Category Archives: Stem Cell Videos

Public release date: 28-Jun-2012 [ | E-mail | Share ]

Contact: Stephanie Desmon sdesmon1@jhmi.edu 410-955-8665 Johns Hopkins Medical Institutions

Johns Hopkins researchers, working with an international consortium, say they have generated stem cells from skin cells from a person with a severe, early-onset form of Huntington's disease (HD), and turned them into neurons that degenerate just like those affected by the fatal inherited disorder.

By creating "HD in a dish," the researchers say they have taken a major step forward in efforts to better understand what disables and kills the cells in people with HD, and to test the effects of potential drug therapies on cells that are otherwise locked deep in the brain.

Although the autosomal dominant gene mutation responsible for HD was identified in 1993, there is no cure. No treatments are available even to slow its progression.

The research, published in the journal Cell Stem Cell, is the work of a Huntington's Disease iPSC Consortium, including scientists from the Johns Hopkins University School of Medicine in Baltimore, Cedars-Sinai Medical Center in Los Angeles and the University of California, Irvine, as well as six other groups. The consortium studied several other HD cell lines and control cell lines in order to make sure results were consistent and reproducible in different labs.

The general midlife onset and progressive brain damage of HD are especially cruel, slowly causing jerky, twitch-like movements, lack of muscle control, psychiatric disorders and dementia, and eventually death. In some cases (as in the patient who donated the material for the cells made at Johns Hopkins), the disease can strike earlier, even in childhood.

"Having these cells will allow us to screen for therapeutics in a way we haven't been able to before in Huntington's disease," says Christopher A. Ross, M.D., Ph.D., a professor of psychiatry and behavioral sciences, neurology, pharmacology and neuroscience at the Johns Hopkins University School of Medicine and one of the study's lead researchers. "For the first time, we will be able to study how drugs work on human HD neurons and hopefully take those findings directly to the clinic."

Ross and his team, as well as other collaborators at Johns Hopkins and Emory University, are already testing small molecules for the ability to block HD iPSC degeneration. These small molecules have the potential to be developed into novel drugs for HD.

The ability to generate from stem cells the same neurons found in Huntington's disease may also have implications for similar research in other neurodegenerative diseases such as Alzheimer's and Parkinson's.

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Turning skin cells into brain cells

HOUSTON -

Pandu, the 286-pound Malayan tiger stretched out on the gurney in the Houston Zoo's hospital, had bone chips big ones in his right elbow.

Ivy the leopard, being prepped in another room, also needed medical treatment for her limp. The zoo's 68-pound black cat, which had arthroscopic surgery in 2009, was showing signs of pain again in her elbows.

The zoo staff was worried.

"I imagine we are going to end up euthanizing her at some point if it can't be fixed," said Beth Schaefer, the zoo's curator of carnivores and primates.

With two big cats needing attention, surgeon Brian Beale of Gulf Coast Veterinary Specialists and stem-cell specialists at InGeneron Inc. donated their services to treat the animals. While Beale removed bone chips and cleaned the joints during arthroscopic surgery, InGeneron staffers produced stem cells from each animal's body fat.

When the surgeries were complete, Beale injected the stem cells, which had taken about two hours to process for each big cat, into the animals' joints to promote faster healing.

Pandu, always a big baby looking for attention, was moving a bit slowly the day after surgery.

Feisty Ivy pretended nothing was wrong.

A week after the surgery, Houston Zoo veterinarian Lauren Howard says neither animal has suffered complications. Pandu, with mild swelling, was released into his exhibit half-days on Monday.

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Stem cells help some ailing Houston Zoo creatures

ScienceDaily (June 27, 2012) University of British Columbia scientists, in collaboration with an industry partner, have successfully reversed diabetes in mice using stem cells, paving the way for a breakthrough treatment for a disease that affects nearly one in four Canadians.

The research by Timothy Kieffer, a professor in the Department of Cellular and Physiological Sciences, and scientists from the New Jersey-based BetaLogics, a division of Janssen Research & Development, LLC, is the first to show that human stem cell transplants can successfully restore insulin production and reverse diabetes in mice. Crucially, they re-created the "feedback loop" that enables insulin levels to automatically rise or fall based on blood glucose levels. The study is published online June 27 in the journal Diabetes.

After the stem cell transplant, the diabetic mice were weaned off insulin, a procedure designed to mimic human clinical conditions. Three to four months later, the mice were able to maintain healthy blood sugar levels even when being fed large quantities of sugar. Transplanted cells removed from the mice after several months had all the markings of normal insulin-producing pancreatic cells.

"We are very excited by these findings, but additional research is needed before this approach can be tested clinically in humans," says Kieffer, a member of UBC's Life Sciences Institute. "The studies were performed in diabetic mice that lacked a properly functioning immune system that would otherwise have rejected the cells. We now need to identify a suitable way of protecting the cells from immune attack so that the transplant can ultimately be performed in the absence of any immunosuppression."

The research was supported by the Canadian Institutes of Health Research, the Stem Cell Network of Canada, Stem Cell Technologies of Vancouver, the JDRF and the Michael Smith Foundation for Health Research.

Diabetes results from insufficient production of insulin by the pancreas. Insulin enables glucose to be stored by the body's muscle, fat and liver and used as fuel; a shortage of insulin leads to high blood sugar that raises the risk of blindness, heart attack, stroke, nerve damage and kidney failure.

Regular injections of insulin are the most common treatment for the type 1 form of this disease, which often strikes young children. Although experimental transplants of healthy pancreatic cells from human donors have shown to be effective, that treatment is severely limited by the availability of donors.

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Diabetes reversed in mice using stem cells

VANCOUVER In a world's first, University of B.C. scientists have used human embryonic stem cell transplants to reverse diabetes in mice.

A 13-member team, whose work was published Wednesday in the journal Diabetes, showed that as the stem cells matured into insulin-secreting cells (beta-cells in the pancreas), a few dozen diabetic mice were weaned gradually off insulin over a period of months.

The study, which cost at least $500,000, was funded by the Canadian Institutes of Health Research, the Stem Cell Network of Canada, Stem Cell Technologies of Vancouver, the Juvenile Diabetes Research Foundation and the Michael Smith Foundation of Health Research. About half the research team was comprised of scientists from the New Jersey private research and development arm (BetaLogics Venture) of Janssen Pharmaceuticals.

"It took about four to five months for the (stem) cells to become functional in our experiments and the mice were able to maintain good blood glucose levels even when fed a high-glucose diet," said lead author Timothy Kieffer, a UBC professor in the department of cellular and physiological sciences.

Type 1 otherwise known as juvenile diabetes is an autoimmune disease in which a patient's immune system kills off insulin-producing cells in the pancreas. Typically, patients must inject themselves with insulin or use insulin pumps to control their blood glucose levels.

While pancreatic islet cell transplantation pioneered at the University of Alberta several years ago has been shown to be an effective way of reducing dependence on insulin injections, such treatments are costly and cumbersome since they require cells culled from dead bodies; such cells are always in short supply. As well, islet cell transplant patients must forever take anti-rejection drugs that can cause organ damage.

Although the research showed that stem cells have great potential as a diabetes cure, it also revealed there are still a few pitfalls to overcome before agencies like the Food and Drug Administration in the United States or Health Canada approve such a therapy. Some mice developed bone or cartilage growths in areas where the cells were inserted, an unacceptable side-effect that future experiments must resolve.

Another obstacle is that the mice used in the study weren't typical; they were a special strain, bred to be immuno-compromised so they wouldn't reject the human cells as foreign invaders. Studies are continuing at UBC, in many more mice, to determine the feasibility of encapsulating stem cells in a membrane material that won't be recognized as a foreign body and rejected.

Kieffer said he's extremely encouraged by the fact that the mice not only were weaned off their need for insulin but also lived well and long, even though they were bred to be immune-deficient. Still, he said, researchers must find ways to fine-tune the approach so that cells don't evolve into something other than what's desired.

In the early stages of the experiment, some mice developed fluid-filled cysts, a problem that was rectified in the laboratory with a cell culture medium change.

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Canadian scientists use stem cells to reverse diabetes in mice

ScienceDaily (June 27, 2012) Stem cells from patients with a rare form of muscular dystrophy have been successfully transplanted into mice affected by the same form of dystrophy, according to a new study published June 27 in Science Translational Medicine.

For the first time, scientists have turned muscular dystrophy patients' fibroblast cells (common cells found in connective tissue) into stem cells and then differentiated them into muscle precursor cells. The muscle cells were then genetically modified and transplanted into mice.

The new technique, which was initially developed at the San Raffaele Scientific Institute of Milan and completed at UCL, could be used in the future for treating patients with limb-girdle muscular dystrophy (a rare form in which the shoulders and hips are primarily affected) and, possibly, other forms of muscular dystrophies.

Muscular dystrophies are genetic disorders primarily affecting skeletal muscle that result in greatly impaired mobility and, in severe cases, respiratory and cardiac dysfunction. There is no effective treatment, although several new approaches are entering clinical testing including cell therapy.

In this study, scientists focused on genetically modifying a type of cell called a mesoangioblast, which is derived from blood vessels and has been shown in previous studies to have potential in treating muscular dystrophy. However, the authors found that they could not get a sufficient number of mesoangioblasts from patients with limb-girdle muscular dystrophy because the muscles of the patients were depleted of these cells.

Instead, scientists in this study "reprogrammed" adult cells from patients with limb-girdle muscular dystrophy into stem cells and were able to induce them to differentiate into mesoangioblast-like cells. After these 'progenitor' cells were genetically corrected using a viral vector, they were injected into mice with muscular dystrophy, where they homed-in on damaged muscle fibres.

The researchers also showed that when the same muscle progenitor cells were derived from mice the transplanted cells strengthened damaged muscle and enabled the dystrophic mice to run for longer on a treadmill than dystrophic mice that did not receive the cells.

Dr Francesco Saverio Tedesco, UCL Cell & Developmental Biology, who led the study, said: "This is a major proof of concept study. We have shown that we can bypass the limited amount of patients' muscle stem cells using induced pluripotent stem cells and then produce unlimited numbers of genetically corrected progenitor cells.

"This technique may be useful in the future for treating limb-girdle muscular dystrophy and perhaps other forms of muscular dystrophy."

Professor Giulio Cossu, another UCL author, said: "This procedure is very promising, but it will need to be strenuously validated before it can be translated into a clinical setting, also considering that clinical safety for these "reprogrammed" stem cells has not yet been demonstrated for any disease."

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Successful transplant of patient-derived stem cells into mice with muscular dystrophy

Public release date: 27-Jun-2012 [ | E-mail | Share ]

Contact: Bridget Dempsey b.dempsey@qmul.ac.uk 44-207-882-7927 Queen Mary, University of London

Scientists at Queen Mary, University of London have uncovered a link between two genes which shows how stem cells could develop into cancer.

The research, published in the online journal PLoS ONE, found a novel mechanism which could be the catalyst for stem cells changing into a tumour.

Dr Ahmad Waseem, a reader in oral dentistry at Queen Mary, University of London who led the research, said: "It was quite an unexpected discovery. We set out to investigate the role of the stem cell gene Keratin K15 which was thought to be a biomarker for normal stem cells.

"Through our research, we discovered there was link between K15 and the notorious cancer gene FOXM1. We found FOXM1 could target K15 to induce cancer formation."

Cancer develops when there is a problem with stem cells; the cells that carry out internal repairs throughout the human body. The loss of stem cell function leads to uncontrolled growth which ultimately develops into a tumour.

The team went through a process where they used extremely sensitive cell and molecular approaches to establish this link.

The study, funded by the Facial Surgery Research Foundation, Saving Faces, paves the way towards identifying new anti-cancer drugs which could be tailored towards cancer stem cells.

Consultant oral and maxillofacial surgeon Professor Iain Hutchison, founder of Saving Faces and co-author on the study, said: "We are excited about this finding as it could lead to more effective cancer drugs being developed to target cancer stem cells and prevent cancer recurrence."

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Scientists identify new cancer stem cell mechanism

Public release date: 27-Jun-2012 [ | E-mail | Share ]

Contact: Brian Kladko brian.kladko@ubc.ca 604-827-3301 University of British Columbia

University of British Columbia scientists, in collaboration with an industry partner, have successfully reversed diabetes in mice using stem cells, paving the way for a breakthrough treatment for a disease that affects nearly one in four Canadians.

The research by Timothy Kieffer, a professor in the Department of Cellular and Physiological Sciences, and scientists from the New Jersey-based BetaLogics, a division of Janssen Research & Development, LLC, is the first to show that human stem cell transplants can successfully restore insulin production and reverse diabetes in mice. Crucially, they re-created the "feedback loop" that enables insulin levels to automatically rise or fall based on blood glucose levels. The study is published online today in the journal Diabetes.

After the stem cell transplant, the diabetic mice were weaned off insulin, a procedure designed to mimic human clinical conditions. Three to four months later, the mice were able to maintain healthy blood sugar levels even when being fed large quantities of sugar. Transplanted cells removed from the mice after several months had all the markings of normal insulin-producing pancreatic cells.

"We are very excited by these findings, but additional research is needed before this approach can be tested clinically in humans," says Kieffer, a member of UBC's Life Sciences Institute. "The studies were performed in diabetic mice that lacked a properly functioning immune system that would otherwise have rejected the cells. We now need to identify a suitable way of protecting the cells from immune attack so that the transplant can ultimately be performed in the absence of any immunosuppression."

The research was supported by the Canadian Institutes of Health Research, the Stem Cell Network of Canada, Stem Cell Technologies of Vancouver, the JDRF and the Michael Smith Foundation for Health Research.

Diabetes results from insufficient production of insulin by the pancreas. Insulin enables glucose to be stored by the body's muscle, fat and liver and used as fuel; a shortage of insulin leads to high blood sugar that raises the risk of blindness, heart attack, stroke, nerve damage and kidney failure.

Regular injections of insulin are the most common treatment for the type 1 form of this disease, which often strikes young children. Although experimental transplants of healthy pancreatic cells from human donors have shown to be effective, that treatment is severely limited by the availability of donors.

###

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Stem cells can beat back diabetes: UBC research

A mother with three daughters who have Fanconi anemia sued the federal government for the right to compensate bone marrow donors. The U.S. Attorney General will not pursue the case with the Supreme Court, thus making a lower court's ruling law. That means bone marrow donors may now receive vouchers worth up to $3,000. NBC's Dr. Nancy Snyderman reports.

By JoNel Aleccia

Certain bone marrow donors could soon be compensated for their life-saving stem cells after federal officials declined to take the matter to the U.S. Supreme Court, allowing a lower court order to become law.

At least one agency, MoreMarrowDonors.org, hopes to begin a pilot program offering up to $3,000 in scholarships, housing vouchers or charity donations -- but not cash -- in exchange for matching donations of marrow cells derived from blood.

This decision is a total game-changer, said Jeff Rowes, a senior attorney with the Institute for Justice, which filed the lawsuit three years ago on behalf of cancer victims and others seeking bone marrow matches. Any donor, any doctor, any patient across the country can use compensation in order to get bone marrow donors.

That may be the effect of the decision by U.S. Attorney General Eric Holder to forgo a high court review of a 9th U.S. Circuit Court of Appeals ruling that certain kinds of bone marrow donations are exempt from federal rules banning compensation.

Under the ruling, donors who provide marrow cells through a process similar to blood donation, called peripheral blood stem cell apheresis, can be compensated because those cells are no longer regarded as organs or organ parts as defined in the National Organ Transplant Act.

The ruling does not apply, however, to bone marrow obtained through traditional techniques that use a needle to aspirate the cells from the hip.

Although it applies only to nine states covered by the 9th Circuit Court, Rowes expects the effects to be felt nationwide.

The move met with praise from Doreen Flynn, 36, of Lewiston, Maine, the lawsuits namesake and the single mother of three daughters with an incurable blood disorder called Fanconi anemia.

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Bone marrow donors soon may be compensated

HOUSTON (AP) - The Food and Drug Administration is criticizing in a new report the Texas company that stored adult stem cells from Texas Gov. Rick Perry for use in an experimental procedure for his back pain.

An FDA report obtained by the Houston Chronicle (http://bit.ly/MwEHjI ) says CellTex Therapeutics cannot guarantee that the stem cells it takes from patients remain alive and sterile. The report calls into question CellTex's quality control procedures and record-keeping.

CellTex was cast into the national spotlight after Perry announced his run for the GOP nomination for president. Perry would reveal that he had stem cells taken from fat in his own body, grown in a lab, and injected into his back and bloodstream in July.

CellTex CEO David Eller says his lab has already resolved many FDA complaints.

Information from: Houston Chronicle, http://www.houstonchronicle.com

Copyright 2012 The Associated Press. All rights reserved. This material may not be published, broadcast, rewritten or redistributed.

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FDA: Stem cell lab used by Perry has problems

ScienceDaily (June 25, 2012) If you break a bone, you know you'll end up in a cast for weeks. But what if the time it took to heal a break could be cut in half? Or cut to just a tenth of the time it takes now? Qian Wang, a chemistry professor at the University of South Carolina, has made tantalizing progress toward that goal.

Wang, Andrew Lee and co-workers just reported in Molecular Pharmaceutics that surfaces coated with bionanoparticles could greatly accelerate the early phases of bone growth. Their coatings, based in part on genetically modified Tobacco mosaic virus, reduced the amount of time it took to convert stem cells into bone nodules -- from two weeks to just two days.

The key to hastening bone healing or growth is to coax a perfectly natural process to pick up the pace.

"If you break a rib, or a finger, the healing is automatic," said Wang. "You need to get the bones aligned to be sure it works as well as possible, but then nature takes over."

Healing is indeed very natural. The human body continuously generates and circulates cells that are undifferentiated; that is, they can be converted into the components of a range of tissues, such as skin or muscle or bone, depending on what the body needs.

The conversion of these cells -- called stem cells -- is set into motion by external cues. In bone healing, the body senses the break at the cellular level and begins converting stem cells into new bone cells at the location of the break, bonding the fracture back into a single unit. The process is very slow, which is helpful in allowing a fracture to be properly set, but after that point the wait is at least an inconvenience, and in some cases highly detrimental.

"With a broken femur, a leg, you can be really incapacitated for a long time," said Wang. "In cases like that, they sometimes inject a protein-based drug, BMP-2, which is very effective in speeding up the healing process. Unfortunately, it's very expensive and can also have some side effects."

In a search for alternatives four years ago, Wang and colleagues uncovered some unexpected accelerants of bone growth: plant viruses. They originally meant for these viruses, which are harmless to humans, to work as controls. They coated glass surfaces with uniform coverings of the Turnip yellow mosaic virus and Tobacco mosaic virus, originally intending to use them as starting points for examining other potential variations.

But they were surprised to find that the coatings alone could reduce the amount of time to grow bone nodules from stem cells. Since then, Wang and co-workers have refined their approach to better define just what it is that accelerates bone growth.

Over the course of the past four years, they've demonstrated that it's a combination of the chemistry as well as the topography of the surface that determines how long it takes a stem cell to form bone nodules. The stem cells are nestled into a nanotopgraphy defined by the plant virus, and within that nanotopography the cells make contact with the variety of chemical groups on the viral surface.

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Speeding up bone growth by manipulating stem cells