AT THE Hilton Hotel in Chicago, Professor Claude Wischik seems slightly fazed by all the fuss. In the last few days, the unassuming scientist has found himself at the centre of a feeding frenzy more commonly associated with actors and rock stars than academics.
Ever since he told an international conference on Alzheimer's that the team he leads at Aberdeen University had made a major breakthrough in the treatment of the disease, the avid sailor and hillwalker has been caught up in a giddy whirl of intervi
ews and press conferences. News that he and his researchers had developed a drug that appeared to halt the progress of the degenerative brain disorder offered fresh hope to millions of sufferers and their families, and propelled Wischik into the limelight, painting him as a medical saviour.
The sudden celebrity seems to sit uneasily on the shoulders of a man who has spent almost his entire career steadfastly working towards this point. Like most medical breakthroughs, the development of this apparent wonder drug is the result not of one Eureka moment, but of two and a half decades of painstaking research spanning several continents. It is a story not of instant glory, but of sporadic triumphs and major setbacks; of patience, unrelenting graft, faith in science and lifelong commitment to a single goal.
"There were many times when I would have liked to have walked away from the whole thing," admitted Wischik. "There were frustrations; times when solving a particularly knotty problem would take three years. But when it was particularly bleak and dark, this simple thought drove me on: knowing what I already know, do I have any right to turn back?
"When you are doing research, it is like rolling a dice. But you ask yourself, 'Is the science good? Is my thinking right or not?' If it keeps coming back yes, then you know you've got to keep on going."
Wischik has spent 24 years studying the neurofibrillary 'tangles' that first destroy nerve cells critical for memory and then neurons in other parts of the brain in those suffering from Alzheimer's. As a result, he, his team of scientists and TauRx Therapeutics – a Singapore-based company spun out of Aberdeen University – developed Rember, a treatment that dissolves the tangles and seems to halt the disease in its tracks.
In recent trials, patients taking a daily capsule experienced an 81% reduction in cognitive decline over a 12-month period.
Better still, patients who took it for 19 months appeared to experience no significant decline at all. Rather, they had felt more confident and better able to cope with daily life, consultant psychiatrist Dr Donald Mowat revealed.
Last week, the trial findings were hailed by some as the biggest breakthrough in the fight against Alzheimer's since 1907. The scientists involved hope that – eventually – doctors will be able to diagnose the onset of the disease and treat it before patients develop any symptoms.
Seeing 24 years of endeavour finally come to fruition must be incredibly rewarding. But Wischik's response has been muted, partly because he knows there is still much work to be done. Funds – to the tune of $200m (£101m) – have to be raised before phase three trials, on a much larger number of patients, can begin. At best, the drug will be available to Alzheimer sufferers in 2012, and it could take even longer.
In the meantime, Wischik knows he has become the unwitting custodian of sufferers' hopes and fears. "This is not about what a great job I have done. It's about the patients and what we can do to help them," he said.
"And there are so many desperate people out there. In the last few days alone, I have received hundreds of e-mails from people asking for them or their loved ones to be allowed to participate in future trials – even though we are not ready to recruit and even when we do there are procedures that have to be followed.
"At the moment, yes, we can offer sufferers hope, but hope is not enough. What people want is a pill they can take to improve their lives."
Talking to Wischik about his research, you might gain the impression of a hard-working but slightly intense man, with few interests outside science. But a look at his website – where academic papers sit alongside photos of him holidaying in exotic locations such as China and Alaska – reveals an exuberant personality and a lively sense of humour. There's a list of saints with strange claims to fame, such as St Christina the Astonishing, who apparently rose up during her own funeral service, levitated and berated the assembled congregation for their sinful ways. And the family website has pictures of dancing hamsters and a rundown of what happens during their Polish family Christmas. This is a man with a hinterland.
When it comes to the fight against Alzheimer's, however, it is clear Wischik has always been single-minded. Born in France and brought up in Australia, his association with the disease began when he won a scholarship to Cambridge to work as what he calls a "research slave" under the direction of eminent scientists Sir Martin Roth and Sir Aaron Klug.
Back then, remembered Wischik, Alzheimer's was seen as a Cinderella field, far less sexy than the likes of immunology.
Using techniques for which Klug won the Nobel prize, Wischik began studying the structure of the tangles – scientifically known as paired helical filament (PHF) – identified by Alois Alzheimer at the beginning of the 20th century.
He discovered first that the tangles were in fact clusters of a protein called tau, and later that it was possible to dissolve them with compounds that became known as Tau Aggregation Inhibitors.
Over the years, Wischik and his team developed a novel form of methylthioninium chloride (now trademarked as Rember), which seemed to block abnormal tau aggregations without interfering with the protein's normal function.
They also discovered that tangles of tau form in Alzheimer's patients up to 20 years before symptoms develop, raising hopes that a treatment could one day be used to prevent rather than treat the disease.
Working with Professor Franz Theuring at the Charite Hospital in Berlin, they demonstrated the drug reversed tau pathology – and cognitive decline – in transgenic mice.
By the time Wischik moved with his team to take up a chair in mental health at the University of Aberdeen in 1997, the drug had already been patented for the treatment and prevention of Alzheimer's in the US and the European Union. It was another five years, however, before TauRx Therapeutics was founded to help push the research forward.
In addition to their work on Alzheimer's, the scientists are looking at how Rember might also work on the synuclein fibres in the brains of those with Parkinson's disease.
Last week, Alzheimer patients who took part in the phase two trials told how Rember was already improving their lives. Jimmy Hardie, 72, from Boddam, near Peterhead in Aberdeenshire, was diagnosed with Alzheimer's in 2005 after he began to suffer "blank" periods and could not remember what he was about to do. He used to put sugar mistakenly in the fridge and suffered mood swings.
But since he started taking Rember in 2006, his confidence has returned, said his wife Catherine. "Two years ago if Jimmy had gone to his shed, he may have forgotten what he was about to do," she said. "Now he is able to plan what he wants to do, go and get the tools that he needs, and do the task. It is very encouraging."
Sandra Sutherland, 61, had been struggling to focus on her job in accounts for several years when she was diagnosed with Alzheimer's. She tested positive for the disease in 2005 as doctors investigated another medical condition.
Sutherland, who lives with her husband and two sons in Aberdeen and enjoys crosswords and gardening, believes Rember has helped her. "Since I've been on the trial, I feel more confident, more positive. I think my concentration has levelled off and not got any worse," she said.
Alzheimer's charities also welcomed Wischik's report, but cautioned that phase three trials involving a lot more patients were required before the efficacy and safety of the drug could be properly established.
They do not want to raise false hopes, but at the same time they are conscious that unless progress is made, and soon, the developed world will be struggling to cope with its dementia problem (just over half of those suffering from dementia have Alzheimer's).
There are currently 62,500 people with dementia in Scotland, and 700,000 in Britain as a whole. It costs the UK upwards of £17bn a year (£1.4bn in Scotland), more than heart disease, strokes and cancer combined. This is because dementia sufferers – most of whom live in the community – need such a high level of care.
And things are going to get worse. According to World Health Organisation figures, by the year 2050 there will be more than one billion people aged 65 or over, with Wischik estimating that about half will have tau tangles.
Professor June Andrews, director of the dementia services development centre at the University of Stirling, said a breakthrough treatment is desperately needed.
"The thing about dementia is that wherever you live in the developed world, you have the same chance of getting it. If you're over 90 the risk is 30%, so if the number of people over 90 doubles, so does the number of people suffering from dementia."
Andrews said Wischik's research is the most promising news she has heard in a long time. "Of course, tests are needed on a much larger number of people to look at possible side effects, but it does seem that they think they are really onto something here.
"This would be great news in social terms, because a reduction in the number of sufferers would allow us to concentrate resources on providing higher-quality care for those who continue to have dementia. But, to be honest, the best thing about the reports last week was to hear those who had taken part in the trials talking about how Rember had improved their quality of life.
"When you consider what Alzheimer's sufferers at the beginning of their journey have to face – the knowledge that their condition will go on deteriorating until it affects their ability to do almost every little thing – then the possibility that this may no longer be the case is, in human terms, pretty amazing."
