Ground-Breaking Immunotherapy for Childhood Leukemia Gets FDA OK

New treatment may open the door to therapy for other cancers

A new treatment designed to help children and young adults battling recurrence of the most common type of childhood cancer received approval from the U.S. Food and Drug Administration (FDA) this week.

Advertising Policy

Cleveland Clinic is a non-profit academic medical center. Advertising on our site helps support our mission. We do not endorse non-Cleveland Clinic products or services. Policy

The therapy is called CAR-T cell therapy. It involves taking cells from a patient’s blood, re-engineering them in a lab to attack a specific protein on the cancerous cells, and returning them to the patient’s bloodstream, where they seek out and destroy cancer cells, says oncologist Mikkael Sekeres, MD, MS, Director of Cleveland Clinic Cancer Center’s Leukemia Program.

“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,” Dr. Sekeres says. “That immune system is then re-infused back into the patient so that, hopefully, the leukemia will disappear.”

Anti-rejection drugs, which usually are needed after transplants, are unnecessary because the cancer-fighting cells are from the patient’s own body, Dr. Sekeres says.

First leukemia therapy of its kind

The treatment is the first therapy of its kind available in the United States. It is a type of immunotherapy, a one-time infusion that uses the body’s own defenses to attack the cancer.

It’s approved for use in certain children and young adults up to age 25, who are fighting a form of acute lymphoblastic leukemia (ALL). ALL is a cancer of the bone marrow and blood.

Advertising Policy

If ALL recurs,  conventional chemotherapy and radiation often aren’t effective against it, leaving few options for young patients. Clinical trials of the new therapy show an overall remission rate of 83 percent within three months of treatment.

Side effects

However, the treatment does have severe side effects, Dr. Sekeres says. The side effects affect the vascular system, causing high fever and a sharp drop in blood pressure. The drugmaker reports that more than 60 percent of patients had these side effects in clinical trials, but none were fatal.

The arthritis drug tocilizumab, which is sold under the name Actemra, can combat the side effects. On Wednesday, the FDA also approved Actemra as a treatment for the side effects. Under the conditions of the FDA approval, doctors can’t use the new therapy unless they also have Actemra on hand.

The new treatment, to be sold under the brand name Kymriah, won’t be available everywhere. Hospitals and clinics will have to be specially certified to administer the treatment, and health care providers will have to undergo additional training before they can prescribe it.

Clinical trial limitations

One limitation of the trial was that the patients were followed up with after an average of five months, Dr. Sekeres says. A person is generally not deemed to be cured until they have been leukemia-free for at least five years, he says.

Advertising Policy

Currently, this type of therapy is only approved for children who have active, quickly growing, recurrent leukemia, Dr. Sekeres says. But beyond that, this treatment may be the first step toward a new  model for treating other types of cancers as well, he says.

“This is an entirely new version of a therapy — it’s actually what we call a living therapy,” Dr. Sekeres says. “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.” 

Because of the FDA requirements, the treatment likely will  be found only at large medical centers once it becomes available, Dr. Sekeres says.

Advertising Policy