Treatment of Advanced Melanoma Myths, Facts and Clinical Trials

Treatment of Advanced Melanoma: Myths, Facts and Clinical Trials Jedd D. Wolchok, MD, PhD Melanoma-Sarcoma Oncology Service Ludwig Center for Cancer Immunotherapy Memorial Sloan-Kettering Cancer Center Adjuvant Therapy: What to do after surgery? • Careful observation- CT scans, physical exams, blood tests • Clinical trials- mainly of investigational immunotherapies (vaccines and antibodies) • High dose interferon alfa (Intron A) Interferon• Approved in 1995 based on results of ECOG-1684 (9 % survival benefit) • 20 MU/m2 IV, 5 days/week x 4 weeks, then 10 MU/m2 SC, TIW x 11 months • Hepatic, cardiac, constitutional and psychiatric toxicities necessitating dose reduction in 2/3 of patients • ECOG-1690: a confirmatory trial? ECOG-1690 ECOG-1690: OS EORTC Overall Survival from randomization 18961 ITT Population 100 90 80 70 60 50 May 2007 Observation GMK vaccine HR (99.99% CI) 1.57 (0.68,3.64) 1.66 (0.69,3.99) P-value 0.03 0.02 40 30 20 10 0 Stratified for Breslow thickness, LND, ulceration, sex (years) 0 O 35 56 1 477 478 2 Number of patients at risk : 214 221 3 51 57 4 3 4 5 Treatment N 657 657 Observation GMK vaccine High Dose IFN versus GMK (ECOG 1694)    OS HR 1.38 P = 0.023    18961 HR 1.66 P = 0.02 What to do if recurrence happens? • • • • Surgery Radiation Therapy Chemotherapy Immunologic Therapy Metastatic Melanoma—2009 Approved Therapies (USA) Date • DTIC • High-dose interleukin-2 1970s 1998 Many patients will receive both agents Myth: Chemotherapy doesn’t work in melanoma Single Agent DTIC Activity* • Response rate • Median response duration • Median survival • 6-year survival 19% 4 mos 6–9 mos <2% DTIC has never been compared with observation or best supportive care *Hill et al. Cancer 53:1299; 1984 (n=580) Activity of single agents in melanoma Early phase II trials of Dartmouth regimen No. of patients 20 45 32 47 20 147 319 PR 7 18 10 11 11 41 100 CR 4 5 5 6 0 17 40 Resp. Rate 55% 51% 47% 36% 55% 39% 44% Del Prete, et al. McClay, et al. Fierro, et al. Reintgen, et al. Richards, et al. Berd, et al. Total More Recent Phase III Trials DTIC +/- IFN +/- Tam 1.0 Dacarbazine Dacarbazine + TMX Dacarbazine + IFN + TMX DTIC vs Dartmouth 1.0 Proportion Surviving Proportion Surviving 0.8 0.6 0.4 0.2 0.0 Dacarbazine + IFN 0.8 0.6 0.4 0.2 0.0 Dartmouth DTIC 0 10 20 30 Months 40 50 60 0 12 24 36 48 60 72 84 Months Falkson et al. JCO, 1997 Chapman et al. JCO, 1999 Cytotoxic Chemotherapy: Conclusion There is currently no evidence that combination chemotherapy or the addition of IL-2 or IFN is superior to chemotherapy alone. Combinations of minimally active drugs yield minimal activity. High-Dose IL-2 Therapy* 1.0 Probability of continuing response 0.8 CR (n = 17) PR (n = 26) CR + PR (n = 43) • • RR: 16% (43/270) Durable responses – Median 8.9 mos – CR: not reached 0.6 0.4 0.2 0.0 0 10 20 30 40 50 60 70 80 90 100 110 120 130 Duration of response, months *Atkins et al. JCO, 1999 (n=270) IL-2 Toxicity: Constitutional • Rigors/fevers • Myalgias/arthralgias • Fatigue • Nausea/vomiting/anorexia • Diarrhea • Skin rash/pruritis T-cell receptor: Antigen-MHC CD28: B7 CTLA-4: B7 Vaccine? Regulation of T cell activation is a Complex Process TCR Antigen MHC ~ CD28 B7 TCR CD28 Antigen CTLA-4  CTLA-4 : B7 suppression  Termination of response ~ B7 Antigen-specific T cell Activation  TCR : Antigen MHC  CD28 : B7 Co-stimulation MHC TCR CD28 Antigen CTLA-4 ~ B7 Activated T cell  IL-2 secretion  Proliferation  Effector function  Induction of CTLA-4 MHC CTLA-4 Blockade Activates Antigen Specific T-cell Responses Anti-CTLA-4 mAb TCR CD28 Antigen MHC CTLA-4 TCR ~ B7 CD28 Antigen MHC CTLA-4 ~ B7 Oct 2006 April 2009 Pre-Treatment After 4th dose PLX4032, XL281, Sorafenib AZD6244 Important New Findings in Genes Altered in Melanoma- Therapeutic Implications • B-raf: mutations occur in this gene in 70% of melanomas, mainly those on intermittently sun exposed skin. Several new drugs being tested to inhibit B-raf with responses observed. • C-kit: mutations in in this gene in a small subset of patients with melanoma on internal surfaces, the face or the bottom of the foot. Imatinib (Gleevec) has shown efficacy in people preselected with mutations.

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