Daiichi Sankyo Company has signed an investment contract with Cuorips Inc., an Osaka University spin-off venture to receive an option right concerning the worldwide commercialization of iPS-derived cardiomyocyte (iPS-CM) sheet developed by Cuorips.

The iPS-CM sheet is an allogeneic cell therapy product consisting of cardiomyocyte derived from human iPS cells. Its transplantation is expected to provide improvement of cardiac function and amelioration of heart failure and become a new treatment option for patients with severe heart failure, who have no remedies other than heart transplantation or artificial heart implantation.

Based on the cutting-edge cell therapy research targeting heart diseases, the team at the Department of Cardiovascular Surgery, Osaka University Graduate School of Medicine, led by Professor Yoshiki Sawa, has been working on the iPS-CM research and development by participating in the Research Center Network for Realization of Regenerative Medicine, which is run by the Japan Agency for Medical Research and Development (AMED). They are currently preparing for clinical research as well as investigator initiated clinical study.

Cuorips is an Osaka University spin-off venture founded to develop and commercialize iPS-CM sheets based on the research data and technologies developed by the university.

Daiichi Sankyo Group has been conducting research on iPS cell-derived cardiomyocyte and their production, and is currently working on the efficient production process capable for commercial supply.

Daiichi Sankyo and Cuorips are aiming to commercialize iPS-CM sheets as a pioneering treatment for severe heart failure. iPS cells are capable of almost unlimited proliferation and differentiation into any organ, and are expected to be used in the field of cell therapy. There are two types of cell therapy: autologous therapy where the patients own cells are collected, cultured and processed, and allogeneic therapy where a donors cells are collected, cultured and processed.

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Daiichi Sankyo signs Investment contract with Cuorips to commercialize iPS-derived cardiomyocyte sheet - pharmabiz.com

Nalini Ambady, the first Indian-American woman to teach psychology at three major universities in the U.S., died in 2013 due to leukaemia when she was just 54.

For the medical fraternity in Kerala, her native place, it turned the spotlight on the lack of awareness of stem cell transplant, which could have saved her life.

Four years down the lane, doctors say the situation has changed only marginally, as many patients who require the treatment have not been able to do it because of high expenses, lack of matching donors, and lack of facilities at hospitals.

Doctors note that stem cell transplant is being proposed as an effective treatment for cancers such as leukaemia and lymphoma, and primary immune deficiency disorders. Stem cells do not develop normally in such patients and it affects the blood cells that they make. By a transplant, the patient gets new stem cells that can make new and healthy blood cells. Earlier, stem cells were collected from the bone-marrow. Now, it is being collected from blood cells.

Neeraj Sidharthan, bone marrow transplant physician at Amrita Institute of Medical Sciences, Kochi, told The Hindu that in Prof. Ambadys case, though matching donors were found, they had all dropped out.

Lack of awareness is still a major issue though there are some positive signs. In some cases, because of lack of infrastructure, cancer cases are not being diagnosed early and treatment is delayed too, he said.

Ajith Kumar V.T., professor, department of paediatrics, Government Medical College, Manjeri, said donors could not be found often from the same families because of the nuclear family system.

There are not many places where you can match the human leukocyte antigen (HLA) typing with donors. Another problem is the lack of stem cell registries in the State from where matching unrelated donors could be found.

Even if doctors suggest a stem cell transplant, many families dont opt for it because of the high cost involved. If the donor is from the same family, the cost is relatively low. But for unrelated donors, it is very high, Dr. Sidharthan said. The solution, Dr. Ajith Kumar said, was government intervention to set up HLA registries and bone marrow transplant centres. nestCare Foundation, a not-for-profit organisation based in the U.S., had recently approached us expressing interest to set up these facilities in the State. Talks are on, he said.

A.S. Jayanth

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Trust those cells to help cure cancer - The Hindu

Tom Parham remembers the time his Woodrow Wilson baseball team was playing Class AAA power Huntington East.

It was 1980, and the Flying Eagles were hosting the Pony Express at Harry Lewin Field. Not known to be a cavernous venue, the field lent itself to an offensive barrage and Huntington East was the last team standing.

A few weeks later, Parham led Woodrow to the state championship game and a rematch with the Express. Woodrow fell short again, but this time it was by the more purist-friendly score of 2-1.

It was then that Parham knew the Eagles needed a new field.

In stepped Doug Epling, Beckley businessman and community leader. He would later be known for refurbishing the old East Bank High School field for WVU Tech to use, as well as the construction of Linda K. Epling Stadium in Beckley, the home of the West Virginia Miners.

The latter, of course, bears the name of Epling's wife. The Tech field is named for Epling himself.The field he helped build on the Woodrow Wilson campus doesn't have an official name.

That will change Saturday.A ceremony will be held at 2 p.m. in the school cafeteria to officially rename the field for Thomas Parham.

The effort to honor the longtime coach was started by Sheila Brown.

"Words cannot describe how it feels," Parham said. "When Mrs. Brown started talking about it, I always told her, nah (modestly). I just thought, 'Let it go.' And finally she told me in April, 'Well, I'm going to the board. I'm going to ask them.' So she did and they told her what to do (at the next meeting)."

The Raleigh County School Board laid out a plan for Brown, and at the next meeting former coaches, colleagues and friends voiced their support.

Legendary boys basketball coach Dave Barksdale. State championship-winning football coach Pete Culicerto. Fellow New Hope Baptist Church member C.W. Claytor. Even Epling himself. They all showed up to see that Parham got the respect they feel he deserves.

"It was just touching to hear former coaches Coach Barksdale, Coach Culicerto, and I even heard from one of my coaching buddies from out of town, Ron Rose," Parham said. "He told Pete what (he wanted) to say. It was just touching, and a humbling experience."

Parham is being recognized for a career that spanned nearly three decades. He was hired as a biology teacher by Ross Hutchens before the start of the 1974-75 academic year.

"He said, 'I need a good biology teacher. I can get a coach anywhere,'" Parham said, laughing.

His first season as head baseball coach was 1975, and he remained there until his retirement in 2000. Along the way, his teams rolled up over 200 wins and appeared in the state tournament five times. Two of those trips resulted in runner-up finishes the 1980 meeting with Huntington East, and in 1983 against Martinsburg.

And the list of star players Parham coached seems endless Chuck Tate, Andy "Bam Bam" Wakefield, Larry Maiolo, Mason Basham, Larry Hickman, Joe Joe Maiolo, Larry Pat Farley, Phil Culicerto, Tim Epling, Phil Lane, Ronnie Fama, John O'Dell.

There were many others, and many of themare members of the Woodrow Wilson Baseball Hall of Fame.

"I was fortunate I came across some good ball players," Parham said. "You don't like to toot your own horn, but like a fella said, we put Woodrow Wilson baseball on the map."

Another was Ronnie Scott, who went on to work for NASA in Florida before returning to Beckley in 2010. Sadly, he passed away in May at age 59.

"He wanted to see baseball dominant again like it was when he played," Parham said.

When Parham retired from baseball in 2000 he stayed on as a biology teacher for one more year it was the emphatic end of an era at the school. Not only did Parham retire, but Culicerto retired after the 1999 football season, and Barksdale left the bench just months before Parham to take a coaching job in Aiken, S.C.

"Indeed it was," said Parham, now 74. "I enjoyed working with Coach Culicerto (as an assistant football coach). He was a great football coach, and he was a baseball supporter. He had seven sons play baseball for me. Three of them played in the state tournament."

After his retirement, Parham's was a familiar face in the stands at Woodrow baseball games. But in 2009, his ability to be a spectator slowed down when it was discovered that he had cancer.

Parham was diagnosed with multiple myeloma, a blood cancer that develops in the plasma cells located in bone marrow. The cancer did eventually go into remission, but Parham was still getting checkups when something told him he needed to go to Johns Hopkins in Baltimore.

It was there that he was introduced to autologous stem cell transplant. It's a procedure that involves collecting the patient's stem cells and following it up with high doses of chemotherapy or a combination of chemo and radiation. The process kills cancer cells while also killing blood-producing cells left in the bone marrow.

The collected stem cells are later transplanted back into the patient, allowing the marrow to produce new blood cells.

"I met a very interesting and a caring doctor up there, Dr. (Ivan) Borrello. He told me (about the transplant)," Parham said. "As a matter of fact, they have been doing this since 1980. He asked, 'What do you think about a stem cell transplant?' And I said yeah. Anything to get rid of this cancer.

"At that time my cancer was in remission, so he couldn't do anything. He said we would have to wait until it comes back. He hoped it didn't come back, but if it does ..."

It did, and in February he had the procedure performed.

"It came back in 2016, and when you're over 70 they don't usually do these things," Parham said. "But he felt like I was in good shape, which I think well, I know I am. I went through it, successful, no problems whatsoever.

"After teaching biology, I thought I knew some things. Now I know I know some things."

Just like baseball.

Email: gfauber@register-herald.com and follow on Twitter @GaryFauber

Excerpt from:
Woodrow Wilson baseball field to be renamed for Tom Parham - Beckley Register-Herald

What causes the body to age?

The Greek Philosopher Aristotle thought it was the hearta hot, dry organ at the seat of intelligence, motion and sensation.

Fast-forward a few centuries, and the brain has overthrown the heart as master of thought. But its control over bodily agingif anywas unclear. Because each organ has its own pool of stem cells to replenish aged tissue, scientists have long thought that the body has multiple aging clocks running concurrently.

As it turns out, thats not quite right.

This week, a study published in Nature threw a wrench into the classical theory of aging. In a technical tour-de-force, a team led by Dr. Dongsheng Cai from the Albert Einstein College of Medicine pinpointed a critical source of aging to a small group of stem cells within the hypothalamusan ancient brain region that controls bodily functions such as temperature and appetite.

Like fountains of youth, these stem cells release tiny fatty bubbles filled with mixtures of small biological molecules called microRNAs. With age, these cells die out, and the animals muscle, skin and brain function declines.

However, when the team transplanted these stem cells from young animals into a middle-aged one, they slowed aging. The recipient mice were smarter, more sociable and had better muscle function. Andget thisthey also lived 10 to 15 percent longer than mice transplanted with other cell types.

To Dr. David Sinclair, an aging expert at Harvard Medical School, the findings represent a breakthrough in aging research.

The brain controls aging, he says. I can see a day when we are implanted with stem cells or treated with stem cell RNAs that improve our health and extend our lives.

Its incredible to think that a tiny group of cells in one brain region could be the key to aging.

But to Cai, there are plenty of examples throughout evolution that support the theory. Experimentally changing a few of the 302 neurons in the nematode worm C. elegans is often sufficient for changing its lifespan, he says.

Of course, a mammalian brain is much more complicated than a simple worm. To narrow the problem down, Cai decided to zero in on the hypothalamus.

The hypothalamus has a classical function to regulate the whole bodys physiology, he says, so theres a natural logic for us to reason that the hypothalamus might be involved in aging, which was never studied before.

Even so, it was a high-risk bet. The hippocampusbecause of its importance in maintaining memory with ageis the most popular research target. And while the hypothalamus was previously somehow linked to aging, no one knew how.

Cais bet paid off. In a groundbreaking paper published in 2013, he found that a molecule called NF-kappaB increased in the hypothalamus as an animal grew older. Zap out NF-kappaB activity in mice, and they showed much fewer age-related symptoms as they grew older.

But heres the kicker: the effects werent limited to brain function. The animals also better preserved their muscle strength, skin thickness, bone and tendon integrity. In other words, by changing molecules in a single part of the brain, the team slowed down signs of aging in the peripheral body.

But to Cai, he had only solved part of the aging puzzle.

At the cellular level, a cornucopia of factors control aging. There is no the key to aging, no single molecule or pathway that dominates the process. Inflammation, which NF-kappaB regulates, is a big contributor. As is the length of telomeres, the protective end caps of DNA, and of course, stem cells.

Compared to other tissues in the body, stem cells in the brain are extremely rare. So imagine Cais excitement when, just a few years ago, he learned that the hypothalamus contains these nuggets of youth.

Now we can put the two threads together, and ask whether stem cells in the hypothalamus somehow regulate aging, he says.

In the first series of experiments, his team found that these stem cells, which line a V-shaped region of the hypothalamus, disappear as an animal ages.

To see whether declined stem cell function contributes to aging, rather as a result of old age, the researchers used two different types of toxins to wipe out 70 percent of stem cells while keeping mature neurons intact.

The results were striking. Over a period of four months, these mice aged much faster: their muscle endurance, coordination and treadmill performance tanked. Mentally, they had trouble navigating a water maze and showed less interest in socializing with other mice.

All of these physiological changes reflected an acceleration in aging, Cai and team concluded in their article.

And the consequences were dire: the animals died months earlier than similar transgenic animals without the toxin treatment.

If the decline in stem cell function is to blame for aging, then resupplying the aged brain with a fresh source of stem cells should be able to reinvigorate the animal.

To test this idea, the team isolated stem cells from the hippocampus of newborn mice, and tinkered with their genes so that they were more resilient to inflammation.

We know the aged hypothalamus has more inflammation and that hurts stem cells, so this step was necessary, explained the authors.

When transplanted into middle-aged mice, they showed better cognitive and muscular function four months later. Whats more, they lived, on average, 10 percent longer than mice transplanted with other cell types. For a human, that means extending an 85-year life expectancy into 93. Not too shabby.

But the best was yet to come. How can a few cells have such a remarkable effect on aging? In a series of follow-up experiments, the team found that the pool of biological molecules called microRNAs was to thank.

microRNAs are tiny molecules with gigantic influence. They come in various flavors, bearing rather unimaginative names like 106a-5p, 20a-5p and so on. But because they can act on multiple genes at the same time, they pack a big punch. A single type of microRNA can change the way a cell workswhether it activates certain signaling pathways or makes certain proteins, for example.

While most cells make microRNAs, Cai found that the hypothalamus stem cells have a unique, very strong ability to pack these molecules up into blobs of membrane and shoot them out like a bubble gun.

Once outside the cell, the microRNAs go on a fantastic voyage across the brain and body, where they tweak the biology of other tissues.

In fact, when the team injected purified little bubbles of microRNAs into middle-aged mice, they also saw broad rejuvenating effects.

Cai explains: we dont know if the microRNAs are pumped out to directly affect the rest of the body, or if they first act on different areas of the brain, and the brain goes on to regulate aging in the body.

Even so, the aging field is intrigued.

According to Dr. Leonard Guarente, an aging biologist at MIT, the study could lead to new ways to develop anti-aging therapies.

Whats more, its possible the intervention could stack with other known rejuvenating methods, such as metformin, young blood or molecules that clean out malfunctioning cells.

Its possible that stem-cell therapy could boost the hypothalamus ability to regulate aging. However, scientists still need to know how stem cells link with the hypothalamus other main role, that is, releasing hormones.

Of course, injecting cells into the brain isnt a practical treatment. The team is now working hard to identify which of the thousands of types of microRNAs control aging and what exactly they do.

Then the goal is to validate those candidate anti-aging microRNAs in primates, and eventually, humans.

Of course humans are more complex. However, if the mechanism is fundamental, you might expect to see effects when an intervention is based on it, says Cai.

Stock Media provided by digitalreflections / Pond5

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Breakthrough Stem Cell Study Offers New Clues to Reversing Aging - Singularity Hub

FREMONT, Calif., Aug. 03, 2017 (GLOBE NEWSWIRE) -- Asterias Biotherapeutics, Inc. (NYSE MKT:AST), a biotechnology company pioneering the field of regenerative medicine, today announced that two additional clinical sites have opened to enroll subjects for the companys ongoing SCiStar Phase 1/2a clinical study of AST-OPC1 in complete cervical spinal cord injury (SCI). The additional clinical sites include: Thomas Jefferson University Hospital, in partnership with Magee Rehabilitation Hospital, in Philadelphia, PA; and UC San Diego Health in San Diego, CA. Asterias now has eight clinical sites throughout the country enrolling patients in the study.

We are excited about the clinical site openings at Thomas Jefferson University Hospital and UC San Diego Health, stated Dr. Edward Wirth III, Chief Medical Officer of Asterias Biotherapeutics. These sites provide additional geographical reach and previous experience with spinal cord injury trials to our SCiStar study. We have recently reported completion of enrollment in four out of five cohorts in our SCiStar study so we hope these institutions will also participate in a future, larger study of AST-OPC1.

Each of the two additional clinical sites is recognized in the treatment of SCI:

The two additional clinical sites join existing clinical sites for the SCiStar study at the Medical College of Wisconsin in Milwaukee, Shepherd Medical Center in Atlanta, University of Southern California (USC) jointly with Rancho Los Amigos National Rehabilitation Center in Los Angeles, Indiana University, Rush University Medical Center in Chicago and Santa Clara Valley Medical Center in San Jose jointly with Stanford University.

Asterias has completed enrollment and dosing in four of the five planned SCiStar study cohorts and enrolled twenty patients in the SCiStar study. Twenty-five patients have been administered AST-OPC1 after including patients from a previous Phase 1 safety trial and results-to-date continue to support the safety of AST-OPC1. In June 2017, Asterias reported 9 month data from the AIS-A 10 million cell cohort that showed improvements in arm, hand and finger function observed at 3-months and 6-months following administration of AST-OPC1 were confirmed and in some patients further increased at 9-months. The company intends to complete enrollment of the entire SCiStar study later this year, with multiple safety and efficacy readouts anticipated during the remainder of 2017 and 2018.

About the SCiStar Trial

The SCiStar trial is an open-label, single-arm trial testing three sequential escalating doses of AST-OPC1 administered at up to 20 million AST-OPC1 cells in as many as 35 patients with subacute motor complete (AIS-A or AIS-B) cervical (C-4 to C-7) SCI. These individuals have essentially lost all movement below their injury site and experience severe paralysis of the upper and lower limbs. AIS-A patients have lost all motor and sensory function below their injury site, while AIS-B patients have lost all motor function but may have retained some minimal sensory function below their injury site. AST-OPC1 is being administered 21 to 42 days post-injury. Patients will be followed by neurological exams and imaging procedures to assess the safety and activity of the product.

Asterias has received a Strategic Partnerships Award grant from the California Institute for Regenerative Medicine, which has provided $14.3 million of non-dilutive funding for the Phase 1/2a clinical trial and other product development activities for AST-OPC1.

Additional information on the Phase 1/2a trial, including trial sites, can be found at http://www.clinicaltrials.gov, using Identifier NCT02302157, and at the SCiStar Study Website (www.SCiStar-study.com).

About AST-OPC1

AST-OPC1, an oligodendrocyte progenitor population derived from human embryonic stem cells originally isolated in 1998, has been shown in animals and in vitro to have three potentially reparative functions that address the complex pathologies observed at the injury site of a spinal cord injury. These activities of AST-OPC1 include production of neurotrophic factors, stimulation of vascularization, and induction of remyelination of denuded axons, all of which are critical for survival, regrowth and conduction of nerve impulses through axons at the injury site.

In a previous Phase 1 clinical trial, five patients with neurologically complete, thoracic spinal cord injury were administered two million AST-OPC1 cells at the spinal cord injury site 7-14 days post-injury. Based on the results of this study, Asterias received clearance from FDA to progress testing of AST-OPC1 to patients with cervical spine injuries in the current SCiStar study, which represents the first targeted population for registration trials. Asterias has completed enrollment in the first four cohorts of this study. Results to date have continued to support the safety of AST-OPC1. Additionally, Asterias has recently reported results suggesting reduced cavitation and improved motor function in patients administered AST-OPC1 in the SCiStar trial.

About Asterias Biotherapeutics

Asterias Biotherapeutics, Inc. is a biotechnology company pioneering the field of regenerative medicine. The company's proprietary cell therapy programs are based on its pluripotent stem cell and immunotherapy platform technologies. Asterias is presently focused on advancing three clinical-stage programs which have the potential to address areas of very high unmet medical need in the fields of neurology and oncology. AST-OPC1 (oligodendrocyte progenitor cells) is currently in a Phase 1/2a dose escalation clinical trial in spinal cord injury. AST-VAC1 (antigen-presenting autologous dendritic cells) is undergoing continuing development by Asterias based on promising efficacy and safety data from a Phase 2 study in Acute Myeloid Leukemia (AML), with current efforts focused on streamlining and modernizing the manufacturing process. AST-VAC2 (antigen-presenting allogeneic dendritic cells) represents a second generation, allogeneic cancer immunotherapy. The company's research partner, Cancer Research UK, plans to begin a Phase 1/2a clinical trial of AST-VAC2 in non-small cell lung cancer in 2017. Additional information about Asterias can be found at http://www.asteriasbiotherapeutics.com.

FORWARD-LOOKING STATEMENTS

Statements pertaining to future financial and/or operating and/or clinical research results, future growth in research, technology, clinical development, and potential opportunities for Asterias, along with other statements about the future expectations, beliefs, goals, plans, or prospects expressed by management constitute forward-looking statements. Any statements that are not historical fact (including, but not limited to statements that contain words such as "will," "believes," "plans," "anticipates," "expects," "estimates") should also be considered to be forward-looking statements. Forward-looking statements involve risks and uncertainties, including, without limitation, risks inherent in the development and/or commercialization of potential products, uncertainty in the results of clinical trials or regulatory approvals, need and ability to obtain future capital, and maintenance of intellectual property rights. Actual results may differ materially from the results anticipated in these forward-looking statements and as such should be evaluated together with the many uncertainties that affect the businesses of Asterias, particularly those mentioned in the cautionary statements found in Asterias' filings with the Securities and Exchange Commission. Asterias disclaims any intent or obligation to update these forward-looking statements.

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Asterias Biotherapeutics Opens Two Additional Clinical Sites for ... - GlobeNewswire (press release)

School of Public Health (SPH) student Jake Maxon (Burnsville, MN) became interested in policy while working among microscopes and petri dishes. After getting his neuroscience degree from Brown University, Maxon researched spinal cord injuries, which involved stem cell research.

Policy around stem cells brought a different kind of challenge to our research, and I wanted to know more about how policies are created and implemented, he says.

So Maxon enrolled in SPHs Public Health Administration and Policy program, where hes been able to work with many Twin Cities policy organizations, including as a grant reviewer for the Ryan White HIV/AIDS Program and as a policy intern for Hennepin County.

But he wanted to understand policy on a federal level. So he applied to the White House Internship Program. After a six-month selection and vetting process, he was assigned to work alongside the three-person team in the Office of National AIDS Policy, which works to create an integrated approach to the prevention, care, and treatment of HIV/AIDS.

Weve advanced medical care for HIV/AIDS patients, so many people in our country forget that HIV is still an epidemic and its still a public health crisis, says Maxon. More than 4,300 residents in Hennepin County alone are currently living with HIV/AIDS.

While in Washington, D.C., Maxon continued working on his degree and now, nearly finished, finds himself in a new role as coordinator for Hennepin Countys HIV Positively Hennepin strategybased off of the national strategy he helped implement at the White House.

The vision is to create a county where all residents living with HIV/AIDS have healthy, vibrant lives, where there is equitable access to HIV prevention and care, and where there are no new HIV infections.

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A bold vision - UMN News

One week ago, MIT Technology Review reported that scientists at an Oregon-based lab had modified the DNA of human embryos using the gene-editing technique known as CRISPR. That was a first for the United States; until then, such a procedure had only ever been done in China.

The researchers, led by Shoukhrat Mitalipov from Oregon Health and Science University, had altered the gene behind an unspecified inherited disease in a number of one-cell embryos. These embryos werent allowed to develop for more than a few days, and there was never any intention to implant them into a womb. The story fueled another cycle of discussion about designer babies, and fears that a Gattaca-style world was just around the corner.

But the full details of the experiment, which are released today, show that the study is scientifically important but much less of a social inflection point than has been suggested. This has been widely reported as the dawn of the era of the designer baby, making it probably the fifth or sixth time people have reported that dawn, says Alta Charo, an expert on law and bioethics at the University of Wisconsin-Madison. And its not.

Given the persistent confusion around CRISPR and its implications, I've laid out exactly what the team did, and what it means.

Who did the experiments?

Shoukhrat Mitalipov is a Kazakhstani-born cell biologist with a history of breakthroughsand controversyin the stem cell field. He was the scientist to clone monkeys. He was the first to create human embryos by cloning adult cellsa move that could provide patients with an easy supply of personalized stem cells. He also pioneered a technique for creating embryos with genetic material from three biological parents, as a way of preventing a group of debilitating inherited diseases.

Although MIT Tech Review name-checked Mitalipov alone, the paper splits credit for the research between five collaborating teamsfour based in the United States, and one in South Korea.

What did they actually do?

The project effectively began with an elevator conversation between Mitalipov and his colleague Sanjiv Kaul. Mitalipov explained that he wanted to use CRISPR to correct a disease-causing gene in human embryos, and was trying to figure out which disease to focus on. Kaul, a cardiologist, told him about hypertrophic cardiomyopathy (HCM)an inherited heart disease thats commonly caused by mutations in a gene called MYBPC3. HCM is surprisingly common, affecting 1 in 500 adults. Many of them lead normal lives, but in some, the walls of their hearts can thicken and suddenly fail. For that reason, HCM is the commonest cause of sudden death in athletes. There really is no treatment, says Kaul. A number of drugs are being evaluated but they are all experimental, and they merely treat the symptoms. The team wanted to prevent HCM entirely by removing the underlying mutation.

They collected sperm from a man with HCM and used CRISPR to change his mutant gene into its normal healthy version, while simultaneously using the sperm to fertilize eggs that had been donated by female volunteers. In this way, they created embryos that were completely free of the mutation. The procedure was effective, and avoided some of the critical problems that have plagued past attempts to use CRISPR in human embryos.

Wait, other human embryos have been edited before?

There have been three attempts in China. The first twoin 2015 and 2016used non-viable embryos that could never have resulted in a live birth. The thirdannounced this Marchwas the first to use viable embryos that could theoretically have been implanted in a womb. All of these studies showed that CRISPR gene-editing, for all its hype, is still in its infancy.

The editing was imprecise. CRISPR is heralded for its precision, allowing scientists to edit particular genes of choice. But in practice, some of the Chinese researchers found worrying levels of off-target mutations, where CRISPR mistakenly cut other parts of the genome.

The editing was inefficient. The first Chinese team only managed to successfully edit a disease gene in 4 out of 86 embryos, and the second team fared even worse.

The editing was incomplete. Even in the successful cases, each embryo had a mix of modified and unmodified cells. This pattern, known as mosaicism, poses serious safety problems if gene-editing were ever to be used in practice. Doctors could end up implanting women with embryos that they thought were free of a disease-causing mutation, but were only partially free. The resulting person would still have many tissues and organs that carry those mutations, and might go on to develop symptoms.

What did the American team do differently?

The Chinese teams all used CRISPR to edit embryos at early stages of their development. By contrast, the Oregon researchers delivered the CRISPR components at the earliest possible pointminutes before fertilization. That neatly avoids the problem of mosaicism by ensuring that an embryo is edited from the very moment it is created. The team did this with 54 embryos and successfully edited the mutant MYBPC3 gene in 72 percent of them. In the other 28 percent, the editing didnt worka high failure rate, but far lower than in previous attempts. Better still, the team found no evidence of off-target mutations.

This is a big deal. Many scientists assumed that theyd have to do something more convoluted to avoid mosaicism. Theyd have to collect a patients cells, which theyd revert into stem cells, which theyd use to make sperm or eggs, which theyd edit using CRISPR. Thats a lot of extra steps, with more risks, says Alta Charo. If its possible to edit the embryo itself, thats a real advance. Perhaps for that reason, this is the first study to edit human embryos that was published in a top-tier scientific journalNature, which rejected some of the earlier Chinese papers.

Is this kind of research even legal?

Yes. In Western Europe, 15 countries out of 22 ban any attempts to change the human germ linea term referring to sperm, eggs, and other cells that can transmit genetic information to future generations. No such stance exists in the United States but the Food and Drug Administration will not fund research that makes such modifications. Separately, federal agencies like the National Institutes of Health are banned from funding research that ultimately destroys human embryos. But the Oregon team used non-federal money from their institutions, and donations from several small non-profits. No taxpayer money went into their work.

Why would you want to edit embryos at all?

Partly to learn more about ourselves. By using CRISPR to manipulate the genes of embryos, scientists can learn more about the earliest stages of human development, and about problems like infertility and miscarriages. Thats why biologist Kathy Niakan from the Crick Institute in London recently secured a license from a British regulator to use CRISPR on human embryos.

The Oregon team has more immediate goals in mind. Through their work, they hope to eventually give people with HCM the certainty that they would not pass on their condition to their children. If we had the freedom to do this, we could theoretically remove HCM in a generation, says Kaul. Thats the potential and we have to let the potential and reality meet someday.

In February, an expert committee convened by the U.S. National Academy of Sciences (and chaired by Charo) offered qualified support for germ-line editing. In a report, they said that such editing shouldnt be used to enhance healthy people, but could be permitted to treat or prevent disease and disability, provided certain criteria were met. The technique would need to become much safer and more efficient, and a stringent oversight system should be set in place. It should be an option of last resort for couples who have a serious genetic disease and have no other way of producing a healthy child. But remember that the Oregon team havent done anything even close to this yet. They just edited embryos for basic research purposesa use that the NAS report wholeheartedly endorsed.

How do people with HCM feel about this?

I reached out to an advocacy organization that raises awareness of HCM, but havent heard back. But John Jefferies, a cardiologist at Cincinnati Children's Hospital Medical Center, says, I think those caring for these patients would greatly welcome this move. The medical therapies we have for this disease are limited and do not reverse the cardiac [problems]. This offers a potential cure for the disease by avoiding it.

Arent there already other ways of doing that?

Yes, and therein lies the debate. A couple could opt for preimplantation genetic diagnosis (PGD), where their sperm and eggs are introduced in a lab, and the resulting embryos are genetically screened to find those that are free of disease genes. This technique already works well, so why bother with gene-editing at all? If one of the wannabe parents has a copy of an HCM-causing mutation, then half of the resulting embryos will carry that mutationand be discarded. But if Oregon team gets their technique working perfectly, then every embryo could be potentially implanted. Theyre not trying to supplant PGD. Theyre trying to bolster it.

But these days with IVF, the tendency is to put in one embryo at a time to avoid having twins or triplets, says Charo. If it doesnt work after a few times, youre less likely to succeed. So its not clear to me how relevant this is for preventing genetic disease. Mitalipov disagrees. IVF is not efficient and with this procedure, we hope patients will be able to become pregnant on just one cycle, he says. He also he sees this as a moral issue. You have no right to throw away 50 percent of these embryos when you can correct them. Its very 19th-century. Some people say that our work is ethically wrong but I think it is ethically right.

Does the editing approach have limitations?

Yes, and they are important ones. CRISPR works by cutting DNA at a precise point. A cell then uses a matching piece of DNA as a template for repairing the cut. Its like tearing a misprinted page from a book and using a pristine edition to fill out the missing text. Mitalipovs team offered the embryos a pristine copy of the MYBPC3 gene to be used in the repair process. But to their surprise, the embryos largely ignored this gift. Instead, they used the healthy copy of the gene from the egg to repair the CRISPR-sliced mutant version from the sperm. That means that this technique would not work if both parents have HCM. If both pass a mutant version of MYBPC3 to an embryo, theres no healthy copy to use as a template. We still need to figure out how to correct those, says Mitalipov.

When can we expect such editing to be commonplace?

Not for a while. The technique would need to be refined, tested on non-human primates, and shown to be safe. The safety studies would likely take 10 to 15 years before FDA or other regulators would even consider allowing clinical trials, wrote bioethicist Hank Greely in a piece for Scientific American. The Mitalipov research could mean that moment is 9 years and 10 months away instead of 10 years, but it is not close. In the meantime, Kaul says, Well get the method to perfection so that when its possible to use it in a clinical trial, we can.

Isnt this a slippery slope toward making designer babies?

In terms of avoiding genetic diseases, its not conceptually different from PGD, which is already widely used. The bigger worry is that gene-editing could be used to make people stronger, smarter, or taller, paving the way for a new eugenics, and widening the already substantial gaps between the wealthy and poor. But many geneticists believe that such a future is fundamentally unlikely because complex traits like height and intelligence are the work of hundreds or thousands of genes, each of which have a tiny effect. The prospect of editing them all is implausible. And since genes are so thoroughly interconnected, it may be impossible to edit one particular trait without also affecting many others.

Theres the worry that this could be used for enhancement, so society has to draw a line, says Mitalipov. But this is pretty complex technology and it wouldnt be hard to regulate it.

Wait, havent I read about DIY gene-editors, who are using CRISPR in their basement labs?

Yes, but none of those people are using the technique to edit human embryos. Mitalipovs work is essentially a form of IVF. Its not simple IVF either, he says. Everything needs to be done exactly the way we did it. Youd need to do a biopsy with every embryo to screen for off-target mutations. You cant do it at home.

So, this isnt the start of Gattaca?

I doubt it.

Brave New World?

Unlikely.

Does this discovery have any social importance at all?

Its not so much about designer babies as it is about geographical location, says Charo. Its happening in the United States, and everything here around embryo research has high sensitivity. She and others worry that the early report about the study, before the actual details were available for scrutiny, could lead to unnecessary panic. Panic reactions often lead to panic-driven policy ... which is usually bad policy, wrote Greeley.

Read more here:
The Designer Baby Era Is Not Upon Us - The Atlantic

Some big scientific breakthroughs are set to take place this year.

And fortunes could be made with a few off-the-radar biotech pioneers.

What I am talking about are the handful of small biotech companies applying regenerative medicine platforms to degenerative diseases and ailments like paralysis.

These degenerative diseases have always been tough to treat, and few therapies are available. Thats not surprising, since weve lacked until recently the ability to create healthy and functioning human cells to replace ones lost in the body to injury and disease.

Now, though, a handful of breakthrough companies are aiming to correct that lack with new pluripotent stem cell technology.

Stem cells have the ability to reproduce and change into functional cells and tissues. And pluripotent stem cells are the most potent. They can turn into any cell type in the human body. Furthermore, they can divide and reproduce without end.

This makes them an ideal starting point for manufacturing cell-based therapies.

One company using this platform to help heal spinal cord injuries and paralysis is already heaving great success in FDA trials with a new cell line.

Their recent FDA Phase 1 trial data shows that patients treated with a new cell line are seeing a significant return of nerve function thanks to these grafts.

And last month, this same company announced nine-month follow-up data for patients that were given a 10 million cell dose to their injury. Even though this is only half of what researchers believe will be a full dose, at least 50% of patients have already shown signs of recovery.

This includes two levels of improvement in motor function, as well as improvements in arm, hand and finger function.

The nine-month data confirms what the company reported at three and six months, too.

Showing us that the new cell therapy has great durability. The introduced cells help heal the injury, and the result is more than temporary.

Under the accumulating evidence, the FDA has also decided to approve an expansion to this trial to include patients with spinal cord injury at the C4 location which is in the middle of the neck.

This is a very significant development.

The C4 spinal location is one of the most common locations for a spinal cord injury, and it often results in paralysis from the neck down.

And now the FDA is allowing the company to expand its treatment window from 1430 days after injury to 2142 days.

All these data bode very nicely for the future.

And not only for the developers of this specific cell therapy, but for other small biotech companies also trying to carve out market share in the pluripotent stem cell therapy space.

According to the National Spinal Cord Injury Statistical Center, there are some 17,000 new spinal cord injuries per year. This represents a large potential market for any company that masters this new technology and provides viable treatments for degenerative injuries.

Even more promising is that this new cell technology may also be used to treat cancer. And another group of tiny biotech companies are leading the way in research for this application, too.

We hope to see the data from these other FDA trials focusing on developing cancer vaccines during the second half of 2017.

If any of these new trial results comes back positive as early indications are showing, then were looking a tsunami sized wave of new opportunities in biotech. And for the handful of small companies and their investors, this should be a game changer.

As it stands currently, most of the companies making the push into regenerative medicine using new stem cell technology remain largely undiscovered.

As the FDA data become more actionable and these treatments move closer to market introduction, there will be some great plays to make.

And when that happens Ill be right here to make sure you have the best chance for the big score with this new technology.

To a bright future,

Ray Blancofor The Daily Reckoning

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Off-the-Radar Biotechs About to Break Out - Daily Reckoning

All of our blood vessels are lined with a type of cell called endothelial cells. To form vessels, individual endothelial cells begin to create empty holes in themselves, called vacuoles. They then connect with other endothelial cells that have done the same thing and the linked vacuoles form tubes, which ultimately become capillaries. Here, the researchers took endothelial cells and mixed them with either fibrin -- a protein involved in blood clotting -- or a semi-synthetic material called gelatin methacrylate (GelMA), which can be easily 3D-printed. When mixed with fibrin, the endothelial cells formed tubes fairly easily, but that wasn't the case with the GelMA. However, when the researchers added in another type of cell, a stem cell found in bone marrow, the endothelial cells were then able to form tubes in the GelMA.

"We've confirmed that these cells have the capacity to form capillary-like structures, both in a natural material called fibrin and in a semi-synthetic material called gelatin methacrylate, or GelMA," Gisele Calderon, the lead author of the study, said in a statement, "The GelMA finding is particularly interesting because it is something we can readily 3D print for future tissue-engineering applications."

The benefits of this method over others include cells that can be patient-specific, reducing the risk of immune system complications, and growth environments that are well suited for organ and vasculature growth -- they're reproducible, not likely to induce immune responses and help boost cell growth and vessel development. Along with making 3D-printed organs more viable, this method will also allow for the development of tissue that could make for more effective and efficient drug testing. In a statement, Jordan Miller, whose lab the work was done in, said, "Preclinical human testing of new drugs today is done with flat two-dimensional human tissue cultures. But it is well-known that cells often behave differently in three-dimensional tissues than they do in two-dimensional cultures. There's hope that testing drugs in more realistic three-dimensional cultures will lower overall drug development costs."

You can watch a video of the cells beginning to form tubes here and Calderon explaining her work in the video below.

Excerpt from:
Researchers are closer to working capillaries in 3D-printed organs - Engadget

July 10, 2017 Credit: CC0 Public Domain

Investigators from Brigham and Women's Hospital (BWH) and the Harvard Stem Cell Institute have a potential solution for how to kill tumor cells that have metastasized to the brain. The team has developed cancer-killing viruses that can deliver stem cells via the carotid artery, and applied them to metastatic tumors in the brain of clinically relevant mouse models. The investigators report the elimination of metastatic skin cancer cells from the brain of these preclinical models, resulting in prolonged survival. The study, published online this week in the journal PNAS, also describes a strategy of combining this therapy with immune check point inhibitors.

"Metastatic brain tumors - often from lung, breast or skin cancers - are the most commonly observed tumors within the brain and account for about 40 percent of advanced melanoma metastases. Current therapeutic options for such patients are limited, particularly when there are many metastases," says Khalid Shah, MS, PhD, director of the Center for Stem Cell Therapeutics and Imaging (CSTI) in the BWH Department of Neurosurgery, who led the study. "Our results are the first to provide insight into ways of targeting multiple brain metastatic deposits with stem-cell-loaded oncolytic viruses that specifically kill dividing tumor cells."

In their search for novel, tumor-specific therapies that could target multiple brain metastases without damaging adjacent tissues, the research team first developed different BRAF wild type and mutant mouse models that more closely mimic what is seen in patients. They found that injecting patient-derived, brain-seeking melanoma cells into the carotid artery of these preclinical models resulted in the formation of many metastatic tumors throughout the brain, mimicking what is seen in advanced melanoma cancer patients. The injected cells express markers that allow them to enter the brain and are labelled with bioluminescent and fluorescent markers to enable tracking by imaging technologies.

To devise a potential new therapy, the investigators engineered a population of bone marrow derived mesenchymal stem cells loaded with oncolytic herpes simplex virus (oHSV), which specifically kills dividing cancer cells while sparing normal cells. Previous research by Shah and his colleagues shows that different stem cell types are naturally attracted toward tumors in the brain. After first verifying that stem cells injected to the brain would travel to multiple metastatic sites and not to tumor-free areas in their model, the team injected stem cells loaded with oHSV into the carotid artery of metastasis-bearing mice.. Injecting the stem cells loaded with oHSV into the carotid artery, a likely strategy for clinical application, led to significantly slower tumor growth and increased survival, compared with the models that received unaltered stem cells or control injections. The oHSV loaded stem cells are ultimately killed by oHSV mediated oncolysis, preventing the engineered cells from persisting within the brain, which is an important safety component in the therapeutic use of these stem cells.

Due to an increasing body of evidence which suggests that the host immune response may be critical to the efficacy of oncolytic virotherapy, Shah and his colleagues also developed an immunocompetent melanoma mouse model and explored treating with both stem cell loaded oHSV and immune checkpoint blockers such as the ones that target the PD-1/PD-L1 pathway. They found that PD-L1 immune checkpoint blockade significantly improved the therapeutic efficacy of stem cell based oncolytic virotherapy in melanoma brain metastasis.

"We are currently developing similar animal models of brain metastasis from other cancer types as well as new oncolytic viruses that have the ability to specifically kill a wide variety of resistant tumor cells," said Shah, who is also a professor at Harvard Medical School and a principal faculty member at the Harvard Stem Cell Institute. "We are hopeful that our findings will overcome problems associated with current clinical procedures. This work will have direct implications for designing clinical trials using oncolytic viruses for metastatic tumors in the brain."

Explore further: Stem-cell-based therapy promising for treatment of breast cancer metastases in the brain

More information: Wanlu Du el al., "In vivo imaging of the fate and therapeutic efficacy of stem cell-loaded oncolytic herpes simplex virus in advanced melanoma," PNAS (2017). http://www.pnas.org/cgi/doi/10.1073/pnas.1700363114

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Stem cell-based therapy for targeting skin-to-brain cancer - Medical Xpress

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We know from experience that patients having scars find them particularly unattractive and mentally stressful. Scars form after wounds have finished healing, when deeper skin layers have been injured. Skin injuries can be caused by an accident, a skin disease or burns. So-called pregnancy stretch marks are also scars.

At first, scars will be red due to the large number of blood vessels. The scar tissue then gradually lightens in color, because the amount of collagenous fibers increases over time. From a medical point of view, scar tissue is an inferior kind of tissue and if put under a certain amount of pressure, so-called scar hernias can be a result thereof.

The formation of scars cannot be prevented after the deeper skin layers have been injured. The chances of the scar healing without too many traces increase, if the wound is treated well during the healing process.

If your wound healing process is already completed and scar tissue has formed, further treatment depends on the cause of the injury and type of scarring. In any case, we require your autologous stem cells obtained from your body fat. It is necessary to extract a small amount of your bodys own fat in order to obtain the stem cells. In accordance with your wishes, liposuction is carried out with microcannulas or regular cannulas.

The question as to whether the scars will be treated with stem cells only or if scar tissue has to be removed depends on the scar itself:

Post-surgery care is minimal: Treatment is on an outpatient basis; afterwards, you are fully mobile and normally can go back to work without any restrictions. We will provide you with individual recommendations for your post-treatment care according to the extent and type of area treated and will give you support during the healing process.

Schedule consultation appointment

Last updated: February 24, 2015

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Treatment of Scars with Stem Cells :: Stem Cell Skin ...

Firefighter Dustin Luebke puts his life on the line everyday protecting and serving the community. Never did he think his life would be threatened in a different way.

So now in 2017, Im going through it for the third time. So now its again recurring Hodgkins, said Luebke.

It all started in March of 2014. After 12 rounds of chemotherapy and six months in remission, it came back. He needed a stem cell transplant and was able to use his own stem cells.

Shortly after completing a year of chemotherapy after the transplant, it came back for the third time.

Its tough to swallow the first time and the second time. And then with the third time, its frustrating, said Luebke.

This father to three little girls will eventually need a bone marrow transplant.

But with this time, right now Im just doing an immunotherapy and were hoping that brings it back down to a cellular level and I can be on that for as long as until it stops working. So then it would require a stem cell transplant with a donor this time, said Luebke.

Thats where you and I can do our part and become part of the bone marrow registry and potentially be the match and save a life like Luebke's.

You fill out a short questionnaire. It talks about your health history and some personal questions, like how willing would you be to become a donor and then the swabbing process is really simple. We just swab each of your cheeks for a couple of minutes and then youre done, said Jalisa Spittler, transplant coordinator.

Spittler says 70% of patients who need a donor dont have a match in their family making the bone marrow registry their only hope.

We do have a lot of patients here that are waiting for matches that we just cant find for them. So its really helpful if we can create a diverse list with tons of people from here in South Dakota, said Spittler.

I got three little girls to raise and beautiful wife at home so I gotta stick around for a few more years, said Luebke.

Its pretty tough to realize that now youre relying on someone else where before it was all the medicine and just chemotherapy and now youre relying on somebody else with healthy stem cells to keep you going, said Luebke.

Sometimes it takes months to find a match.

Its taxing on them because they have to take more chemotherapy the longer it takes us to find a match for them. And the more chemotherapy they take, the harder it is on their body to get through the transplant. So its really important that we have a huge number of people to look at first, said Spittler.

Theres many ways that you can help out with people lives. And whether its in a fire, on a medical call and even helping somebody with life itself and furthering their life and making it better so they dont have to do chemotherapy anymore, said Luebke.

Luebke is a hero to this family and the community.

The first step to being someones cure is to join Be the Match Registry.

This Tuesday at the Oyate Community Center in Sioux Falls, there will be a bone marrow registry drive from noon to 7PM. It is put on by Avera, the city of Sioux Falls, and Be the Match. Registering takes less than 15 minutes.

For more information, just call 877-AT-AVERA.

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Avera Medical Minute AMcK: Firefighter with recurrent Hodgkin Lymphoma will need bone marrow transplant in future ... - KSFY

By Reuters By Reuters July 8 at 8:47 AM

Stem cell tourism in which patients travel to developing countries for unproven and potentially risky therapies should be more tightly regulated, according to a group of international health experts.

With hundreds of medical centers around the world claiming to be able to repair tissue damaged by conditions such as multiple sclerosis and Parkinsons disease, tackling unscrupulous advertising of such procedures is crucial.

These therapies are advertised directly to patients with the promise of a cure, but there is often little or no evidence to show they will help or that they will not cause harm, the 15 experts wrote in the journal Science Translational Medicine.

Some types of stem cell transplant mainly using blood and skin stem cells have been approved by regulators after full clinical trials found they could treat certain types of cancer and grow skin grafts for burn patients.

But many other potential therapies are only in the earliest stages of development and have not been approved by regulators.

Stem cell therapies hold a lot of promise, but we need rigorous clinical trials and regulatory processes to determine whether a proposed treatment is safe, effective and better than existing treatments, said one of the 15, Sarah Chan of Britains University of Edinburgh.

The experts called for global action, led by the World Health Organization, to introduce controls on advertising and to agree on international standards for the manufacture and testing of cell- and tissue-based therapies.

The globalization of health markets and the specific tensions surrounding stem cell research and its applications have made this a difficult challenge, they wrote. However, the stakes are too high not to take a united stance.

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'Stem-cell tourism' needs tighter controls, say medical experts - The ... - Washington Post

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LIMA Employers in the greater West Central Ohio region will collect $33 million in rebates from the Ohio Bureau of Workers Compensation in checks that will be mailed beginning next week.

BWC Administrator/CEO Sarah Morrison, in Lima to present a ceremonial check to local business leaders, said employers are free to spend their rebates as they wish, but she hopes they will consider investing in workplace safety.

We work with employers all over Ohio to prevent injuries and illness in the workplace, and they will tell you that investing in safety is a wise business decision, said Morrison, speaking at a press conference at the Lima/Allen County Chamber of Commerce. Safe workplaces mean fewer injuries, fewer medical claims and a stable workforce, all of which leads to a healthy bottom line for a business.

Morrison was joined by chamber President/CEO Jed Metzger and Tony Daley of Limas Spallinger Millwright Services Inc. Metzger and Daley accepted the check on behalf of employers in the entire region, which includes Allen, Auglaize, Shelby, Hancock, Putnam, and Van Wert counties.

Ohio Gov. John Kasich proposed the rebate in March. Its the third such rebate in the last four years, made possible by an improving safety climate, prudent fiscal management and strong investment returns. The plan to distribute rebates to more than 200,000 Ohio employers during the month of July was approved by BWCs Board of Directors in April. Visitbwc.ohio.govfor more details and eligibility requirements.

The plan also includes a $44 million investment innew health and safety initiativesto promote a healthy workforce and a culture of safety in every Ohio workplace. This includes a new wellness program for small employers, funding for programs to help firefighters and those who work with children and adults with disabilities, and an education campaign to address common injuries at work and in the home.

A healthy economy depends on a strong and healthy workforce, Morrison continued. And when the economy is healthy, we all benefit.

Rebate checks will be mailed in phases starting July 10.

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Antabuse doctors - Antabuse interactions with perfume - Van Wert independent

Sure, the main purpose of sex isprocreation, but according to one researcher, in as little as 30 years, we may have more efficient and cheaper ways of making babiesthangood ole fashioned intercourse. According to Hank Greely, the director of StanfordLaw Schools Center for Law and Biosciences, human reproductionmay become automated faster than you realize.

Greely believes that within three decades, people will no longer have sex as a way to reproduce, and instead relyongenetically edited embryos grown from skin-derived stem cells, not the combination of an egg or sperm, The Independent reported. According to Greely, this processensures that the embryo is free from any devastating genetic diseases, and wouldalso be cheaper in the long run because of the money it would save in healthcare over the years. Whats more, Greely predicts that couples would be able to choose other genetic traits in their children, such as physical features and intelligence.

Read: What Is A Three Parent IVF Technique? Worlds First Baby Born Using DNA From Three Parents, But How?

I dont think were going to be able to say this embryo will get a 1550 on its two-part SAT, Greely said this week at Aspen Ideas Festival, Quartz reported, But, this embryo has a 60% chance of being in the top half, this embryo has a 13% chance of being in the top 10%I think thats really possible.

The idea may soundfar-out, but according to Quartz, it already happens on a much smaller and limited scale as a way to prevent certain diseases. Although extremely expensive at the moment, advances in stem cell technology willhelp to drive down the cost. In addition, the amount that the government would save on not having to take care of sick babies would also make this more cost-efficient.

Making babies from skin cellsrather than a traditional egg fertilized with spermmaysound like its straight out of Hollywood, butthetechnology is quickly advancing. The skin cells, one from the mother and the other from the father, are coaxed into becoming an egg or sperm cell, The New York Times reported. This also means that one day same-sex couples may be able to have biologically related children. So far, vitro gametogenesis (IVG), or making sex cells from stem cells, has only worked on mice.

Theres also the worry that being able to genetically manipulate your offspring may lead to a eugenicssituation where less favorable traits get written out of the human genome forever. However, Greely also believes this is not the case.

This is not designer babies or super babies, said Greely, Quartz reported. This is selecting embryos. You take two people, all you can get out of a baby is what those two people have.

Just because well no longer need sex for procreation doesnt mean the activity is going out of fashion anytime soon. It's agreat way to createfuture generations, and sexis also good for your physical and mental health, as well as keeping couples together.

See Also:

Designer Babies: The Truth Behind Preimplantation Genetic Diagnosis

Scientists Edit Human Embryo Genes For First Time Ever: A Step Toward Disease-Free Future?

Link:
Sex For Reproduction May Be Obsolete Within 30 Years Thanks To New Technology, Professor Predicts - Medical Daily

Times Now

'Custom-made' babies to be in demand in 30 years, says expert Times Now The process will involve taking a female skin sample to create stem cells, which is then used to create eggs. These eggs are then fertilised with sperm cells, resulting in a selection of embryos. While Greely does acknowledge that ethical issues could ... People will 'stop having sex to procreate within 30 years', says scientistMetro Sex Not Necessary for Reproduction: Stanford Professor's Prediction Suggests a Bizarre FutureIndia.com PLAYING GOD: 'End to sex' in 30 years as parents will DESIGN their babies in the labExpress.co.uk Christian Post -Deccan Chronicle all 11 news articles »

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'Custom-made' babies to be in demand in 30 years, says expert - Times Now

This report describes and evaluates cell therapy technologies and methods, which have already started to play an important role in the practice of medicine. Hematopoietic stem cell transplantation is replacing the old fashioned bone marrow transplants. Role of cells in drug discovery is also described. Cell therapy is bound to become a part of medical practice.

Stem cells are discussed in detail in one chapter. Some light is thrown on the current controversy of embryonic sources of stem cells and comparison with adult sources. Other sources of stem cells such as the placenta, cord blood and fat removed by liposuction are also discussed. Stem cells can also be genetically modified prior to transplantation.

Cell therapy technologies overlap with those of gene therapy, cancer vaccines, drug delivery, tissue engineering and regenerative medicine. Pharmaceutical applications of stem cells including those in drug discovery are also described. Various types of cells used, methods of preparation and culture, encapsulation and genetic engineering of cells are discussed. Sources of cells, both human and animal (xenotransplantation) are discussed. Methods of delivery of cell therapy range from injections to surgical implantation using special devices.

Cell therapy has applications in a large number of disorders. The most important are diseases of the nervous system and cancer which are the topics for separate chapters. Other applications include cardiac disorders (myocardial infarction and heart failure), diabetes mellitus, diseases of bones and joints, genetic disorders, and wounds of the skin and soft tissues.

Regulatory and ethical issues involving cell therapy are important and are discussed. Current political debate on the use of stem cells from embryonic sources (hESCs) is also presented. Safety is an essential consideration of any new therapy and regulations for cell therapy are those for biological preparations.

The cell-based markets was analyzed for 2016, and projected to 2026. The markets are analyzed according to therapeutic categories, technologies and geographical areas. The largest expansion will be in diseases of the central nervous system, cancer and cardiovascular disorders. Skin and soft tissue repair as well as diabetes mellitus will be other major markets.

The number of companies involved in cell therapy has increased remarkably during the past few years. More than 500 companies have been identified to be involved in cell therapy and 306 of these are profiled in part II of the report along with tabulation of 291 alliances. Of these companies, 170 are involved in stem cells. Profiles of 72 academic institutions in the US involved in cell therapy are also included in part II along with their commercial collaborations. The text is supplemented with 64 Tables and 22 Figures. The bibliography contains 1,200 selected references, which are cited in the text.

Key Topics Covered:

Part I: Technologies, Ethics & Regulations

Executive Summary

1. Introduction to Cell Therapy

2. Cell Therapy Technologies

3. Stem Cells

4. Clinical Applications of Cell Therapy

5. Cell Therapy for Cardiovascular Disorders

6. Cell Therapy for Cancer

7. Cell Therapy for Neurological Disorders

8. Ethical, Legal and Political Aspects of Cell therapy

9. Safety and Regulatory Aspects of Cell Therapy

Part II: Markets, Companies & Academic Institutions

10. Markets and Future Prospects for Cell Therapy

11. Companies Involved in Cell Therapy

12. Academic Institutions

13. References

For more information about this report visit https://www.researchandmarkets.com/research/hpj9sh/cell_therapy

Source: Jain PharmaBiotech

Media Contact:

Laura Wood, Senior Manager press@researchandmarkets.com

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To view the original version on PR Newswire, visit:http://www.prnewswire.com/news-releases/global-cell-therapy-report-2017---technologies-markets-and-companies-2016-2026-with-profiles-of-key-companiesinstitutions-300483356.html

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Global Cell Therapy Report 2017 - Technologies, Markets and Companies 2016-2026 with Profiles of Key Companies ... - PR Newswire (press release)

Equine mesenchymal stromal cells have been shown to have antibacterial properties, raising the possibility they could aid in healing troublesome skin wounds in humans and horses.

Mesenchymal stem cells, or MSCs, are multipotent connective-tissue cells that can differentiate into a variety of cell types, including bone cartilage, muscle and fat.

Chronic skin wounds in humans are common and their treatment is often complicated by pathogenic bacteria. Therefore, safe and innovative treatments to reduce the bacterial load in such wounds are needed.

MSCs have been reported to provide local hormonal signals that promote healing in skin wounds. However, the effects of equine MSCs on the growth of bacteria commonly found in skin wounds has not, until now, been explored.

Researchers from the College of Veterinary Medicine at New Yorks Cornell University have been the first to show that equine MSCs possess antimicrobial properties which stymied the growth of Escherichia coli (E. coli) and Staphylococcus aureus (S. aureus).

The MSCs did so in part by secreting antimicrobial peptides and depolarizing the bacterial cell membranes.

Rebecca Harman, Gerlinde Van de Walle, Steven Yang, and Megan He, writing in the journal Stem Cell Research & Therapy, said they focused on the antibacterial properties of MSCs from horses, as this animal model offered a readily translatable model for therapies in humans.

The study team described the laboratory experiment they set up, in which MSCs were isolated from the blood of healthy horses. The bacteria were cultured in the presence of MSCs and an MSC conditioned medium a processed fluid containing all factors secreted by the cells.

They found that both the MSCs and the MSC conditioning medium inhibited the growth of both bacteria, and that the conditioning medium depolarized the cell membranes of these bacteria.

The conditioning medium was found to contain four antimicrobial peptides, cystatin C, elafin, lipocalin 2, and cathelicidin. These appeared to be at least partially responsible for the antibacterial action.

They also looked for the presence of beta defensin 2 in equine MSCs since it has been found to be secreted by human umbilical cord-derived MSCs. It belongs to a widespread family of antimicrobial peptides found in most mammals, including horses. To the surprise of the research team, they could not detect beta defensin 2 in equine MSCs.

Our results, they concluded, demonstrate that equine MSCs inhibit bacterial growth and secrete factors that compromise the membrane integrity of bacteria commonly found in skin wounds.

There appeared to be a difference in the underlying mechanisms targeting each species, withdifferent secreted factors appearing to target different bacteria.

It will be interesting, they said, to study the effects of the MSC conditioning medium on additional bacterial species commonly found in equine skin wounds. The findings will likely be relevant to human as well as veterinary medicine, they said.

Since we found that equine MSCs secrete a variety of antimicrobial peptides that appear effective against both gram-positive [S. Aureus]and gram-negative [E.Coli] bacteria, these cells may serve as a broad-spectrum treatment to control bacterial growth and kill bacteria, without leading to resistance.

The study team said they now intended to evaluate the effectiveness of equine MSCs in healing both acute and chronic wounds.

Antimicrobial peptides secreted by equine mesenchymal stromal cells inhibit the growth of bacteria commonly found in skin wounds Rebecca M. Harman, Steven Yang, Megan K. He and Gerlinde R. Van de Walle Stem Cell Research & Therapy 2017 8:157 DOI: 10.1186/s13287-017-0610-6

The study, published under a Creative Commons License, can be read here.

Read more from the original source:
Mesenchymal stromal cells from horses show potential for healing skin wounds - Horsetalk

Stem cell therapy: the next wave in regenerative medicine?

All it involved was a quick injection no different, really, than a flu shot.

A few weeks later, Bill Ambrose realized hed become significantly less reliant on taking Aleve for knee pain, and he was re-learning how to walk without shuffling his feet.

Surgery, it turned out, might not be necessary after all.

Last November, Ambrose scheduled knee surgery to alleviate discomfort in his knees caused by what orthopedic doctors called true bone-on-bone at the joint. But for one reason or another, he kept missing pre-surgery and the surgery never happened.

The next month, Ambrose met with Dr. Bill Nolan, of Cherry Street Health Group, to discuss advertising space in the Danvers Herald.

For the purpose of full disclosure, Ambrose is an employee of Gatehouse Media Company, and he works in the advertising department for Wicked Local, the local branch of GHM newspapers.

After Nolans ads ran inthe Jan. 5issue of the Herald, Ambrose said he reached out to Nolan again. This time, for himself.

Nolans practice offered a solution to his knee pain an alternative to knee surgery he had never considered before: stem cell therapy.

Essentially, the solutionCherry StreetHealth Group offered was an injection of amniotic fluid into Ambrose's knee joint. The stem cells and other growth factorsin the fluid would allow for the regeneration of the cartilage at the joint.

I became interested so I decided to go ahead with it, Ambrose said.

He brought in scans to show Nolan, who said, contrary to what orthopedic doctors had told him, he didnt have true bone on bone. There was still a small space between the bones.

I decided to have one leg done and my knee started getting much better, he said.

Satisfied with the results of the first injection, Ambrose decided to get his left knee done in April.

I still experience some pain in [the left knee], but I get up in the morning and theres very little pain at all, he said in an interview a few weeks following the appointment.

The stem cell option

In the U.S., there are three ways that stem cells are used, Nolan said. Theyre either taken from bone marrow, fat cells, or the amniotic membrane of a healthy c-section from a consenting woman.

When stem cellsare injected into the body,they're expected to increase space at the joint, rebuild cartilage, and ultimately, provide more stability in the joint. As many as 570 businesses across the country advertise some kind of stem cell therapy, according to a 2016 paper.

Stem cell therapy is not necessarily a new discovery, but it is relatively recent in the world of regenerative medicine.Stem cells were first used as much as century ago, first for eye procedures and as filler for the spinal cord, according to Regenexx, which claims to have pioneered orthopedic stem cell treatments in 2005.

Adult stem cells are retrieved directly from the patient, either frombone marrow or fat cells,and concentrated beforeits reinjectedinto the patient's site of pain.

In the case of amniotic fluid therapy,amniotic fluid, which contains stem cells and other growth factors, is injected into the site. These cellshave been shown to "expand extensively" and show "high renewal capacity,"according to research published in the National Library of Medicine.

We know that as you age, your stem cell count decreases,Nolan said, explaining the benefit of using cells from the amniotic membrane. We know that when we get it from the amniotic membrane, theres a large amount of stem cells that are present. From the amniotic membrane, there are no antibodies or antigens, so its safe for anyone to get.

At Cherry Street Health Group, theproduct usedis produced by General Surgical and distributed by RegenOMedix, according to Nolan.The product, which is called ReGen Anu RHEO, is American Tissue Bank approved and FDA cleared.

RHEO is marketed as "a human tissue allograft derived from placental tissue; amniotic membrane and amniotic fluid."Its a"powerful combination" of amniotic fluid and mesencymal stem cells, which are known to differentiate into a variety of cell types, according to RegenOMedix.It also contains growth factor proteins andis "rich" in other necessary components for tissue regeneration.

The product is non-steroidal and comes with no side effects, and the company says no adverse events have been recorded using the product.

Nolan said stem cell therapy has been offered as a treatmentat Cherry Street since 2016.

Across the U.S., there are as many as 56 businesses marketing some form of amniotic stem cellsto its consumers, according to the same paper.

At Rush University Medical Center in Chicago, for example, orthopedic surgeon Adam Yanke enrolled one of his patients into an experimental amniotic cell therapy treatment program. The woman, a 65-year-old suffering from osteoarthritis in both knees, told reporters the injections were "by far the most effective pain treatment" she had tried, and so farthat relief has lasted up to a year.

But while the use of amniotic fluid therapyas a regenerative medicine is becoming increasingly popular throughout the U.S.,the use of amniotic stemcellsdoesn't comewithout concern from some within the community.

Dr. Chris Centeno, who specializes in regenerative medicine andthe clinical use of adult stem cells, has blogged numerous times for Regenexx on the "scam" of using amniotic stem cells most recently in sharply worded post on May 22.

"Regrettably, we have an epidemic on our hands that began when sales reps began telling medical providers thattheir dead amniotic and cord tissues had loads of live cells on it," he wrote.

Nolan said he was familiar with Centeno's posts.

"A lot of the stem cell stuff is new," he said. "Some of the products out there ... They were doing testing on them and not finding cells."

Cherry Street Health Group has treatedabout 50patients with this form of regenerative medicine and had significant success, according to Nolan. Although Nolan owns the health group on Cherry Street in Danvers, the stem cell treatments are provided under the medical practice of Dr. Pat Scanlan.

Weve had really, really amazing success, Nolan said. Weve had over 95 percent success of all the patients weve had in the office. Its been a game changer from a practice standpoint.

The "worst thing" that could happen is there might not be any regeneration, he explained.

"You might get pain relief, but no regeneration," Nolan said. "But from what weve seen, there have been no negative side effects."

At Cherry Street, knees are the most commonly treated joints, followed by hips, shoulders and the lower back. The cervical spine is the least common.

"I hesitated on the surgery, and I'm gladI did," Ambrose said. "Even if[the stem cells]don't do any more than what they've done, its been well worth it."

Patients who do present with true bone on bone, however, are not candidates for this form of therapy, Nolan said.

The cost comparison

At Cherry Street Health Group, the cost of the injection comes toroughly $4,000 per knee, a cost that isn't covered by insurance. By comparison, health-care providers often charge insurers more than $18,000 for knee replacement surgeries in the Boston area, according to a report by the Blue Cross and Blue Shield Association.

The report, however, doesn't account for what the patient actually pays.

Nolan said when other factors of post-op are considered time off of work, rehabilitation time and cost the out-of-pocketcost for surgery compared to stem cell treatment is comparable.

"When you really boil it down, it can be the same or, in a lot of cases, a savings," he said.

Ambrose said it "boggles his mind" that more people don't choose this treatment over surgery.

"Why would you spend $40,000 on a car and not want to spend $4,000 on a knee?," he said."Its crazy. Yes, its out of pocket. So what? We buy a lot of stuff we dont need, and then for something like this, something that people, if they do it, theyll be glad they did it. Its just hard to convince them to do it."

In arecent report in STAT news, a health news start up of the Boston Globe, a study of orthopedic procedures in the U.S. suggested an estimated one-third of knee replacement surgeries are inappropriate. More than 640,000 of these surgeries are performed each year, making for a $10 billion dollar industry in knee surgery.

The study said that evidence isn't limited to just knee surgeries.

"There's a lot that needs to change when we look at health care in general,"Nolan said. "It's really no surprise that something like doing this regenerative medicine is going to take time for it to really take off."

Read more:
Danvers health group offers alternative solution to surgery - Wicked Local North of Boston

For over 20 years autologous hematopoietic stem cell treatment (AHSCT) has been a therapy for autoimmune diseases such as multiple sclerosis, rheumatoid arthritis and lupus; however, the exact mechanism of action remains unclear. Recent clinical research has also been exploring the use of stem cell therapy for type 1 diabetes, another autoimmune disease which affects over 422 million individuals globally.

Type 1 diabetes, formerly known as juvenile or insulin-dependent diabetes, is a chronic condition where little or no insulin is produced by the pancreas. Immune cells attack pancreatic beta cells which produce insulin, leading to inflammation. Insulin is an essential hormone for energy production as it enables the breakdown of sugars to enter the cells and produce energy. The onset of type 1 diabetes occurs when significant inflammation damages beta cells and results in insufficient maintenance of glucose haemostasis (balance of insulin and glucagon to maintain blood glucose levels).

Therapies currently used in type 1 diabetes treatment include insulin administration, blood glucose monitoring and screening for common comorbidities and diabetes-related complications. However, these treatments fail to reduce the damage on a patients immune system. The use of autologous hematopoietic stem cells as a potential type 1 diabetes therapy is based upon the ability of the stem cells to reset the immune system. Autologous hematopoietic stems cells are retrieved from a patients own bone marrow or peripheral blood (blood which circulates the body and contains red blood cells, white blood cells and platelets) and after conditioning are injected intravenously.

A recent study by Ye and colleagues published in Stem Cell Research & Therapy (2017) investigated the effects AHSCT had on the immune response in recently diagnosed diabetes type 1 patients. The study included 18 patients (12-35 years old) with type 1 diabetes who had been diagnosed within less than 6 months. Of these 18 participants, 10 received a traditional insulin injection as treatment and eight received AHSCT. An additional 15 patients who matched in age, gender and BMI of these two groups were enrolled as a control group.

To test the effects of the treatment on immune response, patients peripheral blood cells were assessed. Samples were taken before they started treatment and then again 12 months after either the AHSCT or insulin-only therapies were administered.

Before treatment, peripheral blood cell distribution was almost equivalent in the two groups; however, after 12 months a significant difference was observed. The results of this clinical trial showed that patients receiving AHSCT exhibited significantly reduced development and function of Th1 and Th17 cells (types of T cells which cause inflammation in autoimmune diseases), compared to those only receiving the insulin treatment.

The inhibition of T-cell proliferation and function, along with decreased production of cytokines (pivotal chemical messengers which aid an immune response) observed in patients receiving AHSCT treatment suggests there is a strong link between the therapy and effects on the patients immune response. This may explain why AHSCT results in better therapeutic effects when compared with insulin-only traditional therapy.

The authors note that the small number of participants and length of the study are the two main limitations. Future clinical studies should include a larger number of patients and long-term follow up, especially since AHSCT can cause damage to the bone marrow and lead to potentially serious infections.

Progression of type 1 diabetes, as mentioned above, results in unavoidable immune damage from inflammation. This study suggests the combination of therapies including AHSCT treatment and high-dose immunosuppressive drugs may be a potential new therapeutic approach to type 1 diabetes. It is hypothesized that this combination has the ability reset the immune system and increase the recovery capacity of beta cells. Further clinical studies are essential though, to shed more light on the mechanism and use of stem cell therapy for type 1 diabetes.

Written By:Lacey Hizartzidis, PhD

Original post:
Stem Cell Therapy for Type 1 Diabetes - Medical News Bulletin