Novel ALL Immunotherapy Gets FDA Breakthrough Designation

The FDA has granted breakthrough therapy designation to a novel treatment known as CTL019.

The US Food and Drug Administration has granted breakthrough therapy designation to a novel treatment known as CTL019, in development by Novartis and researchers at the University of Pennsylvania in Philadelphia. The therapy is intended for patients with relapsed/refractory acute lymphoblastic leukemia (ALL).

“This Breakthrough Therapy designation underscores the potential of CTL019 as a life-saving therapy for patients with relapsed/refractory ALL, who are in desperate need of new treatment options," said David Epstein, a division head at Novartis, in a press release. Breakthrough designation allows for an expedited development and review process for new therapies that could yield substantial, meaningful improvements over existing treatments.

CTL019 is the first immunotherapeutic approach to receive breakthrough designation. The therapy works by reprogramming a patient’s own T cells into what are known as chimeric antigen receptor T cells. These cells, injected back into the patient, are capable of attacking cancerous B cells, specifically cells that express the protein CD19.

The breakthrough designation comes after only limited-though promising-results so far. Data from early studies were presented at the American Society of Hematology’s annual meeting in New Orleans last December. In one study, 15 of 32 adult chronic lymphocytic leukemia patients responded to CTL019.

Responses in both adult and pediatric ALL patients were much stronger. Nineteen of 22 pediatric patients with ALL had a complete remission with the agent, five of whom subsequently relapsed. The first five adult patients with ALL treated with CTL019 all experienced a complete remission, the longest of which extended for 6 months following the treatment. The relapses seen in both groups seemed to be in patients with new tumor cells that did not express CD19. A report on two pediatric ALL patients who received the drug and experienced remissions also appeared in the New England Journal of Medicine in April.

In the second phase of development, a phase II study of adult relapsed/refractory ALL is now ongoing. It will include about 67 patients with an estimated completion date of 2017.

"This is a major milestone as we are now one step closer in helping address the high unmet needs of this patient population," said Carl H. June, MD, of the Abramson Cancer Center at the University of Pennsylvania, in the press release. "We are excited about the strength of the positive early data seen in pediatric and adult patients with relapsed/refractory acute lymphoblastic leukemia and look forward to building upon these findings as we continue advancing the CTL019 clinical program in phase II trials."