Ten years ago, the bones currently in your body did not actually exist. Like skin, bone is constantly renewing itself, shedding old tissue and growing it anew from stem cells in the bone marrow. Now, a new technique developed at Caltech can render intact bones transparent, allowing researchers to observe these stem cells within their environment. The method is a breakthrough for testing new drugs to combat diseases like osteoporosis.

The research was done in the laboratory of Viviana Gradinaru (BS '05), assistant professor of biology and biological engineering and a Heritage Medical Research Institute Investigator. It appears in a paper in the April 26 issue of Science Translational Medicine.

In healthy bone, a delicate balance exists between the cells that build bone mass and the cells that break down old bone in a continual remodeling cycle. This process is partially controlled by stem cells in bone marrow, called osteoprogenitors, that develop into osteoblasts or osteocytes, which regulate and maintain the skeleton. To better understand diseases like osteoporosis, which occurs when loss of bone mass leads to a high risk of fractures, it is crucial to study the behavior of stem cells in bone marrow. However, this population is rare and not distributed uniformly throughout the bone.

"Because of the sparsity of the stem cell population in the bone, it is challenging to extrapolate their numbers and positions from just a few slices of bone," says Alon Greenbaum, postdoctoral scholar in biology and biological engineering and co-first author on the paper. "Additionally, slicing into bone causes deterioration and loses the complex and three-dimensional environment of the stem cell inside the bone. So there is a need to see inside intact tissue."

To do this, the team built upon a technique called CLARITY, originally developed for clearing brain tissue during Gradinaru's postgraduate work at Stanford University. CLARITY renders soft tissues, such as brain, transparent by removing opaque molecules called lipids from cells while also providing structural support by an infusion of a clear hydrogel mesh. Gradinaru's group at Caltech later expanded the method to make all of the soft tissue in a mouse's body transparent. The team next set out to develop a way to clear hard tissues, like the bone that makes up our skeleton.

In the work described in the new paper, the team began with bones taken from postmortem transgenic mice. These mice were genetically engineered to have their stem cells fluoresce red so that they could be easily imaged. The team examined the femur and tibia, as well as the bones of the vertebral column; each of the samples was about a few centimeters long. First, the researchers removed calcium from the bones: calcium contributes to opacity, and bone tissue has a much higher amount of calcium than soft tissues. Next, because lipids also provide tissues with structure, the team infused the bone with a hydrogel that locked cellular components like proteins and nucleic acids into place and preserved the architecture of the samples. Finally, a gentle detergent was flowed throughout the bone to wash away the lipids, leaving the bone transparent to the eye. For imaging the cleared bones, the team built a custom light- sheet microscope for fast and high-resolution visualization that would not damage the fluorescent signal. The cleared bones revealed a constellation of red fluorescing stem cells inside.

The group collaborated with researchers at the biotechnology company Amgen to use the method, named Bone CLARITY, to test a new drug developed for treating osteoporosis, which affects millions of Americans per year.

"Our collaborators at Amgen sent us a new therapeutic that increases bone mass," says Ken Chan, graduate student and co-first author of the paper. "However, the effect of these therapeutics on the stem cell population was unclear. We reasoned that they might be increasing the proliferation of stem cells." To test this, the researchers gave one group of mice the treatment and, using Bone CLARITY, compared their vertebral columns with bones from a control group of animals that did not get the drug. "We saw that indeed there was an increase in stem cells with this drug," he says. "Monitoring stem cell responses to these kinds of drugs is crucial because early increases in proliferation are expected while new bone is being built, but long-term proliferation can lead to cancer."

The technique has promising applications for understanding how bones interact with the rest of the body.

"Biologists are beginning to discover that bones are not just structural supports," says Gradinaru, who also serves as the director of the Center for Molecular and Cellular Neuroscience at the Tianqiao and Chrissy Chen Institute for Neuroscience at Caltech. "For example, hormones from bone send the brain signals to regulate appetite, and studying the interface between the skull and the brain is a vital part of neuroscience. It is our hope that Bone CLARITY will help break new ground in understanding the inner workings of these important organs."

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Transparent Bones Enable Researchers to Observe Stem Cells Inside - Laboratory Equipment

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In my opinion, one of the hardest things to accept is a new type of medical treatment, particularly when it changes the philosophy, parameters and overall results that we are expecting and basically used to receiving. Stem cells are undoubtedly no exception to this rule.

About six weeks ago, Eduardo K (a Cuban doctor with a Masters Degree from the University of Pittsburg in internal medicine and nephrology), brought his wife Maria to our institute, in order to assess the possibility of using stem cells to cure the severe chronic pain in her ankle; a pain so severe that it was basically hindering her ability to walk. Dr. K also expressed his extreme hesitation and concerns about having his wife involved in an invasive ankle surgery at this stage of her adult life.

However, while conducting our usual examining process, reviewing her medical records and MRIs and thoroughly discussing my overall recommendations about a potential stem cell transplant, I quickly realized that Dr. K was not a true believer in Stem Cell therapies, since he thought that there was not much medical evidence of their actual effectiveness and he ultimately also confessed that his wife had basically dragged him to accompany her to this particular appointment.

As always, I respectfully explained the reality that stem cells actually repair the damaged cartilage in a microscopic type fashion and thus, while this repair process would not be clearly reflected immediately on future X-rays, I assured them that the pain she was suffering from will soon subside and possibly even completely disappear. In addition, I expressed that I was extremely confident that she would also regain her mobility skills after the procedure, even if this improvement could not be easily detected via a radiological image.

Since Marias options were somewhat limited, added to the fact that months of traditional physical therapy, injections, medications and previous surgeries had completely failed her, Dr. K finally agreed to grant his wifes wishes to have her stem cell transplant (from her own bone marrow and fat) performed, although he was still very skeptical about the process and was showing little enthusiasm.

This morning, both of them attended our follow up appointment (six weeks after the procedure) and surprisingly, Maria and Dr. K happily confirmed that she felt at least 60 percent better, something that no previous traditional medical treatments had been able to accomplish. It was then that I explained to them that her stem cells had acted much faster than expected (something that possibly taught Dr. K an interesting lesson).

As we began to say our goodbyes, the doctor told me (first in English, then in Spanish) that: in spite of my skepticism about stem cell therapies, I can personally attest that the successful results seen on my wife have been irrefutable, and with a smile on both of their faces, they gratefully thanked my staff and I for this amazing improvement.

As I continued to replay the words expressed by this doctor over and over in my mind, I quickly realized how truly incredulous human beings tend to be, with most of us often needing to fail several times at accomplishing something before being able to realize and accept that we were truly mistaken in the first place!

So if you, a friend or relative would like to receive Stem Cell or PRP treatments, please call us at 305-598-7777. For information visit: http://www.stemcellmia.com (available in both English and Spanish), or watch our amazing video-testimonies on our YouTube Chanel and also please follow us on Facebook and Twitter. If you would like to ask a question directly to Dr. Castellanos, please do so via his direct email: stemdoc305@gmail.com.

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The irrefutable success of stem cell treatments - Miami's Community Newspapers

N

ow you see it, now you dont: Scientists have used a chemical technique to make mouse bones turn transparent. The technique has been used in the past to make brains and kidneyssee-through, but this marks the first time its been used in hard tissues.

The ability to see within a bone couldhave implications for research into bone diseases, by letting researchers get a more accurate picture of bones internal structure.

The technique is called CLARITY, and since 2013, when it was first described, it has been deployed on a wide variety of mammalian tissues and inplants. Caltech neuroscientistViviana Gradinaru, an original developer of the technique, even cleared an entire mouses body in 2014 (except for its bones, which were unaffected, she said).

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The approach works by chemically locking proteins and DNA in place with a hydrogel, after which researchers wash away fats within the tissue. Lipids refract light, so this washing step makes CLARITY-treated tissues transparent.

Flexible 3-D printed scaffolds could mend broken bones

In this case, Gradinaruwanted to look at bone marrow and count the number of stem cells that could ultimately produce new bone cells.

Bone is not a static organ. It iscontinuously changed. The bones we have in our body, we didnt have them 10 years ago, she explained. Acontinuous process of bone cell death and bone cell growth ishappening, spurred by progenitor cells in a bones soft, spongy marrow.

But looking for these cells can bechallenging. There arent that many progenitor cells, soextrapolating the number and distribution based ona small sample isnt ideal. Researchers can slice the bone, but cuttingcan damage the edges. Putting images of the sliced bones back together into a coherent, 3-D picture is very difficult, too.A clear bone avoidsslicing altogether.

Doug Richardson, director of imaging at the Harvard Center for Biological Imaging, said the paper represented a step forward in bone clearing. (Richardson was not involved in this research.)

This technique has the potential to monitor bone health or disease progression over larger volumes with greater accuracy, he said.

Gradinarus team has already demonstrated one possible application. They found that a drug for osteoporosis, currently being developed by Amgen, triggered an increase in the number of stem cells in CLARITY-treated bone.Some Amgen scientists were coauthors of the paper.

Using CLARITY let the team more effectively measure the rate of this increase.This is very important, because you want a controlled increase too much of an increase can lead to tumors, Gradinarusaid.

Other uses could be on the horizon. Being able to make a mouse or rat skull see-through could be useful for Gradinarus fellow neuroscientists who use implants in their research and want to establish the exact position of the impact after experiments are done.

Theres still more work to be done. For instance, finding a way to tagthe samples with antibodies without having to cut a bone in half, as researchers did in this paper would be ideal.Gradinaru also wouldnt mind some speed improvements:In this case, the CLARITY process took nearly a month.

Its not a fast method, by any means, Gradinaru said. However, the result theres no substitute for getting 3-D access to the intact bone marrow.

Kate Sheridan can be reached at kate.sheridan@statnews.com Follow Kate on Twitter @sheridan_kate

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Scientists turn bones transparent to let them see into marrow - Stat - STAT

Piero Anversa

Brigham and Womens Hospital and its parent healthcare network have agreed to pay $10 million to the U.S. government to resolve allegations it fraudulently obtained federal funding.

The case, which involves three former Harvard stem cell researchers, dates back several years. In 2014, Circulation retracted a paper by Piero Anversa, Annarosa Leri, and Jan Kajstura, among others, amidst a university investigation into misconduct allegations. Anversa and Leri whose lab was described as filled with fear by one former research fellow later sued the hospital for notifying journals of that investigation. They lost.

In the agreement announced today by the Department of Justice (DOJ), Partners Healthcare and Brigham and Womens Hospital have agreed to pay the government $10 million to settle allegations that the researchers fraudulently obtained funding from the National Institutes of Health:

The settlement resolves allegations that Dr. Anversa, along with Dr. Annarosa Leri and Dr. Jan Kajstura, knew or should have known that their laboratory promulgated and relied upon manipulated and falsified information, including confocal microscope images and carbon-14 age data for cells, in applications submitted for NIH research grant awards concerning the purported ability of stem cells to repair damage to the heart. The government alleges that problems with the work of the laboratory included improper protocols, invalid and inaccurately characterized cardiac stem cells, reckless or deliberately misleading record-keeping, and discrepancies and/or fabrication of data and images included in applications and publications. The government contends that, at the direction of these BWH scientists, the Anversa laboratory included false scientific information in claims to NIH in order to obtain and use funds from NIH grants.

John Thomas, a partner with Gentry Locke who represents whistleblowers who raise allegations of misconduct, told us that other cases have settled for large amounts such as a recent settlement with Columbia University for $9.5 million. But there are other elements that make this latest announcement noteworthy, he said.

Specifically, most settlements involve some black and white failure in research administration, such as misrepresenting a researchers qualifications on a grant, misreporting effort, or conducting the research at a different facility (such as the Columbia case), said Thomas. The latest decision, in contrast, goes to the science itself.

As a result, he said:

This demonstrates the government is still willing to step in even when its not an administrative issue, even when it goes to the merits of the actual misconduct allegations. I think it shows that when the government awards grants, one of the things its intending to get is good and honest science. The science matters.

We reached out to Anversa and Levi; in response, we received a statement from their attorney:

It is outrageous that BWH has sought to unfairly tarnish the reputations of Drs Anversa and Leri, scientists who have pioneered groundbreaking work in cardiac stem cell reproduction and who have litigation pending against BWH and its president, Betsy Nabel.

Neither Dr. Anversa nor Dr. Leri was involved in the settlement process. BWH self-reported allegations in its own way for its own purposes. It has long been known that Drs. Anversa and Leri relied on the work of a senior scientist in the lab in presenting data for grants and in publications. No one has ever shown that either Dr. Anversa or Dr. Leri participated in the fraud or was aware of it at the time.

BWHs allegations and settlement cannot take away from Drs. Anversa and Leris contributions to the science of stem cell replication. There are Phase II trials being conducted by NIH currently on stem cell treatments based on their work. Taxpayer money was rightly used to further the fight against heart disease, a leading cause of death in this country.

According to the U.S. Attorneys Office, Brigham and Womens Hospital brought the allegations to the governments attention:

After learning of the allegations of research misconduct in the Anversa laboratory, BWH investigated the allegations, disclosed its concerns to the U.S. Department of Health and Human Services, Office of the Inspector General (OIG) and Office of Research Integrity, and then worked cooperatively with OIG and the Department of Justice to explain the bases for the allegations.

The three researchers Anversa, Leri, and Kajstura are no longer based at Brigham and Womens Hospital. Earlier this year, Anversa and Leri published a paper about cardiac progenitor cells that lists an affiliation at the Swiss Institute for Regenerative Medicine.

In todays statement, Acting U.S. Attorney William D. Weinreb said:

Individuals and institutions that receive research funding from NIH have an obligation to conduct their research honestly and not to alter results to conform with unproven hypotheses.

Like Retraction Watch? Consider making atax-deductible contribution to support our growth. You can also follow uson Twitter, like uson Facebook, add us to yourRSS reader, sign up on ourhomepagefor an email every time theres a new post, or subscribe to ourdaily digest. Clickhere to review our Comments Policy. For a sneak peek at what were working on,click here.

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Ottawa Sun

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

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Tough time: Jonathan Pitre battles kidney complications - Ottawa Sun

This weekend the community will be given the chance to save the life of one of its youngest residents. On Friday, April 28, the AmVets Post No. 94 will host a bone marrow registration drive from 3-7 p.m.

Wade Wachter is the son of Adam and Jenni (Duvall) Wachter of Perryville and the grandson of Terri and Lori Duvall, Robyn Roy, and Rodney and Barb Wachter.

On the outside, little Wade is a normal kid though he has been battling a very rare form of bone marrow failure disorder called Schwachman Diamond Syndrome. This dysfunction of the bone marrow requires a lifesaving transplant. He currently takes medication daily and has routine biopsies to monitor for potential leukemia developments in his body.

This disease is so rare that funding is hard to find, which limits the number of possible treatments available. DKMS is the nonprofit group leading the charge to find a bone marrow match for Wachter.

Recent tests show that Wachter will need an immediate transplant for his best chance to have a normal childhood, and according to the DKMS website only 30 percent of patients find a donor inside their families. Nearly 14,000 patients require donations from matched individuals outside of their family line each year. Out of more than 800,000 donors in the U.S., and over 6 million worldwide, 6 out of 10 patients are still unable to find a compatible donor.

We thank DKMS and our community for working with us to help find Wade a bone marrow match, said Jenni Wachter, Wades mother. From the outside, Wade may look like your average 6-year-old child, when really he has been facing a life-threatening battle for years. Our hope is to grow the bone marrow registry to help increase the chances of finding Wade a match so he can move forward towards a healthy and happy life.

Potential donors include anyone who is in good general health between the ages of 18 to 55. Registration is free and only requires filling out a simple form and a quick swab of the inside of each cheek. DKMS covers the $65 registration and processing fee for each supporter, but donations will be accepted to cover costs.

There are two ways to donate once a match has been found. The first method is the Peripheral Blood Stem Cell (PBSC) donation. This is a non-surgical, outpatient procedure that collects blood stem cells via the bloodstream. It takes about 4-8 hours on 1-2 consecutive days. This method is used in 75 percent of all cases. The other donation method is by direct bone marrow procedure. It is a 1-2 hour surgical procedure, done under anesthesia, where a syringe collects marrow cells from the back of the pelvic bone. This method is only used in about 25 percent of the cases, usually when the patient is a child.

Anyone unable to attend the drive that wishes to register as a potential donor may do so online at http://www.dkms.org. The Perryville AmVets Post No. 94 is located at 1203 W. Saint Joseph Street.

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Bone marrow drive set for local youth - Perry County Republic Monitor

A New Type of Stem Cell

While much has been gleaned about the power of stem cells over the last few decades, researchers from the Salk Institute and Peking Universityin China recently found out theres plenty left to discover and invent. Nature, it seems, will always keep you guessing.

In a study published in the journal Cell, the team of researchers revealed they had succeeded in creating a new kind of stem cell thats capable of becoming any type of cell in the human body. Extended pluripotent stem cells or EPS cells are similar to induced pluripotent stem cells(iPS cells), which were invented in 2006.

The key difference between the two is that iPS cells are made from skin cells (called fibroblasts) and EPS cells are made from a combination of skin cells and embryonic stem cells. iPS cells are the hallmark of stem cell research and can be programmed to become any cell in the human body hence the pluripotent part of their name. EPS cells, too, can give rise to any type of cell in the human body, but they can also do something very different something unprecedented, actually: they can create the tissues needed to nourish and grow an embryo.

The discovery of EPS cells provides a potential opportunity for developing a universal method to establish stem cells that have extended developmental potency in mammals, says Jun Wu, one of the studys authors and senior scientist at the Salk Institute, in the organizations news release.

When a human or any mammalian egg gets fertilized, the cells divide up into two task forces: one set is responsible for creating the embryo, and the other set creates the placenta and other supportive tissues needed for the embryo to survive (called extra-embryonic tissues). This happens very early in the reproductive process so early, in fact, that researchers have had a very hard time recreating it in a lab setting.

By culturing and studying both types of cells in action, researchers would not only be able to understand the mechanism that drives it, but hopefully could shed some light on what happens when things go wrong, like in the case of miscarriage.

The researchers at the Salk Institute managed to form a chemical cocktail of four chemicals and a type of growth factor that created a stable environment in which they could culture both types of cells in an immature state. They could then harness the two types of cells for their respective abilities.

What they discovered was that not only were these cells extremely useful for creating chimeras (where two types of animal cells or human and animal cells are mixed to form something new), but were also technically capable of creating and sustaining an entire embryo.At least in theory: while they were able to sustain both human and mouse cells, the ethical considerations of creating a human embryo this way have prevented them from attempting it.

That being said, theres no shortage of applications for this type of stem cell: researchers will be able to use them to model diseases, regenerate tissue, create and trial drug therapies, and study in depth early reproductive processes like implantation. Human-animal chimeras may also help engineer organs for transplant or, you know, give rise to the next superhero.

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Researchers Invent Stem Cell Capable of Becoming an Entire Embryo - Futurism

Science Daily

Discovery offers new hope to repair spinal cord injuries: Scientists ... Science Daily Scientists have created a special type of neuron from human stem cells that could potentially repair spinal cord injuries. These cells, called V2a interneurons, ... and more »

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Discovery offers new hope to repair spinal cord injuries: Scientists ... - Science Daily

Coalition Duchenne funded cardiosphere-derived cell studies in Duchenne carried out by Eduardo Marbn MD, the director of the Cedars-Sinai Heart Institute. This work was later licensed for commercial development by Capricor.

(PRWEB) April 25, 2017

Coalition Duchenne, a charity based in Newport Beach, California, committed to raising awareness for Duchenne muscular dystrophy, and funding for Duchenne research, congratulates Capricor Therapeutics on positive six-month results from its randomized CAP-1002 (cardiosphere-derived cells) Phase I/II HOPE clinical trial in Duchenne announced today.

Coalition Duchenne funded studies carried out by Eduardo Marbn MD, the director of the Cedars-Sinai Heart Institute, on cardiosphere-derived cells in Duchenne. This work was later licensed for commercial development by Capricor.

"We are excited that our work with Dr. Marbn is evolving and could become a treatment for Duchenne through Capricor's stellar efforts. It is the cardiomyopathy associated with Duchenne that causes us to lose so many boys and young men with Duchenne. Their hearts fail them when they still have so many dreams to be fulfilled. We must strengthen those hearts," said Catherine Jayasuriya, the founder and executive director of Coalition Duchenne.

The six-month results showed statistically-significant improvements in systolic thickening of the inferior wall of the heart and in the function of the middle and distal upper limb in patients treated with CAP-1002 as compared to control patients. In addition, differences observed in several other cardiac and skeletal muscle measures, including cardiac scar, were consistent with a treatment effect.

"These initial positive clinical results build upon a large body of preclinical data which illustrate CAP-1002's potential to broadly improve the condition of those afflicted by Duchenne, as they show that cardiosphere-derived cells exert salutary effects on cardiac and skeletal muscle," said Linda Marbn, PhD, Capricor's president and chief executive officer.

John L. Jefferies, MD, Professor of Pediatric Cardiology and Adult Cardiovascular Diseases at the University of Cincinnati and Director, Advanced Heart Failure and Cardiomyopathy, and Principal Investigator of the HOPE Trial, said, "In HOPE, we saw potential effects in both the heart and skeletal muscle that appear quite compelling in an exploratory trial."

Coalition Duchenne is hopeful that CAP-1002 will move forward rapidly and become a treatment for Duchenne. Such a development will realize a simple, but potentially game changing, lateral thought by a mother of a young man with Duchenne. Catherine's 24-year-old son Dusty Brandom has the cardiomyopathy exhibited by all boys and young men with Duchenne. In November 2011, Catherine read an Economist article titled "Repairing Broken Hearts" featuring the research of Dr. Eduardo Marbn and others. She immediately thought about Dusty's heart. Catherine's quest, both as a mother and a leader in the Duchenne community, became to convince the researchers to apply stem cell technology to Duchenne. She wrote to all of the researchers mentioned in the article and pushed her simple thesis that Duchenne related cardiomyopathy would be a good candidate for therapy. Only Dr. Eduardo Marbn responded.

About Coalition Duchenne

Coalition Duchenne was founded in 2011 to raise global awareness for Duchenne muscular dystrophy, to fund research, and to find a cure for Duchenne. Coalition Duchenne is a 501c3 non-profit corporation. Its vision is to change the outcome for boys and young men with Duchenne, to rapidly move forward to a new reality of longer, fulfilled lives, by funding the best opportunities for research and creating awareness.

Coalition Duchenne has several research initiatives that are making advances in potential cardiac and pulmonary treatments for sufferers of Duchenne muscular dystrophy.

Through its Duchenne Without Borders initiative, Coalition Duchenne is helping medically underserved boys and young men with Duchenne worldwide. For example, Coalition Duchenne provides Ambu Bags to Duchenne families in rural Borneo to help maintain pulmonary function.

Catherine Jayasuriya, the founder and executive director of Coalition Duchenne, produced and directed the award winning documentary Dusty's Trail: Summit of Borneo (2013) which has screened internationally and is included in the syllabus at teaching institutions worldwide.

For more information about Coalition Duchenne, visit http://www.coalitionduchenne.org.

About Duchenne muscular dystrophy

Duchenne muscular dystrophy is a progressive muscle wasting disease. It is the most common fatal genetic disease that affects children. Duchenne occurs in 1 in 3,500 male births, across all races, cultures and countries. Duchenne is caused by a defect in the gene that codes for the protein dystrophin. This is a vital protein that helps connect the muscle fiber to the cell membranes. Without dystrophin the muscle cells become unstable, are weakened and lose their functionality. Life expectancy ranges from the mid teenage years to age 30.

About CAP-1002

CAP-1002 consists of allogeneic cardiosphere-derived cells, or CDCs, a type of cardiac progenitor cell. CDCs have been the subject of over 100 peer-reviewed scientific publications and have been administered to approximately 140 human subjects across several clinical trials. CAP-1002 is currently being evaluated in the randomized, double-blind, placebo-controlled Phase II ALLSTAR Clinical Trial in adults who have suffered a large heart attack and in the Phase I/II HOPE Clinical Trial in boys and young men with Duchenne.

About Capricor Therapeutics

Capricor Therapeutics, Inc. is a clinical-stage biotechnology company developing first-in-class biological therapies for cardiac and other medical conditions. Capricor's lead candidate, CAP-1002, is a cell-based candidate currently in clinical development for the treatment of Duchenne muscular dystrophy, myocardial infarction (heart attack), and heart failure. Capricor is exploring the potential of CAP-2003, a cell-free, exosome-based candidate, to treat a variety of disorders. For more information, visit http://www.capricor.com.

For the original version on PRWeb visit: http://www.prweb.com/releases/2017/04/prweb14274775.htm

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Coalition Duchenne Congratulates Capricor Therapeutics on Positive Six-Month Results from its Randomized CAP ... - Benzinga

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The way to the Red Planet and other mysterious worlds is being inspired by the villainous Khan from the blockbuster films, according to new research.

The use of stem cell technology may mean the difference between life and death on any attempt to travel beyond Earth into the wilderness of space.

So the first person to walk on Mars is likely to be selected from the growing group of people whose parents took the step to store their child's stem cells at birth.

Stem cells are 'blank' cells that can be reprogrammed to turn into any other cell in the body, enabling the replacement of damaged cells.

More and more British parents, including TV presenter Natalie Pinkham and dancer Darcey Bussell, are paying more than 2,000 to freeze samples from their babies' umbilical cords at birth.

Stem cells are also found in bone marrow and some body tissue, but the procedure to harvest them from umbilical cords is less risky.

Adventurous Mars pioneers will have to be especially prepared for the dangerous trip, which could expose them to cancer and other diseases, through carefully researched gene therapy.

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We wince at the thought of genetically engineered humans

Mark Hall

Mark Hall, spokesperson for the UK's leading stem cell storage and diagnostics company StemProtect, said: "We wince at the thought of genetically engineered humans.

"And we are not going to create a Khan from Star Trek specifically to get to another planet. Getting humans to Mars and beyond will be both expensive and dangerous.

"But the scientific by-products - such as huge leaps in stem cell medicine - will benefit humanity for centuries to come."

Genetic engineering has featured in two Star Trek movies, and a number of TV episodes.

IG

1 of 14

This still image strikes an uncanny resemblance to a figure of a woman

Khan, who appeared in Space Seed and Star Trek II: The Wrath of Khan, was modified to make him stronger and to give him greater stamina and intellectual capacity than a regular human.

Mr Hall said: "The first human to walk on Mars may not even be born yet - but that's an advantage."

StemProtect believes advanced medical techniques will be required to cope with the rigours of interplanetary space.

While a trip to Mars may appear "just around the corner" in galactic terms, it is highly possible exposure to radiation along the way could lead to the astronauts developing leukaemia and other cancers even before they arrived.

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This means future travellers will have to be 'immunised' before they leave Earth.

Mr Hall said: "There was an article in The Times suggesting elephants would make ideal Martian travellers because they'd be largely immune to the radiation.

"But those laughing at the ridiculous sounding headline completely missed the point - the fact is scientists are already working on ways of getting humans there and back alive."

Recent research has shown radiation in deep space increase the risk of leukaemia while long term exposure to micro gravity may leave astronauts open to infection.

The three year round trip to Mars would affect humans at the stem cell level, leaving them with a drastically lowered immune system, NASA funded scientists say.

And NASA's own findings say stem cells may be crucial to the future of space travel, particularly how they respond in a low gravity environment.

One study showed stem cells flown in space and then cultured back on Earth had greater ability to self renew and generate any cell type, changing more easily into specialised heart muscle cells, for instance.

Mr Hall said an astronaut will have to be prepared for the journey "quite literally at the stem cell level."

He explained: "That means working with the best and most effective stem cells available to the patient - those harvested from the umbilical cord at birth."

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The therapies required to 'immunise' humans to space travel are still being researched.

And with most space based science, it can only mean huge benefits to mankind back down on Earth when it comes to fighting otherwise deadly conditions and diseases.

Stem cells have the ability to treat a potentially infinite range of illnesses and diseases.

Stem cell therapy is already being used all over the world to treat some cancers and stroke victims - and there is fast progress being made in many other areas, including Parkinson's and Alzheimer's disease.

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SUPERHUMANS: Mars 'will be colonised by genetically engineered Star Trek-style beings' - Express.co.uk

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In your biology class, you may have learned that lungs help us breathe while bone marrow, found in flat bones such as the hip bone, produces red and white blood cells through a process called hematopoiesis. Now, a new study conducted by University of California, San Francisco (UCSF) researchers has discovered that in addition to being a crucial part of our respiratory system, lungs also play a major role in the production and storage of blood cells.

Like many medical breakthroughs, the scientists stumbled upon this discovery by accident. The team, led by Professor Mark R. Looney, was trying to observe how platelets (cells that form clots to stop bleeding) circulating in the lungs interact with the immune system in mice. To trace the cells path, the rodents had been genetically modified so that the platelets appeared a glowing green.

To the scientists' astonishment, the lungs were filled with megakaryocytes the cells responsible for producing platelets. Though experts have always known of the existence of these cells inside the lungs, the numbers had been believed to be tiny. Emma Lefranais, who co-wrote the study, says, "When we discovered this massive population of megakaryocytes that appeared to be living in the lung, we realized we had to follow this up."

Further examination revealed that the megakaryocytes in the lungs were producing over 10 million platelets, or more than half the total platelets, produced by a mouse, every hour. The researchers also noticed the large population (1 million per mouse lung) of blood stem cells (which produce red blood cells) as well as megakaryocyte progenitor cells (which generate megakaryocyte cells) on the periphery of the lungs. Looney says, To our knowledge, this is the first description of blood progenitors resident in the lung.

To investigate the significance, the scientists conducted three studies. First, they transplanted lungs from normal mice into the genetically engineered ones to see how the blood stem cells move throughout the body. By following the fluorescent cells, they discovered that megakaryocytes originate in the bone marrow but migrate to the lungs to produce platelets.

To test the practical applications of this discovery and see if it would be useful in the treatment of disorders like lung inflammation, Looneys team injected the fluorescent megakaryocyte progenitor cells into mice with low platelet counts. To their delight, the transplanted cells got to work immediately, restoring the platelet count to normal levels within a short time. What was even more encouraging is that the effect lasted for several months.

Finally, the researchers transplanted healthy lungs in which all the cells had been fluorescently tagged into mice whose bone marrow was not producing blood cells or platelets. The researchers found that the glowing green megakaryocyte progenitor cells instantly migrated from the lungs to the bone marrow, where they helped to produce platelets and other critical blood components, like neutrophils, B cells, and T cells.

While the scientists, who published their findings in the journal Nature on March 22, 2017, still need to test if human lungs are as effective, the findings are being hailed as a major breakthrough. Traci Mondoro from the US National Heart, Lung, and Blood Institute, says, "Looney and his team have disrupted some traditional ideas about the pulmonary role in platelet-related hematopoiesis, paving the way for further scientific exploration of this integrated biology."

Resources: newatlas.com, UCSF.edu

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Lungs Don't Just Help Us Breathe They Produce Blood, Too - DOGOnews

April 20, 2017 07:00 ET | Source: David Steenblock, D.O. Inc.

SAN CLEMENTE, Calif., April 20, 2017 (GLOBE NEWSWIRE) -- David Steenblock, an osteopathic physician based in San Clemente, CA, uses stem cells and other therapies to achieve significant and improved lifestyle outcomes for many stroke patients.

Many of our cases have demonstrated improved mobility for stroke victims who received stem cell therapy, along with other therapies, including chelation and hyperbaric oxygen, says Dr. Steenblock.

One patient who suffered a stroke several years ago, came to Dr. Steenblocks clinic to undergo the full stroke program. This included EDTA chelation, a procedure that removes heavy metals from the blood, and hyperbaric oxygen therapy, along with stem cells from his bone marrow to effect healing and restoration.

After having the bone marrow stem cells, the patients eyesight improved, and both of his knees, which hadnt been working well, were back to functioning almost normally. In addition, his hip joint went back to normal function and he believes his balance when walking has improved tremendously.

The EDTA Chelation Therapy, a treatment used to remove heavy metals from the blood, was used with hyperbaric oxygen, which can lead to significant neurologic improvements for stroke patients.

Dr. David Steenblock is a leading-edge physician in many fields of medicine, from stroke care, to acute brain trauma, to generative and cell-based medicine in the treatment of ALS, Cerebral Palsy and other chronic and degenerative diseases. For more information about Dr. Steenblocks work in stem cell therapies, visit http://www.stemcellmd.org

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David Steenblock, D.O., Uses Stem Cells and Other Therapies for Stroke Patients - GlobeNewswire (press release)

WDTN	Ryan Custer may join stem cell study WDTN CINCINNATI, Ohio (WDTN) The Wright State basketball player who injured his spinal cord during an accident at a party this month is getting some much needed good news. According to a post on Facebook, Ryan Custer might be participating in a medical ...

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NEW YORK--(BUSINESS WIRE)--

DKMS, the international non-profit leading the fight against blood cancer, teamed with the film industry and launched #CastingforaHero, a campaign designed to raise awareness about diversity in bone marrow donation and to urge more people to swab their cheeks and register as potential lifesaving bone marrow/blood stem cell donors. The campaign was first launched by actor/producer Vin Diesel, has been supported on social media by Fast franchise players Tyrese Gibson, Cris Bridges (Ludacris), Don Omar and the brother of late actor Paul Walker, Cody Walker, and will be joined by other cast members to support the campaign this month. It has also garnered support by a number of celebrities including Guardians of the Galaxy actress, Zoe Saldana and Larenz Tate. Tate appeared on the television show POWER, alongside Charlie Murphy, who passed away earlier this month from complications related to blood cancer.

This Smart News Release features multimedia. View the full release here: http://www.businesswire.com/news/home/20170417005737/en/

Diesel launched the campaign on his Instagram (@vindiesel) posting, Today, premiere day (April 8), I am proud to launch#CastingforaHero- a campaign to save lives by increasing the multicultural community's presence in the worldwide bone marrow registries.

The campaign was conceived by DKMS through a partnership with Samantha Vincent, (Executive Producer, the Fate of the Furious) and Frank E. Flowers (Director, Haven) after they lost a family member to leukemia and became aware of the overwhelming odds faced by minorities and those of mixed race backgrounds of finding an unrelated match due to being underrepresented on the registry. Of all donors registered only 6% are African American, 9% are Hispanic/Latino, 6% are Asians, and 4% are Mixed Race.

In partnership with the community, one of the nations premier cross cultural creative advertising agencies, the #castingforahero concept was developed and executed through social and experiential channels leveraging key influencers with the goal of raising awareness and activating younger donors. The campaign was launched with the support of Universal Pictures, Saturday, April 8 during the Fate of the Furious #F8 premiere at Radio City Music Hall in NYC, with #castingforahero photo booths present at the F8premiere after party which gave VIP guests an opportunity to register.

In the companion video, written and directed by Flowers and produced by Andrew Molina, Anne McCarthy (Casting Director, Furious 7) and her associates audition real-life unknown actors for a hero role where they are asked to cold-read scripts about the lack of diversity on the bone marrow registry. The actors become emotional upon learning the scripts are in fact about themselves and their own chances to beat a disease like leukemia if there are not enough potential donors registered who share the same ancestry. The video calls for more trueheroes potential bone marrow/blood stem cell donors to join the effort to fight blood cancer and help find more matches for people of all ethnic backgrounds.

The newly launched website, castingforahero.com, allows people to create their own casting photo with custom skins representing unique identities and share on their social media platforms, while directing them to register with DKMS as a potential lifesaving donor.

Each year thousands of people lose the fight against blood cancer because they are unable to find their hero: a lifesaving bone marrow match, said Katharina Harf, co-founder of DKMS US. #CastingforaHero will help bring attention to the need for more diversity among potential bone marrow donors. By registering to become a DKMS donor, you can change the odds and become a life-saving hero yourself.

For more information about #CastingforaHero, visit http://www.castingforahero.com. To learn more about DKMS or register as a potential lifesaving donor, please visit http://www.dkms.org/register or @dkms.us.

About DKMS

DKMS is an international nonprofit organization dedicated to eradicating blood cancers like Leukemia and other blood-related illnesses inspiring both men and women around the world to register as bone marrow and blood stem cell donors. DKMS is providing patients with a second chance at life, working closely with families from diagnosis to transplant and beyond. The donor journey begins with a swab of the cheek that takes less than 60 seconds and can be the action that leads to a lifesaving transplant. DKMS, originally founded in Germany in 1991 by Dr. Peter Harf, has organizations in Poland, Spain, the United Kingdom and the United States. The U.S. office was started in 2004. Globally, DKMS has registered more than 7.2 million people. To join the fight against blood cancer or for more information, please go to dkms.org or @DKMS.us.

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DKMS Creates Celebrity Driven #Castingforahero Campaign - Yahoo Finance

BY: DUSTIN BATTY

Stem cell research is a controversial topic that is often vilified in the minds of the general public. This is in part because of the vast mainstream media coverage of the debates surrounding the use of embryonic stem cells, and their tendency to refer to the issue simply as the stem cell controversy rather than specifying that the problematic stem cells are those harvested from embryos.

Embryonic stem cells aside, though, there is still some discussion in bioethical circles about the harvesting of stem cells from bone marrow and even from skin. According to a study exploring an alternative method of obtaining stem cells, the debates surrounding the extraction of stem cells from even a mildly invasive procedure such as a skin biopsy are particularly relevant when one is procuring cells from vulnerable populations, such as children and individuals with intellectual disability. The study was undertaken to prove the viability of a non-invasive method of procuring stem cells from individuals with Down syndrome.

The method used by the researchers was surprisingly successful. They managed to extract cells from urine samples that were able to become induced pluripotent stem cells (iPSC), which means that the cells were altered so they could act like stem cells. Notably, the iPSCs obtained from the urine samples were superior to those harvested from skin biopsies and other methods because theyd had no exposure to ultraviolet light, and thus their DNA was generally undamaged.

Perhaps the most significant advantage that iPSCs from urine samples have over other methods is their completely non-invasive nature. This is particularly true when collecting stem cells from individuals with Down syndrome; in the past, a significant percentage of such individuals or their parents or guardians have refused to go forward with skin biopsies, limiting the availability of material for developing treatment methods. Research ethics boards have also been known to prevent the wide-scale use of skin biopsies in individuals with DS [Down syndrome]. This new method is expected to relieve the anxieties of the individuals involved, and should be easily accepted by ethics boards as well.

The researchers expect that the use of this method will improve both the quality of cells used and the quantity available to be studied. This increased availability is important to the efficient continuation of research into treatments for Down syndrome. Although such research begins with the use of lab mice to test the viability of new treatment methods, mouse physiology is so much simpler than that of humans that such tests arent sufficient. Eventually, the treatment needs to be tested on human cells. Stem cells are particularly useful for these kinds of tests because they are able to grow into a variety of different cells, which can be tested with the treatment individually.

The researchers conclude with the assurance that the techniques they implemented could be useful not only for research into Down syndrome, but also in the study of other neurodevelopmental and neurodegenerative disorders.

Providing better quality cells with increased participation and no ethical concerns, this new method of harvesting stem cells could be the answer that medical researchers were looking for.

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Stem cells can now be gathered from urine samples - The Plaid Zebra (blog)

The Cardiology Advisor (registration)

Theraputic Stem Cell-Sheet Transplantation May Improve ... The Cardiology Advisor (registration) A small phase I study identified positive benefits from utilizing stem cell-sheet transplantation as a therapy for ischemic and dilated cardiomyopathy. SCOTS Eye Stem Cell Study Exceeds Research GoalsMilTech all 23 news articles »

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Theraputic Stem Cell-Sheet Transplantation May Improve ... - The Cardiology Advisor (registration)

Ottawa Sun

Jonathan Pitre 'anxious' as he readies for his second transplant Thursday Ottawa Sun Boileau goes into surgery at the University of Minnesota Masonic Children's Hospital at 5:30 a.m. Thursday to have bone marrow drawn from her hip. Surgeons will bore two holes into her pelvis and withdraw the bone marrow, a material rich in stem cells; ... and more »

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Jonathan Pitre 'anxious' as he readies for his second transplant Thursday - Ottawa Sun

Virginia Western Community College will host a student-led Be the Match donor drive on April 19 from 10 a.m. to 2 p.m. in the courtyward between the Fralin Center and Business Science Building and the Pedestrian bridge. Through the drive, potential donors will learn if they could provide life-saving bone marrow or peripheral blood stem cell (PBSC) transplants.

At the drive, potential donors will complete a registration form with contact information, health information and a signed agreement to join the Be The Match Registry. To help you complete the form, bring along:

Personal identification (such as a driver's license or passport)

Contact information for two family members or friends who would know how to reach you in the future if your contact information changes

You will provide a swab of cheek cells to be tissue-typed. We will use the results to match you to patients

During the drive, an individual who has battled leukemia and received a stem cell transplant will speak to perspective donors on the importance of donation. Please join us to learn how you could help those in need.

For more information on Be the Match, visit http://www.bethematch.org.

Submitted by Josh Meyer

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VWCC to host bone marrow donor drive April 19 - Roanoke Times

April 6, 2017 A mouse heart section showing human progenitor cells that formed functional human blood vessels. Purple color signifies human blood vessels, red staining signifies the blood vessels of the mouse that received the human cell implants. Credit: Jalees Rehman.

Researchers from the University of Illinois at Chicago have identified a molecular switch that converts skin cells into cells that make up blood vessels, which could ultimately be used to repair damaged vessels in patients with heart disease or to engineer new vasculature in the lab. The technique, which boosts levels of an enzyme that keeps cells young, may also circumvent the usual aging that cells undergo during the culturing process. Their findings are reported in the journal Circulation.

Scientists have many ways to convert one type of cell into another. One technique involves turning a mature cell into a "pluripotent" stem cellone that has the ability to become any type of celland then using chemical cocktails to coax it into maturing into the desired cell type. Other methods reprogram a cell so that it directly assumes a new identity, bypassing the stem-cell state.

In the last few years, scientists have begun to explore another method, a middle way, that can turn back the clock on skin cells so that they lose some of their mature cell identity and become more stem-like.

"They don't revert all the way back to a pluripotent stem cell, but instead turn into intermediate progenitor cells," says Dr. Jalees Rehman, associate professor of medicine and pharmacology at UIC, who led the team of researchers. Progenitor cells can be grown in large quantities sufficient for regenerative therapies. And unlike pluripotent stem cells, progenitor cells can only differentiate into a few different cell types. Rehman calls this method to produce new cells "partial de-differentiation."

Other groups have used this technique to produce progenitor cells that become blood vessel cells. But until now, researchers had not fully understood how the method worked, Rehman said.

"Without understanding the molecular processes, it is difficult for us to control or enhance the process in order to efficiently build new blood vessels," he said.

His group discovered that the progenitors could be converted into blood vessel cells or into red blood cells, depending on the level of a gene transcription factor called SOX17.

The researchers measured the levels of several genes important for blood vessel formation. They saw that as progenitor cells were differentiating into blood vessel cells, levels of the transcription factor SOX17 became elevated.

When they increased levels of SOX17 even more in the progenitor cells, they saw that differentiation into blood vessel cells was enhanced about five-fold. When they suppressed SOX17, the progenitor cells produced fewer endothelial cells and instead generated red blood cells.

"It makes a lot of sense that SOX17 is involved because it is abundant in developing embryos when blood vessels are forming," Rehman said.

When the researchers embedded the human progenitor cells into a gel and implanted the gels in mice, the cells organized into functional human blood vessels. Skin cells that had not undergone a conversion did not form blood vessels when similarly implanted.

When they implanted the progenitor cells into mice that had sustained heart damage from a heart attack, the implanted cells formed functional human blood vessels in the mouse heartsand even connected with existing mouse blood vessels to significantly improve heart function.

The human adult skin cells used by Rehman's team can easily be obtained by a simple skin biopsy.

"This means that one could generate patient-specific blood vessels or red blood cells for any individual person," Rehman said. Using such personalized cells reduces the risk of rejection, he said, because the implanted blood vessels would have the same genetic makeup as the recipient.

Rehman and his colleagues noticed something else about the progenitor cells - they had elevated levels of telomerase - the "anti-aging" enzyme that adds a cap, or telomere, to the ends of chromosomes. As the caps wear away a little bit each time a cell divides, they are believed to contribute to aging in cells, whether in the body or growing in culture in the laboratory.

"The increase in telomerase we see in the progenitor cells could be an added benefit of using this partial de-differentiation technique for the production of new blood vessels for patients with cardiac disease, especially for older patients," Rehman said. "Their cells may already have shortened telomeres due to their advanced age. The process of converting and expanding these cells in the lab could make them age even further and impair their long-term function. But if the cells have elevated levels of telomerase, the cells are at lower risk of premature aging."

While telomerase has benefits, the enzyme is also found in extremely high levels in cancer cells, where it keeps cell division in overdrive.

"We were concerned about the risk of tumor formation," Rehman said, but the researchers didn't observe any in these experiments. "But to truly determine the efficacy and safety of these cells for humans, one needs to study them over even longer time periods in larger animals."

Explore further: Adult stem cells help build human blood vessels in engineered tissues

More information: Lianghui Zhang et al, SOX17 Regulates Conversion of Human Fibroblasts into Endothelial Cells and Erythroblasts via De-Differentiation into CD34Progenitor Cells, Circulation (2017). DOI: 10.1161/CIRCULATIONAHA.116.025722

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Turning skin cells into blood vessel cells while keeping them young - Medical Xpress

Yesterday we highlighted the GTCbio Stem Cell Summit in Boston. Another company we've been closely following is also represented at the conference, Asterias Biotherapeutics (NYSE: AST). Asterias is developing therapies based on pluripotent stem cells to address significant unmet medical needs in neurology and oncology. According to Boston Children's Hospital, pluripotent stem cells are the body's master cells, potentially able to produce any cell or tissue the body needs to repair itself. Like all stem cells, pluripotent stem cells are also able to self-renew, meaning they can perpetually create more copies of themselves.

The company draws its name from the common starfish, Asterias rubens, which has the ability to regenerate lost limbs even in adulthood. The company's lead product candidate, AST-OPC1, is currently in a Phase 1/2a dose ranging trial for spinal cord injury. As Patrick Cox discussed two months ago, Asterias' clinical trial has been generating significant interest.

In the company's earnings call last week, CEO Steve Cartt highlighted the ongoing clinical and corporate progress:

Source: Asterias Biotherapeutics Corporate Presentation, Jan. 24, 2017

We note that the company's cash position is much better than it was a year ago. As shown in the chart above, Asterias' spinal cord trial is funded by a grant from the California Institute of Regenerative Medicine (CIRM), which has certainly eased the burden. As of the end of February 2017, the company's cash and cash equivalents totaled $19.9 million and combined with its available-for-sale securities (mostly BioTime stock - see below*) totaled $33.5 million. Ryan Chavez, CFO, said, "We believe we currently have sufficient capital to fund operations through at least the first quarter of 2018."

* Readers may recall that Asterias was spun out of BioTime (NYSE: BTX) in 2014. BioTime remains the dominant shareholder, controlling 47% of the outstanding common and three out of the nine board seats (with a fourth board seat occupied by Broadwood Capital, BioTime's largest shareholder). Also, as mentioned above, Asterias holds about $13 million worth of BioTime stock. While the kids all seem to be playing well in the sandbox, this close relationship merits watching.

Jane Lebkowski, Asterias' President of Research and Development, was part of an immunotherapy roundtable at the Stem Cell Summit yesterday, discussing the company's research in therapeutics for lung cancer. Today, April 6th, at 10:40am ET, Dr. Michael West, CEO of BioTime, will deliver the conference keynote address entitled "The Promise of Pluripotency in the Manufacture of Advanced Cell-Based Therapeutics" - which sounds like a direct advertorial for a key BioTime asset - Asterias Biotherapeutics.

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Asterias (AST): Blazing the Stem Cell Trail for Spinal Cord Injury - Equities.com