Antibody Immunotherapy for Systemic and Local Amyloidosis

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Amyloidosis is a group of rare but very serious, usually fatal diseases caused by the body's own proteins. Normally, proteins are safely destroyed or recycled at various times after production and are replaced by newly produced molecules to conduct their functions. However, there are about 30 different human proteins that tend to behave differently. Instead of being simply and safely destroyed and replaced, these proteins instead clump together in long strings to form microscopic, thread-like fibres, called amyloid fibrils. Mysteriously, for reasons that are not understood, amyloid fibrils are not promptly recycled or destroyed. Instead, they accumulate and persist in the spaces between cells in tissue and organs. The fibrils themselves are not directly harmful but, by enlarging the distance between cells and, especially, the distance of cells from their essential nourishing blood supply, amyloid deposits damage tissue structure and function, leading to disease. It is as if a full theatre with a play in progress was progressively and then completely filled with foam packing chips so that the audience could not see or hear the actors, find the exits, or, eventually, move or even breathe.Amyloidosis is difficult to diagnose and very challenging to treat. No treatments exist for some forms of the disease whilst treatments for other forms are very toxic and/or have limited efficacy. Existing treatments aim to reduce the amount of the particular amyloid-forming protein in each case, or to interfere with its conversion into amyloid fibrils. These approaches sometimes produce clinical benefit but, even so, many patients are treated too late and die before they can benefit.A novel approach was therefore devised by Professor Sir Mark Pepys FRS, a world leader in amyloidosis. He used specially designed antibodies to activate the body's natural defence mechanisms to directly remove amyloid from the tissues. This approach has proved successful, first in experimental animals and then in amyloidosis patients, providing clinical proof of concept. Together with Sir Greg Winter FRS, Nobel Laureate 2018, and world leading authority on antibody drugs, Immutrin Ltd was founded to create and develop novel, transformative, antibody treatments for amyloidosis.Exciting progress has delivered a unique range of new antibodies, now ready for the next drug development stage, which is conversion into a safe and effective human form that can be clinically tested in patients.