Burzynski Research Institute (BRI) has announced that the FDA has given the company permission to conduct an open-label study of Antineoplastons A10 and AS2-1 in patients >3 months of age with a diffuse intrinsic brainstem glioma (DIPG).

Study subjects will be placed in one of five treatment groups based on their age and whether or not they have received prior treatment for DIPG. The primary study endpoint is a decrease in the size of the tumor, either a partial response (>= 50% decrease in the size of the tumor) or a complete response (disappearance of the tumor). The study is in its final organizational stage and will begin accruing patients as soon as this organizational stage is completed.

DIPG is primarily a disease of childhood, with the majority of patients being between five and 10 years of age. However, infants and adults can also be affected. It is the most common brainstem tumor in children, representing 75%-80% of childhood brainstem tumors, and affecting an estimated 300 children in the U.S. each year.

The prognosis for children with DIPG is significantly worse than that of other primary brainstem tumors. The standard of care for patients with newly-diagnosed DIPG is radiation therapy (RT), which appears to control tumor growth for a short period of time, prolonging survival by approximately three months. Within three to eight months after completion of RT, most patients with DIPG will show progression of their disease. No chemotherapeutic agent has ever demonstrated a significant improvement in outcome beyond that achieved by RT alone.