Annette Bakker, PhD, Chief Executive Officer of the Children’s Tumor Foundation, discusses repurposing shelved assets for rare diseases.
The Children’s Tumor Foundation is an organization dedicated to developing treatments for patients with neurofibromatosis (NF). NF is a group of rare genetic conditions that cause tumors to develop in the nervous system. The main types of NF are Type 1 (NF1) and NF2-related schwannomatosis (NF2-SWN). Symptoms can include skin changes, bone abnormalities, optic gliomas, and tumors on the nerve tissue or under the skin, acoustic neuromas, hearing loss, ringing in the ears, poor balance, brain and/or spinal tumor, and cataracts at a young age. Schwannomatosis causes schwannomas, pain, numbness, and weakness.
Repurposing Shelved Assets
Dr. Bakker recently spoke about the importance of repositioning drugs for rare diseases at the 2025 World Orphan Drug Congress (WODC). When small biotech companies begin development on a new drug and then are bought out by major pharmaceutical companies, these drugs may become shelved assets. This means the drugs to do get further developed and initiatives are stopped, typically for business and strategic reasons. Because these drugs still have efficacy and safety potential and usually, phase 1 data, they are a huge opportunity for the rare disease community.
The challenge lies in getting the rights to these treatments or convincing the company to start those clinical studies up again. For the latter, that can be particularly challenging since the original project team is often no longer at the company and the data may be difficult to organize. There is also the challenge of convincing upper management of the decision to repurpose.
Despite these challenges, two drugs have recently been approved through this process, selumetinib and mirdametinib. With the help of the Children’s Tumor Foundation, systems are being put in place to further progress in repurposed drugs. A preclinical hub has been set up to connect academia, industry, and the medical community. The hub strives to streamline access to treatment options by accelerating drug discovery, increasing testing, and making screening quick and efficient. More information on this preclinical hub can be found at https://www.ctf.org/for-pharma/
For more information on CTF, visit https://www.ctf.org/
To learn more about NF and other rare neurological conditions, visit https://checkrare.com/diseases/neurology-nervous-system-diseases/
