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NEW DRUG THERAPY DISCOVERED AT ROCHESTER

Investigators at the University of Rochester Medical Center have also uncovered a second drug that may be promising for Juvenile Batten disease in terms of brain receptor changes. The study, highlighted in the January edition of Experimental Neurology, explains how investigators improved the motor skills of feeble mice that model the disease, helping them to better their scores on successive coordination tests.  

http://www.urmc.rochester.edu/pr/news/story.cfm?id=1821  

We contacted the team at Rochester who informed us:
"The article is about a drug (compound from a company in Hungary) without even a real name yet – it’s called EGIS-8332. Not tested in humans, but there’s a similar drug called Talampanel (for seizures) that they’re testing in adults. Dr. Pearce’s hope is that one day, he can get Talampanel’s company to agree for him to try it in mice, and then, years out, hopefully humans". 

As Dr Sara Mole at UCL advises: The new drug EGIS-8332 will almost certainly need to be tested for safety in humans before it can be used in a trial in JNCL patients. It will also be useful to compare the effects of EGIS-8332 and Talampanel in the same mouse model. 

While optimistic about these findings, Dr Pearce stresses that this work is preliminary. Work is still going on to see if this drug has managed to protect brain cells or change the course of the disease within the brain. The drug acts to block the effects of having too much glutamate present within the brain, some thing which appears to happen in several forms of Batten disease and is the subject of investigation in Dr Pearce and Dr Cooper’s labs. 

We asked Dr Pearce at Rocester: Is this drug to be trialled on other types of NCL other than JNCL? and were advised:  AMPA receptor dysregulation which EGIS-8332 targets has not been studied in mouse models of other NCLs yet.   

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