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A protein puzzle: Untangling the mysterious condition of amyloidosis

Nature Medicine volume 23, pages 266–269 (2017)Cite this article

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A Correction to this article was published on 07 April 2017

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Pepys and his team had been treating the patient with a combination therapy for a degenerative disease known as amyloidosis. The condition arises when amyloid clumps—aggregates of misfolded proteins that stick together—build up in organs. Pepys has been working to find a treatment for amyloidosis since the 1980s, but he describes the progress made thus far as “agonizingly slow.” Even after his success with Patient 8 in 2013, the combination treatment is only just recently beginning to be tested in a phase 2 trial.

Scientists have known about amyloidosis for more than 160 years, but initially thought—mistakenly, on the basis of iodine staining of the tissue—that it was starch buildup. Its name reflects this history: amylum is Latin for starch. In 1959, scientists discovered that these aggregates were made of protein, and within a decade, discerned that the proteins took the form of zigzagged structures known as β-pleated sheets. A specific type of amyloid protein called beta-amyloid is commonly associated with Alzheimer's disease. It remains unclear whether the accumulation of beta-amyloid plaques is a causal driver of disease pathogenesis in Alzheimer's, however; as such, its classification as a true form of amyloidosis is an ongoing matter of debate among experts. By contrast, in the kind of amyloidosis that Pepys treats, scientists largely agree that the presence of amyloid deposits is what causes disease.

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Change history

  • 23 March 2017

    In the March 2017 issue, the piece “A protein puzzle: Untangling the mysterious condition of amyloidosis” (Nat. Med. 23, 266–269, 2017) incorrectly stated that the FDA placed Ionis’ NEURO-TTR trial on hold. The FDA had placed a related trial called CARDIOTTR on hold after some patients in the NEURO-TTR trial developed complications. The error has been corrected in the HTML and PDF versions of this article.

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  1. Shraddha Chakradhar is Nature Medicine's associate news editor in Boston.,

    Shraddha Chakradhar

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Chakradhar, S. A protein puzzle: Untangling the mysterious condition of amyloidosis. Nat Med 23, 266–269 (2017). https://doi.org/10.1038/nm0317-266

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