Groundbreaking stem cell study kicks off in Northwest Florida – WEAR
By NEVAGiles23
A local stem cell study is changing the future of orthopedics.
A new study taking place at the Andrews Institute in Northwest Florida could shape the future of orthopedic surgery.
The goal of the study, spearheaded by Dr. Adam Anz and already eight years in the making, is to use stem cells to regrow cartilage.
If approved, it will be the first orthopedics study of its kind done in the United States and only the second in the entire world.
Stem cells are currently utilized most in cancer research and treatments, but Dr. Anz of the Andrews Institute wants to change that by putting regenerative medicine to the test, using stem cells to regrow knee cartilage.
The Andrews Institute already uses stem cells in certain therapies, but this new method could be a game changer.
"The bone marrow aspirate, which we're studying for knee arthritis and we can offer to patients, is the 1990's technology of stem cells," Dr. Anz said. "What we're studying is the modern way to harvest many more stem cells. That's the reason the FDA has said you need to bring this through our process before you just offer it to people."
Through a process called apheresis, stem cells are harvested from the patient with help from a synthetic hormone that promotes the body to generate more stem cells.
"Through this process we can collect millions of cells," Dr. Anz said. "Just 140 milliliters -- about a half of a coke can -- will have 140 million stem cells."
The stem cells will then be sorted, divided and injected into the patient's knee. Excess cells are stored in a nitrogen freezer at negative 181 degrees Celsius until the next round of injections, a process to be repeated over the next two years.
"If this study is successful, this will be the first approved in orthopedics in the United States," said Dr. Anz.
The study begins in May. Dr. Anz believes it will take about another five to seven years before the FDA can approve it for use in patients.
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Groundbreaking stem cell study kicks off in Northwest Florida - WEAR
Two weeks after his transplant, Jonathan Pitre battles kidney complications – Ottawa Citizen
By daniellenierenberg
Ottawa Citizen | Two weeks after his transplant, Jonathan Pitre battles kidney complications Ottawa Citizen Pitre, 16, was infused two weeks ago with stem-cell rich blood and bone marrow drawn from his mother's hip. The procedure, conducted as part of an ongoing clinical trial at the University of Minnesota Masonic Children's Hospital, is the only treatment ... |
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Two weeks after his transplant, Jonathan Pitre battles kidney complications - Ottawa Citizen
World’s 1st Stem Cell Transplant from Donor to Man’s Eye Shows Promise of Restoring Sight – EnviroNews (registration) (blog)
By daniellenierenberg
(EnviroNews World News) Kobe, Japan For more than two million Americans, straight lines may look wavy and the vision in the center of their eye may slowly disappear. Its called age-related macular degeneration (AMD), and there is no cure. But that may change soon.
A surgical team at Kobe City Medical Center General Hospital in Japan recently injected 250,000 retinal pigment epithelial (RPE) cells into the right eye of a man in his 60s. The cells were derived from donor stem cells stored at Kyoto University. It marked the first time that retinal cells derived from a donors skin have been implanted in a patients eye. The skin cells had been reprogrammed into induced pluripotent stem cells (iPS), which can be grown into most cell types in the body.
The procedure is part of a safety study authorized by Japans Ministry of Health that will involve five patients. Each will be followed closely for one year and continue to receive follow-up exams for three additional years. Project leader Dr. Masayo Takahashi at Riken, a research institution that is part of the study, told the Japan Times, A key challenge in this case is to control rejection. We need to carefully continue treatment.
A previous procedure on a different patient in 2014 used stem cells from the individuals own skin. Two years later, the patient reported showing some improvement in eyesight. But the procedure cost $900,000, leading the study team to move forward using donor cells. They expect the costs to come down to less than $200,000.
Among people over 50 in developed countries, AMD is the leading cause of vision loss. According to the National Eye Institute, 14 percent of white Americans age 80 or older will suffer some form of AMD. The condition is almost three times more common among white adults than among people of color. Women of all races comprise 65 percent of AMD cases.
The lack of a cure has led some to try unproven treatments. Three elderly women lost their sight after paying $5,000 each for a stem cell procedure at a private clinic in Florida. Clinic staff used liposuction to remove fat from the womens bellies. They then extracted stem cells from the fat, which were injected into both eyes of each patient in the same procedure, resulting in vision loss in both eyes. Two of the three victims agreed to a lawsuit settlement with the company that owned the clinic.
Stem cell therapy is still at an early stage. As of January 2016, 10 clinical uses have been approved around the world, all using adult stem cells. These include some forms of leukemia and bone marrow disease, Hodgkin and non-Hodgkin lymphoma and some rare inherited disorders including sickle cell anemia. Stem cell transplants are now often used to treat multiple myeloma, which strikes more than 24,000 people a year in the U.S.
Clinical trials to treat type 1 diabetes, Parkinsons disease, stroke, brain tumors and other conditions are being conducted. The first patient in a nationwide clinical study to receive stem cell therapy for heart failure recently underwent the procedure at the University of Wisconsin School of Medicine and Public Health. An experimental treatment at Keck Medical Center of USC last year on a paralyzed patient restored the 21-year-old mans use of his arms and hands. Harvard scientists see stem cell biology as a path to counter aging and extend human lifespans. But the International Society for Stem Cell Research warns that there are many challenges ahead before these treatments are proven safe and effective.
The U.S. Food and Drug Administration (FDA) regulates stem cells to ensure that they are safe and effective for their intended use. But, that doesnt stop some clinics from preying on worried patients. The FDA warns on its website that the hope that patients have for cures not yet available may leave them vulnerable to unscrupulous providers of stem cell treatments that are illegal and potentially harmful.
While there is yet no magic cure for AMD, the Japan study and others may one day lead there. The Harvard Stem Cell Institute (HSCI) in Boston is currently researching retina stem cell transplants. One approach uses gene therapy to generate a molecule that preserves healthy vision. Another involves Muller cells, which give fish the ability to repair an injured retina.
But these therapies are far off. We are at about the halfway mark, but there is still a precipitous path ahead of us, Takahashi said.
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World's 1st Stem Cell Transplant from Donor to Man's Eye Shows Promise of Restoring Sight - EnviroNews (registration) (blog)
Lab-Grown Neurons Could Help Heal Spinal Injuries and Restore Movement – Seeker
By Dr. Matthew Watson
The spinal cord is a rope of nerves relaying messages from the brain to every organ, muscle, and nerve ending in the body. The cells that make up the spinal cord arent a homogeneousmass, but rather a combination of dozens of specialized neurons, each with its own important role to play in guiding signals and impulses to the right destination.
This week, a team of California researchers announced the successful production of a lab-grown neuron that could help heal spinal cord injuries by reestablishing the connection between brain and muscle. In apaperpublished inProceedings of the National Academy of Sciences, researchers from the Gladstone Institutes and University of California campuses in San Francisco and Berkeley described how they grew human spinal cord neurons from stem cells and successfully introduced the lab-grown cells into the spines of healthy mice.
Todd McDevitt is a senior investigator at Gladstone and lead author of the study. He said that his team chose the targeted neuron, called a V2a interneuron, because it serves as a long relay cable between the neurons in the brain and the motor neurons that connect directly to muscle. V2a interneurons are, in fact, some of the longest cells in the body, able to extend their axon the nerve fibers that transmit electrical impulses across several vertebrae.
Its one cell stretching out up to 1,000 times longer than a normal human cell, said McDevitt. These outstretched neurons, as long as several centimeters, seem to play a critical role in relaying messages along the spinal cord. So if they are damaged in a traumatic injury, the brain-muscle connection may be severed, potentially leading to paralysis.
But if those critical V2a interneurons could be regenerated in an injured spine, the researchers wondered, perhaps the spinal cord could re-establish the connection and heal itself.
For the past three years, McDevitt and his team have been working to culture viable human V2a interneurons from pluripotent stem cells. The process, known as differentiation, attempts to replicate in the lab the natural development of neurons from undifferentiated stem cells in a human embryo.
Decades of research in developmental biology have provided clues to how genes in a developing embryo direct different proteins and other chemical factors to create all manner of specialized cells. The trouble is that most of the recipes for these chemical cocktails were derived from studying animal embryos.
Obviously, for good reasons, we dont do experiments on human embryos, McDevitt said. You have to take a leap of faith from the developmental biology knowledge we have from worms and flies and think about how we can apply that really important biological information to the human context.
RELATED:Brain Implant Helps 'Locked-In' ALS Woman Communicate
After experimenting with round after round of chemical combinations, the researchers landed on a process that can now produce a sizable batch of human V2a interneurons in a little over two weeks. The first step was to inject the cells into the spinal cords of healthy mice and see if the cells survived. They did even better.
Within two weeks, we saw a number of these cells extend their axons over long distances five millimeters reliably, but some even longer than that, McDevitt said, adding that the wiry cells are also making important connections. Even though theyre mice, we see these human cells that appear to be connecting to other neurons.
Does this mean were close to a human therapy using injections of healthy neurons to repair damaged spines? Not quite. Trials will first need to be run with injured mice before any human subjects can be tested. Plus, its entirely possible that V2a interneurons only fix very specific types of spinal injuries, or none at all. It might require the production of other spinal cord neurons, or a combination of several, to find the most effective treatment.
At the most basic level, this work shows that we can successfully introduce a new type of spinal neuron made from human pluripotent stem cells, McDevitt said. I see it as a step in whats probably going to be a much bigger effort by the field.
WATCH: Are We Close to Repairing Spinal Cord Injuries?
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Lab-Grown Neurons Could Help Heal Spinal Injuries and Restore Movement - Seeker
California’s $3-billion bet on stem cells faces final test : Nature News … – Nature.com
By Sykes24Tracey
Xianmin Zeng/Buck Inst.
Nerve cells derived from human stem cells, in work supported by the California Institute for Regenerative Medicine.
When California voters approved US$3billion in funding for stem-cell research in 2004, biologists flocked to the state, and citizens dreamed of cures for Parkinsons disease and spinal-cord injuries. Now, the pot of money one of the biggest state investments in science is running dry before treatments have emerged, raising questions about whether Californians will pour billions more into stem-cell research.
If they dont, that could leave hundreds of scientists without support, and strand potentially promising therapies before they reach the market. Its an issue of great concern, says Jonathan Thomas, chair of the board for the California Institute for Regenerative Medicine (CIRM) in Oakland.
CIRM is now doling out its final $650million, and its leaders are seeking money from the private sector to carry projects beyond 2020, when the money will run out. Advocates are also surveying voters to determine whether a new request for funding stands a chance in state elections next year. But critics argue against this way of funding research.
California voters saw major opportunities for stem cells in 2004 when they passed Proposition 71, which included an agreement to create the corporation that became CIRM. The move was a reaction to then-US president George W. Bushs decision in 2001 to restrict federal funds for work on human embryonic stem cells.
Since CIRM rolled out its first grants in 2006, it has funded more than 750 projects and reported alluring results from clinical trials. In March, a trial partially funded by CIRM showed that nine out of ten children born with severe combined immunodeficiency or bubble-boy disease a potentially lethal condition in which a persons immune system does not function properly, were doing well up to eight years after treatment (K.L.Shaw etal. J. Clin. Invest. http://doi.org/b6bp; 2017). They no longer need injections to be able to go to school, play outside or survive colds and other inevitable infections.
A dozen facilities constructed by CIRM have helped to push California to the forefront of research on ageing and regenerative medicine. Many grant recipients were early-career academics who had not been able to enter the stem-cell field previously because of the federal restrictions which were loosened in 2009 and the high cost of getting started in this kind of work. That barrier makes it difficult for researchers to gather the preliminary data typically required to win grants from the US National Institutes of Health (NIH).
To milk its remaining $650 million, CIRM partnered last year with the contract-research organization QuintilesIMS in Durham, North Carolina, to carry out clinical trials. CIRM leaders hope that this move will help to guide 40 novel therapies into trials by 2020.
Bob Klein, the property developer who put Proposition 71 on the ballot and established CIRM, isnt waiting for the money to run out. He leads an advocacy group, Americans for Cures, which will soon poll voters to see whether they would approve another $5 billion in funding. If it looks like at least 70% of Californians support that plan, hell start a campaign to put another initiative on the ballot in 2018.
Klein hopes that Californians will rise in support of science at a time when the Trump administration has proposed drastic cuts to the NIH budget. If public enthusiasm is not so strong, Klein says, hell aim for the 2020 elections, when voter turnout should be higher because it will coincide with the next presidential race.
Currently, CIRMs leaders are seeking other sources of support. The majority of our projects will not be ripe for interest from big pharma and the venture-capitalist community by the time we run out of funds, Thomas says. He has been courting large philanthropic foundations and wealthy individuals to raise money to continue the work.
John Simpson, who directs stem-cell oversight work at the advocacy group Consumer Watchdog in Washington DC, plans to oppose any effort to extend CIRM. I acknowledge their scientific advances, but we should not let a flawed process go further, he says. Simpson dislikes the model of using a vote to secure research funding through public bonds, because then the state lacks budgetary control.
Oversight of CIRM has been a problem in the past. In 2012, the US Institute of Medicine found that some scientists vetting grant proposals for CIRM had conflicts of interest. In response, CIRM altered its procedures but the public still felt betrayed. Jim Lott, a member of the state board that oversees CIRMs finances, says that he is not satisfied with the changes. He also argues that CIRM may not have been strategic enough in directing research. Some people say if they had a better focus, they might have achieved cures.
But researchers argue that expectations for cures after only a decade are unrealistic, given the typical pace of drug development. It would be a catastrophe for California if people say CIRM did not do what it was expected to do, says Eric Verdin, president of the Buck Institute for Research on Aging in Novato, California. Theyve built the foundation for the field and attracted people from around the world you cant just now pull the plug.
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California's $3-billion bet on stem cells faces final test : Nature News ... - Nature.com
UMN research team fixes broken hearts with 3D-printed tissue patch – Minnesota Daily
By Sykes24Tracey
A research team at the University of Minnesota found a way to heal broken hearts.
Researchers used a 3D printer to create protein patches that mimic heart tissue to treat post-heart attack scars. The research is in collaboration with the University of Wisconsin-Madison and the University of Alabama-Birmingham.
Brenda Ogle, a University biomedical engineering professor and lead researcher for the project, said she and her team have investigated proteins that surround cells in the body for 15 years. The team has been studying how the proteins also called the extracellular matrix influence stem cell behavior.
For many years, weve been trying to develop optimum formulation that can support stem cells in new cardiac [cell] types, Ogle said, adding that theyve focused on cardiac cell types to figure out a way to strengthen them after the muscle cells are damaged and die during a heart attack. Its one of the cell types in the body that cant be recovered.
The team successfully treated mice with the patches and is now planning to test the method on larger animals.
Molly Kupfer, a doctoral student who is part of Ogles team, said a heart attack occurs when there is a blockage in a primary blood vessel that delivers oxygen and nutrients to the heart.
When that happens, you have cell death in the area of the heart that doesnt receive the appropriate oxygen and nutrients, Kupfer said. Those cells that die arent able to recover."
Typically, after a heart attack, the blood clot in the heart is removed at a hospital, and if the heart has not been damaged too badly, doctors monitor the heart long-term, prescribe medicine and regularly check for signs of heart failure, Ogle said.
What you get instead after a heart attack is scar tissue forming, and that scar tissue ultimately fails, Ogle said.
Associate Professor Brenda Ogle places a 3D printed biopatch on a mouse heart in Nils Hasselmo Hall on Tuesday, April 25, 2017. Her research team induces heart attacks in mice, which causes a dead area of cardiac cells. The patch is placed in this dead zone and mimics the cells of the native heart that aren't able to be replenished on their own.
Kupfer said she worked with Paul Campagnola and his lab at the University of Wisconsin to print the patches; the cells were prepared at the University of Minnesota.
Campagnola, a biomedical engineering professor, said he initially developed the underlying printing technology in 2000.
"The idea of the patch is it could actually behave like native cardiac tissue and assist the function of the heart, Kupfer said, adding that the method used to print the patches results in extremely high resolution structures.
Ogle said before applying the patch to the animal hearts theyre currently testing on, they take a scan of the scarred tissue and create a digital template for the 3D-printer to follow and print the proteins in the same pattern.
Campagnola said the patch provides a stable space for cells to grow and be implanted in damaged areas.
Cardiac cells are also added to the patch when it covers a damaged area. Ogle said it not only provides a support structure, but transplants healthy cells that will eventually become integrated into the heart, stabling it structurally and functionally.
A huge aha moment was when [the cardiac cells] started to beat on this patch synchronously and spontaneously, she said. When that happened, we realized that this could be a viable therapy for the heart, a way to replace those lost muscle cells.
Through the research group at the University of Alabama, Ogle said a study was conducted where the patch was tested on dead or dying tissue in mice hearts and the group saw improvement in the mice after four weeks.
The project was funded through a series of grants from the National Institutes of Health, the National Science Foundation with support from the University, she said.
The group has since received larger funds from the NIH to run a study using the patch on larger animals within the next year.
Ogle said it would take about 10 years until the patch can be used on human patients in a clinical setting.
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UMN research team fixes broken hearts with 3D-printed tissue patch - Minnesota Daily
Are baby, wisdom teeth the next wave in stem cell treatment? – CNN
By Dr. Matthew Watson
It's based on experimental research that suggests stem cells extracted from the pulp of these teeth might someday regrow a lost adult tooth or offer other regenerative medicine benefits -- some potentially life-saving.
"So I'll try not to get emotional here, but my husband was diagnosed with acute myeloid leukemia in 2011," said Bassetto, of Naperville, Illinois, head of a sales team at a software company.
In 2012, her husband, James, had a stem cell transplant to restore his bone marrow and renew his blood.
"He was very fortunate. He was one of six kids, and his brother was a perfect match," she said. She noted that her two children, Madeline, 23, and Alex, 19, may not be so lucky if they develop health problems, since they have only each other; the chance of two siblings being a perfect stem cell match is only 25%.
Unfortunately, her husband's stem cell transplant was not successful. He developed graft-versus-host disease, where his brother's donated stem cells attacked his own cells, and he died shortly afterward.
However, she says, the transplant had given him a chance at a longer life.
Last year, when her son saw a dentist for wisdom tooth pain, a brochure for dental stem cell storage caught Bassetto's eye and struck a chord.
"I know stem cells have tremendous health benefits in fighting disease, and there's a lot ways they're used today," she said. "Had my husband had his own cells, potentially, his treatment could have been more successful."
Medical breakthroughs happen all the time, said Bassetto. "Who knows what potential there is 20 years, 40 years down the road, when my son is an adult or an aging adult?
"Almost like a life insurance policy, is how I viewed it," she said.
Some scientists see storing teeth as a worthwhile investment, but others say it's a dead end.
"Research is still mostly in the experimental (preclinical) phase," said Ben Scheven, senior lecturer in oral cell biology in the school of dentistry at the University of Birmingham. Still, he said, "dental stem cells may provide an advantageous cell therapy for repair and regeneration of tissues," someday becoming the basis for reconstructing bone tissue, retinas and even optic neurons.
Dr. Pamela Robey, chief of the craniofacial and skeletal diseases branch of the National Institute of Dental and Craniofacial Research, acknowledges the "promising" studies, but she has a different take on the importance of the cells.
"There are studies with dental pulp cells being used to treat neurological disorders and problems in the eye and other things," Robey said. The research is based on the idea that these cells "secrete factors that encourage local cells to begin the repair process."
"The problem is, these studies have really not been that rigorous," she said, adding that many have been done only in animals and so provide "slim" evidence of benefits. "The science needs a lot more work."
Robey would know. Her laboratory discovered dental stem cells in 2003.
"My fellows, Songtao Shi and Stan Gronthos, did the work in my lab," Robey said. "Songtao Shi is a dentist, and basically he observed that, when you get a cavity, you get what's called 'reparative dentin.' In other words, the tooth is trying to protect itself from that cavity, so it makes a little bit of dentin to kind of plug the hole, so to speak."
Dentin is the innermost hard layer of tooth that lies beneath the enamel. Underneath the dentin is a soft tissue known as pulp, which contains the nerve tissue and blood supply.
Observing dentin perform reparative work, Shi hypothesized that this must mean there's a stem cell within the tooth that's able to activate and make dentin. So if you wanted to grow an adult tooth instead of getting an implant, knowing how to make dentin would be the start of the process, explained Robey.
Pursuing this idea, Shi, Gronthos and the team conducted their first study with wisdom teeth. They discovered that pulp cells in these third molars did indeed make dentin, but the cells found in baby teeth, called SHED (stem cells from human exfoliated deciduous teeth), had slightly different properties.
"The SHED cells seem to make not only dentin but also something that is similar to bone," Robey said. This "dentin osteogenic material" is a little like bone and a little like dentin -- "unusual stuff," she said.
There is a meticulous process for extracting stem cells from the pulp.
"We very carefully remove any soft tissue that's adhering to the tooth. We treat it with disinfectant, because the mouth is not really that clean," Robey said, laughing.
Scientists then use a dental drill to pass the enamel and dentin -- "kind of like opening up a clam," said Robey -- to get to the pulp. "We take the pulp out, and we digest it with an enzyme to release the cells from the matrix of the pulp, and then we put the cells into culture and grow them."
According to Laning, even very small amounts of dental pulp are capable of producing many hundreds of millions of structural stem cells.
Harvesting dental stem cells is not a matter of waiting for the tooth to fall out and then quickly calling your dentist. When a baby tooth falls out, the viability of the pulp is limited if it's not preserved in the proper solution.
American Academy of Pediatric Dentistry President Dr. Jade Miller explained that "it's critical that the nerve tissue in that pulp tissue, the nerve supply and blood supply, still remain intact and alive." Typically, the best baby teeth to harvest are the upper front six or lower front six -- incisors and cuspids, he said.
For a child between 5 and 8 years of age, it's best to extract the tooth when there's about one-third of the root remaining, Miller said: "It really requires some planning, and so parents need to make this decision early on and be prepared and speak with their pediatric dentist about that."
Bassetto found the process easy. All it involved was a phone call to the company recommended by her dentist.
"They offer a service where they grow the cells and save those and also keep the pulp of the tooth without growing cells from it," she said. "I opted for both." From there, she said, the dentist shipped the extracted teeth overnight in a special package.
Bassetto said she paid less than $2,000 upfront, and now $10 a month for continued storage.
So is banking teeth something parents should be doing?
In a policy statement, the American Academy of Pediatric Dentistry "encourages dentists to follow future evidence-based literature in order to educate parents about the collection, storage, viability, and use of dental stem cells with respect to autologous regenerative therapies."
"Right now, I don't think it is a logical thing to do. That's my personal opinion," said Robey of the National Institute of Dental and Craniofacial Research. As of today, "we don't have methods for creating a viable tooth. I think they're coming down the pike, but it's not around the corner."
Science also does not yet support using dental pulp stem cells for other purposes.
"That's not to say that in the future, somebody could come up with a method that would make them very beneficial," Robey said.
Still, she observed, if science made it possible to grow natural teeth from stem cells and you were in a car accident, for example, and lost your two front teeth, you'd probably be "very happy to give up a third molar to use the cells in the molar to create new teeth." Third molars are fairly expendable, she said.
Plus, Robey explained, it may not be necessary to bank teeth: Another type of stem cell, known as induced pluripotent stem cells, can be programmed into almost any cell type.
"It's quite a different story than banking umbilical cord blood, which we do know contains stem cells that re-create blood," Robey said.
"So cord blood banking -- and now we have a national cord blood bank as opposed to private clinics -- so there's a real rationale for banking cord blood, whereas the rationale for banking baby teeth is far less clear," Robey said.
And there's no guarantee that your long-cryopreserved teeth or cells will be viable in the future. Banking teeth requires proper care and oversight on the part of cryopreservation companies, she said. "I think that that's a big question mark. If you wanted to get your baby teeth back, how would they handle that? How would they take the tooth out of storage and isolate viable cells?"
Provia's Laning, who has "successfully thawed cells that have been frozen for more than 30 years," dismissed such ideas.
"Cryopreservation technology is not the problem here," he said. "Stem cells from bone marrow and other sources have been frozen for future clinical use in transplants for more than 50 years. Similarly, cord blood has a track record of almost 40 years." The technology for long-term cryopreservation has been refined over the years without any substantial changes, he said.
Despite issues and doubts, Miller, of the pediatric dentistry academy, said parents still need to consider banking baby teeth.
A grandparent, he is making the decision for his own family.
"It's really at its infancy, much of this research," he said. "There's a very strong chance there's going to be utilization for these stem cells, and they could be life-saving."
He believes that saving baby teeth could benefit not only his grandchildren but also their older siblings and various other family members if their health goes awry and a stem cell treatment is needed.
"The science is strong enough to show it's not science fiction," Miller said. "There's going to be a significant application, and I want to give my grandkids the opportunity to have those options."
Aside from cost, Miller said there are other considerations: "Is this company going to be around in 30, 40 years?" he asked. "That's not an easy thing to figure out."
Having taken the leap, Bassetto doesn't worry.
"In terms of viability, you know, if something were to happen with the company, you could always get what's stored and move it elsewhere, so I felt I was protected that way," she said. She feels "pretty confident" with her decision and plans to store her grandchildren's baby teeth.
Still, she concedes that her circumstances may be rare.
"Not everybody's going to be touched by some kind of disease where it just hits home," Bassetto said. "For me, that made it a no-brainer."
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Are baby, wisdom teeth the next wave in stem cell treatment? - CNN
Bare bones: Making bones transparent – Science Daily
By JoanneRUSSELL25
Science Daily | Bare bones: Making bones transparent Science Daily Ten years ago, the bones currently in your body did not actually exist. Like skin, bone is constantly renewing itself, shedding old tissue and growing it anew from stem cells in the bone marrow. Now, a new technique developed at Caltech can render ... Scientists turn bones transparent to let them see into marrow Tissue-Clearing Technique Works on Bone |
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Bare bones: Making bones transparent - Science Daily
Tough time: Jonathan Pitre battles kidney complications – Ottawa Sun
By raymumme
Ottawa Sun | Tough time: Jonathan Pitre battles kidney complications Ottawa Sun Pitre, 16, was infused two weeks ago with stem-cell rich blood and bone marrow drawn from his mother's hip. The procedure, conducted as part of an ongoing clinical trial at the University of Minnesota Masonic Children's Hospital, is the only treatment ... |
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Tough time: Jonathan Pitre battles kidney complications - Ottawa Sun
Bone marrow drive for Allums siblings at ULM, other locations – Monroe News Star
By Dr. Matthew Watson
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A bone marrow drive for James Christopher Allums, 21, and his sister Elizabeth, 3, is Monday, May 1 at locations throughout northeast Louisiana.
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The News Star 11:33 a.m. CT April 26, 2017
University of Louisiana Monroe(Photo: Courtesy image)
A bone marrow drive for James Christopher Allums, 21, and his sister Elizabeth, 3, is Monday, May 1 at locations throughout northeast Louisiana.
University of Louisiana Monroe Medical Laboratory Science faculty and students are helping organize the drive. The drive on campus is 9 a.m.-5 p.m. in the SUB and Quad.
May 1 is National Fanconi Anemia Day. James Christopher and Elizabeth suffer from this disease, which is fatal without a bone marrow or stem cell transplant. They are the children of Chris and Ellen Allums.
Melanie Chapman, assistant professor to the School of Health Professions, said, "This is a wonderful opportunity for ULM Warhawks to fly high by working together and setting aside our busy agendas to give two great kids, and possibly others, the chance to live out their years. I am privileged to be a part of ULM and this community effort."
Bone marrow drive locations:
Times vary and new locations may be added. For information, check Facebook The Friends of James Christopher and Elizabeth Allums or visit caringbridge.org and search James Christopher Allums .
MORE NEWS;The Fabulous Equinox Orchestra takes the stage at ULM Friday
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Bone marrow drive for Allums siblings at ULM, other locations - Monroe News Star
Bone marrow drive set for local youth – Perry County Republic Monitor
By raymumme
This weekend the community will be given the chance to save the life of one of its youngest residents. On Friday, April 28, the AmVets Post No. 94 will host a bone marrow registration drive from 3-7 p.m.
Wade Wachter is the son of Adam and Jenni (Duvall) Wachter of Perryville and the grandson of Terri and Lori Duvall, Robyn Roy, and Rodney and Barb Wachter.
On the outside, little Wade is a normal kid though he has been battling a very rare form of bone marrow failure disorder called Schwachman Diamond Syndrome. This dysfunction of the bone marrow requires a lifesaving transplant. He currently takes medication daily and has routine biopsies to monitor for potential leukemia developments in his body.
This disease is so rare that funding is hard to find, which limits the number of possible treatments available. DKMS is the nonprofit group leading the charge to find a bone marrow match for Wachter.
Recent tests show that Wachter will need an immediate transplant for his best chance to have a normal childhood, and according to the DKMS website only 30 percent of patients find a donor inside their families. Nearly 14,000 patients require donations from matched individuals outside of their family line each year. Out of more than 800,000 donors in the U.S., and over 6 million worldwide, 6 out of 10 patients are still unable to find a compatible donor.
We thank DKMS and our community for working with us to help find Wade a bone marrow match, said Jenni Wachter, Wades mother. From the outside, Wade may look like your average 6-year-old child, when really he has been facing a life-threatening battle for years. Our hope is to grow the bone marrow registry to help increase the chances of finding Wade a match so he can move forward towards a healthy and happy life.
Potential donors include anyone who is in good general health between the ages of 18 to 55. Registration is free and only requires filling out a simple form and a quick swab of the inside of each cheek. DKMS covers the $65 registration and processing fee for each supporter, but donations will be accepted to cover costs.
There are two ways to donate once a match has been found. The first method is the Peripheral Blood Stem Cell (PBSC) donation. This is a non-surgical, outpatient procedure that collects blood stem cells via the bloodstream. It takes about 4-8 hours on 1-2 consecutive days. This method is used in 75 percent of all cases. The other donation method is by direct bone marrow procedure. It is a 1-2 hour surgical procedure, done under anesthesia, where a syringe collects marrow cells from the back of the pelvic bone. This method is only used in about 25 percent of the cases, usually when the patient is a child.
Anyone unable to attend the drive that wishes to register as a potential donor may do so online at http://www.dkms.org. The Perryville AmVets Post No. 94 is located at 1203 W. Saint Joseph Street.
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Bone marrow drive set for local youth - Perry County Republic Monitor
TGF-/MAPK signaling mediates the effects of bone marrow mesenchymal stem cells on urinary control and interstitial … – UroToday
By NEVAGiles23
This study aimed to explore the role of the transforming growth factor-/mitogen activated protein kinase (TGF-/MAPK) signaling pathway in the effects of bone marrow mesenchymal stem cells (BMSCs) on urinary control and interstitial cystitis in a rat model of urinary bladder transplantation.
A urinary bladder transplantation model was established using Sprague-Dawley rats. Rats were assigned to normal (blank control), negative control (phosphate-buffered saline injection), BMSCs (BMSC injection), sp600125 (MAPK inhibitor injection), or protamine sulfate (protamine sulfate injection) groups. Immunohistochemistry, urodynamic testing, hematoxylin-eosin staining, Western blotting, enzyme-linked immunosorbent assay, and MTT assay were used to assess BMSC growth, the kinetics of bladder urinary excretion, pathological changes in bladder tissue, bladder tissue ultrastructure, the expression of TGF-/MAPK signaling pathway-related proteins, levels of inflammatory cytokines, and the effects of antiproliferative factor on cell proliferation.
Compared with normal, negative control, BMSCs, and sp600125 groups, rats in the PS group exhibited decreased discharge volume, maximal micturition volume, contraction interval, and bladder capacity but increased residual urine volume, bladder pressure, bladder peak pressure, expression of TGF-/MAPK signaling pathway-related proteins, levels of inflammatory cytokines, and growth inhibition rate. Levels of inflammatory cytokines and the growth inhibition rate were positively correlated with the expression of TGF-/MAPK signaling pathway-related proteins.
Our findings demonstrate that the TGF-/MAPK signaling pathway mediates the beneficial effects of BMSCs on urinary control and interstitial cystitis.
American journal of translational research. 2017 Mar 15*** epublish ***
Ya Xiao, Ya-Jun Song, Bo Song, Chi-Bing Huang, Qing Ling, Xiao Yu
Urological Research Institute of PLA, The First Affiliated Hospital, Third Military Medical UniversityChongqing 400037, P. R. China; Department of Urology, The Second Affiliated Hospital, The Third Military Medical UniversityChongqing 400037, P. R. China., Department of Urology, The Second Affiliated Hospital, The Third Military Medical University Chongqing 400037, P. R. China., Urological Research Institute of PLA, The First Affiliated Hospital, Third Military Medical University Chongqing 400037, P. R. China., Department of Urology, Tongji Hospital, Tongji Medical College of Huazhong University of Science & Technology Wuhan 430030, P. R. China.
PubMed http://www.ncbi.nlm.nih.gov/pubmed/28386345
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TGF-/MAPK signaling mediates the effects of bone marrow mesenchymal stem cells on urinary control and interstitial ... - UroToday
A New Technique Transforms Human Skin Into Brain Cells – Futurism – Futurism
By LizaAVILA
From Skin to Brain
The brain is one of the most vital organs in the human body, so damage to the brain from injury or aging can have major impacts on peoples quality of life.Neurological disorders representsome of todays most devastating medical conditions that are also difficult to treat.Among these is Alzheimers disease.
Usually, research involving Alzheimers rely on brain cells from mice. Now, neurobiologists from the University of California, Irvine (UCI) have developed a method that could allow the use of human cells instead of animal ones to help understand neurological diseases better.
In their study, which was published in the journal Neuron, the researchers found a way to transform human skin cells into stem cells and program them into microglial cells. The latter make up about 10 to 15 percent of the brain and are involved in the removing dead cells and debris, as well as managing inflammation. Micgrolia are instramentalin neural network development and maintenance, explained researcher Mathew Blurton Jones, fromUCIs Department of Neurobiology & Behavior.
Microglia play an important role in Alzheimers and other diseases of the central nervous system. Recent research has revealed that newly discovered Alzheimers-risk genes influence microglia behavior, Jones said in an interview for a UCI press release. Using these cells, we can understand the biology of these genes and test potential new therapies.
The skin cells had been donated by patients from UCIs Alzheimers Disease Research Center. These were firstsubjected toa genetic process to convert them into induced pluripotent stem (iPS) cells adult cells modified to behave as an embryonic stem cell, allowing them to become other kinds of cells. These iPS cells were then exposed to differentiation factors designed to imitate the environment of developing microglia, which transformed them into the brain cells.
This discovery provides a powerful new approach to better model human disease and develop new therapies, said UCI MIND associate researcher Wayne Poon in the press release. The researchers, in effect, have developed a renewable and high-throughput method for understanding the role of inflammation in Alzheimers disease using human cells, according to researcher Edsel Abud in the same source.
In other words, by using human microglia instead of those from mice, the researchers have developed a more accurate toolto study neurological diseases and to develop more targeted treatment approaches. In the case of Alzheimers, they studied the genetic and physical interactions between the diseases pathology and the induced microglia cells. These translational studies will better inform disease-modulating therapeutic strategies, Abud added in the press release.
Furthermore, they are now using these induced microglia cells in three-dimensional brain models. The goal is to understand the interaction between microglia and other brain cells, and how these influence the development of Alzheimers and other neurological diseases.
This is all made possible by reprogrammable stem cells. Indeed, this study is one more example of how stem cells arechanging medicine.
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A New Technique Transforms Human Skin Into Brain Cells - Futurism - Futurism
A mechanism shared by healing wounds and growing tumors – The Rockefeller University Newswire
By NEVAGiles23
Cancerous cells in a skin tumor become locked in an abnormal state as a result of the activation of a gene-regulating element (green).
Like an image in a broken mirror, a tumor is a distorted likeness of a wound. Scientists have long seen parallels between the two, such as the formation of new blood vessels, which occurs as part of both wound healing and malignancy.
Research at The Rockefeller University offers new insights about what the two processes have in commonand how they differat the molecular level. The findings, described April 20 in Cell, may aid in the development of new therapies for cancer.
Losing identity
At the core of both malignancy and tissue mending are stem cells, which multiply to produce new tissue to fill the breach or enlarge the tumor. To see how stem cells behave in these scenarios, a team led by scientists in Elaine Fuchss lab compared two distinct types found within mouse skin.
One set of stem cells, at the base of the follicle, differentiates to form the hair shaft; while another set produces new skin cells. Under normal conditions, these two cell populations are physically distinct, producing only their respective tissue, nothing else.
But when Yejing Ge, a postdoc in the Fuchs lab, looked closely at gene activity in skin tumors, she found a remarkable convergence: The follicle stem cells expressed genes normally reserved for skin stem cells, and vice versa. Around wounds, the researchers documented the same blurring between the sets of stem cells.
Master switches
Two of the identity-related genes stood out. They code for so-called master regulators, molecules that play a dominant role in determining what type of tissue a stem cell will ultimately producein this case, hair follicle or skin. The researchers suspect that stress signals from the tissue surrounding the damage or malignancy kick off a cycle that feeds off itself by enabling the master regulators to make more of themselves.
Access to DNA is the key. To go to work, master regulators bind to certain regions of DNA and so initiate dramatic changes in gene expression. The researchers found evidence that stress signals open up new regions of DNA, making them more accessible to gene activation. By binding in these newly available spots, master regulators elevate the expression of identity-related genes, including the genes that encode the master regulators themselves.
Locked in
While wounds heal, cancer can grow indefinitely. The researchers discovered that while stress signals eventually wane in healing wounds, they can persist in cancerand with prolonged stress signaling, another region of DNA opens up to kick off a separate round of cancer-specific changes.
Tumors have been described as wounds that never heal, and now we have identified specific regulatory elements that, when activated, keep tumor cells locked into a blurred identity, Ge says.
The scientists hope this discovery could lead to precise treatments for cancer that cause less collateral damage than conventional chemotherapy. We are currently testing the specificity of these cancer regulatory elements in human cells for their possible use in therapies aimed at killing the tumor cells and leaving the healthy tissue cells unharmed, Fuchs says.
Elaine Fuchs is the Rebecca C. Lancefield Professor, head of the Robin Chemers Neustein Laboratory of Mammalian Cell Biology and Development, and a Howard Hughes Medical Institute investigator.
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A mechanism shared by healing wounds and growing tumors - The Rockefeller University Newswire
Researchers Invent Stem Cell Capable of Becoming an Entire Embryo – Futurism
By raymumme
A New Type of Stem Cell
While much has been gleaned about the power of stem cells over the last few decades, researchers from the Salk Institute and Peking Universityin China recently found out theres plenty left to discover and invent. Nature, it seems, will always keep you guessing.
In a study published in the journal Cell, the team of researchers revealed they had succeeded in creating a new kind of stem cell thats capable of becoming any type of cell in the human body. Extended pluripotent stem cells or EPS cells are similar to induced pluripotent stem cells(iPS cells), which were invented in 2006.
The key difference between the two is that iPS cells are made from skin cells (called fibroblasts) and EPS cells are made from a combination of skin cells and embryonic stem cells. iPS cells are the hallmark of stem cell research and can be programmed to become any cell in the human body hence the pluripotent part of their name. EPS cells, too, can give rise to any type of cell in the human body, but they can also do something very different something unprecedented, actually: they can create the tissues needed to nourish and grow an embryo.
The discovery of EPS cells provides a potential opportunity for developing a universal method to establish stem cells that have extended developmental potency in mammals, says Jun Wu, one of the studys authors and senior scientist at the Salk Institute, in the organizations news release.
When a human or any mammalian egg gets fertilized, the cells divide up into two task forces: one set is responsible for creating the embryo, and the other set creates the placenta and other supportive tissues needed for the embryo to survive (called extra-embryonic tissues). This happens very early in the reproductive process so early, in fact, that researchers have had a very hard time recreating it in a lab setting.
By culturing and studying both types of cells in action, researchers would not only be able to understand the mechanism that drives it, but hopefully could shed some light on what happens when things go wrong, like in the case of miscarriage.
The researchers at the Salk Institute managed to form a chemical cocktail of four chemicals and a type of growth factor that created a stable environment in which they could culture both types of cells in an immature state. They could then harness the two types of cells for their respective abilities.
What they discovered was that not only were these cells extremely useful for creating chimeras (where two types of animal cells or human and animal cells are mixed to form something new), but were also technically capable of creating and sustaining an entire embryo.At least in theory: while they were able to sustain both human and mouse cells, the ethical considerations of creating a human embryo this way have prevented them from attempting it.
That being said, theres no shortage of applications for this type of stem cell: researchers will be able to use them to model diseases, regenerate tissue, create and trial drug therapies, and study in depth early reproductive processes like implantation. Human-animal chimeras may also help engineer organs for transplant or, you know, give rise to the next superhero.
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Researchers Invent Stem Cell Capable of Becoming an Entire Embryo - Futurism
‘Minibrains’ in a dish shed a little light on autism and epilepsy – 89.3 KPCC
By daniellenierenberg
Tiny, 3-D clusters of human brain cells grown in a petri dish are providing hints about the origins of disorders like autism and epilepsy.
An experiment using these cell clusters which are only about the size of the head of a pin found that a genetic mutation associated with both autism and epilepsy kept developing cells from migrating normally from one cluster of brain cells to another, researchers report in the journal Nature.
"They were sort of left behind," says Dr. Sergiu Pasca, an assistant professor of psychiatry and behavioral sciences at Stanford. And that type of delay could be enough to disrupt the precise timing required for an actual brain to develop normally, he says.
The clusters often called minibrains, organoids or spheroids are created by transforming skin cells from a person into neural stem cells. These stem cells can then grow into structures like those found in the brain and even form networks of communicating cells.
Brain organoids cannot grow beyond a few millimeters in size or perform the functions of a complete brain. But they give scientists a way to study how parts of the brain develop during pregnancy.
"One can really understand both a process of normal human brain development, which we frankly don't understand very well, [and] also what goes wrong in the brain of patients affected by diseases," says Paola Arlotta, a professor of stem cell and regenerative biology at Harvard who was not involved in the cell migration study. Arlotta is an author of a second paper in Nature about creating a wide variety of brain cells in brain organoids.
Pasca's team began experimenting with organoids in an effort to learn more about brain disorders that begin long before birth. Animal brains are of limited use in this regard because they don't develop the way human brains do. And traditional brain cell cultures, which grow as a two-dimensional layer in a dish, don't develop the sort of networks and connections that are thought to go awry in disorders like autism, epilepsy and schizophrenia.
"So the question was really, can we capture in a dish more of these elaborate processes that are underlying brain development and brain function," Pasca says.
He was especially interested in a critical process that occurs when cells from deep in the brain migrate to areas nearer the surface. This usually happens during the second and third trimesters of pregnancy.
So Pasca's team set out to replicate this migration in a petri dish. They grew two types of clusters, representing both deep and surface areas of the forebrain. Then they placed deep clusters next to surface clusters to see whether cells would start migrating.
Pasca says the cells did migrate, in a surprising way. "They don't just simply crawl, but they actually jump," he says. "So they look for a few hours in the direction in which they want to move, they sort of decide on what they want to do, and then suddenly they make a jump."
Pasca suspected this migration process might be disrupted by a genetic disorder called Timothy syndrome, which can cause a form of autism and epilepsy. So he repeated the experiment, using stem cells derived from the skin cells of a person who had Timothy syndrome.
And sure enough, the cells carrying the genetic mutation didn't jump as far as healthy cells did. "They moved inefficiently," Pasca says.
Next Pasca wondered if there might be some way to fix the migration problem. He thought there might be, because Timothy syndrome causes cells to let in too much calcium. And he knew that several existing blood pressure drugs work by blocking calcium from entering cells.
So the team tried adding one of these calcium blockers to the petri dish containing clusters of brain cells that weren't migrating normally. And it worked. "If you do treat the cultures with this calcium blocker, you can actually restore the migration of cells in a dish," Pasca says.
Fixing the problem in a developing baby wouldn't be that simple, he says. But the experiment offers a powerful example of how brain organoids offer a way to not only see what's going wrong, but try drugs that might fix the problem.
Still, to realize their full potential, brain organoids need to get better, Arlotta says. This means finding ways to keep the cell clusters alive longer and allowing them to form more of the types of brain cells that are found in a mature brain.
Arlotta's team has developed techniques that allow brain cell clusters to continue growing and developing in a dish for many months. And what's remarkable, she says, is that over time the clusters automatically begin creating structures and networks like those in a developing brain.
"Using their own information from their genome, the cells can self-assemble and they can decide to become a variety of different cell types than you normally find," she says.
In one experiment, a brain organoid produced nearly all the cell types found in the mature retina, Arlotta says. And tests showed that some of these retinal cells even responded to light.
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'Minibrains' in a dish shed a little light on autism and epilepsy - 89.3 KPCC
Discovery offers new hope to repair spinal cord injuries: Scientists … – Science Daily
By raymumme
Science Daily | Discovery offers new hope to repair spinal cord injuries: Scientists ... Science Daily Scientists have created a special type of neuron from human stem cells that could potentially repair spinal cord injuries. These cells, called V2a interneurons, ... |
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Discovery offers new hope to repair spinal cord injuries: Scientists ... - Science Daily
Custer considered for stem cell study | The Miami Student – Miami Student
By JoanneRUSSELL25
WSU student fractured spine at Oxford party
Ryan Custer, a Wright State University student who was severely injured at an Oxford party, is being considered for a stem cell study at Rush University Hospital in Chicago. The 19-year-old, a first-year forward for the Raiders varsity basketball team, will be evaluated for five days before doctors determine if he qualifies for the study.
Custer suffered a severe spinal injury after jumping into a makeshift pool at a party on S. Main Street on Saturday, April 8. Custer collided with another persons knee when he slid into the pool, causing the injury. Custer was immediately transported to the University of Cincinnati Medical Center where he underwent surgery on his spine that night.
Feeling in Custers legs has not returned, and he has only recently regained some movement in his fingers.
Custer was transported from the UC Medical Center to Rush Hospital on Sunday, April 22. According to a post from the Ryan Custer Recovery Care Page, a Facebook page updated almost daily by Custers family, he spent the first day in Chicago getting acclimated in his new room in Rushs ICU and meeting the doctor who will lead the study, Dr. Richard Fessler.
Dr. Fessler, a renowned spinal surgeon, has focused his research on developing and refining new ways to perform minimally invasive spinal surgeries. In 2010, Fessler performed surgery on former Indianapolis Colts quarterback Peyton Manning, which Custer was happy to learn, the post said.
The five-day period of testing began Monday, and, if selected for the study, treatment for Custer will begin on Friday. The study, called the SCiStar study, will evaluate how the injection of AST-OPC1, particular neural cells produced from human embryonic stem cells, at a single time 14 to 30 days after an injury can benefit the patients recovery.
According to the SCiStar webpage, the studys researchers are seeking adults between the ages of 18 and 69 who recently experienced a spinal cord injury in the neck which resulted in a loss of feeling below the site of the injury in addition to some paralysis in the arms and legs.
HBO has contacted Dr. Fessler about following a patient through this research process.
Ryan thinks it would be cool to do it, so we said yes,an April 22 Facebook post reads. Another step in the plan God has mapped out for Ryan.
A fundraising page created for Custer, The Ryan Custer 33 Recovery Fund, is close to raising its entire $100,000 goal. At the time of publication, the fund was just about $4,000 shy of the 100k mark.
Over 6,500 people have liked the page and are following along with Custers recovery through the familys Facebook updates.
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Custer considered for stem cell study | The Miami Student - Miami Student
Coalition Duchenne Congratulates Capricor Therapeutics on Positive Six-Month Results from its Randomized CAP … – Benzinga
By raymumme
Coalition Duchenne funded cardiosphere-derived cell studies in Duchenne carried out by Eduardo Marbn MD, the director of the Cedars-Sinai Heart Institute. This work was later licensed for commercial development by Capricor.
(PRWEB) April 25, 2017
Coalition Duchenne, a charity based in Newport Beach, California, committed to raising awareness for Duchenne muscular dystrophy, and funding for Duchenne research, congratulates Capricor Therapeutics on positive six-month results from its randomized CAP-1002 (cardiosphere-derived cells) Phase I/II HOPE clinical trial in Duchenne announced today.
Coalition Duchenne funded studies carried out by Eduardo Marbn MD, the director of the Cedars-Sinai Heart Institute, on cardiosphere-derived cells in Duchenne. This work was later licensed for commercial development by Capricor.
"We are excited that our work with Dr. Marbn is evolving and could become a treatment for Duchenne through Capricor's stellar efforts. It is the cardiomyopathy associated with Duchenne that causes us to lose so many boys and young men with Duchenne. Their hearts fail them when they still have so many dreams to be fulfilled. We must strengthen those hearts," said Catherine Jayasuriya, the founder and executive director of Coalition Duchenne.
The six-month results showed statistically-significant improvements in systolic thickening of the inferior wall of the heart and in the function of the middle and distal upper limb in patients treated with CAP-1002 as compared to control patients. In addition, differences observed in several other cardiac and skeletal muscle measures, including cardiac scar, were consistent with a treatment effect.
"These initial positive clinical results build upon a large body of preclinical data which illustrate CAP-1002's potential to broadly improve the condition of those afflicted by Duchenne, as they show that cardiosphere-derived cells exert salutary effects on cardiac and skeletal muscle," said Linda Marbn, PhD, Capricor's president and chief executive officer.
John L. Jefferies, MD, Professor of Pediatric Cardiology and Adult Cardiovascular Diseases at the University of Cincinnati and Director, Advanced Heart Failure and Cardiomyopathy, and Principal Investigator of the HOPE Trial, said, "In HOPE, we saw potential effects in both the heart and skeletal muscle that appear quite compelling in an exploratory trial."
Coalition Duchenne is hopeful that CAP-1002 will move forward rapidly and become a treatment for Duchenne. Such a development will realize a simple, but potentially game changing, lateral thought by a mother of a young man with Duchenne. Catherine's 24-year-old son Dusty Brandom has the cardiomyopathy exhibited by all boys and young men with Duchenne. In November 2011, Catherine read an Economist article titled "Repairing Broken Hearts" featuring the research of Dr. Eduardo Marbn and others. She immediately thought about Dusty's heart. Catherine's quest, both as a mother and a leader in the Duchenne community, became to convince the researchers to apply stem cell technology to Duchenne. She wrote to all of the researchers mentioned in the article and pushed her simple thesis that Duchenne related cardiomyopathy would be a good candidate for therapy. Only Dr. Eduardo Marbn responded.
About Coalition Duchenne
Coalition Duchenne was founded in 2011 to raise global awareness for Duchenne muscular dystrophy, to fund research, and to find a cure for Duchenne. Coalition Duchenne is a 501c3 non-profit corporation. Its vision is to change the outcome for boys and young men with Duchenne, to rapidly move forward to a new reality of longer, fulfilled lives, by funding the best opportunities for research and creating awareness.
Coalition Duchenne has several research initiatives that are making advances in potential cardiac and pulmonary treatments for sufferers of Duchenne muscular dystrophy.
Through its Duchenne Without Borders initiative, Coalition Duchenne is helping medically underserved boys and young men with Duchenne worldwide. For example, Coalition Duchenne provides Ambu Bags to Duchenne families in rural Borneo to help maintain pulmonary function.
Catherine Jayasuriya, the founder and executive director of Coalition Duchenne, produced and directed the award winning documentary Dusty's Trail: Summit of Borneo (2013) which has screened internationally and is included in the syllabus at teaching institutions worldwide.
For more information about Coalition Duchenne, visit http://www.coalitionduchenne.org.
About Duchenne muscular dystrophy
Duchenne muscular dystrophy is a progressive muscle wasting disease. It is the most common fatal genetic disease that affects children. Duchenne occurs in 1 in 3,500 male births, across all races, cultures and countries. Duchenne is caused by a defect in the gene that codes for the protein dystrophin. This is a vital protein that helps connect the muscle fiber to the cell membranes. Without dystrophin the muscle cells become unstable, are weakened and lose their functionality. Life expectancy ranges from the mid teenage years to age 30.
About CAP-1002
CAP-1002 consists of allogeneic cardiosphere-derived cells, or CDCs, a type of cardiac progenitor cell. CDCs have been the subject of over 100 peer-reviewed scientific publications and have been administered to approximately 140 human subjects across several clinical trials. CAP-1002 is currently being evaluated in the randomized, double-blind, placebo-controlled Phase II ALLSTAR Clinical Trial in adults who have suffered a large heart attack and in the Phase I/II HOPE Clinical Trial in boys and young men with Duchenne.
About Capricor Therapeutics
Capricor Therapeutics, Inc. is a clinical-stage biotechnology company developing first-in-class biological therapies for cardiac and other medical conditions. Capricor's lead candidate, CAP-1002, is a cell-based candidate currently in clinical development for the treatment of Duchenne muscular dystrophy, myocardial infarction (heart attack), and heart failure. Capricor is exploring the potential of CAP-2003, a cell-free, exosome-based candidate, to treat a variety of disorders. For more information, visit http://www.capricor.com.
For the original version on PRWeb visit: http://www.prweb.com/releases/2017/04/prweb14274775.htm
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Coalition Duchenne Congratulates Capricor Therapeutics on Positive Six-Month Results from its Randomized CAP ... - Benzinga
Perryville family organizing bone-marrow drive Friday for ailing 6-year-old boy – Southeast Missourian
By Sykes24Tracey
Wade Watcher
A Perryville family is organizing a bone-marrow registration drive in hopes of finding a match for their 6-year-old son, who needs a bone-marrow transplant.
Wade Watcher's mother Jenni said for the most part, he's a regular 6-year-old.
"Active and funny and adorable," she said. "He's smart and loves to draw. He likes playing basketball. He's a pretty awesome kid."
But for him to continue leading a normal childhood, Watcher likely would need a bone-marrow transplant.
"We knew that he had a rare disease when he was a baby, and so yearly we have to get a bone marrow biopsy to see if his bone marrow is failing," Watcher said. "It had been fairly normal until December. ... It showed his bone marrow was in the stages of failing and that it was kind of like a waiting game to see if he needs to be sent for a bone marrow transplant or not."
Wade, who suffers from Shwachman-Diamond syndrome, a rare congenital disorder, is stable, but his mother said they don't know for how long.
So they're organizing a registration drive for members of the community to sign up to have their cheek swabbed and see whether they may be a match. The drive will be from 3 to 7 p.m. Friday at the Amvets Post 94 in Perryville.
Watcher said she's not sure how many people are scheduled to participate, but she to register as many people ranging in age from 18 to 55 years old people as possible.
"Anybody that can would be amazing," she said. "It would provide a lot of help for our family as well as other families."
Registration involves filling a form and having a cheek swabbed for about 30 seconds. Donor recruitment coordinator Olivia Haddox said people typically shy away from such drives because they are unsure of what it may mean if they are "matched" with a person in need.
"People are surprised to find how easy it is just to register, but then the next question is always, 'What's going to happen if I get that call?'" she said. "We definitely get that a lot."
There are two ways for the donation to happen if a match is found, she said. About 80 percent of the time, donations are done via peripheral blood stem-cell donation, a four- to eight-hour session in which blood is taken from one arm and filtered through an aphoresis machine to separate the blood from the stem cells. After taking the stem cells, the blood is returned to the donor's body.
"That can kind of be compared to a lengthier platelet or plasma donation," Haddox said. "You don't actually even lose any blood that day; you just lose some stem cells, and you regenerate those in about a week, so what you give you do get back," she said.
People usually watch Netflix while donating, she said, and minor side effects more often come from the series of injections donors receive before the procedure to boost the stem cells. Those injections can cause some fatigue or other side effects.
"Nothing so severe that it might keep anyone out of work," Haddox said. "It's just kind of your body preparing for the donation."
The other, less-common method is an outpatient procedure whereby liquid marrow from the lower back pelvic area is removed.
"And you're actually put under for this procedure, so you're not awake when it happens and you don't feel anything when it happens," Haddox said. "Afterwards, what most people tell me they feel is just a tenderness and a bruising around the site where they removed the marrow. A lot of people equate this to saying, 'I felt like I fell on some ice, and I had a bruise on my hip for a few days.'"
If people can't attend the drive, swab kits can be ordered at dkms.org.
(573) 388-3627
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Perryville family organizing bone-marrow drive Friday for ailing 6-year-old boy - Southeast Missourian