INDIANAPOLIS -- Doctors at Riley Hospital for Children have been working for months to get systems in place to be able to treat kids with a newly approved cancer treatment.
The treatment, called Kymriah, aims to give some patients a second chance after first-line drugs have failed. This may happen in up to a fifth of patients. It was approved by the FDA this week for patients living with a very aggressive cancer – Acute Lymphoblastic Leukemia – who are also under the age of 25.
Each dose of Kymriah contains a patient's own immune cells, which are sent to a lab to be genetically modified using a virus. This therapy -- known as chimeric antigen receptor T-cell therapy, or CAR-T -- gives the cells the ability to recognize and kill the source of the cancer.
“You have to engineer them so they’re able to produce a marker on the surface on these immune cells that will be able to bind and interact with the Leukemia cells to get rid of those cells,” said Dr. David Delgado with Riley Hospital for Children.
The long term remission rate for patients treated with CAR-T is 80 percent.
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“These are patients who are very difficult to treat and are often very unresponsive to therapy so this is often unheard of to see this high of a response rate from any type of therapy we’ve used before,” said Dr. Delgado.
He says he has patients right now in Indianapolis who could benefit from the treatment.
“I’m not sure we know exactly what timeframe we’ll be able to offer it,” said Dr. Delgado. “We’re in the process of getting ready to be able to offer and treat those patients and we hope that it happens as soon as we’re ready to safely administer this treatment.”
Dr. Delgado says they hope to be able to start administering the treatment by the end of the year.
“I expect this treatment will be available in the future for many different forms of cancer in both children and adults,” said Dr. Delgado.
The first patient to be treated with CAR-T, Emily Whitehead, is now five years cancer free – but getting the treatment out of trial and approved by the FDA has taken years and scores of money.
“Over the past 20 years, LLS has invested $40 million in CAR-T immunotherapy, supporting the development of this revolutionary therapy by funding more than 15 researchers and companies around the world.”
One criticism of the new treatment has been the extreme cost – reports show it currently costs about $475,000.
Dr. Delgado says they recognize that issue and are already working with insurance companies to make CAR-T accessible to Hoosiers.
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