2014 Summer Science Update

John Crispino, PhD, Scientific Advisor

If it’s summer, it’s time for two important meetings where researchers present their latest findings on MPNs: In the US, it’s the American Society of Clinical Oncology (ASCO) meeting, and in Europe it’s the European Hematology Association Meeting (EHA). Here’s a brief summary of key findings:

PRM-151 shows promising results in Phase II myelofibrosis study

Dr. Srdan Verstovsek presented early results for PRM-151 in treating myelofibrosis. PRM-151 is an experimental agent that mimics a normal human protein, pentraxin 2, which acts to prevent fibrosis in response to tissue damage. The study included a total of 27 patients; seven of them showed notable responses, including five who displayed decreases in the degree of bone marrow fibrosis. PRM-151 was safe and well tolerated, with no evidence of myelosuppression. More than half of the patients remain on study beyond 24 weeks. These early results are encouraging. Stay tuned for additional updates!

Ruxolitinib effective for advanced PV in patients resistant to hydroxyurea

Dr. Alessandro Vannucchi presented results from the Phase III RESPONSE trial of Ruxolitinib in patients with advanced PV who were resistant or intolerant to hydroxyurea. Ruxolitinib was superior to best available therapy, with the majority of patients showing a reduction in spleen size and a significant decrease in symptoms. Ruxolitinib was well tolerated, and the majority of patients showed a durable response. These results suggest that Ruxolitinib may be a viable therapy for individuals with advanced PV. Equally important, continuing studies could lead to FDA approval of ruxolitinib for PV – which, in turn, means the cost could be covered by insurance.

Exciting new study on the stem cell niche and its relationship to the MPNs

This is painfully technical, but bear with me. You all know about JAK2 inhibitors; this is something totally different that could lead to a whole new way to treat MPNs. Dr. Simón Méndez-Ferrer demonstrated that sympathetic nerve fibers are consistently reduced in the bone marrow of MPN patients. Enhancement of these cells restored normal sympathetic regulation and blocked MPN progression in a mouse model. Next step: Find out what happens in humans. Dr. Méndez-Ferrer and colleagues are planning a Phase II study in Europe to test the effects of beta-3-sympathomimetic agonists on the disease course and mutant allele burden in MPN patients. That’s the technical part – but it could lead to extremely good news.

Looking forward – eagerly

There’s a lot happening in MPN research – probably more than at any time in the past. I look forward to reporting significant advances on a regular basis. Look for my next update after the American Society of Hematology Meeting (ASH) in December.

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