For most of her life Paula Meir has lived with the knowledge that she could have inherited a fatal disease for which there is no cure.

For most of her life Paula Meir has lived with the knowledge that she could have inherited a fatal disease for which there is no cure.

It killed her mother and brother and influenced every decision Paula has made in life, including whether or not to have children of her own.

In April, when I interviewed Paula for an article about her newly-published book, Your Life Your Way, a practical guide to managing life's problems shot through with wisdom from her own experience, she told me her father had advised her many years ago that she shouldn't let fears about Huntington's disease stop her becoming a mother if that's what she wanted. Science would no doubt find a cure by the time it mattered, should the worst happen.

Paula, who lives in Norwich, went ahead and had her children, who are now young adults, but, she told me eight months ago, there was still no cure for Huntington's.

At the start of last week though, a story hit the headlines that changed everything.

Results from a ground-breaking drug trial offered new hope to people with Huntington's disease.

The experimental drug, injected into spinal fluid, was shown to lower levels of the harmful protein responsible for the devastating inherited brain disorder.

IONIS-HTTRx is the first pharmaceutical agent designed to target the root cause of Huntington's.

A total of 46 patients with early-stage Huntington's disease took part in the trial, conducted at nine centres in the UK, Germany and Canada.

The study confirmed that the treatment worked as hoped.

Lead researcher Professor Sarah Tabrizi, head of University College London's Huntington's Disease Centre, said: 'The results of this trial are of ground-breaking importance for Huntington's disease patients and families.

'For the first time a drug has lowered the level of the toxic disease-causing protein in the nervous system, and the drug was safe and well-tolerated.

'The key now is to move quickly to a larger trial to test whether (the drug) slows disease progression.'

Paula, who is now 47, said news of the drug trial had left her feeling very emotional.

'When you have lived for 32 years hoping for a cure and it was only 20-odd years ago that the gene responsible for causing this disease was even identified, this is a big deal. It is a huge relief that there is hope for all those who are affected. We can't say this is a cure yet but even if it stops the progression that is a massive step forward.'

Paula's mother died of complications caused by Huntington's disease, which damages nerve cells in the brain and progressively affects movement, thinking processes and behaviour. She saw her mother battle alcoholism, attempt suicide, sectioned and cruelly sent to an institution as the disease took hold.

Paula has chosen not to have the test that now exists for the Huntington's gene. One of her sisters has and is free of it. Another brother died of the disease. If you have the faulty gene, Huntington's tends to show itself around the same age it affected your parent. In Paula's case, that would be late 30s or early 40s

'Ironically, although living with not knowing has been incredibly challenging it was preferable to a definitive answer,' she says. 'But this (drug trial) might well change how people will view testing for the gene if it could lead to getting onto some kind of treatment programme. I don't know if it changes my attitude to getting tested. I would have to think about that. I would hope though, that having reached the age of 47 my chances of having the gene are reduced. This is a massive breakthrough but it doesn't mean the research work and need for funding is over.

'When my dad told me all those years ago: 'Don't worry, science will find the answer,' I did not think it would take another 32 years to come up with something.

'For a lot of people though, along with the joy and hope about this news there will be mixed emotions because of all the people whose illness is already too advanced and will never have the chance to benefit from it.'

A 'potential game-changer'

Huntington's disease is a progressive neurological disorder that normally appears in adulthood and causes involuntary movements, psychiatric symptoms, and dementia. It affects an estimated 10,000 people in the UK. A further 25,000 are thought to be at risk. Patients usually die within 20 years after the onset of symptoms.

The new drug, developed by Ionis Pharmaceuticals, contains an 'antisense' molecule consisting of a single strand of chemically-modified DNA.

The DNA strand stops the genetic instructions for making the Huntington's protein getting through to manufacturing machinery in brain cells.

Levels of the toxic HTT protein are reduced, thereby minimising damage to the brain.

Trial patient Peter Allen, 51, who lives in Chelmsford, told the BBC: 'You end up in almost a vegetative state, it's a horrible end.

'It's so difficult to have that degenerative thing in you. You know the last day was better than the next one's going to be.'

His mother Stephanie, uncle Keith and grandmother Olive, have all died from Huntington's disease. Tests show his sister Sandy and brother Frank will develop the condition.

Experts say there is a long way to go but it is possible the same 'gene silencing' technique could have potential in combatting other neurodegenerative disorders that feature the build-up of toxic proteins in the brain, such as dementia and Parkinson's.

Dr Philippa Brice, from the genomics research charity PHG Foundation, said: 'This is a potential game-changer, not only for Huntington's disease patients but also for genomic medicine in general. Though more work needs to be done, if gene silencing lives up to this promise we could be on the brink of some of the personalised treatments that patients with severe genetic diseases need so badly. But this will also raise further questions for society and for policy-makers about how best to use such a powerful technique.'