Transcript Influencing Change in Research, Treatment Protocols, and New

Influencing Change in Research,
Treatment Protocols, and New Drug
Development
Mission/History
The MPN Research Foundation is a non-profit corporation established
in 1999.
The Foundation’s primary objectives:
•
Stimulate original academic science and pre-clinical research in
pursuit of new treatments and eventually a cure
•
Foster communication and collaboration in the scientific
community in order to accelerate research of MPNs
•
Create a powerful patient advocacy group in order to educate
patients and the public, and to influence government and
industry health organizations on issues affecting MPN patients
Accomplishments
Medical Research
• To date, the MPN Research Foundation has
awarded over $8 million dollars for MPN medical
research and will commit up to $2 million more
over the next 2 years
• Awarded thirty multi-year research grants
• Established a Scientific Advisory Board of
accomplished researchers
Website Statistics
Over 9,000 unique visitors per month
MPN Research Foundation Objectives
Continue to fund and/or support the most relevant and ultimately
effective MPN research
Increase membership to include as much of MPN community as
possible
Seek ways to bring the power of our growing MPN database to bear
on issues of interest to the MPN community (e.g., clinical trials, FDA
approval, reimbursement issues, environmental studies, etc.)
Provide the most comprehensive and accurate information on MPN
science and treatment to the MPN community through website,
symposia, and other channels
MPN Research Foundation Capitalizes on Key
Scientific Discoveries
•Following the JAK2 gene mutation in created MPN Research Alliance which
moved this discovery from the bench to the bedside
•Funded first large-scale bio bank of MPN patient samples. The Mayo Clinic
collected patient tissue samples which has provided critical support to
understanding causes and disease progression.
•Harnessing the power of full genome sequencing
•Funding two grants for genetic sequencing of key cohorts of MPN patients
•Data deposited in dbGaP to be shared with qualified researchers worldwide
Scientific Advances
•Funded first large MPN scale tissue bank and associated data bank at Mayo, enabling rapid testing
of new JAK2 inhibiting drugs.
•Funded MPD Research Alliance that enabled pre-clinical studies of JAK inhibitors in animal models
of MPNs.
•Funded the development of a knock-in JAK2V617 mouse model which enabled the identification
of an MPN stem cell population that is not affected by JAK2 Inhibitors.
•Funded continued research on the TET2 mutation and the effects of TET2 loss on MPNs, establishing
the precise frequency of the TET2 MPN mutation and its correlaton with the MPN phenotype.
•Funded the identification of Ikaros and CUX1 as genes that are lost during progression to AML
possibly leading to new targets for therapeutic intervention.
•Funded a study to understand the basis of interferon, specifically pegasys, mechanisms of action in
treating MPNs, in anticipation of a phase 3 study of this drug in polycythemia vera patients.
•Supported SNP array and candidate sequencing projects that have identified several genomic loci
that are altered in MPNs. More research in these areas is ongoing.
Recent Grant Programs
•New Investigator Awards
•Established Investigator Awards
•Donor Directed Awards
MF Challenge
2012 New Initiative
•What is the MF Challenge?
LLS & MPNRF Partnership
Focus of initiative on MF and fibrosis
Concept grants for New Ideas
Challenge Grant reviews June 2012
Where the MPN Research Foundation Gets
Funding
•Patients, Family Members
•Foundations
•Biopharma
•Partners
What is the MF Challenge?
•Scientific Goal
•Approach
•Partners
Background
•Consequences of Myelofibrosis
•Unmet need
•Perfect timing
Funding the MF Challenge
•Initial Funding
•MFC Goal
•Follow-up Funding
•Timeline
MF Challenge Outcome
•Identify fibrosis cause/reversal/cure
•Advance MF science
•Strengthen partnerships to reach shared goals
•Platform possible new drug development