Copyright 2004 The Christchurch Press Company Limited
The Press (Christchurch, New Zealand)
May 29, 2004, Saturday
SECTION: NEWS; NATIONAL; Pg. 4
LENGTH: 1671 words
HEADLINE: Disease victim fights for life Rilutek and MND
BYLINE: MARTIN Yvonne REID Shirley
People afflicted with Motor Neurone Disease (MND) face a creeping death that can only be slowed by an expensive and unsubsidised drug. YVONNE MARTIN speaks to a nurse who has become a patient herself.
For Nelson nurse Shirley Reid menial tasks like cooking meals, washing dishes and ironing are no longer mundane. They are signs of independence to be cherished as long as humanely possible. Three years from now she may need a cast of caregivers to direct her daily routines.
Reid, 56, was diagnosed with the debilitating and deadly Motor Neurone Disease (MND) last August.
Diagnosis usually carries a three-year death sentence, but the disease acts differently in every patient.
Millions of nerve cells or motor neurones in Reid's spine are dying off quicker than they can be replaced. Starved of nerve stimulation, her muscles are gradually weakening and wasting.
Walking is now an effort and she uses a frame or stick to get around. She still has good upper arm movement, but for how long she cannot know.
MND is slowly and silently invading her body, causing irreparable damage.
"Its progression is insidious," said Reid. "It's reasonably slow, but when you look back a year ago I didn't have to use a stick."
The statistics surrounding MND or amyotrophic lateral sclerosis are grim.
About 300 New Zealanders are affected at any one time and it kills an average of 87 a year. Usually their breathing becomes so difficult they lapse into a coma and die.
"MND is the worst disease in medicine," said University of Sydney neurology professor John Pollard.
"It's a disease in which the muscles waste, including the muscles of breathing and swallowing. People who are fully conscious and whose minds are completely unimpaired realise that they are finding it difficult to breathe.
"They face gasping for breath and being unable to get enough air and that is terrifying."
They also lose the ability to swallow, said Pollard.
"At least in the early stage you can enjoy food. But then that becomes difficult and the food goes down into your lungs. It's a horrible thought. Then you waste away and cannot move your arms or legs."
Famed British cosmologist Professor Stephen Hawking, 62 -- whose book A Brief History of Time sold millions worldwide -- is imprisoned in his body with a form of MND. All his movement is concentrated in the one finger he uses to activate his computerised voicebox.
"He only survives because he has so much money that he can afford to have a nurse looking after him and all that expensive equipment," said Pollard.
"For most people it is a death sentence and the average lifespan from diagnosis to death is three years."
Money is also determining how long MND patients survive in New Zealand.
The only known drug to stem the disease's progress, Rilutek, is available here, but not registered or subsidised by the Government's drug-buying agency Pharmac.
Patients on the drug can live better for longer -- anywhere from two months to more than 18 months, depending on which study you believe.
But only the affluent or the fortunate can afford the price tag, $ 883 for 28 days worth of tablets. The rest die sooner.
Had MND patients been born in Australia, where Rilutek is subsidised for certain patients, a month's supply would cost them $ 4.20 with a concession card, or $ 26 for others.
Rilutek, also subsidised in 15 OECD countries, is unlikely to be funded in New Zealand in the short-term.
The Pharmacology and Therapeutics Advisory Committee (PTAC) looked at Rilutek on Pharmac's behalf last year. It considered that the drug might extend a patient's life by "approximately two to four months".
There was a lack of evidence of improved quality of life. The committee recommended that Rilutek "provided a modest benefit at a disproportionately high cost".
It was "unlikely to be a cost-effective treatment in New Zealand", it said.
"Is the life of a MND sufferer in New Zealand not worth prolonging?" asked Reid's husband, John.
"It is yet another example of the cruel and devious way our health system operates. People die so that budgets can be met. Politicians set the rules and walk away."
The Reids have waged a campaign, through emails and letters to newspapers, urging Pharmac to reconsider and drug manufacturer Aventis Pharma not to give up. Aventis, which indicated an interest in supplying Rilutek, said it had been put off making a full application for funding by the committee's negative response.
"An application for Rilutek would take many hours to prepare and cost significant amounts of money," said Aventis's New Zealand business manager Andrew Baker.
"We do not believe, based on the feedback that we have received, that this exercise has any value whatsoever."
Reid, a former public health nurse, first noticed something was awry when she began cramping during a class two years ago. When cycling the Central Otago rail trail with friends she lagged behind and felt fatigued.
"A couple of days later we were staying at Karitane. I walked to the beach and felt my legs dragging," she said.
Reid spent three months trawling the internet trying to diagnose herself. A battery of tests eliminated all other diseases except the dreaded MND.
"In the end I told the doctor, 'I've come here expecting to be diagnosed with MND'. I made it kind of easy for him, I suppose," she said. Yet confirmation from her physician was still devastating.
"Everybody lives to be nearly 100 in our family and you assume you will too. The information pack that you get gives an average life expectancy of 30 months."
Reid's quest for answers took her to Pollard in Sydney, a renowned professor of neurology. He recommended Rilutek to stunt the disease's progress.
Reid, who is paying for the drug privately, believes it is slowing her inevitable downward spiral. Although it cannot repair the muscle damage in her feet, she believes it is keeping her arms mobile and her spirits high. She has had no side effects.
"You don't feel so helpless and you know you are doing as much as anyone in the world with MND," said Reid.
Neurologist Roger Pamphlett, a senior lecturer at the University of Sydney, said the drug does work.
"The first studies show that it increased lifespan by three months but now more recent studies indicate that it might be 11 or 12 months, which is quite good for something that kills you in three to five years," he said.
The longer survival time could be because people on Rilutek saw their doctor more frequently, meaning problems were picked up earlier. Quality of life studies were under way.
"It's very important for people to have hope as well. This is definitely something that works and that is very good for patients, physically as well as psychologically," said Pamphlett.
Doctors hoped other drugs could be found to further inhibit the disease's progress.
British scientists have also reported that they may have developed a gene therapy treatment that slows the onset and progression of a form of MND in mice. It extended life expectancy by 30 per cent.
Pharmac acting chief executive Cristine Della Barca said the PTAC would consider new data if it was submitted and could potentially change its recommendation, if justified.
Pharmac had a fixed budget of $ 541 million to fund 2500 medicines and related devices. It was considering another 50 proposals for funding.
"It's our job to look at the evidence and the benefits and the cost-effectiveness of all pharmaceuticals," said Della Barca.
"It does require pretty thorough analysis and consideration of their needs, in the wider context of how far we stretch a budget."
Another MND sufferer, Elizabeth Street in Wellington, said Rilutek was too expensive for consideration when she was diagnosed four years ago.
"I looked at all the research going on. There always seemed to be a solution around the corner. But four years on, there isn't," said the former librarian.
Street believes Rilutek should be subsidised so patients can have a choice early on.
"Shirley and John have got the guts and the gumption to follow this through.
"And they still have the energy to do it," she said.
THE DISEASE
MND covers a group of conditions in which nerve cells called motor neurones die off. Motor neurones tell muscles what to do. With no nerves to activate them, muscles gradually weaken and waste.
MND is also known as amyotrophic lateral sclerosis (ALS) or Lou Gehrig's disease.
Symptoms include:
twitching
cramps
general fatigue
tightness and spasms of the arms and legs.
The disease affects people differently and its rate of progression varies. It results in creeping paralysis, difficulties in speaking and swallowing, and eventually death.
About 300 New Zealanders have MND. Average life expectancy after contracting the disease is three to five years. About 87 people die of MND each year. There is no known cause or cure.
THE DRUG
Rilutek is a benzothiazole which works by delaying the onset and progression of symptoms of MND.
Esitmates on how long Rilutek extends life vary. The drug's manufacturer, Aventis Pharma, claims three to four months is average. Some patients get an extra 12 to 18 months of life on Rilutek.
The longer a MND patient is symptom-free, or the slower the progress of the disease, the better their quality of life.
Some patients are paying for Rilutek privately. The unsubsidised drug costs $ 883 for 28 days of medication. In Australia, where the drug is funded, it costs $ NZ4.20 a month for concession cardholders and $ 26.21 for other patients.