(RxWiki News) Most patients with a slow-growing form of non-Hodgkin lymphoma respond well to therapy at first. But the disease can return and become resistant to therapy. A new medication may change this pattern.
In a phase ll trial, almost all of the patients with indolent (slow-growing) non-Hodgkin lymphoma (NHL) responded to the investigational medication called idelalisib.
These patients had undergone a number of previous treatments, and standard therapies no longer worked for many of the individuals.
Idelalisib is currently being reviewed by the US Food and Drug Administration (FDA) and may be available to patients later this year.
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Ajay Gopal, MD, a member of Fred Hutchinson Cancer Research Center's Clinical Research Division, served as the study’s lead author.
Non-Hodgkin lymphoma is a term that refers to various types of cancer that develop in the body’s lymphatic system. NHL can start almost anywhere and spread to any organ.
Nearly 70,000 Americans will be diagnosed with NHL this year, and about a third of those will have an indolent non-Hodgkin lymphoma (iNHL).
Indolent NHL is traditionally treated with a targeted therapy called Rituxan (rituximab) and chemotherapy. This treatment is usually effective in the beginning, but the disease is known to return, sometimes bringing with it life-threatening infections and other complications.
To study the overall response rate for idelalisib, 125 iNHL patients between the ages of 37 and 84 were recruited from 41 sites in the US and Europe. Most of the patients (89 percent) had stage lll or IV iNHL.
All of the participants had undergone at least six previous treatments, had become resistant to Rituxan and chemotherapy or had seen their lymphoma return within six months following traditional therapy.
During the trial, participants were given 150 mg of idelalisib, an oral medication, twice a day.
The response rate was 57 percent (71 of 125) of patients, and 6 percent met the criteria for a complete response.
The median time to response was 1.9 months and the median response duration was 12.5 months. Median progression-free survival (period during which the disease did not get worse) was 11 months.
The most common side effects were diarrhea (43 percent), fatigue and nausea (30 percent each), cough (29 percent) and fever (28 percent).
The most serious adverse events included neutropenia (lowering of infection-fighting white blood cells ), which was seen in 27 percent of the patients, elevations of an enzyme in the blood (aminotransferase) that can signal organ damage (13 percent), diarrhea (13 percent) and pneumonia (7 percent).
According to the authors of this study, “Idelalisib monotherapy resulted in tumor reductions in 90 percent of the patients, with 57 percent meeting the criteria for an objective tumor response.”
This medication did not appear to be a cure for iNHL, but it holds promise for controlling the disease for long periods of time, Dr. Gopal said in a statement. He added that medications like idelalisib represent a highly targeted and less harmful approach to treating cancer.
The FDA accepted a New Drug Application for idelalisib earlier this month, with a target review date September 11, 2014. The FDA gave the medication a Breakthrough Therapy designation for the treatment of chronic lymphocytic leukemia (CLL) based on the results of another clinical trial.
Results from this iNHL trial were published January 22 in the New England Journal of Medicine.
The research was sponsored by Gilead Sciences, the maker of idelalisib and the National Institutes of Health and by philanthropic gifts from Frank and Betty Vandermeer.
A number of the authors disclosed financial relationships with Gilead Sciences and other pharmaceutical companies.