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FEATURES
Game-Changer
Super-charged by philanthropy, our experts are forging the future of pediatric surgery.
A family’s transformational gift is helping to create healthier tomorrows for families around the globe.
The Ripple Effect
One key to solving the mental health crisis is strong partnerships between Boston Children’s and schools.
DEPARTMENTS
Profiles
10 Dr. Stuart Orkin
22 Dr. Maria Gutierrez-Arcelus
Big Questions
14 How Do You Turn $30m into $360m?
28 Can an Immediate Impact Last Forever?
New Funding
Accelerating Autism Research
Engineering an End to Joint Pain
“Truly Life-Changing”
THIS ISSUE OF Boston Children’s Hospital Magazine is filled with celebrations—some in the making for decades.
Near and dear to the scientific community and the 20 million people globally impacted by sickle cell disease is Casgevy, a ‘breakthrough’ gene therapy treatment that is truly life-changing for afflicted patients. Countless researchers contributed to the work; however, one stands out.
It’s a pleasure to see accolades pour in for Stuart Orkin, who has devoted much of his remarkable career to making the fundamental discoveries and devising the therapeutic strategy behind Casgevy. I had the privilege of training in Stu’s laboratory very early in my own career, so I’ve seen this story develop almost from the beginning.
Stu’s work—his commitment to discovery, to solving difficult problems, to absolute excellence— exemplifies the Boston Children’s community. I believe this mindset stems from our unique environment, where research and care are fused. Our patients inspire our scientific inquiries and, so often, our findings directly impact the children and families in our care.
I’m especially excited to be at Boston Children’s now. We’re seeing a convergence of developments, like widespread use of computation and artificial intelligence, a rapidly growing armamentarium of approaches to treat disease and new applications in nanotechnology, that are advancing earlier diagnoses and more effective personalized therapies.
As philanthropists, you play a critical role in this important work, enabling researchers to pursue their boldest ideas and transform them into tomorrow’s breakthroughs.
On behalf of Boston Children’s—thank you. Thank you for championing science, for driving innovation and for caring about children everywhere. Your support means the world to us.
With best wishes,
NANCY C. ANDREWS, MD, PHD EXECUTIVE VICE PRESIDENT AND CHIEF SCIENTIFIC OFFICER
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Again this season, Nick Pivetta is serving as our Red Sox champion for Boston Children’s Hospital. His community program includes hospital bedside visits and treating discharged patients and their families to a special night at Fenway Park. Thank you, Nick!
JOYFUL
Seize the joy and celebrate life’s special moments with our families, caregivers and fans— even when they’re superstars themselves. Join us at #PauseForJoyBCH
YALL!!! I AM SO SHOOK! OTB WITH AN ABSOLUTE LEGEND, MARRYANNE! Her and her pupper Cooper are actually 10 year veterans of the Pawprints pro- gram! Cooper actually just won an award not long ago! Marryanne absolutely adores helping out the kiddos in every possible way and stepped right up to the plate! Without a single hesitation she hopped on board at $1000/year! my highest donor ever!! THANK YOU SO MUCH MARRYANNE AND COOPER!!
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4 years ago today my daughter got her second chance at life all because of an amazing surgeon. Forever grateful. I will never stop celebrating this day!
Peter Weinstock
Once in a lifetime when you get to work and collaborate with a childhood friend (an ‘at my wedding’ kinda friend) on something groundbreaking for healthcare. AND it gets featured on Must See TV!!
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Had the honor to work with my dear friend Dr. Darren Orbach and his incredible team at Boston Children's Hospital Interventional Radiology to deliver the first fetal intervention to repair a brain Vein of Galen Malformation (VOGM) prior to birth. Combination of cutting edge interventional and surgical techniques by Dr. Orbach’s team, assisted by 3D printing and immersive tech delivered by Immersive Design Systems at Boston Children’s Hospital. Awesome partnership to make the impossible possible!
Quick photo before we are drenched! A little rain won’t stop us from repping team
Surgical Innovation
Game-Changer
AN INCREDIBLE TEAM, STACKED WITH THE WORLD’S MOST TALENTED PHYSICIANSCIENTISTS AND SUPER-CHARGED THROUGH PHILANTHROPY, IS FORGING THE FUTURE OF PEDIATRIC SURGERY.
Game-Changer
CANCER COULD HAVE SIDELINED CHAD. INSTEAD, IT’S BEEN A TRIUMPHANT RETURN TO THE FIELD FOR #10.
“It completely blindsided me.”
CHAD HAS BEEN devoted to lacrosse since the first grade. His parents, Keith and Jen Palumbo, rare ly miss the chance to watch him play. The family was at a tournament when Chad noticed the lump.
Keith and Jen wasted no time. The next day, Chad, then 17, and his parents met with an adult urologist, who assured them the lump was almost certainly benign. But surgery revealed rhabdomyo sarcoma, a rare pediatric cancer.
“It completely blindsided me,” says Chad. Fortunately, when Keith and Jen started search ing for a medical center with expertise in the con dition, they didn’t have to look far.
Richard Lee, MD, director of pediatric urolog ic oncology, worked with Natalie Collins, MD, PhD, at Dana-Farber/Boston Children’s Cancer and Blood Disorders Center, to develop a com prehensive treatment plan that included robot-as sisted laparoscopic surgery and chemotherapy. Understanding the route the tumor would take if it metastasized, Dr. Lee removed several lymph nodes, using minimally invasive techniques. Ro botic surgery helped shorten Chad’s recovery time, allowed for extremely precise removal of the po tential cancerous tissue and facilitated nerve-spar ing techniques.
The team even advised Chad about staying in shape during treatment, which enabled him to prepare for his eagerly awaited return to lacrosse, says Keith. “When you meet experts who know ex actly what they’re dealing with,” Jen says, “it takes away so much uncertainty.” After their son’s re covery, Keith and Jen saw an opportunity to bring high-quality surgical care to more kids.
The couple established the Palumbo Family Directorship in Minimally Invasive Surgery and an additional research and training fund, with the goal of fueling innovation in robotic surgery and disseminating expertise to hospitals around the world. “The Palumbos’ commitment gives us an amazing opportunity to reduce the impact of surgery for countless young people,” says Dr. Lee.
Keith recently joined Boston Children’s Hospital Trust Board and is focused on advancing the hos pital’s mission through philanthropy. The family also shares their insights with others who receive a rhabdomyosarcoma diagnosis.
“When someone has saved your child’s life,” says Jen, “you can’t do enough to thank them.”
ROBOTS: A KINDER CUT
Robotic surgery allows surgeons to make the smallest and fewest incisions possible to accomplish a task, which decreases the impact of surgeries. It reduces pain, recovery time and scarring and allows for more intricate procedures. Children return to school, family and friends sooner than when undergoing traditional 'open' operations. These benefits are especially important for children with complex disorders, who will face multiple surgeries before they reach adulthood.
Surgical Innovation
PART 2
WHAT’S ON THE HORIZON?
We asked some of our surgical chiefs about the future innovations they’re most excited about. Here’s what they had to say.
THE MARK OF EXPERTISE
“Urological conditions can cause serious kidney problems, but we have no way to identify which children are at risk. Our team has focused on identifying biomarkers of kidney damage, and soon, we’ll use these to individualize care and prevent damage.”
Carlos R. Estrada Jr., MD, MBA
Urologist-in-Chief
CURES BEFORE BIRTH
“It’s not a huge leap from curing congenital disorders to preventing them from happening in the first place. Boston Children’s has an incredible opportunity to join our rich expertise in gene and cell therapies with our newly expanded Fetal Care and Surgery Center. This marriage of basic science and fetal intervention will open the door to stem cell treatment, enzyme replacement and, eventually, gene therapy administered in utero.”
Steven J. Fishman, MD Surgeon-in-Chief and Chief of Surgery
TRACING THE ROOTS
“In craniofacial surgery, we’re exploring the genetic origins of many disorders that bring children into the operating room. We’re now looking to molecular biology to help us understand how anomalies develop and advance new treatments that reverse the effects of these diseases.”
John G. Meara, MD, DMD, MBA Plastic Surgeon-in-Chief
• • •
INDUCING INJURIES TO HEAL THEMSELVES
“In orthopedics, there have long been injuries that struggle to heal, requiring removal of the damaged tissue and replacement with metal and plastic or tissue taken from other places. I’m most excited about the possibility of using biomaterials to enable the regeneration of those tissues. I hope the launch of our Center for Orthopedic Research Translation will enable our laboratory discoveries to become the treatments and cures of tomorrow.”
Martha Murray, MD Orthopedic Surgeonin-Chief
VALVES THAT GROW WITH A CHILD’S HEART
“Valve disease—and a lack of appropriate options— continues to be the key driver of shortened lifespans for kids with heart defects. The biggest breakthrough coming in pediatric cardiovascular surgery is going to be the work we and others are doing to build synthetic heart valves designed for children.”
Pedro J. del Nido, MD Chairman, Department of Cardiac Surgery
SMILES WITHOUT SURGERY
“Surgical removal of benign, but aggressive, jaw tumors can cause functional and aesthetic issues. New medications could offer a noninvasive alternative therapy that won’t harm healthy tissue.”
Bonnie L. Padwa, MD, DMD
Oral Surgeon-in-Chief
ACTING SOONER TO SAVE THE BRAIN
“We’re shifting to earlier intervention, minimally invasive procedures and repair—rather than removal—of tissue to protect the developing brain. We’re particularly excited about our work to test a new device to modulate brain activity and halt seizures in children with epilepsy.”
Mark Proctor, MD Neurosurgeon-in-Chief
• • •
BRAIN SURGERY BEFORE BIRTH
“One project poised to make a big impact is fetal treatment for hydrocephalus. Fluid buildup in the brain can cause permanent damage. With this new technique, we’ll intervene before a baby is born, saving young lives and revolutionizing prenatal care.”
Alireza Shamshirsaz, MD Chief, Division of Maternal-Fetal Medicine and Surgery
TOOLING UP: NEW DEVICES FOR SAFER SURGERY
Compared to adults, children rarely undergo operations, so industry has little incentive to invest in pediatric surgical devices. Boston Children’s innovators are filling the gap to deliver new tools that will improve care for kids everywhere.
A MODEL OF EXCELLENCE
David Hoganson, MD, and a team of engineers use patient-specific imaging data to build custom, multicomponent 3D computer models that physicians can rotate, magnify and pull apart to gain new insights into the patient’s anatomy. This state-of-the-art technology allows surgeons to carefully plan their approach virtually before a procedure, while also guiding them in the operating room. More than 1,000 patients have benefited from this program, the largest and most comprehensive of its kind in the country.
DEEP BRAIN ROBOT
Surgeons are using robotic devices more frequently in the operating room, but, in cardiac and neurosurgery, robots’ capabilities are limited. Engineer Pierre Dupont, PhD, wants to change that. He created a prototype twoarmed, joystick-controlled robot that can help surgeons remove deep-brain tumors faster and with less trauma to healthy tissue.
VIRTUAL TWINS FOR SURGICAL TRAINING
Benjamin Warf, MD, has traveled the world training surgeons to perform the novel hydrocephalus repair he pioneered. Now, he can teach others without leaving home. With a sci-fi-worthy training system developed by EDUCSIM and MIT.nano Immersion Lab, motion-tracking gloves and body markers capture Dr. Warf’s movements as he performs the procedure in Boston. At the same time, a surgical resident anywhere in the world views Dr. Warf’s avatar through virtual reality goggles and then copies the technique on a surgical mannequin.
Technologies like this one will help Boston Children’s surgical leaders share their expertise with colleagues around the globe, improving care for children everywhere.
TRAINING THE NEXT GENERATION OF … INVENTORS?
Heung Bae Kim, MD, knows some of the best ideas come from newly minted surgeons. But developing a device or technology is a complicated process that isn’t covered in medical school. To help trainees take their ideas from the drawing board to the bedside, Dr. Kim operates a Surgical Innovation Fellowship that gives surgeon-inventors access to engineers, prototype specialists and clinical trial experts.
A CUTTING-EDGE SOLUTION—LITERALLY
Surgeons frequently dissect tissue with electrocautery pencils. When blood inevitably pools at the incision site, they switch to a suction tool to remove it.
Alex Yang, MD, MBA, a former surgical innovation fellow, developed a combination tool that makes toggling between those functions quicker and easier. The low-cost device is awaiting approval by the U.S. Food and Drug Administration.
A RESIDENT OPERATES ON A DOLL-LIKE MODEL WITH VIRTUAL INSTRUCTION FROM DR. BENJAMIN WARF.
STICK-TO-IT-IVENESS
Even at rest, heart muscle works twice as hard as leg muscle during a sprint. This level of force, plus moisture from blood, means current surgical glues don’t work for heart repair.
Pedro J. del Nido, MD, partnered with engineers to develop a light-activated, biodegradable glue that is stretchy and strong enough to close a hole in a beating heart. The adhesive has been approved for use in Europe.
PHOTOGRAPH BY Jonathan Kozowyk
Dr. Stuart Orkin: The World’s Most Influential Hematologist Oncologist
Indeed, his impact over five decades is almost too big to measure.
WHEN STUART ORKIN, MD, describes his work, he uses terms like “micrograms” and “kilobase pairs” because what he’s measuring is so small. When everyone else talks about his work, they use words like “global” and “seismic.”
Indeed, his impact over five decades is almost too big to measure. Only recently, Dr. Orkin’s research gave hope to 20 million people worldwide affected by sickle cell disease when the U.S. Food and Drug Administration approved Casgevy, a novel genome editing technology, to treat the condition. Dr. Orkin’s dedication and commitment to share his work with the wider scientific community provided the foundation for that cure.
tion that the cancer drug hydroxyurea prevents sickle cells from forming. But repeated attempts to restart fetal hemoglobin fizzled. Researchers all but gave up and refocused their attention on other medical mysteries.
Serendipity then intervened. Vijay Sankaran, MD, PhD, who was pursuing his dual degrees at the time, arrived in Dr. Orkin’s lab. Encouraged by Dr. Orkin’s mentor, David Nathan, MD, Dr. Sankaran renewed the hunt, this time using dna banked away by the National Institutes of Health.
At the same time, 4,000 miles away in Sardinia, scientists were conducting a genome-wide search for clues to thalassemia. Joel Hirschhorn, MD, PhD, was familiar with their work and connected the teams. That led Drs. Sankaran and Orkin to identify the bcl11a gene as the place to look for the fetal hemoglobin switch.
OPPPOSITE, CLOCKWISE FROM TOP: DR. ORKIN READS THE DNA SEQUENCE OF A GENE (1985); DRS. SANKARAN (L) AND BAUER, ON THE COLLABORATIVE HUNT; THE CANADA GAIRDNER INTERNATIONAL AWARD, ONE OF NUMEROUS DR. ORKIN HAS RECEIVED; DRS. ORKIN AND NATHAN MARVEL AT A PHOTO DURING AN EVENT HONORING DR. ORKIN. ABOVE: COUNTLESS POST-DOCS AND TRAINEES BENEFITED FROM DR. ORKIN’S TRAINING AND COFFEE-FUELED MENTORSHIP.
Dr. Orkin began his foray into sickle cell in 1978, during his fellowship at Boston Children’s and Dana-Farber Cancer Institute. While researching thalassemia, a related blood disorder, he attended “the most exciting” conference. “This was where the first cloning of the human globin gene was described,” says Dr. Orkin, referring to the key regulator of red blood cell development. Conference organizers promoted the theory that synthesizing fetal hemoglobin would deliver a treatment for sickle cell, sparking a career-long mission.
By the early 1980s, Dr. Orkin, working with Haig Kazazian, MD, from Johns Hopkins Medicine, was cloning globin genes from patients with thalassemia and had amassed a catalog of mutations. They hoped to find the switch that told the body to resume making fetal hemoglobin, which doesn't sickle, and stop making adult hemoglobin. “But our research didn’t answer the central question: How do you make a red blood cell,” says Orkin.
That changed in 1989. Dr. Orkin and Leonard Zon, MD, then a fellow, identified the gata-1 gene as the “master regulator” of blood cell development—a discovery that shed light on the genetic principles of fetal hemoglobin production.
Over the next decade and a half, researchers racked up some wins, most notably the realiza-
The search was far from over. The reinvigorated team had to locate the portion of the gene that contained the switch. Dan Bauer, MD, PhD, joined the quest in the Orkin laboratory and, in 2013, suggested Dr. Orkin look in the non-coding portion of bcl11a. That narrowed the search to 10,000 base pairs of dna—still huge ground to cover. Fortunately, their search coincided with the 2015 arrival of crispr gene editing.
Their progress inspired the Doris Duke Foundation to award Drs. Orkin and Bauer critical grants to identify core genetic sequences that upon crispr intervention create healthy red blood cells. They partnered with Massachusetts Institute of Technology’s Feng Zhang, PhD, a crispr inventor, to locate the precise section of dna to edit.
The groundbreaking work by Dr. Orkin and colleagues enabled Vertex and crispr Therapeutics to develop Casgevy. The life-changing gene therapy received fda approval in 2023.
However, the $2.2 million per-treatment price tag and limited availability mean few patients with severe sickle cell disease will get relief soon. To reach more people, there will need to be additional clinics capable of administering the gene therapy, or a drug will need to be delivered in a pill. True to form, Dr. Orkin and team are pursuing both options. “We’re learning as much as we can about bcl11a, how it works, to get clues about the best way to make small molecules and targeted therapies,” says Orkin.
Big Questions
HOW DO YOU TURN $30M INTO $360M?
WHEN IT LAUNCHED 15 years ago, the Venture Philanthropy Network (vpn)—a group of likeminded families who have long supported translational research at Boston Children’s—was, much like the science it championed, something of an experiment. “The idea was to see under the hood of an organization, to really see how good it was. And when people saw the proof of concept, the hope was that they would be excited and give more money,” says Trust Board Member Matthew Sidman, who helped start vpn with his wife, Lori, in 2009. “We also wanted to bring in other couples, so it wasn’t just a charitable experiment but also a social experiment.”
The group coalesced around a then fledgling idea: the Translational Research Program, or trp, a hospital effort to advance some of its most exciting bench science into clinical application.
By every metric, the experiment has been a smashing success.
vpn members have partnered with Boston Children’s and the local business community to raise $30 million for the program since the group’s launch. And trp researchers have used the data and early scientific discovery supported by those donations to draw in an additional $360 million in federal and private foundation grants.
That’s a staggering 12X “return on investment,” Sidman says, but still only accounts for a portion of the TRP’s accomplishments. With support from vpn, the trp has funded more than 60 investigator-career awards. Those recipients have filed patents in nearly 60% of cases and launched 35 clinical trials.
Along the way, trp scientists have dramatically improved outcomes for premature infants, built and sold a vaccine development company in a deal worth $3.3 billion, created cell-on-a-chip technology to better find therapies for rare heart diseases and revolutionized the treatment of anterior cruciate ligament (acl) tears.
More than half of the awardees’ research has resulted in changes in how Boston Children’s—and the world— treats patients.
“What is special about vpn is the community of caring that it has built, and the continued success of vpn stems from the strength of that community and the incredible work being done by the researchers at the hospital,” says Trust Board Member Ted Pappendick, who serves with his wife, Erica, as one of the co-chair families of the group. They also established the Pappendick Family Therapeutic Acceleration Award to support trp science.
With new members joining the group in recent years, Pappendick says vpn’s future is bright, and it remains a unique opportunity for a group of donors to watch their philanthropic impact multiply.
OPPOSITE, CLOCKWISE FROM TOP LEFT: A NOVEL HEART-ON-A-CHIP TECHNOLOGY ALLOWS FOR RAPID LABORATORY TESTING OF THERAPIES TO FIGHT RARE HEART DISEASE; A STARTUP COMPANY THAT WILL PRODUCE THE COLLAGEN SCAFFOLD AT THE CENTER OF A REVOLUTIONARY APPROACH TO TREATING ACL TEARS HAS RECEIVED FDA APPROVAL AND SERIES A FUNDING; OMEGAVEN, APPROVED BY THE FDA IN 2018 TO TREAT BABIES WITH FATAL LIVER DISEASE, HAS SAVED THOUSANDS OF INFANTS’ LIVES; GSK PURCHASED AFFINIVAX, A VACCINE PIPELINE DEVELOPMENT COMPANY, FOR $3.3 BILLION IN TOTAL COMPENSATION (BOTTOM LEFT AND RIGHT).
New Funding
THE PRINCIPAL INVESTIGATOR
Wendy Chung, MD, PhD Chief, Department of Pediatrics
THE SCIENTIFIC QUESTION
ACCELERATING AUTISM RESEARCH
How can we better understand autism’s vast spectrum of severity, symptoms and comorbidities to improve children’s lives?
THE FUNDING
A longtime supporter of Dr. Chung’s work, the Simons Foundation was very happy to continue its partnership with her when she joined Boston Children’s in 2023. Two Simons Foundation grants enable Dr. Chung to continue leading two transformative research projects: SPARK (Simons Powering Autism Research for Knowledge) and Simons Searchlight.
WHY IT’S IMPORTANT
Autism affects 1 in 36 children in the United States. The causes of autism and the biological pathways leading to its disparate symptoms are not completely understood. While research has led to many advancements, significant gaps remain in knowledge and in the availability of effective treatments. To address these gaps, SPARK and Simons Searchlight are collecting robust data from diverse people with autism and their families—and making that data freely accessible to scientists everywhere to accelerate breakthroughs.
WHAT IT SUPPORTS
The longitudinal SPARK study is helping researchers understand the complexity of autism by recruiting and engaging a large cohort of U.S. participants (currently almost 330,000 individuals, including 139,000 with autism) and compiling both quantitative and qualitative data, such as genetic information and personal observations. Simons Searchlight is an international project investigating the hundreds of gene variants implicated in autism. Importantly, recruitment of Black, Latino and other underrepresented populations is a central aim of both studies.
CAROL KAPLAN
ENGINEERING AN END TO JOINT PAIN
Boston
Can researchers grow “off-the-shelf” tissue to heal arthritis?
THE FUNDING
Turning an exciting discovery in the lab into a viable therapy can be costly and time consuming.
The Blavatnik Therapeutics Challenge Awards at Harvard Medical School seek to advance promising therapeutics by supporting the commercial success of early-stage discoveries and propelling them closer to the clinic.
WHY IT’S IMPORTANT
Osteoarthritis (OA), the most common form of arthritis, is a painful reality for millions of people—one for which there is currently no cure. The protective cartilage that cushions every joint breaks down with age and doesn’t heal when injured, making OA a leading cause of pain and disability in the United States. Being able to replace damaged cartilage with healthy tissue would significantly improve people’s quality of life and mobility.
WHAT IT SUPPORTS
Using cartilage engineered from stem cells, Dr. Craft’s team already has successfully repaired damaged tissues in rat models. The Blavatnik award will allow them to test their groundbreaking approach in a large animal study, the next phase in advancing the research toward eventual human trials.
“By funding the translation of our work into a pre-clinical model, this award is a crucial bridge between our discovery and the clinic, where it could potentially improve the lives of countless patients,” says Dr. Craft. Long term, this therapy could become broadly accessible and easily produced, stored and delivered to surgical teams as needed.
THE PRINCIPAL INVESTIGATOR THE SCIENTIFIC QUESTION
April Craft, PhD
Children’s Orthopedic Center and Harvard Stem Cell Institute
MAKING A WORLD OF DIFFERENCE
INSPIRED BY THEIR DAUGHTER’S PASSION TO IMPROVE CHILDREN’S LIVES AROUND THE GLOBE, ANN AND TONY RYAN’S TRANSFORMATIONAL GIFT IS HELPING TO CREATE HEALTHIER TOMORROWS FOR MILLIONS OF FAMILIES THROUGH THE MARGARET C. RYAN GLOBAL HEALTH PROGRAM.
WHEN SHE MET intern Maggie Ryan 10 years ago, Michelle Niescierenko, MD, MPH, spotted it at once. “There was a depth of caring unusual in someone so young,” says the Grousbeck-Fazzalari Chair in Global Health. A kinship ensued as the two shared a commitment to finding solutions to keep children from dying of preventable conditions.
But as Maggie was about to launch her career in global health, a car accident cut her life short. Her parents, Tony and Ann, searched for a way to honor their daughter.
“Maggie talked so enthusiastically about the dynamic Boston Children’s doctor who inspired her,” says Ann.
After meeting Dr. Niescierenko, the Ryans understood the tremendous impact philanthropy could have on children worldwide. They established the Maggie Ryan Endowed Fellowship in Global Health in 2017. Their gift enabled Ryan Fellow Lexie Schmid, DNP, to complete dozens of deployments, delivering training that has reached thousands of health care workers.
As the program grew, the family’s connection with Drs. Niescierenko and Schmid deepened. In 2023, Ann and Tony recognized the program had arrived at an inflection point—it was time to scale. They made a transformational gift, naming the Margaret C. Ryan Global Health Program and creating the Susanna Ryan Endowed Fellowship in Global Health, in honor of Maggie’s grandmother in 2024.
A SELFLESS LEADER, MAGGIE WANTED TO DEDICATE HER LIFE TO THE SERVICE OF OTHERS.
ANNIE SPRATT (TOP); COURTESY OF THE RYAN FAMILY
“There was a depth of caring unusual in someone so young.”
“Our goal is to drive collaborative, sustainable and strategic growth, so children everywhere can access the care they need,” explains Tony.
Different Countries, Similar Challenges
In the United States, clinics serving indigenous communities often don’t have pediatricians or pediatric nurses. Kids miss wellness checks and vaccines. Conditions like asthma or diabetes go undetected. The consequences can be life-threatening.
The federal government asked Boston Children’s for help. At South Dakota’s Cheyenne River Health Center, our pediatricians and nurses are caring for children and training staff to treat emergencies, support newborns and manage kids’ health needs. The Global Health Program is helping to build the local workforce with the region’s first pediatric residency program. Dr. Niescierenko and colleagues
drew from global experiences to develop this model.
In 2017, Liberia had two pediatricians. Boston Children’s and the Liberian Ministry of Health launched a residency program. Today, the country has more than 30 pediatricians, with training led by local faculty. Equally important, caregivers have learned to treat childhood diseases, including malaria, typhoid fever and more.
In Bihar, India, most hospitals weren’t equipped to care for children with traumatic injuries. Local leaders wanted change. The Global Health Program and a humanitarian partner mentored doctors and nurses, who then trained their colleagues to respond to emergencies. The interventions worked. Children are being treated successfully, and Bihar is rolling out the improvements at 37 emergency departments serving 130 million people.
In addition to meeting local needs, our experts respond to humanitarian crises. They’ve delivered pediatric trauma training for 8,000 health care workers in Ukraine, mental health services for thousands of Syrian children and early childhood nutrition programs for millions in Africa.
“The Ryans’ extraordinary generosity enables us to substantially expand our efforts to raise the level of health care for every child, no matter where they live. We have much work to do, and with this gift, we’re positioned to make a sustained impact: locally, nationally and globally,” says President and ceo Kevin B. Churchwell, MD.
Dr. Maria GutierrezArcelus: Diseases,Decoding One Cell at a Time
AS A CHILD, Maria Gutierrez-Arcelus, PhD, traveled with her family from their home in Mexico to Boston Children’s, where her little brother was diagnosed with a brain tumor. A clinical trial saved his life—and opened his sister’s eyes to the transformational power of research.
“A few years later, in high school, I learned that by studying dna you could understand how diseases develop and that by editing genes you could cure diseases,” says Dr. Gutierrez-Arcelus. “That motivated me to go into genomics.”
She returned to Boston Children’s in 2021 as principal investigator of her own laboratory. There, scientists use advanced genetic sequencing and other state-of-the-art techniques to study gene regulation in autoimmune disorders like lupus.
In every patient with lupus, the body’s immune system attacks healthy tissue, yet people’s symptoms vary widely. This heterogeneity makes the disease hard to treat—especially childhood-onset lupus. Now, with funding from the Lupus Research Alliance, Dr. Gutierrez-Arcelus is investigating the biological processes that lead to this variability.
“We know certain B cells play an important role in the development of lupus. Our goal is to analyze how they respond to stimuli, how the responses vary between individuals and how genetic susceptibility to lupus affects B cell responses. These
learnings could help us design personalized therapies,” says Dr. Gutierrez-Arcelus.
Another project on the horizon will study the pathogenesis of lupus in Latino and Black people.
“Lupus is more common in these populations compared to white people but, because they’re understudied, it’s unknown whether this is because of genetics, environmental factors or both,” says Dr. Gutierrez-Arcelus. “In collaboration with colleagues in Mexico and Houston, we’ll analyze samples from these patients, sequence their genomes and examine the external triggers that might influence their higher incidence of lupus.”
Central to this project will be the use of singlecell sequencing, a new technology that allows researchers to analyze one cell at a time—a crucial step in identifying the subsets of cells involved in disease development. The revolutionary tool is available throughout Boston Children’s thanks to funding from the Manton Foundation for the Manton Cell Discovery Network (cdn). Dr. Gutierrez-Arcelus sits on its steering committee.
“With this technology, we can pinpoint the genetic factors that impact biological function—not just in lupus but in numerous conditions. My role in the cdn is to educate Boston Children’s investigators about these capabilities that will help us find answers for our patients.”
DR. GUTIERREZARCELUS VISITED BOSTON CHILDREN’S AS A CHILD WHEN HER BROTHER WAS DIAGNOSED WITH A BRAIN TUMOR. NOW, SHE’S HERE LEADING A PIONEERING LAB.
THE RIPPLE EFFECT
How schools can help solve the behavioral health crisis
WITH FAMILIES FACING months- to year-long wait lists for behavioral health appointments, it’s easy to feel discouraged about the nation’s chances of stemming the crisis. However, there is cause for hope. We have a model for how to reach most kids now.
“Schools are uniquely positioned to support and enhance students’ social and emotional lives and behavioral health,” says Shella Dennery, PhD, director of Boston Children’s Hospital Neighborhood Partnerships (bchnp) Youth spend most of their time each week in school, making it an ideal place to address their needs. A national leader in school behavioral health, bchnp aims to uplift students and teachers by providing services, consultation, training and support. bchnp staff and educators work together to nurture students’ sense of belonging. The goals? Create an open climate. Catch issues before they snowball. Promote a culture of prevention.
Since starting in 2002, bchnp has partnered with 40 Boston Public Schools. Over the past decade, the program has helped 15,000 students, and its impact continues to grow.
Supporting Teachers, Supporting Kids
More than 1 in 3 teachers leave the field because of classroom management challenges and concerns about students’ behavioral health. bchnp enhances teachers’ well-being by growing their knowledge and confidence to understand and address behavioral and emotional health. bchnp team members are embedded within schools and meet
regularly with educators to discuss particular students and plan group or classroom interventions. They lead peer-mentoring programs for refugee and immigrant youth, create lessons on substance use and screen time, and facilitate referrals for more intensive therapy and other services.
bchnp offers free programs to help school staff expand their knowledge and skills through the Clough Foundation Training and Access Project (tap). Since its inception, tap has reached more than 11,000 school professionals across the United States through in-person training and online courses. Attendees learn how to help students experiencing grief and loss, partner with families and support kids after a behavioral health crisis. tap plans to create a video supplement for educators nationwide, amplifying the program’s impact.
“What’s so powerful about bchnp is how it creates a culture of mental wellness within schools by focusing on prevention and early identification,” says Boston Children’s Psychiatrist-in-Chief Stacy Drury, MD, PhD. “As this culture becomes embedded in schools, it spreads to the community. This creates a protective effect for more kids than we could otherwise reach.”
To further advance change, the bchnp team is leading efforts to expand state support for schoolwide behavioral health approaches. “We’re partnering with Boston Children’s Office of Government Relations to ensure that there are enough social workers and counselors in every school,” says Dr. Dennery.
“The more we bolster behavioral health resources in schools, the better chances we have of slowing the crisis,” says Dr. Drury.
“WHAT’S SO POWERFUL ABOUT BCHNP IS HOW IT CREATES A CULTURE OF MENTAL WELLNESS WITHIN SCHOOLS BY FOCUSING ON PREVENTION AND EARLY IDENTIFICATION.”
WHAT DOES COMPREHENSIVE SCHOOL-BASED BEHAVIORAL HEALTH LOOK LIKE?
3 Group counseling focused on peer and interpersonal relationships
3 Individual counseling for students facing challenges at home, in school and in the community
3 Crisis management services that connect students and their families to school and community resources
3 Professional development and consultation services focused on building the capacity of school communities to address social, emotional and behavioral health
Big Questions
CAN AN IMMEDIATE IMPACT LAST FOREVER?
MARY SHEA AND EVE ENDICOTT have never met, but they have much in common. Both spent most of their careers helping others, Mary as a nurse and Eve at conservation nonprofits. They both have a connection to Boston Children’s that stretches back decades. And both have a plan to ensure the hospital can continue providing lifesaving care long into the future.
UNFORGETTABLE EXPERIENCES In 1976, Mary graduated from Children’s Hospital Boston School of Nursing (1889-1978) and later worked here for several years, first as a registered nurse and then a pediatric nurse practitioner. Even after her nursing career led her elsewhere, the hospital’s ongoing commitment to educating nurses throughout their careers—a clear signal of how highly it values the profession—left a lasting impression on her. With multiple advanced degrees herself, Mary remains convinced that prioritizing a learning culture among nurses today is vital for tomorrow’s medical breakthroughs.
Eve also was moved by an experience more than 20 years ago, when her young son was rushed to Boston Children’s by ambulance with a life-threatening condition. Eve still remembers the surgeon who treated her sick child and the relief she felt seeing her wheel him into surgery, knowing her son was in exceptional hands. Today, that little boy works in conservation himself and climbs the world’s highest mountains in his free time.
CREATING A LEGACY
Both women knew they wanted to make an enduring impact on Boston Children’s because of the difference it made in their own lives. They sought to honor their past experiences through meaningful gifts that could meet their philanthropic and financial goals and benefit the hospital for years to come.
Mary dedicated a current-use fund to the Nursing Career Lattice Program, which helps hospital employees from diverse backgrounds obtain a bachelor’s degree in nursing. She also named the hospital as a beneficiary in her estate plan, creating an endowed fund to support nursing education in perpetuity.
“I never thought I’d be able to have this kind of impact while I’m still alive,” says Mary. “I’m getting to see the effects of my giving now and creating something that will last forever.”
Eve also found a way to achieve her present and future goals, by establishing three charitable gift annuities supporting the Every Child Fund, global health initiatives and free care. The annuities give her a reliable, fixed retirement income while furthering the hospital’s long-term mission to ensure all families, even those without insurance, can receive high-quality care.
“I will never forget the doctor who saved my son’s life,” says Eve. “She told me she would take care of him, and I knew she would. Now, I’m giving back so that other parents can have the same peace of mind.”
OPPOSITE: STRONG NURSING MAKES ALL THE DIFFERENCE FOR PATIENTS LIKE SAMUEL.
MICHAEL GODERRE
CHAMPIONS FOR CHILDREN’S CELEBRATES 20 YEARS
WHAT DOES IT TAKE to bring together Boston’s biggest names in sports, business and medicine for one night every year? It’s a layup when the evening benefits sick kids through the Champions for Children’s fundraiser. Founding chair and current Trust Board chair Rob Griffin has partnered with a dedicated committee and scores of sponsors to grow the initiative into a star-studded affair that has raised over $80 million to help the hospital serve families around the globe.
Since becoming an annual event in 2004, Champions for Children’s has elicited unwavering support from all local professional sports teams and become one of the most successful fundraisers in the city’s history. Over the years, attendees have arrived to discover everything from duck boats recreating a victory parade to appearances by the Dropkick Murphys and Jay Leno.
But it’s not just Boston’s A-list who takes home the “W.” The evening’s true champions— our patients—sashay into the event ready to create peak-childhood memories. The heart of Champions for Children’s is a tradition rooted in connection as athletes and kids get to know each other before the show. The sports-tacular occasion carries on with dinner, a spirited live auction and the annual “Champion Award” to recognize all-stars who enrich the lives of children. Past recipients have included Patrice Bergeron, Devin McCourty, Tim Wakefield and Paul Pierce. Newly formed bonds continue long after the lights dim.
“Champions for Children’s energizes many of the more than 1,000 guests to get involved with Boston Children’s in other ways, too,” says Griffin. “This event instills a commitment that extends far beyond the night.”
Looking ahead, longtime Champions ambassador, title sponsor and five-time chair John Fish is confident the event’s legacy will live on: “Champions for Children’s will attract the next generation of leaders to carry forward its extraordinary mission. As we celebrate this milestone, we’re not just commemorating the past—we’re laying the groundwork for a future where every child has the chance to thrive.”
(L-R) WBZ ANCHOR AND CHAMPIONS EMCEE DAVID WADE, ROB GRIFFIN, DR. KEVIN CHURCHWELL, JOHN FISH SPORTING HIS PRIZED CHAMPIONS JACKET, WBZ ANCHOR AND CHAMPIONS EMCEE LISA HUGHES AND BOSTON CHILDREN’S PATIENT CLAIRE. BELOW: NEW ENGLAND PATRIOTS STAR DAVID ANDREWS CONNECTS WITH KIDS. 2023 CHAMPION AWARDEE CODY DAVIS WOWS A FAN.
LEARN MORE ABOUT CHAMPIONS FOR CHILDREN’S
JOINING FORCES
EVERY FALL, Britt Melsheimer’s family and friends lace up their high tops to participate in the Hoops for a Heart basketball tournament. The inspiration for the Westport, Connecticut, fundraiser is her son Evan, who was born with a single ventricle heart defect. Eager to help her baby and all families affected by the rare condition, Britt sought answers from the expert who saved her son’s life.
Rahul Rathod, MD, created the FORCE Registry in 2020, which harnesses data from hospitals worldwide and uses artificial intelligence to predict a child’s need for intervention before a life-threatening event. Britt is fiercely committed to Dr. Rathod’s vision, and the money she raises through the Fundraise Your Way program has helped give the international consortium a solid foundation to expand, strengthen and support families around the globe. FORCE continues to grow, with 36 participating institutions and over 3,700 patients enrolled. “When I see Dr. Rathod, he’s so excited to tell me how much the registry has grown and reminds me that my belief in him was crucial to how far we have come today,” says Britt.
Events
CREATE YOUR OWN FUNDRAISER
DAN DELIVERS HIGH HOPES
WHEN Olivia arrived three months early, Dan Silvia (now Uncle Dan) was already a longtime supporter of Boston Children’s. For eight years, Dan has participated in Extra Life, which unites gamers around the world to fundraise for their local Children’s Miracle Network (CMN) hospital. His niece’s birth encouraged Dan to elevate his efforts.
Like Dan, “Olivia needed help getting started in life” and received care at Boston Children’s. Dan remembers his mom’s stories of feeling like a patient herself as nurses tended so thoughtfully to their family when he needed surgery as a baby in 1981. Now years later, Dan noticed, “Staff decorated Olivia’s room with a collage of her milestones—from her 1-month birthday to her first procedure and her first burp.”
These unique connections warranted an equally unique celebration, thought Dan, and Extra Life, which combines gaming, creativity and philanthropy, was the perfect fit. So, Dan established The High-Altitude Dice Roll. The fundraiser films a 20-sided die being lifted into near-space via weather balloon to encourage donations based on the number rolled at landing. But it’s not only the stakes that are high! Dan also includes a USB containing pre-recorded messages of hope to be carried into the stratosphere and displayed on screens throughout Boston Children’s.
The campaign excited new audiences about all the ways to give—a perfect fit for Extra Life, which has brought together gamers from all walks of life to help kids since 2008.
LEARN MORE ABOUT EXTRA LIFE
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MAKE AN INFINITE IMPACT
Join our CEO, Kevin B. Churchwell, MD, and a select group of caring philanthropists to take on the toughest health challenges facing children around the world. Become a founding member of the Boston Children’s CEO’s Circle of Impact today and give lifesaving answers to kids for generations to come. Learn more at bostonchildrens.org/ceoscircle
