The Future of Parkinson's Disease Therapy

Today, there are more than 200 potential Parkinson’s therapies in clinical trials. A drug called IPX066 is undergoing FDA review, which is expected to be concluded by the end of 2012. Promising agents in more advanced stages of development include Preladenant, Pardoprunox, and ODM-101. 

Current research efforts to stop the progression of the disease—and eventually to cure Parkinson’s—focus on developing effective stem cell transplants, gene-based therapies, and agents that might protect brain cells from damage. 

In the future, doctors may be able to transplant healthy dopamine-producing cells into the brain and replace the cells that are lost due to Parkinson’s disease. Although this line of research is very early, important progress is being made. 

For example, researchers at Memorial Sloan-Kettering Cancer Center reported in 2011 that they created dopamine-producing brain cells from stem cells and implanted them into the brains of animals to learn how these cells would function.

Promising gene therapies are also in early research. In 2012, researchers in the United Kingdom reported on the first experimental gene therapy to succeed in generating dopamine in a patient’s brain. The treatment—tried in only 15 people so far—injected genes for three different enzymes linked to dopamine production. More studies are needed to measure the safety and effectiveness of such approaches. 

Genetics advances are providing a “molecular foundation” on which to build new therapies for Parkinson’s by interfering in processes that cause the disease and that make people susceptible to it. 

Researchers have identified a number of genetic mutations that play a role in Parkinson’s, including mutations of SNCA, a gene that makes the protein alpha-synuclein, and LRRK2, found in the DNA of significant numbers of people with Parkinson’s from certain ethnic groups. Both SNCA and LRRK2 are targets in drug research programs. 

Cowen and Company, Therapeutic Categories Outlook 2012 
Parkinson’s Disease Foundation. New Hope for Parkinson’s Stem Cell Therapy; Genetics: A Foundation for Future Parkinson’s Treatments

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