The Canadian Genetic Diseases Network promotes advances in genetic research, the training of Canada's best young researchers, and the creation of new and beneficial health care partnerships with Canada's premiere universities, medical centres, companies and the Medical Research Council.
The Canadian Genetic Diseases Network is a leading participant in the Federal Government�s Network�s of Centres of Excellence Program. This program, now in its sixth year, is a bold and imaginative approach to generating significant Canadian economic activity and benefits from our expenditures on first class scientific research.
The scientists within this Network represent a consortium of 38 of Canada�s leading genetic researchers at 12 universities, 9 hospitals and 11 core technologies facilities across the country. These scientists have repeatedly demonstrated that they are at the forefront in discovering and elucidating the fundamental mechanisms of genetically transmitted diseases. They have gained international recognition for this work, which has led to a much better understanding of the genetic basis of tragic diseases like Huntington�s, Cystic Fibrosis, Muscular Dystrophy, Alzheimer�s, breast cancer and diabetes.
Increasingly, the results of our researcher�s efforts are being sought out by industrial partners as well as investors in biomedical research. Network management, in close cooperation with the scientific teams, is now focusing on accelerating the pace of commercialization, which already has resulted in industrial partnerships and more recently several new commercial ventures.
The Network�s scientific program in the study of genes (�genomics�) is at the core of the exciting medical revolution currently underway. For most of the 20th century, medicinal chemistry drove the pharmaceutical industry. However, by the 1980�s molecular and structural biology were spurring new innovations that made it possible to understand the genetic basis of disease. Today, the driving force for new pharmaceutical discoveries to treat disease is the biological information derived from genomics research. In addition, the new field of gene therapy is being born and the Network will play an increasing role in this field as it develops.
Inevitably, these new opportunities also bring with them significant challenges, not the least of which is to maintain critical mass in emerging scientific disciplines such as bioinformatics and combinatorial chemistry, which will enable us to continue to apply the fundamental discoveries of genomics research, to the economic benefit of Canada.
As a country, we are in danger of falling behind in training scientists in these new fields, and the international competition for such people, both from industry and academia, is intense. Clearly the Network, because it is based on a unique partnership between government, industry and our universities, will ensure that we remain competitive and capitalize on the results of our own research investments.
I look forward to continued partnership with this dynamic Network and its group of researchers, physicians and managers to create the scientific and industrial infrastructure of the 21st century.
Canadian
Approximately 60% of Canadians will develop or die from a disease with a
significant genetic component. Over 50% of children admitted to paediatric
hospitals are admitted because of genetically-transmitted illnesses. The
scientists and physicians of the Canadian Genetic Diseases Network, located
at universities and medical centres across Canada, are making great strides
in research and have received national and international recognition.
Canadian industry and venture capital organizations are responding to these
scientific advances by forming Canadian partnerships and new ventures to
develop new diagnostics and therapies .
Genetic
Genetics is the transmission of biological information from one generation
to the next. Genes are units of biological information which are used by
cells and organs to determine the development and function of the human
body. Genetic variation among individuals means that each of us has a
unique genetic make-up that influences our susceptibility to disease.
Diseases
Genetic diseases are caused by changes or mutations in the body's genes.
These mutations may be inherited from previous generations or occur in the
cells of an individual's body. Single mutations in critical parts of genes
can lead to onset of disease. Combinations of genes are implicated in many
common diseases not usually classed as genetic, and genetic predispositions
can result in disease if triggered by environmental factors.
Network
A nationwide consortium of Canada's top investigators and core-technology
facilities in human genetics, partnered with colleagues from industry to
conduct leading-edge research within an "institute without walls". Core
funded through the Medical Research Council of Canada by the federal
Networks of Centres of Excellence program since 1990, the Canadian Genetic
Diseases Network is a unique R&D; initiative aimed at achieving international
competitiveness in scientific research with social and economic benefits.
At the forefront of research
In 1994-1995 Network investigators made ground-breaking advances in the
study of early-onset Alzheimer's disease , Huntington disease, juvenile
onset diabetes, spinal muscular atrophy, fragile X syndrome, and breast
and ovarian cancer.
Discovery of causative genes and how genes cause disease paves the way for developing diagnostics and therapies which will benefit current and potential sufferers of devastating genetic diseases.
Training opportunities for young scientists
Graduate students and post doctoral fellows in the Canadian Genetic
Diseases Network train alongside senior Network scientists who are leaders
in their fields.
The Network provides optimum training opportunities for the next generation of Canadian scientists, thereby contributing to a critical mass in genetic research and enhancing Canada's scientific competitiveness.
Economic growth, jobs
Partnerships and collaborations forged with industry lead to an efficient
diffusion of new technologies and improved healthcare products, and
enhanced research infrastructure.
Creation of a new industry receptor capacity through commercialization of Network technologies and attraction of international investments means benefits to the Canadian economy and high quality jobs.
Innovative healthcare
In times of limited healthcare resources, efficient and cost-effective
medical technologies are beneficial to both the user and the healthcare
system.
Research innovations lead to better medical care for diseases which currently have no effective therapies; such advances will improve the quality of life of our afflicted neighbors and fellow Canadians.
The first year of our renewal, 1994-1995, has resulted in stirring collaborative research which has explored new frontiers in medicine. The pooling of intellectual, physical and clinical resources has again shown that the whole is greater than the sum of the parts and has resulted in major scientific advances in a number of illnesses. The list of scientific achievements over this period is remarkable. Network scientists have discovered two genes that cause familial Alzheimer's disease, identified new genes involved in apoptosis, participated in the identification of genes in breast and ovarian cancer, identified the gene for Wilson's disease, participated in the discovery of the gene loci for diabetes, developed an animal model with gene targeted disruption for Huntington disease, developed new technology for protein sequencing, and for DNA sequencing, and established databases on mutations in populations used as models world-wide.
The Network has also played a major role in ensuring that these discoveries are commercialized to the benefit of Canada. We have been able to achieve these goals as a result of increased capability to address our fundamental mission. This includes the hiring of our Commercial Director, increasing collaboration among investigators, and pooling of resources. The changing fiscal environment for our Network, with decreasing budgets over the next few years will, however demand that we find new revenue sources for supporting our mission.
One way we are pursuing this goal is through the establishment of corporate entities across Canada that have been formed to commercialize our research and return resources to the Network for continued discovery and research. These companies represent partnerships among the universities, Network investigators and companies and venture capitalists in Canada. One looks forward to the future when the milestones for these companies are achieved and additional resources are returned to ensure that the pipeline for discovery is well supported in this Network. To that end, we have continued to encourage innovation by providing support for ideas in their earliest stages of development � ideas that could not easily gain support elsewhere.
The Network is continuing to discover new ways to understand how genes cause disease and the challenge in the future will be to understand how these advances will influence the practice of medicine. It am confident that members of this Network will continue to play a leading role in this transformation. We are fortunate to have people of great talent and imagination at all levels of our organization � students, investigators and management. This synergy of creativity, energy and dedication will allow us to take full advantage of the opportunities as we move to the future.
"Nature" August 31, 1995 and June 29, 1995
Early onset Alzheimer's disease
Dr. Peter St George-Hyslop of the University of Toronto led the team which discovered two genes responsible for early-onset Alzheimer's disease.
Fellow Network principal investigator, Dr. Johanna Rommens, collaborated closely in cloning both the genes with the mutations specific to early- onset Alzheimer's disease. The first gene was found on chromosome 14 and is responsible for the most severe form of the disease. The second gene discovery on chromosome 1 adds to the understanding of the biochemical processes of the disease and the eventual design of rational treatments.
Early onset Alzheimer's disease affects individuals as young as 30 years. It progresses faster than the more common late onset forms of the disease but it is otherwise identical. About 250,000 Canadians suffer from Alzheimer's disease.
"Cell" June 2, 1995
New functions of the Huntington disease gene
Dr. Michael Hayden of the University of British Columbia and his team, won an international race to create an animal model in which the Huntington disease gene has been deleted. The findings add important novel insights into the role the HD gene plays in embryonic growth and development.
Network post doctoral fellow, Dr. Jamal Nasir, worked closely with Dr. Hayden in developing the "knock out" mouse model where specific parts of the gene are deleted in every cell. The findings indicate that a previously overlooked area in the brain may play a crucial role in the pathology of the disease.
Huntington disease is an inherited neurological condition which commonly starts in mid-life and leads to death after progressive deterioration of brain cells and brain function over 10-15 years. The disease affects 1 in 10,000 Canadians. Approximately 1 in 2,500 persons are at risk for this disorder.
�Nature Genetics� October 1, 1994
Juvenile Onset Diabetes
Dr. Leigh Field of the University of Calgary discovered two locations for genes which contribute to juvenile onset diabetes. This is a common form of diabetes which develops in childhood and is caused by changes in several genes. The discovery is a step forward in identifying the genes responsible for the disease.
Juvenile onset diabetes affects 1 in every 300 children and approximately 100,000 Canadians in total. The disease frequently causes complications such as blindness and kidney failure. Sufferers need daily insulin injections to survive.
"Cell" January 13, 1995
Spinal muscular atrophy (SMA)
The Network played a key collaborative role in the discovery of a gene involved with spinal muscular atrophy by a team at the Children's Hospital of Eastern Ontario, led by Dr. Alex MacKenzie. The critical large scale genomic DNA sequencing portion of the project was directed by Dr. Robert Korneluk using the Network's advanced core technology facilities.
A potentially important benefit in the isolation of the gene is the discovery of a novel mammalian gene involved in the prevention of programmed cell death, a phenomenon implicated in diseases as diverse as HIV and cancer as well as neuromuscular disorders such as Lou Gehrig�s disease, and other neurodegenerative disorders.
SMA is characterized by the loss of cells known as motor neurons resulting in weakness and wasting of the voluntary muscles. The most severe form has a fetal or post-natal onset with affected children rarely surviving their first few years. The disease is the most common monogenetic cause of death in Canadian infants, affecting approximately 1,000 children and adults.
"Nature Genetics" January 1, 1996
Fragile X syndrome of inherited mental retardation
The team is the first to shed light on the normal function of the FMR1 gene and to determine the role of the protein produced by the gene. The absence of the protein causes fragile X syndrome.
In November, 1995 American Journal of Human Genetics published the team's report that a high prevalence of fragile X unaffected carriers exists in the general population (1 in 259 women). Fragile X mental retardation syndrome is the most common specific cause of mental retardation in children after Down's syndrome, affecting one boy in 1,500 and one girl in 2,500.
"Nature" December 21, 1995 and "Science" October 7, 1994
Breast and Ovarian Cancer
Dr. Steven Narod of Women's College Hospital at the University of Toronto and Dr. Patricia Tonin of McGill's Department of Medicine collaborated with an international team of scientists in the discovery of the second gene for hereditary breast cancer (BRCA2). Drs. Narod and Tonin were part of the team which discovered the BRCA1 gene in 1994.
As with the BRCA1 gene, it is expected that women who inherit the BRCA2 gene may have up to an 85% chance of developing breast cancer. Members of families with the BRCA2 gene may also be at greater risk for other cancers, including ovarian, prostate, throat, and male breast cancer.
BRCA1 and BRCA2 genes together account for over 80% of families in Canada which are considered at high risk for breast cancer.
The Canadian Genetic Diseases Network has created new opportunities for the commercialization of medical research in the dynamic field of human genetics.
1995 ushered in a new era in Network achievements with the launching of several major commercial ventures across Canada: ApoptoGen Inc. (Ottawa), Alzheimer's Canada Inc. (Toronto), and Neurovir Inc. (Vancouver). Commercial funding has been secured for ApoptoGen, and is being finalized for Neurovir. A major diagnostic pharmaceutical collaboration is being developed for Alzheimer�s Canada. In addition, a new Montr�al venture, Sequana Canada, is being planned.
These ventures are based on Network research projects. A new Network "Strategic Fund", earmarked to provide "top-up" funding for research projects with strong commercial potential. facilitates the acceleration of promising research developments and serves as seed funding for Network commercial ventures. Financial returns to the Network come through equity partnerships, sharing in royalties, and new R&D; funding associated with the new ventures.
The Network has developed a broad and strategic approach to intellectual property and has made a heavy investment in leading and implementing patent analysis and filings in cooperation with university and hospital partners. This has created a strong foundation for commercial investments.
The Network is positioned to add considerable value to Canada and the field of medical genetics through the commercialization of research projects, and through new partnerships with universities, hospitals, companies and venture capital organizations.
Strategic Fund:
The following initiatives with strong commercial
potential have been supported by the fund to date:
Genetics of osteoporosis |
$60,000.00 |
Early-onset Alzheimer�s |
$75,000.00 |
Apoptosis |
$50,000.00 |
Gene therapy for the |
$60,000.00 |
Commercialization of biomedical discoveries requires strong intellectual property. Investors need the confidence that the multi-million dollar investments will provide economic returns at the end of what is often a 5-10 year development horizon for new diagnostics and therapeutics.
CGDN Management works closely with investigators and university/hospital partners, who retain ownership of intellectual property developed, to create robust intellectual property positions which can underpin commercial activities. The teams formed by the Network concentrate on filing early to optimize the downstream commercial benefits from Network research. The Network shares in added-value created by these efforts through the sharing of ultimate royalty flows, or by taking equity stakes in new ventures.
The Network is uniquely positioned to add value at each stage of development and has participated and led the formation of several new commercial endeavours including Alzheimer�s Canada (Toronto), Neurovir (Vancouver) and Apoptogen (Ottawa).
Intellectual Property:
Building blocks for new commercial ventures
1994-5 | Cumulative | |||
Patent Filings | 8 | 35 | ||
Licences | 4 |
6 |
Research Phase (I.P. Emerges)
Incubation Phase (Promising opportunity)
Early Seed Stage (Opportunity confirmed)
Seed Financing
Commercialization
The members of this Network have succeeded in creating a unique environment which promotes world-class science and it�s exploitation. Many factors have contributed to this environment but chief among the principles are consensus, cooperation, and trust. It through broad consensus among the participants of this Network from science and industry that it is possible to achieve dramatic results in this competitive world. Through collaborations, it has been possible to maintain internationally competitive research, in spite of limited resources at individual institutions.
This new �networking� is essential in today�s world. No longer is it possible to assemble under one roof the components to provide the research, development and commercialization expertise to carry projects from discovery through to new diagnostics and therapies. This Network is a leader in the world with a new model on how to conduct R&D; efficiently but with total dedication to quality.
The dedication extends to many different facets. In training there is the dedication to make the experience of our students both vigorous and creative. The Network�s training programs involve situations which they would not encounter anywhere else including learning to manage collaborations, effective communication, and patents and commercialization. The Network trainees will be tomorrow�s leaders in science and industry in Canada.
The Network�s industrial partners are now reaching further back into the scientific process to engender the technology transfer and investments necessary to commercialize early-stage technologies. This is a difficult and challenging process. However the methods developed for strategic investments in early stage technologies is already resulting in new ventures and major potential for upside economic impact and employment.
In providing the management and coordination for the operation of this dynamic organization, it has been necessary to develop and employ an entirely new set of skills for management of discovery research, intellectual property, and early stage ventures. The ability to create, along with our university and hospital partners, positions in intellectual property which are robust and internationally competitive has been one area where this Network has excelled.
As we look ahead, not only to the success of new ventures and new infrastructure for molecular medicine in Canada, we also are cognizant that this Network must change. We must structure ourselves to be more independent with a wide variety of funding sources, and to be even more focused on adding value to existing efforts. We will need to work more closely with the Medical Research Council to ensure that the nations� top discovery projects are funded, and that the infrastructure exists to ensure that achievements are exploited to Canadian benefit. Furthermore our partnerships with disease foundations, foreign research organizations, venture capital groups must be strengthened.
I am proud to work with the researchers, partners and management team which together make this Network a success. Although we will become more �corporate�, we must retain the collegiality, the dedication, and the trust which has characterized the successful operations of this Network over the last six years.
To the Canadian public:
To educational institutions, community groups, and schools
...through lectures, seminars, newsletters, visits to laboratories
To federal government representatives:
...through meetings with
...through articles and interviews
MacLeans Bioworld |
CBC Television News CBC Radio: |
To the scientific community
...through excellence in Science:
Aloys Alzheimer Award 1995: American Medical Writers
Association Prize Department of Microbiology Teaching Genetics Society of Canada Award of Gold Medal, Canadian Association Henry Friesen Award, The Canadian J.P. Lecocq Prize, Acad�mie des Sciences, McCalla Fellowship 1995-96: Michael Smith Award of MRC Distinguished Scientist Award: |
NCIC Research Science Award: Pehr Edman Award: Le prix L�o-Pariseau 1994 (Acfas): Prix du Qu�bec (Wilder Penfield): Senior Scientist Award (MRC): Elected Fellow, Appointed to the Alva Appointed Chair of Breast Appointed Chair, Department Appointed Director, |
...through scientific meetings and workshops: The Genetics of Obstructive Lung Disease: Molecular Medicine & Science: Sixth International Society of |
...participated in and supported: Bionet XII Annual Meeting, Vancouver Gene Therapy in Canada - Current Status, 19th International Herpesvirus Ninth International Biotechnology OECD Workshop on Gene Delivery Pacific Northwest Biotechnology R&D; '94 - Technological |
...and aims to produce a new generation of researchers
trained in the highly collaborative Network culture.
The Network Merck Frosst Award for 1994-95 ($34,000 in total) provided crucial project support and professional encouragement for Mr. Frank Merante, Graduate Student at Toronto's Hospital for Sick Children and Dr. Silvia Videl, post-doctoral fellow at Montreal's McGill University.
"The Network/Merck Frosst Studentship Award has gone a long way in
furthering my research career, and has aided in the publication of a
substantial amount of research results from the laboratory. I anticipate
my PhD defense in September and have obtained a Post Doctoral Fellow
position..."
Creating and funding positions for university summer students in the laboratories of Network investigators across the country is the backbone of our hands-on undergraduate training program. In 1994 and 1995, 46 of Canada's best and brightest science students, headed for careers in genetic research or medicine, were awarded 3-month work experience positions.
"The time I have spent at the Canadian Genetic Diseases Network has been
beneficial towards some important decisions regarding my future
occupational goals. Because of the interesting opportunities in genetic
research that were revealed to me, I am considering a career in this field."
One of the unique benefits for Network graduate students and post-doctoral fellows is access to inter-node training exchanges across Canada. The $40,000 "Visiting Researcher" fund in 1994-95 enabled young scientists to work in Network labs across the country, learn specialized techniques of value to their own projects and research centres, while networking with senior scientist mentors.
Perhaps the most highly rated training activity of the Network year is the three day Annual Scientific Meeting. The years 1994 and 1995 brought together over 75 post-doctoral fellows and graduate students in this highly interactive event. Both young scientists and Canada's leading geneticists meet to participate in plenary sessions, brain storming, core technology study sessions, and career-enhancement workshops with representatives from industry. The meeting was held in British Columbia in 1994 and in Quebec in 1995. The 1996 Annual Scientific Meeting will be held in Vancouver in conjunction with the Fourth International Nature Genetics Conference.
Network scientists-in-training
| Post-doctorates | 70 | Graduate students | 86 | Research Associates | 21 | Technicians | 35 |
In keeping with the NCE program mandate to promote funding partnerships and lever existing R&D; resources in innovative ways, the Canadian Genetic Diseases Network raised 50% of annual resources from the private sector, international sources and provincial governments and other sources during 1994-95 (see Fig.1). Support was provided in cash, and in-kind through capital equipment, infrastructure, and collaborations.
Looking ahead, it is expected that 1995-96 and beyond will see a substantial increase in private-sector based Network resources, with Network partnerships and affiliations assisting in counterbalancing the impact of declining federal government funding (see Fig.2 & Fig.3). In the process, the Network is honing research projects to create value in transfer technologies, and is building partnerships with industry through new commercial initiatives.
A sustained core of investment, however, dedicated to the highest quality basic research in human molecular genetics, remains crucial to the health and future of the Canadian Genetic Diseases Network, and the projects which form the pipeline of research and trainees which will attract future investments.
New Investment initiatives are already coming on stream, for instance, the new Centre for Molecular Medicine and Therapeutics in Vancouver and the Neurgene International Joint Collaborative Project with JRDC through the University of Ottawa. These new projects, enabled by Network partnerships, are assisting in launching a new era in both discovery research and exploitation.
The Network challenge for the future is continued championing of Canadian research combined with bold new developments in Canadian receptor companies and industrial partnerships.