Clinical research students at the Icahn School of Medicine at Mount Sinai conduct and report on a wide variety of research. Below are recent highlights.
Evaluating an Objective Measure of Language in Minimally Verbal Autism: Automated Vocal Analysis in Phelan-McDermid Syndrome
Student: Jacquelin Rankine
Mentor: Alex Kolevzon, MD
My study aims to evaluate the use of an automated digital language processor (LENA) in children with Phelan-McDermid Syndrome, a form of autism characterized by severe language impairment. Despite advances in the study and treatment of autism spectrum disorders (ASD), up to 30 percent of individuals with autism remain nonverbal or minimally verbal. Further research in minimally verbal autism is hindered by the lack of an objective tool for measuring language ability in this population. The LENA device is worn by the affected child in specially designed clothing and allows naturalistic recording of the child’s speech for periods of up to 16 hours. I then analyze the recorded speech through the LENA software for measures of language ability and social reciprocity. While the LENA device has been validated for use in populations with ASD or specific language impairment alone, my study provided the first validation of its use in individuals with minimally verbal autism.
Natural versus synthetic Toll-like receptor (TLR) agonists used in a Flt3L-primed in situ lymphoma vaccine
Student: Paul Peng
Mentor: Joshua Brody, MD
My project uses a murine lymphoma model to study the capability of "natural" TLR agonists (nTLRa)--such as live/attenuated/killed bacterial or viral components in prophylactic vaccines--to activate tumor-antigen-loaded DCs. Nearly all lymphomas remain incurable with standard therapies. A novel immunotherapeutic approach called "in situ vaccination" recently demonstrated Phase I/II efficacy in treating indolent lymphomas. In this vaccine maneuver, a dendritic cell (DC) growth factor called Flt3L and a Toll-like receptor (TLR) agonist are injected at the site of a patient’s tumor, which then also receives local irradiation. The resulting immunostimulatory environment allows DCs to uptake tumor antigens, become activated by the TLR agonist, and cross-present their antigens to cytotoxic T cells for induction of anti-tumor immunity. I hypothesize that certain nTLRa or combinations of nTLRa will have greater efficacy than synthetic TLR agonists to induce a systemic anti-tumor response.
My responsibilities include design of in situ vaccination experiments in a mouse lymphoma model; collecting flow cytometric characterization of immune cells after the vaccine maneuver; analyzing flow cytometry and enzyme-linked immunosorbent assay cytotoxic cell killing assay results; as well as reporting the final results in a manuscript.
Student: Ammar Siddiqui
Mentor: Ula Hwang, MD, MPH
The focus of my research year has been on analyzing a large dataset to identify disparities in the quality of pain care in the emergency department. The best thing I like about my project is the freedom it gives me to direct my growth as a researcher: Since no data collection is involved, I have the luxury to focus on design, statistical analysis and scientific writing - all areas which I have had little experience with, and which both my mentor and I wanted to target for improvement. While I still have a lot to learn, my PORTAL classes have made the road far less daunting. I honestly can't imagine doing this year without the foundation in stats and study design that PORTAL classes and journal clubs have given me. Additionally, I have the benefit of a long-standing mentor who has been invested in both my growth and my research career in all the four years I have worked with her - a relationship that Dr. Zier helped set up the summer of MS1. My responsibilities include designing the study design, as well as analysis, and manuscript-writing.
Student: Lianna Lipton
Mentor: Rosalind J. Wright, MD MPH
I have spent my scholarly year working in the Department of Pediatrics on a prospective pregnancy cohort study conducted at Mount Sinai and Harvard. My study involves following mother-child dyads through pregnancy and several years after the child is born. My research aim is to assess the influence of perinatal stress on children’s respiratory health and neurodevelopment. For my thesis project, I survey mother-child pairs to assess the influence of maternal stress and dietary intake on neurobehavioral outcomes in 2.5-year old children. I measure stress in several ways, including a negative life event questionnaire and cortisol levels. I assess child behavior with a behavioral questionnaire, which mothers complete when their children are 2.5 years old.
My responsibilities include running a six-month study lab visit with the mother and infant to assess stress, autonomic function, and behavior; Assisting with data coding and analysis of cardiac and respiratory autonomic data collected from the six-month lab visit; Completing phone calls to collect child behavioral data; and ongoing data entry and analysis.
Student: Brett Marinelli
Mentor: Howard Levin, MD; Timothy Harkin, MD; Raul San-Jose, PhD
The goal of my PORTAL thesis is to integrate variations of existing algorithms to create an assessment protocol for CT images of emphysema patient to better guide therapy. Large, population-wide studies of COPD have accumulated considerable imaging data available for public research. In the context of non-invasive procedures, useful emphysema phenotypes have been identified via CT for their susceptibility to intervention. Expanding on this prior work with the help of pulmonologists, radiologists and computer scientists I assess hundreds of available emphysema CT images available through the COPDGene project, and also test the protocol retrospectively in patients who have undergone non-invasive interventions.
My role in the project includes: Maintaining 2TB imaging database from the NIH; developing and applying image analysis programs; and performing project design, statistical analysis and manuscript writing.
Student: Amanda Leiter
Mentor: Matthew Galsky
For my project, I’ve conducted research related to improving cancer clinical trial awareness and recruitment. I pilot a crowdsourcing platform to obtain input from researchers, physicians, and patients on the design of a prostate cancer clinical trial. For the second part of the project, I analyze predictors of low clinical trial awareness, a known barrier to clinical trial recruitment, in a national survey dataset, as knowledge of barriers to awareness can help target trial education efforts.
My role on the project includes project design, statistical analysis, and manuscript writing.
Patient experience on therapy for hepatitis C virus, the impact of successful treatment on liver stiffness, and the meaning of sustained virologic response
Student: Jillian Nickerson
Mentor: Andrea Branch
My project consists of three studies: A prospective cohort study evaluating side effects and health related quality of life in patients with HCV treated with sofosbuvir and simeprevir; a retrospective case-control study analyzing changes in liver stiffness to estimate liver fibrosis in patients who achieved sustained virologic response (SVR) compared with those who were non-responders to treatment; and a assessing the long-term consequences of SVR.
My responsibilities on the project include designing the study; conducting literature reviews; submitting IRB protocol; recruiting and consenting patients; interviewing patients over the phone; performing statistical analysis of data; writing abstracts and manuscripts; presenting at national meetings; and retrospectively reviewing electronic medical records
Survival and long-term outcomes following bioprosthetic vs mechanical aortic valve replacement in patients aged 50 to 69 years
Student: Yuting P Chiang
Mentor: Joanna Chikwe (Professor, Dept Cardiovascular surgery)
My PORTAL thesis is on my retrospective study of aortic valve replacement using the Statewide Planning and Research Cooperative System. My focus is on the younger age range of patients, 50 to 69, to investigate whether outcomes differed between those receiving bioprosthetic versus mechanical aortic valve replacement. This project exposes me to the use of advanced software to conduct large reviews of surgical outcomes and is helping me contribute to the debate on which surgical approach is best in this younger age group. My study has led to a first author publication in the Journal of the American Medical Association.
My role in the project includes coming up with the study focus and design, dataset acquisition, statistical analysis, data interpretation, and drafting of the manuscript.