First Gene Therapy Treatment Approved

Aug 30, 2017

The approved CAR T gene therapy treatment, now known as Kymriah, received early funding from ACGT in 2004, while in development by Dr. Carl June of the University of Pennsylvania. Novartis ultimately partnered with Dr. June and the Penn Medicine team to fund clinical trials that showed an exceptional 83% success remission rate with pediatric and young adult patients with a form of acute lymphoblastic leukemia (ALL). With this approval, cancer centers will be established around the country to make this specialized therapy readily available to relapsed, refractory ALL patients with no other treatment options.

Congratulations to gene therapy pioneer Dr. Carl June, ACGT Research Fellow/Scientific Advisory Council Member, who led the research and trials leading to this breakthrough.

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