Amyloid is an abnormal protein produced in the bone marrow. Amyloidosis is a rare disease caused by extracellular and intracellular deposition of amyloid which alters the normal activities of the tissue. Commonly affected organs by amyloidosis encompass kidney, heart, nervous system, digestive tract, liver and spleen. Severe form of amyloidosis in later stages can lead to permanent organ failure and in extreme cases can be life-threatening also. There are over 30 different types of amyloid proteins discovered so far and treatment can only help in managing the symptoms and regulating the production of amyloid protein and not the permanent cure. About 90% of the amyloid formed due to the aggregation of misfolded proteins whereas, 10% of amyloidosis is due to presence of components such as apolipoprotein-E (apoE), glycosaminoglycans (GAGs) and serum amyloid P-component (SAP). According to American Society of Hematology the prevalence rate of amyloidosis in the United States is approximately 1 in 90,666 of which 60 – 65 % of the patient are men. Prominent symptoms of the disease include fatigue, weight loss, breathlessness, peripheral oedema, carpal tunnel syndrome, postural hypotension and sensory change. In the United Kingdom systemic amyloidosis is responsible for approximately one in 1,200 deaths each year.
Amyloidosis can be broadly classified into 3 major categories namely AA amyloidosis, AL amyloidosis and Transthyretin amyloidosis. In general amyloidoses are generally denoted by a capital “A” signifying the amyloid present followed by an abbreviation for the fibril protein. AA amyloidosis is a normal sequence serum amyloid – A protein produced mainly in the liver in response to multiple cytokines. AA mostly occur in various chronic inflammatory disorders and is one of the most common systemic amyloidosis across the globe. Some of the conditions associated with AA include: rheumatoid arthritis, alzheimer disease, psoriasis, tuberculosis, renal cell carcinoma, hodgkin disease and non-hodgkin lymphoma.
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AL amyloidosis or light chain amyloidosis monoclonal plasma cell disorder closely related to multiple myeloma. Organs involved in the AL amyloidosis include heart, kidney, peripheral nerve, gastrointestinal tract, respiratory tract and other organs. Treatment for AL amyloidosis in most instances is similar to multiple myeloma i.e. chemotherapy. Selected patient are treated with high-dose melphalan and autologous stem-cell transplantation. Alternative therapy includes rituximab, etanercept, thalidomide, iododoxorubicin and lenalidomide. In about about 80% the cases AA amyloidosis is fetal with median survival rate of 1-2 years only. Transthyretin amyloidosis (ATTR) is one of the most frequent inherited systemic forms of amyloidosis. ATTR is an autosomal-dominant disease but is associated with at least 100 different transthyretin mutations. Other forms of amyloidosis include heavy-chain amyloidosis, beta2 -microglobulin amyloidosis, hereditary renal amyloidosis and Central nervous system amyloidosis.
Major drivers for the growth of the global amyloidosis market include favourable investment and reimbursement facilities in developed economic countries. On the other hand high cost involved in treatment and limited awareness may hinder the growth of the overall market. Biopsy is the widely accepted diagnostic tool for amyloidosis and in about two-third of the patient alkylator-based chemotherapy is effective in managing amyloidosis. Noninvasive tests such as echocardiography of the heart and X-ray of bone abnormalities may provide supportive information but are not sufficient for the diagnosis of amyloidosis. Some of the prominent players operating in global amyloidosis market include Pfizer Inc., GlaxoSmithKline plc, Celgene Corporation, Alnylam Pharmaceuticals, Inc., Takeda Pharmaceutical Company Limited, Prothena Corporation plc. Premier institutes and universities promoting the development of amyloidosis clinical trial studies include Stanford University, Boston University, IRCCS Policlinico San Matte and Umea University.