New Gene Therapy for Cancer Offers Hope to Those With No Options Left – NBCNews.com

Posted: March 31, 2017 at 10:46 pm

Dimas Padilla, 43, of Kissimmee, is in remission from non-Hodgkin's lymphoma after receiving an experimental cancer therapy called CAR-T. Here, he poses with his wife, Dimas Padilla. NBC News

"These are patients who really are without hope," Locke said.

"Patients who at best could expect to have a one in 10 chance of having a complete disappearance of their lymphoma," he added. "So the results are really exciting and remarkable."

More than 80 percent of the 101 patients who got the treatment were still alive six months later. "Only about half the patients who (went) on this study could expect to even be alive six months after the therapy," Locke said.

Padilla is one of them. When the cancer came back most recently time, his lymph nodes were bulging. "They were so bad that they moved my vocal cords to the side and I was without my voice for almost three months," he said.

"They kept growing and my face was swelling, and I thought I was going to choke while I was sleeping."

Padilla was among the last patients enrolled in the trial.

"Once they infused the cells in my body, within two to three days all my lymph nodes started melting like ice cubes," he said.

The treatment is no cake walk. Just as with a bone marrow transplant, the patient's immune system must be damaged so that the newly engineered T-cells can do their work. That involves some harsh chemotherapy.

It's so harsh that it killed three of the patients in the trial. Padilla says he still has some memory loss from his bout with the chemo.

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"I had some fevers and I was shaking and a little bit of memory loss but it was temporary," he said. "I will say that it was pretty intense for like a week, but in my second week, second week and a half, I was starting to feel more normal. I was able to start walking and the shaking was not as bad as it was in the beginning," he said.

And when he got the news that his lymphoma was gone at least for now Padilla was delighted.

"I kissed my wife. I probably kissed the doctor," he said.

The company developing the treatment, Kite Pharma, sought Food and Drug Administration approval for the therapy on Friday.

It carries the tongue-twisting name of axicabtagene ciloleucel, and it's the first commercial CAR-T product to get into the FDA approval process.

It's far too early to say any of the patients were cured, Locke cautions. And such a difficult treatment course is really only for patients in the most desperate condition.

"The patients in this trial were really without options," he said.

But Locke is sold on the approach. "This is a revolution. It's a revolution in cancer care. This is the tip of the iceberg," he said.

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New Gene Therapy for Cancer Offers Hope to Those With No Options Left - NBCNews.com

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