Steps toward a HIV cure using gene therapy: Which hurdles need to be overcome?

10 November 2014
In a unique joint publication, researchers of the Berlin-Brandenburg Center for Regenerative Therapies (BCRT) and members of regulatory agencies map out a high-level roadmap for a gene therapy to cure HIV. In spite of 30 years of intense research, there is still no effective therapy for HIV infection. Currently, antiretroviral therapy (ART) is the standard of care which has managed to improve the quality of life of HIV infected individuals but is unable to cure the disease. Recent advances in autologous hematopoietic stem cell gene therapy applying 'anti-HIV genes' have shown promising results in providing a functional cure for the disease. The infection can be permanently controlled by an immune system engineered to be resistant to HIV, which also reduces the chance of passing on HIV to other uninfected individuals.

Mohamed Abou El-Enein and his co-authors* discuss how to establish a strong safety and efficacy profile for such a stem cell gene therapy product. They describe which steps have to be taken in order to move this therapy quickly and safely into a clinical application. The roadmap, established by the authors, also discusses the differences between the EU and US regulations for gene therapy as well as preclinical and clinical study requirements. The authors, who are either from academic institutions or working as regulators state: "The inherent difficulties in performing such work have been imposing for academic physicians and scientists, but are not insurmountable if a structured approach such as this 'roadmap' is followed". The paper is considered a unique joint publication by an academic group and regulators (from the European Medicines Agency and National Authorities) and can be seen as a paradigm for a dialogue between both parties in order to advance translational medicine.

The BCRT is an interdisciplinary translational center with the goal of enhancing endogenous regeneration by cells, biomaterials and factors. They can be used to develop and implement innovative therapies and products. The primary focus of the center is on diseases of the immune system, the musculoskeletal system and the cardiovascular system. At the BCRT clinicians and researchers are working closely together on the medicine of the future: Targeted, personalized medicine that depends on the early recognition of patients' individual healing potential.

*M Abou-El-Enein, G Bauer, P Reinke, M Renner, C K Schneider. A roadmap toward clinical translation of genetically-modified stem cells for treatment of HIV. Trends Mol Med. 2014

Dr. Mohamed Abou El-Enein
Berlin-Brandenburg Center for Regenerative Therapies (BCRT)
Charité - Universitätsmedizin Berlin
Phone: +49 30 450 539 594

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