A Q&A with Marco Baptista, Ph.D., chief science officer, Christopher & Dana Reeve Foundation
Silos, as many have come to learn, are not relegated to farming alone. In the clinical research field, as is the same across many industries, knowledge and data have become siloed — separated and partitioned off — both within organizations and across industry and academia. Whether it’s a result of protective measures or a byproduct of ineffectual communication and collaboration efforts, siloing has played a significant role in inhibiting innovation.
Yet, lately, there has been increased conversation around transparency and collaboration for the benefit of scientific innovation. And that’s quite apparent at the Christopher & Dana Reeve Foundation.
In this Q&A, Chief Science Officer Marco Baptista, Ph.D., discusses the foundation’s focus on and approach to data and knowledge sharing, as well as patient engagement, as it works to advocate for and fund the development of spinal cord injury (SCI) therapeutics.
One of the tenets of the foundation’s approach to clinical research is the pursuit of “shared knowledge.” Since your arrival, what actions has the foundation taken and what partnerships has it pursued to that end?
Baptista: Successful collaborations depend not only on shared resources but shared knowledge. The Reeve Foundation is increasing transparency throughout the field by leveraging the Open Data Commons, a platform co-developed by our partners at the University of Alberta (working with the University of California San Francisco) for academics, scientists, and industry peers designed to facilitate broad data knowledge. In doing so, we seek to catalyze more efficient use of scientific data — basic and translational — and increase the possibility of fresh insights. Further, we aim to de-prioritize competition between researchers to help provide a solution for the greater common good by sharing development ideas and early data related to the fundamental challenges that often slow everyone down. This requires a sea change in the field, where scientists and others prioritize progress over individual and organizational interests.
Increasing transparency systematically – around lab gains and failures – helps remove long-entrenched barriers to discovery, eliminates time wasted duplicating unsuccessful hypotheses, and allows researchers to refine or redirect their focus as more data becomes publicly available. Most significantly, data sharing supports more expansive success throughout the field, accelerating drug discovery and device development as more pieces of the puzzle become visible to everyone.
You work closely with the North American Clinical Trials Network (NACTN), and one of the foundation’s goals in doing so is to embrace data sharing. Why is data sharing important to advance discoveries in SCI, and how does NACTN support that?
In 2006, the Reeve Foundation launched the NACTN, a consortium of neurosurgery departments at hospitals across the country that provides a crucial launchpad for coordination and collaboration, to accelerate drug discovery and device development.
Our long history with NACTN has focused on expanding the research infrastructure to facilitate more trials focused on a wide range of investigational devices and therapeutic targets and exploring both acute and chronic phases of the injury. The complexity of SCI requires a diverse scientific pipeline, so these have included, for example, bioelectrical stimulation, cell replacement, axon repair and regeneration, and various investigational compounds. Today, as NACTN prepares to publish its findings, we are looking toward a future where the data helps enable the entire field.
Your approach to education and collaboration involves all players — including academic institutions, Big Pharma, and patients and caretakers of those with SCI. What role does each play in supporting your research?
Each player in the scientific ecosystem plays a crucial role in moving the needle to achieve our ultimate mission – finding cures for paralysis. Academic medical centers are able to conduct important lab science; from small biotechs to Big Pharma, industry players not only bring significant financial resources to the table but have the business acumen to bring new therapies to market; and those living with or caring for someone living with SCI bring crucial experience to the process. Their voices need to be heard at every stage of the scientific continuum, from R&D to market rollout, so that future drugs, devices, and surgical interventions take into account the needs, desires, and limitations of the community that can benefit.
By bringing everyone under one roof, the Reeve Foundation seeks to help generate new partnerships to increase innovation. Our role is one of convener, spurring collaboration to break down the silos that limit and slow progress.
In nearly every endeavor, there’s always room for improvement. In what ways would you like pharma companies to become more involved in your research?
The Reeve Foundation believes that partnerships with pharmaceutical and medical device companies are a critical component of speeding progress toward cures. Scientific advances that lead to widely available therapies represent a long road – much longer than is acceptable to those living with spinal cord injuries.
By collaborating closely with industry, we are playing a more active role in accelerating tomorrow’s innovations. Success in these partnerships lies in creating collaborative frameworks designed to overcome traditional hurdles and improve the pace of progress: knowledge and data sharing that leads to better-designed trials and increasing the volume and diversity of clinical trials so that more studies are in the pipeline. Industry also can benefit from our deep knowledge and personal connections with our community. Together we can ensure that those with lived experience inform the creation of products and medical procedures that will have the biggest impact on the lives of those we serve.
Looking internally, and because part of the foundation’s mission is to be transparent, tell me about a time when something didn’t go according to plan, in the lab or elsewhere. How was that remedied?
One of the roles Reeve plays in the arena is actively monitoring current science and helping translate and advance the most promising research – so when a pivotal trial doesn’t meet its primary outcome, that “miss” is felt deeply by our foundation and all those impacted by spinal cord injury. The reality is that the failure rate for clinical trials, especially those testing therapeutics in the central nervous system, is high. There are many reasons for trials not meeting their primary endpoints, and it is important that learnings from these clinical trials are shared with the community so that future trials may be successful.
Two recent trials that Reeve was following closely and, along with the wider paralysis community, shared high hopes for serve as examples of studies that did not meet their primary endpoints: the RESET trial testing AXER-204, a “Nogo receptor decoy” (a class of potential treatments for spinal cord injury that block or neutralize growth inhibitors that are highly expressed after injury and that play a role in restricting neuronal growth and neuroplasticity), from ReNetX Bio, and INSPIRE 2.0, which tested a Neuro-Spinal Scaffold from InVivo Therapeutics. There is much to learn from these highly anticipated trials, and the Reeve Foundation looks forward to the scientific community understanding and applying those lessons to future studies. As such, we’re continuing to monitor the follow-up of these trials and hope to provide the companies with opportunities for the information to be disseminated when ready.
You’ve said before that there are about 100 SCI trials and that 9% of trials involving central nervous system (CNS) research achieve regulatory approval. What is the foundation doing to help increase the odds of successful clinical trials in SCI?
That stat is based on those clinical trials on our radar, and my estimate was looking at all the trials underway recently but focusing strictly on drug therapeutics and devices and trying to deduce unique approaches – so, looking at novel trials. It further builds on a paper (Dietz et al. 2022) that analyzed clinicaltrials.gov and found that of the current approximately 1,150 trials focused on improving outcomes, many were redundant and had low enrollment and that more than 75% with “completed” status do not have results posted. Moreover, the phase of some trials is incorrectly classified as “not applicable” despite testing a drug or biological compound.
Our ultimate vision is to enable cures for spinal cord injury and improve quality of life for individuals and families impacted by paralysis. Our strategic imperative toward that end is to create a robust clinical pipeline whereby more and better clinical trials increase the chances of success. We define robust as greater numbers of clinical trials; greater diversity of trials investigating devices, therapeutics, and drug targets; and smarter trials that are designed to be informative even if they fail.
In addition, the foundation is also floating the idea of an “independent, collaborative fund that will be uniquely positioned to spark innovation and high-level investment.” What’s the status of the project, and what problem will it aim to solve?
The fund is an exciting development at the Reeve Foundation and is also related to our strategic approach toward the creation of a stronger clinical pipeline. One of our goals to that end is to increase investment opportunities for grant-awarding institutions as well as venture philanthropy funds to activate an investment market to cure paralysis.
The creation of this independent, collaborative fund is already underway, building on models that have already been successfully adopted by therapeutic categories as diverse as cystic fibrosis, type 1 diabetes, and Duchenne muscular dystrophy — resulting in new treatment options for patients and billions of dollars redeployed to the respective organizations’ missions.
Our fund will capitalize on recent scientific and technological breakthroughs to spark innovation and high-level investment in scalable, evidence-based, cure-oriented therapies for SCI. Once established, 100% of every dollar will be invested in cure-focused opportunities, with all returns reinvested back into the fund to support the next generation of therapies.
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.