Myeloproliferative neoplasms: a precision medicine-based approach
Project Title: Myeloproliferative neoplasms: a precision medicine-based approach
Project Duration: 2023-2033
MOHCCN Consortium: Marathon of Hope - Québec
Investigators: Drs. N. Szuber, J-S. Delisle, L. Mollica, D-C. Roy, L. Busque
Partners:
- Maisonneuve-Rosemont Research Cente
- Groupe Québécois de Recherche en LMC-NMP
- Canadian MPN Group
Aim/goals:
- Prospectively/retrospectively collect and analyze samples from appropriately consented patient populations with primary or secondary (post-polycythemia vera/essential thrombocytosis) myelofibrosis (MF).
- Evaluate the interplay between age/gender and genomic constitution in these cohorts.
- Correlate genomic data with disease complication rates and survival outcomes.
- Identify novel putative therapeutic targets.
- Expand and enhance opportunities for patients and for research activities in rare blood cancers.
- Improve access to genomic precision medicine for patients across Quebec, including rural areas.
Summary:
Myeloproliferative neoplasms (MPN) are stem-cell derived hematological malignancies characterized by uncontrolled cellular proliferation. They are rapidly becoming a high-priority field of research due to their increasing prevalence, potentially devastating clinical consequences and high symptom burden, as well as lack of disease-modifying therapeutics, and the growing focus on principles of precision medicine as their complex genetic landscape is progressively elucidated. Clinically, MPN patients, particularly those with MF, suffer substantial and multi-dimensional disease burden (physical, emotional), with no available disease modifying therapy or effective cure. The economic burden of MPN is also high, with the lifetime medical cost of treating a single MF patient estimated at $266,000, including $228,356 of resource costs.