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Ampyrone (4-Aminoantipyrine) is a Direct Agonist of Human Tyrosinase and Potential Therapeutic for Oculocutaneous Albinism and Disorders of Hypopigmentation.

Dolinska, M.; Wang, Y.; Coussens, N.; Kalaskar, V.; Eraslan, Z.; Grondin, S.; Bonica, J.; Toay, S.; Hall, M.; Shen, M.; Boxer, M.; Chen, Q.; Gross, S.; Attarwala, N.; Jittayasothorn, Y.; Alur, R.; Shukla, D.; Kee, R.; Deyoung, C.; Sha, C.; Adams, D.; Loftus, S. K.; Cogliati, T.; Sergeev, Y. V.; Zippin, J. H.; Brooks, B.

2025-10-15 pharmacology and toxicology
10.1101/2025.10.13.682036 bioRxiv
Show abstract

Significant loss of pigmentation can increase visual disability, skin cancer risk, and psychosocial stress. Tyrosinase (TYR) catalyzes the first and rate-limiting step of melanin synthesis. Inhibitors of TYR are well established and are currently used in clinical settings; however, there is a dearth of direct activators of TYR. Here, using a unique human TYR construct, high-throughput screening, and computational analysis techniques, we identified ampyrone as a TYR activator. Ampyrone increased the in vitro catalytic activity of the intramelanosomal domain of human TYR (hTYR) and its hypomorphic variant, P406L, a cause of oculocutaneous albinism type 1B (OCA1B). Moreover, ampyrone induced melanin synthesis in both wild-type and OCA1B human melanocytes, as well as 3-dimension (3D) human skin cultures. Our results reveal ampyrone as a lead compound for first-in-class TYR activators, potentially accelerating the discovery of novel therapies for patients with genetic and acquired diseases of hypopigmentation.

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