DUBLIN, Feb. 9, 2021 /PRNewswire/ -- The "Cell Therapy Market by Cell Type, Therapy Type, Therapeutic Area, and End User: Global Opportunity Analysis and Industry Forecast, 2020-2027" report has been added to ResearchAndMarkets.com's offering.

The global cell therapy market accounted for $7,754. 89 million in 2019, and is expected to reach $48,115. 40 million by 2027, registering a CAGR of 25. 6% from 2020 to 2027.

Cell therapy involves administration of somatic cell preparations for treatment of diseases or traumatic damages. Cell therapy aims to introduce new, healthy cells into a patient's body to replace diseased or missing ones.

This is attributed to the fact that specialized cells, such as brain cells, are difficult to obtain from human body. In addition, specialized cells typically have a limited ability to multiply, making it difficult to produce sufficient number of cells required for certain cell therapies. Some of these issues can be overcome through the use of stem cells. In addition, cells such as blood and bone marrow cells, mature, immature & solid tissue cells, adult stem cells, and embryonic stem cells are widely used in cell therapy procedures.

Moreover, transplanted cells including induced pluripotent stem cells (iPSCs), embryonic stem cells (ESCs), neural stem cells (NSCs), and mesenchymal stem cells (MSCs) are divided broadly into two main groups including autologous cells and non-autologous cells. Development of precision medicine and advancements in Advanced Therapies Medicinal Products (ATMPS) in context to their efficiency and manufacturing are expected to be the major drivers for the market. Furthermore, automation in adult stem cells and cord blood processing and storage are the key technological advancements that fuel growth of the market for cell therapy.

In addition, growth in aging patient population, The rise in cell therapy transplantations globally, and surge in disease awareness drive growth of the global cell therapy market. Furthermore, The rise in adoption of human cells over animal cells for cell therapeutics research, technological advancements in field of cell therapy, and increase in incidences of diseases such as cancer, cardiac abnormalities, and organ failure are the key factors that drive growth of the global market.

Moreover, implementation of stringent government regulations regarding the use of cell therapy is anticipated to restrict growth of the market. On the contrary, surge in number of regulations to promote stem cell therapy and increase in funds for research in developing countries are expected to offer lucrative opportunities to the market in the future.

The global cell therapy market is categorized on the basis of therapy type, therapeutic area, cell type, end user, and region. On the basis of therapy type, the market is segregated into autologous and allogenic. By therapeutics, it is classified into malignancies, musculoskeletal disorders, autoimmune disorders, dermatology, and others.

The global cell therapy market is categorized on the basis of therapy type, therapeutic, cell type, end user and region. On the basis of therapy type, the market is segregated into autologous and allogenic. By therapeutic area, it is classified into malignancies, musculoskeletal disorders, autoimmune disorders, dermatology, and others. On the basis of cell type, it is segregated into stem cell therapy and non-stem cell type. On the basis of end user, it is segregated into hospital & clinics and academic & research institutes. On the basis of region, the market is studied across North America, Europe, Asia-Pacific, and LAMEA.

Key Benefits

Key Topics Covered:

Chapter 1: Introduction1.1. Report Description1.2. Key Benefits for Stakeholders1.3. Key Market Segments1.4. Research Methodology1.4.1. Secondary Research1.4.2. Primary Research1.4.3. Analyst Tools & Models

Chapter 2: Executive Summary2.1. Key Findings of the Study2.2. Cxo Perspective

Chapter 3: Market Overview3.1. Market Definition and Scope3.2. Key Findings3.2.1. Top Player Positioning3.2.2. Top Investment Pockets3.2.3. Top Winning Strategies3.3. Porter's Five Forces Analysis3.4. Impact Analysis3.4.1. Drivers3.4.1.1. Technological Advancements in the Field of Cell Therapy3.4.1.2. The Rise in Number of Cell Therapy Clinical Studies3.4.1.3. The Rise in Adoption of Regenerative Medicine3.4.2. Restraint3.4.2.1. Developing Stage and Pricing3.4.3. Opportunity3.4.3.1. High Growth Potential in Emerging Markets3.5. Impact of Covid-19 on Cell Therapy Market

Chapter 4: Cell Therapy Market, by Cell Type4.1. Overview4.1.1. Market Size and Forecast4.2. Stem Cell4.2.1. Key Market Trends and Opportunities4.2.2. Market Size and Forecast, by Region4.2.3. Market Size and Forecast, by Type4.2.3.1. Bone Marrow, Market Size and Forecast4.2.3.2. Blood, Market Size and Forecast4.2.3.3. Umbilical Cord-Derived, Market Size and Forecast4.2.3.4. Adipose-Derived Stem Cell, Market Size and Forecast4.2.3.5. Others (Placenta, and Nonspecific Cells), Market Size and Forecast4.3. Non-Stem Cell4.3.1. Key Market Trends and Opportunities4.3.2. Market Size and Forecast, by Region

Chapter 5: Cell Therapy Market, by Therapy Type5.1. Overview5.1.1. Market Size and Forecast5.2. Autologous5.2.1. Key Market Trends and Opportunities5.2.2. Market Size and Forecast, by Region5.2.3. Market Analysis, by Country5.3. Allogeneic5.3.1. Key Market Trends and Opportunities5.3.2. Market Size and Forecast, by Region5.3.3. Market Analysis, by Country

Chapter 6: Cell Therapy Market, by Therapeutic Area6.1. Overview6.1.1. Market Size and Forecast6.2. Malignancies6.2.1. Market Size and Forecast, by Region6.2.2. Market Analysis, by Country6.3. Musculoskeletal Disorders6.3.1. Market Size and Forecast, by Region6.3.2. Market Analysis, by Country6.4. Autoimmune Disorders6.4.1. Market Size and Forecast, by Region6.4.2. Market Analysis, by Country6.5. Dermatology6.5.1. Market Size and Forecast, by Region6.5.2. Market Analysis, by Country6.6. Others6.6.1. Market Size and Forecast, by Region6.6.2. Market Analysis, by Country

Chapter 7: Cell Therapy Market, by End-user7.1. Overview7.1.1. Market Size and Forecast7.2. Hospitals & Clinics7.2.1. Key Market Trends and Opportunities7.2.2. Market Size and Forecast, by Region7.2.3. Market Analysis, by Country7.3. Academic & Research Institutes7.3.1. Key Market Trends and Opportunities7.3.2. Market Size and Forecast, by Region7.3.3. Market Analysis, by Country

Chapter 8: Cell Therapy Market, by Region8.1. Overview8.2. North America8.3. Europe8.4. Asia-Pacific8.5. LAMEA

Chapter 9: Company Profiles9.1. Allosource9.1.1. Company Overview9.1.2. Company Snapshot9.1.3. Operating Business Segments9.1.4. Product Portfolio9.1.5. Key Strategic Moves and Developments9.2. Cells for Cells9.2.1. Company Overview9.2.2. Company Snapshot9.2.3. Operating Business Segments9.2.4. Product Portfolio9.3. Holostem Terapie Avanzate Srl9.3.1. Company Overview9.3.2. Company Snapshot9.3.3. Operating Business Segments9.3.4. Product Portfolio9.4. Jcr Pharmaceuticals Co. Ltd.9.4.1. Company Overview9.4.2. Company Snapshot9.4.3. Operating Business Segments9.4.4. Product Portfolio9.4.5. Business Performance9.4.6. Key Strategic Moves and Developments9.5. Kolon Tissuegene, Inc.9.5.1. Company Overview9.5.2. Company Snapshot9.5.3. Operating Business Segments9.5.4. Product Portfolio9.5.5. Key Strategic Moves and Developments9.6. Medipost Co. Ltd.9.6.1. Company Overview9.6.2. Company Snapshot9.6.3. Operating Business Segments9.6.4. Product Portfolio9.6.5. Business Performance9.7. Mesoblast Ltd9.7.1. Company Overview9.7.2. Company Snapshot9.7.3. Operating Business Segments9.7.4. Product Portfolio9.7.5. Business Performance9.8. Nuvasive, Inc.9.8.1. Company Overview9.8.2. Company Snapshot9.8.3. Operating Business Segments9.8.4. Product Portfolio9.8.5. Business Performance9.9. Osiris Therapeutics, Inc.9.9.1. Company Overview9.9.2. Company Snapshot9.9.3. Operating Business Segments9.9.4. Product Portfolio9.10. Stemedica Cell Technologies, Inc.9.10.1. Company Overview9.10.2. Company Snapshot9.10.3. Operating Business Segments9.10.4. Product Portfolio

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Worldwide Cell Therapy Industry to 2027 - Profiling Allosource, Medipost and Mesoblast Among Others - PRNewswire

Cardiac Stem Cells Comments Off on Worldwide Cell Therapy Industry to 2027 – Profiling Allosource, Medipost and Mesoblast Among Others – PRNewswire | February 10th, 2021

Paragon Biosciences, a life science innovator that creates, invests in and builds life science companies in biopharmaceuticals, cell and gene therapy and synthetic biology utilizing artificial intelligence, has launched CiRC Biosciences, a cell therapy company developing treatments for serious diseases with high, unmet needs with an initial focus on the eye."The addition of CiRC Biosciences to our portfolio builds upon our cell and gene therapy platform, an area that has tremendous potential to address serious genetic diseases," said Jeff Aronin, founder, chairman and chief executive officer, Paragon Biosciences. "CiRC Biosciences gives us the science to target retinal diseases that could lead to vision restoration with numerous other applications in the years ahead."CiRC Biosciences is currently advancing pre-clinical development of chemically induced retinal cells for vision restoration in Geographic Atrophy Age-Related Macular Degeneration (Dry AMD), which is the most common cause of irreversible vision loss over the age of 65, and advanced Retinitis Pigmentosa (RP), a genetic disorder that causes tunnel vision and eventual blindness. There are no U.S. Food & Drug Administration (FDA) approved treatments to restore vision loss in Dry AMD or RP.The company's novel mechanism of action is designed for direct chemical conversion of fibroblasts into other cell types using a cocktail of small molecules in an 11-day chemical conversion process. Pre-clinical studies have shown efficacy in blind mice that demonstrated vision restoration. CiRC Biosciences has provisional patent applications to protect its platform."Our technology transforms ordinary skin cells into specialized retinal cells using a cocktail of small molecules," said Sai Chavala, M.D., co-founder and chief scientific officer, CiRC Biosciences. "This process is potentially safer, quicker, more cost effective and easier to manufacturer than using traditional stem cells. Working with Paragon Biosciences to build and advance CiRC Biosciences provides us the opportunity to efficiently progress this technology through research and development stages.CiRC Biosciences first reported its discovery in the highly respected scientific journal Nature (April 15, 2020). A recently published New England Journal of Medicine article (Nov. 5, 2020) discussed CiRC's technology of using chemically induced cells to restore retinal function. The article concluded, "The new and emerging strategies for the rescue, regeneration, and replacement of photoreceptors suggest a bright future in the fight to preserve and restore vision in blinding eye diseases."The abstract in Nature is available here.Access to the NEJM article is available here.

Excerpt from:
Paragon Biosciences Expands Cell And Gene Therapy Platform - Contract Pharma

Skin Stem Cells Comments Off on Paragon Biosciences Expands Cell And Gene Therapy Platform – Contract Pharma | February 10th, 2021

Introduction

Head and neck squamous cell carcinoma (HNSCC) is one of the major causes of cancer-associated illness and death, with more than 600,000 newly diagnosed cases worldwide each year1 and a continuously increasing incidence rate.2 HNSCC includes cancers of the oral cavity, pharynx, and larynx. The anatomical structures of the head and neck can be damaged by the tumor itself or treatments such as surgical resection and chemoradiotherapy, which sometimes cause speech, swallowing, and breathing impairments.3,4 Patients with HNSCC have been shown to bear greater psychological distress than those with other types of cancer.5

Despite the currently available therapies, patients with advanced HNSCC still experience poor outcomes.68 For example >50% of patients with locoregionally advanced HNSCC experience recurrence or metastases development within 3 years of treatment.911 Treatment options for patients with the recurrent and metastatic disease following progression after a platinum-based regimen are limited, and the median overall survival of such patients is less than 7 months.1215

The recurrence and metastasis of HNSCC are facilitated by immune evasion;16 therefore, as one of the methods to inhibit immune evasion, the use of programmed death 1 (PD-1)/programmed death ligand 1 (PD-L1) pathway inhibitors is considered effective in the treatment of recurrent HNSCC.1719 Nivolumab, a fully human IgG4 antiPD-1 monoclonal antibody, has shown remarkable antitumor efficacy and safety when administered to patients with recurrent HNSCC whose disease had progressed within 6 months of platinum-based chemotherapy;19 Furthermore, nivolumab treatment has been shown to improve the quality of life of these patients.20 However, PD-1 inhibitors can upregulate T cells in vivo, which may lead to the development of graft-versus-host disease (GVHD) in patients after allogeneic hematopoietic stem cell transplantation (allo-HSCT).2123 To the best of the authors knowledge, no studies have investigated the safety and efficacy of nivolumab in patients with HNSCC after allo-HSCT. Here, we report the case of a patient who experienced excellent control of left buccal squamous cell carcinoma with nivolumab after the failure of platinum-based chemotherapy despite receiving allogeneic bone marrow transplantation.

Without any family history of tumor, a 33-year-old man was diagnosed with Philadelphia chromosome-positive T cell acute lymphoblastic leukemia on March 19, 2014. He received one course of vincristine and prednisone therapy and four courses of vincristine, daunorubicin, cyclophosphamide, and prednisone therapy. He was in complete remission at the end of therapy. Subsequently, allogeneic bone marrow transplantation was performed; the donor was his human leukocyte antigen (HLA)-haploidentical sibling (sister). He experienced chronic GVHD (c GVHD) of the oral cavity and skin 3 months after transplantation, for which he was treated with steroid hormone- and cyclosporine-based therapies. Skin rejection lasted for more than 3 years. Imatinib mesylate was administered for 2 years after transplantation, and his leukemia was well controlled.

In August 2018, the patient developed an ulcer of approximately 0.5 0.5 cm size in the left buccal mucosa; the ulcer was slightly painful and covered with white moss. In September 2018, the patient was admitted to Peking University Stomatological Hospital, where a biopsy of the buccal mucosa was performed. The pathology results showed the presence of squamous cell carcinoma in the left cheek. Unfortunately, this patient was not a right candidate for HNSCC in terms of exposure to risk factors, such as long terms of smoking and drinking. On October 10, 2018, 18F-fluorodeoxyglucose-positron emission tomography/computed tomography (CT) showed that the mass in the left cheek was metabolically active, which is consistent with the activity of a malignant tumor. One course of an adjuvant therapy regimen (nimotuzumab [200 mg d0] + docetaxel [60 mg d1, 8]+ nedaplatin [60 mg d2, 3]) was administered on October 26, 2018. Following this, the patient developed degree II thrombocytopenia and redness, swelling, and ulceration of the cheek, which had discharge with a peculiar smell. On November 29, 2018, a head and neck CT scan showed a left buccal malignant tumor with the destruction of the neighboring mandibular bone and lymph node enlargement in the left submaxillary region and right carotid sheath. The CT examination revealed disease progression. Following a multidisciplinary consultation in our hospital, surgery was not recommended; instead, a chemotherapy-based comprehensive treatment was recommended as a better option for the patient. The patient received chemotherapy with albumin paclitaxel (200 mg d1, 8)+ bleomycin (15,000 units d2, 9) from November 30, 2018 to January 9, 2019. On another CT scan, the curative effect was evaluated as partial remission (showed in Video 1, Figure 1A); subsequently, two courses of a chemotherapy regimen comprising nivolumab (140 mg d1) + albumin paclitaxel (200 mg d1, d8) were administered. A CT examination showed stable disease (SD) on March 12, 2019, following which the patient was administered 120 mg of nivolumab once every 2 weeks from March 15 to May 23, 2019. Another CT examination was performed on May 28, 2019 (showed in Video 2, Figure 1B). During the therapy course, the related tumor markers showed an overall downward trend, the new metastases did not appear, the patients status became better than before. Subsequently, another CT examination performed in August 02, 2019 showed the extent of the tumor was obvious reduction than before (showed in video 3, Figure 1C). And the corresponding CT report in August 02, 2019 was described as follows Compared with the CT on 28 May, 2019, the extent of the tumor in the left cheek became obviously smaller, the tubercle in the left submandibular and the lymph nodes in the left neck also became smaller. There were no other significant changes in this image. Most importantly, the patient did not develop any form of GVHD following nivolumab administration.

Figure 1 Head and neck CT images showing tumor before (A) and after treatment with nivolumab (B, C, respectively).

Abbreviation: CT, computed tomography.

Note: The arrows indicate the maximum length diameter of tumor or tumor site.

Reliable data on the clinical safety and efficacy of nivolumab in the treatment of recurrent or metastatic HNSCC have been obtained in a Phase III randomized clinical trial (CheckMate 141).19 In this trial, 361 patients with recurrent HNSCC for whom disease had progressed within 6 months after platinum-based chemotherapy were enrolled between May 29, 2014, and July 31, 2015. The median follow-up duration for overall survival (OS) was 5.1 months (range, 016.8 months). OS was significantly greater in patients randomized to receive nivolumab than in those who received standard second-line, single-agent systemic therapy with either methotrexate, docetaxel, or cetuximab (hazard ratio, 0.70; 97.73% confidence interval (CI), 0.510.96; P = 0.01). The median OS was 7.5 months (95% CI, 5.59.1) in the nivolumab group versus 5.1 months (95% CI, 4.06.0) in the standard therapy group. The one-year survival was also greater in patients who received nivolumab than in those who received standard therapy (36.0%vs. 16.6%). Furthermore, the response rate was higher in those who received nivolumab than in those who received standard therapy (13.3% vs 5.8%); however, the median progression-free survival was not significantly different between the groups (2.0 vs 2.3 months; P=0.32). In this study, patients who were treated with nivolumab had a longer OS than those treated with standard therapy, regardless of tumor PD-L1 expression or p16 status. Grade 3 or 4 treatment-related adverse events occurred in 13.1% of patients who received nivolumab and 35.1% of those who received standard therapy. Physical function, role functioning, and social functioning were stable in the nivolumab group, whereas they were substantially worse in the standard therapy group.20 Moreover, among Asian patients, the survival benefits were consistent with the global group.24

It was unclear whether nivolumab could be used in patients with recurrent HNSCC after allo-HSCT, though Khaddour et al proved the efficacy and safety of Pembrolizumab in patients who underwent allo-HSCT after relapsed and refractory Szary Syndrome and cutaneous squamous cell carcinoma.25 However, some case reports (Table 1) and clinical trials (Table 2) have reported the efficacy and safety of nivolumab when administrated to patients with recurrent hematological malignancies (mostly Hodgkins lymphoma) after allo-HSCT.

Table 1 Case Reports of Nivolumab Use After Allo-HSCT

Table 2 Studies on Nivolumab Use After Allo-HSCT

In Herbaux et al, nivolumab (3 mg/kg, once every 2 weeks) was administered to 20 patients with Hodgkins lymphoma who experienced relapse after allo-HSCT. The overall response rate was 95%, the 1-year progression-free survival rate was 58.2%, and the 1-year OS rate was 78.8%.26 Compared with other treatment options, nivolumab was more effective in these patients.2730 Haverkos et al reported results after a median follow-up duration was 428 days (range, 133833 days). After treatment with PD-1 inhibitors [nivolumab 3 mg/kg, once every 2 weeks (n = 28) and pembrolizumab (n =3)], the overall response rate of 31 patients with relapsed lymphoma after allo-HSCT was 77%, the median progression-free survival was 591 days (range,400644 days), and 68% of the patients survived to the end of the study.23 These two studies showed that nivolumab is effective when administered to patients with recurrent blood cancers after allo-HSCT, which is consistent with the results of several other case reports3134 and case series.35,36 The PD-1/PD-L1 pathway plays a key role in the regulation of the balance among T cell activation, T-cell tolerance, and immune-mediated tissue damage. This pathway protects healthy cells from excessive inflammatory or autoimmune responses.37,38 Some studies have shown that the activation of the PD-1/PD-L1 pathway can reduce acute and chronic GVHD, whereas its blockade can accelerate the graft-versus-host response and increase the associated mortality.21,22,39 It is unclear whether the PD-1 inhibitor nivolumab increases the risk of GVHD and the associated mortality in patients after allo-HSCT.23,26 Some clinical studies and case reports have shown that nivolumab treatment-related GVHD and consequent death in patients after allo-HSCT might be affected by the following factors. First, GVHD after antiPD-1 treatment has been observed most frequently in matched sibling donor transplants; for which Haverkos et al reported an incidence of 75%.23 In a Phase I pilot study, without GVHD or G3/G4 immune toxicity after receiving multiple doses of nivolumab was only among one patient whose donor source was Haploidentical+cord blood Fludarabine.40 Second, a history of GVHD, especially for the acute GVHD, may lead to an increased risk of nivolumab treatment-related GVHD after allo-HSCT. In a French cohort, all patients who presented with acute GVHD after nivolumab treatment had a prior history of acute GVHD, among which three patients presented with steroid-refractory nivolumab-induced GVHD, and GVHD was not observed among patients without a history of GVHD.26 This phenomenon was also observed in Steinerovs medical report.41 In the study by Haverkos et al, 63% of patients with a history of GVHD prior to antiPD-1 treatment developed treatment-emergent GVHD after receiving antiPD-1.23 Third, the shorter the interval between transplantation and nivolumab use, the greater the risk of GVHD. In the study by Herbaux et al, the median intervals between transplantation and nivolumab use in cases with the presence and absence of GVHD were 8.5 months and 28.5 months, respectively.26 In another study by Wang et al, the reported four patients all experienced immune-related adverse events following nivolumab treatment and the median time from transplantation to nivolumab use was 7.8 months.40 Fourth, dose is a risk factor for nivolumab treatment-related GVHD. In a case report, chronic skin GVHD was observed when the dose of nivolumab was adjusted from 0.5 mg/kg to 2 mg/kg.33 Other factors, such as immunosuppressive therapy at the time of nivolumab administration, may also influence nivolumab treatment-related GVHD. Recently, a comprehensive literature review was launched by Awais et al to assess the safety and efficacy of the use of checkpoint inhibitors (ipilimumab, nivolumab and pembrolizumab) in blood cancers before and after allo-HSCT. Collective data showed that checkpoint inhibitors use after allo-HSCT for post-transplant relapse had higher efficacy but the risk of GVHD was significant. Moreover, the investigation indicated that higher drug doses, shorter intervals between checkpoint inhibitors exposure and allo-HSCT and prior history of GVHD had a positive correlation with the risk of GVHD.42

In the present case, HNSCC was effectively controlled without any nivolumab treatment-related acute or chronic GVHD after nivolumab administration, while the weight loss being the only adverse event. After comprehensive analysis, we found that many factors may impede the development of nivolumab treatment-related GVHD in our patient. On one hand, the appropriate donor, no use of checkpoint inhibitors prior to allo-HSCT, the long interval between nivolumab administration and allo-HSCT (36 months) and the standard dose use of nivolumab were the negative factors for GVHD development. On the other hand, the chronic GVHD of the oral cavity and skin before nivolumab use might lead to the development of GVHD. However, it remained unknown what role the immunosuppressant therapy played in the occurrence of GVHD, though we definitely known that immunosuppressant was administered more than 2 years after allo-HSCT and discontinued for 2 years before treatment with nivolumab in our patient. Finally, whether the two primary cancers in our case affected the efficacy and safety of nivolumab by some unknown pathways were unclear, which needed further exploration.

Nivolumab has been shown to be effective in patients with HNSCC for whom platinum-based therapy has failed. However, little is known about the efficacy and safety of nivolumab in patients with HNSCC who have undergone allo-HSCT. Our case report shows that nivolumab could be used effectively and safely in such patients, however, more clinical trials are required to confirm these results.

This study was approved by the Medical Ethics Committee of Tianjin Medical University Cancer Institute and Hospital. The authors state that they have obtained verbal and written informed consent from the patient for the inclusion of their medical and treatment history within this case report.

This work was supported by the Tianjin Science and Technology Commission (18ZXXYSY00070), Key Task Project of Tianjin Health and Family Planning Commission (16KG128), Anticancer Key Technologies R&D Program of Tianjin (12ZCDZSY16200), and Natural Science Foundation of Tianjin (18JCYBJC91600).

The authors declare no potential conflicts of interest with respect to the research, authorship, and/or publication of this article.

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24. Kiyota N, Hasegawa Y, Takahashi S, et al. A randomized, open-label, Phase III clinical trial of nivolumab vs. therapy of investigators choice in recurrent squamous cell carcinoma of the head and neck: a subanalysis of Asian patients versus the global population in checkmate 141. Oral Oncol. 2017;73:138146. doi:10.1016/j.oraloncology.2017.07.023

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[Full text] Successful Use of Nivolumab in a Patient with Head and Neck Cancer Aft | OTT - Dove Medical Press

Skin Stem Cells Comments Off on [Full text] Successful Use of Nivolumab in a Patient with Head and Neck Cancer Aft | OTT – Dove Medical Press | February 10th, 2021

Cultures with treated stem cells had a 50% higher stem cell survival rate than untreated cultures. Image by carolem41

Treating equine donor stem cells with a growth factor called TGF-2 may allow them to avoid tripping the immune response in recipients, according to new research.

The work carried out at North Carolina State University could simplify the stem cell treatment process for ligament and tendon injuries in horses, and may also have implications for human stem cell therapies.

Mesenchymal stem cell therapy is a promising avenue for treating musculoskeletal injuries, particularly tendon and ligament injuries, in horses.

Mesenchymal stem cells are adult stem cells found in bone marrow that act as repair directors, producing secretions that recruit healing-related paracrine factors to the site of injury.

Just as blood cells have types, depending upon which antigens are on the blood cells surface, mesenchymal stem cells have differing sets of major histocompatibility complex molecules, or MHCs, on their surfaces.

If the MHCs of donor and recipient arent a match, the donors stem cells cause an immune response. In organ transplants, MHCs are carefully matched to prevent rejection.

These treatments arent like a bone marrow transplant or an organ transplant, says Lauren Schnabel, associate professor of equine orthopedic surgery at the university and corresponding author of the study, reported in the journal Frontiers in Cell and Developmental Biology.

Since the mesenchymal stem cells are being used temporarily to treat localized injury, researchers once thought that they didnt need to be matched that they wouldnt cause an immune response. Unfortunately, that isnt the case.

Schnabel and Alix Berglund, a research scholar at the university and lead author of the paper, wanted to find a way to use mesenchymal stem cell therapy without the time, effort and additional cost of donor/recipient matching.

Since these cells dont have to be in the body as long as an organ does, hiding them from the immune system long enough for them to secrete their paracrine factors could be a way around donor/recipient matching, Berglund says. Downregulating expression of the MHC molecules could be one way to do this.

The researchers cultured stem cells and lymphocytes, or T cells, from eight horses, cross-pairing them in vitro so that the stem cells and lymphocytes had differing MHC haplotypes.

In one group, stem cells had been treated with transforming growth factor beta (TGF-2) prior to being added to the lymphocytes in the culture media; the other group was untreated. TGF-2 is a cell-signaling molecule produced by white blood cells that blocks immune responses.

Cultures with treated stem cells had a 50% higher stem cell survival rate than untreated cultures.

We use mesenchymal stem cells to treat musculoskeletal injuries particularly tendon injuries in horses very effectively, Schnabel says.

And while you can extract the secretions from the stem cells, you get better results with the cells themselves. Stem cells arent just a reservoir of secretions, theyre a communications hub that tells other cells what they should be doing. So finding a way to utilize these cells without stimulating immune response gives us better treatment options.

This is a promising pilot study, Berglund says. Our next steps will be to further explore the immune response in vivo, and to look at human cells in vitro, as this work has excellent potential to help humans with these injuries as well.

The research was supported by the National Institutes of Health and the Morris Animal Foundation. Research specialist Julie Long and statistician James Robertson, both with the university, also contributed to the work.

TGF-b2 Reduces the Cell-Mediated Immunogenicity of Equine MHC-Mismatched Bone Marrow-Derived Mesenchymal Stem Cells Without Altering Immunomodulatory PropertiesAlix K. Berglund, Julie M. Long, James B. Robertson, Lauren V. SchnabelCell Dev. Biol., 04 February 2021 https://doi.org/10.3389/fcell.2021.628382

The study, published under a Creative Commons License, can be read here.

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Researchers curb local immune response in horses receiving stem cell injury therapy - Horsetalk

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Cord blood collection process is the easiest and safest for both mother and the baby in normal and C-Section delivery. Cord blood is collected after the baby is born and the umbilical cord is clamped and cut. Read here to know more.

A baby's umbilical cord contains blood-forming stem cells which can rebuild the immune system

As a parent to be, there are multiple things on your mind to consider and plan before the little one arrives. Between scan appointments and packing hospital bags, you certainly are on the lookout for the best for your baby. Every mother is always anxious to give birth to a healthy baby and assure that it has a healthy future. When you want to give it a healthy future, you need to think about life saving benefits. One of the most important decision which is made at birth for the baby and its family is preserving its cord blood from umbilical cord. The umbilical cord, which connects the baby and the mother in womb has life-saving benefits.

After the birth of the baby, the blood left in the umbilical cord has life-saving cells that can potentially treat over 80 medical conditions pertaining to blood disorders. Cord Blood Banking is the procedure of safely collecting blood from umbilical cord and placenta and preserving it in a sterile environment, thereby ensuring access to stem cells for one's lifetime.

The stem cells which is preserved can be used for blood related disorders. In simple words, stem cells can act like the body's own repair kit and help the body heal from life threatening diseases.

With several advancements, community cord-blood banking is a frontier in medical practice that will help secure not just your baby's future but also your extended family's health. Despite its benefits, there remains many common misconceptions about cord blood banking. Dr Anjali Kumar, Director, Obstetrics and Gynecology, C K Birla Hospital, Gurgaon debunks the myths and facts about Cord Blood banking.

Cord blood can secure baby's future and also your extended family's healthPhoto Credit: iStock

Also read:Expert Reveals Common Skin Problems In Newborn Babies And How To Take Care Of Them

Fact: Cord blood collection process is the easiest and safest for both mother and the baby in normal and C-Section delivery. Cord blood is collected after the baby is born and the umbilical cord is clamped and cut. The cord blood that is collected is blood that would normally be discarded after birth, so collection doesn't affect your baby's blood supply during pregnancy or delivery.

Fact: Cord Blood extracted from baby's umbilical cord is rich and can be easily available for the family and the child if preserved at birth. Incase of bone marrow or other source of stem cells, the donor has to be registered and should be a match to the family. Cord blood may be accessed more quickly than stem cells from an adult donor who may have registered for donation years ago. The donor must be located, consented, tested and harvested. The extraction of stem cells through Bone Marrow can be painful as well.

Also read:Newborn Care During Winter Season: Here's A Complete Guide For Parents

Fact: According to The Indian Council of Medical Research (ICMR) guidelines commercial banking of Cord Tissue is not permitted to be collected and preserved.

Fact: A baby's umbilical cord contains blood-forming stem cells which be transplanted and rebuild the immune system and bone marrow by saving a patient life with a certain life-threatening condition such as leukemia, lymphoma or thalassemia. Infusion of these cells can also treat patients with inherited genetic disorders, bone marrow failure or inherited immune deficiency.

Fact: It is difficult to find a matching donor of Indian origin of the times hence its important to bank baby's umbilical cord. Incase even if a match is found, it can also be incredibly costly to obtain donor stem cells. The cost to obtain a unit of stem cells can be anywhere between 15-20 lakhs and upwards.

Also read:What Is The Best Time To Give Water To Your Newborn?

(Dr Anjali Kumar, Director, Obstetrics and Gynecology, C K Birla Hospital, Gurgaon)

Disclaimer: The opinions expressed within this article are the personal opinions of the author. NDTV is not responsible for the accuracy, completeness, suitability, or validity of any information on this article. All information is provided on an as-is basis. The information, facts or opinions appearing in the article do not reflect the views of NDTV and NDTV does not assume any responsibility or liability for the same.

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Cord Blood Banking: Myths And Facts You Should Be Aware Of, As Per Expert - NDTV Doctor

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Three years ago, wading in the sun-warmed waters of the Florida Keys, I felt a sharp pinch and looked down at my feet in surprise. My friend Jen and I had driven down from Miami for a weekend full of strong Cuban coffee and Hemingways six-toed cats. Tempted by water so warm and aquamarine it was almost a cliche, we had stopped to swim at a roadside beach on Bahia Honda Key. I had eased in, careful to drag my feet slowly across the seafloor in a dance known to beachgoers as the stingray shuffle, hoping to alert any local sealife to my approach. But not careful enough.

My foot throbbing, I stumbled back to the beach toward Jen, who wondered if I might have stepped on some glass. But in the next half hour, as my ankle and foot ballooned and the pain ratcheted upfrom stinging to aching, from aching to bone breakingit became clear I must have been stung by something. Then my foot started to turn blue, and we drove to the hospital.

Bahia Honda? the nurse said. Youre the fourth person to come in with a stingray sting from there today.

The pain didnt subside until the next day, when my foot had returned to its normal color. That was the start of a six-week recovery, which also involved crutches, painkillers, heavy-duty antibiotics, and a horrible rash. I wouldnt wish the experiencewhich involves a level of discomfort that some have compared to a gunshot woundon anyone. But in retrospect, its an interesting one to consider. Because, it turns out, animal venoms like the one coursing through my veins on Bahia Honda Key are sought after for drug development, with seven FDA-approved drugs derived from venom toxins on the market so far. Harnessing their power to hurt opens up a world of possibilities for healing.

The Bahia Honda beach where the author had a run-in with a stingray. Image Credit: Giuseppe Milo, Flickr (CC BY 2.0)

Chemical biologist Mand Holford, who studies venom science at her lab at Hunter College, compares what was happening in my foot in the moment after the sting to a cluster bomb. The toxins in animal venom have been engineered by evolution over many millennia to incapacitate by affecting some component in the blood, brain, or cell membranes, she says. Youre getting invaded with 200 to 300 different toxins, all trying to figure out how to reach their target, moving through and rupturing cell membranes, doing all sorts of damage.

The nurse at the emergency room told me stingrays were migrating through the area, their path bringing them close to the cove where I went wading. Stingrays deliver their venom through one or more serrated barbs that lie along their tails. While at rest, a stingray keeps its barb tucked away, immunologist Carla Lima told me in an email. But when it feels threatenedsay, by the feet of a clueless human out for a swimit pushes its tail perpendicular to its body, puncturing that humans flesh with its venom-laden spine.

Lima studies toxins in venomous fish at the Butantan Institute in So Paolo, Brazil. Her research into stingray venom has shown that whats in that venom actually changes as a stingray matures. In the freshwater species she studieswhose venom properties are better explored than the marine stingray that got methe venom of young rays tends to contain toxins that cause pain to the target. Lima hypothesizes this may be to chase predators away. In contrast, the toxins in adult venom have a necrotizing effect, meaning they destroy tissue, which would be helpful for hunting.

Peptides, short chains of amino acids that play key roles in the biological functions of all kinds of organisms, make up a large part of most animal venomsand some are only found in those venoms. Lima and other researchers have identified the peptides porflan and orpotrin as two of the elements in the freshwater stingrays toxic cocktail, along with a number of different proteases, which are enzymes that break down peptides.

As I sat cradling my foot on the beach in Bahia Honda, similar proteases and related proteins worked to break down the structure of cells in my heel, helping the venom spread further, and to prompt an inflammatory reaction that led to the swelling I saw. The peptides, on the other hand, likely caused the arteries to constrict and blood to pool, creating more inflammation and blocking circulationperhaps the cause of my foot turning blue.

A southern stingray (Dasyatis americana) cruises the ocean floor off Grand Turk Island in the Caribbean. Image Credit: Nate Madden, Shutterstock

That a substance that causes so much pain and wreaks so much biological havoc can be used in medicine is what Holford calls the yin and yang of nature. And the fact that damage and healing are, at least in this case, two sides of the same coin forms the basis for the work she does in her lab, identifying new drug applications for various components of animal venom.

Venoms have great potential to contribute to drug development because they are both potent and highly targeted, Holford says, with peptides that fit physically into cell receptors and change how those cells function. Thanks to this dynamic, venom-based drugs can work almost instantaneously. And theyre not what people in the pharmaceutical business call leaky, meaning they tend to only act on the intended cell component and dont stop at other spots along the way causing side effects.

Most stingray venom research, like Limas, takes place in areas where stingrays pose a threat to people: tropical spots like Brazil and Australia. On a drug-development level, we still dont know much about it, Lima says. But we do know a lot about other venomsin particular those created by cone snails and snakes.

For one thing, not all venom toxins cause pain. Some peptides present in snake venom focus on manipulating proteins in the wound so blood flows freely, acting as natural anticoagulants. Other peptides in Gila monster venom promote insulin production, helpful for a hungry lizard that hasnt eaten for awhile. And yet other peptides in cone-snail venom do the opposite of what stingray venom does: paralyze and suppress pain, keeping the snails prey from going into fight-or-flight mode and slowing it down until the (also slow) snail can come nab it for a snack.

This last type of venom is one of the focuses at Holfords lab. Many cone-snail venom peptides are rich in cysteine amino acids, whose structure she compares to Velcro. That makes it relatively easy for them to stick in the hourglass-shaped pores on the surface of cells that let important minerals like sodium, calcium, and potassium flow in and out. The free movement of those minerals is part of how cells talk to each other.

With those channels shut down, neurons cant communicate with one another to indicate pain. Thats what makes Prialt, the commercial version of the cone snails ziconotide peptide, an effective pain medication. Holford and her colleagues are also exploring the potential of other related cone-snail peptides to help dampen signals firing too fast in someone having a heart attack or an epileptic seizure.

She even sees possible applications here for cancer treatment. Current chemotherapy regimens dont discriminate between normal cells and tumor, she says. But because venom peptides work on specific receptorsreceptors that some tumors grow too many of as part of their developmentthey could help create a cancer drug that specifically starves cancer cells of essential minerals, stopping their growth.

The saw-scaled viper (Echis carinatus) is one of the deadliest snakes in India, and its venom is the basis of the blood-thinning drug Tirofiban. Image Credit: Sagar Khunte, Wikimedia Commons (CC BY-SA 4.0)

The venom that nearly ruined my Florida Keys vacation (though I still got to enjoy some beautiful sunsets, and the seafood was fantastic) was incredibly sophisticated, honed by evolution to inflict pain and physiological damage with laser precision. It was almost comforting to learn this in the weeks after, as I hobbled around on my crutches and watched with fascinated disgust as the wound developed a stingray-shaped blister. (My boyfriend said it was a sign I was developing superpowers, but sad to say none appeared.)

We know from nature that these peptides work, Holford says. What we dont know is massive: where they work, how they work, how effective they are. And thats a huge game of Wheres Waldo. Holford and her colleagues have come up with a protocol for finding new venom components that have potential in drug applications, then figuring out how to get them there. The first step is a practical look at the natural world: identifying which animal species are creating venom, especially venom that can be extracted manually. Next, the team uses new technologies that Holford refers to as the omics genomics, transcriptomics, proteomicsto identify the toxins within those venoms, by examining the instructions the animals' DNA and RNA contain and the proteins built by following those instructions.

From there, the team is able to use that genetic code to manufacture more of a chosen peptide in the lab, which is especially useful when it comes to studying venoms that are produced in small quantities in nature. They then test the synthetic toxin on the animals natural prey to make sure its effective and further tweak it to ensure its as specifically targeted as it can be for humans. And finally, they start to think about drug delivery. Does this drug need to cross the blood-brain barrier? Would it work if administered orally? These are essential questions, since potential drugs that cant be delivered effectively cant really be drugs at all.

Much like the experience of the sting itself, the possibilities for new drugs here are dizzying. Most venom-based drugs on the market are derived from a single peptide. But my stingrays venom (just like other naturally occurring venoms) featured hundreds of peptides. And with the advent of the omics, drug development with venom has become more efficient. Time- and resource-intensive experiments can now be run much more quickly using computer modeling, making the whole process more viable and opening up a whole world of drug prospects.

Lima and her colleagues in Brazil, for example, are continuing to explore the realm of fish venom. One synthetic peptide derived from the venom of a species of toadfish shows particular promise. A 2017 study suggested that peptide, known as TnP, has powerful anti-inflammatory and therapeutic effects in mice. Effects that could potentially help stem the autoimmune reactions that lead to spinal cord damage in patients with multiple sclerosis.

As Holford and her team navigate the new technological landscape, theyre also looking for ways to simplify their process. One innovation Holford is excited about is organoids, in this case, venom glands grown independently in a laboratory. Growing organoids would make acquiring venom samples much easier, she says, and would not require sacrificing an animal for the initial sample.

Thats especially important with climate change and habitat loss fueling a looming biodiversity collapse that could take with it undiscovered venoms with the capacity to heal. In 10 years were heading toward this major shift thats coming if we dont change our attitudes and lifestyle, she says. We could lose a lot of things on the planet that are potentially lifesaving.

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I got stung by a stingray, and all I got was this deeper understanding of venom medicine - NOVA Next

Spinal Cord Stem Cells Comments Off on I got stung by a stingray, and all I got was this deeper understanding of venom medicine – NOVA Next | February 6th, 2021

Every year, about 10,000 people in the U.S. need a stem cell transplant but cant find a donor.

The intense medical procedure, which can help those with leukemia, lymphoma, sickle cell anemia and other blood diseases, can save lives but securing a donor can be like finding a needle in a haystack.

Be The Match is a nonprofit, national registry where people can sign up to donate their stem cells. More than 35 million people around the world have volunteered yet only a small percentage of those donors are Americans, and even the registry admits most Americans dont know it exists.

The mother of a 12-year-old boy with leukemia has set out to change that.

Mandy Goldman is a hairdresser who lives with her husband and four children outside Boston. She remembers the devastating day five years ago when doctors told her the chemotherapy they gave her son Mateo Goldman, 9 years old at the time, didnt work.

They told us that our only option of curing Mateo was a bone marrow transplant, she says, a risky procedure that often involves a host of complications. But they had no other choice, she says.

The family got to work on the monumental task finding Mateo Goldman a close enough match.

Linda Matchan first reported the Goldman familys experience for The Boston Globe. In her research, she found very, very few people had any awareness of the need for bone marrow and stem cells donors. The awareness campaign around the subject is severely lacking compared to other campaigns like the importance of donating blood, she says.

For example, there's a little boy right now in North Carolina named Thor Forte, who's 10 and has sickle cell disease. And he has been waiting for literally half his life, five years, for a donor to be available, Matchan says. He's a tough match, but they finally did find somebody. And then when the time came for the procedure, the person backed out. So two years later, the boy is still waiting.

Fortunately, quickly after finding out Mateo Goldman didnt match with anyone in his family, he was paired with a donor on the registry from Germany. Mandy Goldman says Laura Stterlin of Frankfurt was ready to go and donate, ultimately saving her son.

Mateo Goldman wrote Stterlin, whose name he did not know at the time, a thank you note reading: Dear Donor, thank you for giving me the bone marrow. You feel like youre already part of my family, he says.

And unlike usual Make-A-Wish requests, Mateo Goldman asked to meet Stterlin in person halfway across the world. The trip to Germany was planned for summer of 2020 but has since been canceled due to the pandemic.

In 2019 when she was reporting this story, Matchan had a trip planned to Germany. She ended up meeting Stterlin and hearing the story of how she became a donor. Stterlin said she was at a sporting event with her husband when she got hungry and went on the hunt for some grub.

Dear Donor, thank you for giving me the bone marrow. You feel like youre already part of my family.

Germany has a robust public service campaign to get citizens to donate bone marrow, Matchan says. So it came to no surprise to Stterlin when she came across a kiosk to sign up.

Just three months later, she got a call and an email from the registry saying that there is somebody in the United States for whom she could be a match and was asked if she would donate, Matchan says. A couple of days later, she went into the hospital and did the donation.

Stterlins stem cells then crossed the Atlantic Ocean, making their way to America during a snowstorm.

The cells started working in Mateo Goldman right away but not without some difficulties, Mandy Goldman says. He battled total body stiffness from graft-versus-host disease, a complication of the transplant.

But, you know, Matteo's an amazing kid, she says, so through it all, he was smiling and making the best of it, even though he was suffering for a lot of the time.

Two years later, in July of 2020, the cancer came back. But since Mateo Goldmans first transplant, the science had evolved greatly.

So much so that his older brother, Leo Goldman, became a candidate to donate his cells for the second stem cell transplant.

I didn't realize how I could get my brother's cells, Mateo Goldman, now 12 years old, says. Once that sank in, I felt that it would connect me and my brother more.

Right before Christmas last year, the family got extraordinary news: Mateo Goldman had zero cancer in his bone marrow, Mandy Goldman says.

Now the mom of four is on a mission to raise awareness on stem cell donations and share the story of how it saved her sons life.

The amazing feeling Leo got from being able to be the person who saved his brother's life is something he's going to carry with him forever, she says. And even Laura [Stterlin], she gave him three and a half years of his life that we get to spend with him. I just really want to educate people about how empowering it is to do something so incredible for somebody else.

When she started talking to others to raise awareness, she was shocked to discover how fearful people were in committing to be a donor.

If people could see the trauma these patients go through her son had a drain placed in his stomach, total body radiation, chemotherapy that left him head-to-toe in a skin-burning rash she says then maybe they wouldnt be scared to dedicate a small action for someone whose only cure is through a stem cell transplant.

Once people are educated about how much of a difference it makes, she says, then I feel like they would do it.

Click here to learn more about the Be The Match Registry.

Tinku Rayproduced and edited this interview for broadcast.Serena McMahonadapted it for the web.

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After Bone Marrow Donation Saves 9-Year-Old Boy With Cancer, Boston Mom Fights To Raise Awareness - Here And Now

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Leukemia is a type of cancer that affects the blood. The two most common types in children are acute lymphoblastic leukemia and acute myelogenous leukemia.

In a person with leukemia, blood cells are released into the bloodstream before they are fully formed, so there are fewer healthy blood cells in the body.

Below, we describe the types of childhood leukemia, the symptoms, and the treatments. We then look at when to contact a doctor, what questions to ask, and where to find support.

Childhood leukemia is the most common form of cancer in children. It affects up to 3,800 children under the age of 15 in the United States each year.

Leukemia occurs when bone marrow releases new blood cells into the bloodstream before they are fully mature.

These immature blood cells do not function as they should, and eventually, the number of immature cells overtakes the number of healthy ones.

Leukemia can affect red and white blood cells and platelets.

The bone marrow produces stem cells. A blood stem cell can become a myeloid stem cell or a lymphoid stem cell.

Lymphoid stem cells become white blood cells. Myeloid stem cells can become:

Leukemia is typically acute or chronic, and chronic types are rare in children. They can include chronic myeloid leukemia or chronic lymphocytic leukemia.

Most childhood leukemias are acute, meaning that they progress quickly and need treatment as soon as possible.

Acute lymphoblastic leukemia (ALL) is the most common type in children, accounting for 75% of childhood leukemia cases.

It affects cells called lymphocytes, a type of white blood cell.

In a person with ALL, the bone marrow releases a large number of underdeveloped white blood cells called blast cells. As the number of these increases, the number of red blood cells and platelets decreases.

There are two subtypes of ALL: B-cell and T-cell.

In most childhood cases of ALL, the cancer develops in the early forms of B-cells. The other type, T-cell ALL, typically affects older children.

Research from 2020 reports that the majority of people diagnosed with ALL are under 18 and typically between 2 and 10 years old.

The American Cancer Society report that children under 5 years old have the highest risk of developing ALL and that this risk slowly declines until a person reaches their mid-20s.

The outlook for ALL depends on the subtype, the persons age, and factors specific to each person.

Myeloid leukemias account for approximately 20% of childhood leukemia cases, and most myeloid leukemias are acute.

Acute myelogenous leukemia (AML) affects white blood cells other than the lymphocytes. It may also affect red blood cells and platelets.

AML can begin in:

Juvenile myelomonocytic leukemia (JMML) accounts for approximately 12% of leukemia cases in children.

This rare type is neither acute nor chronic. JMML begins in the myeloid cells, and it typically affects children younger than 2 years.

Symptoms can include:

The symptoms of leukemia may be nonspecific similar to those of other common childhood illnesses.

A doctor will ask how long the child has been experiencing the symptoms, which can include:

Children may experience specific symptoms depending on the type of blood cell that the leukemia is affecting.

A low number of red blood cells can cause:

A low number of healthy white blood cells can cause infections or a fever with no other sign of an infection.

A low platelet count can cause:

Various factors can increase a childs risk of leukemia, and most are not preventable.

The following genetic conditions can increase the risk of leukemia:

Also, having a sibling with leukemia may increase the risk of developing it.

These can include exposure to:

If a child has symptoms that might indicate leukemia, a doctor may perform or request:

A bone marrow aspiration involves using a syringe to take a liquid sample of bone marrow cells. The doctor may give the child a drug that allows them to sleep through this test.

During the diagnostic process, a person might ask:

The doctor may recommend a variety of treatments for childhood leukemia, and the best option depends on a range of factors specific to each person.

The treatment usually consists of two phases. The first aims to kill the leukemia cells in the childs bone marrow, and the second aims to prevent the cancer from coming back.

The child may need:

Before or during treatment, a person might ask the doctor:

Questions to ask after the treatment might include:

Children who have undergone leukemia treatments require follow-up care, as the treatments often cause late effects.

These can develop in anyone who has received treatment for cancer, and they may not arise for months or years after the treatment has ended.

Treatments that can cause late effects include:

These complications may affect:

The late effects that may come can also depend on the type of treatment and the form of leukemia.

Because many leukemia symptoms can also indicate other issues, it can be hard to know when to contact a doctor.

Overall, it is best to seek medical advice if a child shows symptoms or behaviors that are not normal for them.

If a child has received a leukemia diagnosis, the effects can extend to parents, other family members, caregivers, and friends.

A person can find support and additional resources from:

The following organizations based in the United Kingdom also provide support and guidance:

Childhood leukemia can affect mental health, as well as physical health.

Learn more about mental health resources here.

According to the American Cancer Society, most children with leukemia have no known risk factors. There is no way to prevent leukemia from developing.

Because there are very few lifestyle-related or environmental causes of childhood leukemia, it is very unlikely that a caregiver can do anything to help prevent the disease.

A childs outlook depends on the type of leukemia. It is important to keep in mind that current estimates do not take into account recent advances in technology and medicine.

For example, the most recent 5-year survival rate estimates reflect the experiences of children who received their diagnoses and treatments more than 5 years ago.

The American Cancer Society report that the 5-year survival rate for children with ALL is 90%. The same rate for children with AML is 6570%.

Childhood leukemia is typically acute, which means that it develops quickly. As a result, a person should contact a doctor if they notice any of the symptoms.

The most common type of childhood leukemia is ALL, representing 3 out of 4 leukemia cases in children.

Treatment may include a combination of chemotherapy, targeted drugs, immunotherapy, stem cell transplants, surgery, and radiation.

The prognosis depends on the type of leukemia and the childs age.

This diagnosis can affect mental as well as physical health, and the effects can extend to caregivers, family members, and friends. Many different resources are available for support.

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Leukemia in children: Symptoms, causes, treatment, outlook, and more - Medical News Today

Bone Marrow Stem Cells Comments Off on Leukemia in children: Symptoms, causes, treatment, outlook, and more – Medical News Today | February 6th, 2021

Following stem cell transplant or treatment with CAR T-cell therapies, patients with hematologic malignancies and coronavirus disease 2019 (COVID-19) tend to have favorable outcomes, especially if they are diagnosed in complete remission (CR) and further out from their cell infusion, according to Miguel-Angel Perales, MD, underscoring that care should not be delayed despite the ongoing pandemic.

Delayed therapy results in patients with relapse or progression of disease who did not receive the intended cellular therapy; [weve seen this happen] in 34% of cases, Perales, chief of the Adult Bone Marrow Transplant Service at Memorial Sloan Kettering Cancer Center (MSKCC), said during a presentation delivered at the 2021 AACR Virtual Meeting on COVID-19 and Cancer.1 Given that we can avoid the risk of nosocomial transmission, I think this clearly indicates that we should be careful about how we manage these patients and not try to delay their care.

In his talk, Perales highlighted registry data detailing the impact of the pandemic on cellular treatment in patients with cancer, outcomes of patients who were infected with the virus and received hematopoietic cell transplantation, and the impact of virus-related delays in care.

Data reported to the ASH Research Collaborative COVID-19 Registry for Hematology, a global reference tool available to the public, showed that as of January 15, 2021, a total of 813 malignant and non-malignant cases of COVID-19 were reported, with just over 500 cases reported in the United States alone.2

When looking at cellular therapies received prior to a diagnosis with the virus, 10 patients had received CAR T-cell therapies (6 recovered, 4 died), 46 patients had undergone allogeneic stem cell transplantation (34 recovered, 7 died, 5 had unknown outcome), and the majority, or 78 patients, had undergone autologous stem cell transplantation (67 recovered, 7 died, 4 had unknown outcome).

An earlier analysis of data collected from this registry showed that among the first 250 patients for whom data were collected, the overall mortality rate was 28% (95% CI, 23%-34%).3 However, in patients with moderate to severe COVID-19 infection, the mortality rate was even higher, at 42% (95% CI, 34%-50%). This is a condition that has significantly impacted our patients with hematologic malignancies, noted Perales.

Another registry, of the Center for International Blood & Marrow Transplant Research (CIBMTR), requires the inclusion of outcomes of patients who have undergone transplantation or received CAR T cells.4 As of January 15, 2021, data for 1258 patients from 195 centers were reported to the registry and showed that 50.08% of patients had undergone allogeneic transplantation and 44.66% had undergone autologous transplantation. Only a small percentage of patients received cell therapy, according to Perales.

The age of patients at the time of infection ranged from less than 20 years to older than 70 years, with the majority of patients between the ages of 60 years and 69 years. When looking at infections by region, 29.35% of cases were reported in the Midwest, 23.44% were reported in the Northeast, and 22.73% were reported in the South. The majority of cases occurred within the first 2 years of their infusion. A total of 614 casesalmost half of all patientshad their infection resolve, while 58 experienced improvement; 187 patients had died.

In a subsequent paper, investigators examined risk factors associated with death from COVID-19 in recipients of allogeneic transplantation based on data from the CIBTR registry.5 Results from the multivariate analysis showed that age greater than 50 years (P = .016), male gender (P = .006), and COVID-19 infection in less than 12 months following transplantation (P = .019) were all significantly associated with increased risk of death.

Interestingly, race and ethnicity were not significant in this series, noted Perales. Similarly, when we look at patients [who have undergone] autologous transplant, the only factor that we saw was the diagnosis of lymphoma versus myeloma. Other factors were not significant.

In another analysis, investigators examined outcomes of patients following transplant who were infected with the virus at MSKCC. Of the first 77 patients diagnosed between March 15, 2020 and May 7, 2020, 37 had undergone autologous transplant, 35 had undergone allogeneic transplant, and 5 had received CAR T-cell therapy.6

The disease distribution was as expected, according to Perales. Thirty-eight percent of patients had plasma cell disease, 23% had acute leukemia, 23% had aggressive non-Hodgkin lymphoma (NHL), 5% had Hodgkin lymphoma, 4% had chronic myeloid leukemia, 4% had myelodysplastic syndrome, and 3% had indolent NHL.

When you look at day [of infection] post infusion, you see there was a significant range, said Perales. In fact, the number of patients were diagnosed with COVID-19 several months or even years after their cell therapy. These are the demographics of 77 patients, but this is representative of the patients that we transplant at our center.

Notably, 44% of patients did not have any comorbidities. Investigators also examined the home medications that patients were receiving at the time of their COVID-19 diagnosis. Here, 10 patients were receiving steroids, 18 were receiving immunomodulatory agents, 4 were receiving anticoagulation agents, and 14 were receiving immunosuppressive drugs.

Almost half, or 48%, of patients had mild COVID-19 infection, so they were not admitted to the hospital. Twenty-six percent of patients had moderate infection, and thus, were admitted to the hospital, while 22% had severe infection and were either admitted to the intensive care unit or died.

In that group, the majority of them actually had active malignancy, unlike the other 2 groups where the majority actually were in remission, said Perales. Patients who required high levels of oxygen [were often those who] had active malignancy.

Results from a univariate analysis looking at the predictors of disease severity revealed significant associations between the presence of comorbidities and infiltrates on imaging at the time of diagnosis. Overall, however, we were able to see favorable outcomes with patients after COVID-19 infection, said Perales. Two-thirds of patients actually had a resolution. We did see 14 deaths, which represented 18% of patients. This was 41% of patients who were admitted, but particularly those with an active malignancy.

Among patients who were admitted to the hospital but had a malignancy that was in remission, the mortality rate was 21%. This was due, in part, to the fact that in many cases, patients or their family members decided to forego aggressive medical care.

Additional data revealed that COVID-19 was linked with a drop in lymphocyte populations across the board, added Perales. Notably, lymphopenia with COVID-19 was not found to impair long-term immune reconstitution in patients who had undergone bone marrow transplant.

When looking at survival in patients after infection with COVID-19, overall outcomes were found to be favorable.

Investigators also examined the risk of nosocomial infections in patients who had undergone transplantation or received cellular treatment in light of the pandemic. They looked at a series of 44 cases.

In March 2020, 2 healthcare workers were exposed at MSKCC and 3 patients had documented COVID-19 infection. One patient was receiving treatment in the inpatient setting, but the patient did have frequent visits from family members, according to Perales. So, its unclear when or how the exposure occurred, Perales said. The patient ended up dying.

Two additional patients may have been exposed in the donor room while they were collecting the stem cell from the autologous transplant, added Perales. One patient eventually died from the virus.

Again, its unclear whether these patients were infected in the center or in the community, as COVID-19 was very prevalent at the time, said Perales. Importantly, we have not seen any additional cases of potential or definite COVID-19 nosocomial infection since March 2020 at our center.

When examining the impact of the pandemic on treatment delays, in March 2020, investigators started to prospectively collect data from patients whose transplant or cellular therapy was delayed as a result of the impact of the virus on resources at the hospital, particularly the capability of using intensive care unit beds.1

Results showed that 85 patients delayed treatment; of those patients, 29 have not received their intended cellular treatment. Sixteen were supposed to receive autologous transplant, 12 were supposed to undergo allogeneic transplant, and 1 was supposed to receive CAR T-cell therapy.

Of the 56 patients who eventually proceeded to treatment, 62% received autologous transplant, 67% received allogeneic transplant, and 86% received CAR T-cell therapy. The biggest reason for not proceeding to treatment with autologous transplant and CAR T-cell therapy was because they were deferred due to good disease control. Other reasons included was because of a new comorbidity (12%) or they died from the virus. The most prominent reason for not proceeding to allogeneic transplant during the pandemic was progression of disease (42%).

We conclude that patients who are recipients of allogeneic transplant, and particularly those with acute leukemia, as much as possible should proceed to their indicated therapy and not be delayed, concluded Perales.

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Perales Examines the Impact of COVID-19 on Recipients of Cellular Therapies for Cancer - OncLive

Bone Marrow Stem Cells Comments Off on Perales Examines the Impact of COVID-19 on Recipients of Cellular Therapies for Cancer – OncLive | February 6th, 2021

Image for representation purpose only.

A tech entrepreneur, who is determined to live for 180 years, claims his bizarre strategies will soon be as popular as cell phones. Dave Asprey firmly believes one of the keys to living longer is skipping breakfast. The American lifestyle guru has asserted that he can at least live to the year 2153 by using a variety of techniques, including a cold cryotherapy chamber sitting and intermittent-fasting. The 47-year-old millionaire devised the term 'Biohacking' to detail his methods of turning back the biological clock. In an effort to survive for as long, Dave has spent over $1,000,000 on hacks and techniques to improve the overall functioning of his body. He has parts of his bone marrow removed to re-inject his own stem cells back into his body.

Appearing on ITVs This Morning today, he joined host Holly Willoughby for a virtual conversation. Holly quizzed Dave why he wants to live so long, to which he replied that as a curious person, he feels there is a lot in the world that can be fixed and improved that he thinks he has not done yet. Dave confirmed that some of the things he wants to pioneer are expensive. However, there are other ways that are free of cost like fasting.

Dave also speculated that after applying his methods for longer life, people under 40 will be 'happy and highly functional' over a 100-years-old. He stated that he won't be the only one as his wife both is also racing to get to 180 years old. In comparison to others, Dave has set himself up for much less inflammation by controlling what he eats and how he sleeps, besides several other anti-ageing treatments. Explaining the benefits of reintroducing his own stem cells in his body, he said people heal when they are young. With age, the stem cells of the body get exhausted, so he opts for ways which gives him more stem cells.

Dave also follows cryotherapy, which is also known as cold therapy. It involves using low temperatures to treat a variety of tissue lesions. He is also taking cold showers for more than ten years. Dave uses another technique to live a long life known as intermittent fasting. This involves controlling the number of times that one eats meals to create periods of fasting over a certain period. According to Dave, it helps in disease prevention as fasting periods lets his body to 'repair itself' while not digesting food.

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US Man Who Wants to Live for 180 Years Re-injects His Own Stem Cells, Spends Rs 87 Lakh - News18

Bone Marrow Stem Cells Comments Off on US Man Who Wants to Live for 180 Years Re-injects His Own Stem Cells, Spends Rs 87 Lakh – News18 | February 6th, 2021

The reduced osteogenic differentiation of bone marrow mesenchymal stem cells (BMSCs) is the typical characteristics of pediatric aplastic anemia (AA) pathogenesis. Long non-coding RNA MEG3 is reported to promote osteogenic differentiation of BMSCs via inducing BMP4 expression.This study aims to investigate the mechanism of DNMT1/MEG3/BMP4 pathway in osteogenic differentiation of BMSCs in pediatric AA.BMSCs were isolated and purified from bone marrows of pediatric AA patients (n=5) and non-AA patients (n=5). The expression of DNMT1, MEG3, and BMP4 in isolated BMSCs was detected using quantitative real-time PCR and western blot analysis. Osteogenic differentiation was determined using Alizarin red staining. The methylation of MEG3 promoter and the interaction between DNMT1 and MEG3 promoter were detected using methylation-specific PCR and chromatin immunoprecipitation assay, respectively.Lowly expressed MEG3 and BMP4 and highly expressed DNMT1 were observed in BMSCs of pediatric AA patients. The overexpression of MEG3 promoted osteogenic differentiation of BMSCs. Luciferase reporter assay showed that MEG3 overexpression increased transcriptional activity of BMP4. The inhibitor of methylation, 5-azacytidine, suppressed DNMT1 expression and reduced methylation of MEG3 promoter. Overexpression of DNMT1 increased the binding between DNMT1 and MEG3 promoter. The simultaneous overexpression of DNMT1 and MEG3 restored the inhibition of osteogenic differentiation caused by DNMT1 overexpression alone.Our findings indicated that DNMT1 mediated the hypermethylation of MEG3 promoter in BMSCs, and DNMT1/MEG3/BMP4 pathway modulated osteogenic differentiation of BMSCs in pediatric AA.

PubMed

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Hypermethylation-mediated downregulation of long non-coding RNA MEG3 inhibits osteogenic differentiation of bone marrow mesenchymal stem cells and...

Bone Marrow Stem Cells Comments Off on Hypermethylation-mediated downregulation of long non-coding RNA MEG3 inhibits osteogenic differentiation of bone marrow mesenchymal stem cells and… | February 6th, 2021

The mum of a Stockton toddler says time is running out to find a match for her son who is battling a rare blood cancer.

Little Mason Arrowsmith, one, was diagnosed with Juvenile Myelomonocytic Leukaemia (JMML) on Christmas Eve last year.

The condition is a rare type of slowly developing blood cancer that occurs in young children.

Although it was discovered at an early stage, doctors say Mason needs a stem cell or bone marrow transplant for the best chance of survival.

In a desperate attempted to help, his mum Katie Jordan will donate her own bone marrow later this month, but a full match is urgently needed.

"The hospital has been absolutely amazing, but unfortunately after searching worldwide there is no match for Mason due to his bone marrow being so rare," Katie said.

"We need to get him to transplant as soon as, the hospital is looking at the end of February, using me as his first donor in the hope that this will help him until we find a better match.

"I would give my life for Mason but unfortunately, I can only donate my bone marrow three times in a lifetime and Im not a full match for him.

"Children with JMML live around 12 months after diagnosis, we need to find mason a better match. Twelve months is not long enough for us to have with our boy, we are living the worst possible nightmare."

As Mason does not have a close enough match within his family - the only potential cure is through a stem cell transplant from an unrelated donor.

The family has now launched a 'Masons Mission' to encourage people to support blood cancer charity Anthony Nolan in raising urgent funds to add more donors to the stem cells register.

There is currently a blacklog of around 25,000 potential donors due to the impact of the coronavirus pandemic and the charity needs to raise up to an extra 500,000 to add people to the register, from ordering more swab packs to analysing completed swabs in its laboratory.

Any one of the 25,000 people who have applied to join the Anthony Nolan could be a match for Mason or one of the 2,300 patients in the UK, who need a stem cell transplant from a donor each year.

Katie added: "We have set our target at 10,000 which does seem a lot but this would help to cover 250 registrations and kits and allow us to continue our search for my baby."

Henny Braund, Chief Executive of Anthony Nolan said: "Were doing all we can to find a stem cell donor to give Mason a second chance of life.

"A perfect storm of the coronavirus pandemic, and a surge of 40,000 incredible people who have been inspired to join the Anthony Nolan register in the last month by patients, like Mason means that were in urgent need.

"The best thing people can do is support Anthony Nolans work financially. By giving anything, together we can add all potential lifesavers to the register, and give patients like Mason hope."

Anthony Nolan recruits people aged 16-30 to the stem cell register as research has shown younger people are more likely to be chosen to donate.

They also carry out ground-breaking research to save more lives and provide information and support to patients after a stem cell transplant, through its clinical nurse specialists and psychologists, who help guide patients through their recovery.

It costs 40 to recruit each potential donor to the register, so Anthony Nolan relies on financial support.

You can support Masons Mission here.

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Family 'living worst nightmare' as they desperately seek donor for tot with rare blood cancer - Teesside Live

Bone Marrow Stem Cells Comments Off on Family ‘living worst nightmare’ as they desperately seek donor for tot with rare blood cancer – Teesside Live | February 6th, 2021

Researchers at the Catholic University of Korea St. Marys Hospital have recently proved the efficacy of bone marrow-derived stem cells to treat ototoxicity hearing loss, the hospital said Thursday.

The team, led by Professor Park Kyoung-ho of the Department of Otolaryngology, conducted an experiment on animal models with ototoxic sensorineural hearing, or sudden hearing loss.

They utilized Catholic MASTER cells, bone marrow stem cells developed by the Catholic Institute of Cell Therapy, to compare the stem cell injection group with the controlled group.

The result showed that animals started to recover their hearing after three weeks. Five weeks later, they recovered normal hearing at 8000Hz, 16000Hz and 32000Hz frequency.

Ototoxic hearing loss is caused when a person ingests chemicals or certain medications that adversely affect the inner ear functions. Major symptoms related to the illness are dizziness, false hearing, and hearing loss, which permanently defects hearing functions. Elders with such symptoms should have medical consultations as they are a high-risk group, the hospital said.

We have proved the efficacy of our bone marrow stem cells in recovering hearing, said Professor Park, who doubles as the director of the Stem Cell Institute. Through the results, we expect to provide new treatment opportunities for patients with hearing loss.

The test results were published in the Korean Journal of Otorhinolaryngology-Head and Neck Surgery.

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Stem cells efficacy confirmed in treating ototoxic hearing loss - Korea Biomedical Review

Bone Marrow Stem Cells Comments Off on Stem cells efficacy confirmed in treating ototoxic hearing loss – Korea Biomedical Review | February 4th, 2021

The outbreak of the Covidpandemic has made many patients reluctantto undergotreatments. While their apprehension seems to overpower them, doctors need to ensure thatstrict guidelines and protocols which assure the best quality service are followed.

Among elective surgeries andtransplants, bone marrow transplant cases have increased substantially in the past few months. Adhering to guidelines for pre-transplant evaluation and the management of a common complication, graft versus host disease (GVHD)is essential.

With the diversity of practice and expertise, the following guidelines will provide a pivotal tool for learning about the rapidly updated therapy landscape in Hematopoietic stem cell transplantation (HSCT).

The guidelines intended to provide a systematic approach for transplantation and help streamline clinical practices and educate new generations of physicians-in-training. Additionally, guidelines can help to evaluate a potential transplant recipient anddetermine if the patient is an eligible candidate for the procedure.

Types and selection of transplantation:

Selection of the type of transplantation for a patient depends on factors such as the type of malignancy, availability of a suitable donor, age of the recipient, the ability to collect a tumor-free autograft, the stage, the malignancy's susceptibility to the GVM effect, and status of disease -- bone marrow involvement, the bulk of disease, chemosensitivity to conventional chemotherapy. This method is particularly applicable for Autologous or Allogeneic Transplantation where one can have a sibling donor or a matched unrelated donor. In the case of a matched unrelated donor, ensure that the collection is adequate and stem cells are available well in time especially if they are imported from countries in Europe.

A haploidentical transplant is another type of transplant that uses healthy, blood-forming cells from a half-matched donor to replace the unhealthy ones. The ideal donor in this case is a family member.

That said, for bone marrow transplant blood products are the backbone and it is important to ensure to have adequate supply before you begin with the transplant.

What are the guidelines and protocols that can be adopted in current times?

Some measures for consideration are: Minimize face-to-face visits including monitoring and consider shifting to telehealth where feasible. Some adaptive community measures like the hospital in the home services, community practices for blood collection, imaging, and support services. For radiation oncology treatment, consider reducing fractions when supported by evidence Consider alternative and less resource-intensive treatment regimes. Minimize unnecessary visitors to cancer centers, for instance, limiting to only patients and their essential caregivers based on frailty and language needs Screen for possible symptoms of COVID-19 and triage patients for admission. If necessary, the admission has to be directed to oncology/hematology departments rather than emergency departments. Immunocompromised patients are likely to have atypical presentations of COVID-19 For suspected checkpoint inhibitor-related pneumonitis prioritizes COVID-19 testing for an early decision regarding corticosteroid therapy.

These are some guidelines that you should heed during a bone marrow transplant. While it is imperative to be updated about the guidelines, timely intervention can reduce the other possible complications during the process.

(The author is the Director, Medical Oncology and Hemato Oncology, atFortis Cancer Institute, Bangalore)

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Understanding bone marrow transplant: The guidelines and the protocols - The New Indian Express

Bone Marrow Stem Cells Comments Off on Understanding bone marrow transplant: The guidelines and the protocols – The New Indian Express | February 4th, 2021

Dave Asprey  |  Photo Credit: Twitter

A man who is determined to live until he is 180 years old says that his bizarre methods will soon be as popular as mobile phones. American millionaire tech entrepreneur Dave Asprey, 47, believes he will live to the year 2153 'at least' by using techniques such as sitting in a cold cryotherapy chamber and intermittent-fasting.

Dave coined the term 'Biohacking' to describe his methods of turning back the biological clock.

He got parts of his bone marrow removed to have the stem cells injected back into his body for $25,000 (Rs 18 lakh).

He speculated that people who are under 40 years of age will be "happy and highly functional" at 100 after applying his methods.

When This Morning's Holly Willoughby asked him why he wants to live so long, he replied, "I'm curious, I think there's a lot of things we can fix and improve in the world and I don't feel like I'm at all done yet."

Dave believes that he won't be the only one to be live for so long.

"The things I am working to pioneer, some of them are expensive, some of them are free like fasting. This will be like cell phones, everyone has cell phones - everyone will have anti-ageing. Change can happen rapidly in society," he said. "There will be many people who are under 40 right now who [will be] walking around under their own power, perfectly happy, highly functional, who are more than 100 years old."

Dave has spent an estimated $1 million on techniques and hacks to try and improve his body's overall functioning.

He added, "I set myself up to have much less inflammation than most people do, by controlling what I eat and how I sleep and a lot of other anti-ageing treatments."

He explained why he re-introduced his stem cells in his body, saying, "When we're young, we have a ton of stem cells and we heal like young people. As we age our stem cells get exhausted, so I do things like intermittent fasting which give me more stem cells and then I take my own stem cells and move them around the body so I heal and move like a young person."

Dave also believes in the benefits of cryotherapy, also known as cold therapy, which is the use of low temperatures in medical therapy to treat a variety of tissue lesions. He has been having cold showers for over ten years.

Another technique that Dave uses to live a long life is intermittent fasting, which involves restricting times that you eat meals to create periods of fasting over a certain period.

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Man who wants to live for 180 years spends Rs 18 lakh to re-inject his own stem cells - Times Now

Bone Marrow Stem Cells Comments Off on Man who wants to live for 180 years spends Rs 18 lakh to re-inject his own stem cells – Times Now | February 4th, 2021

- DKMS-BMST continues to urge Indians to step up to be a potential lifesaver!

BENGALURU, India, Feb. 4, 2021 /PRNewswire/ -- February 4th is marked as World Cancer day every year. This day is observed to spread awareness about the disease and its increasing burden. In line with the theme for this year "Create a futurewithout cancer. The time to act isnow", DKMS BMST Foundation India, a non-profit organization with a mission to provide a second chance at life to blood cancer and blood disorder patients in India makes an appeal to people to come forward and register as potential blood stem cell donors.

On World Cancer Day 2021, DKMS is also celebrating a milestone of providing more than 90,000 blood cancer patients across 57 countries with a second chance at life, since it was founded almost 30 years ago in 1991. DKMS is an international non-profit organization that helps provide patients with lifesaving blood stem cell transplants. DKMS has presence in India, Germany, USA, Poland, UK, Chile, and South Africa.

Patrick Paul, CEO, DKMS BMST Foundation India, says, "DKMS is proud to be the world's leading donor center, accounting for nearly 30% of the total donor pool. While, this is a global milestone, when it comes to India, the fact is that the Indian donors are highly underrepresented in the global database. This is why it becomes difficult for doctors to find a matching blood stem cell donor for Indian patients. While DKMS has registered over 10.5 million donors and has provided over 90,000 patients with a second chance at life globally, it is critical to highlight that only over 43,000 Indian donors are part of this donor pool."

In India, every year, over one lakh people are diagnosed with a form of blood cancer and it remains one of the leading causes of cancer-related deaths among children. Most people are unaware that a life-threatening disease like blood cancer can be treated and in most of the cases, a stem cell transplant is the patient's only chance for survival. For instance, 15-year-old Maheer from Gujarat, India, is one of the blood cancer survivors who had received a lifesaving blood stem cell donation in 2012. He was able to find his matching blood stem cell donor, Dr. Sita, who hails from Germany. Today, he leads a normal, healthy and happy life. He is in grade 9 and loves to travel, read and swim.

Today, more than 37 million potential unrelated donors are listed worldwide with stem cell donor centers and registries, of which only 0.03% are Indians. Currently, in India, the biggest challenge is the lack of awareness about blood stem cell transplant and the importance of registering as a potential blood stem cell donor. The entire procedure is safe and secure. Once the blood stem cells are collected from a donor, they are infused into the patient through a transplant process which then moves through the bloodstream and settles in the bone marrow. These new blood stem cells begin to increase in numbers and produce red blood cells, white blood cells, and platelets, resulting in the replacement of the patient's diseased cells and that's how a blood cancer patient gets a second chance at life. This situation can only be improved by recruiting many more potential stem cell donors from India.

This World Cancer Day, one can take a pledge to become a potential lifesaver. Registration takes only 5 minutes. If one between 18 and 50 years and in good health, the first step to register as a blood stem cell donor by ordering the home swab kit at http://www.dkms-bmst.org/register.

SOURCE DKMS BMST Foundation India

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World Cancer Day 2021: DKMS Announces The Milestone Of Giving 90,000 Blood Cancer Patients Worldwide A Second Chance At Life - PR Newswire India

Bone Marrow Stem Cells Comments Off on World Cancer Day 2021: DKMS Announces The Milestone Of Giving 90,000 Blood Cancer Patients Worldwide A Second Chance At Life – PR Newswire India | February 4th, 2021

Up Market Research (UMR) recently published a report entitled, the Hematopoietic Stem Cell Transplantation (HSCT) Market, describing the crucial aspects of the market by conducting an in-depth analysis of the current trend, emerging threats, and future market assessment. This report takes into account the adverse impact of the COVID-19 pandemic on the market for the period of 2020-2020 and provides a detailed information about how the market will perform during the forecast period, 2020-2027. Our research team presents the report in a simplistic manner supported by fact and actual figures that will assist clients to arrive an informed decision about their investment plans and business strategies.

Request Free Exclusive Sample on Hematopoietic Stem Cell Transplantation (HSCT) Market Report @ https://www.upmarketresearch.com/home/requested_sample/30593

The report provides a holistic view of the market offering key insights of the market behavior over last four years and key assessment of the future market performance. It includes a systematic analysis of historical data for the period, 2015-2020 and draws upon assessment of the market performance for the forecast period, 2020-2027 by observing 2020 as the base year. With the reference to the available data, it provides vital insights on key factors such as drivers, restrains, trends, challenges, and opportunities of the Hematopoietic Stem Cell Transplantation (HSCT) market growth.

This report provides a comprehensive outlook on the key segments and sub-segmentations that includes the complete information about the product types, applications, end-users and regions. It offers latest information regarding the growth rate, volume, and size of the market in respect to each segment and also explains the market performance of these segments in the respective five regions. Moreover, it discusses a wide range of the emerging market scope and potential drawbacks present in the segments.

Hematopoietic Stem Cell Transplantation (HSCT) Market Report Includes:

The Global Hematopoietic Stem Cell Transplantation (HSCT) Market Report is segmented into:

By Types:

AllogeneicAutologous

By Applications:

Peripheral Blood Stem Cells Transplant (PBSCT)Bone Marrow Transplant (BMT)Cord Blood Transplant (CBT)

By Regions:

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The report covers the competitive landscape of various major global players, their current market positions, and key business strategies adopted to mark their major footprint in the market. This includes information about the product launch, expansion of the production facilities or plants, adoption of new technologies, latest merger & acquisition, partnership, and collaboration of the key players. It furthers provides concrete information about the existing market scope for the new entrants and the current competitive levels and scenario for the emerging players in the global market.

The Hematopoietic Stem Cell Transplantation (HSCT) Market Report Covers the Following Companies:

Regen Biopharma IncChina Cord Blood CorpCBR Systems IncEscape Therapeutics IncCryo-Save AGLonza Group LtdPluristem Therapeutics IncViaCord I

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Hematopoietic Stem Cell Transplantation (HSCT) Market Incredible Possibilities, Growth With Industry Study, Detailed Analysis And Forecast To 2027 ...

Bone Marrow Stem Cells Comments Off on Hematopoietic Stem Cell Transplantation (HSCT) Market Incredible Possibilities, Growth With Industry Study, Detailed Analysis And Forecast To 2027 … | February 4th, 2021

Excitement took wing in the scientific community in the early 1990s, when the first gene therapy trial showed significant success, only to crash at the end of the decade with a patients tragic death.

Twenty years later, the excitement is back and greater than before. Although safety remains a concern, investigators are breaking ground in cell and gene therapy, and many believe that ultimately, a string of cured cancers will follow.

In 2017, the excitement over these therapies returned in spades when the FDA signed off on a cell-therapy drug for the first time, approving the chimeric antigen receptor (CAR) T-cell treatment tisagenlecleucel (Kymriah; Novartis) for patients with B-cell precursor acute lymphoblastic leukemia. At last, scientists had devised a way to reprogram a persons own T cells to attack tumor cells.

Were entering a new frontier, said Scott Gottlieb, MD, then-FDA commissioner, in announcing the groundbreaking approval.

Gottlieb was not exaggerating. The growth in CAR T-cell research is exploding. Although only a handful of cell and gene therapies are on the market, the FDA predicted in 2019 that it will receive more than 200 investigational new drug applications per year for cell and gene therapies, and that by 2025, it expects to have accelerated to 10 to 20 cell and gene therapy approvals per year.

We can absolutely cut the number of cancer deaths down so that one day in our lifetimes it can be a rare thing for people to die of cancer, said Patrick Hwu, MD, president and CEO of Moffitt Cancer Center in Florida and among gene therapys pioneers. It still may happen here and there, but itll be kind of like people dying of pneumonia. Its like, He died of pneumonia? Thats kind of weird. I think cancer can be the same way.

Essentially, you can kill any cancer cell that has an antigen that is recognized by the immune cell, Hwu said. The key to curing every single cancer, which is our goal, is to have receptors that can recognize the tumor but dont recognize the normal cells.

Community oncologists will need to be increasingly familiar about the various products, including their immediate and longer-term risks, Bo Wang, MD, and Deepu Madduri, MD, recently wrote in OncologyLive.1 It is key to understand the optimal time for referring these patients to an academic institution, as well as how to manage the requisite post CAR T-cell therapy in the community setting. Madduri is an assistant professor of medicine, hematology and medical oncology, as well as associate director of cellular therapy service, and director of clinical operations with the Center of Excellence for Multiple Myeloma at The Tisch Cancer Institute and the Icahn School of Medicine at Mount Sinai in New York, New York. Wang is a third-year clinical fellow in hematology/oncology at Mount Sinai.

Early referral to academic centers and hospitals equipped to deliver therapies is crucial for patients eligible for therapy. However, as advances continue in the field, community practices may be called upon to administer therapies in their clinic.

The Community Oncology Alliance (COA) envisions a broader role for the settings in which CAR T-cell therapies can be administered. When the Centers for Medicare & Medicaid Services (CMS) was considering coverage for CAR T-cell therapies in 2019, COA officials argued against limiting approvals to hospitals.

It is important to understand that there are state-of-the-art community oncology practices that have significant experience and capabilities in administering highly complex treatments, COA officials wrote in a letter to CMS. For example, stem cell transplants, which are similar in complexity to CAR T therapy, are performed successfully in the community oncology practice setting.2

Broader use of gene therapies depends on several factors, including navigating the logistics of gene therapies, addressing the high costs, and managing toxicities.3

Autologous CAR T-cell therapies involve a manufacturing process that requires coordination between the treating facility and the processing facility. Following leukapheresis, patients may require maintenance therapy to control disease progression during the manufacturing time, which can take 3 to 5 weeks.

In terms of cost, gene and cell therapies can cost from $375,000 to $475,000 per dose and they may face coverage restrictions from payers. Approvals could take weeks to obtain.3,4

Because of cytokine release syndrome and neurotoxicities associated with CAR T-cell therapy, the FDA mandates risk evaluation and mitigation strategy training for centers.

Further, providers may find that real-world experiences with gene therapies are different from those seen in the clinical trial setting, according to Ankit J. Kansagra, MD.

In a presentation at the 2020 American Society of Clinical Oncology Virtual Education Program, Kansagra, an assistant professor of medicine and Eugene P. Frenkel, MD, Scholar in Clinical Medicine at Harold C. Simmons Comprehensive Cancer Center in Dallas, Texas, said that in practice patients may be older and have more aggressive disease, with double- and triple-hit lymphomas.4

Specifically, Kansagra noted that medications such as steroids and/or tocilizumab (Actemra) to prevent or treat cytokine release syndrome or other toxicities were more frequently used in the real-world setting than what had been seen in clinical trials.

As it stands now, only a fraction of eligible patients are receiving CAR T-cell therapies, Kansagra said. Potentially, 9750 patients a year may be eligible for CAR T-cell therapies in approved and upcoming hematologic indications. From 2016 to 2019, a total of 2058 patients received CAR T-cell infusion.4

Next steps for transplanting these novel therapies to clinical practice will require changes in key areas, Kansagra said, such as supply chain management, patient support, and financial systems (Figure).4

Figure. Next Steps for Effective Delivery of Gene and Cell Therapies4

Meanwhile, multiple myeloma experts advise providers to be ready for change. As commercially available myeloma CAR T-cell therapies are approved, it will be even more important for community oncologists to better understand these therapies so they can offer them to their patients, Wang and Madduri wrote.1

Cell therapy involves cultivating or modifying immune cells outside the body before injecting them into the patient. Cells may be autologous (self-provided) or allogeneic (donor-provided); they include hematopoietic stem cells and adult and embryonic stem cells. Gene therapy modifies or manipulates cell expression. There is considerable overlap between the 2 disciplines.

Juliette Hordeaux, PhD, senior director of translational research for the University of Pennsylvanias gene therapy program, is cautious about the FDAs predictions, saying shed be thrilled with 5 cell and/or gene therapy approvals annually.

For monogenic diseases, there are only a certain number of mutations, and then well plateau until we reach a stage where we can go after more common diseases, Hordeaux said.

Safety has been the main brake around adeno-associated virus vector [AAV] gene therapy, added Hordeaux, whose hospitals program has the institutional memory of both Jesse Gelsingers tragic death during a 1999 gene therapy trial as well as breakthroughs by 2015 Giants of Cancer Care winner in immuno-oncology Carl H. June, MD, and others in CAR T-cell therapy. Sometimes there are unexpected toxicity [events] in trials.I think figuring out ways to make gene therapy safer is going to be the next goal for the field before we can even envision many more drugs approved.

In total, 3 CAR T-cell therapies are now on the market, all targeting the CD19 antigen. Tisagenlecleucel was the first. Gilead Sciences received approval in October 2017 for axicabtagene ciloleucel (axi-cel; Yescarta), a CAR T-cell therapy for adults with large B-cell non-Hodgkin lymphoma. Kite Pharma, a subsidiary of Gilead, received an accelerated approval in July 2020 for brexucabtagene autoleucel (Tecartus) for adults with relapsed/ refractory mantle cell lymphoma.

Another CD19-directed therapy under FDA review for relapsed/refractory large B-cell lymphoma, is lisocabtagene maraleucel (liso-cel; JCAR017; Bristol Myers Squibb). Idecabtagene vicleucel (ide-cel; bb2121; Bristol Myers Squibb) is under priority FDA review, with a decision expected by March 31, 2021. The biologics license application for ide-cel seeks approval for the B-cell maturation antigendirected CAR therapy to treat adult patients with multiple myeloma who have received at least 3 prior therapies.5

The number of clinical trials evaluating CAR T-cell therapies has risen sharply since 2015, when investigators counted a total of 78 studies registered on the ClinicalTrials. gov website. In June 2020, the site listed 671 trials, including 357 registered in China, 256 in the United States, and 58 in other countries.6 Natural killer (NK) cells are the research focus of Dean A. Lee, MD, PhD, a physician in the Division of Hematology and Oncology at Nationwide Childrens Hospital in Columbus, Ohio. He developed a method for consistent, robust expansion of highly active clinical-grade NK cells that enables repeated delivery of large cell doses for improved efficacy. This finding led to several first-in-human clinical trials evaluating adoptive immunotherapy with expanded NK cells under an FDA investigational new drug application. Lee is developing both genetic and nongenetic methods to improve tumor targeting and tissue homing of NK cells. His efforts are geared toward pediatric sarcomas.

The biggest emphasis over the past 20 to 25 years has been cell therapy for cancer, talking about trying to transfer a specific part of the immune system for cells, said Lee, who is also director of the Cellular Therapy and Cancer Immunology Program at Nationwide Childrens Hospital, at The Ohio State University Comprehensive Cancer Center Arthur G. James Cancer Hospital, and at the Richard J. Solove Research Institute.

However, Lee said, NKs have wider potential. This is kind of a natural swing back. Now that we know we can grow them, we can reengineer them against infectious disease targets and use them in that [space], he said.

Lee is part of a coronavirus disease 2019 (COVID-19) clinical trial, partnering with Kiadis, for off-the-shelf K-NK cells using Kiadis proprietary platforms. Such treatment would be a postexposure preemptive therapy for treating COVID-19. Lee said the pivot toward treating COVID19 with cell therapy was because some of the very early reports on immune responses to coronavirus, both original [SARS-CoV-2] and the new [mutation], seem to implicate that those who did poorly [overall] had poorly functioning NK cells.

The revolutionary gene editing tool CRISPR is making its initial impact in clinical trials outside the cancer area. Its developers, Jennifer Doudna, PhD, and Emmanuelle Charpentier, PhD, won the Nobel Prize in Chemistry 2020.

For patients with sickle cell disease (SCD), CRISPR was used to reengineer bone marrow cells to produce fetal hemoglobin, with the hope that the protein would turn deformed red blood cells into healthy ones. National Public Radio (NPR) did a story on one patient who, so far, thanks to CRISPR, has been liberated from the attacks of SCD that typically have sent her to the hospital, as well from the need for blood transfusions.7

Its a miracle, you know? the patient, Victoria Gray of Forest, Mississippi, told NPR.

She was among 10 patients with SCD or transfusion-dependent beta-thalassemia treated with promising results, as reported by the New England Journal of Medicine.8

Stephen Gottschalk, MD, chair of the department of bone marrow transplantation and cellular therapy at St Jude Childrens Research Hospital, said, Theres a lot of activity to really explore these therapies with diseases that are much more common than cancer.

Animal models use T cells to reverse cardiac fibrosis, for instance, Gottschalk said. Using T cells to reverse pathologies associated with senescence, such as conditions associated with inflammatory clots, are also being studied.

CAR T, I think, will become part of the standard of care, Gottschalk said. The question is how to best get that accomplished. To address the tribulations of some autologous products, a lot of groups are working with off-the-shelf products to get around some of the manufacturing bottlenecks. I believe those issues will be solved in the long run.

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Harnessing the Potential of Cell and Gene Therapy - OncLive

Cardiac Stem Cells Comments Off on Harnessing the Potential of Cell and Gene Therapy – OncLive | February 4th, 2021

With her sun-kissed hair and flawless golden skin, it's easy to forget that Heidi Klum is in her mid 40s. Genetics certainly help. But so do Heidi Klum's favourite skincare products by Glossier and Drunk Elephant, plus a reminder of home courtesy of German beauty brand The Cream by Augustinus Bader.

In a rare selfie, Klum showed off the Perfectil Hair Skin And Nails Vitamins she takes daily, alongside this smorgasbord of beauty products, which proves the supermodel takes her skincare seriously.

Here are some of her favourite products:

More from womanandhome:

Drunk Elephant T.L.C. Framboos Glycolic Resurfacing Night Serum

This night serum contains a hefty dose of glycol acid to exfoliate built-up dead skin cells and resurface dull, uneven skin. Expect brighter, smoother skin when you wake.

Drunk Elephant T.L.C. Sukari Babyfacial 25% AHA + 2% BHA Mask

If you like to feel a product working, you'll positive love this mask. Like an AHA/BHA facial in a bottle, it resurfaces skin to reveal greater clarity and improved skin tone.

Drunk Elephant C-Firma Day Serum

This vitamin C day serum is packed with antioxidants to protect skin from urban aggressors.

Glossier Solution

A liquid exfoliator that you apply a bit like toner, it buffs away dead dulling skin with a single swipe.

Mario Badescu Drying Lotion

We know, we know, even supermodels get spots. This cult zit lotion contains salicylic acid, sulfur, and zinc oxide to dry up blemishes - fast.

Augustinus Bader The Rich Cream

The brainchild of a stem cell scientist no less, this overnight treatment uses amino acids and vitamins to re-energise cells to repair damage more effectively.

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Heidi Klum loves to pamper her skin with goodies from Drunk Elephant and Glossier - woman&home

Skin Stem Cells Comments Off on Heidi Klum loves to pamper her skin with goodies from Drunk Elephant and Glossier – woman&home | February 4th, 2021

Gaining more fat cells is probably not what most people want, although that might be exactly what they need to fight off diabetes and other diseases. How and where the body can add fat cells has remained a mystery - but two new studies from UT Southwestern provide answers on the way this process works.

The studies, both published online in Cell Stem Cell, describe two different processes that affect the generation of new fat cells. One reports how fat cell creation is impacted by the level of activity in tiny organelles inside cells called mitochondria. The other outlines a process that prevents new fat cells from developing in one fat storage area in mice - the area that correlates with the healthy subcutaneous fat just under the skin in humans. (Both studies were done in mice.)

In the second study, a commonly used cancer drug was able to jump-start healthy fat cell creation in mice, a finding that raises the possibility of future drug treatments for humans.

While fat isn't popular, as long as people overeat they will need a place to store the excess calories, explains Philipp Scherer, Ph.D., director of the Touchstone Center for Diabetes Research at UT Southwestern and senior author of the first study focusing on mitochondria. There are two options, he says: squeezing more lipids (fat) into existing fat cells and ballooning their size, leading to problems such as inflammation and, eventually, diabetes; or creating new fat cells to help spread the load. Fat stored properly - in fat cell layers under the skin (subcutaneous fat) that aren't overburdened instead of around organs (visceral fat) or even inside organs - is the healthy alternative, he says.

Problems follow if existing fat cells are left on their own to become engorged, adds Rana Gupta, Ph.D., associate professor of internal medicine and senior author of the second study. "When these cells are so overwhelmed that they can't take it anymore, they eventually die or become dysfunctional, spilling lipids into places not intended to store fat."

Those lipids may move into the liver, leading to fatty liver disease; to the pancreas, resulting in diabetes; or even to the heart, causing cardiovascular disease, Gupta says. Visceral, or belly fat, may surround the organs, creating inflammation.

The healthiest place to store fat is in subcutaneous fat, adds Gupta. Ironically, that is where mice in his study were least able to create new fat cells, despite the fact that stem-cell-like progenitor cells primed to become fat cells were present there as well, he says.

Gupta's study identified a process that prevents progenitor cells from developing into fat cells in mouse subcutaneous inguinal fat.

The protein HIF-1a (short for hypoxia-inducible factor-1 alpha) is central to the process. It kicks off a series of cellular actions that ultimately inactivate a second protein called PPARgamma, the key driver of fat cell formation.

These proteins are found in both humans and mice. In fact, in a culture of human subcutaneous fat cell progenitors, HIF-1a also inhibited new fat cells from being created, according to Gupta.

In Gupta's mouse study, researchers used a genetic approach to inhibit HIF-1a and found that the progenitor cells could then make subcutaneous inguinal fat cells and fewer were inflamed or fibrotic.

Next, they tested the cancer drug imatinib (brand name Gleevec) and found it had the same effect. The cancer drug was tried because it was known to have beneficial effects against diabetes in cancer patients with both diseases, Gupta says.

In Scherer's study, researchers manipulated a protein called MitoNEET in the outer membrane of the precursor cells' mitochondria, organelles known as the cells' power plants. The resulting mitochondrial dysfunction and drop in cell metabolism caused precursor cells to lose the ability to become new fat cells and increased inflammation.

"This study shows we can manipulate the precursor cells' willingness to become fat cells," Scherer says. "The ability to recruit new fat cells by tickling these pre-fat cells to become fat cells is very important and has profound beneficial effects on health, particularly in the obesity-prone environment that we all live in."

He says his goal is now to design a drug that could stimulate mitochondrial activity.

"Understanding the mechanism is an important first step," Scherer says, referring to the findings from the two studies. "We will have to learn in the future how to manipulate these processes pharmacologically."

Reference: Joffin N, Paschoal VA, Gliniak CM, et aI. Mitochondrial metabolism is a key regulator of the fibro-inflammatory and adipogenic stromal subpopulations in white adipose tissue. Cell Stem Cell. doi:doi.org/10.1016/j.stem.2021.01.002

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

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Two Studies Shed Light on How and Where the Body Can Add New Fat Cells - Technology Networks

Skin Stem Cells Comments Off on Two Studies Shed Light on How and Where the Body Can Add New Fat Cells – Technology Networks | February 4th, 2021