“Dancing molecules,” a new treatment for acute spinal cord injuries developed at Northwestern University, has been granted Orphan Drug Designation by the U.S. Food and Drug Administration (FDA). This innovative therapy was created by Samuel I. Stupp, a pioneer in regenerative nanomedicine, who first introduced the platform in 2021 through a study published in Science.
The FDA’s Orphan Drug program aims to support treatments for rare diseases or conditions with benefits like tax credits for clinical trials and up to seven years of market exclusivity post-approval. Amphix Bio, a company originating from Stupp’s laboratory at Northwestern, is managing the FDA approval process and plans to begin trials on spinal cord injury patients by late 2026.
Stupp stated, “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.” The therapy involves injecting liquid into the injured area of the spinal cord where it forms nanofibers that support cell growth and help re-establish connections severed by injury.
The National Spinal Cord Injury Statistical Center reports about 18,000 new cases of acute spinal cord injury annually in the United States. Dr. James Guest from the University of Miami commented on this approach as having potential for breakthroughs in neural regeneration.
Northwestern University holds financial interests in Amphix Bio, as does Samuel Stupp.