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Getting involved with drug repurposing

Patient groups are in an ideal position to drive drug repurposing projects for several reasons:

  • Patient groups can often independently decide what to fund and can choose to support research not typically funded by industry or the government. This can be particularly advantageous for rare diseases, which are unlikely to receive the same level of investment as common conditions.
  • As patient groups often have more established networks and closer relationships with patients than clinicians, academics or pharmaceutical industry representatives, they are often better equipped to identify potential clinical trial recruitment opportunities.
  • Many patient groups have close relationships with researchers and clinicians, allowing them to drive repurposing projects where government or industry have shown little interest.
  • Patient groups can unite for research funding, and often have crowdsourcing/crowdfunding tools at their disposal.

However, it is worth noting that drug repurposing projects require thorough planning, collaboration and careful consideration of potential roadblocks. This section describes the steps required to organise a drug repurposing project (summarised in the figure below) and details the important considerations at each stage of the project.


How to get involved

When considering getting involved in drug repurposing research, there are three main questions a patient group should ask:

1.     What is needed from a new repurposed treatment for your condition? This might include the benefits your patient population would like to receive from a treatment, the symptoms it should target and the side effects/risk that patients are willing to tolerate. You should also consider whether the perception of the condition and obstacles faced vary in different patient cohorts.

2.     What is your current position, and what avenues does this open up for research? Consider how many patients your organisation is in direct contact with, as well as what kind of contact you have with researchers and clinicians in the field. It is also worth considering whether a patient registry has been set up for the condition in question and whether there is accessible data about your disease online. Think about what financing routes are currently available to your organisation.

3.     What is the current status of treatment development? Assess the existing treatment options for the condition, any opportunities for treatment development, and whether a current therapy can be improved. In particular, patient groups should determine whether there are already existing repurposing candidates or pharmaceutical companies that are interested in your condition.

Answering these questions may help as you look to determine how you can get involved in repurposing research. This can be through a number of avenues, from fundraising to support projects, to collaborating with repurposing research partners.

Figure showing the steps to organising a drug repurposing project in outlined boxes spaced evenly along a large arrow. From left to right the boxes read ‘Getting started’, ‘Selecting a drug repurposing candidate’ and ‘Testing in clinical trials’. At the bottom of each box is an icon representing the stage of the drug repurposing project. These are a head with a brain inside for ‘Getting started’, a magnifying glass over three pills for ‘Selecting a drug repurposing candidate’ and a doctor with a patient sat on an examination table for ‘Clinical trial testing.

Choosing a repurposing partner

If you are looking to collaborate with bioinformatics, biotechnology or pharmaceutical companies on a drug repurposing project, it is important to choose a partner that complements the expertise your patient group has. Consider whether you need a partner with drug repurposing expertise, access to innovative technologies, expert disease knowledge or other resources for conducting research. You may also need a partner with an expansive network, that knows the location of study centres and has contacts within the field. Finally, your research partner’s ethical and cultural beliefs should align with your own to ensure successful collaboration.

Case Study: Partnering with Healx to repurpose drugs for rare diseases

Ian Roberts (Chief Technology Officer) and Karine Proulx (Drug Discovery Alliance Manager) from Healx, a bioinformatics company focused on drug discovery, provide an insight into how Healx’s artificial intelligence (AI) technology can be used to accelerate the generation of treatments for rare diseases. With the recent launch of the Rare Treatment Accelerator programme, designed to speed up the in silico to clinical trial phase for the development of repurposed treatments, Karine describes how patient groups can apply to collaborate with Healx on repurposing projects. Details on the application process can be found on Healx’s website here.