The NIH (National Institute of Health – North America) recently invited SCI -related organisations / individuals to express their opinion about Spinal Cord Injury research priorities (send your own input here . Feel free to copy-paste our input below if you like it). On July 29th, we sent the following feedback on behalf of the endParalysis foundation’s board & scientific committee, including Prof. Jerry Silver, Prof. Elly Hol, Dr. Janneke Stolwijk, Prof. Joost Verhaagen, Dr. Mark Bacon, Christal Powell, Corinne Jeanmaire and Jaap Pipping.
We thank the NIH for the opportunity to comment on Spinal Cord Research priorities. The SCI community is suffering. The SCI community is dying, as mentioned by one of the participants in the NIH’s February panel. We would like to suggest a focused, target-driven and disruptive approach increasing the chances of improving quality of life now as well as the potential for real recovery in the future.
We recommend to:
1. SET-UP 1 MEDIUM/ LT GOAL & 1 SHORT-TERM GOAL + ALIGN WITH THE SCI-COMMUNITY RECOVERY PRIORITIES
1.1. Assign TOP priority: “Accelerate research & development of therapies for repair, plasticity and improved functional recovery from post-acute to chronic SCI” (Theme B)
- A recovery breakthrough will make all other themes irrelevant. This very ambitious goal is the only one offering potential for a complete, global and long-term solution as well as a huge return on investment
- This concerns the biggest number of people, i.e. up to 3 million people living with SCI worldwide, not to mention the spin-off benefits for other neurological conditions
- Chronic SCI represents the only potentially attractive market for the Biotech /Pharmaceutical Industry, as well as a multi-billion USD Healthcare/Welfare public saving opportunity
- Research on Chronic Injury is more likely to lead to conclusive clinical trials and thus to therapies coming to market. As shown in a 2012 study, both high spontaneous recovery rate and complexity of enrolling patients just hours after injury makes it scientifically very difficult (and quite questionable ethically!) to test a therapy at acute timing.
1.2. Assign Second/Short Term priority to “Improve evidence for, and implementation of, functional recovery with neuromodulation and use-dependent plasticity” (Theme C).
As a 2nd priority, we suggest streamlining the testing/implementation of neuro modulation and comparable treatments, as:
- These treatments are already technically available and safeness proven.
- While not providing any actual regeneration, they might offer a short-term potential to improve many patients’ quality of life if properly focused on specific areas like hand function.
- There is a need to produce factual evidence beyond the few available anecdotal clues, of whether these techniques can guarantee/contribute to a real bladder, bowel and sexual recovery as alluded by some researchers or companies.
1.3. Align funding & strategies with the known SCI community’s priorities
This means privileging the research projects that can contribute to an actual motor/functional & sensory recovery in the following priority areas:
- Recovery of bladder, bowel and sexual function
- Hand and arm functions
- Therapies realistically marketable/applicable to people living with CHRONIC SCI, also taking into account the pain factor, another priority of SCI community
2. OPTIMIZE THEME B (REPAIR, PLASTICITY, FUNCTIONAL RECOVERY AFTER CHRONIC SCI).
Besides improving quality of life, we need to use the smartest minds and disruptive approaches to fight paralysis, and win. We suggest:
2.1. To greatly increase the funding allocated to chronic research studies through goal-driven funding criteria.
To date, the NIH only funds a low number of chronic studies, simply because most grant requests have an acute focus. As a result, countless studies don’t go through to the clinic. We believe that all funding bodies – including the NIH – can & should change that by using funding criteria such as clinical relevance & chronic focus.
2.2. To create a disruptive environment alongside the NIH’s traditional grant system to enable breakthroughs.
We suggest a moonshot approach/incentive prize/3.0 project towards:
- A goal-driven & integrative approach, from bench to market (multi-functional teams that include researchers, clinicians, patients, and market representatives) to ensure the clinical & market relevance of research projects
- Increased data sharing (positive & negative results), in a virtual network/platform
- Special funding designated for reproduction studies of major findings
- A fast-track testing of combination therapies, guaranteeing both speed & safety;
- “Blue sky” approaches to explore new breakthrough strategies.
- The establishment of a flexible roadmap targeting, e.g.:
(a) A few partial recovery goals, in line with the community priorities
(b) Key accelerators such as an improved & standardized chronic animal model, better imaging, organoids, glial scar neutralization, motor-neurons replacement;
3. OPTIMIZE THEME C (NEUROMODULATION & ALIKE)
- A large-scale, coordinated testing/implementation of those treatments, using a short-track process for a quick and large scale execution, yet enabling a scientific analysis of outcomes
- Focus on the community’s priority recovery targets
- Test the therapies without extended physiotherapy to guarantee clear outcome measures and applicability in real life.