By Jonathan Springston, Editor, AHC Media

The FDA on Wednesday approved the first gene therapy in the United States, which could help researchers open doors to new cures for cancer and other life-threatening illnesses.

Kymriah (tisagenlecleucel) is a cell-based gene therapy approved for use in patients up to 25 years of age suffering from B-cell precursor acute lymphoblastic leukemia, a cancer of the bone marrow and blood, that is refractory or in second or later relapse. It’s a customized regimen grounded in an individual patient’s unique T-cells. Clinicians collect T-cells and send them to a manufacturing center where technicians modify those cells with a chimeric antigen receptor. This new blend targets and kills leukemia cells that feature the CD19 on the surface.

“Kymriah is a first-of-its-kind treatment approach that fills an important unmet need for children and young adults with this serious disease,” Peter Marks, MD, PhD, director of the FDA’s Center for Biologics Evaluation and Research (CBER), explained in a statement. “Not only does Kymriah provide these patients with a new treatment option where very limited options existed, but a treatment option that has shown promising remission and survival rates in clinical trials.”

Although the treatment demonstrated promising results in one multicenter trial, the FDA granted approval with some caveats. Because of some severe side effects, specifically cytokine release syndrome, the agency placed a boxed warning on Kymriah. Also, because of other potential neurological events, the treatment will include a risk evaluation and mitigation strategy, which will help clinicians administer the regimen safely.

Nevertheless, the release of this treatment regimen marks a big step forward in the field of immunotherapy.

“We’re entering a new frontier in medical innovation with the ability to reprogram a patient’s own cells to attack a deadly cancer,” FDA Commissioner Scott Gottlieb, MD, said in a statement. “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.”

For more information about Kymriah and immunotherapy, check out the National Institutes of Health’s interesting back story on the evolution of this treatment strategy.