The Culture of Different MKTG_150064494_2018 Service Line Big Book Full_FIN | Page 24

Apheresis machines like this one
collect and seperate blood products
with extraordinary precision.
Pediatric hematologist Daniel
Ambruso, MD, works with Terumo,
the machines’ manufacturer, to
refine their capabilities even further.
CENTER FOR CANCER AND BLOOD DISORDERS
CANCER
Biobanking a Better
Future for Bone
Marrow Failure
LEADERSHIP:
Lia Gore, MD, Chief,
Pediatric Oncology,
Hematology and Bone
Marrow Transplant
The Ergen Family
Chair, Pediatric Cancer
READ MORE
ABOUT CANCER:
“A Step Ahead of
Papi’s Cancer,” p. 18
“The Uncharted
World of Childhood
Polyposis,” p. 26
“Detecting
Endocrinopathy
After a Brain Tumor,”
p. 67
“A Preventive
Look at Bladder
Dysfunction After
Cancer,” p. 82
Severe aplastic anemia is rare
— about 100 cases per year
nationally, many with no clear
cause. Collaboration, says
pediatric hematologist
Taizo Nakano, MD, will be
key to improving outcomes.
He and his team at Children’s
Hospital Colorado have
collaborated with Boston
Children’s Hospital to build a
biobank to study the disease.
“Already,” he says, “we’ve
identified novel gene mutations
that disrupt unstudied pathways
in bone marrow failure.”
Through the North American
Pediatric Aplastic Anemia
Consortium — the first
organization of its kind —
prospective clinical trials are
underway, targeting these
mutations for potential new
therapies. One day, Dr. Nakano
hopes, they may lead to better,
more personalized options
for care.
Refining Apheresis for a
New Cancer Paradigm
CANCER AND BLOOD DISORDERS
150+
Bone marrow transplants performed
(2016-2017)
97%
100-day survival rate for bone marrow
transplants (2014-2016)
400+
Ongoing clinical trials
10
National research consortia memberships:
Childhood Ependymoma Research Network
Pediatric hematologist Daniel Ambruso, MD, looks
in on one of the patients at Children’s Hospital
Colorado’s Blood Donor Center for Apheresis
Clinic today. A tube snakes from the teen’s left arm
to a machine the size of a nightstand. At 3,400
revolutions per second, the machine will remove
red blood cells damaged by sickle cell anemia,
replace them with donor cells, and pump the blood
back into his other arm.
Dr. Ambruso knows the machine well. He’s
been working with apheresis machines for 40
years. And since 2015, he and his team have
worked with Terumo, their manufacturer, to
refine their capability.
They’re capable of much.
For example, another patient in clinic today had
a bone marrow transplant for aplastic anemia, a
process that starts with harvesting her donor’s
ste m cells through apheresis. The patient’s
immune system reacted to the transplant,
unfortunately, but apheresis can help there, too.
She’s having her blood removed and treated with a
photosensitive chemical and UV light to kill off the
T cells causing the reaction.
The machines isolate blood components with
astonishing precision. Their applications are
many, but one in particular will likely be crucial for
children with cancer in the coming years.
“The immunological Rosetta Stone is that the body
stops recognizing cancer cells,” says Dr. Ambruso.
“These machines are going to be instrumental
in gathering T cells to be modified into chimeric
antigen receptors (see “A Living Therapy,” next
page) — but you have to optimize them to do it.”
The apheresis team will not only help with that
optimization process, but also, through a research
donor program, will deliver the blood products
Terumo needs to test it.
“This partnership helps us and it helps them,”
Dr. Ambruso says. “It’s a wonderful collaboration.”
New Agents in Neuroblastoma Treatment “These machines are going to be instrumental
in gathering T cells to be modified into chimeric
antigen receptors.”
Pediatric Bone Marrow Transplant Consortium D A N I E L A M B R U S O, M D
Pediatric Brain Tumor Consortium Associate Medical Director, Belle Bonfils Memorial Blood Center
Children’s Oncology Group
Children’s Oncology Group Phase I Consortium
CONNECT Brain Tumor Consortium
Dana Farber Leukemia Consortium
Pediatric Cancer Immunotherapy Network
Neurofibromatosis Consortium
The Culture of Different
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