These news stories were the top conversations in the field and were often included in NeurologyLive®'s coverage in movement disorders such as Parkinson disease, Huntington disease, and essential tremor, among others.
The world of medical and clinical news was alive with updates on the development of new agents, FDA decisions on promising therapies, and research into better understanding disease pathology and patient care in 2021. As these news stories broke, the NeurologyLive® team was working tirelessly to provide succinct coverage of the through lines by offering the latest information on announcements and insights from experts in patient care and from the industry to keep the clinical community up to date.
For patients with movement disorders, such as Parkinson disease (PD), essential tremor, Huntington disease (HD), and more, ongoing conversations covered topics such as the attempts to overtake levodopa’s standard and improve ON time in PD, the pipeline failures and costs of genetic testing in HD, and the attempts to find therapies for rare disorders such as Friedrich ataxia.
To offer a look back on some of those stories—many of which are still continuing and will extend into 2022—take a look at some of the coverage provided by the NeurologyLive® team that was most-viewed this year. These stories just scratch the surface of our coverage in AD. You can read more news in movement disorders, hear experts share insight on the top conversations in the field, and learn more about the ongoing discussions on our Movement Disorders clinical focus page.
Click the buttons below to learn more about each story.
To date, Duopa—combination levodopa/carbidopa (AbbVie)—remains the only treatment that offers up to 16 continuous hours of the therapies together to help control motor fluctuations in advanced PD. Its enteral suspension is administered using a small, portable infusion pump that delivers carbidopa and levodopa directly into the small intestine through a tube placed during an outpatient procedure. In February, AbbVie has announced that it would be conducting a new phase 3 study called M15-736 (NCT04380142), which would measure the efficacy, safety, and tolerability of continuous subcutaneous infusion of ABBV-951, its investigational soluble formulation of carbidopa and levodopa prodrugs (foslevodopa/foscarbidopa), in patients with advanced PD.1
Later in the year, in October, the company announced that ABBV-951 was statistically superior to oral levodopa/carbidopa in reducing motor fluctuations, meeting the primary end point of increase from baseline in ON time (measured in hours) without troublesome dyskinesia after 12 weeks based on the PD Diary. At the time, Michael Severino, MD, vice chairman and president, AbbVie, said in a statement that the company was “committed to addressing the continued needs of patients and are encouraged by these results that highlight a potential alternative treatment option for those affected by advanced Parkinson disease."
This news was reflective of the ongoing conversations in PD about addressing OFF time and developing therapies that can improve upon the gold standard set by levodopa. Shortly after AbbVie’s data announcement Fernando Pagan, MD, director, Movement Disorders Program, and medical director, MedStar Georgetown National Parkinson Foundation Center of Excellence, and his colleague Yasar Torres-Yaghi, MD, board-certified movement disorder neurologist, MedStar Georgetown University Hospital, developed continuing medical education (CME) topics for advanced therapeutics in movement disorders to attempt to help address this issue in the clinic.
The CME were being released by Parkinson & Movement Disorder Alliance (PMD Alliance) in partnership with the pair’s institution, MedStar Georgetown University Hospital, beginning October 22, 2021. These activities are a sneak peek of the PMD Alliance’s Advanced Therapeutics in Movement Related Disorders Congress, set for May 2022.
In September, at the International Parkinson and Movement Disorders Society (MDS) Virtual Congress 2021, data presented by David R. Lynch, MD, PhD, professor of neurology, University of Pennsylvania Perelman School of Medicine, showed continued positive effect of treatment with the investigational omaveloxolone (Reata Pharmaceuticals) in patients with Friedrich ataxia (FA).2 The results showed a difference in modified Friedrich’s Ataxia Rating Scale between the omaveloxolone and placebo groups at the end of the end of the placebo-controlled MOXIe Part 2, differences that were preserved at the end of the delayed-start period.
Lynch, speaking to the difficulty of treating FA, later told NeurologyLive® that “at this moment, the current unmet need is everything. That is to say, we have no way to slow the progression, and most of the symptomatology is fully treated in a very modest manner. [Omaveloxolone] doesn't directly address the individual symptomatology but does go at the mechanism of the disease, the events immediately downstream from the deficiency of the causal protein for frataxin.”
Similar challenges were observed merely weeks later in the spinocerebellar ataxia type 2 (SCA2) community, as a multicenter, double-blind, placebo-controlled trial found no evidence that riluzole (Rilutek; Sanofi) could improve clinical or radiological outcomes in patients with SCA2.
Also in September, it was announced that dosing had begun in the phase 1b/2a SELECT-HD trial (NCT05032196), which will evaluate Wave Life Sciences’ investigational agent WVE-003 in patients with Huntington disease (HD).3 SELECT-HD is a multicenter, randomized, placebo-controlled clinical trial that will assess the safety and tolerability of single and multiple-ascending intrathecal doses of WVE-003 in patients with early-stage HD who carry SNP3 in association with their cytosine-adenine guanine (CAG) expansion. It is expected to enroll approximately 36 patients, and is designed to be adaptive, with dose escalation and dosing frequency being guided by an independent committee.
The news came months after the company discontinued trials of its 2 other HD agents, WVE-120102 and WVE-120101, which failed to show efficacy. A similar failure was observed just days before when Roche announced the discontinuation of dosing in the phase 3 GENERATION HD1 study (NCT03761849) of tominersen, the first agent to successfully target and reduce levels of mutant huntingtin (mHTT) protein in patients with HD. Although, despite these setbacks, hope has remained in the HD pipeline, with a number of therapies retaining potential. In May, Voyager Therapeutics received FDA clearance for its investigational new drug (IND) application for VY-HTT01, a gene therapy candidate for the treatment of HD, which allowed it proceed with its planned phase 1/2 clinical trial.
In September, data from a study of HD Centers of Excellence (COE) suggest that there is another, perhaps less-discussed challenge in HD care: The pronounced variation in the cost of predictive genetic testing for HD despite the use of a standardized protocol among the centers. The average cost of undergoing the HD testing process was found to be $1157.12, ranging from as low as $275.00 to as high as $3640.00. Victor Sung, MD, director, University of Alabama at Birmingham (UAB) Huntington's Disease Clinic, codirector, UAB School of Medicine Neuroscience Module, and director, Birmingham VAMC Deep Brain Stimulation Program, told NeurologyLive® that although these average costs are only moderately high, “future studies should endeavor to better understand factors contributing to this cost variation between sites and explore other cost-related barriers in order to improve access to predictive genetic testing in HD.”