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.
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The treatment, known as chimeric antigen receptor T-cell therapy, or CAR-T, involved extracting Novick’s own white blood cells, genetically modifying them into cancer-killers, and then infusing them back into his body.
It had never been done before, but scientists had high hopes gene therapy could revolutionize cancer treatments and Novick was one of dozens of patients with relapsed acute lymphoblastic lymphoma who were testing it at sites across the country.
For Novick, the results were life-changing and possibly life-saving. The Nashville musician has been in remission for two years, graduated from Belmont University in May and is planning to go to law school.
“I don’t really have anything to complain about,” Novick said. “My energy level is good. My hair hasn’t come back, which I’m slightly bitter about, but all things in perspective, I’m good.”
His results mimic the overall results of the clinical trials — an 89 percent survival rate after six months and 79 percent survival rate after a year, with most patients in remission by that point. The safety and effectiveness of the treatment so far led the U.S. Food and Drug Administration to approve it Wednesday for commercial use.
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.
But there’s questions about what happens next with a gene therapy hitting the open cancer care market for the first time.
It will be sold as Kymriah, a product of Swiss pharmaceutical giant Novartis. Novartis has set a price of $475,000 for the treatment, which drew concern from David Mitchell, a cancer survivor and founder of the advocacy group Patients for Affordable Drugs.
Wall Street analysts said that might actually be a bargain, and the company could have charged much more given the breakthrough nature of the drug. Physicians who participated in the study said it could save the health care system money if it replaces traditional treatments like bone marrow transplants that can have serious complications.
Myers said there’s going to be significant regulatory hoops to jump through before hospitals can start performing the CAR-T treatment commercially, because it involved genetic engineering. They will also have to negotiate with insurance companies to determine which will cover the treatment and at what price.
“Technically it could be commercially available, but it’s not quite as simple as now we have some powder in a vial and we can ship it to you and you can give it to your patient,” Myers said.
In the mean time, Children’s Mercy will continue to enroll patients in the study if they qualify. It’s funded in part by money raised during the “Big Slick Celebrity Weekend.”
Novick, whose parents Gail and David are Kansas City natives, said the CAR-T treatments were a breeze compared to chemotherapy and bone marrow transplant. He said he understands the concerns about the price of the treatment, but hopes it will be accessible to other patients.
“At what price is a life?” Novick said. “I don’t think there’s too many people that would not want to find a way to pay the cost of that. It’s a very unfortunate situation to have to be in to say, ‘Well we have this option, it could potentially save your life, but we can’t afford it.’”