Influencing Change in Research, Treatment Protocols, and New

Drug Development

Mission/HistoryThe 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

AccomplishmentsMedical 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 StatisticsOver 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

•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.

Scientific Advances

•New Investigator Awards

•Established Investigator Awards

•Donor Directed Awards

Recent Grant Programs

•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

MF Challenge2012 New Initiative

Where the MPN Research Foundation Gets Funding

•Patients, Family Members

•Foundations

•Biopharma

•Partners

•Scientific Goal

•Approach

•Partners

What is the MF Challenge?

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