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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 ... and more »

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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 Stem Cells Comments Off on Bone marrow drive for Allums siblings at ULM, other locations – Monroe News Star | April 26th, 2017

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

Bone Marrow Stem Cells Comments Off on Bone marrow drive set for local youth – Perry County Republic Monitor | April 26th, 2017

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

Bone Marrow Stem Cells Comments Off on TGF-/MAPK signaling mediates the effects of bone marrow mesenchymal stem cells on urinary control and interstitial … – UroToday | April 26th, 2017

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

Skin Stem Cells Comments Off on A New Technique Transforms Human Skin Into Brain Cells – Futurism – Futurism | April 26th, 2017

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

Skin Stem Cells Comments Off on A mechanism shared by healing wounds and growing tumors – The Rockefeller University Newswire | April 26th, 2017

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

Skin Stem Cells Comments Off on Researchers Invent Stem Cell Capable of Becoming an Entire Embryo – Futurism | April 26th, 2017

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

Skin Stem Cells Comments Off on ‘Minibrains’ in a dish shed a little light on autism and epilepsy – 89.3 KPCC | April 26th, 2017

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, ... and more »

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Discovery offers new hope to repair spinal cord injuries: Scientists ... - Science Daily

Spinal Cord Stem Cells Comments Off on Discovery offers new hope to repair spinal cord injuries: Scientists … – Science Daily | April 25th, 2017

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

Spinal Cord Stem Cells Comments Off on Custer considered for stem cell study | The Miami Student – Miami Student | April 25th, 2017

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

Cardiac Stem Cells Comments Off on Coalition Duchenne Congratulates Capricor Therapeutics on Positive Six-Month Results from its Randomized CAP … – Benzinga | April 25th, 2017

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.

tgraef@semissourian.com

(573) 388-3627

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Perryville family organizing bone-marrow drive Friday for ailing 6-year-old boy - Southeast Missourian

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The way to the Red Planet and other mysterious worlds is being inspired by the villainous Khan from the blockbuster films, according to new research.

The use of stem cell technology may mean the difference between life and death on any attempt to travel beyond Earth into the wilderness of space.

So the first person to walk on Mars is likely to be selected from the growing group of people whose parents took the step to store their child's stem cells at birth.

Stem cells are 'blank' cells that can be reprogrammed to turn into any other cell in the body, enabling the replacement of damaged cells.

More and more British parents, including TV presenter Natalie Pinkham and dancer Darcey Bussell, are paying more than 2,000 to freeze samples from their babies' umbilical cords at birth.

Stem cells are also found in bone marrow and some body tissue, but the procedure to harvest them from umbilical cords is less risky.

Adventurous Mars pioneers will have to be especially prepared for the dangerous trip, which could expose them to cancer and other diseases, through carefully researched gene therapy.

1 of 16

We wince at the thought of genetically engineered humans

Mark Hall

Mark Hall, spokesperson for the UK's leading stem cell storage and diagnostics company StemProtect, said: "We wince at the thought of genetically engineered humans.

"And we are not going to create a Khan from Star Trek specifically to get to another planet. Getting humans to Mars and beyond will be both expensive and dangerous.

"But the scientific by-products - such as huge leaps in stem cell medicine - will benefit humanity for centuries to come."

Genetic engineering has featured in two Star Trek movies, and a number of TV episodes.

IG

1 of 14

This still image strikes an uncanny resemblance to a figure of a woman

Khan, who appeared in Space Seed and Star Trek II: The Wrath of Khan, was modified to make him stronger and to give him greater stamina and intellectual capacity than a regular human.

Mr Hall said: "The first human to walk on Mars may not even be born yet - but that's an advantage."

StemProtect believes advanced medical techniques will be required to cope with the rigours of interplanetary space.

While a trip to Mars may appear "just around the corner" in galactic terms, it is highly possible exposure to radiation along the way could lead to the astronauts developing leukaemia and other cancers even before they arrived.

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This means future travellers will have to be 'immunised' before they leave Earth.

Mr Hall said: "There was an article in The Times suggesting elephants would make ideal Martian travellers because they'd be largely immune to the radiation.

"But those laughing at the ridiculous sounding headline completely missed the point - the fact is scientists are already working on ways of getting humans there and back alive."

Recent research has shown radiation in deep space increase the risk of leukaemia while long term exposure to micro gravity may leave astronauts open to infection.

The three year round trip to Mars would affect humans at the stem cell level, leaving them with a drastically lowered immune system, NASA funded scientists say.

And NASA's own findings say stem cells may be crucial to the future of space travel, particularly how they respond in a low gravity environment.

One study showed stem cells flown in space and then cultured back on Earth had greater ability to self renew and generate any cell type, changing more easily into specialised heart muscle cells, for instance.

Mr Hall said an astronaut will have to be prepared for the journey "quite literally at the stem cell level."

He explained: "That means working with the best and most effective stem cells available to the patient - those harvested from the umbilical cord at birth."

GETTY STOCK IMAGE

The therapies required to 'immunise' humans to space travel are still being researched.

And with most space based science, it can only mean huge benefits to mankind back down on Earth when it comes to fighting otherwise deadly conditions and diseases.

Stem cells have the ability to treat a potentially infinite range of illnesses and diseases.

Stem cell therapy is already being used all over the world to treat some cancers and stroke victims - and there is fast progress being made in many other areas, including Parkinson's and Alzheimer's disease.

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SUPERHUMANS: Mars 'will be colonised by genetically engineered Star Trek-style beings' - Express.co.uk

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Skin stem cells used to generate new brain cells: Study to advance ... Science Daily Using human skin cells, neurobiologists have created a method to generate one of the principle cell types of the brain called microglia, which play a key role in ... and more »

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Skin stem cells used to generate new brain cells: Study to advance ... - Science Daily

Skin Stem Cells Comments Off on Skin stem cells used to generate new brain cells: Study to advance … – Science Daily | April 25th, 2017

One of the finalists in the Governors Business Plan Contest, Cellular Logistics, is making another run at the top prize as it pursues commercialization of its biomaterials.

Two years ago, the company submitted a plan and made it to the semifinal round before being eliminated.

That was premature, but a good experience to go through in terms of preparation for this year, where we have a much more clear path and stronger team, said Adam Bock, CFO for Cellular Logistics. Weve been here before, but its always exciting.

The company just finished up a family and friends funding round last quarter, which raised $400,000. Its still in its growth stages, according to Bock, who characterizes Cellular Logistics as a virtual company at this point.

We have our business and tech team who operate wherever we need to be -- no dedicated office or lab, Bock said. All work has been done at Wisconsin Institute for Medical Research.

He added the company will be prepared to transition into a commercial space in about six months, saying were right at that crossover point.

Eric Schmuck, Cellular Logistics chief science officer, invented the companys patented biomaterials and heads the studies currently being done to test them.

He initially had the idea as a grad student in the UW-Madison Department of Physiology, where he was working with materials on which to grow stem cells. He realized the material he was experimenting with had unique characteristics which could be applicable in other ways.

It was novel; no one had done anything like it, Schmuck said. WARF agreed to patent the idea in 2011. Weve been tinkering with it in UW ever since.

The company is in the preclinical phase, currently testing an injectable supportive extracellular matrix in the hearts of mice. While results for these tests will have to wait until they are complete, Schmuck says that for earlier iterations, tests have been extremely promising.

He has seen really good restoration of function, with earlier tests, including powerful reduction in cardiac dilation -- a good sign for heart health.

In fact, earlier tests suggest the companys biomaterials are able to reverse or inhibit progression to heart failure, Schmuck says. They would be used in humans to repair damaged cardiac muscles, for patients who have experienced recent heart attacks or heart failure.

The company will be seeking approval from the FDA eventually, Schmuck said, adding that human trials could be undertaken in two or three years.

Bock says winning the contest would be spectacular, and that taking part is a lot of fun. The top 12, or Diligent Dozen, will be announced in late May, with the end of the contest coming in early June.

--By Alex Moe WisBusiness.com

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Cellular Logistics pursuing commercialization, top spot in Governor's Business Plan Contest - Wisbusiness.com

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The biggest national project to treat patients with cerebral palsy (CP) through injection of stems cells from umbilical cord blood (UCB) into the brain began its trial run in March. The project is jointly undertaken by Royan Institute, Childrens Medical Center (affiliated to Tehran University of Medical Sciences), and the Iran Blood Transfusion Organization (IBTO). In the first phase, it will provide treatment to 130 children with CP between the ages 5-13. The treatment was tested in September 2016 on children with CP in some hospitals, and the results were highly satisfactory. Approximately four in every 1,000 children in Iran have CP while in the developed countries the rate is 2 to 2.5 per 1000 live births. Cerebral palsy is an umbrella term for the effects of damage to a developing brain by various causes. It is connected with a range of symptoms, including muscle weakness and movement problems. The damage to the brain usually occurs early on in its development, either in the baby during pregnancy or during the period soon after birth. Symptoms may include difficulties in walking, balance and motor control, eating, swallowing, speech or coordination of eye movements. Some people affected by CP also have some level of intellectual disability. No two people with cerebral palsy are affected in exactly the same way. The IBTO plans to expand the storage of stems cells from umbilical cord blood to 100,000 samples from the current 80,000, said Ali Akbar Pourfathollah, head of the organization, ILNA reported. Around 75,000 samples have been stored in private banks and 5,000 in public banks, but the number will surpass 100,000 soon, he added.

Valuable Source for Treatment Umbilical blood is a valuable source of hematopoietic stem cells which can be used for treatment of many malignant diseases such as leukemia. Hundreds of transplants have been performed using stem cells from such blood, which is easy and risk free. The use of stem cells reduces the risk of viral diseases transmission and incidence of Graft Versus Host Disease (GVHD). The ability to perform organ transplants is among the benefits of umbilical cord blood transfusion. Using stems cells is also one of the best ways to treat blood diseases since the method has a success rate of 70% worldwide. Storage of stem cells is a valuable investment. So far, 27 cord blood banks have been launched across the country. There are two types: public and private banks for stem cell storage. The former does not charge a fee for storage. But in the latter, the cost of collection and genetic testing is about $645 and the annual charge for storage is $33, according to ISNA. Pourfathollah said the IBTO is looking to store stem cells in medical cases when a patient needs to receive treatment from matched unrelated donors. In the past Iranian year (ended March 20), out of the 8,000 stem cell transplantations in the country, only 100 were from matched unrelated donors and the rest came from sibling (or related) donors. IBTO is also looking to set up coagulation/transfusion and HLA/immunogenetics laboratories in the country on par with international standards. The Immunogenetics and HLA Laboratory provides human leukocyte antigen (HLA) typing, HLA antibody identification and post-transplant engraftment monitoring services. These tests are required for patients undergoing evaluation for organ transplantation, recipients of bone marrow/stem cell transplants, patients requiring platelet transfusions from HLA-matched donors, and patients undergoing evaluation of particular health conditions.

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Treating Cerebral Palsy With UCB Stem Cells - Financial Tribune

Bone Marrow Stem Cells Comments Off on Treating Cerebral Palsy With UCB Stem Cells – Financial Tribune | April 24th, 2017

It was Thursday afternoon, and the little girl from near Cologne, Germany, and the 40-year-old Duluth woman had known each other for less than 24 hours. But it was obvious that Edwards already had bonded with Ina and her little sister Mila.

They were together because the girls' mother had given Edwards a much greater gift: the gift of life.

"By your donation, I still get to be a mom," Edwards told Daniela Halfkann, 30. "(You're) a mom, so you completely understand how important it is to be here with your children."

Edwards, the mother of 15-year-old twin boys and the wife of Duluth Fire Chief Dennis Edwards, is alive because of the stem cell transplant she received at the Mayo Clinic on Oct. 31, 2014. As a result, she said, she is in remission from the rare and aggressive form of leukemia with which she had been diagnosed that June.

All she was told at the time of the transplant was that the donor was a woman from Germany.

Halfkann had registered as a potential stem cell or bone marrow donor at the large insurance company where she works in Cologne, she said. One day she received a call, saying her donation was needed.

After the six-hour procedure, Halfkann was told nothing more than that the recipient was a woman in the United States.

After a two-year waiting period required in Germany, the two women learned each other's identities last October and connected via Facebook.

Their meeting in Duluth was arranged by Amanda Schamper, Midwest donor recruitment coordinator for DKMS, the Germany-based organization that facilitated the donation.

Halfkann made the trip along with husband Stefan and their daughters, leaving their home at 3 a.m. on Tuesday and arriving at the Duluth International Airport at 5 p.m. on Wednesday.

Like Edwards, DKMS wants to raise awareness of the need for people to enter the registry, said Schamper, who also traveled to Duluth for the occasion.

She said 14,000 patients are in need of a peripheral blood stem cell or bone marrow donation, but fewer than half will get one because there's no match on the registry.

"We're looking for a particular protein in our DNA," she explained.

Only in 30 percent of cases are siblings a match. Edwards' brother and sister both had been screened, she said, and neither was a match for her.

Finding a match "is equated to finding your genetic twin, or winning the genetic lottery," Schamper said.

If more people were on the registry a process that only requires taking a swab from your cheek there would be more potential matches. But only 2 percent of eligible Americans are registered, Schamper said.

When the Halfkanns arrived at the gate on Wednesday, Dennis and Merissa Edwards, along with sons Caden and Jaxon, were waiting at the gate.

It was an emotional moment.

"It was hard for me," Merissa Edwards said on Friday, speaking to Daniela Halfkann. "I was crying. I was so emotional, so happy to meet you and hug you."

She wiped away a tear. "I still am."

"It was amazing," Halfkann responded. "I cried at the gate, too."

The Halfkanns, who are staying at the Edgewater, initially focused on recovery from jet lag. But Edwards is making sure they'll get a full taste of Duluth and Minnesota before beginning their return trip to Germany next Saturday. That includes visits to the Mall of America, the Great Lakes Aquarium and a trip up the North Shore.

A "thank-you party," open to the public, is planned on Sunday afternoon. Halfkann also will be recognized on Monday during the Saints Sports Awards ceremony at the College of St. Scholastica, where Edwards is an administrative assistant in the athletics department.

Recovery from the ravages of leukemia has been a long process, Edwards said, but she remains in remission. She gets a PET scan every six months to make sure that's still the case; the next one takes place next week.

Edwards shares her story, she said, not to call attention to herself but to highlight the need for people to take the simple step of registering as a potential donor.

"It's so important for us to help other people keep their families together and save a mother or father or son or daughter," she said. "The more people we can encourage to cheek-swab and get on the registry, the more lives we can help save and help families stay together."

TO LEARN MORE

For more information and to learn how to get on the bone marrow and peripheral blood stem cell registry, visit dkms.org.

IF YOU GO

The thank-you party for Daniela Halfkann will be from 2 to 5 p.m. on Sunday at The Other Place Bar and Grill, 3930 E. Calvary Road.

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By Marilynn Marchione The Associated Press

Tummy tucks really hurt. Doctors carve from hip to hip, slicing off skin, tightening muscles, tugging at innards.

Patients often need strong painkillers for days or even weeks, after the procedure but Mary Hernandez went home on just over-the-counter ibuprofen.

The reason could be the yellowish goo smeared on her 18-inch wound as she lay on the operating table. The Houston woman was helping test a novel medicine aimed at avoiding opioids, potent pain relievers fueling an epidemic of overuse and addiction.

Vicodin, OxyContin and similar drugs are widely used for bad backs, severe arthritis, damaged nerves and other ailments. They work powerfully in brain areas that control pleasure and pain, but the body adapts to them quickly, so people need higher and higher doses to get relief.

This growing dependence on opioids has mushroomed into a national health crisis, ripping apart communities and straining police and health departments.

Every day, an overdose of prescription opioids or heroin kills 91 people, and legions more are brought back from the brink of death. With some 2 million Americans hooked on these pills, evidence is growing that theyre not as good a choice for treating chronic pain as once thought.

Drug companies are working on alternatives, but have had little success.

Twenty or so years ago, they invested heavily and failed miserably, said Dr. Nora Volkow, director of the National Institute on Drug Abuse.

Pain is a pain to research. Some people bear more than others, and success cant be measured as objectively as it can be with medicines that shrink a tumor or clear an infection. Some new pain drugs that worked well were doomed by side effects Vioxx, for example, helped arthritis but hurt hearts.

Some fresh approaches are giving hope:

Bespoke drugs, as Volkow calls them. These target specific pathways and types of pain rather than acting broadly in the brain. One is Enbrel, which treats a key feature of rheumatoid arthritis and, in the process, eases pain.

Drugs to prevent the need for opioids. One that Hernandez was helping test numbs a wound for a few days and curbs inflammation. If people dont have big pain after surgery, their nerves dont go on high alert and theres less chance of developing chronic pain that might require opioids.

Funky new sources for medicines. In testing: Drugs from silk, hot chili peppers and the venom of snakes, snails and other critters.

Novel uses for existing drugs. Some seizure and depression medicines, for example, can help some types of pain.

The biggest need, however, is for completely new medicines that can be used by lots of people for lots of problems. These also pose the most risk for companies and patients alike.

Bumpy road

In the early 2000s, a small biotech company had a big idea: blocking nerve growth factor, a protein made in response to pain.

The companys drug, now called tanezumab, works on outlying nerves, helping to keep pain signals from muscles, skin and organs from reaching the spinal cord and brain good for treating arthritis and bad backs.

Pfizer Inc. bought the firm in 2006 and expanded testing. But in 2010, some people on tanezumab and similar drugs being tested by rivals needed joint replacements.

Besides dulling pain, nerve growth factor might affect joint repair and regeneration, so a possible safety issue needed full investigation in a medicine that would be the first of its type ever sold, said one independent expert, Dr. Jianguo Cheng, a Cleveland Clinic pain specialist and science chief for the American Academy of Pain Medicine.

Regulators put some of the studies on hold. Suddenly, some people who had been doing well on tanezumab lost access to it. Phyllis Leis in Waterfall, a small town in south-central Pennsylvania, was one.

I was so angry, she said. That was like a miracle drug. It really was. Unless you have arthritis in your knees and have trouble walking, youll never understand how much relief and what a godsend it was.

Her doctor, Alan Kivitz of the Altoona Center for Clinical Research, has helped run hundreds of pain studies and consults for Pfizer and many other companies.

You rarely get people to feel that good as many of them did on the nerve growth factor drugs, he said.

A drug with that much early promise is unusual, said Ken Verburg, who has led Pfizers pain research for several decades. When you do see one, you fight hard to try to bring one to the market, he said.

An independent review ultimately tied just a few serious joint problems to tanezumab and the suspension on testing was lifted in August 2012. But a new issue nervous system effects in some animal studies prompted a second hold later that year, and that wasnt lifted until 2015.

Now, Eli Lilly & Co. has joined Pfizer in testing tanezumab in late-stage studies with 7,000 patients. Results are expected late next year about 17 years after the drugs conception.

Avoiding pain

What if a drug could keep people from needing long-term pain relief in the first place? Heron Therapeutics Inc. is testing a novel, long-acting version of two drugs the anesthetic bupivacaine and the anti-inflammatory meloxicam for notoriously painful operations such as tummy tucks, bunion removal and hernia repair.

Company studies suggest it can numb wounds for about three days and cut patients need for opioids by 30 to 50 percent.

Theres a good chance of preventing brain responses that lead to chronic pain if patients can get through that initially very rough period, said Dr. Harold Minkowitz, a Houston anesthesiologist who consults for Heron and treated Hernandez in the tummy tuck study.

Hernandez was part of an experiment testing the drug vs. a placebo and doesnt know whether she got the drug or a dummy medicine. But she hurt less than she expected to and never filled a prescription for pain pills.

The goal would be to have half or more of patients not requiring an opiate after they go home, said Herons chief executive, Barry Quart. You have far fewer opiates going out into society, far fewer opiates sitting in medicine cabinets that make their way to a high school.

Studies so far are mid-stage too small to prove safety and effectiveness but Heron plans more aimed at winning approval.

On the horizon

Many companies have their eyes on sodium channel blockers, which affect how nerves talk to one another and thus might help various types of pain. Others are testing cell therapies for nerve pain. Stem cells can modulate immune responses and inflammation, and might overcome a raft of problems, said Cheng of the pain medicine academy.

Some companies, including Samumed, Centrexion Therapeutics and Flexion Therapeutics, are testing long-acting medicines to inject in knees to relieve arthritis pain. Samumeds aims to regenerate cartilage.

And then theres marijuana. A cannabis extract is sold as a mouth spray in Britain for nerve pain and other problems from multiple sclerosis. But cannabinoid research in the United States has been hampered by marijuanas legal status. A special license is needed and most researchers dont even try to obtain one, said Susan Ingram, a neurosurgery scientist at Oregon Health & Science University.

She is studying cannabinoid receptors in the brain, looking at how pain affects one type but not another. Such work might someday lead to drugs that relieve pain but dont produce a high or addiction.

Selective activity has precedent: The drug buprenorphine partially binds to opioid receptors in the brain and has become an extraordinarily successful medication for treating addiction, said Volkow, of the national drug institute.

It has shown pharmaceutical companies that if you come up with a good intervention, there is an opportunity to recover their costs, she said.

Tummy tucks really hurt. Doctors carve from hip to hip, slicing off skin, tightening muscles, tugging at innards. Patients often need strong painkillers for days or even weeks, after the procedure but Mary Hernandez went home on just over-the-counter ibuprofen. The reason could be the yellowish goo smeared on her 18-inch wound as she lay on the operating table. The Houston woman was helping test a novel medicine aimed at avoiding opioids, potent pain relievers fueling an epidemic of overuse and addiction. Vicodin, OxyContin and similar drugs are widely used for bad backs, severe arthritis, damaged nerves and other ailments. They work powerfully in brain areas that control pleasure and pain, but the body adapts to them quickly, so people need higher and higher doses to get relief. This growing dependence on opioids has mushroomed into a national health crisis, ripping apart communities and straining police and health departments. Every day, an overdose of prescription opioids or heroin kills 91 people, and legions more are brought back from the brink of death. With some 2 million Americans hooked on these pills, evidence is growing that theyre not as good a choice for treating chronic pain as once thought. Drug companies are working on alternatives, but have had little success. Twenty or so years ago, they invested heavily and failed miserably, said Dr. Nora Volkow, director of the National Institute on Drug Abuse. Pain is a pain to research. Some people bear more than others, and success cant be measured as objectively as it can be with medicines that shrink a tumor or clear an infection. Some new pain drugs that worked well were doomed by side effects Vioxx, for example, helped arthritis but hurt hearts. Some fresh approaches are giving hope: n Bespoke drugs, as Volkow calls them. These target specific pathways and types of pain rather than acting broadly in the brain. One is Enbrel, which treats a key feature of rheumatoid arthritis and, in the process, eases pain. n Drugs to prevent the need for opioids. One that Hernandez was helping test numbs a wound for a few days and curbs inflammation. If people dont have big pain after surgery, their nerves dont go on high alert and theres less chance of developing chronic pain that might require opioids. n Funky new sources for medicines. In testing: Drugs from silk, hot chili peppers and the venom of snakes, snails and other critters. n Novel uses for existing drugs. Some seizure and depression medicines, for example, can help some types of pain. The biggest need, however, is for completely new medicines that can be used by lots of people for lots of problems. These also pose the most risk for companies and patients alike.Subhead: Bumpy road In the early 2000s, a small biotech company had a big idea: blocking nerve growth factor, a protein made in response to pain. The companys drug, now called tanezumab, works on outlying nerves, helping to keep pain signals from muscles, skin and organs from reaching the spinal cord and brain good for treating arthritis and bad backs. Pfizer Inc. bought the firm in 2006 and expanded testing. But in 2010, some people on tanezumab and similar drugs being tested by rivals needed joint replacements. Besides dulling pain, nerve growth factor might affect joint repair and regeneration, so a possible safety issue needed full investigation in a medicine that would be the first of its type ever sold, said one independent expert, Dr. Jianguo Cheng, a Cleveland Clinic pain specialist and science chief for the American Academy of Pain Medicine. Regulators put some of the studies on hold. Suddenly, some people who had been doing well on tanezumab lost access to it. Phyllis Leis in Waterfall, a small town in south-central Pennsylvania, was one. I was so angry, she said. That was like a miracle drug. It really was. Unless you have arthritis in your knees and have trouble walking, youll never understand how much relief and what a godsend it was. Her doctor, Alan Kivitz of the Altoona Center for Clinical Research, has helped run hundreds of pain studies and consults for Pfizer and many other companies. You rarely get people to feel that good as many of them did on the nerve growth factor drugs, he said. A drug with that much early promise is unusual, said Ken Verburg, who has led Pfizers pain research for several decades. When you do see one, you fight hard to try to bring one to the market, he said. An independent review ultimately tied just a few serious joint problems to tanezumab and the suspension on testing was lifted in August 2012. But a new issue nervous system effects in some animal studies prompted a second hold later that year, and that wasnt lifted until 2015. Now, Eli Lilly & Co. has joined Pfizer in testing tanezumab in late-stage studies with 7,000 patients. Results are expected late next year about 17 years after the drugs conception.Subhead: Avoiding pain What if a drug could keep people from needing long-term pain relief in the first place? Heron Therapeutics Inc. is testing a novel, long-acting version of two drugs the anesthetic bupivacaine and the anti-inflammatory meloxicam for notoriously painful operations such as tummy tucks, bunion removal and hernia repair. Company studies suggest it can numb wounds for about three days and cut patients need for opioids by 30 to 50 percent. Theres a good chance of preventing brain responses that lead to chronic pain if patients can get through that initially very rough period, said Dr. Harold Minkowitz, a Houston anesthesiologist who consults for Heron and treated Hernandez in the tummy tuck study. Hernandez was part of an experiment testing the drug vs. a placebo and doesnt know whether she got the drug or a dummy medicine. But she hurt less than she expected to and never filled a prescription for pain pills. The goal would be to have half or more of patients not requiring an opiate after they go home, said Herons chief executive, Barry Quart. You have far fewer opiates going out into society, far fewer opiates sitting in medicine cabinets that make their way to a high school. Studies so far are mid-stage too small to prove safety and effectiveness but Heron plans more aimed at winning approval.Subhead: On the horizon Many companies have their eyes on sodium channel blockers, which affect how nerves talk to one another and thus might help various types of pain. Others are testing cell therapies for nerve pain. Stem cells can modulate immune responses and inflammation, and might overcome a raft of problems, said Cheng of the pain medicine academy. Some companies, including Samumed, Centrexion Therapeutics and Flexion Therapeutics, are testing long-acting medicines to inject in knees to relieve arthritis pain. Samumeds aims to regenerate cartilage. And then theres marijuana. A cannabis extract is sold as a mouth spray in Britain for nerve pain and other problems from multiple sclerosis. But cannabinoid research in the United States has been hampered by marijuanas legal status. A special license is needed and most researchers dont even try to obtain one, said Susan Ingram, a neurosurgery scientist at Oregon Health & Science University. She is studying cannabinoid receptors in the brain, looking at how pain affects one type but not another. Such work might someday lead to drugs that relieve pain but dont produce a high or addiction. Selective activity has precedent: The drug buprenorphine partially binds to opioid receptors in the brain and has become an extraordinarily successful medication for treating addiction, said Volkow, of the national drug institute. It has shown pharmaceutical companies that if you come up with a good intervention, there is an opportunity to recover their costs, she said.

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Researchers look for new ways to reduce suffering, end addiction ... - The Columbus Dispatch

Spinal Cord Stem Cells Comments Off on Researchers look for new ways to reduce suffering, end addiction … – The Columbus Dispatch | April 23rd, 2017

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A Lincoln student is set to save someone's life after finding out he was a match for someone who needed a bone marrow transplant.

John-Paul Dickie, the vice-president of academic affairs at the University of Lincoln, said he joined the register after his flatmate told him about it.

He doesn't know the identity of the person whose life he saved, but said he was delighted that he's been able to help someone who was desperate for a transplant.

He said: "My flatmate was involved with Lincoln Marrow, a student-led group trying to sign people up to the British Bone Marrow Register. He was telling me the benefits of it, including the fact it could potentially save someone's life.

READ MORE: Selfless mum marks 50th blood donation with daughter's first

"I signed up in February 2014, so it was a surprise when I heard back earlier this year that I was a potential match. I had some samples taken and eventually I had a date set for the operation in May.

"I'm looking forward to it, as it's an amazing way to help somebody.

"However I'm also a bit hesitant as it will require me to be strapped to a machine for four or five hours. My partner will be there to keep me company and I'll have books and TV to stop me from getting too bored."

When bone marrow is damaged it prevents a person from creating healthy blood cells and transplants like this help to treat the condition.

The transplant requires taking stem cells from the blood or bone marrow of one person and giving them to another.

John-Paul added: "There are two ways to take stem cells. One is taking them out of your back using a needle, which is painful but only 10 per cent of people have. Fortunately, I'm having the more common method in which blood is taken out of one arm, the stem cells are removed and then it is returned in the other arm."

READ MORE: 'Gordon was denied stem cell treatment, but I'll hold him in my heart forever'

Most people who need stem cells will be a match with a close family member. However, if this doesn't work then they will have to wait on the British Bone Marrow Registry.

"It's a great way to contribute and help save someone's life. All you have to do is give a sample of spit to get on the register, the process is so simple and easy. If you're able to do it, I would definitely encourage you to give it a try.

"The procedure is anonymous in case something goes wrong. You can find out their age and sex, but at the moment I don't know anything. After two years, you can apply to find out who they are."

Bone marrow donors need to be aged between 17 and 40 and already registered as a blood donor.

If you meet these criteria interested in signing up to the British Bone Marrow Register, visit their website for more information at: http://www.nhsbt.nhs.uk/bonemarrow/

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'It's amazing!' Student discovers he's a potential life-saving bone marrow match - Lincolnshire Echo

Bone Marrow Stem Cells Comments Off on ‘It’s amazing!’ Student discovers he’s a potential life-saving bone marrow match – Lincolnshire Echo | April 23rd, 2017

BROCKPORT When Chris Cella laced up his boxing gloves last Saturday night in preparation for his war within the squared circle, he was fighting for more than his undefeated fight record.

In front of the sellout crowd in Huntington, W.V., he made quick work of his opponent, working his body hard and knocking him out two minutes into the first round.

After his hand was raised cementing the victory the ring announcer handed Cella the microphone.

It was then that he shared his motivation for the win.

This fight tonight was for a special friend, a little girl named Shea. Shes the toughest warrior of all. This win is for you Shea, said Cella.

The week before the fight, Shea and her parents came out to Cellas gym, BC Boxing, where they wished him luck for his upcoming fight.

I first met Shea when she was five and her aunt brought her to the gym to train. When she came to wish me luck she stole my heart. Her beautiful smile and contagious laugh filled the gym with so much love. Shes been more of an inspiration to me that she will ever know. Shes a fighter and warrior in every sense of the word, and it felt great to get the win in her honor, Cella said.

Shea has been unable to make it to the gym for almost a year now.

Now 8 years old, Shea was diagnosed in August with AML leukemia, a type of cancer of the blood and bone marrow with excess immature white blood cells, weeks before she was to start second grade at DuBois Central Catholic School.

Since her diagnosis, Sheas journey toward being cancer-free hasnt been an easy one.

Shes had adverse reactions to her appendix and gallbladder from the chemotherapy treatments and both had to be removed.

She also had a stem cell transplant this January and struggled with with graft versus host disease of the stomach and skin, which occurs when donor bone marrow or stem cells attack the recipient.

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I know that seems like a lot of bad, but Shea had a positive outlook on this whole experience and is nothing but smiles, Sheas mother Misty McKinney said. We are hoping with in a month or two we can come home for good.

Shea cant wait to come home and have a huge pool party and get back to boxing.

Shea said she felt very special and happy, when Cella dedicated his fight to her. The boxing community at the gym has had the familys back since her diagnosis, even taking up collections of toys and gift cards at Christmastime in an attempt to give the girl a holiday she wouldnt forget.

Shea is one of the biggest inspirations Ive ever met, Cella said. She fights so hard every day, it makes my fight seem meaningless.

To keep up to date on Sheas journey and ways you can help, friend Pray for Shea on Facebook.

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Fight For Shea: Brockport man dedicates bout to DuBois girl with leukemia - The Courier-Express

Bone Marrow Stem Cells Comments Off on Fight For Shea: Brockport man dedicates bout to DuBois girl with leukemia – The Courier-Express | April 23rd, 2017