Скачать или смотреть Repurposing Shelved Assets for Rare Diseases

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.

Dr. Bakker recently spoke about the importance of repositioning drugs for rare disease 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 and out of the companies they are being held in. Further challenges arise from the project team being reassigned or no longer at the company, unorganized data, and convincing upper management of the decision to repurpose.

Despite these challenges, two drugs have recently been approved through this process. With the help of the CTF, systems are being put in place to further progress in repurposed drugs. A preclinical hub has been set up to quickly derisk assets and platform trials allow companies to place their drugs into testing. The foundation currently has about 60 ongoing clinical trials.

Chapters:
Introduction 00:00
The Children's Tumor Foundation 00:30
Repositioning Drugs for Rare Diseases 1:01
Challenges With Shelved Assets 2:33
Success Stories 3:42
Finding Clients and Funding 5:37