Juno’s CAR-T Cancer Trial on Halt After Two Deaths
The most important clinical trial of breakthrough chimeric antigen receptor T-cell (CAR-T) based cancer therapy has been put on hold by the U.S. Food and Drug Administration (FDA) after the death of two patients.
The FDA put hold on the Phase II clinical trial of JCAR015, reportedly called as ROCKET, which was being conducted on adult relapsed or refractory B-cell acute lymphoblastic leukemia patients. The step was taken after two patients died last week which apparently was treatment-related.
Both the deaths were faced by patients under 25 years of age affected with cerebral edema. On the common grounds, both the patients were on the preconditioning regimen of fludarabine. Though earlier, one death was reported but action wasn’t taken owing to other reasons.
Juno Pharmaceuticals certainly suspects that it is due to the fludarabine preconditioning regimen which caused the problem. Thus, it has proposed to the FDA that it could continue the ROCKET trial using JCAR015 with the cyclophosphamide preconditioning only. Previously, the company had started the trial using cyclophosphamide only but had added fludarabine later.
According to the reports, the FDA has asked Juno to submit four documents: a revised patient informed consent form, a revised investigator brochure, a revised trial protocol and a copy of the presentation to the agency.
As per Juno, FDA would take 30 days at most in order to expedite its review. This will take place only after the company submits these documents by next week.
Figure 1. Key components of Juno CAR technology (photo credit: Juno Therapeutics).
Juno’s CAR-T technology takes the patient’s own T cells and then engineers them genetically in order to recognize and combat cancer cells. It is used in combination with standard chemotherapy that includes fludarabine and cyclophosphamide.
Juno’s trades have certainly dropped by more than 25% on news of the trial halt.
Featured image credit: JEREMY DWYER-LINDGREN © bizjournals.com