Category Archives: Stell Cell Research


One stem cell treatment stabilizes macular degeneration, another blinds 3 patients – CNN

The macula is the spot in the center of your eye's retina. When that tissue begins to thin and break down, this is referred to as macular degeneration, a blurring of the sharp central vision necessary for driving, reading and other close-up work. Most people develop this disease as they age.

For the latest study, researchers led by Dr. Michiko Mandai of the laboratory for retinal regeneration at RIKEN Center for Developmental Biology in Japan tested an experimental stem cell treatment on a 77-year-old woman diagnosed with "wet," or neovascular age-related, macular degeneration.

The "wet" form of the disease involves blood vessels positioned underneath the pigment epithelium (a layer of retinal cells) growing through the epithelium and harming the eye's photoreceptor cells. In Japan, wet age-related macular degeneration is the most common form, but in Caucasian populations, only about 10% of people with age-related macular degeneration gets that form.

The "dry" form involves the macula breaking down without growth of blood vessels where they're not supposed to be.

To stop the progress of wet macular degeneration, the researchers performed surgery to transplant a sheet of retinal pigment epithelial cells under the retina in one of the patient's eye.

The transplanted cells had been derived from autologous induced pluripotent stem cells, which are reprogrammed cells. They were created using cells from the connective tissue of the woman's skin.

One year after surgery, the transplanted sheet remained intact, and there was no evidence of lasting adverse effects. Although the patient showed no evidence of improved eyesight, her vision had stabilized.

"This research serves multiple purposes," wrote Peter Karagiannis, a science writer, in an email on behalf of Dr. Shinya Yamanaka, the Nobel Prize-winning co-author of the study and director of the Center for iPS Cell Research and Application at Kyoto University. From the patient's perspective, the study shows that induced pluripotent stem cells can alleviate the problems associated with age-related macular degeneration.

"From a greater medical perspective, however, the bigger impact is that it shows iPS cells can be used as cell therapies," the email said, adding that newly initiated stem cell research applications at the center include Parkinson's disease and thrombocytopenia, a lack of platelets in the blood.

The American story, like the Japanese story, begins with patients slowly losing their sight as a result of macular degeneration -- in this case, three women ages 72 to 88, two of whom had the "dry" form.

Each patient paid $5,000 for the procedure at an unnamed clinic in Florida, the authors noted. Some of the patients, including two of the three women described in the paper, learned of the so-called clinical trial on ClinicalTrials.gov, a registry database run by the US National Library of Medicine. However, the consent form and other written materials did not mention a trial.

The procedure took less than an hour and began with a standard blood draw and the removal of fat cells from each patient's abdomen. To obtain stem cells, the fat tissue was processed with enzymes, while platelet-dense plasma was isolated from the blood. The stem cells were mixed with the plasma and injected into both eyes.

Complications may have been caused by contamination during stem cell preparation, or the stem cells might have changed into myofibroblasts, a type of cell associated with scarring, after injection, the authors wrote.

Before the surgery, the women's vision ranged from 20/30 to 20/200. After treatment and complications, the patients were referred in June 2015 to two university-based ophthalmology practices, including the University of Miami, where lead author Dr. Ajay E. Kuriyan was practicing.

"Many stem-cell clinics are treating patients with little oversight and with no proof of efficacy," Kuriyan and his co-authors wrote in the paper, acknowledging that it is difficult for patients to know whether a stem cell therapy -- or a clinical trial -- is legitimate.

One red flag is that the patients were required to pay for their procedure; another is that both eyes were treated at once, the authors said. Legitimate clinical trials do not require payment, and for any experimental treatment of the eyes, a good doctor would observe how one eye responds before attempting the second eye.

Another problem for unsavvy patients: Listings on ClinicalTrials.gov are not fully scrutinized for scientific soundness, noted the authors.

Today, the clinic is no longer performing these eye injections, the authors said, but it is still seeing patients. In October 2015, months after the procedures had been performed, the Food and Drug Administration released more specific guidelines for stem cell treatments.

Writing on behalf of the FDA in an editorial alongside the paper, Drs. Peter W. Marks, Celia M. Witten and Robert M. Califf say there's an absence of compelling evidence, yet some practitioners argue that stem cells have a unique capacity to restore health because of their ability to differentiate into whatever cell is necessary for repairing a defect. Another argument is that clinical trials are too complex for all except large industrial sponsors.

Despite the shadow cast by some stem cell experiments, the Japanese study earned praise from the scientific community.

Michael P. Yaffe, vice president of scientific programs at the New York Stem Cell Foundation Research Institute, said the RIKEN study was "incredibly thorough, careful and well-documented."

"Many experts in the field of regenerative medicine believe that the treatment of macular degeneration and other retinal diseases will be among the first areas of success in the use of stem cell-derived tissues," said Yaffe, whose foundation was not involved in the RIKEN study.

Yaffe said this optimism stems from preliminary studies using retinal cells derived from stem cells in animals. Scientists are also hopeful because the procedures to generate pure cells of the correct type and surgical techniques necessary for transplantation have already been developed.

"A number of research groups are moving toward developing stem cell-based treatments for age-related macular degeneration and other retinal diseases," Yaffe said.

The National Eye Institute at the National Institutes of Health is planning a similar study using patient-specific pluripotent stem cells, according to Kapil Bharti, a Stadtman Investigator in the Unit on Ocular Stem Cell & Translational Research at the institute. After getting approval to conduct a phase I safety trial, the institute will treat 10 to 12 patients to check safety and tolerability of stem cell-based eye tissue transplants.

"Data from 10 to 12 patients is needed to show that the implanted cells are indeed safe," he said, adding that the trial is likely to begin in 2018.

"While researchers have used embryonic stem cell derived cells to treat age-related macular degeneration, (the RIKEN study) is the first study that used induced pluripotent stem cells," said Bharti, who was not involved in the research.

Both induced pluripotent stem cells and embryonic stem cells can be used to make other kinds of cells of the body, explained Bharti. However, induced pluripotent stem cells can be derived from adult skin or blood cells, rather than from embryos.

"Another big scientific advantage with induced pluripotent stem cells is that they can be made patient-specific (because it's the patient's own cells), reducing the chances of tissue rejection," he said.

P. Michael Iuvone, a professor of ophthalmology and director of vision research at Emory University School of Medicine, also noted the importance of using the patient's own stem cells.

Past studies have used embryonic stem cells to treat age-related macular degeneration, but there were problems related to rejection, when the body refuses to accept a transplant or graft, explained Iuvone, who was not involved in the latest study. In the new RIKEN study, the researchers took the patient's own cells and converted them into retinal cells to avoid these complications.

"The results from the standpoint of the graft taking and surviving without any signs of any kind of toxicity or tumorigenicity are very positive," Iuvone said. "But the weakness is, they only had one patient, and it's very difficult to make any conclusions from one patient."

He noted that the RIKEN researchers planned to work with more patients, but in 2014, the Japanese government passed a law that said regenerative medicine clinical trials could be performed only at medical institutions, not at research institutions such as RIKEN.

Though the experiment was performed on a woman with wet age-related macular degeneration, it also might be useful for "dry" age-related macular degeneration, which is more common in the United States, according to Iuvone.

Currently, there are some effective treatments for age-related macular degeneration.

"The standard of care in most cases is to give injections of drugs that inhibit the growth hormones that is called vascular epithelial growth factor, or VEGF," Iuvone said. "For most people, it at least slows the progression and in some cases actually improves visual acuity."

Laser treatments have also been used but are on the decrease because of side effects. "Given the fact that the VEGF treatments seem to be effective, I think that most clinicians have turned to that," Iuvone said.

Bharti believes the RIKEN study is a major milestone in the field. "We and others are learning from the Japan study," he said.

Susan L. Solomon, CEO of the New York Stem Cell Foundation Research Institute, agrees.

"This study represents a fundamental advance in regenerative medicine, in the use of stem cell-derived tissues and in the treatment of eye disease," she said. However, additional work and many more studies are needed, she said, before a safe and efficacious stem cell-based treatment will be available "to the broad and growing population with retinal disease" -- all of us, growing older.

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One stem cell treatment stabilizes macular degeneration, another blinds 3 patients - CNN

Transform 815: Xue Jun-Li’s stem cell research in Rockford sheds light on motor neuron diseases – Rockford Register Star

By Isaac GuererroStaff writer

Xue-Jun Li spends her days studying motor neurons, the cells that send signals from the brain to the body's voluntary muscles.

Motor neuron diseases are degenerative, which means that the muscles don't stop working all at once. When the motor neurons begin to fail, one slowly loses control of the arms and legs, the ability to swallow and, eventually, to breathe.

Scientists know very little about the human nervous system and what causes the motor neurons to stop functioning. Li has published more than 37 peer-reviewed manuscripts and has developed human stem cell models for spastic paraplegias and spinal muscular atrophy, which is the leading genetic cause of death for infants.

Li joined the University of Illinois College of Medicine at Rockford as an associate professor in the Biomedical Sciences Department last May. Her research is supported by the National Institute of Health and ALS Association, among others.

I love the satisfaction of new discoveries, new ideas you get from working in the lab, the interaction with students, she said. But any breakthrough you make is a collaboration.

Her stem cell research is concerned with, among other things, an attempt to better understand microtubule defects that disrupt signals from the brain to motor neurons along the spinal cord. Mitochondrial defects the mitochondria are the energy factories of the cells are another focus.

This fall, Li will teach a class that's a first for the university: stem cells and regenerative engineering.

Isaac Guerrero: 815-987-1361; iguerrero@rrstar.com;@isaac_rrs

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Transform 815: Xue Jun-Li's stem cell research in Rockford sheds light on motor neuron diseases - Rockford Register Star

Strip mall stem cells – HealthNewsReview.org

Michael Joyce is a multimedia producer with HealthNewsReview.org and tweets as @mlmjoyce.

If you had been in Salt Lake City last month, savoringyour morningcoffee, and watching this channel 4 morning show, you might have been treated to this videopromising a revolutionary treatment that assures you dont have to live with back pain.

Youd also be assured that the doctor being interviewed, Dr. Kahn, works at the only place in Utah that does this particular type of stem cell procedure.

But had you found this story online as Dr. Leigh Turner did, and tweeted it our way you might have noticed, tucked way down atthe bottom of the page, thislonely line: This story includes sponsored content (the video didnt mention this at any point during its 5-minute run time, though its possible the broadcast version was preceded by a disclaimer).

Dr. Turner is a bioethicist at the University of Minnesota who, along with studying direct-to-consumer marketing and medical tourism, has a special interest in unproven stem cell therapies. Last summer he co-authored a paper which documented 351 companies across the United States marketing unapproved & unlicensed stem cell procedures. Dr. Khans Wasatch Pain Solutions is one of these businesses. A quick glance at the companys websiteoffers this:

If that is the case then why is Dr. Kahn using an unlicensed, non-FDA-approved treatment that is not supported by controlled research?

The market for these unregulated, unapproved, and unproven stem cell therapies is expanding, says Dr. Leigh Turner. And the list of conditions they claim to treat has expanded to 30 or 40 different diseases. In reflecting on the promotionalcontent of the video, Turner also noticed something else disturbing:

What strikes me in watching the video is just how commonplace a video like thishas become. These businesses and their claims are everywhere, and people dont even seem to blink anymore. Its just become part of the consumer landscape.

And what about the media landscape? A quick glance at the KUCW/KTVX- Channel 4 website reveals foursponsored videos in just the past fourmonths from Dr. Khans Wasatch Pain Solutions ALL promoting the use of the Regenexx stem cell procedures. I ask Turner what he thinks of the media coverage of stem cell interventionsin general.

The media coverage of these unproven stem cell treatments has been highly variable, says Turner. Ive seen insightful and critical examples of investigative reporting that deserve credit. But then there are videos like this one that can lead to misinformation. Its not asking the questions that need to be asked. You cant just sit back and allow someone to make these claims and assertions and not ask the questions that might help your viewers.

And Turner is right. There is some great writing out there. Like this piece by Julia Belluz of Vox warning of the consequences of hyping unproven stem cell research. And another compelling read published today by BuzzFeed which takes on the issue of harm head-on. News storiesthat dont do such a great job tend toward the anecdotal and feature some of the more wildly speculative uses of stem cells in treating things like Alzheimers disease, multiple sclerosis, or even the damaged throwing arms of major league pitchers. But even some of these articles do well in bringing up important issues likeoutrageous treatment costs, lack of FDA approval, possible placebo effects, pervasive skepticism within the medical community at large, and the availability ofwell-established alternative therapies. But many reporterscould do a much better job in simply asking for data. Asking about conflicts of interest. Asking about risks. And simplyfollowing the money. We offer a primer to help journalists address many of these issues.

But beyond the strip mall stem cell injectionsfor whatever ails you, and journalists asking tough questions or not asking any, is perhaps something even more disconcerting. Again, Dr. Leigh Turner:

If this was just about a few businesses here and there I could see them flying under the radar. But when you have 351 of them as our study shows then it suggests a much bigger problem that raises bigger questions. Where is the FDA? Where is the FTC? What about assuring truthfulness, accuracy and honesty in advertising toconsumers? And where are the state medical boards? When you have doctors making promotional claims not backed up by evidence, and who are performing procedures that are not part of the contemporary, evidence-based practice of medicine, then we clearly need external regulators involved who are making decisions based on evidence and not based on financial gain. Where are they?

Lets be fair here.Whether the setting is strip malls or ivory towers is not the issue; the issue is evidence. I think the strip mall settings of many of these businesses speak more to the burgeoning commodification of stem cell therapies than anything else. But the evidence that these stem cells as many practitioners claim are somehow intrinsically capable of sensing the environment they are injected into, and precisely targetwhatever functions require repair, is not proven. Complex cellular systems dont operate that way, as correctly pointed out by FDA scientists in this opinion piece published in todays New England Journal of Medicine.

It is possible some of these stem cell therapies may eventually prove to be safe. Or even efficacious. But until that is clarified with high quality research, the 5 Ws &1H of journalism should be applied to every single story about these unproven stem cell therapies: WHERE is the data? WHATare the real risks and benefits of this therapy? WHY arent alternative therapies being discussed? WHO is making the money here and are there conflicts of interest at play? WHEN will see scientific studies backing revolutionaryclaims and why dont you have them now?

And we end with HOW? How can our state and federal regulators ignore this? How can journalists not give into the hyperbole for clicks and ratings, and instead choose hard questions seekingreal information? And how can we as consumers stop falling for this misleading infotainment and go about demanding more integrity from our health care providers and regulators?

I dont think the answers will come from your local strip mall.

The segment on the WFAA-TV, Dallas, Good Morning Texas program, was headlined "The latest on

Joy Victory is deputy managing editor of HealthNewsReview.org. She tweets as @thejoyvictory. At this moment,

Joy Victory is deputy managing editor of HealthNewsReview.org. She tweets as @thejoyvictory. Lastweek about a

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Strip mall stem cells - HealthNewsReview.org

Some genetic variations difficult to evaluate using current stem cell modeling techniques – Science Daily

Some genetic variations difficult to evaluate using current stem cell modeling techniques
Science Daily
Some heritable but unstable genetic mutations that are passed from parent to affected offspring may not be easy to investigate using current human-induced pluripotent stem cell (hiPSC) modeling techniques, according to research conducted at The Icahn ...

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Some genetic variations difficult to evaluate using current stem cell modeling techniques - Science Daily

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

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

The fountain of youth may reside in an embryonic stem cell gene named Nanog.

Caltech scientists have converted cells of the lower-body region into facial tissue that makes cartilage, in new experiments using bird embryos. The researchers discovered a "gene circuit," composed of just three genes, that ...

Scientists at the University of Newcastle, UK, have used a combination of small molecules to turn cells isolated from human skin into Schwann cells - the specialised cells that support nerves and play a role in nerve repair. ...

Johns Hopkins stem cell biologists have found a way to reprogram a patient's skin cells into cells that mimic and display many biological features of a rare genetic disorder called familial dysautonomia. The process requires ...

(Phys.org)A team of researchers affiliated with New York and Dalhousie Universities, in the U.S. and Canada respectively, has found a possible intermediate cell type that might help understand the evolutionary process ...

German researchers succeed in obtaining brain and spinal cord cells from stem cells of the peripheral nervous system.

Scientists at the University of North Carolina at Chapel Hill have pinpointed a key genetic switch that helps soil bacteria living on and inside a plant's roots harvest a vital nutrient with limited global supply. The nutrient, ...

So they can't use smartphones or WiFi, but bacteria have evolved some seriously complex strategies to communicate with one another. And the resulting interactions are a delicate balance of cooperation and, in some cases, ...

New research led by the UK's Centre for Ecology & Hydrology has revealed for the first time that flower-rich habitats are key to enhancing the survival of bumblebee families between years.

The evolution of land animals has been shaped by barriers such as oceans and mountains which have divided them and sent them down different genetic paths.

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 ...

(Phys.org)A trio of researchers with Anglia Ruskin University in the U.K. and the Australian National University has found that the male fiddler crab uses its oversized claw to get the attention of a prospective mate and ...

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

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


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)

"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)

Some Genetic Variations Difficult to Evaluate Using Current Stem Cell Modeling Techniques – Bioscience Technology

Some heritable but unstable genetic mutations that are passed from parent to affected offspring may not be easy to investigate using current human-induced pluripotent stem cell (hiPSC) modeling techniques, according to research conducted at The Icahn School of Medicine at Mount Sinai and published March 14, in the journalStem Cell Reports. The study serves to caution stem cell biologists that certain rare mutations, like the ones described in the study, are difficult to recreate in laboratory-produced stem cells.

Stem cell-based disease modeling involves taking cells from patients, such as skin cells, and introducing genes that reprogram the cells into human-induced pluripotent stem cells (hiPSCs). These "master cells" are unspecialized, meaning they can be pushed to become any type of mature cell needed for research, such as skin, liver or brain. The hiPSCs are capable of renewing themselves over a long period of time, and this emerging stem cell modeling technique is helping elucidate the genetic and cellular mechanisms of many different disorders.

"Our study describes how a complex chromosomal rearrangement genetically passed by a patient with psychosis to her affected son was not well recreated in laboratory-produced stem cells," says Kristen Brennand, Ph.D, Associate Professor of Genetics and Genomic Sciences, Neuroscience, and Psychiatry at the Icahn School of Medicine, and the study's senior investigator. "As stem cell biologists dive into studying brain disorders, we all need to know that this type of rare mutation is very hard to model with induced stem cells."

To investigate the genetic underpinnings of psychosis, the research team used hiPSCs from a mother diagnosed with bipolar disease with psychosis, and her son, diagnosed with schizoaffective disorder. In addition to the normal 46 chromosomes (23 pairs), the cells in mother and son had a very small extra chromosome, less than 1/10th normal size. This microduplication of genes is increasingly being linked to schizophrenia and bipolar disorders, and the extra chromosomal bit, known as a marker (mar) element, falls into the category of abnormally duplicated genes.

For the first time, the Mount Sinai research team tried to make stem cells from adult cells with this type of mar defect. Through the process, they discovered that the mar element was frequently lost during the reprogramming process.

While mar elements in the general population are rare (less than .05 percent in newborn infants), more than 30 percent of individuals with these defects are clinically abnormal, and mar elements are also significantly more likely to be found in patients with developmental delays.

The study found that the mother's cells were mosaic, meaning some cells were normal while others were not, and the hiPSCs the team created accurately replicated that condition: some were normal and some had the extra mar chromosome. But the technique did not work well with the son's cells. While all of his cells should have had the mar element, as with his mother, some of the reprogrammed stem cells did not contain the extra bit of chromosome.

"We realized we kept losing the mutation in the stem cells we made, and the inability to recreate cells with mar elements may hamper some neuropsychiatric research," says Dr. Brennand. "The bottom line is that it is essential that stem cell biologists look for existing mar elements in the cells they study, in order to check that they are retained in the new stem cells."

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Some Genetic Variations Difficult to Evaluate Using Current Stem Cell Modeling Techniques - Bioscience Technology

Vitamin C can target and kill cancer stem cells, study shows – Medical News Today

Cancer is currently one of the top killers worldwide, and the number of cancer cases is only expected to rise. Although there are a number of therapies available, most of them are toxic and cause serious side effects. New research examines the impact of the natural vitamin C on cancer cell growth.

Cancer is the second leading cause of death and disease worldwide, accounting for almost 9 million deaths in 2015, according to the World Health Organization (WHO).

The global number of new cases of cancer are expected to grow by around 70 percent in the next 20 years.

In the United States, the National Cancer Institute (NCI) estimate that almost 40 percent of U.S. men and women will have developed cancer at one point during their lives.

There are various treatment options available for cancer, but they are not always effective; most of them are toxic, and they tend to have a variety of side effects.

In some more aggressive cases, the cancer does not respond to treatment, and it is believed that cancer stem-like cells are the reason why the cancer comes back and metastasizes.

New research, published in the journal Oncotarget, examines the effectiveness of three natural substances, three experimental drugs, and one clinical drug in stopping the growth of these cancer stem cells (CSCs.)

The study was conducted by researchers from the University of Salford in Manchester in the United Kingdom, and was led by Dr. Gloria Bonuccelli.

In total, the researchers measured the impact of seven substances: the clinical drug stiripentol, three experimental drugs (actinonin, FK866, and 2-DG), and three natural substances (caffeic acid phenyl ester (CAPE), silibinin, and ascorbic acid (vitamin C).)

The research focused on the bioenergetic processes of CSCs, which enable the cells to live and multiply. The study aimed to disrupt the CSCs' metabolism and ultimately prevent their growth.

Of all the substances tested, the team found that actinonin and FK866 were the most effective. However, the natural products were also found to prevent the formation of CSCs, and vitamin C was 10 times more effective than the experimental drug 2-DG.

Additionally, the study revealed that ascorbic acid works by inhibiting glycolysis - the process by which glucose is broken down within the cell's mitochondria and turned into energy for the cell's proliferation.

Dr. Michael P. Lisanti, professor of translational medicine at the University of Salford, comments on the findings:

"We have been looking at how to target cancer stem cells with a range of natural substances including silibinin (milk thistle) and CAPE, a honey-bee derivative, but by far the most exciting are the results with vitamin C. Vitamin C is cheap, natural, nontoxic and readily available so to have it as a potential weapon in the fight against cancer would be a significant step."

"This is further evidence that vitamin C and other nontoxic compounds may have a role to play in the fight against cancer," says the study's lead author.

"Our results indicate it is a promising agent for clinical trials, and as an add-on to more conventional therapies, to prevent tumor recurrence, further disease progression, and metastasis," Bonuccelli adds.

Vitamin C has been shown to be a potent, nontoxic, anticancer agent by Nobel Prize winner Linus Pauling. However, to the authors' knowledge, this is the first study providing evidence that ascorbic acid can specifically target and neutralize CSCs.

Learn how 300 oranges' worth of vitamin C can impair cancer cells.

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Vitamin C can target and kill cancer stem cells, study shows - Medical News Today

Stem cell reprogramming factor controls change in cellular energy generation – Science Daily

Stem cell reprogramming factor controls change in cellular energy generation
Science Daily
Now, research led by the University of Tsukuba has solved the mystery surrounding one of the reprogramming factors, KLF4. The study was published in Stem Cell Reports. KLF4 together with other reprogramming transcription factors is used in the lab to ...

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Stem cell reprogramming factor controls change in cellular energy generation - Science Daily