NIH ANNOUNCES SECOND PHASE OF RARE DISEASE CLINICAL RESEARCH

Wednesday, October 07, 2009

NIH ANNOUNCES SECOND PHASE OF RARE DISEASE CLINICAL RESEARCH

HHT RECEIVES FUNDING FOR BRAIN AVM RESEARCH

OCTOBER 6, 2009 -The National Institutes of Health just announced a second phase of funding for the Rare Diseases Clinical Research Network (RDCRN).This research award will be funded over the next five years.Originally launched in 2003, the RDCRN addresses rare diseases through consortia and supports related to natural history, epidemiology, diagnosis, and treatment.

Historically, patient recruitment has been one of the primary barriers to rare disease research sincea raredisease is defined as acondition that affects less than 200,000 people in the United States. The RDCRN was authorized in legislation known as the Rare Diseases Act of 2002 to address this problem.

Each consortium in the network includes relevant patient advocacy groups. These patient advocacy group representatives serve as research partners within their own consortia. Collectively, the Coalition of Patient Advocacy Groups (CPAG) represents the perspective and interests of all patient advocacy organizations associated with the RDCRN. One of the requirements of the grant was the involvement of a strong patient advocate group. As a result, the HHT Foundation is an integral partof CPAG .

READ the NIH Press Release about the second phase of RDCRN and the 19 new and returning consortia. The Brain Vascular Malformation Consortium is the third project listed and HHT is part of this consortia.

HHT FOUNDATION ADVOCATES FOR
BRAIN AVM RESEARCH

I am very excited to share the press release from the Office of Rare Disease and the funding of the National Institutes of Health (NIH) Research Consortium for HHT. We have a seat at the NIH table after so many years of advocacy!

It was my privilege to represent our consortia as the patient advocacy organizationlast week.The NIH received over 60 consortia research funding applications. It is especially gratifying that our group was one of 13 newly fundedconsortia.

Brain Vascular Malformation Consortium

Principle Investigator: William Young, MD - University of California, San Francisco

NIH Collaborators: ORDR, NINDS

Diseases to be Studied: Vascular malformations, Cerebral Cavernous Malformation progression, Sturge-Weber Syndrome, and HHT

I wish to express my sincere gratitude to Dr. William Young, University of California, San Francisco and Dr. Marie Faughnan, University of Toronto for leading this initiative. In addition, this research would not be possible without the participation of our HHT Centers of Excellence at the Mayo Clinic, Medical College of Georgia, Oregon Health Sciences University, University of Alberta, University of Pennsylvania, University of Toronto, University of Utah, Washington University, and Yale University School of Medicine.

One requirement of this proposal was the involvement of a strong patient advocacy organization. I am thrilled to work toward theadvancement this crucial research and the mission of the HHT Foundation!

Warmest Regards,

Marianne S. Clancy

Executive Director

HOW DOES THIS RESEARCH BENEFIT ME?

This will be the first large-scale study of brain AVMs in HHT patients, who will be recruited throughnine HHT Centers across North America and through the HHT Foundation. The goal of the study is to determine what genetic and clinical factors signal high risk for hemorrhage from brain AVMs. This study should ultimately help doctors make decisions about brain AVM treatment for individual patients and will drive further research in brain AVM therapies.

HOW CAN I PARTICIPATE IN THIS

BRAIN AVM RESEARCH STUDY?

If you have HHT and have been diagnosed with a Brain AVM, whether the Brain AVM has been treated or untreated, you will be eligible to participate in this extremely important Brain AVM Research Study.

TO LEARN MORE... ATTEND ONE OF THE UPCOMING HHT PATIENT AND FAMILY CONFERENCES!

EAST COAST: If you live on the east coast, it would be extremely beneficial for you to attend our Mid-Atlantic Region Patient and Family Conference in Philadelphia on Saturday, November 7th. Dr. Marie Faughnan will be speaking about the NIH-Funded Multi-Center Research Studies.

WEST COAST: The HHT Foundation will be hosting a regional conference on the west coast in Spring 2010. The date and location are to be determined.

CENTRAL US: The HHT Foundation's National Patient and Family Conference will take place in St. Louis, Missouri on October 22-24, 2010.

IF YOU WANT TO HAVE YOUR NAME ADDED TO THE PARTICIPANT LIST...

Call or email the HHT Foundation to put your name on the "interested" participant list. Once the study protocol is complete, we will provide you with more information on how to move forward as a research study participant.

HHT Foundation International

P.O. Box 329

Monkton, MD21111

800-448-6389 (U.S.)

410-357-9932 (International)

Email: nicole.schaefer@hht.org

Website: http://hht.org

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