Huntingtons breakthrough may stop disease

[nitpicker]

http://www.bbc.com/news/health-42308341

Huntington’s breakthrough may stop disease
By James Gallagher
Health and science correspondent, BBC News

11 December 2017

The defect that causes the neurodegenerative disease Huntington's has been corrected in patients for the first time, the BBC has learned. An experimental drug, injected into spinal fluid, safely lowered levels of toxic proteins in the brain. The research team, at University College London, say there is now hope the deadly disease can be stopped. Experts say it could be the biggest breakthrough
in neurodegenerative diseases for 50 years.
(snip)

Huntington's is caused by an error in a section of DNA called the huntingtin gene. Normally this contains the instructions for making a protein, called huntingtin, which is vital for brain development. But a genetic error corrupts the protein and turns it into a killer of brain cells. The treatment is designed to silence the gene.

On the trial, 46 patients had the drug injected into the fluid that bathes the brain and spinal cord. The procedure was carried out at the Leonard Wolfson Experimental Neurology Centre at the National Hospital for Neurology and Neurosurgery in London. Doctors did not know what would happen. One fear was the injections could have caused fatal meningitis. But the first in-human trial showed the drug was safe, well tolerated by patients and crucially reduced the levels of huntingtin in the brain.
(snip)

Doctors are not calling this a cure. They still need vital long-term data to show whether lowering levels of huntingtin will change the course of the disease. The animal research suggests it would. Some motor function even recovered in those experiments.
(snip)

Peter, Sandy and Frank - as well as their partners Annie, Dermot and Hayley - have always promised their children they will not need to worry about Huntington's as there will be a treatment in time for them.
(snip)

He, along with the other trial participants, can continue taking the drug as part of the next wave of trials. They will set out to show whether the disease can be slowed, and ultimately prevented, by treating Huntington's disease carriers before they develop any symptoms.
(snip)

[The Wielding Truth]

1. Oh. I hope so. I hope this is a cure.

[Rollo]

2. sounds promising, but...

Won't this treatment require repeated regular spinal injections? Isn't there considerable risk associated with that sort of regimine, aside from the possible side effects of the drug?

[Canoe52]

3. KnR

[murielm99]

4. I know a family in my community

who have been affected by this cursed disease. Three generations!

As I understand it, people can opt to be tested at age eighteen, to see if they will develop the disease. Not everyone cares to be tested.

This treatment sounds very experimental. Many doctors tell people that the way to stop the disease is to opt out of having children. Having seen the effects of it in one family, I would concur.

[Cicada]

5. Some day a similar trick may help ALS, Alzheimers, Parkinsons and Trumpism