An experimental pill could turn a lethal cancer into a chronic, but manageable disease like high blood pressure. If approved for treatment for a common form of leukemia, the compound replaces toxic chemotherapy treatments.
Chronic lymphocytic leukemia, or CLL, is a cancer of the immune system’s B cells, which produce antibodies, the frontline soldiers against bacterial and viral invaders. But when B cells become cancerous, they accumulate in the patient’s organs, including the lymph nodes, pea-shaped organs under the arms and in the groin that help the body recognize and fight infections. With CLL, the nodes swell many times their normal size.
Richard Furman, a cancer researcher at Weill Cornell Medical College in New York, says the drug idelalisib
, taken twice a day, causes the cancer to melt away.
“When I say melt away, you can literally see the lymph nodes shrink over the course of a couple of days. It works that quickly, which is really wonderful," said Furman.
The standard treatment for CLL is Rituxan, an infusion drug that destroys diseased B cells, but only for a time before the patient relapses. With repeated rounds of chemotherapy, Furman says the leukemia eventually becomes resistant to Rituxan and patients no longer respond. The cancer is fatal.
Investigators led by Furman compared a combination of idelalisib and Rituxan, to Rituxan and placebo in a group of 220 CLL patients from around the world (19 medical centers in the U.S. and five other countries).
Six months into the study, 13 percent of those receiving only Rituxan responded to the therapy compared to 81 percent of the Idelalisib combination group.
And 92 percent of participants in that group were still alive a year after the study began, compared to 80 percent in the Rituxan-only group.
The contrast was so significant that the study was halted early so all the patients could receive Idelalisib.
“And with an agent like idelalisib, which is extraordinarily well-tolerated and extraordinarily effective, my hope is that we can make CLL a chronic disease, sort of akin to high blood pressure where patients are able to take a pill a day and keep it in check," said Furman.
The company that makes idelalisib has asked U.S. regulators to approve the drug within six months so it can be made available to people with chronic lymphocytic leukemia. An article on idelalisib is published in The New England Journal of Medicine.