Friday saw a pre-conference meeting of the Children’s Tumor Foundation NF Preclinical Consortium (NFPC), a collaboration of six top-tier labs – at UCSF, House Ear Institute, Washington University, Cincinnati Children’s Hospital Medical Center and two groups at Harvard Medical School – each focused on genetically-engineered mouse models of different NF tumors. Individually, these are highly successful NF research groups; collectively NFPC is a preclinical drug testing powerhouse. Drugs are tested in parallel in the multiple tumor types of NF1 and NF2 to ensure that we do not miss an opportunity: if a drug is not effective in one NF tumor type we can see if it might be effective in another. NFPC is overseen by a committee of seasoned researchers, many from industry, some with 20+ years of experience in drug development. A $4M multi-year commitment for the Children’s Tumor Foundation, NFPC should maximize our chances of identifying drug candidates for NF clinical trials. So far NFPC has made tremendous strides, attracted a collaboration with Novartis and further industry partnerships are in planning.
An important element of advancing interesting preclinical drug candidates is to establish a link with the clinical trialists. That in mind after NFPC met Friday we had a one hour ‘crossover’ meeting with the physicians of the Phase II NF clinical trials consortium which is funded by the Congressionally Directed Medical Research Program. This group is a clinical trials version of NFPC connecting multiple clinical sites. Because of the variety of tumor types (plexiform, optic pathway etc.) and other manifestations (bone abnormalities, learning disabilities) seen in NF, an effort like this is required in order to recruit sufficient patients for a clinical trial in a timely manner. In the past year or so the Clinical Trials Consortium has launched 2 trials (for plexiform tumors and learning disabilities) and more are pending.
Scientists and clinicians can often live in two different worlds but the joint meeting of the 2 groups opened a dialog on the drugs in the preclinical pipeline, and those most likely to be of clinical interest. Though early days, this meeting emphasizes the importance of collaboration and open communication among our NF community to ensure that we advance promising drug treatment as quickly as possible to the clinic. We should see this theme continuing through the 2009 NF Conference.
