Healing others with music – liherald
By daniellenierenberg
By Stephanie Banat
17-year-old Samantha Horowitz is teaching the world about the healing powers of music.
A lifelong Merrick resident, Horowitz is a senior at Calhoun High School who for the past three years has been the sole vocalist in the production of a musical documentary, Second Chance, based on her mother, Tara Notricas, long battle with mast cell disease.
Some of the songs were written from my perspective, and some were written from my moms perspective, Horowitz explained. Music has given us the freedom to express things that we couldnt put into words and I truly believe its a huge part of the reason that my mom is here with us today.
In honor of her creative, healing effort, the Herald is proud to name Horowitz its 2021 Person of the Year.
Since Notricas early 20s, she had suffered from a number of physical maladies of unknown causes, including episodes of anaphylactic shock, hair loss and other issues.
It wasnt until April 2011, after consultations with scores of specialists, that Notrica was finally diagnosed with mast cell activation syndrome, a rare disorder caused by abnormal or overly active mast cells that affects multiple organ systems, including the gastrointestinal, neurological, endocrine, cardiac and respiratory systems.
It took a huge toll on me and my family, Horowitz said. I was 5 at the time, and I didnt understand what was going on. I just knew that my mom was sick, and that she couldnt be the mom she wanted to be for my brother, Jared, and I.
In 2018, Notrica endured a stem cell transplant, which was unsuccessful. Next, that June, her doctors offered her the option of receiving a bone marrow transplant, which, they said, she had a 50-50 chance of surviving. Nonetheless, Notrica decided to go forward with the procedure.
At this time, the whole music process really started picking up, her daughter said, because there were now a lot more emotions we were experiencing to write about because there were some days that my mom woke up and really didnt think she was going to make it.
Just two weeks before the bone marrow transplant, the family began filming a documentary, directed by Rochester-based filmmakers Matthew White and Brian Gerlach. The film documents Notricas health journey, and focuses on the weeks leading up to the transplant. Its title, Second Chance, comes from one of its songs, which is about Notrica getting a second chance at life, and getting to experience everything she had missed out on because of her illness.
Since 2017, Horowitz has written and recorded 11 original songs for the film. Her music career, however, started long before the documentary.
Ive had a passion for singing since I was around 3 or 4 years old, she said. In elementary school I did musical theater, and then in middle school I began writing my own original songs.
In 2017, at age 13, she wrote her first song for the documentary, alongside her mother and her vocal coach, former American Idol contestant and Merrick native Robbie Rosen. The ballad, called Brave the Storm, was written to show Notrica that she wasnt facing her illness alone, her daughter said.
Another one of her favorite songs from the documentary, Horowitz said, is called Carry On, which she wrote from her mothers perspective. This song is basically my mom saying that if it came down to it and she didnt make it, she wants my family to carry on without her, Samantha said, because shed always be a part of us and would always be watching over us.
Now, nearly three years after the transplant, and after facing a multitude of complications from it, Notrica is still under medical care at home.
The biggest thing, Horowitz said, is throughout this whole process of my mom being sick, whats always brought her a sense of comfort is music. Not just her favorite artists on the radio, but really the fact that I could sing to her and bring her joy and show her that there are things in life that are certainly worth fighting for not just her family, but also things like music.
Aside from her music, Horowitz has earned academic accolades throughout her high school career, and is a member of Calhouns national, math, science, English, social studies and world language honor societies. She is also a peer tutor for other students.
Rosen, who has gotten to know Horowitz well over the past four years, spoke about her dedication to the film and her ability to balance her various responsibilities despite the hardships shes faced. Shes been through so much since her childhood, Rosen said, so I think that her ability to keep it together, get the grades that she does, focus on music the way she does, and persevere through everything is a testament to who she is, her strength and her talent.
Calhoun Principal Nicole Hollings also noted Horowitzs many strengths, and the reasons that she is an ideal role model for others. Aside from being an outstanding student who has taken rigorous courses throughout high school, Hollings said, Samantha has been involved in many community service opportunities, and has always given her time and help to others who need it. She is truly a role model to others, showing how to be strong, caring, and how to live life in the moment, making every moment count, no matter how difficult it might be to do that.
Horowitz said that her mothers health journey has inspired her to major in biology when she starts college next year, and that she plans to go into the medical field. Im really interested in studying the correlation between music and someone healing, she said, Although this journey has caused me a lot of suffering, its made me extremely passionate about what I want to do with my future, and honestly, it has made me into who I am today.
Aside from sharing the familys ordeal, the documentary raises awareness of rare diseases, educates about bone marrow transplants, encourages people to become bone marrow donors and promotes State Senate Bill S1377, which would require school districts to establish medical hardship waiver policies.
But Horowitz said that her overall goal in creating the documentary is to help others who may be going through similar struggles. The main purpose isnt just to share my moms story or to get our music out there, she said, but really, its for people who are going through similar situations to see that they arent alone because its not easy for everyone to talk about their condition the way my mom does, and not everyone has a family member that can make songs about their journey to comfort them but I believe this film has the power to change peoples perspectives on life and to show them that music truly is a coping mechanism.
She added that she hoped the film would teach people not to take life for granted, and to make the best of every negative situation.
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Healing others with music - liherald
Who can donate stem cells or bone marrow? | Stem cell and …
By daniellenierenberg
Find out who can be a stem cell or bone marrow donor, and how to register.
A stem cell or bone marrow transplant is an important treatment for some people with types of blood cancer such as leukaemia, lymphoma and myeloma.
A transplant allows you to have high doses of chemotherapy and other treatments. The stem cellsare collected from the bloodstream or the bone marrow.Peoplehave a transplant either:
To be a donor you need to have stem cells that match the person you are donating to. To find this out, you have a blood test to look at HLA typing or tissue typing.
Staff in the laboratory look at the surface of your blood cells. They compare them to the surface of the blood cells of the person needing a transplant.
Everyone has their own set of proteins on the surface of their blood cells. The laboratory staff look for proteins called HLA markers and histocompatibility antigens. They check for 10 HLA markers. The result of this test shows how good the HLA match is between you and the person who needs the cells.
Abrother or sisteris most likely to be a match. There is a 1 in 4 chance of your cells matching.This is called a matched related donor (MRD) transplant.Anyone else in the family is unlikely to match. This can be very frustrating for relatives who are keen to help.
Sometimes if your cells are a half (50%) match, you might still be able to donate stem cells or bone marrow to a relative. This is called a haploidentical transplant.
You can't donate stem cells or bone marrow to your relative if you're not a match.
It's sometimes possible to get a match from someoneoutside of the family. This is calleda matched unrelated donor. To find a matched unrelated donor, it'susually necessary to search large numbers of people whose tissue type has been tested. So doctorssearch national and international registers to try to find a match for your relative.
Even if you can't donate to your relative, you might be ableto become a donor for someone else. You can do this by contacting one of the UK registers.
There are different donor registersin the UK.These work with each otherand with international registersto match donors with people who need stem cells. This helps doctors find donors for their patients as quickly as possiblefrom anywhere in the world.
Each registry has specific health criteriaand listmedical conditions that mightpreventyou from donating. Check their websitefor this information. Once registered, the organisation will contactyou if you are a match for someone who needs stem cells or bone marrow.
British Bone Marrow Registry (BBMR)
To register with the BBMR, you mustbe a blood donor. BBMR would like toregister those groups they are particularly short of ontheir register.This includes men between the ages of 17 and 40. And womenaged between 17 and 40 who are from Black, Asian, and minority ethnicities and mixed ethnicity backgrounds.
You have a blood test for tissue typing. Your details are kept on file until you are 60.
Anthony Nolan
You must be aged between 16 and 30 to register with Anthony Nolan. You have a cheek swab to test fortissue typing. Your details are kept on the register until you are 60.
Welsh Bone Marrow Donor Registry
You must be aged between 17 and 30 and your details are kept on the register until you are 60. You have a blood test for tissue typing.
DKMS
To register you must be aged between 17 and 55. You havea cheek swab for tissue typing. Your details stay on the register until your61st birthday.
This page is due for review. We will update this as soon as possible.
Link:
Who can donate stem cells or bone marrow? | Stem cell and ...
Bone marrow: Function, diseases, transplants, and donation
By daniellenierenberg
Bone marrow is the spongy tissue inside some of the bones in the body, including the hip and thigh bones. Bone marrow contains immature cells called stem cells.
Many people with blood cancers, such as leukemia and lymphoma, sickle cell anemia, and other life threatening conditions rely on bone marrow or cord blood transplants to survive.
People need healthy bone marrow and blood cells to live. When a condition or disease affects bone marrow so that it can no longer function effectively, a marrow or cord blood transplant could be the best treatment option. For some people, it may be the only option.
This article looks at everything there is to know about bone marrow.
Bone marrow is soft, gelatinous tissue that fills the medullary cavities, or the centers of bones. The two types of bone marrow are red bone marrow, known as myeloid tissue, and yellow bone marrow, known as fatty tissue.
Both types of bone marrow are enriched with blood vessels and capillaries.
Bone marrow makes more than 220 billion new blood cells every day. Most blood cells in the body develop from cells in the bone marrow.
Bone marrow contains two types of stem cells: mesenchymal and hematopoietic.
Red bone marrow consists of a delicate, highly vascular fibrous tissue containing hematopoietic stem cells. These are blood-forming stem cells.
Yellow bone marrow contains mesenchymal stem cells, or marrow stromal cells. These produce fat, cartilage, and bone.
Stem cells are immature cells that can turn into a number of different types of cells.
Hematopoietic stem cells in the bone marrow give rise to two main types of cells: myeloid and lymphoid lineages. These include monocytes, macrophages, neutrophils, basophils, eosinophils, erythrocytes, dendritic cells, and megakaryocytes, or platelets, as well as T cells, B cells, and natural killer (NK) cells.
The different types of hematopoietic stem cells vary in their regenerative capacity and potency. They can be multipotent, oligopotent, or unipotent, depending on how many types of cells they can create.
Pluripotent hematopoietic stem cells have renewal and differentiation properties. They can reproduce another cell identical to themselves, and they can generate one or more subsets of more mature cells.
The process of developing different blood cells from these pluripotent stem cells is known as hematopoiesis. It is these stem cells that are needed in bone marrow transplants.
Stem cells constantly divide and produce new cells. Some new cells remain as stem cells, while others go through a series of maturing stages, as precursor or blast cells, before becoming formed, or mature, blood cells. Stem cells rapidly multiply to make millions of blood cells each day.
Blood cells have a limited life span. This is around 120 days for red blood cells. The body is constantly replacing them. The production of healthy stem cells is vital.
The blood vessels act as a barrier to prevent immature blood cells from leaving bone marrow.
Only mature blood cells contain the membrane proteins required to attach to and pass through the blood vessel endothelium. Hematopoietic stem cells can cross the bone marrow barrier, however. Healthcare professionals may harvest these from peripheral, or circulating, blood.
The blood-forming stem cells in red bone marrow can multiply and mature into three significant types of blood cells, each with its own job:
Once mature, these blood cells move from bone marrow into the bloodstream, where they perform important functions that keep the body alive and healthy.
Mesenchymal stem cells are present in the bone marrow cavity. They can differentiate into a number of stromal lineages, such as:
Red bone marrow produces all red blood cells and platelets and around 6070% of lymphocytes in human adults. Other lymphocytes begin life in red bone marrow and become fully formed in the lymphatic tissues, including the thymus, spleen, and lymph nodes.
Together with the liver and spleen, red bone marrow also plays a role in getting rid of old red blood cells.
Yellow bone marrow mainly acts as a store for fats. It helps provide sustenance and maintain the correct environment for the bone to function. However, under particular conditions such as with severe blood loss or during a fever yellow bone marrow may revert to red bone marrow.
Yellow bone marrow tends to be located in the central cavities of long bones and is generally surrounded by a layer of red bone marrow with long trabeculae (beam-like structures) within a sponge-like reticular framework.
Before birth but toward the end of fetal development, bone marrow first develops in the clavicle. It becomes active about 3 weeks later. Bone marrow takes over from the liver as the major hematopoietic organ at 3236 weeks gestation.
Bone marrow remains red until around the age of 7 years, as the need for new continuous blood formation is high. As the body ages, it gradually replaces the red bone marrow with yellow fat tissue. Adults have an average of about 2.6 kilograms (kg) (5.7 pounds) of bone marrow, about half of which is red.
In adults, the highest concentration of red bone marrow is in the bones of the vertebrae, hips (ilium), breastbone (sternum), ribs, and skull, as well as at the metaphyseal and epiphyseal ends of the long bones of the arm (humerus) and leg (femur and tibia).
All other cancellous, or spongy, bones and central cavities of the long bones are filled with yellow bone marrow.
Most red blood cells, platelets, and most white blood cells form in the red bone marrow. Yellow bone marrow produces fat, cartilage, and bone.
White blood cells survive from a few hours to a few days, platelets for about 10 days, and red blood cells for about 120 days. Bone marrow needs to replace these cells constantly, as each blood cell has a set life expectancy.
Certain conditions may trigger additional production of blood cells. This may happen when the oxygen content of body tissues is low, if there is loss of blood or anemia, or if the number of red blood cells decreases. If these things happen, the kidneys produce and release erythropoietin, which is a hormone that stimulates bone marrow to produce more red blood cells.
Bone marrow also produces and releases more white blood cells in response to infections and more platelets in response to bleeding. If a person experiences serious blood loss, yellow bone marrow can activate and transform into red bone marrow.
Healthy bone marrow is important for a range of systems and activities.
The circulatory system touches every organ and system in the body. It involves a number of different cells with a variety of functions. Red blood cells transport oxygen to cells and tissues, platelets travel in the blood to help clotting after injury, and white blood cells travel to sites of infection or injury.
Hemoglobin is the protein in red blood cells that gives them their color. It collects oxygen in the lungs, transports it in the red blood cells, and releases oxygen to tissues such as the heart, muscles, and brain. Hemoglobin also removes carbon dioxide (CO2), which is a waste product of respiration, and sends it back to the lungs for exhalation.
Iron is an important nutrient for human physiology. It combines with protein to make the hemoglobin in red blood cells and is essential for producing red blood cells (erythropoiesis). The body stores iron in the liver, spleen, and bone marrow. Most of the iron a person needs each day for making hemoglobin comes from the recycling of old red blood cells.
The production of red blood cells is called erythropoiesis. It takes about 7 days for a committed stem cell to mature into a fully functional red blood cell. As red blood cells age, they become less active and more fragile.
White blood cells called macrophages remove aging red cells in a process known as phagocytosis. The contents of these cells are released into the blood. The iron released in this process travels either to bone marrow for the production of new red blood cells or to the liver or other tissues for storage.
Typically, the body replaces around 1% of its total red blood cell count every day. In a healthy person, this means that the body produces around 200 billion red blood cells each day.
Bone marrow produces many types of white blood cells. These are necessary for a healthy immune system. They prevent and fight infections.
The main types of white blood cells, or leukocytes, are as follows.
Lymphocytes are produced in bone marrow. They make natural antibodies to fight infection due to viruses that enter the body through the nose, mouth, or another mucous membrane or through cuts and grazes. Specific cells recognize the presence of invaders (antigens) that enter the body and send a signal to other cells to attack them.
The number of lymphocytes increases in response to these invasions. There are two major types of lymphocytes: B and T lymphocytes.
Monocytes are produced in bone marrow. Mature monocytes have a life expectancy in the blood of only 38 hours, but when they move into the tissues, they mature into larger cells called macrophages.
Macrophages can survive in the tissues for long periods of time, where they engulf and destroy bacteria, some fungi, dead cells, and other material that is foreign to the body.
Granulocytes is the collective name given to three types of white blood cells: neutrophils, eosinophils, and basophils. The development of a granulocyte may take 2 weeks, but this time reduces when there is an increased threat, such as a bacterial infection.
Bone marrow stores a large reserve of mature granulocytes. For every granulocyte circulating in the blood, there may be 50100 cells waiting in the bone marrow to be released into the bloodstream. As a result, half the granulocytes in the bloodstream can be available to actively fight an infection in the body within 7 hours of it detecting one.
Once a granulocyte has left the blood, it does not usually return. A granulocyte may survive in the tissues for up to 45 days, depending on the conditions, but it can only survive for a few hours in circulating blood.
Neutrophils are the most common type of granulocyte. They can attack and destroy bacteria and viruses.
Eosinophils are involved in the fight against many types of parasitic infections and against the larvae of parasitic worms and other organisms. They are also involved in some allergic reactions.
Basophils are the least common of the white blood cells. They respond to various allergens that cause the release of histamines, heparin, and other substances.
Heparin is an anticoagulant. It prevents blood from clotting. Histamines are vasodilators that cause irritation and inflammation. Releasing these substances makes a pathogen more permeable and allows for white blood cells and proteins to enter the tissues to engage the pathogen.
The irritation and inflammation in tissues that allergens affect are parts of the reaction associated with hay fever, some forms of asthma, hives, and, in its most serious form, anaphylactic shock.
Bone marrow produces platelets in a process known as thrombopoiesis. Platelets are necessary for blood to coagulate and for clots to form in order to stop bleeding.
Sudden blood loss triggers platelet activity at the site of an injury or wound. Here, the platelets clump together and combine with other substances to form fibrin. Fibrin has a thread-like structure and forms an external scab or clot.
Platelet deficiency causes the body to bruise and bleed more easily. Blood may not clot well at an open wound, and there may be a higher risk of internal bleeding if the platelet count is very low.
The lymphatic system consists of lymphatic organs such as bone marrow, the tonsils, the thymus, the spleen, and lymph nodes.
All lymphocytes develop in bone marrow from immature cells called stem cells. Lymphocytes that mature in the thymus gland (behind the breastbone) are called T cells. Those that mature in bone marrow or the lymphatic organs are called B cells.
The immune system protects the body from disease. It kills unwanted microorganisms such as bacteria and viruses that may invade the body.
Small glands called lymph nodes are located throughout the body. Once lymphocytes are made in bone marrow, they travel to the lymph nodes. The lymphocytes can then travel between each node through lymphatic channels that meet at large drainage ducts that empty into a blood vessel. Lymphocytes enter the blood through these ducts.
Three major types of lymphocytes play an important part in the immune system: B lymphocytes, T lymphocytes, and NK cells.
These cells originate from hematopoietic stem cells in bone marrow in mammals.
B cells express B cell receptors on their surface. These allow the cell to attach to an antigen on the surface of an invading microbe or another antigenic agent.
For this reason, B cells are known as antigen-presenting cells, as they alert other cells of the immune system to the presence of an invading microbe.
B cells also secrete antibodies that attach to the surface of infection-causing microbes. These antibodies are Y-shaped, and each one is akin to a specialized lock into which a matching antigen key fits. Because of this, each Y-shaped antibody reacts to a different microbe, triggering a larger immune system response to fight infection.
In some circumstances, B cells erroneously identify healthy cells as being antigens that require an immune system response. This is the mechanism behind the development of autoimmune conditions such as multiple sclerosis, scleroderma, and type 1 diabetes.
These cells are so-called because they mature in the thymus, which is a small organ in the upper chest, just behind the sternum. (Some T cells mature in the tonsils.)
There are many different types of T cells, and they perform a range of functions as part of adaptive cell-mediated immunity. T cells help B cells make antibodies against invading bacteria, viruses, or other microbes.
Unlike B cells, some T cells engulf and destroy pathogens directly after binding to the antigen on the surface of the microbe.
NK T cells, not to be confused with NK cells of the innate immune system, bridge the adaptive and innate immune systems. NK T cells recognize antigens presented in a different way from many other antigens, and they can perform the functions of T helper cells and cytotoxic T cells. They can also recognize and eliminate some tumor cells.
These are a type of lymphocyte that directly attack cells that a virus has infected.
A bone marrow transplant is useful for various reasons. For example:
Stem cells mainly occur in four places:
Stem cells for transplantation are obtainable from any of these except the fetus.
Hematopoietic stem cell transplantation (HSCT) involves the intravenous (IV) infusion of stem cells collected from bone marrow, peripheral blood, or umbilical cord blood.
This is useful for reestablishing hematopoietic function in people whose bone marrow or immune system is damaged or defective.
Worldwide, more than 50,000 first HSCT procedures, 28,000 autologous transplantation procedures, and 21,000 allogeneic transplantation procedures take place every year. This is according to a 2015 report by the Worldwide Network for Blood and Marrow Transplantation.
This number continues to increase by over 7% annually. Reductions in organ damage, infection, and severe, acute graft-versus-host disease (GVHD) seem to be contributing to improved outcomes.
In a study of 854 people who survived at least 2 years after autologous HSCT for hematologic malignancy, 68.8% were still alive 10 years after transplantation.
Bone marrow transplants are the leading treatment option for conditions that threaten bone marrows ability to function, such as leukemia.
A transplant can help rebuild the bodys capacity to produce blood cells and bring their numbers to acceptable levels. Conditions that may be treatable with a bone marrow transplant include both cancerous and noncancerous diseases.
Cancerous diseases may or may not specifically involve blood cells, but cancer treatment can destroy the bodys ability to manufacture new blood cells.
A person with cancer usually undergoes chemotherapy before transplantation. This eliminates the compromised marrow.
A healthcare professional then harvests the bone marrow of a matching donor which, in many cases, is a close family member and ready it for transplant.
Types of bone marrow transplant include:
A persons tissue type is defined as the type of HLA they have on the surface of most of the cells in their body. HLA is a protein, or marker, that the body uses to help it determine whether or not the cell belongs to the body.
To check if the tissue type is compatible, doctors assess how many proteins match on the surface of the donors and recipients blood cells. There are millions of different tissue types, but some are more common than others.
Tissue type is inherited, and types pass on from each parent. This means that a relative is more likely to have a matching tissue type.
However, if it is not possible to find a suitable bone marrow donor among family members, healthcare professionals try to find someone with a compatible tissue type on the bone marrow donor register.
Healthcare professionals perform several tests before a bone marrow transplant to identify any potential problems.
These tests include:
In addition, a person needs a complete dental exam before a bone marrow transplant to reduce the risk of infection. Other precautions to lower the risk of infection are also necessary before the transplant.
Bone marrow is obtainable for examination by bone marrow biopsy and bone marrow aspiration.
Bone marrow harvesting has become a relatively routine procedure. Healthcare professionals generally aspirate it from the posterior iliac crests while the donor is under either regional or general anesthesia.
Healthcare professionals can also take it from the sternum or from the upper tibia in children, as it still contains a substantial amount of red bone marrow.
To do so, they insert a needle into the bone, usually in the hip, and withdraw some bone marrow. They then freeze and store this bone marrow.
National Marrow Donor Program (NMDP) guidelines limit the volume of removable bone marrow to 20 milliliters (ml) per kg of donor weight. A dose of 1 x 103 and 2 x 108 marrow mononuclear cells per kg is necessary to establish engraftment in autologous and allogeneic marrow transplants, respectively.
Complications related to bone marrow harvesting are rare. When they do occur, they typically involve problems related to anesthetics, infection, and bleeding.
More:
Bone marrow: Function, diseases, transplants, and donation
BioRestorative Therapies, Inc. Releases Year-End Message – BioSpace
By daniellenierenberg
MELVILLE, N.Y., Dec. 20, 2021 (GLOBE NEWSWIRE) -- BioRestorative Therapies, Inc. (the Company" or BioRestorative) (NASDAQ:BRTX), a life sciences company focused on adult stem cell-based therapies, today released the following year-end message.
As we reach the end of 2021, we are inspired by the many healthcare workers and biopharmaceutical companies that have worked to combat the COVID-19 pandemic. This year has been environmentally difficult, but we have seen incredible advancements in our sector which have reinforced the importance of our mission to become a clinical stage company. Since our emergence from Chapter 11 in 2020, we have sought to take positive steps at BioRestorative Therapies with the goal of making it a preeminent cell therapy company. During 2021, we achieved important transformational milestones, which created meaningful intrinsic value and advanced us toward our stated strategic goals.
In November of this year, we closed on a $23 million capital raise and concurrently listed our securities on the Nasdaq Capital Market. This is a very significant development as we are now fully funded to complete our Phase 2 trial for our lead clinical candidate, BRTX-100, for the treatment of chronic lumbar disc disease (CLDD.) During this process, we have attracted many new institutional fundamental investors as well as some retail investors. With that accomplished, I would like to briefly discuss the status of our programs and the opportunities that lie ahead of us.
BRTX-100 is our lead program for the treatment of CLDD, one of the leading causes of lower back pain. Our solution is a one-time injection of 40 million mesenchymal stem cells derived from a patients own bone marrow and expanded ex vivo before re-injection. Two things make us optimistic about this program. First, in connection with our IND filing, we referred the FDA to prior human clinical studies from different institutions that demonstrated the safety/feasibility of using mesenchymal stem cells to treat disc orders. This data not only enabled us to accelerate our clinical program and initiate a Phase 2 trial, but we believe it substantially reduces risk in offering compelling guidance on the use of cell-based interventions to treat lower back pain. Second, our manufacturing of BRTX-100 involves the use of low oxygen conditions, which ensures that the cells have enhanced survivability after introduction into the harsh avascular environment of the injured disc which has little or no blood flow. The benefits of this process are significant and are illustrated well in our recent Journal of Translational Medicine publication. Our approach is akin to transplant medicine in which specific cell types are used to replace the ones which have been lost to disease. We believe that transplanting targeted cells can offer a more attractive safety profile and potentially an improved clinical outcome. We remain optimistic that we will see significant positive clinical outcomes as we proceed with our clinical trial.
The most significant milestones we achieved in 2021 include:
Our 2022 objectives include the initiation of enrollment for our BRTX-100 clinical trial, the development of our overall product profiles via manufacturing and delivery system improvements, and the entering into of technology validation and enabling partnerships to accelerate our clinical timelines.
Some of the events and milestones that we hope to accomplish in 2022 include:
This is an exciting time to be part of the BioRestorative family. As we enter 2022 with a well-capitalized balance sheet to fully fund our Phase 2 trial, we look to accelerate our research and development pipeline. We do not take for granted that our technologies give us an opportunity to make a profound impact on the everyday lives of many people. We are grateful for the opportunity to validate such technologies; it is what we do and what we believe is the center of our core competencies.
Visit our website at http://www.biorestorative.com for more information about BioRestorative.
Thank you to the BioRestorative family for your loyalty and ongoing support.
I wish you and all those near and dear to you a wonderful Holiday Season and the very best for 2022 and beyond.
Very truly yours,
Lance AlstodtPresident, CEO and Chairman of the Board
About BioRestorative Therapies, Inc.
BioRestorative Therapies, Inc. (www.biorestorative.com) develops therapeutic products using cell and tissue protocols, primarily involving adult stem cells. Our two core programs, as described below, relate to the treatment of disc/spine disease and metabolic disorders:
Disc/Spine Program (brtxDISC): Our lead cell therapy candidate, BRTX-100, is a product formulated from autologous (or a persons own) cultured mesenchymal stem cells collected from the patients bone marrow. We intend that the product will be used for the non-surgical treatment of painful lumbosacral disc disorders or as a complementary therapeutic to a surgical procedure. The BRTX-100 production process utilizes proprietary technology and involves collecting a patients bone marrow, isolating and culturing stem cells from the bone marrow and cryopreserving the cells. In an outpatient procedure, BRTX-100 is to be injected by a physician into the patients damaged disc. The treatment is intended for patients whose pain has not been alleviated by non-invasive procedures and who potentially face the prospect of surgery. We have received authorization from the Food and Drug Administration to commence a Phase 2 clinical trial using BRTX-100 to treat chronic lower back pain arising from degenerative disc disease.
Metabolic Program (ThermoStem): We are developing a cell-based therapy candidate to target obesity and metabolic disorders using brown adipose (fat) derived stem cells to generate brown adipose tissue (BAT). BAT is intended to mimic naturally occurring brown adipose depots that regulate metabolic homeostasis in humans. Initial preclinical research indicates that increased amounts of brown fat in animals may be responsible for additional caloric burning as well as reduced glucose and lipid levels. Researchers have found that people with higher levels of brown fat may have a reduced risk for obesity and diabetes.
FORWARD-LOOKING STATEMENTS
This letter contains "forward-looking statements" within the meaning of Section 27A of the Securities Act of 1933, as amended, and Section 21E of the Securities Exchange Act of 1934, as amended, and such forward-looking statements are made pursuant to the safe harbor provisions of the Private Securities Litigation Reform Act of 1995. You are cautioned that such statements are subject to a multitude of risks and uncertainties that could cause future circumstances, events or results to differ materially from those projected in the forward-looking statements as a result of various factors and other risks, including, without limitation, those set forth in the Company's latest Form 10-K filed with the Securities and Exchange Commission (SEC) and other filings made with the SEC. You should consider these factors in evaluating the forward-looking statements included herein, and not place undue reliance on such statements. The forward-looking statements in this letter are made as of the date hereof and the Company undertakes no obligation to update such statements.
CONTACT:
Email: ir@biorestorative.com
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BioRestorative Therapies, Inc. Releases Year-End Message - BioSpace
Communication between cells plays a major role in deciding their fate > News > USC Dornsife – USC Dornsife College of Letters, Arts and Sciences
By daniellenierenberg
Findings from a new study could point the way to new treatments for blood diseases including cancers such as leukemia and lymphoma. [3 min read]
In this schematic, cells (black spheres) within each well are committed to a specific fate, but external stimuli, such as cell-to-cell communication, can force cells out of one state and into another. (Illustration: Courtesy of Adam MacLean.)
Scientists have found a way to prove that biochemical signals sent from cell to cell play an important role in determining how those cells develop.
The study from researchers at the USC Dornsife College of Letters, Arts and Sciences was published in the journal Development on Dec. 22.
A little background:
Whats new:
We discovered that the communication process can change the formation of blood cell types dramatically, and that cells that are closer to one another have a greater influence on each others fate, MacLean said.
A controversy resolved
Researchers trying to determine what early factors nudge a cell down one developmental path or another have wondered if random fluctuations within the cell are enough to decide which path is taken. Many models have suggested they were, but recent breakthrough studies showed that random fluctuations were not enough, that something else drives cells toward their fate.
The model MacLean and Rommelfanger have developed appears to put an end to the controversy altogether. They show that cell-to-cell communication can, in fact, be the deciding factor that sets cells along a certain path.
Why it matters:
By understanding how blood cell fate decisions are made, MacLean said, we get closer to being able to identify leukemia cells of origin, and in theory we can design strategies to control or alter cell fate decision-making and stop the development of cancer.
The research could help improve cancer therapies such as bone marrow transplant.
Better understanding stem cell fate decisions, as our study provides, could provide new insight to improve clinical outcomes for these diseases, MacLean said.
More than just blood
This new model has important implications beyond the blood system.
Our model is broadly applicable, so researchers working on other cell types can apply it to find out for those other cells how important cell-to-cell communication may be, said MacLean.
Whats next:
The role of cell-to-cell communication in determining cell fate is in its nascent stages, says MacLean, but further experiments and future technologies to integrate these new types of data with sophisticated models should help expand understanding.
In addition, the team is developing methods to study the regulation of key genes involved in cell fate decisions, which should further advance their overall theoretical model.
About the study
This work was supported by National Science Foundation grant DMS 2045327 and a USC Women in Science and Engineering Top-up Fellowship.
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Communication between cells plays a major role in deciding their fate > News > USC Dornsife - USC Dornsife College of Letters, Arts and Sciences
Importance of stem cells-Past, present and future – Express Healthcare
By daniellenierenberg
Dr Pradeep Mahajan, Regenerative Medicine Researcher, StemRx Bioscience Solutions highlights the importance and other aspects of stem cell technology
Globally, we are seeing a change in the type of age-specific, chronic, debilitating diseases. Thus, the manner in which we diagnose and treat such diseases is also seeing a paradigm shift. From empirical use of drugs to target-specific treatments, we are now advancing towards molecular dysfunction-based therapies.
I have been in the field of clinical medicine and surgery for over 3 decades now and I have always been fascinated by new research. Among the substantial advances in the healthcare field, I believe regenerative medicine and cell-based therapy have been game changers. We saw hematopoietic stem cells being used to treat blood cancers and related diseases for over 3-4 decades. Now we are seeing an expansion in the applications of stem cells in treating various acute, chronic, lifestyle, and even genetic and congenital diseases. The need arose because conventional medicine is gradually losing potency in treating diseases and patients are often left at the mercy of nature to take its course.
With increasing knowledge of stem cells, the trend to utilise the endogenous repair mechanisms of the human body gained popularity. Cells, growth factors and other biological products, when present at the right site; at the right moment, stimulate the natural healing mechanisms of the body and aid in management of health conditions. Cell-based therapy thus marked the beginning of a new era in regenerative medicine.
Stem cells are present in several tissues, namely, embryo, umbilical cord, placenta, as well as adult body tissues. These are the master cells of the body that have roles in development of the body, repairing and regenerating injured tissues (at a cellular level), and maintaining homeostasis even in an healthy individual. Of course, we have all heard of ethical issues regarding the use of embryonic stem cells as well as their tumor-forming issue. Regarding umbilical cord stem cells, the trend of banking this tissue has just begun; therefore, the majority of us would not have the umbilical cord as a source of stem cells. Keeping in mind these aspects, researchers started focusing on adult stem cells that can be derived from different tissues of the human body. The common sources are bone marrow, fat tissue, peripheral blood, and teeth, among others. The chief advantage is that, the source being autologous, the therapy is safe and is not associated with side effects.
Coming to the diseases that can be treated using stem cellswe have just scratched the tip of the iceberg. There are several health conditions that plague mankindarthritis, diabetes, nerve-related conditions, traumatic injuries, etc. Conventionally, one would be prescribed medications (often for prolong periods or even for their lifetime) or be advised surgery. Nonetheless, in several cases, the quality of life of a patient is compromised. The various properties of stem cells help reduce swelling in the body, regulate the immune system, enhance the functioning of other cells, and create a healthy environment for health cells to thrive. Through this, one can target a myriad of pathologies at the molecular level, in a minimally/non-invasive manner.
Patients today are quite aware of the benefits of regenerative medicine and cell based therapy, but there is still a long distance to cover. Countries are promoting research and development in the field of regenerative medicine and cell-based therapy. Research advances pertaining to introducing products with cell and scaffold based technology through tissue engineering are underway. Bioactive scaffolds that are capable of supporting activation and differentiation of host stem cells at the required site are being developed. In the future, it will be possible to use human native sites as micro-niche/micro-environment for potentiation of the human bodys site-specific response. Another breakthrough in the field of cell-based therapy is immunotherapy that aims to utilise certain parts of a persons immune system and stimulate them to fight diseases such as cancer.
The scope of cell-based therapy is endless. All we need is more research, awareness, and implementation to permit reach of the treatment to every stratum of the society. Soon, we will talk about treating diseases with cells and not pills and knives!
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Importance of stem cells-Past, present and future - Express Healthcare
Dihydroartemisinin Promoted Bone Marrow Mesenchymal Stem Cell Homing and Suppressed Inflammation and Oxidative Stress against Prostate Injury in…
By daniellenierenberg
Although bone marrow mesenchymal stem cells (BMMSCs) are effective in treating chronic bacterial prostatitis (CBP), the homing of BMMSCs seems to require ultrasound induction. Dihydroartemisinin (DHA) is an important derivative of artemisinin (ART) and has been previously reported to alleviate inflammation and autoimmune diseases. But the effect of DHA on chronic prostatitis (CP) is still unclear. This study aims to clarify the efficacy and mechanism of DHA in the treatment of CBP and its effect on the accumulation of BMMSCs. The experimental CBP was produced in C57BL/6 male mice via intraurethrally administeredE. colisolution. Results showed that DHA treatment concentration-dependently promoted the accumulation of BMMSCs in prostate tissue of CBP mice. In addition, DHA and BMMSCs cotreatment significantly alleviated inflammation and improved prostate damage by decreasing the expression of proinflammatory factors such as TNF-, IL-1, and chemokines CXCL2, CXCL9, CXCL10, and CXCL11 in prostate tissue of CBP mice. Moreover, DHA and BMMSCs cotreatment displayed antioxidation property by increasing the production of glutathione peroxidase (GSH-Px), SOD, and decreasing malondialdehyde (MDA) expression. Mechanically, DHA and BMMSCs cotreatment significantly inhibited the expression of
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Dihydroartemisinin Promoted Bone Marrow Mesenchymal Stem Cell Homing and Suppressed Inflammation and Oxidative Stress against Prostate Injury in...
City of Hope presents leading-edge research on blood cancer therapies and its vaccine to reduce stem cell transplant complications at American Society…
By daniellenierenberg
DUARTE, Calif.--(BUSINESS WIRE)--City of Hope doctors presented data on an investigational bispecific antibody for multiple myeloma and the CMVPepVax, a City of Hope-developed vaccine against the cytomegalovirus, at this years ASH Annual Meeting.
City of Hope continues to be a leader in innovative research on investigational immunotherapies for blood cancers and improving stem cell transplants, said Eileen Smith, M.D., City of Hopes Francis & Kathleen McNamara Distinguished Chair in Hematology and Hematopoietic Cell Transplantation. New research at this years ASH conference includes promising investigational immunotherapies for lymphoma, multiple myeloma, leukemia and other blood cancers and an update on a City of Hope-developed vaccine to prevent a virus that can cause serious complications in stem cell transplant recipients.
Here are highlights of City of Hope research presented at the ASH conference:
Investigational bispecific antibody for multiple myeloma is well-tolerated and effective
Bispecific antibodies are an emerging immunotherapy against blood cancers. City of Hopes Elizabeth Budde, M.D., Ph.D., presented at this years ASH conference on mosunetuzumab. The research demonstrated that mosunetuzumab is a safe and effective investigational bispecific antibody for follicular lymphoma.
Talquetamab is an investigational therapy that is also demonstrating encouraging results for the treatment of relapsed multiple myeloma, according to a study led by Amrita Krishnan, M.D., director of the Judy and Bernard Briskin Center for Multiple Myeloma Research at City of Hope and chief, Division of Multiple Myeloma.
Talquetamab targets the G protein-coupled receptor family C group 5 member (GPRC5D) that has a high expression on malignant plasma cells and is limited on normal human tissue. The first-in-class bispecific antibody directs T cells to kill multiple myeloma cells by binding to both GPRC5D and CD3 receptors.
Patients with relapsed or difficult to treat multiple myeloma in the Phase 1 study received recommended Phase 2 doses as an injection on a weekly or biweekly basis. By increasing the doses slowly, researchers hope that will help to minimize the severity of cytokine release syndrome.
Krishnan presented data on 55 patients. For the study, 30 patients who received the therapy weekly (and their results were evaluable, meaning they could be included in the study) and 23 people who received it on a biweekly schedule were included. The study is ongoing.
In the weekly cohort, the overall response rate was 70% and there was a very good partial response or better in 57% of patients.
The response numbers are very strong and whats also remarkable is that the responses were durable and deepened over time in both groups, Krishnan said.
Cytokine release syndrome occurred in 73% of the weekly dose cohort, but only one patient had a severe case and it was treatable. Other side effects included neutropenia and dysgeusia.
We are excited that our results demonstrated that talquetamab is well-tolerated and highly effective at the Phase 2 dose level and with tolerable side effects, Krishnan said.
Further studies of the therapy on its own or in combination with other treatments for multiple myeloma are underway.
City of Hope-developed vaccine to prevent cytomegalovirus shows safety, tolerability
Despite therapies to help prevent the cytomegalovirus (CMV), which can flare up in blood marrow/stem cell transplant recipients who are immunocompromised, CMV infections are one of the most common complications in these patients. Furthermore, the antiviral drugs used to prevent flare-ups are toxic, expensive and increase the risk of other opportunistic infections.
City of Hope has developed an anti-CMV vaccine, known as CMVPepVax. At this years ASH conference, the results of a Phase 2 trial using CMVPepVax were reported by Ryotaro Nakamura, M.D., City of Hopes Jan & Mace Siegel Professor in Hematology & Hematopoietic Cell Transplantation in the Division of Leukemia.
The double blinded, placebo-controlled, randomized Phase 2 trial enrolled stem cell transplant recipients from four transplant centers, including City of Hope. Nakamura reported on data from 32 patients in the vaccine arm and 29 patients in the placebo arm.
CMVPepVax was delivered via injections 28 days after transplant and 56 days after the procedure.
Trial results demonstrated that there was no difference in CMV reactivation in both arms.
CMVPepVax was well-tolerated in patients with no increase in adverse side effects. Transplant outcomes were also similar between the two groups when comparing one-year overall survival, relapse-free survival, nonrelapse mortality, relapse and acute graft-versus-host disease (GVHD).
Significantly higher levels of CMV-targeting T cells were measured in patients in the vaccine arm who did not have CMV in their bloodstream. In patients who had the CMVPepVax injections, robust expansion of functional T cells also occurred.
Our results confirm that CMVPepVax is safe to use and provides an immune response, Nakamura said. Although the vaccine did not reduce the presence of CMV in the bloodstream, there were favorable CD8 T cell responses, which are protective in principle, but maybe didn't recover fast enough to prevent CMV from reactivating.
Next steps include researching whether stem cell donors who receive the vaccine can transfer immunity to patients, as well as providing a booster to patients. This may lead to faster immune responses after transplant.
Using probiotics for stem cell transplant patients
City of Hope is a leader in bone marrow and stem cell transplantation it was one of the first cancer centers nationwide to perform a bone marrow transplant and has performed more than 17,000 bone marrow/stem cell transplants since 1976. Because of this leadership, City of Hope doctors and scientists are investigating how to make the transplant process better, as well as how to deal with complications that may arise from the procedure, such as GVHD.
Led by Karamjeet S. Sandhu, M.D., an assistant professor in City of Hope's Division of Leukemia in the Department of Hematology & Hematopoietic Cell Transplantation, a City of Hope study examined how adding the probiotic CBM 588 to transplant recipients diets might decrease inflammation in the gut and lower the risk of GVHD. The results were discussed in an oral presentation at the ASH conference.
Sandhu explained that the body hosts microbial communities, known as the microbiome. These microbes help the body in several metabolic processes, such as digesting food, strengthening the immune system, protecting against other bacteria and producing vitamins, including B vitamins.
Recent studies have shown the microbiome can play a role in cancer risk and how a persons body responds to cancer treatment. In people with blood cancers who receive a transplant, there is a direct link between the health of microbiome and survival.
Imbalance among these microbial species have also been associated with several transplant complications including GVHD, said Sandhu, M.D. He added that the imbalance also contributes to morbidity and mortality.
For the study, Sandhu and his team used Clostridium Butyricum Miyairi 588 (CBM588), a probiotic strain that has been used in Japan for several decades to manage diarrhea caused by antibiotics or infections. CBM588 is a butyrate-producing bacteria present in the spore form in soil and food. Administration of CBM588 has shown anti-inflammatory and immune modulating effects, as well as evidence of anti-cancer activity.
This was the first study of CBM588 among bone marrow/stem cell transplant recipients. Fifteen patients received the current standard of care therapies to prevent GVHD and 21 received CBM588 in addition to standard of care for GVHD.
Our study demonstrated that CMB588 is safe and feasible to use in this patient population without increasing mortality, Sandhu said. We even noted an improvement in gastrointestinal GVHD, but further studies are needed to prove the effect and mechanism of action among recipients of bone marrow/stem cell transplantation.
Joint study examines somatic mutations in CMML patients, impact on stem cell transplants
Chronic myelomonocytic leukemia (CMML) is a rare form of leukemia that primarily affects older adults. The only potential cure at this time is allogeneic hematopoietic cell transplantation, also known as a stem cell transplant.
Research has shown that somatic mutations genetic changes that are acquired during life and not inherited are an important factor in determining prognosis for CMML patients. However, limited data are available regarding their impact on outcomes after CMML patients receive transplant.
A joint study between City of Hope and Center for International Blood and Marrow Transplant Research (CIBMTR) analyzed the relationship between somatic mutations in CMML and their impact on stem cell transplants.
Additionally, the study aimed to evaluate two separate scoring systems commonly used in nontransplant CMML patients, the CMML-specific prognostic scoring system (CPSS) and molecular CPSS (CPSS-Mol), which takes into account the somatic mutations, to find out if they can predict the results of a transplant.
Led by City of Hopes Matthew Mei, M.D., an associate professor in City of Hopes Division of Lymphoma, Department of Hematology & Hematopoietic Cell Transplantation, the study included 313 patients across 78 different transplant centers, all of whom underwent a comprehensive mutation analysis of 131 genes performed at City of Hope under the supervision of Raju K. Pillai, M.D., director of Pathology Core Laboratories in Beckman Research Institute of City of Hope.
The study found that 93% of patients had at least one mutation identified, and the median number of mutations was three. The most frequently mutated genes were ASXL1 (62%), TET2 (35%), KRAS/NRAS (33% combined) and SRSF2 (31%); TP53 was mutated in 3% of patients.
Both the CPSS and CPSS-Mol were predictive of overall survival after transplant; however, neither system was able to identify patients who were at an increased risk of relapse. Furthermore, the incorporation of somatic mutations did not appear to refine the prognosis.
Our study is the largest analysis of CMML patients who underwent a stem cell transplant with paired mutation analysis, Mei said. Overall, patients with CMML remain at high risk for relapse after transplant. Novel therapies aimed at decreasing relapse and making transplants safer, as well as improved methods of predicting outcomes of transplant in CMML, are still critically needed.
Additional research on chimeric antigen receptor (CAR) T therapy and stem cell transplantation presented at ASH
Tanya Siddiqi, M.D., director of City of Hope's Chronic Lymphocytic Leukemia Program, also presented a poster on the Transcend NHL 001 trial at the ASH conference, and Ibrahim Aldoss, M.D., associate professor, City of Hope's Division of Leukemia, presented a poster on the outcomes of allogeneic hematopoietic cell transplantation in adults with Ph-like acute lymphoblastic leukemia.
City of Hope is a leader in blood cancer research and treatment. The National Cancer Institute-designated comprehensive cancer center has performed more than 17,000 bone marrow/stem cell transplants and is a leader in chimeric antigen receptor (CAR) T therapy, with nearly 800 patients treated with immune effector cells, including CAR T therapy, and nearly 80 open or completed trials.
About City of Hope
City of Hope is an independent biomedical research and treatment center for cancer, diabetes and other life-threatening diseases. Founded in 1913, City of Hope is a leader in bone marrow transplantation and immunotherapy such as CAR T cell therapy. City of Hopes translational research and personalized treatment protocols advance care throughout the world. Human synthetic insulin, monoclonal antibodies and numerous breakthrough cancer drugs are based on technology developed at the institution. A National Cancer Institute-designated comprehensive cancer center and a founding member of the National Comprehensive Cancer Network, City of Hope is ranked among the nations Best Hospitals in cancer by U.S. News & World Report. Its main campus is located near Los Angeles, with additional locations throughout Southern California and in Arizona. Translational Genomics Research Institute (TGen) became a part of City of Hope in 2016. AccessHope, a subsidiary launched in 2019, serves employers and their health care partners by providing access to NCI-designated cancer center expertise. For more information about City of Hope, follow us on Facebook, Twitter, YouTube or Instagram.
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City of Hope presents leading-edge research on blood cancer therapies and its vaccine to reduce stem cell transplant complications at American Society...
Analysis of Peripheral Blood Mononuclear Cells Gene Expression Highlights the Role of Extracellular Vesicles in the Immune Response following…
By daniellenierenberg
Abstract: Hematopoietic stem cell transplantation (HSCT) is an effective treatment method used in many neoplastic and non-neoplastic diseases that affect the bone marrow, blood cells, and immune system.The procedure is associated with a risk of adverse events, mostly elated to the immune response after transplantation. The aim of our research was to identify genes, processes and cellular entities involved in the variety of changes occurring after allogeneic HSCT in children by performing a whole genome expression assessment together with pathway enrichment analysis. We conducted a prospective study of 27 patients (aged 1.518 years) qualified for allogenic HSCT. Blood samples were obtained before HSCT and 6 months after the procedure. Microarrays were used to analyze gene expressions in peripheral blood mononuclear cells. This was followed by Gene Ontology (GO) functional enrichment analysis, Kyoto Encyclopedia of Genes and Genomes (KEGG) pathway enrich- ment analysis, and proteinprotein interaction (PPI) analysis using bioinformatic tools. We found 139 differentially expressed genes (DEGs) of which 91 were upregulated and 48 were downregulated. Blood microparticle, extracellular exosome, B-cell receptor signaling pathway, complement activation and antigen binding were among GO terms found to be significantly enriched. The PPI analysis identified 16 hub genes. Our results provide insight into a broad spectrum of epigenetic changes that occur after HSCT. In particular, they further highlight the importance of extracellular vesicles (exosomes and microparticles) in the post-HSCT immune response.
Stem Cells Market to Witness Gigantic Growth by 2026 LSMedia – LSMedia
By daniellenierenberg
Advance Market Analytics published a new research publication on Stem Cells Market Insights, to 2026 with 232 pages and enriched with self-explained Tables and charts in presentable format. In the Study you will find new evolving Trends, Drivers, Restraints, Opportunities generated by targeting market associated stakeholders. The growth of the Stem Cells Market was mainly driven by the increasing R&D spending across the world.
Some of the key players profiled in the study are:
Smith & Nephew (United Kingdom),Celgene Corporation (United States),BIOTIME, INC. (United States),Cynata (Australia),Applied Cell Technology (Hungary),STEMCELL Technologies Inc. (Canada),BioTime Inc. (United States),Cytori Therapeutics, Inc. (United States),Astellas Pharma Inc. (Japan),U.S. Stem Cell, Inc. (United States),Takara Holdings. (Japan)
Get Free Exclusive PDF Sample Copy of This Research @ https://www.advancemarketanalytics.com/sample-report/72815-global-stem-cells-market-1
Scope of the Report of Stem Cells
The stem cell is used for treating chronic diseases such as cardiovascular disorders, cancer, diabetes, and others. Growing research and development in stem cell isolation techniques propelling market growth. For instance, a surgeon from Turkey developed a method for obtaining stem cells from the human body without enzymes which are generally used for the isolation of stem cells. Further, growing healthcare infrastructure in the developing economies and government spending on the life science research and development expected to drive the demand for stem cell market over the forecasted period.
Market Trend:
Emphasizing On Development of Regenerative Medicine
Technological Advancement in Stem Cell Harvesting and Isolation Techniques
Market Drivers:
Rising Prevalence of Chronic Diseases such as Cardiovascular Disorders, Cancer, and others
Growing Healthcare Infrastructure in the Developing Economies
Challenges:
Lack of Awareness Regarding Stem Cell Therapy in the Low and Middle Income Group Countries
Opportunities:
Growing Demand for Cellular Therapies
Rising Application of Autologous Therapy
The titled segments and sub-section of the market are illuminated below:by Type (Adult Stem Cells (Neuronal, Hematopoietic, Mesenchymal, Umbilical Cord, Others), Human Embryonic Stem Cells (hESC), Induced Pluripotent Stem Cells, Very Small Embryonic-Like Stem Cells), Application (Regenerative Medicine (Neurology, Orthopedics, Oncology, Hematology, Cardiovascular and Myocardial Infraction, Injuries, Diabetes, Liver Disorder, Incontinence, Others), Drug Discovery and Development), Technology (Cell Acquisition (Bone Marrow Harvest, Umbilical Blood Cord, Apheresis), Cell Production (Therapeutic Cloning, In-vitro Fertilization, Cell Culture, Isolation), Cryopreservation, Expansion and Sub-Culture), Therapy (Autologous, Allogeneic)
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Region Included are: North America, Europe, Asia Pacific, Oceania, South America, Middle East & Africa
Country Level Break-Up: United States, Canada, Mexico, Brazil, Argentina, Colombia, Chile, South Africa, Nigeria, Tunisia, Morocco, Germany, United Kingdom (UK), the Netherlands, Spain, Italy, Belgium, Austria, Turkey, Russia, France, Poland, Israel, United Arab Emirates, Qatar, Saudi Arabia, China, Japan, Taiwan, South Korea, Singapore, India, Australia and New Zealand etc.
Strategic Points Covered in Table of Content of Global Stem Cells Market:
Chapter 1: Introduction, market driving force product Objective of Study and Research Scope the Stem Cells market
Chapter 2: Exclusive Summary the basic information of the Stem Cells Market.
Chapter 3: Displaying the Market Dynamics- Drivers, Trends and Challenges of the Stem Cells
Chapter 4: Presenting the Stem Cells Market Factor Analysis Porters Five Forces, Supply/Value Chain, PESTEL analysis, Market Entropy, Patent/Trademark Analysis.
Chapter 5: Displaying market size by Type, End User and Region 2015-2020
Chapter 6: Evaluating the leading manufacturers of the Stem Cells market which consists of its Competitive Landscape, Peer Group Analysis, BCG Matrix & Company Profile
Chapter 7: To evaluate the market by segments, by countries and by manufacturers with revenue share and sales by key countries (2021-2026).
Chapter 8 & 9: Displaying the Appendix, Methodology and Data Source
Finally, Stem Cells Market is a valuable source of guidance for individuals and companies in decision framework.
Read Detailed Index of full Research Study at @ https://www.advancemarketanalytics.com/reports/72815-global-stem-cells-market-1
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Stem Cells Market to Witness Gigantic Growth by 2026 LSMedia - LSMedia
Stem cell therapy holds immense promise for the treatment of patients with non-healing ischemic leg wounds – Ibcworldnews
By daniellenierenberg
Mysuru
An 88-year-old gentleman presented to Manipal Hospital Mysore with blackish discoloration of the heel of left foot. He was diabetic & was on regular treatment for the same. For the current problem, he had already received several medications including intra venous antibiotics with little improvement. Upon examination he was detected to have Critical Limb Ischemia (CLI) with gangrene of heel of left foot. Large number of such patients end up with amputation of leg. Our aim in such situation is to first try to save the limb. Amputation should be the last resort when everything else fails. Said Dr. Upendra Shenoy Cardiothoracic and Vascular Surgeon Manipal Hospital Mysore, while giving details about the patient. While addressing the media Dr. C B Keshavamurthy Consultant Interventional Cardiology, Manipal Hospital Mysore said, Patients angiogram showed diffuse disease in all blood vessels of the leg with critical blocks in many areas.
We performed angioplasty on the limb. The procedure improved the blood supply to the limb, but additional treatment was required to restore blood circulation to the foot and toes. Dr. Shenoy and team decided to implement stem cell therapy, hybrid procedure of peripheral angioplasty with stem cell injection. First of its kind procedure in Mysore. Stem cell therapy involves the injection of stem cells obtained from the bone marrow of healthy individuals.
These stem cells can transform themselves into different tissues according to the requirement. In this case, the stem cells stimulate the formation of new blood vessels, said Dr Upendra Shenoy while explaining about the therapy. Dr Shenoy further added, On the day after angioplasty, we injected the stem cell into the calf muscles of the patient.
The dose depends upon the weight of the patient. If the weight is below 60 kg, the doctor injects about 150 million stem cells. In patients with more than 60 kg, the dose is about 200 million.
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Stem cell therapy holds immense promise for the treatment of patients with non-healing ischemic leg wounds - Ibcworldnews
Systemic Mastocytosis Treatments Gain Hope Due To Increasing Novel Treatment Options – PRNewswire
By daniellenierenberg
PALM BEACH, Fla., Dec. 21, 2021 /PRNewswire/ -- FinancialNewsMedia.com News Commentary - Systemic mastocytosis is rare disease which affects very few people and it causes due to C-kit mutation which leads to higher number of mast cell production in the body resulting in accumulation of excessive mast cells in the internal body organs such as spleen, liver, bone marrow and small intestine etc. Recently, the World Health Organization (WHO) updated the prognosis, diagnosis and systemic mastocytosis treatment guidelines for the disease which in turn helped to bring uniformity in the approach by healthcare professionals. The manufacturers in the systemic mastocytosis treatment market are focusing on evaluating possible treatment options to cure the disease by investing heavily in the research & development. Various leading manufacturers are focusing on gaining FDA approval to respective drugs for the systemic mastocytosis treatment to enhance their revenue generation. A reportfrom Future Market Insights said:"Increasing awareness about the systemic mastocytosis treatment as well as symptoms of the disease due to extended effort by non-profit organizations, governmental associations and through other platforms expected to drive the growth of the systemic mastocytosis treatment market Increasing approvals and launches of drugs for the systemic mastocytosis treatment expected to drive the growth of the market Increasing spending on research & development by various pharmaceutical companies to develop novel systemic mastocytosis treatment expected to further fuel the growth of market. Increasing early diagnosis rate subsequently followed by increasing treatment seeking rate further expected to drive the growth of the systemic mastocytosis treatment market." Active companies in the markets today include: Hoth Therapeutics, Inc. (NASDAQ:HOTH), Longeveron Inc. (NASDAQ: LGVN), Bristol Myers Squibb (NYSE: BMY), Takeda Pharmaceutical Company Limited (NYSE: TAK), Amgen (NASDAQ: AMGN).
Future Market Insights continued:"The global systemic mastocytosis treatment market is expected to experience significant growth due to increasing novel treatment options. By drug class, systemic mastocytosis treatment market is expected to be dominated by the mast cell stabilizers due to superior efficacy. By indication, systemic mastocytosis treatment market is expected to be dominated by indolent systemic mastocytosis (ISM) due to higher prevalence. By route o administration, systemic mastocytosis treatment market is expected to be dominated by injectables. By distribution channel, systemic mastocytosis treatment market is expected to be dominated by the retail pharmacies due to higher patient footfall. The global systemic mastocytosis treatment market is expected to be dominated by the North America due to comparatively higher prevalence of the disease. Europe systemic mastocytosis treatment market is expected to be second most lucrative market due to higher treatment seeking rate. Latin America expected to show gradual growth in the systemic mastocytosis treatment market due to steadily increasing diagnosis. Asia-Pacific is emerging systemic mastocytosis treatment market due to increasing diagnosis subsequently followed by treatment. Middle East & Africa is the least lucrative systemic mastocytosis treatment market due to least diagnostic rate and lower awareness about the symptoms."
Hoth Therapeutics, Inc. (NASDAQ:HOTH) BREAKING NEWS: Hoth Therapeutics Announces Submission of Orphan Designation Application for HT-KIT to Treat Mastocytosis Hoth Therapeutics, Inc., a patient-focusedclinical-stage biopharmaceutical company, announced it submitted an Orphan Drug Designation Application to the US Food and Drug Administration (FDA) for HT-KIT for the treatment of mastocyctosis. HT-KIT is an antisense oligonucleotide that targets the proto-oncogene cKIT by inducing mRNA frame shifting, resulting in apoptosis of neoplastic mast cells. The KIT signaling pathway is implicated in multiple diseases, including all types of mastocytosis (such as aggressive systemic mastocytosis (ASM), mast cell leukemia (MCL), and systemic mastocytosis with associated hematological neoplasm (SM-AHN)), acute myeloid leukemia, gastrointestinal stromal tumors, and anaphylaxis.
Drugs intended to treat orphan diseases (rare diseases that affect less than 200,000 people in the US)are eligible to apply for Orphan Drug Designation (ODD), which provides multiple benefits to the sponsor during development and after approval. Hoth intends to pursue these benefits as part of the drug development for HT-KIT for treatment of mastocytosis, pending designation of the ODD application.
Benefits of Orphan Drug Designation - Under the Orphan Drug Act, drug companies can apply for ODD, and if granted, the drug will have a status which gives companies exclusive marketing and development rights along with other benefits to recover the costs of researching and developing the drug. A tax credit of 50% of the qualified clinical drug testing costs awarded upon drug approval is also possible. Regulatory streamlining and provide special assistance to companies that develop drugs for rare patient populations. In addition to exclusive rights and cost benefits, the FDA will provide protocol assistance, potential decreased wait-time for drug approval, discounts on registration fees, and eligibility for market exclusivity after approval.
Key benefits of ODD:
Hoth recently announcedthat its novelanti-cancer therapeuticexhibited highly positive results in humanized mast cell neoplasm models, representative in vitro and in vivo models for aggressive, mast cell-derived cancers such as mast cell leukemia and mast cell sarcoma. CONTINUED Read the Hoth Therapeutics full press release by going to: https://ir.hoththerapeutics.com/news-releases
In other news and developments of note in the markets this week:
Amgen (NASDAQ: AMGN) recently announced that the U.S. Food and Drug Administration (FDA) has approved Amgen and AstraZeneca'sTezspire (tezepelumab-ekko) for the add-on maintenance treatment of adult and pediatric patients aged 12 years and older with severe asthma.
Tezspirewas approved following a Priority Review by the FDA and based on results from the PATHFINDER clinical trial program. The application included results from the pivotal NAVIGATOR Phase 3 trial in whichTezspiredemonstrated superiority across every primary and key secondary endpoint in patients with severe asthma, compared to placebo, when added to standard therapy.
Longeveron Inc. (NASDAQ: LGVN), a clinical stage biotechnology company developing cellular therapies for chronic aging-related and certain life-threatening conditions, recently announced that the U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation (ODD) for Lomecel-B for the treatment of Hypoplastic Left Heart Syndrome (HLHS), a rare and life-threatening congenital heart defect in infants.
ODD is intended to assist and encourage companies to develop safe and effective therapies for the treatment of rare diseases or conditions. ODD positions Longeveron to be able to potentially leverage a range of financial and regulatory benefits, including government grants for conducting clinical trials, waiver of FDA user fees for the potential submission of a marketing application, and certain tax credits. Receiving ODD may also result in the product receiving seven years market exclusivity upon approval for use in the rare disease or condition for which the product was designated if all of the statutory and regulatory requirements are met.
Bristol Myers Squibb (NYSE: BMY) recently announced thatOrencia(abatacept) was approved by the U.S. Food and Drug Administration (FDA) for the prophylaxis, or prevention, of acute graft versus host disease (aGvHD), in combination with a calcineurin inhibitor (CNI) and methotrexate (MTX), in adults and pediatric patients 2 years of age and older undergoing hematopoietic stem cell transplantation (HSCT) from a matched or 1 allele-mismatched unrelated donor (URD).
"Orenciais the first FDA-approved therapy to prevent acute graft versus host disease following hematopoietic stem cell transplant, a potentially life-threatening complication that can pose a comparatively higher risk to racial and ethnic minority populations in the U.S. due to difficulty finding appropriately matched donors," said Tina Deignan, senior vice president, U.S. Immunology, Bristol Myers Squibb. "With this fourth indication forOrencia,Bristol Myers Squibb draws on its legacy and expertise in both immunology and hematology to deliver an important treatment option for patients in a disease with high unmet need.
Takeda Pharmaceutical Company Limited (NYSE: TAK) announced that the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) has recommended the approval of intravenous (IV) vedolizumab for the treatment of adult patients with moderately to severely active chronic pouchitis, who have undergone proctocolectomy and ileal pouch-anal anastomosis (IPAA) for ulcerative colitis (UC), and have had an inadequate response with or lost response to antibiotic therapy. The CHMP opinion will now be reviewed by the European Commission. If approved, vedolizumab will become the first treatment indicated for active chronic pouchitis across the European Union.
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Systemic Mastocytosis Treatments Gain Hope Due To Increasing Novel Treatment Options - PRNewswire
Jasper Therapeutics to Present Data on JSP191 Conditioning in SCID patients at the 2021 American Society of Hematology Annual Meeting – Yahoo Finance
By daniellenierenberg
JSP191 is well tolerated with no treatment-related adverse events in dose-escalation study
Single-agent conditioning with JSP191 is associated with engraftment, immune reconstitution, and clinical benefit
REDWOOD CITY, Calif., Dec. 08, 2021 (GLOBE NEWSWIRE) -- Jasper Therapeutics, Inc. (NASDAQ: JSPR), a biotechnology company focused on hematopoietic cell transplant therapies, today announced that data on JSP191 showing long-term benefits of hematopoietic stem cells (HSC) engraftment following targeted single-agent JSP191 conditioning in the treatment of severe combined immunodeficiency (SCID) will be presented at the 2021 American Society of Hematology (ASH) Annual Meeting.
The accepted abstract is published and available on the ASH website here.
Title: JSP191 As a Single-Agent Conditioning Regimen Results in Successful Engraftment, Donor Myeloid Chimerism, and Production of Donor Derived Nave Lymphocytes in Patients with Severe Combined Immunodeficiency (SCID)Session: 721. Allogeneic Transplantation: Conditioning Regimens, Engraftment and Acute Toxicities; Novel Conditioning Approaches. Hematology Disease Topics & Pathways:Abstract: 554Date and Time: Sunday, December 12, 2021, 4.45 p.m. ET
Our ongoing study shows JSP191 to be well tolerated with no treatment-related adverse events across multiple patients ranging from 3 months to 38 years old, said Kevin N. Heller, M.D., Executive Vice President, Research and Development. In this study six of nine non-IL2RG patients with prior hematopoietic cell transplant (HCT), dosed in the initial JSP191 dose escalation (0.1, 0.3, 0.6 and 1.0 mg/kg), achieved HSC engraftment, nave donor T lymphocyte production, and demonstrated clinical improvement. As this trial continues to enroll, the 0.6 mg/kg dose will continue to be evaluated as the potential recommended Phase 2 dose (RP2D) based on HSC engraftment, clinical outcomes and an optimal half-life allowing for integration within existing transplant protocols. We believe that with these initial successful clinical findings, we are one step closer, and uniquely positioned to deliver a targeted non-genotoxic conditioning agent to patients with SCID.
Story continues
About Jasper Therapeutics
Jasper Therapeutics is a biotechnology company focused on the development of novel curative therapies based on the biology of the hematopoietic stem cell. The company is advancing two potentially groundbreaking programs. JSP191, an anti-CD117 monoclonal antibody, is in clinical development as a conditioning agent that clears hematopoietic stem cells from bone marrow in patients undergoing hematopoietic cell transplantation. It is designed to enable safer and more effective curative allogeneic hematopoietic cell transplants and gene therapies. In parallel, Jasper Therapeutics is advancing its preclinical mRNA engineered hematopoietic stem cell (eHSC) platform, which is designed to overcome key limitations of allogeneic and autologous gene-edited stem cell grafts. Both innovative programs have the potential to transform the field and expand hematopoietic stem cell therapy cures to a greater number of patients with life-threatening cancers, genetic diseases and autoimmune diseases than is possible today. For more information, please visit us at jaspertherapeutics.com.
Forward-Looking Statements
Certain statements included in this press release that are not historical facts are forward-looking statements for purposes of the safe harbor provisions under the United States Private Securities Litigation Reform Act of 1995. Forward-looking statements are sometimes accompanied by words such as believe, may, will, estimate, continue, anticipate, intend, expect, should, would, plan, predict, potential, seem, seek, future, outlook and similar expressions that predict or indicate future events or trends or that are not statements of historical matters. These forward-looking statements include, but are not limited to, statements the proposed business combination between AMHC and Jasper Therapeutics, the estimated or anticipated future results and benefits of the combined company following the business combination, including Jasper Therapeutics business strategy, expected cash resources of the combined company and the expected uses thereof, current and prospective product candidates, planned clinical trials and preclinical activities and potential product approvals, as well as the potential for market acceptance of any approved products and the related market opportunity. These statements are based on various assumptions, whether or not identified in this press release, and on the current expectations of the respective management teams of Jasper Therapeutics and AMHC and are not predictions of actual performance. These forward-looking statements are provided for illustrative purposes only and are not intended to serve as, and must not be relied on by an investor as, a guarantee, an assurance, a prediction or a definitive statement of fact or probability. Actual events and circumstances are difficult or impossible to predict and will differ from assumptions. Many actual events and circumstances are beyond the control of Jasper Therapeutics and AMHC. These forward-looking statements are subject to a number of risks and uncertainties, including general economic, political and business conditions the outcome of any legal proceedings that may be instituted against the parties regarding the Business Combination; the risk that the potential product candidates that Jasper Therapeutics develops may not progress through clinical development or receive required regulatory approvals within expected timelines or at all; risks relating to uncertainty regarding the regulatory pathway for Jasper Therapeutics product candidates; the risk that clinical trials may not confirm any safety, potency or other product characteristics described or assumed in this press release; the risk that Jasper Therapeutics will be unable to successfully market or gain market acceptance of its product candidates; the risk that Jasper Therapeutics product candidates may not be beneficial to patients or successfully commercialized; the risk that Jasper Therapeutics has overestimated the size of the target patient population, their willingness to try new therapies and the willingness of physicians to prescribe these therapies; the effects of competition on Jasper Therapeutics business; the risk that third parties on which Jasper Therapeutics depends for laboratory, clinical development, manufacturing and other critical services will fail to perform satisfactorily; the risk that Jasper Therapeutics business, operations, clinical development plans and timelines, and supply chain could be adversely affected by the effects of health epidemics, including the ongoing COVID-19 pandemic; the risk that Jasper Therapeutics will be unable to obtain and maintain sufficient intellectual property protection for its investigational products or will infringe the intellectual property protection of others; the potential inability of the parties to successfully or timely consummate the proposed transaction; the risk of failure to realize the anticipated benefits of the proposed transaction and other risks and uncertainties indicated from time to time in AMHCs public filings, including its most recent Annual Report on Form 10-K for the year ended December 31, 2020 and the proxy statement/prospectus relating to the proposed transaction, including those under Risk Factors therein, and in AMHCs other filings with the SEC. If any of these risks materialize or AMHCs and Jasper Therapeutics assumptions prove incorrect, actual results could differ materially from the results implied by these forward-looking statements. There may be additional risks that neither AMHC nor Jasper Therapeutics presently know, or that AMHC or Jasper Therapeutics currently believe are immaterial, that could also cause actual results to differ from those contained in the forward-looking statements. In addition, forward-looking statements reflect AMHCs and Jasper Therapeutics expectations, plans or forecasts of future events and views as of the date of this press release. AMHC and Jasper Therapeutics anticipate that subsequent events and developments will cause AMHCs and Jasper Therapeutics assessments to change. However, while AMHC and Jasper Therapeutics may elect to update these forward-looking statements at some point in the future, AMHC and Jasper Therapeutics specifically disclaim any obligation to do so. These forward-looking statements should not be relied upon as representing AMHCs and Jasper Therapeutics assessments of any date subsequent to the date of this press release. Accordingly, undue reliance should not be placed upon the forward-looking statements.
Contacts:John Mullaly (investors)LifeSci Advisors617-429-3548jmullaly@lifesciadvisors.com
Lily Eng (media)Real Chemistry206-661-8627leng@realchemistry.com
Jeet Mahal (investors)Jasper Therapeutics650-549-1403jmahal@jaspertherapeutics.com
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Jasper Therapeutics to Present Data on JSP191 Conditioning in SCID patients at the 2021 American Society of Hematology Annual Meeting - Yahoo Finance
American Hospital Dubai launches first and only autologous stem cell transplant centre in the UAE – Gulf News
By daniellenierenberg
Image Credit: Supplied
American Hospital Dubai launches the first and only autologous stem cell transplant department in Dubai. It is the first private hospital in the UAE to offer in-housestem cell transplant of patients stem cells, without the need for a donor. The services include laboratory diagnostics, chemotherapy, stem cell mobilisation, collection, storage and re-infusion with individualised care in specialised rooms.
stem cell transplant of patients stem cells, without the need for a donor. The services include laboratory diagnostics, chemotherapy, stem cell mobilisation, collection, storage and re-infusion with individualised care in specialised rooms.
The DHA-licensed stem cell unit is another step forward for American Hospital Dubais comprehensive cancer care programme, established more than 12 years ago.
The unit has a team of European- and US-qualified medical consultants, subspecialists and allied staff, with an international affiliation for multidisciplinary case review and discussions.
The units nurses are highly skilled in Bone Marrow Transplant (BMT) procedures, with experience in apheresis (separating blood components), cellular therapy, and post-transplant care.
The non-surgical transplant procedure is akin to a blood transfusion. It involves stimulating the stem cells, present mainly in bone marrow, by medication to travel out into the blood. This process, called Peripheral Blood Stem Cell collection, is more common in stem cell transplants for cancer treatment than harvesting stem cells directly from the bone marrow for a BMT.
Welcoming the launch, Dr Tarek Dufan, Chief Medical Officer, American Hospital Dubai, said, The stem cell transplant unit is another milestone in American Hospital Dubais commitment to delivering the most advanced healthcare to UAE and the region. Our cutting-edge Cancer Care Department has been a leader in oncology, and the stem cell unit expands our expertise in offering the latest cancer treatments and management.
Dr Maroun El Khoury, Director of Cancer Centre, said, American Hospital Dubais autologous stem cell transplant unit is the only one of its kind in the UAE. We have highly trained staff specialised in stem cell transplant and care management, excellent in-house laboratory services, radiation facilities, and psychological support systems to deliver a complete and compassionate care experience for patients.
The unit, led by Dr Shabeeha K. Rana, Consultant Haematologist and Director of Stem Cell Transplantation and Cellular Therapy at American Hospital Dubai, includes Dr Maroun El Khoury, Director of Cancer Centre; Dr Faraz Khan, Consultant Haematologist/Oncologist; Dr Julieta Zuluaga, Specialist Haematology and Stem Cell Transplantation; Dr Mona Tareen, Pain Management/Palliative Care Consultant; and Dr Melanie Schlatter, Clinical Psychologist.
The unit will treat haematological cancers such as multiple myeloma, lymphoma, certain types of leukaemia and amyloidosis (build-up of a rare protein called amyloid in the body). In addition, it will treat non-haematological conditions such as germ cell tumours and autoimmune diseases such as multiple sclerosis, Crohns, and ulcerative colitis.
The unit provides patients with support groups who have undergone stem cell transplants as an invaluable psychological tool. Every opportunity is made available to patients to provide feedback, ask questions, and inform and educate themselves with written material resources and emotional support for pre-and post-treatment phases.
American Hospital Dubais stem cell transplant unit follows strict selection criteria before accepting patients to ensure the highest adherence to care quality, safety and efficacy.
This content comes from Reach by Gulf News, which is the branded content team of GN Media.
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American Hospital Dubai launches first and only autologous stem cell transplant centre in the UAE - Gulf News
The Anthony Nolan legacy: Three boys given hope of beating deadly blood diseases – The Mirror
By daniellenierenberg
Fifty years ago a boy was born whose brief life would bring hope for thousands of people diagnosed with blood disease.
Anthony Nolans struggle with deadly Wiskott Aldrich Syndrome and his mum Shirleys tireless campaign to save him by finding a suitable bone marrow donor moved the world.
Shirley established the worlds first register of volunteer donors here in the UK.
Tragically, she never did find a suitable match for Anthony and he died when he was seven years old.
But the register became his legacy, recruiting donors around the world. Their bone marrow and stem cells have saved more than 20,000 patients with leukaemia and other blood disorders.
Alan Corby spent six months in the next isolation room to Anthony at Westminster Childrens Hospital. Neither were expected to survive.
Alan said: Much of the time he was the only person I could see. We would talk and play card games like Twist through the glass.
When I was well enough I moved to my local hospital. I went back to see Anthony a few months later, but he had passed away.
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His life may have been short, but it had an incredible impact. Thousands have been given a second chance of life thanks to him and his mum.
On what would have been Anthonys 50th birthday, the Mirror meets three boys given hope of beating deadly blood diseases by his legacy.
Visit anthonynolan.org for more information or to join the donor register.
Georgie McAvoy knows the heartache Shirley Nolan endured searching for a donor to save her son.
Because her little boy Daniel was born with the same rare disease that killed Anthony.
Daniel, two, has Wiskott Aldrich Syndrome which prevents his blood cells from fighting infection and clotting properly. His only hope is a bone marrow transplant to reset his immune system.
A first transplant in June last year failed as Daniel body rejected his donor cells and relapsed.
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He is now preparing to undergo another gruelling course of chemotherapy, followed by a second transplant and will spend Christmas recovering in hospital.
Georgie, 31, said: We have been through so much, but Daniel is still fighting. He has coped with everything that has been thrown at him and he keeps smiling.
We are so grateful for the donor register and everything that Shirley Nolan did it is the reason that Daniel is still alive.
Daniels parents realised something was wrong when he began suffering nosebleeds and they found blood in his nappy when he was three weeks old.
He then developed sepsis and spent 11 days fighting for his life in intensive care.
Georgie and dad Andrew, 38, even asked the hospital chaplain to christen Daniel is his cot as they feared he might not survive.
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Georgie, from Huntingdon in Cambridge, said: The doctors told us he needed a bone marrow transplant to save his life, but that some children didnt make it to transplant.
It was devastating. We didnt know if he would start to walk or go to school. I remember thinking, I need him to be christened in case something happens.
It was emotional. Daniels big sister Holly wore a christening gown made from my wedding dress and Id planned for Daniel to wear it too, but that obviously wasnt possible.
Daniel was eventually diagnosed with Wiskott-Aldrich syndrome, a rare genetic disorder that affects one in every one million boys, in May last year after an unrelated hernia operation.
Neither his parents nor Holly, four, were a suitable match, so their only hope was to find a donor through the stem cell register, which the charity Anthony Nolan managed within two months.
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Daniel was due to undergo the transplant in March last year, but his procedure was postponed after the Covid pandemic began for fear there would be a shortage of doctors, nurses, or beds.
Georgie said: That was really scary. Daniel had been through all his preparation and we were ready to go, then everything blew up before our eyes. We didnt know what would happen.
Daniel continued to deteriorate, picking up more infections until his transplant finally went ahead at Great Ormond Street Hospital at the end of June as doctors could not risk waiting any longer.
He was only allowed one parent with him as he underwent chemotherapy to remove his immune system ahead of the transplant.
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Daniel returned home in August but suffered a drug relating seizure, then graft versus host disease as his body tried to reject the donor cells and spent last Christmas in hospital.
Georgie said: The last two relapses have been particularly difficult. During the last one he began vomiting digested blood. His stomach had to be drained constantly.
At that point they said there were no more options, we had to do another transplant and we needed a different donor as his body had rejected the first.
It will be hard spending another Christmas in hospital, but we feel so lucky to have found another donor to give Daniel a second chance. That wouldnt have happened without Anthony Nolan.
Time is running out for Alife Pinckney to find a lifesaving stem cell donor.
The eight year-old from Exeter relies on weekly blood transfusions to top up his critically low levels of platelets. That has bought Alfie more time, but his condition is getting worse.
His desperate family know his only hope is a transplant, but his mixed British and Chinese heritage makes it harder to find a matching tissue type to prevent his body rejecting the donor cells.
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Alfies mum Lily, said: Its so hard to watch your child in pain and be incapable of helping. Its tearing me apart. Our only hope is to encourage as many people as possible to join the register.
Alfie developed Aplastic Anaemia when he was five years-old. It means his body cannot produce the platelets he needs for his blood to clot properly and he cannot fight infection.
At the time his British-born parents Lily and Charles were living and working in Hong Kong. They returned to the UK just before lockdown last year to be near family and step up Alfies treatment just like Shirley Nolan moved home from Australia to search for a donor for Anthony.
They hoped they had found a donor earlier this year when a woman in Brazil was confirmed as a matching tissue type, but that fell through.
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Since then, Alfie has continued to deteriorate as his body burns through the weekly platelet transfusions and he suffered a terrifying haemorrhage.
Dad Charles said: He had a huge, uncontrollable nosebleed and bleeding from the gums. He was clutching the kitchen bin, vomiting blood, screaming Daddy, help me.
We rushed him into the high dependency unit and I was mopping blood of his arms, face, and torso as several doctors and nurses tried to keep him alive. It was harrowing.
Its so easy to join the register. It only takes three minutes to swab your checks, then you can get on with your life. But that could help to save Mason or another childs life.
Katie Jordan got the devastating news that little Mason had blood cancer on Christmas Eve last year.
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The only cure for his Juvenile Myelomonocytic Leukaemia was a bone marrow transplant but mum Katie was not a good match, nor was anyone on the donor register.
Most children with the disease only survive for 12 months after diagnosis. So Katie, a single mum like Shirley, launched her own campaign to save her son.
Masons Mission raised nearly 54,000 for Anthony Nolan, helping the charity to test the backlog of 25,000 swab samples that built up during the pandemic and add them to the donor register.
Katie, from Stockton-on-Tees said: I was living my worst nightmare. It was heartbreaking to think that Christmas could have been our last together.
I would give my life for Mason, but I wasnt a match. So I did everything I could to find a donor.
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Thankfully Anthony Nolan did find a suitable donor two months later and Mason had a successful stem cell transplant in March this year.
He was rushed back to hospital over the summer after developing blisters all over his body and spent a week fighting for his life in intensive care before they subsided.
But the two year-old made a full recovery and is now looking forward to a happy, healthy Christmas.
Katie said: We were lucky that we found a donor so quickly. When they told us, I couldnt stop crying. I would love to meet his donor one day and thank them."
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The Anthony Nolan legacy: Three boys given hope of beating deadly blood diseases - The Mirror
Mesenchymal Stem Cells Market Growth Drivers 2021, Industry Share-Size, Global Demand, Emerging Trends, Opportunities in Grooming Regions, Key Players…
By daniellenierenberg
Report Ocean presents a new report on Mesenchymal Stem Cells Market size, share, growth, industry trends, and forecast 2026, covering various industry elements and growth trends helpful for predicting the markets future. The global mesenchymal stem cells market size to reach USD 2,518.5 Million by 2026, growing at a CAGR of 7.0% during forecast period, according to a new research report published . The report Mesenchymal Stem Cells Market, [By Source (Bone Marrow, Umbilical Cord Blood, Peripheral Blood, Lung Tissue, Synovial Tissues, Amniotic Fluids, Adipose Tissues); By Application (Injuries, Drug Discovery, Cardiovascular Infraction, Others); By Region]: Market Size & Forecast, 2018 2026 provides an extensive analysis of present market dynamics and predicted future trends. The market was valued at USD 1,335.1 million in 2017. In 2017, the drug discovery application dominated the market, in terms of revenue. North America region is observed to be the leading contributor in the global market revenue in 2017.
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In order to produce a holistic assessment of the market, a variety of factors is considered, including demographics, business cycles, and microeconomic factors specific to the market under study. Mesenchymal Stem Cells Market report 2021 also contains a comprehensive business analysis of the state of the business, which analyzes innovative ways for business growth and describes critical factors such as prime manufacturers, production value, key regions, and growth rate.
The Centers for Medicare and Medicaid Services report that US healthcare expenditures grew by 4.6% to US$ 3.8 trillion in 2019, or US$ 11,582 per person, and accounted for 17.7% of GDP. Also, the federal government accounted for 29.0% of the total health expenditures, followed by households (28.4%). State and local governments accounted for 16.1% of total health care expenditures, while other private revenues accounted for 7.5%.
This study aims to define market sizes and forecast the values for different segments and countries in the coming eight years. The study aims to include qualitative and quantitative perspectives about the industry within the regions and countries covered in the report. The report also outlines the significant factors, such as driving factors and challenges, that will determine the markets future growth.
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These stem cells mainly function for the replacement of damaged cell and tissues. The potential of these cell is to heal the damaged tissue with no pain to the individual. Scientists are majorly focusing on developing new and innovative treatment options for the various chronic diseases like cancer. Additionally, the local governments have also taken various steps for promoting the use of these stem cells.
The significant aspects that are increasing the development in market for mesenchymal stem cells consist of enhancing need for these stem cells as an efficient therapy option for knee replacement. Raising senior populace throughout the world, as well as increasing frequency of numerous persistent conditions consisting of cancer cells, autoimmune illness, bone and cartilage diseases are elements anticipated to enhance the market development throughout the forecast period. The mesenchymal stem cells market is obtaining favorable assistance by the reliable federal government policies, as well as funding for R&D activities which is anticipated to influence the market growth over coming years. According to the reports released by world health organization (WHO), by 2050 individuals aged over 60 will certainly make up greater than 20% of the globes population. Of that 20%, a traditional quote of 15% is estimated to have symptomatic OA, as well as one-third of these individuals are expected to be influenced by extreme specials needs. Taking into consideration all these aspects, the market for mesenchymal stem cells will certainly witness a substantial development in the future.
Increasing demand for better healthcare facilities, rising geriatric population across the globe, and continuous research and development activities in this area by the key players is expected to have a positive impact on the growth of Mesenchymal Stem Cells market. North America generated the highest revenue in 2017, and is expected to be the leading region globally during the forecast period. The Asia Pacific market is also expected to witness significant market growth in coming years. Developing healthcare infrastructure among countries such as China, India in this region is observed to be the major factor promoting the growth of this market during the forecast period.
The major key players operating in the industry are Cell Applications, Inc., Cyagen Biosciences Inc. Axol Bioscience Ltd., Cytori Therapeutics Inc., Stem cell technologies Inc., Celprogen, Inc. BrainStorm Cell Therapeutics, Stemedica Cell Technologies, Inc. These companies launch new products and undertake strategic collaboration and partnerships with other companies in this market to expand presence and to meet the increasing needs and requirements of consumers.
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Polaris Market Research has segmented the global mesenchymal stem cells market on the basis of source type, application and region:
Mesenchymal Stem Cells Source Type Outlook (Revenue, USD Million, 2015 2026)
Bone Marrow
Umbilical Cord Blood
Peripheral Blood
Lung Tissue
Synovial Tissues
Amniotic Fluids
Adipose Tissues
Mesenchymal Stem Cells Application Outlook (Revenue, USD Million, 2015 2026)
Injuries
Drug Discovery
Cardiovascular Infraction
Others
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Mesenchymal Stem Cells Regional Outlook (Revenue, USD Million, 2015 2026)
North America
U.S.
Canada
Europe
Germany
UK
France
Italy
Spain
Russia
Rest of Europe
Asia-Pacific
China
India
Japan
Singapore
Malaysia
Australia
Rest of Asia-Pacific
Latin America
Mexico
Brazil
Argentina
Rest of LATAM
Middle East & Africa
What are the aspects of this report that relate to regional analysis?
The reports geographical regions include North America, Europe, Asia Pacific, Latin America, the Middle East, and Africa.
The report provides a comprehensive analysis of market trends, including information on usage and consumption at the regional level.
Reports on the market include the growth rates of each region, which includes their countries, over the coming years.
How are the key players in the market assessed?
This report provides a comprehensive analysis of leading competitors in the market.
The report includes information about the key vendors in the market.
The report provides a complete overview of each company, including its profile, revenue generation, cost of goods, and products manufactured.
The report presents the facts and figures about market competitors, alongside the viewpoints of leading market players.
A market report includes details on recent market developments, mergers, and acquisitions involving the key players mentioned.
What is the key information extracted from the report?
Extensive information on factors estimated to affect the Market growth and market share during the forecast period is presented in the report.The report offers the present scenario and future growth prospects Market in various geographical regions.The competitive landscape analysis on the market as well as the qualitative and quantitative information is delivered.The SWOT analysis is conducted along with Porters Five Force analysis.The in-depth analysis provides an insight into the Market, underlining the growth rate and opportunities offered in the business.
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Here’s Why Longeveron Is Surging Today – Motley Fool
By daniellenierenberg
What happened
Shares of Longeveron (NASDAQ:LGVN), a clinical-stage biotechnology company headquartered in Miami, are soaring in response to good news from the U.S. Food and Drug Administration (FDA). Investors excited about an orphan drug designation for the company's stem cell treatment pushed the stock 36.7% higher as of 10:37 a.m. ET on Friday.
Longeveron is developing bone marrow-derived mesenchymal stromal cells for the treatment of a variety of age-related disorders including Alzheimer's disease. Today, the FDA granted the company's lead candidate, Lomecel-B, an orphan designation for the treatment of a rare condition called hypoplastic left heart syndrome.
Image source: Getty Images.
Generally, orphan drug designations aren't something to get excited about. The FDA hands them out like candy to just about anyone that intends to develop a drug for underserved patients with a rare condition.
If approved to treat hypoplastic left heart syndrome, this orphan drug designation can begin assisting Longeveron with a handful of useful benefits. The most important one is seven years of market exclusivity.
There isn't much of a difference between the bone marrow-derived stem cells in Lomecel-B and the stem cells biomedical scientists have been researching for decades. They've been renamed medical signaling cells in recent years, but evidence of a significant benefit for specific indications remains elusive.
Longeveron is now a good stock to buy, and betting against it could prove disastrous, too. In the first quarter of 2022, Longeveron intends to begin clinical trials with Lomecel-B as a treatment for Alzheimer's disease.
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Here's Why Longeveron Is Surging Today - Motley Fool
Sanford Health opens study of cell injections for arthritis – Sanford Health News
By daniellenierenberg
Sanford Health is continuing to explore the potential of orthobiologics and regenerative medicine with a study that may advance treatment for those living with osteoarthritis.
This trial known as ENDURE, which is run in full compliance with the FDA, will examine the safety and effectiveness of adult, adipose (fat)-derived stem cells.
Im really excited to get the trial started, said Donella Herman, M.D., a specialist in sports medicine at Sanford Health. Im even more excited about a year from now because I feel like were just going to learn so much about how effective this treatment is and what it is effective for.
Learn more: Orthopedics regenerative medicine at Sanford Health
Essentially, orthobiologics and regenerative medicine tap into the bodys natural healing ability.
Orthobiologics, a part of regenerative medicine, refers to the use of biological substances found in the body to help treat muscle and bone issues.
This may include injuries to muscles, tendons, and ligaments and as the focus of this trial more chronic conditions such as osteoarthritis.
When used properly by qualified providers, cell therapies are proving to be effective treatment options.
Our goals are to collect data for the different branches of orthobiologics, Dr. Herman said. Do people with knee osteoarthritis respond better with PRP (platelet rich plasma) or bone-marrow derived cells or fat-derived stem cells? Which works best? Which works for the longest time? Thats the kind of information were looking for. Its that kind of ratio that we just dont have right now.
One of the obstacles in regenerative medicine at this point, Dr. Herman said, is that its often being marketed by people with dubious clinical qualifications. They use underdeveloped research and make unfounded claims.
Its become a little bit of a wild, wild west with stem cells and PRP, Dr. Herman said. There are pop-up shops all over the place. Its one thing to have access to these things and allow access to people, but if youre not doing that in a responsible way and using that opportunity to build on a knowledge base, its kind of smoke and mirrors.
In sharp contrast to that scenario, this trial aims to add to the bank of knowledge in regenerative medicine.
We know we can tell people what were putting in their knee or their hip or their shoulder because were testing, Dr. Herman said. A lot of other places theyre just injecting it. They dont know if the stem cells are viable or not.
A typical candidate for the study, as described by Dr. Herman, might have osteoarthritis in their wrist and would not want to sacrifice a loss of motion that could come with surgery. They also would likely have exhausted the steroid injections treatment options that offer temporary relief.
We think of regenerative medicine as a possible alternative therapy that may help people put off surgery, Dr. Herman said. I dont ever tell people it means you will never need surgery but what it does is hopefully buy a little time until youre ready for that. There are lots of times where well do steroid injections until those fail and then they have to get a new joint. Were hoping for this to give us a bridge.
Once a patient is approved to be part of the ENDURE study, the procedure includes:
Follow-ups are then completed in person and over the phone.
Qualified candidates must be 18 or older and:
The cost associated with the study is not currently covered by insurance. By enrolling in the ENDURE study, however, participants may be playing a role in advancing regenerative cell research that could have the potential to improve care for future generations.
Those interested in hearing more about possible eligibility in the study can call (605) 328-3700.
Posted In Innovations, Orthopedics, Research, Specialty Care, Sports Medicine
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Sanford Health opens study of cell injections for arthritis - Sanford Health News
Representation learning of RNA velocity reveals robust cell transitions – pnas.org
By daniellenierenberg
Significance
The recently introduced RNA velocity methods, by leveraging the intrinsic RNA splicing process, have shown their unique capability of identifying the directionality of the cell differentiation trajectory. However, due to the minimal amount of unspliced RNA contents, the estimation of RNA velocity suffers from high noise and may result in less reliable trajectories. Here, we present Velocity Autoencoder (VeloAE), a tailored autoencoder to denoise RNA velocity for more accurate quantification of cell transitions. Through various biological systems, we demonstrate its effectiveness for correcting the inferred trajectory and its interpretability for linking the learned dimensions to underlying biological processes.
RNA velocity is a promising technique for quantifying cellular transitions from single-cell transcriptome experiments and revealing transient cellular dynamics among a heterogeneous cell population. However, the cell transitions estimated from high-dimensional RNA velocity are often unstable or inaccurate, partly due to the high technical noise and less informative projection. Here, we present Velocity Autoencoder (VeloAE), a tailored representation learning method, to learn a low-dimensional representation of RNA velocity on which cellular transitions can be robustly estimated. On various experimental datasets, we show that VeloAE can both accurately identify stimulation dynamics in time-series designs and effectively capture expected cellular differentiation in different biological systems. VeloAE, therefore, enhances the usefulness of RNA velocity for studying a wide range of biological processes.
Author contributions: C.Q. and Y.H. designed research; C.Q. performed research; C.Q. contributed new reagents/analytic tools; C.Q. analyzed data; and C.Q. and Y.H. wrote the paper.
The authors declare no competing interest.
This article is a PNAS Direct Submission.
This article contains supporting information online at https://www.pnas.org/lookup/suppl/doi:10.1073/pnas.2105859118/-/DCSupplemental.
VeloAE is an open-source Python package available at GitHub, https://github.com/qiaochen/VeloAE. All the analysis notebooks for reproducing the results are also available in this repository. Previously published data were used for this work (8, 10, 1925).
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Representation learning of RNA velocity reveals robust cell transitions - pnas.org
Former England and Everton footballer Gary Stevens’ son tragically dies aged four – Mirror.co.uk
By daniellenierenberg
Jack Stevens was diagnosed with juvenile myelomonocytic leukaemia and underwent stem cell treatment but has sadly lost his battle as his father's former clubs pay tribute
The young son of former England footballer Gary Stevens has died tragically aged four.
Stevens son Jack was diagnosed with a rare form of blood cancer.
Just a year ago his family were hopeful of a recovery after Jack underwent a stem cell transplant following his diagnosis with juvenile myelomonocytic leukaemia.
The stem cells Jack received from older brother Oliver had engrafted, meaning they had entered Jacks bone marrow, enabling him to produce his own white blood cells.
The transplant procedure was brought forward after Jack responded to two rounds of preparatory chemotherapy, negating an original plan for five cycles of treatment.
He was discharged to a nearby apartment and during the week lived with mum Louise.
Stevens a brilliant right-back in Howard Kendalls magical mid-1980s Everton team and the couples other sons, Oliver and Josh, would join Jack and Louise at weekends.
This morning Everton said: "Everyone at Everton is deeply saddened to learn that Gary Stevens four-year-old son, Jack, has passed away following his courageous battle with leukaemia.
"Our thoughts are with Gary and his family at this incredibly sad time."
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Stevens, 59, began his football career with Everton, representing the Toffees for six seasons and making over 200 appearances between 1982-1988.
He then transferred to Glasgow Rangers where he played a part in their treble-winning season in 1993.
After another six-year stint, he returned to Merseyside to finish his career plying his trade for Tranmere Rovers.
Stevens earned 46 caps for England during his playing days before moving into physiotherapy after his retirement.
Tranmere shared Everton's post and issued their own touching message, which read: "The thoughts of everyone at Tranmere Rovers are with Gary Stevens and his family at this sad time."
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Former England and Everton footballer Gary Stevens' son tragically dies aged four - Mirror.co.uk