The Jacobi Medallion: A Celebration of Excellence

Updated on Aug 6, 2025 | Alumni, Community, Featured, Research

Seated, from left: Steven J. Burakoff, MD; Ming-Ming Zhou, PhD; Amy J. Goldberg, MD, FACS; Daniel Maklansky, MD; Nina A. Bickell, MD, MPH; and David C. Thomas, MD, MHPE; standing, from left: Kenneth L. Davis, MD; Annapoorna S. Kini, MD, MRCP, FACC; Zahi A. Fayad, PhD; Reginald W. Miller, DVM, DACLAM; Burton A. Cohen, MD; Sandra K. Masur, PhD; and Dennis S. Charney, MD.

Nine accomplished physicians and researchers were honored with the 2019 Jacobi Medallion—one of Mount Sinai’s highest awards—at a special event held at The Plaza on Thursday, March 14.

Dennis S. Charney, MD, Anne and Joel Ehrenkranz Dean, Icahn School of Medicine at Mount Sinai, and President for Academic Affairs, Mount Sinai Health System, addressed nearly 400 alumni, donors, faculty, and staff gathered for the awards ceremony, including Kenneth L. Davis, MD, President and Chief Executive Officer, Mount Sinai Health System. The Jacobi Medallion honors Mount Sinai alumni and faculty who have distinguished themselves in education, clinical and patient care, and biomedical science, or in extraordinary service to The Mount Sinai Hospital, the Icahn School of Medicine at Mount Sinai, or The Mount Sinai Alumni Association.

“Our honorees have excelled as educators, administrators, researchers, and clinicians—they truly show the range of excellence that Mount Sinai is known for,” Dr. Charney said.

Dr. Charney noted the many significant accomplishments at the Icahn School of Medicine during the last year. These include initiatives to improve faculty, trainee, and student wellness, and to reduce racism and bias in medicine and medical education. He also spotlighted major genetic discoveries in the areas of cancer, inflammatory diseases, autism, and Alzheimer’s disease, and important research that is leading to new treatments for cancers, heart disease, diabetes, and depression—all as Mount Sinai embarks on new plans to expand, rebuild, and modernize its research labs, hospitals, and ambulatory care centers.

“Thank you, alumni community, for your commitment to Mount Sinai as we continue to excel as a world-class medical school and health system,” Dr. Charney said.

Burton A. Cohen, MD, President of The Mount Sinai Alumni Association and Associate Clinical Professor of Radiology, delivered opening remarks, and Sandra K. Masur, PhD, Chair of the Jacobi Medallion Award Selection Committee and Professor of Ophthalmology, introduced the recipients.

The honorees are:

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Nina A. Bickell, MD, MPH
Associate Director, Community Engaged and Equity Research, The Tisch Cancer Institute
Co-Director, Center for Health Equity and Community Engaged Research
Co-Director, Cancer Prevention and Control, The Tisch Cancer Institute
Professor, Department of Population Health Science and Policy
Professor, Department of Medicine, Division of General Internal Medicine
Icahn School of Medicine at Mount Sinai

Nina Andrea Bickell, MD, MPH, is a Professor of Population Health Science and Policy, a Professor of Medicine (General Internal Medicine), Co-Director of the Center for Health Equity and Community Engaged Research, Associate Director for Community Engaged and Equity Research at The Tisch Cancer Institute and Co-Lead of the Cancer Prevention and Control Program. At Mount Sinai since 1995, Dr. Bickell has been working with the Harlem community and safety net hospitals throughout the NYC metropolitan area to reduce disparities in cancer care. Her work assessing the role of race, obesity, insulin resistance and aggressive breast cancer have expanded collaborations nationally. Dr. Bickell has extensive experience in large-scale, multi-center trials with regional, national, and international collaborations designed to understand causes of racial disparities, promote health equity and improve the quality of cancer care. Clinically, Dr. Bickell sees patients at Mount Sinai’s Internal Medicine Associates as a primary care physician serving East and Central Harlem.

 

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Steven J. Burakoff, MD
Dean for Cancer Innovation
Chief, Division of Pediatric Hematology-Oncology
Lillian and Henry M. Stratton Professor of Cancer Medicine
Professor, Department of Medicine
Professor, Department of Oncological Sciences
Professor, Jack and Lucy Clark Department of Pediatrics, Jack Martin Division of Pediatric Hematology-Oncology
Icahn School of Medicine at Mount Sinai

Steven J. Burakoff, MD is currently the Dean for Cancer Innovation and Chief, Pediatric Oncology at the Icahn School of Medicine at Mount Sinai. Dr. Burakoff was previously the Director of The Tisch Cancer Institute from 2007-2017 at the Icahn School of Medicine at Mount Sinai. Prior to coming to New York, Dr. Burakoff was the Margaret M. Dyson Professor at Harvard Medical School, the first recipient of the Harvard Medical School Excellence in Mentoring Award and was a member of the Board of Trustees at the Dana Farber Cancer Institute. Dr. Burakoff’s contributions to the field of Immunology are demonstrated by his authorship of more than 300 publications in peer-reviewed journals and his receiving the Lifetime Achievement Award from the American Association of Immunologists. Dr. Burakoff is a member of the Board of Directors of the Damon Runyon Cancer Foundation and the Joint Scientific Advisory Board for Stand Up to Cancer. He also serves as a member of the External Advisory Committee of several Cancer Centers.

 

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Zahi A. Fayad, PhD
Director, Translational and Molecular Imaging Institute
Lucy G. Moses Professorship in Medical Imaging and Bioengineering
Vice Chair for Research and Professor, Department of Radiology
Professor, Department of Medicine, Division of Cardiology
Icahn School of Medicine at Mount Sinai

Zahi A. Fayad, PhD currently serves as a Professor of Radiology and Medicine (Cardiology) at the Icahn School of Medicine at Mount Sinai where he is the founding Director of the Translational and Molecular Imaging Institute and Vice Chair for Research in the Department of Radiology. Dr. Fayad’s interdisciplinary research is dedicated to the detection and prevention of cardiovascular disease with seminal contributions in the field of multimodality biomedical imaging and nanomedicine. A faculty member of the Icahn School of Medicine at Mount Sinai since 1997, his work has recently expanded to understanding the effect of stress on the immune system and cardiovascular disease. The author of more than 500 peer-reviewed publications, 50 book chapters, and 500 meeting presentations, Dr. Fayad is currently the principal investigator of numerous NIH grants, sub-contracts, and pharmaceutically-funded clinical trials. He is the recipient of multiple prestigious awards and has trained over 100 postdoctoral fellows, clinical fellows, and students.

 

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Amy J. Goldberg, MD, FACS, MSSM ‘87
The George S. Peters, MD and Louise C. Peters Chair in Surgery
Professor and Chair, Department of Surgery, Lewis Katz School of Medicine at Temple University
Surgeon-in-Chief, Temple University Health System

Amy J. Goldberg, MD, FACS is a 1987 graduate of the Mount Sinai School of Medicine. She currently serves as Professor and Chair of the Department of Surgery at the Lewis Katz School of Medicine, Surgeon-in-Chief of Temple University Health System, Medical Director of Perioperative Services at Temple University Hospital, and is a newly elected Director of the American Board of Surgery. Dr. Goldberg is well recognized as a superb educator – winning several teaching awards including Temple University’s highest teaching honor, The Great Teacher Award in 2018, the Lindback Award, and multiple Golden Apple Awards. Her contributions to academic medicine have earned numerous accolades throughout her career. Dr. Goldberg is widely recognized as an expert in violence prevention and improved outcomes for victims of trauma. She is also a nationally celebrated clinician scholar with a robust portfolio of highly regarded publications and presentations.

 

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Annapoorna S. Kini, MD, MRCP, FACC
Director, Cardiac Catheterization Laboratory
Director, Structural Heart Disease Program
The Mount Sinai Hospital
Director, Interventional Cardiology Fellowship Program
Zena and Michael A. Wiener Professor of Medicine
Icahn School of Medicine at Mount Sinai

Annapoorna S. Kini, MD, MRCP, FACC, is the endowed Zena and Michael A. Wiener Professor of Medicine at the Zena and Michael A. Wiener Cardiovascular Institute at the Icahn School of Medicine at Mount Sinai. An internationally acclaimed leader in the field of percutaneous coronary intervention and heart valve therapy, she is the Director of the Cardiac Catheterization Laboratory and Director of the Structural Heart Intervention at The Mount Sinai Hospital. Dr. Kini is known for her expertise in performing complex coronary interventions and has contributed substantially in making The Mount Sinai Hospital’s Cardiac Catheterization Laboratory one of the busiest and best in the world. She is highly regarded in giving quality services to adults with coronary artery disease and various forms of structural heart disease which includes Transcatheter aortic valve replacement and Transcatheter mitral valve therapies. Dr. Kini is also an excellent teacher dedicated to the teaching of both cardiology and interventional fellows.

 

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Daniel Maklansky, MD
Radiologist, New York Medical Imaging Associates
Attending Radiologist, The Mount Sinai Hospital
Associate Clinical Professor, Department of Radiology
Icahn School of Medicine at Mount Sinai

Daniel Maklansky, MD is a partner at New York Medical Imaging Associates, an Attending Radiologist at The Mount Sinai Hospital and an Associate Clinical Professor of Radiology at the Icahn School of Medicine at Mount Sinai. Dr. Maklansky, a graduate of the State University of New York Downstate Medical School, has been associated with Mount Sinai, its patients, residents and fellows, and attending physicians since beginning his radiology residency here in 1959. Dr. Maklansky has authored or co-authored at least 24 peer-reviewed publications as well as multiple chapters in the gastrointestinal radiology literature. He has also trained countless medical students, radiology residents and gastroenterology fellows, both in weekly conferences that he co-chaired over many years, as well as in rotations through his office. Dr. Maklansky has been very involved with the Association of the Attending Staff of Mount Sinai, and served as the Scientific Course Director of its annual symposium over many years.

 

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Reginald W. Miller, DVM, DACLAM
Dean for Research Operations and Infrastructure
Associate Professor, Center for Comparative Medicine and Surgery
Associate Professor, Department of Environmental Medicine and Public Health
Icahn School of Medicine at Mount Sinai

Reginald W. Miller, DVM, DACLAM serves as the Dean for Research Operations and Infrastructure and is responsible for the oversight of the Icahn School of Medicine at Mount Sinai Biosafety, Environmental Health and Safety, Laboratory Safety including the Select Agents and Toxins Program (Operations), Core Facilities and Shared Resources as well as Basic Science Laboratory Infrastructure (Infrastructure). Dr. Miller is also the Senior Research Integrity Officer (RIO) for the Mount Sinai Health System. In this role, he is responsible for conducting reviews of allegations of research misconduct and ensuring that a program on the Responsible Conduct of Research is in place.

 

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David C. Thomas, MD, MHPE
System Vice Chair for Education, Department of Medicine
Associate Dean for Continuing Medical Education
Professor, Department of Medicine
Professor, Department of Medical Education
Professor, Department of Rehabilitation and Human Performance
Icahn School of Medicine at Mount Sinai

David C. Thomas, MD, MHPE serves as the System Vice Chair for Education in the Department of Medicine and is the Associate Dean for Continuing Medical Education (CME) at the Icahn School of Medicine at Mount Sinai. Dr. Thomas is a Professor of Medicine, Medical Education and Rehabilitation and Human Performance. As a valued and dedicated Clinician-Educator at the Icahn School of Medicine at Mount Sinai, Dr. Thomas has developed programs and taught at all levels of the continuum of education – from Undergraduate Medical Education to Graduate Medical Education and Continuing Medical Education. Dr. Thomas has been affiliated with Mount Sinai for over 20 years and has dedicated his entire career to the care of underserved and vulnerable patients. In 2004, he Co-Founded the East Harlem Health Outreach Partnership (EHHOP), a student run, attending-directed clinic for uninsured members of the East Harlem community with the goal of providing equitable access to high quality, comprehensive healthcare to all.

 

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Ming-Ming Zhou, PhD
Co-Director, Drug Discovery Institute
Dr. Harold and Golden Lamport Professorship in Physiology and Biophysics
Professor and Chair, Department of Pharmacological Sciences
Professor, Department of Oncological Sciences
Icahn School of Medicine at Mount Sinai

Ming-Ming Zhou, PhD is the Dr. Harold and Golden Lamport Professorship in Physiology and Biophysics and Chairman of the Department of Pharmacological Sciences at the Icahn School of Medicine at Mount Sinai. He is also Co-Director of the Drug Discovery Institute, and Professor of Oncological Sciences. His research interest is directed at better understanding of the basic principles that governs epigenetic regulation of gene transcription in human biology of health and diseases. The Zhou Lab was the first to discover the bromodomain as the lysine-acetylated histone binding domain (‘chromatin reader’) in gene transcription (Nature 1999), and demonstrate druggability and therapeutic potential of modulating bromodomain/acetyl-lysine binding in gene expression to treat a wide array of human diseases including cancer and inflammation. This concept has had a transformative impact in epigenetic drug discovery in the pharmaceutical industry. Dr. Zhou was elected as a fellow of the American Association for the Advancement of Science in 2012.

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$12.5 Million Grant to Advance Study of Hormone’s Role in Weight Gain and Bone Loss

Updated on Jun 30, 2022 | Featured, Research, Women's Health

Mone Zaidi, MD, PhD, center, with team members in the Department of Medicine (Endocrinology, Medicine and Bone Disease), from left: Sakshi Gera, PhD, Postdoctoral Fellow; Funda Korkmaz, MD, Associate Researcher; Jameel Iqbal, MD, PhD, Senior Faculty; Olena Hadeliya, Associate Researcher; Daria Lizneva, MD, PhD, Instructor; Anisa Gumerova, MD, PhD, Associate Researcher; Alina Rahimova, MD, Biomedical Informatician; Nanette Fraticelli, Grants Coordinator; and Tony Yuen, PhD.  View a video on the FSH project.

Researchers at the Icahn School of Medicine at Mount Sinai have been awarded a $12.5 million grant from the National Institute on Aging for a study seeking to advance the treatment of two public health hazards in older adults—osteoporosis and obesity. The four-part project will focus on follicle-stimulating hormone (FSH), which rises at menopause and could be responsible for the weight gain and bone loss that many women experience in middle age.

FSH was known for years to be an important part of the reproductive system. But the groundbreaking research of Mone Zaidi, MD, PhD, Professor of Medicine (Endocrinology, Diabetes and Bone Disease) at the Icahn School of Medicine at Mount Sinai, and his team showed in a mouse model that FSH also plays a direct role in bone loss and belly-fat gain—and that blocking FSH would reverse those effects. Dr. Zaidi has now developed a “humanized” monoclonal antibody to block FSH signaling, and it will be tested in mice during the new project.

“This next stage will bring us closer to creating an effective therapy with an FSH-blocking antibody aimed at preventing and treating both obesity and osteoporosis,” says Dr. Zaidi, who is principal investigator of the overall project. Targeting and blocking FSH was found in past studies to be effective in male as well as female mice, Dr. Zaidi adds, so its benefits could extend to both genders in people. He will oversee and serve as director for the four new studies, which will be conducted at Mount Sinai and other academic medical centers around the country. Mount Sinai will receive the bulk of the grant—$2 million a year for five years—and will serve as the data center for the project.

The first study, conducted at Mount Sinai, will determine if blocking FSH in mice will change bone mass and body composition across their lifespans, inducing them to live longer. The second study will explore whether the new monoclonal FSH-blocking antibodies can prevent fat gain and bone loss in mice, and if they can also treat existing obesity and osteoporosis. This investigation will be led by Tony Yuen, PhD, Assistant Professor of Medicine (Endocrinology, Diabetes and Bone Disease) at the Icahn School of Medicine at Mount Sinai, and performed collaboratively with UT Southwestern Medical School. For the third study, also in mice, Mount Sinai will team up with Maine Medical Center Research Institute (MMCRI) to study the effects of FSH on bone marrow fat deposits, which are associated with osteoporosis during aging and in menopause. And the fourth project will study the correlation between FSH, body fat, and fracture risk in people from the AGES-Reykjavik cohort of 12,000 women and men between 66 and 93 years of age. This epidemiology project will be led by scientists at the University of California, San Francisco.

The FSH research builds on a long-term collaboration between Dr. Zaidi, director of The Mount Sinai Bone Program, and Clifford Rosen, MD, senior scientist at MMCRI and co-director of the four projects. The results of their previous work were published in the journal Nature in 2017 and were among the eight “notable advances” in biomedicine named that year by Nature Medicine. Dr. Zaidi is hopeful that his team’s work will progress to clinical trials in humans within two years. “What would be fascinating and incredibly rewarding,” he says, “is if we can actually show a significant increase in lifespan while also regulating obesity and osteoporosis through a single, FSH-blocking agent.”

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Research Is Customizing Artificial Pancreas for Pregnancy

Mar 19, 2019 | Featured, Patient Stories, Research

From left: Carol J. Levy, MD; Camilla Levister, NP; Co-Investigator Grenye O’Malley, MD, Assistant Professor of Medicine (Endocrinology, Diabetes and Bone Disease); and Clinical Research Coordinator Selassie Ogyaadu, MD, MPH.

An artificial pancreas system tailored to the specific, and daunting, challenges faced by pregnant women with type 1 diabetes is the goal of a study to be conducted by a consortium of four leading institutions, including the Icahn School of Medicine at Mount Sinai. The project, funded by the National Institutes of Health, is the first of its kind in the United States.

“Pregnant women with type 1 diabetes typically test their blood sugars seven to eight times per day, and many wear glucose sensors, but they still struggle tremendously to keep their blood sugar levels in target ranges,” says Carol J. Levy, MD, Clinical Director of the Mount Sinai Diabetes Center, and the project’s principal investigator at the Icahn School of Medicine.

If blood sugar levels are too low for a prolonged period of time, a pregnant woman can feel poorly, or pass out, and risks seizures. If levels rise too high, there are risks to her unborn child, including malformations, delayed lung maturity, placental malfunction, or fetal death. “The use of customized technology provides an important opportunity to improve patient and fetal outcomes,” says Dr. Levy, Associate Professor of Medicine (Endocrinology, Diabetes and Bone Disease), Icahn School of Medicine at Mount Sinai. “We are excited to be part of the team evaluating this important area of research designed to improve care and reduce patient burden.”

The clinical trials will be conducted by specialists at three sites: Mount Sinai; the Mayo Clinic in Rochester, Minnesota; and the Sansum Diabetes Research Institute in Santa Barbara, California.

The overall principal investigator is Eyal Dassau, PhD, an expert on algorithm design and Director of the Biomedical Systems Engineering Research Group at Harvard University’s John A. Paulson School of Engineering and Applied Sciences.

Each patient participating in the artificial pancreas portion of the study will wear a Dexcom G6 continuous glucose monitoring device and an insulin pump. Both devices are linked with a software algorithm on a smartphone, which identifies a personalized blood-glucose range and prompts doses of insulin with reduced input from the patient. This is also known as a closed loop system because it uses a customized algorithm to close the decision-making loop between the glucose reading and the delivery of insulin, with the goal of improving blood sugar control.

In the artificial pancreas, or closed loop system, a controller—consisting of a software algorithm on a smartphone—receives readings from a continuous glucose sensor and prompts doses from an insulin pump, with reduced input from the patient.

The only artificial pancreas approved by the U.S. Food and Drug Administration is the Medtronic 670G. But it is designed for blood glucose targets between 120 milligrams/deciliter (mg/dL) and 180 mg/dL, while pregnant women with type 1 diabetes aim for a narrower range—under 90 mg/dL while fasting and 130 to 140 mg/dL one hour after meals.

“This is not easy to do, even for the most meticulous patients,” says Dr. Levy. Another challenge is that hormonal shifts in each trimester change the level of resistance to insulin, making the proper dose “a moving target.”

The first clinical trial in the grant—Longitudinal Observation of Insulin Requirements and Sensor Use in Pregnancy (LOIS-P)—is now enrolling 50 pregnant women with type 1 diabetes and will follow their glycemic outcomes into the postpartum period, providing guidance in refining the algorithm. The trial is named for the late Lois Jovanovic, MD, who was a former director of the Sansum Institute and a role model for many in the study of diabetes, including Dr. Levy. The overall project is intended to progress from a sequence of in-clinic studies to a safe and effective at-home clinical trial.

“I view this work as critical for patients, and I have a personal perspective as well, since I have had type 1 diabetes for 48 years,” Dr. Levy says. “I managed my diabetes with my health care team through two pregnancies, and every day was a challenge. Caring clinicians are important, and any support that people with type 1 diabetes can have provides a huge difference. Every time I enroll a patient in the LOIS-P study, I feel as if we are one step closer to a real answer for many women.”

For more information about the clinical trials, call 212-241-9089.

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Changing the Paradigm for Patients With Treatment-Resistant Depression

Mar 14, 2019 | Featured, Psychiatry, Research

Dennis S. Charney, MD

The U.S. Food and Drug Administration on March 5 approved SPRAVATO™ (esketamine) CIII nasal spray for the treatment of treatment-resistant depression. Dennis S. Charney, MD, Anne and Joel Ehrenkranz Dean, Icahn School of Medicine at Mount Sinai, is a co-inventor of a method of treatment, which is patented and part of the drug application for SPRAVATO, a product of the Janssen Pharmaceutical Companies of Johnson & Johnson.

“As a researcher, you strive to come up with new treatments for the patient, especially in terms of finding answers to the most debilitating diseases,” says Dr. Charney, who is also President for Academic Affairs, Mount Sinai Health System. “To know that you oversaw the early development of an approach that can make a difference in the lives of countless individuals is extremely rewarding.”

Esketamine represents the first new mechanism of action in decades to treat major depressive disorder. Delivered in the form of a nasal spray, esketamine works differently than the three classes of antidepressants that are currently on the market. The drug works on the N-methyl-D-aspartate (NMDA) receptor, an ionotropic glutamate receptor in the brain. In contrast, widely used antidepressants target different neurotransmitters—serotonin, serotonin and norepinephrine, and norepinephrine and dopamine—and can take weeks or even months to work.

An estimated 30 percent of people who are treated for depression do not respond to therapies. Treatment-resistant depression (TRD) is a devastating disease that is associated with greater morbidity, higher health care costs, and various comorbid conditions.

“We commend Dr. Charney and his colleagues for their work in changing the paradigm for patients with treatment-resistant depression,” says Kenneth L. Davis, MD, President and Chief Executive Officer of the Mount Sinai Health System. “Through his commitment to innovation and science, Dr. Charney has inspired countless researchers to leverage new technologies and create discoveries to benefit the lives of patients around the world—while at the same time leading the Icahn School of Medicine to unparalleled growth and high national rankings.”

In his role as Dean of the Icahn School of Medicine, Dr. Charney says that many younger researchers look to him for advice. “I tell them that in science you’ve got to keep trying, to keep pushing,” he says. “There is a lot to be gained by working in a small group that is engaged in the give and take of scientific interaction. Researchers should understand that failure is going to happen, but they can use that failure as a stepping stone to discovery.”

Conflicts of Interest Disclosure: Dr. Charney is named as co-inventor on patents filed by the Icahn School of Medicine at Mount Sinai (ISMMS) relating to the treatment for treatment-resistant depression, suicidal ideation, and other disorders. ISMMS has entered into a licensing agreement with Janssen Pharmaceuticals, Inc., and it has and will receive payments from Janssen under the license agreement related to these patents for the treatment of treatment-resistant depression and suicidal ideation under this agreement. Consistent with the ISMMS Faculty Handbook, Dr. Charney is entitled to a portion of the payments received by the ISMMS. Since SPRAVATO has received regulatory approval for treatment-resistant depression, ISMMS and thus, through the ISMMS, Dr. Charney will be entitled to additional payments, beyond those already received, under the license agreement.

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$4 Million Grant for Study of Sickle Cell Treatment

Mar 6, 2019 | Featured, Research, Sickle Cell Disease, Your Health

Jeffrey Glassberg, MD, left, administered a lung-function test to Eric Gay, a participant in the clinical trial.

An inhaled treatment that might improve lung function— and reduce the painful symptoms—of patients with sickle cell disease (SCD) is being studied in a clinical trial funded by a $4 million grant from the National Institutes of Health awarded to researchers at the Icahn School of Medicine at Mount Sinai.

“Inhaled corticosteroids, a well-established treatment for asthma, offer a creative new approach to treating sickle cell disease, with the potential to dramatically improve patient outcomes,” says Jeffrey Glassberg, MD, principal investigator of the study, and Associate Professor of Emergency Medicine, and Medicine (Hematology and Medical Oncology). SCD affects about 100,000 people in the United States and is more prevalent in certain ethnic groups, such as African Americans and Hispanics.

he inherited disease is caused by a mutation in hemoglobin, a protein inside red blood cells that carries oxygen from the lungs to the rest of the body. In SCD, abnormal hemoglobin forms long rods and distorts red blood cells into a sickle shape. The repeated damage to red cells causes the blood to become inflamed and sticky, causing pain, infections, stroke, and potentially, early death. The only organs in the body that can reverse sickling are the lungs, but they are highly inflamed in SCD. That is why researchers have proposed using inhaled steroids as a treatment.

“We hypothesize that if patients take an asthma medicine that reduces inflammation in the lungs, it might improve their ability to put oxygen into their blood and make their sickle cell better,” says Dr. Glassberg, Director of the Comprehensive Program for Sickle Cell Disease at the Icahn School of Medicine. The phase II trial—Inhaled Mometasone to Promote Reduction in Vaso-occlusive Event (IMPROVE 2)—is seeking to recruit 80 patients who have sickle cell disease but do not have asthma. They will be randomized, with one group receiving a placebo and the other receiving an inhaled treatment of the steroid mometasone furoate once a day for 48 weeks. The dose will be low, 220 micrograms, to avoid side effects associated with steroids, such as weight gain or bone disorders.

The primary outcome studied will be the level of a biomarker called soluble vascular cell adhesion molecule (sVCAM). “This is something that you measure in the blood, and it correlates very well with how bad somebody’s sickle cell disease is at the time,” says Dr. Glassberg. “The sVCAM level goes up when they are sick, and it goes back down when they get better.” Patients will also keep a daily diary of their pain and quality of life, and return regularly for tests of lung function.

A prior study, IMPROVE 1, established the feasibility of the current trial, with results published in March 2017 in the American Journal of Hematology. It involved 52 patients, who took the inhaled treatment for 16 weeks. Sickle cell symptoms tend to be seasonal, with some patients reporting more pain in colder months, so Dr. Glassberg says the longer IMPROVE 2 study will provide a broader understanding of the treatment. His group is seeking to enroll 20 people a year and to complete the study in June 2023.

Dr. Glassberg sees a future role for inhaled steroids as part of a drug “cocktail,” along with drugs like hydroxyurea that interfere with sickling. A low-dose inhaled steroid treatment would work in concert with these drugs, improving the flow of oxygenated blood. “This is especially appealing because inhaled steroids are inexpensive, widely available, and do not require sophisticated equipment, so they can be used anywhere in the world,” Dr. Glassberg says. “I think this treatment has the potential for a big health impact.”

For information about enrollment in the clinical trial, call 212-241-3650.

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Uncovering the Biology of Neurodegeneration

Mar 5, 2019 | Featured, Research

Ivan Marazzi, PhD

Ivan Marazzi, PhD, Assistant Professor of Microbiology at the Icahn School of Medicine at Mount Sinai, received $2.5 million in funding from the Chan Zuckerberg Initiative (CZI) to further the understanding of the underlying causes of neurodegenerative disorders such as amyotrophic lateral sclerosis (commonly known as Lou Gehrig’s disease), and Alzheimer’s and Parkinson’s diseases.

The Initiative was established by Facebook founder Mark Zuckerberg and pediatrician Priscilla Chan, his wife. The award is part of a $64 million commitment to fund early-career investigators and collaborative science teams to launch the CZI Neurodegeneration Challenge Network, which aims to bring together basic scientists from neuroscience, cell biology, biochemistry, immunology, and genomics.

Cori Bargmann, PhD, Head of Science for CZI, says: “We’re excited to welcome the first group of CZI Neurodegeneration Challenge Network grantees. Together, their work will increase our knowledge of the basic biology of these diseases—and we need that knowledge to develop better treatments.”

Dr. Marazzi studies epigenetic- and chromatin-mediated mechanisms—the heritable alterations that cause genes to turn on or off—and the cellular response to pathogens or cellular differentiation. The major focus of his research is the unique and shared molecular pathways underlying inflammatory, infectious, and neurodegenerative diseases to uncover the relationship between seemingly unrelated diseases to find effective therapeutic interventions.

Earlier work from Dr. Marazzi provided a new paradigm for how mutations can confer both susceptibility to infection and predisposition to neurodegeneration. He was the senior investigator of two groundbreaking studies published in Nature Immunology and Cell that identified a link between innate immune dysfunctions and congenital defects in two proteins controlling RNA metabolism—the RNA exosome and senataxin.

“These studies revealed how we can discover genes linked to disease—in this case, neurodegeneration—by looking at the natural symbiosis between a host and a pathogen,” says Dr. Marazzi. “Our goal is to use this discovery as a foundation to elucidate the relationship between innate immune defects and neurodegeneration to find effective therapeutic interventions.”  

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