Spinogenix has been awarded a research grant from the U.S. Department of Defense (DOD) to further test its lead compound as a potential treatment of amyotrophic lateral sclerosis (ALS), the company announced.
“We are pleased that the DoD has recognized the potential of our novel drug candidate to change the course of disease progression in ALS,” Stella Sarraf, PhD, founding CEO at Spinogenix, said in a press release. The grant’s amount and its duration were not disclosed in the release.
ALS is characterized by the progressive loss of motor neurons, or the nerve cells involved in the control of voluntary movement. Although some ALS treatments can slow nerve cell degeneration, they are not disease modifying and provide only a modest survival benefit. Spinogenix also noted in its release that not all patients tolerate these treatments well.
Spinogenix’s lead therapy candidate is a small, orally bioavailable, molecule designed to induce an increase in synapses — the point of contact between two nerve cells that allows them to communicate. Its goal is to restore these neuronal connections and reverse patients’ decline in cognition and motor function, two faculties often affected in neurodegenerative diseases.
Spinogenix reports that the compound’s mechanism is well understood, and believed to work across all the diseases where synapse loss occurs, regardless of the underlying disease mechanism.
With this grant from the DOD’s Congressionally Directed Medical Research Programs, Spinogenix aims to study the potential therapy using induced pluripotent stem cells (iPSCs) derived from both ALS patients and healthy controls.
iPSCs are stem cells generated from mature cells derived from the skin or blood that can give rise to different cell types, including nerve cells, depending on the particular chemical cues they are given. When these cells are derived directly from patients, they generate cellular models that mimic the disease’s genetic and clinical diversity.
Additional experiments will also be conducted in ALS animal models.
Under the grant, Spinogenix will collaborate with researchers Rita Sattler, PhD, at the Barrow Neurological Institute, and Justin Ichida, PhD, at the Keck School of Medicine of the University of South Carolina.
“Spinogenix’s novel approach has the potential to demonstrate that replacing lost synapses may result in drugs that can provide a meaningful benefit for patients with ALS,” said Merit Cudkowicz, MD, director of the Sean M. Healey and AMG Center for ALS at Mass General Hospital.
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