The Experts below are selected from a list of 33 Experts worldwide ranked by ideXlab platform
John Nemunaitis - One of the best experts on this subject based on the ideXlab platform.
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A review of Contusugene Ladenovec (Advexin) p53 therapy.
Current opinion in molecular therapeutics, 2009Co-Authors: Neil Senzer, John NemunaitisAbstract:Contusugene Ladenovec (Advexin; INGN-201; Introgen Therapeutics Inc) is a replication-impaired, non-integrating, serotype 5 adenoviral vector that carries the p53 gene under the control of the CMV promoter. Deletion or mutation of the p53 gene has been observed in approximately half of malignancies in patients with cancer and p53 pathway dysfunction was observed in the majority of others, thereby providing the rationale for p53 restoration in the treatment of cancer. Advexin has demonstrated a consistent safety profile and clinical efficacy as a monotherapy, as well as in combined modality regimens with chemotherapy and radiation. Additive or synergistic effects have been observed in a variety of tumor types, including NSCLC, squamous cell carcinoma of the head and neck, hepatocellular carcinoma, glioma, and breast, prostate and colorectal cancers. The identification of biomarkers may help direct research in tumor-specific therapeutics.
Boca Raton - One of the best experts on this subject based on the ideXlab platform.
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Ark floats gene therapy's boat, for now
2008Co-Authors: Boca RatonAbstract:In August, gene therapy’s turbulent ride through the clinical rapids took a new twist as Ark Therapeutics released positive top-line results from a phase 3 trial of its adenoviral gene therapy Cerepro (sitimagene ceradenovec) for malignant brain tumors. Although the news boosted the London-based firm’s shares, the course to market authorization and registration remains strewn with uncertainty—as Introgen, of Austin, Texas, found, to its cost, when the US Food and Drug Administration (FDA) recently refused its Biologics License Application (BLA) for Advexin (Contusugene Ladenovec), an orphan-designated adenoviral gene therapy for treating head and neck cancer and Li-Fraumeni syndrome. Ark’s ‘study 904’, which was approved by the UK Gene Therapy Advisory Committee in 2004, randomized 236 people with brain cancer to receive Cerepro plus standard care or standard care alone, which consists either of surgery and radiotherapy or of surgery and radiotherapy plus the alkylating drug Temodar/Temodal (temozolomide) from Schering-Plough in Kenilworth, New Jersey. Subjects given Cerepro and temozolomide showed a 42-day improvement over standard care in median survival, reaching significance (P < 0.032). Side effects hemiparesis, aphasia and pyrexia could be blamed on ganciclovir, which is part of the Cerepro protocol and is “pretty toxic,” says analyst Stephen Dunn of
Neil Senzer - One of the best experts on this subject based on the ideXlab platform.
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A review of Contusugene Ladenovec (Advexin) p53 therapy.
Current opinion in molecular therapeutics, 2009Co-Authors: Neil Senzer, John NemunaitisAbstract:Contusugene Ladenovec (Advexin; INGN-201; Introgen Therapeutics Inc) is a replication-impaired, non-integrating, serotype 5 adenoviral vector that carries the p53 gene under the control of the CMV promoter. Deletion or mutation of the p53 gene has been observed in approximately half of malignancies in patients with cancer and p53 pathway dysfunction was observed in the majority of others, thereby providing the rationale for p53 restoration in the treatment of cancer. Advexin has demonstrated a consistent safety profile and clinical efficacy as a monotherapy, as well as in combined modality regimens with chemotherapy and radiation. Additive or synergistic effects have been observed in a variety of tumor types, including NSCLC, squamous cell carcinoma of the head and neck, hepatocellular carcinoma, glioma, and breast, prostate and colorectal cancers. The identification of biomarkers may help direct research in tumor-specific therapeutics.
Masayuki Yamato - One of the best experts on this subject based on the ideXlab platform.
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Re-examination of regulatory opinions in Europe: possible contribution for the approval of the first gene therapy product Glybera
Molecular Therapy - Methods & Clinical Development, 2015Co-Authors: Natsumi Watanabe, Kazuo Yano, Kenichiro Tsuyuki, Teruo Okano, Masayuki YamatoAbstract:The first commercially approved human gene therapy in the Western world is Glybera (alipogene tiparvovec), which is an adenoassociated viral vector encoding the lipoprotein lipase gene. Glybera was recommended for marketing authorization by the European Medicines Agency in 2012. The European Medicines Agency had only ever reviewed three marketing authorization applications for gene therapy medicinal products. Unlike in the case of Glybera, the applications of the first two products, Cerepro and Contusugene Ladenovec Gendux/Advexin, both of which were for cancer diseases, were withdrawn. In this report, we studied the European public assessment reports of the three gene therapy products. During the assessment process, Glybera was re-examined and reviewed for a fourth time. We therefore researched the re-examination procedure of the European Union regulatory process. Approximately 25% of the new medicinal products initially given negative opinions from the Committee for Medicinal Products for Human Use were ultimately approved after re-examination from 2009 to 2013. The indications of most medicines were changed during the re-examination procedure, and the products were later approved with a mode of approval. These results suggested that the re-examination system in the European Union contributed to the approval of both several new drugs and the first gene therapy product.
Natsumi Watanabe - One of the best experts on this subject based on the ideXlab platform.
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Re-examination of regulatory opinions in Europe: possible contribution for the approval of the first gene therapy product Glybera
Molecular Therapy - Methods & Clinical Development, 2015Co-Authors: Natsumi Watanabe, Kazuo Yano, Kenichiro Tsuyuki, Teruo Okano, Masayuki YamatoAbstract:The first commercially approved human gene therapy in the Western world is Glybera (alipogene tiparvovec), which is an adenoassociated viral vector encoding the lipoprotein lipase gene. Glybera was recommended for marketing authorization by the European Medicines Agency in 2012. The European Medicines Agency had only ever reviewed three marketing authorization applications for gene therapy medicinal products. Unlike in the case of Glybera, the applications of the first two products, Cerepro and Contusugene Ladenovec Gendux/Advexin, both of which were for cancer diseases, were withdrawn. In this report, we studied the European public assessment reports of the three gene therapy products. During the assessment process, Glybera was re-examined and reviewed for a fourth time. We therefore researched the re-examination procedure of the European Union regulatory process. Approximately 25% of the new medicinal products initially given negative opinions from the Committee for Medicinal Products for Human Use were ultimately approved after re-examination from 2009 to 2013. The indications of most medicines were changed during the re-examination procedure, and the products were later approved with a mode of approval. These results suggested that the re-examination system in the European Union contributed to the approval of both several new drugs and the first gene therapy product.