A new leukemia drug that shows a lot of promise is being developed by the biotechnology company, Agios, a Cambridge, Massachusetts based company. Before the news came out about the advances Agios was making in perfecting the leukemia drug AG-221, there were some doubts if the July public stock offering that raised over $100 million for the company was worth it, as Agios hadn’t been able to produce any results that its products were helping anyone.
However, the pill that Agios has developed, known as AG-221, has demonstrated its effectiveness enough to make the medical world sit up and take note. It’s been tested out in 10 patients who have acute myeloid leukemia with tumors. Not just any tumors, though, but ones which have a mutated protein known as IDM2 in a certain specific gene. The protein IDH2 is known to promote the growth of cancer cells.
AML develops the blood and bone marrow of mostly adult humans. It’s the most common type of leukemia that affects adults.It’s often fatal and, if it’s not treated quickly enough, the disease progresses rapidly.
Out of the 10 AML patients that the new Agios drug AG-221 has been tested out on, three were forced into not taking the drug any longer because they had become too sick due to their leukemia. They have since died.
However, out of the seven who were left in the study, six showed marked improvement. Three of these six people experienced a complete remission, all signs of cancer cells that had been in their blood having disappeared. Two of the other three were close to having complete remissions, but they still had blood platelet levels which were depleted. They platelet levels were increasing, though, and, given time, it’s expected that these two people would also have complete remissions.
Even with patients who have complete remissions, they generally would have to have a bone marrow transplant. Dr. Eytan Stein from the Memorial Sloan Kettering Cancer Center, New York, at a press conference called this sort of data “unheard of.” He stated that, with the patients who had a complete remission after taking AG-221, “all bets were off,” and a bone marrow transplant might not be “necessary.”
The Agios drug AG-221 has not yet been involved in a placebo study, and studies involving more people also have to be done before the drug can make its way to the market. It might take years before AG-221 will get FDA approval and will be sold to the general public, but it is a drug that definitely shows a lot of promise.
For Agios CEO David Schenkein, the initial success of AG-221 offers proof that the scientific ideas behind his company, creating medicines that are targeted to specific metabolic cell processes, will result in bringing much-needed drugs to the marketplace faster and more efficiently than has been the case in the past.
Patients are continuing to receive the experimental drug AG-221 developed by Agios.One patient is in the fifth cycle of treatment. Additional patients are being enrolled in the study, and are being treated with higher doses of AG-212. Agios is testing out the dosages that would be necessary for twice-per-day and once-per-day treatments.
Agios is working in collaboration with the Celgene company in developing AG-221. AG-120 is another cancer drug they’re working on. It targets a different mutated cancer-growing protein, IDM1.
The study involving AG-221 has been small, so far, but with the patients that are being added to the initial study group, if beneficial results keep on being demonstrated, the drug might be fast-tracked by the FDA. Still, placebo studies involving more patients are necessary before AG-221 will become approved by the FDA and become widely available.
Written by: Douglas Cobb