“It’s more effective than any known treatment for these highly resistant refractory cancers. It pioneers a trail for the future of cancer regulation and future therapies in this class. We were one of the first sites since the study began to get involved.”
Children’s Mercy patients benefit from first FDA-approved cancer gene therapy
KC Business Journal:
Children’s Mercy contributed to a trial of the first U.S.-approved cancer gene therapy, which the Food and Drug Administration announced Wednesday.
Novartis Pharmaceutical Corp.'s new CAR-T therapy, Kymriah, is for children and young adults with acute lymphoblastic leukemia that is resistant to treatment or has relapsed.
“It’s more effective than any known treatment for these highly resistant refractory cancers. It pioneers a trail for the future of cancer regulation and future therapies in this class,” said Dr. Doug Myers, a hematologist-oncologist and associate professor of pediatrics at Children’s Mercy.
“We were one of the first sites since the study began to get involved.”
Acute lymphoblastic lymphoma is the most common childhood cancer, with about 3,100 patients younger than 20 diagnosed every year, according to the National Cancer Institute.
Children’s Mercy has treated six patients with Kymriah since the multisite clinical trial opened in 2014. Myers said outcomes were in line with the broader study results, which showed 83 percent remission rates after one dose of Kymriah in the trial's 63 participants.
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The leukemia kept coming back and Lucas Novick was running out of options.
In 2015, Novick had already had chemotherapy and a bone marrow transplant, but had relapsed a second time. He was 25 years old and worried he wouldn’t live to see 30.
“At that point in time it was like, ‘Well, we can’t do (another) transplant, we’ll probably do chemotherapy, but it’s not looking good,” Novick said.
Novick was thrown a lifeline in the form of a cutting-edge gene therapy treatment at Children’s Mercy Hospital in Kansas City.
Dr. Doug Myers, a pediatric oncologist at Children’s Mercy, said CAR-T should not be considered a “cure for cancer.” There’s not nearly enough data. But what researchers have seen is encouraging.
“There really isn’t anything else out there that has provided this duration of remission for this many patients for this particular disease,” Myers said. “That’s why it’s so exciting.”
Children’s Mercy has hosted six patients for the CAR-T trial. Every one of them has gone into remission.
Learn more about the Children's Research Institute at Children's Mercy.
Learn more about the Children's Mercy Cancer Center.