A Novel Personalized Cell Therapy for Chronic Limb-Threatening Ischemia

Peripheral vascular disease affects medium/small-vessels which are less amenable to surgical intervention. Stem/progenitor cell (SPCs) treatments with bone-marrow-derived cells show promising outcomes in treating vascular diseases, albeit not without adverse events related to cell mobilization and collection. A unique technology for production of enriched endothelial progenitor cells (EnEPCs) named BGC101 was developed. Requiring 250ml of blood obtained via a standard blood draw procedure makes it available in every clinic even to severely ill patients (no need for anesthesia or Mobilization). BGC101 is supplied the day after in ready-to-use syringes. BGC101 safety and efficacy was shown in a nude mice hind limb ischemia model.

We performed a "first-in-human" study in critical limb ischemia (CLI) patients characterized by rest pain and/or ulceration, and with no option for revascularization. A single treatment session of BGC101 intramuscular injections into the gastrocnemius was followed for acute safety during 48±6hours in the hospital and long-term safety and efficacy at 1week and at 1, 3, 6 and 12months.

The first cohort results have exceeded the expected safety and efficacy primary endpoints. BGC101 is found to be safe with therapeutic effects in both objective outcomes including amputation free survival, increased leg blood flow and wound healing, and, in subjective outcomes including walking ability, reduction of pain, decreased usage of narcotic medications and improved quality of life after a single treatment.

These observations indicate that BGC101 derived from a standard blood draw within less than one day, shows promising results in the treatment of CLI.