Community rallies for 12-year-old from Pine Island battling leukemia

PINE ISLAND, Minn. (KTTC) -- A Pine Island sixth grader is preparing to return to school after a nearly year-long battle with leukemia. Lacey McClain, 12, was diagnosed with T-cell acute lymphoblastic leukemia last year and has undergone extensive treatment at Mayo Clinic Hospital, Saint Marys Campus since the diagnosis.

We thought she had mono, said Lacey's mom, Chrissie Jurrens. We took her in to make sure that was the case and the doctor came to us and said her white cell blood count was way higher than normal."

In fact, Lacey's white blood cell count was more than 20 times above normal. On October 29, 2013, she was diagnosed and immediately began aggressive chemotherapy treatment. It wasn't until January that Lacey began going into remission. Her battle didn't end there. Doctors told the family Lacey would also need a stem cell transplant.

Chemotherapy isn't easy on anybody's body, but she did a really good job of keeping her composure and staying strong and just knowing she was going to get through it, said Jurrens.

Months after going into remission, Lacey received a stem cell transplant on June 3. The transplant meant a 49-day stay in the hospital. In total, the 12-year-old has spent more than 100 days in the hospital.

She was my strength and I tried the best I could to be hers," said Jurrens. "We just stuck together."

Since her homecoming in late July, Lacey has spent much of her time resting and keeping up with doctor's appointments in order to get the all clear to go back to school next week. Despite, a compromised immune system, she'll be allowed to return to classes and a normal routine.

I'm excited for her, but I'm nervous because I know that a lot of germs are picked up in school and that's where most kids get sick. I kind of have mixed emotions about it, said Jurrens.

The family hasn't made their journey to recovery alone, Jurrens said there's been an outpouring of support from the community, including a benefit and 5K planned for Saturday, September 13.

She's not totally capable of being that normal kid yet, said Jurrens. I know that she's going to get there and I know that we're going to have good times again and I'm going to see her doing the things that I want to see her doing again."

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Community rallies for 12-year-old from Pine Island battling leukemia

UCSD Medical School Holding Clinical Trials For Stem Cell …

UC San Diego is initial site for first-in-human testing of implanted cell therapy.

Researchers at the University of California, San Diego School of Medicine, in partnership with ViaCyte, Inc, a San Diego-based biotechnology firm specializing in regenerative medicine, have launched the first-ever human clinical trial of a stem cell-derived therapy for patients with Type 1 diabetes.

The trial will assess the safety and efficacy of a new investigational drug called VC-01, which was recently approved for testing by the U.S. Food and Drug Administration. The two-year trial will involve four to six testing sites, the first being at UC San Diego, and will recruit approximately 40 study participants.

The goal, first and foremost, of this unprecedented human trial is to evaluate the safety, tolerability and efficacy of various doses of VC-01 among patients with type 1 diabetes mellitus, said principal investigator Robert R. Henry, MD, professor of medicine in the Division of Endocrinology and Metabolism at UC San Diego.

He is also chief of the Section of Endocrinology, Metabolism & Diabetes at the Veterans Affairs San Diego Healthcare System.

We will be implanting specially encapsulated stem cell-derived cells under the skin of patients where its believed they will mature into pancreatic beta cells able to produce a continuous supply of needed insulin.

Previous tests in animals showed promising results. We now need to determine that this approach is safe in people.

Development and testing of VC-01 is funded, in part, by the California Institute for Regenerative Medicine, the states stem cell agency, the UC San Diego Sanford Stem Cell Clinical Center and JDRF, the leading research and advocacy organization funding type 1 diabetes research.

Type 1 diabetes mellitus is a life-threatening chronic condition in which the pancreas produces little or no insulin, a hormone needed to allow glucose to enter cells to produce energy.

It is typically diagnosed during childhood or adolescence, though it can also begin in adults. Though far less common than Type 2 diabetes, which occurs when the body becomes resistant to insulin, Type 1 may affect up to 3 million Americans, according to the JDRF.

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UCSD Medical School Holding Clinical Trials For Stem Cell ...

How to tell good stem cells from the bad: Yale researchers answer key question

The promise of embryonic stem cell research has been thwarted by an inability to answer a simple question: How do you know a good stem cell from a bad one?

Yale researchers report in the Sept. 4 issue of the journal Cell Stem Cell that they have found a marker that predicts which batch of personalized stem cells will develop into a variety of tissue types and which will develop into unusable placental or tumor-like tissues.

Scientists have been unable to capitalize on revolutionary findings in 2006 that adult cells could be made young again with the simple introduction of four factors. Hopes were raised that doctors would soon have access to unlimited supplies of a patients own iPSCs induced pluripotent stem cells that could be used to repair many types of tissue damage. However, efforts to direct these cells to therapeutic goals have proved difficult. Many attempts to use cells clinically have failed because they form tumors instead of the desired tissue.

The team of Yale Stem Cell Center researchers led by senior author Andrew Xiao identified a variant histone a protein that helps package DNA which can predict the developmental path of iPSC cells in mice. An accompanying paper in the same journal by researchers at the Whitehead Institute at MIT and Hebrew University in Israel also identifies at different marker that also appears to predict stem cell fate.

The trend is to raise the standards and quality very high, so we can think about using these cells in clinic, Xiao said. With our assay, we have a reliable molecular marker that can tell what is a good cell and what is a bad one.

Lead author of the paper is Tao Wu of Yale.

Research is funded by Yale and Connecticut Stem Cell Foundation.

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How to tell good stem cells from the bad: Yale researchers answer key question

New Research Method Opens Door to Therapy with Human Muscle Stem Cells Promising Method Developed

27.08.2014 - (idw) Max-Delbrck-Centrum fr Molekulare Medizin (MDC) Berlin-Buch

Stem cells are essential for the repair of muscle damage, but all attempts to manipulate human muscle stem cells for therapy have thus far failed. Now Dr. Andreas Marg and Prof. Simone Spuler of the Experimental and Clinical Research Center (ECRC), a joint cooperation between the Max Delbrck Center (MDC) and the Charit, have shown how this might work. They developed a method in which they did not isolate the muscle stem cells, but rather cultivated, proliferated and transplanted them along with their muscle fibers. Using this method in mice, they were able to successfully regenerate muscle tissue. Thus they have opened the door for the use of muscle stem cells to treat muscle diseases.* "Muscle stem cells, which we also refer to as satellite cells, can awaken in their stem cell niche after decades of quiescence and can then repair damaged muscle tissue," Professor Spuler explained. At the ECRC in Berlin-Buch, the neurologist heads the University Outpatient Clinic for Muscle Disorders and the Department of Muscle Sciences. She and her team are exploring the causes of muscle diseases. Evidence shows that satellite cells are active in people with severe muscle diseases such as Duchenne muscular dystrophy, a severe genetic disease in which the muscles degenerate. "But at some point, she added, the reservoir is depleted of muscle stem cells and muscle wasting cannot be stopped."

All attempts to rebuild muscle tissue by transplanting satellite cells in patients with Duchenne muscular dystrophy have failed. The transplanted cells are not viable. Furthermore, the use of other cells with potential to regenerate muscle cells has shown little success. These cells have only limited potential to regenerate muscle. But how is it possible to nevertheless use the bodys own self-renewal potential and the reconstruction potential of satellite cells?

The offer of developmental biologist Professor Carmen Birchmeier (MDC) to participate in the network project on satellite cells (SatNet) of the Federal Ministry of Education and Research pointed Professor Spuler and her co-workers in the right direction. One of the topics of the project was to elucidate why satellite cells rapidly lose their regeneration potential if they are kept in a cell culture. This led to the idea to cultivate the satellite cells together with the surrounding muscle tissue to see whether the cells, if they remain in their accustomed milieu, might possibly regenerate better.

Muscle biopsy specimens from young and old donors After due approval and written, informed consent, Professor Spuler and Dr. Marg obtained specimens of fresh thigh muscle tissue from patients between 20 and 80 years of age from neurosurgeons of Helios Klinikum Berlin-Buch, which like the MDC is located close to the ECRC.

From the biopsy specimens, Professor Spuler and her co-workers dissected more than 1000 muscle fiber fragments, each about 2-3 millimeters long. Remarkably, the researchers found the number of stem cells in the individual tissue specimens to be independent of the age of the donor and that thousands of myoblasts developed from a small number of satellite cells. After further developmental steps, these fuse into muscle fibers.

Dr. Marg: Satellite cells need to have their local milieu around them Professor Spuler and her co-workers cultivated the muscle fiber fragments with the satellite cells, initially for up to three weeks. During this time, the satellite cells increased by 20- to 50-fold, but numerous connective tissue cells also developed in these cultures. To prevent this, the researchers concurrently subjected the muscle fragments to oxygen deprivation (hypoxia) and to cooling (hypothermia) at 4 degrees Celsius. Under these conditions, only satellite cells are able to survive in their stem cell niche, in contrast to the connective tissue cells. Apparently, the satellite cells receive the proper nutrients in their own local milieu, Dr. Marg said.

First success in mice The ECRC researchers then tried out their therapy approach in mice in which muscle regeneration had been inhibited by irradiation. They grafted the muscle fragments containing the satellite cells, which following the hypothermia had been kept for two weeks in culture dishes, into the tibalis anterior muscle. The researchers found that the muscles of animals that had been treated with these fiber fragments regenerated particularly well.

Objective: to couple satellite cells with gene therapy However, a genetic muscle disease cannot be successfully treated alone by transplanting muscle fragments. Professor Spuler: The idea is therefore to equip the satellite cells additionally with a healthy gene that repairs the defective gene and then to transfect it with the aid of a non-viral gene taxi into the muscles to be treated. In a first experiment with a reporter gene in the Petri dish, Professor Spuler and her co-workers proved that this is possible in principle. The reporter gene fluoresces green when it is transfected into the satellite cell. As gene taxi the researchers use the Sleeping Beauty transposon a jumping gene that can change its position in the genome. This transposon technique was developed several years ago by Dr. Zsuzsanna Izsvk (MDC) and Dr. Zoltn Ivics (Paul Ehrlich Institute, Frankfurt) and is considered to be a very promising delivery vehicle (vector) for gene therapy.

Before the method developed by Professor Spuler and her group can be used to benefit patients, some hurdles remain to be taken. So far, the transplantation has succeeded in small mice muscles. In clinical trials, the scientists and physicians want to determine whether this technique can be used in large human thigh muscles, which may be severely altered due to a muscular disease.

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New Research Method Opens Door to Therapy with Human Muscle Stem Cells Promising Method Developed

Removing Programming Material After Inducing Stem Cells Could Improve Their Regeneration Ability

Durham, NC (PRWEB) August 22, 2014

Human induced pluripotent stem cells (hiPSCs) have great potential in the field of regenerative medicine because they can be coaxed to turn into specific cells; however, the new cells dont always act as anticipated. They sometimes mutate, develop into tumors or produce other negative side effects. But in a new study recently published in STEM CELLS Translational Medicine, researchers appear to have found a way around this, simply by removing the material used to reprogram the stem cell after they have differentiated into the desired cells.

The study, by Ken Igawa, M.D., Ph.D., and his colleagues at Tokyo Medical and Dental University along with a team from Osaka University, could have significant implications both in the clinic and in the lab.

Scientists induce (differentiate) the stem cells to become the desired cells, such as those that make up heart muscle, in the laboratory using a reprogramming transgene that is, a gene taken from one organism and introduced into another using artificial techniques.

We generated hiPSC lines from normal human skin cells using reprogramming transgenes, then we removed the reprogramming material. When we compared the transgene-free cells with those that had residual transgenes, both appeared quite similar, Dr. Igawa explained. However, after the cells differentiation into skin cells, clear differences were observed.

Several types of analyses revealed that the keratinocytes cells that make up 90 percent of the outermost skin layer that emerged from the transgene-free hiPSC lines were more like normal human cells than those coming from the hiPSCs that still contained some reprogramming material.

These results suggest that transgene-free hiPSC lines should be chosen for therapeutic purposes, Dr. Igawa concluded.

Human induced pluripotent stem cell (hiPSC) lines have potential for therapeutics because of the customized cells and organs that can potentially be induced from such cells, Anthony Atala, M.D., editor of STEM CELLS Translational Medicine and director of the Wake Forest Institute for Regenerative Medicine. This study illustrates a potentially powerful approach for creating hiPSCs for clinical use.

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The full article, Removal of Reprogramming Transgenes Improves the Tissue Reconstitution Potential of Keratinocytes Generated From Human Induced Pluripotent Stem Cells, can be accessed at http://stemcellstm.alphamedpress.org/content/early/2014/07/14/sctm.2013-0179.abstract.

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Removing Programming Material After Inducing Stem Cells Could Improve Their Regeneration Ability

Vanderbilt researchers find that coronary arteries hold heart-regenerating cells

PUBLIC RELEASE DATE:

20-Aug-2014

Contact: Craig Boerner craig.boerner@vanderbilt.edu 615-322-4747 Vanderbilt University Medical Center

Endothelial cells residing in the coronary arteries can function as cardiac stem cells to produce new heart muscle tissue, Vanderbilt University investigators have discovered.

The findings, published recently in Cell Reports, offer insights into how the heart maintains itself and could lead to new strategies for repairing the heart when it fails after a heart attack.

The heart has long been considered to be an organ without regenerative potential, said Antonis Hatzopoulos, Ph.D., associate professor of Medicine and Cell and Developmental Biology.

"People thought that the same heart you had as a young child, you had as an old man or woman as well," he said.

Recent findings, however, have demonstrated that new heart muscle cells are generated at a low rate, suggesting the presence of cardiac stem cells. The source of these cells was unknown.

Hatzopoulos and colleagues postulated that the endothelial cells that line blood vessels might have the potential to generate new heart cells. They knew that endothelial cells give rise to other cell types, including blood cells, during development.

Now, using sophisticated technologies to "track" cells in a mouse model, they have demonstrated that endothelial cells in the coronary arteries generate new cardiac muscle cells in healthy hearts. They found two populations of cardiac stem cells in the coronary arteries a quiescent population in the media layer and a proliferative population in the adventitia (outer) layer.

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Vanderbilt researchers find that coronary arteries hold heart-regenerating cells

Coronary arteries hold heart-regenerating cells

Endothelial cells residing in the coronary arteries can function as cardiac stem cells to produce new heart muscle tissue, Vanderbilt University investigators have discovered.

The findings, published recently in Cell Reports, offer insights into how the heart maintains itself and could lead to new strategies for repairing the heart when it fails after a heart attack.

The heart has long been considered to be an organ without regenerative potential, said Antonis Hatzopoulos, Ph.D., associate professor of Medicine and Cell and Developmental Biology.

"People thought that the same heart you had as a young child, you had as an old man or woman as well," he said.

Recent findings, however, have demonstrated that new heart muscle cells are generated at a low rate, suggesting the presence of cardiac stem cells. The source of these cells was unknown.

Hatzopoulos and colleagues postulated that the endothelial cells that line blood vessels might have the potential to generate new heart cells. They knew that endothelial cells give rise to other cell types, including blood cells, during development.

Now, using sophisticated technologies to "track" cells in a mouse model, they have demonstrated that endothelial cells in the coronary arteries generate new cardiac muscle cells in healthy hearts. They found two populations of cardiac stem cells in the coronary arteries -- a quiescent population in the media layer and a proliferative population in the adventitia (outer) layer.

The finding that coronary arteries house a cardiac stem cell "niche" has interesting implications, Hatzopoulos said. Coronary artery disease -- the No. 1 killer in the United States -- would impact this niche.

"Our study suggests that coronary artery disease could lead to heart failure not only by blocking the arteries and causing heart attacks, but also by affecting the way the heart is maintained and regenerated," he said.

The current research follows a previous study in which Hatzopoulos and colleagues demonstrated that after a heart attack, endothelial cells give rise to the fibroblasts that generate scar tissue.

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Coronary arteries hold heart-regenerating cells

Cedars-Sinai Medical Tip Sheet for August

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Newswise Expert on Parkinsons Disease and Depression Available for Interviews Michele Tagliati, M.D., director of the Movement Disorders Program at Cedars-Sinai Medical Center, is available to discuss Parkinsons disease and depression. An internationally renowned expert in all types of movement disorders, including Parkinsons Disease, he believes it is important to understand several aspects of the disease: Parkinsons Disease (PD) is not only a movement disorder; more than half of patients diagnosed with PD also suffer from depression. Many patients say that depression is the most disabling symptom of PD (not shaking or difficulty walking, etc.) Depression is intrinsic to PD, but differs from bipolar or major depression. Its symptoms may include general apathy (loss of interest in life), fatigue, insomnia, lack of appetite, etc. Patients may or may not recognize these as symptomatic of depression, so it is important for their physicians to proactively ask appropriate questions that can help identify depression. While depression can be a major problem for patients with PD, its important to realize that help is available. Medications and counseling can help reduce the severity of symptoms. CONTACT: Sandy Van, 808-526-1708; Email sandy@prpacific.com

Unique Study Focuses on Combined Treatment Approach for Locally Advanced Pancreatic Cancer Investigators at the Cedars-Sinai Samuel Oschin Comprehensive Cancer Institute are developing a novel, multistep investigational treatment for one of the most complex and difficult-to-treat forms of the disease, locally advanced pancreatic cancer. Locally advanced pancreatic cancer has the lowest survival rate of any solid tumor, with a cumulative five-year survival rate of only 4 percent for all stages of disease. Surgery is rarely an option for patients because tumors often involve vital blood vessels. Chemotherapy and radiotherapy given concurrently remain the mainstay treatment, yet to-date, no treatment has had a significant impact on improving outcomes. CONTACT: Cara Martinez, 310-423-7798; Email cara.martinez@cshs.org

Research Initiative Will Examine Innovative Strategies to Deliver Care More Efficiently Cedars-Sinai has launched an applied research center to improve the value of patient care inside the medical center and beyond its walls by strengthening patient-doctor bonds and bringing greater efficiency to the delivery of clinical services. Brennan Spiegel, MD, MSHS, will lead the new Cedars-Sinai Center for Outcomes Research and Education, or CS-CORE. As director of Health Services Research, he will oversee a digital health strategy aimed at enhancing healthcare quality and reducing excessive use of resources. CONTACT: Duke Helfand, 310-248-6608; Email duke.helfand@cshs.org

Tissue Collection Aids Search for Neurologic and Neuromuscular Disease Causes and Cures Like other major research centers studying genetic causes of uncommon and poorly understood nervous system disorders, Cedars-Sinai maintains a growing collection of DNA and tissue samples donated by patients. What sets Cedars-Sinais Repository of Neurologic and Neuromuscular Disorders apart is its special emphasis on tissue collection part of its focus on creating future individualized treatments for patients. CONTACT: Sandy Van, 808-526-1708; Email sandy@prpacific.com

Congenital Heart Disease Specialists Develop Nonsurgical Technique to Correct Birth Defects in Premature Infants A new technique for repairing the most common cardiac birth defect in newborns, commonly referred to as a hole in the heart, has been used successfully to mend the condition in six premature infants without subjecting the tiny patients to open-heart surgery. The technique for repairing the condition, patent ductus arteriosus, also called PDA, was published online by the peer-reviewed medical journal Catheterization and Cardiovascular Interventions, the official journal of The Society for Cardiovascular Angiography and Interventions. CONTACT: Sally Stewart, 310-248-6566; Email sally.stewart@cshs.org

Cedars-Sinai Immunotherapy Expert Honored for Work in Kidney Transplantation Ashley Anh Vo, PharmD, administrative director of the Transplant Immunotherapy Program at the Cedars-Sinai Comprehensive Transplant Center, has been named the 2014 Clinician of Distinction by the American Society of Transplantation for her work in developing anti-rejection drug protocols for patients. CONTACT: Laura Coverson, 310-423-5215; Email laura.coverson@cshs.org

Cedars-Sinai Heart Institute Opens First-of-its-Kind Research Stem Cell Clinic for Cardiac Patients Regenerative medicine experts at the Heart Institute have opened a new clinic to evaluate heart and vascular disease patients for participation in stem cell medical studies. The clinic is believed to be the first at a major U.S. academic medical center dedicated to matching patients with appropriate stem cell clinical trials, whether those research interventions are available at the medical center or at other institutions. CONTACT: Sally Stewart, 310-248-6566; Email sally.stewart@cshs.org

Childbirth Experts Debate Best Delivery Practices at Third Annual Birth Community Day Doctors, nurses, midwives and doulas debated healthy labor and delivery practices including the use of the synthetic hormone Pitocin to hasten childbirth and vitamin K to speed blood clotting in newborns -- at the third annual Cedars-Sinai Birth Community Day. CONTACT: Laura Coverson, 310-423-5215; Email laura.coverson@cshs.org

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Cedars-Sinai Medical Tip Sheet for August

Mayo Clinic researching ALS stem cell treatment

by KING 5 HealthLink

KING5.com

Posted on August 7, 2014 at 7:08 PM

Time with grandchildren is especially precious for Linda Leight. Just like Lou Gherig, she has ALS, Amyotrophic Lateral Sclerosis It will eventually paralyze nearly all muscles in the body. With Linda, it started with her voice.

The disease has slowed her speech. Eventually she won't be able to breathe. There is no cure.

So with husband Jerry by her side, she enrolled in a study using stem cells from a patient's own fat and injecting them into the spine.

"We're hopeful that the stem cells will provide a protection for the neurons that remain in the spinal cord and the brain and hopefully slow down the disease and prolong lifespan," said Dr. Nathan Staff.

Just like Lou Gehrig, Linda has her own baseball card to raise awareness. Even if the research doesn't help her, she's hopeful for her grandchildrens' generation.

Another study is using stem cells from a patient's bone marrow. Scientists remain cautious as all of this research is still in the early stages.

Although ALS can run in families, the vast majority of cases don't have a genetic link. One group at higer risk: military veterans who served during the Gulf War. They are almost twice as likely to develop ALS as the general population.

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Mayo Clinic researching ALS stem cell treatment

After Mom Saved By Stem Cell Transplant, Son Donates His Own

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CHICAGO (CBS) A Barrington womans son was inspired to pay it forward after she was given the gift of life

WBBM Newsradios Regine Schlesinger reports Joanne Sullivan received a life-saving stem cell transplant from an anonymous donor in Germany two-and-a-half years ago, after she was diagnosed with myelofibrosis, a form of bone marrow cancer.

I just feel terrific, she said.

The gift motivated her three sons to sign up with a registry for potential bone marrow and stem cell donors.

On Joannes birthday, her youngest son, Bryant, donated his stem cells to someone else.

It was the best birthday gift, I think, to have given her, he said.

Joannes transplant surgeon, Dr. Mrinal Patnaik, said 24 million people worldwide have registered for the transplant list, but he said thats only a drop in the bucket.

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After Mom Saved By Stem Cell Transplant, Son Donates His Own