Transcriber: Ellen Maloney Reviewer: Denise RQ
How many of you know someone who has autism?
Raise your hands.
How many of you know teachers or therapists
that work with children with autism?
And how many of you are familiar with the heartbreak and the difficulty
that families encounter with children with autism?
So almost all of you had raised your hands,
and I'm sure that those of you who are watching this
will have similar experiences.
Autism is on the rise.
Just last month,
the Centers for Disease Control put the estimates at one in 68 children,
and one in 42 boys being diagnosed with autism.
Just to put that in perspective,
that represents an almost 80% increase in autism in just 10 years.
We have an epidemic on our hands.
We need new solutions.
And at Curemark we recognise that new solutions, innovation, and change
comes about as a result of disruptive technology.
I became involved with disruption at a very early age.
It started for me in the sixth grade.
That's me, with the circle around my face.
(Laughter)
I was the winner of the sixth grade science fair project.
My project was called
"Coronary Artery Bypass: The Future of Cardiac Care".
(Laughter)
I was fascinated with the heart back then
because Christiaan Barnard had just done the first heart transplant the year before
and my grandmother, who had rheumatic fever as a child,
had one of the first pig valve transplants.
So the heart fascinated me, and I wanted to do this project.
I wanted to make, what I called, or what we call today, a virtual bypass.
Back then, virtual meant that I had to go to the hobby store and get tubes.
I dyed the tubes blue, with the help of my mom,
representing the vein in the leg, and red, the artery that needed to be bypassed.
We used these tubes to show that in this virtual bypass.
Then I needed to figure out how to show the heart.
I took one of these old Westclox clocks;
most of you probably don't know them, but you would wind them,
and they had a very large sound that they made
when they would wake you up.
I took plastic wood,
covered the Westclox clock with the plastic wood,
and what does the heart and that clock have in common?
They both tick, that's right, they both tick.
So back then, in my sixth grade mind, I thought,
"How interesting, how amazing
that you can take a tube
and go around another tube, almost like going around a traffic jam,
and have something important for people."
Prior to the bypass, people either died
or they were permanently disabled with multiple heart attacks.
So I thought, "Wow, isn't this really cool that this will change things?"
I knew then that this was important,
and I didn't realize
that it actually represented what is "disruptive technology".
It would be called that today if we found the bypass today.
Why is that?
Because it's novel,
because it solves a problem,
because it's enduring,
and it improves people's lives
simply and elegantly.
Let's fast forward to the 1990s.
I'm in practice,
I'm seeing children and parents and a troubling trend emerged.
That trend was children coming into my office
who were five years old who didn't speak.
Who were nine years old, and weren't potty-trained.
Who were six years old and never played a game with another child.
Even more troubling was what the parents said to me.
The parents said,
"My child won't look at me," "My child won't hug me,"
"My child will never say, 'I love you mom, ' or 'I love you dad.'"
These children were in their own worlds,
and really what we were seeing was the beginning of autism.
So I thought, "OK, I'm seeing these children,
nothing seems to be working that other doctors are doing,
I need to find something; see if I can find a pattern,
something about them that maybe would give us a clue
or give me a clue about what was going on for them."
So I looked for patterns, as I do often, and found none.
In fact, the hallmark was that there weren't patterns.
For example, a core symptom of autism is speech deficit.
I looked at speech and some children don't speak at all,
and some children speak a little bit or intermittently.
Some recite whole passages to books and videos.
I was amazed at the lack of pattern.
But what emerged even further was what the parents would say to me.
Beyond their heartbreaking vision, or the heartbreaking understanding
of the child not speaking, or talking, or looking at them,
they also talked about what they ate.
Which I found to be really curious because the parents described it as,
"a white food diet," "a tan food diet", "soft food diet".
But the reality was, when I really looked at it,
it was a diet that was high in carbohydrate and very low in protein.
That intrigued me; it became a curious thing, so I thought,
"You know what? Maybe there's a physiological basis here.
Everyone's thinking about behaviour,
but maybe there's something physiological."
I started doing blood, and urine, and stool tests on these kids,
and found something really interesting.
The first nine children I did,
all had low levels of an enzyme called Chymotrypsin
which digests protein.
Then I did more testing, and even as I did more testing, I looked at this.
So here you have the first group of kids I did,
with autism and without autism.
The red dots represent children without autism,
and the blue dots, with autism.
Ten is the dividing line.
A value of ten Chymotrypsin and above is normal, and below is abnormal.
Here, a pattern emerged.
Children with autism had these low levels of this enzyme.
So I start testing more children,
and then I'm having dinner one night at my brother's home.
The whole family's there.
As usual he says, "So Joan, what's up?"
Let me give you a little background on my brother; he's a rocket scientist.
He actually developed the sun sensors that go on satellites
that guide our cellphones and GPSs; that's what he did when he was in his 20s.
He says to me, "So, what's going on?"
And I said, "Well, I did all this testing on these kids,
on this autism thing that seems to be emerging;
no one knows what causes it, no one knows what to do about it,
but I tested these kids, and it looks like they are lacking an enzyme."
And he says, "Wow, that seems like it could be really important."
And I said, "Yeah, it could be, but I don't really know."
He said, "You know what, Joan? You should really patent that."
I said, "What? Patent what? Patent that?"
He said, "Yes, because if it's going to have a value for the children,
it needs a commercial value.
And you know what?
I'll have a patent attorney call you in the morning."
And Jay did that.
He had his patent attorney call me in the morning,
and then we started writing patents.
The first patent went in in 1999, later that year,
and it was granted in 2003,
and now we have dozens of patents in Curemark.
But having a patent alone was not enough.
I kept thinking, "What about this? What if this could really be important?
What if there's a piece of this that could be really beneficial to the children?
What should I do?"
This is a gut-brain thing.
Wow, gut-brain thing?
That, for me, was even a big paradigm shift.
I think out of the box to begin with.
I thought, "Wow, this is disruptive!"
But others thought it was crazy, and foolish, and silly.
But that's when I'll be forever grateful to my dear friends Susan and Eric.
Susan and Eric will tell you Curemark began at their kitchen table.
One day, we're sitting there, at the island in their kitchen,
Susan, Eric, and myself, and we're talking about this, just like I'm talking to you.
They've known me for 20 years at this point,
and they also know that I'll dog whatever problem it is to death.
Whether that's a child's problem or whatever it is.
We're talking and in the middle of this conversation, Eric gets up,
he goes to the desk that they have in the kitchen,
he pulls out a checkbook,
and he writes me a check for 200,000 dollars.
He says, "Dr Joan, just go and do it,
because if you don't do it, you will never know
if it's going to work for these kids or not.
Quit practice and just go for it."
And I did.
I quit practice, 25 years of practice.
And I did that.
I went out there to see if this enzyme could be beneficial.
I had to tackle autism; autism is a big spectrum disorder.
It's a spectrum disorder; that means there's a big constellation of symptoms.
Therefore, it needed a constellation of ideas and understanding.
Let me just give you a little background here.
This is autism spectrum disorder, these are the core symptoms.
Associated with those core symptoms are secondary symptoms,
a whole bunch of them.
Then you add in GI issues, and you add in seizure disorders,
and you have a very simplistic, but view of autism.
Even today, there is nothing approved to address any of those symptoms.
Nothing.
The only thing that's approved
is something for irritability that results in this,
and they're anti-psychotics, and they don't address autism.
So I knew then, and I know now
that what had to happen needed to be very disruptive.
Because nothing worked.
Many things were tried, and nothing worked.
So when you think about disruption, you think about Silicon Valley.
Disruptive is Silicon Valley's middle name, right?
So any fleeting idea that they have, people throw money at it,
they want to do IPOs, they want to do all these things.
Why is that?
Because it's quick to market,
it's easy to do, perhaps,
and your return on investment is great.
In life sciences, where clinical trials, and FDA, and all the rest of those things
come into play,
you have to publish and all the rest of that,
nobody's throwing money at that.
It's not happening.
Silicon Valley: everything is pushed forward.
And in the life sciences, everything is pushed back.
It's a very hard place to be.
So where is Curemark today?
Well, Curemark has been fast-tracked by FDA which means that we have a product
that has a high likelihood of being approved
and meets a very large, unmet medical need.
Secondly, we've raised over 50 million dollars
in the absence of venture capital,
and we performed the holy grail of clinical trials;
a double-blind, randomized, placebo-controlled trial
of children with autism,
and we met our clinical end-points, and that's all good.
Now we're in the process of submitting our new drug application,
a pre-application under the fast-track program.
We've made great progress, great, great progress,
in terms of getting this forward.
(Applause)
Thank you.
(Applause)
So, what have I learned?
I've learned a lot.
One of the things I've learned
is that you need curiosity.
For disruption to occur you need curiosity.
You need to be looking at things and wondering, "Why?"
You need a belief; a belief in what you're doing,
and knowing you can take your product, and test it against anybody's or anything,
and you have belief in that product, and in the outcome.
You need to believe in yourself.
You need to be fearless.
And you have to have a tenacity; an incredible tenacity to see it through.
Because without these three pieces, you cannot disrupt the status quo.
Indeed, Curemark has disrupted the status quo in many areas,
but three main ones.
One is in drug discovery and development.
We looked at patients and their needs.
We developed a drug to meet those needs.
We took it through rigorous clinical trials,
and now, we'll bring it, hopefully, back to the patients.
It's patient-centered drug discovery.
We've disrupted the bio-tech financing model.
We have raised money in the absence of venture capital, from individuals.
Not from individuals with children with autism
but individuals who understand our mission, who understood this,
and understand why I was doing what I was doing.
And, we disrupted a medical paradigm
where digestion is digestion, and brain function is brain function,
and never the twain shall meet.
Even when the byproduct of protein digestion
is the very building block,
is amino acids of neurotransmitters like serotonin and dopamine,
all have major, major action in the brain.
So we've done much disruption, and we're not done yet.
We continue to discover things, to look at unmet needs,
and to change the paradigms, wherever we are.
And when we're all together as a team,
I remind my team every time why we come to work everyday.
We come to work every day because patients need treatments,
and families need hope,
and you know what?
After all, it's all about the kids.
Thank you.
(Applause)