FDA Approves CAR-T Cell Treatment for Pediatric Patients with Acute Lymphoblastic Leukemia
This week marks a huge milestone for pediatric cancer research.
The U.S. Food and Drug Administration (FDA) announced Wednesday that is has approved Novartis CAR-T cell therapy CTL019—now Kymriah—for pediatric patients with acute lymphoblastic leukemia (ALL)! The decision comes after an advisory committee unanimously voted in favor of approval at a meeting on July 12 — at which Emily Whitehead Foundation co-founder Tom Whitehead spoke.
The Whitehead family shares the following statement in response to this historic announcement:
Over the past five years, we have been focused on doing everything we can to move T-cell therapy toward FDA approval. We believed in this therapy from the very beginning, and it is incredibly exciting to see it granted FDA approval.
Not only is this the first gene cell therapy in the world to be approved by the FDA but also it was approved for children first. This almost never happens as most therapies are approved for adults first while children are left as an afterthought. We believe more therapies like this will be approved in the coming months and years and we hope that this immunotherapy revolution continues for many other cancers to become treatable and cured.
We are so proud that Emily is a pioneer for this treatment, which provided families with hope and gave children a chance to survive and live normal lives. We are immensely grateful to and inspired by Dr. Stephan Grupp, Dr. Carl June, Dr. Bruce Levine, and the many, many others who were involved in moving this therapy forward. Words will never be enough to tell this brilliant and compassionate team of doctors and researchers how thankful we are to them.
While Emily’s story is well known worldwide, there are many other stories to tell. Families who, like us, were told that there were no other options to treat their child’s leukemia. We are inspired by these families whose children also became pioneers of this treatment. Doctors learned something from every single child who received T-cells.
We believe that every child’s story deserves to be told. It is for this reason that our inaugural Believe Ball’s theme is “Write a Better Story”. We want everyone to know the bravery of all children fighting cancer. We want to remember those children who fought valiantly yet lost their lives to this monster of a disease. Children deserve so much more and we want to write a better story for them. One that ends with a cure.
It has been incredible to be part of this journey. It isn’t often in life that you get to be part of something that truly changed lives.
Tom and Kari Whitehead, Co-founders
About CAR-T and Emily Whitehead
In May 2012, Emily Whitehead became the first child in the world to receive CAR-T cell therapy, treated at Children’s Hospital of Philadelphia (CHOP). The treatment was developed by a team of researchers at Penn Medicine, led by Dr. Carl June, in collaboration with Novartis. Today, Emily is five years cancer free. In the years since, the trial has expanded in the U.S. and around the world.
Read Emily's story, as shared by Parker Institute for Cancer Immunotherapy.
Acknowledgements in the Media
Emily keeps smiling. Her smile gives me hope. Seeing her grow from a young girl struggling with a frightening disease into a poised young woman who looks forward to starting the 7th grade, but still spends time to be an ambassador for immunotherapy, is one of the greatest joys I’ve had as NIH Director.
FDA Approves First-of-Its-Kind Cancer Treatment — WebMD (August 30, 2017)
FDA approves first-ever T-cell cancer treatment, pioneered at Penn, CHOP — Philly.com (August 30, 2017)
New breakthroughs in cancer research — ABC World News Tonight (August 30, 2017)
FDA Approves First CAR-T Cell Therapy for Pediatric Acute Lymphoblastic Leukemia — NIH Director’s Blog (August 30, 2017)