CAR-T therapies should be made by academic medical centers – STAT

Draw blood from someone with cancer. Engineer their blood cells to seek and destroy cancer. Reinfuse the cells and watch the cancer melt away. Chimeric antigen receptor T cell therapy (CAR-T) sounds like science fiction. But its the next frontier in cancer therapy.

Were weaponizing individuals immune systems to destroy cancer and add years to their lives. Its incredibly exciting. But at hundreds of thousands of dollars per dose, insurance companies and the U.S. government are struggling to figure out how to pay for these breakthrough treatments.

High prices not only pose a challenge to patient access, but they also raise a fundamental question: Are we creating these therapies the wrong way?

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CAR-T immunotherapy is being tested for a range of cancers, and now holds the potential to benefit more than 1 million Americans who live with or are in remission from blood cancers. A single dose typically costs around $400,000. What makes the price so high? Having drug corporations make the cells.

Under the current system, a hospital extracts blood cells from a patient and sends them to a drug companys manufacturing plant to be genetically engineered. It takes the company two to six weeks to engineer the cells, increase their number, perform quality and safety tests, and ship them back to the hospital to be reinfused into the patient. Under this system, there have been well-documented problems with the engineering process, as well as with shipping and handling.

Theres a better way, one that will lower the price, enable more precise and individualized targeting for specific patients, and allow for a faster process: Let medical centers do this.

Long before pharmaceutical companies took control of CAR-T, medical centers made these treatments. Cancer centers like the University of Pennsylvania, the National Cancer Institute, Memorial Sloan Kettering Cancer Center, Fred Hutchinson Cancer Center, Baylor University, and others figured out how to engineer CAR-T cells and ran the initial trials to test them. Drug companies were later involved mainly as a means to scale up production.

There are clear advantages to keeping CAR-T treatment in medical centers, closer to patients.

First, academic medical centers are well-equipped to make these therapies. They already safely handle stem cells every day. They also routinely perform autologous stem cell transplants (these use a patients own stem cells), which require in-house doctors and specialists to preserve and protect stored blood cells, wash and radiate them if needed, and sometimes select cell populations for clinical use.

Hospitals and academic medical centers can accomplish the CAR-T process more quickly because they do not need to ship cells to and from a drug company plant. This is a critical issue, because most patients currently treated with CAR-T have not responded to other treatments and their health is seriously compromised; some patients die between the time stem cells are removed from them and the time theyre supposed to come back to the hospital for re-infusion.

Second, there are different regulations around stem cells than there are for drugs. That means CAR-T therapies created at hospitals and academic medical centers would not have to go through the yearslong FDA regulatory process. And the treatments developed would allow hospitals to target more effectively and quickly each individuals specific cancer, which is off the table in the current system. Patients with advanced cancer do not have the luxury of time. Although this paradigm shift would require greater regulatory flexibility from the FDA, it would make CAR-T therapy far more effective.

Experience making CAR-T in leading medical centers show that locally engineered CAR-T cells can be made for less than half of what they are currently priced by pharmaceutical companies. We know this is possible because Switzerland is already doing it this way, and pricing CAR-T therapy between $150,000 and $200,000. Multiply that by the 10,000 individuals in the U.S. with the types of cancer for which CAR-T therapy is currently approved and we would save almost $2 billion a year. Factor in future CAR-T approvals and we will save many times that amount.

Making this change requires bucking the system. It means the FDA must redefine CAR-T from a drug to the autologous blood product it is. Its a move that would save not only money but lives because it can target cancer better and destroy it faster.

We took a fork in the CAR-T road a few years back and went the wrong way. There is still time to change course for the good of the many Americans who need this lifesaving treatment.

David Mitchell, who is living with incurable blood cancer, is the founder of Patients for Affordable Drugs. Saad Kenderian, M.D., is a physician-scientist and assistant professor of medicine and immunology at the Mayo Clinic in Rochester, Minn. S. Vincent Rajkumar, M.D., is a hematologist and professor of medicine at the Mayo Clinic. The views expressed are the authors personal views and do not necessarily reflect the policy or position of the Mayo Clinic.

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CAR-T therapies should be made by academic medical centers - STAT