Chimeric antigen receptor (CAR) T-cell therapy, described as a "living drug," is now available for patients with relapsed/refractory multiple myeloma who have been treated with four or more prior lines of therapy.
The US Food and Drug Administration (FDA) said these patients represent an "unmet medical need" when it granted approval for the new product idecabtagene vicleucel (ide-cel; Abecma), developed by bluebird bio and Bristol-Myers Squibb.
Ide-cel is the first CART-cell therapy to gain approval for use in multiple myeloma. It is also the first CAR T-cell therapy to target B-cell maturation antigen.
Previously approved CAR T-cell products target CD19 and have been approved for use in certain types of leukemia and lymphoma.
All the CAR T-cell therapies are customized treatments that are created specifically for each individual patient from their own blood. The patient's own T cells are removed from the blood, are genetically modified and expanded, and are then infused back into the patient. These modified T cells then seek out and destroy blood cancer cells, and they continue to do so long term.
In some patients, this has led to eradication of disease that had previously progressed with every other treatment that had been tried results that have been described as "absolutely remarkable" and "one-shot therapy that looks to be curative."
However, this cell therapy comes with serious adverse effects, including neurologic toxicity and cytokine release syndrome (CRS), which can be life threatening. For this reason, all these products have a risk evaluation and mitigation strategy, and the use of CAR T-cell therapies is limited to designated centers.
In addition, these CAR T-cells products are phenomenally expensive; hospitals have reported heavy financial losses with their use, and patients have turned to crowdfunding to pay for these therapies.
The FDA noted that approval of ide-cel for multiple myeloma is based on data from a multicenter study that involved 127 patients with relapsed/refractory disease who had received at least three prior lines of treatment.
The results from this trial were published in February 2021 in the New England Journal of Medicine (NEJM).
An expert not involved in the trial described the results as "phenomenal."
Krina Patel, MD, an associate professor in the Department of Lymphoma/Myeloma at the University of Texas MD Anderson Cancer Center, Houston, Texas, said that "the response rate of 73% in a patient population with a median of sixlines of therapy, and with one-third of those patients achieving a deep response of complete response or better, is phenomenal.
"We are very excited as a myeloma community for this study of idecabtagene vicleucel for relapsed/refractory patients," Patel told Medscape Medical News at the time.
The lead investigator of the study, Nikhil Munshi, MD, of Dana-Farber Cancer Institute, Boston, Massachusetts, commented: "The results of this trial represent a true turning point in the treatment of this disease. In my 30 years of treating myeloma, I have not seen any other therapy as effective in this group of patients."
In my 30 years of treating myeloma, I have not seen any other therapy as effective in this group of patients. Dr Nikhil Munshi
Both experts highlighted the poor prognosis for patients with relapsed/refractory disease. Recent decades have seen a flurry of new agents for myeloma, and there are now three main classes of agents: immunomodulatory agents, proteasome inhibitors, and anti-CD38 antibodies.
Nevertheless, in some patients, the disease continues to progress. For patients for whom treatments with all three classes of drugs have failed, the median progression-free survival is 3 to 4 months, and the median overall survival is 9 months.
In contrast, the results reported in the NEJM article showed that overall median progression-free survival was 8.8 months, but it was more than double that (20.2 months) for patients who achieved a complete or stringent complete response.
Estimated median overall survival was 19.4 months, and the overall survival was 78% at 12 months. The authors notethat overall survival data are not yet mature.
The patients who were enrolled in the CAR T-cell trial had undergone many previous treatments. They had undergone a median for six prior drug therapies (range, three to 16), and most of the patients (120, 94%) had also undergone autologous hematopoietic stem cell transplant.
In addition, the majority of patients (84%) had disease that was triple refractory (to an immunomodulatory agent, a proteasome inhibitor, and an anti-CD38 antibody), 60% had disease that was penta-exposed (to bortezomib, carfilzomib, lenalidomide, pomalidomide, and daratumumab), and 26% had disease that was penta-refractory.
In the NEJM article, the authors report that about a third of patients had a complete response to CAR T-cell therapy.
At a median follow-up of 13.3 months, 94 of 128 patients (73%) showed a response to therapy (P < .001); 42 (33%) showed a complete or stringent complete response;and 67 patients (52%) showed a "very good partial response or better," they write.
In the FDA announcement of the product approval, the figures for complete response was slightly lower. "Of those studied, 28% of patients showed complete response or disappearance of all signs of multiple myeloma to Abecma, and 65% of this group remained in complete response to the treatment for at least 12 months," the agency noted.
The FDA also noted that treatment with Abecma can cause severe side effects. The label carries a boxed warning regarding CRS, hemophagocytic lymphohistiocytosis/macrophage activation syndrome, neurologic toxicity, and prolonged cytopenia, all of which can be fatal or life threatening.
The most common side effects of Abecma are CRS, infections, fatigue, musculoskeletal pain, and a weakened immune system. Side effects from treatment usually appear within the first 1 to 2 weeks after treatment, but some side effects may occur later.
The agency also noted that, to further evaluate the long-term safety of the drug, it is requiring the manufacturer to conduct a postmarketing observational study.
"The FDA remains committed to advancing novel treatment options for areas of unmet patient need," said Peter Marks, MD, PhD, director of the FDA's Center for Biologics Evaluation and Research.
"While there is no cure for multiple myeloma, the long-term outlook can vary based on the individual's age and the stage of the condition at the time of diagnosis. Today's approval provides a new treatment option for patients who have this uncommon type of cancer."
For more from Medscape Oncology, join us on Twitter and Facebook.
Read more:
First CAR T-Cell Therapy for Multiple Myeloma: Abecma - Medscape
- SUM Hospital conducts Odishas first stem cell transplantation - The Times of India - April 24th, 2024
- Cancer treatment: What is stem cell therapy, and how does it work? - Moneycontrol - April 24th, 2024
- The murky, unregulated world of anti-ageing stem cell therapy - The Telegraph - April 24th, 2024
- Drug Prototype Promising in Treatment of Pulmonary Disease - Medscape - April 20th, 2024
- Man Paralyzed In Surfing Accident Regains Ability to Walk After Stem Cell Treatment - The Inertia - April 20th, 2024
- Medical breakthrough: 8-year-old boy first in New York to receive FDA-approved gene therapy for rare inherited blood disorder - WABC-TV - April 20th, 2024
- Stem Cell Treatment Helped A Man Who Was Paralyzed From The Neck Down To Stand On His Own - Bored Panda - April 20th, 2024
- UM, CUHK jointly hold symposium on stem cells and regenerative medicine - gcs.gov.mo - April 20th, 2024
- Paralysed patient now able to walk as stem cell therapy shows promising results - WION - April 15th, 2024
- Andrew Steele Sees Aging as the Root of Disease - Worth - Worth Magazine - April 15th, 2024
- The astonishing effect of stem cell implants - as groundbreaking treatment 'transforms' life for those with brain damage ... - Daily Mail - April 15th, 2024
- Consumer Alert on Regenerative Medicine Products Including Stem Cells - FDA.gov - April 13th, 2024
- This Startup Wants To Be OpenAI Of Stem Cell Therapy, Targets $250B Market - Forbes - April 13th, 2024
- Brain-cell transplants are the newest experimental epilepsy treatment - MIT Technology Review - April 3rd, 2024
- Global Regenerative Medicine Market to Touch Valuation of USD 472.95 Billion by 2032, at 25.86% CAGR: Astute ... - Yahoo Finance - April 1st, 2024
- Explosive Growth Projected for Stem Cell Market, Expected to Surpass $37 Billion by 2030 As Revealed In New... - WhaTech - April 1st, 2024
- Integrating New Therapies With Autologous Stem Cell Transplant in Myeloma - Targeted Oncology - April 1st, 2024
- New tool provides researchers with improved understanding of stem cell aging in the brain - Medical Xpress - March 30th, 2024
- The ISSCR announces 2024 election results - EurekAlert - March 28th, 2024
- Exosomes and Stem Cells Are the Future of Anti-Aging - NewBeauty Magazine - March 28th, 2024
- Charles River Extends Gene Therapy Alliance with NUS Medicine - Contract Pharma - March 22nd, 2024
- Exciting opportunities in stem cell technology and regenerative biology in Indian healthcare industry - Express Healthcare - March 22nd, 2024
- Charles River Announces Extension of Gene Therapy Manufacturing Alliance with NUS Yong Loo Lin School of Medicine - Yahoo Finance - March 22nd, 2024
- Opinion: The revolutionary sickle cell therapies - MSN - March 22nd, 2024
- 10 Years of Kansas Regenerative Medicine Center - KSAL - March 22nd, 2024
- BioCardia and StemCardia Announce Biotherapeutic Delivery Partnership - Diagnostic and Interventional Cardiology - March 17th, 2024
- Cynata Therapeutics' breakthrough in regenerative medicine - Finance News Network - March 17th, 2024
- New Positive Data Presented on Briquilimab Conditioning in Patients with Fanconi Anemia - GlobeNewswire - March 17th, 2024
- Novel 3D stem cell therapy to treat critical limb ischaemia - Drug Target Review - March 15th, 2024
- Breaking Boundaries: Pharmicell's Bold Vision for Healthcare Transformation and Monumental Growth - The Worldfolio - March 15th, 2024
- CuSTOM Organoid Research Evolving From Tool to Treatment - Research Horizons - Research Horizons - March 15th, 2024
- BioCardia and StemCardia Announce Partnership to Deliver Cell-Gene Therapy for Remuscularization of Heart - Cath Lab Digest - March 15th, 2024
- Calidi Biotherapeutics and City of Hope Announce Funding from the California Institute for Regenerative Medicine ... - Business Wire - March 13th, 2024
- Iron restriction keeps blood stem cells young, researchers find - Phys.org - March 13th, 2024
- Vitamin A could have a key role in both stem cell biology and wound healing: Study - Medical Dialogues - March 9th, 2024
- Trends in Stem Cell Transplantation Refusal for Myeloma Treatment - Targeted Oncology - March 7th, 2024
- Stem Cell Therapy Market (CAGR) of 31.1%, Future of Market Size Emerging Technologies and their Impact on ... - Taiwan News - March 7th, 2024
- This Swedish startup wants to reduce the cost, and controversy, around stem cell production - TechCrunch - March 6th, 2024
- Advancing stem cell therapy: The regulatory process to get to clinical trials - The Economic Times - March 6th, 2024
- Transitioning from traditional surgical methods to the innovative use of stem cells - pharmaphorum - March 6th, 2024
- 'Mini organs' are grown from human stem cells taken during the late stages of pregnancy for the first time - m - Daily Mail - March 6th, 2024
- Stem cell transplant cures man of HIV and leukemia, one of five to achieve full remission - Fox 28 - March 3rd, 2024
- Stem Cell Therapy Market to Boost USD 137.5 Billion by 2034 and Projected to grow at 32.10% CAGR with increasing ... - Yahoo Finance - March 1st, 2024
- Studies expand use of stem cell therapies - WPLG Local 10 - March 1st, 2024
- Innovative therapy targets and destroys leukemia stem cells - News-Medical.Net - February 27th, 2024
- Unlocking the Potential of Stem Cells: The Medical Revolution of the Century - EIN News - February 27th, 2024
- Pelage Pharmaceuticals Announces $16.75M Series A Financing led by GV to Revolutionize Regenerative Medicine ... - Yahoo Finance - February 27th, 2024
- Say Her Name - Henrietta Lacks, The Real Mother Of Stem - Her Campus - February 27th, 2024
- California Man Free of HIV And Cancer in Astonishing Medical Recovery - ScienceAlert - February 25th, 2024
- $93 million for research into improved health for children, young people and the aged, medical devices, mental health ... - Department of Health - February 25th, 2024
- Stem Cells in Regenerative Medicine - PMC - National Center for ... - February 24th, 2024
- The Promise and Reality of Stem Cell Therapy: What You Need to Know - Corporate Wellness Magazine - February 22nd, 2024
- Stem Cell Therapy: A Beacon of Hope for Patients with Untreatable Conditions - Corporate Wellness Magazine - February 22nd, 2024
- Leading Experts in Stem Cell Therapy: A Global Overview - Corporate Wellness Magazine - February 22nd, 2024
- Can Stem Cells Help Reverse the Effects of Aging? - Corporate Wellness Magazine - February 22nd, 2024
- Stem Cell Therapy for Cancer: Hope on the Horizon? - Corporate Wellness Magazine - February 22nd, 2024
- Stem Cells and Autism: A Closer Look at Innovative Treatments - Corporate Wellness Magazine - February 22nd, 2024
- Regrowth and Restoration: The Science Behind Stem Cell Therapy for Hair - Corporate Wellness Magazine - February 22nd, 2024
- Brain Recovery: The Role of Stem Cells in Stroke and Cerebral Palsy Treatments - Corporate Wellness Magazine - February 22nd, 2024
- Turning Back the Clock: The Science of Stem Cells in Anti-Aging - Corporate Wellness Magazine - February 22nd, 2024
- Insights into cellular therapies for cancer treatment - Drug Target Review - February 22nd, 2024
- The Role of Stem Cells in Rheumatoid Arthritis Recovery - Corporate Wellness Magazine - February 22nd, 2024
- Dr Ernesto Romeros vision transforms immunotherapy regenerative medicine into a global leader in stem cell treatment - Khaleej Times - February 22nd, 2024
- World-Renowned Doctors Leading the Way in Stem Cell Research and Treatment - Medical Tourism Magazine - February 20th, 2024
- Choosing the Best: A Guide to America's Premier Stem Cell Therapy Providers - Medical Tourism Magazine - February 20th, 2024
- Navigating Through the Possibilities of Stem Cell Treatment for Eye Disorders - Medical Tourism Magazine - February 20th, 2024
- Global Leaders in Stem Cell Therapy: Where Excellence Meets Innovation - Medical Tourism Magazine - February 20th, 2024
- Excellence in Stem Cell Treatment: How the US is Pioneering Global Healthcare Solutions - Medical Tourism Magazine - February 20th, 2024
- Clarifying Memory Loss Treatments: The Role of Stem Cells - Medical Tourism Magazine - February 20th, 2024
- Battling Inflammation: Stem Cell Therapy for Rheumatoid Arthritis - Medical Tourism Magazine - February 20th, 2024
- Digestive Health and Stem Cells: Treating Ulcerative Colitis and Crohn's Disease - Medical Tourism Magazine - February 20th, 2024
- Top Stem Cell Clinics in the USA: Pioneering Treatments for Chronic Conditions - Medical Tourism Magazine - February 20th, 2024
- Stem Cells and Autism: Understanding the Potential for Innovative Treatments - Medical Tourism Magazine - February 20th, 2024
- Stopping the awakening of sleeping Acute Myeloid Leukaemia stem cells to prevent disease relapse - University of Birmingham - February 20th, 2024
- City of Hope Achieves Ground Breaking Treatment for Blood Cancer and HIV - Medriva - February 18th, 2024
- Regenerative Medicine Market Size to Worth USD 95 Billion by 2032 - InvestorsObserver - February 18th, 2024
- North America Organoids Market Projected to Reach US$ 5.35 Billion by 2030, Riding on a CAGR of 21.5% - GlobeNewswire - February 18th, 2024
- Chinese team tests lung treatment that may be first to reverse COPD damage - South China Morning Post - February 15th, 2024
- Why leukemic stem cells not harmed by chemotherapy begin to grow and produce AML cells after treatment - Medical Xpress - February 15th, 2024
- Regenerative Medicine Market is Expected to Reach $49.0 Billion | MarketsandMarkets - Yahoo Finance - February 15th, 2024
Recent Comments