Children's Cancer Therapy Development Institute

Due Diligence, Overdue ... ?

In 2014 Steve Perrin from ALS-TDI published an editorial in Nature entitled, "Make mouse studies work". While we ourselves believe in mouse models, Dr Perrin makes important points that persist as first principles – and are worth mentioning to safeguard patients and stockholders alike.

In summary, for then and now, many promising treatments fail in human clinical trials because preclinical animal studies are often poorly designed. Although over 80% of drugs that succeed in animal studies fail in humans, this discordance is often due to poorly designed preclinical research rather than the animal models themselves. Weak animal studies can lead to costly and ineffective clinical trials, wasting money and valuable time for patients with terminal illnesses. In ALS research, successes inspired new guidelines to improve animal testing by using better disease assessment, monitoring, and statistical models. While these methods require more time and resources, they reduce false positives and help prevent unnecessary clinical trials, ultimately saving both money and lives.

To read Lucy's full summary and perspective, see https://artisanbio.com/due-diligence-overdue/

Hot Off the Press! This Scientific Reports paper from cc-TDI describes the beautiful complexity of at least 11 (yes, eleven) subtypes of cancer cells found in rhabdomyosarcoma. Curiously, human cell lines only reflect a few.

This work was done by cc-TDI lab members Guak-Kim Tan and Zu-Yuan Qian with help from cc-TDI student researchers Sofia Tosoni and Gillian St John. Collaborators included Kavya Kannan, Hollis Wright and Melvin Lathara (datma/ODA), Sonja Chen (Nationwide Children's Hospital) and Brian Rubin (Cleveland Clinic). Acknowledgements go to Mattie Clark for important technical contributions.

Funding was made possible by Avery's Rhabdo Blast! project, the Ally Anderson AlveolarRhabdomyosarcoma Research Fund at Arms Wide Open Childhood Cancer Foundation, The Megan's Mission Foundation and NIH.

For the full article, https://www.nature.com/articles/s41598-026-57841-3