Replacement Therapy
Will the Real Stem Cell Please Stand Up
Stem cell replacement (SCR) therapy for a wide variety of medical diseases has been a topic of discussion for many years now. The term “stem cell” first appeared in the scientific literature as far back as 1868. A German biologist named Ernst Haeckel used the term to describe the single-celled organism that acted as an ances- tor cell to all living things in history. Jump forward to August 9, 2001 when President George W. Bush signed an order authorizing the use of federal funds for research on a limited number of existing human embryon- ic stem cell lines.
In January of 2009 the Geron Corpora- tion announced the FDA’s announcement of approval for a limited phase 1 trial for a new treatment of spinal cord injuries based entirely on human embryonic stem cells. Later that year, President Barack Obama signed Executive Order 13505 which loos- ened restrictions on human embryonic stem cell research. In July of 2009 the NIH issued revised guidelines for federal funding for stem cell research. Currently the National Institutes of Health approves of at least 13 new human 
embryonic stem cell lines for federal funding.
After a hundred and fifty years and many millions of research dollars spent … what do we know about stem cell replacement therapy, specifically its possible role in lung disease? Just exactly what is a stem cell? Stem cells are found throughout the body and have several main characteristics:
- They can renew themselves by simple cell division;
- They can differentiate themselves, be- come different in the process of growth or take on properties of several different cell structures and tissues based on need;
- They can be transplanted into other organisms where they will continue to divide and differentiate.
Adult stem cells can be found in the skin, bone marrow, brain, blood vessels, liver and skeletal muscle. Theoretically, stem cells can be used to heal, or even regenerate, damaged tissue. It was believed that there were no stems cells in the lungs themselves. Recently researchers at Brigham Women’s Hospital found stem cell evidence in 12 adult donor lungs and nine lungs from fetuses that had died of natural causes. And much to every- one’s amazement, these stem cells were able to divide and form new lung structures!
Stem cell replacement therapy is not being offered to patients with any form of lung dis- ease in the United States. Stem cell pioneers see this therapy as perhaps a promising new direction to take in finding new ways to treat all lung disease, including COPD. Finding adult stem cells in adult human lungs eliminates at least one of the powerful ethical dilemmas concerning stem cell replacement therapies – the need for fetal blood from aborted fetuses. In the past five years stem cells have been harvested primarily from bone marrow. The harvested cells can then be returned to the lungs via intravenous or in some cases, nebulized back into your lungs.
It is much safer to replace your own stem cells than to use a donor’s stem cells
There is a great deal of controversy regarding the use of stem cell therapy to treat COPD. For stem cell treatments to be clinically significant, many millions of stem cells need to be transplanted back into the designated recipient. Harvested stem cells do not reproduce without outside manipulation to produce even larger quantities. The FDA says that manipulating naturally harvested stem cells with other agents technically turns the stem cells into prescription drugs, which will bring more regulation.
How exactly can stem cell therapy theoretically help patients with lung disease? It seems possible that there may be anti-inflammatory benefits to stem cell therapy. It is entirely possible that stem cell therapy may actually trigger the production of reparative growth factors. It is also possible that over time stem cell therapy could accelerate the regeneration of the alveoli and blood vessels of the lungs. Much of the hope for beneficial effects has actually been seen in mice. It is a huge leap to speculate on any possible effects on the human lung, based on itty bitty mice lungs.
So why aren’t there hundreds of studies being done all over the world on stem cell therapy? We must begin all discussions with costs and end with return on investment. One of the more controversial aspects of SCR therapy is the wide variation in costs. Costs at SCR centers show a range between $6,000 to a whopping $64,500! The vast majority of these centers reside outside the continental United States. These fixed costs do not include transportation, hotel or food expenses. SCR centers can be found all over the world. As might be expected, the therapy is not covered by any known health insurance plan.
The American Lung Association’s State- ment on Stem Cells and Cell therapies for Lung Diseases states, “As yet, there is very little known about the short- and long-term effects of administering any type of stem cell therapy to patients with lung diseases. At present there are only a small number of approved clinical trials in the United States and Canada. We are hopeful there will be more in the future. However you may come across information on the internet or other sources about stem cells being administered to patients with lung disease. We caution all patients to carefully consider the claims of benefit being made by many of these pro grams as they have not been substantiated. Because of the potential for harm, the lack of any proven benefit, and the high fees that many of these programs charge, we caution you not to participate in these or any other comparable unauthorized or unapproved stem cell administrations, unless indepen- dent, credible, reliable and objective sources of information are available to substantiate the information and claims being made.” The ALA recommends the International Society for Stem Cell Research (ISSCR) as a reliable source for information. If you’d like more detailed information please visit www. closerlookatstemcells.org.
ISSCR, in its recently published hand- book, tells us, “The ISSCR is very concerned that stem cell therapies are being sold around the world before they have been proven safe and effective. Stem cell therapies are nearly all new and experimental. In these early stages, they may not work, and there may be downsides. Like any new drug, stem cell therapies must be assessed and meet certain standards before receiving approval from national regulatory bodies to be used to treat people.”
This leads me to a topic sometimes known as “Stem Cell Tourism.”
Stem cell tourism is when people travel to another country to receive treatments unavailable to them at home. It exists chief- ly because most stem cell “treatments” are unproven and not readily available from rep- utable local medical services. Stem cell tour- ism is sustained because of a religious-like belief in the promise of the regenerative powers of stem cells.
Medical Tourism is also becoming popular with United States companies. We learned of a furniture and auto parts manufacturer in western North Carolina who gave employees a choice for their surgery: Pay a co-pay in the United States or have the procedure done abroad for free. One lady had weight loss surgery and another man had a knee replacement at a private hos- pital in Costa Rica. Nearly one million Americans go overseas for procedures every year, according to the U.S. Centers for Disease Control and Prevention. While it all sounds too good to be true, medical experts cautioned that there are serious concerns about “medical tourism” and having procedures done overseas. Glenn Cohen, co-director of the Petrie-Flom Center for Health Law Policy at Harvard University, said there is a risk of post-operative complications and said there have been documented cases of people dying or developing in- fections after having surgeries in foreign countries.
The problem of “questionable” stem cell clinics has been growing over the past five to 10 years. Stem cell centers can be found in many countries around the word. Since they are outside the purview of the FDA, they can make all kinds of claims and offer stem cell treatments for fatal or incurable diseases like ALS, spinal cord injury and even strokes. Targeting mostly affluent westerners, costs can easily exceed $100,000. Look closely at the various Internet ads for stem cell centers. You will most commonly find testimonials from patients who have had near miraculous responses. A logical question might be “Why not then publish your data and undergo rigorous peer review?” It is not unusual to find stem cell centers just over the border from modern medicine practiced here in the United States. Mexico is convenient for both medical staff and most patients. There are around 20 stem cell replacement clinics in Tijuana alone. Are they selling desperate pa- tients a 21st century version snake oil? There will always be someone who swears they got better from whatever ailed them in the first place by taking a couple of teaspoons. You can read testimonial after testimonial on the websites of the replacement centers describ- ing fantastic results. It would seem there is no better indication for Caveat Emptor – Let the Buyer Beware!
After consulting with many experts, they are not aware of any stem cell research being done. This does not mean that some- where within the academic community of the United States there isn’t solid research happening as I write this. Undoubtedly there are legitimate studies being done.
Stem cell therapy for lung disease is in its infancy. Theoretically it has the possibility of bringing great hope to many patients who have exhausted other forms of medical therapy to treat their lung disease. On paper, stem cell replacement therapy looks like it may be the next “BIG” thing. However, the biochemical, and technical bridges that need to be crossed are substantial.
Do you remember when Fen-phen was the hottest new diet pill? It was actually on the September 1996 cover of Time magazine, the same year it was placed on the market. Sales in that first year were $300 million as more than 18 million prescriptions were filled. Fen-phen was pulled off the mar- ket in September 1997 by the FDA as there were at least 75 reports of Fen-phen induced heart injuries. Some patients had taken the drug for as little as a month and developed serious cardiac complications including the uniformly fatal Primary Pulmonary Hyper- tension.
Let us heed the message so powerfully sent by the Fen-phen disaster. Stem cell research is so very promising in the possible treatment of so many diseases and chronic conditions. If you or a loved one is contemplating stem cell replacement therapy, spend the time investigating your particular situation. Make sure you meet and talk with your family physician, specialist, and certainly spend your time wisely in gathering your data.
Happy Holidays!
John R. Goodman BS RRT is Executive Vice President of Technical/ Profes- sional Services at Transtracheal Ser- vices, Denver, CO, who says “All You Need Is Love!”