NeuroScientific Biopharmaceuticals (ASX: NSB) has confirmed positive outcomes from a Special Access Scheme trial of its patented StemSmart mesenchymal stem cell (MSC) therapy on patients with fistulising Crohn’s disease.
StemSmart treatment of five patients, which commenced in October, was reported to be safe and well tolerated, with no serious adverse events observed.
The trial’s cohort consisted of two female and three male subjects between 18 and 49 years of age.
Four patients demonstrated a clinical response defined by the closure of more than 50% of fistula openings or a decrease of more than 50% in fistula discharge, while the remaining patient showed a partial response.
The results set the foundation for the company’s anticipated entry into the global market for Crohn’s disease treatment, which is estimated at US$13.8 billion.
Patient Improvements
Assessment using internationally recognised scoring systems for Crohn’s disease showed that all patients experienced an improvement in the Crohn’s Disease Activity Index, Perianal Disease Activity Index, and Inflammatory Bowel Disease Questionnaire Quality of Life Index.
Magnetic resonance imaging indicated a trend toward healing of fistulas for all patients but was considered too early in the treatment to observe fistula closure.
Furthermore, all patients with surgical implants to ensure drainage and help prevent infection of fistulas (known as setons) had at least one implant removed, which is believed to be a significant indicator of fistula healing.
Patients participating in the Special Access Scheme trial were living with fistulising Crohn’s disease, one of the most severe and debilitating complications of inflammatory bowel disease often resistant to conventional treatments.
Each patient was approved by Australia’s Therapeutic Goods Administration for treatment using StemSmart therapy under the Category B pathway designed to address unmet medical needs for those with serious and life-threatening conditions.
‘Exceptional’ Response Rate
NeuroScientific chief medical officer Dr Cathy Cole said the company was pleased with the trial outcomes.
“A clinical response rate of 80% to a novel treatment for a serious, debilitating, and long-standing medical condition that largely affects younger adults is exceptional,” she said.
“Considering these fistula patients were treated in a real-world setting and had limited treatment options available, the response is outstanding and offers new hope for clinical recovery.”
Dr Cole said the company would proceed to a Phase 2 trial focused on frequency and timing of MSC doses and MRI assessments.
NeuroScientific’s StemSmart product is derived from adult human donor bone marrow-sourced MSCs and produced using a patented manufacturing process designed to improve therapeutic activity and clinical response.
Early indications from a previous Phase 2 trial into refractory Crohn’s disease suggest StemSmart MSCs are potent, efficacious, and safe.
