The FDA has approved a clinical study of inhaled stem cell therapy for COPD, allowing SMSbiotech to begin testing SMS cells in U.S. patients. The therapy uses nebulized stem cells that activate the lungs' natural repair mechanisms, with early Australian trials showing no adverse events.
- The FDA issued a Study May Proceed notification for SMSbiotech's inhaled stem cell therapy, clearing the way for phase 1b clinical trials in COPD patients.
- SMS cells are proprietary allogeneic stem cells derived from human blood that activate targeted multi-gene activity modulation (TMAM) to stimulate lung tissue repair.
- Early trial data from Melbourne, Australia shows no therapy-related adverse events in the first cohort after 28 days of safety monitoring.
- The treatment offers simple nebulized administration, eliminating the need for invasive delivery or complex genetic modification required by traditional stem cell therapies.
- SMS cells demonstrate structural and functional lung recovery in preclinical COPD animal models, supporting potential for human therapeutic benefit.
A potential therapy for COPD using inhaled stem cells cleared a hurdle in the research phase as the U.S. Food and Drug Administration (FDA) completed its review of an Investigational New Drug (IND) application and issued a “Study May Proceed” notification.
According to a news release from SMSbiotech, the FDA action clears the way for the company to begin a phase 1b clinical study of Small Mobile Stem (SMS) cells in patients with COPD and expands the development of the treatment into the United States.
The company is already investigating nebulized SMS cells in an ongoing phase 1b clinical trial, “A Study to Investigate Small Mobile Stem Cells (SMS) in Participants Aged 39 to 69 Years With Chronic Obstructive Pulmonary Disease,” in Melbourne, Australia.
That study is testing 18 participants with three different concentrations of the drug with no control group. The primary outcome will be assessed via the following measures:
- Incidence of treatment
- Emergent adverse events
- Abnormalities gathered via vital signs review and physical examination
The first cohort has been dosed and has completed the protocol-specified 28-day safety monitoring period with no therapy-related adverse events. Dose escalation to the second of three cohorts is underway.
Discovered and patented by SMSbiotech, SMS cells are a proprietary population of native, allogeneic adult stem cells derived from human blood. In preclinical studies, SMS cells have been shown to selectively bind to endogenous mesenchymal stem and progenitor cells in the lung. The resulting interaction stimulates multi-gene regenerative activity associated with alveolar tissue repair. The company calls this cellular interaction targeted multi-gene activity modulation (TMAM).
The SMS platform differs from traditional stem cell therapies that may require invasive delivery, complex genetic modification or specialized surgical settings in the following ways. Instead, the SMS model offers:
- Simple nebulized administration.
- Preclinical evidence of alveolar repair. SMS cells demonstrated structural and functional lung recovery in standard COPD animal models, consistent with TMAM-associated alveolar tissue regeneration.
- Off-the-shelf, allogeneic supply. The cells offer naturally low immunogenicity with a scalable manufacturing process designed to ensure consistent availability.
“FDA authorization to proceed represents an important milestone for the science, the platform and the team that built it,” said Abdulkader Rhamo, PhD, founder, president and chief scientific officer of SMSbiotech. “SMS cells appear to engage the lung’s own regenerative machinery through the TMAM cellular interaction, supported by a reproducible manufacturing process that is independent of donor availability. We now look forward to advancing the program with U.S. patients.”
