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March 2003 - from LDNZ Secretary, Jenny Noble

Hello everyone – John, Jenny and Dianne hope you all enjoyed a long and restful summer holiday.

Paul and I along with Sarah and Hayden headed to Wanaka where we spent ten glorious days in the sun doing absolutely nothing. Since coming home I have been busy doing work for our 1st National Lysosomal Family Conference. Haven’t we come a long way in three years?

When I think back to the very first contact I had with John, and we talked about our horror stories of diagnosis, no information, no support, and isolation. We both came to the same conclusion that we needed to do something about all the problems we had experienced in the past. Who would believe that now we are organising our first ever New Zealand Lysosomal Conference on Friday 12th - Sunday 14th Sept 2003.

Planning is now well underway, the venue is booked, funding has been applied for, the program is being fine-tuned, and registration forms are being put together. We hope to have these out to you all by the end of May.

I would like to encourage you all to attend this very important Conference. It is an opportunity to break that isolation, to meet other families who have the same problems that you do, but most importantly of all is to hear first hand the advancements in new therapy's and treatments for Lysosomal Diseases.

LDNZ Website

Our website has been generating a lot of interest over the last 3 months, with a total of 10,000 hits over this period. This is very exciting - people are looking at the site reading our information and our stories. Since Hayden and Sarah’s story has been on the web we have received many e-mails wanting more information about Mucolipidosis. The internet is truly a wonderful tool for making contact with other families around the world.

I would like to take this opportunity to invite other families to send John your story to be included on the family links and story page. If you would rather not do that but would like to share with other New Zealand families please send something to me via mail or e-mail to be included in our next news letter.

NZORD Website

John has been very busy over the last few weeks putting this site together. He expects it to be on the net towards the end of this March. Look out for it on www.nzord.org.nz

Secretary/Treasurer

At our last annul meeting LDNZ accepted Judith Forman’s resignation as secretary and Jenny Noble was voted into the position of Secretary / Treasurer. John remains as Chairman.

Other events

• 2nd Australian Batten Disease Conference 18th-19th October 2003. This conference is to be held at Sea World Nara Resort, Gold Coast, Queensland. For more information contact Vanessa Anderson gvjcando@ozemail.com.au
• The 8th International Symposium on MPS and Related Diseases will be held from 10th-14th June 2004 at Rheingoldhalle, Mainz, Germany.

Chairperson's report to LDNZ Trustees - 8 January 2003

Here is my report on the financial year to 30 June 2002, with some additional commentary on events through to the end of 2002.

Fundraising - the negative

It is particularly frustrating to note that so few of the hotel casino funds are willing to give to small groups such as ours. Being a national organisation also puts us at a disadvantage when many have a local focus. There is currently much public discussion about the ethics and controls over these funds, which are the largest charitable funds in NZ. Being directly asked "what's in it for us" by one hotel owner does not inspire confidence in the integrity of the system, and could readily lead to a conclusion that having a local group that is willing to come back to the hotel for functions, would certainly enhance our prospects of success.

Our experience, despite some limited support that was gratefully received, is that for groups such as ours it is just not worth the time and effort involved in applying to these funds. We are marginalised by higher profile activities such as sports, or larger health interest groups, by structural problems of national versus local conflicts, and by apparent self-interest in the allocation process. Let us hope that the news rules for managing the allocation of these grants, do achieve the intended results of equity and transparency.

Fundraising - the positive

We had much better success this time, compared to previous years. There are usually very few sources of funds available to us so the grants of $3500 from each of Genzyme Australia and from TKT were vital to our continued operation, as they provided the core funding of our essential activities. Additional donations came from unsolicited donations from members of the public, particularly associated with media publicity we were able to generate, and these totalled nearly $1500. A substantial grant of $5000 for general operating costs was received from the Frimley Foundation. In addition there was a $4400 travel grant to the Chairperson from Novazyme /Genzyme which passed through our books.

After the close of the financial year there were further grants of $2000 from the Rehabilitation Welfare Trust, and a startling but very welcome $10,645 received from various agents and business divisions of AMP. This latter grant was closely associated with the publicity surrounding the innovative treatment of Hayden and Sarah Noble with Pamidronate, and the remarkable results achieved, plus Jenny Noble's role in presenting the results of this experimental treatment to the International conference on Lysosomal diseases in Paris.

But the crowning glory of our fundraising activities was the $16,200 received from the Paul Newman Foundation. This was granted specifically to assist with our first ever family conference in New Zealand. The end result of this is a very sound current financial position with all commitments met and $22,000 on term deposit earmarked for our family conference later this year, and $6700 available for general expenses and services. This compares very well with just $6500 received in the previous financial year. However considerable additional fundraising will be required to ensure our conference is a success.

Conference attendances

Because of the rarity of Lysosomal diseases and the isolation of NZ patients and families, attending conferences is important to share knowledge and experiences, and learn many of the basic facts about the disorder that would be almost taken for granted in more common conditions. This is particularly relevant to those with younger children and recent diagnoses.

Building on the previous year's success in getting individuals or families to relevant conferences, in 2002 we supported five attendances at the Australian MPS conference, two to the International Lysosomal Meeting in Paris, and one to the International Pompe meeting in England. The Chairperson also attended a Lysosomal diseases summit and a board meeting of the International Mannosidosis and related disease organisation, in the US.

Finding families

Our contact list for affected NZ families had a 10% increase to 51 during 2002, though some contacts have been lost through address changes. The survey of Paediatric clinics did not produce the numbers expected, but it is clear that the Metabolic Service is having an impact in connecting families to us.

I estimate there are probably another 20 affected families that have a diagnosis but have not made contact with us, as well as probably 50 or more that have not yet had an accurate diagnosis of their Lysosomal disease.

Support and information for families

Our key function in this area is the provision of information on the website. The nature of the internet is such that it is difficult to know who it is accessed by and for what purpose, but we get more than 2000 hits per month from more than 500 distinct users. Enquiries frequently come from overseas as well as within NZ, and it is clear that some families read the site thoroughly before making direct contact with us.

Direct support has included advice to families about dealing with education and social support issues, as well as sharing experiences in managing particular aspects of the particular condition, or suggesting additional sources of practical or clinical advice. At times it can even include a good long chat to enable the parent or patient to unload some of the frustrations they have to cope with. There were about 15 instances of direct support to NZ families during 2002, and about an equal number of responses to overseas families seeking information.

Influencing health policy

There is often a pattern to the issues that arise and we have taken up some of the problem areas with the Health Ministry. One particular problem has been poor transition planning for those with complex and chronic health conditions, when they are due to move on from paediatric to adult health services. LDNZ worked closely with the Paediatric Society to identify some of the problems and produce a suggested set of solutions. This will now be discussed with the Ministry of Health.

Genetic Modification

We have been active in this controversial area as we are well aware of the importance of GM and similar modern biotechnology, in producing treatment options for Lysosomal diseases. Following on from our submissions to the Royal Commission in 2000, last year saw us keep up our public profile on these issues by supporting AgResearch's application to develop transgenic cows with the aim of producing therapeutic proteins in their milk. This technique has already been applied experimentally for one Lysosomal disease, and it is a promising area of research towards treatment of these complex diseases.

Publicity

The Holmes Show feature of Hayden and Sarah Noble's treatment, and other media coverage, has been the best publicity we could ever have hoped for. It has raised awareness, despite the spelling and pronunciation of Lysosomal being severely mangled in the programme. The mention of the parents who did everything was the perfect counterpoint to the other publicity that week of the parents who did nothing.

Significant donations have been associated with this publicity and this has helped achieve our currently sound financial position. Jenny and Paul Noble deserve special recognition for the work they have done for Hayden and Sarah, as well as their contribution to LDNZ. There remains a possibility of further publicity on the same topic with later follow-up reports on Hayden and Sarah's progress.

Planning for our 2003 Family conference

This work is well under way and the timetable is being adhered to. All the indicators are for a successful event. Special thanks to Jenny Noble for taking on the key role of conference organiser.

Quick summary

A good year of activity and fundraising, with the promise of more positive activity to come in 2003.

John Forman
Chairperson