How Gene Therapy Could Revolutionize Medicine

According to a story from Australasian Science Magazine, the dawn of the gene therapy “revolution” is upon us. While there are only a handful of gene therapies that have actually been approved, they have often been completely transformational in the treatment of whatever disease they have been designed to act on. Just recently, a gene therapy for beta thalassemia earned a conditional approval in the EU, and the spinal muscular atrophy gene therapy called Zolgensma (designed by Novartis), was recently approved in the US. This drug grabbed headlines as the most expensive drug ever, being priced at $2.1 million for a single administration.

However, a single dose is all it takes for these therapies to produce exceptionally good results. Zolgensma appears to allow kids born with spinal muscular atrophy to develop relatively normally; just ten years ago, the diagnosis was effectively a death sentence. The beta thalassemia gene therapy can help free patients from their dependence on blood transfusions. There is no denying that gene therapy can allow us to make huge strides towards cures and improved outcomes for patients.

An interesting thing about gene therapy is that its origins can be traced back to the need to treat rare, genetic disorders. Historically rare diseases have been shamefully ignored and neglected by the broader medical community and treatment advances come in fits and starts and often take a long time to appear. Some of the very first gene therapy testing, which took place in 1990, was on two children born with the rare immune system disorder severe combined immunodeficiency (SCID). These early results were not a breakthrough at the time, but they did show the potential of the gene therapy approach.

The development of gene therapy was not always a safe or smooth path. Before scientists were able to perfect the adeno-associated viral vectors that most therapies use today, less refined strategies could lead to fatal complications. 

More companies are starting to delve into the gene therapy field. This is not necessarily because they are suddenly committed to developing a bunch of treatments for rare diseases, but because it is starting to become clear that gene therapy could be transformational for the medical field. Gene therapy may also finally help trigger some much needed conversations about the cost of medicine and the value of human lives.

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