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The Year Ahead in NF Research: Preclinical Drug Testing

By December 28, 2010December 18th, 2023Awareness, Science & Research

The following is excerpted from an article by Dr. Kim Hunter-Schaedle in the Foundation’s Holiday Newsletter.

In order for a drug to be deemed viable for clinical trials it is necessary that it first return promising preclinical results. In 2006, preclinical drug testing was identified as a major gap in NF research. Today, it is the Children’s Tumor Foundation’s largest area of focus through our NF Preclinical Consortium (NFPC) and Drug Discovery Initiative (DDI) Awards. The major NF trials of the past few years had their origins in positive findings from preclinical testing – including Lovastatin for NF1 learning disabilities trials and both lapatinib and rapamycin for NF1 plexiform tumors trials.

Beginning in 2009, CTF’s NF Preclinical Consortium took on a major challenge: to test drugs in parallel in models of different NF1 and NF2 tumors. Guided by an Advisory Board heavy in industry expertise, the NFPC Centers have conducted a sophisticated level of drug testing that, if positive, will translate to the clinic. NFPC has built up a growing pipeline of candidate drugs and established collaborations with companies including Novartis, Genentech and Avila Therapeutics. Building on these accomplishments, in November 2010 CTF announced the intent to support expansion (to seven sites) and continuation (for two more years) of NFPC with an addition $3.75 million commitment through 2013. This will be a competitive expansion: both new and existing Centers may apply.

The DDI Awards program, offering funding for preclinical drug testing, continues to flourish. We have now funded over 35 DDIs, an investment of $900,000. DDI success
is measured by a few key parameters: whether industry collaborations are established (20 to date), whether follow-on funding is secured from NIH, DOD or elsewhere ($4 million to date), and whether the studies are published (18 to date). DDI has jump-started a number of ideas that are new and unusual, and at such an early stage they are not likely secure funding elsewhere. DDI also helps investigators access drugs from industry through our DDI Toolbox. For example, through our ad hoc Schwannomatosis Awards program, we funded Dr. Larry Sherman (Oregon Health Sciences University) to develop the first mouse model of schwannomatosis pain, helped access appropriate drugs for testing from AstraZeneca and Pfizer, and through DDI Award funding we support the drug testing.

DDI Awards will continue into 2011. We hope to see some of these ideas advancing to the clinic, and to support advancement of these projects to the clinic through our Advanced DDI Awards and Clinical Research Awards programs.