MPD Blood Cancer Treatment Begins Human Trials
Researchers at the Dana-Farber Cancer Institute have begun Phase I clinical trials of a new drug designed to treat myeloproliferative diseases (MPD) that sometimes develop into acute myelogenous leukemia (AML).
Scientists at Brigham and Women’s Hospital have already used the new drug, an orally-administered compound known as TG101348, to successfully treat chronic blood cancers in mice. Brigham and Women’s animal study showed the investigational drug dramatically increased survival rates without causing toxicity.
The TG101348 drug (designed by biopharmaceutical company TargeGen, Inc) works by targeting JAK2, a protein that promotes blood cell growth.
Mutations in JAK2 can produce runaway red and white blood cell growth leading to bone marrow damage, blood clots, painful spleen enlargement, excessive bleeding and infections. While some patients live for years with MPD, others develop AML — a potentially fatal form of blood cancer.
MPD affects approximately 80,000 patients in the US and limited treatment options currently exist. But TG101348’s promising results in mice have the scientists enthusiastic about testing it’s effectiveness in human trials. As one Dana-Farber researcher said,
“A betting person would be excited about this.”
Richard Stone, MD
Oncologist, Dana-Farber Cancer Institute
If you’d like to learn more about the TG101348 study, a report has been published in the April 7, 2008, edition of the journal Cancer Cell.
Source: Brigham and Women’s Hospital News
Related Links: American Cancer Society; National Marrow Donor Program; Scientific American
Related Podcast: MMP099 – Acute Myelogenous Leukemia from The Medical Minute
Technorati Tags: polycythemia vera; National Institutes of Health; Howard Hughes Medical Institute; MPD Foundation; Leukemia and Lymphoma Society; Mayo Clinic; hematologic malignancies
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