Two UA researchers are heading efforts to find therapies for Parkinson’s disease patients that may provide alternatives to long-term reliance on medications.
Torsten Falk, an assistant professor of neurology, is one of the main researchers trying to identify the gene most compatible with neuron cells targeted by Parkinson’s.
He discovered and cloned the Kir2.3 gene, which involves the affected area in the brains of Parkinson’s patients. Thereby, he created a “”one-shot treatment”” that restores brain stimulation, he said.
Parkinson’s disease begins when a group of brain cells malfunction and die off, producing a chemical called dopamine, which is responsible for sending information to the brain. The amount of dopamine produced in the brain gradually decreases, slowing the delivery of messages and making it difficult for a person to perform or control basic movements.
The reason why the cells are vulnerable is unknown, but it may stem from environment or genetics, Falk said.
“”The current therapies affect the entire brain, not just the dopamine part, creating instability and increasing a person’s erratic movements,”” he said.
About 1.5 million Americans have Parkinson’s disease, and 60,000 people are diagnosed each year, according to the National Parkinson Foundation. The disease is the second-most-common neurogenerate disorder, affecting 15 percent of the population before the age of 50.
Many treatment options exist to help treat the
disease and its various symptoms, which include weak coordination and balance and body tremors, but there is currently no cure, said Brian McKay, an assistant professor of ophthalmology and vision science.
A gene therapy approach has been used on other diseases not related to the brain. But it may work for Parkinson’s because doctors are able to inject the gene directly into the problem area and thus diminish the danger of it spreading to other parts of the brain or body, Falk said.
McKay is working on another approach, using retinal pigment epithelium cells (RPE) to support neurons in the area of the brain affected by Parkinson’s.
While gene therapy uses viruses to stimulate neurons toward making a new protein, RPE treatment uses actual living tissue to help remaining neurons survive, McKay said.
This approach reduces Parkinson’s patients’ reliance on medication to treat symptoms, he said.
“”Our hope is that we can produce cells that will be useful in treating Parkinson’s disease and slow the progression of the disease,”” McKay said.
Although these approaches won’t rid patients of the disease, it will provide them with a better therapy and another option than their current medications, which are often insufficient in the long run, Falk said.
Falk estimated that the gene therapy treatment will be available to patients in clinical trials within the next five to 10 years.
“”Our research looks very promising,”” he said. “”But we will not know until we actually get there.””