This year, the bioprinting community has discovered ways to speed up precision in 3D bioprinting. Even though experts have warned us that 3D printed organs might not be available for a long time, we cant help the excitement after witnessing crucial progress in 2019 that gets us closer to the possibility of having functional, compatible and working organs and tissues, especially after researchers made significant progress with different tissues and structures. Other relevant research highlights of this year include new bioprinting machines and bioinks, innovation centers and projects from Australias bioprinting research community, and a map of bioprinting companies that gave us a clear grasp of the main biotech hubs around the world.

Bioprinters and bioinks

In early November we learned that researchers at Harvard UniversitysWyss Institute for Biologically Inspired Engineeringcreated a fast multimaterial 3D printer. Thanks to a unique 3D printed printhead design, users can seamlessly switch between multiple different materials up to 50 times per second. The 3D printing technique called Multimaterial Multinozzle 3D (MM3D) printing could revolutionize the process of printing complex structures and is just one of the many advances in 3D bioprinting coming from Wyss.

On the European front, former regenHu CEO and founder, Mark Thurner, embarked on a new journey after launching his second company, mimiX Biotherapeutics, to bioprint in the operating room using sound. The all new bioprocessing technology called Sound Induced Morphogenesis (SIM) will be launched commercially in the summer and has already demonstrated with scientific evidence that it offers tissue engineering strategies to overcome todays obstacles, for example, the creation of dense networks of cells suitable for micro vascularization.

New bioprinters became commercially available in 2019, including CELLINKs bioprinting platform for complex structures, the Bio X6, as well as the Lumen X, a digital light processing bioprinter resulting from a joint collaboration between the seasoned company and Volumetric that is designed to enhance inventions in creating more substantial vascular structures. Another Swedish-based biotech company called Fluicell released a high-resolution bioprinting technology in both 2D and 3D called Biopixlar, capable of creating complex tissue-like structures where positioning of individual cells can be controlled from a gamepad, the novel feature that allows users to control the system just like they would a videogame was well received.

CELLINK BIO X 6 (Image credit: CELLINK)

Bioink developments this year were plentiful. Companies like Allevi turned out liver-specific bioinks, Biogelx launched their first product range of synthetic bioinks for a variety of 3D printing applications, and the Tessenderlo Group released their first gelatin bioink in their Claro series of tissue-engineering products. As far as academic researchers go, they are not lagging behind, ateam of researchers atTexas A&M University have developed a 3D printable hydrogel bioink containing mineral nanoparticles that can deliver protein therapeutics to control cell behavior, while researchers at the Rensselaer Polytechnic Institute and Yale University, turned living human skin cells into a bioink to print artificial skin, which then grows its own blood vessel system. In years to come, once these amazing advances hit the pre-clinical and clinical phases we will see an even bigger revolution in bioprinting.

Cardiac tissue engineering

(Image credit: Tel Aviv University)

The Tel Aviv University story about researchers making significant progress with 3D bioprinting by introducing a new concept for engineering fully personalized cardiac patches to repair heart defects, became quite the hype of the year, especially after many news outlets around the world began using the words 3D printed and the human heart in the same headline. Leading many to believe that a functional beating heart that could replace organ transplant was just around the corner. Although researchers actually printed a cellularized heart-like structure with a natural architecture to demonstrate the potential of the approach for organ replacement, the focus of their work was on a novel 3D printing technique that uses patients stem cells and extracellular matrix (ECM) to create a personalized hydrogel as a bioink to 3D print thick, vascularized, and perfusable cardiac patches that completely match the immunological, cellular, biochemical, and anatomical properties of the patient, regenerating a previously defective or infarcted heart part.

Some of our most seasoned interviewees suggested that bioprinted organs in the long-term future might not be anatomically designed to look like our organs, but all that matters is that they carry the functions that humans require to live.

A growing bioprinting landscape for Australia

Many of our bioprinting stories this year revolved around biotechnology discoveries, new labs and collaborative research efforts in Australia. The approach to science and research that the countrys experienced professionals have, are consistently about teamwork and collaboration, leading us to believe that perhaps theyre onto something. Constant efforts to enroll researchers in projects between different universities have been aplenty, as well as the myriad of opportunities that they have generated this year to get together and engage in biotechnology to advance the field. Integrated research labs across various universities are booming as more and more students become interested in the engineering, design, medical and biochemical aspects of biofabrication. Leading bioprinting experts Gordon Wallace, Professor at the University of Wollongong, and Jason Chuen, Vascular Surgeon and Director of the 3D Medical Printing Laboratory in Melbourne, have been actively heading and participating in conferences and seminars across the country.

With breakthrough developments like 3D Alek, a bioprinter that replicates human ears for patients with microtia, to creating their own bioinks at the lab, researchers understand that the success of their work comes from sharing knowledge and creativity among peers.

Mapping the companies that make bioprinting successful

Bioprinting world map by 3DPrint.com

To get a better grasp of the landscape that has been building up and what we can expect for the future of bioprinting, 3DPrint.com decided to map out all the companies that are working on developing both bioprinters and bioinks to advance biofabrication. Our Bioprinting World Map offers a snapshot of some of the hubs around the world where biotechnology is taking off, as well as potential startups that could revolutionize the next generation of bio machines. As some of the smaller and new companies are scaling up, coming up with new technology to tackle a competitive environment (such as Aspect Biosystems and CTI Biotech), a few are struggling to stay afloat, like Organovo, and a great deal of university spin-out businesses represent some of the cutting edge research and innovation that is undertaken in faculties and institutes (like OxSyBio, a spin-off from the University of Oxford).

Overall, 2019 was a year of highs. Looking ahead to 2020, we can expect a continued surge in bioprinting research and development as well as an ecosystem of collaboration among scientists. We should also expect top research institutions and leading companies to continue flirting with new technologies to harness the power of 3D bioprinting, as well as continue investigating the functionality of tissues for regenerative medicine. Finally, it will be important to closely analyze the growing popularity of new methods that arise and that may inspire emerging trends in the field.

Join the discussion of this and other 3D printing topics at3DPrintBoard.com.

See original here:
The Year in Review: Bioprinting in 2019 - 3DPrint.com

Cardiac Stem Cells Comments Off on The Year in Review: Bioprinting in 2019 – 3DPrint.com | December 14th, 2019

Transparency Market Research (TMR) has published a new report on the global cell separation technology market for the forecast period of 20192027. According to the report, the global cell separation technology market was valued at ~ US$ 5 Bn in 2018, and is projected to expand at a double-digit CAGR during the forecast period.

Cell separation, also known as cell sorting or cell isolation, is the process of removing cells from biological samples such as tissue or whole blood. Cell separation is a powerful technology that assists biological research. Rising incidences of chronic illnesses across the globe are likely to boost the development of regenerative medicines or tissue engineering, which further boosts the adoption of cell separation technologies by researchers.

Expansion of the global cell separation technology market is attributed to an increase in technological advancements and surge in investments in research & development, such as stem cell research and cancer research. The rising geriatric population is another factor boosting the need for cell separation technologies Moreover, the geriatric population, globally, is more prone to long-term neurological and other chronic illnesses, which, in turn, is driving research to develop treatment for chronic illnesses. Furthermore, increase in the awareness about innovative technologies, such as microfluidics, fluorescent-activated cells sorting, and magnetic activated cells sorting is expected to propel the global cell separation technology market.

Request a PDF Sample on Cell Separation Technology Market Report

https://www.transparencymarketresearch.com/sample/sample.php?flag=S&rep_id=1925

North America dominated the global cell separation technology market in 2018, and the trend is anticipated to continue during the forecast period. This is attributed to technological advancements in offering cell separation solutions, presence of key players, and increased initiatives by governments for advancing the cell separation process. However, insufficient funding for the development of cell separation technologies is likely to hamper the global cell separation technology market during the forecast period. Asia Pacific is expected to be a highly lucrative market for cell separation technology during the forecast period, owing to improving healthcare infrastructure along with rising investments in research & development in the region.

Rising Incidences of Chronic Diseases, Worldwide, Boosting the Demand for Cell Therapy

Incidences of chronic diseases such as diabetes, obesity, arthritis, cardiac diseases, and cancer are increasing due to sedentary lifestyles, aging population, and increased alcohol consumption and cigarette smoking. According to the World Health Organization (WHO), by 2020, the mortality rate from chronic diseases is expected to reach 73%, and in developing counties, 70% deaths are estimated to be caused by chronic diseases.

Southeast Asia, Eastern Mediterranean, and Africa are expected to be greatly affected by chronic diseases. Thus, the increasing burden of chronic diseases around the world is fuelling the demand for cellular therapies to treat chronic diseases. This, in turn, is driving focus and investments on research to develop effective treatments. Thus, increase in cellular research activities is boosting the global cell separation technology market.

Increase in Geriatric Population Boosting the Demand for Surgeries

The geriatric population is likely to suffer from chronic diseases such as cancer and neurological disorders more than the younger population. Moreover, the geriatric population is increasing at a rapid pace as compared to that of the younger population. Increase in the geriatric population aged above 65 years is projected to drive the incidences of Alzheimers, dementia, cancer, and immune diseases, which, in turn, is anticipated to boost the need for corrective treatment of these disorders. This is estimated to further drive the demand for clinical trials and research that require cell separation products. These factors are likely to boost the global cell separation technology market.

According to the United Nations, the geriatric population aged above 60 is expected to double by 2050 and triple by 2100, an increase from 962 million in 2017 to 2.1 billion in 2050 and 3.1 billion by 2100.

Request for a Discount on Cell Separation Technology Market Report

https://www.transparencymarketresearch.com/sample/sample.php?flag=D&rep_id=1925

Productive Partnerships in Microfluidics Likely to Boost the Cell Separation Technology Market

Technological advancements are prompting companies to innovate in microfluidics cell separation technology. Strategic partnerships and collaborations is an ongoing trend, which is boosting the innovation and development of microfluidics-based products. Governments and stakeholders look upon the potential in single cell separation technology and its analysis, which drives them to invest in the development of microfluidics. Companies are striving to build a platform by utilizing their expertise and experience to further offer enhanced solutions to end users.

Read more here:
Cell Separation Technology Market is Expected to Elevate to a Value of US$ 13.6 Bn by 2027 - Techi Labs

Cardiac Stem Cells Comments Off on Cell Separation Technology Market is Expected to Elevate to a Value of US$ 13.6 Bn by 2027 – Techi Labs | December 14th, 2019

Transparency Market Research (TMR)has published a new report on the globalcell separation technology marketfor the forecast period of 20192027. According to the report, the global cell separation technology market was valued at ~US$ 5 Bnin 2018, and is projected to expand at a double-digit CAGR during the forecast period.

Overview

Cell separation, also known as cell sorting or cell isolation, is the process of removing cells from biological samples such as tissue or whole blood. Cell separation is a powerful technology that assists biological research. Rising incidences of chronic illnesses across the globe are likely to boost the development of regenerative medicines or tissue engineering, which further boosts the adoption of cell separation technologies researchers.

Expansion of the global cell separation technology market is attributed to an increase in technological advancements and surge in investments in research & development, such asstem cellresearch and cancer research. The rising geriatric population is another factor boosting the need for cell separation technologies Moreover, the geriatric population, globally, is more prone to long-term neurological and other chronic illnesses, which, in turn, is driving research to develop treatment for chronic illnesses. Furthermore, increase in the awareness about innovative technologies, such as microfluidics, fluorescent-activated cells sorting, and magnetic activated cells sorting is expected to propel the global cell separation technology market.

Want to know the obstructions to your companys growth in future? Request a PDF sample here

North America dominated the global cell separation technology market in 2018, and the trend is anticipated to continue during the forecast period. This is attributed to technological advancements in offering cell separation solutions, presence of key players, and increased initiatives governments for advancing the cell separation process. However, insufficient funding for the development of cell separation technologies is likely to hamper the global cell separation technology market during the forecast period. Asia Pacific is expected to be a highly lucrative market for cell separation technology during the forecast period, owing to improving healthcare infrastructure along with rising investments in research & development in the region.

Rising Incidences of Chronic Diseases, Worldwide, Boosting the Demand for Cell Therapy

Incidences of chronic diseases such as diabetes, obesity, arthritis, cardiac diseases, and cancer are increasing due to sedentary lifestyles, aging population, and increased alcohol consumption and cigarette smoking. According to the World Health Organization (WHO), 2020, the mortality rate from chronic diseases is expected to reach73%, and in developing counties,70%deaths are estimated to be caused chronic diseases. Southeast Asia, Eastern Mediterranean, and Africa are expected to be greatly affected chronic diseases. Thus, the increasing burden of chronic diseases around the world is fuelling the demand for cellular therapies to treat chronic diseases. This, in turn, is driving focus and investments on research to develop effective treatments. Thus, increase in cellular research activities is boosting the global cell separation technology market.

To Obtain All-Inclusive Information On Forecast Analysis Of Global Market, Request A PDF Brochure Here.

Increase in Geriatric Population Boosting the Demand for Surgeries

The geriatric population is likely to suffer from chronic diseases such as cancer and neurological disorders more than the younger population. Moreover, the geriatric population is increasing at a rapid pace as compared to that of the younger population. Increase in the geriatric population aged above 65 years is projected to drive the incidences of Alzheimers, dementia, cancer, and immune diseases, which, in turn, is anticipated to boost the need for corrective treatment of these disorders. This is estimated to further drive the demand for clinical trials and research that require cell separation products. These factors are likely to boost the global cell separation technology market.

According to the United Nations, the geriatric population aged above 60 is expected to double 2050 and triple 2100, an increase from962 millionin 2017 to2.1 billionin 2050 and3.1 billion2100.

Productive Partnerships in Microfluidics Likely to Boost the Cell Separation Technology Market

Technological advancements are prompting companies to innovate in microfluidics cell separation technology. Strategic partnerships and collaborations is an ongoing trend, which is boosting the innovation and development of microfluidics-based products. Governments and stakeholders look upon the potential in single cell separation technology and its analysis, which drives them to invest in the development ofmicrofluidics. Companies are striving to build a platform utilizing their expertise and experience to further offer enhanced solutions to end users.

Stem Cell Research to Account for a Prominent Share

Stem cell is a prominent cell therapy utilized in the development of regenerative medicine, which is employed in the replacement of tissues or organs, rather than treating them. Thus, stem cell accounted for a prominent share of the global market. The geriatric population is likely to increase at a rapid pace as compared to the adult population, 2030, which is likely to attract the use of stem cell therapy for treatment. Stem cells require considerably higher number of clinical trials, which is likely to drive the demand for cell separation technology, globally. Rising stem cell research is likely to attract government and private funding, which, in turn, is estimated to offer significant opportunity for stem cell therapies.

Biotechnology & Pharmaceuticals Companies to Dominate the Market

The number of biotechnology companies operating across the globe is rising, especially in developing countries. Pharmaceutical companies are likely to use cells separation techniques to develop drugs and continue contributing through innovation. Growing research in stem cell has prompted companies to own large separate units to boost the same. Thus, advancements in developing drugs and treatments, such as CAR-T through cell separation technologies, are likely to drive the segment.

As per research, 449 public biotech companies operate in the U.S., which is expected to boost the biotechnology & pharmaceutical companies segment. In developing countries such as China, China Food and Drug Administration(CFDA) reforms pave the way for innovation to further boost biotechnology & pharmaceutical companies in the country.

Global Cell Separation Technology Market: Prominent Regions

North America to Dominate Global Market, While Asia Pacific to Offer Significant Opportunity

In terms of region, the global cell separation technology market has been segmented into five major regions: North America, Europe, Asia Pacific, Latin America, and the Middle East & Africa. North America dominated the global market in 2018, followed Europe. North America accounted for a major share of the global cell separation technology market in 2018, owing to the development of cell separation advanced technologies, well-defined regulatory framework, and initiatives governments in the region to further encourage the research industry. The U.S. is a major investor in stem cell research, which accelerates the development of regenerative medicines for the treatment of various long-term illnesses.

The cell separation technology market in Asia Pacific is projected to expand at a high CAGR from 2019 to 2027. This can be attributed to an increase in healthcare expenditure and large patient population, especially in countries such as India and China. Rising medical tourism in the region and technological advancements are likely to drive the cell separation technology market in the region.

Launching Innovative Products, and Acquisitions & Collaborations Key Players Driving Global Cell Separation Technology Market

The global cell separation technology market is highly competitive in terms of number of players. Key players operating in the global cell separation technology market include Akadeum Life Sciences, STEMCELL Technologies, Inc., BD, Bio-Rad Laboratories, Inc., Miltenyi Biotech, 10X Genomics, Thermo Fisher Scientific, Inc., Zeiss, GE Healthcare Life Sciences, PerkinElmer, Inc., and QIAGEN.

These players have adopted various strategies such as expanding their product portfolios launching new cell separation kits and devices, and participation in acquisitions, establishing strong distribution networks. Companies are expanding their geographic presence in order sustain in the global cell separation technology market. For instance, in May 2019, Akadeum Life Sciences launched seven new microbubble-based products at a conference. In July 2017, BD received the U.S. FDAs clearance for its BD FACS Lyric flow cytometer system, which is used in the diagnosis of immunological disorders.

Read more here:
Cell Separation Technology Market Overview, Growth Forecast, Demand and Development Research Report to 2027 - VaporBlash

Cardiac Stem Cells Comments Off on Cell Separation Technology Market Overview, Growth Forecast, Demand and Development Research Report to 2027 – VaporBlash | December 14th, 2019

JavaScript is disabled on your browser. Please enable JavaScript to use all the features on this page.Highlights

Transplantation of iPSC-derived neural precursor cells (NPCs) shows beneficial effects for spinal cord injury (SCI).

Because unsafe iPSC-NPC lines can form tumors after grafting, provisions to attenuate this risk are substantially important.

Clinical application for SCI patients using iPSCs will be conducted in the near future.

For the past few decades, spinal cord injury (SCI) has been believed to be an incurable traumatic condition, but with recent developments in stem cell biology, the field of regenerative medicine has gained hopeful momentum in the development of a treatment for this challenging pathology. Among the treatment candidates, transplantation of neural precursor cells has gained remarkable attention as a reasonable therapeutic intervention to replace the damaged central nervous system cells and promote functional recovery. Here, we highlight transplantation therapy techniques using induced pluripotent stem cells to treat SCI and review the recent research giving consideration to future clinical applications.

Spinal cord injury

Neural precursor cells

Induced pluripotent stem cells

Clinical application

neural precursor cells

induced pluripotent stem cells

high mobility group box-1

swine leukocyte antigen

mixed lymphocyte reaction

human leukocyte antigen

peripheral blood mononuclear cells

directly reprogrammed neural precursor cells

-secretase inhibitor

herpes simplex virus type I thymidine kinase

oligodendrocyte progenitor cells

the Center for iPS Cell Research and Application

American Spinal Injury Association

chondroitin sulfate proteoglycans

Recommended articlesCiting articles (0)

2019 The Japanese Society for Regenerative Medicine. Production and hosting by Elsevier B.V.

Here is the original post:
Cell therapy for spinal cord injury using induced ...

Spinal Cord Stem Cells Comments Off on Cell therapy for spinal cord injury using induced … | December 12th, 2019

ESPCs derived from pig provide important implications for developmental biology, organ transplantation, regenerative medicine, disease modeling, and screening for drugs.

FREMONT, CA: Stem cell therapy, usually applied to humans, is now extended to animals too. It is a regenerative treatment applied to cats, dogs, pigs, and other animals. It includes removing cells from bone marrow, blood or fat, umbilical cords, and the cell can grow into any kind of cell and can repair damaged tissues. The regenerative therapy has been successful in animals. It can be used mainly for the treatment of spinal cord and bone injuries along with the problems with tendons, ligaments, and joints. One of the breakthroughs is the embryonic stem cell lines obtained from the pig.

Scientists have derived Expanded Potential Stem Cells (EPSCs) from pig embryos for the first time. They offer the groundbreaking potential to study embryonic development and produce translational research in genomics and regenerative medicine. Embryonic stem cells (ESC) are derived from the inner cells of early embryos called blastocysts. They are pluripotent cells as they can develop into various cell types of the body in the culture dish. The newly derived porcine EPSCs isolated from pig embryos are the first well-characterized cell lines worldwide. Their pluripotent ability provides important implications for developmental biology, organ transplantation, regenerative medicine, disease modeling, and screening for drugs.

The stem cells can renew themselves, showing that they can be kept in culture indefinitely while showing the typical morphology and gene expression patterns of embryonic stem cells. Because somatic cells have a limited lifespan, they cannot be used for such applications, and therefore the new stem cells are better suited for the lengthy selection process. These porcine stem cell lines can easily be edited with new genome editing techniques like CRISPR/Cas, and are currently the simplest, most versatile and precise method of genetic manipulation.

The EPSCs have a greater capacity to develop into numerous cell types of the organism as well as into extraembryonic tissue, the trophoblasts, rending them very unique and, thus, their name. This capacity is valuable for the future promising organoid technology where organ-like small cell aggregations are grown in 3D aggregates and used for research into early embryo development, various disease models, and testing of new drugs in Petri dishes. Also, they offer a unique possibility to investigate functions or diseases of the placenta in vitro.

More:
How Will Animals Get Benefitted by Stem Cell Therapy? - Medical Tech Outlook

Spinal Cord Stem Cells Comments Off on How Will Animals Get Benefitted by Stem Cell Therapy? – Medical Tech Outlook | December 12th, 2019

SAN DIEGO, Dec. 12, 2019 /PRNewswire/ -- Aspen Neuroscience, Inc. today announced its launch following a $6.5 million seed round led by Domain Associates and Axon Ventures and including Alexandria Venture Investments,Arch Venture Partners,OrbiMedand Section 32 to develop the first autologous cell therapies for Parkinson's disease. Aspen's proprietary approach was developed by the company's co-founders, Jeanne F. Loring, Ph.D., Professor Emeritus and founding director of the Center for Regenerative Medicine at The Scripps Research Institute, and Andres Bratt-Leal, Ph.D., a former post-doctoral researcher in Dr. Loring's lab. The company was initially supported by Summit for Stem Cell, a founding partner and non-profit organization which provides a variety of services for people with Parkinson's disease. Aspen is led by industry veteran Howard J. Federoff, M.D., Ph.D., as Chief Executive Officer.

Parkinson's disease is characterized by the loss of specific brain cells that make the chemical dopamine. Without dopamine, nerve cells cannot communicate with muscles and people are left with debilitating motor problems. Aspen is focusing on human pluripotent stem cells, cultured cells that can become any cell type in the human body. The company's research is specific to induced pluripotent stem cells (iPSCs), which it develops by taking a skin biopsy from a person with Parkinson's disease and turning the tissue into pluripotent stem cells using genetic engineering. Aspen then differentiates the pluripotent stem cells into dopamine-releasing neurons that can be transplanted into that same person (autologous), thereby restoring the types of neurons lost in Parkinson's disease.

As an autologous cell therapy for Parkinson's disease, Aspen's treatment would eliminate the need for immunosuppression because the neurons are transplanted back into the same patient from which they were generated. The use of immunosuppression is necessary with currently available cell therapies for Parkinson's disease and when transplanting cells from one patient to another (allogeneic) to prevent rejection but can pre-dispose the patient to life-threatening complications including infection and add cost to the patient and health system. Aspen is the only company in the world offering an autologous neuron replacement therapy for Parkinson's disease.

Aspen encompasses a powerful executive leadership team including Dr. Federoff who, in addition to his leadership roles at the UC Irvine Health System, was the Executive Vice President for Health Sciences and the Executive Dean of Medicine at Georgetown University. Dr. Federoff also has significant biotech industry experience including co-founding MedGenesis Therapeutix and Brain Neurotherapy Bio, as well as leading the U.S. Parkinson's Disease Gene Therapy Study Group. The company is also proud to announce the addition of several experienced and well-known members to its leadership team including Edward Wirth, M.D., Ph.D., as Chief Medical Officer.

Dr. Wirth currently serves as the Chief Medical Ofcer for Lineage Cell Therapeutics where he oversees clinical development of its two therapeutic programs for spinal cord injuries and lung cancer. He received his M.D. and Ph.D. from the University of Florida in 1994 and remained to conduct postdoctoral research including leading the University of Florida team that performed the rst human embryonic spinal cord transplant in the U.S. Dr. Wirth went on to serve as the Medical Director for Regenerative Medicine at Geron Corporation where the world's rst clinical trial of human embryonic stem cell (hESC)-derived product occurred which demonstrated initial clinical safety.

Drs. Federoff and Wirth are joined by Dr. Loring, as Chief Scientific Officer; Jay Sial, as Chief Financial Officer; Andres Bratt-Leal, Ph.D., as Vice President of Research and Development; Thorsten Gorba, Ph.D., as Senior Director of Manufacturing and Naveen M. Krishnan, M.D., M.Phil., as Senior Director of Corporate Development.

"Aspen is developing a restorative, disease modifying autologous neuron therapy for people suffering from Parkinson's disease," said Dr. Federoff. "We are fortunate to have such a high-caliber scientific and medical leadership team to make our treatments a reality. Our cell replacement therapy, which originated in the laboratory of Dr. Jeanne Loring and was later supported by Summit for Stem Cell and its President, Ms. Jenifer Raub, has the potential to release dopamine and reconstruct neural networks where no disease-modifying therapies exist."

Aspen's lead product (ANPD001) is currently undergoing investigational new drug (IND)-enabling studies for the treatment of sporadic Parkinson's disease. Aspen is also developing a gene-edited autologous neuron therapy (ANPD002) that is in the research stage and targeted toward familial forms of Parkinson's disease beginning with the most common genetic variant in the gene encoding glucocerebrosidase (GBA). Aspen leverages proprietary machine-learning tools and artificial intelligence to ensure quality control during manufacturing and to deliver a safe and reproducible product for each cell line.

"Aspen's financial backing, combined with its experienced and proven leadership team, positions it well for future success," said Kim P. Kamdar, Ph.D., Partner at Domain Associates, one of Aspen's seed investors. "Domain prides itself on investing in companies that can translate scientific research into innovative medicines and therapies that make a difference in people's lives. We clearly see Aspen as fitting into that category, as it is the only company using a patient's own cells for replacement therapy in Parkinson's disease."

About Aspen Neuroscience

Aspen Neuroscience Inc. is a development stage, private biotechnology company that uses innovative genomic approaches combined with stem cell biology to deliver patient-specific, restorative cell therapies that modify the course of Parkinson's disease. Aspen's therapies are based upon the scientific work of world-renowned stem cell scientist, Dr. Jeanne Loring, who has developed a novel method for autologous neuron replacement. For more information and important updates, please visithttp://www.aspenneuroscience.com.

View original content to download multimedia:http://www.prnewswire.com/news-releases/aspen-neuroscience-launches-with-6-5-million-seed-funding-to-advance-first-of-its-kind-personalized-cell-therapy-for-parkinsons-disease-300973830.html

SOURCE Aspen Neuroscience

Read the rest here:
Aspen Neuroscience Launches With $6.5 Million Seed Funding to Advance First-of-its-Kind Personalized Cell Therapy for Parkinson's Disease - P&T...

Spinal Cord Stem Cells Comments Off on Aspen Neuroscience Launches With $6.5 Million Seed Funding to Advance First-of-its-Kind Personalized Cell Therapy for Parkinson’s Disease – P&T… | December 12th, 2019

Parkinsons disease is characterized by the loss of specific brain cells that make the chemical dopamine. Without dopamine, nerve cells cannot communicate with muscles and people are left with debilitating motor problems. Aspen is focusing on human pluripotent stem cells, cultured cells that can become any cell type in the human body. Many health technology startups are on the raise to cure this disease. At the forefront is Aspen Neuroscience, a healthtech startup developing first-of-its-kind personalized cell therapy for Parkinsons disease.

Aspen Neuroscience is a development stage, private biotechnology company that uses innovative genomic approaches combined with stem cell biology to deliver patient-specific, restorative cell therapies that modify the course of Parkinsons disease.

Today,Aspen Neuroscience announced its official launch with $6.5 million seed financingto develop the first autologous cell therapies for Parkinsons disease.The round was led by Domain Associates and Axon Ventures and including Alexandria Venture Investments, Arch Venture Partners, OrbiMed and others.

Aspens proprietary approach was developed by the companys co-founders, Jeanne F. Loring, Ph.D., Professor Emeritus and founding director of the Center for Regenerative Medicine at The Scripps Research Institute, and Andres Bratt-Leal, Ph.D., a former post-doctoral researcher in Dr. Lorings lab. The company was initially supported by Summit for Stem Cell, a founding partner and non-profit organization which provides a variety of services for people with Parkinsons disease. Aspen is led by industry veteran Howard J. Federoff, M.D., Ph.D., as Chief Executive Officer.

The companys research is specific to induced pluripotent stem cells (iPSCs), which it develops by taking a skin biopsy from a person with Parkinsons disease and turning the tissue into pluripotent stem cells using genetic engineering. Aspen then differentiates the pluripotent stem cells into dopamine-releasing neurons that can be transplanted into that same person (autologous), thereby restoring the types of neurons lost in Parkinsons disease.

As an autologous cell therapy for Parkinsons disease, Aspens treatment would eliminate the need for immunosuppression because the neurons are transplanted back into the same patient from which they were generated. The use of immunosuppression is necessary with currently available cell therapies for Parkinsons disease and when transplanting cells from one patient to another (allogeneic) to prevent rejection but can pre-dispose the patient to life-threatening complications including infection and add cost to the patient and health system. Aspen is the only company in the world offering an autologous neuron replacement therapy for Parkinsons disease.

Aspen encompasses a powerful executive leadership team including Dr. Federoff who, in addition to his leadership roles at the UC Irvine Health System, was the Executive Vice President for Health Sciences and the Executive Dean of Medicine at Georgetown University. Dr. Federoff also has significant biotech industry experience including co-founding MedGenesis Therapeutix and Brain Neurotherapy Bio, as well as leading the U.S. Parkinsons Disease Gene Therapy Study Group. The company is also proud to announce the addition of several experienced and well-known members to its leadership team including Edward Wirth, M.D., Ph.D., as Chief Medical Officer.

Dr. Wirth currently serves as the Chief Medical Ofcer for Lineage Cell Therapeutics where he oversees clinical development of its two therapeutic programs for spinal cord injuries and lung cancer. He received his M.D. and Ph.D. from the University of Florida in 1994 and remained to conduct postdoctoral research including leading the University of Florida team that performed the rst human embryonic spinal cord transplant in the U.S. Dr. Wirth went on to serve as the Medical Director for Regenerative Medicine at Geron Corporation where the worlds rst clinical trial of human embryonic stem cell (hESC)-derived product occurred which demonstrated initial clinical safety.

Drs. Federoff and Wirth are joined by Dr. Loring, as Chief Scientific Officer; Jay Sial, as Chief Financial Officer; Andres Bratt-Leal, Ph.D., as Vice President of Research and Development; Thorsten Gorba, Ph.D., as Senior Director of Manufacturing and Naveen M. Krishnan, M.D., M.Phil., as Senior Director of Corporate Development.

Aspen is developing a restorative, disease modifying autologous neuron therapy for people suffering from Parkinsons disease, said Dr. Federoff. We are fortunate to have such a high-caliber scientific and medical leadership team to make our treatments a reality. Our cell replacement therapy, which originated in the laboratory of Dr. Jeanne Loring and was later supported by Summit for Stem Cell and its President, Ms. Jenifer Raub, has the potential to release dopamine and reconstruct neural networks where no disease-modifying therapies exist.

Continue reading here:
Aspen Neuroscience launches with $6.5M seed funding to develop personalized and autologous cell therapy for Parkinson's disease - TechStartups.com

Spinal Cord Stem Cells Comments Off on Aspen Neuroscience launches with $6.5M seed funding to develop personalized and autologous cell therapy for Parkinson’s disease – TechStartups.com | December 12th, 2019

LentiGlobin, Bluebird Bios investigational gene therapy for sickle cell disease (SCD), continues to show promising results in SCD patients participating in the companys Phase 1/2 HGB-206 clinical trial, according to the latest study data.

The new findings which included data from additional patients treated in the trial, updated data from those previously reported, and exploratory analyses were presented at the 61st American Society of Hematology (ASH) Annual Meeting and Exposition, held Dec. 6-10 in Orlando, Fla.

LentiGlobinisa gene therapy that has been developed to increase the levels of hemoglobin the protein that transports oxygen in the blood in people with SCD.

The therapy works by delivering functional copies of a modified form of the beta-globin gene (A-T87Q-globin gene) into patients red blood cell precursors, known as hematopoietic stem cells, or HSCs. Once these precursors differentiate, their red blood cells start producing a modified version of hemoglobin, called HbAT87Q.

By boosting the production of this anti-sickling form of the protein, LentiGlobin reduces the proportion of defective hemoglobin in patients red blood cells. That, in turn, reduces the sickling and destruction of these red blood cells and other complications associated with SCD.

The safety and efficacy of LentiGlobin is currently being evaluated in three groups identified as A-C of SCD patients participating in Bluebirds ongoing open-label, Phase 1/2 HGB-206 trial (NCT02140554).

Those in group A were treated per the original trial protocol. Meanwhile, those in groups B and C received an enhanced treatment protocol, approved in 2016, that is designed to increase the therapys efficiency. In groups A and B, patients HSCs were extracted from the bone marrow, while in group C, they were extracted from the blood.

As of the data cutoff date of August 26, 2019, seven participants in group A, two in group B, and 17 in group C had been treated with LentiGlobin. According to new data presented at the meeting, only two patients from group A required regular blood transfusions after the treatment.

In addition, the updated findings revealed that the levels of anti-sickling HbAT87Q remained stable in all participants from groups A and B over a post-treatment follow-up period of three years. Similarly, levels of total hemoglobin also were found to have remained stable in both patient groups over a two-year follow-up.

At the trial participants last visit, the median levels of anti-sickling HbAT87Q were 0.9 g/dL among those from group A, and 3.6 g/dL and 7.1 g/dL in the two patients from group B. The median levels of total hemoglobin were 9.0 g/dL among patients from group A, and 11.3 g/dL and 13.0 g/dL among those from group B.

Normal levels of hemoglobin in the blood range from 12.5 to 17.5 g/dL.

Among 12 patients from group C who were followed for at least six months, the median levels of anti-sickling HbAT87Q made up at least 40% of their total hemoglobin. At their last visit, the levels of anti-sickling HbAT87Q ranged from 2.7 to 9.0 g/dL, and the levels of total hemoglobin from 9.3 to 15.2 g/dL.

In groups A and B, LentiGlobin reduced the frequency of painful vaso-occlusive crises (VOCs) and acute chest syndrome (ACS) in the two years following treatment.

Nine patients from group C who were followed for at least six months had experienced four or more VOCs or ACS episodes in the two years prior to receiving LentiGlobin. Treatment with the gene therapy led to a reduction of 99% in the frequency of annual VOCs and ACS. In this group, there were no reports of ACS or severe VOCs for up to 21 months following treatment.

Moreover, among those from group C, LentiGlobin reduced the levels of different markers of red blood cells destruction, including reticulocytes, lactate dehydrogenase (LDH), and bilirubin.

LentiGlobins safety profile was consistent with previous data. No serious adverse events related to treatment were reported during the study. Only one mild, non-serious event of hot flush was found to be related to LentiGlobin. That event was rapidly resolved and did not require treatment.

Exploratory analyses were performed in a sub-group of patients from all three groups. In 12 participants who had been followed for at least six months, more than 70% of the individuals red blood cells were found to contain the anti-sickling HbAT87Q at the last study visit, these analyses showed. Moreover, in four of these patients, nearly all their red blood cells (90%) were positive for HbAT87Q.

In addition, exploratory analyses revealed that participants red blood cells were less prone to sickling following treatment with LentiGlobin.

At ASH, the growing body of data from our clinical studies of LentiGlobin for SCD reflects results from 26 treated patients with up to four years of follow-up, David Davidson, MD, Bluebird Bios chief medical officer, said in a press release.

We continue to observe patients treated in Group C producing high levels of gene-therapy derived anti-sickling hemoglobin, HbAT87Q, accounting for at least 40% of total hemoglobin in those with six or more months of follow-up, and exploratory assays show that HbAT87Q is present in most red blood cells of treated patients, Davidson said.

The robust production of HbAT87Q was associated with substantial reductions of sickle hemoglobin, HbS, as well as improvement in key markers of hemolysis [red blood cells destruction]. Most importantly, patients in Group C have not experienced any episodes of acute chest syndrome or serious vaso-occlusive crises following LentiGlobin for SCD treatment, he added.

The company is recruiting participants with transfusion-dependent -thalassemia (TDT) for a Phase 3 trial (NCT03207009) testing LentiGlobin. Moreover, according to the companys pipeline, there is a Phase 2/3 trial planned in sickle cell disease for this gene therapy.

Joana is currently completing her PhD in Biomedicine and Clinical Research at Universidade de Lisboa. She also holds a BSc in Biology and an MSc in Evolutionary and Developmental Biology from Universidade de Lisboa. Her work has been focused on the impact of non-canonical Wnt signaling in the collective behavior of endothelial cells cells that make up the lining of blood vessels found in the umbilical cord of newborns.

Total Posts: 94

Margarida graduated with a BS in Health Sciences from the University of Lisbon and a MSc in Biotechnology from Instituto Superior Tcnico (IST-UL). She worked as a molecular biologist research associate at a Cambridge UK-based biotech company that discovers and develops therapeutic, fully human monoclonal antibodies.

Read more from the original source:
LentiGlobin Gene Therapy Continues to Show Promising Results in SCD, Updated Trial Data Shows - Sickle Cell Anemia News

Bone Marrow Stem Cells Comments Off on LentiGlobin Gene Therapy Continues to Show Promising Results in SCD, Updated Trial Data Shows – Sickle Cell Anemia News | December 12th, 2019

According to an analysis of interim results from a randomized study of young patients with high- or intermediate risk B-cell precursor acute lymphoblastic leukemia (B-ALL) at first relapse, the overall efficacy and safety of post-reinduction therapy with the bispecific T-cell engager (BiTE) blinatumomab outperformed conventional chemotherapy. The findings from this study were presented at the 61st American Society of Hematology (ASH) Annual Meeting and Exposition held in Orlando, Florida.

Disease relapse, particularly early relapse, following initialtreatment of children, adolescents, and young adults with B-ALL is a marker ofpoor prognosis. While allogeneic hematopoietic stem cell transplantation (HSCT) is typicallyconsidered the treatment of choice for these patients, barriers to itsimplementation can include concerns related to adverse events associated withreinduction and subsequent consolidation chemotherapy, and the presence ofminimal residual disease (MRD) following administration of second-remission reinductiontherapy. Those patients who experienceearly bone marrow relapse, and those with MRD greater than 0.1% in the settingof a prolonged CR, at the end of reinduction therapy are considered to have high-riskand intermediate-risk disease, respectively.

Blinatumomab is an artificial, bispecific monoclonal antibody-basedconstruct created from the fusion of single-chain variable fragments from 2different antibodies. In the case of blinatumomab, targets include the CD3receptor on T cells and CD19 on B cells, resulting in the formation of a link betweenthese 2 cell types.

Currently, blinatumomab is approved by the US Food and DrugAdministration (FDA) for the treatment of adult and pediatric patients with B-ALL in first or second CR,with minimal residual disease (MRD) greater than or equal to 0.1%, as well asfor patients with relapsed/refractory B-ALL.2

In this phase 3 Childrens Oncology Group study (AALL1331; ClinicalTrials.gov Identifier: NCT02101853), patients with B-ALL in first relapse between the ages of 1 and 30 years with bone marrow blasts less than 25% and/or failure to clear extramedullary disease following reinduction chemotherapy (UKALLR3 regimen3) were randomly assigned in a 1:1 ratio following risk assessment to receive either 2 blocks of intensive consolidation chemotherapy according to the UKALLR3 regimen3 or two 4-week cycles of otumumab separated by a 1-week break. Allogeneic HSCT was scheduled following these treatments.

The primary end point of the trial was intent-to-treat disease-freesurvival (DFS), with secondary study end points including MRDresponse, overall survival (OS), and ability to proceed to HSCT.

A planned interimanalysis of 208 patients, performed followingthe occurrence of approximately 60% of expected events,included only those with high- (67%) or intermediate-risk (33%) disease. Patientages ranged from 1 to 27 years, with a median age of 9 years.

At a medianfollow-up of 1.4 years, some of the key efficacy findings from this analysis includedrates of 2-year DFS in the intention-to-treat (ITT) population of 41.0% forpatients receiving chemotherapy and 59.3% for those treated with blinatumomab (P =.050). Rates of 2-year OS forpatients in these 2 study arms were 79.4% (blinatumomab) and 59.2% (chemotherapy),(P =.005).

The percentages ofthose achieving undetectable MRD after reinduction chemotherapy were only 22%and 18% in the chemotherapy and blinatumomab arms, respectively. Followingblock 2 of chemotherapy (ie, first cycle of consolidation chemotherapy) orcycle 1 of blinatumomab, rates of undetectable MRD increased to 29% in thechemotherapy arm and 76% in the blinatumomab arm (P <.0001).

Regarding resultsrelated to MRD response, all of the benefit of blinatumomab with respect to MRDclearance appeared to occur in the first cycle, commented PatrickA. Brown of the Sidney Kimmel ComprehensiveCancer Center, Johns Hopkins University, Baltimore, Maryland, who was thepresenting study author.

Furthermore, 45%of patients in the chemotherapy arm compared with 73% of those in the blinatumomabarm were able to proceed to HSCT (P<.0001).

Regarding patientsafety, 4 and 0 patients receiving blinatumomab or chemotherapy, respectively,experienced a postinduction, induction-related toxic death.

In addition, thefrequencies of specific adverse events were considerably higher in thechemotherapy vs the blinatumomab arm. For example, rates of grade 3 or higher febrileneutropenia were 44% and 46% for patients receiving the 2nd and 3rd blocks ofthe UKALLR3 regimen, respectively, but only 4% and 0% of patients receivingcycle 1 and cycle 2 of blinatumomab, respectively (P <.001). Similar differences between the 2 study arms wereobserved with respect to the rates of infections and sepsis.

For patientsreceiving blinatumomab, low-grade cytokine release syndrome (CRS), occurring in22% of patients, was generally limited to the to the first cycle. Seizuresoccurred in 4% and 0% of patients during cycles 1 and 2, respectively, and the incidenceof mostly low-grade encephalopathy was 14% in cycle 1 and 11% in cycle 2.

Accordingto the results of this scheduled interim analysis, the prespecified monitoring thresholdto the primary end point of DFS was not crossed. However, based on the overallresults of the study, the data monitoring committee recommended permanentclosure of study randomization for patients with high- or intermediate-riskdisease, with those in these risk groups immediately crossed over to theblinatumomab arm.

We believe that blinatumomab constitutes anew standard of care in this setting, concluded Dr Brown.

Disclosure:Some of the authors disclosed financial relationships with the pharmaceuticalindustry. For a full list of disclosures, please refer to the originalabstract.

Read more of Cancer Therapy Advisors coverage of ASHs annual meeting by visiting the conference page.

References

Go here to read the rest:
Blinatumomab May Become New Standard of Care for Post-Reinduction Therapy in Young Patients With B-ALL - Cancer Therapy Advisor

Bone Marrow Stem Cells Comments Off on Blinatumomab May Become New Standard of Care for Post-Reinduction Therapy in Young Patients With B-ALL – Cancer Therapy Advisor | December 12th, 2019

by Tyler English | published Dec. 11th, 2019

illustration by Darius Serebrova

Ever wonder where those neon yellow, green, blue and pink fish came from? You know, the ones that have all the matching accessories: tanks, decorations, rocks and their own special ultraviolet light? Well, as it turns out, a team ofscientists in Singapore were the first ones to genetically modify fish to glow in such a way.

Genetic editing in small animals and plants has been aroundsince the 1970s, according to Synthego, a company that providesgeneticallyedited stem cells. Starting with plants and bacteria, scientists began to explore the realm of DNA and genetics. As their understanding of the proteins grew, so did their curiosity.

When scientists learned how to modify the genes of small, simple organisms, they began to wonder, "How could this be applied to humans?"

The scientific community is stirring with the emergence of CRISPR DNA,more specifically known as the CRISPR-Cas9 protein.CRISPR stands forClustered Regularly Interspaced Short Palindromic Repeats.CRISPR is a faster, cheaper and more accurate way of editing the genome, according to theNational Institute of Health.By sending in two different pieces of CRISPR DNA,scientists are able to modify genes. To do so, theycutout areas of genes that aren'tperforming how they should be or as they're expected to.

Dr. Sandi Connelly, a principallecturer in the Thomas H. Gosnell School of Life Sciences, explained how DNA works and what the CRISPRCas-9 protein actually does. Connelly compared DNA to a street of houses each person has different foundations that sprout out different and unique homes.

CRISPR is a piece of DNA, and we [scientists] attach to it an enzyme ...it cuts the DNA at a very specific place like a pair of scissors, Connelly said. When we look at CRISPR, typically we look at CRISPR Cas-9."

Whereas CRISPR is the DNA itself, Cas-9 is the enzyme, a specialized protein that splits the DNA.Connelly said that this allows for both the CRISPR DNA and the original DNA to stick together like magnets. However, due to the specificity of this technique, scientists need to know where in the DNA they'relooking.

Using those same enzymes, we can cut [and] place back inthe good gene, Connelly said.

Now, this technique would not be doneby injecting the CRISPR DNA directlyinto a fully grown adult. Instead,scientists would take a sample of a persons bone marrow and alter the genes of those cells. Since bone marrow is responsible for producing red blood cells, the new altered bone marrow will produce cells with the new DNA.

Connelly saidthechangeswouldnot be instantaneous.The human body replaces a majority of its cells within 13 days, soit would take around two weeks for the newly edited gene to be present in the human body.

The ability to now alter genes of morecomplexorganisms brings with it a variety of applications. Plants can be changed to increase nutritional value and pesticidal properties,whereas bacteria can be used to generate hormones and medicines.

Dr. David Holtzman,an adjunct professor in the College of Science, understands how gene editing is used and what it could be used for.

Most people are familiar with it [gene editing] for things like modifying plants ...[but] there is a lot of misunderstanding about gene editing, Holtzman said.

There is a lot of misunderstanding about gene editing.

CRISPR has begun to work its way into at-home kits, where those with some scientificexpertise can genetically modify their own plants to glow or be a different color. This is fairly simple in the world of gene editing as it is changing a simple expressed trait one that isbiologically shown.

Genes decide what traits a person has, but that persons environment and what happens to their body determines how those traits are expressed. As gene editing becomes more and more innovative, Holtzman said that there are limitations to what gene editing can and cannot do.

It turns out most traits are more than one gene, Holtzman said.

Holtzman used hair color as an example. Numerous genes and sections of DNA code for what an individual's hair colorwill be. Itcan behard and time-consuming to find the right area of the DNA to target for modification.

Connelly talked about the idea of changing hair coloras well,but took it a few steps further. Shesuggestedthat we may start wanting to create offspring that all have blonde hair and blue eyes, which realistically we could accomplish. This then opens parents up to the ideas of having all male children or all female children.

In recent years, science has progressed faster than we could have thought.What appeared to be science fiction in the past is inching ever closer to our scientific reality.

The ability to do [new]things happens a lot faster than our understanding of what we are doing, Holtzman said.

Regardless of the potential scientific progress that could be made, Holtzman, Connelly and other members of the scientific community are having conversations about what should be done with this technology. Where should the limits lie, and how far should humans gowith genetic technology?

"Where should the limits lie, and how far should humansgowith genetic technology?"

If our parents changed our genes, they would also be changing the genes of all of our descendants by extension. Did they consent to something like that?

Some might argue, whether we gene edit or not, we dont really have control over what our parents did, Holtzman said. There is the possibility that if we changed [certain genes]then we can change them back.

Reversal isn't a guarantee, though.

Holtzman mentioned ways in which gene editing could greatly improve the quality of life for all humankind, such as curing Alzheimers disease. Connelly brought up how easy it would be to reduce the effects of aging using genetic modification.

The consequences of the choices made nowmay not affect the generation making them. As the movement to improve the genetic composition of the human race pushes forward,plots in sci-finovelsmay no longer be abstract, distant futures. Rather, for better or worse, they could bethe reality we are setting up for generations to come.

Read the original:
Science Fiction Becoming Reality - Reporter Magazine

Bone Marrow Stem Cells Comments Off on Science Fiction Becoming Reality – Reporter Magazine | December 12th, 2019

DORVAL, QC, Dec. 12, 2019 /CNW/ - Novartis Pharmaceuticals Canada Inc. is pleased to announce that sites in Ontario have been certified in accordance with applicable requirements to treat eligible patients with Kymriah (tisagenlecleucel), the first chimeric antigen receptor T cell (CAR-T) therapy that received regulatory approval in Canada. Patients with relapsed/refractory (r/r) pediatric and young adult B-cell acute lymphoblastic leukemia (ALL) and adult r/r diffuse large B-cell lymphoma (DLBCL) may be eligible to be treated with Kymriah at one of the initially certified Canadian treatment sites. This news follows the recent decision by the Ontario government to fund Kymriah and publication of guidelines by Cancer Care Ontario regarding the enrolment process and criteria for patients to receive CAR-T cell therapy.ii

"Early results show that patients who receive CAR-T cell therapy are either seeing their cancer go into remission or can use CAR-T as a bridge to a stem cell transplant," said Christine Elliott, Deputy Premier and Minister of Health. "We are pleased that Ontario is among the first provinces in Canada to offer CAR-T cell therapy to eligible pediatric and adult patients."

Per the guidelines, eligible patients are now able to access Kymriah from The Hospital for Sick Children (SickKids) in Toronto and Hamilton Health Sciences. Princess Margaret Cancer Centre in Toronto and The Ottawa Hospital are working to complete the process to become certified treatment sites and join the network of qualified Ontario centres in 2020.

"Novartis would like to acknowledge and applaud the collaboration of all stakeholders involved, including Cancer Care Ontario, in helping to ensure patients have access to the first CAR-T therapy approved in Canada for patients with B-cell ALL and DLBCL who historically have poor outcomes. Having treatment sites certified in Ontario allows eligible patients to be treated with CAR-T therapy and is in line with our mission to provide rapid access to life-changing therapies to all Canadians," said Christian Macher, Country President & General Manager, Oncology, Novartis Pharmaceuticals Canada Inc. "Novartis is committed to bringing additional qualified Canadian treatment centres into the network to give patients the opportunity to be treated closer to home."

Due to the sophisticated and individualized nature of Kymriah, treatment sites that are part of the network are required to be FACT-accredited (Foundation for the Accreditation of Cellular Therapy). This means they are qualified to perform intravenous infusion of stem cells collected from the bone marrow of a donor, also referred to as allogeneic hematopoietic stem cell transplantation (alloSCT) and have experience with cell therapies and treating leukemia and lymphoma to facilitate safe and seamless delivery of Kymriah to eligible patients.

About KymriahKymriah (tisagenlecleucel), a CD19-directed genetically modified autologous T-cell immunocellular therapy, is approved to treat two life-threatening cancers that have limited treatment options and historically poor outcomes, demonstrating the critical need for new therapies for these patients.

Kymriah is approved by Health Canada for use in pediatric and young adult patients 3 to 25 years of age with B-cell acute lymphoblastic leukemia (ALL) who are refractory, have relapsed after allogenic stem cell transplant (SCT) or are otherwise ineligible for SCT, or have experienced second or later relapse; and for the treatment of adult patients with relapsed or refractory (r/r) large B-cell lymphoma after two or more lines of systemic therapy including diffuse large B-cell lymphoma (DLBCL) not otherwise specified, high grade B-cell lymphoma and DLBCL arising from follicular lymphomai.

Kymriah is a one-time treatment that uses a patient's own T cells to fight and kill cancer cells. Bringing this innovative therapy to Canadian patients requires collaboration among many health system stakeholders.

Kymriah (tisagenlecleucel) Important Safety InformationThe full prescribing information for Kymriah can be found at: http://www.novartis.ca

Novartis Leadership in Cell and Gene Therapy Novartis is at the forefront of investigational immunocellular therapy and was the first pharmaceutical company to significantly invest in CAR-T research, work with pioneers in CAR-T and initiate global CAR-T trials. Active research programs are underway targeting other hematologic malignancies and solid tumors, and include efforts focused on next generation CAR-Ts that involve simplified manufacturing schemes and gene edited cells.

About Novartis in CanadaNovartis Pharmaceuticals Canada Inc., a leader in the healthcare field, is committed to the discovery, development and marketing of innovative products to improve the well-being of all Canadians. In 2018, the company invested $52 million in research and development in Canada. Located in Dorval, Quebec, Novartis Pharmaceuticals Canada Inc. employs approximately 1,000 people in Canada and is an affiliate of Novartis AG, which provides innovative healthcare solutions that address the evolving needs of patients and societies. For further information, please consult http://www.novartis.ca.

About NovartisNovartis is reimagining medicine to improve and extend people's lives. As a leading global medicines company, we use innovative science and digital technologies to create transformative treatments in areas of great medical need. In our quest to find new medicines, we consistently rank among the world's top companies investing in research and development. Novartis products reach more than 750 million people globally and we are finding innovative ways to expand access to our latest treatments. About 109,000 people of more than 140 nationalities work at Novartis around the world. Find out more at http://www.novartis.com.

Kymriah is a registered trademark.

SOURCE Novartis Pharmaceuticals Canada Inc.

For further information: Novartis Media Relations, Daphne Weatherby, Novartis Corporate Communications, +1 514 633 7873, E-mail: camlph.communications@novartis.com

http://www.novartis.ca

Read the original post:
Novartis completes certification of initial sites in Ontario for first approved Canadian CAR-T therapy, Kymriah (tisagenlecleucel)[i] - Canada...

Bone Marrow Stem Cells Comments Off on Novartis completes certification of initial sites in Ontario for first approved Canadian CAR-T therapy, Kymriah (tisagenlecleucel)[i] – Canada… | December 12th, 2019

iPS cells may help halt failing vision

Getty

By Andy Coghlan

A woman in her 80s has become the first person to be successfully treated with induced pluripotent stem (iPS) cells. A slither of laboratory-made retinal cells has protected her eyesight, fighting her age-related macular degeneration a common form of progressive blindness.

Such stem cells can be coaxed to form many other types of cell. Unlike other types of stem cell, such as those found in an embryo, can be made from adult non-stem cells a discovery that in 2012.

Now, more than a decade after they were created, these stem cells have helped someone. at the RIKEN Laboratory for Retinal Regeneration in Kobe, Japan, and her team took skin cells from the woman and turned them into iPS cells. They then encouraged these to form retinal pigment epithelial cells, which are important for supporting and nourishing the retina cells that capture light for vision.

The researchers made a slither of cells measuring just 1 by 3 millimetres. Before in 2014, they first removed diseased tissue on her retina that was gradually destroying her sight. They then inserted the small patch of cells they had created, hoping they would become a part of her eye and stop her eyesight from degenerating.

Now the results are in. Published today, they show that the treatment hasnt made the womans vision any sharper, but it does seem to have prevented further deterioration with her vision now stable for more than two years. Since the graft, the woman says her vision is brighter.

Takahashi and her team have done incredible work, and deserve all the praise they get for this project, says , director of the Center for iPS Cell Research and Application at Kyoto University, who won the Nobel prize for and collaborated on this work. This is a landmark study and opens the door to similar treatments for many diseases, he says.

This first iPSC-derived retinal graft is an important landmark in the field of retinal regeneration, says at University College London, and head of a trial at Moorfields Eye Hospital in London of similar grafts made instead from human embryonic stem cells.

One worry about this approach is that turning the stem cells into new tissues could lead to cancer-causing genetic mutations though the team found no evidence of this in the treated woman. However, a trial of the technique in another person was cancelled in 2015, after tests revealed that the cells intended to be given to the man had developed genetic abnormalities.

But although it has taken many years to bring , many private centres around the world have been advertising unregulated treatments purporting to use stem cells for some time.

A second study published today shows just how badly some unregulated treatments described as stem cell therapies can go wrong. Three case reports of women given such treatments for age-related macular degeneration detail how one woman went blind and the vision of the other two became much worse.

All three ended up seeking emergency treatment in 2015, after each paid $5000 to a private clinic to receive injections of their own fatty tissue into their eyes.

Patients and physicians in the US should be made aware that not all stem cell clinics are safe, and that stem therapy as provided in private clinics in the US is unproven and potentially harmful, says at the University of Miamis Bascom Palmer Eye Institute, Florida, who subsequently treated two of the women.

Albini advises people to be suspicious of any procedure involving payment. Most legitimate research in the US does not require patients to pay for the experimental procedures, he says, adding that people should check whether a trial has been registered with the US Food and Drug Administration. Be aware that if it sounds too good to be true, it may indeed not be true.

Journal reference: New England Journal of Medicine, DOI: ;

:

More on these topics:

Continue reading here:
Vision saved by first induced pluripotent stem cell treatment - Stock Daily Dish

IPS Cell Therapy Comments Off on Vision saved by first induced pluripotent stem cell treatment – Stock Daily Dish | December 12th, 2019

The idea of a cell therapy for Parkinsons disease starts out simple: Symptoms of the progressive disease are largely driven by the deaths of dopamine-producing neurons found deep within the brain. With lower levels of the neurotransmitter come the characteristic tremors, rigidity and slow movements.

By replacing those lost nerve cells with new dopamine producers, researchers hope to renew the brains connection to the bodys muscles and improve a persons overall motor function.

But in the brain, everything becomes more complicated. On top of the risk of immune system rejection that comes with any kind of living tissue transplant, its important to make sure the implanted cells function correctly and do not pick up any dangerous genetic mutations as they grow.

How ICON, Lotus, and Bioforum are Improving Study Efficiency with a Modern EDC

CROs are often at the forefront of adopting new technologies to make clinical trials more efficient. Hear how ICON, Lotus Clinical Research, and Bioforum are speeding database builds and automating reporting tasks for data management.

Now, a new company, Aspen Neuroscience, aims to tackle both obstacles at once.

First, the startup hopes to avoid any harmful immune reactions by using a patients own cells as a starting point. Then, Aspen plans to implement a rigorous quality control program employing whole genome sequencing and artificial intelligence to make sure the cells stay in line as theyre processed and readied for the procedure.

And to do it, the San Diego-based company is starting out with $6.5 million in seed money plus an impressive roster of names.

They are led by neurology researcher Howard Federoff, previously vice chancellor for health affairs and CEO of the University of California, Irvine health system as well as the executive dean of medicine at Georgetown University. Hes joined by Aspen co-founder and stem cell scientist Jeanne Loring, founding director and professor emeritus of the Center for Regenerative Medicine at the Scripps Research Institute.

Meanwhile, the seed round was led by Domain Associates and Axon Ventures with additional backing from Alexandria Venture Investments, Arch Venture Partners, OrbiMed and Section 32.

Aspen looks to combine its expertise in stem cell biology, genomics and neurology to offer the first autologous cell therapy for Parkinsons diseasewhile others in the space have pursued allogeneic routes, or therapies derived from donors other than the patient.

The process starts with a culture of the patients skin cells, which are then genetically induced to become pluripotent stem cellsor cells capable of differentiating into any other cell type in the body. These are then chemically nudged further to transform into precursor versions of the dopamine-producing neurons, which are typically found in the midbrain and regions responsible for the movement of limbs.

We can say without any equivocation that we can produce the population of cells necessary to transplant, and in a short enough period of time to have a potential beneficial impact on the evolution of the disease, said Federoff, who has also served as chair of the NIHs Recombinant DNA Advisory Committee and helped lead the U.S. Parkinsons Disease Gene Therapy Study Group.

We envisage that this will set back the clock on patients who have Parkinsons, unlike any other therapy that we know of, he told FierceMedTech in an interview.

The number of cells needed would be much smaller compared to other cell therapies and cancer treatments. The healthy human brain contains only about 200,000 dopamine-producing nerve cells, split between its two hemispheres, while patients with Parkinsons disease have lost about 50% or more of those neurons.

Aspen aims to evaluate two doses: one that aims to replace about 60% to 65% of a persons normal cell complement and another larger treatment, Federoff said.

Those smaller doses, as well as starting with a patients donor cells, help make the treatment safer to produce by requiring fewer steps. Each cycle of cell division and multiplication to increase their numbers carries the risk of introducing genetic mutations.

As the cells are grown, they are consistently evaluated with data-driven techniques pioneered by Lorings laboratory. Using whole genome RNA sequencing, Aspen will match the cells up at every stage with a genetic barcode taken from each patient at the start. This will allow them to look for changes, duplications or deletions in the pluripotent stem cell genome.

If the cells harbor mutations that are cancer drivers, we don't want to put those into people, Loring said. The only way is to check the sequencing before we transplant them.

The cells used in the transplant procedure arent fully grown; as neuron progenitors, they mimic the development steps seen in the brain of a growing fetus after theyre placed in the body as they wire themselves up to other neural structures and begin to form new networks of their own.

We anticipate that they will manufacture and release dopamine in a manner that is consistent with synaptic neurotransmission and the process of communicating from cell-to-cell, said Federoff. They will take up dopamine from synapses when it has done its business, bring it back into the cell, and prepare it for another synaptic release.

These are not just dopamine pumps, theyre real neurons, added Loring. They will genuinely replace the cells that have been lost in every way.

Aspen plans to pursue two courses of therapy, for the two major types of Parkinsons disease. Their lead candidate is for idiopathic, or sporadic Parkinsons, while their second is a CRISPR-edited version of the therapy designed to address one of the diseases most common genetic mutations, linked to about 5% of cases.

This would not only aim to restart dopamine production in this orphan indication, but also restore the damaged enzyme GBA, which is seen as an underlying cause. Federoff and Loring expect their sequencing-based quality check system will also help catch any off-target edits linked to the use of CRISPR-Cas9.

The company has yet to secure permission from the FDA to officially launch clinical trials, but the agency has signed off on Aspens plans to prepare a trial-ready cohort of Parkinsons disease patients in the meantime. This would include the initial stages of recruitment and testing, including the selection of patients capable of having their skin cells made into pluripotent stem cells.

After it receives its go-ahead from the FDA, Aspen plans to hit the ground running,enrolling at least 176 participants in a phase 1/2 study that includes a randomized stage to determine clinical benefits.

Read the original here:
By turning stem cells into brain cells, Aspen Neuroscience hopes to rewind the progress of Parkinson's disease - FierceBiotech

Skin Stem Cells Comments Off on By turning stem cells into brain cells, Aspen Neuroscience hopes to rewind the progress of Parkinson’s disease – FierceBiotech | December 12th, 2019

A Campus meeting in November reviewed the arguments for and against donor conception, and the sometimes difficult ethical arguments raised by the prospect of a donor-conceived child. 'Artificial' sperm cells derived from testicular tissue or stem cells may resolve some of those arguments.

The problem is especially acute in cancers diagnosed in prepubertal boys in whom there are no sperm cells available for storage. Their only option for future fatherhood in the face of cancer treatment is adoption or donor sperm. And this, added Goossens, is not an exceptional problem. Incidence rates are around 17 cases per 100,000 population, with leukemia and CNS tumours the most commonly diagnosed. So the usual pathway to fertility preservation in these young cases is for the oncologist to warn of the risk to future fertility from the cancer treatments and refer to the fertility clinic. Biopsy of testicular tissue, of course, must be performed before any radio- or chemotherapy.

Goossens described two experimental techniques, spermatogonial stem cell retrieval and transplantation, and homotopic tissue grafting. The danger in the former procedure is a risk of introducing malignancy, so banked tissue must be free of malignant contamination. Experiments in mouse-to-mouse models have demonstrated spermatogenesis from tissue grafting, and most recently fully functional conception and delivery in a non-human primate (Grady). Similarly, experiments in mouse models with spermatogonial stem cell transplantation have so far proved efficient, with spontaneous pregnancy already possible.

Of course, the objective of this impressive experimental work is not merely a resolution to the question of genetic continuity in couples faced with third-party donation, but the future fertility and long-term quality of life of so many unfortunate young boys. Advances in cancer treatment have led to the increased survival of all children with cancer, and with it a new imperative for the restoration of their fertility. Not all cancer treatments cause complete testicular damage, but around one-third of children having treatment for pediatric cancers will end up infertile. Following the proof-of-concept study which saw the birth of Grady - in which testicular samples removed from prepubertal monkeys was frozen, thawed and regrafted under scrotal skin - the research group declared that their next logical step, with safety and feasibility apparent, is human trials.

1. Fayomi AP, Peters K, Sukhwani M, et al. Autologous grafting of cryopreserved prepubertal rhesus testis produces sperm and offspring. Science 2019; 363: 1314-1319.

Go here to see the original:
CAMPUS: EGG DONATION - Artificial sperm cells to remove the genetic worries of sperm donation - ESHRE

Skin Stem Cells Comments Off on CAMPUS: EGG DONATION – Artificial sperm cells to remove the genetic worries of sperm donation – ESHRE | December 12th, 2019

MELVILLE, N.Y., Dec. 12, 2019 (GLOBE NEWSWIRE) -- BioRestorative Therapies, Inc. (BioRestorative or the Company) (BRTX), a life sciences company focused on stem cell-based therapies, today announced that the Israeli Patent Office has issued BioRestorative a Notice of Allowance on its patent application for a method of generating brown fat stem cells. This is the eighth patent issued, in the United States and other countries, for the Companys brown fat technology related to BioRestoratives metabolic program (ThermoStem Program).

Once issued in Israel, the final patent will allow for a method of isolating and differentiating a non-embryonic human brown adipose-derived stem cell into functional human brown adipocytes and a method of identifying compounds that modifies metabolic activity of human brown adipocytes. The technology is applicable for potential therapeutic uses for treating a wide range of degenerative and metabolic disorders, including but not limited to diabetes, obesity, hypertension and cardiac deficiency.

We continue to drive innovative and novel technology focusing on transformative therapies for our brown fat program, said Mark Weinreb, CEO of BioRestorative Therapies. We are pleased to add to our intellectual property library this recently issued patent by the Israeli Patent Office for our metabolic program to help power disruptive ways to treat metabolic disorders.

About BioRestorative Therapies, Inc.

BioRestorative Therapies, Inc. (www.biorestorative.com) develops therapeutic products using cell and tissue protocols, primarily involving adult stem cells. Our two core programs, as described below, relate to the treatment of disc/spine disease and metabolic disorders:

Forward-Looking Statements

This press release contains "forward-looking statements" within the meaning of Section 27A of the Securities Act of 1933, as amended, and Section 21E of the Securities Exchange Act of 1934, as amended, and such forward-looking statements are made pursuant to the safe harbor provisions of the Private Securities Litigation Reform Act of 1995. You are cautioned that such statements are subject to a multitude of risks and uncertainties that could cause future circumstances, events or results to differ materially from those projected in the forward-looking statements as a result of various factors and other risks, including, without limitation, whether the Company will be able to consummate the private placement and the satisfaction of closing conditions related to the private placement and those set forth in the Company's Form 10-K filed with the Securities and Exchange Commission. You should consider these factors in evaluating the forward-looking statements included herein, and not place undue reliance on such statements. The forward-looking statements in this release are made as of the date hereof and the Company undertakes no obligation to update such statements.

CONTACT:Email: ir@biorestorative.com

Read the original:
BioRestorative Therapies Receives Patent in Israel For Its Metabolic Program - Yahoo Finance

Cardiac Stem Cells Comments Off on BioRestorative Therapies Receives Patent in Israel For Its Metabolic Program – Yahoo Finance | December 12th, 2019

KRAS mutations occur in approximately 20% of people with non-small cell lung cancer, and some previous studies have suggested that these mutations are associated with a poorer response to treatment, said Dr. Jonathan Cheng, vice president, oncology clinical research, Merck Research Laboratories. It was therefore encouraging to see in this exploratory analysis that KEYTRUDA monotherapy was associated with a survival benefit in certain patients with metastatic nonsquamous non-small cell lung cancer, regardless of KRAS mutational status.

The objective of the exploratory analysis was to assess the prevalence of KRAS mutations and their association with efficacy in the KEYNOTE-042 trial. Of the 1,274 untreated patients with metastatic nonsquamous NSCLC whose tumors expressed PD-L1 (TPS 1%) enrolled in KEYNOTE-042, 301 patients had KRAS evaluable data (n=232 without any KRAS mutation; n=69 with any KRAS mutation, including n=29 with the KRAS G12C mutation). Tissue tumor mutational burden (tTMB) and KRAS mutational status were determined by whole-exome sequencing (WES) of tumor tissue and matched normal DNA (blood). Patients were randomized 1:1 to receive KEYTRUDA 200 mg intravenously every three weeks (Q3W) (n=637) or investigators choice of chemotherapy (pemetrexed or paclitaxel) (n=637). Treatment continued until progression of disease or unacceptable toxicity. The primary endpoint was OS with a TPS of 50%, 20% and 1%, which were assessed sequentially. The secondary endpoints were PFS and ORR.

Findings from this exploratory analysis showed that KEYTRUDA monotherapy was associated with improved clinical outcomes, regardless of KRAS mutational status, in patients with metastatic nonsquamous NSCLC versus chemotherapy. In this analysis, KEYTRUDA reduced the risk of death by 58% (HR=0.42 [95% CI, 0.22-0.81]) in patients with any KRAS mutation and by 72% (HR=0.28 [95% CI, 0.09-0.86]) in patients with the KRAS G12C mutation compared to chemotherapy. The safety profile of KEYTRUDA was consistent with what has been seen in previously reported studies among patients with metastatic NSCLC.

Additional efficacy results from this exploratory analysis showed:

With Any KRAS Mutation

With KRAS G12CMutation

Without Any KRAS Mutation

KEYTRUDA Mono-therapy

(N = 30)

Chemo-therapy

(N = 39)

KEYTRUDA Mono-therapy(N = 12)

Chemo-therapy(N = 17)

KEYTRUDA Mono-therapy

(N = 127)

Chemo-therapy(N = 105)

OS, median, mo(95% CI)

28 (23-NR)

11 (7-25)

NR (23-NR)

8 (5-NR)

15 (12-24)

12 (11-18)

OS, HR(95% CI)

0.42 (0.22-0.81)

0.28 (0.09-0.86)

0.86 (0.63-1.18)

ORR, %(95% CI)

56.7

18.0

66.7

23.5

29.1

21.0

PFS, median, mo(95% CI)

12 (8-NR)

6 (4-9)

15 (10-NR)

6 (4-8)

6 (4-7)

6 (6-8)

PFS, HR(95% CI)

0.51 (0.29-0.87)

0.27 (0.10-0.71)

1.00 (0.75-1.34)

Data from an exploratory analysis of KEYNOTE-189 (Abstract #LBA5), which evaluated KRAS mutations and their association with efficacy outcomes for KEYTRUDA in combination with pemetrexed and platinum chemotherapy, were also presented in a mini-oral session today at the ESMO Immuno-Oncology Congress 2019. KEYNOTE-189 was conducted in collaboration with Eli Lilly and Company, the makers of pemetrexed (ALIMTA).

About Lung Cancer

Lung cancer, which forms in the tissues of the lungs, usually within cells lining the air passages, is the leading cause of cancer death worldwide. Each year, more people die of lung cancer than die of colon and breast cancers combined. The two main types of lung cancer are non-small cell and small cell. Non-small cell lung cancer (NSCLC) is the most common type of lung cancer, accounting for about 85% of all cases. Small cell lung cancer (SCLC) accounts for about 10 to 15% of all lung cancers. Lung cancer can also be characterized by the presence of different biomarkers, including PD-L1, KRAS, ALK, EGFR and ROS1. KRAS mutations occur in about 20% of NSCLC cases. Between 2008 and 2014, the five-year survival rate for patients diagnosed in the U.S. with advanced NSCLC was only 5%.

About KEYTRUDA (pembrolizumab) Injection, 100mg

KEYTRUDA is an anti-PD-1 therapy that works by increasing the ability of the bodys immune system to help detect and fight tumor cells. KEYTRUDA is a humanized monoclonal antibody that blocks the interaction between PD-1 and its ligands, PD-L1 and PD-L2, thereby activating T lymphocytes which may affect both tumor cells and healthy cells.

Merck has the industrys largest immuno-oncology clinical research program. There are currently more than 1,000 trials studying KEYTRUDA across a wide variety of cancers and treatment settings. The KEYTRUDA clinical program seeks to understand the role of KEYTRUDA across cancers and the factors that may predict a patients likelihood of benefitting from treatment with KEYTRUDA, including exploring several different biomarkers.

Selected KEYTRUDA (pembrolizumab) Indications

Melanoma

KEYTRUDA is indicated for the treatment of patients with unresectable or metastatic melanoma.

KEYTRUDA is indicated for the adjuvant treatment of patients with melanoma with involvement of lymph node(s) following complete resection.

Non-Small Cell Lung Cancer

KEYTRUDA, in combination with pemetrexed and platinum chemotherapy, is indicated for the first-line treatment of patients with metastatic nonsquamous non-small cell lung cancer (NSCLC), with no EGFR or ALK genomic tumor aberrations.

KEYTRUDA, in combination with carboplatin and either paclitaxel or paclitaxel protein-bound, is indicated for the first-line treatment of patients with metastatic squamous NSCLC.

KEYTRUDA, as a single agent, is indicated for the first-line treatment of patients with NSCLC expressing PD-L1 [tumor proportion score (TPS) 1%] as determined by an FDA-approved test, with no EGFR or ALK genomic tumor aberrations, and is stage III where patients are not candidates for surgical resection or definitive chemoradiation, or metastatic.

KEYTRUDA, as a single agent, is indicated for the treatment of patients with metastatic NSCLC whose tumors express PD-L1 (TPS 1%) as determined by an FDA-approved test, with disease progression on or after platinum-containing chemotherapy. Patients with EGFR or ALK genomic tumor aberrations should have disease progression on FDA-approved therapy for these aberrations prior to receiving KEYTRUDA.

Small Cell Lung Cancer

KEYTRUDA is indicated for the treatment of patients with metastatic small cell lung cancer (SCLC) with disease progression on or after platinum-based chemotherapy and at least one other prior line of therapy. This indication is approved under accelerated approval based on tumor response rate and durability of response. Continued approval for this indication may be contingent upon verification and description of clinical benefit in confirmatory trials.

Head and Neck Squamous Cell Cancer

KEYTRUDA, in combination with platinum and fluorouracil (FU), is indicated for the first-line treatment of patients with metastatic or with unresectable, recurrent head and neck squamous cell carcinoma (HNSCC).

KEYTRUDA, as a single agent, is indicated for the first-line treatment of patients with metastatic or with unresectable, recurrent HNSCC whose tumors express PD-L1 [combined positive score (CPS) 1] as determined by an FDA-approved test.

KEYTRUDA, as a single agent, is indicated for the treatment of patients with recurrent or metastatic HNSCC with disease progression on or after platinum-containing chemotherapy.

Classical Hodgkin Lymphoma

KEYTRUDA is indicated for the treatment of adult and pediatric patients with refractory classical Hodgkin lymphoma (cHL), or who have relapsed after 3 or more prior lines of therapy. This indication is approved under accelerated approval based on tumor response rate and durability of response. Continued approval for this indication may be contingent upon verification and description of clinical benefit in the confirmatory trials.

Primary Mediastinal Large B-Cell Lymphoma

KEYTRUDA is indicated for the treatment of adult and pediatric patients with refractory primary mediastinal large B-cell lymphoma (PMBCL), or who have relapsed after 2 or more prior lines of therapy. This indication is approved under accelerated approval based on tumor response rate and durability of response. Continued approval for this indication may be contingent upon verification and description of clinical benefit in confirmatory trials. KEYTRUDA is not recommended for the treatment of patients with PMBCL who require urgent cytoreductive therapy.

Urothelial Carcinoma

KEYTRUDA is indicated for the treatment of patients with locally advanced or metastatic urothelial carcinoma (mUC) who are not eligible for cisplatin-containing chemotherapy and whose tumors express PD-L1 [CPS 10] as determined by an FDA-approved test, or in patients who are not eligible for any platinum-containing chemotherapy regardless of PD-L1 status. This indication is approved under accelerated approval based on tumor response rate and duration of response. Continued approval for this indication may be contingent upon verification and description of clinical benefit in the confirmatory trials.

KEYTRUDA is indicated for the treatment of patients with locally advanced or metastatic urothelial carcinoma (mUC) who have disease progression during or following platinum-containing chemotherapy or within 12 months of neoadjuvant or adjuvant treatment with platinum-containing chemotherapy.

Microsatellite Instability-High (MSI-H) Cancer

KEYTRUDA is indicated for the treatment of adult and pediatric patients with unresectable or metastatic microsatellite instability-high (MSI-H) or mismatch repair deficient (dMMR).

This indication is approved under accelerated approval based on tumor response rate and durability of response. Continued approval for this indication may be contingent upon verification and description of clinical benefit in the confirmatory trials. The safety and effectiveness of KEYTRUDA in pediatric patients with MSI-H central nervous system cancers have not been established.

Gastric Cancer

KEYTRUDA is indicated for the treatment of patients with recurrent locally advanced or metastatic gastric or gastroesophageal junction (GEJ) adenocarcinoma whose tumors express PD-L1 (CPS 1) as determined by an FDA-approved test, with disease progression on or after two or more prior lines of therapy including fluoropyrimidine- and platinum-containing chemotherapy and if appropriate, HER2/neu-targeted therapy. This indication is approved under accelerated approval based on tumor response rate and durability of response. Continued approval for this indication may be contingent upon verification and description of clinical benefit in the confirmatory trials.

Esophageal Cancer

KEYTRUDA is indicated for the treatment of patients with recurrent locally advanced or metastatic squamous cell carcinoma of the esophagus whose tumors express PD-L1 (CPS 10) as determined by an FDA-approved test, with disease progression after one or more prior lines of systemic therapy.

Cervical Cancer

KEYTRUDA is indicated for the treatment of patients with recurrent or metastatic cervical cancer with disease progression on or after chemotherapy whose tumors express PD-L1 (CPS 1) as determined by an FDA-approved test. This indication is approved under accelerated approval based on tumor response rate and durability of response. Continued approval for this indication may be contingent upon verification and description of clinical benefit in the confirmatory trials.

Hepatocellular Carcinoma

KEYTRUDA is indicated for the treatment of patients with hepatocellular carcinoma (HCC) who have been previously treated with sorafenib. This indication is approved under accelerated approval based on tumor response rate and durability of response. Continued approval for this indication may be contingent upon verification and description of clinical benefit in the confirmatory trials.

Merkel Cell Carcinoma

KEYTRUDA is indicated for the treatment of adult and pediatric patients with recurrent locally advanced or metastatic Merkel cell carcinoma. This indication is approved under accelerated approval based on tumor response rate and durability of response. Continued approval for this indication may be contingent upon verification and description of clinical benefit in the confirmatory trials.

Renal Cell Carcinoma

KEYTRUDA, in combination with axitinib, is indicated for the first-line treatment of patients with advanced renal cell carcinoma (RCC).

Selected Important Safety Information for KEYTRUDA

Immune-Mediated Pneumonitis

KEYTRUDA can cause immune-mediated pneumonitis, including fatal cases. Pneumonitis occurred in 3.4% (94/2799) of patients with various cancers receiving KEYTRUDA, including Grade 1 (0.8%), 2 (1.3%), 3 (0.9%), 4 (0.3%), and 5 (0.1%). Pneumonitis occurred in 8.2% (65/790) of NSCLC patients receiving KEYTRUDA as a single agent, including Grades 3-4 in 3.2% of patients, and occurred more frequently in patients with a history of prior thoracic radiation (17%) compared to those without (7.7%). Pneumonitis occurred in 6% (18/300) of HNSCC patients receiving KEYTRUDA as a single agent, including Grades 3-5 in 1.6% of patients, and occurred in 5.4% (15/276) of patients receiving KEYTRUDA in combination with platinum and FU as first-line therapy for advanced disease, including Grade 3-5 in 1.5% of patients.

Monitor patients for signs and symptoms of pneumonitis. Evaluate suspected pneumonitis with radiographic imaging. Administer corticosteroids for Grade 2 or greater pneumonitis. Withhold KEYTRUDA for Grade 2; permanently discontinue KEYTRUDA for Grade 3 or 4 or recurrent Grade 2 pneumonitis.

Immune-Mediated Colitis

KEYTRUDA can cause immune-mediated colitis. Colitis occurred in 1.7% (48/2799) of patients receiving KEYTRUDA, including Grade 2 (0.4%), 3 (1.1%), and 4 (<0.1%). Monitor patients for signs and symptoms of colitis. Administer corticosteroids for Grade 2 or greater colitis. Withhold KEYTRUDA for Grade 2 or 3; permanently discontinue KEYTRUDA for Grade 4 colitis.

Immune-Mediated Hepatitis (KEYTRUDA) and Hepatotoxicity (KEYTRUDA in Combination With Axitinib)

Immune-Mediated Hepatitis

The rest is here:
Data from Exploratory Analysis Show Merck's KEYTRUDA (pembrolizumab) Improved Overall Survival as Monotherapy for the First-Line Treatment of...

Cardiac Stem Cells Comments Off on Data from Exploratory Analysis Show Merck’s KEYTRUDA (pembrolizumab) Improved Overall Survival as Monotherapy for the First-Line Treatment of… | December 12th, 2019

Transparency Market Research (TMR)has published a new report on the globalcell separation technology marketfor the forecast period of 20192027. According to the report, the global cell separation technology market was valued at ~US$ 5 Bnin 2018, and is projected to expand at a double-digit CAGR during the forecast period.

Overview

Cell separation, also known as cell sorting or cell isolation, is the process of removing cells from biological samples such as tissue or whole blood. Cell separation is a powerful technology that assists biological research. Rising incidences of chronic illnesses across the globe are likely to boost the development of regenerative medicines or tissue engineering, which further boosts the adoption of cell separation technologies by researchers.

Request PDF Sample of Cell Separation Technology Market Report @https://www.transparencymarketresearch.com/sample/sample.php?flag=S&rep_id=1925

Expansion of the global cell separation technology market is attributed to an increase in technological advancements and surge in investments in research & development, such asstem cellresearch and cancer research. The rising geriatric population is another factor boosting the need for cell separation technologies Moreover, the geriatric population, globally, is more prone to long-term neurological and other chronic illnesses, which, in turn, is driving research to develop treatment for chronic illnesses. Furthermore, increase in the awareness about innovative technologies, such as microfluidics, fluorescent-activated cells sorting, and magnetic activated cells sorting is expected to propel the global cell separation technology market.

North America dominated the global cell separation technology market in 2018, and the trend is anticipated to continue during the forecast period. This is attributed to technological advancements in offering cell separation solutions, presence of key players, and increased initiatives by governments for advancing the cell separation process. However, insufficient funding for the development of cell separation technologies is likely to hamper the global cell separation technology market during the forecast period. Asia Pacific is expected to be a highly lucrative market for cell separation technology during the forecast period, owing to improving healthcare infrastructure along with rising investments in research & development in the region.

Enquiry for Discount on Cell Separation Technology Market Report @https://www.transparencymarketresearch.com/sample/sample.php?flag=D&rep_id=1925

Rising Incidences of Chronic Diseases, Worldwide, Boosting the Demand for Cell Therapy

Incidences of chronic diseases such as diabetes, obesity, arthritis, cardiac diseases, and cancer are increasing due to sedentary lifestyles, aging population, and increased alcohol consumption and cigarette smoking. According to the World Health Organization (WHO), by 2020, the mortality rate from chronic diseases is expected to reach73%, and in developing counties,70%deaths are estimated to be caused by chronic diseases. Southeast Asia, Eastern Mediterranean, and Africa are expected to be greatly affected by chronic diseases. Thus, the increasing burden of chronic diseases around the world is fuelling the demand for cellular therapies to treat chronic diseases. This, in turn, is driving focus and investments on research to develop effective treatments. Thus, increase in cellular research activities is boosting the global cell separation technology market.

Increase in Geriatric Population Boosting the Demand for Surgeries

The geriatric population is likely to suffer from chronic diseases such as cancer and neurological disorders more than the younger population. Moreover, the geriatric population is increasing at a rapid pace as compared to that of the younger population. Increase in the geriatric population aged above 65 years is projected to drive the incidences of Alzheimers, dementia, cancer, and immune diseases, which, in turn, is anticipated to boost the need for corrective treatment of these disorders. This is estimated to further drive the demand for clinical trials and research that require cell separation products. These factors are likely to boost the global cell separation technology market.

According to the United Nations, the geriatric population aged above 60 is expected to double by 2050 and triple by 2100, an increase from962 millionin 2017 to2.1 billionin 2050 and3.1 billionby 2100.

Productive Partnerships in Microfluidics Likely to Boost the Cell Separation Technology Market

Technological advancements are prompting companies to innovate in microfluidics cell separation technology. Strategic partnerships and collaborations is an ongoing trend, which is boosting the innovation and development of microfluidics-based products. Governments and stakeholders look upon the potential in single cell separation technology and its analysis, which drives them to invest in the development ofmicrofluidics. Companies are striving to build a platform by utilizing their expertise and experience to further offer enhanced solutions to end users.

Stem Cell Research to Account for a Prominent Share

Stem cell is a prominent cell therapy utilized in the development of regenerative medicine, which is employed in the replacement of tissues or organs, rather than treating them. Thus, stem cell accounted for a prominent share of the global market. The geriatric population is likely to increase at a rapid pace as compared to the adult population, by 2030, which is likely to attract the use of stem cell therapy for treatment. Stem cells require considerably higher number of clinical trials, which is likely to drive the demand for cell separation technology, globally. Rising stem cell research is likely to attract government and private funding, which, in turn, is estimated to offer significant opportunity for stem cell therapies.

Biotechnology & Pharmaceuticals Companies to Dominate the Market

The number of biotechnology companies operating across the globe is rising, especially in developing countries. Pharmaceutical companies are likely to use cells separation techniques to develop drugs and continue contributing through innovation. Growing research in stem cell has prompted companies to own large separate units to boost the same. Thus, advancements in developing drugs and treatments, such as CAR-T through cell separation technologies, are likely to drive the segment.

As per research, 449 public biotech companies operate in the U.S., which is expected to boost the biotechnology & pharmaceutical companies segment. In developing countries such as China, China Food and Drug Administration(CFDA) reforms pave the way for innovation to further boost biotechnology & pharmaceutical companies in the country.

Global Cell Separation Technology Market: Prominent Regions

North America to Dominate Global Market, While Asia Pacific to Offer Significant Opportunity

In terms of region, the global cell separation technology market has been segmented into five major regions: North America, Europe, Asia Pacific, Latin America, and the Middle East & Africa. North America dominated the global market in 2018, followed by Europe. North America accounted for a major share of the global cell separation technology market in 2018, owing to the development of cell separation advanced technologies, well-defined regulatory framework, and initiatives by governments in the region to further encourage the research industry. The U.S. is a major investor in stem cell research, which accelerates the development of regenerative medicines for the treatment of various long-term illnesses.

The cell separation technology market in Asia Pacific is projected to expand at a high CAGR from 2019 to 2027. This can be attributed to an increase in healthcare expenditure and large patient population, especially in countries such as India and China. Rising medical tourism in the region and technological advancements are likely to drive the cell separation technology market in the region.

Launching Innovative Products, and Acquisitions & Collaborations by Key Players Driving Global Cell Separation Technology Market

The global cell separation technology market is highly competitive in terms of number of players. Key players operating in the global cell separation technology market include Akadeum Life Sciences, STEMCELL Technologies, Inc., BD, Bio-Rad Laboratories, Inc., Miltenyi Biotech, 10X Genomics, Thermo Fisher Scientific, Inc., Zeiss, GE Healthcare Life Sciences, PerkinElmer, Inc., and QIAGEN.

These players have adopted various strategies such as expanding their product portfolios by launching new cell separation kits and devices, and participation in acquisitions, establishing strong distribution networks. Companies are expanding their geographic presence in order sustain in the global cell separation technology market. For instance, in May 2019, Akadeum Life Sciences launched seven new microbubble-based products at a conference. In July 2017, BD received the U.S. FDAs clearance for its BD FACS Lyric flow cytometer system, which is used in the diagnosis of immunological disorders.

Continued here:
Cell Separation Technology Market : Industry Overview, Trends and Growth Opportunities Forecasted Till 2027 - VaporBlash

Cardiac Stem Cells Comments Off on Cell Separation Technology Market : Industry Overview, Trends and Growth Opportunities Forecasted Till 2027 – VaporBlash | December 12th, 2019

How do you know if your skin is dry? For starters, it feels rough to the touch and, in extreme cases, it may start flaking and you may even feel a tearing sensation.

The good news is that dryskin can be easily avoided, and the most efficient way to hydrate parched skin is with a hydrating sheet mask. All it takes is 15 to 20 minutes and youre on your way to plumped, glowing skin.

These days, hydration sheet masks are loaded with so much extra goodness vitamins, minerals, amino acids,organic acids, plant stem cells andpeptidesthat gointo the formulas that these can also help fight wrinkles, eliminate dark spots and lighten skin tone.

CHARLOTTE TILBURY INSTANT MAGIC FACIAL DRY SHEET MASK, S$76

Yes, this is a dry mask that you can get at Sephora. The magic all comes from the warmth of your skin: The bio-mimetic vector delivery system turns a combo of ingredients (crocus bulb extract, plant stem cells, peptides and vitamin B3) from solid to liquid.

Wear the dry mask by looping the hoops over each ear then simply activate the formula by massaging upwards to move the mask into place. After 15 minutes, take it off then gently tap the remaining essence into the skin. Youll soon notice a glow that is similar to the one you get after a super shiok facial.

But dont throw away the mask just yet the dry formula is engineered for reuse. In fact, you can use it three more times. Just slip it back (the formula side facing inward) into the resealable foil pouch for when you need it next. So really, thats four masks for the price of one.

UTENA PURESA SHEET MASK HYALURONIC ACID 5S, S$12.90

You can pick this up at Watsons. There are five masks to a pack and each one combines the benefits of hyaluronic acid and royal jelly extract, infused with a gel-like essence that works to hydrate skin like a jelly mask. To make the most of it, first smoothen the jelly bits onto the face before placing the sheet over the face.

But theres more: Peek into the packaging and youll see lots of jelly bits left. Apply these remaining bits anywhere else you want to hydrate. We personally like to spread it over the neck, the back of the palms and even the elbows. Yes, it also works as a body hydration gel.

SK II FACIAL TREATMENT MASK, S$127

Love SK IIs Facial Treatment Essence? Then youll love this sheet mask because its drenched with so much Facial Treatment Essence that it feels as if youve dunked your face into a tub of Pitera.

It contains 50 micro-nutrients like vitamins, minerals, amino acids and organic acids to condition skin's natural functions. Twenty minutes is all it takes to rehydrate, clarify complexion and have crystal clear skin.

LA MER THE TREATMENT LOTION HYDRATING MASK, S$45

Each sheet mask is infused with a full ounce of La Mers liquid energy skin hydrator and its equipped with Japanese skin-hugging technology that delivers a concentrated surge of healing hydration directly onto the skin to nourish and soften fine lines.

STARSKIN RED CARPET READY HYDRATING BIO-CELLULOSE SECOND SKIN FACE MASK, S$18

Another mask you can pick up at Sephora is one with a difference. Instead of prepping your face, you prep the mask massage the still-closed sachet to distribute the serum liquid evenly before opening.

The exclusive Bio Cellulose sheet a thin biodegradable microfibre is infused with a delicious cocktail of coconut juice, Amino acids and brown algae that work together to strengthen the skin barrier and promote moisture retention. Dull and dehydrated skin doesnt stand a chance.

More here:
Got dry skin but no time for a facial? These hydration sheet masks are just as good - CNA

Skin Stem Cells Comments Off on Got dry skin but no time for a facial? These hydration sheet masks are just as good – CNA | December 12th, 2019

Materials science company W. L. Gore & Associates Inc. (Newark, DE) has announced a joint venture with the Mayo Clinic (Rochester, MN) to further develop a therapeutic system using stem cells and bio-absorbable scaffolds to treat a condition affecting patients with Crohns disease. Avobis Bio, based in Delaware, will draw on the expertise of scientists and medical professionals from both organizations to build on the encouraging results of an investigational treatment for perianal fistulas.

A debilitating condition that affects patients with Crohns disease, perianal fistulas are painful tunneling wounds connecting the anus to the skin, explained Gore in a press release. Few healing options exist, and patients endure multiple surgeries and ongoing risk of life-threatening complications, said Gore.

"Perianal fistulas are truly life-altering for Crohn's patients, and treatment options have eluded gastroenterologists and surgeons for years," added William Faubion Jr., MD, a Mayo Clinic gastroenterologist who specializes in inflammatory bowel diseases.

The innovative treatment involves harvesting a patients own mesenchymal stem cells, which then are populated on Gore's bioabsorbable polymer scaffold and surgically implanted in the fistula. A phase I clinical trial showed that 76% of patients healed within a year. If this outcome is validated in a larger trial, Gore said that it would dramatically exceed outcomes achieved with existing treatments.

This project is the initial focus of Avobis Bio, which describes its overall mission as an exploration of the use of mesenchymal stem cells combined with enabling bioabsorbable scaffolds that enhance the effectiveness of the cells in stimulating the body to heal.

Delivering a patients mesenchymal stem cells on a synthetic scaffold that biodegrades over time may be a first-of-its-kind medical therapy, noted Joe Carlson, a reporter at the Minneapolis-based Star Tribune reporting on the joint venture. If successful, Avobis Bio may one day offer a variety of tissue and organ-repair therapies combining Mayo's stem cell expertise and Gore's medical materials, he wrote.

Gore is perhaps best known to the public for its Gore-Tex outerwear, but the privately held $3.7 billion engineering and manufacturing firm sells products in an array of industries, including a line of medical devices designed to repair nonnatural holes in body organs, added Carlson. Mayo has used Gore-made devices for many years.

Original post:
Gore, Mayo Clinic team up to deliver breakthrough medical therapies - Plastics Today

Skin Stem Cells Comments Off on Gore, Mayo Clinic team up to deliver breakthrough medical therapies – Plastics Today | December 12th, 2019

This article highlights some of the most recent drug target discoveries that could be used to develop and design a treatment for pancreatic cancer.

Scientists investigating pancreatic cancer have identified new targets which, with further research, could be the basis for developing future therapies. Listed below are five of the most recent target discoveries, in order of their journal publication dates, with the newest first.

Scientists at the Queen Mary University of London, UK and Zhengzhou University, China have developed a personalised vaccine system that may be able to delay the onset of pancreatic cancer.

Cells taken from mice, mutated chemically into pancreatic cancer cells and then infected with Adenovirus (AdV) as a prime or Vaccinia virus (VV) as a boost, create a vaccine product. The virus kills the cancerous cells in such a way that their antigens are released and are therefore able to prime the immune system to prevent pancreatic cancer returning.

Injection of the virus-infected cells into mice destined to develop pancreatic cancer doubled their survival rate, compared to their unvaccinated counterparts. The vaccine also delayed the onset of the condition in these mice.

Using cells from the recipient of the vaccine enables the immune system to respond to the exact antigens seen in tumour cells of the individual, resulting in a vaccine regime tailored to them.

Through this international collaboration, we have made progress towards the development of a prophylactic cancer vaccine against pancreatic cancer, said Professor Yaohe Wang, leader of the study, from Queen Mary University of London and the Sino-British Research Centre at Zhengzhou University in China.

Researchers at Sanford Burnham Prebys Medical Discovery Institute in the US have identified that a combination of two anti-cancer compounds, already approved for use to treat other cancers, shrank pancreatic tumours in mice.

Our study identifies a potential treatment combination that can immediately be tested against these aggressive tumours. We are already meeting with oncologists at Oregon Health & Science University, US to discuss how to advance this discovery into clinical evaluation, explained Dr Zeev Ronai, a professor in Sanford Burnham Prebys Tumor Initiation and Maintenance Program, also senior author of the study.

Scientists used L-asparaginase to starve pancreatic tumours of asparagine, an amino acid required by cells for protein synthesis. However, the tumour cells did not die, instead switching on a stress response pathway whereby they could produce asparagine themselves. Scientists then used an MEK inhibitor to block the stress response pathway, causing the pancreatic tumour to shrink.

L-asparaginase is already US Food and Drug Administration (FDA) approved to treat leukaemias and similarly the MEK inhibitor is approved for the treatment of solid tumours, including melanoma skin cancer.

This research lays the basis for the inhibition of pancreatic tumour growth by a combined synergistic attack based on asparagine restriction and MAPK signalling inhibition, says Dr Eytan Ruppin, chief of the Cancer Data Science Library at the National Cancer Institute (NCI) and co-author of the study.

Scientists from the Max Planck Institute for Biology of Ageing, Germany have identified that YME1L, a protease in the membrane of mitochondria, is activated when a cell uses glycolysis to produce energy anaerobically.

scientists were able to reduce tumour growth by switching off the glycolysis signalling pathway in the mitochondria

Cells adapt to oxygen deficiency by switching their energy supply to glycolysis, which ferments sugar without oxygen. This switch is often necessary in old age, as the cells in the body become poorly supplied with oxygen and nutrients.

Cancer cells can also face this problem; prior to angiogenesis, tumours are poorly perfused and so the tissue is deprived of oxygen. Oxidative stress in tumours drives the switch-on of multiple pathways. This includes the glycolysis pathway that alters the behaviour of the mitochondria to provide tumour cells with energy despite being starved of oxygen.

Scientists found that the YME1L protease is activated during the conversion to glycolysis. YME1L appears altered and breaks down various proteins in the organelles, preventing the formation of new mitochondria and causing the remaining organelles to change their metabolism. This process eventually stops as YME1L begins to degrade itself at high activity.

Researchers examined cancer cells originating from patients with pancreatic tumours and were able to reduce tumour growth by switching off the glycolysis signalling pathway in the mitochondria, with reproducible results both in the petri dish and in pancreatic tumours in mice.

There is currently no treatment available for pancreatic cancer. I believe that this protease can be a very interesting therapeutic target because we have seen that the signalling pathway is also active in human patients with pancreatic cancer, explained Thomas Langer, the Max Planck Director, continuing: However, there are no known substances that have an effect on this protease.

Researchers at the Crick Institute have identified cancer stem cells as a driver of pancreatic cancer growth. These cells can metastasise and differentiate into different tumour types to continue the spread of cancer.

Cancer stem cells appear at all stages of cancer growth so being able to identify where they are present could be vital in both targeting cancer and developing new treatments, according to the researchers. Analysis of gene expression in the cancer stem cells identified a protein, CD9, is present on tumour surfaces during development and when it is more established. This protein could therefore be used as a marker to help locate these cells.

A further development of the study established that this protein is not just a marker of cancer stem cells, but also promotes their malignant behaviour. By altering the amount of CD9 in tumour cells in mice, researchers found that reduced levels of this protein caused smaller tumours to form and increasing levels of CD9 created more aggressive cells able to form large tumours quickly.

These cells are vital to pancreatic cancer and if even just a few of them survive chemotherapy, the cancer is able to bounce back. We need to find effective ways to remove these cells and so stop them from fuelling cancer growth. However, we need more experiments to validate the importance of CD9 in human pancreatic cancer, says Victoria Wang, lead author and member of the Adult Stem Cell Laboratory at the Crick Institute.

A look into cancer stem cell metabolism also revealed CD9 increases the rate tumour cells take up glutamine, an amino acid which helps provide energy for cancer growth.

Now we know this protein is both linked to cancer stem cells and helps cancer growth, this could guide the development of new treatments that are targeted at the protein and so cut off the supply of glutamine to cancer stem cells, effectively starving the cancer, says Axel Behrens, corresponding author and group leader in the Adult Stem Cell Laboratory at the Crick Institute.

Scientists at Tel Aviv University, Israel have found that PJ34, a small molecule, causes human pancreatic cancer cells to self-destruct. The researchers tested PJ34 on xenografts (transplants) of human pancreatic tumours in mice.

this mechanism also exists in other types of cancer and therefore the treatment could be valuable for use on those resistant to current therapies

The mice were treated with a molecule called PJ34, which is permeable in the cell membrane but affects human cancer cells exclusively. This molecule causes an anomaly during the duplication of human cancer cells, provoking their rapid cell death. Thus, cell multiplication itself resulted in cell death in the treated cancer cells, explains Professor Malca Cohen-Armon, project lead at Tel Aviv Universitys Sackler Faculty of Medicine.

The treatment consisted of daily PJ34 injections for 14 days and four weeks later there was a relative drop of 90 percent in the number of cancer cells within the tumours of the mice. Cohen-Armon also noted there were no adverse side-effects observed in the mice.

This mechanism similarly exists in other types of cancer and therefore the treatment could be valuable for use on those resistant to current therapies. The molecule PJ34 is being tested in pre-clinical trials according to FDA regulations before clinical trials begin.

The rest is here:
Five recent drug target discoveries for pancreatic cancer - Drug Target Review

Skin Stem Cells Comments Off on Five recent drug target discoveries for pancreatic cancer – Drug Target Review | December 12th, 2019