“This white paper represents the work of a cross-sector group of MND experts in academia and industry that have combined to agree a consensus on the key reasons for poor translational outcomes in MND. We have generated recommendations for the optimisation and conduct of pre-clinical studies to improve confidence that a drug will show efficacy in human trials. We hope this guidance will catalyse high quality pre-clinical drug discovery efforts and create a shared vision for all stakeholders involved in MND drug discovery research.”
The Guiding Principles underpin a new £5m funding call from LifeArc, also announced today, which aims to identify medicines used in other diseases and repurpose them for MND.
Paul Wright, MND Translational Challenge Leader at LifeArc, who was a member of the working group, said:
“It’s critical that we embed best practice in all aspects of our research programmes, which is why we’re excited to align our new £5 million drug repurposing funding call to the Guiding Principles for ALS Drug Discovery and Development. By partnering with the MND community to fund the most robust science, we will increase the chance of new treatments reaching people living with MND, faster.”
Jessica Lee, now Director of Research at My Name’5 Doddie Foundation, led the development of this guidance in her previous role at Medicines Discovery Catapult. She said:
“This project was a huge collaborative effort that brought together leading minds over a year-long period. We are so grateful for the time and energy of all members of the expert working group and everyone that attended the online workshop and completed the Delphi survey. We hope that this new guidance will raise the bar for pre-clinical MND research and help to accelerate the development of effective new treatments.”
Dr Nicola Heron, Chief Strategy Officer at Medicines Discovery Catapult, said:
“MND is a devastating disease with limited treatment options, so it is essential the drug discovery community works together to help change this. These Guiding Principles, released today, are the result of a collaborative effort to help innovators find new treatments and improve patient outcomes.”
Dr Brian Dickie MBE, Director of Research at the MND Association, said:
“As with many other neurodegenerative diseases, the history of MND clinical trials is filled with negative or, at best, modestly positive outcomes. With many potential drug targets emerging from the explosion of new knowledge about MND in recent years, the Guiding Principles will improve the translation of new therapies from the lab, help the drug development community to ‘pick winners’ for clinical trials and hopefully result in more effective therapies for this devastating disease.”
