FDA Approves CAR T-Cell Therapy for Leukemia

FDA Approves CAR T-Cell Therapy for Leukemia

The US Food and Drug Administration approved a new leukemia treatment, which the agency considers the first gene therapy it has cleared to hit the market in the United States.

An FDA panel in July voted unanimously to recommend the therapy.

"We're entering a new frontier in medical innovation with the ability to reprogram a patient's own cells to attack a deadly cancer", FDA Commissioner Scott Gottlieb, MD, said in the release. "New technologies such as gene and cell therapies hold out the potential to transform medicine".

"We believe this treatment can change the world", says Tom Whitehead, who frequently speaks about his daughter's experience and testified before the FDA about the treatment.

The primary efficacy analysis was based on phase II results from CCTL019B2202 (B2202), a single-arm, global trial of 63 patients who received a single dose of tisagenlecleucel.

Based on available data, patients on the treatment have had an 89% chance of surviving at least six months and a 79% chance of surviving at least a year, with most being relapse-free at that point.

Researchers detected no minimal residual disease among responding patients.

Investigators concluded that tisagenlecleucel was associated with clinically meaningful remissions. Median duration of response was not reached at a median follow-up of 4.8 months. "More research is needed to make this therapy more effective for a broader population, to reduce the severe side effects that patients experience during treatment, and ultimately to find a broader application beyond blood cancers".

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Sixty-eight patients were infused with tisagenlecleucel, and 63 were evaluable for efficacy. Other possible severe side effects could include serious infection, low blood pressure, kidney injury, fever and decreased oxygen.

In its registrational ELIANA trial, 49% of patients treated with Kymriah experienced grade 3 or 4 CRS.

Juno Therapeutics Inc past year reported a handful of patient deaths during trials of its CAR-T therapy for leukemia.

Kymriah is the first gene therapy in the US that works with a person's own blood cells to destroy childhood leukemia, ushering in a new approach to the treatment of cancer and other serious and life-threatening diseases.

The drug has shown promising remission and survival rates in clinical trials, the FDA said.

The treatment, to be sold under the name Kymriah, was approved for patients up to 25 years of age who have relapsed or were not helped by initial treatment for B-cell acute lymphoblastic leukemia.

Tisagenlecleucel is the first auto T-cell therapy to demonstrate early, deep and durable remission in this patient population, and it likely will become the new standard of care, according to Stephan A. Grupp, MD, PhD, Yetta Deitch Novotny professor of pediatrics at Perelman School of Medicine at University of Pennsylvania and director of the Cancer Immunotherapy Frontier Program at Children's Hospital of Philadelphia.

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