Dystonia Medical Research Foundation & Tyler's Hope
for a Dystonia Cure Join Forces to Pursue New Drug Targets

Two dystonia organizations are collaborating on groundbreaking medical research that could ultimately lead to new treatments for this debilitating neurological disorder. The Dystonia Medical Research Foundation (DMRF) and Tyler's Hope for a Dystonia Cure are co-funding the second phase of a contract with BioFocus, a drug target discovery company, to pursue targets for new potential dystonia drugs. The DMRF launched the first phase of the project in 2009.

The major objective of the project is to identify proteins that modify the effects of a genetic mutation that results in dystonia. This is accomplished by silencing selected genes and monitoring the effects on a protein called torsinA, which is abnormal when the dystonia gene mutation is present. The identified genes and proteins will be potential drug targets and used in subsequent drug discovery programs to develop therapeutics to alleviate dystonia symptoms. The first phase of the project was completed in 2010 resulting in an assay to test for genes and proteins that interact with torsinA. The project is now poised to enter phase two, which screens the assay.

"This is exactly the type of project Tyler's Hope is interested in funding--high risk, high return," says Rick Staab, President of Tyler's Hope. Two of Staab's three children are affected by dystonia.

"No one else is doing this work," says DMRF Vice President of Science Deborah Kilpatrick, PhD. "If we don't pursue this, who else will? We have an obligation to push the research forward to the next level. This is really exciting work." Dr. Kilpatrick lives with a form of dystonia that causes painful muscle contractions and spasms in the neck and shoulder muscles.

The treatments currently available for dystonia suppress symptoms without actually altering the disease process. No single therapy is effective for a majority of patients. The joint DMRF-Tyler's Hope contract with BioFocus is an effort to rationally design novel treatments specifically engineered to interrupt or reverse the dystonia disease mechanism at the cellular level.
 
Dystonia is a neurological movement disorder that causes muscles to contract and spasm involuntarily. The involuntary muscle contractions force the body into repetitive and often twisting movements as well as awkward, irregular postures. Dystonia may affect a single body area or be generalized throughout multiple muscle groups. Dystonia affects men, women, and children of all ages and backgrounds. Estimates suggest that no less than 300,000 people in North America are affected. Dystonia causes varying degrees of disability and pain, from mild to severe.

Founded in 1976, the Dystonia Medical Research Foundation (DMRF) is a 501(c)3 organization dedicated to serving all people with dystonia and their families. Since its inception, the DMRF has grown from a small family-based foundation into a dynamic membership-driven dedicated to advancing research, promoting awareness and education, and supporting the well being of affected individuals and families.

Tyler's Hope for a Dystonia Cure is a non-profit 501(c)3 organization founded in 2006 to passionately pursue solutions and a cure to the pain and limitations caused by DYT1 dystonia. Tyler's Hope is committed to funding neurology research to prevent and cure DYT1 dystonia; while creating awareness of this disorder.

BioFocus offers comprehensive target to candidate drug discovery services, backed by centralized project management.

For more information on the collaboration between the DMRF and Tyler's Hope, contact the Dystonia Medical Research Foundation at 312-755-0198 or dystonia@dystonia-foundation.org

 

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