Neurology Central

A peek behind the paper – Melissa Bowerman on spinal muscular atrophy and the need for continued funding

Take a look behind the scenes of a recent Editorial published in Future Neurology entitled, ‘Funding for spinal muscular atrophy research must continue’, as we ask author Melissa Bowerman (Keele University, UK) about the main challenges surrounding spinal muscular atrophy (SMA) research and funding, including how these challenges might be overcome.

What inspired you to write this piece?

The current state of the SMA research field was the inspiration for this editorial. I have been involved in SMA research for 15 years now. I consider myself privileged to be part of this amazing community consisting of families, researchers and clinicians and to have seen the development and emergence of these new, exciting and life-changing SMN gene-based and enhancing therapies. But even in the early days, when the clinical trial results were being presented at meetings and conferences, it was clear that these SMN-dependent treatments were unfortunately not a ‘cure’. There was a very realistic possibility that additional treatments would have to be developed to support the SMN therapies and alleviate symptoms that patients could develop or maintain as they aged. However, this was not immediately clear to the wider community.

Indeed, since the US FDA approval of Spinraza™ (nusinersen) almost 3 years ago, charities have faced difficulties in raising funds to support SMA research and several funding agencies are sending a message (directly or indirectly) that SMA research is not a priority. I therefore felt the need to communicate why it was of utmost importance to continue investing in SMA research.

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At present, what would you say are the main challenges surrounding SMA research and funding?

In my opinion, the main challenges revolve around getting the public and scientific communities on board with the idea that fundamental and translational SMA research remain of utmost importance. Another challenge is reluctance, sometimes encountered within the SMA field, to support non-traditional “out-of-the-box” type of research endeavours. The new therapeutic era for SMA has to be accompanied by a seismic shift in our research questions so that we can support the SMN gene-based and enhancing therapies and importantly, patients throughout their lives.

How could these challenges be overcome?

By engaging with the public and greater scientific community to communicate clearly why continued investment in SMA research is important. Beyond this, we should pay close attention to the ongoing longitudinal studies of patients that have received SMN gene-based and enhancing therapies, which will shed light on the potential limitations of these treatments and any new or remaining therapeutic needs. We also have to discuss with patients and their families to get their direct inputs and perspectives on the knowledge and therapy gaps that they consider important. And finally, we need companies/industry to work together and with the SMA community (families, researchers and clinicians) to help reach our common goal of optimizing therapeutic development, benefits and delivery to patients.

What do you think the next steps are for SMA research?

I think that we need to embrace novelty and not be afraid to think above and beyond what has already been done. In terms of fundamental research, we still don’t fully understand the impact of SMA on all cells and tissues in the body and how the different SMN gene-based and enhancing therapies are affecting their function. In terms of translational research, we should exploit drug-repositioning strategies as much as possible, whereby existing and approved drugs currently in use for other diseases and conditions can be repurposed to improve specific SMA symptoms. If we make these our priorities, we will be well placed to serve the SMA community to the best of our abilities.

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