The 2011 Neurofibromatosis Conference doesn’t officially start til Saturday, but the action kicked off early Wednesday & Thursday with a specially convened workshop of 20 of the world’s experts in the arena of NF1 learning disabilities. Two thirds of kids with NF1 are affected by some type of learning disability, and the Children’s Tumor Foundation has helped spearhead significant progress in this area the past few years which has included the first clinical trials testing the drug Lovastatin. CTF has played a key role in driving discussions and collaborations since 2006 through the group LeaDNet the Learning Disabilities Network). This week’s LeaDNet workshop headed by Dr. Maria Acosta (Children’s National Medical Center) and Dr. Alcino Silva (UCLA) considered the state of the learning disabilities field today and began to define a strategic plan to accelerate clinical progress in the next few years. Many topics were discussed in the packed day-and-a-half LeaDNet agenda. Firstly, there is a clear need for better diagnosis of the variety of learning disabilities seen in NF1, and to address this, clinicians will now to share more data and figure out standardized ways to classify patients, including firming up potential links with autism and ADHD. This is to be accomplished in part by increasing the use of neuroimaging.
In the arena of mice, there are now a large number of genetically engineered mouse models of NF1 learning disabilities. Researchers agreed it is important to pool this information and figure out which mouse models are most useful, and what aspects of clinical NF1 learning disabilities they represent. At the bench, researchers now think about NF1 learning disabilities as having potentially multiple genetic and molecular causes, which opens up the opportunity to explore new drug targets. Perhaps the most exciting discussions centered around how to diagnose NF1 learning disabilities in young infants. This will be particularly useful once drug or even gene-based therapies are available, and there is the possibility to ‘reverse’ the learning disabilities early in life.
The next steps for LeaDNet will be to expand the recommendations from the meeting and develop next steps including a workplan; and to develop a white paper of consensus recommendations from this week’s meeting. To maintain momentum, the LeaDNet group plans to meet again in early 2012. Look for more updates later this year!
