LCT's Huntington's disease treatment shows potential

The results signify a leap forward in the area of Huntingdon's research and similarly related diseases. There is currently no other neuroprotective cell transplant product targeting the treatment of Huntington's disease itself, not just the symptoms.

Huntington's disease is a devastating neurological disease that currently has no cure or treatment. It is an inherited disease that progresses rapidly with dementia and progressive movement difficulties. More than 1 in 100,000 people are affected by HD.

Living Cell has revealed that brain cell damage in primates treated with NtCell was 5 times less than in control animals affected by HD (approximately 50 per cell death versus 10 per cell).

NtCell is unique in its approach as the treatment uses natural pig cells that are encased in a bio-polymer capsule (biocapsule) developed from seaweed. The cells used are choroid plexus brain cells, which produce spinal cord fluid and a range of neurotrophins, or protective proteins, for the repair and function of the brain.

The procedure involves transplantation of the biocapsule into the region of the brain predominantly affected by HD, known as the striatum, which was lesioned by a chemical that mimics the HD process.

The biocapsules act as an immune barrier, allowing the therapeutic cocktail of proteins produced by the cells to leave the capsule. As a result the body's immune system can't reject the cells and no immunosuppression is required for this treatment.

"The product appears to protect brain tissue that would otherwise die and has the potential to forestall or prevent the debilitating consequences of neurodegenerative disease,"​ said Al Vasconcellos, CEO of LCT.

Currently, genetic screening is the only means in providing a pre-warning to identify individuals that will ultimately suffer from HD prior to their developing symptoms. However, the process is not 100 per cent effective.

The study placed 20 biocapsules into the brains of 7 primates. Four animals were treated with NtCell and three control animals received identical capsules without live cells. The results confirm similar results observed in Huntingdon rat models, also performed by LCT.

LCT's latest primate study provides the essential data required by regulatory authorities such as the FDA before trials in humans can be approved.

"The long term survival of the choroid plexus cells in the brain delivered in this way opens the strong possibility that this treatment approach could be used effectively in disorders such as Alzheimer's and Parkinson's diseases as well as stroke and Lou Gehrig's disease,"​ commented LCT's medical director, Bob Elliott.

DrugResearcher.com previously reported on the work​ that LCT has been doing with pig islet cells that were retrieved from a diabetic patient nine years after they were transplanted in an operation that did not require the use of immunosuppressive drugs.

The pig islets were also encapsulated with a seaweed-derived coating alginate capsule to protect them from immune rejection hence no immune suppressive drugs were needed for this transplant.

Speaking at the time Paris Brooke, general manager of Living Cell Technologies​ told DrugResearcher.com:​ "The cells are coated with a proprietary formulation of a naturally occurring gel called alginate. Used for many decades as a food additive or surgical implant, alginate comes from seaweed and is what protects the cells of the seaweed from the harsh aquatic environment."​

"The ability to control the effective pore size dictates the capsules permselectivity (what molecules are allowed to pass through the wall). This proprietary encapsulation technology enables the company to effectively transplant replacement cells, without the use of crippling immunosuppressive drugs,"​ he added.