Surgical procedure. Image by Pfree2014 – Own work, CC BY-SA 4.0
“Dancing molecules”, as dubbed by the researchers, is a promising new treatment for acute spinal cord injuries developed at Northwestern University. The novel drug has received Orphan Drug Designation from the U.S. Food and Drug Administration (FDA).
Developed by regenerative nanomedicine pioneer Samuel I. Stupp, the therapy harnesses molecular motion to reverse paralysis and repair tissues after traumatic spinal cord injuries.
Stupp first introduced the platform in 2021 in a study published in the journal Science (“Bioactive scaffolds with enhanced supramolecular motion promote recovery from spinal cord injury”). In that study, a one-time injection administered 24 hours after severe injury helped mice regain the ability to walk. This occurred just four weeks after treatment.
The FDA’s Orphan Drug program is designed to encourage and support the development of treatments for rare diseases or conditions. The designation’s benefits include financial incentives such as tax credits for clinical trials, exemption from user fees and up to seven years of market exclusivity after approval.
Amphix Bio, a company spun out from Stupp’s Northwestern laboratory, is designed to help navigate the FDA approval process. The firm is targeting late 2026 for the first trials in spinal cord injury patients and is now completing safety studies required for regulatory approval to begin human trials.
“Since we first published our results in 2021, we have validated the ability of these molecules to regenerate functional neural tissue in new animal models, different types of spinal cord injury and in other neurodegenerative diseases,” Stupp says in a research brief. “This gives us confidence that we are on the right track in developing a new solution to treat this debilitating and very challenging condition.”
To administer the treatment, researchers inject the liquid therapy into the region where a spinal cord injury occurred. The liquid then gels into a network of nanofibers, which serve as a scaffold to support cell growth. These nanofibers contain bioactive signals that trigger potent regenerative pathways, enabling motor neurons from the brain to regrow past the injury site and connect to the lower spinal cord. This re-establishes connections that were severed by injury.
By controlling the collective motion of the “dancing molecules” within the fibres, scientists discovered that intensifying the molecular motion increased the therapy’s signalling power and led to greater tissue regeneration and functional improvement in the injured animals.
As a sign of the importance of the research, according to the U.S. National Spinal Cord Injury Statistical Center at the University of Alabama at Birmingham, the U.S. has approximately 18,000 new cases of acute spinal cord injury each year.