According to clinicaltrials.gov, there are currently almost
clinical studies for heart disease, more than 10,000
studies for diabetes, almost 6,000
studies for breast cancer, and more than 4,000
studies for leukemia. For decades, we have been raising money
for research for some of the world's most common diseases. And it
worked (although there is still work to be done). Many people are
living longer because of what we have learned from clinical studies
of these diseases.
But there is another side to this story. Clinicaltrials.gov
316 clinical studies for osteosarcoma and only 19 studies for
Lennox-Gastaut Syndrome (LSG), a rare and severe form of
According to the National
Organization for Rare Disorders (NORD), there are approximately
7,000 rare diseases. For a disease to be considered rare, it must
affect fewer than 20,000 people. NORD estimates that these 7,000
rare diseases affect nearly 30,000 million Americans. People with
rare diseases often find themselves struggling to find treatment
options because there are often few--if any--approved treatments.
Because these diseases affect fewer people, they often also suffer
from a lack of clinical research.
It is often difficult to justify spending critical and often
limited research dollars on a study of an incredibly rare disease
because the impact may be relatively small. But define impact to a
parent of a child with osteosarcoma. Although
osteosarcoma is the most common bone cancer in children, as far as
cancers go, it is rare, affecting just 400 children younger than
age 20 each year.
If you want to talk about impact, let's talk about Zach Sobiech, who
passed away earlier this year after a four-year battle with
osteosarcoma. Zach wrote a song called "Clouds" as a way
to deal with dying, and in doing so, he moved so many of
us--not just to tears but to action. The Zach Sobiech
Osteosarcoma Fund at Children's Cancer Research Fund is
bringing together a team of scientists to conduct a multi-phase
clinical trial for osteosarcoma. All donations made to Zach's fund
go directly to research.
On a more personal note,
my great nephew, Justin, has LGS. At nearly 4 years old, Justin
cannot communicate or follow simple commands. We don't know when
he's feeling hurt, hungry, or sick. He can have as many as 30
seizures a day, and that's after taking up to eight medications to
"control" the seizures. The seizures impact his ability to learn,
and he suffers from significant intellectual delays. So many of the
treatments and tests Justin has undergone are considered
experimental, and there is simply not enough research done in LGS
to know how to stop the seizures.
As frustrating as the lack of research is for families
struggling with rare diseases, there have been some promising
developments. The National
Pediatric Research Network Act was signed into law in November.
According to U.S. Sen. Sherrod Brown (D-OH), the National
Institutes of Health (NIH) dedicates only about 5 percent of its
annual extramural research budget to pediatric research. The Act
will provide for the establishment of a National Pediatric Research
Network, which will allow children's hospitals around the country
to share data and conduct research into new treatments and possible
cures for rare diseases.
NIH already has an
Undiagnosed Diseases Program, which focuses on advancing
medical knowledge about rare and common diseases. To learn more
about this fascinating and elite program, check out this
60 minutes special from 2012.
So many people need our help, and clinical research can--and
has--made a significant impact on the lives of so many. We must
continue to be voices for kids like Zach and Justin with the hopes
that every dollar raised toward research is one tiny step closer to
finding answers and cures.
As health communicators, it's easy to talk about the well-known
diseases that garner money and celebrity endorsements. But we must
remain diligent in our work for all diseases and conditions-no
matter how small the population. Because someone's life depends on