Stem Cell Clinic

Cutting-edge stem cell therapy proves safe, but will it ever be effective? – Science Magazine

By adminMarch 15, 2017Uncategorized

Masayo Takahashi (second from left) treated macular degeneration with retinal tissue grown from iPS cells.

Kyodo News/Contributor/getty images

By Dennis NormileMar. 15, 2017 , 5:00 PM

Its official: The first use of induced pluripotent stem (iPS) cells in a human has proved safe, if not clearly effective. Japanese researchers reported in this weeks issue of The New England Journal of Medicine (NEJM) that using the cells to replace eye tissue damaged by age-related macular degeneration (AMD) did not improve a patients vision, but did halt disease progression. They had described the outcome at conferences, but publication of the details is an encouraging milestone for other groups gearing up to treat diseased or damaged organs with the versatile replacement cells, which are derived from mature tissues.

This initial success is pretty momentous, says Alan Trounson, a stem cell scientist at the Hudson Institute of Medical Research in Melbourne, Australia. But the broader picture for iPS therapies is mixed, as researchers have retreated from their initial hopes of creating custommade stem cells from each patients tissue. That strategy might have ensured that recipients immune systems would accept the new cells. But it proved too slow and expensive, says Shinya Yamanaka of Kyoto University in Japan, who first discovered how to create iPS cells and is a co-author of the NEJM paper. He and others are now developing banks of premade donor cells. Using stocks of cells, we can proceed much more quickly and cost effectively, he says.

Even so, clinical work is progressing more quickly than I had expected, says Yamanaka, who did his groundbreaking work just a decade ago. His collaborator on this trial, Masayo Takahashi of the RIKEN Center for Developmental Biology in Kobe, Japan, had a head start. An ophthalmologist, Takahashi was familiar with the ravages of AMD, a condition that progressively damages the macula, the central part of the retina, and is the leading cause of blindness in the elderly.

Takahashi started investigating treatments for AMD in 2000, a time when the only cells capable of developing into all the tissues of the body had to be extracted from embryos. But she was stymied by immune reactions to these embryonic stem (ES) cells. When Yamanaka announced that he could induce mature, or somatic cells, to return to an ES celllike state, Takahashi quickly changed course to develop a treatment based on iPS cells.

Her team finally operated on the first patient, a 77-year-old Japanese woman with late-stage AMD, in September 2014. They took a sample of her own skin cells, derived iPS cells, and differentiated them into the kind of retinal cells destroyed by the disease. A surgeon then slipped a small sheet of the cells into the retina of her right eye.

An operation on a second patient was called off because a number of minor genetic mutations had crept into his iPS cells during processing, and uncontrolled growthcancerhas been a worry with such cells. These changes do not directly induce cancer, but we wanted to make safety the first priority, Yamanaka says. Also, Takahashi says, AMD drugs had stabilized the patients condition so there was no urgency in subjecting him to the risks of surgery, which include hemorrhaging and retinal damage.

Immediately after surgery the first patient reported her eyesight was brighter. Takahashi says the surgery halted further deterioration of her eye, even without the drug injections still being used to treat her other eye, and there were no signs of rejection of the graft as of last December.

Clinical work is progressing much more quickly than I expected.

The result is a proof of principle that iPS cellbased therapy is feasible, says Kapil Bharti, a molecular cell biologist at the U.S. National Institutes of Healths National Eye Institute in Bethesda, Maryland, who is also developing iPS cells for treating AMD. Takahashi says once her team gains more experience with the technique they will extend it to patients with earlier-stage AMD in an effort to preserve vision.

Last month, Takahashi won approval to try the procedure on another five patients with late-stage AMD. But this time, instead of using iPS cells derived from each patient, the team will draw on banked cells from a single donor. It takes time to create iPS cells, and a lot of time for the safety evaluation, Yamanaka says. It is also costly, at nearly $900,000 to develop and test the iPS cells for the first trial, Takahashi adds.

Using donor cells to create the iPS cells will make it more difficult to ensure immune compatibility. But Yamanaka says that donor iPS cells can be matched to patients based on human leukocyte antigen (HLA) haplotypessets of cell-surface proteins that regulate immune reactions. HLA-matched cells should require only small doses of immunosuppressive drugs to prevent rejection, Takahashi saysand perhaps none at all for transplantation into the immune-privileged eye.

Kyoto Universitys Center for iPS Cell Research and Application, which Yamanaka heads, has been developing an iPS cell bank. Just 75 iPS cell lines will cover 80% of the Japanese population through HLA matching, he says. Trounson, a past president of the California Institute for Regenerative Medicine, a stem cell funding agency, says banked iPS cells have advantages. Donor iPS cells may be safer than cells derived from older patients, whose somatic cells may harbor mutations. And Jordan Lancaster, a physiologist at the University of Arizona in Tucson, likes the speed of the approach. He is devising patches for heart failure patients based on iPS-derived myocardial cells that will be premanufactured, cryopreserved, and ready to use at a moments notice.

Patient-specific iPS cells will still have clinical uses. For one thing, Bharti says it will be difficult for cell banks to cover all HLA haplotypes. And a patients own iPS cells could be used to screen for adverse drug reactions, says Min-Han Tan, an oncologist at Singapores Institute of Bioengineering and Nanotechnology, who recently published a report on the approach.

Other human trials are not far behind. Yamanaka says his Kyoto University colleague Jun Takahashi (Masayo Takahashis husband) will launch trials of iPS-derived cells to treat Parkinsons disease within 2 years. Bharti hopes to start human trials of iPS cells for a different type of macular degeneration next year. And as techniques for making and growing iPS cells improve, researchers can contemplate treatments requiring not just 100,000 cells or sothe number in Takahashis retinal sheetsbut millions, as in Lancasters heart patches.

As clinical use approaches, Takahashi cautions that researchers need to keep public expectations realistic. For now, iPS treatments may help but wont fully reverse disease, she says. Regenerative medicine is not going to cure patients in the way they hope.

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Cutting-edge stem cell therapy proves safe, but will it ever be effective? - Science Magazine

Mesoblast’s (MESO) Regenerative Cell Therapy Proves Itself in Back Pain Trial – Economic Calendar

By adminMarch 15, 2017Uncategorized

This morning, Australian biotech company Mesoblast Ltd (NASDAQ:MESO) announced encouraging phase 2 trial data regarding its regenerative therapy for chronic lower back pain (CLBP). Mesoblasts proprietary allogeneic Mesenchymal Precursor Cells (MPCs) are given to patients via a single injection and, in the clinical trial, have proved to spark significant improvement in both lower back pain and function that lasted for at least 36 months.

The sustained benefits on pain and function over three years seen with a single injection of Mesoblasts cell therapy have the potential to transform the treatment paradigm for chronic low back pain due to disc degeneration, said Dr. Hyun Bae, the trial investigator and Director of the Spine Institute.

According to Bae, there is growing evidence that disc degeneration can be healed instead of needing to be replaced or fused. Researchers are quickly arriving at this inflection point in the treatment of lower back pain, which is especially critical given the ongoing opioid abuse epidemic sweeping the nation.

Researchers believe that MPCs are turned on by signals in damaged tissues that spark factors to block damaging inflammation and begin repairing the afflicted area.

The phase 2 trial in question involved 100 patients, who were treated with either six million or 18 million MPCs via one intra-discal injection or a placebo of either saline or hyaluronic aid. The primary endpoints were a 50 percent drop in Visual Analog Scale (CAS), which is a pain metric, and a 15-point reduction in Oswestry disability index (ODI) at both the one and two year marks.

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The primary endpoint composite at the two-year mark was met by 41 percent of patients in the 6 million MPC arm, 35 percent in the 18 million arm, with just 18 percent and 13 percent in the placebo groups. The phase 2 results support the companys criteria for its ongoing phase 3 program.

Mesoblast is confident that the durable treatment outcomes in lower back pain are part of an overarching mechanism that might also have a positive effect in other types of chronic conditions where a single injection of MPCs can lead to sustained benefits. Two of these potential indications, according to Mesoblast, are chronic heart failure and biological-resistant rheumatoid arthritis.

Following the trial data being released, Maxim Groups Jason Kolbert reaffirmed a Buy rating on Mesoblasts stock and maintained a price target at $14.00. The upside indicated is 81 percent.

The market opportunity is significant and the treatment fits ideally within the existing paradigm the analyst told investors this morning. Injecting cells versus steroids with a better AE profile and now a better long-term result.

Earlier this month, Mesoblast announced that its MSC-100-IV therapy had been given Fast Track designation by the Food and Drug Administration (FDA) for its use in children with steroid refractory acute Graft Versus Host Disease (aGVHD). This type of designation shortens the FDA review path from ten months to six months, and certain portions of the Biologics License Application (BLA) can be sent in on a rolling basis rather than when everything is finished.

The author of this article holds no position in any of the stocks mentioned above.

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Mesoblast's (MESO) Regenerative Cell Therapy Proves Itself in Back Pain Trial - Economic Calendar

3 Women Blinded By Unproven Stem Cell Treatments – NPR

By adminMarch 15, 2017Stem Cell Treatment

Scientists have long hoped that stem cells might have the power to treat diseases. But it's always been clear that they could be dangerous too, especially if they're not used carefully.

Now a pair of papers published Wednesday in the New England Journal of Medicine is underscoring both the promise and the peril of using stem cells for therapy.

In one report, researchers document the cases of three elderly women who were blinded after getting stem cells derived from fat tissue at a for-profit clinic in Florida. The treatment was marketed as a treatment for macular degeneration, the most common cause of blindness among the elderly. Each woman got cells injected into both eyes.

In a second report, a patient suffering from the same condition had a halt in the inexorable loss of vision patients usually experience, which may or may not have been related to the treatment. That patient got a different kind of stem cell derived from skin cells as part of a carefully designed Japanese study.

The Japanese case marks the first time anyone has given induced pluripotent stem (iPS) cells to a patient to treat any condition.

"These two reports are about as stark a contrast as it gets," says George Q. Daley, Harvard Medical School's dean and a leading stem cell researcher. He wrote an editorial accompanying the two papers. "It's really striking."

The report about the three women in their 70s and 80s who were blinded in Florida is renewing calls for the Food and Drug Administration to crack down on the hundreds of clinics that are selling unproven stem cell treatments for a wide variety of medical conditions, including arthritis, autism and stroke.

"One of the big mysteries about this particular case and the mushrooming stem cell clinic industry more generally is why the FDA has chosen to effectively sit itself out on the sidelines even as this situation overall grows increasingly risky to patients," says Paul Knoepfler, a University of California, Davis, stem cell researcher who has studied the proliferation of stem cell clinics.

"The inaction by the FDA not only puts many patients at serious risk from unproven stem cell offerings, but also it undermines the agency's credibility," Knoepfler wrote in an email.

In response to a query from Shots, an FDA spokeswoman wrote in an email that the agency is in the process of finalizing four new guidelines aimed at clarifying how clinics could use stem cells as treatments. The agency also noted that it had previously issued a warning to patients.

In the meantime, "consumers are encouraged to contact FDA and the appropriate state authorities in their jurisdictions to report any potentially illegal or harmful activity related to stem cell based products," the FDA email says.

Other researchers say the cases should stand as a warning to patients considering unproved stem cell treatments, especially those tried outside carefully designed research studies.

"Patients have to be wary and tell the difference between the snake oil salesmen who are going to exploit them and the kind of slow, painstaking legitimate clinical trials that are also going on," Daley says.

The New England Journal of Medicine report did not name the Florida clinic, but noted that the treatment was listed on a government website that serves as a clearinghouse for research studies. The sponsor is listed as Bioheart, Inc., which is part of U.S. Stem Cell Inc. in Sunrise, Fla.

Kristen Comella, the scientific director of U.S. Stem Cell, would not discuss the cases. "There were legal cases associated with eye patients that were settled under confidentiality, so I am not permitted to speak on any details of those cases due to the confidentiality clause," Comella said by phone.

She acknowledged, however, that the clinic had been performing the stem cell procedures. They were discontinued after at least two patients suffered detached retinas, she says.

But Comella defended the use of stem cells from fat tissue to treat a wide variety of other health problems.

"We have treated more than 7,000 patients and we've have had very few adverse events reported. So the safety track record is very strong," Comella says. "We feel very confident about the procedures that we do, and we've had great success in many different indications."

According to the New England Journal of Medicine report, The Florida clinic was using adult stem cells, which circulate in various parts of the body, including in fat tissue. While those cells may someday be turn out to be useful for treating disease, none have been proven to work.

The body produces a variety of stem cells. The kind that have generated the most excitement and controversy are human embryonic stem cells, which are derived from early human embryos and can be coaxed to become any kind of cell in the body.

Scientists are also excited about iPS cells, which can be made in the laboratory by turning any cell in the body, such as skin cells, into cells that resemble embryonic stem cells.

Those are the cells that were tested by the Japanese scientists. The stem cells were converted into retinal pigment epithelium (RPE) cells, which are the cells that are destroyed by macular degeneration.

"This represents a landmark," says Daley. "It's the first time any patient has been treated with cellular derivatives of iPS cells. So it's definitely a world first."

Daley noted that the scientists only treated one of the patient's eyes in case something went wrong, to ensure remaining vision would not be threatened in the other eye.

After at least a year, no complications had occurred and the patient had not experienced any further deterioration of vision in the treated eye. While that is promising, more patients would have to be treated and followed for much longer to know whether that approach is successful, Daley says.

"Given that macular degeneration is the most frequent cause of vision loss and blindness in the elderly and our population is aging, the prevalence of macular degeneration is going up dramatically," Daley says. "So to be able to preserve or even restore sight would be a really remarkable medical advance."

Despite the potentially encouraging results with the first patient, Daley noted that the Japanese scientists decided not to treat a second patient and suspended the study. That's because they discovered worrisome genetic variations in the RPE cells they had produced for the second patient.

"They weren't certain these would cause problems for the patient, but they were restrained enough and cautious enough that they decided not to go forward," Daley says. "That's what contrasts so markedly with the approach of the second group, who treated the three patients with an unproven stem cell therapy that ended up have devastating effects on their vision."

In this case, the New England Journal of Medicine report says, patients paid $5,000 each to receive injections of solutions that supposedly contained stem cells that were obtained from fat removed from their abdomens through liposuction.

Even though the safety and effectiveness of this procedure is unknown, all three patients received injections in both eyes.

"That's what led to these horrible results," says Thomas Albini, a retina specialist at the University of Florida's Bascom Palmer Eye Institute, who helped write the report.

Before the procedure, all three women still had at least some vision. Afterwards, one woman was left completely blind while the other two were effectively blind, Albini and his colleagues reported.

The cases show that patients need to be warned that something that "sounds too good to be true may indeed be too good to be true and may even be horrible," Albini says.

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3 Women Blinded By Unproven Stem Cell Treatments - NPR

RenovaCare: Stem Cell Treatment Heals Burns In Weeks Not Months – Seeking Alpha

By adminMarch 15, 2017Stem Cell Treatment

RenovaCare Inc. (OTCQB:RCAR) is a New York City-based biotechnology company developing its patented CellMist and SkinGun stem cell technologies for treating burns in weeks or less as well as treating chronic and acute wounds, acne scarring, and skin defects and diseases. In December, it received a U.S. patent for its SkinGun device.

Before joining RenovaCare, CEO Thomas Bold was CEO of StemCell Systems. He has more than 15 years of experience in medical biotechnology device manufacturing and stem cell platform development.

Harlan Levy: How does your CellMist technology specifically work?

Thomas Bold: Doctors isolate a high concentration of the most desirable stem cell population from a very small donor sample of the patient's own skin and suspended in the liquid CellMist Solution. It's then gently sprayed onto wound sites using our SkinGun, which looks like Captain Kirk's particle-beam gun, the "Phaser" in the Star Trek TV series.

The isolated cells include cells that proliferate rapidly in order to achieve quick re-epithelialization. This is the stage at which a burn is technically considered "healed" and patients are often discharged. The average person would recognize this healing phase as the point at which the wound develops a thin, shiny, pink-colored protective layer.

H.L.: What are existing burn treatments, and how do they compare with the SkinGun treatment?

T.B.: Traditional skin grafting has been the treatment for burns and wounds for centuries. More recently, mesh grafting has become the latest standard of care. This process surgically removes large sheets of healthy skin from the patient. Following this painful donor procedure, the sheet is punctured in a grid-like pattern to form an expandable mesh. Surgeons pull this mesh as wide as feasible and surgically stitch this skin to the patient's wound. The procedure is extremely painful, creates an additional wound at each donor site and results in poor cosmetic outcomes, often with scarred and deformed skin.

This transplanted skin can result in restricted joint movement and is unable to grow with the patient. Consequently, mesh graft patients require months and sometimes up to a year of physical therapy and can face psychological problems from the permanent disfigurement of scarring. In addition, long-term pain management with painkillers is very often necessary.

With the RenovaCare treatment technology, by spraying the patient's stem cells, the SkinGun overcomes the need for removing large sheets of donor skin, and the resultant healing does not require prolonged physical therapy. The spray procedure is gentle, and the skin that regrows looks, feels, and functions as the original skin that it replaces. Most often the healing process takes only a week.

It's very important to note here that a sheet of meshed skin covers only up to six times its original donor area. The RenovaCare system covers up to 100 times its donor skin sample. This is why the donor skin sample can be so small compared to the injured treatment area.

H.L.: What about scars and infection potential compared with conventional treatments?

T.B.: A wound heals from the edges towards the middle. The bigger the wound, the longer this process takes. And the longer this process takes, the higher the risk of infection and scarring.

Imagine a large burn of 20, 30, 40 percent of your total body surface. With our CellMist System, the doctor sprays the patient's own stem cells with a highly regenerative capacity onto the wound and, by doing so, creates tens of thousands of little regenerative islands across the wound. These islands grow outwards, ultimately connecting to each other to create a protective epithelial skin layer that covers the wound.

Experts believe the formation of this pink-colored layer marks the moment of re-epithelization where the risk of infection is reduced and the patient's wound is effectively healed. Beyond this stage, the cosmetic healing process also happens entirely natural to produce a scar-free result where, finally, skin color, tone and pigmentation are restored.

Since the RenovaCare spray procedure uses the patient's own stem cells, there isn't the risk of tissue rejection, infection, or ongoing immuno suppression therapy.

H.L.: What results have you found for patients using the SkinGun?

T.B.: We have many examples of patients recovering from severe burns within a week or two, scar-free, and walking away with unlimited joint restrictions.

In the case of one patient with severe electrical burns to over a third of his body, we were able to spray his wounds with 23 million stem cells isolated from a tiny two-inch-by two-inch sample of his own skin. Within five days of treatment, his chest and arms were already healed. Four days later, the patient was discharged from the hospital.

It's also important to note that reconstructive surgery for burn patients is especially challenging when tackling joints in the body. To this end, the authors of a case study in the reputable journal "Burns," said, "Cell-spray grafting is also especially suitable for hands and joint areas, where prolonged times to re-epithelization may significantly impact functionality and esthetic outcome."

H.L.: What different uses does the SkinGun have beside burns?

T.B.: Currently, we are focusing on severe second-degree burns, but we see the RenovaCare technology also applicable for other indications such as cosmetic procedures targeting skin pigmentation disorders, scar treatment, and other related conditions.

Our goal is to bring to market the world's most advanced technology for skin repair using a patient's own stem cells.

H.L.: Is there a record of the SkinGun use in the States and abroad?

T.B.: Having treated 72 burn patients to date, the company's early clinical target is burns with follow-on indications, including chronic wounds and cosmetic procedures.

H.L.: How much research went into creating the SkinGun and over what time period?

T.B.: The birth of RenovaCare technology goes back to the early 2000s in Berlin, Germany. Researchers, at that time, were trying to "grow" skin by seeding stem cells inside multi-dimensional bioreactors. They soon discovered that these artificial chambers were no match for the growth of the same cells when transplanted inside a human body; thus, the birth of a concept to use a patient's own wound as a natural bioreactor.

A study published in "Advances in Plastic Surgery" highlights 19 early patients with deep dermal wound burns to the face and neck, complex three-dimensional surfaces. Researchers achieved such outstanding results using our cell spray that they refused to perform further skin grafting. Instead, surgeons adopted our founding technology as their standard of care.

Let me quote from the surgeons' study, which states

"We refuse to perform a prospective randomized study with groups in which traditional skin grafting and/or wound healing are still applied for the therapy for deep dermal burns due to the excellent results in our study. The method of CEA spray application has become our standard of care for these indications. The faster wound closure, the promotion of spontaneous wound healing by keratinocyte application, as well as the preservation of donor sites are further advantages of the method."

The same paper concluded that "using a spray technique results in excellent cosmetic outcomes compared with any other method."

H.L.: How has the technology changed since then?

T.B.: Since the time of this early approach, our technology has evolved and matured significantly. Our cell isolation no longer requires complex procedures, culturing, expansion, and processing time, and our stem cell spray device no longer requires multiple hand-assembled parts. Its independent power and flow-control unit has been condensed in size from a 2-foot cube down to a 9-volt battery placed inside the handle of a single handheld spray gun.

H.L.: What is the potential market for the technology in dollars and number of patients?

T.B.: Conservatively speaking, the market for our technology exceeds $50 billion. There are nearly a million people who suffer from burns each year in the U.S. alone. According to the American Burn Association, burn injuries continue to be one of the leading causes of accidental death and injury in the U.S, and one civilian fire death occurs every two hours and forty minutes.

H.L.: How much would you estimate the treatment cost may be for each different use?

T.B.: The SkinGun technology is currently under development and not approved for clinical use in the U.S., so it's too early to talk about what the treatment will cost. We have always been mindful of reimbursement, and nearly two years ago, we commissioned an investigation into the reimbursement pathway for our CellMist System. We know that reimbursement opportunities are available by way of current coding and practices.

We have further investigated and evaluated the "bundling" approach currently advocated for by insurers and are confident that that our technology is well placed to take advantages of any shift towards such a model.

H.L: What is the schedule to get Federal Drug Administration clearance?

T.B.: In order to achieve FDA clearance for the CellMist System and the SkinGun, we will be working to show our technology is safe and prove its efficacy within applicable clinical trial formats and according to the relevant regulatory requirements. I can't speculate as to how long the FDA clearance process will take, and, therefore, it's hard to speculate when our product will be commercialized.

H.L.: What other products are you investigating and how may they work?

T.B.: We are focusing on bringing the SkinGun and our stem cell spray technology to market at this time.

H.L.: What is your background, including age, education, prior employment?

T.B.: Before joining RenovaCare I worked as the CEO of StemCell Systems GmbH, a Berlin-based biomedical company engaged in the development and commercialization of advanced cell culture bioreactors. I have more than 15 years of professional business experience in the field of medical biotechnology device manufacturing, stem cell culture technology platform development and regenerative medicine research project management and product development. I also co-founded several start-up companies in Germany.

Disclosure: I/we have no positions in any stocks mentioned, and no plans to initiate any positions within the next 72 hours.

I wrote this article myself, and it expresses my own opinions. I am not receiving compensation for it (other than from Seeking Alpha). I have no business relationship with any company whose stock is mentioned in this article.

Editor's Note: This article discusses one or more securities that do not trade on a major U.S. exchange. Please be aware of the risks associated with these stocks.

Patients Lose Sight After Stem Cells Are Injected Into Their Eyes – New York Times

By adminMarch 15, 2017Stell Cell Research

New York Times

Patients Lose Sight After Stem Cells Are Injected Into Their Eyes
New York Times
All three women found U.S. Stem Cell because it had listed a study on a government website, clinicaltrials.gov provided by the National Institutes of Health. Two later told doctors they thought they were participating in government-approved research ...
3 Women Blinded By Unproven Stem Cell TreatmentsNPR
Experimental stem cell treatment stabilizes macular degenerationCNN
Cutting-edge stem cell therapy proves safe, but will it ever be effective?Science Magazine
Inquirer.net -Kaiser Health News -EurekAlert (press release) -Bascom Palmer Eye Institute
all 44 news articles »

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Patients Lose Sight After Stem Cells Are Injected Into Their Eyes - New York Times

Longeveron Achieves Milestone in Groundbreaking Stem Cell Trial for Alzheimer’s Disease – PR Newswire (press release)

By adminMarch 15, 2017Stell Cell Research

"The impact of Alzheimer's disease is vast, far exceeding the medical community's current ability to treat it," said Joshua M. Hare, M.D., Longeveron's Co-Founder and Chief Science Officer. "Regenerative medicine and cell-based therapies offer a promising new approach to close this gap and address the urgent need for effective therapies to combat the condition."

An important component in the progression of Alzheimer's disease is neuroinflammation. Longeveron was recently awarded a $1 million Part the Cloud Challenge on Neuroinflammation grant from the Alzheimer's Association to help support this research.

"Adult stem cells are very potent anti-inflammatories. The characteristic amyloid plaques found in the brains of Alzheimer's disease patients produce inflammation, and stem cells can reduce inflammation," explained Bernard S. Baumel, M.D., Principal Investigator for the trial. "Alzheimer's also impairs the brain's ability to adequately produce new brain cells in the memory area known as the hippocampus. Stem cells can stimulate the brain to produce these new cells needed to form memory. We believe that an infusion of LMSCs may improve the condition or at least halt the progression of the disease."

Prior research shows that adult MSCs target and reduce inflammation, promote tissue repair and improve brain function in mouse models of Alzheimer's disease. Longeveron's trial is the first U.S. clinical study of exogenously administered mesenchymal stem cells derived from the bone marrow of healthy adult donors for treating Alzheimer's disease.

To learn about participating in the clinical trial, visit: https://clinicaltrials.gov/ct2/show/NCT02600130

About Longeveron

Longeveron is a regenerative medicine therapy company founded in 2014. Longeveron's goal is to provide the first of its kind biological solution for aging-related diseases, and is dedicated to developing safe cell-based therapeutics to revolutionize the aging process and improve quality of life. The company's research focus areas include Alzheimer's disease, Aging Frailty and the Metabolic Syndrome. Longeveron produces LMSCs in its own state-of-the-art cGMP cell processing facility. http://www.longeveron.com

Contact: Suzanne Liv Page spage@longeveron.com 305.342.9590

To view the original version on PR Newswire, visit:http://www.prnewswire.com/news-releases/longeveron-achieves-milestone-in-groundbreaking-stem-cell-trial-for-alzheimers-disease-300424206.html

SOURCE Longeveron

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Longeveron Achieves Milestone in Groundbreaking Stem Cell Trial for Alzheimer's Disease - PR Newswire (press release)

From skin to brain: Stem cells without genetic modification – Phys.Org

By adminMarch 15, 2017Stell Cell Research

March 15, 2017 by Grove Potter The four images, from left to right, show Keratinocyte-derive neural crest stem cells turning into neurons as shown by typical neuronal morphology. Credit: University at Buffalo.

A discovery, several years in the making, by a University at Buffalo research team has proven that adult skin cells can be converted into neural crest cells (a type of stem cell) without any genetic modification, and that these stem cells can yield other cells that are present in the spinal cord and the brain.

The practical implications could be very significant, from studying genetic diseases in a dish to generating possible regenerative cures from the patient's own cells.

"It's actually quite remarkable that it happens," says Stelios T. Andreadis, PhD, professor and chair of UB's Department of Chemical and Biological Engineering, who recently published a paper on the results in the journal Stem Cells.

The identity of the cells was further confirmed by lineage tracing experiments, where the reprogrammed cells were implanted in chicken embryos and acted just as neural crest cells do.

Stem cells have been derived from adult cells before, but not without adding genes to alter the cells. The new process yields neural crest cells without addition of foreign genetic material. The reprogrammed neural crest cells can become smooth muscle cells, melanocytes, Schwann cells or neurons.

"In medical applications this has tremendous potential because you can always get a skin biopsy," Andreadis says. "We can grow the cells to large numbers and reprogram them, without genetic modification. So, autologous cells derived from the patient can be used to treat devastating neurogenic diseases that are currently hampered by the lack of easily accessible cell sources."

The process can also be used to model disease. Skin cells from a person with a genetic disease of the nervous system can be reprogrammed into neural crest cells. These cells will have the disease-causing mutation in their chromosomes, but the genes that cause the mutation are not expressed in the skin. The genes are likely to be expressed when cells differentiate into neural crest lineages, such as neurons or Schwann cells, thereby enabling researchers to study the disease in a dish. This is similar to induced pluripotent stem cells, but without genetic modification or reprograming to the pluripotent state.

The discovery was a gradual process, Andreadis says, as successive experiments kept leading to something new. "It was one step at a time. It was a very challenging task that took almost five years and involved a wide range of expertise and collaborators to bring it to fruition," Andreadis says. Collaborators include Gabriella Popescu, PhD, professor in the Department of Biochemistry in the Jacobs School of Medicine and Biomedical Sciences at UB; Song Liu, PhD, vice chair of biostatistics and bioinformatics at Roswell Park Cancer Institute and a research associate professor in biostatistics UB's School of Public Health and Health Professions; and Marianne Bronner, PhD, professor of biology and biological engineering, California Institute of Technology.

Andreadis credits the persistence of his then-PhD student, Vivek K. Bajpai, for sticking with it.

"He is an excellent and persistent student," Andreadis says. "Most students would have given up." Andreadis also credits a seed grant from UB's office of the Vice President for Research and Economic Development's IMPACT program that enabled part of the work.

The work recently received a $1.7 million National Institutes of Health grant to delve into the mechanisms that occur as the cells reprogram, and to employ the cells for treating the Parkinson's-like symptoms in a mouse model of hypomyelinating disease.

"This work has the potential to provide a novel source of abundant, easily accessible and autologous cells for treatment of devastating neurodegenerative diseases. We are excited about this discovery and its potential impact and are grateful to NIH for the opportunity to pursue it further," Andreadis said.

The research is described in the journal Stem Cells under the title "Reprogramming Postnatal Human Epidermal Keratinocytes Toward Functional Neural Crest Fates."

Explore further: Embryonic gene Nanog reverses aging in adult stem cells

More information: Vivek K. Bajpai et al, Reprogramming Postnatal Human Epidermal Keratinocytes Toward Functional Neural Crest Fates, STEM CELLS (2017). DOI: 10.1002/stem.2583

Journal reference: Stem Cells

Provided by: University at Buffalo

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From skin to brain: Stem cells without genetic modification - Phys.Org

Study identifies molecular clues for age-related intestinal issues – Medical Xpress

By adminMarch 15, 2017Stem Cell Medical Center

March 14, 2017 These microscopic images show structural changes in the intestine of an older mouse approaching 2 years of age (right) compared to intestine from a young 2-3 month old mouse (left). The older intestine has a smaller number of intestinal crypts (mucosal glands) that are wider. Researchers report in Cell Reports that intestinal stem cells (ISCs) age along with the animals, causing detrimental changes to intestinal structures and their function. They also show function can be restored by reactivating the signaling of a key molecule lost in aging intestinal stem cells. Credit: Cincinnati Children's

Intestinal stem cells rejuvenate daily so bowels will stay healthy and function normally, but a new study in Cell Reports suggests they also age along with people and lose their regenerative capacity.

Reporting their data online March 14, researchers suggest that reactivating the signaling of a key molecule lost in aging intestinal stem cells could restore healthy intestinal function in older people. The study was conducted in mice and human intestinal organoids by scientists at Cincinnati Children's Hospital Medical Center and the Institute for Molecular Medicine, Stem Cells and Aging at the University of Ulm in Germany.

Researchers say their study is the first to indicate that intestinal stem cells - which allow different intestinal cell types to renew themselves - age as people do. It also is the first to provide clear evidence that diminished signaling in intestinal stem cells (ISCs) by the Wnt protein - which is important to cell proliferation and renewal - drives the aging process and loss of ISCs' ability to rejuvenate intestinal tissues.

"Deterioration of Wnt signaling in aging intestinal stem cells may help explain imbalances in food and nutrient absorption in older people, but our paper also shows that biology allows us to reverse this process by restoring Wnt signaling," said Hartmut Geiger, PhD, senior investigator of the study. "Pharmacologic interventions will still need to be designed based on this finding, but we know now where to start."

Geiger is a member of the Division of Experimental Hematology and Cancer Biology at Cincinnati Children's.

Old vs. Young

In tests that compared the intestines of young mice (2 to 3 months old) with those of aged mice (approaching 2 years old), researchers noticed significant differences in the structural architecture of intestine.

Intestines from older mice showed a reduced number of crypts (mucosal glands), and the structures were longer and wider than in younger animals. Older intestines also had longer villi (absorptive tissues) and an elevated number of cells. But the researchers also noticed there were fewer numbers of actively dividing cells in the intestines of the older mice.

Study authors then looked for different characteristics in the ISCs of young and old mouse intestines. Although they did not observe differences in the numbers of ISCs, they did see a decreased presence of molecular markers that denote ISC function. This led them to conclude that ISC function diminishes in older intestines.

Finding Wnt

To identify genetically driven molecular processes that prompt aging and diminished function in ISCs (and in Paneth and goblet cells they form) the researchers subjected ISCs to RNA sequencing analysis. These tests showed significant reductions in gene expression in the older cells, including the down regulation of molecular pathways involving the genes PPAR, SMAD and Wnt.

Because Wnt plays a particularly prominent role in regulating ISCs, the researchers focused on this pathway. In young and old mice they tested the presence and strength of the Wnt signaling pathway in cells of the mesenchyme - which forms connective and skeletal tissues. The mesenchyme has recently been identified in other studies as providing a supportive environment for ISCs to form.

The authors also tested Wnt signaling in different intestinal cells from young and old mice, specifically goblet and Paneth cells that secrete proteins and antimicrobial peptides that are important to sustaining healthy intestines. In both instances they observed a decline in Wnt in intestinal tissues from the older animals.

In testing on lab-generated human and mouse intestinal organoids, the researchers also observed declines in Wnt signaling in older intestines along with altered intestinal structures and indications of diminished function.

Molecular restoration

Researchers concluded their study by testing the restoration of Wnt signaling in intestines by adding Wnt3a (an inducer of Wnt signaling) in aged mouse and human organoid models, which were generated with donated human cells. This rejuvenated the regenerative potential of ISCs in the mouse and human modeling systems. It also boosted the generation of Paneth and goblet cells and prompted an increase in the number of intestinal crypts and other critical structures.

The scientists are following up the current study by trying to define the extent to which therapeutic intervention to rejuvenate ISCs might be beneficial to humans.

Explore further: Scientists wage fight against aging bone marrow stem cell niche

More information: "Canonical Wnt Signaling Ameliorates Aging of Intestinal Stem Cells" Cell Reports, DOI: 10.1016/j.celrep.2017.02.056, http://www.cell.com/cell-reports/fulltext/S2211-1247(17)30254-1

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Study identifies molecular clues for age-related intestinal issues - Medical Xpress

City of Hope Hosts Second California Institute for Regenerative Medicine Alpha Stem Cell Clinics Symposium – Business Wire (press release)

By adminMarch 15, 2017Stem Cell Clinic

DUARTE, Calif.--(BUSINESS WIRE)--The stories of patients who have benefited from stem cell therapies funded by the California Institute for Regenerative Medicine (CIRM) will be highlighted at the second symposium of the institutes Alpha Stem Cell Clinics Network on Thursday, March 23, at City of Hope.

CIRM, the states stem cell agency, has developed a network of Alpha Stem Cell Clinics that are focused on innovative stem cell-based therapies. The network, which consists of three clinics located at City of Hope, UCSD, and UCLA/UC Irvine, will showcase its most successful clinical trials at the daylong symposium.

Scientists, clinicians, patient advocates, and business and insurance experts, as well as CIRM and City of Hope representatives, are hosting the meeting to address various aspects of making more stem cell therapies a reality for patients.

CIRMs Alpha Clinics are working day in and day out to find cures for such debilitating diseases as glioblastoma, leukemia and other cancers, as well as blinding eye diseases, spinal cord injuries, HIV, hemophilia, diabetes and other catastrophic conditions for which there are no current cures, said John A. Zaia, M.D., the Aaron D. Miller and Edith Miller Chair in Gene Therapy, who is also director of the Center for Gene Therapy and principal investigator of City of Hopes Alpha Stem Cell Clinic. This symposium brings us together to share the advancements weve made in delivering stem cell treatments to patients, and to discuss how we can accelerate their development.

Highlights of Alpha Clinics: Making the Future a Reality for Stem Cell-Based Therapies

Registration starts at 7 a.m. There will be a welcome address at 8 a.m. with panels concluding at 5 p.m. View the schedule here.

About City of Hope

City of Hope is an independent research and treatment center for cancer, diabetes and other life-threatening diseases. Designated as one of only 47 comprehensive cancer centers, the highest recognition bestowed by the National Cancer Institute, City of Hope is also a founding member of the National Comprehensive Cancer Network, with research and treatment protocols that advance care throughout the world. City of Hope is located in Duarte, California, just northeast of Los Angeles, with communityclinics throughout Southern California. It is ranked as one of "America's Best Hospitals" in cancer by U.S. News & World Report. Founded in 1913, City of Hope is a pioneer in the fields of bonemarrowtransplantation, diabetes and numerous breakthrough cancer drugs based on technology developed at the institution. For more information about City of Hope, follow us on Facebook, Twitter, YouTube or Instagram.

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City of Hope Hosts Second California Institute for Regenerative Medicine Alpha Stem Cell Clinics Symposium - Business Wire (press release)

The tip of the iceberg: A look at stem cell science at Stanford – Scope (blog)

By adminMarch 15, 2017Stem Cell Clinic

For outsiders, its easy to get confused about the status of stem cell science. If you have questions, a recentStanford Magazine article offers a reader-friendlyentre.

The piece introduces the pioneering work of Irving Weissman, MD, who was the first researcher to isolate stem cells from adult tissue. And it highlights the amazing outcomefrom a recent clinic trial on stem cell therapy for stroke led by neurosurgeonGary Steinberg, MD, PhD. One woman who used a wheelchair often before the procedure no longer needed it following the procedure. As Steinberg saidin the article:

This was a huge surprise We thought the circuits were dead or irreversibly injured, but theyre clearly not; they can be resurrected. Were still trying to figure out exactly how we are resurrecting them.

Maria Grazia Roncarolo, MD, co-director of theInstitute of Stem Cell Biology and Regenerative Medicine, is one of the researchers working to bring the innovative basic science work taking place at Stanford into the clinic.

My mission is to help the university to build a brand in stem cell therapy, sheexplained. What we are now pushing into the clinic is just the tip of the iceberg But what we really want to build is a translational research program that would allow all the potential stem cell therapies to surface and be offered to patients in need.

Previously: Stem cells create faithful replicas of native tissue, according to Stanford study, Stroke of luck: Stem-cell transplants show strong signs of efficacy in clinical safety trial for strokeand Stanford scientists describe stem-cell and gene-therapy advances in scientific symposium Image by Ghanson

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The tip of the iceberg: A look at stem cell science at Stanford - Scope (blog)