Fred Hutchinson Cancer Research Center Partners With GlaxoSmithKline to Develop Muscular Dystrophy Therapeutics

   Fred Hutchinson Cancer Research Center Partners With GlaxoSmithKline to
                   Develop Muscular Dystrophy Therapeutics

Collaboration represents first U.S. institution chosen to participate in GSK
academic-industry partnership

PR Newswire

SEATTLE, Dec. 10, 2012

SEATTLE, Dec. 10, 2012 /PRNewswire/ --Fred Hutchinson Cancer Research Center
and GlaxoSmithKline PLC (GSK) today announced a partnership to develop
therapeutics to treat an inherited form of muscular dystrophy. 

The goal of the new agreement is to develop a small-molecule-based medicine to
potentially reverse facioscapulohumeral muscular dystrophy, or FSHD, by
inhibiting the activity of a protein that is incorrectly expressed by the DUX4
gene in people with the disease. The protein activity is what damages muscle
cells and leads to progressive muscle weakness and atrophy in FSHD patients.

The genetic and disease mechanisms of FSHD were discovered by an international
team of scientists led by Stephen Tapscott, M.D., Ph.D., a member of the Fred
Hutch Human Biology Division, in a series of studies published between 2010
and early 2012. Tapscott will lead the Fred Hutch work in the GSK

The team's discoveries also have implications for developing cancer
immunotherapies because researchers also discovered that DUX4 regulates
cancer/testis antigens. Cancer/testis antigens are encoded by genes that are
normally expressed only in the human germ line but are also abnormally
expressed in various tumor types, including melanoma and carcinomas of the
bladder, lung and liver. This knowledge will give researchers a way to
manipulate the expression of cancer/testis antigens, potentially opening the
opportunity to use these antigens in a cancer vaccine.

The partnership with GSK is a first of its kind for Fred Hutch, which is also
the first U.S.-based institution to sign on with GSK's "Discovery Partnership
with Academia" (DPAc) program. GSK launched the program last year to combine
the insight and creativity of the academic world with GSK's drug-discovery
expertise to turn innovative research into medicines that benefit patients.

GSK currently has DPAc collaborations in place with Cambridge University and
the University of Dundee in the U.K.

Unlike traditional licensing agreements in which the licensee is given full
control to develop a discovery, the collaboration will involve GSK and Fred
Hutch scientists working together to develop, test and hopefully bring to
market a clinical treatment.

"GSK has huge expertise in developing agents against protein activity, so our
opportunity to work with them is fantastic," Tapscott said.

"At GSK we believe that combining our drug-discovery expertise with the
in-depth disease knowledge of specialist academic groups can seed innovation
and help speed up the discovery and development of new medicines," said Pearl
Huang, global head of DPAc. "We're excited to be expanding our academic
program in North America and are looking forward to working closely with
scientists like Dr. Tapscott, whose deep understanding of disease biology will
complement our own work in this field."

FSHD affects about one in 20,000 individuals and usually begins in late
adolescence. The effects start around the facial and upper-extremity muscles
and eventually progress to muscles in the lower extremity. People with more
severe FSHD become wheelchair bound and their life spans are often shortened.

In an era of flat federal research funding, this collaboration signals an
increasing interest on the part of Fred Hutch to develop partnerships that
further its lifesaving and innovative research.

"We're looking for more creative academic-industry partnerships like this one
between Fred Hutch and GSK," said Ulrich Mueller, vice president of industry
relations and technology transfer at Fred Hutch.

Tapscott's research on FSHD, which provides the scientific basis for the
collaboration with GSK, was funded by Friends of FSH Research, the National
Institute of Neurological Disorders and Stroke, and the National Institute of
Arthritis and Musculoskeletal and Skin Diseases.

Financial terms of the Fred Hutch-GSK partnership were not disclosed.

At Fred Hutchinson Cancer Research Center, home to three Nobel laureates,
interdisciplinary teams of world-renowned scientists seek new and innovative
ways to prevent, diagnose and treat cancer, HIV/AIDS and other
life-threatening diseases. Fred Hutch's pioneering work in bone marrow
transplantation led to the development of immunotherapy, which harnesses the
power of the immune system to treat cancer with minimal side effects. An
independent, nonprofit research institute based in Seattle, Fred Hutch houses
the nation's first and largest cancer prevention research program, as well as
the clinical coordinating center of the Women's Health Initiative and the
international headquarters of the HIV Vaccine Trials Network. Private
contributions are essential for enabling Fred Hutch scientists to explore
novel research opportunities that lead to important medical breakthroughs. For
more information visit or follow Fred Hutch on Facebook, Twitter
or YouTube.

Dean Forbes
206-605-0311 cell
206-667-2896 desk

SOURCE Fred Hutchinson Cancer Research Center

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