Global LAM Patient Summit 2008

LAM TREATMENT ALLIANCE GLOBAL LAM PATIENT SUMMIT
11-13 January 2008
Oxford, United Kingdom
SUMMIT REPORT

Fifty seven researchers,clinicians, patients and patient family members representing seventeen countries met in Oxford UK in January.

Bronwyn Gray represented both NZ and Australian LAM patients and their families in her capacity as Vice President of LARA (LAM Australasia Research Alliance) and as Director of the New Zealand LAM Trust.

The aim of the Summit, which was a follow on from the meeting hosted by the LAM Treatment Alliance in Stockholm February 2007, was to involve patients in progress towards finding a treatment for LAM. Discussion re collaboration and coordinated efforts to make the fastest possible progress in the quest for a treatment for LAM dominated the meeting.

There was much discussion on establishing ways in which patients can support and facilitate research. It was agreed that the most limiting factor for research is not lack of interest from researchers or lack of money — but access to LAM tissue and LAM patient data.

Scientists are often deterred by the small number of patients in any given country and this may be even more so in Australia and New Zealand — given our relatively small populations.

The meeting was addressed by representatives from the American based National Disease Research Interchange (NDRI); NDRI has a very strong track record and commitment in the field of tissue procurement and distribution, and has been coordinating fresh and fixed LAM tissue in the USA for the past year.

It was decided that NDRI would extend its service and manage the procurement of LAM tissue within Europe; thus creating and widening an efficient global research network.

There was discussion on ways in which countries in the Australasia/Asia Pacific region could set up an efficient, coordinated and managed network for the procurement and distribution to researchers of fresh LAM tissue; NDRI has agreed to investigate options for assisting in our region and working to provide a solution. NDRI’s website can be viewed at www.ndriresource.org.

Further discussion involved clinical trials and off-label drug use involving drugs which included sirolimus ( rapamycin); Dr Ulrich Costabel ( Germany) emphasised the importance of clinical trials and of having sufficient patient numbers to give statistically significant results, however, clinicians at the meeting acknowledged that fast declining patients do not have the luxury of time and may not be able to wait for trials.

Information on the designing of a Global LAM database — LAMSight by MIT ( Massachusetts Institute of Technology) was presented at the meeting. This database would give the opportunity for LAM patients around the world to register their details; this is important so that researchers can learn how many patients exist in each country and access other general information about them. This will help in the design of clinical trials and will also enable researchers and other patients ( with permission) to make contact with news about developments in research and clinical trials. All data will be extremely well protected from unauthorised access as patient privacy is a high priority in the database design.

The meeting was extremely valuable and it is hoped that LARA and the NZ LAM Trust continue to work together with basic scientists and LAM investigators in both our countries as well as furthering the connections made at the APSR meeting in Brisbane in November 2007.

Participants:

• S. Ben Hur – Patient. Accompanied by Husband Guy Fesherman, Israel 
• Cezar Caluschi – Patient Family, Romania
• Havi Carel PhD – Patient. Accompanied by Husband Samir Okasha, LTA Summit Organizing Committee Bristol, United Kingdom 
• Alissa Caron – LAM Treatment Alliance Volunteer. Oxford University – Public Health Department Oxford, England
• Álvaro Casanova Espinosa, MD – Clinician-Researcher. Hospital Universitario, La Princesa Madrid, Spain 
• Ulrich Costabel MD FCCP – Ruhrlandklinik Germany 
• Vincent Cottin MD PhD – Clinician-Researcher. Hopital Louis Pradel, Reference Center for Orphan Lung Diseases Lyon, France 
• Sheila Curristin PhD – Researcher. National Disease Resource Interchange (NDRI) Philadelphia, PA USA 
• Stephanie Dreyer – Patient. Austin, Texas USA 
• Corine Durand – Patient. Poitiers, France 
• Jim Egan MD – Clinician-Researcher. Mater Hospital Dublin, Ireland 
• Patricia Elliott – Patient. Accompanied by Husband Richard Elliott, Sandyfurd Co. Dublin, Ireland 
• Richard Elliott – Patient Family. Husband of Patricia Elliott, Sandyford Co. Dublin, Ireland 
• Reina Endo – Patient. Hayama, Japan 
• Ian Eslick – Researcher. MIT Media Lab, Cambridge, MA USA
• Lucy Falconer – Patient. Aylesbury, United Kingdom
• Amy Farber PhD – Patient. Accompanied by Husband Michael Nurok. LTA Summit Organizing Committee Cambridge, MA, USA 
• Maryam Fathi – Clinician-Researcher. Karolinksa University Hospital Stockholm, Sweden 
• Guy Fesherman –Patient Family. Accompanying Wife S. Ben Hur Israel 
• Michelle Gonsalves – Patient. Tours, France 
• Prof. Alfredo Gorio – Clinician-Researcher. University of Milano; IRCCS Humanitas General Hospital, Rozzano, Milan Milan, Italy
• Bronwyn Gray – Patient Family. Auckland, New Zealand 
• Sergio Harari MD – Clinician-Researcher Ospedale San Giuseppe Milan, Italy 
• Caroline Heckman PhD – Researcher. University of Helsinki, Finland 
• Tuija Helander – Patient. Accompanied by Husband Teijo Hietanen Eura, Finland 
• Simon Johnson MD – Clinician-Researcher. University of Nottingham Nottingham, UK 
• Jan Johnson – Patient. Organization Representative UK LAM database manager UK LAM Action coordinator UK 
• Krista Kenner – Patient Family. Seattle, Washington USA 
• Jacques Lacronique MD – Clinician-Researcher. Cochin Hospital, France 
• Gonzalo Lainez Gutierrez – Patient Family. Husband of N. Navarro, LTA Oxford Summit Organizing Committee La Rioja, Spain 
• Romain Lazor MD – Clinician-Researcher. Reference Center for Orphan Lung Diseases in Lyon, France, and Bern University Hospital, Switzerland Bern, Switzerland 
• Sherry Marts PhD – Researcher-Facilitator. Society for Women’s Health Research Washington, DC 
• Sara Melloni – Patient. Accompanied by Partner Sebastiano D’Avanzo London, United Kingdom 
• Eleonora Miano – Patient. Catania, Italy 
• Joel Moss MD PhD – Clinician-Researcher. National Heart, Lung, and Blood Institute (NHLBI) USA 
• Anke Müller – Patient. LTA Oxford Summit Organizing Committee, Germany 
• N. Navarro – Patient. Accompanied by Husband Gonzalo Lainez Gutierrez LTA Summit Organizing Committee La Rioja, Spain 
• Tommaso Nicolosi MD – Clinician-Researcher. Cannizzaro Emergency Hospital Catania, Italy 
• Caroline Nilsson – LTA Intern Medical Student. Karolinska Institutet Stockholm Sweden 
• Michael Nurok MD PhD – Clinician-Researcher, Patient Family. Husband of Amy Farber Harvard Medical School Brigham and Women’s Hospital Cambridge, MA USA 
• Berit Oberg – Patient Family. Stockholm, Sweden 
• Samir Okasha – Patient Family. Married to Havi Carel Bristol, United Kingdom
• Maria Luz Vila Otero – Patient. Vigo, Spain 
• Viera Reptova – Patient. Vienna, Austria 
• David Rodman MD – Clinician-Researcher. Novartis Institute for Biomedical Research Cambridge, MA USA 
• Meredith Scott – LTA Staff. Cambridge, MA USA
• Flavia Patitucci Sobroza – Patient. Accompanied by Mother Solange Patitucci Sobroza Belo-Horizonte-Minas Gerais, Brazil 
• Solange Patitucci Sobroza – Patient Family. Mother of Flavia Patitucci Sobroza Belo-Horizonte-Minas Gerais, Brazil 
• Susanne – Patient. Germany 
• Anne Tattersfield MD – Clinician-Researcher. Thurgarton, United Kingdom 
• Jeff Thomas – Researcher. National Disease Resource Interchange (NDRI) Philadelphia, PA USA 
• Henrik Watz MD – Clinician-Researcher. Pulmonary Research Institute at Hospital Grosshansdorf Germany
• Vicky Whittemore PhD – Clinician-Researcher. Tuberous Sclerosis Alliance Silver Spring, MD USA
• Kai-Feng Xu MD PhD – Clinician-Researcher. Peking Union Medical College Peking, China