Guest Column | May 28, 2024

Reeve Foundation Advances SCI Trials & Treatments With Symposium, Industry Partnerships

A conversation with Marco Baptista, chief scientific officer, Christopher & Dana Reeve Foundation

Doctors board room meeting-GettyImages-1365555907

The good news is that drug development for therapies treating the central nervous system (CNS) is on the rise, as Reeve Foundation CSO Marco Baptista shares in this interview. The bad news is that of those, spinal cord injury (SCI) therapies are not well represented, he explains. However, with the Reeve Foundation’s support and the help of AI, advanced trial designs, the identification of genetic biomarkers, and most importantly, partnerships, the tide is turning.

In this interview, Baptista delves into the current landscape of SCI clinical trials, exploring the challenges that continue to impede progress and the strategies — at Reeve Foundation and beyond — being employed to overcome them.

What are some challenges for clinical trials studying a new therapy for spinal cord injuries?

Once known as the “graveyard of neurobiology,” advances in understanding the complex underlying biology of spinal cord injury (SCI) has evolved substantially — paving the way for a new era in SCI research. This more comprehensive understanding of the biology of injury combined with 21st century medical and technological gains, from AI to novel trial designs to genetic biomarkers, is invigorating the field.

To capitalize on current momentum, however, it is imperative that innovators, funders, and others combine our collective resources to build a more robust clinical pipeline. While the unmet needs of the SCI community continue to grow, exacerbated by the COVID-19 pandemic, scientific and economic barriers endure and stymie progress.

Over the last several years, we’ve seen the central nervous system (CNS) pipeline grow, accounting today for roughly 14% of the overall industry R&D pipeline, making it the second largest therapy area after oncology by number of assets in development. Yet SCI trials are so few that they don’t represent a significant share of this CNS R&D pie.

Traditionally, SCI innovation has been driven by academia and emerging start-ups — two sectors that face financial obstacles and expertise in bringing new therapies to market. Mid-size and larger companies, however, are beginning to focus more of their resources in CNS, including SCI. For example, both AbbVie and Mitsubishi are developing biologics targeting RGMa, a strong inhibitor of axon regeneration; an RGMa antibody may hold promise as a therapeutic agent for SCI. The incentive for progress among companies of all sizes is also becoming clearer: the global CNS market forecast projects an estimated $169 billion by 2027.

Success for SCI will require investment along the scientific continuum to address long-held challenges, from early-stage pharmacokinetic-pharmacodynamic (PKPD) analysis, an often-overlooked step that leads to failures down the line, to improved patient engagement in trial design and recruitment. Work remains to be done in supporting and listening to diverse patients so that their real-life needs are being addressed and they are able to remain active participants for the duration of a trial.  

Recently, there have been major breakthroughs in neuromodulation-based trials. How has the Reeve Foundation been involved in this research boom?

Significant medical breakthroughs via high-tech neuromodulation studies are beginning to open a promising door to products that can improve the quality of life for those impacted by SCI and paralysis.

Over the last four decades, the Reeve Foundation seeded the field with nearly $145 million invested broadly in SCI research. These investments critically boosted the work of early pioneers of neuromodulation, helping demonstrate that damaged spinal cords can adapt and be retrained with epidural stimulation. As the scope of research expanded, Reeve-fueled efforts have sought to restore long-sought functional gains, including hand dexterity, bladder function, and blood pressure regulation.

From our earliest investments, part of our goal in funding such studies has been to move high-risk, high-reward endeavors into a category of “de-risked” science. This remains a key priority for all of Reeve’s grantmaking, and within the concept of neurostimulation, we believe that restoring mobility via implants and other neurosurgical interventions holds great potential to make life-changing improvements for those living with SCI.

The Reeve Foundation recently announced funding for two new projects with .NeuroRestore, a research, innovation, and treatment center that develops and applies bioengineering strategies involving neurosurgical interventions to restore neurological function.

One of these studies is investigating the possibility of reversing upper limb paralysis, while the other will examine if the technology can be used to restore functionality and bladder control in individuals with incomplete spinal cord injury. For a person living with SCI/paralysis, these functions can make a world of difference in independence and quality of life.

Considering all the bottlenecks of SCI trials, which do you see as the easiest to address? What about the most critical?

One of the Reeve Foundation’s goals is to help facilitate solution-oriented conversation around the varied bottlenecks, especially among smaller biotech companies in the early stages of clinical trials. While bottlenecks are, by definition, difficult to resolve, there are key steps that we believe are critical to pressing ahead.

Breaking down silos, for instance, is mission-critical for the field. In 2023, we launched with Lineage Cell Therapeutics the first annual SCI Investor Symposium, designed to bring together diverse SCI stakeholders, including biotechnology companies and people with lived experience, to discuss research advances, challenges, and future trends. By assembling leaders from across the SCI landscape, we aim to increase innovation, decrease knowledge gaps, and troubleshoot problems before they lead to scientific failures. This June, we are convening numerous key players at the second annual SCI Investor Symposium, with highly anticipated conversations about the enablers of success and inclusion of patients and caregivers at every research stage.

What are some other ways in which the Reeve Foundation collaborates with companies conducting clinical trials?

SCI is a complex condition, and every injury is unique. Success requires shared knowledge and collaboration across the field. As an SCI convenor and arbiter of strong and effective research, we support grant-based trials in both the academic and for-profit sectors while also working to align and guide the field toward what we believe are the most promising scientific avenues. Thus, our work goes far beyond funding individual studies and includes an imperative to forge partnerships that connect and support the entire industry.

Recently, the Reeve and Kessler Foundations announced a pilot study to revolutionize bladder management for individuals with SCI. This collaboration is utilizing the Butterfly iQ™ — a portable ultrasound device that enables those living with SCI to monitor their bladder volume at home, potentially mitigating the risk of urinary tract infections (UTIs), which often result from improper bladder management. This study is an example of research funding that was driven by community input regarding unmet need, and that brings academia, industry, and nonprofit expertise together to problem-solve.

Another example is our work with the University of Alberta, a three-year venture funded by the Reeve Foundation to accelerate data sharing among researchers via the Open Data Commons for Spinal Cord Injury (ODC-SCI). The partnership supports data transparency and seeks to enable researchers to share findings in a protected space with a network of academics, scientists, and industry peers. The ODC-SCI can help address the ongoing challenges facing the field by aiding researchers in the responsible and necessary information-sharing process so that they can begin to analyze successes and failures together — and avoid duplicating the failures.

About The Expert:

Marco Baptista, Ph.D., leads the Reeve Foundation’s mission to advance cures for spinal cord injury and paralysis. He oversees the foundation’s research portfolio and collaborative approach to accelerating the field toward meaningful treatments.

Prior to joining the Reeve Foundation, Baptista served as vice president, research programs at the Michael J. Fox Foundation for Parkinson’s Research. In this role, he worked to develop an aggressive and innovative agenda for accelerating research and drug development for Parkinson’s disease. Prior to that, he served as an associate principal scientist at Merck, leading a preclinical Parkinson’s program focused on improving the understanding of genetic causes of Parkinson’s disease.

Baptista earned an undergraduate degree in psychology from the University of Toronto and a Ph.D. in neuroscience from McMaster University, Canada.