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News Article | March 2, 2017
Site: www.eurekalert.org

(New York, NY - March 2, 2017) --Mount Sinai researchers have discovered that a rheumatoid arthritis drug can block a metabolic pathway that occurs in tumors with a common cancer-causing gene mutation, offering a new possible therapy for aggressive cancers with few therapeutic options, according to a study to be published in Cancer Discovery. Ramon Parsons, MD, PhD, Ward-Coleman Chair in Cancer Research and Chair of the Department of Oncological Sciences at the Icahn School of Medicine at Mount Sinai, led a team that studied how a mutation of the PTEN gene rewires a metabolic pathway in tumors, channeling increased amounts of the amino acid glutamine into the pathway, speeding up DNA production, and causing uncontrolled growth of the tumor. The team discovered that leflunomide, an oral rheumatoid arthritis drug approved by the U.S. Food and Drug Administration, blocks an enzyme in this pathway and damages the DNA created through the pathway, killing PTEN mutant cancer cells while leaving healthy cells untouched. Parsons and his team transplanted human breast cancer cells into mice to test leflunomide's efficacy. Leflunomide drastically reduced the breast cancer tumors in the mice. "Finding successful targeted therapies for cancer is a challenging but important goal in the face of insufficient treatment options," said Dr. Parsons, who discovered the PTEN gene. "Targeted therapies that are tumor-specific are much needed, and identifying changes based on specific tumor suppressor or oncogene alterations will facilitate this effort. Due to the high mutation rate of PTEN in cancer, the effects of PTEN could be at the heart of targeted therapy." This discovery has implications in aggressive cancers with the PTEN mutation and few treatment options such as triple negative breast cancer, prostate cancer, endometrial cancer, and glioblastoma, a brain cancer. Dr. Parsons hopes to create a clinical trial to further test leflunomide in patients with breast and colon cancer. This research was funded by NCI R01CA082783, R01CA155117, and P01CA97403 (R.P.), and partially supported by NIH grants 5P01CA120964 and 5P30CA006516 (J.MA.), and R01 GM041890 and the Breast Cancer Research Foundation (L.C.). The Mount Sinai Health System is an integrated health system committed to providing distinguished care, conducting transformative research, and advancing biomedical education. Structured around seven hospital campuses and a single medical school, the Health System has an extensive ambulatory network and a range of inpatient and outpatient services--from community-based facilities to tertiary and quaternary care. The System includes approximately 7,100 primary and specialty care physicians; 12 joint-venture ambulatory surgery centers; more than 140 ambulatory practices throughout the five boroughs of New York City, Westchester, Long Island, and Florida; and 31 affiliated community health centers. Physicians are affiliated with the renowned Icahn School of Medicine at Mount Sinai, which is ranked among the highest in the nation in National Institutes of Health funding per investigator. The Mount Sinai Hospital is on the "Honor Roll" of best hospitals in America, ranked No. 15 nationally in the 2016-2017 "Best Hospitals" issue of U.S. News & World Report. The Mount Sinai Hospital is also ranked as one of the nation's top 20 hospitals in Geriatrics, Gastroenterology/GI Surgery, Cardiology/Heart Surgery, Diabetes/Endocrinology, Nephrology, Neurology/Neurosurgery, and Ear, Nose & Throat, and is in the top 50 in four other specialties. New York Eye and Ear Infirmary of Mount Sinai is ranked No. 10 nationally for Ophthalmology, while Mount Sinai Beth Israel, Mount Sinai St. Luke's, and Mount Sinai West are ranked regionally. Mount Sinai's Kravis Children's Hospital is ranked in seven out of ten pediatric specialties by U.S. News & World Report in "Best Children's Hospitals." For more information, visit http://www. or find Mount Sinai on Facebook, Twitter and YouTube.


News Article | February 28, 2017
Site: www.prweb.com

The Colon Cancer Foundation, a New York-based nonprofit dedicated to the fight against colorectal cancer, today announced a massive new public awareness campaign called “Protect Your Butt!” The new campaign will focus on the Colon Cancer Foundation’s commitment to saving lives through colon cancer awareness, prevention, and translational research programs focused on a cure and optimal care for those most affected by this disease. The campaign also will break through all stigmas and fears around colon cancer and screening, and significantly increase awareness of the disease. Over 50,000 Americans die from colon cancer each year making colon cancer the #2 cancer in the USA. The kickoff event for the Protect Your Butt! Campaign is the “World’s Largest Booty Shake,” scheduled for Wednesday, March 1, in Central Park. The World’s Largest Booty Shake is expected to draw thousands of colon cancer survivors, those passionate about the cause, and more. “Our kick-off booty shake event is just the beginning,” said Dr. Thomas Weber, the foundation’s founder. “Too many people avoid talking about colon cancer because they are unaware or scared. Our new multifaceted Protect Your Butt! Campaign will reach millions of Americans through large events, social media, email marketing, and television. We are literally going to shake this up, raise money for the cure, and save lives.” The World’s Largest Booty Shake is sponsored by the Mount Sinai Health System, and partners of the Colon Cancer Foundation, Epigenomics and Bracco. These three leading health providers focus on colon cancer prevention, screening, and research. “We are proud to be the presenting sponsor for the World’s Largest Booty Shake,” said David Greenwald, MD, Director of Clinical Gastroenterology and Endoscopy at The Mount Sinai Hospital. “Colon cancer is the No. 2 cancer killer in the United States. By raising awareness and encouraging screening, we can save more lives and change this statistic.” The World’s Largest Booty Shake event starts at noon on Wednesday, March 1, at the Central Park Band Shell, south of Bethesda Terrace between 66th and 72nd streets. Headlining the World’s Largest Booty Shake will be rising R&B star Mark MK and DJ Theo, who will debut the new R&B single: “PYB – Protect Your Butt!” The new single can be downloaded on iTunes, Spotify, Tidal, Amazon, and other services, and part of the proceeds will go towards colon cancer research. All money raised during the Protect Your Butt! Campaign helps to raise awareness of the nation’s second leading cancer killer and provides funds for colorectal cancer research, education, and prevention programs for the underserved. For more information about the Colon Cancer Foundation, visit http://www.coloncancerchallenge.org. The Colon Cancer Foundation (coloncancerchallenge.org) is a 501(c)(3) not-for-profit organization registered in New York state and listed by the Federal IRS as a public charity dedicated to reducing colorectal cancer incidence and death. Its mission includes supporting research into the causes and cures for colorectal cancer, increasing public awareness, educating the public about the importance of early detection and forming strategic partnerships in the fight against colorectal cancer. The Mount Sinai Health System is an integrated health system committed to providing distinguished care, conducting transformative research, and advancing biomedical education. Structured around seven hospital campuses and a single medical school, the Health System has an extensive ambulatory network and a range of inpatient and outpatient services—from community-based facilities to tertiary and quaternary care. The System includes approximately 7,100 primary and specialty care physicians; 12 joint-venture ambulatory surgery centers; more than 140 ambulatory practices throughout the five boroughs of New York City, Westchester, Long Island, and Florida; and 31 affiliated community health centers. Physicians are affiliated with the renowned Icahn School of Medicine at Mount Sinai, which is ranked among the highest in the nation in National Institutes of Health funding per investigator. The Mount Sinai Hospital is in the “Honor Roll” of best hospitals in America, ranked No. 15 nationally in the 2016-2017 “Best Hospitals” issue of U.S. News & World Report. The Mount Sinai Hospital is also ranked as one of the nation’s top 20 hospitals in Geriatrics, Gastroenterology/GI Surgery, Cardiology/Heart Surgery, Diabetes/Endocrinology, Nephrology, Neurology/Neurosurgery, and Ear, Nose & Throat, and is in the top 50 in four other specialties. New York Eye and Ear Infirmary of Mount Sinai is ranked No. 10 nationally for Ophthalmology, while Mount Sinai Beth Israel, Mount Sinai St. Luke's, and Mount Sinai West are ranked regionally. Mount Sinai’s Kravis Children’s Hospital is ranked in seven out of ten pediatric specialties by U.S. News & World Report in "Best Children's Hospitals." For more information, visit http://www.mountsinai.org, or find Mount Sinai on Facebook, Twitter and YouTube. Epigenomics is a molecular diagnostics company focused on blood-based detection of cancers using its proprietary DNA methylation biomarker technology. The company develops and commercializes diagnostic products across multiple cancer indications with high medical need. Epigenomics' lead product, Epi proColon, is a blood-based screening test for the detection of colorectal cancer. Epi proColon has received approval from the U.S. Food and Drug Administration (FDA) and is currently marketed in the United States, Europe, and China and selected other countries. Epigenomics’ second product, Epi proLung®, is in development as a blood-based test for lung cancer detection. Bracco Imaging S.p.A., part of the Bracco Group, is one of the world’s leading companies in the diagnostic imaging business. Headquartered in Milan, Italy, Bracco Imaging develops, manufactures and markets diagnostic imaging agents and solutions that meet medical needs. Bracco Imaging offers a product and solution portfolio for all key diagnostic imaging modalities: X-ray Imaging (including Computed Tomography-CT, Interventional Radiology, and Cardiac Catheterization), Magnetic Resonance Imaging (MRI), Contrast Enhanced Ultrasound (CEUS), and Nuclear Medicine through radioactive tracers. The diagnostic imaging portfolio is completed by a range of medical devices and advanced administration systems for contrast imaging products.


News Article | February 23, 2017
Site: www.eurekalert.org

Diabetes is a leading cause of kidney disease, a serious, often fatal complication that is difficult to diagnose in early, potentially treatable stages. Now, a research team at the Icahn School of Medicine at Mount Sinai has revealed biological pathways involved in diabetic kidney disease, providing hope that both early diagnostic tests and targeted treatment can be designed. The study, published in Diabetes, shows that oxidative stress in the "power plants" within a population of kidney cells progressively impairs the ability of the bean-shaped organs to strain blood for waste products and produce urine. The research team also found a cellular receptor that can be blocked to modulate that stress reaction. Blocking that receptor saved the kidneys in mice genetically destined to develop diabetic kidney failure. About 30 percent of patients with type 1 (juvenile onset) diabetes and 10 to 40 percent of those with type 2 (adult onset) diabetes eventually will suffer from kidney failure, according to the National Kidney Foundation. When that happens, patients must turn to dialysis or kidney transplantation, if available. "Diabetic kidney disease is one of the major causes of death in diabetic patients, and is also the leading single cause of end-stage renal disease in the United States," says the study's senior investigator, Ilse S. Daehn, PhD, Assistant Professor of Medicine (Nephrology) at the Icahn School of Medicine at Mount Sinai. "Our findings open new diagnostic opportunities for early detection and potential therapeutic strategies to protect against further renal damage in patients." The study's findings essentially offer a "fundamental paradigm shift in our understanding of the development and treatment of diabetic kidney disease," says Dr. Daehn, who is also a member of The Charles Bronfman Institute for Personalized Medicine. Investigators focused on the kidney's glomerulus -- globular bodies, full of capillaries and other structures, that serve as the first stage and the key unit in the filtration of blood for waste products to be expelled in urine. The research team studied three different cell types that interact within the glomerulus, using two sets of mice. One group naturally develops diabetic kidney disease and the other group is naturally resistant to the disorder. They discovered that in mice prone to kidney disease, endothelial cells were affected. In these wafer-like cells, which form the inner lining of blood vessels, the mitochondria -- cellular subunits that act like power plants, producing energy -- were stressed, and so made excess amounts of reactive oxygen species (ROS). These are molecules that have important roles in cell signaling but, when overproduced, can damage cell proteins and DNA. This process begins to destroy podocytes, cells that wrap around and work with capillaries and the other cell types in the glomerulus. The glomerulus eventually becomes brittle, the capillaries collapse, and kidneys become leaky, shedding essential body proteins. Progressive damage leads to kidney failure, resulting in end-stage kidney disease. The research team was able to measure, in susceptible mice, molecules linked to excess ROS, suggesting that a biomarker could be developed that signals early development of kidney disease in humans. And knowing that ROS excess leads to kidney disease implies that agents that collect ROS molecules within the kidney might provide a potential therapy, Dr. Daehn says. Investigators then looked for "upstream" regulators of mitochondrial stress within the endothelium in the glomerulus and discovered a pathway that helps manage this oxidative stress. This pathway produced excess quantities of a cell receptor, endothelium receptor-A, as well as its ligand -- the protein that binds to the receptor. This discovery means that a small molecule that blocks the ligand from binding to its receptor might tamp down production of mitochondrial ROS, thus halting damage to the glomerulus, Dr. Daehn says. The researchers used an experimental small molecule, BQ-123, to specifically block this receptor and found that mice that were destined to develop diabetic kidney disease were spared from the disorder. Researchers tested their hypothesis by looking at urine and kidney biopsies from patients with diabetic kidney disease. They found molecules in the urine linked to oxidative stress and rapid disease progression, and biopsies that showed increased mitochondrial DNA damage and increased endothelium receptor-A expression. "These findings in human samples go a long way to substantiate our hypotheses, which is exciting because it represents a new way forward to understanding and treating diabetic kidney disease," Dr. Daehn says. Among other researchers from the Icahn School of Medicine who co-authored the study was senior investigator Erwin Böttinger,, MD, Professor of Medicine (Nephrology). The research team also includes investigators from Columbia University in New York and the University of Gothenberg in Sweden. The study was supported by National Institutes of Health Grants 5U01DK060995, 5R01DK056077 and R01DK097253. The authors report no conflicts of interest. The Mount Sinai Health System is an integrated health system committed to providing distinguished care, conducting transformative research, and advancing biomedical education. Structured around seven hospital campuses and a single medical school, the Health System has an extensive ambulatory network and a range of inpatient and outpatient services--from community-based facilities to tertiary and quaternary care. The System includes approximately 7,100 primary and specialty care physicians; 12 joint-venture ambulatory surgery centers; more than 140 ambulatory practices throughout the five boroughs of New York City, Westchester, Long Island, and Florida; and 31 affiliated community health centers. Physicians are affiliated with the renowned Icahn School of Medicine at Mount Sinai, which is ranked among the highest in the nation in National Institutes of Health funding per investigator. The Mount Sinai Hospital is in the "Honor Roll" of best hospitals in America, ranked No. 15 nationally in the 2016-2017 "Best Hospitals" issue of U.S. News & World Report. The Mount Sinai Hospital is also ranked as one of the nation's top 20 hospitals in Geriatrics, Gastroenterology/GI Surgery, Cardiology/Heart Surgery, Diabetes/Endocrinology, Nephrology, Neurology/Neurosurgery, and Ear, Nose & Throat, and is in the top 50 in four other specialties. New York Eye and Ear Infirmary of Mount Sinai is ranked No. 10 nationally for Ophthalmology, while Mount Sinai Beth Israel, Mount Sinai St. Luke's, and Mount Sinai West are ranked regionally. Mount Sinai's Kravis Children's Hospital is ranked in seven out of ten pediatric specialties by U.S. News & World Report in "Best Children's Hospitals." For more information, visit http://www. , or find Mount Sinai on Facebook, Twitter and YouTube.


News Article | February 15, 2017
Site: www.eurekalert.org

Scalp cooling can lessen some chemotherapy-induced hair loss - one of the most devastating hallmarks of cancer - in certain breast cancer patients, according to a new multicenter study from UC San Francisco, Weill Cornell Medicine and three other medical centers. A majority of the study's patients, all women with stage 1 or 2 breast cancer who underwent scalp cooling, retained more than half of their hair after completing chemotherapy, the investigators learned. The study, which tracks patients over five years, used standardized photographs to grade hair loss. The study will be published Feb. 14 in JAMA, the Journal of the American Medical Association. "Hair loss is almost universal among breast cancer patients receiving adjuvant chemotherapy and is one of the most distressing of adverse side effects," said first author Hope S. Rugo, MD, the corresponding author who led the study. Rugo is a UCSF professor of medicine specializing in breast cancer research and treatment, and director of the breast oncology and clinical trials education program at the UCSF Helen Diller Family Comprehensive Cancer Center. "We found that scalp cooling during commonly used chemotherapy regimens was well tolerated and was associated with significantly less hair loss, as well as improvement in several quality-of-life indicators," Rugo said. "While further research is needed, the data suggest that when scalp cooling is successful at decreasing hair loss, it could improve the treatment experience for women undergoing adjuvant chemotherapy for early-stage breast cancer." Breast cancer is the most common cancer in women around the world, both in developed countries and less developed ones, according to the World Health Organization. Scalp cooling has been used in more than 30 countries as a way to potentially prevent hair loss in patients receiving chemotherapy; in Europe it's been used for several decades. Two types of cooling caps are typically used: frozen caps that need to be replaced every half hour, or cooling systems that continually circulate coolants into a cap during the entire chemotherapy session. Scalp cooling is thought to reduce hair loss due to reduced delivery of chemotherapy to the scalp and hair follicle, Rugo said. The cold temperatures also are thought to slow the hair follicle cell division, making the cell less susceptible to the damaging effects of chemotherapy. In the United States, scalp cooling has been limited because of factors including insufficient scientific data and concern about the theoretic risk of scalp metastases. For the JAMA study, researchers investigated the effectiveness of one device: the DigniCap scalp cooling system manufactured by the Swedish public company Dignitana AB, which partly funded the research. In December 2015, based on preliminary results from the study, the U.S. Food and Drug Administration cleared the DigniCap for use in the U.S., the first and only cooling cap to date to receive such clearance. In the JAMA paper, 122 women with stage 1or stage 2 breast cancer were studied - all received non-anthracycline adjuvant chemotherapy, which generally causes severe hair loss. Of those women, 101 were enrolled in scalp cooling; 16 others, also undergoing chemotherapy but not scalp cooling, were in the control arm. Scalp cooling began 30 minutes prior to each chemotherapy cycle and involved a close fitting of the silicone cap on the patient's head, followed by an insulating neoprene cap. The silicone cap was then gradually cooled. The DigniCap is set to cool at 3 degrees Celsius (37 degrees Fahrenheit) with a temperature variance of plus or minus 2 degrees. Of 101 patients who underwent scalp cooling, 67 of them (66.3 percent) retained half or more of their hair, the authors wrote. In the parallel control group, all the patients lost their hair. Additionally, three of five quality-of-life measures were significantly better for the women who underwent scalp-cooling, including feeling more physically attractive. "Enabling a woman to preserve her hair during chemotherapy is empowering," said senior author Tessa Cigler, MD, MPH, an assistant professor of clinical medicine in the Weill Cornell Breast Center at Weill Cornell Medicine. "Scalp cooling allows patients to protect their privacy and maintain their self-esteem and sense of well-being. This study provides long-awaited evidence for an effective and practical scalp cooling method." The mean age of the cold cap patients was 53 years. Some 77 percent of the patients were white, 9 percent were black and nearly 11 percent were Asian. The study was conducted between August 2013 and October 2014. The average duration of chemotherapy was 2.3 months. Many of the patients reported mild headaches or scalp pain associated with the scalp cooling. Two patients discontinued scalp cooling due to feeling cold. There has been no evidence of scalp metastases in any patient after approximately 30 months of follow up. All patient follow up will continue for a total of five years. The study was funded partially by the Lazlo Tauber Family Foundation (awarded to UCSF); the Anne Moore Breast Cancer Research Fund (awarded to Weill Cornell Medicine); and the Friedman Family Foundation (awarded to Mount Sinai Beth Israel). Dignitana AB supported the design and conduct of the study, including collection, management, analysis and interpretation of the data. Study co-authors also included researchers from the Icahn School of Medicine at Mount Sinai, New York; Wake Forest Baptist Health Medical Center; and the Jonsson Comprehensive Cancer Center at UCLA. From UCSF, co-authors are Michelle E. Melisko, MD, and Laura Esserman, MD, MBA; from Weill Cornell Medicine, Anne Moore, MD, was also a co-author. A complete list of authors can be found in the paper. About UCSF: UC San Francisco (UCSF) is a leading university dedicated to promoting health worldwide through advanced biomedical research, graduate-level education in the life sciences and health professions, and excellence in patient care. It includes top-ranked graduate schools of dentistry, medicine, nursing and pharmacy; a graduate division with nationally renowned programs in basic, biomedical, translational and population sciences; and a preeminent biomedical research enterprise. It also includes UCSF Health, which comprises three top-ranked hospitals, UCSF Medical Center and UCSF Benioff Children's Hospitals in San Francisco and Oakland, and other partner and affiliated hospitals and healthcare providers throughout the Bay Area. Please visit http://www. . About Weill Cornell Medicine: Weill Cornell Medicine is committed to excellence in patient care, scientific discovery and the education of future physicians in New York City and around the world. The doctors and scientists of Weill Cornell Medicine - faculty from Weill Cornell Medical College, Weill Cornell Graduate School of Medical Sciences, and Weill Cornell Physician Organization - are engaged in world-class clinical care and cutting-edge research that connect patients to the latest treatment innovations and prevention strategies. Located in the heart of the Upper East Side's scientific corridor, Weill Cornell Medicine's powerful network of collaborators extends to its parent university Cornell University; to Qatar, where an international campus offers a U.S. medical degree; and to programs in Tanzania, Haiti, Brazil, Austria and Turkey. Weill Cornell Medicine faculty provide comprehensive patient care at New York-Presbyterian/Weill Cornell Medical Center, New York-Presbyterian/Lower Manhattan Hospital and New York-Presbyterian/Queens. Weill Cornell Medicine is also affiliated with Houston Methodist. For more information, visit Weill.Cornell.edu.


News Article | June 29, 2016
Site: www.techtimes.com

Ticked Off! Here's What You Need To Know About Lyme Disease Federal health officials have green-lighted the first pill that can treat all major forms of hepatitis C, a liver infection caused by the blood-borne hepatitis C virus (HCV) On Tuesday, the Food and Drug Administration (FDA) gave its approval to Gilead Sciences' combination pill Epclusa, the latest in a series of drug approvals that can reshape how the viral disease is treated. The new drug combines sofosbuvir, which was green-lighted by federal regulators in 2013, and the new drug velpatasvir. It is the first drug capable of treating all major forms of HCV. Six distinct HCV strains are currently known. Treatment recommendations and the duration of treatment often depend on knowing the particular genotype of the virus. The pill's broad indication makes it easier to use compared with five other hepatitis drugs that were recently approved by the FDA, as each of these were tailored to different viral strains or stages of the liver disease. Epclusa can be used by adult patients with or without liver damage. Patients with moderate to severe cirrhosis can take the drug in combination with the drug ribavirin. "This approval offers a management and treatment option for a wider scope of patients with chronic hepatitis C," said Edward Cox, FDA's Center for Drug Evaluation and Research Office of Antimicrobial Products director. Data from three clinical trials showed that the pill cleared the virus in 95 to 99 percent of 1,558 patients infected with HCV without cirrhosis or with mild cirrhosis after three months of treatment. Another clinical trial showed that treatment with Epclusa in combination with ribavirin cured 94 percent of patients with moderate to severe cirrhosis. Among the drug's side effects include headache and fatigue. "Building on the established backbone of sofosbuvir, Epclusa demonstrated consistently high cure rates across all genotypes, including among patients with genotype 2 and 3, who traditionally have required ribavirin or other multi-pill regimens," said  Ira Jacobson, from Mount Sinai Beth Israel, who is principal investigator in the clinical trials. The new drug, which needs to be taken daily, costs $74,760 for a 12-week course of treatment, or about $890 per pill. This makes the newly approved drug less expensive than Sovaldi, which costs about $1,000 per pill, and another Gilead hepatitis C treatment Harvoni, which costs $1,125 per pill. California-based Gilead has been criticized for the cost of its hepatitis C drugs, but these remain to be top sellers. In 2015, Harvoni was the world's top-selling prescription drug with over $18 billion in global sales. Data from the Centers for Disease Control and Prevention showed that hepatitis C affects at least 2.7 million people in United States. The disease killed more than 19,000 in 2014. © 2017 Tech Times, All rights reserved. Do not reproduce without permission.


News Article | February 15, 2017
Site: www.eurekalert.org

A population of cells in early development may give rise to the ventricular chambers of the heart, but not the atria, according to a study led by researchers from the Mindich Child Health and Development Institute at the Icahn School of Medicine at Mount Sinai and published today in Nature Communications. Congenital heart defects are the most common type of birth defect, affecting 35,000 babies in the United States each year, according to the U.S. Department of Health and Human Services. Many of these defects originate as the heart chambers are forming. While much is known about the development of the heart, the formation of the four distinct chambers of the heart has lacked thorough understanding. Using a model that traces cell lineage in mice, investigators studied the protein-coding gene Foxa2, primarily associated with endoderm and ectoderm development during embryogenesis. They discovered a population of progenitor cells expressing Foxa2 during early development that gave rise to cardiovascular cells of both the left and right ventricular chambers, but not the atria. Their research showed that atrial-ventricular segregation may occur long before the morphological establishment of differentiated cardiac structures. "An in-depth understanding of the formation of the heart chambers will enable us to better comprehend the biology behind detrimental heart defects and how best to address them," said lead investigator Nicole Dubois, PhD, Assistant Professor in the Department of Cell, Developmental and Regenerative Biology at the Icahn School of Medicine at Mount Sinai. "In addition to informing our understanding of early heart development, we hope that these findings will also lead to new protocols for the generation of ventricular cardiomyocytes in cell culture that could potentially be used in therapeutic settings." "There is a lot we still don't understand about this population, or the function of Foxa2 during the formation of the heart, but we think these findings provide a powerful new system to answer some of the most relevant open questions about how early heart development occurs," said Evan Bardot, PhD student and first author of the Nature Communications study. The National Institutes of Health (NIH/NHLBI) and the Mindich Child Health and Development Institute supported this research. The Mount Sinai Health System is an integrated health system committed to providing distinguished care, conducting transformative research, and advancing biomedical education. Structured around seven hospital campuses and a single medical school, the Health System has an extensive ambulatory network and a range of inpatient and outpatient services -- from community-based facilities to tertiary and quaternary care. The System includes approximately 6,100 primary and specialty care physicians; 12 joint-venture ambulatory surgery centers; more than 140 ambulatory practices throughout the five boroughs of New York City, Westchester, Long Island, and Florida; and 31 affiliated community health centers. Physicians are affiliated with the renowned Icahn School of Medicine at Mount Sinai, which is ranked among the highest in the nation in National Institutes of Health funding per investigator. The Mount Sinai Hospital is ranked as one of the nation's top 10 hospitals in Geriatrics, Cardiology/Heart Surgery, and Gastroenterology, and is in the top 25 in five other specialties in the 2014-2015 "Best Hospitals" issue of U.S. News & World Report. Mount Sinai's Kravis Children's Hospital also is ranked in seven out of ten pediatric specialties by U.S. News & World Report. The New York Eye and Ear Infirmary of Mount Sinai is ranked 11th nationally for Ophthalmology, while Mount Sinai Beth Israel is ranked regionally. For more information, visit http://www. , or find Mount Sinai on Facebook, Twitter and YouTube.


News Article | February 22, 2017
Site: www.eurekalert.org

An early-stage clinical trial has found that, compared to a placebo, a novel medication significantly reduces potentially life-threatening episodes of swelling of the airway as well as the hands, feet, and abdomen of patients affected by a rare genetic disorder. The findings were published in the New England Journal of Medicine. Although the small study was designed to test safety of the drug, the findings were so obviously promising that an expedited phase III clinical study has just started enrolling patients at the Icahn School of Medicine at Mount Sinai and other locations. The study's co-lead author, Paula Busse, MD, is an allergist-immunologist at Mount Sinai and an associate professor at the Icahn School of Medicine. The disorder, hereditary angioedema (HAE), causes recurrent and unpredictable attacks of swelling throughout the body. What triggers these attacks is not known. The multicenter, double-blind, placebo-controlled phase 1b study of 37 patients with type 1 or 2 HAE found that all participants that used a 300-mg dose of the drug, lanadelumab, were attack-free, as were 82 percent who took 400 mg. Only 27 percent of participants who used a placebo were incident-free. "Lanadelumab appears to stop attacks of HAE before they start, and this prophylactic approach may represent an exciting new advance for patients," says Dr. Busse, who treats dozens of patients with the disorder. "Given the uncertainty of when an attack may happen, some HAE patients don't want to travel," she says. "The disease can produce such swelling for three to five days that a face is disfigured, hands can't be used, it is difficult to walk, and the abdomen can become very painful. Having a drug that can provide an ongoing buffer against HAE will be wonderful." Current therapies for preventing HAE attacks work, but there are some drawbacks, Dr. Busse adds. A drug known as a C-1 inhibitor needs to be given intravenously every three or four days, and another approach, the use of pills called androgens (a modified testosterone), can produce unwanted hair growth in women, affect mood, cause weight gain, affect lipid levels and liver function, and inhibit growth in children. Lanadelumab, on the other hand, is a monoclonal antibody that is injected every two weeks and was found to be well tolerated by patients in this trial. HAE is an autosomal, dominantly inherited blood disorder caused by a deficiency or dysfunction of a regulatory gene known as the C1 inhibitor. The C1 inhibitor protein normally suppresses the production of another protein called bradykinin, which is the cause of swelling. Without a C1 inhibitor that functions properly, a person produces more bradykinin and consumes less of it. Bradykinin causes the swelling (angioedema) by making vessels dilate and leak fluid. The study included extensive blood analysis, which closely tracked how the drug successfully suppressed production of bradykinin. "If the phase III clinical study confirms the benefit we have seen, lanadelumab will make life much easier for HAE patients," says Dr. Busse. "Many patients who don't like and therefore don't use current treatments will likely opt for this new therapy, which will hopefully reduce morbidity and mortality, and improve the quality of life of patients with HAE." Dr. Busse worked with researchers from a number of universities nationally, and with a research team from Dyax, the developer of Lanadelumab and supporter of the trial. The Mount Sinai Health System is an integrated health system committed to providing distinguished care, conducting transformative research, and advancing biomedical education. Structured around seven hospital campuses and a single medical school, the Health System has an extensive ambulatory network and a range of inpatient and outpatient services--from community-based facilities to tertiary and quaternary care. The System includes approximately 7,100 primary and specialty care physicians; 12 joint-venture ambulatory surgery centers; more than 140 ambulatory practices throughout the five boroughs of New York City, Westchester, Long Island, and Florida; and 31 affiliated community health centers. Physicians are affiliated with the renowned Icahn School of Medicine at Mount Sinai, which is ranked among the highest in the nation in National Institutes of Health funding per investigator. The Mount Sinai Hospital is in the "Honor Roll" of best hospitals in America, ranked No. 15 nationally in the 2016-2017 "Best Hospitals" issue of U.S. News & World Report. The Mount Sinai Hospital is also ranked as one of the nation's top 20 hospitals in Geriatrics, Gastroenterology/GI Surgery, Cardiology/Heart Surgery, Diabetes/Endocrinology, Nephrology, Neurology/Neurosurgery, and Ear, Nose & Throat, and is in the top 50 in four other specialties. New York Eye and Ear Infirmary of Mount Sinai is ranked No. 10 nationally for Ophthalmology, while Mount Sinai Beth Israel, Mount Sinai St. Luke's, and Mount Sinai West are ranked regionally. Mount Sinai's Kravis Children's Hospital is ranked in seven out of ten pediatric specialties by U.S. News & World Report in "Best Children's Hospitals." For more information, visit http://www. , or find Mount Sinai on Facebook, Twitter and YouTube.


News Article | February 16, 2017
Site: www.eurekalert.org

(New York, NY - February 16, 2017) --Mount Sinai researchers have created a novel model that shows the step-by-step progression from normal blood cells to leukemia and its precursor diseases, creating replicas of the stages of the disease to test the efficacy of therapeutic interventions at each stage, according to a study to be published in Cell Stem Cell. This research marked the first time scientists have been able to transplant leukemia from humans to a test tube and then into mice for study, a landmark feat that will allow for valuable research to help find therapies for blood cancer patients in the future. "The new model will empower investigation into the cellular and molecular events underlying the development of leukemia in ways that were not possible before," said Eirini P. Papapetrou, MD, PhD, Associate Professor of Oncological Sciences, Medicine, Hematology, and Medical Oncology at the Icahn School of Medicine at Mount Sinai. "These findings provide a framework to aid investigation into disease mechanisms, drug responses, and the cellular and molecular events driving leukemia progression." Scientists used CRISPR (Clustered Regularly Interspaced Short Palindromic Repeat), a new, cutting-edge genome editing technology, to convert blood cells from patients with myelodysplastic syndrome (MDS) and acute myeloid leukemia (AML) to particular stem cells (called induced pluripotent stem cells) that can mimic all stages of disease progression, from a healthy state to pre-malignancy and finally full-blown leukemia. Though scientists believe that cancer develops through a step-by-step process by which a normal cell transforms to a fully malignant cell through intermediate stages, recreating the steps was challenging with previous tools. Scientists were able to manipulate the leukemia in a test tube environment, both by genetically modifying the disease-ridden stem cells at certain stages to revert to a pre-cancerous state, and by altering them so they would either progress to a severe or mild form of MDS. The ability to manipulate the leukemia to regress or progress will allow future researchers to test therapies that may be most potent at a particular stage, thus saving or extending a patient's life. Memorial Sloan Kettering Cancer Center was a valuable collaborator in this research. The lab of Michael G. Kharas, PhD, performed some of the mouse transplantation experiments in the study. "We are encouraged by the discovery that it was possible to generate potent engraftable leukemia derived from AML induced pluripotent stem cells," said Dr. Kharas, the co-corresponding author. "This work shows that integrated patient cell reprogramming and cancer genetics is a powerful way to dissect cancer progression." The progression model created through this research could also be used to develop models for more complex cancers, including solid tumors, the researchers said. The research was funded by grants from the National Institutes of Health from the National Heart, Lung, and Blood Institute and the National Institute of Diabetes and Digestive and Kidney Diseases (R01HL121570 and R00DK087923); the Damon Runyon Cancer Research Foundation, the Edward P. Evans Foundation, the Ellison Medical Foundation, the Henry and Marilyn Taub Foundation, the Babich Family Foundation and Alex's Lemonade Stand Foundation. The Mount Sinai Health System is an integrated health system committed to providing distinguished care, conducting transformative research, and advancing biomedical education. Structured around seven hospital campuses and a single medical school, the Health System has an extensive ambulatory network and a range of inpatient and outpatient services--from community-based facilities to tertiary and quaternary care. The System includes approximately 7,100 primary and specialty care physicians; 12 joint-venture ambulatory surgery centers; more than 140 ambulatory practices throughout the five boroughs of New York City, Westchester, Long Island, and Florida; and 31 affiliated community health centers. Physicians are affiliated with the renowned Icahn School of Medicine at Mount Sinai, which is ranked among the highest in the nation in National Institutes of Health funding per investigator. The Mount Sinai Hospital is on the "Honor Roll" of best hospitals in America, ranked No. 15 nationally in the 2016-2017 "Best Hospitals" issue of U.S. News & World Report. The Mount Sinai Hospital is also ranked as one of the nation's top 20 hospitals in Geriatrics, Gastroenterology/GI Surgery, Cardiology/Heart Surgery, Diabetes/Endocrinology, Nephrology, Neurology/Neurosurgery, and Ear, Nose & Throat, and is in the top 50 in four other specialties. New York Eye and Ear Infirmary of Mount Sinai is ranked No. 10 nationally for Ophthalmology, while Mount Sinai Beth Israel, Mount Sinai St. Luke's, and Mount Sinai West are ranked regionally. Mount Sinai's Kravis Children's Hospital is ranked in seven out of ten pediatric specialties by U.S. News & World Report in "Best Children's Hospitals." For more information, visit http://www. or find Mount Sinai on Facebook, Twitter and YouTube.


Mount Sinai Beth Israel Receive Award from the Department of Defense to Study Gulf War Illness with electroCore’s Non-Invasive Vagus Nerve Stimulation Therapy Basking Ridge, NJ, November 17, 2016 --( This study will be led by Benjamin Natelson MD, an internationally recognized leader in clinical care and research into medically unexplained pain and fatigue. The trial will be conducted at the Pain & Fatigue Study Center at Mount Sinai Beth Israel in Manhattan. “While pharmaceutical treatments are available for the treatment of Gulf War Illness, they have drawbacks,” says Dr. Natelson. “These drugs don’t work for all patients, the effect frequently only lasts a couple of months and the side effects are often so severe as to preclude their use so there is a real need for further research into treatment of this debilitating condition.” Having been previously involved with surgically implanted vagus nerve stimulation (VNS), in a fibromyalgia safety and tolerability study which resulted in several of the patients becoming pain free, Dr Natelson was keen to run a randomized sham controlled trial to rule out a placebo effect and to determine whether non-invasive VNS could be effective in gulf war illness. “VNS is already an FDA-approved treatment for epilepsy and depression and the drugs used to treat epilepsy and depression are in the same class as newly approved drugs to treat the widespread pain characteristic of fibromyalgia, a common diagnosis in Gulf War veterans,” says Dr Natelson. ElectroCore's proprietary, non-invasive electrical stimulation therapy works by the patient placing the device on the skin over the vagus nerve in the neck. This activates the fibers of the vagus nerve bundle that ascend to the brain to activate pain-related centers. The result of this activation is reduced pain. In the first phase of the study, veterans will be assessed for body-wide pain and headache. In the second phase half of the veterans will be given an active gammaCore nVNS device, and half will be given a sham (inactive) device. These devices will be used by the veterans three times a day for ten weeks, with periodic assessments of the severity of their pain and headaches. After the randomized phase, all veterans will receive active gammaCore devices to use for a further ten weeks in an open label phase. The final assessment of the study, lasting 20 weeks in total, will provide the investigators with information about the effectiveness of nVNS therapy in relieving both widespread pain and migraine headaches. Should the study produce results of sufficient interest, the United States Army has indicated that it will strongly consider supporting a larger pivotal study into VNS therapy for the treatment of widespread pain in Gulf War Illness. Basking Ridge, NJ, November 17, 2016 --( PR.com )-- The Pain & Fatigue Study Center at Mount Sinai Beth Israel in New York has been awarded a United States Army Medical Research grant to conduct a study into the treatment of veterans of the 1990-1991 Gulf War who have Gulf War Illness using electroCore’s non-invasive vagus nerve stimulation (nVNS) therapy gammaCore. The $703,272 grant will be used to fund a randomized clinical trial involving more than 40 veterans with widespread pain and migraines, a common complaint of gulf war veterans.This study will be led by Benjamin Natelson MD, an internationally recognized leader in clinical care and research into medically unexplained pain and fatigue. The trial will be conducted at the Pain & Fatigue Study Center at Mount Sinai Beth Israel in Manhattan.“While pharmaceutical treatments are available for the treatment of Gulf War Illness, they have drawbacks,” says Dr. Natelson. “These drugs don’t work for all patients, the effect frequently only lasts a couple of months and the side effects are often so severe as to preclude their use so there is a real need for further research into treatment of this debilitating condition.”Having been previously involved with surgically implanted vagus nerve stimulation (VNS), in a fibromyalgia safety and tolerability study which resulted in several of the patients becoming pain free, Dr Natelson was keen to run a randomized sham controlled trial to rule out a placebo effect and to determine whether non-invasive VNS could be effective in gulf war illness.“VNS is already an FDA-approved treatment for epilepsy and depression and the drugs used to treat epilepsy and depression are in the same class as newly approved drugs to treat the widespread pain characteristic of fibromyalgia, a common diagnosis in Gulf War veterans,” says Dr Natelson.ElectroCore's proprietary, non-invasive electrical stimulation therapy works by the patient placing the device on the skin over the vagus nerve in the neck. This activates the fibers of the vagus nerve bundle that ascend to the brain to activate pain-related centers. The result of this activation is reduced pain.In the first phase of the study, veterans will be assessed for body-wide pain and headache. In the second phase half of the veterans will be given an active gammaCore nVNS device, and half will be given a sham (inactive) device. These devices will be used by the veterans three times a day for ten weeks, with periodic assessments of the severity of their pain and headaches. After the randomized phase, all veterans will receive active gammaCore devices to use for a further ten weeks in an open label phase. The final assessment of the study, lasting 20 weeks in total, will provide the investigators with information about the effectiveness of nVNS therapy in relieving both widespread pain and migraine headaches.Should the study produce results of sufficient interest, the United States Army has indicated that it will strongly consider supporting a larger pivotal study into VNS therapy for the treatment of widespread pain in Gulf War Illness. Click here to view the list of recent Press Releases from electroCore

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