Stem Cell Clinic

Father-of-three raises cash for last chance MS treatment – Pontefract and Castleford Express

By Stem Cell Treatment

Phil Swindin, from Darrington, was diagnosed with Multiple Sclerosis in 2004, and has suffered major problems with his health ever since.

His symptoms have now escalated, leaving him unable to walk more than a metre without support, and relying on caffeinated drinks for short bursts of energy.

He hopes to travel to a private facility in Mexico for a new type of treatment known as Autologous Haematopoietic Stem Cell Transplantation (AHSCT), which has been linked with improvements in MS.

Phil said: Living with MS is constant and terrifying, as you just get more and more disabled and whatever you do very little can be done.

My MS symptoms have fluctuated and reared its ugly head at the most inappropriate times and limited my normal physical or mental ability.

Its caused major hidden problems in mobility, mental health, energy levels and so many other symptoms.

I long to do simple things like doing things with my kids, playing football or going for a walk but its out of the question.

All other avenues have failed so Im left with only one option, to fundraise for private treatment.

Phil and his wife Alison launched the fundraiser last month, and have already raised more than 28,000.

The couple also hope to organise a ball in the Spring to help boost funding,

Alison said: People have just been wonderful. People who we havent spoken to for years have been in touch and their words of support are fantastic.

It sort of feels much more achievable than when we started it. Were just trying to keep it moving, with more things happening all the time.

Phils treatment is currently booked for April, but will have to be postponed if the funds are not raised before then.

Phil has also been supported by Ackworth mum-of-two Kate Dawson, who successfully underwent the treatment in 2017 and described the results as life-changing.

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Father-of-three raises cash for last chance MS treatment - Pontefract and Castleford Express

Moving story of bone marrow donor’s amazing 30 year bond with the man he saved – Mirror Online

By Stem Cell Treatment

There was a head-scratching moment when Martin Swales answered his front door and a priest handed him a letter.

The mystery was quickly solved. It contained a thank you note from someone whose life Martin had saved.

He knew his bone marrow had been given to someone called Jan and imagined it was a woman in Britain.

In fact the recipient was dad Jan Zemek 4,500 miles away in the US.

And Martins gift of life has led to an extraordinary 30-year bond between the pair, who are like blood brothers.

Jan named his second daughter Martina in honour of his hero and Martin is godfather to his third girl.

Retired welder Martin, 58, of Guisborough, North Yorks, said: Donating bone marrow didnt just save Jans life, it changed mine as well.

The first time I met Jan, I put my arms around him and he hugged me back.

It felt natural, like I was welcoming my brother. It feels like our two families have become one.

They each have three grown-up children and have visited each other for baptisms, graduations, and weddings.

Martin recently went to Switzerland, where Jan lives with his family, to celebrate 30 years since the transplant and present his blood brother with a Walk of Fame plaque.

It includes the touching message: Stood strong, fought hard, and won. You are a survivor.

The mens amazing and heart-warming story dates from 1986 when Martin joined the Anthony Nolan stem cell register after an appeal to save two girls living in the North East.

He was not a match for the girls but in 1989 was called by the register because he could be for Jan.

Martin said: It was quite a shock because Id pretty much forgotten about the register. They told me I was a possible match for someone and what was involved. I said yes straight away. I wanted to help if I could.

Despite the discomfort, Martin gave bone marrow from his hip at a clinic in Harley Street that August. Doctors extracted it from inside his hip using a long needle. Today most donations are no more invasive than giving blood.

Martin spent two nights in hospital. He said: It doesnt take long but at the time I was suffering from sciatica so I think I found it a bit more painful than most. It was an uncomfortable journey home on the train.Anthony Nolan covered the cost of the trip.

Jan, a 27-year-old dad, was diagnosed with leukaemia in 1987. Initially doctors kept the news from him as no treatment was available in the Czech Republic, where he lived.

Jan said: I was diagnosed one year after the Chernobyl tragedy, weve never known if that radiation was to blame for my cancer. I suddenly grew very tired, nobody knew the reason.

I didnt know how sick I was because the doctors wouldnt tell me.

My wife, who was then my girlfriend, went to the same doctors and they told her, Dont marry this guy, dont have children with him. He is going to die in two years.

But Radka ignored their warning and insisted on marrying Jan in 1987.

His only hope was a bone marrow transplant. Weeks later he left for the US with his dad, who planned to be his donor.

Jan said: A few months earlier, I read in the paper the opera singer Jos Carreras was diagnosed with a similar blood disease and was going to the same US centre for a transplant.

They arrived with less than 40 in their pockets and discovered a transplant from his dad would give Jan only a 15 per cent chance of survival.

Instead doctors advised them to find a donor. It took two years and 10,000 to test potential donors before they found a perfect match in Martin.

By then Jan and Radka had become parents to their first daughter, Jana.

Jan needed to raise more than 100,000 to fund the transplant.

He said: It was such a huge amount of money to raise but when you are dying you have no choice.

There were 12 rival local radio stations but they all got together to run a joint appeal, which they broadcast at the same time. It was incredible.

Jan did a sponsored run, gave talks about his ordeal to church congregations to request donations, and wrote to celebrities, especially those with links to the Czech Republic.

Donald Trump s ex-wife Ivana gave 1,000, as did One Flew Over the Cuckoos Nest director Milos Forman. Jan said: The response was crazy. So many people donated 20 dollars or 50.

Martins bone marrow was flown to the Fred Hutchinson Cancer Research Center in Seattle, where Jan was waiting in an isolation room.

He had been blasted with chemo and radiotherapy so his immune system would not attack Martins transplanted cells.

Normally, under strict anonymity rules to protect donor and recipient, Martin and Jan would have been unable to contact each other for years.

But a priest from the North East of England working at the hospital recognised Martins address when the bag of bone marrow arrived.

He offered to take a photo of Jan, a thank you letter, and a Czech garnet stone to Martin when he returned home in 1990.

Martin said: I was stunned. I had no idea my bone marrow had travelled so far. Knowing Id helped a young father, just like me, brought home how important it was and how easily it could have been me waiting for a stranger to save my life.

I wrote straight back. The priest also brought a letter from a couple whose daughter was in the same hospital.

Her transplant didnt work. Sadly she died, but they wrote to thank me for saving Jan. Responding to them was much harder. How do you find the right words?

Martin and Jan kept in touch. When Jans second daughter was born in 1991, he and Radka named her after Martin.

Jan said: How do you repay someone who saved your life? Naming our daughter after Martin was our way of showing him we would never forget what he did for us.

Hes not just the man who saved my life. He is a nice guy. Thats why were so close.

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Jan, 59, and his family moved to Switzerland, where he landed a job with a sports marketing firm that works with World Athletics.

In 1992 his job brought him to Crystal Palace in South London and he spent a few days with Martin and family.

Jans youngest daughter Michaela was born in 1995 and he invited Martin and his family to Switzerland for the baptism and asked him to be godfather.

The two families continued to visit each other and holidayed together in the Czech capital Prague. When Jans eldest, Jana, was studying at Newcastle University, she regularly spent weekends with Martin and his wife Tracey.

Martin said: It meant so much to visit Jan for the 30 anniversary of his transplant earlier this year.

"They showed us the sights and we went up the mountains. It was brilliant. I could never have imagined this when I joined the stem cell register all those years ago.

He added: I hope Martin and I will be able to celebrate another anniversary together in ten years.

The Anthony Nolan register matches potential donors to patients needing stem cell transplants and does vital research. To join, donate or find out more, see anthonynolan.org .

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Moving story of bone marrow donor's amazing 30 year bond with the man he saved - Mirror Online

Stem Cell Therapy Market 2020 Analysis by Treatment, Application, Technology, Therapy With Regional Outlook & Forecast To 2025 – The Picayune…

By Stem Cell Treatment

Growth of Global Stem Cell Therapy Market Size is favored by cumulative factors like development of advanced genome-based cell analysis techniques, soaring awareness of the therapeutic potency of stem cells, detection of novel stem cell lines, and developments in infrastructure related to stem cell banking and processing, and increasing public-private investments for development of stem cell therapies.

In the upcoming years, North America is likely to dominate the global stem cell therapy market share. The U.S. dominates the market which is followed by Canada. Stem cell therapy is powered to cure diseases like diabetes and cancer. There is an increase in occurence of such diseases and higher awareness for the treatment of such diseases is propelling stem cell therapy market. New product innovation & launching, increase in a number of clinical trials for evaluation of the therapeutic potential and higher adoption of these therapies & procedures is fueling market growth. Presence of numerous centers and organization that are engaged in R&D related to new stem cell therapy is likely to led lucrative market growth.

Get more insights at:Global Stem Cell Therapy Market 2020-2025

Europe holds second position in the lucrative market of stem cell therapy. This is attributed to development of efficient & advanced technologies and growing investment in healthcare industry.

The Asia Pacific is projected to exhibit the highest growth in the market. The key countries that are expected to experience fastest development are India and China. The higher adoption of grants by several funding agencies and government for scientific researches is anticipated to stimulate the regional market growth. Investment of leading players for launch and development of newer technologies and increase in healthcare expenditure are also anticipated to up surge the Asia-Pacific market growth of stem cell therapy.

The Middle East & Africa and Latin America regions are expected to show slow growth in the forecast period. Rising awareness about the SC therapies, growing expenditure in the healthcare sector, rapid development in the infrastructure of healthcare and adoption of western technologies and lifestyle is expected to fuel the growth of the stem cell therapy market.

In terms of types of global stem cell therapy market is bifurcated into autologous stem cell therapy and allogeneic stem cell therapy. The segment of allogeneic stem cell therapy is likely to command largest global stem cell therapy market share. This is attributed to easy production scale-up process, escalating commercialization of allogeneic stem cell therapy products, the extensive therapeutic applications of allogeneic stem cells and mounting number of clinical trials related to allogeneic stem cell therapies.

Based on therapeutic application, the global stem cell therapy industry has wide range of categories which includes musculoskeletal disorders, cardiovascular diseases, gastrointestinal diseases, wounds and injuries, surgeries, and other applications. The musculoskeletal disorders sector is anticipated to dominate the global stem cell therapy market by holding largest share. This is attributed to the high prevalence of bone & joint diseases, musculoskeletal disorders, escalating availability of stem cell-based products for the treatment of musculoskeletal disorders and higher patient preference for effective & early treatment strategies.

Get more details about Global Stem Cell Therapy Market:

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The global stem cell therapy market is niche industry with soaring global and local companies involved in the commercialization and development of stem cell therapy products. Anterogen Co., Ltd. (South Korea), MEDIPOST Co., Ltd. (South Korea), Osiris Therapeutics, Inc. (U.S.) and Pharmicell Co., Ltd. (South Korea) are the prominent market players of the global stem cell therapy market. The major strategies adopted by leading market players include expansions, partnerships, new product launches and approvals, and agreements to achieve growth in the global stem cell therapy market.

Key segments of Global Stem Cell Therapy Market

Based on cell source, the market has been segmented into,

Based on therapeutic application, the market has been segmented into,

Based on region, the market has been segmented into,

What to expect from the upcoming report on Global Stem Cell Therapy Market:

Future prospects and current trends of the global stem cell therapy market by the end of forecast period (2018-2025)

Information regarding technological progressions as well as innovations taking place in developing economies

Supportive initiatives by government likely to influence the market dynamics

Trends, drivers, opportunities, restraints, challenges and key developments in the market

In-depth analysis of different market segmentations including regional segmentations, and product types

Deep analysis about the competitive landscape of the market and the initiatives by them to improve this market

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BrainStorm Cell Therapeutics Wins 2020 ‘Buzz of BIO’ Award for ALS Investigational Therapy – ALS News Today

By Stem Cell Medicine

For its promising investigational therapeutic approach to neurodegenerative diseases such as amyotrophic lateral sclerosis (ALS), BrainStorm Cell Therapeutics is theBuzz of BIO 2020 winnerin the Public Therapeutic Biotech category.

The Buzz of BIO contest identifies U.S. companies with groundbreaking, early-stage potential to improve lives. The event also is anopportunity to make investor connections that could take products to the next phase.

Ten biotechnology companies are nominated in each of the three categories ofBuzz of BIO: Public Therapeutic Biotech, Private Therapeutic Biotech, and Diagnostics and Beyond. In the Public Therapeutic Biotech category that BrainStorm won, nominated companies must be actively developing a publicly traded human treatment intended for review by theU.S. Food and Drug Administration (FDA).

As a developer of autologous cellular therapies treatments that use a patients own cells and tissues for debilitating neurodegenerative diseases, BrainStorm is now testing its NurOwn therapy for safety and effectiveness. The treatment involves extracting, from human bone, marrow-derived mesenchymal stem cells (MSCs), which are capable of differentiating into other cell types. The MSCs are then matured into a specific cell type that produces neurotrophic factors compounds that promote nervous tissue growth and survival. They are then reintroduced to the body via injection into muscles and/or the spinal canal.

Backed by a California Institute for Regenerative Medicine grant, Brainstorm has fully enrolledits randomized, double-blind, placebo-controlled Phase 3 clinical trial (NCT03280056) at six U.S. sites in California, Massachusetts, and Minnesota. Some 200 ALS patients are participating. A secondary safety analysis by the trials independent Data Safety Monitoring Board (DSMB) revealed no new concerns. Every two months, study subjects will be given three injections into the spinal canal of either NurOwn or placebo.

The trial is expected to conclude late this year. Results will be announced shortly afterward.

In a Phase 2 study (NCT02017912), which included individuals with rapidly progressing ALS, NurOwn demonstrated a positive safety profile as well as prospective efficacy.

The use of autologous MSC cells to potentially treat ALS was given orphan drug status by both the FDA and the European Medicines Agency.

Thanks to everyone who voted for BrainStorm during the Buzz of BIO competition,Chaim Lebovits, BrainStorm president and CEO, said in a press release. The entire management team at BrainStorm was very pleased with the results of this competition, and we look forward to presenting to an audience of accredited investors who may benefit from the companys story. We thank the BIO[Biotechnology Innovation Organization] team for singling out BrainStorms NurOwn as a key technology with the potential to improve lives.

As a contest winner, BrainStorm is invited to givea presentation at theBio CEO & Investor Conference, to be held Feb. 1011 in New York City, along with exposure to multiple industry elites and potential investors.

NurOwn cells also are being tested in a Phase 2 clinical study (NCT03799718) in patients with progressive multiple sclerosis.

Mary M. Chapman began her professional career at United Press International, running both print and broadcast desks. She then became a Michigan correspondent for what is now Bloomberg BNA, where she mainly covered the automotive industry plus legal, tax and regulatory issues. A member of the Automotive Press Association and one of a relatively small number of women on the car beat, Chapman has discussed the automotive industry multiple times of National Public Radio, and in 2014 was selected as an honorary judge at the prestigious Cobble Beach Concours dElegance. She has written for numerous national outlets including Time, People, Al-Jazeera America, Fortune, Daily Beast, MSN.com, Newsweek, The Detroit News and Detroit Free Press. The winner of the Society of Professional Journalists award for outstanding reporting, Chapman has had dozens of articles in The New York Times, including two on the coveted front page. She has completed a manuscript about centenarian car enthusiast Margaret Dunning, titled Belle of the Concours.

Total Posts: 6

Ins holds a PhD in Biomedical Sciences from the University of Lisbon, Portugal, where she specialized in blood vessel biology, blood stem cells, and cancer. Before that, she studied Cell and Molecular Biology at Universidade Nova de Lisboa and worked as a research fellow at Faculdade de Cincias e Tecnologias and Instituto Gulbenkian de Cincia. Ins currently works as a Managing Science Editor, striving to deliver the latest scientific advances to patient communities in a clear and accurate manner.

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BrainStorm Cell Therapeutics Wins 2020 'Buzz of BIO' Award for ALS Investigational Therapy - ALS News Today

Cell and Advanced Therapies Supply Chain Management Industry Report, 2019-2030 – GlobeNewswire

By Stem Cell Medicine

Dublin, Jan. 03, 2020 (GLOBE NEWSWIRE) -- The "Cell and Advanced Therapies Supply Chain Management Market, 2019-2030: Focus on Technological Solutions" report has been added to ResearchAndMarkets.com's offering.

Cell and Advanced Therapies Supply Chain Management Market: Focus on Technological Solutions, 2019-2030 report features an extensive study of the growing supply chain management software solutions market.

The focus of this study is on software systems, including cell orchestration platforms (COP), enterprise manufacturing systems (EMS), inventory management systems (IMS), laboratory information management systems (LIMS), logistics management systems (LMS), patient management systems (PMS), quality management systems (QMS), tracking and tracing software (TTS), and other such platforms that are being used to improve / optimize various supply chain-related processes of cell and advanced therapies.

One of the key objectives of the report was to understand the primary growth drivers and estimate the future size of the supply chain management software solutions market. Based on multiple parameters, such as number of cell and advanced therapies under development, expected pricing, likely adoption rates, and potential cost saving opportunities from different software systems, we have developed informed estimates of the evolution of the market, over the period 2019-2030.

In addition, we have provided the likely distribution of the current and forecasted opportunity across:

Advanced therapy medicinal products, such as cell and gene therapies, have revolutionized healthcare practices. The introduction of such treatment options has led to a paradigm shift in drug development, production and consumption. Moreover, such therapies have actually enabled healthcare providers to treat several difficult-to-treat clinical conditions.

In the past two decades, more than 30 such therapy products have been approved; recent approvals include Zolgensma (2019), RECELL System (2018), AmnioFix (2018), EpiFix (2018), EpiBurn (2018), Alofisel (2018), LUXTURNA (2017), Yescarta (2017), and Kymriah (2017). Further, according to a report published by The Alliance for Regenerative Medicine in 2019, more than 1,000 clinical trials are being conducted across the globe by over 900 companies.

In 2018, around USD 13 billion was invested in this domain, representing a 73% increase in capital investments in this domain, compared to the previous year. It is worth highlighting that, based on an assessment of the current pipeline of cell therapies and the historical clinical success of such products, it is likely that around 10-20 advanced therapies are approved by the US FDA each year, till 2025.

The commercial success of cell and advanced therapies is not only tied to whether they are capable of offering the desired therapeutic benefits, but also on whether the developers are able to effectively address all supply chain requirements. The advanced therapy medicinal products supply chain is relatively more complex compared to the conventional pharmaceutical supply chain. As a result, there are a number of risks, such as possible operational inefficiencies, capacity scheduling concerns, process delays leading to capital losses, and deliverable tracking-related issues, which need to be taken into consideration by therapy developers.

This has generated a need for bespoke technological solutions, which can be integrated into existing processes to enable the engaged stakeholders to oversee and manage the various aspects of the cell and advanced therapies supply chain, in compliance to global regulatory standards. Over the years, several innovative, software-enabled systems, offering supply chain orchestration and needle-to-needle traceability, have been developed.

The market has also recently witnessed the establishment of numerous partnerships, most of which are agreements between therapy developers and software solutions providers. Further, given the growing demand for cost-effective personalized medicinal products, and a myriad of other benefits of implementing such software solutions, the niche market is poised to grow significantly in the foreseen future.

Amongst other elements, the report features:

In order to account for the uncertainties associated with some of the key parameters and to add robustness to our model, we have provided three market forecast scenarios portraying the conservative, base and optimistic tracks of the industry's evolution.

The opinions and insights presented in this study were influenced by discussions conducted with several stakeholders in this domain. The report features detailed transcripts of interviews held with the following individuals:

Key Topics Covered

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

2. EXECUTIVE SUMMARY

3. INTRODUCTION3.1. Context and Background3.2. An Introduction to Cell and Advanced Therapies3.2.1. Classification of Advanced Therapy Medicinal Products3.2.2. Current Market Landscape3.3. Cell and Advanced Therapies Supply Chain3.3.1. Key Processes3.3.2. Challenges Associated with the Cell and Advanced Therapies Supply Chain3.4. Software Solutions for Cell and Advanced Therapies Supply Chain Management3.4.1. Cell Orchestration Platform3.4.2. Enterprise Manufacturing System3.4.3. Inventory Management System3.4.4. Laboratory Information Management System3.4.5. Logistics Management System3.4.6. Patient Management System3.4.7. Quality Management System3.4.8. Tracking and Tracing System3.5. Growth Drivers and Roadblocks3.6. Emergence of Digital Technologies in Supply Chain Management3.6.1. Blockchain Technology3.6.2. Internet of Things3.6.3. Augmented Reality3.6.4. Big Data Analytics3.6.5. Artificial Intelligence

4. CURRENT MARKET LANDSCAPE4.1. Chapter Overview4.2. Cell and Advanced Therapies Supply Chain Management: Overall Market Landscape4.2.1. Analysis by Type of Software Solution4.2.2. Analysis by Key Specification and Benefit4.3.3. Analysis by Application4.3.4. Analysis by End User4.3.5. Analysis by Mode of Deployment4.3.6. Analysis by Scale of Management4.3.7. Analysis by Regulatory Certifications / Accreditations4.3. Cell and Advanced Therapies Supply Chain Management: Developer Landscape4.2.1. Analysis by Year of Establishment4.2.2. Analysis by Location of Headquarters4.2.3. Analysis by Size of Company4.3.4. Analysis by Support Services Offered4.3.5. Leading Developers: Analysis by Number of Software Solutions

5. COMPANY COMPETITIVENESS ANALYSIS5.1. Chapter Overview5.2. Methodology5.3. Assumptions and Key Parameters5.4. Competitiveness Analysis: Overview of Supply Chain Management Software Solution Providers5.4.1. Small-sized Companies5.4.2. Mid-sized Companies5.4.3. Large Companies

6. CORE SUPPLY CHAIN MANAGEMENT SOFTWARE SOLUTIONS: COMPANY PROFILES6.1. Chapter Overview6.2. Brooks Life Sciences6.2.1. Company Overview6.2.2. Financial Information6.2.3. BiobankPro: Software Description6.2.4. Recent Developments and Future Outlook6.3. Cryoport6.3.1. Company Overview6.3.2. Financial Information6.3.3. Cryoportal: Software Description6.3.4. Recent Developments and Future Outlook6.4. MasterControl6.4.1. Company Overview6.4.2. MasterControl Platform: Software Description6.4.3. Recent Developments and Future Outlook6.5. SAP6.5.1. Company Overview6.5.2. Financial Information6.5.3. SAP S/4HANA: Software Description6.5.4. Recent Development and Future Outlook6.6. Savsu Technologies6.6.1. Company Overview6.6.2. Financial Information6.6.3. evo Cold Chain 2.0: Software Description6.6.4. Recent Development and Future Outlook6.7. TraceLink6.7.1. Company Overview6.7.2. Financial Information6.7.3. Digital Supply Chain Platform: Software Description6.7.4. Recent Developments and Future Outlook

7. CELL ORCHESTRATION PLATFORMS: EMERGING TRENDS AND PROFILES OF KEY PLAYERS7.1. Chapter Overview7.2. Supply Chain Orchestration Platforms7.2.1. Key Functions of Supply Chain Orchestration Platforms7.2.2. Advantages of Supply Chain Orchestration Platforms7.2.3. Supply Chain Orchestration Platform Implementation Strategies7.3. Supply Chain Orchestration Platform: Trends on Twitter7.3.1. Scope and Methodology7.3.2. Historical Trends in Volume of Tweets7.3.3. Popular Keywords7.4. Key Industry Players7.4.1. Be The Match BioTherapies7.4.2. Clarkston Consulting7.4.3. Haemonetics7.4.4. Hypertrust Patient Data Care7.4.5. Lykan Bioscience7.4.6. MAK-SYSTEM7.4.7. sedApta Group7.4.8. Stafa Cellular Therapy7.4.9. Title 21 Health Solutions7.4.10. TrakCel7.4.11. Vineti

8. FUNDING AND INVESTMENT ANALYSIS8.1. Chapter Overview8.2. Types of Funding8.3. Cell and Advanced Therapies Supply Chain Management: Recent Funding Instances8.3.1. Analysis by Number of Funding Instances8.3.2. Analysis by Amount Invested8.3.3. Analysis by Type of Funding8.3.4. Analysis by Number of Funding Instances and Amount Invested across Different Software Solutions8.3.5. Most Active Players: Analysis by Amount Invested8.3.6. Most Active Investors: Analysis by Participation8.3.7. Geographical Analysis by Amount Invested8.4. Concluding Remarks

9. PARTNERSHIPS AND COLLABORATIONS9.1. Chapter Overview9.2. Partnership Models9.3. Cell and Advanced Therapies Supply Chain Management: Recent Collaborations and Partnerships9.3.1. Analysis by Year of Partnership9.3.2. Analysis by Type of Partnership9.3.3. Analysis by Partner's Focus Area9.3.4. Analysis by Type of Software Solution9.3.5. Most Active Players: Analysis by Number of Partnerships9.3.6. Analysis by Regions

10. PLATFORM UTILIZATION USE CASES10.1. Chapter Overview10.2. Cell and Advanced Therapies Supply Chain Management: Recent Platform Utilization Use Cases10.2.1. Analysis by Year of Utilization10.2.2. Analysis by User's Focus Area10.2.3. Analysis by Type of Software Solution10.2.4. Most Active Players: Analysis by Number of Utilization Instances10.2.5. Most Active Players: Regional Analysis by Number of Utilization Instances

11. VALUE CHAIN ANALYSIS11.1. Chapter Overview11.2. Cell and Advanced Therapies Value Chain11.2. Cell and Advanced Therapies Value Chain: Cost Distribution11.3.1. Donor Eligibility Assessment11.3.2. Sample Collection11.3.3. Manufacturing11.3.4. Logistics11.3.5. Patient Verification and Treatment Follow-up

12. STAKEHOLDER NEEDS ANALYSIS12.1. Chapter Overview12.2. Cell and Advanced Therapies Supply Chain Management: Needs of Different Stakeholders12.2.1. Comparison of Stakeholder Needs

13. COST SAVINGS ANALYSIS13.1. Chapter Overview13.2. Key Assumptions and Methodology13.3. Overall Cost Saving Potential of Supply Chain Management Software Solutions, 2019-203013.3.1. Cost Saving Potential in Donor Eligibility Assessment, 2019-203013.3.2. Cost Saving Potential in Sample Collection, 2019-203013.3.3. Cost Saving Potential in Manufacturing, 2019-203013.3.4. Cost Saving Potential in Logistics, 2019-203013.3.5. Cost Saving Potential in Patient Verification and Treatment Follow-up, 2019-2030

14. MARKET FORECAST14.1. Chapter Overview14.2. Key Assumptions and Forecast Methodology14.3. Overall Cell and Advanced Therapies Supply Chain Management Solutions Market, 2019-203014.3.1. Overall Cell and Advanced Therapies Supply Chain Management Solutions Market: Distribution by Application14.3.2. Overall Cell and Advanced Therapies Supply Chain Management Solutions Market: Distribution by End User14.3.3. Overall Cell and Advanced Therapies Supply Chain Management Solutions Market: Distribution by Type of Software Solution14.3.4. Overall Cell and Advanced Therapies Supply Chain Management Solutions Market: Distribution by Mode of Deployment14.3.5. Overall Cell and Advanced Therapies Supply Chain Management Solutions Market: Distribution by Geography14.4. Overall Cell and Advanced Therapies Supply Chain Management Solutions Market: Distribution by Application, Type of Software Solution and Mode of Deployment14.4.1. Cell and Advanced Therapies Supply Chain Management Solutions Market for Donor Eligibility Assessment, 2019-203014.4.2. Cell and Advanced Therapies Supply Chain Management Solutions Market for Sample Collection, 2019-203014.4.3. Cell and Advanced Therapies Supply Chain Management Solutions Market for Manufacturing, 2019-203014.4.4. Cell and Advanced Therapies Supply Chain Management Solutions Market for Logistics, 2019-203014.4.5. Cell and Advanced Therapies Supply Chain Management Solutions Market for Patient Verification and Treatment Follow-up, 2019-2030

15. EXECUTIVE INSIGHTS15.1. Chapter Overview15.2. Thermo Fisher Scientific15.2.1. Company Snapshot15.2.2. Interview Transcript: Bryan Poltilove, Vice President and General Manager15.3. Cell and Gene Therapy Catapult15.3.1. Company Snapshot15.3.2. Interview Transcript: Jacqueline Barry, Chief Clinical Officer15.4. McKesson15.4.1. Company Snapshot15.4.2. Interview Transcript: Jill Maddux, Director, Cell and Gene Therapy Product Strategy, and Divya Iyer, Senior Director, Corporate Strategy and Business Development15.5. TrakCel15.5.1. Company Snapshot15.5.2. Interview Transcript: Martin Lamb, Chief Business Officer

16. CONCLUDING REMARKS16.1. Chapter Overview16.2. Key Takeaways

17. APPENDIX 1: LIST OF ADDITIONAL SUPPLY CHAIN MANAGEMENT SOFTWARE SOLUTIONS

18. APPENDIX 2: TABULATED DATA

19. APPENDIX 3: LIST OF COMPANIES AND ORGANIZATIONS

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Cell and Advanced Therapies Supply Chain Management Industry Report, 2019-2030 - GlobeNewswire

miRNAs: A Promising Target in the Chemoresistance of Bladder Cancer | OTT – Dove Medical Press

By Stem Cell Medicine

Zhonglin Cai,1,* Fa Zhang,2,* Weijie Chen,3,* Jianzhong Zhang,1 Hongjun Li1

1Department of Urology, Peking Union Medical College Hospital, Peking Union Medical College, Chinese Academy of Medical Sciences, Beijing, Peoples Republic of China; 2Department of Urology, First Hospital of Lanzhou University, Lanzhou, Gansu, Peoples Republic of China; 3Department of Urology, Shanghai Municipal Hospital of Traditional Chinese Medicine, Shanghai Traditional Chinese Medicine University, Shanghai, Peoples Republic of China

*These authors contributed equally to this work

Correspondence: Hongjun LiDepartment of Urology, Peking Union Medical College Hospital, Peking Union Medical College, Chinese Academy of Medical Sciences, No. 1 Shuaifuyuan, Dongcheng District, Beijing 100730, Peoples Republic of ChinaTel +86 139 0117 1724Email lihongjun@pumch.cn

Abstract: Chemotherapy is an important cancer treatment method. Tumor chemotherapy resistance is one of the main factors leading to tumor progression. Like other malignancies, bladder cancer, especially muscle-invasive bladder cancer, is prone to chemotherapy resistance. Additionally, only approximately 50% of muscle-invasive bladder cancer responds to cisplatin-based chemotherapy. miRNAs are a class of small, endogenous, noncoding RNAs that regulate gene expression at the posttranscriptional level, which results in the inhibition of translation or the degradation of mRNA. In the study of miRNAs and cancer, including gastric cancer, prostate cancer, liver cancer, and colorectal cancer, it has been found that miRNAs can regulate the expression of genes related to tumor resistance, thereby promoting the progression of tumors. In bladder cancer, miRNAs are also closely related to chemotherapy resistance, suggesting that miRNAs can be a new therapeutic target for the chemotherapy resistance of bladder cancer. Therefore, understanding the mechanisms of miRNAs in the chemotherapy resistance of bladder cancer is an important foundation for restoring the chemotherapy sensitivity of bladder cancer and improving the efficacy of chemotherapy and patient survival. In this article, we review the role of miRNAs in the development of chemotherapy-resistant bladder cancer and the various resistance mechanisms that involve apoptosis, the cell cycle, epithelial-mesenchymal transition (EMT), and cancer stem cells (CSCs).

Keywords: miRNAs, chemoresistant, bladder cancer, biomarkers, targeted therapy

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China confirms third birth of gene-edited baby; scientists involved get prison terms – WRAL Tech Wire

By Stem Cell Medicine

A Chinese scientist who set off an ethical debate with claims that he had made the worlds first genetically edited babies three, according to a Chinese court was sentenced to three years in prison because of his research, state media, the Associated Press and the New York Times have reported.

He Jiankui, who was convicted of practicing medicine without a license, was also fined 3 million yuan ($430,000) by a court in the southern city of Shenzhen, Chinas official Xinhua News Agency reported on Monday. Two other researchers involved in the project received lesser sentences and fines.

The verdict said the three defendants had not obtained qualification as doctors, pursued fame and profits, deliberately violated Chinese regulations on scientific research, and crossed an ethical line in both scientific research and medicine, according to Xinhua. It also said they had fabricated ethical review documents.

The court also confirmed a third birth, saying the researchers were involved in the births of three gene-edited babies to two women. It said all three scientists pleaded guilty during the trial, which Xinhua reported was closed to the public because of privacy concerns.

Hes declaration made him a pariah among scientists, cast a harsh light on Chinas scientific ambitions and embroiled other U.S. scientists who were connected to He. Although He offered no proof and did not share any evidence or data that definitively proved he had done it, his colleagues had said it was possible that he had succeeded, The New York Times reported.

U.S. scientists who knew of Hes plans came under scrutiny. Hes former academic adviser, Stephen Quake, a star Stanford University bioengineer and inventor, was cleared of any wrongdoing after an investigation into his interaction with his former student. Rice University has been investigating Michael Deem, Hes doctoral adviser, because of allegations that he was actively involved in the project.

Duke engineers improve CRISPR genome editing with biomedical tails

He, the lead researcher, shocked the scientific world when he announced in November 2018 that he had altered the embryos of twin girls who had been born the same month. He described his work in exclusive interviews with The Associated Press.

The announcement sparked a global debate over the ethics of gene editing. He said he had used a tool called CRISPR to try to disable a gene that allows the AIDS virus to enter a cell, in a bid to give the girls the ability to resist the infection. The identity of the children has not been released, and it isnt clear if the experiment succeeded.

The CRISPR tool has been tested elsewhere in adults to treat diseases, but many in the scientific community denounced Hes work as medically unnecessary and unethical, because any genetic changes could be passed down to future generations. The U.S. forbids editing embryos except for lab research.

He, who is known as JK, told the AP in 2018 that he felt a strong responsibility to make an example, and that society would decide whether to allow the practice to go forward. He disappeared from public view shortly after he announced his research at a conference in Hong Kong 13 months ago, apparently detained by authorities, initially in an apartment in Shenzhen, a city in Guangdong province that borders Hong Kong.

It wasnt clear if the three-year prison term includes any of the time he has already spent in Chinese custody.

A Chinese scientist said the sentence should have been harsher to deter others. Kehkooi Kee, a Tsinghua University researcher who conducts gene-editing research on stem cells, also said that He should be held responsible for any fallout from the experiment on the lives of the babies and their families.

Dr. William Hurlbut, a Stanford University bioethicist whose advice He sought for more than a year before his experiment, said he felt sorry for the scientist, his wife and two young daughters.

I warned him things could end this way, but it was just too late, Hurlbut wrote in an email addressed to the AP; the director of the U.S. National Institutes of Health, Dr. Francis Collins; and gene-editing pioneer Jennifer Doudna at the University of California, Berkeley.

Sad story everyone lost in this (JK, his family, his colleagues, and his country), but the one gain is that the world is awakened to the seriousness of our advancing genetic technologies, Hurlbut wrote.

Dr. Eric Topol, who heads the Scripps Research Translational Institute in California, noted its almost unheard of for a scientist to get imprisoned but in this case the sheer recklessness and unethical behavior warranted it. Topol praised China for standing up for proper medical research conduct.

Doudna told the AP she was concerned about the mysterious legal process in China, but she said the sentences are a step toward bringing this case to closure and send a strong message to discourage other such work. (Doudna is paid by the Howard Hughes Medical Institute, which also supports APs Health and Science Department.)

As a scientist, one does not like to see scientists going to jail, but this was an unusual case, Doudna said. Hes work was clearly wrong in many ways.

Before setting up a lab at the Southern University of Science and Technology of China in Shenzhen, He studied in the U.S. The verdict accused him of colluding with Zhang Renli and Qin Jinzhou, who worked at medical institutes in the same province.

Zhang was sentenced to two years in prison and fined 1 million yuan, Xinhua said. Qin received an 18-month prison sentence, but with a two-year reprieve, and a 500,000 yuan fine.

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What are the odds? Stem cell recipient learns her donor is also in Ottawa – Ottawa Citizen

By Stem Cell Doctors

Colleen LeCours lay in a hospital bed at the General campus of The Ottawa Hospital on August 12, 2016, waiting for the only thing that could save her life a stem cell transplant from a stranger.

The donor could be anywhere in the world if a related blood donor cant be found, the call to find a match goes out to registries all over the globe and the donated stem cells are rushed across international borders.

What LeCours didnt know is that her donor, an 18-year-old Carleton University student named Timothy White, was just one floor below. Similarly, White didnt know that his recipient was in the same hospital.

There are currently more than 450,000 people on the Canadian Blood Services Stem Cell Registry formerly known as OneMatch and 36 million on affiliated international registries. Still, some people never find a match. There are more than 900 Canadians in need of a transplant who have not found a match anywhere in the world.

What were the odds that the match for LeCours, now 57, would be found in the same city?

Astronomical, she said.

The chances that White would even ever be asked to donate were also very low only about one in a thousand. After he agreed to donate, he was not told where the recipient might be. I was told the recipient could be anywhere. They could be in Africa, said White, now 22 and a recent graduate in computer science.

White had signed up for the registry through a cheek swab booth at ComiCon less than six months earlier. A smart place to recruit would-be stem cell donors, he notes. The optimal donor is a male between the age of 17 and 35 and thats the ComiCon demographic.

He decided to register as a potential donor because he grew up in the scouting movement. One of the main philosophies is to do a good turn every day, he said.

The donation was a non-surgical procedure in which Whites blood was removed though a needle, the stem cells were separated from his blood and the remaining blood components returned to his body through another needle. The procedure started at about 8 a.m. and was over by about 5 p.m.

I figured if I gave someone a day for a thousand more days (of life) then I felt it was a fair trade. I have many years of life. Why not spend one day? said White.

LeCourss medical journey started in 2009 with an emergency room visit for abdominal pain. She was eventually diagnosed with Stage 4 follicular lymphoma, a blood cancer that affects infection-fighting white blood cells. At the time, LeCours was working for Gov.-Gen. Michalle Jean and was able to stay on the job most of the time during her six months of treatment.

Four years later, the lymphoma returned. It was back again two years after that, in a more aggressive form. The only treatment was stem cell transplant.

There are two main kinds of stem cell transplants autologous and allogenic. In an autologous transplant, stem cells are collected from a patients own blood and reintroduced after being treated to remove cancer cells. In an allogenic stem cell transplant, the stem cells come from a donor.

At this point, LeCours was a candidate for an autologous transplant. Once again, she underwent aggressive chemotherapy. A year later, the cancer returned.

Doctors told LeCours there wasnt much else they could do and advised her to get her affairs in order. But the hospitals transplant team felt she could be a candidate for an allogenic transplant. Theres risk rejecting donated stem cells can be fatal to the patient.

LeCours learned that her brother was a match. But the medical work-up would last about three months and she couldnt wait that long.

I wasnt sure I wanted to do it but I didnt have much choice, she said. They said, We have someone waiting in the wings.

And I said, He probably has wings.

After the transplant, LeCours recovered as an outpatient in the home of her brother and sister-in-law. It took three months to rebuild her immune system. Her only rejection symptoms were a bit of skin irritation.

In January 2018, LeCours received an email asking if she would like to exchange contact information with her donor. She replied that she would.

A few months later, she got a message with Whites co-ordinates and was astonished to find that her donor was in Ottawa. It took her a few weeks to formulate an email.

I didnt want to scare him. I just wanted him to know how incredibly grateful I was. And I wanted to pay it forward, said LeCours.

After careful consideration, she sent White an email on Oct. 8, 2018.

Today, being Thanksgiving, I have so much to be thankful for, namely you giving your stem cells and saving my life and the success of the stem cells grafting to my bone marrow, LeCours wrote. I cant thank you enough for your wonderful selfless act.

Stem cell donor 18-year-old Carleton University student Timothy White at The Ottawa Hospital, General campus, donating stem cells for Colleen LeCours in August 2016. At the time he did not know that LeCours would be the recipient. Courtesy Timothy White.jpg

She added that she didnt know anything about him except for his name and email address, and asked if they could meet. They got together for the first time over lunch in a burger restaurant.

As soon as I saw him, I broke down, said LeCours.

It has been three and a half years since the transplant and LeCours remains in remission. She invited White to her familys Thanksgiving this year, and the two meet to catch up every few months. Its one of the quirks of stem cell donation that the recipient assumes the blood type of the donor. LeCours, once O-positive, now has blood type A-negative, like White.

Im a grandmother. The fact that my grandson has his moma is huge.

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Understanding the Key Aspects of a Multiple Myeloma Diagnosis – Curetoday.com

By Stem Cell Doctors

A multiple myeloma expert helps newly diagnosed patients understand the standard of care for their disease.

Richter, an assistant professor of medicine at the Tisch Cancer Institute at the Ichan School of Medicine in Mt. Sinai Hospital located in New York City, noted that there are always exceptions to this rule, but the standard of care is to keep patients with multiple myeloma to continue therapy long term.

This standard of care, however, presents unique challenges and questions for newly diagnosed patients about to undergo treatment. In an interview during the 2019 CURE Educated Patient Summit on Multiple Myeloma in Charlotte, North Carolina, Richter had the chance to address the key aspects of a multiple myeloma diagnosis and how he addresses common questions from patients.

CURE: What does transplant eligible and transplant ineligible mean for patients?

Richter: The notion of transparent eligibility in the U.S. is not clearly defined. One of the people who trained me used to say, Do the patients have the tiger? relating back to Rocky, and essentially what this means is people who are younger tend to be more eligible. So, are you able to undergo the intensive nature of that procedure and chemotherapy?

If you're younger and healthier, you're generally transplant eligible. As you get older, with more medical problems, it becomes more of a risk. Everything in medicine from a Tylenol to a transplant has a risk and benefit. If you are 105 years old and had a heart attack last week, you're not going to be eligible. If you're 40 and otherwise healthy, you're eligible and everywhere in between is an evaluation of risks and benefits.

How would you describe the standard of care for patients with multiple myeloma?

In general, the standard of care is to attempt to get people onto three drugs. The three drugs usually mean a steroid, and then either an immunomodulatory drug, a proteasome inhibitor or a monoclonal antibody, and using those different combinations to come up with two or three-drug combinations, and actually in some cases four-drug combinations.

The general discussion of which one makes sense is we generally try to put some on a three-drug combination and the two most common ones now VRd (Velcade, Revlimid, and dexamethasone) is really a very big standard approach. There's some really wonderful, emerging data from the MAIA study, looking at taking Revlimid and dexamethasone and adding Darzalex (daratumumab) as a three-drug regimen for people who are not going on to transplant and some of that data looks amazing.

But for the most part, the precision that we use has to do not so much with the tumor but with the patient. Meaning for some diseases, the precision in the upfront setting is we look at a genetic marker and we target that. But for myeloma our upfront choice of therapy is saying, what are your comorbidities? What are your risks? For someone who has neuropathy, we may avoid Velcade. Someone has heart issues, we may avoid carfilzomib (Kyprolis) and if someone has difficult coming back and forth for long infusions, we may avoid Darzalex. So, most of the precision that we use is custom tailoring it not necessarily to the disease upfront, although that's part of it, but also to the patient.

What is the role of stem cell transplantation in treating patients with multiple myeloma?

The role of transplant is constantly evolving in myeloma. A generation ago, when we didn't have very good drugs, transplant was clearly the best thing to do because we didn't have good medicines. Transplant was the only way to get deep and durable remissions. Nowadays that we have such better therapies and even better ones along the way, it's being called into question about how much do we still need transplant. And it's a case by case basis, some people still clearly benefit from transplant.

It's an important discussion to have with your provider. But the risks have been well established for many years and we know how to manage them very well. Although there are risks for it, they're generally consolidated into a couple weeks to a couple of months, as opposed to being on long term treatment that can have ongoing risk of side effects. So, yes, they may be higher, but it's usually for a self-contained amount of time.

It's still a very important tool in our armamentarium to treat patients. Now, that being said, the majority of patients in the United States do not receive autologous transplant, so only about 30% and part of the reason has to do with the age of patients. The average age of a myeloma patient in the U.S. is 69, and many people in their 70s and 80s have other medical problems that make them not eligible for transplant.

There are some socioeconomic reasons, as well as referral patterns and access to care. I live in New York City, you can throw a rock and hit a transplant center, but there are parts of the country where the closest transplant center is hours and hours and hours away. And if you are older, sick or don't have easy transportation, it may be more difficult. So, many people do not receive transplant. However, many people nowadays may not even need it because our drugs have gotten so much better.

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The ‘supercells’ that cured an infant’s grave genetic illness – FRANCE 24

By Stem Cell Doctors

McLean (United States) (AFP)

When a person's immune system is impaired by a genetic disease, a bone-marrow transplant can be a powerful therapeutic tool, but with a major downside: during the first few months the recipient's defenses against viruses are severely weakened. The slightest infection can lead to a hospital trip.

A still-experimental type of treatment known as T-cell therapy aims to assist during this vulnerable period -- the months during which the body is rebuilding its natural defenses. After two decades of clinical trials, the technology has been refined, and is being used to treat more and more patients, many of them children.

A boy named Johan is one of them.

Today he is a mischievous, smiling toddler with a thick shock of light-brown hair, who never tires, playfully tormenting the family's puppy, Henry.

There is no sign of the three-year-long medical and emotional roller-coaster ride he and his family, who live in an affluent Washington suburb, have been on.

The first traumatic surprise came with the results of a pregnancy test: Johan was not planned.

"That was a huge shock. I cried," said his mother, 39-year-old Maren Chamorro.

- Risky procedure -

She had known since childhood that she carried a gene that can be fatal in a child's first 10 years, chronic granulomatous disease (CGD).

Her brother died of it at the age of seven. The inexorable laws of genetics meant that Maren had a one in four chance of transmitting it to her child.

For their first children, she and her husband Ricardo had chosen in-vitro fertilization, allowing the embryos to be genetically tested before implantation.

Their twins Thomas and Joanna were born -- both disease-free -- seven and a half years ago.

But in Johan's case, a post-birth genetic test quickly confirmed the worst: he had CGD.

After conferring with experts at Children's National Hospital in Washington, the couple took one of the most important decisions of their lives: Johan would receive a bone-marrow transplant, a risky procedure but one that would give him a chance of a cure.

"Obviously, the fact that Maren had lost a sibling at a young age from the disease played a big role," Ricardo confided.

Bone marrow, the spongy tissue inside bones, serves as the body's "factory" for the production of blood cells -- both red and white.

- His brother's immune system -

Johan's white blood cells were incapable of fighting off bacteria and fungal infections. A simple bacterial infection, of negligible concern in a healthy child, could spread out of control in his young body.

Luckily, Johan's brother Thomas, six years old at the time, was a perfect match. In April 2018, doctors first "cleansed" Johan's marrow using chemotherapy. They then took a small amount of marrow from Thomas's hip bones using a long, thin needle.

From that sample they extracted "supercells," as Thomas calls them -- stem cells, which they reinjected into Johan's veins. Those cells would eventually settle in his bone marrow -- and begin producing normal white blood cells.

The second step was preventive cell therapy, under an experimental program led by immunologist Michael Keller at Children's National Hospital.

The part of the immune system that protects against bacteria can be rebuilt in only a matter of weeks; but for viruses, the natural process takes at least three months.

- Hurdles remain -

From Thomas's blood, doctors extracted specialized white blood cells -- T-cells -- that had already encountered six viruses.

Keller grew them for 10 days in an incubator, creating an army of hundreds of millions of those specialized T-cells. The result: a fluffy white substance contained in a small glass vial.

Those T-cells were then injected into Johan's veins, immediately conferring protection against the six viruses.

"He has his brother's immune system," said Keller, an assistant professor at Children's National.

Johan's mother confirmed as much: today, when Thomas and Johan catch a cold, they have the same symptoms, and for nearly the same amount of time.

"I think it's pretty cool to have immunity from your big brother," Maren Chamorro said.

This therapeutic approach -- boosting the body's immune system using cells from a donor or one's own genetically modified cells -- is known as immunotherapy.

Its main use so far has been against cancer, but Keller hopes it will soon become available against viruses for patients, like Johan, who suffer from depressed immune systems.

The chief obstacles to that happening are the complexity of the process and the costs, which can run to many thousands of dollars. These factors currently restrict the procedure to some 30 medical centers in the United States.

For Johan, a year and a half after his bone marrow transplant, everything points to a complete success.

"It's neat to see him processing things, and especially play outside in the mud," his mother said.

"You know, what a gift!"

Her only concern now is the same as any mother would have -- that when her son does fall ill, others in the family might catch the same bug.

2020 AFP

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