Report on neuroblastoma study

The final results from an early phase (I/II) study on combining Soluble Beta-Glucan (SBG) with anti-cancer antibodies in treatment of neuroblastoma in children have been published today. The study, performed at Memorial Sloan-Kettering Cancer Center, showed that the combined treatment with SBG and 3F8 antibodies was well tolerated and the combined treatment gave promising activity against resistant neuroblastoma. Improvement was demonstrated in 14 of 30 patients showing metastasis to the skeleton, whereof 6 of 15 patients showed complete remission of neuroblastoma in bone marrow.

“Neuroblastoma is a very serious form of cancer in infants and young children, with high mortality rates. Given the progressed stage of disease (high-risk stage 4) in the children in the study and the limitations of alternative treatment regimes, we consider these results promising,” says CEO Svein Lien in Biotec Pharmacon ASA.

The patients included in the study had high-risk metastatic neuroblastoma and a history of recurrent or refractory disease. Patients that are more than one year old, as in this study (more than 18 months of age), having disseminated disease (described as "stage 4" in the International Neuroblastoma Staging System Criteria) have very poor prognosis. In the present study only patients with a history of heavy pre-treatment and recurrent or refractory stage 4 neuroblastoma were included.

“We are glad to see confirmation of the positive effects of SBG within the cancer immunotherapy area, and have in co-operation with Memorial Sloan-Kettering Cancer Center secured a very broad and worldwide patent platform protecting this combined treatment,” says Lien.

When the current study was initiated, Biotec Pharmacon agreed to support a follow up phase II study at Memorial Sloan Kettering Cancer Center (MSKCC). However, the company will not immediately move onto the next clinical phase.

”Based on the experience from our earlier clinical studies with SBG, we need to secure a correct formulation of the product before entering into new clinical studies. This work is currently ongoing.” says Lien.

Study details:

The objective of the study was to define the clinical toxicity of the combined treatment and to assess the biological effects of the treatment regimen. The study was a dose escalating study, where groups of patients were treated with an increasing amount of SBG given per orally combined with a fixed amount of monoclonal anti-neuroblastoma antibodies (3F8) given intravenously. SBG was given in increasing dosing steps from 10 mg/kg, increasing to 20 mg/kg and subsequently up to 200 mg/kg per day, each increasing interval of 20 mg. The treatment was scheduled to be given in 4 cycles of 27 days, and increased up to a total of 8 cycles when allowable, with treatment free periods between each cycle. A total of 45 patients were entered into the study, whereof 44 of the patients treated with the combination of the anti-cancer antibody 3F8 and SBG were evaluable for toxicity. A total of 37 patients could be evaluated for treatment response according to the International Neuroblastoma Staging System Criteria.

Of the 30 patients showing metastasis to the skeleton, 14 patients demonstrated improvement in skeletal MIBG during the treatment period, and 6 of 15 patients showed complete remission of neuroblastoma in bone marrow. Overall, 2 patients had complete response to the tumor and was diagnosed symptom free after the treatment period, 3 had partial response, and 20 patient showed stable disease, whereas 12 developed progressive disease already at start of the first treatment cycle and did not receive the intended subsequent cycles of treatment.