‘Lost’ Drug Trial Highlights Potential of Repurposed, Repositioned, and Rescued Compounds

First Patient Treated with Lost Drug Bisantrene in Clinical Trial in Israel

With Australian specialty pharmaceutical company Race Oncology’s announcement last week that the first patient had been treated with the “lost” drug Bisantrene in a clinical trial for relapsed/refractory acute myeloid leukemia (AML) in Israel, other firms may be reevaluating the potential for similarly repurposed, repositioned, or rescued drugs to become breakthrough treatments against the odds.

“This is a major milestone for Race, because it’s the first treatment with Bisantrene since the drug disappeared more than 25 years ago,” said Race Oncology CEO Peter Molloy. “Treating the first patient is the culmination of our efforts over the last three years to resurrect this forgotten drug and see it used to save patients’ lives.”

According to the company’s announcement, Bisantrene was lost after a series of mergers in the early 1990s, despite its potential as a treatment for AML. The patient in Israel is the first of 12 to be recruited as part of the Bisantrene trial at Sheba Medical Center.

Race Oncology has submitted an Investigational New Drug application to the U.S. Food and Drug Administration for conducting a U.S. registration trial, and is pursuing investigator-initiated Phase II trials, such as the one in Israel, which is being led by hematologist Prof. Arnon Nagler.

Meanwhile, the broad issues involved in bringing shelved drugs back into development for their original, or even new, indications is the focus of the upcoming 8th Annual Drug Repositioning, Repurposing, and Rescue Conference this September in Washington, D.C. The event covers patient advocacy efforts, new partnerships, systematic repositions and data analytics, cutting-edge technologies, regulatory and legal guidance, and key therapeutic areas for repurposing.

The authors of a recent review article in Nature Reviews Drug Discovery noted that, “[g]iven the high attrition rates, substantial costs, and slow pace of new drug discovery and development, repurposing of ‘old’ drugs to treat both common and rare diseases is increasingly becoming an attractive proposition because it involves the use of de-risked compounds, with potentially lower overall development costs and shorter development timelines. …[H]owever, there are…major technological and regulatory challenges that need to be addressed.”

Other resources for those who are interested in this topic include:

“Drug repurposing can be seen as a response to the productivity issues in current drug development, as well as a strategy to reduce development times and as a source of low-cost treatments to meet the increased demands and unmet needs of cancer patients,” notes the ReDO Project’s main page. “We feel that repurposing may represent an untapped source of novel therapies.”

Editor: Gary W. Cramer