And, though they never reached the same level of nimbleness as healthy mice did, they were fierce candidates for the title of most improved players. Over the course of the testing, they achieved nearly the same degree of improvement in their before and after coordination scores as healthy mice did. In fact, almost second for second.
It seems we may have corrected some sort of motor learning deficit in the diseased mice, Pearce said.
While optimistic about these findings, Pearce stressed the importance of reminding affected families that this work is preliminary.
Much research is yet needed, Pearce said. The prospect of offering this sort of investigational medicine to affected children is still years out.
Still, he is further encouraged that a drug called Talampanel very similar to the blocking compound used by his team in Rochester is currently in phase II clinical trials for treating epileptic seizures.
Unlike most anticonvulsants, which typically target cells known as NMDA receptors, Talampanel works by partially blocking AMPA receptors.
This orally active new drug would be an obvious choice for clinical trials with juvenile Batten disease patients, Pearce said. Especially since they routinely suffer seizures, as well.
Though only 150 children in the United States suffer Batten disease, Pearce is hopeful that this research will also likely also inform research efforts for a dozen or so of its cousins other uncommon genetic diseases, each characterized by a glitch with the cells toxin-ridding mechanisms, the lysosomes.
Some of these lysosomal-storage diseases, as theyre called, include Krabbe disease (to which Buffalo Bills quarterback Jim Kelly lost his son, Hunter, in 2005), Tay-Sachs and metchromatic leukodystrophy.
Our research might indeed open doors for learning how other neurological disorders might benefit from drugs that regulate AMPA receptors, Pearce said.
|Contact: Becky Jones|
University of Rochester Medical Center