Treatments Amenable to Immunoisolation and Transplantation
Any disease that can be treated by the release of a cellular product (hormone, protein, neurotransmitter, etc.) is a candidate for transplantation of immunoisolated cells.
Encapsulife has focused on diabetes because of the prevalence of the disease and the potential to achieve large-scale operations and to make a substantial medical and humanitarian impact. However, in addition to immunoisolation and transplantation of pancreatic islets to address diabetes, encapsulation and transplantation strategies can be envisioned for hepatocytes (liver disease), neurons (CNS), and cells secreting clotting factors (hemophilia). There are an estimated two-dozen potential diseases where an encapsulation/transplantation strategy could result in a "functional cure" for suffering patients. Here are five diseases that show particular promise:
- Liver Disease The University of Minnesota is studying the development of an artificial liver that can serve to keep patients alive if their liver fails. The device uses liver cells that it embeds in a collagen gel. This machine is intended to help keep patients alive while they await a liver transplant. Cell encapsulation technology could provide the mechanism to encapsulate the cells used in this liver machine.
- Huntington's Disease This disease is caused by the death of nerve cells in the striatum the part of the brain that controls movement and intellectual capacity. Studies have detailed how encapsulated hamster cells were implanted in three monkeys. The implanted cells prevented the death of nerve cells in the monkeys, thereby holding off the onset of Huntington's disease. Over 25,000 people in the U.S. currently suffer from Huntington's disease.
- Hemophilia Hemophilia comes in two forms, A and B. Both are caused by low levels or the complete absence of a blood protein essential for clotting. Hemophilia A patients lack the blood-clotting protein, factor VIII, and hemophilia B patients lack factor IX. About 20,000 people suffer from hemophilia in the United States.
- Lysosomal storage disease Deficiency in lysosomal enzymes has devastating effects causing severe skeletal and organ abnormalities, often accompanied by serious neuro-degeneration and early death. In spite of their catastrophic nature and relative frequency of occurrence (one in ten thousand births), there is no cure or even definitive treatment.
- Growth Hormone The success of treating growth hormone deficient patients with recombinant human growth hormone has prompted the development of somatic gene therapy protocols for its treatment. However, growth hormone treatment is also being considered for patients with non-growth hormone deficient diseases such as ageing, osteoporosis, surgical trauma, thermal injuries and athletic activities. Non-autologous somatics gene therapy is a novel approach, which may lead to a potential cost effective method of gene-based therapy.