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Agio’s new blood cancer drug AG-221 scores

Cambridge, Massachusetts-based Agio Pharmaceuticals’ experimental new blood cancer drug AG-221 recently demonstrated significantly promising anti-cancer activity in an initial Phase I study of people suffering from relapsed AML (acute myeloid leukemia).  Dr. Eytan Stein of Memorial Sloan Kettering Cancer Center in New York, the lead investigator of the cancer drug study told Reuters that AML is the most common kind of acute leukemia found in adults.

Specifically, it’s both a blood cancer and bone marrow cancer that advances rapidly if not treated.  The blood cancer drug AG-221 focuses on a gene mutation present in up to 15 percent of people with AML.  Those in Stein’s study suffered from AML that had advanced after or did not react to as many as four other prescribed treatments.

blood cancer drug

Agio has created a promising experimental new blood cancer drug AG-221

According to the report, all the patients had a “genetic mutation in the leukemic cells” which the blood cancer drug was created to affect.  It’s codenamed IGH2 or isocitrate dehydrogenase-2. Six out of seven patients evaluated after a 28-day cycle of treatment with the blood cancer drug, AG-221 had what the investigators called “objective responses” to the medication.  Furthermore, three of the six experienced complete remission.

Two were reported to have experienced “complete remission with incomplete platelet recovery” which means that the leukemia had left the bone marrow but their blood platelet count had not reverted to normal.  The new blood cancer drug treatment resulted in significantly lower levels of 2HG– a specific cancer metabolite–in the blood.  This metabolite is thought to be responsible for reprogramming white blood cells and thereby nullifying their power to fight infection.

blood cancer drug


The study participants were given either 30 milligrams or 50milligrans of the blood cancer drug AG-221 twice daily.  Data was not yet available on another portion of the study involving 2 additional higher dosages.

Stein, who recently presented the team’s findings at the AACR (American Association for Cancer Research) meeting in San Diego, California stated: “I’m very excited about what has happened with those patients so far who have responded.  He also noted that the data was only preliminary.

He opined: “If the results are confirmed (in soon-to-come larger trials) that would be a remarkably exciting result.”  He also revealed that in this first small group of participants they observed no drug-related side effects “which is very different from chemotherapy.”  He noted that they hadn’t expected “the impact seen with the lowest doses” in the first few participants.

Stein concluded: “We were actually kind of surprised that at the first dose level we’re seeing dramatic clinical activity. That usually doesn’t happen.”

(Image courtesy of ThePetrizzoGroup and SecGov)

About Will Phoenix

W. Scott Phoenix, B.A., B.S. was born in Hawaii, raised in Pennsylvania and resides in California. He has been a published writer since 1978. His work has appeared (under various names) in numerous places in print and online including Examiner.com. He is a single parent of three children and has also worked as an actor, singer and teacher. He has been employed by such publications as the Daily Collegian and the Los Angeles Times.