Rome 2007 |
by Karen, Dundee
Dear All
The weekend started at 5pm on Friday evening with each attending countries Patient Group (22 countries in total attending) giving a PowerPoint demonstration (or just a talk in the case of Scotland as we are such a new group with no data as yet) about the problems they experience, what they know about Alpha numbers and known statistics.
I noted that Ireland stated that they had 2 Zamira trials which were double blind that they have positive feelings about.
I spoke to Larry Warren and he advised me that the PowerPoint presentations would be published on the Alfa Europe web site (http://www.alfaeurope.org/). This site is not yet fully functional and only has a front page which contains links to member countries web sites.
I have spoken with Andrew Deans in Scotland and I believe he will be organising for the Saltire Flag to link to the Alpha1 Awareness Site (Andrew Willis’ site). I am sure when the presentations are published you will find some of the statistics interesting.
From the Patient Group presentations it became glaringly apparent that all countries are suffering from the same problems as us for example the lack of awareness of Alpha1 and lack of further research funding. It was also
apparent from the weekend that COPD is gaining a lot of press and funding and perhaps we could tap into that, for example the Asthma Foundations may be able to give us money to boost our own funds for the charity.
All of the following is based solely upon my notes. Lin will be scanning and forwarding to the group printed abstracts from the program which details what each person spoke about in more detail and more accurately that we can explain which I am sure you will find informative and
interesting.
Maurizio Luisetti from Italy gave a talk about Where we are, Where we are going to. He said that the S gene was born some 15,000 years ago and the Z mutation between 2-6,000 years ago. He also stated that there were 2 studies currently underway looking for a secondary gene responsible for lung decline.
This session finished at about 9pm when we went for dinner. After dinner it was time for bed as we were to start again at 9am on Saturday morning.
Saturday started obviously with breakfast (and lots of tea for Lin!). John Walsh from Miami began the day with his presentation on Patients Empowerment. I am again hoping that you will be able to review his PowerPoint presentation but even more so that the video of his
presentation will be made available as it really drove home to me that we have to be responsible for our own wellbeing. I will endeavour to obtain a copy of this video for the group.
Francesco Callea from Italy then spoke about Pathogenesis of Liver Injury in Alpha1 – this I am afraid went mainly over my head as it was technical medical. One thing I found surprising was that Liver damage does not occur with either S or Nul.
Claudio Tantucci spoke about The Assessment of Lung Function again from Italy and again over my head. It was at times difficult with the Italian accent and the medical content.
Then David Lomas (whom so many have mentioned on this site) spoke. What a lovely man…he certainly came down to our level and his presentation was both informative and amusing. He spoke about the Pathogenesis of Lung Disease. My understanding of what he said is that they care concentrating and working on getting the Antitrypsin out of the liver so that it is
therefore able to protect the lung. Once the Antitrypsin is out of the liver they will then need to find a way to stabilise it for the lungs. They have data to support that this can work using mice and test tubes but it is as yet a Hypothesis in that nobody else has agreed that their data
is accurate. He retires in 22 years and he is hopeful for something good by then and would anticipate a cure in 40 years if he is right. He dedicated his presentation to Robin Muskett.
Eeva Piitulainen from Sweden spoke about the Natural History of AAT deficiency in the Childhood – the PowerPoint presentation gave interesting statistics on a study which was started back in the 70’s.
Niels Seersholm from Denmark – Heterozygous: A Risk? I noted that he stated that there was only an increased risk of liver disease for MZ – so if you didn’t smoke no increased risk of lung disease.
The answer to a question raised is – Alpha1 Foundation (I believe this to be the US foundation as I heard of 3 groups called this during the weekend!) is funding some Stem Cell research into liver disease.
Robert Stockley from ADAPT said that the repair of lungs was working in rats. In October 2008 we will see the results of the current trial. We should take antibiotics at the first sign of infection to prevent damage to the lungs.
Mark Brantly from the USA told us about aerosolized trials of which there a number underway currently which he is excited about and we should have the results of these in about 3.5 years. With regard to Gene Therapy they are looking at an Adeno Associated Virus – they haven’t had any noticeable results yet in AAT levels but they are about to start trialling higher doses.
My Conclusion
On the whole the weekend made me aware that there are many Alpha1 Patient Groups throughout the world all working towards the same goals but reinventing the wheel. We need to raise awareness of Alpha1 and find,
what was called at the conference, the “C Word” - what is surely at the centre of all of our hearts a CURE!
It was stated at the conference that awareness needed to be raised at a medical level to ensure that other Alpha’s are diagnosed early to ensure that they can make the necessary lifestyle changes which will reduce the damage to their lungs substantially. In the absence of a cure this is
critical to our wellbeing.
We also need to make the Health Authorities, Common Aim Groups and Government more aware so that we can obtain better funding and awareness to help the medical professionals who are committed and able to progress
the essential objective of working towards the C word. It may perhaps be considered somewhat selfish but I know what I would prefer the medical profession to be concentrating on – and it is the C word rather than
awareness! So anything we can do to help with the awareness is surely of benefit to us, our children and Alpha1 friends and cousins.
Having spoken with Andrew Deans and expressed my views on this he is in agreement that the Scottish Patient Group (when we get going) should be closely linked to both the Yahoo Group by way of Patient Support but also
to the Alpha1 Awareness Charity Group. I also hope that we can find a way for these 2 groups (plus Scotland) to work closely together to achieve ‘win win’ targets and realise benefits for all.
As I understand it the Yahoo Group Funds go mainly towards funding flowers/cards/etc for those of us who are in hospital and to subsidise the Group Meetings. Could this not be included as an aim of the charity too perhaps widening to include others in the Adapt Patient Group? Surely if we raise funds from the Yahoo Group these could go into one big pot meaning we reach the target level required to achieve and maintain Charitable Status. On this note Lin and I spoke with a chap from Baxter over dinner on Saturday night and took the bull by the horns and asked for a donation…perhaps cheeky…but “if your not fast your last” as they say here in Scotland. He initially said he had no funds but cornered Lin the following morning saying he may be able to find a couple of hundred to assist…every little counts!
We should share information and resources and try to work together as one team. One focused army with a common aim is surely more powerful and would have more presence and influence than lots of smaller armies all working independently. I hope that a common approach will go some way to achieving our objectives on a National level and with the help of the European Groups being co-ordinated by Alfa Europe on a much wider scale.
Best wishes
Karen
Rome 2007
|
|
|