Cell Therapy Technique to Treat Low Back Pain Funded with $2 … – Genetic Engineering & Biotechnology News

Researchers at Cedars-Sinai have received a $2 million grant from theCalifornia Institute for Regenerative Medicine (CIRM) to develop a new cell therapy that helpsimprove quality of life for patients with degenerated discs and chronic lower back pain.

Dmitriy Sheyn, PhD, assistant professor in the departments of orthopedics, surgery, and biomedical sciences, leads this new project in collaboration with Debiao Li, PhD, director of the Biomedical Imaging Research Institute and professor of biomedical sciences and imaging, and Hyun Bae, MD, professor of orthopedics and co-medical director of Spine Education at Cedars-Sinai.

We are extremely grateful for CIRMs support, said Li, who also holds the Karl Storz chair in minimally invasive surgery in honor of Dr. George Berci. We are committed to finding a better way to treat the millions of people who suffer from this painful condition and medical imaging can play an important role.

The team of investigators, which includes biomaterials experts, imaging experts, pain management experts, and spine surgeons, hopes the research will lead to the development of a novel injectable therapeutic for back pain and intervertebral disc degeneration, which is the most common cause of lower back pain.

There is an urgent need for a long-lasting stem cell therapy that targets the underlying pathogenesis of intervertebral disc degeneration, explained Sheyn. The goal is to have an off-the-shelf treatment accessible to different population groups suffering from this type of pain.

Lower back pain is one of the most common conditions that eventually lead to surgical interventions, chronic pain, and use of opioids in the United States. At least 80% of the adult population is suffering from lower back pain, and 40% of these cases originate in intervertebral disc degeneration.

Once degeneration cascade starts, it is very difficult to slow it down or reverse it, noted Bae. This award will help propel us into the next phase of research, where we hope to develop a therapy that can one day be widely available, cost-effective, and accessible to all.

To date, most treatments for this condition are limited to invasive surgical interventions, such as disc replacement and spinal fusion, or pain management that does not address the underlying cause of intervertebral disc degeneration.

Researchers have tried to find ways to fix this problem, such as injecting stem cells, but there have been many challenges with this approach.

To help combat this problem, the team will be testing a new method using iPSC-derived notochordal cells that are delivered in a special microgel. The team will combine the treatment and MRI technology to develop and optimize the stem cell-loaded microgel component and to test it in large animals.

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Cell Therapy Technique to Treat Low Back Pain Funded with $2 ... - Genetic Engineering & Biotechnology News

Global Stem Cell Therapy Market Is Projected To Grow At A 17% Rate Through The Forecast Period – EIN News

Stem Cell Therapy Global Market Report 2023 Market Size, Trends, And Global Forecast 2023-2027

The Business Research Companys Stem Cell Therapy Global Market Report 2023 Market Size, Trends, And Forecast 2023-2027

The growth in the stem cell therapy market is due to the rising prevalence of chronic diseases. North America region is expected to hold the largest stem cell therapy market share. Major players in the stem cell therapy market include Anterogen, JCR Pharmaceuticals, Medipost, Osiris Therapeutics, Pharmicell, Astellas Pharma, Cellectis, Celyad.

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Trending Stem Cell Therapy Market Trend The companies in the stem cell therapy market are increasingly investing in strategic partnerships. A strategic partnership is a mutually beneficial agreement between two companies that do not compete directly with each other.

Stem Cell Therapy Market Segments By Type: Allogeneic Stem Cell Therapy, Autologous Stem Cell Therapy By Cell Source: Adult Stem Cells, Induced Pluripotent Stem Cells, Embryonic Stem Cells By Application: Musculoskeletal Disorders, Wounds and Injuries, Cancer, Autoimmune Disorders, Other Applications By End-User: Hospitals, Clinics By Geography: The global stem cell therapy market is segmented into North America, South America, Asia-Pacific, Eastern Europe, Western Europe, Middle East and Africa.

Read more on the global stem cell therapy market report at: https://www.thebusinessresearchcompany.com/report/stem-cells-therapy-global-market-report

Stem cell therapy refers to a form of regenerative medicine that uses stem cells or their byproducts to stimulate the body's natural repair process in damaged, malfunctioning, or wounded tissue. It is the next step in the transplantation of organs, replacing donor organswhich are scarcewith cells.

Stem Cell Therapy Global Market Report 2023 from TBRC covers the following information: Market size date for the forecast period: Historical and Future Market analysis by region: Asia-Pacific, China, Western Europe, Eastern Europe, North America, USA, South America, Middle East and Africa. Market analysis by countries: Australia, Brazil, China, France, Germany, India, Indonesia, Japan, Russia, South Korea, UK, USA.

Trends, opportunities, strategies and so much more.

The Stem Cell Therapy Global Market Report 2023 by The Business Research Company is the most comprehensive report that provides insights on stem cell therapy global market size, drivers and stem cell therapy trends, stem cell therapy global market major players, stem cell therapy share and competitors' revenues, market positioning, and stem cell therapy global market growth across geographies. The stem cell therapy global market report helps you gain in-depth insights on opportunities and strategies. Companies can leverage the data in the report and tap into segments with the highest growth potential.

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Regenerative Medicine Market is Expected to Reach $40.6 Billion | MarketsandMarkets. – Yahoo Finance

MarketsandMarkets Research Pvt. Ltd.

Chicago, Feb. 24, 2023 (GLOBE NEWSWIRE) -- Regenerative medicine is an emerging field of medicine that is focused on using the bodys natural healing processes to repair and regenerate damaged tissue. This technology has the potential to revolutionize medicine and offer a range of treatments for a variety of diseases and conditions. In the near future, regenerative medicine has the potential to transform the way we treat many illnesses and injuries, ranging from heart disease and diabetes to spinal cord injuries and hearing loss. Regenerative medicine is already being used to treat certain conditions, such as diabetes, blindness, and spinal cord injuries. The technology is also being used to regenerate tissue and organs, such as skin, bone, and even heart tissue. In the future, regenerative medicine could be used to treat a variety of diseases and conditions, including Alzheimers and Parkinsons.

Regenerative Medicine market in terms of revenue was estimated to be worth $12.2 Billion in 2022 and is poised to reach $40.6 Billion by 2027, growing at a CAGR of 27.2% from 2022 to 2027 according to a latest report published by MarketsandMarkets. Market growth is driven by the rising prevalence of chronic diseases, genetic disorders, and cancer; rising investments in regenerative medicine research; and the growing pipeline of regenerative medicine products. However, the high cost of cell and gene therapies and ethical concerns related to the use of embryonic stem cells in research and development are expected to restrain the growth of this market during the forecast period.

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Regenerative Medicine Market Scope:

Report Coverage

Details

Market Revenue in 2022

$12.2 Billion

Estimated Value by 2027

$40.6 Billion

Growth Rate

Poised to grow at a CAGR of 27.2%

Market Size Available for

20202027

Forecast Period

20222027

Forecast Units

Value (USD Billion)

Report Coverage

Revenue Forecast, Competitive Landscape, Growth Factors, and Trends

Segments Covered

Product, Application, and Region

Geographies Covered

North America, Europe, Asia Pacific, Latin America, Middle East & Africa

Report Highlights

Updated financial information / product portfolio of players

Key Market Restraints

Shortage of skilled professionals

Key Market Drivers

Availability of funding and rising investments in R&D

Notable Regenerative Medicine mergers and acquisitions for 2021 2022 include:

Johnson & Johnson and Lineage Cell Therapeutics: Johnson & Johnson completed the acquisition of Lineage Cell Therapeutics in early 2021, which will help the company expand its presence in the regenerative medicine field.

Baxter International and Xygen Medical: In February 2021, Baxter International announced its acquisition of Xygen Medical, a leading developer of regenerative medicine technologies.

Vertex Pharmaceuticals and Mast Therapeutics: In March 2021, Vertex Pharmaceuticals announced its acquisition of Mast Therapeutics, a biopharmaceutical company focused on regenerative medicine.

Takeda Pharmaceuticals and TiGenix: In April 2021, Takeda Pharmaceuticals announced its acquisition of TiGenix, a regenerative medicine company focused on stem cell therapies.

Novartis and Endocyte: In May 2021, Novartis announced its acquisition of Endocyte, a biopharmaceutical company focused on developing regenerative medicine treatments for cancer.

Astellas Pharma and CellSight: In June 2021, Astellas Pharma announced its acquisition of CellSight, a regenerative medicine company focused on developing cell therapies.

Cellectis and Tmunity: In July 2021, Cellectis announced its acquisition of Tmunity, a regenerative medicine company focused on developing cell therapies for cancer.

Roche and Cell Design Labs: In August 2021, Roche announced its acquisition of Cell Design Labs, a regenerative medicine company focused on developing cell therapies for cancer.

Medtronic and CartiHeal: In September 2021, Medtronic announced its acquisition of CartiHeal, a regenerative medicine company focused on developing cell therapies for orthopedic applications.

Novo Nordisk and TiGenix: In October 2021, Novo Nordisk announced its acquisition of TiGenix, a regenerative medicine company focused on developing stem cell therapies.

Story continues

Growth Drivers of Regenerative Medicine Market from macro to micro:

Increasing Prevalence of Chronic Diseases: Chronic diseases, such as heart diseases, cancer, diabetes, and neurological disorders, are on the rise globally. This is driving the adoption of regenerative medicines as a potential treatment for such diseases.

Government Initiatives: Governments around the world are increasingly investing in regenerative medicine research and development to support the development of innovative therapies.

Growing Investment: As the potential of regenerative medicine becomes more apparent, the number of venture capital and private equity investments in the field is increasing.

Technological Advancements: Recent advances in stem cell research and 3D printing technology have enabled the development of novel regenerative medicine therapies.

Increasing Adoption of Advanced Therapy Medicinal Products (ATMPs): ATMPs are becoming more widely accepted and adopted, which is fueling the growth of the regenerative medicine market.

Growing Focus on Precision Medicine: Precision medicine is gaining traction in healthcare, as it allows for more personalized treatments. This is driving the development of regenerative medicine therapies.

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Hypothetic challenges of Regenerative Medicine market in near future:

Regulation: Regulation of regenerative medicine products varies from country to country. In some countries, there are no regulations in place for the approval or access to regenerative medicine products. This could become a major challenge for companies operating in the regenerative medicine market by limiting their ability to bring their products to market.

Cost: Cost of regenerative medicine products is currently high due to the high cost of production and research involved. This could limit the accessibility of the products and result in a slower growth of the market.

Accessibility: Accessibility is a major challenge for companies operating in the regenerative medicine market. There is limited access to the products for patients in certain parts of the world due to the lack of availability of the products. This could result in slower growth of the market.

Clinical Trials: Clinical trials are required in order to assess the safety and efficacy of regenerative medicine products. This could prove to be a challenge due to the long time frames required for the trials and the associated costs.

Reimbursement: Reimbursement for regenerative medicine products is currently limited in many countries. This could limit the access to the products for patients, as well as the growth of the market.

Top 3 use cases of Regenerative Medicine market:

Cell Therapy: Cell therapy involves the use of adult stem cells to repair or replace damaged or diseased cells, tissues, and organs. This can be used to treat a wide range of medical conditions, including diabetes, Parkinsons disease, stroke, heart disease, and more.

Gene Therapy: Gene therapy is a form of regenerative medicine that involves using genetic material to modify existing cells or create new ones to replace damaged or dysfunctional ones. This can be used to treat genetic disorders, cancer, and other conditions.

Tissue Engineering: Tissue engineering is a process of regenerating or replacing damaged tissues and organs using cells, scaffolds, and bioactive molecules. This can be used to treat musculoskeletal injuries, skin injuries, and other conditions.

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Regenerative Medicine Market is Expected to Reach $40.6 Billion | MarketsandMarkets. - Yahoo Finance

Wells Fargo Lights / Fralin Researchers Emphasize Importance of … – The Roanoke Star

Rare diseases Affect 1 in 10 Americans

On Feb. 28, the top of Wells Fargo Tower in downtown Roanoke will be illuminated with a show of pink, green, purple, and blue. Its part of an effort to shine a light on important but uncommon diseases in recognition ofRare Disease Day, which takes place annually on the last day of February.

We all have diagnoses, and we all have disabilities and abilities, saidStephanie DeLuca, an associate professor at the Fralin Biomedical Research Institute at VTC and co-director of itsNeuromotor Research Clinic. DeLuca and co-directorSharon Rameypioneered the use of a high-intensity therapy that has allowed children with cerebral palsy and other movement disorders to make rapid gains.

They have alsoapplied some of their researchto children diagnosed with rare diseases, including CASK disorders. According to the National Institutes of Health, there were just 130 documented cases of the disorder as of 2020. Its about being willing to try, and not just assuming that because you have a rare diagnosis that there are not positive changes that can be made, DeLuca said. Sometimes its that first step that can lead to a lot of learning that can impact many people. Its one of the things rare diseases can teach us.

More than 7,000 rare diseases affect 30 million people in the United States, according to the National Institutes of Health. Nearly one in 10 Americans is facing a rare disease.

For most rare genetic conditions, the problem is related to changes in a single gene.Rare diseases share some of the same genetic pathways as more common illnesses, however, so by studying them researchers can develop a better understanding the mechanisms of disease that apply to more common health conditions.

Thebenefits ofrare disease researchstretchfar beyond a few affectedindividuals and theirfamilies, said Michael Friedlander, Virginia Techs vice president for health sciences and technology and executive director of the Fralin Biomedical Research Institute.Our institutetakes a broad approach, coordinating effortswith scientists worldwide.

Anthony-Samuel LaMantia, a world-renowned geneticist, professor, and director of the research institutesCenter for Neurobiology Research, investigates DiGeorge syndrome, a disorder that occurs when a small part of chromosome 22 is missing. It affects one in 4,000 people.

Researchers in the Fralin Biomedical Research InstitutesCenter for Vascular and Heart Researchstudy rare diseases that affect electrical signaling in the heart. Fewer than 200,000 Americans are living with Brugada syndrome, a rare disease that can cause sudden cardiac death. Patients with Brugada syndrome usually have mutations in the SCN5A gene, which encodes proteins that regulate sodium channel function in the heart.

Researchers led bySteven Poelzing, a professor at the Fralin Biomedical Research Institute and co-director of the Virginia Tech Translational Biology, Medicine, and Health Graduate Program,study Brugada syndrome to understandhow faulty sodium channels influence cardiac function and heart rhythms.

Nearly one in eight adult cancer patients in the U.S. have a rare form of cancer. They can be challenging to identify, often resulting in delayed diagnosis after symptom onset. Even after diagnosis, treatment options and clinical trials are more limited.

Virginia Tech researchers are targeting glioblastoma, an aggressive form brain cancer with an average survival time of 15 months after diagnosis.

Zhi Shengand his lab are exploring new therapies for glioblastoma multiforme. Sheng is an assistant professor at the Fralin Biomedical Research Institute and aVirginia Tech Cancer Research Alliancemember.Samy Lamouille, an assistant professor at the Fralin Biomedical Research Institute, was given aSeale Innovation Fundgrant to test a novel therapeutic approach to eradicate glioblastoma cancer stem cells. And Associate Professor Jennifer Munson has developed a novel3D tissue-engineered modelof the glioblastoma microenvironment to help learn why the tumors return and how to best eradicate them.

Fewer than 1 percent of children diagnosed with diffuse midline pontine glioma, an aggressive and rare form of pediatric brain cancer, are still alive within five years of diagnosis. Fralin Biomedial Research Institute Assistant ProfessorJia-Ray Yu, who last yearlaunched a new laboratoryon the Childrens National Research and Innovation Campus in Washington, D.C., is investigating the biology of two enzymes that show promise as targets for combination therapies to treat pediatric brain cancer.

Researchers continue to make progress, but fewer than 500 rare diseases have Food and Drug Administration-approved treatments. Because the number of people affected by any one diagnosis is small, there is little economic incentive to invest the millions of dollars needed for research and clinical trials required to develop effective therapies.

The National Institutes of Health also reports that those with rare conditions experience medical costs three to five times higher than for more common illnesses.

A rare disease, as defined in the Orphan Drug Act, affects fewer than 200,000 people, Friedlander said. But the fundamental scientific discoveries that emerge when we work to understand their cellular and molecular processes provides immense value.

Friedlander also serves on theVirginia Department of Healths Rare Disease Council, which advises the General Assembly and the Office of the Governor on the needs of individuals with rare diseases. He works with people with rare disease, care providers, researchers, family members, and program leaders to improve prevention, treatment and support services.

Its vital that we continue to investigate the mechanisms and treatments of rare diseases to advance our understanding of human health, and help patients with rare diseases while informing new therapies for more common disorders, Friedlander said.

Leigh Anne Kelley

Link:
Wells Fargo Lights / Fralin Researchers Emphasize Importance of ... - The Roanoke Star

Adult Stem Cells for Regenerative Therapy – PubMed

Cell therapy has been identified as an effective method to regenerate damaged tissue. Adult stem cells, also known as somatic stem cells or resident stem cells, are a rare population of undifferentiated cells, located within a differentiated organ, in a specialized structure, called a niche, which maintains the microenvironments that regulate the growth and development of adult stem cells. The adult stem cells are self-renewing, clonogenic, and multipotent in nature, and their main role is to maintain the tissue homeostasis. They can be activated to proliferate and differentiate into the required type of cells, upon the loss of cells or injury to the tissue. Adult stem cells have been identified in many tissues including blood, intestine, skin, muscle, brain, and heart. Extensive preclinical and clinical studies have demonstrated the structural and functional regeneration capabilities of these adult stem cells, such as bone marrow-derived mononuclear cells, hematopoietic stem cells, mesenchymal stromal/stem cells, resident adult stem cells, induced pluripotent stem cells, and umbilical cord stem cells. In this review, we focus on the human therapies, utilizing adult stem cells for their regenerative capabilities in the treatment of cardiac, brain, pancreatic, and eye disorders.

Keywords: Blood disorders; Cardiospheres; Diabetes mellitus; Myoblasts; Neurogenesis; Regenerative therapy; Stem cells; Stroke.

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Adult Stem Cells for Regenerative Therapy - PubMed

Stem cell – Adult stem cells | Britannica

Some tissues in the adult body, such as the epidermis of the skin, the lining of the small intestine, and bone marrow, undergo continuous cellular turnover. They contain stem cells, which persist indefinitely, and a much larger number of transit amplifying cells, which arise from the stem cells and divide a finite number of times until they become differentiated. The stem cells exist in niches formed by other cells, which secrete substances that keep the stem cells alive and active. Some types of tissue, such as liver tissue, show minimal cell division or undergo cell division only when injured. In such tissues there is probably no special stem-cell population, and any cell can participate in tissue regeneration when required.

The epidermis of the skin contains layers of cells called keratinocytes. Only the basal layer, next to the dermis, contains cells that divide. A number of these cells are stem cells, but the majority are transit amplifying cells. The keratinocytes slowly move outward through the epidermis as they mature, and they eventually die and are sloughed off at the surface of the skin. The epithelium of the small intestine forms projections called villi, which are interspersed with small pits called crypts. The dividing cells are located in the crypts, with the stem cells lying near the base of each crypt. Cells are continuously produced in the crypts, migrate onto the villi, and are eventually shed into the lumen of the intestine. As they migrate, they differentiate into the cell types characteristic of the intestinal epithelium.

Bone marrow contains cells called hematopoietic stem cells, which generate all the cell types of the blood and the immune system. Hematopoietic stem cells are also found in small numbers in peripheral blood and in larger numbers in umbilical cord blood. In bone marrow, hematopoietic stem cells are anchored to osteoblasts of the trabecular bone and to blood vessels. They generate progeny that can become lymphocytes, granulocytes, red blood cells, and certain other cell types, depending on the balance of growth factors in their immediate environment.

Work with experimental animals has shown that transplants of hematopoietic stem cells can occasionally colonize other tissues, with the transplanted cells becoming neurons, muscle cells, or epithelia. The degree to which transplanted hematopoietic stem cells are able to colonize other tissues is exceedingly small. Despite this, the use of hematopoietic stem cell transplants is being explored for conditions such as heart disease or autoimmune disorders. It is an especially attractive option for those opposed to the use of embryonic stem cells.

Bone marrow transplants (also known as bone marrow grafts) represent a type of stem cell therapy that is in common use. They are used to allow cancer patients to survive otherwise lethal doses of radiation therapy or chemotherapy that destroy the stem cells in bone marrow. For this procedure, the patients own marrow is harvested before the cancer treatment and is then reinfused into the body after treatment. The hematopoietic stem cells of the transplant colonize the damaged marrow and eventually repopulate the blood and the immune system with functional cells. Bone marrow transplants are also often carried out between individuals (allograft). In this case the grafted marrow has some beneficial antitumour effect. Risks associated with bone marrow allografts include rejection of the graft by the patients immune system and reaction of immune cells of the graft against the patients tissues (graft-versus-host disease).

Bone marrow is a source for mesenchymal stem cells (sometimes called marrow stromal cells, or MSCs), which are precursors to non-hematopoietic stem cells that have the potential to differentiate into several different types of cells, including cells that form bone, muscle, and connective tissue. In cell cultures, bone-marrow-derived mesenchymal stem cells demonstrate pluripotency when exposed to substances that influence cell differentiation. Harnessing these pluripotent properties has become highly valuable in the generation of transplantable tissues and organs. In 2008 scientists used mesenchymal stem cells to bioengineer a section of trachea that was transplanted into a woman whose upper airway had been severely damaged by tuberculosis. The stem cells were derived from the womans bone marrow, cultured in a laboratory, and used for tissue engineering. In the engineering process, a donor trachea was stripped of its interior and exterior cell linings, leaving behind a trachea scaffold of connective tissue. The stem cells derived from the recipient were then used to recolonize the interior of the scaffold, and normal epithelial cells, also isolated from the recipient, were used to recolonize the exterior of the trachea. The use of the recipients own cells to populate the trachea scaffold prevented immune rejection and eliminated the need for immunosuppression therapy. The transplant, which was successful, was the first of its kind.

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Stem cell - Adult stem cells | Britannica

About Adult Stem Cell Therapy – University of Kansas Medical Center

Adult Stem Cell Therapy 101

The initial concept of regenerative medicine dates all the way back to 330 BC, when Aristotle observed that a lizard could grow back the lost tip of its tail.

Slowly over time, humans have grown to understand regenerative medicine, and how it may change the way we treat diseases. It's been only relatively recently that adult (non-embryonic) stem cell therapy, a type of regenerative medicine, has gathered fast momentum.

Adult (non-embryonic) stem cells are unspecialized or undifferentiated cells, which means they have yet to develop into a specific cell type. Found in most adult tissues, adult stem cells have two primary properties:

Simply put, adult stem cells have the potential to grow into any of the body's more than 200 cell types.

Adult stem cells have been found in most parts of the body, including brain, bone marrow, blood vessels, skin, teeth and heart. There are typically a small number of stem cells in each tissue. Due to their small number and rate of division (growth), it is difficult to grow adult stem cells in large numbers.

Scientists at the Midwest Stem Cell Therapy Center are working to understand how to grow large amounts of adult stem cells in cell culture. These scientists are also working with more "primitive" stem cells, isolated from the umbilical cord after normal births.

Stem cell transplants, also referred to as bone marrow transplants, have been done since the late 1960s and are well-established treatments for blood cancers and bone marrow failure conditions. Umbilical cord blood also has stem cells that can be used for transplantation for these diseases.

Stem cell transplants for other diseases that use bone marrow, umbilical cord cells or other sources of stem cells are still experimental and need to viewed as such.

The practice of stem cell therapy is not new: One of the oldest forms of it is the bone marrow transplant, which has been actively practiced since the late 1960s. Since then, scientists haven't slowed down with the advancement of adult stem cell therapy.

Every day, scientists worldwide are researching new ways we can harness stem cells to develop effective new treatments for a host of diseases. In the case of a patient suffering with a blood cancer such as leukemia, a bone marrow transplant will replace their unhealthy blood cells with healthy ones.

This same concept inserting healthy cells so they may multiply and form new tissue or repair diseased tissue can be applied to other forms of stem cell therapy.

Stem cell research continues to advance as scientists learn how an organism develops from a single cell and how healthy cells replace damaged cells.

For example, the Midwest Stem Cell Therapy Center is collaborating to investigate the potential of a select group of umbilical cord stem cells in the treatment of Amyotrophic Lateral Sclerosis (ALS, or Lou Gerhig's disease).

Developing a stem cell treatment that has been shown to be both safe and efficacious is not as simple as removing stem cells from one part of the body and putting it in another.

Working with appropriate regulatory agencies, the Midwest Stem Cell therapy Center is conducting R&D activities that will permit the Center to conduct human clinical trials on a variety of diseases over the next several years.

Similar to the development of a new drug, this process when completed, will assure patients in both clinical trials and eventually patients using the approved product, that the product is safe for use in humans and the stem cells being administered are effective in treating the injury or disease they are being used for.

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About Adult Stem Cell Therapy - University of Kansas Medical Center

Scientists Discover Protein Partners that Could Heal Heart Muscle | Newsroom – UNC Health and UNC School of Medicine

A protein that helps make neurons also works to reprogram scar tissue cells into heart muscle cells, especially in partnership with a second protein, according to a study led by Li Qian, PhD, at the UNC School of Medicine.

CHAPEL HILL, N.C. Scientists at the UNC School of Medicine have made a significant advance in the promising field of cellular reprogramming and organ regeneration, and the discovery could play a major role in future medicines to heal damaged hearts.

In a study published in the journal Cell Stem Cell, scientists at the University of North Carolina at Chapel Hill discovered a more streamlined and efficient method for reprogramming scar tissue cells (fibroblasts) to become healthy heart muscle cells (cardiomyocytes). Fibroblasts produce the fibrous, stiff tissue that contributes to heart failure after a heart attack or because of heart disease. Turning fibroblasts into cardiomyocytes is being investigated as a potential future strategy for treating or even someday curing this common and deadly condition.

Surprisingly, the key to the new cardiomyocyte-making technique turned out to be a gene activity-controlling protein called Ascl1, which is known to be a crucial protein involved in turning fibroblasts into neurons. Researchers had thought Ascl1 was neuron-specific.

Its an outside-the-box finding, and we expect it to be useful in developing future cardiac therapies and potentially other kinds of therapeutic cellular reprogramming, said study senior author Li Qian, PhD, associate professor in the UNC Department of Pathology and Lab Medicine and associate director of the McAllister Heart Institute at UNC School of Medicine.

Scientists over the last 15 years have developed various techniques to reprogram adult cells to become stem cells, then to induce those stem cells to become adult cells of some other type. More recently, scientists have been finding ways to do this reprogramming more directly straight from one mature cell type to another. The hope has been that when these methods are made maximally safe, effective, and efficient, doctors will be able to use a simple injection into patients to reprogram harm-causing cells into beneficial ones.

Reprogramming fibroblasts has long been one of the important goals in the field, Qian said. Fibroblast over-activity underlies many major diseases and conditions including heart failure, chronic obstructive pulmonary disease, liver disease, kidney disease, and the scar-like brain damage that occurs after strokes.

In the new study, Qians team, including co-first-authors Haofei Wang, PhD, a postdoctoral researcher, and MD/PhD student Benjamin Keepers, used three existing techniques to reprogram mouse fibroblasts into cardiomyocytes, liver cells, and neurons. Their aim was to catalogue and compare the changes in cells gene activity patterns and gene-activity regulation factors during these three distinct reprogrammings.

Unexpectedly, the researchers found that the reprogramming of fibroblasts into neurons activated a set of cardiomyocyte genes. Soon they determined that this activation was due to Ascl1, one of the master-programmer transcription factor proteins that had been used to make the neurons.

Since Ascl1 activated cardiomyocyte genes, the researchers added it to the three-transcription-factor cocktail they had been using for making cardiomyocytes, to see what would happen. They were astonished to find that it dramatically increased the efficiency of reprogramming the proportion of successfully reprogrammed cells by more than ten times. In fact, they found that they could now dispense with two of the three factors from their original cocktail, retaining only Ascl1 and another transcription factor called Mef2c.

In further experiments they found evidence that Ascl1 on its own activates both neuron and cardiomyocyte genes, but it shifts away from the pro-neuron role when accompanied by Mef2c. In synergy with Mef2c, Ascl1 switches on a broad set of cardiomyocyte genes.

Ascl1 and Mef2c work together to exert pro-cardiomyocyte effects that neither factor alone exerts, making for a potent reprogramming cocktail, Qian said.

The results show that the major transcription factors used in direct cellular reprogramming arent necessarily exclusive to one targeted cell type.

Perhaps more importantly, they represent another step on the path towards future cell-reprogramming therapies for major disorders. Qian says that she and her team hope to make a two-in-one synthetic protein that contains the effective bits of both Ascl1 and Mef2c, and could be injected into failing hearts to mend them.

Cross-lineage Potential of Ascl1 Uncovered by Comparing Diverse Reprogramming Regulatomes was co-authored by Haofei Wang, Benjamin Keepers, Yunzhe Qian, Yifang Xie, Marazzano Colon, Jiandong Liu, and Li Qian. Funding was provided by the American Heart Association and the National Institutes of Health (T32HL069768, F30HL154659, R35HL155656, R01HL139976, R01HL139880).

Media contact: Mark Derewicz, 919-923-0959

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Scientists Discover Protein Partners that Could Heal Heart Muscle | Newsroom - UNC Health and UNC School of Medicine

Organoids: science fiction or the future of pre-clinical studies? – Lexology

New technologies based on human cells are increasingly seen as key to reducing the time and cost in bringing drugs to market. This is an evolving area of study, though the field itself, which developed out of study into 3D cell architecture, is not new. These approaches replicate human physiology to study diseases, treatments, and for drug-development purposes. In parallel to major advances made from a medical and scientific standpoint, novel legal and ethical questions arise.

The definition of organoid is problematic and covers a range of cell culture techniques. Scientists have developed ways of culturing organ-specific tissue from human stem cells or progenitor cells to re-create important aspects of the 3D anatomy e.g. the pancreas, kidney, liver, thyroid, retina, and brain, to recapitulate key organ features. A list of definitions can be found at the end of this article.

A potential game-changer

Breakthroughs in stem cell technology and tissue engineering are driving the change. In addition to this, trends in other areas, like cell and gene therapy and personalised medicine have acted as catalysts. By simulating organ function, specific disease states can be modelled and studied. This is especially useful when looking at rare diseases, how tissue and microbiota interact, or how drugs interact with each other.

The use of organoids holds great promise in several medical-scientific areas like disease modelling, precision medicine, and transplantation. One of the most notable possibilities is to supplement or to a certain extent replace animal models during pre-clinical studies in drug development.

The field is at an early stage, but the trend is an upward one. Companies involved are seeing increasing demand for their services from pharma, biotech, research and academic institutes as well as the cosmetics industry. According to a recent market report the annual growth rate is set to increase by 37.4% during 2022-2028. Cambridge-based CN Bio recently doubled the size of its laboratory facilities due to demand. In the US, Hesperos Inc., a contract research organisation (CRO) provides a multi-organ chip platform (Human-on-a-Chip) based in Florida filed for a USD 20 million IPO. Another fascinating company is Labskin, who make full thickness human skin models, providing reproduceable results for microbiome research. Labskin just announced the first ever commercially available pigmented skin-equivalent in a joint project with Bradford University. These new models incorporate melanocytes, the cells that give skin its colour and present a huge opportunity to study melanomas.

The need for new and/or updated regulatory frameworks

For the use of these new models to be considered by regulatory authorities across the globe in the evaluation of safety and efficacy of drugs (subject to valid scientific demonstrations), regulatory frameworks will have to be adapted on a jurisdiction-by-jurisdiction basis.

In the US, the FDA Modernization Act of 2021 has been introduced to amend the Federal Food, Drug, and Cosmetic Act. The amendment strikes animal and inserts nonclinical tests or studies to be used in the evaluation of safety and efficacy of drugs, such as MPS, cell-based assays and computer models. In addition, the FDA recently approved the first clinical trial using efficacy data collected from a microphysiological system. More data may be needed to convince regulators, but the impetus - and interest - is there.

In the EU, is a 3-year Science With and For Society (SwafS) project, funded under HORIZON2020. The project is being coordinated by the University of Oslo, Norway and involves major research institutions across the EU. It is currently being carried out with the objective of developing a comprehensive regulatory framework for organoid research and organoid-related technologies. In the meantime, the European Parliament is working on legislation aimed at reducing the number of animal studies. The European Medicines Agency already recognized that organoids and organ-on-chip may become suitable alternatives to animal models during medicines development, in its wider effort to promote the 3R principles (replace, reduce and refine).

Further legal and ethical challenges to be addressed

The use of organoids and other MPS raises critical legal and ethical issues, which must be urgently addressed to allow their wide-spread utilization as part of pre-clinical studies within an acceptable framework.

In particular, since organoids are grown from human cells, initial donors rights must be respected and efficiently enforced. The original cells may come from foetal or adult tissues they may be pluripotent stem cells (PSCs), adult stem cells taken from specific tissues and reengineered somatic cells. Key challenges notably relate to the donors informed consent, (which issue has already been addressed by many scholars), research on human embryos and data protection issues. Complete anonymity of human tissue has been shown to be neither possible (due to the identifiable nature of DNA) nor desirable (as the data is necessary to validate prediction models). The donor must be able to control, to some extent, the subsequent use of his/her samples; in practice, consent may not be easily withdrawn (or with very limited effect) once organoids have been successfully developed and used in a pre-clinical study.

Strong quality standards should further be developed and complied with when it comes to producing organoids for pre-clinical purposes. Indeed, a set of specifications for the production of human 3D organoids used as medicines has been proposed. One could imagine specific guidelines applicable to the production of organoids for drug development purposes, to ensure consistent production methods as well as reliability as pre-clinical models. This calls for a wider systemic approach as to the regulation of human tissue and cells intended for human versus research application. On this point, it is interesting to note that a proposal for a regulation on standards of quality and safety for substances of human origin intended for human application is currently being examined in the EU, which expressly excludes their use in research that does not involve application to the human body.

Moreover, specific issues are triggered depending on the type of laboratory-cultivated model. For instance, the development of human cerebral organoids raises questions in terms of moral status and legal protection. Indeed, studies suggest that developed neuronal models show complex electrical activity the human cerebral organoids (sometimes referred to as mini-brains) can command a muscle connected thereto, be receptive to stimuli and may even exhibit a rudimentary form of consciousness. This raises questions as to the core definition of human being and, from a legal standpoint, personhood, the beginning and end of life, as well as the legal protection that should be awarded to such in vitro models (how they can be engineered, used, destroyed etc). In addition, the existence of sophisticated sentient models creates uncertainty regarding what moral status should be awarded to them. These issues are particularly complex as legal, ethical, philosophical, societal and political aspects are necessarily intertwined, and the way they are addressed may greatly vary from one jurisdiction to another.

Further legal and ethical challenges should be addressed in connection with the production and use of organoids beyond pre-clinical studies such as their potential patentability, their commercialization (while certain countries like France forbids the commercialization of human products and elements), their use for transplantation, the articulation with regulations on genome editing, chimeras and human cloning etc.

Conclusion

The rapid progress of scientific research around organoids and other MPS bears the potential to revolutionize many aspects of medical and pharmaceutical research. In particular, they hold great promise in the pursuit of a suitable (and potentially more reliable) alternative to the use of animal models in pre-clinical studies. Beyond this, legal and ethical challenges should be addressed in connection with the production and use of organoids in other applications such as their potential patentability, their commercialization (while certain countries like France forbids the commercialization of human products and elements), their use for transplantation, and the articulation with the regulations on genome editing, the creation of chimeras and human cloning.

Definitions:

Microphysiological systems (MPS): an umbrella term for organ-on-chip (OOC), organoids or tumoroids (stem cells grown in a dish).

Organ-on-a-chip (OOC): from the field of microfluidics, a multi-channel 3-D cell culture. An integrated circuit that simulates the activities, mechanics and physiological response of an entire organ or an organ system.

Organoid: means resembling an organ. Organoids are defined by three characteristics. The cells arrange themselves in vitro into three-dimensional organization that is characteristic for the organ in vivo, the resulting structure consists of multiple cells found in that particular organ and the cells execute at least some of the functions that they normally carry out in that organ.

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Organoids: science fiction or the future of pre-clinical studies? - Lexology

Turning Back the Clock on ALS Cells Reveals New Mechanism – Technology Networks

At the current time, there is no cure for amyotrophic lateral sclerosis (ALS). Things may be about to change, however. Researchers at FAU and the University of California San Diego (UCSD) have identified a protein that already displays pathological characteristics at an early stage of the neurological disease. The team has published their discovery, that could lead to a new approach for treating the disease, in the journal Acta Neuropathologica.

In summer 2014, amyotrophic lateral sclerosis, or ALS for short, received a lot of attention through a social media campaign. In the ice bucket challenge, millions of people across the globe emptied a bucket of ice cold water over their heads to simulate a feeling of paralysis due to the extreme cold. In Germany, approximately 6,000 to 8,000 people are living with ALS, and approximately 2,000 new cases of the disease, that proves fatal within just a few years, are diagnosed every year. ALS is a motor neuron disease, that means it damages the nerve cells that control our muscles, explains Prof. Dr. Beate Winner. During the first phase, muscles become weaker, before wasting away and finally leaving patients unable to swallow or breathe independently. The social media campaign was used to raise money for research into ALS.

Beate Winner is a professor for stem cell models for rare neural diseases at FAU, head of the Department of Stem Cell Biology, and speaker for the Center for Rate Diseases at Universittsklinikum Erlangen. Her laboratory investigates what triggers neurodegenerative diseases of the nervous system such as ALS in the hope of discovering new treatment options as a result. We have known for roughly 15 years that during the end stage of ALS, the protein TDP-43 found in neurons becomes insoluble and starts to form clumps, explains Winner. It loses its normal functions and adopts toxic properties. Even though these pathological changes are not yet noticeable in patients, the fate of the nerve cells is already sealed. Winner continues, We wanted to know whether we could find causes for ALS at an early stage of development before the TDP-43 changes.

She started her quest together with Prof. Dr. Jrgen Winkler and PD Dr. Martin Regensburger from the Department of Molecular Neurology at Universittsklinikum Erlangen. The researchers used an innovative technique. They extracted a small skin sample from the upper arm of ALS patients and healthy people in a control group and reprogrammed it into what are known as induced pluripotent stem cells, cells that are equivalent to a very early stage of human development and that can in theory develop into any cell within the human body. These stem cells were then transformed into nerve cells. Basically, we turned the clock back and generated neurons imitating the developmental stage of a fetus, explains Winner. The fact that cells from adult people can be reprogrammed back into pluripotent stem cells was discovered by Shinya Yamanaka, who received the Nobel Prize for Medicine in recognition of his work.

The Erlangen researchers searched for insoluble proteins in the cell samples using mass spectrometry, a high-throughput procedure. They were successful. In the nerve cells of ALS patients they discovered an RNA-binding protein named NOVA1. In the neurons, the protein demonstrated changes including a greatly increased degree of insolvency, but not yet the typical pathological characteristics of TDP-43, explains Dr. Florian Krach, member of the FAU team and lead author of the study. The cells in the control group did not display these changes.

Armed with these findings, Krach moved to the laboratory of the renowned RNA biologist and bioinformatics specialist Prof. Gene Yeo at the University of California in San Diego (USA), funded by the Bavaria California Technology Center (BaCaTeC). Thanks to specialized experiments and computer-assisted analysis he was able to investigate what NOVA1 binds to in RNA molecules and what influence it has on alternative splicing in human neurons. Alternative splicing is an extremely complex and ingenious mechanism that humans use to multiply their repertoire of proteins, explains Krach. Sections of an RNA messenger molecule are either cut or added, thereby hindering, extending or changing the function of proteins altogether.

It has been known for some time that the alternative splicing process is unregulated in ALS patients. It is also known that TDP-43 influences this process. The team of researchers from Erlangen suspected, however, that other RNA-binding proteins are responsible for the pathological processes in early stages of the disease before TDP-43 changes. This suspicion has now been confirmed with the discovery of the impaired functioning of NOVA1.

We have made a pioneering discovery, but it is only one first step towards possibly being able to detect ALS in the early stages, says Beate Winner. Follow-up studies with larger cohorts could deepen our understanding of the importance of RNA-binding proteins. The researchers hope that their work will help contribute to developing new therapy concepts before neurons cross the point of no return.

Reference:Krach F, Wheeler EC, Regensburger M, et al. Aberrant NOVA1 function disrupts alternative splicing in early stages of amyotrophic lateral sclerosis. Acta Neuropathol. 2022. doi:10.1007/s00401-022-02450-3

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Turning Back the Clock on ALS Cells Reveals New Mechanism - Technology Networks