[Congressional Record Volume 155, Number 142 (Monday, October 5, 2009)]
[Senate]
[Pages S10105-S10106]
From the Congressional Record Online through the Government Publishing Office [www.gpo.gov]




                             BATTEN DISEASE

  Mr. DURBIN. Mr. President, I recently heard from the friends and 
family of a young boy in Illinois named Jasper Duinstra who was 
diagnosed earlier this year with late infantile neuronal ceroid 
lipofuscinosis, often referred to as Batten disease.
  Batten disease is a rare and devastating childhood disease, affecting 
between 2 and 4 of every 100,000 live births in the United States. Due 
to an enzyme deficiency, waste accumulates in the child's brain, 
causing cells to become dysfunctional and eventually die. This results 
in seizures, mental impairment and progressive loss of sight and motor 
skills. Sadly, there are no known treatments to halt or reverse the 
symptoms of Batten disease and the disease is fatal.
  The physical, emotional, and financial toll of this disease is 
devastating. But Jaspers family and friends have rallied around him to 
create a sense of hope and have motivated many people in their 
community to team together in pursuit of a cure for this disease. There 
is a sense of urgency behind the need to increase funding for Batten 
and

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other rare childhood disorders. The urgency grows everyday when mothers 
and fathers watch their childrens health rapidly deteriorate.
  Jasper Duinstras friends and family have formed a nonprofit 
organization called Jasper Against Batten, and children from 20 
elementary schools have mobilized one of the largest kids helping kids 
initiatives on behalf of this group. The money these students are 
raising will go toward research for a treatment and maybe one day, a 
cure.
  Jasper Duinstra is just one of the thousands of children who need our 
support in the fight against Batten disease and other fatal orphan 
diseases. In addition to private efforts to raise money through groups 
like Jasper Against Batten, the National Institutes of Health is 
funding research in Batten disease and other rare diseases.
  While the number of Americans affected by any particular rare disease 
may be very small, over 6,000 rare diseases have been identified. Taken 
together, these diseases affect about 25 million Americans. The burden 
of these diseases is great, not only because of the number of people 
affected but because too often there are few or no treatments available 
for people suffering from them.
  The Orphan Drug Act provides some incentives for drug companies to 
develop drugs for rare diseases. This has been a successful effort, and 
more than 200 drugs and biological products for rare diseases have been 
brought to the U.S. market. However, despite the success in finding 
treatments for some rare diseases, others such as Batten disease have 
seen relatively little progress over the last several decades. Today, 
there are promising experimental treatments, but they need to find 
their way more quickly to these children who are rapidly deteriorating.
  In addition to searching for new and more readily available 
treatments, some scientists are also searching for ways to use existing 
drugs to treat rare diseases that have few options for treatment. A 
Chicago-based research foundation called Partnership for Cures has 
teamed with Jasper Against Batten and is now doing just that. In 
partnership with the National Institutes of Health, they are currently 
screening thousands of drugs that have already been approved by the 
Food and Drug Administration to see whether there are beneficial side 
effects that could slow down the progression of rare orphan diseases, 
starting with Batten.
  I know Jasper's family is heartbroken, and I commend his family, 
friends, and the Chicago community for responding to tragedy with 
action. With biomedical researchers, clinicians, and community 
partners, Jasper's family is leading the fight to find a cure for 
Batten disease and for quicker access to treatments for children with 
many fatal orphan diseases.

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