Is there a cure for BPDCN?

Is there a cure for BPDCN?

Because BPDCN is so rare, and in addition to that, it's been difficult to nail down the diagnosis. So it's kind of rare on top of rare historically. It's been thought to be an elusive entity. But one of the good things about this entity, and I think why we're having this interview, is because we're starting to understand it's its own unique entity within all of the myeloid leukemia. And so the latest W.H.O. and ICC both recognize BPDCN as its own blood cancer.

And the reason why I bring that up is because as we think about cures for this disease, we still just barely understanding the biology of it. And so the 2 or 3 breakthroughs that our group has been fundamentally interested in has been, number one, that all of these cells express a marker called CD123. So that's IL-3 receptor alpha. And that'll be important later on when we talk about treatments. Two, a lot of these cells signal through BCL2, which is an internal pathway that is being targeted in both AML and lymphoid diseases.

And then three, the disease appears to be hyper susceptible still to cytotoxic chemotherapy, steroids from either an ALL based or AML or lymphoma based regimen. And so for us and BPDCN, I would answer the question by saying we are able to cure a small fraction of patients, usually involving either multi-agent chemotherapy, CNS prevention, and or CD123 therapy, and usually coupling it with a stem cell transplant. So we are able to cure. However, historically it's been very poor prognosis. And so we're urgently looking for other new therapies.

Many of the hematologic malignancies that we treat, the only cure is a hematopoietic stem cell transplantation. So a bone marrow transplant—that is a curative modality even for BPDCN. We typically treat patients with BPDCN with therapies to try to remit the disease and then use a transplant. If a transplant is applicable to a patient—because not every patient is eligible for a transplant—but use a transplant to consolidate and cure when possible with BPDCN.