Stanford's CAR22 therapy granted breakthrough designation by FDA

Stanford Cancer Institute members Matthew Frank, MD, PhD, and David Miklos, MD, PhD, have received FDA breakthrough therapy designation on a trial using CAR22 to treat patients with relapsed/refractory large B-cell lymphoma that has progressed after prior CAR19 therapy.

This designation, also known as fast track, facilitates the development and expedites the review of drugs to treat serious conditions and fill an unmet medical need. To achieve this designation, preliminary clinical evidence indicates that the drug may demonstrate substantial improvement over available therapy on a clinically significant endpoint(s).

Dr. Frank is the trial principal investigator. The IND 19103 for “autologous T-cells transduced with lentiviral vector (CD22.BB.Z) expressing chimeric antigen receptor (CD22-CAR); following fludarabine and cyclophosphamide” is held by Stanford University. Dr. Miklos is the medical director for the IND.

Results for the first three subjects have already been published in the journal Blood, and Dr. Frank has presented ongoing updates at international meetings. Two or three more subjects will be enrolled in this trial where the primary endpoint requires a three-month follow-up. As such, results are expected to be published in the next six months.

Dr. Frank says, “We are very encouraged by the phase one clinical trial results demonstrating that CD22 CAR T-cell therapy is a safe and highly effective treatment that often results in durable remission for patients with large B-cell lymphoma who have progressed after prior CD19 CAR T-cell therapy. We have seen patients who were near death return to a normal life again. We are grateful that this therapy has this potential for patients and their families.”

[Image: National Cancer Institute]