FDA Panel Recommends Approval for the First Gene Therapy in Leukemia Treatment


A FDA panel opened a new era in medicine unanimously recommending that the agency approve the first-ever treatment that genetically alters a patient’s own cells to fight cancer, transforming them into what scientists call “a living drug” that powerfully bolsters the immune system to shut down the disease.

The treatment will be The First Gene Therapy ever to reach the market throw FDA approval. 

A single dose of the resulting product has brought long remissions, and possibly cures, to scores of patients in studies who were facing death because every other treatment had failed. The panel recommended approving the treatment for B-cell acute lymphoblastic leukemia that has resisted treatment, or relapsed, in children and young adults aged 3 to 25.

In 2012, as a 6-year-old patient was treated in a study at the Children’s Hospital of Philadelphia. Severe side effects — raging fever, crashing blood pressure, lung congestion — nearly killed her. But she emerged cancer free, and has remained so. 

Her father said to FDA comitte“I hope that someday all of you on the advisory committee can tell your families for generations that you were part of the process that ended the use of toxic treatments like chemotherapy and radiation as standard treatment, and turned blood cancers into a treatable disease that even after relapse most people survive.”

The main evidence  presented by the sponsor to the FDA came from a study of 63 patients who received the treatment from April 2015 to August 2016:

  • 52 of them, (82.5%) went into remission — a high rate for such a severe disease.
  • 11 of them died

As NY times sahred on their interview: “It’s a new world, an exciting therapy,” said Dr. Gwen Nichols, the chief medical officer of the Leukemia and Lymphoma Society, which paid for some of the research that led to the treatment. The next step,  will be to determine “what we can combine it with and is there a way to use it in the future to treat patients with less disease, so that the immune system is in better shape and really able to fight. This is the beginning of something big.”