April 17, 2017 by Hannah L. Robbins SMN protein (red) is necessary for the survival of spinal cord neurons (motor neurons) responsible for breathing and all movement. Harvard researchers have found a compound that stabilized this protein in mouse and human motor neurons. This may lead to the development of new treatments for motor neuron diseases including Spinal Muscular Atrophy and Lou Gehrigs disease. Credit: Natalia Rodriguez-Muela

Harvard Stem Cell Institute (HSCI) researchers have identified a compound that helps protect the cells destroyed by spinal muscular atrophy (SMA), the most frequent fatal genetic disease in children under 2 years of age.

SMA is a neurodegenerative disease targeting motor neurons, the long nerve cells that relay messages from the brain to the muscles and that are, consequently, responsible for bodily movements, including walking, swallowing, and even breathing. Patients with milder forms of SMA experience muscle wasting that may confine them to a wheelchair, while the more severe forms cause paralysis and death before the second birthday.

About one in 50 people are genetic carriers of the disease.

Because of a dysfunctional gene, many motor neurons in SMA patients are unable to produce adequate amounts of a protein called survival of motor neuron (SMN). The deficiency causes cellular stress and eventually cell death. Rather than fixing the gene, which has been the strategy of many labs looking to develop SMA therapies, the Harvard team has identified a compound that helps stabilize the SMN protein both in human neurons in a dish and in mouse models.

The findings were published in the journal Cell Reports.

"This discovery opens up new lines of drug interrogation," said Lee Rubin, HSCI principal faculty member and the senior author on the study. Rubin's lab, which operates out of in Harvard's Department of Stem Cell and Regenerative Biology, uses induced pluripotent stem cells (iPS cells) to make human models of neurological diseases.

In 2015, Rubin made a variety of neuronal types from the iPS cells of SMA patients in order to determine why motor neurons in particular were targeted, and found they experienced a fatal stress response similar to motor neurons affected by amyotrophic lateral sclerosis (ALS), the late-onset neurodegenerative disease more commonly known as Lou Gehrig's disease.

Additionally, some SMA-affected motor neurons were dying before others, though all of the neurons had the same genetic mutations and were experiencing the same stressful environment.

"Clearly, some motor neurons were surviving, so the next question was whether this is random or if there is a molecular explanation," Rubin said.

Early on in their most recent study, the researchers found that within a single petri dish of motor neurons derived from an SMA patient, some produced up to four times as much SMN protein as their neighbors. Over time, those motor neurons with higher levels of SMN were more likely to survive after exposure to toxic environments and stressors.

When the team analyzed motor neurons derived from ALS patients, they found similar results: Motor neurons with higher levels of SMN were likelier to survive than those with lower levels.

"The surprise was when we looked in a control culture and also saw differences between the individual neurons," Rubin said.

"It is clear that the SMN protein is necessary for all motor neuron survival, not just motor neurons targeted by ALS or SMA," said Natalia Rodrguez-Muela, a postdoctoral fellow in Rubin's lab and co-first author on the paper. The results suggest that if the team could increase the amount of SMN protein in any single motor neuron, they would be able to rescue the cell.

During a cell's life span, proteins are constantly being made and degraded, made and degraded again. To interrupt the process of breaking down the SMN protein, the researchers looked at a family of proteins called Cullins, which act as a part of the cell machinery that regulates protein degradation.

In 2011, the Rubin lab had determined that an enzyme called GSK3b helps control SMN stability. Nearly all proteins degraded by GSK3b are flagged for degradation by a pathway that involves a specific member of the Cullin family. Rubin said the researchers hypothesized that if they could block that Cullin-mediated process, the SMN proteins would not be flagged for degradation and would remain stable longer.

The researchers, led by co-first author Nadia Litterman, then dosed human and murine motor neurons with a compound known to block the specific Cullin and found that exposure to the compound made SMN proteins more stable and more abundant. As a consequence, the compound promoted survival of all motor neurons, both in human cells in the dish and in mouse models.

Additionally, mice with SMA, even the more severe forms of the disease, had some of their symptoms improve after exposure to the compound.

"This process points to an unexplored therapeutic direction that could benefit patients of not one, but two separate diseases," Rubin said.

Explore further: Hope against disease targeting children

More information: Natalia Rodriguez-Muela et al. Single-Cell Analysis of SMN Reveals Its Broader Role in Neuromuscular Disease, Cell Reports (2017). DOI: 10.1016/j.celrep.2017.01.035

Journal reference: Cell Reports

Provided by: Harvard University

This story is published courtesy of the Harvard Gazette, Harvard University's official newspaper. For additional university news, visit Harvard.edu.

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Scientists find evidence that ALS and SMA could be treated with a ... - Medical Xpress

Spinal Cord Stem Cells Comments Off on Scientists find evidence that ALS and SMA could be treated with a … – Medical Xpress | April 17th, 2017

When you think of tough athletes, football and hockey players quickly come to mind.

But a bowler?

Someone who learned that with determination and the love of family, friends, teammates and one anonymous bone marrow donor living 1,500 miles away striking down a rare and deadly blood disease is indeed possible?

Cameron Hurwitz stands 4-foot-11 and weighs 84 pounds with Skittles in his pockets.

But the Brighton (Rochester, N.Y.) High School freshman is a big man on the lanes, leading the Barons this season with a 216.5 average, making the coveted six-man state tournament composite team, where he led Section V to a third-place finish, and being named All-Greater Rochester for the second time in three seasons.

He has rolled three 300-games (two sanctioned) and just recently recorded a personal-best 799 series in competition.

There was a time when opponents sized up Hurwitz and took him for an easy mark. No more.

Hes pretty well-known now, Brighton coach Jason Wasserman said. What they cant believe is thathes only in ninth grade and doing as well as he is. He reads lane conditions as good as anyone out there. Hes able to make adjustments on the fly, he knows what equipment to use at what time and then hes just so consistent with his shots.

Thats what happens when you bowl nearly every day from the time youre eye level to a ball rack. When you have parents, Caryn and Scott Hurwitz, who nurture your gifts with unconditional love. When a big brother, Reese, a senior on the Brighton team with a fine 210 average of his own and is headed to Purdue to bowl, is always there to cheer the strikes and help you handle the splits and open frames of life.

Cameron, 14, a hard-throwing right-hander, throws a ball that takes a sharp, last-second right-to-left hook into the pocket that makes pins explode like fireworks on the Fourth of July.

He has had many mentors but in large part he is a self-taught prodigy.

As a big PBA fan who would like to compete on tour someday, he has long watched bowling on television and the internet. He reads bowling magazines, studies the history of the gameand can recite the career statistics of PBA stars. His favorite player is a kindred spirit, 5-foot-5 Norm Duke, a family friend whose autograph he wears proudly on his green Storm bowling shirt.

For good measure, Cameron drills his own balls, customizes his own bowling shoes (blue and fluorescent green on this day), and has ideas for other bowling products that his dad, who owns a motorcycle parts manufacturing business, helps bring to life. Some have already caught the attention of people in the industry.

I think it came from watching the pros on television all the time and picking it up, Cameron said when asked where his style and passion for all things bowling comes from. I love all the physics behind bowling and just the fact you have to use your mind to be able to perform. Anybody of any size can be great at bowling as long as you know the right way to do it and as long as you know what each piece of equipment does for a particular oil pattern.

Bowling alone during off-hours, wearing a mask to prevent against infection, Cameron Hurwitz never gave up on dream of normal life and returning to Brighton High School team.(Photo: CARYN HURWITZ)

Understanding bowling science helped Cameron enjoy his best season so far, but it was medical science that got him back on the lanes.

A little more than two years ago while in the seventh grade, Cameron was getting ready to leave for the Section V tournament when his mother spotted black-and-blue marks on his arms and legs. A phone call to their family doctor led to blood work, which led to instructions to take her son to the emergency room immediately.

He had extremely low platelets, which clot your blood, and they told us to pack a bag, youll be there for many days, Caryn Hurwitz said.

It was six days to be exact, during which Cameron was diagnosed with Aplastic Anemia, a rare and serious blood disorder in which the body stops making enoughnew white and red cells and platelets.

His bone marrow had just shut down and with so few platelets he was at great risk, and with no immunity he couldnt be around people, Caryn Hurwitz said.

While undergoing treatments at Golisano Childrens Hospital, Cameron was unable to attend school and was quarantined at home for over five months. When given the OK by doctors, his lone escape was making trips to area bowling centers where generous owners allowed him to practice during off-hours to the public.

Encouraged by upticks in his white cell counts, Camerons caregivers couldnt say no when he begged to compete in the prestigious United States Bowling Congress Junior Gold national championships in the Chicago area in July 2015. While wearing an antiviral mask and in between receiving seven-hour blood transfusions at a Chicago hospital, Cameron made the televised final, placing second in the U12 division.

The boy behind the mask became a media celebrity and inspiration in the bowling community. He made the cover of Bowlers Journal and PBA stars became his fans. Hall of Famer Pete Weber posted a good luck video message on Facebook to Cameron.

Hed bowl without hardly any oxygen (in his bloodstream), Caryn Hurwitz said. I dont think people really understood how hard it was for him, but as long as he could go, even with the low blood counts, he kept bowling. When I think about, Im amazed.

Unfortunately for Cameron, the treatments he received didnt produce the desired results and as his eighth-grade school year began, he was placed on the national Be the Matchbone marrow registry.

Waiting times for a match can vary, but in Camerons case one was found in just a few months. And on Dec. 29, 2015 he underwent a transplant at Boston Childrens Hospital, a painstaking procedure where a patients body is re-started with new stem cells that need time to grow and take hold.

Six weeks in the hospital were followed by six more months of isolation, school tutoring, the entire Hurwitz family living in the germ-free lane, and the family bonding like an alleys glued wooden strips.

Throughout his recovery, Cameron kept bowling after hours, determined to be ready for his freshman season. Bowling had become his medicine.

For the full story, visit the Rochester (N.Y.) Democrat and Chronicle

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Bone Marrow Stem Cells Comments Off on N.Y. bowler rolls on following bone marrow transplant – USA TODAY High School Sports | April 17th, 2017

Having worked at University Malaya Medical Centres (UMMC) Paediatrics Department for 20 years, senior consultant paediatric oncologist Prof Dr Hany Mohd Ariffin has had to tell her fair share of parents that there is nothing more that can be done for their terminally ill child.

As head of the Paediatric Haematology-Oncology and Bone Marrow Transplantation Unit, this is usually because there is no suitable donor available for a life-saving bone marrow transplant for the child.

Bone marrow transplants, also called stem cell transplants, are used in conditions where the patients bone marrow is damaged or destroyed by disease or intensive cancer treatment, and is unable to carry out its job of producing healthy red blood cells, white blood cells and platelets.

Because white blood cells or leukocytes are part of the immune system that protects our body against foreign invaders, it is critical in such a procedure to match the so-called immunological fingerprints of the patient and the donor.

As Prof Hany explains, these fingerprints are known as human leukocyte antigens (HLAs).

HLAs help the immune system distinguish between the bodys own cells and foreign cells, usually bacteria and viruses that infect us, so that our white blood cells can find and destroy them.

It is crucial that a bone marrow donor and the patient have the same HLAs in order to minimise the chances of the donated bone marrows white blood cells considering its new host body as foreign and attacking it.

Perfect match needed

Standard bone marrow transplantations require that all 10 HLAs in both patient and donor are a match.

As HLAs are inherited half from each parent, this means that only a patients siblings are a possible perfect match.

Explains Prof Hany: If you look at statistics, out of four, one sibling will be completely matched, one sibling will be completely not matched, and two siblings would be half-matched.

So, the chances of finding a match is 25%, but that is statistical randomisation.

In the real world, you can have 10 siblings and all of them might not be matched with you.

If a patient does not have a sibling that matches perfectly with them, or does not have a sibling at all, their only other option is to check for an unrelated match in international stem cell registries or blood banks.

However, Prof Hany notes that this usually requires a sum of RM100,000 for a unit of bone marrow and at least three to four months of waiting two luxuries not all patients have.

She adds: But it is not easy to get a good match for Asians as these registries are usually Caucasian.

And its even worse if you are an Indian patient, as you cant even go to a Taiwanese blood bank.

In the case of Muhammad Yusuff Iskandar Mohd Hambali, time was a critical factor.

The firstborn of two teachers had been referred to UMMC at 10 months of age for recurrent pneumonia.

His mother, secondary school physical education teacher, Aduratun Nasyihin Mokhtar shares: He started falling sick at the age of seven months he had a persistent cough.

Initially, the doctor thought it was pertussis, but it didnt get better after three months as pertussis should, so he was admitted to the hospital.

However, none of the antibiotics they tried worked, so he was referred to UMMC to check his lungs.

This filepic shows a thalassaemia patient with his infusion pump machine for iron-chelating therapy. Thalassaemia is one of the conditions curable by a bone marrow transplant.

It was in UMMC that Yusuff, as he is called, was discovered to have X-linked severe combined immunodeficiency (SCID).

This rare genetic condition, also known as bubble boy disease, results in the malfunction or lack of two specialised white blood cells called T and B cell lymphocytes.

This means that Yusuff effectively had a non-existent immune system.

This was the reason he could not fight off the pneumonia. In fact, his lungs had deteriorated so badly that he was on oxygen therapy from the age of eight months.

In addition, the Mycobacterium bovis in his BCG vaccination had spread to his back, he had chronic diarrhoea and he was very much underweight.

Yusuff needed a bone marrow transplant, and he needed it fast.

Having reached out to her international colleagues at that time, Prof Hany says: One thing constant in all their advice was that if we delayed the procedure, he would never get better from his disseminated BCG, his pneumonia would just worsen, and once you reach a critical point, there would be no turning back.

He would have been dead by six months.

The problem was that Yusuff was then an only child.

Although his mother was pregnant with his younger sister at that time, she would not have been born in time to help him, assuming that she was a match for him in the first place.

With no time to waste, Prof Hany and her team decided to try a procedure called haplo-identical bone marrow transplantation.

On whether she and her team were ready to carry out the new procedure, Prof Hany says that you will never be ready until a life is dangling precariously in front of you. Photo: The Star/Samuel Ong

In this procedure, only five out of 10 HLAs need to be matched in order for the donor to be able to give bone marrow to the patient.

The beauty of this procedure is that you always have two parents (to donate), says Prof Hany.

So, Yusuffs father, sports science and physical education teacher Mohd Hambali Din @ Ismail, could now donate his bone marrow cells to his son.

First though, Yusuff needed to be fattened up via nutritional fluids infused into his veins, his pneumonia brought under control and his M. bovis infection treated with anti-tuberculosis therapy.

This was so that he would be in a decent enough condition to withstand the procedure.

Following the protocol established by Johns Hopkins University in the United States, but modified to suit Yusuffs condition, Prof Hany and her team first killed off Yusuffs remaining bone marrow cells through chemothera-py, before infusing 30ml of his fathers donated bone marrow into him.

Prof Hany explains that it takes two to three weeks for the new bone marrow cells to grow, during which time the patient is completely vulnerable to any infection.

This is why they remain in a completely sealed room where the air is hepa-filtered, they receive no visitors, and their food and linen are completely sterile, she says.

He was also treated with high-dose cyclophosphamide, a chemotherapy drug that targets T cell lymphocytes.

This was in order to destroy the half-matched mature T cells that came with his fathers donated bone marrow.

T cells are your soldier cells. His fathers T cells would recognise Yusuff as foreign and destroy everything in their wake.

And that is what has precluded mismatched transplants all this while, explains Prof Hany.

After the mature T cells are destroyed, she says: What you then get are T cells from stem cell origin, which learn to tolerate the environment of being in Yusuffs body, and therefore, they will be less aggressive and more friendly to these cells that they consider foreign.

Despite that, Yusuff still experienced graft-versus-host disease (GvHD) where his new white blood cells attacked the cells of his skin, gut and lungs.

In between, he also had two episodes of sepsis and he had to go to the ICU once.

He also had to go on the ventilator at one point, says Prof Hany.

She explains that GvHD, which is due to aggressive donor white blood cells, and infections, which are due to the still incomplete immune system, can co-exist, creating a dilemma for the medical team.

On the one hand, to ameliorate GvHD, you have to give steroids (in addition to standard immunosuppresants) to dampen down the immune system.

You dampen down the immune system, then you allow bacteria and fungi to grow.

And that is why it is very challenging, she says.

She admits: For the first 20 days, it was all very smooth and you think, Wah, Im a hero, but then the challenges came.

There were certain moments when I thought, Thats it, were going to lose him.

It took 149 days after the transplant before Yusuff was deemed well enough to be sent home.

And it was one year before Prof Hany and her team felt confident enough to declare him cured.

We estimate anything between six months to a year for the new bone marrow cells to grow and propagate.

So usually, after a year, if the GvHD doesnt appear anymore, it is very unlikely to suddenly appear, she explains.

This first anniversary of Yusuffs transplant, celebrated at UMMC on April 6, was not just sweet because of Yusuffs survival, it was also the opening of a new path for Prof Hany and her team.

On a personal note, there were many times when you have this period of self-doubt.

So, you think that we are just a bunch of stupid, gung-ho people, who are unrealistic; this is not America, this cannot be done that sort of feeling.

There were some moments when you think, have I done a disservice to this child? Would if it have been better to just let go, for the parents to just let go? Is God just testing me? shares Prof Hany.

However, a few months after Yusuffs transplant, she received the case of a baby boy with myelodysplastic syndrome.

Myelodysplastic children will progress to develop acute myeloid leukaemia within a year, and it is only curable with transplant, or not it is certain death by two years, she explains.

And this patient had two siblings, both of whom were only half-matched.

But we were able to offer a transplant to this child, because we knew that from the experience of Yusuff, if he has very bad GvHD of the gut, skin, lung, we would be able to handle it been there, done that.

We were already scarred for life, she says with a laugh. And in fact, due to their prior experience, Prof Hany and her team were able to more precisely determine the amount of donated bone marrow cells needed for transplant.

As a result, she says: The second patient sailed through and was discharged after only five weeks, as opposed to five months for Yusuff.

Explaining the potential impact of having this treatment option available, Prof Hany shares that bone marrow transplantation is a cure for conditions like leukaemia, blood disorders like thalassaemia, congenital defective immune systems and certain rare congenital metabolic conditions.

The major reason why transplants are not being done is because of the lack of an available donor, she says.

But haplo-identical bone marrow transplantation now opens the way for many more potential donors to help the patient.

The learning curve is steep, Prof Hany admits, but adds that after Yusuff, they were able to apply what they learnt to their second patient with great effect.

Im not saying it is easy, but I think it is worth developing further, because it can solve one of the greatest health problems in our country, which is inherited blood disorders.

Giving the example of thalassaemia, she estimates that it costs some RM3.5mil to treat a patient with regular blood transfusions and iron-chelating therapy for 30 years.

A haplo-identical bone marrow transplant costs approximately RM45,000 and will cure the patient.

The risk of dying from this procedure usually because of infections and GvHD during the period when the patient has no working immune system is estimated to be about 10%.

This is at the upper limit for standard bone marrow transplants, where the risk ranges from 5% to 10%.

She adds that studies have shown that the risk of severe GvHD is similar for haplo-identical transplants and sibling-matched transplants, which are both lower than transplants from an unrelated donor.

Although Yusuff is the first successful haplo-identical bone marrow transplant patient in the country, to the best of Prof Hanys knowledge, she believes that the procedure can be easily done in other major hospitals around the country.

The facilities are already there and specialists trained in bone marrow transplants need only learn the procedure once before they should be able to conduct it, she says.

So its not just having a big celebration to tell the world that we saved one boy with SCID, its having the ability to tell parents that there is always hope, as we can now do haplo-identical transplants in our centre, says Prof Hany.

It is about no longer having to tell parents that nothing more can be done for their terminally ill child.

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New hope with haplo-identical bone marrow transplant - Star2.com

Bone Marrow Stem Cells Comments Off on New hope with haplo-identical bone marrow transplant – Star2.com | April 15th, 2017

A team of biomedical engineering researchers, led by the University of Minnesota, has created a revolutionary 3D-bioprinted patch that can help heal scarred heart tissue after a heart attack. The discovery is a major step forward in treating patients with tissue damage after a heart attack.

The research study is published today in Circulation Research, a journal published by the American Heart Association. Researchers have filed a patent on the discovery.

According to the American Heart Association, heart disease is the No. 1 cause of death in the U.S. killing more than 360,000 people a year. During a heart attack, a person loses blood flow to the heart muscle and that causes cells to die. Our bodies cant replace those heart muscle cells so the body forms scar tissue in that area of the heart, which puts the person at risk for compromised heart function and future heart failure.

In this study, researchers from the University of Minnesota-Twin Cities, University of Wisconsin-Madison, and University of Alabama-Birmingham used laser-based 3D-bioprinting techniques to incorporate stem cells derived from adult human heart cells on a matrix that began to grow and beat synchronously in a dish in the lab.

Watch a video of the cells beating on the patch.

When the cell patch was placed on a mouse following a simulated heart attack, the researchers saw significant increase in functional capacity after just four weeks. Since the patch was made from cells and structural proteins native to the heart, it became part of the heart and absorbed into the body, requiring no further surgeries.

This is a significant step forward in treating the No. 1 cause of death in the U.S., said Brenda Ogle, an associate professor of biomedical engineering at the University of Minnesota. We feel that we could scale this up to repair hearts of larger animals and possibly even humans within the next several years.

Ogle said that this research is different from previous research in that the patch is modeled after a digital, three-dimensional scan of the structural proteins of native heart tissue. The digital model is made into a physical structure by 3D printing with proteins native to the heart and further integrating cardiac cell types derived from stem cells. Only with 3D printing of this type can we achieve one micron resolution needed to mimic structures of native heart tissue.

We were quite surprised by how well it worked given the complexity of the heart, Ogle said. We were encouraged to see that the cells had aligned in the scaffold and showed a continuous wave of electrical signal that moved across the patch.

Ogle said they are already beginning the next step to develop a larger patch that they would test on a pig heart, which is similar in size to a human heart.

The research was funded by the National Science Foundation, National Institutes of Health, University of Minnesota Lillehei Heart Institute, and University of Minnesota Institute for Engineering in Medicine.

In addition to Ogle, other biomedical engineering researchers who were part of the team include Molly E. Kupfer, Jangwook P. Jung, Libang Yang, Patrick Zhang, and Brian T. Freeman from the University of Minnesota; Paul J. Campagnola, Yong Da Sie, Quyen Tran, and Visar Ajeti from the University of Wisconsin-Madison; and Jianyi Zhang, Ling Gao, and Vladimir G. Fast from the University of Alabama,

To read the full research paper entitled Myocardial Tissue Engineering With Cells Derived from Human Induced-Pluripotent Stem Cells and a Native-Like, High-Resolution, 3-Dimensionally Printed Scaffold, visit the Circulation Research website.

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3D-printed patch can help mend a broken heart - UMN News

Cardiac Stem Cells Comments Off on 3D-printed patch can help mend a broken heart – UMN News | April 14th, 2017

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'Cradle of life' stem cells taken from skin samples were developed into three-dimensional brain-like organisms capable of exchanging signals between each other in a network.

The petri dish cells behave in a similar way to the brain cells which produce messenger dopamine from neurons - and scientists hope they will be able to use them to come up with a cure for Parkinson's.

Dopamine maintains smooth body movements, but when the neurons die off, tremors, rigid muscles and other Parkinson's disease symptoms begin to take over.

The new developments mean scientists can now use the cells to test what environmental factors like pollutants have on the onset of the disease and potentially find a cure.

Lead author Professor Jens Schwamborn said: "Our cell cultures open new doors to brain research.

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"We can now use them to study the causes of Parkinson's disease and how it could possibly be effectively treated."

Our cell cultures open new doors to brain research

Professor Jens Schwamborn

The stem cells can be transformed into any cell type of the human body but cannot produce a complete organism.

PHD student Anna Monzel developed a procedure to convert the stem cells into brain cells as part of her doctoral thesis.

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Tremor - One of the most noticeable signs of Parkinson's is a tremor that often starts in the hands or fingers when they are relaxed

She said: "I had to develop a special, precisely defined cocktail of growth factors and a certain treatment method for the stem cells, so that they would differentiate in the desired direction."

Prof Schwamborn from the Luxembourg Centre for Systems Biomedicine at Luxembourg University said: "Our subsequent examination of these artificial tissue samples revealed that various cell types characteristic of the midbrain had developed."

"The cells can transmit and process signals.

"We were even able to detect dopaminergic cells - just like in the midbrain."

The scientists say their petri dish study can also reduce the amount of animal testing in brain research.

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Because cell cultures in the petri dishes are of human origin in some aspects they resemble human brains more than the brains of lab animals such as rats or mice.

Professor Schwamborn added: "There are also attractive economic opportunities in our approach.

"The production of tissue cultures is highly elaborate.

"In the scope of our spin-off Braingineering Technologies Sarl, we will be developing technologies by which we can provide the cultures for a fee to other labs or the pharmaceutical industry for their research."

The study was published in the Stem Cell Reports journal.

Link:
Scientists one step closer to turning stem cells into BRAIN | Health ... - Express.co.uk

Skin Stem Cells Comments Off on Scientists one step closer to turning stem cells into BRAIN | Health … – Express.co.uk | April 14th, 2017

(NAPSI)You may be surprised to learn that multiple myeloma is the second most common cancer of the blood, after leukemia. It starts in plasma cells, a type of white blood cell. In time, myeloma cells collect in the bone marrow and may damage the solid part of the bone and eventually harm other tissues and organs, such as the skeleton and the kidneys.

In fact, there are approximately 114,000 new cases diagnosed every year. If you or a loved one is among the 230,000 people living with multiple myeloma worldwide there are a few facts you should know.

What Can Be Done

For many people with the disease, an autologous stem cell transplant may be an answer for eligible patients. This involves collecting the patient's own blood-forming stem cells and storing them. He or she is then treated with high doses of chemotherapy or a combination of chemotherapy and radiation. This kills cancer cells but also eliminates the remaining blood-producing stem cells in the bone marrow. Afterward, the collected stem cells are transplanted back into the patient, so the bone marrow can produce new blood cells.

To help people learn more about the disease and its treatments, the Multiple Myeloma Journey Partners Program was created.

This peer-to-peer education program for patients, caregivers and health care providers leverages storytelling as a tool to improve the patient experience. Journey Partners are multiple myeloma patients who have experienced similar emotions, faced the same challenges and asked the same questions about living with the disease. A Multiple Myeloma Journey Partner will come to any community in which 10 or more people would like to attend the free one-hour educational seminar. The main benefit is that multiple myeloma patients know they're not alone, and the program provides educational resources and services that help patients and families navigate their journey to achieve the best possible outcomes.

As John Killip, a Multiple Myeloma Journey Partner, puts it, "It was conversations with my support group, family and health care providers that influenced my decision to have a stem cell transplant in 2008, when I was first diagnosed with multiple myeloma, at the age of 65. Mentoring other multiple myeloma patients is one of the highlights of my life. I became a Journey Partner to share my story and help others with the disease make sense of the diagnosis and overcome the fear of the unknown."

Learn More

For more information or to request a program, you can visit http://www.mmjourneypartners.com. Anyone interested in becoming a Multiple Myeloma Journey Partner can contact the program coordinator listed on the website. The program is sponsored by Sanofi Genzyme, the specialty care global business unit of Sanofi focused on rare diseases, multiple sclerosis, immunology, and oncology.

On the Net:North American Precis Syndicate, Inc.(NAPSI)

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Understanding Multiple Myeloma - Caswell Messenger

Bone Marrow Stem Cells Comments Off on Understanding Multiple Myeloma – Caswell Messenger | April 11th, 2017

A team of scientists from the University of California, Los Angeles has been able to synthesize an artificial thymus, a human organ that is important to the bodys immune system. An artificial thymus, they say, could produce necessary cancer-fighting T-cells for the body.

On demand.

T-cells, of course, are white blood cells which naturally fight diseases that develop in or infect the body. These T-cells are artificial, though, so they would have to be engineered to target specific forms of cancer, in order to be effective. Still, if this is manageable, then it could provide scientists and health practitioners with additional natural defensesalbeit, bionicfor attacking disease.

The thymus rests in front of the heart. It uses stem cells from the blood to make immune-boosting T-cells, which literally circulate throughout the body to specifically target things that dont belong. In this case, the thymus would create T-cells that could seek out specific cancerous growths without jeopardizing the health of existing tissue.

For the study, the Japanese researchers looked at 27 patients who had received transplants form stem cells that had been taken from their own thigh muscles. These patients showed no sign of any major complications; most patients also showed significant improvement with their symptoms.

Research team member Gay Crooks comments, We know that the key to creating a consistent and safe supply of cancer-fighting T-cells would be to control the process in a way that deactivates all T-cell receptors in the transplanted cells, except for the cancer-fighting receptors. It is important, of course, to take stem cells from the patient who needs them because the body is likely to reject any foreign stem cells (and their byproducts). Apparently, they have been at this study for more than two decades but, unfortunately, the researchers acknowledge that past attempts only showed modest results. From these results, though, they were able to devise a method for producing sheets of muscle stem cells which could then be attached to the inner layer of the sac (which encloses the heart). These stem cells will stimulate healing through the production of chemicals which encourage cardiac regeneration, though the stem cells, themselves, do not survive in the long term.

The results of this study have been published in the scientific journal Nature Methods.

Read more:
Can An Artificial Thymus, Made from Stem Cells, Pump Out Enough T-Cells To Fight Cancer? - Dispatch Tribunal

Cardiac Stem Cells Comments Off on Can An Artificial Thymus, Made from Stem Cells, Pump Out Enough T-Cells To Fight Cancer? – Dispatch Tribunal | April 9th, 2017

April 6, 2017 7:01 PM

NEW YORK (CBSNewYork) Theres potentially exciting news for the two and a half million people around the world struggling with multiple sclerosis.

There is no known cure, but now an experimental treatment in Israel may be able to reverse the symptoms, CBS2s Dr. Max Gomez reports.

MS is a progressive degenerative disease where the insulation around nerve fibers in the brain and spinal cord starts to break down. Its the immune system attacking the insulation.

Medications can slow the disease but dont stop it. Stem cells may be much better.

As Dr. Max reports, walking on a treadmill is a big step for Malia Litman. She had been a top trial attorney in Dallas until she was diagnosed with multiple sclerosis 18 years ago. Slowly, the disease robbed her of her balance, her mobility and her energy.

You can imagine how contracted my world had become, she says.

After she fell and broke her leg, she was in a wheelchair for weeks. Her MS medicines werent really working anymore.

Her search for alternative treatments led toDr. Dimitrios Karussis.

Answers for our diseases and our medical problems are hidden inside our body, hesays.

Karussis heads the experimental stem cell research atHadassah Medical Organization in Israel. He harvests an MS patients own adult stem cells from their bone marrow, then injects them back into their spinal fluid.

As neurologists, we have never seen or even believed that it is possible to reverse any disability, he says.

Litman says within 24 hours of her first treatment, I picked up my leg and went, Oh my god, and I just started crying.

She says her speech is more clear and she has more energy, and shes adamant its not a placebo effect, pointing to a number of tests before and after treatment that show improvement.

Karussis says one patient was even able to walk again.

Researchers are now collaborating with teams at the Mayo Clinic and Harvard, finishing a double-blind study to prove its effectiveness.

Look what I can do now! Its amazing, Litman says.

She still uses her walker but can now get on her rowing machine. After four treatments, shes reactivated her law license and is taking on a case.

I feel like I have my life back. I dont care if I walk with a walker the rest of my life. Although I think I may actually be able to walk again with a couple more treatments, she says.

As Dr. Max reports, the theory is that the stem cells are somehow spurringthe regeneration of the insulating nerve sheaths that are deteriorating in MS.

However, the course of the disease is so variable that Litmans improvement may not be due to the stem cell treatment. Thats why the double-blind studyis so important.

Hadassah Medical Organizations researchers are also looking at the treatments effect on ALS patients.

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Experimental Stem Cell Treatment Might Reverse Symptoms Of Multiple Sclerosis - CBS New York

Bone Marrow Stem Cells Comments Off on Experimental Stem Cell Treatment Might Reverse Symptoms Of Multiple Sclerosis – CBS New York | April 7th, 2017

Scientists report another step in the use of stem cells to help treat people with debilitating heart failure.

In an early study of 27 patients, Japanese researchers used patients' own muscle stem cells to create a "patch" that was placed on the heart.

Over the next year, the patients generally showed small improvements in their symptoms -- including the ability to walk without becoming breathless and fatigued.

However, experts cautioned that while the results are encouraging, there's a lot of work left ahead before stem cells can be used to treat heart failure.

"They've shown that this approach is feasible," said Dr. Eiran Gorodeski, a heart failure specialist at the Cleveland Clinic in Ohio.

But it's not clear whether the stem-cell tactic was actually effective, said Gorodeski, who was not involved in the study.

RELATED: Antidepressant No Help to Heart Failure Patients

That's because the study didn't include a comparison group that did not receive stem cells.

So it's possible, Gorodeski explained, that the "modest" symptom improvements would have happened anyway. All of the patients were on standard medications, and some had heart devices implanted.

Stem cells are primitive cells that mature into the various cells that make up the body's tissues. In the past 15 years or so, scientists have tried to use the cells to help repair some of the damage seen in heart failure.

Heart failure is a progressive disease where the heart muscle is too damaged to efficiently pump blood throughout the body. It often arises after a heart attack.

Symptoms of heart failure include fatigue, breathlessness and swelling in the limbs. The condition cannot be cured, although medications and implantable devices can treat the symptoms.

In the new study, the researchers used stem cells from the patients' own thigh muscle to create a patch they placed on the heart.

That's in contrast to many past studies, where researchers have injected stem cells -- often from patients' bone marrow -- into the heart.

The patch tactic could have some advantages, said senior researcher Dr. Yoshiki Sawa, of Osaka University.

He said animal research suggests that cells in sheet form survive for a longer period, compared to injections.

To test the safety of the approach, Sawa's team recruited 27 patients who had debilitating symptoms despite standard heart failure therapies. The scientists extracted stem cells from each patient's thigh muscle, then cultured the cells so that they formed a sheet.

The sheet was placed on each patient's heart.

The tactic appeared safe, the researchers said, and there were signs of symptom improvements over the next six months to a year.

Why would stem cells from the thigh muscle affect the heart? It's not clear, Sawa acknowledged.

The stem cells don't grow into new heart muscle cells. Instead, Sawa explained, they seem to produce chemicals called cytokines that can promote new blood vessel growth in damaged areas of the heart. The theory, he said, is that "hibernating" cells in the heart muscle can then function better.

Still, it's too soon to know what the new findings mean, said Gorodeski.

This type of trial, called phase 1, is designed to look at the safety and feasibility of a therapy, Gorodeski said. It takes later-phase trials -- where some patients receive the treatment, and others do not -- to prove that a therapy actually works.

Those trials are underway, Sawa said.

Other studies are further along. Last year, researchers reported on a trial testing infusions of stem cells taken from the bone marrow of patients with severe heart failure.

Patients who received the therapy were less likely to die or be hospitalized over the next year, versus those given standard treatment only. But the study was small, and the stem cells had only a minor impact on patients' heart function.

So it's not clear why the stem-cell patients fared better, Gorodeski said.

For now, he stressed, all stem-cell therapies for heart failure remain experimental.

"There's no cell therapy that we can offer patients right now," Gorodeski said.

The message for patients, he added, is that heart failure can be treated, and researchers are looking for "innovative" ways to improve that treatment.

The study was published April 5 in theJournal of the American Heart Association.

Read more here:
Can Stem Cell 'Patch' Help Heart Failure? - Everyday Health (blog)

Bone Marrow Stem Cells Comments Off on Can Stem Cell ‘Patch’ Help Heart Failure? – Everyday Health (blog) | April 7th, 2017

Home Science & Tech

By Malak Abason April 5, 2017

Lungs are a crucial organ in many animals, including humans. While their function has always seemed pretty straightforward to take in oxygen, transfer it into blood, and exhale carbon dioxide scientists have found a previously unrecognized function of the lungs of mice: blood production.

The study, which was published in Nature by researchers at the University of California San Francisco, was performed by inserting fluorescent protein into the mouses genome.

The protein caused the platelets (small blood cells that bind together to help create blood clots when a blood vessel is damaged), in the mouse to glow, allowing scientists to trace the platalets paths. What they found was a massive number of megakaryocytes, a stem cell that produces in the lungs.

When researched further, scientists found that the lung was producing over 10 million blood-producing platelets per hour, and the platelets produced by the lung accounted for the majority of platelets in the mouses circulatory system. Researchers are theorizing that the megakaryocytes are created in the bone marrow, but then travel to the lung to produce platelets.

While it is known that human lungs produce platelets and produce blood, as small amounts of megakaryocytes have been found in lungs before, if these findings are reproduced in humans, it will prove that the sheer amount that lungs produce has been greatly underestimated.

The study also found a reservoir of stem cells with the ability to become blood cells in the lungs. Researchers implanted lungs with the fluorescent megakaryocyte cells into mice that had been engineered to have no blood stem cells in their bone marrow, and found that the fluorescent cells travelled from the lungs to the marrow, and helped to produce platelets and other ingredients in blood, including neutrophils. In cases where the bone marrow is dealing with platelet or stem cell deficiency, these stem cells were able to leave the lung and contribute to the refilling of platelets in the marrow.

If further research indicates that these findings also apply to humans which they very well may, considering the genetic and biological similarities between mice and humans it will not only disprove the current theory that states the bone marrow accounts for most of the human bodys platelet production, but it will also affect how scientists approach treating blood diseases in humans, particularly ones that result in a platelet deficiency, such as thrombocytopenia.

Link:
Lungs of mice found to produce blood - The Manitoban

Bone Marrow Stem Cells Comments Off on Lungs of mice found to produce blood – The Manitoban | April 5th, 2017

April 5, 2017

Stem cell differentiation can now be seen thanks to a combination of machine learning and microfabrication techniques developed by scientists at the RIKEN Quantitative Biology Center in Japan. The results, published in PLOS One, followed the differentiation of human mesenchymal stem cells (MSC) which are easily obtained from adult bone marrow.

MSCs have proven to be important for regenerative medicine and stem cell therapy because they can potentially repair many different types of organ damage, as they have the ability to differentiate into various cell types including bone, muscle and fat. Depending on the way the cells are grown the results can be quite different and so controlling differentiation is an important goal.

Observing MSC differentiation under different conditions is an essential step in understanding how to control the process. However, this has proved challenging on two fronts. First, the physical space in which the cells are grown has a dramatic impact on the results, causing significant variation in the types of cells into which they differentiate. Studying this effect requires consistent and long lasting spatial confinement. Second, classifying the cell types which have developed through manual observation is time consuming.

Previous studies have confined cell growth with fibronectin on a glass slide. The cells can only adhere and differentiate where the fibronectin is present and are thus chemically confined. However, this procedure requires high technical skill to maintain the confinement for an extended period of time. To overcome this, the first author of the study, Nobuyuki Tanaka, decided to look for a new way to confine them. Using a simple agarose gel physical confinement system, he found that he could maintain them for up to 15 days. Tanaka says, "It was wonderful to be able to do this, because agarose gel is a commonly used material in biology laboratories and can be easily formed into a micro-cast in a PDMS silicone mold."

He continues, "The advantage of this system is that once the PDMS molds are obtained the user only needs agarose gel and a vacuum desiccator to create highly reproducible micro-casts." The vacuum pump pulls the agarose gel into the mold. He explains, "We provided the protocol to our coauthors at ETH Zurich and they performed the agarose micro-casting and conducted the stem cell differentiation study. Stem cells were captured in the micro-structures and their differentiation was controlled under the captured condition."

Tanaka's paper also describes an automated cell type classification system, using machine learning, which reduces the time and labor needed to analyze cells. "Combined together, these tools give us a powerful way to understand how stem cells differentiate in given conditions."

According to Yo Tanaka, leader of the Laboratory for Integrated Biodevice, where the research was conducted, "We hope this will break down the barriers that have hindered research in this area so far and help to establish harmony between biologists and engineers. The focus of engineers has traditionally been to develop new technologies, but scientists prefer to use well established technologies. However, if our newly developed technology is simple enough it can spread rapidly, this is our goal."

Explore further: Stem cells seem speedier in space

More information: PLOS One (2017). DOI: 10.1371/journal.pone.0173647

Journal reference: PLoS ONE

Provided by: RIKEN

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This is really great! Getting stem cells to differentiate in to desired adult cells is what is holding back stem cell therapies. This is a MAJOR step in that direction!

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New technique helps researchers determine how stem cells differentiate - Phys.Org

Bone Marrow Stem Cells Comments Off on New technique helps researchers determine how stem cells differentiate – Phys.Org | April 5th, 2017

A small, but promising study suggests that stem cells from a childs own cord blood may offer an effective treatment for autism symptoms.

Most children on the spectrum who received an infusion from their own umbilical cord blood showed improvements in behavior, communication and socialization, among other measures, while experiencing no significant downsides from the treatment.

The findings come from a study of 25 kids with autism ages 2 to 5 published Wednesday in the journal Stem Cells Translational Medicine.

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All of the children who participated in the research had their cord blood banked at birth. For the trial, the kids were given a series of behavioral and functional assessments before receiving a one-time cord blood infusion. Follow-up assessments were conducted at six and 12 months after the infusion.

Not only did the researchers find that the treatment was safe, but parent reports as well as clinical assessments indicated that more than two-thirds of the children saw improvements in autism symptoms.

Most of the behavioral gains were seen in the first six months after the infusion, the study found, but they were sustained over the following six months.

We are pleased that this study demonstrated the safety of treating children with ASD with their own cord blood, said Joanne Kurtzberg, a pediatric bone marrow transplant specialist at Duke Health who worked on the study. Were also encouraged that, while small and non-randomized, there were observed improvements in a majority of the children reported by clinicians and parents.

While the findings are encouraging, researchers said that further study involving more participants is needed before any firm conclusions can be reached about the effectiveness of cord blood infusions.

We are now hoping to replicate these preliminary results in a Phase II randomized clinical trial for which enrollment is nearly complete, Kurtzberg said.

Originally posted here:
Stem Cells Show Promise For Treating Autism - Disability Scoop

Bone Marrow Stem Cells Comments Off on Stem Cells Show Promise For Treating Autism – Disability Scoop | April 5th, 2017

Alexes Harris tells KUOW's Katherine Banwell her story.

When ProfessorAlexes Harris learned she had a rare form of leukemia, she knew she was in a fight for her life. But she didn't realize how difficult it would be to find a bone marrow match as a woman of color. This is her story.

I have a rare blood cancer called myelodysplastic syndrome.

I was diagnosed in May 2016 after a year of various tests.Prior to being diagnosed, my only health complaints were a random onset of what felt like asthma attacks during my cycling classes (the only reason I went to the doctor), feeling very tired, and not always thinking clearly. I was told that if I did not begin treatment right away I would have two years to live.

Im a 41-year-old mother of a 9 year old and 5 year old (and wife to an amazing husband), so my only true option was to begin treatment.

After being presented with treatment options, we opted for an intensive round of in-patient chemotherapy, which I underwent in June 2016 and managed symptoms in July, 2016.During my initial diagnosis I learned that I would eventually need a bone marrow or stem cell transplant. This would be my only hope of a cure.

We immediately started research to learn about how matches were found and I discovered that because I am a person with a mixed race and ethnic background (African American, Filipino and white) I would have a difficult time finding a full donor match.

While whites have a 75 percent chance of finding a full match in the existing bone marrow registry, African Americans only have a 19 percent likelihood of finding a match. African Americans comprise only 7 percent of the United States registry.

And, it is projected that by 2017 our likelihood of finding a match will only raise to 21 percent. Within the United States registry, the likelihood for finding a full match is higher for people of Mexican (37 percent), Chinese (41 percent), South Asian (33 percent), Hispanic Caribbean (40 percent) and Native American (52 percent) ancestry than for African Americans, but still significantly lower than the likelihood for whites.

Finding a non-related full match is difficult if you are a person of color, especially people of mixed race origin. Having a 100 percent match is crucial in predicting positive outcomes post-transplant. While the Seattle Cancer Care Alliance has been searching for a match, today, I still do not have a full bone marrow donor match and am moving forward with an alternative stem cell transplant using donated umbilical cord blood. My transplant for using cord blood was in September.

This is why we are organizing a national bone marrow donation registry campaign.I want to make my cancer matter, so my great friends stepped in to make this happen. Our goal is to have 4,000 new people registered by this effort. We need people of all backgrounds to become potential matches to help people like me live.

I am a professor of sociology and teach about social stratification, inequality and racial outcomes in institutional processing.I research class and racial differences in criminal justice processing and outcomes. I am the daughter of a black and Filipino man, wife to a black man, sister to black men, and mother of a black son and daughter.I live in the United States and, as many of us know, understand the racial inequalities in our broader society.Many times I feel overwhelmed about the lack of ability to make institutional differences, be it in our systems of education, criminal justice and health care.

Yet, when it comes to bone marrow donation, and other blood products and organ donation, we can make a difference. We can, for ourselves, save ourselves. Becoming involved in donation empowers us in a way like no other to alleviate health care disparities.

You can learn a lot about my story and this campaignatteamalexes.com. We had bone marrow registries in five cities last fall Seattle, Los Angeles, Houston, Washington, D.C., and New York.

Please consider signing up for the bone marrow registry. You can literally be a superhero and save someones life.

Dr. Alexes Harris is a professor of sociology at the University of Washington. This essay was originally published on her personal website.

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I'm a woman of color with cancer. Here's why I can't find a bone ... - KUOW News and Information

Bone Marrow Stem Cells Comments Off on I’m a woman of color with cancer. Here’s why I can’t find a bone … – KUOW News and Information | April 1st, 2017

It offers hope for sufferers of diseases hitherto thought incurable

Adult Stem Cell Therapy has gained popularity in developed countries as an alternative to the conventional treatment of many diseases. There are several studies and clinical trials conducted in the United States to support this.

Some current line of treatments are not typically effective, and some can cause detrimental side effects. Medicine is evolving to a more natural and more effective means with the use of stem cells. Due to the numerous religious and ethical issues that comes with the use of embryonic stem cells, todays medicine is moving towards the application of Adult Stem Cell Therapy. This article highlights some new applications of adult stem cells in conditions and diseases that has posed a problem in our society for far too long.

Sickle Cell Anemia

Tissue-based treatments is already evident in the United States of America. In 2012, a patient was successfully cured of sickle cell disease after receiving achemotherapy-free stem cell transplant for sickle cell disease. Additional patients have been successfully treated since then. Two studies conducted in 2014 and 2015 have shown that the use of adult stem cell therapy can greatly reduce complications or even stop the progression of the diseases by providing stem cells to the needed areas. This reduces the need for surgeries for many of these patients.

Diabetes

According to International Diabetes Federation (IDF), there were over 40,000 deaths due to diabetes documented in Nigeria in 2015. Treatment for diabetes has been a focal point for medical research for many years. Consequently, some studies and clinical trials conducted have shown that Adult Adipose (fat) Stem Cell Transplantation can lower and regulate sugar levels resulting in reducing or eliminating the amount of medication or insulin that patients need to take.

In a recently conducted clinical trial, some of the patients achieved insulin independence that remained stable for a median time of 29 months, and another patient for 43 months ongoing. In fact, all the patients studied showed substantial improvement in their dependence on insulin and overall diabetic condition.

Sexual Dysfunction

The emergence of Regenerative Medicine (which includes Adult Stem Cell and Platelet Rich Plasma therapy) has positively impacted the sexual life of both women (O-Shot) and men (P-Shot), and the treatment is also being used for urinary incontinence, etc. There is now ample evidence to show that O-Shot helps women increase their sexual responses, the ability to have Vaginal Orgasm, arousal from clitoral stimulation, sexual desire and natural lubrication, arousal from G-spot stimulation, as well as decrease pain during intercourse and tighten vaginal opening.

Furthermore, P-Shotin men regenerates damaged penile tissues faster and stronger than most traditional treatments. In most cases, treated men see increase in length up to 1 inch or more and girth up to 3/4 inch or more while also increasing their sexual stamina.

Arthritis

Adult stem cells transplantationhas also been studied in arthritis, and there has been some positive reports about its efficacy. In 2014, the effects of stem cells for articular cartilage regeneration was studied.They studied the effect of stem cells injection in treating osteoarthritis of the knee, and the results showed significant improvement.

Neurological Disorders (e.g. Spinal Cord Injury)

The usefulness of stem cell therapy in neurological disorders like Multiple Sclerosis, Cerebral Palsy, Spinal Cord Injury, etc. has been shown in different studies and clinical trials. The prognosis for spinal cord injuries is generally believed to be poor. However, recent researches and case studies are changing this ideology as the value of adult stem cell therapy for patients with spinal cord injuries is emerging.

An example of this can be seen in a case study published in 2015. In this case study, a patient with functional loss below the lesion level due to a motor vehicle accident failed standard therapy but saw clinically meaningful improvements after multiple adult stem cell treatments. Stem cell transplantations over a period of months led to the restoration of the patients ability to move lower extremities against gravity, control the body trunk, and the ability to control the bladder. The patient was also able to stand as well as walk with the aid of hip and knee ortheses. The sensation level also increased.

Conclusion

Regenerative medicine involving adult stem cells is continually being studied and researched to gather more evidence to enable harnessing its clinical potentials. The use of adult stem cells for clinical therapy is now a reality for many patients who were not able to shed the yoke of many diseases that conventional medicine provided very little hope of permanent relief for.

One new innovation is the Umbilical Cord Stem Cells transplantation that is now done without the need for HLA matching. This allows anyone to be treated for conditions or diseases where applicable. In case of sickle cell disease, case studies have been published on the use of Umbilical Cord Stem Cells from HLA matching donors to remedy sickle cell disease; however, case studies are yet to be published on the efficacy of the Umbilical Cord Stem Cells not requiring HLA matching for the treatment of sickle cell disease. I am cautious to note that the results may differ from case to case, so not every sickle cell patient will be a good candidate for the treatment.

Currently, Adult Stem Cell Therapy is now seen as a viable therapeutic alternative for joint and back pain, sexual dysfunction, diabetes, End Stage Renal Disease on hemodialysis, arthritis, etc. The treatments are gaining popularity among patients and doctors because it is natural and can help repair and regenerate most parts of the human tissues.

Ikudaiyisi is the Medical Director of Glory Wellness and Regenerative Centre in USA, Lagos and Abuja and can be reached on info@glorywellness.org

Excerpt from:
How Adult Stem Cell Therapy Is Breaking Glass Ceiling Of Modern Medicine - Leadership Newspapers

Spinal Cord Stem Cells Comments Off on How Adult Stem Cell Therapy Is Breaking Glass Ceiling Of Modern Medicine – Leadership Newspapers | March 31st, 2017

SOUTH SAN FRANCISCO, CA--(Marketwired - March 29, 2017) - VistaGen Therapeutics Inc. (VTGN), a clinical-stage biopharmaceutical company focused on developing new generation medicines for depression and other central nervous system (CNS) disorders, announced today that the European Patent Office (EPO) has issued a Notice of Intention to Grant the Company's European Patent Application for AV-101, its oral CNS prodrug candidate in Phase 2 development for major depressive disorder (MDD). The granted claims covering multiple dosage forms of AV-101, treatment of depression and reduction of dyskinesias associated with L-DOPA treatment of Parkinson's disease will be in effect until at least January 2034.

"We are extremely pleased to receive the EPO's notice of intention to grant significant CNS-related patent claims for AV-101, another substantial step forward in our plan to secure a broad spectrum of intellectual property protection for AV-101 covering multiple CNS indications," stated Shawn Singh, Chief Executive Officer of VistaGen.

About AV-101

AV-101 (4-CI-KYN) is an oral CNS prodrug candidate in Phase 2 development in the U.S. as a new generation treatment for major depressive disorder (MDD). AV-101 also has broad potential utility in several other CNS disorders, including chronic neuropathic pain and epilepsy, as well as neurodegenerative diseases, such as Parkinson's disease and Huntington's disease.

AV-101 is currently being evaluated in a Phase 2 monotherapy study in MDD, a study being fully funded by the U.S. National Institute of Mental Health (NIMH) and conducted by Dr. Carlos Zarate Jr., Chief, Section on the Neurobiology and Treatment of Mood Disorders and Chief of Experimental Therapeutics and Pathophysiology Branch at the NIMH, as Principal Investigator.

VistaGen is preparing to advance AV-101 into a 180-patient, U.S. multi-center, Phase 2 adjunctive treatment study in MDD patients with an inadequate response to standard FDA-approved antidepressants, with Dr. Maurizio Fava of Harvard University as Principal Investigator.

About VistaGen

VistaGen Therapeutics, Inc. (VTGN), is a clinical-stage biopharmaceutical company focused on developing new generation medicines for depression and other central nervous system (CNS) disorders. VistaGen's lead CNS product candidate, AV-101, is a new generation oral antidepressant drug candidate in Phase 2 development for major depressive disorder (MDD). AV-101's mechanism of action is fundamentally differentiated from all FDA-approved antidepressants and atypical antipsychotics used adjunctively to treat MDD, with potential to drive a paradigm shift towards a new generation of safer and faster-acting antidepressants. AV-101 is currently being evaluated by the U.S. National Institute of Mental Health (NIMH) in a Phase 2 monotherapy study in MDD being fully funded by the NIMH and conducted by Dr. Carlos Zarate Jr., Chief, Section on the Neurobiology and Treatment of Mood Disorders and Chief of Experimental Therapeutics and Pathophysiology Branch at the NIMH. VistaGen is preparing to launch a 180-patient Phase 2 study of AV-101 as an adjunctive treatment for MDD patients with inadequate response to standard, FDA-approved antidepressants. Dr. Maurizio Fava of Harvard University will be the Principal Investigator of the Company's Phase 2 adjunctive treatment study. AV-101 may also have the potential to treat multiple CNS disorders and neurodegenerative diseases in addition to MDD, including chronic neuropathic pain, epilepsy, Parkinson's disease and Huntington's disease, where modulation of the NMDAR, AMPA pathway and/or key active metabolites of AV-101 may achieve therapeutic benefit.

VistaStem Therapeutics is VistaGen's wholly owned subsidiary focused on applying human pluripotent stem cell technology, internally and with collaborators, to discover, rescue, develop and commercialize proprietary new chemical entities (NCEs), including small molecule NCEs with regenerative potential, for CNS and other diseases, and cellular therapies involving stem cell-derived blood, cartilage, heart and liver cells. In December 2016, VistaGen exclusively sublicensed to BlueRock Therapeutics LP, a next generation regenerative medicine company established by Bayer AG and Versant Ventures, rights to certain proprietary technologies relating to the production of cardiac stem cells for the treatment of heart disease.

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For more information, please visit http://www.vistagen.com and connect with VistaGen on Twitter, LinkedIn and Facebook.

Forward-Looking Statements

The statements in this press release that are not historical facts may constitute forward-looking statements that are based on current expectations and are subject to risks and uncertainties that could cause actual future results to differ materially from those expressed or implied by such statements. Those risks and uncertainties include, but are not limited to, risks related to the successful launch, continuation and results of the NIMH's Phase 2 (monotherapy) and/or the Company's planned Phase 2 (adjunctive therapy) clinical studies of AV-101 in MDD, and other CNS diseases and disorders, protection of its intellectual property, and the availability of substantial additional capital to support its operations, including the development activities described above. These and other risks and uncertainties are identified and described in more detail in VistaGen's filings with the Securities and Exchange Commission (SEC). These filings are available on the SEC's website at http://www.sec.gov. VistaGen undertakes no obligation to publicly update or revise any forward-looking statements.

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VistaGen Therapeutics Receives European Patent Office Notice of Intention to Grant European Patent for AV-101 - Yahoo Finance

Cardiac Stem Cells Comments Off on VistaGen Therapeutics Receives European Patent Office Notice of Intention to Grant European Patent for AV-101 – Yahoo Finance | March 31st, 2017

In Brief A Japanese man has become the first recipient of donated, reprogrammed stem cells as a treatment for macular degeneration. If the treatment proves effective against the age-related eye condition, it could halt or prevent the vision loss of the 10 million people in the U.S. who have macular degeneration. A New Treatment for Macular Degeneration

Macular degeneration is the leading cause of progressive vision loss with almost 10million Americans affected by the disease. Currently, there are no known cures for the conditionalthough stem cells might change that entirely.

Macular degeneration occurs when the central portion, the macula, of the retina is deteriorated. This is where our eyes record images and send them to the brain through the optic nerve. The macula is known for focusing our vision, controlling our ability to read, recognize faces, and see objects clearly.

A Japaneseman in his sixties is the worlds first person to receive induced pluripotent stem (iPS) cells donated by a different individual. Rather than tip-toeing around the ethics of embryonic stem cells, scientists were able to remove mature cells from a donor and reprogram them into an embryonic state, which then could be developed into a specific cell-type to treat the disease. Physicians cultivated donated skin cells that were transplanted onto the mans retina to halt the progression of his age-related macular degeneration.

While the mans first surgery was a success, the doctors have said they will make no more announcements about his progress until they have completed all five of the planned procedures. While the effectiveness of this technique cannot be evaluated until the fate of the donated cells and the progression of the patientsmacular degenerationhave been fully monitored, there is increasing interest inusing iPScells for theraputic purposes.

A similar therapy was performed at the Kobe City Medical Center General Hospital in Japan in September 2014, but with a slight difference. In this case, the patient received her own skin cells reprogrammed into retinal cells. As hoped, a year after the surgery her vision had no decline, seemingly halting the macular degeneration. Four more patients in the clinical trial are expected to receive donor cells as well.

The donor-cell procedure, if successful, could help pave the way for the iPS cell bank thatShinya Yamanaka is establishing. An iPS cell bank would allow physicians find theperfect iPS donor per each patients biological signatures. Yamanaka is a Nobel-prizewinning scientist at Kyoto University who pioneered the iPS cells.

Yamanakas idea of a iPS cell bank has the potential torevolutionize modern medicine. It would provide patients with ready-made cells immediately, givinga widespread population access to more treatment options bylower all-around costs. While the risk of genetic defects or a poor donor match still remains, the new procedurecould offer enormous advantagescompared toother alternatives.

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APPEAL: Delroy Anglin, star of TV's 'Cant' Pay? We'll Take it Away!' (photo credit: DCBL)

DELROY ANGLIN, a star bailiff of the TV series, 'Cant Pay? Well Take it Away!' has launched an appeal to encourage more African and Caribbean people living in the UK to join the Stem Cell Register.

The #Match4Delroy appeal which is to be led by blood cancer charity, the African Caribbean Leukaemia Trust (ACLT) is hoping to find an unmatched donor for Delroy who requires a lifesaving stem cell, specifically, a bone marrow transplant if he is to beat his battle with leukaemia.

Delroy, aged 56, was diagnosed with Acute Myeloid Leukaemia (AML) last November. Since his diagnosis, Delroy has managed with drugs and receiving two rounds of chemotherapy, but tests show the leukaemia remains. Doctors have confirmed Delroy will need an urgent stem cell transplant to beat the illness.

With siblings having a one in four chance of being a match, it was no surprise out of Delroys five siblings, none were found to be a match to help their brother.

The pain and anguish of dealing with a loved one being diagnosed with blood cancer is something Delroy and his family are all too familiar with, as it was 40 years ago, Delroys brother lost his battle against leukaemia which makes Delroys illness that much more painful for his loved ones to deal with.

Delroys sister Janet Hills, who is Chair at the Met Black Police Association (MBPS) and President at National Black Police Association, or NBPA, said:

When I tell people that Delroy from Cant Pay? Well Take it Away! is my brother, there is an immediate outpouring of warmth and love. I'm praying this appeal turns that love into action. FAMILY: Janet Hills of the Met Black Police Association and Delroy Anglin's sister (photo credit: David Sillitoe/The Guardian) ACLT is the preferred charity for the MBPS and the NBPA. Our members continually engage with the charity and organise community events to raise awareness and funds. If you love Del on the show as much as I love him as my brother, then please, please, please make that commitment today to join the stem cells (bone marrow) register.

Delroy is being supported by his loving family which includes his children and mother. His daughter Domenique Anglin said:

Dad is an active, charismatic person, he loves socialising with his family and friends. He is a fantastic father to my siblings and a wonderful grandfather too. I am appealing on his behalf to all Caribbean and African people in the UK and abroad to join the register, in the hope they might be the match that saves his life.

Anglin said:

Its going to be difficult to find me a perfect matched donor unless we have a lot more Caribbean and African people on the register. It takes 15 minutes to register and is almost painless to donate. I want to beat this illness, but I will only be able to do so, with the help of the Caribbean and African community.

I am requesting for more people of Caribbean and African heritage to join the register to help me and others like me.

Beverley De-Gale, ACLT co-founder said:

Delroys life has done a complete 360 in the last four months. From being on a popular documentary series to being diagnosed with a life-threatening illness. We hope fans of the show come together and help save the life of the individual they have come to love on-screen, in addition to Joe-public who dont watch the show.

If youre 16 55 and in good health, you could potentially be the person to save Delroys life.

You could be a #Match4Delroy. Join the Stem Cell Register now, by clicking here.

Read every story in our hardcopy newspaper for free by downloading the app.

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Lifesaving donor needed for TV debt collector Delroy - The Voice Online

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March 27, 2017 07:02 ET | Source: Cellect Biotechnology Ltd.

Cellects technology, ApoGraft, aims to become a game changerin stem cells transplantations for cancer treatments

Company gets green light from DSMB Board for enrolling additional 2 cancer patients for ApoGraft transplantation treatments

TEL AVIV, Israel, March 27, 2017 (GLOBE NEWSWIRE) -- Cellect Biotechnology Ltd. (Nasdaq:APOP) (TASE:APOP), a developer of stem cell selection technology, announced today that the first stem cell transplant procedure has been successfully performed using its ApoGraft technology in the Companys Phase I/II clinical trial in a blood cancer patient.

Up to 50 percent of stem cell transplant procedures, such as bone marrow transplants, result in life-threatening rejection disease, known as Graft-versus-Host-Disease (GvHD). Cellects ApoGraft technology is aiming to turn stem cell transplants into a simple, safe and cost effective process, reducing the associated severe side effects, such as rejection and many other risks.

Dr. Shai Yarkoni, Cellects CEO said, After 15 years of research, this is the first time we have used our technology on a cancer patient suffering from life-threatening conditions. It is a first good step on a road that we hope will lead to stem cell based regenerative medicine becoming a safe commodity treatment at every hospital in the world.

Based on the successful transplantation results, the independent Data and Safety Monitoring Board (DSMB) approved the enrollment of 2 additional patients for ApoGraft treatment to complete the first study cohort as planned.

About GvHD

Despite improved prophylactic regimens, acute GvHD disease still occurs in 25% to 50% of recipients of allogeneic stem cell transplantation. The incidence of GvHD in recipients of allogeneic stem cells transplantation is increasing due to the increased number of allogeneic transplantations survivors, older recipient age, use of alternative donor grafts and use of peripheral blood stem cells. GvHD accounts for 15% of deaths after allogeneic stem cell transplantation and is considered the leading cause of non-relapse mortality after allogeneic stem cell transplantation.

About ApoGraft01 study

The ApoGraft01 study (Clinicaltrails.gov identifier: NCT02828878), is an open label, staggered four-cohort, Phase I/II, safety and proof-of-concept study of ApoGraft process in the prevention of acute GvHD. The study, which will enroll 12 patients, aims to evaluate the safety, tolerability and efficacy of the ApoGraft process in patients suffering from hematological malignancies undergoing allogeneic stem cell transplantation from a matched related donor.

About Cellect Biotechnology Ltd.

Cellect Biotechnology is traded on both the NASDAQ and Tel Aviv Stock Exchange (NASDAQ:APOP)(NASDAQ:APOPW)(TASE:APOP). The Company has developed a breakthrough technology for the isolation of stem cells from any given tissue that aims to improve a variety of stem cell applications.

The Companys technology is expected to provide pharma companies, medical research centers and hospitals with the tools to rapidly isolate stem cells in quantity and quality that will allow stem cell related treatments and procedures. Cellects technology is applicable to a wide variety of stem cell related treatments in regenerative medicine and that current clinical trials are aimed at the cancer treatment of bone marrow transplantations.

Forward Looking Statements This press release contains forward-looking statements about the Companys expectations, beliefs and intentions. Forward-looking statements can be identified by the use of forward-looking words such as believe, expect, intend, plan, may, should, could, might, seek, target, will, project, forecast, continue or anticipate or their negatives or variations of these words or other comparable words or by the fact that these statements do not relate strictly to historical matters. For example, forward-looking statements are used in this press release when we discuss Cellects aim to make its ApoGraft technology a game changer in stem cell transplantations for cancer treatments and procedures, Cellects Apograft technology aiming to turn stem cell transplants into a simple, safe and cost effective process, reducing the associated severe side effects, such as rejection and many other risks, Cellects hope that stem cell based regenerative medicine will become a safe commodity treatment at every hospital in the world and that Cellects technology is expected to provide pharma companies, medical research centers and hospitals with the tools to rapidly isolate stem cells in quantity and quality that will allow stem cell related treatments and procedures. These forward-looking statements and their implications are based on the current expectations of the management of the Company only, and are subject to a number of factors and uncertainties that could cause actual results to differ materially from those described in the forward-looking statements. In addition, historical results or conclusions from procedures, scientific research and clinical studies do not guarantee that future results would suggest similar conclusions or that historical results referred to herein would be interpreted similarly in light of additional research or otherwise. The following factors, among others, could cause actual results to differ materially from those described in the forward-looking statements: changes in technology and market requirements; we may encounter delays or obstacles in launching and/or successfully completing our clinical trials; our products may not be approved by regulatory agencies, our technology may not be validated as we progress further and our methods may not be accepted by the scientific community; we may be unable to retain or attract key employees whose knowledge is essential to the development of our products; unforeseen scientific difficulties may develop with our process; our products may wind up being more expensive than we anticipate; results in the laboratory may not translate to equally good results in real clinical settings; results of preclinical studies may not correlate with the results of human clinical trials; our patents may not be sufficient; our products may harm recipients; changes in legislation; inability to timely develop and introduce new technologies, products and applications, which could cause the actual results or performance of the Company to differ materially from those contemplated in such forward-looking statements. Any forward-looking statement in this press release speaks only as of the date of this press release. The Company undertakes no obligation to publicly update or review any forward-looking statement, whether as a result of new information, future developments or otherwise, except as may be required by any applicable securities laws. More detailed information about the risks and uncertainties affecting the Company is contained under the heading Risk Factors in Cellect Biotechnology Ltd.'s Annual Report on Form 20-F for the fiscal year ended December 31, 2016 filed with the U.S. Securities and Exchange Commission, or SEC, which is available on the SEC's website, http://www.sec.gov and in the Companys period filings with the SEC and the Tel-Aviv Stock Exchange.

Related Articles

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Cellect Announces Successful First Cancer Patient Stem Cell Transplant - GlobeNewswire (press release)

Bone Marrow Stem Cells Comments Off on Cellect Announces Successful First Cancer Patient Stem Cell Transplant – GlobeNewswire (press release) | March 30th, 2017

KAMLOOPS My curiosity was sparked when I read that a stem cell centre was opening in Kamloops (Kamloops This Week, March 21, 2017).

So I went to the location of the centre at 470 Columbia St only to find a parking lot. Thinking that the address might be wrong, I searched the directory of the medical building next door and found that no stem cell centre was listed.

The Stem Cell Centers website lists Kamloops as the only one in Canada. Dr. Richard Brownlee is named as the surgeon with more information coming soon.

Stem cell therapy, says the website, can help with orthopedic or pain management, ophthalmological conditions, cardiac or pulmonary conditions, neurological conditions, and auto-immune diseases, among many other conditions and disease that results in damaged tissue.

One of the ophthalmological conditions they treat is macular degeneration. If your vision is fading due to macular degeneration, you know its time to seek help. Our non-invasive Stem Cell Therapy treatment might be the solution for you.

I wanted get Dr. Brownlees reaction to news that an unproven stem cell treatment had resulted in blindness according to the New England Journal of Medicine as reported in the Globe and Mail, March 20, 2017.

This week, the New England Journal of Medicine (NEJM) reported on three individuals who went blind after receiving an unproven stem cell treatment at a Florida clinic. The patients paid thousands of dollars for what they thought was a clinical trial on the use of stem cells to treat macular degeneration.

The writer of the Globe and Mail article, Timothy Caulfield, Research Chair of the in Health Law and Policy at the University of Alberta, doesnt name the Florida clinic.

The Stem Cell Centers website refers optimistically to treatment for macular degeneration at a Florida clinic, although apparently not theirs since no Florida clinic appears on their list. It tells of how Doug Oliver suffered from macular degeneration before stem cells were extracted from his hip bone and injected them into his eyes. Almost immediately, Olivers eyesight started to improve. I began weeping, he said.

Caulfield encourages caution. Health science gets a lot of attention in the popular press. People love hearing about breakthroughs, paradigm shifts and emerging cures. The problem is, these stories are almost always misleading. It can also help to legitimize the marketing of unproven therapies.

Reports from the Stem Cell Centers own website are cautionary as well. It quotes an abstract from a study done by the Southern California College of Optometry on how stem cells might ultimately be used to restore the entire visual pathway.

The promise of stem cell research is phenomenal. Scientific American (Jan., 2017) reports that brains can be grown in a lab dish from stem cells taken from skin. These samples can be used to research brain disorders ranging from schizophrenia to Alzheimer's disease, and to explore why only some babies develop brain-shrinking microcephaly after exposure to the Zika virus.

However, Dr. George Daley, dean of Harvard Medical School, concludes that there are only a handful of clinical applications available and they are for skin and blood-related ailments.

Practice, it seems, has not yet matched the promise of stem cell research.

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Stem cell centre coming to Kamloops? - CFJC Today Kamloops

Skin Stem Cells Comments Off on Stem cell centre coming to Kamloops? – CFJC Today Kamloops | March 30th, 2017

Stem cells are undifferentiated biological cells that can differentiate into specialized cells and can divide (through mitosis) to produce more stem cells. They are found in multicellular organisms. In mammals, there are two broad types of stem cells: embryonic stem cells, which are isolated from the inner cell mass of blastocysts, and adult stem cells, which are found in various tissues. In adult organisms, stem cells and progenitor cells act as a repair system for the body, replenishing adult tissues. In a developing embryo, stem cells can differentiate into all the specialized cellsectoderm, endoderm and mesoderm (see induced pluripotent stem cells)but also maintain the normal turnover of regenerative organs, such as blood, skin, or intestinal tissues.

There are three known accessible sources of autologous adult stem cells in humans:

Stem cells can also be taken from umbilical cord blood just after birth. Of all stem cell types, autologous harvesting involves the least risk. By definition, autologous cells are obtained from ones own body, just as one may bank his or her own blood for elective surgical procedures.

Adult stem cells are frequently used in medical therapies, for example in bone marrow transplantation. Stem cells can now be artificially grown and transformed (differentiated) into specialized cell types with characteristics consistent with cells of various tissues such as muscles or nerves. Embryonic cell lines and autologous embryonic stem cells generated through Somatic-cell nuclear transfer or dedifferentiation have also been proposed as promising candidates for future therapies.[1] Research into stem cells grew out of findings by Ernest A. McCulloch and James E. Till at the University of Toronto in the 1960s.[2][3]

The classical definition of a stem cell requires that it possess two properties:

Two mechanisms exist to ensure that a stem cell population is maintained:

Potency specifies the differentiation potential (the potential to differentiate into different cell types) of the stem cell.[4]

In practice, stem cells are identified by whether they can regenerate tissue. For example, the defining test for bone marrow or hematopoietic stem cells (HSCs) is the ability to transplant the cells and save an individual without HSCs. This demonstrates that the cells can produce new blood cells over a long term. It should also be possible to isolate stem cells from the transplanted individual, which can themselves be transplanted into another individual without HSCs, demonstrating that the stem cell was able to self-renew.

Properties of stem cells can be illustrated in vitro, using methods such as clonogenic assays, in which single cells are assessed for their ability to differentiate and self-renew.[7][8] Stem cells can also be isolated by their possession of a distinctive set of cell surface markers. However, in vitro culture conditions can alter the behavior of cells, making it unclear whether the cells will behave in a similar manner in vivo. There is considerable debate as to whether some proposed adult cell populations are truly stem cells.

Embryonic stem (ES) cells are stem cells derived from the inner cell mass of a blastocyst, an early-stage embryo.[9] Human embryos reach the blastocyst stage 45 days post fertilization, at which time they consist of 50150 cells. ES cells are pluripotent and give rise during development to all derivatives of the three primary germ layers: ectoderm, endoderm and mesoderm. In other words, they can develop into each of the more than 200 cell types of the adult body when given sufficient and necessary stimulation for a specific cell type. They do not contribute to the extra-embryonic membranes or the placenta.

Nearly all research to date has made use of mouse embryonic stem cells (mES) or human embryonic stem cells (hES). Both have the essential stem cell characteristics, yet they require very different environments in order to maintain an undifferentiated state. Mouse ES cells are grown on a layer of gelatin as an extracellular matrix (for support) and require the presence of leukemia inhibitory factor (LIF). Human ES cells are grown on a feeder layer of mouse embryonic fibroblasts (MEFs) and require the presence of basic fibroblast growth factor (bFGF or FGF-2).[10] Without optimal culture conditions or genetic manipulation,[11] embryonic stem cells will rapidly differentiate.

A human embryonic stem cell is also defined by the expression of several transcription factors and cell surface proteins. The transcription factors Oct-4, Nanog, and Sox2 form the core regulatory network that ensures the suppression of genes that lead to differentiation and the maintenance of pluripotency.[12] The cell surface antigens most commonly used to identify hES cells are the glycolipids stage specific embryonic antigen 3 and 4 and the keratan sulfate antigens Tra-1-60 and Tra-1-81. The molecular definition of a stem cell includes many more proteins and continues to be a topic of research.[13]

There are currently no approved treatments using embryonic stem cells. The first human trial was approved by the US Food and Drug Administration in January 2009.[14] However, the human trial was not initiated until October 13, 2010 in Atlanta for spinal injury victims. On November 14, 2011 the company conducting the trial announced that it will discontinue further development of its stem cell programs.[15] ES cells, being pluripotent cells, require specific signals for correct differentiationif injected directly into another body, ES cells will differentiate into many different types of cells, causing a teratoma. Differentiating ES cells into usable cells while avoiding transplant rejection are just a few of the hurdles that embryonic stem cell researchers still face.[16] Many nations currently have moratoria on either ES cell research or the production of new ES cell lines. Because of their combined abilities of unlimited expansion and pluripotency, embryonic stem cells remain a theoretically potential source for regenerative medicine and tissue replacement after injury or disease.

Human embryonic stem cell colony on mouse embryonic fibroblast feeder layer

The primitive stem cells located in the organs of fetuses are referred to as fetal stem cells.[17] There are two types of fetal stem cells:

Adult stem cells, also called somatic (from Greek , of the body) stem cells, are stem cells which maintain and repair the tissue in which they are found.[19] They can be found in children, as well as adults.[20]

Pluripotent adult stem cells are rare and generally small in number, but they can be found in umbilical cord blood and other tissues.[21] Bone marrow is a rich source of adult stem cells,[22] which have been used in treating several conditions including spinal cord injury,[23] liver cirrhosis,[24] chronic limb ischemia [25] and endstage heart failure.[26] The quantity of bone marrow stem cells declines with age and is greater in males than females during reproductive years.[27] Much adult stem cell research to date has aimed to characterize their potency and self-renewal capabilities.[28] DNA damage accumulates with age in both stem cells and the cells that comprise the stem cell environment. This accumulation is considered to be responsible, at least in part, for increasing stem cell dysfunction with aging (see DNA damage theory of aging).[29]

Most adult stem cells are lineage-restricted (multipotent) and are generally referred to by their tissue origin (mesenchymal stem cell, adipose-derived stem cell, endothelial stem cell, dental pulp stem cell, etc.).[30][31]

Adult stem cell treatments have been successfully used for many years to treat leukemia and related bone/blood cancers through bone marrow transplants.[32] Adult stem cells are also used in veterinary medicine to treat tendon and ligament injuries in horses.[33]

The use of adult stem cells in research and therapy is not as controversial as the use of embryonic stem cells, because the production of adult stem cells does not require the destruction of an embryo. Additionally, in instances where adult stem cells are obtained from the intended recipient (an autograft), the risk of rejection is essentially non-existent. Consequently, more US government funding is being provided for adult stem cell research.[34]

Multipotent stem cells are also found in amniotic fluid. These stem cells are very active, expand extensively without feeders and are not tumorigenic. Amniotic stem cells are multipotent and can differentiate in cells of adipogenic, osteogenic, myogenic, endothelial, hepatic and also neuronal lines.[35] Amniotic stem cells are a topic of active research.

Use of stem cells from amniotic fluid overcomes the ethical objections to using human embryos as a source of cells. Roman Catholic teaching forbids the use of embryonic stem cells in experimentation; accordingly, the Vatican newspaper Osservatore Romano called amniotic stem cells the future of medicine.[36]

It is possible to collect amniotic stem cells for donors or for autologuous use: the first US amniotic stem cells bank [37][38] was opened in 2009 in Medford, MA, by Biocell Center Corporation[39][40][41] and collaborates with various hospitals and universities all over the world.[42]

These are not adult stem cells, but rather adult cells (e.g. epithelial cells) reprogrammed to give rise to pluripotent capabilities. Using genetic reprogramming with protein transcription factors, pluripotent stem cells equivalent to embryonic stem cells have been derived from human adult skin tissue.[43][44][45]Shinya Yamanaka and his colleagues at Kyoto University used the transcription factors Oct3/4, Sox2, c-Myc, and Klf4[43] in their experiments on cells from human faces. Junying Yu, James Thomson, and their colleagues at the University of WisconsinMadison used a different set of factors, Oct4, Sox2, Nanog and Lin28,[43] and carried out their experiments using cells from human foreskin.

As a result of the success of these experiments, Ian Wilmut, who helped create the first cloned animal Dolly the Sheep, has announced that he will abandon somatic cell nuclear transfer as an avenue of research.[46]

Frozen blood samples can be used as a source of induced pluripotent stem cells, opening a new avenue for obtaining the valued cells.[47]

To ensure self-renewal, stem cells undergo two types of cell division (see Stem cell division and differentiation diagram). Symmetric division gives rise to two identical daughter cells both endowed with stem cell properties. Asymmetric division, on the other hand, produces only one stem cell and a progenitor cell with limited self-renewal potential. Progenitors can go through several rounds of cell division before terminally differentiating into a mature cell. It is possible that the molecular distinction between symmetric and asymmetric divisions lies in differential segregation of cell membrane proteins (such as receptors) between the daughter cells.[48]

An alternative theory is that stem cells remain undifferentiated due to environmental cues in their particular niche. Stem cells differentiate when they leave that niche or no longer receive those signals. Studies in Drosophila germarium have identified the signals decapentaplegic and adherens junctions that prevent germarium stem cells from differentiating.[49][50]

Diseases and conditions where stem cell treatment is being investigated include:

Stem cell therapy is the use of stem cells to treat or prevent a disease or condition. Bone marrow transplant is a crude form of stem cell therapy that has been used clinically for many years without controversy. No stem cell therapies other than bone marrow transplant are widely used.[64][65]

Research is underway to develop various sources for stem cells, and to apply stem cell treatments for neurodegenerative diseases and conditions, diabetes, heart disease, and other conditions.[66]

In more recent years, with the ability of scientists to isolate and culture embryonic stem cells, and with scientists growing ability to create stem cells using somatic cell nuclear transfer and techniques to created induced pluripotent stem cells, controversy has crept in, both related to abortion politics and to human cloning.

Stem cell treatments may require immunosuppression because of a requirement for radiation before the transplant to remove the patients previous cells, or because the patients immune system may target the stem cells. One approach to avoid the second possibility is to use stem cells from the same patient who is being treated.

Pluripotency in certain stem cells could also make it difficult to obtain a specific cell type. It is also difficult to obtain the exact cell type needed, because not all cells in a population differentiate uniformly. Undifferentiated cells can create tissues other than desired types.[67]

Some stem cells form tumors after transplantation; pluripotency is linked to tumor formation especially in embryonic stem cells, fetal proper stem cells, induced pluripotent stem cells. Fetal proper stem cells form tumors despite multipotency.[citation needed]

Hepatotoxicity and drug-induced liver injury account for a substantial number of failures of new drugs in development and market withdrawal, highlighting the need for screening assays such as stem cell-derived hepatocyte-like cells, that are capable of detecting toxicity early in the drug development process.[68]

Some of the fundamental patents covering human embryonic stem cells are owned by the Wisconsin Alumni Research Foundation (WARF) they are patents 5,843,780, 6,200,806, and 7,029,913 invented by James A. Thomson. WARF does not enforce these patents against academic scientists, but does enforce them against companies.[69]

In 2006, a request for the US Patent and Trademark Office (USPTO) to re-examine the three patents was filed by the Public Patent Foundation on behalf of its client, the non-profit patent-watchdog group Consumer Watchdog (formerly the Foundation for Taxpayer and Consumer Rights).[69] In the re-examination process, which involves several rounds of discussion between the USTPO and the parties, the USPTO initially agreed with Consumer Watchdog and rejected all the claims in all three patents,[70] however in response, WARF amended the claims of all three patents to make them more narrow, and in 2008 the USPTO found the amended claims in all three patents to be patentable. The decision on one of the patents (7,029,913) was appealable, while the decisions on the other two were not.[71][72] Consumer Watchdog appealed the granting of the 913 patent to the USTPOs Board of Patent Appeals and Interferences (BPAI) which granted the appeal, and in 2010 the BPAI decided that the amended claims of the 913 patent were not patentable.[73] However, WARF was able to re-open prosecution of the case and did so, amending the claims of the 913 patent again to make them more narrow, and in January 2013 the amended claims were allowed.[74]

In July 2013, Consumer Watchdog announced that it would appeal the decision to allow the claims of the 913 patent to the US Court of Appeals for the Federal Circuit (CAFC), the federal appeals court that hears patent cases.[75] At a hearing in December 2013, the CAFC raised the question of whether Consumer Watchdog had legal standing to appeal; the case could not proceed until that issue was resolved.[76]

Read more: Stem cell Wikipedia, the free encyclopedia

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Stem cell Wikipedia, the free encyclopedia IPS Cell ...

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