New NIH Director Emphasizes Practical Science

A highly accomplished geneticist who is not afraid to express his religious convictions, Dr. Francis Collins assures the public that his focus as the new director of the National Institutes of Health (NIH) will be on science, not religion.

As Dr. Collins publically stated today, "The NIH director needs to focus on science. I have no religious agenda for the NIH."

In his first interview before formally greeting the employees of the $40-billion agency that he has been appointed to direct, Dr. Collins added, "Here we are at a circumstance where I think our country is seeking maybe to redefine our image a bit in the world, from being the soldier to the world to being perhaps the doctor to the world. I’d like to see that happen."

President Obama announced the appointment of Dr. Collins as the new director of NIH on July 8 of this year, a decision which was met with mixed reactions from the media. Although Dr. Collins enjoys a widely respected, sterling reputation among scientists, less-informed critics in the media were concerned, at least initially, that the religious views which Dr. Collins espouses may interfere with an objective and impartial scientific approach to directing NIH. Among other responses, an article in U.S. News & World Report at the time was entitled "Obama Names an Evangelical to Lead the NIH", claiming that "Obama this week picked a Bible-believing, loud, and proud evangelical Christian to head the National Institutes of Health." Today, however, Dr. Collins may have put such concerns to rest.

Although Dr. Collins has been described as an "evangelical Christian who has defended the inherent compatibility of science and religious beliefs", it is clear that any personal religious views to which Dr. Collins might ascribe have certainly not interfered with his scientific objectivity in the past, and he has already directed a number of very high-profile scientific projects. Several other media sources offered more scientifically based assessments of his appointment, such as the Wall Street Journal which simply reported that, "Obama Plans to Name Renowned Geneticist to Head NIH."

Dr. Collins earned a B.S. in chemistry from the University of Virginia in 1970, a Ph.D. in physical chemistry from Yale in 1974, and an M.D. from the University of North Carolina at Chapel Hill in 1977. He first distinguished himself at the University of Michigan where he gained recognition for his pioneering work in positional cloning, a type of genetic screening and gene identification technique. In 1993 he accepted an invitation to succeed Dr. James Watson (Nobel laureate and co-discoverer with Dr. Francis Crick of the helical structure of DNA) as director of the National Human Genome Research Institute (NHGRI), one of 27 institutes within NIH, which he directed from 1993 to 2008. During this time Dr. Collins also became director of the Human Genome Project, an international research project which resulted in 2003 in the successful mapping of the genetic sequences of 3.1 billion chemical base pairs in the human DNA. Along with Craig Venter, founder and former president of Celera Genomics as well as the founder of The Institute for Genomic Research, Dr. Collins was awarded the "Biography of the Year" award in 2000 by the A&E Network, and U.S. News & World Report together with the Harvard Center for Public Leadership named both Collins and Venter "America’s Best Leaders" in 2005.

In 2006 Dr. Collins authored the book, "The Language of God: A Scientist Presents Evidence for Belief", in which he describes scientific discoveries as an "opportunity to worship". Dr. Collins awknowledges that he has been highly influenced by C.S. Lewis, especially by the book "Mere Christianity" to which Collins attributes his conversion to Christianity at the age of 27. Prior to his appointment as the new director of NIH, Dr. Collins has most recently served as president of The BioLogos Foundation, which he founded in 2009, the mission of which is to "contribute to the public voice that represents the harmony of science and faith."

Dr. Collins has received numerous awards and honors throughout his career, including election into the National Academy of Sciences and the Institute of Medicine. Additionally, he is the recipient of the Presidential Medal of Freedom, the highest civilian honor awarded by the President of United States.

As President Obama himself stated when he made the official announcement on July 8, "My administration is committed to promoting scientific integrity and pioneering scientific research, and I am confident that Dr. Francis Collins will lead the NIH to achieve these goals."

This year the NIH will have $40 billion at its disposal for the funding of medical research, including that which is to be conducted on human embryonic stem cells.

UC-Davis Receives Adult Stem Cell Grants

The California Institute for Regenerative Medicine (CIRM), the state legislative stem cell agency, has announced today the awarding of two separate multi-million-dollar, multi-year grants to the University of California at Davis for the R&D of adult stem cell therapies in the treatment of neurodegenerative diseases as well as inherited blood and metabolic disorders.

The two grants include $2.7 million to be devoted to the development of a treatment for Huntington’s disease using the type of adult stem cell known as mesenchymal stem cells (MSCs), and a separate grant worth $4.2 million to develop a safe treatment of infants in utero who may be predisposed to inherited blood cell disorders such as sickle cell anemia and thalassemia, among others.

According to Dr. Jan Nolta, director of the Stem Cell Program at UC-Davis and a principal investigator for the neurodegenerative diseases grant, "Today’s award from CIRM gives us a terrific boost because it opens the door to developing an innovative and effective therapeutic tool for victims of neurodegenerative disorders like Huntington’s disease. Diseases of the brain are incredibly challenging to treat. What we hope to do is create a stem-cell-based treatment that can deliver therapeutic molecules directly into damged cells in the brain."

In the U.S., Huntington’s disease occurs with a frequency of one person per every 10,000 people. Dr. Nolta has been working with MSCs for the past 20 years, during which time the cells have demonstrated their vast therapeutic versatility. Under the new grant, Dr. Nolta and her colleagues intend to re-engineer the MSCs to deliver siRNA (small interfering ribonucleic acid) to damaged areas of the brain, where the siRNA can prevent the mutant proteins that are characteristic of Huntington’s disease from being produced by cells. Hoping to exploit the natural "search and rescue" capacity of MSCs to target damaged regions of the brain, Dr. Nolta expects that such a therapy will preclude the need for systemic infusion of cell-based therapies into the CNS (central nervous system).

According to Judy Roberson, president of the Northern California chapter of the Huntington’s Disease Society of America, "This is the best news. Families like mine have been waiting for an intervention for Huntington’s since the gene was located 16 years ago. My husband and his brother and thousands of others died waiting for something, anything. Now, thanks to CIRM support and the research at UC-Davis, there’s real hope for the first time."

In the second grant, a team of UC-Davis researchers led by Dr. Alice Tarantal will be exploring an in utero model to assess the viability of transplanted stem cells in the treatment of inherited blood disorders, before irreversible damage from the diseases has occurred. Professor of pediatrics and associate director of the UC-Davis Stem Cell Program, Dr. Tarantal explains, "Our team is poised to address crucial bottlenecks that have delayed these therapies from reaching our youngest patients. We greatly appreciate the support provided by CIRM to help us work toward these goals. Infants with congenital blood diseases can be diagnosed before they are born. We hope it may be possible to treat these young patients early and thus avoid the many problems such diseases can cause."

With more than 125 scientists and physicians on its faculty, UC-Davis is currently in the process of constructing a new Institute for Regenerative Cures in Sacramento, a facility that is supported by the CIRM and which will comprise 90,000 square-feet of state-of-the-art research laboratories and clinics.

Adult Stem Cells Treat Parkinson’s

At Jaslok Hospital in Mumbai, 55-year-old Bhawarlal Jain has received autologous adult stem cell therapy for the treatment of Parkinson’s disease. Mr. Jain has suffered from advanced symptoms of the disease for 6 years, which included spondylitis – inflammation of the vertebrae – and other joint pain since 2004. According to Jain, "I managed my business until my speech became so impaired that I had to repeat everything at least four times to be understood."

Then Mr. Jain came to Jaslok Hospital in February of this year, where a new clinical trial was being conducted in which ten Parkinson’s patients were being enrolled for autologous (in which the donor and recipient are the same person) adult stem cell therapy. In addition to enrolling in the study, Mr. Jain also became the first volunteer.

As he explains, "When I heard I was going to be the first human to be treated using stem cell transplant, I was excited and scared. I had told my family that I may never return." Ten days after receiving the stem cells, however, he is no longer afraid, and any doubts or trepidation that he may have had have now been replaced with optimism. As his wife, Sukhi, explains, "I was scared initially but now I am happy that we opted for this operation." As Dr. Pettarusp Wadia, a neurologist at Jaslok Hospital, adds, "All we did was tell him about the procedure and he volunteered himself."

Specifically, the procedure utilizes the type of adult stem cell known as mesenchymal stem cells (MSCs), which are harvested from each patient’s own bone marrow via the hip, and then returned to the patient therapeutically after being processed in the laboratory. According to Dr. Paresh Doshi, head of the team who performed the eight-hour-long procedure, "We chose the stem cells from the marrow of the patient’s hip bone as these cells are readily isolated. They can expand in culture and the body can accept them easily. Small quantities of these cells were injected at an interval of every one millimeter in his brain."

Although Mr. Jain is already showing significant improvement, he will continue to be monitored for the next 18 months.

Watching Adult Stem Cells Repair the Brain

Adult stem cells are consistently proving to offer a viable and efficacious therapy for a number of diseases and injuries, especially in the treatment of neurodegenerative diseases such as Alzheimer’s, Parkinson’s, and Huntington’s disease, among others. Now, researchers have visually observed, in real time, the regenerative power of adult stem cells in a new animal model of Huntington’s disease.

Led by Dr. Yoram Cohen of the School of Chemistry at Tel Aviv University (TAU) in Israel, scientists used mesenchymal stem cells (MSCs) derived from bone marrow to treat animals in whom the disease had been artificially induced. In a novel approach to the study, Dr. Cohen and his colleagues also utilized "in-vivo MRI" (magnetic resonance imaging) to track the progress of the MSCs in the brains of the animals. The cinematic result clearly demonstrated the natural ability of the MSCs to target damaged or unhealthy tissue and repair the degeneration.

As Dr. Cohen explains, "By monitoring the motion of these cells, you get information about how viable they are, and how they can benefit the tissue. We have been able to prove that these stem cells travel within the brain, and only travel where they are needed. They read the chemical signaling of the tissue, which indicates areas of stress. And then they go and try to repair the situation."

The in-vivo MRI was conducted at the Strauss Centre for Computational Neuro-Imaging, where Dr. Cohen and his colleagues were able to observe, in real time, the activity of the live, active MSCs, which had been labeled with magnetic iron oxide nanoparticles, thereby allowing the MSCs to be tracked throughout the brain.

As Dr. Cohen further explains, "Cells that go toward a certain position that needs to be rescued are the best indirect proof that they are live and viable. If they can migrate toward the target, they are alive and can read chemical signaling." Additionally, he adds, "Bone marrow-derived MSCs bypass ethical and production complications, and in the long run, the cells are less likely to be rejected because they come from the patients themselves. This means you don’t need immunosuppressant therapy."

In actuality, since MSCs are "immune privileged", "universal donor" cells, they do not pose any risk of immune rejection even when they are not derived from the same patient to whom the cells are administered. In other words, there is no risk of immune rejection even when MSCs are administered allogeneically (in which the donor and recipient are not the same person), therefore autologous (in which the donor and recipient are the same person) administration is unnecessary, as is immunosuppressant therapy even when the MSCs are administered allogeneically.

Nevertheless, the results of the study offer further concrete evidence for both the natural "homing" ability as well as the powerful regenerative capacities of MSCs, especially in the treatment of neurodegenerative diseases for which conventional medicine currently offers no known treatment or cure.

Adult Stem Cells Evaluated for the Treatment of Stroke

The biotech company Stemedica Cell Technologies has requested a pre-IND (investigational new drug) meeting with the U.S. FDA (Food and Drug Administration) for the purpose of discussing the use of Stemedica’s proprietary line of allogeneic adult stem cells as treatment for ischemic stroke.

Specializing in the R&D of adult stem cell therapies, Stemedical Cell Technologies has developed proprietary products composed of the type of adult stem cell known as mesenchymal stem cells (MSCs), which have already been shown in a number of independent studies to constitute a safe and efficacious therapeutic modality for a wide variety of injuries and diseases.

According to Nikolai Tankovich, M.D., Ph.D., president and chief medical officer of Stemedica, "Following a stroke, there is little therapy to offer patients to promote recovery other than physical, occupational, and speech therapy. Treatment with allogeneic mesenchymal bone marrow cells may offer new hope to patients with chronic neurological or age-related, neurodegenerative diseases." Indeed, since MSCs are known to be "immune privileged", "universal donor" cells, they pose no risk of immune rejection and therefore do not need to be administered autologously (in which the donor and recipient are the same person) but instead can be administered allogeneically (in which the donor and recipient are not the same person), as an "off-the-shelf" item.

The requested pre-IND meeting with the FDA is the preliminary step in the formal authorization process required before clinical trials can be initiated. According to Maynard Howe, Ph.D., vice chairmand and CEO of the company, "Stemedica’s facility will manufacture clinical grade stem cells as it prepares for FDA approval of an IND to begin clinical trials."

Incorporated in 2005 and headquartered in San Diego, Stemedica describes itself as a "specialty biopharmaceutical company" which, within the U.S., is "currently developing regulatory pathways for traumatic injury, stroke and wound repair." As further described on the company’s website, "Outside the United States, Stemedica provides its adult stem cells to hospitals and research centers that are conducting studies under protocols approved by the appropriate regulatory agencies. These studies are focused on the treatment of neurodegenerative disease, sight restoration and wound repair."

Adult Stem Cells Perk Up Pooch

Micki, an 11-year-old German Shepherd in Vancouver, Canada, had developed age-related osteoarthritis in 3 legs. As a result, the dog underwent a ligament operation that was not entirely successful and was accompanied by daily, heavy doses of anti-inflammatory medication, prescribed for indefinite use. Occasionally, Micki was in so much discomfort that a leg brace was required. When a second operation was prescribed for the ligament of a different leg, the dog’s owner, Sandy Draibye, a lawyer, decided that enough was enough. Instead of consenting to further surgery for her dog, Sandy opted for autologous adult stem cell therapy instead.

As Micki’s vet, Amanda Booth, explained, "This is her kid." Although Dr. Booth was personally unfamiliar with the stem cell treatment, she agreed to learn how to administer it anyway. She then harvested adipose (fat) tissue from the dog’s thigh and sent the tissue to the U.S. company Vet-Stem in California, where the stem cells are isolated and returned to Dr. Booth within 48 hours for injection directly into the dog’s injured ligaments and joints.

According to Dr. Booth, "If the other three legs had been in good shape, I would have held off on the stem cell surgery. I researched it before I agreed to do it. My first criterion was finding someone other than the company that says it works." After receiving a number of enthusiastic responses from other vets, Dr. Booth decided to take the plunge and begin acquiring her own experience with the procedure.

As previously reported a number of times on this website, the U.S. company Vet-Stem continues to see consistently high success rates in both canine and equine clinical applications, with an 80% efficacy rate and a 100% safety rate in the animals that are treated with Vet-Stem’s autologous adult stem cell procedure. In other words, 80% of the animals treated are found to experience improvement in their condition with a reduction and often a full elimination of the need for medication, while adverse side effects have not been reported in any of the treated animals.

Companies such as Vet-Stem in the U.S. and VetCell in the U.K. have accumulated numerous documented cases of the benefits of autologous adult stem cell therapy in animals. To name just a few of the advantages, adult stem cell therapy yields faster healing and shorter recovery times than surgical treatments do, and adult stem cell therapy does not pose a risk of any side effects like medications do. Additionally, since the adult stem cells are autologous, there is no risk of immune injection. The U.K. company VetCell derives the autologous adult stem cells from the animal’s bone marrow, and to date has treated approximately 1,700 horses with an 80% success rate. By comparison, the U.S. company Vet-Stem derives the autologous adult stem cells from the animal’s adipose (fat) tissue, and to date has treated over 2,000 dogs and over 3,000 horses, also with an 80% success rate. With both companies, the procedure is quick, simple, and minimally invasive. Although the treatment is more expensive than conventional veterinary procedures, the adult stem cell treatment actually works, and noticeable improvement is seen almost immediately in all cases, not just in the 80% of cases that exhibit a complete recovery. By sharp contrast, however, conventional surgical and pharmacological therapies, which might initially be less expensive than stem cell therapy, only have a 30% success rate and therefore in the long-term are actually more expensive when repeated treatment is needed, or when improvements are not seen at all. Additionally, reinjury is significantly lower in animals who receive autologous adult stem cell therapy, due to the mechanism of action by which these stem cells activate the healing process. As Dr. David Mountford, a veterinary surgeon and chief operating officer at VetCell, explains, "After 3 years, the reinjury rate was much lower in stem-cell-treated animals: about 23% compared with the published average of 56%" for animals treated with conventional therapies.

Not only do the stem cells automatically target the injured tissue, but they also stimulate other endogenous stem cells which in turn are mobilized into action and participate in the healing and repair process. Although improvements are usually dramatic and immediate, even after the first injection, additional injections may be necessary, depending upon the age and condition of the animal. Very few animals ever need more than a total or 2 or 3 treatments, however, before they are fully restored to their natural, pain-free state of mobility – which contrasts dramatically with conventional therapies such as most prescription medications which may need to be taken indefinitely, without ever producing any tangible signs of improvement and while even possibly causing further damage to the animal through dangerous side effects and other associated risks.

Ordinarily, injuries of the bones, joints, tendons and ligaments result in scarring of the tissue, which not only prevents full healing but also often leads to further injuries at a later time. Conventional medical therapies do nothing to address the problem of scar tissue directly, and surgical procedures actually make the problem worse by increasing the severity of tissue scarring which in turn merely exacerbates later complications that will inevitably result from the scar tissue, since such tissue can never be fully rehabilitated. Adult stem cell therapy, however, allows for the full and complete healing of tissue without scarring, which not only reduces the risk of re-injury of the same tissue at a later date but also restores full physical performance and function, usually very quickly and dramatically. Such is the case in humans as well as in animals. According to Dr. Robert Harman, veterinarian and founding CEO of Vet-Stem, "Our success in animals is directly translatable to humans, and we wish to share our evidence that stem cells are safe and effective." Additionally, adipose-derived stem cells have been shown in a number of studies to exhibit highly beneficial immunomodulatory properties – which reduce inflammation, among other benefits – in addition to stimulating the regeneration of cartilage and other tissue. (E.g., "Non-expanded adipose stromal vascular fraction cell therapy for multiple sclerosis", by N.H. Riordan et al., published in the Journal of Translational Medicine in April of 2009, of which Dr. Harman is a coauthor). As Dr. Harman further adds, "In the last couple of years, evidence has come out that the cells we use reduce inflammation and pain, and help lubricate the joint. About 200,000 hip replacements are done every year in humans. That’s a very good target for someone to look at cell therapy."

Vet-Stem uses exclusively adult stem cells, derived from each animal’s own tissue. Since the cells are autologous (in which the donor and recipient are the same animal), there is no risk of immune rejection. More specifically, the stem cells that are harvested in Vet-Stem’s procedure are mesenchymal stem cells, which are highly potent adult stem cells that are also found in bone marrow and umbilical cord blood. Numerous scientific and clinical studies have been published in the peer-reviewed medical and veterinary literature detailing the regenerative properties of mesenchymal stem cells.

No embryonic stem cells are ever used in Vet-Stem’s therapies, since embryonic stem cells are highly problematic in the laboratory, whether they are of human or non-human origin. Among other problems, the risk of teratoma (tumor) formation disqualifies embryonic stem cells for use as a clinical therapy, even in animals. Adult stem cells, however, do not pose such risks and are therefore rapidly accumulating a consistent history of successful clinical treatments in veterinary, as well as in human, medicine.

According to Sandy Draibye, owner of Micki, the 11-year-old German Shepherd, "It’s a lot of money, but she’s a lot of dog. I am not wealthy, but I can afford it. Everybody adores their dog. I don’t think they would give it a second thought."

In fact, as Sandy points out, most humans are so impressed by the news of such dramatic recovery in animals that people are becoming increasingly curious about the use of autologous adult stem cell therapy for the treatment of human ailments. As Sandy adds, "They’re wondering about their own joints."

Pro-Embryo Group Sues NIH

A pro-embryo group has filed a lawsuit against the National Institutes of Health (NIH) alleging that the new stem cell policy of the Obama administration violates federal law. According to the members of the group, they filed the court case in order to protect embryos. In fact, one of the plaintiffs listed in the filing is named simply as "embryos".

The lawsuit was filed in federal district court in Washington, D.C. in a 28-page-long document. Besides "embryos", additional plantiffs include Dr. James Sherley, Dr. Theresa Deisher, and Nightlight Christian Adoptions, each of whom is listed "individually and as next friend for Plantiff Embryos". Shayne and Tina Nelson, William and Patricia Flynn, and Christian Medical Association are also listed as plaintiffs, but not as "next friend for Plaintiff Embryos".

Dr. Deisher is director of R&D at AVM Biotechnology in Seattle, and Dr. Sherley is currently an adult stem cell researcher at the Boston Biomedical Research Institute although he is perhaps best remembered as the former Massachusetts Institute of Technology professor who went on a hunger strike in 2007 in order to protest a lack of faculty diversity at M.I.T. after he was denied tenure. Nightlight Christian Adoptions is an organization which encourages adoptions of left-over embryos from fertility clinics.

The defendants are listed as Kathleen Sebelius, Secretary of the Department of Health and Human Services, in addition to the Department of Health and Human Serivces itself, and Dr. Francis Collins, director of NIH, in addition to NIH itself.

Complaints filed in the lawsuit assert that the new federal policy on human embryonic stem cell research enacted by the Obama administration violates the Dickey-Wicker amendment. According to Drs. Sherley and Deisher, federal guidelines "authorize public funding of research that depends upon and, indeed, requires the destruction of living human embryos."

In March of last year, Nightlight and others filed a similar lawsuit in the U.S. District Court in the state of Maryland. According to Tony Mazzaschi of the Association of American Medical Colleges in Washington, D.C., the new lawsuit doesn’t stand "a chance in hell" of succeeding.

(Please see the numerous related articles on this website, including but not limited to: "Obama Decrees Changes in Embryonic Stem Cell Research, Though Not What One Might Expect", dated March 9, 2009; "Obama Rescinds Bush-Era Executive Order Pushing for More Ethical Stem Cell Research", dated March 10, 2009; "Obama Signs Law Restricting Federal Funding of Embryonic Stem Cell Research", dated March 11, 2009; "A High-Profile Proponent of Embryonic Stem Cell Research Sharply Criticizes Obama’s Policy", dated March 13, 2009; "Members of The President’s Council on Bioethics Object to Obama’s Stem Cell Policy", dated March 26, 2009; "NIH Issues Guidelines Restricting Embryonic Stem Cell Research", dated April 17, 2009; "Embryonic Stem Cell Advocates Protest NIH Guidelines", dated May 25, 2009; "Pros and Cons of the New NIH Guidelines", dated June 3, 2009; "NIH Receives Nearly 50,000 Comments", dated June 5, 2009; "Obama Scraps Bioethics Council", dated June 17, 2009; "NIH Issues New Guidelines", dated July 7, 2009; "New Director of NIH Chosen", dated July 8, 2009; "New NIH Stem Cell Guidelines are Slowing Research", dated July 14, 2009; and "Clinical Results From Embryonic Stem Cells are ‘Decades Away’", dated July 15, 2009, among other articles).

Embryonic Stem Cell Trial Delayed

In the latest chapter of the ongoing saga over the first clinical trial ever to be conducted with human embryonic stem cells (hESCs), a new delay has presented itself.

Specifically, representatives of the Geron Corporation have announced today that U.S. federal regulators have placed a halt on the clinical trial that Geron was scheduled to conduct, before even one patient could be enrolled in the study.

Although a number of people among the non-scientific communities have expressed surprise over what they consider to be an unexpected announcement, there are many among the scientific community who find such a delay to be completely unsurprising and even predictable.

Frought with doubts and problems from the very start, the clinical trial was intended to use hESCs in the treatment of spinal cord injury. Because it would have been the first such study ever to be conducted with hESCs, the proposed clinical trial has repeatedly been the subject of widespread speculation and controversy. Now, this new ruling by the FDA, which brings all the momentum of the entire project to a grinding halt, has merely incited a new round of speculation and controversy.

The U.S. federal regulators decided to impose a halt on the clinical trial as a direct result of new data that Geron disclosed to the FDA, regarding dose escalation in preclinical animal studies. In January of this year, the FDA lifted an additional halt that it had previously imposed on Geron in May of 2008. This time, the new halt is based on safety concerns triggered by the new higher-dose data.

According to Joseph Pantginis, an analyst with the Merriman Curhan Ford Group in New York, "They are at the forefront, which a lot of times is a bit of a curse. They have to hit all the hurdles and be the first one to learn about how these cells behave."

In an official statement issued by representatives of Geron, the company "will work closely with the FDA to facilitate their review of the new data and to release the clinical hold."

Anna Krassowska, a spokesperson for Geron, stated to the press that the company had no further comment.

The news release posted on Geron’s website was brief, at only two paragraphs in length, not counting the customary "forward-looking statements" and "safe harbor" disclaimers. As stated on the comapny’s website, "GRNOPC1, a cell therapy for neurologically complete, subacute spinal cord injury, has been placed on clinical hold by the FDA pending the agency’s review of new nonclinical animal study data submitted by the company. A clinical hold is an order that the FDA issues to a sponsor to delay a proposed trial or to suspend an ongoing trial. … No patients have yet been treated in this study."

As the New York Times article concludes, "While thousands of patients around the world have been treated with adult stem cells and have shown mixed results, no humans have been given cells derived from embryos in an approved trial."

Indeed, it would seem as though, once again, embryonic stem cells remain confined to the experimental, laboratory stage, unable to progress to clinical trials. Meanwhile, by comparison, numerous types of adult stem cells have already advanced to Phase III clinical trials and beyond, and in fact have already been in use as viable therapies in clinics around the world for years.

Adult Stem Cells Treat Heart Failure

At 68 years of age, Brent Benson was suffering from heart failure. As a biochemist who previously conducted tests for the Environmental Protection Agency, Benson was no stranger to physiology and medical science. By his own assessment, he says, "I’m essentially dying of heart failure. I was so far down, I had to try."

Now, however, approximately 5 months after receiving autologous adult stem cell therapy, Benson describes himself very differently. "My life is full and busy," he says. "I had lost that prior to this operation."

Autologous (in which the donor and recipient are the same person) adult stem cell therapy is catching on. Increasingly used in the treatment of a wide variety of diseases and injuries throughout the world, the procedure has shown such consistent success in patient improvement that the news finally seems to be spreading.

In Brent Benson’s particular case, he was able to participate in a clinical trial conducted at the University of Utah in Salt Lake City. After his own adult stem cells were extracted from his bone marrow via his hip, the cells were processed in a laboratory and later injected directly into his heart. He received a total of 30 injections, after which time his heart has exhibited considerable improvement, with his ejection fraction – a measure of the heart’s ability to pump blood – increasing from 15% to over 30%. While an ejection fraction between 55 and 70% is considered normal for healthy people, doctors expect Benson’s ejection fraction to continue to improve.

Heart failure is one of the most prevalent of all diseases, especially in developed nations. The American Heart Association estimates that in the U.S. alone there are more than 5.7 million people who live with heart failure, with approximately 670,000 new cases being diagnosed each year.

Dr. James Willerson is president of the Texas Heart Institute in Houston and a principal investigator of a separate clinical trial sponsored by NIH (the National Institutes of Health), also for the study of adult stem cell therapies in the treatment of heart failure. As Dr. Willerson explains, "When you put (adult) stem cells into a heart, some can differentiate to become blood vessels and others to become heart muscle cells." Additionally, Dr. Willerson adds, these types of adult stem cells, which are known as mesenchymal stem cells (MSCs), also contain growth factors and other beneficial "substances that recruit other cells and promote life," which makes the MSCs particularly efficacious as a clinical therapy. According to Dr. Willerson, "I believe we will be able to regenerate the whole heart of a human being with stem cells."

Dr. Douglas Losordo, director of the Program in Cardiovascular Regenerative Medicine at Northwestern University’s Feinberg School of Medicine, is conducting a pilot study in which 10 patients with heart failure are being treated with their own autologous adult stem cells, derived not from their bone marrow but instead from each patient’s own peripheral blood. As enthusiastic as he is about adult stem cell therapy, however, he is quick to point out that the field is not without its challenges. As Dr. Losordo explains, "Even as a wildly optimistic guy, I don’t imagine that anything I do on a single day in the cath lab is going to reverse 30 years of disease."

Similarly, Dr. Amit Patel, director of Cardiovascular Regenerative Medicine at the University of Utah and the leader of the study in which Mr. Benson is participating, believes that patients should not be shy about seeking adult stem cell treatment, but he also encourages them to conduct their own research and find a medical facility with specialists in a wide variety of fields, who can offer other forms of treatment in addition to adult stem cell therapy. According to Dr. Patel, "This is just one component of the entire continuum of heart failure treatment. It’s the sexiest one, but it’s only one component."

In a comment which is regarded by many as being more than just slightly odd, Dr. Patel also adds that, "With any cell-based therapy, there’s such a high placebo effect. There’s so much hype about the potential of stem cells. It’s positive reinforcement." In specific reference to Brent Benson, however, who lives at an altitude of 9,000 feet, Dr. Patel further acknowleges that, "Elevation, that’s the ultimate stress test." The fact that Benson has regained his energy and vigor following his adult stem cell therapy, while residing at such a high elevation, is concrete evidence for the powerful regenerative capacity of adult stem cells, and probably cannot be attributed solely to "the placebo effect". On the other hand, if someone with clinical heart failure can recover to the extent that their ejection fraction improves from 15% to more than 30%, for no other reason than "the placebo effect", then perhaps more attention should be paid to this grossly misunderstood phenomenon, and perhaps pharmaceutical companies might want to begin bottling and marketing "the placebo effect".

Whether inspite of or because of sudden, remarkable improvement in critically ill patients which is often explained as "just the placebo effect", Dr. Losordo adds that patients should not be afraid to pursue adult stem cell treatment, though he acknowledges that some patients have a difficult time knowing where or how to start. As he explains, "In my opinion, most patients are far too shy about picking up a phone and starting a line of inquiry. All the people involved in this stuff at medical centers, we want to help folks out."

In Brent Benson’s particular case, one of his cardiologists told him, "You don’t have a thing to lose. You don’t have much time left." For most people, a prognosis such as this would be enough to cure them of their shyness.

Not only is Benson now able to be physically active, without fatigue, while tending to the fruit trees on his farm and while also working on the deck at his home in Utah, at an eleveation of 9,000 feet, but also, as Benson now asserts, "I’ve gotten my sense of humor back."

Not bad, for "the placebo effect".

Wanted: Adult Stem Cell Donors

If you are between the ages of 18 and 60, in good health, and would like to do a good deed, the "Be The Match" registry would like to solicit the donation of your adult stem cells.

Operated by the National Marrow Donor Program, the Be The Match registry is the largest in the world, arranging approximately 4,300 transplants a year. Although originally begun for bone marrow donations, the registry has now expanded to include adult stem cell donations, for which the process is relatively simple, quick, and provides a valuable, life-saving service to others.

Upon signing up, new registrants will receive a collection kit in the mail, with which a swab of cheek cells may be collected and returned to the registry. Those registrants who are identified as a potential match will then be contacted for further testing.

Of particular interest to the registry are the type of adult stem cells known as hematopoietic stem cells, which are found in umbilical cord blood, peripheral blood, and bone marrow. Umbilical cord blood is the most easily collected, as it can be cryogenically stored immediately after a child’s birth. Bone marrow donation is the most difficult to collect, since it must be surgically extracted under general anesthesia, a process which is contraindicated in many patients who are already weakened by age or disease. The collection of adult stem cells from peripheral blood is performed by a process known as apheresis, in which blood that is removed from one arm is passed through an apparatus that extracts the stem cells and then returns the remaining blood cells to the other arm.

Currently approximately 60% of the registry’s transplant procedures involve adult stem cells that are derived from peripheral blood, while 20% of the procedures use adult stem cells derived from umbilical cord blood, and the remaining 20% of the procedures involve bone marrow.

As described on the website of the registry, "Donors never pay for donating and are never paid to donate. All medical costs are paid by the patient’s medical insurance or by the patient, sometimes with assistance from the National Marrow Donor Program (NMDP)." Nevertheless, the website also adds that, "The total cost to add a new member to the Be The Match Registry is about $100. This includes the cost of the testing needed to match donors to searching patients and related costs."

Individuals interested in donating their cells to the registry, or in finding a match for their own medical needs, may obtain more information at www.marrow.org.