KL-50 explained

KL-50 (also known as RR-004 by its developer Pharminox Limited) is a drug candidate designed to treat glioblastoma. It functions by alkylating DNA yielding O6-(2-fluoroethyl)guanine which causes DNA interstrand crosslinks. Half of the cancers are unable to repair the DNA damage.^{[1] [2] [3] [4]}

The KL-50 molecule was patented by Pharminox Limited in 2013, detailing its potential use in treating MMR-/MGMT- gliomas.^[5]

Notes and References

1. . 10.26434/chemrxiv-2023-zwj94 . Mechanism of Action of KL-50, a Novel Imidazotetrazine for the Treatment of Drug-Resistant Brain Cancers . 2023 . Huseman . Eric . Lo . Anna . Fedorova . Olga . Elia . James . Gueble . Susan . Lin . Kingson . Sundaram . Ranjini . Burgenske . Danielle . Oh . Joonseok . Liu . Jinchan . Menges . Fabian . Rees . Matthew . Ronan . Melissa . Roth . Jennifer . Batista . Victor . Crawford . Jason . Sarkaria . Jann . Pyle . Anna . Bindra . Ranjit . Herzon . Seth .
2. Huseman . Eric D. . Lo . Anna . Fedorova . Olga . Elia . James L. . Gueble . Susan E. . Lin . Kingson . Sundaram . Ranjini K. . Oh . Joonseok . Liu . Jinchan . Menges . Fabian . Rees . Matthew G. . Ronan . Melissa M. . Roth . Jennifer A. . Batista . Victor S. . Crawford . Jason M. . Pyle . Anna M. . Bindra . Ranjit S. . Herzon . Seth B. . Mechanism of Action of KL-50, a Candidate Imidazotetrazine for the Treatment of Drug-Resistant Brain Cancers . Journal of the American Chemical Society . 30 May 2024 . 146 . 27 . 18241–18252 . 10.1021/jacs.3c06483. 38815248 . 11409917 . July 10, 2025 .
3. Web site: Slow and steady action makes brain cancer drug candidate more selective . Chemical & Engineering News . en.
4. Leslie . Mitch . New Strategy May Thwart Glioblastoma Resistance . Cancer Discovery . 2 November 2022 . 12 . 11 . 2488–2489 . 10.1158/2159-8290.CD-NB2022-0055.
5. US . 2013338104 . application.