The Cure Tay-Sachs Foundation is the leading private funder of Tay-Sachs research committed to finding therapies and a cure for this 100% fatal and heinous disease. Many of us know the personal heartache of losing our children. Sadly, our kids never had a chance. We have been working to change that.

I am delighted to share that a clinical trial request has been submitted to the FDA for a new, potential treatment for Tay-Sachs disease. Known as IB1001, this type of drug has been used in compassionate-use studies in Europe for many years to treat a number of neurogenerative diseases and lysosomal storage disorders like Tay-Sachs, and has been demonstrated to be very safe, well-tolerated, and most of all, effective.

As you all know, a US clinical study with IB1001 would be this community's first opportunity to intervene in the quick decline of our Tay-Sachs kids. Finally, perhaps, a breakthrough for a disease where we have nothing available. This gives us hope!!

The FDA does a magnificent job of bringing oversight and cadence to the process of approving clinical trials for new drugs like IB1001 that have the potential to help so many of our patients and our families. However, since a majority of our kids die so young, it will be unethical to apply the typical "before and after" benchmarks to determine IB1001's effectiveness. Collectively, I know we all believe that the FDA will find a better way to assess the effectiveness of IB1001 given the "death in infancy" nature of our disease, and help give our kids their first treatment option as soon as possible. This gives us hope!!

I have reached out the management team of the company (IntraBio) sponsoring this clinical trial. They have confirmed that based the safety and efficacy observed in previous studies, they intend to conduct a clinical trial with IB1001 for the treatment of Tay-Sachs patients in Europe, and hope to be able to open the study to US patients. At this time, they could not comment further on the details of their interactions with the FDA.

As we all know, "Time is Brain" with Tay-Sachs. We as a community of 3000 strong are excited, motivated and ready to work with the FDA to get this trial approved expeditiously!!

Please send questions, comments or thoughts to: Rick.Karl@curetay-sachs.org