In recent decades, with the advancement of medical science, faulty genetic makeup of individuals has been blamed for a number of diseases. A potential cure for such gene related diseases has been gene therapy which has been in the news a lot of late.
Intended to provide a cure for rare eye diseases, a new gene therapy has been developed by Sparks Therapeutics which is being touted as the biggest breakthrough which also involved a unanimous backing from U.S. Food and Drug Administration’s advisory panel.
But does gene therapy deserve all that hype and what exactly is gene therapy?
And a simple explanation of how it all works, and why she’s excited about it, was all explained by Canaan Venture Partners’ biotech investor Nina Kjellson during a detailed television interview.
Gene therapy is a technique that uses genes to treat disease, Kjellson explains, possibly in the most simplest of terms.
Early trials were not uniformly successful when the first patients were tested for treatment with gene therapy about twenty-five years ago. An 18-year-old Jesse Gelsinger, who got an infusion of trillions of genetically engineered viruses into his liver, died as a result of the initial gene therapy tests in one notable case.
However, gene therapy, which treats a rare condition that causes a form of blindness that often starts in childhood, is a therapy that has at least delivered better results for Sparks, compared to those 20 years ago.
Carrying of a healthy version of the gene into the retina of the ailing patient is done using a modified version of a virus in the new and potential breakthrough gene treatment. The gene inserted into the retina can be thought of as a good copy of the gene.
And what appears to be the crazy part is billions of these viruses — or “vectors” — would be injected by the doctors into both of the patient’s eyes. The aim of the process is to reach the cells where it’s needed.
Even though the FDA usually follows the advice of the advisory committee, it hasn’t approved Sparks’ therapy just yet.
But the price of such treatment is a big obstacle that remains with the treatment. This is because insurance companies might decline to cover the very high price of $1 million which is the expected costs for each patient.
Starting with genetic disorders caused by mutations in single genes, and because of the fact that they could treat a wide variety of medical conditions, these experimental therapies have a lot of promise as far as Kjellson is concerned.
And these serious types of health diseases could be fixed at its root through gene therapy some day and replace drugs and surgery and this the hope of scientists, even though they agree that its still early days.
(Adapted from CNBC)