Report by Dr John Kolbe
Green Lane Hospital, Auckland
On behalf of the Scientific Organising Committee

The inaugural New Zealand/Australia LAM (Lymphangioleiomyomatosis) Symposium was held on Waiheke Island in New Zealand's Hauraki Gulf on May 3-5, 2002. Prominent US investigators were joined by New Zealand and Australian researchers with a wide variety of interests including airway smooth muscle, matrix proteoglycans, gene therapy and transplantation. The Symposium was organised by Bronwyn Gray, Director of the New Zealand LAM Charitable Trust and was an enormous success with free and frank exchange of ideas between leading researchers who addressed the problem of LAM from widely different angles.

LAM is an uncommon condition occurring in 1-5/million women, although substantial under-diagnosis is likely. LAM may occur in isolation (sporadic) or as part of the spectrum of disease in tuberous sclerosis (TS). Mutations in two genes (TSC1, which encodes hamartin, and particularly TSC2 which encodes tuberin) have been implicated; with germ cell mutations reported in the disease associated with TS and somatic mutations in sporadic LAM. The abnormal cell in LAM has a smooth muscle phenotype and while research focuses on treatment or a cure for LAM, it is recognised that a better understanding of smooth muscle proliferation and migration has implications for a variety of other conditions including asthma and idiopathic pulmonary fibrosis.

Frank McCormack from the University of Cincinnati, described the research initiatives funded by the US LAM Foundation, provided an overview of research into the condition and described data that implicated the TSC2 gene product, tuberin in the regulation of critical molecular pathways that modulate smooth muscle growth.

Deborah Hunter, from the Anderson Cancer Centre in Texas, outlined the 3 major research directions (genetics, smooth muscle biology and hormones) in this condition and described an animal model and in vitro systems used to study the condition.

The symposium was an enormous success and one which greatly exceeded the expectations of the Scientific Organising Committee. The generosity of spirit shown by the delegates in giving up their time, and in the free and frank exchange of information was unique in my experience. Delegates from very different backgrounds and who might never otherwise have interacted on scientific matters engaged in discussions on new concepts and hypothesis about LAM, new research directions and novel ways of interpreting and applying research data. The fact that a cure for LAM remains currently elusive was acknowledged and presentations on control of dyspnoea, grief and loss and the role of the primary care doctor were very well received; as were the talks on the role of lung transplantation and the potential role of gene therapy. There was universal acclaim for the content and worthiness of the meeting from a medical and scientific point of view. Quotes form Australian researchers included: "Don't thank me (for attending) I got an enormous amount out of this meeting". "This is the best meeting I've been to in the last decade".

The success of the meeting reflected not only the choice of delegates but the attention to detail in all respects and the hard work and generosity of an enormous number of people. Merv Merrilees on the Scientific Organising Committee deserves special mention. The large number of sponsors were acknowledged as was the ongoing support of the New Zealand LAM Trust from Merck Sharp & Dohme. Heartfelt thanks were extended to the owners of the houses in which delegates stayed but also to the tremendously generous owners of Longhouse, Waiheke Island. A debt of gratitude is also owed to that small army of helpers who organised, "watered"/"wined"! and fed and transported delegates. Things ran like clockwork in a wonderfully understated way. This meeting could not have taken place without Bronwyn Gray - and its success is a testament to her enthusiasm, effectiveness, diligence and filial love.

For the NZ LAM Trust and its Trustees, this will be an enormously hard act to follow, but represents a phenomenally successful first major initiative of the Trust.

To quote Winston Churchill: "This is not the end, this is not the beginning of the end, but it may be the end of the beginning".

Bronwyn Gray, NZ LAM Trust Director, (right) presents a cheque for $3000 to Masters student Elyshia Hankin (centre) for her invaluable research work into the causes of LAM. Professor Merv Merrilees (left), Head of Department of Anatomy with Radiology at Auckland University School of Medicine is the primary investigator.

New Zealand LAM Trust Director Bronwyn Gray recently attended the annual American LAM Foundation Basic and Clinical Science Conference held in Cincinnati at the end of March. The Conference hosted LAM patients and their families along with the largest gathering to date of scientists, clinicians and physicians from around the world.

Associate Professor Merv Merrilees, Head of the Department of Anatomy with Radiology at Auckland University School of Medicine, joined with 80 researchers from Rotterdam, London, Moscow, Milan, Toronto, Nottingham and all over the United States to present papers and take part in discussion topics which covered diverse areas of LAM science research.

Over the three days, 26 papers were presented and covered major aspects of LAM science and research with sessions on LAM smooth muscle proliferation, hormone receptors, LAM pathology and cell biology as well as the genetics of LAM.

Philadelphia-based New Zealand scientist and leading cell biologist, Dr Jilly Evans chaired the cell biology session at which Associate Professor Merv Merrilees presented his paper.

Running concurrently with the science sessions was the LAM patient and family conference attended by approximately 100 patients from countries including the USA, Canada, Brazil, Thailand, New Zealand and Australia.

The LAM Trust gratefully acknowledges the Auckland Medical Research Foundation in its decision to assist with funding towards two return airfares for the keynote speakers to the inaugural LAM Science Symposium in May 2002.

Funds from the Sir Harcourt Caughey Fund have been granted by the AMRF in recognition of the importance of next year's symposium. The keynote speakers; Dr Frank McCormack and Dr Deborah Hunter will also give clinical lectures in Auckland before they return to the USA.

New Zealand born London-based chef Peter Gordon gave his support to the LAM Trust in 2001 by cooking for 100 guests at the LAM Trust's Fundraising Dinner.

The event, held at Point 5 Nine Restaurant in Point Chevalier, raised $25,000.

This money is currently being used for two LAM science projects at the University School of Medicine.

Auckland University Masters student Elyshia Hankin has this month received a $5000 grant from Merck Sharp& Dohme(NZ) Ltd.

Elyshia is working under Professor Merv Merrilees at the School of Anatomy and her work is with New Zealand's first LAM science project. The MSD grant is in recognition of Elyshia's commitment and focus to this project.

New Zealander Dr Jilly Evans, a Director of MERCK in the USA and recent key note speaker at Auckland's Knowledge Wave Conference has recently been appointed to the Scientific Advisory Board of the American LAM Foundation.

Dr Evans, a world renowned cell biologist is an expert in molecules that have been implicated in smooth muscle proliferation.

Founding LAM Trust member Bronwyn Gray has accepted a position on the Board of NZORD (New Zealand Organisation for Rare Disorders). This national body was recently established to give a voice to the many groups and individuals suffering from rare disease.