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DISCLAIMERThe opinions, preferences,hairstyle, etc. containedherein are solely the
wearer’s own and may not reflect those of her family, friends or employers, past
and present.
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Who Am I? Personally Wife Mother Daughter Sister Cousin Aunt Niece Friend Boston Area Resident Patient Advocate
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
Professionally
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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
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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
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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
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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
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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
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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
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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
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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
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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