Archive for the ‘Skin Stem Cells’ Category

MedicalResearch.com Interview with:

Dr. Ke Cheng, PhDProfessor, Department of Molecular Biomedical Sciences, NCSUProfessor, UNC/NCSU joint Department of Biomedical Engineering

MedicalResearch.com: What is the background for this study? What are exosomes?

Response: People are developing lots cosmetic products to keep a healthy and young appearance, like antioxidants, growth factors, peptides and more recently, stem cell products. Also, people are seeking more effective solutions for better absorption, like lotion, mask, laser and fillers. What is used for the treatment and how to deliver it are vitally important to the final effect and lasting time.

Exosomes are nano-sized small vehicles containing proteins, nucleic acids, and they are messengers for cell communication and regulation. Here we use skin cell-secreted exosomes to fight skin aging.

MedicalResearch.com: What are the main findings?

Response: We are proposing a cell-free, needle-free treatment to treat aged skin. Exosomes-treated skin was significantly refined and improved compared to commercial retinoids. Whats more, needle-free jet injector is excellent for the delivery of exosomes. The exosomes can reach deep dermis and still maintain their biological activity.

MedicalResearch.com: What should readers take away from your report?

Response: Exosomes play an important role in cell-free treatment and clinical translation. I think skin cell-derived exosomes hold great potential in rejuvenating skin cells, also, they can penetrate dermis well via jet injection methods. For lotions and mask, bioactive factors are hard to penetrate through stratum corneum, you must apply them daily to keep skin hydrated and smooth. This needle-free injection could achieve a long-lasting effect.

MedicalResearch.com: What recommendations do you have for future research as a result of this study?

Response: Cells are communicating with each other by secreting messengers. It is important to realize the messengers could be different when cells are at different state, like 2D culture, 3D spheroids, treated with drugs and cocultured with different types of cells.

MedicalResearch.com: Is there anything else you would like to add? Any disclosures?

Response: The limitation of this study is the lack of immunological study. Even though it is cell-free treatment, we would like to do more assessment in the future.

Citation:

Needle-Free Injection of Exosomes Derived from Human Dermal Fibroblast Spheroids Ameliorates Skin Photoaging. Shiqi Hu, Zhenhua Li, Jhon Cores, Ke Huang, Teng Su, Phuong-Uyen Dinh, Ke Cheng*.

ACS Nano. 26 Aug 2019.https://doi.org/10.1021/acsnano.9b04384

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Last Modified: Sep 19, 2019 @ 6:21 pm

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Needle-free injection of exosomes enable skin repair after UVB damage - MedicalResearch.com

Stem cells are biological cells which have the ability to distinguish into specialized cells, which are capable of cell division through mitosis. Amniotic fluid stem cells are a collective mixture of stem cells obtained from amniotic tissues and fluid. Amniotic fluid is clear, slightly yellowish liquid which surrounds the fetus during pregnancy and is discarded as medical waste during caesarean section deliveries. Amniotic fluid is a source of valuable biological material which includes stem cells which can be potentially used in cell therapy and regenerative therapies. Amniotic fluid stem cells can be developed into a different type of tissues such as cartilage, skin, cardiac nerves, bone, and muscles. Amniotic fluid stem cells are able to find the damaged joint caused by rheumatoid arthritis and differentiate tissues which are damaged. Medical conditions where no drug is able to lessen the symptoms and begin the healing process are the major target for amniotic fluid stem cell therapy. Amniotic fluid stem cells therapy is a solution to those patients who do not want to undergo surgery. Amniotic fluid has a high concentration of stem cells, cytokines, proteins and other important components. Amniotic fluid stem cell therapy is safe and effective treatment which contain growth factor helps to stimulate tissue growth, naturally reduce inflammation. Amniotic fluid also contains hyaluronic acid which acts as a lubricant and promotes cartilage growth.

With increasing technological advancement in the healthcare, amniotic fluid stem cell therapy has more advantage over the other therapy. Amniotic fluid stem cell therapy eliminates the chances of surgery and organs are regenerated, without causing any damage. These are some of the factors driving the growth of amniotic fluid stem cell therapy market over the forecast period. Increasing prevalence of chronic diseases which can be treated with the amniotic fluid stem cell therapy propel the market growth for amniotic fluid stem cell therapy, globally. Increasing funding by the government in research and development of stem cell therapy may drive the amniotic fluid stem cell therapy market growth. But, high procedure cost, difficulties in collecting the amniotic fluid and lack of reimbursement policies hinder the growth of amniotic fluid stem cell therapy market.

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The global amniotic fluid stem cell therapy market is segmented on basis of treatment, application, end user and geography: Segmentation by Treatment Allogeneic Amniotic Fluid stem cell therapy Autologous Amniotic Fluid stem cell therapy Segmentation by Application Regenerative medicines Skin Orthopedics Oncology Fetal tissue reconstruction Kidney regeneration Regeneration of neural tissue Cardiac regeneration Lung epithelial regeneration Others Drug research and development Segmentation by End User Hospital Ambulatory Surgical Centers Specialty Clinics Academic and Research Institutes Segmentation by Geography North America Latin America Europe Asia-Pacific Excluding China China Middle East & Africa

Rapid technological advancement in healthcare, and favorable results of the amniotic fluid stem cells therapy will increase the market for amniotic fluid stem cell therapy over the forecast period. Increasing public-private investment for stem cells in managing disease and improving healthcare infrastructure are expected to propel the growth of the amniotic fluid stem cell therapy market.

However, on the basis of geography, global Amniotic Fluid Stem Cell Therapy Market is segmented into six key regions viz. North America, Latin America, Europe, Asia Pacific Excluding China, China and Middle East & Africa. North America captured the largest shares in global Amniotic Fluid Stem Cell Therapy Market and is projected to continue over the forecast period owing to technological advancement in the healthcare and growing awareness among the population towards the new research and development in the stem cell therapy. Europe is expected to account for the second largest revenue share in the amniotic fluid stem cell therapy market. The Asia Pacific is anticipated to have rapid growth in near future owing to increasing healthcare set up and improving healthcare expenditure. Latin America and the Middle East and Africa account for slow growth in the market of amniotic fluid stem cell therapy due to lack of medical facilities and technical knowledge.

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Some of the key players operating in global amniotic fluid stem cell therapy market are Stem Shot, Provia Laboratories LLC, Thermo Fisher Scientific Inc. Mesoblast Ltd., Roslin Cells, Regeneus Ltd. etc. among others.

The report covers exhaustive analysis on: Amniotic Fluid Stem Cell Therapy Market Segments Amniotic Fluid Stem Cell Therapy Market Dynamics Historical Actual Market Size, 2012 2016 Amniotic Fluid Stem Cell Therapy Market Size & Forecast 2016 to 2024 Amniotic Fluid Stem Cell Therapy Market Current Trends/Issues/Challenges Competition & Companies involved Amniotic Fluid Stem Cell Therapy Market Drivers and Restraints

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

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

Request to view TOC at https://www.persistencemarketresearch.com/toc/23101?source=atm

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Amniotic Fluid Stem Cell Therapy Market New Growth Opportunities By2018 2026 - My Health Reporter

The Kapamilya stars were among the first to try Belo Medical Group's latest facial treatment, the Salt Facial.

Leila Alcasid, as part of her preparations for the recently held ABS-CBN Ball 2019, had it at the Belo Beauty Suite.

Did you know she went to the ball without any make-up foundation right after she got the treatment?

Continue reading below

Other Kapamilya stars that tried this treatment before the ball were Piolo Pascual, Enrique Gil, Sue Ramirez, Heaven Peralejo, Mccoy De Leon, Barbie Imperial, and Daniella Stranner.

This three-step Salt Facial that helps "restore, replenish, and rejuvenate" the skin in an hour or less.

Dra. Vicki Belo said this treatment is perfect for those who have oily, acne-prone skin.

Continue reading below

It also has great results on minimizing wrinkles and even erasing stretch marks.

She said, "It goes to the dermal level. It uses salt, which has so many benefits for the skin, and we're bringing that machine. It's a brand new machine that we're bringing in, and we've seen amazing results for stretch marks."

Dra. Vicki and her team particularty timed the introduction of the treatment with the ABS-CBN Ball precisely to demonstrate to the stars its immediate results without skin irritation or downtime.

We chose it for the ball because, of course, we have to have calm skin for the night that will catch the light so it will reflect back and they will keep glowing.

Continue reading below

The treatment actually starts with the aesthetician cleaning the face with a cold facial cleanser that is very soothing to the skin.

This is followed by the natural sea salt exfoliation that removes dead skin cells.

Sea salt is a natural product that has anti-bacterial and anti-microbial properties, which kill pimple-causing bacteria.

The process uses a wand-like tool connected to the exfoliating machine that uses closed-loop pressure with microfine medical-grade sea salt.

The result is tighter and healthier skin that better absorbs skincare products.

Continue reading below

After this, a Mandelic or Glycolic acid will be applied on the face, depending on the client's needs. This stem cell-rich gel are massaged and infused on the skin using ultrasound technology.

The last part is the LED light therapy, which is adjusted according to the condition of the skin.

Continue reading below

Blue light is used to minimize inflammation and kill acne-causing bacteria. Meanwhile, red light promotes collagen production and also helps promote healing.

Dra. Vicki said that it also comes in green for pigmentation.

Immediate results can be noticed after the treatment. The skin feels softer, and glowing in an instant.

The Salf Facial will soon be available at Belo Clinics nationwide.

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Leila Alcasid's facial can dry acne fast in just one session - Philippine Entertainment Portal

Stem cells are biological cells which have the ability to distinguish into specialized cells, which are capable of cell division through mitosis. Amniotic fluid stem cells are a collective mixture of stem cells obtained from amniotic tissues and fluid. Amniotic fluid is clear, slightly yellowish liquid which surrounds the fetus during pregnancy and is discarded as medical waste during caesarean section deliveries. Amniotic fluid is a source of valuable biological material which includes stem cells which can be potentially used in cell therapy and regenerative therapies. Amniotic fluid stem cells can be developed into a different type of tissues such as cartilage, skin, cardiac nerves, bone, and muscles. Amniotic fluid stem cells are able to find the damaged joint caused by rheumatoid arthritis and differentiate tissues which are damaged. Medical conditions where no drug is able to lessen the symptoms and begin the healing process are the major target for amniotic fluid stem cell therapy. Amniotic fluid stem cells therapy is a solution to those patients who do not want to undergo surgery. Amniotic fluid has a high concentration of stem cells, cytokines, proteins and other important components. Amniotic fluid stem cell therapy is safe and effective treatment which contain growth factor helps to stimulate tissue growth, naturally reduce inflammation. Amniotic fluid also contains hyaluronic acid which acts as a lubricant and promotes cartilage growth.

With increasing technological advancement in the healthcare, amniotic fluid stem cell therapy has more advantage over the other therapy. Amniotic fluid stem cell therapy eliminates the chances of surgery and organs are regenerated, without causing any damage. These are some of the factors driving the growth of amniotic fluid stem cell therapy market over the forecast period. Increasing prevalence of chronic diseases which can be treated with the amniotic fluid stem cell therapy propel the market growth for amniotic fluid stem cell therapy, globally. Increasing funding by the government in research and development of stem cell therapy may drive the amniotic fluid stem cell therapy market growth. But, high procedure cost, difficulties in collecting the amniotic fluid and lack of reimbursement policies hinder the growth of amniotic fluid stem cell therapy market.

Get Full Report Overview at https://www.persistencemarketresearch.com/market-research/amniotic-fluid-stem-cell-therapy-market.asp

The global amniotic fluid stem cell therapy market is segmented on basis of treatment, application, end user and geography: Segmentation by Treatment Allogeneic Amniotic Fluid stem cell therapy Autologous Amniotic Fluid stem cell therapy Segmentation by Application Regenerative medicines Skin Orthopedics Oncology Fetal tissue reconstruction Kidney regeneration Regeneration of neural tissue Cardiac regeneration Lung epithelial regeneration Others Drug research and development Segmentation by End User Hospital Ambulatory Surgical Centers Specialty Clinics Academic and Research Institutes Segmentation by Geography North America Latin America Europe Asia-Pacific Excluding China China Middle East & Africa

Rapid technological advancement in healthcare, and favorable results of the amniotic fluid stem cells therapy will increase the market for amniotic fluid stem cell therapy over the forecast period. Increasing public-private investment for stem cells in managing disease and improving healthcare infrastructure are expected to propel the growth of the amniotic fluid stem cell therapy market.

However, on the basis of geography, global Amniotic Fluid Stem Cell Therapy Market is segmented into six key regions viz. North America, Latin America, Europe, Asia Pacific Excluding China, China and Middle East & Africa. North America captured the largest shares in global Amniotic Fluid Stem Cell Therapy Market and is projected to continue over the forecast period owing to technological advancement in the healthcare and growing awareness among the population towards the new research and development in the stem cell therapy. Europe is expected to account for the second largest revenue share in the amniotic fluid stem cell therapy market. The Asia Pacific is anticipated to have rapid growth in near future owing to increasing healthcare set up and improving healthcare expenditure. Latin America and the Middle East and Africa account for slow growth in the market of amniotic fluid stem cell therapy due to lack of medical facilities and technical knowledge.

Get Sample Copy of this report at https://www.persistencemarketresearch.com/samples/23101

Some of the key players operating in global amniotic fluid stem cell therapy market are Stem Shot, Provia Laboratories LLC, Thermo Fisher Scientific Inc. Mesoblast Ltd., Roslin Cells, Regeneus Ltd. etc. among others.

The report covers exhaustive analysis on: Amniotic Fluid Stem Cell Therapy Market Segments Amniotic Fluid Stem Cell Therapy Market Dynamics Historical Actual Market Size, 2012 2016 Amniotic Fluid Stem Cell Therapy Market Size & Forecast 2016 to 2024 Amniotic Fluid Stem Cell Therapy Market Current Trends/Issues/Challenges Competition & Companies involved Amniotic Fluid Stem Cell Therapy Market Drivers and Restraints

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

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

Request Methodology on this report at https://www.persistencemarketresearch.com/methodology/23101

Read more:
Amniotic Fluid Stem Cell Therapy Market to Create Lucrative Opportunities for Existing Companies as Well as New Players - Herald Space

Stromal vascular fraction skin treatment is a type of stem cell therapy based on isolation of adipose tissue during liposuction or lipo-aspiration procedures of patients own body. In stromal vascular fraction treatment isolation of tissue contains fat cells, blood cells, and endothelial cells, as well as a large fraction of adipose-derived mesenchymal stem cells which provides regenerative properties and have positive anti-aging properties. A stromal vascular fraction is considered as a personalized stem cell therapy and effective tropical or injectable treatment.

With increasing age, regenerative and repair properties of skin are less effective due to decrease in stem cell count, and therefore, stromal vascular fraction treatment contains stem cell provides a boost in repair and maintenance mechanism of the skin leaving smooth, healthy, radiant skin. Stromal vascular fraction is a naturally occurring stem cell found in bundles of adipose tissues and are the primary source of growth factors along with macrophages and other cells. Due to the presence of growth factors, the stromal vascular fraction is utilized to decrease inflammation present in many diseases. A stromal vascular fraction is adopted in the treatment of rheumatoid arthritis, joint replacement, osteoarthritis, diabetes, Crohn's disease, and others.

Stromal Vascular Fraction Market: Overview

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Stromal vascular fraction is a combination of adipose-derived stromal cells (ADSCs), endothelial cells (ECs), endothelial precursor cells (EPCs), smooth muscle cells, macrophages, pericytes, and pre-adipocytes in the aqueous state. Stromal vascular fraction is advantageous over alternative medical treatments as SVF has the ability to regulate patients own system with the main focus on cell repair and regulation of defective cells. Stromal vascular fraction is a promising field for disease prophylaxis and currently are in clinical trials.

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

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

Stromal Vascular Fraction Market: Segmentation

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The globalstromal vascular fraction marketcan be segmented on the basis of type of therapy, end-user, and region.

By Therapy Type SVF Isolation Products Enzymatic Isolation Non-enzymatic Isolation Automated POC Devices SVF Aspirate Purification Products SVF Transfer Products

By End-user Hospitals Specialty Clinics Stem Cell Banks/Laboratories Others

By Application Cosmetic Soft-tissue Orthopedic Others

By Region North America Latin America Europe Asia Pacific (APAC) South Korea Middle East and Africa (MEA)

In its last part, the report offers insights on the key players competing in the global market for stromal vascular fraction. With detailed profiling of each of the key companies active on the competitive landscape, the report provides information about their current financial scenario, revenue share at a global level, development strategies, and future plans for expansion. Strategic collaborations, mergers, and acquisitions have also been considered as a key strategy among a majority of leading companies in the market.

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Originally posted here:
Stromal Vascular Fraction Market by Top Key Players, Size, Subdivision & Market Dynamics Forces - Commerce Gazette

Everyone has heard of the seven wonders of the ancient world. Today I present my verdict on seven wonder discoveries in biological research, listed in rough chronological order.

Wonder 1: Cell TheoryCell theory, formulated in 1839, made three assertions: (a) All organisms are composed of basic units called cells; (b) The cell is the basic unit of structure and organisation in all living organisms; (c) New cells arise only from pre-existing cells.

Cell theory rationalised basic biology and contradicted the common notion that life can arise spontaneously from non-living matter.

Wonder 2: The Theory of Evolution Through Natural Selection.This is the most significant-ever insight into biology and was jointly proposed in 1858 by Charles Darwin (1809-1882) and Alfred Russell Wallace (1823-1913). It was already known that life on earth changes over long time periods. The theory of evolution explained the mechanism underpinning these changes natural selection. This theory draws all biology together into one unified framework. In its absence, biology would be reduced to a vast catalogue of unrelated observations.

Wonder 3: The Chemistry of LifeLife, unlike mechanical machines, works close to room temperature and without the assistance of significant temperature or pressure gradients. How then do cells grow, divide and create local order in a world that otherwise moves towards increasing disorder? This question was answered by biochemistry the chemistry of life.

A living cell is the end product of co-ordinated reactions between its innumerable chemical constituents metabolism. These reactions must proceed fast enough at room temperature to sustain life, and this is enabled by protein catalysts called enzymes, discovered in the 1800s. Cell metabolism is driven by energy, ultimately supplied by sunlight.

Wonder 4: Antibiotics and VaccinationsAntibiotics, discovered in 1928, are used to kill pathogenic microbes, and vaccination to provide immunity against disease has been in widespread use since 1900. Vaccination has eliminated many deadly diseases, including smallpox and polio.

Average life expectancy in 1900 was 50 years; today that figure is 82 years. Antibiotics and immunisation are one significant cause of this improvement. If we lost antibiotics and vaccinations today, life expectancy would gradually revert to 50-60 years.

Wonder 5: Discovering the Structure of DNAFaithful inheritance of parental characteristics by offspring is essential for biological evolution. The nature of the cells hereditary material was identified as nucleic acid (DNA) in 1944. In 1953, James Watson (born in 1928) and Francis Crick (1916-2004) made the greatest biological discovery of the 20th century when they solved the structure of DNA, a discovery that also indicated how DNA replicates and transmits genetic information from generation to generation.

DNA is a long molecule made of four sub-units A,T,G,C. Genetic information is encoded in the linear sequence (genes) of these subunits, dictating the structure/activity of all cell proteins, including all enzymes, thereby controlling the activities of the cell. Understanding DNA unlocked the secret of the molecular logic of life.

Wonder 6: CloningEvery animal body cell, including cells of the early embryo, contains a full set of the animals genes. Therefore, inducing any body cell into developing as embryonic cells develop should produce a genetic copy of the animal a clone. In 1996 Dolly the sheep was cloned in this manner.

Cloning offers many huge potential benefits, eg cloning animals, genetically modified to secrete a human hormone, to produce herds secreting large quantities of the hormone for use by patients deficient in the hormone. Cloning could also rebuild endangered animal populations or revive extinct animals.

Wonder 7: Induced Pluripotent Stem CellsThe human body is composed of over 200 tissues/organs skin, liver etc. Each tissue cell is differentiated to do a particular job. But all tissue cells develop from embryonic undifferentiated pluripotent stem cells.

In 2006 Shinya Yamanaka discovered how to transform differentiated body cells into pluripotent stem cells. These induced pluripotent stem cells (IPSC) can be coaxed to develop into specific adult tissues.

IPSCs have huge potential in many areas, eg regenerative medicine. New organs could be grown from patients own IPSCs to replace failing organs and without fear of immune rejection.

IPSCs are extremely useful for many research purposes, eg testing new drugs, and can substitute for stem cells extracted from human embryos, thereby avoiding ethical problems.

William Reville is an emeritus professor of biochemistry at UCC

See more here:
Story of life: Seven wonders of biological research - The Irish Times

Cancer stem cells (CSCs) refer to the cells obtained from tumor that posses potential to reproduce all types of cancer cells found in a cancer sample. Cancer stem cells are planned to grow in tumors as a separate population and thereby cause deterioration and metastasis of existing tumor through generation of new tumor. Thus, with advancement in technology especially in cancer stem cells research area, therapies specific to targeting cancer stem cells are expected to improve quality of life and survival cases of cancer patients with metastatic diseases.

Morbidity and mortality rate of cancer is rising at a faster speed worldwide and thus prevention of cancer and cancer treatment is grabbing attention of cancer researchers globally. Stem cells and cell therapy have shown significant potential to treat cancer effectively. Cancer stem cells (CSCs) have been tested on animal models and have also shown satisfactory results. However, human testing of cancer stem cells is still in its developing stage owing to stringent regulations and ethical issues associated with the same.

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Worldwide cancer research activities are increasing rapidly owing to rising burden of mortality rate of cancer. Cancer stem cells are under research for various types of cancers such as lung cancer, breast cancer, colorectal cancer, skin cancer, brain cancer and bone cancer. Government initiative to boost the cancer research activities and availability of funds are some of the factors that are driving the global cancer stem cells (CSCs) market towards growth. While on the other hand, ethical issues involved in the stem cells research and stringent regulations to perform human trials are some of the factors that are restraining the growth of the global cancer stem cells (CSCs) market.

Geographically, global cancer stem cells market is segmented into North America, Europe, Asia Pacific and Rest of the world (RoW) regions. Currently, North America is leading the global cancer stem cells (CSCs) market and is followed by Europe. Factors such as highly developed research infrastructure, well defined regulatory norms, availability of research funds, availability of skilled research and healthcare professionals and supportive economy are driving the North American cancer stem cells market towards growth. Asia Pacific is lucrative market for cancer stem cells. Governments in the Asia Pacific countries mainly, India and China are taking initiative to boost the healthcare and biotechnology industry in the respective countries and thus, research and development activities in these countries are swiftly increasing.

Apart from India and China, Japan will play a significant role in the cancer stem cells market. Japanese government is heavily investing in healthcare industry in order to improve the healthcare facilities in the country and thus rising cancer treatment are expected to escalate the cancer stem cells treatment market in Japan. Latin American countries namely, Brazil, Mexico and Argentina are expected to contribute more to cancer stem cells market than other countries in the rest of the world region. While on the other hand, African countries and Middle Eastern countries are expected to show slow or no growth rate in the global cancer stem cells (CSCs) market.

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Some of the major players in the global cancer stem cells market are AdnaGen GmbH, Advanced Cell Diagnostics, Inc., AVIVA Biosciences Corporation, Celula, Inc., Epic Sciences, Inc., Fluxion Biosciences, Inc., Rarecells USA, Inc. and Silicon Biosystems, S.p.A.

This research report analyzes this market on the basis of its market segments, major geographies, and current market trends. Geographies analyzed under this research report include North America Asia Pacific Europe Rest of the World

This report provides comprehensive analysis of Market growth drivers Factors limiting market growth Current market trends Market structure Market projections for upcoming years

This report is a complete study of current trends in the market, industry growth drivers, and restraints. It provides market projections for the coming years. It includes analysis of recent developments in technology, Porters five force model analysis and detailed profiles of top industry players. The report also includes a review of micro and macro factors essential for the existing market players and new entrants along with detailed value chain analysis.

Reasons for Buying this Report This report provides pin-point analysis for changing competitive dynamics It provides a forward looking perspective on different factors driving or restraining market growth It provides a six-year forecast assessed on the basis of how the market is predicted to grow It helps in understanding the key product segments and their future It provides pin point analysis of changing competition dynamics and keeps you ahead of competitors It helps in making informed business decisions by having complete insights of market and by making in-depth analysis of market segments It provides distinctive graphics and exemplified SWOT analysis of major market segments

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Cancer Stem Cells Market to Increase at Steady Growth Rate 2016-2024 - The Check Chronicle

Updated as of September 19, 2019

With one in 10 Americans over 65 currently living with symptomatic Alzheimers disease, you probably know someone affected by this disease. Worldwide, 50 million people live with symptomatic Alzheimers, making it the most common form of dementia. It commonly affects people over 65, but less than 4 percent of the estimated 5.7 million Americans affected have early-onset Alzheimers with symptoms beginning before age 65.

By 2050, nearly 14 million Americans are projected to suffer from this disease. Alzheimers is 6th leading cause of death in the US, making Alzheimers disease a top health concern. Unfortunately, there is no cure, but current medications and management strategies may improve symptoms, prolonging patient independence. In honor of November being Alzheimers Awareness month, we evaluated the current therapies, drugs in the pipeline and disease outlook.

Overview

Alzheimers disease is a degenerative brain disease that typically begins in late middle age or old age. Degeneration of brain cells, called neurons, cause the symptoms of progressive memory loss, impaired thinking, disorientation and mood and personality changes.

Risk factors: The greatest risk factors are old age, having a family history of Alzheimers and carrying a mutation in a certain gene called apolipoprotein E 4 (APOE4). Environmental and lifestyle factors, such as diet and exercise, also contribute to disease development.

The risk of Alzheimers doubles every five years after the age of 65, with nearly 1 in 3 people age 85 or older developing the disease. People with the APOE4 gene variant are thought to have an increased risk for developing late-onset Alzheimers, but thats not a steadfast rule: inheriting the gene variant does not mean the person will definitely get Alzheimers and some Alzheimers patients do not have the APOE4 gene variant.

Causes: Alzheimers develops as a result of a complex interaction between many risk factors, all resulting in neuron damage and death. The buildup of misfolded proteins, such as the tau protein and -amyloid, create the hallmark protein clumps called tangles and plaques seen in Alzheimers brains. While these protein clumps are thought to cause neuron death by blocking nerve cell communication and function, the exact relationship between the protein clump formation and neuron death is still unclear.

The four stages of Alzheimers: Based on the severity of dementia symptoms, Alzheimers can be characterized into four stages: preclinical, mild (early-stage), moderate (middle-stage) and severe (late-stage). The preclinical stage encompasses all the unseen changes in the brain, such as plaque accumulations, happening years before symptoms arise. Mild Alzheimers patients can still function independently, although they begin forgetting familiar words or locations of objects. As the dementia progresses to become moderate, the patient becomes more forgetful, has greater difficulty doing daily tasks and experiences personality and behavioral changes. At this stage, they may still remember significant life events. The moderate disease stage is the longest, often lasting for many years. Finally, severe Alzheimers patients can no longer respond to their environment, carry a conversation and control their movement or bowels. As the disease progresses, patients require an increasing level of care for daily activities.

Life expectancy: The earlier the diagnosis, the longer the life expectancy is: people diagnosed in their 60s to early 70s can live as long as 7 to 10 years, whereas those diagnosed in their 90s only average a 3-year life expectancy. Alzheimers patients live an average of four to eight years after their diagnosis, but can live as long as 20 years post-diagnosis. However, its difficult to link one disease to life expectancy, especially as you age, due to the many variables that influence life expectancy.

Cost: The cost burden of Alzheimers is as high as its prevalence: Alzheimers medications can range from $177 to $400 monthly, adding up to an annual prescription drugs estimated cost of $3,000. It will cost Americans an estimated $277 billion, including $186 billion in Medicare and Medicaid payments, to care for Alzheimers patients by the end of 2018. By 2050, this cost is projected to be more than $1.1 trillion, accounting for over four-fold increases in government spending through Medicare and Medicaid, as well as out-of-pocket expenses. Up to $7.9 trillion in medical and care costs could be saved by diagnosing earlier and more accurately.

Diagnosis Strategies

Although there is no specific test for Alzheimers disease, doctors use a variety of exams, imaging and lab testing to diagnose the disease.

Physical and neurological exams can test reflexes, coordination and memory. Brain imaging is used to rule out other physical abnormalities, such as tumors, stroke or other traumas, that can cause Alzheimers-like symptoms. Imaging can now be used to detect the specific changes that occur in the brains of living Alzheimers patients, not just in post-mortem analysis. Structural imaging techniques, such as magnetic resonance imaging (MRI) and computed tomography (CT), are used to rule out other physical injuries as well as assess Alzheimers-related brain shrinkage. Functional imaging, such as functional MRI (fMRI) and positron emission tomography (PET), can measure brain cell function by tracking the cells sugar and oxygen use.

Specific radioactive molecules, called radiotracers, can be used to detect -amyloid plaques via PET imaging. Three radiotracers have been approved by the U.S. Food and Drug Administration (FDA) since 2012: Amyvid (18F-florbetapir), Vizamyl (18F-flutametamol) and Neuraceq (18F-florbetaben).

Genetic testing can reveal if someone has a mutation, such as the APOE4 gene variant, that may increase their risk for developing Alzheimers. However, it is generally not recommended for Alzheimers diagnosis due to the lower accuracy, as many factors contribute to disease development. The exception is early-onset Alzheimers: Anyone with a family history of early Alzheimers can be screened for certain gene mutations, such as amyloid precursor protein (APP), presenilin-1 (PS-1) and presenilin-2 (PS-2).

Developing better diagnostic testing could facilitate earlier diagnoses, possibly leading to better outcomes. Future testing includes more sensitive mental ability exams and measuring key disease-associated proteins, called biomarkers, in the blood or spinal fluid.

Current Therapies

While there is no cure for Alzheimers disease, a handful of drugs have been approved by the FDA and shown to somewhat slow symptom progression. They can be broken down into two categories: cholinesterase inhibitors, which increase the amount of the neurotransmitter acetylcholine in the brain, resulting in more cell-to-cell communication; and NMDA receptor antagonists, which also alter how brain cells communicate.

Cholinesterase inhibitors include Eisais Aricept (donepezil) and Novartis Exelon (rivastigmine), both approved for all stages of Alzheimers, as well as Janssen Pharmaceuticals Razdyne (galantamine), which is approved for mild to moderate Alzheimers. Allerganhas two NMDA receptor antagonist-based drugs, Namenda (memantine) and the combination drug Namzaric (donepezil and memantine), which are both approved for moderate to severe Alzheimers. Antidepressants and anti-anxiety medications are sometimes prescribed as well to help control behavioral symptoms.

Unfortunately, these drugs can cause potentially severe side effects and arent overwhelmingly effective compared to placebo, although they have helped stave off mental decline for a while in some patients. However, the need for more effective drugs is clear.

Drug Pipeline

A variety of targeted therapies are currently being explored through clinical trials, including drugs against the tau protein, which forms distinctive tangles in Alzheimers brains; the -amyloid protein, which forms plaques in the Alzheimers brain; -secretase (BACE), an enzyme that cuts amyloid precursor protein (APP) into -amyloid; and the 5-HT2A serotonin receptor, which is involved in cognition and memory by mediating neurotransmitters, such as acetylcholine and glutamate.

The Alzheimers drug development market includes many large players, including Eli Lillywith six drugs (two in Phase 1, two in Phase 2 and two in Phase 3);Biogen with five drugs (two in Phase 1, one in Phase 2 and two in Phase 3); Roche, in collaboration with Genentech, AC Immune, and MorphoSys, with three drugs (two in Phase 2 and one in Phase 3); Eisai, in collaboration with Biogen, with one drug in Phase 3; and Eisai alone with one drug in Phase 2 (as of September 13, 2019).

As of September 13, 2019, there are over 670 active/recruiting/not yet recruiting clinical trials for Alzheimers listed on clinicaltrials.gov. According to a paper published in July 2019, there were 132 drugs in development for Alzheimers: 28 drugs in 42 Phase 3 trials, 74 drugs in 83 Phase 2 trials, and 30 drugs in 31 Phase 1 trials. The figure and legend below, taken from the July 2019 paper, shows all the drugs in clinical trials for Alzheimers as of February 2019.

UsAgainstAlzheimers released their 2019 Alzheimers Drug Pipeline report also in July 2019, where they focused on 98 late-stage Alzheimers drugs in development that could potentially reach the market in the next 5-10 years: 26 drugs in Phase 3 trials, and 72 drugs in Phase 2 trials. Their report shows that, despite some large Phase 3 failures this year, the Alzheimers pipeline is still robust.

The following analysis of some Alzheimers drugs in the pipeline will briefly discuss how each drug works and where it is in clinical trials. This information was up to date as of September 13, 2019. Any text in italics represents failed or terminated trials.

Note: This article is not meant to be completely comprehensive and may unintentionally exclude some drugs in development or clinical trials, especially those trials outside of the United States.

Phase 1

Biogen is exploring multiple antibody drugs against the -amyloid and tau proteins, including a Phase 1 trial studying the anti-tau antibody BIIB076 in 48 healthy and Alzheimers patients; a Phase 2 trial (TANGO) examining the anti-tau antibody BIIB092 (gosuranemab) in 528 early-stage Alzheimers patients; a Phase 2 trial in collaboration with Eisai studying the anti--amyloid antibody BAN2401 in 800 early-stage Alzheimers patients; and a Phase 3 trial (Clarity AD) studying BAN2401 in 1566 early Alzheimers patients.

Unfortunately, in March 2019, Biogen and its partner Eisai decided to end all studies involving another one of its anti--amyloid antibodies called aducanumab (previously called BIIB037), including their two Phase 3 trials (ENGAGE and EMERGE) each studying 1605 early-stage Alzheimers patients, a Phase 2 trial (EVOLVE) in 500 Alzheimers patients with mild cognitive impairment (MCI) or mild dementia due to Alzheimers, and a Phase 1 trial (PRIME) in 197 very mild (prodromal) or mild Alzheimers patients. The studies were stopped because they did not meet their clinical endpoints of slowing cognitive and functional impairment, not due to any safety concerns of the drug.

Eli Lilly is pursuing two chemical entities, a Tau Morphomer and an O-GlcNAcase Inhibitor, in Phase 1 clinical trials for Alzheimers.

Proclara Biosciencescombined a part of the human immunoglobulin protein with their unique protein technology, called General Amyloid Interaction Motif (GAIM), to create their fusion protein drug NPT088, which targets both -amyloid and tau proteins. Their Phase 1a safety trial showed that intravenous NPT088 is safe and well-tolerated in 40 healthy patients. Data from their Phase 1b dosing trial in Alzheimers patients is expected in 2019.

Cognition Therapeutics drug candidate CT1812 is a small molecule pill that disrupts -amyloid binding to a receptor called sigma-2 on brain cells, which is thought to prevent the proteins toxicity. CT1812 has been or is being studied in six clinical trials, including one recruiting Phase 1 trial with 18 mild to moderate Alzheimers patients, one recruiting Phase 1/2 trial with 21 mild to moderate Alzheimers patients, and one recruiting Phase 2 trial with 120 mild to moderate Alzheimers patients. CT1812 was well-tolerated and penetrated the brain very well in 80 healthy patients and 19 mild to moderate Alzheimers patients with mild to moderate side effects. Although the treated Alzheimers patients had lower levels of Alzheimers-related proteins (such as neurogranin and synaptotagmin-1, markers of synaptic damage) in their cerebrospinal fluid, they didnt show significantly different cognitive functioning compared with the placebo group after 28 days of treatment.

Samus Therapeuticsis developing a positron-emitting molecule, called 124I-PU-AD, that inhibits a certain protein complex called epichaperone complex, which reduced tau proteins in the brain, restored long-term memory and increased survival in preclinical animal models. 124I-PU-AD is also being used as a PET imaging agent to study the epichaperone complex in the brains of Alzheimers patients. They have completed an early Phase 1 trial in 5 Alzheimers and certain cancer patients to evaluate the molecules metabolism. Another Phase 1 study is currently recruiting 24 healthy volunteers to evaluate the safety and tolerance of the drug.

Janssen Research & Development is examining the ability of a radioactive PET imaging agent, called [18F]MNI-1020, to bind to the tau protein in Alzheimers patients. An early Phase 1 trial studied the safety and brain uptake efficacy of a single injection of the imaging agent in 15 Alzheimers and healthy age-matched patients. That study also compared the location of tau (using [18F]MNI-1020) and -amyloid (using Amyvid (florbetapir)) in patients with suspected Alzheimers.

Longeveron collects stem cells from healthy adult donors to create their own Longeveron mesenchymal stem cells (LMSCs), which have been shown to reduce inflammation and promote cell regeneration. Their Phase 1 clinical trial is currently recruiting 30 Alzheimers patients to evaluate the safety and efficacy of LMSCs.

Athira Pharmas small molecule drug NDX-1017 designed to restore lost or build new connections in the brain. Their Phase 1 trial is currently recruiting to evaluate the drugs safety in two parts, with Part A involving up to 56 healthy young and elderly participants and Part B involving 44 healthy, mild cognitive impairment or mild to moderate Alzheimers patients.

Cortexyme, Inc.is developing COR388, a first-in-class bacterial protease inhibitor that targets the bacteria Porphyromonas gingivalis, which is present in Alzheimers patients brains and cerebrospinal fluid and thought to contribute to the disease. Two completed Phase 1 trials have shown that COR388 is safe and well-tolerated in 58 healthy and nine Alzheimers patients. A Phase 2/3 trial is currently enrolling 573 mild to moderate Alzheimers patients to assess the drugs efficacy, safety, and tolerability.

Allergan was pursuing a small molecule drug called AGN-242071 that selectively targeted certain receptors in the brain, called muscarinic receptors, which may treat symptomatic cognitive deficits and behavioral symptoms in Alzheimers.

Unfortunately, Allergan decided to withdraw their Phase 1 trial evaluating the safety and tolerability of the drug prior to patient recruitment in November 2018.

Corium Internationalhas developed a novel delivery method for an approved drug, a once-weekly skin patch (the Corplex Donepezil Transdermal System) that delivers a sustained dose of donepezil. The patchs safety and drug profile were examined in multiple Phase 1 trials, which showed great skin tolerability and comparable dosages between the weekly patch and the currently prescribed daily donepezil pills. Corium is also developing a once-weekly skin patch to deliver memantine

Cognoptix has taken a different approach, developing an eye test called Sapphire II to catch and diagnose Alzheimers much earlier by detecting -amyloid deposits in their eyes. A fluorescent drug that binds to the -amyloid protein (Aftobetin-HCl) is administered to the eye as an ointment and binding is detected with the Sapphire II laser device. Their Phase 1 study determined the optimal dosing of the fluorescent drug in 15 participants and is currently recruiting 10 normal and 20 mild cognitive impairment (MCI) or mild Alzheimers patients for dose testing. If the dosing is optimal, then 30 more MCI and 30 more mild Alzheimers patients will be recruited, totaling 105 participants.

Phase 1/2

Ionis Pharmaceuticalsis collaborating with Biogen to study their antisense oligonucleotide drug IONIS-MAPTRx (also called BIIB080), which may reduce tau protein production and its accumulation in brain cells, in a Phase 1/2 trial in 44 mild Alzheimers patients.

QR Pharma, Inc.s small molecule drug Posiphen inhibits APP, tau and -synuclein (involved with Parkinsons disease) protein synthesis. They are currently recruiting 24 Alzheimers patients for their Phase 1/2 dosage study (DISCOVER).

Following their successful Phase 1 trial (SEAD) in 15 Alzheimers patients, Ausio Pharmaceuticalsbrought their estrogen receptor activating drug S-equol (also called AUS-131) to a Phase 1/2 trial (SEAD2), which is currently recruiting 40 Alzheimers patients to test the drugs tolerability and whether or not it affects cognitive abilities. Activating the estrogen receptors on mitochondria is thought to promote mitochondrial functioning, which could restore the reduced mitochondrial activity seen in Alzheimers patients. Less mitochondrial activity is thought to contribute to -amyloid protein build-up in the brain.

Nature Cell Co. is studying a fat cell-derived mesenchymal stem cell (MSC) therapy called AstroStem in an active Phase 1/2 study involving 21 mild to moderate Alzheimers patients.

Phase 2

Eli Lilly has two ongoing Phase 2 trials studying antibody drugs: one active trial (TRAILBLAZER-ALZ) evaluating the tolerability and efficacy of a humanized anti--amyloid antibody, called donanemab (LY3002813 or N3pG-A MAb), in 266 early symptomatic Alzheimers patients; and another currently recruiting trial evaluating the safety and efficacy of a humanized anti-tau antibody, called zagotenemab (LY3303560), in 285 early symptomatic Alzheimers patients.

Roche, in partnership with AC Immune, is studying crenezumab (RG7412), an anti--amyloid antibody drug that binds to -amyloid similar to Eli Lillys solanezumab. Crenezumab is being investigated in an active Phase 2 trial involving 252 non-symptomatic adults with a family history of Alzheimers who have a particular genetic mutation (autosomal-dominant PSEN1 E280A). Baseline data for 242 of the enrolled patients were presented at the Alzheimers Association International Conference in August 2019. Another not yet recruiting Phase 2 trial is in the works to study the effect of crenezumab on the longitudinal tau burden via PET imaging of 150 patients enrolled in the active Phase 2 trial (NCT01998841).

Crenezumab was being investigated in three Phase 3 trials: CREAD 1 evaluating the drugs safety and efficacy in 813 mild Alzheimers patients; CREAD 2 studying the drugs safety and efficacy in 750 mild Alzheimers patients; and an open-label extension trial (CREAD OLE) examining long-term drug treatment in 149 Alzheimers patients. Unfortunately, in January 2019, Roche discontinued all CREAD trials due to the interim analysis showing crenezumab was unlikely to meet the primary endpoint of improving cognition.

Genentech (a subsidiary of Roche) is partnering with AC Immune to develop the anti-tau antibody drug RO7105705 (also called RG6100 and MTAU9937A), which recognizes tau tangles and is meant to block their spread between cells. An active Phase 2 trial involving 457 prodromal to mild Alzheimers patients is studying the drugs safety and effect on cognitive function.

AbbVies humanized antibody drug ABBV-8E12, which targets the tau protein, is being evaluated for its safety and efficacy in an active Phase 2 trial involving 400 early-stage Alzheimers patients. An extension study to study the drugs long-term safety and tolerability is currently enrolling patients from the Phase 2 study (NCT02880956) by invitation.

Avid Radiopharmaceuticals, a wholly-owned subsidiary of Eli Lilly, is developing the PET imaging agent 18F-AV-1451 (also called Flortaucipir F 18 or F 18 T807), a molecule that binds to the tau protein, allowing researchers to study tau in living patients. There are multiple Phase 2 or Phase 2/3 trials studying the imaging agents safety and efficacy, with five Phase 2 trials currently recruiting or not yet recruiting: one to evaluate the agents safety and tau binding via PET imaging in 250 healthy, Alzheimers, traumatic brain injury and depression patients; one (ADRC proj 1) to compare tau tangles in the brain with cerebrospinal fluid CSF biomarkers and cognitive status in 80 Alzheimers patients; one (DIAN Project, AV ADAD) to study the presence of tau tangles in the brain and cognitive status in 130 adults; one to study the uptake and binding in 80 older HIV-positive adults with and without HIV-associated neurocognitive disorders and HIV-negative age-matched controls; and one (Protocol Z) to study tau and amyloid lesions in the brains of 80 APOE4+ adults with normal cognition or early-stage symptomatic Alzheimers.

Neurotrope Bioscience is developing bryostatin-1, a small molecule that activates protein kinase C (PKC), a protein that is important for learning and memory. This drug stimulates synapse repair and growth, activates -amyloid degrading enzymes and prevents tau tangle formation and neuron death. A Phase 2 trial evaluating the safety and efficacy of bryostatin-1 in 147 moderate to severe Alzheimers patients showed positive results: the lower (20 g) dose improved cognition and the ability to care for oneself. This prompted a second Phase 2 trial to study the drugs safety and efficacy at the lower dose in 108 moderately severe to severe Alzheimers patients.

Unfortunately, Neurotrope announced that the second Phase 2 trial did not show statistically significant improvement in memory, indicating it did not meet its primary endpoint of a change in the Severe Impairment Battery (SIB) test total score from baseline to week 13.

EIP Pharma is pursuing a small molecule called neflamapimod (VX-745) that inhibits an enzyme, called p38 MAPK, found in the neurons that is involved in inflammation and possibly -amyloid toxicity. Neflamapimod previously showed clinical activity in rheumatoid arthritis patients before being licensed to EIP Pharma. They are currently conducting a Phase 2b efficacy study (REVERSE-SD) in 161 participants with mild Alzheimers. A Phase 3 study is scheduled to start in the third quarter of 2020. Another Phase 2 trial is recruiting 40 Alzheimers patients to study neflamapimod on brain inflammation.

Actinogen Medicalis studying a drug called xanamem, which inhibits a cortisol-producing enzyme in the brain, ultimately blocking local production of cortisol, known as the stress hormone. While blood cortisol levels tend to rise with age, its particularly raised in patient with certain diseases, such as Alzheimers. Long-term high cortisol levels can be toxic to brain neurons, so preventing cortisol production in the brain may help slow cognitive decline and -amyloid plaque formation. After assessing xanamems safety and dosing in two Phase 1 trials, a Phase 2 trial (XanADu) assessed the drugs safety and efficacy in 186 early-stage Alzheimers patients.

Boehringer Ingelheims drug BI 425809 is a glycine transport inhibitor designed to regulate signaling in the brain that contributes to cognitive impairment. An active Phase 2 trial is studying the safety and effect on cognition of multiple dosages of the drug in 611 Alzheimers patients.

Neurocentriais developing a dietary supplement called MMFS, which contains a molecule called L-threonic acid magnesium salt (L-TAMS) that increases synapse density in portions of the brain needed for memory and executive functioning, such as the prefrontal cortex and hippocampus. Two previous studies showed improved cognition in mild to moderate Alzheimers patients, prompting the active Phase 2 trial that is recruiting 12 mild Alzheimers patients to examine the drugs safety and effect on cognition.

Alkahestis studying intravenously administered plasma-derived product called GRF6019, which is isolated from human plasma (a component of the blood) that has been shown to enhance neurogenesis and improve learning and memory in animals. Matching donor and patients blood types is not needed because the donor-specific antibodies (called immunoglobulins) are removed. A Phase 2 trial in 40 mild to moderate Alzheimers patients studied the safety and feasibility of GRF6019. Another Phase 2 trial is currently recruiting 20 severe Alzheimers patients to study the safety, tolerability, and cognitive benefits of the drug.

Suven Life Sciences drug SUVN-502 specifically inhibits a certain serotonin receptor (called 5-HT6), which is thought to improve cognition and memory. SUVN-502 in combination with donepezil and memantine was shown to increase the concentration of neurotransmitters, like acetylcholine. An active Phase 2 trial is testing the effect of this triple combination therapy on cognition in 563 moderate Alzheimers patients. An expanded access program is also available for eligible patients to receive the drug without being evaluated for safety and efficacy.

Neurim Pharmaceuticalsis taking a different approach by developing a drug, called piromelatine, that binds to and activates melatonin and serotonin receptors in the brain, promoting sleep and therefore neuroprotective effects. This drug was safe and promoted deeper and more REM sleep in a Phase 2 clinical trial in adults with insomnia. Given the link between sleep and Alzheimers, Neurim decided to study piromelatines effects on cognition in 500 mild Alzheimers patients in an active dose-ranging Phase 2 trial.

Eisai, in collaboration withPurdue Pharma, is studying their orexin receptor antagonist drug lemborexant in a Phase 2 trial involving 62 mild to moderate Alzheimers patients. The orexin receptor is involved in the regulation of sleep. Lemborexant binds to the orexin receptor, preventing orexin from binding, which should decrease wakefulness and promote falling and staying asleep naturally. Sleep, especially at appropriate hours, is troublesome for Alzheimers patients whose circadian rhythms tend to be dysregulated.

Phase 2/3

Novartis has partnered with Amgen and the Banner Alzheimer's Instituteto pursue Novartis drug umibacestat (CNP520), which inhibits BACE1, an enzyme involved in -amyloid production. After a successful Phase 2 trial safety study in 124 healthy elderly patients, there were two Phase 2/3 trials: one (Generation S1) to test the efficacy of CNP520 against an investigational immunotherapy drug (CAD106, a vaccine against a fragment of the -amyloid protein) in 481 non-symptomatic older patients with two copies of the APOE4 gene; and one (Generation S2) to test the drugs effect on cognition and underlying Alzheimers pathology in 1145 non-symptomatic older patients with at least one APOE4 allele and elevated brain -amyloid levels.

Unfortunately, both Phase 2/3 trials were discontinued in July 2019 due to worsening cognitive function seen during interim data analysis. As umibacestat was meant to delay the onset of symptoms, participants in the study will discontinue the investigational treatment and discuss further treatment options with their doctors.

TauRx Therapeutics drug TRx0237 (also called LMTX) is their second-generation tau protein aggregation inhibitor, which aims to both dissolve existing tau tangles and prevent new tangles from forming. Two previous Phase 3 clinical trials studied the safety and efficacy of high doses (150-250 mg/day) and a low dose control (8 mg/day) of the drug in 800 mild and 891 mild to moderate Alzheimers patients. Surprisingly, they found that the low dose was as beneficial as the higher doses, prompting a current Phase 2/3 trial (LUCIDITY) recruiting 375 early Alzheimers patients studying TRx0237 at low doses (8 and 16 mg/day). An expanded access program is also available to provide the drug to patients who have previously participated in a TauRx clinical trial but do not qualify for an ongoing trial.

Axsome Therapeutics is pursuing a treatment for agitation associated with Alzheimers and have been granted fast track status for their drug AXS-05, which combines dextromethorphan and bupropion. Dextromethorphan (called DM and commonly known as an over-the-counter cough suppressant) inhibits serotonin and norepinephrine transporters and the NMDA receptor at high doses. Bupropion increases the bioavailability of dextromethorphan and inhibits norepinephrine and dopamine reuptake. A Phase 2/3 trial (ADVANCE) is currently recruiting 435 Alzheimers patients to study the safety of AXS-05 and its effect on agitation.

Phase 3

Eli Lilly has a Phase 3 anti--amyloid antibody drug called solanezumab (LY2062430), which binds to soluble -amyloid monomers. The primary endpoints of trials involving this drug is to slow memory and cognitive decline. The drug is associated with 11 listed trials, including an active Phase 3 trial (A4) involving 1150 not yet symptomatic adults with evidence of amyloid plaque build-up in their brains, and a currently recruiting Phase 2/3 large collaboration trial (DIAN-TU) comparing solanezumab and gantenerumab in 490 non-symptomatic adults known to have an Alzheimers disease-causing mutation. This collaboration includes Eli Lilly, Roche, Avid Radiopharmaceuticals, Janssen, Alzheimers Association, National Institute on Aging (NIA), Accelerating Medicines Partnership (AMP), and Washington University School of Medicine.

Two Phase 3 trials (EXPEDITION and EXPEDITION 2) were completed previously and involved 1040 Alzheimers patients each. Although there was no difference in cognition between the treated and placebo groups, patients with mild Alzheimers did show slower cognitive decline compared to placebo, prompting further studies.

Unfortunately, the next three Phase 3 trials (EXPEDITION 3, EXPEDITION EXT and EXPEDITION PRO) were terminated due to lack of meeting primary endpoints, including slowing cognitive decline, and insufficient evidence that solanezumab would likely demonstrate a meaningful benefit to patients with prodromal Alzheimers.

Roche is currently investigating gantenerumab, an anti--amyloid antibody drug that binds and neutralizes -amyloid plaques. Gantenerumab, brought back after failing in previous clinical trials, is involved in four Phase 3 trials: two active trials studying the drugs effect on cognitive function in 799 prodromal and 389 mild Alzheimers patients; and two currently recruiting trials studying the drugs effect on cognition in 760 early Alzheimers patients each.

Eisai, in collaboration with Biogen, is studying their small molecule BACE1 inhibitor elenbecestat (also called E2609) in two Phase 3 trials (MISSION AD1 and MISSION AD2) currently recruiting 950 early-stage Alzheimers patients each. Inhibiting BACE1 is thought to interfere with -amyloid production.

Unfortunately, the companies announced that they were discontinuing their MISSION AD1 and AD2 Phase 3 trials on September 13, 2019. The decision was made based on results from a safety review that showed an unfavorable risk-benefit ratio of elenbecestat.

Avid Radiopharmaceuticals and Eli Lilly reported positive results earlier this year from their Phase 3 trial on the tau-binding PET imaging agent flortaucipir F 18 (18F-AV-1451 or Tau imaging agent) in Alzheimers patients. The trial met its two primary endpoints, successfully predicting both the disease-related role of tau in the brain and an Alzheimers diagnosis. PET imaging was performed on 156 end-of-life patients with cognition ranging from normal to dementia, with 67 of these patients being evaluated post-mortem. Flortaucipir could significantly detect Alzheimers-related changes in the brain, including both tau and -amyloid plaque densities. Being able to accurately image and diagnose Alzheimers patients is a critical component in understanding the disease and being able to manage it. There are currently 33 studies listed on clinicaltrials.gov for flortaucipir and Alzheimers.

AZTherapies, Inc. is studying the combination drug ALZT-OP1, which consists of the inhaled drug cromolyn and oral drug ibuprofen, both of which are anti-inflammatory. Inflammation in the brain is thought to trigger neuronal death, which causes progressive brain damage. Cromolyn was also shown to prevent -amyloid aggregation in one study. A Phase 3 trial (COGNITE) is currently studying the effect of this combination drug on cognitive decline in 620 early-stage Alzheimers patients.

ACADIA Pharmaceuticals drug pimavanserin (previously called ACP 103) is a selective serotonin inverse agonist (SSIA), meaning it both binds to serotonin receptor subtype 5-HT2A and blocks serotonin signaling. Following a few Phase 2 trials specifically in Alzheimers patients, there are currently three recruiting Phase 3 trials for a broader range of dementia patients: an efficacy study examining pimavanserins ability to prevent relapse of dementia-related psychosis symptoms in 356 dementia patients, a safety study in 300 patients with neurodegenerative disease, and an open-label extension study examining the drugs long-term safety in 300 patients with neurodegenerative disease who previously participated in another pimavanserin clinical trial by ACADIA.

Intra-Cellular Therapiesis developing lumateperone (ITI-007), a molecule that simultaneously affects serotonin, dopamine and glutamate signaling, which play important roles in multiple mental illnesses. Following a Phase 1b/2 study, they recruited 177 dementia patients, including Alzheimers patients, for a Phase 3 trial studying the safety and efficacy of the drug for reducing agitation.

However, the Phase 3 trial was terminated early due to interim data analysis indicating lumateperones lack of efficacy.

AVANIR Pharmaceuticals drug AVP-786 combines two approved drugs: deuterated dextromethorphan (d6-DM), which has better bioavailability and less side effects than regular DM, and an ultra-low dose of quinidine, which slows the metabolism of d6-DM by inhibiting an enzyme (CYP 2D6) that breaks down d6-DM. AVP-786 is a second-generation version of Nuedexta (formerly AVP-923), which is currently approved to treat pseudobulbar affect (PBA). Currently, there are four recruiting or active Phase 3 trials studying the safety and efficacy of AVP-786 in treating agitation in Alzheimers patients: one recruiting 412 Alzheimers patients with moderate to severe agitation worldwide, one active study involving 522 Alzheimers patients in the US, one completed study involving 410 Alzheimers patients in the US and a long-term extension study recruiting 700 patients who have completed previous clinical trials of AVP-786 by Avanir.

Otsuka Pharmaceutical Co.and Lundbeck are collaborating to develop brexpiprazole (brand name Rexulti) for treating agitation and behavioral symptoms in Alzheimers patients. Rexulti, which binds to and activates a particular dopamine receptor (D2), is currently FDA approved to treat schizophrenia and as an add-on treatment for major depression disorder. Two Phase 3 trials examining brexpiprazole at either fixed or flexible doses in a total of 703 Alzheimers patients showed reduced agitation compared to the placebo. They are currently recruiting for three Phase 3 trials: one evaluating the safety, efficacy, and tolerability in 225 Alzheimers patients with dementia-associated agitation in the US; one studying long-term treatment in 157 Alzheimers patients with dementia-associated agitation in Japan; and a 12-week extension study for 250 Alzheimers patients with dementia-associated agitation who were previously enrolled in other Otsuka trials studying brexpiprazole. They are also recruiting for a Phase 2/3 study in 407 Alzheimers patients with dementia-associated agitation in Japan.

Merck Sharp & Dohme Corp., a subsidiary of Merck, is studying their FDA approved drug suvorexant (previously called MK-4305, brand name Belsomra) to treat insomnia in Alzheimers patients. Currently approved for insomnia patients, the small molecule drug works by inhibiting the orexin receptor in the signaling system involved in wakefulness. Their Phase 3 trial studying suvorexants safety and efficacy at improving sleep in 285 Alzheimers patients and patients with insomnia concluded in October 2018, but results have not been posted yet.

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Alzheimer's Disease Insight Report: Current Therapies, Drug Pipeline and Outlook - BioSpace

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Anti-Ageing Drugs Market 2019 In-depth Analysis by Leading Players: Nu Skin, BIOTIME, Elysium Health - Daily Research Chronicle

Once weve conquered our diets, instituted a regimen of exercise and saunas and cold plunges, doused ourselves in NMN and resveratrol and Metformin and benign viruses, quit smoking and cut down our drinking and remembered to wear our seatbelts, theres one main obstacle remaining in the way of an extra-long and healthy life: our guilt.

Whether its hard-wired or a result of societal expectations, we tend to feel that old farts should not outstay their welcome. Leave some room for future generations, we grumble under our breath, out of earshot of elderly relatives. Youre already taking up too much of the housing stock, making it near-impossible for millennials to buy homes. You want to bankrupt Social Security and Medicare too?

Just last month, Ezekiel Emanuel, the chair of the University of Pennsylvanias department of medical ethics (and a chief architect of Obamacare) confirmed that he stood by his controversial 2014 essay: Why I hope to die at 75. Despite the onslaught of anti-aging research, Emmannuel (now 62) said his main arguments still held water: That people in their 80s who were still vigorous were not doing meaningful work; that authors above 75 were not producing brand-new books but simply re-ploughing old furrows.

Let's leave aside the fact that's a pretty weird metric to judge the worth of a life -- sorry, grandma, time to go, you're not doing meaningful work or writing new books! Emanuel's argument ignores what biologists like Sinclair are telling us. The more we age in good health, the more useful we will be.

Sinclair, as you might expect, could not disagree with Emanuel more. First of all, he says, lets assume everyone stopped dying of age-related causes tomorrow and they wont, even under the most extreme anti-aging regimen. But if they do, thats only 100,000 extra people per day sticking around. (Around 150,000 people die every day, roughly two-thirds of them from age-related causes.)

Compare that to the worlds current growth rate. More than 350,000 babies arrive every 24 hours. Earth's population is growing because of the size of the average family in the developing world, not because more people are living longer. The main way to bring it down is to educate more women and move more families into cities where, by the way, we shouldnt blame Baby Boomers for the lack of housing. We simply need to build more.

Total human population should level off at around 11 billion around the time your century dawns, whether or not the aged continue to die. And as for the threat of climate change well, perhaps the older generation will start to pay more attention when theyre actually going to live with the effects themselves. Or when they have to look their great-great-grandchildren in the eyes and explain their inaction.

Secondly, a healthy longevity boom would actually take an enormous burden off the healthcare system. Reducing just one of the major killers like heart disease, even by 10 percent, could savetrillions of dollars, money that can then be reinvested in medical research or just returned to patients in the form of lower costs. And thats the whole point of treating aging as the ultimate disease, the one that effectively produces all the others. (For example, Sinclair writes, smoking makes lung cancer five times more likely, but just living from 20 to 70 increases your chances of getting the disease a thousandfold, even if youve never sucked on a cancer stick.)

Aging is by far the biggest risk factor in any disease, by an order of magnitude, Sinclair says; having volunteered in nursing homes with his wife, he knows whereof he speaks. Dont delude yourself: Getting old and getting sick is not fun, for you or for your family. So I believe we have an obligation to preserve our health for as long as possible.

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The end of aging: Are you ready to live to 150? - Mashable

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Rocket Pharmaceuticals (RCKT) is a best in class gene therapy company with five shots on goal and strong data to support its current valuation. The two largest assets, RP-L102, a lentiviral gene therapy for Fanconi Anemia and RP-A501, an AAV gene therapy for Danon Disease are each worth multiples of the current share price, if successfully commercialized. The management team is highly experienced and have successfully commercialized many products at predecessor companies. The board of directors are both experienced and proven money makers on wall street in the world of biotech. The shareholder base is strong with top quality investors and the company has sufficient cash on the balance sheet for at least two years, during which multiple value drivers will report out. Commercialization of the most advanced products could occur in the 2021 timeframe. While never an investment attribute alone, I would note that there have been multiple acquisitions in gene therapy during the last 18 months (AVXS, ONCE) at eye-watering valuations and large cap pharma is struggling to find pipeline assets and return on productivity for internal pipeline assets remains at a multi decade low.

This report provides an overview of the company and details of the most advanced product in development, RP-L102 for Fanconi Anemia, as this is the primary focus for investors currently. The company's largest pipeline asset, RP-A501 for Danon Disease will become a focus for investors during 2020.

RCKT has Five Programs. Four will be in the Clinic in 2019

Source: Company data

Pipeline has > $1bn in Revenue Potential

Source: Company data, my estimates

Plenty of Catalysts Anticipated During Next 12 months

With five assets either in, or almost in the clinic, there are multiple catalysts expected during the next twelve months.

Source: Company data, my estimates

The company finished 2Q 2019 with $257 million of cash on its balance sheet and during the last 12 months the company burnt $66.5 million of cash. This is expected to increase during 2020 and 2021 as multiple pivotal trials start and consensus forecasts suggest that the company will spend $99 million in 2020 and $98.5 million in 2021. Therefore the company has sufficient cash on its balance sheet for approximately 2.5 years during which time, there will be multiple clinical catalysts that will hopefully drive the share price higher, allowing the company to raise additional equity in late 2020 to fund the company to break even in the 2023 timeframe. In the current environment, investors need to avoid any company that requires substantial financing.

Rocket Pharmaceuticals trades with a market capitalization of just $546 million. As of June 30, 2019, the company had cash of $ 258 million and debt of $ 46 million. Compared to other companies in the gene therapy space, RCKT trades at a significant discount. The company is well capitalized with approximately two years of cash on the balance sheet and there are a number of value creating catalysts during the next 12 months. Additionally, whilst never a reason to solely own a biotech company, I would note that there have been a number of acquisitions in the gene therapy space during the past few years. Large-cap pharma and biotech is short on products and long on cash and they need to make acquisitions.

Selected M&A in the Gene Therapy Sector: 2016-2019

Source: Bloomberg, Company data

RCKT is currently covered by 8 Wall Street Sell Side analysts, as shown below. Notably, Large banks including Goldman Sachs, Jefferies, JP Morgan, Morgan Stanley, Citi and Barclays Capital are all missing. As the company evolves into a commercial company during the next several years, it is likely that some of these brokers will initiate coverage of the stock, thereby improving liquidity.

Source: Bloomberg

As with all biotechnology stocks, there are significant risks associated with this investment and under a worst case outcome, there is 100% downside. The most obvious risk is that the pipeline products fail in clinical development. While Rocket has five assets in its pipeline, and success in any one of these is likely enough to justify the current valuation, negative clinical trial data would clearly have a negative impact on the company's share price. Under the outcome that all five pipeline assets fail in development, the stock is likely worth zero.

We are also in an uncertain political environment with an election looming in 2020. It is unlikely that either party will be arguing for higher drug prices and biotech stocks often underperform during these periods. Investors can mitigate this risk by being short a number of lower quality biotech companies and long a number of higher quality biotech companies. In my opinion, investors need to be long biotech stocks that are financed through 2021 and have multiple catalysts during the next 12 months. Being short companies in the opposite camp likely generates a good return as well.

Currently this company is not really exposed to foreign exchange rate or interest rate risks but these factors may become relevant in years to come.

This report will start with a primer on exactly what gene therapy is and then a detailed analysis of Rocket's lead asset where clinical data has been evolving during the last 24 months.

Gene therapy refers to technologies that can insert genes into cells, thereby expressing the proteins encoded by the genes. Gene therapies consist of two key elements - the gene of interest, and a vector that carries the gene into the host's target cells. Over the years a number of vectors have been used, although most efforts now employ viruses to carry the target genes. In creating a gene therapy, most of the viral genome is replaced by the therapeutic gene of interest. This eliminates the ability of the virus to replicate and cause disease, and permits relatively large target genes to be carried. The manipulated genome is inserted into a viral vector and when the virus is given to a patient, it is taken up by the patient's cells where it delivers its DNA to the nucleus. The cell then makes the target protein using the new gene as if it were encoded by the cell's own genetic material. Importantly, this process of gene transfer can be conducted ex vivo or in vivo depending upon the application.

Although gene therapy has the potential to treat a wide range of conditions, orphan monogenic diseases are particularly well suited for this approach. There are a number of scientific, economic, and logistical attributes of severe, monogenic orphan diseases that make them ideal candidates for the development of gene therapies by small biotechnology companies. First, by their nature as monogenic diseases, their causes are defects in a single gene. The pathogenesis of the disease is often well understood, and its treatment can be straightforward: by placing a functional copy of the gene in affected tissues, the disease process can be functionally cured/halted. Second, as orphan disorders affect a relatively small number of patients, on the order of several thousand individuals, the clinical trial programs can be conducted in tens of patients, rather than thousands. Such trials are less expensive to run and the logistics are within the capabilities of even small biotech companies. Third, most monogenic orphan diseases have no currently available disease altering therapies. Therefore the unmet need is high and any safe and effective therapy will likely be embraced. Fourth, the FDA has been flexible in its requirements for licensure in severe orphan diseases, routinely granting accelerated approvals based on surrogate markers that are reasonably likely to predict clinical benefit. Finally, innovative, and effective orphan therapies still have pricing flexibility in most worldwide markets such that companies can achieve attractive risk-adjusted returns on their research and development investment. Therefore, the orphan business model is well established and has repeatedly generated high returns for small cap biotechnology companies.

Rocket is building a comprehensive gene therapy technology platform to address serious, rare diseases. Rocket is developing both ex vivo lentiviral-based gene therapy technologies as well as adeno-associated virus (AAV) technologies to be used in vivo. Rocket also has early preclinical efforts in gene editing such as CRISPR/Cas9 (Clustered Regularly Interspaced Short palindromic Repeat/CRISPR-associated protein-9 nuclease) in its pipeline.

RCKT is Focusing on both In Vivo and Ex Vivo Gene Therapies

Source: Company data

What is a Lentiviral Vector?

Lentiviruses are a genus of retroviruses that includes the human pathogen human immunodeficiency virus (HIV). Like all retroviruses, lentiviruses are RNA viruses that encode reverse transcriptase (RT). Once a virion infects a cell, RT converts the virus' RNA genome into a DNA copy. This DNA copy is then integrated into the host genome using the virally encoded integrase. Once integrated into the host genome, the virally encoded genes are expressed and copied alongside host genes using the normal host gene expression and replication machinery. Lentivirus-based gene therapy approaches seek to co-opt the viral integration process to stably introduce genes of therapeutic interest into the human genome. Unfortunately, every insertion event is associated with a theoretical risk of causing disease (insertional mutagenesis) due to disruption of the host genome at the site of integration. As a result, lentiviral gene therapy programs take several steps to limit the ability of the virus to generate unnecessary insertion events.

Lentiviral (and retroviral more generally) gene therapy is most often deployed in an ex vivo process whereby cells are removed from the body, transfected with a lentivirus encoding the gene of interest, and then reintroduced into the patient. In Rocket's programs, it is transducing hematopoietic stem cells (HSCs) isolated from patients with defined monogenic diseases in order to insert a normal copy of the gene that is defective in these patients. The transduced HSCs are then infused back into the patient so that they will engraft. Although historically the patient's native hematopoietic system is ablated to improve engraftment, Rocket and its academic collaborators have pioneered a lentiviral approach that requires no or minimal chemotherapy.

HSCs are a self-renewing cell type that reconstitutes the patient's hematopoietic system, thus providing permanent, life-long expression of the normal gene from this one-time treatment. Because HSCs differentiate to form a variety of terminal cell types, this general approach is potentially applicable to a variety of genetic diseases in a modular, repeatable fashion. The ex vivo use of HSCs rather than in vivo treatment of all cells dramatically reduces the number of insertion events required to generate a therapeutic effect thereby reducing the risk of insertional mutagenesis. In addition, Rocket's use of the patient's own cells (an autologous transplant) is an important attribute of lentiviral gene therapy, as this should avoid some of the serious immune complications associated with allogeneic transplants such as graft-versus host disease (GVHD), which require management with harsh immunosuppressive therapies and can be fatal.

The lentiviral vector Rocket uses is based on the HIV virus. The vector takes advantage of the virus' natural ability to integrate into the host genome in both dividing and nondividing cells in order to efficiently deliver the chosen genetic payload. However the vector has been modified in a number of ways to render it nonpathogenic. Virtually all the viral genes have been removed to make room for the transgene and eliminate the virus' ability to replicate. The infectious viral particles are generated by co-transfecting producer cells with separate plasmids containing the "gutted" viral backbone and transgene, the viral capsid proteins and viral polymerase to make viral RNA from the DNA plasmid, reverse transcriptase to make DNA from the virus' RNA, and VSV - a pantropic envelope protein that allows infection of a variety of human cell types (not just CD4+ T cells). This results in the production of infectious viral particles carrying the viral RNA, reverse transcriptase protein, and viral integrase protein. When the virus infects target cells, it is thus able to undergo the process of reverse transcription and integration into the genome, but because the natural viral genes are not present, it can only undergo this single cycle of transduction and cannot replicate or infect other cells. To make doubly sure of this, the terminal ends of the viral genome are also modified to be "self-inactivating," so that they would no longer be recognized for excision even if the necessary viral proteins were to become present in the cell. Thus, the transgene is stably inserted into the host genome. For those readers who would like additional information on lentival gene therapy I recommend you reed this report available on PubMed. Kenneth Lundstrom does a great job discussing the pros and cons of each approach.

AAV is a naturally occurring non-pathogenic virus that is not known to cause any disease in humans. AAV has a number of advantages as a delivery vehicle for in vivo applications of gene therapy. AAV vectors do not replicate inside the host cell, preventing their spread to unintended tissues, and they typically integrate at a very low level into the host cell's genome, reducing the risk of insertional mutagenesis. Moreover, cellular tropism can be effectively modulated by using the natural tropism of different AAV serotypes, synthetically engineering the AAV capsid, and/or altering the transgene's promoter sequence. AAV vectors are also able to transduce non-dividing cells (such as RPE cells in the retina), and once incorporated into a host cell, they can drive the expression of a therapeutic protein for years. Last, AAV vectors can carry a good amount of genetic material, up to 4.5kb permitting them to target a range of indications. Since AAVs are non-replicating and generally non-integrating, the viral genome is typically not copied when an infected cell divides. Therefore, there is a theoretical risk that the efficacy of AAV based therapy in dividing cells could wane as an increasing number of divisions occurs.

A large number of clinical trials of AAV gene therapy are either under way, or have been completed. Applications have been diverse, ranging from hemophilia to REP65-mediated blindness and Parkinson's disease. AAV is versatile, and can be delivered through a number of routes of administration including intravenous, intramuscular, intrapleural, intravitreal, subretinal, and intracranial. For example, in lysosmal storage disorder (LSD) and hemophilia, AAV gene therapies are delivered systemically via intravenous (i.v.) route of administration and liver cells are transduced. In more localized diseases such as retinal dystrophy, choroideremia, X-linked retinoschisis (XLRS), the gene therapies are directly injected into the eye. In advanced Parkinson's disease, the gene therapy candidate is injected intracranially.

Fanconi Anemia - A rare disease with limited treatment options and a median survival of 29 years

Fanconi Anemia (FA) is a rare autosomal recessive DNA repair-deficiency syndrome characterized by aplastic anemia and progressive bone marrow failure. Though FA is a blood disorder, broad complications across a number of organ systems are associated with the syndrome such as defects of the eyes, ears, bones, kidneys and the heart. Perhaps most important, up to 30% of patients with FA develop leukemia, myelodysplastic syndrome (MDS), and or solid tumors at ages between 5 and 15. The median life span for FA patients is approximately 29 years.

Disease Progression: Unmet need for a treatment for FA

Source: Kutler et al, Blood 101:1249, 2003

FA is a complex disease with abnormalities in at least 18 genes associated with the disorder. These genes typically belong in the FANC gene family (FANC A-G, FANC CJ, FANC CL, and FANC M). The FANC gene family is associated with the DNA repair pathway. A mutation in any of these genes renders cells unable to properly repair damaged DNA. FANC A, B, C, E, F, G, L and M) form a nuclear complex termed the FA core complex. The FA core complex is required for monoubiquitination of the FANCD2 protein. Monoubiquintination of the FANCD2 protein allows for FANCD2 to translocate to sites of DNA damage to facilitate BRCA2/FAN CD1 and FANC E function in homologous recombination for DNA repair. Due to mutations in this DNA repair machinery, FA patients are simply unable to repair DNA damage that occurs naturally as cells divide, are exposed to mutagens, etc. Depending upon the exact DNA insult that occurs, unrepaired DNA can lead to abnormal cell death (most commonly) or uncontrolled cell growth. The abnormal cell death in turn creates FA's characteristic anemia and other organ defects. In other cases unrepaired DNA damage leads to uncontrolled cell growth and the development of a leukemia, tumor, or MDS. While it is extremely uncommon for any one DNA insult to generate cancer rather than cell death, DNA damage is occurring constantly within millions of cells in any human. Therefore, with millions of potentially oncogenic unrepaired mutations occurring it is unsurprising that FA patients have a significantly increased risk of developing cancer.

Approximately 60% of FA cases are due to mutations in the FANC A gene (the specific genetic abnormality that Rocket's lead program addresses). Approximately half of FA patients are diagnosed prior to age 10 while about 10% are diagnosed during adulthood. The remaining ~40% of FA patients are diagnosed during their teenage years. Birth defects such as undeveloped skull, eyes, or abnormalities in radial bones, kidney, skeleton, or skin pigmentation often facilitate early diagnosis. The definitive test for FA is a chromosome breakage test using crosslinking agents (dieposxybutane or mitomycin C) in isolated patient blood cells. While blood cells from healthy volunteers are able to correct most of the crosslinking agent induced DNA damage, FA patients' cells are incapable of correcting the damage from DEB or MMC treatment. Other methods of diagnosis include the use of molecular genetic testing on the 18 genes associated with FA such as sequencing analysis. The only curative therapy for FA is hematopoietic stem cell transplantation (HSCT) (there is good information on this here).

However, HSCT has a number of notable difficulties and complications. For one, it can be difficult to find a matched donor so that the transplant can be performed with a reasonable likelihood of success. Even when a suitable match is found, HSCT confers a high degree of morbidity and mortality, particularly in FA patients. Recent advances in conditioning regimens and supportive care have reduced treatment-related mortality from 38% or higher to 5-10% at most centers; nonetheless, such rates of death due to the procedure are notable. Moreover, HSCT can have major short and long-term complications including veno-occlusive disease, infections, infertility, secondary malignancies and graft-versus-host disease. GvHD can be particularly problematic and can evolve into a life-long condition causing serious damage to the lung, skin and mucosa. In severe cases GvHD can also be deadly. Conditioning chemotherapy is also inherently mutagenic and is therefore associated with additional risk of tumors developing post-transplant (secondary malignancy). FA patients are unable to repair these mutations that occur throughout the body during conditioning. Therefore HSCT confers a particularly high risk of secondary malignancy to FA patients. For example, the chance of an FA patient developing a new malignancy such as squamous cell carcinoma is estimated to be ~4x higher post HSCT. Thus, while HSCT is curative of FA's characteristic hematological manifestations, "cured" patients remain at an elevated risk of experiencing morbidity/mortality.

There can be spontaneous improvement in a small fraction of FA patients due to somatic mosaicism. Somatic mosaicism results from the spontaneous, random mutations that occur during normal cell division and proliferation. The cells clonally derived from the initial mutant cell have a different genotype than their neighbors. Somatic mosaicism has been reported in patients with FA. In cells of FA patients, the reversion of a pathogenic FA allele to a functional wild type allele confers a survival advantage on the cell vs. its non-reverted sibling cells. The cell(s) with the wild type reversion exploit this survival advantage to gradually populate the bone marrow. Up to 10-15% of FA patients develop somatic mosaicism resulting in disease stabilization or even improvement in bone marrow function for a prolonged period of time. This observation supports the theory that a very small percentage of corrected cells is sufficient to change the clinical course of FA. Somatic mosaicism therefore provides a rational as to why gene therapy may be successful in the treatment of FA patients and RCKT refer to somatic mosaicism as natural gene therapy.

Somatic mosaicism in FA leads to stabilization/correction of blood counts, in some cases for decades. This uncommon variant results from a reverse mutation and demonstrates that a modest number of gene-corrected hematopoietic stem cells can repopulate a patient's blood and bone marrow with corrected (non-FA) cells.

Source: Soulier, J., et al. (2005) Detection of somatic mosaicism and classification of Fanconi anemia patients by analysis of the FA/BRCA pathway. Blood 105: 1329-1336

Commercial launch likely in 2021/22 with >$1bn potential.

RP-L102 is a lentiviral vector that employs the phosphoglycerate kinase (PGK) promoter to express the FANCA gene. Expression is further facilitated by inclusion of the Woodchuck Hepatitis virus posttranscriptional regulatory element (WPRE). RP-L102 was licensed from the Centro de Investigaciones Energeticas, Medioambientales Y Technologicas (CIEMAT) in Madrid, Spain. CIEMAT is the Investigational Medicinal Product Dossier (IMPD) sponsor of the ongoing Phase I/II FANCOLEN-1 study of RP-L102 in patients with FA. Rocket is entitled to the data and commercial rights to the drug product generated under the CIEMAT sponsored IMPD.

RP-L102 gene therapy could have significant advantages over HSCT for FA patients. Perhaps the most notable advantage is that RP-L102 is being developed by Rocket and its academic collaborators without the use of bone marrow conditioning with chemotherapy agents. In contrast, all HSCT protocols require chemotherapy conditioning. The lack of conditioning confers a number of advantages. For example, without the use of chemotherapy agents, patients do not need to be hospitalized, and treatment can occur outside of a transplant-unit. Most important, FA patients have a diminished ability to correct damage to genetic material like that typically caused by chemotherapeutic agents. Therefore, by avoiding chemotherapy conditioning, the FA patients should not have an increased risk of head and neck cancer or leukemia. Moreover, because of their toxicities in FA bone marrow transplants are indicated specifically for patients with signs of bone marrow failure. RPL102 should enable treatment earlier in the disease course, well before bone marrow failure. This will allow patients to avoid the risks associated with the low blood counts of bone marrow failure, including anemia, infections and hemorrhages.

Gene Therapy Value Proposition: Early, Low-toxicity Intervention to Prevent Hematologic Failure

Source: Company data

RCKT recently presented data at the American Society of Hematology of the first four patients treated with RCKT's lentivial gene therapy for FA.

Bone Marrow Engraftment: Increasing Levels Provide Evidence of Potential Survival Advantage of Gene-Corrected FA Cells

Source: Company data

Increases of Corrected Leukocytes Support Restoration of Normal Bone Marrow Function Consistent with Mosaic Phenotype

Source: ASH 2018

Functional Correction of Bone Marrow

Source: ASGCT 2018

RCKT is a best in class gene therapy company with multiple shots on goal. During the next 12 months, data will likely emerge on many of these assets and if successful, should lead to considerable upside. This report focuses on the company's lead asset and data that has been presented to date is extremely supportive of a likely successful outcome, which would lead to considerable upside. As with all biotech investments, there are obviously significant downside risks and the worst case outcome for this stock is that it ends up at zero. However, with 5 pipeline assets in development, this risk is lower than biotech companies that are reliant upon a single driver of value.

Disclosure: I am/we are long RCKT. I wrote this article myself, and it expresses my own opinions. I am not receiving compensation for it. I have no business relationship with any company whose stock is mentioned in this article.

Read more:
Blast Off With Rocket Pharmaceuticals - Seeking Alpha

Stem cells can do a lot of things - they can heal damaged tissue, reduce inflammation and restore function to damaged tissues. Did you know that stem cells can also improve your skin's quality and reduce the signs of aging? Innovations Stem Cell Center offers fat stem cell therapy for not only a wide array of medical conditions, but also for powerful anti-aging benefits.

How Can Stem Cell Therapy Improve Skin Quality?

Stem cells can help improve skin quality by helping to heal tissues that have been damaged by:

Aging. The aging process causes the breakdown of skin cells and skin quality, leaving the skin looking dull and dirty. Skin also loses elasticity and tightness.

Genetics. Genetics plays a large part in how your skin ages, and it's hard to fight it with over-the-counter products and treatments.

Poor diet. Lack of quality nutrition can negatively impact both the body and the skin. When the skin is not supported through a healthy diet, skin becomes dull, drab and damaged.

Environment. Environmental factors that affect the skin include pollution, dirt and germs. These things clog the pores, dull your appearance and lead to blemishes, acne and breakouts. Environmental factors also include prolonged exposure to the sun, which can cause pigmentation problems and destroy collagen and elastin.

How Is Stem Cell Therapy Used for the Skin?

One of the ways stem cell therapy is used for the skin is through a stem cell face-lift procedure. During this treatment, Dr. Johnson harvests stem cells from unwanted fat taken from another area of your body, such as your lower back or abdomen.

After the cells are harvested, they are separated from the blood and other tissue and then injected into your face with tiny needles.

The stem cell face-lift can be combined with other procedures, such as the facial fat transfer. Combining the procedures increases the odds of stem cell survival and boosts the anti-aging benefits.

What Are the Benefits of Stem Cell Therapy for the Skin?

Patients who undergo stem cell therapy for anti-aging benefits see changes in their skin such as:

Are you interested in learning more about stem cell therapy and its benefits for your skin? Call Innovations Stem Cell today at 214-256-1462 to learn more.

Read the rest here:
Stem Cells for Skin Quality - innovationsstemcellcenter.com

It can be hard to tell the difference between doctors conducting responsible clinical trials and clinics selling unproven treatments. One common differentiator is the way a treatment is marketed. Most specialized doctors receive patient referrals, while clinics selling stem cell treatments tend to market directly to patients, often through persuasive language on the Internet, Facebook and in newspaper advertisements.

Clinics peddling unproven stem cell treatments frequently overstate the benefits of their offerings and use patient testimonials to support their claims. These testimonials can be intentionally or unintentionally misleading. For example, a person may feel better immediately after receiving a treatment, but the perceived or actual improvement may be due to other factors, such as an intense belief that the treatment will work, auxiliary treatments accompanying the main treatment, healthy lifestyle changes adapted in conjunction with the treatment and natural fluctuations in the disease or condition. These factors are complex and difficult to measure objectively outside the boundaries of carefully designed clinical trials. Learn more about why we need to perform clinical trials here.

Beware of clinics that use persuasive language, including patient testimonials, on the Internet, Facebook and newspapers, to market their treatments, instead of science-based evidence.

Visit link:
Nine Things To Know About Stem Cell Treatments A Closer ...

These are 4-cell stage mouse embryos.

Researchers have found a way to transform skin cells into the three major stem cell types that comprise early-stage embryos. The work (in mouse cells) has significant implications for modeling embryonic disease and placental dysfunctions, as well as paving the way to create whole embryos from skin cells.

Researchers at the Hebrew University of Jerusalem (HU) have found a way to transform skin cells into the three major stem cell types that comprise early-stage embryos. The work (in mouse cells) has significant implications for modelling embryonic disease and placental dysfunctions, as well as paving the way to create whole embryos from skin cells.

As published in Cell Stem Cell, Dr. Yossi Buganim of HUs Department of Developmental Biology and Cancer Research and his team discovered a set of genes capable of transforming murine skin cells into all three of the cell types that comprise the early embryo: the embryo itself, the placenta and the extra-embryonic tissues, such as the umbilical cord. In the future, it may be possible to create entire human embryos out of human skin cells, without the need for sperm or eggs. This discovery also has vast implications for modelling embryonic defects and shedding light on placental dysfunctions, as well as solving certain infertility problems by creating human embryos in a petri dish.

Back in 2006, Japanese researchers discovered the capacity of skin cells to be reprogrammed into early embryonic cells that can generate an entire fetus, by expressing four central embryonic genes. These reprogrammed skin cells, termed Induced Plutipotent Stem Cells (iPSCs), are similar to cells that develop in the early days after fertilization and are essentially identical to their natural counterparts. These cells can develop into all fetal cell types, but not into extra-embryonic tissues, such as the placenta.

Now, the Hebrew University research team, headed by Dr. Yossi Buganim, Dr. Oren Ram from the HUs Institute of Life Science and Professor Tommy Kaplan from HUs School of Computer Science and Engineering, as well as doctoral students Hani Benchetrit and Mohammad Jaber, found a new combination of five genes that, when inserted into skin cells, reprogram the cells into each of three early embryonic cell types iPS cells which create fetuses, placental stem cells, and stem cells that develop into other extra-embryonic tissues, such as the umbilical cord. These transformations take about one month.

The HU team used new technology to scrutinize the molecular forces that govern cell fate decisions for skin cell reprogramming and the natural process of embryonic development. For example, the researchers discovered that the gene Eomes pushes the cell towards placental stem cell identity and placental development, while the Esrrb gene orchestrates fetus stem cells development through the temporary acquisition of an extrae-mbryonic stem cell identity.

To uncover the molecular mechanisms that are activated during the formation of these various cell types, the researchers analyzed changes to the genome structure and function inside the cells when the five genes are introduced into the cell. They discovered that during the first stage, skin cells lose their cellular identity and then slowly acquire a new identity of one of the three early embryonic cell types, and that this process is governed by the levels of two of the five genes.

Recently, attempts have been made to develop an entire mouse embryo without using sperm or egg cells. These attempts used the three early cell types isolated directly from a live, developing embryo. However, HUs study is the first attempt to create all three main cell lineages at once from skin cells. Further, these findings mean there may be no need to sacrifice a live embryo to create a test tube embryo.

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UMSOM Researchers Discovered that Pigment-Producing Stem Cells Can Help Regenerate Vital Part of Nervous System

Neurodegenerative diseases like multiple sclerosis (MS) affect millions of people worldwide and occur when parts of the nervous system lose function over time. Researchers at the University of Maryland School of Medicine (UMSOM) have discovered that a type of skin-related stem cell could be used to help regenerate myelin sheaths, a vital part of the nervous system linked to neurodegenerative disorders.

The discovery into these types of stem cells is significant because they could offer a simpler and less invasive alternative to using embryonic stem cells. This early stage research showed that by using these skin-related stem cells, researchers were able to restore myelin sheath formation in mice.

This research enhances the possibility of identifying human skin stem cells that can be isolated, expanded, and used therapeutically. In the future, we plan to continue our research in this area by determining whether these cells can enhance functional recovery from neuronal injury, saidThomas J. Hornyak, MD, PhD, Associate Professor and Chairman of theDepartment of Dermatology, and Principal Investigator in this research. In the future, we plan to continue our research in this area by determining whether these cells can enhance functional recovery from neuronal injury.

Using a mouse model, Dr. Hornyaks team of researchers discovered a way to identify a specific version of a cell known as a melanocyte stem cell. These are the precursor cells to the cells in skin and hair follicles that make a pigment know as melanin, which determines the color of skin and hair. These melanocyte stem cells have the ability to continue to divide without limit, which is a trait that is not shared by other cells in the body. Additionally, the researchers discovered that these stem cells can make different types of cells depending on the type of signals they receive. This research was published inPLoS Genetics.

Importantly, unlike the embryonic stem cell, which must be harvested from an embryo, melanocyte stem cells can be harvested in a minimally-invasive manner from skin.

Dr. Hornyaks research team found a new way to not only identify the right kind of melanocyte stem cells, but also the potential applications for those suffering from neurodegenerative disorders. By using a protein marker that is only found on these specialized cells, Dr. Hornyaks research group was able to isolate this rare population of stem cells from the majority of the cells that make up skin. Additionally, they found that there exist two different types of melanocyte stem cells, which helped in determining the type of cells they could create.

Using this knowledge, the UMSOM researchers determined that under the right conditions, these melanocyte stem cells could function as cells that produce myelin, the major component of a structure known as the myelin sheath, which protects neurons and is vital to the function of our nervous system. Some neurodegenerative diseases, like multiple sclerosis, are caused by the loss of these myelin-producing, or glial, cells which ultimately lead to irregular function of the neurons and ultimately a failure of our nervous system to function correctly.

Dr. Hornyak and members of his laboratory grew melanocyte stem cells with neurons isolated from mice that could not make myelin. They discovered that these stem cells behaved like a glial cell under these conditions. These cells ultimately formed a myelin sheath around the neurons that resembled structures of a healthy nerve cell. When they took this experiment to a larger scale, in the actual mouse, the researchers found that mice treated with these melanocyte stem cells had myelin sheath structures in the brain as opposed to untreated mice who lacked these structures.

This research holds promise for treating serious neurodegenerative diseases that impact millions of people each year. Our researchers at the University of Maryland School of Medicine have discovered what could be a critical and non-invasive way to use stem cells as a therapy for these diseases,said UMSOM Dean,E. Albert Reece, MD, PhD, MBA, Executive Vice President for Medical Affairs, UM Baltimore, and the John Z. and Akiko K. Bowers Distinguished Professor.

Learn more: UMSOM Researchers Discover Certain Skin-Related Stem Cells Could Help in Treating Neurogenerative Diseases

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Researchers at the Hebrew University of Jerusalem (HU) have found a way to transform skin cells into the three major stem cell types that comprise early-stage embryos. The work (in mouse cells) has significant implications for modelling embryonic disease and placental dysfunctions, as well as paving the way to create whole embryos from skin cells.

As published in Cell Stem Cell, Dr. Yossi Buganim of HUs Department of Developmental Biology and Cancer Research and his team discovered a set of genes capable of transforming murine skin cells into all three of the cell types that comprise the early embryo: the embryo itself, the placenta and the extra-embryonic tissues, such as the umbilical cord. In the future, it may be possible to create entire human embryos out of human skin cells, without the need for sperm or eggs. This discovery also has vast implications for modelling embryonic defects and shedding light on placental dysfunctions, as well as solving certain infertility problems by creating human embryos in a petri dish.

Back in 2006, Japanese researchers discovered the capacity of skin cells to be reprogrammed into early embryonic cells that can generate an entire fetus, by expressing four central embryonic genes. These reprogrammed skin cells, termed Induced Plutipotent Stem Cells (iPSCs), are similar to cells that develop in the early days after fertilization and are essentially identical to their natural counterparts. These cells can develop into all fetal cell types, but not into extra-embryonic tissues, such as the placenta.

Now, the Hebrew University research team, headed by Dr. Yossi Buganim, Dr. Oren Ram from the HUs Institute of Life Science and Professor Tommy Kaplan from HUs School of Computer Science and Engineering, as well as doctoral students Hani Benchetrit and Mohammad Jaber, found a new combination of five genes that, when inserted into skin cells, reprogram the cells into each of three early embryonic cell types iPS cells which create fetuses, placental stem cells, and stem cells that develop into other extra-embryonic tissues, such as the umbilical cord. These transformations take about one month.

The HU team used new technology to scrutinize the molecular forces that govern cell fate decisions for skin cell reprogramming and the natural process of embryonic development. For example, the researchers discovered that the gene Eomes pushes the cell towards placental stem cell identity and placental development, while the Esrrb gene orchestrates fetus stem cells development through the temporary acquisition of an extrae-mbryonic stem cell identity.

To uncover the molecular mechanisms that are activated during the formation of these various cell types, the researchers analyzed changes to the genome structure and function inside the cells when the five genes are introduced into the cell. They discovered that during the first stage, skin cells lose their cellular identity and then slowly acquire a new identity of one of the three early embryonic cell types, and that this process is governed by the levels of two of the five genes.

Recently, attempts have been made to develop an entire mouse embryo without using sperm or egg cells. These attempts used the three early cell types isolated directly from a live, developing embryo. However, HUs study is the first attempt to create all three main cell lineages at once from skin cells. Further, these findings mean there may be no need to sacrifice a live embryo to create a test tube embryo.

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Materials provided by The Hebrew University of Jerusalem. Note: Content may be edited for style and length.

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Embryo stem cells created from skin cells | SciSeek

The complications of the little boys genetic skin disease grew as he did. Tiny blisters had covered his back as a newborn. Then came the chronic skin wounds that extended from his buttocks down to his legs.

By June 2015, at age 7, the boy had lost nearly two-thirds of his skin due to an infection related to the genetic disorder junctional epidermolysis bullosa, which causes the skin to become extremely fragile. Theres no cure for the disease, and it is often fatal for kids. At the burn unit at Childrens Hospital in Bochum, Germany, doctors offered him constant morphine and bandaged much of his body, but nothing not even his fathers offer to donate his skin worked to heal his wounds.

We were absolutely sure we could do nothing for this kid, Dr. Tobias Rothoeft, a pediatrician with Childrens Hospital in Bochum, which is affiliated with Ruhr University. [We thought] that he would die.

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As a last-ditch effort, the boys father asked if there were any available experimental treatments. The German doctors reached out to Dr. Michele De Luca, an Italian stem cell expert who heads the Center for Regenerative Medicine at the University of Modena and Reggio Emilia, to see if a transplant of genetically modified skin cells might be possible. De Luca knew the odds were against them such a transplant had only been performed twice in the past, and never on such a large portion of the body. But he said yes.

The doctors were ultimately able to reconstruct fully functional skin for 80 percent of the boys body by grafting on genetically modified cells taken from the boys healthy skin. The researchers say the results of this single-person clinical trial, published on Wednesday in Nature, show that transgenic stem cells can regenerate an entire tissue. De Luca told reporters the procedure not only offers hope to the 500,000 epidermolysis bullosa patients worldwide but also could offer a blueprint for using genetically modified stem cells to treat a variety of other diseases.

To cultivate replacement skin, the medical team took a biopsy the size of a matchbook from the boys healthy skin and sent it to De Lucas team in Italy. There, researchers cloned the skin cells and genetically modified them to have a healthy version of the gene LAMB3, responsible for making the protein laminin-332. They grew the corrected cultures into sheets, which they sent back to Germany. Then, over a series of three operations between October 2015 and January 2016, the surgical team attached the sheets on different parts of the boys body.

The gene-repaired skin took, and spread. Within just a month the wounds were islands within intact skin. The boy was sent home from the hospital in February 2016, and over the next 21 months, researchers said his skin healed normally. Unlike burn patients whose skin grafts arent created from genetically modified cells the boy wont need ointment for his skin and can regrow his hair.

And unlike simple grafts of skin from one body part to another, we had the opportunity to reproduce as much as those cells as we want, said plastic surgeon Dr. Tobias Hirsch, one of the studys authors. You can have double the whole body surface or even more. Thats a fantastic option for a surgeon to treat this child.

Dr. John Wagner, the director of the University of Minnesota Masonic Childrens Hospitals blood and marrow transplant program, told STAT the findings have extraordinary potential because, until now, the only stem cell transplants proven to work in humans was of hematopoietic stem cells those in blood and bone marrow.

Theyve proven that a stem cell is engraftable, Wagner said. In humans, what we have to demonstrate is that a parent cell is able to reproduce or self-renew, and differentiate into certain cell populations for that particular organ. This is the first indication that theres another stem cell population [beyond hematopoietic stem cells] thats able to do that.

The researchers said the aggressive treatment outlined in the study necessary in the case of the 7-year-old patient could eventually help other patients in less critical condition. One possibility, they noted in the paper, was to bank skin samples from infants with JEB before they develop symptoms. These could then be used to treat skin lesions as they develop rather than after they become life-threatening.

The treatment might be more effective in children, whose stem cells have higher renewal potential and who have less total skin to replace, than in adults, Mariaceleste Aragona and Cdric Blanpain, stem cell researchers with the Free University of Brussels, wrote in an accompanying commentary for Nature.

But De Luca said more research must be conducted to see if the methods could be applied beyond this specific genetic disease. His group is currently running a pair of clinical trials in Austria using genetically modified skin stem cells to treat another 12 patients with two different kinds of epidermolysis bullosa, including JEB.

For the 7-year-old boy, life has become more normal now that it ever was before, the researchers said. Hes off pain meds. While he has some small blisters in areas that didnt receive a transplant, they havent stopped him from going to school, playing soccer, or behaving like a healthy child.

The kid is doing quite well. If he gets bruises like small kids [do], they just heal as normal skin heals, Rothoeft said. Hes quite healthy.

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Researchers at the Hebrew University of Jerusalem say they have found a way to transform skin cells into the three major stem cell types that comprise early-stage embryos.

The discovery could pave the way to creating entire human embryos out of human skin cells, without the need for sperm or eggs, the researchers say. And it could also have vast implications for modeling embryonic defects and shedding light on placental dysfunctions, as well as solving certain infertility problems by creating human embryos in a petri dish, a Hebrew University statement said.

You could say we are close to generating a synthetic embryo, which is a really crazy thing, said Dr. Yossi Buganim of the universitys Department of Developmental Biology and Cancer Research, who led the study that was published in Cell Stem Cell.

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This discovery could allow researchers in future to generate embryos from sterile men and women, using only their skin cells, and generate a real embryo in a dish and implant the embryo in the mother, Buganim said in a phone interview.

Researchers at the Hebrew Hebrew University of Jerusalem say they have found a way to transform skin cells into the three major stem cell types that comprise early-stage embryos; the image shows 4-cell stage mouse embryos (Kirill Makedonski)

Buganim and his team discovered a set of five genes capable of transforming murine skin cells into all three of the cell types that make up the early embryo: the fetus itself, the placenta and the extra-embryonic tissues, such as the umbilical cord.

In 2006, Japanese researchers Kazutoshi Takahashi and Shinya Yamanaka discovered the capacity of skin cells to be reprogrammed into early embryonic cells that can generate an entire fetus through the use of four central embryonic genes. These genes reprogrammed the skin cells into induced pluripotent stem cells (iPSCs), which are similar to cells that develop in the early days after fertilization and are essentially identical to their natural counterparts. These cells can develop into all fetal cell types, but not into extra-embryonic tissues, such as the placenta.

The Japanese researchers discovered that the four central embryonic genes can be used to rejuvenate the skin cells to function like embryonic stem cells, explained Buganim.

After fertilization of the egg, the cell divides into 64, creating a bowl of cells that make up the three crucial parts of an embryo the epiblast, the inner cell mass which gives rise to the fetus itself; the primitive endoderm that is responsible for the umbilical cord; and a third part, the trophectoderm, that is responsible for creating the placenta.

What the Japanese managed to do, Buganim said, was to transform the skin cells into fetus stem cells. But that is not enough to create an entire embryo, he said, because the other parts are also needed those that develop the umbilical cord and the placenta.

Dr. Yossi Buganim of The Hebrew Universitys Department of Developmental Biology and Cancer Research (Shai Herman)

The breakthrough of the Hebrew University team, Buganim said, was creating with five genes all of the three essential compartments that make up the embryonic and extra-embryonic features necessary for the creation of an in-vitro embryo. The work was done with mice, and the team is now starting to apply the same research to human embryos, he added.

The researchers used five genes that are completely different from those used by the Japanese researchers, Buganim noted. The genes the Israeli researchers used are those that play a role in the early development of the embryo. They specify and direct what each cell will develop into, whether the umbilical cord, the placenta or the fetus itself.

The team used new technology to study the molecular forces that dictate how each of the cells develop. For example, the researchers discovered that the gene Eomes pushes the cell toward placental stem cell identity and placental development, while Esrrb orchestrates the development of fetus stem cells, attaining first, but just temporarily, an extra-embryonic stem cell identity.

It was our idea to use those genes, Buganim said.

The researchers then combined these five genes in such a way that, when inserted into skin cells, they managed to reprogram the cells into each of three early embryonic cell types in the same petri dish.

The discovery will enable researchers to better understand and address embryonic malfunctions and diseases such as placental insufficiencies or miscarriages, he said. This could enable researchers to use a dish to model the embryonic cells and identify early markers for risk.

The challenges ahead, however, are still huge, said Buganim. An embryo is a three dimensional structure. We need to learn how to put this all together to generate a real embryo. We need to identify the ratios of placental stem cells, umbilical cord cells and iPS cells, which create the fetuses, and in what scaffold to place them, he said.

These cells know how to stick together, Buganim said. I need to give them the proper environment and the proper ratio to organize themselves into a real embryo.

The study was done by Buganim together with Dr. Oren Ram from Hebrew Universitys Institute of Life Science and Professor Tommy Kaplan from the universitys School of Computer Science and Engineering, as well as doctoral students Hani Benchetrit and Mohammad Jaber.

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Hebrew University researchers create embryo stem cells ...

Photo Credit: Hebrew U

A new, groundbreaking study by the Hebrew University of Jerusalem (HU) found a way to transform skin cells into the three major stem cell types that comprise early-stage embryos. This work has significant implications for modelling embryonic disease and placental dysfunctions, as well as paving the way to create whole embryos from skin cells.

As published in Cell Stem Cell, Dr. Yossi Buganim of HUs Department of Developmental Biology and Cancer Research and his team discovered a set of genes capable of transforming murine skin cells into all three of the cell types that comprise the early embryo: the embryo itself, the placenta and the extraembryonic tissues, such as the umbilical cord. In the future, it may be possible to create entire human embryos out of human skin cells, without the need for sperm or eggs. This discovery also has vast implications for modelling embryonic defects and shedding light on placental dysfunctions, as well as solving certain infertility problems by creating human embryos in a petri dish.

Back in 2006, Japanese researchers discovered the capacity of skin cells to be reprogrammed into early embryonic cells that can generate an entire fetus, by expressing four central embryonic genes. These reprogrammed skin cells, termed Induced Plutipotent Stem Cells (iPSCs), are similar to cells that develop in the early days after fertilization and are essentially identical to their natural counterparts. These cells can develop into all fetal cell types, but not into extra-embryonic tissues, such as the placenta.

Now, the Hebrew University research team, headed by Dr. Yossi Buganim, Dr. Oren Ram from the HUs Institute of Life Science and Professor Tommy Kaplan from HUs School of Computer Science and Engineering, as well as doctoral students Hani Benchetrit and Mohammad Jaber, found a new combination of five genes that, when inserted into skin cells, reprogram the cells into each of three early embryonic cell typesiPS cells which create fetuses, placental stem cells, and stem cells that develop into other extraembryonic tissues, such as the umbilical cord. These transformations take about one month.

The HU team used new technology to scrutinize the molecular forces that govern cell fate decisions for skin cell reprogramming and the natural process of embryonic development. For example, the researchers discovered that the gene Eomes pushes the cell towards placental stem cell identity and placental development, while the Esrrb gene orchestrates fetus stem cells development through the temporary acquisition of an extraembryonic stem cell identity.

To uncover the molecular mechanisms that are activated during the formation of these various cell types, the researchers analyzed changes to the genome structure and function inside the cells when the five genes are introduced into the cell. They discovered that during the first stage, skin cells lose their cellular identity and then slowly acquire a new identity of one of the three early embryonic cell types, and that this process is governed by the levels of two of the five genes.

Recently, attempts have been made to develop an entire mouse embryo without using sperm or egg cells. These attempts used the three early cell types isolated directly from a live, developing embryo. However, HUs study is the first attempt to create all three main cell lineages at once from skin cells. Further, these findings mean there may be no need to sacrifice a live embryo to create a test tube embryo.

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Hebrew U Researchers Created Embryo Stem Cells from Skin ...

Pluripotent stem cells are master cells. Theyre able to make cells from all three basic body layers, so they can potentially produce any cell or tissue the body needs to repair itself. This master property is called pluripotency. Like all stem cells, pluripotent stem cells are also able to self-renew, meaning they can perpetually create more copies of themselves.

There are several types of pluripotent stem cells, including embryonic stem cells. At Childrens Hospital Boston, we use the broader term because pluripotent stem cells can come from different sources, and each method creates a cell with slightly different properties.

But all of them are able to differentiate, or mature, into the three primary groups of cells that form a human being:

Right now, its not clear which type or types of pluripotent stem cells will ultimately be used to create cells for treatment, but all of them are valuable for research purposes, and each type has unique lessons to teach scientists. Scientists are just beginning to understand the subtle differences between the different kinds of pluripotent stem cells, and studying all of them offers the greatest chance of success in using them to help patients.

Types of pluripotent stem cells:

All four types of pluripotent stem cells are being actively studied at Childrens.

Induced pluripotent cells (iPS cells):Scientists have discovered ways to take an ordinary cell, such as a skin cell, and reprogram it by introducing several genes that convert it into a pluripotent cell. These genetically reprogrammed cells are known as induced pluripotent cells, or iPS cells. The Stem Cell Program at Childrens Hospital Boston was one of the first three labs to do this in human cells, an accomplishment cited as the Breakthrough of the Year in 2008 by the journal Science.

iPS cells offer great therapeutic potential. Because they come from a patients own cells, they are genetically matched to that patient, so they can eliminate tissue matching and tissue rejection problems that currently hinder successful cell and tissue transplantation. iPS cells are also a valuable research tool for understanding how different diseases develop.

Because iPS cells are derived from skin or other body cells, some people feel that genetic reprogramming is more ethical than deriving embryonic stem cells from embryos or eggs. However, this process must be carefully controlled and tested for safety before its used to create treatments. In animal studies, some of the genes and the viruses used to introduce them have been observed to cause cancer. More research is also needed to make the process of creating iPS cells more efficient.

iPS cells are of great interest at Childrens, and the lab of George Q. Daley, MD, PhD, Director of Stem Cell Transplantation Program, reported creating 10 disease-specific iPS lines, the start of a growing repository of iPS cell lines.

Embryonic stem cells:Scientists use embryonic stem cell as a general term for pluripotent stem cells that are made using embryos or eggs, rather than for cells genetically reprogrammed from the body. There are several types of embryonic stem cells:

1. True embryonic stem cell (ES cells)These are perhaps the best-known type of pluripotent stem cell, made from unused embryos that are donated by couples who have undergone in vitro fertilization (IVF). The IVF process, in which the egg and sperm are brought together in a lab dish, frequently generates more embryos than a couple needs to achieve a pregnancy.

These unused embryos are sometimes frozen for future use, sometimes made available to other couples undergoing fertility treatment, and sometimes simply discarded, but some couples choose to donate them to science. For details on how theyre turned into stem cells, visit our page How do we get pluripotent stem cells?

Pluripotent stem cells made from embryos are generic and arent genetically matched to a particular patient, so are unlikely to be used to create cells for treatment. Instead, they are used to advance our knowledge of how stem cells behave and differentiate.

2. Stem cells made by somatic cell nuclear transfer (ntES cells)The term somatic cell nuclear transfer (SCNT) means, literally, transferring the nucleus (which contains all of a cells genetic instructions) from a somatic cellany cell of the bodyto another cell, in this case an egg cell. This type of pluripotent stem cell, sometimes called an ntES cell, has only been made successfully in lower animals. To make ntES cells in human patients, an egg donor would be needed, as well as a cell from the patient (typically a skin cell).

The process of transferring a different nucleus into the egg reprograms it to a pluripotent state, reactivating the full set of genes for making all the tissues of the body. The egg is then allowed to develop in the lab for several days, and pluripotent stem cells are derived from it. (Read more in How do we get pluripotent stem cells?)

Like iPS cells, ntES cells match the patient genetically. If created successfully in humans, and if proven safe, ntES cells could completely eliminate tissue matching and tissue rejection problems. For this reason, they are actively being researched at Childrens.

3. Stem cells from unfertilized eggs (parthenogenetic embryonic stem cells)Through chemical treatments, unfertilized eggs can be tricked into developing into embryos without being fertilized by sperm, a process called parthenogenesis. The embryos are allowed to develop in the lab for several days, and then pluripotent stem cells can be derived from them (for more, see How do we get pluripotent stem cells?)

If this technique is proven safe, a woman might be able to donate her own eggs to create pluripotent stem cells matching her genetically that in turn could be used to make cells that wouldnt be rejected by her immune system.

Through careful genetic typing, it might also be possible to use pES cells to create treatments for patients beyond the egg donor herself, by creating master banks of cells matched to different tissue types. In 2006, working with mice, Childrens researchers were the first to demonstrate the potential feasibility of this approach. (For details, see Turning pluripotent stem cells into treatment).

Because pES cells can be made more easily and more efficiently than ntES cells, they could potentially be ready for clinical use sooner. However, more needs to be known about their safety. Concerns have been raised that tissues derived from them might not function normally.

Read more about pluripotent stem cells by following these links:

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Pluripotent Stem Cells 101 Boston Children's Hospital

Stem Cell Therapy has been around for quite some time, but due to high cost it was primarily used for recovery in athletes and the financial elite. However, with the progression of science and knowledge, stem cell therapy has become much more widely used and financially attainable.

Tampa Rejuvenation is the first in Tampa Bay to utilize the benefits of stem cell therapy for the purpose of anti-aging and sexual performance. We realize as our patients age, their bodies no longer have the regenerative properties to attain the desired results from using their growth factors alone as with our PRP, or Plasma Rich Platelet, therapy. Although many patients will still yield improvement with the PRP alone, the magnitude of cytokines and growth factors in your blood as you age will deplete with age. By implementing stem cell therapy, the number of growth factors are exponential allowing our bodies to regenerate on a magnitude that is otherwise unattainable with some results lasting for 3-5 years.

Stem Cell Therapy can be used to restore vitality to the skin, encourage the growth of hair, and even restore sexual performance and pleasure.

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Stem Cell Therapy for Anti-Aging and Sexual Performance ...

Are there enough stem cells in your knees to heal the damage of osteoarthritis? If yes, why arent those stem cells fixing your knees now? Is it a lack of numbers?

Marc Darrow MD, JD. Thank you for reading my article. You can ask me your questions about bone marrow derived stem cells using the contact form below.

In 2011, doctors at the University of Aberdeen published research in the journal Arthritis and rheumatism that provided the first evidence that resident stem cells in the knee joint synovium underwent proliferation (multiplied) and chondrogenic differentiation (made themselves into cartilage cells) following injury.(1)This paper, presenting the idea that stem cells in an injured knee increased in numbers in preparation of healing has been cited by more than 40 medical studies.

If the stem cells in your knee synovial lining are abundant and have the ability to rebuild cartilage after injury, why isnt your knee fixing itself?

One of those 40 studies was performed by researchers at theUniversity of Calgary in 2012. Among their questions, if the stem cells in the knee synovial lining are abundant and have the ability to rebuild cartilage after injury, why isnt the knee fixing itself? Here is what they published:

Since osteoarthritis leads to a progressive loss of cartilage and synovial progenitors (rebuilding) cells have the potential to contribute to articular cartilage repair, the inability of osteoarthritis synovial fluid Mesenchymal progenitor cells (stem cell growth factors) to spontaneously differentiate into chondrocytes suggests that cell-to-cell aggregation and/or communication may be impaired in osteoarthritis and somehow dampen the normal mechanism of chondrocyte replenishment from the synovium or synovial fluid. Should the cells of the synovium or synovial fluid be a reservoir of stem cells for normal articular cartilage maintenance and repair, these endogenous sources of chondro-biased cells would be a fundamental and new strategy for treating osteoarthritis and cartilage injury if this loss of aggregation & differentiation phenotype can be overcome.(2)

This research was supported in a study from December 2017 In Nature reviews. The paper suggested that recognizing that joint-resident stem cells are comparatively abundant in the joint and occupy multiple niches (from the center of the joint to the out edges) will enable the optimization of single-stage therapeutic interventions for osteoarthritis.(3) The idea is to get these native stem cells to repair.

Now we know that there are many stem cells in the knee, when there is an injury there are more stem cells. If we can figure out how to get these stem cells turned on to the healing mode, the knee could heal itself of early stage osteoarthritis. So the problem is not the number of stem cells, BUT, communication.

This failure to communicate was also seen in other research. In 2016, another heavily cited paper, this time fromTehran University for Medical Sciences, noted that despite their larger numbers,the native stem cells act chaotically and are unable to regroup themselves into a healing mechanism and repair the bone, cartilage and other tissue. Introducing bone marrow stem cells into this environmentgets the native stem cells in line and redirects them to perform healing functions. The joint environmentis changed from chaotic to healing because of communication.(4) It should be pointed out that 62 medical studies cited the research in this papers findings).

A recentpaper from a research team inAustralia confirms how this change of joint environment works. It starts with cell signalling a new communication network is built.

University of Iowa research published in theJournal of orthopaedic research

Serious meniscus injuries seldom heal and increase the risk for knee osteoarthritis; thus, there is a need to develop new reparative therapies. In that regard, stimulating tissue regeneration by autologous (from you, not donated) stem/progenitor cells has emerged as a promising new strategy.

(The research team) showed previously that migratory chondrogenic progenitor cells (mobile cartilage growth factors) were recruited to injured cartilage, where they showed a capability in situ (on the spot) tissue repair. Here, we tested the hypothesis that the meniscus contains a similar population of regenerative cells.

Explant studies revealed that migrating cells were mainly confined to the red zone (where the blood is and its growth factors) in normal menisci: However, these cells were capable of repopulating defects made in the white zone (the desert area where no blood flows. Migrating cell numbers increased dramatically in damaged meniscus. Relative to non-migrating meniscus cells, migrating cells were more clonogenic, overexpressed progenitor cell markers, and included a larger side population. (They were ready to heal) Gene expression profiling showed that the migrating population was more similar tochondrogenic progenitor cells (mobile cartilage growth factors) than other meniscus cells. Finally, migrating cells equaledchondrogenic progenitor cells in chondrogenic potential, indicating a capacity for repair of the cartilaginous white zone of the meniscus. These findings demonstrate that, much as in articular cartilage, injuries to the meniscus mobilize an intrinsic progenitor cell population with strong reparative potential.(6)

The intrinsic progenitor cell population with strong repair potential are in your knee waiting to be mobilized.

So what are we to make of this research?There are a lot of stem cells in a knee waiting to repair. The problem is they are confused and not getting the correct instructions. Stem cell therapy can fix the communication problem and begin the repair process anew.

A leading provider of bone marrow derived stem cell therapy, Platelet Rich Plasma and Prolotherapy11645 WILSHIRE BOULEVARD SUITE 120, LOS ANGELES, CA 90025

PHONE: (800) 300-9300

1 Kurth TB, Dellaccio F, Crouch V, Augello A, Sharpe PT, De Bari C. Functional mesenchymal stem cell niches in adult mouse knee joint synovium in vivo. Arthritis Rheum. 2011 May;63(5):1289-300. doi: 10.1002/art.30234.

2 Krawetz RJ, Wu YE, Martin L, Rattner JB, Matyas JR, Hart DA. Synovial Fluid Progenitors Expressing CD90+ from Normal but Not Osteoarthritic Joints Undergo Chondrogenic Differentiation without Micro-Mass Culture. Kerkis I, ed.PLoS ONE. 2012;7(8):e43616. doi:10.1371/journal.pone.0043616.

3 McGonagle D, Baboolal TG, Jones E. Native joint-resident mesenchymal stem cells for cartilage repair in osteoarthritis. Nature Reviews Rheumatology. 2017 Dec;13(12):719.

4Davatchi F, et al. Mesenchymal stem cell therapy for knee osteoarthritis: 5 years follow-up of three patients. Int J Rheum Dis. 2016 Mar;19(3):219-25.

5. Freitag J, Bates D, Boyd R, Shah K, Barnard A, Huguenin L, Tenen A.Mesenchymal stem cell therapy in the treatment of osteoarthritis: reparative pathways, safety and efficacy a review.BMC Musculoskelet Disord. 2016 May 26;17(1):230. doi: 10.1186/s12891-016-1085-9. Review.

6 Seol D, Zhou C, et al. Characteristics of meniscus progenitor cells migrated from injured meniscus. J Orthop Res. 2016 Nov 3. doi: 10.1002/jor.23472.

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Stem cell numbers in a damaged knee - Dr. Marc Darrow is a ...

Where do stem cells come from? Learn the basics of master cells to better understand their therapeutic potential.

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Where do stem cells come from? You have probably heard of thewonders of stem cell therapy. Not only do stem cells make research for future scientific breakthroughs possible, but they also provide the basis for many medical treatments today. So, where exactly are they from, and how are they different from regular cells? The answer depends on the types of stem cells in question.

There are two main types of stem cells adult and embryonic:

Beyond the two broader categories, there are sub-categories. Each has its own characteristics. For researchers, the different types of stem cells serve specific purposes.

Many tissues throughout the adult human body contain stem cells. Scientists previously believed adult stem cells to be inferior to human embryonic stem cells for therapeutic purposes. Theydid not believe adult stem cells to be as versatile as embryonic stem cells (ESCs), because they are not capable of becoming all 200 cell types within the human body.

While this theoryhas notbeen entirely disproved, encouraging evidence suggests that adult stem cells can develop into a variety of new types of cells. They can also affect repair through other mechanisms.

In August 2017, the number of stem cell publications registered in PubMed, a government database, surpassed 300,000. Stem cells are also being explored in over 4,600 cell therapy clinical trials worldwide. Some of the earliest forms of adult stem cell use include bone marrow and umbilical cord blood transplantation.

It should be noted that while the term adult stem cell is used for this type of cell, it is not descriptive of age, because adult stem cells can come from children. The term simply helps to differentiate stem cells derived from living humans as opposed to embryonic stem cells.

Embryonic stem cells are controversial because they are made from embryos that are created but not used by fertility clinics.

Because adult stem cells are somewhat limited in the cell types they can become, scientists developed a way to genetically reprogram cells into what is called an inducedpluripotent stem cell or iPS cell. In creating inducedpluripotent stem cells, researchers hope to blend the usefulness of adult stem cells with the promise of embryonic stem cells.

Both embryonic stem cells (ESCs) and induced pluripotent stem cells (iPSCs) are known as pluripotent stem cells.

Pluripotent stem cells are a type of cell that has the capacity to divide indefinitely and create any cell found within the three germ layers of an organism: ectoderm (cells forming the skin and nervous system), endoderm (cells forming pancreas, liver, endocrine gland, and gastrointestinal and respiratory tracts), and mesoderm (cells forming connective tissues, and other related tissues, muscles, bones, most of the circulatory system, and cartilage).

Embryonic stem cells can grow into a much wider range of cell types, but they also carry the risk of immune system rejection in patients. In contrast, adult stem cells are more plentiful, easier to harvest, and less controversial.

Embryonic stem cells come from embryos harvested shortly after fertilization (within 4-5 days). These cells are made when the blastocysts inner cell mass is transferred into a culture medium, allowing them to develop.

At 5-6 days post-fertilization, the cells within the embryo start to specialize. At this time, they no longer are able to become all of the cell types within the human body. They are no longer pluripotent.

Because they are pluripotent, embryonic stem cells can be used to generate healthy cells for disease patients. For example, they can be grown into heart cells known as cardiomyocytes. These cells may have the potential to be injected into an ailing patients heart.

Harvesting stem cells from embryos is controversial, so there are guidelines created by the National Institutes of Health (NIH) that allow the public to understand what practices are not allowed.

Scientists can harvest perinatal stem cells from a variety of tissues, but the most common sources include:

The umbilical cord attaches a mother to her fetus. It is removed after birth and is a valuable source of stem cells. The blood it contains is rich in hematopoietic stem cells (HSC). It also contains smaller quantities of another cell type known as mesenchymal stem cells (MSCs).

The placenta is a large organ that acts as a connector between the mother and the fetus. Both placental blood and tissue are also rich in stem cells.

Finally, there is amniotic fluid surrounding a baby while it is in utero. It can be harvested if a pregnant woman needs a specialized kind of test known as amniocentesis. Both amniotic fluid and tissue contain stem cells, too.

Adult stem cells are usually harvested in one of three ways:

The blood draw, known as peripheral blood stem cell donation, extracts the stem cells directly from a donors bloodstream. The bone marrow stem cells come from deep within a bone often a flat bone such as the hip. Tissue fat is extracted from a fatty area, such as the waist.

Embryonic donations are harvested from fertilized human eggs that are less than five days old. The embryos are not grown within a mothers or surrogates womb, but instead, are multiplied in a laboratory. The embryos selected for harvesting stem cell are created within invitro fertilization clinics but are not selected for implantation.

Amniotic stem cells can be harvested at the same time that doctors use a needle to withdraw amniotic fluid during a pregnant womans amniocentesis. The same fluid, after being tested to ensure the babys health, can also be used to extract stem cells.

As mentioned, there is another source for stem cells the umbilical cord. Blood cells from the umbilical cord can be harvested after a babys birth. Cells can also be extracted from the postpartumhuman placenta, which is typically discarded as medical waste following childbirth.

The umbilical cord and the placenta are non-invasive sources of perinatal stem cells.

People who donate stem cells through the peripheral blood stem cell donor procedure report it to be a relativelypainless procedure. Similar to giving blood, the procedure takes about four hours. At a clinic or hospital, an able medical practitioner draws the blood from the donors vein in one of his arms using a needle injection. The technician sends the drawn blood into a machine, which extracts the stem cells. The blood is then returned to the donors body via a needle injected into the other arm. Some patients experience cramping or dizziness, but overall, its considered a painless procedure.

If a blood stem cell donor has a problem with his or her veins, a catheter may be injected in the neck or chest. The donor receives local anesthesia when a catheter-involved donation occurs.

During a bone marrow stem cell donor procedure, the donor is put under heavy sedation in an operating room. The hip is often the site chosen to harvest the bone marrow. More of the desired red marrow is found in flat bones, such as those in the pelvic region. The procedure takes up to two hours, with several extractions made while the patient is sedated. Although the procedure is painless due to sedation, recovery can take a couple of weeks.

Bone marrow stem cell donation takes a toll on the donorbecause it involves the extraction of up to 10 percent of the donors marrow. During the recovery period, the donors body gradually replenishes the marrow. Until that happens, the donor may feel fatigued and sore.

Some clinics offer regenerative and cosmetic therapies using the patients own stem cells derived from the fat tissue located on the sides of the waistline. Considered a simple procedure, clinics do this for therapeutic reasons or as a donation for research.

Stem cells differ from the trillions of other cells in your body. In fact, stem cells make up only a small fraction of the total cells in your body. Some people have a higher percentage of stem cells than others. But, stem cells are special because they are the mothers from which specialized cells grew and developed within us. When these cells divide, they become daughters. Some daughter cells simply self-replicate, while others form new kinds of cells altogether. This is the main way stem cells differ from other body cells they are the only ones capable of generating new cells.

The ways in which stem cells can directly treat patients grow each year. Regenerative medicine now relies heavily on stem cell applications. This type of treatment replaces diseased cells with new, healthy ones generated through donor stem cells. The donor can be another person or the patient themselves.

Sometimes, stem cells also exert therapeutic effects by traveling through the bloodstream to sites that need repair or by impacting their micro-environment through signaling mechanisms.

Some types of adult stem cells, like mesenchymal stem cells (MSCs), are well-known for exerting anti-inflammatory and anti-scarring effects. MSCs can also positively impact the immune system.

Conditions and diseases which stem cell regeneration therapy may help include Alzheimers disease, Parkinsons disease, and multiple sclerosis (MS). Heart disease, certain types of cancer, and stroke victims may also benefit in the future. Stem cell transplant promises advances in treatment for diabetes, spinal cord injury, severe burns, and osteoarthritis.

Researchers also utilize stem cells to test new drugs. In this case, an unhealthy tissue replicates into a larger sample. This method enables researchers to test various therapies on a diseased sample, rather than on an ailing patient.

Stem cell research also allows scientists to study how both healthy and diseased tissue grows and mutates under various conditions. They do this by harvesting stem cells from the heart, bones, and other body areas and studying them under intensive laboratory conditions. In this way, they get a better understanding of the human body, whether healthy or sick.

With the following stem cell transplant benefits, its not surprising people would like to try the therapy as another treatment option.

Physicians harvest stem cell from either the patient or a donor. For an autologous transplant, there is no risk of transferring any disease from another person. For an allogeneic transplant, the donor is meticulously screened before the therapy to make sure they are compatible with the patient and have healthy sources of stem cells.

One common and serious problem of transplants is the risk of rejecting the transplanted organs, tissues, stem cells, and others. With autologous stem cell therapy, the risk is avoided primarily because it comes from the same person.

Because stem cell transplants are typically done through infusion or injection, the complex and complicated surgical procedure is avoided. Theres no risk of accidental cuts and scarring post-surgery.

Recovery time from surgeries and other types of treatments is usually time-consuming. With stem cell therapy, it could only take about 3 months or less to get the patient back to their normal state.

As the number of stem cell treatments dramatically grew over the years, its survival rate also increased. A study published in the Journal of Clinical Oncology showed there was a significant increase in survival rate over 12 years among participants of the study. The study analyzed results from over 38,000 stem cell transplants on patients with blood cancers and other health conditions.

One hundred days following transplant, the researchers observed an improvement in the survival rate of patients with myeloid leukemia. The significant improvements we saw across all patient and disease populations should offer patients hope and, among physicians, reinforce the role of blood stem cell transplants as a curative option for life-threatening blood cancers and other diseases.

With the information above, people now have a better understanding of the answer to the question Where do stem cells come from? Stem cells are a broad topic to comprehend, and its better to go back to its basics to learn its mechanisms. This way, a person can have a piece of detailed knowledge about these master cells from a scientific perspective.

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Where Do Stem Cells Come From? | Basics Of Stem Cell Therapy

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Where Do Stem Cells Come From? | Basics Of Stem Cell ...

By Insoo Hyun

Stem cells are undifferentiated cells that have the capacity to renew themselves and to specialize into various cell types, such as blood, muscle, and nerve cells. Embryonic stem cells, derived from five-day-old embryos, eventually give rise to all the different cells and organ systems of the embryo. Embryonic stem cells are pluripotent, because they are capable of differentiating along each of the three germ layers of cells in the embryo, as well as producing the germ line (sperm and eggs). The three germ layers are the ectoderm (skin, nerves, brain), the mesoderm (bone, muscle), and the endoderm (lungs, digestive system).

During later stages of human development, minute quantities of more mature stem cells can be found in most tissue and organ systems, such as bone marrow, the skin, and the gut. These are somatic stem cells, responsible for renewing and repairing the bodys specialized cells. Although the lay public often refers to them as adult stem cells, researchers prefer to call them multipotent because they are less versatile than pluripotent stem cells, and because they are present from the fetal stage of development and beyond. Multipotent stem cells can only differentiate into cells related to the tissue or organ systems from which they originated for instance, multipotent blood stem cells in bonemarrow can develop into different types of blood cells, but not into nerve cells or heart cells.

While multipotent stem cell research has been around for nearly 50 years and has led to clinical therapies for leukemia and other blood disorders, the field of human embryonic stem cell research is still relatively new, and basic discoveries have yet to be directly transitioned into clinical treatments. Human embryonic stem cells were first isolated and maintained in culture in 1998 by James Thomson and colleagues at the University of Wisconsin. Since then, more than a thousand different isolateslines of self-renewing embryonic stem cellshave been created and shared by researchers worldwide.

The main ethical and policy issues with stem cells concern the derivation and use of embryonic stem cells for research. A vocal minority of Americans objects to the destruction of embryos that occurs when stem cells are derived. Embryonic stem cell research is especially controversial for those who believe that five-day-old preimplantation human embryos should not be destroyed no matter how valuable the research may be for society.

To bypass this ethical controversy, the Presidents Council on Bioethics recommended in 2005 that alternative sources of pluripotent stem cells be pursued. Some alternatives have been developed, most notably, the induced pluripotent stem (iPS) cells human skin cells and other body cells reprogrammed to behave like embryonic cells. But embryonic stem cell research will remain needed because there are some questions only they have the potential to answer.

Embryonic stem cells are necessary for several aims of scientific and biomedical research. They include addressing fundamental questions in developmental biology, such as how primitive cells differentiate into more specialized cells and how different organ systems first come into being. By increasing our knowledge of human development, embryonic stem cells may also help us better understand the causes of fetal deformations.

Other important applications lie in the areas of disease research and targeted drug development. By deriving and studying embryonic or other pluripotent stem cells that are genetically-matched to diseases such as Parkinsons disease and juvenile diabetes, researchers are able to map out the developmental course of complex medical conditions to understand how, when, and why diseased specialized cells fail to function properly in patients. Such disease-in-a-dish model systems provide researchers with a powerful new way to study genetic diseases. Furthermore, researchers can aggressively test the safety and efficacy of new, targeted drug interventions on tissue cultures of living human cells derived from disease-specific embryonic stem cells. This method of testing can reduce the risks associated with human subjects research.

One possible way of deriving disease-specific stem cells is through a technique called somatic cell nuclear transfer (SCNT), otherwise known as research cloning. By replacing the DNA of an unfertilized egg with the DNA of a cell from a patients body, researchers are able to produce embryonic stem cells that are genetically-matched to the patient and his or her particular disease. SCNT, however, is technically challenging and requires the collection of high-quality human eggs from female research volunteers, who must be asked to undergo physically burdensome procedures to extract eggs.

A much more widespread and simpler technique for creating disease-specific stem cells was pioneered in 2006 by Shinya Yamanaka and colleagues in Kyoto, Japan. They took mouse skin cells and used retroviruses to insert four genes into them to to create iPS cells. In 2007, teams led by Yamanaka, James Thomson, and George Daley each used similar techniques to create human iPS cells. The iPS cell approach is promising because disease-specific stem cells could be created using skin or blood samples from patients and because, unlike SCNT, it does not require the procurement of human eggs for research.

However, despite these advances, scientists do not believe iPS cells can replace human embryonic stem cells in research. For one, embryonic stem cells must be used as controls to assess the behavior and full scientific potential of iPS cells. Furthermore, iPS cells may not be able to answer some important questions about early human development. And safety is a major issue for iPS cell research aimed at clinical applications, since the cell reprogramming process can cause harmful mutations in the stem cells, increasing the risk of cancer. In light of these and other concerns, iPS cells may perhaps prove to be most useful in their potential to expand our overall understanding of stem cell biology, the net effect of which will provide the best hope of discovering new therapies for patients.

Many who oppose embryonic stem cell research believe for religious or other personal reasons that all preimplantation embryos have a moral standing equal to living persons. On the other hand, those who support embryonic stem cell research point out that not all religious traditions grant full moral standing to early-stage human embryos.

According to Jewish, Islamic, Hindu, and Buddhist traditions, as well as many Western Christian views, moral standing arrives much later during the gestation process, with some views maintaining that the fetus must first reach a stage of viability where it would be capable of living outside the womb. Living in a pluralistic society such as ours, supporters argue, means having to tolerate differences in religious and personal convictions over such theoretical matters as when, during development, moral standing first appears.

Other critics of embryonic stem cell research believe that all preimplantation embryos have the potential to become full-fledged human beings and that they should never have this potential destroyed. In response, stem cell supporters argue that it is simply false that all early-stage embryos have the potential for complete human life many fertility clinic embryos are of poor quality and therefore not capable of producing a pregnancy (although they may yield stem cells). Similarly, as many as 75% to 80% of all embryos created through intercourse fail to implant. Furthermore, no embryos have the potential for full human life until they are implanted in a womans uterus, and until this essential step is taken an embryos potential exists only in the most abstract and hypothetical sense.

Despite the controversies, embryonic stem cell research continues to proceed rapidly around the world, with strong public funding in many countries. In the U.S., federal money for embryonic stem cell research is available only for stem cell lines that are on the National Institutes of Health stem cell registry. However, no federal funds may be used to derive human embryonic stem cell lines; NIH funds may only be used to study embryonic stem cells that were derived using other funding sources.

Despite the lack of full federal commitment to funding embryonic stem cell research in the U.S., there are wide-ranging national regulatory standards. The National Academy of Sciences established guidelines in 2005 for the conduct of human embryonic stem cell research. (See Resources.) According to these guidelines, all privately and publicly funded scientists working with embryonic stem cells should have their research proposals approved by local embryonic stem cell research oversight (ESCRO) committees. ESCRO committees are to include basic scientists, physicians, ethicists, legal experts, and community members to look at stem-cell-specific issues relating to the proposed research. These committees are also to work with local ethics review boards to ensure that the donors of embryos and other human materials are treated fairly and have given their voluntary informed consent to stem cell research teams. Although these guidelines are voluntarily, universities and other research centers have widely accepted them.

At the global level, in 2016 the International Society for Stem Cell Research (ISSCR) released a comprehensive set of professional guidelines for human stem cell research, spanning both bench and clinical stem cell research. (See Resources.) Unlike the NAS guidelines, the ISSCR guidelines go beyond American standards, adding, for example, the recommendation that stem cell lines be banked and freely distributed to researchers around the world to facilitate the fields progress on just and reasonable terms.The potential for over-commercialization and restrictive patenting practices is a major problem facing the stem cell field today, which may delay or reduce the broad public benefit of stem cell research. The promise of broad public benefit is one of thejustifying conditions for conducting stem cell research; without the real and substantial possibility for public benefit, stem cell research loses one of its most important moral foundations.

However, providing useful stem-cell-based therapies in the future is not a simple proposition, either. Developing a roadmap to bring stem cell research into the clinic will involve many complex steps, which the new ISSCR guidelines help address. They include:

These and other difficult issues must be sorted out if stem cell research in all its forms is to fulfill its promise.

STEM CELL GLOSSARY

Newer ethical issues in stem cell research go far beyond the embryo debate, since they encompass all stem cell types, not just human embryonic stem cells, and because they involve human subjects who, despite what one may think about the moral status of preimplantation embryos, are unequivocally moral persons. No other emerging issue better encapsulates the above concern than the growing phenomenon of stem cell tourism. At present, stem cell-based therapies are the clinical standard of care for only afew conditions, such as hematopoietic stem cell transplants for leukemia and epithelial stem cell-based treatments for burns and corneal disorders. Unfortunately, some unscrupulous clinicians around the world are exploiting patients hopes by purporting to provide for large sums of money effective stem cell therapies for many other conditions. These so-called stem cell clinics advance claims about their proffered stem cell therapies without credible scientific rationale, transparency, oversight, or patient protections.

The administration of unproven stem cell interventions outside of carefully regulated research protocols endangers patients and jeopardizes the legitimate progress of translational stem cell scientific research. Patients who travel for unproven stem cell therapies put themselves at risk of physical and financial harm.

The ISSCR guidelines are a good point for thinking about this important problem. The guidelines allow for exceptional circumstances in which clinicians might attempt medically innovative care in a very small number of seriously ill patients, subject to stringent oversight criteria. These criteria include: independent peer review of the proposed innovative procedure and its scientific rationale; institutional accountability; rigorous informed consent and close patient monitoring; transparency; timely adverse event reporting; and a commitment by clinician-scientists to move to a formal clinical trial in a timely manner after experience with at most a few patients. By juxtaposing some current stem cell clinics against the standards outlined in the ISSCR guidelines, one may easily identify some clinics shortcomings and call into question the legitimacy of their purported claims of providing innovative care to patients.

Moving beyond past debates about embryo status to issues concerning the uses of all varieties of stem cells, one can begin to focus the bioethical discourse on areas that have a much broader consensus base of shared values, such as patient and research subject protections and justice. Justice may also call on regulatory and oversight bodies to include a greater involvement of community and patient advocates in the oversight of research. Dealing with the bioethics of stem cell research demands that we wrestle with these and other tough questions.

Insoo Hyun, PhD, is an associate professor of bioethics at Case Western Reserve University.

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Stem Cells - The Hastings Center

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