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X Seminario I.T.M.O. Neoplasie a bassa incidenza Istituto di Oncologia Policlinico di Monza Monza 07 Maggio2012? Cambia l algoritmo terapeutico Michele Maio Medical Oncology and Immunotherapy, Department of Oncology University Hospital of Siena, Istituto Toscano Tumori SIENA, ITALY

Evaluation of Clinical Responses: Differences in the mechanism of action of tumour vaccines vs direct anti-tumoural drugs (chemotherapy, oncogene-inhibitors ) Chemotherapy/ Target therapy Tumor cell destruction Immunotherapy Immune system activation Tumor cell destruction

Chemotherapy/Targeted Agents and Immuno-therapy Differ in Action and Outcome Response Chemotherapy and Targeted Therapies IMMUNOTHERAPY 0 6 12 Time (months)

Clinical considerations: ipilimumab or vemurafenib? Ipilimumab Vemurafenib Biological selector No Yes (BRAF mutation) Response Variable, lower rate, durable Rapid, high rate, limited duration Complete responses Rare Rare Survival benefit Yes Yes Long-term survival benefit Yes? Toxicities Experience Manageability Substantial Generally manageable, can be severe Accumulating Generally manageable, skin neoplasms

Treatment decision making for metastatic melanoma? Molecular subtyping c-kit mutation Wild type BRAF mutation Tumour load low LDH normal Tumour load high LDH high Clinical trial of c-kit inhibitor Ipilimumab Vemurafenib Clinical trial or chemotherapy Ipilimumab

Slow progressive decline in tumor volume Baseline 10 mg/mq Ipi Q3 wks x 4 Induction W12 W24 10 mg/mq Ipi Q12 wks Maintenance W108

IPILIMUMAB PATTERN OF RESPONSE Baseline W12 PD ipilimumab 10 mg/kg q3w x 4 Induction W24 SD ipilimumab W 108 SD 10 mg/kg q12w Maintenance

Histopathology of cutaneous biopsy at week 56 Haematoxylin and eosin staining depicting strong regressive changes both in flat and nodular areas of the tumor biopsy; neoplastic melanocytes were virtually absent throughout the whole lesion.

Histopathology of liver biopsy at week 102 Histological examination of a liver melanoma mts showed massive necrosis of melanocytes. On left, well-preserved fibroblats with rare lymphocytes inside a fibrotic septum, and melanophages are recognizable (original magnification 200x)

Treatment RO5185426 Pt # 201191-90654 June 2010 April 2012 (28 cycles)

NIBIT-M1 trial Phase II Study Combining Ipilimumab and Fotemustine in Patients with Metastatic Melanoma: the NIBIT-M1 Trial Siena - AOUS PI dr. Michele Maio Milano - HSR PI dr. Lorenzo Pilla Milano - I.E.O. PI Dr. Alessandro Testori Milano - INTFondazione I.R.C.C.S. PI dr. Mario Santinami Padova - IOV I.R.C.C.S. PI dr.ssa Vanna Chiarion Sileni Genova - INT PI dr.ssa Paola Queirolo Meldola - IRSTi PI dr. Ruggero Ridolfi Napoli -Fondazione G. Pascale PI Dr. Paolo Antonio Ascierto C R T Clinical Research Technology

SIENA JULY 8, 2010 - FPFV

NIBIT-M1-Patient 1005 Baseline W28

NIBIT-M1-Patient 1005 Baseline W28

NIBIT-M2 A randomized, Phase III study of Fotemustine versus the Combination of Fotemustine and Ipili mumab in Patients with Metastatic Melanoma with brain metastasis

Clinical Protocol MO25743 An open-label, single-arm, multicenter study to evaluate the efficacy of Vemurafenib in metastatic melanoma patients with brain metastases

Aug 20th, 2011 Fat-sat T2w Gd-T1w Sep 26th, 2011 Courtesy of Alfonso Cerase, MD, Unit NINT Neuroimaging and Neurointervention, Dpt of Neurological and Sensorineural Sciences Azienda Ospedaliera Universitaria Senese, Policlinico Santa Maria alle Scotte, Siena, Italy

What will be the role of clinical trials?

MEDICAL ONCOLOGY AND IMMUNOTHERAPY DEPT. OF MEDICAL ONCOLOGY UNIVERSITY HOSPITAL OF SIENA Maresa Altomonte Giovanni Amato Luana Calabrò Sara Cantamessa Ornella Cutaia Riccardo Danielli Anna Maria Di Giacomo Ester Fonsatti Angela Iacovelli Andrea Lazzeri Marilena Piccinelli Eliana Pittiglio Massimo Resti Sergio Speranza Sandra Coral Francesca Colizzi Alessia Covre Elisabetta Fratta Huges Nicolay Giulia Parisi Luca Sigalotti Clelia Miracco Maurizio Biagioli