Archive for the ‘Bone Marrow Stem Cells’ Category

Thats how Valerie Mitchell of Joliet is expressing the pre-holiday news that her son Owen Buell, 2, is showing no evidence of disease in regards to the neuroblastoma hes been fighting all year.

We are really happy, Mitchell said. I still cannot believe he is cancer-free. Everyone is really overjoyed about it, especially being around Christmastime.

On Friday, Owen had a number of scans including CT MRI, MIBG and an echocardiogram, along with bone marrow and hearing tests. Mitchell said. All scans came back clear, she said.

He fought as hard as he could and beat cancer, Mitchell said.

But Owen must remain cancer-free for the next five years before the word remission can be used, she said. In addition, Owen also has more treatments ahead of him: six months of immunotherapy, which Mitchell said will be extremely painful and hard on the body.

Owen will need a five to six-day stay in the hospital each month and a pain pump just to receive the treatments, Mitchell said. But the treatment is necessary to eliminate any remaining cancer cells in Owens body; otherwise new tumors or spots of cancer may form.

Were all really tired, Mitchell said. But we can push through knowing that hes going to be cancer-free. Its just one more step and then he should be good.

When Owen was diagnosed in February, he had two tumors and 21 spots of cancer, Mitchell said. His father Brian was working a job and a half at the time and he and Mitchell shared the family van.

Since then, Owen has undergone many scans, a central line placement, five rounds of chemotherapy, surgery to remove tumors, a stem cell harvest, two stem cell transplants that required a three-month hospital stay, 12 rounds of radiation and 10 days of being intubated in the hospitals intensive care unit, Mitchell said.

Owen now also has damage to one kidney and high blood pressure, Mitchell said. The COVID-19 pandemic made treatments even harder on Owen and his family, she added, especially since Owen's brothers Elliott and Bentley, age 4.were just 7 and 4 when Owen was diagnosed.

We didn't have the help everyone was offering in fear Owen would catch this virus, Mitchell wrote on her Facebook page. We couldn't go anywhere in between treatment to cheer Owen up. We couldn't bring him into the store to pick out a new toy or get him out of the house. He couldn't go swimming; he couldn't go to any arcades; he couldn't even have his father or siblings by his side undergoing surgery or chemo. It wasn't/ isnt fair that Owen had to suffer as much as he did. But we are happy he is still here.

Mitchell said the family celebrated Owens good news with pizza, cake and silly string. And she said Owen is going to go crazy with happiness when he sees all the Christmas gifts toys theyve bought for him.

In the meantime, Owen is enjoying the holiday season like any other 2-year-old.

Hes already playing with the Christmas tree bulbs, Mitchell said.

Donate to the "Help for baby Owen Buell and his Family" GoFundMe page at bit.ly/3n0MThy.

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Joliet 2-year-old gets pre-holiday gift: tests that show he's cancer-free - The Herald-News

GAITHERSBURG, Md., Dec. 07, 2020 (GLOBE NEWSWIRE) -- NexImmune, a clinical-stage biotechnology company developing a novel approach to immunotherapy designed to employ the bodys own T cells to generate a specific, potent and durable immune response that mimics natural biology, today announced that City of Hopes Monzr Al Malki, M.D., delivered an oral presentation at the 62nd American Society of Hematology (ASH) Annual Meeting and Exposition featuring initial data from the Phase 1/2 trial of NEXI-001 in AML. Entitled Preliminary Results of the First-in-Human Study of NEXI-001, a Multi-Antigen Specific CD8+ T Cell Product, in Acute Myeloid Leukemia (AML) Patients with Relapsed Disease after Allogeneic Hematopoietic Cell Transplantation (Allo-HSCT) Demonstrate Early Signs of Safety, Tolerability and Robust Immune Responses, the presentation included responses following a single infusion of the experimental therapy.

These data represent safety and tolerability results from the first five patients treated and reflect a median of four months of follow-up with infusion doses ranging from 50-200 million total T cells. As noted by Dr. Al Malki in his presentation, there have been no cases of acute Graft versus Host Disease (aGvHD), Cytokine Release Syndrome (CRS), immune cells-associated neurological syndromes (ICANs), or infusion related reactions (IRRs) reported to-date, nor have there been any treatment-related adverse events (AEs) observed.

Biomarker data characterizing initial immunologic responses for the first three patients analyzed were also shared. Absolute lymphocyte counts, or ALC, were followed over time after the administration of lymphodepleting therapy, and showed a rapid return to baseline levels for each patient assessed (range 3 to 35 days). In addition, data on T cell reconstitution after lymphodepletion demonstrated that a single infusion of NEXI-001 T cells triggered a broad, rapid and robust immune response, inclusive of both CD8+ and CD4+ T cell types. TCR analysis showed the presence, persistence, expansion and migration of individual NEXI-001 T cell clones from the peripheral blood to the bone marrow of each patient. Finally, the immune phenotype of individual T cell subtypes in each NEXI-001 product were maintained in the peripheral blood of each patient at all time points measured, up to two months. These included sustained populations of T stem-cell-like memory and T central memory subtypes.

Early results from this Phase 1/2 trial suggest that infusion of the NEXI-001 product is well-tolerated and capable of triggering early, robust and persistent cell-mediated immune responses, said Dr. Al Malki, the trials lead investigator and associate clinical professor in City of Hopes Department of Hematology & Hematopoietic Cell Transplantation. The initial data are encouraging, and we look forward to dosing more patients with longer follow-up in order to more fully characterize the clinical potential of this exciting new cell therapy.

Relapse after allo-HSCT is the leading cause of death in patients with AML and represents a significant challenge for treating physicians. There are no approved therapies, and current treatment options are limited. Donor lymphocyte infusions (DLIs) represent the current standard of care but are associated with modest Graft versus Leukemia (GvL) responses and high rates of life-threatening GvHD-associated toxicities. There is significant need for new cellular therapies with potential to enhance the benefits of GvL while decreasing the incidence of GvHD-related toxicities.

Han Myint, M.D., Chief Medical Officer at NexImmune, added, While still early in this trial, we believe the initial data reported, combined with the unique and consistent composition of each NEXI-001 product, may offer a cell therapy with potential to decouple the benefits of GvL from the toxicities associated with GvHD, which would be transformative for both allogeneic stem cell transplant patients and the physicians that provide care for them.

About the Phase 1/2 NEXI-001 Clinical TrialThe first clinical trial with NEXI-001 is a prospective, multi-center, open-label, single-arm, dose-escalating Phase 1/2 study that aims to enroll between 22 to 28 patients. The primary objective is to assess the safety and tolerability of a single infusion of NEXI-001 T cells in patients with AML who have either minimum residual disease (MRD) or relapsed disease after a human leukocyte antigen (HLA)-matched allo-HSCT. Secondary objectives include signals of immunologic responses and preliminary anti-tumor activity. Additional analysis will assess the in vivo persistence, proliferation, functionality and TCR repertoire of NEXI-001 T cells as measured in blood and bone marrow samples.

This study includes two phases. The initial Safety Evaluation Phase determines the safety and tolerability of a single infusion of NEXI-001 at escalating dose levels. In the second part of the study, the Dose Expansion Phase, investigators further define safety and will also evaluate the initial efficacy of NEXI-001 T cells at the dose established in the Safety Evaluation Phase. Once a Recommended Phase II Dose has been determined, safety, tolerability and initial clinical response will become the objectives of the expansion phase of the trial, which is expected to begin in [the first quarter] of 2021.

NEXI-001 products contain populations of CD8+ T cells directed against HLA 02.01-restricted peptides from the WT1, PRAME and Cyclin A1 antigens, each of which is commonly over-expressed on AML blasts and leukemic stem cells. Each NEXI-001 product is composed of T cell memory subtypes that combine anti-tumor potency with long-term persistence. Of significance to this Phase 1/2 trial, each patient-specific experimental cell therapy product also contains very low proportions of T cell subtypes with potential to cause GvHD-related toxicities.

About NexImmuneNexImmune is a clinical-stage biotechnology company developing unique approaches to T cell immunotherapies based on its proprietary Artificial Immune Modulation (AIM) technology. The AIM technology is designed to generate a targeted T cell-mediated immune response and is initially being developed as a cell therapy for the treatment of hematologic malignancies. AIM nanoparticles act as synthetic dendritic cells to deliver immune-specific signals to targeted T cells and can direct the activation or suppression of cell-mediated immunity. In cancer, AIM-expanded T cells have demonstrated best-in-class anti-tumor properties as characterized by in vitro analysis, including a unique combination of anti-tumor potency, antigen target-specific killing, and long-term T cell persistence. The modular design of the AIM platform enables rapid expansion across multiple therapeutic areas, with both cell therapy and injectable products.

NexImmunes two lead T cell therapy programs, NEXI-001 and NEXI-002, are in Phase 1/2 clinical trials for the treatment of relapsed AML after allo-HSCT and multiple myeloma refractory to at least three prior lines of therapy, respectively. The Companys pipeline also has additional preclinical programs, including cell therapy and injectable product candidates for the treatment of solid tumors, autoimmune disorders and infectious diseases.

For more information, visit http://www.neximmune.com.

Media Contact:Mike BeyerSam Brown Inc. Healthcare Communications312-961-2502mikebeyer@sambrown.com

Investor Contact:Chad RubinSolebury Trout+1-646-378-2947crubin@soleburytrout.com

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Preliminary Results from NexImmune's Phase 1/2 Trial of NEXI-001 in AML Presented at 62nd ASH Annual Meeting and Exposition - GlobeNewswire

HealthA new stem cell treatment could restore eyesight in some people

Friday, 4th December 2020, 3:18 pm

Researchers discovered that the cells of damaged retinas could be repaired by injecting genetically modified stem cells into the eye.

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The news comes as comedian Al Murray pushed for stem cell donors to come forward, ahead of a charity gig for blood cancer organisation DKMS.

Heres everything you need to know about the scientific discovery - and how you can donate your own stem cells to save the lives of people with blood cell diseases.

Stem cells are produced by bone marrow, and they have the ability to grow into different types of blood cells such as red and white blood cells and platelets.

A stem cell or bone marrow transplant replaces damaged blood cells with healthy ones and can be used to treat conditions affecting the blood cells, like leukaemia and lymphoma.

The transplant involves destroying the unhealthy blood cells and replacing them with the stem cells removed from the blood or bone marrow.

Often, stem cells are taken from one person - usually a close family member or a match with the same or similar tissue type - and they are transferred to the person that needs them.

How could they be used to treat vision damage?

Researchers in Barcelona recently discovered that modified stem cells could potentially help to cure problems with vision.

They found that the cells of damaged eye retinas send out a rescue signal to attract the stem cells that can repair damage.

Stem cells were genetically engineered to make them more sensitive to those signals.

The modified stem cells were transplanted back into mice and human tissue samples and the researchers found that they flocked to the retina cells in large numbers.

In turn, that kept the tissue of the retina alive and functioning.

The new technique is a breakthrough in stem cell research as it suggests stem cells could help to improve sight, and potentially could cure blindness in the future.

Retinal damage is currently incurable and can cause visual disabilities and blindness, especially in older people.

How can stem cells treat conditions?

Stem cells can already be used to treat a number of conditions where the bone marrow is damaged and unable to produce its own healthy blood cells.

Transplants can be used to treat people suffering from different forms of cancer, with someone elses tem cells replacing the patients blood cells that are damaged or destroyed.

Conditions that stem cell transplants can treat include leukemia and lymphoma, which are cancers affecting white blood cells, myeloma, which affects plasma cells, severe aplastic anaemia (bone marrow failure), and other blood disorders.

A stem cell transplant will usually only be carried out if other treatments have been exhausted, but it could save someones life.

How can I donate stem cells?

When its not possible to use someones own stem cells to treat their condition, they need to come from a donor.

However, to improve the chances of the transplant being successful, the donated cells need to have a very similar genetic marker to the patients.

As the number of donors has recently decreased, charities are urgently encouraging healthy people to donate stem cells.

You are able to register to be a donor on the NHS Blood and Transplant website.

The Anthony Nolan charity also takes sign ups, and is specifically looking for younger donors between age 16 and 30.

You will be asked to fill out an application form and will be sent a swab pack so you can be added to the register.

If you ever come up as a match for a patient, you will be contacted by the charity.

Even if you cant join the register, you can donate to Anthony Nolan to help to grow the stem cell register.

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How do you donate stem cells? Donating cells can help treat cancer, blindness and other conditions - heres how - The Scotsman

Groundbreaking research in stem cells has propelled scientists understanding of neurodegenerative diseases, including Parksinsons. Could stem cell therapies one day help cure Alzheimers?

Clinical trials of stem cell therapies are now underway to repair the damaged cells of people with Parkinsons disease and age-related macular degeneration. Being Patient spoke with Jack Price, professor of developmental neurobiology at Kings College London and author of the book The Future of Brain Repair, about the potential and challenges of repairing the brain with stem cell therapy.

Being Patient: What is stem cell therapy?

Prof. Jack Price: Its the transplantation of stem cells, either directly into the brain or in a way that gives them access to the brain and influence the brain, to bring about a therapeutic effect.

Being Patient: Are there stem cells in the brain?

Prof. Jack Price: For many years, neuroscientists didnt think there were stem cells in the brain. We now know there are. We know about a population [of stem cells] thats become very important in our understanding of Alzheimers disease and in mood disorders like anxiety and depression. These are stem cells that are found in a part of the brain called the hippocampus.

But by and large, the brain doesnt have stem cells, unlike skin and other tissues in the body. The blood is the classic [example]: Theres a population of stem cells in the bone marrow that regenerates blood all the time.

Being Patient: What makes stem cells so special and why are they a focus of research?

Prof. Jack Price: The definition of stem cells is a population of cells that gives rise to other types of cells. In neural stem cells, precursor cells can make adult brain cells, nerve cells, glial cells, all the different cell types that make up the brain. If you have a disease like Alzheimers or any other neurodegenerative disease, where we know the key pathology is the loss of nerve cells, your brain doesnt normally have the ability to replace those lost brain cells. The idea was [that] if you put stem cells where the loss of brain cells has taken place, maybe those stem cells would replace the lost cells.

Being Patient: What is the potential of stem cell therapy in treating neurodegenerative diseases?

Prof. Jack Price: Theres a piece of absolutely brilliant stem cell science that was done by Shinya Yamanaka in Kyoto in 2006. He showed you could effectively take any cell through a very straightforward genetic manipulation that he discovered, [and] turn them into what we call pluripotent stem cells, which are cells that can make any cell type in the body. They also have an ability that other stem cells generally dont: They can build tissue. If you grow them in a little culture dish, they can start to make little pieces of brain called organoids or cerebroids. This was a groundbreaking technology.

In Parkinsons disease, theres enormous progress and clinical trials are underway now. We know more about the pathology of Parkinsons disease [than in Alzheimers]. The pathology of Alzheimers turns out to be quite complex, and weve had, over the years, quite a few ideas about how it worked. But [turning] those into actual therapies hasnt quite worked as we expected, and we keep having to go back and rethink whats going on in Alzheimers.

The pathology of Parkinsons disease is also difficult. Its not trivial. But at the same time, one thing is clear: a lot of the pathology is associated with the loss of a particular population of nerve cells the midbrain dopaminergic cells. We can start with these pluripotent stem cells and make them make precisely the right type of dopaminergic cell that we know is lost in Parkinsons disease.

This is built on 30 [to] 40 years of research of people trying to find exactly the right cell type to work [with] in Parkinsons disease. They had some early success and fell backwards. But this technology looks much more precise than everything anybodys ever tried before.

In age-related macular degeneration, the disease of the eye where you lose your retinal photoreceptors, there are very clever strategies now where people are using these pluripotent stem cells to make a thing called a retinal pigment epithelium. It lies behind the retina, but its what supports the photoreceptors. It turns out, thats what goes wrong in age-related macular degeneration.

Being Patient: Are there any stem cell therapy approved to treat brain disorders?

Prof. Jack Price: There are no licensed stem cell therapy for any brain disorders anywhere in the world for the simple reason [that] nobody has shown one works. There are a lot of stem cell clinics in the U.S. and somewhat fewer elsewhere who are offering cell therapies that are untested. Theyll put stem cells into you for any disorder youve got. Those cell therapies do not work.

A lot of genuine companies are trying to get these cell therapies to work in clinical trials and falling flat on their face quite often, despite their best efforts. 90% of clinical trials fail, and thats clinical trials of conventional drugs by drug companies that know what theyre doing.

What do you suppose is the chance with a stem cell therapy [that] we dont really understand how it works, [that] we dont quite know how to manufacture it properly, [and that] we dont quite know what cells we really want, of working? The chance is almost zero.

The interview has been edited for length and clarity.

Contact Nicholas Chan at nicholas@beingpatient.com

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Repairing the Brain With Stem Cells? A Conversation With Prof. Jack Price - Being Patient

An effective new treatment to restore vision is on the horizon that works by injecting genetically modified stem cells into the eye to mend the damaged retina.

Researchers found that the cells of damaged retinas send out a rescue signal to attract the stem cells that repair eye damage.

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They identified two of these cell signals known as Ccr5 and Cxcr6 and then genetically engineered the stem cells to make them more sensitive to those signals.

When these modified stem cells were transplanted back into mice and human tissue samples in the lab they flocked to the retina cells in much greater numbers, keeping the tissue of the damaged retina alive and functioning.

The technique holds promise for improving sight in people with poor vision and potentially even to cure blindness altogether but the researchers cautioned that any such development was some years away and required much bigger studies to confirm their findings.

One of the main hurdles in using stem cells to treat damaged eyesight is low cell migration and integration in the retina, says Pia Cosma, at the Centre for Genomic Regulation in Barcelona.

After the cells are transplanted they need to reach the retina and integrate through its layers. Here we have found a way to enhance this process using stem cells commonly found in the bone marrow, but in principle can be used with any transplanted cells, Dr Cosma said.

There is still considerable work to be done, but our findings could make stem cell transplants a feasible and realistic option for treating visual impairment and restoring eyesight, she said.

Retinal damage, which is currently incurable, inevitably leads to visual disabilities and in most cases blindness. With a growing and ageing population, the number of people affected by retinal damage is estimated to increase dramatically over the next few decades.

Stem cell therapies have been touted as one way of treating degenerative retinal conditions. Stem cells can be transplanted into the eye, releasing therapeutic molecules with neuroprotective and anti-inflammatory properties that promote the survival, proliferation and self-repair of retinal cells. The stem cells can also generate new retinal cells, replacing lost or damaged ones.

The researchers used mesenchymal stem cells, which are found in bone marrow and can differentiate into lots of types of cells, including retinal cells that respond to light.

Mesenchymal stem cells can also be easily grown outside an organism, providing abundant starting material for transplantation compared to other cell sources such as hematopoietic stem cells.

The study is published in the journal Molecular Therapy.

Excerpt from:
Treatment to restore vision by injecting stem cells into the eye could help people with damaged eyesight - iNews

This article was originally published here

J Pharmacol Exp Ther. 2020 Nov 30:JPET-AR-2020-000277. doi: 10.1124/jpet.120.000277. Online ahead of print.

ABSTRACT

ONO-4641 is a second-generation sphingosine 1-phosphate (S1P) receptor modulator that exhibits selectivity for S1P receptors 1 and 5. Treatment with ONO-4641 leads to a reduction in magnetic resonance imaging disease measures in patients with relapsing-remitting multiple sclerosis. The objective of this study was to explore the potential impact of ONO-4641 treatment based on its immunomodulatory effects. Severe aplastic anemia is a bone marrow (BM) failure disease, typically caused by aberrant immune destruction of blood progenitors. Although the T helper type-1-mediated pathology is well described for aplastic anemia, the molecular mechanisms driving disease progression remain undefined. We evaluated the efficacy of ONO-4641 in a mouse model of aplastic anemia. ONO-4641 reduced the severity of BM failure in a dose-dependent manner, resulting in higher blood and BM cell counts. By evaluating the mode of action, we found that ONO-4641 inhibited the infiltration of donor-derived T lymphocytes to the BM. ONO-4641 also induced the accumulation of hematopoietic stem cells in the BM of mice. These observations indicate, for the first time, that S1P receptor modulators demonstrate efficacy in the mouse model of aplastic anemia and suggest that treatment with ONO-4641 might delay the progression of aplastic anemia. Significance Statement ONO-4641 is a second-generation sphingosine 1-phosphate (S1P) receptor modulator selective for S1P receptors 1 and 5. In this study, we demonstrated that ONO-4641 regulates the trafficking of T lymphocytes along with hematopoietic stem and progenitor cells leading to alleviation of pancytopenia and destruction of bone marrow in a bone marrow failure-induced mouse model mimicking human aplastic anemia.

PMID:33257316 | DOI:10.1124/jpet.120.000277

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Sphingosine 1-phosphate Receptor Modulator ONO-4641 Regulates Trafficking of T Lymphocytes and Hematopoietic Stem Cells and Alleviates Immune-Mediated...

Newswise Researchers from The University of Kansas Cancer Center are involved in the presentation of nearly 50 research studies at the 62ndAmerican Society of Hematology (ASH) Annual Meeting, to be held virtually Dec. 5-8 because of the COVID-19 pandemic. With more than 18,000 members from nearly 100 countries, the ASH is the world's largest professional society serving both clinicians and scientists around the world who are working to conquer blood diseases.

The KU Cancer Center is one of only 71 cancer centers designated by the National Cancer Institute because they meet rigorous standards for transdisciplinary, state-of-the-art research focused on developing new and better approaches to preventing, diagnosing and treating cancer. Its catchment area includes the state of Kansas as well as western Missouri.

These 49 research studies represent the hard work of our many researchers focused on blood diseases, said Roy Jensen, M.D., director of the KU Cancer Center. This includes innovations in immunotherapy, advances in leukemia and significant work in stem cell transplants. While the conference is virtual this year, the KU Cancer Center will be well represented.

While a full list of abstracts involving KU Cancer Center researchers can be found online, three of the most significant are listed below.

# # #

About The University of Kansas Cancer Center:

The University of Kansas Cancer Center is transforming cancer research and clinical care by linking an innovative approach to drug discovery, delivery and development to a nationally-accredited patient care program. Our consortium center includes cancer research and health care professionals associated with the University of Kansas Medical Center and The University of Kansas Health System; the University of Kansas, Lawrence; The Stowers Institute for Medical Research; Childrens Mercy; and in partnership with members of the Masonic Cancer Alliance.

About the University of Kansas Medical Center:

The University of Kansas Medical Centers mission is to educate exceptional health care professionals through a full range of undergraduate, graduate, professional, postdoctoral and continuing education programs in the schools of Medicine, Nursing and Health Professions. KU Medical Center also advances the health sciences through world-class research programs; provides compassionate and state-of-the-art patient care in an academic medical center environment; and works with communities in every Kansas county to improve the health of Kansans.

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Cancer center is a contributor to 49 research studies at the 62nd American Society of Hematology Annual Meeting - Newswise

NEW YORK, Dec. 03, 2020 (GLOBE NEWSWIRE) -- IN8bio, Inc., a clinical-stage biotechnology company focused on developing innovative allogeneic, autologous and genetically modified gamma-delta T cell therapies for the treatment of cancers (IN8bio or the Company), today announced an upcoming presentation that provides an update of the ongoing Phase I clinical trial of their product candidate INB-100 at the 62nd American Society of Hematology Annual Meeting & Exposition (ASH), which will take place virtually from December 5 to 8, 2020. INB-100 is designed for the treatment of patients with leukemia undergoing hematopoietic stem cell transplantation with haploidentical donors.

The poster and accompanying narrated slide presentation is titled, First-in-Human Phase I Trial of Adoptive Immunotherapy with Ex Vivo Expanded and Activated gamma delta T-Cells Following Haploidentical Bone Marrow Transplantation and Post-BMT Cyclophosphamide and reviews the study design and provides a brief update on enrollment and patient status.

The company reported that, as of abstract submission, three female subjects with acute leukemia had been enrolled in the INB-100 Phase 1 trial, of whom two had been dosed, and that no treatment-related adverse events had been recorded. The trial is continuing to enroll and treat patients. The abstract for the presentation can be found at https://ash.confex.com/ash/2020/webprogram/Paper142876.html.

The poster and slide presentation are jointly authored by the scientific and physician investigators from IN8bio and The University of Kansas Cancer Center (KU Cancer Center), and will be presented by the studys Principal Investigator, Dr. Joseph McGuirk, Schutte-Speas Professor of Hematology-Oncology, Division Director of Hematological Malignancies and Cellular Therapeutics and Medical Director, Blood and Marrow Transplant at KU Cancer Center.

This preliminary data report from KU Cancer Center with our allogeneic product candidate, INB-100, demonstrates the absence of significant GvHD in these initial patients, said William Ho, Chief Executive Officer of IN8bio. This suggests that gamma delta T-cells delivered as an off-the-shelf allogeneic cell therapy may be well tolerated and have significant potential to treat patients with serious and life-threatening cancers.

Story continues

Dr. McGuirk, commented, Potentially curative stem cell transplants using partially matched donors -- called haploidentical transplants have greatly expanded access to stem cell transplantation. The infusion of donor-derived gamma delta T-cells from the stem cell donor, offers the hope of diminishing this risk of relapse and curing more patients.

About IN8bioIN8bio is a clinical-stage biotechnology company focused on developing novel therapies for the treatment of cancers, including solid tumors, by employing allogeneic, autologous and genetically modified gamma-delta T cells. IN8bios technology incorporates drug-resistant immunotherapy (DRI), which has been shown in preclinical studies to function in combination with therapeutic levels of chemotherapy. IN8bio is currently conducting two investigator-initiated Phase 1 clinical trials for its lead gamma-delta T cell product candidates: INB-200 for the treatment of newly diagnosed glioblastoma, which is a difficult to treat brain tumor that progresses rapidly, and INB-100 for the treatment of patients with acute leukemia undergoing hematopoietic stem cell transplantation. For more information about the Company and its programs, visit http://www.IN8bio.com.

Forward Looking StatementsCertain statements herein concerning the Companys future expectations, plans and prospects, including without limitation, the Companys current expectations regarding the curative potential of its product candidates, constitute forward-looking statements. The use of words such as may, might, will, should, expect, plan, anticipate, believe, estimate, project, intend, future, potential, or continue, the negative of these and other similar expressions are intended to identify such forward looking statements. Such statements, based as they are on the current expectations of management, inherently involve numerous risks and uncertainties, known and unknown, many of which are beyond the Companys control. Consequently, actual future results may differ materially from the anticipated results expressed in such statements. Specific risks which could cause actual results to differ materially from the Companys current expectations include: scientific, regulatory and technical developments; failure to demonstrate safety, tolerability and efficacy; final and quality controlled verification of data and the related analyses; expense and uncertainty of obtaining regulatory approval, including from the U.S. Food and Drug Administration; and the Companys reliance on third parties, including licensors and clinical research organizations. Do not place undue reliance on any forward-looking statements included herein, which speak only as of the date hereof and which the Company is under no obligation to update or revise as a result of any event, circumstances or otherwise, unless required by applicable law.

Contact:IN8bio, Inc.Kate Rochlin, Ph.D.+1 646.933.5605info@IN8bio.com

Investor Contact:Julia Balanova+ 1 646.378.2936jbalanova@soleburytrout.com

Media Contact:Ryo Imai / Robert Flamm, Ph.D.Burns McClellan, Inc.212-213-0006 ext. 315 / 364Rimai@burnsmc.com / rflamm@burnsmc.com

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IN8bio announces first-in-human Phase 1 trial Update from The University of Kansas Cancer Center using INB-100, IN8bios Gamma Delta T-cell product...

Final Report will add the analysis of the impact of COVID-19 on this industry.

According to the latest industry research Bone Marrow Transplant Market share is predicted to gain better growth in upcoming years. Global Bone Marrow Transplant market report is one of the best sources of research data which provided by industry experts. Report gives facts about Covid-19 impact, geographical breakdown, top manufactures, type wise and applications wise segmentation. In Bone Marrow Transplant market report the growth rate, revenue, market shares, sales, production, consumption, manufacturers in particular areas are regionally analysed.

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Bone marrow transplant refers to the replacement of diseased or damaged bone marrow with healthy tissue or bone marrow stem cells in order to treat blood cancer or various cases of anemia. Depending on the source of bone marrow or stem cells, bone marrow transplant procedures are classified as peripheral stem cell transplant (PSCT) or conventional bone marrow transplant.

Bone Marrow Transplant Industry Segmentation:

Bone Marrow Transplant Market by Top Manufacturers:Lonza Group Ltd., Merck Millipore Corporation., Sanofi-Aventis LLC., AllCells LLC., STEMCELL Technologies., American Type Culture Collection (ATCC) Inc. By ProcedureAutologous Bone Marrow Transplant, Allogeneic Bone Marrow TransplantBy Disease IndicationLeukemia, Lymphoma, Myeloma, Myelodysplasia, Myeloproliferative Neoplasms, Aplastic Anemia, Solid tumors, Sickle cell Anemia, OthersBy End UserHospitals, Multispecialty Clinics, Ambulatory Surgical Centers

The study procedure elaborates the analysis of several features affecting the industry, with the government policy, Bone Marrow Transplant market forecast environment, technological innovation, competitive landscape, historical data, present trends in the market, forthcoming technologies and the technical progress in associated industry.

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Bone Marrow Transplant market report is outcome of comprehensive primary and secondary research accepted by analysts having years of experience in the Bone Marrow Transplant industry. All the qualitative and quantitative aspects of the industry have been covered and the collected information has been examined and accessible in the form of easily understandable charts, graphs and tables.

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Detailed TOC of 2019-2024 Global and Regional Bone Marrow Transplant Industry Production, Sales and Consumption Status and Prospects Professional Market Research Report

Chapter 1 Bone Marrow Transplant Industry Overview

1.1 Definition

1.2 Brief Introduction by Major Type

1.3 Brief Introduction by Major Application

1.4 Brief Introduction by Major Regions

1.4.1 United States

1.4.2 Europe

1.4.3 China

1.4.4 Japan

1.4.5 India

Chapter 2 Production Bone Marrow Transplant Market Analysis

2.1 Global Production Market Analysis

2.1.1 2012-2017 Global Capacity, Production, Capacity Utilization Rate, Ex-Factory Price, Revenue, Cost, Gross and Gross Margin Analysis

2.1.2 2012-2017 Major Manufacturers Performance and Market Share

2.2 Regional Production Market Analysis

2.2.1 2012-2017 Regional Market Performance and Market Share

2.2.2 United States Market

2.2.3 Europe Market

2.2.4 China Market

2.2.5 Japan Market

2.2.6 India Market

2.2.7 Rest Regions Market

Chapter 3 Bone Marrow Transplant Sales Market Analysis

3.1 Global Sales Market Analysis

3.1.1 2012-2017 Global Sales Volume, Sales Price and Sales Revenue Analysis

3.1.2 2012-2017 Major Manufacturers Performance and Market Share

3.2 Regional Sales Market Analysis

3.2.1 2012-2017 Regional Market Performance and Market Share

3.2.2 United States Market

3.2.3 Europe Market

3.2.4 China Market

3.2.5 Japan Market

3.2.6 India Market

3.2.7 Rest Regions Market

Chapter 4 Consumption Market Analysis

4.1 Global Consumption Market Analysis

4.1.1 2012-2017 Global Consumption Volume Analysis

4.2 Regional Consumption Market Analysis

4.2.1 2012-2017 Regional Market Performance and Market Share

4.2.2 United States Market

4.2.3 Europe Market

4.2.4 China Market

4.2.5 Japan Market

4.2.6 India Market

4.2.7 Rest Regions Market

Chapter 5 Production, Sales and Consumption Market Comparison Analysis

5.1 Global Production, Sales and Consumption Market Comparison Analysis

5.2 Regional Production, Sales Volume and Consumption Volume Market Comparison Analysis

5.2.1 United States

5.2.2 Europe

5.2.3 China

5.2.4 Japan

5.2.5 India

5.2.6 Rest Regions

Chapter 6 Major Manufacturers Production and Sales Market Comparison Analysis

6.1 Global Major Manufacturers Production and Sales Market Comparison Analysis

6.1.1 2012-2017 Global Major Manufacturers Production and Sales Market Comparison

6.2 Regional Major Manufacturers Production and Sales Market Comparison Analysis

6.2.1 United States

6.2.2 Europe

6.2.3 China

6.2.4 Japan

6.2.5 India

6.2.6 Rest Regions

Chapter 7 Major Type Analysis

7.1 2012-2017 Major Type Market Share

Chapter 8 Major Application Analysis

8.1 2012-2017 Major Application Market Share

Chapter 9 Bone Marrow Transplant Industry Chain Analysis

9.1 Up Stream Industries Analysis

9.1.1 Raw Material and Suppliers

9.1.2 Equipment and Suppliers

9.2 Manufacturing Analysis

9.2.1 Manufacturing Process

9.2.2 Manufacturing Cost Structure

9.2.3 Manufacturing Plants Distribution Analysis

9.3 Industry Chain Structure Analysis

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For the first time, researchers have demonstrated how the gut microbiome the community of microorganisms living in the gut can influence the immune system in humans. Their work could lead to new treatments for immune-related conditions.

The researchers at Memorial Sloan Kettering Cancer Center in New York, NY, tracked the recovery of patients gut microbiota and immune system after bone marrow transplants (BMTs) following treatment for blood cancers.

Healthcare professionals use chemotherapy and radiation therapy to destroy cancerous blood cells in conditions such as leukemia and lymphoma. After completion of the treatment, which also kills healthy immune cells, specialists inject patients with stem cells from a donors blood or bone marrow.

These donated cells slowly restore patients ability to make their own blood cells.

However, patients have to take antibiotics in the first few weeks after the transplant because they are still vulnerable to infections. These upset the balance of their gut microbiota, killing friendly bacteria and allowing dangerous strains to thrive.

Once patients immune systems are strong enough, they can stop taking the antibiotics, which allows their gut microbiota to recover.

The researchers at Sloan Kettering used this unique opportunity to study how the microbiota affects the immune system.

The scientific community had already accepted the idea that the gut microbiota was important for the health of the human immune system, but the data they used to make that assumption came from animal studies, explains systems biologist Joao Xavier, who is co-senior author of the paper with his former postdoc Jonas Schluter.

The parallel recoveries of the immune system and the microbiota, both of which are damaged and then restored, gives us a unique opportunity to analyze the associations between these two systems, says Dr. Schluter, who is now an assistant professor at NYU Langone Health in New York, NY.

Using blood and fecal samples from more than 2,000 patients treated at the cancer center between 20032019, the researchers were able to track daily changes in their gut microbiota and the number of immune cells in their blood.

Our study shows that we can learn a lot from stool biological samples that literally would be flushed down the toilet, says Dr. Xavier. The result of collecting them is that we have a unique dataset with thousands of data points that we can use to ask questions about the dynamics of this relationship.

The researchers used a machine-learning algorithm to identify patterns in the data, which included information about patients medications and the side effects they experienced.

One of the findings was that the presence of three types of gut bacteria called Faecalibacterium, Ruminococcus 2, and Akkermansia was associated with increased blood concentrations of immune cells called neutrophils.

By contrast, two types called Rothia and Clostridium sensu stricto 1, were associated with reduced numbers of these immune cells.

Computer simulations by the researchers predicted that enriching microbiota with the three friendly genera would speed up the recovery of patients immune systems.

This research could eventually suggest ways to make BMTs safer by more closely regulating the microbiota, says co-author Marcel van den Brink.

The study appears in Nature.

Concluding their paper, the authors write:

Our demonstration that the microbiota influences systemic immunity in humans opens the door toward an exploration of potential microbiota-targeted interventions to improve immunotherapy and treatments for immune-mediated and inflammatory diseases.

A previous study found that having a greater diversity of bacterial species in the gut is associated with a better chance of survival after a stem cell transplant. This research also found that a low diversity of bacteria increased the likelihood of potentially fatal graft-versus-host disease, when the donor immune cells attack the recipients tissues.

In 2018, the Sloan Kettering researchers published results from a clinical trial in which they used fecal transplants to restore patients microbiota after treatment for blood cancer.

They used the patients own fecal matter, which had been collected and frozen before the bone marrow transplant and antibiotic treatment disrupted their gut microbiota.

Original post:
Gut bacteria can help rebuild the immune system - Medical News Today

Vancouver, British Columbia--(Newsfile Corp. - December 1, 2020) - Hemostemix Inc. (TSXV: HEM) (OTCBB: HMTXF), a clinical stage biotechnology company with a patented stem cell technology platform, has successfully, following lengthy litigation, obtained its entire clinical trial database from Medrio Inc., which was hosting the database for Aspire Heath Science LLC. For more details on the litigation, please refer to today's news release.

For more information, please view the InvestmentPitch Media "video" which provides additional information about this news and the company. If this link is not enabled, please visit http://www.InvestmentPitch.com and enter "Hemostemix" in the search box.

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The company's principal business is to develop, manufacture and commercialize blood-derived stem cell therapies to treat various diseases not adequately addressed by current therapeutics. The company's process for harvesting stem cells is less invasive, as the stem cells are taken from a patient's blood, which is a simplified process as compared to taking stem cells from fatty tissue or bone marrow.

Critical limb ischemia or CLI, a severe blockage in the arteries of the lower extremities, which markedly reduces blood-flow, is deadliest form of peripheral arterial disease or PAD, with limited treatment options and no current approved drug treatments. The company's lead product, ACP-01 is the subject of a Phase II clinical trial of its safety and efficacy in patients with advanced CLI, who have exhausted all other options to save their limb from amputation.

Hemostemix owns 91 patents related to its products and manufacturing processes and has entered into a contract with a new clinical research organization which is completing the midpoint statistical analyses of the efficacy of ACP-01. A winner of the World Economic Forum Technology Pioneer Award, Hemostemix developed and is commercializing its lead product ACP-01.

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Hemostemix announced the abstract and interim results presented to the 41st annual Canadian Society for Vascular Surgery meeting, which noted healing of ulcers and resolution of ischemic rest pain occurred in 83 per cent of patients studied by lead investigators at the University of British Columbia and the University of Toronto with outcomes maintained for up to 4.5 years.

Thomas Smeenk, CEO, stated: "As every biotech investor knows, it is all about the data! Blinded, we will know in short order if our HS 12-01 midpoint results equal the interim clinical trial results that 83 per cent of patients followed for up to 4.5 years experienced. Fortunately, we have a lot of data of the efficacy of ACP. For example, we have the clinical trial results of the 41 patients treated for cardiomyopathy. And, we have the results of the 106 ischemic heart disease patients on maximal medical therapy who had no option for revascularization, who experienced significant improvement."

Hemostemix is addressing a huge potential market. According to The Sage Group LLC, in the United States alone, approximately 20 million people are affected by PAD, and it is estimated that approximately 7 to 8 million people in the United States and Europe suffer from CLI. The Sage Group estimates that in the United States, medical costs attributable to CLI amount to US$25 billion annually.

The company has also announced a non-brokered private placement. The company is looking to raise gross proceeds of up to $2.5 million from the placement of up to 250 million units priced at $0.01 per unit. The units consist of 1 share and 1 warrant, with each warrant exercisable at $0.05 for 12 months, subject to an acceleration clause. The shares are currently trading at $0.01. However, the company has obtained shareholder approval for a 20 for one share consolidation.

In addition to accredited investors, this placement is available under certain security exemptions to existing shareholders, friends and family, and those investors having received advice under the investment dealer exemption.

For more information, please visit the company's website at http://www.hemostemix.com, contact Thomas Smeenk, President, CEO and Co-Founder at 905-580-4170 or by email at TSmeenk@hemostemix.com.

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InvestmentPitch Media Video Discusses Hemostemix Successfully Obtaining all Clinical Trial Data and Announcement of $2.5 Million Unit Offering - Video...

DUBLIN--(BUSINESS WIRE)--The "Multiple Myeloma Drugs Market: Global Industry Trends, Share, Size, Growth, Opportunity and Forecast 2020-2025" report has been added to ResearchAndMarkets.com's offering.

The global multiple myeloma drugs market grew at a CAGR of around 9% during 2014-2019. Looking forward, the publisher expects the market to witness moderate growth during the next five years.

Multiple myeloma, or Kahler's disease, refers to a form of blood cancer that primarily affects the plasma cells. Some of the most common types of multiple myeloma drugs include chemotherapeutic agents, corticosteroids and immunomodulatory agents. These pharmaceutical drugs aid in promoting bone healing, prevent hypercalcemia, bone fracture, spinal cord compression and anemia, while minimizing the need for chemotherapy. The chemotherapeutic agents include various anthracycline antibiotics and alkylating agents, such as melphalan, doxorubicin, vincristine and liposomal doxorubicin. The targeted therapy drugs include proteasome inhibitor, such as bortezomib, and various other compounds, including dexamethasone, prednisone and thalidomide.

Significant developments in the healthcare sector, along with the increasing prevalence of hematological cancer, is one of the key factors driving the growth of the market. Multiple myeloma is usually caused by specific genetic abnormalities, and the treatment of this disease involves drugs that modulate the immune system and aid in enhancing the efficiency of chemotherapies, radiation therapies, stem cell transplants and platelet transfusion.

Furthermore, rising consumer awareness regarding the benefits of biologic therapy drugs, which utilize the body's immune system to identify and attack the myeloma cells, is also providing a boost to the market growth. Additionally, various technological advancements, such as the development of microRNA therapeutics and nanomedicines for the treatment of multiple myeloma, is acting as another growth-inducing factor. These medicines are used to facilitate the delivery of macromolecular agents into the bone marrow and catalyze antitumor responses. Other factors, including the rising healthcare expenditures and extensive research and development (R&D) activities in the field of medical sciences, are projected to drive the market further.

Companies Mentioned

Key Questions Answered in This Report:

Key Topics Covered:

1 Preface

2 Scope and Methodology

2.1 Objectives of the Study

2.2 Stakeholders

2.3 Data Sources

2.3.1 Primary Sources

2.3.2 Secondary Sources

2.4 Market Estimation

2.4.1 Bottom-Up Approach

2.4.2 Top-Down Approach

2.5 Forecasting Methodology

3 Executive Summary

4 Introduction

4.1 Overview

4.2 Key Industry Trends

5 Global Multiple Myeloma Drugs Market

5.1 Market Overview

5.2 Market Performance

5.3 Market Forecast

6 Market Breakup by Therapy

6.1 Targeted Therapy

6.1.1 Market Trends

6.1.2 Market Forecast

6.2 Biologic Therapy

6.2.1 Market Trends

6.2.2 Market Forecast

6.3 Chemotherapy

6.3.1 Market Trends

6.3.2 Market Forecast

6.4 Others

6.4.1 Market Trends

6.4.2 Market Forecast

7 Market Breakup by Drug Type

7.1 Immunomodulatory Drugs

7.1.1 Market Trends

7.1.2 Market Forecast

7.2 Proteasome Inhibitors

7.2.1 Market Trends

7.2.2 Market Forecast

7.3 Histone Deacetylase Inhibitors

7.3.1 Market Trends

7.3.2 Market Forecast

7.4 Monoclonal Antibody Drugs

7.4.1 Market Trends

7.4.2 Market Forecast

7.5 Steroids

7.5.1 Market Trends

7.5.2 Market Forecast

7.6 Others

7.6.1 Market Trends

7.6.2 Market Forecast

8 Market Breakup by End-User

8.1 Men

8.1.1 Market Trends

8.1.2 Market Forecast

8.2 Women

8.2.1 Market Trends

8.2.2 Market Forecast

9 Market Breakup by Distribution Channel

9.1 Hospital Pharmacies

9.1.1 Market Trends

9.1.2 Market Forecast

9.2 Retail Pharmacies

9.2.1 Market Trends

9.2.2 Market Forecast

9.3 Online Pharmacies

9.3.1 Market Trends

9.3.2 Market Forecast

9.4 Others

9.4.1 Market Trends

9.4.2 Market Forecast

10 Market Breakup by Region

10.1 North America

10.2 Asia Pacific

10.3 Europe

10.4 Latin America

10.5 Middle East and Africa

11 SWOT Analysis

12 Value Chain Analysis

13 Porters Five Forces Analysis

14 Price Indicators

15 Competitive Landscape

15.1 Market Structure

15.2 Key Players

15.3 Profiles of Key Players

For more information about this report visit https://www.researchandmarkets.com/r/8yriem

Excerpt from:
Outlook on the Multiple Myeloma Drugs Global Market to 2025 - by Therapy, Drug Type, End-user, Distribution Channel and Region -...

SOUTH SAN FRANCISCO, Calif.--(BUSINESS WIRE)--Imago BioSciences, Inc., (Imago) a clinical-stage biotechnology company developing innovative treatments for myeloid diseases, today announced the expansion of its global Phase 2b clinical study evaluating bomedemstat (IMG-7289) for the treatment of advanced myelofibrosis (MF) into Hong Kong, where the first patient has now been enrolled and dosed at the Department of Medicine, Queen Mary Hospital and the University of Hong Kong. Myelofibrosis is a rare bone marrow cancer that interferes with the production of blood cells.

In addition to Hong Kong, the Phase 2b study continues to actively enroll patients in the U.S., U.K., and E.U. The study is in the final stages of completing enrollment and continues to dose patients to evaluate safety, tolerability and efficacy.

Patients with myelofibrosis around the world are still in need of new treatment options, said Hugh Young Rienhoff, Jr. M.D., Chief Executive Officer, Imago BioSciences. We are progressing well with enrollment and are pleased to continue expanding our global Phase 2 study into new geographies like Hong Kong. We are encouraged by the signs of clinical activity and safety of bomedemstat as a treatment alternative for patients who do not benefit from the current standards of care.

Bomedemstat is an inhibitor of lysine-specific demethylase 1 (LSD1), an epigenetic regulator critical for self-renewal of malignant myeloid cells and the differentiation of myeloid progenitors. Data presented at the 25th European Hematology Association (EHA) Annual Congress in June demonstrated that the first-in-class LSD1 inhibitor was well tolerated with no dose-limiting toxicities or safety signals. Furthermore, recent data demonstrates the potential of bomedemstat as a monotherapy in intermediate-2 and high-risk patients with myelofibrosis who have become intolerant of, resistant to or are ineligible for a Janus Kinase (JAK) inhibitor.

Bomedemstat was recently granted PRIME (PRIority MEdicines) designation by the European Medicines Agency (EMA) for the treatment of MF. The EMA reviewed bomedemstat non-clinical and clinical data from the ongoing Phase 2 study. The PRIME initiative was launched by the EMA in 2016 to provide proactive and enhanced support to the developers of promising medicines with the view of accelerating their evaluation to reach patients faster.

About Bomedemstat (IMG-7289)

Bomedemstat is an orally available small molecule discovered and developed by Imago BioSciences that inhibits lysine-specific demethylase 1 (LSD1 or KDM1A), an enzyme shown to be vital in cancer stem/progenitor cells, particularly neoplastic bone marrow cells. In non-clinical studies, bomedemstat demonstrated robust in vivo anti-tumor efficacy across a range of myeloid malignancies as a single agent and in combination with other therapeutic agents. Bomedemstat is an investigational agent currently being evaluated in ongoing clinical trials (ClinicalTrials.gov Identifier: NCT03136185, NCT04262141, NCT04254978 and NCT04081220).

Bomedemstat has U.S. FDA Orphan Drug and Fast Track Designation for the treatment of myelofibrosis and essential thrombocythemia, Orphan Drug Designation for treatment of acute myeloid leukemia and PRIME designation by the European Medicines Agency for the treatment of MF.

Bomedemstat is being evaluated in two open-label Phase 2 clinical trials for the treatment of advanced myelofibrosis (MF) and essential thrombocythemia (ET), bone marrow cancers that interfere with the production of blood cells. MF patients who are resistant to a Janus Kinase (JAK) inhibitor are eligible for the study of bomedemstat. ET patients who have failed one standard of care treatment are eligible for the bomedemstat ET study.

About Imago BioSciences

Imago BioSciences is a clinical-stage biopharmaceutical company focused on discovering and developing novel therapeutics for the treatment of hematologic disorders targeting epigenetic enzymes. Imago has developed a series of compounds that inhibit LSD1, an epigenetic enzyme critical for cancer stem cell function and blood cell differentiation. Imago is advancing the clinical development of its first LSD1 inhibitor, bomedemstat, for the treatment of myeloid neoplasms. Imago BioSciences is backed by leading private, corporate, and public investors including Farallon Capital Management, LLC., funds and accounts advised by T. Rowe Price Associates, Inc., funds and accounts managed by Blackrock Advisors, LLC., Surveyor Capital (a Citadel company), Irving Investors, Kingdon Capital Management, 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. The company is based in South San Francisco, California. To learn more, visit http://www.imagobio.com, http://www.myelofibrosisclinicalstudy.com, http://www.etclinicalstudy.com and follow us on Twitter @ImagoBioRx, Facebook and LinkedIn.

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Imago BioSciences Expands Phase 2 Clinical Trial of Bomedemstat (IMG-7289) for the Treatment of Myelofibrosis into Hong Kong - Business Wire

The mRNA technology used to create the Pfizer and Moderna vaccines for COVID is being applied to ... [+] many other medical treatments in addition to vaccines.

While the vaccines for Covid-19 seem to have been created in record time, the technology making them possible has been decades in development. The two vaccine candidates produced by Pfizer/BioNTech and Moderna are unlike any other vaccine thats come before. Should they achieve commercial success, it could usher in a new era of medical science not just for vaccines, but for cancer treatments, blood disorders, and gene therapy.

The two new vaccines are the first ever to use mRNA, which stands for messenger RNA, to generate immunity. Historically, vaccines have used dead or weakened viruses to imitate an infection, spurring the body to make antibodies against that virus without danger of getting sick. Measles, polio, and some seasonal flu shots are examples of vaccines made with whole virus particles.

Other vaccines use only certain fragments of the virus, called antigens, that provoke an immune response. To make this type of vaccine, the genetic code for the desired viral antigen molecule is put into yeast or bacteria cells. These microbes can be grown rapidly and inexpensively, and they can churn out massive quantities of antigen. Then the molecule must be purified to clinical standards so that its safe to inject into healthy people. Prevnar and Gardasil are examples of this type of vaccine.

These methods work well, but they require enormous research and development efforts. A laboratory could spend years optimizing the methods for producing one virus protein, but those methods wouldnt automatically translate to mass-producing a different protein.

For every new protein, you start over. Its a brand-new procedure every step of the way, explains immunologist Drew Weissman of the Perelman School of Medicine at the University of Pennsylvania. Weissman is one of the pioneering scientists behind the mRNA vaccine.

The way I see it, the mRNA platform is much better, its much quicker, and its cheaper, says Weissman. Thats the trilogy of what you need to improve vaccines. With mRNA, the steps are the same, no matter what virus the vaccine is targeting. This makes it easily customizable. Once an mRNA manufacturing facility is up and running, it can easily be deployed to make vaccines against any number of viral antigens.

A strand of mRNA carries the instructions for making one protein. Your cells normally make their own mRNA strands and use them as blueprints to manufacture all the proteins your body needs to function.

The vaccine slips a new strand of mRNA into the cell, like an extra page in the blueprint. This mRNA contains the instructions for making the coronavirus spike protein, and the cell reads it the same way it reads its own mRNAs, using it to build the viral protein. The immune system recognizes that protein as foreign, and starts making antibodies against it. Then, if youre exposed to the actual virus, those antibodies will be available to stop the infection. Astonishingly, in animal tests, mRNA vaccines appear to induce immunity that lasts much longer than live virus vaccines.

The beauty of mRNA is that its temporary. Your cells wont keep cranking out spike protein forever. Like an Instagram story, the mRNA fades away after a certain amount of time, because you dont need to keep making coronavirus protein forever in order to maintain the protective immunity.

Another big advantage of mRNA is that its rapidly customizable. Once scientists know the genetic sequence of a viral protein, they can make the mRNA in the lab and package it into a vaccine in a matter of weeks.

Originally envisioned as a way to deliver gene therapy, mRNA had to overcome some serious challenges before arriving at todays big moment. In 2005, Weissman and his colleague, Katalin Karik, solved one of the most difficult problems facing mRNA. In its natural form, the molecule sparks an excessive immune reaction, igniting inflammation that damages the body. To avoid this, they changed the structure of the mRNA just enough to fool the immune sentries.

Similar to DNA, RNA is made up of a series of chemical letters, a kind of code that the cell translates to make a protein. Modifying the chemical structure of one of those letters allowed the information to remain intact, and eliminated the signal that triggered the bodys immune alarms.

Before the coronavirus pandemic hit, Weissmans lab was working on vaccines for influenza, herpes, and HIV. Those will all be going into phase I clinical trials within the next year, he says. But vaccines are only the beginning of what mRNA can do.

Often in the case of genetic diseases, the problem is that a broken gene fails to produce a protein that the body needs for healthy function. The idea of gene therapy is simple: send in a healthy copy of the broken gene, which the cells can use to make the protein. Most times, researchers use viruses to deliver the gene, but viruses can cause problems of their own. Delivering mRNA to the cell without a virus circumvents some of these issues.

To ferry the mRNA into cells, it is encapsulated in a fatty coating called a lipid nanoparticle (LNP). Weissmans lab has been experimenting with ways to modify the LNP so that it can home in on certain cell types.

In sickle cell disease (SCD), a broken hemoglobin gene prevents blood cells from carrying oxygen ... [+] efficiently, and causes them to take on a rigid, sickle-shaped form.

My lab has figured out how to specifically deliver the LNP to bone marrow stem cells, Weissman says. This could lead to an inexpensive and practical cure for sickle cell anemia. An mRNA molecule can be programmed to encode the beta-hemoglobin gene, which is defective in sickle cell disease. That mRNA would be sent directly to the bone marrow cells using the specially targeted LNPs, enabling the bone marrow to produce healthy red blood cells that contain functioning beta-hemoglobin.

All that would need to be done is to give people a single intravenous injection of the mRNA LNP, and youll cure their sickle cell anemia, Weissman says. By contrast, the current FDA-approved gene-editing therapy for sickle cell requires the patients bone marrow be removed, treated, and then returned to the bodyan expensive and invasive procedure. The mRNA treatment could be simple enough to deliver in lower-income countries, where sickle cell disease impacts the health of millions of people.

An up-and-coming strategy for fighting cancer is a so-called cancer vaccine, which uses immune cells called dendritic cells (DCs). DCs perform surveillance for the immune system. When they detect something that shouldnt be there, whether its a virus, a bacteria, or even a cancer cell, the DCs chew it up, break it into its component molecules, and then show those foreign molecules to the immune cells that make antibodies.

Dendritic cells chew up viruses or other foreign bodies, and present the pieces to other immune ... [+] cells. T cells and B cells both play a role in mounting a long-lasting immunity against the pathogen.

When cancer grows slowly, though, it can slip past the DC surveillance network. To give the immune system a boost, a patients DCs are taken out and artificially loaded with tumor-specific proteins, or antigens. Back inside the body, the cells stimulate the generation of antibodies against the tumor.

Using mRNA to deliver the tumor antigen information to the DCs could provide a way to make this process easier, cheaper, and safer. BioNTech is currently conducting clinical trials on cancer vaccines for triple-negative breast cancer, metastatic melanoma, and HPV-positive head and neck cancers. Called FixVac, the vaccines include multiple tumor antigens that are frequently found across different patients. Early data published in September 2020 showed promise, suggesting that the mRNA therapy generates a lasting immune response, comparable to more expensive methods.

Karik, who is now a senior vice president at BioNTech, and Weissman both speak with an air of inevitability, as if they have only been waiting patiently for the world to catch up with their discovery. The two scientists told their stories recently at the 2nd annual mRNA Day celebration in San Diego, hosted by Trilink BioTechnologies in honor of their recently opened facility there. After hearing the tumultuous history of the technology and seeing promising new data, one attendee asked, what would you say was the turning point for mRNA therapeutics?

Karik responded simply, When people read our [2005] paper. We were waiting for somebody to respond, we did a lot of experiments, but we waited and waited. It was just too early for most people.

Weissman agreed. I think we were early, he said. It finally caught on, and it will hopefully change the world.

Full coverage and live updates on the Coronavirus

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The New Coronavirus Vaccine Is Changing The Future Of Medicine - Forbes

This article was originally published here

J Vasc Interv Radiol. 2020 Nov 25:S1051-0443(20)30769-7. doi: 10.1016/j.jvir.2020.09.003. Online ahead of print.

ABSTRACT

PURPOSE: To evaluate safety and efficacy of angiogenesis induced by intraarterial autologous bone marrow-derived stem cell (BMSC) injection in patients with severe peripheral arterial disease (PAD).

MATERIALS AND METHODS: Eighty-one patients with severe PAD (77 men), including 56 with critical limb ischemia (CLI) and 25 with severe claudication, were randomized to receive sham injection (group A) or intraarterial BMSC injection at the site of occlusion (group B). Primary endpoints included improvement in ankle-brachial index (ABI) of > 0.1 and transcutaneous pressure of oxygen (TcPO2) of > 15% at mid- and lower foot at 6 mo. Secondary endpoints included relief from rest pain, > 30% reduction in ulcer size, and reduction in major amputation in patients with CLI and > 50% improvement in pain-free walking distance in patients with severe claudication.

RESULTS: Technical success was achieved in all patients, without complications. At 6 mo, group B showed more improvements in ABI of > 0.1 (35 of 41 [85.37%] vs 13 of 40 [32.50%]; P < .0001) and TcPO2 of > 15% at the midfoot (35 of 41 [85.37%] vs 17 of 40 [42.50%]; P = .0001] and lower foot (37 of 41 [90.24%] vs 19 of 40 [47.50%]; P < .0001). No patients with CLI underwent major amputation in group B, compared with 4 in group A (P = .0390). No significant difference was observed in relief from rest pain or > 30% reduction in ulcer size among patients with CLI or in > 50% improvement in pain-free walking distance among patients with severe claudication.

CONCLUSIONS: Intraarterial delivery of autologous BMSCs is safe and effective in the management of severe PAD.

PMID:33248918 | DOI:10.1016/j.jvir.2020.09.003

Original post:
Randomized, Double-Blind, Placebo-Controlled Trial to Evaluate Safety and Therapeutic Efficacy of Angiogenesis Induced by Intraarterial Autologous...

There are 206 bones in the human body ranging in size from the tiniest, found in your ear, to the largest, in your thigh.

We have just one heart roughly the size of your fist in our chest and continuously pumping about eight pints of blood.

206 bones and only one heart. But how are the two connected? Does the quality of your bones affect your heart?

BHF-funded researcher Dr Zahra Raisi-Estabragh and her team at Queen Mary University of London recently discovered that poor bone quality is in fact linked to poor heart health.

In this study, they used the UK Biobank the world's largest biomedical database to study the link between osteoporosis and cardiovascular health. Osteoporosis affects over 3 million people in the UK and results in brittle and weak bones that are more likely to break.

We know that osteoporosis and heart disease share a number of risk factors such as increasing age, a sedentary lifestyle, and smoking but are these risk factors all they share? Is there something we havent uncovered yet? This is what Dr Raisi-Estabragh and the team of researchers set out to find.

They discovered that lower bone density (the amount of bone mineral in bone tissue) is linked to stiffer arteries (a sign of poor heart health). They also found that people with poor bone quality have a higher risk of dying from coronary heart disease when the arteries supplying the heart with blood get clogged up with fatty deposits.

With this wealth of information available, the next step will be to identify more of the factors that leave people at high risk of each disease and inform public health policy aimed at improving both bone and heart health.

At the University of Manchester, BHF-funded researcher Dr John Bowes is looking at why coronary heart disease is more common in people with rheumatoid arthritis. Rheumatoid arthritis is an autoimmune disease when your immune system (which usually fights infection) attacks the cells that line your joints by mistake, making the joints swollen, stiff and painful. Over time, this can damage the joints, cartilage and nearby bone.

We know that coronary heart disease causes around half of the early deaths of people with rheumatoid arthritis. Unfortunately, however, there is no effective method to measure the risk of heart disease in people with arthritis, because the risk factors are different in these people. In fact, screening tools based on the usual risk factors (such as high blood pressure, high cholesterol, smoking) are not reliable in predicting the risk of heart disease in people with arthritis.

In comes Dr John Bowes and his team their aim is to create a tool that does just that. First, they will use genetic data and statistical analyses to understand why these two diseases frequently occur together. Based on these results, an accurate tool to identify which people with rheumatoid arthritis are most at risk of coronary heart disease could then be developed.

Your heart is a remarkable organ. It beats about 60 to 100 times every minute, pumping blood and oxygen all around your body with each heartbeat. But theres more to it than that. Your heart also produces a small number of hormones chemical messengers released into the blood and carried to target organs.

BHF-funded researcher Professor Svetlana Reilly and her team at the University of Oxford have recently discovered a new heart hormone calcitonin previously thought to only be produced by the thyroid gland. Published in Nature, the research revealed that cells in the atria (the top chambers of the heart) produce more calcitonin than cells in the thyroid.

It has long been known that calcitonins role in our bodies is to help regulate bone density. However, the Oxford researchers found that calcitonin also plays an important role in reducing atrial scarring. Scarring disrupts the electrical impulses travelling through the atria, which in turn causes the heart to beat in an irregular manner this is known as atrial fibrillation(AF). People with AF may experience dizziness, palpitations, shortness of breath, and tiredness. They are also more at risk of having a stroke.

Around 1.4 million people in the UK have been diagnosed with AF. For most people, this is a shock and emotionally challenging but new research brings new hope. Professor Reilly and her team are making great strides in this area and are hopeful that this bone and heart hormone could lead to new therapies to control or prevent this potentially devastating condition.

Another extraordinary part of the human body is bone marrow the spongy tissue deep inside some of your bones, such as your hip and thigh bones. Bone marrow is essential in repairing tissue damage because it contains stem cells, which can turn into any type of cell in the body, including blood cells.

At the University of Bristol, BHF-funded researcher Professor Paolo Madeddu is studying how diabetescauses damage to bone marrow. We know that diabetes can gradually damage blood vessels, which increases the risk of heart and circulatory diseases. This blood vessel damage impairs blood flow to bone marrow, which in turn decreases the amount of stem cells produced by bone marrow and with it the bodys ability to repair itself.

Using powerful microscopes and scanning machines, the researchers are now investigating the blood circulatory system of bone marrow in around 150 people with diabetes. They hope to find new ways to protect cells in the bone marrow from the damage caused by diabetes.

Maintaining healthy blood vessels and normal bone marrow function is important for people with diabetes, as this could help prevent heart and circulatory diseases in these people diseases which still kill one in four in the UK, and still cause heartbreak in every family.

Help us power our life saving research

We can all agree that 2020 has been a year like no other, but the fact that there are researchers out there using a Nobel Prize-winning discovery to help us beat heartbreak forever to help us live in a world free from the fear of heart and circulatory diseases is exactly what we all needed to know right now.

Read more BHF research blogs

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How are bones and the heart connected? - British Heart Foundation

MELBOURNE, Australia and BAAR, Switzerland, Nov. 29, 2020 (GLOBE NEWSWIRE) -- Telix Pharmaceuticals Limited (ASX: TLX, Telix, the Company) announces it has entered into an agreement with Scintec Diagnostics GmbH (Scintec) to acquire TheraPharm GmbH (TheraPharm), a Swiss-German biotechnology company developing innovative diagnostic and therapeutic solutions in the field of hematology.

The acquisition of TheraPharm provides Telix with access to a portfolio of patents, technologies, production systems, clinical data and know-how in relation to the use of Molecularly Targeted Radiation (MTR) in hematology and immunology. TheraPharm is developing antibody MTR technology against CD66, a cell surface target highly expressed by neutrophils (a type of white blood cell) and tumor-infiltrating lymphocytes. As such, the technology has potentially very broad applications in the diagnosis and treatment of hematologic diseases (e.g. blood cancers), lymphoproliferative disorders and immune-mediated diseases (e.g. lupus, and multiple sclerosis). Of particular interest is the demonstrated use of the technology to safely and effectively perform bone marrow conditioning (BMC) prior to bone marrow stem cell transplant.

Telix CEO, Dr. Christian Behrenbruch stated, Telix is committed to extending and improving the lives of patients with serious diseases. As such, the acquisition of TheraPharm and its MTR assets are uniquely aligned to Telixs mission and technical strengths in antibody engineering and radiochemistry. TheraPharms technology has a significant role to play in BMC and stem cell transplantation across a broad range of blood cancers and rare diseases. The current approach to BMC employs highly toxic drugs that have a poor morbidity and mortality profile, and for which many patients are ineligible. MTR offers an excellent safety profile that may greatly expand the number of patients able to undergo life prolonging stem cell transplantation while greatly reducing the hospitalisation burden and cost associated with such procedures.

TheraPharm co-founder and Managing Director, Dr. Klaus Bosslet added, Over the past 5 years, TheraPharm, in collaboration with Dr. Kim Orchard from the University of Southampton (UK), has made excellent progress developing 90Y-besilesomab for the treatment of hematologic cancers and several related conditions including multiple myeloma, leukemia and amyloidosis. This unique asset is a logical addition to Telixs portfolio, offering a potentially rapid development path to a first commercial indication for the treatment of patients with SALA, while at the same time having potentially broad applications for stem cell transplantation in patients with more common cancers of the blood, including multiple myeloma and leukemia. We look forward to joining the Telix team in order to expedite the development of products for this under-served field.

Full transaction details, including financial terms, can be found via the Telix website and ASX portal here.

About Hematopoietic Stem Cell Transplant (HSCT)

Bone marrow conditioning (BMC) followed by hematopoietic stem cell transplantation (HSCT) is presently performed to treat patients with hematologic malignancies (blood cancers), with the objective of extending patient survival or achieving cure. HSCT is also performed for a broad range of non-cancer conditions. HSCT is preferentially performed in countries of high income (Europe >30,000, Americas >20,000, worldwide >65,000 p.a., respectively) and is growing at around 5% annually.

About Systemic Amyloid Light-Chain Amyloidosis (SALA)

SALA is a rare, but serious protein deposition disease, caused by a protein known as amyloid that is produced by abnormal plasma cells residing in the bone marrow. As amyloid accumulates in the organs of the body, organ function will eventually deteriorate, ultimately causing organ failure. SALA has an estimated prevalence of 30,000 and 45,000 in United States and Europe, respectively and while a rare disease, SALA portends a very poor prognosis, with a median survival from diagnosis of ~11 months if untreated.

The current standard of care comprises of induction therapy (typically cyclophosphamide, bortezomib, dexamethasone) plus high dose melphalan BMC, followed by HSCT. This approach is typically only accessible to a small proportion of patients (<20%) who are able to tolerate induction therapy and melphalan BMC.

About Telix Pharmaceuticals Limited

Telix is a clinical-stage biopharmaceutical company focused on the development of diagnostic and therapeutic products using Molecularly Targeted Radiation (MTR). Telix is headquartered in Melbourne, Australia with international operations in Belgium, Japan and the United States. Telix is developing a portfolio of clinical-stage oncology products that address significant unmet medical needs in prostate, kidney and brain cancer. Telix is listed on the Australian Securities Exchange (ASX: TLX). For more information visit http://www.telixpharma.com.

AboutTheraPharm GmbH

TheraPharm is a biotechnology company specialised in the research, development and manufacturing of monoclonal antibodies for targeted radiation of hematopoietic malignant and non-malignant diseases, lymphoproliferative diseases, conditioning for allogeneic stem cells as well as in diagnostics of inflammatory diseases and bone marrow metastases.

See the article here:
Telix Pharmaceuticals Limited Acquires TheraPharm GmbH, Broadening Reach to Hematologic Cancers and Transplant Medicine - BioSpace

Report covers a detailed analysis of the Hematopoietic Stem Cell Transplantation (HSCT) Market in the estimated forecast period. It analyses and determines the effect of the external factors which are impacting the revenues and the growth of the market in the estimated forecast period. In addition, with the emergence of the global pandemic, the effect of COVID-19 is also analyzed in the report. Based on the type and applications, introduction of new products and research involved in the development of new products is one of the major aspects which is likely to have an impact on the Hematopoietic Stem Cell Transplantation (HSCT) Market. Similarly, the impact of the COVID-19 on the manufacturing and the effect of the demand for these products is also one of the major aspects which are likely to have an impact on the growth of the market in the estimated forecasts period.

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The comprehensive list ofKey Market Playersalong with their market overview, product protocol, key highlights, key financial issues, SWOT analysis, and business strategies:Regen Biopharma IncChina Cord Blood CorpCBR Systems IncEscape Therapeutics IncCryo-Save AGLonza Group LtdPluristem Therapeutics IncViaCord Inc

The following points are involved along with an in-depth study of each point for Hematopoietic Stem Cell Transplantation (HSCT) Market:

Manufacture Analysis Manufacture of the Hematopoietic Stem Cell Transplantation (HSCT) is analyzed with respect to different applications, types and regions.

Resource and Consumption In extension with sales, this segment studies Resource and consumption for the Hematopoietic Stem Cell Transplantation (HSCT) Market. Import export data is also provided by region if applicable.

Additionally, we provide customized report as per our clients requirement.

Historical year: 2015-2019Base year: 2019Forecast period: 2020-2027

Focus on segments and sub-section of the Market are illuminated below:

Geographical Analysis: United States, Europe, China, Japan, Southeast Asia & India, Rest of World etc.

Type Analysis:AllogeneicAutologous

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

Each and every segment has been detailed in the report with its market share, revenue, basic data, and highest growing segment globally.

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Global Hematopoietic Stem Cell Transplantation (HSCT) Competitive Analysis:

The key players are aiming innovation to increase efficiency and product life. The long-term growth opportunities available in the sector are captured by ensuring constant process improvements and economic flexibility to spend in the optimal schemes. Company profile section of top players includes its basic information like company legal name, website, headquarters, subsidiaries, its market position, history and 5 closest competitors by Market capitalization / revenue along with contact information.

Industrial Dynamics:

Hematopoietic Stem Cell Transplantation (HSCT) Market Report concentrates on market dynamics which give holistically view on driving factors mainly impacting on growth of Hematopoietic Stem Cell Transplantation (HSCT) market. It also includes restraints that have negative impact along with lucrative opportunities in near future so that readers get an idea about the profitability of market. It also gives complete information about the market scenario due to attack of corona virus. Thus, it also provides threats to the market.

This report is very useful for the new entrants as it offers them with the idea about the different approaches towards the market and the impact of COVID-19.

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Global Hematopoietic Stem Cell Transplantation (HSCT) Market 2020 Impact of COVID-19, Future Growth Analysis and Challenges | Regen Biopharma Inc,...

(TNS) - Scientists at Bay Area universities, laboratories, biotechnology companies and drug manufacturers are fashioning drug concoctions out of blood plasma, chimpanzee viruses and cells taken from bone marrow in the race to rid the world of COVID-19.

The microbial treasure hunt is not just to find a cure which may not be possible but to control the debilitating health problems caused by the coronavirus.

Major progress has been made this year. The antiviral drug remdesivir, produced in Foster City, has improved recovery times, and the steroid dexamethasone has cut the number of deaths in severely ill patients.

What follows is a list of some of the most promising medications and vaccines with ties to the Bay Area:

Antibodies

and Immunity

Mesenchymal stem cells / UCSF and UC Davis Medical Center:

UCSF Dr. Michael Matthay is leading a study of whether a kind of stem cell found in bone marrow can help critically ill patients with severe respiratory failure, known as ARDS. Matthay hopes the stem cells can help reduce the inflammation associated with some of ARDS' most dire respiratory symptoms, and help patients' lungs recover.

In all, 120 patients are being enrolled at UCSF Medical Center, Zuckerberg San Francisco General Hospital, the UC Davis Medical Center in Sacramento and hospitals in Oregon and Texas. He said the trial, which includes a small number of ARDS patients who don't have COVID-19, should have results by summer or fall 2021. So far, 28 patients are enrolled in San Francisco.

Lambda-interferon / Stanford University:

Lambda-interferon is a manufactured version of a naturally occurring protein that had been used to treat hepatitis, and researchers hoped it would help patients in the early stages of COVID-19.

Stanford researchers completed their trial of lambda-interferon and found that it did not boost the immune system response to coronavirus infections.

"That trial did not find any difference in outcomes between the treatment and placebo," said Yvonne Maldonado, chief of pediatric infectious diseases at Lucile Packard Children's Hospital at Stanford, where 120 patients were enrolled in the trial. "It didn't work."

Antiviral drugs

Remdesivir / Gilead Sciences ( Foster City):

Remdesivir, once conceived as a potential treatment for Ebola, was approved by the Food and Drug Administration in October for use on hospitalized COVID-19 patients.

Trademarked under the name Veklury, the drug interferes with the process through which the virus replicates itself. It was one of the drugs given to President Trump and has been used regularly in hospitals under what is known as an emergency use authorization.

It was approved after three clinical trials showed hospitalized coronavirus patients who received remdesivir recovered five days faster on average than those who received a placebo. Patients who required oxygen recovered seven days faster, according to the studies.

Gilead now plans to conduct clinical trials to see how remdesivir works on pediatric patients, from newborns to teenagers, with moderate to severe COVID-19 symptoms. Remdesivir is also being studied with steroids and other drugs to see if it works better as part of a medicinal cocktail. An inhalable form of the drug is also being developed.

Favipiravir / Fujifilm Toyama Chemical ( Stanford University):

This antiviral drug, developed in 2014 by a subsidiary of the Japanese film company to treat influenza, is undergoing numerous clinical studies worldwide, including a trial involving 180 patients at Stanford University.

Stanford epidemiologists are testing favipiravir to see if it prevents the coronavirus from replicating in human cells, halts the shedding of the virus and reduces the severity of infection. Unlike remdesivir, it can be administered orally, so it can be used to treat patients early in the disease, before hospitalization is necessary.

The Stanford study has so far enrolled about 90 patients, who are given the drug within 72 hours of when they were first diagnosed with COVID-19. Half of them get a placebo. People can enroll by emailing treatcovid@stanford.edu.

Monoclonal antibodies

REGN-COV2 / Regeneron Pharmaceuticals / Stanford School of Medicine:

The REGN-COV2 cocktail is the same one Trump received, and Stanford is one of dozens of locations nationwide where clinical trials are being held. Two separate trials are under way at Stanford one for hospitalized patients, the other for outpatients. A third trial is about to begin for people who aren't sick but are in contact with carriers of the virus.

Regeneron halted testing on severely ill patients requiring high-flow oxygen or mechanical ventilation after the independent Data and Safety Monitoring Board determined that the drug was unlikely to help them.

The drug is a combination of two monoclonal antibodies lab-made clones of the antibodies produced naturally in people who have recovered from COVID-19. The antibodies bind to the virus' spike protein and block the virus' ability to enter cells.

Dr. Aruna Subramanian, professor of infectious diseases at Stanford and lead investigator for the inpatient trial, said the 21 hospitalized patients in the study receive a high dose like Trump, a lower dose or a placebo. Subramanian plans to expand the inpatient trial to 45 patients. The outpatient study has enrolled a little more than 40 of the 60 patients researchers intend to sign up.

"There's enough promising evidence that it helps people early in the infection," Subramanian said. "What we don't know is whether it helps people who are pretty sick but not critically ill."

Bamlanivimab / Eli Lilly / Stanford and UCSF:

Stanford and UCSF are testing the Eli Lilly monoclonal antibodies on outpatients after the pharmaceutical company halted trials on hospitalized COVID-19 patients because of adverse results.

Dr. Andra Blomkalns, chair of emergency medicine at Stanford and the lead in the Eli Lilly outpatient trial, said she is now enrolling older people with comorbidities like heart disease, chronic lung disease, a history of strokes and severe obesity shortly after they test positive.

The hypothesis is that the bamlanivimab monotherapy, which is very similar to the Regeneron monoclonals, might work best early in the infection. Although about 400 patients have been enrolled in the Lilly phase 3 trials nationwide, to date fewer than 10 have been enrolled at Stanford and UCSF.

Matthay, who headed up the Lilly monoclonal study with LY-CoV555 at UCSF, said the cancellation of this inpatient trial was disappointing, but "just because this one did not work, doesn't mean another one won't work for hospitalized patients."

Blomkalns said the testing criteria has been changing. She expects the outpatient trial to open soon to adolescents ages 12 and up to determine whether the drug can be used as a preventive.

Designer monoclonal antibodies / Vir Biotechnology, San Francisco:

Scientists at Vir are studying several types of monoclonal antibodies, including a type engineered to activate T cells, which can search out and destroy cells infected with the coronavirus. A study published in the journal Nature in October found that monoclonals, modified to bind with certain receptors, stimulated T cells and improved the human immune response.

"By observing and learning from our body's powerful natural defenses, we have discovered how to maximize the capacity of antibodies through the amplification of key characteristics that may enable more effective treatments for viral diseases," said Herbert Virgin, the chief scientific officer at Vir and co-author of the study.

A similarly modified monoclonal antibody, leronlimab, is being studied in coronavirus clinical trials by its Washington state drugmaker, CytoDyn, which has developed drugs to treat HIV. The company's chief medical officer is in San Francisco, and the company that does laboratory tests of leronlimab is in San Carlos.

Anti-inflammatory drugs

Colchicine / UCSF ( San Francisco and New York):

The anti-inflammatory drug commonly used to treat gout flare-ups is being studied by scientists at UCSF and New York University. The drug short-circuits inflammation by decreasing the body's production of certain proteins, and researchers hope that it will reduce lung complications and prevent deaths from COVID-19.

Preliminary results from a clinical trial found that "Colchicine can be effective in reducing systemic symptoms of COVID-19 by inhibiting inflammatory biomarkers."

Selinexor / Kaiser Permanente:

Kaiser hospitals in San Francisco, Oakland and Sacramento are studying selinexor, an anticancer drug that blocks a key protein in the cellular machinery for DNA processing. Preliminary findings during the trials indicated that low doses of selinexor helped hospitalized patients with severe COVID-19. The drug has both antiviral and anti-inflammatory properties, and it's administered orally, according to Kaiser's Dr. Jacek Skarbinski.

Vaccines

VXA-COV2-1 / Vaxart, South San Francisco:

The biotechnology company Vaxart is testing VXA-COV2-1, the only potential vaccine in pill form. It uses the genetic code of the coronavirus to trigger a defensive response in mucous membranes. The hope is that the newly fortified membranes will prevent the virus from entering the body.

"It's the only vaccine (candidate) that activates the first line of defense, which is the mucosa," said Andrei Floroiu, Vaxart's chief executive. He said intravenous vaccines kill the virus after it is inside the body, but this one stops it beforehand.

See original here:
Coronavirus Updates: The Latest Treatments and Vaccines - GovTech

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The Report Segments for Hematopoietic Stem Cell Transplantation (HSCT) Market Analysis & Forecast 20192025 are as:Global Hematopoietic Stem Cell Transplantation (HSCT) Market, by ProductsAllogeneicAutologous

Global Hematopoietic Stem Cell Transplantation (HSCT) Market, by ApplicationsPeripheral Blood Stem Cells Transplant (PBSCT)Bone Marrow Transplant (BMT)Cord Blood Transplant (CBT)

The Major Players Reported in the Market Include:Regen Biopharma IncChina Cord Blood CorpCBR Systems IncEscape Therapeutics IncCryo-Save AGLonza Group LtdPluristem Therapeutics IncViaCord Inc

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Hematopoietic Stem Cell Transplantation (HSCT) Market To Witness Huge Growth By 2027 | Regen Biopharma Inc, China Cord Blood Corp, CBR Systems Inc,...

Bone marrow aspiration and trephine biopsy are usually performed on the back of the hipbone, or posterior iliac crest. An aspirate can also be obtained from the sternum (breastbone). For the sternal aspirate, the patient lies on their back, with a pillow under the shoulder to raise the chest. A trephine biopsy should never be performed on the sternum, due to the risk of injury to blood vessels, lungs or the heart.

The need to selectively isolate and concentrate selective cells, such as mononuclear cells, allogeneic cancer cells, T cells and others, is driving the market. Over 30,000 bone marrow transplants occur every year. The explosive growth of stem cells therapies represents the largest growth opportunity for bone marrow processing systems.

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Europe and North America spearheaded the market as of 2018, by contributing over 74.0% to the overall revenue. Majority of stem cell transplants are conducted in Europe, and it is one of the major factors contributing to the lucrative share in the cell harvesting system market.

In 2018, North America dominated the research landscape as more than 54.0% of stem cell clinical trials were conducted in this region. The region also accounts for the second largest number of stem cell transplantation, which is further driving the demand for harvesting in the region.

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Asia Pacific is anticipated to witness lucrative growth over the forecast period, owing to rising incidence of chronic diseases and increasing demand for stem cell transplantation along with stem cell-based therapy. Japan and China are the biggest markets for harvesting systems in Asia Pacific. Emerging countries such as Mexico, South Korea, and South Africa are also expected to report lucrative growth over the forecast period. Growing investment by government bodies on stem cell-based research and increase in aging population can be attributed to the increasing demand for these therapies in these countries.

Major players operating in the global bone marrow processing systems market are ThermoGenesis (Cesca Therapeutics inc.), RegenMed Systems Inc., MK Alliance Inc., Fresenius Kabi AG, Harvest Technologies (Terumo BCT), Arthrex, Inc. and others.

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Bone Marrow Processing Systems Market Top Participant to Focus on Regional Expansion - Murphy's Hockey Law

Brilliant news as Keisha announces daughter Aurora is back in remission Photo Keisha Pile-Gray

Amazing news has been announced today (November 30) nine-year-old Aurora Pile-Gray from Westbrook is in remission.

Aurora was diagnosed with stage 4 Burkitts Lymphoma after becoming poorly towards the end of April.

The rare cancer affects blood and bone marrow. Aurora has been undergoing chemotherapy and her family were looking at CAR-T cell therapy and an allogeneic transplant.

Earlier this year, after 4 gruelling cycles of intensive chemo, Aurora had been given the news that she was in remission, however this was short lived.

In October the family were told the cancer had in fact spread to Auroras bones and that the youngster was in need of a bone marrow transplant. The situation was also complicated by Auroras mixed ethnicity, making it that much more difficult to find a match.

Aurora is currently being treated by Royal Marsden Hospital and Great Ormond Street Hospital.

But in brilliant news Auroras mum Keisha has today revealed her daughter is now in remission.

Posting to facebook Keisha said: Aurora is officially back in remission!!

Her bone marrow assessments show no cancer cells present, and no cancer cell regeneration on new cells in both the solid and liquid part of her biopsy.

We were aware about the liquid aspirate a week ago, but weve been anxiously waiting for news on the solid part up until today!

The transplant team have also sourced an 11/12 donor match which means that Aurora will have one more round of chemotherapy and will move forward with transplant in January!

We dont know much about her donor other than her stem cells will be coming from a 36 year old female, with 2 children. We arent sure if we are allowed to get in contact before two years but just know, if you read this and its you, or of you know who it could be, we owe our entire life to you and would give you the world.

Days like today make our heart burst with pride and love and just how strong she has been throughout this whole ordeal. We are forever in awe of how she has tackled this journey and we are absolutely overjoyed that things are starting to look up!

Theres still a long way to go, but shes already come this far, we are all so over the moon, and I can barely get my words out, so for now, we are celebrating that out little lion is fighting on.

Matching bone marrow donors is a much more complex process than matching blood type. It relies on matching individual tissue type, and genetic markers that are found on most cells in the body. These markers are used by the immune system as a way to distinguish what cells are supposed to be in the body, and which arent. The markers must be as closely matched as possible between host and guest, to prevent the body rejecting the new bone marrow. Everyones tissue type is inherited, so often bone marrow donations come directly from a donor with the same ethnic background.

Theres a lack of individuals from ethnic minorities on the register, and as a whole only 2% of the entire UK population is currently signed up to become a bone marrow donor despite having a 1 in 800 chance you would be a match for someone.

Keisha added: Auroras in remission but we still have to get her through transplant and theres a 90% chance it could return within a year. Were made up, but still very apprehensive.

This month Keisha and Aurora were announced as joint winners, alongside Westgates Wilfred Jenkins, after our call out for Thanet heroes of 2020.

And Keishas latest update here

Find out how to join the bone marrow register:

http://www.anthonynolan.org

http://www.dkms.org

A fundraising page has been set up to help the family in case treatment abroad is needed.

Related

Read more here:
Family 'over the moon' as nine-year-old Aurora confirmed as being back in remission - The Isle of Thanet News

The researchers noted a weakening of possible repair mechanisms of blood vessels in patients who showed clinically significant vasoplegia

Vasoplegia, where vaso refers to blood vessels and plegia stands for paralysis, is a condition where the patient exhibits a low blood pressure, even in the presence of normal or increased output of blood from the heart. When this occurs as a complication of cardiopulmonary bypass surgery, there is a chance that it can lead to multiple organ failure and even death. Now, a diverse group of researchers including clinicians, computational biologists and biotechnologists have come together to study how this may be predicted early on based on clinical observations, so that effective treatment may be given.

Also Read | Mumbais first robot-assisted cardiac surgery

In a pilot study involving 19 patients who underwent elective cardiac surgery, the researchers measured the circulating counts of endothelial progenitor cells and hematopoietic stem cells at different points in time starting from when the patient was being anaesthetised to until 24 hours after the surgery. They find that in a statistically significant number of people in the group that showed clinically significant vasoplegia, there was a blunting of the endothelial progenitor cell response. Also, in the group that did not show clinically significant vasoplegia, they observed that there was no such blunting.

We can say there appears to be a pattern, which is well worth exploring in a larger cohort of patients and further delineating this particular response as a biomarker in predicting a potentially devastating complication following cardiac surgeries, says Dr. Paul Ramesh Thangaraj, from the department of cardiothoracic surgery, Apollo Hospitals, Chennai, who is one of the PIs of the study. This research is published in the journal PLOS ONE.

Hematopoietic and endothelial progenitor cells play an important role in repair of damaged tissues and inner lining of the blood vessels called the endothelium, respectively. Usually, these cells reside in the bone marrow; however, in response to injury to a tissue or a blood vessel, they come out into the circulation from the bone marrow and home into the site of injury for tissue repair, says Madhulika Dixit from the Department of Biotechnology, Indian Institute of Technology Madras, in an email to The Hindu.

Prof Dixit describes using flow cytometry to measure the counts during the surgery and afterwards. The cells were identified by means of expression of specialised cell surface receptors. For this, at regular intervals the blood withdrawn from the patient was subjected to flow cytometry. We checked for time-dependent changes in circulating counts of progenitor cells during the course of cardiopulmonary bypass in patients.

Also Read | Minimally invasive cardiac surgery the best bet

One of the key challenges was to get significant patterns in this small dataset, according to Rahul Siddharthan, from The Institute of Mathematical Sciences, Chennai, who was one of the people involved in formal analysis. In this case, we have two data sets, with two-valued outcomes [non-vasoplegic or vasoplegic], and the goal is to see how other measured parameters can predict them, he says. There are very sophisticated machine-learning algorithms available these days for such tasks. In this case the most basic algorithm, logistic regression, is good enough, says Prof. Siddharthan.

As he explains, in both cases, the idea is to look at a single value (change in circulating progenitor cells at two timepoints) and in seeing its predictive power for the output. The trend is clear, that for non-vasoplegic patients, the level of circulating progenitor cells increases, while for vasoplegic patients, it stays flat or decreases. There are exceptions but the finding is statistically significant even on this small study, says Prof. Siddharthan.

With a larger study, Dr.. Paul Ramesh envisages even developing a risk score for predicting vasoplegia as a complication following surgery.

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Pilot study finds potential signal indicative of loss of tone in blood vessels after cardiac surgery - The Hindu

The elated family of a nine-year-old girl battling a rare cancer has revealed she is back in remission and a bone marrow donor has been found.

The odds had been stacked against Aurora Pile-Gray, from Westgate, who was diagnosed with stage 4 Burkitt Lymphoma in May, which affects the blood and bone marrow.

Not only did she need a bone marrow transplant, but in September her parents were told her cancer was more aggressive than ever, just two weeks after being given the all-clear.

But now, mum Keisha, who also has a baby son Oscar and two-year-old daughter Ada-Ireland, has announced the news they have all been desperately waiting for - that brave Aurora, who has undergone gruelling rounds of chemotherapy, is in remission.

A donor has also been found meaning she will have a transplant in the new year.

Writing on Facebook, Keisha said: "Her bone marrow assessments show no cancer cells present, and no cancer cell regeneration on new cells in both the solid and liquid part of her biopsy.

"We were aware about the liquid aspirate a week ago, but we've been anxiously waiting for news on the solid part up until today.

"The transplant team have also sourced an 11/12 donor match which means that Aurora will have one more round of chemotherapy and will move forward with transplant in January.

"We don't know much about her donor other than her stem cells will be coming from a 36-year-old female, with two children.

"We aren't sure if we are allowed to get in contact before two years but just know, if you read this and it's you, or if you know who it could be, we owe our entire life to you and would give you the world.

"Days like today make our heart burst with pride and love at just how strong she has been throughout this whole ordeal.

"We are forever in awe of how she has tackled this journey and we are absolutely overjoyed that things are starting to look up.

"There's still a long way to go, but she's already come this far, we are all so over the moon, and I can barely get my words out, so for now, we are celebrating that our little lion is fighting on."

The community has been rallying round to help the family raise money towards life-saving cell treatment abroad for the St Crispin's pupil.

For more on her journey see growingpainspaperplanes.wordpress.com.

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Family's joy as schoolgirl, 9, back in remission - Kent Online

A one-year-old boy has been diagnosed with a condition so rare only one in a million people suffer from it.

Max Gardner was diagnosed with aplastic anaemia - a condition that means the bone marrow and stem cells do not produce enough blood cells and is fatal if untreated.

He was diagnosed after his parents, Connor Gardner and Rachel Nicholson, who live in Hebburn, became alarmed by significant bruising and rashes all over his body.

The couple took him to South Tyneside District Hospital, where he was incorrectly diagnosed with immune thrombocytopenic purpura, a condition which a child will grow out of.

However, as Maxs condition worsened, he ended up at the Royal Victoria Infirmary in Newcastle, where doctors conducted tests which showed he had the much rarer aplastic anaemia.

Connor said: He looked like he was a child abuse victim; we were really worried about what people would think, as he was covered in bruises.

We took him to the RVI for further tests, and they realised that maybe the condition was worse. Initially, we thought he would be diagnosed with leukaemia, but the consultant told us that it was aplastic anaemia after a bone marrow biopsy, which was administered under anaesthetic.

They told us about the condition, and that the outcome could lead to death if Max was to catch any type of sickness bug, as his immune system was non-existent.

We got our emotions out after we got the diagnosis we had a cry but we knew that we needed to be there for Max and help him get better.

The only way to cure aplastic anaemia fully is with a bone marrow transplant, and both Connor, 29, and Rachel, 27, were tested to see if they were matches.

Fortunately, Rachel was a near-perfect match, a very rare scenario.

Connor said: Usually they would use siblings for the transplant but Max does not have any. There is about a 25% chance that me or Rachel would be a match, and then there is about a 1% chance that it would be a 9/10 match.

The condition that Max has affects one in a million people, so it is very unfortunate for Max to have had this condition, but it is lucky that his mother has been a near-perfect match.

Chemotherapy is the next stage before you have the transplant, but that can lead to wiping out fertility, so we agreed to a new trial that would give Max the best chance of being able to have children of his own when he grows up.

They take a biopsy of one of his testicles and they store it for future; it is the best chance he has of having a child when he is older if he is infertile.

The family now have to shield for two weeks, before Max and his mother head back to hospital and onto the transplant ward, where he will spend the next two months.

Fortunately, Rachel can stay with Max during this time, but Connor can only see his son at specific visiting hours and has to isolate, so that the risk of spreading any illness is at a minimum.

He said: Max starts his chemotherapy on December 10, which takes place over five days, and during that time Rachel will be getting treatment so that the hospital can help harvest her bone marrow.

Then, when she goes to give the transplant, she will be there for four hours while the machine separates the bone marrow before it is given to Max.

Then he gets a bone marrow transplant, which is very similar to a normal blood transfusion."

Connor and Rachel have set up a fundraising page to help pay for the added costs of not working and to help them support them through this tough time.

He said: We have been overwhelmed with the support that people have given us and the GoFundMe page has been a great way for people to give us time.

I have been taken back by the generosity of total strangers.

Connor stressed the importance of raising awareness for bone marrow transplants, and had his fiance not been a very rare match, they would likely have had to wait for a match on the donor register.

I think it is important to raise awareness of the Anthony Nolan page. We have been lucky enough to get a donor for Max through his mam, but there are lots of people out there who have not been so lucky and are waiting for a donor.

We have met a little girl who is eight years old and she hasnt got a match yet, so we are just hoping that people will join the donor list as it may save someones life.

You can donate to the fundraising campaign by visiting here.

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Family's heartache after Hebburn boy diagnosed with one in a million condition - Chronicle Live