UC San Diego Health First in Nation to Offer New Treatment for Hereditary Amyloidosis

UC San Diego Health is the first hospital in the nation to offer a new injectable medication to patients with nerve damage caused by hereditary transthyretin amyloidosis, a rare disease that can be fatal without management.

Previously, patients with this systemic disease were tethered to long, intravenous infusion therapy sessions every three weeks. Now, they receive one injection every three months.

UC San Diego Health began offering the treatment in July.

The injection is specifically indicated for polyneuropathy, a condition where nerves are damaged by hereditary transthyretin amyloidosis.

“This medication fits under a class of drugs called ‘silencers,’ which decrease production of the protein that creates amyloid,” said Marcus Urey, M.D., cardiologist at UC San Diego Health and assistant professor of medicine at UC San Diego School of Medicine, in a written statement. 

“After receiving the medication in clinical trials, polyneuropathy or pain caused by amyloidosis progressed much slower and symptoms improved in some patients,” Urey said.

There are more than 30 types of amyloidosis that can affect different organs. Hereditary transthyretin amyloidosis occurs when an abnormal protein accumulates in the bloodstream and eventually deposits in a person’s organs, causing them to become dysfunctional and, in some cases, fail.

Symptoms can range from shortness of breath, numbness in the hands, constipation or diarrhea.

Often affecting multiple organs, patients with amyloidosis require a multidisciplinary team of medical experts for treatment.

UC San Diego Health is one of a few centers in California that utilizes a comprehensive approach for treating amyloidosis. Obtaining the Food and Drug Administration-approved silencer drug, Amvuttra, was made possible by a team of medical experts.

Urey believes this is just the beginning in finding a potential cure for amyloidosis.