The drug AT2101 slowed down the progression of Parkinson's disease in mice, in a new study, which if proven effective in humans, would make it the only drug on the market to go beyond merely treating the symptoms of the disease.
In the study, which was published in the October edition of Neurotherapeutics, researchers at UCLA genetically engineered mice to produce too much alpha-synuclein, to develop similar deficits of humans with Parkinson's. After four months of the mice receiving AT2101, their motor functions improved, brain inflammation stalled and levels of alpha-synuclein reduced (alpha-synuclein is a protein that accumulates in patients with Parkinson's, thought to cause the destruction of neurons in the brain that make dopamine). A total of 152 male mice were used in the study.
"This is only a first indication that the drug may work," said Marie-Francoise Chesselet, the study's senior author who also teaches neurology at UCLA and is the director of the UCLA Center for the Study of Parkinson's Disease. "Mice never mimic exactly what happens in humans, but this still has the potential to stop the disease from progressing."
The mice in the study did not carry a mutated Gaucher gene, a gene that causes Gaucher disease and less than five percent of Parkinson's cases. This means, according to Chesselet, that AT2101, a drug created by Amicus Therapeutics to treat Gaucher, could apply to the majority of Parkinson's patients who lack that mutated gene. It also "reinforces the notion that the enzyme that is abnormal in patients with Gaucher disease has something to do with Parkinson's disease," said Chesselet.
The main limitation of this study is that the drug was only given to mice at the earliest stage of the disease. Since the disease gets progressively worse over time for most, and many aren't diagnosed at the earliest stage, the drug working in later stages of the disease will be vital for its usefulness. Researchers hope, in future studies, to test the drug in more advanced stages of the disease.
A lot of people could benefit from a drug that slows down the progression of Parkinson's disease, as the disease affects around a million Americans, with 60,000 a year being diagnosed, and four million people worldwide. It's a degenerative disease of the central nervous system that causes tremors, body trembling, impaired coordination, slow movements and difficulty walking and talking. The onset of the disease happens, on average, at around 60 years of age and can cause complications that lead to death, like choking and pneumonia. It's rarely genetic and typically starts on one side of the body, eventually debilitating the other side of the body. The cause of the disease is unknown.
Parkinson's was not the original target of AT2101, as
"Clearly this technique has application in other diseases, like Parkinson's, which speaks to the promise of pharmacological chaperones like At2101," said William Baird, CFO of Amicus Therapeutics.
As of now, no cure exists for Parkinson's, though drugs such as carbidopa and levodopa and surgery can treat the symptoms. No drug has been proven to slow down the progression of the disease. That's why AT2101 could have large implications for Parkinson's and the people who suffer from it, if it goes on to work in humans at various stages of the disease.
