FDA panel endorses first gene-altering therapy for leukemia patients

Emily Whitehead with her father Tom Whitehead

The Food and Drug Administration advisory panel voted 10-0 on Wednesday to recommend approval of the treatment developed by the University of Pennsylvania and Novartis Corp.

The agency is not required to follow its committees' recommendations, but it usually does. "We are hopeful about the promise of immunotherapy approaches for cancer patients, who deserve new and more effective treatments".

"This is a major advance", said panel member Dr. Malcolm A. Smith of the National Cancer Institute. Patient advocacy groups praised the FDA's findings and the impact the new treatment could have on patients with ALL.

"We believe that when this treatment is approved, it will save thousands of children's lives around the world", Emily's father, Tom Whitehead, told the panel. While patients on this engineered vehicle cell therapy have been repeatedly hit by potentially lethal cases of cytokine release syndrome and neurological toxicity with the threat of new malignancies, advisers were clearly willing to accept the risk in order to achieve the potential gains.

"While CAR-T is a promising new type of immunotherapy, it is not commercially available and we have yet to complete our evaluation", said T.J. Crawford, a spokesman for Aetna Inc.

The treatment from Novartis would be the first of its kind and is targeted to children who are out of options. The drugmaker is seeking approval to use the treatment for patients aged 3 to 25 with a blood cancer called acute lymphoblastic leukemia whose disease has spread or failed to respond to standard treatment.

More than 600 patients in the USA each year reach that critical stage. ALL accounts for a quarter of all cancers in children under age 15.

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Typically, young patients with ALL who fail chemotherapy only have up to a 30 percent chance of survival. Eleven patients died - four from side effects and seven from the cancer. To do that, researchers use gene therapy to change patients' bone marrow cells, which make blood and immune cells, to recognize cancer cells.

Samit Hirawat, Novartis' head of oncology global development, explained the process by which the treatment is created with patients' own cells that are transferred from the patient, moved to a dedicated manufacturing facility in Morris Plains, New Jersey where the cells undergo enrichment and are then cryopreserved and returned to the clinical site where patients are infused. The whole testing process took 16 weeks on average, which FDA advisers noted could be too long for severely ill patients.

CAR-T therapies also present a gnarly regulatory challenge for the FDA: how do you assure the potency and purity of a complex, living drug that must be made fresh for each patient?

Vehicle T cell therapies, however, have led to several deaths in the past. Dr. Carl June, leader of the Penn research team, said, "It is encouraging to see the FDA panel's recommendation and continued momentum behind this innovative therapy". Novartis is also working on other gene therapies for additional types of cancer and an aggressive brain tumor.

The treatment's short-term side effects, including fever and hallucinations, are often intense as the body's revved-up immune system goes on the attack.

The vote also underscores ODAC's ingrained acceptance of moderate risk when it comes to treating patients dying of cancer. GlaxoSmithKline's Strimvelis is being targeted to treat severe combined immunodeficiency with a $670,000 price tag.

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