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AlloVir Research Presented at the 2021 Transplantation & Cellular Therapy Meeting Digital Experience – Business Wire
CAMBRIDGE, Mass.--(BUSINESS WIRE)--AlloVir (Nasdaq: ALVR), a late clinical-stage cell therapy company, today announced results of a subgroup analysis from a Phase 2, proof-of-concept study (CHARMS) evaluating the companys lead product candidate, Viralym-M (ALVR105), an allogeneic, off-the-shelf, multi-virus specific investigational T-cell therapy (VST), in allogeneic hematopoietic stem cell transplant (allo-HSCT) recipients with virus-associated hemorrhagic cystitis (V-HC). These data are being presented in an oral presentation during the Transplantation & Cellular Therapy (TCT) Meeting of the American Society for Transplantation and Cellular Therapy (ASTCT) and the Center for International Blood & Marrow Transplant Research (CIBMTR). Additionally, two separate oral presentations characterize the high economic and clinical burden of V-HC and double-stranded (ds) DNA viral infections in allo-HSCT recipients. Preclinical data was also presented in a poster presentation on ALVR109, AlloVirs virus-specific T-cell therapy targeting SARS-CoV-2, the virus responsible for COVID-19.
The data from the Phase 2 CHARMS study highlight Viralym-M's potential to treat and possibly prevent multiple viral infections and viral diseases. The findings presented at TCT show that this novel virus-specific T cell therapy has the potential to rapidly and effectively resolve macroscopic hematuria in allo-HSCT recipients with virus-associated hemorrhagic cystitis a disease that currently has no effective treatment options and causes significant morbidity and increased risk of mortality, said Agustin Melian, MD, Chief Medical Officer and Head of Global Medical Sciences of AlloVir. We have recently initiated our Phase 3, pivotal study of Viralym-M for the treatment of virus-associated hemorrhagic cystitis and look forward to advancing this therapy through development for patients in need.
Data of Viralym-M in fifty-eight allo-HSCT recipients with at least one treatment-refractory viral infection caused by BK virus (BKV), cytomegalovirus (CMV), adenovirus (AdV), Epstein Barr virus (EBV), human herpesvirus 6 (HHV-6), and/or JC virus (JCV) were evaluated in the CHARMS Phase 2 study. The subgroup analysis presented at TCT included 26 patients who received intravenous VST infusions for the treatment of V-HC due to infection with BKV (n=23), AdV (n=2) and BKV and AdV (n=1). Infusions were well tolerated with mild, grade 1, de novo skin rash from graft-versus-host disease (GVHD) occurring in 15% of patients (n=4). In the 20 patients with available V-HC grading, resolution of macroscopic hematuria was observed in 60% and 80% of patients at two- and six-weeks post-infusion, respectively. In comparison, resolution of macroscopic hematuria was observed in <10% and 30% of patients at weeks two and six, respectively, in a contemporary cohort of allo-HSCT recipients (n=33) with V-HC who were not treated with Viralym-M.
Health economic outcomes data was also presented in two separate oral presentations at the conference. The two presentations analyzed U.S. claims data to compare health care reimbursement, health resource utilization, and clinical outcomes in pediatric and adult allo-HSCT recipients with V-HC and those without V-HC, and allo-HSCT recipients with or without dsDNA infections, respectively. Both studies found that allo-HSCT recipients with V-HC and those with any dsDNA infection had higher reimbursement costs, increased hospital and ICU length of stay, and increased hospital readmission rates. The presence of V-HC or any dsDNA viral infection was associated with a higher risk of mortality.
In addition, a poster presentation at the conference demonstrated the in vitro effector and safety profile of ALVR109, an allogeneic, off-the-shelf investigational VST therapy designed to target SARS-CoV-2, the virus that causes the severe and life-threatening viral disease, COVID-19. These data suggest the potential for using these VSTs to treat COVID-19 in hospitalized high-risk patients to prevent the development of severe disease. A clinical trial evaluating these banked, off-the-shelf SARS-CoV-2 specific T cells has been initiated at the Center for Cell and Gene Therapy, Baylor College of Medicine (BCM), Texas Children's Hospital, and Houston Methodist Hospital.
Viral Infections in Immunocompromised Patients
In healthy individuals, virus-specific T cells (VSTs) from the bodys natural defense system provide protection against numerous disease-causing viruses. However, in patients with a weakened immune system these viruses may be uncontrolled. Viral diseases are common and can cause potentially devastating and life-threatening consequences in immunocompromised patients. For example, up to 90% of patients will reactivate at least one virus following an allogeneic stem cell transplant and two-thirds of these patients reactivate more than one virus, resulting in significant and prolonged morbidity, hospitalization, and premature death. Typically, when viruses infect immunocompromised patients, standard antiviral treatment does not address the underlying problem of a weakened immune system and therefore many patients suffer with life-threatening outcomes such as multi-organ damage and failure, and even death.
Viralym-M (ALVR105) is an allogeneic, off-the-shelf, multi-virus specific investigational T-cell therapy targeting five devastating viral pathogens: BK virus, cytomegalovirus, adenovirus, Epstein-Barr virus, and human herpesvirus 6. Viralym-M has the potential to transform care for transplant recipients as well as individuals who are at high risk for opportunistic viral infections by reducing or preventing disease morbidity and dramatically improving patient outcomes. Three pivotal and proof-of-concept clinical (POC) trials are ongoing and actively recruiting patients in indications such as treatment of virus-associated hemorrhagic cystitis and multi-virus prevention following allo-HSCT, and preemptive treatment of BK viremia in adult kidney transplant recipients. Additional pivotal and POC trials are expected to initiate for the treatment of CMV and the treatment of AdV in allo-HSCT recipients and in CMV for solid organ transplant recipients, respectively. For more information on the ongoing clinical trials visit clinicaltrials.gov.
Viralym-M has received Regenerative Medicine Advanced Therapy (RMAT) designation from the U.S. Food and Drug Administration (FDA), as well as PRIority MEdicines (PRIME) and Orphan Drug Designations (ODD) from the European Medicines Agency.
AlloVir is a leading late clinical-stage cell therapy company with a focus on restoring natural immunity against life-threatening viral diseases in pediatric and adult patients with weakened immune systems. The companys innovative and proprietary technology platforms leverage off-the-shelf, allogeneic, multi-virus specific T-cells targeting devastating viruses for patients with T-cell deficiencies who are at risk from the life-threatening consequences of viral diseases. AlloVirs technology and manufacturing process enables the potential for the treatment and prevention of a spectrum of devastating viruses with each single allogeneic cell therapy. The company is advancing multiple mid- and late-stage clinical trials across its product portfolio. For more information visit http://www.allovir.com.
This press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995, as amended, including, without limitation, statements regarding AlloVirs development and regulatory status of our product candidates, the planned conduct of its preclinical studies and clinical trials and its prospects for success in those studies and trials, and its strategy, business plans and focus. The words may, will, could, would, should, expect, plan, anticipate, intend, believe, estimate, predict, project, potential, continue, target and similar expressions are intended to identify forward-looking statements, although not all forward-looking statements contain these identifying words. Any forward-looking statements in this press release are based on managements current expectations and beliefs and are subject to a number of risks, uncertainties and important factors that may cause actual events or results to differ materially from those expressed or implied by any forward-looking statements contained in this press release, including, without limitation, those related to AlloVirs financial results, the timing for the initiation and successful completion of AlloVirs clinical trials of its product candidates, whether and when, if at all, AlloVirs product candidates will receive approval from the U.S. Food and Drug Administration, or FDA, or other foreign regulatory authorities, competition from other biopharmaceutical companies, the impact of the COVID-19 pandemic on AlloVirs product development plans, supply chain, and business operations and other risks identified in AlloVirs SEC filings. AlloVir cautions you not to place undue reliance on any forward-looking statements, which speak only as of the date they are made. AlloVir disclaims any obligation to publicly update or revise any such statements to reflect any change in expectations or in events, conditions or circumstances on which any such statements may be based, or that may affect the likelihood that actual results will differ from those set forth in the forward-looking statements. Any forward-looking statements contained in this press release represent AlloVirs views only as of the date hereof and should not be relied upon as representing its views as of any subsequent date.
Jasper Therapeutics Announces Launch of New Clinical Trial with National Heart, Lung, and Blood Institute to Evaluate JSP191 in Sickle Cell Disease -…
REDWOOD CITY, Calif.--(BUSINESS WIRE)--Jasper Therapeutics, Inc., a biotechnology company focused on hematopoietic cell transplant therapies, today announced the launch of a Phase 1/2 clinical trial to evaluate JSP191, Jaspers first-in-class anti-CD117 monoclonal antibody, as a targeted, non-toxic conditioning regimen prior to allogeneic transplant for sickle cell disease (SCD). Jasper Therapeutics and the National Heart, Lung, and Blood Institute (NHLBI) have entered into a clinical trial agreement in which NHLBI will serve as the Investigational New Drug (IND) sponsor for this study.
SCD is a lifelong inherited blood disorder that affects hemoglobin, a protein in red blood cells that delivers oxygen to tissues and organs throughout the body. Approximately 300,000 infants are born with SCD annually worldwide, and the number of cases is expected to significantly increase. Currently, hematopoietic stem cell transplantation (HSCT) is the only cure available for SCD.
"This clinical trial agreement with the NHLBI expands the development of JSP191 for transplant conditioning and could bring curative transplants to more patients in need," said Kevin N. Heller, M.D., Executive Vice President, Research and Development, of Jasper Therapeutics. "We look forward to collaborating with the NHLBI and learning more about the potential for JSP191 in patients living with sickle cell disease."
JSP191 (formerly AMG 191) is a first-in-class humanized monoclonal antibody in clinical development as a conditioning agent that clears hematopoietic stem cells from the bone marrow. JSP191 binds to human CD117, a receptor for stem cell factor (SCF) that is expressed on the surface of hematopoietic stem and progenitor cells. The interaction of SCF and CD117 is required for stem cells to survive. JSP191 blocks SCF from binding to CD117 and disrupts critical survival signals in stem cells leading to cell death. This creates space in the bone marrow for engraftment of donor or gene-corrected transplanted stem cells.
Preclinical studies have shown that JSP191, as a single agent, safely depletes normal and diseased hematopoietic stem cells, including in animal models of severe combined immunodeficiency (SCID), myelodysplastic syndromes (MDS), and sickle cell disease (SCD). Treatment with JSP191 creates the space needed for transplanted normal donor or gene-corrected hematopoietic stem cells to successfully engraft in the host bone marrow. To date, JSP191 has been evaluated in more than 90 healthy volunteers and patients.
JSP191 is currently being evaluated in two separate Jasper Therapeutics-sponsored clinical studies in hematopoietic cell transplant. The first clinical study is evaluating JSP191 as a sole conditioning agent in a Phase 1/2 dose-escalation and expansion trial to achieve donor stem cell engraftment in patients undergoing hematopoietic cell transplant for SCID. Blood stem cell transplantation offers the only potentially curative therapy for SCID. JSP191 is also being evaluated in combination with another conditioning regimen in a Phase 1 study in patients with MDS or acute myeloid leukemia (AML) who are receiving hematopoietic cell transplant. For more information about the design of these clinical trials, visit http://www.clinicaltrials.gov (NCT02963064 and NCT04429191).
Additional studies are planned to advance JSP191 as a conditioning agent for patients with other rare and ultra-rare monogenic disorders and autoimmune diseases.
About Jasper Therapeutics
Jasper Therapeutics is a biotechnology company focused on the development of novel curative therapies based on the biology of the hematopoietic stem cell. The companys lead compound, JSP191, is in clinical development as a conditioning antibody that clears hematopoietic stem cells from bone marrow in patients undergoing a hematopoietic cell transplant. This first-in-class conditioning antibody is designed to enable safer and more effective curative hematopoietic cell transplants and gene therapies. For more information, please visit us at jaspertherapeutics.com.
Stem Cell Therapy Market Size and Forecast (2021-2027) | By Top Leading Players NuVasive, Osiris Therapeutics, JCR Pharmaceutical, Pharmicell, Chiesi…
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New Jersey, United States,- The Stem Cell Therapy Market research report produced by Market Research Intellect focuses on some key aspects of the market such as profitability, market share, key regions, production, and key players. This Stem Cell Therapy report also provides the readers with detailed figures which have been used to evaluate the Stem Cell Therapy market in the historic year and its expected growth in the years to come. In addition, the analysis also predicts the CAGR at which Stem Cell Therapy is expected to grow and the main drivers of market growth. All current technological innovations, industry trends, and market data are presented in the Global Stem Cell Therapy report for the forecast period. An in-depth understanding of the Stem Cell Therapy industry based on the market size, growth, opportunity, and development plans suggested in the analysis of the report.
Additionally, this report offers a comprehensive analysis of the supply chain, regional marketing, opportunities, challenges, and market factors to accurately predict the global Stem Cell Therapy market. The Stem Cell Therapy report also provides an in-depth analysis of the research methodology and approach, data sources and authors of the study. The Stem Cell Therapy report also includes detailed production data such as interview log, gross margins, shipping, and business distribution, which can help the consumer understand the competitive landscape.
Additionally, the report provides an in-depth assessment of the Stem Cell Therapy market, highlighting data on various aspects including opportunities, market drivers, and threats. However, this data can help suppliers make the right decision before investing in the Stem Cell Therapy market. In addition, the research report has been developed based on an in-depth analysis of the target market as well as information from market professionals. The report focuses on the spanning market landscape and growth prospects for the forecast period. The Stem Cell Therapy Market Watch also provides a comprehensive overview of the major retailers operating in the target market.
The most important Companies in this Stem Cell Therapy Market Watch are:
Stem Cell Therapy Market Segment Analysis
The study report contains certain segments by type and application. Each type provides information on products in the forecast period from 2015 to 2027. The application segment also provides consumption information for the forecast period from 2015 to 2027. Understanding the segments will help determine the importance of various factors for market growth.
The report further studies the market segmentation based on the types of products offered in the market and their end-uses/uses.
While segmenting the Market by Stem Cell Therapy Types, the Report includes:
While segmenting the Market by Stem Cell Therapy Applications, the report covers the following application areas:
Stem Cell Therapy Market Report Scope
Business opportunities in the following regions and countries:
? North America (USA, Canada and Mexico)
? Europe (Germany, Great Britain, France, Italy, Russia, Spain and Benelux countries)
? Asia Pacific (China, Japan, India, Southeast Asia, and Australia)
? Latin America (Brazil, Argentina and Colombia)
Key Points of the Stem Cell Therapy Market Report:
Stem Cell Therapy market research coverage: It includes key market segments, information on key manufacturers, the volume of supply in the reporting years, the global Stem Cell Therapy market and research objectives. It also contains links to the departmental study identified in the report based on item type and applications.
Stem Cell Therapy Market Overview: This area focuses on key research, market pace, serious situation, market drivers, models and problems despite the obviously visible signs.
Stem Cell Therapy Market Production by Regions: The report has information related to imports and travel expenses, revenue, creation, and key players of the respective local markets that is currently being reviewed.
Stem Cell Therapy Manufacturer Market Profile: This section provides a detailed analysis of each market player. This part also features SWOT research, items, generation, value, limit and other necessary elements of a single player.
How will the report help your business grow?
? This document provides statistics on the value (in USD) and size (in units) of the Stem Cell Therapy industry from 2021 to 2027.
? The report also details major competitors in the market that will have a greater impact on Stem Cell Therapys business.
? Comprehensive understanding of the fundamental trends affecting each sector despite the greatest threat, the latest technologies and opportunities that can create a global Stem Cell Therapy market for both supply and demand.
? The report will help the client identify the key results of the major market players or rulers of the Stem Cell Therapy sector.
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In a zebrafish model, researchers have found that the protein NAPMT can trigger muscle stem cells to proliferate and heal muscle damage.
Researchers at the Australian Regenerative Medicine Institute at Monash University, Australia, have discovered a factor that triggers muscle stem cells to proliferate and heal. In a mouse model of severe muscle damage, injections of this naturally occurring protein led to the complete regeneration of muscle and the return of normal movement after severe muscle trauma.
According to the researchers, growing these stem cells in the lab and then using them to therapeutically replace damaged muscle has been difficult.
The scientists studied the regeneration of skeletal muscle in zebrafish, which are transparent allowing the scientists to witness regeneration in living muscle.
By studying the cells that migrated to a muscle injury in these fish the scientists identified a group of immune cells, called macrophages, which appeared to have a role in triggering the muscle stem cells to regenerate.
What we saw were macrophages literally cuddling the muscle stem cells, which then started to divide and proliferate. Once they started this process, the macrophage would move on and cuddle then next muscle stem cell and pretty soon the wound would heal, said lead researcher of the study Professor Peter Currie. He added that macrophages flock to any injury or infection site in the body, removing debris and promoting healing.
The research team found that there were eight genetically different types of macrophages in the injury site and that one type, in particular, was the cuddler. Further investigation revealed that this macrophage released a substance called NAMPT.
By removing these macrophages from the zebrafish and adding the NAMPT to the aquarium water the scientists found they could stimulate the muscle stem cells to grow and heal, effectively replacing the need for the macrophages.
The team highlight that experiments placing a hydrogel patch containing NAPMT into a mouse model of severe muscle wasting led to what Professor Currie called significant replacement of the damaged muscle. The researchers are now in discussions with a number of biotech companies about taking NAMPT to clinical trials for the use of this compound in the treatment of muscle disease and injury.
The findings are published inNature.
Leukemia is a type of cancer that affects the blood and bone marrow, where blood cells are formed. All types of leukemia cause rapid, uncontrolled growth of abnormal bone marrow and blood cells.
The main differences between the types include how fast the disease progresses and the types of cells it affects.
There are four main types of leukemia, which we describe in detail below:
Lymphocytic leukemia affects the lymphocytes, a type of white blood cell. Myeloid leukemia can affect the white blood cells, red blood cells, and platelets.
According to the National Cancer Institute, roughly 1.5% of people in the United States will receive a leukemia diagnosis at some point.
In this article, explore the four main types, their symptoms, the treatment options available, and the outlook.
The full name of this type of cancer is acute lymphocytic leukemia, and acute means that it grows quickly. Lymphocytic means that it forms in underdeveloped white blood cells called lymphocytes.
The disease starts in the bone marrow, which produces stem cells that develop into red and white blood cells and platelets.
In a healthy person, the bone marrow does not release these cells until they are fully developed. In someone with ALL, the bone marrow releases large quantities of underdeveloped white blood cells.
There are several subtypes of ALL, and the subtype may influence the best course of treatment and the prognosis.
One subtype is B-cell ALL. This begins in the B lymphocytes, and it is the most common form of ALL in children.
Another subtype is T-cell ALL. It can cause the thymus, a small organ at the front of the windpipe, to become enlarged, which can lead to breathing difficulties.
Overall, because ALL progresses quickly, swift medical intervention is key.
As research from 2020 acknowledges, healthcare providers still do not know what causes ALL. It may occur due to genetic factors or exposure to:
Although genetic factors may play a role, ALL is not a familial disease.
Learn more about ALL here.
ALL is the most common form of leukemia in children.
The risk of developing it is highest in children under 5 years old. The prevalence slowly rises again in adults over 50.
ALL symptoms can be nonspecific difficult to distinguish from those of other illnesses.
They may include:
In a person with AML, the bone marrow makes abnormal versions of platelets, red blood cells, and white blood cells called myeloblasts.
The full name of this disease is acute myeloid leukemia, and acute refers to the fact that it is fast-growing.
It forms in one of the following types of bone marrow cell:
Doctors classify AML by subtype, depending on:
AML can be difficult to treat and requires prompt medical attention.
Learn more about AML here.
The most common risk factor is myelodysplastic syndrome, a form of blood cancer that keeps the body from producing enough healthy blood cells.
Other factors that increase the risk of developing AML include:
Most people who develop AML are over 45. It is one of the most common types of leukemia in adults, though it is still rare, compared with other cancers.
It is also the second most common form of leukemia in children.
Symptoms of AML can vary and may include:
CLL is the most common form of leukemia among adults in the U.S. and other Western countries.
There are two types. One progresses slowly, and it causes the body to have high levels of characteristic lymphocytes, but only slightly low levels of healthy red blood cells, platelets, and neutrophils.
The other type progresses more quickly and causes a significant reduction in levels of all healthy blood cells.
In someone with CLL, the lymphocytes often look fully formed but are less able to fight infection than healthy white blood cells. The lymphocytes tend to build up very slowly, so a person might have CLL for a long time before experiencing symptoms.
Learn more about CLL here.
Genetic factors are the most likely cause. Others might include:
CLL is rare in children. It typically develops in adults aged 70 or over. However, it can affect people as young as 30.
CLL typically causes no early symptoms. When symptoms are present, they may include:
Also, 5090% of people with CLL have swollen lymph nodes.
CML is a slow-growing type of leukemia that develops in the bone marrow.
The full name of CML is chronic myeloid leukemia. As the American Cancer Society explain, a genetic change takes place in the early forms of the myeloid cells, and this eventually results in CML cells.
These leukemia cells then grow, divide, and enter the blood.
CML occurs due to a rearrangement of genetic material between the chromosomes 9 and 22.
This rearrangement fuses a part of the ABL1 gene from chromosome 9 with the BCR gene from chromosome 22, called the Philadelphia chromosome. The result of this fusion is called BCR-ABL1.
BCR-ABL1 produces a protein that promotes cell division and stops apoptosis, the process of cell death, which typically removes unneeded or damaged cells.
The cells keep dividing and do not self-destruct, resulting in an overproduction of abnormal cells and a lack of healthy blood cells.
This occurs during the persons lifetime and is not inherited.
CML typically affects adults. People aged 65 and older make up almost half of those who receive a CML diagnosis.
The symptoms of CML are unclear, but they may include:
The symptoms may vary, depending on the type of leukemia. Overall, a person should get in touch with a doctor if they experience:
Learn more about the symptoms of leukemia here.
Treatment for ALL typically involves three basic phases: induction, consolidation, and maintenance. We describe these in detail below.
Treatment for AML involves the first two phases. The induction phase may include treatment with the chemotherapy drugs cytarabine (Cytosar-U) and daunorubicin (Cerubidine) or idarubicin (Idamycin). The doctor may also recommend targeted drugs.
The goal of this phase is to kill the leukemia cells, causing the cancer to go into remission, using chemotherapy.
The doctor may recommend:
People having chemotherapy may need to see their doctors frequently and spend time in the hospital, due to the risk of serious infections and complications.
This phase of the treatment lasts for about 1 month.
Even if the treatment so far has led to remission, cancer cells may be hiding in the body, so more treatment is necessary.
The consolidation phase may involve taking high doses of chemotherapy. A doctor may also recommend targeted drugs or stem cell transplants.
This phase, consisting of ongoing chemotherapy treatments, usually lasts for 2 years.
Since CLL tends to progress slowly, and its treatment can have unpleasant side effects, some people with this condition go through a phase of watchful waiting before starting the treatment.
For a person with CML, the focus is often on providing the right treatment for the phase of the illness. To do this, a doctor considers how quickly the leukemia cells are building up and the extent of the symptoms. Stem cell transplants can be effective, but further treatment is necessary.
Overall, the initial treatment tends to include monoclonal antibodies, targeted drugs, and chemotherapy.
If the only concern is an enlarged spleen or swollen lymph nodes, the person may receive radiation or surgery.
If there are high numbers of CLL cells, the doctor may suggest leukapheresis, a treatment that lowers the persons blood count. This is only effective for a short time, but it allows the chemotherapy to start working.
For people with high-risk disease, doctors may recommend stem cell transplants.
A persons prognosis depends on the type of leukemia.
Learn more about survival rates for people with leukemia here.
About 8090% of adults with ALL experience complete remission for a while during treatment. And with treatment, most children recover from the disease.
Relapses are common in adults, so the overall cure rate is 40%. However, factors specific to each person play a role.
The older a person is when they receive an AML diagnosis, the more difficult it is to treat.
More than 25% of adults who achieve remission live for 3 years or more after treatment for AML.
A person may live for a long time with CLL.
Treatments can help keep the symptoms under control and prevent the disease from spreading. However, there is no cure.
Stem cell transplants can cure CML. However, this treatment is very invasive and is not suitable for most people with CML.
The United Kingdoms National Health Service estimate that 70% of males and 75% of females live for at least 5 years after receiving a CML diagnosis.
The earlier a person receives the diagnosis, the better their outlook.
Leukemia is a type of cancer that affects the blood and bone marrow. It can affect people of all ages.
There are four main types of leukemia. They differ based on how quickly they progress and the types of cells they affect.
Treatments for all types of leukemia continue to improve, helping people live longer and more fully with this condition.
See the original post:
Types of leukemia: Prevalence, treatment options, and prognosis - Medical News Today
Network-based screen in iPSC-derived cells reveals therapeutic candidate for heart valve disease – Science
Machine learning for medicine
Small-molecule screens aimed at identifying therapeutic candidates traditionally search for molecules that affect one to several outputs at most, limiting discovery of true disease-modifying drugs. Theodoris et al. developed a machine-learning approach to identify small molecules that broadly correct gene networks dysregulated in a human induced pluripotent stem cell disease model of a common form of heart disease involving the aortic valve. Gene network correction by the most efficacious therapeutic candidate generalized to primary aortic valve cells derived from more than 20 patients with sporadic aortic valve disease and prevented aortic valve disease in vivo in a mouse model.
Science, this issue p. eabd0724
Determining the gene-regulatory networks that drive human disease allows the design of therapies that target the core disease mechanism rather than merely managing symptoms. However, small molecules used as therapeutic agents are traditionally screened for their effects on only one to several outputs at most, from which their predicted efficacy on the disease as a whole is extrapolated. In silico correlation of disease network dysregulation with pathways affected by molecules in surrogate cell types is limited by the relevance of the cell types used and by not directly testing compounds in patient cells.
In principle, mapping the architecture of the dysregulated network in disease-relevant cells differentiated from patient-derived induced pluripotent stem cells (iPSCs) and subsequent screening for small molecules that broadly correct the abnormal gene network could overcome this obstacle. Specifically, targeting normalization of the core regulatory elements that drive the disease process, rather than correction of peripheral downstream effectors that may not be disease modifying, would have the greatest likelihood of therapeutic success. We previously demonstrated that haploinsufficiency of NOTCH1 can cause calcific aortic valve disease (CAVD), the third most common form of heart disease, and that the underlying mechanism involves derepression of osteoblast-like gene networks in cardiac valve cells. There is no medical therapy for CAVD, and in the United States alone, >100,000 surgical valve replacements are performed annually to relieve obstruction of blood flow from the heart. Many of these occur in the setting of a congenital aortic valve anomaly present in 1 to 2% of the population in which the aortic valve has two leaflets (bicuspid) rather than the normal three leaflets (tricuspid). Bicuspid valves in humans can also be caused by NOTCH1 mutations and predispose to early and more aggressive calcification in adulthood. Given that valve calcification progresses with age, a medical therapy that could slow or even arrest progression would have tremendous impact.
We developed a machine-learning approach to identify small molecules that sufficiently corrected gene network dysregulation in NOTCH1-haploinsufficient human iPSC-derived endothelial cells (ECs) such that they classified similar to NOTCH1+/+ ECs derived from gene-corrected isogenic iPSCs. We screened 1595 small molecules for their effect on a signature of 119 genes representative of key regulatory nodes and peripheral genes from varied regions of the inferred NOTCH1-dependent network, assayed by targeted RNA sequencing (RNA-seq). Overall, eight molecules were validated to sufficiently correct the network signature such that NOTCH1+/ ECs classified as NOTCH1+/+ by the trained machine-learning algorithm. Of these, XCT790, an inverse agonist of estrogen-related receptor (ERR), had the strongest restorative effect on the key regulatory nodes SOX7 and TCF4 and on the network as a whole, as shown by full transcriptome RNA-seq.
Gene network correction by XCT790 generalized to human primary aortic valve ECs derived from explanted valves from >20 patients with nonfamilial CAVD. XCT790 was effective in broadly restoring dysregulated genes toward the normal state in both calcified tricuspid and bicuspid valves, including the key regulatory nodes SOX7 and TCF4.
Furthermore, XCT790 was sufficient to prevent as well as treat already established aortic valve disease in vivo in a mouse model of Notch1 haploinsufficiency on a telomere-shortened background. XCT790 significantly reduced aortic valve thickness, the extent of calcification, and echocardiographic signs of valve stenosis in vivo. XCT790 also reduced the percentage of aortic valve cells expressing the osteoblast transcriptional regulator RUNX2, indicating a reduction in the osteogenic cell fate switch underlying CAVD. Whole-transcriptome RNA-seq in treated aortic valves showed that XCT790 broadly corrected the genes dysregulated in Notch1-haploinsufficient mice with shortened telomeres, and that treatment of diseased aortic valves promoted clustering of the transcriptome with that of healthy aortic valves.
Network-based screening that leverages iPSC and machine-learning technologies is an effective strategy to discover molecules with broadly restorative effects on gene networks dysregulated in human disease that can be validated in vivo. XCT790 represents an entry point for developing a much-needed medical therapy for calcification of the aortic valve, which may also affect the highly related and associated calcification of blood vessels. Given the efficacy of XCT790 in limiting valve thickening, the potential for XCT790 to alter the progression of childhood, and perhaps even fetal, valve stenosis also warrants further study. Application of this strategy to other human models of disease may increase the likelihood of identifying disease-modifying candidate therapies that are successful in vivo.
A gene networkbased screening approach leveraging human disease-specific iPSCs and machine learning identified a therapeutic candidate, XCT790, which corrected the network dysregulation in genetically defined iPSC-derived endothelial cells and primary aortic valve endothelial cells from >20 patients with sporadic aortic valve disease. XCT790 was also effective in preventing and treating a mouse model of aortic valve disease.
Mapping the gene-regulatory networks dysregulated in human disease would allow the design of network-correcting therapies that treat the core disease mechanism. However, small molecules are traditionally screened for their effects on one to several outputs at most, biasing discovery and limiting the likelihood of true disease-modifying drug candidates. Here, we developed a machine-learning approach to identify small molecules that broadly correct gene networks dysregulated in a human induced pluripotent stem cell (iPSC) disease model of a common form of heart disease involving the aortic valve (AV). Gene network correction by the most efficacious therapeutic candidate, XCT790, generalized to patient-derived primary AV cells and was sufficient to prevent and treat AV disease in vivo in a mouse model. This strategy, made feasible by human iPSC technology, network analysis, and machine learning, may represent an effective path for drug discovery.