VIDEO: 'Exciting new developments' in GVHD treatment, understanding

Editor’s note: This is an automatically generated transcript. Please notify editor@healio.com if there are concerns regarding accuracy of the transcription.

There are a lot of exciting new developments in the treatment and just the overall understanding of graft-versus-host disease. And I can try and break it down based on whether we're talking about acute graft-versus-host disease or chronic graft-versus-host disease, and then also within the treatment space and the prevention space.

Starting with chronic graft-versus-host disease — because that's where most of the innovation has occurred —we have two FDA-approved drugs, ibrutinib [Imbruvica; Janssen, Pharmacyclics] and ruxolitinib [Jakafi, Incyte], for the treatment of chronic graft-versus-host disease. And relevant to me specifically, since I'm a pediatric bone marrow transplanter, I like to think of where these drugs are also indicated in children. And then the third FDA approval came with belumosudil (Rezurock, Kadmon/Sanofi], where children 12 years and older are eligible to get this drug. So, a lot of sort of therapeutic options available for treatment of chronic graft-versus-host disease, whereas previous to these three drugs, the options and choices for our patients were quite few.

What is probably exciting and in the threshold, perhaps, of approval is axatilimab [SNDX-6352; Incyte/Syndax Pharmaceuticals]. We saw some very interesting data presented at both the American Society of Hematology and also at the American Society of Transplantation and Cellular Therapy on the tolerability and responses in chronic graft-versus-host disease with axatilimab. What is interesting there is it's studying a novel pathway of CSFR1 receptor on macrophages targeting fibrosis, whereas all previous agents have been looking just at T-cell and B-cell targeting. So a novel pathway, a novel drug, additional opportunities for our patients. So, I think that's where things are quite exciting.

And in the sort of prevention phase, and this would span both acute and chronic graft-versus-host disease, would be the study of using a single high dose of oral vitamin A to prevent acute and chronic graft-versus-host disease. Those data were presented last year at a TCT meeting, and are now published. And I think what is interesting about this particular strategy is that it's a single dose given once before the onset of chemotherapy — so before any nausea and vomiting. It is a large dose. The maximum dose is a quarter of a million units of vitamin A. What it does is it allows for our dendritic cells of our intestinal tract to become more tolerogenic, especially in a presumed homeostatic environment. And with the single sort of dose of a drug, we're able to see in a randomized double-blinded placebo-controlled clinical trial effects of reduced moderate to severe chronic graft-versus-host disease, and reduced acute gastrointestinal graft-versus-host disease. So, the very clinically relevant types of both acute and chronic GVHD. And I think what is quite interesting about that, too, is it's an affordable strategy. It's very cheap. Vitamins are technically not even drugs. You know, they're available as supplements over the counter. So, I think there's a lot of exciting things happening within the space.