The new drug reduces toxic proteins in people with Huntington's disease

is a deadly genetic neuropathy. On May 6th data on a drug expected to treat Huntington's disease was published in the New England Journal of Medicine, which blocks mutations in proteins that cause brain damage in Huntington's patients.
results were first published in December 2017, and an editorial described the trial as a ground-breaking breakthrough. The synthetic short-chain DNA drug, called HTTPRx, could stop the production of the mutant Huntington's protein, according to a new paper. None of the 46 subjects involved in the trial reported any serious adverse events, indicating that the drug was safe for humans.
paper also provides exciting details behind the experiment: HTTRx lowers levels of the mutated Huntington protein in the cerebrospinal fluid, reversing hunting-like motor and cognitive symptoms in mice. The reduction of mutant proteins in the patient's cerebrospinal fluid was dose-dependent: the larger the dose, the greater the reduction of mutant proteins within a certain dose range.
However, the authors report that taking into account data from five groups of patients treated with different doses, the symptoms of the disease remained generally unchanged and that "no meaningful difference was found between patients treated with placebo and patients treated with HTTPX, regardless of dosage levels".
now, all eyes are on a key clinical trial aimed at recruiting 660 people with Huntington's disease. The first patient was registered in January and the last patient data is expected to be collected in March 2022. The study was large enough and time long enough for scientists to measure the drug's effects on Huntington's disease, and to show whether the drug slowed or halted the development of Huntington's disease
(Source: Xu Shaoliang, China Science Daily)