Our grantees at the Parkinson’s Institute have found new genes associated with Parkinson’s disease. These will help the scientists understand the origins of the disease, develop drugs and also screen for people who are at risk.
Until now, scientists knew only a small number of gene mutations that play a role in Parkinson’s disease. These are ones that you can get screened for through organizations like 23 and Me, which look for known mutations that can put you at higher risk of some diseases.
But many people who don’t have any of those known mutations still get the disease, and some who have the mutations never show symptoms. Clearly, there’s more going on in the disease than those few known genes would suggest. Scientists have thought there are probably additional genes waiting to be discovered, as well as environmental risk factors like exposure to certain chemicals.
The Parkinson’s Institute scientists worked with the Michael J. Fox Foundation and Population Diagnosics, Inc to find the additional genes. A press release on the Parkinson’s Institute website has more about the collaboration.
It’s true that finding a gene is a big leap from finding a cure, but it’s a step in that direction. One immediate benefit is that doctors might be able to do a better job of identifying people who might be at risk of developing the disease and help them get early treatment. That doesn’t stop the disease, but can certainly help people start managing their symptoms.
The genes will also help those scientists working to develop therapies, which is the stem cell connection in this research. We’ve funded the Parkinson’s Institute to collect skin samples from people with a genetic risk of developing Parkinson’s disease, then convert those skin cells into reprogrammed iPS stem cells and mature those into neurons.
The neurons they’ve already developed from people with Parkinson’s disease behave very differently than normal nerve cells. By studying those cells, the scientists can learn more about what causes the disease and also look for drugs that return those cells to normal function.
Now that they know about the additional genes, they can develop neurons in a lab dish from people with those mutations and compare them with other Parkinson’s neurons. Also, they might be able to learn more about whether some drugs work better in people with different mutations.
We have more about CIRM funding for Parkinson’s disease on our Parkinson’s disease fact sheet.
Amy Adams