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Prof Amit Nathwani
UCL Cancer Institute, Paul O'Gorman Building
72 Huntley Street
London
WC1E 6BT
Tel: 2076796225
Fax: 2076796222
Appointment
  • Research Department of Haematology
  • Cancer Institute
  • Faculty of Medical Sciences
Research Summary

The main thrust of my research is the development of novel treatment avenues for haematological disorders and cancer using gene transfer technology. I am in addition interested in stem cell biology
My group has developed a distinctive approach for gene therapy of haemophilia B using a self complementary adeno-associated viral vector (AAV), in collaboration with St Jude Children's Research Hospital in Memphis Tennessee. This vector is significantly more potent than conventional AAV vectors. We have demonstrated the preclinical safety and efficacy of this new vector system and are about to commence a Phase I/II study in subjects with severe haemophilia B. Other clinical targets that are currently being evaluated include: Haemophilia A, Congenital FVII and FX deficiency, urea cycle and lysosomal storage disorders.
Haemato-oncology research is focused on the development of two emerging strategies: immunotherapy and anti-angiogenesis. Gene-therapy approaches are central to each of these strategies. Immunotherapy strategies using engineered T cells are being evaluated for the treatment of chronic lymphocytic leukaemia, a disease that remains incurable. Angiogenesis is required for the growth of tumours including haematological malignancies thus making it an important target for anti-cancer strategies. Long-term expression of an angiogenesis inhibitor is likely to be required and the role of gene therapy-mediated delivery of these agents is being evaluated.
Recently we have begun a program of research on the use of induced pluripotent stem (iPS) cells for the treatment of degenerative conditions as well as disease models. Through funding from the UKRMP/MRC we evaluating methods for the generation of iPS cells in a GMP environment. In addition, through EU framework support we are assessing the potential of iPS cells as a source of cells for the treatment of malignancies and or organ failure.  
Gene therapy for haematological malignancies: My haemato-oncology research is focused on the development of two emerging strategies: immunotherapy and anti-angiogenesis. Gene-therapy approaches are central to each of these strategies.

Immunotherapy strategies are directed against chronic lymphocytic leukaemia, a disease that remains incurable. Current emphasis is on the adoptive transfer of bi-specific gene modified T cells which recognise CMV infected cells through their native receptor and CLL via a tumour antigen specific receptor. Our ultimate goal is to adoptively transfer bi-specific cells immediately after front line therapy in patients with poor prognosis CLL.
Angiogenesis is required for the growth of tumours including haematological malignancies thus making it an important target for anti-cancer strategies. Long-term expression of an angiogenesis inhibitor is likely to be required for the successful treatment of malignancies. Therefore, gene therapy-mediated delivery of these agents is an alternative way to provide long-term expression of these therapeutic proteins. Vectors expressing a number of different angiogenesis inhibitors are currently being designed and tested in vitro and in vivo in murine models of leukaemia and lymphoma.

Teaching Summary

A. Medical students
i) Introduction to Blood groups
ii) Basic blood transfusion
iii) Introduction to leukaemias including CLL

B. Foundation year 1 and 2 teaching
Use of blood products, decision pathway

C. SpR Teaching at the NBS and UCLH
(i) Basic blood transfusion
(ii) Use of blood products and special requirements
(iii) Red cell transfusion, reactions and substitutes
(iv) Management of transfusion reaction
(v) Treatment options in CLL

D. MSc course in transfusion
Clinical decision in the use of blood products

E. UCLH Trust Induction Course in Transfusion
Blood transfusion training for HO and SHO

Appointments
2008 Group Leader   Foundation for Liver Research, United Kingdom
2007 Honorary Consultant Haematologist   Royal Free Hospital, United Kingdom
2006 Professor and Honorary Consultant Haematologist   UCL Cancer Institute and University College London Hospitals, United Kingdom
2001 Visiting Physician Scientist   St Jude Children’s Research Hospital, United States
2001 Honorary Consultant Haematologist   NHS Blood and Transplant, United Kingdom
2001 – 2006 Senior Lecturer and Honorary Consultant Haematologist Department of Haematology University College London Medical School and Hospital, United Kingdom
1997 – 2001 Wellcome Trust Advanced Training Fellow   St. Jude Children’s Research Hospital in Memphis, United States
1993 – 1997 Senior Registrar in Haematology   The University College London Rotation, United Kingdom
1990 – 1993 MRC Clinical Scientific Fellow   MRC Clinical Research Centre, Harrow, United Kingdom
1988 – 1990 Registrar in Haematology   Hammersmith Hospital Rotation, United Kingdom
Academic Background
1997 PhD Doctor of Philosophy – Molecular Biology Open University
1988 MRCP Member of the Royal College of Physicians – Clinical Medicine Royal College of Physicians
1984 MBBS Bachelor of Medicine/Bachelor of Surgery – Clinical Medicine University of Aberdeen
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