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Ref Type Journal Article
PMID (31325145)
Authors Kaley TJ, Panageas KS, Mellinghoff IK, Nolan C, Gavrilovic IT, DeAngelis LM, Abrey LE, Holland EC, Lassman AB
Title Phase II trial of an AKT inhibitor (perifosine) for recurrent glioblastoma.
Journal Journal of neuro-oncology
Vol 144
Issue 2
Date 2019 Sep
Abstract Text Perifosine (PRF) is an oral alkylphospholipid with antineoplastic effects and reasonable tolerability. It inhibits signaling through the PI3/AKT axis and other cascades of biologic importance in glioblastoma, and has promising pre-clinical activity in vitro and in vivo. Therefore, we conducted a phase II open-label single-arm clinical trial of perifosine for patients with recurrent glioblastoma (GBM).We planned to accrue up to 30 adults with recurrent GBM with a minimum Karnofsky Performance Status of 50 following radiotherapy but without other restrictions on the number or types of prior therapy. Concurrent p450 stimulating hepatic enzyme inducing anticonvulsants were prohibited. Patients were treated with a loading dose of 600 mg PRF (in 4 divided doses on day 1) followed by 100 mg daily until either disease progression or intolerable toxicity. The primary endpoint was the 6-month progression free survival (PFS6) rate, with at least 20% considered promising. Accrual was continuous but if 0 of the first 12 patients with GBM reached PFS6, then further accrual would terminate for futility. Patients with other high grade gliomas were accrued concurrently to an exploratory cohort.Treatment was generally well tolerated; gastrointestinal toxicities were the most common side effects, although none resulted in treatment discontinuation. However, there was limited to no efficacy in GBM (n = 16): the PFS6 rate was 0%, median PFS was 1.58 months [95% CI (1.08, 1.84)], median overall survival was 3.68 months [95% CI (2.50, 7.79)], with no radiographic responses. There was a confirmed partial response in one patient with anaplastic astrocytoma (n = 14).PRF is tolerable but ineffective as monotherapy for GBM. Preclinical data suggests synergistic effects of PRF in combination with other approaches, and further study is ongoing.


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Molecular Profile Treatment Approach
Gene Name Source Synonyms Protein Domains Gene Description Gene Role
Therapy Name Drugs Efficacy Evidence Clinical Trials
Drug Name Trade Name Synonyms Drug Classes Drug Description
Perifosine KRX-0401|NSC-639966 Akt Inhibitor (Pan) 19 Perifosine (KRX-0401) is a synthetic alkylphospholipid that inhibits AKT resulting in apoptotic induction and antitumor activity (PMID: 16418332, PMID: 28864978, PMID: 31325145).
Gene Variant Impact Protein Effect Variant Description Associated with drug Resistance
Molecular Profile Indication/Tumor Type Response Type Therapy Name Approval Status Evidence Type Efficacy Evidence References
Unknown unknown glioblastoma multiforme no benefit Perifosine Phase II Actionable In a Phase II trial, Perifosine (KRX-0401) was well tolerated, but did not demonstrate activity in patients with recurrent glioblastoma multiforme, with a 6-month progression-free rate of 0% (0/16), a median progression-free survival of 1.58 months, and a median overall survival of 3.68 months (PMID: 31325145). 31325145
Unknown unknown astrocytoma not applicable Perifosine Phase II Actionable In a Phase II trial, Perifosine (KRX-0401) was well tolerated, but demonstrated limited activity in patients with anaplastic astrocytoma (n=14), with only 1 patient achieved a partial response (PMID: 31325145). 31325145