BRIDGEPORT, Conn. (WTNH) The Gift of Life Marrow Registry organized the meeting Thursday between a bone marrow donor from Connecticut and the recipient whose life was saved by the donation.

Jennie Bunce, 25, of Redding donated her marrow. According to a representative for Gift of Life, Bunce was studying physical therapy and joined Gift of Life through a sorority event at North Carolinas High Point University in 2016.

I never win or get picked for anything, but it just felt like the right thing to do, Bunce told Gift of Life. Im just incredibly happy and grateful to be part of something so special. Its similar to holding the door open for someone or helping a friend in a time of need.

Across the country in Mesa, Arizona, father-of-6, Mark Roser, 33, was battling Acute Lymphoblastic Leukemia. He found out about the diagnosis after he broke a hip in 2018 and had continued weakness. Roser was told he needed a bone marrow transplant to survive.

The hardest part was knowing, no matter how hard I worked, that what I did would not be a deciding factor in my ability to receive this gift, said Roser.

The match was made by Gift of Life in about six months, and the transplant took place in Phoenix.

She is a hero to all the people in my life, said Roser.

She gave me life, she gave my children a future with their dad, she gave my wife a chance to hold her husband, to have someone hold her back. She allowed me to go to work, to play, to see things from a different perspective. I am grateful for every moment I have, and its because of her.

According to Gift a Life, medical privacy laws dictate that recipients and donors must remain anonymous and wait at least a year before meeting.

The two came face-to-face for the first time Thursday in Bridgeport at the Boca Oyster Bar.

Since its start in 1991, the Gift of Life Registry 349,000 individuals who have donated blood stem cells or bone marrow to save a life. The program has facilitated 16,800 matches and over 3,500 transplants.

To learn more about the organization and/or how to donate: https://www.giftoflife.org/.

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Bone marrow recipient comes face-to-face with CT donor for the first time - WTNH.com

Bone Marrow Stem Cells Comments Off on Bone marrow recipient comes face-to-face with CT donor for the first time – WTNH.com | October 18th, 2019

Company Announcement

Copenhagen, Denmark; October 18, 2019 Genmab A/S (Nasdaq:GMAB) announced today that the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) has issued a positive opinion recommending broadening the existing marketing authorization for DARZALEX (daratumumab) in the European Union. The recommendation is for the use of DARZALEX in combination with lenalidomide and dexamethasone (Rd) as treatment for newly diagnosed adult patients with multiple myeloma who are ineligible for autologous stem cell transplant (ASCT). The Type II variation application, based on the Phase III MAIA (MMY3008) study, was submitted to the EMA by Janssen Pharmaceutica NV in March 2019. In August 2012, Genmab granted Janssen Biotech, Inc. an exclusive worldwide license to develop, manufacture and commercialize daratumumab.

We are encouraged that the CHMP are recommending a broadening of the current DARZALEX marketing authorization in the European Union to include DARZALEX in combination with lenalidomide and dexamethasone as a possible treatment for patients newly diagnosed with multiple myeloma. This would give patients another treatment option, in addition to the already approved combination of daratumumab plus bortezomib, melphalan and prednisone in this same setting, said Jan van de Winkel, Ph.D., Chief Executive Officer of Genmab.

About the MAIA (MMY3008) studyThe Phase III study (NCT02252172) is a randomized, open-label, multicenter study that includes 737 newly diagnosed patients with multiple myeloma who are not candidates for high dose chemotherapy and ASCT. Patients were randomized to receive either treatment with daratumumab in combination with lenalidomide (an immunomodulatory drug) and dexamethasone (a corticosteroid) or treatment with lenalidomide and dexamethasone alone. In the daratumumab treatment arm, patients received 16 milligrams per kilogram (mg/kg) weekly for first 8 weeks (Cycles 1 and 2), every other week for 16 weeks (Cycles 3 to 6) and then every 4 weeks (Cycle 7 and beyond) until progression of disease or unacceptable toxicity. Lenalidomide is administered at 25 mg orally on days 1 through 21 of each 28-day cycle, and dexamethasone is administered at 40 mg once a week for both treatment arms. Participants in both treatment arms will continue Rd until disease progression or unacceptable toxicity. The primary endpoint of the study is progression free survival.

About multiple myelomaMultiple myeloma is an incurable blood cancer that starts in the bone marrow and is characterized by an excess proliferation of plasma cells.1 Approximately 16,830 new patients were expected to be diagnosed with multiple myeloma and approximately 10,480 people were expected to die from the disease in the Western Europe in 2018.2 Globally, it was estimated that 160,000 people were diagnosed and 106,000 died from the disease in 2018.3 While some patients with multiple myeloma have no symptoms at all, most patients are diagnosed due to symptoms which can include bone problems, low blood counts, calcium elevation, kidney problems or infections.4

About DARZALEX (daratumumab)DARZALEX (daratumumab) intravenous infusion is indicated for the treatment of adult patients in the United States: in combination with bortezomib, thalidomide and dexamethasone as treatment for patients newly diagnosed with multiple myeloma who are eligible for autologous stem cell transplant; in combination with lenalidomide and dexamethasone for the treatment of patients with newly diagnosed multiple myeloma who are ineligible for autologous stem cell transplant; in combination with bortezomib, melphalan and prednisone for the treatment of patients with newly diagnosed multiple myeloma who are ineligible for autologous stem cell transplant; in combination with lenalidomide and dexamethasone, or bortezomib and dexamethasone, for the treatment of patients with multiple myeloma who have received at least one prior therapy; in combination with pomalidomide and dexamethasone for the treatment of patients with multiple myeloma who have received at least two prior therapies, including lenalidomide and a proteasome inhibitor (PI); and as a monotherapy for the treatment of patients with multiple myeloma who have received at least three prior lines of therapy, including a PI and an immunomodulatory agent, or who are double-refractory to a PI and an immunomodulatory agent.5 DARZALEX is the first monoclonal antibody (mAb) to receive U.S. Food and Drug Administration (U.S. FDA) approval to treat multiple myeloma. DARZALEX is indicated in Europe in combination with bortezomib, melphalan and prednisone for the treatment of adult patients with newly diagnosed multiple myeloma who are ineligible for autologous stem cell transplant; for use in combination with lenalidomide and dexamethasone, or bortezomib and dexamethasone, for the treatment of adult patients with multiple myeloma who have received at least one prior therapy; and as monotherapy for the treatment of adult patients with relapsed and refractory multiple myeloma, whose prior therapy included a PI and an immunomodulatory agent and who have demonstrated disease progression on the last therapy6. The option to split the first infusion of DARZALEX over two consecutive days has been approved in both Europe and the U.S. In Japan, DARZALEX is approved in combination with lenalidomide and dexamethasone, or bortezomib and dexamethasone, for the treatment of adults with relapsed or refractory multiple myeloma and in combination with bortezomib, melphalan and prednisone for the treatment of patients with newly diagnosed multiple myeloma who are ineligible for autologous stem cell transplant. DARZALEX is the first human CD38 monoclonal antibody to reach the market in the United States, Europe and Japan. For more information, visit http://www.DARZALEX.com.

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Daratumumab is a human IgG1k monoclonal antibody (mAb) that binds with high affinity to the CD38 molecule, which is highly expressed on the surface of multiple myeloma cells. Daratumumab triggers a persons own immune system to attack the cancer cells, resulting in rapid tumor cell death through multiple immune-mediated mechanisms of action and through immunomodulatory effects, in addition to direct tumor cell death, via apoptosis (programmed cell death).5,6,7,8,9,10

Daratumumab is being developed by Janssen Biotech, Inc. under an exclusive worldwide license to develop, manufacture and commercialize daratumumab from Genmab. A comprehensive clinical development program for daratumumab is ongoing, including multiple Phase III studies in smoldering, relapsed and refractory and frontline multiple myeloma settings. Additional studies are ongoing or planned to assess the potential of daratumumab in other malignant and pre-malignant diseases in which CD38 is expressed, such as amyloidosis, NKT-cell lymphoma and B-cell and T-cell ALL. Daratumumab has received two Breakthrough Therapy Designations from the U.S. FDA for certain indications of multiple myeloma, including as a monotherapy for heavily pretreated multiple myeloma and in combination with certain other therapies for second-line treatment of multiple myeloma.

About Genmab Genmab is a publicly traded, international biotechnology company specializing in the creation and development of differentiated antibody therapeutics for the treatment of cancer. Founded in 1999, the company has two approved antibodies, DARZALEX (daratumumab) for the treatment of certain multiple myeloma indications, and Arzerra (ofatumumab) for the treatment of certain chronic lymphocytic leukemia indications. Daratumumab is in clinical development for additional multiple myeloma indications, other blood cancers and amyloidosis. A subcutaneous formulation of ofatumumab is in development for relapsing multiple sclerosis. Genmab also has a broad clinical and pre-clinical product pipeline. Genmab's technology base consists of validated and proprietary next generation antibody technologies - the DuoBody platform for generation of bispecific antibodies, the HexaBody platform, which creates effector function enhanced antibodies, the HexElect platform, which combines two co-dependently acting HexaBody molecules to introduce selectivity while maximizing therapeutic potency and the DuoHexaBody platform, which enhances the potential potency of bispecific antibodies through hexamerization. The company intends to leverage these technologies to create opportunities for full or co-ownership of future products. Genmab has alliances with top tier pharmaceutical and biotechnology companies. Genmab is headquartered in Copenhagen, Denmark with core sites in Utrecht, the Netherlands and Princeton, New Jersey, U.S.

Contact: Marisol Peron, Corporate Vice President, Communications & Investor Relations T: +1 609 524 0065; E: mmp@genmab.com

For Investor Relations: Andrew Carlsen, Senior Director, Investor RelationsT: +45 3377 9558; E: acn@genmab.com

This Company Announcement contains forward looking statements. The words believe, expect, anticipate, intend and plan and similar expressions identify forward looking statements. Actual results or performance may differ materially from any future results or performance expressed or implied by such statements. The important factors that could cause our actual results or performance to differ materially include, among others, risks associated with pre-clinical and clinical development of products, uncertainties related to the outcome and conduct of clinical trials including unforeseen safety issues, uncertainties related to product manufacturing, the lack of market acceptance of our products, our inability to manage growth, the competitive environment in relation to our business area and markets, our inability to attract and retain suitably qualified personnel, the unenforceability or lack of protection of our patents and proprietary rights, our relationships with affiliated entities, changes and developments in technology which may render our products or technologies obsolete, and other factors. For a further discussion of these risks, please refer to the risk management sections in Genmabs most recent financial reports, which are available on http://www.genmab.com and the risk factors included in Genmabs final prospectus for our U.S. public offering and listing and other filings with the U.S. Securities and Exchange Commission (SEC), which are available at http://www.sec.gov. Genmab does not undertake any obligation to update or revise forward looking statements in this Company Announcement nor to confirm such statements to reflect subsequent events or circumstances after the date made or in relation to actual results, unless required by law.

Genmab A/S and/or its subsidiaries own the following trademarks: Genmab; the Y-shaped Genmab logo; Genmab in combination with the Y-shaped Genmab logo; HuMax; DuoBody; DuoBody in combination with the DuoBody logo; HexaBody; HexaBody in combination with the HexaBody logo; DuoHexaBody; HexElect; and UniBody. Arzerra is a trademark of Novartis AG or its affiliates. DARZALEX is a trademark of Janssen Pharmaceutica NV.

1 American Cancer Society. "Multiple Myeloma Overview." Available at http://www.cancer.org/cancer/multiplemyeloma/detailedguide/multiple-myeloma-what-is-multiple-myeloma.Accessed June 2016.2 Globocan 2018. Western Europe Fact Sheet. Available at http://gco.iarc.fr/today/data/factsheets/populations/926-western-europe-fact-sheets.pdf Accessed March 20183 Globocan 2018. World Fact Sheet. Available at http://gco.iarc.fr/today/data/factsheets/populations/900-world-fact-sheets.pdf. Accessed December 2018.4 American Cancer Society. "How is Multiple Myeloma Diagnosed?" http://www.cancer.org/cancer/multiplemyeloma/detailedguide/multiple-myeloma-diagnosis. Accessed June 20165 DARZALEX Prescribing information, September 2019. Available at: https://www.accessdata.fda.gov/drugsatfda_docs/label/2019/761036s024lbl.pdf Last accessed September 20196 DARZALEX Summary of Product Characteristics, available at https://www.ema.europa.eu/en/medicines/human/EPAR/darzalex Last accessed October 20197De Weers, M et al. Daratumumab, a Novel Therapeutic Human CD38 Monoclonal Antibody, Induces Killing of Multiple Myeloma and Other Hematological Tumors. The Journal of Immunology. 2011; 186: 1840-1848.8 Overdijk, MB, et al. Antibody-mediated phagocytosis contributes to the anti-tumor activity of the therapeutic antibody daratumumab in lymphoma and multiple myeloma. MAbs. 2015; 7: 311-21.9 Krejcik MD et al. Daratumumab Depletes CD38+ Immune-regulatory Cells, Promotes T-cell Expansion, and Skews T-cell Repertoire in Multiple Myeloma. Blood. 2016; 128: 384-94.10Jansen, JH et al. Daratumumab, a human CD38 antibody induces apoptosis of myeloma tumor cells via Fc receptor-mediated crosslinking.Blood. 2012; 120(21): abstract 2974.

Company Announcement no. 50CVR no. 2102 3884LEI Code 529900MTJPDPE4MHJ122

Genmab A/SKalvebod Brygge 431560 Copenhagen VDenmark

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CHMP Issues Positive Opinion Recommending DARZALEX (Daratumumab) in Combination with Lenalidomide and Dexamethasone in Frontline Multiple Myeloma -...

Bone Marrow Stem Cells Comments Off on CHMP Issues Positive Opinion Recommending DARZALEX (Daratumumab) in Combination with Lenalidomide and Dexamethasone in Frontline Multiple Myeloma -… | October 18th, 2019

Results of an ex vivo study evaluating the phenotypic and growth characteristics of T cells collected by leukapheresis from cohorts of patients with newly diagnosed or relapsed/refractory multiple myeloma support use of chimeric antigen receptor (CAR)-T therapy earlier in course of the disease. The hypothesis-generating findings from this study were published in Blood Advances.

While CAR-T therapy targeted against the B-cellmaturation antigen (BCMA) has been associated with promising results inpatients with multiple myeloma, nearly all of the patients responding to thisapproach eventually develop progressive disease. Hence, strategies to optimizepatient selection for CAR-T therapy in the setting of multiple myeloma arebeing actively pursued.

Theratio of CD4 to CD8 T cells and/or the frequency of the CD81 CD45RO2 CD271 T-cell memory phenotype were usedin this study as surrogates for the clinical effectiveness of CAR-T therapysince previous studies ofCAR-T therapy in patients with chronic lymphocyticleukemia and multiple myeloma showed that of all baseline patient- anddisease-related characteristics considered, clinical response to CAR-T therapywas associated only with this T-cell ratio and/or the frequency of this subsetof memory T cells in the premanufacturing leukapheresis product.

Twocohorts of patients where compared in this study: 38 patients with newly diagnosedmultiple myeloma who had participated in clinical trials of induction therapy andon whom leukapheresis was performed before consolidation therapy and autologousstem cell transplantation (ASCT); and 25 patients with relapsed/refractorymultiple myeloma enrolled in a phase 1 clinical trial of anti-BCMA CAR-Ttherapy and on whom leukapheresis was performed during a washout period shortlyfollowing study enrollment.

Inboth patient cohorts, leukapheresis samples were exposed ex vivo to anti-CD3and anti-CD28 monoclonal antibodies covalently linked to magnetic beads toprovide stimulatory/costimulatory signals for T-cell proliferation and theexpansion of functional T cells.

The 2 patient cohorts were similar with respect to median age (ie, 55 years; 58 years [relapsed/refractory]), although the time from multiple myeloma diagnosis was 222 days for those treated with induction therapy and 4.6 years for those with relapsed/refractory disease.

Inaddition, differences in the median number of prior lines of therapy (1 vs 7),and bone marrow cellularity occupied by myeloma plasma cells (13% vs 65%) wereobserved when the former and latter cohorts were compared at the time thatleukapheresis was performed.

Akey finding from this study was a significantly higher frequency of T cellswith the CD81 CD45RO2CD271 T-cell memory phenotype(43.9% vs 29.0%; P =.001), as well asa significantly higher median CD4/CD8 ratio (2.6 vs 0.87; P <.0001) in the postinduction versus the relapsed/refractorypatient cohort.

Inaddition, the CD4/CD8 ratio was also significantly higher in the postinductioncohort compared with responders to anti-BCMA CAR-T therapy from the relapsed/refractorycohort (2.6 vs 1.3; P= .0009); however,while higher in the postinduction cohort, the difference in the frequency of Tcells with the CD81 CD45RO2CD271 T-cell memory phenotypewas not statistically significant when these 2 groups were compared.

Regardingcapacity for ex vivo proliferation during manufacturing, significantly highernumbers of population doubling by day 9 (PD9) were observed for thepostinduction cohort compared with either the overall relapsed/refractorycohort or the group of responders within the relapsed/refractory cohort.

Ourresults suggest that CAR T cells manufactured from leukapheresis samplesobtained after response to induction therapy would be, on average, moreclinically effective than those obtained from heavily relapsed/refractorymultiple myeloma patients, the study authors concluded.

Reference

Garfall AL, Dancy EK, Cohen AD, et al. T-cell phenotypes associated with effective CAR T-cell therapy in postinduction vs relapsed multiple myeloma. Blood Adv. 2019;3:2812-2815.

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CAR-T Therapy May Be More Effective When Administered Earlier in the Multiple Myeloma Treatment Continuum - Cancer Therapy Advisor

Bone Marrow Stem Cells Comments Off on CAR-T Therapy May Be More Effective When Administered Earlier in the Multiple Myeloma Treatment Continuum – Cancer Therapy Advisor | October 18th, 2019

BALTIMOREand PHILADELPHIA and IRVINE, Calif., Oct. 15, 2019 (GLOBE NEWSWIRE) -- WindMIL Therapeutics and the University of California, Irvine (UCI) today announced that the first patients have been identified in an investigator-sponsored study for the collection of bone marrow from patients with gliomas. The study will evaluate generating marrow infiltrating lymphocytes (MILs) for these patients through WindMILs proprietary cellular activation and expansion process. The study is being conducted at UCI.

Patients suffering with glioblastoma are in great need of new, promising treatments that might advance the current standard of care, said Daniela A. Bota, MD, PhD, director of theUCI Health Comprehensive Brain Tumor Program,seniorassociate dean for clinical research, UCI School of Medicine and clinical director, UCI Sue & Bill Gross Stem Cell ResearchCenter. The University of California, Irvine is excited toplay a key role in research that may lead to a clinical trial that enlists the immune system in novel ways to fight this terrible disease.

Gliomas are the most common of the malignant brain tumors. Glioblastoma, the most common glioma, has a five-year survival of less than 5 percent. Additional treatment options are urgently needed for these patients. Adoptive immunotherapy is a possible approach for gliomas and the use of MILs, a cell therapy that is naturally tumor-specific, is one such treatment option.

The bone marrow is a unique niche in the immune system to which antigen-experienced memory T cells traffic and are then maintained. WindMIL has developed a proprietary process to select, activate and expand these memory T cells into MILs. Because memory T cells in bone marrow occur as a result of the immune systems recognition of tumor antigens, MILs are specifically suited for adoptive cellular immunotherapy and are able to directly eradicate or facilitate eradication of each patients unique cancer. WindMIL is currently studying MILs in multiple myeloma, non-small cell lung cancer and squamous cell carcinoma of the head and neck, and plans to expand into other solid tumors.

WindMIL is looking forward to working with the University of California, Irvine on this exciting project and is optimistic that MILs may offer the potential to help patients with these hard-to-treat diseases, said Monil Shah, PharmD, MBA, Chief Development Officer at WindMIL.

About WindMIL Therapeutics

WindMIL Therapeutics is a clinical-stage company developing a novel class of autologous cell therapies based on marrow infiltrating lymphocytes (MILs) for cancer immunotherapy. As the leader in cellular therapeutics emanating from bone marrow, WindMIL translates novel insights in bone marrow immunology into potentially life-saving cancer immunotherapeutics for patients. WindMIL believes that Cell Source Matters and the companys proprietary process to extract, activate and expand these cells offers unique immunotherapeutic advantages, including inherent poly-antigen specificity, high cytotoxic potential and long persistence. For more information, please visit: http://www.windmiltx.com.

About UCI Health

UCI Healthcomprises the clinical enterprise of the University of California, Irvine. Patients can access UCI Health at primary and specialty care offices across Orange County and at its main campus, UCI Medical Center in Orange, California. The 417-bed acute care hospital provides tertiary and quaternary care, ambulatory and specialty medical clinics and behavioral health and rehabilitation services. UCI Medical Center features Orange Countys only National Cancer Institute-designated comprehensive cancer center, high-risk perinatal/neonatal program and American College of Surgeons-verified Level I adult and Level II pediatric trauma center and regional burn center. UCI Health serves a region of nearly 4 million people in Orange County, western Riverside County and southeast Los Angeles County. Follow us onFacebookandTwitter.

About the University of California, Irvine

Founded in 1965, UCI is the youngest member of the prestigious Association of American Universities. The campus has produced three Nobel laureates and is known for its academic achievement, premier research, innovation and anteater mascot. Led by Chancellor Howard Gillman, UCI has more than 36,000 students and offers 222 degree programs. Its located in one of the worlds safest and most economically vibrant communities and is Orange Countys second-largest employer, contributing $5 billion annually to the local economy. For more on UCI, visitwww.uci.edu.

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WindMIL Therapeutics and University of California, Irvine Announce Collaboration to Collect Bone Marrow from Patients with Gliomas to Develop Marrow...

Bone Marrow Stem Cells Comments Off on WindMIL Therapeutics and University of California, Irvine Announce Collaboration to Collect Bone Marrow from Patients with Gliomas to Develop Marrow… | October 16th, 2019

A case study published in Leukemia and Lymphoma described a patient with a diagnosis of Waldenstrm Macroglobulinemia (WM) that had subsequently undergone histological transformation to refractory high grade B-cell lymphoma and was successfully treated with CD19-targeted chimeric antigen receptor (CAR)-T cell therapy.1

WMis a type of B-cell non-Hodgkin lymphoma(NHL), typically characterized by overproduction of monoclonal immunoglobulinM, as well as infiltration of malignant lymphoplasmacytic cells into the bonemarrow.

Although considered incurable, WM often follows an indolent course andsome patients can be asymptomatic for long periods. Rarely, the diseasetransforms into a more aggressive form of NHL that has been associated with apoor prognosis.

The patient described in the case studywas a 71-year-old man who was first diagnosed with WM in 1998. The patient wasmonitored without undergoing active treatment for a period of 12 years, atwhich time he developed anemia and splenomegaly. At that time, he underwenttreatment with 6 cycles of fludarabine and rituximab and achieved a partial response totreatment. Following a worsening of symptoms 4 years later, the patient wastreated with 6 cycles of bendamustine and rituximab.

Biopsyof an enlarged cervical lymph node performed at that time revealed high-gradeB-cell lymphoma that was clonally related to the previously seenlymphoplasmacytic infiltrate, consistent with transformation.

Thepatient subsequently achieved a complete response to 6 cycles of rituximab,cyclophosphamide, doxorubicin, vincristine, prednisone (R-CHOP) plus ibrutinibfollowed by 6 months of ibrutinib maintenance therapy that lasted for 18months.

Salvagetherapy included 2 cycles of rituximab, dexamethasone, cytarabine, cisplatin(R-DHAP) followed by 1 cycle of rituximab plus high-dose cytarabine, followedby autologous stem cell transplantation several months later.

Asthe patients disease was considered to be chemorefractory based on subsequent imagingand pathological analyses, he was treated with axicabtagene ciloleucel, aCD19-targeted CAR-T cell therapy currently approved for the treatment of adultpatients with relapsed or refractory large B-cell lymphoma, including high gradeB-cell lymphoma, after 2 or more lines of systemic therapy.2

Althoughthe patient experienced pancytopenia, grade 1 cytokine release syndrome, andgrade 1 neurotoxicity following CAR-T therapy, he achieved a complete response1 month following treatment.

Notably,there was no evidence of either underlying WM or transformed disease at 6 and 12months follow-up.

Thestudy authors noted that longer term follow up in this patient will beinformative, as late relapses have occurred even in patients who achieve a deepresponse after transplant. CAR-T cell therapy may be an effective treatment forrelapsed or refractory WM that has not yet undergone histologicaltransformation, as CD19 is almost universally expressed on lymphoplasmacyticlymphoma cells. The researchers concluded that further analysis of this iswarranted in the context of clinical trials.

References

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Benefit of CD19-Targeted CAR-T Therapy in Patients With Transformed Waldenstrm Macroglobulinemia - Cancer Therapy Advisor

Bone Marrow Stem Cells Comments Off on Benefit of CD19-Targeted CAR-T Therapy in Patients With Transformed Waldenstrm Macroglobulinemia – Cancer Therapy Advisor | October 16th, 2019

Organizers of this years Light the Night, which is held in support of blood cancer research, hope members of the community can join them Oct. 19 as they take to the Rotary Trail to raise awareness and funds for the eradication of various blood cancers.

Leukemia, lymphoma, Hodgkins lymphoma and myeloma can all be beat, but for every inspirational tale of perseverance and survival, there are even more about those who could not hang on long enough and died as a result of the fast moving diseases that affect blood cells, bone marrow and lymph nodes to name a few.

I lost my niece to leukemia 11 years ago, she was 19, said Westlock organizer Joanne Rimmer, with tears in her eyes as fresh as the day she lost her niece.

I had positive thoughts the whole time, I thought she was going to get over it and everything was going to be back to normal and it didnt work out. I didnt want other families to go through that, so I thought it was a good thing to help raise money.

It has been 11 years and Im still crying. Its one way we can do something to say we really miss her.

Rimmer also has a close friend who was able to survive leukemia after a donation of stem cells from her brother saved her life. She also has another friend in Manitoba who is currently fighting off leukemia, with some success. These are her reasons for lighting up the night, so called because participants often hold lanterns of different colours that denote how thatindividual has been affected.

White lanterns are carried by survivors, gold is in remembrance and red is in support, which together makes for quite a sea of colour moving through the town.

Rimmer, who has been taking part in the event in one form or another for 11 years and helping to organize the Westlock event for the last six years is a proponent of having a bare-bones event with little flash or overhead to make sure as much money as possible is donated to the Leukemia and Lymphoma Society of Canada.

Any sponsors or potential contributors are asked to donate directly to the cause instead of providing other supports, which are appreciated, but ultimately unneeded.

I want every dollar that gets raised to actually go towards what were actually raising money for. I dont want to waste it on silly things.

Rimmer is urging those who want to participate to start collecting sponsors, and to register at http://www.lightthenight.ca. Participants should meet at the Rotary Spirit Centre after 6:30 p.m. Oct. 19 for the walk that will begin at 7 p.m., winding it way east on the Rotary Trail to the healthcare centre, then eventually on to the pool. All are welcome to join, from babies in strollers to their great-great parents, said Rimmer, who also noted the event will take place rain or shine.

Link:
Light the Night Oct. 19 - Westlock News

Bone Marrow Stem Cells Comments Off on Light the Night Oct. 19 – Westlock News | October 16th, 2019

DUBLIN--(BUSINESS WIRE)--The "Cell Harvesting Market" report has been added to ResearchAndMarkets.com's offering.

This report provides a summary of the cell harvesting market, including a market snapshot and profiles of key players in the market. It provides an exhaustive segmentation analysis of the market with in-depth information about each segment. The overview section of the report provides a description of market trends and market dynamics, including drivers, restraints and opportunities. it provides information about market developments and future trends that can be useful for organizations, including wholesalers and exporters. It provides market positionings of key players using yardsticks of revenue, product portfolio, and recent activities. It further includes strategies adopted by emerging market players with strategic recommendations for new market entrants. Readers will also find historical and current market sizes and a discussion of the market's future potential. The report will help market players and new entrants make informed decisions about the production and exports of goods and services.

The report includes:

Key Topics Covered:

Chapter 1 Introduction

Chapter 2 Summary and Highlights

Chapter 3 Market and Technology Background

Chapter 4 Market Breakdown by Type of Harvesting

Chapter 5 Market Breakdown by End User

Chapter 6 Market Breakdown by Application

Chapter 7 Market Breakdown by Component/Equipment

Chapter 8 Market Breakdown by Region

Chapter 9 New Developments and Patent Review

Chapter 10 Analysis of Market Opportunities

Chapter 11 Company Profiles

For more information about this report visit https://www.researchandmarkets.com/r/lahvpd

About ResearchAndMarkets.com

ResearchAndMarkets.com is the world's leading source for international market research reports and market data. We provide you with the latest data on international and regional markets, key industries, the top companies, new products and the latest trends.

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Cell Harvesting Market 2019 with Detailed Profiles of Key Companies Including General Electric, Perkinelmer and Thermo Fisher - ResearchAndMarkets.com...

Bone Marrow Stem Cells Comments Off on Cell Harvesting Market 2019 with Detailed Profiles of Key Companies Including General Electric, Perkinelmer and Thermo Fisher – ResearchAndMarkets.com… | October 14th, 2019

Jennifer Adair, a senior scientist at Fred Hutch, speaks at the 2019 GeekWire Summit. (GeekWire Photo / Kevin Lisota)

Genetically editing cells using CRISPR could be the answer to curing genetic disorders such as sickle cell anemia. But in order for the technology to be available for people in countries like Nigeria where around a quarter of the population carries the sickle cell trait the technology will need to become substantially cheaper and less invasive.

Thats where gold nanoparticles come in.

Scientists at the Fred Hutchinson Cancer Research Center are devising an approach that vastly simplifies how CRISPR is applied. Their goal is to create a safe process for gene editing that takes place entirely within the body of a patient.

In order to edit human stem cells using CRISPR today, scientists have to follow a process that involves removing the cells from a patients bone marrow, electrocuting those cells, and modifying them with engineered virus particles.

The process gets even more invasive from there. We actually have to treat these patients with chemotherapy, radiation or other agents in order for these cells that were genetically manipulated to be taken up, Jennifer Adair, a senior scientist at Fred Hutch, said during a talk at the 2019 GeekWire Summit.

The researchers think theyve figured out the first step, which is delivering CRISPR to blood stem cells inside the body. Theyre doing that using gold nanoparticles that are about a billionth the size of a grain of table salt and able to smuggle in RNA, DNA and a protein.

Weve been able to show that not only can we make these, but they passively deliver all of those components to blood stem cells, then we do get genetic editing. And weve been able to go on to show that we can correct the sickle cell defect using this approach, said Adair.

The nanoparticles are big enough to carry the CRISPR payload but small enough to infiltrate cell membranes. Gold is a useful medium since it isnt harmful to humans.

The Fred Hutch team published their work with gold nanoparticles earlier this year in the journal Nature Materials. The system safely edited 10 to 20 percent of the target cells, which the researchers hope will increase as the method is refined.

In an ideal world, clinicians would be able to deliver gene therapy through a syringe, a process that might be accomplished in a single office visit. Adair previously published research on agene therapy in a box concept, a table-top device that could provide gene therapy treatments without the need for expensive medical infrastructure.

We need to develop technologies that make gene editing simpler, more affordable and more accessible to patients around the world, Adair said.

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Fred Hutch scientist on how gold nanoparticles could bring CRISPR to the developing world - GeekWire

Bone Marrow Stem Cells Comments Off on Fred Hutch scientist on how gold nanoparticles could bring CRISPR to the developing world – GeekWire | October 13th, 2019

Gaucher disease can predispose patients to rare types of blood cancer, and doctors should be vigilant for the development of these malignancies, a case report suggests.

The study, A case of bony lytic lesions in a patient with Gaucher disease, was published in the journal Clinical Case Reports.

The most common symptoms of Gaucher disease are reduced platelet count, enlarged liver and spleen, and lesions in the bones. This disease also has been associated with an increased risk ofblood disorders.

Researchers in Canada reported the case of a 57-year-old man who developed a rare type of blood cancer cell probably related to Gaucher disease, as he had Gaucher cells cells that accumulate abnormal amounts of a fat molecule (glucocerebroside) characteristic of the condition in his bone marrow.

The man had a scalp lesion that did not heal and progressively increased in size. Doctors performed a biopsy and discovered it was caused by a plasmacytoma, a rare form of blood cancer in which myeloma cells form a tumor in the bones or soft tissues.

At the time of biopsy, the patient had mild anemia, low levels of platelets, and a family of blood proteins called gamma globulins characteristic of myeloma. His kidney function and calcium levels were normal.

Further tests showed that the man had several bony lytic lesions spots of bone damage caused by cancerous myeloma cells and abone marrow biopsy showed infiltration of both plasma cells and Gaucher cells. That led to a diagnosis of plasma cell myeloma probably associated with Gaucher disease.

Gaucher cells infiltrating the bone marrow may mask the extent of abnormal plasma cell infiltrates, and immunohistochemical staining [a method that identifies abnormal cells in biopsies] can be invaluable in identifying the true burden of plasma cells for appropriate classification of suspected plasma cell neoplasia, the researchers said.

The investigators also noted that the man had a history of abnormal spleen size and reduced blood cell count. A bone marrow biopsy performed years earlier showed the presence of possible Gaucher cells.

Gaucher disease should be considered in the differential diagnosis of unexplained hepatomegaly [abnormal liver size], splenomegaly [abnormal spleen size], or cytopenias [reduced blood cell count], the investigators said. They added that further research of the previous symptoms might have allowed diagnosing Gaucher disease before the cancer appeared.

The patient received a combination of chemotherapy, cyclophosphamide, and Velcade (bortezomib), followed by high doses of melphalan and autologous stem cell transplantation. He tolerated the transplant well and was discharged with the recommendation of long-term follow-up.

Alejandra has a PhD in Genetics from So Paulo State University (UNESP) and is currently working as a scientific writer, editor, and translator. As a writer for BioNews, she is fulfilling her passion for making scientific data easily available and understandable to the general public. Aside from her work with BioNews, she also works as a language editor for non-English speaking authors and is an author of science books for kids.

Total Posts: 20

Ins Martins holds a BSc in Cell and Molecular Biology from Universidade Nova de Lisboa and is currently finishing her PhD in Biomedical Sciences at Universidade de Lisboa. Her work has been focused on blood vessels and their role in both hematopoiesis and cancer development.

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Gaucher Might Be LInked to Rare Types of Blood Cancer, Report Suggests - Gaucher Disease News

Bone Marrow Stem Cells Comments Off on Gaucher Might Be LInked to Rare Types of Blood Cancer, Report Suggests – Gaucher Disease News | October 13th, 2019

Blood, of course, plays a crucial role in keeping our bodies alive and functioning.

Red blood cells carry oxygen from our lungs to our muscles. White blood cells are the first responders of our immune systems, detecting infections and foreign agents and triggering the immune response needed to deal with the problem. Plasma, the liquid part of blood, transports not only the cells but also proteins, such as antibodies, and hormones, such as insulin, to every part of the body. It is a beautifully complex system that is the key to our bodies functioning correctly.

Parasites, viruses and bacteria all use the circulatory system to spread around the body. When cancer metastasises and spreads to other parts of the body, it is through tumour cells circulating within the bloodstream. There are also myriad blood cancers, such as leukaemias and lymphomas, and blood disorders, such as sickle cell anaemia, not to mention autoimmune diseases like diabetes or lupus. Sepsis, also known as blood poisoning, is a deadly overreaction to an infection which also has its roots in blood.

Magnetic blood filtration is a tool which enables the physical removal of specific substances from the bloodstream

Most of these diseases are treated with drugs or chemotherapies, some with great success, others, much less. There are also various methods of physically extracting different components from the blood by circulating a patients blood outside of the body through whats known as an extracorporeal circuit. Dialysis, for example, removes excess toxins from the blood, acting as a substitute for kidneys when they fail. Plasmapheresis and leukapheresis are methods of removing harmful antibodies from the plasma or white blood cells from the blood. Similar techniques are also used to harvest stem cells from the blood, which can then be used in cancer therapies (known as stem cell transplantation), if the donor and patient are a match.?

Magnetic blood filtration

Despite these methods, millions of people still die every year from blood-borne diseases. At MediSieve a small, London-based start-up company we are using nanotechnology to develop a new technology which we think can transform our ability to tackle these conditions. Magnetic blood filtration (MBF) is a tool which enables the physical removal of specific substances from the bloodstream. It is similar to other extracorporeal procedures like dialysis, but instead of removing every component of a particular size or weight, MBF removes highly specific targets to address the specific medical issue, thereby removing only the substance that doctors want to remove. Alongside this high specificity, various targets, both big and small, can be removed simultaneously, raising the prospect of removing for example specific cells and harmful antibodies in a single procedure.

To achieve this, we use magnetic nanoparticles coated with binding moieties, such as antibodies, which bind specifically to the desired targets in the blood. These particles are infused into the blood within the extracorporeal circuit, binding to their targets. The blood then flows through a magnetic filter which captures the magnetic particles and the targets bound to them, while the rest of the blood flows back into the patient. Several different particles can be used in the same procedure in order to capture different components. Using this method, anything for which there is a specific antibody or other binding moiety can theoretically be removed directly from the bloodstream. I think that in the long-term the technology could be used to remove specific cells, antibodies, bacteria, viruses, toxins, drug molecules and inflammatory cytokines, the drivers of immune conditions such as sepsis.

The MediSieve Filter is a disposable, single-use device in which the magnetic particles and their targets are captured. It is inserted into the MediSieve Magnet, a reusable medical device which activates the filter. Both can be incorporated into a variety of existing extracorporeal systems and integrate with standard blood pumps and tubing sets.

We are currently developing treatments for malaria, sepsis and leukaemia. The Filter and Magnet have completed pre-clinical testing and are now ready for clinical trials, which we hope to start shortly. Magnetic particles for various clinical targets are currently being developed and validated in the laboratory, with promising results. Animal trials for these are expected to start in 2020.

Malaria

Our potential treatment for malaria is the closest to market because malaria infected cells, uniquely, have naturally occurring magnetic properties is it therefore possible to remove them from the blood using the MediSieve Filter without the infusion of any magnetic particles. The magnetic properties arise from a core aspect of the malaria parasites lifecycle. After infecting a red blood cell, the parasite consumes the protein part of haemoglobin, leaving behind an iron-based waste-product known as haemozoin, which is stored inside the cell. Haemozoin is paramagnetic, thereby giving infected cells their unique magnetic properties.

MBF could be used in highly severe malaria cases in which the patient is hospitalised and at high risk of death. Currently, these patients receive intravenous drugs such as artesunate which can achieve parasite clearance in 36-48 hours; parasite clearance rate is the key indicator of patient recovery, and it can take up to eight doses of IV drugs to achieve complete clearance. Mortality in these cases can be as high as 20%.?

Using MBF alongside the first dose of IV drugs could drastically accelerate parasite clearance rate. We claim that, depending on the patient size and initial level of infection, this approach can remove over 90% of red blood cells containing haemozoin in just two hours. Because they have higher quantities of haemozoin, MBF is better at removing later stage infected cells, whereas drugs are much more effective against earlier stage cells, so they should be complimentary.

MBF has the additional benefit of removing free circulating haemozoin, also known as the malaria toxin, which should also improve the treatment for the patient since drugs can cause the large-scale release of haemozoin as infected cells die.

According to the WHO, in 2017 there were 219 million cases of malaria and 435,000 deaths, mostly children. While overall malaria cases and deaths have been trending downwards in recent years, the number of hospitalised patients is increasing as healthcare infrastructure improves in malaria endemic countries and more patients gain access to hospitals. In the future, MBF could be adapted for use in mobile clinics to reach harder to access areas.

While ourinitial target is severe malaria patients, I also believe MBF could be a valuable tool in the fight against drug-resistant malaria strains, which have been emerging in SE Asia and are causing great concern if drug resistance spreads to Africa, the effect could be catastrophic. It can also be used to treat patients for whom drugs cannot be used, such as pregnant women.

Sepsis

Sepsis is one of the leading causes of death in the developed world with more than 1.9M cases in Europe and the US and published mortality rates of 29% - 50%. Sepsis is a complex syndrome in which bacteria or other pathogens create a dysregulated immune response which can escalate to organ failure and death. The immune response creates an overproduction of pro-inflammatory cytokines, while cell damage over time creates damage-associated molecular patterns (DAMPs) that sustain the syndrome. ?

Our approach to sepsis, which we call SepSieve, uses a cocktail of different particles to remove a number of targets from a patients bloodstream: specific pro-inflammatory cytokines (IL-1, IL-6 and IL-18), DAMPs (HMGB-1), endotoxins (LPS), and gram-negative bacteria. This multi-modal approach tackles the disease from two key angles: Removing the pathogens and endotoxins that trigger the immune response and reducing magnitude of the immune response and preventing the cascade towards septic shock.

Like in malaria, SepSieve would be used alongside existing frontline treatments, specifically antibiotics. While antibiotics are critical for treatment of sepsis, the bacterial cell death they cause releases LPS which accelerates the dysregulated immune response MBF could remove the LPS to prevent the condition from worsening. The main benefit of MBF in sepsis is therefore not so-much the removal of bacteria itself (which is tackled by antibiotics and in any case is not present exclusively in the bloodstream), but rather the removal of all the other components driving the disease.

Gram-negative bacteria such as E. coli account for approximately 50% of sepsis patients, but thanks to the removal of other substances, particularly HMGB-1 and the inflammatory cytokines, I think the combined approach could benefit all sepsis patients. Since magnetic filtration is a purely physical method, it can also target and remove pathogens which are resistant to antibiotics, which again are a huge concern with increasing occurrences of resistant infections in hospitals.

Like in malaria, wwe plan to apply sepsis treatment to hospitalised patients and specifically those in Intensive Care Units. These are the most severe cases and those who stand to benefit the most from the treatment. The idea is to intervene early to prevent the sepsis cascade, in which the disease escalates eventually causing organ failure and death.

In fact, we managed to secure grants worth a total of 1.56M from Innovate UK, the UKs government grant funding body, and the UK National Institute of Health Research to develop and validate our sepsis particles. Currently being tested in human blood models in the companys laboratories, we plan to start animal trials in 2020 which, if successful, will be followed by clinical trials in 2021.

Leukaemia

One of the advantages of the particles we develop to remove pro-inflammatory cytokines for sepsis is that they can also be used in other diseases. This includes auto-immune diseases and cytokine storms such as cytokine release syndrome (CRS), a common side-effect of newer leukaemia treatments known as CAR T-cell therapies.?

In CAR-T therapies, T-cells, a type of white blood cell, are modified to attack cancer cells in a patients bone marrow. Taken either directly from the patient or from a matching donor, the modified cells are infused into the patient in order to directly attack the cancer. Results of clinical trials have been mixed, but these cell therapies are seen as a huge leap forward for leukaemia treatment.?

The problem is that the infused T-cells trigger massive immune reactions within the patient. Indeed, that is the intention the immune reaction is intended to kill the cancer cells but it can easily escalate into the condition called CRS. The result is similar to sepsis an immune over-reaction which attacks the patient and can be fatal. Immune mediators can be used to calm this reaction, but they then prevent the infused CAR-T cells from having their effect, eliminating the therapeutic benefit of the treatment.

Our proposal is to use MBF in CRS patients to remove cytokines from the bloodstream. This should calm the immune reaction, alleviating patient suffering and eliminating the risk of death. But since MBF only removes cytokines from the bloodstream, it shouldnt affect the immune effect of the CAR-T cells in the bone marrow, so the therapeutic benefit should be maintained. In addition, MBF can be stopped at will, so it can be used to control the immune response by maintaining the correct balance of cytokines this is of course not possible with immune mediators which are infused into the patient.

A further benefit that MBF can provide in leukaemia patients is the removal of leukaemia cells from the bloodstream leukaemia patients commonly have very high white blood cell counts due to circulating leukaemia cells. These cause a number of issues such as a reduction in immune function, making patients more vulnerable to infection. They can also prevent certain chemotherapies from working effectively, since they block the drug from targeting cancer cells in the bone marrow. High white blood cell counts also increase the risk of side-effects during treatment, since the sudden death of such a large numbers of cells causes debris to circulate in the blood, putting strain on the body and causing immune reactions like CRS; this is known as Tumour Lysis Syndrome. ?

We are currently focussing development on their sepsis particles, but plan to trial their cytokine particles in CRS at the same time as they are trialled in sepsis, since the pre-clinical validation for each disease is the same. The particles to remove white blood cells, however, are at an earlier stage and will be developed further down the line.

Our ambitions for MBF are certainly large. In the long-term we want to revolutionise the way in which blood-borne diseases are treated. Going far beyond malaria, sepsis and leukaemia, we want to develop treatments for all blood-borne diseases if its in the blood, and doctors want it out, we want to be able to take it out.

My vision is that hospitals all around the world will have Magnetic Blood Filtration Units which will address a huge variety of patients. Only time will tell if this can be achieved, or even if our technology will work at all after all, there have, as of yet, been no clinical trials.

However, the ability to remove specific substances from blood would clearly be of benefit to huge numbers of patients. It is something that we cannot do today, but we certainly should want to be able to do tomorrow. Whether it is MediSieve who gets us there or not remains to be seen.

Author:

Dr George Frodsham is CEO and founder of MediSieve

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It's in the blood - Lab News

Bone Marrow Stem Cells Comments Off on It’s in the blood – Lab News | October 13th, 2019

This article, written byKatharine Sedivy-Haley, University of British Columbia, originally appeared on The Conversation and is republished here with permission:

When I was applying to graduate school in 2012, it felt like stem cells were about to revolutionize medicine.

Stem cells have the ability to renew themselves, and mature into specialized cells like heart or brain cells. This allows them to multiply and repair damage.

If stem cell genes are edited to fix defects causing diseases like anemia or immune deficiency, healthy cells can theoretically be reintroduced into a patient, thereby eliminating or preventing a disease. If these stem cells are taken or made from the patient themselves, they are a perfect genetic match for that individual, which means their body will not reject the tissue transplant.

Because of this potential, I was excited that my PhD project at the University of British Columbia gave me the opportunity to work with stem cells.

However, stem cell hype has led some to pay thousands of dollars on advertised stem cell treatments that promise to cure ailments from arthritis to Parkinsons disease. These treatments often dont help and may harm patients.

Despite the potential for stem cells to improve medicine, there are many challenges as they move from lab to clinic. In general, stem cell treatment requires we have a good understanding of stem cell types and how they mature. We also need stem cell culturing methods that will reliably produce large quantities of pure cells. And we need to figure out the correct cell dose and deliver it to the right part of the body.

Embryonic, 'induced and pluripotent

Stem cells come in multiple types. Embryonic stem cells come from embryos which makes them controversial to obtain.

A newly discovered stem cell type is the induced pluripotent stem cell. These cells are created by collecting adult cells, such as skin cells, and reprogramming them by inserting control genes which activate or induce a state similar to embryonic stem cells. This embryo-like state of having the versatile potential to turn into any adult cell type, is called being pluripotent.

However, induced pluripotent and embryonic stem cells can form tumours. Induced pluripotent stem cells carry a particularly high risk of harmful mutation and cancer because of their genetic instability and changes introduced during reprogramming.

Genetic damage could be avoided by using younger tissues such as umbilical cord blood, avoiding tissues that might contain pre-existing mutations (like sun-damaged skin cells), and using better methods for reprogramming.

Stem cells used to test drugs

For now, safety concerns mean pluripotent cells have barely made it to the clinic, but they have been used to test drugs.

For drug research, it is valuable yet often difficult to get research samples with specific disease-causing mutations; for example, brain cells from people with amyotrophic lateral sclerosis (ALS).

Researchers can, however, take a skin cell sample from a patient, create an induced pluripotent stem-cell line with their mutation and then make neurons out of those stem cells. This provides a renewable source of cells affected by the disease.

This approach could also be used for personalized medicine, testing how a particular patient will respond to different drugs for conditions like heart disease.

Vision loss from fat stem cells

Stem cells can also be found in adults. While embryonic stem cells can turn into any cell in the body, aside from rare newly discovered exceptions, adult stem cells mostly turn into a subset of mature adult cells.

For example, hematopoietic stem cells in blood and bone marrow can turn into any blood cell and are widely used in treating certain cancers and blood disorders.

A major challenge with adult stem cells is getting the right kind of stem cell in useful quantities. This is particularly difficult with eye and nerve cells. Most research is done with accessible stem cell types, like stem cells from fat.

Fat stem cells are also used in stem cell clinics without proper oversight or safety testing. Three patients experienced severe vision loss after having these cells injected into their eyes. There is little evidence that fat stem cells can turn into retinal cells.

Clinical complications

Currently, stem cell based treatments are still mostly experimental, and while some results are encouraging, several clinical trials have failed.

In the brain, despite progress in developing treatment for genetic disorders and spinal cord injury, treatments for stroke have been unsuccessful. Results might depend on method of stem cell delivery, timing of treatment and age and health of the patient. Frustratingly, older and sicker tissues may be more resistant to treatment.

For eye conditions, a treatment using adult stem cells to treat corneal injuries has recently been approved. A treatment for macular degeneration using cells derived from induced pluripotent stem cells is in progress, though it had to be redesigned due to concerns about cancer-causing mutations.

A path of cautious optimism

While scientists have good reason to be interested in stem cells, miracle cures are not right around the corner. There are many questions about how to implement treatments to provide benefit safely.

In some cases, advertised stem cell treatments may not actually use stem cells. Recent research suggests mesenchymal stem cells, which are commonly isolated from fat, are really a mixture of cells. These cells have regenerative properties, but may or may not include actual stem cells. Calling something a stem cell treatment is great marketing, but without regulation patients dont know what theyre getting.

Members of the public (and grad students) are advised to moderate their excitement in favour of cautious optimism.

Katharine Sedivy-Haley, PhD Candidate in Microbiology and Immunology, University of British Columbia

This article is republished from The Conversation under a Creative Commons license. Read the original article.

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BEYOND LOCAL: Expert recommends 'path of cautious optimism' about the future of stem cell treatment - CollingwoodToday

Bone Marrow Stem Cells Comments Off on BEYOND LOCAL: Expert recommends ‘path of cautious optimism’ about the future of stem cell treatment – CollingwoodToday | October 12th, 2019

Stem cell therapy, which All Blacks prop Owen Franks used to help fix a damaged shoulder, is raising hopes of a whole range of medical breakthroughs.

But there's a way to go before the medical establishment is convinced.

In late 2017, US Food and Drug Administration (FDA) Commissioner ScottGottliebhad this to say:"We're at the beginning of a paradigm change in medicine with the promise of being able to facilitate regeneration of parts of the human body, where cells and tissues can be engineered to grow healthy, functional organs to replace diseased ones; new genes can be introduced into the body to combat disease; and adult stem cells can generate replacements for cells that are lost to injury or disease."

REGEN CELLULAR

Dr Hassan Mubark takes blood from All Blacks prop Owen Franks.

Yet, as an indication of how far there is still to go, the FDA has also warnedpeople in the USagainst "unscrupulous providers" offering stem cell products that were unapproved and unproven.

READ MORE:*Rugby World Cup 2019: All Black Owen Franks thrown a stem cell lifeline*Owen Franks hits back at critics following omission from Rugby World Cup squad*Stem cell therapy for All Black Israel Dagg as he hits comeback trail with Crusaders*Experimental stem cell treatment shows results for Waikato woman with MSA Cerebella*Stem cell clinics accused of taking advantage of patients*Reported stem cell treatment could give hope to Michael Schumacher

"Researchers hope stem cells will one day be effective in the treatment of many medical conditions and diseases," it said, thenadded: "Stem cells have been called everything from cure-alls to miracle treatments. But don't believe the hype."

Looking at just the area of deteriorating joints, it's easy to see how stem cell therapies, if they deliver on the promise,could make life much better for many people with osteoarthritis who are in pain and have restricted movement.

Last week, Otago University researchers predictedthe number of knee replacement surgeries needed for osteoarthritis would increase from around 5000 a year in 2013 to abut9000 in 2038.

AP

Former Formula One champion Michael Schumacher received devastating head injuries in a ski accident six years ago. Last month it was reported he has undergone stem cell treatment in Paris.

Osteoarthritis is the area where ReGen Cellular,the clinic where Franks had the therapy, has done most of its work in the past two to three years, although ithas recently expanded its services to include a range of diagnosed auto-immune conditions, among them rheumatoid arthritis, multiple sclerosis, and type 1 diabetes.

ReGensaid 55 per cent of its patients were aged over 60, 35 per cent were 40-60 and 10 per cent were sports-based.

Theclinic usesPure Expanded Stem Cell (PESC) therapy, which involves taking 40 grams - about a teaspoon - of fat from around a patient's stomach. Mesenchymal stem cells (MSCs)in that sample are then multiplied in the clinic's Queenstown laboratory for about eight weeks. At the end of that process 100 million to 200 million cells have been produced.

Otago University

Otago University, Christchurch regenerative medicine research team have invented a bio-ink - a gel-like substance mixed with human stem cells - to be used with a bio-printer to make human body parts. Video shows the printer using bio-ink to make a body part.

For the treatment of osteoarthritis, between 50m and 100m stem cells are injected into larger joints, with 25m to 50m into smaller joints. ReGen said the therapy provided immediate pain reduction and increased mobility. MRI scans showed cartilage could and did regenerate.

ReGendescribedMSCs as the cells that "wake up damaged or lazy cells". Slightly more technically, Nature.com said MSCs wereadult stem cells present in multiple tissues, including the umbilical cord, bone marrow and fat.MSCscan self-renew by dividing and can differentiate into multiple tissues including bone, cartilage, muscle and fat cells, and connective tissue.

ReGen director of patient care Marcelle Noble said the clinic believed its treatments, if offered early enough, would save the public health system hundreds of millions of dollars through lessened replacement surgeries, and would save ACC millions of dollars in lengthy rehabilitation programmes.

The treatment for two knees was half the price of one knee replacement surgery within the public health system, she said. ReGen advertises osteoarthritis treatment for a single joint at $12,500 and for two joints at $15,000.

GETTY IMAGES

Former All Black Israel Dagg had stem cell therapy for an injured knee, but in the end had to give the game away because of the injury.

So far mainstream funding hadnot been offered for the therapy, Noble said. But the clinic had a "big breakthrough" earlier this year when two insurers in New Zealand accepted patients'PESC therapy claims. In July, ACC accepted consultation by ReGen's chief medical officer Dr Hassan Mubark.

ReGen only had data for the past five years on the success of its therapy, but the fact patients were returning to have other areas of their body treated was an indication of how people feltthe therapy was improving their quality of life, Noble said.

Globally, "massive" R&D spending was going into stem cell research. More therapies would become available and stem cell treatment would become "commonplace".

At any one time ReGen had 50-75 patients' cells growing in its incubators, Noble said. Of the patients treated, 40 per cent hadailments in therknees, 30 per cent in their hips, 20 per cent in their shoulders. The final 10 per cent were for sports and other issues, including problems with tendons, muscles, cartilage tears, fingers, elbows, ankles and hands.

SUPPLIED

Dr Ron Lopert undergoing part of the PESC treatment.

The first patient to undertake ReGen's PESC therapy was retired GP Dr Ron Lopert, who lives in Tauranga.

For five to 10 years, he had beengetting aches and pains in his hips after playing sport, and the problem was becoming more noticeable, he said. In 2013 he had an x-ray that showed he had moderate to severe osteoarthritis in both hips,more severein his right hip.

He stopped playing all sports and started researching different forms of treatment. Ideally, he wanted to be able to get some of his own cartilage back and reverse the osteoarthritis. It seemedPESCshould do that.

In 2015, aged 61, he had the therapy, with stem cells being injected into each hip joint.Within weeks henoticed an improvement in the range of motion and a decrease in pain, Lopert said.Some of that was just the anti-inflammatory component of stem cell injection, but he thought he also received a longer term benefit from cartilage regeneration.

SUPPLIED

Dr Lopert on his recent travels. He says he has much less hip pain.

He put the success of the procedure at75 per centin terms of symptoms and function, and100 per cent when it came to avoiding invasive surgery."I opted for a much more natural treatment where my own tissue is regenerating, instead of a metal prosthesis," Lopert said.

He was not sure all the improvement came from the stem cell treatment. As well as avoiding overuse of the joints, which meant he hadn't returned to playing sport, he had also switched to an anti-inflammatory diet.

His left hip continued to have hardly any symptomsbut he had started noticing the "odd twinge now and then" in his right hip.

"The vast majority of days it's fine provided I'm just walking and doing ordinary things. On the odd occasion I might carry something heavy, then I would notice it the next day and it (right hip) would stay painfulintermittentlyfor the next couple of days," Lopert said.

Sean Gallup

In this picture from February, German Chancellor Angela Merkel looks through a microscope at brain organoids grown from stem cells.

Some of his stem cells had been retained after the treatment, and he was booked in for a follow-up injection for his right hip at the end of October.

He expected the therapy would become a "go to" treatment, and would become an early intervention for osteoarthritis. But more independent research was needed to confirm the success of the treatment. "The evidence is slowly building up but there needs to be more before the Government will accept it," Lopert said.

In his case, he thought there had been cartilage regeneration in his hips, but that was based on his symptoms. "It would have been nice had I had MRI scans before and after the injection for objective evidence," he said.

From the perspective of the medical establishment, the New Zealand Orthopaedic Association said it supported a position statement on stem cell therapy produced by the Royal Australian College of Surgeons.

That paper, approved in mid-2018,noted stem cell therapy was a "rapidly advancing" area, but many proposed stem cell therapies were experimental and not yet proven. It did not support surgeons administering stem cell therapy outside of an ethically approved registered clinical trial.

"Whilst there may be scope for innovative treatment in the future, currently, the clinical effectiveness and safety of stem cell therapies remain scientifically unproven," RACS said.

In this country, an ACC spokesperson said ACC did not have an official position on stem cell therapy for the treatment of injuries. An internationally standardised evidence-based healthcare approach was used to help ACC decide how it covered injuries and funded treatments.

Dr HassanMubark, ReGen's chief medical officer, was a healthcare provider contracted to ACC in the specialty of rheumatology, and ACC had funded consultation fees with Mubark, the spokesperson said. Those consultations were for diagnostic and treatment planning purposes and did not need prior approval from ACC.

ACC had to consider legislative criteria when deciding whether to fund any particular treatment. There would be many reasons why ACC might decide to fund a client to see a rheumatologist for an opinion on the diagnosis and possible management of their condition. That would not commit ACC to funding any proposed treatment but would provide the client and ACC with information to help decision-making.

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Stem cell therapy helped Owen Franks but there's still plenty to prove - Stuff.co.nz

Bone Marrow Stem Cells Comments Off on Stem cell therapy helped Owen Franks but there’s still plenty to prove – Stuff.co.nz | October 12th, 2019

By JLNJ Staff | October 10, 2019

(Courtesy of Ezer Mizion) Flying 35,000 feet above the Atlantic Ocean is not an easy job! But Ofer had already spent 17 years as a fighter pilot in the IDF defending the State of Israel. In 2003 he left the reserves and joined El-Al full time. Most people dont realize that being a pilot is a very dangerous profession. When you know it is dangerous you are safe but when you think it is easy, when youre a cowboy, you are unsafe! A pilots job is to always be alert in case something happens. Ofer always remained alert with hundreds of travelers under his wing, quite literally!

But after 16 years of flying for El-Al, Ofer started to feel fatigued. It became difficult for me to walk up with steps to the plane from the tarmac. I thought I was starting to get old or out of shape. But the truth was far more devastating: after routine blood tests, Ofer was diagnosed with leukemia!

I was immediately rushed to the hospital. When I arrived they couldnt even find bone marrow inside my body for a biopsy. I had very little bone marrow left in my body.

Ofer started to think about his future. He thought, Will I ever be able to fly again? Will I be able to see my children again? Will I get to meet my grandchildren?

It was a very difficult time in my life. I was very lucky to have the best doctors in Israel. Shortly after Jan 1, 2017, Ofer was told that Ezer Mizion had a perfect bone marrow match for him! He was thrilled, but still very hesitant. I knew I was not yet out of the woods. I was on a new medication and I was starting to feel better. I did not know if I wanted to risk a transplant with possible complications. Ofer decided to take a vacation to Moscow. He had always traveled the world and Moscow was one place he had never visited but had always wanted to see. The doctors told me if I get even a small virus I can forget about the whole transplant. I put my faith in God and said, if it is meant to be, then I will return and have the transplant.

On Feb. 28, Ofer landed back in Tel Aviv, and March 1 started his preparations for a transplant.

Pushing through all the negative thoughts, Ofer decided to fight. He was absolutely determined to overcome this illness and would go to any lengths to get better.

A short six weeks later Ofer was released from the hospital and returned to his family.

David Bugoslavski was in the middle of his military service on Mt. Hermon when he received a call from Ezer Mizion that he is a perfect match for a cancer patient. Ironically, David wasnt supposed to have his phone on him while he was in the middle of active duty. Yet, as he explains, fate thought otherwise. He knew that Ezer Mizion needed him, and while he did not know Ofer personally, he jumped at the opportunity to save the pilots life.

Thanks to Davids transplant, Ofer is alive today. While the recovery process is slow and there has been some turbulence along the way, Ofer has his life back. One of Ofers dreams had always been to fly a Boeing Dreamliner. Unfortunately, due to his medical history, this dream will never come to fruition in his capacity as a pilot but he still loves to travel the world, even if hes sitting in the back of the plane.

David was able to jump on a once-in-a-lifetime opportunity to save a life. Ofer was able to be the recipient of a special and unique kindness, having his life literally saved by someone else. As Ofer explained so beautifully, David: without you, I wouldnt be here... For me, you are part of the family.

Ezer Mizions bone marrow registry has close to 1 million registrants, with over 550,000 of them IDF soldiers. At Ezer Mizion, no matter who you are or where you come from, your life matters. Ofer and David are just one example of the lifesaving mission of Ezer Mizion taking flight. At Ezer Mizion, unconditional love is not just a term thrown around, but a philosophy that is in the very DNA of the organization. As Dr. Bracha Zisser, director and founder of Ezer Mizions National Bone Marrow Registry says, We have created a true connection of blood between two people who did not know each other at all up to that point. A connection that would not have happened without the unconditional immediate enlistment of David or, as Ofer called him, my angel.

Join Ezer Mizion on November 9 at Congregation Keter Torah in Teaneck at 7:30 p.m. for an Evening of Heroes: a beautiful musical Havdalah by Shulem Lemmer, meet real IDF heroes who have saved lives by donating their stem cells, and a fireside chat with Bret Stephens and Nachum Segal. Learn more about Ezer Mizion and RSVP for the Evening of Hereos by going to http://www.eveningofheroes.com, or contact Ryan Hyman, national director of development, at [emailprotected] or 718-853-8400 ext.109.

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The Connection Deeper Than Blood - Jewish Link of New Jersey

Bone Marrow Stem Cells Comments Off on The Connection Deeper Than Blood – Jewish Link of New Jersey | October 12th, 2019

PENSACOLA, Fla. (WKRG) Osteoarthritis affects millions of people in the US. Symptoms range from minor pain to crippling pain that compromises quality of life. A groundbreaking study is underway at four prestigious research facilities in the United States. One of those is right here on the Gulf Coast. Tonight, Drexel Gilbert is on the road in Gulf Breeze.

Lori Jamison is a Pensacola native who, as a teenager, played basketball at Pine Forest High School. Today, she suffers from osteoarthritis in her knee. She believes its a result of basketball injuries.

I get stiffness, it interferes with my mobility. Sometimes its like a sharp needle going down your leg. When I go to the movie theater, I have to sit on the back row so I can stretch it out, Jamison said. She is participating in a clinical trial at Andrews Research and Education Foundation in Gulf Breeze.

The research is studying stem cell treatment for osteoarthritis in the knee. AREF is one of only four facilities in the country participating in the study. The others are Emory Orthopedics & Spine Center, Duke University and Sanford Health. Researchers hope it leads to FDA approval for the treatment. If that happens, it could be life-changing for patients.

Hopefully reduce their pain if not actually get rid of their pain. That is our goal. We want to delay, if not prevent, total knee replacement, said Dr. Josh Hackel, who is the primary investigator for the Andrews phase of the study. Were comparing three different stem cell sources. Bone marrow from their pelvis, adipose- thats tissue from their belly fat- and the third is umbilical cord tissue donated from pregnant mothers.

The bone marrow and belly fat stem cells are harvested from the study participants, under local anesthesia. The stem cells are later implanted into the knee joint using ultrasound guidance to implant the cells into the knee joint.

Jamison has already undergone stem cell harvesting.

It was very easy, very convenient, no downtime after the procedure was done, Jamison said

This $13 million clinical trial is being funded entirely by a grant from Bernie Marcus, founder of the Marcus Foundation and co-founder of Home Depot. Osteoarthritis is an issue that is close to the philanthropists heart because his mother was left disabled by the illness at a young age.

There will be around 120 participants at each of the four sites. There are plenty of openings. If youd like to be considered for the study, call AREF at 850-916-8591.

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Drexel on the Road: Stem cell study for osteoarthritis - WKRG News 5

Bone Marrow Stem Cells Comments Off on Drexel on the Road: Stem cell study for osteoarthritis – WKRG News 5 | October 12th, 2019

The 2019 Nobel Prize in Medicine awarded for research in cellular responses to oxygen By Benjamin Mateus 10 October 2019

In the course of a lifetime, the human heart will beat more than three billion times. We will have taken more than 670 million breaths before we reach the end of our lives. Yet, these critical events remain unconscious and imperceptible in everyday life, unless we exert ourselves, such as running up several flights of stairs. We quickly tire, stop to take deep breaths and become flushed.

With the deepening comprehension by medical science of how our bodies work, we have come to better understand the fundamental importance of oxygen to life. Every living organism relies on it in one form or another. However, how cells and tissues can monitor and respond to oxygen levels remained difficult to elucidate. It has only been late in the 20th century with advances in cellular biology and scientific instrumentation that these processes have finally been explained.

On Monday, the 2019 Nobel Prize in Physiology or Medicine was awarded jointly to three individuals: William G. Kaelin, Jr., Sir Peter J. Ratcliffe, and Gregg L. Semenza. Specifically, their discoveries helped elucidate the mechanisms for lifes most basic physiologic processes.

They were able to discover how oxygen levels directly affect cellular metabolism, which ultimately controls physiological functions. More importantly, their findings have significant implications for the treatments of conditions as varied as chronic low blood counts, kidney disease, patients with heart attacks or stroke and cancers. One of the hallmarks of cancer is its ability to generate new blood vessels to help sustain its growth. It also uses these oxygen cellular mechanisms to survive in low oxygen environments.

Dr. William G. Kaelin Jr. is a professor of medicine at Harvard University and the Dana-Farber Cancer Institute. The main focus of his work is on studying how mutations in what are called tumor suppressor genes lead to cancer development. Tumor suppressor genes are special segments of the DNA whose function is to check the integrity of the DNA before allowing a copy of itself to be made and undergo cell division, which prevents cells from propagating errors. Cellular mechanisms are then recruited to fix these errors or drive the cell to destroy itself if the damage is too severe or irreparable.

His interest in a rare genetic disorder called Von Hippel-Lindau disease (VHL) led him to discover that cancer cells that lacked the VHL gene expressed abnormally high levels of hypoxia-regulated genes. The protein called the Hypoxia-Inducible Factor (HIF) complex was first discovered in 1995 by Gregg L. Semenza, a co-recipient of the Nobel Prize. This complex is nearly ubiquitous to all oxygen-breathing species.

The function of the HIF complex in a condition of low oxygen concentration is to keep cells from dividing and growing, placing them in a state of rest. However, it also signals the formation of blood vessels, which is important in wound healing as well as promoting the growth of blood vessels in developing embryos. In cancer cells, the HIF complex helps stimulate a process called angiogenesis, the formation of new blood vessels, which allows the cancer cells to access nutrition and process their metabolic waste, aiding in their growth. When the VHL gene is reintroduced back into the cancer cells, the activity of the hypoxia-regulated genes returns to normal.

Dr. Gregg L. Semenza is the founding director of the vascular program at the Johns Hopkins Institute for Cell Engineering. He completed his residency in pediatrics at Duke University Hospital and followed this with a postdoctoral fellowship at Johns Hopkins. His research in biologic adaptations to low oxygen levels led him to study how the production of erythropoietin (EPO) was controlled by oxygen. EPO is a hormone secreted by our kidneys in response to anemia. The secretion of EPO signals our bone marrow to produce more red blood cells.

His cellular and mouse model studies identified a specific DNA segment located next to the EPO gene that seemed to mediate the production of EPO under conditions of low oxygen concentration. He called this DNA segment HIF.

Sir Peter J. Ratcliffe, a physician and scientist, trained as a nephrologist, was head of the Nuffield Department of Clinical Medicine at the University of Oxford until 2016, when he became Clinical Research Director at the Francis Crick Institute. Through his research on the cellular mechanisms of EPO and its interaction between the kidneys and red cell production, he found that these mechanisms for cellular detection of hypoxia, a state of low oxygen concentration, were also present in several other organs such as the spleen and brain. Virtually all tissues could sense oxygen in their micro-environment, and they could be modified to give them oxygen-sensing capabilities.

Dr. Kaelins findings had shown that the protein made by the VHL gene was somehow involved in controlling the response to low oxygen concentrations. Dr. Ratcliffe and his group made the connection through their discovery that the protein made by the VHL gene physically interacts with HIF complex, marking it for degradation at normal oxygen levels.

In 2001, both groups published similar findings that demonstrated cells under normal oxygen levels will attach a small molecular tag to the HIF complex that allows the VHL protein to recognize and bind HIF, marking it for degradation by enzymes. If the oxygen concentration is low, the HIF complex is protected from destruction. It begins to accumulate in the nucleus where it binds to a specific section of the DNA called hypoxia-regulating genes, which sets into motion the necessary mechanisms to respond to the low oxygen concentration.

The ability to sense oxygen plays a vital role in health and various disease states. Patients who suffer from chronic kidney failure also suffer from severe anemia because their ability to produce EPO is limited. This hormone is necessary for the stem cells in our bone marrow to produce red blood cells. Understanding how cancer cells utilize oxygen-sensing mechanisms has led to a variety of treatments that targets these pathways. The ability to elucidate these mechanisms offers insight into directions scientists and researchers can take to design or create novel treatments.

The WSWS recently published its 75,000th article. Become a monthly donor today and keep up this vital work. It only takes a minute. Thank you.

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The 2019 Nobel Prize in Medicine awarded for research in cellular responses to oxygen - World Socialist Web Site

Bone Marrow Stem Cells Comments Off on The 2019 Nobel Prize in Medicine awarded for research in cellular responses to oxygen – World Socialist Web Site | October 12th, 2019

PENNY Lancaster was reduced to tears as she handed five-year-old Ronnie Musselwhite the Young Hero gong at The Suns Who Cares Wins health awards last night.

Spurs fan Ronnie bravely offered to give his older sister Ebonie a bone marrow transplant last year.

Eight-year-old Ebonie had leukaemia and it was her only hope.

Mum Christine Jenkins, 40, said: Ronnies stem cells worked perfectly. They did what they were supposed to do but the leukaemia came back somewhere new.

Ebonie, of Crawley, West Sussex, nominated her younger brother before her death in June.

Rod Stewarts partner Penny chatted with Ronnie about his love of football and Spurs.

She said: To say I am humbled to be here is an understatement. Sometimes you think life has turned a corner on you, but then someone else turns up to give you some inspiration.

"The courage this little man has shown is absolutely incredible. Hes only five years old, hes lost a sister.

"He was incredibly shy to stand up in front of everyone to collect his award, but he again was so brave.

Christine said: We want Ronnie to know that what he did still worked, was still brave, even though he lost his sister.

1

PM Boris Johnson was also at the awards held at The Suns London HQ near The Shard and paid tribute to our NHS heroes.

He presented an award to a pair of quick-thinking hospital porters who saved the life of a seven-week-old baby boy.

Nick Evans, 48, and Ruth Lowe, 47, sprang into action after Logan Clifford stopped breathing.

His parents, Sarah and Mike were visiting a relative at the Princess Royal Hospital in Telford, Shrops, when they noticed Logans lips had turned blue.

Sarahs screams alerted Ruth, who shouted for Nick. He grabbed Logan and performed CPR as he ran half a mile down the corridor to A&E.

Nick continued CPR until the resuscitation team took over and the porters stayed by Logans parents side until they knew he was going to be OK. Sarah, 30, has called the two porters my heroes.

As he handed the pair the Ultimate Lifesaver trophy, the PM said: The NHS is revered around the world, and in no small part due to the heroes working in it every day.

He added: My experience of the NHS is like everybody else in the NHS - one of admiration and love.

"It is the most extraordinary institution in the world. If our country was an omelette then the NHS is the egg white that holds the great British cake together.

Virgin Radio DJ Chris Evans presented the Best Nurse gong to Liz Monaghan, 53. She set up the widely praised Purple Rose initiative, which aims to improve the care for patients and their loved ones in the last days of their life.

Liz, who works at the Florence Nightingale Hospice, based at the Stoke Mandeville Hospital in Aylesbury, Bucks, said: Im a little embarrassed to have won. Im a small part of a big team.

DJ Chris said: Youve got to prepare yourself for nights like this because otherwise they hit you like an express train.

Who Cares Wins Awards: The winners

BEST HEALTH CHARITY

Winner: Matt Hampson Foundation

Former English rugby union player Matt Hampson set up a charity to help others after being left paralysed in a scrum in 2005.

Other nominees: Superhero Foundation and Team Domenica

BEST NEONATAL SPECIALIST

Winner: Professor Kypros Nicolaides

Professor Nicolaides performed pioneering keyhole surgeon on Sherrie Sharps unborn son Jaxon. By extraordinary coincidence, as a young surgeon, he also operated on Sherries mother when she was in the womb.

Other nominees: Dr Vesna Pavasovic and Professor Massimo Caputo

UNSUNG HERO

Winner: Therapeutic Care Volunteers at South Tees NHS Foundation Trust

30 therapeutic care volunteers, who all have a learning or physical disability, give up their time to support patients with spinal injuries at The James Cook University Hospital in Middlesbrough. They include Ify Nwokoro.

Other nominees: Ben Slack and Rob Allen

GROUNDBREAKING PIONEER

Winner: Guys and St Thomas London Auditory Brainstem Implant (ABI) Service

Leia Armitage, eight, was born with a rare form of deafness and was never expected to speak. But she now can thanks to pioneering brain surgery and speech therapy carried out by Guys and St Thomas London Auditory Brainstem Implant (ABI) Service.

Other nominees: Dr Helen Spencer and Girish Vajramani

BEST DOCTOR

Winner: Dr Matthew Boulter

Dr Boulter served in Afghanistan, teaches wild trauma to army medics and his surgery became the first in Cornwall to be given veteran friendly accreditation.

Other nominees: Margaret France and Dr Bijay Sinha

BEST MIDWIFE

Winner: Jane Parke

Jane helped deliver the youngest surviving twin boys in Britain when they were born at 22 weeks last year. She flew 190 miles with their mum Jennie Powell to a specialist neonatal unit.

Other nominees: Charlotte Day and Nagmeh Teymourian

ULTIMATE LIFESAVER

Winner: Ruth Lowe and Nick Evans

Porters Ruth and Nick saved the life of Sarah and Mike Cliffords seven-week-old baby Logan. He stopped breathing as they walked through the main entrance of The Princess Royal Hospital in Telford to visit a sick relative.

Other nominees: Dr Mark Forrest and Mike Merrett

BEST NURSE

Winner: Liz Monaghan

Liz is the Matron of the Florence Nightingale Hospice in Aylesbury, Bucks, and came up with the idea for the widely praised Purple Rose initiative to improve the care for patients in the last days of their lives.

Other nominees: Margaret Ballard and Carlton DeCosta

MENTAL HEALTH HERO

Winner: Ben West

Ben lost his brother Sam, 15, to suicide last year and since his death, has campaigned tirelessly to raise awareness for mental health.

Other nominees: Beth Gregan and Catherine Benfield

YOUNG HERO

Winner: Ronnie Musselwhite

Ronnie offered to help his sister Ebonie by giving her a bone marrow transplant when she was diagnosed with a rare form of leukaemia. Ebonie nominated her brother for his bravery before she died in June.

Other nominees: Bella Field and Kaitlyn Wright

I only walked ten metres into the room tonight and I already nearly burst into tears three times.

TV star Christine Lampard gave the Best Neonatal Specialist award to Prof Kypros Nicolaides, 66.

He was nominated by Sherrie Sharp, 29, of Horsham, West Sussex, for saving the life of her unborn baby son and her own.

After scans revealed Jaxson had spina bifida, Sherrie was offered a termination. But she contacted Prof Nicolaides, a surgeon at Kings College Hospital, London.

He had saved her life 30 years earlier when she developed a rare blood disorder in her mums womb.

He agreed to perform ground-breaking surgery on Jaxson while he was in Sherries womb.

Prof Nicolaides said: I was delighted to be able to help. Sherrie said: He has saved so many generations of my family. Hes our guardian angel.

The Who Cares Wins Awards were set up in 2017 by The Sun to honour the nations heroic doctors, nurses, midwives, other NHS staff and volunteers.

The Duchess of York presented an award to the parents of Natasha Ednan-Laperouse, 15, who died of an allergic reaction to a sandwich from Pret.

The duchess said: Can I just say to The Sun, I think youre incredible. Every minute Im sitting there and thinking Im so lucky. The NHS, The Sun and all of you, this is what makes Britain so great.

Lorraine Kelly, who presented the awards, said: Earlier on this year my dad was very sick and we honestly thought we were going to lose him.

"It was really difficult and it was only because of the efforts of the NHS hes still here. Its fantastic.

Who Cares Wins Awards: The winners

BEST HEALTH CHARITY

Nominees: Superhero Foundation

Team Domenica

Winner: Matt Hampson Foundation

Former English rugby union player Matt Hampson set up a charity to help others after being left paralysed in a scrum in 2005.

BEST NEONATAL SPECIALIST

Nominees: Dr Vesna Pavasovic

Professor Massimo Caputo

Winner: Professor Kypros Nicolaides

Professor Nicolaides performed pioneering keyhole surgeon on Sherrie Sharps unborn son Jaxon. By extraordinary coincidence, as a young surgeon, he also operated on Sherries mother when she was in the womb.

UNSUNG HERO

Nominees: Ben Slack

Rob Allen

Winner: Therapeutic Care Volunteers at South Tees NHS Foundation Trust

30 therapeutic care volunteers, who all have a learning or physical disability, give up their time to support patients with spinal injuries at The James Cook University Hospital in Middlesbrough. They include Ify Nwokoro.

GROUNDBREAKING PIONEER

Nominees: Dr Helen Spencer

Girish Vajramani

Winner: Guys and St Thomas London Auditory Brainstem Implant (ABI) Service

Leia Armitage, eight, was born with a rare form of deafness and was never expected to speak. But she now can thanks to pioneering brain surgery and speech therapy carried out by Guys and St Thomas London Auditory Brainstem Implant (ABI) Service.

BEST DOCTOR

Nominees: Margaret France

Dr Bijay Sinha

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Penny Lancaster is reduced to tears as she gives bone marrow donor Ronnie Musselwhite, 5, The Suns Young H - The Sun

Bone Marrow Stem Cells Comments Off on Penny Lancaster is reduced to tears as she gives bone marrow donor Ronnie Musselwhite, 5, The Suns Young H – The Sun | October 12th, 2019

NEW YORK, Oct. 07, 2019 (GLOBE NEWSWIRE) -- BrainStorm Cell Therapeutics Inc.(NASDAQ: BCLI), a leading developer of adult stem cell therapeutics for neurodegenerative diseases, today announced that the United States Patent and Trademark Office (USPTO) has issued a Notice of Allowance for BrainStorm's new US Patent Application, number: 15/113,105, titled: Method of Qualifying Cells'.

The allowed claims cover a pharmaceutical composition for MSC-NTF cells secreting neurotrophic factors (NurOwn) comprising a culture medium as a carrier and an isolated population of differentiated bone marrow-derived MSCs that secrete neurotrophic factors.

Patent families protecting NurOwn have previously issued in the United States, Japan, Europe, Hong-Kong and Israel.

"This allowance further expands the patent protection of the NurOwn Cellular Therapeutic Technology Platform and enables us to accelerate clinical development for new neurodegenerative indications, commented BrainStorm President and CEO,Chaim Lebovits.

About NurOwn

NurOwn (autologous MSC-NTF) cells represent a promising investigational therapeutic approach to targeting disease pathways important in neurodegenerative disorders. MSC-NTF cells are produced from autologous, bone marrow-derived mesenchymal stem cells (MSCs) that have been expanded and differentiated ex vivo. MSCs are converted into MSC-NTF cells by growing them under patented conditions that induce the cells to secrete high levels of neurotrophic factors. Autologous MSC-NTF cells can effectively deliver multiple NTFs and immunomodulatory cytokines directly to the site of damage to elicit a desired biological effect and ultimately slow or stabilize disease progression. BrainStorm is currently conducting a Phase 3 pivotal trial of autologous MSC-NTF cells for the treatment of amyotrophic lateral sclerosis (ALS). BrainStorm also recently received U.S. FDA acceptance to initiate a Phase 2 open-label multicenter trial in progressive MS and enrollment began in March 2019.

About BrainStorm Cell Therapeutics Inc.

BrainStorm Cell Therapeutics Inc. is a leading developer of innovative autologous adult stem cell therapeutics for debilitating neurodegenerative diseases. The Company holds the rights to clinical development and commercialization of the NurOwn technology platform used to produce autologous MSC-NTF cells through an exclusive, worldwide licensing agreement. Autologous MSC-NTF cells have received Orphan Drug status designation from the U.S. Food and Drug Administration (U.S. FDA) and the European Medicines Agency (EMA) in ALS. BrainStorm is currently enrolling a Phase 3 pivotal trial in ALS (NCT03280056), investigating repeat-administration of autologous MSC-NTF cells at six sites in the U.S., supported by a grant from the California Institute for Regenerative Medicine (CIRM CLIN2-0989). The pivotal study is intended to support a filing for U.S. FDA approval of autologous MSC-NTF cells in ALS. BrainStorm also recently received U.S. FDA clearance to initiate a Phase 2 open-label multicenter trial in progressive Multiple Sclerosis. The Phase 2 study of autologous MSC-NTF cells in patients with progressive MS (NCT03799718) started enrollment in March 2019. For more information, visit the company's website at http://www.brainstorm-cell.com

Safe-Harbor Statements

Statements in this announcement other than historical data and information constitute "forward-looking statements" and involve risks and uncertainties that could cause BrainStorm Cell Therapeutics Inc.'s actual results to differ materially from those stated or implied by such forward-looking statements. Terms and phrases such as "may", "should", "would", "could", "will", "expect", "likely", "believe", "plan", "estimate", "predict", "potential", and similar terms and phrases are intended to identify these forward-looking statements. The potential risks and uncertainties include, without limitation, risks associated with BrainStorm's limited operating history, history of losses; minimal working capital, dependence on its license to Ramot's technology; ability to adequately protect the technology; dependence on key executives and on its scientific consultants; ability to obtain required regulatory approvals; and other factors detailed in BrainStorm's annual report on Form 10-K and quarterly reports on Form 10-Q available at http://www.sec.gov. These factors should be considered carefully, and readers should not place undue reliance on BrainStorm's forward-looking statements. The forward-looking statements contained in this press release are based on the beliefs, expectations and opinions of management as of the date of this press release. We do not assume any obligation to update forward-looking statements to reflect actual results or assumptions if circumstances or management's beliefs, expectations or opinions should change, unless otherwise required by law. Although we believe that the expectations reflected in the forward-looking statements are reasonable, we cannot guarantee future results, levels of activity, performance or achievements.

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BrainStorm Announces Notice of US Patent Allowance for NurOwn Cellular Therapeutic Technology Platform - Yahoo Finance

Bone Marrow Stem Cells Comments Off on BrainStorm Announces Notice of US Patent Allowance for NurOwn Cellular Therapeutic Technology Platform – Yahoo Finance | October 9th, 2019

A newly designed viral vector the vehicle that delivers a gene therapyto a patients cells for use insickle cell anemia is more efficient than earlier vectors at introducing healthy copies of genes into stem cells and can be produced in greater amounts, studies in animal models show.

The study Development of a forward-orientated therapeutic lentiviral vector for hemoglobin disorders was published in the journal Nature Communications.

Hemoglobin is the protein in red blood cells that binds oxygen, allowing oxygen to be transported around the body. Mutations in the HBBgene, which encodes a component of hemoglobin, causessickle cell.

Gene therapies involve either altering the mutated gene or introducing a healthy version of that gene to the body. Still under development for sickle cell, an estimated 27 patients have undergone experimental gene therapy. One strategy involves removing hematopoietic stem cells (which function to produce blood cells) from a patients bone marrow. A healthy copy of the HBB gene is then introduced into the cells using a modified, harmless virus known as a viral vector. The cells are then transplanted back into the patient where they will produce healthy red blood cells.

Traditionally, viral vectors for sickle cell have been designed in a way known as reverse structural orientation. This means that the HBB gene is translated or read from right to left, like reading an English sentence backwards. The reverse structural orientation design ensures that a key section of the gene (known as intron 2), which is necessary for the production of high levels of the HBB gene, is retained during viral vector preparation.

However, this design makes preparing the viral vectors more difficult, and decreases the efficiency of introducing the gene into the stem cells.

Researchersat the National Institutes of Healthdesigned a new viral vector, one in which the HBB gene is forward orientated and read from left to right. Genes essential for the virus were inserted into intron 2, meaning that only vectors that retained intron 2 would be produced (a type of positive selection).

Our new vector is an important breakthrough in the field of gene therapy for sickle cell disease, John Tisdale, MD, chief of the Cellular and Molecular Therapeutic Branch at the National Heart, Lung, and Blood Institute (NHLBI) and the studys senior author, said in a press release.

Its the new kid on the block and represents a substantial improvement in our ability to produce high capacity, high efficiency vectors for treating this devastating disorder, he added.

The researchers compared the new vectors to traditional reverse-orientated vectors in mouse and monkey models. The new vectors were four to 10 times more efficient at introducing the healthy HBBgene into the stem cells, and could carry up to six times more HBB genes compared to the conventional vectors.

Furthermore, the new vectors remained incorporated into the cells of monkeys up to four years after a transplant. These vectors could also be produced in greater amounts, which may lessen the time and costs required for large-scale vector production.

The researchers hope that these characteristics will make gene therapy for sickle cell disease more effective and increase its use. The new vector design still needs to be tested in clinical trials in patients.

Our lab has been working on improving beta-globin vectors for almost a decade and finally decided to try something radically different and it worked, Tisdale said.

These findings bring us closer to a curative gene therapy approach for hemoglobin disorders, he added.

Patricia holds her Ph.D. in Cell Biology from University Nova de Lisboa, and has served as an author on several research projects and fellowships, as well as major grant applications for European Agencies. She also served as a PhD student research assistant in the Laboratory of Doctor David A. Fidock, Department of Microbiology & Immunology, Columbia University, New York.

Total Posts: 94

Margarida graduated with a BS in Health Sciences from the University of Lisbon and a MSc in Biotechnology from Instituto Superior Tcnico (IST-UL). She worked as a molecular biologist research associate at a Cambridge UK-based biotech company that discovers and develops therapeutic, fully human monoclonal antibodies.

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New Viral Vector for Sickle Cell Gene Therapy Likely to Be More Effective, NIH Study Says - Sickle Cell Anemia News

Bone Marrow Stem Cells Comments Off on New Viral Vector for Sickle Cell Gene Therapy Likely to Be More Effective, NIH Study Says – Sickle Cell Anemia News | October 9th, 2019

DORVAL, QC, Oct. 9, 2019 /CNW/ - Novartis Pharmaceuticals Canada Inc. is pleased to announce that sites in Quebec have been certified in accordance with applicable requirements to treat eligible patients with Kymriah (tisagenlecleucel), the first chimeric antigen receptor T cell (CAR-T) therapy that received regulatory approval in Canada. Patients with relapsed/refractory (r/r) pediatric and young adult B-cell acute lymphoblastic leukemia (ALL) and adult r/r diffuse large B-cell lymphoma (DLBCL) may be eligible to be treated with Kymriah at one of the initially certified Canadian treatment sites. This news coincides with the Quebec government announcement that Kymriah is now reimbursed for eligible patients under the Rgie de l'assurance maladie du Qubec (RAMQ)ii.

Eligible patients in Quebec are now able to access Kymriah from the Centre hospitalier universitaire (CHU) Sainte-Justine and Maisonneuve-Rosemont Hospital (HMR) in Montreal.

"Novartis feels it is important to acknowledge the collaborative effort by all stakeholders involved to ensure Canadians have access to the first approved CAR-T therapy for patients with B-cell ALL and DLBCL who historically have poor outcomes. With treatment centers certified in Quebec, this allows patients with these two life-threatening cancers the opportunity to be treated with CAR-T therapy," said Daniel Hbert, Medical Director, Novartis Pharmaceuticals Canada Inc. "Novartis is committed to bringing additional qualified treatment centers from other parts of the country into the network to give Canadians the opportunity to be treated closer to home."

Due to the sophisticated and individualized nature of Kymriah, treatment sites that are part of the network are required to be FACT-accredited (Foundation for the Accreditation of Cellular Therapy), qualified to perform intravenous infusion of stem cells collected from the bone marrow of a donor, also referred to as allogeneic hematopoietic stem cell transplantation (alloSCT) and have experience with cell therapies, leukemia and lymphoma to facilitate safe and seamless delivery of Kymriah to eligible patients.

"We are thrilled with this news because we will now be able to treat patients at our institution with the knowledge that their therapy will be publicly funded. We see this as a significant step forward. The young patients we see who have refractory or relapsed B-cell ALL are desperately in need of a new treatment option. Kymriah brings hope to patients who are literally in a fight for their life." said Dr. Henrique Bittencourt, hematologist at the CHU Sainte-Justine in Montreal and Associate Professor, Department of Pediatrics, Universit de Montral.

"The expertise at HMR has raised the profile of our organization, which is a major Quebec, Canadian and worldwide pole for health innovation. Thanks to the dedicated work of our care, research and teaching teams, patients can now access this new treatment with demonstrated effectiveness and impact on quality of life," said Sylvain Lemieux, President and CEO, Centre intgr universitaire de sant et de services sociaux (CIUSSS) de l'Est-de-l'le-de-Montral.

About Kymriah Kymriah (tisagenlecleucel), a CD19-directed genetically modified autologous T-cell immunocellular therapy, is approved to treat two life-threatening cancers that have limited treatment options and historically poor outcomes, demonstrating the critical need for new therapies for these patients.

Kymriah is approved by Health Canada for use in pediatric and young adult patients 3 to 25 years of age with B-cell acute lymphoblastic leukemia (ALL) who are refractory, have relapsed after allogenic stem cell transplant (SCT) or are otherwise ineligible for SCT, or have experienced second or later relapse; and for the treatment of adult patients with relapsed or refractory (r/r) large B-cell lymphoma after two or more lines of systemic therapy including diffuse large B-cell lymphoma (DLBCL) not otherwise specified, high grade B-cell lymphoma and DLBCL arising from follicular lymphomai.

Kymriah is a one-time treatment that uses a patient's own T cells to fight and kill cancer cells. Bringing this innovative therapy to Canadian patients requires collaboration among many health system stakeholders.

Kymriah (tisagenlecleucel) Important Safety InformationThe full prescribing information for Kymriah can be found at: http://www.novartis.ca

Novartis Leadership in Cell and Gene TherapyNovartis is at the forefront of investigational immunocellular therapy and was the first pharmaceutical company to significantly invest in CAR-T research, work with pioneers in CAR-T and initiate global CAR-T trials. Kymriah, the first approved CAR-T cell therapy in Canada, is the cornerstone of this strategy. Active research programs are underway targeting other hematologic malignancies and solid tumors, and include efforts focused on next generation CAR-Ts that involve simplified manufacturing schemes and gene edited cells.

About Novartis in CanadaNovartis Pharmaceuticals Canada Inc., a leader in the healthcare field, is committed to the discovery, development and marketing of innovative products to improve the well-being of all Canadians. In 2018, the company invested $52 million in research and development in Canada. Located in Dorval, Quebec, Novartis Pharmaceuticals Canada Inc. employs approximately 1,000 people in Canada and is an affiliate of Novartis AG, which provides innovative healthcare solutions that address the evolving needs of patients and societies. For further information, please consult http://www.novartis.ca.

About NovartisNovartis is reimagining medicine to improve and extend people's lives. As a leading global medicines company, we use innovative science and digital technologies to create transformative treatments in areas of great medical need. In our quest to find new medicines, we consistently rank among the world's top companies investing in research and development. Novartis products reach more than 750 million people globally and we are finding innovative ways to expand access to our latest treatments. About 108,000 people of more than 140 nationalities work at Novartis around the world. Find out more at http://www.novartis.com.

Kymriah is a registered trademark.

References_____________________________________________i Novartis Pharmaceuticals Canada Inc., Kymriah Product Monograph. January 7, 2019.ii Quebec Ministry of Health and Social Services press release. October 8, 2019. Available at: https://www.newswire.ca/fr/news-releases/la-therapie-car-t-cell-maintenant-disponible-au-quebec-821953237.html

SOURCE Novartis Pharmaceuticals Canada Inc.

For further information: Novartis Media Relations, Daphne Weatherby, Novartis Corporate Communications, +1 514 633 7873, E-mail: camlph.communications@novartis.com

http://www.novartis.ca

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Novartis completes certification of initial sites in Quebec for first approved Canadian CAR-T therapy, Kymriah (tisagenlecleucel)(i) - Canada NewsWire

Bone Marrow Stem Cells Comments Off on Novartis completes certification of initial sites in Quebec for first approved Canadian CAR-T therapy, Kymriah (tisagenlecleucel)(i) – Canada NewsWire | October 9th, 2019

Registration is now open for World Cord Blood Day 2019 (November 15th) including the official online conference (free event) which will feature numerous cord blood transplant doctors and cellular therapy researchers such as Dr. Joanne Kurtzberg, Dr. Karen Ballen, Dr. Elizabeth Shpall, Dr. Wise Young and Dr. Filippo Milano. In addition, free educational events will be held around the globe.

TUCSON, Ariz., Oct. 9, 2019 /PRNewswire/ -- World Cord Blood Day (November 15th, 2019) is a free event and open to the public. In addition to events worldwide, World Cord Blood Day will feature a free online conference. Renowned researchers and leading transplant doctors will give introductory presentations for the public as well as academic lectures specifically designed for healthcare professionals.

Attendees will learn about the 40,000+ cord blood transplants performed since 1988 to treat over 80 life-threatening diseases including sickle cell anemia, thalassemia, lymphoma and leukemia. In addition, attendees will learn about exciting advances in the emerging field of regenerative medicine to potentially treat autism, cerebral palsy, spinal cord injury and more.

As the host and organizer of World Cord Blood Day 2019, Save the Cord Foundation is proud to announce the following speakers for this year's program (in order of appearance): Dr. Joanne Kurtzberg (Duke Department of Pediatrics, Duke Center for Autism and Brain Development), Dr. Karen Ballen (University of Virginia), Dave Murphy and Monroe Burgess (Quick Specialized Healthcare Logistics), Dr. Wise Young (Rutgers University), Dr. Elizabeth Shpall (MD Anderson Cancer Center), Dr. Filippo Milano (Fred Hutchinson Cancer Research Center). In addition, attendees will hear from Dr. Alexes Harris who beat cancer thanks to a cord blood transplant from a donor and young Luke Fryer who was treated for cerebral palsy with his own cord blood in a clinical trial.

The morning session will focus on the success of cord blood transplants over the past 30 years and how transplant doctors use cord blood stem cells today, namely, to fight blood cancer. The afternoon session will look at new directions in cord blood research. Attendees will receive updates on several ground-breaking clinical trials using cord blood in regenerative medicine, cellular therapy and more. To view the full agenda, please visit: https://www.worldcordbloodday.org/online-medical-conference-agenda.html

Organized and hosted by Save the Cord Foundation (501c3 non-profit), this year's event is officially sponsored by Quick Specialized Healthcare Logistics. Inspiring Partners include the Cord Blood Association (CBA), Be the Match (NMDP), World Marrow Donor Association (WMDA-Netcord), AABB Center for Cellular Therapy and Foundation for the Accreditation of Cellular Therapy (FACT).

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Visit http://www.WorldCordBloodDay.org to learn how you can participate and/or host an event. Join us on social media using the hashtags: #WCBD19 and #WorldCordBloodDay.

About Save the Cord Foundation

Save the Cord Foundation (a 501c3 non-profit) was established to advance cord blood education. The Foundation provides non-commercial information to parents, health professionals and the public regarding methods for saving cord blood, as well as current applications using cord blood and the latest research. Learn more at http://www.SaveTheCordFoundation.org.

About Quick Specialized Healthcare Logistics

Quick is the trusted logistics leader serving the Healthcare and Life Science community for almost 40 years. Quick safely transports human organs and tissue for transplant or research, blood, blood products, cord blood, bone marrow, medical devices and personalized medicine, 24/7/365. Quick's specially trained experts work with hospitals, laboratories, blood banks and medical processing canters, and utilize the safest routes to ensure integrity, temperature control and chain of custody throughout the transportation process. Learn more at http://www.quickhealthcare.aero.

Media contact:Charis Ober225955@email4pr.com520-419-0269

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World Cord Blood Day 2019 to Welcome Leading Transplant Doctors and Pioneering Cellular Therapy Researchers - Yahoo Finance

Bone Marrow Stem Cells Comments Off on World Cord Blood Day 2019 to Welcome Leading Transplant Doctors and Pioneering Cellular Therapy Researchers – Yahoo Finance | October 9th, 2019