First Embryonic Stem-Cell Treatment on Patient Begins
It was announced this Monday that an American patient with a spinal cord injury that has caused paralysis, became the first person in the world treated with an embryonic stem cell based drug. The injections of the cell therapy grown from embryonic stem cells were administered at Shepherd Center in Atlanta. No details regarding the patient's age or sex were released as the individual wished to remain anonymous. Only an individual with a paralyzing spinal cord injury suffered less than 14 days before being treated could qualify for entry into the study.
This treatment marks a milestone in medical research, as it is the first human trial of an embryonic stem cell based therapy. In the evolving field of what is termed regenerative medicine, this marks a major advance. Though the research is preliminary, stem cell researchers are encouraged to see these trials begin so soon after the discovery of the properties and potential of stem cells. It has only been 12 years since the discovery of human embryonic stem cells. Scientist soon speculated on the regenerative properties of such cells, leading to the concept of regenerative medicine.
The patient was the first to be enrolled and treated in a safety trial, known as GRNOPC1. Geron, an American company, is running the trial, which is the first clinical study of any such cell treatment. Geron Corporation is based in Menlo Park, California. They have worked in conjunction with researchers at the University of California, Irvine, in the development of this drug model. The company's stated mission is to develop first-in-class biopharmaceuticals for the treatment of cancer and chronic degenerative diseases, including spinal cord injury, heart failure and diabetes. They are also actively working on advancing an anti-cancer drug and as well as a cancer vaccine that target the enzyme telomerase. Multiple clinical trials for different cancers are ongoing.
Embryonic stem cell-derived neural cells have been used by researchers to treat nervous system disorders in animal models. In spinal cord injuries, neural cells derived from animal embryonic stem cells and injected into the spinal cord injury site produced significant recovery of the animal's ability to move and bear weight. In addition to spinal cord injury, GRNOPC1 may have value for other central nervous system indications, such as Alzheimer's disease, Canavan disease and multiple sclerosis. Genron has entered into collaborations with academic groups to test GRNOPC1 in animal models of these diseases.
It was just in January of 2009 that Genron announced it had received approval from the FDA for a study of its stem-cell treatment for spinal cord injuries. The trial was approved for up to 10 patients. At that time, the announcement capped more than a decade of advances in the company's labs. It also came in conjunction with an expected shift in U.S. policy toward support of embryonic stem-cell research.
Despite it’s significance, this medical first does not come without controversy. Many have voiced ethical concerns. Basic bio-science researchers believe embryonic stem cells to be the most versatile stem cells. Using such cells they have regrown tissue and cured diseases in animals. However, to get these cells, scientists have to destroy an embryo, which many feel is akin to abortion. A judge in August blocked federal funding of embryonic stem cell research. Accordingly, researchers are proceeding with caution, being well aware of the medical complexity and social sensitivities involved in this line of work. Development of this drug has been funded totally with private dollars and his been underway for years. Embryonic stem cells are derived from embryos that develop from eggs that have been fertilized in vitro—in an in vitro fertilization clinic—then donated for research purposes with informed consent of the donors. They are not derived from eggs fertilized in a woman's body.
Some have argued that adult stem cells can be used for these purposes, avoiding the moral questions raised by many over the use of embryonic stem cells. To date, it is not clear if adult stem cells hold the same potential, though research continues. An adult stem cell is an undifferentiated cell, found among differentiated cells in a tissue or organ. These cells can renew themselves and have the potential to differentiate into some or all of the major specialized cell types of the tissue or organ. The primary roles of adult stem cells in a living organism are to maintain and repair the tissue in which they are found.
This first patient treatment is the very beginning of human trials, labeled phase one trials. Nearly 40% of all drugs developed never make it past phase one, which is a trial based totally on the safety of the drug or treatment. Researchers note that despite this advance, it could be years before we know if any drug developed at this stage will actually work. The trial is formally named: hESC-Derived Oligodendrocytes - GRNOPC1 for short.
Clearly, for many suffering with chronic, debilitating, or incurable diseases and injuries this research holds great promise. Only time will tell if the promise will be realized.