MSKNewsFALL 2019 The Next Generation - How MSK Kids Is Changing the Face of Pediatric Cancer Care - Memorial Sloan Kettering Cancer Center
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MSKNews
FALL 2019
MEMORIAL SLOAN KETTERING CANCER CENTER
The Next
Generation
How MSK Kids Is
Changing the Face of
Pediatric Cancer Care
IT ION
L E D
C IA
SPE
Photo: Karsten Moran
Fall 2019 | 1
MSKHappenings
The graduates celebrate. Photo: Rick DeWitt
MSK’s Class of 2019 was feted on June 6 In his keynote address, MSK Presi-
Grads Seize the at the 13th annual Pediatric Convocation, dent and Chief Executive Officer Craig
Day at Pediatric which recognizes current and former
MSK Kids patients who are graduating
B. Thompson encouraged graduates to
take risks and not fear failure in their
Convocation from high school. The event is a happy
reunion for families and care teams, and
years ahead.
Finally, Nina Pickett, Senior Director
MSK Kids Chair Andrew Kung speaks at the festivities as children take center stage during the day’s events. Photos: Rick DeWitt
By Meredith Begley and a time to look ahead to all that awaits of Pediatrics, recognized the teachers
Ann Marie Sanchez MSK’s graduating class. who instruct children of all ages at
This year’s convocation was attended
by 37 graduates and their families. Paul
MSK Kids. She thanked them for their
devotion to their students and their
Kids Walk for MSK Kids: Raising
Meyers, Vice Chair for Clinical Affairs flexibility in teaching under unique Funds for Pediatric Cancer Research
and Chief of the Sarcoma Service, emceed. circumstances.
Mary Maher, principal of the New After processing out of the audito- By Meredith Begley and Ann Marie Sanchez
York City Department of Education’s rium to “Pomp and Circumstance,”
Hospital Schools Program, was on hand the graduates celebrated with a lunch- In 2001, Sophie and her classmates founded Kids Walk for MSK Kids to raise money
to present an award to each student for eon and spent time catching up with for neuroblastoma research at Memorial Sloan Kettering, where her younger
their dedication to academics, sports, familiar faces. l brother, Simon, was undergoing treatment for the disease. Today, Simon is cancer
service, and other interests. free and pursuing a PhD, and the event created in his honor is still going strong.
On May 4, more than 3,000 patients, families, students, and other supporters
participated in the 19th Kids Walk in Central Park.
Only 4 percent of federal cancer funding goes to pediatric cancer research.
New York Yankees Partnership Is a Home Run To address this gap, 100 percent of every dollar raised from Kids Walk directly
supports pediatric cancer research at MSK Kids. In 2019, the 1.5-mile walk raised more
By Meredith Begley and Ann Marie Sanchez than $1 million, bringing the total raised by Kids Walk to $7 million since 2001,
with more than $4 million raised in the last four years. These funds provide MSK Kids
Talk about a winning combo: Since 2006, researchers and doctors with the resources they need to improve the lives of children
the New York Yankees and MSK Kids with cancer and make life-changing discoveries.
have teamed up to strike out childhood Andrew Kung, Chair of MSK Kids, says, “Kids Walk is a special event in that it
cancer — and have some fun too. MSK features kids raising money to support other young people with cancer. With these
Kids patients and their families have been donations, MSK Kids has uncovered new treatments, giving hope to children and
treated to a personalized Bronx Bombers their families.”
experience at every home game this Thanks in part to donations from Kids Walk over the years, MSK Kids has developed
season. Fans can buy T-shirts and make treatment strategies that have changed outcomes for advanced neuroblastoma.
direct donations year-round through the Children with this cancer once had a survival rate below 5 percent. It is now nearly
Yankees Universe Fund, which raises 50 percent.
money to provide MSK Kids with critical As the largest pediatric oncology program in the nation, MSK Kids treats more
funding for the development of treatment children and young adults with cancer than any other hospital in the United States.
advances in childhood cancer care. l For over a century, MSK has led the crusade in research and patient care for children
with all types of the disease, from the most common to the rarest. Kids Walk has
From left: MSK fans at Yankee Stadium;
Yankee star Aaron Judge (left) with a supporter.
also supported two clinical trials for leukemia. Without the funding that participants
Photos: Courtesy of the New York Yankees and donors provide through Kids Walk, these initiatives might not be possible. l
2 | MSKNews Fall
Fall2019
2019 | | 3
MSKIn the Lab
DEAR READERS,
ON THE COVER
Like every child, teen, and young By Meredith Begley
adult in the MSK Kids community,
this issue of MSK News, dedicated
to our youngest patients, is unique. When 12-year-old Bryana’s knee started She has had two surgeries, during which
This special edition is a testament hurting last year, her family doctor Dr. Prince was able to successfully remove
to the resilience of our patients thought it was just growing pains. But the remaining tumor. Despite numerous “Rather than normal
an MRI revealed something far worse: hospitalizations, she maintained a posi-
cells becoming cancerous
and their families, the tenacity and
dogged focus of our researchers, and osteosarcoma, a rare bone cancer that tive attitude throughout, relying on a
the unwavering commitment of our tends to affect children, teens, and young
adults. After meeting a specialist near
personal mantra to get her through. “My
blood counts might be low, but my spirits
because of random
dedicated doctors, nurses, and staff.
their home in Florida, Bryana’s mother are high,” she says. Following a summer mutations, PGBD5 itself
produces the mutations
These are the stories of the people decided to take her to New York City, vacation to Japan, Bryana is back at
behind MSK Kids, the nation’s largest where they used to live. school and in her normal routine. She has
pediatric oncology program. “I knew MSK,” says Bryana’s mom, no evidence of disease. l and turns cells malignant.
It suggested a whole
Myrna. “There was no question.”
We hope you come away feeling as
At MSK, Bryana and Myrna met
new category of human
inspired by what you read here as
orthopedic surgeon Daniel Prince, who
we were while putting the issue to-
specializes in bone regeneration in chil-
gether. Please let us know what you
dren. “After we met Dr. Prince, I immedi- cancers that result
think at publicaffairs@mskcc.org.
ately said, ‘Mommy, I want to get treated
here,’ ” Bryana recalls. “This didn’t feel
from the process.”
—The MSK News Editorial Team
like a hospital, and I didn’t feel scared.”
Bryana underwent 29 rounds of che- —Alex Kentsis
motherapy under the care of pediatric
oncologists Paul Meyers and Leonard
Wexler, who are specialists in sarcoma.
Alex Kentsis has shed important light on the process by which an abnormally active gene can trigger the development of pediatric cancers. Photos: Rick DeWitt
CRACKING When cancer strikes a child, the fates In 2017, Dr. Kentsis’s team made a
THE CODE OF seem especially cruel. After all, cancer major discovery: A gene called PGBD5
CHILDHOOD is typically seen as a disease of aging.
A person’s risk rises through the years
can become abnormally activated in
children, unleashing mayhem in various
CANCERS as genetic mutations pile up due to cell types. The protein made by PGBD5
DNA copying mistakes when cells divide snips DNA segments and flips them to a
or exposure to environmental factors. different location, which can drastically
By Jim Stallard
Researchers have long been confounded alter normal gene function and trigger
by why tumors develop in children, who cancer.
presumably haven’t lived long enough “This discovery explained a long-
to accumulate these mutations. standing conundrum as to how pediatric
Memorial Sloan Kettering cancer cancers develop,” Dr. Kentsis says. “Rather
biologist and pediatric oncologist Alex than normal cells becoming cancerous
Kentsis has spent most of his career because of random mutations, PGBD5
looking for an answer to why children itself produces the mutations and turns
get cancer. In the past few years, his cells malignant. It suggested a whole
Sloan Kettering Institute laboratory has new category of human cancers that
begun to unravel the mystery. result from the process.”
Bryana and her mom at MSK, and (bottom left) with surgeon Daniel Prince. Photos: Karsten Moran
4 | MSKNews Fall 2019 | 5
affects soft tissue in the kidneys, liver, Hanno Steen of Boston Children’s Michael Ortiz, an MSK Kids clinical
and brain.
“Despite years of intense laboratory
Hospital. The researchers were looking
for a molecular indicator, or biomarker,
investigator and pediatric oncologist,
confirmed through laboratory experiments
“Despite years of
studies of childhood tumors, this was a that would make it possible to identify that prohibitin is high in Wilms’ tumor intense laboratory
new cause of mutations that the scien- children whose cancers would resist cells. It is even higher in children with
tific world had missed,” says Andrew treatment. They collected urine from advanced disease. “Prohibitin is required studies of childhood
Kung, Chair of MSK Kids. “It represented
a major advance in understanding human
children with various kidney tumors and
from healthy children.
for the survival and growth of cancer
cells in response to chemotherapy,” Dr.
tumors, [PGBD5]
cancer in general and pediatric tumors The team analyzed thousands of Kentsis says. “Normally, chemotherapy was a new cause of
in particular.” proteins and looked for ones in the urine
associated with certain tumors. Early in
kills the tumor cells, but high levels of
prohibitin enable them to survive.”
mutations that the
An Inviting Drug Target the project, the PGBD5 protein stood out Prohibitin is also found in high levels scientific world had
missed. It represented
PGBD5’s means of creating genomic as a biomarker for kidney rhabdoid tumors, in a variety of other solid tumors, including
havoc suggests a therapeutic strategy which led to the jumping genes discovery. neuroblastoma. Based on these findings,
for targeting the cancer cells it creates. Researchers then realized that a Dr. Kentsis thinks drugs that target pro- a major advance in
For a cell to survive, it must use special protein called prohibitin was linked to hibitin are expected to help people with
proteins to fix breaks in the DNA. In treatment resistance in Wilms’ tumor, Wilms’ tumor, neuroblastoma, and other understanding human
a cancer cell, a transposase, such as
PGBD5, creates so many DNA breaks
the most common type of kidney
cancer in children. Almost 90 percent
cancers that persist despite treatment.
“Research like this will allow us
cancer in general and
that the cell is especially dependent on of children with this cancer can be to devise new therapeutic strategies pediatric tumors in
these proteins.
Dr. Kentsis’s team reported that
cured with a combination of surgery,
chemotherapy, and radiation. The team
to eliminate toxic chemotherapy for
children who won’t benefit and to over-
particular.”
recently developed drugs could kill found that prohibitin was present at come treatment resistance in children
PGBD5-producing tumor cells by inter- high levels in children whose Wilms’ tumor for whom current therapy is not suffi- —Andrew Kung
fering with the cells’ DNA repair system. returned after surgery and chemotherapy. cient to cure,” Dr. Kentsis says. l
Research by Alex Kentsis and Michael Ortiz (right) could help pave the way for treatments of such
As a result, the cancer cells become
childhood cancers as rhabdoid tumor and Wilms’ tumor. Photo: Kreg Holt overwhelmed by DNA damage and
self-destruct.
In the future, children with cancer
Jumping Genes could potentially have their tumors
The PGBD5 protein is a DNA transposase, analyzed to see whether they make
a type of enzyme that organisms use to the PGBD5 protein and, if so, receive In 2016, then 3-year-old Caroline
rearrange segments of DNA. Called these targeted inhibitors. This treatment was diagnosed with stage IV
transposons or “jumping genes,” these strategy has already emerged as a Wilms’ tumors in both kidneys.
pieces of mobile DNA move from one promising way to treat other cancers. She was treated by a team of
site on a chromosome to another. (For Drugs called PARP inhibitors appear MSK Kids specialists, and today,
an example, look at corn. When trans- to be effective in women with breast the cancer is in remission. She
posons alter pigment-controlling genes, or ovarian cancers that have mutations and her family — including big
varying colors of kernels appear on a in BRCA genes, which also regulate brother Andy, mom Debbie, and
single ear. The discovery of transposons DNA repair. dad Kevin — have raised more
was recognized with a Nobel Prize.) “This has implications for people with than $200,000 to support the
For decades, researchers thought that a wide range of cancers that make work of MSK Kids oncologist
transposons did not play much of a role PGBD5,” Dr. Kentsis says. Michael Ortiz, who is working to
in how human cells function; they were develop better ways to diagnose
considered remnants of evolution. But A Reliable Biomarker and treat Wilms’ tumor. l
beginning in 2015, Dr. Kentsis’s team led for Wilms’ Tumors
Photo: Karsten Moran
research revealing that PGBD5 could The discovery of PGBD5’s role in child-
cut and paste DNA in human cells. These hood cancers occurred somewhat by
genetic rearrangements were especially chance. A decade ago, Dr. Kentsis began
prominent in human rhabdoid tumors, a collaboration with Elizabeth Mullen of
a rare, aggressive childhood cancer that the Dana-Farber Cancer Institute and
6 | MSKNews Fall 2019 | 7
MSKIn the Clinic
Inside MSK Kids: Jonathan’s Journal
By Jonathan, Age 13
Hi! My name’s Jonathan. I’m from New York, and I’m in eighth grade. I love traveling
with my family, snorkeling, and building Lego cars.
When I was 9, I found out I had leukemia. When my parents took me to MSK Kids,
I was scared. Treatment was really hard, but everyone I met made me feel like they
had my back.
For two years, I spent a lot of time on the ninth floor in the Pediatric Ambulatory
Care Center, getting chemotherapy and other kinds of treatments. Sometimes I got
really sick and I had to stay in the hospital overnight.
Now, I come back to MSK Kids every few months for checkups. A photographer
tagged along during my most recent visit so you could meet some of the awesome
people who take care of me.
p This is my nurse practitioner, Joe. He was one of the first people I met when I came to MSK Kids. He always helped me during my treatment days and
made sure I was getting what I needed. Now, I see him for regular checkups, when he takes my blood pressure, listens to my heart, and tells me about
my test results. After that, we like to swap traveling stories.
p The first person I see when I get to the ninth
floor is Matt. He greets everyone and helps me
get my appointment started. p People like Caitlin and the rest of the IV room
staff were there for me when I was getting
chemo, so I stop by for a high five.
p This is the MSK Kids fish tank. You can’t miss it! There are lots of cool things on the ninth floor for kids
to see and do while they’re here.
p This is Robin. He’s a mind body therapist. He taught me martial arts, tai chi, and meditation to help
keep me calm during treatment. My favorite meditation was when we took a “trip” to the beach.
p Dr. Steinherz was in charge of my treatment
and made sure I got what I needed to get rid of
my leukemia.
p Say cheese! At MSK Kids, you see the same nurses the
p
whole time you’re here, so you can get to know
This is my friend Jess. Playing Legos with her helped me one another. I’ve known Paige for four years
p
get through my chemo treatments. She even brought me now. I have to give her a hug. She’s awesome!
my favorite Lego sets to help cheer me up when I was sick
in the hospital. Jess is a child life specialist. Her job is to
help kids get used to being in the hospital. She also gets
to plan fun stuff for kids and families to do.
Photos: Karsten Moran
8 | MSKNews Fall 2019 | 9
A B C D
IS FOR IS FOR
AZ
ARRAY OF BINGO IS FOR
EXPERTS NIGHT CHILD LIFE
MSK Kids has specialists in a IS FOR
Our specialists help young
variety of fields — including
Every Monday and
Wednesday, youngsters patients and their families DIAGNOSTICS
radiation oncology, physical and their siblings can get develop strategies for coping MSK uses the most-advanced
and occupational therapy, together for an evening with and adjusting to treatment. techniques and tools to ensure
and radiology — working of play and prizes. that patients receive accurate
alongside primary teams and up-to-date information
to provide the most about their disease.
expert care.
E F G
Check out some of the
TO many offerings available IS FOR
H
IS FOR FRIDAY NIGHT IS FOR
to young patients and EDUCATION CANDY CART GIANT FISH TANK
IS FOR
their families at MSK Kids. Step off the ninth floor into MSK
As part of the New York City
Department of Education’s
A beloved tradition
since 1989, the cart Kids and one of the first things HOLIDAY PARTIES
you’ll see is the 162-gallon fish With support from The Society of
By Meredith Begley Hospital Schools Program, brings joy to children
tank, where residents Nemo MSK and other generous donors,
full-time teachers are staying overnight
and Dory are often seen MSK Kids celebrates special days
available in the Pediatric in the hospital.
swimming around. of the year, including Valentine’s
Ambulatory Care Center or
Day, Mardi Gras, the Fourth of July,
at the bedside for students
Halloween, and more.
in pre-K through high school.
I K L M N
IS FOR IS FOR
J
INTEGRATIVE IS FOR LONG-TERM IS FOR IS FOR
MEDICINE KIDS BEING KIDS FOLLOW-UP MEDICAL PLAY NURSES
Our unique Integrative Medicine IS FOR The abundance of programming at MSK Kids A team of dedicated experts in A miniature operating room, At MSK Kids, our award-
Service offers kid-friendly yoga, JUGGLERS allows young people to retain a sense of survivorship is committed to helping lifelike puppets, and doll-size winning nurses know the
creative movement, music therapy, Members of Healthy normalcy during their time in the hospital. pediatric cancer survivors maintain medical equipment help younger little details that make each
karate, and more to complement Humor visit MSK Kids the highest possible level of health kids become familiar with the child an individual: their
traditional medical care. three times a week to and manage any needs. hospital environment. favorite flavor of juice, for
clown around with example, or the artists on
the children. 2021 their latest playlist.
2020
2019
P Q R S T
IS FOR
TYA@MSK
O
IS FOR IS FOR
IS FOR QUICK SCHEDULING RESEARCH The diagnosis and treatment
OUTPATIENT IS FOR The collaboration between IS FOR experience of teens and young
CARE PSYCHOSOCIAL A cancer diagnosis can
turn a parent’s world our doctors and researchers SUPPORTIVE CARE adults is unique. Our Teen and
More than 90 percent of the CARE upside down. Our experts means that young patients
Our pain and palliative care
Young Adult Program
can benefit from the latest (TYA@MSK) offers services
care delivered at MSK Kids is Our robust psychosocial care team aim to schedule an initial program staff works hard to
advances in cancer care, to decrease isolation,
done on an outpatient basis, includes psychologists, psychiatrists, appointment within make children, teens, and young
many of which were devel- improve connections
so kids can spend more time social workers, chaplains, physical 24 hours of a family’s adults as comfortable as possible
oped across the street in among peers, and
out of the hospital. and occupational therapists, child life first call to MSK Kids. while they’re under our care.
our research labs. address psychological
specialists, and teachers.
and social needs.
U V IS FOR
VOLUNTEERS
W Y Z IS FOR
ZOOM
X
IS FOR
IS FOR IS FOR YEARLY
UNPARALLELED More than 100 WHOLE-GENOME IS FOR CELEBRATIONS
Little ones about to have
surgery can ride into
EXPERTISE volunteers come SEQUENCING X-TRA SPECIAL During MSK’s annual the operating room
through MSK Kids
Our team of experts, made up of each week, offering
It’s one of the ways that
SIBLING PROGRAMS talent show, children and in style on remote-
people across a variety of specialties, MSK Kids understands staffers wow the crowd. control cars.
respite for caregivers The Specially Important Brothers
is world-renowned in treating all and targets tumors At Pediatric Prom, kids of
and assistance with and Sisters program ensures that
pediatric cancers, from the most with more-effective all ages don their finest
group activities. the siblings of young patients are
common to the most complex. treatments. for a fancy party.
recognized, valued, and cared for.
Rihanna was born with a tumor on her
upper right arm almost as large as
her head. After chemotherapy failed to
shrink it, her parents brought her to
Memorial Sloan Kettering, hoping for a
different outcome. “MSK’s doctors gave
me a sense of confidence that they knew
what they were doing,” says Rihanna’s
mother, Ana.
And the MSK team delivered: They
determined that Rihanna’s cancer was
caused by a mutation in a gene called
NTRK and focused her care plan on
attacking the cancer-driving effects of
that mutation.
At the time, MSK Kids pediatric
oncologist Neerav “Neal” Shukla was
leading a clinical trial for larotrectinib
(Vitrakvi®), a drug that specifically
targets NTRK. Within days of starting
Rihanna on larotrectinib, the tumor be-
gan to shrink. Five months later, the
Above: Neerav “Neal” Shukla is Director of the
remaining tumor was removed, allowing Pediatric Translational Medicine Program. Photo:
Rihanna to have full function of her arm. Karsten Moran. Right: Dr. Shukla with Rihanna
Now 2, she has shown no sign of the and her mother, Ana. Photo: Ana Plaza
GETTING TO THE ROOT disease. “She is a happy, healthy toddler,”
Ana says. A major effort is underway to fully deliver
OF PEDIATRIC CANCERS
Rihanna’s story illustrates the power on the promise of precision medicine for
of precision medicine. This practice is our youngest patients.
grounded in uncovering the genetic Successfully treating one child, like
Targeting the Gene Mutations That Tumors Thrive On changes that drive a tumor’s growth Rihanna, can do a lot to accelerate drug
and then finding the best drugs to development to help many more kids.
stop them. Over the past two decades, The trial she participated in resulted in
By Julie Grisham
precision medicine has helped many the simultaneous approval of larotrec-
adults with cancer, leading to dozens of tinib for both children and adults with
more effective, less toxic drugs for can- NTRK-driven tumors. In the past, children
cers of the lung, colon, breast, and more. often had to wait until after a drug was
But the progress has been slower approved in adults before pediatric trials
for children. MSK Kids is changing that: were launched.
“As the largest pediatric oncology program in the
world, we are well-positioned to deliver on the promise
of precision medicine and to learn from every
child who we have the privilege of caring for.”
Above: MSK Kids Chair Andrew Kung.
—Andrew Kung Photo: Rick DeWitt
Rihanna before her first birthday. Photo: Ana Plaza
12 | MSKNews Fall 2019 | 13
program, all children cared for at MSK
are offered comprehensive tumor testing.
Being able to deliver therapies to the
right target requires identifying the root
cause of each tumor. This interdisciplinary
approach is being spearheaded by genet-
icist Elli Papaemmanuil and her team of
data scientists.
“We want to understand the genomic
drivers of cancer in children, identify the
key targets, and understand which pa-
tients will respond to targeted therapies.
In this way, we can develop a treatment
approach that is patient tailored and Pediatric oncologist Kim Kramer (left) with researcher Nai-Kong Cheung. Photo: Rick DeWitt
data driven,” Dr. Papaemmanuil explains.
Her lab has performed in-depth Geneticist and data scientist Elli Papaemmanuil.
analyses on tumors from more than Photo: Michael Prince Addressing an Urgent Need with Science
120 children treated at MSK. “Our
preliminary data have opened our eyes
to the diversity and complexity of “Our preliminary data When MSK researcher Nai-Kong Cheung developed a drug called omburtamab
in his laboratory, his work filled a need for better therapies to treat neuroblastoma
pediatric cancers,” she says. have opened our when it spreads to the brain, previously an incurable disease.
“We have developed and optimized Omburtamab, an antibody linked to a radioactive isotope that seeks out
the processes required to deliver tumor eyes to the diversity cancer cells, delivers a big dose of radiation while sparing healthy cells. MSK
analyses quickly enough to benefit
patients,” she adds. “We have shown
and complexity of pediatric oncologist Kim Kramer is excited about what this drug could mean for
her young patients. “It has the potential to have tremendous impact not only on
that this is possible.” pediatric cancers.” neuroblastoma but on other cancers that express the protein that omburtamab
targets,” she says.
Pediatric oncologist Julia Glade Bender is part of the Pediatric Translational Medicine Program. How Research Translates Dr. Kramer has enrolled dozens of patients in her ongoing international
—Elli Papaemmanuil
Photo: Karsten Moran to Treatment clinical trial for omburtamab, which was given a Breakthrough Therapy
Part two of precision medicine is devel- Designation from the US Food and Drug Administration in June 2017.
Making Progress in the Lab oping drugs that can potentially target Doctors, molecular pathologists, and “We understand how urgently these new drugs are needed,” Dr. Kramer says.
These efforts are guided by MSK’s the changes driving children’s tumors. data and laboratory scientists work Dr. Cheung is also working on another drug for neuroblastoma called
Pediatric Translational Medicine Program Once a tumor has been characterized, together to make treatment decisions naxitamab, which received the FDA’s Breakthrough Therapy Designation in
(PTMP), which is led by Dr. Shukla. members of the PTMP’s clinical research based on what they’ve already learned 2018. Of his team’s commitment to developing new drugs for pediatric cancers,
The PTMP is involved in genomically team take over and start working on about the underpinnings of pediatric tumors. he asks, “If we don’t take on this challenge, who will?”
characterizing patients’ tumors as well as treatments matched to changes in the If a child appears to be a good In another area, MSK medical oncologist Renier Brentjens is collaborating
developing new therapies. Through this cancer genome. candidate for a treatment matched to a with MSK Kids Chair Andrew Kung to develop engineered chimeric antigen
genetic change in their tumor, the next receptor T cell therapies for osteosarcoma, a type of bone cancer. “We are
challenge is gaining access to the drug. optimistic that this approach will address the tremendous challenges these
The best option is to enroll the child in patients face,” Dr. Kung says. l
an ongoing clinical trial. This is what
happened with Rihanna.
“We aim to have a clinical trial resources, including finding a company scientists who specialize in pediatrics.
available for every patient, but even willing to supply a drug and getting For example, clinical trials developed by
common mutations are present in only permission from the US Food and Drug MSK’s Early Drug Development Service
1 or 2 percent of pediatric cancers,” says Administration to administer it. It can be can now include children as young as 12.
Julia Glade Bender, a pediatric oncolo- a lengthy and labor-intensive process. (Previously, the age requirement was 18,
gist who is part of the PTMP. “Doing “We anticipate that many of the as it is for most clinical trials.)
a clinical trial for every genomic abnor- drugs we test in individual patients can “As the largest pediatric oncology
mality that we find is just not feasible.” eventually benefit a greater number of program in the world, we are well-
For rarer mutations, a more children with cancer,” says Dr. Glade positioned to deliver on the promise of
specialized approach may be needed. Bender. So far, at least five drugs first precision medicine and to learn from
“This is when we develop a single- given as SPUs at MSK Kids have pro- every child who we have the privilege
patient use [SPU] treatment plan,” gressed into pediatric clinical trials. of caring for,” says MSK Kids Chair
Nancy Bouvier, a research project manager, with pediatric oncologist Filemon Dela Cruz, a member of Dr. Glade Bender says. These compas- MSK’s efforts to develop drugs Andrew Kung. l
the Pediatric Translational Medicine Program. Photo: Karsten Moran sionate-use plans require tremendous for kids go beyond the doctors and
14 | MSKNews Fall 2019 | 15
MSKSociety MSKInterview
we could better predict the rebuilding of
THE SOCIETY WELCOMES A NEW PRESIDENT the immune system after transplant. Plus,
it increased survival chances and there were
fewer dangerous side effects for the child.
LUCY, AGE 12: What is different about
getting a transplant at MSK?
Over the past ten years we have made
major progress in making BMTs safer and
Medical oncologist Santosha Vardhana,
one of the 2019 Scholars Prize recipients, more effective for children. At MSK Kids,
with his family half of the BMTs we do are for diseases
that aren’t cancer, so gene therapy is
SUPPORTING going to be a big game changer.
TALENTED 8 Questions with Here’s how it works, Lucy: We take a
child’s own stem cells, modify them
RESEARCHERS J. J. Boelens outside the body, and then give the child
WHO ARE By Meredith Begley
their gene-corrected cells back after
NEW PARENTS chemotherapy. Because we are using a
kid’s own cells, there is no risk of graft-
Established in 2016 and first versus-host disease, which is when the
awarded in 2017, The Society Photo: Rick DeWitt new cells attack the body. I predict that
Scholars Prize honors postdocs gene therapy will be available for most
Kate Allen was named the 37th Society President in May. Born and raised in Austin Jaap-Jan “J. J.” Boelens joined MSK Kids in 2018 as Chief of the Stem Cell
performing at the highest levels noncancerous blood diseases in five to ten
and a graduate of the University of Virginia, Mrs. Allen has lived in New York City
who are also managing familial Transplantation and Cell Therapy Service. For this very special edition of MSK News, years. We already have access to gene
for 24 years. Her involvement with The Society of MSK began on the Associates some of our MSK Kids patients asked him the questions they were most curious about.
obligations and adjusting to being therapy for some diseases in clinical trials
Committee. During eight years of service, she co-chaired the Bunny Hop, the
new parents. The merit-based prize here at MSK Kids. We also have chimeric
Antique Show (now known as TEFAF), and The Society Campaign, finally serving as
is awarded annually to at least antigen receptor T cell and other immuno-
Vice Chair of Membership. Upon moving to the Administrative Board, she co-chaired
ten full-time postdoc researchers. JAMES, AGE 4: You’re a new doctor favorite depends on the day, my mood, therapy treatments, which rev up the body’s
the Spring Ball and served on the Executive Committee as Secretary from 2017
It provides a cash award of up to here at MSK Kids. Where did you the weather, the season, deadlines, how own immune system to fight cancer. It is
to 2019. Mrs. Allen is active on the board of her summer church in Maine, Christ
$1,250 per year for up to four years. l come from? busy I am — it all factors in! such an exciting time!
Church Dark Harbor, and regularly volunteers at her children’s schools. She and her
I moved from the Netherlands last August
husband, Chris, have two children, Katie, 14, and Dillon, 11. l
with my wife and my three girls, ages 17, NAOMI, AGE 18: What are you studying? MATTHEW, AGE 8: What do you like to
14, and 7, plus our two cats and our horse. That’s a big question with a long answer, do when you’re not working?
It was not easy to move the whole family, Naomi. I have been studying bone marrow Definitely travel, Matthew. And I like to be
but they were all excited to take this step. transplants (BMTs) for the past 15 years. I active. I like hiking and scuba diving.
PRIZE RECOGNIZES CONTRIBUTIONS TO THE The Netherlands is pretty small, and we noticed that in general, the process wasn’t
HECTOR, AGE 9: What do you like best
were looking for some adventure. standard from start to finish. So I started
FIELD OF PEDIATRIC ONCOLOGY to analyze how the chemotherapy and im- about New York?
JULIA, AGE 13: Why did you come to munotherapy we gave before a transplant So many things, Hector! The city is really
The Society of Memorial Sloan Kettering Prize is given annually to recognize MSK Kids? affected how the immune system rebuilt diverse, and the food is great. You can get
an individual who has made significant or groundbreaking contributions to the Coming here was an opportunity for me itself afterward. We looked at everything everything here, and it’s super easy.
field of pediatric oncology. The 2019 award was given to Kevin Shannon, a professor to give kids treatments that could give — the drugs and the dose, and how much
in the department of pediatrics at the University of California, San Francisco. them a second life — a very long second of the drugs were in the blood — and then OMAR, AGE 15: What has surprised you
His research into RASopathies, a group of rare genetic conditions, has advanced life, like six or seven decades. It’s giving asked how all of that related to survival the most about MSK Kids?
the understanding of the causes of and treatments for various childhood and adult them their whole life back after they have outcomes in each patient. The blood levels Lots of things, Omar, but probably the
cancers. Dr. Shannon has also played a key role in training several leaders in the field. been so sick. It is very gratifying. of the drug in the body after we adminis- large number of patients we get to see
He currently serves on the External Advisory Board of MSK’s Human Oncology and ter the medication — what we call drug here. I really want more people to know
Pathogenesis Program. He has served on the National Cancer Institute’s Board EMILY, AGE 11: Your shirts are really exposure — is a really good way to predict about MSK Kids, especially about BMTs
of Scientific Advisors, the Pediatric Cancer Working Group for the NCI’s Cancer cool! How do you pick them? a child’s response to treatment. After we and cellular therapy. We have so many
Moonshot Initiative, and the American Cancer Society’s Council for Extramural Grants. l That’s a very important question, Emily! adjusted the dose based on weight and more resources than other centers do,
MSK Kids Chair Andrew Kung and 2019 Society Prize
They need to be a little bit colorful, and other variables, and started measuring the and we want to be able to share what we
winner Kevin Shannon. Photo: Rick DeWitt
they need to be a certain motif or theme. actual drug levels in the body, we figured learn here in a way that makes it easier
They are usually by a couple of designers out how to get to a consistent drug for other doctors around the world to
The Society of Memorial Sloan Kettering, founded in 1946, is a volunteer-led organization within MSK dedicated to promoting the well-being of
patients, supporting cancer research, and providing public education on the early prevention, detection, and treatment of cancer. who have shops on the Internet. My exposure for everyone. The result was that help more kids. l
16 | MSKNews Fall 2019 | 17
MSKPeople
ENDOWED CHAIRS APPOINTMENTS AND PROMOTIONS
Prasad Adusumilli Michalis Aristophanous Lisa Carter-Harris Irene Cheung
Surgeon Medical Physicist Behavioral Scientist Clinical Immunologist
Promoted to Member; Department Appointed as Associate Clinical Appointed as Associate Member, Promoted to Laboratory Member;
Dana Pe’er was appointed to the Alan and of Surgery, Thoracic Service Member; Department of Medical Memorial Hospital; Department of Pediatric General Oncology Service
Physics, Radiotherapy Physics Psychiatry and Behavioral Sciences,
Sandra Gerry Endowed Chair.
Service Behavioral Sciences Service
Ian Ganly Daniel Gomez Jan Grimm Chris Kotsen
Christopher Lima was appointed Chair of the Structural Biology Surgeon Radiation Oncologist Radiologist Clinical Psychologist
Program at SKI. Dr. Lima is a fellow of the American Academy of Arts Promoted to Member, Memorial Appointed as Member, Memorial Promoted to Member; Molecular Appointed as Associate Clinical
and Sciences and a Howard Hughes Medical Institute Investigator. Hospital; Department of Surgery, Hospital; Department of Radiation Pharmacology Program, Sloan Member; Department of Psychiatry
Martin Tallman was appointed to the Cassidy Head and Neck Service Oncology, External Beam Kettering Institute; Department and Behavioral Sciences, Behavioral
Family Endowed Faculty Chair. Radiotherapy Service, Manhattan of Radiology, Molecular Imaging Sciences Service, Tobacco
and Therapy Service Treatment Program
MSK KUDOS MSKNews
Kathryn Anderson, Maria Jasin, and International Association for the Inquiries about MSKNews should be addressed to:
Dana Pe’er were named to Crain’s 2019 Study of Lung Cancer. Memorial Sloan Kettering
list of Notable Women in Health Care. Department of Communications
Michel Sadelain was awarded the
Piro Lito was selected to join the Jacob Heskel Gabbay Award in 1275 York Avenue
2019 class of Pew-Stewart Scholars Biotechnology and Medicine New York, NY 10065
for Cancer Research by the Pew from the Rosenstiel Basic Medical Galina Levin Rosna Mirtcheva-Trocheva Phone: 212-639-3573
Diagnostic Radiologist Radiologist www.mskcc.org
Charitable Trusts and the Alexander Sciences Research Center at Appointed as Associate Clinical Appointed as Associate Clinical
and Margaret Stewart Trust. Brandeis University. Member; Department of Radiology, Member; Department of Radiology,
Body Imaging Service Molecular Imaging and Therapy Ami Schmitz Editors
John Maciejowski was named a Craig B. Thompson was recognized Service Vice President, Margaret Breuninger
Pew Biomedical Scholar by in Becker’s Hospital Review’s list of Editorial and Content Andrew Roth
the Pew Charitable Trusts. 105 Physician Leaders to Know. Strategy
Blima Marcus was honored by Santosha Vardhana received a Randi Press Special thanks to Rachel
Andrea Schietinger was named
The Jewish Week in its annual Burroughs Wellcome Fund Career Creative Director Corke, program manager
an inaugural member of the
36 under 36 special section. Award for Medical Scientists. of MSK Kids, for her
Cancer Research Institute’s
Maria Rubinstein received the fourth Anas Younes received the 2019 Lloyd J. Old Scientists Taking Kristin Glick contributions and support.
annual Young Investigators Award Distinguished Leadership Award Risks Program and awarded a Associate Art Director
from Gateway for Cancer Research. from the Lymphoma Research research grant.
Foundation. Michael Morris Milind Rajadhyaksha
Charles Rudin received the Paul A. Medical Oncologist Optical Imaging Specialist
Promoted to Member; Department Promoted to Member; Department NCI-designated Comprehensive Cancer Center ©2019
Bunn, Jr. Scientific Award from the
of Medicine, Genitourinary of Medicine, Dermatology Service
Oncology Service
18 | MSKNews Fall 2019 | 19MSKBefore You Go
SOMEWHERE OVER THE RAINBOW
By Andrew Roth
In January, nearly 50 children and teens with cancer and their siblings
lent color to their experiences at MSK, with help from fashion designer
Tanya Taylor and her team, in a color therapy painting class.
The class took place in the Pediatric Ambulatory Care Center’s Recreation Center. Color therapy
is about associating color to help people express emotions. In this class, Ms. Taylor used prompts to help
participants paint, including “What do you feel when you’re happy?”
The class was part of an ongoing partnership between MSK Kids and Ms. Taylor, who previously redesigned the exam room
curtains at MSK Kids and produced head scarves for adults and children undergoing chemotherapy, with profits going to MSK.
In the summer of 2018, her team painted the Art Shack at Sunrise Day Camp, a free summer camp for children with cancer and
their siblings.
An art installation was created with 42 of the student paintings from the color therapy class; it’s currently on display in the entry
lobby of the Pediatric Ambulatory Care Center. l
Left and right: Children painting during Ms. Taylor’s color therapy class.
Center: The finished mural. Photos: Rick DeWittYou can also read
