The FOP gene discovery is relevant to every disease that affects
the formation of bone and every disease that affects the
formation of the skeleton. Answers to FOP are relevant to many
common skeletal conditions such as unwanted bone growth
that forms after hip replacement surgery, athletic injury,
brain injury, spinal cord injury, soft tissue injury, burns, war
wounds, valvular heart disease, and even bone spurs from
osteoarthritis. Eventually, through progress being made
in FOP research, some of which has been highlighted in this
report, it might be possible to harness the FOP gene,
and create bone in a more controlled way where it is
desperately needed such as in fractures that do not heal,
surgical spine fusions, severe bone loss from trauma,
osteoporosis, tumors, and congenital malformations.
The FOP gene discovery is a great beacon of hope for all
of us in the FOP community, and for all of us in a much
wider global community who are affected with common skeletal
disorders. Better treatments for FOP are not just a dream - they
are now a likelihood, and a cure is a distinct possibility.
During this past year, we have begun to turn the FOP
gene discovery into insight, and insight into development.
wider global community who are affected with common skeletal
disorders. Better treatments for FOP are not just a dream - they
are now a likelihood, and a cure is a distinct possibility.
During this past year, we have begun to turn the FOP
gene discovery into insight, and insight into development.
We have set our sights on the distant horizon. But, we won't
get there by wishing it, and we cannot do it alone. We need
your help.
The Lachance family from Grand Bend, Ontario,
Canada visits the FOP Laboratory in Philadelphia.
What needs to be done? During this past
year, we have expanded the network of physicians and scientists
who are working on FOP through targeted identification and
funding of those who can help most and who can help the fastest
- wherever they may be. We need to expand that program, and we
need your help.
What needs to be done? During the
past year, we have begun to develop cellular and animal models
to determine how the renegade FOP gene acts at the
molecular level. We need that knowledge and those critical
models to design new drugs and to test them. We need
to continue that work, to expand it, and we need your help.
What needs to be done? During this
past year, we have begun to crystallize the mutant FOP
What needs to be done? During this
past year, we have begun to crystallize the mutant FOP
protein to study its atomic structure, its catalytic
domains, and its interactions with other key proteins in the
molecular relay-switch that triggers catastrophic new bone
formation. Such knowledge will be needed to custom-design
the best medications to block the mutant switch. We need to
continue that work, to expand it, and we need your help.
What needs to be done? During the
past year, we have begun to model the active site of
What needs to be done? During the
past year, we have begun to model the active site of
the broken switch, and are beginning to use that knowledge
to design the best methods to block, jam, or bypass it. We
need to continue that work, to expand it, and we need your help.
What needs to be done? During the
past year, we have begun to study the mechanisms by which
the inflammatory microenvironment of an injury triggers the
renegade FOP switch to form new bone. When we understand
that better, we'll be able to use that knowledge to apply the brakes
to a run-away process. We need to continue that work, to expand
What needs to be done? During the
past year, we have begun to study the mechanisms by which
the inflammatory microenvironment of an injury triggers the
renegade FOP switch to form new bone. When we understand
that better, we'll be able to use that knowledge to apply the brakes
to a run-away process. We need to continue that work, to expand
it, and we need your help.
What needs to be done? During the
past, year, we have begun to scour the world's available libraries
of medicinal compounds to identify those that may block
What needs to be done? During the
past, year, we have begun to scour the world's available libraries
of medicinal compounds to identify those that may block
the abnormal FOP switch and its downstream molecular circuits.
We need to continue that work, to expand it, and we need
your help.
These goals and the tasks they imply are easy to articulate
and all have been started, but they need funding to be
These goals and the tasks they imply are easy to articulate
and all have been started, but they need funding to be
fulfilled.
We need your help to continue these vital programs and
We need your help to continue these vital programs and
ensure their success, to do more, to do it faster, to expand our
horizons, and to make sure that no clue is ignored.
FOP is an uncommon condition of uncommon brutality,
but there is finally a chance to do something intelligent
and rational to interrupt the inexorable progression of what has
FOP is an uncommon condition of uncommon brutality,
but there is finally a chance to do something intelligent
and rational to interrupt the inexorable progression of what has
been described as a "horrible nightmare disease." Chemistry
combined with compassion will lead to orphan drug development,
to more effective treatments for those with FOP, and for those
with more common forms of heterotopic ossification. We have
worked hard to get this far, and your generosity has helped
get us there. But, we need your help to go farther. We
all know what needs to be done, and we need your help to
prevail.
As it is said many times, cause and cure are the two
words that propel us and provide the guiding principle for all we
do: to discover the cause of FOP, and to use that knowledge to
develop effective treatments and eventually a cure. Many said
that identifying the gene mutation that causes FOP couldn't be
done.
In 2006, with your help, we reached the summit of a great
mountain and discovered the genetic cause of FOP. In 2007, we
headed for a higher and more distant summit - the treatment and
cure of FOP.
We need to continue that journey, to expand it, and we need your help.
As it is said many times, cause and cure are the two
words that propel us and provide the guiding principle for all we
do: to discover the cause of FOP, and to use that knowledge to
develop effective treatments and eventually a cure. Many said
that identifying the gene mutation that causes FOP couldn't be
done.
In 2006, with your help, we reached the summit of a great
mountain and discovered the genetic cause of FOP. In 2007, we
headed for a higher and more distant summit - the treatment and
cure of FOP.
We need to continue that journey, to expand it, and we need your help.