Puppies benefit from stem cell treatment for children with spina bifida – University of California

A pair of English bulldog puppies are the first patients to be successfully treated with a unique therapy a combination of surgery and stem cells developed at the University of California, Davis, to help preserve lower-limb function in children with spina bifida.

Because dogs with the birth defect frequently have little control of their hindquarters, they also have little hope for a future. They are typically euthanized as puppies.

At their postsurgery re-check at 4 months old, however, the siblings, named Darla and Spanky, showed off their abilities to walk, run and play to their doctor,veterinary neurosurgeon Beverly Sturges.

The initial results of the surgery are promising, as far as hind limb control, said Sturges. Both dogs seemed to have improved range of motion and control of their limbs.

The dogs have since been adopted, and continue to do well at their home in New Mexico.

Spina bifida occurs when spinal tissue improperly fuses in utero, causing a range of cognitive, mobility, urinary and bowel disabilities in about 1,500 to 2,000 children born in the U.S. each year. The dogs procedure, which involved surgical techniques developed byfetal surgeon Diana Farmerof UC Davis Health together with a cellular treatment developed by stem cell scientistsAijun WangandDori Borjesson, director of the universitysVeterinary Institute for Regenerative Cures, represents a major step toward curing spina bifida for both humans and dogs.

Farmer pioneered the use of surgery prior to birth to improve brain development in children with spina bifida. She later showed that prenatal surgery combined with human placenta-derived mesenchymal stromal cells (PMSCs), held in place with a cellular scaffold, helped research lambs born with the disorder walk without noticeable disability.

Sturges wanted to find out if the surgery-plus-stem-cell approach could give dogs closer-to-normal lives along with better chances of survival and adoption. At 10-weeks old, Darla and Spanky were transported from Southern California Bulldog Rescue to the UC Davis veterinary hospital, where they were the first dogs to receive the treatment, this time using canine instead of human PMSCs.

Another distinction for Darla and Spanky is that their treatment occurred after birth, since prenatal diagnosis of spina bifida is not performed on dogs, Sturges explained. The disorder becomes apparent between 1 and 2 weeks of age, when puppies show hind-end weakness, poor muscle tone, incoordination and abnormal use of their tails.

UC Davis is the only place where this type of cross-disciplinary, transformational medicine could happen, according to Farmer.

Its rare to have a combination of excellent medical and veterinary schools and strong commitment to advancing stem cell science at one institution, she said.

UC Davis is also home to the One Healthinitiative aimed at finding novel treatmentslike thesefor diseases that affect both humans and animals.

Ive often said that I have the greatest job on the planet, because I get to help kids, Farmer said. Now my job is even better, because I get to help puppies too.

With additional evaluation and U.S. Food and Drug Administration approval, Farmer and Wang hope to test the therapy in human clinical trials. Sturges and Borjesson hope to do the same with a canine clinical trial. They hope the outcomes of their work help eradicate spina bifida in dogs and humans.

In the meantime, the team wants dog breeders to send more puppies with spina bifida to UC Davis for treatment and refinements that help the researchers fix an additional hallmark of spina bifida incontinence. While Darla and Spanky are very mobile and doing well on their feet, they still require diapers.

Further analysis of their progress will determine if the surgery improves their incontinence conditions, Sturges said.

Funding for this project was provided by the Veterinary Institute for Regenerative Cures (VIRC) at the UC Davis School of Veterinary Medicine, and the Surgical Bioengineering Lab at the UC Davis School of Medicine. Private donations to the veterinary school for stem cell research also contributed to this procedure. Farmer and Wangs spina bifida research is supported by funding from the National Institutes of Health, the California Institute for Regenerative Medicine, Shriners Hospitals for Children and the March of Dimes Foundation.

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Puppies benefit from stem cell treatment for children with spina bifida - University of California

FDA to crack down on ‘unscrupulous’ stem cell clinics …

FILE -- The Sawgrass Medical Center, where the U.S. Stem Cell Clinic operates, in Sunrise, Fla., March 15, 2017. The Food and Drug Administration issued a warning letter to U.S. Stem Cell and announced a crackdown on other dangerous stem cell clinics, while at the same time pledging to ease the path to approval for companies and doctors with legitimate treatments in the growing field. (Scott McIntyre/The New York Times)

The U.S. Food and Drug Administration on Monday promised a nationwide crackdown on unscrupulous stem cell clinics, following years of reports that some clinics have offered lofty claims of potential medical benefits with scant, if any, medical evidence.

These dishonest actors exploit the sincere reports of the significant clinical potential of properly developed products, as a way of deceiving patients and preying on the optimism of patients facing bad illnesses. This puts the entire field at risk, wrote FDA Commissioner Dr. Scott Gottlieb, who was appointed in May. Products that are reliably and carefully developed will be harder to advance if bad actors are able to make hollow claims and market unsafe science.

Gottliebs statement, published Monday on the FDAs website, highlighted a warning letter issued to one Florida stem cell clinic on Thursday and the direct seizure of products from another clinic in California on Friday. Gottlieb said the agency is aware of other cases where unproven stem cell treatments have clearly harmed patients, and he promised additional enforcement actions in the coming months.

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‘Reprogrammed’ Stem Cells Fight Parkinson’s Disease in Monkeys – Voice of America

LONDON

Scientists have successfully used "reprogrammed" stem cells to restore functioning brain cells in monkeys, raising hopes the technique could be used in the future to help patients with Parkinson's disease.

Since Parkinson's is caused by a lack of dopamine made by brain cells, researchers have long hoped to use stem cells to restore normal production of the neurotransmitter chemical.

Now, for the first time, Japanese researchers have shown that human induced pluripotent stem cells (iPS) can be administered safely and effectively to treat primates with symptoms of the debilitating disease.

So-called iPS cells are made by removing mature cells from an individual often from the skin and reprogramming them to behave like embryonic stem cells. They can then be coaxed into dopamine-producing brain cells.

The scientists from Kyoto University, a world-leader in iPS technology, said their experiment indicated that this approach could potentially be used for the clinical treatment of human patients with Parkinson's.

In addition to boosting dopamine production, the tests showed improved movement in affected monkeys and no tumors in their brains for at least two years.

The human iPS cells used in the experiment worked whether they came from healthy individuals or Parkinson's disease patients, the Japanese team reported in the journal Nature on Wednesday.

"This is extremely promising research demonstrating that a safe and highly effective cell therapy for Parkinson's can be produced in the lab," said Tilo Kunath of the MRC Center for Regenerative Medicine, University of Edinburgh, who was not involved in the research.

The next step will be to test the treatment in a first-in-human clinical trial, which Jun Takahashi of Kyoto University told Reuters he hoped to start by the end of 2018.

Any widespread use of the new therapy is still many years away, but the research has significantly reduced previous uncertainties about iPS-derived cell grafts.

The fact that this research uses iPS cells rather human embryonic stem cells means the treatment would be acceptable in countries such as Ireland and much of Latin America, where embryonic cells are banned.

Excitement about the promise of stem cells has led to hundreds of medical centers springing up around the world claiming to be able to repair damaged tissue in conditions such as multiple sclerosis and Parkinson's.

While some treatments for cancer and skin grafts have been approved by regulators, many other potential therapies are only in early-stage development, prompting a warning last month by health experts about the dangers of "stem-cell tourism."

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'Reprogrammed' Stem Cells Fight Parkinson's Disease in Monkeys - Voice of America

FDA announces first US gene therapy approval for cancer treatment – CNN

The treatment, called Kymriah, aims to give some patients a second chance after first-line drugs have failed. This may happen in up to a fifth of patients, according to the FDA.

Each dose of Kymriah contains a patient's own immune cells, which are sent to a lab to be genetically modified using a virus. This therapy -- known as chimeric antigen receptor T-cell therapy, or CAR-T -- gives the cells the ability to recognize and kill the source of the cancer.

"We've never seen anything like this before and I believe this therapy may become the new standard of care for this patient population," said Dr. Stephan Grupp, director of cancer immunotherapy at Children's Hospital of Philadelphia, which spearheaded this research.

Based on available data, patients on the treatment have had an 89% chance of surviving at least six months and a 79% chance of surviving at least a year, with most being relapse-free at that point.

Most patients with ALL recover through other treatments such as radiation, chemotherapy and stem cells. But if the cancer recurs, the prognosis is poor.

"Kymriah is a first-of-its-kind treatment approach that fills an important unmet need for children and young adults with this serious disease," Dr. Peter Marks, director of the FDA's Center for Biologics Evaluation and Research, said in a statement.

The one-time treatment has a boxed warning for cytokine release syndrome or CRS, a life-threatening side effect that can cause blood pressure to drop dangerously low. It is caused by overactive genetically modified immune cells. The FDA said hospitals and clinics must become certified to distribute the treatment, meaning they are prepared to recognize and treat CRS and other potentially fatal neurological events. Novartis said it hopes to have an initial network of 20 treatment centers within a month with plans to expand that to 32 by the end of the year.

Kymriah has a $475,000 price tag; however, patients who do not respond within a month of treatment will not be charged, according to Novartis.

"Novartis is collaborating with (Centers for Medicaid Services) to make an outcomes-based approach available to allow for payment only when pediatric and young adult ALL patients respond to Kymriah by the end of the first month. Future potential indications would be reviewed for the most relevant outcomes-based approach," the drug company said in a statement.

On Wednesday, the FDA also expanded approval for another drug, tocilizumab, to treat CRS in patients 2 and older.

Novartis is required to conduct followup study to assess the safety of the treatment long-term.

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FDA announces first US gene therapy approval for cancer treatment - CNN

Dr. Pawel Muranski to Head New Cellular Immunotherapy Laboratory at NewYork-Presbyterian/Columbia University … – Newswise (press release)

Newswise New York (August 31, 2017) Scientific innovator and physician Dr. Pawel Muranski has joined NewYork-Presbyterian and Columbia University Medical Center (CUMC) as director of cellular immunotherapy at the newly established Good Manufacturing Practices (GMP) cell production lab and assistant director of Transfusion Medicine and Cellular Therapy. He will also serve on the faculty of CUMC as Assistant Professor of Medicine, Pathology and Cell Biology, a principal investigator at Columbia Center for Translational Immunology (CCTI) and a member of Columbias Herbert Irving Comprehensive Cancer Center.

Were thrilled to have Dr. Muranski joining us to continue his innovative work, said Dr. Gary Schwartz, chief of the Division of Hematology/Oncology at NewYork-Presbyterian/CUMC and the Clyde 56 and Helen Wu Professor of Oncology (in medicine) at CUMC. His approach to T cell-based therapy holds so much potential and could revolutionize care for cancer patients, transplant patients and others.

Dr. Muranski is a hematologist who specializes in bone marrow transplantation and in developing adoptive T cell therapies, in which white blood cells called T lymphocytes are removed from a patient or a donor and then programmed to target viral infections, leukemic cells and solid tumors. Adoptive transfer of T cells, including Chimeric Antigen Receptor (CAR)-T therapy has shown great promise in early trials of patients with leukemia, lymphoma and several solid cancersin some cases leading to a complete remission.

Dr. Muranskis research will continue to focus on exploiting and enhancing the capability of engineered T cells to recognize and target cancerous cells or dangerous viruses. He has a particular interest in developing CD4+ T helper cellsthe master orchestrators of immune responseas a potentially powerful weapon against cancer. His T cells can also target viral infections in patients whose immune systems have been weakened by bone marrow or organ transplantation, cancer treatment, or autoimmune diseases.

Despite recent spectacular advances in the field of cancer immunotherapy, very few institutions have GMP laboratories with the capacity to grow and manipulate T cells, said Dr. Muranski. NewYork-Presbyterian and Columbia University Medical Center are now positioned to become leaders in cutting-edge cellular immunotherapies. Im excited to work with the team here on developing a comprehensive program that brings these innovative treatments to our patients.

In addition to his work in the GMP lab, Dr. Muranski will be working with Dr. Prakash Satwani, a pediatric hematologist and oncologist at NewYork-Presbyterian and associate professor of pediatrics at CUMC, on an upcoming major CAR-T cell initiative. He will also work closely with Dr. Markus Mapara, director of the Adult Blood and Marrow Transplantation Program at NewYork-Presbyterian/Columbia and professor of medicine at CUMC.

Dr. Muranski trained as a fellow at the Surgery Branch, National Cancer Institute (NCI), National Institutes of Health (NIH) in Bethesda, Maryland, where he performed innovative studies aimed at understanding of the role of CD4+ T cells as mediators of curative anti-tumor immunity. Most recently, he served in Hematology Branch, National Heart, Lung and Blood Institute (NHLBI) at the NIH, where his research focused on using T cell-based therapies to prevent viral infections in patients undergoing donor-based stem cell transplantation for blood cancers.

He earned his medical degree from the Medical University of Warsaw in Poland before completing a research fellowship at the Institute for Molecular Medicine and Genetics, Medical College of Georgia and a residency at St. Francis Hospital in Evanston, Illinois. He completed a clinical fellowship in hematology and oncology at the National Institutes of Health in Bethesda, Maryland.

NewYork-Presbyterian

NewYork-Presbyterian is one of the nations most comprehensive, integrated academic healthcare delivery systems, whose organizations are dedicated to providing the highest quality, most compassionate care and service to patients in the New York metropolitan area, nationally, and throughout the globe. In collaboration with two renowned medical schools, Weill Cornell Medicine and Columbia University Medical Center, NewYork-Presbyterian is consistently recognized as a leader in medical education, groundbreaking research and innovative, patient-centered clinical care.

NewYork-Presbyterian has four major divisions:

Columbia University Medical Center

Columbia University Medical Centerprovides international leadership in basic, preclinical, and clinical research; medical and health sciences education; and patient care. The medical center trains future leaders and includes the dedicated work of many physicians, scientists, public health professionals, dentists, and nurses at the College of Physicians and Surgeons, the Mailman School of Public Health, the College of Dental Medicine, the School of Nursing, the biomedical departments of the Graduate School of Arts and Sciences, and allied research centers and institutions. Columbia University Medical Center is home to the largest medical research enterprise in New York City and State and one of the largest faculty medical practices in the Northeast. The campus that Columbia University Medical Center shares with its hospital partner, NewYork-Presbyterian, is now called the Columbia University Irving Medical Center. For more information, visit cumc.columbia.eduorcolumbiadoctors.org.

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Dr. Pawel Muranski to Head New Cellular Immunotherapy Laboratory at NewYork-Presbyterian/Columbia University ... - Newswise (press release)

For Lowell native, stem cell match becomes a match as friends – Lowell Sun

From left to right: Richard Stone, a doctor at Dana-Farber Cancer Institute in Boston, poses with Peter Karalekas (center), 76, and Matthew Churitch, 22. Churitch donated stem cells to Karalekas two years ago, and he visited Dana-Farber with Karalekas earlier this summer. (Courtesy photo)

BOSTON -- After winding his way through Massachusetts, Connecticut, New Hampshire and Maine for 76 years, Peter Karalekas has a proclamation: He's a Southerner now.

He still lives in Kittery, Maine, just about an hour from the Lowell middle school where he taught for 21 years.

He has no plans to move.

Rather, Karalekas considers himself a Southerner because of his stem cells.

He never exactly felt all that sick.

Karalekas worked tirelessly for decades, first as a teacher and coach at the James S. Daley Middle School in Lowell and then as the owner of a half-dozen T-Bones restaurants across New Hampshire.

Even despite the 12-hour days, seven days a week, in the grind of the restaurant industry, Karalekas felt healthy and rarely fell ill.

Peter Karalekas, left, a 76-year-old former Lowellian, smiles during his first meeting with Matthew Churitch, 22, of Nashville, Tennessee, who helped save Karalekas life by donating stem cells. (Courtesy photo)

The two, who do not have children, moved to Kittery 17 years ago.

Everything started to change in 2014.

Karalekas recalls being "short-winded," but he had very few other symptoms when he was diagnosed with myelodysplastic syndrome, a rare type of cancer in which the bone marrow is damaged and cannot produce enough blood cells.

The prognosis was not good.

"They said the only thing that would save me was a stem cell transplant," Karalekas said. "Otherwise, I had a couple of months to live, because my cells were all dropping drastically.

He went onto a registry, hoping for a donor to pop up, but doctors told him it could take from six months to two years to find the right match. Even with a transplant, Karalekas said, his chances of success were "30 to 40 percent."

The call came four weeks later.

Matthew Churitch got his call quickly, too.

He joined the National Marrow Donor Program's Be the Match Registry in 2014, the summer between his freshman and sophomore years at Clemson University. His mother had been on the registry to donate for years. Churitch's decision was simple: When a friend was diagnosed with leukemia, he knew he should sign up, too.

He did the requisite cheek swab, unsure if he would ever even be contacted to donate. By the time he had finished the following semester, he got the call.

A match was found.

Churitch went through several more levels of testing and preparation to donate stem cells to a stranger. He went to Clemson's student health center to have blood drawn.

He returned to his native Nashville, Tennessee, going to a medical center 10 days in a row to receive shots in his stomach that would stimulate his bone marrow and prepare his cells for transplant.

He sat for eight hours, a needle in each arm as his stem cells were filtered out so they could be transferred to Boston.

"Getting the shots isn't fun," he said. "You're pretty sore afterward for a few weeks. But knowing that the person on the other end is in hundreds and hundreds times more pain than any donor would ever go through -- that kind of pushed me through."

Karalekas and Churitch first connected via an anonymous letter, per the transplant registry's rules, updating Churitch on Karalekas's lengthy, isolated recovery. They were able to speak directly after a year.

Churitch dialed Karalekas' number on a lengthy walk to class, took a deep breath and hit the call button. Moments later, both men were crying and laughing.

"That was really awesome, just being able to hear his voice and recognize that there's somebody else on the other end of this," Churitch said. "A lot of people don't get the chance to connect with their recipients or their donors."

Karalekas wanted more. He told his wife early on that he wanted to meet his "angel from heaven," so when Churitch graduated Clemson earlier this year, Karalekas paid to bring the 22-year-old and his mother to New England.

In late June, Karalekas and his wife pulled into a pickup lane at Logan International Airport in Boston.

"I got out of the car, I charged over, and I gave them both a huge hug," Karalekas said.

Karalekas showed Churitch and his mother around for five days.They went on a private tour of Fenway Park; they wandered the historic streets of Portsmouth, New Hampshire; they visited Dana-Farber together to meet the team that treated Karalekas.

Both families quickly bonded. Karalekas recalls his brother George asking Churitch about his portable phone charger, expressing curiosity about how convenient it was. A few weeks later, a brand-new portable charger arrived at George's door, a gift from Churitch.

In January, Karalekas and his wife will vacation in Arizona and will cheer on Churitch's mother -- without Churitch even present -- in the Phoenix Marathon.

Donor and recipient talk every week.

"It's like we're a very, very close-knit family now," Karalekas said. "He's the son we never had."

Churitch is now in his first year at the University of South Carolina School of Medicine Greenville with hopes of becoming a physician. He hopes to use Karalekas's experience as inspiration for any patients facing future hardship, and he hopes that others, especially young people, will see their success and join the registry.

"You never know where that will take you," he said. "You can gain a friend for life, impact somebody and their family in need."

Karalekas said he feels he has a new life: His chances of beating the disease are now 97 percent, he says, up from the 30 percent or 40 percent when he started treatment. Thanks to the transplant from a handsome, athletic college student in Tennessee.

"I said, 'I'm a Southerner now,'" Karalekas said. "My stem cells are 99 percent this gentleman. I'm 99 percent him."

Follow Chris on Twitter @ChrisLisinski.

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For Lowell native, stem cell match becomes a match as friends - Lowell Sun

Brain cell replacement for Parkinson’s boosted by monkey study – The San Diego Union-Tribune

A brain cell replacement therapy reduced Parkinsons disease symptoms in monkeys, Japanese researchers report in a study released Wednesday. The positive result boosts prospects to test the therapy in people.

The goal is to implant neurons derived from stem cells into the brains of Parkinsons patients, a project pursued by scientists in San Diego, New York, Britain and Sweden as well as in Japan. If all goes well, the neurons will function as replacements for those destroyed in the disease.

In addition, human testing of a related brain cell therapy from Carlsbads International Stem Cell Corp. is already under way in Australia.

While treatments exist for the movement disorders caused by Parkinsons, none of them actually halt progression. Replacing the brain cells destroyed in Parkinsons holds the promise of actually reversing the disease.

Moreover, success with Parkinsons could pave the way to treating many other neurodegenerative diseases, such as ALS (Lou Gehrigs disease) and perhaps Alzheimers, along with brain and spinal cord injuries. These afflictions cost hundreds of billions annually, and most importantly, produce immense suffering in patients and caregivers.

Years of extensive research are required before any such therapy can be tried in people. Testing in monkeys or other primates is often regarded as the last step before human treatment can be contemplated.

The study was published in the journal Nature. Its senior author was Jun Takahashi, a prominent stem cell researcher at Kyoto University in Kyoto, Japan. Go online to j.mp/parkips for the study.

There is precedent to suggest the therapy might work. Beginning decades ago, brain cells taken from human fetuses have been implanted into the brains of Parkinsons patients, with mixed results. Some patients experienced improved movement control. But others gained nothing, or experienced uncontrolled movements.

Scientists in the field say using stem cells should provide improved results. Stem cells can be made in greater quantity than the limited number of fetal brain cells available. In addition, the stem cells and neurons made from them can be analyzed for quality before implantation.

The study was praised by regenerative medicine researcher Tilo Kunath at the University of Edinburgh, in comments provided by the UK Science Media Centre.

This is extremely promising research demonstrating that a safe and highly effective cell therapy for Parkinsons can be produced in the lab, Kunath said.

Such a therapy has the potential to reverse the symptoms of Parkinsons in patients by restoring their dopamine-producing neurons. The next stage will be to test these therapies in a first-in-human clinical trial.

In the study, researchers produced neurons that secrete dopamine, a neurotransmitter deficient in Parkinsons disease. These neurons were made from human stem cells derived from both healthy people and those with Parkinsons.

The researchers then implanted the human neurons into 10 monkeys whose own dopamine-making neurons had been destroyed. The monkeys were given immunosuppressive drugs to prevent rejection of the human cells.

The human neurons integrated into the brains of the monkeys and functioned as dopamine-making neurons. The monkeys improved in movement ability, save for one monkey that became ill and was euthanized. Both cells from healthy and Parkinsons patients were effective.

A companion study in Nature Communications demonstrated a method of immune-matching the cells to reduce the immune response. Takahashi was also senior author of that study. Go online to j.mp/ipsimmune for the study.

Both studies used artificial embryonic stem cells, called induced pluripotent stem cells (IPS). These act-alike cells are not derived from embryos, but are genetically reprogrammed from adult cells, usually skin cells.

The IPS cells appear to act virtually identically to embryonic stem cells, but dont raise the ethical objections many have to using embryonic stem cells. These cells were invented in 2006 by a team led by Shinya Yamanaka, a co-author of the Nature Communications study.

Moreover, the cells can be made from the patients themselves, which is not expected to cause an immune reaction. This is the approach taken by the San Diego team, including scientists at The Scripps Research Institute.

Carlsbads International Stem Cell Corp. uses a different approach. It starts with unfertilized, or parthenogenetic, human egg cells. These are grown into immature neurons that are implanted. The cells are expected to grow not only into dopamine-making neurons, but other kind of brain cells that preserve the remaining neurons.

The Australian clinical trial has gathered evidence of safety, and continued testing is under way determine efficacy.

The Nature study dovetails with research by the San Diego group, Summit for Stem Cell, (www.summitforstemcell.org), including scientists at The Scripps Research Institute and doctors at Scripps Health.

The group proposes to treat Parkinsons patients with neurons grown from their own IPS cells. The scientists have received funding from the California Institute for Regenerative Medicine, the states stem cell agency.

The studies support the personalized approach that we are taking for a neuron replacement therapy for Parkinson's disease patients, said Jeanne Loring and Andres Bratt-Leal, stem cell scientists at The Scripps Research Institute.

Two points from the studies should be highlighted, Loring and Bratt-Leal said by email.

Parkinson's disease is a late-onset disorder, they said. That means that there was nothing wrong with the neurons that people with Parkinson's were born with. Few PD patients have a family history of the disease, which suggests that genetic mutations did not cause their disease.

So for the great majority of patients, transplantation of their own neurons is a promising approach to relieving symptoms, without having to take expensive and risky immunosuppressive drugs, they said.

The Summit for Stem Cell scientists are members of an international partnership of laboratories developing neuron replacement therapies for Parkinsons, called GForce PD.

Takahashi belongs to the partnership, as do scientists in the UK, Sweden and New York. These use both embryonic and IPS stem cells. The Summit for Stem Cell effort is the only one using patient-matched IPS cells, Loring and Bratt-Leal said.

Brain cells reprogrammed to make dopamine, with goal of Parkinsons therapy

Parkinson's stem cell therapy shows signs of safety

Parkinson's therapy funded by California's stem cell agency

Dopamine-making neurons can be chemically controlled in animal model of Parkinson's

Stem cell clinical trial for Parkinson's begins

Summit for Stem Cell

bradley.fikes@sduniontribune.com

(619) 293-1020

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Brain cell replacement for Parkinson's boosted by monkey study - The San Diego Union-Tribune

Collaboration Key to 3D Bioprinting Stem Cell Research Success at BioFab3D@ACMD in Australia – 3DPrint.com

Medical researchers and scientists have created all kinds of medical marvels, from brain tissue and cartilage to a heart and a pancreas, by 3D printing stem cells. In Australia, Swinburne University of Technology PhD candidateLilith Caballero Aguilar is currently collaborating on a project with surgeons and researchers at BioFab3D@ACMD, the countrys first bioengineering facility based in a hospital, about how stem cells are fed once theyre inside the body. She is working to develop methods to control the rate of release for growth factors, which stem cells need for development once theyve been implanted, and the research could help doctors use biological 3D printing techniques to regenerate damaged or missing tissue.

Caballero Aguilar says that working alongside surgeons and other university researchers at the facility has had a major impact on her work.

We complement each other. If I have doubt, we can discuss it and reshape the project as we go, which helps to reach a better outcome.At the end of the day, everyone is doing a bit of work in a big project. It feels very rewarding,Caballero Aguilar said.

The facility was established through a partnership between Swinburne, St Vincents Hospital Melbourne, the ARC Centre of Excellence for Electromaterials Science, the University of Melbourne,RMIT University, and the University of Wollongong Australia. Biology experts, surgeons, researchers, and biomedical engineers work at the facility to pioneer innovations, like nerves, re-engineered limbs, and tissues.

Cellink Inkredible Bioprinters [Image: Swinburne]

Caballero Aguilars stem cell work is part of two of the facilitys major research projects, one which focuses on repairing damaged muscle fibers and another regarding damaged cartilage regeneration; both are using advanced technologies, like bioprinting, to implant materials into the body, including the handheld 3D Biopen that allows surgeons to draw biomaterials into a patient directly and has been successfully tested, using knee cartilage, on six sheep.

BioPen

She is working to manipulate polymer materials into release mechanisms for stem cell growth factors, which would form part of the 3D bioink drawn into the body. Controlling the delivery of growth factors is very important stem cells take at least six weeks to grow into tissue, so the growth factors need to be slowly released over the entire time period.Caballero Aguilar shakes an oil and water solution at an intense rate, which is called the emulsion method, to create microspheres, which are crosslinked to form a substance thats able to hold the growth factors.

Swinburne Professor of Biomedical Electromaterials Science Simon Moulton, who is Caballero Aguilars supervisor, said that the success of her stem cell research project was helped along by the opportunity to collaborate directly with orthopaedic surgeons and muscle specialists at St Vincents Hospital.

Swinburne PhD candidate Lilith Caballero Aguilar and Professor Simon Moulton in a lab at BioFab3D@ACMD. [Image: Swinburne]

Professor Moulton said, Without this space, Liliths project would be a much smaller project without the translation benefit.It still would be great research done at a very high level, she would have publications and be able to graduate, but working in this collaborative environment, she can achieve all of that, while also having her research go into a clinical outcome that actually has benefit to patients.

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Collaboration Key to 3D Bioprinting Stem Cell Research Success at BioFab3D@ACMD in Australia - 3DPrint.com

British Mitochondria Study Could Provide New Approaches to Treating ALS – ALS News Today

Researchers at University of Exeter Medical School in the United Kingdom have started an ambitious project to investigate how mitochondria flaws may cause the neuron cell death associated with ALS.

Not only may their insights provide a better understanding of how amyotrophic lateral sclerosis arises, but it also may lay a foundation for the development of new treatments.

Mitochondria are the cells power plants, converting nutrients to energy. Researchers believe abnormalities in their functioning may contribute to ALS.

The team, led by Dr. Akshay Bhinge of Exeters Living Systems Institute, noted that mutations in the TBK1 and OPTN genes were recently linked to ALS. The genes take part in a cell process called mitophagy, or the removal of defective mitochondria.

Although studies suggest that mutations in the genes can cause disease, researchers have no idea how.

To find out, the research team plans to use induced pluripotent stem cells. These are stem cells that, in the lab, are forced back into development to become stem cells. Researchers will use a gene editing tool called CRISPR-Cas9 to introduce mutations they find in patients.

The cells will then be grown into spinal motor neurons so researchers can study the mitophagy process. Among other things, they will want to how many mitochondria end up in cell compartments involved in breaking down worn-out cell components.

They will also look at whether the mutations affect how neurons signal, and if those neurons are more prone to dying than typical neurons.

Understanding the major effects of a mutation is a key step toward understanding how a disease such as ALS arises. The insight may not necessarily provide information that allows researchers to develop drugs that stop or slow the disease process, however.

To obtain information that may enable them to develop drugs, the team will also examine how a mutation affects the activity of all the genes in a cell. Using this information, they will then attempt to reverse the defects.

It is possible that other mutations such as the faulty SOD1 and FUS genes that are linked to ALS that runs in families may alter the same molecular pathways that the TBK1 and OPTN genes do, and thus cause the same molecular flaws. To test for this possibility, the team will examine movement-control neurons that have these mutations.

The researchers are in the midst of pursuing a collaboration with a pharmaceutical company that could help them turn their findings into therapies.

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Frawley, Mindell/Brody, Calkins Awards Recognize 5 for Excellence – UB School of Medicine and Biomedical Sciences News

Resident Asma Mursleen, MD (center) with Roseanne C. Berger, MD (left), and Michael E. Cain, MD was honored for her research at the 20th annual Scholarly Exchange Day.

Published August 30, 2017

Trainees and a student in the departments of Medicine, Biomedical Engineering and Pediatrics have received awards for their research.

The two trainees to receive support from theThomas F. Frawley, MD, Residency Research Fellowship Fundare:

Asma Mursleen, MD Resident in theDepartment of Medicine Project Title: Defining the Role of CDC-derived Exosomes on Macrophage Polarization and Modulation of Cardioprotection Following Myocardial Infarction

Amanda Przespolewski, DO A 2017 alumna of the hematology/oncology fellowship Project title: Dual Enhancement of Immune Responses and Inhibition of Marrow Vasculature in Acute Myeloid Leukemia

The awardsupports medical or surgical residents, fellows and new graduates for whom research represents a primary interest and passion.

Frawley, a 1944 graduate of the medical school, was a nationally recognized endocrinology researcher, president of the American College of Physicians and chair of medicine at Saint Louis University School of Medicine.

The 2017 recipients of the Eugene R. Mindell, MD, and Harold Brody, MD 61, PhD, Clinical Translational Research Awardare:

Yongho Bae, PhD Assistant professor in theDepartment of Pathology and Anatomical Sciences Project Title: Effect of Arterial Stiffening on Vascular Smooth Muscle Cell Mechanotransduction

Kyle Indiana Mentkowski Masters candidate in the Department of Biomedical Engineering Project Title: Development of a Targeted Cardiomyocyte Delivery System Utilizing Cardiosphere-Derived Cell Exosomes

The award recognizes junior research scientists for the best basic science research that seeks to solve a clinical problem.

Mindell chairedUBs Department of Orthopaedics from 1964 to 1988. A past president of the American Board of Orthopaedic Surgery, he is credited withinitiating the boards certifying process for orthopaedic surgeons.

Brody was the chair of anatomy and cell biology from 1971 to 1992. He founded UBs Brain Museum, a world-class collection of brain specimens and slides.

The 2017 honorees for the Evan Calkins, MD, Fellowship for Community-Based Researchare:

Raed Al Yacoub, MD Resident in the Department of Medicine Project Title: Enhancing the Prevention of Microvascular Complications of Diabetes Type 2: A Resident-Led QI Project

Prerana Baranwal, MD Resident in the Department of Pediatrics Project Title: Addressing Childhood Obesity Through Dyslipidemia Screening: Measuring Frequency of Dyslipidemia Screening with Substitution of Random Lipid Panel for Fasting Lipid Panel

The award supports residents, fellows and junior faculty who conduct community-based research or quality improvement projects.

Calkins was chair of the UB Department of Internal Medicine, division chief of geriatrics and founder of the geriatrics fellowship. He served as director of medicine at Meyer Memorial Hospital (now Erie County Medical Center) for 12 years.

The award is a product of his conviction that medical institutions have an obligation to improve the quality of, and access to, health care throughout the community.

Amro Elshoury, MBBCh, a trainee in the hematology/oncology fellowship, received an honorable mention for the Frawley award. Elshourys project was: The Effect of Extra-Physiologic Oxygen Shock / Stress (EPHOSS) On Human Bone Marrow Stem Cell Viability And Multi-Potency.

Roseanne C. Berger, MD, senior associate dean for graduate medical education, presented the awards at this years Scholarly Exchange Day.

The keynote speaker,Steven D. Schwaitzberg, MD, professor and chair of surgery, presented a talk titled Preparing Students and Residents for 21st Century Surgery.

Michael E. Cain, MD, vice president for health sciences and dean, Jacobs School of Medicine and Biomedical Sciences, gave school updates and introductory remarks at the event.

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Frawley, Mindell/Brody, Calkins Awards Recognize 5 for Excellence - UB School of Medicine and Biomedical Sciences News