The recurrent observation of accumulation and aggregation of mutant proteins in different neurodegenerative disorders indicates the possibility of a shared pathogenic mechanism. Recent data suggest that elimination of mutant protein accumulation can lead not only to a halt of symptomatic progression but also to regression of the disease.
As a physician and a scientist, I study a rare disorder, Gaucher disease. I have been motivated to continue to study Gaucher disease for the past 25 years because I feel that our understanding of basic aspects of this disorder are still lacking, and that my patients with Gaucher disease, whom I have grown to know and love, will benefit from these discoveries.
Parkinson disease is a common disorder that is known to have a complex inheritance. This means that unlike Gaucher disease, which results from mutations in one specific gene, Parkinson disease can have many causes and many genes likely contribute to how the disease presents. Patients with Parkinson disease develop a resting tremor that often begins on one side, but they can also have slowed movements, unstable posture and gait, and at times, dementia. It affects about 1.5% of the population over age 65, and the risk increases with advancing age. There are disorders associated with Parkinson disease called the Lewy body dementias that have similar manifestations, but more progressive dementia.
The Parkinson’s and Brain Research Foundation is supporting leading medical scientists in the United States and Europe who are pursuing a 21st century approach to finding a cure for Parkinson’s disease. Prominent researchers at Harvard University, the National Institutes of Health, and University College London discovered the most common genetic risk factor for Parkinson disease – mutation of the GBA gene – a gene that causes a rare hereditary brain disorder called Gaucher. Carriers of this Gene are predisposed to the development of Parkinson’s – and – Recent and unexpected findings show the possibility of a shared pathogenic mechanism. The Parkinson’s and Brain Research Foundation is seeking a cure for Parkinson’s by funding medical research with prominent scientists who are focusing on the GBA Link. Medical history is awash with examples of progress being made on one disease as a result of links to another disease. Our International Scientific Advisory Board chooses cutting-edge multi-disciplinary medical research – research that takes a 21st Century approach – a new approach to solve the mystery of Parkinson’s.
One Small Discovery – can unlock the mystery. Your Gift – can help find a cure.
The only thing incurable, is our passion!
Ted Meyer was six years old the first time he got involved in medical research, by donating a sample of bone marrow. He had just been diagnosed with Gaucher disease, and his parents hoped their son’s participation might help him and others with the potentially fatal inherited metabolic disorder.