Burzynski Research Institute (BRI) has started patient enrolment for a Phase 2 study of Antineoplastons A10 and AS2-1 in patients below the age of 3 months with diffuse intrinsic brainstem glioma (DIPG) disease.
It has been reviewed by US Food and Drug Administration (FDA) and approved by Institutional Review Board (IRB), and is an open-label, single-arm study, where 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 study's primary study endpoint is a decrease in tumour size, either a partial response (= 50% decrease in the size of the tumour) or a complete response (disappearance of the tumour).
DIPG is mainly a disease of childhood, with the majority of patients being between five and ten years of age, although infants and adults can also be affected by the disease.
It is the most common brainstem tumour in children, representing 75%-80%, and affecting around 300 children in the US each year.
Prognosis for children with DIPG is worse than that of other primary brainstem tumours.
The standard of care for patients with newly diagnosed DIPG is radiation therapy (RT), which seems to control tumour growth for a short period of time, prolonging survival by nearly three months.
Within three to eight months after completion of RT, most patients with DIPG will demonstrate progression of their disease.
BRI stated that no chemotherapeutic agent has so far demonstrated major improvement in outcome beyond that achieved by RT alone.