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Promising treatments for hearing loss are on the horizon.
Miniature
Functional 3D replicas provide powerful windows into the body’s inner
DEPARTMENTS
Profiles
8 Dr. Beth Stevens 16 Dr. John Kheir • Big Questions
12 How Can Parents Encourage Healthy Gaming? 24 Can Investing in Mental Health Help Curb School Shootings?
Accelerating Vision Research
Philanthropy Propels Young Athletes
First Person
“Our Greatest Impact”
EARLY IN MY CAREER at Boston Children’s, I witnessed a miracle: stem cells grown in a dish. In theory, those immature cells could be coaxed to form any tissue in the body, offering a powerful new tool for studying development and disease. What was theoretical then is reality today. As you will read in this issue, researchers are growing stem cells into organoids—mini-models of brain, lung, bone marrow, and more. These organoids are speeding discovery of safer, more effective treatments for everything from pediatric brain cancer to chronic diarrhea to genetic hearing loss. The impact is astonishing.
Impact is a word that readily springs to mind when thinking about Boston Children’s. For me, as for many of you, that impact has been personal and profound.
My dad was born with a heart defect that couldn’t be diagnosed let alone cured when he was young. It wasn’t discovered until he failed his World War II physical. People with his condition rarely lived beyond their 20s, but my dad reached 92. One of the hospital’s great heart surgeons, Robert Gross, MD, perfected a technique to correct the defect. He accepted my father as an adult patient and saved his life.
Boston Children’s became my parents’ charity of choice. It’s been mine and my husband’s, too. Most recently, we made a planned gift that provides us with income in retirement, allowing us to have our greatest impact. We are forever thankful for the dedication, determination, and sheer talent of the researchers and clinicians who keep families like mine whole.
With gratitude,
LAURIE BECKELMAN RETIRED COMMUNICATIONS DIRECTOR
Socially Speaking
FOLLOW Lori Tolentino
It’s amazing to be SEEN with Stephen, Dr. Scoobily our first African American male Healthcare clown at Boston Children’s Hospital. I have the goosebumps from all of the love and silliness and whacky music tunes we shared and received. Dr. Scoobily you have this magical soul that these children and families appreciate. When we get to interact and connect with families that look like us and they see us, it’s something I can’t really put into words but WOW do I feel it. Laughter League has been doing an amazing job making sure our teams represent who we are and it’s an honor to be part of this unique journey.
JOYFUL
Celebrate life’s special moments with our families, caregivers, and followers—even when they’re superstars themselves.
Zachary can continue being a silly and outgoing 7-year-old, thanks to the generous blood donations.
Enjoying the rooftop garden
Nora is not a fan of getting pokes in the hospital! She is, however, a fan of playing with doctor toys. Nora’s child life specialists have been capitalizing on her love of playing doctor and utilizing medical play to help her prepare for procedures in the hospital.
If you see Dr. Nora around Boston Children’s Hospital, make sure to ask her for a checkup!
We’ve been spending this week at Boston Children’s Hospital as Josephine is treated for bronchiolitis/ pneumonia and this has truly been the most humbling experience of my life. We’ve been staying in the ER for a few nights and then in the neurology unit because it was the only room available in the entire hospital. Ex- periencing the hallways, the sights, and the sounds is something that will be engrained in us forever. This is the most extraordinary place on earth for any child to come to and we are so blessed to have it in our back- yard. But no kid ever deserves to be here.
Life really slowed down for me this week, showed me that every moment spent with family is precious, and needs to be cherished.
Brad Zapenas
The Gift of
BROTHERS EMILIANO (LEFT) AND JP DELIGHT IN NEWFOUND MOBILITY AS GRATEFUL FAMILIES POWER RESEARCH THAT CHANGES LIVES
Movement
“Shore, look what I can do!”
With that, Emiliano takes off across the room, showing his doctor, Benjamin Shore, MD, MPH, the progress he’s made since his last surgery.
When Emiliano first came to Boston Children’s in 2017 from his home in Mexico, his cerebral palsy (CP) had caused the muscles and tissues around his knees to tighten so severely that he couldn’t straighten his legs—a common challenge among patients with CP. He and his family had a simple goal: to help Emiliano stand.
Dr. Shore proposed a novel technique: placing screws asymmetrically across the knees’ growth plates to guide the bone’s trajectory. Three years later, Emiliano’s knees straightened, and he could stand. With that breakthrough, Dr. Shore then prescribed additional interventions to realign his feet and legs, providing greater stability and mobility.
Today, Emiliano loves demonstrating the things he can do, including walking, biking, and playing sports. To celebrate his progress, as well as that of his younger brother JP, who also has CP, their parents Mariana Canales and Luis Lugo-Platt established the Lugo Canales Family Fund. Their support allows Dr. Shore and his team to expand their innovative approach to guided growth of the bone from the knee to the hip joint—an early intervention that may prevent hip dislocation and the need for more complex surgery in the future. The family hopes to give other kids with neuromuscular diagnoses like CP the chance for similar success.
Different Goals, Same Commitment
Across Boston Children’s Orthopedic Center, researchers are pushing boundaries to ensure kids with a variety of conditions can live their best lives. Grateful families are often the engines behind the scenes, ensuring this work can benefit countless children.
“Orthopedic problems are the most common problems patients face in their lifetime, yet less than 2% of federal funding for medical research goes to orthopedics,” says Orthopedic Surgeon-inChief Martha Murray, MD. “Philanthropy bridges the gap to bring new technologies and treatments to the clinic.”
It also advances research that directly impacts care. When their daughter tore her anterior cruciate ligament (ACL), Ian and Isabelle Loring were in-
trigued by research led by Mininder Kocher, MD, MPH, chief of Boston Children’s Sports Medicine Division, comparing four common surgical options for pediatric ACL tears.
For the past 15 years, the Lorings have supported this groundbreaking ACL study, which analyzes data to improve outcomes for young athletes who injure their knees. The study already has yielded important insights that are shaping surgeons’ understanding of knee health, including post-surgery growth, injury prevention in both knees, and the psychological components of injury and recovery.
This patient-centered, comprehensive approach impressed Andrea and Ted Killory, whose children, Aidan and Caroline, are active teenagers who find identity and community through sports. Boston Children’s experts have helped both kids heal from various injuries and conditions.
In 2023, Andrea Bauer, MD, a hand specialist, surgically corrected a congenital thumb difference in Aidan’s right hand to give him improved joint stability. And Cynthia Stein, MD, MPH, a sports medicine doctor, continues to care for Caroline as she recovers from a competitive rowing injury. Last year, the Killorys established the Ted and Andrea Killory Orthopedics Research Fund, which supports investigations led by Drs. Bauer and Stein.
Their gift enables research into questions that otherwise would be difficult to fund, including which physical therapy approaches are most effective after certain surgeries, how to increase access to sports medicine services for all kids, and what methods will improve medical education for future providers.
“We’ve had a wonderful experience at Boston Children’s,” says Ted. “Now, we hope to accelerate research so others can receive the same benefits.”
“In orthopedics, our goal is both to improve patients’ health and how well they function in their environment,” says Dr. Shore. “With the resources these gifts provide, we can teach the world what we learn. It amplifies these positive outcomes many times over.”
DR. KOCHER REVIEWS A YOUNG PATIENT’S MRI SCAN.
New Funding
THE PRINCIPAL INVESTIGATORS
David Hunter, MD, PhD Ophthalmologist-in-Chief
Lois E.H. Smith, MD, PhD Professor of Ophthalmology
THE SCIENTIFIC QUESTION
How can scientists improve care for children with vision-threatening conditions?
THE FUNDING
For more than two decades, the Knights Templar Eye Foundation, Inc. (KTEF) has supported Boston Children’s researchers working to treat, cure, and prevent vision loss. Now, through a gift matched by the Children’s Hospital Ophthalmology Foundation, the organization has established the KTEF Research Endowment.
WHY IT’S IMPORTANT
Visual impairment in children can lead to developmental delays, learning disabilities, depression, and other negative consequences. Without treatment, these young people may require lifelong assistance. As leaders in pediatric ophthalmic disease research, Boston Children’s investigators have the potential to improve outcomes and enhance the quality of life for all individuals affected by vision-threatening conditions. “This endowment ensures that our experts can continue conducting basic science and translational research to advance our understanding of sight-threatening conditions,” says Dr. Hunter.
WHAT IT SUPPORTS
The KTEF Research Endowment will be overseen initially by Drs. Hunter and Smith. Scientists selected for endowment support will conduct research to advance our understanding of numerous disorders, including strabismus, or crossed eyes; retinopathy of prematurity, a disease that can blind preterm infants; amblyopia, or “lazy eye”; and nystagmus, a condition with uncontrollable, rhythmic eye movements. They also will explore the use of artificial intelligence to refine ophthalmic imaging, use data science to build a database of pediatric eye disease, and work to develop novel therapies to correct or prevent pediatric forms of blindness.
HAVING PEOPLE THAT BELIEVE IN YOU AND GIVE YOU OPPORTUNITIES—I CAN’T SAY HOW IMPORTANT THAT’S BEEN FOR MY CAREER.
Dr. Beth Stevens: Protecting Brain Health
NOT MANY PEOPLE can say they’ve been so engrossed in a conversation about glial cells that they almost missed a flight—but there aren’t many people like Beth Stevens, PhD. At the time, she was interviewing for a postdoctoral position in the lab of the late neuroscientist Ben Barres, MD, PhD, a pioneer in understanding these cells that support neurons and make up more than half the brain.
“Ben said I could spend my career studying glia and there would always be more to learn. That’s what gets any scientist excited—the idea that you’re never going to run out of cool questions to ask,” says Dr. Stevens.
Since starting her own lab at Boston Children’s in 2008, Dr. Stevens has asked dozens of these questions—and come up with game-changing answers. Her team helped demonstrate that the immune cells of the brain—a type of glia called microglia—prune extra synapses during development. Then, they discovered that a pathogen-fighting protein known as C1q acts as an “eat me” signal in the healthy brain, tagging synapses for the microglia to eliminate.
These findings prompted Dr. Stevens to ask her next compelling question: Could this process that helps wire the brain normally during development become dysregulated and lead to the loss of synapses and to cognitive dysfunction in neurological diseases?
Now, Dr. Stevens is investigating the roles of
microglia, C1q, and other “eat me” signals in conditions that manifest throughout the lifespan, from autism to neuropsychiatric disorders to Alzheimer’s disease. Her lab recently showed that this developmental immune-pruning mechanism is turned back on in early-stage Alzheimer’s and Huntington’s disease and that blocking C1q lessens synapse loss and cognitive defects in preclinical disease models.
“I think our work will catalyze the discovery of therapeutics as well as biomarkers that clinicians can use to predict who is going to develop neuroimmune-related synapse loss across disease progression,” says Dr. Stevens. “This would be relevant not just to one disorder but many.”
Early on, Dr. Stevens made a bet that studying development and neurodegeneration in parallel would further our understanding of both—and philanthropy enabled her to pursue this visionary path. “You can’t get large grant funding for risky projects. That was true when I launched my lab and is still true today.”
Nevertheless, Dr. Stevens encourages her trainees to take risks and follow the unexpected. Being a good mentor, she says, is as important as being a good scientist.
“I feel a huge responsibility to support the next generation of investigators and engage young scientists from diverse backgrounds. Having people that believe in you and give you opportunities—I can’t say how important that’s been for my career.”
THE POWER OF PHILANTHROPY WE ARE GRATEFUL TO THE SUPPORTERS THAT FUEL DR. STEVENS’ WORK: ALZHEIMER’S ASSOCIATION, CHAN ZUCKERBERG INITIATIVE, CURE ALZHEIMER’S FUND, DAUTEN FAMILY FOUNDATION, JONATHAN AND JEANNIE LAVINE, SIMONS FOUNDATION.
ADAPTIVE SPORTS
PHILANTHROPY PROPELS YOUNG ATHLETES
Ian, 9, grins from ear to ear as he sprints down the TRACK at new balance. He’s reaping both physical and social benefits from his exuberant participation in Adaptive Track and Field—one of more than 15 activities offered through the New Balance Foundation Adaptive Sports Program at Franciscan Children’s
The program gives kids with disabilities—such as autism, developmental delays, and other differences—the opportunity to take part in sports while learning new skills, building bonds, and boosting physical activity.
The impacts are obvious and important. Kids are stronger and faster. They’ve built friendships. Their confidence has bloomed.
Adaptive Sports Program participation has skyrocketed from 68 children in 2021 to 450 as of September 2024. Keeping up with demand can pose a challenge, says Program Director Paul Ward, but a generous gift from the DICK’s Sporting Goods Foundation means that the program—like its young participants—can continue to grow and thrive.
“We know the benefits of sport—confidence, teamwork, resilience, and a sense of belonging (to name a few)—can transform lives and build stronger communities,” says Rick Jordan, vice president, at The DICK’S Foundation
A NEW TEAM FOR KIDS
Boston Children’s and Franciscan Children’s joined forces in 2023 with a shared goal of building healthy bodies and strong minds. Together, the partners aim to reshape pediatric mental health and rehabilitative care in Massachusetts and beyond.
Big Questions
HOW CAN PARENTS ENCOURAGE HEALTHY GAMING?
A SEDENTARY TEEN IN THE basement, mashing a controller for hours, neglecting schoolwork and personal hygiene, spraying bullets across a screen. A typical gamer, right? Not so fast.
Parents of avid gamers can somewhat relax. Doomsday views are unwarranted, including the myth that video games make individuals violent, says Gino Mortillaro, MD, medical director of Boston Children’s Emergency Psychiatric Service
Parents should take heart in gaming’s positive aspects, says Dr. Mortillaro. Strong social benefits top these, with esports helping many kids make new friends and connect over popular culture. Play also fosters problem-solving skills, teamwork, and creativity. And studies have shown improved hand-eye coordination and cognitive benefits. All that said, as with any aspect of parenting, there are no shortcuts when it comes to nurturing healthy habits. Dr. Mortillaro shares the following tips.
3 RESEARCH GAMES FOR PROBLEMATIC ELEMENTS. “Reviews on Common Sense Media and the Entertainment Software Ratings Board websites will help you better understand a game’s age rating and content.”
3 UNDERSTAND ONLINE INTERACTIONS. Educate yourself and your child on how to restrict in-game communication to known friends and family, as well as how to avoid sharing personal information with strangers.
3 SET CLEAR BOUNDARIES. Share your expectations for how your child must balance playing time with responsibilities such as homework, family time, and chores. Use parental controls to enforce these.
3 WATCH OUT FOR IN-GAME PURCHASES. “Research has shown in-game purchases can fuel addictive behavior, much like gambling targeted to children. In extreme cases, children have stolen credit cards to run up charges.”
3 ASK FOR HELP. If your child develops signs of problematic gaming—difficulties with sleep, school, relationships, or other aspects of their overall well-being—reach out to their primary care provider or to Boston Children’s Clinic for Interactive Media and Internet Disorders.
3 PLAY! “Gaming with your child is great for your relationship. It‘s also the single best way to understand each game’s content and how to set guardrails for your child.”
Dr. Mortillaro encourages parents to seize gaming’s myriad opportunities to bond with their child, on screen and off. Families can attend gaming conventions, watch game-based movies, create game-inspired art or music, and make up backyard games together.
It’s a recommendation that comes from experience. As a child, the doctor made up songs to the tune of the Super Mario theme song while playing with cousins. Later, weeknight gaming “dates“ during his college years helped Dr. Mortillaro bolster a long-distance relationship with his now-husband.
But his most treasured gaming memory is creating a 3-D Pokemon village as a teen with his mother. “She actually got a bunch of construction paper, markers, and cardboard so the drawing could stand upright, and then put it in an armoire alongside her good china. I felt I had created something really valuable.”
“I wanted to make the day brighter for other families in an emergency.”
INSPIRED BY HER GRANDSON’S CARE, MAUREEN CASELLA JOINED BOSTON CHILDREN’S CEO’S CIRCLE OF IMPACT, A GROUP OF PHILANTHROPISTS TAKING ON THE TOUGHEST CHALLENGES IN CHILD HEALTH AND HELPING FAMILIES EVERYWHERE, INCLUDING DAWSON’S (PICTURED HERE).
Beth Ann Fricker
Profiles
Dr. John Kheir: Preventing Fatal Cardiac Arrest
WHEN A PATIENT goes into cardiac arrest, tragedy—brain damage or even death—can ensue. For years, cardiac intensivist John Kheir, MD, has sought to outsmart the biochemical processes that lead to these outcomes.
Oxygen is the central player in cardiac arrest. When our bodies are deprived of it, the heart stops working. Breathing ceases.
About 15 years ago, Dr. Kheir came up with an innovative approach to cardiac arrest: temporarily halt the catastrophic cascade by injecting an oxygen nanoparticle solution directly into the bloodstreams of critically ill patients. It’s a brilliant idea, but, until recently, it proved to be exceptionally difficult to implement.
Last year, Dr. Kheir and his team scored a win and demonstrated that injectable oxygen saved large animals from severe brain damage and death during cardiac arrest.
But during the interim, he had pivoted and devised another bold solution, aiming to safeguard the brain during longer low-oxygen environments, like cardiac surgery or when transitioning a patient to ECMO following cardiac arrest.
During these events, the body processes oxygen abnormally—leading to the creation of destructive molecules known as reactive oxygen species. These rogue species tear apart DNA and the protective membranes surrounding cells, putting patients in jeopardy.
In their quest to protect patients, the research team enlisted an unlikely ally: hydrogen. Dr. Kheir
drew inspiration from how these partners on the periodic table interact. When introduced to oxygen-starved cells, hydrogen “soaks up” the toxic oxygen species like a sponge, effectively neutralizing them and reducing cell damage. This breakthrough has already progressed to the bedside.
In a clinical trial at Boston Children’s, Dr. Kheir’s team is administering a hydrogen gas mixture to patients following cardiac arrest.
Vision, perseverance, and expert knowledge certainly were key to advancing the project. But many potentially lifesaving projects don’t progress beyond early stages of research. Philanthropy can provide a critical bridge from idea to implementation. Support from the Pappendick Family Therapeutic Acceleration Award fueled the hydrogen trial and the experiments needed to satisfy the U.S. Food and Drug Administration’s (FDA) safety requirements. When additional costs threatened to derail the project, an award from the Mooney Family Initiative for Translational and Clinical Studies in Rare Diseases allowed for the last-minute modifications that kept the trial moving forward.
“Philanthropy allows people like me—who see patient problems and know they have the tools to create solutions—to innovate and take risks. It moves medicine forward,” says Dr. Kheir.
And momentum continues. The FDA recently approved expanding the hydrogen trial to other pediatric medical centers, and Dr. Kheir is working to bring the therapy to adult stroke and cardiac arrest patients.
PHILANTHROPY ALLOWS PEOPLE LIKE ME TO INNOVATE AND TAKE RISKS. IT MOVES MEDICINE FORWARD.
Hearing Loss
BOSTON CHILDREN’S IS ADVANCING TREATMENTS, IMPROVING CARE, AND LEADING CONFIDENCE-BOOSTING PROGRAMS FOR KIDS EVERYWHERE.
CAT AND KIT SHOW THE SIGN FOR CAMP.
IT’S NOT EVERY DAY you meet a mouse—especially one created from your genetic blueprint. But that’s exactly what happened when Lila visited the Translational Hearing Genomics Lab at Boston Children’s.
When she was 10, her parents, Louis and Jennifer, were shocked to discover their daughter had hearing loss due to a change in the gene TMPRSS3. While exploring treatments, they found a study that led them to Jeffrey Holt, PhD, and Eliot Shearer, MD, PhD, experts in gene therapy for hearing loss.
“In our hearts, we didn’t think gene therapy would help Lila,”
says Jennifer. “But after learning about Boston Children’s research, we knew it was going to happen. The question was when.”
They established the Louis and Jennifer Hernandez Hearing Fund in 2019, enabling scientists to study TMPRSS3 hearing loss, which affects thousands of patients. Dr. Shearer and Karl Koehler, PhD, created mouse models based on Lila’s exact genetic change, and inner ear organoids (human tissue cultures grown from stem cells) from her own cells. Now, they’re testing a gene therapy developed by Dr. Holt to see if it can restore hearing for Lila and others like her.
Enhancing Care
Louis and Jennifer aren’t the first to ask about gene therapy for hearing loss. In the clinic, Dr. Shearer sees many families curious about these treatments, and he believes millions of children could be eligible. He envisions a future in which otolaryngologists diagnose a child and create a gene therapy specifically for them. It’s an ambitious goal—each of the 150 genes implicated in hearing loss requires its own therapeutic.
Dr. Shearer and his colleagues rely on Boston Children’s hearing loss registry to inform their studies
JOHN GILLOOLY
and accelerate the path to diagnosis. Siblings Sofia and Pedro De Garay helped expand it through the De Garay Family Hearing Loss Research Fund after Margaret Kenna, MD, MPH, treated a family member. The registry contains thousands of patients’ clinical information and has benefitted many children, including Valeria, a youngster from Mexico City.
Her mother, Rossi, sought answers for her daughter after inconclusive tests. Using the registry, Dr. Kenna diagnosed Valeria with congenital cytomegalovirus (CMV), a main culprit of hearing loss, and recommended cochlear implants for Valeria. Rossi was inspired to give back.
“I wish more people could get the same treatment,” says Rossi. “When I decided to support Boston Children’s, I divided my fund into two parts: the VCL Research Fund to advance Dr. Shearer’s research, and the VCL Observership in Audiology and Speech Language Pathology to help bring Boston Children’s quality of care to Mexico.”
For Valeria, a diagnosis opened doors to the best treatment for her.
Many kids aren’t as lucky. Despite improvements in screening, current tests miss 30% of children with hearing loss, particularly those with milder or progressive forms. Another problem? Diagnosis can take months, narrowing the treatment window and causing kids to miss key milestones. Our researchers are working to change that.
“The earlier you provide hearing for children, the better,” says Dr. Shearer. “We’re trying to improve newborn screening by developing a genetic analysis platform to provide results within days, as opposed to months.”
ANSWERS FOR HEARING LOSS
COCHLEAR IMPLANTS BYPASS DAMAGED PARTS OF THE EAR TO STIMULATE AUDITORY NERVES AND PROVIDE A SENSE OF SOUND.
ON THE HORIZON GENE THERAPIES RESTORE HEARING BY TREATING THE UNDERLYING CAUSES OF DEAFNESS.
LEFT: Philanthropists Kimberly Hsu-Barber and Jeff Barber with 2024 Boston Investment Conference awardee Dr. Holt.
RIGHT: Dr. Shearer (middle) operates on a young patient.
He’s testing the diagnostic plat form that screens for CMV and ge netic conditions on several hundred children. He hopes to get the aver age age of diagnosis from 9 months to 3 months old.
Dr. Shearer’s team is also im proving research and care for His panic and Latino individuals, who typically have inconclusive test re sults because they’re historically underrepresented in clinical stud ies. Dr. Shearer conducted the first genetic study on 30 Mexican chil dren using current methods and diagnosed almost half of that pop ulation. Now, he’s mentoring Mex ican audiologists and physicians through the Escuchar Sin Fron teras program and training them to perform genetic screening and care for children with cochlear implants.
Creating a Ripple Effect
Support from grateful families has enabled our experts to estab lish a biobank of stem cells from children with genetic hearing loss. This will allow them to create organoids and test therapies. Dr. Shearer, who received funding from the Translational Research Program in 2024, says he couldn’t have reached this point without philanthropy.
“I’m excited to talk to parents of children with hearing loss, to tell them about treatments on the horizon.”
Meanwhile, Lila, now 18, still thinks about that mouse and the possibilities it represents for deaf and hard-of-hearing children.
“I’m thrilled to see the progress in hearing loss research,” says Lila. “I hope this work can help people like me.”
CAMP MEMORIES WITH CAT AND KIT
Meet 7th graders Cat and Kit. They’re athletes. Cat skates with a roller derby team. Kit’s a soccer goalie. They share a fondness for school dances and theater. The girls, who both have hearing loss, met at Boston Children’s Success Leadership Camp last spring.
Cat started thinking about camp in 4th grade. “I was obsessed.”
It’s easy to see why. Designed for middle schoolers, the weekend is jam-packed with team-building games, American Sign Language lessons, and leadership development.
The best part of camp? It’s a toss-up. “It’s nice to be around other kids who understand hearing loss,” says Kit. But the opportunity to plan a recreational event for younger kids with hearing loss allowed campers to test their newfound leadership skills.
“We had to create games and explain the rules,” says Kit. “And make sure everyone stayed safe,” adds Cat. Not surprising, the kindergartners loved the game “Run and Scream,” a favorite among older kids as well.
What’s next for the duo? Both are working on signing and staying connected to their camp peers. They’re also counting the days until Success Leadership Camp 2025.
Big Questions
CAN INVESTING IN MENTAL HEALTH HELP CURB SCHOOL SHOOTINGS?
Mom, I’m scared … I heard gunshots … I’m hiding in math class ... I love you.
Texts like these fuel parents’ nightmares.
The statistics are grim. Since the Columbine shooting in 1999, more than 338,000 students have experienced gun violence. Today, in the United States, there’s nearly one school shooting every day, leaving many parents feeling powerless. But Heidi Ellis, PhD, a clinical psychologist, says we owe it to our children to act swiftly and with optimism.
Mental illness doesn’t cause violence. However, young people who commit violence often struggle with problems that mental health providers can help address, says Dr. Ellis.
Dr. Ellis and her team have engaged in research and programming over the past 10 years to understand and prevent youth radicalization to violence. “We’ve found that these acts are an expression of psychological distress by young people who are suffering. They’re experiencing bullying. They don’t feel like they belong. These kids are like every other kid—only they’re misdirected on how to gain some sense of meaning and purpose.”
Boston Children’s partners with schools to foster environments where all children feel valued and receive mental health support. The hospital is expanding its efforts to identify struggling students, addressing issues before they reach a crisis point. However, communities urgently need experts who can work with students who demonstrate risk factors for violence but don’t pose an immediate risk of harm to others.
Most mental health providers haven’t been trained— and many are unwilling—to treat kids at risk for targeted violence. “Anecdotal data from youth referred to our program suggest that 1 of 3 had been turned away by mental health providers,” says Dr. Ellis.
To address this gap, Boston Children’s has launched
the nation’s first clinic that specializes in treating young people who need intensive help to move off their current pathway.
The team works to address the psychological drivers of violence and re-engage kids in their families, schools, and communities. Treatment can involve therapy to help them heal from bullying or self-loathing. But it also extends outside the clinic: coaching parents and teachers on how to minimize stressors, while increasing support for students with tutoring and school clubs. Community partners also help the clinic bolster protective factors through skill-building and mentorship.
“We have the tools to build a system of care that reaches youth before they engage in horrific acts,“ says Dr. Ellis. “We need the political will, resources, and training to make these services available in all communities.”
KNOW THE SIGNS*
The red flags of an individual at risk of violence:
3 Withdrawing suddenly from friends, family, and activities (including online)
3 Bullying, especially targeted at differences in race, religion, gender, or sexual orientation
3 Demonstrating excessive irritability, impatience, or anger
3 Experiencing chronic loneliness or social isolation
3 Expressing thoughts of harming themselves or someone else
3 Making threats toward a place, a person, or themselves
3 Bragging about access to weapons
3 Recruiting accomplices or audiences for an attack
3 Harming animals intentionally
Call 911 if you suspect an immediate threat. If you see early signs of troubling behaviors, reach out to MAPP@childrens. harvard.edu
*ADAPTED FROM THE
ORGANOIDS: THE BODY’S MACHINERY IN MINIATURE
Microcosms of life, organoids could hold the key to treating diseases.
STEM CELL-DERIVED ORGANOIDS SHOWING HAIR AND NERVE CELLS
MAKING AN ORGANOID —a small, functional, 3D model of an organ, tissue, or other biological specimen—is a lot like following a recipe. Stem cells, the main ingredient in the dish, are mixed with a blend of chemicals present during normal embryonic development. The types, amounts, and timing of chemicals determine what kind of organoid the cells will become: a little more of this, a little less of that, and scientists can coax a miniature ear into being; tweak the ingredients and that ear transforms into craniofacial skin. While not exact scaleddown reproductions of full organs, these specimens contain living cells that can grow, divide, communicate, and perform cellular functions, just like cells within human bodies.
At Boston Children’s, scientists are using organoids to study human development, probe disease origins, and test promising therapeutics. Some of these investigators are generating organoids from cells collected during patient biopsies to explore personalized approaches to care; others start with stem cells from humans or mice that can differentiate into specific cell types in the body. Pioneers in the field, our researchers also have created novel organoids, including first-ever models of the lung, bone marrow, and hair-bearing skin.
From head to toe, these organoids are transforming what scientists can learn in the lab and accelerating the pace of life-changing discoveries.
PEDIATRIC BRAIN TUMORS are the leading cause of childhood cancer death—a statistic Xin Tang, PhD, is working to change. His lab uses brain tumor organoids to reconstruct the microenvironment in human brains and understand how brain tumor cells and immune cells, or microglia, interact. The organoids allow Dr. Tang to explore the dynamics between microglia and tumor cells, evaluate anti-cancer drugs, and stimulate microglia to fight tumors. Importantly, the Tang team can observe impacts of anti-cancer therapies on both tumor and normal brain cells in the same organoid—critical for determining which therapies and approaches are both effective and safe.
SKIN & INNER EAR
EARLIER IN HIS career, Karl Koehler, PhD, focused his research on inner ear organoids to study hearing loss and balance disorders. After realizing that a simple modification to the ear cell culture would produce a skin organoid instead, he began investigating sensory organs more broadly. Now, his lab uses ear, skin, and retinal organoids to model diseases and test gene therapies.
One such disease is Usher syndrome—an inherited condition that causes deafness and blindness in
AIRWAY ORGANOID GROWN FROM HUMAN LUNG LAVAGE FLUID SAMPLE
BRAIN
MICHAEL GODERRE
DR. MACHLUS
DR. KIM
DR. THIAGARAJAH (BACK RIGHT) WITH HIS LAB TEAM IN 2021
Organoids are transforming what scientists can learn in the lab and accelerating the pace of life-changing discoveries.
children. The Koehler lab is working to develop a gene therapy that could target both organ systems at the same time.
Dr. Koehler also is exploring therapeutics for epidermolysis bullosa (EB), a congenital disorder that makes skin blister at the slightest touch and, in its most severe forms, may be fatal during infancy. Using skin organoids, he can mimic the wounds caused by EB and test different methods to treat, cure, and prevent the disease.
With its extraordinary ability to not only grow hair but also generate nerves, the skin organoid created by Dr. Koehler may lead to improvements in scar and burn treatment, drug and cosmetic testing, and baldness interventions.
HUMAN LUNG organoids are generally derived from lung tissue, which isn’t easily obtained, or stem cells that require complex manipulation. These hurdles aren’t stopping Carla Kim, PhD, from using organoids to advance our understanding of both adult-onset and pediatric diseases. Working with tumor organoids derived from mouse lung cells, the Kim lab discovered cellular changes that occur during the earliest stages of lung cancer and is testing these new targets for treatments. In another study, Dr.
LUNG
Kim and colleagues successfully transplanted cells from healthy lung organoids into mice with damaged lungs, offering early evidence that cell therapy may reverse disease. Now, her lab has refined a protocol for growing organoids from patients’ lung fluid samples, expanding the scope of respiratory diseases that can be modeled from human cells.
BONE MARROW
EVERY 15 SECONDS , someone with a blood disorder, a traumatic injury, or other serious condition needs a transfusion of platelets—the cells that help blood clot—because scientists have yet to discover a way to trigger the body to make more platelets when needed. To help find better solutions for these patients, Kellie Machlus, PhD, and a team of colleagues created the first-ever human bone marrow organoids. Now, the Machlus lab is testing therapeutics that can stimulate platelet production from platelet mother cells, megakaryocytes. INTEST -INES
CREATING ORGANOIDS from patients’ own cells, Jay Thiagarajah, MD, PhD, is discovering new ways to help children with congenital enteropathy and other causes of malnutrition. While his lab pursues gene therapy approaches to
reversing these conditions, the team also found that an anti-diarrheal already approved for use in adults appeared safe and effective in the pediatric organoid model. Dr. Thiagarajah hopes to start a clinical trial of this medication at Boston Children’s in 2025 and eventually expand it to sites around the world. Its success could greatly enhance patients’ quality of life.
In other gastrointestinal research, specialized gut organoids developed by Daniel Zeve, MD, and David Breault, MD, are fueling the work of Jason Zhang, MD, PhD. The Zhang lab discovered that certain bacterial compounds induce the body to make tremendous amounts of GLP1—a hormone that helps control blood sugar and appetite. This finding could lead to probiotic treatments for diseases like obesity and type 2 diabetes that produce fewer side effects than current GLP-1 medications.
BUOYED BY progress and supported by generous philanthropists, our investigators are leveraging their organoid research to ask and answer more questions and apply their work to new areas. In the years ahead, they hope to study additional diseases; improve existing models; develop safer, more effective, and personalized treatments; and disseminate their findings to benefit patients everywhere.
THE POWER OF PHILANTHROPY WE ARE GRATEFUL TO OUR PHILANTHROPIC SUPPORTERS: AGA RESEARCH FOUNDATION, THE CYSTIC FIBROSIS FOUNDATION, THE ELSA U. PARDEE FOUNDATION, F. M. KIRBY NEUROBIOLOGY CENTER INNOVATION AWARD, G. HAROLD AND LEILA Y. MATHERS CHARITABLE FOUNDATION, GLOBAL FOR GOOD FUND INC., HARVARD STEM CELL INSTITUTE, THE HERNANDEZ FAMILY, THE LEONA M. AND HARRY B. HELMSLEY CHARITABLE TRUST, LONGFONDS ACCELERATE, STEPHEN AND BETH MCPARLAND, NASPGHAN FOUNDATION, THE SLIFKA FAMILY, THE THORACIC FOUNDATION, THE TKO STRONG FOUNDATION, THE USHER SYNDROME SOCIETY.
“The human connection at the Walk is so special. I love spending time as a community and seeing hospital leaders in person—knowing they’re supporting patients and employees. We’re thrilled we can direct our support to programs that benefit Latino families.”
EVA, CO-CHAIR AND TEAM CAPTAIN FOR BOSTON CHILDREN’S HISPANIC OUTREACH LATINOS, AMIGOS & ALLIES (HOLA)
“At the Walk, I learned that Boston Children’s treats more kids with rare diseases than any hospital in the world, and I was like, ‘That’s why I’m here!’”
BIA
“I walk in honor of my daughter, Harper. Having a child with a disability or health problem can be isolating, but the Walk helps me feel a little less alone. You’re surrounded by families who get it. It’s unspoken, but it’s like they’re saying, ‘I see you. I hear you.’”
EMILY
“When I was diagnosed with a benign brain tumor at 5, my family traveled to Boston Children’s for my surgery, and I continued receiving care for over a decade. Today, I want to have an immediate impact. Yoga Reaches Out provides the perfect opportunity to help kids.”
EMMA
WHEN: May 4 WHERE: The TRACK at new balance JOIN US:
bostonchildrens.org/yoga
“Boston Children’s is the place you never expect to need—until you do. My family participates in as many events as possible to show our gratitude and to support families like ours. We’ve loved doing the Eversource Walk, and now I lead my own team as a captain for Yoga Reaches Out!”
HEIDI
REGISTER FOR YOGA REACHES OUT
401 PARK DRIVE, SUITE 602 BOSTON, MA 02215-3354
CHILDREN’S LEGACY CIRCLE
Grandparents Give a Gift with Many Benefits
Price and Peggy van der Swaagh remain thankful for the lifesaving cancer treatment their grandson received at Boston Children’s. To demonstrate their gratitude, they selected a tax-savvy strategy that allows them to maximize their impact while saving on taxes using their IRAs.
The qualified charitable distribution, known as the IRA charitable rollover, allows philanthropists 70½ and older to:
• donate up to $108,000 per year to eligible charities, such as Boston Children’s.
• satisfy all or part of their required minimum distributions.
• unlock savings and reduce their taxable income by the amount directed to charity.
The van der Swaaghs employ this strategy and encourage others to consider the same, noting, “If you are philanthropic in nature, this is a wonderful way to meet your minimum distribution.”
Learn more about these and other philanthropic strategies at plannedgiving.bostonchildrens.org
We have the highest regard for Boston Children’s Hospital. It’s why we use our IRAs to support their ongoing fight to make childhood cancer a thing of the past.
