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Palifosfamide is the active moiety of ifosfamide (IFA) [1].

IFA is alkylating agents which are active against a variety of pediatric sarcomas such as rhabdomyosarcoma (RMS), Ewing's sarcoma (ES), osteosarcoma (OS) and other undifferentiated soft tissue sarcomas [1].

In human OS cell lines SaOS-2, OS229 and OS230, Palifosfamide lysine has broad activity with IC50 ranging from 2.25 to 6.75 μM. While OS222 had the IC50 of 31.5 μM [1].

In CB17 female SCID mice, palifosfamide lysine (100 mg/kg) administered intravenously for three consecutive days, the mean weight loss was less than 15% and complete recovery to baseline within 4 weeks of treatment. While, doses higher than 100 mg/kg for three consecutive days lead to either greater than 20% loss of body weight or death. In NCr-nu/nu mice bearing established orthotopic mammary MX-1 tumor xenografts, palifosfamide suppressed MX-1 tumor growth by greater than 80% with 17% complete antitumor responses [2].

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