These results are fantastic,” said David Steensma, MD, a hematologist-oncologist at Dana-Farber Cancer Institute and Harvard Medical School, Boston. “We have long wanted agents for AML [acute myelogenous leukemia] like we have for APL [acute promyelocytic leukemia], noncytotoxic chemotherapy approaches that release cells stuck in immature stages. Unfortunately, IDH2 is only present in less than 20% of AML.”

An implication that can be drawn from these data is that patients with relapsed/refractory AML should undergo molecular testing for the IDH2 mutation, and if the mutation is present, they could be treated with AG-221.

“Patients refractory to conventional therapies who might otherwise be enrolled in hospice could achieve an excellent result, just like a patient I saw treated at our institution with AG-221 who almost certainly would have died without this type of therapy. Now this patient has undergone stem cell transplantation,” Dr. Steensma said.

Dr. Steensma said that if further studies confirm these encouraging results, AG-221 could be transformative, allowing patients with no other alternatives a chance for curative transplant. He also said AG-221 may be useful in other malignancies that harbor IDH2 mutations, including glioblastoma multiforme. ■

Disclosure: Dr. Steensma reported no potential conflicts of interest.