Sep 19, 2024
Is 'Five Years' Finally Here?
Sam Maddox
U2FP’s Annual Symposium is upon us - a little more than a week away - happening September 27-28 in Atlanta, Georgia (register here). This is the third and final installment in my series attempting to contextualize and summarize the scientific presentations at this year’s gathering.
Session four may propose an answer to the old SCI research trope, give us five years and we’ll have something for you. Everybody with SCI hears this, for the last 40 years. Well, is five years finally here?
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Dave Marver, CEO of device startup ONWARD Medical and James Guest, MD, PhD, a prominent SCI researcher/clinician from the Miami Project, will present study details from a recent clinical trial for skin surface (transcutaneous) spinal cord stimulation, sponsored by ONWARD Medical.
Measuring the effect of tSCS on upper limb mobility in quads, the device improved function and sensation in almost three in four participants. The discussion will include ONWARD’s efforts to gain regulatory approval for the ARC-EX transcutaneous device, ongoing trials for an implanted device (ARC-IM, epidural stimulation) and other strategies in the pipeline, including a combination of an implanted brain signal sensor with epidural stim (ARC-BCI).
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The day will end with a conversation between artists Freaque and Reveca Torres, both living with SCI. They will describe their creative endeavors and how their unique point of view informs their work. A selection of their paintings and drawings have been incorporated into this year’s program.
The Future in Combination
Session five will kick off the second day with a discussion of combination therapies, likely necessary to offer the greatest potential for recovery, especially in chronic SCI.
Candace Floyd, PhD, directs the Neurotrauma Translational Research Center at Emory University. (She also helped organize this symposium, representing title sponsor Emory. Thank you Candace!)
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Here she will report on a study combining eSCS and a potentially regenerative drug in a large animal model. The stim units are the same as those used in humans; the drug is NervGen’s NVG-291 (the company’s Chief Medical Officer to talk later today) which is being studied clinically now; it may promote axonal regeneration, neuroplasticity and remyelination to shore up disrupted spinal cord nerves.
The use of a pig model makes the study very translational – pigs are physiologically similar to humans and therefore results can be clinically relevant. Floyd’s study also includes the input of persons with lived-experience in SCI regarding research design and priorities. This combinatorial work is the brainchild of U2FP and is funded by a coalition of SCI organizations: the Bryon Riesch Paralysis Foundation, Conquer Paralysis Now, Get Up Stand Up 2 Cure Paralysis Foundation, Morton Cure Paralysis Fund, and United Paralysis Foundation.
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Mark Anderson, PhD, is a scientist at the Swiss Federal Institute of Technology. He will discuss fascinating recent work to reengineer the spinal cord combining biologic, genetic and chemical engineering to regenerate growth after SCI. Anderson and his colleagues in the Gregoire Courtine lab take a step-wise approach to regeneration and recovery of function.
In a recent study they showed substantial recovery of walking function in 27 of 30 animals, using a complicated series of steps to mimic developmental neurobiology (to turn on and guide the growth of walking-specific spinal cord nerves to connect appropriately below the injury lesion site – just the way we formed the nervous system when we were infants). It’s complex and far from translation, but resets the bar for SCI regeneration. Listen to this recent CureCast interview with Mark and his colleague, Jordan Squiar.
Kajana Satkunendrarajah, PhD, Medical College of Wisconsin, will present on her recent work to restore breathing function in people with high level cervical injuries using a combination of gene therapy and designer drugs to activate receptors to induce regeneration among a sub-population of interneurons associated with respiratory function. Animal studies have been quite promising. She thinks the work has potential to significantly reduce ventilator dependency, improve wheelchair mobility and overall quality of life for people living with cervical SCI. Dr. Satkunendrarajah also discussed her work on this episode of our CureCast podcast.
Commercializing Cures
Session six will discuss the challenges and promise of commercializing regenerative treatments for SCI. NervGen’s Chief Medical Officer, Daniel Mikol, MD, PhD, will provide an update on the company’s Phase 2 trial in chronic SCI at the Shirley Ryan Ability Center in Chicago.
NervGen’s molecule (NVG), may help spinal cord axons to grow past the injury scar area; U2FP has covered this drug and trial since they emerged from preclinical studies in the Jerry Silver lab. Mikol will talk about trial design, the evolution of outcomes measurements and the emerging use of biomarkers and other non-motor score measures. He will also discuss plans for future trials.
Jessica Kwok, PhD, at the University of Leeds is co-founder of Neurosolv, a company that developed a two-part, non-surgical SCI therapy based upon neural signaling Interventions and direct treatment of the glial scar to target scar-related sugar molecules called chondroitin sulfate proteoglycans or CSPGs.
The company says the oral therapy works better than invasive techniques and can potentially treat acute and chronic SCI. Kwok will provide an update on plans for a Phase 1 trial using Perineline. The therapy has an Orphan Designation from the European Medicines and has been approved for use in a congenital liver dysfunction; this should provide regulatory acceleration in Europe and the US for the SCI therapy.
Michael Lauw, of Lineage Cell Therapeutics will discuss the company’s ongoing oligodendrocyte precursor cells (OPC1) trials and the company’s plans to launch a chronic injury cohort with their cell line. These are the same cells the company Geron pioneered in a small number of acute SCI patients beginning in 2009; the line was acquired by Asterias, then Lineage.
This presentation will give a history of OPC1 cells, which have been transplanted in about 25 patients. The cells are designed to replace or support cells that are absent or broken due to traumatic injury. Lauw will discuss what has been learned, what progress has been made, and what is needed to make cell therapies a viable and commercially available treatment.
Poster Session
Session seven will feature a poster session, showcasing works in progress on a single 3 x 5 board including hypothesis, data, and analysis. This is an opportunity for graduate students and scientist-trainees to present their work and get feedback from colleagues and - especially in this setting - people with lived SCI experience.
This interaction is critical to bridge the gaps between science studies and the unmet needs of the SCI community. Young scientists learn to communicate complex science to laypeople, and people with SCI gain a better understanding of the challenges inherent in scientific discovery.
Strategy - Breakout Groups
A breakout session from U2FP’s Symposium last year in Minneapolis.
Session eight continues U2FP’s efforts to facilitate in-person conversation about several intriguing topics. Symposium participants will stay in place in five groups; hosts for each session rotate to meet with each of the groups to discuss:
- Social Media: An increasingly important means of messaging and information, but mostly unfiltered and uncurated. How do we break through the noise? Hosts: Matt Edwards, Quinn Brett, Reveca Torres and Gabriel Rodreick.
- Whole Body Translation: SCI is not a set of symptoms but a single system, can it be treated as a whole? Host: David Magnuson
- Federal CAN: a proposal to shift successful ($40 million so far) SCI cure research funding advocacy from state programs to federal agencies (NIH, DOD, etc. Hosts: Jason Stoffer, Jake Beckstrom
- Commercialization: It’s beginning to happen, there may be a business in SCI treatments. How can the SCI community help? Hosts: Daniel Mikol, Michael Lauw
- An Untapped Resource: Activity Based Therapy (ABT) Centers can be an essential collaborator in clinical trials. Hosts: Christel Mitrovich, Tommy Sutor
Each of these topic rounds will ask questions that echo the proximity frame described on day one: How do you see/understand/engage with this information, this challenge, from your point of view? And how and where do our points of view diverge and converge?
U2FP is committed to accelerating curative therapies for chronic spinal cord injury. We want to hear your point of view and want you to gain new insights by being in proximity to new people and new perspectives. Considering joining us if you haven't already signed up.
Stay curious,
