Why Children's National Medical Center, and why the Cooperative International Neuromuscular Research Group?
It is a coincidence, not a cause, that both families' children are being seen at
Children's National Medical Center.
The reality is that the Children's research, led by Dr. Eric
Hoffman, who was on the team at Harvard who discovered the dystrophin
gene in 1986 (and the first scientist to clone the gene), is among
the most aggressive in the world. As clearly one of the top two or
three most accomplished and published scientists in this arena, Hoffman
leads one of the most sophisticated and cutting-edge laboratories conducting
genetic research, and his passion for decades now has been to drive
treatments into the marketplace for this generation of sufferers of
Duchenne Muscular Dystrophy.
While this may represent an oversimplification,
much of the great promise of Hoffman's research is that he, almost alone in the
scientific universe, has the capability (and financial muscle in his
large lab) to unravel the cascade of the genetic expression of Duchenne Muscular Dystrophy.
This work is leading to multiple targets for the broader scientific
community to “shoot at” as treatments (and even potential
cures) for DMD. Because of Hoffman's research, DMD sufferers
needn't have all their eggs in the basket singularly of fixing
the flawed dystrophin gene either through gene therapy or manipulation
of stem cells.
Admittedly, some of the treatments that could
arise from this research may be long-term goals. And parents of Duchenne Muscular Dystrophy kids, no matter the age,
are filled with desperate needs for research that will, at a minimum,
slow the progression of the horrible DMD curve while the ultimate 'cure' is
being sought. Hence the Foundation to Eradicate Duchenne's commitment to the
Cooperative International Neuromuscular Research Group, an elaborate
network of 20 institutions in 10 countries actually engaged in
clinical
trials on kids with Duchenne Muscular Dystrophy. The Foundation to Eradicate Duchenne is the largest private contributor
to this network.
The Cooperative International Neuromuscular Research Group was formed by Dr. Diana Escolar, Erik
Henricson and Dr. Eric Hoffman during the year 2000, lead since its
conception by Dr. Escolar.
The immediate purpose of
CINRG is to take FDA approved drugs and other
non-regulated supplements (such as sports-medicine supplements) that
seem to delay the progression of Duchenne Muscular Dystrophy in laboratory animals and run
trials on DMD kids to see if they have a similar effect. While most
clinical trials take about 3 to 5 years to complete, this group has
developed and is conducting 7 protocols in the 3 years of its existence
and there are some encouraging early results The current thinking is
that DMD treatment will not be one 'silver bullet,' even
if gene therapy is ever a reality, but rather a combination (cocktail)
of these drugs and supplements and perhaps other genetic modifying
therapies. Certainly, these first trials are steps along the way that
can improve the quality and length of lives for children (and adults)
with Duchenne Muscular Dystrophy.
Support for the clinical trials network is
absolutely essential. Many parents and organizations focus their
efforts on the long-term goals
of gene and cell therapies that will 'fix' the genetic
flaw, and we applaud this. But there are two points to be made. The
first is that we have heard scientific presentations from researchers
around the world, and none of them, even the most optimistic among
them, is willing to wager that full-fledged genetic repair of the dystrophin
gene, or full-fledged dystrophin expression through whatever means,
is going to be available in the marketplace during the coming five
or six years. None of them. Secondly, even if some magical gift should
fall down from the sky, the approval process of the Food and Drug Administration
is extremely rigorous, expensive, and time-consuming.
Clinical trials are expensive, very expensive. They involve scores
of highly trained professionals, hundreds of thousands of dollars worth
of equipment to measure muscle strength, software programs to ensure
that every kid is being evaluating according to the same standards,
and a sustained effort to grow an infrastructure that can pass the
muster of difficult governmental processes. In the Duchenne Muscular
Dystrophy world, there is only one alternative for this network: the
Cooperative
International Neuromuscular Research Group. We are of the belief
that if CINRG were to wither on the vine for lack of sufficient funding,
there is little hope for major breakthroughs to come to the market
that will impact this generation of those afflicted with Duchenne Muscular Dystrophy. Fortunately,
due to governmental earmarks and private donations, the
CINRG network
is growing, and more drugs with encouraging potential are being lined
up for trial in the coming year.
Visit the
Cooperative International Neuromuscular Research Group for more
information and current trials. Not only are funds necessary
to sustain CINRG, but the patient pool of candidates for trials is
essential, and we encourage all parents of Duchenne Muscular Dystrophy kids to consider whether
their children – and the entire Duchenne Muscular Dystrophy community – could benefit
from enrollment in any of the various clinical trials.
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Children's National Medical Center | The Cooperative International Neuromuscular Research Group
