Impacts of CIHR-funded research: Neurodegenerative Diseases
Preventing blindness
Alzheimer's drug could work for glaucoma
Overview
Glaucoma is the second-leading cause of blindness in Caucasians in North America and the leading cause among African-Americans. In Canada, one per cent of people over 40 suffer from the disease. Dr. Adriana Di Polo of the University of Montreal looked at whether a drug approved in both Canada and the United States for treating Glaucoma could be used for treating Alzheimer's disease. She found that the drug is extremely effective at protecting neurons and function. The drug, marketed under the name Reminyl, was tested in an animal model in which glaucoma acts much as it does in humans.
Impact
Dr. Di Polo and her team have a patent on the use of Galantamine for glaucoma and other optic neuropathies. They are in conversation with companies who could help test the drug in humans. Since the drug is already approved for human use, physicians can prescribe it to their patients for glaucoma, known as "off-label" use.
First Published
CIHR Health Research Results, 2005-06; updated 2009
Stopping Alzheimer's in its tracks
Diabetes compound proves effective in blocking beta amyloid
Overview
The loss of memory and other functioning in Alzheimer's disease is caused by the action of a protein called beta amyloid, which destroys brain cells. It is generally only possible to treat the symptoms of the disease. But a discovery by two researchers from the University of Alberta offers an opportunity to prevent or delay the progression of Alzheimer's disease by stopping the action of beta amyloid. Drs. Jack Jhamandas and David MacTavish have found that a compound developed to prevent destruction of insulin-producing cells in diabetes is also effective in blocking the pathways through which beta amyloid destroys brain cells, improving the cells' chances of survival.
Impact
The work undertaken by Drs. Jhamandas and MacTavish suggests that the drug they were testing is effective in human tissue samples as well as in animal models. They continue their work in this area with continuing support from CIHR.
First Published
CIHR Health Research Results, 2004-05; updated 2009
A genetic clue to common form of dementia
Frontotemporal dementia strikes people under 65
Overview
Frontotemporal dementia attacks the frontal and temporal parts of the brain that are responsible for a number of functions including reasoning, planning, perception and memory. It typically strikes people between the ages of 40 and 70. Drs. Ian Mackenzie and Howard Feldman of the University of British Columbia and the Vancouver Coastal Health Research Institute have discovered that a mutation in a gene called progranulin causes an inherited form of the disease – a finding that came as a surprise to them.
Impact
A treatment arising from this discovery is still elusive. However, the researchers are now able to measure progranulin protein levels in blood, which opens the door to more accurate diagnosis of sub-types of dementia and improvements in monitoring treatment response. There are also some candidate drugs that could restore progranulin expression.
First Published
CIHR Health Research Results, 2005-06; updated 2009
Enlisting immune system cells to eat plaque
Bone marrow stem cells help produce microglia
Overview
Alzheimer's disease is characterized by the formation of plaques in the brain. A team led by Dr. Serge Rivest from Laval University has discovered a new way to fight the plaques. The team used bone marrow stem cells to produce immune cells known as microglia. These cells were able to digest the plaque created by Alzheimer's disease. People with the disease have microglia, but in their natural form, they are unable to eliminate the plaque. The research, conducted in mice, demonstrated increased numbers of microglia and stabilization of cognitive decline, indicating that targeting these immune cells could have potential for treating Alzheimer's disease.
Impact
Dr. Rivest and his team are looking for partners to test the finding in people with mild cognitive impairment, to gain clinical data to support the work.
First Published
CIHR Health Research Results, 2005-06; updated 2009
Uncovering the genetic roots of diseases
Translating findings into treatments
Overview
In the years since the mapping of the human genome, research has held out tantalizing possibilities of understanding and treating genetic disorders. Dr. Michael Hayden of the University of British Columbia is best known for his groundbreaking research on Huntington disease that most recently has provided evidence of a potential novel treatment for this devastating neurodegenerative disease. He has also made important discoveries about the role genes play in coronary artery disease and adverse drug reactions. Dr. Hayden has co-founded three pharmaceutical companies to commercialize his discoveries. One, Aspreva Pharmaceuticals Inc., tests existing medications as potential treatments for people suffering from rare and overlooked diseases. In 2008, Dr. Hayden was named Canada's Health Researcher of the Year.
Impact
Dr. Hayden's research on Huntington's disease has led to the development of a predictive genetic test for the disease. Xenon Pharmaceuticals, Inc., one of the companies he has co-founded announced in June 2009 a strategic alliance with Merck & Co., Inc., to discover and develop novel candidates for treating heart disease. Other drug development products include ones for pain, obesity, iron overload and anemia.
First Published
CIHR Health Research Results, 2003-04; updated 2009
The genetic basis of Alzheimer's disease
Gene discovery leads to drug development
Overview
Some types of Alzheimer's disease, particularly those that feature early onset of the disease, have their basis in genes. Dr. Peter St. George-Hyslop of the University of Toronto has discovered two genes that cause the early onset of Alzheimer's disease. The genes were found to be a component of an enzyme that generates amyloid beta peptide, the feature that makes up amyloid plaque, a principal feature of the disease. He has developed a drug to prevent the onset of the disease and halt its progress after it strikes.
Impact
The genes discovered by Dr. St. George-Hyslop have fundamentally changed how scientists think about the origins and development of Alzheimer's disease. And the enzyme found to contain the genes is now the target of a number of compounds to inhibit the enzyme, some of which are in human clinical trials. Among them is a compound developed by Dr. St. George-Hyslop and his team called scyllo inositol which has been shown to be successful at inhibiting Alzheimer's disease in a mouse model and is now in phase 2 clinical trials with a Canadian biotech company called Transition Therapeutics.
First Published
CIHR Health Research Results, 2003-04; updated 2009
A sweet solution to Alzheimer's disease
Sugar-like substance halts disease in mice
Overview
A team of researchers from the University of Toronto, led by Dr. JoAnne McLaurin, has identified a drug that can halt Alzheimer's disease in mice. The drug, a sugar-like substance known as scyllo-cyclohexanehexol, blocked the accumulation of amyloid beta, which, if unstopped, kills brain cells and triggers the formation of the plaques that are characteristic of Alzheimer's disease. Dr. McLaurin has received permission from Health Canada to proceed with human trials of this promising new drug.
Impact
Clinical trials of ELND005, as it is now known, are underway, run by Toronto-based Transition Therapeutics, Inc., in collaboration with Elan Pharmaceuticals. Phase I trials have been completed and Phase II trials are underway, slated to end in the second quarter of 2010. The U.S. Food and Drug Administration has given ELND005 fast-track designation to facilitate review of the drug.
First Published
CIHR Health Research Results, 2006-07; updated 2009
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