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Ref Type Journal Article
PMID (35046062)
Authors Hoes LR, van Berge Henegouwen JM, van der Wijngaart H, Zeverijn LJ, van der Velden DL, van de Haar J, Roepman P, de Leng WJ, Jansen AML, van Werkhoven E, van der Noort V, Huitema ADR, Gort EH, de Groot JWB, Kerver ED, de Groot DJ, Erdkamp F, Beerepoot LV, Hendriks MP, Smit EF, van der Graaf WTA, van Herpen CML, Labots M, Hoeben A, Morreau H, Lolkema MP, Cuppen E, Gelderblom H, Verheul HMW, Voest EE
Title Patients with Rare Cancers in the Drug Rediscovery Protocol (DRUP) Benefit from Genomics-Guided Treatment.
Journal Clinical cancer research : an official journal of the American Association for Cancer Research
Vol 28
Issue 7
Date 2022 Apr 01
URL
Abstract Text Patients with rare cancers (incidence less than 6 cases per 100,000 persons per year) commonly have less treatment opportunities and are understudied at the level of genomic targets. We hypothesized that patients with rare cancer benefit from approved anticancer drugs outside their label similar to common cancers.In the Drug Rediscovery Protocol (DRUP), patients with therapy-refractory metastatic cancers harboring an actionable molecular profile are matched to FDA/European Medicines Agency-approved targeted therapy or immunotherapy. Patients are enrolled in parallel cohorts based on the histologic tumor type, molecular profile and study drug. Primary endpoint is clinical benefit (complete response, partial response, stable disease ≥ 16 weeks).Of 1,145 submitted cases, 500 patients, including 164 patients with rare cancers, started one of the 25 available drugs and were evaluable for treatment outcome. The overall clinical benefit rate was 33% in both the rare cancer and nonrare cancer subgroup. Inactivating alterations of CDKN2A and activating BRAF aberrations were overrepresented in patients with rare cancer compared with nonrare cancers, resulting in more matches to CDK4/6 inhibitors (14% vs. 4%; P ≤ 0.001) or BRAF inhibitors (9% vs. 1%; P ≤ 0.001). Patients with rare cancer treated with small-molecule inhibitors targeting BRAF experienced higher rates of clinical benefit (75%) than the nonrare cancer subgroup.Comprehensive molecular testing in patients with rare cancers may identify treatment opportunities and clinical benefit similar to patients with common cancers. Our findings highlight the importance of access to broad molecular diagnostics to ensure equal treatment opportunities for all patients with cancer.

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Molecular Profile Treatment Approach
Gene Name Source Synonyms Protein Domains Gene Description Gene Role
Therapy Name Drugs Efficacy Evidence Clinical Trials
Drug Name Trade Name Synonyms Drug Classes Drug Description
Gene Variant Impact Protein Effect Variant Description Associated with drug Resistance
Molecular Profile Indication/Tumor Type Response Type Therapy Name Approval Status Evidence Type Efficacy Evidence References
BRAF V600E large cell carcinoma predicted - sensitive Cobimetinib + Vemurafenib Case Reports/Case Series Actionable In a clinical study, a patient with poorly differentiated large cell carcinoma harboring BRAF V600E experienced a complete response lasting at least 2 years on combination treatment with Cotellic (cobimetinib) and Zelboraf (vemurafenib) (PMID: 35046062). 35046062