FDA approves gene therapy that could fight type of leukemia

Charlotte Gifford is almost 5 years old with dreams of looking like her favorite Disney princess.

“Rapunzel, because she has the long hair,” says Charlotte.

Charlotte lost her own long locks after being diagnosed last March with b-cell acute lymphoblastic leukemia, the most common form of leukemia to develop in children and young adults.

“Once we got the word it was leukemia. Our world came down… it crumbled,” says Erica Gifford, Charlotte’s mom.

Erica and Adam Gifford are thrilled the Food and Drug Administration announced approval of the first gene therapy in the United States. Car t-cell gene therapy can be used for Charlotte’s type of leukemia, affecting  anyone under 25 years old.

“It’s a new treatment with an entirely different flavor,” says Dr. Daniel Weisdorf, Professor of Medicine and Chief Division of Hematology, Oncology and Transplantation. “It’s not a drug, but a way to take the patient’s own cells, their normal cells, and engineer the cells so they will specifically and uniquely attack the leukemia cells.”

Weisdorf witnessed success from this treatment, as some patients from University of Minnesota Health and Masonic Cancer Center were part of the clinical trials.

“The treatments are remarkably effective but complicated to administer because the patients get very sick,” says Weisdorf.

The Leukemia and Lymphoma Society has helped fund research for decades, and today, Executive Director Teri Cannon is celebrating a major medical milestone.

“Nowadays 90 percent of young people diagnosed with leukemia survive,” says Cannon, “Those kids that relapse and standard therapy doesn't work for them, 83 percent of the kids who have used car t-therapy in the clinical trial have survived. So that's going to bring us a lot closer to that 100 percent that is going to make parents happy.”

As for Charlotte, she's doing well and in remission. Hopefully she'll never need the this newly approved gene therapy reserved for patients whose cancer has returned. For the Giffords, this major medical advancement offers their family and others options…and hope.

“You don’t know how important it is until your own child is diagnosed with cancer,” says Gifford.

Charlotte continues steroid, chemo and physical therapy. Her parents have started a Go Fund Me Page.