Developing Therapeutics for Benign Hematologic Diseases
In This Video
- Hanny Al-Samkari, MD, is a classical hematologist and clinical investigator at the Center for Hematology at the Mass General Cancer Center
- He conducts hemostasis research on the use of systemic therapies to treat hereditary hemorrhagic telangiectasia (HHT)
- With a better understanding of the pathophysiology of HHT, doctors are now treating the specific underlying problem, which is excessive vascular endothelial growth factor, with drugs that were originally developed to treat cancer
- His research is also exploring the development of therapeutics for a number of benign hematologic diseases
Hanny Al-Samkari, MD, is a classical hematologist and clinical investigator at the Center for Hematology at the Mass General Cancer Center. He conducts hemostasis research on the use of systemic therapies to treat hereditary hemorrhagic telangiectasia. His research is also exploring the development of therapeutics for a number of benign hematologic diseases.
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Transcript
I think one of the most exciting areas within the hemostasis fields in the modern day is the use of systemic therapies to treat hereditary hemorrhagic telangiectasia, or HHT. This is the second most common hereditary bleeding disorder in the world and yet has no FDA-approved treatments. For decades and decades we've been treating HHT with just local hemostatic procedures that are generally temporizing, but over the last couple of decades, we've understood the pathophysiology of HHT much better and are now treating the specific underlying problem, which is excessive vascular endothelial growth factor in these patients. And we're doing this with drugs that were originally developed to treat cancer, drugs like bevacizumab, that were effective in managing cancer but certainly not a paradigm shift, and now using those drugs to manage patients with HHT. And the data we've collected thus far is very, very encouraging with the use of these drugs to cause the abnormal blood vessels that form along mucosal surfaces in the nasal cavity and the GI tract that cause chronic bleeding and anemia in this disease, to get these vessels to just involute and go away, really to make them disappear, which stops the bleeding, resolves the anemia and patients feel much, much better.
So in addition to a lot of the exciting work we're doing in HHT, we're really looking forward to the development of therapeutics for a number of benign hematologic diseases, drugs that are able to treat a number of hereditary hemolytic anemias using novel mechanisms that address the underlying cause of the anemia, as well as drugs are to raise the platelet count in cancer patients receiving chemotherapy that would otherwise be impossible to administer safely.
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