Road to a Cure

At our last golf tournament Lance Johnston who is the Executive Director of the BDSRA made a bold prediction of being able to have significant treatment or cure for Batten’s Disease in two years. This type of public prediction for Lance is very uncharacteristic of him. Lance is a quiet unassuming person who is very careful about what he says in public.

Given this background, we attended the BDSRA conference in July with the purpose of learning more about developments from researchers involved in this ground breaking discovery. During the conference we were able to spend time with a researcher from the University of Iowa who is developing the procedure Lance referred to at the golf tournament.

For years, researchers at the University of Iowa have been working with gene therapy as a method to repair the brain cells that Batten Disease affects. By repairing these cells, it is believed that the motor functions lost by this disease and the general health of the child can be returned to pre disease onset. So far, researchers have seen results of this type.

The main stumbling block with this type of treatment has been delivering the gene therapy medicine to the brain without drilling through the skull. Well, now the University of Iowa researches believe they have found a way to use the brain’s natural defensive mechanisms to deliver the gene therapy treatments. This development is huge not only for Batten Disease but will also impact other brain diseases.

To date, research has been successful on various types and sizes of animal models. Current plans are to continue monitoring the process of these models for the remainder of 2010. If the results are positive, as expected, researchers will begin the process of applying this research to human trials in early 2011. This means working closing with the FDA and ensuring they are adhering to the various government regulations

Paula and I felt it was important to inform you of this ground breaking development and potential cure for Batten Disease. Over the years, proceeds from the Melissa Froio Foundation have been used to fund research at the University of Iowa. We truly appreciate your support and generosity to help make this situation possible. This means your support is leading to a major breakthrough for the cure to Batten Disease and potentially helps other brain diseases.

Click here to view the summary of this research written by Dr. Colleen Stein who was kind enough to spend a few hours with us at the BDSRA conference explaining her research and critiquing our notes and understanding of their work. We encourage you to read this as it provides a detailed overview of their work. Please feel free to send me any questions or comments you may have for Dr. Stein and I will ensure she responds to them.

We are now on the ‘Road to a Cure” and could not be there without your help and support. Again we thank you and hope you continue to support us in the future.