First Gene Therapy For Leukemia Approved By FDA

Phillip Cunningham
September 1, 2017

Although China has approved a gene therapy cancer drug and two gene therapy treatments for inherited genetic diseases are on the market in Europe, this is the first to win approval from FDA.

The first cancer drug approved by US regulators to use a patient's own cells to fight common childhood cancers is priced at a whopping $475,000. The historic action makes the first gene therapy available in the United States. Clinical trials of the "living drug" showed 83 percent of patients who received the treatment were free of cancer within three months.

"This is a turning point in the fight against B-cell ALL that opens up opportunities for patients across the world who desperately need new options", said Carl June, MD, the Richard W. Vague Professor in Immunotherapy in the department of Pathology and Laboratory Medicine in Penn's Perelman School of Medicine and director of the Center for Cellular Immunotherapies in the Abramson Cancer Center. The price is well below the $700,000 that was estimated before the approval of the drug.

The drug Kymriah was developed by Novartis Pharmaceuticals and the University of Pennsylvania.

However, this type of therapy carries risk of severe side effects.

"The therapy", STAT reports, "is made by harvesting patients' white blood cells and rewiring them to home in on tumors". For others, Kymriah serves as a "bridge to transplant", keeping them alive longer enough to undergo this therapy.

The treatment process, known as auto T-cell therapy, will be used to fight the recurrence of the B-cell where cancer has not responded to treatment or returned after initial treatment, something that occurs in approximately 15-20% of patients.

"The Novartis approval is also a momentous milestone in the path for cell therapies reaching patients - the final objective of those of us involved in developing therapies", said Catherine Bollard, President, ISCT and invited for the FDA Oncologic Drugs Advisory Committee Novartis CAR-T decision as an "invited subject matter expert". "The established path to commercialization taken by Novartis will provide considerable motivation for cell therapy scientists and development companies as well as investors looking to benefit from the ongoing success in the industry".

Kymriah does have side effects, causing cytokine release syndrome, a systemic response to the proliferation of vehicle T-cells that can cause flu-like symptoms such as a high fever, and neurological events. Penn and Novartis are also investigating the next generation of vehicle therapies for multiple myeloma, and for solid tumors, through trials in glioblastoma, mesothelioma, and ovarian and pancreatic cancer.

Epstein said he envisioned cell therapies having much shorter life cycles than traditional drugs. Instead of getting an updated therapy that works better on a disease every decade or so, we might begin to see second-generation cell therapies in just a few years.

For one, the treatments would have to treat more types of cancer than the one Kymriah was approved for on Wednesday. "The lessons learned from the clinical trials of vehicle T-cell operations will assist us in being able to provide to the larger population". And previous year, Juno said five people in its clinical trials had died, all from cerebral edema.

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