A man with an unusual form of motor neuron disease experienced an improvement in his symptoms after receiving a drug designed to target the specific genetic mutation that causes his illness. One year after treatment, the patient reported that he is still doing well.
According to the journal *Nature*, researchers say the initial results are exciting and lay the groundwork for more treatments for neurodegenerative diseases caused by rare mutations.
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The man suffered from a slowly progressing form of motor neuron disease, also known as amyotrophic lateral sclerosis (ALS), which is caused by a rare mutation that leads to the accumulation of proteins contributing to the death of motor neurons.
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He received an RNA therapy called “antisense oligonucleotide therapy.” Unlike standard gene therapy, which modifies a person’s genes to treat a genetic disease, this new therapy uses short strands of genetic material to target the RNA produced by the gene and reduce the amount of protein produced.
Steve Vucic, a neurologist and ALS researcher at the University of Sydney in Australia, said the results represent an “exciting first step,” although it is too early to know whether the treatment could halt the progression of the disease or serve as a cure.
This will require monitoring the trial participant for another two or three years and testing the drug on more people, he noted.
Source: KYPE
