UK & Ireland Fanconi Anaemia Family/Clinical Network

We are pleased to welcome Dr Michael Milsom to the UK and Ireland FA Research Network.

Mick is a British scientist who has just taken up a research group leader position at the German Cancer Research Center (DKFZ) and the Heidelberg Institute for Stem Cell Research and Experimental Medicine (HI-STEM). He has a long standing interest in research into the field of Fanconi anemia and the main focus of his research group is the biology of the hematopoietic stem cell defect that is inherent to cells defective in the Fanconi signaling pathway. Having trained with Dr David Williams at Cincinnati Children’s Medical Center and Harvard Medical School, his interest in Fanconi spans both basic research into the mechanistic basis for the disease and translational research into novel experimental therapies for FA such as cell and gene therapy.

Molecular biologist Dr Spencer Collis and Consultant Neurosurgeon Mr Thomas Carroll have received funding from Yorkshire Cancer Research to try to uncover a new way of treating brain tumours by capitalising on their knowledge of Fanconi Anaemia to increase the effectiveness of  current chemotherapy drugs. More

The Division of Experimental Hematology and Cancer Biology at the Cincinnati Children’s Research Foundation has an opening for a Postdoctoral Fellow interested in DNA repair and Fanconi anemia. More details

International Fanconi Anaemia Gene Therapy Working Group -  “a real platform for the accelerated development of collaborative clinical studies.”

November 16th saw the first meeting of an International Fanconi Anaemia Gene Therapy Working Group, organised and funded by the Fanconi Hope Charitable Trust working in association with the British  Society for Gene Therapy and the Fanconi Anemia Research Fund (FARF). This meeting, hosted by Prof Adrian Thrasher at the Institute of Child Health and ably chaired by Prof Jakub Tolar from the University of Minnesota, brought together 14 leading Gene Therapy and Fanconi Anaemia experts from across the globe to create an action plan for gene therapy trials in Fanconi Anaemia. The need to run trials collaboratively stems from the very rare nature of the condition, since individual countries have insufficient patient bases.
After many hours of productive and intellectually stimulating discussion on topics such as vector design, transduction, trial design, ethics, safety and toxicology and finally application processes and centre selection the attendees agreed that there was a basis for moving to an internationally coordinated set of gene therapy trials such that data from clinical trials set up under different regulatory systems in different countries can be shared.
What happens next is of course key to the success of the project. Formal minutes with details of the action plan will be circulated shortly by Prof Tolar and it was agreed within the meeting that notwithstanding interim actions, the Group should reconvene formally in 12 months time. The Fanconi Anemia Research Fund has suggested that the next meeting of the group may be organised to coincide with its 23rd Annual Scientific Symposium in Barcelona next autumn.

Prof Thrasher, Chairman of the BSGT said afterwards that the meeting was “a real platform for the accelerated development of collaborative clinical studies.”
Fanconi Hope would like to thank all the participants from the UK, Europe and the US for lending their support to this ground-breaking project, to Prof Thrasher for hosting and Prof Tolar for chairing the meeting and to Bev Mayhew the Executive Director of FARF for her strong support and FARF representation at the meeting.

Attendees at the meeting  (in alphabetical order)

Dr Michael Antoniou
Head of Nuclear Biology Group
King’s College London School of Medicine
Department of Medical and Molecular Genetics, Guy’s Hospital
London,
UK

Dr Cynthia Bartholomä
Division of Translational Oncology
National Center for Tumor Diseases , Heidelberg
German Cancer Research Center
GERMANY

Dr Juan Bueren
Head of the Hematopoietic Gene Therapy Division.
CIEMAT, Madrid
SPAIN

Prof Marina Cavazzana-Calvo
Hopital Necker-Enfants Malades, Biotherapy Department, Paris
FRANCE

Mr Thomas Carroll
Chairman – Fanconi Hope Charitable Trust,
Consultant Neurosurgeon
Lead Clinician, Department of Neurosurgery, Royal Hallamshire Hospital, Sheffield,
UK

Mr Bob Dalgleish
Secretary, Fanconi Hope Charitable Trust
UK

Dr Anne Galy
Head of Immunology and Gene Therapy Group,
GENETHON,
Evry 
FRANCE

Prof Helmut Hanenberg
Department of Pediatric
Hematology & Oncology,
Children’s Hospital,
Heinrich Heine University
Duesseldorf,
GERMANY
and Riley Hospital for Children, Indianapolis
USA

Prof Hans-Peter Kiem
Professor of Medicine / Adjunct Professor of Pathology
University of Washington School of Medicine,
Seattle
USA

Mrs Bev Mayhew
Executive Director
Fanconi Anemia Research Fund
USA

Prof Luigi Naldini
Director,
San Raffaele Telethon Institute for
Gene Therapy, Milan
ITALY

Dr Raffaele Renella
Fellow in Pediatric Hematology/Oncology
Children’s Hospital – Dana-Farber Cancer Institute
Harvard Medical School, Boston
USA

Dr Paula Rio
Gene Therapy Researcher
CIEMAT
Madrid
SPAIN

Dr Julian Sevilla
Gene Therapy Researcher
CIEMAT
Madrid
SPAIN

Prof Adrian Thrasher
Consultant in Paediatric Immunology
Wellcome Trust Senior Clinical Fellow
NIHR Senior Investigator
Centre for Immunodeficiency
Molecular Immunology Unit
Institute of Child Health
London
UK

Prof Jakub Tolar
Division of Hematology-Oncology and Blood and Marrow Transplantation
University of Minnesota Medical School
USA

Prof Els Verhoeyen
Ecole Normale Supérieure de Lyon
Unité de Virologie Humaine
FRANCE

We are now on the countdown to raising our first £100,000, with only £6000 to go!

This amazing figure, achieved in less than two years, is due to the magnificent efforts of our FA families and friends across the UK and Ireland.

This has enabled us to publish the first UK Standards of Care, hold Clinician and Family meetings and provide over £30,000 for UK FA research. We will also shortly be funding an international gene therapy workshop and making a further significant donation to UK FA research.

Thank you everyone!

National Institutes of Health grant expands OHSU’s 15-year research effort into the cause, prevention, treatment of the disease.

Oregon Health & Science University is partnering with the University of Oregon and Harvard Medical School to expedite basic science research into new and existing drugs and compounds that may prevent the complications associated with Fanconi anemia, an inherited condition that can lead to bone marrow failure and cancer.

With a new $10.7 million grant from the National Heart Lung and Blood Institute, a branch of the NIH, the research teams will screen and sort thousands of drug candidates in mice at OHSU, zebra fish at UO and human cell lines at Harvard.  more

Many thanks are to due to Dr Helen Walden of Cancer Research UK for organising the first UK FA Research Conference, held on May 24th.

 A big thank you also to Cancer Research UK for funding the event. In supporting this event and in funding FA research, Cancer Research UK has shown its commitment to FA research as a means to furthering research into cancers in general.

The conference was attended by 25 researchers from the FA  molecular/cell biologist community and also a representative from the FA gene therapy community, Prof  Linda Lako, whose research Fanconi Hope is currently funding.

A representative from Fanconi Hope was kindly invited to attend and to speak briefly at the outset to explain the charity’s role in trying to bring together the UK FA research community and in expediting FA research.

It was very encouraging to see so many of the the researchers meeting for the first time and discussing ways of helping each other. It was also gratifying to realise that there were now in excess of 25 researchers from across the UK working in just this one area of FA research – molecular biology.  When you add the researchers working in other aspects of FA research it proves there is a significant body of research now in the UK devoted to Fanconi Anaemia.

One of the conclusions from the event was that there was an appetite for further such events. Fanconi Hope fully supports this and intends to be actively involved in the organisation of future research conferences not only in the molecular biology field but in FA research more generally.

The programme of events can be downloaded here.

Prof Lako discussing her Gene TherapyResearch	Prof Neil McDonald and Dr Helen Walden in discussion during the coffee break	Dr Ambrose Cole discusses the FA Core Complex	Question Time

Thomas Carroll and Bob Dalgleish of Fanconi Hope attended the British Society for Gene Therapy’s Annual Conference in London on March 29th. Thomas Carroll spoke at the invitation of the BSGT president, Professor Adrian Thrasher on the Public Engagement Day on  ”Fanconi…hope: from ‘Saviour Sibling’ to gene therapy”. Thomas attended for the whole duration of the 3 day conference and was able to meet with a large number of researchers in the gene therapy community to promote the concept of using FA as a target for their research. Fanconi Hope’s sponsorship of the event, entailed covering travel expenses for  Dr Juan Bueren, one of the world’s leading researchers of gene therapy in Fanconi Anaemia, who presented on his ground-breaking work. Fanconi Hope also paid for a ‘bag drop’ which allowed us to target the gene therapy research community with an information sheet to encourage them to consider using FA as the basis of their research.

Fanconi Hope has Trustee membership of the BSGT and is now an official charity sponsor.  Our logo can now be seen on their Charity partners web page. We recognise the tremendous significance of gene therapy as a potential cure for FA and are seeking to expedite the move from basic research to clinical trials through helping to link relevant groups and individuals and through lobbying for Eu funding to support trials.

Our Information Sheet for the Gene Therapy research community	Thomas Carroll presenting on “Fanconi…hope: from ‘Saviour Sibling’ to gene therapy”	Thomas (left) in discussion withProf Adrian Thrasher (2nd L), current BSGT president and Prof Len Seymour, past president.(on right)

Fanconi Hope is one of a number of groups sponsoring the British Society of Gene Therapy Annual Conference to be held in London, 29th -31st March. The event is chaired by Professor Adrian Thrasher, the BSGT President, who some of you may recall, gave a presentation at our last Family Meeting.

Thomas Carroll, our Chairman, will be giving a talk entitled “Fanconi…hope: from ‘Saviour Sibling’ to gene therapy” on Monday 31st March and Dr Juan Bueren, who also presented at our Family Meeting will be talking on Wed 31st March on “Gene Therapy and Cell Reprogramming Approaches in Fanconi Anemia”

Fanconi Hope, who have Trustee membership of the BSGT, are sponsoring Dr Bueren’s attendance expenses and paying for a ‘bag drop’ which allows us to disseminate targetted Fanconi Hope literature to all those involved in Gene Therapy. This material will seek to persuade them to consider using Fanconi Anaemia as a basis for Gene Therapy research and trials.

We are pleased to welcome our three latest Research Network Members, Prof Majlinda Lako, Peter McHugh and Dr Michael Carty:-

Prof Lako’s group at Newcastle University focuses their research on understanding of the basic biology of human embryonic stem cells, their self-renewal and differentiation to haematopoietic, retinal and corneal lineages.

Peter McHugh runs the DNA Damage and Repair Research Group at Weatherall Institute of Molecular Medicine, University of Oxford, John Radcliffe Hospital, Oxford Peter’s group has an increasing interest in FA, and has recently published their first paper in this area. Peter regularly participates in the US FARF, is giving a lecture this year, and is a regular grant reviewer for the FARF.

Michael Carty’s group at the DNA Damage Response laboratory at NUI, Galway have an interest in Fanconi Anaemia as part of their research into the responses of cells to DNA damaging agents such as cisplatin and mitomycin C, and to mitosene derivatives synthesized by colleagues in the School of Chemistry at NUI, Galway.