Batten Disease

Floor Speech

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 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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