- M102 is a candidate of disease-modifying drugs for motor neuron disease (MND) discovered by Sheffield scientists in 2013
- The University of Sheffield’s partnership with Aclipse Therapeutics provided a £ 1.6m grant from the Medical Research Council (MRC) to advance the development of M102 translation.
- The MRC grant follows funding from FightMND to bring M102 to clinical trials with patients
- MND – also known as Amyotrophic Late Sclerosis (ALS) – is a devastating disease that affects 5,000 people in the UK and 450,000 people worldwide
Researchers from the University of Sheffield Institute for Translational Neuroscience (SITraN) have received a £ 1.6 million grant from the Medical Examination Council (MRC). The grant will support their partnership with Aclipse Therapeutics to advance the development of M102 translation – a drug candidate for the treatment of motor neuron disease (MND).
Neuroprotective properties of M102 were discovered by SITraN researchers in 2013. Preclinical models showed potential for M102 to slow the progression of MND, which affects a patient’s ability to walk, talk, eat and breathe.
The drug candidate activates the NRF2 (associated erythroid nuclear factor 2) and HSF1 (heat shock factor 1) signaling pathways, which can work together to protect motor neurons from injury.
The Principal Investigator on the project, Dr. Richard Mead explains: “M102 has the potential to delay disease progression in familial and sporadic MND patients.”
“The MRC grant will allow us to develop patient stratum biomarkers that will be applied in M102 clinical trials, which may enable a personalized treatment approach in MND. This means we can identify those who do and do not respond to M102 so that we can focus on M102 so that we can focus on the treatment of those MND patients most likely to benefit.In addition, we will, in collaboration with Aclipse, all improvements required to reach the clinic. “
Project co-ordinator Dr Laura Ferraiuolo said: “This project uses a methodology developed by my lab team to allow us to identify gene signatures that discriminate between carriers. and non-responders of selected drugs.Our future goal is to be able to identify the best drug for each patient, making a major step forward in drug effectiveness and patient well-being . “
Project co-applicant Professor Dame Pamela Shaw, Director of SITraN and NIHR Sheffield Biomedical Research Center, said: “This funding award from the MRC is great news for MND patients in dire need effective treatment to address this life-threatening neurodegenerative disease. “
“Together with my SITraN colleagues, Dr Richard Mead and Dr Laura Ferraiuolo, we led the MND biology research that allowed the development of M102, including the discovery of a potentially detailed treatment modality. exists for M102 in MND, so we are extremely grateful that MRC funding will allow us to undertake this exciting therapeutic approach in human clinical trials. “
“We greatly appreciate the support from the MRC for our vision for a modern and comprehensive pathomechanism approach to the treatment of MND patients,” said Raymond K. Houck, Head of Aclipse Therapeutics.
He continued: “The MRC award, together with our recent FightMND award, is accelerating the development of M102 into its first human clinical studies and demonstrating the biology of M102 and its potential for precision treatment for the treatment of MND.
“The research funding from these programs will be crucial as they will support the completion of our new drug inspection (IND) -enabling work and the regulatory filters for first human studies. Importantly, M102 may have applications in a wide range of conditions associated with adverse effects on neuronal function such as Friedreich’s ataxia, Huntington’s disease and Parkinson’s disease. ”
MND – also known as Amyotrophic Late Sclerosis (ALS) – affects approximately 5,000 people in the UK and 25,000 people in the US; with the expectation that numbers will rise. MND is a disorder that affects the nerves – or motor neurons – in the brain and spine that connect the nervous system to muscles to allow the body to move. The messages from these nerves gradually stop reaching the muscles, leading to weakening, stiffen and eventually detoxification.
Currently, there is no cure for MND and no effective treatments to stop or reverse the progression of this devastating disease.
###
Contact the media: Amy Huxtable, Media Relations Officer, University of Sheffield, 0114 222 9859, [email protected]
Notes to editors
University of Sheffield
With nearly 29,000 of the brightest students from more than 140 countries, studying alongside more than 1,200 top scholars from around the globe, the University of Sheffield is one of the leading universities in the world.
Sheffield is a member of the UK’s renowned Group of leading research-led institutions, offering world-class teaching and research excellence across a wide range of subjects.
United by the power of discovery and understanding, university staff and students are committed to finding new ways to transform the world in which we live.
Sheffield is the only university to appear in the Sunday Times Top 100 Non-Profit Groups for Work for 2018 and for the past eight years it has been ranked in the top five universities in the UK for Student Satisfaction with Times Higher Education.
Sheffield has six Nobel Prize winners among alumni and students and its alumni progress to careers with great responsibility and influence around the world, making a significant contribution to the fields they chose.
Global research partners and clients include Boeing, Rolls-Royce, Unilever, AstraZeneca, Glaxo SmithKline, Siemens and Airbus, as well as many UK and overseas government agencies and charitable foundations.
Aclipse Therapeutics
Aclipse Therapeutics develops modern and differentiated drugs to treat orphan diseases with unmet medical needs. Our main drug candidate, M102, is being developed for the treatment of ALS with potential use in other neurodegenerative diseases such as Friedreich’s ataxia, Huntington’s disease and Parkinson’s disease. M102 targets multiple disease pathomechanisms and enables a precise treatment approach to identify patients who are more likely to benefit from the drug. Aclipse has a highly experienced orphan drug management team and a clinical advisory board of the world’s leading ALS physicians. For more information about Aclipse, visit the website at http: // www.
Disclaimer: AAAS and EurekAlert! they are not responsible for the accuracy of press releases posted to EurekAlert! by sending institutions or for using any information through the EurekAlert system.