A successful example of a generic drug repurposed for off-label use is sirolimus, for the treatment of autoimmune lymphoproliferative syndrome (ALPS). In 2004, Cures Within Reach funded a single researcher to run a study of repurposed drugs in an ALPS mouse model, which in less than 36 months demonstrated potential efficacy of sirolimus, a generic transplant drug. The charity then provided further support for a pilot study in six patients, five of which were in complete remission after 90 days. Results were published open access to promote off-label use and monitoring of the treatment is ongoing in the literature. Cures Within Reach estimate that use of sirolimus saves $50,000 per patient per year and hugely improves their health and quality of life. Based on this success, Cures Within Reach have funded follow-on research to repurpose the same drug in five additional autoimmune diseases with remission seen in the majority of patients. For more information on the repurposing of sirolimus for ALPS, visit Cures Within Reach’s website here