Stem Cell Clinic

Targeted Cancer Therapies Market Regional Landscape, Production, Sales & Consumption Status and Prospects 2025 – Commerce Gazette

By Gene Therapy Clinics

Targeted cancer therapies are drugs that are actively involved in blocking the growth of cancer by interfering with specific molecules which are responsible for the growth, progression, and spread of cancerous cells. These therapies are also known as precision medicines. Targeted cancer therapy is different from standard chemotherapy treatment as these therapies target only cancerous cells without affecting the normal cells. Also targeted cancer therapy block tumour cell proliferation, whereas standard chemotherapy kills the tumour cells. Recently targeted cancer therapies have gained increasing focus in anti-cancer drug development industry as these therapies forms the main branch of precision medicine i.e. a form of medicine that uses molecular diagnostic techniques to prevent, diagnose, and treat cancer. Targeted cancer therapies market comprises of the drugs used as precision medicine for treating malignant and benign tumours. Many drugs in targeted cancer therapies have been approved by FDA to treat various types of cancer and have been commercialized, whereas numerous cancer therapies are being studied in clinical trials and many are in preclinical testing.

Targeted cancer therapies market is growing, this is attributed to increasing prevalence of various types of cancers such as lung cancer, breast cancer, colorectal cancer, prostate cancer, lymphoma, leukaemia, melanoma etc. Also increasing awareness regarding molecular diagnostic techniques such as liquid biopsy to detect malignancy is expected to drive the market for targeted cancer therapies over the forecast period. Growing healthcare expenditure, and rising insurance coverage, aids in the revenue growth of targeted cancer therapies market. Increasing number of new targeted anti-cancerous drugs also drives the market for targeted anti-cancer drugs. However higher pricing of these drugs along with higher pricing of the molecular diagnostic tests to detect cancer is expected to hamper the growth of the targeted cancer therapies market over the forecast period.

The targeted cancer therapies market is segment based on the therapy type, end user and application

Targeted cancer therapies market is segmented into following types:

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By Therapy Type Hormone Therapies Signal Transduction Inhibitors Gene Expression Modulators Apoptosis Inducers Angiogenesis Inhibitors Immunotherapies Monoclonal Antibodies

By Disease Indication Gastrointestinal Lung Cancer Breast Cancer Colorectal Cancer Leukemia Lymphoma Melanoma Prostate Cancer Others

By End User Hospitals Cancer and Radiation Therapy Centers Clinics

Targeted cancer therapies market revenue is expected to grow at a significant rate, over the forecast period. The market is anticipated to perform well in the near future due to increasing awareness regarding various cancer types and their treatment protocols. Also the targeted cancer therapies market is expected to expand globally due increasing prevalence of cancer and increasing preference of oncologists to prescribe targeted anti-cancer drugs for the patients. The targeted therapies for lung cancer is anticipated to grow with a fastest CAGR over the forecast period, attributed to increasing number of smokers globally. Increasing competition among anti-cancer drug manufacturers, increasing investment in R&D and increasing number of new drug launches are the major factors estimated to drive the revenue growth of targeted cancer therapies market.

Depending on geographic region, the targeted cancer therapies market is segmented into five key regions: North America, Latin America, Europe, Asia Pacific (APAC) and Middle East & Africa (MEA).

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North America is occupying the largest regional market share in the global targeted cancer therapies market owing to the presence of more number of market players, high patient awareness levels, increasing healthcare expenditure and relatively larger number of R&D exercises pertaining to drug manufacturing and marketing activities in the region. Also Europe is expected to perform well in the near future due to increasing inclination of oncologists and physicians in the region prescribing targeted anti-cancerous drugs to the patients suffering from cancer.

Asia Pacific is expected to grow at the fastest CAGR because of increasing prevalence of different types of cancers in the region, thus boosting the market growth of targeted cancer therapies market throughout the forecast period.

Key players of targeted cancer therapies market includes Abbott Laboratories, Bayer HealthCare AG, GlaxoSmithKline plc, OncoGenex Pharmaceuticals Inc., Hospira Inc., Boehringer Ingelheim GmbH, AstraZeneca, Aveo Pharmaceuticals and many more. The companies in targeted cancer therapies market are increasingly engaged in strategic partnerships, collaborations, mergers and acquisitions to capture a greater pie of market share as the market is in the nascent stage.

The research report presents a comprehensive assessment of the market and contains thoughtful insights, facts, historical data, and statistically supported and industry-validated market data. It also contains projections using a suitable set of assumptions and methodologies. The research report provides analysis and information according to categories such as market segments, geographies, types, technology and applications.

The report covers exhaustive analysis on: Market Segments Market Dynamics Market Size Supply & Demand Current Trends/Issues/Challenges Competition & Companies involved Technology Value Chain

Regional analysis includes North America (U.S., Canada) Latin America (Argentina, Mexico, Brazil, Rest of Latin America) Europe (Germany, Italy, France, U.K., Spain, Russia, Rest of Europe) Asia Pacific (China, India, Japan, Rest of APAC) Middle East and Africa (Rest of MEA, S. Africa)

The report is a compilation of first-hand information, qualitative and quantitative assessment by industry analysts, inputs from industry experts and industry participants across the value chain. The report provides in-depth analysis of parent market trends, macro-economic indicators and governing factors along with market attractiveness as per segments. The report also maps the qualitative impact of various market factors on market segments and geographies.

Report Highlights: Detailed overview of parent market Changing market dynamics in the industry In-depth market segmentation Historical, current and projected market size in terms of volume and value Recent industry trends and developments Competitive landscape Strategies of key players and products offered Potential and niche segments, geographical regions exhibiting promising growth A neutral perspective on market performance Must-have information for market players to sustain and enhance their market footprint.

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Targeted Cancer Therapies Market Regional Landscape, Production, Sales & Consumption Status and Prospects 2025 - Commerce Gazette

Precision Cancer Therapies Market to Witness Comprehensive Growth by 2017 2025 – Technology Trend

By Gene Therapy Clinics

Precision medicine (PM) can be defined as predictive, personalized, and preventive healthcare services delivery model. Precision cancer therapies is an additional option for patients suffering from cancer however it cannot completely replace the existing cancer treatments. Currently, researchers are making progress in the field of precision cancer therapies however many new and innovative drugs are currently in clinical trials. Precision cancer therapies include drugs or other substances which block the growth of cancer. Precision cancer therapies are also termed as molecular targeted therapy, or targeted molecular therapies, and precision medicines. Researchers are involved in developing anticancer drug developments via precision cancer therapies.

Precision Cancer Therapies Market: Segmentation

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Precision cancer therapies market can be segmented on the basis of the type of therapies, end users, and regions: Hormone Therapy Immunotherapies Targeted Therapy Monoclonal Antibody Therapy Gene Therapy

Precision cancer therapies market can be segmented on the basis of different end users in the market: Hospitals Diagnostic Centers Oncology Clinics Research Institutes

Precision Cancer Therapies Market: Dynamics

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Precision cancer therapies market is primarily driven by a few key factors such as the increasing prevalence of cancers, such as lung cancer, breast cancer, prostate cancer, melanoma and other types of cancers. The market is likely to grow owing to the increasing awareness regarding molecular diagnostic techniques which is expected to fuel the growth of precision cancer therapies market. The rising insurance coverage and growing healthcare expenditure by the government are among the factors which would aid the growth of precision cancer therapies market over the forecast years.

Precision cancer therapies market, however, faces various challenges such as the high cost of new and innovative therapies which prevent the wide prevention of these diseases. Precision cancer therapies market has various drugs which are still in various stages of clinical trials which refrain the products from the market. Precision cancer therapies market faces tremendous challenges due to the low awareness regarding the new diagnosis and treatment measures. Low-income countries and rising economies are coming forward to address such issues for precision cancer therapies market.

Precision Cancer Therapies Market: Region-wise Outlook

Based on geography, the precision cancer therapies market can be segmented into five major regions: North America, Europe, Asia-Pacific, Latin America and Middle East & Africa. At present, North America holds a leading position in the precision cancer therapies market due to the increasing incidence of cancer in the region which is followed by Europe. The major driving factors which have driven the growth of the precision cancer therapies market in this region is constant support of healthcare organizations in the development of new treatment methods, technological advancement in finding innovative treatment measures, and a rise in funding in public and private sector. Following North America, European countries are also anticipated to show steady growth in the precision cancer therapies market. Asia Pacific is expected to grow at the fastest CAGR because of increasing prevalence of different types of cancers in the region, thus boosting the market growth of precision cancer therapies market throughout the forecast period. The factors which would fuel the growth of precision cancer therapies market in Asia-Pacific are various multinational companies are setting up their operations in this region and aiming to gain huge revenue share from emerging countries, rising healthcare concerns, and improving healthcare scenario of the region. Precision cancer therapies market would evolve at a rapid rate across the regions. However, North America would maintain its position in the precision cancer therapies market, though, we are anticipating emerging economies such India, China, Brazil, Russia to have the highest growth in precision cancer therapies market.

Precision Cancer Therapies Market: Key Players

Precision cancer therapies market holds a huge number of players operating in the segment for years with expertise and experience. Various multinational companies are involved in the manufacturing of products which are utilized in the treatment of cancer. Such companies are Abbott Laboratories, Bayer HealthCare AG, GlaxoSmithKline plc, OncoGenex Pharmaceuticals Inc., Hospira Inc., Boehringer Ingelheim GmbH, AstraZeneca, Aveo Pharmaceuticals among others. Precision cancer therapies market has the presence of many regional players which have a huge market share in the emerging countries.

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Precision Cancer Therapies Market to Witness Comprehensive Growth by 2017 2025 - Technology Trend

Biological Product Manufacturing Market Analysis, Size, Regional Outlook, Share, Trend, Growth, Analysis and Forecast – Rapid News Network

By Gene Therapy Clinics

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Biological products or biologics are therapeutic preparations that consist of natural complex biomolecules derived from living things. Active pharmaceutical ingredients (API), vaccines serum, and hematological products (whole blood and plasma derivatives), recombinant DNA products, cell cultures (micro-organisms or eukaryotic cells), gene therapy and cell therapy products, antigens, allergens, antivenoms, etc. are some examples of biological products. Due to the recent advances in supply chain management and favorable federal regulations, the biological products manufacturing industry has become a lucrative market for startups, contractual manufacturing companies, and strategic collaborations, equipped with skilled workers and affordable pricing. Increase in investments for R&D by various organizations, with an aim of manufacturing cost reduction and economical yet sustainable biological product manufacturing are serving as opportunities for many companies and collaborations in biological product manufacturing industries.

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Major market players in the global biological product manufacturing market have various advantages such as cutting edge technologies, superior research and development, technologically advanced instruments, and skilled workforce to ensure proper assembly and delivery of the product. The biological product manufacturing market is driven by extensive R&D, along with increasing demand from health care providers across the world for advanced biological products, and growing focus by market players to provide quality products. However, competitive costs of therapeutic products, requirement of highly skilled labor for high value low volume biological product manufacturing, limited availability foreign capital, and stringent regulatory affairs regarding the manufacture of biological products are restraining the global biological product manufacturing market.

The global biological product manufacturing market has been segmented based on product type, end-user, application, and region. In terms of product type, the market is classified into bio-pharmaceutical products and biotechnological/biological products. The bio-pharmaceutical products segment includes vaccines, immunoglobulins, serum and other blood related product, allergens, antigens, antivenoms, and toxoids. The biotechnological/biological products segment includes micro-organism or eukaryotic cell culture, recombinant DNA technology products, gene therapy product, and cell therapy product. The biopharmaceuticals products segment accounted for the largest share of the global biological product manufacturing market. Based on end-user, the biological product manufacturing market has been segmented into hospitals, research and academic institutes, clinics and specialty trials, diagnostic labs, and others. The hospitals end-user segment accounted for the largest share of the global biological product manufacturing market, followed by research and academic institutes.

Superior technological advancements serve as a major opportunity in the biological product manufacturing market. Rising incidence of chronic diseases across the world, availability of better diagnostic facilities, advances in the drug discovery and pharmaceutical R&D sector, and rising government initiatives are some of the factors attributed to the high growth of the biological product manufacturing market. Increasing focus on discovery of heat labile or temperature sensitive products, advanced biopharmaceutical research in medicine, and invention of effective means to treat various diseases are expected to boost the growth of the biological product manufacturing market.

In terms of region, the global biological product manufacturing market has been segmented into five regions: North America, Europe, Asia Pacific, Latin America, and Middle East & Africa. The market in Asia Pacific is projected to expand at a higher CAGR during the forecast period driven by factors such as low labor cost, less capital investment for manufacturing, increasing adoptions of new techniques in manufacturing of biological product, and favorable governmental policies. North America dominated the global biological product manufacturing market in 2015 due to factors such as new and technologically advanced products introduced in the market, significant investments in research and development of advanced products, and increasing patient demand for better health care facilities.

Key players in the biological product manufacturing market are Lonza, Advanced Life Sciences Holdings, Inc., Pfizer, Inc., Johnson & Johnson, Invitrogen, Amgen, Abbott Laboratories, Piramal Healthcare, and Shenhua Group Corp. Ltd. The report offers a comprehensive evaluation of the market. It does so via in-depth qualitative insights, historical data, and verifiable projections about market size. The projections featured in the report have been derived using proven research methodologies and assumptions. By doing so, the research report serves as a repository of analysis and information for every facet of the market, including but not limited to: Regional markets, technology, types, and applications. The study is a source of reliable data on: Market segments and sub-segments Market trends and dynamics Supply and demand Market size Current trends/opportunities/challenges Competitive landscape Technological breakthroughs Value chain and stakeholder analysis The regional analysis covers: North America (U.S. and Canada) Latin America (Mexico, Brazil, Peru, Chile, and others) Western Europe (Germany, U.K., France, Spain, Italy, Nordic countries, Belgium, Netherlands, and Luxembourg) Eastern Europe (Poland and Russia) Asia Pacific (China, India, Japan, ASEAN, Australia, and New Zealand) Middle East and Africa (GCC, Southern Africa, and North Africa) The report has been compiled through extensive primary research (through interviews, surveys, and observations of seasoned analysts) and secondary research (which entails reputable paid sources, trade journals, and industry body databases). The report also features a complete qualitative and quantitative assessment by analyzing data gathered from industry analysts and market participants across key points in the industrys value chain. A separate analysis of prevailing trends in the parent market, macro- and micro-economic indicators, and regulations and mandates is included under the purview of the study. By doing so, the report projects the attractiveness of each major segment over the forecast period. Highlights of the report: A complete backdrop analysis, which includes an assessment of the parent market Important changes in market dynamics Market segmentation up to the second or third level Historical, current, and projected size of the market from the standpoint of both value and volume Reporting and evaluation of recent industry developments Market shares and strategies of key players Emerging niche segments and regional markets An objective assessment of the trajectory of the market Recommendations to companies for strengthening their foothold in the market Note:Although care has been taken to maintain the highest levels of accuracy in TMRs reports, recent market/vendor-specific changes may take time to reflect in the analysis.

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Biological Product Manufacturing Market Analysis, Size, Regional Outlook, Share, Trend, Growth, Analysis and Forecast - Rapid News Network

Google Bans Advertising on Unproven Medical Treatments (like Stem Cells) – Bedford Bulletin

By Gene Therapy Clinics

Closing out the first week of September, Google said it will no longer allow the posting of ads for unproven or experimental medical techniques.This includes, mostly, advertising that discusses or describes stem cell therapy, cellular therapy, and gene therapy.The Mountain View, CA-based technology giant said that this complex decision comes as a means to quell a rise in bad actors who try to take advantage of vulnerable people by offering untested, deceptive treatments.

In a recent blog post, Google said, These treatments can lead to dangerous health outcomes and we feel they have no place on our platforms, specifically ads for medical treatments with no established biomedical or scientific basis.

If this seems arbitrary to you then you may not have seen any of the onslaught of new ads from stem cell clinics across the United States looking to sell unapproved therapies which, they claim, can treat a wide range of ailments. This might include everything from arthritis to Alzheimers disease, from macular degeneration to multiple sclerosis.

And if you have not seen any of this type of advertising, you might want to be prepared to start.Stem cell clinics have been growing quickly as an emerging direct-to-consumer industry.And their growth may be largely due to the excitement generated by advertising the vast number of conditions that stem cells can supposedly treat.As a matter of fact, scientists and medical associations alike have commented that these unsupported claims make stem cell therapy like a modern snake oil that specifically aims to prey on seriously illand terribly vulnerablepatients.

While stem cell research has certainly had its breakthroughs, claiming that it can treat such a broad canvas of ailments is dangerous. Unfortunately, some stem cell treatments have already resulted in serious injury in some patients. Specifically, at least five women have reported going blind after a stem cell clinic injected their product directly into their eyes.

At the same time, some stem-cell industry representatives are criticizing Googles new ad policy. The argument is that this ban unfairly discriminates against good companies because there is no determination as to which companies actually provide a safe, FDA-verified procedure.Of course, Google should not have be a regulator in this industry, so the ban is more of an attempt to avoid any involvement with the potential risks.

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Google Bans Advertising on Unproven Medical Treatments (like Stem Cells) - Bedford Bulletin

Global Induced Pluripotent Stem Cells (iPSCs) Market 2019 Industry Growth with CAGR 12.7% in Forecast to 2024 – Indian Columnist

By Induced Pluripotent Stem Cells

MarketResearchNest.com adds Global Induced Pluripotent Stem Cells (iPSCs) Market Growth (Status and Outlook) 2019-2024new report to its research database. The report spread across 115 pages with multiple tables and figures in it.

According to this study, over the next five years the Induced Pluripotent Stem Cells (iPSCs) market will register a 12.7% CAGR in terms of revenue, the global market size will reach US$ 113.1 million by 2024, from US$ 70 million in 2019. In particular, this report presents the global revenue market share of key companies in Induced Pluripotent Stem Cells (iPSCs) business, shared in Chapter 3.

This report presents a comprehensive overview, market shares, and growth opportunities of Induced Pluripotent Stem Cells (iPSCs) market by product type, application, key manufacturers and key regions and countries.

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This study considers the Induced Pluripotent Stem Cells (iPSCs) value generated from the sales of the following segments:

Segmentation by product type: breakdown data from 2014 to 2019 in Section 2.3; and forecast to 2024 in section 10.7.

Human iPSCs had a market share of 89% in 2018, followed by Mouse iPSCs.

Segmentation by application: breakdown data from 2014 to 2019, in Section 2.4; and forecast to 2024 in section 10.8.

Academic Research is the largest segment of Induced Pluripotent Stem Cells (iPSCs) application,with a share of 32% in 2018.

This report also splits the market by region: Breakdown data in Chapter 4, 5, 6, 7 and 8.

Americas, United States, Canada, Mexico, Brazil, APAC, China, Japan, Korea, Southeast Asia, India, Australia, Europe, Germany, France, UK, Italy, Russia, Spain, Middle East and Africa, Egypt, South Africa, Israel, Turkey, GCC Countries

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The report also presents the market competition landscape and a corresponding detailed analysis of the major vendor/manufacturers in the market. The key manufacturers covered in this report: Breakdown data in in Chapter 3.

In addition, this report discusses the key drivers influencing market growth, opportunities, the challenges and the risks faced by key players and the market as a whole. It also analyzes key emerging trends and their impact on present and future development.

Research objectives

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Global Induced Pluripotent Stem Cells (iPSCs) Market 2019 Industry Growth with CAGR 12.7% in Forecast to 2024 - Indian Columnist

Adipose Tissue-derived Stem Cells Market Size Set for Rapid Growth and Trend by2018 2028 – My Health Reporter

By Induced Pluripotent Stem Cells

Adipose tissue is rich in multi potent stem cells that have the capability to differentiate into a number of cell types including adipocytes, osteocytes, chondrocytes and others, in vitro. These Adipose Tissue-derived Stem Cells are used for a number of applications including stem cell differentiation studies, regenerative medicine, cell therapy, tissue engineering and development of induced pluripotent stem cell lineage. Adult stem cells such as the Adipose Tissue-derived Stem Cells have a very good potential for regenerative medicine. The Adipose Tissue-derived Stem Cells show higher yields compared with other stem cell sources. Some of the regenerative medicine applications using Adipose Tissue-derived Stem Cells include skin, bone and cartilage regeneration.

Although, Adipose Tissue-derived Stem Cells have the ability to differentiate into different cell types in vitro, unlike the embryonic stem cells they lack the ability to differentiate into all types of organs and tissues of the body. Derivation of stem cells from adipose tissue have a number of advantages including that fat tissue contain 100 to 1000 times more mesenchymal stem cells than the bone marrow. Furthermore the method of collection of fat tissue is relatively easier and is less invasive than that of bone marrow collection. Although Adipose Tissue-derived Stem Cells have a potential to be used in cell-based therapy, there are a number of challenges the Adipose Tissue-derived Stem Cells market has to face. Some of the challenge include the safety issue for the clinical use of Adipose Tissue-derived Stem Cells, development and differentiation of the cells, delivery of the cells in vivo and immune response after the transplant.

The global Adipose Tissue-derived Stem Cells market is segmented based on product type and end user. Based on product type the Adipose Tissue-derived Stem Cells can be categorized into cell line and reagent & kits. Cell line can be further classified based on the source of the adipose tissue such as human and rodents. Based on reagents the Adipose Tissue-derived Stem Cells market is further classified as media & sera and kits. Based on application the Adipose Tissue-derived Stem Cells market is classified into regenerative medicine, cell therapy, tissue engineering, and other applications such as cell differentiation studies and other similar research. End users of Adipose Tissue-derived Stem Cells market are biotechnology companies and academic and research institutes.

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The Global Adipose Tissue-derived Stem Cells market is classified on the basis of product type, end user and region:

Based on the Product Type, Adipose Tissue-derived Stem Cells market is segmented into following: Cell Line Human Adipose Tissue-derived Stem Cells Rat Adipose Tissue-derived Stem Cells Reagents & Kits Media & Sera Kits

Based on the application, Adipose Tissue-derived Stem Cells market is segmented into following: Regenerative medicine Tissue engineering Cell therapy Others

Based on the end user, Adipose Tissue-derived Stem Cells market is segment as below: Biotechnology companies Academic & Research Institutes

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Growing research activities using stem cells along with the growth of regenerative medicine and cell therapy the global Adipose Tissue-derived Stem Cells market is set to expand considerably during the forecast period. However, ethical concerns and stringent regulations may hinder the growth of the global Adipose Tissue-derived Stem Cells market.

On the basis of geography, global Adipose Tissue-derived Stem Cells market is segmented into six major regions that include North America, Latin America, Europe, Asia-Pacific excluding China, China and Middle East & Africa. North America is expected to be the most lucrative Adipose Tissue-derived Stem Cells market owing to increased research activity of stem cells. Furthermore government support for regenerative and stem cell based studies along with cell therapy studies is driving the growth of the Adipose Tissue-derived Stem Cells market in the region. Changing government regulations in china is supporting the research activity that supports the growth of the adipose tissue-derived stem cell market in the region at a considerable rate.

Key participants operating in the Adipose Tissue-derived Stem Cells market are: Lonza, ThermoFisher Scientific, Celprogen, Inc, American CryoStem, Rexgenero Ltd, iXCells Biotechnologies, Merck KGaA, Lifeline Cell Technology, and others.

The report covers exhaustive analysis on: Adipose Tissue-derived Stem Cells Market Segments Adipose Tissue-derived Stem Cells Market Dynamics Historical Actual Market Size, 2013 2017 Adipose Tissue-derived Stem Cells Market Size & Forecast 2018 to 2026 Adipose Tissue-derived Stem Cells market Current Trends/Issues/Challenges Competition & Companies involved Adipose Tissue-derived Stem Cells Market Drivers and Restraints

Regional analysis includes North America Latin America Europe Asia Pacific Excluding China China Middle East & Africa

Report Highlights: Shifting Industry dynamics In-depth market segmentation Historical, current and projected industry size recent industry trends Key Competition landscape Strategies for key players and product offerings Potential and niche segments/regions exhibiting promising growth A neutral perspective towards market performance

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Adipose Tissue-derived Stem Cells Market Size Set for Rapid Growth and Trend by2018 2028 - My Health Reporter

Stem cell therapy is promising, but we need more donors from more races – The Star Online

By Stem Cell Treatment

KUALA LUMPUR: Stem call therapy holds out great promise for treating blood cancer and other disorders, but the number of registered donors in Malaysia is very low, says the Health Ministry.

Its Minister Datuk Seri Dr Dzulkefly Ahmad said the Malaysian Stem Cell Registry (MSCR) which was established 19 years ago only listed 28,291 donors so far.

With this small number in the registry, matching donors were found (only) in 16 cases, he said.

Among the diseases that could be cured through stem cell therapy are thalassaemia, leukaemia, bone marrow disorder, lymphoma and multiple myeloma, he added when opening the inaugural World Marrow Donor Day celebration in Malaysia at the Ampang Hospital on Thursday (Sept 19).

This year's theme is Be the match, be a donor.

Dr Dzulkefly noted that Asians only made up 15% of the 34 million registered stem cell donors worldwide, and the bulk of Asian donors are Chinese.

Hence, the chances of a Chinese patient finding a matching donor in the global registry is higher compared to Malays, Indians, as well as the bumiputra in Sabah, Sarawak, and other races, he said.

Dr Dzulkefly noted that 70% of global stem cell donors were Caucasian, even though 88% of the global population were non-Caucasians.

He also lauded the Ampang Hospital-based Haematology Department for taking the initiative to organise the inaugural World Marrow Day celebration in Malaysia to raise awareness on the need to increase the pool of public donors to increase the chances of finding matching donors.

Perhaps many people are not aware that stem cell donations can also be made by just donating blood, he said.

Dr Dzulkefly added that up till 2018, Ampang Hospital had performed 2,111 stem cell transplants.

Of the number of cases, 797 involved donors from among relatives, 53 involved matching donors from among non-relatives, 49 cases involved unmatched donors from among relatives (haplodentical), and 10 cases involved stem cells from umbilical cords.

Most of the stem cells from matching non-relative donors were sourced from abroad, he said.

Dr Dzulkefly added that the government could not afford to absorb the high cost involved in sourcing and importing matching stem cells from abroad.

Therefore it was pertinent to establish a large pool of local public donors.

With the decline in the fertility rate in Malaysia since 2013, it would be increasingly difficult to find matching donors from among close relatives, he said.

He noted that only a few hospitals in Malaysia had the expertise to carry out stem cell transplants to treat blood disorders.

Ampang Hospital is the biggest centre where 60% of stem cell transplants in the country are carried out here, he said.

Other hospitals that had the expertise to carry out stem cell transplant include Penang Hospital and Sultanah Aminah Hospital in Johor.

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Stem cell therapy is promising, but we need more donors from more races - The Star Online

CAR T-Cell Therapy for Pediatric Patients: The Latest Updates – Dana-Farber Cancer Institute

By Stem Cell Treatment

SUMMARY: Researchers are learning how best to use this powerful modality while avoiding serious toxicities.

Since it was approved by the U.S. Food and Drug Administration (FDA) in the fall of 2017, a form of the powerful and promising therapy known as CAR T-cell therapy has been used to treat certain young patients with B-cell acute lymphoblastic leukemia (ALL) who have relapsed or didnt respond to standard regimens.

Today, researchers and physicians at Dana-Farber/Boston Childrens Cancer and Blood Disorders Center say they are learning how best to use the specific CAR T-cell treatment known as Kymriah for which patients, and at what point in their cancer treatment and how to predict and treat the serious toxicities that can occur. Theyre also laser-focused on another question: Can CAR T-cell therapy work for other pediatric patients?

CAR T-cell therapy is a form of gene therapy in which a patients own immune cells are removed, engineered in a lab to hone their cancer-fighting abilities, and returned to the body. In clinical trials, Kymriah showed encouraging remission and survival rates in B-cell ALL patients who had few other options leading to its FDA approval.

Kymriah is approved for patients 25 years old and below whohave B-cell ALL expressing the CD19 protein, and who have not responded tostandard therapy or are in a second or later relapse. The great majority ofpatients with B-cell ALL are successfully treated with standard methods, butthose who dont respond have an unfavorable prognosis. Kymriah has been shownto be effective in many of these cases.

CAR T-cell therapy is a complete paradigm shift, says Christine Duncan, MD, a senior physician at Dana-Farber/Boston Childrens. It started with CD19, which is a specific form of pediatric ALL. Now that it has expanded, were learning a lot more about the obstacles to therapy and how we need to pick the right patients for the treatment, so we can move forward to other high-risk populations.

Some patients receive CAR T-cell therapy and then undergo stem cell transplantation, while for others its the reverse, say Duncan and Steven Margossian, MD, PhD, who is a senior physician in the stem cell transplant program at Dana-Farber/Childrens.

Generally, we are using CAR T as a bridge to a transplant;CAR T gets the patient into a good remission and then we take them totransplant, says Margossian. They do well with a transplant.

But some patients whose leukemia relapses after a stem celltransplant are treated with Kymriah as definitive therapy.

Kymriah treatments are custom-made for each individual. Initially, the patient undergoes apheresis to remove lymphocytes known as T cells, which are frozen, packaged, and sent to a pharmaceutical laboratory. There, the T cells are equipped with whats known as a chimeric antigen receptor (CAR) so that they will home in on the CD19 protein on the patients leukemia cells. This takes about 24 to 28 days; meanwhile, the patient may receive a cycle of chemotherapy to combat the leukemia. When the CAR T cells are returned to Dana-Farber/Boston Childrens, they are reinfused into the patient, where they seek out and destroy the cancer cells.

CAR T-cell therapy can trigger serious side effects,including cytokine release syndrome. There are medications that can block thisreaction, and we are researching the correct way to give the medication: shouldit be as a rescue medicine or can you give it pre-emptively? Margossian says. Neurologicaltoxicities are also possible.

We are very good at managing the patients through the complications we always have regular meetings for every CAR T patient that comes in, and we are always prepared to transfer the patient to the ICU if needed, Margossian notes.

The long-term effects of CAR T-cell therapy are also still unclear,but doctors are working to understand them.

It took many years to figure out how to correctly followpatients who received a stem cell transplant, says Duncan. Now were tryingto figure out how to follow patients who received CAR T-cell therapy. This issomething well learn over time.

Meanwhile, variations on the approved use of Kymriah are being studied in new clinical trials. Margossian says one trial will be for patients with ALL who are in remission but have high levels of detectable cancer cells. Another trial will test Kymriah in pediatric B-cell lymphomas. Also on the horizon is a clinical trial of CAR T-cells as a bridge to transplant for patients with acute myeloid leukemia (AML).

Farther off are CAR T-cell therapy trials for solid tumorssuch as bone cancers and neuroblastoma, which present unique challenges, accordingto Margossian. One hurdle is that the cancer-specific molecules that CAR Tcells bind to are often inside the cancer cells of solid tumors, not on thesurface, as they are in blood cancers, and therefore are more difficult totarget.

Its exciting to see CAR T-cell studies developing andexpanding to very high-risk patient populations, says Duncan. Were veryhopeful that CAR T-cell therapy will expand to entirely new populations.

Read more here:
CAR T-Cell Therapy for Pediatric Patients: The Latest Updates - Dana-Farber Cancer Institute

CRISPR fix in mice may lead to muscular dystrophy therapy – Futurity: Research News

By Stem Cell Treatment

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The CRISPR gene editing technique may provide the means for lifelong correction of the genetic mutation responsible for Duchenne muscular dystrophy, a new study with mice shows.

Duchenne muscular dystrophy (DMD), a rare but devastating genetic disorder, causes muscle loss and physical impairment. Children with DMD have a gene mutation that interrupts the production of a protein known as dystrophin. Without it, muscle cells weaken and eventually die. Many children lose the ability to walk, and muscles essential for breathing and heart function ultimately stop working.

Research has shown that CRISPR can be used to edit out the mutation that causes the early death of muscle cells in an animal model, says Dongsheng Duan, professor in medical research in the molecular microbiology and immunology department at the University of Missouri School of Medicine and senior author of the paper in Molecular Therapy.

However, there is a major concern of relapse because these gene-edited muscle cells wear out over time. If we can correct the mutation in muscle stem cells, then cells regenerated from the edited stem cells will no longer carry the mutation. A one-time treatment of the muscle stem cells with CRISPR could result in continuous dystrophin expression in regenerated muscle cells.

For the study, researchers explored whether they could efficiently edit muscle stem cells from mice. They first delivered the gene editing tools to normal mouse muscle through AAV9, a virus that the US Food and Drug Administration recently approved to treat spinal muscular atrophy.

We transplanted AAV9 treated muscle into an immune-deficient mouse, says lead author Michael Nance, a MD-PhD program student in Duans lab. The transplanted muscle died first then regenerated from its stem cells. If the stem cells were successfully edited, the regenerated muscle cells should also carry the edited gene.

The researchers reasoning was correctthey found abundant edited cells in the regenerated muscle. They then tested if they could use CRISPR to edit muscle stem cells in a mouse model of DMD. Similar to what they found in normal muscle, the stem cells in the diseased muscle were also edited. Cells regenerated from these edited cells successfully produced dystrophin.

This finding suggests that CRISPR gene editing may provide a method for lifelong correction of the genetic mutation in DMD and potentially other muscle diseases, Duan says.

Our research shows that CRISPR can be used to effectively edit the stem cells responsible for muscle regeneration. The ability to treat the stem cells that are responsible for maintaining muscle growth may pave the way for a one-time treatment that can provide a source of gene-edited cells throughout a patients life.

With more study, the researchers hope this stem cell-targeted CRISPR approach may one day lead to long-lasting therapies for children with DMD.

Additional coauthors are from the University of Missouri, the National Center for Advancing Translational Sciences, Johns Hopkins School of Medicine, and Duke University. The National Institutes of Health, the Department of Defense, the Jackson Freel DMD Research Fund, Hope for Javier, and the Intramural Research Program of the National Center for Advancing Translational Sciences funded the work.

Source: University of Missouri

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CRISPR fix in mice may lead to muscular dystrophy therapy - Futurity: Research News

When You Need Stem Cells, You May Appreciate the Donor Registry – 93.1 WIBC Indianapolis

By Stem Cell Treatment

INDIANAPOLIS--Getting a diagnosis of leukemia or sickle cell disease can mean months or years of treatment, and that doesn't necessarily work. Sometimes a bone marrow or stem cell transplant is the answer and can be a cure. But, sometimes donors are hard to find.

"In any given family each child has about a 30 percent chance of having a match within their family, especially if they have siblings. The likelihood of a match becomes higher, the more siblings you have," said Dr. Jodi Skiles, who is director of the pediatric stem cell program at Riley Children's Health.

But, when the siblings aren't a match, families have to turn to the world-wide stem cell donor registry.

"There's a stem cell registry. It's called 'Be the Match', and it really is a mechanism for volunteers to sign up to be a donor," said Skiles.

LINK: Be The Match website

Brittany Pittman's daughter was nine years old when she needed a donor.

"I got the phone call when I was at work on Valentine's Day 2017, saying that she tested positive for leukemia," she said. Pittman, of Greenwood, took her daughter to Riley the next day.

"I just remember coming home. Everybody's crying. I just asked 'em like what was wrong. They didn't answer," said Alayna Pittman, 12.

"She did some rounds of IV chemo. We were eventually told that her leukemia wasn't going away with just the IV chemo, that she needed a stem cell transplant," said Pittman. "All of our family members were tested and we were not a match for Alayna."

That's when they turned to the registry. After two weeks a match was found and testing on both the donor and Alayna began. Within a month she was ready for the transplant. Alayna was in the hospital for six weeks and had to have oral chemotherapy for two years. She is considered cured, an outcome Skiles said is typical for people who get stem cell transplants.

She said leukemia, lymphoma and Sickle Cell disease are some of the most common of 40 to 50 illnesses that can be treated with stem cell transplants.

"It's a really simple process. You just have to swab the inside of your cheek and give us your information so that when we have a patient that is in need, we can search the registry to see if you happen to be a match to that patient," said Skiles.

Though it's generally tougher for minorities to find a match, when you donate, it may not necessarily be to someone in Indiana.

"The match program is a national and global program. Signing up as a potential donor in Indiana means you could be donating cells for somebody anywhere in the world."

She said http://www.BetheMatch.org is the portal to find out about donating.

PHOTOS: Chris Davis/Emmis

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When You Need Stem Cells, You May Appreciate the Donor Registry - 93.1 WIBC Indianapolis

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