Don't Like Your Genes? Pay to Trade!

The world of medicine has arrived at the point we've all been waiting for; we can finally preform gene replacement! Don't worry about the cool Million Plus Euro that it will cost you just yet because it won't be approved for sale until April 2015. The Dutch biotech company UniQure has created and tested a gene replacement regimen for lipoprotein lipase deficiency (LPLD) and long-term results are promising. 

LPLD- What is it?  

Lipoprotein lipase (LPL) is needed to break down triglycerides into free fatty acids that the body can then use. Without these necessary enzymes, the triglycerides build up in the capillaries of the digestion system and can result in serious health complications, the most common of which is pancreatitis (Inflammation and dysfunction of the pancreas which is responsible for releasing the majority of the enzymes needed to digest all food ingested). The LPLs are normally made in the muscle cells and then transported to the digestive capillaries, but with LPLD there is an error in the gene that codes for these LPLs causing either dysfunctional LPLs to be made which then get destroyed by the body’s immune system or simply not enough LPLs are made. LPLD is a rare disease impacting only a small number of people in the population.

Glybera - How Gene Replacement Works

Glybera, the commercial name of the gene replacement therapy, takes advantage of our friend the virus, alipogene tiparvovec, which is rendered harmless before use. As seen in the image (left), the new DNA for the gene that codes LPL is inserted into the virus (referred to as a vector) and the person impacted by LPLD is simply infected with the virus through a series of injections. The virus then invades the cells of the body like any other virus and begins to transcribe and translate the needed gene into the proteins that form LPLs. The alipogene virus is a great vector because the body has a minimal immune response to it that prevents it from being cleared from the body. The human trials are lasting into their 6^th year with almost zero complications and continued expression of the LPL gene.

The Major Considerations

As the first major treatment of its kind to reach the market, it will serve as a foundation that all future gene replacement approaches will be modeled after. As mentioned in the beginning the current estimated price for a 65kg patient will be around 1 Million Euro (Glybera won’t reach the U.S. until 2018 at the earliest). The pricing structure is considered valid by many in the industry and actually matches other replacement therapies that are currently employed to treat rare diseases. Other gene replacement treatments being developed for more common diseases will have a lower cost due to the larger consumer pool. The drug company is mostly concerned with recouping the huge investments that were required to develop the therapy. Only 23-30% of the cost will be considered royalties for UniQure; the rest will be to cover the expense of making the vectors themselves.

It is important also to consider the implications of the future potential of gene replacement. We aren’t to the point of changing our eye and hair color with genes yet, but as this market begins to form within the medical community, its important to remember there is always the potential for long-term impacts that don’t seem obvious. This project could bring a whole new meaning the term designer genes.   

Resources: 

http://www.uniqure.com/uploads/Publications/GL008_clinical_2010.pdf

http://news.yahoo.com/exclusive-first-gene-therapy-drug-sets-million-euro-173717146--finance.html

Image:

http://en.wikipedia.org/wiki/Alipogene_tiparvovec#mediaviewer/File:AAV_Gene_Therapy.jpg