researchers are already taking steps
to educate patients about the technology. The National Institutes of Health is
launching a study this month to examine
opinions on CRISPR among up to 150
sickle-cell patients, parents of patients,
and health-care providers.
Vence Bonham, a researcher on genomics and health disparities at the National
Human Genome Research Institute who is
leading the study, says it’s important that
scientists designing clinical trials consider
patients’ beliefs and concerns. Gauging the
views of people who are most likely to be
affected by a new scientific advance seems
like a no-brainer, but it’s something that’s
rarely been done in medical research. “This
technology has been moving very quickly,
but the disease and advocacy communities
have not really been part of the conversation,” Bonham says.
Participants in the NIH study will
first be asked about their knowledge of
CRISPR. Then they’ll watch an educa-
tional video about the technology and
answer a second set of questions to see
how the video may have influenced their
knowledge or beliefs. After that, they’ll
participate in focus groups with other
patients, parents of patients, or health-
care providers to talk about using CRISPR
for sickle-cell disease. Bonham hopes the
study will “inform the development of
clinical trials to make them more appro-
priate and respectful of the concerns of
Biree Andemariam, director of the
New England Sickle Cell Institute at the
University of Connecticut Health Cen-
ter, has talked to her adult patients about
CRISPR’s potential for treating sickle-cell
disease. “Patients are very intrigued by it.
They think it sounds wonderful,” she says.
But Andemariam says there can also
be trust issues between sickle-cell patients
and their health-care providers. Black
patients may be suspicious of signing up
for clinical trials, particularly given his-
torical examples of medical experimenta-
tion on African-Americans without their
consent. The infamous Tuskegee study,
for example, left African-American men
with syphilis deliberately untreated in an
experiment that ran from 1932 to 1972.
“The Tuskegee experiment is fresh in
a lot of people’s minds even though that
was decades ago,” says Andemariam, who
is also chief medical officer for the Sickle
Cell Disease Association of America.
If a CRISPR cure for sickle-cell even-
tually reaches the market, one major
question is who will have access to it. Isaac
Odame, a Ghana-born physician at the
Hospital for Sick Children in Toronto,
says patients in Africa already have trou-
ble paying for hydroxyurea, a common
medication used to treat the disease. The
drug costs one to two dollars per day, but
even that is too expensive for many, he
says. He worries the cost of CRISPR will
put a cure out of reach for most of the
“For 90 percent of people with sickle-cell disease who live in this world, this will
still be something far too expensive for
them to have access to,” he says.
Until CRISPR is available, sickle-cell
patients will have to cope with other treatments. To manage his disease, Nazaire
recently underwent apheresis, a transfusion procedure that removed and replaced
some of his red blood cells in an attempt
to decrease the proportion of sickled ones.
He’s in less pain than before, but the benefits could wear off over time.
To Nazaire and others, CRISPR represents the promise of a better, longer life.
“When you’re faced with something that’s
desperate and life-threatening, you want
to see something done about it,” he says.
“I think this is something that needs to be
used. It could be beneficial for the world.”
Nazaire’s sickle-cell-inspired paintings.