Address by An Tanaiste and Minister for Enterprise, Trade and Employment, Mary Harney, TD

At the launch of Cystinosis Foundation Ireland - Irish Stock Exchange

Tuesday, November 5^th, 2003

"I am very honoured to have been invited here this evening to launch Cystinosis Foundation Ireland.

I must say it is an unusual venue for such a launch - it's probably a first, to have an event of this type held on the floor of the Irish Stock Exchange. But then I suppose what we are doing here this evening is not unrelated to what happens on this floor during normal business hours. Tonight's event could be seen as the flotation of a new venture, for which funding is needed. We are here to raise awareness and to seek capital.

We are privileged indeed this evening to be in the presence of some very distinguished company - Dr Jerry Schneider, Professor of Paediatrics at the University of California in San Diego and Dr Donald Cairns, Associate Head of the School of Pharmacy at Aberdeen University.

Dr Schneider and Dr Cairns are the two world leaders in the search for a cure for Cystinosis and I know their presence here this evening will lend great insight and expert assistance to this very important event.

If there is any silver lining attached to this disease known as Cystinosis, it is that thankfully relatively few people suffer from it - about 1 case in every 200,000 live births worldwide and just nine known cases in Ireland. That of course is of little comfort to the families and loved ones of the 6 children and three adults in this country who must battle this degenerative illness every day of their lives.

Thankfully the prognosis for sufferers is somewhat better now, than was the case in the past, and while new treatments are being developed to at least keep the disease at bay, this can involve a harrowing regime of medication, which is both demanding and invasive, and which brings with it some difficult side effects.

However it does represent progress, and progress in turn breeds hope. The quest for improved medications and ultimately a cure must continue, and that's where events like tonight come in. I know the Foundation Director, Sue Maguire, is keen not just to raise awareness but also to raise funds as well, so that they can begin to contribute to that campaign.

The early diagnosis of Cystinosis is key when it comes to the effective treatment of the illness and that's why raising awareness is important. If you manage to diagnose Cystinosis within six months to two years of birth, it yields greater results from the point of view of treatment. However the rareness of the disease means that awareness even amongst members of the medical profession, is low, and that is something the Foundation will aim to address.

It is heartening indeed to know that all medications, feeds, syringes etc for sufferers are presently covered by either Medical Cards or the Long Term Illness Scheme which is administered by the Department of Health.

But we should also perhaps look further down the line and think of doing more to tackle the underlying cause of this debilitating disease. You may know that the Government is currently putting an enormous amount of funding into a variety of basic and applied research projects in this country through Science Foundation Ireland and the Medical Research Council.

Knowledge is the great resource of the future and our aim is to cultivate intellectual property here, as an indigenous resource so that one day it will become our greatest national asset.

It is in this context that I see a link with this evening's event. I would like very much to see some of that funding being steered towards research into Cystinosis and who knows, it may not be beyond the bounds of possibility that Irish scientists and researchers could themselves become world leaders in searching for a cure. Perhaps we might be able to persuade Drs Schneider and Cairns to consider Ireland as a location for Cystinosis research.

Can I conclude by wishing Cystinosis Foundation Ireland every success. There is a great sense of commitment and resolve in this room this evening and I think we will be hearing an awful lot more about this issue in the coming months and years. Can I commend also the great courage and bravery of those who live with Cystinosis and my genuine hope for all of you is that the quest for better and even more successful treatments, will be a speedy one."

Notes for Editors

Cystinosis is a very rare metabolic disease which arises from the over accumulation of the amino acid cystine, in certain organs of the body such as the kidney, eye, muscle, pancreas, and brain.

It symptoms include muscle wasting, difficulty in breathing and swallowing, diabetes, blindness, hypothyroidism, gonadism, late puberty and dementia.

If left untreated, kidney function declines over time to the stage where renal replacement therapy (dialysis or kidney transplantation) becomes necessary.

Cystinosis is an inherited condition, brought about by a genetic abnormality.

Cystinosis has an incidence, in developed countries, of about 1 case in every 200,000 live births.

There are only 9 known sufferers in Ireland, 6 children and 3 adults

Dr. Schneider treated his first patient with Cystinosis in 1965. After many years of research Dr Schneider produced a new drug called Cysteamine, which proved to be a major breakthrough in the treatment of Cystinosis and brought hope to sufferers where little had previously existed.

Dr. Cairns has worked on the development of drugs used in the treatment of Cystinosis and continues to research new and more effective treatments.

The specific treatment for cystinosis, Cystagon. is believed to be effective but its side effects can be severe. Many suffer severe gastric irritation which in turn affects an already poor appetite, causes vomiting and in many cases, ulcers.

However, especially for teenagers the worst side effect is the after smell, it has been described variously as being like cabbage, sour milk and even dirty nappies. Just try and imagine being a vulnerable and/or rebellious teenager being told that they stink.

Apart from this, for the drug to work to maximum effect it has to be administered every six hours, day and night, resulting in severe restrictions on lifestyle; play times and all social activities must be scheduled to fit in with "med time".

Most of the children need supplementary nutrition usually given by an overnight feed; therefore "sleepovers" and any other unplanned visits are impossible.

The parents of these children have had to try and assimilate and understand information that medical students take five years to learn!

They have become adept at setting up feeding bags and disconnecting them at 3.30am half asleep; they must do a crash course in bio-chemistry to understand which of the numerous medications cannot be given in conjunction with others (some of our kids are on up to 10 different preparations, some given four times, daily); they must learn how to give injections and all of this while still trying to maintain "normal" family life.

Packing for even a weekend away means at least one suitcase is full of meds, needles, syringes, capsules, liquids, feeding pump, feeding bags cans of nutritional supplements....all for a "break" away!

Monthly blood tests must be carried out and hospital stays can be frequent. Fortunately at the moment, most of the children are doing well.

ENDS

ETE 1153