ATLANTA – A new clinical study can begin enrolling newly injured patients with a spinal cord injury today to test the safety and effectiveness of a new investigational drug called SUN13837. The study aims to determine whether the drug – initially administered within 12 hours of injury and then daily for 28 days – improves function in adults with acute spinal cord injury (SCI) compared to a placebo.
“If proven effective, certain patients might experience less spinal cord damage or improved function with this drug,” said Keith Tansey, M.D., Ph.D., Shepherd Center’s director of spinal cord injury research and the study’s principal investigator at the hospital. Shepherd is one of the first U.S.-based rehabilitation centers to participate in this Phase II, randomized, placebo-controlled, double-blinded study.
SUN13837, which is also being studied in people with ALS (Lou Gehrig’s disease), mimics beta fibroblastic growth factor (bFGF) properties.
“This is exciting because growth factors like FGF keep neurons alive and healthy, and promote axonal regrowth,” said Nick Boulis, M.D., lead investigator on the study and assistant professor of neurosurgery at Atlanta’s Emory University Hospital and Grady Memorial Hospital, one of the initial collaborating trauma centers.
To date, these growth factor therapies have been plagued by delivery issues because they are proteins, Dr. Boulis explained. But this investigational compound is smaller.
“As a small molecule, we will be able to give it intravenously, which means earlier in the process,” he added. “I could even envision giving it in the field ultimately.”
Unlike bFGF, SUN13837 does not cause a proliferation of cells treated with it. While the exact mechanism of action is still unknown, researchers have some theories.
“This drug might save injured tissue from dying and/or provide better connections between nerve cells after injury in a way similar to how growth factors present during nervous system development help to ‘wire up’ the nervous system,” Dr. Boulis said.
The study, sponsored by Asubio Pharmaceuticals Inc., part of the Daiichi Sankyo R&D group, is expected to enroll 164 patients at 60 acute trauma centers. Patients will be selected based on baseline cervical motor level of injury (ASIA Impairment Scale A or complete C-4 to -7 spinal cord injuries) and randomly assigned to receive the placebo or active treatment.
Researchers will determine if patients receiving SUN13837 are more likely to respond to the treatment (and to what extent) compared to those in the placebo group. A person who has responded to treatment is viewed as someone who has a greater potential to perform typical activities of daily living, such as eating or bathing, independently (or with minimal assistance). Moreover, the drug level in the bloodstream will be compared to the response to determine the relationship between dose, effect (efficacy or clinical symptoms) and safety.
SUN13837 has shown neuroprotectant properties in previous animal studies.
“In pre-clinical studies, use of this drug improved locomotor scores, but it is not clear whether the reason was neuroprotection at the injury site, regeneration at the injury site or neural plasticity below the level of spinal cord injury,” Dr. Tansey said. Neural plasticity is the idea that the spinal cord can adjust itself functionally by reorganizing neural circuits and allowing some recovery of lost function.
Still, cautious optimism is needed until this and other studies are conducted, researchers said. But they are hopeful this study may open new doors for adults with acute SCI.
“Even a minor neurological improvement in these patients can have a tremendous impact on daily functioning,” explained Issi Clesson, RN, director of clinical trials at Shepherd Center. “Being able to offer a medication, if approved, rather than an invasive procedure that exposes the spinal cord would be simpler to implement and easier on the acute SCI patient. Since the investigational product is not derived from stem cell sources, it is not controversial.”
An added benefit of the study design is the collaboration between trauma centers and rehabilitation and spine centers.
“I’m excited about this trial because it will bring together the rich resources of Shepherd Center with the strength of Grady’s neurotrauma and emergency room resources and Emory University’s strength in translational spinal cord regenerative medicine,” Dr. Boulis added.
Not only will Shepherd Center serve as a study site monitoring patient outcomes, its therapists will be in the field to train trauma centers on neurological assessments to determine which patients qualify for the study.
For more information on spinal cord injury research, visit www.shepherd.org/research/spinal-cord-injury.