Next wave of innovation and discovery

Over the past 70 years, new surgical techniques and medical therapies, some of which were developed at the Stanford School of Medicine and Packard Childrens, have evolved and greatly improved outcomes for children with almost every type ofcongenital heart disease.

Heart defects that were once universally fatal can now be surgically improved. As patients born with heart disease survive longer, there are now more adults than children in the United States with congenital heart disease. However, further advancements are still needed to ensure a healthier future for patients, many of whom continue to face a compromised quality of life and require subsequent surgeries.

Surgical intervention can repair, but it rarely can truly cure, said pediatric heart surgeonFrank Hanley, MD, who is also the Lawrence Crowley, MD, Endowed Professor in Child Health at the School of Medicine and executive director of the Betty Irene Moore Childrens Heart Center. Children who have received complex surgical intervention to repair a cardiac abnormality require careful monitoring and specialized care throughout their life span. We imagine a day when a child born with a poorly working aortic valve, rather than undergoing multiple open-heart operations throughout his lifetime, instead receives a replacement valve engineered from his own stem cells. Dr. and Mrs. Moores gift comes at a critical juncture enabling us to advance beyond surgical repair to the discovery of transformational treatments and interventions and, ultimately, to true cures.

The center has an overall survival rate of 98 percent. Beyond survival alone, the goal is now to ensure an excellent overall outcome from normal brain function for even the most fragile patients, to the ability for children to perform well in school and to exercise and enjoy an active life into adulthood.

We are committed to providingbabies and children with heart disease and their families with the happiest, healthiest lives possible, from the early identification of problems, to expert intervention, and finally to a lifetime of care and support, saidStephen Roth, MD, MPH, chief of pediatriccardiologyand director of the Betty Irene Moore Childrens Heart Center.

Dr. and Mrs. Moores incredible gift will not only bolster our clinical capabilities for children and families receiving care now in the Betty Irene Moore Childrens Heart Center, it will also accelerate basic and translational research by Stanford Medicine faculty and scientists to develop more precise techniques to predict, prevent and cure, said Lloyd Minor, MD, dean of the School of Medicine. When it comes to achieving precision health, we must think as big as we can not just about treating disease, but about making and keeping people healthy and nowhere is this more true than in children.

In 2017, Packard Childrens will complete its major expansion, becoming the most technologically advanced, family-friendly and environmentally sustainable childrens hospital in the nation. The Moores gift will enable the Childrens Heart Center to expand its state-of-the-art clinical and research facilities, train the future leaders of cardiovascular medicine and surgery, and improve the field of pediatric cardiology and pediatric cardiovascular surgery through innovative research. In addition, the center will expand its clinical facilities, including a newly designed outpatient center.

Packard Childrens established the Childrens Heart Center in 2001 to focus more expertise and resources on congenital heart disease, the most common type of birth defect worldwide. Each year, approximately 40,000 children in the United States are born with heart defects, and an additional 25,000 children develop some kind of acquired heart disease.

The center has gained recognition as a national and international destination program for several highly specialized surgical procedures, and is also a full-service cardiology program that cares for patients with all forms of cardiovascular conditions. Under the leadership of Hanley and Roth, the center receives more than 25,000 patient visits annually and performs 80 to 90 percent of all cardiac surgical care for children in northern and central California.

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FINDINGS

UCLA researchers have developed a stem cell gene therapy cure for babies born with adenosine deaminase-deficient severe combined immunodeficiency, a rare and life-threatening condition that can be fatal within the first year of life if left untreated.

In a phase 2 clinical trial led by Dr. Donald Kohn of theEli and Edythe Broad Center of Regenerative Medicine and Stem Cell Researchat UCLA, all nine babies were cured. A 10th trial participant was a teenager at the time of treatment and showed no signs of immune system recovery. Kohns treatment method, a stem cell gene therapy that safely restores immune systems in babies with the immunodeficiency using the childs own cells, has cured 30 out of 30 babies during the course of several clinical trials.

Adenosine deaminase-deficient severe combined immunodeficiency, also known as ADA-SCID or bubble baby disease, is caused by a genetic mutation that results in the lack of the adenosine deaminase enzyme, which is an important component of the immune system. Without the enzyme, immune cells are not able to fight infections. Children with the disease must remain isolated in clean and germ-free environments to avoid exposure to viruses and bacteria; even a minor cold could prove fatal.

Currently, there are two commonly used treatment options for children with ADA-SCID. They can be injected twice a week with the adenosine deaminase enzyme a lifelong process that is very expensive and often does not return the immune system to optimal levels. Some children can receive a bone marrow transplant from a matched donor, such as a sibling, but bone marrow matches are rare and can result in the recipients body rejecting the transplanted cells.

The researchers used a strategy that corrects the ADA-SCID mutation by genetically modifying each patients own blood-forming stem cells, which can create all blood cell types. In the trial, blood stem cells removed from each childs bone marrow were corrected in the lab through insertion of the gene responsible for making the adenosine deaminase enzyme. Each child then received a transplant of their own corrected blood stem cells.

The clinical trial ran from 2009 to 2012 and treated 10 children with ADA-SCID and no available matched bone marrow donor. Three children were treated at the National Institutes of Health and seven were treated at UCLA. No children in the trial experienced complications from the treatment. Nine out of ten were babies and they all now have good immune system function and no longer need to be isolated. They are able to live normal lives, play outside, go to school, receive immunizations and, most importantly, heal from common sicknesses such as the cold or an ear infection. The teenager, who was not cured, continues to receive enzyme therapy.

The fact that the nine babies were cured and the teenager was not indicates that the gene therapy for ADA-SCID works best in the youngest patients, before their bodies lose the ability to restore the immune system.

The next step is to seek approval from the Food and Drug Administration for the gene therapy in the hopes that all children with ADA-SCID will be able to benefit from the treatment. Kohn and colleagues have also adapted the stem cell gene therapy approach to treat sickle cell disease and X-linked chronic granulomatous disease, an immunodeficiency disorder commonly referred to as X-linked CGD. Clinical trials providing stem cell gene therapy treatments for both diseases are currently ongoing.

Kohn is a professor of pediatrics and microbiology, immunology and molecular genetics at the David Geffen School of Medicine at UCLA and member of the UCLAChildrens Discovery and Innovation Institute at Mattel Childrens Hospital. The first author of the study is Kit Shaw, director of gene therapy clinical trials at UCLA.

The research was published in the Journal of Clinical Investigation.

The research was funded by grants from the U.S. Food and Drug Administrations Orphan Products Clinical Trials Grants Program (RO1 FD003005), the National Heart, Lung and Blood Institute(PO1 HL73104 and Z01 HG000122), the California Institute for Regenerative Medicine (CL1-00505-1.2 and FA1-00613-1), the UCLA Clinical and Translational Science Institute (UL1RR033176 and UL1TR000124) and the UCLA Broad Stem Cell Research Center.

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Investopedia

Cellect Succeeds In First Stem Cell Transplant (APOP) Investopedia It includes more than half the stem cell transplant procedures, including bone marrow transplant, resulting in a serious rejection disease called Graft-versus-Host-Disease (GvHD). GvHD is a medical disorder which results from receipt of transplanted ... Cellect Announces Successful First Cancer Patient Stem Cell TransplantP&T Community Why Cellect Biotechnology Ltd. (APOP) Stock Is Soaring TodayInvestorplace.com all 28 news articles »

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Stelios Andreadis, PhD, is leading a team of researchers who have discovered how to convert adult skin cells into stem cells without modifying their genetics.

UB researchers have found that adult skin cells can be converted into neural crest cells without any genetic modification.

The discovery, which was several years in the making, proves that these stem cells can yield other cells that are present in the spinal cord and brain.

The applications could be very significant, ranging from studying genetic diseases in a dish to generating possible regenerative cures from a patients own cells.

Its actually quite remarkable that it happens, says Stelios Andreadis, PhD, professor of biomedical engineering, who recently published a paper on the results, titled Reprogramming Postnatal Human Epidermal Keratinocytes Toward Functional Neural Crest Fates, in the journal Stem Cells.

The identity of the cells was further confirmed by lineage tracing experiments, where the reprogrammed cells were implanted in chicken embryos and acted just as neural crest cells do.

This image shows Keratinocyte-derived neural crest stem cells turning into neurons as shown by typical neuronal morphology.

Stem cells have been derived from adult cells before, but not without adding genes to alter the cells. The new process yields neural crest cells without addition of foreign genetic material. The reprogrammed neural crest cells can become smooth muscle cells, melanocytes, Schwann cells or neurons.

In medical applications this has tremendous potential because you can always get a skin biopsy, says Andreadis, who is also professor and chair of the Department of Chemical and Biological Engineering in the School of Engineering and Applied Sciences.

We can grow the cells to large numbers and reprogram them without genetic modification. So, autologous cells derived from the patient can be used to treat devastating neurogenic diseases that are currently hampered by the lack of easily accessible cell sources, he says.

The process can also be used to model disease. Skin cells from a person with a genetic disease of the nervous system can be reprogrammed into neural crest cells. These cells will have the disease-causing mutation in their chromosomes, but the genes that cause the mutation are not expressed in the skin.

The genes are likely to be expressed when cells differentiate into neural crest lineages, such as neurons or Schwann cells, thereby enabling researchers to study the disease in a dish. This is similar to induced pluripotent stem cells, but without genetic modification or reprogramming to the pluripotent state.

The discovery was a gradual process, taking almost five years, Andreadis says, as successive experiments kept leading to something new.

It was one step at a time. It was a very challenging task that involved a wide range of expertise and collaborators to bring it to fruition, he says.

Collaborators include:

Andreadis credits the persistence of his then-doctoral student, Vivek K. Bajpai, for sticking with it.

He is an excellent and persistent student, Andreadis says. Most students would have given up.

The research was supported by grants from the National Institutes of Health (NIH).

Andreadis also credits a seed grant from UBs Office of the Vice President for Research and Economic Developments IMPACT program that enabled part of the work.

The work recently received a $1.7 million NIH grant to delve into the mechanisms that occur as the cells reprogram, and to employ the cells for treating the Parkinsons-like symptoms in a mouse model of hypomyelinating disease.

This work has the potential to provide a novel source of abundant, easily accessible and autologous cells for treatment of devastating neurodegenerative diseases, Andreadis says. We are excited about this discovery and its potential impact and are grateful to NIH for the opportunity to pursue it further.

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Forbes

Four Ways to Younger Skin Right Now Forbes Her Hydrating and Plumping Serum No1 combats the environmental stressors that skin faces every day to detoxify and rejuvenate the face and subsequently enacting anti-aging properties. By using plant-stem cells, hyaluronic acid, marine snail peptides ...

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Malia

March is Multiple Sclerosis Month and Hadassah leads the world in the stunning success and implementation of the research at Hadassah Hospital in Jerusalem. Malia is just one of the many stories of triumph in Prof. Dimitrios Karussis program.

Malia was a top Dallas trial attorney-a senior partner in a law firm and the mother of three children, the youngest, age five. Her energy level was high and her life was full. And then, 18 years ago, she was diagnosed with multiple sclerosis (MS).

In the beginning, her symptoms were mild. For example, she was a little unsteady in her walking. She began taking a weekly injection of Avonex because, as her doctor told her, "You wouldn't want me to treat you for high blood pressure only after you had a heart attack."

Malia took Avonex for about 10 years. Still, her life became a journey of declining energy and mobility. Her biggest fear was that she would lose her vision and not be able to drive her children around. Fortunately, she didn't experience any problems with her vision, but her gait did get worse, as did the fatigue.

Ultimately, she could only walk a very short distance with a walker. Her balance became more unsteady. Her speech was harder to understand. And the fatigue got so bad that even taking a shower was so exhausting that she needed a nap afterward.

Malia tried four or five different medicines, but none seemed to help. As she explained, however, the promise of these medicines is only to slow the progression of the disease, not to improve upon one's symptoms.

At one point on this odyssey of decline, Malia fell and broke her leg and found herself in a wheelchair for six weeks. She began to research other treatment options, surfing the Internet extensively. It was how she came upon the innovative stem-cell work of Prof. Dimitrios Karussis, senior neurologist at Hadassah Hospital Ein Kerem and head of its Multiple Sclerosis Center. She learned that Prof. Karussis was conducting a unique MS clinical trial involving the injection of a patient's own bone marrow-derived stem cells directly into the spinal cord.

When she met Prof. Karussis for the first time, she was walking with a walker, but couldn't walk any real distance. It was December 2014. In the spring of 2015, Malia, who was accepted into one of Prof. Karussis' clinical trials, traveled back to Hadassah to have a bone marrow extraction, which would be the basis for her stem-cell transplant. As she explains, just a syringe of cells is extracted, but then these cells are enhanced in culture for six months. In the fall of 2016, Malia received her first infusion. The actual infusion, itself, Malia relates, took only five minutes!

"I got the infusion at 2 p.m.," Malia recalled, "and eight hours later, I did something I had not done in two to three years." She explained that before the infusion, whenever she got out of bed, to get back in, she would have to lift her right leg with her hands. This time, post infusion, after she got up to go to the bathroom, she was able to get back into bed by just lifting her right leg onto the bedwithout holding it! She called the kids; her husband, David, made a video of her new ability. When her doctors came in that next morning to see how she was doing, she showed them her new skill. "Everyone was sobbing," she recalled; "we were all so excited by how amazing it was."

Malia said: "The most important thing of all was that the level of my fatigue was so improved."

Taking herself back to the night she returned home from that first infusion, Malia recalled: "We arrived back home at midnight. The next morning at 8, I was up writing my blog. Then I unpacked, did laundry, and made a five-course dinner for my husband, after which he went to bed, exhausted from the jet lag. I, however, stayed up to crochet a new blanket until midnight."

That renewed energy has never disappeared since Malia started getting stem-cell infusions at Hadassah. She received two more in 2016 and the most recent in February 2017. The kind of improvements varied with each infusion-sometimes the improvement in her gait was most noticeable; other times, it was the clarity of her speech. With the 2017 infusion, it was her balance. A year or so ago, she could only balance herself on a "whole body vibration machine" for about a minute and a half. Then she worked this up to five minutes. After this fourth infusion, she could stay on the machine for 12 minutes and even let go of the bar with one hand.

In addition, before every infusion, a baseline measurement of her abilities is taken. The most recent baseline, Malia reported, was higher than ever before. By the same token, her overall physical improvements are the best they've been.

Malia's recovery is not without its setbacks. As the stem-cell treatment wears off, she does experience some backsliding in her improvements-perhaps in her speech or the way she walks. Nevertheless, after she has a treatment, the improvements return or surpass the previous ones.

"I had forgotten what it was like to feel like a regular person, like someone with a normal level of energy; I had forgotten what it was like to feel good," Malia said.

When she is out and about with her walker, she sees people looking at her with sympathy. But she doesn't feel sorry for herself, she notes, because she has gotten her life back. Malia feels blessed that she has come this far. "Until you've been there, you can't really understand just how important it is to feel like a regular person."

Malia had stopped practicing law when she was diagnosed with MS. But just recently she reactivated her law license. She took on a case, with a colleague. And in the beginning of April, she will be going to trial.

"When you feel like you can do things you used to do, life is as it should be," Malia said.

Malia no longer takes any medication for MS and being an MS patient is no longer a central focus of her life. But, she is now a stem-cell activist and fundraiser on behalf of Hadassah because "I feel I have been given a gift and I want to ensure that more people with MS can be helped."

While she doesn't know the other participants in the clinical trial, she did meet one man from London last time she was at the hospital for her infusion. As she relates, this man was "giddy with excitement, lifting his cane in the air with one hand." They began to talk and he explained that he just had his first stem-cell infusion and, overnight, he was able to lift his cane up in the air with his right hand when for 15 years he had been unable to even hold anything in that hand.

"What I would like Hadassah supporters around the world to understand," Malia related, "is that yes, I get the wonderful benefits of Prof. Karussis' stem-cell treatments (and, of course, she does not minimize his trailblazing hard work and miraculous success), "but I also was surprised to learn that I have this large Hadassah cheering squad that gives me hope and supports me-making me believe in possibilities for improvement. As Malia recalled, when she sits with a visitor from Hadassah and sees the look of awe on the face of this Hadassah supporter because of the leap in her improvement, it gives her renewed strength. "There are many things I want to do with the rest of my life," Malia said, "and raising money for Hadassah is at the top of the list."

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Lacey Miller will probably never forget her sons first birthday, at least not for the usual reasons. She threw him a party at a park near their home in Culver. Throughout the whole thing, she couldnt walk and could barely see, merely moving was a challenge.

I just kind of sat there and everyone cleaned up and they got me back to the house, she said.

Miller has a form of multiple sclerosis, a disease that disrupts communication between the brain and the rest of the body. The symptoms numbness, difficulty concentrating, blurred vision and fatigue come in cycles and tend to worsen over time.

When she was diagnosed in 2012 at age 27, Millers symptoms werent so bad: little things like numbness in her legs and blurry vision. It wasnt until three years later, when she became pregnant with her son, that things went downhill fast. She had to be induced into labor early because she had so much trouble walking. After that, there was vertigo, vision loss and walking became more difficult. Her worsening symptoms culminated in an emergency room trip following her sons first birthday party.

I was going to be in a wheelchair in probably a year, she said.

Internet exploration brought her to a study at Northwestern University in Chicago that is further exploring the idea of, in a sense, resetting the immune systems of multiple sclerosis patients. Multiple sclerosis develops when a persons immune system attacks the protective covering of nerve fibers in the central nervous system, making it difficult for the brain to communicate with the rest of the body.

Miller applied to join the study in June 2016 and was approved in September. She started her testing and treatment in early January, embarking on what would be months of grueling treatments and travel.

Through it all, Miller thought about her now-18-month-old son, Emmerick.

Even on the days when it was like, This sucks. This is really hard, it was like, Im doing this for him so he can have a mom who can run down to the park with him and do things with him, she said.

Thats a win

A handful of prominent studies in recent years have raised hope that a procedure called hematopoietic stem cell transplantation can prevent further disability among patients with multiple sclerosis.

A study on 25 multiple sclerosis patients published last month gave further hope. Three years after undergoing stem cell transplants, disability symptoms in nearly 80 percent of the patients had not worsened, according to the study, published in the in the Journal of the American Medical Association Neurology. At five years, nearly 70 percent of the patients symptoms still had not worsened.

Dr. Linda Griffith, an author on the study and a medical officer at the National Institute of Allergy and Infectious Diseases, which sponsored the study, said being able to halt existing symptoms is a big advance.

To us, thats a win if they dont get worse, she said. We have no notion of being able to cure MS here and make it go away. Its not going to go away. Its a bad disease. But medical investigators are really thrilled and excited when they can find that the disease isnt getting worse.

Dr. Richard Burt, a professor of medicine and chief of immunotherapy at Northwesterns Feinberg School of Medicine, found similar results in a 2015 study he and his team published in the Journal of the American Medical Association. Five years after undergoing stem cell transplants, only 10 percent of multiple sclerosis patients had worsened symptoms.

The majority got better and stayed better, he said. Thats a paradigm change.

Creating hope

The stem cell transplant procedure involves extracting a patients bone marrow stem cells using a long needle and using chemotherapy and other toxic medications to clear out the rest of the cells. In multiple sclerosis patients, the patients own stem cells are then put back into the body.

It is now immature, just like if you were a child again and your immune system was learning all over again whats its supposed to be reacting against versus not, Griffith said.

While it sounds exciting, Griffith cautions the research is far from conclusive. She hearkens back to the hype around early studies that showed the procedure could be helpful for breast cancer patients. It ultimately wasnt.

What needs to happen next, she said, is a randomized study that compares groups of multiple sclerosis patients who either receive the transplant or the medications typically used to treat the condition, following them over time.

Not for everyone

Burt, the first doctor to perform these stem cell transplants for multiple sclerosis patients in the U.S., performed the procedure on Miller.

He cautions that only a specific subset of multiple sclerosis patients will benefit from the procedure. They must have a common form of the condition called relapse-remitting, meaning symptoms come in waves that recur over time. He doesnt perform the procedure on people who have progressed to a later stage, as it would be too late to help them, he said. Imaging on their brain and spine must show new lesions, indicating the disease is active.

Its frustrating because people learn about this on Facebook and want to get it, she said. Theyre upset when we decline them.

Burt is confident, however, that he knows who his procedure can help and who it cant help.

We developed this over many decades, he said.

When Miller, who is now 32, went in for testing with Burt, it was in the middle of an attack. She could barely walk into the office at Northwestern; her dad had to hold her arm for balance.

Although she was accepted into the study, getting her health insurance carrier on board proved to be a struggle and logistical issues delayed her ability to officially participate. Burt is still treating her under the study protocols, however, on whats referred to as a compassionate basis.

Millers first trip to Chicago was a short one for pre-study testing: imaging to check the progression of her multiple sclerosis, among other evaluations. She flew back to Chicago on Jan. 21 for what would be a two-week trip. First, they gave her chemotherapy to kill her stem cells and suppress her immune system. That meant she lost her hair.

Then they put a long needle into her neck to remove the remaining stem cells. She still has a tiny, round scar where the needle went in.

On Feb. 12, she flew back to Chicago again to have her stem cells put back into her body. She was discharged on March 1.

Sometimes, Burt said his patients notice right away the so-called MS fog, the fatigue and inability to think clearly, is lifted once they finish the procedure.

Miller said that was the case for her. She noticed other things right away, too. Walking around downtown Chicago was much easier after the surgery than before. She no longer had to use a cane or wall to guide her and she no longer had to stop and rest.

The other day, her fiance, Chris, asked her what the score of the basketball game was. She told him.

He was like, Can you see that? she said. I was like, Yeah! I didnt realize things are just so crisp and clear.

The near-constant numbness in her legs also isnt as bad as it used to be.

We were kind of joking the other day because my fiance touched my foot, she said. I was like Oh my god, I can feel that! I hadnt been able to feel my feet.

No more drugs

Burt theorizes that in addition to helping peoples quality of life, stem cell transplants could also help them financially, a point he hopes to flesh out in his current research.

A 2015 study in the journal Neurology found multiple sclerosis drugs cost patients roughly $60,000 per year. The price of the drugs increased annually between 1993 and 2013 at rates of five to seven times higher than the rate of prescription drug inflation, according to the study.

Miller, who works as a juvenile parole officer in Jefferson County, estimates she was spending about $500 a month on the drugs after her insurance paid its portion. She had to quit one medication a shot she gave herself three times per week because it gave her flu-like symptoms. Not having to take medications anymore was one of the main reasons Miller said she wanted to enroll in Burts study.

Im kind of one of those people that I dont even like taking Tylenol, she said. Id rather just not take anything.

Miller isnt currently on any multiple sclerosis medications. She said she hasnt felt this good in years. She returned to work part time this week.

Burt routinely travels to medical centers around the world explaining the procedure in hopes other neurologists will perform the stem cell transplants on multiple sclerosis patients. The older ones tend to be more set in their ways, but he believes the technique will catch on among younger doctors.

The procedure is currently being performed in England, Sweden and Brazil, Burt said. A hospital in India recently expressed interest, too.

At the end of the day, we want to help people throughout the world, Burt said, and in fact this is spreading throughout the world.

Reporter: 541-383-0304,

tbannow@bendbulletin.com

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A small stem cell trial in which patients with severe spinal injuriesappeared to make remarkable progress is still showing excellent results, according to the company conductingthe research.

One of the patients in the trial is 21-year-old Kris Boesen, from Bakersfield, California, whose story we reported on last year.A car crash had left theBakersfield, California native with three crushed vertebrae, almost no feeling below his neck, and a grimprognosis. Doctors believed he would live the rest of his life as a paraplegic.

Enter stem cell therapy. Most treatments for serious spinal injuries concentrate on physical therapy to expand the range of the patients remainingmotor skills and to limit further injury, not to reverse the actual damage. But last April, as part of an experimental phase 2 clinical trial called SCiStar, researchers injected Boesen with 10 million stem cells. By July, hehad recovered use of his hands to the point where he could use a wheelchair, a computer and a cellphone, and could take care of most of his daily living needs.In recent months his progress has continued, says his father.

Boesen is not the only patient to have improved in the trial, according toAsterias Biotherapeutics, which is conducting the research. Boesen is part of a cohort of six patients who were experiencing various levels of paralysis and were injected with the 10 million stem cell dose. In a Jan. 24update, the company saidfive of those patientshad improved either one or twolevels on a widely used scale to measuremotor function in spinal injury patients.

On Tuesday, Asterias issued a newupdate, announcingthat the sixth patient in the cohort has experienced a similar improvement.

While spontaneous recovery for spinal injury patients does occur,the likelihood of all six patients recovering to the degree they haveis less likely, researchers say.

This is as good as you could hope at this point, said Charles Liu, Boesens neurosurgeon and director of the USC Neurorestoration Center. So far all the evidence is pointing in the right direction.

To measure improvement in spinal injury patients, researchers use two yardsticks: the Upper Extremity Motor Scale, or UEMS, and the International Standards for Neurological Classification of Spinal Cord Injury, or ISNCSCI. On the UEMS scale,patients are scored from 0 to 5 on theirability to use five key muscles in the wrists, elbows and fingers. The ISNCSCI scale assesses where damage has occurred along the different levels of the cervical vertebrae, which generally determines the scope of impairment to the body and the level of care needed.

For instance, if a patient has sustained damage at the fourth cervical vertebra down, known as C-4, at the base of the neck, it generally means that person is paralyzed from the neck down, requiring round-the-clock care and a ventilator to breathe.A patient with a C-5 injury may not be able to move her arms or hands, requiring about 6 to 12 hours per day of assisted care; and at the C-6 level, better motor function mayallow a patient to take care of most of herdaily living needs on her own.

Which is all to say that even one level of recovery could substantially improve the daily life ofa spinal injury patient.

According to Asterias, all six patients in the 10million-cell cohort have improved their general UEMS scores, and jumped at least one motor level on the ISNCSCI scale on one or both sides of their body.

Two patients have improvedtwo motor levels on one side; and one patient,Boesen, has improved two motor levels on both sides.

Steve Cartt, president and CEO of Asterias, said anotherpatient, Jake Javier of Danville, California, has gonefrom partial paralysis to being able to use his hands well enough to considerpursuing a computer science career.

Throws Like a Regular Throw

In September, Boesens father, Rod Boesen, told us how excited he wasthat his son had regained some feeling in one of his feet. Last week, at11 months post-injection, the elder Boesensaid Kris has continued to improve.

Now he can move his toe and his knee together at the same time, Boesen said. Theyre about to give him a manual wheelchair now [instead of a motorized one]. He can grip with his hands enough to use a manual one.

Boesen said the movement in his sons arms and hands has greatlyimproved since September.Kris, a formerhigh school pitcher, had beenflinging a ball to his dog like people throw hand grenades, Boesen said. They kind of cradle them and thats how Kris would do it. But now he throws like a regular throw, tosses that ball down the hall, has that release point down, and just wings it.

Asterias is currently recruiting patients for a trial in which theyll receive 20 million stem cells, the optimal dose, according to company researchers. Two patients have already started the 20 million stem cell therapy, and six-month results from those patients will be released in the fall, Cartt said.

Patients who received 2 million stem cells in an earlier phase of the study have not shown much change in their condition, according to the Jan. 24 update.

Guarded Optimism

While Boesens father is impressed with the results, the optimism of researchers inside and outside the studyhas been guarded.The trial is still in its early stages, and the sample size is small, said Paul Knoepfler, a cell biology professor and stem cell researcher at UC Davis, who is not involved in the SCiStar study.

As a scientist, I still would want to wait for more data, Knoepfler said. Its certainly interesting, but its still early. Its a phase 2 trial.

To address the issue of small sample size, Asterias islooking at historical data to determinethe level of improvement for patients in similar circumstances who did not receive stem cell therapy. The company has said it found a meaningful difference in the recovery of its study patients compared to the norm.

Liu said one of the most importantresults is the lack of significant side effects or other negative outcomes resulting from the treatment to date.

Thats very significant to me, Liu said. Thats the first thing you look for, is anyone hurt from this therapy.

There was also a concern, he said, that some patients might regress over time, once the initial injection of stem cells wore off. Thathasyet to occur.

No one has lost anything theyve gained, Liu said. We were very happy to see that. This is all very promising.

The next step for the SCiStartrial will be to establish a control group, Cartt said.

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A two-step method to make microglia Nature.com Microglia have been reported in some disease models to have beneficial effects; however, research into their potential as a cell therapy is limited by the lack of means to produce readily grafted, autologous microglial cells. Now, in Nature ...

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For a soldier who suffered a spinal cord injury on the battlefield, the promise of regenerative medicine is to fully repair the resulting limb paralysis. But that hope is still years from reality.

Not only powerful, but efficient. Studying diseases in lab-created tissue may help reduce the price tag now roughly $1.8 billion for bringing a new drug to market, which is one of the reasons Ashton received a National Science Foundation CAREER Award for advancing tissue engineering of the human spinal cord. During the projects five-year funding period, his lab in the Wisconsin Institute for Discovery will fine-tune the technology for growing a neural tube, the developmental predecessor of the spinal cord, from scratch.

As the neural tube matures and diversifies during the development of a human embryo, it gives rise to the two core parts of our central nervous system: the brain and spinal cord. By replicating this process in a dish, Ashton hopes to develop a platform for research that is highly reproducible and can be broadly disseminated. Biologists could simply add their cells to Ashtons starter tissue to build a model of whichever spinal cord disease they desire.

By starting with cells from an individual patient, researchers will be able to target disease therapies to a particular genetic background a concept known as personalized medicine. Drug tests in engineered spinal cords may become an intermediate step between animal models and clinical trials of patients affected by Lou Gehrigs disease, multiple sclerosis or spinal cord injuries, helping to bridge the differences between a human and rodent spinal cord.

Weve cured spinal cord diseases in a lot of rodents over the years, but only a small percentage of those drugs work in humans, Ashton says. If we can make the engineered tissue as close as possible to whats in our body, this will eventually translate to better drugs.

The recipe Ashton and fellow UWMadison engineering professors Lih-Sheng (Tom) Turng and David Beebe plan to follow to coax stem cells into forming a neural tube goes something like this: First, they use water-soluble Lego-type molds to create microscale cavities within a jelly-like substance. Then they add human neural stem cells into these cavities, and let the cells coalesce as they do naturally to form neural tube-resembling tissue.

Next, they add signaling molecules whose variable concentration instructs the stem cells to turn into different types of neurons and neuron-supporting cells. Last comes the greatest challenge: getting these specialized cells to connect to one another and form electric circuits that give the spinal cord its function.

The complexity of the central nervous system exists because specific circuits have to form over very long distances. If any part of a circuit goes awry, you lose function, Ashton explains. The biggest open question is whether the tissue we create in vitro will have the proper wiring of different cell types to yield circuits similar to those in our body.

Ashtons CAREER Award will also fund educational activities that range from expanding outreach programs for underrepresented minority K-12 students, to creating a website and exhibit for the public, to educating Madison-area high school teachers about tissue engineering.

Ashton, who is African-American, comes from a socially active family background his grandfather was a minister and president of the Virginia chapter of the NAACP. He has assisted the nonprofit group 100 Black Men of Madison with its K-12 mentoring programs for years.

He plans to use the new grant to develop virtual interactive lab experiments and matching teaching modules.

I hope to attract more students to the fast-growing field of regenerative medicine, Ashton says, and to motivate people to continue to fund this kind of research so that we can develop therapies to cure diseases, instead of just treating their symptoms.

This article has been republished frommaterialsprovided by University of Wisconsin-Madison. Note: material may have been edited for length and content. For further information, please contact the cited source.

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Growing a Neural Tube in a Dish | Technology Networks - Technology Networks

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Twice a year, I strip down to my underwear, don a paper gown and subject myself to a full-body examination at the dermatologists office. These are done twice as often as most other patientsand for good reason. Not only am I freckly and fair-skinned, Ive had an unhealthy relationship with the sun, which makes me more susceptible to skin cancer.

During my teens and 20s, when I was a lifeguard and camp counselor, I spent the majority of my summers outdoors. Like my peers, Id wanted to achieve the perfect tan. Id worn sunscreen, but it was SPF 4barely any protection, compared with what doctors recommend today.

Now, Im paying the price. This past decade, Ive had a handful of suspicious-looking moles removed. Recently, my dermatologist sent me to a medical photographer for a full-body photo session to document my moles, in case they change.

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My situation isnt unique. Countless people worldwide didnt protect themselves adequately from the suns ultraviolet rays during their youth. Decades ago, doctors didnt preach about sun protection, and researchers didnt realize that the suns ultraviolet rays could cause skin changes that can lead to melanoma, the deadliest form of skin cancer.

The most important reason for the increase in melanomas is thought to be due to increased exposure to ultraviolet radiation from sun and artificial tanning sources, says John J. DiGiovanna, staff clinician in the dermatology branch of the National Cancer Institutes Center for Cancer Research in Bethesda, Maryland.

Melanoma is only the ninth most commonly diagnosed cancer across Europe, but its rates have been rising sharply since the 1980s, six-fold among some groups.

Every year, 100,000 new cases of melanoma are diagnosed in Europe, says John Haanen, head of medical oncology at the Netherlands Cancer Institute in Amsterdam. Caucasians are at greatest risk, especially those with fair skin, red hair and freckles. Risk rises after age 40especially sun worshippers. Many experts refer to the increased prevalence as an epidemic.

I would not call it a melanoma epidemic but a skin cancer epidemic, says Reinhard Dummer, director of the Skin Cancer Centre at University Hospital Zrich. We expect in Switzerland that one out of five persons will develop skin cancers once in their lives.

Cultural changes over several decades are likely to blame. Bathing suits have gotten skimpier, and seaside vacations have become more common, exposing pale office workers to intense sunlight for short periods.

In Europe, low-cost air travel has increased the ability for people to travel to sunny, warmer climates for a week here and there, says Alex Menzies, medical oncologist at Melanoma Institute Australia, the country with the highest melanoma rates in the world. Intermittent exposure to the sun with burning is a major risk for melanoma.

Even if youve endured decades worth of sun exposure, there is hope.

The earlier you notice melanoma, the greater your chances are of being cured. Surgery is the primary treatment. If you picked up an early-changing mole, you could have a virtually normal life expectancy, says Girish Patel, lead investigator for the Skin Cancer Stem Cell Research Program at Cardiff University.

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Regular skin checks and meaningful lifestyle changes to limit further damage from the sun help improve the odds. Since Imogen Cheese, 37, of Gloucestershire, England, was diagnosed with stage II melanoma in 2013, shes screened by her medical team every three months. I cover up to avoid the midday sun, says Imogen. I wear high factor SPF, I am active and eat a healthy balanced diet. So far, her cancer has not progressed.

Researchers have made great strides in the treatment of advanced melanoma. One option: Targeted therapy, which can be given to stage IV patients with specific genetic mutations.

Melanoma researchers in Australia have been involved with targeted therapy research since the beginning, about seven years ago. We do testing on their tumors to look if there are any mutations in certain genes in the tumor, says Menzies. We have targeted therapy that can attack the BRAF mutation, which is found in about 50 percent of tumors from patients. If we give tablets for BRAF-mutant melanoma, almost every patient will have shrinkage of the tumor. On average, it will keep things under control for one year, and the one-year survival rate has improved to 70 percent, from 30 percent five years ago.

Five years after John Ambrose, 67, of New South Wales, Australia, had a grade IV skin melanoma removed he began coughing up blood. His disease had spread to both lungs and his prognosis was poor. He joined a targeted therapy clinical trial in 2013, and within three months, his tumors shrank by 70 percent. After 18 months, he had clear scans. Today, John travels, plays golf and spends time with his grandchildren.

My situation has not stopped me living a normal life, he says.

Texas native Jesse Thomas, 57, also benefited from targeted therapy after being diagnosed with stage IV melanoma in 2013, with tumors on his neck, liver and spine. Genomic testing revealed Jesse had an uncommon V600K BRAF mutation, and his oncologist was able to pinpoint a targeted therapy for him.

They expected the cancer to stop growing, but it actually shrank, Jesse says. Theres no way to cure it, but I am controllable.

Targeted therapy is only for stage IV patients, but researchers are studying its effects on stage III patients. We should know within a couple of years whether these treatments are beneficial, says John Haanen.

Researchers have been able to stimulate the T-cells in some melanoma patients immune systems to fight cancer, with astounding results.

T-cells kill off viruses and other things, Menzies says, but with cancer, theyre sitting there around the tumor, asleep. They know that the tumor is foreign, but the tumor has turned them off, stopping them from killing it. Immunology drugs turn on the T-cells and they kill the tumor.

Melanoma researchers consider immunology the biggest breakthrough in decades.

This is our penicillin moment in oncology, Menzies says. Melanoma can be turned into a chronic disease, and many people will not die from it in the near future if we continue to go the way were going.

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Immunotherapy doesnt work for everyone, but it can be quite effective. Cardiff Universitys Patel says, In the 45 or so percent of people who respond, they can respond for very long periods of time.

In 2013, Cardiff resident Vicky Brown, 62, was shocked to learn that a lump in her breast was actually melanoma, not breast cancer. Shed had early-stage melanoma in 2006, which returned in her breast and lungs.

Through a clinical trial, Brown received intravenous doses of two immunotherapy drugs. Within weeks, her tumors shrank. She discontinued the drugs due to side effects, but it kept the melanoma in check for a year. In 2015, after new lung tumors appeared, she received more immunology treatments. The drugs again shrank her tumors.

I am hoping this couple of doses will give me more time again, Vicky says. My grandson is now nine months old. I want to be able to make memories for him, as well as my four-year-old granddaughter.

Researchers are working to get more patients to have a positive response to the treatment. The notion is that clearly, if we can do it in a few, we should be able to do it in the majority, says Patel.

For years, researchers tried creating a melanoma vaccine, to no avail. Now, researchers are combining the success of immunotherapy with the concept of vaccines, leading to personalized melanoma treatments.

As we better understand how the immune system recognizes the melanoma cells, we are developing so-called personalized vaccines, Haanen says. We are starting now in metastatic patients and if this concept works well move to earlier stages.

Hein Jambroers, 50, of Roermond, Netherlands, has benefited from a personalized treatment called adoptive cell therapy (ACT). He was diagnosed with stage II melanoma in 2009, but a year later, he had stage IV disease, with tumors on his right leg and liver, and was told that he had less than six months to live.

After getting some short-term benefit from targeted therapy, Hein was referred to an ACT clinical trial in 2011. Doctors at the Netherlands Cancer Institute harvested some of his white blood cells, then monitored them in a laboratory to identify the healthiest T-cells to fight melanoma. They were replicated in large numbers. Hein received chemotherapy to kill his existing T-cells, then got an infusion of the laboratory-created T-cells, which basically gave him a new immune system that shrank his tumors within three months.

Hes what doctors call a complete responder. Hes had clean scans ever since; no trace of melanoma.

Complete responders have an excellent prognosis, says Haanen, who treated Hein. Cure is always difficult to say, but very long-term remissions which could be cureare seen in the majority of complete responders and in some partial responders.

Hein, who expected to die, is cautiously optimistic. Im very positive about my future, but Im always on a state of alert, he says. I sit in the shade. I cream up with sunscreen. I even do it for my child and my wife. I dont want to tempt the fates.

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Soon, doctors may defeat cancer by attacking stem cells.

Skin stem cells make thousands of healthy skin cells. Melanoma stem cells work similarly, except they make thousands of malignant melanoma cells. Researchers are targeting melanoma stem cells to stop tumors from spreading.

Its like killing off the queen bee, Patel says. The whole hive then dies away, because youve gotten to the cell thats giving rise to everything.

This is vastly different from chemotherapy, which aims to kill as much cancer as possible. Stem cells make up only one to three per cent of some skin cancers.

If you got rid of the cancer stem cell population, the whole tumor could not proliferate, Patel says. If you take the bulk of a tumor and regrow it in a mouse without stem cells, it cant form. But if you take a small part of the cancer stem cell population, it grows back fully.

Researchers have begun clinical trials, and treatments could be available in a decade.

Despite sun damage that I endured during my youth, Im optimistic that Im doing everything that I can to stay ahead of any problems that may crop up. Ive got photos of all of my moles and freckles now, which I use for monthly self-exams. Ill bring them to my dermatologist for my next full-body examination. Ive also been raising my children with 21st century values for sun exposureplenty of high-SPF sunscreen, hats and time in the shadeso hopefully the next generation wont have the melanoma worries that my generation does.

If youve been diagnosed with advanced melanoma, heres what patient advocates recommend:

See a specialist

Seek a facility where doctors specialize in melanoma. Our recommendation for patients is to get into a melanoma center of excellence, says Bettina Ryll, founder of Melanoma Patient Network Europe in Uppsala, Sweden. The new immunotherapies have very different side effects from anything weve ever had before, so you dont want to have a physician who has never seen this.

Consider a clinical trial

Availability of immunotherapy and targeted therapy varies in Europe. Cost is a factor in many countries. Many patients enter clinical trials to receive these drugs. A promising clinical trial may be farther from home than youd prefer, but the extra drive could be worth it. Rory Bernard, 47, of Clermont-Ferrand, France, travels four hours to Paris for targeted therapy treatments, which have shrunk his tumors and extended his life. The dermatologist said, If you stay here, youre dead in six months, says Rorys wife, Gilly Spurrier-Bernard, founder of Melanoma France. My aim is to inform patients that if they want to get the best treatment, they may need to move around. Translation translation transl translation translation transl translation translation.

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Sun Exposure Is No Joke. You Need to Get Your Skin Checked ASAP - Reader's Digest

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Science Daily

First patient cured of rare blood disorder Science Daily The transplant technique is unique, because it allows a donor's cells to gradually take over a patient's bone marrow without using toxic agents to eliminate a patient's cells prior to the transplant. ... treatment options have been limited because they ... Stem Cell Transplant Cures a Man With Rare Blood DisorderNature World News Doctors cure first patient with rare blood disorderIANS all 3 news articles »

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First patient cured of rare blood disorder - Science Daily

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Electroacupuncture releases stem cells to relieve pain, promote tissue repair, study finds Science Daily "The acupuncture stimulus we're giving these animals has a rapid effect on neuroanatomical pathways that connect the stimulus point in the arm to responsive neurons in the spinal cord and into a region in the brain called the hypothalamus. In turn, the ...

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Electroacupuncture releases stem cells to relieve pain, promote tissue repair, study finds - Science Daily

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Stem Cell Therapies for Degenerative Disc Disease Clinical Pain Advisor (registration) MSCs derived from bone marrow have been successfully differentiated into cardiopoietic cells and used in treatment of heart failure. Fourth- and fifth-generation techniques use genetically modified MSCs and induced pluripotent stem cells (iPSCs), ...

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The new report says the three women, in their 70s and 80s, paid $5,000 to be treated in 2015 for age-related macular degeneration. Participants can also report their concerns to the Office for Human Research Protections within the U.S. Department of Health and Human Services.

The "devastating outcomes" experienced by the women raise the "need for oversight of such clinics and for the education of patients by physicians and regulatory bodies", the paper said.

The women all suffered detached retinas, vision loss, and hemorrhages in their eyes.

"We don't mean to say all stem cell clinical studies are risky", coauthor Dr. Thomas Albini of the University of Miami told Reuters Health in a telephone interview.

Paul Knoepfler, a stem-cell scientist at the University of California at Davis who is a frequent critic of the clinics, said he didn't understand why the FDA and the NIH have not moved more aggressively to ensure patient safety. They sought treatment at a Florida clinic that had announced a study to treat the condition on clinicaltrials.gov, a federal database of research studies. Two out of the three patients found the trial through the website, which doesn't fully vet trials for scientific soundness. "Platelet count increased to 1.01m3 following the treatment and there were remarkable improvements in other symptoms", said Geeta Shroff, Stem Cell Specialist, Director, Nutech Mediworld. Stem cell clinics have cropped up all over the United States in recent years and are operating in a self-perceived regulatory loophole. Stem cells were then extracted from the fat and injected into their eyes. Albini says the complications could have come from injecting a contaminant into the eye, or from the fact that the stem cells may have turned into myofibroblasts after the injections, which are cells associated with scarring.

The Japanese case marks the first time anyone has given induced pluripotent stem (iPS) cells to a patient to treat any condition.

Legitimate medical research seldom requires patients to pay and, in the case of eye treatments, only one eye is treated at a time so doctors can gauge its effectiveness, the Kuriyan team said.

Although the women had moderate vision loss prior to the stem cell treatments, a year later their vision ranged from total blindness to 20/200, which is considered legally blind.

And even if the interventions were done well, they say, there is no evidence that they could have restored the patients' vision. They first cultivate stem cells to form the retinal pigmented epithelial cells that are needed to restore a damaged retina.

Shoddy stem cell preparation may have led to some of the complications, said the study authors.

The episode, described Wednesday in an article in the New England Journal of Medicine, represents one of the most egregious examples of patient injury involving a stem-cell clinic. The company also noted that it does not now treat eye patients.

The paper also mentions that the women believed that they were taking part in a clinical trial because they were aware of the clinic's work on the ClinicalTrials.gov website run by the U.S. National Library of Medicine. In other words, the company claims the study was stopped before patients were enrolled. In fact, doctors have done bone marrow transplant, a procedure where stem cell transplantation is performed.

"There's this perception that there are all these stem cell therapies out there that are close to clinical application that. are being held back by regulators and if they just step back, there would be all these treatments", he said. However, it can be hard for patients to distinguish between trials that are legitimate, and those that are not, the authors wrote.

"There's no excuse for not designing a trial properly and basing it on preclinical research", added study Jeffrey Goldberg, also a study author, of Stanford University's School of Medicine.

Researchers from the Luxembourg Centre for Systems Biomedicine (LCSB) of the University of Luxembourg and an global team have now identified an ingenious mechanism by which the body orchestrates the regeneration of red and white blood cells from progenitor cells.

See if a trial is affiliated with an academic medical center - that's a good sign it is legitimate, they say.

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3 women blinded after receiving stem cell therapy for macular degeneration - ClickLancashire

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Scientists at The University of Queensland have taken a significant step forward in cardiac disease research by creating a functional beating human heart muscle from stem cells.

Dr James Hudson and Dr Enzo Porrello from the UQ School of Biomedical Sciences collaborated with German researchers to create models of human heart tissue in the laboratory so they can study cardiac biology and diseases in a dish.

The patented technology enables us to now perform experiments on human heart tissue in the lab, Dr Hudson said.

This provides scientists with viable, functioning human heart muscle to work on, to model disease, screen new drugs and investigate heart repair.

The UQCardiac Regeneration Laboratoryco-leaders have also extended this research and shown that the immature tissues have the capacity to regenerate following injury.

In the laboratory we used dry ice to kill part of the tissue while leaving the surrounding muscle healthy and viable, Dr Hudson said.

We found those tissues fully recovered because they were immature and the cells could regenerate in contrast to what happens normally in the adult heart where you get a dead patch.

Our goal is to use this model to potentially find new therapeutic targets to enhance or induce cardiac regeneration in people with heart failure.

Studying regeneration of these damaged, immature cells will enable us to figure out the biochemical events behind this process.

Hopefully we can determine how to replicate this process in adult hearts for cardiovascular patients.

UQ scientists create beating human heart muscle from The University of Queensland on Vimeo.

Each year, about 54,000 Australians suffer a heart attack, with an average of about 23 deaths every day.

The UQ research has been supported by the National Health and Medical Research Council (NHMRC) and the National Heart Foundation.

Heart Foundation Queensland CEO Stephen Vines said the charity was excited to fund such an important research project.

Heart attack survivors who have had permanent damage to their heart tissue are essentially trying to live on half an engine, Mr Vines said.

The research by Dr Hudson and Dr Porello will help unlock the key to regenerating damaged heart tissue, which will have a huge impact on the quality of life for heart attack survivors.

Dr Hudson and Dr Porello are deserved recipients of our highest national research accolade the Future Leader Fellowship Award.

The research is published in Circulation and Development.

Media: Dr James Hudson, j.hudson@uq.edu.au; Kim Lyell, k.lyell@uq.edu.au, 0427 530 647.

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Scientists create 'beating' human heart muscle for cardiac research - UQ News

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A discovery, several years in the making, by a University at Buffalo research team has proven that adult skin cells can be converted into neural crest cells (a type of stem cell) without any genetic modification, and that these stem cells can yield other cells that are present in the spinal cord and the brain.

The practical implications could be very significant, from studying genetic diseases in a dish to generating possible regenerative cures from the patient's own cells.

"It's actually quite remarkable that it happens," said Stelios T. Andreadis, Ph.D., professor and chair of UB's Department of Chemical and Biological Engineering, who recently published a paper on the results in the journal Stem Cells.

The identity of the cells was further confirmed by lineage tracing experiments, where the reprogrammed cells were implanted in chicken embryos and acted just as neural crest cells do.

Stem cells have been derived from adult cells before, but not without adding genes to alter the cells. The new process yields neural crest cells without addition of foreign genetic material. The reprogrammed neural crest cells can become smooth muscle cells, melanocytes, Schwann cells or neurons.

"In medical applications this has tremendous potential because you can always get a skin biopsy," Andreadis said. "We can grow the cells to large numbers and reprogram them, without genetic modification. So, autologous cells derived from the patient can be used to treat devastating neurogenic diseases that are currently hampered by the lack of easily accessible cell sources."

The process can also be used to model disease. Skin cells from a person with a genetic disease of the nervous system can be reprogrammed into neural crest cells. These cells will have the disease-causing mutation in their chromosomes, but the genes that cause the mutation are not expressed in the skin. The genes are likely to be expressed when cells differentiate into neural crest lineages, such as neurons or Schwann cells, thereby enabling researchers to study the disease in a dish. This is similar to induced pluripotent stem cells, but without genetic modification or reprograming to the pluripotent state.

The discovery was a gradual process, Andreadis said, as successive experiments kept leading to something new. "It was one step at a time. It was a very challenging task that took almost five years and involved a wide range of expertise and collaborators to bring it to fruition," Andreadis said. Collaborators include Gabriella Popescu, Ph.D., professor in the Department of Biochemistry in the Jacobs School of Medicine and Biomedical Sciences at UB; Song Liu, Ph.D., vice chair of biostatistics and bioinformatics at Roswell Park Cancer Institute and a research associate professor in biostatistics UB's School of Public Health and Health Professions; and Marianne Bronner, Ph.D., professor of biology and biological engineering, California Institute of Technology.

Andreadis credits the persistence of his then-Ph.D. student, Vivek K. Bajpai, for sticking with it.

"He is an excellent and persistent student," Andreadis said. "Most students would have given up." Andreadis also credits a seed grant from UB's office of the Vice President for Research and Economic Development's IMPACT program that enabled part of the work.

The work recently received a $1.7 million National Institutes of Health grant to delve into the mechanisms that occur as the cells reprogram, and to employ the cells for treating the Parkinson's-like symptoms in a mouse model of hypomyelinating disease.

"This work has the potential to provide a novel source of abundant, easily accessible and autologous cells for treatment of devastating neurodegenerative diseases. We are excited about this discovery and its potential impact and are grateful to NIH for the opportunity to pursue it further," Andreadis said.

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From Skin to Brain: Stem Cells Without Genetic Modification - Bioscience Technology

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Monday, March 13, 2017 3 a.m. CDT

BRUSSELS (Reuters) - Belgian biotech group Tigenix said on Monday its medical trial with a novel treatment for patients at risk of heart failure after a coronary attack was successful.

The group said patients treated in its PhaseI/II trial of donor-derived expanded cardiac stem cells (AlloCSC) showed no side-effects and all of them continued to live after 30 days, six months and a year.

Tigenix added that in one subgroup of trial patients associated with a poor long-term outlook, there was a larger reduction in the size of infarction, tissue death due to inadequate blood supply.

"This is the first trial in which it has been demonstrated that allogeneic cardiac stem cells can be transplanted safely through the coronary tree," one of the doctors in the trial said.

The group said it would now analyze the data from the trial and decide on how to proceed with its research.

(Reporting by Robert-Jan Bartunek; editing by Philip Blenkinsop)

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Belgium's Tigenix says heart attack stem cell trial successful - KFGO

Cardiac Stem Cells Comments Off on Belgium’s Tigenix says heart attack stem cell trial successful – KFGO | March 15th, 2017

A longtime product developer is bringing a peptide ingredient to the US market that has been researched for a unique property promoting the growth of bone marrow stem cells.

Called DH Stemogen, the product is the brainchild of Dr Marvin Heuer MD who has a history of product development with sports nutrition company MuscleTech. Dr. Heuer has a background in clinical research, having spent many years in drug development at Glaxo Smith Kline. He also runs a contract research firm, Heuer M.D. Research Inc. and is the CEO of omega-3 supplement manufacturer Blue Ocean Nutrascience.

The new product, called DH Stemogen is based on a Cyclo-{L-ALA-L-GLU(TRP-OH) peptide that was developed by a Russian biochemist.

Its a peptide that is a mimic of a naturally occurring thymic peptide,Dr. Heuer told NutraIngredients-USA. Heuer was promoting the launch of the product at the recent Expo West trade show in Anaheim, CA. At Heuer M.D. Research, as a company we are out looking for novel ingredientsto bring out, hopefully in the nutraceutical area.

We got interested in Prof. Vlad Deigins peptide research, Dr. Heuer said (Deigin is associated with the Institute of Bioorganic Chemistry at the Russian Academy of Sciences in Moscow.)We looked at this particular compound that he was launching as an ingredient in Russia about a year ago.

The peptide in DH Stemogen targets a particular type of stem cell hematopoietic cells (HSC). Stem cells in general are the building blocks of our bodies. These cells are able to transform themselves into almost any type of cell. There are various sources of stem cells in an adult body. One of the most important of them comprises the bone marrow, where the HSCs are produced. HSCs transform into all the main cell types in our blood, including red blood cells and white blood cells. Dr. Heuer said there is some evidence that those cells are able to reconstruct other body tissues by transforming into the specific tissue type cell such as liver, nervous tissue, kidney and skin.

These properties would seem to make Stemogen a natural for a healthy aging product positioning, Dr. Heuer said. But Deigins research, trending as it does over into disease endpoints, is a little problematical when it comes to supporting US-style structure function claims, he admitted. Other countries dont make the same hard and fast distinctions between dietary ingredients meant for supplement applications and active pharmaceutical agents meant for drugs, he said.

We are going to be very cautious about making structure/function claims,Dr. Heuer said. The product at the moment saysSupport your immune system and Support healthy levels of stem cells in your blood.

We are about to begin a whole profile of research in the U.S. and Canada, he added.

Dr. Heuer said one thing thats unique about the ingredient (and something that he says Deigin has patented) is a structural twist that improves the peptides stability. The criticism of some other novel peptides has been that interesting as their properties might be, once they hit the stomachs gastric fluid they blow apart into their constituent amino groups and all those novel properties are lost.

He has a patent on the way he makes this with a hex ring on the end that protects it in the GI tract and allows it to be absorbed,he said.

Bringing a synthetic analogue of a naturally occurring peptide to market as a dietary ingredient would seem to pose significant regulatory challenges. Dr. Heuer said hes confident there is a way through that thicket. The plan is to start first with a GRAS filing, and Dr. Heuer said he believes that the peptide would fall under the amino acid category in the DHSEA definitions of what constitutes a dietary ingredient.

Certainly there is a precedent of complex peptides being sold on the market, he said.

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Peptide aimed at stem cell genesis debuts on supplement market - NutraIngredients-usa.com

Bone Marrow Stem Cells Comments Off on Peptide aimed at stem cell genesis debuts on supplement market – NutraIngredients-usa.com | March 15th, 2017

RenovaCare Inc. (OTCQB:RCAR) is a New York City-based biotechnology company developing its patented CellMist and SkinGun stem cell technologies for treating burns in weeks or less as well as treating chronic and acute wounds, acne scarring, and skin defects and diseases. In December, it received a U.S. patent for its SkinGun device.

Before joining RenovaCare, CEO Thomas Bold was CEO of StemCell Systems. He has more than 15 years of experience in medical biotechnology device manufacturing and stem cell platform development.

Harlan Levy: How does your CellMist technology specifically work?

Thomas Bold: Doctors isolate a high concentration of the most desirable stem cell population from a very small donor sample of the patient's own skin and suspended in the liquid CellMist Solution. It's then gently sprayed onto wound sites using our SkinGun, which looks like Captain Kirk's particle-beam gun, the "Phaser" in the Star Trek TV series.

The isolated cells include cells that proliferate rapidly in order to achieve quick re-epithelialization. This is the stage at which a burn is technically considered "healed" and patients are often discharged. The average person would recognize this healing phase as the point at which the wound develops a thin, shiny, pink-colored protective layer.

H.L.: What are existing burn treatments, and how do they compare with the SkinGun treatment?

T.B.: Traditional skin grafting has been the treatment for burns and wounds for centuries. More recently, mesh grafting has become the latest standard of care. This process surgically removes large sheets of healthy skin from the patient. Following this painful donor procedure, the sheet is punctured in a grid-like pattern to form an expandable mesh. Surgeons pull this mesh as wide as feasible and surgically stitch this skin to the patient's wound. The procedure is extremely painful, creates an additional wound at each donor site and results in poor cosmetic outcomes, often with scarred and deformed skin.

This transplanted skin can result in restricted joint movement and is unable to grow with the patient. Consequently, mesh graft patients require months and sometimes up to a year of physical therapy and can face psychological problems from the permanent disfigurement of scarring. In addition, long-term pain management with painkillers is very often necessary.

With the RenovaCare treatment technology, by spraying the patient's stem cells, the SkinGun overcomes the need for removing large sheets of donor skin, and the resultant healing does not require prolonged physical therapy. The spray procedure is gentle, and the skin that regrows looks, feels, and functions as the original skin that it replaces. Most often the healing process takes only a week.

It's very important to note here that a sheet of meshed skin covers only up to six times its original donor area. The RenovaCare system covers up to 100 times its donor skin sample. This is why the donor skin sample can be so small compared to the injured treatment area.

H.L.: What about scars and infection potential compared with conventional treatments?

T.B.: A wound heals from the edges towards the middle. The bigger the wound, the longer this process takes. And the longer this process takes, the higher the risk of infection and scarring.

Imagine a large burn of 20, 30, 40 percent of your total body surface. With our CellMist System, the doctor sprays the patient's own stem cells with a highly regenerative capacity onto the wound and, by doing so, creates tens of thousands of little regenerative islands across the wound. These islands grow outwards, ultimately connecting to each other to create a protective epithelial skin layer that covers the wound.

Experts believe the formation of this pink-colored layer marks the moment of re-epithelization where the risk of infection is reduced and the patient's wound is effectively healed. Beyond this stage, the cosmetic healing process also happens entirely natural to produce a scar-free result where, finally, skin color, tone and pigmentation are restored.

Since the RenovaCare spray procedure uses the patient's own stem cells, there isn't the risk of tissue rejection, infection, or ongoing immuno suppression therapy.

H.L.: What results have you found for patients using the SkinGun?

T.B.: We have many examples of patients recovering from severe burns within a week or two, scar-free, and walking away with unlimited joint restrictions.

In the case of one patient with severe electrical burns to over a third of his body, we were able to spray his wounds with 23 million stem cells isolated from a tiny two-inch-by two-inch sample of his own skin. Within five days of treatment, his chest and arms were already healed. Four days later, the patient was discharged from the hospital.

It's also important to note that reconstructive surgery for burn patients is especially challenging when tackling joints in the body. To this end, the authors of a case study in the reputable journal "Burns," said, "Cell-spray grafting is also especially suitable for hands and joint areas, where prolonged times to re-epithelization may significantly impact functionality and esthetic outcome."

H.L.: What different uses does the SkinGun have beside burns?

T.B.: Currently, we are focusing on severe second-degree burns, but we see the RenovaCare technology also applicable for other indications such as cosmetic procedures targeting skin pigmentation disorders, scar treatment, and other related conditions.

Our goal is to bring to market the world's most advanced technology for skin repair using a patient's own stem cells.

H.L.: Is there a record of the SkinGun use in the States and abroad?

T.B.: Having treated 72 burn patients to date, the company's early clinical target is burns with follow-on indications, including chronic wounds and cosmetic procedures.

H.L.: How much research went into creating the SkinGun and over what time period?

T.B.: The birth of RenovaCare technology goes back to the early 2000s in Berlin, Germany. Researchers, at that time, were trying to "grow" skin by seeding stem cells inside multi-dimensional bioreactors. They soon discovered that these artificial chambers were no match for the growth of the same cells when transplanted inside a human body; thus, the birth of a concept to use a patient's own wound as a natural bioreactor.

A study published in "Advances in Plastic Surgery" highlights 19 early patients with deep dermal wound burns to the face and neck, complex three-dimensional surfaces. Researchers achieved such outstanding results using our cell spray that they refused to perform further skin grafting. Instead, surgeons adopted our founding technology as their standard of care.

Let me quote from the surgeons' study, which states

"We refuse to perform a prospective randomized study with groups in which traditional skin grafting and/or wound healing are still applied for the therapy for deep dermal burns due to the excellent results in our study. The method of CEA spray application has become our standard of care for these indications. The faster wound closure, the promotion of spontaneous wound healing by keratinocyte application, as well as the preservation of donor sites are further advantages of the method."

The same paper concluded that "using a spray technique results in excellent cosmetic outcomes compared with any other method."

H.L.: How has the technology changed since then?

T.B.: Since the time of this early approach, our technology has evolved and matured significantly. Our cell isolation no longer requires complex procedures, culturing, expansion, and processing time, and our stem cell spray device no longer requires multiple hand-assembled parts. Its independent power and flow-control unit has been condensed in size from a 2-foot cube down to a 9-volt battery placed inside the handle of a single handheld spray gun.

H.L.: What is the potential market for the technology in dollars and number of patients?

T.B.: Conservatively speaking, the market for our technology exceeds $50 billion. There are nearly a million people who suffer from burns each year in the U.S. alone. According to the American Burn Association, burn injuries continue to be one of the leading causes of accidental death and injury in the U.S, and one civilian fire death occurs every two hours and forty minutes.

H.L.: How much would you estimate the treatment cost may be for each different use?

T.B.: The SkinGun technology is currently under development and not approved for clinical use in the U.S., so it's too early to talk about what the treatment will cost. We have always been mindful of reimbursement, and nearly two years ago, we commissioned an investigation into the reimbursement pathway for our CellMist System. We know that reimbursement opportunities are available by way of current coding and practices.

We have further investigated and evaluated the "bundling" approach currently advocated for by insurers and are confident that that our technology is well placed to take advantages of any shift towards such a model.

H.L: What is the schedule to get Federal Drug Administration clearance?

T.B.: In order to achieve FDA clearance for the CellMist System and the SkinGun, we will be working to show our technology is safe and prove its efficacy within applicable clinical trial formats and according to the relevant regulatory requirements. I can't speculate as to how long the FDA clearance process will take, and, therefore, it's hard to speculate when our product will be commercialized.

H.L.: What other products are you investigating and how may they work?

T.B.: We are focusing on bringing the SkinGun and our stem cell spray technology to market at this time.

H.L.: What is your background, including age, education, prior employment?

T.B.: Before joining RenovaCare I worked as the CEO of StemCell Systems GmbH, a Berlin-based biomedical company engaged in the development and commercialization of advanced cell culture bioreactors. I have more than 15 years of professional business experience in the field of medical biotechnology device manufacturing, stem cell culture technology platform development and regenerative medicine research project management and product development. I also co-founded several start-up companies in Germany.

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