Scenario
Gene therapy has ventured out of the clinical research lab and is now available for treating patients. Glybera is used for patients with fat problems, especially those who have hypertriglyceridaemia and therefore face problems of clumped fat leading to pancreatitis-like symptoms and other high-fat related problems (problems with lipases). With Glybera, the virus infects cells and genetic reconstruction leads to normal function of lipase(s) and fat usage. The blood is cleared of excess fatty material and build-up. Prolonged hyperlipidaemia is halted. There is less chances of pancreatitis occurring following high fat intake (eg after makan kenduri). Patients no longer have to rush to A&E, complaining of excruciating pain of supposedly pancreatitis. Reduced occurrence of prolonged hyperlipaemia means less chances for formation of oxidised LDL (ox-LDL). Less ox-LDL means less chances for macrophages to foam cell conversion in the arterial intima, and therefore reduced atherosclerosis, hence reduced possibilities of ischaemic heart disease (IHD). So, overall, a priori, gene therapy is good for patients with genetic problems such as fat-related problems.
Other applications
Glybera should also be good for diabetics since diabetics have prolonged lipaemia. Prolonged lipaemia is a hallmark of those who have IGT (impaired glucose tolerance) before they progress to full blown diabetes. It is a big hope that Glybera can help IGT patients. IGT patients are difficult to find or trace since they don't come to hospital (they are not ill or sick and don't have diabetes). IGT is a pre-diabetic state. The other category of patients is those who have IFG (impaired fasting glucose). When fasted, IFG patients have high blood glucose. I have not seen clinical trials of Glybera in IGT and IFG patients. So we have to wait till someone does the research and the research findings are out. That's the future.
More on gene therapy at BBC News:
http://www.bbc.co.uk/news/health-20179561
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