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# FDAers Promote Using Cancer Drugs for Rare Diseases
- URL: https://www.fdaweb.com/fdaers-promote-using-cancer-drugs-for-rare-diseases/
- Published: 2016-07-28T12:00:00.000Z
- Updated: 2026-09-14T21:19:00.000Z
- Author: David McFarland
- Tags: Drugs, #legacy-id-D5136334

Four FDA National Center for Toxicological Research staffers say that it is likely that some cancer drugs could be repositioned to treat rare diseases. In an opinion [article](http://www.cell.com/trends/pharmacological-sciences/fulltext/S0165-6147%2816%2930077-3?ref=fdaweb.com) in the journal *Trends in Pharmacological Sciences*, the four look at the possible association between cancers and rare diseases across clinical observation, crosstalk between germline mutation and somatic mutation, and shared biological pathways, and conclude that by using systematic drug-repositioning approaches and taking safety issues into consideration, oncology drugs have great potential for reuse in treating rare diseases.

“It is encouraging that some efforts have been made to explore the reuse of oncology drugs in rare disease therapy,” the authors say. “However, more systematic and sophisticated strategies will be necessary to accelerate the process…. Genomic-based drug repositioning has particular potential because both rare diseases and cancers have a prominent genetic component.”

The authors say that in the near future, novel genomics techniques are likely to provide more extensive data on the specific genetic variations that underlie both cancers and rare diseases, making the association between cancers and rare diseases more precise. “Additional data and in-depth interpretation is a must to further reaffirm and verify the proposed hypothesis here,” the article concludes. “Nevertheless, the unmet need for orphan drugs is now not only a medical challenge but also a social issue. We anticipate that our paper could provide out-of-the-box thinking in this field and stimulate the community to investigate the potential of oncology drugs in the development of novel therapeutics for rare diseases.”

Questions still remaining, the FDAers say, include: 

- the role genetic mutations in rare diseases play in the development or evolution of cancer;
- whether rare disease treatment with oncology drugs identified in screening will translate into clinical efficacy;
- how the severe side effects of oncology drugs can be reduced or eliminated when the drugs are repurposed to treat rare diseases; and
- how regulatory agencies can balance the trade-off between efficacy and toxicity issues when reviewing potential submissions concerning repurposing of oncology drugs for rare disease therapy.