My name is Robert Prezant.
I'm the Dean of the College
of Science and Mathematics
here at Montclair
State University,
and I want to welcome all of you
to this second Emerging Science
program.
The Emerging Science
lectures had their roots
in the excitement generated by
the creation of a new science
building, right over there--
short walk right across campus.
The new Center for
Environmental and Life Sciences
opened up with our first
Emerging Science program.
And we thought it was
such a good idea that we
wanted to continue the program.
The Center for Environmental and
Life Sciences is really a model
for partnering-- partnering
between our faculty
and our students, and
partnering with industry
in state-of-the-art research
focused on pharmaceutical
and medicinal chemistry and
focused on environmental
science and
environmental management.
It's the home of our Sokol
Institute for Pharmaceutical
Life Sciences, and it's the home
for our PhD in Environmental
Management.
Related to this
evening's presentation,
we do in the College of
Science and Mathematics
have a full array of
pharmaceutical research,
including programs in
pharmaceutical chemistry.
But the College of
Science and Mathematics
is a full-array college
with every STEM discipline,
just about, represented.
And to that end, in fulfilling
the "It's All Here" mantra--
it's all here at Montclair
State University--
and to better
clarify some of what
we do in the College of
Science and Mathematics,
before we introduce our
very special speaker
for the evening, we're going
to have a short video to share
that takes a quick
look at some of what
we do in the sciences and some
of the other science-related
disciplines here at Montclair.
[VIDEO PLAYBACK]
- Well, it's been a
very exciting year
at Montclair State University.
As we looked at the
university, there
are a number of areas where we
really believed, first of all,
that we could excel, and
second of all, that there
was a tremendous social need.
And the sciences was
one of those areas.
We now have a new
School of Nursing,
which is just rising from the
ground in spectacular fashion.
We have growing programs in
public health, strong programs
also growing in nutrition and
food science, in psychology,
in exercise science.
We're just unstoppable.
- The story of the sciences,
not just at Montclair State
but anywhere, is the fact that
the sciences are the solution.
Within the College of
Science and Mathematics,
we have an array of institutes
and centers like our PSEG
Institute for
Sustainability Studies,
our Bristol-Myers Squibb Science
Teaching and Learning Center,
and our Center for
Quantitative Obesity
Research, as a few examples.
These are really critically
important, and primarily
because we're able to reach
out across the college,
even across the university,
to bring other disciplines
under one umbrella.
- We have a group of medicinal
chemists who are on that side,
and we have biochemists
and biologists.
So we do research in
a variety of areas--
a little bit in
the cancer arena,
a lot in global health
and parasitology.
- Passaic River Institute
was established back in 2004.
The function of the
institute is to promote
environmental
research in education.
We want the institute to
become the go-to place
in the region for any type of
water science-related question.
- Within the College of
Science and Mathematics,
we have a large
number of faculty
who are working on critical
issues that are important
not just to the individual,
the neighbor down the block,
but to the nation
and to the world.
- Montclair State University
has been a member of the LIGO
collaboration since 2013.
LIGO stands for the Laser
Interferometer Gravitational
Wave Observatory.
And it's really a
massive science project
that's trying to detect
gravitational waves produced
by colliding neutron
stars and black holes.
It was the first
time that we actually
see gravitational waves
passing over the Earth.
- My laboratory is primarily
concerned with jellyfish venom.
We've gotten very interested in
the effects of jellyfish venom
and the proteins in the venom.
Our ultimate goal
is to try and get
some of these proteins
once they've been cloned,
looking for
pharmacological relevance.
Could we develop new drugs?
- My laboratory lately has
evolved into a really exciting
area of research, I think.
There's a big problem now that
we use antibiotics too much
and we develop what is
called antibiotic resistance.
Bacterium become
resistant to antibiotics.
How is it that bacteria
become resistant?
But what we don't
know is, how is it
that bacteria respond to an
antibiotic at the beginning,
before they become resistant?
- Health care is
changing right now.
New Jersey has lots of needs
with a diverse population.
The Montclair State
University School of Nursing
is designed to
address those needs.
It's an amazing,
wonderful opportunity.
- Our Public Health faculty
are engaged in research
in a number of important areas.
We have colleagues
who are looking
at HIV and AIDS prevention
and communication
in communities of color.
We have folks looking at
adolescent reproductive health.
- There is a higher interest
in science and mathematics
and computer science and
information technology,
in cybersecurity,
today than ever before.
We have to make sure we are
accessible for the students who
want to head in that direction.
- Many of the young
scientists that we recruit
need the support of
scholarships in order
to pursue these
difficult fields.
And they need to study, and they
need to spend time in the lab,
and they need to work
with their faculty.
So if they're having to
hold down two jobs in order
to just pay their
tuition, they're
never going to be able to have
the time necessary to really
excel in the field.
And so the scholarship
support for science students
is absolutely critical
to their success.
- What I'm working on are
nuisance and invasive species
in marine environments.
So I'm working with different
species of jellyfish
and figuring out
how they got here,
and studying their populations.
And I'm also working with an
invasive species of seagrass
down in St. John's in
the US Virgin Islands,
and figuring out
how closely related
all those different
populations are
and why it's
spreading so quickly.
- We don't know the full
sequence of hyaluronic acid
from the Atlantic sea nettle.
They're a very common
jellyfish in this area.
So I'm working on getting
the sequence out of it,
comparing the sequence
to various creatures,
because hyaluronidase gets
used in a lot of venoms
throughout the animal kingdom.
- This is a toxin protein
from the venom of Atlantic sea
nettle.
So we have modeled this protein
using different algorithms
and different softwares.
First we had to isolate the
gene from the jellyfish,
and it generates
this model for us.
- It's amazing to be able to
come in here every day to do
research.
The school has also provided
me a [INAUDIBLE] scholarship
to do research here, which helps
not only fund me but also fund
the lab as well.
- All the monies
that we get here
allow us to work and
spend most of our time
on campus and in the labs
rather than going out and trying
to work on the side.
So it's really, really helpful,
and it gives you an opportunity
to really immerse yourself
in the campus environment
and in your department.
- You never know who the
leaders are on campus
or who's going to benefit
from the scholarship
at the end of the day.
So that person could be the
next president, the next doctor,
the next lawyer.
You never know.
So providing a scholarship
just helps mold
the society we're living in.
- When I speak with someone who
is thinking about contributing
to the sciences
and the development
of the sciences at Montclair
State University, what
I try to say to
them is, you are,
if you do this,
connecting yourself
with the future in a way
that is so dynamic that you
are creating the
scientists of tomorrow.
You're part of the expression
of how humanity moves forward
from one century to the
next with more knowledge,
more ability to care
for people in need.
[END PLAYBACK]
I now have the opportunity--
the very pleasing opportunity--
to be able to introduce
John Crawley, Chairman
and CEO of Amicus Therapeutics.
The reality is that we
previously invited John
to offer a keynote address
at the last PharmFest
here at Montclair.
And it was clear from the
response of the audience
that we needed to
have him back--
and have him back to talk to
a more diverse audience, not
just pharmaceutical executives.
John's exceptional
involvement with biotechnology
was energized by
the 1998 diagnosis
of two of his children
with Pompe disease,
a severe neuromuscular disorder.
In his drive to find
a cure for them,
he left his position at a
major pharmaceutical company
and became a serious
entrepreneur, lobbyist,
and industry guide for promoting
solutions to rare diseases.
As the Co-founder,
President, and CEO
of Novazyme Pharmaceuticals,
a biotech startup pursuing
research on experimental
treatments for Pompe disease,
John almost, by
sheer will of spirit,
promoted and eventually found
a path that would eventually
help stop the rapid progress
of this otherwise poorly
studied disease.
In 2001, Novazyme was acquired
by Genzyme Corporation
and John continued
to play a key role
in the development of a
drug for Pompe disease
as senior vice president.
And as many of you know,
John and his family
have been featured by
The Wall Street Journal
and are subjects of
a book, The Cure--
How a Father Raised $100
Million and Bucked the Medical
Establishment in a Quest
to Save His Children,
written by Pulitzer
Prize-winning journalist Geeta
Anand.
And John's story was
the basis for a movie,
Extraordinary Measures,
starring Brendan Frazier
and Harrison Ford.
John himself is the author of a
personal inspirational memoir,
Chasing Miracles--
The Crowley Family Journey
of Strength, Hope, and Joy.
And because it's not enough to
be an innovator, entrepreneur,
CEO, author, and
basis for a movie,
John is also a
commissioned officer
in the US Naval Reserve,
assigned to the United States
Special Operations Command
with service in Afghanistan.
He graduated with
a baccalaureate
in Foreign Service from
Georgetown University and a JD
from the University
of Notre Dame
law school, and an
MBA from Harvard.
In fact, the remarkable
efforts of the Crowley family
were recognized by Notre Dame by
the 2011 Family Exemplar Award.
Not surprisingly, John serves
on the executive committee
of the National
Board of Directors
for the Make a Wish
Foundation of America
and is a founding board member
of the Global Genes Project.
Now, before John gets up here
and shares part of his story
with us, I think we have a short
trailer to set as a premise.
[VIDEO PLAYBACK]
- In both kids, it's hard.
That's the real
threat to their lives.
- How much more time do we have?
- Megan, maybe a year.
Patrick, less.
- I wish that we had
a drug to treat Pompe,
but we simply don't.
I'm sorry.
- Dr. Stonehill?
- Yeah?
- This is John Crowley.
All the researchers
out there say
that you're a
genius on the verge
of a scientific breakthrough
- I'm not on the
verge of anything.
- How much would it take
to prove your theory?
- The odds against
you are crushing.
Where does that leave your
kids, when the dad has
no job and no health insurance?
- You're right.
This is crazy.
But I can't just sit around
and wait for my kids to die.
- I promised him
that we'd raise 500.
- That's all?
- Thousand.
- Is it 500 or 1,000?
- 500,000.
- Are you totally insane?
- Apparently.
- Is it worth it,
the Stonehill guy?
- He's really eccentric,
but his science is way ahead
of everybody else's.
- Do you have a wife?
- Um, ex-wives.
Two of 'em.
- How come?
- Because I'm so easy
to get along with.
- Figure any dude
in a business suit
can help a raise venture
capital and run the company.
But who's gonna be half as
motivated as a dad who's
trying to save his own kids?
- We can do this if
we push ourselves,
work around the clock.
- I already work
around the clock!
- Great, cure
diseases in theory,
but never help a single
human being in reality.
- I can't cure your kids.
You know that.
But I think I can
save their lives.
- What if he succeeds too late?
Then what?
- You're in clinical trials
by the end of the year
or we pull the plug.
- Nobody is going to tell
me how to run my lab.
- We're outta time.
- Are you crazy?
You've jeopardize
your chances of ever
getting your kids treated.
- Do you remember
you told me that I
should stop chasing miracles?
- Well, don't get
your hopes up, kid.
It's a Hail Mary.
- He's not gonna kiss me, is he?
- I will restrain him.
[END PLAYBACK]
So again, thank you
President Cole, Dean Prezant,
for having me here to speak.
I was really struck when
I listened to that video
in the beginning that
talked about Montclair State
University, and the commitment
to science and to research.
And one thing that
really struck me is it
really is, at its
core, about a mission
to develop entrepreneurs,
sometimes entrepreneurs
with very technical skills
in science and biology
and any number of disciplines
that are promoted and fostered
here at the university.
But when President Cole
talked about helping humanity
from century to century toward a
mission to help people in need,
the core of what we've done
in my now almost 20 years
in pharmaceutical
and biotechnology
is to help people in
need to make medicine.
So what I'd like to
share with you tonight
are just some of my
thoughts, my reflections,
on what it takes to be a
successful entrepreneur.
Two elements that are
critical to be a successful
entrepreneur-- specific
to science and research,
but I think maybe applicable
more broadly in the field--
and some characteristics
of what I've
seen of some of the very
best entrepreneurs who
I've worked with,
who I've studied,
and one who I've lived it.
So with that as a
premise, if I can,
I'm happy to stay afterwards
and answer any questions
about our family's journey
and our building businesses
to make medicines
to help people.
But let me begin by answering--
I used to say in these
lectures two questions.
There's now a third
question that I've
gotten quite frequently
in the last few weeks.
So first two questions are, how
did a movie come to be made?
Second is, how are
the kids today?
There's now a third question.
How in the heck did we get
invited to the Oval Office,
and how was Megan featured
in the president's speech
before a joint session of
Congress three weeks ago?
So I will answer
that one, as well.
So the movie came to be made
because of our children's
diagnosis, our second and third
children, Megan and Patrick,
born in December 1996
and March of 1998.
By the end of 1997, we realized
that Megan wasn't doing
the things that a
typical one-year-old,
two-year-old might do--
wasn't pulling up in a crib,
wasn't taking those first steps
at a year.
So you go from test to
test to doctor to doctor.
And by this time, we were
pregnant with our third child,
Patrick.
And then on a Friday
the 13th, Megan
was diagnosed with this rare
form of a muscular dystrophy
known as Pompe disease.
Pompe can be diagnosed in
various forms from infancy
through later in adult years.
In every case, it's a defect
in the same gene leading
to a deficiency
in the same enzyme
and the buildup of
glycogen in the muscles.
So a very severe and
rapidly progressing
form of a neuromuscular disease
or a muscular dystrophy.
We were told too that our
Patrick had a 25% chance
of having the disease, as well.
So that set us off on what
became a multi-year journey,
now a career--
a mission, a vocation
in some regards--
to try to alter the
course of that disease,
and now, to make a difference
in hopefully many more
rare genetic disorders.
Through all of that--
I won't take it through
a lot of what we did.
There's a pretty
decent movie they
made about it and some books.
But at its core, it was
about trying to beat nature
and trying to beat time.
So we went from the sense of
hopelessness, of frustration,
of shock, denial, eventually
very quickly settling
on determination,
determination to do
everything we could to change
the course of that disease.
And then moving from
the not-for-profit world
to starting a small
biotech in March of 2000,
eventually selling that to
a larger biotech company,
Genzyme.
And within Genzyme, helping
to develop that first medicine
that Megan and Patrick both
received in January of 2003.
In the summer of
2002, we were told
that they wouldn't live
another six to 12 months,
that time was up--
so very much about beating
time as much as nature.
As tough as those years
were, as tough as 2002 was,
2003 was a great year.
A number of people
began learning
about our journey, and the
kids with the 27th and 28th
in a clinical study to receive
that lifesaving enzyme therapy.
They received it and continue
to receive a Pompe medicine now
every other week,
intravenous therapy.
And they will for the
rest of their lives
or until newer
technologies come along.
So a lot of people
followed that story.
So how did a movie
come to be made?
Well, by the summer
of 2003 the kids
were getting much,
much stronger,
and they were doing things
that the couldn't do before.
A lot of people followed
that journey, one of whom
was Geeta Anand, a writer
for The Wall Street Journal.
Geeta wrote a
front-page piece that
appeared in The Wall Street
Journal in August of 2003.
When it came out, I
was on the west coast.
And my wife called me at about
7:30 in the morning east coast
time to let me know that we were
on the front page of The Wall
Street Journal, I had
to grab the paper.
And so when I was at the
airport later that morning,
I finally got a hold of it.
And there it was.
It was a title,
"For His Sick Kids."
And there's my pencil
picture, when the journal used
to do those pencil pictures.
My wife's.
They tell me I'm the
first in my business
school class to
ever have his pencil
picture on the cover of
The Wall Street Journal
and not having been
shot at or indicted.
So I took that as a good sign.
A lot of people read that,
one of whom was Harrison Ford.
And he had made lots of movies
that we're all familiar with,
but he'd not made a
real-life story before.
So he reached out
to producers of his,
friends who had made Erin
Brockovich, made the World
Trade Center movie, and they
decided to reach out to us.
It took us a while.
It took me about a year to get
comfortable with that notion.
We were very comfortable with
Geeta and her professionalism,
and telling our family's
journey through a book.
Hollywood was a different world.
My dad was a cop up in
Bergen County growing up,
so we didn't go to a lot of
Hollywood premiere parties
as kids.
So getting used to that
world was different.
But we trusted Harrison.
We trusted the producers.
And they did a beautiful
job in the screenplay
that Bob Jacobs wrote, and
then eventually in the film.
So that's how a movie
came to be made.
It came out in January of 2010.
And we use that film as a
vehicle to tell our story,
but it's really the story
of many, many people--
millions of families--
who live with any one
of 7,000 rare diseases
that in the United
States alone affect
more than 30 million people.
So taken together,
not so rare at all.
So that's how a movie
came to be made.
The kids today--
Patrick is 19
years old, a senior
in high school down in
Princeton in public school,
where we live.
And Megan is a sophomore at
Notre Dame out in South Bend.
Still in wheelchairs, still both
on ventilators, still both with
24/7 nursing.
The disease never
affects the mind,
so incredibly smart,
tough, precocious kids,
and as spirited as
you could imagine.
And you got a flavor
for it in the trailer,
but the young lady--
they screened over 2,000
actresses to try
to find somebody,
because you just can't pretend
at seven years old to have
that spunk and fire.
So they had to find somebody
who is so naturally kind
of gifted and full of spit
and fire to play our Megan.
And they did in young
Meredith, who played Megan.
So that's the kids today.
The visit to the White House was
certainly a very special event.
And the president honoring
our daughter Megan
at that speech,
how it came about
was when I saw that the
joint session to Congress
was going to be on
February 28, the last day
of February for a decade now
has been traditionally rare
disease day.
If you don't work or live in
the world of rare diseases,
you probably didn't know that.
So we've been working
really hard for a decade
to promote it.
We do a lot of events
in Washington around it,
a lot of community events,
a lot of online events.
So when I saw that,
through some folks
I knew in Washington, in
politics, in the White House,
I reached out a
couple of times to try
to let them know the unmet
needs and just how prevalent
all these diseases were,
and that if we could get
some words of encouragement
in the speech for Rare Disease
Day, it would be very helpful.
And the Friday
before the speech,
I heard from the White House
that in the final draft
there were two sentences
talking about rare diseases,
the needs of the community,
and Rare Disease Day, which
I thought was terrific.
After that, apparently a lot of
behind the scenes conversations
at the White House occurred.
We knew nothing about it until
we were out in South Bend
visiting our daughter Megan
for my wife's birthday.
And I got a voicemail
from a 202 area code
that I didn't recognize.
So about 20 minutes
later, I listened to it.
I had to listen to it twice.
And it was the
White House calling,
inviting my daughter Megan
to be the guest of honor
at the president's joint
session of Congress
and for a meeting
in the Oval Office
to discuss the needs of people
living with rare diseases.
I played that
voicemail for Megan,
and she started to
turn red and tears
started to flow down her face.
And without saying a word,
she looked at me, like,
is this a joke?
And I looked at her
without saying a word.
I said, no, honey,
I don't think it is.
And I think in that
moment, she realized
the enormity of on
her little shoulders
rested the responsibility to be
a proxy for millions of people
living in wheelchairs,
people with special needs,
people in the rare
disease community.
And she certainly
represented all of us
quite well, and with a great
amount of poise and dignity
and strength, and a lot of
wit throughout that day.
So it was a great
event for our family
and has led to some very,
very good discussions
about the needs of people
living with rare diseases.
So if I can, in the balance
of time I have, let me
come back to this notion
of being an entrepreneur,
particularly in the
life science field
and in taking some of
the great ideas that
come from universities
like Montclair State,
to take those ideas, the
theoretical research,
the practical
application of science
that students are taught--
and I had a great tour of one
of the medicinal
chemistry labs here
and saw the quality of
research and the commitment
to education, and to developing
people for careers in science,
which is what we need.
Our most limited capital in
biotechnology with good ideas
is not money.
There's plenty of
financial capital.
It's finding the people
to work with us--
people who have the technical
skills, but also something
a little extra,
something special,
people who want to
live that mission.
There are a lot of great
industries and businesses.
You can make blue jeans
and automobiles and iPods,
and they're all very noble
careers and important.
But I think what we
do in pharmaceuticals,
in biotechnology, is just
a little extra special.
And with that comes
extra responsibility.
The notion of making
great medicines
and getting them to as many
patients as quickly as possible
is really at the core of
who we are and what we do.
So what do you need
to be successful?
I think there are
two key elements.
One is a sense of vision.
When we started my
current company,
Amicus, here in
New Jersey, it was
based on technology out of the
university licensed and funded
early by some venture capital
investors in labs sponsored
by the State of New Jersey in
North Brunswick on Route 1.
We had shared lab space,
and I think four of us
in two cubicles--
so four or five people starting
this company in January
of '05 to really launch it.
Though, we had a
really big vision.
We wanted to affect a range
of human genetic diseases,
with the idea that if we
could help alleviate some pain
and suffering of
people living with some
of these rare diseases,
that in itself
is a good and
perhaps noble goal.
But we really
believed-- and continue
to believe-- that by studying
these human genetic disorders,
we will unlock the secrets
to how we treat much more
prevalent diseases.
You think about the Alzheimer's,
the Parkinson's, many cancers
today, most of them
really are just
a series of human
genetic diseases
that we don't yet
completely understand.
Some of the great
advances in immunotherapy
in cancer in the
last several years,
geared in many, many cases
to particular mutations,
and understanding the
human genome itself.
Those are great advances.
And if we can do that
and understand and unlock
the secrets of the human genome
and human genetic medicine,
I really believe that we
can fundamentally impact
human health much more broadly.
So we started with
a really big vision,
and we had some
really good teachers
and some good templates
along the way.
I'll give you two
brief examples.
One, the entire biotechnology
industry is only 41 years old.
It was started in 1976 by a
29-year-old venture capitalist
and a 30-something-year-old
research scientist
at a university.
In this case, it was
UC-San Francisco.
The doctor, Herb Boyer,
and a venture capitalist
named Bob Swanson--
and Boyer had just published
some of his academic research
on protein engineering
and making medicines out
of host protein
and animal cells,
with the idea that you
could develop therapeutics
for a range of disorders.
So Swanson, the venture
capitalist, read those papers.
He literally went
knocked on Boyer's door.
He said who he was,
talked about ideas,
and spent hours together.
And Boyer kind of wrote
out a bunch of theories,
explained his
papers, how that can
translate to make a medicine.
So Swanson was excited.
He eventually went
back to his partners
at the VC firm south of
San Francisco and said,
this is terrific
cutting-edge science.
And he had to get the money
for an investment, millions
of dollars.
So OK, that's what
venture capitalists do.
But think of the risk-taking.
They had to bet on
somebody, Dr. Boyer
and his colleagues, who
had never been in industry,
had never made a medicine.
They had to start a new company.
OK, that's what
venture capitalists do.
But they actually had to
start a whole new industry.
So you think of the
levels of risk-taking,
but the vision that they had.
And it succeeded.
And that company they founded
was called Genentech, the first
and arguably the most successful
of any biotechnology company
in our industry, going on to
develop medicines in cancer
and other fields that
have fundamentally
changed the course of life
for many, many people.
Bob Swanson died in the
1990s of a brain tumor,
and after that Herb Boyer,
the science founder,
was interviewed.
And he said something.
He said, when Bob Swanson
walked into my office that day,
he changed my life and
he changed the lives
of millions of other people.
And they only did that
because of the risk-taking
and because of the
vision they had.
It wasn't how they were going
to raise the first dollar
or build a business
plan or pay the rent.
I'm sure those were all
day-to-day concerns.
But they started with
a big, big picture.
It's also the idea
that started Celgene.
So Celgene started
in the mid-1980s,
with the idea that you could
potentially take thalidomide--
now, thalidomide was
used in the early 1960s
to treat nausea as a
side effect of pregnancy.
Never actually approved
here in the United States.
But a number of people,
including Dr. Sol Barer,
had the idea that you
could take it and actually
use it as a powerful
anti-cancer medicine.
This was a drug that
nobody wanted to touch,
a whole field of molecules
nobody wanted to go near.
That's a pretty big vision.
It took them from, the
founding of Celgene
here in New Jersey to approval
of that medicine, 17 years,
almost going bankrupt three
times as a public company
in the 1990s.
And today, Celgene employs, I
think, around 10,000 people,
with a market cap of
almost $100 billion.
And again, the lives that it
has saved and fundamentally
changed, the research that
it's funded around the world,
including here at
Montclair, is and will
continue to change the
lives of many, many people.
But they started with a
really, really big vision
of what they wanted to
do, fundamentally alter
the landscape the
treatment of cancers.
And now they're involved in
immunology and inflammation,
and now in neurology as well.
And even at a $100 billion
market cap, a lot more room
for Celgene to continue to
grow and to fundamentally
change people's lives.
That's a really,
really big vision.
So I had some good examples,
some good templates.
Sol, who was one of one of my
great mentors over the years,
served on our board for
nine years at Amicus.
So when I thought about
putting this company together,
I had to think, all right,
yeah, we need to raise the money
and we've got to pay the
rent and hire the people.
And those activities have to be
the foundation of what you do,
and sometimes they
can consume your days.
But we tried to start
with a really big vision.
What did we want this to look
like 10, 15 years from then?
So about now.
We talked about the
areas we wanted to be in
and the diseases we
wanted to impact.
We didn't accomplish
everything we set out to do.
It usually takes longer
and costs more money
in our business.
But we've done a lot.
And I think where we are today,
with about 350 employees,
about a $1 billion value,
a marketed drug in one
rare disease, other drugs in
clinical studies for a range
of diseases, programs in
more prevalent disorders
like Parkinson's, in our first
now little more than a decade,
I think we've laid a
really good foundation.
Plenty of mistakes along
the way, plenty of setbacks.
I've said this is an industry
that's a privilege to work in.
But it's also a tough one.
You've got to have the stomach
for the roller, coaster
because almost
everything we try,
unlike our brethren
in high-tech,
the basic technology
doesn't work.
And that's something
you have to get used to.
No matter how smart
the people are,
the great buildings you
build, the equipment you
buy, it's really
tough to trick nature
and to try to change
the course of a disease.
So it rarely works.
But when it does,
I still believe
it's the best job you can have.
So that vision
and risk-taking is
essential to be a successful
entrepreneur in our industry.
The other element is you
have to be patient-focused.
So everybody will say that.
Every company you go to
today, even the new startups,
will say, we are
completely patient-focused.
Some are.
Increasingly, they are.
We tried to do it a little
bit differently at Amicus.
And maybe it was
inspired initially
by our family's journey.
But it's now carried by
all 350 people at Amicus.
Even the name itself,
"amicus" in Latin, of course,
meaning friend.
So we thought if you
Latin it up a little bit,
it sounds a little
more sophisticated
than "Friend Biotechnology."
But the core purpose
of what we wanted to do
was if you were
somebody living with one
of these diseases
or a family member,
what would you want
the company to do?
What would you want
them to think about?
I went back to Sol's advice
to me-- make great medicines
and you will build great value.
So what we've tried to do,
though, is to think about, OK,
at every step of the
way, how do we do it
and how do we do it maybe
a little bit differently?
So from early on,
we put together
a scientific advisory board.
Most every company does.
We put together
medical advisory boards
for each of our diseases.
But then we did something
a little bit different.
We put together a
patient advisory board.
So we brought in people
living with the disease
or their family
members, about a dozen
for each of the diseases
we were involved in,
put them under
confidentiality agreements.
We don't pay them.
We pay their travel to
meetings, but we don't pay them.
And when I first proposed
this to my board,
they were a little
uncomfortable with it.
They're were
thinking, gosh, John,
if you're going to show them
everything that we're doing--
the confidential
regulatory interactions,
the preclinical data,
the clinical strategy--
somebody is going
to go on Facebook
or tweet it or talk
in patient meetings.
And I have to tell you,
in 12 years of doing this
across multiple
diseases, we have never
had one breach of
confidentiality.
In some regards, I think
they hold that trust with us
more sacred than any of our
consultants or advisors.
And it's made our
programs better.
It's not only the right thing to
do, the right message to send.
It has made our programs better.
The example I'll give you--
two years ago, my
daughter Megan turned 18.
And she said, Dad, I'm 18.
Can I sit on your
patient advisory board?
I said, I don't know.
Call Ms. Jane and ask her.
Jane's our chief patient
advocate at the company--
something else we
have that's different,
the only biotech company with
a C-suite level executive
for patients, a chief
patient advocate.
So Megan called
Jane Gershkowitz.
And Jane said, Megan,
we'd love to have you.
It was down in Orlando.
So of course, Megan calls me.
Dad, I've got to get to Orlando.
So we get her down there.
And I wasn't there for
this part of the meeting,
but they went
around and they were
talking about the
protocols for what
would become our new study
with a new medicine in Pompe
disease.
Our doctors had proposed
looking at six-minute walk test,
forced vital capacity--
some of the classic measures
of neuromuscular disease.
And they went around, and people
on the patient advisory board
really dug deep into it, added a
lot of inside of what mattered,
what didn't matter,
what was practical--
things like biopsies, skin
biopsies, muscle biopsies.
Would patients be
willing to undergo those?
Then they got to Megan, and
she was surprisingly shy.
She's a pretty
outgoing young lady.
It's hard to understand
Megan when she speaks.
The young lady who
played her in the movie
was able to speak more
clearly than our Megan can,
and I think that was a
little of her reticence.
But it got to her,
and they asked
the question going around.
What would make the
most difference for you?
And Megan said two things.
She said, one, I'd like
to breathe for one minute
without my ventilator.
And we were thinking
about developing protocols
to wean people from ventilators,
or how much stronger
all these technical measures
of pulmonary function
could become.
And she said that because
she knows still today,
although her heart is fixed
with her current medicine, that
is the thing that could be the
most life-threatening aspect
of Pompe for her.
If that trach is knocked
and a nurse for some reason
doesn't hear it or
can't clear the airway,
that's what could kill her.
So she said, number one, if I
could breathe for one minute
without my vent.
And then she said, number
two, if I could just
speak a little more clearly
so when I go to college
I can make more friends.
And I think there was that
silence in the room where
all of our technical
assessments,
all of the experts'
experiences, were really
put into perspective of, for
an 18-year-old young lady who
had lived and
thrived and survived
with this disease, what
would make a difference?
What would be a meaningful
endpoint for her?
And so we went back to it and
designed a range and composite
of endpoints in that study.
So just one small vignette of
how one patient's experience
made that program a
much better program.
If you come to our building,
our campus down in Cranbury
north of Princeton,
you'll see we've
got patient portraits decorating
all the hallways, and banners.
What we also, though, do is
the artist who takes them.
It's called "Living
in the Light."
So it's shot, the pictures
taken, in natural light.
And of course,
the double meaning
of living in the light
with rare diseases.
What he also does, what Levi
also does, the artist, is he
goes and spends a full
day, sometimes two days,
with each of the persons
who he takes a picture of.
And when I asked
him, I said, Levi,
I don't want you to
then put together
a plaque next to the picture
that has their biography.
We'll figure that out.
Tell us something special about
them, about their uniqueness,
about their spirit, their
hopes, their dreams.
And he did that.
And if you come to
our campus today,
you'll see dozens and
dozens of these pictures.
And we add to them a
couple of times a year.
We have seven offices
in Europe now.
Our offices in Europe have
some of those portraits
and have some
specific to Europe,
sometimes specific to the
countries that they're in.
But it's another
aspect of living that.
The other thing we do in terms
of being patient-focused is
at the beginning and end
of every major business
decision or discussion,
frame it in terms
of what's best for patients.
Literally, if you
had this disease
and you were sitting at this
table, what would you do?
What would matter?
And it really helps
change your perspective,
and it keeps you very,
very honest to the mission.
So we don't do it
perfectly by any means,
but I think in some
regards we do it
in a matter that is perhaps
just a bit different than some
of our peers.
Although increasingly, I'm
seeing this perspective
more and more brought
into companies,
from the smallest of biotechs
to the largest of big pharma.
And I think that's a
very, very good thing.
Just this past December,
President Obama
signed into law the
21st Century Cures Act.
And it specifically
mandates the US FDA
to take into account the patient
perspective at every stage
of drug development.
And I think that's a very,
very good advancement
for all of us--
and certainly
something we emphasized
in our meeting in the Oval
Office with the president,
how important that is to bring
in the perspective of people
who actually live
with these disorders.
So let me just spend a few
minutes, and then I'll wrap up.
If those are the essential
elements of success for being
an entrepreneur-- and whether
that is at the bench-level
research or whether you're
running one of these companies
someday--
that vision and risk-taking
and that patient-centered drug
development in medicine--
I think the title of
this talk is "When
Drug Research is Personal."
It's kind of easy to say how
it was personal for our family,
but it's personal in
every single case.
Even touring the labs
here, taking a look at some
of the basic research
going on, some day--
and it may have
already happened--
some molecule that a student or
graduate student here develops
goes on to actually treat or
maybe someday cure a disease.
That's personal.
That's personal drug research.
So some of the traits
I've seen of some
of the great entrepreneurs--
one is hope.
In some respects, what we do
in our business, we sell hope.
When I first came into
biotechnology in March of 2000,
I left my position
of Bristol-Myers.
I went to this small
startup in Oklahoma.
We had a handful of people,
$37,000 in seed capital.
And at 31, 32 years
old, I was the best
they could do for a CEO--
so not so good.
So I went out to
Boston to go to the bio
show, Biotech Industry-- or
now Innovation Organization.
It's the trade show
for all of biotech,
and I just wanted to be
a part of it-- to listen,
to take it in, to understand
this new industry.
I heard it was a little
different than pharmaceuticals.
I went to the
keynote that night,
and there were maybe 5,000
of us in the audience.
And the keynote speaker
was a quadriplegic
in a wheelchair on a ventilator,
and a big strong guy.
You could tell at
some point in life
he was a tremendous athlete.
He came out and he spoke.
And the first word that he
said was "biotechnology."
He said, that's a
great big word that
for people like me
just means hope.
He said it's hope
that someday you
may develop a treatment that
could treat my paralysis.
Maybe someday I could hold my
wife's hand on the beach again
or throw a ball to my kids.
And then he said
something really profound.
He said-- I'll
never forget this--
it's not just hope for me
and people like me today.
It's hope for people
who don't even
know that they need hope today.
I listened and I wasn't
quite sure what he meant.
And then he said,
it's for the kid who's
going to be on a football field
in a year and break his neck,
or the young lady in a diving
accident or a car accident,
who two years, three
years, five years from now
is going to go online,
and their lives
are going to be
turned upside down.
And they're going to
look for research--
and he pointed out
in the audience--
that some of you may not
have yet even thought of.
He said, that's
what hope really is
and that's what
biotech really is.
And he was right.
And that man was Christopher
Reeve, and a couple of years
later he died from
complications of his paralysis.
But I'm certain someday,
when paralysis is effectively
treated or cured, it will be
because of some of the wheels
that Chris and his
wife Dana set in motion
with their ideas, their
passion, their funding,
their foundation.
And it was a real
lesson for me in hope.
I always hoped with
our kids and others
living with Pompe that
we could beat nature
and we could beat time.
And we've beaten it a bit.
We haven't beaten it completely.
The game's still going on.
But almost weekly, I hear
from a new family with Pompe,
where a baby was born
and just diagnosed,
or an adult at age 50 was
diagnosed with a late onset
form of disease.
And they're thankful
for the work that
was done by thousands of people
that led to a treatment today,
and they're really
hopeful that we
may be able to come up with the
next best answers in the years
ahead.
So hope, and implicit in
that hope for the future,
and realizing that failure
is OK because we will learn,
and from that we'll
will derive an awful lot
of our future basis
for new medicines.
Persistence is a
really important trait.
We talked a little bit about
having the stomach for biotech,
and the roller coaster
for drug development.
I mentioned that
biotech is 41 years old.
You can actually go
back further than that,
maybe 70 years or so, and
one of the first true biotech
researchers was
actually Dr. Jonas Salk.
So a researcher
at the University
of Pittsburgh Medical Center--
he had been given a grant by the
Infantile Paralysis Foundation,
what we now call
the March of Dimes.
And it was to research vaccines
for the treatment of polio.
So polio is one of
the great health
scourges of the 1940s and '50s.
FDR suffered with it, of course.
His early work received
millions of dollars of funding,
and it failed.
After a couple of
years, it didn't work.
Eventually, he
developed a vaccine
that was tried in a
clinical trial in 1.5
million schoolchildren,
approved, and then eventually
went on to change the
course of the world--
the world's health, and
certainly, of polio itself.
But I was reading an article
a number of years ago.
It was from a magazine
ironically called Wisdom.
And it was years after that
medicine finally worked,
and Dr. Salk reflected on
his failure, not his success.
And he talked about
that first vaccine.
He described how they
had such great hope,
such enormous investment in
it, and how it didn't work.
And that day, he
was so devastated
when he got all those results
that he went to, he said,
a park.
And he sat on a bench,
thought about how
he's going to tell his wife,
his family, the people who
had invested all that
money, his lab colleagues.
And he looked out and he
saw dozens of children.
And when he looked
out at those kids,
he said he realized that
without a vaccine some of them
would get sick, some
of them may die.
And he said that he
realized in that moment
the enormity of his work,
and he went back to it
with a renewed vigor.
So that was his inspiration,
seeing those kids
in that playground.
And he was certainly persistent.
And some day, somebody-- if
Dr. Salk had just walked away--
would have come up with
a vaccine for polio.
But it would have
taken more time.
And how much human
suffering would
have occurred in those years?
So persistence--
incredibly important.
So is optimism.
This is something
you've got to believe in
and you've got to, in
a measured fashion,
try to convey that
we can do this.
I talked about Megan's spunk
and spirit for years growing up.
And she went to public
schools in Princeton.
Our household was
always really busy
with nurses and therapists
coming and going.
And between it all, we ended
up getting two Jack Russell
puppies, because we
didn't think we had enough
going on in the house.
But every morning,
Megan would come
rolling in in her wheelchair.
And every morning,
I'd ask her as I'd
be getting ready and
shaving, doing whatever,
putting my tie on,
I'd always ask you
the same question for years.
Megan, how are you today?
And for years, she answered me
with the same one-word answer,
awesome.
I'm doing awesome.
And I have to tell you,
if you've met Megan,
if you saw her then or today
and you saw a young lady,
then maybe 10 years old, in
a wheelchair on a ventilator,
even when she was sick or
had a respiratory infection,
and I gave you a piece of
paper to write 100 adjectives,
I bet you you wouldn't
write the word "awesome."
But that's what she thought.
That's what she wanted
the world to believe,
because that's
what she believed.
And I think even in that moment
in Congress three weeks ago
when the camera panned
on Megan and when
I and Aileen stood
next to her and had
the leaders of our nation--
Americans, not
Republicans or Democrats--
stand and applaud
her, and watching
her handle it with that grace.
And when she just
waved and smiled,
that was a really proud moment.
And I thought back
to all those years
when she'd just answer
with the word "awesome."
I'm doing awesome.
And I think if she
could tell anybody,
you could almost
sense in the chamber
and for much of what
I've heard afterwards,
it wasn't applause out of pity.
It was applause out of respect
and admiration and love,
whatever you want to call it.
Because I think
in some small way,
they got that she was
awesome, but so is
everybody else like her.
One of my favorite
stories since I
was talking to the senior leader
at one of our pharma companies
here in the state, nice
conversation a few weeks ago
and he said he was watching
it with his daughter.
And she's a 16-year-old
with cerebral palsy.
I didn't know that he
had a daughter with CP.
And he said, John,
I was watching this
and I was surprised, and
I'd never met your daughter,
and to see that.
And he said, and then I
looked over at my daughter
and she had tears
running down her face
and she had no idea who Megan
or John or what Pompe was.
And he looked at her and
he said, she looked at me
and said, Dad,
look at that girl.
She's just like me.
So that sense of
optimism that Megan
has shared with me every day,
with Aileen, with our family,
really has touched
so many people.
And that, I think, is one of
the most essential elements
of being a successful
entrepreneur.
You've got to believe.
So the last thing
I'll leave you with
is this lesson, and that's
of humility and time itself.
So I mentioned we really
kicked off in 2005.
We went public in 2007
with a lot of fanfare.
And years earlier
in my first company,
I couldn't get anybody
to give us money.
By the time Amicus came,
we had some experience,
a track record.
The markets were pretty good.
So we raised a lot of money.
We went around.
Investment banks take you on
these road shows for your IPO.
We had people literally
throwing money
at our little startup company.
We raised $75
million in that IPO.
They valued our business
at $300 million bucks.
Lots of back-slapping.
We went to New York.
We closed the deal.
We had a great board meeting.
The next day, I was on CNBC.
We were interviewed
by the newspapers.
We closed the stock exchange.
For an entrepreneur,
that's a pretty good day.
I bring the whole company
up from the Princeton area.
They have a celebration.
Got them hotel rooms.
Told them enjoy and have fun.
Time to go back
to work tomorrow.
But I went home.
And I'd been gone for about
two weeks, and went in
and checked on the kids.
Although a very successful
trip, it was a very long trip.
So I went and checked
on John Jr. and Patrick.
And they're pretty sound
sleepers, so they were sleeping
and I gave him a kiss
and they didn't stir.
And then I went in
and checked on Megan.
Megan's a very light sleeper.
And I kissed her and she woke
up, and she threw her arms open
and said, Daddy.
She said, Daddy!
I said, Meg.
She said, I missed you.
I missed you too.
She said, give me a hug.
I gave her a hug.
And she said, how'd your
big business trip go?
And I had my suit on, whatever.
I said, well Megs, you'd be
pretty proud of the old man.
We did pretty well.
She said, I know, Daddy.
I saw you on TV.
I said, oh, well, how'd I look?
And she's laying in
her bed and she's
tethered to her ventilator,
and she says, well, Daddy, she
said, you looked
really, really--
so I'm thinking,
you know, powerful
or whatever-- she said,
you looked really,
really kind of short.
So then there's that
kind of awkward silence.
And then she said, but Dad, that
yellow tie looked really good
on camera.
Thanks, Megs.
And then she asked
me, are you going
to be here in the morning?
I said, I'll be here.
She said, would you
take me to school?
I said, yes, Megan,
I'll take you to school.
And she said, great,
good night, I love you.
I love you.
And I think in that moment--
not because she had teased me.
Kids tease me all the time.
I think it was because
in that moment,
I realized the true
humility of success,
and how fleeting success is.
And I realized too that
she truly absolutely
didn't care about what
plane we were flown
around in or what banks or
how much money we raised
or what TV show I was on.
What she cared about,
nine or 10 years old then,
was that her dad was on a
really long business trip,
he was home, and he was
going to get to take her
to school the next day.
And you think
about what do we do
and why do we teach
in a university.
Why do we make these medicines?
At the end of the day,
all we're making them for
is to buy more time--
quality of life and
time with each other.
That's all it is.
And for me, it taught
me in that one moment
that's why we did all of
this and continue to do it,
for more moments like that.
So thanks.
I'll be around for a while.
I'm happy to take
your questions.
Thanks for all you
do at Montlcair.
[APPLAUSE]
I think we now have a better
appreciation about why we've
invited John to come back.
I just wanted to come up
and ask you to thank him
one more time for
what I thought was
an inspirational and
informative talk.
Thank you, John.
[APPLAUSE]
