Mayo Clinic researchers expand CAR-T cell therapy options

Volume 9, Issue 3, December 2020

Summary

A new facility to produce clinical-grade immunotherapies can lower the cost and increase patient access.

Photograph of Hong Qin, M.D., Ph.D.

Hong Qin, M.D., Ph.D.

The first time Hong Qin, M.D., Ph.D., saw images of how chimeric antigen receptor (CAR)-T cell therapy obliterated a tumor, he was captivated and inspired by this revolutionary treatment. As the director of Regenerative Immunotherapies at Mayo Clinic in Florida, Dr. Qin and his team are now playing a pivotal role in accelerating the latest CAR-T cell and other regenerative immunotherapy discoveries from bench to bedside to address unmet patient needs.

Dr. Qin's team is working with the Mayo Clinic departments of Hematology and Oncology, the Center for Regenerative Medicine and Mayo Clinic Cancer Center to develop first-in-class CAR-T cell products, procedures and treatments. The goal is to expand regenerative immunotherapy options to treat more types of cancers and potentially to treat neurological and autoimmune disorders.

"When I was in medical school, I was taught that blood cancer was incurable," Dr. Qin said. "But with CAR-T cell therapy, a significant number of patients with B-cell acute lymphoblastic leukemias can survive long term without the tumor coming back." Therapeutic benefits like this are unparalleled, and they may go beyond traditional chemotherapy or radiation therapy, he said. "I believe that CAR-T and regenerative immunotherapies in general will open a new era for Mayo Clinic to treat cancer patients."

Immunotherapies unleash the body's defense mechanisms to fight bacteria, viruses and diseases, including cancer. CAR-T cell therapy seeks to harness the power of the immune system by genetically modifying cells, essentially equipping them to go on search-and-destroy missions to kill cancer. These engineered cells act like a living drug continually working within the body to cure disease.

The key hurdles to bringing CAR-T cell therapy to more patients are cost and access. It's expensive and there are long waits for clinical trials. Mayo Clinic is addressing those hurdles through CAR-T translational research being conducted by Dr. Qin.

New laboratory space will be made available in Florida for discovery science with the hope of attracting researchers with innovative ideas for developing new regenerative immunotherapy products unique to Mayo Clinic. For example, could scientists identify CAR-T therapies with fewer side effects that are easier on patients? Could investigators discover ways to apply CAR-T cell therapy to solid tumors, providing new treatment options for many more types of cancer?

Mayo Clinic is one of only a few medical research centers that have made significant investments in facilities where clinical-grade biotherapies can be manufactured on-site. The new Discovery & Innovation Building on the Florida campus will deploy Current Good Manufacturing Practices (cGMP) in a facility where new patient-ready immunotherapies can be manufactured under strict, sterile, quality-control measures that meet U.S. Food and Drug Administration guidelines. That could eventually increase patient access to CAR-T and other regenerative immunotherapies through clinical trials and could lower the cost through in-house supply.

"With the cGMP facility we can help move new discoveries toward an initial clinical trial in order to perform critical first-in-man evaluation," Dr. Qin said. "This platform is so important because it empowers us to readily translate discoveries to the patient."

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