Scientists in Brazil have used adult stem cells harvested from umbilical cord blood to treat muscular dystrophy.

Rather than referring to one disease, the term “muscular dystrophy” actually refers to a group of hereditary disorders of genetic origin and varying severity, depending upon the degree to which the dystrophin gene is defective or absent. Located at Xp21, the dystrophin gene codifies dystrophin, an essential component in the protein complex that is responsible for the membrane stability of muscle cells. A complete absence of the gene causes more severe forms of the disease such as the Duchenne form (DMD), whereas the presence of a defective gene causes milder forms of the disease such as the Becker form (BMD).

In this study, which was condcuted by Dr. Tatiana Jazedje and colleagues at the Human Genome Research Center in Sao Paulo, Brazil, the scientists took CD34+ adult stem cells derived from umbilical cord blood and established co-cultures which combined the stem cells with myoblasts from a patient who had been diagnosed with DMD. The CD34+ stem cells were already known to differentiate into muscle cells and to express dystrophin in vivo, but Dr. Jazedje and her colleagues were the first to show that this particular progenitor cell is also capable of regenerating muscle dystrophin in vitro, as the stem cells were found to have differentiated into mature myotubes after 15 days, while dystrophin-positive regions were also detected through immunofluorescence analysis.

As the authors concluded in their article, “Our findings showed that umbilical cord blood CD34+ stem cells have the potential to interact with dystrophic muscle cells restoring the dystrophin expression of DMD cells in vitro. Although utilized within the context of DMD, the results presented here may be valid to other muscle-related therapy applications.”

Scientists in the U.K. have been forced to stop their research with human-animal hybrid experimentation because of a lack of research funds.

Less than a year after the controversial laboratory techniques were legalized in the U.K., funding agencies are now refusing to finance the research of human-animal hybrids. Although Britain has enjoyed the status of a world leader in this field, it is now feared that existing research projects may be brought to a final and permanent end within weeks.

The experimentation has proven to be highly controversial from an ethical perspective, since it fuses human cells with nonhuman mammalian eggs in a laboratory process which thereby creates a new species and a new living creature which is simultaneously both yet neither human and nonhuman. Numerous ethical debates over the procedure are still raging, and may or may not be a direct cause of the current funding drought. No specific explanation was given for the lack of funds, other than “competition” from other projects.

Two of the three license holders who are legally permitted to create hybrid embryos in the U.K. have been denied research funds, namely, Dr. Stephen Minger of King’s College London and Dr. Lyle Armstrong of the Center for Life at Newcastle University. The third license holder, Professor Justin St. John of Warwick University, is still in the process of preparing a grant proposal.

According to Dr. Minger, whose work has not yet started, even a year after his license was issued, “The problem has been a lack of funding. We haven’t been able to buy equipment, £80,000 to £90,000 worth. We put in a grant proposal last year but it wasn’t successful and we’re dead in the water. We’re discussing whether it is worth the time to re-submit our application. People reviewing grants may be looking at this from a completely different moral perspective, and how much that has influenced people’s perception about whether this should be funded, we don’t know.”

Dr. Armstrong of Newcastle University, who has created 278 hybrid embryos from human cells that were fused into cow ova, is now unable to continue working with the part-human, part-bovine embryos due to having been denied funding. According to Dr. Armstrong, “It seems a lot of effort for nothing. We are investigating other avenues to keep this work going but it is depressing that Britain seems happy to create a nice regulatory environment for this work but then not to provide money for it.”

The licenses were originally issued by the Human Fertilisation and Embryology Authority through the Human Fertilisation and Embryology Bill, which allowed for the legal creation of animal-human hybrid embryos for stem cell research and which was passed after much debate by Parliament in May of 2008 and backed by both Gordon Brown and David Cameron.

Apparently, even though the British Parliament may have finished debating this topic, it would seem as though the rest of the populace has not.

Stanford University scientists have announced the discovery of adult stem cells that are located in human testes and which exhibit a capacity for differentiation that appears to be similar to that of embryonic stem cells.

The discovery is the result of studies that were conducted on 19 men who were treated for infertility by Dr. Paul Turek of San Francisco. Testicular tissue samples that were obtained from the men yielded abundant quantities of stem cells which were later found to exhibit multipotency in their differentiation capability when injected into mice.

The discovery is similar to previous reports that were published in the journal Nature by a team of scientists at the University of Tubingen in Germany, who had reported similar results from stem cells isolated from the testes of mice and which were found to differentiate into a variety of mouse tissue types. This latest study, however, is among the first to be conducted in humans.

According to Dr. Renee A. Reijo-Pera, who led the team of scientists, “We have a battery of tools now and we’re moving rapidly down the long road toward their use in human medicine. I’m really amazed at the progress the science is making, and I’m certain we’ll be ready for clinical trials of some stem cell therapies within the next 5 to 10 years.”

According to Alan Trounson, president of the California Institute for Regenerative Medicine, the primary focus of which is funding for embryonic stem cell research, “This is extremely interesting and important work.”

Whether or not men will be eager to donate testicular tissue for the harvesting of stem cells, however, is yet to be seen.

Scientists have made a discovery with adult stem cells in the fruit fly, Drosophila melanogaster, which may have important implications for humans.

In a study led by Dr. Michael Buszczak, formerly of the Department of Embryology at the Howard Hughes Medical Institute Research Laboratories in Baltimore, Maryland, and currently in the Department of Molecular Biology at the University of Texas Southwest Medical Center in Dallas, researchers have reported the identification of a protease-encoding gene in Drosophila which is commonly required in germline, epithelial and intestinal stem cells.

Known as the histone H2B ubiquitin protease “scrawny” (scny) gene, the gene encodes a ubiquitin-specific protease and is a common requirement in stem cells within diverse tissue types, since such stem cells share the common need for a chromatin configuration that promotes self-renewal. Chromatin proteins contain the genetic instructions that direct cell function, and are known to regulate multiple types of stem cells.

Among other effects, Dr. Buszczak and his colleagues observed that mutant fruit flies who lacked functional copies of the scrawny gene suffered a premature loss of stem cells in various tissue types which included their skin, intestinal and reproductive tissue.

As the authors conclude in their paper, “Our findings suggest that inhibiting H2B ubiquitylation through ‘scny’ represents a common mechanism within stem cells that is used to repress the premature expression of key differentiation genes, including Notch target genes.”

Although the scrawny gene has only been identified in fruit flies thus far, similar genes are suspected of performing similar functions in other multicellular organisms such as humans. According to Dr. Allan C. Spradling, director of the Carnegie Institution’s Department of Embryology, “Our tissues and indeed our very lives depend upon the continuous functioning of stem cells, yet we know little about the genes and molecular pathways that keep stem cells from turning into regular tissue cells – a process known as differentiation. This new understanding of the role played by scrawny may make it easier to expand stem cell populations in culture, and to direct stem cell differentiation in desired directions.”

This is not the first time that important clinical therapies for people have been developed from research conducted on the humble fruit fly. Indeed, the unpretentious Drosophila melanogaster has played a central role in the advancement of human medical science for over the past century, ever since the American geneticist and embryologist, Dr. Thomas Hunt Morgan, began studying the common fruit fly in 1906 while at Columbia University. Although a few other scientists prior to Dr. Morgan had conducted experiments with Drosophila, Dr. Morgan became the first person to demonstrate, by studying successive generations of the fruit fly, that genes are transmitted from parents to offspring via chromosomes, which constitute the molecular mechanism of heredity. Such a discovery established the foundation for the entire field of modern genetics, and Dr. Morgan was awarded the 1933 Nobel Prize in Physiology or Medicine, “for his discoveries concerning the role played by the chromosome in heredity.” Later, when Dr. Morgan relocated to the California Institute of Technology, he established the Division of Biology at Cal Tech which subsequently produced 7 Nobel Prize winners. To this day, in honor of Dr. Morgan and over half a century after his death in 1945, the Genetics Society of America still awards the annual Thomas Hunt Morgan Medal to one of its members, for outstanding contributions to the field of genetics. As a direct result of Dr. Morgan’s discoveries, Drosophila melanogaster continues to serve as a “model organism” of study for genetics and developmental biology, and as such this fruit fly has yielded a number of important discoveries that are applicable to a variety of other species, not only humans. Currently Drosophila is still being studied as a genetic model for many of the most perplexing of human diseases such as diabetes, cancer, and a number of the neurodegenerative diseases including Alzheimer’s, Huntington’s and Parkinson’s diseases, among others.

Like all other species – whether flora or fauna, vertebrate or invertebrate – fruit flies have stem cells too. Unlike many species, however, fruit flies also exhibit a number of traits that lend themselves desirably toward scientific investigation, such as a fast reproductive cycle within a short lifespan, an ease of culturability in large numbers, and a genotype in which mutations are easily inducible and easily trackable by phenotype from one generation to the next. All things considered, fruit flies make a much more compliant and cooperative laboratory specimen to study than humans.

Imagine a high-tech sachet the size of a tea-bag, filled with very small beads, and each bead in turn contains thousands of adult stem cells. Then imagine that each adult stem cell has been individually bioengineered to produce a drug that is specifically designed to treat stroke. Although such a therapy may have originally began as the stuff of imagination, it is now a reality.

Developed by the British company Biocompatibles, these stem cell “beads” have already been used to treat a number of patients. A man in Germany who received the treatment last year following a stroke has already regained his speech and the use of his right arm, although such a recovery was considered so extraordinary that it has also generated some controversy.

The stem cells in the beads are human adult mesenchymal stem cells which have been obtained from healthy donors and “programmed” to secrete CM1, a synthetically produced replica of a naturally occurring protein, GLP-1, which has been found to prevent damaged brain cells from dying through its strong anti-apoptotic effect. The full delivery mechanism consists of the cluster of human adult stem cells encapsulated in alginate beads, which protect the stem cells from an immunological response by the patient, and which constitute a retrievable implant that is removed after 14 days.

According to Dr. Peter Stratford, chief scientific officer of Biocompatibles, “We use stem cells to produce the protein because we want a reasonably low level of protein, but constantly produced, within the injured site. So in patients with a hemorrhagic stroke we have access to the brain since the surgeons are going to remove the blood clots anyway, so we can put the cells into the place where they’re needed and produce the therapeutic protein over a 2 to 3 week period in exactly the right place, and prevent the surrounding cells and tissue from dying. Each one of the cell beads is about half a millimeter in diameter and contains a few thousand stem cells that are producing the protein. And all in all, there are a few thousand cells within the containment that we implant in the brain.”

The implant is expected to be available on the European market within 5 years.

A familiar Latin adage attributed to the second century Roman poet Juvenal states: “Mens sana in corpore sano” (a healthy mind in a healthy body). Nearly 2 millennia later, scientists are still discovering new scientific proof of such timeless wisdom.

The leading Harvard Medical School associate clinical professor of psychiatry, Dr. John J. Ratey, would like you to know that physical exercise offers a number of neurological and even intellectual benefits. Of course, most people were probably already aware of such a claim, at least intuitively if not scientifically, although they may not have fully understood the precise mechanisms underlying such phenomena. In his new book, Dr. Ratey sheds light on the compelling science behind this important topic by elucidating the many ways in which physical activity stimulates various parts of the brain, including, among other components, the brain’s own endogenous stem cells. These stem cells, which naturally reside within the brain throughout life, even into the advanced decades of adulthood, are capable of being prompted and directed in their formation of new brain cells by external stimuli such as physical exercise. In “Spark: The Revolutionary New Science of Exercise and the Brain”, Dr. Ratey explores the connection not just between physical and mental health, but between specific types of physical exercise and cognitive acuity.

For years, neurophysiologists have already been studying the various factors, of both genetic and environmental origin, that influence the constant re-patterning of neural network connections which in turn provide the cellular basis upon which new information and experiences are processed. Now Dr. Ratey offers further depth and breadth of insight into the cellular and molecular processes of the brain, and into the essential role that physical exercise plays in catalyzing such mechanisms. As the title suggests, the resulting message is nothing short of “revolutionary” in conveying the absolutely critical importance of physical activity.

Among other benefits, Dr. Ratey explains, physical exercise improves the “fitness” of the neocortex, which in turn improves mental agility and mood as well as cognitive processes that require attention, alertness and motivation. But not all physical activity is created equal, and some types are more effective than others at accomplishing specific goals. For the greatest intellectual benefit, aerobic exercise in combination with “complex activity” has been found to maximize “brain power”. According to Dr. Ratey, “A fast-paced workout boosts the production of a protein called brain-derived neurotrophic factor. I call it Miracle-Gro for the brain, and physical activity is one of the best ways to release this brain-nourishing protein. A workout at the gym or a brisk walk also seems to build better connections between brain cells. Studies show that regular physical activity may increase the production of cells in the hippocampus, the region of the brain involved in learning and memory. The end result is a brain that’s better able to perform in school, at home or on the job.” Underlying the continuous replenishing of brain cells are none other than the brain’s own stem cells, which are stimulated into action by physical exercise and without which new brain cells and connections between neurons could not be formed.

Dr. Ratey adds, “Aside from elevating endorphins, exercise regulates all of the neurotransmitters targeted by antidepressants. It wakes up the brain and gets it going and improves self-esteem, which is one component of depression. Exercise also boosts dopamine, which improves mood and feelings of wellness. Studies have shown that chronic exercise increases dopamine storage in the brain. The process of getting fit is all about building up your aerobic base. The more you work your heart and lungs, the more efficient they become at delivering oxygen to your body and brain.”

As Dr. Ratey further explains, “We need to change the way we think about exercise. We really need to understand that exercise keeps the brain functioning well, and then realize that it also happens to be good for the body. We tend to think about it the other way around, but in fact it readies the cells in the brain to be optimal. We are made to move and people aren’t moving anymore.”

Devotees of the “mind-body medicine” that was so popular in the 1990s will be particularly interested in this book by Dr. Ratey, which advances the mind-body connection an order of magnitude further by examining with rigorous scientific objectivity the inseparability of physical health and cognitive performance. Even for those people who may not fall into the category of Olympic athletes, however, Dr. Ratey still offers hope by encouraging the reader to find motivation in the knowledge that even moderate exercise can sharpen memory and improve mental function.

Since the publication of Dr. Ratey’s book, Harvard Medical School has begun offering seminars on the subject through their Department of Continuing Education. Other universities and health conscious organizations are following the trend.

The book is coauthored with Eric Hagerman and published by Little, Brown & Company, 2008.