So, good afternoon. I am very pleased to
be here at IGI to have an opportunity
to have a conversation with you about some
of the work that I'm doing and to learn
about all the exciting things that are
happening here at this Institute. My
meetings this morning really
were so enjoyable there just kind of
hear both about common interests that
individuals here the Institute have with
regards to sickle cell disease as well
as some of the exciting things and the
ways you're pushing forward this issue
of translation of gene editing into
clinical care. So, I want to have a
conversation that's focused on this
frame about searching for a cure and
gene editing in sickle cell disease. What
I will do is I'm gonna just take a
minute and talk about what I described
is the hundred and ten year legacy of
this disease and why this legacy is
important as you do your research as
researchers here to understand that
history and to put your research in
context of that history and the
potential future related to this disease
and then frame around gene editing
horizon that actually you can give that
talk probably much better than I can but
for us to talk about this issue of gene
editing on the horizon. But then I
want to really spend my time with you
this afternoon to talk about issues with
regards to fairness and respect and
transnational cooperation related to
gene editing and identify three areas
that I think are important for that
conversation. So, sickle cell disease as
probably everybody in this room knows is
a disease of the blood a blood disorder
disease of a single change in mutation
of the beta-globin gene, but the
complications that arise from this
disease
are really varied based on an individual
and I think that's an important context
to understand about this disease if
you've seen one individual living with
sickle cell disease you've seen one
individual living with sickle cell
disease because the phenotypic variation
of the disease is quite varied where
some individuals have very severe
problems and a very short life and
others live a much higher quality of
life in a much longer life. But some of
the common phenotypic complications
include strokes, leg ulcers, priapism, as
well as, in organ damage that
significantly impacts the lives of
individuals living with this disease. So,
recognizing the different genotypes with
sickle cell disease and recognizing the
variation with regards to severity of
the disease I think is an important
part of this conversation when we talk
about curative genetic therapies like
gene editing and who is the appropriate
individuals to actually participate in
these clinical trials and the ultimate
ability to have this treatment in
clinical care. But to put this in history
we need to talk about the history of
sickle cell disease and so I like to
start in 1910 with Dr. James Herrick but
to take a minute and say this is a
disease that was known in sub-Saharan
Africa for hundreds of years before
James Herrick, but James Herrick in
Chicago, Illinois was the first
individual to identify and describe
sickle cell disease. James Herrick
and his resident, Ernest Irons, had an
opportunity to care for a patient and the
patient's name was Walter Clement Noel
and Walter Clement Noel was a dental
student in Chicago who went to Dr.
Herrick for treatment and at the time of
going with pain episodes and with
chronic inability to do his daily life and
quality of his life was not good
but he was a dental student and I want to
highlight that, the first sickle cell
disease
identified individual was a dental
student in Chicago when he was diagnosed.
So, I put some context to think about
this disease and the history of this
disease that in 1910 this young man from
a Caribbean island from Grenada had
sickle cell disease and had lived a long
life, was a dental student when it was
first diagnosed. And to put that in
context to think about this disease in
our situation today in 2020 and looking
for a cure so the story of Walter
Clement Noel is this: he came to dental
school, he was identified by James
Herrick, they took a blood smear and they
identified these sickle cells under the
blood smear, and two years later Dr.
Herrick reported this case study but Mr.
Noel continued to be treated by Dr.
Herrick and then returned to his Island
in Grenada and lived for a number of
more years as a dentist on that island.
So, this story tells us a couple things
about this recognition of this disease
and this history of this disease of
both that the disease should not just be
seen as a disease of individuals with
severe complications that are not able
to be educated, not be able to live a
full life, and not be able to do things
that they want but also disease with
these complications that individuals
have the resilience to continue to do
things to make sure that they have a
very high quality of life. So, the story
of Walter Clement Noel there's a number
of papers that have been published about
this particularly by Dr. Todd Savitt
that I would encourage you if you're
interested in knowing the history and
the beginning of sickle cell disease
within the European and American medical
literature.
So, let's talk about the last hundred
years and a couple things that happened
so 1949 is a major milestone when we
talk about sickle cell disease and Linus
Pauling so that year published in
science was a commentary that stated
that sickle cell disease was the first
molecular disease so line is calling
played a really major role with regards
to our understanding of the molecular
basis of sickle cell disease but also
was important regarding an ethical
question so a couple years later our
Nobel laureate Linus Pauling said that
we should put a tattoo on the forehead
of all individuals with sickle cell
disease so that they would not marry
someone and then ultimately have someone
a child with the disease so both he had
this great scientific basic background
and understanding of the molecular basis
of the disease but raised a really
interesting ethical question so how do
we think about this how do we explore
this of this great scientist but also
had these ethical laughs that I would
identify related to sickle cell disease
so nineteen ninety eight years after
sickle cell disease was first identified
the first fda-approved drug for sickle
cell disease was approved by the FDA
Hydrox areaa hydroxyurea as many of you
may know was actually a cancer treatment
but it increased fetal hemoglobin of
individuals living with the disease and
Heydrich shariah has been the primary
drug for sickle cell disease for the
history of this disease in our
recognition of its disease it is
currently used with starting with
infants through adulthood to increase
fetal hemoglobin today but it continues
not to be able to be a benefit to
everyone not everyone actually has their
fetal hemoglobin increased with the use
of hydroxyurea and it has various
complications that
some individuals living with the disease
not to want to use this treatment so
this third side I don't know if you can
see because it's the the screen is not
that great is a paper that was published
by dr. Sophie Lance cron and dr. Carlton
Heywood in 2005 and it was a paper that
reported the age and what related to
mortality and identified at that time
that the average age and mortality for a
male is 43 and for a woman was 45 but it
also showed in this study that as the
actual level of life increased for
children the mortality rate was
decreasing meaning that people were
dying at an earlier age with sickle-cell
disease and that they focused this on
really related to health care
experiences that individuals were having
and barriers with regards to access to
quality healthcare so this tension of as
individuals grew older they were
actually dying because of barriers with
regards to quality access to good care
and raise the question about how do we
grapple with this issue with regards to
actual health services as we also move
forward for additional treatments and
then in 2009 it's a major work with
regard to bone marrow transplant we're
taking place in sickle cell disease and
really the first curative therapies were
being developed with regards to bone
marrow transplant and then in 2017 I
would argue there was a major shift that
has occurred with this disease so first
in 2017 there was a second drug approved
by the FDA for use with sickle cell
disease and diary and then in 2019
several things happened that I think
have been a great milestone for the
future of sickle cell disease there were
two new drugs
that were approved by the FDA in 2019
but then in 2019 there was the
announcement of the first case of gene
editing CRISPR gene editing of an
individual with sickle-cell disease was
announced in 2019 so we've gone through
these last 110 years with regards to
sickle cell disease a disease that is a
disease of discrimination of individuals
going to the emergency room and being
perceived to be drug seeking and not
getting appropriate care a disease where
historically was not receiving the
research support for the number of
individuals with this rare genetic
disease and a disease that has been
racialized in the United States setting
and we now are here today in 2020 with
sickle cell disease gene editing on the
horizon and the great work that's going
on here at this Institute and in other
institutions across this country and
across the world so what does this mean
and how do we think about this with
regards to the future of this disease
and what are some important issues for
us to think about so I just share with
you a few articles that have come out in
the last four years including by some of
the investigators in the room here today
and the work that's been done here will
be at the Institute but if you think
about these articles and that all of
these articles have come out in the last
four years and where we are today with
regards to sickle cell disease and the
potential that we have going forward and
I think that's the setting that we have
to look at and as you know there are
really two major approaches that are
currently focused on with regards to
gene editing and particularly focused on
CRISPR gene editing one is modification
bcl11a gene to increase fetal hemoglobin
and the other is to modify the beta
globin gene to actually to modify the
mutation and the work that's happening
here and happening
other institutions that are so I want to
tell you a little about some of the
research that's going on in my research
group at the National Human Genome
Research Institute I'm an intramural
researcher in our social and behavioral
research branch and within that branch
as was stated I lead our health
disparities unit and I see sickle cell
disease as a health disparity disease a
genetic despite a disease and with the
horizon of gene editing and other
curative genetic therapies I became
really interested in wanting to have
conversations with the actual community
I was at a American Society of
Hematology meeting back in 2015 and it
was very clear that this was on the
horizon but all the conversation was
about the science and none of it was
about what was the perspective
potentially of communities with regards
to this potential new treatment and so I
became very interested in wanting to
move forward a project to actually
listen to individuals in the community
and get their views so we spent 2017 and
2018 in the field collecting data from
adults living with sickle cell disease
from parents with a child with sickle
cell disease and from health care
providers all hematologists who care for
individuals living with sickle cell
disease and we looked at that
information to help share and inform
what the perspectives were of the
community with regards to the potential
of these clinical trials coming to the
clinic and a willingness of individuals
to participate in these trials so this
was before the first fda-approved
clinical trial for gene editing was
actually approved by the FDA for a
CRISPR gene editing trial so hopefully
my work has helped to inform some of the
efforts that are currently going on and
hopefully the work that we're doing can
also move forward to help as we continue
down this road to develop these new
treatments so it
with this study we conducted 15 focus
groups with adults living with disease
parents of a child and their providers
we used some standard measures that had
been used by the pew with regards to
gene editing and we wanted to share our
views of the sickle cell disease
community with the general public and
with the general public of African
Americans to look at differences we
conducted three pilot focus groups to
help modify our instrument and our
survey guide as well as our focus group
guide and then we launched our study to
go into the community and to gather this
information and now we've done various
analyses of this work and have published
two papers and have a third paper that
hopefully will be published soon from
that work so I want to share with you
some of the voices from the individuals
that participated in our focus group
study and kind of put a frame with
regards to why I think your work is so
important but also that we must struggle
with what I argue are some important
ethical and social questions with
regards to the integration of gene
editing into clinical care so let me
just start with what I would say is the
take-home message from our study the
take-home message was that there was
overall great optimism about the
potential of gene editing and other
curative genetic therapies for
sickle-cell disease so a couple quotes
it's exciting it's what I've been
waiting for one patient said another set
I'm very optimistic
it's another possible option for sickle
cell patients and unfortunately we don't
have many so this was the message that I
want to share with you today that this
community recognizing 110 year legacy of
discrimination of racism of lack of
CAIR is optimistic about the potential
of gene editing and other curative
therapies for sickle-cell disease that
is the take-home message from our study
and the work that we've conducted but
you must put that in context with
regards to the broader issues of how do
we actually do this how we actually
integrate this treatment in the clinical
care
so in 2017 the National Academies of
science published a report on human
genome editing which I actually think
they did a really great job and I think
that this is going to actually be a
foundation with regards to the
principles that we should be having a
conversation as we go forward because I
think that the principles that they laid
out which are the seven principles that
they think are important for human
genome editing are going to be important
part of our conversation as we move
forward the research and move forward
the clinical care
so promoting well-being transparency do
care
responsible science respect for person
fairness and transnational cooperation
and I want to just highlight a couple of
those principles around some issues that
I think that we're gonna have an
obligation and a responsibility to
struggle with so the first one is
related to this issue of providing
quality information to individuals and
the question how do we ensure disease
communities have access to quality
accessible and scientifically accurate
information I think this is a
fundamental question that the field the
researchers the clinicians as well as
the patient community are going to have
to struggle with and I think that
there's some really exciting
opportunities to collaborate on this
question not just for sickle cell
disease but use sickle cell disease as a
model for other diseases where gene
editing will be important
because this question about how do you
make sure people have information to
make an informed decision is essential
to this conversation and I know this
Institute is funding individual
investigators and thinking about this
issue it's thinking about it as a broad
education effort but this question about
informed information so a couple quotes
from our study I'm not clear on how
exactly it works is it something that
they do in a thing and then they put it
into your body how does that work
do they inject it in your bone marrow in
your blood how does the CRISPR get on
your DNA chain to make the changes one
adult said so this question about how
does this actually work to help me make
a decision whether this is something
that's of interest to me
what does it actually do for them on a
daily basis well they have less pain
will it hot like renal failure or
progression plus any complications a
physician said as you see this is a very
well-educated group and it's not that we
don't understand or that we're resistant
to research but you need to explain in a
way that people will understand also be
a credible source one individual said so
this message of having high quality
scientific information is essential for
this community to help make decisions
whether this is a treatment that they're
interested in and without that
information that lack of that
information I argue that that is an
ethical lapse on the part of the
scientific community I want every risk
associated with it like even if it
didn't happen
I want the theoretical risk and what
might happen what may happen one adult
with sickle cell disease so this
question of understanding
the benefits the chances that something
will go wrong that potentially could
cause harm to that individual or to that
individuals child so what do people do
when they want information they type
into Google whatever they're looking for
right so one of the first things we all
do and we go and we find what's on the
web so my group my research group at the
National Human Genome Research Institute
it's actually looking at what is
currently on the internet about sickle
cell disease and gene therapy and gene
editing okay so we're looking broadly
and all cure to genetic therapies for
sickle cell disease to study actually
the quality and the accessibility of the
information it's on the web
so we're conducting a study but looking
at what's on the web and then providing
an assessment of that material we are
not including things that are podcasts
or news stories about individual
patients or news articles about
individual patients but we are including
anything that would be described using
the criteria that we developed as
patient education materials so we're
particularly focused on this question of
if I'm a patient or if I'm a parent and
I'm looking for information to help me
better understand this protocol this
treatment this science what would I find
on the web so when we find these
materials on the web we are using an arc
the agency for Healthcare Research and
quality assessment tool called the
patient education materials assessment
tool or Pima to actually to score each
of these individual documents that we
find on the web so we're going through
in a standardized way
with two reviewers taking each document
independently and coding and scoring the
document and then comparing their their
findings and then having a third person
if they disagreed make a final judgment
with regards to the quality of the
material following this arc guideline so
we're looking at understandability we're
looking at actionability what action can
you take relate to the information we're
looking at scientific accuracy a
presentation of risk and benefits and
we're looking at readability and we're
using tool tools of the flesh Kincaid
grade level and the smog to assess these
tools okay so we are now in the process
of doing this work you may have seen
some of these websites before that are
included we try to identify all of the
tools and then we had a librarian a
medical librarian separate from us to go
through to identify tools on the web so
we had an independent person also go
through to identify the tools that we
were able to find and so this is an
ongoing project in my research group but
the message that I want to share with
you is there's very few tools online and
the quality is variable with regards to
the tools that are there and I argue
that we are at a time now where we
really need as a community to come
together to make sure that there's very
high-quality information on the web
that's accessible to patients and
parents and providers that can help them
in being informed so this is before
getting to the stage of a clinical trial
to be informed about the science and the
risk and the benefits and the questions
that they need to ask when they are
talking to their physician or a
researcher about a clinical trial so the
second area that I want to talk about is
respect for a person
the principle of respect for person and
I think that this is a real time issue
that I think we all need to be thinking
about a related to the excitement of
these new therapies and the issue is the
story of the individuals who are first
in humans who are participating in these
clinical trial so the principle of
respect the first in human clinical
trial participants and I think this is
another area that we as researchers have
an obligation to be protective of
individuals who are first in human
clinical trials so this is a quote from
the Belmont report that I think is
timely with regards to this question
persons are treated in an ethical manner
not only by respecting their decision
and protecting them from harm but also
making efforts to secure their
well-being so think about that the
well-being of individual so this is not
a health risk this is an issue of
thinking about someone and their
well-being and how the story of their
participation as a first inhuman
participant can have an impact on their
well-being so I just use a couple
examples here of stories that we've all
seen over the last year and these are
stories that are exciting stories
they're exciting stories of individuals
who have had great positive outcomes
today
but think about what could go wrong
think about what this could mean with
regards to discrimination what this
could mean with regards to
misinformation what this can mean to
them to their families and how do we
think about this from a well-being
perspective I became really interested
in this because I had several reporters
over this last year
call me at several times throughout the
year to ask me to respond to a story
that they were about to publish and to
talk about a participant in a trial and
I would always say no I can't talk I'm
happy to talk to you but I can't talk
about an individual in a trial one I
don't know this individual - I'm not
part of that trial but is that right
and so I think we have to struggle as we
move forward with our enthusiasm about
the science to be protective of
individuals who are leading the front
edge of moving this science into
clinical care and what do we do with
regards to how we deal with identity and
privacy protection of an individual and
the well-being of an individual who has
participated in one of these studies
have basically made a commitment to be
part of research and made a commitment
to help lead the science forward on how
do we protect their well-being so this
is an article that I would encourage to
all of you who have an interest in this
area they just recently came out in the
Hastings Center report by author Frank
and he has his article is not focused on
the media and the stories
it's really focus much more on narrative
medicine and and issues that we need to
be thinking about but I think it applies
directly to this issue that I'm raising
here and this issue of privilege in the
briefest terms we need to understand how
to tell respectful stories in which the
characters are fully acknowledged fellow
participants not one-dimensional object
of a knowing gaze the problem is
narration itself the problems particular
version of narration 'el privilege and I
think we have an obligation in our work
to be careful and thoughtful with
regards to not putting individual study
participants in a place where they
perceive to have their privileged their
privilege taken away and his article
talks about people need to tell stories
about other people
people tell multiple kinds of stories
and different listeners or readers and
different media how people feel injured
or you searched or diminished by stories
told about themselves is a complex issue
and if people are put at risk by having
stories told about them they can also
gain comfort from having their stories
told so I'm not saying that we should
not have media part of this discourse
about the excitement and what is
happening in this research but then we
be very careful and very thoughtful of
how we tell someone else's story so how
do we equity engage the public and share
this science while also respecting the
first in human participants so the last
area I want to focus on is with regards
just global justice and equity and
curative therapies and I too here think
that the academies got it right and I
think that there's two principles that
are clearly highlighted here one is fair
and transnational cooperation and I
think this is where there is both
exciting work going on but I think where
we all need to be humbled as we think
about a disease where the burden of the
disease is not in the United States it's
not in Italy it's not in Canada is not
in Belgium it's not where these trials
are going on it's in sub-saharan Africa
and India that is where the burden of
this disease is and that we must always
recognize that as we move forward our
work and our research to recognize that
the burden of individuals that are
living with sickle cell disease it's not
here and how do we hope and help and
guide our work to actually make an
impact in these areas of the world where
millions of people are living with this
disease versus a hundred thousand and
how do we do that in a way that is both
cost effective and appropriate but it's
accessible to individuals that live in
these countries that are far away from
San Francisco Berkeley California so
there are exciting things happening in
this area as many of you know and ask
you some help with the Gates Foundation
or collaborating on a new major
initiative to actually focus on new
approaches that will hopefully reach
more individuals and it will be
accessible to more individuals with
regards to gene editing and curative
therapies and I am really excited to see
this work I'm excited to have the
conversation this morning about the
things that are happening here to help
to move that work forward but I think we
also must just be very humble about the
potential of this and how do we actually
recognize that this type of a treatment
is not available today but today also
what's not available is the standard of
care for sickle-cell disease so when we
think about the standard of care today
is to start
a child out on penicillin making sure
that individuals held appropriate
vaccinations to put people in Hydrox
area and that the vast majority of
individuals living in sub-saharan Africa
and India do not have access to these
treatments today and as we think about
how we move forward to actually have
cure to genetic therapies now we also
must be thinking about how do we
actually expand access to standard of
care today so that we can move forward
so I want to share with you another
project that my group is starting and
that's a project that we're doing in
sub-saharan Africa in the context where
approximately half of individuals don't
live to age of five with sickle-cell
disease the vast majority of individuals
don't live to be adults but that there
are still millions of individuals who do
reach adulthood but sickle cell disease
so my group at the National Human Genome
Research Institute is starting a
collaboration in Sierra Leone and Sierra
Leone which is in West Africa is one of
the poorest countries in Africa it was
affected by Ebola
and it had a major war that occurred in
that country for a number of years but
it also has a very high prevalence level
of sickle cell disease so it
collaborators in Sierra Leone as well as
investigators here in the United States
from Sierra Leone we are starting a
project to actually take a study to see
early on around sickle cell disease in
adulthood to study individuals to
actually to increase the number of
individuals who are getting newborn
screening in Sierra Leone and to think
about these questions of curative
therapies in a country like Syria so
we're really excited to be doing this
work to think mostly about the ethical
questions and social questions but also
what is the current state of treatment
in Sierra Leone today and how can we
help increase that quality of treatment
and conduct research in that setting so
how do we ensure that these new
technologies are available to even the
most low resource or disadvantaged
populations and how do we bring current
standard of care to low resource and
disadvantaged population so I want to
frame this talk and frame this issue
around truth fairness what does that
mean fairness and how do we explore that
and how do we explore this principle
with regards to the exciting things that
are happening within the field of
gene-editing
and sickle cell disease and can we as we
move forward the technology and the
development of new treatments think
about the context of the principle of
fairness so I have two last quotes that
I want to share with you to have the
sickle cell population move this forward
and then not have available for them
equally would be extremely traumatic to
the community when physician said I
would say don't mess it up if you are
one really talking about it impacting
the sickle cell population you have to
be very careful that the other rare
diseases that have more resources don't
take it over and the sickle cell
population gets left in the dusts
because they've been left in the dust
with so many other things that there
already are skeptics a physician said so
this question of fairness is an area
that we must explore and in our
conversation our question and answers
today I would like to really talk about
fairness but before I do that I have to
acknowledge my research group so these
are the researchers the folks that work
with me in my research group at the
genome Institute I and
I want to highlight I always do this now
I'm out on the road training
opportunities at the National Human
Genome Research Institute I am hiring a
postdoc to come and think about these
social and ethical questions related to
gene editing and sickle cell disease if
you're interested you know reach out to
the our website we're looking for
individuals who want to come and spend a
couple years in Bethesda struggling with
some of these issues as well as I'm
looking for post backs who are also
interested in these issues that I always
bring to my lab so with that I would
like to say thank you and that I look
forward to our conversation the first
thank you very much and the story comes
to mind when shared the very clear
that's basically everything everyone so
my question is um how would you want to
effectively educate people about risks
at our very low probability without
having them fear of things that are very
unlikely yeah no I think that's a great
question so I think that's why it's so
important that we create really high
quality information that's available for
people and that there's conversations so
that people can debate what's actually
at higher risk and lower risk with
regards to going wrong as well as
potential impact on individuals lives
over their life time and that then that
gets communicated and you know one of
the things that's difficult is how do
you take information where you're
showing risk in the risk of very very
low but it's important that you share
that with individuals but I think that
you can do that in a way that you
separate out those things that are
higher risk potentially going wrong and
still showing that they're two very rare
that would ever occur and those things
that are really remote and that are
unlikely to ever happen and so it's it's
this is not easy but I think we can do
this we can create accessible
information that can distinguish things
that are higher risk that people just
need to recognize as they make decisions
and those things that are quite remote
but what I argue is if you know
something is a potential risk to hide it
would be wrong and so we don't want to
do that from an ethical perspective but
we clearly don't want to
create a feeling of fear when the
information the data does not support
that the chances of that risk occurring
will happen a question related to the
studies the resources their destinations
so as the lines between credible and
incredible sources become increasingly
blurred I'm kind of wondering how
organizations like the National
Institute of Health are altering their
methods to educate the public I mean
obviously there's a wealth of
information out there very credible
information debunking misinformation
about vaccines their effectiveness and
their safety but that hasn't done
anything to stop be the anti-vaxxer
movement so I guess you worry about
excluding things like blog posts Twitter
in these analysis of what's available to
people how yeah so this is my research
this is not the government doing this as
a researcher independent investigator in
the social behavioral research branch
these are my research questions so this
is not the Institute's educational
materials
that's first comment I want to make so I
think the challenge you always have to
kind of create a parameter around what
you're doing and we made a judgment that
if you were a parent and you were trying
to make a decision about this something
I want to learn more about for my
potentials for my child that one of the
first things you want is information
that would be considered health
educational information and so that's
why we decided that that was the scope
of this not saying that the blogs or
social media other kind of information
won't be of importance and the stories
of individual participants will not be
important but if you're going to assess
about the quality the scientific
accuracy and the quality I think
I argue you need to start there in that
information that has been created with
an expectation that individual patients
and their families would be looking at
that information for information to help
them understand the science and so
that's kind of how we made our decision
but I argue that we have to look grow
more broadly and then we need to come up
with more creative ways to provide
different avenues for people to get
information and that you know various
other approaches like videos like
podcasts will ultimately be extremely
important but if you didn't if you can't
get those things that are articulate as
patient health information correct then
I think we got a problem
and so that's where why we focused on
what do you think about the role
companies and insurance companies in so
I had some great conversations this
morning that this institute thinks about
the big picture
and I argue that researchers have an
obligation think about the big picture
and that it's not just the insurance
companies and the pharmaceutical
companies they could screw it up
actually if the researchers can screw it
up too and I think that by everybody
kind of coming together on certain
efforts and then conducting that
research to help develop models to
actually
use the chance of things going wrong is
an obligation for the researchers just
as is the obligation I argue for the
pharmaceutical companies and for those
individuals who are the investment
bankers and everybody involved if you
really want to move this forward so it's
integrated into clinical care that we
have to avoid some of the pitfalls that
have happened historically
and I think sickle cell disease you know
the potential is so exciting and we we
can see that this number of individuals
that could be impacted but we also know
this legacy right and so we want to make
sure we don't repeat that legacy with
regards to things happening that can
either push back the science for 20
years or create another level of
mistrust again my take-home message was
the community is optimistic they're
hopeful they're willing to participate
in these trials that was clearly the
message I got that's clearly the
evidence is being seen with regards to
the trials that are currently open so I
think I think this is a unique
opportunity for everybody to work
together in some ways to do some things
to actually make money to do great
science but to actually benefit people
and so so I push it back on the
researcher is how do you work with the
government how do you work with the
pharmaceutical companies how do you work
with you know the various groups that
are at the table to help to move things
forward and a epical and appropriate way
eight things for the talk so disease so
I'm curious what would you say are kind
of key success factors from translating
intention to
to enact that both on the clinical side
as well as the public engagement and
actually executing that effectively so I
think that's important to have these
kind of dialogues that you're having and
to bring people here and to have those
conversations but also go to people so I
think you know leave Berkeley and go out
across the country and engage with
people and having conversations and
engage with the various stakeholders
industry stakeholders also around these
issues and government stakeholders I
think that's an important step I also
would think that one of the things is to
listen and to listen to the various
communities and their experiences and
what's happening within those
communities what's working listen to the
physicians that are caring for this this
patient population and what are the
actual day-to-day barriers you know how
will this work you know you know will
that parent be able to to get that child
to continues treatment or follow-up or
will that adult be able to actually take
off you know for the number of weeks to
participate in this kind of chore
how do we do this in a way that is
respectful so I think you guys are just
everything I heard this morning about
what's going on here it just seems like
yes you guys are getting it right so now
you got to share that with others if
you're getting it right and enter and to
bring more people into your tent I think
that's really important how do you think
about fairness in that context as well
where there's a possibility that there's
a development of a technology here that
ultimately mean all right reach and Bri
effective person here but there's now
also a focus happening elsewhere is
they're not sure if I'm praising this
right
but what is that balance of fairness of
focusing on people here and focusing on
people elsewhere and making sure that
all can can benefit from the technology
while not overlooking people in our own
yeah so I you know I think that it's
really important that in high resource
countries that we get this right first
at one level I think you know if you
take Europe and Canada and United States
I don't know you have over a million
people with sickle cell disease if we
can design and develop this to work in
those countries and integrate that into
sub-sahara Africa and India I think that
makes sense at the same time that we do
the basic science research to make this
just so much easier to access in all of
the parts of the world so that we are
not losing sight that the burden is not
here and so we're doing the science to
actually bring this to Sierra Leone at
the same time that we're making it work
in Oakland California and so I you know
I feel very very confident that we have
to see if we can make this work in the
United States and and in a way that
people can access this as a treatment
that need this treat then that we can
make the decisions at whose most
appropriate for this treat and and then
we can figure out how to pay for it and
so you know I do think we need to think
about that one of the things I'm really
interested in also I didn't talk about
today is thinking about high-income
countries in and to do some comparative
analysis because our health care system
is so different from the UK and France
and Canada and how do we do this in
these different kinds of healthcare
systems and what can we learn from these
other countries that can help us here in
the United States so so I think that
there's a lot with
the collaboration in these countries
that have the ability to do this type of
treatment today and in an experimental
way to help us both integrate this into
our clinical care but then think about
low resource countries
