Amyloidoses are rapidly progressive life-threatening diseases that can occur at any age. There are numerous forms of amyloid diseases, which also play a role in Alzheimer’s disease. In light-chain amyloidosis, plasma cells in bone marrow produce large amounts of defective antibody proteins that clump into large molecules. These are deposited in various organs, blood vessels and nerves and cause damage up to functional failure. An estimated 5,000 people per year suffer from light-chain amyloidosis (AL) in the EU. A late diagnosis followed by aggressive chemotherapy and expensive stem cell therapy shows only short-term improvement so far. Fifty percent of patients do not survive despite treatment or have to cancel because of side effects.
The causes of AL amyloidosis are so far unexplored. Alongside myeloma and lymph gland tumors, unexplained factors also account the for the sudden increase in the production of plasma cells. Amyloidosis frequently remains undetected for a long time because of various symptoms and thus develops into a disease with serious organ dysfunction and is often fatal. The disease could be treated with a targeted therapy with low side effects, and this could significantly improve the patients’ quality of life.
Orphanbiotec’s evaluation process enabled the rapid identification of a suitable molecule for light-chain amyloidosis. The next validation and efficient development of a novel drug preparation focused on the pathomechanism of amyloidosis will take place in order to be able to offer a specific treatment for this disease. This effective treatment with minimal side effects, combined with regular monitoring, significantly increases the survival rate of patients and allows them a high quality of life. A potential therapy would be the first in this field and makes it possible to reduce high health expenditures for non-standard therapies with serious side effects.