FDA votes to recommend gene-therapy leukemia treatment

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The FDA doesn't have to follow advisory panels' recommendations.

Novartis' groundbreaking cancer T-cell therapy CTL019 has won unanimous backing from advisers to the FDA, paving the way for approval in the coming months.

The gene therapy, also known as CTL019, uses a new technology called chimeric antigen receptor T-cell therapy, or CAR-T for short. "This is a major advance, and is ushering in a new era", said Malcolm Smith, a paediatric oncologist at the National Institutes of Health in Bethesda, Maryland. In a news release, the drugmaker said that it "continues to invest in the necessary infrastructure for the potential commercialization of CTL019, including manufacturing and the establishment of a network of certified treatment centers".

"Our daughter was going to die and now she leads a normal life", said Tom Whitehead, of Philipsburg, Pennsylvania.

Another patient, 12-year-old Emily Whitehead, was present at the panel's meeting, along with her parents, to push for approval. Around 60 percent of them are young adults and children. Effective treatment options are limited, and in paediatric and young adult patients with B-cell ALL that have relapsed multiple times or become refractory to treatment, the five-year disease-free survival is less than 10-30 percent. That happens to more than 600 patients in the US each year.

Other CAR-T therapies are on the horizon, including a promising one from Kite Pharma.

The FDA also discussed mandating a patient registry that would track patients for up to 15 years.

In a key test, results were far better than chemotherapy and even newer types of cancer drugs. Other pharmaceutical companies developing CAR-T therapies are Juno Therapeutics (JUNO), and Bluebird bio (BLUE) in collaboration with Celgene (CELG). These include the possible risk that the virus used to engineer the cells could acquire the ability to replicate, or that improper insertion of the foreign gene could turn the T cells cancerous. That means taking out the cells, reengineering them, reaching a critical mass of genetically modified cells, and then putting them back into the patient-all of which can take several weeks to complete.

The approval of the drug is likely, especially in light of the positive results of experiments in the field of cell modification to fight diseases. So the researchers also equipped them with interleukin-15, a protein created to help them stick around in the body for many months.

This revolutionary new method of treating blood cancer is customized to target a specific type of cancer, that is, it is adjusted according to the DNA information of a particular patient.

If approved by the FDA, CAR-T therapies could cost up to $500,000 and generate billions of dollars for their developers. Speaking during the public hearing, Megan Polanin, a senior fellow at the National Center for Health Research who specializes in evidence-based mental health interventions, urged the FDA to take it slow in approving the vehicle T therapy.

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. She also noted that once the therapy is marketed, patients will be lost to the follow-up that's needed to determine long-term effects. ACGT believed so strongly in cell and gene therapy and moved to embrace the science, that it focused all its resources on helping scientists we thought had the most exciting, novel ideas on how to treat cancers.