Toward individualized medicine: The Jackson Laboratory’s contributions
The limits of medicine today
The practice of medicine is largely reactive. Often we can’t see diseases coming until they have arrived. Typically we wait until their onset and then try to treat or cure them. Often our rescue efforts are too little, too late.
Most of the deadliest diseases that afflict us—cancer, diabetes, heart disease, Alzheimer’s and others—are beyond our current level of understanding. Because we don’t know how genetic and environmental factors interact to cause them, our efforts to treat these diseases are often imprecise, unpredictable and ineffective.
The drugs and treatments we devise are tested on broad populations and are prescribed using statistical averages. However, we know that individuals may respond to a given drug differently than the population average, given their unique genetic makeup and body chemistry. Doctors usually can’t discern who will tolerate a drug, who will have adverse reactions, and what the best dose is for a particular patient.
The promise of medicine tomorrow
As our grasp of genetic science strengthens, the way we maintain our personal health will change. Medicine will increasingly be tailored to each person’s unique genetic makeup. This new approach will open a whole new future of better health care. For each individual we will be better able to:
- understand how genetics and environmental factors influence disease.
- predict the onset of diseases before they occur.
- intervene with therapies and lifestyle changes to prevent or manage diseases.
- prescribe the right drugs in the appropriate doses so patients benefit without adverse reactions.
- treat or cure diseases with tailored therapies.
These dramatic improvements are within the realm of possibility because we are living through a scientific revolution of historic proportions. The sequencing of the human genome and other genomes is giving us powerful new tools for research and discovery. We now have a working “parts list” of all the genes in a cell and are beginning to understand how those parts work together. Scientists around the world—including those at The Jackson Laboratory—are using this information to discover the genetic mechanisms underlying human health and disease. As our understanding grows, medicine is becoming more proactive, precise and powerful, allowing new prevention, diagnosis and treatment options tailored to each individual’s unique genome.
This genomic revolution is changing our lives in the 21st century just as profoundly as the Industrial Revolution shaped the 19th century world. Many of us will benefit, and our children and grandchildren will live longer and healthier lives.
We’re already starting to see the benefits today. For example, we now have genetic tests can determine how well a patient will respond to the blood thinner warfarin,the breast cancer drug Tamoxifen and the blood clot preventative Plavix. This knowledge helps doctors prescribe the right medicines and the right doses for each individual patient, and avoid medicines that won’t work or may bring unwanted side effects.
More developments like these are coming every day, and they are leading us toward better medicine based on each person’s unique genetic makeup.
The Jackson Laboratory’s contributions to individualized medicine
The Jackson Laboratory is a leader in the genomic revolution. Discovering the genetic basis for preventing, treating and curing human disease is our mission.
The Laboratory is home to a research work force of more than 500 people, including more than 200 who have doctoral degrees. Virtually all of these scientists specialize in mouse genetics, and they comprise the largest such body of specialists anywhere in the world.
Humans and mice are very similar genetically, sharing more than 95 percent of their genomes. Mice get most of the same diseases that people get, and for many of the same genetic reasons. Therefore, experimental findings in mice often correlate to human biology. Jackson scientists conduct experiments in mice that would be impossible or unethical if attempted in human beings. Researchers worldwide use mice in the same way, and they turn to The Jackson Laboratory for its expertise. In fact, the Laboratory provides 4,000 lines of genetically defined mice to 16,000 laboratories around the world.
Our expertise in experimental genetics is leading to better treatments and cures for cancer, diabetes, Alzheimer’s, cardiovascular disease, and many other conditions that undermine human health and longevity. We are applying that expertise toward understanding, treating and curing genetic diseases and conditions that confront individual people.
Understanding genetic complexity
States of human health and disease are governed not by a single gene but by complex interactions among hundreds of genes and environmental factors such as diet and exercise. A new approach to studying this biological complexity is called systems genetics, and it’s critical to a future of individualized medicine.
The Jackson Laboratory is a leader in systems genetics. Mining vast databases with powerful statistical tools, our geneticists, mathematicians and computer scientists are generating new insights into the genetic determinants of health and disease. The Laboratory also houses and maintains the international Mouse Genome Informatics database, the world’s most comprehensive collection of mouse genetic data. Our expertise in systems genetics and traditional research methods gives The Jackson Laboratory a distinctive edge in the quest to understand disease. It also bring us closer to the day when medicine is targeted to an individual’s distinctive genetic composition.
For instance, Jackson’s systems genetics group is mapping the dozens of genetic interactions that give rise to type 1 diabetes. Others are deciphering the gene network that controls levels of HDL, the “good” cholesterol, in our blood. Researchers in the Jackson Aging Center are untangling the web of genetic and environmental factors that make us grow old.
Recent research at The Jackson Laboratory helped explain why some patients do not respond as expected to Gleevec, an effective drug for some leukemias. It also identified an additional drug to be used with Gleevec that promises to help these patients.
Connecting experimental genetics to medical practice
Scientists at The Jackson Laboratory work closely with researchers at leading medical centers around the world to apply Jackson’s experimental genetics research to the treatment of human diseases. These scientists also work with the pharmaceutical and biotechnology industries to bring new medications and therapeutic devices to market.
For instance, the Laboratory contributed to groundbreaking work into a new spinal muscular atrophy treatment that acts as a genetic “spell-check” to repair defective genes. This offers new hope for effective intervention in this devastating early childhood disease.
The Laboratory is pursuing potential new partnerships with other organizations that can apply its unique research capacities in systems genetics. One collaboration with a leading medical school would determine the genetic factors involved in responses to new cancer drugs. Another collaboration with the pharmaceutical industry would help determine the genetics underlying drug toxicity.
Research at the Laboratory has led the way to organ transplants, bone marrow transplants and medical use of stem cells. Five Nobel Prize winners have worked at the Laboratory in various capacities. Jackson’s mouse models and resources have catalyzed the work of 15 more Nobel laureates. The most recent are Cappecchi, Evans and Smithies, the 2007 Nobelists who discovered how to “knock out” specific genes in mice.
The non-profit Jackson Laboratory shares its genetics expertise and resources with the global research community through education programs. Every year more than 1,500 scientists from around the world attend Jackson courses and conferences. Every summer some of the nation’s best and brightest high school and college students conduct research and learn under the mentorship of Jackson faculty.
The Jackson Laboratory: Leading the search for tomorrow’s cures
Teams of Jackson scientists and collaborators worldwide are discovering the genetic mechanisms that control human health and disease. Their work is contributing to a future in which medicine is precisely targeted to the unique genetic makeup of each person. We will have more precise and powerful ways to prevent, diagnose and treat some of our worst genetic diseases. These benefits are beginning to arrive, and they will help our children and grandchildren live longer and healthier lives.
