To date, only nonembryonic human stem cells have been used in cell-based gene therapy studies. The inherent limitations of these stem cells, as discussed below, have prompted scientists to ponder and explore whether human embryonic stem cells might overcome the current barriers to the clinical success of cell-based gene therapies.

Gene therapy is a relatively recent, and still highly experimental, approach to treating human disease. While traditional drug therapies involve the administration of chemicals that have been manufactured outside the body, gene therapy takes a very different approach: directing a patient's own cells to produce and deliver a therapeutic agent. The instructions for this are contained in the therapeutic transgene (the new genetic material introduced into the patient). Gene therapy uses genetic engineeringthe introduction or elimination of specific genes by using molecular biology techniques to physically manipulate genetic materialto alter or supplement the function of an abnormal gene by providing a copy of a normal gene, to directly repair such a gene, or to provide a gene that adds new functions or regulates the activity of other genes.

Clinical efforts to apply genetic engineering technology to the treatment of human diseases date to 1989. Initially, gene therapy clinical trials focused on cancer, infectious diseases, or disorders in which only a single gene is abnormal, such as cystic fibrosis. Increasingly however, efforts are being directed toward complex, chronic diseases that involve more than one gene. Prominent examples include heart disease, inadequate blood flow to the limbs, arthritis, and Alzheimer's disease.

The potential success of gene therapy technology depends not only on the delivery of the therapeutic transgene into the appropriate human target cells, but also on the ability of the gene to function properly in the cell. Both requirements pose considerable technical challenges.

Gene therapy researchers have employed two major strategies for delivering therapeutic transgenes into human recipients (see Figure 11.1. Strategies for Delivering Therapeutic Transgenes into Patients). The first is to "directly" infuse the gene into a person. Viruses that have been altered to prevent them from causing disease are often used as the vehicle for delivering the gene into certain human cell types, in much the same way as ordinary viruses infect cells. This delivery method is fairly imprecise and limited to the specific types of human cells that the viral vehicle can infect. For example, some viruses commonly used as gene-delivery vehicles can only infect cells that are actively dividing. This limits their usefulness in treating diseases of the heart or brain, because these organs are largely composed of nondividing cells. Nonviral vehicles for directly delivering genes into cells are also being explored, including the use of plain DNA and DNA wrapped in a coat of fatty molecules known as liposomes.

Figure 11.1. Strategies for Delivering Therapeutic Transgenes into Patients.

( 2001 Terese Winslow)

The second strategy involves the use of living cells to deliver therapeutic transgenes into the body. In this method, the delivery cellsoften a type of stem cell, a lymphocyte, or a fibroblastare removed from the body, and the therapeutic transgene is introduced into them via the same vehicles used in the previously described direct-gene-transfer method. While still in the laboratory, the genetically modified cells are tested and then allowed to grow and multiply and, finally, are infused back into the patient.

Gene therapy using genetically modified cells offers several unique advantages over direct gene transfer into the body and over cell therapy, which involves administration of cells that have not been genetically modified. First, the addition of the therapeutic transgene to the delivery cells takes place outside the patient, which allows researchers an important measure of control because they can select and work only with those cells that both contain the transgene and produce the therapeutic agent in sufficient quantity. Second, investigators can genetically engineer, or "program," the cells' level and rate of production of the therapeutic agent. Cells can be programmed to steadily churn out a given amount of the therapeutic product. In some cases, it is desirable to program the cells to make large amounts of the therapeutic agent so that the chances that sufficient quantities are secreted and reach the diseased tissue in the patient are high. In other cases, it may be desirable to program the cells to produce the therapeutic agent in a regulated fashion. In this case, the therapeutic transgene would be active only in response to certain signals, such as drugs administered to the patient to turn the therapeutic transgene on and off.

To date, about 40 percent of the more than 450 gene therapy clinical trials conducted in the United States have been cell-based. Of these, approximately 30 percent have used human stem cellsspecifically, blood-forming, or hematopoietic, stem cellsas the means for delivering transgenes into patients.

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11. Use of Genetically Modified Stem Cells in Experimental ...

Stem Cell Therapy: Helping the Body Heal Itself

Stem cells are natures own transformers. When the body is injured, stem cells travel the scene of the accident. Some come from the bone marrow, a modest number of others, from the heart itself. Additionally, theyre not all the same. There, they may help heal damaged tissue. They do this by secreting local hormones to rescue damaged heart cells and occasionally turning into heart muscle cells themselves. Stem cells do a fairly good job. But they could do better for some reason, the heart stops signaling for heart cells after only a week or so after the damage has occurred, leaving the repair job mostly undone. The partially repaired tissue becomes a burden to the heart, forcing it to work harder and less efficiently, leading to heart failure.

Initial research used a patients own stem cells, derived from the bone marrow, mainly because they were readily available and had worked in animal studies. Careful study revealed only a very modest benefit, so researchers have moved on to evaluate more promising approaches, including:

No matter what you may read, stem cell therapy for damaged hearts has yet to be proven fully safe and beneficial. It is important to know that many patients are not receiving the most current and optimal therapies available for their heart failure. If you have heart failure, and wondering about treatment options, an evaluation or a second opinion at a Center of Excellence can be worthwhile.

Randomized clinical trials evaluating these different approaches typically allow enrollment of only a few patients from each hospital, and hence what may be available at the Cleveland Clinic varies from time to time. To inquire about current trials, please call 866-289-6911 and speak to our Resource Nurses.

Cleveland Clinic is a large referral center for advanced heart disease and heart failure we offer a wide range of therapies including medications, devices and surgery. Patients will be evaluated for the treatments that best address their condition. Whether patients meet the criteria for stem cell therapy or not, they will be offered the most advanced array of treatment options.

Allogenic: from one person to another (for example: organ transplant)

Autogenic: use of one's own tissue

Myoblasts: immature muscle cells, may be able to change into functioning heart muscle cells

Stem Cells: cells that have the ability to reproduce, generate new cells, and send signals to promote healing

Read more:
Stem-Cell Therapy and Repair after Heart Attack and Heart ...

Russell A. Foulk, M.D. was born in Logan, Utah while his father played football for Utah State University. He has become an internationally renowned expert in the treatment of infertility. He has a strong passion for overcoming infertility and has helped several thousands of couples from around the world to achieve their dreams of having a baby. This drive has led him to establish active fertility treatment centers in four different states in order to enable all those who suffer from infertility to find an affordable and effective way to get pregnant.

He graduated magna cum laude from Brigham Young University with dual majors in microbiology and Asian studies, and dual minors in zoology and Korean. He finished at the top of his class while receiving his doctorate of medicine from the University of California, San Diego, School of Medicine. During his post-graduate training in Ob/Gyn at U.C. Irvine Medical Center and his fellowship in reproductive medicine at U.C. San Francisco, he received a prestigious National Institute of Health grant, published numerous papers and presented before many national organizations. While on faculty at U.C. San Francisco, his research endeavors focused on implantation physiology, in which he shares two patents on unique immunologic proteins expressed by placental cells. He continues his research interests through multi-center investigative trials and basic research into the mechanisms of implantation and stem cells.

Dr. Foulk is an associate clinical professor at the University of Washington, Department of Ob/Gyn and at the University of Nevada, School of Medicine. He routinely teaches medical students, residents and general education courses. He has set up several IVF centers in Nevada, Idaho, Utah, Hawaii, Belgrade andSerbia. He has served for 8 years on the Board of Directors of the Pacific Coast Reproductive Society and recently served as the President of the Society.

On a regular basis, he presents at international meetings and local conferences. Nationally, he has appeared as a fertility expert on the Oprah Winfrey show twice, the Montel Williams show, the Leeza Gibbons show, Inside Edition and German national television. The British Broadcasting Company profiled Dr. Foulk's expertise in a medical program on reproductive ethics. Recently, he presented in Valencia, Spain and in Japan on the Tokyo Broadcasting Company for his work in third party reproduction. He has been quoted in the Associated Press, Reuters, Wall Street Journal and many of the nation's newspapers.

Child magazine in 2005 ranked Dr. Foulk's center in Nevada among the top six in the nation. In 2009, The Nevada Center for Reproductive Medicine was ranked among the 17 "Fertility Centers of Excellence" by the Contemporary Ob/Gyn Journal.

Dr. Foulk is a founding member of StemLifeline, a stem cell research company. In 2006, he was invited to Washington D.C. by Dr. Bill Frist, the Senate Majority Leader, to brief him on embryology and stem cell research. While there, Dr. Foulk met with President George W. Bush for 1 hour in the White House Oval Office before his vote on the subject. On a personal note, Dr. Foulk has been married for over 25 years. He and his wife have 5 children. He is active in his community and church affairs.

CURRICULUM VITAE

Russell A. Foulk, M.D.

Associate Clinical Professor University of Nevada, School of Medicine University of Washington, School of Medicine

Utah Fertility Center 1988 W. 930 N. Suite B Pleasant Grove, Utah 84062 Phone: 801-492-9200 Fax: 801-492-3764

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Dr. Foulk - Welcome to Nevada Center for Reproductive Medicine

Top 10 list of important, easy to understand facts for patients about stem cell treatments

For better or worse, I am in the unique position of being a stem cell scientist and also a patient. Looking on the bright side this gives me a unique perspective on things.

I know there are thousands of people out there looking for more practical information about stem cell therapies and treatments. These folks understandably are using the Internet to look for some clear, good info on stem cell treatments either for themselves or their loved ones. Too often the info that is out there is either wrong, misleading, or overly complex.

So in this post I want to address this need speaking as a scientist, patient advocate and cancer survivor in the form of 10 key facts to help you guide your way through the jungle of stuff out there about stem cells.

1) Stem cells are essentially a type of drug or biological and possibly permanent in nature. Yeah, they are extremely unusual drugs, but they are drugs. The FDA considers them drugs. Unlike other drugs, once a patient receives a stem cell drug, it will not necessarily simply go away like other drugs because a stem cell drug consists of living cells that often behave in unpredictable ways. What this means is if the stem cells are doing bad things your doctor has no way to stop it.

2) Like any medical product, even aspirin, stem cells treatments will have side effects. Not maybe. Definitely. Our hope is the side effects will be relatively mild.

3) The only stem cell treatment explicitly approved by the FDA for use in the U.S. is bone marrow transplantation. What this means is that any other stem cell treatment you see advertised on Facebook or Google or elsewhere that indicates it will be given to you inside the U.S. may in fact be illegal and unsafe. The exception to this is if it is part of an FDA-approved clinical trial.

4) If you venture outside the U.S. for a stem cell treatment, use extra caution and have a knowledgeable physician inside the U.S. guiding you. We have to avoid the trap of thinking that only the U.S. can offer advanced medical treatments, but on the other hand within the U.S. you have the added safety of the FDA, which is trying to protect you. In the vast majority of other countries regulatory agencies are practically non-existent or are far less strict than the FDA.

5) Stem cells are not a cure all. I am as excited as anybody about the potential of stem cells to treat a whole bunch of diseases and injuries, but they are not some kind of miracle cure for everything. When a doctor offers to inject some kind of stem cells or a stem cell-derived product into a patient either into the bloodstream or into a specific place that is injured such as a shoulder, we just do not know at this point if it will do any good with the exception of bone marrow transplant.

6) Dont let celebrities be your guide to medical care. The number of famous people getting stem cell treatments is increasing including sports stars and politicians. Dont let what these folks do influence what you decide to do about your health. Just because they are famous do not believe for one minute that they are any more informed than you or your personal doctor about medical treatments or stem cells. If anything I think sometimes famous people are more reckless with their health than average people like you and me.

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Patients guide to treatments | Knoepfler Lab Stem Cell Blog