This winter, it’s not just Philadelphians who have bundled up against the cold. The Pavilion—Penn Medicine’s new, $1.5 billion hospital set to open in 2021—also received a weather-proofed “jacket” for the season. With the building’s exterior wrap in place, the construction team has turned attention to the 500 patient rooms, 47 operating rooms, and other spaces for patients, their families, and hospital staff.
A Medical Milestone and an “Impossible” Birth
When Jennifer Gobrecht was 17, doctors told her that she would never carry her own child. But in November 2019, she achieved a seemingly impossible dream: she delivered a son. Benjamin Thomas Gobrecht was the first baby born as part of Penn Medicine’s Uterus Transplantation for Uterine Factor Infertility trial and the second baby in the United States to be born using a transplanted uterus from a deceased donor. “
“As a woman it meant everything to me to be able to have that journey of becoming parents in the way that most people around you get to experience,” Jennifer said. “It’s all thanks to a truly incredible team of doctors and nurses and the selfless donor who made my dream of motherhood come true.”
PSOM Ranks 3rd in the Nation for NIH Research Grants: Breakdown by the Numbers
The Perelman School of Medicine (PSOM) consistently ranks among the top medical schools in the nation in research funding from the National Institutes of Health (NIH). In fiscal year 2019, PSOM received more than $493 million in awards across 970 projects, earning the third highest total among medical schools. This federal funding empowers Penn investigators to make inroads in a wide range of research areas, including those highlighted in the examples below.
“The NIH funding we receive attests to the excellence of our faculty and the impact of their groundbreaking discoveries,” said J. Larry Jameson, MD, PhD, executive vice president of the University of Pennsylvania for the Health System and dean of the Perelman School of Medicine. “It also reflects the outstanding breadth of Penn Medicine’s research enterprise and the many ways in which it is improving health.”
500,000 PEOPLE are living with Parkinson’s disease in the United States. Penn is leading a multi-institutional effort to detect Parkinson’s in the brain and track its progression through imaging. Supported by a five-year, $20 million grant, the team aims to create two tracers that will bind to specific proteins in the brain. This study has the potential to shift the ways in which researchers utilize molecular imaging tools and improve the study of Parkinson’s treatments.
4,200 PATIENTS who received lung transplants will become part of a study to better understand the biological processes that can lead to post-transplant complications. Patients may experience a drop in lung function—a symptom of a form of chronic lung allograft dysfunction (CLAD) that is the leading cause of death among lung transplant recipients. Funded by a seven-year, $9.8 million grant, researchers will perform long-term phenotyping using biosamples, as well as data collected at routine clinical visits and exams conducted at six-months intervals to assess lung function and quality of life. The multi-site study aims to better understand the mechanisms that drive CLAD, who is at heightened risk for its different forms, and how to develop targeted treatments that improve long-term, post-transplant outcomes.
26 INVESTIGATORS across the U.S. and U.K. received a five-year, $9.7 million grant to establish CONNECT-TBI, a program that studies traumatic brain injury (TBI) and neurodegenerative diseases. The program is investigating the effects of TBI, which often include changes like memory loss, confusion, and depression. The team is also exploring the mechanisms of TBI-related neurodegeneration (TReND), and aims to define all subtypes of TReND and understand their progression. Of particular interest is chronic traumatic encephalopathy, a condition associated with repeated concussions in contact sports.
5 RESEARCH GRANTS totaling more than $22 million were awarded to teams exploring opioid use disorder (OUD) prevention and treatment. In the prevention effort, Penn is studying interventions that reduce pain and opioid use among patients with kidney failure as the lead institution of the Hemodialysis Opioid Prescription Effort (HOPE) consortium. Seven Penn Medicine sites were also designated specialized clinical centers for the NIH’s Early Phase Pain Investigation Clinical Network (EPPIC-Net). Projects aiming to improve treatment include a study of the impact of a collaborative care model—which places social workers trained in mental health care in primary care offices—on the treatment of OUD and psychiatric disorders. (See “Ending the Isolation” on p. 22.) Another team is exploring the use of extended-release injectable naltrexone to reduce overdose and relapse risk. Researchers are also developing neuroimaging methods to assess the impact of opioid exposure on early brain development.
Penn Forges Powerful Philly Partnerships
A celebration of this new partnership was held at the Perelman Center for Advanced Medicine. Gritty, the iconic Philadelphia Flyers mascot, got down on one fuzzy knee and shared a moment with Brian Sennett, MD, following the announcement.
Home Team Hat Trick
Whether a Philadelphia Flyers hockey player is injured on the ice, an arena staff member is sick, or Gritty suffers a mishap with a t-shirt cannon, Penn Medicine providers will be there to offer care. In January, Penn Medicine announced a major partnership with Comcast Spectacor, becoming the official health system for the Flyers and Wells Fargo Center. Penn Medicine will serve as the team’s official medical services provider, the preferred provider for front office employees, and a teammate in joint community health initiatives.
“Not only will we provide the most advanced care for these players as needed on game nights, but they will also have access to the wide range of providers off the ice,” said Brian Sennett, MD, chief of Sports Medicine and vice chair of Orthopaedic Surgery. “We look forward to working with the team, and to keeping the players safe and healthy for many seasons to come.”
Same SEPTA Station, New Neighborly Name
SEPTA’s Regional Rail riders who have passed through West Philadelphia have been able to watch Penn Medicine’s new Pavilion rise higher and higher with each daily commute—but that likely isn’t the only major development they’ve noticed. Following a newly forged partnership between SEPTA and the University of Pennsylvania Health System (UPHS), University City Station has been renamed Penn Medicine Station. In January, new signage, maps, and interactive screens were unveiled, and Penn and SEPTA aim to to identify other opportunities to enhance the rider experience for the 6,400 passengers who pass through the station each day.
As the construction of the Pavilion enters its final stages, a connecting bridge will be constructed to facilitate easier, more convenient travel between the station and the hospital for patients, visitors, and staff. “As the gateway to the Pavilion and our West Philadelphia medical campus, thousands of people each day pass through this SEPTA station en route to work, visit, and receive care in our facilities,” said Kevin Mahoney, CEO of UPHS. “We’re so thrilled for the opportunity to have the same station with a healthy new name.”
Penn HealthX Endowment Supports Students’ Entrepreneurial Spirit
Since 2013, Penn HealthX—a student-run organization within the Perelman School of Medicine—has empowered students who are interested in health care management, entrepreneurship, technology, and business to explore the many ways that they can use their medical degree outside of the clinic. Now, an endowed fund will create a scholarship to support medical students pursuing an MBA at the Wharton School of the University of Pennsylvania. The Roderick T. Wong Penn HealthX Program was made possible through the generosity of Roderick Wong, MD’03, a member of the program’s advisory board and a managing partner of RTW Investments LLC, a health care investment firm in New York.
Penn HealthX is integrated into the medical school curriculum and awards a Certificate in Healthcare Management, Entrepreneurship, and Technology. The nationally recognized program offers opportunities to connect with visionary faculty, alumni, and industry leaders through an annual conference and regular interdisciplinary seminars and workshops. The HealthX Venture Fund also offers grants of up to $5,000 to teams developing projects to fill health care gaps. This new endowment will offer an additional level of financial support to students as they design and pursue innovative ventures that can shape the future of health care.
Tree of Life Tragedy Plants Seeds for Community Health
As Elliot C. Rabinowitz, MD’12, stood before dozens of staff, faculty, and supporters in the Perelman Center for Advanced Medicine on a blustery December afternoon, he admitted that it would be hard to sum up his “quirky and inspiring uncle Jerry.” Jerry Rabinowitz, BA’73, MD’77, had an infectious belly laugh, a penchant for bow ties, and a talent for sharing pearls of wisdom when you needed them most. He held patients’ hands without gloves at the height of the HIV/AIDS epidemic when many other doctors were uncomfortable and unfamiliar with the disease. He regularly visited his elderly patients long after his peers stopped making house calls. To the family physician, “medicine was about creating relationships and making sure people felt seen and heard.”
In 2018, Rabinowitz was nearing retirement and shared his plans to pay it forward by getting involved in the training of the next generation of doctors. But on October 27, he was among 11 congregants killed at the Tree of Life synagogue in Pittsburgh by a gunman who had shared anti-Semitic conspiracy theories on social media; Rabinowitz had run toward the sound of gunfire in an effort to tend to his wounded community members. Rabinowitz was unable to directly share his knowledge and experience with young physicians, but his legacy lives on—both in the stories shared by the family, friends, and patients who adored him, and now through a new scholarship named in his honor.
“In his humanistic approach to care and his commitment to serving the community, Dr. Rabinowitz exemplified the mission of family medicine,” said Matthew Press, MD, MSc, interim chair of Family Medicine and Community Health in the Perelman School of Medicine. “What an inspiring legacy for our students, our trainees, our faculty, and for me personally.” The Jerry Rabinowitz, C’73, M’77 Memorial Scholarship is an endowed scholarship that will be awarded annually to a Penn third-year family medicine resident who embodies Rabinowitz’s dedication to treating not just an illness or injury, but the whole patient, as well as his selfless devotion to community outreach.
This year, Sarah L. Smith-Benjamin, MD, received the inaugural scholarship. Described as a role model for her peers, an asset to Penn, and a positive force in the community, during her residency, she has volunteered as a preceptor at the Heart Health Bridge to Care Clinic—an interdisciplinary, student-run clinic that helps uninsured patients in West Philadelphia manage their hypertension.
“When I was a child and thought about becoming a doctor someday, Dr. Rabinowitz was the kind of physician I imagined being,” Smith-Benjamin said. “During my journey to becoming a physician, there have been times when I’ve felt caught up in the minutiae of the day-to-day and frustrated with the things that make it hard to connect with patients. Every time this happens, I go back to the community. There’s a lot to be said for meeting people where they are to empower them to improve their health. Dr. Rabinowitz knew about that very well, and I’m honored to carry his legacy with me into all of my future work.”
“Harnessing the Power” of Penn Providers to Combat the Opioid Crisis
As the nation grapples with the devastating impact of the opioid epidemic, overdoses continue to occur at an alarming rate. According to the Centers for Disease Control and Prevention, 130 Americans die each day from an opioid overdose—and about three of them are from Philadelphia. Staff across Penn Medicine have been hard at work combatting the deadly toll of opioid use disorder (OUD) at every point of contact. They are reducing prescribing and introducing alternative pain management protocols during all kinds of patient encounters. For patients with OUD, they offer medication-assisted treatment in the emergency department and connect patients with certified recovery specialists. To save lives, they provide free naloxone trainings in the community. To grow their impact, they lead dedicated research efforts.
Central to this work has been Jeanmarie Perrone, MD, a professor Emergency Medicine, director of Medical Toxicology, and a founding member of Penn Medicine’s opioid task force. Under the leadership of Perrone, the newly launched Penn Medicine Center for Addiction Medicine and Policy will enhance the health system’s already robust prevention, treatment, and education efforts. The center will focus on developing harm reduction policies—including naloxone dissemination—and addiction treatment initiatives, all with the goal of increasing access to evidence-based therapies, improving patient outcomes, and mitigating the stigma of OUD.
“Through this center, we’re harnessing the power of many providers across Penn Medicine to integrate evidence-based treatments for substance used disorders in both our hospitals and outpatient practices,” Perrone said. “It’s our hope that we will be able to continue evolving our standard of care to support this vulnerable patient population.”
“It’s Not Just My Cure I’m Chasing”
David C. Fajgenbaum, MD’13, MBA’15, MSc, was in his third year at the Perelman School of Medicine when he finished an exam, walked down the hall to the emergency department, and received news that changed the course of his life. Fajgenbaum had been a Division 1 athlete at Georgetown University and fastidiously maintained a healthy diet and a rigorous exercise routine, so when he started experiencing night sweats, fatigue, abdominal pain, and fluid accumulating in his legs, the quarterback-turned-medical student knew it was more than a common cold. During that fateful ED visit in 2010, he learned that his liver, kidneys, and bone marrow were all failing, and he needed to be hospitalized immediately—and by the time he was given the formal diagnosis of idiopathic multicentric Castleman disease (iMCD) a few months later, he was near death.
Over the decade that has followed, Fajgenbaum has undergone intense bouts of chemotherapy, experienced four near-fatal relapses, and had his last rites read to him multiple times. He also graduated medical school, married his college sweetheart, became a father, wrote a book, and found a drug that has kept him in remission for nearly six years. Now an assistant professor of Medicine in Translational Medicine and Human Genetics at PSOM, executive director of the Castleman Disease Collaborative Network, and director of the Center for Study & Treatment of Castleman’s & Inflammatory Lymphadenopathies, Fajgenbaum is paving the way for new treatment options for patients like him who don’t respond to siltuximab, the only FDA-approved therapy for the disease.
Castleman’s disease—which is diagnosed in about 5,000 people of all ages each year in the United States—is like a rare hybrid of an autoimmune condition and cancer. iMCD is the most severe subtype, with 35 percent of patients dying within five years of diagnosis. Fajgenbaum currently serves as both the principal investigator and Patient 1 in a clinical trial to determine whether Sirolimus, the drug he initially tested on himself in 2014, can lead to sustained remission in other patients.
The possibility of relapse can feel like a sword of Damocles hanging over him, but it inspires action, Fajgenbaum said in a conversation on the Ten Percent Happier podcast. “I have the opportunity based on the laboratory that I run and the work that I do to really make a difference. It’s not just my cure that I’m chasing,” he said. Thousands of rare diseases do not currently have a FDA-approved drugs to treat them, but just as Fajgenbaum found unexpected success with an immunosuppressant for patients with kidney transplants, there may be other options hiding in plain sight.
“Sometimes we hope, pray, and wish for something to happen, and we stop there. I hoped someone would find a drug for me, that progress would be made. But then I realized that if I wanted it to happen, I needed to do it,” he said. As a man “living in overtime,” every second counts. Fajgenbaum plans to spend those seconds—however many he has left—finding answers and driving change.
From Micro to Macro
Whether exploring cellular mechanisms to engineer breakthroughs or eliminating barriers to enhance community health, Penn Medicine researchers are tackling questions that will impact the immune system and public health system alike.
Immune Cell Champions
A CRISPR First: A groundbreaking clinical trial—the first of its kind performed in human patients in the United States—has found that immune cells edited using CRISPR/Cas9 technology can persist, thrive, and function months after a cancer patient receives them. Researchers from the Abramson Cancer Center partnered with the Parker Institute for Cancer Immunotherapy and Tmunity Therapeutics to edit T cells and safely infuse them back into the patients, two with multiple myeloma and one with sarcoma. Three edits were made to reprogram the cells to seek out and destroy tumors, followed by the insertion of a T cell receptor that told the edited cells to target a specific antigen. This marked the first-ever sanctioned investigational use of multiple edits to the human genome. This work is the latest milestone in Penn’s history as cellular and gene therapy pioneers.
GPS for CAR T Cells: When engineered CAR T cells are infused into a patient’s body to fight disease, where do they go, and how long do they keep fighting? Penn researchers developed a novel way to track these cells in mouse models. First, they tagged the T cells with a bacterial protein—a reporter gene—to differentiate them from normal immune cells. Then, they created a radiotracer with an affinity for the bacterial protein, which caused the tagged CAR T cells to “light up” on a PET scan. This allowed the team to track their movements in real-time. The research team plans to test the reporter gene/radiotracer pairing in a clinical trial with humans.
T Cells Get Tired, Too: Though the immune system is supposed to combat disease, T cells can become “exhausted” and ineffective. To determine what triggers exhaustion, Penn researchers worked backwards, examining the molecular mechanisms of newly formed “precursor” T cells. They found that a key transcription factor known as TCF-1 programs a cell’s trajectory toward either exhaustion or becoming an “effector” cell that fights disease and potentially acts as a self-renewing “memory” cell that can respond rapidly in the event of another infection. Understanding the developmental path of T cells opens up the possibility of re-wiring them to prevent exhaustion.
Public Health Pioneers
Economic Erosion and Opioids: When automotive assembly plants close, a rise in opioid-related deaths is likely to follow. A Penn-led study examined these deaths over a 17-year period in 112 manufacturing counties, 29 of which experienced the closure of an automotive assembly plant during the study period. Five years after these plants’ closures, overdose mortality rates were 85 percent higher than anticipated when compared to unaffected counties. These rates were particularly high among non-Hispanic white men between 18 and 34 years old, as well as their 35-and-older counterparts. These findings suggest that declining economic opportunities, combined with increased access to overprescribed medications, caused the opioid crisis to hit these particular areas hardest.
Infant Vaccine Innovation: Why can’t infants receive vaccines for illnesses like the flu and chickenpox soon after birth? It’s all down to the maternal antibodies still in their systems, which protect them in some ways, but also fight back—too hard—against vaccines. Waiting six to 12 months for the antibodies to subside can leave infants susceptible to diseases that can be prevented in older children. Penn researchers explored whether a mRNA-LMP (nucleoside-modified mRNA encapsulated in lipid nanoparticles) vaccine—which provokes a more powerful immune response than a traditional vaccine—could overcome maternal antibodies in young mice. They found that it slipped under the antibodies’ radar, prompting the mouse’s own immune system to respond.
When Zip Code Affects Survival: If you experience sudden cardiac arrest, your location may have a bigger impact on your chance of survival than you realize. Penn researchers examined data from more than 27,000 cardiac arrest events and found that people who live in largely Hispanic communities are less likely to receive bystander CPR when compared to people living in predominantly non-Hispanic neighborhoods—resulting in a staggering 44 percent lower likelihood of survival. By partnering with local organizations that serve Philadelphia’s Latinx and Hispanic communities like Congreso de Latinos Unidos, developing educational initiatives that break down language and economic barriers, and providing accessible CPR training through the Mobile CPR Project, Penn aims to address this disparity.
Honors & Awards
The honors and awards listed below are just a few of the highlights among Penn Medicine’s highly lauded leaders, faculty, staff, and trainees. For more honors, click here.
Virginia M.Y. Lee Earns $3 Million Breakthrough Prize
While Americans are living longer, debilitating neuro-degenerative diseases are compromising quality of life for millions across the nation and placing a tremendous financial strain on the health care system. But what if we could understand what prompts brain and nervous system cells to function abnormally and use those discoveries to slow the progression of neurodegenerative diseases—or prevent them altogether?
Virginia M.Y. Lee, PhD, MBA’84, director of the Center for Neurodegenerative Disease Research and the John H. Ware 3rd Professor in Alzheimer’s Research in the Department of Pathology and Laboratory Medicine at the Perelman School of Medicine, is dedicated to answering that question. Lee was recently awarded the Breakthrough Prize in Life Sciences by the Breakthrough Prize Foundation, which is sponsored by a cohort of prominent Silicon Valley figures: Sergey Brin, Priscilla Chan and Mark Zuckerberg, Ma Huateng, Yuri and Julia Milner, and Anne Wojcicki. Lee was recognized for her trailblazing research studying underlying mechanisms of neurodegenerative diseases.
By investigating how different forms of misfolded pathological proteins travel from cell to cell, Lee aims to understand the roles they play in the progression of Alzheimer’s, Parkinson’s, and other dementias and movement disorders. This transformative research serves as a springboard to identify advanced treatments—work that will be supported by the $3 million prize.
“I am really optimistic that maybe some treatment for Alzheimer’s and Parkinson’s will become available in the next, let’s say, one or two decades,” Lee said. “I am deeply honored to be a recipient of the Breakthrough Prize. It is very gratifying to know that our research on neurodegenerative diseases is being recognized by the scientific community.”
American College of Physicians Honors Jonathan A. Epstein
Established in 1958, the Harriet P. Dustan Award for Science as Related to Medicine is bestowed annually by the American College of Physicians (ACP) upon a nominee whose outstanding scientific work has contributed to the advancement of internal medicine and merited national or international recognition. During the ACP’s scientific conference in April 2020, this award will be presented to physician-scientist Jonathan A. Epstein, MD, Executive Vice Dean and Chief Scientific Officer of the Perelman School of Medicine, and the William Wikoff Smith Professor of Cardiovascular Research.
Epstein is a renowned stem cell biologist, developmental biologist, and cardiovascular biologist. His work primarily focuses on the molecular mechanisms of cardiovascular development and has directly impacted the creation of new therapeutic agents for heart failure and myocardial infarction. This past fall, Epstein and his team published a first-of-its-kind study that found CAR T-cell therapy could be a viable treatment for heart disease. The researchers used genetically modified T cells in mouse models to target and remove fibroblasts that contributed to cardiac fibrosis, or stiffening of the heart. While in its early stages, this research “marks a significant step forward in our efforts to treat—and potentially reverse—a condition that accelerates the progression of heart failure,” he said.
Six Faculty Members Elected to National Academy of Medicine
Six faculty members from the Perelman School of Medicine at the University of Pennsylvania were elected to the National Academy of Medicine—one of the nation’s highest honors in biomedicine. They were among 90 new U.S. and 10 international members elected by their peers for accomplishments and contributions to the advancement of the medical sciences, health care, and public health.
This year’s members include Charles S. Abrams, MD, GME’91, the founding director of the Penn/Children’s Hospital of Philadelphia (CHOP) Blood Center for Patient Care & Discovery, vice-chair for research and chief scientific officer in the Department of Medicine, and the Francis C. Wood Professor in the Department of Pathology and Laboratory Medicine; Beverly L. Davidson, PhD, director of the Raymond G. Perelman Center for Cellular and Molecular Therapeutics, chief scientific strategy officer at CHOP, the Arthur V. Meigs Chair in Pediatrics at CHOP, and a professor of Pathology and Laboratory Medicine; George Demiris, PhD, FACMI, a Penn Integrates Knowledge University Professor with joint faculty appointments in the School of Nursing and in the Department of Biostatistics, Epidemiology and Informatics; James Eberwine, PhD, the Elmer Holmes Bobst Professor of Pharmacology and co-director of the Penn Program in Single Cell Biology; Stephan A. Grupp, MD, PhD, director of CHOP’s Cancer Immunotherapy Program and Translational Research for the Center for Childhood Cancer Research, and a professor of Pediatrics; and Guo-li Ming, MD, PhD, the Perelman Professor of Neuroscience and a member of Institute of Regenerative Medicine.
Eydie Miller-Ellis Receives FOCUS Award for Advancement of Women in Medicine
Eydie Miller-Ellis, MD, is committed to ensuring that underrepresented groups are seen and heard. For example, she connects with her African American patients through their shared background and uses her family history with glaucoma to help patients better understand the disease. She is also proud to serve as a co-investigator of Penn’s Primary Open-Angle African-American Glaucoma Genetics study, which focuses on glaucoma in patients of African descent—a group historically excluded from genome-wide association studies. (See “Correcting a Blind Spot” in Penn Medicine, Spring/Summer 2018.)
Miller-Ellis serves as the chief of Glaucoma Service and director of the Glaucoma Fellowship Program at the Scheie Eye Institute, vice chair for Faculty Affairs and Diversity, and a professor of Clinical Ophthalmology—and still finds the time to mentor her peers. She also serves as the co-director of the Rabb-Venable Excellence in Research Program for the National Medical Association, Ophthalmology Section, through which she aims to increase the number of underrepresented groups in ophthalmology residencies and academic ophthalmology.
Miller-Ellis' commitment to the advancement of women and people of color in medicine earned her the 2019 FOCUS Award for the Advancement of Women in Medicine. This award is presented annually to a faculty member whose efforts have promoted the success, leadership, and quality of life for Penn women in academic medicine.
“Many women struggle to balance their professional and family responsibilities, and finding visible role models who understand the special challenges face in academia can be difficult." Miller-Ellis said. "I am now in a position to ensure junior faculty have access to opportunities to speak at national meetings, work on committees, build their confidence, and advance their careers. I do my best to support women as they navigate these daily challenges.”