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NHLBI: Exploring Research Directions In Hope Of A Widely Available Cure For Sickle Cell Disease
By NAPS,
North American Precis Syndicate
. New York
,
NY
.(NAPSI)—September is not just “back-to-school” month;
it’s National Sickle Cell Awareness Month—a month selected to
call attention to a life-long illness affecting around 100,000 Americans, the
majority of whom are African American or Hispanic.
Sickle cell disease is accompanied by severe pain attacks and poor oxygen
delivery throughout the body that can cause critical damage to organs.
Currently, the only cure is hematopoietic stem cell transplantation (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 recipient to generate red
blood cells that do not sickle as do the cells in those with sickle cell
disease—the characteristic that gives the disease its name.
Unfortunately, many people with sickle cell disease in 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 Institute (NHLBI), a leader of
federally funded research efforts on sickle cell disease, is actively
exploring several safe and effective treatment options, and leading several
initiatives designed to find a widely available cure for sickle cell disease.
The institute supports many research efforts that yield new therapies,
optimize current treatments, enhance pain management, and improve bone marrow
transplant procedures to increase the body’s acceptance of donor stem
cells.
Image
credit: Laurel Mendelsohn, Emilia Barbu, NHLBI
Mixture of normal and sickle-shaped red blood cells taken from a
blood sample of a person with sickle cell disease, as viewed through a
fluorescent microscope.
One promising strategy that is being studied in a clinical trial—a
study that tests how well a new medical approach works in people—is
half-match bone marrow transplants. Bone marrow transplants can eliminate
sickle cell disease, 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 completed the Transcranial Doppler with Transfusions
Changing to Hydroxyurea (TWiTCH) trial. Based on earlier research that was
funded by NHLBI, regular blood transfusions have become the standard of care
for reducing the risk 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 complications 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 promise for sickle cell disease treatment is gene editing,
which involves changing the DNA. An NHLBI research group recently showed that
correcting the sickle mutation in hematopoietic stem cells from patients with
sickle cell disease, and then transplanting them in mice, resulted in enough
normal hemoglobin to have a potential benefit. Another recent study in a
patient showed that replacing the sickle mutation by gene insertion (gene
therapy) resulted in complete clinical 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 conducting early research using small molecule drugs. In one
recent NHLBI-funded 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 approach could become a cost-effective,
widely available treatment for sickle cell disease both in developed and
developing countries. However, 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
participate in research studies are critical partners in discovering
potential therapies and new approaches that improve our understanding of
sickle cell disease. Although new preventive and treatment strategies might
take years to develop, patients should take heart that today there are
effective treatments that can help reduce symptoms and prolong life.
To find out more about how you or a loved one can participate in a
clinical trial, go to clinicaltrials.gov and search sickle cell disease.
On the Net:North American Precis Syndicate, Inc.(NAPSI)
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