Efficacy shown in Mouse Models
Lysosomal Storage Diseases
Mucopolysaccharidosis Type VII is a typical lysosomal storage disease due to the deficiency of an enzyme beta-glucuronidase, resulting in catastrophic chronic consequences . It is inherited as an autosomal recessive genetic disease in humans. The same disease occurs in a murine model, the GUS mouse. When this murine mutant is implanted with microcapsules containing cells engineered to secrete beta-glucuronidase, many of the organ and behavioural pathologies were significantly improved.
A universal hallmark of cancer is uncontrolled cell growth. Recent developments in cancer treatment have focused on using potent biologicals to either increase killing or inhibit growth of cancer cells. When tumor-bearing mice were implanted with encapsulated cells engineered to secrete molecules with cancer killing (cytokines) or growth inhibition (angiostatin) properties, their survival and tumor burden were significantly improved. The efficacy of this treatment was further enhanced when multiple types of molecules were delivered at the same time.
Hemophilia is a coagulation disorder in humans caused by the genetic deficiency of blood clotting factors such as Factor VIII or IX. When cells engineered to secrete such factors were encapsulated in alginate microcapsules, they provided a persistent source of such factors not only in the petri-dish, but also in mutant mice suffering from the same disorder.
Growth hormone deficiency leads to growth retardation and dwarfism. When dwarf mice genetically deficient in growth hormone were implanted with encapsulated cells engineered to secrete growth hormone, significant growth enhancement (>20%) was achieved. This was the first proof-of-principle in the efficacy of the microencapsulation platform technology.