On June 22, the Adult Polyglucosan Body Disease Research Foundation hosted its first Biomarker Workshop to facilitate a vigorous exchange among researchers and patient advocates regarding potential biomarkers for drug discovery and development targeting APBD.
The Workshop — hosted on a virtual platform — was organized by the Foundation’s Pharmaceutical Development Committee and brought in well-known researchers within the APBD community as well as experts from other fields to open a dialogue on potential biomarkers.
Biomarkers can be specific cells, molecules, genes, gene products, enzymes, or hormones. They provide quantifiable biologic parameters that can be measured serially. Well-known examples include: hemoglobin A1c levels in diabetes, PSA for prostate disease, LDL and HDL in cardiovascular disease, and rheumatoid factor in rheumatoid arthritis.
Dr. Deberah Goldman, a member of the Foundation’s Board of Directors, shared her experience as a patient and the cry of her heart that time is of the essence for our patients. She shared, “I am hopeful that the scientists will focus their attention on APBD biomarkers to drive early clinical diagnosis, develop prevention strategies, and evaluate new therapeutic options. We currently have no treatments for APBD; we need a treatment or a cure now.”
The Workshop was attended by 30 clinicians, researchers, our Board of Directors and members of the patient community. We are thankful for the support of the Chan Zuckerberg Initiative in making this kick-off collaborative initiative possible.
Stay tuned as we work to translate the ideas and opportunities presented during this Workshop.
We look forward to sharing a summary of the Workshop proceedings with our community, as well as hosting a Q&A session to engage and educate our patients.