@derailroaded @EricTopol Donate your data or your organs?I prefer the 1rst 1 Health is too serious to be left to pol (forget the NASA gate).
— CATHERINE COSTE (@cathcoste) September 17, 2013
Patients Share DNA For Cures (WSJ, September 17,2013) |
Article by RON WINSLOW
"Patients with rare and deadly diseases are getting a powerful new boost.
Cancer foundations and other nonprofit patient groups are investing
tens of millions of dollars to build genetic databases in an effort to
speed drug development and jumpstart clinical trials.
The databases are designed to collect DNA and other information from
patients with hard-to-treat diseases. The material can be analyzed for
certain genetic mutations and made available to scholars and
pharmaceutical companies."
On Tuesday, the Leukemia & Lymphoma Society is set to announce a
three-year, $8.2 million project that combines the resources of the
Oregon Health Sciences University, gene-sequencing company Illumina Inc.,
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and Intel Corp.
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Funded by the society, the resulting database will contain DNA
information and analysis gathered from 900 patients with acute myeloid
leukemia.
Array Biopharma Inc.
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is the first of what the society expects will be a host of companies
that will test new and existing compounds against any potential drug
targets revealed in the patient data.
The plan is to have
clinical trials for relapsed AML patients underway within one year and
for newly-diagnosed patients by the end of the second year. "We're
talking about an aggressive timeline for moving this data into the
clinic," says Brian Druker, director of the Knight Cancer Institute at
OHSU and leader of the project.
The latest collaborative efforts show how powerful advocacy groups
are using their clout to urge cooperation over competition in the
academic and corporate spheres. A main goal of the databases is to lower
some of the costs and risks that might discourage pharmaceutical firms
from investing in potential new therapies.
Next week, the Multiple Myeloma Research Foundation is hosting a
symposium in New York City where it plans to reveal the first data from
an ongoing $40 million, 1,000-patient study. Called Compass, the
project is dedicated to mapping the genetic and clinical characteristics
of the blood cancer.
The organization is making raw data from the study available to
academic and industry researchers that could help them find information
about at least 10 different mutations believed to fuel the cancer.
"Our goal is to accelerate cures," says Kathy Giusti, founder and chief executive officer of the foundation.
One factor that makes the DNA databases possible is the rapidly
declining cost of DNA sequencing. "The cost to sequence 900 patients a
decade ago—that would have been an impossible project to fund," Dr.
Druker says. "Now it's actually a reasonable amount of money."
George Mulligan, director of translation medicine at Takeda Pharmaceuticals Inc.'s
4502.TO -0.43%
Millennium Pharmaceuticals unit, says groups like the myeloma
foundation can serve as an "honest broker" with "the influence and the
clout" to bring a variety of research interests together. Millennium
developed Velcade, a multiple-myeloma drug.
The myeloma
foundation has raised nearly $250 million since it was founded in 1998,
when the median survival rate was about 3-1/2 years. Thus far, the
organization has supported 47 trials involving 24 drugs, six of which
have reached the market.
To make their money work even harder, patient foundations
increasingly make grants contingent upon researchers agreeing to share
data and expertise—even with potential competitors. They say that such
open access allows the research enterprise as a whole to operate more
efficiently than just one drug company or academic group working alone.
"We see ourselves as a catalyst for collaboration," says John Walter,
chief executive of the leukemia group. "It's not a conventional
approach."
While cancer groups are paving the database route, others— focusing
on disparate conditions including Parkinson's disease—are following a
similar path. Later this year, the Michael J. Fox Foundation will begin
enrolling the first of 600 patients in a Parkinson's disease study,
hoping to gain insight into how two recently discovered genetic
mutations affect progression of the debilitating condition. The DNA data
will complement an ongoing $50 million study co-funded by 13 drug
companies to identify other biological markers of Parkinson's so
researchers can better study potential treatments.
"It's a very exciting set of experiments that will open a lot of
doors and give us a lot of insight that previously wasn't there," says
David Schenkein, chief executive officer of Agios Pharmaceuticals Inc.,
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a Cambridge Mass. biotechnology company.
All three foundations
have made their mark promoting this kind of collaborative
research—efforts that have helped several drugs come to market.
The Michael J. Fox Foundation, founded in 2000 by the actor after his
Parkinson's diagnosis two decades ago, has funded the development of
several research tools, including chemicals and animal models of the
disease. Last week, Biogen Idec Inc. and Amicus Therapeutics Inc.
announced an agreement to pursue possible Parkinson's drugs based on
research at Amicus that the foundation supported.
A big hurdle for Parkinson's drugs is a lack of validated biological
markers that enable researchers to track whether a treatment is actually
working."It's made it very high-risk [for companies] to move forward
with these types of therapies," says Todd Sherer, CEO of the foundation.
The $50 million collaboration between the foundation and 13 drug
companies is an effort to address that issue while reducing the risk to
individual firms." (http://online.wsj.com/article/SB10001424127887323342404579079453190552312.html)
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