMA in multiple myeloma.

The data that enticed BMS and researchers came from a single-center trial and was recently published in the New England Journal of Medicine, showing a 71 percent response rate among multiple myeloma patients who received the autologous CAR T-cell therapy, dubbed MCARH109, after five or more prior treatments. BMS gained the rights to develop the CAR T-cell therapy in 2019, when it acquired Celgene, which had previously acquired Juno Therapeutics. Juno bought the rights to develop CAR T-cell therapies using antibodies targeting GPRC5D from Memorial Sloan Kettering in 2016.

In the Phase I trial published in NEJM, patients were treated at MSK, and their autologous T cells were engineered to target the GPRC5D protein in MSK's Cell Therapy Cell Engineering Facility. The trial enrolled 17 multiple myeloma patients, all of whom had received at least three prior therapies, but most of whom received more than that. The median number of prior treatment lines was six, and eight patients had already received BCMA-directed autologous CAR T-cell therapy.

Across all four dose levels of the GPRC5D-directed CAR T-cell therapy, 71 percent of patients responded, and 35 percent experienced a complete response or stringent complete response. After a median follow-up of 10.1 months, 50 percent of the patients who experienced at least a partial response remain progression-free, and the therapy was generally safe with side effects comparable to those seen with B-cell maturation antigen (BCMA)-directed CAR T-cell therapies.

According to Renier Brentjens, the deputy director of the Roswell Park Comprehensive Cancer Center and a co-senior author on the Phase I study, one of the most encouraging findings was that of the eight patients who'd already received BCMA-targeted CAR T-cell therapy, 75 percent responded to MCARH109.

"That was key, because it means that with this type of technology, you get more than one chance at bat," said Brentjens, who worked at MSK at the start of the trial. "If you don't get long-term remission, but you get a year of disease-free survival out of one [CAR T-cell therapy], then we know that we still have another option in our back pocket."

The finding that GPRC5D-directed CAR T-cell therapy could be effective for multiple myeloma patients who have already received BCMA-directed CAR T-cell therapy comes on the heels of US market approval of two multiple myeloma CAR T-cell therapies that target BCMA. Last year, the US Food and Drug Administration approved 2seventy Bio (formerly Bluebird Bio) and BMS's Abecma (idecabtagene vicleucel) as a fifth-line treatment for relapsed or refractory multiple myeloma patients, and in March, Janssen and Legend Biotech netted approval for Carvykti (ciltacabtagene autoleucel) for the same patient population. These drugs are approved in Europe in this setting, too.

The arrival of CAR T-cell therapy in multiple myeloma has been encouraging for patients whose cancers haven't responded to prior treatment regimens. Abecma and Carvykti both led to high response rates in pivotal trials, and many patients have benefited from the autologous therapies since they've entered the US and European markets. But as Brentjens and colleagues wrote in their recent NEJM paper, these therapies "have not generated survival curves with a plateau in patients with multiple myeloma, and most patients are likely to have an eventual relapse."

When, eventually, most patients do experience disease progression following BCMA-directed CAR T-cell therapy, the treatment options are limited, highlighting a significant need for a next-line therapy like MCARH109.

Possible dual-targeting approach

Beyond the possibility that MCARH109 might offer multiple myeloma patients another option after BCMA-directed CAR T-cell therapy, Brentjens suggested there might be a role for combined CAR T-cell infusions that target both BCMA and GPRC5D.

"The idea is that, if the tumor can somehow dodge one CAR T cell by, for example, decreasing or losing expression of BCMA well, it's a lot more difficult to get rid of both targets at the same time," Brentjens said. "If target antigen loss is a reason why patients get relapsed disease, one would predict that that would occur less frequently than if you're going after two targets rather than one on the tumor cell."

Clinical trials evaluating this approach are already underway in the academic setting, Brentjens said. He and his colleagues at MSK have recently begun enrolling patients into a Phase I trial combining BCMA- and GPRC5D-targeted CAR T-cell therapy for relapsed or refractory multiple myeloma patients after at least three prior treatment lines.

Investigators exploring the dual receptor targeting approach will need to be extra careful about patient toxicity, Brentjens acknowledged. "You would think that the toxicity would be additive, so the toxicity you see with one therapy would be seen at the same time as the toxicity you see with the other but biology can sometimes surprise us," he said. "You need to be vigilant to look at whether there is a synergistic degree of toxicity if you get both [CAR T-cell therapies] at the same time."

As far as the efficacy of the dual-targeting approach goes, Brentjens said that the data he and colleagues have generated to date "certainly justifies trying both at the same time."

"I'm very excited about it," he said of the new combined CAR T-cell trial. "I think we're one of the first to try to see if this approach might be feasible, and hopefully we'll see that the remissions are longer term when compared to one or the other treatment alone."

Brentjens also suggested he could see a future situation in which patients could have their cells analyzed prior to undergoing CAR T-cell therapy, and a higher expression of one versus the other target be it BCMA or GPRC5D could determine which therapy a patient receives. That said, because BCMA-targeted CAR T-cell therapy is currently FDA approved whereas GPRC5D-directed CAR T-cell therapy is not, the prospect of that sort of biomarker-directed approach to sequencing cell therapies is not right around the corner; clinical trials would first need to provide enough support for MCAR109 to score regulatory approval.

This may well be on the horizon, though. BMS has recently launched a dose-escalation study of MCARH109 at nine sites across the country and is planning to enroll 77 relapsed or refractory multiple myeloma patients. Participants in this trial must have three or more prior therapies, though prior BCMA-targeted CAR T-cell therapy is not a requirement for enrollment.

BMS did not provide further information about this trial or its overall approach to studying MCARH109.

See more here:
Early Data on GPRC5D-Directed CAR T-Cell Therapy Open Doors for New Myeloma Treatment Strategies - Precision Oncology News

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