Early-stage trial for Parkinson’s disease therapy shows signs of promise

Scientists have reported early success in a trial of an experimental cell therapy for Parkinson’s disease, raising hope for patients. Bemdaneprocel therapy is at an early stage and the year-long trial involved just 12 patients, but the positive outcome is viewed as significant after decades of setbacks in the hunt for an effective treatment. Developed by BlueRock therapeutics, Bemdaneprocel was shown to be safe and the data gave indication that patients may have benefited.

“The data from this phase 1 open label study are extremely encouraging,” said Claire Henchcliffe, a neurologist at the University of California, Irvine, who was one of the study’s principal investigators. Click here to learn more.

New INGREZZA® (valbenazine) capsules show improvements in chorea associated with HD

Neurocrine Biosciences, Inc. has announced new data on FDA-approved INGREZZA® (valbenazine) capsules for the treatment of adults with chorea associated with Huntington's disease (HD), including exploratory results from the Phase 3 KINECT®-HD study, which showed consistently greater improvements in HD chorea with INGREZZA versus placebo from Week 2 to Week 12.

“The exploratory analysis from our KINECT-HD study further exemplifies the value of INGREZZA as a now approved medication for HD chorea with improvements seen with treatment as early as two weeks,” said Eiry W. Roberts, M.D., Chief Medical Officer at Neurocrine Biosciences. Click here to learn more.

Study into patients’ views of FA's impact opens in Europe

An observational study in Europe is using an app to better understand how Friedreich’s ataxia (FA) affects patients’ health-related quality of life, as well as their economic and psychosocial well-being. Called PROFA, the study is taking place across Germany, Austria and France and currently enrolling eligible patients, ages 12 and older.

“The in-depth and multidisciplinary real-time data assessment will provide a better understanding of FA's impact on patients’ everyday life, firming the basis for the design of improved care...and future clinical and healthcare research trials,” researchers wrote. Click here to learn more.

Lixisenatide shows promise in phase 2 trial as adjunct therapy for early Parkinson's disease

Recent findings from the phase 2 LixiPark trial showed that participants with Parkinson's disease (PD) treated with lixisenatide (Adlyxin, Sanofi) had improvements in motor function. These results suggest that the therapy, a glucagon-like peptide 1 receptor (GLP1-R) agonist, may potentially have disease-modifying effects when utilized as an adjunct to other treatments for PD.

Following one year of treatment, patients given lixisenatide daily showed less disability on the Movement Disorder Society-Unified Parkinson's Disease Rating Scale. Click here to learn more.

Research opportunity for HD patients and caregivers

Rare Patient Voice (RPV) is recruiting patients and caregivers living in the United States for an important Huntington's research opportunity. This is a 30-minute online survey, and the compensation is $60. Please sign up here to receive an e-mailed invitation to a screening survey to see if you qualify.

RPV connects patients and caregivers with the opportunity to voice their opinions through surveys and interviews to improve medical products and services, while earning cash rewards.