At the Neurobiology of Learning and Memory Laboratory (NLML) in Tampa, Florida, a research team led by Edwin Weeber Ph.D. have recently claimed unexpected results while investigating Alzheimer’s Disease.
The aim of the NLML’s research is to find a way to restore UBE3A function to neurons in order to address the learning and motor planning issues caused by lack of UBE3A. This is in contrast to research attempting to re-activate the (dormant) paternal copy of the UBE3A gene (e.g. the topoisomerase inhibitor research described above).
According to Edwin Weeber:
“…this proposal does not focus, necessarily, on understanding mechanisms of AS but rather treating AS.”
The NLML team have indicated that their initial investigations have suggested that the brain and the neurons form correctly in an individual with AS, indicating that the neurons could function normally if UBE3A function could be restored. Their research suggests that the cognitive disruption observed in Angelman syndrome may result from disruption in CaMKII signaling. Importantly, αCaMKII is not produced until after birth, suggesting that postnatal therapeutic intervention may be possible.
The team is exploring several approaches to rescue the learning and memory deficits in AS mouse models. They state that the issues caused by loss of UBE3A could be compensated for by finding drugs or strategies to enhance CaMKII activity in neurons. Thus the NLML proposal is that if drugs or strategies can be found to enhance activity in neurons, this might also benefit individuals with Angelman syndrome. What’s more, if they concentrate their research on drugs that were already FDA-approved the process of bringing an effective treatment to market could be significantly quicker.
To this end the team identified Minocycline – a tetracycline-class antibiotic – as having the qualities they needed. This drug is primarily used to treat acne and other skin infections such as MRSA, as well as Lyme disease. The NLML team claim that Minocycline’s superior ability to cross the blood–brain barrier is the main reason for its effectiveness in treating their AS mouse model.
The next stage of research is to conduct human trials on AS individuals to establish the treatment’s effectiveness and the correct dosage. These trials started in May 2012, with the results due to be published in February 2013.
For more information go to: http://www.weeberlab.com/clinical_trials.html