On 7th and 8th May, Ataxia UK’s Research Communications Officer, Scarlett Parr-Reid, attended Beacon for Rare Diseases and Remedi4All’s second International Drug Repurposing Conference (IDR25), held in Amsterdam.
Drug repurposing involves finding new therapeutic uses for drugs that are already approved for use in other conditions. One example of this which was showcased at IDR25 is the drug Nitisinone. It was originally developed for a condition called Tyrosinemia type 1, but the Alkaptonuria Society in the UK spearheaded research and funding for trials in Alkaptonuria, leading to its approval as the first licensed treatment for this rare disorder.
The event brought together over 300 researchers, clinicians, patient advocates, members of industry, policymakers and more united in the mission to find treatments for rare diseases for 2 inspiring days of talks, panels, workshops, posters and networking. Some of the key themes discussed included the importance of patient input into the design and outcomes of clinical trials, the need for patient organisations and industry representatives such as pharmaceutical companies to hold discussions with drug regulators as early and often as possible during drug development, and the value of harnessing tools such as AI to expedite the process of finding and even testing repurposed drugs for rare diseases.
This event comes at a time when drug repurposing is garnering more attention than ever before. There are over 7000 rare diseases with known molecular mechanisms. Only 500 of these have approved treatments. According to a recent review paper, fewer than a quarter of rare conditions have had clinical trials.
The SIMPATHIC Consortium and its 22 international partners led by the Dutch Radboud University Medical Center and Amsterdam University Medical Centers showcased their work at the conference. They have developed a new approach to expedite the repurposing of existing drugs for groups of patients with rare neurological disorders. The consortium has been awarded an €8.8 million grant from the Horizon Europe program to further develop this innovative method. The researchers are using a new technology to test the efficacy of existing drugs in patients with neurological disorders, requiring only a tube of blood or a small piece of skin from the patient. These materials contain stem cells that the researchers culture into nerve cells. They subsequently test how they respond to a variety of existing drugs. Euro-ataxia one of the patient groups representing people with neurological conditions on this project, and Spinocerebellar ataxia type-3 (SCA3) is one of the neurological conditions which will be studied.
Dr Tessa Braam (pictured to the right), a clinician and researcher working on the SIMPATHIC project who presented a poster titled ‘The SIMPATHIC Basket Trial: Targeting Shared Pathomechanisms’ which was runner-up in the IDR25 poster competition, had this to say,
“As someone who is simultaneously a patient, physician, and researcher from Amsterdam UMC and Radboudumc, I found it profoundly valuable and meaningful to see the multifaceted dimensions of drug repurposing so thoughtfully highlighted and seamlessly integrated throughout the conference. Therefore, I was grateful for the opportunity to draw attention to the SIMPATHIC project through my poster presentation. As outlined in our recently published journal paper which you can read , we are developing a basket trial to evaluate a drug repurposing candidate — sildenafil citrate — across multiple diseases simultaneously. One of the possible diseases is SCA3. We are currently working intensively to translate preclinical data into concrete inclusion criteria, outcome measures, and a corresponding trial design. To stay informed about our progress, please keep an eye on the website for future updates.”
Read more about the work of the SIMPATHIC Consortium here.
IDR2025 highlighted a number of barriers to accessing repurposed drugs for rare diseases and how these can be overcome, including a lack of information on repurposed drugs reaching GPs, limited connection to secondary care specialists for GPs to ask specific questions about rare conditions such as ataxias, and a lack of access by patients to repurposed drugs currently being trialled, which can vary widely by postcode.
Scarlett had this to say about the event,
“The passion and drive from those working in the rare diseases space to bring patient voices into the drug repurposing and clinical trial landscape at every stage was palpable. This event showcased the incredible hard work that is going on to bring treatments to those with rare diseases sooner by harnessing tools such as AI drug screening platforms, sharing clinical trial data for rare diseases across research groups to de-duplicate efforts, and multi-national collaboration that bridges the gap between researchers, regulators and pharmaceutical industries.
There has been a step-change in drug repurposing in the last few years, and this event has highlighted that. We have work to do, but by coming together and sharing knowledge and tools, we will get there a lot sooner than we might have done with fragmented research efforts.”
One of the conference speakers, Prof Stefan Gold, Head of the Neuropsychiatry Research Group at Charité – Universitätmedizin (University Hospital) Berlin, outlined powerfully the problem of the traditional one-drug one-condition approach to drug development and the need for drug repurposing,
“Developing one drug for one condition at a time is like building a new stadium for each football game, rather than [with drug repurposing] building a stadium to play multiple football games, or even to host concerts and other events.”
One example of a drug repurposing research project Ataxia UK has recently funded is a project looking at repurposing the anti-depressant drug Vortioxetine for the treatment of Spinocerebellar ataxia type-3 (SCA3) which you can read about about here.
Read more about Remedi4All here.
Read more about Beacon for Rare Diseases here.
