DRUG TRIAL FOR JUVENILE BATTEN DISEASE

Finally, after 180 years, we may be able to do something about Juvenile Batten disease. Even though we know what happens in the disease process, we still do not fully understand the cause of the disease. David Pearce, Ph.D. and his lab have been working on this aspect for quite some time and may have discovered some of the answers. It appears that the disease causes the body to fight itself by the way of an “autoimmune response”. The question then becomes; can anything be done about this? Please see attached sheet from Dr. Pearce.

BDSRA funded Dr. David Pearce to test the use of a drug, Mycophenolate Mofetil (CellCept) on mice to determine if it has any affect on the progression of the disease. The results show it does have a positive effect on the progression of the disease in mice. Will it do the same in children? That has to be determined.

This drug is often used for children who have had organ transplants and is FDA approved. Although it is considered to be safe, there is always the possibility of a child having an adverse reaction, just as there is with any drug.

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 BDSRA does not know how many children with Juvenile Batten disease would be included in the trial, what the criteria for inclusion will be, the start date or duration of the trial, how selection will be made or anything else. All this is yet to be determined. There are no guarantees that any of this will work, either.

What BDSRA does know is that there is no funding available to do this trial. The estimated start up cost for the trial is $500,000.00. We also know that if this trial is to happen, it will be up to us to make it happen. The actual cost to start the trial is being worked out by the Batten Center Group.

In today's' world, $500,000.00 is not a lot of money. To many of us it does seem like an enormous amount, but if everyone will join together, we can make it happen. We finally have the opportunity to do something for the children. Let's not let this get away from us.

If you are interested in contributing to the clinical trial, donations may be sent to the BDSRA, 166 Humphries Dr. , Reynoldsburg , OH , 43068 .  Make sure to mark all donations for "juvenile trial."   If you are interested in fundraising for the clinical trial, you may call the BDSRA office at 1-800-448-4570 and get signed up to "Make the Juvenile Trial Happen."