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Funding Research

CCM3 Action is committed to advancing research. To this end, CCM3 Action regularly awards seed grants for basic, animal model, or translational research projects that will significantly decrease the timeline for development of an effective therapy for CCM. The purpose of these seed grants is to provide funds to researchers to test new hypotheses and generate preliminary data that will facilitate submission of traditional grant applications in one year’s time.  

2013 Mitchell Asbury Memorial Award

In 2013, CCM3 Action awarded the $25,000 Mitchell Asbury Memorial Award to the laboratory of Dr. Douglas Marchuk at Duke University for a project entitled “Therapy for CCM3 Disease.”  From Dr. Marchuk’s project summary:

We have created mouse models with targeted mutations in CCM3… We have in parallel developed high resolution magnetic resonance (MR) imaging and stereotactic localization techniques which enable the detection and characterization of…lesions [in mice], even at the earliest stages of development.  Using this novel platform we will test the hypothesis that Ccm3 lesion burden can be reduced with either one of two drugs, fasudil, a specific Rho kinase inhibitor, and simvastatin, a non-specific kinase inhibitor in wide clinical use for other clinical indications.  This work shows whether these drugs that show efficacy in our Ccm1 and Ccm2 mouse models will show similar efficacy in Ccm3 mice. Our long term goal is to find new approaches to the prevention and treatment of all forms of CCM, including the most devastating form, CCM3.

2014 MadoroM Research Award

In 2014, CCM3 Action awarded the $25,000 MadoroM Research Award to the laboratory of Drs. Ian Scott and Brent Derry at the University of Toronto SickKids Hospital for their work with C.elegans (worms) and zebrafish. From their proposal:

We propose that the CCM3 signaling pathway is conserved from C. elegans to humans, and this provides a unique opportunity to exploit the powerful genetics of C. elegans to delineate the entire pathway.  Moreover, we will define in an unbiased manner the CCM3 pathway and conduct functional analysis of these genes in the neurovasculature of zebrafish.  The combined powers of using C. elegans to identify CCM3 pathway genes and the zebrafish neurovasculature to investigate conserved functions in a vertebrate model will lead to drug targets that can be exploited to treat patients.  In parallel, we will conduct a screen in the zebrafish CCM3 model of kinase inhibitors to search for potential therapeutic agents.

2014 Asbury-Lee Award

Additionally, in 2014, CCM3 Action will award $10,000 to the lab of Dr. Adriana Beltran at the University of North Carolina at Chapel Hill for her study “Patient Stem Cells to Study CCM3 Disease.”  A CCM3 Action family has donated blastocysts with a mutation in the CCM3 gene to Dr. Beltran’s lab.  This grant will enable these blastocysts as well as blood samples from individuals who have a CCM3 mutation to be used to further research as described by Dr. Beltran below:

I propose to generate human embryonic stem cells (hESCs) and induced pluripotent stem cells (iPSCs) as powerful novel models for studying CCM3 function, including phenotypes resulting from patient CCM3 mutations. These cells have the capability to self-renew and differentiate into endothelial cells, providing an unlimited source of human cells harboring CCM3 mutations for in vitro differentiation and small molecule drug discovery. The derived CCM3+/- hESC lines will be donated to the NIH Human Embryonic Stem Cell Registry to make them available to other investigators. This is a once-in-a-lifetime opportunity for both CCM researchers and patients.

Future Awards

In 2013 and 2014, CCM3 Action issued its request for proposals at the Angioma Alliance International CCM Scientific Meeting in early November with 3-page letter of intent submission deadlines in mid- to late-December. Awards were announced in early January and funds were disbursed in February. Additionally, in 2014, CCM3 Action limited award applicants to laboratories that had representatives in attendance at the scientific meeting. If funding allows, it is anticipated future awards will be announced at the International Scientific Meeting, held each November, and awards will be limited to meeting attendees.