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BIG NEWS: FDA Approves Groundbreaking CAR T Cell Therapy for Kids With Leukemia

by Becky C. Weaver, Chief Mission Officer, St. Baldrick's Foundation
August 30, 2017

St. Baldrick’s supporters, this is a day to celebrate!  You have helped make history. Today, the FDA approved the first gene therapy available in the United States. Called Kymriah, it’s an entirely new way of treating cancer and it’s saving lives.

Breaking News

This “living drug” is for patients with a type of acute lymphoblastic leukemia (ALL), a cancer of the blood and bone marrow and the most common form of childhood cancer. Scientists genetically modify a patient’s own immune cells in the lab, then infuse these new cells back into the patient’s body. These modified cells – called CAR T cells or chimeric antigen receptor T cells – then prompt the child’s own immune system to attack and kill leukemia cells. 

As with any breakthrough, this progress has been years in the making.  The St. Baldrick’s Foundation has been supporting immunotherapy research since 2012, including scientists in the lab of Dr. Stephan Grupp of Children’s Hospital of Philadelphia.

Dr. Grupp treated the first child with CAR T cell therapy, a girl named Emily who is now more than five years cancer free, after once being given a slim chance of surviving the night. Dr. Grupp is also a key member of the Pediatric Cancer Dream Team, supported since 2013 jointly by the St. Baldrick’s Foundation and Stand Up to Cancer.

“This is a landmark moment for cancer research and for pediatric oncology, as a whole new kind of cancer treatment, CAR T therapy, is approved,” Dr. Grupp said. “It is amazing to me as a pediatric oncologist to see the first approval of a new treatment like this in pediatric leukemia.”

The Dream Team has been instrumental in helping to understand why the treatment works for some patients and not others, researching how to best deal with the dangerous side effects some patients experience, and helping to push the drug approval over the finish line as quickly as possible.

Read more about the Dream Team’s incredible work with CAR T cell therapy >

This new therapy “represents a watershed moment in the decades long efforts to improve outcomes from cancer,” said Dr. John Maris, a pediatric oncologist at The Children’s Hospital of Philadelphia and leader of the SU2C-St. Baldrick’s Pediatric Cancer Dream Team.  “Not only is it exceedingly rare for new drug approvals directed squarely at childhood cancers, but this drug is the first gene therapy of any kind to show dramatic and long-standing remission for patients with literally no other therapeutic options.”

“The FDA has approved today a transformative therapy for children with refractory leukemia.  It represents the first in an entirely new class of cancer therapies that holds promise for all cancer patients.  The support of the St. Baldrick’s Foundation during the development of this therapy was indispensable for teaching physicians how to render it as effectively and safely as possible.”  Dr. Crystal L. Mackall, Professor of Pediatrics and Internal Medicine at Stanford University, associate director of Stanford Cancer Institute, and co-leader of the Stand Up to Cancer – St. Baldrick’s Pediatric Dream Team.

This CAR T cell therapy is groundbreaking in more ways than one.

First, Kymriah’s success is stunning. Given to children and young adults whose leukemia could not be cured by any other means, the treatment produced remission within three months for 83% of patients.  It has already saved the lives of many children, like 2016 St. Baldrick’s Ambassador Phineas.

Second, it was approved first for children, adolescents and young adults.  Almost all new therapies are approved first for adults, and eventually, maybe, for kids.  Not this time!

And last, it is only the beginning of a whole new era that can change everything – for children and adults.

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

St. Baldrick’s supporters have played a key role in bringing all this to reality.

“This is a remarkable achievement and a hallmark day for our community and our children most importantly,” said Kathleen Ruddy, CEO of the St. Baldrick’s Foundation. “We are so grateful for the many scientists who have labored to realize this day, and for every donor and volunteer who has helped to make this possible. We will continue to support the best childhood cancer research, no matter where it is happening, to make progress like this available to patients with all types of childhood cancers.”

Join us and be a part of revolutionary research like this. Donate today.

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