31-Oct-2019

Ingredients

Since ancient times medicinal plants have been used for their health beneficial properties, to protect and promote the skin and for treatment of various diseases.

Plantshave an enormous capacity to produce complex chemical molecules with bioactive properties.The market for botanicals is expanding with an increasing demand for plant bioactives. It is therefore important to produce the plant raw material a sustainable way. Often traditional production does not support this.

Plant cell cultivation enables sustainable production of high-quality plant raw material. Based on this technique it is possible to target and enrich specific cell types, such as plant stem cells.

Since the cultivation takes place in a clean and controlled environment, the produced plant raw material is free from adulteration, pollution, pesticides and herbicides. Besides cell enrichment, it is further possible to increase the production of bioactives through the MET (Metabolic Enhancement Technology).

For bioactives it is also important to consider their availability in the final product, otherwise their beneficial properties will not be available to our cells. Some bioactives are not available in dry cells even when these are grinded.

This can be due to that they are tightly bound to a cell structure, such as the cell wall. However, these can be made accessible through extraction where these actives are released from their bound position.

The extract with the highest quality and health beneficial properties are high in concentration and standardised to selected actives or group of molecules. This way it is possible to ensure that the extract is always the same in terms of properties and efficacy.

In vitro Plant-tech develops and produces high quality plant raw material and extracts using the plant cell cultivation technology. We are proud of our green and sustainable production platform, producing superior products with compassion for nature.

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Plant bioactives, combining tradition with technology - Cosmetics Business

Skin Stem Cells Comments Off on Plant bioactives, combining tradition with technology – Cosmetics Business | November 1st, 2019

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Newswise Three researchers at theEli and Edythe Broad Center of Regenerative Medicine and Stem Cell Research at UCLAhave received awards totaling more than $18 million from the California Institute for Regenerative Medicine, the states stem cell agency.

The recipients are Dr. Sophie Deng, professor of ophthalmology at the UCLA Stein Eye Institute;Yvonne Chen, a UCLA associate professor of microbiology, immunology and molecular genetics; and Dr. Caroline Kuo, a UCLA assistant clinical professor of pediatrics. The awards were announced at a CIRM meeting today.

Dengs four-year, $10.3 million award will fund a clinical trial for a blinding eye condition called limbal stem cell deficiency. Limbal stem cells are specialized stem cells in eye tissue that help maintain the health of the cornea. Because of genetic defects or injuries caused by infections, burns, surgeries or other factors, some people do not have enough limbal stem cells, which results in pain, corneal scarring and blindness.

The approach she is testing involves extracting a small number of limbal stem cells from a persons eye, multiplying them in a lab, and then transplanting them back into the eye, where they could regenerate the cornea and restore vision. The research will be conducted in collaboration with theUCLAUCI Alpha Stem Cell Clinic, a partnership between UCLA and UC Irvine.

The grants awarded to Chen and Kuo are for projects that are heading toward the FDAs investigational new drug application process, which is required by the agency before a phase 1 clinical trial the stage of testing that focuses on a treatments safety.

Chens two-year, $3.2 million award will fund efforts to create a more effectiveCAR T cell therapyfor multiple myeloma, a blood cancer that affects white blood cells. The research will evaluate a specialized form of CAR T therapy that simultaneously targets two markers, BCMA and CS1, commonly found on multiple myeloma cells. CAR T therapies that target BCMA alone have been effective in clinical trials, but the presence of BCMA on multiple myeloma cells is not uniform.

Previous research has shown that the marker CS1 is present in around 90% of multiple myeloma cells. A CAR T therapy that targets both markers could potentially help more patients and reduce the likelihood of a cancer relapse.

Kuos 2 1/2-year, $4.9 million award, will support the development of a stem cell gene therapy for a deadly immunodeficiency called X-linked hyper IgM syndrome, or XHIM.

The syndrome, which is caused by a mutation in the CD40LG gene, results in invasive infections of the liver, gastrointestinal tract and lungs. Currently, the only potential cure is a bone marrow transplant from a matched donor, which carries life-threatening risks and is often less effective for XHIM patients than patients with other forms of immune deficiency. Even with current treatments, only 30% of people with the syndrome live to age 30.

Kuo will evaluate a stem cell gene therapy that corrects the genetic mutation that causes XHIM. After removing blood-forming stem cells from a person with the syndrome, the therapy would use a genetic engineering technique called CRISPR to insert a correct copy of the affected gene into the DNA of the stem cells. The corrected blood-forming stem cells would be infused back into the patient, where they could regenerate a healthy immune system.

She will collaborate with Dr. Donald Kohn, a UCLA distinguished professor of microbiology, immunology and molecular genetics who has successfully treated two other immune deficiencies bubble baby disease and X-linked chronic granulomatous disease with a similar therapy.

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Three UCLA scientists receive grants totaling more than $18 million - Newswise

Bone Marrow Stem Cells Comments Off on Three UCLA scientists receive grants totaling more than $18 million – Newswise | October 31st, 2019

Ezer Mizion, the worlds largest Jewish bone marrow registry, will host its Evening of Heroes for the Teaneck, Bergenfield, and New Milford communities on Saturday, November 9, at Congregation Keter Torah in Teaneck.

The evening begins with a musical Havdalah and mini-concert by the chasidic superstar Shulem Lemmer, the first chasidic singer to sign with Universal Records. Then Ezer Mizion will introduce IDF heroes who defend the State of Israel and have saved lives with their stem cells.

A stem cell recipient will recount the day he received a call letting him know that Ezer Mizion had identified a stem cell match for him a match that saved his life. Bret Stephens, a New York Times Pulitzer Prize-winning columnist, and Nachum Segal will give a fireside chat about innovations from Israel, including the export of more than 60 percent of Ezer Mizions stem cell transplants.

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There will be a swabbing station for people who meet the basic criteria for donations. Israeli wines and shuk foods will be served.

The program aims to bring awareness of the organizations role in saving hundreds of lives around the world every year with its growing bone marrow registry. It has more than 1 million potential stem cell donors, and more than 550,000 of these donors are from the IDF. There is no cost to attend the adults-only evening; RSVPs are requested. For more information, go to eveningofheroes.com; email Ezer Mizions national director of development, Ryan Hyman, at ryan@ezermizionusa.org or call him at (718) 853-8400, ext. 109.

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Ezer Mizion's Evening of Heroes is November 9 in Teaneck - The Jewish Standard

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The National Institutes of Health (NIH) and the Bill & Melinda Gates Foundation will each invest $100 million over the next four years to speed the development of affordable gene therapies for sickle cell disease (SCD) and the human immunodeficiency virus (HIV) on a global scale.

This unprecedented collaboration focuses from the get-go on access, scalability and affordability of advanced gene-based strategies for sickle cell disease and HIV to make sure everybody, everywhere has the opportunity to be cured, not just those in high-income countries, said NIH Director Francis S. Collins, MD, PhD.

Seventy-five percent of babies born with SCD live in sub-Saharan Africa. It is hoped that experimental gene therapies would advance to clinical trials in the United States and relevant African countries within the next seven to 10 years, and that safe, effective, and inexpensive gene therapies be made available globally, including in low-resource settings where the cost and complexity of these therapies make them inaccessible to many.

In recent years, gene-based treatments have been groundbreaking for rare genetic disorders and infectious diseases, Trevor Mundel, MD, PhD, president of the global health program at the Bill & Melinda Gates Foundation said in a news release.

While these treatments are exciting, people in low- and middle-income countries do not have access to these breakthroughs. By working with the NIH and scientists across Africa, we aim to ensure these approaches will improve the lives of those most in need and bring the incredible promise of gene-based treatments to the world of public health, he added.

Hemoglobin is the protein in red blood cells that binds oxygen, allowing oxygen to be transported around the body. Mutations in the HBBgene, which encodes a component of hemoglobin, result in the formation of sickle hemoglobin that causes sickle cell anemia.

Currently, gene therapies for SCD involves altering the patients own hematopoietic stem cells (bone marrow cells that divide and specialize to produce blood cells including red blood cells). Genes are introduced into the cells using a modified, harmless virus (known as a viral vector). The cells are then transplanted back into the patient where they will produce healthy red blood cells. Gene therapy has an advantage over a bone marrow transplant, as it circumvents the complications associated with a bone marrow donation.

The first goal of the collaboration between the NIH and the Gates Foundation is to develop an easy-to-administer gene-based intervention to correct the mutations in the HBBgene or deliver a functional gene that will promote the production of normal levels of hemoglobin without the need to extract cells from patients and modify them in the lab before introducing the cells back. However, this strategy, known as in vivotreatment, requires the advancement of more efficient delivery systems that can deliver the gene therapy specifically to hematopoietic stem cells.

A second goal of the collaboration will be to work together with African partners and bring potential therapies to clinical trials.

Further research is required to understand the burden of SCD in sub-Saharan Africa and to screen newborns at high risk for the disease, a task that the National Heart, Lung and Blood Institute (NHLBI) has started to tackle by building the necessary infrastructure for clinical research.

The NIH and the Gates Foundation will help boost this infrastructure to allow point-of-care screening (for example, when infants receive vaccinations), and to initiate a standard of care. This will occur outside of the official collaboration.

Our excitement around this partnership rests not only in its ability to leverage the expertise in two organizations to reduce childhood mortality rates in low-resource countries, but to bring curative therapies for sickle cell disease and HIV to communities that have been severely burdened by these diseases for generations, said Gary H. Gibbons, MD, director of the NHLBI.

A persons health should not be limited by their geographic location, whether rural America or sub-Saharan Africa; harnessing the power of science is needed to transcend borders to improve health for all, he added.

Matshidiso Rebecca Moeti, the regional director for Africa at the World Health Organization said, We are losing too much of Africas future to sickle cell disease and HIV.

Beating these diseases will take new thinking and long-term commitment. Im very pleased to see the innovative collaboration announced today, which has a chance to help tackle two of Africas greatest public health challenges, Moeti added.

Patricia holds her Ph.D. in Cell Biology from University Nova de Lisboa, and has served as an author on several research projects and fellowships, as well as major grant applications for European Agencies. She also served as a PhD student research assistant in the Laboratory of Doctor David A. Fidock, Department of Microbiology & Immunology, Columbia University, New York.

Total Posts: 94

Margarida graduated with a BS in Health Sciences from the University of Lisbon and a MSc in Biotechnology from Instituto Superior Tcnico (IST-UL). She worked as a molecular biologist research associate at a Cambridge UK-based biotech company that discovers and develops therapeutic, fully human monoclonal antibodies.

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SCD, HIV Gene Therapy Efforts Get $200M from NIH, Gates Foundation - Sickle Cell Anemia News

Bone Marrow Stem Cells Comments Off on SCD, HIV Gene Therapy Efforts Get $200M from NIH, Gates Foundation – Sickle Cell Anemia News | October 31st, 2019

TEL AVIV, Israel, Oct. 31, 2019 /PRNewswire/ -- BioLineRx Ltd. (NASDAQ: BLRX) (TASE: BLRX), a clinical-stage biopharmaceutical company focused on oncology, announced today that it will deliver the following poster presentations at the Society for Immunotherapy of Cancer(SITC) 34th Annual Meeting to take place November 6-10, 2019 at the Gaylord National Hotel & Convention Center in Baltimore, Maryland:

About BL-8040

BL-8040 is a short synthetic peptide that functions as a high-affinity best-in-class antagonist for CXCR4, a chemokine receptor over-expressed in many human cancers, where it has been shown to be correlated with poor prognosis, and plays a key role in tumor growth, invasion, angiogenesis, metastasis and therapeutic resistance. CXCR4 is also directly involved in the homing and retention of hematopoietic stem cells (HSCs) and various hematological malignant cells in the bone marrow.

In a number of clinical and pre-clinical studies, BL-8040 has shown a critical role in immune cell trafficking, tumor infiltration by immune effector T cells and reduction in immunosuppressive cells within the tumor niche, turning "cold" tumors, such as pancreatic cancer, into "hot" tumors (i.e., sensitizing them to immune check point inhibitors). BL-8040-mediated inhibition of the CXCR4-CXCL12 (SDF-1) axis has also shown robust mobilization of HSCs for transplantation in hematological malignancies.

BL-8040 was licensed by BioLineRx from Biokine Therapeutics and was previously developed under the name BKT-140.

About BioLineRx

BioLineRx is a clinical-stage biopharmaceutical company focused on multiple oncology indications. The Company'slead program, BL-8040, is a cancer therapy platform currently being evaluated in a Phase 2a study in pancreatic cancer in combination with KEYTRUDA and chemotherapy under a collaboration agreement with MSD. BL-8040 is also being evaluated in a Phase 2b study in consolidation AML and a Phase 3 study in stem cell mobilization for autologous bone-marrow transplantation. In addition, the Company has an ongoing collaboration agreement with Genentech, a member of the Roche Group, evaluating BL-8040 in combination with Genentech's atezolizumab in two Phase 1b/2 solid tumor studies.

BioLineRx is developing a second oncology program, AGI-134, an immunotherapy treatment for multiple solid tumors that is currently being evaluated in a Phase 1/2a study.

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

Various statements in this release concerning BioLineRx's future expectations constitute "forward-looking statements" within the meaning of the Private Securities Litigation Reform Act of 1995. These statements include words such as "may," "expects," "anticipates," "believes," and "intends," and describe opinions about future events. These forward-looking statements involve known and unknown risks and uncertainties that may cause the actual results, performance or achievements of BioLineRx to be materially different from any future results, performance or achievements expressed or implied by such forward-looking statements. Some of these risks are: changes in relationships with collaborators; the impact of competitive products and technological changes; risks relating to the development of new products; and the ability to implement technological improvements. These and other factors are more fully discussed in the "Risk Factors" section of BioLineRx's most recent annual report on Form 20-F filed with the Securities and Exchange Commission on March 28, 2019. In addition, any forward-looking statements represent BioLineRx's views only as of the date of this release and should not be relied upon as representing its views as of any subsequent date. BioLineRx does not assume any obligation to update any forward-looking statements unless required by law.

Contact:Tim McCarthyLifeSci Advisors, LLC+1-212-915-2564tim@lifesciadvisors.com

or

Tsipi HaitovskyPublic Relations+972-52-598-9892tsipihai5@gmail.com

View original content:http://www.prnewswire.com/news-releases/biolinerx-to-present-two-posters-at-the-society-for-immunotherapy-of-cancer-sitc-2019-300948943.html

SOURCE BioLineRx Ltd.

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BioLineRx to Present Two Posters at the Society for Immunotherapy of Cancer (SITC) 2019 - P&T Community

Bone Marrow Stem Cells Comments Off on BioLineRx to Present Two Posters at the Society for Immunotherapy of Cancer (SITC) 2019 – P&T Community | October 31st, 2019

Zion Market Research published a new 110+ pages industry researchCell Isolation Market by Product (Instruments and Consumables), by Cell Type (Animal and Human), by Cell Source (Adipose Tissue, Embryonic/Cord Blood Stem Cells, and Bone Marrow), by Technique (Surface Marker-Based Cell Isolation, Centrifugation-Based Cell Isolation, and Filtration-Based Cell Isolation), by Application (Cancer Research, Biomolecule Isolation, Tissue Regeneration & Regenerative Medicine, Stem Cell Research, In Vitro Diagnostics, and Others), and By End-User (Hospitals & Diagnostic Laboratories, Research Laboratories & Institutes, Biotechnology & Biopharmaceutical Companies, and Others): Global Industry Perspective, Comprehensive Analysis, and Forecast, 20182025.

TheGlobal Cell Isolation Market Is Expected To Reach Around USD 15.16 Billion By 2025complete outline is crystal clear penned down in the GlobalCell Isolation Marketresearch report such that not only an unskilled individual but also a professional can easily extrapolate the entire Cell Isolation Market within a few seconds.The research study covers research data which makes the document a handy resource for managers, analysts, industry experts, and other key people get ready-to-access and self-analyzed study along with TOC, graphs and tables to help understand the market size, share, trends, growth drivers and market opportunities and challenges.

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The Cell Isolation Market research report covers major industry player profiles that include:

Becton, Dickinson, and Company, Thermo Fisher Scientific, Inc., Merck KGaA, Beckman Coulter Inc., Terumo BCT, Bio-Rad Laboratories, Inc.

This report employs the SWOT analysis technique for the assessment of the development of the most remarkable market players. It additionally considers the latest upgrades while assessing the development of leading market players. Moreover, in the global Cell Isolation Market report, the key product categories of the global Cell Isolation Market are included. The report similarly demonstrates supportive data related to the dominant players in the market, for instance, product offerings, revenue, segmentation, and business synopsis. The global Cell Isolation Market is as well analyzed on the basis of numerous regions.

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Global Cell Isolation Market: Regional Analysis

To understand the competitive landscape in the market, an analysis of Porters five forces model for the market has also been included. The study encompasses a market attractiveness analysis, wherein all segments are benchmarked based on their market size, growth rate, and general attractiveness. This report is prepared using data sourced from in-house databases, secondary and primary research team of industry experts.

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The report answers important questions that companies may have when operating in the Global Cell Isolation Market. Some of the questions are given below:

What is the current CAGR of the Global Cell Isolation Market?

Which product is expected to show the highest market growth?

Which application is projected to gain a lions share of the Global Cell Isolation Market?

Which region is foretold to create the most number of opportunities in the Global Cell Isolation Market?

Will there be any changes in market competition during the forecast period?

Which are the top players currently operating in the global market?

How will the market situation change in the coming years?

What are the common business tactics adopted by players?

What is the growth outlook of the Global Cell Isolation Market?

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Cell Isolation Market ||Becton, Dickinson, and Company, Thermo Fisher Scientific, Inc., Merck KGaA - Industry News Info

Bone Marrow Stem Cells Comments Off on Cell Isolation Market ||Becton, Dickinson, and Company, Thermo Fisher Scientific, Inc., Merck KGaA – Industry News Info | October 31st, 2019

Cell Harvesting MarketReport has newly added to its massive repository. Different industry-specific methods have been used for analyzing the market carefully. The informative data has been inspected through primary and secondary research techniques. The global Cell Harvesting market has been analyzed by focusing on different verticals of the businesses such as market trends, regional outlook, competitive landscape, key players, business approaches, technologies, and standard operating procedures.An exclusiveCell Harvesting Marketresearch report contains a brief on those trends which may enable the companies operating into know to strategize and the current sector to their small enterprise expansion. The investigation report analyses the market size, industry share, growth, key sections, CAGR, and drivers.

Top Companies in the GlobalCell HarvestingMarket:PerkinElmer (US), Brandel (US), TOMTEC (US), Pall Corporation (Danaher), Connectorate (Switzerland), Scinomix (US), ADSTEC (Japan), Sartorius, Terumo Corporation.

Cell harvesting usually for use in cancer or other treatment. Usually the cells are removed from the patients own bone marrow. Stem cells can be harvested from the blood or bone marrow. Umbilical cords have been saved as a future source of stem cells for the baby.

https://www.marketinsightsreports.com/reports/05161234118/global-cell-harvesting-market-size-status-and-forecast-2019-2025/inquiry?mode=46

The market engineering comprises the structured, systematic and theoretically founded procedure of analyzing, designing, introducing and also quality assuring of markets as well as their legal framework regarding simultaneously their market mechanisms and trading rules, systems, platforms and media, and their business models.

This report segments the globalCell HarvestingMarket on the basis ofTypesare:ManualAutomated

On The basis OfApplication,the GlobalCell HarvestingMarket is Segmented into:BiopharmaceuticalStem Cell Research

https://www.marketinsightsreports.com/reports/05161234118/global-cell-harvesting-market-size-status-and-forecast-2019-2025/discount?mode=46

Geographically, this report is segmented into several key Regions, with production, consumption, revenue (million USD), and market share and growth rate ofCell HarvestingMarketthese regions, from 2018 to 2025 (forecast), covering

North America,Europe,China,Japan, Southeast Asia, India, North America(USA, Canada and Mexico)Europe(Germany, France, UK, Russia and Italy)Asia-Pacific(China, Japan, Korea, India and Southeast

-Comprehensive assessment of all opportunities and risk in the market. Cell Harvesting market recent innovations and major events.-Detailed study of business strategies for growth of the market-leading players.-Conclusive study about the growth plot of Cell Harvesting market for forthcoming years.-In-depth understanding of market-particular drivers, constraints and major micro markets.-Favourable impression inside vital technological and market latest trends striking the market.

https://www.marketinsightsreports.com/reports/05161234118/global-cell-harvesting-market-size-status-and-forecast-2019-2025?mode=46

MarketInsightsReportsprovides syndicated market research on industry verticals including Healthcare, Information and Communication Technology (ICT), Technology and Media, Chemicals, Materials, Energy, Heavy Industry, etc.MarketInsightsReportsprovides global and regional market intelligence coverage, a 360-degree market view which includes statistical forecasts, competitive landscape, detailed segmentation, key trends, and strategic recommendations.

Irfan Tamboli (Head of Sales) Market Insights ReportsPhone: + 1704 266 3234 | +91-750-707-8687sales@marketinsightsreports.com|irfan@marketinsightsreports.com

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Cell Harvesting Market Global Strategies and Insight driven transformation 2019-2024 - The Chicago Sentinel

Bone Marrow Stem Cells Comments Off on Cell Harvesting Market Global Strategies and Insight driven transformation 2019-2024 – The Chicago Sentinel | October 31st, 2019

Hair care is being taken to the next level, by utilising recombinant DNA technology to restore one's confidence and crowning glory.

Such hair restoration products or treatments are made using recombinant DNA - or DNA cloning - where selected pieces of DNA from different organisms are combined to construct artificial DNA.

At Ageless Medi-Aesthetics, its latest AnteAge MD Hair Treatment is a non-invasive procedure that involves applying the AnteAge MD Hair Growth Factor solution or serum - made from potent recombinant growth factors and cytokines - onto skin prepared with microneedling.

Dr Lam Bee Lan, director of Ageless Medi-Aesthetics, told The New Paper: "Recombinant DNA technology is more efficient in producing large amounts of artificial messenger proteins effective for skin and hair renewal compared with stem cells derived from plants."

Methods of hair restoration are often divided into two broad categories - invasive techniques and topical and/or oral solutions. They can either be expensive or linked to side effects such as erectile dysfunction, ejaculatory dysfunction and loss of libido.

But Dr Lam cautioned that before treatments are prescribed, patients must consult with a physician to ascertain if they are suitable for them.

"Treatments based on recombinant DNA technology should be worked in as a first-line treatment when you start to experience more hair loss than usual, or as part of a regular routine in maintaining a full head of hair.

"For more severe hair loss, patients should consider a hair transplant," she said.

While there are minimal side effects such as occasional soreness and redness that will resolve within one to two hours, Dr Lam noted that most patients will experience slowing down of hair loss after the first session, while new hair will grow after the second session.

Home-grown scalp specialist PHS Hairscience has also explored stem cell technology and cell signalling technology since 2014 to treat hair loss or greying hair on the cellular level.

Ms Anita Wong, its chief executive and founder, told TNP: "As the body ages or changes due to reasons such as stress or lifestyle choices, cell functions can deteriorate, and cell activity that directly impacts new hair growth or melanin (hair pigment) production becomes less than optimal."

PHS Hairscience's marquee treatment, Miracle Stem Cell Solution, leverages on stem cell science and cell signalling to reactivate dormant follicle cells to promote hair growth. At $297 a session, it can be complemented with the FEM/HOM Thickening range of products.

She said: "These active botanical stem cells also work to increase the life span of hair follicles so your hair can remain in the anagen (growth) phase of the hair growth cycle for a longer period of time.

"Keeping the hair in this growth phase will maximise the length and thickness of new hair, as well as stop the existing strands from shedding."

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Restore your crowning glory with recombinant DNA tech - The New Paper

Skin Stem Cells Comments Off on Restore your crowning glory with recombinant DNA tech – The New Paper | October 31st, 2019

The question remains unanswered as to whether a peer-to-peer collaborative model will prosper where medtech companies that are in some instances one step ahead of big pharma in terms of drug development are happy to be a third- party provider to big pharma that have the budgets and networks to truly deliver the regenerative medicine revolution.

In a recent document published by the UK government in response to the Regenerative Medicine Inquiry by the House of Commons Science and Technology Committee, policymakers stressed the importance of commercialising new therapies to meet the changing needs of the health sector.

In the UK, the Regenerative Medicine Expert Group (RMEG) has been tasked with developing an NHS regenerative medicine strategy to ensure the NHS is fully prepared to deliver innovative treatment and that regulations support and not hinder its delivery.

The Cell and Gene Therapy Catapult is also continuing to work to bridge the gap between translational research and commercialisation.

However, for the UK to be well-positioned to offer safe and effective regenerative therapies, a strategy is needed that covers the whole value chain from academic research, commercial development and clinical application.

The effect of Brexit on the UKs regenerative medicine sector remains unclear, but the UK has the opportunity to develop an independent framework outside the EU regulatory system to accelerate the development of new therapies and its economic potential while upholding the highest patient safety standards.

In any case, EU and UK regulators need to prioritise the standardisation of regulations governing manufacturing, quality control and the supply chain to keep up with advancements made by the FDA in the US.

Establishing an efficient supply chain for regenerative medicine

The promise of regenerative medicine requires an innovative look at the complete product life cycle, including the development of an efficient distribution network.

Once these novel drugs become mainstream, the entire healthcare ecosystem will have to adapt. Regulatory approval for any drug relies on it safely and successfully fulfilling its medical intent.

As such, information about supply chain management needs to be submitted to the regulator after the completion of phase 3 clinical trials, including packaging, labelling, storage and distribution.

The clinical supply chains required to deliver these therapies are arguably the most complex the industry has seen so far. Regenerative medicine is either personalised or matched to the donor-recipient. They are also highly sensitive to exogenous factors like time and temperature.

Advanced IT solutions and monitoring systems are being developed and employed to ensure end-to-end traceability. These are giving clinicians access to view the progress of therapies and their distribution in real-time and allow users to automatically schedule or amend material collections in line with manufacturing capacity, helping to keep the supply chain as agile as possible.

The live tissues and cells which form the basis of regenerative medicine products are highly sensitive and some have a shelf life of no more than a few hours.

Therefore, materials need to be transported from the site of harvest to manufacturing facilities, and from manufacturing facilities to medical institutions under strictly controlled conditions, within certain times and temperatures, according to cell and tissue requirements.

Temperature-controlled logistics solutions are vital to ensure a safe, effective and financially viable supply chain network for these high-value shipments. Cryopreservation is one technique increasingly being used to deliver medicines at optimum temperature using vapour phase nitrogen; however, many clinical settings remain ill-equipped to handle such equipment.

On-site production is an alternative manufacturing arrangement, particularly for autologous products which are derived from a patients own cells.

However, this throws up a number of compliance and infrastructure challenges, as the hospital would need to comply with a host of regulations including installing a Good Manufacturing Practice (GMP)-licensed clean room.

As a first-generation technology, stakeholders will have a greater tolerance for higher pricing... but only for a limited time period. By streamlining the currently very expensive manufacturing process and improving supply chain management, yields will automatically get larger and costs will slowly come down.

While there are many challenges in the road ahead, 2019 certainly appears to be the start of regenerative medicines move to the big time.

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Delivering the promise of regenerative medicine - PMLiVE

Skin Stem Cells Comments Off on Delivering the promise of regenerative medicine – PMLiVE | October 30th, 2019

Stem Cell Therapy Market research analysis and insights displayed in this report are very thoughtful for the businesses to make enhanced decisions, to build up better strategies about production, marketing, sales and promotion of a particular product. Stem Cell Therapy market report also takes into consideration several major factors such as revenue, cost, gross and gross margin while analysing market data. Various markets at local, regional and international level are thought of in this Stem Cell Therapy report. All this helps in extending their reach towards the success. The use of advanced tools and techniques applied for this report makes it the premium in the class. By understanding clients needs precisely, this report merges business and product information for the sustainable growth in the market. Geron Corporation, Vericel Corporation, Pluristem Therapeutics, Cytori Therapeutics, Fate Therapeutics are some players grooming the market.

Stem Cell Therapy Market is expected to reach USD 15.63 billion by 2025, from USD 7.72 billion in 2017 growing at a CAGR of 9.2% during the forecast period of 2018 to 2025. The Stem Cell Therapy market report contains data for historic year 2016, the base year of calculation is 2017 and the forecast period is 2018 to 2025 (Updated values listed in sample report).

Get Sample of This Research Report:https://databridgemarketresearch.com/request-a-sample/?dbmr=global-stem-cell-therapy-market

Stem cell therapy is the therapy which uses stem cells for the treatment or prevention of a disease. Bone marrow transplant is the widely applicable therapy which is followed by umbilical cord blood. Research is going on to develop various sources (such as cord blood cells, bone marrow and skin) to use these cells for treatment of various disorders like neurodegenerative diseases and conditions such as heart disease, diabetes and other conditions. Some of the major players operating in the global stem cell therapy market are

Others: ViaCyte, Inc, AbbVie, Mesoblast Ltd., Roslin Cells, Regeneus Ltd, ReNeuron Group plc,, International Stem Cell Corporation, Aastrom Biosciences, Inc., Advanced Cell Technology, Cryo Cell International, Cytori Therapeutics, Inc., Geron Corporation, and Invitrogen and others. The global stem cell therapy market is highly fragmented and the major players have used various strategies such as new product launches, expansions, agreements, joint ventures, partnerships, acquisitions, and others to increase their footprints in this market. The report includes market shares of the global stem cell therapy market for global, Europe, North America, Asia Pacific and South America.

Get TOC For Full Analysis Of Report:https://databridgemarketresearch.com/toc/?dbmr=global-stem-cell-therapy-market

Major Market Drivers and Restraints:

Drivers:

Restraints:

Segmentation:

The global stem cell therapy market is segmented based on

Type

Product

Application

End Users

Geographical Segments

On the basis of type, the market is segmented into

Allogeneic stem cell therapy

Autologous stem cell therapy

The allogeneic stem cell therapy segment is expected lead the market because of commercialization of allogeneic stem cell therapy products and wide application with easy scale up process.

Based on products, the market is segmented into

Adult stem cells

Human embryonic stem cells

Induced pluripotent stem cells and others

The adult stem cells accounts highest share in market due to ability to generate trillions of specialized cells which may lower the risks of rejection and repair tissue damage.

Based on application, the market is segmented into

Musculoskeletal disorders

Wounds and injuries

Cardiovascular diseases

Surgeries

Gastrointestinal diseases, and other applications

The musculoskeletal disorders segment leads the market due to availability of stem cell-based products for the treatment of musculoskeletal disorders, high prevalence of musculoskeletal disorders and bone & joint diseases.

Based on end users, the market is segmented into

Therapeutic companies

Cell and tissues banks

Tools and reagent companies

Service companies

The growing number of stem cell donors, improved stem cell banking facilities and because of the research and development therapeutic companies held the largest share in stem cell therapy.

By Geography

North America (U.S., Canada, Mexico)

South America (Brazil, Argentina, Rest of South America)

Europe (Germany, France, United Kingdom, Italy, Spain, Russia, Turkey, Belgium, Netherlands, Switzerland, Rest of Europe)

Asia-Pacific ( Japan, China, South Korea, India, Australia, Singapore, Thailand, Malaysia, Indonesia, Philippines, Rest of Asia Pacific)

Middle East & Africa (South Africa, Egypt, Saudi Arabia, United Arab Emirates, Israel, Rest of Middle East & Africa)

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Stem Cell Therapy Market Trends, Secondary Research With Geron Corporation, Vericel Corporation, Pluristem Therapeutics, Cytori Therapeutics, Fate...

Skin Stem Cells Comments Off on Stem Cell Therapy Market Trends, Secondary Research With Geron Corporation, Vericel Corporation, Pluristem Therapeutics, Cytori Therapeutics, Fate… | October 30th, 2019

MCLEAN, Va.--(BUSINESS WIRE)--

The Thought Leadership & Innovation Foundation (TLI) announces today plans to build on its existing Regenerative Medicine Program through a research collaboration with cellular therapy industry leader RenovaCare. As part of TLIs efforts to conduct vital research in regenerative medicine and chronic disease, this initiative aims to innovate methods for reducing complications from burn and diabetic wounds across large populations.

Our research base, collaborative institutions and long history of innovation align with RenovaCares commitment to breakthrough biomedical technologies, says Bill Oldham, founder and chairman of the Board, TLI. The patented RenovaCare SkinGun technology and its ability to ultra-gently spray stem cells could present a special opportunity for investigations and applications in a wide range of regenerative therapies. Working together, our overall goal is to improve the quality, efficiency and effectiveness of patient care by not only developing new treatment methods, but also by making thoughtful and systematic changes to healthcare and health systems.

TLIs Regenerative Medicine program seeks to adapt new strategies based upon sound scientific evidence, utilizing its infrastructure to support the continuation of scientific and medical work, as well as the development of grant-funded research and other initiatives.

Dr. Robin A. Robinson, who is a TLI Fellow, Vice President of Scientific Affairs, RenovaCare, and named one of the top 100 innovators in medicine by Medicine Maker in 2018, states, This exciting collaboration between RenovaCare and TLIs Regenerative Medicine Program is the first step toward the development of meaningful and quality therapeutic treatments that will benefit patients around the world.

About TLI Foundation:

TLI Foundation is a nonprofit foundation focused on driving innovative thinking and action on global issues relating to health, education and economic empowerment. The organization is committed to fostering transformative change and improving the health and well-being outcomes of communities around the world. Visit https://www.thoughtfoundation.org/

About RenovaCare:

RenovaCare, Inc. is a biotechnology company focused on developing first-of-their-kind autologous (self-donated) stem cell therapies for the regeneration of human organs. Initial products under development target the bodys largest organ, the skin. Investigative clinical use of their flagship technology has shown to be promising new alternatives for patients suffering from burns, and chronic and acute wounds. https://www.renovacareinc.com.

View source version on businesswire.com: https://www.businesswire.com/news/home/20191028005527/en/

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Thought Leadership & Innovation Foundation to Expand Its Regenerative Medicine Program Through New Collaboration with RenovaCare - Yahoo Finance

Skin Stem Cells Comments Off on Thought Leadership & Innovation Foundation to Expand Its Regenerative Medicine Program Through New Collaboration with RenovaCare – Yahoo Finance | October 30th, 2019

Pelizaeus-Merzbacher disease is a genetic malady that leaves neurons without their myelin coating. This deficit has devastating consequences for the boysits X-linkedwho have it. These children have severe developmental delay, so they have inability to walk, inability to talk and perform self-care, says Nalin Gupta, a professor of neurological surgery and pediatrics at the University of California, San Francisco (UCSF). Their neurologic function typically does not improve, and usually they actually die during childhood.

About a decade ago, the biotech firm StemCells Inc. was looking for a neurosurgeon to try out an intervention that might finally offer some help for these children. Because Gupta had experience conducting surgical clinical trials in kids with disabilities, the company approached him to see if he could transplant neural stem cells into the brains of boys with Pelizaeus-Merzbacher disease (PMD)an approach that researchers had considered promising for a range of conditions, but which had yet to be proven effective in a clinical trial for any disease. He agreed.

In 2012, Gupta and colleagues reported that four boys with PMD who had received pluripotent neural stem cells in a Phase 1 clinical trial tolerated the procedure, and imaging techniques that indirectly detect myelin indicated they may have had myelination in their brains one year following the transplant. This August, the researchers reported the results of a long-term follow-up study of those patientsall four are still alive at ages 10, 11, 12, and 13. Patients like these who have symptoms of the disease starting at birth typically die in their teens.

Although the researchers could not directly examine myelinationthat would require autopsiesthe imaging evidence is promising. There were some clinical improvements, too, although with such a small number of patients and no control group in a trial designed to examine safety, its hard to know whether they are attributable to the transplant.

We dont actually have a product that we can use even if we wanted to do a Phase 2 study in this disease.

Nalin Gupta, UCSF

Guptas study is the latest report in a series of clinical trials on neural stem cell transplantation, in which pluripotent neural cells taken, in most cases, from the brains of aborted fetuses are expanded in the lab and then injected into the brains or spinal cords of patients with incurable neurological disorders. These include stroke, multiple sclerosis, ALS, spinal injury, and Parkinsons disease. But for all the effort that has gone in to testing these cells, none have been able to work themselves out of trials and into clinical practice.

When asked which of the human clinical trials have been most successful, Steven Goldman, a professor of neurology and neuroscience at the University of Rochester, replies, So far, none of them, right? To date, no Phase 2 trial to evaluate the efficacy of a neural stem cell treatment has been completed, he points out. And scientists, Gupta included, are less-than-ecstatic about the methods and outcomes of the clinical trials that have been done so far.

Goldman, who was not involved in the PMD experiment, calls it by far the most rigorous and well controlled. But that trial can go no further.

Gupta says he and his colleagues felt that there was sufficient evidence from the Phase 1 trial to justify a Phase 2. They cant proceed, however, because StemCells, the company that funded the research and provided the cells, closed in 2016. We dont actually have a product that we can use even if we wanted to do a Phase 2 study in this disease, he says.

Research on other potential uses for neural stem cells are also affected by a lack of momentum. Theres somewhat of a pause in what people are doing in terms of stem cell therapeutics, says Gupta. Treatments for conditions such as spinal cord injury and stroke hold the most interest for their potential societal benefit, he says, but the complexity of the changes that occur when the brain or spinal cord are injuredmeaning regions composed of multiple cell types and networks of connections are just wiped outmake for a challenging repair. Were probably a long way from being able to transplant a structure that will recapitulate the three-dimensional organization and structure of the brain and spinal cord, he says. Trials for diseases with more specific defects might be more successful, he adds, such as multiple sclerosis, which like PMD involves demyelination.

According to Evan Snyder, the director of the Center for Stem Cells and Regenerative Medicine at the Sanford Burnam Prebys Medical Discovery Institute in La Jolla, California, there havent been enough trials, and certainly not enough under ideal circumstances, to know whether neural stem cell transplantation can be an effective treatment in humans. I think the field is too young to know right now if theyre effective. I think the field can just say that theyre safe, says Snyder. To be able to know whether the cells are effective, you really need to be able to put them into an optimal setting where their mechanism of action is optimal, and that kind of trial has never been done yet, he adds.

The animal research that laid the foundation for the PMD study, and other studies on diseases involving the loss or absence of myelination, took place in a mouse model called shiverer. These animals have a mutation that prevents their oligodendrocytes from making myelin, such that their neurons are badly insulated and cannot efficiently conduct electrical signals. The shiverer mice have problems with motor functions and self-care along with seizures. They also have a tremor, hence their name.

In 1999, Snyders lab reported in PNAS that injecting mouse neural stem cells into the brains of shiverer mice led to the remyelination of neurons as well as some tremor reductionmeasured by dipping each mouses tail in ink and noting the size of the stain it left on a piece of graph paper.

Using the model, Goldmans team later transplanted human glial progenitor cellswhich are derived from neural stem cellsinto shiverer mice, generating chimeras in which mouse neurons became insulated with human myelin. The chimeric mice, as Goldman reported in Cell Stem Cell in 2008, survived longer and had improved neurological phenotypes, including fewer seizures, compared with untreated controls. In 2012, StemCells Inc., in collaboration with researchers at Oregon Health & Science University and elsewhere, reported in Science Translational Medicine that transplanting shiverer mice with human neural stem cells resulted in remyelination in the brain. Also in that issue, Gupta and StemCells described the one-year results from the PMD trial, which used the same cells for transplantation.

Neurons (green) and glia (red) differentiated from human neural stem cells in culture. Nuclei are stained blue.

Evan Snyder

The PMD study was not the first trial launched by StemCells Inc. In 2006, the company launched a Phase 1 trial of neural stem cell transplantation in children with Batten disease, a fatal condition in which children are missing a lysosomal storage enzyme. That study was the first study authorized by the FDA for transplantation with neural stem cells into the brain, says Stephen Huhn, a biotech consultant and the former chief medical officer of the company.

The trial, which was completed in 2009, revealed the treatment to be safe, the authors reported in Journal of Neurosurgery: Pediatrics. Autopies on the brains of several kids who died of the disease during the study suggested that in some patients donor cells had both survived and migrated away from the subcortical and ventricular injection sites and into the basal ganglia, among other locations, Huhn says.

The fact that we saw even glimmers of an effect was for us very promising that cellular therapy could well have a place in the treatment of some neurological disorders.

Stephen Huhn, formerly of StemCells Inc.

The stem cells used for this and other StemCells trials were isolated from the brain of a single aborted fetus, expanded as balls of cells called neurospheres, and frozen for later use. Before injection into patients, the cells were thawed, cultured for two weeks, and dissociated, so that what was injected was no longer a neurosphere but a cluster of cells, according to Huhn. Because the neural stem cells were donor-derived, patients were given immunosuppressant drugs for several months following the transplant to prevent rejection.

Using the same procedure and stock of cells, Gupta and colleagues transplanted neural stem cells into the brains of the four boys with PMD in a Phase 1 trial that began in 2009 and ran through 2012the same trial whose long-term follow-up results came out this summer. One year after transplantation, diffusion tensor imagingan MRI-based technique that lets researchers indirectly observe myelinated axonsof the boys brains suggested that myelination had occurred.

From 2012 to 2015, the company ran a Phase 1/2 trial of neural stem cell transplantation for age-related macular degeneration. The treatment proved safe, and there was also evidence of a treatment effecta slowing of the retinal damage called geographic atrophy and improvements in visual functionin some patients, says Huhn.

At the same time, the firm was engaged in a Phase 1/2 trial of stem cell transplantation for patients with injuries to the thoracic region of the spine. The treatment proved safe, and Huhn notes that several participants seemed to have sensory improvement below the level of injury, which would imply that the stem cells were having a treatment effect.

But the companys run of auspicious results did not last forever: Its Phase 2 trial of neural stem cells to treat cervical spinal cord injury, which began in 2014, terminated two years later after an independent review of the emerging data found that the study was unlikely to show a statistically significant treatment effect, Huhn says. For that same reason, a follow-up study on the same patients also ended in 2016, he adds.

At that point, StemCells Inc. shut down. STAT reported that the reason was disappointing results from the spinal cord study.

Despite stopping, the companys work was not in vain, says Huhn, as it demonstrated that the approach is safe and might be worth pursuing. These are challenging disorders, Huhn says, adding that the fact that we saw even glimmers of an effect was for us very promising that cellular therapy could well have a place in the treatment of some neurological disorders.

According to Snyder, who was not involved in the work, the PMD trial suffered from the limitations of the clinical trials system. The unfortunate thing is the way clinical trials are designed, you only get a patient who has failed every other intervention, is very deep into the disease, and almost has no chance of anything changing the course, he says. This problem is not unique to the PMD trial but applies to all neural stem cell clinical trials to date, Snyder says. In the Batten disease trial, for instance, the patients had little hope of recovery, Snyder notes. Three of the six participants had died of their disease by the time the researchers stopped collecting data. Where stem cells are going to be most useful, ultimately, is going to be the early stages of a disease where there are regions that can be rescued, and where the cells are placed in a position where they can distribute themselves throughout the region that needs to be fixed. And no clinical trial has ever met those [criteria].

Although the follow-up PMD study revealed some myelination, there was not a lot of it, notes Goldman, who was not involved in the work. Theres some evidence for local remyelination around the region of the transplants, but there was nothing that was dispersed or broad, and these patients need really widespread remyelination, he says.

Goldman says he believes that there was not more widespread and robust myelination in the PMD patients because of the cell type used. While neural stem cells can give rise to oligodendrocytes, astrocytes, and neurons, they not very efficient at making oligodendrocytes, he says. And, he adds, they do not migrate much, which is necessary for them to have widespread effects. In contrast, human glial progenitor cells, which are produced from neural stem cells and give rise to both oligodendrocytes and astrocytes, are more migratory, says Goldman, and for this reason, the field has shifted away from neural stem cells and toward glial progenitor cells for transplantation. Goldman has trials of his own in the works using a neural stem cell derivative to treat multiple sclerosis and PMD through a company he cofounded, Oscine Therapeutics.

Other trials are currently underway. Researchers at Emory University and the University of Michigan, with funding from the company Neuralstem, have completed a Phase 1 study of neural stem cells to treat ALS and, according to ClinicalTrials.gov, a Phase 2 clinical trial is ongoing. Theres a Phase 2/3 trial of nasally delivered neural stem cells to treat Parkinsons disease enrolling in China. And theres an active Phase 1 trial for Parkinsons disease in Australia using human parthenogenetic neural stem cells derived from unfertilized eggs, rather than fetal tissue.

This year, Snyder received a California Institute for Regenerative Medicine (CIRM) grant to do work leading up to cell-based therapies for babies who are at risk for developing cerebral palsy due to perinatal asphyxia, or oxygen and blood deprivation in the womb, he says. Within the first few days of life, the researchers plan to do brain imaging to identify babies with regions of the brain where cells are injured but not dead, he says, then transplant neural stem cells. The injurys still very fresh and cells are sort of teetering on a knife edge. They can either go on to die or they can go on to live, and the [transplanted] stem cells make factors that push them in the direction to live, Snyder says. If that happens, the prediction is the babies will do much better.

Theres only a short window, when cells are damaged but not dead, during which a neural stem cell transplant can work, he adds. Other trials in older patients with more advanced disease, he suggests, may have missed their optimal treatment windows. Snyder predicts that if the right patients are transplanted with the right neural stem cells at the right time, I think then, under those circumstances, now youre going to start seeing not just safety but real efficacy.

Ashley P. Taylor is a New Yorkbased freelance reporter. Follow her on Twitter@crenshawseedsand read her work atashleyptaylor.com.

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Neural Stem Cell Transplantation Crawls Toward the Clinic - The Scientist

Spinal Cord Stem Cells Comments Off on Neural Stem Cell Transplantation Crawls Toward the Clinic – The Scientist | October 30th, 2019

Massive inflammation and other changes in the striatum, an area of the brain selectively destroyed in Huntingtons disease (HD), are already present before patients develop any symptoms, a study has found.

These findings could help understand how the disease unravels and why this brain region is particularly sensitive to degeneration in individuals with Huntingtons.

The study, The caudate nucleus undergoes dramatic and unique transcriptional changes in human prodromal Huntingtons disease brain, was published in the journal BMC Medical Genomics.

Huntingtons is a neurodegenerative disease caused by mutations in the huntingtin(HTT) gene and marked by trouble in controlling movement, a progressive loss of thinking ability, and psychiatric problems.

Symptoms, which typically begin when people reach their 30s and 40s, stem from a selective degeneration of certain brain regions, particularly two areas: the basal ganglia, a region deep in the brain thats responsible for functions including movement coordination; and the cortex, the outer and highly twisted layer of the brain which controls thought, behavior, and memory.

Within the basal ganglia, HD targets nerve cells (neurons) of the striatum, especially in two areas known as the caudate nuclei and putamen. These regions can shrink and suffer massive damage as a result of disease progression.

Very little is known about the active disease processes leading to such debilitating symptoms. Obtaining post-mortem brain samples from people with disease-causing mutations who have not yet developed symptoms in other words, still have a largely intact striatum is very rare.

Researchers atBoston University School of Medicine (BUSM) had the opportunity to analyze samples from the striatum more precisely, the caudate nucleus of two HD-positive individuals who had no symptoms at the time of their death.

To pinpoint early drivers of disease, the team compared the activity (expression) of genes those turned on and turned off in the caudate nucleus of these asymptomatic individuals to the prefrontal cortex of 26 symptomatic Huntingtons patients and 56 healthy controls.

Researchers used a high throughput sequencing technology called RNA-Seq to determine gene expression profiles, and a preformed bioinformatics analysis to understand which genes and biological processes were altered.

Our data suggest that the striatum experiences massive inflammation in HD even before symptoms appear, and exhibits a similar gene expression pattern to that observed in prefrontal cortex. Patterns unique to the striatum are also observed, Adam Labadorf, PhD, director of BUs Bioinformatics Nexusand the studys senior author,said in a news release.

In addition to extensive inflammatory processes, the data also suggested that over the diseases course, the striatum undergoes some form of neurogenesis, or the generation of new nerve cells.

While these are only trends that warrant further investigation, researchers propose that active production of neurons could be happening in the striatum during the prodromal phase (before symptom onset) to compensate for the nerve cell loss that precedes symptoms.

The idea that active neurogenesis occurs in the adult brain is controversial, but could lead to exciting discoveries into the innate regenerative capabilities of the central nervous system, Labadorf said.

According to researchers, these findings provide clear evidence that the caudate nucleus is strongly affected in people positive for Huntington disease, before the emergence of any symptoms.

This study presents the most detailed analysis to date of the active disease process in the primarily affected brain region of HD, and although these results do not directly suggest any novel therapies, a better understanding of these processes is likely to lead to them, Labadorfadded.

An important observation was that some genes, like HSPA6, were perturbed across all HD patient samples relative to healthy brains. This set of genes may provide an opportunity to develop prognostic tests for disease progression, the researchers noted.

A robust clinical test measuring disease progression will likely take the form of a panel of key inflammatory and possibly developmental genes measured in the blood or cerebrospinal fluid (the liquid surrounding the brain and spinal cord), they wrote.

Ana is a molecular biologist enthusiastic about innovation and communication. In her role as a science writer she wishes to bring the advances in medical science and technology closer to the public, particularly to those most in need of them. Ana holds a PhD in Biomedical Sciences from the University of Lisbon, Portugal, where she focused her research on molecular biology, epigenetics and infectious diseases.

Total Posts: 79

Ana holds a PhD in Immunology from the University of Lisbon and worked as a postdoctoral researcher at Instituto de Medicina Molecular (iMM) in Lisbon, Portugal. She graduated with a BSc in Genetics from the University of Newcastle and received a Masters in Biomolecular Archaeology from the University of Manchester, England. After leaving the lab to pursue a career in Science Communication, she served as the Director of Science Communication at iMM.

Original post:
Huntington's Marked by Inflammation and Changes in Brain's Striatum Before Symptoms, Study Finds - Huntington's Disease News

Spinal Cord Stem Cells Comments Off on Huntington’s Marked by Inflammation and Changes in Brain’s Striatum Before Symptoms, Study Finds – Huntington’s Disease News | October 30th, 2019

WHEN Laura Farmer-Maia's daughter suddenly became clingy and unhappy, she initially brushed it off as nothing but "a phase".

And given little Beatriz was just three-years-old, the last thing to cross her mind was cancer.

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Yet, months after doctors repeatedly dismissed the symptoms as clinginess, the diagnosis was confirmed - Beatriz had an aggressive childhood cancer known as a neuroblastoma.

The horrifying news came after Beatriz's father Tiago Maia refused to leave the hospital after discovering the potential diagnosis himself on Google.

Shocked, Laura, 39, and Tiago, 40, are now urging all parents to be vigilant and check their kids for signs of the disease.

The mum, who works in advertising, first suspected something was wrong last July, when Beatriz's behaviour dramatically changed.

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She said: "Before she was diagnosed, Beatriz was quite naughty but when she reached two, she suddenly became clingy and picky with her food, and had a fever all the time.

"We took her to the GP who believed it was a virus and after recurrent visits they gave her some antibiotics to cover for a potential bacterial cause, which didnt have any effect.

"Beatriz started to complain that her legs hurt so we took her to A&E, where they did some more tests and still said it might be a virus.

"Its hard to get a diagnosis right when a child is too young to explain how theyre feeling, but in the back of our minds we knew it was something bad.

"We want to spread awareness of the difficulty of diagnosing cancer in young children - if your child doesnt seem right, you should push for further tests."

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Tiago pushed the GP for more tests, and blood tests showed something was wrong, so Beatriz was sent urgently to hospital.

It was there medics finally discovered a lump above her kidney and diagnosed her with neuroblastoma in September last year.

The cancer is aggressive and has a 40 per cent chance of long-term survival.

Everything moved so quickly and we all felt frightened as they carried out the tests

Tiago, originally from Portugal, added: "At the hospital, they twice said it was likely to be a virus and I refused to leave until I saw a specialist.

"I waited for three hours until a more senior doctor was free, and then Beatriz was examined by different specialists who admitted her to do all kinds of tests and observations including X-rays and ultrasounds - it was the last one that confirmed there was a lump.

"When my fears from Google turned out to be true, it was very strange because even though my life had just flipped upside down, I was almost relieved to be right - it was weird and confusing."

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Beatriz was referred straight to Great Ormond Street Hospital in London, where they carried out further tests including scans, blood tests and biopsies.

She began chemotherapy just a week after being diagnosed and underwent eight gruelling rounds of chemo over the next 18 months.

Doctors then carried out a stem cell transplant to regenerate bone marrow destroyed by high dose chemo, which meant Beatriz couldnt leave the hospital for eight weeks.

Tiago, a design director, said: "I was quite scared when Beatriz was diagnosed because my mum and dad had only recently died from cancer I thought of the worst.

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"Everything moved so quickly and we all felt frightened as they carried out the tests.

"Doctors found that the cancer had spread across her body, so she began chemotherapy just weeks after being diagnosed.

"We were told the treatment would last 18 months which was a massive shock to us.

"A week after Beatriz started chemotherapy she massively improved, but it was tricky being in hospital at first.

"Now, she still has periods of discomfort but sometimes shes happy to be in hospital because she has toys and people to come and play with her."

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Laura added: "The stem cell transplant was a hard time for us all because we had to spend a lot of time apart from our other daughter, Clara, six.

"Beatriz was diagnosed in Claras first week of school and it was difficult because that was supposed to be an exciting time for her."

After more scans and hopes of an all-clear, doctors found more metastatic growths still remaining in Beatrizs head, which meant that the cancer hadnt fully cleared up and she had relapsed.

The brave youngster is now undergoing immunotherapy and is due to start a six month medical trial on the NHS at Great Ormond Street Hospital, called the Beacon Trial.

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What is neuroblastoma?

Neuroblastoma is a type of cancer that most commonly afflicts babies and young children.

The disease develops from special nerve cells, known as neuroblasts, which get left behind from the child's development in the womb.

It mostly begins in the sufferer's adrenal glands located above the kidneys but can occur in the nerve tissue that runs along the spinal cord in the neck, chest, abdomen or pelvis.

The vicious illness can then spread to other organs like the bone, bone marrow, lymph nodes and skin.

Neuroblastoma afflicts around 100 children a year in the UK but the cause of the disease is still not known.

Its symptoms can include:

It is uncertain whether the trial will work and, even if Beatriz goes into remission, relapse rates are high but her parents are determined to do everything they can to stop the cancer from returning.

Laura and Tiago are now trying to raise 200,000 to help get their daughter into remission or to keep the cancer away if her treatment goes well.

The money is hoped to go towards further treatment, or if Beatriz gets the all-clear, a special vaccine in New York which helps keep the disease away.

Laura said: "After the stem cell transplant, the end was almost in sight but then she relapsed.

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"Were afraid that the cancer will get worse and worse and want to raise money to help get her into remission the ideal outcome is that the trial works and clears the disease.

"Luckily, compared to other two-year-olds, Beatriz has suffered less side effects with treatment and despite losing her curly hair shes powering through."

You can donate on Beatriz's JustGiving page here.

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Docs said our toddler was just clingy but we learned the truth on Google it was cancer - The Sun

Bone Marrow Stem Cells Comments Off on Docs said our toddler was just clingy but we learned the truth on Google it was cancer – The Sun | October 30th, 2019

By Express News Service

KOCHI:Though the impending examinations, the study leave and the hartal called by the merchants association played spoilsport, nearly 525 youngsters came forward to register themselves as stem cell donors on Tuesday at a camp set up by Smilemakers of Cusat and DATRI of St Teresas College. The organisers were expecting around 1,000 registrations.

Next, we will be holding a camp at Lulu Mall on November 14, said Ramiz Rehman of Smilemakers. Not only students but also teachers and people from outside the campus came in to register. However, one big impediment is the lack of awareness about the stem cell donation process, he said.According to him, there is a common notion that bone marrow aspiration needs to be done to extract stem cells. But this is not the case. The stem cells are extracted from the blood. There is no drilling of bones happens, said Ramiz. According to him, stem cell transfusion is the only treatment that can save the lives of those suffering from leukaemia and thalassemia.

The programme has been organised for the benefit of not only the three siblings diagnosed with thalassemia major hailing from Mattancherry, but for the thousands of patients who have registered with DATRI. Since the possibility of obtaining a match is one in 20 lakh, there is a need to have a lot of people registering as fast as possible, he said. Speaking after inaugurating the registry drive, Poornima Jayaram, actor, said: Everyone will come as one to register themselves if they put themselves in the shoes of the recipient.

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Stem cell registry gets good response - The New Indian Express

Bone Marrow Stem Cells Comments Off on Stem cell registry gets good response – The New Indian Express | October 30th, 2019

MULTIPLE sclerosis campaigners have hailed a huge step forward for patients in Scotland after a stem cell therapy was recommended for use on the NHS for the first time.

Haematopoietic stem cell transplantation (HSCT) has been described as a game-changer for MS after an international clinical trial showed that it could reboot patients immune systems and halt the progress of the disease.

Some patients who had been in wheelchairs prior to treatment said their condition improved so dramatically it was like they had never been diagnosed with MS.

READ MORE: Scots MS patients 'missing out' on pioneering stem cell treatment available in England

The Scottish Health Technologies Group (SHTG) said there is now sufficient evidence for it to recommend making HSCT available on the NHS in Scotland to MS patients who have the relapsing-remitting form of the disease, and who were not responding to drug treatments.

Iain Robertson, chairman of the SHTG, said: Our committee members were able to advise that this treatment should be considered for those with this particular type of MS who have not responded to treatment with disease-modifying therapies.

We hope that our advice will be of use in helping decide the best course of treatment for these patients.

The SHTG also stressed that patients must be made aware of the demands, risks and uncertainties of the treatment, which uses chemotherapy to wipe out patients' 'faulty' immune systems before replenishing it with a transplant of stem cells harvested from their own bone marrow.

It puts patients at high risk from infections, which can be fatal, but the theory is that the treatment works by enabling patients to 'reset' their immune system to stop it attacking the central nervous system as is the case in MS.

READ MORE: Anger of Scots MS patients travelling abroad for stem cell therapy available to some on NHS England

HSCT is not considered an effective treatment for patients with the progressive form of MS, however, as stem cells cannot regrow nerves or repair damaged myelin - the protective sheath which coats nerves.

It will also be unavailable to patients with relapsing-remitting MS who no longer show signs of inflammation on an MRI brain scan.

Scotland has one of the highest rates of MS in the world, but until now Scottish patients seeking HSCT have had to travel overseas to Mexico, Russia and Israel and bankroll their own private treatment at a cost of around 40-60,000.

It has also been available privately in London since 2017, but with a 100,000 price tag.

A small number of MS patients in England have been able to access the treatment on the NHS, however, because there are clinical trials into HSCT taking place at NHS hospitals in Sheffield and London.

Morna Simpkins, director of MS Society Scotland, said: The decision from SHTG to approve HSCT for the treatment of MS is good news and could help in the development of a clear pathway, for people who could potentially benefit, to access it.

We will push to ensure that this decision leads to real change for people with MS by continuing to engage with other groups to offer the treatments, including HSCT, which are right for them.

READ MORE: Stem cells help mother with MS make 'remarkable' recovery

The SHTG said eligible patients must have equal access to the procedures regardless of where they live, but it is unlikely all health boards will be able to provide it.

The MS Society wants a centre, or centres, of excellence set up where patients from across Scotland can be referred.

Lucy Clarke from the Scottish HSCT Network said the recommendation was "a huge step forward" for people in Scotland living with MS.

Ms Clarke underwent HSCT in Russia and credits it with substantially reversing her disability.

She added: This important decision supports HSCT as a treatment option where other treatments have failed. We will continue to push so that this treatment is available to people in Scotland who need it.

A Scottish Government spokeswoman said: We are grateful to the Scottish Health Technologies Group for this important work.

"NHS Boards are expected to consider their advice on technologies in the planning and provision of its services and clinicians are expected to follow their professional judgement, working within the management structure of their Board.

We will work closely with MS Society Scotland, other third sector bodies and the clinical community to consider what the Technologies Groups findings means for provision in Scotland, including the information that needs to be available to people about eligibility and risks.

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Stem cell therapy approved for MS patients in Scotland - HeraldScotland

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DURHAM, N.C., Oct. 29, 2019 (GLOBE NEWSWIRE) -- Chimerix, Inc. (CMRX), a biopharmaceutical company focused on accelerating the development of innovative medicines to treat patients with cancer and other serious diseases, today announced that it will host a live conference call and audio webcast on Tuesday, November 5, 2019 at 8:30 a.m. ET to report financial results for the third quarter ended September 30, 2019, and to provide an operational update.

To access the live conference call, please dial (877) 354-4056 (domestic) or (678) 809-1043 (international) at least five minutes prior to the start time, and refer to conference ID 1693898. A live audio webcast of the call will also be available on the Investors section of the Company's website, http://www.chimerix.com. An archived webcast will be available on the Chimerix website approximately two hours after the event.

AboutChimerix

Chimerixis a development-stage biopharmaceutical company dedicated to accelerating the advancement of innovative medicines that make a meaningful impact in the lives of patients living with cancer and other serious diseases. The two clinical-stage development programs are dociparstat sodium (DSTAT) and brincidofovir (BCV).

Dociparstat sodium is a glycosaminoglycan biologic derived from porcine heparin that has low anticoagulant activity but retains the ability to inhibit activities of several key proteins implicated in the retention and viability of AML blasts and leukemic stem cells in the bone marrow during chemotherapy (e.g., CXCL12, selectins, HMGB1). Mobilization of AML blasts and leukemic stem cells from the bone marrow has been associated with enhanced chemosensitivity and may be a primary mechanism accounting for the observed increases in EFS and OS in Phase 2 with DSTAT versus placebo. Randomized Phase 2 data suggests that DSTAT may also accelerate platelet recovery post chemotherapy via inhibition of platelet factor 4, a negative regulator of platelet production that impairs platelet recovery following chemotherapy. BCV is a lipid conjugate DNA polymerase inhibitor in development as a medical countermeasure for smallpox.For further information, please visit the Chimerix website,www.chimerix.com

CONTACT:

Investor Relations:Michelle LaSpaluto919-972-7115ir@chimerix.com

Will OConnorStern Investor Relations212-362-1200will@sternir.com

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Chimerix to Announce Third Quarter 2019 Financial Results and Provide an Operational Update on November 5, 2019 - Yahoo Finance

Bone Marrow Stem Cells Comments Off on Chimerix to Announce Third Quarter 2019 Financial Results and Provide an Operational Update on November 5, 2019 – Yahoo Finance | October 30th, 2019

The new horror flick on Netflix, Eli, released just in time for Halloween, borrows from The Exorcist and Rosemarys Baby, with touches of The Shining. And it all takes place in what looks like Downton Abbey with the cleaning staff gone.

Eli works; its scary. But the set-up using gene therapy gone awry is unfortunate, superfluous, and even offensive. (Beware, spoilers ahead)

The film opens with 11-year-old Eli dreaming about being able to go outside without his hazmat suit and breathing without his skin reddening and blistering. He awakens and hes inside, in a bubble.

David was diagnosed, four years earlier, with a rare formof severe combined immune deficiency (SCID). It slashes his ability to make the antibodies that protect against infection, unleashes inflammation that reddens his skin, while at the same time turns his immune system against his own tissues, an autoimmune response.

The parents, caring Rose and weirdo Paul, bundle Eli up to take him to a doctor whos going to cure him with a new treatment. Once at the supposedly clean Downton Abbey haunted house, Eli has a decontamination shower.

When the boy meets the doc, she explains that his immune system makes too many bad immunoglobulins, using that word instead of antibodies because it sounds more technical.Eli quickly responds, spouting out that he has mutations in the RAG1 and RAG2 genes (recombination-activating genes).

Elis body cant make the enzymes that mix and match antibody parts, and the proportions of a bunch of immune system cells and proteins go out of whack. His condition is also called Omenn syndrome.

Dr. Horn has two nurses, and all three of them wear purple uniforms.

Good news! Dr. Horn will administer viral gene therapy! I will make you better, like my other patients, she assures the boy.

Rose gingerly begins to unwrap the blue layers that encase her son, bending down and looking like Laura Dern examining dino poo in Jurassic Park or Princess Leia releasing the hologram from R2D2. Mom and boy can finally hug!!!

At night, the house creaks. Eli wanders the spooky halls, glimpsing kids in the windows, mirrors, and reflections, including ghostly girls who look like the twins at the end of the hallway in The Shining.

A redheaded girl outside, Sadie Sink, apparently escaped from playing Max on Stranger Things, seems real.

Im allergic to the world, Eli tells her. Not exactly.

The next morning, Dr. Horn blames Elis ghost sightings on a side effect from immunosuppressants. Why is she trying to suppress an immune system already so impaired?

Next Eli, who looks so much like Tom Petty that I expected him to shriek I Wont Back Down, is strapped down to a table with a contraption holding his head in place, as Dr. Frankenstein asks her nurses to take a reading.

Dr. Horn at first seems to have gotten the basic idea of gene therapy correct: introducing a working copy of the mutant gene aboard viruses into stem cells from bone marrow. And poof! Like a magic trick! itll work, she proclaims.

She proceeds to extract a hunk of pinkish gunk after drilling into a bone, as the immobilized boy twists and grimaces on the table. Satisfied, the doc plops the glob into a Petri dish.

It burns! Eli shrieks.

That means its working, replies the doc. Within seconds, the doctored viruses have apparently hit their targets.

Then Eli awakens. It all seems a dream, but its foreshadowing.

A ghost appears in a bloody nightgown.

The house breathes at night.

A scrawny, dagger-nailed hand grabs Eli and the apparition turns into his father.

When Eli writes his name on a window, the letters rearrange to spell Lie, like Redrum becoming Murder in the mirror in The Shining. Later on, with the E written like a 3, Eli scratched into various furniture surfaces in the house becomes 317317317. What can it mean?

When Eli reports these events, Dr. Horn barks, Its the medication, as if sophisticated gene therapy has suddenly become as mundane as a tab of Tylenol. Shell have to lower the dosage because the second of the three treatments is coming up.

Treatment 2 is indeed brutal. Eli is held in a contraption like the one Hannibal Lecter wears to keep him from eating people and his head bolted like hes Frankenstein.

Were confident that the gene therapy virus is correcting the mutation, Dr. Horn declares, adding that this will burn a little bit, as she presumably delivers more.

When Eli turns red and screams, she assures him that this is supposed to happen. The virus is penetrating the blood-brain barrier, as if said barrier is a superhighway requiring that the bolts hold his head still.

I was speechless.

Barrier refers to the blood vessels in the brain that are closed to large molecules, which keeps toxins out. A widely-used gene therapy vector, AAV9 (adeno-associated virus 9), has been known for a decade to naturally cross the barrier. And that doesnt require torture hardware.

Heres a photo of one of the kids I write about receiving AAV9 gene therapy for a rare neurological disease through an intravenous delivery in her hand!

The doc then attributes Elis reaction to his body initially rejecting the new cells, like any transplant. But if his gene therapy consists of viruses traipsing across the blood-brain barrier, where did cells suddenly come from? Is it the doctoring of stem cells from bone marrow that was in Elis dream, or delivering viruses into the bloodstream?

Elis nocturnal adventures continue. Hes pushed and pulled from unseen forces as the floor turns transparent, revealing scary medical people. As he keeps bellowing the doc orders Haldol and his mom pushes Valium.

The mysterious 317 opens a key pad to an inner sanctum, which looks like the set of the second Indiana Jones film. We see insects alighting, so the place was never a clean room after all.

Eli finds a notebook with case histories of the past patients and the pieces start to fit. Perry. Agnes. Lucas.

After treatment 2, the kids eyes look haunted, their complexions gray, like Eli. After treatment 3, their heads exploded.

Then the religion clues start to fall out.

Eli discovers a photo of nuns that includes his medical team. A huge iron cross sheaths a dagger. The surgical table with Eli across it resembles Christ on the cross.

One reviewer posits that the plot is about gay conversion, pointing to a scene in which Eli literally crawls out of a closet to tell his parents the truth.

The action speeds up and twists as treatment 3 looms.

Dr. Horn dons religious garb, makes the sign of the cross, flings holy water, and babbles about Jesus and the archangel. The boy, having discovered the medical records, has become a liability.

I thought I could cure Eli. The gene therapy would have worked, if he wasnt so strong. But he cant leave here! the enraged doc yells.

But when Eli is tied down and Dr. Crazy is coming at him with the dagger pulled from the cross, he suddenly summons his inner Regan MacNeil (from The Exorcist) and stops the knife in mid-air, turns it around, and forces the doctor to stab herself. She mutters may you find peace and forgiveness in the name of the Lord, channeling Father Damien KarrasThe power of Christ compels you! as he attempts to exorcise Regan.

With the plunging of the dagger, Eli, red-eyed and screaming, rips off his restraints. His parents are thrown to the floor while the nurses and the doc, somehow still living with the dagger in her chest, try to leave.

But Eli, like Anthony in the cornfield episode of the Twilight Zone, points at them and they turn in unison and then elevate, like Regan rising from her bed. The purple ones then float around the room in an eerie circle emanating an unearthly blue glow, as if theyre on one of those centripetal force amusement park rides.

A conspiracy revealed

It turns out that all are in on whats happening, even the nice-seeming mom. And Eli realizes hes never been sick.

What has she been putting inside me? What have you been putting inside me? he shrieks at his parents, conjuring images of Rosemarysdevil spawn.

At that the nurses and doc suddenly flip upside down, the horror equivalent of Regans rotating head, and slam to the floor.

What am I?

Our son.

Eli sets the nurses and doc on fire.

Are you my dad?

I prayed every day! answers dad.

Prayed to whom? the boy bellows.

The Lord didnt answer me, but your father did, Rose utters mysteriously.

And we know.

Eli never had a SCID. Its a twist on Munchausen Syndrome by Proxy, the cause of his symptoms the holy water that mom and then doc sprinkled on him.

But the ultimate cause? Dad is the devil. At that realization, Eli makes his dads head explode.

The other kids, whose bodies indeed turn up, were Elis half-siblings, even Haley. Dad the Devil got around.

Eli is fun, fast and scary. But why did the writers have to tarnish gene therapy? Why use a genetic disease at all? And especially an ultra rare one? I cant help but wonder what motivated the writers to do this.

Ive devoted the past decade to learning about families who have rare genetic diseases and have kids who have had, or wish they could have, gene therapy, writing about them, and accompanying some of them on their journeys.

In addition to my posts here and at my blog DNA Science, I wrote the only book on gene therapy, The Forever Fix, which chronicles the efforts of a few families. The first gene therapy was FDA-approved in late 2017. The technology has indeed been like the mythical phoenix bird, arising from the ashes.

For the families, I resent the use of gene therapy as a plot point.

After viewing the film the other night, I did a final Facebook check. The first thing that popped up: a photo of an exquisite child, on a page for families dealing with Sanfilippo syndrome, a devastating neurological condition.

The boy was now free of the cruel disease, free to be at peace. He was 11. Elis age.

Genetic disease, and especially attempts to treat it, shouldnt be the stuff of horror films.

Ricki Lewis is the GLPs senior contributing writer focusing on gene therapy and gene editing. She has a PhD in genetics and is a genetic counselor, science writer and author of The Forever Fix: Gene Therapy and the Boy Who Saved It, the only popular book about gene therapy. BIO. Follow her at her website or Twitter @rickilewis

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Viewpoint: Netflix's new horror movie 'Eli' is a fright. But why did they have to 'tarnish gene therapy'? - Genetic Literacy Project

Bone Marrow Stem Cells Comments Off on Viewpoint: Netflix’s new horror movie ‘Eli’ is a fright. But why did they have to ‘tarnish gene therapy’? – Genetic Literacy Project | October 30th, 2019

More than 10 million people worldwideabout 1 percent of people over age 60live with Parkinsons disease. There are treatments that can help control symptoms, but there is no cure.

The hallmark of the disease is the death of certain brain cellsneurons that produce dopamine. Most Parkinsons researchers have focused on studying these cells. But what if the disease starts elsewhere? What if it involves not only neurons but other cells that interact with neurons? In particular, what role is played by astrocytes, star-shaped cells that nurture and help form the connections, or synapses, between the neurons?

(This article by Angela Spivey, with photos by Alex Boerner, originally appeared in Duke Medical Alumni News. Read that story here.)

Thats the question a team of Duke researchers led by Cagla Eroglu, PhD, associate professor of cell biology and neurobiology, is exploring, thanks to a $1 million grant from the Chan Zuckerberg Initiative.

Sitting in her office, Eroglu picks up an orange plastic object that resembles a piece of coral, its tentacles branching this way and that. This is a model of a mouse astrocyte, she says. It can interact with 100,000 synapses at the same time. Astrocytes, she explains, infiltrate the brain, touching everything within their reach. They communicate with its synapses, regulating blood flow and metabolism.

Astrocytes from the Greek astron, meaning "star"have traditionally been thought of as support cells. But that thinking is changing. Since astrocytes are in such close contact and continuously communicating with synapses, we are beginning to appreciate that they are also fundamentally involved in regulating brain function, Eroglu says.

Collaborating with Albert La Spada, MD, PhD, Eroglu found that a certain gene known to be important in Parkinsons is more highly expressed in astrocytes than in neurons. And when the researchers mutated that gene in astrocytes, they saw some intriguing changes. This still-unpublished work laid the foundation for their proposal to the Chan Zuckerberg Initiative, which is bringing together experimental scientists from divergent fields to take a fresh look at the causes of neurodegenerative disorders.

There are vanishingly few papers that have delved into how astrocytes are contributing to the Parkinsons disease process, says La Spada, professor of neurology and vice chair of research for the Department of Neurology. This is an area that's been under-studied, and I think that the results that we're generating are suggesting that it deserves more attention.In addition to his long experience studying neurodegenerative diseases, La Spada brings expertise in growing astrocytes from induced pluripotent stem cells (IPSCs). That process starts by growing skin cells from a skin biopsy from a Parkinsons patient. Then we use what's called a reprogramming protocol to basically revert them to stem cells that are pluripotent. Once you create the IPSCs, you could use them to make any cell you wanta muscle cell or a cardiac cell or a neuron or an astrocyte, La Spada says. The beauty of this is, it comes from the patient who has the disease of interest."

His labs expertise will only grow because of the Chan Zuckerberg Initiative, which has formed focus groups for grantees around various areas, such as stem cell modeling, CRISPR gene-editing technology, bioinformatic analysis of data sets, and more. We're meeting other researchers from around the world who are doing really unique things. It's a chance for us all to compare notes, and I think this will accelerate all of our endeavors, La Spada says.

Rounding out the team is Nicole Calakos, MD, PhD, a scientist and clinician who treats patients with movement disorders, including Parkinsons. Calakos says that when she first met Eroglu, she was intrigued by her idea that since astrocytes are involved in sculpting the language of neurons, perhaps they play a role in the events that can lead to disease.

Everybody has been fixated like a magnet on the idea that the problem is the neuron that's dying, Calakos says. Cagla said, Hey, let's think outside of the box of that dead cell. Lets consider whether astrocytes are like the soil around a plant, providing the nutrition, and allowing it to form roots, and maybe that is whats broken. Why aren't we even thinking about this critical piece of the brain?

Eroglu puts it this way: Maybe the problem is loss of connections between neurons, even before they die.

Calakos says that part of the reason she came to Duke was the close intermingling of physicians and bench scientists. Because of how the community is at Duke, Cagla and I had been exchanging ideas and collaborating over the years, she says. The Chan Zuckerberg grant is an opportunity to get together as a formal team. I think it's really forward-thinking of them to have teams of basic scientists and practicing physicians all talking to each other.

The Chan Zuckerberg Initiative was launched in December 2015 by Mark Zuckerberg, founder and CEO of Facebook, and Priscilla Chan, a pediatrician and founder and CEO of The Primary School in East Palo Alto. In addition to her clinical insight, Calakos brings expertise in electrophysiologyreal-time recording and observation of electrical signals coming from brain cells. We can listen to the language of synapses, she says. They speak in electrical currents,which we can measure. Eroglu believes that by learning all they can about how astrocytes support synaptic development and health in the normal brain, they may find ways to stop neurodegenerative diseases like Parkinsons.

We are seeing aging as a part of development, Eroglu says. If your house is built on a strong base, then it might last longer. Whereas, if you build it in another way, it may be there for a while, but gradually start to break down.

This doesn't mean that we are destined to have neurodegeneration and we can't do anything. We may be more predisposed to get the disease, but we may not get it if we have done something else in our lives that helps strengthen our brain. I strongly believe that there will be ways to stop neurodegeneration.We will find a way to strengthen the brain connections. If we can figure out the weakest link, then we could concentrate on solving that.

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The Stars in Our Brains - Duke Department of Neurology

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DUBLIN--(BUSINESS WIRE)--The "Cell Based Assay & High Content Screening Markets Market Forecasts by Application, With Executive and Consultant Guides and including Customized Forecasting and Analysis 2020 to 2024" report has been added to ResearchAndMarkets.com's offering.

Cell Based Assays are a mainstay of drug development and scientific research, but growth is now accelerating as new immuno-oncology markets create unprecedented investment in the race to cure cancer. On top of this new technology is allowing Cell Based Assays to be used to measure any aspect of cell function. This market just keeps on growing with no end in sight. The workhorse of the pharmaceutical industry is becoming a central player in biotechnology.

This is a complex area but this readable report will bring the entire management team up to speed, on both the technology and the opportunity.

The technology is moving faster than the market. Genomics and Immunology are playing a role too. Find opportunities and pitfalls. Understand growth expectations and the ultimate potential market size.

Key Topics Covered

1. Introduction and Market Definition

1.1 What are Cell Based Assays?

1.2 Clinical Trial Failures

1.2.1 Immuno-oncology Plays a Leading Role in Cell Based Assays

1.3 Market Definition

1.3.1 Market Size

1.3.2 Currency

1.3.3 Years

1.4 Methodology

1.4.1 Authors

1.4.2 Sources

1.5 U.S. Medical Market and Pharmaceutical Research Spending - Perspective

1.5.1 U.S. Expenditures for Pharmaceutical Research

2. Cell Based Assays - Guide to Technology

2.1 Cell Cultures

2.1.1 Cell Lines

2.1.2 Primary Cells

2.1.3 Stem Cells

2.1.3.1 iPSC's - The Special Case

2.2 Cell Assays

2.3 Cell Viability Assays

2.3 Cell Proliferation Assays

2.4 Cytotoxicity Assays

2.5 Cell Senescence Assays

2.6 Apoptosis

2.7 Autophagy

2.8 Necrosis

2.9 Oxidative Stress

2.10 2D vs. 3D

2.11 Signalling Pathways, GPCR

2.12 Immune Regulation & Inhibition

2.13 Reporter Gene Technology

2.14 CBA Design & Development

2.15 Cell Based Assays - The Takeaway

3. Industry Overview

3.1 Players in a Dynamic Market

3.1.1 Academic Research Lab

3.1.2 Contract Research Organization

3.1.3 Genomic Instrumentation Supplier

3.1.5 Cell Line and Reagent Supplier

3.1.6 Pharmaceutical Company

3.1.7 Audit Body

3.1.8 Certification Body

4. Market Trends

4.1 Factors Driving Growth

4.1.1 Candidate Growth

4.1.2 Immuno-oncology

4.1.3 Genomic Blizzard

4.1.4 Technology Convergence

4.1.5 The Insurance Effect

4.2 Factors Limiting Growth

4.2.1 CBA Development Challenges

4.2.2 Instrument Integration

4.2.3 Protocols

4.3 Technology Development

4.3.1 3D Assays

4.3.2 Automation

4.3.3 Software

4.3.4 Primary Cells

4.3.5 Signalling and Reporter Genes

4.3.6 The Next Five Years

5. Cell Based Assays Recent Developments

5.1 Recent Developments - Importance and How to Use This Section

5.1.1 Importance of These Developments

5.1.2 How to Use This Section

6. Profiles of Key Cell Based Assay Companies

7. Global Market Size

7.1 Cell Based Assay Global Market Size by Region with Charts

7.2 Cell Based Assays Global Market Size by Type with Charts

8. Global Market by User Type

8.1 Pharmaceutical Market

8.1.1 Pharmaceutical Market by Region with Chart

8.2 Basic Research Market

8.2.1 Basic Research Market by Region with Chart

8.3 Industrial/Cosmetic Market

8.3.1 Industrial/Cosmetic Market by Region with Chart

9. Cell Based Assay by Product Class

9.1 Instrument Market

9.1.1 Instrument Market by Region with Chart

9.2 Reagent Market

9.2.1 Reagent Market by Region with Chart

9.3 Services Market

9.3.1 Services Market by Region with Chart

9.4 Software Market

9.4.1 Software Market by Region with Chart

10. Appendices

10.1 FDA Cancer Drug Approvals by Year

10.2 Clinical Trials Started 2010 to 2016

10.3 Share of Pharma R&D by Country

Companies Mentioned

For more information about this report visit https://www.researchandmarkets.com/r/atsea9

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Global Cell Based Assay & High Content Screening Markets, 2020-2024 | Forecast by Application with Executive & Consultant Guides -...

Cardiac Stem Cells Comments Off on Global Cell Based Assay & High Content Screening Markets, 2020-2024 | Forecast by Application with Executive & Consultant Guides -… | October 30th, 2019