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Treating Spinal Muscular Atrophy With RNA Therapeutics

First Posted: Mar 23, 2017 04:01 AM EDT
Treating Spinal Muscular Atrophy With RNA Therapeutics
Extremely optimistic results recorded in clinical trial of Spinraza. (Image for representation only.)

(Photo : Goodentree/YouTube screenshot)

RNA therapeutics have been around since the 20th century, but the commercialization of the concept was hindered due to various factors. These include competition with conventional drugs, some explained results in clinical trials and, above all, lack of awareness. When pharmaceutical companies realized that RNA therapeutics-based drugs are lagging behind in the market of prevalent chronic diseases, they shifted their focus on developing new RNA drugs that can cure rare and as of yet incurable genetic disorders like spinal muscular atrophy.

One such drug "Spinraza" used for treatment of this spinal muscular atrophy is recently approved by the U.S. government. As of now, the drug is in the clinical trial phase. The trials are indicating highly optimistic response in patients. Cameron, who was born with the disease, is a participant in the clinical trial of Spinraza.

According to his mother and father, there has been remarkable improvement in his condition. While most children fail to survive the first year, Cameron has shown immense progress in terms of body movements. A video shot after only two months of treatment shows that he is now able to move his head and is even trying to reach for his toy, Business Insider reported.

According to Med Page Today, Cameron's case is unprecedented. The article also quoted that Cameron's "Lazarus-like revival points to a new generation of drugs." Though the therapeutic power of RNAs has been realized long back, its true potential is yet to be realized.

Scientists are fully aware of the revolution that RNA therapeutics could bring in the near future. Steven Dowdy from the School of Medicine at the University of California has clearly stated that "RNA therapeutics-that's the future of medicine."

Fierce Biotech reported that many pharmaceutical giants are and will continue experimenting on the development of new RNA-based medicines. Most recently, Arrakis, an RNA therapeutics-based pharmaceutical startup, was set up to focus on the developemnet of new and better RNA medicines for the treatment of acute and chronic diseases. Michael Gilman, ex-CEO of Padlock Therapeutics, who will be the CEO of Arrakis, said, "We view the RNA world as a vast and potentially forbidding source of powerful new medicines for humanity." Also, the new company will work towards mining these RNAs in a systematic manner.

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