Groundbreaking Drug Shows Promise in Treating Spinal Cord Injuries
A groundbreaking drug has shown promising results in the treatment of spinal cord injuries (SCI), according to a recent study published in the British Journal of Clinical Pharmacology. The drug, known as KCL-286, activates retinoic acid receptor beta (RARb) in the spine to promote recovery. In a Phase 1 clinical trial, researchers found that the drug was well tolerated by participants with no severe side effects.
Spinal cord injuries affect millions of people worldwide, with falls and road accidents being the leading causes. Despite the significant healthcare costs and the adverse impact on individuals’ ability to work and their need for long-term care, there are currently no licensed drugs available to address the failure of the adult central nervous system to regenerate. Therefore, finding an effective treatment for spinal cord injuries remains a major unmet clinical need.
Previous research has demonstrated that nerve growth can be stimulated by activating the RARb2 receptor. However, until now, no drug suitable for human use has been developed. KCL-286, an RARb2 agonist, has been specifically designed to address this gap. In a first-in-human study, 109 healthy male participants were divided into two trial groups, receiving different doses of the drug to assess its safety and tolerability.
The study revealed that participants were able to safely take 100mg doses of KCL-286 without experiencing severe adverse events. Professor Jonathan Corcoran, the Director of the Neuroscience Drug Discovery Unit at King’s College London and senior author of the study, expressed his optimism about the potential of the drug, stating, This represents an important first step in demonstrating the viability of KCL-286 in treating spinal cord injuries. This study has shown that a 100mg dose delivered via a pill can be safely taken by humans, and we have also provided evidence that it engages with the correct receptor.
Dr. Bia Goncalves, the senior scientist and project manager of the study, and the study’s first author from King’s College London, highlighted the life-changing impact of spinal cord injuries and their significant physical and mental health consequences. Dr. Goncalves expressed her hope for future research, stating, The outcomes of this study demonstrate the potential for therapeutic interventions for SCI, and I am hopeful for what our future research will find.
Professor Elizabeth Bradbury, co-Head of the Wolfson Centre for Age-Related Diseases at King’s College London, also commented on the significance of this breakthrough, emphasizing that KCL-286 is part of a pipeline of regenerative therapies being developed at the university to address currently incurable spinal cord injuries. Professor Bradbury expressed her enthusiasm for the future, envisioning a world where regenerative therapies can reconnect nerves and restore function to paralyzed muscles, providing relief to the millions of people worldwide living with the lifelong consequences of such injuries.
This groundbreaking research was made possible thanks to funding from the Medical Research Council. As the positive results obtained in the Phase 1 trial pave the way for further research and clinical trials, the hope for a new treatment for spinal cord injuries grows stronger.
In conclusion, the groundbreaking drug KCL-286 has shown promise in treating spinal cord injuries by stimulating nerve growth through the activation of the RARb2 receptor. The drug was well tolerated by participants in a Phase 1 clinical trial, prompting researchers to seek funding for a Phase 2a trial to further study the safety and tolerability of the drug in individuals with SCI. This exciting development brings hope to millions of people worldwide who are living with the lifelong consequences of spinal cord injuries, and it represents a significant step toward finding an effective treatment for this currently unmet clinical need.
