Israel is the first country to incorporate a breakthrough therapy developed for cancer patients suffering from an aggressive type of leukemia which, although extremely common, often leaves them with no other treatment options.
The new therapy, the first of its kind to be developed in twenty years, is by AbbVie, a research-based global biopharmaceutical company. Already after the early stages of the trial, Israel had included the therapy into the state-subsidized health care basket. Results from phase 3 of the therapy study were only presented earlier this month to the virtual 25th European Hematology Association (EHA) Annual Congress that took place on June 11-21.The results from the study constitute a historic breakthrough for patients suffering from acute myeloid leukemia (AML), a relatively common type of leukemia, with 160,000 patients across the globe who live with the disease. The treatment, a combo-therapy of venetoclax and azacitidine, was developed for AML patients who have very little other treatment options since they are often ineligible for bone marrow transplant or intensive chemotherapy.“Patients who are diagnosed with AML face a very difficult disease with few currently approved treatment options, and survival in this patient population is low,” according to Dr. Courtney D. DiNardo from the Department of Leukemia at the MD Anderson Medical Center, chief researcher in the new therapy’s trial study. “Patients and their healthcare providers need access to treatments that have demonstrated both safety and efficacy in well-designed clinical trials,” she said.The combo-therapy, which is simply taken orally, offers these patients an extended period of survival. Patients who took the venetoclax combination achieved improved median overall survival of 14.7 months versus the 9.6 months in the placebo group. Additionally, more than twice as many patients (66.4%) treated with the combo-therapy had a composite complete remission compared to the 28.3 % treated with azacitidine plus placebo.Phase-3 results from the study showed a significant increase in survival rates (34%) for patients who were otherwise ineligible for chemotherapy and were given the combo-therapy of venetoclax and azacitidine. These results were compared to the control group who only received azacitidine and a placebo. Patients who received the combo-therapy showed a median survival of five months.
"AML is an aggressive and difficult-to-treat blood cancer and is typically treated with conventional induction chemotherapy,” DiNardo said. “The five-year survival rate is only 29% and the treatment landscape has not evolved enough in the past three decades – especially for patients who cannot withstand intensive chemotherapy.”The Israeli Leukemia Group had an active role in achieving early access to venetoclax for AML patients in Israel, the fifth largest country in terms of the number of patients enrolled in the study. The country had already incorporated the drug into the state’s healthcare basket after phase-1 results. The move to incorporate the therapy came months after FDA approval, and two years ahead of Europe. It was an unusual step for the Health Ministry, which usually waits for phase-3 trail data before approving a drug. Since AML is so prevalent and aggressive, however, it was the right decision in DiNardo’s eyes. She encourages regulatory agencies to further consider early access.“Given the seriousness and aggressiveness of this AML, I’m hopeful regulatory agencies will work closely with the trial sponsors to determine a pathway to provide accelerated treatment access to patients in a timely fashion,” she said.