Last week I attended the Partners for Cure (P4C) meeting in New
York. This is an annual meetup for an odd mix of academic scientists,
advocacy groups, venture capitalists, social entrepreneurs, and
biotech-pharma folks. In contrast to typical scientific meetings, which
explore the “what” and the “how” of research, P4C is mostly about the
“who” and the “when.” Specifically, who will deliver new cures and how
do we speed up this process? The meeting was sponsored by a group called
Faster Cures and driven by advocates, many fighting a fatal disease.
Throughout the two-day meeting, they reminded scientists that “time =
lives.”
But the theme that intrigued me was not the burning question of
“when” but the disruptive question of “who.” For many of the P4C
attendees,
the traditional model of NIH funding to academic scientists
followed by industry funding of translation to new treatments is not
working. One participant described science that has not helped patients
as “science fair projects.” Others disparaged a culture that focuses on
papers rather than products. Noting that academic scientists are
responsible for teaching, clinical care, and writing grants, many asked
whether the academic engine for research is powered to go as fast and
far as patients need. And for many the incentives of science in academia
seemed disconnected from the growing unmet needs of patients. Success
in academia is measured in number of papers, grant funding, and
promotion to tenure. Success for patients means treatments and cures
that offer a healthy, long life. As just one example, scientists have
few incentives and some disincentives to share data or resources. For
patients, progress may depend on the rapid sharing of information and
crowd sourcing of tough problems.
What made the P4C meeting so inspiring was the chance to witness
some of the new models that are changing our assumptions about “who”
will deliver new cures. There were sessions on experiments in
collaboration or crowd sourcing or new tools for a range of needs
including cancer, Parkinson’s, Alzheimer’s, and serious mental illness.
In fact, after four previous meetings, this was the first that included a
session on serious mental illness. But aside from the disease focus,
the message was that when nonprofits and companies and academia and
government agencies work together, good things can happen.
Some of the most innovative experiments are being developed by disease advocacy groups: (Stroke needs to be in here)
In the Michael J. Fox Foundation’s
Parkinson’s Data Challenge, researchers competed to develop the best
way to benefit patients and clinicians by collecting objective, passive
data on a large scale. (Passive data is generated as an incidental
product of everyday activities, such as using a cell phone.) The winner,
chosen from 630 entries by teams in 21 countries, was a mobile phone
app that analyzed changes in voice quality to detect onset and
progression of Parkinson’s disease.
The Pooled Resource Open-Access
ALS Clinical Trials (PRO-ACT), a joint project of academia, pharma, and
nonprofits, amassed more than 8,600 de-identified subject records from
18 clinical trials on amyotrophic lateral sclerosis treatment into a
single, harmonized dataset that is now being mined by scientists from
more than 30 countries.
The Project Data Sphere Initiative
is a big data initiative led by the Life Sciences Consortium, a task
force of the CEO Roundtable on Cancer. The Initiative provides access to
the data from the control arms of Phase III clinical trials. This
dataset, based on records from thousands of patients, accelerates
research by providing detailed information about the progression of
various tumors.
A new company, Curious, is driving
a patient-centric research movement by helping disease foundations
develop platforms for data sharing and open access.
There were remarkable stories from a new generation of
entrepreneurs. Halle Tecco, co-founder of Rock Health, talked about why
she left Apple to begin a company investing in mobile apps for health.
Jessica Richman, co-founder of uBiome, showed how the world’s largest
crowd-funded citizen-science startup established a unique microbiome map
for science. Josh Sommer, diagnosed with chordoma while an
undergraduate at Duke, has built the Chordoma Foundation to unify and
accelerate global research on this rare disease.
What became clear at P4C was that the “who” question is being
answered in very creative ways. Using registries and sensors and crowd
sourcing, patients are becoming drivers not just passengers on the
research bus. The traditional model of government-funded research in
academia followed by industry research to develop marketed treatments is
being replaced by collaborations between nonprofits, academic
scientists, and biotech with a goal of delivering treatments more
quickly for more people. Science has become global, slowed only by the
rate of data sharing and access. Because time = lives, we need to go
faster. Thankfully, there is a new generation with some fresh ideas.
This just proves once again what
Amy Farber has to say.
For the past five years Farber has been battling
not only her own disease but
also
the wall of resistance erected by those who believe that a patient can
make about as much of a meaningful contribution to the process of
scientific discovery as a laboratory rat.
