Thread by @eperlste

Here are the slides from my #ReeveSummit2020 keynote called “The Future of SCI (Spinal Cord Injury) Research”

We often begin our presentations with a remembrance of Christopher Reeve, and this quote of his in particular has reverberated through the decades.

In fact, this year marks the 25th anniversary of Christopher’s injury. I was a sophomore in high school at the time.

Over the last 40 years, SCI foundations have funded many hundreds of projects without a globally coordinated research agenda that spans bench to bedside and the care-to-cure continuum.

The @ReeveFoundation has spent $138M over the last 40 years on research, spanning neuromodulation, neuroprotection, axonal regeneration, the secondary consequences of the injury (dysautonomia) and rehabilitation.

We imagine this distribution looks similar for other SCI funders.

Despite the enormous combined outlays for research by government funders like @NIH and nonprofits like @ReeveFoundation, @NeilsenFndn, @WFLWorldRun, @BuonicontiFund, @TRFoundation, @SpinalResearch, @SpinalCure, among others, there are 0 (zero) approved SCI therapies.

Although @NIH spending on SCI is ticking upward, there remains a translational valley of death that someone has to fund.

SBIRs/STTRs are supposed to help bridge that gap but these funding mechanisms are not exclusively SCI-focused.

How have other nonprofits done it? The marquee example is @CF_Foundation, which marshaled venture philanthropy in partnership with @VertexPharma. The result is a triple-drug combo approved last fall that treats up to 90% (but not all) CF patients and a $3B return on investment.

The global SCI community is at a crossroads. Ideas that seemed like science fiction 10-15 years ago are now hurtling toward preclinical validation. There’s a growing awareness that funding consortia of academic researchers one lab and one project at a time won’t cut it.

How do we proceed?

First we define the ambition as restoring people to who they were the moment before their injury. Next we form a global alliance of SCI stakeholders. Then we collaborate to develop a technical roadmap that garners global buy-in as SCI respects no borders.

Multiple barriers have historically impeded progress in advancing the SCI research agenda: lack of sustained funding; lack of robust patient registries; lack of standardized tools, platform and models; lack of knowledgeable sharing; lack of a technical roadmap, above all.

Currently, no individual stakeholder is playing the “orchestrator” role in the SCI R&D ecosystem.

We believe that @ReeveFoundation, whose brand and reach are already global, can play a unifying leadership role here.

We propose four primary building blocks to enable us to deliver on a more ambitious R&D agenda:

1) Right talent
2) Sustained funding
3) Strong governance and agile operating model
4) Alliance of partners

Leading R&D focused foundations typically follow a three-stage path from genesis to maturity.

One or more highly motivated families coalesce around a cure vision. They build research-evaluating capacities and organize a researcher and clinician network. Big donors drive impact.

Cure-focused foundations spend their early years funding grants and soliciting proposals. Research funding gradually comes with appropriate strings attached like contractual milestones. Academic-biotech partnerships emerge, culminating in mature venture philanthropy.

Key accelerators have enabled foundations to evolve more rapidly and deliver patient impact quickly. Exemplars include @CF_Foundation, @theMMRF, @CHDIfoundation, @LLSusa, @JainFoundation, @JDRF, @CureDuchenne, @MelanomaReAlli & @TheDDFund

Numerous organizations have developed roadmaps to identify and systematically address barriers that were impeding the development of new interventions and therapies.

Two examples of roadmap making exercises are Accelerating Medical Partnerships (nih.gov/research-train…) and @JDRF (jdrf.org/wp-content/upl…)

Case study 1: @NIH’s Advancing Medical Partnerships

Case study 2: @JDRF’s clinical translation roadmap has led to more efficient allocation of scarce resources across the Type 1 Diabetes landscape and more effective collaboration with sister group TD1 Exchange (t1dexchange.org)

Selection of experts for inclusion in the expert pane is critical to the success of roadmap development that balances improving lives and curing lives, to use @JDRF’s terminology.

The SCI roadmap collaboration will bring leading (and egoless) scientific voices into agreement on the current state of SCI research. Together we will publish a roadmap as a public resource for the global SCI community and harmonize activities toward our common goal of a cure.

Our collaborative SCI roadmap will assess the research landscape, highlight unmet needs of chronically injured patients, identify promising translational opportunities regardless of treatment modality, and establish a rational (and one day predictive) framework for combinations.

6 best practices to develop a robust roadmap:

1) Define how community will collaborate
2) Assemble a community of experts
3) Draft, workshop and revise roadmap
4) Define mission and ambition
5) Define metrics for success
6) Adapt roadmap for a broader range of audiences

My closing argument is that we’re stronger together. SCI doesn’t respect territorial boundaries. A bold international alliance is needed.

This is not an attempt to reproduce other efforts at collaboration — all of which have failed to improve the quality of lives.

The plan follows the alliance. And the funding (venture philanthropy) follows the plan.

Here’s a preview from our friends at @JDRF of what could happen for the SCI community after the completion of a roadmap t1dfund.org

Feedback encouraged!