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The combination of imatinib and fampridine is being investigated as a neoadjuvant therapy for patients with gastrointestinal stromal tumors (GIST) specifically harboring KIT exon 11 mutations. Imatinib is a well-established tyrosine kinase inhibitor that targets the KIT and PDGFR receptors, which are frequently mutated and drive tumor growth in GIST. Fampridine (4-aminopyridine) is a voltage-gated potassium channel blocker traditionally used to improve walking in patients with multiple sclerosis. In this oncology context, fampridine is being repurposed to potentially enhance the antitumor activity of imatinib. The University of California, San Diego is conducting a Phase I study to determine the safety, maximum tolerated dose, and preliminary efficacy of this combination in the neoadjuvant setting before surgical intervention.
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