Dr. John Berk the Assistant Director of the Amyloidosis Center
John Berk, MD, Assistant Director of the center, specializes in familial transthyretin and lung amyloidosis. Dr. Berk designed and conducted the first international randomized controlled trial for familial amyloid polyneuropathy. This NIH- and FDA-funded study found that Diflunisal, an obsolete non-steroidal anti-inflammatory drug, inhibited neurologic disease progression, validated a neurologic metric to map disease progression, generated natural history data critical to future study design, and provided proof-of-concept that protein stabilization could alter the course of a protein folding disorder. The study design also established a blueprint for subsequent ATTR amyloid neuropathy trials that subsequently led to two FDA- approved TTR gene silencing drugs. Dr. Berk serves on two Scientific Advisory Boards on the development of gene editing applications for ATTR amyloidosis and the development of TTR stabilizing drugs for leptomeningeal amyloidosis. Dr. Berk also described low-dose, external beam radiation to treat tracheobronchial amyloidosis, inducing apoptosis of clonal plasma cells generating airway amyloid, a non-surgical option for disease management.