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OCASCR scientists make progress in TSET-funded adult stem cell … – NewsOK.com

By Stell Cell Research

OCASCR scientist Lin Liu at work. Photo provided.

Working together, scientists from Oklahoma State University, the University of Oklahoma Health Sciences Center and the Oklahoma Medical Research Foundation are advancing adult stem cell research to treat some of todays most devastating diseases.

Under the umbrella of the Oklahoma Center for Adult Stem Cell Research (OCASCR), created with funding from the Oklahoma Tobacco Settlement Endowment Trust, these scientists have amassed groundbreaking findings in one of the fastest growing areas of medical research.

We have made exciting progress, said OCASCR scientist Lin Liu, director of the Oklahoma Center for Respiratory and Infectious Diseases and director of the Interdisciplinary Program in Regenerative Medicine at Oklahoma State University.

We can convert adult stem cells into lung cells using our engineering process in petri dishes, which offers the possibility to repair damaged lung tissues in lung diseases, said Liu, whose research primarily focuses on lung and respiratory biology and diseases.

Using our engineered cells, we can also reverse some pathological features. These studies give us hope for an eventual application of these cells in humans.

Adult stem cells in the body are capable of renewing themselves and becoming various types of cells.

Until recently, stem cell treatments were largely restricted to blood diseases. However, new studies suggest many other types of adult stem cells can be used for medical treatment, and the Oklahoma Center for Adult Stem Cell Research was created to promote this branch of research.

OCASCR scientist Lin Liu and his team discussing their work. Photo provided.

Liu said the discipline provides hope for many ailments.

What most fascinated me in stem cell research is the hope that we may be able to use stem cells from our own body; for example, bone marrow or fat tissues to cure lung diseases, Liu said.

It is impossible to know exactly which diseases will respond to treatments.However, results of early experiments suggest many diseases should benefit from this type of research, including lung, heart, Alzheimers and Parkinsons diseases, as well as cancer, diabetes and spinal cord injuries. The field is often referred to as regenerative medicine, because of the potential to create good cells in place of bad ones.

While the application of stem cells can be broad, Liu hopes that his TSET-funded work will help develop treatments for diseases caused by tobacco use.

The goal of my research team is to find cures for lung diseases, Liu said. One such disease is chronic obstructive pulmonary disease (COPD).

COPD is the third leading cause of death in the country and cigarette smoking is the leading cause of COPD.

Cigarette smoking is also a risk factor for another fatal lung disease, idiopathic pulmonary fibrosis (IPF), which has a mean life expectancy of 3 to 5 years after diagnosis, he added.

There is no cure for COPD or IPF. The current treatments of COPD and IPF only reduce symptoms or slow the disease progression.

Using OCASCR/TSET funding, my team is researching the possibility to engineer adult stem cells using small RNA molecules existing in the body to cure COPD, IPF and other lung diseases such as pneumonia caused by flu, Liu said.

This is vital research, considering that more than11 million peoplehave been diagnosed with COPD, but millions more may have the disease without even knowing it, according to the American Lung Association.

Despite declining smoking rates and increased smokefree environments, tobacco use continues to cause widespread health challenges and scientists will continue working to develop treatments to deal with the consequences of smoking.

We need to educate the public more regarding the harms of cigarette smoking, Liu said. My research may offer future medicines for lung diseases caused by cigarette smoking.

Under the umbrella of the Oklahoma Center for Adult Stem Cell Research (OCASCR), created with funding from the Oklahoma Tobacco Settlement Endowment Trust, these scientists have amassed groundbreaking findings in one of the fastest growing areas of medical research. Photo provided.

Liu has been conducting research in the field of lung biology and diseases for more than two decades.

However, his interests in adult stem cell therapy began in 2010 when OCASCR was established through a grant with TSET, which provided funding to Oklahoma researchers for stem cell research.

I probably would have never gotten my feet into stem cell research without OCASCR funding support, he said. OCASCR funding also facilitated the establishment of the Interdisciplinary Program in Regenerative Medicine at OSU.

These days, Liu finds himself fully immersed in the exciting world of adult stem cell research and collaborating with some of Oklahomas best scientific minds.

Dr. Liu and his colleagues are really thriving. It was clear seven years ago that regenerative medicine was a hot topic and we already had excellent scientists in the Oklahoma, said Dr. Paul Kincade, founding scientific director of OCASCR. All they needed was some resources to re-direct and support their efforts. OSU investigators are using instruments and research grants supplied by OCASCR to compete with groups worldwide. TSET can point to their achievements with pride.

The Oklahoma Center for Adult Stem Cell Research represents collaboration between scientists all across the state, aiming to promote studies by Oklahoma scientists who are working with stem cells present in adult tissues.

The center opened in 2010 and has enhanced adult stem cell research by providing grant funding for researchers, encouraging recruitment of scientists and providing education to the people of Oklahoma.

We are fortunate that the collaboration at the Oklahoma Center for Adult Stem Cell Research is yielding such positive results, said John Woods, TSET executive director. This research is leading to ground breaking discoveries and attracting new researchers to the field. TSET is proud to fund that investments for Oklahomans.

Funding research is a major focus for TSET and it comes with benefits reaching beyond the lab. For every $1 TSET has invested at OCASCR, scientists have been able to attract an additional $4 for research at Oklahoma institutions, TSET officials said.

TSET also supports medical research conducted by the Stephenson Cancer Center and the Oklahoma Tobacco Research Center.

For more information, visit http://www.ocascr.org.

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OCASCR scientists make progress in TSET-funded adult stem cell ... - NewsOK.com

Newborn rats get mature hearts by serving as stem cell laboratory – RT

By Stell Cell Research

When it comes to treating heart disease, researchers are hoping that stem cells could prove to be a breakthrough. When immature heart muscle cells were implanted into newborn rats, mature adult cells developed, a new study has found.

Researchers at the Johns Hopkins University School of Medicine successfully injected about 200,000 immature heart muscle cells developed from mouse embryonic stem cells into the lower heart chamber of newborn rats that were engineered without an immune system for their bodies to accept the introduced foreign cells.

Within a month, the cells began to appear as adult heart muscle cells, according to the study, which was recently published in Cell Reports.

Chulan Kwon, leader of the study and member of the Johns Hopkins University School of Medicines Institute for Cell Engineering, said his team's research could represent a key advancement in the study and treatment of heart disease.

"Our concept of using a live animal host to enable maturation of cardiomyocytes can be expanded to other areas of stem cell research and really opens up a new avenue to getting stem cells to mature,"said Kwon.

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Kwon and his research group turned to newborn rats after realizing that cells grown in a lab setting were not transitioning from immature to mature cells likely due to the artificial atmosphere. After the switch, the researchers found the cells developed in the newborn rats had more genetic resemblance to adult heart muscle cells as opposed to immature heart cells.

In addition to a host of other proof-of-concept experiments used to test their findings, Kwon and company confirmed that the new cells could function as normal adult heart muscle cells.

While clinical use of such cells is years away, Kwon said he is cautiously optimistic about the indications of his team's research.

"The hope is that our work advances precision medicine by giving us the ability to make adult cardiomyocytes from any patients own stem cells" to target and treat specific heart diseases, he said.

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Newborn rats get mature hearts by serving as stem cell laboratory - RT

Scientists say new medical diagnostic chip can sort cells anywhere with an inkjet – Wikinews

By Uncategorized

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Thursday, February 9, 2017

On Monday, scientists from the Stanford University School of Medicine announced the invention of a new diagnostic tool that can sort cells by type: a tiny printable chip that can be manufactured using standard inkjet printers for possibly about one U.S. cent each. Lead researchers say this may bring early detection of cancer, tuberculosis, HIV and malaria to patients in low-income countries, where the survival rates for illnesses such as breast cancer can be half those of richer countries.

Existing methods tend to identify cell types using fluorescent or magnetic labels, which take time to attach, but this platform uses the phenomenon of dielectrophoresis: because different kinds of cells have different levels of receptivity to electrical fields, a trait called polarizability, when an electric potential gradient is activated around the chip, different cells are pulled in different directions at different speeds. This allows doctors to diagnose cancer by determining the number of tumor cells in a patient's blood sample. Different chips can be printed to diagnose different diseases.

Physically, the scientists say, the system has two parts. Cells are held in a clear microfluidic chamber made of silicone. The chip itself is an electronic strip that can be printed onto flexible polyester. Most lab-on-a-chip devices must be manufactured by professional staff in specialized facilities called clean rooms and can take weeks, but the chip component of this system can be made almost anywhere in as little as twenty minutes. The chips cost approximately one U.S. cent to produce (US$0.01) and can be reused. For comparison, a standard flow cytometry machine can cost US$100,000 to purchase.

"Enabling early detection of diseases is one of the greatest opportunities we have for developing effective treatments," said lead author and electrical engineer Dr. Rahim Esfandyarpour. "Maybe $1 in the U.S. doesn't count that much, but somewhere in the developing world, it's a lot of money."

Senior author Dr. Ron Davis of the Stanford University Genome Technology Center compared this invention to that of low-cost genome sequencing, which helped lead to personalized medicine.

The findings appeared in the Proceedings of the National Academy of Sciences on Monday.

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Scientists say new medical diagnostic chip can sort cells anywhere with an inkjet - Wikinews

Scientists identify aggressive pancreatic cancer cells and their vulnerability – Medical Xpress

By Uncategorized

February 9, 2017 Credit: University of Texas M. D. Anderson Cancer Center

Researchers have identified a gatekeeper protein that prevents pancreatic cancer cells from transitioning into a particularly aggressive cell type and also found therapies capable of thwarting those cells when the gatekeeper is depleted.

A team from The University of Texas MD Anderson Cancer Center describes this week in the journal Nature a series of preclinical experiments using patient-derived tumor xenografts (PDXs) and mouse models that point to potential treatments for patients with a rapidly-progressing and resistant subgroup of tumor cells.

"Pancreatic cancer cells are characterized by remarkable plasticity, cellular changes that make this malignancy so difficult to treat," said first author Giannicola Genovese, M.D., instructor in Genomic Medicine.

Genovese and colleagues found, in a subset of tumor cells, after the original oncogenic driver fades, depletion of a gene called SMARCB1 results in a cellular change to mesenchymal status, a mobile and invasive cell state.

The team also found a vulnerability for mesenchymal cells: they are overly reliant on accelerated protein production to meet increased metabolic needs.

"Inhibiting proteostasis in combination with standard of care chemotherapy was highly effective in killing these most aggressive subpopulations of pancreatic cancer," Genovese said.

Identify, understand tumor cells to kill them

This led the team to look at a drug called AUY922, an inhibitor of heat shock protein 90, which blocks proteostasis - the creation, folding, distribution and degrading of proteins. Both as a single agent and combined with the chemotherapy gemcitabine, AUY922 increased the response rate and lengthened survival of mice whose tumors faithfully recapitulated key features of human pancreatic cancers.

A key challenge in treating cancer stems from molecular and genomic variability of tumor cells, which causes functional differences across cells that can fuel resistance to treatment.

"We are working to dissect the cell populations within tumors to attempt to understand the functional vulnerabilities of each, then to plan for more rational combinatorial treatment approaches," said Giulio Draetta, M.D., Ph.D., professor of Genomic Medicine and director of MD Anderson's Institute for Applied Cancer Science.

Draetta, who is corresponding author of the paper, noted that identifying the subpopulation of aggressive cells and establishing their vulnerability to proteostasis inhibitors allows a match of treatment to specific cell type. "This is truly functionally defined, personalized medicine."

Path to mesenchymal status

To identify and study the impact of pancreatic cancer cell plasticity, the team established an experimental approach to isolate and characterize single cell clones called "escapers" that spontaneously acquire malignant features. They identified two major sub-populations, one preserving simpler epithelial differentiation, one displaying mesenchymal features.

Profiling the two types of escaper populations revealed that mesenchymal clones are characterized by the extinction of KRAS signaling, a common driver of pancreatic cancer, and the abnormal activation of epigenetic programs regulated by the chromatin remodeling factor SMARCB1.

Lower SMARCB1, shorter life

To explore the clinical relevance of these findings, the researchers analyzed surgically removed tumors from 134 patients and identified a subset of patients whose tumors displayed low levels of SMARCB1, independence from KRAS signaling and who had a dismal prognosis.

Subsequent experiments ablating the SMARCB1 gene in mouse models led to the rapid expansion of mesenchymal sub-populations with powerful growth and metastatic characteristics. Restoring SMARCB1 caused mesenchymal cells to revert to the less aggressive epithelial type, establishing SMARCB1 as a gatekeeper of epithelial identity.

The researchers also found that SMARCB1-deficient cells had increased protein synthesis rates and activation of a number of protein-related stress-response pathways. They also found that expression of the oncogene MYC is required to maintain the mesenchymal state in SMARCB1-deficient cells.

To test the stress-response connection, they ablated a crucial stress response gene, which resulted in tumor regression and prolonged survival in mice.

These findings led to the experiments with the HSP90 inhibitor AUY922, which caused tumor cell death and hindered growth in SMARCB1-deficient mice but had a limited impact on mice with intact SMARCB1. The combination with gemcitabine extended survival in mice transplanted with patient-derived xenografts.

Hunting mechanisms of cell change

"This work represents the first step of a major effort to understand the mechanisms allowing malignant cells to hijack specific gene programs to adapt to stress and survive," Genovese said. "Today we have a detailed map of the genetic landscape driving cancer initiation and progression, but our knowledge of the epigenetic, metabolic and molecular programs conferring on tumors cells the ability to change state are still elusive."

The team is developing novel technological tools to dissect those mechanisms in detail and also collaborates with the Institute for Applied Cancer Science to translate its findings by designing tailored clinical trials to exploit the vulnerabilities of those highly aggressive mesenchymal cells.

Explore further: Loss of key protein unleashes cascade that culminates in rhabdoid tumor formation

More information: Giannicola Genovese et al, Synthetic vulnerabilities of mesenchymal subpopulations in pancreatic cancer, Nature (2017). DOI: 10.1038/nature21064

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Scientists identify aggressive pancreatic cancer cells and their vulnerability - Medical Xpress

Researchers isolate cells that weaken immune response to cancer – ModernMedicine

By Uncategorized

Researchers at the University of Colorado are working on a new form of immunotherapy that would use cells originating in the spleen to fight against cancer.

This new immunotherapy would stifle the work of myeloid-derived suppressor cells (MDSCs), which soothe the immune system and are increased in the presence of cancer cells to decrease the bodys immune response to fight against cancer.

The study, published in Cancer Immunology & Immunotherapy, details how researchers at the University of Colorado Cancer Center used previous studies on mouse splenocytes that showed cancer boosted the number of these cells. Similar results were found in human cancer patients.

MDSCs are immune cells that originate from bone marrow cells and develop in the spleen. While in the spleen, these so-called splenocytes are programmed. While the exact function of these cells isnt clear, cancer patients with high levels of MDSCs tend to have poor prognoses and decreased responses to therapy.

Splenocytes are more abundant and easier to isolate in mice than in humans, making previous studies of this type of response difficult. Not only did the research team draw a correlation between the work of splenocytes in mice and humans, but also new methods for isolating human splenocytes.

The research team at the University of Colorado was able to prove that splenocytes had an immunosuppressive response in humans, blocking the work of T-cells in fighting against cancer, according to the report.

Other cancer therapies exist that use T-cells to fight cancer, but this study shows that the work of MDSCs might weaken the response of those T-cells and other cancer-fighting immune cells.

The study findings help to explain why only 20% to 40% of patients respond to existing immunotherapy, and could pave the way for new treatment to weaken the power of MDSCs in suppressing the T-cells response to cancer.

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Researchers isolate cells that weaken immune response to cancer - ModernMedicine

‘Economical stem cell treatment will revolutionise medicine’ – The Hindu

By Stem Cell Treatment

Cost-effective stem cell treatment has to be the next revolution to transform personalised treatment of patients, said Hunterian Professorship Awardee A.A.Shetty. Speaking at a felicitation function, Dr. Shetty said there is a need to create awareness about stem cells and patient-specific treatment specifically designed for individuals.

It is going to be simple, with minimal complications. Our role is to make it cost effective. Once it happens, it will revolutionise treatment, said Dr. Shetty.

Surgery using stem cell technology is developing at a rapid pace and the role of stem cell therapy in Orthopaedics is gaining importance, said Trauma and Orthopaedic Speciality Hospital (TOSH) Managing Director S.H. Jaheer Hussain. The ability of stem cells to transform into bone and cartilage has given a new dimension in the treatment of osteoarthritis, fracture non union, ligament tears. Stem cells have shown significant clinical results in osteoarthritis and cartilage defects. Recent advances in stem cells centrifuging techniques have lead to the introduction of the new concept of single- stage knee cartilage regeneration.

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'Economical stem cell treatment will revolutionise medicine' - The Hindu

Stanford scientists describe stem-cell and gene-therapy advances in scientific symposium – Scope (blog)

By Stem Cell Treatment

Using stem cells and gene therapy to treat orcure disease may still sound like science fiction, but a scientific meeting here last week emphasizedall the fronts onwhich it is moving closer and closer to fact.

Were entering a new era in medicine, said Lloyd Minor, MD, dean of the School of Medicine, in his opening remarks at the first annual symposium of the schools new Center for Definitive and Curative Medicine. Stanford researchersare poised to use stem cells and gene therapy to amelioratea wide swath of diseases, from common diagnoses such as diabetes and cancerto rare diseases ofthe brain, blood, skin, immune system and other organs. Ultimately, the goal is to create one-time treatments that can provide lifetime cures; hence the definitive and curative part of the centers name. Stanford is a leader in this branch of medical research, Minor said, addingThis is a vital component of our vision for precision health.

Stanford has a long history of leading basic-science discoveries in stem cell biology, andis now engaged in studyingmany different ways those discoveries couldbenefit patients, saidMaria Grazia Roncarolo, MD, who leads the new center.Our job is to produce clinical data so compelling that industry will pick up the product and take it to the next stage, Roncaraolo told the audience.

Among otherevent highlights:

More coverage of the days events is available in a story from the San Jose Mercury News that describeshowAnthonyOro, MD, PhD, and his colleagues are fighting epidermolysis bullosa, a devastating genetic disease of the skin. Oro closed his talk with a slightly goofy photo of a man getting a spray tan. It got a laugh, but his point was serious: Our goal for the cell therapy of the future is spray-on skin to correct a horrible genetic disease.

Ambitious? Yes. Science fiction? In the future, maybe not.

Previously: One of the most promising minds of his generation: Joseph Wu takes stem cells to heart,Life with epidermolysis bullosa: Pain is my reality, pain is my normaland Rat-grown mouse pancreases reverse diabetes in mice, say researchers Photo of Matthew Porteus courtesy of Stanford Childrens

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Stanford scientists describe stem-cell and gene-therapy advances in scientific symposium - Scope (blog)

Genetic profiling can guide stem cell transplantation for patients with myelodysplastic syndrome – Medical Xpress

By Stem Cell Treatment

February 9, 2017 Credit: NIH

A single blood test and basic information about a patient's medical status can indicate which patients with myelodysplastic syndrome (MDS) are likely to benefit from a stem cell transplant, and the intensity of pre-transplant chemotherapy and/or radiation therapy that is likely to produce the best results, according to new research by scientists at Dana-Farber Cancer Institute and Brigham and Women's Hospital.

In a study published in the New England Journal of Medicine, the investigators report that genetically profiling a patient's blood cells, while factoring in a patient's age and other factors, can predict the patient's response to a stem cell transplant and help doctors select the most effective combination of pre-transplant therapies. The findings are based on an analysis of blood samples from 1,514 patients with MDS, ranging in age from six months to more than 70 years, performed in collaboration with investigators from the Center for International Blood and Marrow Transplant Research.

MDS is a family of diseases in which the bone marrow produces an insufficient supply of healthy blood cells. Treatments vary depending on the specific type of MDS a patient has; donor stem cell transplants are generally used for patients with a high risk of mortality with standard treatments.

"Although donor stem cell transplantation is the only curative therapy for MDS, many patients die after transplantation, largely due to relapse of the disease or complications relating to the transplant itself," said the study's lead author, R. Coleman Lindsley, MD, PhD, of Dana-Farber. "As physicians, one of our major challenges is to be able to predict which patients are most likely to benefit from a transplant. Improving our ability to identify patients who are most likely to have a relapse or to experience life-threatening complications from a transplant could lead to better pre-transplant therapies and strategies for preventing relapse."

Researchers have long known that the specific genetic mutations within MDS patients' blood cells are closely related to the course the disease takes. The current study sought to discover whether mutations also can be used to predict how patients will fare following a donor stem cell transplant.

Analysis of the data showed that the single most important characteristic of a patient's MDS was whether their blood cells carried a mutation in the gene TP53. These patients tended to survive for a shorter time after a transplant, and also relapse more quickly, than patients whose cells lacked that mutation. This was true whether patients received standard "conditioning" therapy (which includes chemo- and/or radiation therapy) prior to transplant or received reduced-intensity conditioning, which uses lower doses of these therapies. Based on these results, doctors at Dana-Farber are now working on new strategies to overcome the challenges posed by TP53 mutations in MDS.

In patients 40 years old and over whose MDS didn't carry TP53 mutations, those with mutations in RAS pathway genes or the JAK2 gene tended to have a shorter survival than those without RAS or JAK2 mutations. In contrast to TP53 mutations, the adverse effect of RAS mutations on survival and risk of relapse was evident only in reduced-intensity conditioning. This suggests that these patients may benefit from higher intensity conditioning regimens, the researchers indicated.

The study also yielded key insights about the biology of MDS in specific groups of patients. Surprisingly, one in 25 patients with MDS between the ages of 18 and 40 were found to have mutations associated with Shwachman-Diamond syndrome (a rare inherited disorder that often affects the bone marrow, pancreas, and skeletal system), but most of them had not previously been diagnosed with it. In each case, the patients' blood cells had acquired a TP53 mutation, suggesting not only how MDS develops in patients with Schwachman-Diamond syndrome but also what underlies their poor prognosis after transplantation.

The researchers also analyzed patients whose MDS arose as a result of previous cancer therapy (therapy-related MDS). They found that TP53 mutations and mutations in PPM1D, a gene that regulates TP53 function, were far more common in these patients than in those whose disease occurred in the absence of previous cancer treatment.

"In deciding whether a stem cell transplant is appropriate for a patient with MDS, it's always necessary to balance the potential benefit with the risk of complications," Lindsley remarked. "Our findings offer physicians a guide - based on the genetic profile of the disease and certain clinical factors - to identifying patients for whom a transplant is appropriate, and the intensity of treatment most likely to be effective."

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Genetic profiling can guide stem cell transplantation for patients with myelodysplastic syndrome - Medical Xpress

Stem-cell-derived cells flag a possible new treatment for rare blood … – Medical Xpress

By Stem Cell Treatment

February 8, 2017 Blood stem cells from patients with Diamond-Blackfan anemia dont mature properly (right two columns). Credit: Doulatov et al., Science Translational Medicine (2017)

Researchers at Boston Children's Hospital's Stem Cell Research Program were able, for the first time, to use patients' own cells to create cells similar to those in bone marrow, and then use them to identify potential treatments for a blood disorder. The work was published today by Science Translational Medicine.

The team derived the so-called blood progenitor cells from two patients with Diamond Blackfan anemia (DBA), a rare, severe blood disorder in which the bone marrow cannot make enough oxygen-carrying red blood cells. The researchers first converted some of the patients' skin cells into induced pluripotent stem (iPS) cells. They then got the iPS cells to make blood progenitor cells, which they loaded into a high-throughput drug screening system. Testing a library of 1,440 chemicals, the team found several that showed promise in a dish. One compound, SMER28, was able to get live mice and zebrafish to start churning out red blood cells.

The study marks an important advance in the stem cell field. iPS cells, theoretically capable of making virtually any cell type, were first created in the lab in 2006 from skin cells treated with genetic reprogramming factors. Specialized cells generated by iPS cells have been used to look for drugs for a variety of diseasesexcept for blood disorders, because of technical problems in getting iPS cells to make blood cells.

"iPS cells have been hard to instruct when it comes to making blood," says Sergei Doulatov, PhD, co-first author on the paper with Linda Vo and Elizabeth Macari, PhD. "This is the first time iPS cells have been used to identify a drug to treat a blood disorder."

DBA currently is treated with steroids, but these drugs help only about half of patients, and some of them eventually stop responding. When steroids fail, patients must receive lifelong blood transfusions and quality of life for many patients is poor. The researchers believe SMER28 or a similar compound might offer another option.

"It is very satisfying as physician scientists to find new potential treatments for rare blood diseases such as Diamond Blackfan anemia," says Leonard Zon, MD, director of Boston Children's Stem Cell Research Program and co-corresponding author on the paper with George Q. Daley, MD, PhD. "This work illustrates a wonderful triumph," says Daley, associate director of the Stem Cell Research Program and also dean of Harvard Medical School.

Making red blood cells

As in DBA itself, the patient-derived blood progenitor cells, studied in a dish, failed to generate the precursors of red blood cells, known as erythroid cells. The same was true when the cells were transplanted into mice. But the chemical screen got several "hits": in wells loaded with these chemicals, erythroid cells began appearing.

Because of its especially strong effect, SMER28 was put through additional testing. When used to treat the marrow in zebrafish and mouse models of DBA, the animals made erythroid progenitor cells that in turn made red blood cells, reversing or stabilizing anemia. The same was true in cells from DBA patients transplanted into mice. The higher the dose of SMER28, the more red blood cells were produced, and no ill effects were found. (Formal toxicity studies have not yet been conducted.)

Circumventing a roadblock

Previous researchers have tried for years to isolate blood stem cells from patients. They have sometimes succeeded, but the cells are very rare and cannot create enough copies of themselves to be useful for research. Attempts to get iPS cells to make blood stem cells have also failed.

The Boston Children's researchers were able to circumvent these problems by instead transforming iPS cells into blood progenitor cells using a combination of five reprogramming factors. Blood progenitor cells share many properties with blood stem cells and are readily multiplied in a dish.

"Drug screens are usually done in duplicate, in tens of thousands of wells, so you need a lot of cells," says Doulatov, who now heads a lab at the University of Washington. "Although blood progenitor cells aren't bona fide stem cells, they are multipotent and they made red cells just fine."

SMER28 has been tested preclinically for some neurodegenerative diseases. It activates a so-called autophagy pathway that recycles damaged cellular components. In DBA, SMER28 appears to turn on autophagy in erythroid progenitors. Doulatov plans to further explore how this interferes with red blood cell production.

Explore further: Scientists find that persistent infections in mice exhaust progenitors of all blood cells

More information: "Drug discovery for Diamond-Blackfan anemia using reprogrammed hematopoietic progenitors," Science Translational Medicine stm.sciencemag.org/lookup/doi/10.1126/scitranslmed.aah5645

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Stem-cell-derived cells flag a possible new treatment for rare blood ... - Medical Xpress

Stem cell treatment effective in anterior cruciate ligament tear – Business Standard

By Stem Cell Treatment

IANS | New Delhi February 6, 2017 Last Updated at 20:08 IST

With stem cell treatment effective in several health conditions, including spinal problems, doctors say the medical procedure can also help in speedy recovery of anterior cruciate ligament tear - a common chronic sports injury.

The medical procedure has advantage over surgeries because they are less invasive and focus on regeneration and healing of the tissues and ligaments rather than to cut and replace it.

In a case study, 28-year-old Mohan Verma - - a footballer suffering from anterior cruciate ligament was cured through Stem Cell treatment also known as Human Embryonic Stem Cell (HESC) in just eight months time.

Initially he was told to undergo a surgery and was informed that the complete recovery would take at least a year.

According to the doctors, HESC is used in three phases for ACL, so that the stem cells could grow, repair and regenerate the ligaments, tissues in the knee.

"Each treatment phase lasts 4-6 weeks during which 0.05 ml human embryonic stem cells is injected. The physician continuously administers the HESC. No immune-suppressants are given to the patients. In addition to HESC therapy, the patient receives physiotherapy and occupational therapy," said Geeta Shroff, stem cells specialist at Delhi-based Nutech Mediworld Hospital.

Comparing it to the conventional treatment, Shroff said: "In surgical procedure the graft goes in at a steeper angle than the original ACL which causes compression of the cartilage and hence most of the young athletes undergoing surgery end up with arthritis by the age of 30."

"Moreover the position sense and the strength of the knee can never be restored," she added.

--IANS

rup/rn

(This story has not been edited by Business Standard staff and is auto-generated from a syndicated feed.)

With stem cell treatment effective in several health conditions, including spinal problems, doctors say the medical procedure can also help in speedy recovery of anterior cruciate ligament tear - a common chronic sports injury.

The medical procedure has advantage over surgeries because they are less invasive and focus on regeneration and healing of the tissues and ligaments rather than to cut and replace it.

In a case study, 28-year-old Mohan Verma - - a footballer suffering from anterior cruciate ligament was cured through Stem Cell treatment also known as Human Embryonic Stem Cell (HESC) in just eight months time.

Initially he was told to undergo a surgery and was informed that the complete recovery would take at least a year.

According to the doctors, HESC is used in three phases for ACL, so that the stem cells could grow, repair and regenerate the ligaments, tissues in the knee.

"Each treatment phase lasts 4-6 weeks during which 0.05 ml human embryonic stem cells is injected. The physician continuously administers the HESC. No immune-suppressants are given to the patients. In addition to HESC therapy, the patient receives physiotherapy and occupational therapy," said Geeta Shroff, stem cells specialist at Delhi-based Nutech Mediworld Hospital.

Comparing it to the conventional treatment, Shroff said: "In surgical procedure the graft goes in at a steeper angle than the original ACL which causes compression of the cartilage and hence most of the young athletes undergoing surgery end up with arthritis by the age of 30."

"Moreover the position sense and the strength of the knee can never be restored," she added.

--IANS

rup/rn

(This story has not been edited by Business Standard staff and is auto-generated from a syndicated feed.)

IANS

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Stem cell treatment effective in anterior cruciate ligament tear - Business Standard

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