London, UK – A new phase 1 study has demonstrated the safety and benefits of intravenous cardiosphere-derived cells (CDCs) in patients with various forms of pulmonary arterial hypertension (PAH). The study, published in The Lancet, found encouraging exploratory results and no clinical adverse events in the short or long term in this patient cohort.
“CDCs exhibit potent properties that could positively impact on PAH pathobiology (anti-inflammatory, immune modulating, anti-fibrotic, anti-oxidative and anti-apoptotic),” the authors wrote. “The primary objective of this FDA-authorized phase 1 study was to determine short- and long-term safety profile of CAP-1002 (contains CDCs) administered by central intravenous infusion, using the maximum feasible dose, to patients with IPAH, heritable PAH , PAH associated with connective tissue disease, and PAH associated with HIV.”
The research team conducted an open-label, dose-escalating, phase 1a study on 26 patients with idiopathic PAH, heritable PAH, PAH-associated with connective tissue disease, and PAH associated with HIV undergoing PAH therapy for at least 4 months. Half of the patients received 50 million CDCs and the other half received 100 million CDCs. In phase 1b, 10 patients were randomized to receive 100 million CDCs and 10 to receive placebo infusions.
The results revealed no short- or long-term safety or adverse events with either CDC dose, nor did any patient experience clinical worsening due to PAH.
Encouraging signals were observed, including improved or stabilized right ventricular end diastolic volume index, exercise capacity as measured by the 6-minute walk test, diffusing capacity, hemodynamics, and serum creatinine. These benefits were not found with placebo infusions.
The authors concluded that CDCs clearly warrant further study as an adjunctive therapy for patients with PAH. Given that only single CDC doses were used in this study, the authors suggested that upcoming phase 2 studies should include a repeat dosing design.