CAR T cell therapy has been one of the most exciting medical advances of this decade. As a type of immunotherapy, CAR T treatment consists of collecting patient-derived immune T cells, and genetically engineering them to recognize and fight invasive cancer cells. The first of these therapies to receive U.S. Food and Drug Administration (FDA) approval, Novartis’ Kymriah®, and Gilead’s Yescarta® garnered international attention for their astonishing success rates in clinical trial.
CAR T therapies are now making news once more. At this year’s meeting of the American Society of Hematology (ASH) in Orlando, Fla., Gilead Sciences presented data  from a long-term Phase 2 clinical study. The study showed that among patients who had received Yescarta® at least 3 years ago, nearly half (47%) were still alive at a 39-month follow-up study. The data is being touted as an “astounding” result for patients with relapsed or refractory large B cell lymphoma. If left untreated, people with the disease have a life expectancy of 3-4 months. 
Following the approval of Yescarta® in 2017, the drug has faced some difficulties. It has been criticized for high prices and slow adoption into practice. Developing personalized, cell-based medicines is an expensive proposition, and reimbursement challenges through Medicaid and Medicare have slowed pick up despite interest on behalf of doctors and their patients.
Other challenges also face the adoption of cell therapy products into mainstream medicine; access to the raw materials for these innovative products relies on voluntary donations, from both patients and healthy donors. Sourcing cell therapy starting materials requires reliable access to a large network of diverse and highly-characterized donors. It’s no coincidence that HemaCare, which has provided the leukapheresis starting materials for both Yescarta® and Kymriah®, maintains the largest donor pool in the industry.
There is currently a concerted effort amongst cell therapy suppliers, developers, and regulatory agencies to improve access to high-quality, GMP-compliant cell therapy starting materials. The effort will encompass more extensive quality oversight, greater coordination between donor networks and cell therapy developers, and innovation focused on improving and expanding cell collection infrastructure.
Meanwhile, the adoption of CAR T therapies and other cell therapy products may well be reaching a tipping point. Early clinical success has prompted a surge of cell-based products into clinical trial, and investment in such products continues to grow. The FDA recently announced that it will be bulking up its cell and gene therapy staff to keep up with the field. The agency expects to be approving 10-20 new cell and gene therapies a year by 2025.
The very promising long-term survival results announced at ASH are generating another round of excitement over the promise these therapies offer, especially in view of the fact the current round of CAR T therapies is already facing up and coming competitors from developers who hope to improve on their results. With a goal of increasing efficacy in patients while improving cost efficiency, cell therapy is likely here to stay.
- Helfand C. ASH: Gilead Sciences touts 'astounding' Yescarta® survival results at 3 years. Fierce Pharma. Dec 2019.
- Pfreundschuh M., et al. CHOP-like chemotherapy plus rituximab versus CHOP-like chemotherapy alone in young patients with good-prognosis diffuse large-B-cell lymphoma: A randomised controlled trial by the MabThera International Trial (MInT) Group. Lancet Oncol. 7:379–91. 2006.