The treatment of ‘dance molecules’ receives designation of orphan drugs of the FDA

“Dance molecules”, the new promising treatment for acute spinal cord injuries developed at the Northwestern University, has received the designation of orphan drugs from the United States Food and Medicines Administration (FDA).

Developed by the regenerative pioneer of Nanomedicine Samuel I. Stupp, the therapy takes advantage of the molecular movement to reverse the paralysis and repair the tissues after the traumatic lesions of the spinal cord. Stupp first introduced the platform in 2021 in a study published in the journal Science. In that study, a unique injection administered 24 hours after a serious injury helped mice recover the ability to walk, only four weeks after treatment.

The FDA orphan drug program is designed to foster and support the development of treatments for rare diseases or conditions. The benefits of the designation include financial incentives such as fiscal credits for clinical trials, exemption from user rates and up to seven years of exclusivity of the market after approval.

Amphix Bio, a company left the Northwestern Laboratory of Stupp, is helping to navigate the FDA approval process and has received early comments from the agency on the drug development program for acute spinal cord injuries. Amphix Bio is aimed at the end of 2026 for the first trials in patients with spinal cord injuries and is now completing the safety studies necessary for regulatory approval to begin human tests.

“Since we first publish our results in 2021, we have validated the capacity of these molecules to regenerate the functional neuronal tissue in new animal models, different types of spinal cord injury and in other neurodegenerative diseases,” Stupp said. “This gives us confidence that we are on the right path to develop a new solution to treat this weakening and very challenging condition.”

The scientific director of Amphix Bio, Stupp is a professor of trustees at the Board of Science and Engineering of Materials, Chemistry, Biomedical Medicine in Northwestern, where you have appointments at the McCormick School of Engineering, Weinberg College of Arts and Sciences and Feinberg School of Medicine. He also directs the Center for Regenerative Nanomedicine.

To administer the treatment, researchers inject liquid therapy in the region where a spinal cord injury occurred. The liquid is then gela in a network of nanofibers, which serve as a scaffold to support cell growth. These nanofibers contain bioactive signals that trigger powerful regenerative pathways, which allows the motor neurons of the brain to grow the site of the lesion and connect to the lower spinal cord, restoring the connections that were cut by the lesion.

When controlling the collective movement of the “dance molecules” within the fibers, scientists discovered that intensifying the molecular movement increased the signaling power of therapy and led to a greater regeneration of tissues and a functional improvement in injured animals.

According to the National Center for Lesions of the Bite of the Spinal Bite of the University of Alabama in Birmingham, the United States has approximately 18,000 new cases of acute spinal cord injury every year.

“To experience a spinal cord injury is devastating for patients and their families, since it leads to a life of loss of mobility, high costs of medical care and a great dependence on caregivers,” said Dr. James Guest, neurosurgeon and professor of neurological surgery at the University of Miami and the Miami project to cure paralysis. “The previous treatments have faced many limitations, but Professor Stupp’s team and Amphix’s biography are adopting a completely new approach to neural regeneration, with the potential of an advance in the field.”

Northwestern University has financial interests (that is, capital) in Amphix Bio. Samuel Stupp also has financial interests (that is, capital) and affiliations with Amphix Bio.