Eprodisate and Tramiprosate - two small-molecule compounds for the treatment of amyloidosis
Xianqi Kong, Ph.D.
VP&CSO, Risen (Suzhou) PharmaTech Co., Ltd.
Amyloidosis is a group of diseases in which abnormal protein, known as amyloid fibrils, builds up in tissue. Symptoms depend on the type and are often variable. They may include diarrhea, weight loss, feeling tired, enlargement of the tongue, bleeding, numbness, feeling faint with standing, swelling of the legs, or enlargement of the spleen. There are about 30 different type of amyloidosis, each due to a specific protein misfolding. Some are genetic while others are acquired. They are grouped into localized and systemic forms. In the developed world about 1 per 1,000 people die from amyloidosis. Among amyloidosis, amyloid A is associated with end stage renal disease (ESRD), and amyloid beta (Aβ) with Alzheimer's disease (AD). This talk will introduce the studies on amyloid deposition mechanisms, and the discovery and development of small-molecule therapeutic agents, Eprodisate and Tramiprosate, for the treatment of ESRD and Alzheimer's disease.