FDA Announces First US Gene Therapy Approval For Cancer Treatment
PHILADELPHIA (CBS) — This begins a new era in cancer care. The FDA has approved the first treatment that genetically engineers a patients' own blood cells and turns them into an army of assassins that destroy childhood leukemia.
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It all started with Emily Whitehead, the first pediatric patient at Children's Hospital of Philadelphia to be treated with CAR-T gene therapy in 2012, when she was just 6.
She had relapsed twice with acute lympho-blastic leukemia.
"It was scary really scary," Emily said, back in 2012.
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But the treatment worked for Emily and others .. Remission rates are now over 80 percent.
It becomes the first gene transfer therapy approved by the FDA and will be called Kymriah, developed by doctors and researchers at CHOP and the University of Pennsylvania.
"This is already changing lives of patients with leukemia," Dr. Stephan Grupp said.
The new treatment modifies the patients' own immune T cells, which are collected from the patient, reprogrammed and returned super-charged to seek and destroy cancer cells.
"Broadly speaking, ten years ago, I don't think anybody thought you could productively use the immune system to fight cancer," Dr. Grupp said. "Now, I think most doctors would agree, that in addition to chemotherapy, radiation and surgery, this is the fourth thing: immunotherapy. That's a huge difference."
It's been a long haul for Emily and her family. She suffered a variety of setbacks.
"They keep telling us that Emily is rewriting the history books because she was the fist child in the world with the immune system trained to beat her leukemia," Tom Whitehead, Emily's father, said back in 20016.
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Now history can potentially be changed for other young patients who relapse.
And the therapy is being tested for thousands of additional patients.
A therapy that started with a desperate decision.
"We were just hoping for anything because this was her last chance this was the only thing she had left," Kari, Emily's mother, said.
Emily is 12 now and feeling good.
Novartis, the company that makes the just approved therapy, says it will cost $475,000 because it's customized for every patient.
But there will be no charge if it doesn't work within a month. Expensive, but it's a one-time treatment that's been shown to be a life saver.