Jasper Against Batten Fund

Batten Disease-We are pleased to announce that our hard work has paid off!

A new gene therapy clinical trial began December 7, 2010 at Weill Cornell Medical Center in NYC to treat this currently fatal childhood disease. The organizers of the Jasper Against Batten Fund have raised over $1M in the last 18 months and PFC has worked closely with Weill Cornell researchers to get the funding and approvals in place. Keep your fingers crossed that this repurposed gene therapy will reverse the brain cell destruction that cripples and then kills these otherwise normal children!

Jasper Against Batten Fund

On April 27, 2009 Partnership for Cures created the Jasper Against Batten Fund at Partnership for Cures to fight Batten disease. This fund is set up in honor of Jasper with his Dad Jasper Duinstra, a 4 year old boy recently stricken with Batten Disease, and in support of the many other children afflicted with this catastrophic disease. Jasper was recently diagnosed with the Late Infantile Neuronal Ceroid Lipofuscinosis (LINCL) form of Batten Disease. LINCL patients and other Batten patients suffer from this cruel and rare childhood disorder for which there is currently no known cure. Over time, children affected by Batten disease suffer from epilepsy, mental impairment and progressive loss of sight and motor skills.

In October we funded a joint project at University of Iowa and Rush University Medical Center testing the last stages of a gene vector therapy that would then be ready to move towards a human clinical trial.

We are working with researchers at Johns Hopkins Medical Center to screen a library of over 2500 already approved drugs that might help Batten patients. We are hopeful that we will have candidate drugs that could be used by physicians treating Batten patients in early to mid 2010.

We are working towards funding a grant at a research facility here in the US that has a different gene therapy that could be easily delivered to Batten patients to replace their missing enzyme. We are hoping to fund intermediate stage research with this grant that could propel this research to patients within the next two years.