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Category Archives: Cell Medicine

Cell Medica Acquires Catapult Therapy for Lead WT1-TCR T-Cell Therapy – Genetic Engineering & Biotechnology News (blog)

Posted: June 20, 2017 at 6:45 pm

Cell Medica is buying Catapult Therapy TCR and the firm's gene-modified WT1-TCR (Wilms' tumor 1 proteinT-cell receptor) T-cell therapy candidate. The treatment is currently in Phase I/II development for the potential treatment of acute myeloid leukemia (AML) and myelodysplastic syndrome (MDS).

Catapult Therapy TCR is a special purpose vehicle established by The Cell and Gene Therapy Catapult (CGT Catapult), UCL Business (UCLB), and Imperial lnnovations to develop the WT1-TCR T-cell therapy, which was originally discovered by researchers at University College London (UCL) and Imperial College London. Financial details of the acquisition were not disclosed.

Cell Medical says it plans to apply its Dominant TCR platform to generate a more effective WT1-TCR product that could also feasibly be used to treat challenging solid tumors, including mesothelioma and ovarian cancer. The firm acquired the Dominant TCR technology from UCLB in 2016.

London, U.K.-based Cell Medica and CGT Catapult will carry out further development of the next generation of T cells, and manufacturing process, at the latters recently built large-scale cell and gene therapy manufacturing center at the Stevenage BioScience Catalyst, U.K.A Phase I/II study with the enhanced Dominant WT1-TCR candidate is projected to start during late 2018.

The acquisition of the WT1-TCR cell therapy leverages the investment we made in 2016 for exclusive rights to the Dominant TCR technology, said Gregg Sando, CEO of Cell Medica. Our objective is to show how we can enhance any existing TCR cell therapy with the Dominant TCR technology to create a more effective treatment for patients with solid tumors who otherwise have a very poor prognosis. We are also looking forward to an important collaboration with CGT Catapult to initiate manufacturing at the Stevenage GMP facility, where we will work together on scale-up strategies for commercial production.

With support from Innovate UK, CGT Catapult operates as a Centre of Excellence for Innovation to help drive growth of the U.K.s cell and gene therapy industry and translate early-stage research into new therapies. "We are pleased that Cell Medica has acquired the WT1 T-cell immunotherapy," added Keith Thompson, CEO at CGT Catapult. "With their complementary technologies, they will take over the development of this exciting new therapy. The next-generation product developed in our manufacturing center underlines our ability to support the localization of cell manufacturing processes in the U.K.

Cell Medica is exploiting its proprietary activated T-cell chimeric antigen receptor (CAR) and engineered TCR platforms to develop cellular immunotherapies targeting cancer. Lead product CMD-003 (baltaleucel-T) is being evaluated in the Phase II CITADEL study as a treatment for advanced lymphomas associated with the oncogenic Epstein-Barr virus.In March, Cell Medica raised 60 million (approximately $76 million) in a Series C investment roundto support development of its pipeline.

The firm has an ongoing CAR development partnership with Baylor College of Medicine and is working with UCL to leverage the Dominant TCR technology. Cell Medicas acquisition of Delenex Therapeutics in mid-2016 gave the firm an antibody fragment platform for use in developing anticancer CAR-NKT (natural killer T cells) products, and additional immune cell engineering expertise.

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3D Cell Culture Tech Advances Medical Research and Treatments – GlobeNewswire (press release)

Posted: June 20, 2017 at 6:45 pm

June 20, 2017 05:00 ET | Source: BCC Research

Wellesley, Mass., June 20, 2017 (GLOBE NEWSWIRE) --

Fast-moving developments in 3D cell culture tools and technology are accelerating cancer research and clinical applications, along with other medical research and safety applications. A new report by BCC Research forecasts that some segments could see CAGR as high as 44% in products for pharmaceuticals development through 2021. In another example, neurological safety testing could grow from a $5 million segment in 2015 to $95 million by 2021, according to 3D Cell Cultures: Technologies and Global Markets.

New cell culture products and applications are proliferating even faster than a prior BCC Research report predicted in 2015. Companies such as ThermoFisher, GEHealthcare, MerckMillipore and a range of start-ups and spinouts are pursuing diverse market segments, from cosmetics and skin care to cardiac toxicology and metabolic reactions to new drugs. The new 3D technology allows for groundbreaking visibility into tissue and cancer behaviors in the body, compared with animal testing or more general, 2D in vitro technologies.

Bioreactors with microcarriers are another 3D application seeing rapid market growth. Skin and artificial skin substitutes have been in use for years. Now some of the knowledge behind those advancements are being applied to internal medicine from liver function and metabolic disease and other adjacent fields. These new applications promise not only improved medical safety in determining dosages and tailoring treatments to a patients condition and situation, but also breakthroughs in basic research, drug discovery and development.

Research Highlights

"The standards and best practices emerging in precision cancer care, and the new findings in CNS research could certainly speed up patient-centric care. Some call it personalized medicine, or precision medicine, and it really is a revolution compared with developing mass-market drugs. We are already seeing stem cells and other tools having an impact thanks to 3D Cell Culture technology, says Robert G. Hunter, senior healthcare editor at BCC Research.

About BCC Research

BCC Research is a publisher of market research reports that provide organizations with intelligence to drive smart business decisions. By partnering with industry experts worldwide, BCC Research provides unbiased measurements and assessments of global markets covering major industrial and technology sectors, including emerging markets. For more information, please visit bccresearch.com. Follow BCC Research on Twitter at @BCCResearch.

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3D Cell Culture Tech Advances Medical Research and Treatments - GlobeNewswire (press release)

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Low-cost biochip isolates cells for clinical diagnosis [PreClinical] – 2 Minute Medicine

Posted: June 18, 2017 at 8:42 pm

1. Researchers manufactured a microfluidic biochip, using an inkjet printing system to apply conductive nanoparticles to a polymer substrate.

2. The biochip used an electric field to separate and capture cell populations, allowing for downstream characterization and further analysis.

Evidence Rating Level: 2 (Good)

Study Rundown: Some infectious diseases and cancers can be detected early through the identification of rare cell populations. The sorting and identification of these cells currently require bulky and expensive equipment, preventing their effective use in areas unable to obtain these technologies. The goal of this study was to develop a low-cost, reusable, and effective cell separation platform that could be used for diagnostics in resource-limited areas.

The biochip was designed to have three components: a reusable polyethylene terephthalate substrate with inkjet-printed conductive nanoparticles, a disposable microfluidic platform made of polydimethylsiloxane, and an insulating barrier. The biochip induced dipole moments, resulting in a dielectrophoresis (DEP) force that moved varying cell types based on differences in size and dielectric properties. This allowed for the effective separation and storage of different cell populations. A mixture of breast adenocarcinoma cells, yeast cells, and polystyrene microspheres was used to test the biochip. The three particle types were collected with high separation efficiency. The cells were viable after being on the biochip, indicating they could be used for downstream analyses.

Future work must evaluate the biochip using more clinically relevant cell mixtures. However, the biochip demonstrates potential for diagnostics and research studies on rare cell populations. Without the need for clean rooms or time consuming processing, manufacturing the biochip only requires vector-drawing software and inkjet printing technology. With a production time of around 20 minutes and a materials cost of $0.01 per chip, this biochip could be feasibly produced in developing and low-income areas. Not only can clinical samples be reused after processing on the biochip, but the chip itself is also a reusable platform. This technology could enable faster diagnostic capabilities and early detection of rapidly progressing conditions.

Click here to read the study in PNAS

Relevant Reading: A microfluidic biochip for complete blood cell counts at the point-of-care

In-Depth [in vitro study]: To collect single cells, the biochip used contactless dielectrophorectic-traps and an array of facing electrodes. When particles were introduced to the chip, each cell experienced negative DEP forces that trapped it in a chamber within the electrical field. Once a cell was trapped in a chamber, no other cells could enter.

To optimize the parameters of the biochip, polystyrene microspheres were put in the chip and subjected to various voltages and signal frequencies to determine their effects on the resulting velocity and DEP force. Flow rates were adjusted to optimize the capture efficiency, with higher flow rates resulting in a 510% drop in the efficiency of capturing the microspheres. These parameters were then validated using a breast adenocarcinoma cell line (MDA-MD-231) and yeast cells.

Cells were isolated from the chip and assessed for viability. A 1.5-fold increase in transformation efficiency was noted in the yeast cells, confirming the safety of this technology. A mixture of MDA-MD-231 cells, yeast cells, and streptavidin-coated polystyrene microspheres was put into the biochip. Because each of these particles have different polarization properties, they could be separated by the biochip. Separation efficiency was found to be 79, 88, and 86% for the breast cancer cells, yeast cells, and microspheres, respectively.

Image: PD

2017 2 Minute Medicine, Inc. All rights reserved. No works may be reproduced without expressed written consent from 2 Minute Medicine, Inc. Inquire about licensing here. No article should be construed as medical advice and is not intended as such by the authors or by 2 Minute Medicine, Inc.

2 Minute Medicines The Classics in Medicine: Summaries of the Landmark Trials is available now in paperback and e-book editions.

This text summarizes the key trials in:General Medicine and Chronic Disease, Cardiology, Critical and Emergent Care, Endocrinology, Gastroenterology, Hematology and Oncology, Imaging, Infectious Disease, Nephrology, Neurology, Pediatrics, Psychiatry, Pulmonology, and Surgery.

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Texas has sanctioned unapproved stem cell therapies. Will it change anything? – Science Magazine

Posted: June 17, 2017 at 9:42 am

Texas Governor Greg Abbott just signed a law making it easier for unproven stem cell therapies to be given to patients in his state.

Marjorie Kamys Cotera/Bob Daemmrich Photography/Alamy Stock Photo

By Kelly ServickJun. 15, 2017 , 11:15 AM

Texas Governor Greg Abbott yesterday signed a bill allowing clinics and companies in the state to offer people unproven stem cell interventions without the testing and approval required under federal law. Like the right to try laws that have sprung up in more than 30 states, the measure is meant to give desperately ill patients access to experimental treatments without oversight from the U.S. Food and Drug Administration (FDA).

In a state where unproven stem cell therapies are already offered widely with little legal backlash, bioethicists and patient advocates wonder whether the states official blessing will maintain the status quo, tighten certain protections for patients, or simply embolden clinics already profiting from potentially risky therapies.

You could make the argument thatif [the new law] was vigorously enforcedits going to put some constraints in place, says Leigh Turner, a bioethicist at the University of Minnesota in Minneapolis, who last year co-authored a study documenting U.S. stem cell clinics marketing directly to consumers online, 71 of which were based in Texas. But it would really be surprising if anybody in Texas is going to wander around the state making sure that businesses are complying with these standards, he adds. Either way, Turner says theres powerful symbolic value in setting up this conflict between state law and federal law.

The law, effective 1 September, will allow people with severe chronic or terminal illness to be treated at a clinic that purports to isolate therapeutic stem cells from adult tissuesuch as a patients own fatif their doctor recommends it after considering all other options, and if its administered by a physician at a hospital or medical school with oversight from an institutional review board (IRB). It also requires that the same intervention already be tested on humans in a clinical trial. The law sanctions a much broader set of therapies than federal rules, which already exempt certain stem cell interventions from FDAs lengthy approval process, provided the cells are only minimally manipulated and perform the same function they normally have in body.

The Texas bills clinical trial and IRB requirements seem to weed out some dubious therapies, but the language is too nebulous to protect patients, says Beth Roxland, a bioethicist at New York Universitys Langone Medical Center in New York City. The bill doesnt specify that a trial be conducted in the United States or that the therapy get clearance from FDA for human testing. You could gain access to something [as long as its] being studied in a human somewhere on the planet, she says, which in the stem cell area makes it really very scary.

Awareness about the risks of unproven stem cell therapies is growing. A case report published in The New England Journal of Medicine earlier this year documented three women who lost their vision after receiving purported stem cell injections meant to treat age-related degeneration of the retina. Such risks are also the subject of a news conference today at the annual meeting of the International Society for Stem Cell Research in Boston.

Roxland is also unnerved by a provision in the Texas law that would prevent any state government entity from interfering with a patients access to treatment. Hypothetically, if a state officially gets wind of nefarious doings at a for-profit clinic the state officials are now restrained from doing anything. She notes that that language mirrors a proposal in a federal bill known as the Trickett Wendler Right to Try Act, introduced in the Senate in January, which would prevent the federal government from interfering with a terminally ill patients access to an experimental drug outside of a clinical trial, and would prevent FDA from considering those patients outcomes in its drug approval decisions. Vice President Mike Pence signaled his support for the law in February and met with the family of Trickett Wendler, who advocated for right to try laws before her death from amyotrophic lateral sclerosis in 2015.

Others also believe that the Texas laws approval might signal a coming thaw in federal regulation of stem cell clinics. The FDA obviously doesnt have the manpower to watch over these people, says David Bales, chairman of the advocacy group Texans for Cures in Austin, which pushed for more patient protections in the new bill. We really feel like theyre trying to open up the floodgates.

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Pierre Coulombe, Ph.D. to lead UM Department of Cell & Developmental Biology – University of Michigan Health System News (press release)

Posted: June 17, 2017 at 9:42 am

ANN ARBOR, MI One of the oldest departments at the University of Michigan is about to get a new leader. The U-M Board of Regents today approved the appointment of Pierre A. Coulombe, Ph.D., to lead the Department of Cell and Developmental Biology in the Medical School.

Coulombe will become chair on August 1, and lead one of the nine basic science departments of Michigan Medicine, U-Ms academic medical center. The departments researchers study how structure governs function in cells and tissues throughout the body, and how complex arrays of signals are integrated to foster the proper development of tissues and organs. They also study stem cells, including embryonic stem cells, and train undergraduate, graduate and medical students in cell biology.

The department traces its roots back to 1854, soon after the founding of the Medical School, when it was known as the Department of Anatomy.

Coulombe comes to Michigan from Johns Hopkins University, where he chaired the Department of Biochemistry and Molecular Biology in the Bloomberg School of Public Health for nine years, and held the E.V. McCollum professorship as well as several joint appointments in the School of Medicine. At Hopkins, Coulombe was noted for recruiting and nurturing junior faculty members to success, and developing robust training programs for graduate students and post-doctoral fellows. He was also instrumental in addressing the departments infrastructure needs.

To me, cell and developmental biology are critically important endeavors as one seeks to translate the wealth of knowledge acquired in biochemistry and molecular biology, along with the power of imaging techniques, into a better understanding of how organs and tissues form, and operate, under normal and disease conditions, he says. This knowledge is also important for developing novel therapies for human disease. U-M already is a formidable institution, and otherwise is making a substantial investment into biomedical research. Therefore, I am absolutely thrilled about the opportunity to lead Cell & Developmental Biology, and team up with my new colleagues in the department and at U-M, to fulfill this potential.

In addition to his appointment in Cell & Developmental Biology, Coulombe will have a joint appointment in the U-M Department of Dermatology. His research focuses on understanding how keratin proteins and the nanoscale filaments they form foster an optimal architecture and function in skin and related epithelia, and how disruption of these processes result in diseases ranging from inherited conditions to cancer.

A native of Montral, Qubec, Coulombe earned his undergraduate degree from the Universit du Qubec Montral and his Ph.D. in Pharmacology from Universit de Montral. He completed his postdoctoral fellowship in the Department of Molecular Genetics and Cell Biology & Howard Hughes Medical Institute at the University of Chicago before joining Johns Hopkins School of Medicine in 1992. He is the author of more than 140 peer-reviewed publications and one book, holds one patent, and has received multiple awards in recognition of his research and teaching endeavors.

For more about the U-M Department of Cell and Developmental Biology, visit https://medicine.umich.edu/dept/cell-developmental-biology.

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Induced Cell Turnover: A proposed modality for in situ tissue regeneration & repair – Medical Xpress

Posted: June 15, 2017 at 9:41 pm

June 15, 2017 Credit: Biogerontology Research Foundation, Feinberg School of Medicine & Swammerdam Institute for Life Sciences

Thursday, July 15, 2017, London, UK: Scientists at the Biogerontology Research Foundation, Feinberg School of Medicine at Northwestern University and Swammerdam Institute of Life Sciences at the University of Amsterdam have published a paper on a proposed method of in situ tissue regeneration called Induced Cell Turnover (ICT) in the journal Human Gene Therapy. The proposed therapeutic modality would aim to coordinate the targeted ablation of endogenous cells with the administration of minimally-differentiated, hPSC-derived cells in a gradual and multi-phasic manner so as to extrinsically mediate the turnover and replacement of whole tissues and organs with stem-cell derived cells.

"One of the major hurdles limiting traditional cell therapies is low levels of engraftment and retention, which is caused in part by cells only being able to engraft at locations of existing cell loss, and by the fact that many of those vacancies have already become occupied by ECM and fibroblasts (i.e. scar tissue) by the time the cells are administered, long after the actual occurrence of cell loss. The crux underlying ICT is to coordinate endogenous cell ablation (i.e. induced apoptosis) with replacement cell administration so as to manually vacate niches for new cells to engraft, coordinating these two events in space and time so as to minimize the ability for sites of cell loss to become occupied by ECM and fibroblasts. This would be done in a gradual and multi-phasic manner so as to avoid acute tissue failure resulting from the transient absence of too many cells at any one time. While the notion of endogenous cell clearance prior to replacement cell administration has become routine for bone marrow transplants, it isn't really on the horizon of researchers and clinicians working with solid tissues, and this is something we'd like to change." said Franco Cortese, Deputy Director and Trustee of the Biogerontology Research Foundation, and lead author on the paper.

Cell-type and tissue-specific rates of induced turnover could be achieved using cell-type specific pro-apoptotic small molecule cocktails, peptide mimetics, and/or tissue-tropic AAV-delivered suicide genes driven by cell-type specific promoters. Because these sites of ablation would still be "fresh" when replacement cells are administered, the presumption is that the patterns of ablation will make administered cells more likely to engraft where they should, in freshly vacated niches where the signals promoting cell migration and engraftment are still active. By varying the dose of cell-type targeted ablative agents, cell type and tissue-specific rates of induced turnover could be achieved, allowing for the rate and spatial distribution of turnover to be tuned to the size of the tissue in order to avoid ablating too many cells at once and inadvertently inducing acute tissue failure.

"Cell therapies are limited by low levels of engraftment, and in principal their ability to improve clinical outcomes is limited by the fact that they can only engraft at locations of existing cell loss. Conversely, therapeutic tissue and organ engineering requires surgery, is more likely to introduce biochemical and mechanical abnormalities to tissue ultrastructure through the decellularization process, and is fundamentally incapable of replacing distributed tissues and structures with a high degree of interconnectivity to other tissues in the body. The aim of ICT is to form a bridge between these two main pillars to regenerative medicine, extending the efficacy of cell therapies beyond a patch for existing cell loss and accomplishing the aim of tissue and organ engineering (i.e. the replacement and regeneration of whole tissues and organs) while potentially remaining free of some of their present limitations." said Giovanni Santostasi, co-author on the paper and a researcher at the Feinberg School of Medicine, Northwestern University.

While future iterations of the therapy could use patient-derived cells, such as ESCs derived via somatic cell nuclear transfer (SCNT) or iPSCs derived from nuclear reprogramming, shorter-term applications would likely use existing stem cell lines immunologically matched to the patient via HLA matching. The authors contend that the cloning of adult organisms with normal lifespans from adult somatic cells testifies to the fact that adult cells can be rejuvenated and used to produce a sufficient quantity of daughter cells to replace the sum of cells constituting adult organisms, and that serial cloning experiments (in which this process is done iteratively, using an adult cell of each subsequent generation to derive the next) attests to this fact even more strongly.

"ICT could theoretically enable the controlled turnover and rejuvenation of aged tissues. The technique is particularly applicable to tissues that are not amenable to growth ex vivo and implantation (as with solid organs)such as the vascular, lymphatic, and nervous systems. The method relies upon targeted ablation of old, damaged and/or senescent cells, coupled with a titrated replacement with patient-derived semi-differentiated stem and progenitor cells. By gradually replacing the old cells with new cells, entire tissues can be replaced in situ. The body naturally turns over tissues, but not all tissues and perhaps not optimally. I am reminded of the quote attributed to Heraclitus: 'No man ever steps in the same river twice, for it's not the same river and he's not the same man.'" said Sebastian Aguiar, a coauthor on the paper and researcher at the Swammerdam Institute of Life Sciences, University of Amsterdam.

"Reversing aging in humans will require a multi-step approach at multiple levels of the organismal organization. In situ targeted ablation of the senescent cells and regeneration will be an important component of comprehensive anti-aging therapies." said Alex Zhavoronkov, Chief Science Officer of the Biogerontology Research Foundation.

The researchers originally proposed ICT in 2016 in the context of biomedical gerontology as a possible means of preventing and/or negating age-related phenotypic deviation for the purposes of healthspan extension, and in this new paper they refine the methodological underpinnings of the approach, take a closer look at potential complications and strategies for their deterrence, and analyze ICT in the context of regenerative medicine as an intervention for a broader range of conditions based on disease or dysfunction at the cellular and intercellular level, with potential utilities absent from traditional cell therapies and tissue/organ engineering, the two main pillars of regenerative medicine. The intervention is still very much conceptual, and any potential utilities over other therapeutic modalities within regenerative medicine would need to be verified via preclinical studies, but their hope is to stimulate further research at this interface between geroscience and regenerative medicine.

More information: Francesco Albert Bosco Cortese et al, Induced Cell Turnover: A novel therapeutic modality for in situ tissue regeneration, Human Gene Therapy (2017). DOI: 10.1089/hum.2016.167

Journal reference: Human Gene Therapy

Provided by: Biogerontology Research Foundation

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Stem Cells and Regenerative Medicine Global Summit | Cell … – Technology Networks

Posted: June 15, 2017 at 9:41 pm

The discovery of stem cell has revolutionized the medical world. Ever since the pioneering work of Canadian scientists, Dr. James Till and Dr. Ernest McCulloch, stem cell research has opened up doors to treatments for seemingly incurable conditions and set the groundwork for regenerative medicine.

To support stem cell translation and to help scientists, researchers, and industry members to stay ahead of this constantly evolving field, Clariden Global is proud to present Stem Cells and Regenerative Medicine Global Summit in Toronto, Canada, from 25th 27th September 2017.

The summit will showcase the latest innovations and breakthroughs in stem cell research and medical applications. You will discover the solutions to technical challenges in stem cell expansion, delivery, and integration, and find out how to address rejection and tumor risks of stem cell therapy. The event presents a premium platform for all participants to discover development progress of stem cell medical treatments.

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Transplants using iPS cells put Riken specialist at forefront of regenerative medicine research – The Japan Times

Posted: June 14, 2017 at 4:47 am

When she entered medicine in the mid-1980s, Masayo Takahashi chose ophthalmology as her specialty, she said, because she wanted to have a family and thought the discipline would spare her from sudden work calls in the middle of the night, helping her best balance work and life.

Three decades later, the 55-year-old mother of two grown-up daughters is at the forefront of the nations even the worlds research into regenerative medicine.

In September 2014, she offered a ray of hope to scores of patients with a severe eye condition when her team at the Riken institutes Center for Developmental Biology in Kobe succeeded in a world-first transplanting of cells made from induced pluripotent stem (iPS) cells into a human body.

The operation, conducted as a clinical study, involved creating a retinal sheet from iPS cells, which were developed by Shinya Yamanaka, a researcher at Kyoto University. His 2006 discovery of iPS cells, which can grow into any kind of tissue in the body, won him a Nobel Prize in 2012.

During the 2014 procedure, the retinal sheet was transplanted into a female patient in her 70s with age-related macular degeneration (AMD), an eye disorder that blurs the central field of vision and can lead to blindness. The research team used iPS cells made from the patients own skin cells.

Takahashi made history again in March when she and her team carried out the worlds first transplant of retina cells created from donor iPS cells stocked at Kyoto University. The time and cost necessary for the procedure has been significantly reduced by using the cells, which are made from super-donors, people with special white blood cell types that arent rejected by the immune systems of receiving patients.

Takahashi was in Tokyo last week to speak at the Foreign Press Center and later with The Japan Times. She recounted the highlights of her 25-year research and the numerous legal and other challenges she has overcome.

Takahashi points to the day she led that first iPS transplant surgery Sept. 12, 2014 as the high point of her career so far. Because she worked so hard leading up to the surgery to confirm the safety of the retinal cells, she said that when the operation was over, she was relieved and slept very well.

It wasnt the same for Yamanaka, who provided the stem cells to Takahashi, she said, chuckling. Yamanaka-sensei couldnt sleep well after the surgery because he didnt know about the safety of the cells very well. I should have convinced him.

Some researchers have expressed concern that iPS cell-derived cells have a higher risk of developing cancer. But Takahashi said she knew from the outset that the type her team was making, retinal pigment epithelium (RPE) cells, are extremely unlikely to cause tumors. RPE cells make up the pigmented layer of tissue that supports the light-sensitive cells of the retina.

People in the world think iPS cells are very dangerous because we modify the genes, she said. The retinal pigment epithelium cell is very safe. We knew it from the beginning because we have never seen a metastatic tumor from this cell. Ophthalmologists know very well that this cell is very safe and very good.

The Osaka native said she learned of and became fascinated by the possibility of using stem cells for eye diseases in the mid-1990s, when she took a year off from clinical practice at Kyoto University and spent a year as a researcher at the Salk Institute in San Diego. She moved to Riken in 2006.

More than 2 years have passed since that first iPS surgery, but the transplanted cells remain intact. According to Takahashi, it was not the goal of the research from the outset to improve the eyesight of the patient, who suffered from a very severe case of AMD. Before the surgery, the patient required injections of drugs into her eyeball every two months, but her visual acuity was declining. After the surgery, her acuity stabilized, and more importantly, she is happy, feeling that her vision has brightened and widened, Takahashi said.

Many challenges remain, however, to advance the technology and make it commercially available. One of the issues is cost, Takahashi said, adding that it will take until around 2019 before the cost of the iPS treatment for AMD will fall below 10 million. The first surgery in 2014 cost about 100 million in total, much of which was spent to maintain the clean room and culture the cells.

Still, Takahashi sees a huge potential for iPS cell therapy in her field and beyond.

Every disease has potential to be treated by iPS cell-derived cells or ES (embryonic stem) cell-derived cells in the future, she said, responding to a question on the chances of iPS cells being used to treat ALS, a rare, degenerative neurological disease for which there is currently no cure.

She said she has learned through her experience that some patients are very happy with small improvements.

For ALS, at first, I thought, its a systemic, whole-body disease, so I didnt know how they can fix it, she said. But a doctor (who specializes in ALS) said, its OK, if one finger moves, its (still) OK. So I realized that some benefit will come from cell therapy.

A Matter of Health is a weekly series on the latest health research, technology or policy issues in Japan. It appears on Thursdays.

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Stem Cells and Regenerative Medicine Global Summit – Technology Networks

Posted: June 13, 2017 at 10:42 am

The discovery of stem cell has revolutionized the medical world. Ever since the pioneering work of Canadian scientists, Dr. James Till and Dr. Ernest McCulloch, stem cell research has opened up doors to treatments for seemingly incurable conditions and set the groundwork for regenerative medicine.

To support stem cell translation and to help scientists, researchers, and industry members to stay ahead of this constantly evolving field, Clariden Global is proud to present Stem Cells and Regenerative Medicine Global Summit in Toronto, Canada, from 25th 27th September 2017.

The summit will showcase the latest innovations and breakthroughs in stem cell research and medical applications. You will discover the solutions to technical challenges in stem cell expansion, delivery, and integration, and find out how to address rejection and tumor risks of stem cell therapy. The event presents a premium platform for all participants to discover development progress of stem cell medical treatments.

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Mitochondria behind blood cell formation – Phys.Org

Posted: June 13, 2017 at 10:42 am

June 13, 2017 Mitochondria are tiny, free-floating organelles inside cells. New Northwestern Medicine research has discovered that they play an important role in hematopoiesis, the bodys process for creating new blood cells. Credit: Northwestern University

New Northwestern Medicine research published in Nature Cell Biology has shown that mitochondria, traditionally known for their role creating energy in cells, also play an important role in hematopoiesis, the body's process for creating new blood cells.

"Historically, mitochondria are viewed as ATPenergyproducing organelles," explained principal investigator Navdeep Chandel, PhD, the David W. Cugell Professor of Medicine in the Division of Pulmonary and Critical Care Medicine. "Previously, my laboratory provided evidence that mitochondria can dictate cell function or fate independent of ATP production. We established the idea that mitochondria are signaling organelles."

In the current study, Chandel's team, including post-doctoral fellow Elena Ans, PhD, and graduate students Sam Weinberg and Lauren Diebold, demonstrated that mitochondria control hematopoietic stem cell fate by preventing the generation of a metabolite called 2-hydroxyglutarate (2HG). The scientists showed that mice with stem cells deficient in mitochondrial function cannot generate blood cells due to elevated levels of 2HG, which causes histone and DNA hyper-methylation.

"This is a great example of two laboratories complementing their expertise to work on a project," said Chandel, also a professor of Cell and Molecular Biology and a member of the Robert H. Lurie Comprehensive Cancer Center of Northwestern University.

Paul Schumacker, PhD, professor of Pediatrics, Cell and Molecular Biology and Medicine, was also a co-author on the paper.

Chandel co-authored an accompanying paper in Nature Cell Biology, led by Jian Xu, PhD, at the University of Texas Southwestern Medical Center, which demonstrated that initiation of erythropoiesis, the production of red blood cells specifically, requires functional mitochondria.

"These two studies collectively support the idea that metabolism dictates stem cell fate, which is a rapidly evolving subject matter," said Chandel, who recently wrote a review in Nature Cell Biology highlighting this idea. "An important implication of this work is that diseases linked to mitochondrial dysfunction like neurodegeneration or normal aging process might be due to elevation in metabolites like 2HG."

Explore further: Novel method enables absolute quantification of mitochondrial metabolites

More information: Elena Ans? et al. The mitochondrial respiratory chain is essential for haematopoietic stem cell function, Nature Cell Biology (2017). DOI: 10.1038/ncb3529

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Mitochondria behind blood cell formation - Phys.Org

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