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Hot topics in Gene and Cell Therapy – exclusive video interviews

Professor Nick Lemoine, Director of the Barts Cancer Institute and Editor of the Nature Specialist Journal, Gene Therapy is co-presenting a series of on-line video-cast interviews with distinguished scientists at the American Society of Gene & Cell Therapy in May 2011.


Clinical trials and advances in systemic delivery of gene therapies for Duchenne muscular dystrophy. Professor Xiao Xiao, University of North Carolina	Advances in gene and cell therapies to prevent blindness due to retinal disease. Professor Robin Ali, Institute of Ophthalmology, UCL

Gene therapy for cancer-associated pain and chemotherapy-induced neuropathy. Dr Darren Wolfe, President & CEO, Diamyd Inc	Paracrine mechanisms of stem cell action in tissue repair and regeneration. Dr Victor Dzau, Duke University

Cell and gene therapies. Professor Malcolm Brenner, Baylor College of Medicine, Houston	Developments in oncolytic virotherapy for cancer. Dr David Kirn CEO of Jennerex Biotherapeutics

The American Society of Gene & Cell Therapy (ASGCT) is dedicated to the understanding, development and clinical application of genetic and cellular therapies to alleviate human disease. It is the largest association of individuals involved in gene and cell therapeutics with approximately 2,500 members worldwide.

The ASGCT’s annual meeting aims to exchange information and promote education among professionals and the public. This year, the BCI presented the following posters at the annual meeting.

Targeting of Prostate Cancer Cells with the Novel Replication-Selective Adenoviral Mutant AdΔΔ in Combination with the Phytochemical Drugs Eqoul and Resveratrol
Authors: Virginie S. Adam, Daniel Oberg, Nicholas Lemoine, Gunnel Halldén.

Replication-Selective Adenovirus Expressing the Prodrug-Converting Enzyme CD/UPRT Efficiently Inhibits Tumour Growth in Prostate Cancer Models
Authors: Maria Ekblad, Daniel Oberg, Nicholas Lemoine, Gunnel Halldén.

A Novel Therapeutic Regime To Eradicate Established Tumors with an Effective Induction of Tumor-Specific Immunity
Authors: James Tysome, Xiaozhu Li, Shengdian Wang, Pengju Wang, Dongling Gao, Rathi Gangeswaran, Dong Chen, Nick R. Lemoine, Yaohe Wang

Modification of the Early Gene Enhancer-Promoter Improves the Oncolytic Potency of Adenovirus 11
Authors: Han Hsi Wong, Guozhong Jiang, Rathi Gangeswaran, Ming Yuan, Hexiao Wang, Vipul Bhakta, Heike Muller, Nick Lemoine, Yaohe Wang

Gene therapies are experimental treatments that aim to insert, alter, or remove a gene in a patient’s cells in order to produce benefit by preventing, arresting or reversing the course of a disease. Although gene therapy is still largely experimental and has not yet become a standard way to treat most patients, the results of some studies have produced very promising results in some inherited blood disorders (e.g., Factor IX deficiency) and eye disease (e.g., Leber’s congenital amaurosis), with patients benefiting substantially from gene therapy. Some of the most dramatic advances have been in cancer, for which there are two licensed commercial agents – both based on adenoviral vectors – presently only available in China. Gene therapy is being used to help design stem cell therapies not only for their traditional application in bone marrow transplantation but in the repair or replacement of tissues such as in the heart, eye, brain and other organs.

Click here to find out about our senior researchers working on gene therapy.

Joining forces with Pharma to battle Kidney Cancer

The Barts Cancer Institute (BCI), in a partnership with Affitech A/S, is aiming to translate its lab-based research into a potential, new treatment for kidney cancer.

Antibody Professor Fran Balkwill’s lab was the first to show that a particular protein receptor (chemokine receptor CCR4), normally found on white blood cells, is overexpressed in some cancer cells. Targeting CCR4 and inhibiting its action reduces the survival of cancer cells and their ability to spread, without adversely affecting normal cell function. This is why it has the potential to be a target for new therapies in various tumours, particularly kidney cancer.

Affitech A/S is a company that specializes in developing antibodies for the treatment of cancer and other diseases, and has already developed an anti-CCR4 antibody. It will provide the antibody to the Centre for Cancer & Inflammation at the BCI, so that the anti-CCR4 antibody can be tested, using the research techniques developed in our labs, to determine its ability to block cancer growth and spread. Promising results in the lab will allow Affitech to develop the antibody into a real treatment for people with kidney cancer.

This is an ideal collaboration; the BCI’s research expertise is used to validate a new product, and at the same time, substantiate its research findings, without having to spend time and money developing its own antibody; Affitech can advance their therapeutic programme for an anti-CCR4 antibody, with its potential as a new drug to treat cancer tested using techniques already available.

The future for treatment of kidney cancer

Kidney cancer accounts for approximately 2% of all human cancers and as a result around 100,000 deaths occur per year worldwide. Current treatments are mainly restricted to surgery as well as kinase inhibitors and anti-angiogenic agents. Although we are at the initial phase of targeting CCR4, using Affitech’s therapeutic antibodies, we are excited that we may be able to find another weapon to treat kidney cancer, with the potential for treating other tumour types with further research.