Stem Cell Clinic

I believe that this will be the next generation of medicine. Area … – WTHITV.com

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VINCENNES, Ind. (WTHI) In the past, new moms would be asked if they wanted to save their umbilical cord blood for future, personal use.

But at one area hospital, a new partnership is taking that concept and making it public.

Amelia Nance and her fianc just welcomed their second child on Monday, a baby boy named Finn.

The new mom says, Its neat to be a part of something thats growing in a positive way.

Nance just donated the umbilical cord, cord blood, placenta, and amniotic fluid from Finns birth to Life Line Stem Cell.

Its a partnership at Good Samaritan Hospital thats just weeks old.

However, officials say around 90% of patients take part in the program.

Margaret Suozzi, MSN/RN is the Director of Women & Children Services at Good Samaritan.

She says, Like any program we havent been up and running long enough to have monthly stats yet, but we anticipate that we will be pretty close to that even from the very beginning. And any time you start a new program, its always one of those things where theyre like, What does this mean? What do I do?'

One of the first questions asked is, Does this cost anything? The answer is no. It is a free service.

Since being a new or expecting mom is hard enough, Life Line has narrowed the donation process down to a questionnaire.

Nance says, The form is actually really simple its pretty laid out, open questions, it asks you about your history, your parents history.

If the tissues werent donated they would be properly disposed of by the hospital.

Suozzi says that could mean missing out on countless possibilities to change someone elses life.

Suozzi says, I believe that this will be the next generation of medicine. We are already finding a million things that stem cells can do for our existing patients in other areas: diabetics, wound care, Chrons disease, and many other things. And I think it is the tip of the ice berg. So for our patients to be able to donate to the cause and to be able to help others, is just one sign of Indiana hospitality.

Life Line Stem Cell allows the family that provides the tissue first dibs if a family member could benefit from the blood.

But after that, its donated to a registry for public use.

Thats part of the reason Nance decided she wanted to take part.

The new mom says, I would say its excellent. The fact that, again, it was absolutely no cost to me, it didnt hurt our child, and we could donate it and it could possibly help him out again or you know, one of my family members. Ive had family members thats died of cancer or different diseases. And its nice to know that there can possibly be research done with this blood that would help progress you know, a cure or even just something that would help prolong a positive future for somebody. Whether it be a kid or a child with a disease that might otherwise be painful or negative in their life.

A rep for Life Line Stem Cell says one placenta can be used to heal as many as 100 eyes.

He says amniotic fluid is showing great results in the healing process for burn victims too.

Tricia Crowe is a Life Line Stem Cell Training Manager. She says, It is important that families understand that we are only using hematopoietic blood cells that are found in the umbilical cord and are blood forming. They give rise to red blood cells, white blood cells and platelets.

Suozzi says Good Samaritan is the only hospital in the Southwest region that is offering this program.

For more information on stem cell donation, contact Good Samaritans Women and Infants Center at 812-885-3369 or click here.

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I believe that this will be the next generation of medicine. Area ... - WTHITV.com

Sickle Cell Trait in Blacks Can Skew Diabetes Test Results – Albany Democrat Herald

By Uncategorized

TUESDAY, Feb. 7, 2017 (HealthDay News) -- A blood test commonly used to diagnose and treat diabetes may be less accurate in black people who have the sickle cell anemia trait, a new study says.

The test is called hemoglobin A1C (HbA1C). An A1C reading of 5.7 or more indicates prediabetes or diabetes; below 5.7 is normal, says the American Diabetes Association.

But, the current study found that for blacks with a trait for sickle cell anemia, the A1C test may come back lower than it should. This discrepancy could lead to delays in diagnosis and treatment of diabetes, and it might also affect the management of known diabetes.

When the researchers compared the results of A1C tests to other measures that check blood sugar levels, they showed that when A1C readings were expected to be 6 percent, they only registered 5.7 percent for blacks with sickle cell trait.

"We want to make clinicians aware that things like race and hemoglobin traits can have an effect on A1C. If the A1C numbers don't jibe with blood glucose monitor numbers, this could potentially be a part of that," said Tamara Darsow.

Darsow, who wasn't involved in the study, is senior vice president of research and community programs with the American Diabetes Association.

The test measures the percentage of red blood cells that have become "glycated" over a two- to three-month period. Glycated essentially means the red blood cells have sugar attached to them. That can happen when blood sugar levels are too high (hyperglycemia).

Sickle cell anemia is an inherited disorder that affects the hemoglobin in red blood cells. Hemoglobin is the substance that carries oxygen to the body through the blood. Sickle cell disease causes the hemoglobin to form in a sickle shape instead of the normal rounded disc shape, according to the U.S. National Heart, Lung, and Blood Institute.

For people with sickle cell disease, red blood cells live 10 to 20 days instead of the normal 90 to 120 days.

However, someone with sickle cell trait doesn't have sickle cell disease. To get sickle cell disease, you must inherit the gene from both parents. If you only inherit the sickle cell gene from one parent, you have the sickle cell trait. People with sickle cell trait usually don't have any symptoms of the disease, according to the U.S. Centers for Disease Control and Prevention.

As many as 10 percent of black people in the United States have sickle cell trait, the study authors said. People from Mediterranean and Middle Eastern countries and other parts of the world can also have the trait or the disease, the study's researchers said. The current study's focus was on blacks.

"We found that in people with the sickle cell trait, A1C levels were significantly lower than in people without the sickle cell trait," said the study's lead author, Mary Lacy. She's a doctoral candidate at Brown University School of Public Health in Providence, R.I.

"Hemoglobin A1C is influenced by sugar levels, but also by how old the red blood cells are. Our hypothesis is that people with a sickle cell trait might have red blood cells that live a shorter period of time," Lacy explained.

The current study included more than 4,600 people -- about 1,600 from a study called CARDIA, and about 3,000 from the Jackson Heart Study. Their average age was 52. Both groups had their blood sugar levels measured with A1C, and also with fasting blood sugar tests. In addition, the CARDIA group was given two-hour oral glucose tolerance tests.

When the researchers only used A1C to diagnose diabetes, they saw a major difference -- 29 percent of blacks with a sickle cell trait were diagnosed with prediabetes versus 49 percent of blacks without the sickle cell trait.

Dr. Joel Zonszein is director of the clinical diabetes center at Montefiore Medical Center in New York City. He wasn't surprised by the study's results.

"The hemoglobin A1C is not a perfect test. Results vary, but it's still a very good test. Patients like it because it doesn't matter if they're fasting or not," he said.

Zonszein added that other blood disorders, such as iron deficiency anemia, kidney failure and late pregnancy can affect the results of an A1C test. "We're using a tool that's practical, but not 100 percent reliable," he said.

Darsow said there was already evidence of this discrepancy from smaller studies.

Also, the diabetes association addresses the difference in its Standards of Medical Care in Diabetes -- 2017.

"We don't have good evidence to make screening or treatment guidelines right now, but it's important to understand it can be a potential interference," Darsow said. "We highlight individualized care in our standards, and this is one of the potential things that could impact diabetes management."

The study was published Feb. 7 in the Journal of the American Medical Association.

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Sickle Cell Trait in Blacks Can Skew Diabetes Test Results - Albany Democrat Herald

15 School of Medicine researchers named CZ Biohub investigators – Stanford Medical Center Report

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Fifteen faculty members from the School of Medicine are among the 47 investigators announced today by the Chan Zuckerberg Biohub.

The CZ Biohub is an independent nonprofit medical research organization that has the goal of harnessing the power of science, technology and human capacity to cure, prevent or manage all disease. It is funded through a $600 million commitment by the Chan Zuckerberg Initiative, which was created by Facebook founder Mark Zuckerberg and his wife Priscilla Chan, MD.

The investigators were selected from the three institutions participating in the CZ Biohub: Stanford, UC-San Francisco and UC-Berkeley. Each of the investigators will be given a five-year appointment and up to $1.5 million for research in their respective areas of expertise. More than 700 researchers applied for the funding; the selections were made by an international panel of 60 scientists and engineers.

The investigators include both senior researchers and up-and-coming faculty.

The 47 CZ Biohub investigators were introducing today are quite literally inventing the future of life science research, said Stephen Quake, PhD, co-president of CZ Biohub and professor of bioengineering and applied physics at Stanford. The CZ Biohub is distinguished by our emphasis on technology and engineering, and our researchers are inventing tools to accelerate science for the good of humanity.

We are honored to have so many of our scientists selected to pursuetheirinnovative and ambitious projectsat the Chan Zuckerberg Biohub,said Lloyd Minor, MD, dean of the School of Medicine. If past is prologue, givingsuch inventivethinkersthe freedom to conduct fundamental research will result in trulyoutstanding discoveries, moving us toward a future wherewe can both cure and preventwhat today seems incurable and unpredictable.

The 15 medical school faculty members are:

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15 School of Medicine researchers named CZ Biohub investigators - Stanford Medical Center Report

Cellectis gets US ok to test its universal donor cancer cell therapy – BioPharma-Reporter.com

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US regulators have cleared French biotech Cellectis to trial its off-the-shelf blood cancer cell therapy, UCART123.

The product consists of T-cells modified to target the CD123 antigen on the surface of cancerous cells.

Jennifer Moore, VP of Communications at Cellectis, told us: "CELLforCURE, the largest commercial industrial facility for the production of innovative therapeutic cell therapies in Europe, a subsidiary of the biopharmaceutical group LFB, will perform the cGMP manufacturing of clinical batches of Cellectis allogeneic CART cells."

Unlike autologous cell therapies that are made from the specific patients own cells, UCART123 is composed of lymphocytes harvested from an unrelated, so called universal donor.

The advantage of this allogenic approach is that developers can make and store stocks of cell therapies rather than having to produce a specific batch for each patient, which makes such treatments cheaper according to Loan Hoang-Sayag, Cellectis Chief Medical Officer.

Hoang-Sayag said: Cellectis allogeneic UCART products have the potential to create an important shift with regard to availability, and cost-effectiveness, to make these therapies widely accessible to patient population across the world.

Gene editing

UCART123 - which Cellectis claims is the first allogenic cell therapy to be approved for trials by the US Food and Drug Administration (FDA) -will be tested as a treatment for patients with acute myeloid leukemia (AML) and blastic plasmacytoid dendritic cell neoplasm (BPDCN). The Phase I clinical studies due to start in the first half of the year.

Like other therapies in Cellectis pipeline, UCART123 is produced using Talen gene editing to inset genes that encode a chimeric antigen receptor (CAR) that targets the CD123 antigen.

The gene editing method is also used to prevent the T-cells of which UCART123 is composed from interacting with non-target proteins, thereby reducing side-effects

UCART therapies

One of Cellectis UCART therapies was used to successfully treat a baby girl with leukaemia whose disease had progressed despite chemotherapy.

According to the results of a study published in 2015 , the girl received a single dose (4.5x106/kg) of Cellectis allogeneic engineered T-cell product UCART19 and two months later the girl was cleared of leukaemia.

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Cellectis gets US ok to test its universal donor cancer cell therapy - BioPharma-Reporter.com

Stem Cell Network

By Stem Cell Treatment

It may sound like science fiction, but the research of Stephanie Willerth of the University of Victoria is proving to be anything but. A patients adult cells will be reprogrammed back into their stem cell state.....

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Septic shock is the most severe form of infection seen in intensive care units (ICUs), a sneaky and unpredictable condition according to the Ottawa Health Research Institutes Dr. Lauralyn McIntyre...

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Its been 12 years since Canada passed legislation governing research on human embryos. As it stands, the legislation has not kept pace with the science... ...

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Umbilical cord blood can be a promising source of hematopoietic stem cells (HSCs), used in treating blood diseases...

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Liver transplants save lives, plain and simple. But they also sentence recipients to a lifetime of immune-suppressive drugs, to prevent the body from rejecting the foreign addition....

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Its an exciting time for the Stem Cell Network! We have been very busy over the past few months ensuring that we are able to deliver on our mandate and see research funding and training opportunities provided for...

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Stem Cell Network

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

By Stell Cell Research

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

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

By Stell Cell Research

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

By Stell Cell Research

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

The next weapon against brain cancer may be human skin – The Verge

By Stem Cell Clinic

Human skin can be morphed into genetically modified, cancer-killing brain stem cells, according to a new study. This latest advance has only been tested in mice but eventually, its possible that it could be translated into a personalized treatment for people with a deadly form of brain cancer.

The study builds on an earlier discovery that brain stem cells have a weird affinity for cancers. So researchers, led by Shawn Hingtgen, a professor at University of North Carolina at Chapel Hill, created genetically engineered brain stem cells out of human skin. Then they armed the stem cells with drugs to squirt directly onto the tumors of mice that had been given a human form of brain cancer. The treatment shrank the tumors and extended survival of the mice, according to results recently published in the journal Science Translational Medicine.

The treatment shrank the tumors and extended survival

Usually we think about stem cell therapy in the context of rebuilding or regrowing a broken body part like a spinal cord. But if they could be modified to become cancer-fighting homing missiles, it would give patients with a deadly and incurable brain cancer called glioblastoma a better chance at survival. Glioblastomas typically affect adults, and are highly fatal because they send out a web of cancerous threads. Even when the main mass is removed, those threads remain despite chemotherapy and radiation treatment. This cancer has caused a number of high-profile deaths including Senator Edward (Ted) Kennedy in 2009, and possibly Beau Biden more recently. Approximately 12,000 new cases of glioblastoma are estimated to be diagnosed in 2017.

We really have no drugs, no new treatment options in years to even decades, Hingtgen says. [We] just really want to create new therapy that can stand a chance against this disease.

But theres a problem: brain stem cells arent exactly easy to get. Brain stem cells, more properly known as neural stem cells, hang out in the walls of the brains irrigation canals areas filled with cerebrospinal fluid, called ventricles. They generate the cells of the nervous system, like neurons and glial cells, throughout our lives.

They could be modified to become cancer-fighting homing missiles

A research group at the City of Hope in California conducted a clinical trial to make sure it was safe to treat glioblastoma patients with genetically engineered neural stem cells. But they used a neural stem cell line that theyd obtained from fetal tissue. Since the stem cells werent the patients own, people who were genetically more likely to reject the cells couldnt receive the treatment at all. For the people who could, treatment with the neural stem cells turned out to be relatively safe although at this phase of clinical trials, it hasnt been particularly effective.

More personalized treatments have been held up by the challenge of getting enough stem cells out of the patients own brains, which is virtually impossible, says stem cell scientist Frank Marini at the Wake Forest School of Medicine, who was not involved in this study. You cant really generate a bank of neural stem cells from anybody because you have to go in and resect the brain.

So instead, Hingtgen and his colleagues figured out a way to generate neural stem cells from skin which in the future, could let them make neural stem cells personalized to each patient. For this study, though, Hingtgen and his colleagues extracted the skin cells from chunks of human flesh leftover as surgical waste. That really is the magic piece here, Marini says. Now, all of a sudden we have a neural stem cell that can be used as a tumor-homing vehicle.

That really is the magic piece here.

Using a disarmed virus to infect the cells with a cocktail of new genes, the researchers morphed the skin cells into something in between a skin cell and a neural stem cell. People have turned skin cells back into a more generalized type of stem cell before. But then turning those basic stem cells into stem cells for a certain organ like the brain takes another couple of steps, which takes more time. Thats something that people with glioblastoma dont have.

The breakthrough here is that Hingtgens team figured out how to go straight from skin cells to something resembling a neural stem cell in just four days. The researchers then genetically engineered these induced neural stem cells to arm them with one of two different weapons: One group was equipped with an enzyme that could convert an anti-fungal drug into chemotherapy, right at the cancers location. The other was armed with a protein that binds to the cancer cells and makes them commit suicide in an orderly process called apoptosis.

The researchers tested these engineered neural stem cells in mice that had been injected with human glioblastoma cells, which multiplied out of control to create a human cancer in a mouse body. Both of the weaponized stem cell groups were able to significantly shrink the tumors and keep the mice alive by about an extra 30 days (for scale, mice usually live an average of two years).

Were working as fast as we can.

But injecting the cells directly into the tumor doesnt really reflect how the therapy would be used in humans. Its more likely that a person with glioblastoma would get the bulk of the tumor surgically removed. Then, the idea is that these neural stem cells, generated from the patients own skin, will be inserted into the hole left in the brain. So, the researchers tried this out in mice, and the tumors that regrew after surgery were more than three times smaller in the mice treated with the neural stem cells.

Its a promising start, but it could take a few years still before its in the clinic, Hingtgen says. He and his colleagues started a company called Falcon Therapeutics to drive this new therapy forward. Were working as fast as we can, Hingtgen says. We probably cant help the patients today. Hopefully in a year or two, well be able to help those patients.

One of the things theyll have to figure out first is whether the neural stem cells can travel the much bigger distances in human brains, and whether theyll be able to eliminate every remaining cancer cell. The caveats on this are that, of course, its a mouse study, and whether or not that directly converts to humans is unclear, Marini says. Still, he adds, Theres a very high probability in this case.

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The next weapon against brain cancer may be human skin - The Verge

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