Targeted Therapies

Formerly BioOncology Watch

www.tgt-therapies.com

APRIL 2004

NON-SMALL CELL LUNG CANCER (NSCLC)

Standard combination chemotherapy plus gefitinib.  Two phase II studies in patients with previously treated advanced NSCLC have shown that single-agent gefitinib 250 mg daily is active (response rates were 11.4% and 18.4%).  Thus, Giuseppe Giaccone and colleagues conducted a multicenter, double-blind phase III trial (INTACT 1) in which 1,093 previously untreated patients with unresectable stage III or IV NSCLC were randomized to cisplatin 80 mg/m2 on Day 1 and gemcitabine 1,250 mg/m2 on Days 1 and 8 every 3 weeks plus either gefitinib 500 mg daily, gefitinib 250 mg daily, or placebo for up to 6 cycles.  Gefitinib/placebo was then continued until disease progression.  Patients in both of these studies were not selected on the basis of the presence of tumor EGFR. There were no differences in overall survival, time to progression, or response rate between the treatment groups.  Roy Herbst et al performed a second multicenter, double-blind phase III study (INTACT 2) in which 1,037 previously untreated patients with unresectable stage III or IV NSCLC were randomized to receive paclitaxel 225 mg/m2 and carboplatin area under the concentration/time curve of 6 mg/min/mL on Day 1 every 3 weeks plus either gefitinib 500 mg daily, gefitinib 250 mg daily, or placebo for up to 6 cycles.  Gefitinib/placebo was then continued until disease progression.  Again there were no differences observed in overall survival, time to progression, or response rate between the treatment groups.  These large double-blind, placebo-controlled trials showed that the addition of the EGFR tyrosine kinase inhibitor gefitinib to standard combination chemotherapy doublet regimens did not result in greater clinical benefit than standard chemotherapy alone.    (Giaccone G, et al. J Clin Oncol 2004;22:777-784 and Herbst RS, et al. J Clin Oncol 2004;22:785-794)

 

RENAL CELL CANCER (RCC)

Randomized phase II study of CCI-779.  CCI-779 (Wyeth Research) is a novel mammalian target of rapamycin (mTOR) kinase inhibitor.  In a phase I study CCI-779 was well tolerated over a wide range of doses and one patient with advanced RCC achieved a partial response (PR).  These preliminary findings led Michael Atkins and associates to conduct a phase II study in which 111 patients with advanced RCC (previously treated or those who were not considered to be an appropriate candidate for first-line IL-2-based therapy) were randomized to receive 25, 75, or 250 mg of CCI-779 weekly.  Neither efficacy nor safety was found to be influenced by CCI-779 dose level.  The overall objective response rate was 7% (7 PRs and 1 complete response), the median time to progression was 5.8 months, and the median survival was 15.0 months.  The most common CCI-779-related toxicities were maculopapular rash (76%), mucositis (70%), asthenia (50%), and nausea (43%).  These findings suggested that CCI-779 has modest clinical activity against advanced RCC and further studies are warranted.  (Atkins MB, et al. J Clin Oncol 2004;22:909-918)

 

MYELOFIBROSIS WITH MYELOID METAPLASIA (MMM)

Phase II study of thalidomide.  MMM is the most severe among Philadelphia-negative myeloproliferative disorders. Potentially curative therapies, i.e., stem-cell transplantation, are held in reserve and standard treatment consists of supportive therapies or cytoreductive approaches aimed at reducing tumor burden.  Thalidomide has been evaluated in MMM patients based on its anti-angiogenic properties (D'Amato RJ, et al. Proc Natl Acad Sci USA 1994;91:4082-4085) and the prominent neo-angiogenesis that occurs in MMM.  Pilot studies have suggested that thalidomide may be an effective therapeutic agent for MMM. These findings led Monia Marchetti et al to administer oral thalidomide, starting at 50 mg daily and increasing to 400 mg daily as tolerated, to 63 patients with MMM.  Half of the patients were maintained on a thalidomide dose >100 mg daily and the drop-out rate was 51% at 6 months.  Splenomegaly was decreased by >50% in 19% of patients.  An increase in platelet count of ³ 50 x 109/L was achieved in 22% of patients and an improvement in anemia was observed in 22% of patients. Thirty-nine per cent of red blood cell transfusion-dependent patients became transfusion independent with thalidomide treatments.  Improvements in overall disease severity score occurred in 31% of patients and were associated with reduced fatigue.  Thus, low doses of thalidomide have palliative activity in some patients with MMM and further investigations with controlled trials are warranted.  (Marchetti M, et al. J Clin Oncol 2004;22:424-431)

 

NON-HODGKIN'S LYMPHOMA (NHL)

Rituximab adjuvant therapy following high-dose therapy.  Steven Horwitz and colleagues investigated the use of rituximab treatments following high-dose therapy (HDT) supported by autologous hematopoietic cell transplantation (HCT) in patients with relapsed or refractory aggressive NHL.  Starting at Day 42 after HCT, 4 weekly intravenous infusions of rituximab 375 mg/m2 were administered to 35 patients with relapsed or refractory B-cell lymphoma (25 patients with DLCL, 3 patients with mantle-cell lymphoma, 3 patients with transformed B-cell lymphoma, and 4 patients with other subtypes of B-cell lymphoma).  In addition, patients 5 through 35 received a second 4-week course of rituximab 6 months after HCT.  In 29 evaluable patients with a median follow-up of 30 months, the 2-year overall and event-free survival rates were 88% and 83%, respectively.  All patients who received 2 courses of rituximab did not have measurable B-cell counts at 12 months.  However, B-cell numbers were recovering by 18 to 24 months after HCT and serious infections were not observed despite delayed B-cell recovery.  Grade 3 to 4 neutropenia occurred in 54% of patients but patients responded rapidly to granulocyte-stimulating factors.  These data showed that rituximab adjuvant therapy following HDT supported by HCT for patients with relapsed or refractory aggressive NHL is feasible.  The encouraging outcomes for these patients support the rationale for the current US Intergroup phase III trial in patients with recurrent/refractory DLCL.  (Horwitz SM, et al. Blood 2004;103:777-783)

 

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