This story originally appeared in UCLA Today, a discontinued publication.

Animal research generates new treatments, benefits society

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Research involving laboratory animals at UCLA leads to many medical breakthroughs that improve people's lives and hold promise for additional improvements in diagnoses, treatments and cures.
 
Breast cancer
UCLA studies utilizing mice were the basis for human clinical trials in patients with metastatic breast cancer. This led to the breakthrough breast cancer medication Herceptin, the first cancer-fighting drug to successfully target a specific genetic alteration, thereby limiting side effects such as hair loss and nausea that often accompany conventional therapies. Mouse models of human cancers such as prostate, pancreatic and lung cancer are widely utilized to test innovative cancer-fighting agents.
 
Parkinson's disease
UCLA scientists pioneered deep brain stimulation, a surgical procedure used to treat a variety of disabling neurological symptoms — most commonly the debilitating symptoms of Parkinson's disease, such as tremors, rigidity, stiffness, slowed movement and walking problems. The procedure, made possible through research involving non-human primates, is used to treat Parkinson's patients experiencing only limited benefits from medication alone. Deep brain stimulation also is used to treat essential tremor, a common neurological movement disorder.
 
Alzheimer's disease
Using rodent models, UCLA researchers helped develop a new diagnostic technology that utilizes positron emission tomography (PET) and integrated microfluidic chips that synthesize and label imaging molecules. This technology allows scientists to search throughout the human body for the molecular errors of disease and has lead to important new diagnostic methods for detecting and monitoring a variety of brain disorders, including Alzheimer's disease, Huntington's disease and cancer. Studies continue at UCLA and elsewhere.
 
Artificial heart
The development of the total artificial heart would not have been possible without first testing the device on animals. The first phase of clinical trials on humans began with the implantation of the device in a patient at Jewish Hospital in Louisville, Ky., on July 2, 2001, followed by five more implants at additional medical centers participating in the total artificial heart clinical trials, including UCLA. The study is ongoing.
 
Schizophrenia
Researchers at UCLA have developed new insights into the biochemical processes that contribute to cognitive disabilities affecting behavior, speech and reasoning in schizophrenia patients. Within the prefrontal cortex, a region of the brain especially well-developed in non-human primates, two neurochemicals engage in an intricate balance to coordinate optimal cognition. As research continues, specifying the nature of these interactions holds promise for discovering genes that cause schizophrenia and developing treatments to counteract it. The work also holds promise for treating other neurological and psychiatric disorders, notably Alzheimer's-related dementia, stroke and brain trauma.
 
Pediatric heart valve
Children with congenital heart defects may soon have an alternative to invasive and risky open-heart surgery. UCLA researchers are developing a heart valve using super-memory, shape-elastic metal alloy that can be loaded into a catheter, inserted into a vein in the groin area, guided into place and deployed at a precise location within the heart. To test the device, researchers are using pigs, whose circulatory system closely resembles that of humans.
 
Atherosclerosis
UCLA research utilizing mice led to the development of a peptide that shows promise in treating atherosclerosis, a disease of the large arteries that is the primary cause of heart disease and stroke. UCLA researchers also have used mouse genetics to reveal how oxidation and inflammation contribute to atherosclerosis and to discover new pathways and genes important in the manifestation of the disease. Mouse models are crucial to a better understanding of the disease and to developing new treatments because examining the mechanisms involved in the initiation and progression of atherosclerosis in humans is not feasible.
 
Autism
UCLA researchers have discovered that a U.S. Food and Drug Administration–approved drug reverses brain dysfunction caused by a genetic disease known as tuberous sclerosis complex (TSC). The findings offer new hope for treating learning disorders and seizures associated with autism, as approximately half of TSC patients also suffer from autism. Working with a mouse model for TSC, UCLA scientists tested rapamycin, a drug approved by the FDA to fight tissue rejection following organ transplants. Rapamycin is well known for targeting an enzyme involved in the production of proteins needed for memory — and the same enzyme is regulated by TSC proteins.
 
Childhood blindness
Using mice, UCLA researchers have discovered that a gene missing in infants born with the blinding disease known as Leber's congenital amaurosis is also a key enzyme required for sight. The identity of this enzyme has long been a mystery to scientists. The findings hold promise for curing some forms of congenital blindness through gene therapy. Much of this ongoing research involves the use of mice with gene mutations.
 
Progeria
Hutchinson-Gilford Progeria Syndrome, also known as progeria or HGPS, is a rare, fatal genetic condition characterized by accelerated aging and cardiovascular disease in children. Using a mouse model that closely mirrors the genetic defect that causes many cases of the syndrome in humans, UCLA researchers found that an experimental drug improved bone density, reduced bone fractures, aided weight gain and delayed the onset of the disease. The findings allow researchers to begin targeting treatment for children and make it feasible to consider clinical trials in humans.
 
Stroke
Various animal models are used to test new clot retrieval devices and other medications for stroke. Clot retrieval devices have shown a beneficial effect in reversing strokes. One such device was invented at UCLA.
 
Heart arrhythmia
Heart disease is the leading cause of death in industrialized societies worldwide. The restitution hypothesis, a new concept for developing drugs to treat cardiac fibrillation — one of the most common forms of arrhythmia, or irregular heartbeat — was based on animal research at UCLA. The hypothesis states that the extent to which cardiac electrical activity shortens as heart rate increases is a key factor in whether fibrillation occurs.
 
Organ transplant rejection
UCLA researchers using rats to seek new ways of preventing organ rejection in transplant recipients have identified a gene that helps prevent a patient's immune system from rejecting organs. In addition, promising allochimeric-therapy research seeks to reduce the dosage or shorten the regimen of immunosuppressive medications used to prevent rejection.
 
Gene therapy for cancer
UCLA researchers use miniaturized positron emission tomography (MicroPET) to track the effects of gene therapy on cancer in living rodents. The animal research has led to new methods of imaging gene expression, which directly affects the safety and efficacy of gene therapy trials on human cancer patients. The same work allows researchers to better understand basic cancer biology and how tumors grow and spread, enabling the development of new treatment strategies.
 
Radiation therapy
UCLA oncology specialists have used specially bred mice to design more effective radiation therapies for cancer, with minimal side effects for healthy body tissue. Investigators approximate the clinical situation of patients' daily radiation treatment in mice in order to evaluate the long-term response of the body, organ by organ. The mice used in the research are of a strain known for their longevity and are carefully protected from infection so they will live long enough to be evaluated.
 
Tuberculosis vaccine
The bacterium that causes tuberculosis infects some 2 billion people worldwide, and 8 million new cases of active tuberculosis develop annually. New vaccines to combat the disease are being developed using guinea pigs, which are highly susceptible to infection by the tuberculosis bacterium and develop a disease remarkably similar to tuberculosis in humans. UCLA researchers developed the first vaccine in 100 years that is more potent than the current vaccine.
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