ASH News Daily 2017 - Issue 4 - A-3

Tuesday, December 12, 2017

ASH News Daily

Page A-3
®

GLOBAL PERSPECTIVES

Here, There, and Everywhere
By lynne lederMan, Phd

o you know how challenging it is to practice hematology in a low-resource
setting? During Monday's Global
Hematology Symposium, we heard
about these challenges and about
ways to address them. Dr. Kwaku
Ohene-Frempong described his
work with public and private sectors in Ghana to build comprehensive screening and early intervention for children with sickle cell
disease (SCD).
Early identification of SCD can
improve outcomes by allowing preventive measures like prophylactic
antibiotics to be instituted. Newborn screening programs for SCD
were initiated in New York in 1975
and recommended across the United States in 1987, but only finally
implemented in May 2006. Brazil,
Canada, and some Middle Eastern,
Caribbean, and Western European
countries now screen for SCD as
part of their newborn screening
panels. However, no Sub-Saharan
African countries have established
any universal, public health-based
newborn screening programs.

The National Newborn Screening Programme for SCD in Ghana,
one of a few pilot projects in Africa,
began as a demonstration project in
1993 funded by the National Institutes of the Health National Lung,
Heart, and Blood Institute, adopted
by the Ghanaian Ministry of Health
in 2008 with continuing support
from Brazil, and launched nationally in 2010, free of charge.
Health care for children younger
than five years of age is theoretically free in Ghana, but insufficient
funding has slowed scale-up of the
screening program. Currently, only
about 4 percent of babies born in
Ghana are screened; of these, 1.78
percent have SCD. Of the more
than 8,500 babies screened, about
6400 have been enrolled for care at
the Kumasi Centre for Blood and
Sickle Cell Disease at the Komfo
Anokye Teaching Hospital.
Preparations by the Sickle Cell
Foundation of Ghana for the national scale-up of newborn SCD screening include building SCD treatment
centers across the country and educating and training clinicians, public and community health nurses,
genetic counselors, and laboratory

staff. "Guidelines for Management
of Sickle Cell Disease in Ghana" are
being developed, and a mobile app
is improving data collection.
Session Chair Dr. Leslie Kean
described how ASH members can
volunteer through a variety of opportunities including the ASH partnership with Health Volunteers
Overseas (HVO), which focuses
on adult hematology and oncology in Uganda, Tanzania, and Peru,
and on pediatric hematology and
oncology in Cambodia. Activities
include training courses for local
personnel and ongoing case conferences. For example, the program
has helped institute treatment for
chronic myeloid leukemia in Tanzania and chemotherapy for pediatric
patients in Cambodia. These programs also provide an opportunity
for hematologists and oncologists
in the United States to learn about
health care in other countries.
Volunteers are asked to commit
to a minimum two-week service
time in-country, participate in the
ongoing case conferences, and return for periodic in-country visits.
Dr. Kean said, "Of all the things I
have done in my career, this is abso-

Dr. Kwaku Ohene-Frempong
described his work with
public and private sectors
in Ghana to build
comprehensive screening and
early intervention for children
with SCD. Early identification
of SCD can improve outcomes
by allowing preventive
measures like prophylactic
antibiotics to be instituted.

lutely the most rewarding and enriching for me." She added, "I think
it is one of the most fun things that
I have done, to really see progress
being made and to be able to collaborate on that."
If you are interested in the ASH
HVO program, visit http://www.
hematology.org/Global/204.aspx. Volunteer opportunities for pediatric
or adult hematology/oncology clinicians, laboratory scientists, and
academicians are available.
Dr. Lederman indicated no relevant
conflicts of interest.

EMERGING THERAPIES

And That's the Recipe for Makin' Drugs...
By nina Shah, Md

onday afternoon's Featured Topic Session "Combination Therapies - How
Are We Deciding What to Utilize?"
confirmed the challenges of explosive growth in drug development.
In a rapid-fire but comprehensive
presentation, Dr. Steven Grant of
Virginia Commonwealth University and Dr. Donna Przepiorka of
the U.S. Food and Drug Administration took on the daunting task of
contextualizing the numerous drug
combinations presented throughout the meeting.
The presenters focused on different classes of drugs. Dr. Grant spoke
about novel targeted agents and
small molecule inhibitors, while Dr.
Przepiorka focused on immuneoncology. Amid the sea of abstracts,
the session chairs were able to select
several key studies that exemplified
evolving principles of combination
therapy in our current era.
"One of the main purposes of
this session is to draw attention to
interesting findings involving rationale combinations in various
disease entities," said Dr. Grant.
Indeed, his presentation outlined

Dr. Donna Przepiorka presents during
Monday's Featured Topic session.

how novel agents have been intelligently combined with classical cytotoxic agents or repurposed to an
alternate disease based on sound
preclinical data. He also discussed
trials that examine rational combinations of novel, unapproved
agents with a possible pathway toward multiple approvals.
Dr. Przepiorka echoed this sentiment in the context of immuno-

therapy and took it one step further,
commenting, "What is the basic science coming out that's suggestive
of something new that would work
in combination? And what are the
potential areas of controversy?
People are thinking that it might be
a good combination based on the
abstract but then when you look at
them in greater detail, are we really
over-reading them?" She discussed
recent findings with antibody-drug
conjugates, PD-1 inhibitors, and bispecific antibodies as the anchors of
combination immunotherapy.
In a unique twist, this session appealed to both scientists and clinicians. Though much of the presented
data had emerged from basic scientific investigation, the road to clinical translation was highlighted by
the speakers. As Dr. Przepiorka described, "We wanted to make this
something that is applicable to all the
ASH attendees: for the basic scientists
thinking about ideas for studies, and
also for people in practice ... why
some things that people think might
go together probably shouldn't."
Dr. Grant summarized the concept of rational combination therapy
and stated that, "In terms of predicting how do we go forward? That's

a tall order." He also speculated on
what we can learn from the session,
"At the preclinical level, it's obviously hard to predict which one of
these concepts is going to be successful ... I think one of the more promising approaches is to take agents
that are already effective and then
rationally disable bypass pathways
or resistance-conferring pathways."
Though the myriad drugs are
a more recent phenomenon, the
concept of combination therapy
is not new. As Dr. Przepiorka
pointed out, "We're going to have
to put things into context in order
to get people to see where we're
coming from (especially with the
new basic science information)
and where we want them to go."
Perhaps with the experience from
the past and the innovation of
the present, there is hope for this
multifaceted approach, yet uncertainty remains. "I won't pretend
to be able to predict the right approach," said Dr. Grant, "but if I
knew which approach would be
successful, I'd be buying my ticket
to Stockholm!"
Dr. Shah indicated no relevant conflicts of interest.

http://www.hematology.org/Global/204.aspx http://www.hematology.org/Global/204.aspx

Table of Contents for the Digital Edition of ASH News Daily 2017 - Issue 4

ASH News Daily 2017 - Issue 4 - A-1
ASH News Daily 2017 - Issue 4 - A-2
ASH News Daily 2017 - Issue 4 - A-3
ASH News Daily 2017 - Issue 4 - A-4
ASH News Daily 2017 - Issue 4 - A-5
ASH News Daily 2017 - Issue 4 - A-6
ASH News Daily 2017 - Issue 4 - A-7
ASH News Daily 2017 - Issue 4 - A-8
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