The MDA/ALS Center of Hope provides an important setting for research towards understanding, treating, and curing ALS. That is why the ALS Hope Foundation supports an extensive clinical research program at the Center of Hope. Without research, there would be no progress in our fight against ALS. Clinical Research is a broad term, encompassing clinical trials (studies on the effect of a medicine on the disease or its symptoms) and clinical research that examines other aspects of the illness, such as assistive technology or quality of life. Clinical research at the Center complements our basic research program, in the hope that promising therapeutics identified at the lab bench can be tested for efficacy in the clinic.
For more information, please contact our Research Nurse Coordinator, Kathleen Hatala, at Kathleen.Hatala@tuhs.temple.edu.
Clinical Research: Ongoing Projects
Tirasemtiv Clinical Trial
We are participating in the Phase III clinical trial of the experimental drug called Tirasemtiv to examine whether this drug improves lung function and muscle strength in people with ALS. Previous studies have demonstrated that Tirasemtiv is safe and well-tolerated when administered over 14 days. This drug improved muscle function in SOD1(G93A) mice, the animal model of ALS. The major side effect was dizziness. Learn more about Cytokinetics.
While ALS primarily affects the motor system, up to 40% of PALS have cognitive impairment with predominantly executive dysfunction, while approximately 20% of PALS show behavioral changes, predominantly apathy and disinhibition. These cognitive and behavioral changes add significantly to the burden and distress of caregivers who now have to face the challenges of managing behavioral changes in addition to physical disability. Given this increasing burden, there is a critical need for validated interventions that can help the caregiver manage these behaviors. However, there is no standard and/or validated intervention currently available, nor has there been investigation directed at helping caregivers to manage these behavioral changes in ALS. The overall goal of our study is to characterize the relationship of caregiver burden to cognitive and behavioral impairment in ALS and to develop and perform a feasibility study of a pilot individualized intervention as a way to reduce caregiver burden. Ultimately, this study will provide the necessary background to develop, inform, and guide future targeted and larger scale interventions directed to both PALS and caregivers.
EEG-Based Brain-Computer Interface
By using EEG (brain wave) signals from the scalp to create a signal, the Brain-Computer Interface (BCI) allows people to make selections from a computer screen. This study is intended to evaluate both the complexity of the system and the degree to which each participant will be able to communicate using BCI. Trials will consist of asking the subject to follow a series of simple instructions and to complete certain tasks while using the BCI in our clinic or offices. We are also working to simplify the system as a prelude to home based use.
In a disease like ALS in which the cause is unknown, no animal models of ALS can substitute for understanding how it affects humans. We are currently collecting blood, urine, cerebrospinal fluid, and autopsy materials from people with ALS and other motor neuron diseases to look for clues in the human tissue. All samples are tied to de-identified clinical information in a database to help maximize the usefulness of this precious resource. The anonymous database includes demographic information, environmental exposures, and medical history. This will increase availability of human tissue for research with pertinent corresponding clinical information.