Fighting cancer from within
CAR-T treatment offers young patients hope
The approval of a revolutionary childhood cancer treatment that turns human cells into diseasefighting armies is giving new hope to kids with few other options, and Boston’s own Dana-Farber Cancer Institute may be ready to offer the groundbreaking therapy in a matter of weeks.
“It’s manipulating the body’s own immune system to attack the cancer,” said Dr. Lewis Silverman, clinical director of Dana-Farber’s Pediatric Hematologic Malignancies Center. “This is a completely different paradigm.”
The new treatment for acute lymphoblastic leukemia — the most common childhood cancer — reprograms a patient’s T-cells to attack the disease from within.
Developed by Novartis Pharmaceuticals and the University of Pennsylvania, the breakthrough technique will give new hope to children who aren’t receptive to chemotherapy and radiation, and have, until now, been left with few other options.
Although only about 35 sites nationwide will offer it, Silverman pointed out that the technique is just the beginning and that “more and more therapies are becoming available that are able to redirect the immune system to attack cancer.”
Dana-Farber is expected to begin offering the so-called CAR-T cell therapy in a matter of weeks or months, the hospital said.
About 3,000 people 20 and younger are diagnosed with this type of leukemia each year and Silverman said as many as 20 percent of those patients can’t be cured with standard treatment options.
In a statement, FDA Commissioner Scott Gottlieb hailed CAR-T cell therapy as part of “a new frontier in medical innovation with the ability to reprogram a patient’s own cells to attack a deadly cancer.”
Despite its promise, the technique doesn’t come without complications.
The process would involve removing a patient’s cells and sending them to a lab for engineering, which could take up to four weeks, Silverman said. And once those cells are replaced, fighting the disease from within will take a toll on the child’s body.
Much like the flu causes aches and chills, the body would give a similar response, but much more severe.
Acute lymphoblastic leukemia lends itself to this type of treatment, because each of the cancer cells has a clear marker on its surface for T-cells to target.
But it is likely that lymphomas — cancers of the immune system — will be candidates as well, said Dr. Michael Kelly, chief of the Division of Pediatric Hematology/Oncology at the Floating Hospital for Children at Tufts Medical Center.
He added that once the treatment shows success at a limited number of hospitals, it will be expanded to other institutions.
“This is a really big step forward,” Kelly said. “The FDA will be watching closely.”