Stem Cell Clinic

New Technology from Asymmetrex Promises to End the Era of Elusive Adult Tissue Stem Cells

By adminJanuary 8, 2015Stem Cell Medicine

Boston, MA (PRWEB) January 08, 2015

James Sherley, Director of the new biotech start-up Asymmetrex, LLC (previously, the Adult Stem Cell Technology Center, LLC) says that he is looking forward to laboratories around the globe evaluating the companys most recent exciting new stem cell technology, which allows tissue stem cells to be counted for the first time. The new technology is reported online this week in Stem Cell Research.

With only the purchase of two commercially available antibodies, any basic cell biology lab can evaluate the new technology for counting its favorite adult tissue stem cells, which Asymmetrex also refers to as distributed stem cells. Asymmetrex scientists accomplished the essential proof of principle in the report with cultured mouse hair follicle stem cells. They also showed that cells with the specific detection criterion were found in mouse hair follicles themselves in regions known to contain the stem cells. With collaborator Dr. Jennifer Chen, they demonstrated that cells in experimental cultures enriched for human skeletal muscle stem cells had the criterion, too. The technology is predicted to be universally able to count adult tissue stem cells in many different tissue types and different vertebrate species, including most, if not all, human tissues.

To count tissue stem cells, the first antibody needed is one that identifies chromosomes found in all cells about an hour before they divide to become two cells. The second antibody needed is one that identifies a special set of chromosomes that is found specifically in adult tissue stem cells. Asymmetrexs Director Sherley spent the last 16 years defining properties of these unique chromosomes, which are called immortal chromosomes. By evaluating both of these antibodies cell detection patterns simultaneously, adult tissue stem cells can be identified with sufficient specificity to count them with a high degree of confidence.

The new report shows that getting to the new technology was a rather complicated business. The project started with the work of Dr. Minsoo Noh when he was a doctoral graduate student in Dr. Sherleys lab at the Massachusetts Institute of Technology. In his graduate studies, Dr. Noh applied a bioengineering-bioinformatics approach to identifying genes that were highly associated with the unique properties of adult tissue stem cells. To avoid the previously unsolved problem of impure tissue stem cells, Dr. Noh used a family of cells that were engineered to model the unique properties of tissue stem cells. He was successful in identifying a large number of cellular genes whose expression was highly specific for unique tissue stem cell properties.

With Dr. Nohs success, the research team now faced a common bioinformatics pitfall too many genes to know which to study next. Dr. David Winklers group at CSIRO in Australia, co-authors of the report, provided a solution. The new report details how Winklers team applied a newly emerging probabilistic approach to reduce a thousand-plus member gene set down to a single gene for interrogation, the histone H2A variant H2A.Z. Oddly, H2A.Z was reduced during adult tissue stem cell specific functions, which went against the conventional biomarker concept of being increased. Dr. Yang Hoon Huh, then a post-doctoral fellow with the Sherley team, undertook an intent investigation of H2A.Zs tissue stem cell-associated properties despite its non-conformist expression. Due to his persistent studies, H2A.Z emerged as the key target of the second antibody in the new technology.

The ability to identify adult tissue stem cells specifically means that now, for the first time, they can be counted. This long awaited capability will begin a new era of quantitative stem cell biology and stem cell medicine. Sherley predicts that, It will be as if tissue stem cell biology put on glasses for the first time. Previously, tissue stem cell research, existing stem cell medicine (e.g., bone marrow transplantation), and new regenerative medicine developments have operated in a blurry world of not knowing the actual number of the elusive tissue stem cells involved in experiments or transplantation treatments. The ability simply to count the critical cells will have a major impact on the quality and progress of these important applications for continuing advances in medicine and human health.

******************************************************************************************** Asymmetrex, LLC is a Massachusetts life sciences company. Asymmetrexs founder and director, James L. Sherley, M.D., Ph.D. is the foremost authority on the unique properties of adult tissue stem cells. The companys patent portfolio contains biotechnologies that solve the three main technical problems production, quantification, and monitoring that have stood in the way of successful commercialization of human adult tissue stem cells for regenerative medicine and drug development. In addition, the portfolio includes novel technologies for isolating cancer stem cells and producing induced pluripotent stem cells. Currently, Asymmetrex is employing its technological advantages to pursue commercialization of facile methods for monitoring adult tissue stem cell number and function.

Gamida Cell's NiCord gets FDA and EMA orphan drug status

By adminJanuary 8, 2015Uncategorized

Published 07 January 2015

Gamida Cell, a leader in cell therapy technologies and products for transplantation and adaptive immune therapy, announced that orphan drug designation has been granted by The US Department of Health and Human Services, The FDA Office of Orphan Products Development (OOPD) for the investigational medicinal product NiCord for the treatment of acute lymphoblastic leukemia (ALL), acute myeloid leukemia (AML), Hodgkin lymphoma and myelodysplastic syndrome (MDS).

The FDA orphan drug designation coincides with the positive opinion of the European Medicines Agency's (EMA's) Committee for Orphan Medicinal Products (COMP) regarding NiCord as a treatment for AML. Gamida Cell intends to file for NiCord orphan drug status with the EMA for other indications as well.

"Receipt of orphan drug status for NiCord in the US and Europe advances Gamida Cell's commercialization plans a major step further, as both afford significant advantages. We very much appreciate the positive feedback and support of the FDA and EMA and look forward to continuing what has been a very positive dialogue with these important agencies," said Gamida Cell president and CEO Dr. Yael Margolin.

The FDA and EMA grant an orphan drug designation to promote the development of products that demonstrate promise for the treatment of rare diseases or conditions. Orphan drug designation provides for various regulatory and economic benefits, including seven years of market exclusivity in the U.S. and 10 years in the EU.

NiCord is derived from a single cord blood unit which has been expanded in culture and enriched with stem cells using Gamida Cell's proprietary NAM technology.

It is currently being tested in a Phase I/II study as an investigational therapeutic treatment for hematological malignancies such as leukemia and lymphoma. In this study, NiCord is being used as the sole stem cell source.

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Gamida Cell's NiCord gets FDA and EMA orphan drug status

Cord Blood Banking Leader, Cryo-Cell International, Continues to Support the Advancement of Regenerative Medicine

By adminJanuary 7, 2015Uncategorized

Tampa, FL (PRWEB) January 06, 2015

One million Americans experience acute myocardial infarctions, commonly known as a heart attack, each year and of those, approximately 300,000 to 500,000 individuals develop heart failure. A heart attack occurs when blood stops flowing properly to a part of the heart and the heart muscle is injured and can die because it is not receiving enough oxygen.

Cryo-Cell International has agreed to provide the Center with cord blood collections that have previously been donated to Cryo-Cell International by parents and designated for research use to advance regenerative medicine. These cord blood collections will allow the Centers scientists to continue to investigate the mechanisms whereby stem cells can be beneficial in limiting damage from heart attacks. A team at the Center, led by researcher and cardiology specialist, Robert J. Henning, M.D., has demonstrated in research animals that stem cells obtained from human umbilical cord blood can release a large number of biologically active growth factors and anti-inflammatory chemicals that can limit the substantial heart inflammation, cell injury and cell destruction that occurs with acute heart attacks, significantly reducing the effects of heart attacks, even when administered up to 24 hours after the heart attack.

We are making good progress in our studies thanks to the cord blood stem cells contributed by Cryo-Cell International, reports Henning.

Cryo-Cell International and others have demonstrated that human umbilical cord blood stem cells can be preserved for more than 20 years without loss of cell viability or potency. Consequently, parents who have the foresight to use cord blood banking services upon their babys birth can potentially use these cord blood stem cells years later to provide a regenerative treatment for a family member if an acute heart attack occurs. The Centers scientists hope to bring umbilical cord blood stem cell therapy to the treatment of patients who have experienced heart attacks within the next five years.

Heart disease is still the number one leading cause of death in the United States. We feel very fortunate that we can provide a valuable and consistent source of cord blood banked stem cells to the Center for Cardiovascular Research, said David Portnoy, Chairman and Co-CEO of Cryo-Cell International.

About Cryo-Cell International

Founded in 1989, Cryo-Cell International, Inc. is the world's first and most highly accredited private cord blood bank. More than 500,000 parents from 87 countries trust Cryo-Cell International to preserve their family members' stem cells. Cryo-Cell International's mission is to provide clients with state-of-the-art stem cell cryopreservation services and support the advancement of regenerative medicine. Cryo-Cell International operates in a facility that is FDA registered, cGMP-/cGTP-compliant and is licensed in all states requiring licensure. In addition to earning AABB accreditation for cord blood banking, Cryo-Cell International is also the first U.S. (for private use only) cord blood bank to receive FACT accreditation for voluntarily adhering to the most stringent cord blood quality standards set by any internationally recognized, independent accrediting organization. Cryo-Cell International is ISO 9001:2008 certified by BSI, an internationally recognized, quality assessment organization. Cryo-Cell International is a publicly traded company, OTCQB: CCEL. For more information, please visit http://www.Cryo-Cell.com.

About the University of South Florida Center for Cardiovascular Research

The University of South Florida Morsani College of Medicines Cardiovascular Services Research Unit has been in existence for almost 20 years and evaluates pharmacotherapeutic agents and the latest treatment and devices for cardiovascular disease.

Circadian rhythms regulate skin stem cell metabolism and expansion, study finds

By adminJanuary 7, 2015Uncategorized

UC Irvine scientists studying the role of circadian rhythms in skin stem cells found that this clock plays a key role in coordinating daily metabolic cycles and cell division.

Their research, which appears Jan. 6 in Cell Reports, shows for the first time how the body's intrinsic day-night cycles protect and nurture stem cell differentiation. Furthermore, this work offers novel insights into a mechanism whereby an out of synch circadian clock can contribute to accelerated skin aging and cancers.

Bogi Andersen, professor of biological chemistry and medicine, and Enrico Gratton, professor of biomedical engineering, focused their efforts on the epidermis, the outermost protective layer of the skin that is maintained and healed by long-lived stem cells.

While the role of the circadian clock in processes such as sleep, feeding behavior and metabolism linked to feeding and fasting are well known, much less is known about whether the circadian clock also regulates stem cell function.

The researchers used novel two-photon excitation and fluorescence lifetime imaging microscopy in Laboratory of Fluorescence Dynamics in UCI's Department of Biomedical Engineering to make sensitive and quantitative measurements of the metabolic state of single cells within the native microenvironment of living tissue.

They discovered that the circadian clock regulates one form of intermediary metabolism in these stem cells, referred to as oxidative phosphorylation. This type of metabolism creates oxygen radicals that can damage DNA and other components of the cell. In fact, one theory of aging posits that aging is caused by the accumulative damage from metabolism-generated oxygen radicals in stem cells.

The Andersen-Gratton study also revealed that the circadian clock within stem cells shifts the timing of cell division such that the stages of the cell division cycle that are most sensitive to DNA damage are avoided during times of maximum oxidative phosphorylation.

Other studies in animals have linked aging to disruption of circadian rhythms, and Andersen said that accelerated aging could be caused by asynchrony in the metabolism and cell proliferation cycles in stem cells.

"Our studies were conducted in mice, but the greater implication of the work relates to the fact that circadian disruption is very common in modern society, and one consequence of such disruption could be abnormal function of stem cells and accelerated aging," he said.

Andersen adds that it is possible that future studies could advance therapeutic insights from this research.

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Circadian rhythms regulate skin stem cell metabolism and expansion, study finds

Circadian rhythms regulate skin stem cell metabolism and expansion, UCI study finds

By adminJanuary 7, 2015Uncategorized

Body clock protects cells from metabolism-generated oxygen radical damage during division

Irvine, Calif., Jan. 6, 2015 -- UC Irvine scientists studying the role of circadian rhythms in skin stem cells found that this clock plays a key role in coordinating daily metabolic cycles and cell division.

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Circadian rhythms regulate skin stem cell metabolism and expansion, UCI study finds

Gamida Cell treatment granted orphan drug status

By adminJanuary 7, 2015Stem Cell Treatment

Stem cell therapy developer Gamida Cell has been awarded orphan drug status by the US Food and Drug Administration (FDA) and European Medicines Agency (EMA) for leukemia treatment NiCord. The investigational drug treats acute lymphoblastic leukemia (ALL), acute myeloid leukemia (AML), Hodgkin lymphoma and myelodysplastic syndrome (MDS). Gamida Cell intends to file for NiCord orphan drug status with the EMA for other indications as well.

Gamida Cell president and CEO Dr. Yael Margolin said, "Receipt of orphan drug status for NiCord in the US and Europe advances Gamida Cell's commercialization plans a major step further, as both afford significant advantages. We very much appreciate the positive feedback and support of the FDA and EMA and look forward to continuing what has been a very positive dialogue with these important agencies."

NiCord is derived from a single cord blood unit, which has been expanded in culture and enriched with stem cells using Gamida Cell's proprietary NAM technology. It is currently being tested in a Phase I/II study as an investigational therapeutic treatment for hematological malignancies such as leukemia and lymphoma. In this study, NiCord is being used as the sole stem cell source.

Published by Globes [online], Israel business news - http://www.globes-online.com - on January 6, 2015

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Gamida Cell treatment granted orphan drug status

'CRISPR' science: Newer genome editing tool shows promise in engineering human stem cells

By adminJanuary 6, 2015Stem Cell Medicine

Johns Hopkins study could advance use of stem cells for treatment and disease research

A powerful "genome editing" technology known as CRISPR has been used by researchers since 2012 to trim, disrupt, replace or add to sequences of an organism's DNA. Now, scientists at Johns Hopkins Medicine have shown that the system also precisely and efficiently alters human stem cells.

In a recent online report on the work in Molecular Therapy, the Johns Hopkins team says the findings could streamline and speed efforts to modify and tailor human-induced pluripotent stem cells (iPSCs) for use as treatments or in the development of model systems to study diseases and test drugs.

"Stem cell technology is quickly advancing, and we think that the days when we can use iPSCs for human therapy aren't that far away," says Zhaohui Ye, Ph.D., an instructor of medicine at the Johns Hopkins University School of Medicine. "This is one of the first studies to detail the use of CRISPR in human iPSCs, showcasing its potential in these cells."

CRISPR originated from a microbial immune system that contains DNA segments known as clustered regularly interspaced short palindromic repeats. The engineered editing system makes use of an enzyme that nicks together DNA with a piece of small RNA that guides the tool to where researchers want to introduce cuts or other changes in the genome.

Previous research has shown that CRISPR can generate genomic changes or mutations through these interventions far more efficiently than other gene editing techniques, such as TALEN, short for transcription activator-like effector nuclease.

Despite CRISPR's advantages, a recent study suggested that it might also produce a large number of "off-target" effects in human cancer cell lines, specifically modification of genes that researchers didn't mean to change.

To see if this unwanted effect occurred in other human cell types, Ye; Linzhao Cheng, Ph.D., a professor of medicine and oncology in the Johns Hopkins University School of Medicine; and their colleagues pitted CRISPR against TALEN in human iPSCs, adult cells reprogrammed to act like embryonic stem cells. Human iPSCs have already shown enormous promise for treating and studying disease.

The researchers compared the ability of both genome editing systems to either cut out pieces of known genes in iPSCs or cut out a piece of these genes and replace it with another. As model genes, the researchers used JAK2, a gene that when mutated causes a bone marrow disorder known as polycythemia vera; SERPINA1, a gene that when mutated causes alpha1-antitrypsin deficiency, an inherited disorder that may cause lung and liver disease; and AAVS1, a gene that's been recently discovered to be a "safe harbor" in the human genome for inserting foreign genes.

Their comparison found that when simply cutting out portions of genes, the CRISPR system was significantly more efficient than TALEN in all three gene systems, inducing up to 100 times more cuts. However, when using these genome editing tools for replacing portions of the genes, such as the disease-causing mutations in JAK2 and SERPINA1 genes, CRISPR and TALEN showed about the same efficiency in patient-derived iPSCs, the researchers report.

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'CRISPR' science: Newer genome editing tool shows promise in engineering human stem cells

Scientists Develop Pioneering Method to Define Stages of Stem Cell Reprogramming

By adminJanuary 6, 2015Uncategorized

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Newswise In a groundbreaking study that provides scientists with a critical new understanding of stem cell development and its role in disease, UCLA researchers at the Eli and Edythe Broad Center of Regenerative Medicine and Stem Cell Research led by Dr. Kathrin Plath, professor of biological chemistry, have established a first-of-its-kind methodology that defines the unique stages by which specialized cells are reprogrammed into stem cells that resemble those found in the embryo.

The study was published online ahead of print in the journal Cell.

Induced pluripotent stem cells (known as iPSCs) are similar to human embryonic stem cells in that both cell types have the unique ability to self-renew and have the flexibility to become any cell in the human body. iPSC cells, however, are generated by reprogramming skin or blood cells and do not require an embryo.

Reprogramming is a long process (about one to two weeks) and largely inefficient, with typically less than one percent of the primary skin or blood cells successfully completing the journey to becoming an iPSC. The exact stages a cell goes through during the reprogramming process are also not well understood. This knowledge is important, as iPSCs hold great promise in the field of regenerative medicine, as they can provide a single source of patient-specific cells to replace those lost to injury or disease. They can also be used to create novel disease models from which new drugs and therapies can be developed.

This research has broad impact, because by deepening our understanding of cell reprogramming we have the potential to improve disease modeling and the generation of better sources of patient-specific specialized cells suitable for replacement therapy, said Plath. This can ultimately benefit patients with new and better treatments for a wide range of diseases.

Drs. Vincent Pasque and Jason Tchieu, postdoctoral fellows in the lab of Dr. Plath and co-first authors of the study, developed a roadmap of the reprogramming process using detailed time-course analyses. They induced the reprogramming of skin cells into iPSC, then observed and analyzed on a daily basis or every other day the process of transformation at the single-cell level. The data were collected and recorded over a period of up to two weeks.

Plaths team found that the changes that happen in cells during reprogramming occur in a sequential stage-by-stage manner, and that importantly, the stages were the same across all the different reprogramming systems and different cell types analyzed.

The exact stage of reprogramming of any cell can now be determined, said Pasque. This study signals a big change in thinking, because it provides simple and efficient tools for scientists to study stem cell creation in a stage-by-stage manner. Most studies to date ignore the stages of reprogramming, but we can now seek to better understand the entire process on both a macro and micro level.

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Scientists Develop Pioneering Method to Define Stages of Stem Cell Reprogramming

Pioneering method developed to define stages of stem cell reprogramming

By adminJanuary 6, 2015Uncategorized

In a groundbreaking study that provides scientists with a critical new understanding of stem cell development and its role in disease, UCLA researchers at the Eli and Edythe Broad Center of Regenerative Medicine and Stem Cell Research led by Dr. Kathrin Plath, professor of biological chemistry, have established a first-of-its-kind methodology that defines the unique stages by which specialized cells are reprogrammed into stem cells that resemble those found in the embryo.

The study was published online ahead of print in the journal Cell.

"This research has broad impact, because by deepening our understanding of cell reprogramming we have the potential to improve disease modeling and the generation of better sources of patient-specific specialized cells suitable for replacement therapy," said Plath. "This can ultimately benefit patients with new and better treatments for a wide range of diseases.

Plath's team found that the changes that happen in cells during reprogramming occur in a sequential stage-by-stage manner, and that importantly, the stages were the same across all the different reprogramming systems and different cell types analyzed.

"The exact stage of reprogramming of any cell can now be determined," said Pasque. "This study signals a big change in thinking, because it provides simple and efficient tools for scientists to study stem cell creation in a stage-by-stage manner. Most studies to date ignore the stages of reprogramming, but we can now seek to better understand the entire process on both a macro and micro level."

Plath's team further discovered that the stages of reprogramming to iPSC are different from what was expected. They found that it is not simply the reversed sequence of stages of embryo development. Some steps are reversed in the expected order; others do not actually happen in the exact reverse order and resist a change until late during reprogramming to iPSCs.

"This reflects how cells do not like to change from one specialized cell type to another and resist a change in cell identity," said Pasque. "Resistance to reprogramming also helps to explain why reprogramming takes place only in a very small proportion of the starting cells."

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Pioneering method developed to define stages of stem cell reprogramming

Brainstorm Stem-Cell Therapy Continues to Show Treatment Effect in ALS Patients

By adminJanuary 6, 2015Stem Cell Treatment

By: Adam Feuerstein | 01/05/15 - 10:52 AM EST

Once injected, the NurOwn stem cells bathe the damaged neurons of ALS patients with secretions of nerve growth factors. Brainstorm has a home run on its hands if NurOwn can be shown to slow or halt the progressive destruction of neurons, and if that disease-modifying effect translates into improved muscle function for ALS patients. Monday's update comes from a Phase IIa trial in which 14 ALS patientswere followed for the three months without treatment. At month four, each patient wastransplanted with their own personalized NurOwn therapy and then assessed every month for six months. Brainstorm evaluated NurOwn's impact on ALS disease progression using the ALSFRS score, a commonly used assessment of treatment response and muscle function in ALS patients. Lung function, another commonly used measure of efficacy in ALS clinical trials, was also measured.

Twelve ALS patients were evaluable for response. Of these, 11 patientsshowed aslowing of ALS disease progression at six months compared to baseline, measured either by improved ALSFRS or lung function scores, Brainstorm said. Two other patients enrolled in the study died. Administration of the NurOwn therapy was well tolerated by patients, the company said.

The final Phase IIa data announced Monday were a small improvement over interim results from the same study presented last June. Further, detailed data from the study will be presented at a medical meeting later this year. For perspective purposes, it's important to note that this phase IIa study enrolled a relatively small number of ALS patients and was conducted at a single hospital in Israel. This doesn't necessarily discredit the positive results, but conclusions about NurOwn's ultimate benefit as an ALS therapy can't be drawnuntil data from larger studies are gathered.

Brainstorm is conducting another, larger Phase II study in the U.S., enrolling 48 ALS patients who will be randomized 3:1 to receive a single NurOwn treatment in the muscle and spine, or a placebo treatment. The study is being conducted at two hospitals in Massachusetts, UMass Medical Center and Massachusetts General, and the Minnesota-based Mayo Clinic. The study's primary endpoint is the safety and tolerability of NurOwn, but investigators will also assess ALS patients for efficacy using measures of ALS disease activity and muscle function. The first patient was enrolled into the Phase II study last June and Brainstorm expects results to be ready in the first half of 2016.

The company is also in the planning stages for another Phase II study in which ALS patients will be treated with multiple doses of NurOwn. Must Read: 11 Best Small-Cap Technology Stocks That Could Hit It Big in 2015

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Brainstorm Stem-Cell Therapy Continues to Show Treatment Effect in ALS Patients