CLEVELAND – On Wednesday, the U.S. Food and Drug Administration (FDA) approved a new treatment designed to help children and young adults battling recurrence of the most common type of childhood cancer.
According to Mikkael Sekeres, M.D., of Cleveland Clinic, CAR-T cell therapy is a form of ‘living therapy’ where cells are removed from a patient’s blood, reengineered in a lab, and put back into the bloodstream to fight cancer.
“We take that immune system, treat it and expose it to the leukemia cells itself, so that what grows out is an immune system that is specific for the leukemia – that knows how to attack the leukemia,” said Dr. Sekeres. “That immune system is then re-infused back into the patient so that hopefully, the leukemia will disappear.”
The treatment is the first therapy of its kind available in the United States.
It’s approved for use in certain children and young adults, up to age 25, who are fighting a form of acute lymphoblastic leukemia, or ‘ALL. ALL is a cancer of the bone marrow and blood.
If ALL recurs, it’s often resistant to conventional chemotherapy and radiation, leaving few options for young patients.
Clinical trials studying the new approach show an overall remission rate of 83 percent within three months of treatment, however, Dr. Sekeres said it does have severe side effects – and a hefty price tag.
One limitation of the trial was that the patients were followed up with after an average of five months, whereas a person is generally not deemed to be in remission until they have been leukemia-free for at least five years.
Dr. Sekeres said currently this type of therapy is only approved for children who have active, quickly-growing, recurrent leukemia, but he believes it’s just the beginning.
“This is an entirely different version of a therapy – it’s actually what we call a living therapy,” said Dr. Sekeres. “We’re giving live cells that we take from a patient, and giving them back to that patient. This is a vanguard of cancer therapy and what we’re going to be seeing more and more of in the future.”
The FDA is requiring hospitals that use the new CAR-T cell therapy to have special certification, therefore Dr. Sekeres said it will likely only be found at large medical centers once it becomes available.