The decision to become a living organ donor is a significant one and, among the many factors to weigh, donors should consider potential financial consequences of their altruism.

Medical costs associated with organ, tissue and bone marrow donations don't fall on the donor; they typically are paid by the recipient and often covered by insurance, Medicare or Medicaid.

But other related expenses including travel, lodging and loss of wages due to time out of work are the donor's responsibility.

Most of the roughly 6,000 living donations that occur annually are between relatives and close friends, people who have a vested interest in the recipient's outcome, according to the U.S. Department of Health and Human Services (HHS).

But even people who wouldn't hesitate to help a loved one should know about potential costs.

When Sally McCartin of North Branford donated a kidney to a fellow "hockey mom" in 2013, she knew she'd have to take time off from her job at the state's Department of Revenue Services.

"I was worried about the first two weeks that I would be out of work because I had used up all my sick time getting the necessary testing done to donate," she says. "Initially, I would have gone with no pay for the first two weeks."

The recipient of her kidney offered to cover McCartin's loss of wages, but the expense ultimately was covered by financial donations from McCartin's coworkers and contributions from her union, so she bore none of the cost.

For others, out-of-pocket expenses can be problematic and may deter some from donating altogether, says Sen. Martin Looney, D-New Haven. He proposed legislation in January that would help ease some of the burden.

Under his bill, people who donate organs or bone marrow after Jan. 1, 2017, could deduct up to $10,000 from their income, under the state personal income tax, to cover unreimbursed costs of travel, lodging and lost wages they incur as a result of donating. The bill also aims to allow state employees, beginning in 2018, to take up to 30 days of paid leave from work for organ donation and up to seven days of paid leave for bone marrow donation.

The legislation, co-sponsored by Sen. Catherine Osten, D-Columbia, is before the General Assembly's Public Health Committee and slated to receive a public hearing. It's intended to "limit potential barriers to people agreeing to be a live donor," Looney says.

The matter hits close to home for the senator, who suffered from kidney failure and received a kidney donation from a live donor in December. Nineteen states already have similar laws on the books, Looney says.

Some donors may have some expenses covered by their own insurance, depending on their plan, he says, but not all do.

"In some cases, the out-of-pocket expenses are minimal to the donor, and in other cases they can be substantial," he says, especially when it comes to missed work. "The donor is going to be out of work probably for a minimum of two weeks after the procedure. That would probably be the biggest hardship of all."

Many things can be donated by living donors, according to the HHS, including six vital organs: heart, kidneys, pancreas, lungs, liver and intestines. (In some cases a portion of the organ, such as the liver, is donated. Living heart donations are rare but happen. They occur when a donor has a pulmonary condition that necessitates removal of the heart and lungs for new ones lungs-only operations are considered riskier but the donor's heart is in good enough condition to be transplanted in another patient.)

Donors also may give certain tissues including skin, cornea and blood vessels as well as bone marrow, stem cells and umbilical cord blood.

April has been dubbed "National Donate Life Month," an effort by nonprofit advocacy group Donate Life America to encourage people to register as organ, eye and tissue donors.

About 20 to 25 living donations occur annually at the Hartford Hospital Transplant Program, according to registered nurse and Living Donor Transplant Coordinator Kari Rancourt. Most of them are kidney transplants.

"We do talk a little [with prospective donors] about out-of-pocket expenses," she says. Some donors get help covering costs through social fundraising platforms like GoFundMe, she says.

There also are foundations that offer grants to help would-be donors afford travel and lodging, she adds, which have "helped limit barriers to donations."

McCartin, who donated at Yale-New Haven Hospital, says that she did have to consider the potential expense but that wouldn't have stopped her from donating. She is so passionate about it, she recently began Kid-U-Not, a Connecticut chapter of the American Living Organ Donor Fund that raises funds to assist donors.

"I felt that, if I was healthy enough to donate, then it was a no-brainer," she says. "How could I not help a single mom of three children? I would hope that if I was ever in the same situation someone would step up for me."

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Living Organ Donors: Proposed Tax Deduction Could Help Defray Donation Expenses - Hartford Courant

Healthy cell function relies on well orchestrated gene activity. Via a fantastically complex network of interactions, around 30,000 genes cooperate to maintain this delicate balance in each of the37.2 trillion cellsin the human body.

Broadly speaking, cancer is a disruption of this balance by genetic changes, or mutations. Mutations can trigger over-activation of genes that normally instruct cells to divide, or inactivation of genes that suppress the development of cancer. When a mutated cell divides, it passes the mutation down to its daughter cells. This leads to the accumulation of non-functioning, abnormal cells that we recognise as cancer.

Our laboratoryis focused on understanding how one particular cancer chronic myeloid leukaemiaor CML works. Each year more than 700 patients in the UK andover 100,000worldwide are diagnosed with CML. After recent advances,almost 90%of patients under the age of 65 now survive for more than five years.

Sign Up for DDD's Webinar on Multicomponent Amorphous Solid Dispersion Systems on March 28th!:http://buff.ly/2n5iEqr

But in the vast majority of patients CML is currently incurable and lifelong treatment means that patients must live with side effects and the chance of drug resistance arising. With increasing numbers of CML patients surviving (and treatment costing between 40,000 and 70,000 per patient a year), increasing strain is being placed on health services.

A single mutation

CML is perhaps unique in cancers in that a single mutation, namedBCR-ABL, underlies the disease biology. This mutation originates in a singleleukaemic stem cell, but is then propagated throughout the blood and bone marrow as leukaemia cells take over and block the healthy process of blood production. The presence of BCR-ABL affects the activity of thousands of genes, in turn preventing these cells from fulfilling their normal function as blood cells.

Drugsthat specifically neutralise the aberrant effects of this mutation were introduced to the clinic from the early 2000s. These drugs have revolutionised CML patient care. Many are now able to live relatively normal lives with their leukaemia under good control.

But while these drugs kill the more mature daughter cells of the originally mutated leukaemia stem cell, they have not fully lived up to their initial billing as magic bullets in the fight against cancer. This is because the original seed population of leukaemic stem cellsevade therapy,lying dormant in the bone marrowto stimulate new cancer growth when treatment is withdrawn.

To truly cure CML we must expose, understand the inner workings of, and uproot the leukaemia stem cells. And to do this, we need to learn more about them. How do they survive the treatment that so readily kills their more mature counterparts? Which overactive or inactivated genes protect them?

We believe that the answers to these questions lie in the analysis of biological big data. Genome-scale technologies now allow scientists to measure the activity (or expression) of every gene in the genome simultaneously, in any given population of cells, or even at the level of a single cell. Comparison of expression data generated from leukaemia stem cells with the same data generated from healthy blood stem cells will reveal single genes or networks of genes potentially targetable in the fight against leukaemia.

Big data to the rescue

In a project funded by Bloodwise and the Scottish Cancer Foundation, we have createdLEUKomics. This online data portal brings together a wealth of CML gene expression data from specialised laboratories across the globe, including our own at the University of Glasgow.

Our intention is to eliminate the bottleneck surrounding big data analysis in CML. Each dataset is subjected to manual quality checks, and all the necessarycomputational processingto extract information on gene expression. This enables immediate access to and interpretation of data that previously would not have been easily accessible to academics or clinicians without training in specialised computational approaches.

Consolidating these data into a single resource also allows large-scale, computationally-intensive research efforts by bioinformaticians (specialists in the analysis of big data in biology). From a computational perspective, the fact that CML is caused by a single mutation makes it an attractive disease model for cancer stem cells. However, existing datasets tend to have small sample numbers, which can limit their potential.

The more samples available, the higher the power to detect subtle changes that may be crucial to the biology of the cancer stem cells. By bringing all the globally available CML datasets together, we have significantly increased the sample size, from two to six per dataset to more than 100 altogether. This offers an unprecedented opportunity to analyse gene expression data to expose underlying mechanisms of this disease.

As of March 2017, theportalis up and running in the public domain. We are planning to tour Scotland and present at international conferences, aiming to train researchers in how best to exploit this new resource. Ultimately, we hope that this tool will lead to new ideas and approaches, and attract more funding, in the fight against CML. And while we continue to expand our representation of CML data in real time from research centres all over the world, we also plan to begin incorporating data from other types of leukaemia.

In recent years, targeted therapies have becomehugely importantin cancer research. By providing these data to the CML research community withinLEUKomics, we hope to mobilise new research into cancer-causing leukaemic stem cells, and ultimately design treatments to target them without affecting healthy cells. Our database provides a critical stepping stone in this process.

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How Big Data is Being Mobilized in the Fight Against Leukemia - Drug Discovery & Development

This week a very special delivery was made from space that will help further research that could eventually lead to a mind-blowing, futuristic way to cure diseases: shooting unmanned satellite wombs into orbit and then retrieving from them batches of stem cells that can be used to treat patients. Regardless of the outcome, the scientific experiment will still advance our knowledge of these unique cells.

On Thursday Dr. Abba Zubairat the Mayo Clinic in Jacksonville, Florida, received frozen stem cells grown at the International Space Station. The package was part of the 5,400 pounds of scientific samples and equipment that splashed down on Sunday off the coast of California inside a SpaceX Dragon-10 capsule completing a historic round-trip mission.

Up there, one of the astronauts helped us to image the cells, harvest the cells and freeze them in a way that we can use them here on Earth and compare them to cells we grew here in the lab, Zubair, the principal investigator of the stem cell experiment, told Salon.

Zubairs team will look to see if the culture grown in the near-zero gravity of low-space orbit, about 250 miles above the Earths surface, results in healthier cells than onesgrownin aterrestrial lab. If so then it would helpconfirm the theory that microgravity, which resembles the weightless-likebuoyancyof female womb, is best environment for growing stemscells.

Stem cells, from which all other types of cells originate, are the bodys raw materials, and as such offer immense potential to cure many diseases. Doctors already use stem cells forbone-marrow transplants and treating blood-related diseases like leukemia, as well asfor some eye-related disorders. Researchers believe were only in the very early stages of developing revolutionary stem cell therapiesto combat cancer, Alzheimers disease, Parkinsons disease, Type 1 diabetes, heart disease and strokes. In the future, stems cellscience could even lead to growing organs in a lab that can be transplanted into humans.

But stem cells are finicky. As they replicate in a lab, many of them develop imperfections and have to be discarded. It can take a month to grow the roughly 200,000 cells needed to treat one patient, Zubair said. Gravity might be the culprit.

In nature, these cells start their life after an egg is fertilized. Humans, right from conception, develop almost in a microgravity environment, Zubair said. Fetuses develop in amniotic fluid. Theyre buoyant, which cancels the effect of gravity because theyre suspended in a liquid. Thats how three-dimensional growth in a fluid environment is possible. We think gravity does play a role in the shape and development of the cells and how organs develop.

In other words, if the cells are suspended in fluid, they can grow and move in any direction, producing more of them, compared withhow they grow on a flat surface, like in a petri dish.

This is why stem cells are typically grown in a bioreactor, a common bioengineering tool that gently stirswater containing the seed cells and certain nutrients that promote growth. But because of the way gravity affectsfluids, many of the cells become damaged and cant be used for treatment. (In the language of physics, the problem has to do with something called shearing force.) By placing a bioreactor in the microgravity of orbit, the effects of gravity on liquid mechanics is virtually eliminated.

If growing stem cells in spaceproves to be efficient, thats when things get interesting. Growing stem cells at the International Space Station is anexperimental endeavor, so its not really a viable place to begin manufacturing themin great quantities. But theoretically, Zubair says, bioreactor satellites could be put into orbit and left there to grow cells until theyre remotely called back to Earth or sent wherever future interplanetary pilgrims wind up. As the cost of sending small satellites into low orbit falls, this system could be commercially viable.

There are companies that are interested in developing a floating lab in space to grow not only stem cells but also tissues and organs down the road for human use or for use elsewhere as we hopefully colonize other planets, like Mars, Zubair said.

This might seem out of this world, but the technology for growing stem cells remotely already exists. If space is the place to grow human parts and this research will help to determine that then designing systems and deploying these bioreactor space wombs might not be that far off in the future.

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Space wombs for stem cells: Satellites could help accelerate the discovery of disease cures - Salon

Mumbai:A 45-year-old man, who was suffering from 45 per cent burns due to a chemical spill at work, has been healed with stem cell treatment, said the authorities at a hospital here on Friday.

Ram Naik (name changed) was brought to city-based StemRx Bioscience hospital after receiving first aid in another hospital. Nearly 45 per cent of his upper body was burned due to a chemical spill during work.

The impact of the burns led to a charred look on his face and body. Also, joint mobility due to the burns was reduced. The outer layer of the skin was affected, facial burns were of grade II level and in some instances grade III burns were also present, leading to deeper structures like the subcutaneous tissue also being affected.

According to the doctors, burn wound healing involves a series of complex processes, with healing time and scar tissue being the most important parameters that affect treatment outcomes. Burn injuries, especially severe ones, are proving to have devastating effects on the affected patients.

They said that stem cells have been recently applied in burn wounds to promote superior healing of the wounds. Not only have stem cells been shown to promote better and faster healing of the burn wounds, they are also capable of decreasing inflammation and prevent scar progression and fibrosis.

Therefore, the doctors decided to provide Naik stem cell treatment.

Regenerative Medicine researcher at Stemrx Bioscience hospital Pradeep Mahajan said that within two days, a notable improvement in his condition was observed and the swelling and charred appearance started reducing.

"Mild eyelid movements were noticed and on the third day the burns started drying on the face and he could open his mouth and eyes. Growth factors derived from platelets, cells, fibroblasts, collagen-based gel etc. was used during treatment. In addition, in areas with deep burns, sheets of PGLA coated with cells and growth factors were used," said Mahajan, adding that different medication and treatments were imparted and closed dressing was avoided.

"Blood transfusion and supplementary fluids were given intravenously to maintain systemic homeostasis," said Mahajan.

Stating that on 5th and 6th day following treatment, dry scales from the face and body started peeling off, the doctor's team also observed impressive changes such as new skin forming within a week of treatment with cells and growth factors.

By conventional modalities, it takes more than eight weeks for the patient to heal and many additional months for the patient to be able to regain joint and facial movements.

"By the 10th day of the treatment, dry scales completely peeled off and by the 14th day the patient had no tenderness or burning pain. Joint movements became free as well, Steady rate of progression of healthy skin formation is being noticed. Areas with deep burns are also healing at a rapid rate and I am confident that within a month we will accomplish thorough healing and the patient will be back to normal," Mahajan said.

Medical sciences say that such cases are challenging to manage considering the degree of impairment they result in due to prolonged healing period. Also, through conventional therapeutic modalities healing occurs with scar formation and results in contractures. Chances of systemic complications and infection are also high.

However according to the medical team, by using stem cells, the natural healing potential of the body is used, leading to reduction of healing time and promoting regeneration of affected tissues. This also reduces the mental trauma and financial burden that a patient goes through when under conventional management.

"Stem cell-based therapy has offered a novel and powerful strategy in almost every medical specialty including burns and wound management. Stem cells have proven to have tremendous potential in enhancing wound healing and facilitating skin regeneration," Mahajan said.

Originally posted here:
Man with 45% burns healed with stem cell treatment - Zee News

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

The applications could be very significant, from studying genetic diseases in a dish to generating possible regenerative cures from the patients own cells.

Its actually quite remarkable that it happens, says Stelios T. Andreadis, PhD, professor and chair of UBs Department of Chemical and Biological Engineering, who recently published a paper on the results in the journal Stem Cells.

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

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

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

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

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

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

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

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

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

The research, described in the journal Stem Cells under the title Reprogramming Postnatal Human Epidermal Keratinocytes Toward Functional Neural Crest Fates, was supported by grants from the National Institutes of Health.

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From skin to brain: Stem cells without genetic modification - UB News Center

The study consists of donor and patient pairs for allogeneic hematopoietic cell transplantation. The first part of the study, which is nearing completion, is intended to enroll an initial cohort of 10 donor and recipient pairs, consisting of patients with advanced hematological malignancies and their HLA-matched sibling donors. Interim results show that a single injection of BL-8040 mobilized sufficient amounts of cells required for transplantation at a level of efficacy similar to that achieved by using 4-6 injections of G-CSF, the current standard of care. Furthermore, all recipients transplanted so far have experienced a successful neutrophil engraftment. The recipients will be followed for one year to assess acute and chronic GVHD events. As for the donors, BL-8040 treatment was safe and well tolerated.

Philip Serlin, Chief Executive Officer of BioLineRx, stated, "We are very encouraged by these initial results of the Phase 2 clinical trial for assessing BL-8040, our lead oncology and hematology platform, as a single agent for hematopoietic stem cell mobilization for allogeneic transplantation. Hematopoietic stem cells are increasingly used as part of the treatment regimen for certain types of hematological cancers, as well as for severe anemia and immune deficiency disorders. These results, supporting BL-8040 as a one-day dosing and up-to-two-day collection regimen, for rapid mobilization of substantial amounts of stem cells, represent a significant improvement over the current standard of care, which requires four-to-six daily injections of G-CSF and one-to-four apheresis sessions.If there are no safety concerns regarding graft failure or rejection after the interim safety review of donor-recipient pairs participating in Part 1 of the study, we will continue with Part 2 of the study, which will permit enrollment of recipients with either matched sibling or haploidentical donors, up to a total enrollment in the study of 24 donor-recipient pairs. We are looking forward to the topline results expected by the end of 2017."

"We continue our efforts to maximize the potential of our unique BL-8040 oncology platform, with multiple clinical studies for additional indications up and running or expected to start in 2017, including several combination studies with immune checkpoint inhibitors and a registration study in stem-cell mobilization for autologous transplantation," added Mr. Serlin.

The Phase 2 open-label study is conducted in collaboration with the Washington University School of Medicine, Division of Oncology, and will enroll up to 24 donor/recipient pairs, aged 18-70. The trial is designed to evaluate the ability of BL-8040, as a single agent, to promote stem cell mobilization for allogeneic hematopoietic cell transplantation. On the donor side, the primary endpoint of the study is the ability of a single injection of BL-8040 to mobilize sufficient amounts of cells for transplantation following up to two apheresis procedures. On the recipient side, the study aims to evaluate the time to engraftment rate following transplantation of the BL-8040 collected graft.

The study will also evaluate the safety and tolerability of BL-8040 in healthy donors, as well as graft durability, the incidence of grade 2-4 acute and chronic GVHD, and other recipient related parameters in patients who have undergone transplantation of hematopoietic cells mobilized with BL-8040.

About BL-8040

BL-8040 is a short peptide for the treatment of acute myeloid leukemia, solid tumors, and certain hematological indications. It functions as a high-affinity antagonist for CXCR4, a chemokine receptor that is directly involved in tumor progression, angiogenesis, metastasis and cell survival. CXCR4 is over-expressed in more than 70% of human cancers and its expression often correlates with disease severity. In a number of clinical and pre-clinical studies, BL-8040 has shown robust mobilization of cancer cells from the bone marrow, thereby sensitizing these cells to chemo- and bio-based anti-cancer therapy, as well as a direct anti-cancer effect by inducing apoptosis. In addition, BL-8040 has also demonstrated robust stem-cell mobilization, including the mobilization of colony-forming cells, and T, B and NK cells. BL-8040 was licensed by BioLineRx from Biokine Therapeutics and was previously developed under the name BKT-140.

About Stem Cell Mobilization

High-dose chemotherapy followed by hematopoietic cell transplantation has become an established treatment modality for a variety of hematologic malignancies, including multiple myeloma, as well as various forms of lymphoma and leukemia. Modern peripheral stem-cell harvesting often replaces the use of traditional surgical bone marrow stem-cell harvesting. In the modern method, stem cells are mobilized from the bone marrow using granulocyte colony-stimulating factor (G-CSF), often with the addition of a mobilizing agent such as Plerixafor (Mozobil), harvested from the donor's peripheral blood by apheresis, and infused to the patient after chemotherapy ablation treatment.

An allogeneic hematopoietic cell transplant involves matching a patient's tissue type, specifically their human leukocyte antigen (HLA) tissue type, with that of a related or unrelated donor. HLA proteins are found on all cells of our body and are the main way the immune system tells the difference between our own cells and foreign cells. The closer the HLA match between a donor and recipient, the greater the chance a transplant will be successful. If the HLA match is not close enough, the donor's immune system, which accompanies the donated stem cells, recognizes the HLA mismatch, and will attack the recipient's tissues. This process is known as graft versus host disease (GVHD).

Approximately 70% of people with a hematological malignancy or bone marrow failure syndrome who need an allogeneic transplant have an HLA-identical sibling or unrelated donor available. For patients who need a stem cell transplant but do not have an HLA-matched related or unrelated donor, recent medical advances have made possible the use of a partially matched or haploidentical related donor. A haploidentical related donor is usually a 50% match to the recipient and may be the recipient's parent, sibling or child.

The advantage of having a haploidentical transplant is thatit increases the chance offinding a donoras almost everyone has at least one haploidentical relative. Relatives can usually be asked to donate stem cells much more quickly than unrelated volunteer donors, particularly when the volunteer donors live in other countries, thereby allowing transplants to be done in a more timely manner.

With improvements in medical treatment, complications of a haploidentical transplant, such as GVHD, rejection of the graft and slow recovery of the immune system appear not to be increased compared to transplants using HLA-matched related or unrelated donors. Since this is a relatively new approach to stem cell transplantation, a haploidentical transplant is a treatment option that is not offered at all treatment centers, but is becoming more common.

About BioLineRx

BioLineRx is a clinical-stage biopharmaceutical company focused on oncology and immunology. The Company in-licenses novel compounds, primarily from academic institutions and biotech companies based in Israel, develops them through pre-clinical and/or clinical stages, and then partners with pharmaceutical companies for advanced clinical development and/or commercialization.

BioLineRx's leading therapeutic candidates are: BL-8040, a cancer therapy platform, which has successfully completed a Phase 2a study for relapsed/refractory AML and is in the midst of a Phase 2b study as an AML consolidation treatment and a Phase 2 study in stem cell mobilization for allogeneic transplantation; and BL-7010 for celiac disease and gluten sensitivity, which has successfully completed a Phase 1/2 study. In addition, BioLineRx has a strategic collaboration with Novartis for the co-development of selected Israeli-sourced novel drug candidates; a collaboration agreement with MSD (known as Merck in the US and Canada), on the basis of which the Company has initiated a Phase 2a study in pancreatic cancer using the combination of BL-8040 and Merck's KEYTRUDA; and a collaboration agreement with Genentech, a member of the Roche Group, to investigate the combination of BL-8040 and Genentech's Atezolizumab in several Phase 1b studies for multiple solid tumor indications and AML.

For additional information on BioLineRx, please visit the Company's website athttp://www.biolinerx.com, where you can review the Company's SEC filings, press releases, announcements and events. BioLineRx industry updates are also regularly updated onFacebook,Twitter, andLinkedIn.

Various statements in this release concerning BioLineRx's future expectations constitute "forward-looking statements" within the meaning of the Private Securities Litigation Reform Act of 1995. These statements include words such as "may," "expects," "anticipates," "believes," and "intends," and describe opinions about future events. These forward-looking statements involve known and unknown risks and uncertainties that may cause the actual results, performance or achievements of BioLineRx to be materially different from any future results, performance or achievements expressed or implied by such forward-looking statements. Some of these risks are: changes in relationships with collaborators; the impact of competitive products and technological changes; risks relating to the development of new products; and the ability to implement technological improvements. These and other factors are more fully discussed in the "Risk Factors" section of BioLineRx's most recent annual report on Form 20-F filed with the Securities and Exchange Commission on March 10, 2016. In addition, any forward-looking statements represent BioLineRx's views only as of the date of this release and should not be relied upon as representing its views as of any subsequent date. BioLineRx does not assume any obligation to update any forward-looking statements unless required by law.

Contacts: PCG Advisory Vivian Cervantes Investor Relations +1-212-554-5482 vivian@pcgadvisory.com

or

Tsipi Haitovsky Public Relations +972-52-989892 tsipihai5@gmail.com

SOURCE BioLineRx Ltd.

Excerpt from:
BioLineRx Provides Update on Phase 2 Open-Label Study for BL-8040 as Novel Stem Cell Mobilization Treatment - PR Newswire (press release)

Testing the efficacy of new gene therapies more efficiently ... Science Daily Using a new cellular model, innovative gene therapy approaches for the hereditary immunodeficiency Chronic Granulomatous Disease can be tested faster and ... and more »

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Testing the efficacy of new gene therapies more efficiently ... - Science Daily

By Staff

Electroacupuncture triggers a neurological response that releases stem cells that can relieve injury-induced pain, and help promote tissue repair, says a study in the journal Stem Cells led by Indiana University School of Medicine.

The school outlined its findings in a media release:

Electroacupuncture is a form of acupuncture that uses a small electrical current to augment the ancient Chinese medical practice of inserting fine needles into the skin at pre-determined points throughout the body.

For the study, a team of more than 40 scientists at institutions in the United States and South Korea was led by four senior authors including IU School of Medicines Maria B. Grant, MD, Marilyn Glick Professor of Ophthalmology and co-corresponding author; Mervin C. Yoder, MD, IU Distinguished Professor, Richard and Pauline Klingler Professor of Pediatrics, associate dean for entrepreneurial research at IU School of Medicine, director of the Herman B Wells Center for Pediatric Research and co-corresponding author; and Fletcher A. White, PhD, Vergil K. Stoelting Chair ofAnesthesia, professor of anesthesia, pharmacology and toxicology.

This work is a classic example of the power of team science, where investigators in different institutions with specific expertise worked together to unravel the complexity of how electroacupuncture works to help the body respond to stressors, said Dr. Yoder.

The researchers performed a series of lab tests involving humans, horses and rodents that follow the effects of electroacupuncture from the stimulus of the needle all the way to the brain, resulting in the release of reparative mesenchymal stem cells (MSCs) into the bloodstream.

Depending on the species, electroacupuncture led to activation of the hypothalamusa part of the brain that controls the nervous system and involuntary bodily functions such as heart rate and digestionwithin nine to 22 minutes. The stem cells were mobilized within two hours.

The acupuncture stimulus were giving these animals has a rapid effect on neuroanatomical pathways that connect the stimulus point in the arm to responsive neurons in the spinal cord and into a region in the brain called the hypothalamus. In turn, the hypothalamus directs outgoing signals to stem cell niches resulting in their release, said Dr. White, who is a neuroscientist at the Richard L. Roudebush VA Medical Center in Indianapolis.

The researchers found electroacupuncture treatments resulted in higher thresholds for injury-induced pain, as well as considerable increases in the presence of a type of collagen that promotes tendon repair and anti-inflammatory cells known to be predictors of faster healing time.

Dr. White said these findings could lead to new strategies for tissue repair and pain management related to injuries.

We could potentially capture the MSCs from an individuals blood following electroacupuncture and save the cells for future re-introduction in the patient post-surgery or to treat chronic pain due to an injury, he said.

The horses used in the study had been injured during training for international dressage competitions, and the six people who took part were healthy volunteers, who still showed activation of their hypothalamus through brain imaging.

See more here:
Electroacupuncture Releases Stem Cells to Relieve Pain and More, Study Finds - National Pain Report

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Cesca Therapeutics Inc. (NASDAQ: KOOL), a market leader in automated cell processing and point-of-care, autologous cell-based therapies, today announced the publication in a peer reviewed journal of data from its Critical Limb Ischemia (CLI) feasibility study utilizing the Companys innovative point-of-care technology. The report was published in the Stem Cells International and is available online at https://www.hindawi.com/journals/sci/2017/4137626/ref/.

Results from the seventeen patient clinical study titled, Safety and Effectiveness of Bone Marrow Cell Concentrate in the Treatment of Chronic Critical Limb Ischemia Utilizing a Rapid Point-of-Care System, (the Study) were obtained using Cescas automated point-of-care technology. The single treatment procedure was performed at the patients bedside and took less than 60 minutes. The Study results showed significant improvement in wound healing, rest pain and six-minute walking distance, along with significant reduction in intermittent claudication pain following the treatment.

Dr. Venkatesh Ponemone, Study Director and Executive Director of TotipotentRX, a Cesca subsidiary and the corresponding author of the article commented, We are targeting difficult to treat or life threatening conditions such as CLI with our autologous, cell-based therapies. We believe our innovative point-of-care cell processing systems, such as those used in the Study, can play an important role in optimizing the quality and quantity of target cells used to improve patient outcomes.

Dr. Xiaochun "Chris" Xu, Cesca's Interim CEO added, We are pleased that the Study was recognized and published in a peer reviewed journal. The encouraging data highlights Cescas capability to develop effective automated cellular processing systems. We welcome strategic partners to help us further refine their use in larger clinical settings.

About Cesca Therapeutics Inc.Cesca Therapeutics Inc. (www.cescatherapeutics.com) is engaged in the research, development, and commercialization of cellular therapies and delivery systems for use in regenerative medicine. The Company is a leader in the development and manufacture of automated blood and bone marrow processing systems that enable the separation, processing and preservation of cell and tissue therapeutics. These include:

Forward-Looking StatementThe statements contained herein may include statements of future expectations and other forward-looking statements that are based on managements current views and assumptions and involve known and unknown risks and uncertainties that could cause actual results, performance or events to differ materially from those expressed or implied in such statements. A more complete description of risks that could cause actual events to differ from the outcomes predicted by Cesca Therapeutics' forward-looking statements is set forth under the caption "Risk Factors" in Cesca Therapeutics annual report on Form 10-K and other reports it files with the Securities and Exchange Commission from time to time, and you should consider each of those factors when evaluating the forward-looking statements.

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Cesca Therapeutics (KOOL) Announces CLI Feasibility Study Published in Stem Cells International - StreetInsider.com

March 20, 2017 by Melissa Gaskill Cultured stem cells. Credit: BioServe Inc., University of Colorado

Growing significant numbers of human stem cells in a short time could lead to new treatments for stroke and other diseases. Scientists are sending stem cells to the International Space Station to test whether these cells proliferate faster in microgravity without suffering any side effects.

Therapeutic uses require hundreds of millions of stem cells and currently no efficient way exists to produce such quantities. Previous research suggests that microgravity could help, and the space station is home to the nation's only national lab in microgravity.

Some types of stem cells grow faster in simulated microgravity, according to Abba Zubair, a researcher at the Mayo Clinic in Jacksonville, Florida. Zubair is principal investigator for the Microgravity Expanded Stem Cells investigation, which is cultivating human stem cells aboard the space station for use in clinical trials back on Earth. He holds a doctor of medicine degree in transfusion medicine and cell therapy and a doctorate of philosophy in tumor immunology.

Human stem cells are cells that have not yet specialized in function and can divide into a spectrum of cell types, rejuvenating and repairing tissue throughout a person's lifetime. Stem cells in every organ of the body, including skin and bones, maintain those organs and repair tissue by dividing and differentiating into specialized cells.

Harvesting a person's stem cells and growing enough of them for use in therapies has proven difficult, though. Researchers have successfully grown mesenchymal stem cells, found in bone marrow, but growing sufficient quantities takes weeks. That could be too late for treatment of some conditions.

"Stem cells are inherently designed to remain at a constant number," Zubair explains. "We need to grow them faster, but without changing their characteristics."

The first phase of the investigation, he adds, is answering the question: "Do stem cells grow faster in space and can we grow them in such a manner that they are safe to use in patients?"

Investigators will examine the space-grown cells in an effort to understand the mechanism behind microgravity's effects on them. The long-term goal is to learn how to mimic those effects and develop a safe and reliable way to produce stem cells in the quantities needed.

The second phase will involve testing clinical application of the cells in patients. Zubair has been studying treatment of stroke patients with lab-grown stem cells and plans to compare those results with use of the space-grown stem cells.

"What is unique about this investigation is that we are not only looking at the biology of the cells and how they grow, but focusing on application, how we can use them to treat patients," he says.

The investigation expands existing knowledge of how microgravity affects stem cell growth and differentiation as well as advances future studies on how to produce large numbers of stem cells for treating stroke and other conditions.

The faster that happens, the better for those who could benefit from stem cell therapies.

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Stem cells seem speedier in space - Phys.Org

I recently donated peripheral blood stem cells (PBSC) to a middle-aged man with myelodysplastic syndrome. This is similar medically to a bone marrow donation (though less painful) and much more involved than a blood donation (which I have done regularly since I was seventeen). I found the whole process fascinating and a testament to the glory of mans mind and modern civilization.

The patient with myelodysplastic syndrome lives in central Europe. His bodys bone marrow was no longer producing healthy functioning blood cellsthat is, red blood cells, white blood cells, and plateletsa deficiency that could have resulted in his bodys loss of ability to fight infections or control bleeding, and possibly leukemia. The cure for his disease involved destroying his defective natural bone marrow and replacing it with someone elsesmine.

Bone marrow compatibility between donor and recipient is more complicated than blood types. He needed a donor whose human leukocyte antigen (HLA) proteins most closely matched his own in order to minimize the chance of graft-versus-host disease. His doctors found my data in the Be the Match Registry, where Im registered as a potential donor, and they judged my HLA proteins to be his best hope.

Eight weeks before the operation would take place, I was notified by phone about the match and the donation process. I was then asked whether I was willing to donate. I said yes (and was given several opportunities later to change my mind). In the following weeks, I provided two sets of blood samples to verify that I was healthy enough to donate and still a good match. I was flown out to the donation facility in Michigan to be examined physically, preview the process, and speak with the doctors and nurses who would collect the donation. My donor representative called me periodically to keep me informed and to verify my continuing consent. She also made the arrangements for collecting the samples, managed my travels, and ensured that my expenses were covered.

Here I am holdingthe final product just prior to its transportation to the recipientin central Europe.

As the donation date drew closer, I received ten shots of Filgrastim, a drug designed to stimulate additional blood stem cell formation, one shot in each arm for five days. This increased my white blood cell count far above normal and forced extra blood stem cells into my bloodstream, thus enabling the technicians to run my blood through an apheresis machine, which separated the phases of blood by density using centripetal acceleration. On the donation day, I sat in a comfy chair with an IV in each arm for four hours as a machine took blood from one arm, separated out the stem cells, and returned the rest of my blood via my other arm. While the process continued throughout the morning, the nurses took a few notes here and there, and, as my arms couldnt bend, fed me lunch (chicken wings from Jets Pizza). Once the machine had collected enough stem cells for the recipient (Im fifteen pounds heavier than he is, so it was easier on my body than it could have been), the IVs were removed, my blood was tested one final time to make sure I was OK to drive home, and I left.

My blood stem cells were then transported by private courier to the patient in central Europe. In preparation for the donation, his entire immune system and blood-producing machinery (bone marrow) had been destroyed using myeloablative chemotherapy in order to eliminate any remaining diseased cells and to suppress any immune response from his body to my replacement tissue. My blood stem cells were injected into his bloodstream by IV and then migrated to his bones to replace his destroyed bone marrow and eventually start producing new red blood cells, white blood cells, and platelets. Essentially, my blood and my immune system are regrowing in his body. With these, he inherits my allergies and infectious disease history, and, if all goes well, my life force for another few decades.

Although the organization through which I donated does not pay for stem cells (because payment is against international registry standards), I was treated well and fully reimbursed for expenses. They paid for flights, a half dozen meals, a private driver at one point, hundreds of miles of my own driving, and my stays at nice hotels.

It is worth noting that the Institute for Justice (IJ) recently sued the U.S. attorney general to legalize bone marrow and stem cell donor compensation.1 As the IJ reports, the Ninth Circuit ruled in our favor, holding that the National Organ Transplant Acts ban on donor compensation does not apply to the most common method for donating marrow. This victory is especially helpful for certain minorities and people with multiracial ancestries who face significantly reduced odds of finding unrelated marrow donors. But direct compensation has been met with strong resistance by the major national and international marrow registry organizations, which also lobbied against IJs efforts in court.2 Currently, compensation for donations is being offered only by smaller organizations.

My motivation for donating cannot be reduced to just one reason, but it certainly was not a sacrifice. My reasons varied in depth and weight, but all were self-interested. I thought the process itself was fascinating. I was able to ask the doctors and nurses unlimited questions and to experience firsthand a medical procedure about which I had no previous knowledge. I enjoyed business-class travel, which, as a college student was a significant treat. Most broadly, I participated in an important aspect of the kind of civil society in which I want to live. I want someone to be willing to donate lifesaving tissue to me or my loved ones, should we need it in the future, and I was happy to donate first. The costs were trivialabout twenty-five hours of volunteered time and some minor discomfort. Overall, the experience was positive and spiritually rewarding.

The option to make a donation of this kind did not even exist a few decades ago. It is a function of many interrelated parts of todays modern, relatively free-market, science-oriented cultures. The establishment and maintenance of an international donor registry requires stable, relatively rights-protecting legal systems that enable long-range and large-scale planning among cooperative strangers. To find matches in a timely manner requires the speed and integrating capacities of computers and the Internet. The medical procedure itself requires the kinds of scientific knowledge and expensive technologies made possible by todays relatively free markets. The ability to pay for such a procedure requires substantial personal wealth, which more people have today than ever before. I am exceedingly grateful to live in our rich, science-oriented, relatively capitalist civilization at the time that I do. And I hope the recipient of my donation is able to enjoy many years more of living and loving life as I do.

Related:

Endnotes

1. Bone MarrowNOTA Challenge, Institute for Justice, http://ij.org/case/bonemarrow/.

2. Coalition Says PBSC Donor Compensation Poses Health Risks to Patients and Donors, Be the Match, February 2, 2012, https://bethematch.org/news/news-releases/coalition-says-pbsc-donor-compensation-poses-health-risks-to-patients-and-donors/.

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My Non-Sacrificial Donation of Stem Cells to Save a Life - The Objective Standard

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

Kyodo News/Contributor/getty images

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

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

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

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

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

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

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

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

Clinical work is progressing much more quickly than I expected.

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

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

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

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

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

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

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

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

March 17, 2017 Left: Cross-sectional view of the cerebrum in normal ferret. Neurons are localized in the cerebral cortex, the surface layer of the cerebrum. Since the surface of the cerebrum has folds (gyri), the layer containing neurons winds on its way. Right: Cross-sectional view of the cerebrum in TD ferret. Clusters of neurons (indicated by arrows) are found deep in the cerebrum, which are not detected in the cerebrum of normal ferret. They are called 'periventricular nodular heterotopia,' PNH. In addition, in the surface layer, a larger number of smaller folds (gyri) are seen than normal (indicated by asterisks). They are called polymicrogryri. Credit: Kanazawa University

Thanatophoric dysplasia (TD) is an intractable disease causing abnormalities of bones and the brain. In a recent study of ferrets, which have brains similar to those of humans, researchers using a newly developed technique discovered that neuronal translocation along radial glial fibers to the cerebral cortex during fetal brain development is aberrant, suggesting the cause underlying TD.

In TD cases, the limb and rib bones are shorter than normal, and brain abnormalities manifest, including polymicrogyria and periventricular nodular heterotopia. Previous research has determined that a gene, fibroblast growth factor receptor 3 (FGFR3), is responsible. However, as a result of TD rarity and the difficulty of obtaining brain samples from human patients, the pathophysiology of TD is largely unknown, and effective therapy has not been established.

The present research team of Kanazawa University generated an animal model of TD using ferrets that reproduces the brain abnormalities found in human TD patients. By using this animal model, the team elucidated the formation process of polymicrogyria, one of the abnormalities found in the TD brain. The team has also investigated the formation process of PNH, the other brain abnormality found in human TD patients.

First, PNH was analyzed in terms of composing cell types to reveal that a large number of neurons but few glial cell exist in PNH. In a healthy brain, neurons are found in the cerebral cortex near the brain surface. The researchers believe that during fetal brain development, PNH formation might be induced by the inability of neurons to translocate themselves to the cerebral cortex. The researchers found that the spatial arrangement of radial glial cells was distorted; radial glial fibers are believed to serve as the "track" for neurons to translocate themselves. Thus, the distortion of radial glial fibers seems to be a reason for aberrant localization of neurons.

Research on abnormalities of bones in TD is progressing with iPS cells at Kyoto University, and it is expected that the whole aspect of TD with brain and bone abnormalities would be elucidated and that the therapeutic methods would be developed. The present study on PNH was only possible using the experimental technique for ferrets developed by the research team. This animal model technique could also contribute to studies of other neurological diseases that have been difficult to investigate with conventional model animals.

Explore further: Researchers discover a gene's key role in building the developing brain's scaffolding

More information: Naoyuki Matsumoto et al, Pathophysiological analyses of periventricular nodular heterotopia using gyrencephalic mammals, Human Molecular Genetics (2017). DOI: 10.1093/hmg/ddx038

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Researchers develop new animal model to study rare brain disease - Medical Xpress

By Isaac GuererroStaff writer

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

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

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

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

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

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

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

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

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

BRUSSELS Belgian biotech group Tigenix said on Monday its medical trial with a novel treatment for patients at risk of heart failure after a coronary attack was successful.

The group said patients treated in its PhaseI/II trial of donor-derived expanded cardiac stem cells (AlloCSC) showed no side-effects and all of them continued to live after 30 days, six months and a year.

Tigenix added that in one subgroup of trial patients associated with a poor long-term outlook, there was a larger reduction in the size of infarction, tissue death due to inadequate blood supply.

"This is the first trial in which it has been demonstrated that allogeneic cardiac stem cells can be transplanted safely through the coronary tree," one of the doctors in the trial said.

The group said it would now analyze the data from the trial and decide on how to proceed with its research.

(Reporting by Robert-Jan Bartunek; editing by Philip Blenkinsop)

LONDON A French biotech firm is hoping to gain approval within months for a nuclear medicine targeting the type of cancer that killed Steve Jobs.

NEW YORK Developers of an experimental blood test for autism say it can detect the condition in more than 96 percent of cases and do so across a broad spectrum of patients, potentially allowing for earlier diagnosis, according to a study released on Thursday.

(Reuters Health) - Getting too little sleep in early childhood is linked to cognitive and behavioral problems years later, a U.S. study suggests.

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Belgium's Tigenix says heart attack stem cell trial successful - Reuters

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

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

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

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

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

About Longeveron

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

Contact: Suzanne Liv Page spage@longeveron.com 305.342.9590

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

SOURCE Longeveron

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

Runner's World

Stem Cell Therapy Runner's World This is why researchers and physicians think this therapy may help joint injuries caused by worn-out cartilage; in cell cultures, stem cells can grow new cartilage, and if this can happen in a joint, it may prevent the need for a joint replacement ... Nutrients Boost Stem Cell FunctionProHealth all 19 news articles »

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Stem Cell Therapy - Runner's World

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

Journal reference: Stem Cells

Provided by: University at Buffalo

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The Scientist

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