Dear Visitor,

Please let me open this letter by saying that the Sikorra family is amazingly blessed by this community of friends. Your endless words of encouragement, prayers, laughter and all that you do are what keep us together. Thank you.

We would like to take this opportunity to give you a few updates on some very exciting developments regarding the treatment of Batten disease…

What is Batten Disease? Its technical name is Neuronal Ceroid Lipofusinoes. It is a lysosomal storage disease. Only about 150 children in the United States are living with Juvenile Batten disease, many of them are siblings.

Over time, affected children suffer mental impairment, worsening seizures, and progressive loss of sight and motor skills. As you know, our two sons have this disease. Until recently, there was nothing they could do to slow the progression. However, after 180 years we may be able to do something about Juvenile Batten disease.

David Pearce, PhD, from the University of Rochester, has dedicated his talents, time and I must say, his heart to figuring out this disease. He is amazingly dedicated to the families with children suffering from Juvenile Batten disease. Recently, Dr. Pearce and his lab have discovered that this disease does have an “autoimmune response.” The question then becomes can anything be done about this? This is where it gets EXCITING.

The Pearce lab has been working with a drug called CellCept. This drug is used frequently for children who have had organ transplants and is FDA approved. They tested the use of this drug on mice to determine if it has any affect on the progression of the disease. The results show that it does have a positive effect. The progression of motor and coordination were examined and showed a significant slowing of degeneration. Will it do the same in children? That has yet to be determined.

Dr. Pearce and the group at the Batten Center at the University of Rochester Medical Center are preparing for a human trial. At this time they do not know how many children, what children, the start date or the duration of the trial.

What they do know is it will take approximately $500,000 to start.

The actual cost of the trial is still being worked out by the Batten Center Group. We are committed to getting this moving forward.

There is no guarantee that John and Ben will even be in the trial. We will, of course, do whatever is in our power to make that happen; however there are families and children like ours who are suffering with this beast.

If we can be a part of the treatment for any child with this disease, we will.

Please take time to contact us for references or to discuss what you can do to help these children with their disease.

Thanks,

The Sikorras