Jan 15, 2009 - The Burzynski Research Institute, Inc. (BRI), a biopharmaceutical company developing treatment for cancer based on genomic and epigenomic principles, reached an agreement with the FDA allowing the company to initiate a pivotal Phase III clinical trial of combination antineoplaston therapy plus radiation therapy in patients with newly-diagnosed, diffuse, intrinsic brainstem gliomas.
Antineoplaston therapy (ANP) uses a synthetic version of naturally occurring peptides and amino acid derivatives found in the human body to target and control cancer cells without destroying normal cells. The agreement was made under the FDA’s Special Protocol Assessment (SPA) procedure and means that the design and planned analysis of the Phase III study is acceptable to support a regulatory submission seeking new drug approval.
The primary objective of this randomized multicenter study is to compare overall survival of children with newly-diagnosed diffuse intrinsic brain stem glioma (DBSG) who receive combination antineoplaston therapy (Antineoplastons A10 (Atengenal) and AS2-1 (Astugenal)) plus radiation therapy (RT) versus RT alone.
DBSG are considered to be one of the most difficult types of cancer to treat. It combines highly malignant characteristics with the very difficult location of the brainstem. DBSG are inoperable because they involve most of the brainstem (diffuse and intrinsic). The number of children in the U.S. with brainstem gliomas is approximately 660. Absent treatment, the survival rate from time of diagnosis is six months or less.
At present, there are no standard curative treatments for the disease. RT is the only treatment that may slow its progress, but at two years 93 percent of children with this type of cancer die and none of them survive for five years. Other conventional treatments such as chemotherapy have generally been tried in clinical trials but are shown to be ineffective. There are no pharmacological treatments approved for DBSG at this time.
“We are very pleased by our agreement with the FDA to move forward with a confirmatory study on a type of tumor that has shown itself to be highly treatment resistant and challenged further by severely limited treatment options and clinical trials that could expand and discover new, efficacious therapies,” said Stanislaw R. Burzynski, M.D., Ph.D. “The SPA agreement puts antineoplaston therapy further down a straight path to regulatory approval, enabling the kind of study that should prove its merits as another option to cancer management.”
What is unique about the agreement to the oncology field is that BRI received the approval without financial backing from the government and without a major pharmaceutical partner.
For more information: www.burzynskiclinic.com
October 21, 2025 
