Category Archives: California Stem Cells

April 14, 2017 -- At 55, George Chung of Los Angeles could keep up with skiers decades younger, taking on difficult slopes for hours and hours. "Skiing was my passion," he says.

Then the pain started, and the bad news. He had severe osteoarthritis, the ''wear-and-tear'' type, in both knees. Doctors suggested surgery, but he chose instead an investigational treatment -- injections of stem cells. Two months after the first treatment, he was out of pain. "I had been in pain of various degrees for 6 years," he says.

Now, nine treatments and 3 years later, he is back to intense skiing. Last year, he also took up long-distance cycling, completed five double-century cycling rides, and earned the prestigious California Triple Crown cycling award.

Treatments with stem cells -- which can grow into different types of cells -- are booming in the U.S., with an estimated 500 or more clinics in operation. Some clinics offer treatment for conditions ranging from autism to multiple sclerosis to erectile dysfunction, often without scientific evidence to support how well they work.

Treatment for knee arthritis is especially popular. Its one type of osteoarthritis, which afflicts 30 million Americans. Fees vary, but $2,000 per treatment for knee arthritis is about average. Insurance companies usually deny coverage, although in rare cases they may cover it when done alongwith another, established procedure.

Many doctors and scientists view the growth of stem cell treatments as very promising. But that growth comes as the FDA debates whether to tighten regulations on stem cell clinics after recent reports of patients suffering severe damage from treatment. The only stem cell-based product approved by the FDA is for umbilical cord blood-derived stem cells for blood cancers and other disorders.

In an editorial published March 16 in TheNew England Journal of Medicine, FDA officials warned the lack of evidence for unapproved stem cell treatments is ''worrisome." The officials cited reports of serious side effects, including two people who became legally blind after receiving the treatment in their eyes for macular degeneration.

In another case, a patient who received stem cell injections after a stroke developed paralysis and needed radiation treatment.

The FDA also notes that stem cell treatments potentially have other safety concerns, such as causing tumors to grow. And because patients mayreceive the treatmentsoutside of formal research studies, it can bedifficult to track their side effects.

Doctors say that treating the kneehasless of a chance forcomplications. It is also the body part with perhaps the most research.

Still, even doctors who offer the treatment for arthritic knees say more study is needed.

"We don't have a lot of controlled trials yet," says Keith Bjork, MD, an orthopedist in Amarillo, TX, who has given stem cell treatments to about 500 patients with knee arthritis in the past 5 years. "Their results are the strongest evidence," he says.

The most common side effects are joint stiffness and pain at the injection site as well as swelling, according to the results of one study.

For knee injections, doctors often take stem cells from the patient's bone marrow, fat tissue, or blood. Doctors who do the treatments cite anecdotal evidence as validation that the treatments work.

Marc Darrow, MD, the Los Angeles physical medicine specialist who cares for Chung, says he has done thousands of stem cell treatments. He uses stem cells from the patient's own bone marrow, a process he says is simple and fast.

His patients pain often subsides after knee injections, he says. He also has had cases in which the ''before'' and ''after'' X-rays suggest an increase in cartilage, he says.

Harvey E. Smith, MD, an assistant professor of orthopedic surgery at the Hospital of the University of Pennsylvania, says its clear the treatment has an effect. What is not as clear is how it lessens pain. Researchers are studying whether the stem cells themselves cut inflammation or if they release substances that affect other cells. They also are looking at whether the treatments can regenerate worn-out cartilage.

Published studies have produced mixed results. One from 2014 showed that stem cell injections given aftersurgery to remove torn knee cartilage showed evidence of cartilage regeneration and lessened pain. In March, researchers who reviewed the findings of six studies on stem cells for knee arthritis found that patients reported good results with no serious side effects. More data is needed, however, before researchers can recommend it.

''There is still not enough evidence to suggest this should be routine treatment for knee early osteoarthritis," says Wellington Hsu, MD, the Clifford C. Raisbeck professor of orthopedic surgery at Northwestern University Feinberg School of Medicine. Even so, he says, ''there is very little damage you are going to do with an injection to the knee. I think stem cells appear to be safe in orthopedic applications."

There is, of course, the risk that an investment of a couple thousand dollars will do nothing. But Hsu says that ''you are not going to find the catastrophic cases that will shut down a clinic [as may occur for other body parts].''

For people who have knee arthritis, the most invasive treatment is total knee replacement, Hsu says. Doctors are also testing other injectable therapies, including platelet-rich plasma, hyaluronic acid, and steroids, he says.

Consumers who decide to try stem cell treatments for achy knees should research their doctor and the specifics on the stem cell treatment. It's crucial to ask the clinic where the stem cells come from, Smith says. Ask if they will retrieve them from your own bone marrow or fat tissue, or if they will come from donors. The FDA requires donor cells and tissues to be tested for communicable diseases. There is no consensus on which source is best, but most doctors use stem cells from fat, Hsu says.

The FDA suggests patients who decide to get stem cells for any purpose should speak to their doctor about the potential risks and benefits, and ask whether they are part of an FDA-approved clinical trial. Most often, doctors who offer stem cell treatments are orthopedists, plastic surgeons, or physical medicine and rehabilitation doctors,

The reduction in pain, however, isnt permanent, Smith says. "The effect may last 6 months," he says, citing results from knee studies. When people are paying out of pocket, he adds, they may over-report good effects to feel like they got their money's worth.

Chung, the skier-cyclist, says the investment has been worth it. He plans to continue his injections once or twice a year, as needed, so he can stay active on the bike and the slopes.

SOURCES:

Wellington Hsu, MD, Clifford C. Raisbeck professor of orthopedic surgery, Feinberg Northwestern University School of Medicine, Chicago.

Harvey E. Smith, MD, assistant professor of orthopedic surgery, University of Pennsylvania, Philadelphia.

Keith Bjork, MD, orthopedic surgeon, Amarillo, TX; clinical advisory staff member, Amnio Technology.

Julian Cameron, MD, orthopedic surgeon, Tamarac, FL.

Marc Darrow, MD, Los Angeles physical medicine specialist.

George Chung, stem cell recipient, Los Angeles.

CDC: "Osteoarthritis Fact Sheet."

The Journal of Bone and Joint Surgery: "Adult Human Mesenchymal Stem Cells Delivered via Intra-Articular Injection to the Knee Following Partial Medial Meniscectomy."

The New England Journal of Medicine: "Clarifying Stem-Cell Therapy's Benefits and Risks."

American Academy of Orthopaedic Surgeons annual meeting, presentation: ''Platelet-Rich Plasma, Bone Morphogenetic Protein, and Stem Cells: What Surgeons Need to Know." March 14, 2017, San Diego.

International Society for Stem Cell Research. "Stem Cell Facts."

Andrea Fischer, FDA spokeswoman.

FDA: "Consumer Information on Stem Cells."

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Stem Cells for Knees: Promising Treatment or Hoax? - WebMD

Scientists report in a paper (HGFA Is an Injury-Regulated Systemic Factor that Induces the Transition of Stem Cells into GAlert) in Cell Reports about a new approach to speed recoveryfrom a wide variety of injuries.

Our research shows that by priming the body before an injury, you can speed the process of tissue repair and recovery, similar to how a vaccine prepares the body to a fight infection, said lead authorJoseph T. Rodgers, Ph.D., who began the research during his postdoctoral studies at the Stanford University School of Medicine. He has continued his work in his current position as an assistant professor of stem cell biology and regenerative medicine at The University of Southern California.

This recent study builds upon Dr. Rodgers previous finding: When one part of the body suffers an injury, adult stem cells in uninjured areas throughout the body enter a primed or Alert state. Alert stem cells have an enhanced potential to repair tissue damage.In this new study, he identified a signal that alerts stem cells and showed how it could serve as a therapy to improve healing.

Searching for a signal that could alert stem cells, Dr. Rodgers and colleagues focused their attention on the blood. They injected blood from an injured mouse into an uninjured mouse. In the uninjured mouse, this caused stem cells to adopt an Alert state. The teamidentified the critical signal in blood that alerted stem cellsan enzyme called hepatocyte growth factor activator (HGFA). In normal conditions, HGFA is abundant in the blood, but inactive. Injury activates HGFA, so HGFA signaling can alert stem cells to be ready to heal.

Leveraging this discovery, Dr. Rodgers' group asked the question: What happens if HGFA alerts stem cells before an injury occurs? Does this improve the repair response? They injected active HGFA into mice that received either a muscle or skin injury a couple of days later. The mice healed faster, began running on their wheels sooner, and even regrew their fur better than mice that did not receive the HGFA booster.

These findings indicate that HGFA can alert many different types of stem cells, rousing them from their normal resting or quiescent state and preparing them to respond quickly and efficiently to injury, according to Dr. Rodgers.

We believe this could be a therapeutic approach to improve recovery in situations where injuries can be anticipated, he said, such as surgery, combat, or sports.

This therapeutic approach could prove particularly useful for people with impaired healing, such as older adults or diabetics.

This work shows that there are factors in the blood that control our ability heal, continued Dr. Rodgers. "We are looking at how HGFA might explain declines in healing, and how we can use HGFA to restore normal healing.

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Stem Cells Primed to Be on Alert to Repair Tissue Damage - Genetic Engineering & Biotechnology News

Scientists at the University of California San Francisco have discovered a new function of lungs: They make blood which leads to a new wellspring of stem cells as well.

The astonishing breakthrough comes courtesy of refinement to microscopic video imaging that allows researchers to probe individual cells within blood vessels of a living host's lungs in this case, mice lungs.

The findings have far-reaching implications for human study: Researchers were surprised to find that not only did the lungs produce more blood cells, they did so in volumes that indicated more than half of all platelets in circulation critical for clotting are produced by the lungs.

The significance for the blood stem cells also was compelling. The newly discovered pool of stem cells is capable of restoring blood production when bone marrow stem cells are depleted. This could lead to novel approaches to treating leukemia, a cancer of white blood cells that crowds out red blood cells, and bone cancer, which destroys the body's ability to manufacture red blood cells.

This finding definitely suggests a more sophisticated view of the lungs that theyre not just for respiration but also a key partner in formation of crucial aspects of the blood, said pulmonologist Mark R. Looney, a professor of medicine and of laboratory medicine at the University of California, and the research's senior author. What weve observed here in mice strongly suggests the lung may play a key role in blood formation in humans as well. The report was published online at Nature.com.

The new imaging approach allowed scientists to examine interactions between the immune system and platelets in the lungs. While following the interactions, they discovered a surprisingly large population of cells that produce platelets called megakaryocytes. Though these cells were observed in the lungs previously it was generally though that they exist primarily in bone marrow.

Researchers were baffled and more detailed imaging followed. Once they zeroed in on these cells, they soon realized that they not only took up residence in the lungs, they also were producing 10 million platelets per hour there evidence that more than half of platelet production actually occurs in the lungs (in the mice models).

To be able to track blood stem cells and blood production, researchers transplanted donor lungs to mice with fluorescent-dye-tinted megakaryocytes. They followed the fluorescent cells as they traveled to the new lungs.

In another experiment, scientists wanted to determine if lungs that already had these platelet producers imbedded would spur platelet production in mice with low platelet counts, so they transplanted lungs with fluorescent-tinted megakaryocytes into mice predetermined to have low platelet counts. The transplanted lungs quickly sprung into action and restored normal platelet levels.

In yet another experiment, researchers transplanted healthy lungs with all cells fluorescently tinted into mice without bone marrow blood stem cells. The fluorescent marker cells quickly traveled to the damaged bone marrow and began production of myriad cells including T cells, which are key immune cells.

The exact mechanism behind the bone marrow-lung blood production is not yet known. Its possible that the lung is an ideal bioreactor for platelet production because of the mechanical force of the blood, or perhaps because of some molecular signaling we dont yet know about, said Guadalupe Ortiz-Muoz, a postdoctoral researcher and the researchs co-author. But more research is sure to follow.

Now medical scientists and researchers can zero in on proving in human models that blood components stem cells key among them travel more freely than previously though, which could lead ultimately to advances in treatment options for various blood disorders.

2017 NewsmaxHealth. All rights reserved.

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Surprise - Lungs Make Blood, Too - Newsmax

UCLA researchers have created a new system to produce human T cells, the white blood cells that fight against disease-causing intruders in the body. The system could be utilized to engineer T cells to find and attack cancer cells, which means it could be an important step toward generating a readily available supply of T cells for treating many different types of cancer.

The preclinical study, published in the journal Nature Methods, was led by senior authors Dr. Gay Crooks, a professor of pathology and laboratory medicine and of pediatrics and co-director of theEli and Edythe Broad Center of Regenerative Medicine and Stem Cell Research at UCLA, and Amelie Montel-Hagen, an associate project scientist in Crooks lab.

The thymus sits in the front of the heart and plays a central role in the immune system. It uses blood stem cells to make T cells, which help the body fight infections and have the ability to eliminate cancer cells. However, as people age or become ill, the thymus isnt as efficient at making T cells.

T cells generated in the thymus acquire specialized molecules, called receptors, on their surface, and those receptors help T cells seek out and destroy virus-infected cells or cancer cells. Leveraging that process has emerged as a promising area of cancer research: Scientists have found that arming large numbers of T cells with specific cancer-finding receptors a method known as adoptive T cell immunotherapy has shown remarkable results in clinical trials.

Adoptive T cell immunotherapy typically involves collecting T cells from people who have cancer, engineering them in the lab with a cancer-finding receptor and transfusing the cells back into the patient.

However, adoptive T cell immunotherapy treatments can be time-consuming, and people with cancer might not have enough T cells for the approach to work, according to Dr. Christopher Seet, the studys first author and a clinical instructor who treats cancer patients in the division of hematology-oncology at UCLA.

Since adoptive T cell immunotherapy was first used clinically in 2006, scientists have recognized that it would be more efficient to create a readily available supply of T cells from donated blood cells or from pluripotent stem cells, which can create any cell type in the body. The challenge with that strategy would be that T cells created using this approach would carry receptors that are not matched to each individual patient, which could ultimately cause the patients body to reject the transplanted cells or could cause the T cells to target healthy tissue in addition to cancer cells.

We know that the key to creating a consistent and safe supply of cancer-fighting T cells would be to control the process in a way that deactivates all T cell receptors in the transplanted cells, except for the cancer-fighting receptors, Crooks said.

The UCLA team used a new combination of ingredients to create structures called artificial thymic organoids that, like the thymus, have the ability to produce T cells from blood stem cells. The scientists found that mature T cells created in the artificial thymic organoids carried a diverse range of T cell receptors and worked similarly to the T cells that a normal thymus produces.

Next, the team tested whether artificial thymic organoids could produce the specialized T cells with cancer-fighting T cell receptors. When they inserted a gene that delivers a cancer-fighting receptor to the blood stem cells, they found that the thymic organoids produced large numbers of cancer-specific T cells, and that all other T cell receptors were turned off. The results suggest that the cells could potentially be used to fight cancer without the risk of T cells attacking healthy tissue.

Montel-Hagen said the artificial thymic organoid can easily be reproduced by other scientists who study T cell development. The UCLA researchers now are looking into using the system with pluripotent stem cells, which could produce a consistent supply of cancer-fighting T cells for patients in need of immediate lifesaving treatment.

Kite Pharma holds a license to the artificial thymic organoid method, which is patented by the Regents of the University of California.

The research was supported by the National Institutes of Health (R01 AG049753, 1R21AI119927, P01 HL073104 and T32HL066992), Tower Cancer Research Foundation Career Development Award, the Prostate Cancer Foundation Challenge Award (15CHAL02) and a Jane Coffin Childs Postdoctoral Fellowship, as well as an Innovation Award and a Clinical Fellowship from the UCLA Broad Stem Cell Research Center.

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UCLA develops artificial thymus that produces cancer-fighting cells - University of California

Sunday 4:00 pm - 8:00 pm Arrival and Check-in 6:00 pm Dinner 7:30 pm - 7:40 pm Welcome / Introductory Comments by GRC Site Staff 7:40 pm - 9:30 pm Keynote Session: Cancer Initiation Discussion Leader: Allison Bardin (Genetics and Developmental Biology Unit (INSERM U934), Institut Curie, France) 7:40 pm - 7:50 pm Opening Remarks 7:50 pm - 8:25 pm Ronald Depinho (University of Texas MD Anderson Cancer Center, USA) "Targeting Cancer Specific Vulnerabilities" 8:25 pm - 8:40 pm Discussion 8:40 pm - 9:15 pm Hans Clevers (Hubrecht Institute, The Netherlands) "Lgr5 Stem Cell-Grown Organoids and Their Applications" 9:15 pm - 9:30 pm Discussion Monday 7:30 am - 8:30 am Breakfast 9:00 am - 12:30 pm Stem Cell Biology Discussion Leader: Sean Morrison (University of Texas Southwestern Medical Center, USA) 9:00 am - 9:30 am Hans-Reimer Rodewald (German Cancer Research Center (DKFZ), Germany) "Resolving Hematopoiesis by Endogenous Barcoding" 9:30 am - 9:40 am Discussion 9:40 am - 10:10 am David Traver (University of California, San Diego, USA) "Development of Hematopoietic Stem Cells" 10:10 am - 10:20 am Discussion 10:20 am - 10:50 am Coffee Break 10:50 am - 11:20 am Leanne Jones (University of California, Los Angeles, USA) "Evolution of Stem Cell-Niche Interactions During Aging" 11:20 am - 11:30 am Discussion 11:30 am - 12:00 pm Helen Blau (Stanford University, USA) "Rejuvenation of Muscle Stem Cell Function" 12:00 pm - 12:10 pm Discussion 12:10 pm - 12:25 pm Selected from Poster Abstracts: John Schell (University of Utah, USA) "Limiting Mitochondrial Pyruvate Entry Alters Intestinal Stem Cell Maintenance and Proliferation" 12:25 pm - 12:30 pm Discussion 12:30 pm Lunch 1:30 pm - 4:30 pm Free Time 4:30 pm - 6:00 pm Poster Session 6:00 pm - 8:00 pm Genome Integrity Discussion Leader: Emmanuelle Passegue (Columbia University Medical Center, USA) 6:00 pm - 6:30 pm Andre Nussenzweig (National Cancer Institute, NIH, USA) "Endogenous and Exogenous DNA Breaks" 6:30 pm - 6:40 pm Discussion 6:40 pm - 7:10 pm Allison Bardin (Genetics and Developmental Biology Unit (INSERM U934), Institut Curie, France) "Spontaneous Somatic Stem Cell Mutations" 7:10 pm - 7:20 pm Discussion 7:20 pm - 7:50 pm Bjoern Schmacher (University of Cologne, Germany) "Non-Cell-Autonomous Regulation of the C. elegans p53 Response in Primordial Germ Cells" 7:50 pm - 8:00 pm Discussion 8:00 pm Dinner Tuesday 7:30 am - 8:30 am Breakfast 8:30 am Group Photo 9:00 am - 12:30 pm Epigenome Discussion Leader: Andre Nussenzweig (National Cancer Institute, NIH, USA) 9:00 am - 9:30 am Maarten Van Lohuizen (Netherlands Cancer Institute, The Netherlands) "Role of Polycomb Repressor Complex 2 in NSCLC: Consequences for Epigenetic Therapy" 9:30 am - 9:40 am Discussion 9:40 am - 10:10 am Anne Brunet (Stanford University, USA) "Mechanisms of Neural Stem Cell Aging" 10:10 am - 10:20 am Discussion 10:20 am - 10:50 am Coffee Break 10:50 am - 11:20 am Elaine Fuchs (Rockefeller University, USA) "Skin Stem Cells and Cancer: From Signaling to Chromatin Landscapes" 11:20 am - 11:30 am Discussion 11:30 am - 12:00 pm Thomas Rando (Stanford University, USA) "Epigenetic Determinants of Stem Cell Aging and Rejuvenation" 12:00 pm - 12:10 pm Discussion 12:10 pm - 12:25 pm Selected from Poster Abstracts: Francesco Neri (Leibniz Institute on Aging - Fritz Lipmann Institute (FLI), Germany) "Dnmt3b-Dependent Intragenic DNA Methylation Prevents RNA Polymerase II Spurious Entry on Gene Bodies and Cryptic Transcription Initiations" 12:25 pm - 12:30 pm Discussion 12:30 pm Lunch 1:30 pm - 4:30 pm Free Time 4:30 pm - 6:00 pm Poster Session 6:00 pm - 8:00 pm Stem Cell Niche and Environment Discussion Leader: Leanne Jones (University of California, Los Angeles, USA) 6:00 pm - 6:30 pm Valentina Greco (Yale University, USA) "Capturing the Interface Between Homeostasis and Cancer by Live Imaging" 6:30 pm - 6:40 pm Discussion 6:40 pm - 7:10 pm Sean Morrison (University of Texas Southwestern Medical Center, USA) "Metabolic Mechanisms Regulating Cancer Initiation" 7:10 pm - 7:20 pm Discussion 7:20 pm - 7:50 pm Amy Wagers (Harvard University, USA) "Muscle Stem Cells and Metabolism in Aging, Regeneration and Tumorigenesis" 7:50 pm - 8:00 pm Discussion 8:00 pm Dinner Wednesday 7:30 am - 8:30 am Breakfast 9:00 am - 12:30 pm Clonal Selection Discussion Leader: Andreas Trumpp (German Cancer Research Center / HI-STEM, Germany) 9:00 am - 9:30 am Catriona Jamieson (University of California, San Diego, USA) "The Role of RNA Processing Deregulation in Malignant Reprogramming" 9:30 am - 9:40 am Discussion 9:40 am - 10:10 am Douglas Winton (Cancer Research UK Cambridge Institute, United Kingdom) "Fate and Fidelity of Stem Cells in the Intestinal Epithelium" 10:10 am - 10:20 am Discussion 10:20 am - 10:50 am Coffee Break 10:50 am - 11:20 am Irving Weissman (Stanford University School of Medicine, USA) "Normal and Neoplastic Stem Cell Competitions" 11:20 am - 11:30 am Discussion 11:30 am - 12:00 pm Cedric Blanpain (Universite Libre de Bruxelles, Belgium) "Reprograming Stem Cells During Cancer Initiation" 12:00 pm - 12:10 pm Discussion 12:10 pm - 12:25 pm Selected from Poster Abstracts: Olivier Cinquin (University of California, Irvine, USA) "Quantification of In Vivo Progenitor Mutation Accrual with Ultra-Low Error Rate and Minimal Input DNA Using SIP-HAVA-seq" 12:25 pm - 12:30 pm Discussion 12:30 pm Lunch 1:30 pm - 4:30 pm Free Time 4:30 pm - 6:00 pm Poster Session 6:00 pm - 8:00 pm Stem Cell Metabolism and Signaling Discussion Leader: Helen Blau (Stanford University, USA) 6:00 pm - 6:30 pm Emmanuelle Passegue (Columbia University Medical Center, USA) "HSC, Metabolism and Fate Decisions" 6:30 pm - 6:40 pm Discussion 6:40 pm - 7:10 pm David Sabatini (Whitehead Institute for Biomedical Research, Massachusetts Institute of Technology, USA) "Regulation of Growth by the mTOR Pathway" 7:10 pm - 7:20 pm Discussion 7:20 pm - 7:50 pm Andreas Trumpp (German Cancer Research Center / HI-STEM, Germany) "Quiescence and Dormancy in Normal and Malignant Stem Cells" 7:50 pm - 8:00 pm Discussion 8:00 pm Dinner Thursday 7:30 am - 8:30 am Breakfast 8:30 am - 9:00 am Business Meeting Nominations for the Next Vice Chair; Fill in Conference Evaluation Forms; Discuss Future Site and Scheduling Preferences; Election of the Next Vice Chair 9:00 am - 12:30 pm Plasticity and Heterogeneity Discussion Leader: Maarten Van Lohuizen (Netherlands Cancer Institute, The Netherlands) 9:00 am - 9:30 am Fred De Sauvage (Genentech, USA) "Targeting Intestinal Stem Cells in Cancer" 9:30 am - 9:40 am Discussion 9:40 am - 10:10 am Tannishtha Reya (University of California, San Diego, USA) "Stem Cell Signals in Cancer Heterogeneity and Therapy Resistance" 10:10 am - 10:20 am Discussion 10:20 am - 10:50 am Coffee Break 10:50 am - 11:20 am Christopher Heeschen (Barts Cancer Institute, Queen Mary University of London, United Kingdom) "Novel Precision Medicine Approaches for Pancreatic Cancer Stem Cells" 11:20 am - 11:30 am Discussion 11:30 am - 12:00 pm Henri Jasper (Buck Institute for Research on Aging, USA) "Control of Intestinal Stem Cell Activity: Lessons from Drosophila" 12:00 pm - 12:10 pm Discussion 12:10 pm - 12:25 pm Selected from Poster Abstracts: Jagdeep Nanchahal (University of Oxford, United Kingdom) "HMGB1 Accelerates Regeneration of Multiple Tissues by Transitioning Stem Cells to G(Alert)" 12:25 pm - 12:30 pm Discussion 12:30 pm Lunch 1:30 pm - 4:30 pm Free Time 4:30 pm - 6:00 pm Poster Session 6:00 pm - 8:00 pm Keynote Session: Developmental Pathways Discussion Leader: Hans-Reimer Rodewald (German Cancer Research Center (DKFZ), Germany) 6:00 pm - 6:15 pm Selected from Poster Abstracts: Ana Carolina Dantas Machado (University of Southern California, USA) "Mechanisms of Protein-DNA Recognition Provide Insights into Gene Regulation" 6:15 pm - 6:20 pm Discussion 6:20 pm - 7:00 pm Margaret Goodell (Baylor College of Medicine, USA) "Aging HSCs and Epigenetic Modulation" 7:00 pm - 7:10 pm Discussion 7:10 pm - 7:50 pm Manuel Serrano (Centro Nacional de Investigaciones Oncologicas, Spain) "Tissue Repair: An Integrative View of Senescence and Reprogramming" 7:50 pm - 8:00 pm Discussion 8:00 pm Dinner Friday 7:30 am - 8:30 am Breakfast 9:00 am Departure

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Gordon Research Conferences - 2017 Meeting - Stem Cells ...

ROCKLEDGE, Fla. Gracie Gregory smiles beneath her brilliant blue eyes. Shes sitting on her mothers lap, next to her older sister, Ryleigh, who boasts about Gracie being very sweet and kind.

It wasnt always so. Just a couple years ago, Ryleigh, 11, was scared of her sister when shed throw tantrums and screaming fits.

She wouldve fought and kicked, Ryleigh says, noting that it wouldnt have been possible to sit like this next to Gracie.

Why was she scared of her sister?

Because of the kicking.

Gracie, 7, interrupts: I dont even remember it.

We do, says her mother, Gina Gregory.

Gracie has autism, a condition that affected nearly every aspect of her familys life after she was diagnosed at 2. But a new study is offering hope for the Gregorys and families like them.

Gracie was one of 25 children who took part in the first-of-its-kind study at Duke University in Durham, North Carolina. The goal: to see whether a transfusion of their own umbilical cord blood containing rare stem cells could help treat their autism.

The results were impressive: More than two-thirds of the children showed reported improvements. A larger second trial is underway, one its researchers hope will lead to long-term treatment for children with autism.

Skeptics say there are too many unanswered questions to get excited. Even Duke researchers acknowledge as much. The initial trial, published Wednesday in the journal Stem Cells Translational Medicine, was a safety study, not a controlled, double-blind study with definitive proof of positive results. This study was open-label, meaning everyone the doctors and the families knew that the therapy was being administered.

But for the Gregorys, the change in their daughter has been monumental.

Gone are the days of Gracie throwing fits in long lines at Disney World or during dinner at restaurants. When a tantrum intruded on family outings, her mom and dad wished they had T-shirts that said My kid has autism to ward off judgmental stares.

During autism therapy sessions, Gracie would kick, scream, spit and hit at her occupational therapist. It was horrible to try to get her to sit there, her mother says.

Even just brushing her teeth or combing her hair could set her off.

Gracie, then 5, was on the mild to moderate autism scale, but her parents say the disorder consumed about 75% of their daily routine. After her participation in the study, that figure has been reduced to a mere 10%.

On a scale of 1 to 10, they rate her improvement around an 8 or 9; its been that dramatic. Shes even begun attending a regular school and thriving there, something her parents never thought possible. Shed been in various specialized school programs, and nothing was the proper fit.

Are Gracies changes a result of the cord blood transfusion stimulating her brain? Or did her brain just mature as she got older? Could it be that her parents were subconsciously determined to magnify her improvements, given all their family had been through?

Those are questions the Gregorys still ask. But they do know that their daughters transformation appeared to begin about six months after her transfusion in January 2015 and has continued ever since.

Her fathers favorite adjustment is her newfound affection. Instead of shunning hugs, she now welcomes an embrace.

We will say we dont think its cured her. You still see some of the small idiosyncrasies that she does have, says her father, Wade Gregory. But again, I think its supercharged her learning curve. Its pushed her to do things she normally wouldnt do.

Her mother adds, She got better, and were just thankful for that whether it be the stem cells or not. Were just thankful for what changes have happened.

Billions of cells

Dr. Joanne Kurtzberg shows off a freezer deep inside the bowels of the Carolinas Cord Blood Bank at Duke University Medical Center. Known as a thermogenesis freezer, it stores up to 3,640 units of cord blood left over from babies umbilical cords and placenta at minus 196 degrees Celsius.

Each unit is designated by labels with specially designed adhesive to withstand extremely cold temperatures for decades. There are 14 cord blood freezers in all.

It is the cord blood in those freezers stored or donated by parents in case a serious illness develops thats at the cutting edge of this research.

Kurtzberg, who heads the Robertson Clinical and Translational Cell Therapy Program, has teamed up with Dr. Geraldine Dawson, director of the Duke Center for Autism and Brain Development.

Both saw a great need for medical advances to help treat children with autism. An estimated one in every 68 children in America has some form of autism spectrum disorder, according to the Centers for Disease Control and Prevention.

About 30% never learn to speak, and many children even with early behavioral interventions still struggle to adapt. There also are no FDA-approved medications that improve the core symptoms of autism.

I was very interested in collaborating with people here at Duke who could offer medical approaches that could enhance neuroplasticity, or the brains ability to respond to treatment, Dawson says.

Thats where Kurtzberg comes in. Over the past two decades, she had seen children with inherited metabolic disorders be treated with cord blood after receiving high doses of chemotherapy.

Weve been able to show that with some of these diseases, a cord transplant rescues them from death and also improves their neurologic outcome, she says.

She began wondering: Could cord blood help other children?

About a decade ago, her laboratory began clinical tests of children with cerebral palsy whose parents had banked their cord blood. Again, they saw positive results. And in some of those children who had autistic tendencies, they saw autistic symptoms improve. Another spark went off: What if they tested cord blood specifically for autism?

The safety trial began a little over a year and a half ago. Not only did it find cord blood to be safe, but 70% of the 25 children, age 2 to 6, had behavioral improvements as described by their parents and tracked by the Duke researchers. The research is largely funded by a $40 million donation from the Marcus Foundation, a nonprofit created by Home Depot co-founder Bernie Marcus.

The children traveled to Duke three times over the course of a year. They underwent a series of evaluations such as autism assessments, MRIs and EEGs to track their brain activity. On the first trip, the children received the cord blood infusion along with the intense evaluations. Each child received 1 billion to 2 billion cells, given through an IV in their arms or legs. At six months and then a year later, the children returned for more tests and observations.

Some children, who were not speaking very much, had big increases in their vocabulary and their functional speech, Kurtzberg says. Many children were able to attend to play and have meaningful communication in a way that they werent before. Some children had less repetitive behaviors than they did when they came onto the study.

Adds Dawson, The study was very encouraging. We did see positive results. However, it did not have a comparison group, which is very important in establishing whether a treatment is actually effective.

Both researchers cant stress that enough: that although theyre cautiously optimistic about the results, they want the science to play out. They are now in the midst of the definitive trial on whether cord blood can treat autism a double-blind, placebo-controlled trial involving 165 autistic children, ranging in age from 2 to 8. The FDA has oversight of the study.

During the phase II study, the children on their first visit receive a cord blood infusion either their own or from a donor or they get a placebo. They also undergo a battery of assessment tests and brain monitoring.

On their second visit six months later, the children receive a second infusion with whatever preparation they did not receive the first time and undergo more evaluations. The order of the infusions is not known. Researchers will monitor them for the next year for any sign of behavioral improvements.

Its known as a crossover trial, in which each subject gets the treatment and the placebo but in a different order. Researchers say it would have been nearly impossible to find participants if parents knew that their children might not receive an infusion.

How groundbreaking would it be if the trial shows similar results to the safety study?

If we can show that receiving an infusion of cord blood is more effective for improving social behavior than the placebo, Dawson says, then this will be game-changing.

Kurtzberg adds, Well be extraordinarily encouraged if the second trial shows that the cells benefit children when the placebo does not. We will consider that a breakthrough.

Both researchers were shaped early in life by the struggles families face raising autistic children. As a teen, Dawson babysat twins with autism who lived across the street. It was just an inspiration to devote my career to improving the lives of people with autism, she says.

Kurtzberg was similarly affected. When she was a junior in college, she would visit a girl with severe autism and play with her as a means of behavioral intervention. The family still writes to me, she says.

It is for this reason their longtime devotion to families raising children with autism that both issue a heap of caution. Although theyre excited about the results of the first study, Kurtzberg says, we dont want to mislead people and claim its working before we have definitive proof.

Adds Dawson, Its important for parents who might hear about cord blood as a potential treatment for autism to know that we are working very hard to know the answer to that question. We arent there yet.

Cautious optimism

Kurtzberg has a hypothesis about what may be happening: that certain immune cells within the cord blood are crossing the blood-brain barrier and altering brain connectivity while also suppressing inflammation, which may exist with autism.

I feel more confident now because of our (cerebral palsy) study, which preceded this study and does show benefits, Kurtzberg says.

Dr. Arnold R. Kriegstein, director of the stem cell center at the University of California, San Francisco, says that he hopes there will be breakthrough treatment for children with autism but that much more needs to be known before this will become a reality.

One has to be very careful when interpreting results that havent come from properly controlled, double-blind studies, he said. All I can say is that it would be wonderful if this treatment was effective, but one has to be very cautious before reaching any conclusions.

Even without a placebo effect, he says, many factors could have resulted in an improved outcome in the first study: The growing children could have acquired skills simply through maturation, possibly enhanced by occupational therapy, and their parents may have clung to positive gains, creating a biased outcome.

Thomas Frazier II, chief scientific officer of the advocacy group Autism Speaks, said the results of the initial study were encouraging but that more work needs to be done before the public gets excited. Its too early to get hopeful. Too early to change behavior, he said. I hope people dont go out and spend money on banking cord blood as a result [of this trial].

Kriegstein of UCSF also wonders whether cord blood is really stimulating cells in the brain and creating new connections. There are so many unanswered questions about what might be going on here, it becomes very difficult to evaluate the proposed mechanism, he said.

The question remains: How do these cells injected intravenously wind up in the brain, how do they target the appropriate brain regions, and what are they doing that could improve brain function?

I forgot how bad it was

An 8-year-old boy with autism sits at a table in a room within Dukes Center for Autism and Brain Development. Clinical research specialist Michelle Green watches from behind a two-way mirror. Two cameras in the room feed computer monitors, allowing her to further analyze his behavior.

Dr. Lauren Franz, a clinician, works with the boy in the room.

What kind of things make you feel threatened or anxious? she asks.

Like when Im done with a test, the boy says.

How does it feel when youre frightened or anxious? How does that feel?

Like pretty weird, he says.

The boy is participating in the second trial, and hes returned for his six-month assessment and second infusion. Researchers dont know which infusion he received first: the cord blood or the placebo.

But they track, record and monitor the slightest of details. Although it might seem like an innocuous conversation, researchers will compare the results with those of his first visit and any follow-ups. Was he able to sit still at the table before? Could he articulate his thoughts? Did he talk before the study? Has he improved?

At the Gregorys home in Florida, Gracies parents remember when she went through those same tests. The best investment they ever made, they say, was the $2,000 spent on banking her cord blood. At the time, it was just a precaution; her autism diagnosis didnt come until three months after her second birthday.

They know the desperation of families raising a child with autism of longing for their daughter to have a shot of normalcy in life. You cant quantify it. You cant measure it. You want to see your child succeed, her father says.

Mom and Dad recently watched old home videos, of Gracie singing inaudibly, of her covering her ears when Happy Birthday was sung for her third birthday, of showing no emotion on Christmas when she was 2. I forgot how bad it was, her mother says.

They hope the current study leads to similar successes and results in breakthrough treatment for autistic children everywhere.

Excerpt from:
Stem Cells May Offer Hope for Autism, Study Finds - wnep.com

UCLA researchers have created a new system to produce human T cells, the white blood cells that fight against disease-causing intruders in the body. The system could be utilized to engineer T cells to find and attack cancer cells, which means it could be an important step toward generating a readily available supply of T cells for treating many different types of cancer.

The preclinical study, published in the journal Nature Methods, was led by senior authors Dr. Gay Crooks, a professor of pathology and laboratory medicine and of pediatrics and co-director of the Eli and Edythe Broad Center of Regenerative Medicine and Stem Cell Research at UCLA, and Amelie Montel-Hagen, an associate project scientist in Crooks lab.

The thymus sits in the front of the heart and plays a central role in the immune system. It uses blood stem cells to make T cells, which help the body fight infections and have the ability to eliminate cancer cells. However, as people age or become ill, the thymus isnt as efficient at making T cells.

T cells generated in the thymus acquire specialized molecules, called receptors, on their surface, and those receptors help T cells seek out and destroy virus-infected cells or cancer cells. Leveraging that process has emerged as a promising area of cancer research: Scientists have found that arming large numbers of T cells with specific cancer-finding receptors a method known as adoptive T cell immunotherapy has shown remarkable results in clinical trials.

Adoptive T cell immunotherapy typically involves collecting T cells from people who have cancer, engineering them in the lab with a cancer-finding receptor and transfusing the cells back into the patient.

However, adoptive T cell immunotherapy treatments can be time-consuming, and people with cancer might not have enough T cells for the approach to work, according to Dr. Christopher Seet, the studys first author and a clinical instructor who treats cancer patients in the division of hematology-oncology at UCLA.

Since adoptive T cell immunotherapy was first used clinically in 2006, scientists have recognized that it would be more efficient to create a readily available supply of T cells from donated blood cells or from pluripotent stem cells, which can create any cell type in the body. The challenge with that strategy would be that T cells created using this approach would carry receptors that are not matched to each individual patient, which could ultimately cause the patients body to reject the transplanted cells or could cause the T cells to target healthy tissue in addition to cancer cells.

We know that the key to creating a consistent and safe supply of cancer-fighting T cells would be to control the process in a way that deactivates all T cell receptors in the transplanted cells, except for the cancer-fighting receptors, Crooks said.

The UCLA team used a new combination of ingredients to create structures called artificial thymic organoids that, like the thymus, have the ability to produce T cells from blood stem cells. The scientists found that mature T cells created in the artificial thymic organoids carried a diverse range of T cell receptors and worked similarly to the T cells that a normal thymus produces.

Next, the team tested whether artificial thymic organoids could produce the specialized T cells with cancer-fighting T cell receptors. When they inserted a gene that delivers a cancer-fighting receptor to the blood stem cells, they found that the thymic organoids produced large numbers of cancer-specific T cells, and that all other T cell receptors were turned off. The results suggest that the cells could potentially be used to fight cancer without the risk of T cells attacking healthy tissue.

Montel-Hagen said the artificial thymic organoid can easily be reproduced by other scientists who study T cell development. The UCLA researchers now are looking into using the system with pluripotent stem cells, which could produce a consistent supply of cancer-fighting T cells for patients in need of immediate life-saving treatment.

Kite Pharma holds a license to the artificial thymic organoid method, which is patented by the Regents of the University of California.

The research was supported by the National Institutes of Health (R01 AG049753, 1R21AI119927, P01 HL073104 and T32HL066992), Tower Cancer Research Foundation Career Development Award, the Prostate Cancer Foundation Challenge Award (15CHAL02) and a Jane Coffin Childs Postdoctoral Fellowship, as well as an Innovation Award and a Clinical Fellowship from the UCLA Broad Stem Cell Research Center.

Read more:
Artificial thymus developed at UCLA can produce cancer-fighting T cells from blood stem cells - UCLA Newsroom

Gracie Gregory smiles beneath her brilliant blue eyes. She's sitting on her mother's lap, next to her older sister, Ryleigh, who boasts about Gracie being "very sweet and kind."

It wasn't always so. Just a couple years ago, Ryleigh, 11, was scared of her sister when she'd throw tantrums and screaming fits.

"She would've fought and kicked," Ryleigh says, noting that it wouldn't have been possible to sit like this next to Gracie.

Why was she scared of her sister?

"Because of the kicking."

Gracie, 7, interrupts: "I don't even remember it."

"We do," says her mother, Gina Gregory.

Gracie has autism, a condition that affected nearly every aspect of her family's life after she was diagnosed at 2. But a new study is offering hope for the Gregorys and families like them.

Gracie was one of 25 children who took part in the first-of-its-kind study at Duke University in Durham, North Carolina. The goal: to see whether a transfusion of their own umbilical cord blood containing rare stem cells could help treat their autism.

The results were impressive: More than two-thirds of the children showed reported improvements. A larger second trial is underway, one its researchers hope will lead to long-term treatment for children with autism.

Skeptics say there are too many unanswered questions to get excited. Even Duke researchers acknowledge as much. The initial trial, published Wednesday in the journal Stem Cells Translational Medicine, was a safety study, not a controlled, double-blind study with definitive proof of positive results. This study was open-label, meaning everyone -- the doctors and the families -- knew that the therapy was being administered.

But for the Gregorys, the change in their daughter has been monumental.

Gone are the days of Gracie throwing fits in long lines at Disney World or during dinner at restaurants. When a tantrum intruded on family outings, her mom and dad wished they had T-shirts that said "My kid has autism" to ward off judgmental stares.

During autism therapy sessions, Gracie would kick, scream, spit and hit at her occupational therapist. "It was horrible to try to get her to sit there," her mother says.

Even just brushing her teeth or combing her hair could set her off.

Gracie, then 5, was on the mild to moderate autism scale, but her parents say the disorder consumed about 75% of their daily routine. After her participation in the study, that figure has been reduced to a mere 10%.

On a scale of 1 to 10, they rate her improvement around an 8 or 9; it's been that dramatic. She's even begun attending a "regular" school and thriving there, something her parents never thought possible. She'd been in various specialized school programs, and nothing was the proper fit.

Are Gracie's changes a result of the cord blood transfusion stimulating her brain? Or did her brain just mature as she got older? Could it be that her parents were subconsciously determined to magnify her improvements, given all their family had been through?

Those are questions the Gregorys still ask. But they do know that their daughter's transformation appeared to begin about six months after her transfusion in January 2015 and has continued ever since.

Her father's favorite adjustment is her newfound affection. Instead of shunning hugs, she now welcomes an embrace.

"We will say we don't think it's cured her. You still see some of the small idiosyncrasies that she does have," says her father, Wade Gregory. "But again, I think it's supercharged her learning curve. It's pushed her to do things she normally wouldn't do."

Her mother adds, "She got better, and we're just thankful for that -- whether it be the stem cells or not. We're just thankful for what changes have happened."

Billions of cells

Dr. Joanne Kurtzberg shows off a freezer deep inside the bowels of the Carolinas Cord Blood Bank at Duke University Medical Center. Known as a thermogenesis freezer, it stores up to 3,640 units of cord blood -- left over from babies' umbilical cords and placenta -- at minus 196 degrees Celsius.

Each unit is designated by labels with specially designed adhesive to withstand extremely cold temperatures for decades. There are 14 cord blood freezers in all.

It is the cord blood in those freezers -- stored or donated by parents in case a serious illness develops -- that's at the cutting edge of this research.

Kurtzberg, who heads the Robertson Clinical and Translational Cell Therapy Program, has teamed up with Dr. Geraldine Dawson, director of the Duke Center for Autism and Brain Development.

Both saw a great need for medical advances to help treat children with autism. An estimated one in every 68 children in America has some form of autism spectrum disorder, according to the Centers for Disease Control and Prevention.

About 30% never learn to speak, and many children even with early behavioral interventions still struggle to adapt. There also are no FDA-approved medications that improve the core symptoms of autism.

"I was very interested in collaborating with people here at Duke who could offer medical approaches that could enhance neuroplasticity, or the brain's ability to respond to treatment," Dawson says.

That's where Kurtzberg comes in. Over the past two decades, she had seen children with inherited metabolic disorders be treated with cord blood after receiving high doses of chemotherapy.

"We've been able to show that with some of these diseases, a cord transplant rescues them from death and also improves their neurologic outcome," she says.

She began wondering: Could cord blood help other children?

About a decade ago, her laboratory began clinical tests of children with cerebral palsy whose parents had banked their cord blood. Again, they saw positive results. And in some of those children who had autistic tendencies, they saw autistic symptoms improve. Another spark went off: What if they tested cord blood specifically for autism?

The safety trial began a little over a year and a half ago. Not only did it find cord blood to be safe, but 70% of the 25 children, age 2 to 6, had behavioral improvements as described by their parents and tracked by the Duke researchers. The research is largely funded by a $40 million donation from the Marcus Foundation, a nonprofit created by Home Depot co-founder Bernie Marcus.

The children traveled to Duke three times over the course of a year. They underwent a series of evaluations such as autism assessments, MRIs and EEGs to track their brain activity. On the first trip, the children received the cord blood infusion along with the intense evaluations. Each child received 1 billion to 2 billion cells, given through an IV in their arms or legs. At six months and then a year later, the children returned for more tests and observations.

"Some children, who were not speaking very much, had big increases in their vocabulary and their functional speech," Kurtzberg says. "Many children were able to attend to play and have meaningful communication in a way that they weren't before. Some children had less repetitive behaviors than they did when they came onto the study."

Adds Dawson, "The study was very encouraging. We did see positive results. However, it did not have a comparison group, which is very important in establishing whether a treatment is actually effective."

Both researchers can't stress that enough: that although they're cautiously optimistic about the results, they want the science to play out. They are now in the midst of the definitive trial on whether cord blood can treat autism -- a double-blind, placebo-controlled trial involving 165 autistic children, ranging in age from 2 to 8. The FDA has oversight of the study.

During the phase II study, the children on their first visit receive a cord blood infusion -- either their own or from a donor -- or they get a placebo. They also undergo a battery of assessment tests and brain monitoring.

On their second visit six months later, the children receive a second infusion with whatever preparation they did not receive the first time and undergo more evaluations. The order of the infusions is not known. Researchers will monitor them for the next year for any sign of behavioral improvements.

It's known as a crossover trial, in which each subject gets the treatment and the placebo but in a different order. Researchers say it would have been nearly impossible to find participants if parents knew that their children might not receive an infusion.

How groundbreaking would it be if the trial shows similar results to the safety study?

"If we can show that receiving an infusion of cord blood is more effective for improving social behavior than the placebo," Dawson says, "then this will be game-changing."

Kurtzberg adds, "We'll be extraordinarily encouraged if the second trial shows that the cells benefit children when the placebo does not. We will consider that a breakthrough."

Both researchers were shaped early in life by the struggles families face raising autistic children. As a teen, Dawson babysat twins with autism who lived across the street. "It was just an inspiration to devote my career to improving the lives of people with autism," she says.

Kurtzberg was similarly affected. When she was a junior in college, she would visit a girl with severe autism and play with her as a means of behavioral intervention. "The family still writes to me," she says.

It is for this reason -- their longtime devotion to families raising children with autism -- that both issue a heap of caution. Although they're excited about the results of the first study, Kurtzberg says, "we don't want to mislead people and claim it's working before we have definitive proof."

Adds Dawson, "It's important for parents who might hear about cord blood as a potential treatment for autism to know that we are working very hard to know the answer to that question. We aren't there yet."

Cautious optimism

Kurtzberg has a hypothesis about what may be happening: that certain immune cells within the cord blood are crossing the blood-brain barrier and altering brain connectivity while also suppressing inflammation, which may exist with autism.

"I feel more confident now because of our (cerebral palsy) study, which preceded this study and does show benefits," Kurtzberg says.

Dr. Arnold R. Kriegstein, director of the stem cell center at the University of California, San Francisco, says that he hopes there will be breakthrough treatment for children with autism but that much more needs to be known before this will become a reality.

"One has to be very careful when interpreting results that haven't come from properly controlled, double-blind studies," he said. "All I can say is that it would be wonderful if this treatment was effective, but one has to be very cautious before reaching any conclusions."

Even without a placebo effect, he says, many factors could have resulted in an improved outcome in the first study: The growing children could have acquired skills simply through maturation, possibly enhanced by occupational therapy, and their parents may have clung to positive gains, creating a biased outcome.

Thomas Frazier II, chief scientific officer of the advocacy group Autism Speaks, said the results of the initial study were encouraging but that more work needs to be done before the public gets excited. "It's too early to get hopeful. Too early to change behavior," he said. "I hope people don't go out and spend money on banking cord blood as a result [of this trial]."

Kriegstein of UCSF also wonders whether cord blood is really stimulating cells in the brain and creating new connections. "There are so many unanswered questions about what might be going on here, it becomes very difficult to evaluate the proposed mechanism," he said.

"The question remains: How do these cells injected intravenously wind up in the brain, how do they target the appropriate brain regions, and what are they doing that could improve brain function?"

'I forgot how bad it was'

An 8-year-old boy with autism sits at a table in a room within Duke's Center for Autism and Brain Development. Clinical research specialist Michelle Green watches from behind a two-way mirror. Two cameras in the room feed computer monitors, allowing her to further analyze his behavior.

Dr. Lauren Franz, a clinician, works with the boy in the room.

"What kind of things make you feel threatened or anxious?" she asks.

"Like when I'm done with a test," the boy says.

"How does it feel when you're frightened or anxious? How does that feel?"

"Like pretty weird," he says.

The boy is participating in the second trial, and he's returned for his six-month assessment and second infusion. Researchers don't know which infusion he received first: the cord blood or the placebo.

But they track, record and monitor the slightest of details. Although it might seem like an innocuous conversation, researchers will compare the results with those of his first visit and any follow-ups. Was he able to sit still at the table before? Could he articulate his thoughts? Did he talk before the study? Has he improved?

At the Gregorys' home in Florida, Gracie's parents remember when she went through those same tests. The best investment they ever made, they say, was the $2,000 spent on banking her cord blood. At the time, it was just a precaution; her autism diagnosis didn't come until three months after her second birthday.

They know the desperation of families raising a child with autism -- of longing for their daughter to have a shot of normalcy in life. "You can't quantify it. You can't measure it. You want to see your child succeed," her father says.

Mom and Dad recently watched old home videos, of Gracie singing inaudibly, of her covering her ears when "Happy Birthday" was sung for her third birthday, of showing no emotion on Christmas when she was 2. "I forgot how bad it was," her mother says.

They hope the current study leads to similar successes -- and results in breakthrough treatment for autistic children everywhere.

See original here:
Could stem cells offer hope for autism? - WTAE Pittsburgh

LONDON, April 4, 2017 /PRNewswire/ -- KEY FINDINGS

The global market for stem cell is anticipated to expand at a CAGR of 25.76% during the forecast period of 2017-2025. The rise in neurodegenerative diseases is the primary factor for the growth of the stem cell market.

Download the full report: https://www.reportbuyer.com/product/4807905/

MARKET INSIGHTS The global stem cell market is segmented on the basis of product, technology, application, and geography. The stem cell market of the product is segmented into adult stem cell, embryonic stem cell, induced pluripotent stem cell market and rat neutral stem cell market. The stem cell market of technology is segmented into stem cell acquisition market, stem cell production market, stem cell cryopreservation market and stem cells expansion and sub-culture market. the application of the stem cell market is segmented into stem cells regenerative medicine market and drug discovery and development market. The stem cell market geography is segmented into North America, Europe, Asia-Pacific and rest of the world. the upsurge in neurodegenerative ailments, growing investments in R&D, government subsidy and sustenance, advancements in the applications of stem cell, significant growth in medical tourism, swelling stem cell banking are the major drivers for the stem cell market.

REGIONAL INSIGHTS The Stem Cell market in North America is expected to hold the largest share by 2025. Increased investments in research and development activities for the stem cell market and the presence of popular pharmaceutical market have contributed to the growth of the US market in the North American region. Asia- Pacific is anticipated to grow at CAGR of 26.23%, the fastest growing region among others. The growth of Asia-Pacific region is primarily driven due to growing incidences of chronic lifestyle diseases and government supports and their initiatives. Europe has generated revenue of $13556 million in 2016 which is set to increase by 2025. The Europe stem cell analysis market is primarily driven by the rising prevalence of chronic disorders such as cancer and cardiovascular disorders.

COMPETITIVE INSIGHTS The market players in the stem cell market are Cytori therapeutics Inc., Fibrocell science, Cellartis AB (acquired by Takara holdings Inc.), Biotime Inc., GE Healthcare, Thermo fisher scientific Pvt Ltd, Stem cell technologies, Cellular dynamics international (holding company Fujifilm), Vericel corporation (Aastrom bioscience), Brainstorm cell therapeutics, California stem cell Inc. (Holding company Caladrius biosciences, Inc. ), Beckton Dickinson and company, Stryker corporation, Celgene corporation. Some major companies involved in stem cell are; GE Healthcare, Stem cell technology, Thermo Fisher, Becton, Corning and many others.

Download the full report: https://www.reportbuyer.com/product/4807905/

About Reportbuyer Reportbuyer is a leading industry intelligence solution that provides all market research reports from top publishers http://www.reportbuyer.com

For more information: Sarah Smith Research Advisor at Reportbuyer.com Email: query@reportbuyer.com Tel: +44 208 816 85 48 Website: http://www.reportbuyer.com

To view the original version on PR Newswire, visit:http://www.prnewswire.com/news-releases/global-stem-cell-market-forecast-2017-2025-300434766.html

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GLOBAL STEM CELL MARKET FORECAST 2017-2025 - PR Newswire (press release)

Policy | Facilities | Publishing | Events | People | Research | Funding | Announcement | Trend watch | Coming up

Brexit triggered The UK government made good on its promise to trigger Article 50 on 29March and formally began the process of leaving the European Union. In the run up, Stephen Metcalfe, chair of the House of Commons science select committee, released a report on 22March, produced with scientific and industry groups and research charities, setting out science priorities for the upcoming negotiations over the future relationship between Britain and the EU. These include providing certainty for non-UK EU scientists working in the United Kingdom and maintaining funding levels for research.

Military research The Science Council of Japan (SCJ) on 24 March called on Japans government to keep military research out of universities and institutions. The statement by the SCJ, an independent body that represents 830,000 Japanese scientists and acts as an advisory body to the cabinet, was prompted by recent efforts to involve Japans universities and research institutes in military research. The declaration refers directly to the Acquisition, Technology and Logistics Agency, set up in the defence ministry in 2015 to fund dual-use research. The statement reflects on Japans history of mobilizing science in military efforts a reference to the Second World War and says that scientists must maintain their freedom and autonomy.

GM impasse Crop companies seeking to grow genetically modified varieties of maize (corn) in the European Union were disappointed on 27 March. In a vote, the European Commission failed once more to gain the required majority from the EUs 28 member states to authorize cultivation of two new varieties of maize from Pioneer and Syngenta, and to renew authorization for Monsantos MON810 variety, the only GM crop so far approved in the EU. The European Food Safety Authority has deemed all three varieties safe. The outcome means that the Commission will now make the final decisions on the authorizations itself. But, exploiting 2015 rules that allow member states to opt out of cultivating individual GM crops, 19 have already stated they will ban cultivation of these varieties in all or part of their territories, whatever the Commission decides.

Environmental fears halt India detector The India-based Neutrino Observatory (INO), a facility to be built underground in the southern state of Tamil Nadu, must seek new environmental clearances. The long-delayed INO was finally given budgetary approval in 2015, and is to study masses and other properties of neutrinos. On 20March, the National Green Tribunal in Chennai deemed that the 15-billion rupee (US$220-million) project needs a higher standard of environmental clearance because of the sites proximity to a national park. The projects leaders must now make a new proposal and seek permission from the National Board for Wildlife. Environmentalists are concerned about potential effects on the biodiverse Western Ghats. In 2009, a proposed site was rejected because it was deemed too close to a tiger reserve and an elephant corridor.

Frans Lanting/National Geographic Creative

An aerial view of the Nilgiri Hills in Indias Western Ghats.

Open-access push Four California universities have formally committed themselves to the goal of making all scholarly publishing open access. On 20 March, the University of California (UC) Berkeley, UCDavis, UC San Francisco and California State University, Northridge, signed up to an international initiative called OA2020, launched by Germanys Max Planck Society in 2015. Its aim is to push publishers towards open-access business models. The universities are the first academic institutions in the United States to sign up to the initiatives expression of interest, now endorsed by 82 universities and scholarly organizations worldwide.

Preprint policy For the first time, researchers applying for grants from the US National Institutes of Health (NIH) will be allowed to cite their studies published on preprint servers, including draft manuscripts, in applications. The NIH said on 24 March that the change is intended to speed up the review process and enhance the rigour of researchers work. Open-access advocates have applauded the policy. But critics worry that grant reviewers will not be able to distinguish between peer-reviewed research and early data, and that the policy will promote hype of incomplete results.

Post-Brexit plans A survey of 201 Spanish scientists working in Britain finds that 30% of them have changed their plans because of Brexit. Another 43% are putting off decisions until the outcome of the negotiations that will determine Britains future relationship with the European Union, according to the survey. The poll, of members of the Society of Spanish Researchers in the United Kingdom, was carried out in late 2016. Access to European funds and immigration policy are among the main concerns of the researchers, half of whom do not qualify for permanent residency. An estimated 5,000Spaniards work at public and private UK research institutions.

Pipeline approved The government of US President Donald Trump issued a permit for the construction of the contentious KeystoneXL pipeline on 24March, reversing the decision of the previous administration to block the project. The pipeline would transport crude oil from tar sands in Canada to the Gulf of Mexico and is controversial in part because tar-sands oil is more polluting than conventional crude.

CRISPR patent The European Patent Office announced on 23March that it intends to grant the University of California a key patent on the gene-editing technique CRISPRCas9. The patent would cover the use of the technology in both prokaryotic cells, such as bacteria, and eukaryotic cells, including those of plants and animals potentially the most lucrative application of the technique. By contrast, in February the US Patent and Trademark Office decided that the universitys US patent application did not adequately specify applications of CRISPRCas9 in eukaryotes, and thus did not overlap with another patent from the Broad Institute in Cambridge, Massachusetts, that covers eukaryotic uses of the technology. The scope of the University of Californias European patent could still be narrowed in response to challenges by outside parties.

Moon mission China and Saudi Arabia have signed a memorandum of understanding to collaborate on Chinas Change 4 Moon mission. Change 4 will be the first landing on the far side of the Moon and could offer a peek at a suspected volcanic structure, the history of which has intrigued scientists. Chinas Change 3 mission landed a rover on the Moon in 2013, but the machine lost mobility, disappointing scientists who had hoped for a wide survey of the lunar surface. Saudi Arabia also has an active satellite-launching programme and is partnering with Russia on plans to build an international space station.

Jemal Countess/Getty Images for TIME

AI researchers Andrew Ng, a leading machine-learning researcher, announced on 22 March that he plans to leave the Chinese technology giant Baidu. Ng has been chief scientist at the firms Silicon Valley research lab since 2014, while retaining a role at Stanford University in California. Ng says that he plans to continue to work in artificial intelligence (AI), and hopes to shepherd in the important societal change it offers. The departure is one of two key staff losses at Baidu: on 23 March, Tencent, the Chinese firm behind the social-media application WeChat, announced that it had hired the head of Baidus Big Data Lab, Zhang Tong, to lead its growing AI research efforts.

iPS cell trial A Japanese man in his 60s has become the first person to receive cell transplants derived from another persons induced pluripotent stem (iPS) cells. A team led by Masayo Takahashi at the RIKEN Center for Developmental Biology in Kobe reprogrammed anonymous donor skin cells into retinal cells, and on 28March transplanted them into the retina of the man, who has the eye disease age-related macular degeneration. In a similar 2014 trial by the team the first to use iPS cells in humans a Japanese woman received iPS-derived retinal tissue created from her own skin cells. The latest surgery is expected to pave the way for more applications of iPS cell technology, which offers the versatility of embryonic stem cells with fewer ethical difficulties.

Canadas freeze Canadas budget on 22 March presented scientists with an unexpected freeze on their main basic-research funding streams. Prime Minister Justin Trudeaus government made good on its promises to emphasize innovation and to encourage links between industry and academia, promising to establish Innovation Canada, a platform to coordinate support for Canadian entrepreneurs. But Canadas three major research councils, for the natural, health and social sciences, were given no budget increases. The plan did set aside Can$2million (US$2.5million) to fund the post of chief government science adviser, a key campaign promise of Trudeaus Liberal party.

Gates open access The Bill & Melinda Gates Foundation in Seattle, Washington, will launch its own open-access publishing venture this year, it announced on 23March. The initiative, Gates Open Research, will be modelled on a service begun last year by the Wellcome Trust in London. The Gates Foundations platform is intended to accelerate the publication of articles and data from research funded by the charity. The foundation implemented a publishing policy in January that mandates that the research it funds must be open access as soon as it is published.

Standing up for science Nominations are invited for the John Maddox Prize, which rewards an individual in any country who has promoted sound science and evidence on a matter of public interest. The 2,000 prize puts emphasis on those who have faced difficulty or hostility for their efforts. It is awarded by Nature, the London-based charity Sense about Science and the Kohn Foundation. The deadline for nominations is 31 July.

Around 85% of Natures readers are plagued weekly by invitations from apparently bogus and potentially predatory journals, an online poll answered by more than 5,300 people suggests. Most (52%) reported receiving 15 nuisance invites in the week of the survey; 17% received 10 or more. The invites, usually by e-mail, often ask scientists to publish papers with the journals or to become editors. Predatory journals are those that charge fees to publish papers without providing expected publishing services.

116 AprilThe information age is the focus of the Edinburgh International Science Festival.

26 AprilThe American Chemical Society holds its spring national meeting in San Francisco, California.

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