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Medical Compassionate Corporate Chemistry Provides Hope for FOP

The Greek mathematician and inventor Archimedes
(c.250 B.C.) exclaimed, "Give me where to stand and I will
move the Earth." More than two thousand years later, a famous
advertisement proclaimed, "When the human element is added
to the equation, everything changes." May 30, 2007 will
forever be noted in the annals of FOP history as one of those
rare and defining moments when one could hear and feel the
earth-shattering movement of something one could barely see
- the massive and powerful engines of corporate capitalism
locking in place on a new track, being nudged into a purposeful
articulation by revelational biology, compelling chemistry and most

importantly by the undeniable valence and compassion of the
human spirit.

While no miracle drugs have yet been developed, promised, or

even begun, one of the highlights of 2007 was a unique and mind-
boggling meeting on FOP with a focus on FOP patients, sponsored
and hosted by the research division of a major US pharmaceutical
company. The company's senior vice president and director of
worldwide development was instrumental in organizing the historic
meeting which took place at corporate research headquarters near
Philadelphia, and was attended by senior corporate executives,
research scientists, medicinal chemists and physicians. Three FOP
patients (an adult, a teenager, and an infant) were invited to attend
with family members, and provided, in the words of the senior
executive, "a powerful day that we hope will lead to an improved
approach to addressing a terrible unmet medical need." And, she
continued, "We all feel deeply passionate about helping patients."

The medicinal chemists of the pharmaceutical company have

developed inhibitors for overactive receptors very similar to
the kinds of receptors that cause FOP. Motivated by the FOP
gene discovery, the compelling need of FOP patients, and the
widespread utility of an orphan drug for FOP that might be
used to treat more common conditions of heterotopic ossification,
the company was eager to explore the feasibility of entering
a new frontier of pharmaceutical development. During the
remainder of 2007, tantalizing collaborative experiments,
conducted at the pharmaceutical company and in the FOP
laboratory, provided important insight into possible drug
development for FOP. This exciting work continues. The Earth
has begun to move, one mountain at a time.

Dr. Eileen Shore, Ph.D., Professor of
Orthopaedic Surgery