Brain balls sound like something straight out of a Tim Burton movie: starting as stem cells harvested from patients, they eventually develop into masses of living neurons, jumbled together in misshapen blobs.

Just like the developing brain, these neurons stretch and grow, reaching out skinny branches that grab onto others to form synapsesjunctions where one neuron talks with the next.

And they do talk: previous attempts at growing these brain organoids found that they spark with electrical activity, much like the webs of neurons inside our heads that lead to thoughts and memories.

Theyre creepy. Theyre fascinating. And they may be neuroscientists best bet at modeling developmental disorders like autism in a dish.

Last week, two studies published in the prestigious journal Nature argued for brain balls as a reductionist model for broken brains. In one study, scientists took skin cells from patients with Timothy syndrome, a devastating neurodevelopmental disorder that often ends with childhood death, and grew them into brain balls to study where and how the developing brain veered off track.

In a separate paper, researchers used cutting-edge technology to profile the inhabitants of brain balls as they matured for eight months in a dish. Heres a creepy teaser: some blobs contained retinal neurons that normally allow us to see. Brain balls with eyes?!

As bizarre as that sounds, the fact that brain balls can develop a variety of neuron types with densely packed synapses is a win. Because theyre made from human cells, brain balls may eventually mimic diseases like schizophrenia, autism, or Alzheimers better than mouse models, revealing what went wrong and offering ample test grounds for potential treatments.

Weve never been able to recapitulate these human-brain developmental events in a dish before, says Dr. Sergiu Pasca at Stanford, who led the Timothy syndrome study. Our method lets us see the entire movie, not just snapshots.

Brain balls, better known by their scientific name cerebral organoids, first came onto the neurodevelopmental scene in 2013.

They often begin their short life as run-of-the-mill skin cells. Scientists first transform them back into stem cells. Then, using a chemical concoction of nutrients and signaling molecules, the stem cells are pushed to spontaneously assemble into little Frankenstein blobs of brain tissue.

But the process isnt just random bursts of division and growth. Rather, the way the brain balls mature roughly echoes how a fetuss cortex develops in the womb: the outer edges curl inward, forming outer and deeper layers.

What really sparked scientists interest was this: almost 90 percent of the neurons within a brain ball had active synapses, often spontaneously shooting electrical pulses to others in their network. While scientists believe brain balls arent capable of thinkingthe high-level cognitive processes constantly churning in our headstheyre definitely doing something.

To begin getting some answers, Dr. Paola Arlotta and team at Harvard followed a number of brain balls for nine months as they gradually maturedroughly the amount of time for human gestation, and much longer than any previous attempts.

Periodically, the researchers harvested more than 80,000 brain balls and ran sophisticated genetic tests to figure out their gene expression profile. Like law enforcement using DNA to match a perpetrators identity, this allowed researchers to profile the inhabitants of the organoids.

It was a cellular bonanza: as expected, excitatory neurons and non-neuronal cells called glia both made an appearance. More surprising were inhibitory neurons that dampen network activity, and cells that normally form the corpus callosum, a highway that connects the brains two hemispheres.

But creepiest by far, every single type of retinal cell also made an appearance. Although they couldnt really see in the normal sense, when bathed under light they did fire off electrical signals.

Just like a developing brain, the older they got the more complex the brain balls became. At eight months old, they contained roughly the same density of synapses as a human fetus cortex.

The cells connect witheach other, forming circuits, and once theyre connected, they can synchronize their activity, potentially mimicking higher-order functions of the human brain, says Arlotta.

Thats great, because it means mini brains could be used to study how different types of neurons connect with each other, and how disrupting the process leads to developmental problems.

Thats the direction the second study took. Rather than letting the mini brains grow wild, Pasca and team at Stanford tweaked the protocol to force them into different identities.

As a fetus brain grows, it gradually separates into an outer layer chock full of excitatory neurons, and an inner sanctum where inhibitory neurons reside. A big part of brain wiring is inhibitory neurons reaching out towards the surface and hooking up with their respective partners.

Starting from skin cells collected from patients with Timothy disease, the scientists used distinct chemical concoctions to form two batches of brain balls, each roughly 1/16 of an inch across and containing one million cells. One batch contained mostly inhibitory neurons, mimicking deeper brain regions, whereas the other modeled the cortex.

The spheroid cells were remarkably similar to those from corresponding regions of the human fetal brain, says Dr. J. Gray Camp and Dr. Barbara Treutlein at the Max Planck Institute in Germany, who were not involved in the studies.

The team then stuck the two types of brain blobs together into the same dish, and as expected, the inhibitory ball started nudging its way into the cortical one, until the two fused together.

As it turns out, the inhibitory neurons from Timothy patients were terrible migrants. Rather than smoothly slithering their way into the mesh of excitatory partners, they stuttered, stopped, but somehow ended up much further than theyre supposed to go, as if making up for their inefficiency.

The problem seemed to be the faulty neurons themselves, rather than defective signals from the environment. When researchers fused a Timothy inhibitory ball with a healthy excitatory one, they still fumbled without heads or tails.

But surprisingly, when treated with a chemical normally used for high blood pressure, the Timothy balls calmed down and migrated normally.

Spheroids are opening up new windows through which we can view the normal development of the fetal human brain, says Pasca. More importantly, it will help us see how this goes awry in individual patients.

While the scientists dont know whether the same drug could help babies with Timothy after theyre bornand their basic brain wiring already establishedPasca hopes that there may be a window of opportunity later on in life to correct the misguided migration.

All said, brain balls are an extremely reductionist model of the human brain. Although its hard to say whether the root of Timothy disease is faulty inhibitory neuron migration, its a great place to start looking for answers.

Pasca is rushing to speed up the process of growing spheroids, hoping to develop a giant depository harvested from many patients to screen for drugs that steers them towards a normal developmental path.

Others are a bit more cautious. These new studies show that brain balls whipped up from the same patient or patients with the same disease can express very different genes, warned Camp and Treutlein. The problem is likely more prominent in neurodevelopmental disorders like autism, in which the cause is a lot more heterogeneous.

But the fact that brain organoids behave like actual brains on several fundamental functionsmaking connections, spontaneously firing, responsive to external cuesis promising, so much so that theyre sparking intense ethical debates. Can they eventually see or think? Do they feel? Will consciousness spontaneously emerge without us detecting it?

For now, the mini brains are simply too tiny for higher-level thinking. Only time will tell what theyll eventually become, and how much information these mini brains can provide, says Camp and Treutlein.

Image Credit:PascaLab

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Bizarre Mini Brains Offer a Fascinating New Look at the Brain - Singularity Hub

Skin Stem Cells Comments Off on Bizarre Mini Brains Offer a Fascinating New Look at the Brain – Singularity Hub | May 16th, 2017

Cardiovascular disease is the number one cause of death worldwide in men, women and children, claiming more than 17 million lives each year. The effects of congestive heart failure and acute myocardial infarction (heart attack) present great challenges for doctors and researchers alike.

In this section:

Heart attacks cause damage to the heart muscle, making it less efficient at pumping blood throughout the circulatory system.

Your heart is constructed of several types of cells. For mending damaged heart tissue, researchers generally focus on three specific heart cell types:

Gladstone Institutes. Close up of a mouse heart stained to reveal the important structural protein that helps heart muscle cells to contract (red). The cell nuclei are labeled in magenta.

Despite major advances in how heart disease is managed, heart disease is progressive. Once heart cells are damaged, they cannot be replaced efficiently, at least not as we understand the heart today.

There is evidence that the heart has some repair capability, but that ability is limited and not yet well understood.

Heart failure is a general term to describe a condition in which the hearts blood-pumping action is weaker than normal. How much weaker varies widely from person to person, but the weakness typically gets worse over time. Blood circulates more slowly, pressure in the heart increases, and the heart is unable to pump enough oxygen and other nutrients to the rest of the body. To compensate, the chambers of the heart may stretch to hold more blood, or the walls of the chambers may thicken and become stiff. Eventually, the kidneys respond to the weaker blood-pumping action by retaining more water and salt, and fluid can build up in the arms, legs, ankles, feet, and even around the lungs. This general clinical picture is called congestive heart failure.

Many conditions can lead to congestive heart failure. Among the most common are:

The American Heart Association defines normal blood pressure for an adult as 120/80 or lower. What do those numbers mean? The top number is the systolic pressure that is, the pressure in your arteries when your heart beats, or contracts. The bottom number measures diastolic pressure, or the pressure in your arteries between beats, when the heart refills with blood.

In the early stages of congestive heart failure, treatment focuses on lifestyle changes (healthy diet, regular exercise, quitting smoking, etc.) and specific medications; the goals are to slow down any progression of the disease, lessen symptoms and improve quality of life.

Medications called beta blockers are often prescribed after a heart attack or to treat high blood pressure. Other medications called ACE inhibitors prevent heart failure from progressing.

For moderate to severe congestive heart failure, surgery may be necessary to repair or replace heart valves or to bypass coronary arteries with grafts. In severe cases, patients may be put on fluid and salt restriction and/or have pacemakers or defibrillators implanted to control heart rhythms.

Acute myocardial infarction, or a heart attack, occurs when the blood vessels that feed the heart are blocked, often by a blood clot that forms on top of the blockage. The blockage is a build-up of plaque that is composed of fat, cholesterol, calcium and other elements found in the blood. Without oxygen and other nutrients from the blood, heart cells die, and large swaths of heart tissue are damaged.

After a heart attack, scar tissue often forms over the damaged part of the heart muscle, and this scar tissue impairs the hearts ability to keep beating normally and pumping blood efficiently. The heart ends up working harder, which weakens the remaining healthy sections of the heart; over time, the patient experiences more heart-related health issues.

Doctors often use a procedure called angioplasty to disrupt the blood clot and widen clogged arteries. Angioplasty involves inserting and inflating a tiny balloon into the affected artery. Sometimes this temporary measure is enough to restore blood flow. However, angioplasty is often combined with the insertion of a small wire mesh tube called a stent, which helps keep the artery open and reduces the chances that it will get blocked again.

Other post-heart attack treatments include the regular use of blood thinners (for example, low-dose aspirin) to prevent new clots from forming and other medications to help control blood pressure and blood cholesterol levels. Lifestyle changes, such as lowering salt and fat intake, exercising regularly, reducing alcohol consumption and quitting smoking are also recommended to reduce the chances of a subsequent heart attack.

Scientists and clinicians have long suspected and recently confirmed that a persons genetic makeup contributes to the likelihood of their having a heart attack. Learn more here

The goals of heart disease research are to understand in greater detail what happens in heart disease and why, and to find ways to prevent damage or to repair or replace damaged heart tissue. Scientists have learned much about how the heart works and the roles different cells play in both normal function and in disease, and they are learning more about how cardiomyocytes and cardiac pacemaker cells operate, including how they communicate with each other and how they behave when damage occurs.

Researchers grow cardiomyocytes in the lab from the following sources:

These cells will beat in unison in a culture dish, the same way they do in a living heart muscle. This is exciting to consider, as researchers explore whether they might someday grow replacement tissue for transplantation into patients. However, it is not yet known whether lab-grown cardiomyocytes will integrate or beat in unison with surrounding cells if they are transplanted into the human body.

Gordon Keller Lab. Heart cells beating in a culture dish.

Scientists also use various types of stem cells to study the hearts natural repair mechanisms and test ways to enhance those repair functions. The evidence we have so far suggest thats the heart may have a limited number of cardiac stem cells that may conduct some repair and replacement functions throughout an individuals life, but we dont know where they live in the heart or how they become activated.

Human cells made from iPS cells are also incredibly useful for creating human models of heart disease to get a better understanding of exactly what goes wrong and for testing different drugs or other treatments. They can also be used to help predict which patients might have toxic cardiac side effects from drugs for other diseases such as cancer.

The key to treating heart disease is finding a way to undo the damage to the heart. Researchers are trying several tactics with stem cells to repair or replace the damaged heart tissue caused by congestive heart failure and heart attacks.

Areas under investigation include:

The Europe-wide BAMI clinical trial (the effect of intracoronary reinfusion of bone marrow-derived mononuclear cells on all-cause mortality in acute myocardial infarction) that began in 2014, is testing the infusion of cells from the participants bone marrow into one of the coronary arteries (one of two major arteries that supply the heart) to spark repair activity. However, it is not yet clear whether these cells will support heart repair function or in what way.

Researchers are also exploring transplantation of cardiomyocytes generated from both iPS cells and cardiac progenitor cells. They need to determine whether these transplanted cells survive and function in the body and whether they help speed up the hearts innate repair mechanisms.

Some of these approaches are still being evaluated in the lab while others are already being tested in clinical trials around the world. However, these trials are in their early stages and the results will not be clear for many years. Indeed, some published data conflict in critical ways, so carefully designed and well-monitored trials are key to working out what is safe and effective.

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Heart disease on Stem Cells - ISSCR

Cardiac Stem Cells Comments Off on Heart disease on Stem Cells – ISSCR | May 15th, 2017

Stem cell transplants may advance ALS treatment by repair of blood-spinal cord barrier Science Daily Using stem cells harvested from human bone marrow, researchers transplanted cells into mice modeling ALS and already showing disease symptoms. The transplanted stem cells differentiated and attached to vascular walls of many capillaries, beginning the ... and more »

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A new biomimetic bone tissue may help improve bone marrow transplants.

Engineers at the University of California San Diego have developed a bone-like implant that eliminates the need for donor cells to wipe out the hosts pre-existing cells, by allowing donor cells the space to live and grow.

Weve made an accessory bone that can separately accommodate donor cells. This way, we can keep the host cells and bypass irradiation, bioengineering professor Shyni Varghese, from the UC San Diego Jacobs School of Engineering, said in a statement.

The implants are made of a porous hydrogel matrix that contains calcium phosphate minerals in the outer matrix and donor stem cells that produce blood cells in the inner matrix.

The researchers successfully tested the bone tissues in mice and the donor cells survived for at least six months, while supplying the mice with new blood cells.

The structures matured into bone tissues of the mice that have a working blood vessel network and a bone marrow inside that supplies new blood cells. After a month the implanted marrow contained a mixture of host and donor blood cells, which remained circulating in the bloodstream even after 24 hours.

In the future, our work could contribute to improved therapies for bone marrow disease, Yu-Ru (Vernon) Shih, a research scientist in Vargheses lab and the studys first author said in a statement. That would have useful applications for cell transplantations in the clinic.

The researchers also took stem cells from the implanted marrow and transplanted them into another group of mice with their marrow stem cells eradicated by radiation and drugs. The transplanted cells diffused into the bloodstream of the mice in the second group.

Were working on making this a platform to generate more bone marrow stem cells, Varghese said.

According to Varghese, the implants could only be used in patients with non-malignant bone marrow diseases, where there arent any cancerous cells that need to be eliminated.

The researchers said this discovery indicates that implanted marrow is functional and donor cells can form and survive for long periods of time in the presence of host cells. They also said that the host and donor cells can travel between the implanted marrow and the hosts circulating blood through the blood vessel network formed in the implanted bone tissue.

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Engineered Bone Marrow Improves Transplant Safety - R & D Magazine

Bone Marrow Stem Cells Comments Off on Engineered Bone Marrow Improves Transplant Safety – R & D Magazine | May 12th, 2017

Harvard Gazette

Researchers work to create kidney filtration barrier on a chip ... Harvard Gazette Researchers say their glomerulus-on-a-chip lined by human stem cell-derived kidney cells could help model patient-specific kidney diseases and guide ... and more »

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Researchers work to create kidney filtration barrier on a chip ... - Harvard Gazette

IPS Cell Therapy Comments Off on Researchers work to create kidney filtration barrier on a chip … – Harvard Gazette | May 12th, 2017

Induced pluripotent stem cells have revolutionized stem cell science in the decade since their invention. Theyre yielding clues into the nature of diseases such as cancer and Alzheimers, and are also being tapped for therapy.

But creating these IPS cells is lengthy, complicated and tricky, and the facilities equipped to make them cant accommodate all the scientists whod like to get their hands on them.

A UK-led consortium has removed that bottleneck, by producing 711 lines of ready-to-go IPS cells from healthy individuals. These lines are meant to help scientists understand the normal variations between healthy individuals and those involved in disease, as well as to understand normal human biology and development.

The IPS lines are available for research purposes to academic scientists and industry by contacting the Human Induced Pluripotent Stem Cell Initiative (HipSci), at http://www.hipsci.org and the European Bank for induced Pluripotent Stem Cells at https://www.ebisc.org.

The accomplishment was announced in a study published in Nature. It can be found online at j.mp/711ips.

While many other efforts have generated IPS cells to address rare diseases, this study produces them from healthy volunteers to plumb common genetic variation, Fiona Watt, a lead author on the paper and co-principal investigator of HipSci, from King's College London, said in a statement.

"We were able to show similar characteristics of iPS cells from the same person, and revealed that up to 46 per cent of the differences we saw in iPS cells were due to differences between individuals, Watt said in the statement. These data will allow researchers to put disease variations in context with healthy people."

Andrs Bratt-Leal, director of the Parkinson's Cell Therapy Program at The Scripps Research Institute in La Jolla, agreed.

This kind of study is extremely important because it leads to a deeper understanding of the differences between normal genetic variation and genetic changes that could negatively impact cell behavior, said Bratt-Leal, who was not involved in the study.

This data will help scientists using induced pluripotent stem cells to model diseases as well as scientists developing cell therapies, said Bratt-Leal, who works in the lab of stem cell researcher Jeanne Loring.

Because DNA sequencing has become a routine tool in the lab, enormous amounts of data have been produced, he said. Not only have we have observed a high level of genetic diversity between different people, but also a more subtle variation exists among the cells from an individual person. The next step is a better understanding of how this diversity translates to function and behavior of stem cells and mature cells derived from stem cells.

Loring and Bratt-Leal are studying the use of induced pluripotent stem cells to relieve symptoms of Parkinsons disease. They are in the process of translating the research into a therapy, aided with a grant from the California Institute for Regenerative Medicine.

The work was the product of a large-scale collaboration of scientists from various institutions in the United Kingdom, including the European Molecular Biology Laboratory in Cambridge; Wellcome Trust Sanger Institute in Cambridge; the University of Dundee in Dundee; and the University of Cambridge. Also participating was St Vincent's Institute of Medical Research in Victoria, Australia.

bradley.fikes@sduniontribune.com

(619) 293-1020

UPDATES:

1:00 p.m.: This article was updated with additional details.

This article was originally published at 10:00 a.m.

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Hundreds of new stem cell lines ready to help research - The San Diego Union-Tribune

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Skin cells found at root of balding, gray hair Science Daily The researchers found that a protein called KROX20, more commonly associated with nerve development, in this case turns on in skin cells that become the hair shaft. These hair precursor, or progenitor, cells then produce a protein called stem cell ...

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Brigham and Womens Hospital (BWH) will pay $10 million to resolve allegations that one of their stem cell research laboratories fraudulently obtained grant funding from the National Institutes of Health (NIH), according to a press release.

As per federal regulations and institutional policy requirements, BWH conducted an investigation that identified data integrity concerns in federally funded grant applications submitted by the Anversa lab. After learning of and investigating the allegations of misconduct in the Anversa laboratory, BWH disclosed its concerns to the U.S. Department of Health and Human Services, Office of the Inspector General, and Office of Research Integrity.

BWH independently evaluated the issues relative to the federal false claims requirements, said Lori Schroth, media relations manager at BWH. Following that evaluation, BWH self-disclosed this matter to appropriate government entities and ceased drawing implicated funds.

The settlement resolves the allegations against Dr. Piero Anversa, who ran the laboratory, and Drs. Annarossa Leri and Jan Kajstura. Allegedly, the doctors knew or should have known that their laboratory published and relied upon manipulated and falsified information including microscope images and carbon-14 age data for cells, according to the press release. This information was used in applications for NIH research grant awards concerning the purported ability of stem cells to repair damage to the heart.

The settlement also resolves allegations that the laboratory followed improper protocols, inaccurately characterized cardiac stem cells, and kept recklessly or deliberately misleading records, according to the press release.

Drs. Anversa, Leri, and Kajstura are no longer affiliated with BWH, and the lab has since been closed.

BWH is committed to ensuring that research conducted at the institution is done under the most rigorous scientific standards, and has made significant enhancements to research integrity compliance protocols as a result of this event, said Schroth.

Acting U.S. Attorney William D. Weinreb said in the press release that individuals and institutions that receive research funding from NIH have an obligation to conduct their research honestly and not to alter results to conform with unproven hypotheses.

Medical research fraud not only wastes scarce government resources but also undermines the scientific process and the search for better treatments for serious diseases, Weinreb said, according to the press release. We commend Brigham and Womens for self-disclosing the allegations of fraudulent research at the Anversa laboratory, and for taking steps to prevent future recurrences of such conduct.

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BWH settles research fraud allegations - Mission Hill Gazette

Cardiac Stem Cells Comments Off on BWH settles research fraud allegations – Mission Hill Gazette | May 6th, 2017

Home Health News Mouse teeth shown to hold insight into future stem cell tissue regeneration

The use of stem cells throughout the years has been both a decisive topic and one that holds a lot of promise for potential medical therapy. They are essentially undifferentiated biological cells that havent yet been specialized for a specific purpose. The cells of your heart, stomach, and even your brain have all started out as stem cells, and it wasnt until some point during human development that biological processes channeled them to permanently becoming one type of cell. Scientists and researchers around the globe are always in search of the best way to learn about and harvest these valuable cells, and the latest reports suggest the teeth of rodents are an abundant source.

There are considered two main stem cell types in the body: one is from embryonic development when in the womb, and the other are adult stem cells that exist throughout the body. Harvesting embryonic stem cells has been controversial, as it often seen as unethical, but adult stem cellsfound in organs such as the bone marrow, blood vessel, and liver in mammalsis easier to obtain. Stomach linings, for example, require the constant shedding of their cell linings as the acid wears away at them, and having adult stems cells allows for quick replacement of these sloughed off cells.

Weve all seen mice before, and one of their defining characteristics are their front teeth. What most people arent aware of is that their front teeth, or incisors, constantly grow, as they rely on them to be consistently sharp for burrowing and self-defense, and of course, for eating away at your pantry food. As we grow older our teeth start to wear out, and in nature, once you dont have your teeth anymore, you die. As a result, mice and many other animals from elephants to some primates can grow their teeth continuously. Our labs objective is to learn the rules that let mouse incisors grow continuously to help us one day grow teeth in the lab, but also to help us identify general principles that could enable us to understand the processes of tissue renewal much more broadly, said UC San Franciscos Ophir Klein, MD, Ph.D., a professor of orofacial sciences in UCSFs School of Dentistry and of pediatrics in the School of Medicine.

While not all aspects of this process are fully understood just yet, as the exact signals triggering this process have yet to be identified. It, however, marks an advancement of knowledge in the field, and one that bodes well for the future of stem cell therapy. It may prove beneficial for tissue regeneration to treat everything from severe burns to growing entire organs from scratch.

Related:Stem cells from fat may be useful to prevent aging

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https://www.ucsf.edu/news/2017/04/406836/mouse-teeth-providing-new-insights-tissue-regeneration http://www.sciencedirect.com/science/article/pii/S1934590917300942 http://www.medicalnewstoday.com/info/stem_cell

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Mouse teeth shown to hold insight into future stem cell tissue regeneration - Bel Marra Health

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Few things could start out so simple and yet maybe lead you to save a life. But one woman's firsthand experience led precisely down that road, and now she's getting more people on board.

Be the Match is a program offering free kits for people to submit their DNA through mouth swabs to see if they might one day be a match to donate to someone with blood cancers or other blood-based diseases.

And getting that opportunity is about to become even easier for those in the Roaring Fork Valley this coming First Friday (May 5) and then on Dandelion Day (May 13) in Carbondale.

Erica Borum, who works as a civil engineer for the White River National Forest, is setting up a Be the Match booth at these events, where she'll have registry kits with mouth swabs ready to be used and sent off.

This first step is simple. If you're between 18 and 44 you can participate, but this first step doesn't mean that you're automatically going to be donating to someone.

Your mouth swab puts you on a DNA registry. Once your DNA is on the list, that information is available to doctors looking for donors who match with patients in need for stem cells or bone marrow.

According to Be the Match, each year about 14,000 patients are down to one option for a cure: a transplant from someone outside their family.

Borum is pushing this effort in Carbondale after her own experience a little more than a year ago donating to a man with Hodgkin's lymphoma. She wants to give people this opportunity by bringing the kits to them, but she's also on a mission to demystify the process.

"When people are first addressed with it, it's strange and weird and not something that would be of interest," she said. "I'd like to help make it not so foreign."

Borum first heard about this donation process through a friend, who knew of someone needing a bone marrow transplant for leukemia. The patient was down to her last option, but in the end the procedure worked out for her.

Borum got online and started doing her research on the organization Be the Match.

The chances that you'll be selected are actually quite slim. This isn't as simple as finding a person with matching blood type. Because doctors are looking for someone with highly specific blood markers and other characteristics to give the patient the best shot possible for a good transfer, only about one in 430 people end up being suitable match, she said.

There are two types of transfers: a transfer of peripheral blood stem cells or a bone marrow transplant. Ultimately, the patient's doctor choses which route to go, so donors need to be willing to do either.

Donating blood stem cells, which is what Borum did, is a bit like an extended blood draw.

"The first thing to know is my phobia is needles," said Borum. "But knowing that phobias are completely illogical, I went ahead and sent off my swabs. And there wasn't a great chance that I would be a match anyway.

"The prospect of going through a process that's a little uncomfortable for the benefit of saving someone, it's kind of overwhelming," she said. "To be honest, I didn't give it a second consideration, despite the phobia."

She joined the registry in 2013. The mouth swab process took only about 10 minutes, she said.

A little more than two years later, Borum got that call that she was a preliminary match. Did she want to proceed with the process?

Even agreeing at this point isn't the final say in whether you end up donating. First, Borum had her blood drawn and sent off to reinforce that, yes, she was a match. And after that was confirmed she had to get a physical and undergo some pathogen testing, a chest X-ray and some additional testing to make sure she was healthy.

During this process, the identity of the patient is guarded. All Borum was told was that the patient was a 56-year-old man with Hodgkin's lymphoma.

From there it was a repeated process of "test and we'll let you know, test and we'll let you know," she said.

'ONE POKE AT A TIME'

And as each of those came back good, they check to see if you want to proceed. The donor can pull out at any time. But while the donor is going through this process, the patient is going through a parallel preparation of intensive chemotherapy trying to kill as much of the cancer as possible before the transplant.

This is a critical stage for the patient, and if the donor opts out now, it could be life-threatening for the patient, she said.

"I took it one needle poke at a time."

Leading up to the final blood draw, Borum was given several injections of a drug to boost her cell count to help doctors withdraw the stem cells they were looking for.

On the long end, physicians say the blood draw process could take up to six hours. That was their prediction for Borum, who is more petite. During that draw, they run the first samples to a lab to confirm they're getting the right concentration. Borum's body reacted well to the blood cell-boosting drug, and the final process ended up only taking four hours.

To explain her motivation to go through with this procedure, Borum said it's part of her spiritual practice.

"I did it as part of the practice, and recognizing that a lot of things we do are self-referential and self-serving. And I don't think there's a ton of benefit in that, for myself and for others.

"I think the quote from the Dalai Lama goes: If you want others to be happy, have compassion; if you want to be happy, have compassion.

"And it's all a matter of statistics. If there are more people in the registry, it's more likely someone will have a match. If I can benefit one person, it's little effort on my end to try to boost the numbers."

Because of the statistically low chances of finding a match, getting more people on the registry is essential. And the first month or two after the final transplant is a time critical to find out whether their new blood stem cells are working. This doesn't always have a happy ending, said Borum.

But in her case, it did.

BUILDING A BOND

Her transfer was a little more than a year ago. About two months afterward she got word that her cells were successfully making the patient's blood and he had no sign of cancer cells. The patient, who Borum learned lives in New York, sent her a thank you card a few months later, and recently they've exchanged emails.

And in those exchanges, she got her first glimpses into his life.

"I am a husband of 28 years and the father of two boys and was facing a difficult future," he wrote in April of last year. "I am overwhelmed beyond words with this gift you have given me. Please know that I will live the rest of my life with the warmth of your generosity and will do my best to extend it to others in need."

"His email said that he's now been healthy for one year and two months," said Borum. He's also offered to come to Colorado to meet Borum and thank her in person.

His sickness had taken him to a point where walking was a very difficult task, but now he's running again.

The whole process, they say, is about 20 to 30 hours of your time, usually spread over about four to six weeks, according to Be the Match.

"I think that's a small sacrifice to potentially save someone," said Borum.

The patient in this case wasn't available for this article but for pretty much the best reason ever.

Borum said that after being too sick for so long, he was finally leaving for a long-awaited vacation.

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How you can give yourself a chance to save another's life - Glenwood Springs Post Independent

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Caroline, 11, Elizabeth, 3, Jon Thomas, 13 and James Christopher Allums, 20, do everything as a family. James Christopher and Elizabeth both have a rare medical condition. Their mother, Ellen Allums, said they all go through the process together and support each other with faith and love.(Photo: Courtesy)

Learning that your child has a rare, life-threatening illness is difficult for any family. Everything changes. One family learned that two of their children share the same rare blood disorder.

"That news that we heard was the worst news that we could hear, but it was the best thing that's ever happened to us. It really changed our perspective. It changed our priorities," Chris Allums said.

"We're no longer the same people we were," Ellen Allums said.

Ellen and Chrishave four childrenJames Christopher, 20,Jon Thomas, 13,Caroline,11, andElizabeth, 3.

James Christopher and Elizabeth have Fanconi anemia, a disease that affects the bone marrow's ability to produce blood. Bone marrow or blood stem cell transplants are considered the best treatments, andthey have not yet found a match for either child.

James Christopher was diagnosed 12 years ago and told he had about 18 monthsto live. The family was told he must received a bone marrow transplant.

"We immediately decided that, first of all, we're going to pray and expect a miracle and grow our faith, and next, we're going to try and see if we can find him a bone marrow match and help others along the way, see how many lives that we can affect, that we can save both spiritually and physically," Ellen said.

More than 16,000 people have been added to the worldwide bone marrow registry as a result of drives held on behalf of the Allums. Ellen said they know of at least 41 lives that have been saved because of those efforts, and they're asking more people to commit to donate.

'Looking for a double miracle'

Ellen said a doctor said someone with FA can be like a duck gliding on the water the surface appearance is calm, but people can't see all the effort that goes into staying in motion.

It has a variety of symptoms such as fatigueand can lead to bone marrow or organ failure. Ellen and Chris said FA patients are 500 times more likely to develop some cancers, such as leukemia. James Christopher is subject to constant screenings.

The disease is genetic. According to the National Organization for Rare Disorders, the incidence rate is 1 in 136,000 births. Ellen said her children are two ofsix in Louisiana affected by FA.

Elizabeth's blood counts have been OK, but doctors have said James Christopher has an immediate need for a transplant. DNA needs to be close to an exact match, and many families find a relative who can donate.Elizabeth is a 100 percent match, but she's ineligible because of her FA.

One donor, once found, could help both.A bone marrow transplant won't cure someone with FA, but it can help prolong life.

"Just because you're having to wait doesn't mean the miracle's not going to come. We've been waiting 12 years, but we still have faith that that miracle's coming. Just because it hasn't happened doesn't mean it's not going to. The timing needs to be right," Ellen said. "In our lives, we're looking for an even greater miracle because we're looking for a double miracle, with two children."

FA patients can require blood and platelet transfusions, after which they may become dependent and need additional rounds, which would require a bone marrow transplant quickly.

James Christopher received his first blood transfusion three weeks ago.

"Chris gave. His daddy gave blood to him, and we felt like it was his heavenly father and his earthly father that gave him that blood, and now we're praying and believing that he never has to receive it again," Ellen said.

She said they've dealt with some scary bleeding issues "like Niagara Falls," and James Christopher has almost lost his life a few times. His parents call him a survivor, a warrior. He gets up and stays active daily, even with low blood counts that doctors thinkwould cause fatigue.

"I love to prove doctors wrong. If they give me a boundary, I want to cross it, definitely, when it comes to that," he said. He likes to tell people "keep calm and carry on," like the World War II posters.

Every bump, scratch, scrape and bruise for the siblings is noteworthy, and the whole family works to avoid germs. A simple cough or cold could be devastating, so they're all in tune to notice illness.They're very aware of the importance of handwashing and staying home if ill. Chris said during cold and flu season, they often come in, shower and change clothes before interacting with the others.

Ellen said they respect people who choose not to vaccinate, but all of her children have been vaccinated because measles or chicken pox can kill someone with FA.

All the children home school to help prevent illness. When James Christopher was diagnosed, doctors said it could help him live longer. Chris said all four have excelled fromthe one-on-one time, and they've enjoyed getting to know other families inthe Christian Homeschool Association.

The Allums know their lives are different than those of many other families, but they are running their own race.

"I have to tell you that we have a wonderful life. Sure it's full of hard work, but it's wonderful because of what the Lord has done with it," Chris said.

Read more:Mom says prayer pulled her through transplant|Facing the storm: Mother shares unbelievable story|Big brother to the rescue: Man gives sister half of liver|Man saves 10 in life, death

Joy in the journey

The couple did their homework on hospitals that specialize in the disease and settled on Memorial Sloan Kettering Hospital's cancer center in New York. It had the best survival rates, and they've been going for 12 years.

James Christopher's and Elizabeth's immunity is low, the family cannot travel with the general public. They either have to make the almost 20-hour drive or arrange for a private plane. Ellen said they've had to go there, at times, every three to six months.

The whole family travels to medical appointments.

"Although they don't have the disease, they go through it with them," Ellen said of Jon Thomas and Caroline. She said all of her children have gone to hospitals and played with and prayed for children were facing terminal diagnoses. It's been a blessing to them and a ministry to others.

James Christopher said they try to find fun in the journey. Ellen said they do something fun every time they go to the hospital and embrace John 10:10Jesus came that we might have life and have it abundantly.

James Christopher Allums, 20, holds his sister Elizabeth Allums, 3. The siblings both have a rare medical condition called Fanconi anemia.(Photo: Courtesy)

What happens if there's a match?

"We would be moving to New York for six to eight months for the bone marrow transplant," Ellen said.

Ellen said the a bone marrow recipient with FA will have to go through chemotheraphy for two weeks to kill off the patient's natural bone marrow.

"When the cells are dead, then they receive someone else's bone marrow. It's a liquid, it looks just like an IV, and they lie there and you just pray to God that it's going to take," she said.

After the transplant, the patient is in isolation for 30-40 days. They stay at the transplant hospital for six to eight months and keep a medical mask on for one year. Chris said you hope graph vs. host disease isn't an issue.

Saving lives

She said she used to look at missions that dig wells in other countries and wish they could go save lives, but, after prayer, she realized they are saving people. With the help of family and friends, efforts to add bone marrow donors have helped dozens of people.

"I like to tell people 'You could be the reason someone lives.' ... And I think those words are pretty powerful" Ellen said.

She said the process to donate blood stem cells, which is the most common donation method, involves a needle in each arm for four to six hours.

"It's not even a surgery. It's not like giving a kidney or a lung or a heart, even, but the benefits are that strong. It can truly save a life, but yet all you have to do is like giving blood," Ellen said.

To test for a match, she said, it's even less of a commitment. It takes about five minutes to fill out paperwork and provide a swap from inside the cheek. Anyone 18-55 in good health can register.

The community has come together to help organize a drive for May 1, National Fanconi Anemia Day. A massive drive will take place at more than a dozen locations across northeastern Louisiana, and CenturyLink will be registering employees on-site.Anyone anywhere can order testing kitsonline atdkms.orgorbethematch.org.

A month after testing, people will get a phone call to confirm their position on the registry. Ellen said they pray people will make the commitment.Previous drives for the Allumshave set national records for most registered in one day. Over three days, they tested 5,000 people.

"When people come, we want to educate them on the processin hopes thatwhetherthey are a match in a month or a match in 20 yearsthat they will be committed to beingon that registry to help somebody," Chris said.

They heard of a woman who registered with her family at a previous event andlater developed leukemia. Her sister was found as an instant bone marrow match because theyalreadyhad been tested.

Ellen and Chris said knowing that 41 lives were saved as a result of their family'sefforts makes it all worth it, even though it hasn't been easy.

"But we believe that God is going to heal them both because He told us He would, and we believe that. We hold on to those promises of God. ... and we focus on that. That gives us strength," Ellen said.

Follow Bonnie Bolden on Twitter@Bonnie_Bolden_and on Facebook athttp://on.fb.me/1RtsEEP.

Want to register?

May 1 is National Fanconi Anemia Day, and a more than a dozen locations across northeastern Louisiana will be part of a single registration drive. Times vary and new locations may be added. Check The Friends of James Christopher and Elizabeth Allumson Facebook or visitcaringbridge.organd searchJames Christopher Allums.

Or order a testing kit online at dkms.org or bethematch.org.

Testing sites and times are:

Monroe

West Monroe

Surrounding parishes

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Family seeks 'miracle' for siblings, saves lives in the process - Monroe News Star

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Jewish Chronicle

Bone marrow donor forgot he'd registered Jewish Chronicle My phone rang and when I answered they said someone needed my stem cells. They asked me would I still like to donate? I went in the next day for tests and when I was deemed fit and healthy they got me to come back in for the procedure. On Tuesday the ...

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Technology Networks

Stem Cells Edited to Fight Arthritis Technology Networks Such stem cells, known as SMART cells (Stem cells Modified for Autonomous Regenerative Therapy), develop into cartilage cells that produce a biologic anti-inflammatory drug that, ideally, will replace arthritic cartilage and simultaneously protect ... CRISPR-SMART Cells Regenerate Cartilage, Secrete Anti-Arthritis DrugGenetic Engineering & Biotechnology News all 5 news articles »

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Researchers have showncertain cardiac glycosides can reduce hepatocyte production of aprecursor of LDL cholesterol.

Familial hypercholesterolaemia (FH) is a rare genetic disease that affects the production of functioning low-density lipoprotein (LDL) receptors in the liver. When patients have mutations in both copies of the LDL receptor gene, they do not respond to statins and have limited pharmaceutical treatment options available because of a lack of accurate disease models.

Reporting in Cell Stem Cell on 6 April 2017[1], researchers used FH human hepatocytes derived from induced pluripotent stem cells to screen for existing drugs that might lower apolipoprotein B (apoB) a precursor of LDL cholesterol.

The team found that all nine cardiac glycosides in their drug library reduced levels of apoB in the hepatocytes. In an analysis of historical patient data, the researchers found a reduction in serum LDL-C comparable to that seen with statins in patients taking cardiac glycosides.

The researchers say the results demonstrate the potential of their stem-cell based approach for identifying new treatment candidates for inherited liver diseases.

Citation: Clinical Pharmacist, CP April 2017 online, online | DOI: 10.1211/CP.2017.20202623

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Stem-cell screening finds statin alternative for hypercholesterolaemia - The Pharmaceutical Journal

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Ottawa Citizen

Two weeks after his transplant, Jonathan Pitre battles kidney complications Ottawa Citizen Pitre, 16, was infused two weeks ago with stem-cell rich blood and bone marrow drawn from his mother's hip. The procedure, conducted as part of an ongoing clinical trial at the University of Minnesota Masonic Children's Hospital, is the only treatment ... and more »

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Two weeks after his transplant, Jonathan Pitre battles kidney complications - Ottawa Citizen

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(EnviroNews World News) Kobe, Japan For more than two million Americans, straight lines may look wavy and the vision in the center of their eye may slowly disappear. Its called age-related macular degeneration (AMD), and there is no cure. But that may change soon.

A surgical team at Kobe City Medical Center General Hospital in Japan recently injected 250,000 retinal pigment epithelial (RPE) cells into the right eye of a man in his 60s. The cells were derived from donor stem cells stored at Kyoto University. It marked the first time that retinal cells derived from a donors skin have been implanted in a patients eye. The skin cells had been reprogrammed into induced pluripotent stem cells (iPS), which can be grown into most cell types in the body.

The procedure is part of a safety study authorized by Japans Ministry of Health that will involve five patients. Each will be followed closely for one year and continue to receive follow-up exams for three additional years. Project leader Dr. Masayo Takahashi at Riken, a research institution that is part of the study, told the Japan Times, A key challenge in this case is to control rejection. We need to carefully continue treatment.

A previous procedure on a different patient in 2014 used stem cells from the individuals own skin. Two years later, the patient reported showing some improvement in eyesight. But the procedure cost $900,000, leading the study team to move forward using donor cells. They expect the costs to come down to less than $200,000.

Among people over 50 in developed countries, AMD is the leading cause of vision loss. According to the National Eye Institute, 14 percent of white Americans age 80 or older will suffer some form of AMD. The condition is almost three times more common among white adults than among people of color. Women of all races comprise 65 percent of AMD cases.

The lack of a cure has led some to try unproven treatments. Three elderly women lost their sight after paying $5,000 each for a stem cell procedure at a private clinic in Florida. Clinic staff used liposuction to remove fat from the womens bellies. They then extracted stem cells from the fat, which were injected into both eyes of each patient in the same procedure, resulting in vision loss in both eyes. Two of the three victims agreed to a lawsuit settlement with the company that owned the clinic.

Stem cell therapy is still at an early stage. As of January 2016, 10 clinical uses have been approved around the world, all using adult stem cells. These include some forms of leukemia and bone marrow disease, Hodgkin and non-Hodgkin lymphoma and some rare inherited disorders including sickle cell anemia. Stem cell transplants are now often used to treat multiple myeloma, which strikes more than 24,000 people a year in the U.S.

Clinical trials to treat type 1 diabetes, Parkinsons disease, stroke, brain tumors and other conditions are being conducted. The first patient in a nationwide clinical study to receive stem cell therapy for heart failure recently underwent the procedure at the University of Wisconsin School of Medicine and Public Health. An experimental treatment at Keck Medical Center of USC last year on a paralyzed patient restored the 21-year-old mans use of his arms and hands. Harvard scientists see stem cell biology as a path to counter aging and extend human lifespans. But the International Society for Stem Cell Research warns that there are many challenges ahead before these treatments are proven safe and effective.

The U.S. Food and Drug Administration (FDA) regulates stem cells to ensure that they are safe and effective for their intended use. But, that doesnt stop some clinics from preying on worried patients. The FDA warns on its website that the hope that patients have for cures not yet available may leave them vulnerable to unscrupulous providers of stem cell treatments that are illegal and potentially harmful.

While there is yet no magic cure for AMD, the Japan study and others may one day lead there. The Harvard Stem Cell Institute (HSCI) in Boston is currently researching retina stem cell transplants. One approach uses gene therapy to generate a molecule that preserves healthy vision. Another involves Muller cells, which give fish the ability to repair an injured retina.

But these therapies are far off. We are at about the halfway mark, but there is still a precipitous path ahead of us, Takahashi said.

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Tiny, 3-D clusters of human brain cells grown in a petri dish are providing hints about the origins of disorders like autism and epilepsy.

An experiment using these cell clusters which are only about the size of the head of a pin found that a genetic mutation associated with both autism and epilepsy kept developing cells from migrating normally from one cluster of brain cells to another, researchers report in the journal Nature.

"They were sort of left behind," says Dr. Sergiu Pasca, an assistant professor of psychiatry and behavioral sciences at Stanford. And that type of delay could be enough to disrupt the precise timing required for an actual brain to develop normally, he says.

The clusters often called minibrains, organoids or spheroids are created by transforming skin cells from a person into neural stem cells. These stem cells can then grow into structures like those found in the brain and even form networks of communicating cells.

Brain organoids cannot grow beyond a few millimeters in size or perform the functions of a complete brain. But they give scientists a way to study how parts of the brain develop during pregnancy.

"One can really understand both a process of normal human brain development, which we frankly don't understand very well, [and] also what goes wrong in the brain of patients affected by diseases," says Paola Arlotta, a professor of stem cell and regenerative biology at Harvard who was not involved in the cell migration study. Arlotta is an author of a second paper in Nature about creating a wide variety of brain cells in brain organoids.

Pasca's team began experimenting with organoids in an effort to learn more about brain disorders that begin long before birth. Animal brains are of limited use in this regard because they don't develop the way human brains do. And traditional brain cell cultures, which grow as a two-dimensional layer in a dish, don't develop the sort of networks and connections that are thought to go awry in disorders like autism, epilepsy and schizophrenia.

"So the question was really, can we capture in a dish more of these elaborate processes that are underlying brain development and brain function," Pasca says.

He was especially interested in a critical process that occurs when cells from deep in the brain migrate to areas nearer the surface. This usually happens during the second and third trimesters of pregnancy.

So Pasca's team set out to replicate this migration in a petri dish. They grew two types of clusters, representing both deep and surface areas of the forebrain. Then they placed deep clusters next to surface clusters to see whether cells would start migrating.

Pasca says the cells did migrate, in a surprising way. "They don't just simply crawl, but they actually jump," he says. "So they look for a few hours in the direction in which they want to move, they sort of decide on what they want to do, and then suddenly they make a jump."

Pasca suspected this migration process might be disrupted by a genetic disorder called Timothy syndrome, which can cause a form of autism and epilepsy. So he repeated the experiment, using stem cells derived from the skin cells of a person who had Timothy syndrome.

And sure enough, the cells carrying the genetic mutation didn't jump as far as healthy cells did. "They moved inefficiently," Pasca says.

Next Pasca wondered if there might be some way to fix the migration problem. He thought there might be, because Timothy syndrome causes cells to let in too much calcium. And he knew that several existing blood pressure drugs work by blocking calcium from entering cells.

So the team tried adding one of these calcium blockers to the petri dish containing clusters of brain cells that weren't migrating normally. And it worked. "If you do treat the cultures with this calcium blocker, you can actually restore the migration of cells in a dish," Pasca says.

Fixing the problem in a developing baby wouldn't be that simple, he says. But the experiment offers a powerful example of how brain organoids offer a way to not only see what's going wrong, but try drugs that might fix the problem.

Still, to realize their full potential, brain organoids need to get better, Arlotta says. This means finding ways to keep the cell clusters alive longer and allowing them to form more of the types of brain cells that are found in a mature brain.

Arlotta's team has developed techniques that allow brain cell clusters to continue growing and developing in a dish for many months. And what's remarkable, she says, is that over time the clusters automatically begin creating structures and networks like those in a developing brain.

"Using their own information from their genome, the cells can self-assemble and they can decide to become a variety of different cell types than you normally find," she says.

In one experiment, a brain organoid produced nearly all the cell types found in the mature retina, Arlotta says. And tests showed that some of these retinal cells even responded to light.

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'Minibrains' in a dish shed a little light on autism and epilepsy - 89.3 KPCC

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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By Marilynn MarchioneAP Chief Medical Writer

Tummy tucks really hurt. Doctors carve from hip to hip, slicing off skin, tightening muscles, tugging at innards. Patients often need strong painkillers for days or even weeks, but Mary Hernandez went home on just over-the-counter ibuprofen.

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

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

This growing dependence on opioids has mushroomed into a national health crisis, ripping apart communities and straining police and health departments. Every day, an overdose of prescription opioids or heroin kills 91 people, and legions more are brought back from the brink of death. With some 2 million Americans hooked on these pills, evidence is growing that they're not as good a choice for treating chronic pain as once thought.

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

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

Pain is a pain to research. Some people bear more than others, and success can't be measured as objectively as it can be with medicines that shrink a tumor or clear an infection. Some new pain drugs that worked well were doomed by side effects Vioxx, for instance, helped arthritis but hurt hearts.

Some fresh approaches are giving hope:

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

Drugs to prevent the need for opioids. One that Hernandez was helping test numbs a wound for a few days and curbs inflammation. If people don't have big pain after surgery, their nerves don't go on high alert and there's less chance of developing chronic pain that might require opioids.

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

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

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

One drug's bumpy road

In the early 2000s, a small biotech company had a big idea: blocking nerve growth factor, a protein made in response to pain. The company's drug, now called tanezumab (tah-NAZE-uh-mab), works on outlying nerves, helping to keep pain signals from muscles, skin and organs from reaching the spinal cord and brain good for treating arthritis and bad backs.

Pfizer Inc. bought the firm in 2006 and expanded testing. But in 2010, some people on tanezumab and similar drugs being tested by rivals needed joint replacements. Besides dulling pain, nerve growth factor may affect joint repair and regeneration, so a possible safety issue needed full investigation in a medicine that would be the first of its type ever sold, said one independent expert, Dr. Jianguo Cheng, a Cleveland Clinic pain specialist and science chief for the American Academy of Pain Medicine.

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

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

Her doctor, Alan Kivitz of Altoona Center for Clinical Research, has helped run hundreds of pain studies and consults for Pfizer and many other companies. "You rarely get people to feel that good" as many of them did on the nerve growth factor drugs, he said.

A drug with that much early promise is unusual, said Ken Verburg, who has led Pfizer's pain research for several decades.

"When you do see one, you fight hard to try to bring one to the market," he said.

An independent review ultimately tied just a few serious joint problems to tanezumab and the suspension on testing was lifted in August 2012. But a new issue nervous system effects in some animal studies prompted a second hold later that year, and that wasn't lifted until 2015.

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

Avoiding pain to avoid drugs

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

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

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

Hernandez was part of an experiment testing the drug versus a placebo and doesn't know whether she got the drug or a dummy medicine. But she hurt less than she expected to and never filled a prescription for pain pills.

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

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

On the horizon

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

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

And then there's marijuana. A cannabis extract is sold as a mouth spray in Britain for nerve pain and other problems from multiple sclerosis. But cannabinoid research in the United States has been hampered by marijuana's legal status. A special license is needed and most researchers don't even try to obtain one, said Susan Ingram, a neurosurgery scientist at Oregon Health & Science University.

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

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

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

Marilynn Marchione can be followed at http://twitter.com/MMarchioneAP

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