The therapy will use an intravenous infusion of DNA that inhibits the expression of the transthyretin gene. The infusions will be done every three weeks as part of the study, which will also feature a sample of people receiving a placebo. It will reduce the levels of the gene in the people receiving the infusions and will further inhibit the full expression of the disease.

The study will be done over 18 months and is part of a global multicentre study known as Apollo, which is funded by the American company Alnylam. People receiving the gene therapy will be monitored at the CING.

FAP 1 is a disease that involves the production of transthyretin by the liver, which then turns into an amyloid, an inappropriately folded protein, which affects the nerves nearby and the nervous system as a whole. People exhibit pains and discomfort in the lower gastrointestinal system. If untreated, the disease can be fatal in ten years.

The disease affects about five in 100,000 Cypriots and is carried from generation to generation. It first arrived on the island in the 12th century, with the crusades. In Cyprus, the FAP 1 carriers of the disease are among 22 families mainly from the Kyrenia and Limassol districts. In Limassol, the carriers are mainly from the village of Pachna.

In 1990, the UK offered liver transplants as part of the first attempts of healing people afflicted by the disease, and today about 45 Cypriots have been given transplants. People, however, sometimes have to wait up to 12 months for a transplant making the new therapy all the more necessary.

In Europe, FAP 1 is already being fought with the use of the drug Tafamidis. The medicine slows the progression of the disease until a liver transplant can be done.

Source: Cyprus Mail