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Channel in Nerve Cell May be Key in Unlocking Parkinson Disease Therapy – Pharmacy Times

By adminDecember 3, 2019Induced Pluripotent Stem Cells

Researchers at the University of Cologne have identified Cav2.3 channels as a new mechanism for the development of Parkinson disease which may be the beginning of a new targeted therapy. The findings were published in Nature Communications.1

Parkinson disease is the second most common neurodegenerative disease in which a specific population of dopamine-producing nerve cells in the mid-brain die off selectively. The resulting lack of dopamine then leads to symptoms such as resting tremors, muscle stiffness, and problems executing voluntary movement. It affects more than 6 million people worldwide and is strongly age-dependent.2

It has previously been found that at the cellular level, disturbances in the calcium-dependent signaling pathways are integral to the development of Parkinson disease. Calcium plays a key role in many cellular signaling pathways, and its concentration is therefore regulated very precisely in the cell.2

Deregulation of the calcium balance causes disturbances of the intracellular signaling cascades, which can lead to cell death. Researchers have now shown that excessive calcium influx through specific ion channels, Cav2.3 channels of the so-called R-type, can contribute significantly to the development of Parkinson disease.1

Researchers were able to prevent the death of dopamine-producing nerve cells by genetically switching off the activity of the Cav2.3 channels. The ion channel Cav2.3 has so far not been associated with Parkinson disease. Further research on dopamine-producing neurons, which have developed from human so-called induced pluripotent stem cells, shows that signaling cascades similar to those that cause Parkinson sensitivity in the animal models are also active in human neurons.1

It had been previously hypothesized that another calcium channel, Cav1.3, plays a central role in the development of Parkinson disease. However, a recently completed clinical trial in which Cav1.3 channels were blocked did not show protection against Parkinson disease.2

This new study provides evidence as to why this clinical trial failed to show protective effects and suggests that selective Cav2.3 inhibitors should be tested as a drug to treat Parkinson disease, the authors concluded.2

Reference

First-in-kind Human 3-dimensional Models of Parkinson’s Disease and Progressive Multiple Sclerosis Launching to the International Space Station -…

By adminDecember 3, 2019Induced Pluripotent Stem Cells

LOUISVILLE, Ky.--(BUSINESS WIRE)--The National Stem Cell Foundation (NSCF) announced today that research teams from Aspen Neuroscience and the New York Stem Cell Foundation (NYSCF) Research Institute will send a first-in-kind study of neurodegenerative disease to the International Space Station (ISS) on the nineteenth SpaceX Commercial Resupply Services (CRS-19) mission, scheduled to launch December 4th from the Kennedy Space Center in Cape Canaveral, Florida. This is the second space flight for the research teams. A preliminary experiment was launched to the ISS in July 2019 onboard SpaceX CRS-18 to test custom flight hardware systems and refine post-flight analytical methods in preparation for the SpaceX CRS-19 launch.

The NSCF-funded collaboration between researchers at the NYSCF Research Institute and Aspen Neuroscience will perform the first study of long-term cell cultures of patient-derived induced pluripotent stem cell (iPSC) neural organoids with microglia on the ISS to study Parkinsons disease and primary progressive multiple sclerosis in microgravity. The ability to observe cell interaction, cell signaling, migration, changes in gene expression and the common pathways of neuroinflammation for both diseases in microgravity provides an opportunity to view the biological processes in a way that is not possible on Earth. This innovative approach to modelling disease has the potential to provide valuable new insight into the fundamental mechanisms underlying neurodegenerative disorders that may accelerate biomarker discovery and potential new drug and cell therapy options for patients. These models also offer potential for better translational study and future personalized medicine applications.

The development of patient-specific, 3-dimensional human organoids that incorporate microglia (the inflammatory cells of the immune system implicated in the development of Parkinsons, MS and other neurodegenerative diseases) for observation and study in the unique research environment of microgravity has the potential to enable progress across the field for a wide variety of conditions that affect a significant portion of the global population. The engineering required to facilitate the transport of cells and culture on orbit is being led by space flight engineering partner Space Tango.

Dr. Paula Grisanti, CEO of NSCF said, Supporting this collaboration between world-class research teams during a time of explosive growth in our understanding of the research advances possible in space is a great privilege. We are delighted to be funding such innovative science at the frontier of new drug and cell therapy discovery.

We are thrilled to be working with such a comprehensive team of scientists and fantastic organizations and feel honored to use our technology to better understand neurodegenerative disorders affecting so many persons globally, said Dr. Andres Bratt-Leal, Vice President of Research and Development, Aspen Neuroscience.

We feel privileged to have the opportunity to help understand the behavior of neural cells in microgravity and to help model neurodegenerative disease in such a novel way. We are excited about this fantastic project and look forward to learning the results, said Dr. Jeanne Loring, Chief Scientific Officer, Aspen Neuroscience.

We are excited to collaborate on the first study of progressive multiple sclerosis and Parkinsons patient brain cells in space. This work will provide important insights into the mechanisms behind these diseases and advance targets for future treatments," noted Susan L. Solomon, NYSCF Chief Executive Officer.

There is significant potential to advance our understanding of MS and PD as we initiate these long-term studies of patient cells in microgravity now that we have completed our preliminary tests, said Dr. Valentina Fossati, NYSCF Senior Research Investigator. We look forward to leveraging the unique capabilities of spaceflight research to better understand the role of microglia in multiple sclerosis and Parkinsons disease, as well as how dysfunction in these cells can be targeted therapeutically.

It takes vision, passion, and courage to change the paradigms of current understanding, said Jana Stoudemire, Commercial Innovation Officer at Space Tango. We are honored to support the groundbreaking work of the National Stem Cell Foundation and these recognized leaders in stem cell biology. Their commitment and dedication to advancing the frontiers of science using new tools and new approaches has been inspiring to witness, and has the potential to provide an entirely new perspective on Parkinsons and progressive MS.

To learn more about this unique collaboration, visit https://www.stemcellsinspace.org/.

About The National Stem Cell Foundation (NSCF)

The National Stem Cell Foundation is a 501(c)3 non-profit organization that funds adult stem cell and regenerative medicine research, connects children with limited resources to clinical trials for rare diseases and underwrites the National STEM Scholar Program for middle school science teachers inspiring the next generation of STEM (science, technology, engineering and math) pioneers nationwide. For more information, visit https://nationalstemcellfoundation.org/.

About The New York Stem Cell Foundation (NYSCF) Research Institute

The New York Stem Cell Foundation Research Institute is an independent organization accelerating cures and better treatments for patients through stem cell research. The NYSCF global community includes over 180 researchers at leading institutions worldwide, including NYSCF Druckenmiller Fellows, NYSCF Robertson Investigators, NYSCF Robertson Stem Cell Prize Recipients, and NYSCF Research Institute scientists and engineers. The NYSCF Research Institute is an acknowledged world leader in stem cell research and in developing pioneering stem cell technologies, including the NYSCF Global Stem Cell Array and in enabling large-scale stem cell research for scientists around the globe. NYSCF focuses on translational research in a model designed to overcome the barriers that slow discovery and replace silos with collaboration. For more information, visit http://www.nyscf.org.

About Aspen Neuroscience, Inc.

Aspen Neuroscience is a development stage, private biotechnology company that uses innovative genomic approaches combined with stem cell biology to deliver patient-specific, restorative cell therapies that modify the course of Parkinsons disease. The pipeline technology of Aspen is based upon the scientific work of world-renowned stem cell scientist, Dr. Jeanne Loring, who has developed a novel method for autologous neuron replacement. For more information and important updates, please visit http://www.aspenneuroscience.com.

About Space Tango, Inc.

Space Tango provides improved access to microgravity through their Open Orbit platform for bioengineering and manufacturing applications that benefit life on Earth. With their first operational TangoLab facility installed on the International Space Station in 2016, and a second facility installed in 2017, Space Tango has designed and flown nearly 80 diverse payloads. As a recognized leader in the development of fully automated, remote-controlled systems for research and manufacturing in orbit, Space Tango continues to provide expertise in technology and scientific consulting for industry and academic partners. Leveraging this current work, Space Tango is developing new commercial market segments in space with the announcement of ST-42 a fully autonomous orbital platform designed specifically for scalable manufacturing in space. Space Tango envisions a future where the next important breakthroughs in both technology and healthcare will occur off the planet, creating a new global market 250 miles up in low Earth orbit. For more information, visit http://www.spacetango.com.

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First-in-kind Human 3-dimensional Models of Parkinson's Disease and Progressive Multiple Sclerosis Launching to the International Space Station -...

Examining the ethics of embryonic stem cell research …

By adminDecember 3, 2019Stem Cell Clinics

Last year, President Bush cast the first veto of his presidency when Congress tried to ease the restriction on federal funding of embryonic stem cell research.

Following the recent passage by both houses of Congress of the Stem Cell Research Enhancement Act of 2007, which would permit federal funding of research using donated surplus embryonic stem cells from fertility clinics, the president has once again threatened a veto.

Because neither the House nor the Senate had sufficient votes to override a presidential veto, it appears unlikely this new bill will be enacted into law, further stalling the pace of this research. This bill crosses a moral line that I and others find troubling, stated Bush, following the Senates vote.

SCL: What are the main arguments for and against embryonic stem cell research? MS: Proponents argue that embryonic stem cell research holds great promise for understanding and curing diabetes, Parkinsons disease, spinal cord injury, and other debilitating conditions. Opponents argue that the research is unethical, because deriving the stem cells destroys the blastocyst, an unimplanted human embryo at the sixth to eighth day of development. As Bush declared when he vetoed last years stem cell bill, the federal government should not support the taking of innocent human life.

It is surprising that, despite the extensive public debatein Congress, during the 2004 and 2006 election campaigns, and on the Sunday morning talk showsrelatively little attention has been paid to the moral issue at the heart of the controversy: Are the opponents of stem cell research correct in their claim that the unimplanted human embryo is already a human being, morally equivalent to a person?

SCL: Considering that the moral and political controversy over embryonic stem cell research centers on this very question, why do you think there is so little attention being paid to it? MS: Perhaps this claim has gone unaddressed because stem cell proponents and many in the media consider it obviously falsea faith-based belief that no rational argument could possibly dislodge. If so, they are making a mistake. The fact that a moral belief may be rooted in religious conviction neither exempts it from challenge nor puts it beyond the realm of public debate. Ignoring the claim that the blastocyst is a person fails to respect those who oppose embryonic stem cell research on principled moral grounds. It has also led the media to miss glaring contradictions in Bushs stem cell policy, which does not actually live up to the principle it invokesthat destroying an embryo is like killing a child.

It is important to be clear about the embryo from which stem cells are extracted. It is not implanted and growing in a womans uterus. It is not a fetus. It has no recognizable human features or form. It is, rather, a blastocyst, a cluster of 180 to 200 cells, growing in a petri dish, barely visible to the naked eye.

SCL: What are the contradictions in Bushs stance? MS: Before we address that, it is important to be clear about the embryo from which stem cells are extracted. It is not implanted and growing in a womans uterus. It is not a fetus. It has no recognizable human features or form.

It is, rather, a blastocyst, a cluster of 180 to 200 cells, growing in a petri dish, barely visible to the naked eye. Such blastocysts are either cloned in the lab or created in fertility clinics. The bill recently passed by Congress would fund stem cell research only on excess blastocysts left over from infertility treatments.

The blastocyst represents such an early stage of embryonic development that the cells it contains have not yet differentiated, or taken on the properties of particular organs or tissueskidneys, muscles, spinal cord, and so on. This is why the stem cells that are extracted from the blastocyst hold the promise of developing, with proper coaxing in the lab, into any kind of cell the researcher wants to study or repair.

The moral and political controversy arises from the fact that extracting the stem cells destroys the blastocyst. It is important to grasp the full force of the claim that the embryo is morally equivalent to a person, a fully developed human being.

For those who hold this view, extracting stem cells from a blastocyst is as morally abhorrent as harvesting organs from a baby to save other peoples lives. This is the position of Senator Sam Brownback, Republican of Kansas, a leading advocate of the right-to-life position. In Brownbacks view, a human embryo . . . is a human being just like you and me; and it deserves the same respect that our laws give to us all.

If Brownback is right, then embryonic stem cell research is immoral because it amounts to killing a person to treat other peoples diseases.

SCL: What is the basis for the belief that personhood begins at conception? MS: Some base this belief on the religious conviction that the soul enters the body at the moment of conception. Others defend it without recourse to religion, by the following line of reasoning: Human beings are not things. Their lives must not be sacrificed against their will, even for the sake of good ends, like saving other peoples lives. The reason human beings must not be treated as things is that they are inviolable. At what point do humans acquire this inviolability? The answer cannot depend on the age or developmental stage of a particular human life. Infants are inviolable, and few people would countenance harvesting organs for transplantation even from a fetus.

Every human beingeach one of usbegan life as an embryo. Unless we can point to a definitive moment in the passage from conception to birth that marks the emergence of the human person, we must regard embryos as possessing the same inviolability as fully developed human beings.

SCL: By this line of reasoning, human embryos are inviolable and should not be used for research, even if that research might save many lives.MS: Yes, but this argument can be challenged on a number of grounds. First, it is undeniable that a human embryo is human life in the biological sense that it is living rather than dead, and human rather than, say, bovine.

But this biological fact does not establish that the blastocyst is a human being, or a person. Any living human cell (a skin cell, for example) is human life in the sense of being human rather than bovine and living rather than dead. But no one would consider a skin cell a person, or deem it inviolable. Showing that a blastocyst is a human being, or a person, requires further argument.

Some try to base such an argument on the fact that human beings develop from embryo to fetus to child. Every person was once an embryo, the argument goes, and there is no clear, non-arbitrary line between conception and adulthood that can tell us when personhood begins. Given the lack of such a line, we should regard the blastocyst as a person, as morally equivalent to a fully developed human being.

SCL: What is the flaw in this argument? MS: Consider an analogy: although every oak tree was once an acorn, it does not follow that acorns are oak trees, or that I should treat the loss of an acorn eaten by a squirrel in my front yard as the same kind of loss as the death of an oak tree felled by a storm. Despite their developmental continuity, acorns and oak trees differ. So do human embryos and human beings, and in the same way. Just as acorns are potential oaks, human embryos are potential human beings.

The distinction between a potential person and an actual one makes a moral difference. Sentient creatures make claims on us that nonsentient ones do not; beings capable of experience and consciousness make higher claims still. Human life develops by degrees.

SCL: Yet there are people who disagree that life develops by degrees, and believe that a blastocyst is a person and, therefore, morally equivalent to a fully developed human being. MS: Certainly some people hold this belief. But a reason to be skeptical of the notion that blastocysts are persons is to notice that many who invoke it do not embrace its full implications.

President Bush is a case in point. In 2001, he announced a policy that restricted federal funding to already existing stem cell lines, so that no taxpayer funds would encourage or support the destruction of embryos. And in 2006, he vetoed a bill that would have funded new embryonic stem cell research, saying that he did not want to support the taking of innocent human life.

But it is a striking feature of the presidents position that, while restricting the funding of embryonic stem cell research, he has made no effort to ban it. To adapt a slogan from the Clinton administration, the Bush policy might be summarized as dont fund, dont ban. But this policy is at odds with the notion that embryos are human beings.

SCL: If Bushs policy were consistent with his stated beliefs, how, in your opinion, would it differ from his current dont fund, dont ban policy? MS: If harvesting stem cells from a blastocyst were truly on a par with harvesting organs from a baby, then the morally responsible policy would be to ban it, not merely deny it federal funding.

If some doctors made a practice of killing children to get organs for transplantation, no one would take the position that the infanticide should be ineligible for federal funding but allowed to continue in the private sector. In fact, if we were persuaded that embryonic stem cell research were tantamount to infanticide, we would not only ban it but treat it as a grisly form of murder and subject scientists who performed it to criminal punishment.

SCL: Couldnt it be argued, in defense of the presidents policy, that Congress would be unlikely to enact an outright ban on embryonic stem cell research? MS: Perhaps. But this does not explain why, if the president really considers embryos to be human beings, he has not at least called for such a ban, nor even called upon scientists to stop doing stem cell research that involves the destruction of embryos. In fact, Bush has cited the fact that there is no ban on embryonic stem cell research in touting the virtues of his balanced approach.

The moral oddness of the Bush dont fund, dont ban position confused even his spokesman, Tony Snow. Last year, Snow told the White House press corps that the president vetoed the stem cell bill because he considered embryonic stem cell research to be murder, something the federal government should not support. When the comment drew a flurry of critical press attention, the White House retreated. No, the president did not believe that destroying an embryo was murder. The press secretary retracted his statement, and apologized for having overstated the presidents position.

How exactly the spokesman had overstated the presidents position is unclear. If embryonic stem cell research does constitute the deliberate taking of innocent human life, it is hard to see how it differs from murder. The chastened press secretary made no attempt to parse the distinction. His errant statement that the president considered embryo destruction to be murder simply followed the moral logic of the notion that embryos are human beings. It was a gaffe only because the Bush policy does not follow that logic.

SCL: You have stated that the presidents refusal to ban privately funded embryonic stem cell research is not the only way in which his policies betray the principle that embryos are persons. How so? MS: In the course of treating infertility, American fertility clinics routinely discard thousands of human embryos. The bill that recently passed in the Senate would fund stem cell research only on these excess embryos, which are already bound for destruction. (This is also the position taken by former governor Mitt Romney, who supports stem cell research on embryos left over from fertility clinics.) Although Bush would ban the use of such embryos in federally funded research, he has not called for legislation to ban the creation and destruction of embryos by fertility clinics.

SCL: If embryos are morally equivalent to fully developed human beings, doesnt it then follow that allowing fertility clinics to discard thousands of embryos is condoning mass murder? MS: It does. If embryos are human beings, to allow fertility clinics to discard them is to countenance, in effect, the widespread creation and destruction of surplus children. Those who believe that a blastocyst is morally equivalent to a baby must believe that the 400,000 excess embryos languishing in freezers in U.S. fertility clinics are like newborns left to die by exposure on a mountainside. But those who view embryos in this way should not only be opposing embryonic stem cell research; they should also be leading a campaign to shut down what they must regard as rampant infanticide in fertility clinics.

Some principled right-to-life opponents of stem cell research meet this test of moral consistency. Bushs dont fund, dont ban policy does not. Those who fail to take seriously the belief that embryos are persons miss this point. Rather than simply complain that the presidents stem cell policy allows religion to trump science, critics should ask why the president does not pursue the full implications of the principle he invokes.

If he does not want to ban embryonic stem cell research, or prosecute stem cell scientists for murder, or ban fertility clinics from creating and discarding excess embryos, this must mean that he does not really consider human embryos as morally equivalent to fully developed human beings after all.

But if he doesnt believe that embryos are persons, then why ban federally funded embryonic stem cell research that holds promise for curing diseases and saving lives?

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Examining the ethics of embryonic stem cell research ...

Stem of the problem | Columns | Journal Gazette – Fort Wayne Journal Gazette

By adminDecember 3, 2019Stem Cell Clinics

I have been reading a book, The Man Who Wouldn't Die, that satirizes Silicon Valley and the venture-capital start-up culture. It's reasonably funny. I was reminded, however, that sometimes culture is crazy.

At one point a character casually describes how stem-cell therapy is obviously going to make 50-year-olds feel like 20-year-olds. Grow a new liver and you're good to go, so to speak! The author clearly meant the idea as a joke. However, that idea is out there and taken seriously. Stem cells made national headlines in the late '90s and early 2000s because of some scientific breakthroughs and their promise for future medical treatments.

Just about all of our body is made up of specialized cells. These are cells that have grown to perform some specific function. Some examples are heart muscle cells, say, or red blood cells.

Stem cells are different; they have not yet specialized. That is, they have not developed many of the specific properties that are unique to the different parts of our bodies.

Given the right environment the right chemicals, for examplethey can grow into specialized cells.

There are, of course, all sorts of important details for how stem cells grow, when they specialize and how much flexibility they have in specializing. Somehow, however, there is now an industry that has skipped all that work and is marketing stem cells as a general cure. Alzheimer's disease and joint pain are frequently mentioned. If that makes you suspicious that these clinics are targeting the elderly, you would be right.

A typical treatment involves taking stem cells from a patient's bone marrow or fat and injecting it back into a sore knee or hip or whatever needs fixing. The idea, somewhat vaguely, is that the stem cells will grow to replace whatever is worn down by age.

As is true of all good scams, this one has a good story, one that can pass as proven medicine for many patients. The treatment also benefits by treating a problem with symptoms that can come and go irregularly, so a patient can honestly claim that they recovered in, say,six months.

The body is a complicated machine. It is hard to predict what will cure or not cure any specific problem. Just because a story involving stem cells sounds reasonable is not a sufficient reason to try something.

For a typical patient, we should have good, strong, positive evidence that a treatment will help. A good story is not strong, positive evidence. In the language of science, it is a hypothesis. Potentially true.

It is worth remembering that many, many things are potentially true. We don't try them all out on ourselves when we are sick.

Many of these ideas have in fact been tested in rigorous, peer-reviewed studies. So far, the evidence for positive effects is weak.

One reason these clinics have managed to skirt the rules is that extracting material from a patient then reinjecting requires less oversight for safety. Depending on the details, this treatment is considered, for Food and Drug Administration regulations, something like plasma donation (where blood is extracted, platelets removed, then reinjected into the body).

Just because a process may be safe does not make it good medicine. Medicine is not and should not be a free market. Patients will almost never have enough knowledge to reasonably choose between treatment options.

The FDA is the federal agency in charge of ensuring companies do not offer useless or harmful procedures. Unfortunately, in 2017 the FDA decided to allow stem cell clinics a three-year grace period to describe their procedures for the FDA's evaluation. As a result of that open window, hundreds of clinics have opened nationwide, offering services for which there is no strong evidence.

In the past year the FDA has realized where the industry has gone with this treatment and begun trying to crack down. Some clinics have unsafe procedures; others are misleading patients about treatments. Many clinics are still operating, unfortunately, and it can be difficult for typical patients to recognize the difference between these treatments and proven remedies. Furthermore, these bad actors could potentially make people skeptical of stem cell-based technology entirely.

That would be a shame because there is still great promise for stem cells. That may be cold comfort for those who want a treatment immediately, but that desire is the exact motivation that has led to terrible medicine for centuries.

Christer Watson, of Fort Wayne, is a professor of physics at Manchester University. Opinions expressed are his own. He wrote this column for The Journal Gazette, where his columns normally appear the first and third Tuesday of each month.

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Stem of the problem | Columns | Journal Gazette - Fort Wayne Journal Gazette

Mayo Clinic Transplant Center – Regenerative medicine …

By adminDecember 3, 2019Stem Cell Clinic

Mayo Clinic Regenerative Medicine Consult Service

At Mayo Clinic, an integrated team, including stem cell biologists, bioengineers, doctors and scientists, work together and study regenerative medicine. The goal of the team is to treat diseases using novel therapies, such as stem cell therapy and bioengineering. Doctors in transplant medicine and transplant surgery have pioneered the study of regenerative medicine during the past five decades, and doctors continue to study new innovations in transplant medicine and surgery.

In stem cell therapy, or regenerative medicine, researchers study how stem cells may be used to replace, repair, reprogram or renew your diseased cells. Stem cells are able to grow and develop into many different types of cells in your body. Stem cell therapy may use adult cells that have been genetically reprogrammed in the laboratory (induced pluripotent stem cells), your own adult stem cells that have been reprogrammed or cells developed from an embryo (embryonic stem cells).

Researchers also study and test how reprogrammed stem cells may be turned into specialized cells that can repair or regenerate cells in your heart, blood, nerves and other parts of your body. These stem cells have the potential to treat many conditions. Stem cells also may be studied to understand how other conditions occur, to develop and test new medications, and for other research.

Researchers across Mayo Clinic, with coordination through the Center for Regenerative Medicine, are discovering, translating and applying stem cell therapy as a potential treatment for cardiovascular diseases, diabetes, degenerative joint conditions, brain and nervous system (neurological) conditions, such as Parkinson's disease, and many other conditions. For example, researchers are studying the possibility of using stem cell therapy to repair or regenerate injured heart tissue to treat many types of cardiovascular diseases, from adult acquired disorders to congenital diseases. Read about regenerative medicine research for hypoplastic left heart syndrome.

Cardiovascular diseases, neurological conditions and diabetes have been extensively studied in stem cell therapy research. They've been studied because the stem cells affected in these conditions have been the same cell types that have been generated in the laboratory from various types of stem cells. Thus, translating stem cell therapy to a potential treatment for people with these conditions may be a realistic goal for the future of transplant medicine and surgery.

Researchers conduct ongoing studies in stem cell therapy. However, research and development of stem cell therapy is unpredictable and depends on many factors, including regulatory guidelines, funding sources and recent successes in stem cell therapy. Mayo Clinic researchers aim to expand research and development of stem cell therapy in the future, while keeping the safety of patients as their primary concern.

Mayo Clinic offers stem cell transplant (bone marrow transplant) for people who've had leukemia, lymphoma or other conditions that have been treated with chemotherapy.

Mayo Clinic currently offers a specialty consult service for regenerative medicine within the Transplant Center, the first consult service established in the United States to provide guidance for patients and families regarding stem cell-based protocols. This consult service provides education and consultation for people with many conditions who have questions about the potential use of stem cell therapy. The staff provides guidance to determine whether stem cell clinical trials are appropriate for these individuals. Regenerative medicine staff may be consulted if a doctor or patient has asked about the potential use of stem cell therapies for many conditions, including degenerative or congenital diseases of the heart, liver, pancreas or lungs.

People sometimes have misconceptions about the use and applications of stem cell therapies. This consult service provides people with educational guidance and appropriate referrals to research studies and clinical trials in stem cell therapies for the heart, liver, pancreas and other organs. Also, the consult service supports ongoing regenerative medicine research activities within Mayo Clinic, from basic science to clinical protocols.

Quinn Waters: Weymouth, Massachusetts three-year-old is seeing the world for himself – CBS News

By adminDecember 2, 2019Stem Cell Doctors

Weymouth, Massachusetts Aside from immediate family, no one was allowed in the house to see 3-year-old Quinn Waters of Weymouth, Massachusetts. And more importantly Quinn wasn't allowed out.

"We basically keep him in a bubble just as a precaution," said Quinn's father, Jarlath.

"Even a common cold could be something that will bring him back into the hospital," said Quinn's mother, Tara.

Parents Jarlath and Tara Waters say Quinn's natural immunity was temporarily wiped out after he got a stem cell transplant to treat his brain cancer.

Fortunately, the kid is a fighter and as we first reported a few months ago, he kept a mostly positive attitude.

But it still stunk.

He sees all of this happening and he knows he's stuck inside. And there would be days when Quinn was literally pounding to get out.

Unfortunately, staring out a window is a poor substitute for walking out a door. Quinn's connection to the outside world has been limited to whoever passes by, which hasn't been all that limiting, actually.

"It started out with family members coming to the window," said Jarlath.

Then the neighbors started showing up to entertain the police caught wind and pretty soon topnotch performers were just showing up on Quinn's front lawn.

It turned into a vaudeville stage out there.

"Yeah, the window kind of became his window on the world," Jarlath said.

It got so you never knew what might happen by. One minute it could be a dog parade the next, a team of Irish step dancers everyone brought together by word of mouth and a will to help Quinn get better.

Which his parents say did start happening.

"It's the positive energy from all these people that we believe has gotten him through his sickness, you know. You can never repay, you know (emotional), just maybe pay it forward," Jarlath said.

Being indebted never felt so fortunate.

After this story first aired in August, things got even better for Quinn light-years better. By Halloween, doctors had released him from home confinement and free to be a kid again he rushed outdoors at warp speed.

He also got to drop the puck at a Boston Bruins' game and feel the sand between his toes at the Massachusetts shore.

There is no greater curse than cancer but no greater blessing, than beating it.

To contactOn the Road, or to send us a story idea, email us:OnTheRoad@cbsnews.com.

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Quinn Waters: Weymouth, Massachusetts three-year-old is seeing the world for himself - CBS News

‘We didn’t worry when 2-year-old refused to use potty – then her lips went blue’ – Mirror Online

By adminDecember 2, 2019Stem Cell Doctors

It was back in November 2015 when Lynsay Hughes and her husband Andrew, a project engineer, both 38, first noticed their two-year-old Evie was out of sorts.

Wed begun potty training and it was going really well, but Evie suddenly started refusing to go to the toilet and got very whingy, explains Lynsay, a swimming teacher.

Shed been quite a sickly baby, so initially we didnt think much of it.

When Evie went off her food, got dark circles under her eyes, and seemed to have no energy, Lynsay took her to the GP.

The little girl was then referred to North Tees Hospital, near the familys home in Stockton-on-Tees.

She spent five days there, before eventually being diagnosed with a chronic kidney infection, and discharged.

I just wanted to get her home for Christmas, says Lynsay, also mum to Oscar, now nine.

She was fine for a few days and then her health plummeted her legs, feet and fingers began to swell and her lips went blue.

Andrew took her to the park and she vomited everywhere. We knew something wasnt right.

Lynsay went back to hospital, where Evie was given an ultrasound.

A doctor came out and said, Do you want to wait for your husband? but I never dreamed it would be anything serious so I said, No, just tell me now.

He explained that while he wasnt a paediatric expert, they had found a mass around Evies tummy and wanted us to be transferred to specialists at the Royal Victoria Infirmary in Newcastle.

Andrew met us at the bigger hospital we knew it was more serious, but I still wasnt thinking about cancer.

Even when we went to meet an oncology consultant, I wasnt panicking because I honestly didnt know what the word meant.

The first time we realised it was cancer was when a doctor came in the room and said, Im going to take a biopsy of Evies tumour.

What she had a tumour? Andrew and I just looked at each other in shock.

The couple were soon ushered into a room full of different medical people consultants, nurses, anaesthetists and were told the devastating news: Evie had stage 4 neuroblastoma.

Neuroblastoma is a very rare childhood cancer that affects only about 100 children a year in the UK.

I was in complete shock, recalls Lynsay.

I said to the doctor: Are you telling me my daughter has cancer? he replied: Im not just telling you she has cancer, Im telling you she has the beast of childhood cancers. Those were his exact words.

Andrew burst into tears, but I couldnt take it in, I just held his hand feeling gutted, totally heartbroken.

It was on 22 December 2015 when that devastating diagnosis was confirmed: Evie had a 13cm tumour the most aggressive kind which was wrapped around her main artery.

The disease had also spread to her bones. Knowing staff would be short for the festive season, doctors moved quickly.

On Christmas Eve, the poorly toddler began her first, gruelling round of chemotherapy.

It was only when Evie had her line to receive her chemotherapy fitted into her little body that the enormity of the situation finally sank in, admits Lynsay.

I let out some noise that Andrew tells me was like a shriek and I fell to the floor. It was like the world had stopped.

But somehow we dragged ourselves through Christmas, trying to put on brave faces to watch Oscar open his presents.

Evie was too poorly to care about gifts.

The next year went in a blur of treatment and surgery.

Evie was put on a 10-day cycle of different chemotherapy drugs for 80 days to target the disease in her bones.

Then she had the tumour surgically removed, a stem cell harvest, high intensity chemotherapy with stem cell replacement, and radiotherapy every day for three weeks.

She was also put on a clinical trial for immunotherapy, designed to kill the bad cells while protecting the healthy ones for a further six months.

It was unbelievably traumatic. If Evie woke up in a different room to the one she went to sleep in, or if we werent there, she would scream and scream.

We explained to her that she had a nasty bug in her tummy and the doctors were giving her medicine which would make her feel funny but would make her better in the end.

We named her feeding tube Tommy and her chemo drips Wally and Dolly, to try and make things less scary for her.

She took her beloved Teddy everywhere.

When Evies blonde curls fell out in lumps, my friend who is a hairdresser came and cut it into a bob for her.

We told her she had superpowers, thats why her hair had fallen out.

Her big brother, Oscar, was so sweet when he came to the hospital, he would make friends with the other children on the ward and be so protective of her.

There were times when I was terrified she might die, but I just willed her to be better and wouldnt allow myself to think the worst.

We put on a brave face at home for Oscar, but when I was in bed I would cry and cry and my heart would be racing with the anxiety and stress.

I just had to focus on the next part of the treatment.

In between we tried to do nice things we took her to The Lion King in London as a treat, and we went camping in the Lake District.

Lynsay gave up teaching swimming to be with Evie full time, Andrew cut down his hours as a project engineer, and Lynsays sister would take care of Oscar when both parents had to be at hospital.

Finally, in March 2017, after more than two years of treatment, the family received the news they had been longing to hear: Evie was cancer-free and in remission.

To mark the momentous occasion, the hospital have a special end of treatment bell that children get to ring at the end of their chemotherapy.

It was a huge day for us.

Evie wore a new dress with rainbows on it, and our whole family came to the hospital to watch her ring the bell.

We all cried buckets.

Afterwards, we went to a restaurant in Newcastle where everyone made a fuss of her and had a big party, with Evie getting a knickerbocker glory.

Shes two and half years in remission now and living a normal, healthy life.

Shes in Year One at school, has lots of friends, and her hair has grown back. She loves dancing and drama and riding her bike.

This Christmas we will be surrounded by our family at our home and we are determined to enjoy every second.

Evie is madly into Barbies and Frozen and cant wait for Father Christmas to come. Shes definitely been good!

And we have the best present you could possibly imagine our family, together.

Neuroblastoma (neuro = nerve, blastoma = collection of tumour cells) is a tumour arising from particular nerve cells, which run up the back of the childs abdomen and chest and into the skull, following the line of the spinal cord.

The tumour occurs either in the sympathetic nervous system, or closely associated adrenal glands, and is usually, although not always, located in the abdomen.

Neuroblastoma affects around 100 children each year in the UK. It usually affects children under the age of five, and can occur before a child is born, because it develops from the cells left behind from a babys development in the womb. It is the most common solid tumour in childhood after brain tumours.

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'We didn't worry when 2-year-old refused to use potty - then her lips went blue' - Mirror Online

Upstate SC toddler survives rare cancer and the risky procedure used to treat it – Greenville News

By adminDecember 2, 2019Stem Cell Doctors

Outcome means a special Thanksgiving

Hailie and Treylin Hyman saw the bruising on their baby girls leg as a sign that the active 1-year-old was learning to walk.

But as a blood test would later reveal, little Maci was actually suffering from an extremely rare blood cancer that threatened her life without a risky treatment - atreatmentalmost as dangerous as the disease.

In the beginning, it was very scary, Hailie Hyman told The Greenville News.

I couldnt think of anything but the bad things, she confessed. It was all about the statistics. And the statistics arent good.

Hailie Hyman holds her daughter Maci, 1, before an appointment at the Prisma Health Pediatric Hematology Oncology Center Monday, Nov. 4, 2019.(Photo: JOSH MORGAN/Staff)

Terrifying months followed the diagnosis, punctuated by one critical complication after another, leaving the Boiling Springs couple to wonder if Maci would survive.

Somehow, though, the blue-eyed toddler pulled through.And now her family is looking forward to a special Thanksgiving with much to be grateful for.

The Hymans journey began last February atMacis 1-year-old well-child checkup.

We had no idea anything was wrong, her mom said.But they did a routine (blood test) and a couple of hours later, we got a call saying her platelets were very low.

The Hymans were referred to a hematologist who found other abnormalities in Macis blood and scheduled a bone marrow biopsy to investigate further.

Hailie Hyman holds her daughter Maci, 1, before an appointment at the Prisma Health Pediatric Hematology Oncology Center Monday, Nov. 4, 2019.(Photo: JOSH MORGAN/Staff)

During the procedure, the child suffered an aneurysm in an artery and went into cardiac arrest. The team performed CPR on her for 20 minutes before she was stabilized, her mom said.

Later, in the pediatric intensive care unit, she suffered internal bleeding, too.

It was really hard, she said. There were many nights that I would just pray and pray and pray.

Initially believing Maci had leukemia, doctors subsequently determined she had myelodysplastic syndrome, or MDS.

The condition occurs when abnormal cells in the bone marrow leave the patient unable to make enough blood, according to the American Cancer Society.

Its rare, afflicting as few 10,000 Americans a year, though the actual number is unknown.

Maci Hyman, 1, interacts with hospital staff before an appointment at the Prisma Health Pediatric Hematology Oncology Center Monday, Nov. 4, 2019.(Photo: JOSH MORGAN/Staff)

In children, its rarer still. Most people arediagnosed in their 70s.

We were told that just four out of 1 million children get it every year, Hailie Hyman said.

That made the diagnosis elusive at first, said Dr. Nichole Bryant, a pediatric hematologist-oncologist with Prisma Health-Upstate, formerly Greenville Health System.

Shes the only one Ive seen in my career, she said.

Maci had to have regular blood transfusions, antibiotics and other medications to fight the MDS, Bryant said. But the only hope for a cure was a stem cell transplant at the Medical University of South Carolina in Charleston.

When they said that was the only treatment plan for MDS, I of course went to Google, Hailie Hyman said. I read about transplant patients and ...all the complications. It was terrifying. But no matter how many bad things I saw, we had to do it. There is no other option.

The transplantis extremely risky.

Hailie Hyman looks at a fish tank with her daughter Maci, 1, before an appointment at the Prisma Health Pediatric Hematology Oncology Center Monday, Nov. 4, 2019.(Photo: JOSH MORGAN/Staff)

First, high doses of chemotherapy are given to destroy the diseased bone marrow, leaving the patient without an immune system, so fighting infections becomes a challenge. Then healthy donor marrow is infused.

Its also fraught with potentially life-threatening complications, including graft vs. host disease, which occurs when immune cells from the donor attack the patients body, Bryant said. Other complications include permanent kidney damage and gastrointestinal problems.

They have to go to hell and back, she said. But its the only option for long-term survival.

Maci had a really rough start, suffering lots and lots and lots of complications, Bryant said.

Her kidneys failed, so she wound up on dialysis. When she couldnt breathe on her own, she was put on a ventilator. And because she couldnt eat, she had to be tube fed.

Hailie Hyman looks at a fish tank with her daughter Maci, 1, before an appointment at the Prisma Health Pediatric Hematology Oncology Center Monday, Nov. 4, 2019.(Photo: JOSH MORGAN/Staff)

She had blistering sores in her mouth and throughout her GI tract, her mom said. Because her liver wasnt functioning properly, her abdomen filled up with fluid that had to be drained. She was bleeding so profusely in her lungs that one of them collapsed.

Maci, who was sedated through much of it, was put on full life support, she said.

That night we almost lost her, her mom said. We were in the hallway crying our eyes out. We didnt know what do to or think. It was pretty scary for a while.

Somehow, Maci made it.

There were so many times during her first months that it seemed like she would not survive, Bryant said. So the fact that she is here ... is really a miracle.

Macis family found an unrelated donor through the National Marrow Donor Program, enlisting hundreds of other people to join the registry in the process, Bryant said.

Nichole Bryant, M.D.(Photo: Provided)

It was an important part of their journey that maybe didnt directly benefit Maci, she said. But if everybody did that, we wouldnt have difficulty finding a donor for anybody.

Doctors have no explanation for why Maci got MDS. She didnt carry the genetic mutation for it and there is no family history.

She is a rare child - and not in a good way, her mom said, adding,Youve got to laugh sometimes or youre going to cry.

Maci was admitted to MUSC on June 2 and released on Oct. 14.

The Hymans, both 22, spent the entire time in Charlestonwhile Hailies mom cared for their older daughter, Athena, now 2.

Treylins employer held his welding job open for him. And other friends and family members did what they could to help.

We had many, many people very generously donate to us to cover expenses at home and living expenses where we were, Hailie Hyman said.

We are thankful for everyone who helped us through it the cards, the gifts, the donations. Every single cent is greatly appreciated.

They still need to travel to Charleston once a week to see the transplant doctor. In between, Maci is seen in Greenville.

She's doing well, but recovery from a transplant can take months to years, Bryant said.

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Her kidneys are functioning again so she was able to come off dialysis. But she still must take many medications, including anti-rejection drugs that suppress her immune system and leaveher at risk for infection. And she still must be tube fed.

She is miles ahead of where she was two months ago, Bryant said. But she still has a long way to go. Its a long, long road.

Macis mom says she can be up and playing one day and flopped over on the couch another. She still experiences a lot of nausea and vomiting, but is doing well compared to where she was.

Hailie Hyman pulls her daughter Maci, 1, in a wagon in the hallway before an appointment at the Prisma Health Pediatric Hematology Oncology Center Monday, Nov. 4, 2019.(Photo: JOSH MORGAN/Staff)

So as the nation pauses to give thanks this Thanksgiving, she says the family will be countingtheir many blessings family andfriends, Gods mercy, andthe doctors and nurses who saved Macis life.

She has battled a lot and overcome a lot, she said. I have no doubt she will be able to get through.

Want to know more about becoming a marrow donor? Go to bethematch.org.

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Upstate SC toddler survives rare cancer and the risky procedure used to treat it - Greenville News

13 YEAR OLD BOY WHO SURVIVED RARE CHILDHOOD CANCER TWICE SUPPORTS NEW FUNDRAISING CAMPAIGN – MENAFN.COM

By adminDecember 2, 2019Stem Cell Doctors

(MENAFN - Pressat) 13 year old Ryan Savage, from Billingham in Stockton-on-Tees, features in a new Christmas campaign with Neuroblastoma UK, to raise vital funds for neuroblastoma research.

Ryan was first diagnosed with neuroblastoma, a rare childhood cancer, when he was just three years old. He was given a 5% chance of survival and doctors told his parents that he may not live to see Christmas.

This Christmas campaign aims to #GiveHope to families like Ryan's, by raising money for leading research projects which aim to deliver new, more effective and kinder treatments for children with neuroblastoma.

Ryan's mum Lisa says, 'Christmas is Ryan's favourite time of year - he's not a child who asks for much but he is always just so happy to celebrate Christmas with his family. Every time Christmas comes around, it's a precious reminder to our family that another year has passed since Ryan fought this terrible disease twice, and won.

'Ryan was first diagnosed with neuroblastoma on 29th October 2009 - a date that will be etched on my memory forever. He came home from nursery complaining of stomach pains. He wouldn't eat and the pain got worse, so I took him to A&E that night. Doctors thought it was constipation or trapped wind but two days later, he had a high temperature, was in extreme pain and, worryingly, he couldn't move his legs.

'We took him back to A&E and they did an immediate MRI scan. They found a tumour wrapped around Ryan's spine and we were told that Ryan could have less than two months to live, and he might not see Christmas.

Our world was turned upside-down by a cancer we had never even heard of before.'

Ryan began chemotherapy immediately, followed by surgery to remove the majority of his tumours. After a gruelling year of cancer treatment, Ryan was in remission and the family could celebrate another Christmas together.

But two years later, doctors found another tumour in his chest and their consultant said it was the worst case that she'd ever seen.

'Our little man endured another two really tough years of treatment including chemotherapy, radiotherapy, a stem cell transplant and major surgery. He also needed ribs removed as the cancerous tumours had wrapped around his little bones. By March 2012, the only tumour left in his body was a growth behind his heart. In October 2013, the heart tumour no longer appeared on his scans.

Ryan endured years of treatment including chemotherapy, radiotherapy, a stem cell transplant and major surgery, after having neuroblastoma twice.

'Seeing what Ryan went through, it breaks my heart to think of all those children fighting neuroblastoma this Christmas. They should be at home enjoying the fun of the festive season with their families. I know from personal experience that their parents will be hoping against hope that the treatment their children receive will help them beat neuroblastoma too.

'Little children simply don't deserve to be robbed of their precious early years, nor should they have the threat of their cancer returning constantly hanging over them. But there is still so much more research that needs to be done to prevent children suffering like Ryan did.'

Katherine Mobey, Fundraising Manager at Neuroblastoma UK said, 'Neuroblastoma is a cancer that almost exclusively affects children, with around 100 children diagnosed every year in the UK. Christmas is a time when families should be together and having fun, not going through harsh cancer treatment.

'With our new fundraising campaign, we want to give hope to more families like Ryan's. By funding more vital research, we can help scientists search for new and kinder treatments, give children with neuroblastoma a better quality of life after treatment, and move closer to finding a cure.'

To make a donation, visitwww.neuroblastoma.org.uk/christmas .

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13 YEAR OLD BOY WHO SURVIVED RARE CHILDHOOD CANCER TWICE SUPPORTS NEW FUNDRAISING CAMPAIGN - MENAFN.COM

Heartbreaking photograph shows the reality of childhood cancer – Yahoo News

By adminDecember 2, 2019Stem Cell Doctors

Sophia Soto is pictured tearfully awaiting cancer treatment. [Photo: Caters]

The mother of a cancer survivor has released a heartbreaking photograph to show the reality of the disease.

Sophia Soto, from Florida, was diagnosed with stage four neuroblastoma at just 14 months old after unusual bruises developed around her eyes.

READ MORE:Girl with neuroblastoma declared cancer-free after pioneering treatment

With doctors dismissing it as just a fall, an eye specialist eventually found tumours behind her eyes were to blame.

The toddler endured 60 rounds of chemo, 20 of radiotherapy and a stem cell transplant over six months. She was snapped just before having treatment.

Sophia, now six, has been in remission for five years and medication-free for the past 24 months.

Speaking of her daughters condition, Sophias mother Rosie Soto, 40, said: The picture of Sophia upset really does hone in on the reality of childhood cancer.

She was having a lead put on her chest for her treatment, which she didn't want, hence why Sophia was so upset.

I look back at the picture now and wonder how I did it. It was so hard watching my little girl so ill.

Neuroblastoma is a rare type of cancer that mainly affects babies and young children,according to the NHS.

READ MORE:Childhood Cancer Survivors Are Twice as Likely to Have This Condition

Around 95 youngsters in the UK are diagnosed every year, making up 6% of all childhood-cancer cases,Children with Cancer UK statistics show.

In the US, around 800 are diagnosed annually, also accounting for 6% of all cases,according to the American Cancer Society.

Neuroblastoma develops in specialised nerve cells called neuroblasts, which get left behind during a babys development in the womb.

The disease tends to start in one of the adrenal glands above the kidneys or nerve tissue next to the spinal cord, before spreading.

Mrs Soto became concerned when her daughter develop bruises around her eyes, with no obvious cause.

I kept taking her to the doctors because the bruising wasn't going away, but they just said it must have been from a bump or something, she said.

Sophia wasn't referred for a scan or biopsy until I went to an eye specialist, who knew straight away it was caused by a tumour.

Story continues

She was sent for an MRI, where black spots appeared on the scans confirmed the tumours behind her eyes.

[A] biopsy found tumours on one of her kidneys as well, which led to her stage four neuroblastoma diagnosis.

Sophia discovered she had the disease in March 2014, aged just 14 months.

After extensive treatment, the youngster has been in remission since that November, but still has check-ups every six months.

The tumours behind her eyes cannot be removed, however, doctors believe they are now benign.

READ MORE: Mom beats breast cancer and then books 'dream' trip

Doctors are reluctant to remove the tumours Sophia has behind her eyes as they've said it would be likely the surgery [would] disfigure her face, Mrs Soto said.

Whilst they are tumours, doctors are reasonably confident they are not cancerous so we have decided to not have the surgery right now, but it may be something she has when she's older.

Over the worst, Sophia loves dancing and dreams of one day becoming a vet.

No one can imagine what she went through looking at her now, she just looks like a normal regular child, Mrs Soto said.

Sophia has her moments when she asks about when she was sick and has questions about her treatment scars, but overall she's a pretty happy girl.

If I was to say anything to other parents with children battling cancer, I'd say to not give up, stay positive.

It's really important not to compare your child's process to anyone else as everyone battles illnesses differently.

We're over the moon Sophia is now doing so well. We're really blessed she's such a fighter.

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Heartbreaking photograph shows the reality of childhood cancer - Yahoo News