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Gene Therapy Offers New Hope To Leukemia Patients At Cincinnati Children's

Cincinnati Children's

Cincinnati Children’s Hospital will soon be getting Kymriah, a newly FDA-approved drug, that uses the body’s own immune system to fight cancer – giving new hope to the families of kids with leukemia.

Cincinnati doctors say the acute lymphoblastic leukemia (ALL) patient who will use it first has either relapsed or was never in remission. The child’s body stopped responding to chemotherapy.

A clinical trial at Children’s and other hospitals nationwide saw an 83 percent remission rate with 68 patients who had seemingly reached the end of the road. Dr. Christine Phillips, an oncologist at Cincinnati Children's, says after Kymriah the disease was undetectable.

"Getting the immune system to wake up and do its job and treat the cancer is very exciting,” Phillips says.

The gene therapy takes the patient's own immune cells and reprograms them to fight cancer, as explained in this NPR story.

There are some temporary serious side effects, and the drug is expensive, at $475,000. Novartis says it will only charge patients who get better in a month, and Dr. Phillips says insurance will pay for it.

Gene therapy is showing promise in other formers of cancer. Melanoma and brain cancer are among cancers where immunotherapy is an option.

Javier El-Bietar, a bone marrow transplant doctor at Children's is optimistic about Kymriah for ALL patients.

"Just as little as 5-10 years ago, situations like an ALL relapse after transplant was invariably an end game kind of scenario,” El-Bietar says, “and this changes the ballgame entirely."