PTG-300: ASH 2022 Srdan Verstovsek Verify Phase 3 Clinical Trial
By Srdan Verstovsek, MD, PhD
PTG-300 is the primary session report of the development of new antibody, and that antibody is against a mutated calreticulin. In all the patients with myeloproliferative, neoplasms being ET or PV (diagnosis polycythemia vera) or myelofibrosis, there is a hyperactivity of intercellular signal. JTA pathway, and this is because of our CE mutations in about 30% of the ET patients and 30%. The stakes are high, that's why that was a plenary session. We want to talk about the molecular response where we would be able to eliminate disease or eliminate at least malignant cells.
Inhibition of the growth and the cell death is expected to be seen in the mice. But now we know that the growth of these cells is inhibited and longer exposure to antibody mice would lead, hopefully to elimination of disease (polycythemia vera). The prospects are high that this is a beginning of new era when we talk about the molecular response and potential for elimination of the disease. Rusfertide is an injectable peptide mimicking hepcidin, a protein predominantly generated in hepatocytes. Low levels of are linked to iron overload due to excessive iron absorption, as seen in beta thalassemia, as well as iron deficiency anemia.
Their next step is really to transition from the preclinical to clinical work. Of course we will need to study the safety of the antibody first in most advanced patients, my fibrosis patients, to establish the safety and the side effects. The key takeaway from the presentation is that we are on a brink of a new chapter in development of therapies for myeloproliferative neoplasms et myelofibrosis. We did not at the beginning understand exactly how and why the hyperactive JAK pathway happens. And now after about 8 years from that discovery of calreticulin mutation, we are actually talking about therapies that was specifically a touch rate.
Dr. Srdan Verstovsek is a hematologist-oncologist at MD Anderson Cancer Center and the United Energy Resources, Inc. Professor of Medicine. He oversaw more than 60 early/advanced phase clinical trials of new MPN medications, including ruxolitinib, the only FDA-approved prescription for myelofibrosis (MF) until 2019. He has written 24 book chapters and over 600 peer-reviewed original articles/reviews in prestigious medical journals such as the New England Journal of Medicine, Blood, Leukemia, and Lancet.
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