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0:00
I'm Dr. Sophie Lanzkron,
I am a Professor of Medicine,
and the director of
the division of
Classical Hematology at
the Sidney Kimmel
Medical College at
Thomas Jefferson University.
Today, I'm going to talk about
a comprehensive overview of
sickle cell disease in
the era of gene therapy.
0:21
I'm going to describe
the pathophysiology of
vaso-oclusive events in
sickle cell disease.
Apply some
guideline-based care to
adults with sickle cell disease,
and talk a little bit, at
the end, about gene therapy—
the promise of gene therapy and
the risks associated
with gene therapy.
0:38
I like to start my
talks with this quote.
This is from the New England
Journal of Medicine,
from our colleagues
Power-Hays and McGann.
"There may be no population of
patients whose health
care and outcomes are
more effected by racism than
those with sickle cell disease."
I think that's really true,
and I think that there is data
that supports that this is true.
0:60
On this slide, we did
a study comparing both
NIH (National Institutes of
Health) federal funding for
research for sickle
cell disease and
foundation funding for
sickle cell disease,
and compared that same
funding to cystic fibrosis.
You look, there are about
90 to 100,000 people in
the United States with
sickle cell disease and about
30,000 with cystic fibrosis.
You look at the amount of
NIH funding for both
of these diseases:
about $76 million for
sickle cell disease and
$84 million for cystic fibrosis,
which doesn't sound like
it's terribly different.
But when you think about
the funding per person with the
disease living in the United States,
it's quite remarkable.
They is about $812
per person spent for
research on people with
sickle cell disease,
and almost $3000 per person for
people with cystic fibrosis.
That disparity looks
more stark when
you look at foundation funding.
The Cystic Fibrosis
Foundation has about
$230 million to spend each year,
and the Sickle Cell
Disease Association and
other foundations raise
about $10 million each year.
That equates to about
$102 for people with
sickle cell disease,
and almost $8000 per person for
people with cystic fibrosis.
To think that these
differences in
federal funding for research and
foundation funding for resources
don't make a difference,
I think is unrealistic.
We know that research dollars,
especially federal ones
really make a difference in
the development
of new therapies,
which we desperately need for
people with sickle cell disease.
Foundation funding
can be used for
many things to
support patients who
often live and struggle with
social determinants of health,
providing transportation
to doctors' appointments,
and, additionally,
many other services,
including community
health workers.
Even for the Cystic
Fibrosis Foundation,
they have funded fellows
in pulmonary to become
cystic fibrosis experts.
We know that in
sickle cell disease,
there really aren't enough
experts in the disease,
so this funding disparity
makes a huge difference.
