The development of effective treatments for many proteopathies has been challenging. Because the proteopathies often involve different proteins arising from different sources, treatment strategies must be customized to each disorder; however, general therapeutic approaches include maintaining the function of affected organs, reducing the formation of the disease-causing proteins, preventing the proteins from misfolding and/or aggregating, or promoting their removal. For example, in Alzheimer's disease, researchers are seeking ways to reduce the production of the disease-associated protein Aβ by inhibiting the enzymes that free it from its parent protein. Another strategy is to use antibodies to neutralize specific proteins by active or passive immunization. In some proteopathies, inhibiting the toxic effects of protein oligomers might be beneficial. Amyloid A (AA) amyloidosis can be reduced by treating the inflammatory state that increases the amount of the protein in the blood (referred to as serum amyloid A, or SAA). In immunoglobulin light chain amyloidosis (AL amyloidosis), chemotherapy can be used to lower the number of the blood cells that make the light chain protein that forms amyloid in various bodily organs. Transthyretin (TTR) amyloidosis (ATTR) results from the deposition of misfolded TTR in multiple organs. Because TTR is mainly produced in the liver, TTR amyloidosis can be slowed in some hereditary cases by liver transplantation. TTR amyloidosis also can be treated by stabilizing the normal assemblies of the protein (called tetramers because they consist of four TTR molecules bound together). Stabilization prevents individual TTR molecules from escaping, misfolding, and aggregating into amyloid. Several other treatment strategies for proteopathies are being investigated, including small molecules and biologic medicines such as small interfering RNAs, antisense oligonucleotides, peptides, and engineered immune cells. In some cases, multiple therapeutic agents may be combined to improve effectiveness.
Proteinopathy
Multisystem proteinopathy
Bisalbuminemia
Cryoglobulinemia
Lipiduria
Hemoglobinopathy
Ageing
Proteinuria