There are two ideas behind the use of cells as a medical treatment. The first is to provide a source of missing cells, say to heal a tissue that is injured or to renew a population of cells that are killed off by a disease such as Alzheimer's. The second notion is to manipulate cells to produce a missing substance, such as the protein that is missing in boys affected by Duchenne muscular dystrophy.
Researchers at the McGowan Institute are developing cell-based treatments based on both approaches for a broad array of injuries and conditions, using many different cell types. The Institute is particularly committed to exploring the full potential of adult stem cells. These cells, which exist in all of us as a repair mechanism for tissues lost to trauma, disease, and wear and tear, have been studied for decades, and are already widely used to treat some conditions, such as leukemia.
The McGowan Institute is participating in the country's first two FDA-approved human clinical trials of adult stem cells for cardiac therapy, in which patients' own stem cells are injected into their heart muscle. The patients in the first trial are in need of heart transplants, and will receive their own stem cells at the same time that they are implanted with ventricular assist devices. As donor hearts become available to these patients, the researchers will have an opportunity to examine how the injected stem cells grew and developed in the original hearts.
In the second trial, patients undergoing coronary artery bypass graft surgery will receive their own stem cells. This study will help researchers determine how many stem cells are required to improve heart function.
Urinary incontinence afflicts over 17 million Americans, some of whom receive collagen injections to add bulk to the bladder sphincter. Unfortunately, the injected collagen can sometimes cause an immune reaction. At the McGowan Institute, researchers have developed a rejection-free alternative treatment using a patient's own stem cells from muscle. These are isolated from a small biopsy of the patient's thigh, replicated in the laboratory over several weeks, then injected into the bladder sphincter. The stem cells persist in the bladder for up to six months and grow to become the same sort of muscle that the bladder sphincter is made of, improving its strength.
Cancer Stem Cells
The real culprit in cancer may not be its hallmark rapidly-dividing cells, but the stem cells that give rise to those cells. If indeed there are mutant stem cells at the root of cancers, this new notion may explain why so many cancers recur after radiation and chemotherapy have done away with original tumor: traditional cancer therapies could be leaving the faulty stem cells untouched.
Duchenne Muscular Dystrophy
Duchenne muscular dystrophy is a genetic disorder afflicting one in 3,500 boys, most of whom will die in adolescence or young adulthood as their muscles — including their hearts — weaken. The problem is that their muscle cells don't produce the essential protein dystrophin.
McGowan Institute researchers have identified and isolated a line of stem cells in muscles that offers new hope for Duchenne patients. When these stem cells are genetically engineered to contain a gene encoding for dystrophin, they produce dystrophin in Duchenne-affected muscles. This work may produce a minimally invasive treatment in which the patient's own stem cells are isolated, engineered in the lab to carry a functioning dystrophin gene, then reinjected into muscle.