Scientists are launching the first human trials of a “life-changing” drug that can alleviate the outward signs of Parkinson’s disease.
Two non-profits, Parkinson’s UK and The Michael J. Fox Foundation, have partnered with Neurolixis to support a clinical trial testing the safety, tolerability and early efficacy of drug NLX-112 to help ease dyskinesia and the severity of some non-motor symptoms due to Parkinson’s disease.
NLX-112 tackles the shaking and twitchiness brought on by Parkinson’s treatments and could be available within a year.
NLX-112 works by targeting serotonin-producing brain cells, which are believed to contribute to dyskinesia by converting levodopa into dopamine and releasing it in an erratic manner. NLX-112 stabilises the flow.
NLX-112 is a new compound that is able to selectively activate a sub-type of serotonin receptor called the 5-HT1A receptor, which is usually found in serotonin-producing neurons. Once active, this receptor interferes with serotonin signalling cascades in these nerve cells, lowering their activity.
By reducing the activity of serotonin-producing neurons, NLX-112 is expected to alleviate symptoms of dyskinesia in people with Parkinson’s. Preclinical studies supported by Parkinson’s UK have shown that NLX-112 was effective at easing dyskinesia in animal models of Parkinson’s. These findings provided the foundation for the launch of clinical trials testing NLX-112 in patients.
The results, published in the Neuropharmacology journal, showed that NLX-112 reduced involuntary movements without diminishing the therapeutic effect of the levodopa. The new drug has also been tested on diabetes patients to treat pain and was found to be well tolerated and safe.
Human trials are being organised by a team at the Karolinska Institute in Sweden. They follow a deal between the Michael J. Fox Foundation for Parkinson’s Research in New York and Parkinson’s UK to fund the £1.5m clinical trials.
“We are excited to move to a proof-of-concept trial with NLX-112. The compound has a novel mechanism of action which has been extensively validated in laboratory tests,” said Adrian Newman-Tancredi, CEO of Neurolixis.
“If the positive effects seen in the lab translate into a clinical setting, NLX-112 could significantly improve the quality of life of many people with Parkinson’s.”
Altogether, Parkinson’s UK, The Michael J. Fox Foundation and Neurolixis have raised £1.5 million (about $1.7 million) to fund the two-year study, where Neurolixis’ experimental therapy for levodopa-induced dyskinesia (LID) will be given to Parkinson’s patients for a first time.
“Levodopa-induced dyskinesia can significantly impact quality of life for people with Parkinson’s. This collaboration with Parkinson’s UK is about combining our resources to advance this promising therapeutic approach from Neurolixis as quickly as we can to benefit the patients and families worldwide who navigate dyskinesia in their daily lives,” said Todd Sherer, PhD, CEO of The Michael J. Fox Foundation.
The clinical trial, which will be conducted by researchers at the Karolinska Institute in Sweden, will compare the safety and tolerability of NLX-112 to that of a placebo in 24 patients with dyskinesia. It will also focus on assessing whether NLX-112 can ease dyskinesia, as well as some of Parkinson’s typical non-motor symptoms, including mood and sleep issues. Study findings may enable NLX-112 to move into Phase 3 clinical testing, and potentially a request for its approval as a new disease treatment.