The Progression Marker Project
The Progression Marker Project
In 2015, the MPN Research Foundation assessed the state of MPN research and the Foundation’s relationship to funding science that is having a positive effect on the lives of patients. With only one FDA-approved medication developed since the discovery of JAK2 mutations, we were anxious to understand available prospects for breakthrough therapies for people with PV, ET, and MF. And if there were none, what could we do about it? Although we know many genetic markers for MPN, including correlation with prognosis, we do not have a complete understanding of how the disease progresses to more acute and deadly versions.
We took our idea to Srdan Verstovsek at MD Anderson and developed the idea for the Progression Marker Project. Srdan helped us identify how we might attack this, which is by a multi-year project that would collect samples and data from patients, using both their electronic medical records and their own self-reported experience with symptoms through our patient registry. Our scientific advisors John Crispino (Northwestern) and Raajit Rampal (Memorial Sloan-Kettering) rounded out the trio of advisors.
With this core team, we developed a five-year study during which time we’d enroll several hundred patients at multiple sites, collecting saliva and blood from them every 6 months. Samples would be tested for cytokines and screened for genetic changes, and all samples and data would be housed safely for subsequent analysis by qualified researchers who would be granted access to de-identified data by the Foundation. The goal: to identify genetic markers that lead up to progression from ET/PV to MF in order to identify additional targets for the development of therapies as well as better explain the mechanism by which this disease transforms, in the hope of stopping it.
According to Dr. Verstovsek, part of what is driving this effort is the need for more information “We need rigorous data to move physicians away from "watch and wait" to treatment strategies based on an understanding "why, how and when" people with MPNs should be treated. The Progression Marker Project is designed to provide us with this essential yet missing data.”
We have buy-in from some of the most illustrious MPN researchers around the country who believe this will be essential to getting to the next big discovery for MPN. Far too often the patients we serve are left with incremental improvements in quality of life, and nothing to prevent transformation to a more acute or deadly version of the disease. We want to ensure that there will be transformational therapeutic options for people with MPN in the next ten years. If all of us working on behalf of patients pull together in the same direction, there’s no reason we can’t have the kinds of breakthroughs we are seeing in other diseases.
Working Group: Srdan Verstovsek, Raajit Rampal and John Crispino
Consultants: Rick Winneker, Allison Formal and Helena Ellis of Biobanking Without Borders
Oversight: MPNRF’s Science Advisory Board and a Steering Committee were put in place for this project