In January of this year, to great fanfare and widespread publicity, the U.S. FDA (Food and Drug Administration) gave approval to the Geron Corporation for the commencement of clinical trials with human embryonic stem cells (hESCs). The trials, originally scheduled to begin this summer, were to be the first official U.S. government-approved clinical trials ever conducted with hESCs. On August 18, however, the FDA imposed a halt on the trials, even before the first patient could be enrolled.

Although Geron declined to offer a detailed comment at that time, representatives of the company have now finally issued a formal explanation. According to Geron officials, the FDA halt is the result of "non-proliferative cysts" that were found in preclinical animal studies.

Although the precise, scientific interpretation of "non-proliferative cysts" is highly debatable, nonscientists in the general public seem to be pacified by Geron’s extremely unscientific choice of words.

In actuality, this explanation contradicts initial reports in which Geron representatives attributed the FDA halt to "escalated dosage" that Geron scientists had reported in preclinical animal studies. (Please see the related news article on this website, entitled "Embryonic Stem Cell Trial Delayed", dated August 18, 2009, as originally reported in The New York Times). Now, however, "non-proliferative cysts" would seem to be a different explanation which is altogether entirely separate from "escalated dosage".

For those people with a modest understanding of scientific laboratory procedures, this latest statement by Geron does nothing to clarify matters at all, but, on the contrary, merely seems to obfuscate matters further. For the less scientifically inclined, however, any explanation at all would seem to be enough to restore full confidence and enthusiasm.

According to San Diego based WBB Securities analyst Stephen Brozak, "I think it provides people with a reasonable explanation. Everybody was afraid of the T-word, teratomas, and it clearly wasn’t that."

In fact, the results are not clear at all, and indeed, people have good reason to be cautious of "the T-word", since teratomas are, by definition, a required characteristic not only of embryonic stem cells but of all pluripotent stem cells in general. Since pluripotency is defined as the ability of a cell to differentiate into tissue from all 3 germ layers (the ectoderm, the mesoderm and the endoderm), the formation of a teratoma remains the universal laboratory standard by which embryonic and other pluripotent stem cells are identified. If a cell in the laboratory forms a teratoma, then it is, by formal definition, recognized to be a pluripotent stem cell – and this includes not only embryonic stem cells but also the more recently developed iPS (induced pluripotent stem) cells. Conversely, if a cell cannot form a teratoma in the laboratory, then it is recognized as not being pluripotent.

A very specific type of tumor, teratomas are known for their extremely hideous appearance since they often contain not only bones and organs but also hair and teeth. They appear, in fact, to be just like an embryo, though highly physiologically disorganized, as if all the bones and organs had been disassembled and randomly rearranged. Although teratomas are benign, in the sense that they do not metastasize, they can still pose a serious health hazard and can even be fatal if left untreated. A metastatic, malignant counterpart does exist, known as a teratocarcinoma, and in fact when early teratomas are first detected in the laboratory it is impossible to know at such a primitive stage whether the tumor is the benign or the malignant version. It is therefore a realistic possibility that pluripotent stem cells can cause the formation of malignant teratocarcinomas as well as the benign teratomas.

Since embryonic stem cells (ESCs) are required, by the formal definition of pluripotency, to cause the formation of teratomas, a major concern with medical therapies based upon ESCs is that such therapies should, logically and predictably, also form teratomas in the patients who receive such therapies. Indeed, exactly how, or if, one might be able to flip off the cellular and molecular "switches" that cause pluripotent cells to form teratomas remains a hotly debated point among stem cell scientists.

By sharp contrast, however, adult stem cells are not pluripotent and therefore are not capable of forming teratomas, unlike ESCs and iPS cells. Although such a lack of pluripotency was originally seen as a disadvantage of adult stem cells, it is now widely acknowledged to be a major advantage and one of the primary reasons why adult stem cells have already been in clinical use as clinical therapies, safely, for years, without even one teratoma ever being reported as a side effect.

Geron did not offer a detailed elaboration of the precise features of these "non-proliferative cysts" which were found to form in laboratory animals who received Geron’s novel, proprietary ESC therapy. Teratomas, of course, being nonmalignant, could also be described as "non-proliferative"; furthermore, teratomas are often routinely referred to as "cysts", especially within the medical community itself. It is not uncommon, for example, when teratomas form naturally on or around the ovaries, for a gynecologist to tell the patient that a "cyst" needs to be surgically removed, when in fact the "cyst" is a teratoma. Nevertheless, "cyst" is the more common, colloquial term that is more often used in casual discourse between doctor and patient.

It is not surprising, therefore, that many scientists remain skeptical about Geron’s claim that these "non-proliferative cysts" are not, in fact, the dreaded "T-word", teratomas. There are also many scientists who are wondering why it took Geron so long to issue a formal explanation, thereby apparently deliberately perpetuating the initial reports which cited "escalated dosage" as the reason for the FDA halt. Especially when the formal explanation that is now released to the press consists of such a simple, and simplistic, reason as the appearance of "non-proliferative cysts", many scientists are left wondering why it was exactly that Geron took so long to get around to releasing this explanation to the public.

Allowing separate, independent laboratories to analyze the cysts, and to check specifically for the differentiation of cells into all 3 germ layers, could settle the skepticism once and for all. It could also clarify the apparently erroneous perception, carelessly propagated throughout the public, that "escalated dosage" had something to do with the FDA’s reason for halting Geron’s clinical trial. Thus far, however, Geron has indicated no interest in allowing such a clarification.

Meanwhile, it would appear as though financial analysts are not necessarily scientists, and Stephen Brozak, among others, have now given Geron the new rating of a "strong buy" precisely as a result of Geron’s own description of "non-proliferative cysts". Consequently, shares of Geron’s stocks rose 25 cents, or 3.6%, to $7.18 at the close of trading today – up significantly after having fallen 10% as a result of the FDA halt that was announced on August 18.

According to Joseph Pantginis, an analyst at Merriman Curhan, "I believe that since the worst case scenario did not occur, investors are relieved." In fact, it is not at all known whether or not "the worst case scenario" did or did not occur. Until independent laboratories can check specifically for cells from all 3 germ layers, it cannot be known with any reliability or certainty whether or not these "non-proliferative cysts" are teratomas. Such a stance has nothing whatsoever to do with Geron in particular but rather it is a fundamental premise of the scientific method in general, which is founded upon repeatability and independent verification, without which, claims are meaningless – regardless of who makes such claims and regardless of whether the claims pertain to medical science or to the physics of planetary motion or to any other natural phenomenon.

Nevertheless, Pantginis was quick to add that Geron is not completely out of the woods just yet, as he points out that, "The potential timing of a release of the clinical hold is a complete black box and cannot be projected."

After all, the U.S. FDA actually employs scientists, some of whom might want to see further, independently verified evidence for the absence of cells from all 3 germ layers in these "non-proliferative cysts", before any proclamation about teratomas can be made.

South Korean prosecutors have told a Seoul court that Hwang Woo-suk should receive a four-year prison sentence for the scientific fraud that he committed in 2004 and 2005.

In addition to violation of bioethics laws, Hwang is also being charged with misappropriation of 2.8 billion won (approximately 2.25 million USD) in state funds. Although Hwang has apologized for his fraudulent reports, he has denied personal culpability, claiming that he was misled by junior researchers in his laboratory. Nevertheless, as one of the prosecutors explains, "The disappointment felt by the Korean people is enormous."

Formerly considered to be one of the world’s leading experts in the field of stem cell research, Hwang suffered a catastrophic fall from the prestige that he had previously enjoyed when he was found, in November of 2005, to have fraudulently reported the successful cloning of human embryonic stem cells. Specifically, he had authored two articles which were published in the highly respected journal Science in 2004 and 2005, and which initially catapulted Hwang into the international spotlight for his reports of the world’s first successful creation of human embryonic stem cells by cloning. The articles were later editorially retracted, however, when they were discovered to contain fabricated data, and Hwang quickly fell from fame to infamy. He subsequently confessed to charges of fraud, and was "indicted on embezzelment and bioethics law violations linked to faked stem cell research", according to media reports at the time. Additionally, he was expelled from the Seoul National University which had previously employed him, and the S. Korean government rescinded its financial support of his research and banned him from conducting further experiments on human cloning. To this day, the scandal has impacted S. Korean national law, which brought a formal halt to all experimentation on human cloning until this year. As Dr. Oh Il-hwan, a bioethics professor at the Catholic University Medical School in Korea, explains, "Hwang’s fall discouraged the government from supporting stem cell studies. It also meant that researchers in the field were the objects of scorn." Nevertheless, an investigational team at Seoul National University did verify that Hwang’s team had successfully produced the world’s first cloned dog, an Afghan hound named “Snuppy”.

Hwang is not without his supporters, who have staged high-profile, emotional rallies in the court, imploring a verdict that will allow Hwang a second chance. According to Lee Bong-gu, a lawyer for Hwang, "The prosecutors are trying to tear apart Hwang’s precious scientific evidence."

In 2006, with financial backing from his supporters, Hwang founded Sooam Biotech Research Foundation, which specializes in the cloning of animals and has produced several cloned dogs.

Hwang’s trial has been dragging on for three years and is predicted to continue into a fourth year, having been bogged down by technical testimony from numerous scientists who have testified about Hwang’s research. It is not known when a final verdict might be decreed.

Shannon Miller, a 7-time Olympic Medalist and 9-time World Medalist, has signed a formal endorsement with StemCyte, one of the leading cord blood banks in the world. According to the terms of the agreement, Ms. Miller will be featured in a series of public awareness and advertising campaigns for the company.

In addition to her accomplishments as an Olympic gymnast, Ms. Miller is also a lawyer, having graduated in 2007 from Boston College Law School. She and her husband, John Falconetti, are expecting their first child in November of this year.

According to Ms. Miller, "As a former Olympic gold medalist, I’ve always sought out the best and healthiest decisions in life. As my husband and I have begun planning our family’s future, we have been thoroughly investigating the finest decisions for our baby. Cord blood banking was a no-brainer for us, and StemCyte was the obvious choice for where we wanted to store our baby’s cord blood."

In a statement made by Calvin Cole, vice president of operations in North America for StemCyte, "We were thrilled when Shannon approached StemCyte. We felt that as a world class athlete, expecting mom and role model for millions around the world, she was a perfect fit for StemCyte. As a decorated athlete who knows what it takes to be the best in the world, we’re thrilled that she clearly recognized StemCyte as the world’s best choice for parents and their cord blood banking decisions."

Headquartered in California and New Jersey, StemCyte also has facilities in India and Taiwan. As stated in their press release, StemCyte has supplied over 1,000 cord blood products for over 40 life-threatening diseases to over 200 leading worldwide transplant centers. StemCyte is also involved in the development of new umbilical cord-blood-based therapies, and is supporting the largest clinical study for the use of unrelated cord blood transplantation in the treatment of thalassemia, one of the most common genetic diseases in the world. Additionally, StemCyte is also supporting trials investigating regenerative spinal cord therapies. StemCyte has been contracted by the U.S. Department of HHS (Health and Human Services) to help establish a public National Cord Blood Inventory for unrelated transplant units.

Researchers at the University of Wisconsin at Madison are once again in the news headlines. This time, they have reprogrammed skin cells into retinal cells. The implications of such an accomplishment offer new hope to millions of people who suffer from vision problems.

Led by Drs. David Gamm and Jason Meyer, the team of researchers took ordinary fibroblasts and de-differentiated them into a more primitive state known as iPS (induced pluripotent stem) cells, a procedure which is becoming increasingly common in research laboratories throughout the world. The iPS cells, which resemble embryonic stem cells in their pluripotency, were then re-differentiated into retinal cells. Additionally, the scientists also compared the differentiation of iPS cells into retinal cells with the differentiation of embryonic stem cells into retinal cells, and both processes appeared to progress through the same stages over the same amount of time. Both types of cells developed first into neural cells, which then differentiated further into cells of the primitive eye field, which in turn differentiated further into the more specialized cells of the retina, which is the light-sensitive, image-forming membrane on the inside of the eye, without which vision is not possible. The final cells included retinal pigment epithelium cells as well as photoreceptor cells, both of which are commonly lost in diseases of the eye such as retinitis pigmentosa and age-related macular degeneration, the latter of which is estimated to afflict approximately 30 million people around the world, and which in the U.S. is the leading cause of blindness in people 55 years of age and older.

As Dr. Gamm explains, "If you understand and follow every stage of building a photoreceptor, you’re more confident because you’ve observed the assembly line. We can understand the disease better and see if there are ways to protect the cells or slow down the disorder." It is still not yet known, however, if these new retinal cells can actually function in vivo in the same manner as naturally occurring retinal cells. As Dr. Meyer adds, "That is among the next projects we’re working on."

First pioneered in 2007, simultaneously by Dr. James Thomson of the UW-Madison as well as by Dr. Shinya Yamanaka at Kyoto University in Japan, iPS cell technology is now considered to be one of the most promising fields in regenerative medicine. With the same pluripotency as embryonic stem cells, but without any of the ethical dilemmas and controversies, iPS cells seem to be able to do anything and everything that embryonic stem cells can do.

According to Dr. Robert Lanza, chief scientific officer at Advanced Cell Technology, who did not participate in the current study but has conducted similar research, "This is an important paper. It underscores the enormous potential of iPS cells, not only for treating diseases such as blindness, but as an important tool for understanding eye development. I think it will only be a matter of time before these iPS-derived cells are used in the clinic to help restore vision in patients."

Indeed, according to Dr. Gamm, who believes that clinical trials for retinal cell replacement could be conducted within the next 5 years, "That’s not pie in the sky anymore. I couldn’t have said that, even two years ago."

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.

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.