The classic application for encapsulated cells is the treatment of diabetes, and Cell-in-a-Box® is already being used by various groups world-wide to develop treatments for this debilitating and life-threatening disease. However, other acquired diseases such as hypercholesterolemia are also good targets for the Cell-in-a-Box®. Cells overexpressing specific enzymes would be transplanted into the patients body and Cell-in-a-Box® will ensure long term survival of the transplanted cells, as well as release of the required factors through the porous shell of the capsule.
Similarly, a number cell and gene therapies have already been tested in clinical trials for the treatment of hereditary diseases such as the various forms of mucopolysaccharidosis, haemophilia A and B, Gaucher's disease, lipoprotein lipase deficiency, ornithine transcarbamylase deficiency, Fabry disease, amyotrophic lateral sclerosis, junctional epidermolysis bullosa and many more that have yet to be treated.
Such therapies are highly specific and have great efficacy since usually only a little of the missing enzyme can alleviate or even totally cure the disease. Cell-in-a-Box® technology takes metabolic gene therapy one step further and transfers the site of enzyme production straight into the patient’s body.
To demonstrate the use of Cell-in-a-Box® technology, we have for instance shown that encapsulated insulin producing cells can be implanted into mice where they secrete insulin in a glucose dependent manner that can rescue animals from an otherwise deadly hypoglycaemia, even when the encapsulated cells are from another species.
Advantages of constant enzyme production from encapsulated cells genetically modified to make the missing enzyme:
- Avoids peaks and troughs of classical delivery
- Thus better efficiency
- Thus lower doses required
- Thus less side effects/better safety profile
- Longer treatment times means less hospital visits for patients and thus better compliance