FINDINGS

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

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

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

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

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

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

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

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

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

The research was published in the Journal of Clinical Investigation.

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

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Pioneering stem cell gene therapy cures infants with bubble baby disease - UCLA Newsroom

Bone Marrow Stem Cells Comments Off on Pioneering stem cell gene therapy cures infants with bubble baby disease – UCLA Newsroom | March 29th, 2017

In the United States alone, more than 400,000 lumbar discectomies and 500,000 spinal fusions are performed each year for symptoms related to lumbar disc degeneration. The ability to get these to heal without surgery has been a long-term goal of many patients and physicians alike. The Spine Center continues to be on the forefront of treatment options and is proud to offer stem cell therapy treatments for patients as part of our comprehensive non-operative treatment options.

Adult stem cells are divided into different categories. For example, the types of adult stem cells Dr. Theofilos uses to treat musculoskeletal issues are known as mesenchymal stem cells (MSCs). These are multi-potent cells that can differentiate into bone cells, cartilage cells, or fat cells.

The human body has multiple storage sites for stem cells to repair degenerated and injured structures. Dr. Theofilos has found that obtaining stem cells from the hip bone (iliac bone) is easily performed within minutes and, in most cases, is a fairly painless procedure for the patient. The stem cells are obtained from bone marrow; just minutes later, they are used for treatment.

This procedure is done in our office and after the procedure, the syringe of stem cells is taken to the lab and placed in a specialized machine called a centrifuge. The centrifuge spins the bone marrow solution and stem cells are separated from the non-useful cells. Now, the stem cells are ready for the treatment.

For those whom are ideal candidates, this provides great hope with reduction in pain and improved quality of life without the need for major surgery.

Voted as one of Americas Top Surgeons, Charles S. Theofilos, MD, Neurosurgeon and Founder of The Spine Center is a leading provider of the state-of-the-art, most comfortable and effective surgical, minimally invasive and non-surgical treatment options for a full range of cervical and spinal ailments, including stem cell therapy and artificial disc replacement. He was among a field of 20 top neuro and orthopedic surgeons in the U.S. chosen to participate in the groundbreaking Artificial Disc Study, which compared the clinical outcome of disc replacement versus traditional spinal fusion. A widely sought after educator and lecturer, Dr. Theofilos has offices in Palm Beach Gardens and Port St. Lucie. In an effort to maintain and honor the commitment to our patients, we will continue to accept Medicare and Medicare Advantage insurance plans for all new and follow up appointments.

11621 Kew Gardens Ave., Suite 101;Palm Beach Gardens

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What are mesenchymal stem cells? - Palm Beach Post

Bone Marrow Stem Cells Comments Off on What are mesenchymal stem cells? – Palm Beach Post | March 29th, 2017

Id like to believe that our lives have meaning, that the day-to-day decisions we make steer our steps down a variety of roads in life. As in the poem by Robert Frost, moving in one direction can leave us wondering about what would have happened had we taken the other road.

I dont believe Frost meant that we regret our choices, simply that new realities unfold as we move forward, and they are different than if we had moved left or right instead. Our decisions affect others as well, unfolding and connecting in ways we can only accept and not really understand.

One of my favorite thoughts is that from each difficulty we encounter, no matter how painful, some good will come of it; our tempering or pruning, if you will. It is our place to trust.

This I know. Eleven years ago, Heidi, the daughter of my dear friend Sue Sands, was diagnosed with Leukemia. Her family held a sign-up for Be The Match, the National Bone Marrow Donor Registry, in hopes of finding a stem cell donor to cure her.

I couldnt attend that evening, but shortly after I had a vivid dream in which I was a bone marrow match for Heidi. I knew I had to get tested, so I found another sign-up, did a cheek swab and entered the registry. I wasnt called to be a match for Heidi. In fact, she underwent treatment and was able to recover without a bone marrow transplant.

There was more to come. In 2009 I turned out to be a perfect match for a little girl who happened to be the same age as my daughter. Its more than just matching a blood type; a person must closely match on human leukocyte antigen. I agreed to donate bone marrow and it was a completely anonymous donation.

Let me give you the simplified version of donating bone marrow and stem cells. There are two ways; blood stem cells and bone marrow. In the former, the donor is given a medicine to boost stem cell formation and then the cells are drawn from their blood. In the latter, the donor undergoes surgery where the stem cells are harvested from the back of their hip bones. The stem cells are given to the recipient in about 48 hours, and if all goes well, they are completely cured of their disease.

People tell me they are amazed I would do this for someone I dont know. How could I not? When I was about 23, my mother died of multiple myeloma bone cancer. A little over a year ago, my stepmother succumbed to pancreatic cancer. I know intimately what it is like to feel helpless when a loved one is sick. I know the sense of loss that remains.

I recovered from the bone marrow donation. Yes I was sore for several weeks, and tired, but it felt good to have done something. Yet, there was more to the story. Mayo Clinic doctors had discovered in my pre-donation blood work that I had elevated calcium levels. It was due to a parathyroid tumor, and it was taking calcium from my bones. It didnt affect my ability to donate, but they advised removal of the tumor soon after.

Had I not been a donor, I might never have known about the parathyroid issue until it had severely affected my health. It still gives me shivers to think about it.

Fast forward to this past December. I was working on page layout one afternoon when I received a call from Be the Match. I was a potential match for another patient, this time an adult woman with Leukemia. I am at the top of the preferred age group, but I was the perfect match to potentially save this persons life.

Now, back to the idea of paths intermingling. My friends daughter Heidi, who had beat her leukemia, was dealt a tough blow. A few weeks ago she was diagnosed once again with leukemia, not the same type, but different. It was like lightning striking twice.

My mind instantly went back to my initial dream. What can this mean, that I am called to donate twice, defying so many odds, at the same time she is diagnosed again, against so many odds?

Last week I completed the second bone marrow donation for an unknown recipient. The doctors and nurses on the ninth floor of the Charleton Building at Mayo Clinic were amazing. They took such good care of me and thanked me repeatedly. The procedure went well and my APG employers have been gracious to give me time off for the whole thing.

I am oh so very tired, but the soreness in my hips is better each day. The need to build up the hemoglobin in my blood has made me extra conscientious of my diet. I am thankful for my good health and also for the friends, who brought food, checked in on me and have provided support through this donation process.

What does it all mean? We need each other. What we can do for another person in desperate need, we should do.

I hope and pray for a full recovery for Heidi. They will look for a bone marrow match for her this time around. Maybe I am a match for her after all? The doctor last week said that I could theoretically donate again once I was 100 percent recovered and if my hemoglobin levels were perfect.

Maybe I am not a match, but through my story, others will join the registry and Heidis match will be found. I guess its OK to not know what it all means. Knowing that something good will come is enough.

Reach Publisher and Editor Terri Lenz at 333-3148, or follow her on Twitter.com @KenyonLeader

Reach Publisher and Editor Terri Lenz at 333-3148, or follow her on Twitter.com @KenyonLeader

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Not knowing what it means is OK, follow your path - Southernminn.com

Bone Marrow Stem Cells Comments Off on Not knowing what it means is OK, follow your path – Southernminn.com | March 29th, 2017

The decision to become a living organ donor is a significant one and, among the many factors to weigh, donors should consider potential financial consequences of their altruism.

Medical costs associated with organ, tissue and bone marrow donations don't fall on the donor; they typically are paid by the recipient and often covered by insurance, Medicare or Medicaid.

But other related expenses including travel, lodging and loss of wages due to time out of work are the donor's responsibility.

Most of the roughly 6,000 living donations that occur annually are between relatives and close friends, people who have a vested interest in the recipient's outcome, according to the U.S. Department of Health and Human Services (HHS).

But even people who wouldn't hesitate to help a loved one should know about potential costs.

When Sally McCartin of North Branford donated a kidney to a fellow "hockey mom" in 2013, she knew she'd have to take time off from her job at the state's Department of Revenue Services.

"I was worried about the first two weeks that I would be out of work because I had used up all my sick time getting the necessary testing done to donate," she says. "Initially, I would have gone with no pay for the first two weeks."

The recipient of her kidney offered to cover McCartin's loss of wages, but the expense ultimately was covered by financial donations from McCartin's coworkers and contributions from her union, so she bore none of the cost.

For others, out-of-pocket expenses can be problematic and may deter some from donating altogether, says Sen. Martin Looney, D-New Haven. He proposed legislation in January that would help ease some of the burden.

Under his bill, people who donate organs or bone marrow after Jan. 1, 2017, could deduct up to $10,000 from their income, under the state personal income tax, to cover unreimbursed costs of travel, lodging and lost wages they incur as a result of donating. The bill also aims to allow state employees, beginning in 2018, to take up to 30 days of paid leave from work for organ donation and up to seven days of paid leave for bone marrow donation.

The legislation, co-sponsored by Sen. Catherine Osten, D-Columbia, is before the General Assembly's Public Health Committee and slated to receive a public hearing. It's intended to "limit potential barriers to people agreeing to be a live donor," Looney says.

The matter hits close to home for the senator, who suffered from kidney failure and received a kidney donation from a live donor in December. Nineteen states already have similar laws on the books, Looney says.

Some donors may have some expenses covered by their own insurance, depending on their plan, he says, but not all do.

"In some cases, the out-of-pocket expenses are minimal to the donor, and in other cases they can be substantial," he says, especially when it comes to missed work. "The donor is going to be out of work probably for a minimum of two weeks after the procedure. That would probably be the biggest hardship of all."

Many things can be donated by living donors, according to the HHS, including six vital organs: heart, kidneys, pancreas, lungs, liver and intestines. (In some cases a portion of the organ, such as the liver, is donated. Living heart donations are rare but happen. They occur when a donor has a pulmonary condition that necessitates removal of the heart and lungs for new ones lungs-only operations are considered riskier but the donor's heart is in good enough condition to be transplanted in another patient.)

Donors also may give certain tissues including skin, cornea and blood vessels as well as bone marrow, stem cells and umbilical cord blood.

April has been dubbed "National Donate Life Month," an effort by nonprofit advocacy group Donate Life America to encourage people to register as organ, eye and tissue donors.

About 20 to 25 living donations occur annually at the Hartford Hospital Transplant Program, according to registered nurse and Living Donor Transplant Coordinator Kari Rancourt. Most of them are kidney transplants.

"We do talk a little [with prospective donors] about out-of-pocket expenses," she says. Some donors get help covering costs through social fundraising platforms like GoFundMe, she says.

There also are foundations that offer grants to help would-be donors afford travel and lodging, she adds, which have "helped limit barriers to donations."

McCartin, who donated at Yale-New Haven Hospital, says that she did have to consider the potential expense but that wouldn't have stopped her from donating. She is so passionate about it, she recently began Kid-U-Not, a Connecticut chapter of the American Living Organ Donor Fund that raises funds to assist donors.

"I felt that, if I was healthy enough to donate, then it was a no-brainer," she says. "How could I not help a single mom of three children? I would hope that if I was ever in the same situation someone would step up for me."

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Living Organ Donors: Proposed Tax Deduction Could Help Defray Donation Expenses - Hartford Courant

Bone Marrow Stem Cells Comments Off on Living Organ Donors: Proposed Tax Deduction Could Help Defray Donation Expenses – Hartford Courant | March 27th, 2017

Using video microscopy in a living mouse lung, a team of researchers at the Universities of California, San Francisco (UCSF) & Los Angeles (UCLA), has revealed that the lungs play a previously unrecognized role in blood production.

Visualization of resident megakaryocytes in the lungs. Image credit: Emma Lefranais et al, doi: 10.1038/nature21706.

The team, headed by UCSF Professor Mark R. Looney, found that the lungs produced more than half of the platelets blood components required for the clotting that stanches bleeding in the mouse circulation.

In another finding, the team also identified a previously unknown pool of blood stem cells capable of restoring blood production when the stem cells of the bone marrow, previously thought to be the principal site of blood production, are depleted.

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, Prof. Looney said.

What weve observed here in mice strongly suggests the lung may play a key role in blood formation in humans as well.

The study was made possible by a refinement of a technique known as two-photon intravital imaging.

The authors were using this technique to examine interactions between the immune system and circulating platelets in the lungs, using a mouse strain engineered so that platelets emit bright green fluorescence, when they noticed a surprisingly large population of platelet-producing cells called megakaryocytes in the lung vasculature.

When we discovered this massive population of megakaryocytes that appeared to be living in the lung, we realized we had to follow this up, said team member Dr. Emma Lefranais, from the UCSF Department of Medicine.

More detailed imaging sessions soon revealed megakaryocytes in the act of producing more than 10 million platelets per hour within the lung vasculature, suggesting that more than half of a mouses total platelet production occurs in the lung, not the bone marrow, as researchers had long presumed.

Video microscopy experiments also revealed a wide variety of previously overlooked megakaryocyte progenitor cells and blood stem cells sitting quietly outside the lung vasculature estimated at 1 million per mouse lung.

Proposed schema of lung involvement in platelet biogenesis. The role of the lungs in platelet biogenesis is twofold and occurs in two different compartments: (a) platelet production in the lung vasculature; after being released from the bone marrow or the spleen, proplatelets (a1) and megakaryocytes (a2) are retained in the lung vasculature, the first capillary bed encountered by any cell leaving the bone marrow, where proplatelet formation and extension and final platelet release are observed; (b) mature and immature megakaryocytes along with hematopoietic progenitors are found in the lung interstitium; in thrombocytopenic environments, hematopoietic progenitors from the lung migrate and restore bone marrow hematopoietic deficiencies. Image credit: Emma Lefranais et al, doi: 10.1038/nature21706.

The discovery of megakaryocytes and blood stem cells in the lung raised questions about how these cells move back and forth between the lung and bone marrow.

To address these questions, Prof. Looney, Dr. Lefranais and their colleagues conducted a clever set of lung transplant studies.

First, they transplanted lungs from normal donor mice into recipient mice with fluorescent megakaryocytes, and found that fluorescent megakaryocytes from the recipient mice soon began turning up in the lung vasculature.

This suggested that the platelet-producing megakaryocytes in the lung originate in the bone marrow.

In another experiment, the team transplanted lungs with fluorescent megakaryocyte progenitor cells into mutant mice with low platelet counts.

The transplants produced a large burst of fluorescent platelets that quickly restored normal levels, an effect that persisted over several months of observation much longer than the lifespan of individual megakaryocytes or platelets.

This indicated that resident megakaryocyte progenitor cells in the transplanted lungs had become activated by the recipient mouses low platelet counts and had produced healthy new megakaryocyte cells to restore proper platelet production.

Finally, the researchers transplanted healthy lungs in which all cells were fluorescently tagged into mutant mice whose bone marrow lacked normal blood stem cells.

Analysis of the bone marrow of recipient mice showed that fluorescent cells originating from the transplanted lungs soon traveled to the damaged bone marrow and contributed to the production not just of platelets, but of a wide variety of blood cells, including immune cells such as neutrophils, B cells and T cells.

These experiments suggest that the lungs play host to a wide variety of blood progenitor cells and stem cells capable of restocking damaged bone marrow and restoring production of many components of the blood.

To our knowledge this is the first description of blood progenitors resident in the lung, and it raises a lot of questions with clinical relevance for the millions of people who suffer from thrombocytopenia, Prof. Looney said.

The findings were published online March 22, 2017 in the journal Nature.

_____

Emma Lefranais et al. The lung is a site of platelet biogenesis and a reservoir for haematopoietic progenitors. Nature, published online March 22, 2017; doi: 10.1038/nature21706

This article is based on text provided by the University of California, San Francisco.

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Lungs Play Previously Unknown Role in Blood Production - Sci-News.com

Bone Marrow Stem Cells Comments Off on Lungs Play Previously Unknown Role in Blood Production – Sci-News.com | March 27th, 2017

As people age, so does their blood. The question is: what exactly is in the blood of older people that makes it age? And in the same light, what is in the blood of younger people that can help rejuvenate old blood?

The idea of using young blood to rejuvenate old blood was not an automatic conclusion, of course. While it does seem logical, it remained a theory until tests that involved conjoining (i.e. stitching together) of old and young mice for the purpose of swapping blood revealed that the concept did have merit. With shared blood, the health of younger mice deteriorated while the health of older mice improved.

Another kind of experiment done was non-invasive blood swapping using tubes. The results were similar, though different explanations emerged for the change in health conditions of both old and young mice. When conjoined, the mice shared more than just blood; their organs got affected too. In non-invasive blood swapping, the old blood got diluted.

While these experiments were done on mice, theres a chance they might work in people as well. However, this involves blood donation from young people, which might mean the supply will be limited when it comes to fulfill demand.

As an alternative, a research team at Germanys University of Ulm led by Hartmut Geiger turned to stem cells, specifically, what are being referred to as mother stem cells those stem cells in the bone marrow that produce red and white blood cells, and whose number become fewer and fewer as a person ages. With fewer of these cell-generating cells, older people become more susceptible to conditions like anemia and heart disease. They become less capable of fighting infection as well.

By examining mice bone marrow, Geigers team discovered that older mice have considerably lower levels of a protein known as osteopontin. To check the effect of this protein on blood stem cells, they injected stem cells into mice that had low levels of osteopontin. What happened was, the cells aged much quicker.

However, when they mixed older stem cells with osteopontin and a protein that activates osteopontin, the old stem cells started producing white blood cells as if they were young stem cells. This suggests that osteopontin might indeed have a hand in rejuvenating old stem cells and making them behave as if they were young again.

While majority of blood rejuvenation efforts focus on the liquid part of blood (or plasma), Geiger believes blood cells might also play a vital role since cells can move better in the bodys tissues.

Following the initial results of their experiments, the team is now working on developing a drug that contains osteopontin and its corresponding protein activator. The hope is that this drug can promote youthful behavior in blood stem cells and boost the number of mother stem cells. Ultimately, this can help in the treatment of age-related blood disorders, and possibly boost the immune system of the elderly too so they dont get sick as easily.

Details of the study have been reported in The EMBO Journal.

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Protein Found in Young Blood Could Be The Key To Fight Aging - Wall Street Pit

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Investopedia

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

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Cellect Succeeds In First Stem Cell Transplant (APOP) - Investopedia

Bone Marrow Stem Cells Comments Off on Cellect Succeeds In First Stem Cell Transplant (APOP) – Investopedia | March 27th, 2017

Healthy cell function relies on well orchestrated gene activity. Via a fantastically complex network of interactions, around 30,000 genes cooperate to maintain this delicate balance in each of the37.2 trillion cellsin the human body.

Broadly speaking, cancer is a disruption of this balance by genetic changes, or mutations. Mutations can trigger over-activation of genes that normally instruct cells to divide, or inactivation of genes that suppress the development of cancer. When a mutated cell divides, it passes the mutation down to its daughter cells. This leads to the accumulation of non-functioning, abnormal cells that we recognise as cancer.

Our laboratoryis focused on understanding how one particular cancer chronic myeloid leukaemiaor CML works. Each year more than 700 patients in the UK andover 100,000worldwide are diagnosed with CML. After recent advances,almost 90%of patients under the age of 65 now survive for more than five years.

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But in the vast majority of patients CML is currently incurable and lifelong treatment means that patients must live with side effects and the chance of drug resistance arising. With increasing numbers of CML patients surviving (and treatment costing between 40,000 and 70,000 per patient a year), increasing strain is being placed on health services.

A single mutation

CML is perhaps unique in cancers in that a single mutation, namedBCR-ABL, underlies the disease biology. This mutation originates in a singleleukaemic stem cell, but is then propagated throughout the blood and bone marrow as leukaemia cells take over and block the healthy process of blood production. The presence of BCR-ABL affects the activity of thousands of genes, in turn preventing these cells from fulfilling their normal function as blood cells.

Drugsthat specifically neutralise the aberrant effects of this mutation were introduced to the clinic from the early 2000s. These drugs have revolutionised CML patient care. Many are now able to live relatively normal lives with their leukaemia under good control.

But while these drugs kill the more mature daughter cells of the originally mutated leukaemia stem cell, they have not fully lived up to their initial billing as magic bullets in the fight against cancer. This is because the original seed population of leukaemic stem cellsevade therapy,lying dormant in the bone marrowto stimulate new cancer growth when treatment is withdrawn.

To truly cure CML we must expose, understand the inner workings of, and uproot the leukaemia stem cells. And to do this, we need to learn more about them. How do they survive the treatment that so readily kills their more mature counterparts? Which overactive or inactivated genes protect them?

We believe that the answers to these questions lie in the analysis of biological big data. Genome-scale technologies now allow scientists to measure the activity (or expression) of every gene in the genome simultaneously, in any given population of cells, or even at the level of a single cell. Comparison of expression data generated from leukaemia stem cells with the same data generated from healthy blood stem cells will reveal single genes or networks of genes potentially targetable in the fight against leukaemia.

Big data to the rescue

In a project funded by Bloodwise and the Scottish Cancer Foundation, we have createdLEUKomics. This online data portal brings together a wealth of CML gene expression data from specialised laboratories across the globe, including our own at the University of Glasgow.

Our intention is to eliminate the bottleneck surrounding big data analysis in CML. Each dataset is subjected to manual quality checks, and all the necessarycomputational processingto extract information on gene expression. This enables immediate access to and interpretation of data that previously would not have been easily accessible to academics or clinicians without training in specialised computational approaches.

Consolidating these data into a single resource also allows large-scale, computationally-intensive research efforts by bioinformaticians (specialists in the analysis of big data in biology). From a computational perspective, the fact that CML is caused by a single mutation makes it an attractive disease model for cancer stem cells. However, existing datasets tend to have small sample numbers, which can limit their potential.

The more samples available, the higher the power to detect subtle changes that may be crucial to the biology of the cancer stem cells. By bringing all the globally available CML datasets together, we have significantly increased the sample size, from two to six per dataset to more than 100 altogether. This offers an unprecedented opportunity to analyse gene expression data to expose underlying mechanisms of this disease.

As of March 2017, theportalis up and running in the public domain. We are planning to tour Scotland and present at international conferences, aiming to train researchers in how best to exploit this new resource. Ultimately, we hope that this tool will lead to new ideas and approaches, and attract more funding, in the fight against CML. And while we continue to expand our representation of CML data in real time from research centres all over the world, we also plan to begin incorporating data from other types of leukaemia.

In recent years, targeted therapies have becomehugely importantin cancer research. By providing these data to the CML research community withinLEUKomics, we hope to mobilise new research into cancer-causing leukaemic stem cells, and ultimately design treatments to target them without affecting healthy cells. Our database provides a critical stepping stone in this process.

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How Big Data is Being Mobilized in the Fight Against Leukemia - Drug Discovery & Development

Bone Marrow Stem Cells Comments Off on How Big Data is Being Mobilized in the Fight Against Leukemia – Drug Discovery & Development | March 27th, 2017

Stem cells are the foundation of all our body cells before they differentiate to become specialised cells that grow into our tissues and organs, such as kidney cells, muscle cells, nerve cells, and so on.

They commonly come from two sources: The embryo (embryonic stem cells formed in early development after the human egg is fertilised by a sperm); and adult tissue (adult stem cells, such as those existing in bone marrow to later differentiate to form red blood cells, white blood cells and other components of the blood).

The use of human embryonic stems cells for treatment or research is often frowned upon by some people, as they regard the human embryo as a person that should not be discarded after such endeavours. Consequently, much scientific work has recently been focused on the use of adult stem cells.

THE USE OF STEM CELLS

Stem cells may be beneficial in treating diseases that are amenable to cell replacement. However, this is still a young science, and belief that a particular treatment helps two or three people does not convince the scientific community or the whole society that the treatment will work for everyone so afflicted.

Scientific proof comes from conducting clinical trials, the international gold standard often involving hundreds of people so afflicted and comparing them with an equivalent number of people not afflicted to determine whether a treatment really works for those who receive it.

Whilst many stem cell research projects are currently being conducted in various centres around the world to determine whether they produce benefits, and what may be the possible risks involved, there are also medical clinics that are using stem cells not in a registered research project, but rather in the actual treatment of affected people.

TREATMENT CAN CAUSE HARM

A recent report in the highly respected New England Journal of Medicine informed that three elderly women in Florida had been blinded by an unproven treatment.

They had signed up for a purported clinical trial in 2015 for which they had to pay US$5,000 each. Before surgery, the vision in their eyes varied from 20/30 to 20/200, but within one week after surgery, they experienced a variety of complications, including vision loss, detached retinas and bleeding into their eyes, resulting in total blindness.

The authors of the article from the Standard University School of Medicine sought to make patients, doctors and the various regulatory agencies aware of the risks of such a minimally regulated, patient-funded research. It stated that some clinics appeal to patients that are desperate for care and who hope that stem cells will be their answer, but as in the case of these women, some of these current enterprises are very dangerous.

At this particular clinic, fat cells were taken from the patients abdomens and processed to obtain stem cells which were then injected into their eyes. The patients reported that the entire process took less than one hour. The patients had both eyes treated at once, even though most doctors would opt for a conservative approach to observe how the first eye responds.

THE NEED FOR THE REGULATION OF RESEARCH

The article stated that while there is a lot of well-founded evidence for the positive potential of stem cell treatment for many human diseases, such treatments should be conducted in a well-designed clinical trial based on pre-clinical research.

The treatment done for the women lacked nearly all the components of a properly designed clinical trial, including a hypothesis based on laboratory experiments, the involvement of a control group of people and a treatment group, the safe collection of data, the masking of clinical and patient groups, and plans for follow-up.

Clinics offering stem cell treatments exist in Jamaica, The Bahamas and Cuba. However, while both The Bahamas and Cuba have developed regulations that stipulate in law the conditions to be met for stem cell treatments and research within their jurisdictions, Jamaica has developed no such regulation.

THE MEDICAL ACT DOES NOT PROVIDE PROTECTION

The Medical Act of Jamaica was passed in 1976, but does not mention or provide any guidance or protection regarding research with human participants.

Its focus was to: Register medical practitioners; appoint examiners to conduct exams for people applying for registration, and ensure the maintenance of proper professional conduct by practitioners.An amendment in 2004 added the requirement of continuing medical education for practitioners.

Guyana and St Lucia are the only countries in the Caribbean that have joined the progressive countries who all have legislation governing research with human participants within their borders. Regulations should stipulate the requisite conditions, including that treatment and research be monitored by an appropriate ethics committee to meet all international standards.

Without this, vulnerable people seeking health benefits will unknowingly continue to subject themselves to risks of harm without the protection that proper regulations can provide.

Derrick Aarons MD, PhD is a consultant bioethicist/family physician, a specialist in ethical issues in medicine, the life sciences and research, and is the Ethicist at the Caribbean Public Health Agency (CARPHA). (The views expressed here are not written on behalf of CARPHA)

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Stem cell treatments can go wrong - Jamaica Observer

Bone Marrow Stem Cells Comments Off on Stem cell treatments can go wrong – Jamaica Observer | March 26th, 2017

The suggestion that young blood may be the key toreversingsome of the negative aspects of aging sounds like the setup to a horror movie. In reality, however, itrefers to some groundbreaking work being carried out byscientists at the University of Ulm in Germany.

Theyve been examining the ways that old blood can be made young again, and they hypothesize that it might help fight some of the effects of aging.To achieve this, theyve discovereda protein capable of boosting blood stem cells, which prompts them to act like the stem cells of younger people.

More: Scientists may have discovered how to reverse the natural aging process

Older stem cells have several disadvantages, Dr. Hartmut Geiger, co-author of a paper describing the work, told Digital Trends. Theyresult in fewer red blood cells, and fewer of an important immune cell that fights infection. They also tend to produce more leukemia, which is a cancer of the blood. All of this is linked, at least in part, to the aging of stem cells. If you have younger stem cells, we think that we can reverse some of these effects.

Older stem cells tend to possess these negative characteristics thanks to the declining number of so-called mother stem cells in our bone marrow as we age. This decline triggers the effects Geiger describes.

In trials with mice, Geiger and his team discovered that older mice have lower levels of a protein called osteopontin. They decided to testthe effect of this protein on blood stem cells.

We took the stem cells from aged mice, incubated them with the osteopontin protein, and then gave them back, he continued.These blood stem cells, which had come from older animals, were found to have a lot of the features of stem cells coming from much younger animals in terms of function.

Its still early days for the research, but the suggestion that osteopontin could make stem cells behave in a more youthful way is certainly promising. The team is now developing a drug made up of osteopontin and a protein for activating it.

Hopefully human trials can follow in the not-too-distant future while were still around to benefit from them.

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Young blood: Scientists say this protein could be key to reversing aging process - Yahoo News

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Why it matters to you

Nobody likes getting older. Reversing some of the bad aspects of the process helps us all age gracefully.

The suggestion that young blood may be the key toreversingsome of the negative aspects of aging sounds like the setup to a horror movie. In reality, however, itrefers to some groundbreaking work being carried out byscientists at the University of Ulm in Germany.

Theyve been examining the ways that old blood can be made young again, and they hypothesize that it might help fight some of the effects of aging.To achieve this, theyve discovereda protein capable of boosting blood stem cells, which prompts them to act like the stem cells of younger people.

More: Scientists may have discovered how to reverse the natural aging process

Older stem cells have several disadvantages, Dr. Hartmut Geiger, co-author of a paper describing the work, told Digital Trends. Theyresult in fewer red blood cells, and fewer of an important immune cell that fights infection. They also tend to produce more leukemia, which is a cancer of the blood. All of this is linked, at least in part, to the aging of stem cells. If you have younger stem cells, we think that we can reverse some of these effects.

Older stem cells tend to possess these negative characteristics thanks to the declining number of so-called mother stem cells in our bone marrow as we age. This decline triggers the effects Geiger describes.

In trials with mice, Geiger and his team discovered that older mice have lower levels of a protein called osteopontin. They decided to testthe effect of this protein on blood stem cells.

We took the stem cells from aged mice, incubated them with the osteopontin protein, and then gave them back, he continued.These blood stem cells, which had come from older animals, were found to have a lot of the features of stem cells coming from much younger animals in terms of function.

Its still early days for the research, but the suggestion that osteopontin could make stem cells behave in a more youthful way is certainly promising. The team is now developing a drug made up of osteopontin and a protein for activating it.

Hopefully human trials can follow in the not-too-distant future while were still around to benefit from them.

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Scientists May Have Found A Way To Make Old Stem Cells Act ... - Digital Trends

Bone Marrow Stem Cells Comments Off on Scientists May Have Found A Way To Make Old Stem Cells Act … – Digital Trends | March 25th, 2017

In what could potentially serve as an importantmoment in the quest to 3D-print body parts, a team of scientists from Swedens Sahlgrenska Academy and Chalmers University of Technology have managed to successfully implant human cartilage cells in six-week-old baby mice.

The researchers created a gel composed of human cartilage cells, printed it through a CELLINK3D bioprinter and implanted the material inside the lab mice. Once implanted, the tissue began to grow and proliferate inside the animal, eventually vascularizing, with blood vessels growing inside the implanted material. After two months, the material began to more closely resemble human cartilage, which was further stimulated with the addition of stem cells.

The team worked with local plastic surgeons to implant the material, which could one day be used to create more natural implants for patients who have lost ears, noses or knees due to accidents or diseases like cancer.

There is no solution for missing ears, research lead professor Paul Gatenholm tells TechCrunch. You have plastic and silicone implants, which you need to put in with a titanium screw. Thats it. The surgical procedure is that you put in cartilage from a rib from the patient and carve. Its very painful and the outcome is very bad. If we can use cells from the nose and beef it up with stem cells from the patients bone marrow or fat, we will be able to print that full 3D structure.

Gatenholm adds, hopefully, It will have a great impact on healthcare for tissue regeneration and implementation. I think the first breakthrough will be of the skin and then the cartilage and then the bone. Beyond that, the technology could some day also be used for even more complex human parts like organs.

Gatenholm cites a paper from February of last yearin his discussion. In it, a team of researchers, including regenerative medicine professor Anthony Atala, detail how theyve used 3D bioprinting to construct bone and muscle using computer imaging to translat[e] the model into a program that controls the motions of the printer nozzles, which dispense cells to discrete locations.

Similarly, 3D modeling in the form of CAD (computer-aided design) files can be used by the printer to create better anatomical pieces than plastic surgery modeling.

The process is still a ways from becoming a viable reality for future reconstructive surgery, including all of the regulatory approvals that come with implementing of this sort of invasive procedure. But the teams work marks a promising step in the process, which could some day extend beyond cartilage to other key human tissue.

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Swedish scientists successfully implant 3D-printed human cartilage cells in baby mice - TechCrunch

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Selfless Ray Noble may never meet the stranger whose life he saved.

The 29-year-old registered as a stem cell and bone marrow donor four years ago after a young girl his wife knew was diagnosed with cancer.

If that was my little girl Id want someone to be there for her, he said.

Ive been a blood donor for a while, so I thought why not sign up to the stem cell register as well.

And last year dad-of-one Ray, from Wallsend, made a life-saving donation after being told he was a match for an unknown patient in urgent need of a transplant.

Now blood cancer charity Anthony Nolan have urged more people to follow Rays example after a survey revealed that 50% of young men from the North East could not be encouraged to sign up to a blood stem cell or bone marrow register for any reason.

Every year there are 2,000 people in the UK in need of a bone marrow or stem cell transplant. This is usually their last chance of survival.

For Ray, the path to becoming a blood cancer patients last hope started when a relative of his wifes friend was diagnosed with the disease.

The process was pretty simple, he said.

I followed the instructions on the Anthony Nolan website about how to sign up.

Within a week or two they sent me a spit test, where I basically had to spit into a tube and send it off so it could be analysed.

I then got a card a few weeks later saying I was on the register.

Since signing up, Ray has been identified as a potential match for two patients.

About two years ago Anthony Nolan got in touch to say that I was a potential match for someone and I had to go and give some samples.

On that one they managed to find a closer match - I was eight out of 10 and they found a 10 out of 10, which was obviously better for the patient.

Then around Christmas last year they confirmed that I was a match for someone.

After undergoing several health checks and injections to stimulate the stem cells in his blood, Ray travelled down to Sheffield in April last year to make the donation.

All in all it took about four or five hours, he said. Id been aching a bit before the procedure because of the injections but afterwards I felt totally fine.

Ray, who is dad to two-year-old Ariana, has since convinced several friends and relatives to sign up.

For me its a question of, why not?, he said.

Its not that likely that youre ever going to be asked to donate - its just a case of being on there for someone if they need it.

I always ask people: How would you feel if it was your child or parent or cousin, if they needed a donor and you werent a match - would you want someone to step up and help them?

Every 20 minutes someone in the UK finds out they have a blood cancer.

Around 2,000 people in the UK in need of a bone marrow or stem cell transplant every year. This is usually their last chance of survival.

75% of UK patients wont find a matching donor in their families. So they turn to Anthony Nolan to find them an unrelated donor.

Healthy adults aged between 16 and 30 can sign up for a simple, pain-free test through the Anthony Nolan Trust.

The charity particularly need more young men to sign up. They produce more stem cells than women and are six times more likely to donate, but make up just 15% of the register. They also need more donors from black and minority ethnic backgrounds as they often struggle to find matches for people in these groups.

Check the list of criteria to make sure youre eligible to join and fill in an application form, either online or at an Anthony Nolan recruitment event.

If you come to a recruitment event and your application is OK, you can give your saliva sample there. If you apply online, youll be sent spit kit in the post. All you need to do is spit into a small tube and post it back.

The sample will be tested and the results put in the charitys database. Every time someone needs a transplant, theyll automatically compare their tissue to yours and the 620,000 other individuals on the register.

You can donate your stem cells in two ways.

Nearly 90% of people donate their stem cells quickly and easily in a process similar to giving blood, called peripheral blood stem cell collection.

The other 10% donate through bone marrow, where they give cells from the bone marrow in their pelvis.

If youre on the register, you must be happy to donate stem cells in either way.

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'If that was my little girl I'd want someone to step up': Stem cell donor on lifesaving transplant - ChronicleLive

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A non-surgical treatment that uses a patients own bone marrow stem cells to treat chest pain or angina improved both symptoms and the length of time treated patients could be physically active, according to recent research.

We injected a catalyst molecule that caused bone marrow stem cells to enter the patients blood, then harvested them to re-inject into the patient,said Hadyanto Lim, Ph.D., study senior author.

Thirty minutes after the cell separation procedure finished, the collected stem cells were injected back into the patient through an IV.

Four weeks after receiving the treatment, patients experienced significantly fewer angina-related symptoms, and they were able to exercise at a higher intensity and for a longer period of time.

The studys limitations are the small number of patients and absence of a control group. Because no control group was used, the placebo effect cannot be ruled out, Lim noted.

Although this treatment is currently used to treat some cancers multiple myeloma and lymphoma it will need more investigation before it can be made available to the general public to treat angina, according to Lim.

The GLP aggregated and excerpted this blog/article to reflect the diversity of news, opinion, and analysis. Read full, original post:Hard to treat chest pain may be improved with a patients own stem cells

For more background on the Genetic Literacy Project, read GLP on Wikipedia.

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Injection with own stem cells alleviates chest pains, angina, study finds - Genetic Literacy Project

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

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

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

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

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

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

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

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

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

Thats a win

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

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

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

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

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

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

Creating hope

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

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

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

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

Not for everyone

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

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

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

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

We developed this over many decades, he said.

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

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

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

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

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

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

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

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

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

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

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

No more drugs

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

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

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

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

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

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

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

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

Reporter: 541-383-0304,

tbannow@bendbulletin.com

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Stem cell transplant reduces Culver woman's MS symptoms - Bend Bulletin

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This week a very special delivery was made from space that will help further research that could eventually lead to a mind-blowing, futuristic way to cure diseases: shooting unmanned satellite wombs into orbit and then retrieving from them batches of stem cells that can be used to treat patients. Regardless of the outcome, the scientific experiment will still advance our knowledge of these unique cells.

On Thursday Dr. Abba Zubairat the Mayo Clinic in Jacksonville, Florida, received frozen stem cells grown at the International Space Station. The package was part of the 5,400 pounds of scientific samples and equipment that splashed down on Sunday off the coast of California inside a SpaceX Dragon-10 capsule completing a historic round-trip mission.

Up there, one of the astronauts helped us to image the cells, harvest the cells and freeze them in a way that we can use them here on Earth and compare them to cells we grew here in the lab, Zubair, the principal investigator of the stem cell experiment, told Salon.

Zubairs team will look to see if the culture grown in the near-zero gravity of low-space orbit, about 250 miles above the Earths surface, results in healthier cells than onesgrownin aterrestrial lab. If so then it would helpconfirm the theory that microgravity, which resembles the weightless-likebuoyancyof female womb, is best environment for growing stemscells.

Stem cells, from which all other types of cells originate, are the bodys raw materials, and as such offer immense potential to cure many diseases. Doctors already use stem cells forbone-marrow transplants and treating blood-related diseases like leukemia, as well asfor some eye-related disorders. Researchers believe were only in the very early stages of developing revolutionary stem cell therapiesto combat cancer, Alzheimers disease, Parkinsons disease, Type 1 diabetes, heart disease and strokes. In the future, stems cellscience could even lead to growing organs in a lab that can be transplanted into humans.

But stem cells are finicky. As they replicate in a lab, many of them develop imperfections and have to be discarded. It can take a month to grow the roughly 200,000 cells needed to treat one patient, Zubair said. Gravity might be the culprit.

In nature, these cells start their life after an egg is fertilized. Humans, right from conception, develop almost in a microgravity environment, Zubair said. Fetuses develop in amniotic fluid. Theyre buoyant, which cancels the effect of gravity because theyre suspended in a liquid. Thats how three-dimensional growth in a fluid environment is possible. We think gravity does play a role in the shape and development of the cells and how organs develop.

In other words, if the cells are suspended in fluid, they can grow and move in any direction, producing more of them, compared withhow they grow on a flat surface, like in a petri dish.

This is why stem cells are typically grown in a bioreactor, a common bioengineering tool that gently stirswater containing the seed cells and certain nutrients that promote growth. But because of the way gravity affectsfluids, many of the cells become damaged and cant be used for treatment. (In the language of physics, the problem has to do with something called shearing force.) By placing a bioreactor in the microgravity of orbit, the effects of gravity on liquid mechanics is virtually eliminated.

If growing stem cells in spaceproves to be efficient, thats when things get interesting. Growing stem cells at the International Space Station is anexperimental endeavor, so its not really a viable place to begin manufacturing themin great quantities. But theoretically, Zubair says, bioreactor satellites could be put into orbit and left there to grow cells until theyre remotely called back to Earth or sent wherever future interplanetary pilgrims wind up. As the cost of sending small satellites into low orbit falls, this system could be commercially viable.

There are companies that are interested in developing a floating lab in space to grow not only stem cells but also tissues and organs down the road for human use or for use elsewhere as we hopefully colonize other planets, like Mars, Zubair said.

This might seem out of this world, but the technology for growing stem cells remotely already exists. If space is the place to grow human parts and this research will help to determine that then designing systems and deploying these bioreactor space wombs might not be that far off in the future.

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Space wombs for stem cells: Satellites could help accelerate the discovery of disease cures - Salon

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A good starting point for any scientist in any field is to acknowledge that theres a lot that we dont know. We dont know, for example, why there is more matter than antimatter in the universe. We dont know quite how the evolution of the dinosaurs panned out. And, perhaps most surprisingly of all, we dont know quite how many organs the human body has or what all their functions are.

This January, researchers have announced that a brand new organ had been discovered in our bodies after it had long been mistaken for something else. Now, writing in the journal Nature, a group of researchers from the University of California, San Francisco (UCSF), have found that the lungs in mice have a hidden feature too they help make blood.

Specifically, it appears the lungs produce over half of the platelets the components that bind blood together to stop us bleeding out when were wounded involved in circulation.

So not only do our breathing bags allow us to respire, but they also help keep our cardiovascular system full to the brim. Well thats rather lovely of them.

Thats not all. The researchers also managed to identify a cache of stem cells the type that can differentiate into almost any cell type with the right biological programming that can transform themselves into blood cells.

Bone marrow is thought to be the primary source of such stem cells, so this new revelation suggests that if our bone marrow is damaged and unable to keep up with its regular blood cell manufacture, our lungs can step in to make up for the shortfall.

This finding definitely suggests a more sophisticated view of the lungs that they're not just for respiration but also a key partner in formation of crucial aspects of the blood, senior author Mark Looney, a professor of medicine at UCSF, said in a statement.

A little caveat worth mentioning at this point is that this hasnt been directly imaged in humans, but mice. Nevertheless, the biological workings of these little critters is surprisingly similar to that of humans, which is part of the reason why theyre used in so many medical-themed studies so theres a good chance human lungs also possess the same hidden features.

Using a remarkable technique allowing the platelets to fluoresce, the team were able to directly trace the paths of the mousey platelets, and found they were coming from within the lungs. The megakaryocytes the platelet-producing cells are also seen moving back and forth between the lungs and the bone marrow, depending on where they are needed the most.

Perhaps studying abroad in different organs is a normal part of stem cell education, Looney added.

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Scientists Have Discovered A Secret Function Of Lungs - IFLScience

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Jonathan Pitre readies for his second stem cell transplant, which will take place April 13th at the University of Minnesota Masonic Children's Hospital. Tina Boileau / -

Fully recovered from a series of infections, Jonathan Pitre has received medical clearance to undergo a second stem cell transplant.

Pitre, 16, will check into hospital on the last day of March to begin eight days of high-dose chemotherapy and one day of radiation. His stem cell transplant what doctors call Day Zero is scheduled forApril 13 at the University of Minnesota Masonic Childrens Hospital.

The night before he goes into hospital, Pitre will attend the Ottawa Senators game against the Minnesota Wild at the Xcel Energy Centre in Saint Paul. It will be a good night of fun before it all starts again, said Pitres mother, Tina Boileau.

She shared the latest news on her Facebook page on Wednesday.

After many weeks of tests, procedures and appointments at the hospital, Jonathan got the green light to proceed with the second transplant, she said. He has completely recovered from his infections and his body is as strong as can be This time it will work!

Last September, Pitre suffered nausea, hair loss, fevers and exhaustion in the aftermath of his first transplant, which ultimately failed when his own stem cells recolonized his bone marrow.His second transplant has been delayed because of lung and blood infections.

In an interview earlier this month, Pitre told the Citizen hes staying positive even though he understands the physical test that he faces in hospital.

Its mostly thinking about sticking together with the people you care about, your family, he said . You have to stick to them very, very tightly and tell each other that, Its going to be OK, and that Were stronger than this. Were going through this together, not just alone.

Pitre suffers from a rare, painful and deadly form of epidermolysis bullosa (EB), a blistering skin disease.

Hes the first Canadian to take part in a clinical trial operated by the University of Minnesotas Dr. Jakub Tolar, a pediatric transplant specialist who has adapted stem-cell therapy as a treatment for the most severe forms of EB.Although the procedure comes with the potential for life-threatening complications, it has produced dramatic improvements in two-thirds of those EB patients who have survived the transplant: tougher skin, reduced blistering and better wound healing.

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'Butterfly boy' Jonathan Pitre cleared for second stem cell transplant - Ottawa Citizen

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Fresh young cells

Dennis Kunkle Microscopy/Science Photo Library

By Jessica Hamzelou

BLOOD from the young seems to have healing powers, but how can we harness them without relying on donors? The discovery of a protein that keeps blood stem cells youthful might help.

The rejuvenating properties of young blood came to light in macabre experiments that stitched young and old mice together to share a circulatory system. The health of the older mice improved, while that of the younger ones deteriorated. Other animal studies have since shown that injections of young or old blood have similar effects.

This may work in people too. Young blood is being trialled as a treatment for conditions like Alzheimers, and aged mice that received injections of blood from human teenagers showed improved cognition, memory and physical activity levels.

We think the drug will improve signs of ageing and boost the immune systems of older people

But these studies rely on young people donating their blood: if this became the go-to therapy for age-related disease it would be difficult to get enough donations to fulfil demand.

The stem cells in our blood could provide an alternative approach. Our red and white blood cells are made by stem cells that themselves come from mother stem cells in bone marrow. But as we age, the number of these mother stem cells declines. One of the worlds longest-lived women seemed to only have two left in her blood when she died at age 115.

The decline in mother stem cells causes people to have fewer red blood cells, and white blood cells called B and T lymphocytes. These declines can cause anaemia and weaken the immune system. Usually the immune system in the elderly is not prepared to fight infections very hard, says Hartmut Geiger at the University of Ulm in Germany.

When Geigers team examined the bone marrow in mice, they found that older animals have much lower levels of a protein called osteopontin. To see if this protein has an effect on blood stem cells, the team injected stem cells into mice that lacked osteopontin and found that the cells rapidly aged.

But when older stem cells were mixed in a dish with osteopontin and a protein that activates it, they began to produce white blood cells just as young stem cells do. This suggests osteopontin makes stem cells behave more youthfully (EMBO Journal, doi.org/b4jp). If we can translate this into a treatment, we can make old blood young again, Geiger says.

Its exciting, says Hanadie Yousef at Stanford University in California. But longer term studies are needed to see whether this approach can rejuvenate the whole blood system, she says.

Until now, most efforts to use blood as a rejuvenation agent have focused on plasma, the liquid component, as some believe it carries dissolved factors that help maintain youth. But Geiger thinks the cells in blood might play a key role, because they are better able to move into the bodys tissues.

Both soluble factors and blood cells are likely to be important, says Yousef. While injections of young plasma rejuvenate older animals, the treatment doesnt have as strong an effect as when young and old animals share a circulatory system, she says.

Geigers team is developing a drug containing osteopontin and the activating protein to encourage blood stem cells to behave more youthfully. It should boost the immune system of elderly people, he says.

Such a drug might have benefits beyond fighting infection and alleviating anaemia. The team also think the protein will boost levels of mother stem cells. Having only a small number of such cells has been linked to heart disease, so Geiger says there is a chance that boosting them may help prevent this.

Osteopontin might also be useful for treating age-linked blood disorders, such as myelodysplasias that involve dysfunctional cells, says Martin Pera of the Jackson Laboratory in Bar Harbor, Maine. It is possible that rejuvenating bone marrow stem cells could help with these conditions, he says.

This study provides more evidence that cells can be rejuvenated, says Ioakim Spyridopoulos at Newcastle University, UK. They have made old blood look young again, although whether it acts young or not will have to be shown in clinical trials.

This article appeared in print under the headline Old blood made young again

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Challenging a long-held model about how blood is formed, a study led by UC San Francisco researchers has found that the lungs play a crucial role in the process, producing half of blood platelets and also storing blood-forming stem cells.

The study, performed in mice, also found that blood stem cells and progenitor cells travel freely between the lungs and bone marrow, long considered the primary source of blood production.

If found to occur in humans, this discovery about the lungs role in blood production could provide new approaches for treating blood diseases, pulmonologist Mark R. Looney, M.D., senior author of the study, said in a statement.

Moreover, the success of lung transplantation might be increased by better understanding this process. Immune reaction between donor blood cells in the lungs and the host could contribute to transplant rejection, the study stated.

The study was published Wednesday in the journal Nature. When placed online, the study can be found at j.mp/lungblood.

"This finding definitely suggests a more sophisticated view of the lungs -- that they're not just for respiration but also a key partner in formation of crucial aspects of the blood," Looney said. "What we've observed here in mice strongly suggests the lung may play a key role in blood formation in humans as well."

"Dr. Looney and his team have disrupted some traditional ideas about the pulmonary role in platelet-related hematopoiesis, paving the way for further scientific exploration of this integrated biology," said Traci Mondoro, of the National Heart, Lung and Blood Institute, in the statement.

While it has been known for decades that platelets can be made in the lungs, the study indicates that lung production is a more important factor than previously thought, said Mondoro, project officer at the Translational Blood Science and Resources Branch of the NHLBI, a division of the National Institutes of Health.

Researchers studied the lungs of mice genetically engineered to make a green fluorescent protein in platelets and platelet-making cells called megakaryocytes. They found a larger than expected number of these cells.

Megakaryocytes that release platelets in the lungs originate from extrapulmonary sites such as the bone marrow; we observed large megakaryocytes migrating out of the bone marrow space, the study said. The contribution of the lungs to platelet biogenesis is substantial,accounting for approximately 50% of total platelet production or 10 million platelets per hour.

After discovering this process, the researchers looked for more signs of blood cells residing in the lungs. They found progenitor cells that turn into megakaryocytes, along with blood-forming, or hematopoietic, stem cells. a total of 1 million per mouse lung.

These cells constitute a reservoir that can replenish the bone marrow, the study said.

Under conditions of thrombocytopenia (platelet deficiency) and relative stem cell deficiency in the bone marrow, these progenitors can migrate out of the lungs, repopulate the bone marrow, completely reconstitute blood platelet counts, and contribute to multiple hematopoietic lineages, the study stated. These results identify the lungs as a primary site of terminal platelet production and an organ with considerable hematopoietic potential.

The studys co-first authors are Emma Lefranais and Guadalupe Ortiz-Muoz, both of UCSF. It was supported by the UCSF Nina Ireland Program in Lung Health; the UCSF Program for Breakthrough Biomedical Research, and the National Heart, Lung, and Blood Institute.

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