Personalized Gene Therapy Shows Promise For Certain Blood Cancers
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MIAMI (CBSMiami) -- The FDA has approved a new gene therapy for some aggressive types of lymphoma. The treatment is giving hope to patients who have run out of options.
Judy Wilkins, 59, battled lymphoma for years. The cancer got so aggressive, multiple rounds of chemotherapy did not help and she was not a candidate for stem cell transplant.
"I never really thought at that point it would be making choices on my life. But then the more I realized how sick I really was because I was in total denial about it and that's when Caron said we are doing this," explained Wilkins.
Dr. Caron Jacobson from the Dana Farber Cancer Institute in Boston told Judy about an experimental treatment called CAR-T cell therapy. A patient's own immune cells, known as "T" cells, are engineered to find and kill cancer cells. CAR-T is custom made for each patient and delivered once.
"We give them some gentle chemotherapy for a couple of days in order to help 'T' cells grow better. Once we get them back into patient and then we infuse patient with cells and we wait for them to do their job," explained Dr. Jacobson.
In a nationwide clinical trial, of the 101 patients who underwent treatment with CAR-T, 84-percent had a response. 54-percent had a complete response and went into remission, including Judy.
"This is a tremendous deal," said Dr. Jacobson. "These are patients that have really exhausted the standard options."
Wilkins said, "I'm really lucky, really lucky." She's been in remission for over a year.
"I feel just like me again which has been almost 3 years."
While it is still too early to tell if this therapy is a cure, it gave Judy her life back and she's grateful for another chance.
The treatment costs hundreds of thousands of dollars. It can cause serious side effects and three people died after getting the treatment. The FDA is requiring a long-term safety study as well training for hospitals to quickly spot and manage reactions.
The first CAR-T drug, Kymriah, received approval in August to treat an advanced form of childhood leukemia. There are nearly 200 clinical trials exploring CAR-Ts currently registered with the federal government.
