Targeted Therapies

Formerly BioOncology Watch

www.tgt-therapies.com

JANUARY 2005

HIGHLIGHTS OF THE 46TH ANNUAL MEETING OF THE AMERICAN SOCIETY OF HEMATOLOGY (Dec. 4–7, 2004)

 

CHRONIC MYELOID LEUKEMIA (CML)

BMS-354825: phase I study in imatinib-resistant disease.  Charles Sawyer and colleagues reported preliminary results from a phase I study of BMS-354825, a novel dual SRC/ABL kinase inhibitor, in Philadelphia chromosome–positive CML patients in chronic phase who had experienced hematologic progression or intolerance during prior imatinib treatment. Orally administered doses of 15 to 180 mg/day of BMS-354825 were given to 29 patients. One episode of grade 4 thrombocytopenia was reported: otherwise, study therapy was well tolerated without dose-limiting toxicity. Among 26 evaluable patients (22 patients with imatinib resistance and 4 patients with imatinib intolerance), 19 patients had achieved complete hematologic response and 7 patients had partial response.  To date, 1 complete, 6 major, and 1 minor cytogenetic responses have been observed. Dose escalation continues. These early data demonstrate the effectiveness of BMS-354825 in imatinib-resistant chronic-phase CML. (Abstract 1)

 

MANTLE-CELL LYMPHOMA (MCL)

Rituximab plus HCVAD (R-HCVAD) alternating with rituximab plus high-dose methotrexate-cytarabine (R-M/A). Jorge Romaguera and others administered R-HCVAD alternating every 21 days with R-M/A to patients with previously untreated MCL in a phase II study. The CR/CRu rate after 6 cycles of treatment in 97 evaluable patients was 87%. With a median follow-up of 40 months, the 3-year survival rate was 81%. Grade 4 hematologic toxicity was significant, and 8 deaths were reported (sepsis [3], transformation to MDS/acute leukemia [3], pulmonary hemorrhage [1], and unknown [1]).  These findings indicate that this intensive regimen is effective treatment for patients with previously untreated MCL. Further studies are warranted.  (Abstract 128)

 

CCI-779 for relapsed MCL.  Thomas Witzig et al. reported a high response rate in patients with relapsed cyclin D1–positive MCL treated with single-agent CCI-779, a novel inhibitor of the PI3K pathway at the level of mTOR, in a phase II study. Thirty-five patients received 250 mg of intravenous CCI-779 weekly.  The overall response rate was 38% among 34 evaluable patients (1 CR and 12 PR).  The median time to response was 1 month (range, 1–8 months), and the median duration of response was 5.7 months (95% CI: 5.2–13.2 months). Grade 3 or 4 toxicities occurred in 32 patients; thrombocytopenia was the most commonly reported grade 3/4 adverse event. Platelet counts typically recovered within 1 week.  Only 4 patients were able to tolerate sustained weekly doses of 250 mg per week of CCI without dose reduction. The substantial antitumor activity demonstrated by CCI-779 in this trial warrants further study.  Lower doses of CCI-779 are currently being evaluated in an NCCTG trial.  (Abstract 129)

 

Phase II study of bortezomib.  Andrew Belch and others performed a phase II trial in which patients with advanced MCL who received 0–2 prior chemotherapy regimens were treated with the proteasome inhibitor bortezomib 1.3 mg/m2 IV bolus on days 1, 4, 8, and 11 every 3 weeks. An overall response rate of 33% was observed in 24 evaluable patients. Among the first 14 patients enrolled, 5 serious adverse events occurred in patients with pre-existing edema, dyspnea, or effusions. The eligibility criteria were then amended to exclude such patients. Bortezomib-related ≥grade 2 adverse events included sensory neuropathy (12%), neuropathic pain (12%), myalgia (12%), fatigue (60%), diarrhea (20%), and nausea (14%). Thus, while bortezomib exhibits activity against MCL, higher doses appear unfeasible in the MCL patient population.  (Abstract 608)

 

DIFFUSE B-CELL LYMPHOMA

Rituximab plus CHOP (R-CHOP).  Thomas Miller et al. added 4 infusions of rituximab (375 mg/m2 on days –7, 1, 22, and 43) to 3 cycles of standard CHOP followed by involved-field radiotherapy (RT) in aggressive diffuse B-cell lymphoma patients with limited disease (stage I with at least one adverse prognostic factor: age >60 years, elevated serum LDH, or performance status 2). In 62 evaluable patients, the 2-year progression-free and overall survival rates were 94% and 95%, respectively.  These results compared favorably with a historical experience in a similar group of patients evaluated in SWOG study 8736 who were treated with 3 cycles of CHOP alone followed by involved-field RT. In this historical group, the progression-free and overall survival rates measured at 2 years were 85% and 93%, respectively. Short-term toxicity was similar for patients receiving R-CHOP or CHOP alone. These encouraging results merit further study of the addition of rituximab to standard CHOP treatments.  (Abstract 158)

 

FOLLICULAR LYMPHOMA

Natural history changed by new therapies.  Until recently, the natural history of follicular lymphoma had not changed over 30 years. Richard Fisher and associates analyzed data from recent studies investigating new treatment options. Their analysis showed that a treatment approach utilizing CHOP followed by monoclonal antibody therapy (specifically, rituximab or I131-tositumomab), as compared with ProMACE and CHOP alone chemotherapeutic regimens, has resulted in increases in 4-year progression-free (61% vs. 48% and 46%, respectively) and overall survival (91% vs. 79% and 69%) rates. These data indicate that novel sequential therapies have had a significant impact on the natural history of follicular lymphoma.  (Abstract 583) 

 

Fc gamma receptor polymorphisms.  Polymorphisms of the Fc gamma receptor (at position 158 [CD16] and position 131 [CD32]) have been associated with higher affinity binding of human IgG1, greater in vitro ADCC, and improved outcomes in follicular lymphoma patients following single-agent rituximab therapy. In the present investigation, David Maloney and coworkers evaluated DNA samples from patients with advanced follicular lymphoma (n = 87) treated in a phase II study (SWOG 9800) with sequential CHOP followed by rituximab therapy. They did not find a correlation between Fc gamma receptor phenotype and the 2-year progression-free survival rate. This study demonstrated that the antitumor activity of CHOP followed by rituximab therapy is not dependent on the presence of high-affinity Fc gamma receptor polymorphisms.  (Abstract 589)

 

Minimal residual disease (MRD) analysis.  Jesper Jurlander et al., from the Nordic Lymphoma Group, identified 23 patients with follicular lymphoma who had achieved a clinical CR in a randomized phase II study (Study M39035) with rituximab-based therapy and who also had tumor markers (t[14;18] fusion transcript or clonal rearrangement of Ig heavy chain) detectable by standard DNA-based PCR in the diagnostic bone marrow/blood sample. Blood and marrow samples from these patients were tested for MRD at 10–16 weeks, 38–40 weeks, and 52 weeks following treatment. No evidence of MRD was found at any of the 3 time points in 14 patients. The median duration of clinical CR in these 14 patients was 62 months, compared with 21 months in the 9 patients with detectable MRD at one or more time points (P < 0.005). Nine of 14 patients remained in complete molecular remission at a median follow-up of 62 months. These data showed that long-term molecular remissions are possible in follicular lymphoma patients treated with rituximab-based therapy and that MRD status during the first year was predictive of clinical outcome.  (Abstract 1393)

 

B-CELL LYMPHOMA

Preclinical studies of an anti-CD40 antagonistic antibody.  Wen-Kai Weng et al. utilized a novel anti-CD40 antagonistic monoclonal antibody (CHIR-12.12) to disrupt the CD40/CD40L interaction. In vitro studies showed that CHIR-12.12 inhibited proliferation of follicular lymphoma and CLL/SLL cells in a dose-dependent manner; it was also found to induce effective ADCC of follicular lymphoma cells in a study using purified NK cells from a healthy donor. Thus, CHIR-12.12 blocks CD40/CD40L–induced tumor proliferation and mediates ADCC.  Clinical development of this promising monoclonal antibody is warranted.  (Abstract 3279)

 

Radioimmunotherapy (RIT).  F. Morschhauser et al. treated relapsed or refractory diffuse large B-cell lymphoma in elderly patients with a single dose of 90Y-ibritumomab tiuxetan (Zevalin®) in a phase II study. In 103 evaluable patients, the overall response rate was 44%, but the response rate was 52% in patients previously treated with chemotherapy alone and 19% in patients who had previously received rituximab therapy. No unexpected toxicities were encountered. John Leonard et al. retrospectively analyzed 230 patients from 5 studies to determine the relationship between tositumomab/131I-tositumomab (Bexxar® regimen) antitumor activity and the number of prior chemotherapy regimens. At each successive treatment course, the Bexxar response rate and the duration of response were consistently greater than after chemotherapy. Although the response rate decreased with the number of prior therapies, duration of response remained consistently durable across the range of prior therapies. Andrew Belch evaluated lymphorad-131 (LR131) in 10 patients with relapsed or refractory follicular lymphoma.  LR131 is made up of B Lymphocyte Stimulator (BLyS) protein labeled with iodine 131.  BLyS protein is a B-cell maturation factor that binds to immunoglobulin-positive B cells. LR131 targeted sites of disease seen on CT and PET in 10 of 10 patients. Among 8 evaluable patients, 2 CRs, 2 PRs, and 1 SD were achieved. Treatments were tolerated with mild-to-moderate, reversible toxicity.  (Abstracts 130, 132, and 750)

 

T-CELL LYMPHOMA

Novel agents.  Bexarotene (a synthetic retinoic acid X receptor agonist) was administered orally to 37 patients with T-cell lymphoma for a median of 13 months. Twenty-four patients responded (64.8%), and the peripheral CD8+ T-cell count returned to within normal range in 26 patients after a median of 6.5 weeks of treatment. Denileukin difitox (Ontak®), a fusion protein comprising IL-2 and the enzymatically active domain of diphtheria toxin, was given by IV infusion (18 ug/kg once daily for 5 days every 3 weeks) to patients with relapsed or refractory T-cell lymphoma (patients with cutaneous T-cell lymphoma were excluded). In 7 patients with CD25+ disease, there were 2 CRs, 3 PRs, and 1 SD. Of 7 patients with CD25– disease, there were 2 PRs and 4 SDs.  Overall response rate was 50%, and treatment was well tolerated.  Additional patients are being studied. (Abstracts 744 and 2641)

 

BREAST CANCER (REPORTED FROM THE SAN ANTONIO BREAST CANCER SYMPOSIUM)

Combined blockade of erbB receptor network.  C.L. Arteaga et al. conducted a phase II study in which patients with metastatic breast cancer were treated with trastuzumab 2 mg/kg/week and gefitinib 250–500 mg/day. Eligibility criteria included 0–2 previous chemotherapy regimens for metastatic breast cancer, LVEF ≥50%, no prior exposure to trastuzumab, and breast cancers with HER2 overexpression by immunohistochemistry and/or HER2 gene amplification by FISH. Patients were treated until disease progression. The primary objective was to demonstrate an increase in progression-free survival. Progression-free survival increased from 50% to 65% at 6 months for chemotherapy-naive patients and from 50% to 70% at 3 months for patients previously treated with chemotherapy. Interim analysis of 36 patients showed that median time to progression was only 2.9 months and 2.2 months in the chemotherapy-naive group and the previously treated group, respectively. No ≥grade 3 cardiac toxicities were reported.  Time to progression was shorter than previously reported in patients treated with single-agent trastuzumab, suggesting an antagonistic interaction between trastuzumab and gefitinib. The authors concluded that these findings do not support the further use of trastuzumab-gefitinib combination therapy, and the study was discontinued.  (Abstract 25)

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