Category Archives: Stem Cells

News Release

Thursday, August 13, 2020

National Institutes of Health Director Francis S. Collins, M.D., Ph.D., has selected Rena N. DSouza, D.D.S., M.S., Ph.D., as director of NIHs National Institute of Dental and Craniofacial Research (NIDCR). A licensed dentist, Dr. DSouza is currently the assistant vice president for academic affairs and education for health sciences at the University of Utah, Salt Lake City. There she also serves as a professor of dentistry, the Ole and Marty Jensen Chair of the School of Dentistry and professor of neurobiology and anatomy, pathology and surgery in the School of Medicine and the Department of Biomedical Engineering. She is expected to begin her new role as the NIDCR director later this year.

Dr. DSouza is renowned for her research in craniofacial development, genetics, tooth development and regenerative dental medicine. She has worked as a proponent for NIH for decades, serving on critical advisory committees and as an expert consultant on multiple projects, said Dr. Collins. I look forward to having her join the NIH leadership team later thisyear. I also want to thank NIH Principal Deputy Director Lawrence A. Tabak, D.D.S., Ph.D., for his valuable leadership as the acting director of NIDCR since January 1, 2020.

As NIDCR director, Dr. DSouza will oversee the institutes annual budget of over $475 million, which supports basic, translational and clinical research in areas of oral cancer, orofacial pain, tooth decay, periodontal disease, salivary gland dysfunction, craniofacial development and disorders and the oral complications of systemic diseases. The institute funds approximately 770 grants, 6,500 researchers and 200 organizations. Additionally, NIDCR supports research training and career development programs for approximately 350 people at various stages of their careers, from high school students to independent scientists.

Dr. DSouza has been a principal investigator on multiple NIH and other federal grants since 1987 and has published 140 peer-reviewed journal papers and book chapters. Her research focuses on developmental biology and genetics; matrix biology; biomaterials, tissue engineering and stem cells; and clinical research. Her groups discovery that a novel mutation in PAX9 was responsible for a severe form of human tooth agenesis opened a new field of research to discover genes and mutations as well as therapies for common human inherited disorders of the craniofacial complex.

Dr. DSouzas career honors are significant. She was selected to be the inaugural dean of the University of Utahs School of Dentistry, which was established in 2012. She is currently the elected chair in Dentistry and Oral Health Sciences Section and elected as a fellow of the American Association for the Advancement of Science. She also is a former president of the American Association for Dental Research and the International Association for Dental Research,a fellow of the American College of Dentistsand the recipient of the 2017 American Association for Dental Research Irwin D. Mandel Distinguished Mentoring Award. Dr. DSouza served on the NIH Advisory Committee to the Director in 2013-14, and on NIH study sections. She is a devoted mentor and champion of diversity in the biomedical research workforce. Since 1985, she has served as a volunteer dentist for women in need and people struggling with homelessness in Salt Lake City, Dallas and Houston.

Dr. DSouza received her bachelors degree in dental surgery from the University of Bombay, India, after which she completed her general practice residency. She earned her D.D.S., Ph.D. and masters degree in pathology/biomedical sciences from the University of Texas Health Science Center in Houston.

NIDCR is the nations leading funder of research on oral, dental, and craniofacial health. To learn more about NIDCR, visit https://www.nidcr.nih.gov/.

About the National Institutes of Health (NIH):NIH, the nation's medical research agency, includes 27 Institutes and Centers and is a component of the U.S. Department of Health and Human Services. NIH is the primary federal agency conducting and supporting basic, clinical, and translational medical research, and is investigating the causes, treatments, and cures for both common and rare diseases. For more information about NIH and its programs, visit http://www.nih.gov.

NIHTurning Discovery Into Health

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NIH names Dr. Rena D'Souza as director of the National Institute of Dental and Craniofacial Research - National Institutes of Health

Market Study Report, LLC, has added a detailed study on the Circulating Tumor Cells (CTCS) and Cancer Stem Cells (CSCS) market which provides a brief summary of the growth trends influencing the market. The report also includes significant insights pertaining to the profitability graph, market share, regional proliferation and SWOT analysis of this business vertical. The report further illustrates the status of key players in the competitive setting of the Circulating Tumor Cells (CTCS) and Cancer Stem Cells (CSCS) market, while expanding on their corporate strategies and product offerings.

The report on Circulating Tumor Cells (CTCS) and Cancer Stem Cells (CSCS) market covers the key trends of the industry which impact its growth with reference to the competitive arena and key regions. The study highlights the challenges this industry vertical will face along with the growth opportunities which would support the business development in existing & untapped markets. Besides this, the report also includes few case studies including those which take into account the corona virus pandemic, with an intention to offer a clear picture of this business sphere to all stakeholders.

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Global Circulating Tumor Cells (CTCS) and Cancer Stem Cells (CSCS) Market - Detailed Analysis of Current Industry Figures with Forecasts Growth By...

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New drug candidate targets asthma, COPD, other progressive lung diseases

Shown is a section of mouse lung with progressive lung disease, similar to asthma or COPD. The red staining shows excess mucus production that is characteristic of progressive lung disease and can occur in the aftermath of viral infection or other injury to the lungs. Researchers at Washington University School of Medicine in St. Louis have received a grant to develop a drug to block this process, potentially leading to a new treatment for debilitating lung disorders.

Researchers at Washington University School of Medicine in St. Louis have received a $3.9 million development award supporting new technologies and therapeutics to advance a first-in-class drug to treat debilitating lung diseases, including asthma and chronic obstructive pulmonary disease (COPD).

The research, funded by the Department of Defense, is led by Michael J. Holtzman, MD, the Selma and Herman Seldin Professor of Medicine and director of the Division of Pulmonary & Critical Care Medicine at Washington University.

Holtzmans team has designed a new drug candidate that blocks two important signaling systems in the lungs. These systems cause cells of the respiratory tract to become mucus-producing cells. An overabundance of these cells creates too much mucus, which can block breathing. Evidence suggests that the drug could stop and perhaps even reverse progressive damage from overproduction of mucus that is triggered by aggravations to the lung, including from respiratory viruses, smoking and air pollution.

Obstructive lung diseases, such as asthma and COPD, are the third leading cause of death due to disease in the United States, Holtzman said. But we have no effective treatments that address the root causes of these illnesses or halt disease progression. We can only try to relieve symptoms. This grant will allow us to continue research into a new drug candidate that our group developed and that has shown evidence of stopping and correcting what goes wrong in the lungs when this type of disease process is triggered.

In past research, Holtzman and his colleagues identified specialized stem cells in the lungs that give rise to mucus cells. During development, such stem cells are responsible for growing the lung itself. Some of these stem cells remain in the lungs into adulthood and periodically make new cells to line the inner surfaces of lung tissue, including those cells that make mucus. These stem cells also respond to injury to the lung such as a burn injury from inhaling smoke or a severe viral infection and orchestrate the repair process.

Shown is a section of healthy mouse lung.

We need a certain level of mucus to protect the lungs from viruses or particles that can be inhaled, Holtzman said. The stem cell population is important in maintaining normal lung function and in injury situations, where they help with the healing process. But, unfortunately, in some people these cells can go off the rails. Under certain conditions, particularly a severe respiratory viral infection, these stem cells become reprogrammed so that theyre activated even after the injury or infection is resolved. This leads to overproduction of mucus and excessive inflammation that can interfere with lung function with airway obstruction and difficulty breathing.

The new drug candidate is being designed to be taken by mouth or inhalation and to specifically target two related but distinct signaling molecules known as MAP kinases to control both arms of the immune and inflammatory response. Studying human cells, as well as mouse and pig models of respiratory disease following respiratory viral infections, the researchers found that not only does the drug reduce mucus production, it also nudges the rogue stem cells back toward their quieter and healthier state of readiness. The new grant will support studies in human cells, mice and pigs to establish evidence for the safety and effectiveness of the lead drug candidate and to help determine proper doses for subsequent studies in people. The safety work also will be facilitated by a subcontract to the teams biotechnology company known as NuPeak Therapeutics, which is specially designed for this drug development activity. The goal is to gather data to support approval for a first-in-human clinical trial.

Holtzman said the drug does not treat the viral infection itself; rather, it stops what he calls post-viral disease and its progression, which includes asthma and COPD. Post-viral lung disease also could include COVID-19, as an example of another severe respiratory viral infection that causes progressive and in some cases long-term lung disease in some patients well after the infectious virus has been cleared.

SARS-CoV-2, the virus that causes COVID-19, is similar to the viruses Holtzman and colleagues are working with to study their new drug candidate. The group is gathering key information in patient and animal models to determine whether the same therapeutic strategy could prove useful in treating COVID-19 as it has been for lung disease due to other related respiratory viruses.

Were just beginning to learn how the body responds to SARS-CoV-2, but it is very common for any respiratory viral infection, especially severe infections, to trigger this progressive disease process in some percentage of the patients who contract the virus, Holtzman said. Whats interesting is that the infectious form of the virus is gone when this process ramps up. Patients arent fighting the virus any more, theyre fighting their own immune system. In future work, we will be interested in finding out whether our drug candidate can help shut this process down regardless of the trigger, viral or otherwise.

This work is supported by the Department of Defense, grant number PR190726. Holtzman founded the biotechnology company NuPeak Therapeutics, which is facilitating development of the drug candidate.

Washington University School of Medicines 1,500 faculty physicians also are the medical staff of Barnes-Jewish and St. Louis Childrens hospitals. The School of Medicine is a leader in medical research, teaching and patient care, ranking among the top 10 medical schools in the nation by U.S. News & World Report. Through its affiliations with Barnes-Jewish and St. Louis Childrens hospitals, the School of Medicine is linked to BJC HealthCare.

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Drug development for severe respiratory diseases supported with $3.9 million grant - Washington University School of Medicine in St. Louis

The global GMP cell banking services marketis expected to gain stupendous demand avenues in the forthcoming years. This growth is attributed to increasing demand for GMP Cell banking services from the enterprises engaged in the pharmaceutical and stem cell research industries. Cell banks use a conventional technique named cryopreservation to keep the cells materials preserved. At the same time, cell banks also prevent the natural cell division process; thereby improve the shelf life of products preserved in the cell banks.

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One of the important factors owing to which GMP cell banking services are gaining momentum among scientists and research institutes is the cost-effectiveness of these services. In recent years, there is remarkable growth in the use of GMP cell banking services among gamut of research institutes from all across the world. This scenario depicts that the vendors working in the global GMP cell banking services market will witness stupendous demand opportunities in the forthcoming period.

Government Bodies Increase Flow of Funding to Discover Treatment Options for Rare Diseases

In recent years, there is noteworthy increase in the number of people living with various rare diseases. This situation has compelled scientists working in all worldwide locations to focus on the discovery of novel options to treat these health issues. To accelerate these research activities, government bodies of many countries from all over the world are financially supporting these research projects. This factor is positively impacting on the development of the global GMP cell banking services market.

On regional front, players working in the GMP cell banking services market are projected to gain fantastic development opportunities in North America and Asia Pacific. Presence of substantial mammalian cell is said to be one of the key reasons driving the growth of GMP cell banking services market in North America.

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GMP Cell Banking Services Gain Momentum among Stem Cell Research Institutes Due to Cost-effectiveness and Quality They Offer - TMR Research Blog

McMaster University is terminating a high-profile research institute that investigates novel stem cell and cancer therapies.

The loss of the multimillion dollar Stem Cell and Cancer Research Institute (SCC-RI) may extend to its prominent leader as questions remain about whether Mick Bhatia will stay in Hamilton. Two other researchers Kristin Hope and Karun Singh are already leaving for Toronto.

McMaster and Bhatia tell very different stories about how a university board of governors meeting on June 4 came to include the recommendation to end the nationally known institute.

My vision was to have an international presence and supremacy in stem cells, said Bhatia. McMaster, in the end, in my interpretation, really just didnt have the appetite to go in that direction.

The university claims SCC-RI has run its course because its researchers werent collaborating, which is the entire purpose of an institute.

They werent working together, said Jonathan Bramson vice-dean of research for the faculty of health sciences. We put people together because we think they will work together and achieve a situation where the sum is greater than the parts and that wasnt the case here.

Bhatia says he cant possibly compute that explanation, pointing out he has published at least one paper with every researcher at SCC-RI and the other researchers have done the same.

It doesnt make sense, he said. Its like saying the Raptors arent good at basketball.

In fact, it was collaboration that lured Bhatia to McMaster from California in 2006 in the first place. He was working toward leading a stem cell institute there when Dr. John Kelton, who was dean of the faculty of health sciences at the time, made Bhatia believe it would work better back home in Ontario.

I was completely enchanted of the idea that in Canada you could achieve that level of excellence and there was support to build something, he said. It was a great opportunity that I thought couldnt happen here in Canada and yet here it was in front of me.

The institute was set up with $10 million from Michael G. DeGroote and, over time, got another $15 million from David Braley and $24 million from the Boris family.

We were really starting from scratch, said Bhatia. There were no people working deeply in stem cell biology you had to recruit from outside because there is no pre-existing expertise, equipment or infrastructure.

At its height, SCC-RI had 13,000 square feet of state-of-the art facilities, 130 staff and millions in grants including $13 million from the Ontario Research Fund, roughly $10 million from the Canadian Foundation of Innovation and in the last fiscal year alone its scientists were awarded $3.28 million in research grants.

It had findings that were paradigm shifting for stem cell research, collaborated with biochemists which was a first for the field and took potential new cancer drugs into clinical trials.

John Kelton . was pretty visionary, said Bhatia. He was looking for areas to be truly excellent and his definition of excellence was very akin to mine ... It excited me that you could do this level of science and there was like-minded people thinking about that direction.

But at the 10-year-mark in 2016, Bhatia describes the beginning of a rift between his future vision of SCC-RI and that of McMaster. It was at the same time Kelton retired and was replaced by Dr. Paul OByrne.

We got to a point where I saw this as a Stage 1 achievement, whereas I think they were feeling this is where we needed to be and they were quite happy with it, said Bhatia. I thought we could do more.

About two years ago, funding ran out for two researchers Bhatia had recruited and trained over 10 years, and they ended up leaving McMaster. He also saw no way to recruit the senior scientists he felt he could now attract at the established institute.

By December 2019, Bhatia said he saw the writing on the wall and resigned as director of SCC-RI.

To some degree its understandable, said Bhatia. This type of science and at this level is very, very expensive. It requires an immense commitment There is a certain risk measure that comes with that.

In the wake of Bhatias resignation, McMaster spent about $8,000 on an external review which Bramson said concluded there was no collaboration.

Its a series of individuals who are operating independently, he said. Some of those individuals are quite successful which is great and they continue to operate independently but there was no value gained by having them work together.

Bramson said he doesnt know why they werent collaborating and the review didnt shed any light on that either.

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I cant tell you why people can or cannot work together, he said. Its their choice, they dont have to, youre not obliged to collaborate.

In fact, he says its not in a researchers nature to work together.

Scientists are mavericks, said Bramson. They are stallions, they are not trained to work together, theyre trained to work independently.

He also said this happens all the time about research institutes being terminated.

It didnt work out, I dont really see that as being a surprise, he said. Science is an experiment ... When you create an institute, you dont know how its going to shake out.

At some point between December and the board of governors meeting in June, two other principle investigators announced they were leaving.

People leave for their own reasons, said Bramson. Clearly, if they felt that they were gaining something from being part of the institute, they would have stayed.

Other principle investigators including Dr. Sheila Singh and Dr. Tobias Berg appear to be staying at McMaster and continuing their research independently.

The group was not the reason they were successful, said Bramson. Disbanding the group is not going to diminish that success. Theyre going to continue doing what theyre doing.

No principle investigators responded to The Spectators request for comment. Although Bhatia says the university made it clear to all that it was speaking on the institutes behalf. He was originally unable to speak himself but eventually got permission from McMaster.

For now, Bhatia is continuing on with his research here.

I love Canada, he said. I love McMaster.

But he added his priority is moving stem cell and cancer therapy science forward.

Well have to see how that unfolds, he said.

As for OByrnes decision to recommend terminating the institute, Bhatia says, It was the obvious decision.

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Stem Cell and Cancer Research Institute terminated by McMaster University - TheSpec.com

A recent report published by QMI on mesenchymal stem cells market is a detailed assessment of the most important market dynamics. After carrying out a thorough research of mesenchymal stem cells market historical as well as current growth parameters, business expectations for growth are obtained with utmost precision. The study identifies specific and important factors affecting the market for mesenchymal stem cells during the forecast period. It can enable manufacturers of mesenchymal stem cells to change their production and marketing strategies in order to envisage maximum growth.

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According to the report, the mesenchymal stem cells market has been segmented by source (bone marrow, umbilical cord blood, peripheral blood, lung tissue, synovial tissues, amniotic fluids, adipose tissues), by application (injuries, drug discovery, cardiovascular infraction, others).Insights about the regional distribution of market:The market has been segmented in major regions to understand the global development and demand patterns of this market.

For the mesenchymal stem cells market, the segments by region are North America, Asia Pacific, Western Europe, Eastern Europe, Middle East, and Rest of the World. During the forecast period, North America, Asia Pacific and Western Europe are expected to be major regions on the mesenchymal stem cells market.

North America and Western Europe have been one of the key regions as they have an established healthcare infrastructure for product innovations and early adaptations. This is estimated to drive demand for the mesenchymal stem cells market in these regions. In addition to this, some of the major companies operating in this market are headquartered in these regions.

Asia Pacific is estimated to register a high CAGR mesenchymal stem cells market. The APAC region has witnessed strategic investments by global companies to cater to the growing demand for healthcare solutions in recent years. The Middle East and Rest of the World are estimated to be emerging regions for the mesenchymal stem cells market.

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Market Players Cell Applications, Inc., Cyagen Biosciences Inc. Axol Bioscience Ltd., Cytori Therapeutics Inc., Stem Cell Technologies Inc., Celprogen, Inc.

Reasons to Buy This Report:o It provides niche insights for a decision about every possible segment helping in the strategic decision-making process.o Market size estimation of the mesenchymal stem cells market on a regional and global basis.

o A unique research design for market size estimation and forecast.o Identification of major companies operating in the market with related developmentso Exhaustive scope to cover all the possible segments helping every stakeholder in the mesenchymal stem cells market.

Market Segmentation:By Source:o Bone Marrowo Umbilical Cord Bloodo Peripheral Bloodo Lung Tissueo Synovial Tissueso Amniotic Fluidso Adipose Tissues

By Application:o Injurieso Drug Discoveryo Cardiovascular Infractiono Others

By Region:o North Americao North America, by Country? US? Canada? Mexicoo North America, by Sourceo North America, by Application

o Western Europeo Western Europe, by Country? Germany? UK? France? Italy? Spain? The Netherlands? Rest of Western Europeo Western Europe, by Sourceo Western Europe, by Application

o Asia Pacifico Asia Pacific, by Country? China? India? Japan? South Korea? Australia? Indonesia? Rest of Asia Pacifico Asia Pacific, by Sourceo Asia Pacific, by Application

o Eastern Europeo Eastern Europe, by Country? Russia? Turkey? Rest of Eastern Europeo Eastern Europe, by Sourceo Eastern Europe, by Application

o Middle Easto Middle East, by Country? UAE? Saudi Arabia? Qatar? Iran? Rest of Middle Easto Middle East, by Sourceo Middle East, by Applicationo Rest of the Worldo Rest of the World, by Country? South America? Africao Rest of the World, by Sourceo Rest of the World, by Application

Years Covered in the Study:Historic Year: 2016-2017Base Year: 2018Estimated Year: 2019Forecast Year: 2028

Objectives of this report:o To estimate the market size for mesenchymal stem cells market on a regional and global basis.o To identify major segments in mesenchymal stem cells market and evaluate their market shares and demand.

o To provide a competitive scenario for the mesenchymal stem cells market with major developments observed by key companies in the historic years.o To evaluate key factors governing the dynamics of mesenchymal stem cells market with their potential gravity during the forecast period.Customization:This study is customized to meet your specific requirements:

o By Segmento By Sub-segmento By Region/Countryo Product Specific Competitive Analysis

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Impacts of the COVID-19-Mesenchymal Stem Cells Market Size Current and Future Industry Trends, 2020-2028 - 3rd Watch News

Stem cells have been holding great promise for regenerative medicine for years. In the last decade, several studies have shown that this type of cell, which in Spanish is called mother cell because of its ability to give rise to a variety of different cell types, can be applied in regenerative medicine for diseases such as muscular and nervous system disorders, among others. Researchers and stem cell pioneers Sir John B. Gurdon and Shinya Yamanaka received the Nobel Prize in Physiology and Medicine in 2012 for this idea. However, one of the main limitations in the application of these cell therapies is the quality of the stem cells that can be generated in the laboratory, which impedes their use for therapeutic purposes.Now, a team from the Cell Division and Cancer Group of the Spanish National Cancer Research Centre (CNIO), led by researcher Marcos Malumbres, has developed a new, simple and fast technology that enhances in vitro and in vivo the potential of stem cells to differentiate into adult cells. The research results are published in The EMBO Journal.

In recent years, several protocols have been proposed to obtain reprogrammed stem cells in the laboratory from adult cells, but very few to improve the cells we already have. The method we developed is able to significantly increase the quality of stem cells obtained by any other protocol, thus favouring the efficiency of the production of specialised cell types, says Mara Salazar-Roa, researcher at the CNIO, first author of the article and co-corresponding author.

In this study, the researchers identified an RNA sequence, called microRNA 203, which is found in the earliest embryonic stages before the embryo implants in the womb and when stem cells still have their maximum capacity to generate all the different tissues. When they added this molecule to stem cells in the laboratory, they discovered that the cells ability to convert to other cell types improved significantly.

To corroborate this, they used stem cells of human and murine origin, and of genetically modified mice. The results were spectacular, both in mouse cells and in human cells. Application of this microRNA for just 5 days boosts the potential of stem cells in all scenarios we tested and improves their ability to become other specialised cells, even months after having been in contact with the microRNA, says Salazar-Roa.

According to the study, cells modified by this new protocol are more efficient in generating functional cardiac cells, opening the door to an improved generation of different cell types necessary for the treatment of degenerative diseases.

Malumbres, head of the CNIO Cell and Cancer Division Group, says: To bring this asset to the clinic, collaboration with laboratories or companies that want to exploit this technology is now necessary in each specific case. In this context, Salazar-Roa recently participated, in close collaboration with the CNIOs Innovation team, in prestigious innovation programs such as IDEA2 Global of the Massachusetts Institute of Technology (MIT) and CaixaImpulse of the la Caixa Foundation, from which they also obtained funding to start the development of this technology.

This article has been republished from the following materials. Note: material may have been edited for length and content. For further information, please contact the cited source.

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Quick and Simple Technology Enhances the Potential of Stem Cells To Differentiate Into Adult Cells - Technology Networks

The global artificial blood substitutes market is predicted to register stellar growth rate in the forthcoming years. The presence of a large patient population that requires blood transfusion during surgeries, trauma, and for other blood disorders, which remains unmet due to shortage of blood supply has necessitated creation of artificial blood substitutes.

Artificial blood substitutes are primarily used to mimic oxygen carrying capacity of biological blood and expand the blood volume in the human body. Use of artificial blood substitutes is at present considered an alternate method for blood transfusion. Further research is underway to develop more alternate methods for blood transfusion, including developing human red blood cells (RBCs) from stem cells of donors blood.

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The research report provides an in-depth analysis of the artificial blood substitutes market over the forecast period. The report covers each and every key aspect pertinent to the market, including market dynamics, segmentation, and competitive scenario. The assessment of artificial blood substitutes market presented herein could serve as a valuable guide for both existing market participants, and the ones seeking entry in this market.

Artificial Blood Substitutes Market: Competitive Landscape and Notable Developments

The initial clinical trials for blood substitutes are recorded as early as early 1600, wherein milk, beer, urine, sheeps blood, and perfluorochemicals were administered as blood substitutes for animal and human subjects.

In successive periods, clinical trials of milk transfusion, including goats milk in large quantities were carried out but in vain. Clinical trials also involved injecting human milk that were futile too, which led researchers concede human milk not to be a substitute for blood.

With continual extensive research, over long periods, scientists have attained some success to develop blood substitutes. Artificial blood thus far developed can substitute red blood cells. While biological human blood performs several different functions, artificial blood performs the sole purpose of transporting oxygen and carbon dioxide in the body.

Established biotechnology companies in the ambit are engaged to develop blood substitutes. Such pursuits primarily involve developing oxygen carriers similar or above the capacity of biological blood. With concerted efforts of some top-notch biotechnology companies, namely HEMARINA SA, KaloCyte Inc. and Hemoglobin Oxygen Therapeutics LLC blood substitutes are available as oxygen carrier based on hemoglobin and perfluorocarbon-based oxygen carrier.

Nevertheless, presence of several well-established biotechnology companies engaged in the development of blood substitutes portrays a competitive yet moderately consolidated vendor landscape of the artificial blood substitutes market.

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Artificial Blood Substitutes Market: Key Trends

Worldwide, excessive blood loss due to traumatic injuries and diseases is responsible for vast number of deaths every year. Limited availability of fresh blood and small storage periods of fresh blood for such situations have necessitated development of artificial blood substitutes.

With continual experiments over long periods, scientists have thus far been able to create substitutes to mimic oxygen carrier capacity of biological blood. Development of perfluorochemical-based oxygen carrier and hemoglobin-based oxygen carrier and provide thrust to the artificial blood substitutes market.

Besides this, advent of stem cell therapy is poised to create new opportunities for demand of artificial blood substitutes.

However, on the downside, lower shelf life of artificial blood products and stringent regulatory approval process for these products restrain the growth of artificial blood substitutes market.

Artificial Blood Substitutes Market: Regional Outlook

North America is at the fore for demand within overall artificial blood substitutes market. Presence of advanced healthcare combined with awareness of individuals for alternate demonstrated therapies account for leading revenue share of the region.

Continual advances in stem cell therapy further indicates sustained growth of artificial blood substitutes market in the region.

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Artificial Blood Substitutes Market - Insights into how contours of market will change in coming years - BioSpace

Global Stem Cells Cryopreservation Equipments Market: Competitive Analysis

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The market is segmented into{Liquid Phase, Vapor Phase};{Cord Blood Stem Cells Cryopreservation, Other Stem Cells Cryopreservation}. The geographical presence of the Stem Cells Cryopreservation Equipments market is categorized mainly into North America, Europe, Asia Pacific, Latin America, and the Middle East and Africa. Some of the major industry players included within the Stem Cells Cryopreservation Equipments market study includesChart, Worthington Industries, Cesca Therapeutics, Shengjie Cryogenic Equipment, Sichuan Mountain Vertical, Qingdao Beol,.

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Global Stem Cells Cryopreservation Equipments Market with Coronavirus (COVID-19) Impact Analysis | likewise Industry is Booming Globaly with Top...

No matter how many molecules he threw at them, Paul Tesar couldnt get the brain cells to survive. Or he got them to survive, but then to everyones bafflement they still couldnt do what they were supposed to.

Tesar, a professor of innovative therapeutics at Case Western University, had spent years building stem cell models for multiple sclerosis, growing brain organoids in dishes and then seeing what small molecules restored myelin production. Now he was trying to do the same for other myelin diseases, particularly an ultra-rare genetic condition called Pelizaeus-Merzbacher disease, where a single mutation leads to the death of the myelin-producing neurons, called oligodendrocytes, and can kill patients in infancy.

Weve screened many thousands of small molecule compounds, Tesar toldEndpoints News. But we could not get them to restore function.

Then Tesar got an email from Ionis, the California biotech that had just used an RNA-modifying technology called antisense to build Spinraza, the first FDA-approved drug for the genetic neurological disorder spinal muscular atrophy.

Now, in a study published inNature,Tesar and Ionis have shown they can use a single dose of drug built from that technology to keep those neurons both alive and well-functioning and treat the disease at least in mice. The publication isnt groundbreaking, antisense researchers say, but it shows for the first time that antisense can be used to effectively target oligodendrocytes, an insight its authors hope will open up other rare myelin disorders to therapy.

Its not that its different than everything thats been done before, but it goes further than everything thats gone before, Jon Watts, a professor at the RNA Therapeutics Institute at UMass Medical School who is not affiliated with Ionis or the paper, told Endpoints, both in terms of duration of effect after a single dose, and the real focus in getting the biology, the therapeutic effect in oligodendrocytes.

The applicability to the most famous and common of myelin disorders, multiple sclerosis, is limited, researchers say, both because the therapy relied on having a specific gene to target and because the paper doesnt prove you can get an effect on the peripheral nervous system. Still, Berit Powers, an assistant director at Ioniss neurology research department and a co-author, pointed to several other genetic myelin disorders, known as leukodystrophies. That includes an Ionis program on Alexander disease, a rare childhood condition with Parkinsons-like symptoms.

Were certainly exploring the potential of ASOs in non-monogenic conditions like MS, Powers told Endpoints, using a shorthand for antisense oligonucleotides. But that work is very new.

This is hardly Tesars first foray into biotech. In 2015, he showed in Naturehow certain small molecules could regenerate myelin the holy grail for an MS therapy and founded Convelo Therapeutics around that work. Last year, they partnered with Genentech for an undisclosed sum and an exclusive option to acquire the company.

Myelin is a fatty substance that coats neurons, insulating them and helping electric currents pass through. Tesars lab was broadly interested in the question of why myelin fails, both in MS and rare diseases, and about 7 years ago he got a grant to work from the PMD Foundation.

First, Tesar built stem cell models of the disease, figuring out how different mutations in a single gene, called PLP1, lead oligodendrocyte progenitor cells (the stem cell-like cells that will become oligodendrocytes) to create a toxic RNA and a mutated protein that kills them soon after they differentiate. Then, he tried to suppress that gene with different chemicals, eventually testing over 3,000 different compounds.

He was able to eventually get the oligodendrocytes to survive, but to his surprise, they didnt produce myelin as they should. The surviving cells still couldnt properly function, revealing, he wrote in a 2018 Cell paper a second phase of pathology. A hypothetical treatment, he argued, would have to both keep progenitor cells alive and then treat the survivors in a way that induces myelination.

With antisense, he and Powers Ionis team were able to do both. Antisense oligonucelotides consist of strands of RNA that are a mirror image of the RNA you want to target. The mirror binds to and silences, or turns off, that gene. In the study, the researchers confirmed that PLP1 was disease-causing by knocking out the gene in cell lines with CRISPR. Then they injected mice with antisense strands through the spinal cord, the same way Spinraza is delivered. (You cant use CRISPR to treat the disease in humans, because theres no good way yet of delivering it.)

Powers and Tesar were unsure if they would be able to target oligodendrocytes and progenitor cells. What they found, though, was complete restoration of oligodendrocytes and a profound rescue of neurological function. Myelin, too, was finally restored. Mice that died after 3 weeks now lived for over 200 days.

Ionis hasnt licensed the drug and its unclear yet the implications for other diseases, but researchers say the results could translate into humans quickly, at least by drug development standards.

I do think its very rapidly translatable, Watts said. Based on the data theyre showing here, and based on the unmet need, this appears to be something that could be translated pretty quickly into a Phase I trial.

Excerpt from:
Ionis, leading MS researcher throw antisense at a new type of brain cells - Endpoints News