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CSO Perspectives: A Moment to Remember: First-of-its-Kind Cancer Treatment Approved by FDA

Published on August 31, 2017 in Cornerstone Blog · Last updated 4 months ago


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It was a pivotal moment that has turned into a new era for cancer immunotherapy. On April 17, 2012, Children’s Hospital of Philadelphia researchers for the first time treated a pediatric patient with a cellular therapy that used her own reprogrammed immune cells, called T cells, to attack her aggressive form of blood cancer.

For a very sick and brave little girl, Emily Whitehead, and her parents, that moment turned into tenuous hours and then fearful days when she was on a ventilator, as CHOP physicians figured out how to manage a side effect called cytokine release syndrome. Three precious weeks later, a bone marrow test showed that Emily had completely responded to T cell therapy and was in remission. She remains healthy at age 12.

That moment was only five years ago, so it is remarkable that yesterday the U.S. Food and Drug Administration (FDA) announced the landmark decision to approve the personalized cellular therapy developed by investigators at Penn’s Perelman School of Medicine and CHOP, for the treatment of patients up to 25 years of age with B-cell precursor acute lymphoblastic leukemia that is refractory or in second or later relapse. About 20 percent of the 3,500 pediatric and young adult patients diagnosed every year with ALL in the U.S. relapse or do not respond to conventional treatment.

The approval was granted to Novartis for the chimeric antigen receptor (CAR) T-cell therapy, Kymriah™ (tisagenlecleucel, formerly CTL019). In 2012, Penn and Novartis entered into a global collaboration to further research, develop, and commercialize Kymriah and other CAR-T cell therapies for the treatment of cancers. Kymriah is the first therapy based on gene transfer approved by the FDA, and it is expected to be available through a network of certified treatment centers throughout the U.S. Novartis will create a registry to follow patients for 15 years after being treated to monitor their progress and any potential, future side effects.

Given that children are often at the back of the line for access to new cancer therapies, it is truly amazing that FDA approval for this novel therapy came so quickly and was approved first for a pediatric patient population. Much of that momentum was driven by the extremely hard work of the Penn/CHOP/Novartis collaborators and families like the Whiteheads who crusaded to bring this “living drug” to more children with ALL who had exhausted every other option.

Our research team led by Stephan Grupp, MD, PhD, the Yetta Deitch Novotny Professor of Pediatrics at Penn and director of the Cancer Immunotherapy Frontier Program and chief of the section of Cell Therapy and Transplant at CHOP, learned from each patient they treated. Researchers accumulated enough strong evidence that the FDA took note of their success and designated the CTL019 approach as a Breakthrough Therapy.

It was the first personalized cellular therapy for the treatment of cancer to receive this important classification, which helped to expedite its progress into a global registration trial in 2015, involving 68 children and young adults with advanced ALL treated at 25 centers across the world. In those trials, researchers are seeing overall remission rates over 80 percent.

An FDA advisory panel unanimously recommended approval of the therapy in July 2017, setting the stage for the FDA’s approval this week. After presentation of trial data and testimony from families whose children have received the therapy, one expert on the panel said this was “the most exciting thing I’ve seen in my lifetime.”

I wholeheartedly agree! Thank you to everyone involved in this groundbreaking research — clinical research coordinators, inpatient and outpatient nurses, and so many others — who ensured that all the pediatric patients who received the experimental therapy at CHOP experienced the most advanced care available. Behind the scenes, our Clinical Vector Core also played a key role by creating a high quality vector that made a large part of the CAR-T cell development process work.

It is because of your vision, dedication, and expertise that families battling their child’s relentless form of ALL are now looking at the possibility of a future free of cancer.

To see how far we’ve come in such a short time, look back at the beginnings of this groundbreaking therapy in this video. Read more about the FDA’s approval in this press release.