Neurological Application of Neurotoxins

In 2016, the foundation was pleased to select Lisa M. Williams, MD, as the recipient of the Clinical Research Fellowship Award in Neurological Application of Neurotoxins. Dr. Williams used this funding to continue her focus on dysphagia in neuromuscular diseases while completing a second fellowship at the University of California, Davis Medical Center (UCDMC). Her research efforts as part of this fellowship award began on August 1, 2016, and continued through July 31, 2017.   

“Dysphagia, difficulty with swallowing, is a common deleterious sequela of neuromuscular disease (NMD) associated with significant morbidity and mortality. The prevalence of dysphagia in NMD is variable with reports as high as 56% to 100% in some disorders. As dysphagia worsens, NMD patients commonly experience failure to thrive and are at increased risk of death. Currently, neuromuscular dysphagia research is lacking and there is a need to develop early screening and monitoring tools to assist with management strategies,” said Dr. Williams. 

The AANEM Clinical Research Fellowship Award has helped Dr. Williams’ work to complete three primary aims in neuromuscular dysphagia research, of which, her first aim has successfully been completed. 

“Our first aim was to define a unique classification system for neuromuscular dysphagia. Using a previously captured videoflouroscopy database, we discovered a unique swallowing measure characterizing NMD dysphagia. We found that the pharyngeal constriction ration (PCR), which is an indicator of aspiration, was elevated in patients classified with primary muscle disease or lower motor neuron disease as compared to patients with a predominantly spastic dysphagia. This is a novel measurement that is easily obtained on high resolution videofluoroscopy and may distinctly characterize NMD dyspaghia. PCR is a clinically applicable outcome measurement tool that may be used determine management strategies and efficacy of treatments,” noted Dr. Williams. 

Aim 2, which is currently underway, involves determining the reliability and validity of a comprehensive dysphagia clinical outcome instrument using the 10-item Eating Assessment Tool (EAT-10) in a specific NMD population for use in potential treatment applications for NMD dysphagia. 

Aim 3 will use EAT-10 to determine the effectiveness of management strategies of dysphagia from behavioral to interventional procedures such as Botulinum toxin (BOTOX) in a selective small cohort of patients.  

Dr. Williams is currently a Neuromuscular Medicine Attending at UCDMC. She works as a clinician in the MDA multidisciplinary neuromuscular medicine clinics at UCDMC and is continuing her work investigating dysphagia in NMD through her clinical research as a physician scientist.  

"Our research in neuromuscular dysphagia continues and we have formed a multidisciplinary approach in collaboration with UCDMC’s ENT department to work towards developing regular easily administered assessment and grading tools to inform management in NMDs,” she said.