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iPS Cells for Disease Modeling and Drug Discovery

By Dr. Matthew Watson

Cambridge Healthtech Institutes 4th AnnualJune 19-20, 2019

With advances in reprogramming and differentiation technologies, as well as with the recent availability of gene editing approaches, we are finally able to create more complex and phenotypically accurate cellular models based on pluripotent cell technology. This opens new and exciting opportunities for pluripotent stem cell utilization in early discovery, preclinical and translational research. CNS diseases and disorders are currently the main therapeutic area of application with some impressive success stories resulted in clinical trials. Cambridge Healthtech Institutes 4th Annual iPS Cells for Disease Modeling and Drug Discovery conference is designed to bring together experts and bench scientists working with pluripotent cells and end users of their services, researchers working on finding cures for specific diseases and disorders.

Day 1 | Day 2 | Download Brochure | Speaker Biographies

Wednesday, June 19

12:00 pm Registration Open

12:00 Bridging Luncheon Presentation:Structural Maturation in the Development of hiPSC-Cardiomyocyte Models for Pre-clinical Safety, Efficacy, and Discovery

Nicholas Geissse, PhD, CSO, NanoSurface Biomedical

Alec S.T. Smith, PhD, Acting Instructor, Bioengineering, University of Washington

hiPSC-CM maturation is sensitive to structural cues from the extracellular matrix (ECM). Failure to reproduce these signals in vitro can hamper experimental reproducibility and fidelity. Engineering approaches that address this gap typically trade off complexity with throughput, making them difficult to deploy in the modern drug development paradigm. The NanoSurface Car(ina) platform leverages ECM engineering approaches that are fully compatible with industry-standard instrumentation including HCI- and MEA-based assays, thereby improving their predictive power.

12:30 Transition to Plenary

12:50 PLENARY KEYNOTE SESSION

2:20Booth Crawl and Dessert Break in the Exhibit Hall with Poster Viewing

2:25 Meet the Plenary Keynotes

3:05 Chairpersons Remarks

Gabriele Proetzel, PhD, Director, Neuroscience Drug Discovery, Takeda Pharmaceuticals, Inc.

3:10 KEYNOTE PRESENTATION: iPSC-Based Drug Discovery Platform for Targeting Innate Immune Cell Responses

Christoph Patsch, PhD, Team Lead Stem Cell Assays, Disease Relevant Cell Models and Assays, Chemical Biology, Therapeutic Modalities, Roche Pharma Research and Early Development

The role of innate immune cells in health and disease, respectively their function in maintaining immune homeostasis and triggering inflammation makes them a prime target for therapeutic approaches. In order to explore novel therapeutic strategies to enhance immunoregulatory functions, we developed an iPSC-based cellular drug discovery platform. Here we will highlight the unique opportunities provided by an iPSC-based drug discovery platform for targeting innate immune cells.

3:40 Phenotypic Screening of Induced Pluripotent Stem Cell Derived Cardiomyocytes for Drug Discovery and Toxicity Screening

Arne Bruyneel, PhD, Postdoctoral Fellow, Mark Mercola Lab, Cardiovascular Institute, Stanford University School of Medicine

Cardiac arrhythmia and myopathy is a major problem with cancer therapeutics, including newer small molecule kinase inhibitors, and frequently causes heart failure, morbidity and death. However, currentin vitromodels are unable to predict cardiotoxicity, or are not scalable to aid drug development. However, with recent progress in human stem cell biology, cardiac differentiation protocols, and high throughput screening, new tools are available to overcome this barrier to progress.

4:10 Disease Modeling Using Human iPSC-Derived Telencephalic Inhibitory Interneurons - A Couple of Case Studies

Yishan Sun, PhD, Investigator, Novartis Institutes for BioMedical Research (NIBR)

Human iPSC-derived neurons provide the foundation for phenotypic assays assessing genetic or pharmacological effects in a human neurobiological context. The onus is on assay developers to generate application-relevant neuronal cell types from iPSCs, which is not always straightforward, given the diversity of neuronal classes in the human brain and their developmental trajectories. Here we present two case studies to illustrate the use of iPSC-derived telencephalic GABAergic interneurons in neuropsychiatric research.

4:40 Rethinking the Translational The Use of Highly Predictive hiPSC-Derived Models in Pre-Clinical Drug Development

Stefan Braam, CEO, Ncardia

Current drug development strategies are failing to increase the number of drugs reaching the market. One reason for low success rates is the lack of predictive models. Join our talk to learn how to implement a predictive and translational in vitro disease model, and assays for efficacy screening at any throughput.

5:10 4th of July Celebration in the Exhibit Hall with Poster Viewing

5:30 - 5:45 Speed Networking: Oncology

6:05 Close of Day

5:45 Dinner Short Course Registration

6:15 Dinner Short Course*

*Separate registration required.

Day 1 | Day 2 | Download Brochure | Speaker Biographies

Thursday, June 20

7:15 am Registration

7:15 Breakout Discussion Groups with Continental Breakfast

8:10 Chairpersons Remarks

Jeff Willy, PhD, Research Fellow, Discovery and Investigative Toxicology, Vertex

8:15 Levering iPSC to Understand Mechanism of Toxicity

Jeff Willy, PhD, Research Fellow, Discovery and Investigative Toxicology, Vertex

The discovery of mammalian cardiac progenitor cells suggests that the heart consists of not only terminally differentiated beating cardiomyocytes, but also a population of self-renewing stem cells. We recently showed that iPSC cardiomyocytes can be utilized not only to de-risk compounds with potential for adverse cardiac events, but also to understand underlying mechanisms of cell-specific toxicities following xenobiotic stress, thus preventing differentiation and self-renewal of damaged cells.

8:45Pluripotent Stem Cell-Derived Cardiac and Vascular Progenitor Cells for Tissue Regeneration

Nutan Prasain, PhD, Associate Director, Cardiovascular Programs, Astellas Institute for Regenerative Medicine (AIRM)

This presentation will provide the review on recent discoveries in the derivation and characterization of cardiac and vascular progenitor cells from pluripotent stem cells, and discuss the therapeutic potential of these cells in cardiac and vascular tissue repair and regeneration.

9:15 Use of iPSCDerived Hepatocytes to Identify Treatments for Liver Disease

Stephen A. Duncan, PhD, Smartstate Chair in Regenerative Medicine, Professor and Chairman, Department of Regenerative Medicine and Cell Biology, Medical University of South Carolina

MTDPS3 is a rare disease caused by mutations in the DGUOK gene, which is needed for mitochondrial DNA (mtDNA) replication and repair. Patients commonly die as children from liver failure primarily caused by unmet energy requirements. We modeled the disease using DGOUK deficient iPSC derived hepatocytes and performed a screen to identify drugs that can restore mitochondrial ATP production.

9:45Industrial-Scale Generation of Human iPSC-Derived Hepatocytes for Liver-Disease and Drug Development Studies

Liz Quinn, PhD, Associate Director, Stem Cell Marketing, Marketing, Takara Bio USA

Our optimized hepatocyte differentiation protocol and standardized workflow mimics embryonic development and allows for highly efficient differentiation of hPSCs through definitive endoderm into hepatocytes. We will describe the creation of large panels of industrial-scale hPSC-derived hepatocytes with specific genotypes and phenotypes and their utility for drug metabolism and disease modeling.

10:00 Sponsored Presentation (Opportunity Available)

10:15 Coffee Break in the Exhibit Hall with Poster Viewing

10:45 Poster Winner Announced

11:00 KEYNOTE PRESENTATION: Modeling Human Disease Using Pluripotent Stem Cells

Lorenz Studer, MD, Director, Center for Stem Cell Biology, Memorial Sloan Kettering Cancer Center

One of the most intriguing applications of human pluripotent stem cells is the possibility of recreating a disease in a dish and to use such cell-based models for drug discovery. Our lab uses human iPS and ES cells for modeling both neurodevelopmental and neurodegenerative disorders. I will present new data on our efforts of modeling complex genetic disease using pluripotent stem cells and the development of multiplex culture systems.

11:30 Preclinical Challenges for Gene Therapy Approaches in Neuroscience

Gabriele Proetzel, PhD, Director, Neuroscience Drug Discovery, Takeda Pharmaceuticals, Inc.

Gene therapy has delivered encouraging results in the clinic, and with the first FDA approval for an AAV product is now becoming a reality. This presentation will provide an overview of the most recent advances of gene therapy for the treatment of neurological diseases. The discussion will focus on preclinical considerations for gene therapy including delivery, efficacy, biodistribution, animal models and safety.

12:00 pm Open Science Meets Stem Cells: A New Drug Discovery Approach for Neurodegenerative Disorders

Thomas Durcan, PhD, Assistant Professor, Neurology and Neurosurgery, McGill University

Advances in stem cell technology have provided researchers with tools to generate human neurons and develop first-of-their-kind disease-relevant assays. However, it is imperative that we accelerate discoveries from the bench to the clinic and the Montreal Neurological Institute (MNI) and its partners are piloting an Open Science model. By removing the obstacles in distributing patient samples and assay results, our goal is to accelerate translational medical research.

12:30 Elevating Drug Discovery with Advanced Physiologically Relevant Human iPSC-Based Screening Platforms

Fabian Zanella, PhD, Director, Research and Development, StemoniX

Structurally engineered human induced pluripotent stem cell (hiPSC)-based platforms enable greater physiological relevance, elevating performance in toxicity and discovery studies. StemoniXs hiPSC-derived platforms comprise neural (microBrain) or cardiac (microHeart) cells constructed with appropriate inter- and intracellular organization promoting robust activity and expected responses to known cellular modulators.

1:00Overcoming Challenges in CNS Drug Discovery through Developing Translatable iPSC-derived Cell-Based Assays

Jonathan Davila, PhD, CEO, Co-Founder, NeuCyte Inc.

Using direct reprogramming of iPSCs to generate defined human neural tissue, NeuCyte developed cell-based assays with complex neuronal structure and function readouts for versatile pre-clinical applications. Focusing on electrophysiological measurements, we demonstrate the capability of this approach to identify adverse neuroactive effects, evaluate compound efficacy, and serve phenotypic drug discovery.

1:15Enjoy Lunch on Your Own

1:35 Dessert and Coffee Break in the Exhibit Hall with Poster Viewing

1:45 - 2:00 Speed Networking: Last Chance to Meet with Potential Partners and Collaborators!

2:20 Chairpersons Remarks

Gary Gintant, PhD, Senior Research Fellow, AbbVie

2:25 The Evolving Roles of Evolving Human Stem Cell-Derived Cardiomyocyte Preparations in Cardiac Safety Evaluations

Gary Gintant, PhD, Senior Research Fellow, AbbVie

Human induced pluripotent stem cell-derived cardiomyocytes (hiPSC-CMs) hold great promise for preclinical cardiac safety testing. Recent applications focus on drug effects on cardiac electrophysiology, contractility, and structural toxicities, with further complexity provided by the growing number of hiPSC-CM preparations being developed that may also promote myocyte maturity. The evolving roles (both non-regulatory and regulatory) of these preparations will be reviewed, along with general considerations for their use in cardiac safety evaluations.

2:55 Pharmacogenomic Prediction of Drug-Induced Cardiotoxicity Using hiPSC-Derived Cardiomyocytes

Paul W. Burridge, PhD, Assistant Professor, Department of Pharmacology, Center for Pharmacogenomics, Northwestern University Feinberg School of Medicine

We have demonstrated that human induced pluripotent stem cell-derived cardiomyocytes successfully recapitulate a patients predisposition to chemotherapy-induced cardiotoxicity, confirming that there is a genomic basis for this phenomenon. Here we will discuss our recent work deciphering the pharmacogenomics behind this relationship, allowing the genomic prediction of which patients are likely to experience this side effect. Our efforts to discover new drugs to prevent doxorubicin-induced cardiotoxicity will also be reviewed.

3:25 Exploring the Utility of iPSC-Derived 3D Cortical Spheroids in the Detection of CNS Toxicity

Qin Wang, PhD, Scientist, Drug Safety Research and Evaluation, Takeda

Drug-induced Central Nervous System (CNS) toxicity is a common safety attrition for project failure during discovery and development phases due low concordance rates between animal models and human, absence of clear biomarkers, and a lack of predictive assays. To address the challenge, we validated a high throughput human iPSC-derived 3D microBrain model with a diverse set of pharmaceuticals. We measured drug-induced changes in neuronal viability and Ca channel function. MicroBrain exposure and analyses were rooted in therapeutic exposure to predict clinical drug-induced seizures and/or neurodegeneration. We found that this high throughput model has very low false positive rate in the prediction of drug-induced neurotoxicity.

3:55 Linking Liver-on-a-Chip and Blood-Brain-Barrier-on-a-Chip for Toxicity Assessment

Sophie Lelievre, DVM, PhD, LLM, Professor, Cancer Pharmacology, Purdue University College of Veterinary Medicine

One of the challenges to reproduce the function of tissues in vitro is the maintenance of differentiation. Essential aspects necessary for such endeavor involve good mechanical and chemical mimicry of the microenvironment. I will present examples of the management of the cellular microenvironment for liver and blood-brain-barrier tissue chips and discuss how on-a-chip devices may be linked for the integrated study of the toxicity of drugs and other molecules.

4:25 Close of Conference

Day 1 | Day 2 | Download Brochure | Speaker Biographies

Arrive early to attend Tuesday, June 18 - Wednesday, June 19

Chemical Biology and Target Validation

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iPS Cells for Disease Modeling and Drug Discovery

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CloneR hPSC Cloning Supplement – Stemcell Technologies

By Dr. Matthew Watson

'); jQuery('.cart-remove-box a').on('click', function(){ link = jQuery(this).attr('href'); jQuery.ajax({ url: link, cache: false }); jQuery('.cart-remove-box').remove(); setTimeout(function(){window.location.reload();}, 800); }); }); //jQuery('#ajax_loader').hide(); // clear being added addToCartButton.text(defaultText).removeAttr('disabled').removeClass('disabled'); addToCartButton.parent().find('.disabled-blocker').remove(); loadingDots.remove(); clearInterval(loadingDotId); jQuery('body').append(""); setTimeout(function () {jQuery('.add-to-cart-success').slideUp(500)}, 5000); }); } try { jQuery.ajax( { url : url, dataType : 'json', type : 'post', data : data, complete: function(){ if(jQuery('body').hasClass('product-edit') || jQuery('body').hasClass('wishlist-index-configure')){ jQuery.ajax({ url: "https://www.stemcell.com/meigeeactions/updatecart/", cache: false }).done(function(html){ jQuery('header#header .top-cart').replaceWith(html); }); jQuery('#ajax_loader').hide(); jQuery('body').append(""); setTimeout(function () {jQuery('.add-to-cart-success').slideUp(500)}, 5000); } }, success : function(data) { if(data.status == 'ERROR'){ jQuery('body').append(''); }else{ ajaxComplete(); } } }); } catch (e) { } // End of our new ajax code this.form.action = oldUrl; if (e) { throw e; } } }.bind(productAddToCartForm); productAddToCartForm.submitLight = function(button, url){ if(this.validator) { var nv = Validation.methods; delete Validation.methods['required-entry']; delete Validation.methods['validate-one-required']; delete Validation.methods['validate-one-required-by-name']; if (this.validator.validate()) { if (url) { this.form.action = url; } this.form.submit(); } Object.extend(Validation.methods, nv); } }.bind(productAddToCartForm); function setAjaxData(data,iframe,name,image){ if(data.status == 'ERROR'){ jQuery('body').append(''); }else{ if(data.sidebar && !iframe){ if(jQuery('.top-cart').length){ jQuery('.top-cart').replaceWith(data.sidebar); } if(jQuery('.sidebar .block.block-cart').length){ if(jQuery('#cart-sidebar').length){ jQuery('#cart-sidebar').html(jQuery(data.sidebar).find('#mini-cart')); jQuery('.sidebar .block.block-cart .subtotal').html(jQuery(data.sidebar).find('.subtotal')); }else{ jQuery('.sidebar .block.block-cart p.empty').remove(); content = jQuery('.sidebar .block.block-cart .block-content'); jQuery('').appendTo(content); jQuery('').appendTo(content); content.find('#cart-sidebar').html(jQuery(data.sidebar).find('#mini-cart').html()); content.find('.actions').append(jQuery(data.sidebar).find('.subtotal')); content.find('.actions').append(jQuery(data.sidebar).find('.actions button.button')); } cartProductRemove('#cart-sidebar li.item a.btn-remove', { confirm: 'Are you sure you would like to remove this item from the shopping cart?', submit: 'Ok', calcel: 'Cancel' }); } jQuery.fancybox.close(); jQuery('body').append(''); }else{ jQuery.ajax({ url: "https://www.stemcell.com/meigeeactions/updatecart/", cache: false }).done(function(html){ jQuery('header#header .top-cart').replaceWith(html); jQuery('.top-cart #mini-cart li.item a.btn-remove').on('click', function(event){ event.preventDefault(); jQuery('body').append('Are you sure you would like to remove this item from the shopping cart?OkCancel'); jQuery('.cart-remove-box a').on('click', function(){ link = jQuery(this).attr('href'); jQuery.ajax({ url: link, cache: false }); jQuery('.cart-remove-box').remove(); setTimeout(function(){window.location.reload();}, 800); }); }); jQuery.fancybox.close(); jQuery('body').append(''); }); } } setTimeout(function () {jQuery('.add-to-cart-success').slideUp(500)}, 5000); } // CloneR is a defined, serum-free supplement designed to increase the cloning efficiency and single-cell survival of human embryonic stem cells (ES cells) and induced pluripotent stem cells (iPS cells). CloneR enables the robust generation of clonal cell lines without single-cell adaptation, thus minimizing the risk of acquiring genetic abnormalities.

CloneR is compatible with the TeSR family of media for human ES and iPS cell maintenance as well as your choice of cell culture matrix.

Advantages:

Greatly facilitates the process of genome editing of human ES and iPS cells Compatible with any TeSR maintenance medium and your choice of cell culture matrix Does not require adaptation to single-cell passaging Increases single-cell survival at clonal density across multiple human ES and iPS cell lines

Cell Type:

Pluripotent Stem Cells

Application:

Cell Culture

Area of Interest:

Cell Line Development; Stem Cell Biology; Disease Modeling

Formulation:

Defined; Serum-Free

Document Type

Product Name

Catalog #

Lot #

Language

This product is designed for use in the following research area(s) as part of the highlighted workflow stage(s). Explore these workflows to learn more about the other products we offer to support each research area.

Research Area Workflow Stages for

Workflow Stages

Figure 1. hPSC Single-Cell Cloning Workflow with CloneR

On day 0, human pluripotent stem cells (hPSCs) are seeded as single cells at clonal density (e.g. 25 cells/cm2) or sorted at 1 cell per well in 96-well plates in TeSR (mTeSR1 or TeSR-E8) medium supplemented with CloneR. On day 2, the cells are fed with TeSR medium containing CloneR supplement. From day 4, cells are maintained in TeSR medium without CloneR. Colonies are ready to be picked between days 10 - 14. Clonal cell lines can be maintained long-term in TeSR medium.

Figure 2. CloneR Increases the Cloning Efficiency of hPSCs and is Compatible with Multiple hPSC Lines and Seeding Protocols

TeSR medium supplemented with CloneR increases hPSC cloning efficiency compared with cells plated in TeSR containing ROCK inhibitor. Cells were seeded (A) at clonal density (25 cells/cm2) in mTeSR1 and TeSR-E8 and (B) by single-cell deposition using FACS (seeded at 1 cell/well) in mTeSR1.

Figure 3. CloneR Increases the Cloning Efficiency of hPSCs at Low Seeding Densities

hPSCs plated in mTeSR1 supplemented with CloneR demonstrated significantly increased cloning efficiencies compared to cells plated in mTeSR1 containing ROCK inhibitor (10M Y-27632). Shown are representative images of alkaline phosphatase-stained colonies at day 7 in individual wells of a 12-well plate. H1 human embryonic stem (hES) cells were seeded at clonal density (100 cells/well, 25 cells/cm2) in mTeSR1 supplemented with (A) ROCK inhibitor or (B) CloneR on Vitronectin XF cell culture matrix.

Figure 4. CloneR Yields Larger Single-Cell Derived Colonies

hPSCs seeded in mTeSR1 supplemented with CloneR result in larger colonies than cells seeded in mTeSR1 containing ROCK inhibitor (10M Y-27632). Shown are representative images of hPSC clones established after 7 days of culture in mTeSR1 supplemented with (A) ROCK inhibitor or (B) CloneR.

Figure 5. Clonal Cell Lines Established Using CloneR Display Characteristic hPSC Morphology

Clonal cell lines established using mTeSR1 or TeSR-E8 medium supplemented with CloneR retain the prominent nucleoli and high nuclear-to-cytoplasmic ratio characteristic of hPSCs. Representative images at passage 7 after cloning are shown for clones derived from the parental (A) H1 hES cell and (B) WLS-1C human induced pluripotent stem (iPS) cell lines.

Figure 6. Clonal Cell Lines Established with CloneR Express High Levels of Undifferentiated Cell Markers

hPSC clonal cell lines established using mTeSR1 supplemented with CloneR express comparable levels of undifferentiated cell markers, OCT4 (Catalog #60093) and TRA-1-60 (Catalog #60064), as the parental cell lines. (A) Clonal cell lines established from parental H1 hES cell line. (B) Clonal cell lines established from parental WLS-1C hiPS cell line. Data is presented between passages 5 - 7 after cloning and is shown as mean SEM; n = 2.

Figure 7. Clonal Cell Lines Established Using CloneR Display a Normal Karyotype

Representative karyograms of clones derived from parental (A) H1 hES cell and (B) WLS-1C hiPS cell lines demonstrate that the clonal lines established with CloneR have a normal karyotype. Cells were karyotyped 5 passages after cloning, with an overall passage number of 45 and 39, respectively.

Figure 8. Clonal Cell Lines Established Using CloneR Display Normal Growth Rates

Fold expansion of clonal cell lines display similar growth rates to parental cell lines. Shown are clones (red) and parental cell lines (gray) for (A) H1 hES cell and (B) WLS-1C hiPS cell lines.

STEMCELL TECHNOLOGIES INC.S QUALITY MANAGEMENT SYSTEM IS CERTIFIED TO ISO 13485. PRODUCTS ARE FOR RESEARCH USE ONLY AND NOT INTENDED FOR HUMAN OR ANIMAL DIAGNOSTIC OR THERAPEUTIC USES UNLESS OTHERWISE STATED.

Internal Search Keywords: genome editing | cloning | CRISPR | clone | gene editing | 05888 | 5888 | single cell | accutase

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The addition of human iPS cell-derived neural progenitors …

By Dr. Matthew Watson

JavaScript is disabled on your browser. Please enable JavaScript to use all the features on this page.Highlights

Human iPS cell-derived neural progenitors influence the contractile property of cardiac spheroid.

The contractile function of spheroids depends on the ratio of neural progenitors to cardiac cells.

Neural factors may influence the contractile function of the spheroids.

We havebeen attempting to use cardiac spheroids to construct three-dimensional contractilestructures for failed hearts. Recent studies have reported that neuralprogenitors (NPs) play significant roles in heart regeneration. However, theeffect of NPs on the cardiac spheroid has not yet been elucidated.

This studyaims to demonstrate the influence of NPs on the function of cardiac spheroids.

Thespheroids were constructed on a low-attachment-well plate by mixing humaninduced pluripotent stem (hiPS) cell-derived cardiomyocytes and hiPScell-derived NPs (hiPS-NPs). The ratio of hiPS-NPs was set at 0%, 10%, 20%,30%, and 40% of the total cell number of spheroids, which was 2500. The motionwas recorded, and the fractional shortening and the contraction velocity weremeasured.

Spheroidswere formed within 48 h after mixing the cells, except for the spheroidscontaining 0% hiPS-NPs. Observation at day 7 revealed significant differencesin the fractional shortening (analysis of variance; p=0.01). The bestfractional shortening was observed with the spheroids containing 30% hiPS-NPs.Neuronal cells were detected morphologically within the spheroids under aconfocal microscope.

Theaddition of hiPS-NPs influenced the contractile function of the cardiacspheroids. Further studies are warranted to elucidate the underlying mechanism.

Human iPS cell

Cardiomyocyte

Neural progenitor

Spheroid

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What Are Induced Pluripotent Stem Cells? – Stem Cell: The …

By Dr. Matthew Watson

Today, induced pluripotent stem cells are mostly used to understand how certain diseases occur and how they work. By using IPS cells, one can actually study the cells and tissues affected by the disease without causing unnecessary harm to the patient.For example, its extremely difficult to obtain actual brain cells from a living patient with Parkinsons Disease. This process is even more complicated if you want to study the disease in its early stages before symptoms begin presenting themselves.

Fortunately, with genetic reprogramming, researchers can now achieve this. Scientists can do a skin biopsy of a patient with Parkinsons disease and create IPS cells. These IPS cells can then be converted into neurons, which will have the same genetic make-up as the patients own cells.

Because of IPS cells, researchers can now study conditions like Parkinsons disease to determine what went wrong and why. They can also test out new treatment methods in hopes of protecting the patient against the disease or curing it after diagnosis.

In addition, IPS cells have also been looked to as a way to replace cells that are often destroyed by certain diseases. However, there is still research to be done here.

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What Are Induced Pluripotent Stem Cells? - Stem Cell: The ...

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Stem Cell Key Terms | California’s Stem Cell Agency

By Dr. Matthew Watson

En Espaol

The term stem cell by itself can be misleading. In fact, there are many different types of stem cells, each with very different potential to treat disease.

Stem CellPluripotentEmbryonic Stem CellAdult Stem CelliPS CellCancer Stem Cell

By definition, all stem cells:

Pluripotent means many "potentials". In other words, these cells have the potential of taking on many fates in the body, including all of the more than 200 different cell types. Embryonic stem cells are pluripotent, as are induced pluripotent stem (iPS) cells that are reprogrammed from adult tissues. When scientists talk about pluripotent stem cells, they mostly mean either embryonic or iPS cells.

Embryonic stem cells come from pluripotent cells, which exist only at the earliest stages of embryonic development. In humans, these cells no longer exist after about five days of development.

When isolated from the embryo and grown in a lab dish, pluripotent cells can continue dividing indefinitely. These cells are known as embryonic stem cells.

James Thomson, a professor in the Department of Cell and Regenerative Biology at the University of Wisconsin, derived the first human embryonic stem cell lines in 1998. He now shares a joint appointment at the University of California, Santa Barbara, a CIRM-funded institution.

Adult stem cells are found in the various tissues and organs of the human body. They are thought to exist in most tissues and organs where they are the source of new cells throughout the life of the organism, replacing cells lost to natural turnover or to damage or disease.

Adult stem cells are committed to becoming a cell from their tissue of origin, and cant form other cell types. They are therefore also called tissue-specific stem cells. They have the broad ability to become many of the cell types present in the organ they reside in. For example:

Unlike embryonic stem cells, researchers have not been able to grow adult stem cells indefinitely in the lab, but this is an area of active research.

Scientists have also found stem cells in the placenta and in the umbilical cord of newborn infants, and they can isolate stem cells from different fetal tissues. Although these cells come from an umbilical cord or a fetus, they more closely resemble adult stem cells than embryonic stem cells because they are tissue-specific. The cord blood cells that some people bank after the birth of a child are a form of adult blood-forming stem cells.

CIRM-grantee IrvWeissman of the Stanford University School of Medicine isolated the first blood-forming adult stem cell from bone marrow in 1988 in mice and later in humans.

Irv Weissman explains the difference between an adult stem cell and an embryonic stem cell (video)

An induced pluripotent stem cell, or iPS cell, is a cell taken from any tissue (usually skin or blood) from a child or adult and is genetically modified to behave like an embryonic stem cell. As the name implies, these cells are pluripotent, which means that they have the ability to form all adult cell types.

Shinya Yamanaka, an investigator with joint appointments at Kyoto University in Japan and the Gladstone Institutes in San Francisco, created the first iPS cells from mouse skin cells in 2006. In 2007, several groups of researchers including Yamanaka and James Thomson from the University of Wisconsin and University of California, Santa Barbara generated iPS cells from human skin cells.

Cancer stem cells are a subpopulation of cancer cells that, like stem cells, can self-renew. However, these cellsrather than growing into tissues and organspropagate the cancer, maturing into the many types of cells that are found in a tumor.

Cancer stem cells are a relatively new concept, but they have generated a lot of interest among cancer researchers because they could lead to more effective cancer therapies that can treat tumors resistant to common cancer treatments.

However, there is still debate on which types of cancer are propelled by cancer stem cells. For those that do, cancer stem cells are thought to be the source of all cells that make up the cancer.

Conventional cancer treatments, such as chemotherapy, may only destroy cells that form the bulk of the tumor, leaving the cancer stem cells intact. Once treatment is complete, cancer stem cells that still reside within the patient can give rise to a recurring tumor. Based on this hypothesis, researchers are trying to find therapies that destroy the cancer stem cells in the hopes that it truly eradicates a patients cancer.

John Dick from the University of Toronto first identified cancer stem cells in 1997. Michael Clarke, then at the University of Michigan, later found the first cancer stem cell in a solid tumor, in this case, breast cancer. Now at Stanford University School of Medicine, Clarke and his group have found cancer stem cells in colon cancer and head and neck cancers.

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Catriona Jamieson talks about therapies based on cancer stem cells (4:32)

Stanford Publication: The true seeds of cancer

UCSD Publication: From Bench to Bedside in One Year: Stem Cell Research Leads to Potential New Therapy for Rare Blood Disorder

Updated 2/16

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Advance Stem Cell Therapy in India | Stem Cell Treatment …

By Dr. Matthew Watson

Plan your Stem Cell Therapy in India with Tour2India4Health Consultants

Stem cell therapy in India is performed by highly skilled and qualified doctors and surgeons in India. Our hospitals have state-of-art equipment that increase success rate of stem cell treatment in India. Tour2India4Health is a medical value provider that offers access to the stem cell therapy best hospitals in India for patients from any corner of the world. We offer low cost stem cell therapy at the best hospitals in India.

Stem cells have the ability to differentiate into specific cell types. The two defining characteristics of a stem cell are perpetual self-renewal and the ability to differentiate into a specialized adult cell type.

Serving as a sort of repair system, they can theoretically divide without limit to replenish other cells for as long as the person or animal is still alive. When a stem cell divides, each "daughter" cell has the potential to either remain a stem cell or become another type of cell with a more specialized function, such as a muscle cell, a red blood cell, or a brain cell.

There are three classes of stem cells i.e totipotent, pluripotent and multipotent (also known as unipotent).

Many different terms are used to describe various types of stem cells, often based on where in the body or what stage in development they come from. You may have heard the following terms:

Adult Stem Cells or Tissue-specific Stem Cells: Adult stem cells are tissue-specific, meaning they are found in a given tissue in our bodies and generate the mature cell types within that particular tissue or organ. It is not clear whether all organs, such as the heart, contain stem cells. The term adult stem cells is often used very broadly and may include fetal and cord blood stem cells.

Fetal Stem Cells: As their name suggests, fetal stem cells are taken from the fetus. The developing baby is referred to as a fetus from approximately 10 weeks of gestation. Most tissues in a fetus contain stem cells that drive the rapid growth and development of the organs. Like adult stem cells, fetal stem cells are generally tissue-specific, and generate the mature cell types within the particular tissue or organ in which they are found.

Cord Blood Stem Cells: At birth the blood in the umbilical cord is rich in blood-forming stem cells. The applications of cord blood are similar to those of adult bone marrow and are currently used to treat diseases and conditions of the blood or to restore the blood system after treatment for specific cancers. Like the stem cells in adult bone marrow, cord blood stem cells are tissue-specific.

Embryonic Stem Cells: Embryonic stem cells are derived from very early embryos and can in theory give rise to all cell types in the body. While these cells are already helping us better understand diseases and hold enormous promise for future therapies, there are currently no treatments using embryonic stem cells accepted by the medical community.

Induced Pluripotent Stem Cells (IPS cells): In 2006, scientists discovered how to reprogram cells with a specialized function (for example, skin cells) in the laboratory, so that they behave like an embryonic stem cell. These cells, called induced pluripotent cells or IPS cells, are created by inducing the specialized cells to express genes that are normally made in embryonic stem cells and that control how the cell functions.

Embryonic stem cells are derived from the inner cell mass of a blastocyst: the fertilized egg, called the zygote, divides and forms two cells; each of these cells divides again, and so on. Soon there is a hollow ball of about 150 cells called the blastocyst that contains two types of cells, the trophoblast and the inner cell mass. Embryonic stem cells are obtained from the inner cell mass.

Stem cells can also be found in small numbers in various tissues in the fetal and adult body. For example, blood stem cells are found in the bone marrow that give rise to all specialized blood cell types. Such tissue-specific stem cells have not yet been identified in all vital organs, and in some tissues like the brain, although stem cells exist, they are not very active, and thus do not readily respond to cell injury or damage.

Stem cells can also be obtained from other sources, for example, the umbilical cord of a newborn baby is a source of blood stem cells. Recently, scientists have also discovered the existence of cells in baby teeth and in amniotic fluid that may also have the potential to form multiple cell types. Research on these cells is at a very early stage.

Stem cell therapy is the use of stem cells to treat certain diseases. Stem cells are obtained from the patients own blood bone marrow, fat and umbilical cord tissue or blood. They are progenitor cells that lead to creation of new cells and are thus called as generative cells as well.

The biological task of stem cells is to repair and regenerate damaged cells. Stem cell therapy exploits this function by administering these cells systematically and in high concentrations directly into the damaged tissue, where they advance its self-healing. The process that lies behind this mechanism is largely unknown, but it is assumed that stem cells discharge certain substances which activate the diseased tissue. It is also conceivable that single damaged somatic cells, e.g. single neurocytes in the spinal cord or endothelium cells in vessels, are replaced by stem cells. Most scientists agree that stem cell research has great life-saving potential and could revolutionize the study and treatment of diseases and injuries.

Stem cell therapy is useful in certain degenerative diseases like

If stem cell therapy is an option, a detailed treatment plan is prepared depending on the type of treatment necessary. Once the patient has consented to the treatment plan, an appointment is scheduled for bone marrow extraction. Please note that this is a minimally invasive surgical procedure, so it is important that patients do not take any blood-thinning medication in the ten days prior to the appointment. It is necessary for each patient to consult their own doctor before discontinuing this type of medication.

The treatment procedure include:

Bone Marrow Extraction: Bone marrow is extracted from the hip bone by the physicians. This procedure normally takes around 30 minutes. First, local anesthetic is administered to the area of skin where the puncture will be made. Then, a thin needle is used to extract around 150-200 ml of bone marrow. The injection of local anesthetic can be slightly painful, but the patient usually does not feel the extraction of bone marrow.

Isolation, Analysis and Concentration of the Stem Cells in the Laboratory: The quality and quantity of the stem cells contained in the collected bone marrow are tested at the laboratory. First, the stem cells are isolated. Then a chromatographical procedure is used to separate them from the red and white blood corpuscles and plasma. The sample is tested under sterile conditions so that the stem cells, which will be administered to the patient, are not contaminated with viruses, bacteria or fungi. Each sample is also tested for the presence of viral markers such as HIV, hepatitis B and C and cytomegalia. The cleaned stem cells are counted and viability checks are made. If there are enough viable stem cells, i.e. more than two million CD34+ cells with over 80 percent viability, the stem cell concentrate is approved for patient administration.

Stem Cell Implantation: The method of stem cell implantation depends on the patient's condition. There are four different ways of administering stem cells:

Intravenous administration:

It is important to understand that while stem cell therapy can help alleviate symptoms in many patients and slow or even reverse degenerative processes, it does not work in all cases. Based on additional information, patient's current health situation and/or unforeseen health risks, the medical staff can always, in the interest of the individual patient, propose another kind of stem cell transplantation or in exceptional situations cancel the treatment.

Allogeneic Stem Cell Transplantation: Allogeneic stem cell transplantation involves transferring the stem cells from a healthy person (the donor) to your body after high-intensity chemotherapy or radiation. It is helpful in treating patients with high risk of relapse or who didnt respond to the prior treatment. Allogeneic stem cell transplant cost in India is comparatively less when contrasted with alternate nations.

Autologous Stem Cell Transplant: Patients own blood-forming stem cells are collected and then it is treated with high doses of chemotherapy. The high-dose treatment kills the cancer cells. They are used to replace stem cells that have been damaged by high doses of chemotherapy, used to treat the patient's underlying disease.

The side effects of stem cell therapy differ from person to person. Listed below are the side effects of stem cell therapy :

According to the Indian Council of Medical Research, all is considered to be experimental, with the exception of bone marrow transplants. However, the guidelines that were put into place in 2007 are largely non-enforceable. Regardless, stem cell therapy is legalized in India. Umbilical cord and adult stem cell treatment are considered permissible. Embryonic stem cell therapy and research is restricted.

There is about a 60% to 80% overall success rate in the use of stem cell therapy in both India and around the world. However, success rates vary depending on the disease being treated, the institute conducting the procedures, and the condition of the patient. In order to receive complete information you will have to contact the medical institutes and ask specific questions concerning the patient's condition.

Mrs. Selina Naidoo with her Son from Malaysia

Tour2India4Health has proved to be a blessing in disguise for me. A medical tourism company with everything at par with our expectations has given me the most satisfactory and relieving experience of my life. I went to them for my sons surgery who was suffering from a serious illness and stem cell therapy was the only choice I had. Trust it was heart wrenching to leave my son under any hands on the operation table. Nevertheless, courageously I had to because thats what I was here for and thats what could get my son a new and healthy life. Sitting at a corner outside the operation theatre was taking my heartbeats away with every second. Finally, the surgery was over and I was there in front of the doctor with closed eyes. He declared that the surgery was successful and my son is fine but needs some extra care and some cautious post operative measures for recovery. All through our stay in the hospital, everything went on brilliantly and after my son recovered completely, I came back to my home country. Even after that for many months, I received regular calls to verify and virtually monitor the health of my child. Now, its been 5 years and when I see my child today it feels as if no surgery was ever done on him. Thanks to the doctor who treated him and to the entire team of nurses and travel professionals who displayed extra warmth and care. Thanks is just a small word to say as a mother of a child.

India is the most preferable destination for patients who are looking for low cost stem cell therapy. Indian doctors and healthcare professionals are renowned world over for their skills with many of them holding high positions in leading hospitals in US, UK and other countries around the world. There are significant numbers of highly skilled experts in India, including many who have relocated to India after having worked in the top hospitals across the world.

The Cost of stem cell treatment in India are generally about a tenth of the costs in US and are significantly cheaper compared with even other medical travel destinations like Thailand

*The price for the Stem Cell Therapy is an average collected from the 15 best corporate hospitals and 10 Top Stem Cell Experts of India.

*The final prices offered to the patients is based on their medical reports and is dependent on the current medical condition of the patient, type of room, type of therapy, hospital brand and the surgeon's expertise.

We have worked out special packages of the Stem Cell Therapy for our Indian and International patients. You can send us your medical reports to avail the benefits of these special packages.

You would be provided with 3 TOP RECOMMENDED SURGEONS / HOSPITALS FOR YOUR STEM CELL THERAPY in India.

There are many reasons for India becoming a popular medical tourism spot is the low cost stem cell treatment in the area. When in contrast to the first world countries like, US and UK, medical care in India costs as much as 60-90% lesser, that makes it a great option for the citizens of those countries to opt for stem cell treatment in India because of availability of quality healthcare in India, affordable prices strategic connectivity, food, zero language barrier and many other reasons.

The maximum number of patients for stem cell therapy comes from Nigeria, Kenya, Ethiopia, USA, UK, Australia, Saudi Arabia, UAE, Uzbekistan, Bangladesh.

Cities where top and world renowned Stem Cell Therapy hospitals and clinics situated are :

We have PAN-India level tie ups with TOP Hospitals for Stem Cell Therapy across 15+ major cities in India. We can provide you with multiple top hospitals & best surgeons recommendations for Stem Cell Therapy in India.

India has now been recognized as one of the leaders in medical field of research and treatment. Tour2India4Health Group was established with an aim of providing best medical services to its patients and since then has been working hard in maintaining itself as one of the most professional healthcare tourism providers in India. With a number of world-renowned medical facilities affiliated, we have the resources to offer you the finest medical treatment in India, and help your speedy recovery. Tour2India4Health Group has always believed and practiced providing its patients best surgery and treatment procedure giving a second chance to live a more better and normal life. Our team serves the clientele most comfortable and convenient measures of healthcare services thus, making your medical tour to India very fruitful experience.

Our facilitation:

We has been operating patients from all major countries like USA, United Kingdom, Italy, Australia, Canada, Spain, New Zealand, and Kuwait etc. We have network of selected medical centers, surgeons and physicians around various cities in India, who qualify our assessment criteria to ensure that our core values of Safety, Excellence and Trust are maintained in all our services.

Below are the downloadable links that will help you to plan your medical trip to India in a more organized and better way. Attached word and pdf files gives information that will help you to know India more and make your trip to India easy and memorable one.

Best Stem Cell Therapy in India, Cost of Stem Cell Therapy in India, Stem Cell Therapy Best Hospitals in India, Success Rate of Stem Cell Treatment in India, Stem Cell Therapy Treatment Cost in India, Allogeneic Stem cell Transplant Cost in India, autologous Stem Cell Transplant Cost in India, Stem Cell Therapy in India, Low Cost Stem Cell Therapy India, Stem Cell Benefits in India, Top Stem Cell Centers in India, Best Doctors for Stem Cell Therapy in India, List of Best Stem Cell Treatment Clinics in India, Allogeneic stem cell transplantation, Allogeneic Stem Cell Transplant Cost in India, Autologous Stem Cell Transplant, Autologous Stem Cell Transplant Cost in India

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Advance Stem Cell Therapy in India | Stem Cell Treatment ...

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Autologous iPS cell therapy for Macular Degeneration: From bench-to-bedside

By Dr. Matthew Watson

Presented At:Gibco - 24 Hours of Stem Cells Virtual Event

Presented By:Kapil Bharti - Stadtman Investigator, NIH, Unit on Ocular Stem Cell & Translational Research

Speaker Biography:Dr. Kapil Bharti holds a bachelor's degree in Biophysics from the Panjab University, Chandigarh, India, a master's degree in biotechnology from the M.S. Rao University, Baroda, India, and a diploma in molecular cell biology from Johann Wolfgang Goethe University, Frankfurt, Germany. He obtained his Ph.D. from the same institution, graduating summa cum laude. His Ph.D. work involved research in the areas of heat stress, chaperones, and epigenetics.

Webinar:Autologous iPS cell therapy for Macular Degeneration: From bench-to-bedside

Webinar Abstract:Induced pluripotent stem (iPS) cells are a promising source of personalized therapy. These cells can provide immune-compatible autologous replacement tissue for the treatment of potentially all degenerative diseases. We are preparing a phase I clinical trial using iPS cell derived ocular tissue to treat age-related macular degeneration (AMD), one of the leading blinding diseases in the US. AMD is caused by the progressive degeneration of retinal pigment epithelium (RPE), a monolayer tissue that maintains vision by maintaining photoreceptor function and survival. Combining developmental biology with tissue engineering we have developed clinical-grade iPS cell derived RPE-patch on a biodegradable scaffold. This patch performs key RPE functions like phagocytosis of photoreceptor outer segments, ability to transport water from apical to basal side, and the ability to secrete cytokines in a polarized fashion. We confirmed the safety and efficacy of this replacement patch in animal models as part of a Phase I Investigational New Drug (IND)-application. Approval of this IND application will lead to transplantation of autologous iPS cell derived RPE-patch in patients with the advanced stage of AMD. Success of NEI autologous cell therapy project will help leverage other iPS cell-based trials making personalized cell therapy a common medical practice.

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Autologous iPS cell therapy for Macular Degeneration: From bench-to-bedside

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Human iPS cell-derived dopaminergic neurons function in a …

By Dr. Matthew Watson

Kriks, S. et al. Dopamine neurons derived from human ES cells efficiently engraft in animal models of Parkinsons disease. Nature 480, 547551 (2011)

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Stem Cell Therapy for Neuropathy: What Can We Expect …

By Dr. Matthew Watson

As the body ages, its only natural that some of its processes should break down. Humans become clumsier, stiffer, their reaction times slower, their senses duller. This is often due to the fact that nerves in the extremities grow less sensitive over time, transmitting messages to the brain more slowly and feeling less acutely a condition known as peripheral neuropathy or simply neuropathy.

While some of that is normal, especially in the golden years, neuropathy often manifests in people much too young in their 30s, 40s, or 50s as a result of a disease such as diabetes or autoimmune issues. Unfortunately, the condition can significantly hamper a persons quality of life, making mobility difficult and limiting everyday activities.

The good news? Neuropathy may have a cure, or at least a solid treatment, on the horizon. Stem cells show great promise for a wide variety of conditions, and nerve damage is the latest of these. To see how it can help, its important to understand what stem cell treatment is, what neuropathy is and what causes it, and how the former can address the latter.

In this article:

The body is made of trillions of tissue-specific cells, making up organs, skin, muscle, bone, nerves, and all other tissue. Some of these can renew indefinitely, such as blood cells. Others, however, cannot replace themselves: Once they have divided a certain number of times or become damaged, theyre dead for good. That goes for nerves and brain tissue, for example.

There is, however, an answer. The developing embryo uses stem cells, or master cells capable of differentiating into any kind of tissue in the human body, to transform one fertilized egg into a fully functional baby human. While adult humans lack these pluripotent stem cells that can transform into anything, they do have multipotent stem cells, which are tissue-specific master cells (such as blood cells).

By harvesting these multipotent stem cells from blood or fat tissue, scientists can induce the cells to become pluripotent, meaning theyre now capable of becomingany tissue in the human body. Essentially, researchers have figured out how to reverse-engineer adult stem cells to become all-powerful embryonic cells. This meansstem cells have a huge range of possible uses.

In other cases, multipotent stem cells alone are enough to heal some parts of the human bodysuch as nerves.

Peripheral neuropathymanifests in a number of ways. It causes pain, weakness, and tingling in affected areas, making it hard to lift objects, grasp items, walk competently, and more. Typically it affects the hands and feet most strongly, though it can also cause symptoms in the arms, legs, and face. Not only does it affect motor coordination,but it also makes it hard for the body to sense the environment, including temperature, pain, vibration, and touch.

A more serious manifestation of the disease is autonomic neuropathy, which influences more than the periphery of the body. It also messes with blood pressure, bladder and bowel function, digestion, sweating, and heart rate. Polyneuropathy is when the condition starts at the periphery of the body but gradually spreads inward.

Diabetic neuropathy is the most well-known incarnation of this disease. It is a result of high glucose and fat levels in the blood, which can damage nerves.Other causes include:

If the bad news is there are so many potential causes of neuropathy, the good news is stem cell treatments have the potential to address all of them.

In the case of neuropathy, stem cell treatment is simpler than in other conditions. Mesenchymal stem cells (certain types of multipotent stem cells) releaseneuroprotective and neuroregenerative factors, so when they are injected into the bloodstream they can begin to rebuild nerves and undo the damage caused by the disease. Also, because these stem cells replicate indefinitely, they will offer these benefits for the rest of the patients life.

The basic process is that scientists harvest these cells from the patient (autologous transplant) or from a donor (allogeneic transplant), then cultivate them until they reach certain levels before reinjecting them back into the patient. The stem cells, with the help of hormones and growth factors, seek out and repair the damage done by neuropathy.

The main risks to stem cell treatment include reaction to the injection. In an autologous transplant, the patient may react to the preservatives and other chemicals used by way of necessity. In an allogeneic transplant, the patient may exhibit an immune response to donor cells, or vice versa with the donor cells seeing the patients body as an invader and attacking it. All of the above reactions can prove minor or, on the other end of the spectrum, fatal.

The severity of the problem will, therefore, dictate whether or not it is worth moving forward. Note that those whodochoose to pursue the treatment often have extremely good results.

Unlike some other stem cell treatments, which remain in preliminary stages, stem cell therapy for neuropathy has thus far received serious attention. However, thesmall sample size and difficult conditions of clinical trialsmake it hard to say yet whether this treatment will become widespread or receive FDA approval.Other studies have demonstrated more significant resultsin the treatment of facial pain and may pave the way for future neuropathy treatments using stem cells.

For now, those suffering from neuropathy should seek the advice of a physician. If there are clinical trials available nearby, thats the place to start. Its possible to seek stem cell therapy through a clinic as well as through a clinical study or research institution, but make sure to research the provider thoroughly. With stem cells becoming such a relevantapproach to medical conditions of all kinds, its not safe to conclude that all providers are equally experienced or effective.

If you found this blog valuable, subscribe to BioInformants stem cell industry updates.

As the first and only market research firm to specialize in the stem cell industry, BioInformant research is cited by The Wall Street Journal, Xconomy, AABB, and Vogue Magazine. Bringing you breaking news on an ongoing basis, we encourage you to join more than half a million loyal readers, including physicians, scientists, executives, and investors.

Did this article address your concerns about neuropathy? Let us know in the comments section below.

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Stem Cell Therapy for Neuropathy: What Can We Expect

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What is CAR-T Cell Therapy | CAR-T Definition | Bioinformant

By Dr. Matthew Watson

CAR-T cell therapy is asa type of immunotherapy that teaches T cells to recognize and destroy cancer.CAR-T cell therapy has demonstrated promising results in a range of patients from young and old. In some patients, this can lead to the total elimination of the cancer. In others, there is a significant improvement of the disease.

For those who are facing cancer, it is important to answer the question What is CAR-T? This guide will answer the most common questions about CAR-T cell therapy for readers who want to understand this novel technology platform for treating cancer.

What you need to know about CAR-T therapy and its role in cancer treatment is described below.

CAR-T is pronounced phonetically, as car tee cell.CAR-T is named after a mythical creature called the chimera. A chimera is an animal made of different parts of different animals attached together.

With CAR-T cell therapy, apatientsTcells are modified within a laboratory, so that they they can find and attack cancer cells. Because CAR-T cells combine different parts from different sources, they are called chimera (meaning, blended or fused) antigen receptor T cells.

T cells are a type of white bloodcell that plays a central role in the immune response within humans.T cell that have been genetically altered into CAR-T cells function as living drugs when they are administered to patients.

To understand CAR-T cell therapy,a brief history of immunologymay prove helpful. An antigen is a foreign substance in the body, either a toxin or disease agent or unhealthy cell (as in cancer), that triggers an immune response. The body then produces white blood cells to attack the agent. It does this by binding to it with the use of antigen receptors on the surface of the white blood cells, or lymphocytes. Only then does the body produce antibodies to destroy the foreign or diseased agent.

The problem is T cells, the white blood cells responsible for destroying tumor cells,are not good enough at recognizing it. Therefore, in order to increase the patient immune levels, medical specialists take blood. From the blood, they harvest T cells and add extra antigen receptors to the surface of the cells. They inject those cells back into the patient via blood transfusion, where they multiply and can then attack cancer, either with or without the aid of additional therapies.

Specifically, the antigens can then recognize the protein CD-19, which forms on the surface of B cells, a type of blood cell that frequently becomes cancerous. By knowing which proteins to look for, the modified T cells can hunt them down, attack, and destroy them throughout the bloodstream.

CAR-T cells are defined as T-cells (immune cells) that have been modified to match markers present on the outside of cancer cells, allowing them to selectively find and attack them. To create CAR-T cells, physicians extract T-cells from a patient, genetically alter them, expand them in quantity, and re-infuse them to the patient so that the engineered CAR-T cell can selectively attack cancer cells.

The patient response is then monitored using a variety of tools.

There are four steps involved with the CAR-T cell therapy process.

These steps include:

The patient is then monitored by the attending physicians to document the therapeutic response.

Cancer is a silent killer. Too often, it has devastating results, because the cells in the human body are not adept at killingit. This is the case with T cells, human immune cells whose responsibility is to fight invasion and disease. These cells, also known as T lymphocytes a special type of white blood cell are not always able to recognize and eliminate cancer.A potential new solution may be CAR-T cell therapy.

As theCancer Treatment Centers of Americapoints to CAR-T treatment as a novel way to treat cancer, it could drastically alter the medical outlook for both children and adults. These patients would otherwise be without the possibility of a cure.

However, CAR-T immunotherapy is not a cure-all for every patient. For some, it only works for a short time before the cancer relapses. Other patients respond to it, but suffer such severe side effects that it does almost nothing to ease the symptoms. While researchers work furiously to determine why some treatments work on cancer cells and others do not, they still have not arrived at a firm answer.

During transport and until ready to administer at bedside CAR-T cells must be stored at least -150 Celsius. @SylvesterCancer is the only center in South Florida certified to treat patients with this novel #immunotherapy pic.twitter.com/1LKm6UHzd8

Sylvester Cancer (@SylvesterCancer) August 7, 2018

In 2017, two experimental CAR-T treatments received approval from the U.S. FDA with more in clinical trials:

Kymriah was approved by FDA in August 2017 to be used in children and adults with ALL. In May 2018, the FDA approved Kymriah for a second indication (diffuse large B-cell lymphoma). The second CAR-T product, Yescarta, was approved by FDA in October 2017 for patients with lymphoma.In August 2018, both Kymriah and Yescarta secured European regulatory approval. In September 2018, Health Canada made Kymriah the first CAR-T therapy to receive regulatory approval in Canada.

Numerous companies are also working to perfect the technology of CAR-T cells. Akron Biotechmodifies many types of cells for use in medical treatments.

CAR-T is a new technology. Not only is it expensive to manufacture antigens in a lab and attach them to T cells, it takes a long time and carries a number of different specifications in order for candidates to gain approval for the treatment. So, exactly which candidates can receive therapy?

Both treatment protocols modify T cells to help them recognize and attack diseased B cells in the blood. Patients with either leukemia or B-cell lymphoma may apply for the clinical trial at this time. However, they cannot do so without first trying at least two other cancer therapies of a more standard nature.

Currently, researchers are experimenting with CAR-T therapies for other types of cancers as well. These include leukemia and lymphoma subtypes, as well as non-blood-borne cancers. Its ability to fight solid tumors, or those that do not spread throughout blood or bone marrow, have thus far proven less than impressive.

Physicians make CAR-T cells via a careful process. First, the patient is set up in the hospital and prepped for a blood draw, followed by a long stay. Most patients are quite ill by the time they start CAR-T cell immunotherapy, necessitating they remain in the hospital until the completion of the treatment.

Doctors then take a patients blood and feed it into anapheresis machine. This device separates out the white blood cells, T cells included. Then it feeds the remaining blood back to the patient. This means they do not lose a lot of blood while physicians now have a healthy supply of cells to transform. Doctors then freeze the harvested cells and send them off to a lab.

Lab workers then take the collected T cells and introduce a gene that manufactures the chimeric antigen receptor into the DNA of each cell. Lab workers then grow millions of versions of these cells. Once they have enough, they harvest the cells, freeze them once more and deliver them back to the patient via transfusion.

Both these T cells, plus the ones subsequently manufactured by the patients body, can then bind to and attack the cancer cells.

Because transforming T cells is such a complex process, the treatment is typically a long one for the patient. From beginning to end, the transformation and reintroduction of cells may take up to 3 weeks. During that time, the patient is compromised even more than usual due to the reduction in their T cell population. Thats why they usually stay in the hospital during the entire process. This way, doctors can monitor them and make sure their immunity stays as robust as possible.

Before introducing the modified T cells to the patient, physicians typically give them a round of chemotherapy. This helps to weaken their immune system further, which reduces the chances that existing T cells will outnumber the new ones. Counterintuitively, by depressing the immune system in the short run, doctors give patients the best chance of engineered T cells multiplying and doing their job.

The transfusion itself is typically short and painless, lasting only about an hour. After staying in the hospital for monitoring, patients must come in regularly for a few weeks afterward.

The huge benefit of a treatment like this is the T cell modifications will last for life. Each time a bodys T cells encounter a toxin or disease agent and develop antigen receptors and antibodies to fight it, the person has that ability forever. That means patients who receive modified T cells now have the tools to fight their particular cancer for the remainder of their days.

This makes CAR-T cell therapy more than a treatment. For example, while chemotherapy and radiation are effective, their curative effects end when the treatment ends (or, more accurately, a few days or weeks after the last course). In contrast, modified T cells hang aroundforever, turning this type of immunotherapy into a living drug.

While CAR-T therapies are long-lasting, making them more affordable over a lifetime, it is expensive to access these therapies.Currently, Kymriah and Yescarta are offered at the following prices:

Moreover, possible side effects do exist. These include:

Finally, while the process is very beneficial to some patients, it is extremely time-consuming. Some question where it can actually serve the broader population, considering the necessary time and specialization required.

Do you need a visual look at how CAR-T therapy works? Watch this video from Associated Press.

CAR-T companies are on the rise, supported by growing investment flowing into CAR-T product development and landmark approvals of CAR-T cell therapies by the U.S. FDA, European Medicines Agency (EMA), and Health Canada.

Are you interested to know the identities of the companies developing CAR-T therapies worldwide?

For a limited-time, you can claim the Global Database of CAR-T Cell Therapy Companies and get the CAR-T Funding Brief ($49 value) for FREE:

Overall, T-cell therapy has proven a promising new treatment approach. As its manufacture, administration, and safety profile improve, it will become an important tool in the cancer treatment toolkit.

Do you know anyone in need of a cancer cure? What role could CAR-T therapy play in their treatment? Let us know in the comments below.

What is CAR-T Cell Therapy? | CAR-T Definition

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Japan’s Laws Supporting Accelerated Pathways for Cell …

By Dr. Matthew Watson

In late 2014, Japan passed two new laws that revolutionizedthe commercialization ofcell therapies within the country by providing an accelerated pathway for product approvals. While there has been much discussion about these laws, few people have a clear understanding of the implications of these regulations on a global scale.

Below, we summarize the laws, identify their importance, and most importantly, speak to howJapan has become a gateway country for regenerative medicines.

New regulations accelerating the approval of regenerative therapeutics in Japan took effect November 25, 2014. The significance of these regulations is that they allow companies to receive conditional marketing approval and commercialize regenerative medicine products while clinical trials continue through the later stages.

The accelerated commercialization of cell therapies is part of the economic revitalization plan initiated by Prime Minister Shinz Abe. Under Shinz Abe, Japan has been pursuing regenerative medicine and cellular therapy as key strategies to the Japans economic growth. Japans Education Ministry also indicated that it is planning to spend 110 billion yen ($1.13 billion) on iPS cellresearch during the next 10 years, and the Japanese parliament has been discussing bills that would speed the approval process and ensure the safety of such treatments.[1]

In late 2014, Japan exercised the following acts:

The aim of the first act was to accelerate the clinical application and commercialization of innovative regenerative medicine therapies. It covers clinical research and medical practice using processed cells and specifies the procedure required for clearance to administer cell procedures to humans. These guidelines are very important to the use the cells within clinical stages.

The PMD Acts definition of regenerative medicine includes tissue-engineered products, cell therapy products, and gene therapy products.

The intent of the laws is to accelerate the commercialization of cell therapeutics within Japan by allowing companies to benefit from conditional marketing authorization.

Therefore, cell therapies that show safety and probable efficacy during Phase I and Phase II trials can get conditional approval for up to seven years, during which time:

1) Larger-scale, later-stage clinical trials are performed2) Revenue from the cell therapy is pursued within the Japanese market

During the seven-year conditional approval period, companies must continue to submit clinical trial data to Japans Pharmaceuticals and Medical Devices Agency (PMDA), and subsequentlyapply for final marketing approval or withdraw the product within seven years.

This safety data can then be used by non-Japanese participants, which is a massive benefit to foreign companies, such as those located in the United States. The regulatory environment in Japan provides companies with the unique opportunity to fast track a clinical trial and seek approval of a new cell therapy product within the Japanese market.

As Kaz Hirao, CEO of Cellular Dynamics International (CDI), shared with BioInformant:This has made Japan a gate country for developing innovative cell therapies with the potential to address major unmet medical needs. It has has provided a strategic opportunity to American companies, because they can benefit from fast track applications through doing clinical testing within Japan and subsequently developing its cell therapy across the rest of the world. Numerous American and Australian companies are pursuing this strategy, as well as other companies from other countries worldwide.[2]

Footnotes[1] Dvorak, K. (2014).Japan Makes Advance on Stem-Cell Therapy[Online]. Available at: http://online.wsj.com/news/articles/SB10001424127887323689204578571363010820642. Web. 8 Apr. 2015.[2]Interview with Kaz Hirao, CEO of Cellular Dynamics International (CDI), a FUJIFILM Company. Conducted by BioInformants President/CEO, Cade Hildreth [January 29, 2017]. Available at: https://bioinformant.wpengine.com/cellular-dynamics-cdi-kaz-hirao/.

Japans Laws Supporting Accelerated Pathways for Cell Therapies

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Cell Regeneration Perth | Cell Rejuvenation and Cell Therapy

By Dr. Matthew Watson

There are a lot of theories as to why people change as they get older. Some claim that aging is caused by injuries from ultraviolet light over time, wear and tear on the body, or by-products ofmetabolism. Other theories view aging as a predetermined process controlled by genes.

No single process can explain all the changes of aging. Aging is a complex process that varies as to how it affects different people and even different organs. Most gerontologists (people who study aging) feel that aging is due to the interaction of many lifelong influences. These influences include heredity, environment, culture, diet, exercise and leisure, past illnesses, and many other factors.

Unlike the changes of adolescence, which are predictable to within a few years, each person ages at a unique rate. Some systems begin aging as early as age 30. Other aging processes are not common until much later in life.

Although some changes always occur with aging, they occur at different rates and to different extents. There is no way to predict exactly how you will age.

Some studies have shown that Cell Regeneration treatments have a better effect on people over the age of 35, however this has no clinical evidence to back it up. What we do know is that as we age our bodies do not renew cell turnover at the same rate as it did in our younger years. And there appears to be no end age for these treatments to have some effect.

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Kotton Lab – Boston University Medical Campus

By Dr. Matthew Watson

The Kotton labs goal is advancing our understanding of lung disease and developmental biology with a focus on stem cell biology and gene therapy. We believe that novel treatments for many lung diseases can be realized based on a better understanding of how the lung develops as well as regenerates after lung injury.

We are particularly interested in understanding how lung cells decide and remember who they are. To this end, one focus of our group is defining the genomic and epigenomic programs that regulate lung cell fate. A longer term goal is the de novo generation of the full diversity of lung lineages and transplantable 3D lung tissues from pluripotent stem cells. Our Principal Investigator, Dr. Darrell Kotton, also serves as the founding Director of the Center for Regenerative Medicine (CReM). Take a full tour of the CReM by clicking on our logo above.

Click on the menu to learn more about our research areas and our team

Have forty five minutes for an overview of our last decade? Listen here to Darrells ATS Discovery Series Lecture, Lung Regeneration: An Achievable Mission.

Open Source Works! Click here to access our:iPS Cell Lines, Lentiviral Vectors, Bioinformatics Datasets, or Detailed Protocols!

or read more about our Open Source Biology Philosophyor a recent interview on Darrells approach to sharing our cells

More photos of lab life

See all our latest news on twitter:

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Stem Cell Therapy For Knees | What You Need To Know …

By Dr. Matthew Watson

The main conditions treated by stem cell injections include knee osteoarthritis, cartilage degeneration, and various acute conditions, such as a torn ACL, MCL, or meniscus. Stem cell therapy may speed healing times in the latter, while it can actually rebuild tissue in degenerative conditions such as the former.

Thats a major breakthrough. Since cartilage does not regenerate, humans only have as much as they are born with. Once years of physical activity have worn it away from joints, there is no replacing it. Or at least, there wasnt before stem cell therapy.

Now, this cutting-edge technology enables physicians to introduce stem cells to the body. Thesemaster cells are capable of turning into formerly finite cell types to help the body rebuild and restore itself.

Although it may sound like an intensive procedure, stem cell therapy is relatively straightforward and usually minimally invasive. These days, physicians have many rich sources of adult stem cells, which they can harvest right from the patients own body. This obviates the need for embryonic stem cells, and thereby the need for moral arguments of yore.

Mesenchymal stem cells (MSCs) are one of the main types used by physicians in treating knee joint problems. These cells live in bone marrow, butincreasing evidence shows they also exist in a range of other types of tissue.This means they can be found in places like fat and muscle. With a local anesthetic to control discomfort, doctors can draw a sample of tissue from the chosen site of the body. The patient usually doesnt feel pain even after the procedure. In some cases, the physician may choose to put the patient under mild anesthesia.

They then isolate the mesenchymal stem cells. Once they have great enough numbers, physicians use them to prepare stem cell injections. They insert a needle into the tissue of the knee and deliver the stem cells back into the area. This is where they will get to work rebuilding the damaged tissue. Although the mechanisms arent entirely clear, once inserted into a particular environment, mesenchymal stem cells exert positive therapeutics effectsinto the local tissue environment.

Mechanisms of action of mesenchymal stem cells appear to include reducing inflammation, reducing scarring (fibrosis), and positively impacting immune system function.

Thats not quite enough to ensure a successful procedure, however. Thats why stem cell clinics may also introduce growth factors to the area. These are hormones that tell the body to deliver blood, oxygen,and nutrients to the area, helping the stem cells thrive and the body repair itself.

Physicians extract these growth factors from blood in the form of platelet-rich plasma (PRP). They take a blood sample, put it in a centrifuge and isolate the plasma, a clear liquid free of red blood cells, but rich in hormones needed for tissue repair.

So, what can a patient reasonably expect when it comes to stem cell therapy, in terms of time and cost outlay?

The answers to both of these questions differ depending on the clinic doing the procedure and the patients level of knee degradation. Some clinics recommend a course of injections over time. Meanwhile, others prepare the injection and deliver it back to the patient in only a matter of hours. Either way, the treatment is minimally invasive, with fast healing times and a speedy return to normal (and even high-intensity) activity.

Some quotes for stem cell knee treatment are as low as $5,000. Others cost up to $20,000 or more. Again, this depends on how many treatments a patient needs, as well as how many joints theyre treating at the same time. Because its easier to batch prepare stem cells, a patient treating more than one knee (or another joint) can address multiple sites for far less. The procedure would only cost an addition of about $2,000 or so per joint.

No treatment proves effective every time. However, insofar as patients reporting good results for stem cell injections, the overall evidence does lean in a beneficial direction.Studies at the Mayo Clinic, for instance, indicate that while further research is needed, it is a good option for arthritis in the knee. Anecdotal reports are positive as well. Patients report it as an effective alternative to much more invasive solutions, such as arthroscopic or knee replacement surgery.

Other studies point to the need for caution. Stem cell therapy and regenerative medicine, in general, are only now exiting their infancies. There arent enough high-quality sources from which to draw at this point, so hard and fast conclusions remain elusive. Of the studies that do exist, some contain unacceptably high levels of bias.

Of course, any new treatment will face these kinds of challenges in the beginning. For those who need an answer to knee pain, and havent yet found one that works, its likely worth the risk that it wont prove as effective as they hoped. But what about other risks?

The good news about this form of stem cell therapy is that the patient uses their own cells. That means they completely skip over the dangers that accompany donor cells. The main one of which is graft-versus-host disease (in which the donor cells initiate an immune response against the patients body). Because the cells have all the same antibodies, neither the body nor the reintroduced cells will reject one another.

Also, the relatively low-stakes outpatient nature of the procedure (versus, say, a bone marrow transplant) means that the chances of something going wrong are much reduced.

However, there do exist some risks wherever needles come into play. It is possible to get an infection at the site of the blood draw as well as at the injection site, but these risks are quite low. Other risks include discoloration at theinjection site or soreness. While some people fear the possible growth of stem cells at the site of injection into a tumor, it is unlikely for this to happen, because physicians utilize adult stem cells for these procedures that have a low proliferative capacity.

These adult stem cells tend to be much safe than pluripotent stem cell types. Examples of pluripotent stem cells are embryonic stem cells (derived from embryos) and a type of lab-made stem cell known as induced pluripotent stem cell (iPS cell).

For those who think stem cell therapy could prove beneficial, its time to set up a consultation with your doctor. Multiple factors will influence whether or not its a good idea. These include age, health, andseverity of the condition and other available treatments. However, overall, this form of regenerative medicine is reasonably affordable, very low-risk, and typically effective.

Are you seeking a stem cell treatment for your knees or other joints?To support you,we have partnered withOkyanosa state-of-the-art facility providing patients with advanced stem cell treatments.

The group offers treatments for arange of chronic conditions, includingosteoarthritis and degenerative joint disease, which are leading causes of knee pain.

If you are seeking a stem cell treatment for knee pain or other chronic condition,contact Okyanos for a Free Medical Consultation.

What questions do you still have about stem cell therapy for knees? Ask them below and we will get you answers.

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The Cost Of Stem Cell Therapy And Why It’s So Expensive …

By Dr. Matthew Watson

How much is stem cell therapy? As stated by CBC Canada,the cost of stem cell therapy is $5,000 to $8,000per stem cell treatment for patients. According to a Twitter poll by BioInformant, the cost can be even higher. Our May 2018 poll found that stem cell treatments can cost as much as $25,000 or more. This article explores the key factors that impact the cost of stem cell therapy, including the type of stem cells used within the protocol, the number of treatments required, and the site of theclinic. It also provides pricing quotes from stem cell clinics within the U.S. and worldwide.

In this article:

Stem cell therapy is the use of living cells as therapeutics to treat disease or injury. Read on to learn about the cost requirements of these procedures.

CBC Canadas pricing involves Cell Surgical Network (CSN) following its protocol to remove fat tissue and process it before re-injecting [adipose-derived stem cells] either directly or intravenously into the same patient. Unfortunately, the U.S. FDA and Department of Justice (DOJ) sent this network of stem cell treatment providers a permanent injunction notice in May 2018. Therefore, patients should not seek treatments from the group at this time.Although Cell Surgical Network (CSN) is based in California, it has a network of approximately 100 U.S. treatment centers. They also have three Canadian clinics located in Vancouver, Sudbury,andKamloops.

The controversy such as the one above stirs up questions about the safety of stem cell procedures. Anyone considering stem cell therapy from any tissue or source will benefit from understanding the possible consequences of stem cell therapy and the factors driving costs.

For the patient, a stem cell transplant involves multiple steps, including:

There are also real costs for the doctors who provide stem cell treatments. They have overhead costs, including:

There is also time and expertise required toperform the procedure and offer post-operative care. In some cases, the physician must pay licensing fees to access stem cell sourcing, processing, or delivery technologies.

Stem cell treatment has gained more and more traction over the last decade. It has been helped along by considerable advances in research. In 2017, the number of scientific publications about stem cells surpassed 300,000. The number of stem cell clinical trials has also surpassed4,600 worldwide.

However, stem cell therapy is still expensive. Among the cheapest and easiest options is to harvest adipose-derived stem cells (ADSCs) those that exist in adult fat layers and re-deliver them to the patient. Unlike harvesting from bone marrow or teeth, providers can feasibly remove fat, separate stem cells, then re-inject them into a patient the same day. This approach is typically less expensive than those that require more invasive procedures for harvesting. Because of its practicality in terms of cost, it has become a common approach to stem cell treatment.

Relatively easy harvesting stilldoesnt translate to inexpensive cost, although some are certainly more affordable than others. For orthopedic conditions, the costof stem cell therapy is typically lower, averaging between $5,000 and $8,000. Examples of these types of medical conditions include:

Note that these prices are typically out-of-pocket costs paid by the patientbecause most insurance companies will not cover them. They are considered experimental and unapproved by the FDA. This means patients needing stem cell treatment will need to use their own savings.

Although fat is a frequently utilized source for stem cells, it is also possible for physicians to utilize stem cells from bone marrow. Regenexx provides this service in the U.S. and Cayman Islands. With theRegenexxstem cell injection procedure, a small bone marrow sample is extracted through a needle, and blood is drawn from a vein in the arm. These samples are processed in a laboratory, and the cells it contains are injected into an area of the body that needs repair. On June 19, 2018, ACAP Health, a leading provider in innovative, clinical-based solutions partnered with Regenexx to reduce high-cost musculoskeletal surgeries.ACAP Health is a national leader in employer healthcare expense reduction. It is one of the first healthcare groups to partner with a stem cell treatment group to support insurance coverage to patients.

A recent Twitter poll conducted by BioInformant reported that, on average, patients can expect to spend $25,000 or more on stem cell therapies. According to the poll,

Most likely, those paying lower stem cell treatment costs under $5,000 were pursuing treatment for orthopedic or musculoskeletal conditions. In contrast, those paying higher treatment costs were likely getting treated for systemic or more complex conditions, such as diabetes, multiple sclerosis (MS), neurodegenerative diseases (such as Alzheimers disease or dementia), psoriatic arthritis, as well as the treatment for autism.

In the U.S., treatment protocols vary depending on the clinic and the treating physician. A one-time treatment that utilizes blood drawn from a patient can cost as little as $1,500. However, protocols that utilize a bone marrow or adipose (fat) tissue extraction can run as much as $15,000 $30,000. This is because bone marrow extraction is an invasive procedure that requires a penetrating bone and adipose tissue extraction requires a medical professional trained in liposuction.

For treatments that require a systemic or whole-body approach, the cost tends to be in the higher range, often averaging from $20,000 to $30,000. Examples of the diseases or conditions requiring this type of stem cell treatment include:

These higher costs reflect the complexity of treating these patients and the fact that multiple treatments are often required.

Founded by Dr. Neil Riordan, a globally recognized stem cell expert, theStem Cell Institutein Panama is one of the worlds most trusted adult stem cell therapy centers. Over the past 12 years, the center has performed more than10,000 procedures, making it a widely recognized destination for stem cell treatments.

Working in collaboration with universities and physicians worldwide, its stem cell treatment protocols utilize combinations of allogeneic human umbilical cord blood stem cells and autologous bone marrow stem cells to treat a wide variety of conditions.

A reader of BioInformant was recently treated for psoriatic arthritis at the Stem Cell Institute in Panama in early 2018. The price of his stem cell treatment was $22,000. With travel and lodging included, the total expenses were approximately $30,000.

Because of its proximity to the U.S., Mexico is increasingly becoming a destination for medical tourism.Before choosing a stem cell treatment provider in Mexico, ensure the clinic is fully authorized by COFEPRIS, the Mexican equivalent to the FDA.

One patient who recently shared stem cell treatment quotes with BioInformant found that the treatment for glycogen storage disease, a metabolic disorder that often onsets in infancy and continues into adulthood, would cost $23,900 throughGIOSTAR Mexico.

In contrast, the patient was quoted$33,000 throughCelltex, a U.S.-based company that treats patients in Cancun, Mexico.Celltex follows FDA regulations concerning the export of cells to Mexico and is compliant with the standards and procedures of COFEPRIS. Celltex also has an alliance with a certified hospital in Mexico, which is approved to receive cells and administer them to patients by a licensed physician.

In contrast, the patient was quoted $10,000 from Stem Cell Therapy of Las Vegas and Med Spa, an American clinic. This price difference may reflect regulatory restrictions that prevent U.S. providers from expanding cells. It may also reflect the therapeutic approach used by the clinic, as well as the quality of their expertise.

In Mexico, where certain types of stem cell expansion are allowed that are restricted within the U.S., treatment protocols vary depending on the clinic and the treating physician. A one-time treatment that utilizes peripheral blood from a patient can cost as little as $1,000. In contrast, protocols that utilize more invasive sources of stem cells can run as much as $15,000 $35,000. Examples of invasive procedures includebone marrow and adipose tissue extraction. In some cases, hospitalization may be required, which raises costs. The location of a stem cell facility can factor heavily into thecost of the procedure.

Not every cost associated with treatment gets billed to the patient at the time of the procedure. Hidden costs such as reactions to the treatment, graft-versus-host disease, or disability derived from the treatment can all result in more money to the patient, to insurance, or to the government.

For example, in the case of someone with cancer, it frequently isnt viable to harvest the patients own stem cells because they may contain cancerous cells that can reintroduce tumors to the body. Instead, the patient would receive stem cells by transplant. Treatments that involve cells from another person are called allogeneic treatments. The danger here is that the body may see those cells as invaders and attack them via the immune system, a condition known as graft-versus-host disease (GvHD). The body (host) and the introduced stem cells (graft) then battle rather than coexist.

Transplanted cells often face the risk of being rejected by their host; this article discusses the effect of plasma exchange on acute graft vs. host diseasehttps://t.co/cA3nzFntew

Katie Bunde (@kbuns76) May 29, 2018

In addition to making the stem cell treatments less effective or ineffective, GvHD can be deadly. Roughly30 to 60 percent ofhematopoieticstem cell and bone marrow transplantationpatients sufferfrom it, and of those, 50 percent eventually die. The hospital costs associated with it are substantial.

Another hidden cost is the potential to disrupt a system that formerly functioned adequately. The best current example of this isthe case of Doris Tyler, who received bilateral stem cell injections in her eyes from Drs.RobertHalpernand JamieWalraven of Stem Cell Center of Georgia. According to her, while her vision was failing, it was still good enough to perform various tasks, and now it is not. That means the cost increases for her, as well as potential insurance or disability claims (though again, insurance is unlikely to cover the specific consequences of this action).

Because of tight regulations surrounding stem cell procedures performed in the United States, many stem cell treatment providers provide both on-shore (U.S.-based) and offshore (international) treatment options.Depending on where a treatment is received, patients may have to pay travel, lodging,and miscellaneous expenditures.

For example, Regenexx offers treatments at a wide range of U.S. facilities using non-expanded stem cells. However, it also offers a laboratory-expanded treatment option at a site in the Cayman Islands, which can administer higher cell doses to patients by expanding the cells in culture within a laboratory.

Similarly, Okyanos (pronounced Oh key AH nos) offers treatments to patients at its Florida location and provides more involved stem cell procedures at its offshore site inGrand Bahama. It was founded in 2011 and is a stem cell therapy provider specializing in treatments for congestive heart failure (CHF) and other chronic conditions. It is fully licensed under the Bahamas Stem Cell Therapy and Research Act and adheres to U.S. surgical center standards.

Similarly, Celltex is headquartered in Houston, Texas, but offers stem cell treatments in Cancun, Mexico. Celltex specializes in storing a patients mesenchymal stem cells (MSCs) for therapeutic use.

While no hard evidence yet points to stem cell clinics raising their rates as a result of lawsuits, that is a typical response in industries whose products or services the public perceives as a high risk.

An additional danger to stem cell treatment providers,points out Nature, is the reduction of bottom-line profits through former patients winning suits. If clinics have to pay out the money they earned and then some to individuals suing for damages, they may soon become faced with an unviable business model. That is a definite concern for those hoping to leverage these treatments now and in the future.

As with any other area of medicine, patient evaluations of stem cell providers and treatments run the gamut from extremely satisfied to desolately unhappy. Those like Doris Tyler who have lost their eyesight exist at the negative end of the spectrum. However, many others praise stem cell treatments for their power to heal diseases, boost immunity, fight cancer, and more.

For example, BioInformants Founder and President, Cade Hildreth, had a favorable experience with stem cell therapy. Cade had bone marrow-derived stem cells collected and then had them re-injected into the knee to treat a devastating orthopedic injury. Cade was able to reverse pain, swelling, and scarring to reclaim an elite athletic ability.

As of now, this much is clear. There exists enough interest in America and across the world that stem cell providers are continuing to offer a wide range of treatments. Stem cell treatments also offer the potential to reverse diseases that traditionally had to be chronically managed by drugs. Like most medical practices, stem cell treatments will require further testing to reveal merits and faults. Until then, the public will likely continue to pursue services when medical needs arise.

Although the cost of stem cell therapy is pricey, some patients choose to undergo the treatment because it is more economical than enduring the costs associated with chronic diseases.

Although most stem cell therapy providers do not provide FDA-approved procedures, the Food and Drug Administration (FDA) continues to encouragepatients to pursue approved therapies, even if there is a higher associated treatment cost.

Providers rarely post their prices for stem cell treatments in print or digital media because they want patients to understand the benefits of therapy before making a price decision. Additionally, the price of stem cell treatments varies by condition, the number of treatments required, and the complexity of the procedure, factors that can make it difficult for medical providers to provide cost estimates without a diagnostic visit for the patient. However, in many cases, it is not in the patients best interest to make treatment decisions based on the cost of stem cell therapy. The best way to know whether to pursue stem cell therapy is to explore patient outcomes by condition and compare the healing process to other surgical and non-surgical treatment options.

The cost of stem cell therapy is indeed expensive, especially because the procedures are rarely covered by health insurance. However, with the right knowledge and a clear understanding of the treatment process, the risk of undergoing stem cell therapy can be worth it, especially if it removes the requirement for a lifetime of prescription medication. Although stem cell therapy has associated risks, it has improved thousands of lives and will continue to play in a key role in the future of modern medicine.

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Are you seeking a stem cell treatment? If so, we have partnered with GIOSTAR to help you acccess medical guidance and advice.

In alignment with what we believe at BioInformant, GIOSTARs goal is to offer cutting-edge, extensively researched stem cell therapy options designed to rejuvenate and improve a patients quality of life.

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Up Next: Japan to Supply Human Embryonic Stem Cells (hESC) for Clinical Research

Cost Of Stem Cell Therapy And Why Its So Expensive

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Stem Cell Therapy in Thailand – Beike Biotech – Hospitals

By Dr. Matthew Watson

TREATMENT:hRPE stem cells implantation (human Retinal Pigment Epithelial cells, (adult stem cells) by stereotactic brain injection + nutritious stem cell cocktail treatment (intravenous).

START OF TREATMENT:March 6, 2007.

BEFORE THE TREATMENT: Lindas main symptoms were rigidity and stiffness in the left side of her body. She had mild tremors mainly in her left hand and had difficulty grasping small objects or holding things with her fingers. She would drag her left leg while walking and while at rest the

muscles in her leg and tows would contract. During the night her muscles would contract constantly keeping her regularly from having more than few hours sleep. Her muscles were very weak and she would tire very quickly, her posture was stooped and she suffered from a general tenseness and stiffness in her face, neck and back.

Without the affect of the medications she could not turn her neck and should turn her whole body in order to look back. Every morning, before the medications started to influence, it was difficult getting dressed, getting out of bed or taking a shower.

Before the treatment Linda took her medications every 2-3 hours (Contam 250mg x 8 times a day). One hour after taking the medications Lindas symptoms were hardly noticed, but the medications influence wear out quickly and Lindas every activity was dependant on her next dose of medications.

During the last few years Lindas short term memory was affected up to a level that she quit her job in human resources. Her hand writing was affected too even after taking the medications, it was still very scratchy and hard to read.

Linda also suffered from general anxiety and depression.

AFTER THE TREATMENT:

Lindas first notable change after the surgery was a full night sleep - the first one in 5 years. Within 5 weeks after the stem cell implantation most of Lindas symptoms were gradually gone. Her fingers got their flexibility back and the tremors were gone she could now grasp things, open a door and articulate more precise movements with her fingers.

The cramps in her leg were gone and she stopped dragging her left leg.

I dont need to think anymore about every movement, as I did before she says.

Her muscle tension was significantly reduced, she felt more relaxed and stronger than before.

Her posture became more open and she could now turn her neck more easily. Before leaving the hospital Linda still had some weakness in her muscles but she felt that she is getting stronger every day.

Linda also noticed that her sense of smell and taste that were greatly weakened during the last years were coming back.

A major change in her quality of life was that now her symptoms were unnoticeable with almost half the dosage of the medications she used to take before. Linda is now taking medications 4 times a day (Sinemet 200mg X4 times a day) instead of 8 times of double dosage that she used to take before the treatment.

I was a watch keeper, I used to watch at the clock all the time, I stopped swimming riding bicycle and other activities because I never knew when the medications affect will wear out she says.

Linda hopes that her medications could be gradually reduced even more, and she will keep a close contact with her doctors in China in order to follow up with her condition.

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Stem Cell Therapy for ALS Patients

By Dr. Matthew Watson

Learn about what stem cells are, why they are important and how they are going to revolutionize healing and medical care in Canada.

Not all conditions are effectively treated by PRP injections or stem cell therapy, and with ongoing clinical trials its important to realize what stem cells can and cannot help with. Weve built a comprehensive list of the different types of conditions that stem cell therapy shows promise for, however if you dont find it listed wed recommend checking outDanish health website Doc24.dk. Regular maintenance of health is key to making sure long-term issues dont arise as we age, and part of that is a rich, balanced diet and careful supplementation.

Research on human embryos in general, and stem cell research in particular, has been the subject of public debate in Canada since the late 1980s. In 2002, the Canadian Institute of Health Research (CIHR) issued guidelines for research on human embryonic stem cell lines, which have been revised and reissued several times since 2005 (most recently in 2007). These guidelines regulate the allocation of state funds in the field of research on human embryonic stem cells and concern both the handling of existing stem cell lines and the establishment of new stem cell lines.

The guidelines specify a number of important conditions that must be fulfilled in order for research projects to be eligible for funding. These include, but are not limited to:

The Stem Cell Oversight Committee (SCOC) was set up to ensure that research projects comply with the provisions of the Directive and to address the complex ethical issues surrounding research projects. Any project applying for government funding in the field of stem cell research must first be positively evaluated by the SCOC.

In addition to the regulation of state funding, the Assisted Human Reproduction Act came into force in 2004, which broadly regulates the field of reproductive medicine. Unlike the guidelines of the CIHR, it is not merely a guideline for state funding of certain research activities, but a law that places certain activities under state control and generally prohibits others. Research on human embryos is one of the controlled activities of the Assisted Human Reproduction Act. According to 8 Para. 3, the approval according to 10 Para. 2 requires the consent of the donor after clarification of the intended use. The Assisted Human Reproduction Agency of Canada (AHRAC), established by law, is responsible for granting authorisations and monitoring research activities.

The extraction of ES cells also falls under this section and is therefore permitted in Canada. The use of in vitro embryos for research purposes, including the derivation of stem cells, is subject to the following conditions under the Assisted Human Reproduction Act:

The production of a human clone is prohibited according to 5 a Assisted Human Reproduction Act. This provision also includes so-called therapeutic cloning by nuclear transfer. According to 5 b, the creation of embryos for purposes other than the creation of a human being or the improvement of artificial reproduction procedures is also prohibited. The law does not apply to the handling of already established human embryonic stem cell lines.

The CBC news network and other media responded to Twitter posts and a YouTube live video about unapproved treatments that lately came up. Patients that suffer from chronic pain or disease could benefit from stem-cell therapies. Canadians who have been treated more open by their federal and other regulatory laws about unlicensed stem cell therapies are asking for the legalization or this procedure.

A new company now made it their mission to offer direct-to-customer opportunities for trainees and people in general which can mean a big advantage for a patient. Unproven stories about this training in marketing and science services are offering support for approved stem-cell professionals.

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Stem Cell Therapy for ALS Patients

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Stem Cell Therapy Has a Lot to OfferIt Just May Take Some …

By Dr. Matthew Watson

Stem Cell Therapy Has a Lot to OfferIt Just May Take Some Time to Get There

By: Ashwini Nagappan

In conversation with the New York Times, Dr. Shinya Yamanaka, the director of Kyoto Universitys Center for iPS Cell Research and Application and researcher at the Gladstone Institutes, illuminates the complexities and future of stem-cell research. Yamanaka was jointly awarded the 2012 Nobel Prize in Physiology or Medicine for reconfiguring adult cells back to their pluripotent states. These induced pluripotent stem cells, or iPS cells, have been used as treatments for conditions such as macular degeneration.

However, Yamanaka mentions that these treatments are temporarily suspended because of the possibility of mutations developing in the patients iPS cells. Cancer could be a potential outcome because the production of iPS cells increases the chance of mutations. Researchers are rigorously testing to make sure that there are no cancer-causing mutations and that the cells function as they should. In order to be certain that these cells are safe, they are transplantedinto mice or rats for about a year. Yamanaka approximates that only 100 lines would be needed to cover the Japanese population and 200 lines for the US population.

Yamanaka acknowledges that the potentialfor stem cells may have been too eagerlyanticipated as they can only remedy the small portion of diseases that are caused by a single cell failure such as heart failure. Stem cell therapy cannot target diseases caused by multiple types of cell failures. He mentions an alternative to iPS known as direct cellular reprogramming, which would be beneficial if the patient in question was elderly instead of a younger person, and if the area targeted was larger instead of a small wound.

In essence, Yamanaka highlights the need for an ethical consensus in order to understand how to move forward with advancing stem cell technology. Further, iPS cells are fairly young they are only tenyears old. For patients to be able to receive these treatments requires money and time. In the mean time, Yamanaka recommends arrivingat an ethical consensus onthe use of stem cells.

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FUJIFILM Cellular Dynamics to Establish New Production …

By Dr. Matthew Watson

- Accelerating the practical application of treatments that apply iPS cells towards the early industrialization of regenerative medicine- Making the high quality and highly efficient production of iPS cells a reality

January 4, 2019FUJIFILM Cellular Dynamics, Inc.

FUJIFILM Cellular Dynamics, Inc. (FCDI), a US subsidiary of FUJIFILM Corporation (President: Kenji Sukeno) and a leader in the development and manufacture of human induced pluripotent stem (iPS) cells and tissue-specific cells differentiated from iPS cells, will establish a new cGMP-compliant* production facility with an investment of about 21 million US dollars in order to enhance its production of iPS cells for cell therapy. The facility is scheduled to begin operations during fiscal year ending March 2020.FCDI will use the iPS cells produced at this facility to accelerate development of its regenerative medicine products. In addition, by also conducting contract development and manufacturing of iPS cells and iPS cell-derived differentiated cells, it will expand its business and scale to the industrial stage.

Regenerative medicine is drawing interest as a solution for unmet medical needs. There are high expectations for the practical application of treatments that utilize iPS cells, as these cells possess totipotency and the capacity for infinite reproduction, making it possible to produce a large volume of diverse cells. To fulfill the promise of cell therapy, sophisticated techniques and know-how are required to culture, induce differentiation in, and control the quality of cells.

FCDI will be establishing a new production facility equipped with cell culture facilities appropriate for the production of a large volume of cells, as well as culture facilities appropriate for small-scale, diverse production, and a system capable of highly precise cell quality analyses. By also harnessing world-class technologies for the initialization and induction of differentiation in iPS cells and Fujifilm's advanced engineering technology and image analysis technology, the facility will be capable of efficiently producing high-quality iPS cells.Going forward, FCDI will use the high-quality iPS cells produced at this facility to accelerate the development of regenerative medicine products in the areas of age-related macular degeneration, retinitis pigmentosa, Parkinson's disease, heart diseases, and cancer. FCDI will also contribute to the realization and spread of treatments that utilize iPS cells by widely conducting the contract development and manufacturing of iPS cells and iPS cell-derived differentiated cells.

Currently, FCDI provides iPS cells and iPS cell-derived differentiated cells to public institutions, major pharmaceutical companies, and academia including the California Institute for Regenerative Medicine** and the National Heart, Lung, and Blood Institute*** while accelerating the development of its regenerative medicine products. FCDI will continue to harness its accumulated data, technologies, and know-how related to iPS cells, working together with academic institutions and corporations around the world and utilize the technologies and know-how of Fujifilm group companies including Fujifilm, Japan Tissue Engineering Co., Ltd., FUJIFILM Wako Pure Chemical Corporation, and Irvine Scientific Sales Company, Inc. to further expand its iPS cell-based business and contribute to the elevation of regenerative medicine business to the industrial stage.

Overview of the New Facility

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CTERP International Conference – 2018: About

By Dr. Matthew Watson

CTERP INTERNATIONAL CONFERENCEApril 11-13, 2018Moscow, Russia

In recent years there have been rapid advances in applying the discoveries in cell technologies field into medical practice. Cell technologies are progressing as the result of multidisciplinary effort of scientists, clinicians and businessmen,with clinical applications of manipulated stem cells combining developments in transplantation and gene therapy.Challenges address not only thetechnology itself but also compliancewith safety and regulatory requirements.

The Conference will provide a platform for scientists from basic and applied cell biology fields, practical doctors, and biotech companies to meet and share their experience, to discuss the research associated with developing biomedical clinical products and translating this research into novel clinical applications, challenges of such translational efforts and foundation of bioclusters assisting further developments in cell technology.

The official language of the conference is English.

Conference materials will be published in the Russian Journal of Developmental Biology.

Please download your abstracts in accordance with the journal guidelines (english, russian) for authors provided on their website.

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