MONTREAL (CP) - A new drug cocktail to treat Lou Gehrig's disease could slow the incurable illness, but requires more research money before human trials can begin, researchers said Tuesday.
McGill University scientists said they need at least $250,000 to begin testing the cocktail on Canadians afflicted with the neurodegenerative disease, also known as ALS.
ALS is sometimes called Lou Gehrig's disease, named after the New York Yankees baseball legend who died of the disease in 1941.
The researchers believe the cocktail, consisting of three commonly used drugs, could extend patients' lives.
They said mice that were fed the drugs last year lived up to 20 per cent longer than mice on a regular diet. ALS symptoms, such as muscle deterioration, also developed more slowly in the mice that received the cocktail.
Dr. Jasna Kriz, one of two researchers who developed the treatment, said human trials must begin as soon as possible to help ALS patients, 90 per cent of whom will die within five years of their diagnosis.
"This is a deadly disease," Kriz said after a news conference introducing the experimental treatment.
"Once you have a diagnosis, you know within two to five years you will be completely paralysed or dead."
Researchers hope to test the drug cocktail within three months on ALS patients in Montreal, Edmonton, Vancouver, Halifax and London, Ont.
ALS, or amyotrophic lateral sclerosis, kills the body's motor nerves, gradually causing paralysis and the inability to speak or swallow.
About 2,000 Canadians a year are diagnosed with ALS.
The drug cocktail, developed at McGill by Kriz and Dr. Jean-Pierre Julien, contains three commonly used drugs:
- Minocyline, an antibiotic used to treat acne.
- Riluzole, a drug already used to treat ALS.
- Nimodipine, a drug that blocks calcium channels and is normally used to treat brain hemorrhages and migraine headaches.
Used together, McGill researchers determined the drugs protected the nerve cells of mice from the massive inflammation that accompanies the onset of ALS symptoms.
But there are no guarantees the cocktail's beneficial effects on mice will be transferable to humans, say researchers.
The experimental treatment was published Tuesday in the medical journal Annals of Neurology.
Dr. Angela Genge, director of the ALS clinic at the Montreal Neurological Institute, told the news conference funding hasn't yet been finalized for the second stage of research on humans.
Researchers hope to get money from the Canadian Institutes of Health Research, a federal agency responsible for funding biomedical research in Canada.
The agency contributed $1.5 million to last year's animal trials, along with additional funding from the ALS Society of Canada and the Muscular Dystrophy Association of Canada.
ALS sufferer Neil Rubin, who was at the news conference, said Tuesday he would jump at the opportunity to participate in clinical trials.
The Montreal electronics employee was an avid squash player for many years, but now can hardly walk as his ALS symptoms worsen.
Rubin, 54, said his diagnosis didn't appear as quite as bleak once he heard news of the breakthrough.
"It's very encouraging, very optimistic," said the father of three.
"There are a lot of people out there, hoping, waiting, and I'm one of them."
Rubin said he's prepared for the possibility the drugs might not work on humans.
"We have to play the cards that we're dealt and do the best that we can," said Rubin.
"There's always tomorrow."
© Copyright 2003 The Canadian Press
Canadian scientists have discovered a drug cocktail to treat ALS, or Lou Gehrig's Disease, that they believe could slow the incurable illness and add up to 10 years to patients' lives.
The experimental finding, published today in the journal Annals of Neurology, is expected to be tested in human patients by late summer.
"We could have a preliminary answer within a year," said neuroscientist and senior author Jean-Pierre Julien of McGill University Health Centre in Montreal.
"Right now, ALS is a disease with very little treatment," Dr. Julien said. "If I had the disease, I wouldn't hesitate to take this treatment."
Dr. Julien and collaborator Jasna Kriz tested three drugs: minocycline -- an antibiotic with anti-inflammatory properties; riluzole -- the traditional ALS drug; and nimodipine -- a drug that blocks calcium channels and is normally used to treat brain hemorrhage and for prevention of migraine headaches. All three drugs are on the market but had not been tested in combination for ALS.
Dr. Julien said the cocktail worked brilliantly, extending the lives of experimental mice with ALS by up to 20%. The treated animals were much more active and stronger than the untreated mice.
"The results are very impressive," said Dr. Angela Genge, director of the ALS clinic at the Montreal Neurological Institute and Hospital.
Until recently, scientists have been obsessed with the notion of treating disease
with a single drug or therapy. But the groundbreaking success of AIDS drug cocktails has launched a wave of studies looking at multi-drug treatments for complex diseases such as ALS.
"We would like to have the magic bullet to cure diseases, but in real life, it's probably unrealistic," said Remi Quirion, scientific director of Canada's Institute of Neurosciences, Mental Health and Addiction.
Dr. Quirion said the approach used in Montreal could also be used to treat Alzheimer's disease, muscular dystrophy and multiple sclerosis. "The impact of the three-drug therapy in AIDS has shown that we have to go that route, perhaps more often than not, when treating complex disease," Dr. Quirion said.
Last year, Dr. Julien's lab reported that minocycline -- a common antibiotic -- could by itself slow the onset of ALS. Several clinical trials of the drug are ongoing and scientists say results look good. But Dr. Julien said the three-drug treatment appears to be considerably more effective and he hopes researchers begin clinical trials on it soon.
Susan Graham Walker, spokeswoman for the ALS Society of Canada, said researchers will have no difficulty finding patients for testing. "Anybody with ALS will do anything," she said. "They will have far more people than they can accommodate."
The research was funded by the Canadian Institutes of Health Research in conjunction with the ALS Society of Canada and the Muscular Dystrophy Association of Canada.
In a complementary finding, a team of Italian researchers yesterday reported that stem cells taken from bone marrow helped to regenerate damaged nerve tissue in mice with the animal version of ALS.
"The central nervous system of ALS mice showed a fivefold increase of newly generated neuronal cells with respect to the healthy mice used as controls," said University of Milan researcher Stefania Corti.
"A high level of regeneration in heart and skeletal muscle was also found."
Dr. Julien said the stem cell treatment and his combination drug therapy could work together.
"Eventually, we hope that a combination of these two different targets will dramatically slow down this disease in humans," he said.
