Dataset Information

Neurofibromatosis 2011: a report of the Children's Tumor Foundation annual meeting.

ABSTRACT: The 2011 annual meeting of the Children's Tumor Foundation, the annual gathering of the neurofibromatosis (NF) research and clinical communities, was attended by 330 participants who discussed integration of new signaling pathways into NF research, the appreciation for NF mutations in sporadic cancers, and an expanding pre-clinical and clinical agenda. NF1, NF2, and schwannomatosis collectively affect approximately 100,000 persons in US, and result from mutations in different genes. Benign tumors of NF1 (neurofibroma and optic pathway glioma) and NF2 (schwannoma, ependymoma, and meningioma) and schwannomatosis (schwannoma) can cause significant morbidity, and there are no proven drug treatments for any form of NF. Each disorder is associated with additional manifestations causing morbidity. The research presentations described in this review covered basic science, preclinical testing, and results from clinical trials, and demonstrate the remarkable strides being taken toward understanding of and progress toward treatments for these disorders based on the close interaction among scientists and clinicians.

SUBMITTER: Kalamarides M

PROVIDER: S-EPMC3282898 | biostudies-literature | 2012 Mar

REPOSITORIES: biostudies-literature

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Publications

Neurofibromatosis 2011: a report of the Children's Tumor Foundation annual meeting.

Kalamarides Michel M Acosta Maria T MT Babovic-Vuksanovic Dusica D Carpen Olli O Cichowski Karen K Evans D Gareth DG Giancotti Filippo F Hanemann C Oliver CO Ingram David D Lloyd Alison C AC Mayes Debra A DA Messiaen Ludwine L Morrison Helen H North Kathryn K Packer Roger R Pan Duojia D Stemmer-Rachamimov Anat A Upadhyaya Meena M Viskochil David D Wallace Margret R MR Hunter-Schaedle Kim K Ratner Nancy N

Acta neuropathologica 20111116 3

The 2011 annual meeting of the Children's Tumor Foundation, the annual gathering of the neurofibromatosis (NF) research and clinical communities, was attended by 330 participants who discussed integration of new signaling pathways into NF research, the appreciation for NF mutations in sporadic cancers, and an expanding pre-clinical and clinical agenda. NF1, NF2, and schwannomatosis collectively affect approximately 100,000 persons in US, and result from mutations in different genes. Benign tumor ...[more]

PMID: 22083253

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Project description:Although recent FDA approvals on ipilimumab and sipuleucel-T represent major milestones, the ultimate success of immunotherapy approaches will likely benefit from appropriate combinations with other immunotherapeutic and/or non-immunotherapeutic approaches. However, implementation of ideal combinations in the clinic may still face formidable challenges in regulatory, drug-availability and intellectual property aspects. The 2011 SITC annual meeting hosted a workshop on combination immunotherapy to discuss: 1) the most promising combinations found in the laboratory; 2) early success of combination immunotherapy in clinical trials; 3) industry perspectives on combination approaches, and 4) relevant regulatory issues. The integrated theme was how to accelerate the implementation of efficacious combined immunotherapies for cancer patients. Rodent animal models are providing many examples of synergistic combinations that typically include more than two agents. However, mouse and human immunology differ in a significant number of mechanisms and hence we might be missing opportunities peculiar to humans. Nonetheless, incisive animal experimentation with deep mechanistic insight remains the best compass that we can use to guide our paths in combinatorial immunotherapy. Combination immunotherapy clinical trials are already in progress and preliminary results are extremely promising. As a key to translate promising combinations into clinic, real and "perceived" business and regulatory hurdles were debated. A formidable step forward would be to be able to test combinations of investigational agents prior to individual approval. Taking together the FDA and the industrial perspective on combinatorial immunotherapy, the audience was left with the clear message that this is by no means an impossible task. The general perception is that the road ahead of us is full of combination clinical trials which hopefully will bring clinical benefit to our cancer patients at a fast pace.

Dataset Information

Neurofibromatosis 2011: a report of the Children's Tumor Foundation annual meeting.

Publications

Neurofibromatosis 2011: a report of the Children's Tumor Foundation annual meeting.

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