Gene Therapy Makes Noise in 2017

Gene therapy has successfully made its presence known in the medical field this last year, reported ABC News. Dr. Francis Collins of the National Institutes of Health isn’t afraid to use the word “cure” when it comes to gene therapy. 2017 was a huge year for researchers, finding much answers to treat some cancers and as well as cures for a handful of rare genetic diseases. With gene therapy doctors aim to delete, add or alter DNA to change the root of many diseases. They aim to cure, rather than to treat symptoms. So far, they’ve found success.

This type of gene manipulation is game-changing and U.S. regulators are establishing this therapy as a new mode of medication. More tests are being approved, and successes are being discovered. A gene editing expert at Stanford University says this is indeed one of those times when you’re astonished by science, and those times only happen a few times in a lifetime.

In 2017 the FDA approved CAR-T cell therapies, which have been successful for some types of lymphoma and leukemia. They aim to use this to help with other blood cancers as well. The FDA also approved Luxturna, which is the first gene therapy to cure RPE65-associated leber congenital amaurosis (LCA), a genetic disease that causes blindness. To read more about LCA, click here.

They shared responses from children cured, and nothing is more beautiful than hearing the excitement over seeing colors for the first time. Additionally, gene therapies continued to show progress with hemophilia and sickle cell, and hopefully will have more results in the near future.

While so much has come from gene therapy in the last year, there wasn’t success in trials for all diseases, and unfortunately the procedures are often very expensive. Since it’s also new, researchers will need to see if the results are long lasting, but most are confident that gene therapy will be a “one-time” fix.

With hope and the continued goal to not just treat symptoms, but to cure the underlying diseases, researchers are on the right path to make an incredibly positive impact on the rare genetic disease community.

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