Targeted Therapies

Formerly BioOncology Watch

www.tgt-therapies.com

august 2004

NON-SMALL CELL LUNG CANCER (NSCLC)

Identification of activating mutations that underlie responsiveness to gefitinib. While response rates of only 10% to 19% have been demonstrated in initial clinical studies of gefitinib treatment of patients with chemotherapy-refractory NSCLC, responses to gefitinib have been rapid and profound. These findings led Thomas Lynch and others in Boston to study tumor specimens obtained from NSCLC patients treated with gefitinib in order to search for mutations in the EFGR gene that may explain why some patients are so responsive to gefitinib. Somatic mutations were identified in the tyrosine kinase of the EFGR gene in the tumors of 8 of 9 patients with gefitinib-responsive NSCLC compared to none of 7 patients with NSCLC that did not respond to gefitinib. The mutations were either in-frame deletions or amino acid substitutions clustered around the ATP-binding pocket of the tyrosine kinase domain. All mutations were heterozygous and identical mutations were observed in multiple patients. In vitro studies demonstrated that these EFGR mutants had enhanced tyrosine activity in response to EGF and increased sensitivity to inhibition by gefitinib. This study showed that a subgroup of patients with NSCLC have specific mutations in the EFGR which correlates with increased clinical responsiveness to the tyrosine inhibitor gefitinib. (Lynch TJ, et al. N Engl J Med 2004;350:2129-2139)

 

Bevacizumab plus carboplatin and paclitaxel. Carboplatin and paclitaxel show enhanced tumor growth inhibition when combined with an antiangiogenesis agent. David Johnson and associates randomized 99 patients with advanced or recurrent NSCLC in a phase II trial to one of the following treatments: (1) the anti-vascular endothelial growth factor (VEGF) monoclonal antibody bevacizumab 7.5 mg/kg plus carboplatin (area under the curve = 6) and paclitaxel 200 mg/m2 every 3 weeks (n = 32); (2) bevacizumab 15 mg/kg plus carboplatin and paclitaxel every 3 weeks (n = 35); or (3) carboplatin and paclitaxel alone every 3 weeks (control arm; n = 32). Patients treated with bevacizumab 15 mg/kg plus carboplatin and paclitaxel had a higher response rate (31.5% vs. 18.8%), longer median time to progression (7.4 vs. 4.2 mo), and increased median overall survival time (17.7 vs. 14.9 mo) compared to patients who received carboplatin and paclitaxel alone. Five of 19 control-arm patients who crossed over to single-agent bevacizumab therapy following disease progression achieved stable disease and a 47% 1-year survival rate. Six bevacizumab-treated patients experienced life-threatening hemoptysis or hematemesis (4 events were fatal). Four of these hemorrhages occurred in patients with squamous carcinomas. Thus, bevacizumab in combination with carboplatin and paclitaxel may improve outcomes in patients with advanced or recurrent NSCLC. However, serious bleeding adverse events were observed most frequently in patients with squamous cell lung cancers. A randomized study of carboplatin and paclitaxel with or without bevacizumab in patients with nonsquamous carcinomas is underway. (Johnson DH, et al. J Clin Oncol 2004;22:2184-2191)

 

COLORECTAL CARCINOMA

Bevacizumab added to irinotecan, fluorouracil, and leucovorin (IFL). Herbert Hurwitz et al conducted a multicenter, double-blind trial in which 923 patients with previously untreated metastatic colorectal cancer were randomized to either: (1) IFL plus placebo; (2) IFL plus bevacizumab 5 mg/kg; or (3) fluorouracil and leucovorin plus bevacizumab 5 mg/kg. Randomization to the third treatment arm (fluorouracil, leucovorin, and bevacizumab) was halted after an interim analysis that included the first 313 patients enrolled into the study. The results of this group were not reported. The median durations of progression-free and overall survival were greater for patients treated with IFL plus bevacizumab compared to IFL alone (10.6 vs. 6.2 mo and 20.3 vs. 15.6 mo, respectively). Response rates were higher (44.8% vs. 34.8%) and the median duration of response was longer (10.4 vs. 7.1 mo) in IFL plus bevacizumab-treated patients. Grade 3 hypertension responsive to medical management occurred more commonly in patients receiving bevacizumab (11% vs. 2.3 %). Thus, addition of bevacizumab to IFL chemotherapy improved survival in patients with previously untreated metastatic colorectal cancer. (Hurwitz H, et al. N Engl J Med 2004;350:2335-2342)

 

NON-HODGKIN'S LYMPHOMA (NHL)

Ifosfamide-carboplatin-etoposide (ICE) plus rituximab (RICE). Tarun Kewalramani and colleagues administered 3 cycles of rituximab (375 mg/m2 each cycle) plus ICE (RICE) to 34 patients with diffuse large B-cell lymphoma (DLBCL) prior to autologous stem cell transplantation (ASCT). RICE therapy resulted in a higher complete response (CR) rate compared to a historical control group of 147 consecutive DLBCL patients treated with ICE only (53% vs. 27%). The 2-year progression-free survival rate after ASCT was comparable to that of ICE-treated patients (54% vs. 43%). Febrile neutropenia occurred in 7.5% of delivered cycles of RICE. This study showed that RICE therapy has acceptable toxicity and can result in a high CR rate prior to ASCT in DLBCL patients. Further studies are warranted. (Kewalramani T, et al. Blood 2004;103:3684-3688)

 

Extended rituximab treatments in follicular lymphoma (FL). Michele Ghielmini et al administered standard rituximab (375 mg/m2 weekly x 4 weeks) initially to 202 patients with FL (32% chemotherapy naïve). At week 12, non-progressors (n = 151) were randomized to no further treatment or extended rituximab (375 mg/m2 every 2 mo x 8 mo). At a median follow-up of 35 months, the median event-free survival (EFS) time was longer in the patients given extended rituximab (23 vs. 12 mo). EFS gains were greatest in chemotherapy-naive patients and patients who responded to induction rituximab treatment. The number of the (14;18)-positive cells in peripheral blood and bone marrow at baseline was significantly predictive of a clinical response to rituximab. These data show that extended rituximab treatments improve EFS in patients with FL. (Ghielmini M, et al. Blood 2004;103:4416-4423) 

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