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Emerging Alternatives to Stem Cell Transplant

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Published on September 3, 2021

Are There Alternatives to Stem Cell Transplant?

With new immunotherapy options for MPN patients, what is the role of stem cell transplant? Follow along as Edward A. Faber, Jr., DO, MS, Medical Oncologist, Hematologist & Transplant Specialist at OHC Specialists in Cancer and Blood Disorders, discusses the importance of alternative therapies for patients who are ineligible for transplant, as well as which patients should still consider stem cell transplant as a curative option.

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Transcript | Emerging Alternatives to Stem Cell Transplant

Dr. Faber:
My name is Edward Faber, a blood and marrow and cellular therapy physician here in Cincinnati, Ohio. As someone that has been working in the field for the last decade and a half, I think many of us recognize how beneficial bone marrow or stem cell transplants can be. It does offer the hope of a curative process, but as with many things that are good in life, it comes at a cost. And unfortunately for many of our patients, they can have not only side effects or toxicities, but these can lead unfortunately to their inability to survive the process. And also there is one side effect that we all know as graft-versus-host disease that can be exceptionally limiting to many of our patients. And actually the quality of life can be worse than what they were dealing with from their myeloproliferative disorder. And so, to me, that's where the field has been moving as far as improving these outcomes, better quality of life, better survival, less side effects and less toxicities.

 And in my mind, when we talk about side effects, I say the term toxicity, because those are the things that we don't want. There are certain side effects like dry eyes or dry mouth, or... Those are side effects. We can still have a good quality of life around that, but when toxicities impair quality of life and certainly survival, we need to have improvements. And where is the field of hematology in general, moving and moving very quickly? It's immunotherapy. And there are things like cellular therapies that we call CAR T. There's antibodies that not only recognize one target, but multiple targets. And in my mind, this is the true immunotherapy because when we use donor stem cells and develop and grow a new immune system, we're creating that immune system to recognize the myeloproliferative disorder. And that's the hope for a cure and control, but the side effects and the toxicities that I mentioned earlier, we have to find a way to overcome that.

Why Are Stem Cell Transplant Alternatives Important?

And sometimes we can't fully control that. So, I believe that the hope is using antibodies and say CAR T, where we're using patient's own lymphocytes, may offer hope in limiting these side effects and toxicities while still having efficacy against the underlying blood disorder. Moving forward beyond that is really our way in our understanding of how to control the immune system better. And that has been the hope that I've been reading about since the 1990s, when I was a young student and in graduate school. We've been dreaming about manipulating the immune system for quite some time. And I believe now the science has caught up with our dreams and the old adage that science fact many times comes from science fiction, we're living that right now. And so, it's exciting to see how the field will evolve over the next three, five and 10 years.

I think transplant is still very much indicated. Folks that are a bit younger, under the age of 60, you have a related donor, you have bone marrow available as the stem cell source. Those are the ways that we know a fully matched donor, a patient in good health, we have very good outcomes from that type of patient. Now, if we're able to use some of the available therapies like the JAK2 inhibitors, for example ruxolitinib (Jakafi), everyone knows... Is aware of that drug. There is another next generation called fedratinib (Inrebic). Sometimes we can help folks with their symptoms if the disease behaves unexpectedly, improve their life, improve their performance status that we refer to and then move on to a transplant. That is still a very, very good strategy for many individuals.

Folks that are over 60, certainly approaching 70 or older, that's where we have to make, I believe, more individual decisions based upon other health issues. How matched or unrelated, I should say related or unrelated, the donor may be because there are times that yes, we do have to accept side effects and toxicities because medications offer only a finite time of helping.

I think patients over the age of 60, 65, that may not have the best donor, if we have alternative strategies, especially in the concept of a well-designed clinical trial, that's an excellent opportunity to pursue these other types of immunotherapies or cellular therapies.

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