The results showed the drug lowered the level of the toxic disease-causing protein in the nervous system and did so safely.
According to The Guardian, the phase one trial included 46 men and women with early onset Huntington's disease, who were given four spinal injections, one each month, with an increasing dose each time.
Ionis senior vice president of research Frank Bennett said the protein reductions observed in the study "substantially exceeded our expectations" and that the drug was also well tolerated.
Alzheimer's and Parkinson's are both caused by buildups of harmful proteins in the brain; if a similar technique could be used to stop the production of these proteins, it could slow or cure these diseases (which are far more common than Huntington's).
The Swiss pharmaceutical giant Roche paid a $45 million license fee to take the drug forward to clinical use.
The researcher noted, however, that a much larger, longer trial is needed to show that IONIS-HTTRx can slow the progression of Huntington's disease.
Now treatments only exist for the symptoms of Huntington's, which usually begin to appear between the ages of 30 and 50, leading to their gradual decline over the next 10 to 25 years.
Huntington's Disease is a inherited condition in which nerve cells in the brain break down over time.
The error is found in the huntingtin gene that tells the body to produce the huntingtin protein.
"What we have seen is a really strong, really encouraging piece of chemical evidence that the drug is doing what it's supposed to do, which is lowering the level of the toxic protein in the spinal fluid where it was injected".
Roughly a quarter of participants formed the control group and were given a placebo injection. "This is presumably the most noteworthy crossroads in the historical backdrop of Huntington's since the quality [was isolated]".
The new drug, developed by Ionis Pharmaceuticals, contains an "antisense" molecule consisting of a single strand of chemically-modified DNA. "One day we want to prevent the disease".
They created a snippet of synthetic DNA that messes with the messenger RNA responsible for translating DNA protein codes, effectively blocking the problematic huntingtin protein from every being made.
There is no treatment, but Russell Snell of the Centre for Brain Research said they had identified raised levels of urea in the brain of a transgenic sheep model that matched human brains affected by Huntington's.
A comparison of protein levels before and after the treatment showed "significant" drops after IONIS-HTTRx was administered, UCL said in a press release, suggesting the drug had successfully intercepted the rogue gene.
"Alzheimer's and Huntington's are at opposite ends of the dementia spectrum - so if this holds true for these types, then I believe it is highly likely it will hold true for all the major age-related dementias", Cooper said, in the paper published in the Proceedings of the National Academy of Sciences.
However, Miami Beach is hosting the Art Basel global art fair this weekend, which draws celebrities from around the world. According to TMZ , which obtained an arrest affidavit from the incident, Buress asked an officer to call him an Uber.
It's enough to get by, but saving is still a struggle, even with the university waiving about $12,000 of his tuition costs. A bachelor's degree yields, on average , a 15 percent return, although that percentage will likely decrease.
MISSING CHILD Alert issued for Caitlyn Frisina , W/F, 17yo, 5'7", 135lbs, BR hair & eyes, from Lake City. The department also says they've received tips about the pair being in North Georgia and in SC .
He had called the chancellor to complain about "inacceptable" leaks to the press, the Social Democrat leader said. The message is "simply wrong", said Martin Schulz at a press conference at the SPD headquarter.
South Australia, of course, is no stranger to power outages, as blackouts left much of the area without power last summer. Tesla said it hopes the project "provides a model for future deployments around the world".
In a statement today, British Airways said: " Heathrow is continuing to experience severe adverse weather conditions. Glasgow and Aberdeen Airports were also reporting cancellations to several flights heading to England and Amsterdam.
This was due to a government panel finding that the hospital did not adhere to norms regarding newborn infants. Jain also mentioned that, "Max Hospital is a habitual offender and three notices were served earlier to it".
Under EU rules, 16 favourable votes are needed as a "qualified majority" for renewal before authorisation expires on December 15. Banning glyphosate outright would have shaken Europe's agriculture sector, since it is so widely used.
Previous U.S. administrations have failed to stop North Korea from developing nuclear weapons and a sophisticated missile program. A photo released by North Korea's official Korean Central News Agency (KCNA) shows launching of the Hwasong-15 missile.
Trump to record robocall for Roy Moore
Others were Trump supporters eager to see the president in person. "They're resisting the will of the American people", he said. At that time, according to Nelson, Moore was pursuing a sexual relationship with her and had groped and tried to rape her.
Bayonetta 3 will be a Switch exclusive, Nintendo reveals
Nintendo has also confirmed during The Game Awards that Bayonetta 1 and Bayonetta 2 bundles are also arriving on Nintendo Switch . Buying the digital version of Bayonetta will cost you $29.99/ €29.99/ £29.99 and then Bayonetta 2 will run you $29.99/ €29.99.
Embattled Michigan Rep. John Conyers announces retirement
Following these accusations, House minority leader Nanci Pelosi eventually asked Conyers to step down, as did Rep. Conyers, who was first elected in 1964, easily won re-election past year in the heavily Democratic 13th District.
Tiger Woods finishes 9th on comeback
It was Woods' first competitive tournament since February and a fourth back surgery - a spinal fusion - in April. World No. 1 Dustin Johnson finished in a tie for 14th at 288 after a final-round 72.
The best of Herm Edwards' introduction as ASU's head coach
Edwards also offered up this gem as he paraphrased former Arizona State star Pat Tillman: "Football ignites my soul". State in the Sun Bowl on December 29. "Devils Digest, huh?" said Edwards, who asked where his business was located.
Mega Man X Collection and Mega Man 11 Announced
Capcom said that the first collection will also get an update that allows players to rewind gameplay if they make a mistake. Indeed, even Mega Man afficionados will likely find some trivia and development stories that they haven't heard before.
Google Assistant can now locate a handiman near you
Those include Lorde's Royals and Jayden Smith's "Icon" (some seem to think the crash is well-deserved for that last one). Whatever your reason for using the Home Mini for playing music, don't do it at high, and especially maximum, volume.
Flynn plea raises questions on how Trump could react
Earlier in February, former FBI director James Comey said that Trump asked him to stop the investigation into Flynn. Comey's conference room for a final briefing: Only about 3,000 emails had been potentially work-related.
Jury: Mexican man not guilty in San Francisco pier killing
During the presidential race, then-candidate Donald Trump cited the killing as a reason to toughen US immigration policies. Under local sanctuary city rules, local law enforcement won't hold a person beyond the term of their incarceration.
Why is Trump calling Elizabeth Warren Pocahontas?
Putting a hand on the shoulder of one of the two veterans, he said: "But you know what, I like you because you are special". Q: At the event that the president just did with the Navajo code talkers, he referred to Pocahontas being in the Senate.