FAC8CELL®

Cellular therapy for the treatment of haemophilia.

Fac8Cell® is a liver product aimed at producing factor 8, a blood clotting agent absent or defective in people with haemophilia. There are also potential applications of the treatment in liver failure and inborn errors of metabolism.

Product Demand

• Haemophilia is a lifelong condition and there is currently no cure
• One in 10,000 males born worldwide has haemophilia. It is one of the most expensive diseases to treat, more than $A100,000 per year for each patient
• Inborn errors of metabolism collectively account for about 3% of all causes of inherited diseases causing intellectual handicap
• Acute liver failure from hepatitis and cirrhosis is common. “Liver assist” devices similar to kidney dialysis could be improved by use of encapsulated porcine liver cells.

Product Development

Liver cells from newborn pigs have been grown successfully and encapsulated. Preclinical experiments have been carried out in a mouse model of haemophilia.

Publications

• Garkavenko O, Emerich DF, Muzina M, Muzina Z, Vasconcellos AV, Ferguson AB, Cooper IJ, Elliott RB.
  Xenotransplantation of neonatal porcine liver cells.
  Transplant Proc. 2005 Jan-Feb;37(1):477-80. Read abstract

  Transplant Proc. 2005 Jan-Feb;37(1):477-80.
  Xenotransplantation of neonatal porcine liver cells.
  Garkavenko O, Emerich DF, Muzina M, Muzina Z, Vasconcellos AV, Ferguson AB, Cooper IJ, Elliott RB.
  Living Cell Technologies LTD, Auckland, New Zealand. olga@diatranz.co.nz

  Xenotransplantation of porcine liver cell types may provide a means of overcoming the shortage of suitable donor tissues to treat hepatic diseases characterized by inherited inborn errors of metabolism or protein production. Here we report the successful isolation, culture, and xenotransplantation of liver cells harvested from 7- to 10-day-old piglets. Liver cells were isolated and cultured immediately after harvesting. Cell viability was excellent (>90%) over the duration of the in vitro studies (3 weeks) and the cultured cells continued to significantly proliferate. These cells also retained their normal secretory and metabolic capabilities as determined by continued release of albumin, factor 8, and indocyanin green (ICG) uptake. After 3 weeks in culture, porcine liver cells were loaded into immunoisolatory macro devices (Theracyte devices) and placed into the intraperitoneal cavity of immunocompetant CD1 mice. Eight weeks later, the devices were retrieved and the cells analyzed for posttransplant determinations of survival and function. Post mortem analysis confirmed that the cell-loaded devices were biocompatible, and were well-tolerated without inducing any notable inflammatory reaction in the tissues immediately surrounding the encapsulated cells. Finally, the encapsulated liver cells remained viable and functional as determined by histologic analyses and ICG uptake/release. The successful harvesting, culturing, and xenotransplantation of functional neonatal pig liver cells support the continued development of this approach for treating a range of currently undertreated or intractable hepatic diseases