Date: July 1st, 2000
Over the next few decades, the new field of "regenerative medicine" promises to fundamentally alter the way diseases, especially those affected by the aging process, are approached and treated. Regenerative medicine involves research on ways to renew the body's tissues using natural signals, such as genes and proteins. According to William Haseltine, CEO and chair of Human Genome Sciences, Inc., the incredible strides that biomedical research has made over the past 30 years will be dwarfed by the coming regenerative medicine "revolution." The Human Genome Project, a collaborative effort to sequence the entire length of human DNA, will provide reams of data to scientists and spur their progress.
Regenerative medicine consists of three main avenues of research:
First: the use of genes, proteins, and antibodies as medicines. For example, researchers are studying a protein called vascular endothelial growth factor (VEGF), which may help build blood vessels in the heart, allowing some patients to avoid cardiac surgery. In the future, a patient could receive VEGF gene therapy "and then go home and build their own blood vessels," Haseltine said. Gene therapy may also help cancer patients; for example, clinical trials are examining the effects of providing a normal p53 gene to people with a mutated or lost p53. (Problems with the p53 gene are implicated in more than half of all cancers.)
Many hormones are also being researched. For example, a naturally occurring cardiac hormone called nesiritide is being tested as a short-term treatment for congestive heart failure. Also being researched are antibodies to either fight cancer or help physicians detect it more easily.
Haseltine believes the first treatments of this type should be available within three years, and that within 20 years they will make up half of all medications.
The second avenue of research is tissue engineering, or the use of human cells to rebuild organs, either by injection into diseased tissues or by using cells to reconstruct new healthy organs outside the body as replacements. "One day we will regrow organs for individuals in the laboratory - we will have successful autotransplantation," Haseltine said. Bone, skin, and cartilage are already being created in the laboratory, said Haseltine, and he is part of a project to grow a functioning human heart in this fashion.
The third research field is the use of human pluripotent stem cells as treatments. Stem cells, which are the earliest cells that form after conception, have the ability to develop into many different types of cells, such as muscle cells, nerve cells, heart cells, blood cells and others. Because of this ability, they are enormously important to science and hold great promise for advances in health care. Further research in this area may help scientists generate cells and tissue that could be used for transplantation or replacement for damaged cells resulting from Parkinson's, Alzheimer's, spinal cord injury, burns, stroke, diabetes, and heart disease. Stem cells can also help improve our understanding of the complex events that occur during normal human development, and help us understand what goes wrong to cause diseases and conditions such as birth defects and cancer. And human pluripotent stem cells could change the way we develop drugs and test them for safety and potential efficacy. New medications could first be tested using these stem cells, such as liver cells or skin cells, and only those drugs that are both safe and effective would then be tested on human and animal subjects.
In a recent review of regenerative medicine in the online Journal of Regenerative Medicine (of which Haseltine is the editor), Dr. David Stocum wrote: "The basic research we are doing now will evolve into the regenerative medicine of the future, and eventually into the prevention of the diseases... that regenerative medicine is designed to treat ….regenerative biology and medicine is a science whose time has come."