In terms of history, cellular therapy is a relatively new form of medicine.
Cellular therapy was pioneered in the form of blood and marrow transplantation — the first successful transplants were performed in 1968. Since then, huge advancements have been made to the field, making these therapies more effective for treating cancers and other blood diseases.
The right direction
With the advent of Chimeric Antigen Receptor (CAR) T-cell therapy, a new form of immunotherapy that uses altered T cells to destroy abnormal cancer cells, experts believe we’re moving in the right direction. CAR T-cell therapy is widely considered one of the most promising treatments for patients battling leukemia, lymphoma, and other forms of cancer.
Dr. Navneet Majhail — director of the Cleveland Clinic’s Blood and Marrow Transplant Program and president of the American Society for Transplantation and Cellular Therapy (ASTCT) — said these therapies are transformational, and that the two FDA-approved CAR T drugs now available to treat patients with leukemia and lymphoma will lead the way for the other cellular therapies that are in various stages of development.
“There is an impressive response rate in selected patients, and we are just scratching the surface,” Majhail said. “It is a harbinger of where this field is going.”
Research in the field has accelerated drastically, too. In 2018, industry leaders from the ASTCT, the Center for International Blood and Marrow Transplantation Research, the American Society of Hematology, and the National Cancer Institute published a groundbreaking paper that aims to measure the toxicities associated with CAR T therapies.
Making treatments safe
With advancement comes another question: how should this be regulated?
“It’s a completely new branch of medicine, so it doesn’t fall within the scope of what lawmakers are used to seeing,” said Alycia Maloney, the director of government relations for ASTCT.
This year, the Centers for Medicaid and Medicare (CMS) issued proposed policy changes regarding inpatient and outpatient payment schedules for CAR T therapies. Advocacy groups are on Capitol Hill during these important decision-making moments.
Pricing is a major point of contention. As these drugs become more widely available, advocates want to make sure they’re being priced fairly. As CMS sets a precedent for how CAR T is reimbursed, advocacy groups are engaging with lawmakers to create best practices that allow access to these therapies.
“People’s lives are being saved,” Maloney said. “We’re making sure that we’re engaged in the rule-making process, because legislators are seeking advice from the people within this industry.”
Kate Jacobson, American Society for Transplantation and Cellular Therapy, [email protected]