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Archive for the ‘Bone Marrow Stem Cells’ Category

Types of leukemia: Prevalence, treatment options, and prognosis – Medical News Today

Leukemia is a type of cancer that affects the blood and bone marrow, where blood cells are formed. All types of leukemia cause rapid, uncontrolled growth of abnormal bone marrow and blood cells.

The main differences between the types include how fast the disease progresses and the types of cells it affects.

There are four main types of leukemia, which we describe in detail below:

Lymphocytic leukemia affects the lymphocytes, a type of white blood cell. Myeloid leukemia can affect the white blood cells, red blood cells, and platelets.

According to the National Cancer Institute, roughly 1.5% of people in the United States will receive a leukemia diagnosis at some point.

In this article, explore the four main types, their symptoms, the treatment options available, and the outlook.

The full name of this type of cancer is acute lymphocytic leukemia, and acute means that it grows quickly. Lymphocytic means that it forms in underdeveloped white blood cells called lymphocytes.

The disease starts in the bone marrow, which produces stem cells that develop into red and white blood cells and platelets.

In a healthy person, the bone marrow does not release these cells until they are fully developed. In someone with ALL, the bone marrow releases large quantities of underdeveloped white blood cells.

There are several subtypes of ALL, and the subtype may influence the best course of treatment and the prognosis.

One subtype is B-cell ALL. This begins in the B lymphocytes, and it is the most common form of ALL in children.

Another subtype is T-cell ALL. It can cause the thymus, a small organ at the front of the windpipe, to become enlarged, which can lead to breathing difficulties.

Overall, because ALL progresses quickly, swift medical intervention is key.

As research from 2020 acknowledges, healthcare providers still do not know what causes ALL. It may occur due to genetic factors or exposure to:

Although genetic factors may play a role, ALL is not a familial disease.

Learn more about ALL here.

ALL is the most common form of leukemia in children.

The risk of developing it is highest in children under 5 years old. The prevalence slowly rises again in adults over 50.

ALL symptoms can be nonspecific difficult to distinguish from those of other illnesses.

They may include:

In a person with AML, the bone marrow makes abnormal versions of platelets, red blood cells, and white blood cells called myeloblasts.

The full name of this disease is acute myeloid leukemia, and acute refers to the fact that it is fast-growing.

It forms in one of the following types of bone marrow cell:

Doctors classify AML by subtype, depending on:

AML can be difficult to treat and requires prompt medical attention.

Learn more about AML here.

The most common risk factor is myelodysplastic syndrome, a form of blood cancer that keeps the body from producing enough healthy blood cells.

Other factors that increase the risk of developing AML include:

Most people who develop AML are over 45. It is one of the most common types of leukemia in adults, though it is still rare, compared with other cancers.

It is also the second most common form of leukemia in children.

Symptoms of AML can vary and may include:

CLL is the most common form of leukemia among adults in the U.S. and other Western countries.

There are two types. One progresses slowly, and it causes the body to have high levels of characteristic lymphocytes, but only slightly low levels of healthy red blood cells, platelets, and neutrophils.

The other type progresses more quickly and causes a significant reduction in levels of all healthy blood cells.

In someone with CLL, the lymphocytes often look fully formed but are less able to fight infection than healthy white blood cells. The lymphocytes tend to build up very slowly, so a person might have CLL for a long time before experiencing symptoms.

Learn more about CLL here.

Genetic factors are the most likely cause. Others might include:

CLL is rare in children. It typically develops in adults aged 70 or over. However, it can affect people as young as 30.

CLL typically causes no early symptoms. When symptoms are present, they may include:

Also, 5090% of people with CLL have swollen lymph nodes.

CML is a slow-growing type of leukemia that develops in the bone marrow.

The full name of CML is chronic myeloid leukemia. As the American Cancer Society explain, a genetic change takes place in the early forms of the myeloid cells, and this eventually results in CML cells.

These leukemia cells then grow, divide, and enter the blood.

CML occurs due to a rearrangement of genetic material between the chromosomes 9 and 22.

This rearrangement fuses a part of the ABL1 gene from chromosome 9 with the BCR gene from chromosome 22, called the Philadelphia chromosome. The result of this fusion is called BCR-ABL1.

BCR-ABL1 produces a protein that promotes cell division and stops apoptosis, the process of cell death, which typically removes unneeded or damaged cells.

The cells keep dividing and do not self-destruct, resulting in an overproduction of abnormal cells and a lack of healthy blood cells.

This occurs during the persons lifetime and is not inherited.

CML typically affects adults. People aged 65 and older make up almost half of those who receive a CML diagnosis.

The symptoms of CML are unclear, but they may include:

The symptoms may vary, depending on the type of leukemia. Overall, a person should get in touch with a doctor if they experience:

Learn more about the symptoms of leukemia here.

Treatment for ALL typically involves three basic phases: induction, consolidation, and maintenance. We describe these in detail below.

Treatment for AML involves the first two phases. The induction phase may include treatment with the chemotherapy drugs cytarabine (Cytosar-U) and daunorubicin (Cerubidine) or idarubicin (Idamycin). The doctor may also recommend targeted drugs.

The goal of this phase is to kill the leukemia cells, causing the cancer to go into remission, using chemotherapy.

The doctor may recommend:

People having chemotherapy may need to see their doctors frequently and spend time in the hospital, due to the risk of serious infections and complications.

This phase of the treatment lasts for about 1 month.

Even if the treatment so far has led to remission, cancer cells may be hiding in the body, so more treatment is necessary.

The consolidation phase may involve taking high doses of chemotherapy. A doctor may also recommend targeted drugs or stem cell transplants.

This phase, consisting of ongoing chemotherapy treatments, usually lasts for 2 years.

Since CLL tends to progress slowly, and its treatment can have unpleasant side effects, some people with this condition go through a phase of watchful waiting before starting the treatment.

For a person with CML, the focus is often on providing the right treatment for the phase of the illness. To do this, a doctor considers how quickly the leukemia cells are building up and the extent of the symptoms. Stem cell transplants can be effective, but further treatment is necessary.

Overall, the initial treatment tends to include monoclonal antibodies, targeted drugs, and chemotherapy.

If the only concern is an enlarged spleen or swollen lymph nodes, the person may receive radiation or surgery.

If there are high numbers of CLL cells, the doctor may suggest leukapheresis, a treatment that lowers the persons blood count. This is only effective for a short time, but it allows the chemotherapy to start working.

For people with high-risk disease, doctors may recommend stem cell transplants.

A persons prognosis depends on the type of leukemia.

Learn more about survival rates for people with leukemia here.

About 8090% of adults with ALL experience complete remission for a while during treatment. And with treatment, most children recover from the disease.

Relapses are common in adults, so the overall cure rate is 40%. However, factors specific to each person play a role.

The older a person is when they receive an AML diagnosis, the more difficult it is to treat.

More than 25% of adults who achieve remission live for 3 years or more after treatment for AML.

A person may live for a long time with CLL.

Treatments can help keep the symptoms under control and prevent the disease from spreading. However, there is no cure.

Stem cell transplants can cure CML. However, this treatment is very invasive and is not suitable for most people with CML.

The United Kingdoms National Health Service estimate that 70% of males and 75% of females live for at least 5 years after receiving a CML diagnosis.

The earlier a person receives the diagnosis, the better their outlook.

Leukemia is a type of cancer that affects the blood and bone marrow. It can affect people of all ages.

There are four main types of leukemia. They differ based on how quickly they progress and the types of cells they affect.

Treatments for all types of leukemia continue to improve, helping people live longer and more fully with this condition.

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Types of leukemia: Prevalence, treatment options, and prognosis - Medical News Today

Be The Match encourages people of color to join bone marrow registry – KING5.com

Black patients in need of bone marrow or blood stem cell treatments have a decreased chance of matching with a donor. The Seattle branch hopes to change that.

Seattles Be The Match Collection Center opened up less than a year ago and is celebrating its 100th blood cell donation with an important message: More bone marrow donors of color are needed.

The nonprofit donation center is a part of the National Marrow Donor Program and increases the capacity to collect blood cells in the Pacific Northwest. Seattles Clinical Manager Hannah Erskine said this month is an important time to focus on the donation gap.

In the midst of Black History Month, its important to note that we frankly dont have enough Black and African American donors on the registry, said Erskin.

Only 4% of approximately 22 million donors on the registry are African American, lowering the chances that a Black patient can find a bone marrow donor who is a genetic match.

According to Be The Match data, the likelihood of finding a matched adult donor is only around 23% for an African American or Black patient, versus a 77% match rate for a white patient.

These matched bone marrow or blood stem cell transplants can help cure blood cancers like leukemia and lymphoma, as well as other blood conditions, such as sickle cell disease. Be The Match has coordinated more than 100,000 transplants.

Erskine said registering is a simple mouth swab that will be mailed to potential donors. They will be contacted if they are a match with a patient.

Being a matching blood stem cell donor can potentially save a life. The first step in changing the trend is to join the registry at http://www.bethematch.org.

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Be The Match encourages people of color to join bone marrow registry - KING5.com

Cord Blood Banking Services Market projected to expand at a CAGR of 10.9% from 2019 to 2027 KSU | The Sentinel Newspaper – KSU | The Sentinel…

Transparency Market Research (TMR) has published a new report titled, Cord Blood Banking Services Market Global Industry Analysis, Size, Share, Growth, Trends, and Forecast, 20192027. According to the report, the globalcord blood banking services marketwas valued atUS$ 25.8 Mnin2018and is projected to expand at a CAGR of10.9%from2019to2027.

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Cord Blood Banking Services Market projected to expand at a CAGR of 10.9% from 2019 to 2027 KSU | The Sentinel Newspaper - KSU | The Sentinel...

Jasper Therapeutics Announces Launch of New Clinical Trial with National Heart, Lung, and Blood Institute to Evaluate JSP191 in Sickle Cell Disease -…

REDWOOD CITY, Calif.--(BUSINESS WIRE)--Jasper Therapeutics, Inc., a biotechnology company focused on hematopoietic cell transplant therapies, today announced the launch of a Phase 1/2 clinical trial to evaluate JSP191, Jaspers first-in-class anti-CD117 monoclonal antibody, as a targeted, non-toxic conditioning regimen prior to allogeneic transplant for sickle cell disease (SCD). Jasper Therapeutics and the National Heart, Lung, and Blood Institute (NHLBI) have entered into a clinical trial agreement in which NHLBI will serve as the Investigational New Drug (IND) sponsor for this study.

SCD is a lifelong inherited blood disorder that affects hemoglobin, a protein in red blood cells that delivers oxygen to tissues and organs throughout the body. Approximately 300,000 infants are born with SCD annually worldwide, and the number of cases is expected to significantly increase. Currently, hematopoietic stem cell transplantation (HSCT) is the only cure available for SCD.

"This clinical trial agreement with the NHLBI expands the development of JSP191 for transplant conditioning and could bring curative transplants to more patients in need," said Kevin N. Heller, M.D., Executive Vice President, Research and Development, of Jasper Therapeutics. "We look forward to collaborating with the NHLBI and learning more about the potential for JSP191 in patients living with sickle cell disease."

About JSP191

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

Preclinical studies have shown that JSP191, as a single agent, safely depletes normal and diseased hematopoietic stem cells, including in animal models of severe combined immunodeficiency (SCID), myelodysplastic syndromes (MDS), and sickle cell disease (SCD). Treatment with JSP191 creates the space needed for transplanted normal donor or gene-corrected hematopoietic stem cells to successfully engraft in the host bone marrow. To date, JSP191 has been evaluated in more than 90 healthy volunteers and patients.

JSP191 is currently being evaluated in two separate Jasper Therapeutics-sponsored clinical studies in hematopoietic cell transplant. The first clinical study is evaluating JSP191 as a sole conditioning agent in a Phase 1/2 dose-escalation and expansion trial to achieve donor stem cell engraftment in patients undergoing hematopoietic cell transplant for SCID. Blood stem cell transplantation offers the only potentially curative therapy for SCID. JSP191 is also being evaluated in combination with another conditioning regimen in a Phase 1 study in patients with MDS or acute myeloid leukemia (AML) who are receiving hematopoietic cell transplant. For more information about the design of these clinical trials, visit http://www.clinicaltrials.gov (NCT02963064 and NCT04429191).

Additional studies are planned to advance JSP191 as a conditioning agent for patients with other rare and ultra-rare monogenic disorders and autoimmune diseases.

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

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Jasper Therapeutics Announces Launch of New Clinical Trial with National Heart, Lung, and Blood Institute to Evaluate JSP191 in Sickle Cell Disease -...

Apamistamab Conditioning Treatment Induces High Rates of HCT Success in AML – OncLive

Apamistamab (Iomab-B) conditioning treatment with targeted radioimmunotherapy to the bone marrow resulted in high rates of successful allogeneic hematopoietic stem cell transplants in patients with active, relapsed, or refractory acute myeloid leukemia (AML), according to interim results from the phase 3 SIERRA trial, which were presented virtually at the 2021 Transplant and Cellular Therapies Meetings.1

In these patients with relapsed or refractory AML, we observed high rates of allogeneic stem cell transplant with curative intent [in] 88% of patients on the Iomab-B arm, 18% of patients who were randomized to the conventional care arm achieved complete remission and received standard of care allo-transplant, and an overall rate of 79% of allo-transplant in all enrolled patients, Boglarka Gyurkocza, MD, said in a virtual presentation.

Investigators sought to prove with this study that targeted radiation to the marrow with apamistamab, a radioactive iodine (131I)labeled anti-CD45 antibody, could enable the successful engraftment of patients despite active disease in the marrow. Safety and robust efficacy had previously been demonstrated with the agent in 271 patients treated in 9 different phase 1 and 2 clinical trials.

The SIERRA trial is looking to enroll 150 patients, and the trial is already over 75% enrolled. Recently, an independent data monitoring committee recommended that the trial continue to the planned full enrollment based on a positive pre-planned ad-hoc analysis.2

In the study, patients with active, relapsed, refractory AML are randomized 1:1 to receive either apamistamab conditioning therapy and allogeneic HCT or conventional care. In the control arm, patients who do not achieve a complete remission (CR) by day 42 are allowed to cross over to receive Iomab-B, and those who do have a CR undergo HCT or receive standard-of-care therapy of the physicians choice.

Durable CR (dCR) rate is the primary end point of the study, characterized as complete response at 6 months after initial CR, and the secondary end point is overall survival (OS) rate at 1 year.

Patients are eligible for enrollment if they have marrow blast count 5% or the presence of peripheral blasts, age 55 years, a Karnofsky score 70, and related/unrelated donor matching at human leukocyte antigen (HLA)-A, HLA-B, HLA-C, and DRB-1. Active, relapsed, or refractory AML was defined for the sake of the trial as primary induction failure after 2 cycles of therapy including chemotherapy or 2 cycles of venetoclax (Venclexta) with a hypomethylating agent or low-dose cytarabine, first early relapse after first CR of less than 6 months, relapse refractory to salvage chemotherapy regimen, or second or subsequent relapse. Secondary or treatment-related AML was also allowed.

In the SIERRA trial, patient-specific dosimetry was used to generate an individualized therapeutic dose to target marrow and spare non-hematopoietic organs. Patients in the investigational arm received a dosimetric dose of apamistamab ( 20 mCi) approximately 19 days prior to HCT followed by a therapeutic dose of apamistamab, which is individually calculated for each patient based on an upper limit of 24 Gy to the liver. After, patients remain on radiation isolation for several days before receiving fludarabine conditioning therapy (30 mg/m2/day for 3 days) and finally low-dose total body irradiation (200 cGy) prior to HCT.

Among the first 75% of enrolled patients (n = 113), patients in the apamistamab arm (n = 56) had a median age of 63 years (range, 55-77), 35% had intermediate risk and 61% had adverse risk, the median

percent of marrow blasts at baseline was 29% (range, 4%-95%), and had received a median of 3 prior treatment regimens (range, 1-7). At randomization, 56% were in primary induction failure, 16% were in first early relapse, 15% had relapsed or refractory disease, and 13% were in their second or later relapse.

In the conventional care arm, the median age was 65 years (range, 55-77), 32% had intermediate risk and 63% had adverse risk, median marrow blasts was 20% (range, 5%-97%), and had received a median of 3 prior regimens (range, 1-6). At randomization, 49% were in primary induction failure, 21% were in first early relapse, 21% had relapsed or refractory disease, and 8.8% were in their second or later relapse. Patients who crossed over to receive apamistamab (n = 30) had similar baseline characteristics.

Forty-nine patients in the apamistamab-randomized arm were able to go on and undergo allogeneic HCT compared with 10 patients in the conventional care arm. In the investigational arm, a median of 646 mCi (range, 3541027) of apamistamab was infused at a dose of 14.7 Gy (range, 4.6-32) to the marrow. The median infused CD34-positive cell count was 5.6 x 106/Kg (range, 1.8-208). Forty-five patients received peripheral blood stem cells (PBSCs), 3 received marrow grafts, 17 had related donors, and 31 had unrelated.

Individualized therapy of Iomab-B provided myeloablative doses of radiation to the marrow, Gyurkocza, a medical oncologist at Memorial Sloan Kettering Cancer Center, commented.

These patients had a median of 30 days (range, 23-60) to HCT after randomization and 14 days (range, 9-22) to neutrophil engraftment, with no graft failure reported. Patients also had 18 days (range, 4-39) until platelet engraftment.

We also observed 100% neutrophil and platelet engraftment in patients who received Iomab-B conditioning, despite a heavy leukemia burden, Gyurkocza said.

In patients in the conventional arm who went on to HCT, conditioning regimens for HCT consisted of fludarabine/melphalan in 2, fludarabine/melphalan/total body irradiation in 1, busulfan/fludarabine in 1, cyclophosphamide/fludarabine/total body irradiation in 2, and 4 had no data on conditioning regimens available. Eight of these patients had PBSCs, 2 had marrow, 3 had related donors, 6 had unrelated, and 1 was unreported.

Median days to HCT was 67 (range, 52-104) with 17 days (range, 13-83) to neutrophil engraftment and 22 days (range, 8-35) to platelet engraftment. There was 1 graft failure.

Among the patients who crossed over to receive apamistamab before HCT, the median infused dose was 592 mCi (range, 313-1013) with 15.5 Gy (range, 6.3-42) to the marrow. The median infused CD34-positive cell count was 5.1 x 106/Kg (range, 1.8-16.1). Twenty-eight patients had PBSCs, 2 had marrow, 10 had related donors, and 20 had unrelated.

Patients had a median of 62 days (range, 36-100) to HCT, 14 days (range, 10-37) to neutrophil engraftment, and 19 days (range, 1-38) to platelet engraftment. No graft failure was reported in this group.

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Apamistamab Conditioning Treatment Induces High Rates of HCT Success in AML - OncLive

Novartis, Gates Foundation pursue a simpler gene therapy for sickle cell – STAT

Novartis and the Bill and Melinda Gates Foundation are joining forces to discover and develop a gene therapy to cure sickle cell disease with a one-step, one-time treatment that is affordable and simple enough to treat patients anywhere in the world, especially in sub-Saharan Africa where resources may be scarce but disease prevalence is high.

The three-year collaboration, announced Wednesday, has initial funding of $7.28 million.

Current gene therapy approaches being developed for sickle cell disease are complex, enormously expensive, and bespoke, crafting treatments for individual patients one at a time. The collaboration aims to instead create an off-the-shelf treatment that bypasses many of the steps of current approaches, in which cells are removed and processed outside the body before being returned to patients.

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Sickle cells cause is understood. The people it affects are known. But its cure has been elusive, Jay Bradner, president of the Novartis Institutes for BioMedical Research, told STAT.

We understand perfectly the disease pathway and the patient, but we dont know what it would take to have a single-administration, in vivo gene therapy for sickle cell disease that you could deploy in a low-resource setting with the requisite safety and data to support its use, he said. Im a hematologist and can assure you that in my experience in the clinic, it was extremely frustrating to understand a disease so perfectly but have so little to offer.

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Sickle cell disease is a life-threatening inherited blood disorder that affects millions around the world, with about 80% of affected people in sub-Saharan Africa and more than 100,000 in the U.S. The mutation that causes the disease emerged in Africa, where it protects against malaria. While most patients with sickle cell share African ancestry, those with ancestry from South America, Central America, and India, as well as Italy and Turkey, can also have the hereditary disease.

The genetic mutation does its damage by changing the structure of hemoglobin, hampering the ability of red blood cells to carry oxygen and damaging blood vessels when the misshapen cells get stuck and block blood flow. Patients frequently suffer painful crises that can be fatal if not promptly treated with fluids, medication, and oxygen. Longer term, organs starved of oxygen eventually give out. In the U.S., that pain and suffering is amplified when systemic and individual instances of racism deny Black people the care they need.

Delivering gene therapy for other diseases has been costly and difficult even in the best financed, most sophisticated medical settings. Challenges include removing patients cells so they can be altered in a lab, manufacturing the new cells in high volume, reinfusing them, and managing sometimes severe responses to the corrected cells. Patients also are given chemotherapy to clear space in their bone marrow for the new cells.

Ideally, many of those steps could be skipped if there were an off-the-shelf gene therapy. That means, among other challenges, inventing a way to eliminate the step where each patients cells are manipulated outside the body and given back the in vivo part of the plan to correct the genetic mutation.

Thats not the only obstacle. For a sickle cell therapy to be successful, Bradner said, it must be delivered only to its targets, which are blood stem cells. The genetic material carrying corrected DNA must be safely transferred so it does not become randomly inserted into the genome and create the risk of cancer, a possibility that halted a Bluebird Bio clinical trial on Tuesday. The payload itself mustnt cause such problems as the cytokine storm of immune overreaction. And the intended response has to be both durable and corrective.

In a way, the gene delivery is the easy part because we know that expressing a normal hemoglobin, correcting the mutated hemoglobin, or reengineering the switches that once turned off normal fetal hemoglobin to turn it back on, all can work, Bradner said. The payload is less a concern to me than the safe, specific, and durable delivery of that payload.

For each of these four challenges delivery, gene transfer, tolerability, durability there could be a bespoke technical solution, Bradner said. The goal is to create an ensemble form of gene therapy.

Novartis has an existing sickle-cell project using CRISPR with the genome-editing company Intellia, now in early human trials, whose lessons may inform this new project. CRISPR may not be the method used; all choices are still on the table, Bradner said.

Vertex Pharmaceuticals has seen encouraging early signs with its candidate therapy developed with CRISPR Therapeutics. Other companies, including Beam Therapeutics, have also embarked on gene therapy development.

The Novartis-Gates collaboration is different in its ambition to create a cure that does not rely on an expensive, complicated framework. Novartis has worked with the Gates Foundation on making malaria treatment accessible in Africa. And in October 2019, the Gates Foundation and the National Institutes of Health said together they would invest at least $200 million over the next four years to develop gene-based cures for sickle cell disease and HIV that would be affordable and available in the resource-poor countries hit hardest by the two diseases, particularly in Africa.

Gene therapies might help end the threat of diseases like sickle cell, but only if we can make them far more affordable and practical for low-resource settings, Trevor Mundel, president of global health at the Gates Foundation, said in a statement about the Novartis collaboration. Its about treating the needs of people in lower-income countries as a driver of scientific and medical progress, not an afterthought.

Asked which is the harder problem to solve: one-time, in vivo gene therapy, or making it accessible around the world, David Williams, chief of hematology/oncology at Boston Childrens Hospital, said: Both are going to be difficult to solve. The first will likely occur before the therapy is practically accessible to the large number of patients suffering the disease around the world.

Williams is also working with the Gates Foundation, as well as the Koch Institute for Integrative Cancer Research at MIT, Dana-Farber Cancer Institute, and Massachusetts General Hospital, on another approach in which a single injection of a reagent changes the DNA of blood stem cells. But there are obstacles to overcome there, too, that may be solved by advances in both the technology to modify genes and the biological understanding of blood cells.

Bradner expects further funding to come to reach patients around the world, once the science progresses more.

There is no plug-and-play solution for this project in the way that mRNA vaccines were perfectly set up for SARS-CoV-2. We have no such technology to immediately redeploy here, he said. Were going to have to reimagine what it means to be a gene therapy for this project.

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Novartis, Gates Foundation pursue a simpler gene therapy for sickle cell - STAT

Bone marrow transplant shows signs of curing brave little boy with one in a million condition – Shields Gazette

One-year-old Max Gardner was diagnosed with aplastic anaemia, in October 2020, a serious condition in which the bone marrow and stem cells do not produce enough blood cells.

After Max developed significant bruises and a rash over his body, parents, Connor Gardner and Rachel Nicholson, from Hebburn, were referred to South Tyneside District Hospital, where their brave little boy underwent tests.

Doctors initially believed that Max had an immune disorder but after he was admitted to the Royal Victoria Infirmary (RVI) further tests helped to diagnose him with aplastic anaemia.

The family was told that the condition could be fatal if not treated properly.

Doctors said Max needed to have a bone marrow transplant, which has the potential to cure him.

Dad Connor, 29, and mum Rachel, 27, were both tested to see if they would be a bone marrow match and the pair were overjoyed when Rachel was found to be a 9/10 match.

Max started chemotherapy on January 7 at the RVI and mum Rachel donated stem cells on January 13 at Newcastles Freeman Hospital.

The following day, January 14, Max underwent the transplant at the RVI.

The family is now waiting for the results of a Chimerism Test which will tell them for definite whether the stem cells have worked but signs are already looking positive.

Delighted dad, Connor, said: "His neutrophils [a type of white blood cell that protect us from infections] have been more than 0.50 for three days in a row, which means that he is essentially engrafted, which means that his body is accepting the transplant.

"So it is working, but we still have to wait for the test results."

Doctors say there is no doubt that it has worked with the way the numbers have gone up but they have to officially do it like that to make sure, Connor continued.

"But there is no reason why it shouldnt have [doctors] say.

"He has done really well to get to this stage, he has absolutely sailed through it, everyone is surprised with how well he has done.

This the best outcome we could have hoped for.

But it hasnt been plain sailing for the family, who have also had to face additional challenges during the treatment.

Parents Connor and Rachael initially were not allowed to visit Max at the same time due to Covid rules, however the hospital has now eased the restriction in their case.

The family also became sick with Norovirus in the run-up to the transplant, causing concern over whether it would have to be pushed back.

Thankfully, the transplant went ahead as planned and the family made a good recovery, although Max still needs help with his eating.

Max will now have to remain in hospital for a while longer as he recovers from the transplant.

Connor added: We can feel that we are nearly at the end of it.

"His neutrophils are the highest they have ever been since he became poorly so we feel like we are coming to the end.

The family are sharing Maxs journey to health on Instagram under the name @maxinamillionaajourney and hope his story will encourage people to sign up to the Anthony Nolan register to become a potential donor and help others like Max.

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Bone marrow transplant shows signs of curing brave little boy with one in a million condition - Shields Gazette

After 13 years of trials and tribulations RTP firm G1 wins first FDA approval for cancer drug – WRAL Tech Wire

RESEARCH TRIANGLE PARK After 13 years as a clinical-stage oncology company,G1 Therapeuticsof Research Triangle Park transformed into a commercial-stage company overnight upon the approval of its first drug by the U.S. Food and Drug Administration.

The FDA on Feb. 12 approved G1s trilaciclib, to be marketed as Cosela, for protecting bone marrow from chemotherapy damage in adult patients with extensive-stage small cell lung cancer (ES-SCLC).

Cosela will help change the chemotherapy experience for people who are battling ES-SCLC, said Jack Bailey, the companys chief executive officer. G1 is proud to deliver Cosela to patients and their families as the first and only therapy to help protect against chemotherapy-induced myelosuppression.

Myelosuppression, or damage to the bone marrow, is the most serious and life-threatening side effect of chemotherapy. Chemotherapy-induced myelosuppression reduces the bodys essential supply of white blood cells, red blood cells and platelets, and can lead to increased risks of infection, severe anemia and bleeding.

RTP drug firm G1 secures FDA approval for treatment to prevent chemo damage to bone marrow

These complications impact patients quality of life and may also result in chemotherapy dose reductions and delays, said Jeffrey Crawford,M.D., Geller Professorfor Research in Cancer in theDepartment of MedicineandDuke Cancer Institute. In clinical trials, the addition of trilaciclib to extensive-stage small cell lung cancer chemotherapy treatment regimens reduced myelosuppression and improved clinical outcomes.The good news is that these benefits of trilaciclib will now be available for our patients in clinical practice.

Cosela is expected to be commercially available through G1s specialty distributor partner network in early March, the company said.

G1 is committed to helping patients with in theU.S.gain access to treatment with Cosela through access and affordability programs. Patients and healthcare can call the companys support center at 833-418-6663 for information.

Cosela is intended to be given as a 30-minute infusion four hours prior to chemotherapy treatments containing platinum/etoposide or topotecan. About 90 percent of all patients with ES-SCLC receive at least one of these chemotherapy regimens during their treatment, according to G1.

The approval of Cosela is based on data from three randomized, placebo-controlled trials. Data showed that patients receiving Cosela before the start of chemotherapy had less neutropenia, an abnormally low number of neutrophils, white blood cells that fight bacterial and fungal infection.

Data also showed a positive impact on red blood cell transfusions and other myeloprotective measures.

Chemotherapy is the most effective and widely used approach to treating people diagnosed with extensive-stage small cell lung cancer, Bailey said. However, standard-of-care chemotherapy regimens are highly myelosuppressive and can lead to costly hospitalizations and rescue interventions.

To date, oncologists have relied on rescue therapy, a mix of growth factor agents, antibiotics and red blood cell transfusions, to restore bone marrow after it has been damaged by chemotherapy.

By contrast, trilaciclib provides the first proactive approach to myelosuppression through a unique mechanism of action that helps protect the bone marrow from damage by chemotherapy, Crawford said.

Cosela helps protect bone marrow cells from chemotherapy damage by inhibiting cyclin- dependent kinase 4 and 6, two enzymes involved in cancer cell growth. Inhibiting these enzymes temporarily stops hematopoietic stem cells and progenitor cells in the bone marrow from dividing, making them resistant to damage from chemotherapy drugs that target dividing cells.

Bonnie J. Addario, lung cancer survivor, co-founder and board chair of theGo2 Foundation for Lung Cancer, said many people with extensive-stage small cell lung cancerrely on chemotherapy to extend their lives and alleviate their symptoms.

Unfortunately, the vast majority will experience chemotherapy-induced side effects, resulting in dose delays and reductions, and increased utilization of healthcare services, she said.

G1 shares our organizations goal to improve the quality of life of those diagnosed with lung cancer and to transform survivorship among people living with this insidious disease. We are thrilled to see new advancements that can help improve the lives of those living with small cell lung cancer.

About 30,000 small cell lung cancer patients are treated inthe United Statesannually. SCLC, one of the two main types of lung cancer, accounts for about 10 to 15 percent of all lung cancers but is the more aggressive disease, tending to grow and spread faster than the other type, non-small cell lung cancer.

InJune 2020, G1 announced a three-yearco-promotion agreementwithBoehringer Ingelheimfor Cosela in small cell lung cancer in theU.S.andPuerto Rico. G1 will lead marketing, market access and medical engagement initiatives for Cosela whileBoehringer Ingelheimsoncology commercial team will lead sales force engagement initiatives.

G1 will book revenue and retain development and commercialization rights to Cosela and payBoehringer Ingelheima promotional fee based on net sales.

The three-year agreement does not extend to additional indications that G1 is evaluating for trilaciclib: breast, colorectal, bladder and non-small cell lung cancers.

G1 is a 2008 spin-out of the University of North Carolina at Chapel Hill.

The company raised $108 million in an initial public offering of stock in 2017 after receiving more than $95 million in three rounds of venture capital funding. The North Carolina Biotechnology Center provided two early-stage loans totaling $500,000.

G1s stock is traded on the Nasdaq Global Select Market under the ticker symbol GTHX.

(C) N.C. Biotech Center

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After 13 years of trials and tribulations RTP firm G1 wins first FDA approval for cancer drug - WRAL Tech Wire

Global Hematological Cancers Therapeutics Market Is Expected to Reach USD 82.40 billion by 2028 : Fior Markets – GlobeNewswire

Newark, NJ, Feb. 11, 2021 (GLOBE NEWSWIRE) -- As per the report published by Fior Markets, theglobal haematological cancer therapeutics market is expected to grow from USD 37.88 billion in 2020 and to reach USD 82.40 billion by 2028, growing at a CAGR of 10.2% during the forecast period 2021-2028.

The global hematological cancers therapeutics market is witnessing significant growth in recent years. This growth is attributed to the increased government spending and infrastructure development rate across the globe, increasing blood cancer incidences, and increasing investment in research and development. Other factors propelling the market growth include inventions of novel drugs and growing investment in research and development.

The haematological cancer is a class of cancer that affects one marrow, blood, and lymph nodes. It is mostly caused due to a long exposure of toxic substances like genetic predisposition, ionized radiation and chemical agents, improper assessment, viral infections, and other risks associated with other diseases with decreased immunity. Further, the bone marrow's stem cells develop into red blood cells, white blood cells, or platelets.

The global haematological cancer therapeutics market is expected to witness significant growth, owing to the increasing awareness about the possibility of early diagnosis, rising diagnostics rate, and advancements in biotechnology and pharmaceutical industries. The factors restraining the market growth are lack of awareness among people, high cost of medications, and haematological therapeutics' side effects.

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The key players operating in the global haematological cancer therapeutics market are Johnson & Johnson, Roche, Mindray Medical, Karyopharm Therapeutics, AbbVie, Abbott Laboratories, Celgene, Novartis, Bio-Rad Laboratories, HemoCue AB, Sysmex, and Siemens AG. To gain a significant market share in the global haematological cancer therapeutics market, the key players are now focusing on adopting strategies such as product innovations, mergers & acquisitions, recent developments, joint ventures, collaborations, and partnerships.

Pharmacological therapies segment dominated the market growth and held the largest share of 23.76% in the year 2020On the basis of type, the global haematological cancers therapeutics market is segmented into anaemia treatment, pharmacological therapies, steam cell transplantation, thrombosis treatment, surgery and radiation therapy, neutropenia treatment, and others. Pharmacological therapies segment dominated the market growth and held the largest share of 23.76% in the year 2020. This growth is attributed to the growing pharmaceutical sector, increasing research and developments, and rising cancer prevalence.

Hospitals segment dominated the market and held the largest share of 36.65% in the year 2020On the basis of end-user, the global hematological cancer therapeutics market is segmented into clinical laboratories, hospitals, academic and research institutes, and others. The hospitals segment dominated the market and held the largest share of 36.65% in the year 2020. This growth is attributed to the government and private bodies reimbursement and increased government spending on the hospital infrastructures.

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Regional Segment of Hematological Cancer Therapeutics Market

On the basis of geography, the global haematological cancer therapeutics market is classified into North America, Europe, Asia-Pacific, Middle East & Africa, and South America. Asia-Pacific region held the largest share of 39.68% in the year 2020. This growth is attributed to the increased government hospital expenditure, growing investment in research and development, and increasing awareness about cancer treatments. China holds the largest market share in the region due to investments by key pharmaceutical players to develop new drugs. North America is expected to witness significant growth, owing to the high prevalence of blood cancer patients and rising healthcare infrastructure.

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About the report:The global haematological cancer therapeutics market is analyzed on the basis of value (USD billion). All the segments have been analyzed on global, regional and country basis. The study includes the analysis of more than 30 countries for each segment. The report offers in-depth analysis of driving factors, opportunities, restraints, and challenges for gaining the key insights of the market. The study includes porters five forces model, attractiveness analysis, raw material analysis, and competitors position grid analysis.

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Global Hematological Cancers Therapeutics Market Is Expected to Reach USD 82.40 billion by 2028 : Fior Markets - GlobeNewswire

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

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

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

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

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

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

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

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

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

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

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

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

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

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

Types and selection of transplantation:

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

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

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

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

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

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

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

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

‘Whatever it takes’: Stem cell drive underway to find bone marrow match for girl on Alta. First Nation – CTV Edmonton

EDMONTON -- A little girl with leukemia in northern Alberta is in desperate need of a bone marrow transplant.

Friends and family of 10-year-old Ameilia Powder have set up a stem cell drive to find a match.

"Asking for help is probably one of the most difficult things to do when you're in this situation and really opening your story up to everyone is really hard. but at the end of the day Ameilia needs a bone marrow match," Ameilia's grandmother Jaime Harpe said. "I'll do whatever it takes to get her that match."

Ameilia was diagnosed in March 2020 and went through five months of treatment at the Stollery Children's Hospital before returning home to Fort McKay First Nation in August.

The cancer returned late last month and this time, a bone marrow transplant is the only option to save her life.

"All people have to do is go blood.ca/stemcells and they can register online, and a kit gets sent to you in the mail," organizer Amanda Main said. "You just swab your cheek, pop it back in and you get entered in the data base, that's all you have to do."

Potential matches need to be between the ages of 17 and 35.

A GoFundMe page has already raised more than $11,000.

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'Whatever it takes': Stem cell drive underway to find bone marrow match for girl on Alta. First Nation - CTV Edmonton

Novel Bone Marrow ‘Ingredient’ To Help Arthritic Horses The Horse – TheHorse.com

Regenerative therapies such as stem cells and platelet-rich plasma already play an important role in managing osteoarthritis (OA). Nonetheless, veterinarians have found that response to even these therapies is less than ideal in many cases, prompting researchers to continuously seek novel therapies for this all-too-common musculoskeletal disorder. One of the newest to be unveiled is called bone marrow mononuclear cell (BMNC) therapy. One researcher who presented at the 2020 American Association of Equine Practitioners Convention, held virtually, reported that the equine industry is in critical need for therapies that resolve joint inflammation but preserve tissue healing, and BMNC appears a promising candidate.

Much more than stem cells classically sought for cartilage healing, bone marrow is rich in macrophage progenitor cells, explained James B. Everett, DVM, MS, previously of the Virginia-Maryland College of Veterinary Medicine, who now works at the Equine Surgical Center at ThorSport Farm,in Murfreesboro, Tennessee. Macrophages are a type of white blood cell that play a role in tissue repair and cartilage integrity, and produce the anti-inflammatory mediators, including interleukin-10 (IL-10).

Everett said macrophages in the synovial (joint) membrane are essential for joint health, clearing aggressors, secreting key molecules required for optimal joint function, and forming a shield that protects tissues undergoing repair, similar to a wound scab. However, when the amount of tissue damage overwhelms these housekeeping functions, macrophages stimulate inflammation as a means of recruiting more cells, especially more macrophages, to cope with increased demands for repair.

If this response is efficiently accomplished, macrophages then produce, among other things, high concentrations of IL-10 and resolve the inflammatory process, returning the joint to a healthy state, he said.

Everett emphasized that not all inflammation is bad. This acute inflammation is essential to establish a resolving response, and anti-inflammatory therapies can negatively interfere.

As presented by Everetts colleague Bruno Menarim, DVM, PhD, in a separate session, studies show that BMNCs promote the endogenous resolution of experimentally induced inflammation. To see if these promising features translated to naturally occurring inflammation in live horses, Everetts research team studied 19 horses, dividing them into three treatment groups:

The selected horses were diagnosed with OA in a single joint, and the team injected those joints once with the saline, triamcinolone, or BMNCs. The BMNCs were autologous, meaning veterinarians collected them from each patients own bone marrow aspirate. They processed the aspirate in-house, and the isolated mononuclear cells, composed predominantly of macrophages, were ready to inject into the affected joint within three hours of aspiration.

We found that while objectively assessed lameness (via Lameness Locator) decreased in all three groups, it was only significant in the BMNC-treated horses, said Everett. Further, the treatment was well-tolerated with no adverse events appreciated in this study.

He said that using BMNCs can help reduce the need for chronic use of non-steroidal anti-inflammatory drugs and corticosteroids, which produces potentially harmful consequences. Further, BMNCs preserve the production of molecules such as interleukins and cytokines that are essential for restoring joint homeostasis. Corticosteroids often inhibit these molecules.

The researchers noted that these results support a larger clinical trial using BMNCs in clinical cases of equine OA.

Originally posted here:
Novel Bone Marrow 'Ingredient' To Help Arthritic Horses The Horse - TheHorse.com

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

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

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

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

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

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

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

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

SOURCE DKMS BMST Foundation India

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

Helping others helps Havard through sickness | Community | hannapub.com – The Franklin Sun

As an athlete, Courtlynn Havard has always set goals for herself to improve her game whether on the soccer or softball field. She has worked toward those goals through hard work and perseverance.

Now her goals have shifted slightly.

Courtlynn, a sophomore at Franklin Parish High School, currently has two main goals: to beat aplastic anemia and paroxysmal nocturnal hemoglobinuria (PNH) and to help others.

Helping others for Courtlynn brightens her day, strengthens her and gives her hope for a better tomorrow.

From that desire to help others, the Havard family is sponsoring a bone marrow drive at Life Church in Winnsboro Feb. 20 from 10 a.m. until 2 p.m.

The drive will be administered by DKMS, an international nonprofit organization, whose goal is to find bone marrow donors for people like Havard.

It is simple as a swab in your cheek, said Jaimie Havard, Courtlynns mother. That could save a life. It only takes a few minutes and is so easy for you to give somebody a second chance on life.

Anyone 18-55 with good health can participate in the drive. Participates stay in their car, watch a video on their phone and fill out a short form, said Amy Roseman of DMKS.

They are given a kit with a swab for their cheeks. The whole process takes 7-10 minutes.

In October, Courtlynn went to the doctor with kidney stones. When doctors took her blood, they found her blood count was low.

Her mother and Courtlynn met with an oncologist who gave them disturbing news.

At first the oncologist thought it was leukemia, Jaimie said. You feel like your whole world is collapsing. I didnt know what to say or do. Courtlynn was devastated and crying. I was trying to be strong for her.

The Havards were then sent to LSU Health Shreveport. The medical professionals there performed a bone marrow biopsy and found she had aplastic anemia and PNH.

You never think it can be your child, Jaimie said. It is really an unbelievable, indescribable feeling.

Aplastic anemia is a condition that occurs when your body stops producing enough new blood cells, according to the Mayo Clinic. The condition leaves a person fatigued and more prone to infections and uncontrolled bleeding.

A rare and serious condition, aplastic anemia can develop at any age. It can occur suddenly, or it can come on slowly and worsen over time and can be mild or severe.

Treatment for aplastic anemia might include medications, blood transfusions or a stem cell transplant, also known as a bone marrow transplant.

PNH is a rare acquired, life-threatening disease of the blood. The disease is characterized by destruction of red blood cells (hemolytic anemia), blood clots (thrombosis), and impaired bone marrow function (not making enough of the three blood components).

PNH affects 1-1.5 persons per million of the population and is primarily a disease of younger adults. The median age of diagnosis is 35-40 years of age, with occasional cases diagnosed in childhood or adolescence. PNH is closely related to aplastic anemia.

Courtynns world use to evolve around sports, mud riding with her friends and being very social.

Now because of her weak immune system, she has to be careful and stay at home with her English Spaniel, Old Mack, and go to school virtually. Courtlynn talks to her friends via cell phone, computer and occasionally gets a visit from one that sits on her porch while she socially distances.

I missed my friends the most, Courtlynn said. I am one of those people that get up and go. I dont stay still.

She also goes to doctors whether it be locally to get her blood count tested, or Shreveport to receive platelets and blood or Memphis to St. Judes.

Saving people around the world

One thing that drives Courtlynn through this situation is keeping up with other kids situations, Jaimie said. There are so many stories out there of kids looking for bone marrow transplants.

The Feb. 20 bone marrow drive is a way Courtlynn and her family can help others.

DKMS has been finding matching bone marrow donors for 30 years. They are now in the United States, Germany, Poland, India and South Africa.

Sadly, only 2 percent of Americans have signed up as potential donors, Roseman said. We are hoping we will have really nice support for Courtlynn and the other patients looking for donors.

Reasons vary for the low percentage of potential donors, but Roseman attributes not knowing the need and ease of the process.

You fill out contact information, swab your cheeks and you are put in a data base that is only seen by medical teams searching for donors, Roseman said. We call it, youre a hero in waiting.

The biggest misconception is the donation of bone marrow if you are a match.

You are asked to donate stem cells from the blood stream, Roseman said. It is a very easy process. It is very similar to donating plasma or platelets and takes a morning or afternoon.

Blood is taken from one arm, and the blood is put back into the other arm, Roseman, said. Stem cells lost in the process will regenerate.

You have given someone a second chance in life, Roseman said.

A person may be asked to donate actual bone marrow if he or she is matched to a three year old or younger. This procedure is done in a hospital and takes less than an hour. DKMS pays for the hospital visit and time loss from work.

It is amazing to think about giving someone a second chance on life by giving up a just morning of your time, Roseman said.

A community comes together

The Franklin Parish community has come together in support of Courtlynn and the Havard family.

Boutique shops, individuals and restaurants have come together to raise money for her cause.

During the Feb. 20 bone marrow drive at Life Church a BBQ chicken plate lunch will be on sale for $10. T-shirts will also be on sale at the event. Keep up with all drives and Courtlynns journey on her Facebook page: Courtlynns Compass.

We have a really amazing group of friends and family that have come together, Jaimie said. Our local community has stepped up big time. Complete strangers are showing so much love and support for my baby. It speaks volumes for our little town we live in.

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Helping others helps Havard through sickness | Community | hannapub.com - The Franklin Sun

Why Cynata is hopeful its COVID treatment trial will succeed where others have failed – Business News Australia

Cynata Therapeutics (ASX: CYP), founded by two clever stem cell researchers and one wise Australian techpreneur, is in the process of developing a treatment for COVID-19.

Using its in-house stem cell technology Cymerus, the ASX-listed biotech hopes to treat one of the deadliest complications of COVID-19 -acute respiratory distress syndrome (ARDS).

In doing so Cynata would achieve what competitor Mesoblast (ASX: MSB) couldn't with FDA approval.

By deploying an industrialised approach to stem cell therapeutics, Cynata CEO Ross Macdonald (pictured) is confident the clinical trial process won't leave the company hamstrung.

In 1981 scientists discovered a way to derive embryonic stem cells from early mouse embryos.

The discovery thrilled scientists, and eventually led to the development of a method to do the same in lab-grown human embryos by 1998.

While there have been plenty of discussions surrounding the ethics of using of embryonic stem cells, these major scientific movements have pushed researchers to discover new and inventive ways of treating a whole raft of diseases and infections.

One such researcher, Dr Ian Dixon, saw potential for the use of mesenschymal stem cells (MSCs) - a type of stem cell that can differentiate into a variety of cell types enabling the treatment of many diseases and infections.

However there was still an obstacle to overcome: how do you mass produce enough cells needed to commercialise a treatment?

Luckily, two researchers at the University of Wisconson, Professor Igor Slukvin and Dr Maksym Vodyanik, had invented a biotechnological breakthrough called Cymerus.

The technology was able to do exactly what Dixon needed: the consistent manufacture of MSCs on an ultra-large scale; basically what Henry Ford did to the industrialisation of the auto industry, but for stem cells.

So in 2003 Dixon partnered with the two researchers to start Cynata - now an ASX-listed biotechnology company trialing a number of different treatments for a wide variety of ailments.

Most recently, Cynata's focus has been on developing a treatment for a complication of COVID-19 called acute respiratory distress syndrome (ARDS).

The complication ravages COVID-19 infected patients, destroying their organs through what is known as a cytokine storm. The complication is estimated to kill up to half of COVID-19 patients that suffer from it.

Melbourne-based Cynata is currently in the very early stages of its investigation into whether its MSCs will be able to treat the coronavirus complication overwhelming hospitals globally.

If this all sounds familiar, you might be thinking of another ASX-listed biotech called Mesoblast (ASX: MSB).

In March last year Mesoblast, also based in Melbourne, saw its shares surge after announcing plans to evaluate its stem cell treatment solutions on COVID-19 patients.

The group commenced the arduous clinical trial process to see if its remestemcel-L therapy could treat ARDS by using bone marrow aspirate from healthy donors - a similar approach the company had already taken to treat a condition many suffer from after receiving bone marrow transplants.

Mesoblast was riding high on the ASX following positive announcements surrounding the clinical traila process, especially back in April 2020 when a trial at New York City's Mt Sinai hospital found its remestemcel-L therpay achieved "remarkable" results.

Serious attention gathered around Mesoblast, with the company even securing $138 in funds from investors to continue its important research.

The company went so far as to sign a commercialisation deal for the COVID-19 treatment with Novartis, and the US Food and Drugs Administration (FDA) fast tracked the approvals process for the potential game-changing treatment.

However, in December 2020, Mesoblast hit a stumbling block.

Mesoblast's COVID-19 treatment flunked the test - its remestemcel-L therapy failed to show a lower mortality rate for patients in the prescribed 30-day timeframe of treatment.

At that point Cynata had commenced research into its own ARDS treatment. But did Mesoblast's failure unnerve Cynata CEO Ross Macdonald? Not a chance.

"I'm more confident that our trial will be successful where theirs was a failure," Macdonald said.

"If you use a process like we have developed - we don't rely on multiple different [stem cell] donations. You start with exactly the same material every time."

To explain, Macdonald used the analogy of a local caf; you normally expect a coffee from one caf to taste more or less exactly the same every time you go there - the same beans are used every time.

Whereas Macdonald said Mesoblast's process is like going to the same caf every day, but each visit they use different beans from a different supplier which leads to inconsistency in taste and flavour.

Cynata's approach with its MSCs is in line with the first example - what you get the first time from them will be replicated in each and every dose of the drug - while MSB's is like the latter.

"Yes, you still got the coffee, but the experience of the taste is totally different than it was yesterday," he said.

"The FDA said to Mesoblast, well you've got a manufacturing problem that is reliant upon multiple donors prepared to donate bone marrow and that is flawed.

"So with that in mind it's perhaps not surprising that they had a pretty disappointing result in the clinical trials."

Additionally, Macdonald said the initial investor reactions to MSB's early COVID-19 trail results were overblown.

"The initial data from their trial that got everybody excited was, in my view, quite flawed, because they said "look at how many patients are dying in intensive care units with COVID compared the patients that we treated," he said.

"But the reality of the situation was quite different. The control group at that time - the death rate was way, way higher than you would typically see for ARDS, whether its COVID or anything else. And it was simply because of the chaos that existed in intensive care units in New York in the first wave.

"So we think that the initial enthusiasm was perhaps a little misguided."

When asked why Mesoblast is receiving so much attention compared to Cynata, especially considering the above, Macdonald said it was simply because MSB is bigger and has been around for longer. For context, MSB has a market capitalisation of $1.46 billion, whereas Cynata's is just $94.56 million.

"I'd love to know why there is less attention, and how we can get our market cap above a billion dollars," joked Macdonald.

"I think the answer though is that they've been around for a lot longer than we have, they have spent a hell of a lot more money than we've spent - their monthly spend is more than we've spent for pretty much our entire existence.

"But I think the fundamental reason why is that data drives value in biotech, so the more clinical data you generate that shows your product works, the more attention you attract from investors."

That's not to say Cynata is being totally ignored in favour of the larger Mesoblast.

The company secured a $15 million placement led by $10 million from healthcare investor BioScience Managers in December.

The funds will be used to expand Cynata's clinical development pipeline and scale their operations in Australia.

As such, the company is preparing to expand its clinical development pipeline to include idiopathic pulmonary fibrosis, renal transplantation, and diabetic foot ulcers.

"So we're starting to garner that attention now that says two things - one, cell therapies are definitely a medical revolution and two, Cynata is part of that new generation of companies," Macdonald said.

As for the company's pipeline, in addition to the COVID treatment trials, Cynata is planning on launching three new clinical candidates that will get under way this year.

There's also Cynata's osteoarthritis trial, which Macdonald describes as significant for the biotech company; with 2 million patients in Australia and 30 million in the United States the company is hoping to tap into an $11 billion plus addressable market.

"It will ultimately show whether MSCs are useful in that particularly devastating condition," he said.

"It doesn't just affect people who want to go and play golf or tennis, it affects, particularly manual labourers who can no longer work.

"So the cost to the economy of osteoarthritis is quite significant, which is of course one of the reasons why the Australian Government is funding this trial."

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Business News Australia

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Why Cynata is hopeful its COVID treatment trial will succeed where others have failed - Business News Australia

Anti-Emetic Drug Effectively Blocks CD93 Signaling in Preclinical Evaluations, Suggesting Suitability in CML – Cancer Therapy Advisor

The anti-emetic agent metoclopramide blocked CD93 signaling in cell culture and delayed leukemia development in mice, according to data from a preclinical study published in Cell Reports. The results indicate that CD93 signaling, which is an important regulator of self-renewal and proliferation of murine and human leukemia stem cells (LSCs), could be a potential therapeutic target for the elimination of LSCs in chronic myeloid leukemia (CML).

To characterize the function of CD93 in CML, the researchers first demonstrated that all subsets of LSCs expressed CD93 while more differentiated leukemia granulocytes did not. Though CD93 was shown to encourage self-renewal and proliferation of murine and human LSCs, it notably had no such effect on hematopoietic stem cells.

In an experiment, the investigators injected mice with LSCs that were either proficient or deficient in CD93. Mice with CD93-deficient LSCs were found to incorporate bromodeoxyuridine, which is used to detect proliferating cells, at a lower rate than mice with CD93-proficient LSCs. The finding suggests that proliferation of LSCs is impaired when CD93 is absent.

Next, a drug library was used to screen for compounds that could block CD93 signaling. Among the 240 compounds evaluated in vitro, 10 blocked CD93 signaling; one of the compounds was the anti-emetic agent metoclopramide.

Mice were then treated with either vehicle or metoclopramide. Notably, metoclopramide-treated mice had delayed leukemia development and lived longer than vehicle-treated mice. Among the metoclopramide-receiving mice, most genes were downregulated in the LSCs, particularly genes that promote stem cell maintenance and myeloid differentiation, cell proliferation and survival, response to cytokine signaling, and gene expression.

In vitro exposure to metoclopramide was found to disrupt colony formation in human bone marrow CML stem/progenitor cells. A control experiment showed that metoclopramide had no effect on hematopoietic stem/progenitor cells from humans with healthy bone marrow.

The study authors reasoned that because metoclopramide is a very well-tolerated and cheap anti-emetic drug, its LSC-eradicating activity in patients with CML can be directly tested in clinical drug repurposing studies.

Reference

Riether C, Radpour B, Kallen NM, et al. Metoclopramide treatment blocks CD93-signaling-mediated self-renewal of chronic myeloid leukemia stem cells. Cell Rep. 2021;34(4):108663. doi:10.1016/j.celrep.2020.108663

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Anti-Emetic Drug Effectively Blocks CD93 Signaling in Preclinical Evaluations, Suggesting Suitability in CML - Cancer Therapy Advisor

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

Dave Asprey  |  Photo Credit: Twitter

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

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

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

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

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

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

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

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

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

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

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

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

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

World Cancer Day 2021: What are Blood Cancers, Types, Symptoms, Treatment and data in India? – Jagran Josh

World Cancer Day 2021: It is observed on 4 February every year and this year the theme is "I Am and I Will".The campaign shows that our actions have an impact on everyone around us, within our neighbourhoods, communities, and cities. This year is a reminder of the enduring power of cooperation and collective action i.e. together, all our actions matter.

What are Blood Cancers?

It is a type of cancer that affects blood cells and affects the production and function of blood cells.

This type of cancer starts in the bone marrow which is the main source of blood production.

It occurs when abnormal blood cells start growing out of control and interrupt the function of normal blood cells that fight off infection and produce new blood cells.

World Cancer Day 2021: Current Theme, History and Key Facts

Blood Cancer: Types

Mainly, there are three types of blood cancers namely leukemia, lymphoma, and myeloma.

Leukemia

It is a blood cancer that originates in the blood and bone marrow. It is caused by the rapid production of abnormal white blood cells and interferes with the bone marrow's ability to make red blood cells and platelets. These high numbers of abnormal white blood cells are not able to fight infection.

Lymphoma

This type of blood cancer affects the lymphatic system, which removes excess fluids from the body and produces immune cells. As we know that lymphocytes are a type of white blood cell that fights infection. Therefore, abnormal lymphocytes become lymphoma cells that multiply and collect in the lymph nodes and other tissues. And over time, these cancerous cells impair the immune system of the body.

Myeloma

It is a type of blood cancer that begins in the plasma cells of blood which is a type of white blood cell made in the bone marrow. Plasma cells are white blood cells that produce antibodies to fight against infection and disease in the body. So, myeloma cells prevent the normal production of antibodies which make the immune system weak and susceptible to infection.

Blood Cancer: Symptoms

Loss of appetite, nausea

Fever, chills

Night sweats

Persistent fatigue, weakness

Unexplained weight loss

Bone/joint pain

Shortness of breath

Abdominal discomfort

Frequent infections

Itchy skin or skin rash

Swollen lymph nodes in the neck, underarms or groin

Delirium and confusion

Decreased urination and difficulty while urinating

What is High Grade Metastatic Cancer?

Blood Cancer: Treatment

On the type of cancer, treatment depends and how fast the cancer is progressing, where cancer has spread, and other factors. Some common treatment for blood cancer are:

Chemotherapy: In this anticancer cancer drugs are provided to the patient to interfere with and stop the growth of cancer cells in the body. In blood cancer, in chemotherapy treatment, sometimes several drugs are given together in a set regimen. This treatment may also be given before a stem cell transplant.

Radiation therapy: In this type of cancer treatment high-energy rays are given to kill cancer cells. It may also be given before a stem cell transplant.

Stem cell transplantation: In this type of treatment, healthy stem cells are infused into the patient body to help resume healthy blood production following therapy to destroy malignant blood cells. Stem cells may be collected from the bone marrow, circulating blood, and umbilical cord blood.

Blood Cancer in India

In India, over 20,000 new cases of childhood blood cancer are diagnosed every year of which nearly 15,000 of those cases are leukemia as per Globocan 2020.

The most common type of blood cancer is leukemia that affects children and teens (0-19 years) and one of the leading causes of death.

As per some studies, India ranks 3rd highest in reported cases of Blood Cancer after the US and China. Several factors are responsible like low accessibility of affordable healthcare in rural areas, lack of awareness and education on Blood Cancer, etc.

So, now you may have come to know about the blood cancer, types, symptoms, treatment, etc.

GK Questions and Answers on Types of Cancers

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World Cancer Day 2021: What are Blood Cancers, Types, Symptoms, Treatment and data in India? - Jagran Josh

India Stem Cell Market speedy growth at US$ 1.27 Bn by 2028 with Thermofisher Scientific India, Pluristem Technologies, Becton Dickinson Private…

India Stem Cell Market to surpass huge revenue of USD 1.27 Billion at CAGR +13% by 2028.

Stem cell therapy in India helps in treating several diseases, including leukaemia, lymphoma, thalassemia, Parkinsons, Alzheimers, stroke, cerebral palsy, spinal cord injury, muscular dystrophy, etc. Stem cell therapy in India has shown promising results in India and as well as all over the world.

In comparison, in India it costs INR 10-20 lakh in private hospitals, while in government hospitals it is much cheaper INR 3-6 lakh depending on the type of procedure, he said

On average, private banking of stem cells derived from cord blood costs INR 50,000-70,000. Banks claim to freeze the cells in liquid nitrogen so that it can be used up to 20 years from the date of preservation.

Researchers hope stem cells will one day be effective in the treatment of many medical conditions and diseases. But unproven stem cell treatments can be unsafe so get all of the facts if youre considering any treatment. Stem cells have been called everything from cure-alls to miracle treatments.

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Key players profiled in the report includes:

Thermofisher Scientific India Pvt. Ltd., Pluristem Technologies Ltd., Becton Dickinson Private Limited, Stem Cell Technologies India Pvt. Ltd., Merck Lifescience Pvt. Ltd., Cordlife India Pvt. Ltd., LifeCell International Pvt. Ltd., StemCyte India Therapeutics Private Limited, Stempeutics Research Private Limited, ReeLabs Private Limited, CryoSave, Indu Stem Cell Bank, Path Care Labs Pvt

The aim of the report is to equip relevant players in deciphering essential cues about the various real-time market based developments, also drawing significant references from historical data, to eventually present a highly effective market forecast and prediction, favoring sustainable stance and impeccable revenue flow despite challenges such as sudden pandemic, interrupted production and disrupted sales channel in the India Stem Cell market.

Market segments on the basis of:

This research report is an amalgamation of all relevant data pertaining to historic and current market specific information that systematically decide the future growth prospects of the India Stem Cell market. This section of the report further aims to enlighten report readers about the decisive developments and catastrophic implications caused by an unprecedented incident such as the pandemic that has visibly rendered unparalleled implications across the market.

This report is well documented to present crucial analytical review affecting the India Stem Cell market amidst COVID-19 outrage. The report is so designed to lend versatile understanding about various market influencers encompassing a thorough barrier analysis as well as an opportunity mapping that together decide the upcoming growth trajectory of the market. In the light of the lingering COVID-19 pandemic, this mindfully drafted research offering is in complete sync with the current ongoing market developments as well as challenges that together render tangible influence upon the holistic growth trajectory of the India Stem Cell market.

Besides presenting a discerning overview of the historical and current market specific developments, inclined to aid a future-ready business decision, this well-compiled research report on the India Stem Cell market also presents vital details on various industry best practices comprising SWOT and PESTEL analysis to adequately locate and maneuver profit scope. Therefore, to enable and influence a flawless market-specific business decision, aligning with the best industry practices, this specific research report on the market also lends a systematic rundown on vital growth triggering elements comprising market opportunities, persistent market obstacles and challenges, also featuring a comprehensive outlook of various drivers and threats that eventually influence the growth trajectory in the India Stem Cell market.

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India Stem Cell Geographical Segmentation Includes:

North America (U.S., Canada, Mexico)

Europe (U.K., France, Germany, Spain, Italy, Central & Eastern Europe, CIS)

Asia Pacific (China, Japan, South Korea, ASEAN, India, Rest of Asia Pacific)

Latin America (Brazil, Rest of L.A.)

Middle East and Africa (Turkey, GCC, Rest of Middle East)

Some Major TOC Points:

Chapter 1. Report Overview

Chapter 2. Growth Trends

Chapter 3. Market Share by Key Players

Chapter 4. Breakdown Data by Type and Application

Chapter 5. Market by End Users/Application

Chapter 6. COVID-19 Outbreak: India Stem Cell Industry Impact

Chapter 7. Opportunity Analysis in Covid-19 Crisis

Chapter 9. Market Driving Force

And More

In this latest research publication a thorough overview of the current market scenario has been portrayed, in a bid to aid market participants, stakeholders, research analysts, industry veterans and the like to borrow insightful cues from this ready-to-use market research report, thus influencing a definitive business discretion. The report in its subsequent sections also portrays a detailed overview of competition spectrum, profiling leading players and their mindful business decisions, influencing growth in the India Stem Cell market.

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India Stem Cell Market speedy growth at US$ 1.27 Bn by 2028 with Thermofisher Scientific India, Pluristem Technologies, Becton Dickinson Private...

[Full text] Retrospective Study on Implantation of Autologous-Cultured Osteoblasts | ORR – Dove Medical Press

Introduction

Osteonecrosis of the femoral head is a progressive disorder that causes pain and often progresses to hip joint collapse, finally resulting in disabling arthritis.1,2 It occurs between 30 to 50 years of age, and prevails at a relatively younger age in Asians compared to their western counterparts.3 It is estimated that approximately 20,00030,000 new cases of osteonecrosis are diagnosed in the United States each year, accounting for 10% of total hip arthroplasties performed.4 The Indian Society of Hip and Knee Surgeons has reported that more than 50% of all hip replacements in India are performed for osteonecrosis.5 Many studies have reported osteonecrosis to be more prevalent in men compared to women (3 or 5:1).3 The underlying pathophysiology of osteonecrosis remains unclear; however, it is multifactorial and several traumatic and nontraumatic etiological factors may contribute to its development. Traumatic events that may cause osteonecrosis include femoral neck/head fracture, hip dislocation, or slipped capital femoral epiphysis. Nontraumatic factors include use of steroids, alcoholism, metabolic disorders such as Cushings syndrome, and inherited disorders such as sickle cell disease.6,7 Besides the known traumatic and nontraumatic causes, some cases of osteonecrosis are idiopathic.1,8

Osteonecrosis of the femoral head may progress to secondary arthritis, and degeneration of articulating surface from advanced osteonecrosis necessitates total hip arthroplasty (THA). A primary treatment target of osteonecrosis of femoral head is to delay/prevent progression to osteoarthritis. Core decompression (CD) is the most widely used procedure in clinical practice; however, it has shown poor clinical outcomes, with up to 40% of patients having to undergo THA despite undergoing core decompression procedure.8 Therefore, a more pathophysiological approach may be required to treat osteonecrosis of femoral head. Osteonecrosis is characterized by a reduction in the osteogenic progenitor cells, an increase in osteoblast death, and altered intramedullary vascular supply due to trauma.1 It was observed that the number and function of mesenchymal cells in hematopoietic tissue and stroma of the bone marrow decreased in osteonecrosis patients.2 This observation indicated potential for using bone marrow stromal cells for the treatment of osteonecrosis, and consequently, several clinical studies have demonstrated encouraging results.2 A meta-analysis also showed that treatment with cell therapy compared to core decompression alone increased Harris hip score, decreased necrotic area of femoral head and collapse of femoral head, and reduced THA conversion rate.9 However, a recent randomized study has shown that bone marrow cell implantation in addition to core decompression did not improve THA conversion rate in patients with grade 3 osteonecrosis.10 The ideal treatment goal for osteonecrosis is to facilitate new bone formation in the place of dead bone that can provide pain relief, cease disease progression, prevent joint collapse, and preserve the joint. The fact thatbone marrow aspirate consists of mesenchymal stem cells raised a possibility if bone marrow cells could be differentiated into bone forming cells or osteoblasts and characterized by bone alkaline phosphatase. In a randomized trial, autologous osteoblast implantation was shown to significantly delay the evolution to subchondral fracture and reduce pain compared to bone marrow aspirate.11

OSSGROW (Regrow Biosciences Pvt Ltd., Mumbai, India) is a commercially available technology that involves implantation of autologous adult live-cultured osteoblasts (AALCO) derived from mesenchymal stem cells sourced from the bone marrow aspirate for osteonecrosis of the hip that received conditional marketing approval in India in March 2017.12 Here, we evaluated the efficacy of OSSGROW implantation technique by assessing retrospective data from patients with osteonecrosis who underwent the procedure. We also evaluated the correlation between Ficat-Arlet stages of osteonecrosis and clinical outcomes of the AALCO implantation procedure.

This retrospective, observational, non-comparative study was conducted at 37 centers in India. We retrospectively reviewed the data of patients with osteonecrosis of the femoral head who had undergone OSSGROW (AALCO) from 2010 to 2015. Key inclusion criteria were patients aged 12 years with a confirmed diagnosis of osteonecrosis in one or both hip joints who had undergone AALCO implantation. Diagnosis, analysis, and classification of osteonecrosis were done according to Ficat-Arlet based on radiography, computed tomography (CT) scans, and magnetic resonance imaging (MRI) findings. Patients whose medical records were not complete or were lost to follow-up were excluded from the study.

The protocol was approved by the Institutional Ethics Committee - Regrow Biosciences Pvt Ltd. and as this study was a retrospective study, informed consent was not required to review medical records. We also sought permission from the head of the institutes/departments before data collection. Patient data confidentiality was maintained in this study.

All the patients had undergone AALCO implantation on the recommendation of their consulting orthopedic surgeon after having received an explanation of the complications of osteonecrosis, the therapeutic options available, and the risks involved with the implantation procedure. Osteoblasts from patients were obtained from bone marrow aspiration from the posterior/superior iliac crest. Mesenchymal stem cells from the bone marrow were isolated and differentiated ex vivo into osteoblasts. Osteoblasts were then cultured for approximately 4 weeks under stringent laboratory conditions and multiplied up to 48 million osteoblasts (Figure 1). The cultured cells were implanted using a gel (Tisseel kit from Baxter) at the site of osteonecrosis through a minimally invasive surgery in a 3-step procedure: core decompression, curettage, and injection of osteoblasts (Figure 2).

Figure 1 Microscopic image of osteoblast in culture used for the final cell product before cell implantation.

Figure 2 Steps of osteoblasts implantation. (A) Step 1 Insertion of guide wire in center of lesion as identified on the MRI. (B) Step 2 Guide wire and 8mm cannulated drill for core decompression. The entry point of the guide wire is near the vastus ridge, to prevent a fracture due to stress-riser, greater width of femur and faster healing due to cancellous bone. (C) Step 3 Curettage: a variety of angulated curettes is used to do forage (curettage to remove necrotic bone). This bone is sent for biopsy.

Patients were operated on under spinal anesthesia. Core decompression tunnels were created into the subchondral necrotic lesion of the femoral head, approximately 23 mm away from the joint cartilage, by using 2.0-mm K-wires under fluoroscopic guidance through the greater trochanter and the femoral neck, and over drilled using trephine was performed by the centrally positioned K-wire. Cultured osteoblasts were injected following the curettage, and necrotic tissues were removed.

The patients had to undergo appropriate rehabilitation therapy after the implantation, which included complete bed-rest for 4 weeks post-implantation. After 4 weeks, passive lower limb exercises were performed for 2 weeks following post-implantation. Accordingly, non-weight bearing, partial weight bearing, and full weight bearing exercises were suggested as per the study protocol. Descriptive demographic and clinical data recorded before and after the procedure were collected from patient records. Past medical history, concomitant medications, and surgical treatments undertaken before and after the AALCO were recorded. Pre-existing risk factors for osteonecrosis such as steroid intake, alcohol consumption, comorbid conditions, or trauma were also noted.

Improvement in functional capacity and pain reduction were evaluated using Harris Hip Score (HHS) and visual analog scale (VAS) respectively at the time of pre- and post-operative consultations. Continued use of steroids or alcohol consumption after undergoing the AALCO implantation was recorded. The main outcome of the study was the need for THA (THA conversion rate). Based on these parameters, the treatment outcome was determined to be either improved (better score after AALCO implantation), stable (same condition as before AALCO implantation), or progressive (worse scores following AALCO implantation).

Continuous and quantitative variables were summarized using descriptive statistics and compared using Students t-test or nonparametric test, as applicable. Categorical data were presented as frequency count (n) and percentages (%) and were compared using the 2 test or Fishers exact test. P-values <0.05 were considered significant. All analyses were performed using the SPSS version 10.0.

Data from 64 patients were collected and analyzed as per the study protocol, and 101 hip joints were assessed. The age of patients ranged from 1270 years and BMI ranged from 20.632 kg/m2. The majority of the patients were men (79.7%). The mean duration since diagnosis of osteonecrosis was 7.4 1.6 years and the mean duration of AALCO treatment was 6.3 1.4 years (Table 1). Unilateral involvement of the hip joint was seen in 42.2% of cases. Bilateral involvement of hip joints was seen in 57.8% of patients. The majority of hips diagnosed were grade III (42.1%) and grade IV osteonecrosis (10.5%). While the exact cause for osteonecrosis was not known (idiopathic) in 25% of patients, 35.9% of cases were linked to steroid use and 26.6% to alcohol abuse. Records of concomitant medications revealed that 91.9% of patients were on analgesics, 8.1% were on ayurvedic treatment, and 1 patient took bisphosphonate.

Table 1 Demographic Characteristics

A total of 98 hip joints were assessed as data of 3 patients were not available for changes in mean VAS scores (improvement in pain), before and after the AALCO implantation. As shown in Figure 3A, the mean VAS score reduced significantly after a mean 6.3 years of AALCO treatment compared to the baseline (32.2 32.1 vs 58.8 13.8; mean difference: 26.5 35.2, p = 0.001) indicating significant improvement in pain. Similarly, HHS also improved post-operatively (47.1 12.3 vs 63.7 27.7; mean difference: 16.7 28.7, p = 0.001) showing functional improvement of patients. We categorized patients based on their HSS score (<70: poor, 7080: fair, 8090: good, 90100: excellent). At baseline, 96 hips (98%) had HSS score of <70, each of the two remaining hips had scores of <80 and <90, respectively. Improvement in HSS scores was seen at follow-up with 42 hips (43.3%) with HSS <70, 11 (11.3%) with 7080, 26 (26.8%) with 8090, and 18 (18.6%) with HSS scores of 90100.

Figure 3 (A) Changes in visual analog scale (VAS) and Harris hip scores. (B) Need for hip replacement surgery in different grades of osteonecrosis. (Osteonecrosis graded according to Association Research Circulation Osseous criteria).

The mean follow-up period since diagnosis of osteonecrosis was 6.3 years (range 49 years). Following AALCO treatment, 29 (28.7%) hips underwent THA, indicating that AALCO treatment could prevent and delay THA for 71.3% of hips. The mean time to THA was 3.2 2.0 years (range: 19 years). A total of 9 (39.1%) grade II, 11 (47.8%) grade III, and 3 (13%) grade IV hip joints required THA surgery (Figure 3B). In other words, AALCO treatment could delay THA for up to 3 years in 80% of hips in early stage osteonecrosis (Grades I and II) and 72% of hips in late stage osteonecrosis (Grades III and IV). Univariate analysis showed that the age of the patient, BMI, gender of patients, the side of osteonecrosis, and duration of disease had no effect on the clinical success of the procedure. Following AALCO treatment, 35.9% of patients continued using steroids and 29.7% continued with alcohol consumption. Of the total 29 hip joints that required surgery at follow-up, 20.7% and 41.4% had an associated etiology of alcohol consumption and steroid intake, respectively (Figure 4A). Overall, a significantly greater number of patients with underlying etiologies of alcohol consumption, smoking, or taking steroids required THA compared to those without these etiologies (14 [37.8%] vs 3 [11.1%], p = 0.017).

Figure 4 (A) Need for hip replacement stratified as per etiology of osteonecrosis. (B) Overall outcome stratified as per the grades of osteonecrosis.

Abbreviations: RA, rheumatoid arthritis; SLE, systemic lupus erythematous.

Based on the pre- and post-operative data, the condition of 65.6% of patients improved and 1.6% remained stable following AALCO treatment. Overall, the condition of 65.9% of hips (56/85) in grade I to grade III improved (Figure 4B). For quick reference, the pre- and post-operative radiograph images for a given patient are presented in Figure 5.

Figure 5 Pre- and posttransplantation MRI and X-ray images (A): pre-operative MRI (male patient [35 years]): Ficat and Arlet Stage II B with a subchondral fracture of right hip with a large anterolateral lesion(arrow) involving more than 40% of femoral head and less than 2mm depression at high risk of collapse. Etiology is post steroid AVN. (B) Post-operative MRI at 5 months post-surgery. (C) Post-operative X-ray at 4 years after surgery; anteroposterior (AP) view and lateral view.

We retrospectively studied the clinical outcomes of AALCO treatment. Our results showed that there was a reduction in pain and improvement in joint function following AALCO implantation, as was evident from a statistically significant reduction in the mean VAS score and increase in the HHS score. Of all the hips that underwent the AALCO implantation, 60% improved and 38% worsened with a THA conversion rate of 28%.

AALCO is a minimally invasive, surgical 3-step procedure with each step contributing significantly to the overall effectiveness of the treatment. The first step is core decompression that reduces pressure allowing increased blood flow. In the second step, the necrotic bone is debrided by a curette that promotes new bone formation. The third and most important step is implantation of osteoblasts that form new bone. The THA conversion rate is reported lower with core decompression compared to natural progression of disease, but approximately 40% of patients still required THA.8 Bone marrow cell therapy was shown to improve the THA conversion rate further.9 In a recent randomized trial, implantation of autologous bone marrow aspirate concentrate did not show any improvement in patients with grade 3 osteonecrosis.10 In our study, AALCO implantation avoided THA in 72% of hips in late grade osteonecrosis, suggesting that the technique may even benefit patients in advanced stages of disease; however, our results are limited by the relatively small numbers of patients belonging to each stage.

The differences in the THA conversion may not be directly comparable to those with others may be due to the diversity in the presentation of patients, differences in the follow-up period, or the AALCO technique.13,14 The THA conversion rate certainly remains low with AALCO treatment compared to 75% THA conversion rate reported in patients with natural progression to osteoarthritis resulting from osteonecrosis of the femoral head.15,16 A randomized study found autologous osteoblastic cells implantation to be more efficacious than bone marrow implantation as an adjunct to core decompression. The disease progression rate was found to be 20% in patients who had undergone autologous osteoblasts implantation vs 47% in patients in the bone marrow implantation group.11 Bone alkaline phosphatase-characterized osteoblasts have better regenerative potential compared to heterogeneous bone marrow cells.17,18 Use of these characterized cells could explain the favorable outcomes of AALCO implantation in our study.

Intake of alcohol and/or steroids is known to adversely affect bone renewal by causing an imbalance between the normal progenitor cells and the fat-storing bone marrow progenitor cells.1,19,20 The latter phenotype also leads to fat embolism and arteriosclerosis reducing the blood supply to necrotic tissues.1,19,20 In our study, alcohol and steroid intake were associated with occurrence of osteonecrosis of the femoral head in more than a quarter of patients. These results highlight the adverse impact of alcohol and steroid intake on the progression of osteonecrosis that is already evident in the literature in the pathogenesis of osteonecrosis.2125 As expected, THA conversion rate was also higher among patients who consumed alcohol and/or used steroids compared to those who did not in our study, signifying the adverse impact of alcohol and steroids on the AALCO treatment outcomes. However, a consensus on the specific mechanisms leading to these observations is yet to be reached.

A major limitation of our study was the retrospective data collection, and the lack of assessments of radiographic progression of the affected hips.

The results of this study substantiate the therapeutic potential for AALCO in improving clinical outcomes in terms of pain and functional activity, and reducing the risk of disease progression and the need for THA in patients with osteonecrosis. However, this study was limited by the small sample size and the retrospective data collection limiting the power of study for some subgroup comparisons. Further, clinical studies and long-term trials are warranted to confirm the findings of this study.

Authors acknowledge CBCC Global Research for providing medical writing and submission support funded by Regrow Biosciences Pvt. Ltd.

The authors report no conflicts of interest in this work.

1. Hernigou P, Poignard A, Zilber S, Rouard H. Cell therapy of hip osteonecrosis with autologous bone marrow grafting. Indian J Orthop. 2009;43(1):4045. doi:10.4103/0019-5413.45322

2. Gangji V, Hauzeur J-P, Matos C, De Maertelaer V, Toungouz M, Lambermont M. Treatment of osteonecrosis of the femoral head with implantation of autologous bone-marrow cells: a pilot study. J Bone Joint Surg Am. 2004;86(6):11531160. doi:10.2106/00004623-200406000-00006

3. Vardhan H, Tripathy SK, Sen RK, Aggarwal S, Goyal T. Epidemiological profile of femoral head osteonecrosis in the North Indian population. Indian J Orthop. 2018;52(2):140146. doi:10.4103/ortho.IJOrtho_292_16

4. Moya-Angeler J, Gianakos AL, Villa JC, Ni A, Lane JM. Current concepts on osteonecrosis of the femoral head. World J Orthop. 2015;6(8):590601. doi:10.5312/wjo.v6.i8.590

5. ISHKS registry. Available from: http://www.ishks.com/pdf/ISHKS-registry-2019.pdf. Accessed December 17, 2020. 2019.

6. Xie XH, Wang XL, Yang HL, Zhao DW, Qin L. Steroid-associated osteonecrosis: epidemiology, pathophysiology, animal model, prevention, and potential treatments (an overview). J Orthop Translat. 2015;3(2):5870. doi:10.1016/j.jot.2014.12.002

7. Jaffr C, Rochefort GY. Alcohol-induced Osteonecrosisdose and duration effects. Int J Exp Pathol. 2012;93(1):7879. doi:10.1111/j.1365-2613.2011.00798_1.x

8. Houdek MT, Wyles CC, Martin JR, Sierra RJ. Stem cell treatment for avascular necrosis of the femoral head: current perspectives. Stem Cells Cloning. 2014;7:6570. doi:10.2147/SCCAA.S36584

9. Xu S, Zhang L, Jin H, et al. Autologous stem cells combined core decompression for treatment of avascular necrosis of the femoral head: a systematic meta-analysis. Biomed Res Int. 2017;2017:6136205. doi:10.1155/2017/6136205

10. Hauzeur JP, De Maertelaer V, Baudoux E, Malaise M, Beguin Y, Gangji V. Inefficacy of autologous bone marrow concentrate in stage three osteonecrosis: a randomized controlled double-blind trial. Int Orthop. 2018;42(7):14291435. doi:10.1007/s00264-017-3650-8

11. Hauzeur JP, Toungouz M, Lechanteur C, et al. Autologous osteoblastic cells (PREOBy) versus concentrated bone marrow implantation in osteonecrosis of the femoral head: a randomized study. Revue de Chirurgie Orthopdique et Traumatologique. 2016;102(7):S73. doi:10.1016/j.rcot.2016.08.002

12. Cuende N, Rasko JEJ, Koh MB, Dominici M, Ikonomou L. Cell, tissue and gene products with marketing authorization in 2018 worldwide. Cytotherapy. 2018;20(11):14011413. doi:10.1016/j.jcyt.2018.09.010

13. Pepke W, Kasten P, Beckmann NA, Janicki P, Egermann M. Core decompression and autologous bone marrow concentrate for treatment of femoral head osteonecrosis: a randomized prospective study. Orthop Rev (Pavia). 2016;8(1):6162. doi:10.4081/or.2016.6162

14. Zhao D, Cui D, Wang B, et al. Treatment of early stage osteonecrosis of the femoral head with autologous implantation of bone marrow-derived and cultured mesenchymal stem cells. Bone. 2012;50(1):325330. doi:10.1016/j.bone.2011.11.002

15. Hernigou P, Habibi A, Bachir D, Galacteros F. The natural history of asymptomatic osteonecrosis of the femoral head in adults with sickle cell disease. J Bone Joint Surg Am. 2006;88(12):25652572. doi:10.2106/00004623-200612000-00002

16. Tomaru Y, Yoshioka T, Sugaya H, et al. Ten-year results of concentrated autologous bone marrow aspirate transplantation for osteonecrosis of the femoral head: a retrospective study. BMC Musculoskelet Disord. 2019;20(1):410. doi:10.1186/s12891-019-2797-4

17. Birmingham E, Niebur G, McHugh PE. Osteogenic differentiation of mesenchymal stem cells is regulated by osteocyte and osteoblast cells in a simplified bone niche. Eur Cell Mater. 2012;23:1327. doi:10.22203/eCM.v023a02

18. Prins H-J, Braat AK, Gawlitta D, et al. In vitro induction of alkaline phosphatase levels predicts in vivo bone forming capacity of human bone marrow stromal cells. Stem Cell Res. 2014;12(2):428440. doi:10.1016/j.scr.2013.12.001

19. Cui Q, Wang GJ, Balian G. Steroid-induced adipogenesis in a pluripotential cell line from bone marrow. J Bone Joint Surg Am. 1997;79(7):10541063. doi:10.2106/00004623-199707000-00012

20. Hernigou P, Beaujean F, Lambotte J. Decrease in the mesenchymal stem-cell pool in the proximal femur in corticosteroid-induced osteonecrosis. J Bone Joint Surg Br. 1999;81(2):349355. doi:10.1302/0301-620X.81B2.0810349

21. Sakaguchi M, Tanaka T, Fukushima W, Kubo T, Hirota Y. Impact of oral corticosteroid use for idiopathic osteonecrosis of the femoral head: a nationwide multicenter case-control study in Japan. J Orthop Sci. 2010;15(2):185191. doi:10.1007/s00776-009-1439-3

22. Kubo T, Ueshima K, Saito M, Ishida M, Arai Y, Fujiwara H. Clinical and basic research on steroid-induced osteonecrosis of the femoral head in Japan. J Orthop Sci. 2016;21(4):407413. doi:10.1016/j.jos.2016.03.008

23. Cooper C, Steinbuch M, Stevenson R, Miday R, Watts N. The epidemiology of osteonecrosis: findings from the GPRD and THIN databases in the UK. Osteoporos Int. 2010;21(4):569577. doi:10.1007/s00198-009-1003-1

24. Fukushima W, Fujioka M, Kubo T, Tamakoshi A, Nagai M, Hirota Y. Nationwide epidemiologic survey of idiopathic osteonecrosis of the femoral head. Clin Orthop Relat Res. 2010;468(10):27152724. doi:10.1007/s11999-010-1292-x

25. Kang JS, Park S, Song JH, Jung YY, Cho MR, Rhyu KH. Prevalence of osteonecrosis of the femoral head: a nationwide epidemiologic analysis in Korea. J Arthroplasty. 2009;24(8):11781183. doi:10.1016/j.arth.2009.05.022

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[Full text] Retrospective Study on Implantation of Autologous-Cultured Osteoblasts | ORR - Dove Medical Press

Orthopedic Regenerative Medicine Market Size to Witness A Lucrative Growth Over 2020-2027 | Curasan, Inc., Carmell Therapeutics Corporation, Anika…

The Report Titled on Orthopedic Regenerative Medicine Market which provides COVID19 Impact analysis on Market Size (Production, Capacity, Value, Values & Consumption), Regional and Country-Level Market Size, Segmentation Market Growth, Market Share, Competitive Landscape, Sales Analysis, Impact of Domestic and Market Players. Orthopedic Regenerative Medicine Market detailed study of historical and present/future market data. Economic growth, GDP (Gross Domestic Product), and inflation are some of the elements included in this report to offer crystal clear picture of the Orthopedic Regenerative Medicine industry at global level.

Orthopedic Regenerative Medicine Market competitive landscapes provides details by topmost manufactures like (Curasan, Inc., Carmell Therapeutics Corporation, Anika Therapeutics, Inc., Conatus Pharmaceuticals Inc., Histogen Inc., Royal Biologics, Ortho Regenerative Technologies, Inc., Swiss Biomed Orthopaedics AG, Osiris Therapeutics, Inc., and Octane Medical Inc.), including Capacity, Production, Price, Revenue, Cost, Gross, Gross Margin, Growth Rate, Import, Export, Market Share and Technological Developments.

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Market Segmentation & Coverage:

This research report categorizes the Orthopedic Regenerative Medicine to forecast the revenues and analyze the trends in each of the following sub-markets:

By Procedure Cell TherapyTissue EngineeringBy Cell TypeInduced Pluripotent Stem Cells (iPSCs)Adult Stem CellsTissue Specific Progenitor Stem Cells (TSPSCs),Mesenchymal Stem Cells (MSCs)Umbilical Cord Stem Cells (UCSCs)Bone Marrow Stem Cells (BMSCs)By SourceBone MarrowUmbilical Cord BloodAdipose TissueAllograftsAmniotic FluidBy ApplicationsTendons RepairCartilage RepairBone RepairLigament RepairSpine RepairOthers

Based on Geography, the Orthopedic Regenerative Medicine Market studied across Americas, Asia-Pacific, and Europe, Middle East & Africa. The Americas region surveyed across Argentina, Brazil, Canada, Mexico, and United States. The Asia-Pacific region surveyed across Australia, China, India, Indonesia, Japan, Malaysia, Philippines, South Korea, and Thailand. The Europe, Middle East & Africa region surveyed across France, Germany, Italy, Netherlands, Qatar, Russia, Saudi Arabia, South Africa, Spain, United Arab Emirates, and United Kingdom.

Impact of COVID-19 on this Market:

The pandemic of COVID-19 continues to expand and impact over 175 countries and territories. Although the outbreak appears to have slowed in China, COVID-19 has impacted globally. The pandemic could affect three main aspects of the global economy: production, supply chain, and firms and financial markets. National governments have announced largely uncoordinated, country-specific responses to the virus. As authorities encourage social distancing and consumers stay indoors, several businesses are hit. However, coherent, coordinated, and credible policy responses are expected to offer the best chance at limiting the economic fallout.

We, at Coherent Market Insights, understand the economic impact on various sectors and markets. Using our holistic market research methodology, we are focused on aiding your business sustain and grow during COVID-19 pandemics. With deep expertise across various industries-no matter how large or small- and with a team of highly experienced and dedicated analysts, Coherent Market Insights will offer you an impact analysis of coronavirus outbreak across industries to help you prepare for the future.

The Orthopedic Regenerative Medicine Market on the basis of Business Strategy (Business Growth, Industry Coverage, Financial Viability, and Channel Support) and Product Satisfaction (Value for Money, Ease of Use, Product Features, and Customer Support) that aids businesses in better decision making and understanding the competitive landscape.

Competitive Strategic Window:

Competitive Strategic Window analyses the competitive landscape in terms of markets, applications, and geographies. Competitive Strategic Window helps the vendor define an alignment or fit between their capabilities and opportunities for future growth prospects. During a forecast period, it defines the optimal or favorable fit for the vendors to adopt successive merger and acquisition strategies, geography expansion, research & development, and new product introduction strategies to execute further business expansion and growth.

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The report provides insights on the following pointers:

Market Penetration: Provides comprehensive information on the market offered by the key players

Market Development: Provides in-depth information about lucrative emerging markets and analyzes the markets

Market Diversification: Provides detailed information about new product launches, untapped geographies, recent developments, and investments

Competitive Assessment & Intelligence: Provides an exhaustive assessment of market shares, strategies, products, and manufacturing capabilities of the leading players

Product Development & Innovation: Provides intelligent insights on future technologies, R&D activities, and new product developments

The report answers questions such as:

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What is the competitive strategic window for opportunities in the Global Orthopedic Regenerative Medicine Market?

What are the technology trends and regulatory frameworks in the Global Orthopedic Regenerative Medicine Market?

What are the modes and strategic moves considered suitable for entering the Global Orthopedic Regenerative Medicine Market?

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World Cancer Day 2021: Know All About The Different Types Of Blood Cancer From Expert – NDTV Doctor

World Cancer Day: Blood cancer can be managed with treatments such as chemotherapy, radiation therapy

World Cancer Day 2021: This day is observed on February 4. Blood cancer originates in the blood forming tissues when abnormal blood cells start growing out of control, thereby interrupting the functioning of the normal blood cells. The normal blood cells help strengthen the immune system by fighting infection and producing new blood cells. Most blood cancers begin in the bone marrow where blood is produced. The three most common blood cancers are lymphoma, leukaemia and multiple myeloma. The common symptoms include weakness, shortness of breath, minimal injury resulting in fractures, excessive or easy bruising, bleeding gums, recurrent infections and frequent vomiting sensations. Blood cancer can be managed with treatments such as chemotherapy, radiation therapy and stem cell transplant.

Multiple myeloma

Multiple Myeloma develops in the bone marrow and affects plasma cells of the body. Plasma cells are responsible for producing antibodies that attack infections and diseases. When these cells become cancerous, they collect in the bone marrow and weaken the bones, causing pain on movement. They also produce antibodies that are useless and make the body weaker. Some common symptoms for multiple myeloma include low blood count, high calcium levels, kidney problems and spinal cord compression due to weakened bones.

Also read:Cervical Cancer During Pregnancy: Here's All You Need To Know

Lymphoma

Lymphoma affects the lymphatic system, which is responsible for getting rid of toxins in the body. When the immune cells, or lymphocytes, grow out of control, they collect in the lymph nodes, spleen and in other tissues, and organs. The main types are Hodgkins and non-Hodgkin lymphoma. Some common symptoms for lymphomas include painful swelling in the neck, groin, and armpits, fever and drenching sweats, fatigue, unexplained weight loss and shortness of breath.

Leukaemia

Leukaemia is cancer in the bone marrow that gradually spreads to the bloodstream. It is the most common cause of death due to cancer in India. In Leukaemia, the bone marrow produces metamorphosed cells, that outgrow the healthy blood cells gradually. There are multiple forms of leukaemia, but the diagnosis is determined based on speed of symptom development and the type of blood cells that accumulate. Some common symptoms for leukaemia include severe and frequent infections, recurrent nosebleeds, tiny red spots on the skin and excessive sweating and pain in the bones and joints.

While lymphomas and leukaemia affect both children and adults, Myeloma is more prevalent among adults.

Also read:What To Do When A Cancer Patient Tests Positive For COVID-19?

There are several therapies that can be used for treating the different kinds of blood cancer such as:

While there have been developments and advancement in therapies and treatments available for cancer, a significant portion of the future cancer burden can be prevented if we take necessary precautionary measures in the early stages. Better control on tobacco sale and consumption, dietary changes, expansion and equitable distribution of medical facilities, awareness about education programs and risks, prevention, and knowing the benefits of bone marrow donation can go a long way in reducing the burden of blood cancer.

Also read:Alarming Cancer Symptoms Men Should Not Ignore

(Dr Nitin Sood, Director, Hemato Oncology and Stem Cell Transplant Medical and Haemato Oncology, Cancer Institute, Medanta)

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

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World Cancer Day 2021: Know All About The Different Types Of Blood Cancer From Expert - NDTV Doctor

Bone Marrow Transplantation Market to Surpass US$ 15 Bn By 2027: Acumen Research And Consulting – IT News Online

GlobeNewswire2021-02-01

Acumen Research and Consulting, a global provider of market research studies, in a recently published report titledBone Marrow Transplantation Market Global Industry Analysis, Market Size, Opportunities and Forecast, 2020-2027

LOS ANGELES, Feb. 01, 2021 (GLOBE NEWSWIRE) -- The Global Bone Marrow Transplantation Market size is projected to reach US$ 15 Bn by 2027, with CAGR of 5.7% during 2020-2027

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Disease, cancer, or chemotherapy may damage or kill the bone marrow. A surgical procedure is known as bone marrow transplantation, which replaces compromised or lost bone marrow. Stem cells are collected, processed, frozen and stored in a patient's autologous transplant and then returned to the patient after intensive therapy. Stem cells are obtained from an appropriate donor in an allogeneic transplant and transplanted to the recipient to cure the disease and rebuild the immune system of the recipient.

The growing prevalence of cancer and anemia is the leading catalyst for the growth of global demand for bone marrow transplantation. In turn, global demand for bone marrow transplantation will also expand with advances in technology, enhanced health services, recent evidence of cardiac and neuronal bone marrow transplantation, expanded logistic treatment development and increased per-capita health spending. However, high treatment costs, shortage of bone marrow donors and payment instability in many countries remain a major barrier to the global demand for bone marrow transplantation.

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Cell therapy and tissue engineering products for commercialization

The growing demand for bone marrow transplantation is expected to expand in the industry with several organizations involved in clinical trials for cell therapy and tissue engineering products worldwide. Many of the companies have grown from R&D to full market firms. Over the last five years, some have experienced over 30% growth. Stem cell graft is one of the fastest growing cell therapies on the market, estimated at USD 510 million by the end of 2015.

Small Bone Marrow Repayment and Billing Laps

In developed regions, the inability to pay for organ transplantation techniques in tandem with cost-effective bone marrow transplantation has contributed to the slower implementation of the bone marrow transplantation method, particularly in developing regions. Approximately 30% of the world's people consider their bone transplantation to be financially secure.

The CMS payment scheme, which returns significantly low compared to real bone marrow transplantation prices, underlines the limited scope of drugs. The amount of government interest is 47% higher than the actual cost of the operation. This adds up to nearly US$ 40,000 in transplant costs in US medical transplant hospitals.

The increase in allogeneic demand for bone marrow transplants is driven by a lower incidence of disease recurrence and a strong increase in eligible donors.

Bone marrow transplantation with allogeneic substances is currently leading the bone marrow transplantation type. Demand for allogeneic bone marrow transplantation is driven by large increases in healthy patients and a reduced risk of disease recurrence. The market will soon be dominated by autologous bone marrow transplant due to the increasing number of stem cell banks that store healthy cells in patients and the development of healthy blood cells in patients after treatment with conditioning.

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The lymphoma indicator is behind a significant uptick in new cases of Hodgkin and increased survival levels for post-bone marrow transplantation.

The bone marrow transplantation industry is mainly in the lymphoma group. This consists of 2 types of Hodgkin and Non-Hodgkin lymphoma. 83,180 new cases of lymphoma were registered by the National Cancer Center (NCI) in 2018. Lymphoma progression is driven by a large increase in lymphoma patients and a higher survival rate after bone marrow transplantation. Throughout 2018, 60,300 new cases of leukemia have been reported by the National Cancer Institute, while leukemia is irregular in the development of white blood cells in the bone marrow.

Regional Stance

Europe held significant market share in 2019 in bone marrow transplantation owing to the high number of bone marrow transplants and the expansion of bone marrow registries. With strong potential for treatment, Latin America is expected to see a significant increase in the number of bone marrow transplants.

Key Players & Strategies

Participants include major global Agendia, Sanofi, Diadexus Inc., Abbott, Qiagen N.V., and Others.

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Bone Marrow Transplantation Market to Surpass US$ 15 Bn By 2027: Acumen Research And Consulting - IT News Online

Stem Cell Therapy Market 2021: Global Key Players, Trends, Share, Industry Size, Segmentation, Forecast To 2027 KSU | The Sentinel Newspaper – KSU |…

Stem Cell Therapy Market is valued at USD 9.32 Billion in 2018 and expected to reach USD 16.51 Billion by 2025 with the CAGR of 8.5% over the forecast period.

Rising prevalence of chronic diseases, increasing spend on research & development and increasing collaboration between industry and academia driving the growth of stem cell therapy market.

Scope of Stem Cell Therapy Market-

Stem cells therapy also known as regenerative medicine therapy, stem-cell therapy is the use of stem cells to prevent or treat the condition or disease. Stem cell are the special type of cells those differentiated from other type of cell into two defining characteristics including the ability to differentiate into a specialized adult cell type and perpetual self-renewal. Under the appropriate conditions in the body or a laboratory stem cells are capable to build every tissue called daughter cells in the human body; hence these cells have great potential for future therapeutic uses in tissue regeneration and repair. Among stem cell pluripotent are the type of cell that can become any cell in the adult body, and multipotent type of cell are restricted to becoming a more limited population of cells.

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The stem cell therapy has been used to treat people with conditions including leukemia and lymphoma, however this is the only form of stem-cell therapy which is widely practiced. Prochymal are another stem-cell therapy was conditionally approved in Canada in 2012 for the treatment of acute graft-vs-host disease in children those are not responding to steroids. Nevertheless, hematopoietic stem cell transplantation is the only established therapy using stem cells. This therapy involves the bone marrow transplantation.

Stem cell therapy market report is segmented based on type, therapeutic application, cell source and by regional & country level. Based upon type, stem cell therapy market is classified into allogeneic stem cell therapy market and autologous market.

Stem Cell Therapy Companies:

Stem cell therapy market report covers prominent players like,

Based upon therapeutic application, stem cell therapy market is classified into musculoskeletal disorders, wounds and injuries, cardiovascular diseases, surgeries, gastrointestinal diseases and other applications. Based upon cell source, stem cell therapy market is classified into adipose tissue-derived mesenchymal stem cells, bone marrow-derived mesenchymal stem cells, cord blood/embryonic stem cells and other cell sources

The regions covered in this stem cell therapy market report are North America, Europe, Asia-Pacific and Rest of the World. On the basis of country level, market of stem cell therapy is sub divided into U.S., Mexico, Canada, U.K., France, Germany, Italy, China, Japan, India, South East Asia, GCC, Africa, etc.

Stem Cell Therapy Market Segmentation

By Type

Allogeneic Stem Cell Therapy Market, By Application

Autologous Market, By Application

By Therapeutic Application

By Cell Source

Stem Cell Therapy Market Dynamics

Rising spend on research and development activities in the research institutes and biotech industries driving the growth of the stem cell therapy market during the forecast period. For instance, in January 2010, U. S. based Augusta University initiated Phase I clinical trial to evaluate the safety and effectiveness of a single, autologous cord blood stem infusion for treatment of cerebral palsy in children. The study is estimated to complete in July 2020. Additionally, increasing prevalence of chronic diseases creating the demand of stem cell therapy. For instance, as per the international diabetes federation, in 2019, around 463 million population across the world were living with diabetes; by 2045 it is expected to rise around 700 million. Among all 79% of population with diabetes were living in low- and middle-income countries. These all factors are fuelling the growth of market over the forecast period. On the other flip, probabilities of getting success is less in the therapeutics by stem cell may restrain the growth of market. Nevertheless, Advancement of technologies and government initiative to encourage research in stem cell therapy expected to create lucrative opportunity in stem cell therapy market over the forecast period.

Stem Cell Therapy Market Regional Analysis

North America is dominating the stem cell therapy market due increasing adoption rate of novel stem cell therapies fueling the growth of market in the region. Additionally, favorable government initiatives have encouraging the regional market growth. For instance, government of Canada has initiated Strategic Innovation Fund Program, in which gov will invests in research activities carried out for stem cell therapies. In addition, good reimbursing scheme in the region helping patient to spend more on health. Above mentioned factors are expected to drive the North America over the forecast period.

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