CAMBRIDGE, Mass., Aug. 9, 2016 /PRNewswire/ -- World-renowned scientists and industry leaders converged on Cambridge, MA in early August to present the latest research on Huntington's disease and other devastating brain diseases. The occasion was the Hereditary Disease Foundation's 10th biennial "Milton Wexler Celebration of Life" Symposium, to promote scientific collaboration and innovation. The goal is to accelerate the translation of lab findings into treatments and cures that impact patients' lives.
One of the significant and promising developments discussed by the approximately 300 participants at the Symposium is a Phase 1 clinical trial of a drug called IONIS-HTTRx, which could potentially be the first to target the cause of Huntington's disease. Scientists at Ionis Pharmaceuticals are currently investigating the safety, tolerability and activity of IONIS-HTTRx in patients with early stage Huntington's disease. The drug is designed to reduce production of the huntingtin protein in individuals who have the disease. The current trial will evaluate the safety of IONIS-HTTRx. If it is successful, later-stage trials will be conducted to determine if the drug is effective. It is hoped that reducing the levels of the abnormal protein in this way will slow or halt disease progression.
Huntington's disease is a devastating hereditary disorder which usually strikes between the ages of 30 and 40, causing involuntary movements, loss of intellectual abilities, severe emotional disturbance, dementia, and eventually death. The disease is passed from parent to child through an abnormality in the huntingtin gene. Each child of an affected parent has a 50% chance of inheriting the genetic mistake. Everyone who inherits the abnormal gene will eventually develop the disease.
Dr. Nancy S. Wexler, President of the Hereditary Disease Foundation, noted the significance of this conference as a means of bringing together some of the world's leading researchers: "There are brilliant scientists from around the world working to conquer Huntington's disease and other brain bandits. The Hereditary Disease Foundation's ability to provide funding and nurturing support to these scientists helped lead to the discovery of the very first DNA marker for a genetic disease and then to the discovery of the gene that causes Huntington's disease. The exciting research presented as this year's Symposium suggests that we are on the brink of making new discoveries that will bring hope and healing to the millions of patients and families in the United States and worldwide who are affected by brain disorders."
About Hereditary Disease Foundation
Established in 1968, the Hereditary Disease Foundation is dedicated to finding treatments and cures for a wide range of devastating brain disorders that are estimated to impact millions of people in the United States and worldwide. Huntington's disease has been a major focus for the Foundation, which facilitates collaborative and innovative scientific research to further the understanding of this dreaded disease. A genetic disorder that strikes in the prime of life, Huntington's disease destroys brain cells, bringing on severe and progressive declines in personality, cognitive ability, and mobility.
It was work organized by the Hereditary Disease Foundation that led to the discovery of the genetic marker for Huntington's disease in 1983. Building upon this breakthrough, the Foundation organized and funded a decade-long international collaboration of over 100 scientists in an effort to find the gene. These efforts bore fruit when in 1993 this team of scientists was able to discover the actual gene that causes Huntington's. This work played an essential role in the development of the Human Genome Project. As a disease caused by a mistake on a single gene, Huntington's disease is an ideal model for other brain disorders. Progress toward treatments and cures for Huntington's disease can help in finding ways to treat other illnesses with more complex genetics, such as Parkinson's, Alzheimer's and Lou Gehrig's disease (ALS). For more information, visit http://hdfoundation.org/.
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SOURCE Hereditary Disease Foundation