12 Apr A Parkinson’s ‘game changer,’ backed by Michael J. Fox, could lead to new diagnostics and, someday, treatments

By Matthew Herper, STAT

Michael J. Fox was sitting in his Upper East Side office surrounded by Emmys and an Oscar — one he received not for his acting but for his Parkinson’s philanthropy. (“Humanitarian stuff,” he said nonchalantly.) He wore blue trousers and a T-shirt, and Adidas sneakers with no socks. His hair, years ago always perfect, was a bit disheveled, and he was in constant movement in his chair, a hallmark of the Parkinson’s disease that has defined half of his life.

This past year has been particularly difficult for Fox. As he sipped Coke Zero through a straw — drinking is hard with Parkinson’s — the 61-year-old icon recounted how he had broken multiple bones in a fall, including some in his hand and his face.

“It’s been a terrible year,” he said.

But, he added, in some ways he was “feeling better.” He won that Oscar. A new documentary on his life will be coming out in May. And, most importantly, there was the scientific discovery he wanted to talk about.

“This is the thing,” he said. “This is the big reward. This is the big trophy.”

The trophy is science — and specifically research funded by the Michael J. Fox Foundation for Parkinson’s Research that has resulted in the clearest evidence yet that the presence of a particular misfolded protein, alpha-synuclein, can be used to determine if people have Parkinson’s. It is an advance that may soon be used to develop better diagnostics, but more importantly could rapidly accelerate the search for treatments for the disease.

The new findings, published in The Lancet Neurology, are the result of a 1,123-person study that has cost the Fox Foundation hundreds of millions of dollars since it began in 2010. Right now, alpha-synuclein can only be detected by taking a spinal tap, a difficult and uncomfortable procedure. But scientists say they hope that it could be detected in blood, a skin biopsy, or possibly even in a swab of the nose. An editorial in the medical journal called the test “a game-changer in Parkinson’s disease diagnostics, research, and treatment trials.”

The result is convincing in part because of the unique resource of patient volunteers that Fox was able to bring together, said Vikram Khurana, chair of movement disorders at Brigham & Women’s Hospital. Read more …