New pill 'melts away' leukemia without chemotherapy

The newly-tested drug was so effective that researchers halted the double-blinded trial and asked all the participants to take the new drug.

By Ananth Baliga

Making leukemia a treatable disease could be as easy as popping a pill twice a day, without having to go through debilitating chemotherapy.

Researchers at Weill Cornell Medical College have tested a drug, called idelalisib, that could do away with chemotherapy treatment for patients diagnosed with chronic lymphocytic leukemia, or CCL, the most common form of the disease.


The findings have been published in the New England Journal of Medicine, and show that idelalisib in combination with rituximab could prevent the leukemia from worsening, even in chemotherapy-resistant patients.

In the double-blinded study, 220 chemotherapy-resistant leukemia patients were given either rituximab and idelalisib or rituximab and a placebo. Rituximab is another targeted drug that has been used to treat leukemia. They found that those who received the combination of drugs went longer without their condition worsening as compared to those in the placebo group.

Within six months 93 precent of participants in the combination drug therapy had not worsened, compared to 46 percent in the placebo group. After a year, 92 percent of patients given the new drug were still alive as compared to 80 percent who took only rituximab.


These results were so encouraging that researchers stopped the trial and put all the participants on the combination drug therapy. Patients responded to the medication in as little as a week.

"We saw incredible responses in patients who used idelalisib. Their cancer quickly melted away," says Dr. Furman, an associate professor in hematology and oncology at Weill Cornell Medical College.

"Even if this cancer remains incurable, it now can be treated as if it was a chronic disease with a pill, in the same way that high blood pressure is treated," Furman said.

Furman previously tested ibrutinib, which was used to target mantle cell lymphoma, and was consequently approved by the U.S. Food and Drug Administration. Furman has been using the drug as the first-line treatment, even for patients who were recently diagnosed. Furman said that idelalisib and ibrutinib will hopefully become first-line treatments for B cell lymphomas.

"I am now able to avoid all use of chemotherapy in these patients, which has long been my goal," he said.

[Weill Cornell Medical College] [New England Journal of Medicine]

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