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

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 research - NewsOK.com

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

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

Eli and Edythe Broad add $1 million donation to further stem cell research – USC News

PhilanthropistsEli and Edythe Broad have donated $1 million tosupport eight grants for early-stage stem cell research projects at three California universities including USC.

The gift came at a Feb. 3 symposium marking the 10th anniversary of the stem cell research centers at USC, UCLA and the University of California, San Francisco,established with support from the Broads and the California Institute for Regenerative Medicine (CIRM).

We love scientists because none of you are satisfied with the status quo.

Eli Broad

We love scientists because none of you are satisfied with the status quo, Eli Broad said at the symposium, hosted by UCLA.

Among the groundbreaking discoveriesof stem cell researchers is a cure forinfants born without a functional immune system an inherited condition called Adenosine Deaminase Severe Combined Immunodeficiency, or Bubble Baby Disease.

The traditional treatment is a bone marrow transplant from a matched donor, which carries significant risk of fatal immune rejection or other complications.Donald Kohns lab at UCLA engineered a better source of transplanted cells: the patients own stem cells, in which the disease-causing mutation has been corrected using gene editing. This cure already proved successful in clinical trials, and is now being commercialized with support and funding from CIRM.

Its hard to put into a simple sentence how important this philanthropy has been for all three of our centers, said Owen Witte, director of UCLAs stem cell research center. But I can honestly say that without their philanthropy to help establish these three sites, I dont think UCLA would have accomplished what weve been able to, and Im pretty sure the same holds true for USC and UCSF.

Andy McMahon, director of USCs stem cell research center, highlighted the Broads enormous influence not only on science, but also on art and education.

Art, education and science hold special and personal meanings for us all, said McMahon. Through art, we explore our imaginations. Through education, we realize our potential. Through science, we improve our lives. And through the Broads lifelong and transformative philanthropy, art and education and science have flourished to the great and obvious benefit of all.

In addition to recognizing the Broads, the symposium highlighted recent scientific progress in regenerative medicine.

Arnold Kriegstein, director of UCSFs stem cell research center, shared how his stem cell research spurred an ongoing clinical trial, using the antibiotic Azithromycin as a treatment for Zika patients in Brazil.UCSFs Holger Willenbring is nearing the clinical trial stage with a stem cell-based approach to treating liver scarring, also known as fibrosis or cirrhosis.

Other scientists shared projects at any earlier stage of research and discovery. They painted a vision of a future in which kidney disease, hearing loss, cancer, infertility and even aging itself could become afflictions of the past.

USCs Neil Segil discussedhearing loss, which affects approximately 10 percent of the worlds population and half of the retirement-aged population. Most hearing loss is due to damage to the inner ears sensory cells, which do not regenerate in humans. However, they do regenerate in non-mammals, such as birds thanks to a population of neighboring cells that respond to deafness by differentiating into replacement sensory cells. Segils group is exploring ways to stimulate a similar regenerative response in mammals.

The mutations underlying cancer were the topic for USC researcher Min Yu. Her lab studies patient-derived breast cancer stem cells (CSCs), which break off of the primary tumor, enter the bloodstream and seed the new metastatic tumors that ultimately prove fatal. In analyzing these CSCs, her lab found that certain mutations and gene activity can consistently predict specific patterns of metastasis such as the formation of secondary tumors in the brain versus other organs. These insights could inform the future of personalized medicine.

The updates were well received by the Broads. Edye and I want to thank each of you for your dedication, Eli Broad said. We cannot express how much your work means to us and how we appreciate all you do to improve human health.

More stories about: Research, Stem Cells

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Eli and Edythe Broad add $1 million donation to further stem cell research - USC News

Chancellor appointed to governing board of state stem cell agency – UC Santa Cruz (press release)

Chancellor George Blumenthal (photo by Mike Bolte).

Chancellor George Blumenthal has been appointed to a seat on the governing board of the California Institute for Regenerative Medicine (CIRM), the state agency created by voters in 2004 to fund stem cell research.

Blumenthal's appointment was announced Wednesday, Feb. 8, by California Lt. Gov. Gavin Newsom, who personally invited the campus leader to join the 29-member board.

"The California Institute for Regenerative Medicine is doing outstanding work, and I am delighted to join the board. CIRM support has advanced stem cell research at UC Santa Cruz and across the state," said Blumenthal. "Public support for this work remains strong, and I look forward to playing a role in securing the future of the institute."

Blumenthal was appointed to a six-year term on the board. A professor of astronomy and astrophysics, he said he looks forward to learning a lot more about stem cell research, the projects supported by CIRM, and the benefits to patients.

CIRM funds stem cell research at institutions and companies throughout California, and beyond, with the goal of speeding treatments to patients with unmet medical needs, including those suffering from heart disease, stroke, cancer, diabetes, Alzheimer's, and Parkinson's disease. CIRM began funding scientists in 2006 and today is the world's largest institution dedicated to helping people by advancing human embryonic stem cell research.

UC Santa Cruz researchers across the disciplines have received support from CIRM, including those working in bioinformatics, neurobiology, developmental biology, and genetics. Even astronomers with expertise in adaptive optics for telescopes have been funded to work with electrical engineers and biologists on deep-tissue imaging techniques.

Among the highlights of campus support received over the past decade:

A 2008 grant for $7.2 million to establish a stem cell research center. A 2014 grant to run the data coordination and management program of the Center of Excellence in Stem Cell Genomics A $2.2 million grant in 2009 to fund a program to train stem cell scientists

Jonathan Thomas, chair of the CIRM board said, "We are honored to have someone with Dr. Blumenthals experience and expertise join the board. As Chancellor at UCSC, he has demonstrated a clear commitment to advancing world-class research and earned a reputation as a bold and visionary leader. We look forward to seeing those qualities in action to help advance CIRMs mission."

Former state senator Art Torres serves as vice chair of the CIRM board; Torres is a 1968 graduate of Stevenson College at UC Santa Cruz (B.A., government).

Blumenthal also serves as chair of the California Association for Research in Astronomy (CARA), which manages the W. M. Keck Observatory near the summit of Mauna Kea in Hawaii.

"I am a great champion of cutting-edge technology that expands human understanding, from the telescopes that unlock the secrets of the universe to computers that reveal the intricacies of the human genome," said Blumenthal.

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Chancellor appointed to governing board of state stem cell agency - UC Santa Cruz (press release)

Mars’s frozen pole, Sweden’s climate plan and a stem-cell trial in Japan – Nature.com

Research | Policy | Politics | People | Events | Funding | Awards | Trend watch | Coming up

Stem-cell trial Japan is resuming pioneering clinical research using induced pluripotent stem (iPS) cells. A team led by Masayo Takahashi at the RIKEN Center for Developmental Biology in Kobe will make suspensions of iPS cells derived from retinal cells, and transplant them into people with age-related macular degeneration, an eye condition that can cause blindness. Takahashi started a similar study in 2014 the first to use iPS cells in humans but the cells prepared for the second patient were found to have genetic abnormalities and no other participants were recruited. On 1February, Japans health ministry approved a new five-patient study. This time the team will use banked iPS cells created from anonymous, healthy donor cells rather than from the participants themselves.

Martian polar ice cap sculpted by wind A seasonal layer of carbon dioxide frost coats Marss northern polar ice cap in this image, which was released on 2February by the European Space Agency (ESA). Each winter, carbon dioxide precipitates out of the cold atmosphere and onto the ice cap. The image is a composite of pictures taken between 2004 and 2010 by ESAs Mars Express spacecraft. The distinctive spiral troughs were probably carved by wind. Radar investigation by Mars Express and NASAs Mars Reconnaissance Orbiter revealed that the ice cap consists of many layers of ice and dust extending to a depth of about 2 kilometres.

ESA/DLR/FU Berlin; NASA MGS MOLA Science Team

GM wheat trial A UK research laboratory has been granted permission to begin field trials of a wheat plant that has been genetically modified (GM) to improve photosynthesis. Scientists at Rothamsted Research in Harpenden have already shown that wheat plants modified with a gene from stiff brome grass (Brachypodium distachyon) are more efficient at photosynthesis in greenhouses than conventional wheat, and they now hope to see improved yields from plants grown outside in more realistic conditions. In 2012, GM trials at Rothamsted attracted small but high-profile protests. The labs researchers have been among the leading advocates of such trials in Europe.

Swedish stimulus The Swedish government unveiled plans on 2February to make the country carbon neutral in less than two decades. A law expected to pass through parliament in March would set a binding target of reducing domestic greenhouse-gas emissions from industry and transport by 85% by 2045, relative to 1990 levels. Remaining emissions would be offset by natural carbon capture through forestation and by investment abroad. On announcing the move, Swedens environment minister, Isabella Lvin, said that her country wants to set an example at a time when climate action in the United States is threatening to lose momentum.

Romanian protests Angry Romanian scientists have called on their new government to reverse its order for national science-advisory bodies to immediately stop their work, pending reorganization. The government made the order on 31January, when it also issued a decree giving amnesty to some officials accused of corruption; this was later withdrawn after mass protests. An open letter signed by nearly 600academics and their supporters says that the councils, which are non-political, should be immune to government change. Signatories fear that the proposed reorganization may allow amnesty for politicians who have committed scientific misconduct.

UK science czar The UK governments chief scientific adviser has been appointed to possibly the biggest science job in the country. The government announced on 2February that Mark Walport will take the helm of a new body called UK Research and Innovation (UKRI), which is expected to oversee a pot of more than 6billion (US$7.5billion) in government science spending when it comes into being in 2018. Walports appointment is significant because there are fears that UKRI could reduce the freedom of the nine individual bodies that currently allocate much government science funding.

Researcher on trial An Iranian researcher in disaster medicine, who is accused of collaboration with a hostile government, has been threatened with the death sentence by a judge on Irans revolutionary court, according to close contacts of the scientist. Ahmadreza Djalali, who had been affiliated with research institutes in Italy, Sweden and Belgium, was arrested inApril 2016 during an academic visit to Iran. According to sources close to Djalali, he has been kept in solitary confinement for three months in a Tehran prison and was forced to sign a confession. Djalalis trial is scheduled to start later this month.

Ice station The British Antarctic Survey (BAS) announced on 2February that it had completed moving its HalleyVI research station 23kilometres across the floating ice platform on which it rests. The 13-week operation, which used tractors to tow the stations 8 modules (pictured), was prompted by fears about a growing crack in the Brunt ice shelf. Staff were evacuated last month for the coming Antarctic winter after another unpredictable crack in the ice was discovered. The base, which is designed to be relocated periodically, is ready for re-occupation in November, the BAS said.

British Antarctic Survey

Borehole record The Iceland Deep Drilling Project completed the deepest-ever geothermal well on 25January. After 168days of drilling, the well bottomed out at 4,659metres, just shy of its 5-kilometre goal. But temperatures and pressures were so high at the bottom of the well that fluids were observed behaving in a supercritical fashionas neither liquid nor gasan observation that was one of the projects goals. The well, on Icelands volcanic Reykjanes peninsula, is being used to explore the source of geothermal systems and to see whether supercritical fluids can be tapped as an energy resource.

Indias budget Health research, biotechnology and space science are the main beneficiaries of robust budget increases announced by the Indian government on 1February. Overall, science spending in 2017 by eight ministries (excluding nuclear and defence research) will increase by 11%well above the projected 5% inflation rateto 360billionrupees (US$5.3billion). Health research, including the fight against diseases such as leprosy and measles, will get 31% more government funding. Biotechnology will get an extra 22%, and Indias aspirations in space, including plans to land a rover on the Moon in 2018, will benefit from a 21% budget increase for space science.

Dual tribute The CRISPR gene-editing system, which has transformed biological research and biomedicine, drew yet more major prizes last week. On 31January, the Madrid-based BBVA Foundation announced that its 400,000 (US$427,000) Frontiers of Knowledge Award in Biomedicine would be shared by Francisco Mojica, Emmanuelle Charpentier and Jennifer Doudna. Mojica discovered the CRISPR repeating DNA sequences that some bacteria use to fight viral infections. Charpentier and Doudna developed the universal CRISPR editing toolfor which they have also won the 50-million (US$445,000) Japan Prize, announced on 2February. They share it with cryptographer Adi Shamir.

Women, non-Asian ethnic minorities and disabled people are under-represented in science and engineering in the United States, according to the National Center for Science and Engineering Statistics (NCSES). Women receive about half of all science and engineering degrees but hold less than 30% of jobs in these areas. White men, who in 2015 comprised only 31% of the US population, held nearly half of these jobs. Although female and minority representation has risen, disparities remain.

Source: NCSES

1115 February Biophysicists gather in New Orleans, Louisiana, for the Biophysical Societys 61st annual meeting. go.nature.com/2jtfz17

1216 February At an international meeting in Queenstown, New Zealand, scientists discuss the latest research in advanced materials and nanotechnology. confer.co.nz/amn8

15 February Indias Polar Satellite Launch Vehicle launches a high-resolution Earth-observation satellite from the Satish Dhawan Space Center in Sriharikota. go.nature.com/2jteerk

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Mars's frozen pole, Sweden's climate plan and a stem-cell trial in Japan - Nature.com

The origin of stem cells – Science Daily

The origin of stem cells Science Daily Freiburg plant biologist Prof. Dr. Thomas Laux and his research group have published an article in the journal Developmental Cell presenting initial findings on how shoot stem cells in plants form during embryogenesis, the process of embryonic development. and more »

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The origin of stem cells - Science Daily

UCLA researchers turn stem cells into somites, precursors to skeletal muscle, cartilage and bone – UCLA Newsroom

FINDINGS

Adding just the right mixture of signaling molecules proteins involved in development to human stem cells can coax them to resemble somites, which are groups of cells that give rise to skeletal muscles, bones, and cartilage in developing embryos. The somites-in-a-dish then have the potential to generate these cell types in the lab, according to new research led by senior author April Pyle at theEli and Edythe Broad Center of Regenerative Medicine and Stem Cell Research at UCLA.

Pluripotent stem cells, by definition, can become any type of cell in the body, but researchers have struggled to guide them to produce certain tissues, including muscle. In developing human embryos, muscle cells as well as the bone and cartilage of vertebrae and ribs, among other cell types arise from small clusters of cells called somites.

Researchers have studied how somites develop in animals and identified the molecules that seem to be an important part of that process in animals. But when scientists have tried to use those molecules to coax human stem cells to generate somites, the protocols have been inefficient.

The scientists isolated the minuscule developing human somites and measured expression levels of different genes both before and after the somites were fully formed. For each gene that changed levels during the process, the researchers tested whether adding molecules to boost or suppress the function of that gene in human pluripotent stem cells helped push the cells to become somite-like. They found that the optimal mixture of molecules in humans was different than what had been tried in animals. Using the new combination, they could turn 90 percent of human stem cells into somite cells in just four days.

The scientists followed the cells over the next four weeks and determined that they were indeed able to generate cells including skeletal muscle, bone and cartilage that normally develop from somites.

The new protocol to create somite-like cells from human pluripotent stem cells opens the door to researchers who want to make muscle, bone and cartilage cells in the lab. Pyles group plans to study how to use muscle cells generated from the new somites to treat Duchenne muscular dystrophy, a severe form of muscle degeneration that currently does not have a cure.

Pyle is a UCLA associate professor of microbiology, immunology and molecular genetics. The first author of the study is Haibin Xi; co-authors are Wakana Fujiwara, Karen Gonzalez and Majib Jan of UCLA; Katja Schenke-Layland and Simone Liebscher of Germanys Eberhard Karls University Tbingen; and Ben Van Handel of CarthroniX Inc., a California-based biopharmaceutical company.

The study was publishedin the journal Cell Reports.

The study was funded by a grant from the National Institute of Arthritis and Musculoskeletal and Skin Diseases (R01AR064327) and support from a UCLA Broad Stem Cell Research Center Research Award.

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UCLA researchers turn stem cells into somites, precursors to skeletal muscle, cartilage and bone - UCLA Newsroom

Patients’ cells provide possible treatment for blood disorder – Harvard Gazette

Image courtesy of the Daley Lab/Boston Childrens Hospital

Red blood cells successfully made via induced pluripotent stem cells from a Diamond-Blackfan anemia (DBA) patient. Control iPS cells (left) and DBA iPSc cells (right), showing that DBA blood cells dont mature properly.

Researchers at Boston Childrens Hospitals 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. Induced pluripotent stem cells, which are 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 diseases except for blood disorders, because of technical problems in getting iPS cells to make blood cells.

By Nancy Fliesler, Boston Children's Hospital Communications | January 15, 2015 | Editor's Pick Audio/Video

Sergei Doulatov, co-first author on the paper with Linda Vo and Elizabeth Macari, said the cells have been hard to instruct when it comes to making blood. 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, said Leonard Zon, director of Boston Childrens Stem Cell Research Program and co-corresponding author on the paper with George Q. Daley, This work illustrates a wonderful triumph, said 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 Childrens 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, said Doulatov, who now heads a lab at the University of Washington. Although blood progenitor cells arent 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.

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Patients' cells provide possible treatment for blood disorder - Harvard Gazette

California’s stem cell program ponders a future of new challenges and old promises – Los Angeles Times

Evangelina Padilla Vaccaro of Coronais the new face of stem cell science in California. Born with bubble baby disease that deprived her of a functioning immune system, she was cured with stem cell therapy developed in Donald Kohns lab at UCLA, which has received millions of dollars in grants from the California stem cell program.

Now shes a vivacious 4-year-old, depictedastride a hobby horse and clad in a pink sweatshirt bearing a lightning bolton the programs 2016 annual report under the legend CURED.

Thank you all for the amazing work you do, her father told the program board during its Dec. 13 meeting.

Evangelina represents the great potential of the $3-billion state program, formally known as the California Institute for Regenerative Medicine, or CIRM, but also its dilemma as it ponders its next chapter. Established in 2004 by a nearly 60-40 vote for Proposition 71, CIRM began issuing grants in 2006. Now, after 10 years, the program has committed $2.2 billion of its bond-funded war chest. Its expecting to spend the rest by the end of 2020.

So it wont be long before CIRM must confront the question of whether to fold up shop when its well runs dry, seek outside funding from foundations and industry, or appeal to voters for more public money. If it returns to the ballot, CIRM would have a chance to reconsider its administrative structure, the inflated expectations it gave voters in 2004, its embedded conflicts of interestand even whether it should be limited to funding research into stem cells. All these features of Proposition 71 have created complications during the programsfirst decade.

Robert Klein II, the real estate investor who was the driving force behind Proposition 71 and chaired the institutefor its first seven years, already has said that he intends to place a funding measure on the November 2018 ballot.

CIRM officials, wary of rules limiting how far public agencies can go in lobbying for ballot measures, arent yettaking an official stand on Kleins effort. Were leaving it to Bob, Chairman Jonathan Thomas says; he told the oversight board in December, We keep him updated on CIRMs progress so that his effort is fully informed.

Thomas added, however, that he and other officers have started discussions with philanthropists and medical foundations that could be potential sources of funds to keep CIRM going in the event Bobs measure is not successful.Klein didnt respond to a requestfor comment.

The success of any ballot initiativewill depend on two factors, says Henry T. Greely, director of the Center for Law and the Biosciences at Stanford University: The two big variables are whether any of their clinical trials pay off, and what the Trump administration does.

The public wants evidence that its heavy investment in CIRM has yieldedcures for diabetes, Alzheimers, Parkinsons, spinal cord injuries orother conditions that were touted as research targets by the Proposition 71 campaign. Evangelinas improvement notwithstanding, no CIRM-funded research has yet reached the marketing stage, although CIRM officials say some initiativesare getting close.

Greelys allusion to White House policy harks back to the very genesis of the stem cell program, President George W. Bushs 2001 ban on federal funding for research on stem cellsderived from humanembryos. The imposition of an essentially ideological test forscientific research was what spurred California voters to enactProposition 71 as a constitutional amendment three years later. The measure endowed CIRM with $3 billion in bond revenue to fund California stem cell laboratories and attract world-class researchers.

The program certainly has helped turn California into a global center of stem cell research. California is the place to be if you want to develop stem cell treatments, says Jeanne Loring of Scripps Research Institute in La Jolla, a CIRM grantee who is researching possible treatments for Parkinsons.A lot of my colleagues in other states are envious.

President Obama lifted the Bush ban in 2009, but by then CIRMs role as a bulwarkof Californias research infrastructure was secure. Whether Trump might reimpose the ban is unknown.

Trump himself hasnt left a record of his views on embryonic stem cells, which typically are derived from unused embryos developed for in vitro fertilization and donated for research. ButVice President Mike Pence labeled suchresearch morally wrong in the pages of Christianity Today.

A new ballot campaign would present an opportunity to fix some of the stem cell programs flaws that were written into Proposition 71 and consequently embedded in the state Constitution.

The measure gave CIRM unique exemptions from most legislative oversight and state laws mandating public accountability and transparency. By dictating that seats on CIRMs 29-member oversight board would be filled almost exclusively by representatives of grant-eligible institutions or patient advocacy groups, it institutionalizedconflicts of interest and an atmosphere of cronyism.

The Institute of Medicine of the National Academy of Sciences found in a 2012 report that this setup bedeviled the program with persistent questions about the integrity and independence of some of CIRMs decisions. Indeed, an analysis by David Jensen of the California Stem Cell Report found that about 90% of the $1.7 billion in CIRM grants by 2013 had gone to institutions with present or past representatives on the board.

Lasting damage to CIRMs mission also may have been done by the tenor of the Proposition 71 campaign, which used such high-profile victims of neurological conditions as Michael J. Fox and Christopher Reeve to give voters the impression that money was the sole obstacle to miraculous stem cell cures, and that successful treatments would yield immense profits for the state. Neither claim was realistic, but they set benchmarks for success that CIRM has been unable to meet.

A new funding campaign could give the program a much-needed reboot. The ballot measure could restructure CIRM as an ordinary agency of the state subject to legislative oversight, open meetings lawsand other good-government statutes, says Marcy Darnovsky, executive director of the Berkeley-based Center for Genetics and Society.

Especially in its early years during Kleins chairmanship, the program guarded its independence from oversight truculently. Since then, Darnovsky says, its been more accommodating: Theyve been much better than they have to be by the letter of the law, she says approvingly. But she says the program has never resolved the conflicts of interest inherent in who decides where the money goes and who gets it.

A new campaign could instill more public realismabout the potential of the research being funded. If they ask for money, it would be really important that they level with the people of California and educate them about how science really works, Darnovsky said.

CIRMs leadership knows that the publics inflated expectations threaten to obscurethe programs real accomplishments. With multiple clinical trials of CIRM-funded researchunderway, the first government approval of treatments isexpected in the not-too-distant future, C. Randal Mills, the programs president, said in an interview.

But he acknowledged that expectations need to be tempered with humility at the enormity of the task before us. We dont want to overpromise or overhype. CIRM is doing what it was set up to do, but it might be taking longer than people thought or hoped.

Still, the programs future may depend more on politics than science. If it looks like Washington is flipping off California, that could have political ramifications at the ballot box, Greely says. Some researchers arent optimistic about the prospects for independent, federally funded science under the Trump administration.

The only thing Ican predict, Loring says, is that it will be neutral or negative. It wont be positive.

Keep up to date with Michael Hiltzik. Follow@hiltzikmon Twitter, see hisFacebook page, or emailmichael.hiltzik@latimes.com.

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California's stem cell program ponders a future of new challenges and old promises - Los Angeles Times

Yes there’s hope, but treating spinal injuries with stem cells is not a reality yet – The Conversation AU

The 2017 Australian of the Year award went to Professor Alan Mackay-Sim for his significant career in stem cell science.

The prize was linked to barbeque-stopping headlines equating his achievements to the scientific equivalent of the moon landing and paving the road to recovery for people with spinal cord injuries.

Such claims in the media imply that there is now a scientifically proven stem cell treatment for spinal cord injury. This is not the case.

For now, any clinic or headline claiming miracle cures should be viewed with caution, as they are likely to be trading on peoples hope.

Put simply, injury to the spinal cord causes damage to the nerve cells that transmit information between the brain and the rest of the body.

Depending on which part of the spine is involved, the injury can affect the nerves that control the muscles in our legs and arms; those that control bowel and bladder function and how we regulate body temperature and blood pressure; and those that carry the sensation of being touched. This occurs in part because injury and subsequent scarring affect not just the nerves but also the insulation that surrounds and protects them. The insulation the myelin sheath is damaged and the body cannot usually completely replace or regenerate this covering.

Stem cells can self-reproduce and grow into hundreds of different cell types, including nerves and the cells that make myelin. So the blue-sky vision is that stem cells could restore some nerve function by replacing missing or faulty cells, or prevent further damage caused by scarring.

Studies in animals have applied stem cells derived from sources including brain tissue, the lining of the nasal cavity, tooth pulp, and embryos (known as embryonic stem cells).

Dramatic improvements have been shown on some occasions, such as rats and mice regaining bladder control or the ability to walk after injury. While striking, such improvement often represents only a partial recovery. It holds significant promise, but is not direct evidence that such an approach will work in people, particularly those with more complex injuries.

The translation of findings from basic laboratory stem cell research to effective and safe treatments in the clinic involves many steps and challenges. It needs a firm scientific basis from animal studies and then careful evaluation in humans.

Many clinical studies examining stem cells for spinal repair are currently underway. The approaches fit broadly into two categories:

using stem cells as a source of cells to replace those damaged as a result of injury

applying cells to act on the bodys own cells to accelerate repair or prevent further damage.

One study that has attracted significant interest involves the injection of myelin-producing cells made from human embryonic stem cells. Researchers hoped that these cells, once injected into the spinal cord, would mature and form a new coating on the nerve cells, restoring the ability of signals to cross the spinal cord injury site. Preliminary results seem to show that the cells are safe; studies are ongoing.

Other clinical trials use cells from patients own bone marrow or adipose tissue (fat), or from donated cord blood or nerves from fetal tissue. The scientific rationale is based on the possibility that when transplanted into the injured spinal cord, these cells may provide surrounding tissue with protective factors which help to re-establish some of the connections important for the network of nerves that carry information around the body.

The field as it stands combines years of research, and tens of millions of dollars of investment. However, the development of stem cell therapies for spinal cord injury remains a long way from translating laboratory promise into proven and effective bedside treatments.

Each case is unique in people with spinal cord injury: the level of paralysis, and loss of sensation and function relate to the type of injury and its location. Injuries as a result of stab wounds or infection may result in different outcomes from those incurred as a result of trauma from a car accident or serious fall. The previous health of those injured, the care received at the time of injury, and the type of rehabilitation they access can all impact on subsequent health and mobility.

Such variability means caution needs to accompany claims of man walking again particularly when reports relate to a single individual.

In the case that was linked to the Australian of the Year award, the actual 2013 study focused on whether it was safe to take the patients own nerves and other cells from the nose and place these into the damaged region of the spine. While the researchers themselves recommended caution in interpreting the results, accompanying media reports focused on the outcome from just one of the six participants.

While the outcome was significant for the gentleman involved, we simply do not know whether recovery may have occurred for this individual even without stem cells, given the type of injury (stab wounds), the level of injury, the accompanying rehabilitation that he received or a combination of these factors. It cannot be assumed a similar outcome would be the case for all people with spinal injury.

Finding a way to alleviate the suffering of those with spinal cord injury, and many other conditions, drives the work of thousands of researchers and doctors around the globe. But stem cells are not a silver bullet and should not be immune from careful evaluation in clinical trials.

Failure to proceed with caution could actually cause harm. For example, a paraplegic woman who was also treated with nasal stem cells showed no clinical improvement, and developed a large mucus-secreting tumour in her spine. This case highlights the need for further refinement and assessment in properly conducted clinical trials before nasal stem cells can become part of mainstream medicine.

Its also worth noting that for spinal cord injury, trials for recovery of function are not limited to the use of stem cells but include approaches focused on promoting health of surviving nerves (neuroprotection), surgery following injury, nerve transfers, electrical stimulation, external physical supports known as exoskeletons, nanotechnology and brain-machine interfaces.

Ultimately, determining which of these approaches will improve the lives of people with spinal injury can only be done through rigorous, ethical research.

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Yes there's hope, but treating spinal injuries with stem cells is not a reality yet - The Conversation AU