Gene therapy one step closer to FDA approval

An FDA panel unanimously recommended approval of a cancer treatment for patients who

Looking at 17 year-old camp counselor Justin Pritikin, you’d never imagine he just battled a leukemia relapse.

“When I first got diagnosed, you know, they say you can’t really understand or whatever. I got the gist of it immediately. I was like, ‘OK’” he said.

Not only did he get the gist, Justin was determined to beat it and a new gene therapy has become his best weapon. Scientists are reporting unprecedented success with a new T-cell treatment, using the patient’s own immune system to fight the cancer.

“This is a therapy that uses the patient’s own cells that have been genetically engineered, and when infused back into the patient will locate the tumor cells and kill those tumor cells. That’s never, we have nothing like that in the arsenal today,” said Dr. Lee Greenberger, chief scientific officer of the Leukemia & Lymphoma Society.

Just this week an FDA panel unanimously recommended approval of what’s being dubbed the “second chance” cancer treatment because it works best on patients who’s first line of drugs — traditional chemo and radiation — failed.

“He was originally diagnosed with acute lymphoblastic leukemia back in March 2012 and he was doing great,” said mother Jill Pritikin. “He had standard therapy, which is chemotherapy. For boys, three and a half years is your road map of treatment. And he was off chemo for about a year and four months and then he relapsed just in November 2016 with the exact same cancer.”

“It was horrible watching him go through the regular stuff, the regular chemo. To watch him go through this new T-cell treatment, it’s sort of like you’re part of a magic trick. Like you really can’t believe what they do,” said father Andy Pritikin.

After the white blood, or T-cells are removed from the patient, the new CAR T-cells are put back where they divide and conquer.

“We weighed our options. We looked at bone marrow transplant, it wasn’t a good option at the time. We looked at doing another round of intense chemo, but with my type of cancer it was probably going to come back, so that wasn’t a great option. We looked at a different study, but it wasn’t as promising as this one,” Justin said.

“I can tell you seeing this firsthand, seeing the difference that it makes, seeing all these kids in treatment at hospitals, it’s very moving,” Andy said.

After five years missing school and friends, it took just one T-cell treatment to get him back on his feet, in remission, at the Burlington County summer camp run by his parents.

“I mean, I’m at camp. I got diagnosed in November. I’m working long days and hot hours with many kids who are loud or whatever and I feel fine with it. So it must have worked, I think,” Justin said.

Though scientists warn there can be severe side effects — some nearly fatal — serious infections can occur or a syndrome that resembles sepsis. Fortunately for Justin he had none. Still the benefits, seem to outweigh the cons.

“It can produce dramatic effects to kill tumor cells that are long lasting — years. In fact as best as we know, maybe indefinitely. It looks like, in some patients, like a cure with a single dose of this new therapy cure. That’s truly remarkable,” said Greenberger.

“He’s back to being a 17-year-old full of energy, making me crazy because he’s doing regular 17-year-old things. You know, staying out late with friends. It’s good. I’m optimistic, but I’m realistic at the same time. So you know, you’re never going to shake that feeling that this might not be over, but you know, for now we’re living in the moment. I mean that’s all anybody has, so that’s kind of how we have to approach life,” said Jill.

The FDA could approve the therapy as early as this year.

We’re in this together
For a better-informed future. Support our nonprofit newsroom.
Donate to NJ Spotlight