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Next-Generation Sequencing Market Size to Reach US$ 31,411.3 Mn, Introduction of Rapid Sequencing Technologies Drives the Market: Fortune Business…

August 17, 2020 11:38 ET | Source: Fortune Business Insights

Pune, Aug. 17, 2020 (GLOBE NEWSWIRE) -- The global next-generation sequencing market is forecast to exhibit a remarkable CAGR as the next-generation sequencing platform allows effective sequencing of millions of DNA molecules. Fortune Business Insights in a new report, titled Next-Generation Sequencing: Global Market Analysis, Insights and Forecast, 2019-2026 offers a detailed analysis of the competitive landscape and market dynamics.

The demand for cost-effective, accurate, and fast DNA sequencing technologies is increasing and this is further giving rise to the dominance of next-generation sequencing platforms. This will further give rise to advanced sequencing technologies for clinical purposes, which is anticipated to drive the market. Increasing advancements in bioinformatics are likely to increase the adoption of next-generation sequencing platforms to facilitate the diagnosis of rare diseases.

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As per the report, the global market is expected to rise at a ferocious CAGR of 22.2% between 2018 and 2026. In 2018, the market was worth US$ 6,335.2 Mn and the market will be a howling success as it is projected to reach US$ 31,411.3 Mn by 2026. The demand for next-generation sequencing methodologies is increasing rapidly across several applications such as diagnostics, clinical research among others.

The global economy is in absolute turmoil because of the COVID-19 pandemic outbreak. Some industries remain largely unaffected by the outbreak, some are thriving, but most are in doldrums. Although the healthcare industry is flourishing, certain markets within the industry are experiencing staggered growth. Wading through these troubled times is a difficult task and Fortune Business Insights aims at equipping your business with the most comprehensive market insights, collated and analyzed by our expert and experienced research team.

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Demand for Next-Generation Sequencing Services to Rise at a High Pace

Next-generation sequencing products encompass instruments, consumables, and software. According to the findings from the report, the next-generation sequencing product segment is anticipated to lead the market over the forecast period. This is ascribable to some primary factors such as new product launches, high usage of consumables, and easy availability.

Apart from next-generation sequencing products, next-generation sequencing services are expected to grow at a higher CAGR during the forecast period. The need for novel platforms regarding advancements in bioinformatics and fast DNA data interpretation are paving way for start-ups, especially for next-generation sequencing services. With the introduction of next-generation sequencing, the cost of sequencing has reduced over the years.

This is positively impacting the growth of this segment. Furthermore, the adoption of next-generation sequencing-based testing is increasing across the world. This, coupled with attractive healthcare reimbursement policies, is expected to drive the segments growth in the projected period.

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North America Leads the Way Owing to Active Government Support

Among regions, North America is anticipated to lead the global next-generation sequencing market through the forecast duration. Rapid developments in precision medicine, next-generation DNA sequencing, and diagnostics are creating growth opportunities for the market. The market is likely to expand as companies in the biotechnology sector are continuously involving in research and development (R&D) activities, which fuels the market demand. Adapative Technologies developed a next-generation sequencing diagnostic test called ClonoSEQ for patients suffering from acute lymphoblastic leukemia or ALL and minimal residual disease or MRD. The test received FDA approval in September 2018, which in turn, contributes to the growth of the market in North America.

Increasing investments in precision medicine and a growing number of strategic collaborations are driving the market in Europe. Driven by these factors, the market in this region is expected to expand in the forecast period. Apart from these two regions, Asia Pacific is expected to expand at a remarkable growth on account of rising geriatric population and growing awareness about reproductive health.

Saphetor SA and Swift Biosciences Inc.s Partnership Aims to Target Next-Generation Sequencing Panels

Strategic partnerships and new product launches are increasingly adopted by companies as these help them to maintain their position in the market. Some of the industry developments are mentioned below:

Companies such as Illumina, Inc., Thermo Fisher Scientific Inc., and F. Hoffmann-La Roche Ltd are likely to lead in the global next-generation sequencing market. This is mainly on account of their strong geographical presence and diversified product portfolio. Moreover, Illumina was leading in the global market owing to its rising product sales. New product launches also helped the company to strengthen its market position.

Some of the other players functioning in the global market are Agilent Technologies, Inc., BGI, PerkinElmer Inc., QIAGEN, Eurofins Scientific, PierianDx, Macrogen, Inc. among others.

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Next-Generation Sequencing Market Segmentation:

By Type

Products

Instruments & Software

Consumables

Services

By Application

Diagnostics

Research

Others

By End User

Research Institutes

Healthcare Facilities & Diagnostic Centers

Pharmaceutical & Biotechnological Companies

Contract Research Organization (CROs)

By Geography

North America (the USA and Canada)

Europe (UK, Germany, France, Italy, Spain, and Rest of Europe)

Asia Pacific (Japan, China, India, Australia, Southeast Asia, and Rest of Asia Pacific)

Latin America (Brazil, Mexico, and Rest of Latin America)

Middle East & Africa (South Africa, GCC, and Rest of the Middle East & Africa)

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Next-Generation Sequencing Market Size to Reach US$ 31,411.3 Mn, Introduction of Rapid Sequencing Technologies Drives the Market: Fortune Business...

Global digital PCR market size was USD 224.9 million in 2018 and is projected to reach USD 799.1 million by 2026, exhibiting a CAGR of 17.2% during…

Trusted Business Insights answers what are the scenarios for growth and recovery and whether there will be any lasting structural impact from the unfolding crisis for the Digital PCR market.

Trusted Business Insights presents an updated and Latest Study on Digital PCR Market 2019-2029. The report contains market predictions related to market size, revenue, production, CAGR, Consumption, gross margin, price, and other substantial factors. While emphasizing the key driving and restraining forces for this market, the report also offers a complete study of the future trends and developments of the market.The report further elaborates on the micro and macroeconomic aspects including the socio-political landscape that is anticipated to shape the demand of the Digital PCR market during the forecast period (2019-2029). It also examines the role of the leading market players involved in the industry including their corporate overview, financial summary, and SWOT analysis.

Get Sample Copy of this Report @ Digital PCR Market Size, Share & Industry Analysis, By Type (Droplet Digital PCR, Chip-based Digital PCR, and Others), By Product (Instruments and Reagents & Consumables), By Indication (Infectious Diseases, Oncology, Genetic Disorders, and Others), By End User (Hospitals & Clinics, Pharmaceutical & Biotechnology Industries, Diagnostic Centers, and Aca-demic & Research Organizations), and Regional Forecast, 2020-2029(Includes COVID-18 Business Impact)

The global digital PCR market size was USD 224.9 million in 2018 and is projected to reach USD 799.1 million by 2026, exhibiting a CAGR of 17.2% during the forecast period.

We have updated Digital PCR Market with respect to COVID-19 Impact. Inquire before buying

Digital PCR is a biotechnological advancement of conventional PCR. It is a high-throughput absolute quantitative method that provides a sensitive and reproducible way of measuring the amount of DNA or RNA in a sample. The rise in prevalence of infectious diseases, cancer, and genetic disorders, coupled with new product launches, increasing acquisitions, and the rise in the adoption of dPCR by reference laboratories are the key factors likely to drive the market growth during the forecast period. Moreover, increasing technological advancements in digital PCR are projected to boost the global demand for dPCR products in the upcoming years.

MARKET TRENDS

Shift of Healthcare Providers from Droplet to Chip-based Digital PCR will Drive the Market The shift from droplet to chip-based digital PCR for diagnosis of various diseases has been pivotal in the advancement of dPCR devices. In commercially available droplet dPCR, the droplets have to be transferred manually, leading to underestimation of the targets. Moreover, these kits also poses a risk of cross-contamination during the transfer of the sample. On the other hand, in chip-based digital polymerase chain reaction, droplets always run in channels, reducing the risk of cross-contamination. This innovation is likely to increase its adoption for the diagnosis of various diseases in near future. Increasing incidence of infectious diseases and the growing prevalence of cancer are propelling the shift of healthcare providers for diagnosis of these conditions through chip-based digital PCR devices. This is projected to further propel the market growth during the forecast period.

MARKET DRIVERS

Technological Advancements in dPCR to Boost the Market Growth Digital polymerase chain reaction (PCR) is the refinement of conventional PCR that can be used to directly quantify and clonally amplify nucleic acids. Increasing research and development activities and growing demand for innovative devices have led to the introduction of various such technologically advanced devices offering great benefits to the Digital PCR market. The introduction of such innovative devices such as droplet, chip-based, beam, and crystal dPCR by key market players is anticipated to drive the global market growth during the forecast period. For instance, in November 2019, Bio-Rad Laboratories, Inc. announced the launch of QX ONE Droplet Digital PCR System, which provides an absolute measurement of target DNA molecules with more accuracy, precision, and sensitivity.

Growing Adoption of dPCR Over Real-time PCR to Boost the Market Globally Digital PCR helps to deliver a complete measure of target nucleic acid molecules than the relative measure achieved from the real-time PCR. This absolute DNA quantification allows for reproducibility, sensitivity, and precision, further enabling researchers to quantify smaller differences and precisely measure minor variants. These advantages are leading to growing adoption of dPCR over conventional and other PCR methods, which is likely to augment the digital PCR market growth during the forecast period.

MARKET RESTRAINT

High Cost of Digital PCR Devices and Lack of Awareness in Emerging Countries to Hamper Growth

Despite the increasing incidence of infectious diseases, cancer, and genetic disorders worldwide, and high prevalence of these conditions in emerging countries, the adoption digital polymerase chain reaction techniques is limited in these regions. This is attributable to the lack of awareness about the dPCR and its advanced types in less-developed countries. In addition to this, the high cost of these devices further restricts the market penetration. However, initiatives taken by leading players to enhance the awareness about the availability of dPCR for the diagnosis of various diseases, establishment of strong distribution network, and increased new product launches are likely to create lucrative growth opportunities for the expansion of the market in emerging regions.

SEGMENTATION

By Type Analysis

Droplet Digital PCR Dominated the Global Market

Based on type, the digital PCR market is segmented into droplet, chip-based, and others. The droplet digital polymerase chain reaction segment dominated the market in 2018. Technological advances along with various new product launches by key players have been instrumental in the dominance of droplet dPCR in the global market. For instance, in August 2018, Merck KGaA launched the BioReliance Droplet dPCR Technology for cell and gene therapy development.

Chip-based digital PCR segment is projected to register a significant CAGR during the forecast period. These dPCR devices overcome the cross-contamination drawback of droplet digital PCR leading to the increasing preference of healthcare providers for chip-based dPCR. This factor is projected to fuel the demand for this dPCR type, and drive the digital polymerase chain reaction market growth during the forecast period.

By Product Analysis

Instruments Segment to Generate the Highest Revenue During the Forecast Period In terms of product, the market is segmented into instruments, reagents & consumables. the instruments segment emerged dominant owing to their high costs. The reagents segment held comparatively low market share due to small volumes of reagents required for analysis. However, the segment is expected to grow at a significantly high growth rate owing to growing number of diagnostic procedure globally.

By Indication Analysis

Clinically Proven Effectiveness of dPCR in Infectious Diseases to Aid Dominance of the Segment In terms of indication, the market is segmented into infectious diseases, oncology, genetic disorders, and others. Among them, the infectious diseases segment is anticipated to dominate the dPCR market. The clinically proven potential advantages of droplet dPCR in diagnosis of infectious diseases, including viral, bacterial, and parasitic infections is a primary reason for the dominance of this segment in the global polymerase chain reaction (PCR) market. According to an article published by the National Center for Biotechnology Information (NCBI), dPCR provides a more sensitive, accurate, and reproducible detection of pathogens and is a better choice than real-time polymerase chain reaction for clinical applications. Oncology is among the other major indication areas for dPCR. Growing prevalence of the condition, along with the introduction of new product launches in the market is expected to drive the segment during the forecast period.

By End User Analysis

Higher Adoption of dPCR devices by Diagnostic Centers to Enable Dominance of the Segment Based on end-user, the dPCR market segments include hospitals & clinics, pharmaceutical & biotechnology industries, diagnostic centers, and academic & research organizations. The emergence of diagnostic centers for infectious diseases, genetic diseases and other conditions in developed and emerging countries is attracting a large patient pool suffering from chronic and acute conditions. The increasing number of diagnostic centers and favorable reimbursement support provided by these settings are some of the major factors responsible dominance of the segment. These are some of the factors that are responsible for the dominance of this segment throughout the forecast period.

REGIONAL ANALYSIS

The dPCR market size in North America stood at USD 96.5 million in 2018. The market in the region is characterized by high prevalence and diagnosis rates for infectious diseases. Besides this, high awareness among patient population towards new diagnostic options and availability of advanced dPCR devices in the region, are helping the region dominate the market. Moreover, the rising incidence of various types of cancer and metabolic diseases requiring advanced diagnosis and therapies are fueling the market growth in Europe. For instance, according to GLOBOCAN 2018, around 4,229,662 new cancer cases were reported in Europe. The market in Asia-Pacific is projected to register a comparatively higher CAGR during the forecast period. Growing patient awareness regarding advanced digital polymerase chain reaction devices and new product launches by market players, are projected to drive the digital polymerase chain reaction (dPCR) market in Asia Pacific during the forecast period. The market in Latin America and the Middle East & Africa is currently in nascent stage. However, the developing healthcare infrastructure in these regions and growing prevalence of infectious and genetic diseases are projected to increase the demand for the digital polymerase chain reaction products during the forecast period.

KEY INDUSTRY PLAYERS

Diverse Portfolio of Leading Players and Strong Focus on Partnerships and Acquisition to Strengthen Their Market Position

Competition landscape of the digital PCR market is consolidated with few players holding the dominant share. Bio-Rad Laboratories, Inc., Thermo Fisher Scientific Inc., and Sysmex Inostics are some of the leading industry players. A diversified product portfolio of droplet and chip-based digital PCR along with constant innovations by the companies, leading to new product introductions, are prominent factors responsible for the dominance of these companies. Moreover, increasing focus on partnerships and acquisition strategies in the dPCR is likely to attract more digital PCR market revenue in the future. January 2017 Bio-Rad Laboratories, Inc. acquired RainDance Technologies with an aim to expand their product portfolio in dPCR and liquid biopsy.

LIST OF KEY COMPANIES PROFILED:

KEY INDUSTRY DEVELOPMENTS:

July 2019 Sysmex Inostics launched OncoBEAM test which uses BEAMing enhanced digital PCR for liquid biopsy for cancer patients.

January 2019 QIAGEN announced plans for a global launch in 2020 of a new series of digital PCR platforms. This is based on the technology developed by FORMULATRIX combined with QIAGENs assay development expertise.

REPORT COVERAGE

The report provides a detailed analysis of the market and focuses on key aspects such as leading companies, product types, and leading applications of the product. Besides this, the report offers insights into the market trends and highlights key industry developments. In addition to the aforementioned factors, the Digital PCR market report encompasses several factors that have contributed to the growth of the market over the recent years.

Report Scope & Segmentation

Segmentation

By Type

By Product

By Indication

By End User

By Geography

Looking for more? Check out our repository for all available reports on Digital PCR in related sectors.

Quick Read Table of Contents of this Report @ Digital PCR Market Size, Share & Industry Analysis, By Type (Droplet Digital PCR, Chip-based Digital PCR, and Others), By Product (Instruments and Reagents & Consumables), By Indication (Infectious Diseases, Oncology, Genetic Disorders, and Others), By End User (Hospitals & Clinics, Pharmaceutical & Biotechnology Industries, Diagnostic Centers, and Aca-demic & Research Organizations), and Regional Forecast, 2020-2029(Includes COVID-18 Business Impact)

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Global digital PCR market size was USD 224.9 million in 2018 and is projected to reach USD 799.1 million by 2026, exhibiting a CAGR of 17.2% during...

Brain Stimulators Market: Rise in incidence of neurological disorders is expected to drive the market – BioSpace

Brain stimulation therapy is a procedure to treat certain mental disorders. Brain stimulation therapies involve activating or inhibiting brain activity directly with electricity. Electricity is provided directly by invasive or non-invasive brain stimulators placed on the scalp. Brain stimulators investigate the effects of electricity on basic processes such as gene expression and other aspects of molecular biology, neurochemical regulation, functional brain activity, sensorimotor function, and cognitive and affective processes. The stimulation technique uses a probe to outline patterns of neural connectivity in the brain and provides the information of brain activity in the form of graphs. The functioning of brain stimulators depends on battery powered adapter for the display of output on the patient monitor. Moreover, the system incorporates visual indicators of performance in order to provide the physicians an optimal performance. However, new developments in computer technology and rapidly growing cranial stimulation technique allows the scientific community to focus on the best practice within the medical industry, which is paving the way for early diagnosis and treatment of mental diseases.

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The global brain stimulators market is driven by rapid development in technology, funding for digital health companies, rise in incidence of neurologicaldisorders, increase in government support for research & development, and adverse effects of present diagnostic & drug therapies. According to the U.S. Food and Drug Administration, deep brain stimulators were approved for treatment of movement disorders such as essential tremor, Parkinson's disease and dystonia, and recently, obsessive-compulsive disorder. Rise in prevalence of Parkinson's disease, epilepsy, dystonia, and other chronic pain is expected to drive the global brain stimulators market. However, long approval time, high cost of research & development and devices, and lack of awareness are factors expected to restrain the global brain stimulators market during the forecast period.

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The global brain stimulators market can be segmented based on product type, end-user, application, and region. In terms of product type, the market can be classified into transcranial magnetic stimulators, electroconvulsive therapy stimulators, deep brain stimulators, and others. Based on application, the global brain stimulators market can be categorized into Parkinsons disease, essential tremor, dystonia, and others. In December 2017, Boston Scientific Corporation received the U.S. Food and Drug Administration (FDA) approval for the sale of Vercise Deep Brain Stimulation (DBS) System to treat the symptoms of Parkinsons disease (PD), a degenerative condition that affects more than one million people in the U.S. and 10 million globally. Hence, deep brain stimulators as an invasive device will be more prominently used in the near future. In terms of end-user, the market can be divided into diagnostic laboratories, clinics, hospitals, and others.

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Geographically, the global brain stimulators market can be segmented into North America, Europe, Asia Pacific, and Rest of the World. North America dominated the global market due to advanced technology used in medical devices, increase in incidence of mental illnesses among the geriatric population, significant awareness, and high per capita health care expenditure. Early-phase diagnosis, treatment of disease, and after surgery effects drive the biotechnology and medical device market which in turn is expected to propel the brain stimulators market in North America. Europe was the second largest market for brain stimulators due to favorable government policies regarding health care infrastructure, high incidence of brain diseases due to environmental conditions, and awareness programs about different types of brain disorders.The market in Asia Pacific is likely to grow at a rapid pace due to increase in the geriatric population, rise in awareness among people, and increase in per capita expenditure. Additionally, economic growth supports improvement in health care infrastructure in developing countries such as India, China, Brazil, and South Africa. Hence, these factors are expected to contribute to the growth of the brain stimulators market in Asia Pacific and Rest of the World.

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Major players operating in the global brain stimulators market include Medtronic plc, Boston Scientific Corporation, Abbott Laboratories, Aleva Neurotherapeutics SA, Deep Brain Innovations, LLC, and SceneRay.

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.

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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.

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Brain Stimulators Market: Rise in incidence of neurological disorders is expected to drive the market - BioSpace

Global mucopolysaccharidosis treatment market size was USD 1.98 billion in 2018 and is projected to reach USD 4.37 billion by 2026, exhibiting a CAGR…

Trusted Business Insights answers what are the scenarios for growth and recovery and whether there will be any lasting structural impact from the unfolding crisis for the Mucopolysaccharidosis Treatment market.

Trusted Business Insights presents an updated and Latest Study on Mucopolysaccharidosis Treatment Market 2019-2029. The report contains market predictions related to market size, revenue, production, CAGR, Consumption, gross margin, price, and other substantial factors. While emphasizing the key driving and restraining forces for this market, the report also offers a complete study of the future trends and developments of the market.The report further elaborates on the micro and macroeconomic aspects including the socio-political landscape that is anticipated to shape the demand of the Mucopolysaccharidosis Treatment market during the forecast period (2019-2029). It also examines the role of the leading market players involved in the industry including their corporate overview, financial summary, and SWOT analysis.

Get Sample Copy of this Report @ Mucopolysaccharidosis Treatment Market Size, Share & Industry Analysis, By Treatment (Enzyme Replacement Therapy (ERT), and Others), By Disease Type (Mucopolysaccharidosis Type I, Mucopolysaccharidosis Type II, Mucopolysaccharidosis Type IV A, Mucopolysaccharidosis Type VI, and Others), By Route of Administration (Intravenous, and Intracerebroventricular (ICV)), By End User (Hospitals, Specialty Clinics, and Others), and Regional Forecast, 2020-2029(Includes COVID-18 Business Impact)

The global mucopolysaccharidosis treatment market size was USD 1.98 billion in 2018 and is projected to reach USD 4.37 billion by 2026, exhibiting a CAGR of 10.4% during the forecast period.

We have updated Mucopolysaccharidosis Treatment Market with respect to COVID-19 Impact. Inquire before buying

MPS, also known as mucopolysaccharidosis, refers to a group of key rare metabolic disorders which is caused by the absence, deficiency or malfunctioning of lysosomal enzymes called glycosaminoglycan (GAGs). This inability to completely down the sugar, leads to the abnormal accumulation of certain compounds such as dermatan sulfate, heparan sulfate, and keratan sulfate, which interferes with normal cell function of the body. One of the characteristic symptoms of MPS is the presence of severe neurological symptoms that are present in several types of MPS such as Hunter syndrome and Sanfilippo syndrome and a number of clinical stage companies are involved in the R&D of new therapeutics for the management of these symptoms.MARKET TRENDSIncreasing R&D for Rare Diseases to Drive the Market GrowthMarket trends present in the mucopolysaccharidosis (MPS) treatment market is the increasing number of pipeline candidates from major companies in the therapeutic area of rare diseases or orphan diseases. Since, mucopolysaccharidosis is considered to be a potentially serious orphan disease group, some prominent clinical stage biopharmaceutical companies such as ArmaGen and REGENXBIO Inc. are involved in the clinical trials for new therapeutics. This increasing initiatives into the development of rare diseases therapies is due to the fact that development of major blockbuster drugs is more possible in these rare diseases as compared to the conventional pharmaceutical portfolios. Another driving factor for this trend is that the pharmaceutical companies are mandated to conduct larger outcome studies for conventional medications or therapies. This is applicable for therapies to treat diseases such as heart disease and diabetes in comparison to rare disorders such as the several disease types of mucopolysaccharidosis. This is projected to further positively drive the mucopolysaccharidosis treatment market growth during the forecast period.

MARKET DRIVERS

Need for Advanced Therapeutics Due to Unmet Clinical Needs and Better Treatment Outcomes to Propel Market GrowthThe lack of presence of a wide range of therapeutics for the patients, which has led to the creation of the monopoly of one type of therapy, enzyme replacement therapy is the most critical driver for the market growth. For instance, for the treatment of mucopolysaccharidosis type II (Hunter syndrome), Elaprase is the most commonly used therapeutic, an enzyme replacement therapy (ERT). This ERT is a prohibitively expensive therapy option and often patients from developing countries cannot easily access this therapeutic due to cost constraints. Other therapeutics for MPS treatment include Vimizim (elosulfase alfa) which is administered for the treatment of MPS IV A (Morquio syndrome) and which at the time of launch in the market is estimated to cost US$ 380,000 for the disorders annual treatment. Hence, due to the six-figure costs of most of the enzyme replacement therapy (ERT), the patients in the emerging countries such as India, often cannot afford these therapeutics despite increasing initiatives for improved access from various governments. Anticipated launches of cheaper alternatives and advanced therapeutics is anticipated to drive the growth of the global MPS treatment market during the forecast period.Another critical driving factor is the increasing need for advanced therapeutics due to unmet clinical needs and better treatment outcomes. During the current period therapeutics, such as Elaprase, and ALDURAZYME are not capable of crossing the blood-brain barrier, a key requirement for effective management of the disorder. So, these therapeutics are not able to appropriately address the neurological symptoms and complications of the patients of mucopolysaccharidosis (MPS), who are most severely affected. For instance, in the patients of Hunter syndrome with the most severe symptoms, which affects approximately two-thirds of the total patient population, the neurological symptoms are severely disabling. The therapeutics used currently are not able to treat the neurological symptoms, thus a significant proportion of the population remains in the need for appropriate treatment. This is anticipated to propel the demand for effective therapeutics which aid in the management of all types of the symptoms of the mucopolysaccharidosis and drive the growth of the MPS treatment market size during the forecast period.Increasing Number of Clinical Stage Candidates in R&D Pipelines of Major Players to Drive MarketThere is a significant number of pipeline candidates for the development of advanced therapeutics for an increasing number of rare disorders and the several types of mucopolysaccharidosis is one of the key disease groups for these companies due to the severity of the disease. According to the National Organization for Rare Disorders (NORD) (NORD), the prevalence of all disease types of mucopolysaccharidosis is estimated to be 1 in 25,000 births. In April 2019, it was reported that in Japan, an estimated 150 individuals suffered from mucopolysaccharidosis type II (Hunter syndrome). Such driving factors are further leading to the presence of prominent and important pipeline candidates. ArmaGen, Denali Therapeutics, and REGENXBIO Inc., all have pipeline candidates for the various types of MPS in various stages of clinical trials. The above factors combined with the need for advanced therapeutics is further anticipated to drive the demand for these therapeutics and stimulate the MPS market growth.MARKET RESTRAINTHigh Cost Therapeutics Coupled with Poorer Diagnosis Rates and Low Treatment Rates in Emerging Countries to Restrain the Market In recent times, globally due to sophistication in diagnostics, there is a higher prevalence of MPS in several countries which includes emerging regions such as Asia, there are certain factors that are restraining the widespread usage of these therapies. Due to the high costs associated with enzyme replacement therapies (ERT), which is the mainstay treatment for MPS, one of the major factors limiting the growth of the market is low levels treatment rates in emerging countries. This severely limits the number of patients undergoing treatment, and a significant proportion of the patients of MPS are left without treatment due to their inability to afford treatment. Often, these therapies are not available to the patients in the developing countries due to lack of awareness and also options for the payment of these therapies. The governments in these countries often do not have appropriate awareness regarding these diseases and do not adequately reimburse them. Another key limiting factor is the delays associated with the diagnosis of mucopolysaccharidosis. A study published in 2018, in the Orphanet Journal of Rare Diseases, indicated that there is a failure to shorten the diagnostic delay in the two ultra-orphan diseases of mucopolysaccharidosis types I and mucopolysaccharidosis type III. Factors as such are further expected to restrain the market growth.SEGMENTATIONBy Treatment AnalysisEnzyme Replacement Therapy (ERT) is Expected to Dominate the Global MarketBased on treatment, the global market is segmented into enzyme replacement therapy (ERT), and others. The primary treatment for lysosomal storage disorders, a group of disorders to which MPS belongs, is enzyme replacement therapy (ERT). These enzyme replacement therapies, aim to replace or generate the missing or deficient enzyme in the body, due to which mucopolysaccharidosis occurs. Hence, the enzyme replacement therapy (ERT) segment dominated the mucopolysaccharidosis treatment market share in 2018. The only major therapeutics approved by the regulatory agencies globally treatment of MPS, is enzyme replacement therapy (ERT). For example, in case of mucopolysaccharidosis type II (Hunter syndrome), the only two approved products are Elaprase and Hunterase which are both ERTs, and have been instrumental in the dominance of this segment in the global market.The others segment is anticipated to grow at a comparatively stronger CAGR. The mucopolysaccharidosis (MPS) treatment market growth during the forecast period is anticipated to be driven by the growing number of clinical trials involving the usage of the hematopoietic stem cell transplant (HSCT). Other than stem cell transplantation, a number of prominent clinical stage biopharmaceutical companies are conducting trials on gene therapy including Sangamo Therapeutics, which has a pipeline candidate for Hunter syndrome.By Disease Type Analysis To know how our report can help streamline your business, Speak to Analyst var get_chart_name1=

MPS II (Hunter Syndrome) to Dominate the Disease Type due to Presence of Key Products In terms of disease type, the market is segmented into mucopolysaccharidosis type I also known as Hurler syndrome/Hurler-Scheie syndrome/Scheie syndrome, mucopolysaccharidosis type II also known as Hunter syndrome, mucopolysaccharidosis type IV A also known as Morquio A syndrome, mucopolysaccharidosis Type VI also known as MaroteauxLamy syndrome, and others. The mucopolysaccharidosis type II (Hunter Syndrome) is expected to dominate the market due to the presence of key products such as Hunterase and Elaprase. Both of these products accounts for a significant portion of the market revenue share. The other segments in the global mucopolysaccharidosis treatment market which includes mucopolysaccharidosis type I, mucopolysaccharidosis type IV A and mucopolysaccharidosis Type VI, all of which have key products offerings attributable to them. Such factors are anticipated to drive the market during the forecast period.The others segment includes mucopolysaccharidosis type VII (Sly syndrome) whose product offering of MEPSEVII (vestronidase alfa-vjbk) is anticipated to register a strong growth rate during the forecast period. Growing prevalence of the mucopolysaccharidosis types, along with introduction of major enzyme replacement therapy (ERT) products in the market, is expected to drive the growth of the segment during the forecast period.By Route of Administration AnalysisPresence of Major Therapeutics in Intravenous Form to Aid Dominance of the SegmentIn terms of route of administration, the market is segmented into intravenous, and intracerebroventricular (ICV). The intravenous type is anticipated to account for the majority of the revenue share of the route of administration segment, because most of the therapeutics for MPS treatment are to be administered through intravenous injections only. For example, the most prominent therapeutic for Hunter syndrome, Elaprase is administered intravenously. Intravenous therapeutics holds a monopoly over the mucopolysaccharidosis treatment market and is anticipated to hold control over its market share in the forecast period.The only other approved therapeutic that is not in an intravenous form and the route of administration is intracerebroventricular (ICV), is Hunterase. Growing approvals in other countries of these therapeutic, along with anticipated introduction in other countries across the globe, is expected to drive the growth of the segment during the forecast period.By End User AnalysisIncreased Need for Administration of Therapeutics at Hospitals to Enable Dominance of the SegmentOn the basis of end user, the market is segmented into hospitals, specialty clinics, and others. The key factor aiding in the dominance of hospitals segment is that the therapeutics used in MPS treatment can often be administered by trained medical professionals who can administer these therapeutics in settings such as hospitals with care and proper guidelines. This allows for the proper adherence to the MPS treatment guidelines and administration of critical therapeutics which have to be administered intravenously, in a safe environment to manage the increased risk of infusion site infections.Growing number of specialty clinics, due to increasing need for specialist care of a higher degree for the treatment and management of several types of mucopolysaccharidosis (MPS) patients, are some of the major factors responsible for this segments dominance forecast period. Also, a number of these specialty clinics are based in the developed countries, resulting in a strong market revenue share. Also, the increasing government initiatives such as the Brazilian Governments initiative for the setting up of treatment guidelines is also anticipated to contribute to the segments growth.

REGIONAL ANALYSISNorth

segmentation(get_label12,get_values2,chartid);The mucopolysaccharidosis (MPS) treatment market size in North America stood at USD 1,019.4 million in 2018. Some of the characteristics of this market is higher diagnosis and treatment rates for a number of rare disorders, coupled with adequate reimbursement policies for these therapeutics. These factors, coupled with higher awareness among patient population towards advanced treatment options, increasing diagnostic sophistication, presence of significant clinical trials, and the presence of major clinical stage biopharmaceutical companies are their pipeline candidates, are responsible for highest share of the region in the global market. The market in Europe is anticipated to account for the second largest revenue share during the forecast period. The presence of key products in the region such as Elaprase and ALDURAZYME, is anticipated to drive the demand for MPS treatment in Europe during 2019-2026. Asia-Pacific is projected to register the highest CAGR during the forecast period. Anticipated launch of some therapeutics such as Hunterase in Japan in Asia Pacific, the upcoming product launches in the region due to increased regulatory applications for approvals, and the presence of a strong potential patient population base in the region, together are projected to fuel the mucopolysaccharidosis (MPS) treatment market growth in Asia Pacific during forecast period. The rest of the world market comprises of Latin America and the Middle East & Africa and is currently in nascent stage. However, developing healthcare infrastructure in these regions, increased government initiatives and growing awareness of rare disorders is projected to fuel the mucopolysaccharidosis treatment market demand during forecast period.KEY INDUSTRY PLAYERSKey Product Offerings and Core Focus on Mucopolysaccharidosis of Shire (Takeda Pharmaceutical Company Limited) and BioMarin, to Propel the Company to a Leading Position Competition landscape of mucopolysaccharidosis (MPS) treatment market depicts a competitive landscape dominated by a few key players. Shire (now owned by Takeda Pharmaceutical Company Limited), whose prominent product of Elaprase commands a strong revenue share, and BioMarin are some of the key market players. For example, the product offering of Shire (Takeda Pharmaceutical Company Limited) of Elaprase (idursulfase), its efficiency in terms of treatment outcomes and also its indispensability in treatment of MPS II, are prominent factors responsible for the dominance of the company in the mucopolysaccharidosis treatment. BioMarin has the key product offerings of ALDURAZYME, VIMIZIM, and NAGLAZYME in its product portfolio, covering three types of mucopolysaccharidosis.However, ArmaGen, and REGENXBIO Inc., are some of the prominent clinical stage biopharmaceutical companies such who have entered the clinical trials with their potential drug candidates. This is projected to positively impact the global market as these companies are poised to gain market share during the forecast period through key regulatory approvals.LIST OF KEY COMPANIES PROFILED :Shire (Takeda Pharmaceutical Company Limited)Denali TherapeuticsArmaGenREGENXBIO Inc.Sangamo TherapeuticsBioMarinLysogeneAbeona Therapeutics Inc.Ultragenyx PharmaceuticalGenzyme CorporationOthersKEY INDUSTRY DEVELOPMENTS:February 2020 Lysogene, a key clinical stage biopharmaceutical company, received the FDA fast track designation for the pipeline candidate of LYS-SAF302 gene therapy in MPS IIIA, also known as the Sanfilippo syndrome.April 2019 Abeona Therapeutics, a key integrated leader in cell and gene therapy development, received the FDA Fast Track Designation for their pipeline candidate of ABO-101 for the treatment of Sanfilippo syndrome type B or MPS IIIB.August 2018 Ultragenyx announced the approval of Mepsevii (vestronidase alfa) in Europe for the treatment of mucopolysaccharidosis VII, also known as Sly syndrome.REPORT COVERAGEThe mucopolysaccharidosis treatment market report provides a detailed analysis of the market and focuses on key aspects such as prevalence of key mucopolysaccharidosis types by key regions (2018), pipeline analysis, key industry developments, regulatory scenario- by key regions, overview of emerging treatments for mucopolysaccharidosis, and reimbursement scenario by key regions. Besides this, the report offers insights into the market trends and highlights key industry developments. In addition to the aforementioned factors, the report encompasses several factors that have contributed to the growth of the market over the recent years.

Report Scope & Segmentation

ATTRIBUTEDETAILSStudy Period 2015-2026Base Year 2018Forecast Period 2019-2026Historical Period 2015-2017Unit Value (USD billion)SegmentationBy TreatmentEnzyme Replacement Therapy (ERT)OthersBy Disease TypeMucopolysaccharidosis Type IMucopolysaccharidosis Type IIMucopolysaccharidosis Type IV AMucopolysaccharidosis Type VIOthersBy Route of Administration IntravenousIntracerebroventricular (ICV)By End User HospitalsSpecialty ClinicsOthersBy GeographyNorth America (U.S. and Canada)Europe (U.K., Germany, France, Italy, Spain, and Rest of Europe)Asia-Pacific (Japan, China, India, South Korea, and Rest of Asia-Pacific)Rest of the World

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Quick Read Table of Contents of this Report @ Mucopolysaccharidosis Treatment Market Size, Share & Industry Analysis, By Treatment (Enzyme Replacement Therapy (ERT), and Others), By Disease Type (Mucopolysaccharidosis Type I, Mucopolysaccharidosis Type II, Mucopolysaccharidosis Type IV A, Mucopolysaccharidosis Type VI, and Others), By Route of Administration (Intravenous, and Intracerebroventricular (ICV)), By End User (Hospitals, Specialty Clinics, and Others), and Regional Forecast, 2020-2029(Includes COVID-18 Business Impact)

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Global mucopolysaccharidosis treatment market size was USD 1.98 billion in 2018 and is projected to reach USD 4.37 billion by 2026, exhibiting a CAGR...

From stem cells to islets hope for treatment of type 1 diabetes – News – The University of Sydney

According to Professor Liddle, this study used stem cells derived from human umbilical vein and human fat that were re-programmed to generate human islet-like organoids (HILOs).

Pancreatic islets are regions in the pancreas responsible for the production of hormones and insulin.

Pancreatic islets contain multiple cell types, not just insulin-producing beta cells. The research team created three-dimensional HILOs that not only include beta-like cells (the cells that produce, store and release insulin in the islets of the pancreas), but also other supporting cell types found in normal islets, said Professor Liddle.

Under the microscope, and using gene sequencing analysis, we are able to show that the three-dimensional HILOs are very similar to human islets. When the HILOs are transplanted into diabetic mice, they secrete insulin when blood glucose levels are high, just as normal islets would.

While human pancreatic islet transplantation has been a major advancement in treating severe cases of type 1 diabetes, the availability, quality and limited cellular longevity of this approach limits its application.

Pancreatic islet transplantation currently involves implanting insulin-producing islet cells from a deceased human donor into the liver of a person with type 1 diabetes. When successful, the procedure can control blood glucose levels, reduce the frequency and severity of hypoglycaemic episodes and potentially eliminate the need for regular insulin injections. A number of transplants are usually needed, and immunosuppressant drugs to prevent the immune system from attacking the transplanted cells are also required.

While the procedure is now funded by the Australian Government, pancreatic islet transplantation is currently limited to people with severely unstable type 1 diabetes, particularly those for whom insulin therapy alone is not effective and who experience recurrent and severe hypoglycaemic episodes.

Professor Philip OConnell is Executive Director at The Westmead Institute for Medical Research and pioneered pancreatic islet transplantation in Australia. Almost 20-years ago, he led Australias first pancreatic islet transplantation trials at Westmead Hospital and The Westmead Institute for Medical Research. Today, he continues his research, aiming to improve this procedure and develop islet transplantation as a mainstream treatment for type 1 diabetes.

Professor OConnell, who was not involved in this research study, said, Pancreatic islet transplantation has saved hundreds of lives around the globe however, it has its limitations. For example, pancreatic islets are taken from deceased donors, and the wait for donor islets can be lengthy. Once donor islets are obtained, not all are suitable for transplantation.

This research indicates the potential to alleviate some of these issues. Stem cells derived from readily available human tissues can be expanded then re-programmed into potentially unlimited numbers of islets that are suitable for transplantation.

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From stem cells to islets hope for treatment of type 1 diabetes - News - The University of Sydney

Adipose Tissue-derived Stem Cell Therapy Market Share Analysis, Application, Strategies of Key Players & Forecast to 2025 – Scientect

The Global Adipose Tissue-derived Stem Cell Therapy Market report offers users the detailed study of the market and its main aspects. The study on Global Adipose Tissue-derived Stem Cell Therapy Market, offers profound understandings about the Adipose Tissue-derived Stem Cell Therapy Market covering all the essential aspects of the market. The report provides competitive pipeline landscape of the Global Factors like production, market share, revenue rate, regions and key players define a market study start to end. This report gives an overview of market valued in the year 2019 and its growth in the coming years till 2025.

This study covers following key players: 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. AlloCure Tissue Genesis Antria Cellleris Corestem Celgene Corporation Mesoblast Cytori Therapeutics Pluristem Therapeutics Intrexon Lonza BioRestorative Therapies Pluristem Therapeutics iXCells Biotechnologies Cyagen Celltex Therapeutics Corporation

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The study is done with the help of analysis such as SWOT analysis and PESTEL analysis. It consists of the detailed study of current market trends along with the past statistics. The past years are considered as reference to get the predicted data for the forecast period. The report covers complete analysis of the Adipose Tissue-derived Stem Cell Therapy Market on the basis of regional and Global level. Various important factors such as market trends, revenue growth patterns market shares and demand and supply are included in almost all the market research report for every industry.

There are different marketing strategies that every marketer looks up to in order to ace the competition in the Global market. Some of the primary marketing strategies that is needed for every business to be successful are Passion, Focus, Watching the Data, Communicating the value To Your Customers, Your Understanding of Your Target Market. There is a target set in market that every marketing strategy has to reach. In addition, it also covers political and social factors which is likely to affect the growth of the market. It also covers and analysis several segments which are present in the market. A significant development has been recorded by the market of Adipose Tissue-derived Stem Cell Therapy, in past few years. It is also for it to grow further. Various important factors such as market trends, revenue growth patterns market shares and demand and supply are included in almost all the market research report for every industry.

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Market segment by Type, the product can be split into Segmentation by type: breakdown data from 2015 to 2020 in Section 2.3; and forecast to 2025 in section 10.7. Autologous Stem Cells Allogeneic Stem Cells

Segmentation by application: breakdown data from 2015 to 2020, in Section 2.4; and forecast to 2025 in section 10.8. Therapeutic Application Research Application

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 Middle East & Africa Egypt South Africa Israel Turkey GCC Countries

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. AlloCure Tissue Genesis Antria Cellleris Corestem Celgene Corporation Mesoblast Cytori Therapeutics Pluristem Therapeutics Intrexon Lonza BioRestorative Therapies Pluristem Therapeutics iXCells Biotechnologies Cyagen Celltex Therapeutics Corporation

Market segment by Application, split into Segmentation by application: breakdown data from 2015 to 2020, in Section 2.4; and forecast to 2025 in section 10.8. Therapeutic Application Research Application

One of the ways for the estimation for the growth of the market is estimation of the market share by the regions which is likely to contribute to the growth of the market in the estimated forecast period. In this, the growth and fall of each region is covered which is likely to boost the growth of the Adipose Tissue-derived Stem Cell Therapy Market. In addition, to determine and use precise methods, research methodology such as the qualitative and quantitative data is used for the estimation and determination of the Global Adipose Tissue-derived Stem Cell Therapy Market.

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Adipose Tissue-derived Stem Cell Therapy Market Share Analysis, Application, Strategies of Key Players & Forecast to 2025 - Scientect

Auxolytic’s Nutrient-Based Shut-Off Switch Boosts Cell Therapy Safety Without Transgenes – BioSpace

Auxolytic has developed a nutrient-based safety-switch for cell therapies that doesnt rely on introducing transgenes. The process, auxotrophy, uses the engineered inability of an organism to synthesize a compound required for its survival to allow physicians to turn off a gene therapy if serious side effects develop.

The work currently is in preclinical phases, in humanized cells in mice, and appears promising. When it advances to clinical applications, patients receiving cell therapies (such as CAR T, stem cell, and TCR therapies) containing this safety switch would be given supplements of a particular nutrient uridine, in this case. If the cell therapy went awry, patients could simply stop taking the uridine supplement and the cellular therapy would cease to function.

A paper published in Nature Biotechnology describes how the off switch could be engineered into cell therapies. Basically, it says, the approach knocks out the gene that disrupt(s) uridine monophosphate synthetase (UMPS) in the pyrimidine de novo synthesis pathway in cell lines, pluripotent cells, and primary human T cells.

This knockout makes proliferation of the cell therapy dependent on the external supply of uridine. Therefore, researchers can control cell growth by modulating the uridine supply in vitro and, importantly, in vivo after transplantation.

In the movie Jurassic Park, the dinosaurs were engineered to need lysine. If they escaped, there was no lysine to keep them alive. This therapy is very similar, founder and CEO James Patterson, M.D., Ph.D., told BioSpace.

Rather than lysine, Auxolytic uses uridine as the controlling nutrient. Uridine is important in carbohydrate metabolism and is found in yeast, tomatoes, broccoli, sugarcane and other foods, and also can be produced by the body when inadequate amounts are consumed in the diet.

The quantities available through the diet or produced by the body, however, arent high enough to sustain the engineered cells, Patterson said. Evidence comes from a rare genetic disease, orotic aciduria. Patients with that condition have a mutation in the UMPS gene that causes them to produce insufficient levels of the enzyme that breaks down orotic acid. They often die at very young ages if not supplied with quantities of pure uridine. This shows that a normal diet wont compensate.

Patients of cell therapies that incorporate Auxolytics nutrient-based safety switch likely would be able to eat their usual foods, but with nuridine added as a supplement. The approach Dr. Patterson developed hasnt been tested in patients yet.

This is the same nutrient I worked with in yeast, but now in human cells, Patterson said. Early work shows that only the engineered cell therapy would be affected by uridine modulation. Within one week of withdrawing the uridine, the engineered cells were inactive and unable to proliferate. Normal cells continued to function as usual.

At age 27, Patterson already has worked with many of the thought leaders in medicine and biomedical research while pursuing his M.D. and Ph.D. degrees at the University of Cambridge and the Francis Crick Institute. Beginning early during his university days, he performed research placements at the University of Zurich, The Gurdon Institute, The Whitehead Institute and The Cambridge Stem Cell Initiative.

This nutrient-based approach to controlling cell therapy is the direct result of that body of experience.

During my M.D./Ph.D. training, I became interested in cell therapy and its potential for curing patients, but there were safety risks. My Ph.D. work focused on yeast biology, studying how cells control their size. There, the idea of nutrient-based cell control was commonplace, but no one was working on this in human cells, he said. I became interested in science when I was very young, so during my undergraduate work I made sure I was thinking about the science being done in the labs in addition to what I was learning in lectures. Theres a difference.

Lectures lay the scientific foundation, but lab work is cutting-edge and forward-thinking.

I started working in labs when I was 19 in Zurich. I spent all my summers working in labs, asking a range of questions and working in lots of different systems, Patterson said.

Such broad exposure proved foundational for Auxolytic.

As you can see, this is a yeast technology. Thats not where you usually go to look for cell therapy ideas, he added.

He advises students early on to get into exciting labs that are doing interesting, fundamental science. Ask basic questions of how cells work, for example. You never know what youll find that could be applicable to the clinic. Jumping to clinical research (too early) causes you to lose the blue sky thinking.

Auxotyic, based in Cambridge, UK, is, for now, a virtual company of onebut with ample advisors.

Theyve helped along the way in the academic sector and also in the management of business, Patterson said. Those mentors include seasoned industry veterans who know what it takes to take a drug from bench to bedside, and who understand patenting and licensing.

The next step for Auxolytic, scientifically, is to identify potential applications around selecting for differentiated cells from induced pluripotent stem cells (iPSCs). Much of the scientific work is being done in collaboration with the Matthew H. Porteus lab at Stanford University.

On the business side, he continued, Were excited to get this into the hands of big cell therapy companies that currently are making cell therapies without a safety switch. Were looking to partner with them to get this to patients.

Auxolytic is talking with several interested companies. People recognize the need for a safety switch and are very excited, Patterson said. Discussions are going well.

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Auxolytic's Nutrient-Based Shut-Off Switch Boosts Cell Therapy Safety Without Transgenes - BioSpace

Stem Cell Marketing: Hundreds OF Businesses Pitch Unproven Stem Cell Products To Treat Different Conditions – Technology Times Pakistan

Nature abhors a vacuum, and in the absence of evidence-based therapies to treat or prevent COVID-19, stem cell clinics with a menu of unsubstantiated direct-to-consumer therapies are more than willing to fill the gap.

Ambitious claims are not out of place in the realm of stem cell marketing, in which hundreds of U.S. businesses aggressively pitch unproven stem cell products to treat a variety of conditions, ranging from autism to Parkinsons disease to macular degeneration. It is a gray area of regenerative medicine that rheumatologists and orthopedic providers are typically familiar with, as the promise of stem cell injections to relieve joint pain or bypass invasive surgeries often resonates among patients with osteoarthritis.

Although much of the attention surrounding unproven regenerative medicine has focused on the significantly flashier claims, it is the relatively lowkey promises related to arthritis and other orthopedic conditions that remain the industrys main source of revenue.

A lot of the times when people talk about this market, they are interested in the really far out claims, such as that they treat ALS or spinal cord injuries or Alzheimers Disease, Leigh Turner, PhD, of the University of Minnesota Center for Bioethics and School of Public Health, told Healio Rheumatology. While that is important and the misrepresentations are extreme, individuals with arthritis and similar conditions are really the bread and butter of this marketplace. It is a lot more common.

In the United States, more than 27 million people are affected by OA, with an estimated 9.9 million adults exhibiting OA of the knee. It is to this population that regenerative medicine companies, through hundreds of sports medicine and pain management clinics across the country, direct their most enticing promises that all patients have to do is take cells from bone marrow or blood, process it, and inject them back into the body, and it will have a variety of anti-inflammatory therapeutic effects.

These are widespread advertising claims, Turner said. Its almost so widespread that their existence has kind of been normalized and routinized. When you think about regulatory activity with the FDA and the Federal Trade Commission, much of it has not been in the orthopedic or pain management space; the focus instead has been on the companies making the especially egregious claims.

In 2015, platelet injections for arthritis alone generated more than $93 million in revenue in the United States, according to a 2018 article in The Journal of Knee Surgery. Globally, the regenerative medicine market, including direct-to-consumer stem cell therapy, is expected to reach $5.6 billion by 2025, according to a 2019 report from the market research firm ReportLink.

Many businesses [involved in direct-to-consumer stem cell therapies] appear to be violating federal law and generating a lot of money by doing it but are not really drawing the attention of investigators, regulators or journalists, and remain such a fundamental part of the marketplace, Turner said. It deserves to have a light shown on it.

But now, many of the same regenerative medicine companies offering unproven treatments are adding a new promise to their advertising copy: the prevention and/or treatment of COVID-19.

In a statement published this year in JAMA, Peter Marks, MD, PhD, director of the FDAs Center for Biologics Evaluation and Research, warned physicians of this new development, and asked for their help in cracking down on these clinics.

Proven vs. Unproven Products When it comes to evidence-based, FDA-approved stem-cell therapies, the list of indications is short. According to Turner, these include stem-cell transplants for blood-related cancers and sickle cell maladies.

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Outside the stem-cell arena, the FDA in 2018 approved the regenerative medicine advanced therapy designation to MiMedx Groups AmnioFix Injectable an allogeneic micronized dehydrated human amnion/chorion membrane for use in the treatment of OA of the knee.

Beyond that, there are credible clinical trials taking place, where you have individuals with relevant training and legitimate credentials doing the kind of research genuinely intended to get at these questions for stem cell use in orthopedic diseases and injuries, Turner said. I would say that some of these preliminary studies are certainly encouraging enough to suggest the merits of further research.

In addition, according to Shane A. Shapiro, MD, medical directors for the Regenerative Medicine Therapeutics Program at the Mayo Clinic, the science of cell-based, or cell-derived, therapies for musculoskeletal disorders has made promising progress over the last decade.

We have given this subset of treatments the moniker orthobiologics to help separate these types of treatments from pure mesenchymal stem cell treatments, which are still in various phases of research, and quite far from ready to market or FDA approval, Shapiro told Healio Rheumatology. Additionally, we need to draw the distinction between orthobiologics and biologic agents used as treatments in inflammatory arthritic diseases, which modulate the immune system and have been approved for use in several arthritis and systemic inflammatory diseases.

Among these orthobiologic agents being translated into occasional practice include blood-based therapies, such as platelet rich plasma (PRP), as well as a similar product concentrated from bone marrow product called BMAC, and some fat-based therapies. All of these have begun testing in clinical trials, with some early use in clinical practice to treat arthritis, or to help augment surgical procedures, Shapiro said.

We have the most experience about 15 years with PRP for osteoarthritis and the literature, via several randomized controlled trials at this point, is quite supportive of PRP as a treatment for arthritic pain, he said. However, it is not a cure. PRP has been successful as a cure in a few, but certainly not all, chronic tendinopathies. The other cell-based therapies are further behind in our understanding of how they work and need more research. Still, we try very hard not to call any of these stem cell treatments, because it leads to greater patient confusion.

Moreover, most of this work has been focused in orthopedic and sports medicine, with very little known about their application in rheumatic diseases.

Given that OA and rheumatoid arthritis are different diseases with differing etiology, it would be hard to recommend any of these treatments in routine rheumatologic practice outside of a clinical trial, Shapiro said. Only a few stem-cell therapies have been approved worldwide and the few U.S. approvals treat hematologic malignancies. No other cell therapy product stem cell treatment has been adequately studied for use in rheumatology practice.

Baseless Claims In stark contrast to the work of the Mayo Clinic and other institutions focusing on evidence-based research, treatment claims commonly seen in the direct-to-consumer marketplace have gone well beyond the confines of credibly-designed clinical trials or generating meaningful data, according to Turner.

The businesses that I am concerned the most about are the ones that are about just hanging out a shingle on the internet and making advertising claims about having stem cell therapies for arthritis and a litany of other diseases and injuries, he said. Sure, there are credible studies taking place, but these are businesses that have leaped beyond that. Instead, these businesses are making claims to prospective clients that what they are advertising is safe, and that it will help them with pain relief or pain mitigation or even stronger claims.

A 2016 study co-authored by Turner and published in Cell Stem Cell found 351 U.S. businesses engaged in direct-to-consumer marketing of stem cell interventions offered at 570 American-based clinics.

These businesses, according to Turner, persuade people to spend thousands, or even tens of thousands, of dollars based on amorphous claims often relating to the mechanics of medicinal signaling cells that they are safe, robust and reliable.

interesting reading: Science Has A Racism Problem, Top Science Journal Say Systemic Racism Is Keeping Black People From Entering STEM

What I would say is that it is not a hypothesis that should be dismissed out of hand, but we also shouldnt walk around as though this is all established fact, Turner said. And to their credit, the FDA and the FTC have gradually over the past decade, especially in the last few years, stepped up their enforcement activities against these claims and the businesses that make them.

Typically, such enforcement actions from the FDA come in the form of a warning letter or, more recently, untitled letters used for violations that may not meet the threshold of regulatory significance for a warning letter requesting that a company correct their behavior.

According to Turner, recent warning and untitled letters from the FDA have resulted in some companies scaling back their claims regarding stem-cell therapies. However, as not all of these letters have been made public, it is difficult to know the extent of their use and which companies have received them.

Federal authorities have also sought and obtained permanent injunctions against companies and clinics for making unsubstantiated stem cell claims. The FDA and the U.S. Department of Justice in June 2019 successfully obtained a permanent injunction in a Florida federal court that stopped U.S. Stem Cell Clinic LLC and U.S. Stem Cell Inc., as well as their chief scientific officer, from manufacturing or distributing any and all stromal vascular fraction products, which are fat-tissue derived stem cell products.

However, Turner cautioned, such victories come with an important caveat.

The interesting thing there is that it was a specific stem cell product that the FDA wanted to get a permanent injunction on, Turner said. I think some people assume that if a company gets a warning letter, then its the end of the line, but my understanding with U.S. Stem Cell is that it resulted in a rebranding of the business. They still market stem cell treatments, but they have moved away from fat-derived products. So, its kind of a success for the FDA and the Department of Justice, but also maybe not such a dramatic success if the company can keep on marketing supposed stem cell therapies.

Cracking Down in the Age of COVID In his statement in JAMA, Marks wrote that some of the same clinics that have been advertising unproven regenerative therapies are now offering similarly unproven products for the treatment of complications of COVID-19, and are making claims that are simply not supported by compelling clinical data.

Turner, from his perspective, said the vast majority of these clinics have been largely unaffected by the emergence of COVID-19, with most boasting the same claims as before the pandemic.

However, there are those who have pivoted to take advantage of the spreading coronavirus, he added.

There is an interesting development in the marketplace, having to do with the companies that have seen the pandemic as a marketing opportunity, and are now advertising their stem cell products as treatments or preventive measures for COVID-19, Turner said. Now, I am not seeing that across hundreds of businesses, but it is an activity that is out there in the marketplace. Some of it is being picked up by the FDA and the FTC, and some of it continues to happen, but it hasnt appeared to have attracted the attention of regulators yet.

That may be changing, though. According to Turner, recent unproven claims regarding COVID-19 could give regulators a renewed focus and provide a clear target for corrective actions.

There is a kind of crispness, or a clarity to it, when companies make claims on their website, Facebook page or YouTube video that they have stem cells that are immune-system boosters, or that they will prevent individuals from getting COVID-19, he said. The FDA, the FTC and the Department of Justice have all indicated that they are going to be very aggressive in terms of pursuing businesses that are engaging in misrepresentations about treatment and preventive measures about COVID-19.

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Not in every case, because you can still find some of these businesses, but I would say that they have been as active as I have ever seen them, not just in terms of responding, but responding speed as well, to these misleading claims, he added.

In addition, Marks called on clinicians and patients alike to help federal authorities catch these clinics and companies, by coming forward and reporting any unproven claims or advertisements that they see.

However, that kind of work would require substantial communication between patient and physician regarding the large amount of medical and scientific material surrounding cell therapies circulating around the internet, in clinical practice and elsewhere.

And that, according to Shapiro, is exactly what the Mayo Clinics Regenerative Medicine Therapeutics Program, and its consulting service, have set out to do.

Harnessing Expertise Speaking as director of the program, Shapiro said the Mayo Clinics Regenerative Medicine Therapeutics Program, and its consult service, can help patients understand the available treatment options and provide answers about unmet clinical needs.

In most cases, that means having a conversation about the patients diagnosis, reviewing the standard of care treatment for their diagnosis, and then discussing available regenerative treatment options if there are any or alternatively, helping the patient find a clinical trial they might be a candidate for, Shapiro said.

Important, for patients with arthritis, is to clear up any misconceptions about what the cell therapies can and cannot do in current medical practice, he added. We also help patients understand the drug approval process and why stem cell therapies for arthritis might still need further research before they can be used to treat inflammatory conditions.

According to Shapiro, rheumatologists can help patients avoid unproven therapies by taking the time to explain all legitimate treatment options.

Rheumatologists are the experts in inflammatory arthritis conditions, he said. Patients look to their expertise for guidance. Rheumatologists should be providing patients with all legitimate treatment options, partnering with patients to decide on the best treatment option for their diagnosis, offering reassurance that patients are being presented with all scientifically-validated options, and engaging in a respectful but evidenced-based conversation about the current state of practice when it comes to regenerative therapeutics for inflammatory arthritis.

We can also provide them hope that legitimate scientific research is making great strides when it comes to regenerative treatments for such conditions, he added. There are legitimate scientific reports of bone-marrow stem-cell transplants for severe rheumatoid arthritis offering cures in small studies.

Turner agreed, adding that clinicians and rheumatologists should also help their patients who have been taken in by false claims to report such activities, as well as any adverse events they experience as a result. This, he said, would provide regulators with a better sense of the illicit direct-to-consumer marketplace, and how people are being taken advantage of.

I suspect there is substantial underreporting because the businesses themselves dont really report these kinds of problems, and when patients go to these clinics, they can feel embarrassed that they lost money or were harmed by it, so there can be some hesitancy in coming forward, Turner said.

I think part of it is the difficult work of trying to help patients understand where the current state of research is, and what kinds of treatment exist and which ones should we really be talking about as established, evidence-based therapies, he added. Those kinds of sober, cautious conversations help people sort out credible, evidence-base claims from what we might think of as scams or outright fraud.

the article is originally published at healio.

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Stem Cell Marketing: Hundreds OF Businesses Pitch Unproven Stem Cell Products To Treat Different Conditions - Technology Times Pakistan

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