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.