Archive for the ‘Bone Marrow Stem Cells’ Category

LONDON, UK / ACCESSWIRE / January 15, 2020 / Hemogenyx Pharmaceuticals plc (HEMO.L) ("Hemogenyx" or the "Company"), the biopharmaceutical group developing new therapies and treatments of blood diseases, is pleased to announce the following update on its activities.

As previously announced, Hemogenyx's CDX product has the potential to treat Acute Myeloid Leukemia (AML) directly as well as providing a benign conditioning regimen for blood stem cell replacement therapy. The Company has now carried out extensive work developing treatments for AML and has to date obtained encouraging results.

Hemogenyx has successfully constructed and in vitro tested Chimeric Antigen Receptor (CAR) programmed T cells (HEMO-CAR-T) for potential treatment of AML. HEMO-CAR was constructed using Hemogenyx's proprietary humanized monoclonal antibody against a target on the surface of AML cells. The Company has demonstrated that HEMO-CAR was able to programme human T cells (converted them into HEMO-CAR-T) to identify and destroy human AML derived cells in vitro.

Following the successful completion of these tests, in vivo tests of the efficacy of HEMO-CAR-T against AML are being conducted utilising a model of AML using Advanced peripheral blood Hematopoietic Chimera (ApbHC) - humanized mice developed by Immugenyx, LLC, a wholly-owned subsidiary of Hemogenyx.

Vladislav Sandler, Chief Executive Officer, commented, "We are encouraged by this new data which demonstrates our continuing progress in the development of novel treatments for blood cancers such as AML. The development of HEMO-CAR-T expands Hemogenyx's pipeline and advances it into a cutting-edge area of cell-based immune therapy. We are excited to have developed another product candidate that should, if successful, provide a new and potentially effective treatment for blood cancers for which survival rates are currently very poor."

About AML and CAR-T

AML, the most common type of acute leukemia in adults, has poor survival rates (a five-year survival rate of less than 25% in adults) and is currently treated using chemotherapy, rather than the potentially more benign and effective form of therapy being developed by Hemogenyx. The successful development of the new therapy for AML would have a major impact on treatment and survival rates for the disease.

CAR-T therapy is a treatment in which a patient's own T cells, a type of immune cell, are modified to recognize and kill the patient's cancer cells. The procedure involves: isolating T cells from the patient, modifying the isolated T cells in a laboratory using a CAR gene construct (which allows the cells to recognize the patient's cancer); amplifying (growing to large numbers) the newly modified cells; and re-introducing the cells back into the patient.

Market Abuse Regulation (MAR) Disclosure

Certain information contained in this announcement would have been deemed inside information for the purposes of Article 7 of Regulation (EU) No 596/2014 until the release of this announcement.

Enquiries:

Hemogenyx Pharmaceuticals plc

http://www.hemogenyx.com

Dr Vladislav Sandler, Chief Executive Officer & Co-Founder

headquarters@hemogenyx.com

Sir Marc Feldmann, Executive Chairman

SP Angel Corporate Finance LLP

Tel: +44 (0)20 3470 0470

Matthew Johnson, Vadim Alexandre, Soltan Tagiev

Peterhouse Corporate Finance Limited

Tel: +44 (0)20 7469 0930

Lucy Williams, Duncan Vasey

US Media enquiries

Tel: +1 (323) 646-3249

Lowell Goodman

lowell@corbomitecomms.com

About Hemogenyx Pharmaceuticals plc

Hemogenyx Pharmaceuticals plc ("Hemogenyx") is a publicly traded company (HEMO.L) headquartered in London, with its wholly-owned US operating subsidiaries, Hemogenyx LLC and Immugenyx LLC, located in New York City at its state-of-the-art research facility and a wholly-owned Belgian operating subsidiary, Hemogenyx-Cell SPRL, located in Lige.

Hemogenyx is a pre-clinical stage biopharmaceutical group developing new medicines and treatments to bring the curative power of bone marrow transplantation to a greater number of patients suffering from otherwise incurable life-threatening diseases. Hemogenyx is developing several distinct and complementary product candidates, as well as a platform technology that it uses as an engine for novel product development.

For more than 50 years, bone marrow transplantation has been used to save the lives of patients suffering from blood diseases. The risks of toxicity and death that are associated with bone marrow transplantation, however, have meant that the procedure is restricted to use only as a last resort. Hemogenyx's technology has the potential to enable many more patients suffering from devastating blood diseases such as leukemia and lymphoma, as well as severe autoimmune diseases such as multiple sclerosis, aplastic anemia and systemic lupus erythematosus (Lupus), to benefit from bone marrow transplantation.

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Hemogenyx's CAR-T Cells are Effective Against AML in vitro - Yahoo Finance

PALO ALTO, Calif.--(BUSINESS WIRE)--Jasper Therapeutics, Inc., a biotechnology company focused on hematopoietic cell transplant therapies, today announced the expansion of its Series A financing with an additional investment of $14.1 million led by Roche Venture Fund and with participation from other investors, bringing the total company financing to more than $50 million to date. The initial Series A round was led by Abingworth LLP and Qiming Venture Partners USA, with further investment from Surveyor Capital (a Citadel company) and participation from Alexandria Venture Investments, LLC.

Jasper plans to use the proceeds to advance and expand the study of its lead clinical asset, JSP191. A humanized antibody targeting CD117 on hematopoietic stem cells, JSP191 is designed to replace toxic chemotherapy and radiation therapy as conditioning regimens to prepare patients for curative stem cell and gene therapy. JSP191 is the only antibody of its kind in clinical development as a single conditioning agent for people undergoing curative hematopoietic cell transplantation.

This investigational agent is currently being evaluated in a Phase 1/2 dose-escalation and expansion study as a conditioning agent to enable stem cell engraftment in patients with severe combined immunodeficiency (SCID) who received a prior stem cell transplant that resulted in poor outcome. Initial positive results from this ongoing clinical trial were presented in an oral session at the American Society of Hematology (ASH) Annual Meeting in December 2019. Jasper plans to expand the Phase 1/2 clinical study to include patients with acute myeloid leukemia (AML) or myelodysplastic syndromes (MDS) receiving hematopoietic cell transplant. The development of JSP191 is supported by a collaboration with the California Institute for Regenerative Medicine (CIRM).

About Hematopoietic Cell TransplantationBlood-forming, or hematopoietic, stem cells are rare cells that reside in the bone marrow and are responsible for the generation and maintenance of all blood and immune cells. These stem cells can harbor inherited or acquired abnormalities that lead to a variety of disease states, including immune deficiencies, blood disorders or hematologic cancers. Replacement of the defective or malignant hematopoietic stem cells in the patients bone marrow by transplantation and engraftment of healthy stem cells is the only cure for most of these life-threatening conditions. Successful transplantation is currently achieved by subjecting patients to toxic treatment with radiation and/or chemotherapy followed by transplantation of a donor or gene-corrected hematopoietic cell graft. These toxic regimens cause DNA damage and lead to short- and long-term toxicities, including unwanted damage to organs and prolonged hospitalization. As a result, many patients who could benefit from a hematopoietic cell transplant are not eligible. New approaches that are effective but have minimal to no toxicity are urgently needed so more patients who could benefit from a curative stem cell transplant could receive the procedure.

Safer and more effective hematopoietic cell transplantation regimens could overcome these limitations and enable the broader application of hematopoietic cell transplants in the cure of many disorders. These disorders include hematologic cancers (e.g., myelodysplastic syndrome [MDS] and acute myeloid leukemia [AML]), autoimmune diseases (e.g., lupus, rheumatoid arthritis, multiple sclerosis and Type 1 diabetes), and genetic diseases that could be cured with genetically-corrected autologous stem cells (e.g., severe combined immunodeficiency syndrome [SCID], sickle cell disease, beta thalassemia, Fanconi anemia and other monogenic diseases).

About JSP191JSP191 (formerly AMG 191) is a first-in-class humanized monoclonal antibody in clinical development as a conditioning agent that clears hematopoietic stem cells from bone marrow. JSP191 binds to human CD117, a receptor for stem cell factor (SCF) that is expressed on the surface of hematopoietic stem and progenitor cells. The interaction of SCF and CD117 is required for stem cells to survive. JSP191 blocks SCF from binding to CD117 and disrupts critical survival signals, causing the stem cells to undergo cell death and creating an empty space in the bone marrow for donor or gene-corrected transplanted stem cells to engraft.

Preclinical studies have shown that JSP191 as a single agent safely depletes normal and diseased hematopoietic stem cells, including in an animal model of MDS. This creates the space needed for transplanted normal donor or gene-corrected hematopoietic stem cells to successfully engraft in the host bone marrow. To date, JSP191 has been evaluated in more than 80 healthy volunteers and patients. It is currently being evaluated as a sole conditioning agent in a Phase 1/2 dose-escalation and expansion trial to achieve donor stem cell engraftment in patients undergoing hematopoietic cell transplant for SCID, which is curable only by this type of treatment. For more information about the design of the clinical trial, visit http://www.clinicaltrials.gov (NCT02963064). Clinical development of JSP191 will be expanded to also study patients with AML or MDS who are receiving hematopoietic cell transplant. IND-enabling studies are planned to advance JSP191 as a conditioning agent for patients with other rare and ultra-rare monogenic disorders and autoimmune diseases.

About Jasper TherapeuticsJasper Therapeutics is a biotechnology company focused on hematopoietic cell transplant therapies. The companys lead compound, JSP191, is in clinical development as a conditioning antibody that clears hematopoietic stem cells from bone marrow in patients undergoing a hematopoietic cell transplant. This first-in-class conditioning antibody is designed to enable safer and more effective curative hematopoietic cell transplants and gene therapies. For more information, please visit us at https://jaspertherapeutics.com.

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Jasper Therapeutics Announces Expansion of Series A Financing, Bringing Total Corporate Fundraising to More than $50 Million - Business Wire

Pete McCleave pictured with his children

Sleights residents, June and Mike McCleave's son Peter has Myeloma, a type of bone marrow cancer for which there is currently no cure.

Peter, 42, was diagnosed with the disease three years ago, and time is running short for the man who two years ago was given just seven years to live.

The family is now in a race against time to find a matching stem cell donor, who can provide the transfusion that will extend Peter's life, hopefully long enough for a cure to be found.

Mum, June, said: "We go to myeloma conferences which give details of all the updated work and drugs that are available. They are very hopeful of a cure and are working on one which involves gene therapy, meaning that good cells will attack the cancer. They reckon that in ten years there will be a cure for this."

Peter has been determined to fight the disease. He set up a campaign called 10,000 donors to encourage as many people as possible to register with DKMS, the charity dedicated to defeating blood cancer. To date 33,402 donors have registered because of this campaign and 12 donor matches have been confirmed, Pete is still waiting.

June and Mike have organised an event at Eskdale School for people to go along and take a cheek swab test to see if they are a compatible match for Peter, or others who have the disease.

The event takes place of Tuesday, January 14 from 4.00pm to 7,00pm.

Taking the test is simple and pain free, three cotton swabs (like cotton buds) collect saliva from inside the mouth and are sent for testing. It's a process which is over in seconds, with one swab collecting saliva from the left cheek, one from the right cheek and one from around the mouth.

A DKMS representative will be at the session and will take the swabs to the laboratory for analysis, you will then receive a card a few weeks later confirming you are registered as a potential donor.

See more here:
Sleights family's appeal for blood stem cell donor in Whitby - The Scarborough News

Sickle cell disease is a painful condition that thousands of kids have to endure. The genetic disease impacts the blood, but it can cause organ damage and other issues, including lots of pain.

Most of the time there is no end in sight, which makes it even harder on families. But the bravery of one teen is helping scientists to develop a potential cure that could change the lives of so many.

Helen Obando recently became the youngest person to ever go through a special gene therapy using stem cells.

The usual treatment for sickle cell therapy is a bone marrow transplant form a healthy sibling, but Helen's older sister Haylee also has sickle cell, so that isn't really an option for the family.

The Obandos were excited to learn about an experimental treatment that has the potential to flip the switch on the genetics and actually cure the disease.

Scientists are hoping that the new treatment could help people with a number of genetic conditions using a technique to manipulate the DNA.

Helen had to spend four weeks in the hospital after her infusion to get strong enough to go home, and they don't know yet if the treatment has worked.

The poor girl has gone through a lot. Her pelvis was harmed before she even turned 1, and at 2, her spleen had to be removed. She's had a lot of painful episodes, and while Haylee was able to match with their younger brother Ryan for a bone marrow transplant, that wasn't an option for Helen.

In the Boston Globe, Helen's mom said that she was scared of the gene therapy option when she first heard of it. But she decided that it was worth the risk to have a chance at being healthy.

Six months since the treatment, it's so far, so good. Helen's hemoglobin levels are at a point that she has never achieved. She actually has no signs of sickle cell right now, and that is just amazing.

What a brave girl to go through a risky procedure.It's a big burden for a teenager to bear, but luckily things have worked out well so far. We hope that Helen continues to find success and health in the new year.

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Helen Obando Is The Youngest To Successfully Undergo Sickle Cell Therapy - Moms

Cancer enters your body when cells begin to grow out of control. There are various types of cancer and cells in almost every part of the body can become cancer. Leukemia is a type of cancer which starts in the cells, then develops into different types of blood cells. It starts in early forms of white blood cells. There are different types of leukemia which can be divided into acute and chronic. Acute is fast growing and chronic is slow growing.

An Acute Lymphoblastic Leukemia is a type of leukemia which progresses quickly and if not treated, will be fatal in a couple of months. Acute means fast growing and lymphatic means it develops from the early forms of lymphocytes, which is a type of white blood cell. It all starts in the bone marrow and leukemia cells start to invade the body quickly. They can spread to other parts of the body. Some cancers also start in the organs and then spread to the bone marrow, but they are not leukemia.

There are other types of cancer which start in lymphocytes and are known as lymphomas. Leukemias affect blood and bone marrow and lymphomas affect lymph nodes and other organs. It can sometimes be difficult to tell if a cancer of lymphocytes is lymphoma or leukemia. If at least 20% of the bone marrow has cancerous lymphocytes, the disease is considered to be leukemia. Acute Lymphoblastic Leukemia is the most common childhood cancer and children below the age of five are at the highest risk. It can also occur in adults.

RELATED:Kids Born To Obese Mothers Are More Likely To Develop Leukemia

ALL can increase the chances of bleeding and developing infections in the body. Its symptoms include:

In order to diagnose ALL, the doctor must complete a physical exam and also conduct bone marrow tests and blood tests. Doctors are likely to ask about bone pain, since it is the most common symptom of ALL. Here are a few tests doctors carry out.

The doctor might order a blood count, and people who have ALL may have a blood count which shows low platelet count and a low hemoglobin count. The WBC may or may not have increased. A blood smear might show immature cells circulating in the blood, which are usually found in bone marrow.

This process involves taking a sample of the bone marrow from your breastbone or the pelvis. It is an ideal way to test for increased growth in marrow tissue and reduced production of red blood cells.

An X-ray of the chest can allow the doctor to see if the mediastinum, that is the middle partition of the chest is widened. Further, a CT scan can help the doctor estimate whether the cancer has spread to the spinal cord, brain or to any other part of the body.

There are other tests like a spinal tap, which is used to check if cancer cells have spread around the spinal fluid. Tests on the serum urea and liver function might also be done.

The treatment will help bring the count back to normal. When this happens and the bone marrow looks normal, the cancer is in remission. Acute Lymphoblastic Leukemia can be treated through chemotherapy. You might be asked to stay at the hospital for a few weeks in the first treatment. Later, you can continue the treatment as an outpatient.

For those with a low WBC count, you will be asked to spend time in an isolation room. It ensures that you are protected from contagious diseases and other problems. If leukemia does not respond to chemotherapy, a bone marrow or stem cell transplant might be recommended. The transplanted marrow can be taken from a sibling who is a complete match.There are high chances of cancer remission in case of children.

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You're Probably Ruining Your Teenager's Life & Here's What You're Doing Wrong

Tags:cancer,childhood cancer

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Criss Angel's Son Has Acute Lymphoblastic Leukemia, But What Is It? - Moms

CAMBRIDGE, Mass.--(BUSINESS WIRE)--bluebird bio, Inc. (Nasdaq: BLUE) announced the launch in Germany of ZYNTEGLO (autologous CD34+ cells encoding A-T87Q-globin gene), a one-time gene therapy for patients 12 years and older with transfusion-dependent -thalassemia (TDT) who do not have a 0/0 genotype, for whom hematopoietic stem cell (HSC) transplantation is appropriate but a human leukocyte antigen (HLA)-matched related HSC donor is not available. This is the first time ZYNTEGLO is commercially available.

TDT is a severe genetic disease caused by mutations in the -globin gene that result in significantly reduced or absent adult hemoglobin (HbA). In order to survive, people with TDT maintain hemoglobin (Hb) levels through lifelong chronic blood transfusions. These transfusions carry the risk of progressive multi-organ damage due to unavoidable iron overload. ZYNTEGLO is a one-time gene therapy that addresses the underlying genetic cause of TDT and offers patients the potential to become transfusion independent, which, once achieved, is expected to be lifelong.

Due to the highly technical and specialized nature of administering gene therapy in rare diseases, bluebird bio is working with institutions that have expertise in stem cell transplant as well as in treating patients with TDT to create qualified treatment centers that will administer ZYNTEGLO. bluebird bio has established a collaboration with University Hospital of Heidelberg as the first qualified treatment center in Germany.

In addition, bluebird has entered into value-based payment agreements with multiple statutory health insurances in Germany to help ensure patients and their healthcare providers have access to ZYNTEGLO and that payers only pay if the therapy delivers on its promise. bluebirds proposed innovative model is limited to five payments made in equal installments. An initial payment is made at the time of infusion. The four additional annual payments are only made if no transfusions for TDT are required for the patient.

For patients with TDT, lifelong chronic blood transfusions are required in order to survive. We are thrilled to announce that ZYNTEGLO will now be available for patients in the EU living with this severe disease, says Alison Finger, chief commercial officer, bluebird bio. In addition to confirming manufacturing readiness of our partner, apceth Biopharma GmbH, bluebird has also submitted a dossier to the Joint Federal Committee (G-BA) in Germany for drug benefit assessment. We would like to thank our collaborators for their commitment in helping us transform the healthcare system by accepting innovative payment models, and we look forward to treating our first commercial patient soon.

About LentiGlobin for -Thalassemia (autologous CD34+ cells encoding A-T87Q-globin gene)

The European Commission granted conditional marketing authorization for LentiGlobin for -thalassemia, to be marketed as ZYNTEGLO (autologous CD34+ cells encoding A-T87Q-globin gene) gene therapy, for patients 12 years and older with TDT who do not have a 0/0 genotype, for whom hematopoietic stem cell (HSC) transplantation is appropriate, but a human leukocyte antigen (HLA)-matched related HSC donor is not available.

TDT is a severe genetic disease caused by mutations in the -globin gene that result in reduced or significantly reduced hemoglobin (Hb). In order to survive, people with TDT maintain Hb levels through lifelong chronic blood transfusions. These transfusions carry the risk of progressive multi-organ damage due to unavoidable iron overload.

LentiGlobin for -thalassemia adds functional copies of a modified form of the -globin gene (A-T87Q-globin gene) into a patients own hematopoietic (blood) stem cells (HSCs). Once a patient has the A-T87Q-globin gene, they have the potential to produce HbAT87Q, which is gene therapy-derived hemoglobin, at levels that may eliminate or significantly reduce the need for transfusions.

Non-serious adverse events (AEs) observed during the HGB-204, HGB-207 and HGB-212 clinical studies that were attributed to LentiGlobin for -thalassemia were hot flush, dyspnoea, abdominal pain, pain in extremities, thrombocytopenia, leukopenia, neutropenia and non-cardiac chest pain. One serious adverse event (SAE) of thrombocytopenia was considered possibly related to LentiGlobin for -thalassemia for TDT.

Additional AEs observed in clinical studies were consistent with the known side effects of HSC collection and bone marrow ablation with busulfan, including SAEs of veno-occlusive disease.

The conditional marketing authorization for ZYNTEGLO is valid in the 28 member states of the EU as well as Iceland, Liechtenstein and Norway. For details, please see the Summary of Product Characteristics (SmPC).

The U.S. Food and Drug Administration (FDA) granted LentiGlobin for -thalassemia Orphan Drug status and Breakthrough Therapy designation for the treatment of TDT. LentiGlobin for -thalassemia is not approved in the United States.

bluebird bio has initiated the rolling BLA submission for approval in the U.S., and is engaged with the FDA in discussions regarding the requirements and timing of the various components of the rolling BLA submission. Subject to these ongoing discussions, the company is currently planning to complete the BLA submission in the first half of 2020.

LentiGlobin for -thalassemia continues to be evaluated in the ongoing Phase 3 Northstar-2 and Northstar-3 studies. For more information about the ongoing clinical studies, visit http://www.northstarclinicalstudies.com or clinicaltrials.gov and use identifier NCT02906202 for Northstar-2 (HGB-207) or NCT03207009 for Northstar-3 (HGB-212).

bluebird bio is conducting a long-term safety and efficacy follow-up study (LTF-303) for people who have participated in bluebird bio-sponsored clinical studies of LentiGlobin for -thalassemia. For more information visit: https://www.bluebirdbio.com/our-science/clinical-trials or clinicaltrials.gov and use identifier NCT02633943 for LTF-303.

About bluebird bio, Inc.

bluebird bio is pioneering gene therapy with purpose. From our Cambridge, Mass., headquarters, were developing gene therapies for severe genetic diseases and cancer, with the goal that people facing potentially fatal conditions with limited treatment options can live their lives fully. Beyond our labs, were working to positively disrupt the healthcare system to create access, transparency and education so that gene therapy can become available to all those who can benefit.

bluebird bio is a human company powered by human stories. Were putting our care and expertise to work across a spectrum of disorders including cerebral adrenoleukodystrophy, sickle cell disease, -thalassemia and multiple myeloma, using three gene therapy technologies: gene addition, cell therapy and (megaTAL-enabled) gene editing.

bluebird bio has additional nests in Seattle, Wash.; Durham, N.C.; and Zug, Switzerland. For more information, visit bluebirdbio.com.

Follow bluebird bio on social media: @bluebirdbio, LinkedIn, Instagram and YouTube.

ZYNTEGLO, LentiGlobin, and bluebird bio are trademarks of bluebird bio, Inc.

The full common name for ZYNTEGLO: A genetically modified autologous CD34+ cell enriched population that contains hematopoietic stem cells transduced with lentiviral vector encoding the A-T87Q-globin gene.

Forward-Looking Statements

This release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995, including statements regarding the Companys plans and expectations for the commercialization for ZYNTEGLO (autologous CD34+ cells encoding A-T87Q-globin gene, formerly LentiGlobin in TDT) to treat TDT, and the potential implications of clinical data for patients. Any forward-looking statements are based on managements current expectations of future events and are subject to a number of risks and uncertainties that could cause actual results to differ materially and adversely from those set forth in or implied by such forward-looking statements. These risks and uncertainties include, but are not limited to: the risk that the efficacy and safety results from our prior and ongoing clinical trials of ZYNTEGLO will not continue or be repeated in our ongoing or planned clinical trials of ZYNTEGLO; the risk that the current or planned clinical trials of ZYNTEGLO will be insufficient to support regulatory submissions or marketing approval in the US, or for additional patient populations in the EU; the risk that the production of HbAT87Q may not be sustained over extended periods of time; the risk that we may not secure adequate pricing or reimbursement to support continued development or commercialization of ZYNTEGLO; the risk that our collaborations with qualified treatment centers will not continue or be successful; and that the risk that commercial patients treated with ZYNTEGLO will not achieve or maintain transfusion independence. For a discussion of other risks and uncertainties, and other important factors, any of which could cause our actual results to differ from those contained in the forward-looking statements, see the section entitled Risk Factors in our most recent Form 10-Q, as well as discussions of potential risks, uncertainties, and other important factors in our subsequent filings with the Securities and Exchange Commission. All information in this press release is as of the date of the release, and bluebird bio undertakes no duty to update this information unless required by law.

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bluebird bio Announces Launch in Germany of ZYNTEGLO (autologous CD34+ cells encoding A-T87Q-globin gene) Gene Therapy for Patients 12 Years and Older...

NEW YORK, Jan. 13, 2020 /PRNewswire/ --Actinium Pharmaceuticals, Inc. (NYSE AMERICAN: ATNM) ("Actinium"), announced today that it has entered into an agreement with the University of California, Davis (UC Davis) to utilize Actinium's Antibody Radiation-Conjugate or ARC apamistamab-I-131 for targeted conditioning and replace the chemotherapy conditioning being used in an ongoing Phase 1/2 stem cell gene therapy clinical trial. In the trial, patients with relapsed or refractory HIV-related lymphoma are being treated with autologous stem cell gene therapy. This is the first gene therapy clinical trial that will utilize ARC based conditioning. The clinical trial will be conducted at UC Davis and may be expanded to additional sites in the future.

Dr. Mehrdad Abedi, Professor, Hematology and Oncology at UC Davis and study lead, said, "This collaboration represents an exciting combination of revolutionary technologies that could further our ability to treat patients with HIV and other life-threatening diseases with gene therapy. Despite the advances made in the field of gene therapy, the reliance on non-targeted chemotherapy and external radiation as conditioning regimens is less than optimal and poses a problem that we hope to reduce or eliminate as part of this collaboration by replacing our conditioning regimen in this study with Actinium's ARC based targeted conditioning. Advances in HIV therapies have dramatically improved patient survival, but current therapies require life-long daily use to keep the HIV virus at bay, can have severe side effects, may be overcome by HIV resistance and do not address the needs of all patients like those in this study with HIV-related lymphomas. We envision a future where a single treatment of our stem cell gene therapy can cure patients of their lymphoma and HIV leaving the patient with a new immune system that can fight, be resistant to and prevent the mutation of HIV. Apamistamab-I-131's demonstrated antitumor effect against lymphoma and ability to condition patients in a targeted manner with a demonstrated tolerable safety profile in the bone marrow transplant setting makes it an ideal conditioning agent for this patient population. Based on these factors and extensive supporting clinical data in the Iomab-B program, we selected this ARC as the conditioning agent for the next phase of our trial as we believe antibody radiation-conjugates are more advanced and hold distinct advantages over novel but unproven conditioning technologies such as Antibody Drug Conjugates and naked antibodies that are beginning to be developed albeit at the preclinical stage."

In the current clinical trial, the anti-HIV stem cell gene therapy is produced by taking a patient's own or autologous, blood forming stem cells and genetically modifying them via gene therapy with a combination of three anti-HIV genes. The intended result is for the gene modified bone marrow stem cells to produce a new immune system and newly arising immune cells that are resistant to HIV via a single treatment. Conditioning is necessary prior to adoptive cell therapies such as gene therapy to eliminate certain cell types such as immune cells and stem cells in the bone marrow so the transplanted cells can engraft. Until now, conditioning in this trial, as is typical, used a multi-drug chemotherapy regimen administered over several days. This approach is non-targeted, associated with toxicities that impairs patients and restricts the use and efficacy of cellular therapy. Apamistamab-I-131, which requires just one therapeutic administration, will displace the non-targeted chemotherapy to condition patients in a targeted manner with the goal of reducing conditioning related toxicities and improving patient outcomes. Actinium and UC David will cross-reference their respective Investigational New Drug applications and will work collaboratively to obtain necessary regulatory and institutional approvals. In this clinical collaboration, Actinium will provide drug product, support for its administration and certain trial costs. UC Davis will be responsible for the production of the anti-HIV stem cell gene therapy and overall conduct of the study and its cost.

Dr. Dale Ludwig, Actinium's Chief Scientific Officer, said, "We are excited to be working with Dr. Abedi on this clinical study and we appreciate his recognition of the value of our Iomab-ACT targeted conditioning program may provide in support of gene stem cell therapy. This targeted approach using our CD45 ARC, enables both anti-tumor activity and effective conditioning with the potential for reduced toxicity compared to non-targeted chemotherapy and external radiation in the bone marrow transplant setting. Supported by extensive clinical investigation in 12 trials and over 300 patients, a single therapeutic dose of apamistamab-I-131 is sufficient for conditioning and, due to its dual activity, even a patient with active disease could expect to receive therapy within two weeks, which is anticipated to lead to better outcomes compared to chemotherapy, external beam radiation, or exploratory approaches such as naked antibodies or Antibody Drug Conjugates. In addition, CD45, the target of apamistamab-I-131, is ideal for targeted conditioning, as it is not expressed outside of the haemopoietic system and, because it is a poorly internalizing receptor. An ARC approach which does not require internalization of its radionuclide warhead for target cell killing, is anticipated to be more viable and more effective than Antibody Drug Conjugate approaches which need to internalize their payloads. Given the potential of this ARC targeted conditioning technology for bone marrow transplant, we are grateful to Dr. Abedi for the opportunity to advance the Iomab-ACT program into the promising field of gene stem cell therapy."

Sandesh Seth, Actinium's Chairman and Chief Executive Officer, said, "Actinium is thrilled to be working with UC Davis and honored to now be part of this important trial. It has become evident that better conditioning regimens are needed for cell and gene therapies to reach their full potential. Our team is proud to be the first company to establish a clinical stage targeted conditioning portfolio for both cell and gene therapy. We are pleased to extend our ARC technology for targeted conditioning into these rapidly advancing fields and we are committed to establishing a strong leadership position in enabling these adoptive cell therapies fully realize their great potential for improving patients' lives."

Apamistamab-I-131's demonstrated conditioning and antitumor effect in lymphoma1

Actinium's apamistamab-I-131 ARC has been studied as a targeted conditioning agent in over 300 patients in the bone marrow transplant setting in the Iomab-B Program and is currently being studied in a pivotal Phase 3 clinical (SIERRA) trial in patients with relapsed or refractory acute myeloid leukemia. Clinical proof of concept has been established with Iomab-B for targeted conditioning in high-risk, relapsed or refractory lymphoma patients prior to an autologous stem cell transplant where a favorable safety profile with no dose limiting toxicities and minimal non-hematologic toxicities observed and promising efficacy with median overall survival not reached (range: 29 months to infinity) and 31% of patients in prolonged remission at a median of 36 months follow up (range: 25 41 months)1.

1) Cassaday et al. Phase I Study of a CD45-Targeted AntibodyRadionuclide Conjugate for High-Risk Lymphoma. AACR Clin Cancer Res Published OnlineFirst September 3, 2019

About Actinium Pharmaceuticals, Inc.

Actinium Pharmaceuticals, Inc. is a clinical-stage biopharmaceutical company developing ARCs or Antibody Radiation-Conjugates, which combine the targeting ability of antibodies with the cell killing ability of radiation. Actinium's lead application for our ARCs is targeted conditioning, which is intended to selectively deplete a patient's disease or cancer cells and certain immune cells prior to a BMT or Bone Marrow Transplant, Gene Therapy or Adoptive Cell Therapy (ACT) such as CAR-T to enable engraftment of these transplanted cells with minimal toxicities. With our ARC approach, we seek to improve patient outcomes and access to these potentially curative treatments by eliminating or reducing the non-targeted chemotherapy that is used for conditioning in standard practice currently. Our lead product candidate, apamistamab-I-131 (Iomab-B) is being studied in the ongoing pivotal Phase 3 Study of Iomab-B in Elderly Relapsed or Refractory Acute Myeloid Leukemia (SIERRA) trial for BMT conditioning. The SIERRA trial is over fifty percent enrolled and promising single-agent, feasibility and safety data has been highlighted at ASH, TCT, ASCO and SOHO annual meetings. Apatmistamamb-I-131 will also be studied as a targeted conditioning agent in a Phase 1/2 anti-HIV stem cell gene therapy with UC Davis and is expected to be studied with a CAR-T therapy in 2020. In addition, we are developing a multi-disease, multi-target pipeline of clinical-stage ARCs targeting the antigens CD45 and CD33 for targeted conditioning and as a therapeutic either in combination with other therapeutic modalities or as a single agent for patients with a broad range of hematologic malignancies including acute myeloid leukemia, myelodysplastic syndrome and multiple myeloma. Ongoing combination trials include our CD33 alpha ARC, Actimab-A, in combination with the salvage chemotherapy CLAG-M and the Bcl-2 targeted therapy venetoclax. Underpinning our clinical programs is our proprietary AWE (Antibody Warhead Enabling) technology platform. This is where our intellectual property portfolio of over 100 patents, know-how, collective research and expertise in the field are being leveraged to construct and study novel ARCs and ARC combinations to bolster our pipeline for strategic purposes. Our AWE technology platform is currently being utilized in a collaborative research partnership with Astellas Pharma, Inc.

Forward-Looking Statements for Actinium Pharmaceuticals, Inc.

This press release may contain projections or other "forward-looking statements" within the meaning of the "safe-harbor" provisions of the private securities litigation reform act of 1995 regarding future events or the future financial performance of the Company which the Company undertakes no obligation to update. These statements are based on management's current expectations and are subject to risks and uncertainties that may cause actual results to differ materially from the anticipated or estimated future results, including the risks and uncertainties associated with preliminary study results varying from final results, estimates of potential markets for drugs under development, clinical trials, actions by the FDA and other governmental agencies, regulatory clearances, responses to regulatory matters, the market demand for and acceptance of Actinium's products and services, performance of clinical research organizations and other risks detailed from time to time in Actinium's filings with the Securities and Exchange Commission (the "SEC"), including without limitation its most recent annual report on form 10-K, subsequent quarterly reports on Forms 10-Q and Forms 8-K, each as amended and supplemented from time to time.

Contacts:

Investors:Hans VitzthumLifeSci Advisors, LLCHans@LifeSciAdvisors.com(617) 535-7743

Media:Alisa Steinberg, Director, IR & Corp Commsasteinberg@actiniumpharma.com(646) 237-4087

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Actinium Pharmaceuticals Announces Iomab-ACT Program Gene Therapy Collaboration with UC Davis in Ongoing Clinical Trial for Patients with HIV-Related...

White blood cells circulate around the blood and help the immune system fight off infections.

Stem cells in the bone marrow are responsible for producing white blood cells. The bone marrow then stores an estimated 8090% of white blood cells.

When an infection or inflammatory condition occurs, the body releases white blood cells to help fight the infection.

In this article, learn more about white blood cells, including the types and their functions.

Health professionals have identified three main categories of white blood cell: granulocytes, lymphocytes, and monocytes. The sections below discuss these in more detail.

Granulocytes are white blood cells that have small granules containing proteins. There are three types of granulocyte cells:

These white blood cells include the following:

Monocytes are white blood cells that make up around 28% of the total white blood cell count in the body. These are present when the body fights off chronic infections.

They target and destroy cells that cause infections.

According to an article in American Family Physician, the normal range (per cubic millimeter) of white blood cells based on age are:

The normal range for a pregnant women in the 3rd trimester is 5,80013,200 per cubic millimeter.

If a person's body is producing more white blood cells than it should be, doctors call this leukocytosis.

A high white blood cell count may indicate the following medical conditions:

Surgical procedures that cause cells to die can also cause a high white blood cell count.

If a person's body is producing fewer white blood cells than it should be, doctors call this leukopenia.

Conditions that can cause leukopenia include:

Doctors may continually monitor white blood cells to determine if the body is mounting an immune response to an infection.

During a physical examination, a doctor may perform a white blood cell count (WBC) using a blood test. They may order a WBC to test for, or rule out, other conditions that may affect white blood cells.

Although a blood sample is the most common approach to testing for white blood cells, a doctor can also test other body fluids, such as cerebrospinal fluid, for the presence of white blood cells.

A doctor may order a WBC to:

The following are conditions that may impact how many white blood cells a person has in their body.

This is a condition wherein a person's body destroys stem cells in the bone marrow.

Stem cells are responsible for creating new white blood cells, red blood cells, and platelets.

This is an autoimmune condition wherein the body's immune system destroys healthy cells, including red and white blood cells.

HIV can decrease the amount of white blood cells called CD4 T cells. When a person's T cell count drops below 200, a doctor might diagnose AIDS.

Leukemia is a type of cancer that affects the blood and bone marrow. Leukemia occurs when white blood cells rapidly produce and are not able to fight infections.

This condition causes a person's body to overproduce some types of blood cells. It causes scarring in a person's bone marrow.

Whether or not a person needs to alter their white blood cell count will depend on the diagnosis.

If they have a medical condition that affects the number of white blood cells in their body, they should talk to a doctor about the goals for their white blood cell count, depending on their current treatment plan.

A person can lower their white blood cell count by taking medications such as hydroxyurea or undergoing leukapheresis, which is a procedure that uses a machine to filter the blood.

If a person's white blood cell count is low due to cancer treatments such as chemotherapy, a doctor may recommend avoiding foods that contain bacteria. This may help prevent infections.

A person can also take colony-stimulating factors. These may help prevent infection and increase the number of white blood cells in the body.

White blood cells are an important part of the body's immune system response. There are different types of white blood cell, and each has a specific function in the body.

Certain conditions can affect the number of white blood cells in the body, causing them to be too high or too low.

If necessary, a person can take medication to alter their white blood cell count.

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White blood cells: Function, ranges, types, and more - Medical News Today

BOSTON Helen Obando, a shy slip of a girl, lay curled in a hospital bed in June waiting for a bag of stem cells from her bone marrow, modified by gene therapy, to start dripping into her chest.

The hope was that the treatment would cure her of sickle cell disease, an inherited blood disorder that can cause excruciating pain, organ damage and early death.

Helen, who at 16 was the youngest person ever to undergo the therapy, was sound asleep for the big moment.

It was a critical moment in medical science.

For more than a half-century, scientists have known the cause of sickle cell disease: A single mutation in a gene turns red blood cells into rigid crescent or sickle shapes instead of soft discs. These misshapen cells get stuck in veins and arteries, blocking the flow of blood that carries life-giving oxygen to the body and causing the diseases horrifying hallmark: episodes of agony that begin in babyhood.

Millions of people globally, a vast majority of them Africans, suffer from sickle cell disease. Researchers have worked for decades on improving treatment and finding a cure, but experts said the effort has been hindered by chronic underfunding, in part because most of the estimated 100,000 people in the United States who have the disease are African American, often poor or of modest means.

The disease also affects people with southern European, Middle Eastern or Asian backgrounds, or those who are Hispanic, like Helen.

This is the story of two quests for a sickle cell cure one by the Obando family and one by a determined scientist at Boston Childrens Hospital, Dr. Stuart Orkin, 73, who has labored against the disease since he was a medical resident in the 1970s.

Like many others affected by sickle cell, the Obando family faced a double whammy: not one but two children with the disease, Helen and her older sister, Haylee Obando. They lived with one hope for a cure, a dangerous and sometimes fatal bone marrow transplant usually reserved for those with a healthy sibling as a match. But then they heard about a potential breakthrough: a complex procedure to flip a genetic switch so the body produces healthy blood.

Scientists have been experimenting with gene therapy for two decades, with mixed success. And it will be years before they know if this new procedure is effective in the long term. But if it is, sickle cell disease could be the first common genetic disorder to be cured by manipulating human DNA.

Four weeks after the infusion of stem cells, Helen was strong enough to be discharged. At home, in Lawrence, Massachusetts, on a sofa with her mother by her side, she put a hand over her eyes and started to sob. She and her family wondered: Would it work? Was her suffering really over?

A Familys Nightmare

Sheila Cintron, 35, and Byron Obando, 40, met when she was in the eighth grade and he was a high school senior. They fell in love. Haylee, their first child, was born in 2001, when Cintron was 17.

When a newborn screening test showed that Haylee had the disease, her father asked, Whats sickle cell?

They soon found out.

As the family gathered for her first birthday party, Haylee started screaming inconsolably. They rushed her to the hospital. It was the first of many pain crises.

Doctors warned the parents that if they had another baby, the odds were 1 in 4 that the child would have sickle cell, too. But they decided to take the chance.

Less than two years later, Helen was born. As bad as Haylees disease was, Helens was much worse. When she was 9 months old, a severe blockage of blood flow in her pelvis destroyed bone. At age 2, her spleen, which helps fight bacterial infections, became dangerously enlarged because of blocked blood flow. Doctors surgically removed the organ.

After Helen was born, her parents decided not to have any more children. But four years later, Cintron discovered she was pregnant again.

But they were lucky. Their third child, Ryan Obando, did not inherit the sickle cell mutation.

As Ryan grew up, Helens health worsened. When he was 9, Helens doctors suggested a drastic solution: If Ryan was a match for her, he might be able to cure her by giving her some of his bone marrow, though there would also be major risks for her, including death from severe infections or serious damage to organs if his immune system attacked her body.

As it turned out, Ryan matched not Helen but Haylee.

The transplant succeeded, but her parents asked themselves how they could stand by while one daughter was cured and the sicker one continued to suffer.

There was only one way to get a sibling donor for Helen: have another baby. In 2017, the couple embarked on another grueling medical journey.

Obando had a vasectomy, so doctors had to surgically extract his sperm from his testicles. Cintron had 75 eggs removed from her ovaries and fertilized with her husbands sperm. The result was more than 30 embryos.

Not a single embryo was both free of the sickle cell gene and a match for Helen.

So the family decided to move to Mesa, Arizona, from Lawrence, where the cold, which set off pain crises, kept Helen indoors all winter. The family had already sold their house when they heard that doctors at Boston Childrens were working on sickle cell gene therapy.

Cintron approached Dr. Erica Esrick, a principal investigator for the trial. But the trial wasnt yet open to children.

Figuring Out the Science

Nothing had prepared Orkin for the suffering he witnessed in his 30s as a medical resident in the pediatric hematology ward at Boston Childrens. It was the 1970s, and the beds were filled with children who had sickle cell crying in pain.

Orkin knew there was a solution to the puzzle of sickle cell, at least in theory: Fetuses make hemoglobin the oxygen-carrying molecules in blood cells with a different gene. Blood cells filled with fetal hemoglobin do not sickle. But the fetal gene is turned off after a baby is born, and an adult hemoglobin gene takes over. If the adult gene is mutated, red cells sickle.

Researchers had to figure out how to switch hemoglobin production to the fetal form. No one knew how to do that.

Orkin needed ideas. Supported by the National Institutes of Health and Howard Hughes Medical Institute, he kept looking.

The breakthrough came in 2008. The cost of gene sequencing was plummeting, and scientists were finding millions of genetic signposts on human DNA, allowing them to home in on small genetic differences among individuals. Researchers started doing large-scale DNA scans of populations, looking for tiny but significant changes in genes. They asked: Was there a molecular switch that flipped cells from making fetal to adult hemoglobin? And if there was, could the switch be flipped back?

They found a promising lead: an unprepossessing gene called BCL11A.

In a lab experiment, researchers blocked this gene and discovered that the blood cells in petri dishes started making fetal instead of adult hemoglobin.

Next they tried blocking the gene in mice genetically engineered to have human hemoglobin and sickle cell disease. Again, it worked.

Patients came next, in the gene therapy trial at Boston Childrens that began in 2018.

The trial run by Dr. David Williams, an expert in the biology of blood-forming stem cells at Boston Childrens, and Esrick has a straightforward goal: Were going to reeducate the blood cells and make them think they are still in the fetus, Williams said.

Doctors gave adult patients a drug that loosened stem cells immature cells that can turn into red blood cells from the bone marrow, their normal home, so they floated free in the bloodstream. Then they extracted those stem cells from whole blood drawn from the patient.

The researchers used a disabled genetically engineered AIDS virus to carry information into the stem cells, flipping on the fetal hemoglobin gene and turning off the adult gene. Then they infused the treated stem cells into patients veins. From there, the treated cells migrated into the patients bone marrow, where they began making healthy blood cells.

With the success in adults, the Food and Drug Administration said Boston Childrens could move on to teenagers.

When her mother told her about the gene therapy trial, Helen was frightened. But the more she thought about it, the more she was ready to take the risk.

In the months after the gene therapy infusion at Boston Childrens, her symptoms disappeared.

Helen was scheduled for her six-month checkup Dec. 16. Helens total hemoglobin level was so high it was nearly normal a level she had never before achieved, even with blood transfusions. She had no signs of sickle cell disease.

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At 16, shes a pioneer in the fight to cure sickle cell disease at Boston Childrens - Boston.com

The Cord Blood Stem Cells Market is predicted to worth +6500 Million USD with a CAGR of +30% over the forecast period 2020 to 2025.

Three sources of stem cells are bone marrow, peripheral blood, and cord blood. The blood in the umbilical cord is called cord blood and is collected at the time of delivery. Cord blood is an abundant source of Red Blood Cells (RBCs), white blood cells (WBCs), platelets and hematopoietic stem cells, and is extracted and stored in a private blood bank for the purpose of treating the disease in the future as needed.

Request for Sample Report@ https://www.reportconsultant.com/request_sample.php?id=39736

The global Cord Blood Stem Cells Market analytical report has recently published by Report Consultant to its massive repository. The research report has been summarized with informative and technical details of the dynamics of the market. It has been compiled by using some significant research methodologies such as primary and secondary research techniques. The report also elaborates on the factors which are fueling or hampering the growth of the market. It gives more focus on recent trends and technologies which are boosting the performance of the companies.

Cord Blood Stem Cells Market Key Players:

Cord Blood America Inc, Cryo-Cell International Inc, Cryo-Save AG, Cord Blood Registry Systems Inc, Viacord Inc, China Cord Blood Corporation, Cordlife Group Ltd, Vita 34 AG, Lifecell International Pvt. Ltd, Stemcyte Inc

The Cord Blood Stem Cells Market is segmented by means of storage service, application, and region.

Storage service: Public cord blood bank and Private cord blood bank

Cord blood stem cell market segmentation by application: Blood disease, Cancer, Acute leukemia, Krabbe diseases, and other diseases

Regions: North America (USA, Canada, Mexico), Europe (Germany, France, UK, Italy, Russia), Asia Pacific (China, India, Japan, South Korea, Australia, Indonesia, Malaysia), Middle East and Africa (Bahrain, Egypt, Jordan, Kuwait, Morocco, Oman, Qatar, Saudi Arabia, Syria)

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The report Describes the Cord Blood Stem Cells Market basics like definitions, classifications, applications and industry chain overview, industry policies and plans, product specifications, manufacturing processes, cost structures and so on. Then it analyzed the worlds main region market conditions, including the product price, profit, capacity, production, capacity utilization, supply, demand and industry growth rate, etc. In the end, the report introduced new project SWOT analysis, investment feasibility analysis, and investment return analysis.

Global research Cord Blood Stem Cells Market report highlights:

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What is Cord Blood Stem Cells Market and What Factors will drive the Industry including Leading Players Cord Blood America Inc, Cryo-Cell...

2020 Honorees include Cystic Fibrosis Foundation, Emily Whitehead Foundation, Gift of Life Marrow Registry and Ret. Major General Bernard Burn Loeffke (US Military)

Miami, FL, Jan. 09, 2020 (GLOBE NEWSWIRE) -- The formal ceremony of the 2020 Stem Cell and Regenerative Medicine Action Awards will take place at a gala reception and dinner on January 23, during the 15th annual World Stem Cell Summit (WSCS) at the Hyatt Regency in Miami. Since 2005, the nonprofit Regenerative Medicine Foundation (RMF) (formerly Genetics Policy Institute) has recognized the stem cell and regenerative medicine community's leading innovators, leaders, and champions through its annual awards reception.

Bernard Siegel, Executive Director of Regenerative Medicine Foundation and founder of the World Stem Cell Summit, said, The 2020 Action Awards will recognize three important organizations that are positively impacting the emerging field of regenerative medicine. We will also honor a retired Major General, who has capped off his military and diplomatic career by promoting the cause of world peace through medicine. All of these distinguished honorees will be recognized for their devotion to improving health and developing cures through advocacy, innovation, leadership and inspiration. In addition, the wounded warrior veterans community of South Florida will also receive special recognition at the event.

Meet the 2020 Stem Cell & Regenerative Medicine Action Award Honorees:

Innovation Award: With the motto, We will not rest until we find a cure, the Cystic Fibrosis Foundation is geared towards the successful development and delivery of treatments, therapies and a cure for every person with cystic fibrosis. CF Foundation has added decades to the lives of people with the disease as a direct result of advances in treatment and care made possible through its innovative business model- venture philanthropy. The Foundation recently unveiled its Path to a Cure research agenda aimed at addressing the root genetic cause of the disease and is currently funding industry programs aimed at gene delivery with the goal of progressing into clinical studies in 2021.

Inspiration Award: Emily Whitehead Foundation is a nonprofit organization committed to raising funds to invest in the most promising pediatric cancer research. Tom and Kari Whitehead founded EWF in honor of their daughter Emily, the first child in the world to receive CAR T-cell therapy, training her own cells to fight cancer. Her inspiring story focused public attention on thepotential for cancer immunotherapy to transform cancer treatment,as well as the need to support lifesaving cancer immunotherapy research. The foundation provides support to pediatric cancer patients and promotes awareness of the disease through education and sharing other inspiring stories.

Advocacy Award: Gift of Life Marrow Registry was established in 1991 by Jay Feinberg and his family after Jay received a life-saving bone marrow transplant. Gift of Life is dedicated to saving lives and facilitating bone marrow and blood stem cell transplants for patients with leukemia, lymphoma, sickle cell and other diseases. In 2019, Gift of Life opened the worlds first apheresis center fully integrated within a registry, the Dr. Miriam and Sheldon G. Adelson Gift of Life-Be The Match Stem Cell Collection Center. With the collection center and rapidly expanding donor database, Gift of Life will launch a biobank to advance cellular therapies using allogeneically sourced cells in 2020.

Leadership Award: Ret. Major General Bernard Burn Loeffke, PhD (US Military) is a highly decorated Special Forces officer, diplomat and medical officer.He survived two helicopter crashes and was wounded in combat. After the Vietnam War, he served as the Army Attach at theU.S. Embassy in Moscow, first Defense Attach at the U.S Embassy in Beijing, a staff officer in theWhite House, and Director of the Commission onWhite House Fellows. His last command was Commanding General of Army South. After 35 years in the military, he became a medical officer traveling the world on relief missions to third and fourth world countries. Presently, at age 85, he champions the hydrocephalus and wounded warrior communities. He continues to serve as an inspiration and supporter of building peaceful international relations through medical partnerships and played a pivotal role as a keynote speaker at the inaugural 2019 World Stem Cell Summit CHINA.He is called the Peace General in Latin America. In China, he is simply known as The General, our Friend.

Story continues

To learn more about past honorees and details for sponsoring or attending the upcoming 2020 Stem Cell and Regenerative Medicine Action Awards dinner, please visit, https://www.worldstemcellsummit.com/stem-cell-action-awards/

About the World Stem Cell Summit (WSCS)

Produced by the non-profit Regenerative Medicine Foundation (RMF), and in its 15th year, the World Stem Cell Summit will take place January 21-24, 2020, in Miami, Florida in partnership with Phacilitate Leaders World, as part of Advanced Therapies Week. The Summit is the most inclusive and expansive interdisciplinary, networking, and partnering meeting in the stem cell science and regenerative medicine field. With the overarching purpose of fostering translation of biomedical research, funding, and investments targeting cures, the Summit and co-located conferences serve a diverse ecosystem of stakeholders. For more information about the upcoming World Stem Cell Summit in Miami, please visit: http://www.worldstemcellsummit.com.

About the Regenerative Medicine Foundation (RMF)

The nonprofit Regenerative Medicine Foundation fosters strategic collaborations to accelerate the development of regenerative medicine to improve health and deliver cures. RMF unites the worlds leading researchers, medical centers, universities, labs, businesses, funders, policymakers, experts in law, regulation and ethics, medical philanthropies, and patient organizations. We maintain a trusted network of leaders and pursue our mission by producing our flagship World Stem Cell Summit series of conferences and public days, honoring leaders through the Stem Cell and Regenerative Medicine Action Awards, supporting our official journal partner STEM CELLS Translational Medicine (SCTM), promoting solution-focused policy initiatives both nationally and internationally and creating STEM/STEAM educational projects. For more information about RMF, please visit: http://www.regmedfoundation.org.

Attachments

Joseph DawsonRegenerative Medicine Foundation561-906-4755joseph@regmedfoundation.org

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Stem Cell and Regenerative Medicine Action Awards to be Presented at World Stem Cell Summit on January 23 at the Hyatt Regency Miami - Yahoo Finance

Roshen Confectionery Corporation will allocate UAH 50 million for the overhaul of the oncohematology department and the creation of an autologous bone marrow transplantation department at the National Cancer Institute.

According to the company's press release, the project will last almost two years.

"In the building where the oncohematology and chemotherapy department is located, the roof has been leaking for many years, water leaked from the sewer under the foundation as a result, almost all the walls of the building have a fungus that is simply deadly for people with this disease. In early autumn, Roshen began the overhaul of part of the premises of the second building of the National Cancer Institute. We plan to complete the work in August 2020," Iryna Ponomarenko, the director for social projects development at Roshen Confectionery Corporation, said.

In 2018, the corporation repaired and equipped a room intended for apheresis (collection) of stem cells (for bone marrow transplantation) and donor platelets for a total of UAH 2.9 million.

In total, in 2017-2018 Roshen invested UAH 357 million in social projects.

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Roshen will issue UAH 50 mln for development of National Cancer Institute - Interfax Ukraine

What are the most common types of brain tumors in children, and what treatment options are available?

Brain tumors are the most common solid tumors affecting children, with approximately 4,500 new cases each year in the U.S.

As brain tumors expand or block the normal pathways in the brain, the pressures inside the skull expand. As a result, symptoms of brain tumors can include headaches, seizures, lethargy, nausea and vomiting. A child experiencing progressively worsening symptoms like these should be evaluated by a pediatrician or in the emergency room. The doctors evaluation may include a scan of the brain. If the scan shows a tumor, the next step is a consultation with a neurosurgeon.

The majority of pediatric brain tumors occur in the posterior fossa (located near the bottom of the skull and the brain stem). The most common tumors include medulloblastoma, pilocytic astrocytoma, and ependymoma. Other less common tumors can occur in the cerebral hemispheres (the two main portions of the brain) and include astrocytomas, gangliogliomas, craniopharyngiomas, and germ cell tumors.

Surgery is usually the first step in treatment when a brain tumor is discovered. The goals of surgery are to determine whether the tumor is cancerous and remove all or as much of the tumor as safely as possible. At UVa Childrens Hospital, the latest technologies are utilized to help perform surgery, including intraoperative MRI, navigation, ultrasound and minimally invasive endoscopic surgery. Based on the types of cells found in the brain tumor, additional treatments may be needed. These therapies may include chemotherapy, radiation therapy, proton therapy, stem cell rescue and bone marrow transplantation and/or supportive care for rehabilitation.

More recent treatment options have focused on precision medicine and targeted drug therapy. Targeted drug treatments can cause brain tumor cells to die by blocking abnormalities present within these cells. These drugs are changing how brain tumors are treated while improving outcomes. Current research is focused on understanding the molecular basis of tumor formation and discovery of new targets for treatment.

At UVa, we are committed to providing the best neurosurgical care for children through our multidisciplinary brain tumor team, consisting of neurosurgery, neurology, pediatric oncology and radiation oncology.

For more information, visit childrens.uvahealth.com/services/pediatric-neurosurgery.

Dr. Hasan R. Syed and Dr. John Jane Jr. are pediatric neurosurgeons at UVa Childrens Hospital.

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Ask the Expert: What are the most common types of brain tumors in children? - The Daily Progress

Jasper Therapeutics, Inc., a Palo Alto, Calif.-based biotechnology company focused on hematopoietic cell transplant therapies, expanded its Series A financing with an additional investment of $14.1m.

The round was led by Roche Venture Fund with participation from other investors. This brought the total company financing to more than $50m to date.

The initial Series A round was led by Abingworth LLP and Qiming Venture Partners USA, with further investment from Surveyor Capital (a Citadel company) and participation from Alexandria Venture Investments, LLC.

The company plans to use the proceeds to advance and expand the study of its lead clinical asset, JSP191.

Jasper Therapeutics is a biotechnology company focused on hematopoietic cell transplant therapies. The companys lead compound, JSP191, is in clinical development as a conditioning antibody that clears hematopoietic stem cells from bone marrow in patients undergoing a hematopoietic cell transplant. This conditioning antibody is designed to enable safer and more effective curative hematopoietic cell transplants and gene therapies.

FinSMEs

09/01/2020

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Jasper Therapeutics Raises Additional $14.1M in Series A Financing - FinSMEs

13 January 2020

Promising results from clinical trials give hope for using CRISPR/Cas9 genome editing to treat various heritable diseases and cancer in humans.

It has been seven years since the discovery that the CRISPR/Cas9 defence system, used by microbes to destroy viruses, could be re-engineered to edit the human genome. Since then researchers have carried out an array of experiments to explore potential applications.

Biophysist Dr He Jiankui sparked global controversy concerning the ethics of genome editing when he used CRISPR to genetically modify embryos, resulting in the birth of the first genome-edited babies (see BioNews 977).

Yet researchers worldwide have at the same time been investigating the use of CRISPR for non-heritable changes, modifying the genes in non-embryonic cells to treat a wide range of diseases.

'There's been a lot of appropriate caution in applying this to treating people, but I think we're starting to see some of the results of that work,' said Dr Edward Stadtmauer, a haematologist at the University of Pennsylvania, Philadelphia.

Over a dozen new clinical trials testing CRISPRtherapy on diseases such as cancer, HIV and sickle cell anaemia were listed on the clinicaltrials.gov database last year. One trial in its early stages used CRISPR to treat sickle cell anaemia and beta-thalassaemia, both genetic blood disorders that result in the production of an abnormal form of the oxygen-carrying protein, haemoglobin.

Two patients with these disorders were treated by CRISPR Therapeutics in Cambridge, Massachusetts, and Vertex Pharmaceuticals in Boston, Massachusetts, using CRISPR to inactivate a gene that switches off the production of an alternative form of haemoglobin. Preliminary results of the study suggest that this therapy improved some of the symptoms but the participants will need to be followed for a longer period to be sure.

Results from two other trials, one in which genome-edited blood cells were transplanted into a man to treat HIV infection, and the other in which they were transplanted into three people to treat some forms of cancer, were less successful. In both cases, the transplanted cells flourished in the bone marrow of recipients, without any serious safety concerns, but did not produce a clear medical benefit. The study has been placed on hold while researchers explore ways to boost that percentage, says Hongkui Deng, a stem-cell researcher at Peking University, Beijing, China and a lead author of the work.

Other researchers are trying to move beyond editing cells in vitro. In July 2019 a clinical trial was launched to treat Leber congenital amaurosis 10 (LCA10), a rare genetic disease that causes blindness. The trial, launched by two pharmaceutical companies, Editas Medicine in Cambridge, Massachusetts, and Allergan in Dublin, Ireland, will be the first trial that uses CRISPR to edit cells inside of the body. The researchers are testing AGN-151587 (EDIT-101), which is a novel CRISPR treatment delivered via adeno-associated virus (AAV) directly to the eye's light-sensing photoreceptor cells to remove the mutation that causes LCA10.

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The potential use of CRISPR to treat disease is gaining momentum - BioNews

Last Updated on January 10, 2020

Medically reviewed by Ann S. LaCasce, MD, MMSc

Mantle cell lymphoma is a rare, often aggressive form of non-Hodgkin lymphoma (NHL), a cancer that involves white blood cells known as lymphocytes, which help protect the body from disease. It is named for its origins in the mantle zone a ring of cells within the lymph nodes where B cells (a type of lymphocyte) grow and take on specialized functions. It comprises about 6% of all cases of NHL, usually arises during an individuals early 60s, and is more common in men than women.

The most common symptoms of mantle cell lymphoma include:

At the time of diagnosis,nearly all patients have disease that has spread beyond its initial site.

For most patients, the cause of the disease is unknown, but rates are higher among farmers and people from rural areas.

Itoccurs when B lymphocytes acquire genetic mutations that alter their functionand growth. One such abnormality, found in 90% of cases, causes B lymphocytesto overproduce cyclin D1, a protein that spurs the cells growth. Othermutations can interfere with B cells ability to produce infection-fightingantibodies, leaving patients vulnerable to certain diseases.

A definitive diagnosis requires a biopsy of an affected lymph node or other involved tissue.

Doctors use a variety of scans to determine the diseases stage, or how far it has advanced. These include:

Treatment for mantle cell lymphoma varies depending on patients age and overall health and the stage of the disease. Patients who have yet to develop symptoms and who have a relatively small amount of slow-growing disease may be recommended for active surveillance close monitoring of their health through regular checkups and lab tests. When lymphoma-related symptoms appear or tests show a worsening of the disease, active treatment may begin.

The initial treatment for aggressive mantle cell lymphoma in younger patients often includes a combination of chemotherapy drugs in conjunction with an antibody-based treatment, often followed by a stem cell transplant using patients own stem cells. Older, less-fit patients may undergo less intensive chemotherapy sometimes followed by a prolonged course of antibody therapy.

Other treatments may include drugs known as BTK inhibitors such as acalbrutinib and ibrutinib, which interfere with lymphoma cells internal growth signals.

In patients who relapse after treatment or dont respond to initial treatment, a variety of options may be available, including:

Clinical trials are currently underway of CAR T-cell therapy for patients with mantle cell lymphoma. The therapy, which uses genetically modified immune system T cells to attack tumor cells, has been shown to be effective in patients with other forms of non-Hodgkin lymphoma. Other trials are testing drugs known as bispecific antibodies, artificial proteins that can bind simultaneously to two surface proteins on cells, and targeted agents directed against specific cancer-related proteins.

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What is Mantle Cell Lymphoma and How Is It Treated? - Dana-Farber Cancer Institute

Imago BioSciences, Inc., a clinical-stage biotechnology company developing innovative treatments for myeloid diseases, today announced that the U.S. Food and Drug Administration (FDA) has granted Fast Track designation for the development of bomedemstat (IMG-7289) for the treatment of essential thrombocythemia (ET), a bone marrow disease associated with high platelet counts and potentially catastrophic vascular complications. Bomedemstat inhibits the enzyme LSD1 (lysine-specific demethylase 1), thus preventing excess platelet and neutrophil production.

"The Fast Track designation by the FDA recognizes the need for novel therapeutics for myeloid diseases and mirrors our own urgency in addressing these devastating conditions," said Hugh Young Rienhoff, Jr. M.D., CEO, Imago Biosciences. "ET is a quiet bone marrow cancer than can linger for years. In a subset of patients, the excess of platelets leads to bleeding and clotting including strokes and infractions, each having a significant impact on these patients. With only one FDA approved therapy, one that does not increase overall survival, patients are in desperate need of new options. Based on its mechanism and safety data obtained to date, we believe bomedemstat has the promise to be that new treatment."

The FDA grants Fast Track designation to facilitate development and expedite the review of therapies with the potential to treat a serious condition where there is an unmet medical need. A therapeutic that receives Fast Track designation can benefit from early and frequent communication with the agency, in addition to a rolling submission of the marketing application, with the objective of getting important new therapies to patients more quickly.

About Bomedemstat (IMG-7289)

Bomedemstat is a small molecule discovered by Imago BioSciences that inhibits lysine-specific demethylase 1 (LSD1 or KDM1A), an enzyme essential for production and normal function of megakaryocytes and for self-renewal of malignant hematopoietic stem or progenitor cells. Megakaryocytes are the primary producer of platelets and cytokines that drive essential thrombocythemia pathogenesis.

In non-clinical studies, bomedemstat demonstrated robust in vivo efficacy as a single agent, and in combination with other therapeutics across a range of myeloid malignancy models including the myeloproliferative neoplasms encompassing myelofibrosis, essential thrombocythemia and polycythemia vera.

The FDA has also granted Fast Track designation to bomedemstat for the treatment of myelofibrosis, which is currently being studied in an international Phase 2b study. In this study IMG-7289 was effective in reducing spleen volumes and substantially improved symptom scores in a majority of evaluable patients. For more information visit http://www.clinicaltrials.gov (NCT03136185). Additional clinical studies in hematologic disorders will begin in 2020.

About Imago BioSciences

Imago BioSciences is a clinical-stage, venture-backed pharmaceutical company whose investors include a fund managed by Blackstone Life Sciences, Frazier Healthcare Partners, Omega Funds, Amgen Ventures, MRL Ventures Fund, HighLight Capital, Pharmaron, Greenspring Associates and Xeraya Capital, as well as other corporate and venture investors.

View source version on businesswire.com: https://www.businesswire.com/news/home/20200113005164/en/

Contacts

Ian StoneCanale Communicationsian@canalecomm.com (619) 849-5388

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Imago Receives Fast Track Designation from U.S. FDA for Bomedemstat for Treatment of Essential Thrombocythemia - Yahoo Finance

This week, the American Cancer Society released some very welcome news: the cancer death rate in the U.S. dropped by 2.2% from 2016 to 2017, the largest single-year drop ever recorded.

The drop, which the report attributes to plummeting smoking rates as well as new screening and treatment methods, continues a decades-long trend, as cancer death rates have fallen by nearly 30% since 1991 about 2.9 million fewer deaths.

Dr. Thomas Loughran, director of the University of Virginia Cancer Center, said UVA is in step with this national trend.

The UVA Cancer Center is one of 71 National Cancer Institute-designated treatment centers nationwide and ranked among the nations top 50 cancer centers over each of the past four years (No. 26 last year). The center serves approximately 4 million people in Virginia and West Virginia.

We spoke with Loughran about what he is seeing at UVA and beyond, new treatments and research helping to eradicate cancer, and where he sees cancer treatment in five years.

Q. Why have cancer death rates dropped so significantly?

A. As reports of this latest drop have said, a large part of the decline can be attributed to declining rates of lung cancer. The importance of preventing cancer particularly behavioral interventions like stopping smoking has become more prominent, and there have been remarkable declines in smoking across the United States.

This is a very important focus for us at UVA. We serve a large geographical area 90 contiguous counties in Virginia and West Virginia, including rural Appalachia. Southwest Virginia in Appalachia still has high smoking rates, and as a result, high rates of lung cancer. Education, screening and tobacco cessation programs are critically important, especially in those areas.

Q. What advances in treatment have contributed to falling cancer death rates, nationally and at UVA?

A. Screening technology, especially for the more common cancers like lung, colorectal, prostate and breast cancer, has improved. The latest report probably doesnt fully reflect recent implementation of lung cancer screening using a low-dose CT scan, recommended for high risk individuals and especially those with a history of heavy smoking. That has only been around a few years, and its impact will likely show up in future reports.

The second big factor is the development of immunotherapy [cancer treatments that utilize and help the patients immune system]. UVA has invested quite a lot of institutional resources in becoming a state-of-the-art immunotherapy center, and I am proud to say we are a leader in the field.

We have created a Cancer Therapeutics Program to support the development of new therapies. Dr. Craig Slingluff, who leads that program, is a surgical oncologist internationally famous for immunotherapy treatments for melanoma. To strengthen this program, we have recruited a cadre of leading physician scientists from across the country. Dr. Karen Ballen came here to lead our stem cell and bone marrow transplant program. Dr. Lawrence Lum, the scientific director of the transplant program, has developed a novel therapy using antibodies that bind to both T-cells [patient cells that can kill cancer cells] and tumor cells, forming a bridge between the two that helps the T-cells kill the cancer cells. Dr. Trey Lee is a leader in CAR-T cell therapy.

I could keep going; there are so many great people working on this. We also have a new Good Manufacturing Practice lab, supported by a grant from the commonwealth, that will help us grow and modify T-cells as needed and give them to patients under sterile conditions. That just opened and we are very excited about that program.

Q. What other areas of research have shown great promise?

A. Some of our work in nanotechnology is really unique and exciting. [Biomedical engineering professor] Mark Kester directs UVAs nanoSTAR Institute, which is working on delivering cancer therapies by nanotechnology basically, engineering at a very small scale. For example, nanoliposomes a sort of delivery system for cancer therapy are actually smaller than individual cells and can therefore penetrate cancer cells and release treatment from inside those cells.

We are very excited about early phase trials testing this technology on solid tumors, and we also hope to use it to treat patients with acute leukemia over the next few years.

Q. Looking ahead, where do you see the next big gains coming from?

A. Immunotherapy has revolutionized cancer treatment, but why some patients respond well and some dont remains puzzling. I hope that we can begin to discover why some patients are reacting to these newer treatments differently than others. Once we figure out why some patients respond to immunotherapy, we can begin to make improvements that could benefit a larger percentage of patients with these deadly cancers.

CAR T Cell therapy one method of immunotherapy is very effective against leukemia, lymphoma and cancers of the blood, but not yet against solid tumors. Over the next five years, I hope we can determine how to deliver these T-cells to solid tumors such as those found in lung, colorectal and other common cancers again to make this advance more widely applicable to a larger number of patients.

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Q&A: Cancer Death Rates Are Falling Nationally. Here's What's Happening at UVA - University of Virginia

There was a head-scratching moment when Martin Swales answered his front door and a priest handed him a letter.

The mystery was quickly solved. It contained a thank you note from someone whose life Martin had saved.

He knew his bone marrow had been given to someone called Jan and imagined it was a woman in Britain.

In fact the recipient was dad Jan Zmek 4,500 miles away in the US.

And Martins gift of life has led to an extraordinary 30-year bond between the pair, who are like blood brothers.

Jan named his second daughter Martina in honour of his hero and Martin is godfather to his third girl.

Retired welder Martin, 58, of Guisborough, North Yorks, said: Donating bone marrow didnt just save Jans life, it changed mine as well.

The first time I met Jan, I put my arms around him and he hugged me back.

It felt natural, like I was welcoming my brother. It feels like our two families have become one.

They each have three grown-up children and have visited each other for baptisms, graduations, and weddings.

Martin recently went to Switzerland, where Jan lives with his family, to celebrate 30 years since the transplant and present his blood brother with a Walk of Fame plaque.

It includes the touching message: Stood strong, fought hard, and won. You are a survivor.

The mens amazing and heart-warming story dates from 1986 when Martin joined the Anthony Nolan stem cell register after an appeal to save two girls living in the North East.

He was not a match for the girls but in 1989 was called by the register because he could be for Jan.

Martin said: It was quite a shock because Id pretty much forgotten about the register. They told me I was a possible match for someone and what was involved. I said yes straight away. I wanted to help if I could.

Despite the discomfort, Martin gave bone marrow from his hip at a clinic in Harley Street that August. Doctors extracted it from inside his hip using a long needle. Today most donations are no more invasive than giving blood.

Martin spent two nights in hospital. He said: It doesnt take long but at the time I was suffering from sciatica so I think I found it a bit more painful than most. It was an uncomfortable journey home on the train.Anthony Nolan covered the cost of the trip.

Jan, a 27-year-old dad, was diagnosed with leukaemia in 1987. Initially doctors kept the news from him as no treatment was available in the Czech Republic, where he lived.

Jan said: I was diagnosed one year after the Chernobyl tragedy, weve never known if that radiation was to blame for my cancer. I suddenly grew very tired, nobody knew the reason.

I didnt know how sick I was because the doctors wouldnt tell me.

My wife, who was then my girlfriend, went to the same doctors and they told her, Dont marry this guy, dont have children with him. He is going to die in two years.

But Radka ignored their warning and insisted on marrying Jan in 1987.

His only hope was a bone marrow transplant. Weeks later he left for the US with his dad, who planned to be his donor.

Jan said: A few months earlier, I read in the paper the opera singer Jos Carreras was diagnosed with a similar blood disease and was going to the same US centre for a transplant.

They arrived with less than 40 in their pockets and discovered a transplant from his dad would give Jan only a 15 per cent chance of survival.

Instead doctors advised them to find a donor. It took two years and 10,000 to test potential donors before they found a perfect match in Martin.

By then Jan and Radka had become parents to their first daughter, Jana.

Jan needed to raise more than 100,000 to fund the transplant.

He said: It was such a huge amount of money to raise but when you are dying you have no choice.

There were 12 rival local radio stations but they all got together to run a joint appeal, which they broadcast at the same time. It was incredible.

Jan did a sponsored run, gave talks about his ordeal to church congregations to request donations, and wrote to celebrities, especially those with links to the Czech Republic.

Donald Trump s ex-wife Ivana gave 1,000, as did One Flew Over the Cuckoos Nest director Milos Forman. Jan said: The response was crazy. So many people donated 20 dollars or 50.

Martins bone marrow was flown to the Fred Hutchinson Cancer Research Center in Seattle, where Jan was waiting in an isolation room.

He had been blasted with chemo and radiotherapy so his immune system would not attack Martins transplanted cells.

Normally, under strict anonymity rules to protect donor and recipient, Martin and Jan would have been unable to contact each other for years.

But a priest from the North East of England working at the hospital recognised Martins address when the bag of bone marrow arrived.

He offered to take a photo of Jan, a thank you letter, and a Czech garnet stone to Martin when he returned home in 1990.

Martin said: I was stunned. I had no idea my bone marrow had travelled so far. Knowing Id helped a young father, just like me, brought home how important it was and how easily it could have been me waiting for a stranger to save my life.

I wrote straight back. The priest also brought a letter from a couple whose daughter was in the same hospital.

Her transplant didnt work. Sadly she died, but they wrote to thank me for saving Jan. Responding to them was much harder. How do you find the right words?

Martin and Jan kept in touch. When Jans second daughter was born in 1991, he and Radka named her after Martin.

Jan said: How do you repay someone who saved your life? Naming our daughter after Martin was our way of showing him we would never forget what he did for us.

Hes not just the man who saved my life. He is a nice guy. Thats why were so close.

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Jan, 59, and his family moved to Switzerland, where he landed a job with a sports marketing firm that works with World Athletics.

In 1992 his job brought him to Crystal Palace in South London and he spent a few days with Martin and family.

Jans youngest daughter Michaela was born in 1995 and he invited Martin and his family to Switzerland for the baptism and asked him to be godfather.

The two families continued to visit each other and holidayed together in the Czech capital Prague. When Jans eldest, Jana, was studying at Newcastle University, she regularly spent weekends with Martin and his wife Tracey.

Martin said: It meant so much to visit Jan for the 30 anniversary of his transplant earlier this year.

"They showed us the sights and we went up the mountains. It was brilliant. I could never have imagined this when I joined the stem cell register all those years ago.

He added: I hope Martin and I will be able to celebrate another anniversary together in ten years.

The Anthony Nolan register matches potential donors to patients needing stem cell transplants and does vital research. To join, donate or find out more, see anthonynolan.org .

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Bone marrow donor's amazing 30 year bond with man he saved - Mirror Online

There is also another option on the table: a technology called haplo-identical, where they could use the stem cells from my brother, who is a 50 per cent match.

But it shouldnt have been this hard to find a match, and thats whyI started my campaign to sign more people up to the transplant list.I want to make a difference for other people who have to go through this.

If I dont make it, I want to leave a legacy that the children can look at when theyre older and know that Mummy did everything she could to fight this thing. There can only be one winner with this disease, and it needs to be me.

As told to Jessica Salter

Leukaemia Care is one of three charities supported by this years Telegraph Christmas Charity Appeal. Our others are Wooden Spoon, which works with the rugby community to raise money for disabled and disadvantaged children,and The Silver Line, a telephone support service for lonely elderly people. To donate,visit telegraph.co.uk/charity or call 0151 284 1927 before the end of January

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My agonising two-year wait for a stem-cell donor after being diagnosed with leukaemia - The Telegraph

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MCLEAN, United States (AFP) When a person's immune system is impaired by a genetic disease a bone marrow transplant can be a powerful therapeutic tool, but with a major downside during the first few months the recipient's defences against viruses are severely weakened. The slightest infection can lead to a hospital trip.

A still-experimental type of treatment known as T-cell therapy aims to assist during this vulnerable period the months during which the body is rebuilding its natural defences. After two decades of clinical trials, the technology has been refined and is being used to treat more and more patients, many of them children.

A boy named Johan is one of them.

Today he is a mischievous, smiling toddler, with a thick shock of light-brown hair, who never tires, playfully tormenting the family's puppy, Henry.

There is no sign of the three-year-long medical and emotional roller coaster ride he and his family, who live in an affluent Washington suburb, have been on.

The first traumatic surprise came with the results of a pregnancy test Johan was not planned.

That was a huge shock. I cried, said his mother, 39-year-old Maren Chamorro.

Risky procedure

She had known since childhood that she carried a gene that can be fatal in a child's first 10 years, chronic granulomatous disease (CGD).

Her brother died of it at the age of seven. The inexorable laws of genetics meant that Maren had a one in four chance of transmitting it to her child.

For their first children, she and her husband Ricardo had chosen invitro fertilisation, allowing the embryos to be genetically tested before implantation.

Their twins Thomas and Joanna were born both disease-free seven and a half years ago.

But in Johan's case, a post-birth genetic test quickly confirmed the worst: He had CGD.

After conferring with experts at Children's National Hospital in Washington, the couple took one of the most important decisions of their lives, Johan would receive a bone marrow transplant a risky procedure but one that would give him a chance of a cure.

Obviously, the fact that Maren had lost a sibling at a young age from the disease played a big role, Ricardo confided.

Bone marrow, the spongy tissue inside bones, serves as the body's factory for the production of blood cells both red and white.

His brother's immune system

Johan's white blood cells were incapable of fighting off bacteria and fungal infections. A simple bacterial infection, of negligible concern in a healthy child, could spread out of control in his young body.

Luckily, Johan's brother Thomas, six years old at the time, was a perfect match. In April 2018, doctors first cleansed Johan's marrow using chemotherapy. They then took a small amount of marrow from Thomas's hip bones using a long, thin needle.

From that sample they extracted supercells, as Thomas calls them stem cells, which they reinjected into Johan's veins. Those cells would eventually settle in his bone marrow and begin producing normal white blood cells.

The second step was preventive cell therapy, under an experimental programme led by immunologist Michael Keller at Children's National Hospital.

The part of the immune system that protects against bacteria can be rebuilt in only a matter of weeks; but for viruses, the natural process takes at least three months.

Hurdles remain

From Thomas's blood, doctors extracted specialised white blood cells T-cells that had already encountered six viruses.

Keller grew them for 10 days in an incubator, creating an army of hundreds of millions of those specialised T-cells. The result: A fluffy white substance contained in a small glass vial.

Those T-cells were then injected into Johan's veins, immediately conferring protection against the six viruses.

He has his brother's immune system, said Keller, an assistant professor at Children's National.

Johan's mother confirmed as much: Today, when Thomas and Johan catch a cold they have the same symptoms, and for nearly the same amount of time.

I think it's pretty cool to have immunity from your big brother, Maren Chamorro said.

This therapeutic approach boosting the body's immune system using cells from a donor or one's own genetically modified cells is known as immunotherapy.

Its main use so far has been against cancer, but Keller hopes it will soon become available against viruses for patients, like Johan, who suffer from depressed immune systems.

The chief obstacles to that happening are the complexity of the process and the costs, which can run to many thousands of dollars. These factors currently restrict the procedure to some 30 medical centres in the United States.

For Johan, a year and a half after his bone marrow transplant, everything points to a complete success.

It's neat to see him processing things, and especially play outside in the mud, his mother said.

You know, what a gift!

Her only concern now is the same as any mother would have that when her son does fall ill, others in the family might catch the same bug.

Now you can read the Jamaica Observer ePaper anytime, anywhere. The Jamaica Observer ePaper is available to you at home or at work, and is the same edition as the printed copy available at http://bit.ly/epaperlive

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The supercells' that cured an infants genetic illness - Jamaica Observer

Researchers at Baylor College of Medicine have discovered a new mechanism that helps maintain and repair bones in adults. Ultimately, this could help develop new therapeutic strategies to improve bone healing.

Knee Bones

Osteoporosis is a skeletal disease characterized by reduced bone density and changes in the microarchitecture of bones. These changes weaken the bone and increase the risk of fractures.

Osteoporosis develops particularly in older people. Today, a new study could eventually lead to the development of therapeutic strategies to improve bone regeneration in these patients. Results published in the journal Cell Stem Cell on the 5th December 2019 have laid out a new mechanism that contributes to the maintenance and repair of bones in adults.

Adult bone repair relies on the activation of bone stem cells, which still remain poorly characterized. Bone stem cells have been found both in the bone marrow inside the bone and also in the periosteum: the outer layer of tissue that envelopes bone. Previous studies have shown that these two populations of stem cells share many characteristics; however, they also have unique functions and specific regulatory mechanisms, said Dr. Dongsu Park, assistant professor of molecular and human genetics, pathology and immunology at Baylor College of Medicine.

Of these two populations, periosteal stem cells are the least known. Although scientists know that this is a heterogeneous population of cells that can contribute to the thickness, formation, and repair of bone fractures, no one has yet been able to distinguish between the different subtypes of bone stem cells in order to study the regulation of their different functions.

Read Also:HGH Is Now A Solid Treatment For Osteoporosis According To Studies

Here, however, Dr. Dongsu Park and colleagues were able to develop a technique in mice to identify different subpopulations of periosteal stem cells, define their contribution to the repair of bone fractures and identify the specific factors that regulate their migration and proliferation under physiological conditions.

The researchers identified a specific subset of stem cells that contribute to lifelong bone regeneration in adults. They also observed that periosteal stem cells react to inflammatory molecules, chemokines, which are normally produced in bone injuries.

In detail, periosteal stem cells have receptors that bind to the CCL5 chemokine. The CCL5 chemokine sends a signal to the cells to migrate to the injured bone and repair it. By suppressing the CCL5 gene in rats, the researchers found defects in bone repair that delayed healing. However, when they gave CCL5 to rats that had lost CCL5, the bones recovered faster.

Read Also:The Exciting Future of Joint and Cartilage Repair

Our findings contribute to a better understanding of the healing of adult bones. We believe this is one of the first studies to show that bone stem cells are heterogeneous and that different subtypes have unique properties that are regulated by specific mechanisms, said Dr. Dongsu Park.

In conclusion, this study has allowed for the identification of different stem cell subtypes and their distinguishing markers and their roles in bone repair. This discovery gives insight into new therapeutic strategies for the treatment of bone damage in adults, particularly in the setting of osteoporosis or diabetes. Indeed, people with diabetes may be prone to falls and fractures due to neurological, visual or renal complications. In addition, bone fragility in diabetics is likely to be due to changes in bone remodeling and, in particular, an increase in bone resorption.

Read Also:Implants from Own Stem Cells May Offer Solution to Back Pain, Researchers Say

https://www.cell.com/cell-stem-cell/fulltext/S1934-5909(19)30458-8?

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Researchers at Baylor College of Medicine Discover How to Improve Bone Repair - Gilmore Health News

When Allison Shatford was diagnosed with aplastic anemia in 1984 she was given just a 20 per cent chance of survival.

Before her diagnosis, the now 58-year-old described having headaches, bruising, prolonged periods and lethargy.

Eventually it became too much and she was taken to the Leicester Royal Infirmary (LRI) when she was just 23-years-old.

That's when she was given her diagnosis, two years after she first noticed that something might be wrong.

She spent seven years in and out of hospital before doctors gave her another shot at life.

Now, 28 years since her life-saving procedure Alison has written a book about the "frightening" experience.

"I can remember when I was in Florida with my friend and I could not stop bleeding, it was traumatic and that was two years before I was taken into hospital," she said.

"My bone marrow had completely shut down, it didn't make any red blood cells, plasma or oxygen.

"For the next seven years from the age of 23 I was dependant on the blood and platelet donors three times a week for seven years.

"As a young person at 23 my life was taken by this, but I fought it, I never wanted to die."

In her book she said that doctors told her she was only the 13th person to be diagnosed with aplastic anemia after she underwent a series of bone marrow tests.

Aplastic anemia is a serious condition that affects the blood and can happen suddenly.

It is also called bone marrow failure.

Essentially, the condition means that the bone marrow and stem cells don't produce enough blood cells.

There are three types of blood cell: red, white and platelets.

Red blood cells carry a protein called haemoglobin which carry oxygen around the body.

White blood cells help to fight bacterial infections and viruses.

Platelets help blood to clot.

There are multiple symptoms of aplastic anemia, they are: Fatigue, shortness of breath, rapid or irregular heart rate, pale skin, frequent or prolonged infections, unexplained bruising, nosebleeds and bleeding gums, prolonged bleeding from cuts, rashes, dizziness and headaches.

The condition is normally found withing children and adults over the age of 60.

According to the NHS the best form of treatment is a bone marrow transplant - without treatment the condition can be fatal.

Alison had hormone procedures in order to treat the condition, and she said that doctors at the LRI told her they had never seen anything like it.

She said that from 1984 to 1991 she was constantly in and out of the LRI as well as Hammersmith Hospital in London who were helping with treatment.

"I had a hormone procedure mainly used for aplastic anemia and that made me so much more poorly, I was desperate.

"Six months later I had the second course, the treatment involved hormones from horses and rabbits.

"Everyone would bring me carrots, lettuce and apples," she laughed.

"One week before my 30th birthday the doctors said 'Alison it doesn't look good.'

"Leukemia cells had been laying dormant now they had woken up - I had aplastic anemia and luekemia."

She said the medical professionals said that she had one chance and that was a bone marrow transplant.

After testing members of her family for a potential bone marrow donor, her sister Annette was found to be a match.

"It was so unbelievable it was like looking through a haystack to find a needle, that's how rare it was," she said.

"My sister was a match, she was always on the plane coming backwards and forwards for tests; she lived in Ireland.

"I was given a 20 per cent chance of survival before I travelled to hospital for treatment.

Alison said that she started to get her finances in check and had planned her own funeral because she did not want to burden her family in case she didn't come home.

Fortunately after the transplant and extensive chemotherapy, Alison survived.

"In 1991 they said 'expect to be in there for about 15 weeks'.

"After 4 weeks I came home," she said.

"The only set back I had was in the February when I got pneumonia and that was the only time I couldn't control my own body, I was given morphine that knocked me out, but I'm still here."

Allison also had a stroke seven years ago, but again survived after having a blood clot removed before it reached her brain.

The 58-year-old former carer from Oadby now owns and runs a shop on Welford Road called Alison's Bits & Bobs.

From that shop she has helped to raise over 2000 for charities such as the Anthony Nolan trust.

She has also now written a book about her life.

"I have had my shop here for two years and have given 2000 already.

"I've walked Ben Nevis, sky dived you name it, I've done it.

"I have a portfolio on my shop window of all the media cuttings from 1991.

"In the first piece that was published I said I would write a book, so I did.

"I started writing it in February in my shop and it took me four weeks, I'm delighted with the book."

The book is filled with her memoirs that detail her struggles throughout her life in great depth.

The money made from sales of the 500 copies she has had printed will be donated to the LRI Haematology ward, Hammersmith Hospital and the Anthony Nolan Trust.

Alison lives with an unrivaled sense of positivity despite her ordeals in life.

"I love it here, I can educate and talk and listen to everyone.

"I think everyone deserves a shot at life, I'm still here, I wouldn't want to be anywhere else.

"I just want to give people hope and strength to get through like I did and that's all I can ask for."

You can purchase of a copy of Alison's book at her shop in Welford Road for 14.99.

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The woman who spent 7 years in and out of hospital battling rare condition some doctors had never seen - Leicestershire Live

Colleen LeCours lay in a hospital bed at the General campus of The Ottawa Hospital on August 12, 2016, waiting for the only thing that could save her life a stem cell transplant from a stranger.

The donor could be anywhere in the world if a related blood donor cant be found, the call to find a match goes out to registries all over the globe and the donated stem cells are rushed across international borders.

What LeCours didnt know is that her donor, an 18-year-old Carleton University student named Timothy White, was just one floor below. Similarly, White didnt know that his recipient was in the same hospital.

There are currently more than 450,000 people on the Canadian Blood Services Stem Cell Registry formerly known as OneMatch and 36 million on affiliated international registries. Still, some people never find a match. There are more than 900 Canadians in need of a transplant who have not found a match anywhere in the world.

What were the odds that the match for LeCours, now 57, would be found in the same city?

Astronomical, she said.

The chances that White would even ever be asked to donate were also very low only about one in a thousand. After he agreed to donate, he was not told where the recipient might be. I was told the recipient could be anywhere. They could be in Africa, said White, now 22 and a recent graduate in computer science.

White had signed up for the registry through a cheek swab booth at ComiCon less than six months earlier. A smart place to recruit would-be stem cell donors, he notes. The optimal donor is a male between the age of 17 and 35 and thats the ComiCon demographic.

He decided to register as a potential donor because he grew up in the scouting movement. One of the main philosophies is to do a good turn every day, he said.

The donation was a non-surgical procedure in which Whites blood was removed though a needle, the stem cells were separated from his blood and the remaining blood components returned to his body through another needle. The procedure started at about 8 a.m. and was over by about 5 p.m.

I figured if I gave someone a day for a thousand more days (of life) then I felt it was a fair trade. I have many years of life. Why not spend one day? said White.

LeCourss medical journey started in 2009 with an emergency room visit for abdominal pain. She was eventually diagnosed with Stage 4 follicular lymphoma, a blood cancer that affects infection-fighting white blood cells. At the time, LeCours was working for Gov.-Gen. Michalle Jean and was able to stay on the job most of the time during her six months of treatment.

Four years later, the lymphoma returned. It was back again two years after that, in a more aggressive form. The only treatment was stem cell transplant.

There are two main kinds of stem cell transplants autologous and allogenic. In an autologous transplant, stem cells are collected from a patients own blood and reintroduced after being treated to remove cancer cells. In an allogenic stem cell transplant, the stem cells come from a donor.

At this point, LeCours was a candidate for an autologous transplant. Once again, she underwent aggressive chemotherapy. A year later, the cancer returned.

Doctors told LeCours there wasnt much else they could do and advised her to get her affairs in order. But the hospitals transplant team felt she could be a candidate for an allogenic transplant. Theres risk rejecting donated stem cells can be fatal to the patient.

LeCours learned that her brother was a match. But the medical work-up would last about three months and she couldnt wait that long.

I wasnt sure I wanted to do it but I didnt have much choice, she said. They said, We have someone waiting in the wings.

And I said, He probably has wings.

After the transplant, LeCours recovered as an outpatient in the home of her brother and sister-in-law. It took three months to rebuild her immune system. Her only rejection symptoms were a bit of skin irritation.

In January 2018, LeCours received an email asking if she would like to exchange contact information with her donor. She replied that she would.

A few months later, she got a message with Whites co-ordinates and was astonished to find that her donor was in Ottawa. It took her a few weeks to formulate an email.

I didnt want to scare him. I just wanted him to know how incredibly grateful I was. And I wanted to pay it forward, said LeCours.

After careful consideration, she sent White an email on Oct. 8, 2018.

Today, being Thanksgiving, I have so much to be thankful for, namely you giving your stem cells and saving my life and the success of the stem cells grafting to my bone marrow, LeCours wrote. I cant thank you enough for your wonderful selfless act.

Stem cell donor 18-year-old Carleton University student Timothy White at The Ottawa Hospital, General campus, donating stem cells for Colleen LeCours in August 2016. At the time he did not know that LeCours would be the recipient. Courtesy Timothy White.jpg

She added that she didnt know anything about him except for his name and email address, and asked if they could meet. They got together for the first time over lunch in a burger restaurant.

As soon as I saw him, I broke down, said LeCours.

It has been three and a half years since the transplant and LeCours remains in remission. She invited White to her familys Thanksgiving this year, and the two meet to catch up every few months. Its one of the quirks of stem cell donation that the recipient assumes the blood type of the donor. LeCours, once O-positive, now has blood type A-negative, like White.

Im a grandmother. The fact that my grandson has his moma is huge.

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Originally posted here:
ASTRONOMICAL ODDS: Stem cell recipient and her donor both from Ottawa - Ottawa Sun

Military personnel walk past the Raytheon Missile stand.

Carl De Souza | AFP | Getty Images

Check out the companies making headlines in midday trading:

Raytheon, Lockheed Martin, L3Harris Equity of major aircraft and weapons manufacturers Raytheon, Lockheed Martin and L3Harris rose 1.6%, 3.8% and 3.3%, respectively, in midday trading as U.S.-Iranian tensions flare in the Middle East. The U.S. confirmed it was responsible for a drone strike in Baghdad on Friday that killed Iranian Gen. Qasem Soleimani, Tehran's top military commander and a prominent political fixture in the region.

Incyte Shares of Incyte plunged 10% Friday after the company announced that a Phase III study showed one of its developmental drugs failed to show results that were statistically superior to a placebo. The drug was aimed at treating a disease that arises when donated bone marrow or stem cells attack their new host.

Tesla Tesla's stock climbed 3.8% on Friday after the automaker reported better-than-expected deliveries for its most recent quarter. The electric car company delivered 112,000 vehicles during the fourth quarter, topping consensus estimates of 106,000. Tesla delivered roughly 367,500 vehicles for the full year, a 50% increase from 2018 and within the range that it had given as guidance.

Bank of America Shares of the top U.S. bank fell 1.5% in afternoon trading after BMO Capital Markets downgraded the equity to market perform from outperform, telling clients its valuation re-rating has "run its course." Analyst James Fotheringham added that Bank of America shares now trade at a premium to their long-term average and suggested investors look to cheaper names like Citi and Morgan Stanley in the big-bank space.

Concho Resources, Apache, Devon Energy Shares of Concho, Apache and Devon all traded higher, following crude prices, after the U.S. killed a top Iranian military leader in an airstrike. Concho and Apache each traded higher by more than 1% while Devon advanced 0.8%.

L Brands Shares of L Brands rose nearly 8% after Bank of America upgraded the retail and apparel company to buy from neutral. The bank's analysts cited a strong Bath & Body Works business, potential for a more stable Victoria's Secret and a high dividend yield as reasons for the upgrade. The bank also raised its price target on the stock to $25 per share from $21, which would be a 49% increase from where the stock closed on Thursday.

Humana Humana rose 1.5% after Goldman Sachs added the health care company to its "Conviction Buy" list and told clients it sees sizable upward revisions to earnings estimates due to the recent repeal of a fee on health insurers.

CNBC's Fred Imbert and Jesse Pound contributed to this report.

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Stocks making the biggest moves midday: L3Harris, Tesla, Apache & more - CNBC