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Breakthrough Stem Cell Treatment Could ‘Halt’ Multiple Sclerosis

September 19, 2016 Scientists in Canada have developed a radical treatment that can potentially ‘halt’multiple sclerosis in its tracks. The breakthrough treatment involves effectively destroying a person’s immune system and then building it back up using stem cells. While risky, the process could even reverse the symptoms of aggressive MS. The scientists have warned however that the treatment is dangerous and is only really likely to benefit a certain population of patients who are still in the early stages of the illness. MS occurs when the immune system strips nerve fibres in the brain and spinal cord of myelin, a fatty insulating material without which nerve signals cannot be transmitted properly. Symptoms range from blurred vision or tingling sensations to full blown paralysis. Usually the disease progresses to become more aggressive over time, but some patients find their health deteriorating rapidly. Doctors testing the therapy, known as IAHSCT (immunoablation and autologous hematopoietic stem cell transplantation), took stem cells from patients’ bone marrow and froze them before injecting powerful chemotherapy drugs to destroy the immune system. The stem cells were then transplanted back into the body to generate a new functional immune system with no “memory” of attacking the brain. Results over a period of up to 13 years were dramatic, with not one patient relapsing and 70% experiencing a complete halt in disease progression. In 40% of cases, patients saw lasting reversal of symptoms such as vision loss, muscle weakness and balance problems, the scientists reported in The Lancet medical journal. Some participants were able to return to work or school, regain the ability to drive, or get married and have children. Dr Harold Atkins, from the University of Ottawa in Canada, said: “Our trial is [...]

MS breakthrough: Replacing diseased immune system halts progression and allows repair

June 9, 2016 Clinical trial suggests chemo and blood stem cell combination therapy should be considered for people with early, aggressive MS A clinical trial published in The Lancet, a top medical journal, shows that an intensive procedure that completely wipes out the immune system and then regenerates a new one using blood stem cells can eliminate all signs of damaging brain inflammation in people with early, aggressive multiple sclerosis (MS), and facilitate lasting recovery. Led by Dr. Harold Atkins and Dr. Mark S. Freedman of The Ottawa Hospital and the University of Ottawa, the trial included 24 participants who were followed for up to 13 years. The $6.47 million trial was funded by the MS Society of Canada and its affiliated Multiple Sclerosis Scientific Research Foundation. The research was also supported by The Ottawa Hospital Foundation, The Ottawa Hospital Department of Medicine and Canadian Blood Services. People who are interested in this therapy should speak with their own neurologist, who can request a referral to the Ottawa Hospital MS Clinic or another major hospital with experience in this area. Noth that The Ottawa Hospital cannot treat people without valid Canadian health coverage. “Our trial is the first to show the complete, long-term suppression of all inflammatory activity in people with MS,” said Dr. Atkins, a stem cell transplant physician and scientist at The Ottawa Hospital, and associate professor at the University of Ottawa. “This is very exciting. However, it is important to note that this therapy can have serious side effects and risks, and would only be appropriate for a small proportion of people with very active MS. People with MS who have had significant disability for a long time would likely not benefit.” “This [...]

Exercise May Impact Reduced Disease Activity in Children with MS

Thu, 08/13/2015 - 8:33am Bevin Fletcher, Associate Editor Children with multiple sclerosis (MS) who get regular exercise may have a less active disease, according to a new study published online Wednesday in the journal Neurology. Children responded to questionnaires about tiredness, depression and exercise habits.  The study included 79 children who had experienced a single inflammatory neurological event and 31 children with MS.  MRI brain scans were conducted on 60 of those children to measure brain volume and the amount and type of MS lesions. “Up to three-quarters of children with MS experience depression, tiredness, or memory and thinking impairment,” said study author E. Ann Yeh, M.D., with The Hospital for Sick Children (SickKids) in Toronto. “Our research is important since little is known regarding how lifestyle behaviors may affect the disease. Overall, MS patients who participated in strenuous physical activity were associated with a less active disease, regarding the volume (amount) of lesions, and relapses per year.   There was no difference in whole brain volumes between children who partook in strenuous activity and those who did not, however those who did were more likely to have a lower overall volume of lesions that indicate disease activity, or T2 lesions.  Those that exercised had a median of 0.46 cm3 of T2 lesions, compared to 3.4 cm3 for those who did not.  The active children also had a median of 0.5 relapses per year, compared to one per year for those with no strenuous activity.   Less children (45 percent) with MS reported participating in strenuous activity compared to the other children (82 percent). Those with MS also reported higher levels of tiredness and depression compared to the others studied. “These findings add to the possibility that [...]

Major step for implantable drug-delivery device

MIT spinout signs deal to commercialize microchips that release therapeutics inside the body. Rob Matheson | MIT News Office June 29, 2015 An implantable, microchip-based device may soon replace the injections and pills now needed to treat chronic diseases: Earlier this month, MIT spinout Microchips Biotech partnered with a pharmaceutical giant to commercialize its wirelessly controlled, implantable, microchip-based devices that store and release drugs inside the body over many years. Invented by Microchips Biotech co-founders Michael Cima, the David H. Koch Professor of Engineering, and Robert Langer, the David H. Koch Institute Professor, the microchips consist of hundreds of pinhead-sized reservoirs, each capped with a metal membrane, that store tiny doses of therapeutics or chemicals. An electric current delivered by the device removes the membrane, releasing a single dose. The device can be programmed wirelessly to release individual doses for up to 16 years to treat, for example, diabetes, cancer, multiple sclerosis, and osteoporosis. Now Microchips Biotech will begin co-developing microchips with Teva Pharmaceutical, the world’s largest producer of generic drugs, to treat specific diseases, with licensing potential for other products. Teva paid $35 million up front, with additional milestone payments as the device goes through clinical trials before it hits the shelves. “Obviously, this is a huge validation of the technology,” Cima says. “A major pharmaceutical company sees how this technology can further their efforts to help patients.” Apart from providing convenience, Microchips Biotech says these microchips could also improve medication-prescription adherence — a surprisingly costly issue in the United States. A 2012 report published in the Annals of Internal Medicine estimated that Americans who don’t stick to prescriptions rack up $100 billion to $289 billion annually in unnecessary health care costs from additional hospital [...]

New technology looks into the eye and brings cells into focus

Photo by L. Brian Stauffer Dr. Stephen Boppart led a team that developed a new medical imaging device that can see individual cells in the back of the eye to better diagnose and track disease. From left: postdoctoral researcher Yuan-Zhi Liu, graduate student Fredrick A. South, and professor Stephen Boppart. 6/22/2015 | Liz Ahlberg, Physical Sciences Editor | CHAMPAIGN, Ill. — Eye doctors soon could use computing power to help them see individual cells in the back of a patient’s eye, thanks to imaging technology developed by engineers at the University of Illinois. Such detailed pictures of the cells, blood vessels and nerves at the back of the eye could enable earlier diagnosis and better treatment for degenerative eye and neurological diseases. New technology uses computational techniques to more clearly see individual rods and cones, the cells that detect light in the back of the eye | Graphic by Alex Jerez Roman The technique applies adaptive optics – the method astronomers use to correct telescope images so they can more clearly see stars beyond the twinkling – to the instruments that scan the retina at the back of the eye. However, the Illinois team does the correction computationally, instead of using complex hardware. Led by electrical and computer engineering professor Stephen Boppart, the research team published its work in the journal Nature Photonics. “The eye has always been a bit of a challenge to image. It’s a very complicated organ,” said Dr. Boppart, who also is a medical doctor. “There are many microscopic structures that are hard to see. Many diseases that affect vision also start at the microscopic level, so being able to see those early changes is going to lead to better, earlier [...]

Stem cells as the road to repairing Multiple Sclerosis

A clinical trial set to begin this month in Ottawa will test the safety and efficacy of mesenchymal stem cells to stimulate repair of damaged nerves in MS patients. This article was published simultaneously on Signals Blog. June3, 2015 By Lisa Willemse On any given day at the general campus of The Ottawa Hospital, you’re likely to find a pair of clinician-researchers who possess a keener knowledge of stem cells and their potential as a treatment for Multiple Sclerosis than just about anyone else in the country. Nearly 15 years ago, Drs. Mark Freedman and Harry Atkins, both members of the Ontario Institute for Regenerative Medicine, began work on a protocol that was intended to track the origins of MS by wiping out the patient’s compromised immune system (immunoablation) and rebooting it with a bone marrow (hematopoietic) stem cell transplant. The theory was that the disease would reassert itself and the researchers would be able to identify the earliest stages of MS, something largely unknown, due to the fact that most diagnoses of the disease occur years after initial onset. This knowledge could prove to be vital for Canadians, who have the highest incidence of MS in the world at 291 cases per 100,000. The study, however, didn’t work -- at least, not as expected. Drs. Freedman and Atkins were unable to discover the origins of MS because not a single patient in the study redeveloped the disease. For some, the level of physical function after treatment stayed the same, a bare few deteriorated further, some saw marginal or moderate functional improvement, and, remarkably, some regained almost all the physical abilities they had before diagnosis – but, no matter what their functional improvement, the disease [...]

Regenerative Medicine Study Underscores Lung Regeneration Capacity

April 28, 2015, Reid D'Amoco In diseases like cystic fibrosis, the lungs undergo constant healing and remodeling due to chronic infections. To better understand the repair mechanisms the lungs go through in diseases like CF and COPD, scientists have paid great attention to studying cellular regeneration. Scientists at the University of Pennsylvania and Duke University have discovered that mature lung cells have the ability to repair and differentiate into other types of cells found in the lungs. The primary author, Dr. Rajan Jain, published his findings earlier this month in Nature Communications. Type I cells in the lungs are responsible for the exchange of oxygen and carbon dioxide that occurs during breathing, and type II cells are responsible for secreting surfactants. Pulmonary surfactants are critical to lung function; they prevent drying of the airways, and allow the lungs to expand and contract with each breath. Studies performed in the 1960s and 1970s suggest that type II cells have the ability to regenerate into type I cells in the presence of damage, but Dr. Jain and his team have discovered that type I cells can also give rise to type II cells. These findings suggest that the lungs have much more flexible repairing mechanisms than previously thought. By exploring these mechanisms in greater detail, researchers can begin to understand how these mature lung cells already present within the body can be used to treat lung damage caused by diseases like CF. In lung diseases like cystic fibrosis and COPD, there is no cure, and patients only control their conditions through medications and treatments. By learning how to influence the lung’s ability to regrow or repair damaged tissue, the course of treatments for those with CF would drastically change. [...]

Gladstone Scientists Discover Potential New Treatment for Multiple Sclerosis

Finding could have implications for other autoimmune disorders, such as type I diabetes SAN FRANCISCO, CA—APRIL 27, 2015—Scientists from the Gladstone Institutes have discovered a way to prevent the development of multiple sclerosis (MS) in mice. Using a drug that blocks the production of a certain type of immune cell linked to inflammation and autoimmunity, the researchers successfully protected against the onset of MS in an animal model of the disease. The scientists say the next step is to test this strategy using other autoimmune disorders. “We are very excited about these findings,” says Eric Verdin, MD, a senior investigator at Gladstone and co-senior author on the study. “In light of the significant effect the treatment had on inflammation, the implications of these results will likely extend beyond multiple sclerosis to other types of autoimmune disorders. We are particularly interested in testing this in type I diabetes given the similar pathways involved, and we are already seeing very promising results in preliminary experiments.” In the immune system, two kinds of T cells strike a delicate balance—T helper cells (Th17) activate the immune system, protecting against infections and cancers, while regulatory T cells (Tregs) suppress the system, keeping it in check. A disparity between these cell types, when there are too many Th17 and not enough Tregs, can lead to a hyperactive immune system, resulting in inflammation, tissue damage, and autoimmune disease. In the current study, published in the Journal of Experimental Medicine, the researchers discovered that an important regulatory protein, sirtuin 1 (SIRT1), is involved in the production of Th17 cells. By blocking this protein, the scientists can protect against the onset of autoimmunity. SIRT1 also has a negative impact on Treg maturation and maintenance, so [...]

Drugs Lessen Multiple Sclerosis Damage in Mice

April 27, 2015 At a Glance In a mouse study, 2 drugs already on the market activated stem cells and repaired the type of brain damage seen in multiple sclerosis (MS). More research will be needed to determine whether the drugs could lead to novel MS therapies. MS is a disease in which a nerve-insulating compound called myelin is mistakenly attacked by the immune system. The breakdown of myelin disrupts communication between the brain and the body, leading to muscle weakness, numbness, and problems with vision, coordination, and balance. An oligodendrocyte lays myelin down around long nerve fibers. Illustration by Megan Kern, courtesy of Case Western Reserve University. What triggers the disease is unknown. Anti-inflammatory drugs, which modulate the immune system, can help prevent the episodic relapses common in MS. However, they’re less effective at preventing chronic disease and disability. An alternate approach might be to develop therapies that promote myelin repair. Myelin is laid down around nerve cells by specialized cells called oligodendrocytes in a process called myelination. Oligodendrocytes are derived from stem cells called oligodendrocyte progenitor cells (OPCs). OPCs multiply in the brains of MS patients, but for unknown reasons fail to form oligodendrocytes that can remyelinate nerve cells. A research team led by Dr. Paul J. Tesar at Case Western Reserve University and Dr. Robert Miller at George Washington University investigated whether drugs already approved for other uses could stimulate OPCs and boost myelination. The study was partially funded by NIH’s National Institute of Neurological Disorders and Stroke (NINDS). Results appeared online in Nature on April 20, 2015. The scientists began with mouse OPCs and screened compounds for their ability to boost production of a protein that marks oligodendrocytes. They tested more than 700 [...]

Drugs that activate brain stem cells may reverse multiple sclerosis

March 20, 2015 NIH-funded study identifies over-the-counter compounds that may replace damaged cells Two drugs already on the market — an antifungal and a steroid — may potentially take on new roles as treatments for multiple sclerosis. According to a study published in Nature today, researchers discovered that these drugs may activate stem cells in the brain to stimulate myelin producing cells and repair white matter, which is damaged in multiple sclerosis. The study was partially funded by the National Institute of Neurological Disorders and Stroke (NINDS), part of the National Institutes of Health. An artist’s representation of the study. Scientists found that certain drugs were able to promote remyelination in mouse models of multiple sclerosis. Image courtesy of Case Western Reserve University; Illustrator: Megan Kern Specialized cells called oligodendrocytes lay down multiple layers of a fatty white substance known as myelin around axons, the long “wires” that connect brain cells. Myelin acts as an insulator and enables fast communication between brain cells. In multiple sclerosis there is breakdown of myelin and this deterioration leads to muscle weakness, numbness and problems with vision, coordination and balance. “To replace damaged cells, the scientific field has focused on direct transplantation of stem cell-derived tissues for regenerative medicine, and that approach is likely to provide enormous benefit down the road. We asked if we could find a faster and less invasive approach by using drugs to activate native nervous system stem cells and direct them to form new myelin. Our ultimate goal was to enhance the body’s ability to repair itself,” said Paul J. Tesar, Ph.D., associate professor at Case Western Reserve School of Medicine in Cleveland, and senior author of the study. It is unknown how myelin-producing [...]