Tuesday, 21 November, 2017

FDA approves first-ever gene therapy treatment for acute lymphoblastic leukemia

US clears first 'living drug' for childhood leukaemia Novartis' CAR-T gene therapy, the first approved by FDA, to be priced based on cancer patients' outcomes
Jerome Frank | 31 August, 2017, 01:44

"Kymriah is a first-of-its-kind treatment approach that fills an important unmet need for children and young adults with this serious disease", said Peter Marks, M.D., Ph.D., director of the FDA's Center for Biologics Evaluation and Research, in the news release.

The immunotherapy now can be used in children and young adults with B-cell ALL that will not respond to other therapies, the FDA announced. In June, a clinical trial in China showed promise in treating another blood cancer, multiple myeloma, using reprogrammed T-cells in much the same way Kymriah does.

This first use of CAR-T therapy is aimed at patients desperately ill with acute lymphoblastic leukaemia, which strikes more than 3,000 children and young adults in the USA each year.

The disease is the most common type of childhood cancer in the USA, making up 25 percent of cancer diagnoses for children under 15 years old, according the National Cancer Institute.

With the treatments available now, fewer than 10 percent of patients with the relapsed, hard-to-treat leukemia are alive five years after diagnosis. In the USA, only a few dozen specialized hospitals are now qualified to provide CAR-T treatments, which require retrieving, processing and then returning immune cells to the patient.

"We're entering a new frontier in medical innovation with the ability to reprogramme a patient's own cells to attack a deadly cancer", said FDA commissioner Scott Gottlieb. Some 83 percent of those given the therapy were in remission within three months of treatment, the agency said.

Kymriah is a genetically-modified autologous T-cell immunotherapy.

The FDA based its decision on a clinical trial involving 63 patients with B-cell ALL.

However, tisagenlecleucel (also known as CTL019) can cause severe side effects, particularly a condition called cytokine release syndrome (CRS) that can become life-threatening.

However, this type of therapy is not without risk of severe side effects as the immune system goes into high gear.

A spokeswoman for Switzerland-based Novartis declined to say how much the drug will cost, though analysts have estimated a price of $500,000 or more.

Manufacturing and delivery are more complex in CAR-T therapies than for a typical drug. It is the first gene therapy of its kind to hit the USA market.

The others still are being tracked to see how they fare long-term. More than half were children and teens.

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