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Hyderabad team grows miniature eyes using stem cells The Hindu The iPS cells are produced by genetically manipulating human skin cells to produce embryonic-like stem cells that are capable of forming any cell types of the body. Small portions of the corneal tissue were separated from the miniature eyes and used ...

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Hyderabad team grows miniature eyes using stem cells - The Hindu

Skin Stem Cells Comments Off on Hyderabad team grows miniature eyes using stem cells – The Hindu | June 19th, 2017

17 Jun 2017

Animal models and engineered cells have contributed their fair share of insight into the pathogenesis of amyotrophic lateral sclerosis (ALS), but researchers do not often have the opportunity to peer into afflicted human neurons. In a study published May 30 in Cell Reports, researchers led by Rickie Patani and Sonia Gandhi at University College London offer a new model for human disease by using motoneurons and astrocytes derived from amyotrophic lateral sclerosis (ALS) patient stemcells.

The researchers generated spinal motoneurons and astrocytes from induced pluripotent stem cells (iPSCs) of ALS patients carrying mutations in the valosin-containing protein (VCP) gene. VCP mutations account for 2 percent of familial ALS cases and have been linked to other disorders as well, including hereditary inclusion body myopathy with Paget disease of bone and frontotemporal dementia (IBMPFD), jointly termed multisystemproteinopathy.

Previously, researchers reported that VCP mutations promoted the formation of cytoplasmic aggregates of TDP-43,one of the hallmarks of motoneuron disease, and that these aggregates were at least partially responsible for the VCP-induced neurodegeneration seen in a fly model (Neumann et al., 2007; Ritson et al., 2010).

Few studies have tested what VCP mutations do to human neurons, however. Using an iPSC-derived neuronal model, Virginia Kimonis at the University of California, Irvine, found that the VCP R155H mutation boosted cells levels of TDP-43 and several proteins involved in protein disposal (Dec et al., 2014).

In search of a more complete view of how VCPs effects unfold over time, first co-authors Claire Hall, Zhi Yao, Minee Choi, and Giulia Tyzach used iPSC-derived populations of spinal cord motoneurons and astrocytes to track several cellular functions. The cells originated from two patients (four clones) with VCP mutations and three healthycontrols.

TDP-43 (red) stays in the nucleus (blue) in control motoneurons (left) but leaks into the cytoplasm in three-day old VCP-mutant motoneurons (right). [Cell Reports, Hall et al.,2017.]

More VCP-mutant motoneurons than controls died; they also made fewer synapses with their neighbors, and had less intense and fewer coordinated bursts of firing than controls. Monitoring the whereabouts of TDP-43, the authors found no difference between mutant and normal cells when the cells were at an early stage of differentiation, as neural precursor cells. But by the third day after becoming motoneuron-like, the mutant cells were leaking TDP-43 from their nuclei into the cytoplasm (see image above). At the same time, they had increased levels of markers of endoplasmic reticulum (ER) stress, became less able than controls to survive an ER stress assay, and began producing reactive oxygen species (ROS) at a higherrate.

By day 17, the mutant motoneurons had sickly ER tubules. Some were swollen, others cozied up to mitochondria, both signs of continued ER stress. The neurons also had low mitochondrial membrane potential and low glutathione levels, indicating mitochondrial dysfunction and oxidative stress,respectively.

Putting these findings together, Patani and Gandhi suggested that cyotosolic TDP-43 generates ER stress, which next triggers increased tethering of the ER to mitochondria. This could depolarize mitochondria, which could impair mitochondrial function and ultimately lead to high levels of free radical production and oxidative stress.The findings support a role for TDP-43 wreaking havoc in the cytoplasm, saidPatani.

The mechanism that links TDP-43 and ER stress remains uncertain, said Kimonis. Patani and Gandhi acknowledge the link is probably complex, noting that, in addition to cyotplasmic TDP-43 aggregates inducing ER stress, ER stress itself seems to drive TDP-43 leakage (Walker et al., 2013).

The researchers then probed how mutant VCP might affect astrocytes. Studies of astrocytes in other ALS models have yielded varying results depending on the mutation. Superoxide dismutase 1 (SOD1) mutations, for example, can turn astrocytes into motoneuron killers (see Oct 2011 news),but it is unclear if they endanger the survival of the astrocytes themselves. Conversely, mutations in TDP-43 seem to sicken astrocytes without making them toxic to neighboring neurons (Serio et al., 2013).

On the left, healthy astrocytes (red) protect VCP-mutant motoneurons (yellow) from death (green). VCP-mutant astrocytes are much less protective (right). [Courtesy of ZhiYao.]

Interestingly, Patani and Gandhi found that VCP mutations had both cell-autonomous and non-cell-autonomous effects. They increased astrocytes risk of death and rendered them less able to support the survival of both control and VCP-mutant motoneurons (see image at right). The authors also found that the cell-autonomous effects on astrocytes differed from those in motoneurons. VCP-mutant astrocytes seemed more resistant when challenged with an ER stressor and had only a transient drop in mitochondrial membrane potential and a transient increase in ROS, with no change in glutathionelevels.

The study is a validation of key prior findings using iPS-derived neurons and glia from patients, which is a nice advance, wrote Paul Taylor at St. Jude Childrens Research Hospital in Memphis, Tennessee. The characterization of the role of glia is also interesting and potentially important.

Looking ahead, Patani and Gandhi want to generate isogenic controls, that is, cells derived from the same patients but with repaired VCP mutations. They also want to create iPSC-derived upper motoneurons, and to better understand muscle pathology, Kimonis hopes to extend her investigation of iPSC-derived muscle cells (Llewellyn et al., 2017).

Other model systems will be of value, too. iPSC-derived models are not equivalent to the decades-old cells inhabiting an adult nervous system. A study of dopaminergic neurons, for example, revealed that gene expression and DNA methylation patterns differed between iPSC-derived neurons and their in vivo counterparts (Roessler et al., 2014).

In the long term, Patani hopes that shedding light on the sequence of events that marks motoneuron degeneration in human cells will enable researchers to more effectively search for ALS therapies.MarinaChicurel

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Familial ALS Linked to Both Neuron and Astrocyte Pathology - Alzforum

Spinal Cord Stem Cells Comments Off on Familial ALS Linked to Both Neuron and Astrocyte Pathology – Alzforum | June 18th, 2017

islamabad - New research finds evidence of a link between use of certain hair products, such as dyes and relaxers, and raised risk of breast cancer in women.

In their study report, the researchers explain that there is conflicting evidence on whether use of hair products, some of which contain cancer-causing chemicals, or carcinogens, can raise the risk of breast cancer in women.

Some of the evidence comes from animal testing, and some of it comes from studies in defined human populations. However, research in human populations has tended to focus on hair dyes, with mixed results.

The researchers investigated links between raised risk of breast cancer and use of hair products, with particular focus on the use of hair dyes, use of products for relaxing or straightening hair, and use of creams containing cholesterol or placenta for deep conditioning of hair.

When they analysed the data, the researchers found some significant links between raised risk for breast cancer and use of hair dyes and chemical relaxers, or straighteners, and that the patterns of risk differed between white women and black women.

For example, for black women, they found that use of dark shades of hair dye was linked to an overall higher risk of breast cancer, and an even higher risk of estrogen positive breast cancer.

For white women, the analysis found that use of relaxers, or straighteners, either alone or together with hair dyes, was linked to raised risk of breast cancer.

Among white women, there was also a raised risk of estrogen positive breast cancer with use of dark hair dyes and raised risk of estrogen negative breast cancer with use of relaxers.

The authors conclude that these findings support the idea of a relationship between use of certain hair products and a raised risk of breast cancer. They suggest: Further examination of hair products as important exposures contributing to breast cancer carcinogenesis are necessary.

Meanwhile, a new study, however, finds that the treatment could be more harmful than helpful if cardiac stem cells are involved.

Researchers found that using patients own cardiac stem cells to repair damaged heart tissue may not only be ineffective, but that the stem cells may also develop inflammatory properties that cause further heart damage.

Prof. Leor and colleagues came to their findings by isolating stem cells derived from the cardiac tissue of mice that had left ventricular dysfunction caused by a heart attack.

The team then injected the stem cells back into the hearts of the mice and assessed how they affected heart remodeling and function, compared with a saline solution.

Instead of repairing the rodents damaged heart tissue, the researchers found that the transplanted stem cells developed inflammatory properties, which may increase heart damage.

We found that, contrary to popular belief, tissue stem cells derived from sick hearts do not contribute to heart healing after injury, explains Prof Leor.

Furthermore, we found that these cells are affected by the inflammatory environment and develop inflammatory properties. The affected stem cells may even exacerbate damage to the already diseased heart muscle.

An increasing number of end-stage heart failure patients are turning to stem cell therapy as a last resort, but the researchers believe that the treatment should be approached with caution.

[...] our findings suggest that stem cells, like any drug, can have adverse effects. We concluded that stem cells used in cardiac therapy should be drawn from healthy donors or be better genetically engineered for the patient.

While the findings may come as a blow for many heart failure patients, the study did uncover some information that could help to improve autologous stem cell therapy.

By studying stem cells derived from the heart tissue of mouse models and humans with heart disease, the team was able to identify the gene that causes the stem cells to develop inflammatory properties.

Furthermore, the researchers found that deleting this gene, called TLR4, can shift the stem cells back to a reparative state, a discovery that the team believes could be used to transform autologous stem cell therapy for patients with heart failure.

Our findings determine the potential negative effects of inflammation on stem cell function as theyre currently used, says Prof. Leor. The use of autologous stem cells from patients with heart disease should be modified.

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Hair dyes, relaxers tied to raised breast cancer risk - The Nation

Cardiac Stem Cells Comments Off on Hair dyes, relaxers tied to raised breast cancer risk – The Nation | June 18th, 2017

Each year, more than 700,000 people suffer myocardial infarction, aka a heart attack. Thanks to medical advances, there are myriad ways for a doctor to get the blood properly pumping and save a persons life. A cardiologist might give a patient medication to clear or loosen blockages. Or a doctor might insert a catheter to remove the clot, or place stents in the artery so it stays open.

These interventions have vastly improved survival rates, but they dont heal the damage caused by a cardiac event. The heart is really just one big muscle, and trauma to any muscle does some damage, which becomes scar tissue. Scar tissue on the heart means it functions far less optimally, which eventually leads to heart failure.

Short of a transplant, there isnt a long-term option to fix a damaged ticker. But a team of researchers say theyve come up with a high-tech solution that could revolutionize cardiology. Using 3-D printing technology, Brenda Ogle, an associate professor of biomedical engineering at the University of Minnesota-Twin Cities, has created a patch a doctor could apply to mend a patients broken heart.

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A false-color scanning electron micrograph (SEM) of a blood clot protruding from an arterial entrance in a heart chamber. This type of clot, known as coronary thrombosis, is the usual cause of myocardial infarction (heart attack). P. Motta/G. Macchiarelli/Sapienza University/Science Photo Libary/Getty

The concept is to imprint proteins that are native to the body, says Ogle. Weve used stem cellderived cardiac musclecardiac myocytesand actually mixed those with other cell types needed for blood vessels. This, she says, prevents what would otherwise happen naturally: The formation of a different type cells known as fibroblasts, which secrete scar tissue.

Ogle and her team of 3-D printing experts, clinical cardiologists and stem cell engineers have successfully tried the patch on mice. First, the team induced cardiac arrest in the rodents. When they then placed the cell patch on a mouse, researchers saw a significant increase in the functional capacity of the organ after just four weeks. We generated the continuous electric signal across the patch, and we can pace it: We can increase the frequency of beating up to three hertz, which is similar to a mouse heart, says Ogle who, this past January, published the findings of their experiment in Circulation Research, a journal from the American Heart Association.

The results of the experiment were so inspiring that in June 2016 the National Institutes of Health awarded her team a grant of more than $3 million, so they can now give pigs heart attacks and fix them with the patch. However, it will take some time to see their innovation in surgical departments, since using biological products such as cells requires a long regulatory process and, of course, quality assurance.

The replacement of muscle has been the holy grail for some time, says Ogle. Now we finally have the ability to take stem cells out of the body and develop the protocols to do that.

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How 3D Printing Can Help Mend a Broken Heart - Newsweek

Cardiac Stem Cells Comments Off on How 3D Printing Can Help Mend a Broken Heart – Newsweek | June 18th, 2017

Frances Wang, KXTV 8:33 PM. PDT June 17, 2017

Dawson Deschaine passed away on June 10th after a 2-and-a-half year battle with leukemia. He was only 8-years-old.

While most kids try to earn gold stars for their work, Dawson Deschainefelt a little pride every time he earned a bead.

"The Beads of Courage program is these beads that [represent] every poke, every hospital stay, every bone marrow biopsy, every chemotherapy," said Breanna Deschaine, Dawson's mother.

Dawson was diagnosed with leukemia in January 2015 at just 6-years-old. The battle would last two-and-a-half years. It was all Dawson ever knew.

"He mostly knew nurses, doctors, family," said Jason Deschaine, Dawson's father. "He had a lot more adult conversations than kid ones."

Breanna said he was an old soul. He even drank a cup of coffee every morning (decaf, of course).

"They accidentally sent him coffee [instead of hot chocolate] to his room one day," said Breanna.

Dawson's mom said he was an old soul. After getting coffee instead of hot chocolate, that's what he drank every morning (decaf, of course). pic.twitter.com/eYx7Ed57XX

Dawson battled leukemia for 2-and-a half years. Smiled through it all. Chemo, stem cell transplanted, bone marrow biopsies... pic.twitter.com/NN5NoFSFLI

Now you know an infectious smile like Dawson's comes with some pretty funny stories.

Last August, Nevada County made Dawson an honorary firefighter. They called it 'Dawson Day' with a ceremony and all.

Sweet Dawson passed away this Sat. after battling leukemia. He was only 8 years old. Last yr, he became an honorary Nevada Co. firefighter. pic.twitter.com/RkVOY0WGhD

Dawson was given a badge, his own turnouts, and boots. He even got to respond to an injured biker.

"He kept telling the EMTs how to wrap the leg!" said Breanna.

And his firefighter card, he never took for granted.

"He was getting ready to go to clinic one day," said Jason. "He comes back running in the house saying 'I need my ID Card!'...'No no, I need it. Just in case mom gets pulled over, [I can say police officer,] I am with the fire department.

It's reminiscing and laughing about stories like this that keep his family strong and smiling even when it hurts.

"People always ask how we're doing. As you can tell we smile. Dawson would never let us cry in the room," said Breanna.

Dawson's community made sure his last months in this world was full of adventure. Sadly, he got too sick for his Make-A-Wish: to go to Hawaii and swim with dolphins.

Dawson had plenty of adventures his last 6 months. Sadly, he never got to swim with dolphins in Hawaii... it was his dream . pic.twitter.com/UQeAqIwzW3

On June 10th at 6:05 AM, with his parents and his sister by his side, Dawson passed away.

Even up until his very last moment, he gave his family a thumbs up.

"He couldn't talk very much. Just the thumbs up that he was still good. He was Awesome Dawson," said Breanna, through tears. "It was his signature. No matter what...I told him 'It's OK Dawson. You fought the hardest battle and you won.'"

"You're not supposed to cry!" said Melody, Dawson's grandmother. "He's watching you."

And if Dawson was watching, what would they say?

"I would say thank you, for the opportunity...you pulled our community together and made our family so strong," said Melody. "We all love him very, very much. And miss him."

Breanna said she would read to him again from his favorite book 'Love You Forever.'

"His favorite saying from his favorite book: 'I love you forever. I like you for always. As long as I'm living, my baby you'll be," said Breanna.

Dawson's family hopes to continue his legacy by bringing the Beads of Courage program that got him through his darkest days into more hospitals.

Donations can be made on the Beads of Couragewebsite, under Dawson's name.

And until they see Dawson again, the family says they'll live by this motto: 'Don't cry because it's over. Smile because it happened.'

That is what Dawson would've wanted.

2017 KXTV-TV

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Awesome Dawson: The legacy an 8-year-old boy who battled leukemia leaves behind - ABC10

Bone Marrow Stem Cells Comments Off on Awesome Dawson: The legacy an 8-year-old boy who battled leukemia leaves behind – ABC10 | June 18th, 2017

Asian American Donor Program encourages minorities and mixed heritage to join national registry

Oakland Lisa Marie Evangelista, a 31-year-old Filipina woman who lives in Sacramento, is in a literal fight for her life. Lisa is a speech language pathologist and works at the U.C. Davis Medical Center.

On Dec. 27, 2016, she was diagnosed with Chronic Myelomonocytic Leukemia, a rare and aggressive blood cancer. She needs a bone marrow transplant to survive. Lisas sister is a 5/10 or half match. However, doctors prefer Lisa find a 10/10-donor match. To find a perfect match, Lisa needs a stranger to step forward and help save her life.

Lisa has partnered with the Asian American Donor Program to find a donor similar to her genetic makeup. A bone marrow transplant, which is needed soon, is Lisas only hope for her long-term survival. A committed 10/10 marrow-matching donor must be located to have a successful transplant. Since Lisa is of Filipino, a matching donor will also need to be of Filipino or Asian descent.

More about Lisa

Lisa learned about her diagnosis just nine months after her father died of a blood cancer. Family and friends note how she brings laughter, joy, warmth, and kindness to each day. Lisa is a speech-language pathologist and board certified specialist in swallowing and swallowing disorders. Her clinical interests include the evaluation and treatment of dysphagia resulting from radiation and chemotherapy treatments to the head and neck. She works directly with patients diagnosed with throat cancer. Lisa has lectured at the regional and national levels on pulmonary health and ethical considerations in dysphagia management. Lisa is described by her colleagues as a brilliant clinician and scientific thinker who is devoted to helping her patients. Lisas hobbies include traveling, hiking, and dancing.

Lisa grew up in Laguna Hills in Orange County, California. She attended Laguna Hills High School. From California State University, FresnoLisa received abachelors degree in 2007 and a masters degree in 2009. Lisa received her clinical science doctorate in medical speech-language pathology from the University of Pittsburgh, Pittsburgh, PA in 2014.

Whats the solution?

Minorities are more likely to die of leukemia and other blood cancers because there is a shortage of ethnic and mixed-ethnic donors on the Be The Match national registry. It is vital to expand and build a more diverse registry so everyone has an equal opportunity to survive blood cancers.

Encouraging more people of ethnically diverse backgrounds and those of mixed heritage to be committed and join the Registry, potentially saving a life. Each of us can Be TheOne to Save a Life!

The Asian American Donor Program (AADP,www.aadp.org) is a 27-year-old nonprofit organization, based in Alameda, CA, that works to educate community members about the shortage of ethnic marrow donors and the importance of joining the Be The Match national registry. It is the oldest nonprofit of its kind in the country. AADP staffis dedicated to increasing the availability of potential stem cell donors for patients with life threatening diseases curable by a blood stem cell or marrow transplant.AADP is an official recruitment center for Be The Match.

There is a shortage of committed non-Caucasians on the Be The Match national registry, says Carol Gillespie, the AADP executive director. We need everyone of mixed race ancestry to step forward and join the Registry.When a marrow match is not readily available, patients have to wait longer than is ideal to find a match.Once a match has been found, their disease may have progressed to the point that they are no longer eligible for a transplant.

Shortage of ethnic/multi-ethnic donors

Approximately every three minutes one person in the United States is diagnosed with a blood cancer. An estimated combined total of 172,910 people in the US are expected to be diagnosed with leukemia, lymphoma or myeloma in 2017. New cases of leukemia, lymphoma and myeloma are expected to account for 10.2 percent of the estimated 1,688,780 new cancer cases diagnosed in the U.S. in 2017. (From:http://www.lls.org/http%3A/llsorg.prod.acquia-sites.com/facts-and-statistics/facts-and-statistics-overview/facts-and-statistics)

Of the approximately 816,000 Asians on the Be the Match registry, .5 percent are Filipinos, while Filipino Americans constitute 19.7 percent of Asian Americans (Source: 2010 Census). The Be The Match registry recruits hundreds of thousands of donors each year through an extensive network of more than 155 local and regional Community Engagement Representatives and organizations. You only need to join the Be The Match registry once.

Finding a marrow/stem cell match can be like finding a needle in a haystack, says Gillespie. Multi-racial patients face the worst odds. Those diagnosed with a blood disease need a marrow/stem cell transplant as soon as possible. Building the Registry with committed donors is what patients need. You could potentially match anyone in the world, this is truly a global effort.

Marrow/stem cell matches are very different than blood type matches. Just as we inherit our eyes, hair, and skin color, we inherit our marrow and stem cell tissue type.

For thousands of severely ill blood cancer patients, there is a cure, Gillespie says. You could be the cure. Those whose marrow/stem cells are not a match for a patient in need now may be a match for someone else down the road, anywhere in the world. I encourage multi-ethnic individuals to commit to registering. It is simple to register just a swab of the inside of your cheek.

How you can commit to help

Find a registration drive in your area. Go tohttp://www.aadp.org/drive/.

Register on line here:https://join.bethematch.org/lisa.

You must be 18 to 44 years old and meet general health requirements

Fill out a consent form and do a cheek swab.

Be committed. Be ready to donate to any patient in need.

Contact friends/family and encourage them to go to a registration drive or register online.

Set up a drive in your area or for more information, call AADP at 1-800-593-6667 or visit our websitehttp://www.aadp.org.

Volunteer to help at registration drives.

Please take a few minutes of your time to learn more about how you can help save a life and register as a marrow donor.

Upcoming registration drive

Soy and Tofu Festival, Saturday, June 17 from 11 a.m. to 5 p.m. Open to the public at Saint Marys Cathedral,1111 Gough St., San Francisco, CA 94109.

Malayan SF Outdoor Festival, Philippine Independence Day, Sunday, June 18 from noon to 8 p.m. at Union Square, 333 Post St., San Francisco, 94102.

More about the Asian American Donor Program (AADP)

The Asian American Donor Program (AADP), with its offices in the San Francisco Bay Area,is dedicated to increasing the availability of potential stem cell donors for patients with life threatening diseases curable by a blood stem cell or marrow transplant.

AADP is a community-based nonprofit for social benefit (5013) organization and specializes in conducting outreach and donor registration drives in and with diverse communities. AADP is an official recruitment center of the Be TheMatchregistry.

To learn more about scheduled upcoming marrow drives, visithttp://www.aadp.org/drive/.

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Filipina urgently needs bone marrow donor - Asianjournal.com

Bone Marrow Stem Cells Comments Off on Filipina urgently needs bone marrow donor – Asianjournal.com | June 17th, 2017

MUMBAI: Three-year-old Kinaya Shah was diagnosed with thalassemia at the tender age of three months and has been undergoing regular blood transfusions ever since. The only cure for thalassemia is a bone marrow transplant (BMT), a form of stem cell therapy. Typically, the donor of the stem cells would be a sibling of the patient such that the stem cells of the donor are a near perfect match to those of the patient. The only complication was that Kinaya was a lone child.

So, city doctors in a first used stem cells donated by Kinaya's father - who was only a half or haploidentical match - to cure the child of the blood disorder. "We went to Vellore, Bangalore and Pune but no one was willing to do the transplant without a full match donor," said Kinaya's parents, Aneri and Shripal Shah. They approached Dr Santanu Sen at the Kokilaben Dhirubhai Ambani Hospital, Andheri, in October of 2016, after reading about a similar surgery that he had performed.

While haploidentical bone marrow transplants are carried out to cure leukaemia, it has only been done about half a dozen times for thalassemia in a couple of Indian cities. ``Haploidentical transplants are gradually increasing because of better techniques,'' said Dr Sen.

Dr Sen has completed 36 BMTs in the last two years, of which 12 were haploidentical donors. ``But this is the first time that a haploidentical transplant has been done in western India to cure thalassemia,'' he said.

Chennai-based haematologist Dr Revathy Raja said that there is a 85% chance of cure in thalassemia with a fully matched donor. ``The success rate falls to 70% with a half-match or haploidentical donor. We have hence not started it at our Chennai centre. Hopefully, techniques will further improve in the coming years,'' she said.

In order to perform the surgery, Dr Sen conditioned Kinaya's immune system over three months, with slight chemotherapy, to increase the chances of her body accepting the graft. "We found that her father's stem cells were a 70% match through genetic tests and decided to use them for the transplant. In the case that the graft was rejected we froze a couple of Kinaya's stem cells as insurance. The positive is that children have lower rejection rates for foreign cells as they have barely developed any active immunity," said Dr Sen. "BMT is the most viable treatment to cure thalassemia, the only barrier thus far was the necessity of a full match donor," he added.

However, Vinay Shetty of NGO Think Foundation, which works for thalassemia patients, said that it would be prudent to wait for a statistically significant number of successful halploidentical transplants before recommending it to all patients.

Post the three months of conditioning, stem cells were collected from her father's bone marrow and the transplant was performed on May 10, 2017. After several tests to confirm that the graft was accepted, Kinaya was finally discharged from the hospital on June 13.

"The future of thalassemia treatment probably lies in gene therapy, but at the moment, haploidentical transplants have made BMT much more accessible," said Dr Sen, adding that he has two more cases such as Kinaya lined up. Kinaya is expected to be completely independent of medication and any trace of thalassemia in the coming six months.

What is Thalassemia?

Thalassemia is a genetic blood disorder when the body produces abnormal hemoglobin. Patients require regular blood transplant and well as dietary control to ensure that blood irons level stay suppressed.

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In a first, Mumbai doctors use dad's cells to fight blood disorder - Times of India

Bone Marrow Stem Cells Comments Off on In a first, Mumbai doctors use dad’s cells to fight blood disorder – Times of India | June 17th, 2017

Andrew LeClerc knew something was wrong when he heard voices when no one else was around. Some were those of people he knew, others were unfamiliar, but all had the authentic mannerisms of real people, not his imagination. He was in his early twenties, unsure of his direction in life, and had been taking synthetic marijuana to ease stress from past traumas. Disturbed by the voices, he sought help in an emergency room and voluntarily admitted himself to a psychiatric hospital, not realizing he would be kept there for six days. He was diagnosed with psychosis, but had little interaction with a therapist. You mostly sit around with coloring books, he says. It felt like a punishment, when all he wanted was help.

Afterward, he contacted therapists, but many were booked. An online search led him to a research study at Beth Israel Deaconess Medical Center in Boston for people newly diagnosed with psychotic disorders. In January 2014, he entered a two-year study that compared two approaches to psychotherapy to help manage cognitive impairments and other symptoms. He was also prescribed an antipsychotic medication.

Eventually he was diagnosed with schizophrenia. Now, about four years later, at 26, LeClerc is learning to live with the condition. Its hard for a person whos diagnosed with schizophrenia to be told somethings not real when they think its real, he says. He continues to take antipsychotic medications that help control his hallucinations and lives in an apartment below his parents in Middleton, Massachusetts. Hes hoping to start a small business, putting his love of gardening to work as a landscaper.

But more importantly, hes learned to make peace with his mind. He likes to say: I dont hear voices, I hear my own brain. When voices do appear, he recognizes them as a product of an aberrant auditory cortex, and he thinks about engaging his prefrontal cortexthe decision-making part of the brainto help him distinguish fact from fiction. I have tools to pull myself back to the moment, he says.

Not everyone who struggles with schizophrenia is able to find such stability. The illness takes many forms; symptoms may include hallucinations and delusions, lack of motivation, and cognitive problems similar to dementia. It tends to strike in the late teens and early twenties, robbing young people of their mental stability just as theyre entering adulthood, beginning careers, or pursuing a college degree. Some improve, while others experience a long mental decline.

The treatments that we have are useful but not great, says Matcheri Keshavan, Cobb professor of psychiatry at Harvard Medical School (HMS) and the leader of the study that LeClerc participated in. The medications used to treat schizophrenia are decades old, and only ameliorate symptoms. Like other psychiatric illnesses, schizophrenia has suffered from a lack of investment from pharmaceutical companies. Says Keshavan, We need better medications that really address the underlying cause of this illness.

But those causes are still mysterious. What scientists do know is that schizophrenia tends to run in families. About 70 percent to 80 percent of a persons risk of developing the illness, Keshavan says, can be explained by genetic factors. Recently, theres been a surge of effort to capitalize on that fact. Advances in genetics have made it possible to search not only for clues about schizophrenia and other psychiatric illnesses hidden within thousands of human genomesbut also for potential new treatments.

As a result, theres been a renaissance in research on schizophrenia and other psychiatric disorders, and some cautious optimism. Its been possible to make real if still early progress in understanding what genes and molecules influence these illnesses, says Steven McCarroll, Flier associate professor of biomedical science and genetics. At Harvard, the leading force is the Stanley Center for Psychiatric Disease Research at the Broad Institute, which is pouring new funding and resources into amassing data on the genetics of mental illness.

Bringing the power of genomics to psychiatric disease fulfills a long-held goal for the Stanley Centers director, Steven Hyman, professor of stem cell and regenerative biology. An HMS professor of psychiatry before becoming head of the National Institute of Mental Health (NIMH) in 1996, Hyman was frustrated by the sluggish progress on the science of psychiatric disorders, as research on illnesses like cancer, heart disease, and diabetes marched ahead. Schizophrenia in particular is challenging to study because its uniquely human. Scientists can study limited aspects of psychiatric illness in animals if they can measure an observable behavior, such as avoiding social interactions or grooming excessively. But psychosis is a problem of thinking; animals, as far as is known, dont experience it in any way we can measure. Its also challenging because brain tissue is so inaccessible. We were really hampered, Hyman says, and I, frankly, didnt know all that much more when I was at NIMH in the late 1990s than careful observers knew at the turn of the twentieth century.

Steven Hyman, director of the Stanley Center for Psychiatric Disease Research Photograph by Stu Rosner

When he left the position, Hyman was interested in researching psychiatric disease but didnt see a rigorous path to do so; instead, he accepted a position as Harvards provosttaking what he now refers to as a 10-year timeout. During that time, a revolution occurred. Genetic technologies and vastly expanded computer power opened new paths for studying the biological basis of complex diseases.

The Broad Institute launched the Stanley Center in 2007 under inaugural director Edward Scolnick, thanks to an initial $100 million in private funding from philanthropists Ted and Vada Stanley, aiming to bring much-needed innovation to treatments for psychiatric disease by harnessing the power of genomics. (The Stanleys provided another $650 million in 2014, an unprecedented gift for psychiatric research.) Partly as a result, the center has gathered the worlds largest collection of DNA samples for studying not only psychiatric diseasesincluding schizophrenia, autism, ADHD, and bipolar disorderbut also healthy control subjects. The resulting data are freely available to the public.

The human genome has started to give us a really powerful way into the problem, Steven McCarroll explains, because the key source of scientific leverage that we have is we know that schizophrenia and other psychiatric illnesses are heritablethey aggregate in families. Their molecular secrets are almost certainly hidden in the way our genomes vary from person to person.

Much of the research on genetics and disease has focused on what McCarroll calls genetic sledgehammersgenes that when mutated would almost certainly make you sick. But schizophrenia, like most common diseases, is genetically complex. The hereditary component of the disease may be a product of tens to hundreds of genetic nudges, variations that dont cause disease by themselves, but together make people vulnerable to illness.

Studying genetic nudges requires amassing large numbers of DNA samples to achieve the statistical power to find subtle variations that may contribute to disease, a project thats taken enormous collaborative effort by many scientists and institutions around the world. The Psychiatric Genomics Consortiumthe largest scientific collaboration involving psychiatric diseaseformed in 2007 and comprises hundreds of investigators in 38 countries and nearly a million genetic samples. The Stanley Center has served as the hub for data sharing, aggregation, and analysis to further the consortiums discoveries.

One of the key tools for uncovering the genetic basis of disease is the genome-wide association study (GWAS)a way of quickly sorting through the common variations in genomes to find those that are more common in people with a given trait or disease than in those without. Associate professor of medicine Mark Daly, who leads the analytic hub of the consortium, says that scientists originally thought such studies might uncover a handful or two of DNA variants that could be statistically correlated with schizophrenia. But rather than identifying a few standouts, the consortiums Schizophrenia Working Group found a crowd of genetic associations, each contributing just a tiny amount of risk. A landmark paper published in 2014 in the journal Nature, led by Michael ODonovan of Cardiff University, described 108 different locations in the genome that harbored variants associated with schizophrenia.

GWAS studies can identify only stretches of DNA: like flags on a zoomed-out map of a city, they provide a neighborhood, not the exact address. We know where the variants are, one of which is likely to be the causal variant, but cant say for sure which one, says assistant professor of medicine Ben Neale, who is developing methods to analyze genomic data. Another approach is to sift through genomes in finer-grained detail by directly reading each letter of the DNA sequence. Such work is time-consuming, but it can help uncover rare genetic differences that are linked to disease, many of which have a stronger effect than common variants. Work by the consortium has also analyzed areas of DNA that are deleted or duplicated, called copy number variations. People with schizophrenia tend to have more such variations overall, and the genes they affect can provide clues to the diseases origins.

Meanwhile, the Stanley Center and other institutions are working to collect thousands more DNA samples from people with schizophrenia and other psychiatric disorders, hoping to identify even more genetic associations of risk. Hyman doesnt see such data-gathering as an endless project. We should kill this problem, he says, meaning in some reasonable number of yearsseven to 10we should have proceeded so far in the genetics of schizophrenia, bipolar disorders, autism, perhaps some other disorders, that weve reached diminishing returns in terms of biological information.

But so far, the picture is still incomplete. The vast majority of genetic samples, for instance, come from people of European ancestry. From a purely scientific point of view, it means were missing a large proportion of the worlds genetic diversity, says Karesten Koenen, professor of psychiatric epidemiology at the Harvard T.H. Chan School of Public Health. Most of that diversity is in Africa: There is much more diversity in African genomes than in those of people from other parts of the world.

Koenen is leading an effort through the Stanley Center to launch genetic research on psychiatric disease in Ethiopia, Uganda, Kenya, and South Africa. Their researchers are partnering with researchers and academic and clinical institutions in those countries and will be gathering DNA samples and clinical information from people diagnosed with schizophrenia. We really want to build local capacity, she says, and develop sustainable research programs that can be led by local scientists and clinicians. The center also plans to extend the effort to Latin America, beginning in Mexico.

This effort will help fill in the genetic picture of psychiatric illness, and will also help correct a vast imbalance. Geneticists are beginning to use data to classify patients based on their risk of developing complex diseases, including schizophrenia. But these risk profiles, Koenen says, lose accuracy when applied to people of African descent. As this kind of profiling makes its way into medicine, she says, Theres a risk that if we dont extend this research to Africa, the health disparity and treatment gap will widen.

What will all this data amount to? Theres a misconception, McCarroll says, that the goal of this research is to conjure up a crystal ball genetic test that will give people personalized treatments based on their unique portfolio of genes. Thats not the aim. Our goal, he says, is to understand the core biological processes in the illnesses, so that innovative treatments can be developed that can treat anyone. Scientists hope that the dizzying array of schizophrenia-related genes will converge onto a few basic processes in the brain, once the function of those genes is understood.

But even as scientists have made dramatic leaps in discovering genetic risk factors of complex diseases, the task of understanding how those genes work is a different, and slower, task.

Researchers from Harvard and the Broad Institute have grown human brain organoids, three-dimensional organ models cultured from stem cells, to study the genetics of psychiatric illness. This series shows (clockwise from upper left) growth at 1, 3, 6, and 9 months, with development of synapses indicated in green. Quadrato et al./Nature 2017

McCarroll was lead author of a study making one of the strongest links between a specific genetic variant and its role in schizophrenia. Working with Aswin Sekar (then a graduate student, now a research fellow), he focused on the most powerful signal of risk in GWAS studies to date, a stretch of DNA in chromosome 6 that was known to harbor many genes involved in the immune system. They focused on one called C4, which has a high degree of variability in humans: each of its different forms may be present in multiple copies in one individual. By using both genetic data and postmortem brain tissue, they found that people with schizophrenia are more likely to have variants of the C4 gene that lead to higher levels of one gene product, C4A, in brain cells.

C4A is one of several proteins involved in a type of immunity called the complement pathway, which helps clear damaged cells and harmful microbes from the body. As part of their study, McCarroll and Sekar collaborated with associate professor of neurology Beth Stevens, whose previous research with mice clarified an ingenious connection between the complement pathway and the brain. Scientists know that as the brain develops, it churns out new cells, which form billions of connections called synapses. In adolescence and early adulthood, some of these connections are pared back, a process called synaptic pruning. In mice, Stevens has found, this pruning is mediated by the complement pathway, which triggers immune cells called microglia to attack neural connections: literally nibbling at synapses.

Sekar, McCarroll, and Stevens also worked with professor of pediatrics Michael Carroll, who had developed mice with varying copies of the C4 gene, and showed that too much C4 activity in the animals can lead to excess pruning. Its too much of a good thing, Stevens says. Their finding suggests that schizophrenia, in some cases, may be caused by loss of synapses in adolescencean especially promising result because it supports clinical observations: synaptic pruning coincides with the age when schizophrenia typically emerges, and brain imaging shows that many people with schizophrenia experience a thinning of the prefrontal cortex in the early stages of disease.

Steven McCarroll, Flier associate professor of biomedical science and genetics Photograph by Stu Rosner

McCarroll emphasizes that the C4A variation contributes only a small amount of risk of disease, but may collude with other variants to tip the brain past a threshold. There are a lot of genetic findings that map to synapses, says Hyman, so some of those other variants may contribute to a larger disruption in how synapses are formed and maintained. But other processes are likely at work in schizophrenia as well. Some genetic risk variants relate to a chemical signal in the brain called glutamate, and others to ion channels, proteins that determine how electrical signals propagate in brain cells. There are also others, Hyman adds, that, frankly, just have us scratching our heads.

The work on C4 offers an example of how genetics is beginning to help neuroscience move forward. Its opened up a ton of new directions and strategies for our group, says Stevens. Across Harvard and the Stanley Center, a growing community is launching collaborative projects with the goal of taking psychiatric disease research into new territories.

One priority is developing new models for teasing out the role of genes in the brain. Scientists have been able to study some behaviors that relate to mental illness in animals, but there is no animal model for schizophrenia. Michael Carroll is now working to extend the C4 study by creating humanized mice that carry human C4 genes, which may make it possible to study their function in a living brain.

Other researchers are trying to develop new ways to study psychiatric disease in humans. Paola Arlotta, professor of stem cell and regenerative biology, explains that when scientists are able to get samples of human brain tissuefrom patients undergoing surgery, postmortem donations, or even tissue from fetusesthe cells die quickly. They cant be propagated and studied in a laboratory, so there is no renewable source of the actual endogenous tissue.

Stem cells have emerged as a way around that problem. Scientists can now take cells from the skin or hair and transform them into induced pluripotent stem (iPS) cells that are capable of becoming other cell types, including brain cells. (At the Stanley Center, Arlotta and other scientists are exploring how to transform iPS cells into specific types of brain cells.) The iPS cells allow scientists to study how cells derived from a person of one genetic background differ from those of another person. Scientists can also use the genome-editing tool CRISPR-Cas9 to introduce specific genetic changes and study their effects.

But theres very little that can be learned about psychiatric disease from isolated cells: brain activity depends on the constant chatter of many cells that are intricately connected. Arlotta has been investigating whether neural stem cells can be spun into something that behaves more like human brain tissue. So-called organoidsclusters of millions of cells up to a few millimeters in diametercan be formed from growing stem cells in a nutrient-rich solution. Organoids have already been used to study events that happen in early development: last year, a team of researchers used them to study the effects of the Zika virus on developing brains.

But since psychiatric diseases like schizophrenia emerge later in life, Arlotta wants to make organoids grow larger and live longer, and to understand whether they can mimic some of the properties of an older brain. This is a new tissue were making, she says, and so the questions that we want to answer are: can we develop them for a very long time, can we understand the cellular composition, can we see if these organoids make actual networks and communicate with each other?

To better characterize these cell-based models, Arlotta and her colleague Kevin Eggan, a fellow professor of stem cell and regenerative biology, are collaborating with McCarroll to apply a technology his lab developedDropSeqthat makes it possible to analyze gene activity in individual cells. The technology will provide a detailed, cell-by-cell understanding of what these models may reveal. In a Nature paper published in April, Arlottas team demonstrated that its possible to cultivate human brain organoids for nine months or more. Analysis revealed that the organoids are filled with a diverse mix of brain-cell types, and that these cells actually form interconnected networks, suggesting they may begin to function in ways that brains do.

But how much meaningful information about psychiatric disorders can be gleaned by studying individual cells or clusters of artificial tissue remains unclear. And an even bigger question is how to use these models to study the effects of genetic nudges. Disease genetics, typically, has been studied by altering or removing genes, one at a time, in an animal. Studying a whole suite of subtle genetic variations in a model system is a completely new idea.

There is no playbook, says Hyman. He acknowledges that the work is risky; many of these projects are possible only because the Stanley Centers open-ended funding makes it easier for labs to work together to pursue new ideas. We spend many tens of millions of dollars a year, and were accountable only at the end of the year to our scientific advisory board, and we tell them our strategy, he says. It gives us enormous flexibility, but its an enormous responsibility.

Some scientists and clinicians believe that gathering genetic data and studying cells is a misguided strategy for alleviating psychiatric illness. They see it as reductionist, and argue that it emphasizes the inborn biological origins of illnesses rather than other factorslike abuse, trauma, drug use, and emotional stressthat are known to play a role in their development. Hyman answers, Genes are not fate, but genes have an awful lot to say. Genetics and the environment both undoubtedly contribute to disease, but both ultimately must act on the brainand genetics happens to be a more tractable way to study whats happening in the brain.

Genetics is already providing insights that could help alter the way psychiatric disorders are defined. People have studied disorders with a box around them, says Elise Robinson, assistant professor of epidemiology, who has analyzed genetic differences within and between disorders such as schizophrenia, bipolar disorder, depression, and autism, which are usually defined by clinical categories outlined in the Diagnostic and Statistical Manual of Mental Disorders. But Robinson says the idea of distinct boundaries separating these disorders is not necessarily consistent with biology. Genetically, psychiatric disorders look more like Venn diagrams with large overlaps. People with schizophrenia share 60 percent to 70 percent of genome-wide variation with those who suffer bipolar disorder, and about 25 percent with autism.

Similarly, there is no simple dividing line between people who have a psychiatric illness and those who dont. Genetic risk for schizophrenia is not something you either have or dont have, she says. Theres a little bit of risk in all of us. Natural variations in many different genes, she explains, have been shown to relate to the way people perform on tests of cognitive or emotional skills. Schizophrenia may emerge from some combination of factors that are part of normal variation in the development and functioning of the human brain. This is true for other complex diseases and many normal traits, she adds: height, for instance, is largely determined by genetics, but theres no single geneor even handful of genesthat controls it. Its a quality that emerges from many genetic inputs.

Robinson believes that scientists could learn more about these disorders by cutting across diagnostic boxes and studying genetic variants that are linked to multiple traits and disorders. Only by understanding how these variants affect the brain can researchers begin to understand how they contribute both to normal brain function and to the risk of disease.

Such research could help demystify the experiences of people like Andrew LeClerc. He has learned to talk about his schizophrenia as something he struggles with, not something that defines him. He describes his condition as a mental difference.

LeClerc also appreciates that not all the voices in his head are negative: he sometimes hears words of encouragement or helpful warnings. As he speaks, his thoughts dont always follow the linear paths of normal conversation, but they can take him into deeper places; he has a keen understanding of how humans brains create their own realities. He sees an analogy to his condition in the once-expensive glass pieces he has begun collecting from his local dump. Glass that seems like trash, he says, can be reused or recycled, so it isnt really broken. He describes himself the same way: Im fragile, not broken.

It may take decades before genetic research on schizophrenia yields new treatments for people like LeClerc, but clues about the biological underpinnings of schizophrenia could help in other ways. Patients with psychiatric disorders get blamed for those disorders in our culture in a way that people with diseases in other organs dont, says McCarroll. If this research can provide a firmer biological understanding of whats happening in the brain, he says, I would hope that we could generate more empathy.

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IPS Cell Therapy Comments Off on Probing Psychoses – Harvard Magazine | June 17th, 2017

Rep. Jim Banks, R-3rd, introduced legislation Thursday that would prevent the use of federal funds for stem cell research involving human embryos.

Banks' bill would direct the U.S. Department of Health and Human Services to give priority to medical research with the greatest potential for near-term clinical benefit in human patients and that does not use stem cells from destroyed, discarded or created embryos.

Scientists say embryonic stem cells show potential for transforming into other cells that might repair tissue damaged by disease or injury. Human embryonic stem cells used in research come from donated, unused fertilized eggs developed for in vitro fertilization procedures.

Adult blood stem cells are used to treat leukemia, and adult neural stem cells have been tested for brain disorders and spinal cord injuries.

This bipartisan bill prioritizes stem cell research that has a real impact on patients suffering right now while ensuring that research is conducted ethically without destroying human embryos, Banks, a freshman lawmaker from Columbia City, said in a statement.

Rep. Dan Lipinski, D-Ill., co-sponsored Banks' bill, which is called the Patients First Act of 2017.

The Dickey-Wicker Amendment of 1996 prohibited HHS from funding research using created or destroyed human embryos. But a federal court ruled in 2011 that Dickey-Wicker was ambiguous and did not ban research using stem cells from in vitro fertilization.

The Alliance for Regenerative Medicine, a coalition of medical companies, research institutions and patient advocacy groups that support embryonic stem cell research, had little to say Thursday about Banks' legislation.

As an organization representing the broader global regenerative medicine sector, our position is that we are in favor of government funds supporting the best science in an effort to speed safe and efficacious products to patients in need, Lyndsey Scull, senior communications director for ARM, said in an email.

Scull said ARM would monitor Banks' bill in the legislative process.

Banks' proposal states it would promote the derivation of pluripotent stem cell lines without the creation of human embryos for research purposes and without the destruction or discarding of, or risk of injury to, a human embryo.

The National Institutes of Health defines pluripotent stem cells as those that can give rise to any type of cell in the body except those needed to support and develop a fetus in the womb. They come from embryos and fetal tissue, although induced pluripotent stem cells are genetically reprogrammed cells taken from adult tissues.

In May, Banks led a letter signed by 40 other Republican House members that asked President Donald Trump to replace Dr. Francis Collins as the director of the NIH because of Collins' support for human embryonic stem cell research. Trump announced last week that he is retaining Collins, a geneticist nominated for NIH chief by President Barack Obama and confirmed by unanimous consent by the Senate in 2009.

The NIH is an HHS agency.

bfancisco@jg.net

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Banks targets embryonic stem cell research funding - Fort Wayne Journal Gazette

Spinal Cord Stem Cells Comments Off on Banks targets embryonic stem cell research funding – Fort Wayne Journal Gazette | June 16th, 2017

Eva Feldman, M.D., Ph.D.(Photo: Detroit News file photo)

World-renowned researcher Dr. Eva Feldman is stepping down as director of the University of Michigans A. Alfred Taubman Medical Research Institute, an organization that supports research for cures for debilitating and deadly diseases.

Feldman was the founding director of the institute launched by philanthropist and businessman A. Alfred Taubman, who died in 2015 at age 91. Feldman spent the last 10 years at the helm as the institute developed new drugs, surgeries and therapies for diseases such as adult and childhood cancer, diabetes, cardiovascular disease and, Feldmans research interest, amyotrophic lateral sclerosis.

When Alfred and I began the institute 10 years ago together, we had a vision of creating an institute where clinician scientists would take their most novel discoveries from their laboratory and translate that into patient clinical trials, said Feldman, adding that the team of four has grown to over 200. Weve really been able to realize that vision.

Feldman, 65, said Thursday that she raised the institute from its infancy.

Its now time for the next person to take it through its adolescence, because its still growing, she said. So it just seemed the perfect time to pass the baton to someone to have the next 10 best years of their life the way I just had the 10 best years of my life.

The institute will select the next director soon, Feldman said.

A Russell N. DeJong Professor of Neurology, Feldman will continue to run the ALS clinic at UM and her own laboratory, Program for Neurology Research & Discovery. Her team of 30 scientists have spent years working on human clinical trials that may lead to a treatment for ALS, a neurological disease also known as Lou Gehrigs disease. Most ALS patients lose the ability to walk and talk, and death may occur within three to five years of diagnosis.

In 2010, Feldman started the first human clinical trial approved by the U.S. Food and Drug Administration to use stem cells to treat ALS. The FDA recently approved Feldman to move onto the final, nationwide phase of the trial that involves injecting stem cells into the spinal cord of ALS patients. If approved, the drug could be marketed to patients.

Besides focusing on the trial, Feldman said shell have more time to do a deep dive into why people with diabetes get neurological complications.

She also was elected to the National Academy of Medicine and plans to use her position to help others understand the value of philanthropy.

Philanthropy can be a game changer Al Taubman showed us that and one of my goals is to help other clinician scientists throughout the country understand the process, she said.

Feldman said shes much closer to a cure for ALS, but were not there yet. For now, she wants to press the pause button on the disease.

When the patient comes to me, I want to be able to offer them a therapy so they will stay right where they are the first day I see them, she said. I dont think that stem cells will necessarily make people go back to where they are before they got ill, but if we can stop the disease in its tracks, my patients will be happy and I will be happy.

Feldman was named a 2011 Detroit News Michiganian of the Year. Shes served as the president of the American Neurological Association, the third woman to hold the position in 130 years, and was named one of Americas Top Doctors in 2016.

ssteinberg@detroitnews.com

(313) 222-2156

Twitter: @Steph_Steinberg

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Eva Feldman steps down as Taubman Institute director - The Detroit News

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June 15, 2017

Patients with severe and end-stage heart failure have few treatment options available to them apart from transplants and "miraculous" stem cell therapy. But a new Tel Aviv University study finds that stem cell therapy may, in fact, harm heart disease patients.

The research, led by Prof. Jonathan Leor of TAU's Sackler Faculty of Medicine and Sheba Medical Center and conducted by TAU's Dr. Nili Naftali-Shani, explores the current practice of using cells from the host patient to repair tissueand contends that this can prove deleterious or toxic for patients. The study was recently published in the journal Circulation.

"We found that, contrary to popular belief, tissue stem cells derived from sick hearts do not contribute to heart healing after injury," said Prof. Leor. "Furthermore, we found that these cells are affected by the inflammatory environment and develop inflammatory properties. The affected stem cells may even exacerbate damage to the already diseased heart muscle."

Tissue or adult stem cells"blank" cells that can act as a repair kit for the body by replacing damaged tissueencourage the regeneration of blood vessel cells and new heart muscle tissue. Faced with a worse survival rate than many cancers, many heart failure patients have turned to stem cell therapy as a last resort.

"But our findings suggest that stem cells, like any drug, can have adverse effects," said Prof. Leor. "We concluded that stem cells used in cardiac therapy should be drawn from healthy donors or be better genetically engineered for the patient."

Hope for improved cardiac stem cell therapy

In addition, the researchers also discovered the molecular pathway involved in the negative interaction between stem cells and the immune system as they isolated stem cells in mouse models of heart disease. After exploring the molecular pathway in mice, the researchers focused on cardiac stem cells in patients with heart disease.

The results could help improve the use of autologous stem cellsthose drawn from the patients themselvesin cardiac therapy, Prof. Leor said.

"We showed that the deletion of the gene responsible for this pathway can restore the original therapeutic function of the cells," said Prof. Leor. "Our findings determine the potential negative effects of inflammation on stem cell function as they're currently used. The use of autologous stem cells from patients with heart disease should be modified. Only stem cells from healthy donors or genetically engineered cells should be used in treating cardiac conditions."

The researchers are currently testing a gene editing technique (CRISPER) to inhibit the gene responsible for the negative inflammatory properties of the cardiac stem cells of heart disease patients. "We hope our engineered stem cells will be resistant to the negative effects of the immune system," said Prof. Leor.

Explore further: Adult stem cell types' heart repair potential probed

More information: Nili Naftali-Shani et al, Left Ventricular Dysfunction Switches Mesenchymal Stromal Cells Toward an Inflammatory Phenotype and Impairs Their Reparative Properties Via Toll-Like Receptor-4Clinical Perspective, Circulation (2017). DOI: 10.1161/CIRCULATIONAHA.116.023527

Journal reference: Circulation

Provided by: Tel Aviv University

New University of Otago research is providing fresh insights into how a patient's adult stem cells could best be used to regenerate their diseased hearts.

Genetically engineered human cardiac stem cells helped repair damaged heart tissue and improved function after a heart attack, in a new animal study.

Scientists use mathematical modeling to simulate human mesenchymal stem cell delivery to a damaged heart and found that using one sub-set of these stem cells minimises the risks associated with this therapy. The study, published ...

An international team of researchers, funded by Morris Animal Foundation, has shown that adipose (fat) stem cells might be the preferred stem cell type for use in canine therapeutic applications, including orthopedic diseases ...

A*STAR researchers and colleagues have developed a method to isolate and expand human heart stem cells, also known as cardiac progenitor cells, which could have great potential for repairing injured heart tissue.

(HealthDay)A new method for delivering stem cells to damaged heart muscle has shown early promise in treating severe heart failure, researchers report online July 27 in Stem Cells Translational Medicine.

Researchers at Karolinska Institutet in Sweden have obtained the first 3D snapshots of a sperm protein attached to a complementary egg coat protein at the beginning of fertilisation. The study, which reveals a common egg ...

As we bask in the summer heat, it is easy to take for granted that humans are also prepared for the cold of winter, with overcoats in the closet and home heating systems ready to be fired up as an added assurance against ...

Organs-on-Chips (Organ Chips) are emerging as powerful tools that allow researchers to study the physiology of human organs and tissues in ways not possible before. By mimicking normal blood flow, the mechanical microenvironment, ...

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Researchers from Imperial College London and colleagues have found a potential way to target the receptors that specifically control appetite in mouse brains, potentially without causing other side effects.

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Cardiac stem cells from heart disease patients may be harmful - Medical Xpress

Cardiac Stem Cells Comments Off on Cardiac stem cells from heart disease patients may be harmful – Medical Xpress | June 16th, 2017

Russias Progress 67 (67P) cargo craft is orbiting Earth and on its way to the International Space Station Friday morning carrying over three tons of food, fuel and supplies. Meanwhile, the three member Expedition 52 crew researched a variety of space science on Thursday while preparing for the arrival of the 67P.

Commander Fyodor Yurchikhin and Flight Engineer Jack Fischer will monitor the automated docking of the 67P to the Zvezda service module Friday at 7:42 a.m. EDT. NASA TV will broadcast live the resupply ships approach and rendezvous beginning at 7 a.m. The 67Ps docking will mark four spaceships attached to the space station.

Fischer spent the morning photographing mold and bacteria samples on petri dishes as part of six student-led biology experiments that are taking place inside a NanoRacks module. In the afternoon, he removed protein crystal samples from a science freezer, let them thaw and observed the samples using a specialized microscope.

Flight Engineer Peggy Whitson tended to rodents Thursday morning cleaning their habitat facilities and restocking their food. In the afternoon, she moved to human research swapping out samples for the Cardiac Stem Cells study that is exploring why living in space may accelerate the aging process.

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Station Crew Researches Mold, Rodents and Stem Cells as Cargo Ship Chases Station - Space Fellowship

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Back to Browse Journals International Journal of Women's Health Volume 9

Ahmed Badawy,1 Mohamed A Sobh,2 Mohamed Ahdy,3 Mohamed Sayed Abdelhafez1

1Department of Obstetrics and Gynecology, 2Department of Internal Medicine, 3Department of Clinical Pharmacology, Mansoura University, Mansoura, Egypt

Objective: Attempting in vivo healing of cyclophosphamide-induced ovarian insufficiency in a mouse model using bone marrow mesenchymal stem cells (BMMSCs). Methods: Female BALB/c white mice were used to prepare a model for premature ovarian failure by single intraperitoneal injection of cyclophosphamide (80 mg/kg). Ten mice were injected with BMMSCs and then sacrificed after 21 days for morphometric evaluation of the ovaries. Hormonal profile was evaluated while mice were being sacrificed. Another 10 mice were left for natural breeding with male mice, and 5 of these were injected with BMMSCs. Oocyte-like structures were obtained from 3 mice and were subjected to in vitro fertilization/intracytoplasmic sperm injection. Results: Morphometric analysis of the ovaries demonstrated the presence of newly formed primordial follicles. Contribution of MSCs to the formation of these follicles was proven by a labeling technique. There was a drop in estradiol and rise in follicle-stimulating hormone levels, followed by resumption of the hormonal levels to near normal 21 days after MSCs therapy. The 5 mice that were injected with MSCs became pregnant after natural breeding. Fertilization and further division was reported in 5 oocytes subjected to intracytoplasmic sperm injection, but division did not continue. Conclusion: From this proof-of-concept trial, we can say that healing of damaged ovaries after chemotherapy in mice is possible using in vivo therapy with BMMSCs. This should open the gate for a series of animal studies that test the possibility of in vitro maturation of germinal epithelium of the ovary into mature oocytes.

Keywords: cyclophosphamide, stem cell, POF, ovarian insufficiency

This work is published and licensed by Dove Medical Press Limited. The full terms of this license are available at https://www.dovepress.com/terms.php and incorporate the Creative Commons Attribution - Non Commercial (unported, v3.0) License. By accessing the work you hereby accept the Terms. Non-commercial uses of the work are permitted without any further permission from Dove Medical Press Limited, provided the work is properly attributed. For permission for commercial use of this work, please see paragraphs 4.2 and 5 of our Terms.

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Bone marrow mesenchymal stem cell repair of cyclophosphamide-induced ovarian insufficiency in a mouse model - Dove Medical Press

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Newswise You might think stem cells only exist inside a fetus, but your adult body has a stockpile of stem cells, armed and ready to respond. These remarkable cells can develop into any other type of cell, like muscle or bone or nerve cells.

Researchers know heart attacks and strokes summon these cells. They flock to your heart or brain from all over your body to help you stay alive.

But, scientists did not realize other injuries, like a torn ACL of the knee, could command the army of stem cells to deploy.

Kevin Baker, Ph.D., Beaumont director of Orthopedic Research, conducted a study with Beaumont orthopedic surgeon Kyle Anderson, M.D., and others that revealed ACL tears send a signal to stem cells throughout our body.

After an ACL tear, Dr. Baker and his colleagues found a six-fold increase in stem cells circulating around the knee, similar to the bodys response to a major, life-threatening event like a stroke or heart attack.

However, when the stem cells arrive to help regenerate and repair the injured ligament, they get stuck. They cant get through the thick membrane that surrounds the knee joint.

We think this discovery will help us to understand how the body responds to an ACL injury, and also how post-traumatic osteoarthritis develops after a joint injury, Dr. Anderson said.

Post-traumatic osteoarthritis is a form of arthritis that develops after a knee injury. Its a common injury that affects veterans, athletes and anyone who puts stress and strain on their knees. But, until now, little was known about how the body attempts to heal these injuries.

As we age, the number of stem cells in our body declines. This could explain why your knee joint doesnt heal as well after a trauma when you are older, Dr. Baker said.

Osteoarthritis affects more than 30 million adults in the United States, according to the Centers for Disease Control and Prevention, and many of these cases occur after trauma to a joint. Its also a leading cause of disability.

The next step of our research will be finding methods to get the stem cells inside the joint. If the stem cells can get through the membrane around the knee, they could help speed up the healing process and perhaps delay or prevent arthritis, Dr. Baker added.

The study, funded in part by the American Orthopedic Society of Sports Medicine, is entitled, Acute mobilization and migration of bone marrow-derived stem cells following anterior cruciate ligament rupture. The authors believe it is the first study of its kind to reveal the bodys systemic stem cell response to an ACL injury.

Dr. Baker and Dr. Andersons research will appear in an upcoming edition of the journal Osteoarthritis and Cartilage. Other members of the research team are Perry Altman, M.D., Beaumont orthopaedic surgery resident, as well as Asheesh Bedi, M.D., and Tristan Maerz, Ph.D., of the University of Michigan.

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Researchers Discover Body's Stem Cell Army Hits a Wall When ... - Newswise (press release)

Bone Marrow Stem Cells Comments Off on Researchers Discover Body’s Stem Cell Army Hits a Wall When … – Newswise (press release) | June 16th, 2017

Domainex will expand its two-year-old collaboration with Imperial College London to discover new therapies that reduce heart muscle damage during heart attacks, the partners said today.

Domainex and Imperial aim to discover a treatment that inhibits the enzyme MAP4K4, which is linked to cell death following heart attacks. Since the collaboration was launched in 2015, the partners said, they have discovered novel, potent, and selective MAP4K4 inhibitors using human cardiac muscle grown from human induced pluripotent stem cells (iPSCs).

The inhibitors have shown promise in protecting these cells against oxidative stress, a trigger for cell death during heart attacks, Domainex and Imperial said.

As a result of the progress, Imperial College London said, its Professor Michael Schneider, Ph.D., has secured a follow-on award of 4.5 million (nearly $5.8 million) from the Wellcome Trusts Seeding Drug Discovery initiative to continue the research.

From its Medicines Research Centre near Cambridge, U.K., Domainex said, its researchers will continue to provide integrated drug discovery servicesincluding further biochemical, cellular and biophysical assay screening, and structure-guided medicinal chemistry coupled with drug metabolism, safety, and pharmacokinetic assessment of promising candidates.

Domainex and Imperial said they aim to advance potential treatments into preclinical development and ultimately to clinical evaluation.

"We have already identified a number of very exciting, novel inhibitors through structure-based drug design," Domainex CSO Trevor Perrior said in a statement. The innovative cardiac muscle assay developed by the team here at Domainex working in partnership with Imperial College London, is enabling early testing on human cardiac muscle cells, which will make cardiac drug discovery more efficient and effective in identifying efficacious candidate drugs.

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Domainex, Imperial College London Extend Cardiac Therapy Collaboration - Genetic Engineering & Biotechnology News

Cardiac Stem Cells Comments Off on Domainex, Imperial College London Extend Cardiac Therapy Collaboration – Genetic Engineering & Biotechnology News | June 14th, 2017

Release from the University of Virginia Health System:

CHARLOTTESVILLE, Va., June 14, 2017 - University of Virginia Cancer Center has earned recognition as a national center of excellence for its care of patients with myelodysplastic syndrome (MDS), a cancer of the bone marrow that often leads to leukemia.

UVA is the only center in Virginia to receive this designation from the MDS Foundation for the treatment of this condition, which UVA hematologist Michael Keng, MD, said is often referred to as a bone marrow failure disorder.

Bone marrow produces stem cells that make white blood cells, red blood cells and platelets. In patients with MDS, the marrow does not produce enough healthy cells. When there are not enough healthy cells, there is an increased risk of infection, bleeding, easy bruising and anemia. Approximately 30 percent of patients diagnosed with MDS will progress to a diagnosis of acute myeloid leukemia.

According to the MDS Foundation website, centers of excellence have:

UVA provides tailored care for each MDS patient through a multidisciplinary team. UVAs care team includes medical oncologists/hematologists, pharmacists, care coordinators, nurses, infectious diseases specialists, clinical trial coordinators, and support services such as social workers, case workers, and therapists.

UVA is devoted to providing support, research, treatment and education around MDS to all patients, caregivers, physicians, nurses and other healthcare providers, Keng said.

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UVA Honored as Center of Excellence for Bone Marrow Cancer - NBC 29 News

Bone Marrow Stem Cells Comments Off on UVA Honored as Center of Excellence for Bone Marrow Cancer – NBC 29 News | June 14th, 2017

Dr. Keith Roach, Syndicated Columnist

DEAR DR. ROACH: I hope you can answer some questions about myelodysplastic syndrome. What does it do to your body? Is there a known cause or cure? What is the prognosis? P.B. ANSWER: The myelodysplastic syndromes are a group of similar diseases, specific types of blood cancers, that prevent your bone marrow from working properly. They also can transform into acute leukemia. These are uncommon cancers, with perhaps 30,000 cases per year in the U.S. The specific myelodysplastic syndromes are now categorized by appearance, genetic abnormalities of the cells, and condition of the bone marrow.

MDS may arise from damage to DNA, such as from radiation or other toxic exposures. However, many cases have no known cause, and its likely that these are spontaneous mutations in the bone marrow cells.

Because MDS is a group of related diseases, the treatment and prognosis vary among the different subtypes. However, supporting the bone marrow with transfusions of red blood cells and platelets often is necessary. Medications to stimulate both red and white blood cell production can be used. A few people will be recommended for bone marrow (stem cell) transplant, but the decision to consider this treatment must be made cautiously, as many people who get MDS will not benefit from this treatment due to age or other medical conditions.

The prognosis depends on the age of the person affected and their specific MDS. A person younger than 60 with a low-risk MDS has a median survival (based on data published in 1997) of about 12 years. However, high-risk MDS has a much worse outcome: Half of people succumb within six months. Advances in treatment since these data were published have improved these results, but not as much as hoped.

DEAR DR. ROACH: My 89-year-old mother suffers from fluttering in her heart. She saw an expert in cardiac arrhythmias, who diagnosed her with tachy-brady syndrome and sick sinus syndrome. A nurse also said she has PVCs. She is taking metoprolol, but still has episodes of fluttering. What are these conditions? Are there other medications she could take to correct this heart condition? M.D.P.

ANSWER: Tachy-brady syndrome (from the Greek roots for fast and slow) and sick sinus syndrome are the same thing. The sinus in sick sinus syndrome refers to the sino-atrial node of the heart, which is the hearts natural pacemaker. It is where every beat normally starts. This part of the heart can become diseased, and the heart can beat both too quickly (tachycardia) and, at other times, too slowly (bradycardia). Sick sinus syndrome can come from many different conditions and, rarely, from medications.

Medications are sometimes used for sick sinus syndrome. Beta blockers, like the metoprolol your mother is taking, are given to slow down the tachycardic component of sick sinus, but it can make the bradycardia worse. Most often, the treatment for sick sinus syndrome is a permanent pacemaker. Not everyone needs it, but Im sure your mothers cardiologist is monitoring her and will recommend a pacemaker if needed. If one is necessary, 89 years old is not too old to put in a pacemaker.

PVCs are very common and do not usually indicate disease in the heart, although they are more common in people with heart disease, especially poor blood flow to the heart. Premature ventricular contractions themselves seldom need treatment.

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TO YOUR GOOD HEALTH: 'Myelodysplastic syndrome' covers a range of diseases - Prescott Daily Courier

Bone Marrow Stem Cells Comments Off on TO YOUR GOOD HEALTH: ‘Myelodysplastic syndrome’ covers a range of diseases – Prescott Daily Courier | June 14th, 2017

AUSTIN, Texas -- Gov. Greg Abbott has signed a new law that allows terminally ill or those which chronic diseases receive stem cell treatments in Texas.

Stem cell therapy is the use of stem cells to treat or prevent a disease or condition, and is often patient's last hope for improvement.

Bone marrow transplant is the most widely used stem-cell therapy, and can often help those with multiple sclerosis and other diseases.

House Bill 810, which was introduced by Rep. Tan Parker, R-Flower Mound, passed in both the Texas House and Senate.

"It is easy to fall into the trap of viewing legislation as just words on a piece of paper," said Sen. Paul Bettencourt, R-Houston, the bill's sponsor in the Senate. "But for the many people who are ill with multiple sclerosis and other diseases that stem cell therapy has the hope of solving in our lifetime, I look at this bill, I look at the possibility of what can happen in the 21st Century, with Texas taking the lead on adult stem cell treatments and this bill has the potential to extend lives and make a difference for these patients."

The Texas Medical Board will be responsible for writing the rules for the treatment.

"Everyone has a zest for life. This adult stem cell treatment possibility gets government out of the way to let these new therapies flourish and give these patients hope for a future good quality of life," Bettencourt added.

The legislation takes effect Sept. 1.

-- Value of Stem Cell Therapy --

According to the National Institues of Health, stem cellshave the remarkable potential to develop into many different cell types in the body during early life and growth.

In addition, in many tissues they serve as a sort of internal repair system, dividing essentially without limit to replenish other cells as long as the person or animal is still alive.

When a stem cell divides, each new cell has the potential either to remain a stem cell or become another type of cell with a more specialized function, such as a muscle cell, a red blood cell, or a brain cell.

Doctors say stem cells are important for living organisms for many reasons.

In the 3- to 5-day-old embryo, called ablastocyst, the inner cells give rise to the entire body of the organism, including all of the many specialized cell types and organs such as the heart, lungs, skin, sperm, eggs and other tissues.

In some adult tissues, such as bone marrow, muscle, and brain, discrete populations of adult stem cells generate replacements for cells that are lost through normal wear and tear, injury, or disease.

---

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Governor Signs Law to Allow Chronic, Terminally Ill in Texas to Get Stem Cell Treatments - Spectrum News

Bone Marrow Stem Cells Comments Off on Governor Signs Law to Allow Chronic, Terminally Ill in Texas to Get Stem Cell Treatments – Spectrum News | June 14th, 2017

When she entered medicine in the mid-1980s, Masayo Takahashi chose ophthalmology as her specialty, she said, because she wanted to have a family and thought the discipline would spare her from sudden work calls in the middle of the night, helping her best balance work and life.

Three decades later, the 55-year-old mother of two grown-up daughters is at the forefront of the nations even the worlds research into regenerative medicine.

In September 2014, she offered a ray of hope to scores of patients suffering from a severe eye condition when her team at the Riken institutes Center for Developmental Biology in Kobe succeeded in a world-first transplanting of cells made from induced pluripotent stem (iPS) cells into a human body.

The operation, conducted as a clinical study, involved creating a retinal sheet from iPS cells, which were developed by Shinya Yamanaka, a researcher at Kyoto University. His 2006 discovery of iPS cells, which can grow into any kind of tissue in the body, won him a Nobel Prize in 2012.

During the 2014 procedure, the retinal sheet was transplanted into a female patient in her 70s with age-related macular degeneration (AMD), an eye disorder that blurs the central field of vision and can lead to blindness. The research team used iPS cells made from the patients own skin cells.

Takahashi made history again in March when she and her team carried out the worlds first transplant of retina cells created from donor iPS cells stocked at Kyoto University. The time and cost necessary for the procedure has been significantly reduced by using the cells, which are made from super-donors, people with special white blood cell types that arent rejected by the immune systems of receiving patients.

Takahashi was in Tokyo last week to speak at the Foreign Press Center and later with The Japan Times. She recounted the highlights of her 25-year research and the numerous legal and other challenges she has overcome.

Takahashi points to the day she led that first iPS transplant surgery Sept. 12, 2014 as the high point of her career so far. Because she worked so hard leading up to the surgery to confirm the safety of the retinal cells, she said that when the operation was over, she was relieved and slept very well.

It wasnt the same for Yamanaka, who provided the stem cells to Takahashi, she said, chuckling. Yamanaka-sensei couldnt sleep well after the surgery because he didnt know about the safety of the cells very well. I should have convinced him.

Some researchers have expressed concern that iPS cell-derived cells have a higher risk of developing cancer. But Takahashi said she knew from the outset that the type her team was making, retinal pigment epithelium (RPE) cells, are extremely unlikely to cause tumors. RPE cells make up the pigmented layer of tissue that supports the light-sensitive cells of the retina.

People in the world think iPS cells are very dangerous because we modify the genes, she said. The retinal pigment epithelium cell is very safe. We knew it from the beginning because we have never seen a metastatic tumor from this cell. Ophthalmologists know very well that this cell is very safe and very good.

The Osaka native said she learned of and became fascinated by the possibility of using stem cells for eye diseases in the mid-1990s, when she took a year off from clinical practice at Kyoto University to work as a researcher at the Salk Institute in San Diego. She moved to Riken in 2006.

More than 2 years have passed since that first iPS surgery, but the transplanted cells remain intact. According to Takahashi, it was not the goal of the research from the outset to improve the eyesight of the patient, who suffered from a very severe case of AMD. Before the surgery, the patient required injections of drugs into her eyeball every two months, but her visual acuity was declining. After the surgery, her acuity stabilized, and more importantly, she is happy, feeling that her vision has brightened and widened, Takahashi said.

Many challenges remain, however, to advance the technology and make it commercially available. One of the issues is cost, Takahashi said, adding that it will take until around 2019 before the cost of the iPS treatment for AMD will fall below 10 million. The first surgery in 2014 cost about 100 million in total, much of which was spent to maintain the clean room and culture the cells.

Still, Takahashi sees a huge potential for iPS cell therapy in her field and beyond.

Every disease has potential to be treated by iPS cell-derived cells or ES (embryonic stem) cell-derived cells in the future, she said, responding to a question on the chances of iPS cells being used to treat ALS, a rare, degenerative neurological disease for which there is currently no cure.

She said she has learned through her experience that some patients are very happy with small improvements.

For ALS, at first, I thought, its a systemic, whole-body disease, so I didnt know how they can fix it, she said. But a doctor (who specializes in ALS) said, its OK, if one finger moves, its (still) OK. So I realized that some benefit will come from cell therapy.

A Matter of Health is a weekly series on the latest health research, technology or policy issues in Japan. It appears on Thursdays.

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Transplants using iPS cells put Riken specialist at forefront of regenerative medicine research - The Japan Times

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Scientists have used artificial skin grafts grown from induced pluripotent stem cells to cover the developing spines of rat fetuses while still in the womb.

Asian Scientist Newsroom | June 14, 2017 | In the Lab

AsianScientist (Jun. 14, 2017) - Researchers from Japan have developed a stem cell-based therapy to treat a severe congenital bone defect known as myelomeningocele. Their findings have been published in Stem Cell Reports.

Myelomeningocele is the most serious and common form of spina bifida, a condition in which the backbone and spinal canal do not close before birth, leaving parts of the spinal cord and nerves exposed.

A baby born with this disorder typically has an open area or a fluid-filled sac on the mid to lower back. Most children with this condition are at risk of brain damage because too much fluid builds up in their brains. They also often experience symptoms such as loss of bladder or bowel control, loss of feeling in the legs or feet, and paralysis of the legs.

To develop a minimally invasive treatment to cover large myelomeningocele defects earlier during pregnancy, the researchers first generated artificial skin from human induced pluripotent stem cells (iPSCs), and then successfully transplanted the skin grafts into rat fetuses with the condition.

We provide preclinical proof of concept for a fetal therapy that could improve outcomes and prevent lifelong complications associated with myelomeningoceleone of the most severe birth defects, said senior study author Professor Akihiro Umezawa of Japan's National Research Institute for Child Health and Development.

Since our fetal cell treatment is minimally invasive, it has the potential to become a much-needed novel treatment for myelomeningocele.

The human iPSCs were generated from fetal cells taken from amniotic fluid from two pregnancies with severe fetal disease (Down syndrome and twin-twin transfusion syndrome). The researchers then used a chemical cocktail in a novel protocol to turn the iPSCs into skin cells and treated these cells with additional compounds such as epidermal growth factor to promote their growth into multi-layered skin.

In total, it took approximately 14 weeks from amniotic fluid preparation to 3D skin generation, which would allow for transplantation to be performed in humans during the therapeutic window of 28-29 weeks of gestation.

Next, the researchers transplanted the 3D skin grafts into 20 rat fetuses through a small incision in the uterine wall. The artificial skin partially covered the myelomeningocele defects in eight of the newborn rats and completely covered the defects in four of the newborn rats, protecting the spinal cord from direct exposure to harmful chemicals in the external environment.

Moreover, the engrafted 3D skin regenerated with the growth of the fetus and accelerated skin coverage throughout the pregnancy period. Notably, the transplanted skin cells did not lead to tumor formation, but the treatment significantly decreased birth weight and body length.

We are encouraged by our results and believe that our fetal stem cell therapy has great potential to become a novel treatment for myelomeningocele, Umezawa said. However, additional studies in larger animals are needed to demonstrate that our fetal stem cell therapy safely promotes long-term skin regeneration and neurological improvement.

The article can be found at: Kajiwara et al. (2017) Fetal Therapy Model of Myelomeningocele with Three-dimensional Skin Using Amniotic Fluid Cell-derived Induced Pluripotent Stem Cells.

Source: Cell Press; Photo: Kazuhiro Kajiwara. Disclaimer: This article does not necessarily reflect the views of AsianScientist or its staff.

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3D Artificial Skin Used To Treat Spina Bifida In Rats - Asian Scientist Magazine

IPS Cell Therapy Comments Off on 3D Artificial Skin Used To Treat Spina Bifida In Rats – Asian Scientist Magazine | June 14th, 2017