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Systematic Review

Systematic Review of Gossypol/AT-101 in Cancer Clinical Trials

Department of Nutritional Biochemistry, Institute of Nutritional Sciences, University of Hohenheim, 70599 Stuttgart, Germany
Institute of Nutritional Medicine and Prevention, University of Hohenheim, 70599 Stuttgart, Germany
Department of Internal Medicine VIII, University Hospital Tuebingen, 72076 Tuebingen, Germany
German Cancer Consortium (DKTK), DKFZ Partner Site, 72076 Tuebingen, Germany
Department of Vegetative and Clinical Physiology, Institute of Physiology, University of Tuebingen, 72074 Tuebingen, Germany
Authors to whom correspondence should be addressed.
These authors contributed equally to this work.
Academic Editor: Célia Cabral
Pharmaceuticals 2022, 15(2), 144;
Received: 15 December 2021 / Revised: 17 January 2022 / Accepted: 21 January 2022 / Published: 26 January 2022
(This article belongs to the Section Natural Products)
The potential of gossypol and of its R-(−)-enantiomer (R-(−)-gossypol acetic acid, AT-101), has been evaluated for treatment of cancer as an independent agent and in combination with standard chemo-radiation-therapies, respectively. This review assesses the evidence for safety and clinical effectiveness of oral gossypol/AT-101 in treating various types of cancer. The databases PubMed, MEDLINE, Cochrane, and were examined. Phase I and II trials as well as single arm and randomized trials were included in this review. Results were screened to determine if they met inclusion criteria and then summarized using a narrative approach. A total of 17 trials involving 759 patients met the inclusion criteria. Overall, orally applied gossypol/AT-101 at low doses (30 mg daily or lower) was determined as well tolerable either as monotherapy or in combination with chemo-radiation. Adverse events should be strictly monitored and were successfully managed by dose-reduction or treating symptoms. There are four randomized trials, two performed in patients with advanced non-small cell lung cancer, one in subjects with head and neck cancer, and one in patients with metastatic castration-resistant prostate cancer. Thereby, standard chemotherapy (either docetaxel (two trials) or docetaxel plus cisplatin or docetaxel plus prednisone) was tested with and without AT-101. Within these trials, a potential benefit was observed in high-risk patients or in some patients with prolongation in progression-free survival or in overall survival. Strikingly, the most recent clinical trial combined low dose AT-101 with docetaxel, fluorouracil, and radiation, achieving complete responses in 11 of 13 patients with gastroesophageal carcinoma (median duration of 12 months) and a median progression-free survival of 52 months. The promising results shown in subsets of patients supports the need of further specification of AT-101 sensitive cancers as well as for the establishment of effective AT-101-based therapy. In addition, the lowest recommended dose of gossypol and its precise toxicity profile need to be confirmed in further studies. Randomized placebo-controlled trials should be performed to validate these data in large cohorts. View Full-Text
Keywords: oral gossypol; AT-101; clinical trial; cancer; oncologic patients oral gossypol; AT-101; clinical trial; cancer; oncologic patients
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MDPI and ACS Style

Renner, O.; Mayer, M.; Leischner, C.; Burkard, M.; Berger, A.; Lauer, U.M.; Venturelli, S.; Bischoff, S.C. Systematic Review of Gossypol/AT-101 in Cancer Clinical Trials. Pharmaceuticals 2022, 15, 144.

AMA Style

Renner O, Mayer M, Leischner C, Burkard M, Berger A, Lauer UM, Venturelli S, Bischoff SC. Systematic Review of Gossypol/AT-101 in Cancer Clinical Trials. Pharmaceuticals. 2022; 15(2):144.

Chicago/Turabian Style

Renner, Olga, Mascha Mayer, Christian Leischner, Markus Burkard, Alexander Berger, Ulrich M. Lauer, Sascha Venturelli, and Stephan C. Bischoff. 2022. "Systematic Review of Gossypol/AT-101 in Cancer Clinical Trials" Pharmaceuticals 15, no. 2: 144.

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