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2013 Annual
Dinner & Fly‐in

A Researcher Turned
Patient’s Story & Hope
Sue Dubman
sdubman@gmail.com

DISCLAIMER
The opinions, preferences,
hairstyle, etc. contained
herein are solely the
wearer’s own and may not
reflect those of her family,
friends or employers, past
and present.

Regenerative Medicine

2

Who Am I?
Personally

Professionally

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 Graduate work at
Brown
 20+ years in senior
leadership positions
in biopharma &
government
 CDISC, BRIDG and
Quantum Leap Boards
 Led early translational
platform development
at the NCI

Wife
Mother
Daughter
Sister
Cousin
Aunt
Niece
Friend
Boston Area Resident
Patient
Advocate


3

My Entrance into the World of Parkinson’s
 Diagnosed with Parkinson’s in early 2009
 Floated down the River of De‐Nial for almost two years
 Went ‘public’ in 2012
 Found little to smile about
 Until I finally admitted to myself that I am a
Parkinson’s patient and I will be for the rest of my life
 And recognized that I needed a new purpose
“The purpose of life is a life of purpose”
- Robert Byrne

4

Bringing my professional life into my
personal life
 Since diagnosis, very focused on moving the Parkinson’s
agenda forward
 Today, I am an active Parkinson’s advocate
– Massachusetts Assistant State Director,
Parkinson’s Action Network (PAN)
– Parkinson’s Disease Foundation (PDF)
Advocate and Graduate of Clinical
Research Learning Institute
– Patient Outreach, Fox Trial Finder
 And, a Clinical Trial Participant
 Hoping to leverage years in R&D informatics


5

Why become an advocate (since whatever is
done is unlikely to help me)?
 My hope is that others don’t
have to go through what my
family and I are going through
 As a researcher, I know the
importance of patient
participation
 I also believe in the promise of
science
– And, maybe just maybe, there will
be something in time for me
– But the clock is ticking

6

The Promise of Science
 We have so much technology and so much
knowledge compared to the past
 We have opportunities that we only dreamed of
before and possibilities beyond our imagination
 Regenerative medicine is not my expertise but I do
understand its potential
– Better understanding PD at a cellular level
– Help with development of new progressive, predictive
models of PD
– Cell models of PD generated from stem cells could help
screen drugs more efficiently

7

Tomorrow’s Challenge
 When you go to the capital tomorrow, you need to
make sure Congress understands the ramifications of
their decisions
 NIH supports approximately 300,000 scientists. That
support is good for the economy, creates jobs and
saves lives
 Parkinson’s is a very complex disease
– The ability to use stem cells to replace dopamine‐
producing neurons is no small task
– We need to be realistic – it will cost lots of money
and takes lots of time

8

The Need to Invest
 Science is an investment in the future and we need
to make sure we have appropriate funding
– These investments are for our fellow citizens
– We can’t be satisfied that things are okay
 Investments are needed in both the private and
public sector
– It is not just NIH – it is also FDA
– FDA is particularly under resourced

 We need to develop significant infrastructure to take
advantage of advancements and for that, we need
government’s help

9

The Need to Change
 That doesn’t mean that we shouldn’t
do whatever we can to improve the
way we do science
 We need to embrace  change
 In the biopharma industry, where I
currently work, this means:
–
–
–
–
–
–

More pre‐competitive collaboration
More agile organizations, willing to explore new ideas
Embracing new information technologies
Data standards for improved data sharing and re‐use
Increased patient participation in the research process
And, much, much more


10

Learn from failures as well as successes
 All of us in the PD Community are disappointed in the
Failed phase 2b Trial of Experimental Treatment for
Ceregen’s Gene Therapy, CERE‐120 (AAV‐nuerturin)
 However, this area of science continues to be one of the
most promising potential approaches
 Although this gene may have failed, it did prove that
Gene Therapy:
– Is a technique that can be conducted safely
– Can provide greater understanding as to the nature of the
brain targets, the delivery systems and the surgical
techniques


11

Why Should Congress Care?
 Because it costs a lot and investments not only save
lives but create jobs
 PD affects an estimated 7‐10 million worldwide
 An estimated $25 billion of direct and indirect costs
of PD each year in the U.S. alone, including:
– Treatment
– Social security payments
– Lost income from inability to work

 Medication costs for an individual person with PD
average $2,500 a year, and therapeutic surgery can
cost up to $100,000 dollars per patient

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Summary
 We are making progress but way too slowly
 We need to continue to advocate for
improvements in policy, legislation, R&D
processes and funding:
– To improve quality of life for patients and their caregivers,
family and friends
– To stop or slow progression of Parkinson’s as well as other
severe and disabling diseases


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Thank you for all that you do...


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PD Advocacy in Stem Cell Research
 The Parkinson’s community has been at the forefront of the
struggle to achieve research freedom for scientists working in
the field of embryonic stem cell research
 The Parkinson’s Action Network (PAN) is a founding member
of the Coalition for the Advancement of Medical Research
(CAMR)
– Amy Comstock Rick, President of PAN, is President
– Michael J Fox Foundation, Parkinson’s Disease Foundation
(PDF), the Parkinson Alliance and the National Parkinson’s
Foundation are members
 Parkinson’s Advocacy Organizations along with others also
continue to educate the nation about the importance of
medical and scientific research, including stem cell research

15

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