Page 8 The Skanner September 20, 2017
News
NHLBI: Exploring Research Directions in Hope of a Widely
Available Cure for Sickle Cell Disease
pain management, and
improve bone marrow
transplant procedures
to increase the body’s ac-
ceptance of donor stem
cells.
One promising strat-
egy that is being stud-
ied in a clinical trial – a
study that tests how well
the United States don’t
have a relative who is a
full genetic match and
able to be a donor.
The National Heart,
Lung, and Blood Insti-
tute (NHLBI), a leader
of federally funded re-
search efforts on sickle
cell disease, is actively
“
Bone marrow transplants
can eliminate sickle cell dis-
ease, ridding them of pain-
ful and debilitating symp-
toms, and the need for a
lifetime of pain medications
and blood transfusions
PHOTO COURTESY OF ED UTHMAN VIA FLICKR
exploring several safe
and effective treatment
options, and leading sev-
eral initiatives designed
Peripheral blood smear of sickle cell disease
cells that do not sickle as
do the cells in those with
sickle cell disease—the
characteristic that gives
the disease its name. Un-
fortunately, many people
with sickle cell disease in
to find a widely avail-
able cure for sickle cell
disease. The institute
supports many research
efforts that yield new
therapies, optimize cur-
rent treatments, enhance
a new medical approach
works in people – is
half-match bone marrow
transplants. Bone mar-
row transplants can
eliminate sickle cell dis-
ease, ridding them of
painful and debilitating
symptoms, and the need
for a lifetime of pain
medications and blood
transfusions. This half-
match approach could
make bone marrow
transplants accessible to
more people living with
sickle cell disease.
The NHLBI also com-
pleted the Transcranial
Doppler with Transfu-
sions Changing to Hy-
droxyurea
(TWiTCH)
trial. Based on earlier
research that was fund-
ed by NHLBI, regular
blood transfusions have
become the standard of
care for reducing the risk
DIANA GRIB VIA WIKICOMMONS
S
eptember is not just
“ b a c k - t o - s c h o ol ”
month; it’s National
Sickle Cell Aware-
ness Month—a month
selected to call attention
to a life-long illness af-
fecting around 100,000
Americans, the majori-
ty of whom are African
American or Hispanic.
Sickle cell disease is
accompanied by severe
pain attacks and poor ox-
ygen delivery through-
out the body that can
cause critical damage to
organs. Currently, the
only cure is hematopoi-
etic stem cell transplan-
tation (HSCT)—a costly
procedure in which stem
cells are taken from the
bone marrow or blood of
a healthy donor, and then
injected into the recipi-
ent to generate red blood
of stroke in children with
sickle cell disease. The
TWiTCH study found
that daily treatment with
hydroxyurea— an oral
medicine to help reduce
or prevent several com-
plications of sickle cell
disease – is as effective
as blood transfusions
at reducing blood flow
velocities in the brain, a
key risk factor for stroke.
NHLBI is committed to
additional studies that
can prevent strokes and
other sickle cell-related
complications.
Another area of prom-
ise for sickle cell dis-
ease treatment is gene
editing, which involves
changing the DNA. An
NHLBI research group
recently showed that
correcting the sickle mu-
tation in hematopoietic
A career you can
be proud of.
stem cells from patients
with sickle cell disease,
and then transplanting
them in mice, resulted
in enough normal hemo-
globin to have a potential
benefit. Another recent
study in a patient showed
that replacing the sickle
mutation by gene inser-
tion (gene therapy) re-
sulted in complete clin-
ical remission of sickle
cell disease. However,
longer follow-up in more
patients is required to
confirm the long-term
safety and effectiveness
of gene therapy for sickle
cell disease.
NHLBI is also conduct-
ing early research using
small molecule drugs. In
one recent NHLBI-fund-
ed study, researchers
found a small molecule
that binds to hemoglobin
and increases its ability
to bind to oxygen; this
could reduce sickling
of red blood cells. Since
small molecules can be
easily
administered,
the hope is that this ap-
proach could become
a cost-effective, widely
available treatment for
sickle cell disease both
in developed and devel-
oping countries. Howev-
er, this approach has not
been tested in humans
yet.
Over the next decade,
NHLBI is committed to
conducting and funding
innovative research on
sickle cell disease. Sickle
cell patients who partic-
ipate in research studies
are critical partners in
discovering
potential
therapies and new ap-
proaches that improve
our understanding of
sickle cell disease. Al-
though new preventive
and treatment strategies
might take years to devel-
op, patients should take
heart that today there are
effective treatments that
can help reduce symp-
toms and prolong life.
To find out more about
how you or a loved one
can participate in a clini-
cal trial, go to clinicaltri-
als.gov and search sickle
cell disease.
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