[Congressional Record (Bound Edition), Volume 149 (2003), Part 5]
[Senate]
[Pages 5744-5745]
[From the U.S. Government Publishing Office, www.gpo.gov]




                 FIBRODYSPLASIA OSSIFICANS PROGRESSIVA

  Mr. CORZINE. Mr. President, I rise to call attention to a little-
known, little-understood, devastating ``orphan'' disease fibrodysplasia 
ossificans progressiva, or FOP--which strikes children between age 2 
and 10.
  Normally, when a young child sprains a wrist or an ankle, or bruises 
a knee, there's a natural healing process. But children with FOP--
develop catastrophic bone spurs at the site of the injury that continue 
to grow, encasing major organs and exerting painful, life-threatening 
pressure. According to Dr. Frederic Kaplan, an orthopedic surgeon at 
the University of Pennsylvania, the worldwide center for FOP research, 
the average lifespan for people with this dreadful disease is about 45 
years. But most sufferers are wheel-chair bound by age 20, breathing 
with the greatest difficulty, unable to feed or dress themselves.
  Here's the sad problem: there are perhaps 300 FOP cases in the 
world--at least 12 in my state of New Jersey, 16 in New York, and 13 in 
Pennsylvania. This is the orphan of all orphan diseases. So we need to 
put a human face on this. For me, that face belongs to 10-year old 
Whitney Weldon, of Westfield, New Jersey. When first diagnosed two 
years ago, Whitney did all the things most all children do--run, play 
ball, skip down the street. Now she cannot lift her arms over her head. 
But she is able to ride a special bicycle and enjoy the art she loves, 
and time with her best friend. We want to give her the chance for more 
time, and to do that we need money for research. Right now Whitney's 
only treatment consists of painkillers and anti-inflammatory steroids. 
Nothing stops the bone growth.
  Dr. Kaplan and his research partner at the University of 
Pennsylvania, Dr. Eileen Shore, have received a little more than $1 
million over 4 years from the National Institutes of Health for 
research into the gene that causes FOP and to determine the pathways by 
which this abnormal gene causes extra bone production. This funding is 
a tribute to their progress so far, but there is still such a long way 
to go. Dr. Kaplan's annual budget is $1 million. About 20 percent of 
that comes from NIH funding--the other 80 percent comes from families 
and friends. And I'll tell you something interesting--Dr. Kaplan says 
that even though FOP affects only one person in 2 million, the

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answers that can be found in continued research can shed light on 
osteoporosis and extra-bone formation that occurs after head or spinal 
cord trauma. So there is the very real potential of beneficial effects 
for many millions of people.
  There are some promising avenues. Adult stem cell research and 
examination of bone marrow from FOP sufferers have yielded possible 
directions to pursue. Wider stem cell research would be exponentially 
more helpful.
  Whitney's parents enable her to live as normal a life as possible for 
as long as possible. And she shares fun and confidences with Mackenzie 
Roach, her friend since kindergarten. She also shares one of life's 
extraordinary connecting bonds with Mackenzie. Stephen Roach, 
Mackenzie's father, was killed on September 11 when terrorists bombed 
the World Trade Center. Stephen was very involved in raising funds and 
awareness for FOP. In his memory, Mackenzie's family has created the 
Stephen L. Roach Fund for FOP Research, which to date has raised more 
than $800,000.
  Last March, President Bush declared 2002-2011 as National Bone and 
Joint Decade. That is a very hopeful development, and hope goes a long 
way. When we join that hope with a sustained focus on finding a cure 
for FOP, we will go even further.

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