SinaInnovations Celebrates Past and Looks to Future

Raymond Schinazi, PhD

Scientists whose revolutionary treatments have cured millions of people with hepatitis C and restored sight to patients with a rare form of blindness were among keynote speakers at the seventh annual SinaInnovations conference, held in Stern Auditorium during two consecutive days in October.

Innovation in science and medicine was the theme of this year’s SinaInnovations conference. The event highlighted the Icahn School of Medicine at Mount Sinai’s commitment to groundbreaking research and concluded its yearlong 50th anniversary celebration.

“This conference exemplifies Mount Sinai’s mission to produce the great translational research that improves the lives of our patients through innovation and entrepreneurship,” 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, said as he opened the event.

Katherine A. High, MD

Keynote speaker Raymond Schinazi, PhD, Director of the Center for AIDS Research at the Emory University School of Medicine, spoke about his part in developing an antiviral treatment for hepatitis C that has had a 96 percent cure rate and is considered to be one of the greatest successes in modern medicine. Dr. Schinazi also helped develop TRUVADA for PrEP®, a prophylactic drug that enables people to protect themselves before coming into contact with HIV-1. His next priority, he said, will be  developing a cure for hepatitis B, which affects 400 million people worldwide. “We can do this,” Dr. Schinazi said.

Katherine A. High, MD, President of Spark Therapeutics, spoke about development of a gene therapy for hemophilia and the creation of LUXTURNA™, a treatment for a rare form of blindness caused by a defective gene. Spark Therapeutics’ LUXTURNA is now the first gene therapy for an inherited disease to be approved by the U.S. Food and Drug Administration and the European Commission.

Sri Madabushi, PhD

The conference also featured a session on the promise of data-driven innovation that included Sri Madabushi, PhD, Business Development Director of Google AI Healthcare; and Eric Dishman, Director of the All of Us Research Program of the National Institutes of Health. All of Us is seeking to enroll more than 1 million Americans who will share their personal health data with researchers and clinicians who are advancing precision medicine.

Mr. Dishman said his personal experience is emblematic of the program’s goals. For 23 years, he battled a rare form of kidney cancer, enduring 57 rounds of chemotherapy and radiation. Seven years ago, when he was near death, he had his genome sequenced, and physicians used that data to identify a pancreatic cancer drug that eradicated his disease. “Here I was, a wealthy, college-educated man who knows CEOs and senators, and I barely got access to precision medicine—at the 11th hour. What about everyone else?”

Access to quality health care was the focus of the final session at SinaInnovations. Kelly J. Kelleher, MD, Distinguished Professor of Pediatrics, Psychiatry, and Public Health at Nationwide Children’s Hospital in Columbus, Ohio, said that years ago, Nationwide became troubled by economic and health care disparities in a nearby neighborhood. “Some of our patients came from one of the areas most affected by violence, homelessness, drug addiction, and infant mortality, and I was offering a little bit of amoxicillin for ear infections,” Dr. Kelleher said. “This was unacceptable.” He said Nationwide has adopted a “neighborhood as patient” philosophy, working to incentivize doctors to keep patients healthy, support efforts to build affordable housing, and create job-training programs in the underserved community.

At the conclusion of SinaInnovations, conference leader Scott L. Friedman, MD, Dean for Therapeutic Discovery and Chief of the Division of Liver Diseases at the Icahn School of Medicine, summed up the event’s overarching message of innovation.

“We are a great medical school working to fulfill great ambitions,” he said. “This conference was intended to reflect upon the history of our 50 years as a medical school and project into our future.”

 

A panel on Caring for the Community Through the Lifespan with, from left: R. Shaun Morrison, MD; Nina A. Bickell, MD; Nathan Goldstein, MD; Elizabeth Howell, MD; John Steever, MD; David Blumenthal, MD; Kelly J. Kelleher, MD; and Angela Diaz, MD, PhD.

Fourth Annual Mount Sinai Innovation Awards

Anne Schaefer, MD, PhD, Inventor of the Year

Individuals and teams from the Mount Sinai Health System were honored for significant advances in research, technology, medicine, and health care at the fourth annual Mount Sinai Innovation Awards ceremony, which was held Monday, October 22, in conjunction with the SinaInnovations conference.

Anne Schaefer, MD, PhD, Associate Professor, Neuroscience, and Psychiatry, at the Icahn School of Medicine at Mount Sinai, and a
Seaver Fellow at The Friedman Brain Institute, received the Inventor of the Year Award for her work in micro-RNA, which heralds a potential cure for intractable seizures in certain forms of epilepsy, including Dravet syndrome, a catastrophic childhood disease.

Julio A. Aguirre-Ghiso, PhD, left, recipient of the Deal of the Year Award, with Scott L. Friedman, MD, Dean for Therapeutic Discovery, and leader of the SinaInnovations conference.

Julio A. Aguirre-Ghiso, PhD, Professor, Medicine (Hematology and Medical Oncology), Otolaryngology, and Oncological Sciences, received the Deal of the Year Award for his research into the underlying causes of metastatic disease and relapse that is the basis for a new startup company in New York City.

Drew Kiraly, MD, PhD, and James Young, MD, PhD, received the Faculty Idea Prize for analyzing more than 1,000 metabolites from the serum of patients undergoing active monitoring for epileptic seizure activity. The analyses of Dr. Kiraly, Assistant Professor, Neuroscience, and Psychiatry, and Dr. Young, an Instructor in Neurosurgery and Neurology, will serve as the first step in developing clinically applicable serum biomarkers to help refine and target treatment strategies for epilepsy.

A group of 22 innovators received the 4D Technology Development Program Award for five projects that efficiently move new technologies through a process of discovery, design, development, and delivery:

• Identification of biomarkers for preemptive diagnosis of ocular graft vs. host disease (oGVHD) in patients with hematopoietic cellular transplantation (HCT): Penny A. Asbell, MD; Neeta S. Roy, PhD; James L. Ferrara, MD, DSc; John E. Levine, MD; Eric Kuklinski, BS; and Yi Wei, PhD.

• Automation of radiographic measurements for surgical planning using artificial intelligence: Samuel K. Cho, MD; Varun Arvind, BS; Deepak Kaji, BA; Jun S. Kim, MD; Eric K. Oermann, MD; and Jonathan E. Robinson, MD.

• Development of an epigenetic treatment for Prader-Willi syndrome: Jian Jin, PhD; Yong-Hui Jiang, MD, PhD; and Yan Xiong, PhD.

• Creation of an eye-tracking algorithm for autism: Pilar Trelles, MD; Robert Gilman, MD; Alexander Kolevzon, MD; and Mirko Zimic, PhD.

• Knowledge-based automated radiotherapy planning via deep learning: Yading Yuan, PhD; Yeh-Chi Lo, PhD; and Tzu-Chi Tseng, MS.

The Dean’s Healthcare System Team Science Award, which acknowledges the importance of interdisciplinary teams in translational research, went to 15 members of the DEFINE-FMD Team. The group initiated a large, functional omics study of the genetic and molecular basis of fibromuscular dysplasia (FMD), a disease that predominately affects women and results in stroke and myocardial infarction. The study has enabled the team to identify several disease-causing candidates and begin developing a blood-based diagnostic test. The winners were Jason Kovacic, MD, PhD; Jeffrey W. Olin, DO; Antonio F. Di Narzo, PhD; Valentina d’Escamard,
PhD; Daniella Kadian-Dodov, MD; Haoxiang Cheng, PhD; Annette King, RN, ANP; Bhargravi Vonguru, MS; Emir Bander, MD; Allison Thomas, MS; Rihab Bouchareb, PhD; Sander Florman, MD; Johan LM Björkegren, MD, PhD; Manuel Mayr, MD, PhD; and Ke Hao, PhD.

Four individuals received Trainee Innovation Idea Awards, which highlight research ideas from Mount Sinai trainees that could potentially be translated into a marketable product:

• MD student Aly Valliani: Virtual Contrast

• PhD student Billie Bian: MediTrack

• Postdoctoral fellow Sangeetha Vadakke- Madathil, PhD: Placental stem cells for regeneration of an injured heart

• House staff physician Jorge Andrade Romo, MD: Structural vs. functional foveal avascular zone (FAZ) parameters compared at different stages of diabetic retinopathy.

Corporate sponsors for SinaInnovations included Altice Business; Cisco Systems; Dell Technologies; Fisher Scientific; Gilead Sciences; Jones Day; and the Louis and Rachel Rudin Foundation. Health Hackathon sponsors included Persistent Systems.

Health Hackathon Finds Technology-Based Solutions

Mount Sinai hosted its third annual Health Hackathon, an exciting health care innovation competition that ran from Friday, October 19, through Sunday, October 21. Participants included students from the Icahn School of Medicine at Mount Sinai and 12 other academic institutions, plus professionals with a wide range of backgrounds, such as clinical care, business, basic science, engineering, and software development.

They formed 16 teams that worked together over a 48-hour period to create innovative, technology-based solutions to problems in the field of rare diseases.

The winning teams:

Eye Can Do Technology that allows an immobile person to use eye movements to interact with devices in a smart-home environment.

Mango Tango A smartphone app, called Demeter, that helps patients with metabolic disorders track their diet and assess and manage symptoms.

Walk Thru An ambulatory walker with a portable attachment that helps the user get through self-closing doors without letting go of the walker.

“It’s pretty incredible what people can accomplish when they work with like-minded and not like-minded colleagues to create new ventures,” said Janice L. Gabrilove, MD, the James F. Holland, MD Professor of Medicine and Oncological Sciences, and Director, Clinical and Translational Research Education Program, Icahn School of Medicine.

The Health Hackathon is funded by ConduITS, the Institutes for Translational Sciences at the Icahn School of Medicine, with sponsorship from the software engineering company Persistent Systems. A diverse panel of judges chose three winning teams, which were each awarded a prize of $2,500. These teams, plus a fourth wild-card team, will be invited to participate in an Innovation Showcase sponsored by Mount Sinai Innovation Partners on February 14, 2019, where they will present their pitches to a panel of entrepreneurs.

 

Research at the Icahn School of Medicine at Mount Sinai Included in List of Most Influential in 2018

Two publications from researchers at the Icahn School of Medicine at Mount Sinai have been included in an annual listing of the most influential research publications.

The two publications were included in the annual Altmetric Top 100, which highlights research published in 2018 that has generated significant international online attention and discussion in forums including post-publication peer-review sites, public policy documents, the media, blogs, and social media platforms.

The two publications included in the list are:

  • “Structure and Distribution of an Unrecognized Interstitium in Human Tissues,” which was published in March in Scientific Reports. It was ranked number 14 on the list.
  • “Multiscale Analysis of Independent Alzheimer’s Cohorts Finds Disruption of Molecular, Genetic, and Clinical Networks by Human Herpesvirus,” which was published in June in Neuron. It was ranked number 74 on the list.

Altmetric, which is based in London, tracks and analyzes the online activity around scholarly literature. The firm collates what people are saying about published research outputs in scholarly and non-scholarly forums like the mainstream media, policy documents, social networks, and blogs to provide a more robust picture of the influence and reach of scholarly work.

Altmetric works with some of the biggest publishers, funders, and institutions around the world to deliver this data in an accessible and reliable format.

$43 Million Grant for Study of Environmental Effects on Children’s Health

Robert O. Wright, MD, MPH, and Rosalind J. Wright, MD, MPH

Researchers at the Icahn School of Medicine at Mount Sinai have been awarded a $43 million grant from the National Institutes of Health (NIH) to continue their work in a sweeping five-year research program called Environmental influences on Child Health Outcomes (ECHO). The national ECHO study aims to evaluate how a range of environmental factors, also known as the exposome, affect health during childhood, adolescence, and the transition into adulthood. At Mount Sinai, this work builds on the expertise and resources of the Institute for Exposomic Research, which is co-directed by two international leaders in the field, Rosalind J. Wright, MD, MPH, Horace W. Goldsmith Professor of Pediatrics, and Dean for Translational Biomedical Sciences at the Icahn School of Medicine; and Robert O. Wright, MD, MPH, Professor and Ethel H. Wise Chair of the Department of Environmental Medicine and Public Health.

“Exposomic research is changing the way medicine is practiced by finally addressing the largely unstudied role that our environment plays in our health—and doing so on the grandest possible scale,” says Dr. Robert Wright. In the ECHO program, institutions across the nation will study up to 50,000 families, investigating the influence of environmental exposures on four key areas: perinatal outcomes such as preterm birth; child growth and obesity; respiratory disorders; and neurobehavioral and cognitive difficulties that may affect social and academic functioning in the longer term. The Wrights are principal investigators of a regional ECHO consortium, including sites in New York City, Boston, and Virginia, that will study 5,000 women and children, measuring the exposome across their diverse ethnic backgrounds.

“Synergistic programs”

Mount Sinai has also received two related NIH grants, for a total of $19.4 million, that fund its participation in the Children’s Health Exposure Analysis Resource (CHEAR) program, which is designed to support ECHO. One CHEAR grant funds a Laboratory Hub, led by Dr. Robert Wright, which analyzes environmental chemicals, metabolites, hormones, and other factors. The other supports the Data Repository, Analysis, and Science Center, led by Susan Teitelbaum, PhD, Professor, Environmental Medicine and Public Health, which conducts big data analysis. Across the nation, there are six CHEAR laboratory hubs, and one data center, at Mount Sinai. “ECHO and CHEAR are naturally synergistic programs,” says Dr. Robert Wright. “CHEAR will run exposomic assays on samples collected by ECHO that can identify thousands of chemicals to which children are exposed, and can systematically study which are toxic, which are harmless, and which are beneficial.”

In 2016, Mount Sinai won a large NIH grant during the development phase of ECHO, and since then Dr. Robert Wright and Dr. Rosalind Wright have been part of a select national committee that has shaped the program. Now they are helping set protocols for the researchers who will follow up with mothers and children at multiple stages of life—collecting samples of blood, urine, hair, saliva, and placental tissue, and gathering data on birth weight, preterm birth, growth, cognitive development, and lung development.

Informing better health

One overarching goal of the ECHO study is “solution-oriented research,” Dr. Rosalind Wright says. “We are not just looking for risk factors, we are also measuring factors that might be protective and build resilience. For example, there is evidence that higher intake of antioxidants such as vitamin C and anti-inflammatory fatty acids, like omega-3 fish oils, can buffer the effects of stress and other toxins. We are now looking at the effect of diet on exposure to air pollution and chemical toxicities,” she says. “People in the ECHO program are really excited—we are all in this because we want our research to inform better health.”

National Cancer Institute Leader Visits Mount Sinai

From left: Luis M. Isola, MD, Professor of Medicine (Hematology and Medical Oncology), and Pediatrics; Ramon Parsons, MD, PhD, Director, The Tisch Cancer Institute; Norman E. Sharpless, MD, Director, National Cancer Institute; and William Oh, MD, Deputy Director, The Tisch Cancer Institute, and Associate Director of Clinical & Translational Research for the Institute.

Aging is one of the greatest risk factors for developing cancer, which is most frequently diagnosed among people aged 65-74. But there are no simple explanations for the “multifaceted” science behind this connection, according to Norman E. Sharpless, MD, Director of the National Cancer Institute (NCI).

On Thursday, September 13, Dr. Sharpless addressed the topic in a seminar titled “The Dynamic Interplay between Cancer and Aging,” which he presented before a standing-room-only crowd in Davis Auditorium on The Mount Sinai Hospital campus. Dr. Sharpless has devoted much of his career to studying the connection between cancer and aging. Developing a better understanding of this relationship is particularly important, he said, because people over the age of 65 make up the fastest growing segment of the nation’s population.

Dr. Sharpless was invited to speak about his own research by The Tisch Cancer Institute at the Icahn School of Medicine at Mount Sinai, where he spent the earlier part of the day meeting with leaders of the Institute and medical school. He had lunch with postdoctoral fellows and students who work in Mount Sinai’s cancer-focused laboratories, and met with faculty and staff who oversee Mount Sinai’s Cancer Center Support Grant. The grant provides Mount Sinai with its NCI designation as one of an elite group of U.S. institutions committed to the research and treatment of cancer.

During the day’s meetings, Dr. Sharpless shared his vision for NCI programs and discussed trends in funding and cancer research. The Tisch Cancer Institute received its NCI designation for the fi rst time in 2015 and is preparing to renew the competitive grant in 2019. Since his appointment to the NCI in 2017, Dr. Sharpless has spent time visiting NCI-designated cancer centers around the country. On his recent trip to New York City, he also visited the Albert Einstein Cancer Center.