hello thank you very much it’s wonderful
to be back at stage and I appreciate the
opportunity to talk to you about
something that is very near and dear and
I’m very passionate about cure duchene
tradition has been acknowledged over the
last couple of years for creating a
unique nonprofit business model the
reality is I’d like to say that we set
out to do this purposely but basically
we just applied logic and common sense
to a problem so a little bit of
background I was raised in just a normal
household I had an older brother and
sister my mom used to always call me the
pragmatists in the family and I because
it was my mom telling me that I never
bothered looking up the word to figure
out what it meant and so many years
later and I guess she would she would
tell me that I was always able to shift
from Planet a plan B pretty quickly
without a lot of stress or anxiety and
so I was also very rebellious when I was
young I was especially my teenage years
I was pretty much a rebel looking for a
cause and I would go through very
intense stages of whether it was horses
or rock and roll or travel or whatever
it was I was completely focused on what
i was doing to the point that when I was
in college my friend started calling me
saying that I had monomania and I did
look this word up and I wasn’t real
pleased with the first definition about
mental illness so I like to think that I
attended more towards number two which
was excessive concentration on a single
subject and so we basically ended up
having a huge huge problem our son
Hawkin was born in 1997 when he was five
years old he was diagnosed with Duchenne
muscular dystrophy we had never heard of
this word before and we really didn’t
know what to do and there was a problem
in my life that I honestly did not have
a
Lucien for and it was huge Duchenne
muscular dystrophy is the most common
form of muscular muscular dystrophy it
affects boys it’s an excellent
chromosome disease they’re usually
diagnosed between three and five years
old usually by the time they’re twelve
years old they stop walking confined to
a wheelchair later in teen years they
need ventilation to breathe they’re
unable to move their arms or even use a
keyboard on a computer this is a
terrible prognosis we researched we went
to all the doctors we were basically
told we’re told that there’s nothing you
can do go home and love your child this
disease affects all parts of the body it
affects the brain there’s a lack of
dystrophin which is a protein that’s in
muscles and also in your brain so
there’s cognitive issues many times of
these kids your heart is a muscle and
basically the primary cause of death is
cardiac failure usually with with boys
young men in their early to late 20s so
it was a very serious problem we were
basically told that our son we were
going to lose him as most most parents
were seeing their their sons graduate
from high school so Dan pilota who
actually was a TED talk several years
ago that I heard and really inspired me
I had already been running a non-profit
for a few years and trying to grapple
with just the regulation of the way
things were done there were a couple
muscular dystrophy organizations and
Duchenne organizations and we tried to
work with them and we really had no
intention of going out and starting our
own organization but what we heard was
when we would come up with new ideas new
ways of doing things that’s not the way
we do things here and so we looked at
this and thought well they don’t have a
cure yet for a disease and they’re
really not very far along so maybe we
shouldn’t be doing it their way and so
that was why we created the business
model and started moving forward so Dan
pilota was very articulate and talking
about the dysfunction of how charities
are
and help what the public expects from a
charity it seems that charities are
supposed to operate with very little
money and get big results and he felt
and said basically that it’s it’s
unrealistic to expect that that
frugality is is a measure of success and
morality when in essence we should be
thinking big we should be thinking about
problem solving and having setting and
being rewarded for our accomplishments
and for setting high goals and achieving
them and not worrying about our expense
ratio it’s like any business if you have
to operate on an eighty-five percent
profit margin you’re going to have a
tough time and that’s what nonprofits
are expected to do so he basically said
let’s change the way we think about
changing the world so here to Shen you
know I think a lot of it has to do with
the fact that both my husband and I came
out of business and we started looking
at traditional nonprofits and basically
what you had is we would you know a
non-profit goes out and works really
really hard to raise money from people
like you to to donate in in medical
research what this means is you give a
donation in the form of a grant to an
academic institution and the scientists
there you work on a sponsored research
program and and you know if the if the
science is successful and it’s promising
a biotech or pharmaceutical company may
come in and license that technology and
if that drug is developed and it’s
commercially successful then the biotech
and the pharmaceutical company make a
lot of money and you the nonprofit go
back to your donors and ask for more
money to do it all over again it’s very
very hard raising money so what cured of
shin did is we created what’s called the
venture philanthropy model and actually
we didn’t we didn’t create it it was was
kind of an unknown phrase when we
started this organization but a few
organizations had already had successful
business models
namely actually one cystic fibrosis have
done a fantastic job with this so
basically here to shen we take our
donations and we work with biotech and
pharmaceutical companies as much as we
can the difference between these two
models and what what defines success is
for an academic institution success
means getting a paper published success
for a pharmaceutical or biotech company
means having a drug that’s working so
cure Duchene knows that drug companies
make drugs and so why not partner with
them so we started partnering early with
with drug companies where we would
actually invest and and if there was
commercial success then part of that
return would come back to cure duchene
that we could go and reinvest in further
research and so we still have to go back
to donors because as you’ll see in just
a minute you know weary deploy the funds
and if we’re going to move on to more
projects we do need to continue to raise
money but it’s not a one-way street this
is a case study that actually is a very
successful case study in 2004 cure
duchene and over a couple years invested
two million dollars into a little tiny
biotech company called presenza and the
netherlands and we went through ups and
downs with with their drug development
program and finally in 2015 a larger
biotech company in northern california
bought them called BioMarin and when
that transaction happened are two
million dollars we were able to get back
about seven million dollars from that
which we then redeployed into five
additional research projects four of
them being biotech companies one of them
being an academic institution bamboo
therapeutics is a gene therapy company
that potentially has a cure by
correcting the DNA and we invested money
into that in two thousand
January 2016 august 2016 Pfizer bought
them in the future if that is successful
and hopefully it will be you know at
some point cure edition will be able to
take those funds that we get from that
and reinvested Capricorn era più tix
has the heart stem-cell exosomes it’s
delivered directly to the cardiac and it
is in a phase 12 again we’re awaiting
results for that miles Eric’s resurrect
small companies that cure edition was
the initial funder resurrects was right
out of USC that we created a startup
company and license at technology so
it’s it’s really exciting to see that
one one investment that started with
people like you in the room here by
trusting in an organization that cares
so much about a disease and we have the
best experts in the world you can see
how it’s that the funds that donors give
us are leveraged and able to grow so
this is this is a business model that we
would like to to work with we basically
find the best science areas and another
thing that sets cure edition apart from
the traditional nonprofits most
traditional nonprofits that fund medical
research have you know very very expert
Board of Advisors with the scientists
and they once a year get together and
they do a call for proposals and they
meet and they score and it’s you know
it’s a very high level meeting and they
find the best science well that’s great
but what if what if there’s certain
parts of this disease that aren’t being
addressed by the proposals that they
happen to get and so we take a top-down
view where we basically look at this
disease and we realize that it’s cardiac
its diaphragm muscles failure it’s
brittle bones it’s inflammation and
fibrosis and you know gene replacement
and correcting the gene and all these
different things that have to be taken
care of so we look at it like from the
top down like we have to impose our will
on the science
and so we basically go out and set the
targets that we want to address and we
find the best people to work on that and
if nobody’s doing it we create that and
it’s worked out to be very successful
something else that distinguishes cure
dash in from I think at least at the
nonprofit’s that I’m familiar with in
the muscular dystrophy space is when it
came time to hire our team I did not go
to academic institutions if drug
companies make drugs then I wanted to
hire a professional from the
pharmaceutical business which we did
with 30 we hired a scientist with 30
years biotech and pharmaceutical
experience who can actually help us
through the drug development process we
hired somebody out of a venture capital
firm who is in a Stanford trade Stanford
trained and B that can help us design
these deals and really work with the
biotech companies and so we find the
projects we find the best projects we
fund them and we also foster them by
offering our expertise the patient
access the key opinion leaders and we’re
able to foster these all the way through
to the hopefully commercialization so
venture philanthropy I I believe in my
opinion is the way that nonprofits
should be run if possible they’re
self-sustaining again raising money is
really really hard and will never stop
raising money through our galas and our
fundraising and that’s really the core
but we are able to tell our donors that
we’re going to leverage the money that
you give us and grow it by being smart
without money it’s replicable I’m on
several panels talking to other
nonprofit organizations especially in
the rare disease space trying to help
them understand you know different
financing models whether its equity
convertible notes royalties licensing
agreements there’s a lot to learn and
taken us will taking me personally a
long time to learn that
and and I think this is a model that is
starting to be replicated a lot in
nonprofits it’s it’s a way to
revolutionize the the medical research
and for me the most important part is
this is a way to get meaningful funds
and I’m not talking you know ten twenty
thousand dollar grants we’re talking
million or two million dollars where we
can actually see the project and make it
happen quickly so meaningful funds that
come back to the people that care the
most about what they’re going to do with
the money so we care more than anybody
we care more than the pharmaceutical or
the biotech companies about the success
of that particular drug and so when we
have control over it and we have the say
it goes a lot faster and again back to
Dan pilotis point it’s like let’s let’s
reward success and what is success in
medical research it’s drugs that help
patients not papers that are published
in journals and so the whole venture
philanthropy rewards success by actual
results speaking of results last year we
actually had the first drug approval for
duchene it was a long and rocky road
getting there we learned a lot on the
way lots of mistakes and lots of ways
that we would do things differently in
the future but we do have one drug
approval so we know we can do this this
drug is not a cure we still have a long
way to go it doesn’t treat the primary
cause of death which is cardiac failure
and it only treats about thirteen
percent of the patients but it’s a start
and my husband always uses the Roger
Bannister the four-minute mile that
until Roger Bannister round the
four-minute mile nobody believed they
could do it and after he did that lots
of people made that record and so we
believe that we are so close to being on
the cusp of being able to treat many
more boys in a much more meaningful way
so these are few tiles of arns
estimates and it shows basically you
know a top-down view of treating the
whole disease whether it’s replacing the
dystrophin that’s missing treating the
cardiac failure we funded an initiative
to help companies better understand
clinical trials to accelerate the
process because the FDA was being very
very difficult and so we had to learn
how to work with the FDA to give them
the information that they need it in a
way that they would sign off on it
basically i’ll come back to X onyx and
these other these other ones I mentioned
so just recently this week in fact on
Monday we just announced the formation
of a new company called X onyx and I
don’t know if any of you have heard of
gene editing or crisper cast nine but
it’s taken to scientific world by storm
it’s a means of going in and almost like
using scissors to edit the gene and put
it back together the way you want it to
be and there is so much hope for this
technology to cure so many diseases and
make it not just a chronic issue where
you have to have ongoing treatments but
actually a cure and this is where we’re
moving towards so talk about problem
solving great opportunity the problem is
is that there are three companies three
public companies that are working on
Chris Burke as 9 technology their public
companies which means they’re their
responsibilities to the shareholders and
so they’re going to be working on the
low-hanging fruit first which means
they’re going to be working on diseases
where there’s like a single organ so
right now they’re working on for example
I issues when you look at muscle we have
a lot of muscle in our body and it
spread all over the place so just
looking at the expense of treating an
eyeball versus the muscle throughout
your body with a very very very
expensive delivery system and drug is
overwhelming not to mention how do you
get it throughout the body it’s one
thing to
inject a needle into a specific organ
and cure it it’s another thing to get it
throughout your body so great technology
big problem they don’t want to work on
dish in right now and what it’s like to
be a parent and to see a technology that
could cure your kid and nobody wants to
touch it because it doesn’t fit the
business model at this particular point
so what do you do you find a solution
and our solution was to form a company
called X onyx we licensed the technology
from UT Southwestern dr. Eric Olson is a
premier muscle biologist in in
Christopher technology and we just had
our announcement this week and we are
very very excited this is another
project that I’m extremely excited about
so a company that had a drug that failed
to get approval from the FDA and yet it
was shown to be effective in kids and so
here you’ve got a dichotomy between what
the FDA is seen on paper and what the
parents and the children are seen in
their lives where they’re responding
everywhere from stabilization of the of
the disease which is a good thing
stabilization is really good in duchene
as well as some of the kids actually
regaining function and having a better
lifestyle and riding bikes or they never
will be able to do that before and just
improve quality of life and then the
drug is told from them because the FDA
could not see what the parents saw so
this happened about nine months ago and
the parents have been devastated and I’m
very close with many of those and so we
we worked really hard and again here’s a
big problem a drug that works can’t get
it to the kid so to make a very very
long story short we created another
nonprofit called CD access and we’ve
negotiated with the company to transfer
drug to this non-profit and we are now
in the drug distribution and delivery
business there’s always a way and is
always a plan B
and I encourage all nonprofits to think
outside the box and figure it’s not
about running an organization it’s about
finding a cure so this is Hawkin he was
number 88 on the sage football team he
was talk about finding a solution he
loved he loved football always and when
he was diagnosed with Duchenne it was
just devastating he always had a ball in
his hand all the time when he was young
and so thanks to the coaches here it’s
aight ill and my husband being
persistent Hawkin was able to be part of
the football team and it’s been just a
tremendous tremendous story and stage
has been such a part of our life and
it’s extremely satisfying to be here
he’s now at USC in journalism and
writing up a storm so I would just like
to say thank you and I would like to if
possible inspire people here especially
the kids that if you have some quirks
that people comment on go with it
embrace it you know it’s God gives us
gifts and sometimes they’re not easy
ones and sometimes they make you feel
uncomfortable and and you don’t fit in
really well but there’s a reason why you
have a gift and my my advice to you is
let it let it play itself out and find a
way that you can use that to help other people thank you