A new compound dubbed a "molecular tweezers" has halted Parkinson's disease in its tracks in a promising test of the drug on zebra fish.
UCLA researchers have developed their compound to combat the a-synuclein protein, which, as they point out, is widely believed to lead to Parkinson's by clumping together and then killing brain neurons in a progression of toxic potency. Their new drug, known as CLR01, prevents the toxic clumps and breaks up existing ones that formed. It's dubbed a "molecular tweezers" because it binds to other proteins very selectively and precisely. The researchers say they generated some success in cell cultures and then a transgenic zebra fish model.
Lead researcher Jeff Bronstein, a UCLA professor of neurology, says the results are promising. But he also downplays them in a statement announcing the news.
"We've only stopped Parkinson's in zebra fish," he notes.
We would similarly lower expectations with the study, which is detailed online in the journal Neurotherapeutics. But Bronstein and his team are correct to remind us that the results are worth further consideration, because CLR01 worked "without any evidence of toxicity," and the drug could eventually generate similar results in people, serving as a vital tool to slow or stop Parkinson's and other related neurological disorders, like Alzheimer's disease. But so far, the bid to come up with a viable drug to stop Parkinson's--a nervous system disorder that affects movement and only gets worse--has fallen short. Some treatments help slow the disease, but they don't stop it.
At least the UCLA crew is moving ahead and testing the substance in mice. If all goes well, human trials are likely, the researchers say. The results, if as promising as the zebra fish tests, will generate some serious buzz.
- here's the release
- read the journal abstract