Phenylbutyrate – pro

Sodium phenylbutyrate is an orphan drug, marketed by Ucyclyd Pharma (Hunt Valley, USA) under the trade name Buphenyl and by Swedish Orphan International (Sweden) as Ammonaps. It, along with special diets, has been used to treat urea cycle disorders. It also has received an orphan drug designation by the FDA for treatment of acute promyelocytic leukemia. It appears to have a number of potentially useful effects. Sodium phenylbutyrate was given an orphan drug designation by the FDA for use as an adjunct to surgery, radiation therapy, and chemotherapy for treatment of patients with primary or recurrent malignant glioma. Orphan drug designation has a lower burden of proof than that required for other FDA approved drugs. It has also been advocated for many years by the Burzynski clinic, more recently in combination with other drugs. Dr. Burzynski is conducting clincal trials with these combinations.

Hogarth P, Lovrecic L, Krainc D. Sodium phenylbutyrate in Huntington’s disease: A dose-finding study. Mov Disord. 2007;22(13):1962-1964.

Fujii T, Yokoyama G, Takahashi H, et al. Preclinical studies of molecular-targeting diagnostic and therapeutic strategies against breast cancer. Breast Cancer. 2008;15(1):73-78.

Dyer, Erica S.; Paulsen, Michelle T.; Markwart, Sonja M.; Goh, Meidee; Livant, Donna L.; Ljungman, Mats (2002).”Phenylbutyrate inhibits the invasive properties of prostate and breast cancer cell lines in the sea urchin embryo basement membrane invasion assay.” International Journal of Cancer 101(5): 496-499.

Luis H. Camacho et al, Phase I dose escalation clinical trial of phenylbutyrate sodium administered twice daily to patients with advanced solid tumors Journal Investigational New Drugs Volume 25, Number 2 / April, 2007 , Pages 131-138

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