FDA Panel Recommends Approval

Today a Food and Drug Administration (FDA) panel unanimously recommended that the agency approve a treatment that genetically alters a patient’s own cells to fight leukemia. If the agency does make the approval, the first-ever such treatment will be the start of the “living drug” era of human medicine, in which we harness technology to boost our natural immune system and improve its ability to master formerly unbeatable diseases.

While this gene therapy treatment for leukemia, known as CTL019, will be the first to reach the market, there are more on the way. Treatments for an aggressive type of brain tumor, as well as myeloma and other varieties of leukemia, are also in development.

This technique is a true example of personalized medicine: a unique version of the treatment must be created for every patient from their own cells. After the cells are removed by medical personnel, they are frozen, shipped to Novartis (the therapy’s maker), processed, refrozen, and shipped back to the medical center.

Assuming this treatment is approved, it will be a first for the FDA. Though the approval wouldn’t necessarily be surprising in light of the results of the trials, which were stunning: patients facing death after all other treatments failed who received just a single dose of the gene therapy experienced long remissions that could, in time, prove curative. The FDA panel therefore recommended approval for treatment of relapsed or treatment-resistant B-cell acute lymphoblastic leukemia in children and young adults ages 3 to 25.