New Insights on Therapy for Spinal Cord Injury

Global prevalence of Spinal Cord InjuryI is estimated to be between 0.7 and 1.2 million cases per year, with falls and road accidents being the major causes. Despite incurring a cost of $4 billion per year in direct healthcare and indirect costs in the US alone, there are no licensed drugs that can tackle the intrinsic failure of the adult central nervous system to regenerate, and thus remains a largely unmet clinical need.

A new research has found that the KCL-286 drug — which works by activating retinoic acid receptor beta (RARb) in the spine to promote recovery — was well tolerated by participants in a Phase 1 clinical trial, with no severe side effects.

109 healthy males were divided into one of two trial groups; single ascending dose (SAD) adaptive design with a food interaction (FI) arm, and multiple ascending dose (MAD) arm.

SAD studies are designed to establish the safe dosage range of a medicine by providing participants with small doses before gradually increasing the dose provided. Researchers look for any side effects, and measure how the medicine is processed within the body. MAD studies explore how the body interacts with repeated administration of the drug, and investigate the potential for a drug to accumulate within the body.

Researchers found that participants were able to safely take 100mg doses of KCL-286, with no severe adverse events.

Professor Jonathan Corcoran,  the study’s senior author said, “This represents an important first step in demonstrating the viability of KCL-286 in treating spinal cord injuries. This first-in-human study has shown that a 100mg dose delivered via a pill can be safely taken by humans. Furthermore, we have also shown evidence that it engages with the correct receptor.

“Our focus can hopefully now turn to researching the effects of this intervention in people with spinal cord injuries.”

Dr. Bia Goncalves, a senior scientist and project manager of the study, and the study’s first author from King’s IoPPN said, “Spinal Cord Injuries are a life changing condition that can have a huge impact on a person’s ability to carry out the most basic of tasks, and the knock-on effects on their physical and mental health are significant.

Sources:

Maria B. Goncalves, Tim Mant, Jörg Täubel, Earl Clarke, Hana Hassanin, Daryl Bendel, Henry Fok, John Posner, Jane Holmes, Adrian P. Mander, Jonathan P. T. Corcoran. Phase 1 safety, tolerability, pharmacokinetics and pharmacodynamic results of KCL‐286, a novel retinoic acid receptor‐β agonist for treatment of spinal cord injury, in male healthy participants. British Journal of Clinical Pharmacology, 2023; DOI: 10.1111/bcp.15854

King’s College London. “New orally available drug for spinal cord injury found to be safe and tolerable in healthy participants.” ScienceDaily. ScienceDaily, 10 August 2023. <www.sciencedaily.com/releases/2023/08/230810110422.htm>.

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