Mum shares heartbreaking photo of toddler sobbing through gruelling cancer treatment – The Sun

THIS is the heartbreaking moment a tiny toddler sobs her way through agonising cancer treatment.

Sophia Soto's devastated mum took the photo while her little 14-month-old was having a lead put on her chest.


Sophia was diagnosed with stage 4 neuroblastomaafter tumours were discovered behind her eyes and on her kidney.

And her mum Rosie, 40, is now sharing the harrowing image in a bid to highlight the traumatic reality of childhood cancer.

Speaking about the poignant picture, Rosie, from Florida, USA, said: "The picture of Sophia upset really does home in on the reality of childhood cancer.

"She was having a lead put on her chest for her treatment - which she didn't want - hence why Sophia was so upset.



"I look back at the picture now and wonder how I did it; it was so hard watching my little girl so ill."

Rosie first suspected something was wrong with Sophia after she began developing bruising around her eyes - something she claims doctors repeatedly dismissed as being from bump or fall.

It wasn't until Rosie took Sophia to see an eye specialist that they spotted what they suspected tumours behind her eyes were causing the bruising.

Rosie added: "Sophia kept getting bruising on her eyes and I didn't recall her falling over or anything, so I didn't understand where they were come from.



"I kept taking her to the doctors because the bruising wasn't going away, but they just said it must have been from a bump or something.

"Sophia wasn't referred for a scan or biopsy until I went to see an eye specialist with her who knew straight away that it was caused by a tumour.

"She was sent for an MRI where black spots appeared on the scans confirming the tumours behind her eyes.

"It was then the biopsy which found the tumours on one of her kidneys as well which led to her stage 4 neuroblastoma diagnosis."

Neuroblastoma is a rare type of cancer that mostly affects babies and children and develops from specialised nerve cells left behind from a baby's development in the womb.

I kept taking her to the doctors because the bruising wasn't going away, but they just said it must have been from a bump or something

After being diagnosed in March 2014, the then 14-month-old endured 60 rounds of chemotherapy, 20 rounds of radiation and a stem cell transplant over a six months period.

Thankfully, Sophia, now six, has been in remission for five years and now looks like a completely different child compared to the one in the heart wrenching photograph.

She was told she was in remission in November 2014 and has been medication free for two years.

Sophia isn't yet classified as 'cancer free' so still goes for check ups every six months with specialists.

What are the symptoms of cancer in children?

Cancer symptoms can be very similar to those of other childhood illnesses - and they vary between children.

According toCancer Research UK, there are 15 signs to look out for:



The brave six-year-old still has tumours behind her eyes which cannot be removed due to the placement of them, but doctors believe the tumours are benign and therefore not causing Sophia too much harm.

Rosie added: "Doctors are reluctant to remove the tumours Sophia currently has behind her eyes as they've said it would be likely the surgery to disfigure her face.

"Whilst they are tumours, doctors are reasonably confident that they are not cancerous so we have decided to not have the surgery right now, but it may be something she has when she's older."



Sophia now looks like any other fun-loving child and from her appearance, you would never know she had cancer.

The six-year-old loves to dance and hopes of becoming a vet one day.

Rosie said: "No one can imagine what she went through looking at her now - she just looks like a normal regular child.

"Sophia has her moments when she asks about when she was sick and has questions about her treatment scars, but over all she's a pretty happy girl.




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"If I was to say anything to other parents with children battling cancer, I'd say to them to not give up, stay positive and keep your faith.

"It's really important not to compare your child's process to anyone else as everyone battles illnesses differently as every situation is different.

"We're over the moon that Sophia is now doing so well - we're really blessed that she's such a fighter."


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Mum shares heartbreaking photo of toddler sobbing through gruelling cancer treatment - The Sun

Bone Therapeutics SA: Information on the total number of voting rights and shares – GlobeNewswire

Regulated information

Gosselies, Belgium, 29November 2019, 7am CET BONE THERAPEUTICS (Euronext Brussels and Paris: BOTHE), the leading biotech company focused on the development of innovative cell and biological therapies to address high unmet medical needs in orthopaedics and bone diseases, today announces an increase in the total number of voting rights and shares as a result of the issuance of new shares on 14November 2019 following the conversion of convertible bonds issued on the private placement on 7March 2018. The following information is published in accordance with Article15 of the Belgian Law of 2May 2007 on the publication of major shareholdings in issuers whose shares are admitted to trading on regulated market.

(1) Based on the conversion price of EUR3.3971 (92% of the Volume-Weighted-Averaged-Price of Bone Therapeutics shares on 14November 2019).

About Bone Therapeutics

Bone Therapeutics is a leading biotech company focused on the development of innovative products to address high unmet needs in orthopaedics and bone diseases. Based in Gosselies, Belgium, the Company has a broad, diversified portfolio of bone cell therapy and an innovative biological product in later-stage clinical development across a number of disease areas, which target markets with large unmet medical needs and limited innovation.

Bone Therapeutics core technology is based on its allogeneic cell therapy platform (ALLOB) which uses a unique, proprietary approach to bone regeneration, which turns undifferentiated stem cells from healthy donors into bone-forming cells. These cells can be administered via a minimally invasive procedure, avoiding the need for invasive surgery, and are produced via a proprietary, cutting-edge manufacturing process.

The Companys ALLOB product pipeline includes a cell therapy product candidate that is expected to enter PhaseIIb clinical development for the treatment of difficult-to-heal fractures and a PhaseII asset in patients undergoing a spinal fusion procedure. In addition, the Company is also developing an off-the-shelf protein solution, JTA-004, which is expected to enter PhaseIII development for the treatment of pain in knee osteoarthritis.

Bone Therapeutics cell therapy products are manufactured to the highest GMP (Good Manufacturing Practices) standards and are protected by a broad IP (Intellectual Property) portfolio covering ten patent families as well as knowhow. Further information is available at


Bone Therapeutics SAThomas Lienard, Chief Executive OfficerJean-Luc Vandebroek, Chief Financial OfficerTel: +32 (0) 71 12 10

International Media Enquiries:Consilium Strategic CommunicationsMarieke VermeerschTel: +44 (0) 20 3709

For French Media and Investor Enquiries:NewCap Investor Relations & Financial CommunicationsPierre Laurent, Louis-Victor Delouvrier and Arthur RouillTel: + 33 (0)1 44 71 94

Certain statements, beliefs and opinions in this press release are forward-looking, which reflect the Company or, as appropriate, the Company directors` current expectations and projections about future events. By their nature, forward-looking statements involve a number of risks, uncertainties and assumptions that could cause actual results or events to differ materially from those expressed or implied by the forward-looking statements. These risks, uncertainties and assumptions could adversely affect the outcome and financial effects of the plans and events described herein. A multitude of factors including, but not limited to, changes in demand, competition and technology, can cause actual events, performance or results to differ significantly from any anticipated development. Forward looking statements contained in this press release regarding past trends or activities should not be taken as a representation that such trends or activities will continue in the future. As a result, the Company expressly disclaims any obligation or undertaking to release any update or revisions to any forward-looking statements in this press release as a result of any change in expectations or any change in events, conditions, assumptions or circumstances on which these forward-looking statements are based. Neither the Company nor its advisers or representatives nor any of its subsidiary undertakings or any such person`s officers or employees guarantees that the assumptions underlying such forward-looking statements are free from errors nor does either accept any responsibility for the future accuracy of the forward-looking statements contained in this press release or the actual occurrence of the forecasted developments. You should not place undue reliance on forward-looking statements, which speak only as of the date of this press release.

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Bone Therapeutics SA: Information on the total number of voting rights and shares - GlobeNewswire

Mesenchymal Stem Cells Market Key Trends, Key Players, Challenges And Standardization, Analysis Of Key Players, And Forecast To 2026 – WindStreetz

The Global Mesenchymal Stem Cells Market covers explicit data considering the advancement rate, advertise gauges, drivers, confinements, future based interest, and income during the figure time frame. Further, the report comprises information amassed from various primary and secondary sources.

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The mesenchymal stem cells market is anticipated to grow at a CAGR of 7.0% from 2018 to 2026 according to a new research report published by Alexa Reports Research. The market was valued at USD 1,335.1 million in 2017 and is estimated to reach USD 2,518.5 Million by 2026. In 2017, the drug discovery application dominated the market, in terms of revenue. North America region is observed to be the leading contributor in the global market revenue in 2017.

Mesenchymal stem cells are adult stem cells, which are traditionally found in the bone marrow. However, they can also be parted from other available tissues including peripheral blood, cord blood, fallopian tube. These stem cells mainly function for the replacement of damaged cell and tissues. The potential of these cell is to heal the damaged tissue with no pain to the individual. Scientists are majorly focusing on developing new and innovative treatment options for the various chronic diseases like cancer. Additionally, the local governments have also taken various steps for promoting the use of these stem cells.

The global mesenchymal stem cells market growth is primarily driven by the increasing demand for these stem cells as an effective treatment alternative for knee replacement in the recent past.

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Furthermore, increasing elderly population across the globe, and rising prevalence of various chronic diseases including cancer, autoimmune diseases, bone and cartilage diseases are factors expected to boost the market growth during the forecast period. In addition, effective government policies, and funding for research and development would positively influence the market growth over coming years. However, some of the political point of views, and higher cost of treatment by using mesenchymal stem cells might restraint the growth during the forecast period.

Increasing demand for better healthcare facilities, rising geriatric population across the globe, and continuous research and development activities in this area by the key players is expected to have a positive impact on the growth of Mesenchymal Stem Cells market. North America generated the highest revenue in 2017, and is expected to be the leading region globally during the forecast period. The Asia Pacific market is also expected to witness significant market growth in coming years. Developing healthcare infrastructure among countries such as China, India in this region is observed to be the major factor promoting the growth of this market during the forecast period.

The major key players operating in the industry are Cell Applications, Inc., Cyagen Biosciences Inc. Axol Bioscience Ltd., Cytori Therapeutics Inc., Stem cell technologies Inc., Celprogen, Inc. BrainStorm Cell Therapeutics, Stemedica Cell Technologies, Inc. These companies launch new products and undertake strategic collaboration and partnerships with other companies in this market to expand presence and to meet the increasing needs and requirements of consumers.

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About Us:Alexa Reports is a globally celebrated premium market research service provider, with a strong legacy of empowering business with years of experience. We help our clients by implementing decision support system through progressive statistical surveying, in-depth market analysis, and reliable forecast data. Alexa Reports is a globally celebrated premium market research service provider, with a strong legacy of empowering business with years of experience. We help our clients by implementing decision support system through progressive statistical surveying, in-depth market analysis, and reliable forecast data.

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Mesenchymal Stem Cells Market Key Trends, Key Players, Challenges And Standardization, Analysis Of Key Players, And Forecast To 2026 - WindStreetz

Genome Editing Services, World Markets to 2030: Focus on CRISPR – The Most Popular Genome Manipulation Technology Tool – PRNewswire

DUBLIN, Nov. 28, 2019 /PRNewswire/ -- The "Genome Editing Services Market-Focus on CRISPR 2019-2030" report has been added to's offering.

This report features an extensive study of the current landscape of CRISPR-based genome editing service providers. The study presents an in-depth analysis, highlighting the capabilities of various stakeholders engaged in this domain, across different geographical regions.

Currently, there is an evident increase in demand for complex biological therapies (including regenerative medicine products), which has created an urgent need for robust genome editing techniques. The biopharmaceutical pipeline includes close to 500 gene therapies, several of which are being developed based on the CRISPR technology.

Recently, in July 2019, a first in vivo clinical trial for a CRISPR-based therapy was initiated. However, successful gene manipulation efforts involve complex experimental protocols and advanced molecular biology centered infrastructure. Therefore, many biopharmaceutical researchers and developers have demonstrated a preference to outsource such operations to capable contract service providers.

Consequently, the genome editing contract services market was established and has grown to become an indispensable segment of the modern healthcare industry, offering a range of services, such as gRNA design and construction, cell line development (involving gene knockout, gene knockin, tagging and others) and transgenic animal model generation (such as knockout mice). Additionally, there are several players focused on developing advanced technology platforms that are intended to improve/augment existing gene editing tools, especially the CRISPR-based genome editing processes.

Given the rising interest in personalized medicine, a number of strategic investors are presently willing to back genetic engineering focused initiatives. Prevalent trends indicate that the market for CRISPR-based genome editing services is likely to grow at a significant pace in the foreseen future.

Report Scope

One of the key objectives of the report was to evaluate the current opportunity and the future potential of CRISPR-based genome editing services market. We have provided an informed estimate of the likely evolution of the market in the short to mid-term and long term, for the period 2019-2030.

In addition, we have segmented the future opportunity across [A] type of services offered (gRNA construction, cell line engineering and animal model generation), [B] type of cell line used (mammalian, microbial, insect and others) and [C] different geographical regions (North America, Europe, Asia Pacific and rest of the world).

To account for the uncertainties associated with the CRISPR-based genome editing services market and to add robustness to our model, we have provided three forecast scenarios, portraying the conservative, base and optimistic tracks of the market's evolution.

The research, analysis and insights presented in this report are backed by a deep understanding of key insights generated from both secondary and primary research. All actual figures have been sourced and analyzed from publicly available information forums and primary research discussions. Financial figures mentioned in this report are in USD, unless otherwise specified.

Key Topics Covered

1. PREFACE1.1. Scope of the Report1.2. Research Methodology1.3. Chapter Outlines


3. INTRODUCTION3.1. Context and Background3.2. Overview of Genome Editing3.3. History of Genome Editing3.4. Applications of Genome Editing3.5. Genome Editing Techniques3.5.1. Mutagenesis3.5.2 Conventional Homologous Recombination3.5.3 Single Stranded Oligo DNA Nucleotides Homologous Recombination3.5.4. Homing Endonuclease Systems (Adeno Associated Virus System)3.5.5. Protein-based Nuclease Systems3.5.5.1. Meganucleases3.5.5.2. Zinc Finger Nucleases3.5.5.3. Transcription Activator-like Effector Nucleases3.5.6. DNA Guided Systems3.5.6.1. Peptide Nucleic Acids3.5.6.2. Triplex Forming Oligonucleotides3.5.6.3. Structure Guided Endonucleases3.5.7. RNA Guided Systems3.5.7.1. CRISPR-Cas93.5.7.2. Targetrons3.6. CRISPR-based Genome Editing3.6.1. Role of CRISPR-Cas in Adaptive Immunity in Bacteria3.6.2. Key CRISPR-Cas Systems3.6.3. Components of CRISPR-Cas System3.6.4. Protocol for CRISPR-based Genome Editing3.7. Applications of CRISPR3.7.1. Development of Therapeutic Interventions3.7.2. Augmentation of Artificial Fertilization Techniques3.7.3. Development of Genetically Modified Organisms3.7.4. Production of Biofuels3.7.5. Other Bioengineering Applications3.8. Key Challenges and Future Perspectives

4. CRISPR-BASED GENOME EDITING SERVICE PROVIDERS: CURRENT MARKET LANDSCAPE4.1. Chapter Overview4.2. CRISPR-based Genome Editing Service Providers: Overall Market Landscape4.2.3. Analysis by Type of Service Offering4.2.4. Analysis by Type of gRNA Format4.2.5. Analysis by Type of Endonuclease4.2.6. Analysis by Type of Cas9 Format4.2.7. Analysis by Type of Cell Line Engineering Offering4.2.8. Analysis by Type of Animal Model Generation Offering4.2.9. Analysis by Availability of CRISPR Libraries4.2.10. Analysis by Year of Establishment4.2.11. Analysis by Company Size4.2.12. Analysis by Geographical Location4.2.13. Logo Landscape: Distribution by Company Size and Location of Headquarters

5. COMPANY COMPETITIVENESS ANALYSIS5.1. Chapter Overview5.2. Methodology5.3. Assumptions and Key Parameters5.4. CRISPR-based Genome Editing Service Providers: Competitive Landscape5.4.1. Small-sized Companies5.4.2. Mid-sized Companies5.4.3. Large Companies

6. COMPANY PROFILES6.1. Chapter Overview6.2. Applied StemCell6.2.1. Company Overview6.2.2. Service Portfolio6.2.3. Recent Developments and Future Outlook6.3. BioCat6.4. Biotools6.5. Charles River Laboratories6.6. Cobo Scientific6.7. Creative Biogene6.8. Cyagen Biosciences6.9. GeneCopoeia6.10. Horizon Discovery6.11. NemaMetrix6.12. Synbio Technologies6.13. Thermo Fisher Scientific

7. PATENT ANALYSIS7.1. Chapter Overview7.2. Scope and Methodology7.3. CRISPR-based Genome Editing: Patent Analysis7.3.1. Analysis by Application Year and Publication Year7.3.2. Analysis by Geography7.3.3. Analysis by CPC Symbols7.3.4. Emerging Focus Areas7.3.5. Leading Players: Analysis by Number of Patents7.4. CRISPR-based Genome Editing: Patent Benchmarking Analysis7.4.1. Analysis by Patent Characteristics7.5. Patent Valuation Analysis

8. ACADEMIC GRANT ANALYSIS8.1. Chapter Overview8.2. Scope and Methodology8.3. Grants Awarded by the National Institutes of Health for CRISPR-based8.3.1. Year-wise Trend of Grant Award8.3.2. Analysis by Amount Awarded8.3.3. Analysis by Administering Institutes8.3.4. Analysis by Support Period8.3.5. Analysis by Funding Mechanism8.3.6. Analysis by Type of Grant Application8.3.7. Analysis by Grant Activity8.3.8. Analysis by Recipient Organization8.3.9. Regional Distribution of Grant Recipient Organization8.3.10. Prominent Project Leaders: Analysis by Number of Grants8.3.11. Emerging Focus Areas8.3.12. Grant Attractiveness Analysis

9. CASE STUDY: ADVANCED CRISPR-BASED TECHNOLOGIES/SYSTEMS AND TOOLS9.1. Chapter Overview9.2. CRISPR-based Technology Providers9.2.1. Analysis by Year of Establishment and Company Size9.2.2. Analysis by Geographical Location and Company Expertise9.2.3. Analysis by Focus Area9.2.4. Key Technology Providers: Company Snapshots9.2.4.1. APSIS Therapeutics9.2.4.2. Beam Therapeutics9.2.4.3. CRISPR Therapeutics9.2.4.4. Editas Medicine9.2.4.5. Intellia Therapeutics9.2.4.6. Jenthera Therapeutics9.2.4.7. KSQ Therapeutics9.2.4.8. Locus Biosciences9.2.4.9. Refuge Biotechnologies9.2.4.10. Repare Therapeutics9.2.4.11. SNIPR BIOME9.2.5. Key Technology Providers: Summary of Venture Capital Investments9.3. List of CRISPR Kit Providers9.4. List of CRISPR Design Tool Providers

10. POTENTIAL STRATEGIC PARTNERS10.1. Chapter Overview10.2. Scope and Methodology10.3. Potential Strategic Partners for Genome Editing Service Providers10.3.1. Key Industry Partners10.3.1.1. Most Likely Partners10.3.1.2. Likely Partners10.3.1.3. Less Likely Partners10.3.2. Key Non-Industry/Academic Partners10.3.2.1. Most Likely Partners10.3.2.2. Likely Partners10.3.2.3. Less Likely Partners

11. MARKET FORECAST11.1. Chapter Overview11.2. Forecast Methodology and Key Assumptions11.3. Overall CRISPR-based Genome Editing Services Market, 2019-203011.4. CRISPR-based Genome Editing Services Market: Distribution by Regions, 2019-203011.4.1. CRISPR-based Genome Editing Services Market in North America, 2019-203011.4.2. CRISPR-based Genome Editing Services Market in Europe, 2019-203011.4.3. CRISPR-based Genome Editing Services Market in Asia Pacific, 2019-203011.4.4. CRISPR-based Genome Editing Services Market in Rest of the World, 2019-203011.5. CRISPR-based Genome Editing Services Market: Distribution by Type of Services, 2019-203011.5.1. CRISPR-based Genome Editing Services Market for gRNA Construction, 2019-203011.5.2. CRISPR-based Genome Editing Services Market for Cell Line Engineering, 2019-203011.5.3. CRISPR-based Genome Editing Services Market for Animal Model Generation, 2019-203011.6. CRISPR-based Genome Editing Services Market: Distribution by Type of Cell Line, 2019-203011.6.1. CRISPR-based Genome Editing Services Market for Mammalian Cell Lines, 2019-203011.6.2. CRISPR-based Genome Editing Services Market for Microbial Cell Lines, 2019-203011.6.3. CRISPR-based Genome Editing Services Market for Other Cell Lines, 2019-2030

12. SWOT ANALYSIS12.1. Chapter Overview12.2. SWOT Analysis12.2.1. Strengths12.2.2. Weaknesses12.2.3. Opportunities12.2.4. Threats12.2.5. Concluding Remarks




Companies Mentioned

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Genome Editing Services, World Markets to 2030: Focus on CRISPR - The Most Popular Genome Manipulation Technology Tool - PRNewswire

Visiongain Report Looks at Opportunities within the $1.8bn Acute Myeloid Leukaemia Market – P&T Community

- Global Acute Myeloid Leukaemia Market Forecast to 2029

- Chemotherapy, Targeted Therapy, Cytarabine, Daunorubicin, Midostaurin, Enasidenib

LONDON, Nov. 28, 2019 /PRNewswire/ -- The global acute myeloid leukaemia market is estimated to grow at a CAGR of 13% in the first half of the forecast period. In 2018, the chemotherapy segment held 32% share of the global acute myeloid leukaemia market.

How this report will benefit youRead on to discover how you can exploit the future business opportunities emerging in this sector.

In this brand new 149-page report you will receive 143 charts all unavailable elsewhere.

The 149-page Visiongain report provides clear detailed insight into the acute myeloid leukaemia market. Discover the key drivers and challenges affecting the market.

By ordering and reading our brand-new report today you stay better informed and ready to act.

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Report Scope

Global Acute Myeloid Leukaemia Market forecast to 2029

Forecast of the Global Acute Myeloid Leukaemia market byTreatment Type and Product Type: Chemotherapy: Cytarabine, Daunorubicin, Others Targeted Therapy: Midostaurin, Enasidenib, Others

This report provides individual revenue forecasts to 2029 for these regional andnational markets: North America: US, Canada, and Mexico Europe: Germany, France, United Kingdom, Italy, Spain and rest of Europe Asia-Pacific: Japan, China, India, Australia and Rest of Asia-Pacific LAMEA: GCC, Brazil, South Africa, Rest of LAMEA

Each national market forecast is further segmented by type: chemotherapy and targeted therapy.

Our study discusses the selected leading companies that are the major players in the acute myeloid leukaemia market: Bristol-Myers Squibb Company Celgene Clavis Pharma ASA Daiichi Sankyo Eisai GSK Novartis Roche Sunesis Pharmaceuticals Teva

This report discusses factors that drive and restrain the acute myeloid leukaemia market. This report also discusses the opportunities that can be tapped in this market.

This report discussesPorter's Five Forces analysis of the acute myeloid leukaemia market.

This report also discusses several new agents that are in development for the treatment of acute myeloid leukaemia.

Key questions answered in this report: How is the Acute myeloid leukemia market evolving? What are the drivers and restraints for the growth of the acute myeloid leukemia market? What are the market shares of each segment of the overall acute myeloid leukemia market in 2019? How will each acute myeloid leukemia submarket segment grow over the forecast period and how much revenue will these submarkets account for in 2029? How will the market shares for each acute myeloid leukemia submarket develop from 2019 to 2029? What is the value of the leading acute myeloid leukemia segments in important regions of the world? What will be the main driver for the overall market from 2019 to 2029? How will political and regulatory factors influence the regional markets and submarkets? How will the market shares of the national markets change by 2029 and which geographical region will lead the market by 2029? Who are the leading players and what are their prospects over the forecast period? How will the industry evolve during the period between 2019 and 2029?

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Did you know that we also offer a report add-on service? Email sara.peerun@visiongain.comto discuss any customized research needs you may have.

Companies covered in the report include:

AbbVieAgios Pharmaceuticals, Inc.Ambit Biosciences CorporationAmgenAnimas CorporationAstexB. Braun Melsungen AGBaxter, Becton, Dickinson and Company (BD)BayerBoehringer IngelheimBristol-Myers Squibb Company (Bristol-Myers)Celator Pharmaceuticals, Inc.CelgeneClavis Pharma ASA (Clavis Pharma)CyclacelDaiichi PharmaceuticalDaiichi SankyoEisai Co., Ltd. (Eisai)F. Hoffmann-La Roche Ltd. (Roche)Fresenius SE & Co. KGaAGlaxoSmithKline plc (GSK)GLOBOCANHospiraMedtronicMoog Inc.NeoMedNorsk HydroNovartisOnyx PharmaceuticalsSankyoSeattle GeneticsSmiths Group Plc.,Sunesis Pharmaceuticals, Inc. (Sunesis)TEVA Pharmaceutical Industries Limited (TEVA)

List of Organizations Mentioned in the ReportAmerican Cancer OrganizationBaylor Scott and White Research InstituteBenaroya Research Institute at Virginia MasonBeth Israel Deaconess Medical CenterBreslin Cancer CenterCenter for Blood Disorders and Stem Cell TransplantationCharles A. Sammons Cancer CenterColumbia University MedicalDana Farber Cancer InstituteDuke Cancer CenterEuropean CommissionFred Hutchinson Cancer Research CenterHealth CanadaHerbert Irving Comprehensive Cancer CenterInstitute of Hematology & Blood Diseases Hospital,TianjinJames Graham Brown Cancer CenterLegacy Emanuel Hospital & Health CenterMary Babb Randolph Cancer CenterMassachusetts general HospitalMayo ClinicMD Anderson Cancer CenterMedical College of Wisconsin Cancer CenterMedizinische Klinik und Poliklinik IMemorial Sloan Kettering Cancer CenterMichigan State UniversityNantes University HospitalNational Cancer InstituteNew Jersey Hematology Oncology AssociatesNorthwestern UniversityOchsner Medical CenterPenn State Hershey Cancer Institute-Clinical Trials OfficePerelman Center for Advanced MedicinePrincess Margaret HospitalSt. Francis Cancer CenterSt. Joseph Mercy HospitalSt. Vincent's Comprehensive Cancer CenterSwedish Cancer InstituteThe Gayle and Tom Benson Cancer CenterThe University of Hong KongThe University of IowaThe University of Texas MD Anderson Cancer CenterThomas Jefferson UniversityU.S. Food and Drug Administration (FDA)UCLA Medical Center, Division of Hematology/OncologyUniversity Hospital, CaenUniversity of Kansas Cancer CenterUniversity of Kansas Medical Center Research InstituteUniversity of KentuckyUniversity of LouisvilleUniversity of Michigan Health SystemUniversity of PennsylvaniaUniversity of Texas M.D. Anderson Cancer CenterVA Caribbean Healthcare SystemWeill Cornell Medical CollegeWeill Medical College of Cornell UniversityWest Virginia UniversityWorld Health Organization (WHO)

To see a report overview please e-mail Sara Peerun on

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Visiongain Report Looks at Opportunities within the $1.8bn Acute Myeloid Leukaemia Market - P&T Community

The FutureAnd the End?of AIDS – Columbia University Irving Medical Center

Throughout much of the world, a diagnosis of HIV/AIDS that once meant a certain death sentence now means living with a chronic, controllable disease. If you think about the fact that this epidemic was only recognized in 1981, the progress has been enormous, says David Ho, MD, the AIDS pioneer who was recruited to VP&S this year as the Clyde56 and Helen Wu Professor of Medicine. We do not have a vaccine. We do not have a cure. But we have turned a deadly disease into a manageable condition.

Ho has witnessed that transformation firsthand. He saw some of the earliest AIDS cases in the United States as a resident at Cedars-Sinai Medical Center in Los Angeles in 1980. I will always remember the young man who came to the hospital with a multitude of infections, he recalls of his first case. He was treated but, nevertheless, died within a few weeks after leaving the hospital.

Soon, another young man with pneumonia and other infections came to the hospital, was treated, and, again, died very quickly. It was this second, then third case, that began to raise alarm, says Ho. Within a year, those patients were included in the first case report submitted to the Centers for Disease Control and Prevention on what would become known as the AIDS epidemic.

What he saw in the clinic moved Ho to pursue HIV research throughout the 1980s. His renown grew, and by 1990 Ho had been named scientific director and chief executive officer of the Aaron Diamond AIDS Research Center, or ADARC, the largest independent nonprofit organization dedicated to basic research in HIV/AIDS. In 2019, ADARC moved to Columbia University Irving Medical Center.

Ho's work has played a key role in what is now known about HIV. He helped elucidate the nature of HIV replication, developed a number of antiretroviral drugs, and at the 1996 International AIDS Conference presented his teams breakthrough study results that proved combination therapy could reduce HIV viral loads to undetectable levels for at least one year. That was a turning point in the treatment efforts, says Ho.

Much of ADARCs research focuses on antibodies to prevent HIV transmission. One antibody in particular, engineered by ADARC six years ago, obstructs viral entry into the host cell. It turns out to be extremely powerful in blocking HIV infection in the lab and in laboratory animals, says Ho. He and his team have initiated a year-long, phase 1 trial of the antibody in infected patients and healthy volunteers. The trial is also being conducted at Columbia, led by Magdalena Sobieszczyk, MD, chief of the infectious diseases division.

If all goes well in this and subsequent human trials, Ho foresees the antibody being delivered every few months by subcutaneous injection to protect against HIV infection, much like a long-acting contraceptive prevents pregnancy.

Hos antibody research is one vital endeavor amid a broader and urgent effort to prevent new HIV infections worldwide, and ADARC is joining existing Columbia research programs with the same goal. The goal announced by the U.S. president in early 2019to end AIDS including decreasing the number of new HIV infections by 90% by 2030 in the United Statesis a bold one, notes University Professor and global director for ICAP at Columbia University Wafaa El-Sadr, MD. While there has been a huge scale-up in terms of treatment domestically and globally, we are not on track to reach the goal of HIV prevention in terms of the number of new infections. More than a million are still reported globally every year.

In May, the New England Journal of Medicine published a perspective on the 2030 target by El-Sadr and coauthored by Miriam Rabkin, MD, associate professor of medicine and of epidemiology (in Columbias ICAP) with colleagues at the Fenway Institute and the University of West Virginia. HIV affects the most vulnerable among us, they noted, highlighting the disproportionate number of new cases among people of color, transgender people, people in rural areas who use injectable drugs such as opioids, and those most affected by poverty and unstable housing.

The medical community already has the tools to overcome these challenges, says El-Sadr, who is co-principal investigator of the NIH-funded HIV Prevention Trials Network, or HPTN. In 2016, an HPTN study confirmed that HIV treatment is, itself, a highly effective form of prevention. When you treat people living with HIV, she says, not only does the person being treated benefit, but you also decrease the risk of transmission of HIV to others. Thus, the scale-up of HIV treatment has been a fundamental global priority championed as well by ICAP in the countries where it works.

For individuals who are HIV-negative, several preventive options are now available. An important one is pre-exposure prophylaxis, or PrEP, an approach where persons can protect themselves by taking a daily pill. Post-exposure prophylaxis, on the other hand, offers protection by taking medications after a suspected sexual or occupational exposure to prevent infection.

As co-PI of HPTN, El-Sadr designs and implements HIV prevention research across the United States and in Latin America, Africa, and Asia. Our goal is to design the best possible research to identify new prevention tools and determine how to use them. Then, importantly, we need to get what we know works to the people who need it so we can demonstrate the benefits at a population level. The same values infuse ICAP, the Columbia-based global health center that El-Sadr founded in 2003 to develop and deliver comprehensive, family-focused HIV services and evidence-based initiatives to bolster national health systems.

Despite more positive attitudes toward people living with HIV, El-Sadr notes that stigma still impedes testing, status disclosures, and access to prevention and treatment among many people in high-risk populations. Keep in mind that some of these same populations, even without HIV, are stigmatized, she says. If you are a young man having sex with men in a Southern rural community in the United States, if you are a transgender woman, even in New York City, your whole outlook on life is shaped by prevailing stigma and discrimination. Adding HIV makes it doubly difficult in so many ways.

To fight back, El-Sadr marshals creativity and engagement. Many of the ICAP-led projects she has created train and empower peer educators to share their own experiences, talk about how they overcame similar challenges, and encourage others to participate in research studies as well as programs. That approach has been the backbone of her work since the late 1980s, when she established the first HIV research unit at Harlem Hospital. Peer educators are from the same communities as those we seek to serve. They know what is going to resonate. They also know how to model behaviors. They have been a huge part of our work, whether it be in Tanzania, Swaziland, Kazakhstan, or right here in New York.

Outreach has been central to the peer educators roles, says El Sadr. For example, in Tanzania, the ICAP-supported teams work to reach those most disenfranchised, going wherever they are needed and doing whatever is needed. They organize festive campaigns in village centers, visit artisanal miners, brothels, drug dens, fishing villages where the population is transient. Carrying backpacks loaded with supplies, peer educators talk to people, gain their trust, set up mobile units and tents, or do whatever is needed to get them the prevention and treatment they need. If you get out of the comfort zone of health facilities, clinics, hospitals, or research labs, get creative and collaborative, and sit with the people, listen to their concerns, speak in their language and offer them prevention, offer them treatment, right where theyneed it, guess what? They are quite willing to engage.

With nearly four decades of experience in the field, El-Sadr counsels diligence to achieve the ambitious U.S. HIV goals. First, she says, adequate funding is critically important. Second, the affected communities must be fully engaged. Third, resources and actions must be focused on the populations and locations where they are needed, informed by evidence and data. And finally, hard science must drive policy. We need to do what is supported by evidence, she says. There should be no room for political expediency.

Ho takes a cautiously optimistic stance on the 2030 target. It is possible, but a daunting task, he says. It requires political leadership, political will, and making sure that resources are properly given to the effort.

Over the past decade, ADARC has received nearly $50 million collectively from the Gates Foundation and the NIH to advance its research, with more to come. I think we could move the work we are doing with antibodies into the clinic and into at-risk communities to block HIV transmission even more effectively, says Ho. And I believe over the next five or 10 years, we are going to see tremendous progress in preventing HIV infection, not just with drugs like PrEP, but with antibodies and perhaps even with vaccines.

Decades of innovations, action, and partnerships have ushered in a new era in the global HIV responseone that could mean the end of AIDS. We are at a moment in history where we know enough to stem this epidemic, and we need to take what we know into action, says El-Sadr. At the same time, we must continue to seek new discoveries through research in the laboratory, in the clinic, and in the community. Both discovery and action are needed as we move forward.

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Researchers discover a stem cell therapy that can help heal injured heart – ANI News

ANI | Updated: Nov 29, 2019 20:58 IST

Washington D.C. [USA], Nov 29 (ANI): Researchers have discovered a stem cell therapy that might help the heart recuperate from an attack.This study published in the journal Nature reported that injecting living or even dead heart stem cells into the injured hearts of mice triggers an acute inflammatory process, which in turn generates a wound healing-like response to enhance the mechanical properties of the injured area.Mediated by macrophage cells of the immune system, the secondary healing process provided a modest benefit to heart function after a heart attack, according to the principal investigator Jeffery Molkentin, PhD, director of Molecular Cardiovascular Microbiology a Cincinnati Children's Hospital Medical Center and a professor of the Howard Hughes Medical Institute (HHMI)."The innate immune response acutely altered cellular activity around the injured area of the heart so that it healed with a more optimized scar and improved contractile properties," Molkentin said.The findings build on a 2014 study published by the same research team. As in that earlier study, the current paper shows that injecting c-kit positive heart stem cells into damaged hearts as a strategy to regenerate cardiomyocytes doesn't work.The findings prompted Molkentin and his colleagues to conclude that there is a need to "re-evaluate the current planned cell therapy based clinical trials to ask how this therapy might really work."Researchers worked with two types of heart stem cells currently used in the clinical trials -- bone marrow mononuclear cells and cardiac progenitor cells.As they went through the process of testing and re-verifying their data under different conditions, they were surprised to discover that in addition to the two types of stem cells, injecting dead cells or even an inert chemical called zymosan also provided benefit to the heart by optimizing the healing process. Zymosan is a substance designed to induce an innate immune response.They reported that stem cells or zymosan therapies tested in this study altered immune cell responses that significantly decreased the formation of extracellular matrix connective tissue in the injury areas, while also improving the mechanical properties of the scar itself.Researchers also found that stem cells and other therapeutic substances like zymosan have to be injected directly into the hearts surrounding the area of infarction injury."Most of the current trials were also incorrectly designed because they infuse cells into the vasculature. Our results show that the injected material has to go directly into the heart tissue flanking the infarct region. This is where the healing is occurring and where the macrophages can work their magic," Molkentin explained. (ANI)

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Revolutionizing Injury Recovery With Tendon Stem Cells – Technology Networks

The buildup of scar tissue makes recovery from torn rotator cuffs, jumpers knee, and other tendon injuries a painful, challenging process, often leading to secondary tendon ruptures. New research led by Carnegies Chen-Ming Fan and published inNature Cell Biologyreveals the existence of tendon stem cells that could potentially be harnessed to improve tendon healing and even to avoid surgery.

Tendons are connective tissue that tether our muscles to our bones, Fan explained. They improve our stability and facilitate the transfer of force that allows us to move. But they are also particularly susceptible to injury and damage.

Unfortunately, once tendons are injured, they rarely fully recover, which can result in limited mobility and require long-term pain management or even surgery. The culprit is fibrous scars, which disrupt the tissue structure of the tendon.

Working with Carnegies Tyler Harvey and Sara Flamenco, Fan revealed all of the cell types present in the Patellar tendon, found below the kneecap, including previously undefined tendon stem cells.

Because tendon injuries rarely heal completely, it was thought that tendon stem cells might not exist, said lead author Harvey. Many searched for them to no avail, but our work defined them for the first time.

Stem cells are blank cells associated with nearly every type of tissue, which have not fully differentiated into a specific functionality. They can also self-renew, creating a pool from which newly differentiated cell types can form to support a specific tissues function. For example, muscle stem cells can differentiate into muscle cells. But until now, stem cells for the tendon were unknown.

Surprisingly, the teams research showed that both fibrous scar tissue cells and tendon stem cells originate in the same spacethe protective cells that surround a tendon. Whats more, these tendon stem cells are part of a competitive system with precursors of fibrous scars, which explains why tendon healing is such a challenge.

The team demonstrated that both tendon stem cells and scar tissue precursor cells are stimulated into action by a protein called platelet-derived growth factor-A. When tendon stem cells are altered so that they dont respond to this growth factor, then only scar tissue and no new tendon cells form after an injury.

Tendon stem cells exist, but they must outcompete the scar tissue precursors in order to prevent the formation of difficult, fibrous scars, Fan explained. Finding a therapeutic way to block the scar-forming cells and enhance the tendon stem cells could be a game-changer when it comes to treating tendon injuries.

Reference: Harvey, Flamenco and Fan. 2019.A Tppp3+Pdgfra+ tendon stem cell population contributes to regeneration and reveals a shared role for PDGF signalling in regeneration and fibrosis. Nature Cell Biology.DOI:

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New Link Discovered Between Cells That Burn Fat and Colon Cancer – Clinical OMICs News

A new study by Rutgers University researchers suggests that two genes expressed in the intestinal cells that line the inside of the colon may also be involved in cancer development.

Recent studies have shown that intestinal stem cells can increase in animals on a high fat Western diet, potentially explaining an elevated cancer risk from such a diet.Diet being able to control cell proliferation is an interesting research development, particularly the convergence of dietary factors and dysregulated gene signaling driving malignant transformations and promoting an adenoma-to-adenocarcinoma progression.

This new study suggests a novel connection between HNF4A and HNF4G genes, diet and cancer.Genetic expression of HNF4 has previously been shown by to be heavily influenced by the gut microbiota, which in turn can influence a multitude of intestinal disorders.

Non-host gene regulation was further explored in this study by using a high fat diet to test how these genes work, and the researchers discovered they help co-regulate stem cell proliferation, as well as help intestine cells burn dietary fat. This was done by isolating cells from knockout and control mice and observing intestine stem cell proliferation under conditions of high fat and control. Mice that had both HNF4A and HNF4G knocked out were unable to have their stem cells proliferate under high fat conditions.

Intestinal stem cells undergo constant renewal and fuel the continuous turnover of the lining of the intestine. People naturally lose millions of intestinal cells daily, much like they lose skin cells. If this rate of replication is not closely controlled, it can quickly lead to malignancy. Lack of proliferation can be very problematic for the colon and damaging to lower layers of cells.

This [research] is important because scientists have shown that when theres too much dietary fat in the intestine, stem cell numbers increase, boosting susceptibility to colon cancer, said senior author Michael Verzi, an associate professor in the Department of Genetics in the School of Arts and Sciences at Rutgers UniversityNew Brunswick.

Rutgers scientists believe HNF4A and HNF4G help stem cells burn fat, providing them energy. By linking gene activation, cell replication number, diet and cancer risk, scientists might be able to better understand the cancer development process in high risk patients. Going forward, the researchers plan to continue studying whether these two genes alter stem cell numbers and cancer risk alongside a high fat diet, said Verzi.

Colorectal cancer (of the colon or rectum) is the third most common cancer diagnosed in both men and women in the United States. According to the American Cancer Society, over 100,000 Americans will be diagnosed with colon cancer this year. This cancer is also the second most deadliest in the United States, but due to a combination of increased screening and heightened awareness the death rate has been dropping. However, in patients under the age of 55, the death rate of colon cancer has increased each year by 1% since 2007. Approximately 50,000 colon cancer patients are expected to die in 2019.

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How birds fly: New USC study examines the evolution of flight feathers – USC News

Birds of a feather may flock together, but the feathers of birds differ altogether.

New research from an international team led by USC scientists set out to learn how feathers developed and helped birds spread across the world. Flight feathers, in particular, are masterpieces of propulsion and adaptation, helping penguins swim, eagles soar and hummingbirds hover.

Despite such diversity, the feather shares a common core design: a one-style-fits-all model with option trims for specialized performance. This simplicity and flexibility found in nature holds promise for engineers looking for better ways to build drones, wind turbines, medical implants and other advanced materials.

Those findings, published today in Cell, offer an in-depth look at the form and function of a feather based on a comparative analysis of their physical structure, cellular composition and evolution. The study compares feathers of 21 bird species from around the world.

Weve always wondered how birds can fly in so many different ways, and we found the difference in flight styles is largely due to the characteristics of their flight feathers, said Cheng-Ming Chuong, the studys lead author and a developmental biologist in the Department of Pathology at the Keck School of Medicine of USC. We want to learn how flight feathers are made so we can better understand nature and learn how biological architecture principles can benefit modern technology.

To gain a comprehensive understanding of the flight feather, Chuong formed a multi-disciplinary international team with Wen Tau Juan, a biophysicist at the Integrative Stem Cell Center, China Medical University in Taiwan. The work involved experts in stem cells, molecular biology, anatomy, physics, bioimaging, engineering, materials science, bioinformatics and animal science. The bird species studied include ostrich, sparrow, eagle, chickens, ducks, swallow, owl, penguin, peacock, heron and hummingbird, among others.

They compared feathers using fossils, stem cells and flight performance characteristics. They focused on the feather shaft, or rachis, that supports the feather much like a mast holds a sail, bearing the stress between wind and wing. They also focused on the vane, the lateral branches astride the shaft that give the feather its shape to flap the air. And they examined how evolution shaped the barbs, ridges and hooks that help a feather hold its form and lock with adjacent feathers like Velcro to form a wing. The goal was to understand how a simple filament appendage on dinosaurs transformed into a three-level branched structure with different functions.

We want to learn how flight feathers are made so we can better understand nature and learn how biological architecture principles can benefit modern technology.

Cheng-Ming Chuong

For birds such as ducks, eagles and sparrows that fly in different modes, the scientists noted significant differences in the feather shaft compared to ground-hugging birds. On the rigid exterior, the shaft cortex was thinner and lightweight, while the interior was filled with porous cells resembling bubble wrap, aligned into bands of various orientations and reinforced with ridges that operate like tiny lateral beams. Together, it forms a light, hollow and buoyant structure to enable flight. Cross-sections of feather shafts of different birds show highly specialized shapes and orientations of the inner core and outer cortex.

The flight feather is made of two highly adaptable architectural modules, light and strong materials that can develop into highly adaptable configurations, Chuong said.

The researchers discovered two different molecular mechanisms guiding feather growth. Cortex thickness was governed by bone morphogenetic proteins, which are molecular signals for tissue growth. The porous feather interior, or medulla, relied upon a different mechanism known as transforming growth factor-beta (TGF-b). Both components originate as stem cells in the birds skin.

By contrast, feathers in flightless birds were simpler, consisting of a dense cortex exterior that is more rigid and sturdy with fewer internal struts and cells found in flying birds. The features were especially pronounced for penguins, which use wings as paddles under the water.

As part of the study, the researchers looked at 100 million-year-old feathers, found embedded in amber in Myanmar. These fossils show early feathers lacked one key feature that modern birds have. Specifically, the researchers report that fossil feathers had barb branches and barbules, which form a feather vane by overlapping, but not hooklets. The hooklets, which act like clasps to turn fluffy feathers into a tight flat plane for high-performance flight, evolved later. The scientists also identified WNT2B, another growth factor, as the agent that controls hooklet formation. These also originated from epidermal stem cells.

Taken together, the findings show how feathered dinosaurs and early birds could form a primitive vane by overlapping barbule plates, although that wasnt aerodynamically fit to carry much load. As more complex composite features occurred in the wing, it got heavier, so feather shafts became stronger yet more lightweight, which led to stiffer feathers and sturdy wings that powered flight to carry birds around the world.

Our findings suggest the evolutionary trends of feather shaft and vane are balanced for the best flight performance of an individual bird and become part of the selective basis of speciation, the study said. The principles of functional architectures we studied here may also stimulate bio-inspired designs and fabrication of future composite materials for architectures of different scales, including wind turbines, artificial tissues, flying drones.

Chuong and Juan are co-leaders of the 31-person team, joined by co-authors Randall B. Widelitz, Shuo Wang, Michael Habib, Ting-Xin Jiang, Zhong-Lai Luo and Ping Wu of the Keck School of Medicine of USC; Wei-Ling Chang, Hao Wu, Yung-Chi Lai, Ming Xing Lei, and Shih-Chieh Hung of the China Medical University Hospital in Taiwan; Ming-You Shie, Jui-Ting Hsu, Heng-Li Huang and Yi-Wen Chen of the China Medical University, Taiwan; Chih-Feng Chen, Ping Chi Tang, Hus Chen Cheng, and Yen-Cheng Lin of the National Chung Hsing University in Taiwan; How-Jen Gu, Yu-Kun Chiu, Tse-Yu Lin, Shun-Min Yang, Tsung-Tse Lee, J.C. Tsai and Yeu-Kuang Hwu of the Institute of Physics, Academia Sinica, Taiwan; Cheng-Te Yao of the Endemic Species Research Institute, Taiwan; Shyh-Jou Shieh of the National Cheng Kung University, Taiwan; Ang Li of the University of Texas, Arlington.

Work at USC was supported by the National Institutes of Health (AR 047364, AR 060306) while team members in Taiwan were supported by grants from their own institutes and the Taiwan government.

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