Our Five-Year Research Strategy
2019-24

We are proud that over the last 35 years we have raised and donated £5 million in support of research into ground-breaking findings and initiatives that have been key to the development of drug treatments and access to clinical trials (read more here).

Pharmaceutical companies are now investing heavily in the clinical development of drug treatments; these are important stepping stones that we will continue to monitor and support. Our research funding focus will now be on projects that contribute to:

  • Understanding of the complexities of SMA and the underlying disease mechanisms
  • The pre-clinical development of drug treatments   
  • Improvements in the clinical care and management of people affected by SMA
  • Further supporting, strengthening and building the capacity of the UK SMA scientific and clinical community

Our excellent track record of raising and delivering money into the scientific community, along with our strong connections and collaborations with clinicians, researchers, international groups and the pharmaceutical industry, enable us to ensure we are not duplicating effort when we fund and support initiatives that will ease the path to drug treatments and improvements in clinical care.

Where we can make a difference, we will.
 

Collaboration, accountability and decision making

We believe in collaboration in a networked world. We have close ties with other SMA organisations across the world and we use these to maximise knowledge, expertise and avoid duplication of effort. We are an original founder member of SMA Europe, maintaining active participation in the work of this pan-European consortium of patient and research organisations. Through this body, we work with Cure SMA and The SMA Foundation in the USA. We are members of TREAT-NMD, Genetic Alliance UK, the UK Neurological Association, the UK SMA REACH network and the Association of Medical Research Charities (AMRC).

Our history, our international alliances and requirements of AMRC membership ensure the most rigorous and collaborative vetting of all research funding requests. We ensure that the possibility of funding from Government Sources, the NHS, bodies such as the Wellcome Trust and pharmaceutical companies, have been thoroughly explored.

Funding decisions are managed by our Trust Board supported by its Research Advisory Group and our Research Coordinator.
 

Potential areas considered for funding

While the areas below are non-exhaustive, we believe it is useful to be transparent about the major areas we are most likely to consider for funding research.

  • Laboratory Research that addresses:
    • Understanding of the underlying mechanisms of SMA as a whole-body condition
    • Pre-clinical development of treatments to prevent or to slow the progression of SMA
  • Clinical Care and Management Research that addresses:
    • Improving understanding of the impact on an individual of SMA, with or without treatment, over the course of their life
    • Developing a greater understanding of the impact of treatment on an individual with SMA, compared to the expected outcome without treatment.
    • Delivery of the best standards of care for SMA
    • Development of ethically acceptable newborn screening programmes
  • Building UK SMA Scientific and Clinical Capacity:
    • Supporting young scientists and clinicians to take up a career in the field of SMA, in order to ensure that SMA research is sustainable 
    • Sponsoring collaborative initiatives, including: developing networks; delivering events such as conferences, meetings and workshops
       

How we will implement this strategy

Over the next five years (2019 – 2024), we had intended to support projects related to the above areas via the following mechanisms: 

  • A grant of up to £30,000 a year to support our SMA PhD Partnership with Muscular Dystrophy UK which will fund up to two 4-year SMA related PhD studentships.  
     
  • A pledge of £25,000 a year towards SMA Europe’s call which takes place every two years and funds research that is peer reviewed and selected by the SMA Europe Scientific Advisory Board.

During this strategy period, we did not plan to make further ‘one-off’ grants to support research.

It is with great regret that, due to the impact of COVID-19 on both MDUK and our funding, we are unable to progress this funding at this stage (May 2020) Read more here.

In 2019 / 20, we also made the grants listed below:

  • Treat-NMD UK SMA Patient Registry: £10,000. SMA Support UK has funded the registry since its inception in 2007. Given the changes taking place as to how the various registries will all work together, this will be reviewed at the end of February 2020.
     
  • Research Consortium Coordinator: £15,000. This is an addition to the SMA Trust’s funding of the consortium (January 2016: £1.3m over 3 years) to assist with ensuring sustainability of the developments to date.
     
  • Study led by Dr. Melissa Bowerman, Keele University: £14,000 to assess whether a commercially available drug targeting metabolism could be a potential treatment for SMA, when given in combination with a gene therapy. The project is already partly funded by MDUK and has been reviewed by Action Medical Research (AMR). This is the final part of the required funding.


 

Communication

Our Scientific Research Coordinator, Clinical Care Research Coordinator and Research Coordinator work with our Support Services Team to ensure that the SMA Community is informed as accurately, quickly and effectively as possible of:

  • developments in understanding, treating and managing SMA
  • progress with access to drug treatments in the UK
  • any opportunities to apply for funding for research initiatives
  • research funding decisions
  • outcomes of funded projects
     

Access to drug treatments

Staff will continue to work in their role as patient advocates to ensure this ultimate resaerch outcome becomes a reality for the SMA Community.
 

We spend all money raised wisely,
bringing hope for tomorrow.