National ALS Biorepository Pilot Study

ALS-Pilot Study

Project Details






The Need:The Agency for Toxic Substances and Disease Registry (ATSDR) needed a pilot study to determine the feasibility of creating a biorepository of specimens from persons with Amyotrophic Lateral Sclerosis (ALS) enrolled in the National ALS Registry. How We Helped: McKing developed a protocol with input from an expert panel made up of ALS researchers, neurologists, epidemiologists, advocacy representatives, and federal agency staff. Under this pilot study McKing conducted two collections of biospecimen samples (blood, urine, hair, and nail clippings) at 6-month intervals. Three hundred thirty (330) people from across the U.S. participated in at least one collection. For the postmortem part of the study, tissue sample collection includes the whole brain, spinal cord, cerebral spinal fluid, bone samples, and muscle samples for up to 30 participants. We are collecting a small skin sample from postmortem participants for a complementary contract to create fibroblast cell lines. Participants in this study have been recruited from enrollees in the National ALS Registry. Results: At the conclusion of the pilot study in September 2015, 339 participants were enrolled for biospecimen donation and 330 provided specimens on at least one occasion. For the postmortem part of the study, 30 participants have been enrolled, with 18 collections completed as of December 2015. McKing continues to maintain contact with the participants or their families on a quarterly basis to monitor the progress of the disease. Based on the Pilot Study Performance, ATSDR decided to move forward with establishing the National ALS Biorepository, which includes both collection of specimens and distribution of samples to researchers. McKing currently is helping ATSDR prepare the project protocol for IRB and OMB-PRA approval. Researchers will be able to apply for samples in Spring 2016 and collections are excepted to re-start in Fall 2016.