StemCell Therapy

LentiGlobin, Bluebird Bios investigational gene therapy for sickle cell disease (SCD), continues to show promising results in SCD patients participating in the companys Phase 1/2 HGB-206 clinical trial, according to the latest study data.

The new findings which included data from additional patients treated in the trial, updated data from those previously reported, and exploratory analyses were presented at the 61st American Society of Hematology (ASH) Annual Meeting and Exposition, held Dec. 6-10 in Orlando, Fla.

LentiGlobinisa gene therapy that has been developed to increase the levels of hemoglobin the protein that transports oxygen in the blood in people with SCD.

The therapy works by delivering functional copies of a modified form of the beta-globin gene (A-T87Q-globin gene) into patients red blood cell precursors, known as hematopoietic stem cells, or HSCs. Once these precursors differentiate, their red blood cells start producing a modified version of hemoglobin, called HbAT87Q.

By boosting the production of this anti-sickling form of the protein, LentiGlobin reduces the proportion of defective hemoglobin in patients red blood cells. That, in turn, reduces the sickling and destruction of these red blood cells and other complications associated with SCD.

The safety and efficacy of LentiGlobin is currently being evaluated in three groups identified as A-C of SCD patients participating in Bluebirds ongoing open-label, Phase 1/2 HGB-206 trial (NCT02140554).

Those in group A were treated per the original trial protocol. Meanwhile, those in groups B and C received an enhanced treatment protocol, approved in 2016, that is designed to increase the therapys efficiency. In groups A and B, patients HSCs were extracted from the bone marrow, while in group C, they were extracted from the blood.

As of the data cutoff date of August 26, 2019, seven participants in group A, two in group B, and 17 in group C had been treated with LentiGlobin. According to new data presented at the meeting, only two patients from group A required regular blood transfusions after the treatment.

In addition, the updated findings revealed that the levels of anti-sickling HbAT87Q remained stable in all participants from groups A and B over a post-treatment follow-up period of three years. Similarly, levels of total hemoglobin also were found to have remained stable in both patient groups over a two-year follow-up.

At the trial participants last visit, the median levels of anti-sickling HbAT87Q were 0.9 g/dL among those from group A, and 3.6 g/dL and 7.1 g/dL in the two patients from group B. The median levels of total hemoglobin were 9.0 g/dL among patients from group A, and 11.3 g/dL and 13.0 g/dL among those from group B.

Normal levels of hemoglobin in the blood range from 12.5 to 17.5 g/dL.

Among 12 patients from group C who were followed for at least six months, the median levels of anti-sickling HbAT87Q made up at least 40% of their total hemoglobin. At their last visit, the levels of anti-sickling HbAT87Q ranged from 2.7 to 9.0 g/dL, and the levels of total hemoglobin from 9.3 to 15.2 g/dL.

In groups A and B, LentiGlobin reduced the frequency of painful vaso-occlusive crises (VOCs) and acute chest syndrome (ACS) in the two years following treatment.

Nine patients from group C who were followed for at least six months had experienced four or more VOCs or ACS episodes in the two years prior to receiving LentiGlobin. Treatment with the gene therapy led to a reduction of 99% in the frequency of annual VOCs and ACS. In this group, there were no reports of ACS or severe VOCs for up to 21 months following treatment.

Moreover, among those from group C, LentiGlobin reduced the levels of different markers of red blood cells destruction, including reticulocytes, lactate dehydrogenase (LDH), and bilirubin.

LentiGlobins safety profile was consistent with previous data. No serious adverse events related to treatment were reported during the study. Only one mild, non-serious event of hot flush was found to be related to LentiGlobin. That event was rapidly resolved and did not require treatment.

Exploratory analyses were performed in a sub-group of patients from all three groups. In 12 participants who had been followed for at least six months, more than 70% of the individuals red blood cells were found to contain the anti-sickling HbAT87Q at the last study visit, these analyses showed. Moreover, in four of these patients, nearly all their red blood cells (90%) were positive for HbAT87Q.

In addition, exploratory analyses revealed that participants red blood cells were less prone to sickling following treatment with LentiGlobin.

At ASH, the growing body of data from our clinical studies of LentiGlobin for SCD reflects results from 26 treated patients with up to four years of follow-up, David Davidson, MD, Bluebird Bios chief medical officer, said in a press release.

We continue to observe patients treated in Group C producing high levels of gene-therapy derived anti-sickling hemoglobin, HbAT87Q, accounting for at least 40% of total hemoglobin in those with six or more months of follow-up, and exploratory assays show that HbAT87Q is present in most red blood cells of treated patients, Davidson said.

The robust production of HbAT87Q was associated with substantial reductions of sickle hemoglobin, HbS, as well as improvement in key markers of hemolysis [red blood cells destruction]. Most importantly, patients in Group C have not experienced any episodes of acute chest syndrome or serious vaso-occlusive crises following LentiGlobin for SCD treatment, he added.

The company is recruiting participants with transfusion-dependent -thalassemia (TDT) for a Phase 3 trial (NCT03207009) testing LentiGlobin. Moreover, according to the companys pipeline, there is a Phase 2/3 trial planned in sickle cell disease for this gene therapy.

Joana is currently completing her PhD in Biomedicine and Clinical Research at Universidade de Lisboa. She also holds a BSc in Biology and an MSc in Evolutionary and Developmental Biology from Universidade de Lisboa. Her work has been focused on the impact of non-canonical Wnt signaling in the collective behavior of endothelial cells cells that make up the lining of blood vessels found in the umbilical cord of newborns.

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

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LentiGlobin Gene Therapy Continues to Show Promising Results in SCD, Updated Trial Data Shows - Sickle Cell Anemia News

Autologous stem cell therapy market segment is anticipated to witness around 10% growth throughout the forecast timeframe. Autologous stem cell therapy has high compatibility with patients immune system and showcases strong efficacy. Additionally, it eliminates the need for finding donor and reduces the overall cost of treatment. Aforementioned advantages offered by autologous stem cell therapy will augment its demand thereby, escalating segment growth.

Oncology segment held over 37% revenue in 2018. Increasing prevalence of cancer globally will increase the demand for advanced stem cell Therapy. Metastatic cancer cells are difficult to destroy utilizing conventional methods. Radiations and chemotherapy cannot eradicate the disease completely and also results in several complications. Stem cells have unique properties such as secretion of bioactive factors and migration towards cancer cells promotes tumor targeting that are proven to be effective in treatment of cancer. Aforementioned factors will escalate the segmental growth.Rising government initiatives for promoting stem cell therapy in developed as well as developing countries will propel industry growth. Increasing government spending on research activities aimed at development of stem cell therapy for treatment of life-threatening diseases such as cancer. The government ensures that laboratories, research and academic centers are well-equipped with necessary equipment and advanced devices for facilitating the stem cell therapy research processes. Aforementioned factors are expected to boost the stem cell therapy market growth.

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Increasing efforts undertaken by companies for improving efficacy of stem cell therapy will fuel industry growth. Stem cells possess totipotency that enables them to transform into any differentiated cell. Stem cells play pivotal role in treatment of chronic diseases such as cancer, cardiovascular diseases and degenerative disorders. 0Currently, hematopoietic, embryonic and mesenchymal cells are being researched as they have the ability to cure several complications. Companies commercialize these stem cell products for providing effective therapy that should prove beneficial for the industry growth. However, ethical issues associated with stem cell therapy may affect industry growth to some extent.

Hospitals segment was valued around USD 4 billion in 2018. Significant growth is attributed to increasing prevalence of chronic diseases. Annually, large number of patients prefer stem cell therapy offered at hospitals as a treatment option to avoid complications caused due to invasive methods. Hospitals have dedicated resources allocated for providing uncompromised care to patients that speeds up the recovery process. Moreover, hospitals affiliated with government authorities receives enough funding and have the ability to provide superior quality stem cell therapy. Above mentioned factors will augment patients preference towards hospitals that will exceed segment growth.

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Asia Pacific stem cell therapy market held around 20% revenue in 2018. Substantial revenue share can be attributed to the increasing awareness regarding availability of stem cell therapies. Moreover, government encourages scientists and researchers to carry out studies and thesis on stem cell therapies in order to develop innovative solutions for treating chronic diseases. Also, several developing countries in the region have witnessed industry growth opportunities as government provide funds to establish stem cell centers. For instance, in Singapore JTC Corporation has established Biopolis, that is a biomedical research center of stem cell science. Such government initiatives in Asian countries will positively impact regional market growth.

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Prominent industry players operational in stem cell therapy market are Astellas Pharma Inc, Cellectis, Celyad, Novadip Biosciences, Gamida Cell, Capricor Therapeutics, Cellular Dynamics, CESCA Therapeutics, DiscGenics, OxStem, Mesoblast Ltd, ReNeuron Group and Takeda Pharmaceuticals. Industry players implement various strategies such as collaboration, acquisition, merger, product launch to sustain in the market. For instance, in February 2018, Astellas acquired Universal Cells so that they can utilize proprietary technology to manufacture pluripotent stem cells. This strategy will help Astellas to develop innovative solutions that will provide competitive advantage to company.

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Global Stem Cell Therapy Market 2019-2025 Analyzed by Business Growth, Development Factors, Applications, and Future Prospects - Technology Magazine

A highly effective vaccine to prevent hepatitis B virus (HBV) infection has been available for nearly 40 years, yet millions of people worldwide continue to become infected with the liver-attacking virus and more than 600,000 die from its complications each year, according to the U.S. Centers for Disease Control and Prevention. A new strategic plan from the National Institutes of Health outlines efforts to intensify ongoing HBV research with the goals of developing a cure and improving scientific understanding of the virus while creating improved strategies for screening and treating patients.

HBV is transmitted through sex, contact with infected blood or bodily fluids, or from an infected mother to her baby, and can result in either an acute infection that resolves or a chronic infection. Chronic HBV infection can lead to serious health issues including cirrhosis (scarring of the liver), liver failure or liver cancer. Approximately 80% to 90% of infants infected during the first year of life will develop chronic infection, while only 5% of people infected as adults will become chronically infected. Between 850,000 and 2 million people in the United States have chronic hepatitis B; globally, that number is 257 million, according to the World Health Organization.

Although treatments are available to control HBV infection, they must be taken for years if not for life. Additionally, high medication costs, the need for continuous disease monitoring and adhering to treatment regimens present significant burdens for people with chronic hepatitis B. Even among those treated for chronic hepatitis B, the risk of developing cirrhosis and liver cancer remains elevated.

Scientific discoveries within the past decade suggest that a hepatitis B cure is possible. The new NIH plan defines a hepatitis B cure as a sustained loss of a specific protein on the surface of HBV called hepatitis B virus surface antigen preferably with antibodies against the antigen and undetectable viral DNA after completion of a finite course of treatment. Ideally, a hepatitis B cure would also reduce a persons risk of liver failure and liver cancer. To effectively address the global public health challenge posed by HBV, a curative treatment will need to complement better approaches to screening, follow-up care, and vaccination coverage.

The Strategic Plan for Trans-NIH Research to Cure Hepatitis B focuses on three key research areas. The first priority calls for a better understanding of hepatitis B biology, including the viral and host factors that lead to disease, immunity, reactivation and transmission, as well as the impact of co-infections with other hepatitis viruses and HIV. An improved understanding of these factors and the HBV lifecycle is essential to developing a cure.

The second priority emphasizes the development and sharing of tools and resources to support fundamental research and product development. This includes standardizing and sharing reagents and assays; improving and creating new animal models to study the progression of human liver disease and mother-to-child transmission; and establishing biomarkers for disease progression and response to therapy.

The third priority calls for the creation of strategies to cure and prevent hepatitis B infection. A cure could include approaches to blocking viral replication, stimulating the anti-HBV immune responses or eliminating HBV-infected cells. However, achieving a cure requires strengthening existing public health efforts for promoting hepatitis screening, ensuring that high-risk and underserved populations have access to vaccination, and prioritizing follow-up to care and adherence to treatment, according to the plan.

The strategic plan builds on NIHs ongoing hepatitis B research portfolio and the U.S. National Viral Hepatitis Action Plan. NIH sought input from academia, patient advocacy organizations, private and nonprofit companies, government organizations, and clinical trial networks funded by the NIH in the development of this strategic plan.

Reference

Ortinau et al. (2019) Identification of Functionally Distinct Mx1+SMA+ Periosteal Skeletal Stem Cells. Cell Stem Cell. DOI: https://doi.org/10.1016/j.stem.2019.11.003

This article has been republished from the following materials. Note: material may have been edited for length and content. For further information, please contact the cited source.

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Strategic Plan To Cure Hepatitis B in the Making - Technology Networks

We can already edit genes in human embryos. We can even do it in a way to pass the edits down generations, fundamentally changing a familys genetic makeup.

Doing it well, however, is far more difficult.

Its impossible to talk about human germline genome editing without bringing up the CRISPR baby fiasco. Over a year ago, a rogue Chinese scientist performed an edit on fertilized human embryos that, in theory, makes them resistant to HIV infection. Two twin girls were born, and both had multiple unplanned edits in their genome with unknown health consequencesconsequences that may be passed on to their offspring.

The brash attempt at making scientific history clearly shows that, ethics and morality issues aside, when it comes to germline editingthat is, performing gene edits in egg, sperm, or the embryowere simply technologically not there. Make no mistake: CRISPR may one day wipe out devastating genetic diseases throughout entire family lines, or even the human race. But to harness its power responsibly, there are plenty of technical challenges we need to master first.

This week, Rebecca Lea and Dr. Kathy Niakan at the Human Embryo and Stem Cell Laboratory at the Francis Crick Institute in London, UK, laid out those challenges in a sweeping articlein Nature. CRISPR as a gene editor is getting more specific and efficient by the day, they explained. However, for it to gradually move into germline editing, we also need to understand how the tool tangos with cells during early human development.

The data, they argue, will not only let us zoom into the creation of human life. It will also help inform the debate about potential safe and effective clinical uses of this technology, and truly unlock the doors to the human genome for good.

Correcting dangerous genetic mutations is one reason to pursue germline editing, but CRISPRing human embryos can also unveil insights into the very first stages of human embryo development. Research shows that trying to understand how human embryos form by studying mice might not be the best route, especially when it comes to using those results to tackle infertility and other medical problems. With CRISPR, we have insight into these early stages that were previously completely unattainable. We might only solve infertility issues, but perhaps also allow same-sex couples to have genetic children in the future.

Another argument is that couples already screen for life-threatening mutations during IVF, and using CRISPR on top of that is unnecessary. Not true, the authors argued. When both parents carry a similar mutation that robs them of the ability to have a healthy child, CRISPRnot selection during IVFis the answer. Ultimately, providing more options for patients empowers them to make the choice that is best for their family and circumstances, they said.

This is where it gets complicated.

The big one: were still trying to tease out how CRISPR works in cells that form the embryo, in hopes that we can cut down on potential mistakes.

Let me explain: all cells in the body have a cell cycle, somewhat analogous to a persons life cycle. Many checkpoint life events happen along the way. The cell could decide to divide and have kids, so to speak, or temporarily halt its cycle and stop its own aging. During a cycle, the cells DNA dramatically changes in number and packaging in preparation for its next stage in life.

The problem? The way CRISPR works heavily depends on the cell cycle. Although dubbed an editor, CRISPR actually vandalizes the genome, creating breaks in the DNA strands. What we call gene editing is the cells DNA repair system kicking into high gear, trying to patch up the mess CRISPR left behind. Adult cells that cant be repaired stop their own life cycle at a checkpoint for the greater good. In embryos, however, cells arent nearly as altruistic. Their checkpoints arent fully developed, so they might continue to develop even with severe mutations. Zooming back to the full picture, it means that the resulting early-stage embryo may keep accumulating damage, until it fails in the mothers womb.

To get around this, scientists have tried other ways to push an embryo into accepting a healthy DNA template after a CRISPR snip, which in theory would cut down on unwanted mutations. One idea is injecting the CRISPR machinery at a specific time into fertilized eggs, so it catches the early-stage embryo at just the right time to reduce DNA breaks in both strands. While theoretically possible, the process is kind of like a person trying to jump from a high-speed train into a specific cabin on a rapidly rotating Ferris wheel while blindfolded.

But science is making progress. Although we dont have a detailed movie of cell cycles in human embryos yet, multiple labs are beginning to piece one together, with hopes itll eventually help take off the blindfold when injecting CRISPR. Others are looking into adding CRISPR to sperm before fertilization as an alternative.

At the same time, scientists are also trying to characterize the entire scope of mutations caused by CRISPR. Its not just adding, swapping, or deleting specific letters in genes. Rather, the range of mutations is more complex, including large swaths of genetic rearrangements, unintended cuts relatively far from targeted spots, and other dramatic DNA lesions following CRISPR action. Its perhaps not surprising that the edits in CRISPR babies didnt work as intended.

Base editors, which swap one genetic letter for another, might be a better approach compared to the classic hack-and-paste, the authors said. So far, however, the tools havent yet been validated in embryosnot even those from mice.

Finally, for the edit to make a difference to the child, the embryo has to develop normally inside a womb into a baby. But success rates for assisted reproductive technologies are already fairly low. Add in a dose of genetic editing tool that cuts into an already-sensitive genomic landscape, and it becomes incredibly hard to maintain the health of the edited embryo.

Putting it all together, there is simply not enough data at present to understand the capability of early[embryos] to repair DNA, the authors said.

Far from it. Although theres much we dont yet understand, we do have an impressive range of tools to predict and evaluate mutations in human embryos. Exactly how to determine whether a gene-edited embryo is healthy remains up for debatefor example, is five unexpected mutations considered ok? What about 500 or 5,000?

That said, just having tools to diagnose the genetic health of an embryo from a tiny bit of DNA is already extremely useful, especially if we as a society decide to move into germline editing as a treatment.

With machine learning making an ever-larger splash in computational biology, these predictive tools will only become more accurate. Add to that ever-more-effective CRISPR variations, and were on the right trackas long as any potential applications of embryo editing only come after in-depth public and policy discussions and fit a number of strict ethical and safety criteria, the authors said.

In response to the CRISPR baby scandal, multiple governments and the World Health Organization have all drafted new guidelines or legislation to tap on the brakes. The technology isnt mature enough for clinical use, the authors said, and much more work is needednot just to further improve CRISPR tools, but especially for understanding how it works in human embryos.

Ultimately, were talking about potentially engineering the future of the human race. Tiptoeing, rather than stumbling ahead, is the least we can do. One must ensure that the outcome will be the birth of healthy, disease-free children, without any potential long-term complications, the authors concluded.

Image Credit: Image by marian anbu juwan from Pixabay

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How Far Are We from (Accurately and Safely) Editing Human Embryos? - Singularity Hub

Myelodysplastic syndrome (MDS) is a blood disorder caused due to the production of abnormal blood cells in bone marrow. Bone marrow failure leads to drop in the number of healthy blood cells in the body. In MDS, bone marrow does not produce healthy red blood cells, white blood cells, and/or platelets. Symptoms of MDS in the beginning are no specific than causes of pancytopenia i.e., deficiency of RBC, WBC and platelets. Therefore, the final diagnosis of MDS is done after examination of the cells of bone marrow.

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Bone marrow sample is taken from inside of a bone (usually the hip bone) and examined with a microscope using special stains to look for abnormal and immature cells. According to the American Cancer Society, over 13,000 new cases of MDS occur in the U.S. each year. According to the National Organization for Rare Disorders, 30% of patients with MDS can develop a form of blood cancer known as acute myeloid leukemia. Majority of patients diagnosed with MDS are aged between 65 and 70; however, MDS can affect people of any age and the risk of developing MDS increases with age.

The global myelodysplastic syndrome treatment market is driven by rise in the global geriatric population as MDS is most commonly found in this population and remains an incurable disease. Moreover, significant progress has been made in the diagnosis of MDS with the help of sequencing technologies. Chromosomal abnormalities can be identified in MDS patients with these technologies. These are useful for both diagnosis and prognosis in MDS patients. For instance, patients with chromosome 5q deletions are more likely to respond to lenalidomide. No new drugs have been approved for MDS by the U.S. Food and Drug Administration since 2006. Current available therapies can be efficacious, but are generally not curative. Some of the challenges in developing new treatments are the complexity and heterogeneity of MDS as a disease.

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The global myelodysplastic syndrome treatment market can be segmented based on type of treatment and region. In terms of type of treatment, the market can be classified into supportive therapy, growth factors, chemotherapy (including hypomethylating agents), and stem cell transplant. Cytarabine, azacitidine, decitabine, and lenalidomide are the major drugs used during chemotherapy.

Stem cell transplant is the only cure for MDS; however, majority of patients are not treated with stem cell transplant due to various factors such as high treatment cost, transplant-related deaths, and relapse rate at five years (as high as 40%). The chemotherapy segment is expected to hold major share of the global myelodysplastic syndrome treatment market due to larger application and less complications than other therapies such as stem cell transplant.

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Geographically, the global myelodysplastic syndrome treatment market can be segmented into North America, Europe, Asia Pacific, Latin America, Africa, and Middle East. North America is projected to dominate the global myelodysplastic syndrome market during the forecast period due to factors such as the rise in aging population and growing awareness about the disease among the population coupled with unmet medical needs in this region.

Key players operating in the global myelodysplastic syndrome treatment market are Celgene Corporation, Otsuka Holdings Co., Ltd., Sandoz, Inc., Dr. Reddys Laboratories, Inc., Accord Healthcare Ltd., Mylan N.V., and Pfizer, Inc.

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Myelodysplastic Syndrome Treatment Market: Research Analysis by Basic Information, Manufacturing Base, Sales Area and Regions - Drnewsindustry

SEATTLE--(BUSINESS WIRE)--Omeros Corporation (Nasdaq: OMER) today announced positive data from its pivotal clinical trial of the companys novel investigational complement inhibitor narsoplimab in the treatment of hematopoietic stem cell transplant-associated thrombotic microangiopathy (HSCT-TMA), a frequently lethal complication of HSCT. These preliminary data were recently provided to FDA as part of the companys ongoing interactions with the Agency on the narsoplimab Biologics License Application (BLA). All safety and efficacy endpoints including the composite primary endpoint and the secondary endpoints are agreed with FDA. The reported data support a strongly positive benefit-risk balance.

The primary efficacy endpoint in this single-arm open-label trial of HSCT-TMA patients is the proportion of patients who achieve a highly rigorous set of response criteria that requires both improvement in HSCT-TMA laboratory markers and improvement in clinical status (organ function and transfusions). Patients who did not fully meet these criteria were considered non-responders. The secondary endpoints include survival rates and change from baseline in HSCT-TMA laboratory markers. Consistent with the pre-specified statistical analysis plan for the trial, the primary and secondary endpoints are assessed for (1) all patients who received at least one dose of narsoplimab and (2) patients who received at least 4 weeks of narsoplimab dosing. Patients enrolled in this trial had a high expected mortality rate. In severe cases of HSCT-TMA, mortality can exceed 90 percent.

Primary Efficacy Endpoint:

Secondary Endpoints:

Safety:

The HSCT-TMA patient population enrolled in this trial had multiple high-risk features that portend a poor outcome. These include persistence of HSCT-TMA despite modification of immunosuppression (which was a criterion for entry into the trial), graft-versus-host disease, significant infections, non-infectious pulmonary complications and neurological findings. Patients in the trial had a high expected death rate, with 93 percent of them having multiple risk factors.

Patient enrollment in the pivotal trial has been completed. The details of the endpoints, including the response criteria agreed with FDA, and the number of patients in the trial remain confidential for competitive business reasons.

Last year the company reported data on 19 HSCT-TMA patients treated with narsoplimab on which FDA granted breakthrough therapy designation. The results reported today are even stronger. The response rate remains equally high at 56 percent, while the 100-day survival has improved from 53 percent to 65 percent.

The response rate in this high-risk population would be expected to be 10 to 15 percent with a 100-day survival rate of less than 20 percent. The response rate and 100-day survival achieved with narsoplimab in this trial demonstrate an unprecedented effect in this condition, said Rafael Duarte M.D., Ph.D., F.R.C.P., Associate Professor, Head of Hematology Department and Hematopoietic Transplantation Program, University Hospital Puerta de Hierro Majadahonda, Madrid, Spain, and Secretary of the European Society for Blood and Marrow Transplantation. The other secondary endpoints are equally impressive. The data are consistent with my personal experience with narsoplimab. Patients with severe forms of HSCT-TMA have a dismal prognosis with no treatment currently available. I expect a treatment with this profile would be widely adopted for use in these patients and even lead to increased physician recognition of the disorder.

Omeros reported the initiation of its rolling BLA in October. Narsoplimab, also referred to as OMS721, is Omeros lead human monoclonal antibody targeting mannan-binding lectin-associated serine protease-2 (MASP-2) and has breakthrough therapy designation from FDA for this indication.

The striking results seen in our pivotal trial are tremendously gratifying, said Gregory A. Demopulos, M.D., chairman and chief executive officer of Omeros. Our rolling BLA is underway the nonclinical sections have been submitted and the data from this trial form the efficacy basis of the application. We continue to compile the remaining sections of the BLA and look forward to continued partnership with regulators to make narsoplimab widely available for the treatment of this devastating condition.

Data from this pivotal trial will also support the narsoplimab marketing authorization application for HSCT-TMA in Europe. The data are planned for publication and for presentation at international congresses in the first part of 2020.

In addition to breakthrough therapy designation from FDA, narsoplimab has orphan drug designation in both the U.S. and Europe for HSCT-TMA. Narsoplimab also has been awarded breakthrough therapy designation for immunoglobulin A nephropathy (IgAN), and Omeros has Phase 3 programs for narsoplimab ongoing in IgAN and in atypical hemolytic uremic syndrome (aHUS).

Conference Call and Webcast Details

Omeros management will host a webcast and conference call to present data from its pivotal trial of narsoplimab in HSCT-TMA. The call will be held today at 8:30 a.m. Eastern Time; 5:30 a.m. Pacific Time. To access the live conference call via phone, please dial (844) 831-4029 from the United States and Canada or (920) 663-6278 internationally. The participant passcode is 3774209. Please dial in approximately 10 minutes prior to the start of the call. A telephone replay will be available for one week following the call and may be accessed by dialing (855) 859-2056 from the United States and Canada or (404) 537-3406 internationally. The replay passcode is 3774209.

To access the live or subsequently archived webcast and presentation materials on the internet, go to the companys website at http://www.omeros.com and select Events under the Investors section of the website. To access the live webcast, please connect to the website at least 15 minutes prior to the call to allow for any software download that may be necessary.

About Omeros Corporation

Omeros is an innovative biopharmaceutical company committed to discovering, developing and commercializing small-molecule and protein therapeutics for large-market as well as orphan indications targeting complement-mediated diseases, disorders of the central nervous system and immune-related diseases, including cancers. In addition to its commercial product OMIDRIA (phenylephrine and ketorolac intraocular solution) 1%/0.3%, Omeros has multiple Phase 3 and Phase 2 clinical-stage development programs focused on complement-mediated disorders and substance abuse. In addition, the company has a diverse group of preclinical programs including GPR174, a novel target in immuno-oncology that modulates a new cancer immunity axis recently discovered by Omeros. Small-molecule inhibitors of GPR174 are part of Omeros proprietary G protein-coupled receptor (GPCR) platform through which it controls 54 new GPCR drug targets and their corresponding compounds. The company also exclusively possesses a novel antibody-generating platform.

About HSCT-TMA

Hematopoietic stem cell transplant-associated thrombotic microangiopathy (HSCT-TMA) is a significant and often lethal complication of stem cell transplants. This condition is a systemic, multifactorial disorder caused by endothelial cell damage induced by conditioning regimens, immunosuppressant therapies, infection, GvHD, and other factors associated with stem cell transplantation. Endothelial damage, which activates the lectin pathway of complement, plays a central role in the development of HSCT-TMA. The condition occurs in both autologous and allogeneic transplants but is more common in the allogeneic population. In the United States and Europe, approximately 25,000 to 30,000 allogeneic transplants are performed annually. Recent reports in both adult and pediatric allogeneic stem cell transplant populations have found an HSCT-TMA incidence of approximately 40 percent, and high-risk features may be present in up to 80 percent of these patients. In severe cases of HSCT-TMA, mortality can exceed 90 percent and, even in those who survive, long-term renal sequalae are common. There is no approved therapy or standard of care for HSCT-TMA.

About Narsoplimab

Narsoplimab, also known as OMS721, is an investigational human monoclonal antibody targeting mannan-binding lectin-associated serine protease-2 (MASP-2), a novel pro-inflammatory protein target and the effector enzyme of the lectin pathway of complement. Importantly, inhibition of MASP-2 does not appear to interfere with the antibody-dependent classical complement activation pathway, which is a critical component of the acquired immune response to infection. Omeros controls the worldwide rights to MASP-2 and all therapeutics targeting MASP-2.

Phase 3 clinical programs are in progress for narsoplimab in hematopoietic stem cell transplant-associated thrombotic microangiopathy (HSCT-TMA), in immunoglobulin A (IgA) nephropathy, and in atypical hemolytic uremic syndrome (aHUS). The FDA has granted narsoplimab breakthrough therapy designations for HSCT-TMA and for IgA nephropathy; orphan drug status for the prevention (inhibition) of complement-mediated thrombotic microangiopathies, for the treatment of HSCT-TMA and for the treatment of IgA nephropathy; and fast track designation for the treatment of patients with aHUS. The European Medicines Agency has granted orphan drug designation to narsoplimab for treatment in HSCT and for treatment of primary IgA nephropathy.

Forward-Looking Statements

This press release contains forward-looking statements within the meaning of Section 27A of the Securities Act of 1933 and Section 21E of the Securities Exchange Act of 1934, which are subject to the safe harbor created by those sections for such statements. All statements other than statements of historical fact are forward-looking statements, which are often indicated by terms such as anticipate, believe, could, estimate, expect, goal, intend, likely, look forward to, may, on track, plan, potential, predict, project, prospects, scheduled, should, slated, targeting, will, would and similar expressions and variations thereof. Forward-looking statements, including statements regarding anticipated regulatory submissions, expectations regarding regulatory exclusivities, the timing and results of ongoing or anticipated clinical trials, and the therapeutic application of Omeros investigational product, are based on managements beliefs and assumptions and on information available to management only as of the date of this press release. Omeros actual results could differ materially from those anticipated in these forward-looking statements for many reasons, including, without limitation, availability and timing of data from clinical trials and the results of such trials, unproven preclinical and clinical development activities, regulatory oversight, intellectual property claims, competitive developments, litigation, and the risks, uncertainties and other factors described under the heading Risk Factors in the companys Annual Report on Form 10-K filed with the Securities and Exchange Commission on March 1, 2019. Given these risks, uncertainties and other factors, you should not place undue reliance on these forward-looking statements, and the company assumes no obligation to update these forward-looking statements, whether as a result of any new information, future events or otherwise, except as required by applicable law.

Source: Omeros Corporation

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Omeros Reports Positive Data Across Primary and Secondary Endpoints in Pivotal Trial of Hematopoietic Stem Cell Transplant-Associated Thrombotic...

NEW YORK, Dec. 4, 2019 /PRNewswire/ --

Hunter Syndrome Treatment Market Size, Share & Trend Analysis By Treatment (Enzyme Replacement Therapy, Hematopoietic Stem Cell Transplant), By Region, And Segment Forecasts, 2019 - 2026

Read the full report: https://www.reportlinker.com/p05830601/?utm_source=PRN

The global hunter syndrome treatment market size is expected to reach a value of USD 1.52 billion by 2026, expanding at a CAGR of 7.1%. High unmet needs, robust pipeline, increasing awareness about this rare disease and growing R&D activities for the development of novel therapies are expected to drive market growth over the forecast period.

Hunter syndrome, also referred as mucopolysaccharidosis type II (MPS II), is a rare genetic disorder caused by the missing or malfunctioning iduronate-2-sulfatase enzyme. According to the data published by the National Institute of Neurological Disorders and Stroke, MPS II syndrome occurs in around 1 in every 100,000 to 150,000 male births.

Presently, there are no approved curative therapies for the treatment of Hunter syndrome.The available treatment options such as enzyme replacement therapy (ERT) and hematopoietic stem cell transplant (HSCT) are focused on providing symptomatic relief and management of complications associated with disease progression.

Shire plc's Elaprase (idursulfase) is the only key drug available for the treatment of Hunter syndrome worldwide, with GC Pharma's Hunterase (idursulfase beta) being approved only in South Korea.

Key players are focused on extensive R&D activities for product development and gaining approval as novel therapies.Launch of such novel therapies in the near future is expected to significantly fuel the Hunter syndrome treatment market growth.

For instance, in May 2018, REGENXBIO Inc. received the U.S. FDA's Fast Track designation for its novel drug candidate RGX-121, indicated for the disease treatment.

Further Key Findings from the Study Suggest: The enzyme replacement therapy segment has acquired the largest share in 2018, owing to the increased adoption of Elaprase and the potential approval of Hunterase worldwide The absence of curative therapies for MPS II creates lucrative opportunities for the key players in the market for developing new therapies The launch of late-stage pipeline therapies is expected to drive the market growth during the forecast period Currently, Shire Plc. is one of the leading players, supported by strong sales of their marketed drug ELAPRASE for the disease treatment Major players in the market are adopting inorganic growth strategies such as partnerships and collaborations for the development and commercialization of novel therapies In 2018, North America held a dominant position in the global market, owing to favorable reimbursement scenario, high awareness regarding rare disorders, and presence of major players Some of the key companies in hunter syndrome treatment market include GC Pharma, Sangamo Therapeutics, Inc.; JCR Pharmaceuticals Co Ltd.; RegenxBio Inc.; and Shire Plc. (Takeda Pharmaceutical Company)

Read the full report: https://www.reportlinker.com/p05830601/?utm_source=PRN

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Hunter Syndrome Treatment Market Size, Share & Trend Analysis By Treatment, By Region And Segment Forecasts, 2019 - 2026 - P&T Community

Researchers at Karolinska Institutet have developed a new method to separate different types of a common herpes virus (HHV-6) that has been linked to multiple sclerosis (MS).

By analyzing antibodies in the blood against the most divergent proteins of herpesvirus 6A and 6B, these researchers were able to show that MS-patients carry the herpesvirus 6A to a greater extent than healthy individuals.

This is relevant news since as many as 80 percent of all children are infected with the HHV-6 virus before 2 years of age.

But since it hasnt been possible to tell the variants apart post-infection, it has been difficult to say whether HHV-6A or B is a risk factor for MS, said these researchers in a related press release.

The findings published on November 26, 2019, in Frontiers in Immunology, indicate a potential role for HHV-6A in MS vaccine development.

Multiple sclerosis is an autoimmune disease that affects the central nervous system (CNS). The cause of the disease is unclear, but one plausible explanation is a virus tricks the CNS to attack the bodys own tissue.

MS-related news article

HHV-6 has previously been associated with MS, but in those studies, it wasnt possible to distinguish between 6A and 6B.

HHV-6 infection has been associated with complications of varying severity in hematopoietic stem cell transplant recipients, to a lesser degree in solid organ transplant recipients, and in those who are otherwise immunosuppressed, says the HHV-6Foundation.org.

These researchers have been able to show that HHV-6B can cause mild conditions such as roseola in children, but it has been unclear if HHV-6A is the cause of any disease.

In this study, however, the researchers were able to distinguish between the A and B virus by analyzing antibodies in the blood against the proteinsimmediate-early protein 1A and 1B (IE1A and IE1B)that diverge the most between the 2 viruses.

This is a big breakthrough for both the MS and herpes virus research, says Anna Fogdell-Hahn, associate professor at the Department of Clinical Neuroscience at Karolinska Institutet and one of the studys senior authors, in this press release.

Recent herpes vaccine news

For one, it supports the theory that HHV-6A could be a contributing factor to the development of MS.

On top of that, we are now able, with this new method, to find out how common these different types of HHV-6 are, something we havent been able to do previously.

The researchers compared antibody levels in blood samples of some 8,700 MS-patients against more than 7,200 healthy people whose gender, date of birth, date of the blood sample and other factors matched those with MS.

They concluded that people with MS had a 55 percent higher risk of carrying antibodies against the HHV-6A protein than the control group.

In a sub-group of almost 500 people, whose blood samples were drawn before the onset of the disease, the risk of developing MS in the future was more than doubled if they had a 6A viral infection.

The younger the people were when the virus was first discovered in the blood, the higher the risk was of developing MS in the future.

HHV-6B, on the other hand, was not positively associated with MS.

Instead, MS-patients had lower levels of antibodies toward IE1B than those without MS.

Antibodies toward Epstein-Barr virus (EBV), another herpes virus that is also associated with MS, were analyzed with the same method and the researchers were able to show that individuals affected with both viruses had an even greater risk of MS.

This indicates that several virus infections could be acting jointly to increase the risk of MS.

Both HHV-6A and 6B can infect our brain cells, but they do it in slightly different ways.

Therefore, it is now interesting to go forward and attempt to map out exactly how the viruses could affect the onset of MS, says Anna Fogdell-Hahn.

The research has been financed by grants from the Swedish Research Council, Stockholm County Council, Swedish Brain Foundation, KAW Foundation, Margareta af Ugglas Foundation, MultipleMS Horizon 2020, Multiple Sclerosis Society of Canada and the Swedish Society of Medical Research. Some of the researchers have previously received grants/fees by pharmaceutical companies in various contexts.

Multiple Sclerosis news published by Precision Vaccinations

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Herpes Virus Linked to Multiple Sclerosis - PrecisionVaccinations

Outcome means a special Thanksgiving

Hailie and Treylin Hyman saw the bruising on their baby girls leg as a sign that the active 1-year-old was learning to walk.

But as a blood test would later reveal, little Maci was actually suffering from an extremely rare blood cancer that threatened her life without a risky treatment - atreatmentalmost as dangerous as the disease.

In the beginning, it was very scary, Hailie Hyman told The Greenville News.

I couldnt think of anything but the bad things, she confessed. It was all about the statistics. And the statistics arent good.

Hailie Hyman holds her daughter Maci, 1, before an appointment at the Prisma Health Pediatric Hematology Oncology Center Monday, Nov. 4, 2019.(Photo: JOSH MORGAN/Staff)

Terrifying months followed the diagnosis, punctuated by one critical complication after another, leaving the Boiling Springs couple to wonder if Maci would survive.

Somehow, though, the blue-eyed toddler pulled through.And now her family is looking forward to a special Thanksgiving with much to be grateful for.

The Hymans journey began last February atMacis 1-year-old well-child checkup.

We had no idea anything was wrong, her mom said.But they did a routine (blood test) and a couple of hours later, we got a call saying her platelets were very low.

The Hymans were referred to a hematologist who found other abnormalities in Macis blood and scheduled a bone marrow biopsy to investigate further.

Hailie Hyman holds her daughter Maci, 1, before an appointment at the Prisma Health Pediatric Hematology Oncology Center Monday, Nov. 4, 2019.(Photo: JOSH MORGAN/Staff)

During the procedure, the child suffered an aneurysm in an artery and went into cardiac arrest. The team performed CPR on her for 20 minutes before she was stabilized, her mom said.

Later, in the pediatric intensive care unit, she suffered internal bleeding, too.

It was really hard, she said. There were many nights that I would just pray and pray and pray.

Initially believing Maci had leukemia, doctors subsequently determined she had myelodysplastic syndrome, or MDS.

The condition occurs when abnormal cells in the bone marrow leave the patient unable to make enough blood, according to the American Cancer Society.

Its rare, afflicting as few 10,000 Americans a year, though the actual number is unknown.

Maci Hyman, 1, interacts with hospital staff before an appointment at the Prisma Health Pediatric Hematology Oncology Center Monday, Nov. 4, 2019.(Photo: JOSH MORGAN/Staff)

In children, its rarer still. Most people arediagnosed in their 70s.

We were told that just four out of 1 million children get it every year, Hailie Hyman said.

That made the diagnosis elusive at first, said Dr. Nichole Bryant, a pediatric hematologist-oncologist with Prisma Health-Upstate, formerly Greenville Health System.

Shes the only one Ive seen in my career, she said.

Maci had to have regular blood transfusions, antibiotics and other medications to fight the MDS, Bryant said. But the only hope for a cure was a stem cell transplant at the Medical University of South Carolina in Charleston.

When they said that was the only treatment plan for MDS, I of course went to Google, Hailie Hyman said. I read about transplant patients and ...all the complications. It was terrifying. But no matter how many bad things I saw, we had to do it. There is no other option.

The transplantis extremely risky.

Hailie Hyman looks at a fish tank with her daughter Maci, 1, before an appointment at the Prisma Health Pediatric Hematology Oncology Center Monday, Nov. 4, 2019.(Photo: JOSH MORGAN/Staff)

First, high doses of chemotherapy are given to destroy the diseased bone marrow, leaving the patient without an immune system, so fighting infections becomes a challenge. Then healthy donor marrow is infused.

Its also fraught with potentially life-threatening complications, including graft vs. host disease, which occurs when immune cells from the donor attack the patients body, Bryant said. Other complications include permanent kidney damage and gastrointestinal problems.

They have to go to hell and back, she said. But its the only option for long-term survival.

Maci had a really rough start, suffering lots and lots and lots of complications, Bryant said.

Her kidneys failed, so she wound up on dialysis. When she couldnt breathe on her own, she was put on a ventilator. And because she couldnt eat, she had to be tube fed.

Hailie Hyman looks at a fish tank with her daughter Maci, 1, before an appointment at the Prisma Health Pediatric Hematology Oncology Center Monday, Nov. 4, 2019.(Photo: JOSH MORGAN/Staff)

She had blistering sores in her mouth and throughout her GI tract, her mom said. Because her liver wasnt functioning properly, her abdomen filled up with fluid that had to be drained. She was bleeding so profusely in her lungs that one of them collapsed.

Maci, who was sedated through much of it, was put on full life support, she said.

That night we almost lost her, her mom said. We were in the hallway crying our eyes out. We didnt know what do to or think. It was pretty scary for a while.

Somehow, Maci made it.

There were so many times during her first months that it seemed like she would not survive, Bryant said. So the fact that she is here ... is really a miracle.

Macis family found an unrelated donor through the National Marrow Donor Program, enlisting hundreds of other people to join the registry in the process, Bryant said.

Nichole Bryant, M.D.(Photo: Provided)

It was an important part of their journey that maybe didnt directly benefit Maci, she said. But if everybody did that, we wouldnt have difficulty finding a donor for anybody.

Doctors have no explanation for why Maci got MDS. She didnt carry the genetic mutation for it and there is no family history.

She is a rare child - and not in a good way, her mom said, adding,Youve got to laugh sometimes or youre going to cry.

Maci was admitted to MUSC on June 2 and released on Oct. 14.

The Hymans, both 22, spent the entire time in Charlestonwhile Hailies mom cared for their older daughter, Athena, now 2.

Treylins employer held his welding job open for him. And other friends and family members did what they could to help.

We had many, many people very generously donate to us to cover expenses at home and living expenses where we were, Hailie Hyman said.

We are thankful for everyone who helped us through it the cards, the gifts, the donations. Every single cent is greatly appreciated.

They still need to travel to Charleston once a week to see the transplant doctor. In between, Maci is seen in Greenville.

She's doing well, but recovery from a transplant can take months to years, Bryant said.

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Her kidneys are functioning again so she was able to come off dialysis. But she still must take many medications, including anti-rejection drugs that suppress her immune system and leaveher at risk for infection. And she still must be tube fed.

She is miles ahead of where she was two months ago, Bryant said. But she still has a long way to go. Its a long, long road.

Macis mom says she can be up and playing one day and flopped over on the couch another. She still experiences a lot of nausea and vomiting, but is doing well compared to where she was.

Hailie Hyman pulls her daughter Maci, 1, in a wagon in the hallway before an appointment at the Prisma Health Pediatric Hematology Oncology Center Monday, Nov. 4, 2019.(Photo: JOSH MORGAN/Staff)

So as the nation pauses to give thanks this Thanksgiving, she says the family will be countingtheir many blessings family andfriends, Gods mercy, andthe doctors and nurses who saved Macis life.

She has battled a lot and overcome a lot, she said. I have no doubt she will be able to get through.

Want to know more about becoming a marrow donor? Go to bethematch.org.

Read or Share this story: https://www.greenvilleonline.com/story/news/health/2019/11/27/upstate-sc-toddler-survives-rare-cancer-and-risky-procedure-treat/4158606002/

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Upstate SC toddler survives rare cancer and the risky procedure used to treat it - Greenville News

Dr Sister Ltd continues to offer effective skin treatments to both men and women, and they have been doing this for over 45 years now. Doctor Sister provides a comprehensive portfolio of treatments while at the same time delivering natural-looking, regenerative and enhancing results for the face and body. They are big believers in helping slow down the ageing process for each client, as of course, they are unable to stop time for you.

At Dr Sister Ltd, you can be treated by a professional doctor that trains other practitioners. Dr Sisterhimself has introduced over ten ground-breaking treatments to the UK market, along with eight published books, many articles in international peer reviews medical journals and general press, as well as being one of UKs and Europes leading lecturer and trainer in the field of Aesthetic Medicine.

The treatments offered by Dr Sister Ltd are non-invasive so there is no surgery and no downtime. Some of the skin treatments on offer at Dr Sister Ltd include the following; mini face lift, non-surgical face lift, vampire facial, PRP treatment and PRP injection. That is not an exhaustive list and he is also a renowned hormonal expert.

Dr. Sister has perfected safe, effective, natural-looking treatments, which has made him a worldwide expert and teacher in regenerative and innovative procedures such as Dracula PRP, Mint Lift including the new Stem Cell Facelift.

The PRP treatment (Dr. Sister has his own superior trademarked version called Dracula Therapy) may be unfamiliar to some clients. Dr Sister explains the procedure in great detail on their site. APRP treatment is a powerful anti-ageing treatment that involves using your blood as an injectable treatment (PRP Injection). Dr Daniel Sister was the first to introduce the treatment into the UK, and now he calls it Dracula Therapy.

With the Dracula Therapy or vampire facial, you will notice results within 3-4 weeks, and often only one PRP injection is required. However, the treatment may need to be repeated every 2-6 months because of the on-going ageing process.

The PRP injections generally appeal to patients looking for a more natural approach to facial rejuvenation, which is the rejuvenation process of using their cells. This treatment does not use synthetic fillers or animal products and has no risks or side effects.

At Dr Sister Ltd, they are well known for their aesthetic treatments, in particular, the MINT lift and Dr Sister is the training partner for the MINT lift. It is a PDO thread lift that offers exceptional results. Dr. Sister has been particularly impressed by the results as it provides an immediate and obvious lift, which many of his patients are looking for.

Dr Sister Ltd also mentions that local anaesthetic is used making the procedure pain free, and patients generally return to work and usual activities the following day. There are many benefits such as soft tissue lifting, instant lift, results lasting around 18 months.

If you would like to find out more about the treatments on offer at Dr Sister Ltd, there are many ways to get in touch. You can email press@drdanielsister.com your query, and they will get back to you as soon as possible, or you can go online to their website at https://drdanielsister.com. On their site, you will find all the information about the top treatments, fees, testimonials, and Dr Sister Ltd.

Source:https://thenewsfront.com/dr-sister-ltd-offers-effective-skin-treatments-to-both-men-and-women/

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Dr Sister Ltd Offers Effective Skin Treatments to Both Men and Women - The News Front

A research report on Transplant Diagnostics Market by Persistence Market Research features an in-depth analysis on the latest market trends. The report also includes detailed abstracts about statistics, revenue forecasts and market valuation. The report also offers a detailed analysis on the competitive landscape of the market.

Organ transplantation is the need to relocate organ in order to treat organ failure such as liver, pancreas, lungs, kidney, and heart. Human leukocyte antigens are the antigens found on the surface of the cell that regulates the body recognition and rejects foreign tissue transplant. It is the major histocompatibility complex in the human beings, which is controlled by the genes located on the chromosome six. Human leukocyte antigen (HLA) diagnostic testing is performed to determine the tissue compatibility between the donor and recipient in organ and bone marrow transplant.

In addition, a close match between a donor and recipient HLA marker is essential. It increases the probability of graft survival and minimizes severe immunologic transplant complications. It is performed with the help of non-molecular assay and molecular assay. The non-molecular assay includes serological assay and mixed lymphocyte culture (MLC) assay. In addition, molecular assay comprises PCR-based assay (sequence -pecific primer PCR, and sequence-specific oligonucleotide PCR), and sequencing-based assay (Sanger sequencing and next-generation sequencing). Hospitals, transplant centers, research laboratories, academic institutes, and commercial service providers are the major end-users of transplant diagnostics.

To Remain Ahead Of Your Competitors, Request For A Sample Here @https://www.persistencemarketresearch.com/samples/4768

North America dominates the global market for transplant diagnostics due to continuous reduction in the average cost of gene sequencing, private-public funding for the development of HLA typing technology and rising number of stem cells, and solid organ-based transplantation in the region. Asia is expected to show a high growth rate in the next five years in the global transplant diagnostics market with China and India being the fastest growing markets in the Asia Pacific region. The key driving forces for the transplant diagnostics market in developing countries are the large pool of patients, improving healthcare infrastructure, rising public and private support to develop human leukocyte antigen typing technologies, and growing focus of life science companies in the region.

Growing geriatric population, rising number of soft tissue, solid and stem cell based transplantation, rising prevalence of chronic diseases, growth in robot-assisted laboratory automation of diagnostic procedures, and technological advancement in the field of human leukocyte antigen (HLA) typing are some of the key factors driving the growth of the global transplant diagnostics market. In addition, increase in public-private investment to develop innovative human leukocyte antigen (HLA) testing products, rising application of HLA typing products in clinical diagnostics, improving healthcare infrastructure in developing countries, continuous reduction in average cost of gene sequencing, and increasing installation base of PCR and NGS instruments are driving the growth of the global transplant diagnostic market. However, factors such as high cost of PCR and NGS instruments, limited medical reimbursement for transplantation procedure, and a huge gap between organ donation and transplantation annually act as major restraints for the growth of the global transplant diagnostics market.

To Get Extensive Insights On Key Trends, Request For Customization Here @https://www.persistencemarketresearch.com/request-customization/4768

Increasing shift of preference from serological assay method to genome-based HLA profiling, and rising market penetration in developing countries to develop transplant diagnostic products would pose further opportunities for the growth of the transplant diagnostic market. New product launches and product development are among the major trends for the global transplant diagnostics market.

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Transplant Diagnostics Market to Witness Heightened Revenue Growth During the Forecast Period (2015-2021) - Statsflash

Lynne D. Neumayr, MD; Carolyn C. Hoppe, MD, MPH; Clark Brown, MD, PhD

2 decades; in 2017, L-glutamine oral powder was approved for the prevention of the acute complications of SCD. During the last several years there has been a dramatic increase in research into treatments that address distinct elements of SCD pathophysiology and even new curative approaches that provide new hope to patients and physicians for a clinically consequential disease that has long been neglected.

Am J Manag Care. 2019;25:-S0

For author information and disclosures, see end of text.

Background

Sickle cell disease (SCD) is a common, severe disorder that includes congenital hemolytic anemias caused by inherited point mutations in the -globin gene.1 These mutations result in abnormal hemoglobin polymerization, which leads to a cascade of physiologic consequences, including erythrocyte rigidity, vaso-occlusion, chronic anemia, hemolysis, and vasculopathy.1 This change in the behavior of hemoglobin has profound clinical consequences, including recurrent pain episodes (known as sickle cellrelated pain crises or vaso-occlusive crises), hemolytic anemia, multiorgan dysfunction, and premature death.1 Newborn screening, early immunization, and prophylactic penicillin treatment in infants and children, as well as comprehensive management for pain and disease complications, have improved outcomes in these patients; however, the average life expectancy of a patient with SCD remains only about 40 to 50 years.2,3

Globally, it is expected that approximately 306,000 people are born every year with SCD; an estimated 79% of these births occur in sub-Saharan Africa. In the United States, approximately 100,000 people are living with SCD, including approximately 1 in 365 African Americans and 1 in 16,300 Hispanic Americans.4,5

The impact of SCD on patient quality of life (QOL) has been estimated to be greater than that of cystic fibrosis and similar to that of patients undergoing hemodialysis, which is widely recognized as having a severe impact on QOL.6 Impairments are seen across functional and QOL domains and are particularly profound in terms of pain, fatigue, and physical function.7,8

Management of SCD can be intensive, time-consuming, and costly, particularly in patients with recurrent acute pain episodes. On average, patients with SCD experience approximately 3 vaso-occlusive crises each year, of which at least 1 requires inpatient treatment and 1 requires emergency department management without admission.9 Among patients who require admission, the median length of stay is approximately 6 days.9 More than 90% of acute hospital admissions for patients with SCD are due to severe and unpredictable pain crises, and these crises are responsible for 85% of all acute medical care for these patients.10 Estimates of the lifetime care costs for SCD vary dramatically based on underlying assumptions, from approximately $500,000 to nearly $9 million.11,12

Few options are currently available for the management of SCD. Hydroxyurea, which until recently was the only FDA-approved drug for adults with severe SCD genotypes (and is also used off-label for adults with less severe genotypes and children ages 9 months to 2 years), improves the course of SCD and results in substantial cost savings.13,14 Unfortunately, hydroxyurea is underutilized and treatment adherence is poor for a variety of reasons.15 Recently, L-glutamine became the second drug approved for SCD in the United States.16

Red blood cell (RBC) transfusion is common in patients with SCD for the management of acute complications, and regular or chronic transfusion regimens are used for stroke prevention in at-risk patients. Despite being effective for the management of both acute and chronic complications of SCD,1 transfusion is associated with annual costs exceeding $60,000; it requires routine, costly iron chelation therapy to prevent liver and other organ damage as a result of iron overload; and it is associated with the risk of alloimmunization.12,17 Stem cell transplantation, while potentially curative, is limited by a scarcity of matched donors and the risks for adverse events (AEs) and death.18 Currently under investigation are novel gene therapies that offer considerable hope for a more broadly applicable curative therapy.

This review will first examine our current understanding of the pathogenesis of SCD and explore the broad range of clinical manifestations of this disease. It will then focus on the relatively limited current therapeutic options, recent clinical trials, and near-term therapies for the chronic and acute management of the disease.

The Pathogenesis of SCD

SCD is not a single disorder. Rather, it is a clinical entity that includes a number of heritable hemolytic anemias with widely variable clinical severity and life expectancy. All involve point mutations in the -globin gene, resulting in an abnormal hemoglobin referred to as hemoglobin S (HbS).19 In the most common forms of SCD, which are also the most severe, the patient inherits the sickling gene from both parents and produces HbS exclusively.19 The compound heterozygous forms of SCD are defined by the production of HbS and another abnormal -globin protein.19

The point mutation in the -globin gene results in the substitution of glutamic acid in position 6 with valine in the resulting protein.1 This small change in the amino acid sequence of hemoglobin has profound structural and functional consequences, because under low oxygen conditions, it produces a hydrophobic region in deoxygenated HbS that promotes binding between the 1 and 2 chains of 2 hemoglobin molecules, ultimately resulting in HbS polymerization into rod-shaped structures.

The polymerization of HbS changes both the shape and physical properties of RBCs, resulting in red cell dehydration, increased rigidity, and a variety of deleterious structural abnormalities, including the characteristic sickled RBCs from which the disease gets its name.20 The rigidity of deoxygenated RBCs contributes to vaso-occlusion by impeding their passage through the microcirculation.1 Repeated cycles of tissue hypoxia and reperfusion damage elicits upregulation of adhesion molecules, such as P-selectin and E-selectin, on the vascular endothelium. This promotes adherence of RBCs, white blood cells (WBCs), and platelets, further contributing to a propensity for vaso-occlusive events and a chronic inflammatory state.1,20,21

Hemolytic anemia is an important driver of the pathophysiology of SCD. The average RBC in homozygous SCD survives only approximately 10 to 20 days, compared with 120 days for normal RBCs.22 Destruction and release of the contents of RBCs into the circulation results in progressive endothelial dysfunction and proliferation, which may in part be due to scavenging of nitric oxide (a key regulator of vascular tone) by extracellular hemoglobin.20,23-25 The end result is an impaired vasodilatory response, chronic activation of endothelial cells and platelets, and an ongoing inflammatory state. Exposure of phosphatidylserine, which is normally only found on the inner surface of the RBC membrane, also occurs, and this predisposes cells to premature lysis and promotes the activation of coagulation pathways.26,27 Excess levels of adenosine, often related to stress, are also seen in SCD. Adenosine signaling contributes to the pathophysiology of SCD by stimulating the production of erythrocyte 2,3-bisphosphoglycerate, an intracellular signal that decreases oxygen binding to hemoglobin.28

Clinical Consequences of SCD

SCD is associated with a broad range of acute and chronic complications that have a profound impact on patients, their families, and society. As noted previously, patients with SCD can present with a broad range of manifestations and disease severities depending upon the underlying genetics of their disease; the discussion below primarily refers to the most common homozygous form of the disease.

Acute pain events affect approximately 60% of patients with SCD in any given year.29-31 Such events can begin as early as 6 months of age and may recur throughout the patients life. Acute pain events are responsible for more than three-quarters of hospitalizations in patients with SCD,32 and from the perspective of the patient, they are often considered the most important and disabling consequence of the disease.32,33 Many such events can be managed at home with oral analgesics, hydration, and rest; however, in some cases, patients must be administered opioids in the emergency department or hospital setting to achieve adequate pain control.34 Acute pain events are major contributors to the high healthcare utilization of many patients with SCD.32

Stroke is the most common, and most concerning, long-term risk of homozygous SCD. The risk for stroke in children with SCD is approximately 300 times higher than for children without SCD, and approximately 25% of adults with SCA will have a stroke.20,35 Silent cerebral infarcts occur in 27% of patients by age 6 years and in 37% by age 14 years; the prevalence of silent cerebral infarct in adults is less well defined, although it is likely that progressive injury occurs as patients age.36 Cognitive impairment is seen in 5 to 9 times as many patients with SCD as compared with patients without SCD, likely due to silent repetitive ischemic brain injury.29 The use of transcranial Doppler or MRI to screen patients can help to identify patients who would benefit from additional measures to decrease the frequency and severity of stroke.20

Continued here:
Sickle Cell Disease: Current Treatment and Emerging Therapies - AJMC.com Managed Markets Network

Hailie Hyman holds her daughter Maci, 1, before an appointment at the Prisma Health Pediatric Hematology Oncology Center Monday, Nov. 4, 2019.(Photo: JOSH MORGAN/Staff)

GREENVILLE, S.C.Hailie and Treylin Hyman saw the bruising on their baby girls leg as a sign that the active 1-year-old was learning to walk.

But as a blood test would later reveal, little Maci was actually suffering from an extremely rare blood cancer that threatened her life without a risky treatment - atreatmentalmost as dangerous as the disease.

In the beginning, it was very scary, Hailie Hyman told The Greenville News.

I couldnt think of anything but the bad things, she confessed. It was all about the statistics. And the statistics arent good.

Terrifying months followed the diagnosis, punctuated by one critical complication after another, leaving the Boiling Springs couple to wonder if Maci would survive.

Somehow, though, the blue-eyed toddler pulled through.And now her family is looking forward to a special Thanksgiving with much to be grateful for.

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The Hymans journey began last February atMacis 1-year-old well-child checkup.

We had no idea anything was wrong, her mom said.But they did a routine (blood test) and a couple of hours later, we got a call saying her platelets were very low.

The Hymans were referred to a hematologist who found other abnormalities in Macis blood and scheduled a bone marrow biopsy to investigate further.

Hailie Hyman holds her daughter Maci, 1, before an appointment at the Prisma Health Pediatric Hematology Oncology Center Monday, Nov. 4, 2019.(Photo: JOSH MORGAN/Staff)

During the procedure, the child suffered an aneurysm in an artery and went into cardiac arrest. The team performed CPR on her for 20 minutes before she was stabilized, her mom said.

Later, in the pediatric intensive care unit, she suffered internal bleeding, too.

It was really hard, she said. There were many nights that I would just pray and pray and pray.

Initially believing Maci had leukemia, doctors subsequently determined she had myelodysplastic syndrome, or MDS.

The condition occurs when abnormal cells in the bone marrow leave the patient unable to make enough blood, according to the American Cancer Society.

Its rare, afflicting as few 10,000 Americans a year, though the actual number is unknown.

Maci Hyman, 1, interacts with hospital staff before an appointment at the Prisma Health Pediatric Hematology Oncology Center Monday, Nov. 4, 2019.(Photo: JOSH MORGAN/Staff)

In children, its rarer still. Most people arediagnosed in their 70s.

We were told that just four out of 1 million children get it every year, Hailie Hyman said.

That made the diagnosis elusive at first, said Dr. Nichole Bryant, a pediatric hematologist-oncologist with Prisma Health-Upstate, formerly Greenville Health System.

Shes the only one Ive seen in my career, she said.

Maci had to have regular blood transfusions, antibiotics and other medications to fight the MDS, Bryant said. But the only hope for a cure was a stem cell transplant at the Medical University of South Carolina in Charleston.

When they said that was the only treatment plan for MDS, I of course went to Google, Hailie Hyman said. I read about transplant patients and ...all the complications. It was terrifying. But no matter how many bad things I saw, we had to do it. There is no other option.

The transplantis extremely risky.

Hailie Hyman looks at a fish tank with her daughter Maci, 1, before an appointment at the Prisma Health Pediatric Hematology Oncology Center Monday, Nov. 4, 2019.(Photo: JOSH MORGAN/Staff)

First, high doses of chemotherapy are given to destroy the diseased bone marrow, leaving the patient without an immune system, so fighting infections becomes a challenge. Then healthy donor marrow is infused.

Its also fraught with potentially life-threatening complications, including graft vs. host disease, which occurs when immune cells from the donor attack the patients body, Bryant said. Other complications include permanent kidney damage and gastrointestinal problems.

They have to go to hell and back, she said. But its the only option for long-term survival.

Maci had a really rough start, suffering lots and lots and lots of complications, Bryant said.

Her kidneys failed, so she wound up on dialysis. When she couldnt breathe on her own, she was put on a ventilator. And because she couldnt eat, she had to be tube fed.

Hailie Hyman looks at a fish tank with her daughter Maci, 1, before an appointment at the Prisma Health Pediatric Hematology Oncology Center Monday, Nov. 4, 2019.(Photo: JOSH MORGAN/Staff)

She had blistering sores in her mouth and throughout her GI tract, her mom said. Because her liver wasnt functioning properly, her abdomen filled up with fluid that had to be drained. She was bleeding so profusely in her lungs that one of them collapsed.

Maci, who was sedated through much of it, was put on full life support, she said.

That night we almost lost her, her mom said. We were in the hallway crying our eyes out. We didnt know what do to or think. It was pretty scary for a while.

Somehow, Maci made it.

There were so many times during her first months that it seemed like she would not survive, Bryant said. So the fact that she is here ... is really a miracle.

Macis family found an unrelated donor through the National Marrow Donor Program, enlisting hundreds of other people to join the registry in the process, Bryant said.

Nichole Bryant, M.D.(Photo: Provided)

It was an important part of their journey that maybe didnt directly benefit Maci, she said. But if everybody did that, we wouldnt have difficulty finding a donor for anybody.

Doctors have no explanation for why Maci got MDS. She didnt carry the genetic mutation for it and there is no family history.

She is a rare child - and not in a good way, her mom said, adding,Youve got to laugh sometimes or youre going to cry.

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Maci was admitted to MUSC on June 2 and released on Oct. 14.

The Hymans, both 22, spent the entire time in Charlestonwhile Hailies mom cared for their older daughter, Athena, now 2.

Treylins employer held his welding job open for him. And other friends and family members did what they could to help.

We had many, many people very generously donate to us to cover expenses at home and living expenses where we were, Hailie Hyman said.

We are thankful for everyone who helped us through it the cards, the gifts, the donations. Every single cent is greatly appreciated.

Maci's doing well, but recovery from a transplant can take months to years, Bryant said.

Her kidneys are functioning again so she was able to come off dialysis. But she still must take many medications, including anti-rejection drugs that suppress her immune system and leaveher at risk for infection. And she still must be tube fed.

She is miles ahead of where she was two months ago, Bryant said. But she still has a long way to go. Its a long, long road.

Macis mom says she can be up and playing one day and flopped over on the couch another. She still experiences a lot of nausea and vomiting, but is doing well compared to where she was.

Hailie Hyman pulls her daughter Maci, 1, in a wagon in the hallway before an appointment at the Prisma Health Pediatric Hematology Oncology Center Monday, Nov. 4, 2019.(Photo: JOSH MORGAN/Staff)

So as the nation pauses to give thanks this Thanksgiving, she says the family will be countingtheir many blessings family andfriends, Gods mercy, andthe doctors and nurses who saved Macis life.

She has battled a lot and overcome a lot, she said. I have no doubt she will be able to get through.

Want to know more about becoming a marrow donor? Go to bethematch.org.

Follow Liv Osby on Twitter:@livgnews

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Read or Share this story: https://www.usatoday.com/story/news/health/2019/11/28/south-carolina-toddler-survives-rare-blood-cancer-risky-procedure/4321002002/

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South Carolina toddler survives rare cancer and the risky procedure used to treat it - USA TODAY

What if someone told you that theres a disease you could catch where you couldnt feel any symptoms coming on? And that this occurs every 1.2 seconds somewhere in the world?

What if you were stricken with this disease then there would be a 5% chance youd lose a limb within a year and a 50-70% chance youd be dead in five years? What if you were told that this problem cost more than the five most expensive cancers in the U.S. but far less than one one-thousandth of comparative federal and private funding is spent on attacking it?

Ladies and gentlemen, please allow me to introduce you to the humble diabetic foot ulcer. While the problem may strike at the end of the body, far away from the heart or the brain, its effects are far-reaching.

I have spent my career treating and researching the lower extremity complications of diabetes. Based on my research and experience, I believe our society could eliminate immeasurable suffering if we collectively paid more attention to this problem.

OK, I know this isnt a sexy topic. Foot wounds are ugly. Many people who have them are poor. But bear with me. They are a reality for far too many Americans and people across the globe. The ages of these patients are bimodal, in that there is one population of people who are old and getting older. Conversely, with more and more people being diagnosed with Type 2 diabetes earlier, there is a population that is younger than ever being afflicted with wounds, infections and amputation. Ignoring the problem is an example of ignoring the needs of a silent and vulnerable population.

About 31 million people in the U.S. have diabetes, and about half a billion worldwide.

Diabetic foot ulcers develop because people with diabetes slowly lose the gift of pain. Over many years, people with diabetes lose feeling in their extremities. This occurs first and generally most profoundly in their feet.

Once this occurs, people with diabetes might wear a hole in their foot, just as you or I might wear a hole in a sock or shoe. This hole is called a diabetic foot ulcer.

About half the time, the ulcer will become infected. This increases the risk of further tissue damage and, in the face of frequent vascular disease, high-level amputation. Often all of this occurs with few, if any, symptoms until it is too late.

There is also good news. Studies have suggested that high-level amputations seem to decrease when interdisciplinary care is in place, regardless of the country.

Interdisciplinary teams consist of podiatric and vascular surgeons, the so-called Toe and Flow model. The concept is simple; these two specialists, can manage a great deal of the medical, surgical and biomechanical aspects of healing and aftercare.

When we add core physical therapy to this, then the threesome (what we in the field call Toe, Flow and Go) is really quite formidable. For example, our clinics at the University of Southern California and Rancho Los Amigos in Los Angeles have active participation from more than eight specialists ranging from plastic surgery to prosthetics/orthotics, to occupational therapy to nutrition to general practice to infectious disease to diabetology to nurse case management.

Truly, it takes a village to preserve a limb.

It has long been said in wound care that its not what one puts on a wound that heals it, but what one takes off. That maxim is absolutely true in the diabetic foot. Protection of the wound is key.

The gold standard for protecting the wound has been, believe it or not, to put the patient into a special cast. This device works so well because it protects the foot in a process known as offloading, or taking the burden off the foot. By its design, this cast is not easy to remove.

While this has been my personal favorite device to heal these foot wounds, patients dont like it and most doctors dont, either. In fact, fewer than 2% of centers in the country use this as their primary means of offloading. Reasons for this include fear of putting an open wound into a cast (even though the data largely refute this), the time required to apply and remove it and patients being miserable in a hot and heavy device.

Very recently, tech company offshoots have begun to partner with prosthetic/orthotic companies to create next-gen devices that can coax patients into wearing their protective device rather than forcing it upon them. They are using phone calls and a smartwatch.

After focusing on offloading pressure, the next question is what can be done to heal the wound.

Technologies ranging from fancy vacuums, to donated placental tissue, to repurposing blood cells into a dressing to topical oxygen systems have shown recent promise. Active research is being conducted with stem cell sheets consisting of specialized cells seeded on a clear sheet, spread-on skin, and gene therapy.

As challenging as healing the wound heals, the real challenge is whats next. Following healing, 40% of foot wounds will recur in one year, about two-thirds at three years, and nearly three out of four at five years.

At USC, along with colleagues in the National Health Service in the U.K., we have developed remission clinics designed to extend and promote an active life for this high-risk patient population.

This has also been combined with things like smart insoles, socks and home-based bathmats that can identify wounds before they occur. These technologies will likely initially be subscription-based but may expand beyond that.

Diabetic foot ulcers are common, complex and costly. Theyre sinister in that they come on quietly. Perhaps, though, it is now up to us to alert our own families, communities and leaders to this condition. It is, I believe, only by teaming up that we can stem the tide and preserve not only limbs, but extend lifespan, healthspan and hope.

[ Youre smart and curious about the world. So are The Conversations authors and editors. You can read us daily by subscribing to our newsletter. ]

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Diabetic foot wounds kill millions, but high-tech solutions and teamwork are making a difference - The Conversation US

In autologous stem cell and non-stem cell based therapies, an individuals cell is cultured and then re-introduced to the donors body. Used for the treatment of various bone marrow diseases, autologous stem cell and non-stem cell based therapies allows patients to have normal bone marrow, which gets destroyed in chemotherapy. The various diseases that can be treated with the help of autologous stem cell and non-stem cell based therapies include: multiple myeloma, aplastic anemia, non-Hodgkins lymphoma, Parkinsons disease, Hodgkins lymphoma, thalassemia, and diabetes. Thus, the demand for this therapy is projected to rise over the coming years.

The report is a thorough analysis of theAutologous Stem Cell and Non-Stem Cell Based Therapies Market. Comprising an in-depth analysis of the various factors boosting and inhibiting the growth of the market, this report is a key to making profitable decisions by investing in the correct segment and sub-segment, which is anticipated to make the most progress in the future.

Autologous Stem Cell and Non-Stem Cell Based Therapies Market: Trends and Opportunities

One of the key drivers for this market is the rise in the prevalence of cancer and diabetes among people across all age groups. Moreover, the growing geriatric population is another factor, which is likely to create a heightened demand for autologous stem cell and non-stem cell based therapies. Favorable reimbursement policies across several nations are also aiding the growth of this market.

To obtain all-inclusive information on forecast analysis of Autologous Stem Cell and Non-Stem Cell Based Therapies Market, Request a PDF Brochure Here https://www.transparencymarketresearch.com/sample/sample.php?flag=B&rep_id=4001

Players in the market are striving to achieve therapies that are not only safe and effective but also affordable and easy to use. Players are also investing in extensive research and development so as to speed up the treatment process of autologous stem cell and non-stem cell based therapies. While currently this treatment is quite expensive, government bodies are expected to take up initiatives and make the therapy affordable in the years to come. This is expected to drive the market in the future.

On the other hand, challenges faced by the global autologous stem cell and non-stem cell based therapies market include risks and complications associated with the therapy, such as diarrhea, hair loss, nausea, severe infections, vomiting, heart complications, and infertility.

Autologous Stem Cell and Non-Stem Cell Based Therapies Market: Geographical Analysis

By geography, North America, trailed by Europe is leading in the autologous stem cell and non-stem cell based therapies market, on account of the minimization of risks associated with the therapy. Also, these therapies are highly in demand owing to their ability to treat a large number of infectious diseases. The fact that autologous stem cell and non-stem cell based therapies do not require an outside donor, makes it more convenient and less infectious. All these factors are boosting the growth of the market in North America.

Asia Pacific is projected to show the most promising growth in the years to come with high demand from China, Vietnam, Malaysia, and India. The demand is expected to be high as autologous stem cell and non-stem cell based therapies help in the effective treatment of cardiovascular diseases. Sophisticated healthcare infrastructure and favorable tax and reimbursement policies are also expected to aid the growth of the Asia Pacific autologous stem cell and non-stem cell based therapies market.

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Autologous Stem Cell and Non-Stem Cell Based Therapies Market: Companies Mentioned

Some of the leading players operating in the autologous stem cell and non-stem cell based therapies market are Fibrocell Science, Inc., Aastrom Biosciences, Dendreon Corporation, NeoStem, Inc., BrainStorm Cell Therapeutics, Regeneus Ltd., and Genzyme Corporation.

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Autologous Stem Cell and Non-Stem Cell Based Therapies Market to Observe Strong Development by 2023 - Montana Ledger

Global Blood Therapeutics, Inc. (GBT) confirmed that the FDA has approved Oxbryta (voxelotor) tablets for the treatment of sickle cell disease in adults and children 12 years of age and older. According to the company press release, Oxbryta is the "first and only FDA-approved sickle hemoglobin polymerization inhibitor, a new class of therapy."

Earlier, the company presented updates on corporate developments on 10/8/2019 at its Analyst & Investor Day. Global Blood is a clinical-stage biopharmaceutical company having expertise in blood biology and structural and medicinal chemistry. The company's lead product candidate is voxelotor ('GBT440), an oral, once-daily therapy, designed to modulate hemoglobin affinity for oxygen for the treatment of sickle cell disease (SCD). SCD is an inherited blood disorder caused by a genetic mutation in the beta-chain of hemoglobin, leading to the formation of abnormal hemoglobin known as sickle hemoglobin (HbS).

HbS polymerizes forming rigid rods within a red blood cell (RBC). The inflexible polymer rods arranged in a sickle shape, cause hemolytic anemia (destruction of RBCs) that can result in multi-organ damage and even early death. This sickling process blocks capillaries and small blood vessels. Blocked bloodflow to organs results in inadequate oxygen delivery (hypoxia) to body tissues, which makes the SCD patients suffer recurrent and unpredictable episodes of severe pain, ultimately leading to physical and psychosocial disabilities. Voxelotor is designed to inhibit HbS polymerization.

Voxelotor had been granted priority review for the treatment of SCD, with PDUFA date set to February 26, 2020. Priority review shortens the FDA review time from the standard 10 Months to 6 months. The accelerated approval 3 months ahead of PDUFA speaks for the importance of this novel drug in the eyes of the FDA.

The NDA was based on data from the multi-national Phase 3 HOPE study, which demonstrated statistically significant and sustained improvements in hemoglobin with voxelotor. Looking at the critical need for new SCD treatments, the U.S. FDA earlier granted Breakthrough Therapy, Fast Track, Orphan Drug and Rare Pediatric Disease designations to voxelotor for the treatment of patients with SCD. The European Medicines Agency (EMA) has also included voxelotor in its Priority Medicines (PRIME) program, while the European Commission (EC) has designated voxelotor as an orphan medicinal product for the treatment of patients with SCD.

Design: The efficacy and safety of two dose levels of voxelotor, 1500 mg and 900 mg, administered orally once daily, was compared with placebo in persons with SCD in a multicenter, Phase 3, double-blind, randomized, placebo-controlled trial "HOPE." The percentage of participants who had a hemoglobin response, defined as an increase of more than 1.0 g per deciliter from baseline at week 24 in the intention-to-treat analysis was the primary endpoint. 274 participants in total were randomly assigned in a 1:1:1 ratio to receive a once-daily oral dose of 1500 mg of voxelotor, 900 mg of voxelotor, or placebo. Most participants had sickle cell anemia (homozygous hemoglobin S or hemoglobin S0-thalassemia), and approximately two thirds were receiving hydroxyurea at baseline.

Results: The Phase 3 study met the primary endpoint with nearly 60% of patients achieving >1 g/dL increase in Hb vs. placebo (p<0.001). A significantly higher percentage of participants had a Hb response in the 1500-mg voxelotor group (51%; 95% confidence interval [CI], 41 to 61) than in the placebo group (7%; 95% CI, 1 to 12), in the intention-to-treat analysis. Anemia worsened between baseline and week 24 in more placebo-treated participants than in each voxelotor dose group.

At week 24, the 1500-mg voxelotor group had significantly greater reductions from baseline in the indirect bilirubin level and percentage of reticulocytes than the placebo group. Adverse events (AEs) of grade 3 or above occurred in 26%, 23% and 26%, in the 1500-mg voxelotor group, the 900-mg voxelotor group, and the placebo group respectively. Most AEs were not treatment emergent either with the trial drug or placebo. Voxelotor increased hemoglobin levels and reduced markers of hemolysis significantly. The results are consistent with inhibition of HbS polymerization.

(Image source: Company presentation)

The company in agreement with the U.S. FDA, has already designed the post approval, confirmatory study of voxelotor, utilizing TCD flow velocity as the primary endpoint.

There are millions of SCD patients worldwide with 90,000 to 100,000 in the U.S. and about 60,000 in Europe. SCD is a congenital disease, with symptoms and organ damage starting in the early years of life. It is estimated that worldwide 250,000 to 300,000 children are born annually with SCD, which is concentrated in populations of African, Middle Eastern and South Asian descent. SCD treatment is costly, with average annual cost in the U.S. being more than $200,000 for an adult, which can lead to aggregate expense of more than $8 million over an assumed 50-year lifespan. Newborn screening, which is required by all states in the U.S., and development of new therapeutics is hence a critical need of the market.

GBT raised approximately $197.8 million in net proceeds from a public offering in June 2019 and related over-allotment option exercise in July 2019. GBT had cash, cash equivalents and marketable securities totaling to $731.7 million as of 6/30/2019. Looking at the cash burn of about $61 million in the most recent quarter ending 6/30/2019, GBT's fund position seems to be at an adequate level to carry through the commercialization of voxelotor, and other development activities without further dilution of the stock. Insiders sold a negligible, less than 15000 shares in the last 52 weeks. Institutional holding increased over 2% in 2Q-2019.

The company awarded more than $200,000 in grants to five nonprofit organizations as part of the Access to Excellent Care for Sickle Cell Patients Pilot Program (ACCEL). The grant funding will support projects to improve access to high-quality healthcare for SCD patients in the U.S. The company has launched two SCD awareness campaigns while also hiring all commercial leads as of 1H-2019.

Voxelotor does not have a direct competition as it is attacking the root cause of SCD with a new first-in-class therapy. It faces indirect competition from (1) Bristol-Myers Squibb's (BMY) hydroxyurea (DROXIA or Hydrea) and its generic form, which are approved for "reducing the frequency of painful crises and need for blood transfusions in patients with sickle cell anemia for the treatment of adults with SCD," and (2) Endari (L-glutamine oral powder), marketed by Emmaus (OTCQB: EMMA), approved for the reduction of acute complications in SCD patients of age five years and above. GBT will also face competition from one-time therapies for patients with severe SCD, like hematopoietic stem cell transplantation, gene therapy and gene editing.

bluebird bio, Inc. (BLUE) has a gene therapy candidate in clinical development - LentiGlobin BB305, which the company plans to pursue on an accelerated development path. Pfizers (PFE) rivipansel is in a Phase 3 trial however it failed the treatment goals. Novartis (NVS) crizanlizumab (SEG101), an anti-P-selectin monoclonal antibody for the prevention of vaso-occlusive crises (VOCs) in patients with SCD is in clinical development with a breakthrough designation. The U.S. FDA has accepted for a priority review of crizanlizumab based on its Phase 2 SUSTAIN study results. Estimated PDUFA date is 1/15/2020. GBT also has an anti-P-selectin monoclonal antibody therapy inclacumab, in clinical development, under worldwide, exclusive but non-diagnostic license from Roche (OTCQX:RHHBY).

Various patents covering voxelotor, including its composition of matter, methods of use and a polymorph of voxelotor will expire between 2032 and 2035. Patents that may issue from GBTs pending patent applications relating to voxelotor in the United States or from corresponding foreign patent applications, if issued, are expected to expire between 2032 and 2037. Some patents related to voxelotor are held jointly with the Regents of the University of California.

GBTs other risk is competition. For a long time, Lentiglobin has been talked about as a major competition, and while it is aimed as a curative treatment, it is expensive, and still in a much earlier stage, with the Phase 2/3 trial in planning stage only. Crizanlizumab is also not a real competition either, because it is a downstream therapeutic approach compared to GBTs.

The stock price was near the midpoint of the 52-week high and low before the approval. The approval has pushed the price up by about 20% in the time since the original version of this article was written. However, I strongly believe there's considerably more upside to this stock as the drug gets to the market in the next couple of weeks and starts generating revenue.

Thanks for reading. At the Total Pharma Tracker, we do more than follow biotech news. Using our IOMachine, our team of analysts work to be ahead of the curve.

That means that when the catalyst comes that will make or break a stock, weve positioned ourselves for success. And we share that positioning and all the analysis behind it with our members.

Disclosure: I/we have no positions in any stocks mentioned, and no plans to initiate any positions within the next 72 hours. I wrote this article myself, and it expresses my own opinions. I am not receiving compensation for it (other than from Seeking Alpha). I have no business relationship with any company whose stock is mentioned in this article.

Read more here:
Global Blood Therapeutics: Oxbryta Is Finally Approved - Seeking Alpha

In autologous stem cell and non-stem cell based therapies, an individuals cell is cultured and then re-introduced to the donors body. Used for the treatment of various bone marrow diseases, autologous stem cell and non-stem cell based therapies allows patients to have normal bone marrow, which gets destroyed in chemotherapy. The various diseases that can be treated with the help of autologous stem cell and non-stem cell based therapies include: multiple myeloma, aplastic anemia, non-Hodgkins lymphoma, Parkinsons disease, Hodgkins lymphoma, thalassemia, and diabetes. Thus, the demand for this therapy is projected to rise over the coming years.

The report is a thorough analysis of theAutologous Stem Cell and Non-Stem Cell Based Therapies Market. Comprising an in-depth analysis of the various factors boosting and inhibiting the growth of the market, this report is a key to making profitable decisions by investing in the correct segment and sub-segment, which is anticipated to make the most progress in the future.

Request a PDF Brochure of Autologous Stem Cell and Non-Stem Cell Based Therapies Market Report https://www.transparencymarketresearch.com/sample/sample.php?flag=B&rep_id=4001

Autologous Stem Cell and Non-Stem Cell Based Therapies Market: Trends and Opportunities

One of the key drivers for this market is the rise in the prevalence of cancer and diabetes among people across all age groups. Moreover, the growing geriatric population is another factor, which is likely to create a heightened demand for autologous stem cell and non-stem cell based therapies. Favorable reimbursement policies across several nations are also aiding the growth of this market.

Players in the market are striving to achieve therapies that are not only safe and effective but also affordable and easy to use. Players are also investing in extensive research and development so as to speed up the treatment process of autologous stem cell and non-stem cell based therapies. While currently this treatment is quite expensive, government bodies are expected to take up initiatives and make the therapy affordable in the years to come. This is expected to drive the market in the future.

On the other hand, challenges faced by the global autologous stem cell and non-stem cell based therapies market include risks and complications associated with the therapy, such as diarrhea, hair loss, nausea, severe infections, vomiting, heart complications, and infertility.

Autologous Stem Cell and Non-Stem Cell Based Therapies Market: Geographical Analysis

By geography, North America, trailed by Europe is leading in the autologous stem cell and non-stem cell based therapies market, on account of the minimization of risks associated with the therapy. Also, these therapies are highly in demand owing to their ability to treat a large number of infectious diseases. The fact that autologous stem cell and non-stem cell based therapies do not require an outside donor, makes it more convenient and less infectious. All these factors are boosting the growth of the market in North America.

Asia Pacific is projected to show the most promising growth in the years to come with high demand from China, Vietnam, Malaysia, and India. The demand is expected to be high as autologous stem cell and non-stem cell based therapies help in the effective treatment of cardiovascular diseases. Sophisticated healthcare infrastructure and favorable tax and reimbursement policies are also expected to aid the growth of the Asia Pacific autologous stem cell and non-stem cell based therapies market.

Request for a Discount on Autologous Stem Cell and Non-Stem Cell Based Therapies Market Report https://www.transparencymarketresearch.com/sample/sample.php?flag=D&rep_id=4001

Autologous Stem Cell and Non-Stem Cell Based Therapies Market: Companies Mentioned

Some of the leading players operating in the autologous stem cell and non-stem cell based therapies market are Fibrocell Science, Inc., Aastrom Biosciences, Dendreon Corporation, NeoStem, Inc., BrainStorm Cell Therapeutics, Regeneus Ltd., and Genzyme Corporation.

Read more here:
Autologous Stem Cell and Non Stem Cell Based Therapies Market Shares, Strategies, and Forecasts Analysis - Downey Magazine

Bone marrow is soft, spongy tissue within some bones, including those in the hips and thighs. People with certain blood-related conditions benefit from a transplant that replaces damaged cells with healthy cells, possibly from a donor.

Bone marrow transplants can be lifesaving for people with conditions such as lymphoma or leukemia, or when intensive cancer treatment has damaged blood cells.

This type of transplant can be an intensive procedure, and recovery can take a long time.

Here, we provide an overview of bone marrow transplants, including their uses, risks, and recovery.

Bone marrow contains stem cells. In healthy people, stem cells in bone marrow help create:

If a medical condition such as one that damages the blood or immune system prevents the body from creating healthy blood cells, a person may need a bone marrow transplant.

A person with any of the following conditions may be a candidate for a bone marrow transplant:

There are three types of bone marrow transplant, based on where the healthy bone marrow cells come from.

In many cases, the donor is a close family member, such as a sibling or parent. The medical name for this is an allogenic transplant.

Transplants are more likely to be effective if the donated stem cells have a similar genetic makeup to the person's own stem cells.

If a close family member is not available, the doctor will search a registry of donors to find the closest match. While an exact match is best, advances in transplant procedures are making it possible to use donors who are not an exact match.

In a procedure called an autologous transplant, the doctor will take healthy blood stem cells from the person being treated and replace these cells later, after removing any damaged cells in the sample.

In an umbilical cord transplant, also called a cord transplant, doctors use immature stem cells from the umbilical cord following a baby's birth. Unlike cells from an adult donor, the cells from an umbilical cord do not need to be as close a genetic match.

Before a bone marrow transplant, the doctor will run tests to determine the best type of procedure. They will then locate an appropriate donor, if necessary.

If they can use the person's own cells, they will collect the cells in advance and store them safely in a freezer until the transplant.

The person will then undergo other treatment, which may involve chemotherapy, radiation, or a combination of the two.

These procedures typically destroy bone marrow cells as well as cancer cells. Chemotherapy and radiation also suppress the immune system, helping to prevent it from rejecting a bone marrow transplant.

While preparing for the transplant, the person may need to stay in the hospital for 12 weeks. During this time, a healthcare professional will insert a small tube into one of the person's larger veins.

Through the tube, the person will receive medication that destroys any abnormal stem cells and weakens the immune system to prevent it from rejecting the healthy transplanted cells.

Before entering the hospital, it is a good idea to arrange:

A bone marrow transplant is not surgery. It is similar to a blood transfusion.

If a donor is involved, they will provide the stem cells well in advance of the procedure. If the transplant involves the person's own cells, the healthcare facility will keep the cells in storage.

The transplant typically takes place in several sessions over several days. Staggering the introduction of cells in this way gives them the best chance of integrating with the body.

The healthcare team may also use the tube to introduce liquids such as blood, nutrients, and medications to help fight infection or encourage the growth of bone marrow. The combination depends on the body's response to treatment.

The procedure will temporarily compromise the person's immune system, making them very susceptible to infection. Most hospitals have a dedicated, isolated space for people undergoing bone marrow transplants to help reduce their risk of infection.

After the last session, the doctor will continue to check the blood each day to determine how well the transplant has worked. They will test whether new cells are beginning to grow in bone marrow.

If a person's white blood cell count starts to rise, it indicates that the body is starting to create its own blood, indicating that the transplant has been successful.

The amount of time that it takes for the body to recover depends on:

Many other factors can affect recovery, including:

Some people are able to leave the hospital soon after the transplant, while others need to stay for several weeks or months.

The medical team will continue to monitor the person's recovery for up to 1 year. Some people find that effects of the transplant remain for life.

A bone marrow transplant is a major medical procedure. There is a high risk of complications during and after it.

The likelihood of developing complications depends on various factors, including:

Below are some of the more common complications that people who receive bone marrow transplants experience:

Some people die as a result of complications from bone marrow transplants.

A person who receives a bone marrow transplant may also experience reactions that can follow any medical procedure, including:

The body's response to a bone marrow transplant varies greatly from person to person. Factors such as age, overall health, and the reason for the transplant can all affect a person's long term outlook.

If a person receives a bone marrow transplant to treat cancer, their outlook depends, in part, on how far the cancer has spread. Cancer that has spread far from its origin, for example, responds less well to treatment.

According to the National Marrow Donor Program, the 1-year survival rate among people who have received transplants from unrelated donors increased from 42% to 60% over about the past 5 years.

A bone marrow transplant is a major medical procedure that requires preparation. This involves determining the best type of transplant, finding a donor, if necessary, and preparing for a lengthy hospital stay.

The time that it takes for the body to recover from a transplant varies, depending on factors such as a person's age and overall health and the reason for the transplant.

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Bone marrow transplant: What it is, uses, risks, and recovery - Medical News Today

Nes Ziona, Israel, Nov. 18, 2019 (GLOBE NEWSWIRE) -- Enlivex Therapeutics Ltd. (Nasdaq: ENLV), a clinical-stage immunotherapy company, today reported completion of patient recruitment into the Companys Phase Ib clinical trial in patients with severe sepsis. The study was designed to recruit ten patients, including six treated with a single dose of Allocetra, and four patients treated with two doses of Allocetra.

Enlivex announced on November 4, 2019 positive interim safety and efficacy data from an ongoing trial of off-the-shelf universal Allocetra in patients with severe sepsis. The interim analysis comparing the first six Allocetra-treated patients with 37 severe sepsis patients with equivalent source of infection and disease severity who were hospitalized at the same hospital, demonstrated the potential of single dose Allocetra infusion as therapy for prevention of sepsis-associated organ failure and mortality.

Sepsis is defined as a life-threatening organ dysfunction caused by a dysregulated immune response to infection. Sepsis has been identified by the World Health Organization as a global health priority and currently has no FDA-approved pharmacologic treatment. Sepsis is the third leading cause of mortality in the United States after cardiovascular and cancer diseases and affects approximately 1.7 million adults in the United States each year. Various studies have estimated that up to 50% of severe sepsis hospitalizations culminate in death.

ALLOCETRATMby Enlivex was designed toprovide a novel immunotherapy mechanism of actionthat targets life-threatening clinical indications that are defined as unmet medical needs, includingprevention or treatment of complications associated with bone marrow transplantations (BMT) and/or hematopoietic stem cell transplantations (HSCT); organ dysfunction and acute multiple organ failure associated with sepsis; and enablement of an effective treatment of solid tumors via immune checkpoint rebalancing.

ABOUT ENLIVEXEnlivex is a clinical stage immunotherapy company, developing an allogeneic drug pipeline for immune system rebalancing. Immune system rebalancing is critical for the treatment of life-threatening immune and inflammatory conditions which involve hyper-expression of cytokines (Cytokine Release Syndrome) and for which there are no approved treatments (unmet medical needs), as well as solid tumors immune-checkpoint rebalancing. For more information, visithttp://www.enlivex.com.Safe Harbor Statement: This press release contains forward-looking statements, which may be identified by words such as expects, plans, projects, will, may, anticipates, believes, should, would, could, intends, estimates, suggests, has the potential to and other words of similar meaning, including statements regarding expected cash balances, market opportunities for the results of current clinical studies and preclinical experiments, the effectiveness of, and market opportunities for, ALLOCETRATMprograms. All such forward-looking statements are made pursuant to the safe harbor provisions of the Private Securities Litigation Reform Act of 1995. Investors are cautioned that forward-looking statements involve risks and uncertainties that may affect Enlivexs business and prospects, including the risks that Enlivex may not succeed in generating any revenues or developing any commercial products; that the products in development may fail, may not achieve the expected results or effectiveness and/or may not generate data that would support the approval or marketing of these products for the indications being studied or for other indications; that ongoing studies may not continue to show substantial or any activity; and other risks and uncertainties that may cause results to differ materially from those set forth in the forward-looking statements. The results of clinical trials in humans may produce results that differ significantly from the results of clinical and other trials in animals. The results of early-stage trials may differ significantly from the results of more developed, later-stage trials. The development of any products using the ALLOCETRATMproduct line could also be affected by a number of other factors, including unexpected safety, efficacy or manufacturing issues, additional time requirements for data analyses and decision making, the impact of pharmaceutical industry regulation, the impact of competitive products and pricing and the impact of patents and other proprietary rights held by competitors and other third parties. In addition to the risk factors described above, investors should consider the economic, competitive, governmental, technological and other factors discussed in Enlivexs filings with the Securities and Exchange Commission, including in the Companys most recent Annual Report on Form 20-F filed with the Securities and Exchange Commission. The forward-looking statements contained in this press release speak only as of the date the statements were made, and we do not undertake any obligation to update forward-looking statements, except as required under applicable law.

ENLIVEX CONTACT: Shachar Shlosberger, CFO Enlivex Therapeutics, Ltd. shachar@enlivexpharm.com

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Enlivex Therapeutics Completes Recruitment In Phase Ib Trial Evaluating Safety And Efficacy Of Universal Off-The-Shelf Allocetra In Patients With...

On the morning of April 25, 2018, in Fort Wayne, Indiana, Omarion Jordan came into the world ten-fingers-and-toes perfect. His mother, Kristin Simpson, brought her dark-haired newborn home to a mostly empty apartment in Kendallville, about 30 miles to the north. Shed just moved in and hadnt had time to decorate. Her son, however, had everything he needed: a nursery full of toys, a crib, a bassinet and a blue octopus blanket.

Still, within his first couple of months, he was plagued by three different infections that required intravenous treatments. Doctors thought he had eczema and cradle cap. They said he was allergic to his mothers milk and told her to stop breastfeeding. Then, not long after he received a round of standard infant vaccinations, his scalp was bleeding and covered with green goop, recalled the first-time mother, who was then in her late teens. She took him to the hospital emergency room, where, again, caregivers seemed puzzled by the babys bizarre symptoms, which didnt make any sense until physicians, finally, ordered the right blood test.

What they learned was that Omarion was born with a rare genetic disorder called X-linked severe combined immunodeficiency (SCID), better known as the bubble boy disease. Caused by a mutated gene on the X chromosome, and almost always limited to males, a baby born with X-linked SCID, or SCID-X1, lacks a working immune system (hence the unusual reaction to vaccination). The bubble boy name is a reference to David Vetter, a Texas child born with SCID-X1 in 1971, who lived in a plastic bubble and ventured out in a NASA-designed suit. He died at 12, but his highly publicized life inspired a 1976 TV movie starring John Travolta.

Today, technological advances in hospitals provide a kind of bubble, protecting SCID-X1 patients with controlled circulation of filtered air. Such safeguards are necessary because a patient exposed to even the most innocuous germs can acquire infections that turn deadly. As soon as Omarion tested positive for the disorder, an ambulance carried him to Cincinnati Childrens Hospital in nearby Ohio and placed him in isolation, where he remained for the next few months. I had no idea what would happen to him, his mother recalled.

Approximately one in 40,000 to 100,000 infants is born with SCID, according to the Centers for Disease Control and Prevention. Only about 20 to 50 new cases of the SCID-X1 mutationwhich accounts for about half of all SCID casesappear in the United States each year. For years, the best treatments for SCID-X1 have been bone marrow or blood stem cell transplantations from a matched sibling donor. But fewer than 20 percent of patients have had this option. And Omarion, an only child, was not among them.

As it happened, medical scientists at St. Jude Childrens Research Hospital in Memphis, Tennessee, were then developing a bold new procedure. The strategy: introduce a normal copy of the faulty gene, designated IL2RG, into a patients own stem cells, which then go on to produce the immune system components needed to fight infection. Simpson enrolled Omarion in the clinical study and Cincinnati Childrens Hospital arranged a private jet to transport her and her son to the research hospital, where they stayed for five months.

St. Jude wasnt the first to try gene therapy for SCID-X1. Nearly 20 years ago, researchers in France reported successfully reconditioning immune systems in SCID-X1 patients using a particular virus to deliver the correct gene to cells. But when a quarter of the patients in that study developed leukemia, because the modified virus also disrupted the functioning of normal genes, the study was halted and scientists interested in gene therapy for the disorder hit the brakes.

At St. Jude, experts led by the late Brian Sorrentino, a hematologist and gene therapy researcher, set out to engineer a virus delivery vehicle that wouldnt have side effects. They started with a modified HIV vector emptied of the virus and its original contents, and filled it with a normal copy of the IL2RG gene. They engineered this vector to include insulators to prevent the vector from disturbing other genes once it integrated into the human genome. The goal was to insert the gene into stem cells that had come from the patients own bone marrow, and those cells would then go on to produce working immune system cells. It was crucial for the viral vector to not deliver the gene to other kinds of cellsand thats what the researchers observed. After gene therapy, for example, brain cells do not have a correct copy of the gene, explained Stephen Gottschalk, who chairs St. Judes Department of Bone Marrow Transplantation and Cellular Therapy.

In the experimental treatment, infants received their re-engineered stem cells just 12 days after some of their bone marrow was obtained. They went through a two-day, low-dose course of chemotherapy, which made room for the engineered cells to grow. Within four months, some of the babies were able to fight infections on their own. All eight of the initial research subjects left the hospital with a healthy immune system. The remarkably positive results made news headlines after being published this past April in the New England Journal of Medicine. Experimental gene therapy frees bubble boy babies from life of isolation, the journal Nature trumpeted.

So far, the children who participated in that study are thriving, and so are several other babies who received the treatmentincluding Omarion. As a physician and a mom, I couldnt ask for anything better, said Ewelina Mamcarz, lead author of the journal article and first-time mother to a toddler nearly the same age as Omarion. The children in the study are now playing outside and attending day care, reaching milestones just like my daughter, Mamcarz says. Theyre no different. Mamcarz, who is from Poland, came to the United States to train as a pediatric hematologist-oncologist and joined St. Jude six years ago.

Other medical centers are pursuing the treatment. The University of California, San Francisco Benioff Childrens Hospital is currently treating infant patients, and Seattle Childrens Hospital is poised to do the same. Moreover, the National Institutes of Health has seen success in applying the gene therapy to older patients, ages 3 to 37. Those participants had previously received bone marrow transplants from partially matched donors, but theyd been living with complications.

In the highly technical world of medicine today, it takes teamwork to achieve a breakthrough, and as many as 150 peoplephysicians, nurses, regulators, researchers, transplant coordinators and othersplayed a role in this one.

Sorrentino died in November 2018, but hed lived long enough to celebrate the trial results. In the early 90s, we thought gene therapy would revolutionize medicine, but it was kind of too early, said Gottschalk, who began his career in Germany. Now, nearly 30 years later, we understand the technology better, and its really starting to have a great impact. We can now develop very precise medicine, with very limited side effects. Gottschalk, who arrived at St. Jude a month before Sorrentinos diagnosis, now oversees the hospitals SCID-X1 research. Its very, very gratifying to be involved, he said.

For now the SCID-X1 gene therapy remains experimental. But with additional trials and continued monitoring of patients, St. Jude hopes that the therapy will earn Food and Drug Administration approval as a treatment within five years.

Simpson, for her part, is already convinced that the therapy can work wonders: Her son doesnt live in a bubble or, for that matter, in a hospital. He can play barefoot in the dirt with other kids, whatever he wants, because his immune system is normal like any other kid, she said. I wish there were better words than thank you.

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These Scientists May Have Found a Cure for 'Bubble Boy' Disease - Smithsonian.com