New data show that combination treatment with RAD001 (everolimus) and Sandostatin LAR (octreotide IM) and RAD001 given alone control tumour growth in patients with pancreatic neuroendocrine tumours (NET), a rare and difficult-to-treat form of cancer.

These results, from the RADIANT-1 (RAD001 In Advanced Neuroendocrine Tumours) study, were presented today at the 33rd European Society for Medical Oncology (ESMO) Congress in Stockholm, Sweden.

In the trial, patients with pancreatic NET who became resistant to chemotherapy were given either daily RAD001 combined with monthly Sandostatin LAR or daily RAD001 alone. The results showed that 82 per cent of patients receiving combination therapy and 77 per cent receiving monotherapy had tumours that either decreased in size or remained stable.

"Pancreatic neuroendocrine tumours cause debilitating symptoms related to hormone production and typically claim the lives of patients within five years despite treatment with chemotherapy, which is the current standard of care," said James Yao, associate Professor of Medicine at The University of Texas. "Results from this trial show the promise of RAD001, with or without Sandostatin LAR, to provide tumour shrinkage or stability and to extend time without disease progression in patients who currently have limited treatment options."

The study explores the potential of mTOR inhibition for patients with pancreatic NET by examining RAD001 alone or in combination with standard of care treatment, Sandostatin LAR. RAD001 (proposed brand name Afinitor) is a once-daily oral therapy that continuously inhibits the mTOR protein, a central regulator of cell division and tumour blood vessel growth.

"These findings begin to establish the role of RAD001 as a promising new option for patients with a rare and deadly form of cancer that historically has not responded well to any treatment," said David Epstein, president and CEO of Novartis Oncology. "The combination of RAD001 and Sandostatin LAR may offer a novel treatment approach for disease and symptom control in patients with pancreatic neuroendocrine tumours who are resistant to chemotherapy."

Two phase-III trials investigating the use of RAD001 and Sandostatin LAR are underway in patients with pancreatic NET and carcinoid tumours. The endpoints will be progression-free survival and overall survival.

The term 'neuroendocrine tumour' or 'NET', as defined by the World Health Organization, refers to a diverse mixture of tumours that include pancreatic NET and carcinoid tumours. The development of NET is not completely understood. In some cases, NET can be part of inherited syndromes that affect the endocrine system. Since they are relatively rare among cancers, there is no routine screening. Like many other diseases, lifestyle factors, most notably smoking, may increase risk for NET.
Pancreatic NET are diagnosed in approximately five per million patient cases. Only 55 per cent of people with pancreatic NET will survive for five years. Pancreatic NET are most commonly found in men and women between the ages of 40 and 60 years of age.

RAD001, an oral once daily inhibitor of mTOR, is an investigational drug being studied in multiple tumour types. The safety and efficacy profile of RAD001 has not yet been established in oncology and there is no guarantee that RAD001 will become commercially available for oncology indications.

Sandostatin LAR is a long-acting, injectable depot formulation of octreotide acetate, a somatostatin analogue that exerts similar pharmacologic effects on the human body as the natural hormone somatostatin.