Category Archives: Cell Therapy

The U.S. Food and Drug Administration (FDA) had several approvals this week. Read on to see what the regulatory agency gave the go-ahead to.

GlaxoSmithKline

GSK announced on Thursday that the U.S. Food and Drug Administration (FDA) has approved belimumab (Benlysta), the first-ever treatment for adults with lupus nephritis (LN) who are currently receiving standard therapy.

LN, or inflammation of the kidneys, is a common system of systemic lupus erythematosus (SLE), one of the most prevalent autoimmune diseases. Lupus causes the production of proteins called autoantibodies in the immune system, which attack tissues and organs, including the kidneys. Five out of 10 adults living with lupus will have LN, while eight out of 10 children with lupus are found to have kidney damage, usually caused by LN.

The Alliance for Lupus Research (LRA) President and Chief Executive Officer Kenneth M. Farber extended congratulations to GSK on behalf of the alliance:

"An effective treatment developed specifically for lupus nephritis has been desperately needed. We are particularly proud to see this approval for another indication of belimumab, having funded much of the original research that led to the drug's development. We at the LRA are thrilled to share this news about belimumab and look forward to future approvals of other lupus nephritis treatment options.

Approval was given following positive results of a 104-week BLISS-LN phase III post-approval study which showed belimumab to be a safe and effective therapy for treating LN.

"Approximately 40% of patients with systemic lupus erythematosus develop lupus nephritis, which causes inflammation in the kidneys and can lead to end-stage kidney disease. BENLYSTA is the first medicine approved to treat systemic lupus and adults with active lupus nephritis, an important treatment advance for patients with this incurable autoimmune disease, said GSK Chief Scientific Officer and President of R&D Dr.Hal Barron.

First approved in 2011, belimumab is now the first and only biologic approved for both SLE and LN in over a half-century.

MacroGenics Inc.

MacroGenics, a biopharma company working to make cancer history with the development of innovative monoclonal antibody-based therapeutics, had its first product, MARGENZA, approved by the FDA on Wednesday.

MARGENZA (margetuximab-cmkb), was given the green light, in combination with chemotherapy, the treatment of adult patients with metastatic HER2-positive breast cancer.

An Fc-engineered monoclonal antibody targeting the HER2 oncoprotein, MARGENZA offers hope for patients who have received two or more prior anti-HER2 regimens, with at least one being for metastatic disease.

In a pivotal head-to-head Phase III SOPHIA clinical trial, it became the first HER2-targeted therapy to improve progression-free survival (PFS) versus Herceptin (trastuzumab), when each was combined with chemotherapy. In comparison with trastuzumab plus chemotherapy, MARGENZA showed a statistically significant 24% reduction in the risk of disease progression or death, with an objective response rate of 22% compared with 16% for trastuzumab.

The approval of MARGENZA is an exciting milestone for MacroGenics and, more importantly, it brings a new treatment option to metastatic breast cancer patients. We are grateful for the patients who participated in this study, as well as their families, and everyone who played a role in helping MacroGenics reach this milestone, said MacroGeneics president and CEO Scott Koenig, M.D., Ph.D., at the time of the announcement.

The product launch is expected to take place in March of 2021.

Athenex, Inc.

There will be a brand-new treatment for actinic keratosis on the market next year, with Tuesdays FDA approval of Athenixs Klisyri (tirbanibulin).

This will be the first branded proprietary product for Athenex, which will be launched in the U.S. in partnership with Barcelona-based biopharma company, Almirall. Klisyri will be manufactured by Athenex.

Actinic Keratosis, a rough, scaly skin patch occurring after years of sun exposure, has the potential to turn into squamous cell skin cancer.

The approval is based on two pivotal, randomized, double-blind, vehicle-controlled Phase III studies (KX01-AK-003 and KX01-AK-004) in 702 adults with actinic keratosis of the face or scalp where tirbanibulin demonstrated complete clearance of lesions at day 57 in treated areas in a significantly higher number of patients compared to vehicle.

The FDA approval of Klisyri is a significant milestone for Athenex. Klisyri is a home-grown product discovered and characterized by Athenex scientists and developed from pre-IND to NDA by the Athenex team. We are extremely proud of our teams excellent execution. Approval demonstrates our ability to execute upon the entirety of the drug development and registration process. We are excited to partner with Almirall to bring this first-in-class microtubule inhibitor to patients with actinic keratosis in the U.S., said Athenex Chairman and Chief Executive Officer, Dr. Johnson Lau.

Buffalo, New York-based Athenex is dedicated to the discovery, development and commercialization of novel therapies for the treatment of cancer.

Genentech Inc. (Roche)

Genentech (a member of the Roche group) began the week with a bang, as the FDA on Monday approved a shorter, two-hour infusion time for relapsing and primary progressive multiple sclerosis therapy Ocrevus (ocrelizumab).

Ocrelizumab is a humanized monoclonal antibody designed to target CD20-positive B cells, an immune cell thought to be a key cause of the myelin and axonal damage found in multiple sclerosis. It is the first and only therapy approved for both relapsing MS (RMS), relapsing-remitting MS (RRMS), secondary-progressive MS (SPMS), and primary-progressive MS (PPMS).

More than 170,000 people with MS have been treated with Ocrevus the only approved B-cell therapy with a twice-yearly dosing schedule and it is the most prescribed MS medicine in the U.S., We constantly strive to improve the experience that patients and their physicians have with our medicines, and we believe people with relapsing and primary progressive MS will find the shorter two-hour Ocrevus infusion time to be more convenient, said Genentech Chief Medical Officer and Head of Global Product Development, Levi Garraway, M.D., Ph.D.

Approval was given on the strength of a randomized, double-blind ENSEMBLE PLUS study, which met the primary endpoint, showing similar frequency and severity of IRs for a two-hour Ocrevus infusion time vs. the already approved 3.5-hour time in patients with RRMS. Importantly, a consistent safety profile was maintained.

The new two-hour infusion time was previously approved by the European Medicines Agency (EMA) in May 2020.

When Pigs Fly Allergy-friendly

The week began with big news from the field of genomics, as the FDA on Monday approved a first-of-its-kind intentional genomic alteration (IGA) in pigs that could reduce potential allergic reactions when porcine materials are used for medical purposes in humans.

The alteration was pulled off in a line of domestic pigs called GalSafe pigs, which may be used for either food or human therapeutics.

"Today's first-ever approval of an animal biotechnology product for both food and as a potential source for biomedical use represents a tremendous milestone for scientific innovation," FDA Commissioner Stephen M. Hahn, M.D. said in a statement.

The IGA is meant to eliminate alpha-gal sugar on the surface of the pigs cells, thereby making the resulting food or therapeutics safe for use in people with Alpha-gal syndrome (AGS) which can cause a mild or severe allergic reaction to alpha-gal sugar found in red meat.

The FDA reviewed data that demonstrated that there is no detectable level of alpha-gal sugar across multiple generations of GalSafe pigs.

This particular pig, however, has not been evaluated for use in xenotransplantation products or for transplantation or implantation into humans.

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FDA Approvals: Therapies for Lupus, MS, Cancer and a Genomic Alteration in Pigs - BioSpace

This is the tenth manuscript published in SITCs Cancer Immunotherapy Guidelines series, a critically important effort to educate physicians and other health care providers, and improve outcomes for cancer patients receiving FDA-approved immunotherapies," said SITC President Mario Sznol, MD.

MILWAUKEE (PRWEB) December 18, 2020

The Society for Immunotherapy of Cancer (SITC), the worlds leading member-driven organization dedicated solely to cancer immunotherapy, is pleased to announce the publication of a clinical practice guideline for the management of cell therapy adverse events. Immune effector cell therapies include the three U.S. Food and Drug Administration (FDA)-approved chimeric antigen receptor (CAR) T cell products for treatment of blood-based cancers.

The Society for Immunotherapy of Cancer (SITC) clinical practice guideline on immune effector cell-related adverse events, was published in the Journal for ImmunoTherapy of Cancer (JITC) and is part of the societys long running Cancer Immunotherapy Guidelines program. The guideline was developed by an expert panel of physicians representing multiple medical specialties, patient advocates, and nurses. It covers risk-assessment, identification and management for the most clinically significant side effects associated with immune effector cell therapies.

Immune effector cell therapies, especially the CAR T cell products, have increasingly become an integral component of cancer care for patients with hematologic (blood-based) malignancies, said Stephan Grupp, MD, PhD, co-chair of the SITC Immune Effector Cell-Related Adverse Events Expert Panel. Its crucial for oncologists and hematologists to understand the toxicities that may arise after treatment with these agents, so that they may safely offer these potentially lifesaving therapies to patients.

Immune effector cells are made from a patients own cells or in some cases from cells taken from a donor, usually from the blood. When these immune effector cells are infused back into a patient, they become living drugs that expand and persist in the body to attack cancer cells. This is one reason why immune effector cell therapies can sometimes produce long-lasting disease control, with some patients remaining cancer free for years after treatment. However, the therapies may cause unique side effects which can be similar to severe infections.

Clinicians need to feel comfortable identifying the signs and symptoms of common adverse events such as cytokine release syndrome and neurotoxicity so that they can appropriately intervene, said Marcela Maus, MD, PhD, co-chair of the SITC Immune Effector Cell-Related Adverse Events Expert Panel. In many cases, the adverse events associated with immune effector cell therapies may be safely managed, but prompt recognition is crucial.

The SITC Cancer Immunotherapy Guidelines were developed by leading experts to help hematologists and oncologists determine when and how to best use immunotherapy to treat their patients. The published disease-state specific guidelines provide evidence- and expert consensus-based recommendations on selection of appropriate immunotherapy treatments, toxicity management, biomarkers, and considerations for patient quality of life. SITC has published clinical practice guidelines for acute leukemia, bladder carcinoma, cutaneous melanoma, head and neck squamous cell carcinoma, multiple myeloma, non-small cell lung cancer, prostate cancer and renal cell carcinoma. Additional guidelines in development will cover lymphoma, breast cancer, hepatocellular carcinoma, non-melanoma skin cancer and immune checkpoint inhibitor-related adverse events.

Cell therapy is one of the major advances in cancer treatment in the past decade, and its reasonable to expect more of these cell therapies to be developed, and to see their use extend beyond very specialized treatment centers. SITC recognized the growing importance and use of cell therapy and therefore developed this new clinical practice guideline to assist physicians with management of the unique side effects, said SITC President Mario Sznol, MD. This is the tenth manuscript published in SITCs Cancer Immunotherapy Guidelines series, a critically important effort to educate physicians and other health care providers, and improve outcomes for cancer patients receiving FDA-approved immunotherapies.

SITC is a proponent for collaboration and harmonization of efforts between like-minded organizations whenever possible. SITC wishes to thank the American Society of Hematology (ASH), the American Society for Transplantation and Cellular Therapy (ASTCT), the Foundation for the Accreditation of Cellular Therapy (FACT) at the University of Nebraska Medical Center, and the Emily Whitehead Foundation for providing representatives to serve on SITCs Immune Effector Cell-related Adverse Events Expert Panel to aid in the development of this clinical practice guideline.

In addition to the published manuscript, SITC is also offering several different opportunities to help clinicians understand and implement the guidelines into their practice, including the live webinars and on-demand modules hosted on the SITC website. In 2021, SITC will host a live, free webinar during which attendees will be able to learn more about the recommendations included in this clinical practice guideline and ask questions of expert faculty. Registration information will be available in the coming weeks.

About SITCEstablished in 1984, the Society for Immunotherapy of Cancer (SITC) is a nonprofit organization of medical professionals dedicated to improving cancer patient outcomes by advancing the development, science and application of cancer immunotherapy and tumor immunology. SITC is comprised of influential basic and translational scientists, practitioners, health care professionals, government leaders and industry professionals around the globe. Through educational initiatives that foster scientific exchange and collaboration among leaders in the field, SITC aims to one day make the word cure a reality for cancer patients everywhere. Learn more about SITC, our educational offerings and other resources at sitcancer.org and follow us on Twitter, LinkedIn, Facebook and YouTube.

About JITCJournal for ImmunoTherapy of Cancer (JITC) is the official open access, peer-reviewed online journal of the Society for Immunotherapy of Cancer featuring an impact factor of 10.252. The journal publishes high-quality articles on all aspects of tumor immunology and cancer immunotherapy, on subjects across the basic science-translational-clinical spectrum. JITC publishes original research articles, literature reviews, position papers and practice guidelines, and case reports; invited commentaries may also be commissioned by the journal editors. These articles, freely accessible at jitc.bmj.com, make JITC the leading forum for tumor immunology and cancer immunotherapy research. Follow JITC on Twitter @jitcancer.

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The challenges of gene and cell therapists can be divided into three broad categories based on disease, development of therapy, and funding.

Challenges based on the disease characteristics: Disease symptoms of most genetic diseases, such as Fabrys, hemophilia, cystic fibrosis, muscular dystrophy, Huntingtons, and lysosomal storage diseases are caused by distinct mutations in single genes. Other diseases with a hereditary predisposition, such as Parkinsons disease, Alzheimers disease, cancer, and dystonia may be caused by variations/mutations in several different genes combined with environmental causes. Note that there are many susceptible genes and additional mutations yet to be discovered. Gene replacement therapy for single gene defects is the most conceptually straightforward. However, even then the gene therapy agent may not equally reduce symptoms in patients with the same disease caused by different mutations, and even the samemutationcan be associated with different degrees of disease severity. Gene therapists often screen their patients to determine the type of mutation causing the disease before enrollment into a clinical trial.

The mutated gene may cause symptoms in more than one cell type. Cystic fibrosis, for example, affects lung cells and the digestive tract, so the gene therapy agent may need to replace the defective gene or compensate for its consequences in more than one tissue for maximum benefit. Alternatively, cell therapy can utilizestem cellswith the potential to mature into the multiple cell types to replace defective cells in different tissues.

In diseases like muscular dystrophy, for example, the high number of cells in muscles throughout the body that need to be corrected in order to substantially improve the symptoms makes delivery of genes and cells a challenging problem.

Some diseases, like cancer, are caused by mutations in multiple genes. Although different types of cancers have some common mutations, every tumor from a single type of cancer does not contain the same mutations. This phenomenon complicates the choice of a single gene therapy tactic and has led to the use of combination therapies and cell elimination strategies. For more information on gene and cell therapy strategies to treat cancer, please refer to the Cancer and Immunotherapy summary in the Disease Treatment section.

Disease models in animals do not completely mimic the human diseases and viralvectorsmay infect various species differently. The testing of vectors in animal models often resemble the responses obtained in humans, but the larger size of humans in comparison to rodents presents additional challenges in the efficiency of delivery and penetration of tissue.Gene therapy, cell therapy, and oligonucleotide-based therapy agents are often tested in larger animal models, including rabbit, dog, pig and nonhuman primate models. Testing human cell therapy in animal models is complicated by immune rejections. Furthermore, humans are a very heterogeneous population. Their immune responses to the vectors, altered cells, or cell therapy products may differ or be similar to results obtained in animal models.

Challenges in the development of gene and cell therapy agents: Scientific challenges include the development of gene therapy agents that express the gene in the relevant tissue at the appropriate level for the desired duration of time. There are a lot of issues in that once sentence, and while these issues are easy to state, each one requires extensive research to identify the best means of delivery, how to control sufficient levels or numbers of cells, and factors that influence duration of gene expression or cell survival. After the delivery modalities are determined, identification and engineering of a promoter and control elements (on/off switch and dimmer switch) that will produce the appropriate amount of protein in the target cell can be combined with the relevant gene. This gene cassette is engineered into a vector or introduced into thegenomeof a cell and the properties of the delivery vehicle are tested in different types of cells in tissue culture. Sometimes things go as planned and then studies can be moved onto examination in animal models. In most cases, the gene/cell therapy agent may need to be improved further by adding new control elements to obtain the desired responses in cells and animal models.

Furthermore, the response of the immune system needs to be considered based on the type of gene or cell therapy being undertaken. For example, in gene or cell therapy for cancer, one aim is to selectively boost the existing immune response to cancer cells. In contrast, to treat genetic diseases like hemophilia and cystic fibrosis the goal is for the therapeutic protein to be accepted as an addition to the patients immune system.

If the new gene is inserted into the patients cellularDNA, the intrinsic sequences surrounding the new gene can affect its expression and vice versa. Scientists are now examining short DNA segments that may insulate the new gene from surrounding control elements. Theoretically, these insulator sequences would also reduce the effect of vector control signals in the gene cassette on adjacent cellular genes. Studies are also focusing on means to target insertion of the new gene into safe areas of the genome, to avoid influence on surrounding genes and to reduce the risk of insertional mutagenesis.

Challenges of cell therapy include the harvesting of the appropriate cell populations and expansion or isolation of sufficient cells for one or multiple patients. Cell harvesting may require specific media to maintain the stem cells ability toself-renew and mature into the appropriate cells. Ideally extra cells are taken from the individual receiving therapy. Those additional cells can expand in culture and can be induced to becomepluripotent stem cells(iPS), thus allowing them to assume a wide variety of cell types and avoiding immune rejection by the patient. The long term benefit of stem cell administration requires that the cells be introduced into the correct target tissue and become established functioning cells within the tissue. Several approaches are being investigated to increase the number of stem cells that become established in the relevant tissue.

Another challenge is developing methods that allow manipulation of the stem cells outside the body while maintaining the ability of those cells to produce more cells that mature into the desired specialized cell type. They need to provide the correct number of specialized cells and maintain their normal control of growth and cell division, otherwise there is the risk that these new cells may grow into tumors.

Challenges in funding: In most fields, funding for basic or applied research for gene and cell therapy is available through the National Institutes of Health (NIH) and private foundations. These are usually sufficient to cover the preclinical studies that suggest a potential benefit from a particular gene and cell therapy. Moving into clinical trials remains a huge challenge as it requires additional funding for manufacturing of clinical grade reagents, formal toxicology studies in animals, preparation of extensive regulatory documents, and costs of clinical trials.Biotechnology companies and the NIH are trying to meet the demand for this large expenditure, but many promising therapies are slowed down by lack of funding for this critical next phase.

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Gene & Cell Therapy FAQs | ASGCT - American Society of ...

What are cells and genes?

Cells are the basic building blocks of all living things, and genes can be found deep within cells. Genes are small sections of DNA that carry genetic information and instructions for making proteins, which help build and maintain the body1.

Every person has around 20,000 genes and two copies of each of their genesone from each parent. Small variations in genes result in differences in peoples appearance and, potentially, health1.

Genetic diseases happen when a critical piece or whole section of DNA is substituted, deleted or duplicated2. These changes are called genetic mutations3. Some serious genetic diseases caused by genetic mutations can be passed to future generations4.

Cell therapy and gene therapy are overlapping fields of biomedical research and treatment6. Both therapies aim to treat, prevent, or potentially cure diseases, and both approaches have the potential to alleviate the underlying cause of genetic diseases and acquired diseases6. But, cell and gene therapies work differently.

Cell therapy aims to treat diseases by restoring or altering certain sets of cells orby using cells to carry a therapy through the body5. With cell therapy, cells are cultivated or modified outside the body before being injected into the patient. The cells may originate from the patient (autologous cells) or a donor (allogeneic cells)6.

Gene therapy aims to treat diseases by replacing, inactivating or introducing genes into cells either inside the body (in vivo) or outside of the body (ex vivo)6.

Some therapies are considered both cell and gene therapies. These therapies work by altering genes in specific types of cells and inserting them into the body.

Learn more about how we use cell and gene therapies and why they are important

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What is cell and gene therapy | Novartis

By Pat Anson, PNN Editor

Scientists have developed an experimental drug that can lure stem cells to damaged tissues and help them heal -- a discovery being touted as a major advancement in the field of regenerative medicine.

The findings, recently published in the Proceedings of the National Academy of Sciences (PNAS), could improve the effectiveness of stem cell therapy in treating spinal cord injuries, stroke, amyotrophic lateral sclerosis(ALS), Parkinsons disease and other neurodegenerative disorders. It could also expand the use of stem cells to treat conditions such as heart disease and arthritis.

The ability to instruct a stem cell where to go in the body or to a particular region of a given organ is the Holy Grail for regenerative medicine, said lead authorEvan Snyder, MD, director of theCenter for Stem Cells & Regenerative Medicineat Sanford Burnham Prebys Medical Discovery Institute in La Jolla, CA. Now, for the first time ever, we can direct a stem cell to a desired location and focus its therapeutic impact.

Over a decade ago, Snyder and his colleagues discovered that stem cells are drawn to inflammation -- a biological fire alarm that signals tissue damage has occurred. However, using inflammation as a therapeutic lure for stem cells wasnt advisable because they could further inflame diseased or damaged organs, joints and other tissue.

To get around that problem, scientists modified CXCL12 -- an inflammatory molecule that Snyders team discovered could guide stem cells to sites in need of repair to create a drug called SDV1a. The new drug works by enhancing stem cell binding, while minimizing inflammatory signals.

Since inflammation can be dangerous, we modified CXCL12 by stripping away the risky bit and maximizing the good bit, Snyder explained. Now we have a drug that draws stem cells to a region of pathology, but without creating or worsening unwanted inflammation.

To demonstrate its effectiveness, Snyders team injected SDV1a and human neural stem cells into the brains of mice with a neurodegenerative disease called Sandhoff disease. The experiment showed that the drug helped stem cells migrate and perform healing functions, which included extending lifespan, delaying symptom onset, and preserving motor function for much longer than mice that didnt receive the drug. Importantly, the stem cells also did not worsen the inflammation.

Researchers are now testing SDV1as ability to improve stem cell therapy in a mouse model of ALS, also known as Lou Gehrigs disease, which is caused by a progressive loss of motor neurons in the brain. Previous studies conducted by Snyders team found that broadening the spread of neural stem cells helps more motor neurons survive so they are hopeful that SDV1a will improve the effectiveness of neuroprotective stem cells and help slow the onset and progression of ALS.

We are optimistic that this drugs mechanism of action may potentially benefit a variety of neurodegenerative disorders, as well as non-neurological conditions such as heart disease, arthritis and even brain cancer, says Snyder. Interestingly, because CXCL12 and its receptor are implicated in the cytokine storm that characterizes severe COVID-19, some of our insights into how to selectively inhibit inflammation without suppressing other normal processes may be useful in that arena as well.

Snyders research is supported by the National Institutes of Health, U.S. Department of Defense, National Tay-Sachs & Allied Disease Foundation, Childrens Neurobiological Solutions Foundation, and the California Institute for Regenerative Medicine (CIRM).

Thanks to decades of investment in stem cell science, we are making tremendous progress in our understanding of how these cells work and how they can be harnessed to help reverse injury or disease, says Maria Millan, MD, president and CEO of CIRM. This drug could help speed the development of stem cell treatments for spinal cord injury, Alzheimers, heart disease and many other conditions for which no effective treatment exists.

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New Drug Could Improve Effectiveness of Stem Cell Therapy - Pain News Network

A study published in OncoTargets and Therapy found that CD19 chimeric antigen receptor (CAR) T-cells derived from patients who relapsed after receiving allogeneic hematopoietic cell transplantation (alloHCT) with a low level of donor chimerism were effective for salvage therapy. These T-cells could be restored to complete donor chimerism after 12 days of in vitro culture.

The study included nine patients with B-cell acute lymphocytic leukemia (ALL) who had low donor chimerism levels and relapsed after alloHCT. The first three patients received CD19 CAR T-cell therapy using cells derived from autologous peripheral blood mononuclear cells (PBMCs), comprising a mixture of patient and original donor cells, as their donors could not provide PBMCs.

Samples from the subsequent six patients were investigated in vitro only. The changes in the degree of donor chimerism, function of the CD19 CAR T-cells, and T-cells in all nine patients were analyzed in vitro.

CAR T-cells and T-cells in all nine patients showed complete donor chimerism restoration after a 12-day culture period in vitro. These CD19 CAR T-cells demonstrated strong cytotoxicity toward Nalm 6 cells in vitro except in two patients. In the latter patients, the absolute numbers of all subsets especially the CD8+ T-cell absolute numbers in peripheral blood were very low.

Two patients showed relatively short durations from transplant to recurrence and received chemotherapy after relapse. Among patients receiving CD19 CAR T-cell therapy, the most commonly observed adverse event was grade 1/2 cytokine release syndrome.

No patients had acute graft-versus-host disease during treatment. Among the first three treated patients, the first two achieved complete response with complete restoration of donor chimerism. Patient three, who received the same CD19 CAR-T cell therapy as the first two, did not respond to this therapy.

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Study Observes Changes in Donor Chimerism in Patients with ALL Receiving CAR T-Cell Therapy - DocWire News

New York, Nov. 27, 2020 (GLOBE NEWSWIRE) -- Reportlinker.com announces the release of the report "Asia Pacific Cell Therapy Instruments Market Forecast to 2027 - Covid-19 Impact and Regional Analysis - By Product ; Cell Type ; Process ; End User, and Country" - https://www.reportlinker.com/p05989496/?utm_source=GNW However, the low success rate of cell therapies and the high cost of cell-based research is expected to restrain the market growth during the forecast period.

Cell therapy typically involves the administration of somatic cell preparations by injecting or grafting it into the patients body for the treatment of diseases or traumatic damages.The procedure is used to cure diabetes, neurological disorders, related injuries, several cancer types, bones and joints, and genetic disorders.

Continuous research and development activities have led to unique cell therapeutic instruments for the improvement of immune system and efficient treatment of genetic disorders. Various market players provide several consumables such as reagent kits and enzymes as well as devices, equipment, and software to perform various cell therapy processes.

The use of instruments is essential for handling cell therapies such as NSC, PSC, MSC, T cells, and HSC.These cell therapy products are derived from animals or human cells and thus need to be protected from contamination.

The instruments used in cell therapies help provide protection against contamination and allow scaling up of transplantation. Companies such as Hitachi Chemical Advanced Therapeutics Solutions Corning Incorporated; Thermo Fisher Scientific Inc., MiltenyiBiotec, LLC; Invetech; and Cytiva (General Electric Company) have introduced various equipment and consumables for the cell therapy procedures.

The global COVID-19 emergency has been particularly affecting the supply chain worldwide.The supply chain disruptions, along with the enormous demand for effective therapies for the treatment of COVID-19, have put the healthcare research industry in a crucial situation in the Asia Pacific region.

However, many medical companies have realized the importance of cell therapy in the treatment of COVID 19, which would raise its demand in the coming years.

The Asia Pacific cell therapy instruments market, by product, is segmented into consumables, software, equipment, and systems.The consumables segment held the largest share of the market in 2019 and is expected to register the highest CAGR during the forecast period.

On the basis of cell type, the cell therapy instruments market is segmented into animal cells and human cells. The human cells segment held a larger share of the market in 2019 and is estimated to register a higher CAGR during the forecast period.

On the basis of process, the Asia Pacific cell therapy instruments market is segmented into cell processing; cell preservation, distribution, and handling; and process monitoring and quality control.The cell processing segment held the largest share of the market in 2019 and is estimated to register the highest CAGR during the forecast period.

The Asia Pacific cell therapy instruments market, based on end user, is segmented into life science research companies, research institutes, and other end users. The life science research companies segment accounted for the largest share of the market in 2019 and is anticipated to register the highest CAGR during the forecast period.

A few of the major primary and secondary sources associated with the Asia Pacific cell therapy instruments market are National Center for Biotechnology Information (NCBI); World Health Organization (WHO); Medical Research Future Fund (MRFF); Asia-Pacific Economic Corporation (APEC); and Global Institute of Stem Cell Therapy and Research (GIOSTAR).Read the full report: https://www.reportlinker.com/p05989496/?utm_source=GNW

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Asia Pacific Cell Therapy Instruments Market Forecast to 2027 - Covid-19 Impact and Regional Analysis - By Product ; Cell Type ; Process ; End User,...

I would never have thought that a mutant flu virus could create that big panic in people at each and every corner of the world. In fact, the past few days in quarantine have really made me realize where exactly the humanity is heading.

For majority of us, our lives are on a temporary hold while the world is dealing with the global pandemic of COVID-19. In fact, past three months have served as something of an inflection for many countries and as the number of infected patients have surpassed one million, there is already a sense of worry looming across the industries.

Since Coronavirus is a newly identified pathogen, there is no known pre-existing immunity observed in humans and therefore everyone is assumed to be susceptible. While researchers all across the globe are putting efforts to develop immediate treatment, there are speculations, based on credible evidence, that infected patients treated with Stem Cell Therapy are more likely to survive the disease. Specifically, mesenchymal stem cells can be effectively used to improve patients resistance to the SARS-CoV-2 virus induced pneumonia as these cells have the potential to repair damaged tissues in the patients respiratory system leading to speedy recovery.

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Recent Trends

Recent trends across several social medial platform highlight the immense efforts put by several stakeholders validating the therapeutic potential of stem cells. Specifically, Id like to appreciate the efforts of Azidus Brasil, CAR-T (Shanghai) Biotechnology, Janssen Research & Development, Mesoblast, Millennium Pharmaceutical, Pfizer, Stem Cell Arabia, Tianhe Stem Cell Biotechnologies and Tuohua Biological Technology that have progressed significantly in-terms of development of stem cell-based therapies to defeat this global pandemic of COVID-19 infection.

Looking at the efforts made by different pharmaceutical companies, I felt assured and hopeful as many patients have successfully recovered, while others are at the verge of getting discharged by using these novel classes of regenerative medicines.

What are the Key Hubs for Stem Cell Research?

With the virus strengthening its foothold in several countries across the globe, the threat of pandemic has become real and the question is are we ready?

As per the recent study published by Roots Analysis, the efforts for development of stem cell therapies have been undertaken by players all across the globe, majority of the developers (45%) are based in Asia-Pacific regions; China, South Korea and Japan; followed by developed countries, such as the US, Germany, Belgium, Spain and the UK.

Roots Analysis, in its recent report, has captured the clinical and research landscape of stem cell therapy-based treatment. To know further, check out the report here

Expert Opinion

In fact. several industry stakeholders are quite optimistic about the future market potential of stem cell-based therapies.

Bottom Line: Stay Cautious, Stay Hopeful!!

In the midst of the anxiety, worry, and uncertainty surrounding the COVID-19 pandemic, each day seems to bring news thats worse than the day before. However, remember this is not for the first time any pandemic outbreak has taken place in the history of mankind. About 200,000 (~20%) patients have already been recovered from this disease. The sky is not falling and for sure, life would return to normal. Stay cautious, stay hopeful.

For further information, check out the report here

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About Roots Analysis

Roots Analysis is one of the fastest growing market research companies, sharing fresh and independent perspectives in the bio-pharmaceutical industry. The in-depth research, analysis and insights are driven by an experienced leadership team which has gained many years of significant experience in this sector. If youd like help with your growing business needs, get in touch at [emailprotected]

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Stem Cell Therapy: A Ray of Hope to End Global Pandemic of COVID-19 | Roots Analysis - Khabar South Asia

The report aims to offer a clear picture of the current scenario and future growth of the global Cell Therapy Technologies Market market. The report provides scrupulous analysis of global market by thoroughly reviewing several factors of the market such as vital segments, regional market condition, market dynamics, investment suitability, and key players operating in the market. Besides, the report delivers sharp insights into present and forthcoming trends & developments in the global market.

The report articulates the key opportunities and factors propelling the global Cell Therapy Technologies Market market growth. Also, threats and limitations that have the possibility to hamper the market growth are outlined in the report. Further, Porters five forces analysis that explains the bargaining power of suppliers and consumers, competitive landscape, and development of substitutes in the market is also sketched in the report.

For More Detail Insights, Download Sample Copy of the Report at: https://www.researchdive.com/download-sample/1819

The report reveals various statistics such as predicted market size and forecast by analyzing the major factors and by assessing each segment of the global Cell Therapy Technologies Market market. Regional market analysis of these segments is also provided in the report. The report segments the global market into four main regions including Asia-Pacific, Europe, North America, and LAMEA. Moreover, these regions are sub-divided to offer an exhaustive landscape of the Cell Therapy Technologies Market market across key countries in respective regions. Furthermore, the report divulges some of the latest advances, trends, and upcoming opportunities in every region.

Furthermore, the report profiles top players active in the global Cell Therapy Technologies Market market. A comprehensive summary of 10 foremost players operating in the global market is delivered in the report to comprehend their position and footmark in the industry. The report highlights various data points such as short summary of the company, companys financial status and proceeds, chief company executives, key business strategies executed by company, initiatives undertaken & advanced developments by the company to thrust their position and grasp a significant position in the market.

RESEARCH METHODOLOGY

The research report is formed by collating different statistics and information concerning the Cell Therapy Technologies Market market. Long hours of deliberations and interviews have been performed with a group of investors and stakeholders, including upstream and downstream members. Primary research is the main part of the research efforts; however, it is reasonably supported by all-encompassing secondary research. Numerous product type literatures, company annual reports, market publications, and other such relevant documents of the leading market players have been studied, for better & broader understanding of market penetration. Furthermore, medical journals, trustworthy industry newsletters, government websites, and trade associations publications have also been evaluated for extracting vital industry insights.

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KEY MARKET BENEFITS

KEY MARKET SEGMENTS

The global Cell Therapy Technologies Market market is segmented on the basis of the following:

Global Cell Therapy Technologies Market Market By Product Type:

Global Cell Therapy Technologies Market Market By Applications:

Global Cell Therapy Technologies Market Market By Regions:

Top Leading key players stated in Global Cell Therapy Technologies Market Market report are:

Beckman Coulter, Becton, Dickinson and Company, GE Healthcare, Lonza, Merck KGaA, Miltenyi Biotec, STEMCELL Technologies, Inc., Terumo BCT, Thermo Fisher Scientific

The report also summarizes other important aspects including financial performance, product portfolio, SWOT analysis, and recent strategic moves and developments of the leading players.

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Widening: Turn your losses to profit with Exclusive Insight on Cell Therapy Technologies Market - The Courier

A novel CAR T-cell therapy developed by researchers at UCL and designed to target cancerous tumours, has shown promising early results in children with neuroblastoma, a rare form of childhood cancer.

For this proof-of-principle study, researchers at the UCL Great Ormond Street Institute for Child Health (GOS ICH) and the UCL Cancer Institute modified the patient's own T-cells (a type of immune cell), equipping them to recognise and kill neuroblastoma tumour cells.

Twelve children with relapsed or refractory (where the disease does not respond to treatment) neuroblastoma were treated as part of the Cancer Research UK-funded phase I clinical trial.

The research, published in Science Translational Medicine, is one of the first studies to demonstrate CAR T-cells achieving rapid regression against a solid cancer (non-blood cancer). Although the beneficial effects only lasted a short while, the study provides important evidence that this specific CAR T-cell treatment could be used as a future treatment for children with solid cancers.

Neuroblastoma is a rare type of cancer that mostly affects babies and young children and develops from specialised nerve cells (neuroblasts) left behind from a baby's development in the womb.

Up to 100 children in the UK are diagnosed with neuroblastoma each year. Current treatment for children with an aggressive type of neuroblastoma includes surgical removal, chemotherapy with stem-cell transplant, radiotherapy and antibody therapy. Despite this intensive treatment long-term survival is between 50-60 per cent.

In CAR T-cell therapy, a type of immunotherapy, T-cells are engineered to contain a molecule called a chimeric antigen receptor (CAR) on their surface which can specifically recognise cancerous cells.

For this study the patients' own T-cells were modified with a CAR to target the GD2 surface protein, which is highly abundant on almost all neuroblastoma cells, but found at very low levels in healthy cells.

Researchers found that when using a sufficient dose* of the modified CAR T-cells, this treatment induced rapid reduction in tumour size in some of the patients treated. These effects were transient. Importantly, in all patients the CAR T-cells did not cause any harmful side effects in healthy tissues that express the GD2 molecule.

Lead author, Dr Karin Straathof, Research group leader at UCL GOS ICH and Consultant Paediatric Oncologist at Great Ormond Street Hospital NHS Trust said: "It's encouraging to see the anti-tumour activity induced by these modified T-cells in some of the patients on this study.

"While the anti-tumour activity seen was only transient, it provides an important proof-of-principle that CAR T-cells directed at the GD2 molecule could be used against solid cancers in children.

"New treatments are needed for high-risk neuroblastoma and with more research we hope to develop this further into a treatment that results in lasting responses and increases the number of patients that can be cured."

Senior author, Dr Martin Pule (UCL Cancer Institute) said: "Targeting of solid cancers by CAR T-cells is dependent on their infiltration and expansion within the tumour microenvironment, and thus far fewer clinical responses have been reported.

"The rapid regression in neuroblastoma cells is promising, particularly as this activity was observed in the absence of neurotoxicity which occurs with antibody-based approaches that target GD2."

Dr Pule added: "Targeting neuroblastoma with GD2 CAR T-cells appears to be a valid and safe strategy but requires further modification to promote CAR T-cell longevity."

Dr Sue Brook, medical advisor at Cancer Research UK, said: "Children who have hard to treat cancers like neuroblastoma have limited treatment options open to them, especially when the cancer returns.

"The early results for the GD2 CAR-T treatment look promising, especially due to the initial safety data. However more work is needed on making the response last longer, and we are looking forward to seeing the next steps in its development."

The research team are preparing for their next clinical study in collaboration with Autolus, a clinical-stage biopharmaceutical company developing next-generation, programmed T-cell therapies for the treatment of cancer. This study will evaluate AUTO6NG, which builds on this approach utilising the same GD2 CAR alongside additional programming modules designed to enhance efficacy and persistence.

Reference:Straathof K, Flutter B, Wallace R, et al. Antitumor activity without on-target off-tumor toxicity of GD2chimeric antigen receptor T cells in patients with neuroblastoma.Sci. Transl. Med.2020;12(571). doi:10.1126/scitranslmed.abd6169This article has been republished from the following materials. Note: material may have been edited for length and content. For further information, please contact the cited source.

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CAR T-Cell Therapy Shows Promising Early Results in Children With Neuroblastoma - Technology Networks