Targeted Therapies

Timely Information for Practicing Hematologists and Oncologists

www.tgt-therapies.com

FEBRUARY 2006

 

Glioblastoma

Response to kinase inhibitors.  The epidermal growth factor receptor (EGFR) is a receptor that is frequently overexpressed or mutated in glioblastomas.  However, only 10% to 20% of patients with glioblastoma have a response to the EGFR kinase inhibitors erlotinib and gefitinib.  Previous studies have revealed that EGFR deletion mutant variant III (EGFRvIII), which persistently activates the phosphatidylinositol 3' kinase (PI3K) signaling pathway, is often expressed in glioblastomas.  In addition, the tumor-suppression protein PTEN, which inhibits the PI3K pathway, is commonly lost in glioblastomas. Ingo Mellinghoff and colleagues analyzed the expression of EGFR, EGFRvIII, and PTEN in available pretreatment tissue obtained from 26 patients with recurrent glioblastoma who had received EGFR tyrosine kinase inhibitor therapy (gefitinib or erlotinib) since 2001.  Seven of these patients achieved a response and 19 patients developed rapid progression during therapy.  They found that the coexpression of EGFRvIII and PTEN was strongly associated with clinical response (p <0.001).  Furthermore, these findings were validated in a multicenter experience with 33 glioblastoma patients and in vitro experiments which showed that coexpression of EGFRvIII and PTEN sensitizes glioblastoma cells to erlotinib.  A second tyrosine kinase inhibitor, imatinib, has also been evaluated in glioblastoma patients due to an overexpression of platelet-derived growth factor (PDGF) and PDGF receptor by this tumor.  David Reardon et al report that the combination of imatinib and hydroxyurea achieved at least stable disease in 51% of 33 patients with recurrent glioblastoma that was associated with a 27% progression-free survival rate at 6 months.  These studies represent important steps toward the identification of rational, tumor-specific therapy for glioblastoma.  A perspective editorial by Henry Friedman and Darell Bigner concludes that future studies of agents that target genetic pathways in glioblastoma will have to include analyses of genes and proteins in tumors from patients enrolled in therapeutic trials.  (Mellinghoff IK, et al. N Engl J Med 2005;353:2012-2024; Reardon DA, et al. J Clin Oncol 2005;23:9359-9368; Friedman HS and Bigner DD. N Engl J Med 2005;353:1997-1999)

 

Non-Hodgkin's Lymphoma (NHL)

R-CHOP vs. CHOP in advanced follicular lymphoma (FL).  Wolfgang Hiddeman and others from the German Low-Grade Lymphoma Group conducted a study in which 428 patients with previously untreated, advanced-stage FL were randomized to receive standard CHOP chemotherapy or rituximab plus CHOP (R-CHOP).  CHOP was given in 3-week cycles for 6 to 8 cycles and rituximab 375 mg/m2 was administered on the day before each cycle to patients in the R-CHOP treatment group.  A higher response rate (96% vs. 90%; p = 0.011) and a longer duration of response (p = 0.001) was achieved in the R-CHOP compared to the CHOP treatment group.  In addition, both time to treatment failure and overall survival were superior in R-CHOP-treated patients (p <0.001 and p = 0.016; respectively).  Severe granulocytopenia occurred more frequently in the R-CHOP group (63% vs. 53%; p = 0.01).  However, the proportion of patients with severe infection was low and comparable between the R-CHOP and CHOP alone treatment groups (5% and 7%).  These results demonstrate that the addition of rituximab to CHOP chemotherapy improves clinical outcomes for patients with advanced, previously untreated FL.  (Hiddeman W, et al. Blood 2005;106:3725-3732)

 

Multiple Myeloma (MM)

First-line lenalidomide plus high-dose dexamethasone.  Lenalidomide (Len) is a new immunomodulatory drug (IMiD) that is an analog of thalidomide.  Len has demonstrated more potent preclinical activity than thalidomide and has shown significant efficacy in relapsed or refractory MM when given as monotherapy or in combination with dexamethasone (Dex).  In the current study, S. Vincent Rajkumar and others used Len/Dex to treat 34 patients with previously untreated MM.  Len was given orally 25 mg once daily on days 1 to 21 of a 28-day cycle and Dex was given orally 40 mg daily on days 1-4, 9-12, and 17-20 of each cycle.  Patients also received aspirin (81 mg or 325 mg once daily) as thrombosis prophylaxis.  Thirty-one (91%) patients achieved a response (2 complete responses, 11 very good partial responses, and 18 partial responses).  Among the 3 patients who did not have at least a partial response, 2 had minor responses and one had stable disease.  Grade 3 or 4 neutropenia developed in 12% of patients and grade 3 or 4 thrombocytopenia did not occur in any of the patients.  Overall, 47% of patients experienced grade 3 or 4 nonhematologic toxicity, most commonly fatigue (15%), muscle weakness (6%), anxiety (6%), pneumonitis (6%), and rash (6%).  One patient (3%) developed a pulmonary embolism and recovered with therapy; no other patient developed deep vein thrombosis or pulmonary embolism.  These findings indicate that Len/Dex is a highly active regimen in patients with newly diagnosed MM and is tolerated with manageable side effects.  (Rajkumar SV, et al. Blood;106:4050-4053)

 

Renal cell Carcinoma (RCC)

Activity of SU11248 in metastatic RCC.  SU11248 (sunitinab malate; Pfizer Inc.) is a highly potent inhibitor of certain tyrosine kinases, including vascular endothelial growth factor receptor (VEGFR) and platelet-derived growth factor receptor (PDGFR).  Antiangiogenesis and antiproliferative effects were demonstrated in preclinical experiments.  The recommended dose defined in phase I trials was 50 mg orally once daily for 4 weeks of every 6-week cycle.  Robert Motzer et al. used this regimen of SU11248 in a multicenter phase II study to treat 63 patients with metastatic RCC.  A partial response was achieved in 25 (40%) patients and 17 (27%) patients had stable disease.  The median time to progression was 8.7 months.  The most common adverse event was fatigue and the most common grade 3 or 4 laboratory abnormalities were lymphopenia (32%) and elevated serum lipase (21%).  Four patients were discontinued from the study for a decline in cardiac ejection fraction.  These results showed that the multitargeted receptor tyrosine kinase inhibitor SU11248 is active against metastatic RCC and is tolerated with manageable toxicities.  Further studies are warranted. (Motzer RJ, et al. J Clin Oncol 2006;24:16-24) 

 

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