As part of our commitment to improving translation and accelerating new treatments, we are launching this year’s Advancing Treatments Award (ATA).

The ATA exists to fund the kind of exciting research that backs promising treatment approaches ready to move from lab to clinic.

We’re looking for researchers working on the early-stage development of new treatments to slow, stop or reverse MND disease progression. We’ll also consider funding projects focussed on developing treatments to better manage symptoms, which could dramatically impact the lives of people living with the disease.

This Award, of up to £500k for a maximum of 5 years, aims to help researchers build data packages that will attract onward investment and strategic partnerships, to accelerate promising treatments through further testing and move them towards commercialisation. Projects should have the potential to be effective across a broad range of people living with MND.

Further details

We will accept applications for projects that cover two of the strategic priorities in our research strategy, Catalysing a Cure:

  • Validating therapeutic targets - this includes demonstrating the target’s relevance, differentiation and safety, with a clear outline of how target modulation results in pathway modification and rescue of disease phenotype. 
  • Accelerating new treatments - including the development of new pharmacological interventions and their efficacy in pre-clinical models. We will also support safety and pharmacokinetic studies.

We hosted an online webinar which covered the award round in more detail, and spoke to members of our Research Review Committee who provided advice on how to produce a strong application. You can watch the webinar below.

For more information about the scope of the award and how to apply, visit our Advancing Treatments Award page. Expressions of interest close on Tuesday 9 June at 12pm. Applicants are strongly encouraged to get in touch with the Foundation before submitting an application to discuss your ideas (please email research@myname5doddie.co.uk).

Previous Advancing Treatments Award projects

Dr Tatyana Shelkovnikova, University of Sheffield - 2024 Advancing Treatments Award

“Small molecule hit optimisation for lncRNA NEAT1/2 - a molecular target for neuroprotection in sporadic MND”

Background: NEAT1 is a ribonucleic acid (RNA) molecule that serves as the main building block for microscopic RNA-protein granules called “paraspeckles”. NEAT1-paraspeckles are induced by stress, helping cells to survive. Dr Shelkovnikova and her team previously found that spinal motor neurons of people with MND develop paraspeckles, and that several proteins which are affected by MND-associated mutations are known to regulate NEAT1-paraspeckles. This suggests that boosting NEAT1-paraspeckle production could potentially improve the survival of motor neurons in MND.

Methods: The team aim to generate compound libraries and identify preferred chemotypes capable of boosting NEAT1-paraspeckle production in MND. They will conduct hit-to-lead optimisation using biophysical and cellular assays and perform proof-of-concept studies in neuronal models of MND with additional in vitro and in vivo pharmacokinetic studies.

Expected outcomes: If successful, not only will the project demonstrate the potential of targeting NEAT-1/paraspeckles in the treatment of MND, but the team will also deliver selective small molecules ready for optimisation and identification of a suitable candidate ready for clinical translation.

Professor Pietro Fratta, University College London – 2023 Advancing Treatments Award

“Developing cell specific targeted therapeutics for ALS modifiers”

Background: Nuclear loss of TDP-43 induces a multitude of splicing changes, including the aberrant incorporation of cryptic exons into mature RNA. These events are exceptionally specific and along with others, Professor Fratta and his team have shown they occur only in people with MND and only in the subset of diseased cells. The team have developed a gene therapy approach which takes advantage of this specificity by only targeting degenerating cells.

Methods: The team will further develop their gene therapy approach through optimisation of guide RNAs and expression vectors. They will then generate neuronal cell models to test efficacy of the therapy by rescuing MND molecular and functional phenotypes, before testing in vivo.

Expected outcomes: If successful, the project will provide evidence of this gene therapy approach in vitro and in vivo, generating a data package that will lead to further funding to support pre-clinical toxicity studies and early clinical trials. The resulting treatment could be able to treat the majority of people living with MND.

This new technology gives us the ability to express therapies that we think will be beneficial to MND only where they are needed. This will improve safety, and improved safety means a greater likelihood of being able to test therapies in clinical trials, getting a successful result, and helping people affected by MND.

Professor Pietro Fratta

University College London

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