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Hemophilia Treatment Market: Trends, Applications, Industry Competitive Analysis, Growth,Forecast: 2019 to 2029 – The News Brok

By Gene Therapy Clinics

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 Hemophilia Treatment market.

Trusted Business Insights presents an updated and Latest Study on Hemophilia 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 Hemophilia 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.

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Abstract, Snapshot, Market Analysis & Market Definition: Hemophilia Treatment Market Industry / Sector Trends

Hemophilia Treatment Market size was valued USD 10.5 billion in 2018 and is expected to witness 5.0% CAGR from 2019 to 2025.

U.S. hemophilia treatment market, by disease, 2018 & 2025 (USD Million)

Growing prevalence of hemophilia across the globe will be one of the major market augmenting factors over the forthcoming years. As per the recent statistics published by Centers for Disease Control and Prevention (CDC), hemophilia A affects around 1 in 5,000 live male births in the U.S. Majority of the people with hemophilia are diagnosed at a young age. According to the CDC data, average age of hemophilia diagnosis in the U.S. is 36 months for mild hemophilia, 8 months for moderate hemophilia and 1 month for severe hemophilia. Hence, rise in number of people suffering from hemophilia will enhance the hemophilia treatment market growth in the near future.

Rise in several government initiatives will augment the industry growth over the projection period. Organization including the World Hemophilia Organization along with governments of several countries carry out awareness and diagnosis programs. Government efforts to enhance public health and treat underprivileged people will result in boosting the hemophilia treatment market growth in the near future.

However, high cost of hemophilia medications will be one the major growth impeding factors. High investment, R&D, manufacturing and marketing of drugs by manufacturers is the reason for high cost of the products. High cost of products are unaffordable for the middle and lower income class families, thus resulting in restraining the business growth.

Market Segmentation, Outlook & Regional Insights: Hemophilia Treatment Market

Hemophilia Treatment Market, By Disease

Germany hemophilia treatment market share, by disease, 2018

Hemophilia A treatment market was valued at USD 6,743.7 million in the year 2018. Hemophilia A is a common blood disorder and the prevalence rate is high in developing and underdeveloped regions. As per the recent statistics, around 19,000 people in India suffer from hemophilia and only 15% of the patients are registered. Increase in number of people suffering from hemophilia A and rise in awareness among the people regarding the treatment and medications of hemophilia will result in rise in demand and adoption rate of hemophilia drugs, fueling the business growth.

Hemophilia B treatment market is estimated to grow at 4.0% over the estimation period. As per a recent research article, the prevalence of hemophilia B is quite rare as compared to hemophilia A and the incidence of hemophilia B is around 1 in 30,000 in the U.S. Recent technological advancements has led to growing medications for hemophilia B treatment, hence resulting in boosting the segmental growth.

Hemophilia Treatment Market, By Product

Recombinant factor concentrates market was valued at USD 6,626.9 million in the year 2018. Rise in production of recombinant factor concentrates has provided growth opportunity to treat hemophilia and overcome limited availability of plasma-derived concentrates. Also, increased safety of replacement therapy with the help of recombinant factor concentrates has dramatically enhanced the quality of patient life. Increase in preference of recombinant factor concentrates in treatment of hemophilia will ultimately lead to augmenting the business growth over the forecast period.

Extended half-life products market is estimated to witness a robust growth of 5.3% over the forecast period. Recent research and development has led to the production of new factor concentrates that are efficient and need less frequent injections. The reason for fewer injections are due to the increased half-life of the product. Hence, recent technological advancements and new product launches will lead to increase in demand and adoption rate of extended half-life products, thereby fueling the business growth.

Hemophilia Treatment Market, By Patient

Hemophilia treatment in adults was valued at USD 6,778.1 million in the year 2018. As per a recent research article, around 65% of the people suffering from hemophilia are adults. In some of the adults mild hemophilia is diagnosed in the later stages of life. As the age of patients increases, the need for hemophilia care also increases. There are various possibilities of developing moderate to severe hemophilia in adults. Hence, growing age results in rise in chances of developing hemophilia in adults, thereby boosting the demand for hemophilia treatment in the near future.

Hemophilia treatment in pediatrics is projected to grow at 5.2% over the estimation period. Hemophilia is inherited diseases that are passed to children from gene located on X-chromosome. In approximately one third of children, no family history of hemophilia is found, and the disease occurs due to gene mutation. Children with hemophilia gene bruise easily, bleed more with surgeries and have frequent nose bleedings. According to the National Center for Biotechnology Information, severe form of factor deficiencies including hepatitis A and hepatitis B were diagnosed in the neonates with 52% and 68% of cases respectively. Hence, increase in prevalence of hemophilia in children will augment the hemophilia treatment market growth in the near future.

Hemophilia Treatment Market, By Treatment

Prophylaxis treatment market was valued at USD 7,219.9 million in the year 2018. Long-term prophylaxis treatment is considered as standard of care to prevent chronic arthropathy and joint bleeding in patients with severe hemophilia. As per a recent research, it was observed that the prophylaxis treatment for hemophilia was quite effective and slowed the progression of joint damage. Majority of patients and healthcare providers opt for prophylaxis treatment owing to its efficiency and better end results. Hence, increase in preference of prophylaxis treatment will further augment the business growth in the near future.

On demand treatment market is estimated to grow at 5.2% over the forthcoming years. People suffering from mild to moderate hemophilia are treated with on demand treatment to prevent bleeding. Children with severe hemophilia initially receive prophylactic therapy to prevent bleeding; however, after reaching adulthood, they switch to on-demand treatment for several reasons, such as strict work schedules, inconvenience of visiting doctors or going to a clinic several times a week, along with high expenditure related to continuous prophylactic therapy. Hence, the aforementioned reasons will be responsible for the market growth of on demand treatment segment.

Hemophilia Treatment Market, By Therapy

Factor replacement therapy market was valued at USD 9,574.2 million in the year 2018. As per the Medical and Scientific Advisory Council (MASAC) of National Hemophilia Foundation (NHF), the use of recombinant factor concentrates in children is quite effective as the recombinant factor does not contain actual human blood and cannot transmit harmful viruses including hepatitis. Hence, the use of factor replacement therapy is quite common due to direct infusion of clotting factors, resulting in boosting the segmental growth.

Non-factor replacement therapy is projected to show rapid growth of 38.3% over the estimation period. One of the challenging problems in the treatment of hemophilia is the development of alloantibodies against the infused factor concentrates. Development of inhibitors render the factor replacement therapy ineffective, leading to high risk of morbidity and mortality in patients. Also, there is growing interest in non-factor replacement therapy agents that act by inhibiting anticoagulant pathways or enhancing coagulation. Hence, introduction of new therapeutic agents in hemophilia treatment will enhance the business growth.

Hemophilia Treatment Market, By Drug Class

Coagulation factors market was valued at USD 9,948.3 million in the year 2018. Hemophilia is considered as hereditary blood disease with coagulation time. The use of coagulation factors is quite common and is used mostly in the replacement therapy. Absence or improper functioning of several clotting factors in the blood such as factors VIII, XI, etc. result in causing hemophilia. Hence, use of several clotting factors to replace the absent blood clotting factors will lead to boosting the hemophilia treatment market growth.

Vasopressin drug class market is estimated to grow at 6.0% in the near future. Patients suffering from mild hemophilia use vasopressin drugs for joint and muscle bleeds, bleeding in mucous membranes of nose and mouth and pre and post-surgery. Vasopressin drugs comes in injectable and nasal spray form and is comparatively easy to use. Hence, the aforementioned factors will be responsible for the market growth of vasopressin drug class.

Hemophilia Treatment Market, By Route of Administration

Injectable route of administration for hemophilia treatment was valued at USD 9,629.3 million in the year 2018. Majority of the drugs and factor concentrate products are intravenously given to patients for better and quick results. Intravenous route of administration helps the medications to directly interact with the blood. Hence, majority of healthcare providers opt for injectable or intravenous route of drug administration to treat hemophilia for better and quick results, hence boosting the market growth.

Nasal spray or intra nasal route of administration will grow at 6.0% over the forecast period. Drugs including vasopressin are required to be taken by patients for joint and muscle bleeds, and bleeding in mucous membranes of nose and mouth. Several advantages offered by nasal spray such as ease of use and user convenience will result in growing demand, thereby fueling the business growth.

Hemophilia Treatment Market, By End-use

Hemophilia treatment centers as end-use was valued at USD 4,134.6 million in the year 2018. A substantial proportion of hemophilia treatment is carried out in such centers. During the forecast period, the hemophilia treatment center end-use market segment will continue to lead, owing to rising number of special care provided and peoples increased preference for these centers over other healthcare settings.

Clinics as end-use is estimated to grow at 5.3% in the near future. Clinics provide fast-track treatment and infusion of factor concentrates to patients. Also, immediate and short-time treatment provided during the daytime to people with busy and hectic work schedule will enhance business growth in the near future.

Hemophilia treatment Market, By Region

Europe hemophilia treatment market, by country, 2025 (USD Million)

U.S. dominated the North America hemophilia treatment market and was valued at USD 3,539.3 million in the year 2018. As per the Hemophilia Federation of America, hemophilia A affects around 1 in 5,000 people in the U.S. and approximately 400 new-borns are diagnosed with hemophilia every year. Also, presence of favorable reimbursement and insurance in the country will result in growing demand and adoption rate of hemophilia medications, expanding the hemophilia treatment market growth in the country.

India hemophilia treatment market is projected to grow at 9.2% over the estimation period. Various government initiatives has helped in building necessary infrastructure, empower healthcare providers and patients to self-administer treatment at home, hence helping patients achieve a better quality of life. Recent technological advancements, rise in awareness among people, new product launches and government initiatives will augment the business growth over the forecast period.

Key Players, Recent Developments & Sector Viewpoints: Hemophilia Treatment Market

Major industry players involved in the hemophilia treatment market include Swedish Orphan Biovitrum AB, Sanofi SA, Pfizer, Novo Nordisk, Genentech, CSL Behring, Biogen, among other market players. These players have implemented several strategic initiatives such as collaborations, merger and acquisitions, new product launches and partnerships that have enhanced their financial stability, helped them evolve as major industrial players and gain strong market position.

Hemophilia Treatment Industry Viewpoint

Hemophilia has been an extremely old and critical disease that the global population has been facing. People in ancient times have written and articulated about bleeding problems. Some of the bleeding problems identified have been different as compared to others. But little they knew about blood clots and the disease caused by their deficiency. Until before World War II, doctors learned about hemophilia A and its cause due to deficiency of factor VIII. Several other blood factors were recognized later on and Roman numeral names were given to avoid confusion. Hemophilia research has come a long way with scientists discovering human blood in different groups, hence helping blood transfusions to be more successful. In 1960s, Dr. Judith Pool discovered a procedure to freeze and thaw plasma to get a layer of factor-rich plasma called as cryoprecipitate. Use of cryoprecipitate was the best way of stopping hemophilia bleeding. Later on, the greatest breakthrough that came in hemophilia treatment was the development and introduction of factor concentrates. These clotting factors can be freeze-dried to a powder that can be easily stored and consumed. With the help of these factor concentrates, people suffering from hemophilia can be treated quickly with maximum efficiency. Also, factor concentrates have helped people treat their bleedings at home or at work, resulting in people to lead normal lives. Also, development and introduction of new medications and technologies such as non-factor replacement therapy, extended half-life products, gene therapy, etc. will augment the growth of hemophilia treatment market growth in the near future.

Key Insights Covered: Exhaustive Hemophilia Treatment Market 1. Market size (sales, revenue and growth rate) of Hemophilia Treatment industry. 2. Global major manufacturers operating situation (sales, revenue, growth rate and gross margin) of Hemophilia Treatment industry. 3. SWOT analysis, New Project Investment Feasibility Analysis, Upstream raw materials and manufacturing equipment & Industry chain analysis of Hemophilia Treatment industry. 4. Market size (sales, revenue) forecast by regions and countries from 2019 to 2025 of Hemophilia Treatment industry.

Research Methodology: Hemophilia Treatment Market

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Hemophilia Treatment Market: Trends, Applications, Industry Competitive Analysis, Growth,Forecast: 2019 to 2029 - The News Brok

Study Identifies New Set of Genes That May Explain Why People with Down Syndrome Have a Higher Risk of Leukemia – DocWire News

By Induced Pluripotent Stem Cells

A study which appeared in the journal Oncotarget sheds light on why people with Down syndrome are at higher risk of Leukemia. Researchers pinpointed a new set of genes overexpressed in endothelial cells of individuals with Down syndrome, thus creating an environment conducive for leukemia.

Down syndrome occurs in approximately in one in 700 babies, and individuals with the syndrome not only development physical impairments, they have a greatly augmented risk of developing leukemia. Specifically, people with Down syndrome have a 500-fold risk of developing acute megakaryoblastic leukemia (AMKL) and a 20-fold risk of being diagnosed with acute lymphoblastic leukemia (ALL).

In this study, researchers used skin samples from patients with Down syndrome to create induced pluripotent stem cells (iPSC). They subsequently differentiated the iPSC cells into that were then endothelial cells. The researchers observed that the endothelial cell genetic expression produced altered endothelial function throughout cell maturation. We found that Down syndrome, or Trisomy 21, has genome-wide implications that place these individuals at higher risk for leukemia, says co-lead author Mariana Perepitchka, BA, Research Associate at the Manne Research Institute at Lurie Childrens via a press release. We discovered an increased expression of leukemia-promoting genes and decreased expression of genes involved in reducing inflammation. These genes were not located on chromosome 21, which makes them potential therapeutic targets for leukemia even for people without Down syndrome.

Our discovery of leukemia-conducive gene expression in endothelial cells could open new avenues for cancer research, said co-lead author Yekaterina Galat, BS, Research Associate at the Manne Research Institute at Lurie Childrens.

Fortunately, advances in iPSC technology have provided us with an opportunity to study cell types, such as endothelial cells, that are not easily attainable from patients, stated senior author Vasil Galat, PhD, Director of Human iPS and Stem Cell Core at Manne Research Institute at Lurie Childrens and Research Assistant Professor of Pathology at Northwestern University Feinberg School of Medicine. If our results are confirmed, we may have new gene targets for developing novel leukemia treatments and prevention.

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Study Identifies New Set of Genes That May Explain Why People with Down Syndrome Have a Higher Risk of Leukemia - DocWire News

Patenting Stem Cell Inventions in India- What to Expect? – Lexology

By Induced Pluripotent Stem Cells

Stem cells offer hope as a promising treatment option for various diseases and are the future of medicine. Embryonic stem cells, have been at the heart of many debates globally, in view of the embryonic destruction or manipulation that their generation may require. Converging between research and law, patent law and policy grant yet throw their own challenges to obtaining exclusivity.

In India, in addition to satisfying the criteria of novelty and inventive step, inventions need to fall outside the realm of Section 3 of the Patents Act, to be patentable. Presenting an additional bar to patentability, Section 3 enlists inventions which are not patentable. Owing to this section it is oftentimes the case that the claim scope granted in India is quite different from that granted in other jurisdictions.

Public order and morality

Over the years, the Indian Patent Offices perspective on the issue of patentability of inventions involving embryonic stem cells, appears to have changed. This change in stance is apparent from the changes in the Manual of Patent Office Practice and Procedure. The 2005 draft of said guidelines treated the use of human or animal embryos for any purpose against public order and morality and prohibited the same from patentability. This restriction however, was removed from the subsequent draft of the guidelines and has not reappeared ever since.

Inspite of this change in the guidelines, the Patent Office till date raises the public order and morality objection under section 3(b) of the Patents Act, on stem cell related inventions (both methods and stem cell products). The concern most frequently expressed is the possibility of destruction of human embryos. The prosecution history of several cases shows that an objection on public order and morality has been raised even if the claims do not call out embryonic stem cells but the specification mentions the possibility of use of embryonic stem cells. The objection is frequently overcome by excluding any reference to embryonic stem cells from the claims and by disclaiming the use of embryonic stem cells in the operation of the invention.

However, the approach of treating stem cell research against public order and morality appears to be in contrast to public policy in India. The National Guidelines for Stem Cell Research (published by ICMR and DBT under the Ministry of Science and Technology) prescribe conditions subject to which research on stem cells should be conducted. The conditions include verification that the blastocysts used are spare embryos. The guidelines also permit establishment of new human embryonic stem cell lines from spare embryos subject to the approval of certain committees. Clearly, these government guidelines permit safe and responsible stem cell research, including research on embryonic stem cells.

Moreover, it is a well-known fact that not every invention involving embryonic stem cells would necessitate destruction of human embryos and a lot of research is based on embryonic stem cell lines. Therefore, the indiscriminate imposition of objections under Section 3(b) requires change.

Parts of Plants or Animals and Products of Nature

While claims relating to methods of isolation and propagation of stem cells are frequently granted, the Indian Patent Office appears to have never granted even a single application with claims directed to stem cells per se.

This brings us to another common objection frequently encountered in stem cell applications, namely, Section 3(j) which prohibits from patentability plants and animals in whole or any part thereof other than micro-organisms but including seeds, varieties and species and essentially biological processes for production or propagation of plants and animals. Another commonly encountered objection is of Section 3(c) which bars the patentability of any living thing or non-living substance occurring in nature.

There is no judicial precedent that could throw light on what exactly constitutes parts of plants and animals under Section 3(j). The Patent Office considers any cell or tissue derived from plants or animals as parts of plants or animals leading to refusal of cell claims under this ground. Claims related to compositions comprising stem cells are also frequently refused as the compositions are treated as indirectly claiming stem cells. There have been some exceptions though, such as patent number 333231, where a composition comprising stem cells was granted.

A moot issue here is whether cells are actually parts of animals/plants or whether they can be treated as microorganisms. While the Patents Act permits the patentability of microorganisms (that do not occur in nature), the term microorganism has not been defined in either the Act or the manuals that the Patent Office has issued so far. In fact, even the TRIPS agreement which mandates member states to grant patents in relation to microorganisms does not define the term. The European Patent Office recognizes all generally unicellular organisms with dimensions beneath the limits of vision which can be propagated and manipulated in a laboratory. (T 0356/93) as microorganisms.

Since the Patents Act does not limit the scope of the term microorganism and if one were to accept the literary or dictionary meaning of the term microorganism, it would appear that the Patents Act does not prohibit from the scope of patentability cells, which are not visible to the naked eye or which are so small that they require a microscope for viewing.

Moreover, stem cells like induced pluripotent stem cell and human parthenogenetic stem cells, which are somatic cells or oocytes that have been induced to develop the characteristics of unrestrained propagation and ability to develop into any cell type, are markedly distinct from the parent cell from which they are derived and are new cell types altogether. Such cells are indeed creations of man and cannot qualify as an animal part. They are also not living substances that occur in nature and being purely man made fall outside the prohibitory restraint of Section 3(c).

In the absence of judicial precedents and well defined guidelines, the law in India in relation to patentability of stem cell research is at a nascent stage. The Indian Patent Office has been following an unwritten code in the examination of these applications but the approach currently adopted is debatable. It is important to offer robust patent protection to encourage innovation in all fields. While there has been some change in the Patent Offices approach to patentability of stem cells and claims related to methods of producing, culturing and isolation of stem cells, culture media for stem cells, etc., are commonly granted, there is still a lot that can be patented but is currently not. Hopefully, India will see some judicial precedents in the future that will clarify the patentability issues that this field is struggling with.

This article was first published by Legal Era

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Patenting Stem Cell Inventions in India- What to Expect? - Lexology

Scientists May Have Discovered a Way to to Slow Aging by Direct Reprogramming of Human Cells – SciTechDaily

By Induced Pluripotent Stem Cells

Skin fibroblasts were successfully reprogrammed into the smooth muscle cells (red) and endothelial cells (white) which surround blood vessels. The cells nuclei are shown in blue. Credit: Bersini, Schulte et al. CC by 4.0

Salk study is the first to reveal ways cells from the human circulatory system change with age and age-related diseases.

Salk scientists have used skin cells called fibroblasts from young and old patients to successfully create blood vessels cells that retain their molecular markers of age. The teams approach, described in the journal eLife on September 8, 2020, revealed clues as to why blood vessels tend to become leaky and hardened with aging, and lets researchers identify new molecular targets to potentially slow aging in vascular cells.

The vasculature is extremely important for aging but its impact has been underestimated because it has been difficult to study how these cells age, says Martin Hetzer, the papers senior author and Salks vice president and chief science officer.

Research into aging vasculature has been hampered by the fact that collecting blood vessel cells from patients is invasive, but when blood vessel cells are created from special stem cells called induced pluripotent stem cells, age-related molecular changes are wiped clean. So, most knowledge about how blood vessel cells age comes from observations of how the blood vessels themselves change over time: veins and arteries become less elastic, thickening and stiffening. These changes can contribute to blood pressure increases and a heightened risk of heart disease with age.

From left: Martin Hetzer and Simone Bersini. Credit: Salk Institute

In 2015, Hetzer was part of the team led by Salk President Rusty Gage to show that fibroblasts could be directly reprogrammed into neurons, skipping the induced pluripotent stem cell stage that erased the cells aging signatures. The resulting brain cells retained their markers of age, letting researchers study how neurons change with age.

In the new work, Hetzer and his colleagues applied the same direct-conversion approach to create two types of vasculature cells: vascular endothelial cells, which make up the inner lining of blood vessels, and the smooth muscle cells that surround these endothelial cells.

We are among the first to use this technique to study the aging of the vascular system, says Roberta Schulte, the Hetzer lab coordinator and co-first author of the paper. The idea of developing both of these cell types from fibroblasts was out there, but we tweaked the techniques to suit our needs.

The researchers used skin cells collected from three young donors, aged 19 to 30 years old, three older donors, 62 to 87 years old, and 8 patients with Hutchinson-Gilford progeria syndrome (HGPS), a disorder of accelerated, premature aging often used to study aging.

The resulting induced vascular endothelial cells (iVECs) and induced smooth muscle cells (iSMCs) showed clear signatures of age. 21 genes were expressed at different levels in the iSMCs from old and young people, including genes related to the calcification of blood vessels. 9 genes were expressed differently according to age in the iVECs, including genes related to inflammation. In patients with HGPS, some genes reflected the same expression patterns usually seen in older people, while other patterns were unique. In particular, levels of BMP-4 protein, which is known to play a role in the calcification of blood vessel, were slightly higher in aged cells compared to younger cells, but more significantly higher in smooth muscle cells from progeria patients. This suggests that the protein is particularly important in accelerated aging.

The results not only hinted at how and why blood vessels change with age, but confirmed that the direct-conversion method of creating vascular endothelial and smooth muscle cells from patient fibroblasts allowed the cells to retain any age-related changes.

One of the biggest theoretical implications of this study is that we now know we can longitudinally study a single patient during aging or during the course of a treatment and study how their vasculature is changing and how we might be able to target that, says Simone Bersini, a Salk postdoctoral fellow and co-first author of the paper.

To test the utility of the new observations, the researchers tested whether blocking BMP4 which had been present at higher levels in smooth muscle cells developed from people with HGPS could help treat aging blood vessels. In smooth muscle cells from donors with vascular disease, antibodies blocking BMP4 lowered levels of vascular leakiness one of the changes that occurs in vessels with aging.

The findings point toward new therapeutic targets for treating both progeria and the normal age-related changes that can occur in the human vascular system. They also illustrate that the direct conversion of fibroblasts to other mature cell types previously successful in neurons and, now, in vascular cells is likely useful for studying a wide range of aging processes in the body.

By repeating what was done with neurons, weve demonstrated that this direct reprogramming is a powerful tool that can likely be applied to many cell types to study aging mechanisms in all sorts of other human tissues, says Hetzer, holder of the Jesse and Caryl Philips Foundation Chair.

The team is planning future studies to probe the exact molecular mechanisms by which some of the genes they found to change with age control the changes seen in the vasculature.

Reference: Direct reprogramming of human smooth muscle and vascular endothelial cells reveals defects associated with aging and Hutchinson-Gilford progeria syndrome by Simone Bersini, Roberta Schulte, Ling Huang, Hannah Tsai and Martin W Hetzer, 8 September 2020, eLife. DOI: 10.7554/eLife.54383

Other researchers on the study were Ling Huang and Hannah Tsai of Salk. The work was supported by grants from the National Institutes of Health, the NOMIS Foundation and an AHA-Allen Initiative in Brain Health and Cognitive Impairment award made jointly through the American Heart Association and the Paul G. Allen Frontiers Group. Simone Bersini was supported by the Paul F. Glenn Center for Biology of Aging Research at the Salk Institute.

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Scientists May Have Discovered a Way to to Slow Aging by Direct Reprogramming of Human Cells - SciTechDaily

Induced Pluripotent Stem Cells Market Global Growth Analysis and Forecast to 2024 | Top Players (BlueRock Therapeutics, Corning Life Sciences, EMD…

By Induced Pluripotent Stem Cells

The Induced Pluripotent Stem Cells market analysis is provided for the international markets including development trends, competitive landscape analysis, and key regions development status. The report provides key statistics on the market status of the Induced Pluripotent Stem Cells manufacturers and is a valuable source of guidance and direction for companies and individuals interested in the industry.

Complete report on Induced Pluripotent Stem Cells market spread across 148 pages, profiling companies and supported with tables and figures is now available @ https://www.insidemarketreports.com/sample-request/6/463100/Induced-Pluripotent-Stem-Cells

Our industry professionals are working reluctantly to understand, assemble and timely deliver assessment on impact of COVID-19 disaster on many corporations and their clients to help them in taking excellent business decisions. We acknowledge everyone who is doing their part in this financial and healthcare crisis.

The global Induced Pluripotent Stem Cells market 2019 research is a professional and in-depth study on the current state of the industry and provides a basic overview of the industry including definitions, classifications, applications and industry chain structure. The Induced Pluripotent Stem Cells market analysis is provided for the international markets including development trends, competitive landscape analysis, and key regions development status. Development policies and plans are discussed as well as manufacturing processes and cost structures are also analyzed. This report also states import/export consumption, supply and demand Figures, cost, price, revenue and gross margins.

This report presents the worldwide Induced Pluripotent Stem Cells market size (value, production and consumption), splits the breakdown (data status 2015-2019 and forecast to 2024), by manufacturers, region, type and application. This study also analyzes the market status, market share, growth rate, future trends, market drivers, opportunities and challenges, risks and entry barriers, sales channels, distributors and Porters Five Forces Analysis.

Companies profiled and studied for this Induced Pluripotent Stem Cells market report include BlueRock Therapeutics, Corning Life Sciences, EMD Millipore, Lonza Group, Promega, Thermo Fisher Scientific and others.

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Induced Pluripotent Stem Cells Market Global Growth Analysis and Forecast to 2024 | Top Players (BlueRock Therapeutics, Corning Life Sciences, EMD...

Study: The Speed Neurons Fire Impacts Their Ability to Synchronize – Lab Manager Magazine

By Induced Pluripotent Stem Cells

Research conducted by the Computational Neuroscience Unit at the Okinawa Institute of Science and Technology Graduate University (OIST) has shown for the first time that a computer model can replicate and explain a unique property displayed by a crucial brain cell. Their findings, published Sept. 8 ineLife, shed light on how groups of neurons can self-organize by synchronizing when they fire fast.

The model focuses on Purkinje neurons, which are found within the cerebellum. This dense region of the hindbrain receives inputs from the body and other areas of the brain in order to fine-tune the accuracy and timing of movement, among other tasks.

"Purkinje cells are an attractive target for computational modeling as there has always been a lot of experimental data to draw from," said professor Erik De Schutter, who leads the Computation Neuroscience Unit. "But a few years ago, experimental research into these neurons uncovered a strange behavior that couldn't be replicated in any existing models."

These studies showed that the firing rate of a Purkinje neuron affected how it reacted to signals fired from other neighboring neurons.

Cell membranes have a voltage across them due to the uneven distribution of charged particles, called ions, between the inside and outside of the cell. Neurons can shuttle ions across their membrane through channels and pumps, which changes the voltage of the membrane. Fast firing Purkinje neurons have a higher membrane voltage than slow firing neurons.

Image modified from "How neurons communicate: Figure 2," by OpenStax College, Biology (CC BY 4.0)

The rate at which a neuron fires electrical signals is one of the most crucial means of transmitting information to other neurons. Spikes, or action potentials, follow an "all or nothing" principleeither they occur, or they don'tbut the size of the electrical signal never changes, only the frequency. The stronger the input to a neuron, the quicker that neuron fires.

But neurons don't fire in an independent manner. "Neurons are connected and entangled with many other neurons that are also transmitting electrical signals. These spikes can perturb neighboring neurons through synaptic connections and alter their firing pattern," explained De Schutter.

Interestingly, when a Purkinje cell fires slowly, spikes from connected cells have little effect on the neuron's spiking. But, when the firing rate is high, the impact of input spikes grows and makes the Purkinje cell fire earlier.

"The existing models could not replicate this behavior and therefore could not explain why this happened. Although the models were good at mimicking spikes, they lacked data about how the neurons acted in the intervals between spikes," De Schutter said. "It was clear that a newer model including more data was needed."

Fortunately, De Schutter's unit had just finished developing an updated model, an immense task primarily undertaken by now former postdoctoral researcher, Dr. Yunliang Zang.

Once completed, the team found that for the first time, the new model was able to replicate the unique firing-rate dependent behavior.

In the model, they saw that in the interval between spikes, the Purkinje neuron's membrane voltage in slowly firing neurons was much lower than the rapidly firing ones.

"In order to trigger a new spike, the membrane voltage has to be high enough to reach a threshold. When the neurons fire at a high rate, their higher membrane voltage makes it easier for perturbing inputs, which slightly increase the membrane voltage, to cross this threshold and cause a new spike," explained De Schutter.

The researchers found that these differences in the membrane voltage between fast and slow firing neurons were because of the specific types of potassium ion channels in Purkinje neurons.

"The previous models were developed with only the generic types of potassium channels that we knew about. But the new model is much more detailed and complex, including data about many Purkinje cell-specific types of potassium channels. So that's why this unique behavior could finally be replicated and understood," said De Schutter.

When a group of Purkinje neurons fire rapidly, loose synchronization occurs. This can be seen by the spikes occurring in groups at regular intervals (highlighted in yellow). When Purkinje neurons fire slowly, this synchronization does not occur.

OIST

The researchers then decided to use their model to explore the effects of this behavior on a larger-scale, across a network of Purkinje neurons. They found that at high firing rates, the neurons started to loosely synchronize and fire together at the same time. Then when the firing rate slowed down, this coordination was quickly lost.

Using a simpler, mathematical model, Dr. Sungho Hong, a group leader in the unit, then confirmed this link was due to the difference in how fast and slow firing Purkinje neurons responded to spikes from connected neurons.

"This makes intuitive sense," said De Schutter. He explained that for neurons to be able to sync up, they need to be able to adapt their firing rate in response to inputs to the cerebellum. "So this syncing with other spikes only occurs when Purkinje neurons are firing rapidly," he added.

The role of synchrony is still controversial in neuroscience, with its exact function remaining poorly understood. But many researchers believe that synchronization of neural activity plays a role in cognitive processes, allowing communication between distant regions of the brain. For Purkinje neurons, they allow strong and timely signals to be sent out, which experimental studies have suggested could be important for initiating movement.

"This is the first time that research has explored whether the rate at which neurons fire affects their ability to synchronize and explains how these assemblies of synchronized neurons quickly appear and disappear," said De Schutter. "We may find that other circuits in the brain also rely on this rate-dependent mechanism."

The team now plans to continue using the model to probe deeper into how these brain cells function, both individually and as a network. And, as technology develops and computing power strengthens, De Schutter has an ultimate life ambition.

"My goal is to build the most complex and realistic model of a neuron possible," said De Schutter. "OIST has the resources and computing power to do that, to carry out really fun science that pushes the boundary of what's possible. Only by delving into deeper and deeper detail in neurons, can we really start to better understand what's going on."

- This press release was originally published on theOIST website

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Study: The Speed Neurons Fire Impacts Their Ability to Synchronize - Lab Manager Magazine

Global Autologous Cell Therapy Market Is Expected to Reach USD 42.68 Billion by 2027 : Fior Markets – GlobeNewswire

By Stem Cell Treatment

September 09, 2020 16:00 ET | Source: Fior Markets

Newark, NJ, Sept. 09, 2020 (GLOBE NEWSWIRE) -- As per the report published by Fior Markets, theglobal autologous cell therapy market is expected to grow from USD 9.29 billion in 2019 and to reach USD 42.68 billion by 2027, growing at a CAGR of 21.00% during the forecast period 2020-2027.

The primary determinants attributing to the growth of the autologous cell therapy business are the increasing incidence of chronic diseases such as cancer, a blood disorder, autoimmune diseases, and others. An increase in the population undergoing severe conditions is also generating a requirement for market growth. Autologous cell therapy is increasing due to the moderate risk of complexities connected with autologous treatment. Other factors expected to propel the market are the affordability, enhanced survival rate of patients, no chance of graft-versus-host diseases, and no obligation to identify an HLA-matched donor.

Autologous cell therapy (ACT) is an innovative therapeutic intervention that employs an individuals cells, that are cultured and extended outside the body, and reintroduced into the donor. The advantages of the autologous cell therapy approach include minimizing risks from systemic immunological reactions and bio-incompatibility. Also, disease transmission related with cells or grafts that are not cultivated from the individual gives added benefits. So far, this kind of treatment has been utilized successfully to help counteract chronic inflammation, bioengineer skin substitutes, wound healing, treat burns and pressure ulcers, and enhance postoperative healing. The therapy is recognized as a safer and effective technology compared with the existing transplant technologies, such as xenotransplants and allogeneic. Autologous transplants promote in mitigation of risks connected with disease transmission, bio-incompatibility, and immunological reactions. The increasing frequencies of fatality and morbidity of cancer and ample funding from the government, as well as many private facilities in order to restrict the growth of cancer, has currently made the procedure for cancer the topmost priority. The growth of widespread diseases and a large number of stem cell helpers are the critical factors propelling the demand of the market. Autologous stem cell therapy technology (a form of regenerative cell therapy) changes treatments by launching several new therapies. Its range is vast and promising for the future despite challenges. It is a unique therapeutic platform improving in the field of regenerative medication. It is acknowledged as an effective and safer technology. And it also serves as an internal repairing system. Hence the number of therapies based on stem cells is comparatively higher.

The cost of the processing is not affordable; however, this mode of treatment will undoubtedly experience extensive market growth by the intervention of government organizations. There are several investments and endowments done to the research facilities by the private and public sectors promoting the growth of the research facilities. Moreover, the increasing incidence of complicated diseases and the advancement of technology will drive the segment's demand.

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Key players operating in the global autologous cell therapy market include Bayer AG, Daiichi Sankyo Co. Ltd., Takeda Pharmaceutical Co. Ltd., Teva Pharmaceutical Industries Ltd., Holostem Terapie Avanzate Srl, Vericel Corp., Osiris Therapeutics Inc., Brainstorm Cell Therapeutics Inc., Sumitomo Chemical Co. Ltd., and FUJIFILM Holdings Corp. To gain a significant market share in the global autologous cell therapy market, the key players are now focusing on adopting strategies such as product innovations, mergers & acquisitions, recent developments, joint ventures, collaborations, and partnerships.

Bone marrow segment dominated the market and held the largest market share of 23.38% in the year 2019 The source segment includes bone marrow, epidermis, mesenchymal stem cells, hematopoietic stem cells and chondrocytes. Bone marrow segment held the largest market share of 23.38% in the year 2019. Bone marrow is the hub for most stem cells, and extensive research and development activities for bone marrow-derived stem cells promote market growth.

Cancer segment dominated the market and valued at USD 1.82 billion in the year 2019 The application segment includes cancer, neurodegenerative diseases, wound healing, orthopedic, cardiovascular diseases and autoimmune diseases. Cancer segment dominated the market and valued at USD 1.82 billion in the year 2019. The primary source of stem cells is embryonic stem cells for therapeutic targets due to their large totipotency and indefinite lifespan. These advantages are expected to propel the growth of the market in fatal therapeutic areas.

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Regional Segment Analysis of The Autologous Cell Therapy Market

On the basis of geography, the global autologous cell therapy market is classified into North America, Europe, South America, Asia Pacific, and Middle East and Africa. North America is expected to show the largest share in the autologous cell therapy product over the forecast period. The United States is a significant contributor to crucial market merchants and research businesses established in the country. The region has numerous pipeline designs that are promoting the market requirement. In June 2019, Celgene and Evotec SE extended their collaboration to incorporate a new iPSC, which now targets toward the betterment of disease-modifying procedures for patients suffering from neurodegenerative diseases.

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About the report: The global autologous cell therapy market is analyzed on the basis of value (USD Billion). All the segments have been analyzed on global, regional and country basis. The study includes an analysis of more than 30 countries for each segment. The report offers in-depth analysis of driving factors, opportunities, restraints, and challenges for gaining the key insight of the market. The study includes porter's five forces model, attractiveness analysis, raw material analysis, and competitor position grid analysis.

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Global Autologous Cell Therapy Market Is Expected to Reach USD 42.68 Billion by 2027 : Fior Markets - GlobeNewswire

AgeX Therapeutics and Lineage Cell Therapeutics Announce Expansion of Agreement Related to ESI Clinical-grade Pluripotent Stem Cell Lines for…

By Stem Cell Treatment

Sept. 9, 2020 12:00 UTC

ALAMEDA, Calif. & CARLSBAD, Calif.--(BUSINESS WIRE)-- AgeX Therapeutics, Inc.(AgeX: NYSE American: AGE), a company focused on developing and commercializing innovative therapeutics for human aging, and Lineage Cell Therapeutics, Inc.. (Lineage: NYSE American and TASE: LCTX), a clinical-stage biotechnology company developing novel cell therapies for unmet medical needs, and ES Cell International Pte Ltd. (ESI), a subsidiary of Lineage, today announced the broadening of their collaborative relationship with regard to ESI stem cell lines. ESI cell lines are current Good Manufacturing Practice (cGMP)-compatible, registered with the National Institutes of Health (NIH), and widely studied as a potential source for the industrial-scale manufacture of any cell type in the human body. Neither party made or received any cash payments in connection with this arrangement.

Both Lineage and AgeX are pioneering important aspects of regenerative medicine. Working together, we have amended our agreement regarding ESI cell lines derived under cGMP to be optimal for the business needs of each company, stated Brian M. Culley, Lineages CEO. In particular, Lineage has acquired exclusivity for the use of ESI cell lines in spinal cord injury and certain oncology indications. On the other hand, AgeX has gained greater flexibility and independence to support its efforts toward licensing certain technologies and cell lines to third parties. With this step complete, we next intend to explore additional opportunities to collaborate with AgeX on promising tissue regenerating projects.

The ESI cell lines are recognized for being the first clinical-grade human pluripotent stem cell lines created under cGMP as described in the publication Cell Stem Cell (2007;1:490-4). It may become possible to generate potentially limitless quantities of all the cell types of the human body from these master cell banks with a wide array of potential therapeutic applications. These cell lines are listed on the NIH Stem Cell Registry and are among the best characterized and documented stem cell lines available globally. Importantly, ESI cells are among only a few pluripotent stem cell lines from which a derived cell therapy product candidate has been granted FDA investigational new drug (IND) clearance to commence human studies.

Key to the creation of shareholder value is the placement of these important assets in the hands of collaborators to advance the development of a vast number of regenerative therapies, said Michael West, Ph.D., AgeXs CEO. Our collaborative relationship with Lineage led to this streamlined process that may facilitate the commercialization of these applications to the benefit of shareholders of each company. Since the beginning of the year, AgeX has entered into new research and commercial arrangements utilizing an array of its technology platforms, such as UniverCyteTM for the engineering of universally transplantable cells, PureStem for the manufacture and derivation of cells, and an ESI cell line as source material for deriving cellular therapeutics.

About AgeX Therapeutics, Inc

AgeX Therapeutics, Inc. (NYSE American: AGE) is focused on developing and commercializing innovative therapeutics for human aging. Its PureStem and UniverCyte manufacturing and immunotolerance technologies are designed to work together to generate highly defined, universal, allogeneic, off-the-shelf pluripotent stem cell-derived young cells of any type for application in a variety of diseases with a high unmet medical need. AgeX has two preclinical cell therapy programs: AGEX-VASC1 (vascular progenitor cells) for tissue ischemia and AGEX-BAT1 (brown fat cells) for Type II diabetes. AgeXs revolutionary longevity platform induced Tissue Regeneration (iTR) aims to unlock cellular immortality and regenerative capacity to reverse age-related changes within tissues. AGEX-iTR1547 is an iTR-based formulation in preclinical development. HyStem is AgeXs delivery technology to stably engraft PureStem cell therapies in the body. AgeXs core product pipeline is intended to extend human healthspan. AgeX is seeking opportunities to establish licensing and collaboration arrangements around its broad IP estate and proprietary technology platforms and therapy product candidates. For more information, please visit http://www.agexinc.com or connect with the company on Twitter, LinkedIn, Facebook, and YouTube.

About Lineage Cell Therapeutics, Inc.

Lineage Cell Therapeutics is a clinical-stage biotechnology company developing novel cell therapies for unmet medical needs. Lineages programs are based on its robust proprietary cell-based therapy platform and associated in-house development and manufacturing capabilities. With this platform Lineage develops and manufactures specialized, terminally differentiated human cells from its pluripotent and progenitor cell starting materials. These differentiated cells are developed to either replace or support cells that are dysfunctional or absent due to degenerative disease or traumatic injury or administered as a means of helping the body mount an effective immune response to cancer. Lineages clinical programs are in markets with billion dollar opportunities and include three allogeneic (off-the-shelf) product candidates: (i) OpRegen, a retinal pigment epithelium transplant therapy in Phase 1/2a development for the treatment of dry age-related macular degeneration, a leading cause of blindness in the developed world; (ii) OPC1, an oligodendrocyte progenitor cell therapy in Phase 1/2a development for the treatment of acute spinal cord injuries; and (iii) VAC, an allogeneic dendritic cell therapy platform for immuno-oncology and infectious disease, currently in clinical development for the treatment of non-small cell lung cancer and in preclinical development for additional cancers and as a vaccine against infectious diseases, including SARS-CoV-2, the virus which causes COVID-19. For more information, please visit http://www.lineagecell.com or follow the Company on Twitter @LineageCell.

About ESI

ES Cell International Pte Ltd (ESI). Established in 2000, ESI, a wholly owned subsidiary of Lineage Cell Therapeutics, Inc., developed ESI hESC lines in compliance with the principles of current Good Manufacturing Practices and has made them available to various biopharmaceutical companies, universities and other research institutions, including AgeX. These ESI cell lines are extensively characterized and most of the lines have documented and publicly available genomic sequences.

Forward-Looking Statements for AgeX

Certain statements contained in this release are forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. Any statements that are not historical fact including, but not limited to statements that contain words such as will, believes, plans, anticipates, expects, estimates should also be considered forward-looking statements. Forward-looking statements involve risks and uncertainties. Without limitation, such risks include those associated with the use of human pluripotent stem cell lines in the research, development, and use of therapies for the treatment of human diseases, disorders, and injuries, and risks associated with commercializing the pluripotent stem cell lines. Actual results may differ materially from the results anticipated in these forward-looking statements and as such should be evaluated together with the many uncertainties that affect the business of AgeX Therapeutics, Inc. and its respective subsidiaries, particularly those mentioned in the cautionary statements found in more detail in the Risk Factors section of its most recent Annual Reports on Form 10-K and Quarterly Reports on Form 10-Q filed with the Securities and Exchange Commissions (copies of which may be obtained at http://www.sec.gov). Subsequent events and developments may cause these forward-looking statements to change. Undue reliance should not be placed on forward-looking statements, which speak only as of the date on which they were made. AgeX specifically disclaims any obligation or intention to update or revise these forward-looking statements as a result of changed events or circumstances that occur after the date of this release, except as required by applicable law.

Forward-Looking Statements for Lineage

Lineage cautions you that all statements, other than statements of historical facts, contained in this press release, are forward-looking statements. Forward-looking statements, in some cases, can be identified by terms such as believe, may, will, estimate, continue, anticipate, design, intend, expect, could, plan, potential, predict, seek, should, would, contemplate, project, target, tend to, or the negative version of these words and similar expressions. Such statements include, but are not limited to, statements relating to the potential commercialization of ESI cell lines. Forward-looking statements involve known and unknown risks, uncertainties and other factors that may cause Lineages actual results, performance or achievements to be materially different from future results, performance or achievements expressed or implied by the forward-looking statements in this press release, including risks and uncertainties inherent in Lineages business and other risks in Lineages filings with the Securities and Exchange Commission (the SEC). Lineages forward-looking statements are based upon its current expectations and involve assumptions that may never materialize or may prove to be incorrect. All forward-looking statements are expressly qualified in their entirety by these cautionary statements. Further information regarding these and other risks is included under the heading Risk Factors in Lineages periodic reports with the SEC, including Lineages Annual Report on Form 10-K filed with the SEC on March 12, 2020 and its other reports, which are available from the SECs website. You are cautioned not to place undue reliance on forward-looking statements, which speak only as of the date on which they were made. Lineage undertakes no obligation to update such statements to reflect events that occur or circumstances that exist after the date on which they were made, except as required by law.

View source version on businesswire.com: https://www.businesswire.com/news/home/20200909005398/en/

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AgeX Therapeutics and Lineage Cell Therapeutics Announce Expansion of Agreement Related to ESI Clinical-grade Pluripotent Stem Cell Lines for...

Tweet Chat Recap: Evaluating Treatment Approaches for Relapsed/Refractory DLBCL – Targeted Oncology

By Stem Cell Treatment

Targeted Oncology was joined by Kami J. Maddocks, MD, associate professor of clinical internal medicine, Division of Hematology, The Ohio State University Comprehensive Cancer CenterJames, for the discussion of a 76-year-old man with relapsed/refractory diffuse large B-cell lymphoma (DLBCL) in a recent tweet chat. In this case scenario, the patient presented with stage IV high-risk disease and received R-CHOP (Rituximab [Rituxan], cyclophosphamide, doxorubicin, vincristine, prednisone), and radiotherapy.

Although the treatment appeared well-tolerated, the patient presented with similar symptoms as at diagnosis after completing 6 cycles with complete response to the therapy. According to the work-up, the patient is ineligible for transplant.

The patient was ineligible for stem cell transplantation (SCT), which Maddocks speculates may be due to the patients age, although other considerations could include comorbidities or intolerance to R-CHOP. Eligibility is the first thing she considers for her patients as it is currently the standard of care and the only curative approach for patients to receive salvage chemotherapy followed by consolidation with autologous SCT.

Maddocks told Targeted Oncology, In some patient cases, [the reason for ineligibility] is age even though there's no specific age cutoff, but we know that it's harder on the marrow as patients get older to collect stem cells and get that aggressive salvage chemotherapy. Patient comorbidities [can also impact eligibility], so heart conditions, lung conditions, renal insufficiency can be a problem. Performance status and then lastly, just if the patient had trouble getting to their initial chemotherapy with R-CHOP or had a lot of complications, then it's probably going to be harder for them to tolerate even more aggressive or intensive therapy.

In a twitter poll ahead of the chat, Targeted Oncology asked what the next best line of therapy for this patient might be, with 4 potential different treatment options. The option that drew the most attention, however, was the recently approved regimen of tafasitamab (Monjuvi) and lenalidomide (Revlimid).

Maddocks tweeted, All these options are potential therapeutic choices for this patient, but the combination of tafasitamab/lenalidomide is the only option approved in this setting. The treatment has a promising ORR [overall response rate], and CR [complete response], and the remissions for patients who respond are durable!

During the tweet chat, Maddocks reviewed each of the different treatment options in the poll, and why she selected this combination regimen as the next best line of therapy for this particular patient. Following the chat, she spoke with Targeted Oncology to share further insights on each of these therapeutic approaches and the importance of the FDAs approval of tafasitamab plus lenalidomide in this setting.

The combination of tafasitamab plus lenalidomide held the majority vote, which Maddocks agreed would be the next best line of therapy for this patient.

For patients who are not candidates or considered eligible for a salvage chemotherapy followed by autologous SCT, the tafasitamab/lenalidomide combination was recently approved in the setting of first relapse, and it's the only approved therapy in this setting, Maddocks said. Historically, we would give some sort of palliative chemotherapy approach if patients were candidates and interested in pursuing therapy, or consideration of clinical trial, but this is the only therapy approved in this setting.

The approval of tafasitamab in combination with lenalidomide includes an indication for patients who are not eligible for autologous SCT, as describes the patient in our case. This regimen was approved on the basis of the phase 2 L-MIND (NCT02399085) clinical trial, which explored this use of this regimen in 81 patients with relapsed/refractory DLBCL. Two-year follow-up demonstrated an ORR of 58.5%, which included CRs in 41.3% of patients and partial responses (PRs) in 17.5% of patients. In addition, 15.0% achieved stable disease, and the median duration of response was 34.6 months (95% CI, 26.1-34.6).1

I think this patient case is the perfect example of where this can fit into the treatment landscape, Maddocks explained. For patients who first relapse from the standard R-CHOP therapy, the toxicities were generally manageable, and with the response rate, this is a great option for patients at first relapse who are not going to be candidates for a transplant. I think maybe patients who go on to get palliative chemotherapy or maybe patients who get treatment with plans to go to transplant but just don't tolerate it and dont look like they're going to [undergo] aggressive therapy, this may be an option for those patients too, understanding that there is some role for CAR T in a set of those patients.

This study, which was presented during the 25th Congress of the European Hematology Association (EHA), demonstrated that the majority of toxicities were hematologic, and most were reversible. The most common grade 3 hematologic treatment-emergent adverse events (TEAEs) were neutropenia in 49.4% of patients, thrombocytopenia in 17.3%, and febrile neutropenia in 13.2%.1

These were able to be managed by holding the dose growth factor, and there was a population of patients who had to be dose-reduced on the lenalidomide. The starting dose was 25 mg, so the majority were able to maintain 20 mg if they were dose-reduced, although a few had to be reduced more than once, Maddocks said. The most common grade 3/4 or serious AEs were infection, probably not surprisingly, and overall, that's probably similar to what you see with other options in this setting. There was a small number of infusion reactions, but these were all grade 1 in the trial and were easily managed.

Non-hematologic TEAEs of grade 3 included pneumonia in 8.6% of patients and hypokalemia in 6.2%. Serious AEs reported included pneumonia in 8.6%, febrile neutropenia in 6.2%, and pulmonary embolism in 3.7%, as well as bronchitis, lower respiratory tract infection, atrial fibrillation, and congestive cardiac failure in 2.5% each.1

Given the safety profile of this combination of tafasitamab plus lenalidomide, this regimen is particularly suitable for a large proportion of patients with DLBCL, Gilles Salles, MD, PhD, lead author of L-MIND, toldTargeted Oncology. We do know that the median age of occurrence of DLBCL is in the late 60s, and there are many, many patients that are over 70 and that are not usually transplant eligible. Clearly this is a great opportunity for patients to receive this non-cytotoxic regimen.

Although this regimen is an exciting opportunity for patients with DLBCL and relapsed/refractory disease, 1 challenge that needs to be addressed is the potential use of tafasitamab plus lenalidomide in sequence with CAR T-cell therapy. There is very little experience, if any, of patients receiving the combination regimen after receiving CAR T-cell therapy. The combination and CAR T cells both target the same antigen, CD19, which can be problematic. As its known that some patients will lose CD19 expression on CAR T-cell therapy, the regimen may no longer be an effective treatment option.

For those patients that had failed CAR T-cell therapy, substantial proportions, about 30% of them, may have lost CD19 expression and then may not be eligible anymore for this regimen. There is, however, a substantial proportion of patients that retains CD19 and in whom tafasitamab/lenalidomide can be used as a treatment option, Salles commented.

A large proportion of patients will maintain CD19 expression following CAR T-cell therapy, so tafasitamab plus lenalidomide may still be effective in a percentage of patients.

Its hard to say because we dont have a lot of data, but we do know there are other CD19-directed therapies outside of CAR T cell development, Maddocks told Targeted Oncology. I think in the next few years, were going to see patients treated both pre- and post-CAR T with other CD19-directed therapies, and well have more information on this.

The combination of polatuzumab vedotin (Polivy) plus bendamustine (Bendeka) and rituximab (BR) was approved by the FDA as treatment of patients with relapsed/refractory DLBCL after 2 prior lines of therapy in June 2019 based on the findings from the phase 1b/2 GO29365 (NCT02257567) clinical trial. Although this option is also not FDA-approved for the treatment of patients after first relapse, Maddocks noted that this was the only treatment evaluated in a randomized trial. The study had included patients who were ineligible for transplant.

Significant improvements were observed with polatuzumab vedotin plus BR compared with BR alone in an international, multicenter, open-label study, particularly in regard to the ORR, CRs, progression-free survival (PFS), and overall survival (OS). CRs were observed in 40.0% of the patients with the combination versus 17.5% with BR alone. Survival rates favored the combination as well, with a median PFS of 9.5 months with the combination versus 3.7 months with BR alone (HR, 0.36; 95% CI, 0.21-0.63; P <.001) and a median OS of 12.4 months versus 4.7 months (HR, 0.42; 95% CI, 0.24-0.75; P =.002), respectively.2

The addition of polatuzumab did increase toxicity from the standpoint of cytopenias, but that didn't really translate to increased serious infections. It did add neuropathy as a side effect, but most of that was reversible, so I think this was a regimen that, by the addition of polatuzumab, was something that you could offer patients that did give them somewhat of a better overall response and was more durable than just giving them a palliative chemotherapy alone, Maddocks added. This is also a regimen that's been used in patients who were not able to achieve a remission to bridge them to CAR T or in some patients after CAR T, and so I can understand why this was definitely one of the more favorable choices.

In the study, grade 3/4 neutropenia was observed more frequently in the combination arm (42.6%) compared with the BR alone arm (33.3%), but the occurrence of grade 3/4 infections was comparable between the 2 groups (23.1% vs. 20.5%, respectively). In addition, the study authors noted that although many of the fatal AEs occurred after disease progression, 11 patients in the BR arm experienced fatal AEs compared with 9 in the combination arm, infection being the most common, which was the cause in 4 patients in each arm.2

Although the regimen appeared tolerable in this setting, Maddocks tweeted, it is more attractive than chemotherapy alone and understandable why it was chosen [as the second-best option in the Twitter poll].

Among the treatment options considered in our twitter poll ahead of the tweet chat, selinexor (Xpovio) only caught the attention of 16.7% of voters, similar to CAR T-cell therapy. However, both of these options are currently only approved in patients who have received at least 2 prior lines of therapy, which this case did not.

In regard to selinexor in particular, Maddocks tweeted, Looking at the single arm phase 2 data, it also has the lowest overall response rates of all the options listed with an ORR of 28%.

Selinexor received its approval from the FDA in June 2020, which is indicated for the treatment of adult patients with relapsed/refractory DLBCL, not otherwise specified, who have received at least 2 prior systemic therapies. This is the only oral single-agent therapy approved in this setting, and it is also the only nuclear export inhibitor approved by the FDA for use in hematologic malignancies.

The agent was approved on the basis of the phase 2b SADAL clinical trial, which demonstrated an ORR of 29% with 13% CRs and 16% PRs. The responses achieved in the study were durable, which led to a median duration of response of 9.2 months in the overall population (95% CI, 4.8-23.0) and 13.5 months in those who had achieved a CR (95% CI, 9.3-23.0).3

The most common treatment-related AEs were cytopenias and gastrointestinal/constitutional symptoms, which were generally reversible and manageable with dose modifications and/or standard supportive care approaches. The most common on-hematologic AEs, which were mostly grade 1/2, were nausea (52.8%), fatigue (37.8%), and anorexia (34.6%). The most common grade 3/4 AEs included thrombocytopenia (39.4%), neutropenia (20.5%), and anemia (13.4%). No treatment-related grade 5 AEs were observed.

CAR T-cell therapy, on the other hand, offers a unique option to this patient case even though it is still only approved in patients who have progressed or relapsed after 2 prior therapies or SCT. The TRANSCEND-PILOT-017006 (NCT03483103) study is evaluating the potential for CAR T-cell therapy lisocabtagene maraleucel (liso-cel) as treatment of patients with relapsed/refractory aggressive B-cell non-Hodgkin lymphoma who have received at least 1 prior therapy and are ineligible for SCT. While this does appear promising for introducing CAR T-cell therapy earlier on for patients with DLBCL, the treatment is not available off trial and is not a standard approach.

Maddocks told Targeted Oncology, It's very clear who's eligible for autologous transplant by age and comorbidities, but with CAR T, it's not so clear all the time who is going to be a candidate. There's not as great of data or information on who is going to be a candidate for that or not. Probably more patients are going to be a candidate for transplant, but there is still going to be patients that are comorbidities that they're not going to be a candidate for CAR T cells, and while they're approved in this setting and they can be very effective, there's also logistical issues, including that right now there's only certain centers, most often transplant centers, that are able to administer CAR T cells, so the patient has to have access to a center, they have to be able to get through the time that their leukapheresis cells are sent out and then sent back, and there's still barriers to cost and insurance in some patients, too.

This particular patient case does represent a challenge, Maddocks said. Historically, this is not a patient that's going to be a candidate for an autologous SCT, and that's going to be the only curative approach. CAR T is not approved in this setting, which is the other curative approach we know outside of patients who are unable to get to autologous STC, or at least appears to be likely curative for a percentage of patients.

Overall, CAR T-cell therapy is not a viable treatment option for the patient depicted in our tweet chat discussion, although it can still offer curative opportunities to a select group of patients with DLBCL who are ineligible for transplant.

In conclusion, tafasitamab plus lenalidomide helps fulfill the unmet need of patients who are in first relapse but are ineligible for transplant, which is the only curative option for patients with relapsed/refractory DLBCL. Although CAR T cells appear hopeful in this space, more research needs to be done to further determine their role in the treatment paradigm.

When you look at relapsed DLBCL, in general, and have these options, it's exciting for our patients to be able to have these. All of these have come up in the last 1 to 2 years, CAR T being a little bit longer than the other 3 regimens, but they all have offered patients tolerable therapy in the setting of previously not having these options.

Reference

1. Salles G, Duell J, Gonzlez-Barca E, et al. Long-term outcomes from the phase II L-MIND study of Tafasitamab (MOR208) plus lenalidomide in patients with relapsed or refractory diffuse large B-cell lymphoma. Presented at: Presented at: EHA25 Virtual; June 11-21, 2020. Abstract EP1201.

2. Sehn LH, Herrera AF, Flowers CR, et al. Polatuzumab Vedotin in Relapsed or Refractory Diffuse Large B-Cell Lymphoma.J Clin Oncol. 2019;38(2):155-165. doi: 10.1200/JCO.19.00172

3. Kalakonda N, Cavallo F, Follows G, et al. A phase 2b study of selinexor in patients with relapsed/refractory (r/r) diffuse large B-cell lymphoma (DLBCL).Hematol Oncol. 2019;37(S2). doi: 10.1002/hon.31_2629

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Tweet Chat Recap: Evaluating Treatment Approaches for Relapsed/Refractory DLBCL - Targeted Oncology

Seeing the eye like never before | Newsroom – UW Medicine Newsroom

By Stem Cell Treatment

While there is no cure for blindness and macular degeneration, scientists have accelerated the process to find a cure by visualizing the inner workings of the eye and its diseases at the cellular level.

In an effort led by UW Medicine, researchers successfully modified the standard process of optical coherence tomography (OCT) to detect minute changes in response to light in individual photoreceptors in the living eye.

The results were published Sept. 9 in Science Advances.

We have now accelerated the life cycle of vision restoration, said lead author Vimal Prabhu Pandiyan, a ophthalmology researcherat the University of Washington School of Medicine.

The study was fundedin partby the National Eye Institutes Audacious Goals Initiative, which embraces bold ideas in helping people to see better.

The OCT modifications outlined in the study will help researchers who want to test therapiessuch as stem cells or gene therapy to treat retinal disease. They now have the tools to zoom in on the retina to evaluate whether the therapy is working.

Corresponding author Ramkumar Sabesan, a UW assistant research professor of ophthalmology, said the only wayto objectively measure the eye currently is to look at a wide retinal area. Sabesan said researchers currently can attach electrodes on the cornea but it captures a large area with around 1 million cells. Now they are talking about nanometers, or one billionth of a meter a small fraction of the size of a cell, providing orders of magnitude improvement.

Since photoreceptors are the primary cells affected in retinal generation and the target cells of many treatments, noninvasive visualization of their physiology at high resolution is invaluable, the researchers wrote.

Cone photoreceptors are the building blocks of sight, capturinglight and funneling information to the other retinal neurons. They are a key ingredient in how we process images and patterns of light falling on the retina.

Optical coherence tomography has been around since the 1990s. In this study, researchers used OCT with adaptive optics, line-scanning and phase-resolved acquisition to deliver the concept of Thomas Youngs interference to the human eye. With the ability to zoom in on the retina at high speeds, they found that cone photoreceptors deform at the scale of nanometers when they first capture light and begin the process of seeing.

As Sabesan explained: You can imagine a picture that looks visually and structurally normal. But when we interrogate the inner working of the retina at a cellular scale, we may detect a dysfunction sooner than what other modalities can do. A doctor then can prescribe medication to intervene early or follow the time-course of its repair via gene therapy or stem cell therapy in the future.

We will now have a way to see if these therapies are acting in the way they should, Sabesan said.

The study also involved researchers at Stanford University, University of California,Berkeley, and University of California, Riverside.

The study was funded by NIH grants U01EY025501, EY027941, EY029710, EY025501, and P30EY001730; Research to Prevent Blindness Career Development Award; Foundation Fighting Blindness; Murdock Charitable Trust; Burroughs Welcome Fund Careers at the Scientific Interfaces; and Unrestricted grant from the Research to Prevent Blindness.

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Seeing the eye like never before | Newsroom - UW Medicine Newsroom

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