Yesterday, the Food and Drug Administration (FDA) announced that it had approved Kymriah as the first gene therapy procedure for use in the United States. Kymriah is an immunotherapy that is intended to treat B-cell acute lymphoblastic leukemia—an aggressive form of cancer that has shown itself to be particularly resistant to existing treatments. MIT Technology Review gives an excellent breakdown of the procedure and its implication:

The therapy, which will be marketed as Kymriah by Novartis, is a customized treatment that uses a patient’s own T cells, a type of immune cell. A patient’s T cells are extracted and cryogenically frozen so that they can be transported to Novartis’s manufacturing center in New Jersey. There, the cells are genetically altered to have a new gene that codes for a protein—called a chimeric antigen receptor, or CAR. This protein directs the T cells to target and kill leukemia cells with a specific antigen on their surface. The genetically modified cells are then infused back into the patient.

In a clinical trial of 63 children and young adults with a type of acute lymphoblastic leukemia, 83 percent of patients that received the CAR-T therapy had their cancers go into remission within three months. At six months, 89 percent of patients who received the therapy were still living, and at 12 months, 79 percent had survived. 

An estimated 3,100 patients aged 20 and younger in the U.S. are diagnosed with acute lymphoblastic leukemia each year, making it the most common childhood cancer, according to the National Cancer Institute. Current treatment options include chemotherapy and stem-cell patients, but about 600 pediatric and young adult patients with the disease relapse each year, and many patients remain incurable.

This is a monumental moment for precision medicine and genetic modification, and will hopefully mark the beginning of great things to come. As FDA Commissioner Scott Gottlieb noted in the Agency’s press release:

“We’re entering a new frontier in medical innovation with the ability to reprogram a patient’s own cells to attack a deadly cancer. … New technologies such as gene and cell therapies hold out the potential to transform medicine and create an inflection point in our ability to treat and even cure many intractable illnesses. At the FDA, we’re committed to helping expedite the development and review of groundbreaking treatments that have the potential to be life-saving.”

Between yesterday’s approval, the FDA’s decision earlier this week to produce a framework for regenerative medicine, and Dr. Soukhrat Mitalipov’s experiment in human embryonic genetic modification from earlier this month, we could be on the cusp of a true revolution in applied genomic science. So long as Commissioner Gottlieb continues embracing the need for a more innovation-friendly FDA, the future of genetic modification and gene therapy treatments will alleviate the pain and suffering of millions of Americans suffering from debilitating diseases. The future can’t come soon enough.