XVth International Symposium on Amyloidosis
Twenty members of our research and clinical teams attended and made presentations at the XVth International Symposium on Amyloidosis in Uppsala Sweden in July. It was a wonderful opportunity to discuss amyloid research with investigators from around the world and to initiate collaborations. Several of the presentations were made by our students and trainees, and brief highlights of these and other contributions from our group are described in the following paragraphs.
Dr. Lawreen Connors and her laboratory team detailed basic studies on the transthyretin (TTR) protein focused on identifying differences in the amyloid forming properties among inherited TTR mutants and the age-related wild-type form. Dr. Olga Gursky, a longtime Boston University biophysicist and new member of the amyloid research team, presented structural stability studies of Apolipoprotein A1 showing what causes mutant forms of the protein to become amyloid deposits. Several basic studies on AL amyloid proteins included one from Dr. Elena Klimtchuk on a cell model that expresses both immunoglobulin heavy and light chains, an important tool to investigate how light chain proteins form amyloid fibrils. In another study Dr. Tatiana Prokaeva presented an analysis of a newly available test of HevyLite to identify clonal plasma cell dyscrasia in AL amyloidosis.
A number of clinical AL amyloidosis studies were presented by Dr. Vaishali Sanchorawala and the clinical team; these included the validation of a new renal staging algorithm, the value of the 6-minute walk test to help measure a patients’ functional status, and results from clinical trials of pomalidomide and idelalisib. Using our extensive patient database, Dr. Sanchorawala, in collaboration with Optum and Prothena Biosciences Inc., presented the quality of life in AL amyloidosis following treatment and the association between health-related quality of life and mortality. This is the first time such a detailed analysis has been performed or presented. It used data on 1822 patients with AL amyloidosis and documented significant health-related quality of life deficits in physical and mental functioning compared to a general population. The results showed that the most significant quality of life deficits were related to physical functioning. Also using our database, Dr. John Berk presented quality of life changes in patients with transthyretin amyloidosis, both inherited and age-related wild-type forms. Dr. Martha Skinner participated in a panel discussion on high-dose chemotherapy/stem cell transplantation in AL amyloidosis and she was chosen to give the final summary of the Symposium.
