Amyloidosis Disease Treatment

No cure has been found for amyloidosis. Treatment consists of slowing disease progression, treating underlying causes, and reducing symptoms. Because of the wide range of organs susceptible to protein buildup, treatment must be individualized according to causes and symptoms.

Biopsy Samples and Pathology Results

A diagnosis of amyloidosis often begins when routine tests reveal high levels of protein in the urine. However, a definitive diagnosis requires the gathering and analysis of a biopsy sample. Biopsy samples for suspected amyloidosis are usually gathered from the abdomen, rectum, or bone marrow.

Pathologists are physicians who specialize in disease action at the cellular level. Amyloidosis deposits contain a protein called serum amyloid P (SAP). By exposing the biopsy sample to antibodies, pathologists can detect the presence of SAP. They may also stain the biopsy sample with a dye called Congo Red. Congo Red fuses to amyloid proteins. When examined under polarized light, the fused Congo Red proteins have a distinct apple green color.

Treatment

If amyloidosis results from an underlying condition, treatment concentrates on the primary disease. Treatment that cures, controls, or slows the underlying disorder will slow the progression of amyloidosis. If no underlying cause of protein deposits can be discovered, a number of choices are available to treat the disease itself.

Colchicine and FMF

The anti-inflammatory medication colchicine prevents acute attacks of gouty arthritis. Colchicine has produced positive results when used to treat Familial Mediterranean Fever (FMF), a hereditary form of protein buildup that produces periodic attacks of high fever and pain in the joints, abdomen and chest. Clinical trials indicate that colchicine may prevent FMF attacks.

Myeloma Drugs and Amyloidosis

If a plasma cell disorder is the suspected cause of amyloidosis, drugs used to treat multiple myeloma and Waldenstrom’s macroglobulemia may be effective. Plasma cell disorders cause increased bone marrow antibody production. Myeloma medications include the alkylating agents chlorambucil, melphalan, and cyclophosphamide.

Liver, Heart, and Kidney Transplants

If the heart, liver, or kidneys are affected, an organ transplant may be appropriate. Organ transplants do not cure amyloidosis; eventually, proteins will begin to accumulate in the transplanted organ. In a best-case scenario, years of symptom relief can be gained through transplants.

Clinical trials indicate that liver transplants are an effective treatment for the hereditary forms of amyloidosis. If the disease affects the kidneys, transplants and dialysis both improve survival rates.

Transplants have some drawbacks. Waiting lists for suitable donor organs are long. After an organ transplant, the recipient must use long-term immunosupressing drugs to prevent organ rejection. These drugs have a number of adverse side effects, including the risk of serious infections.

Clinical Trails: Stem Cell Transplants and Interferon Alfa

Clinical trials are examining new treatment methods. Peripheral stem cell transplantation treats the disease by replacing diseased stem cells (the immature cells used to make blood cells). High dose chemotherapy is used to kill diseased and abnormal stem cells. Healthy stem cells are then introduced to the body to replace the destroyed cells. The healthy cells may have been collected form the person undergoing treatment, or may come from a donor. Stem cell research has shown that the procedure may slow the progress of amyloidosis.

Clinical trials are also investigating the effectiveness of treating amyloidosis with a combination of chemotherapy drugs, the steroid dexamethasone and interferon alpha. Interferon alpha is a naturally occuring protein in the body that boosts the immune system, making it easier for the immune system to target abnormal cells.

Amyloidosis is a rare disease. Accordingly, the condition receives less funding and research involvement than more widespread disorders. Recruiting clinical trial participants is also made more difficult by the condition’s rarity. If you have been diagnosed with amyloidosis, please consider participating in a medical trial. Often clinical trials provide participants with novel therapies and professional care they would otherwise be unable to access.