A.V.L.T.

From 1st July 2000 to 31st December 2003, AVLT spent € 281,603 on the “Laboratory for the Development of Pharmacologic and Pharmacogenomic Therapy of Thalassemia” (ThalLab), operating at the Biotechnology Center of Ferrara University, Italy, as can be seen on the page “Progetti di ricerca – Spese sostenute per il ThalLab” on the Internet site
www.talassemiaricerca.unife.it
Furthermore, AVLT is committed to spending a further 500,000 in the period from 1st January 2004 to 31st December 2008 to pay for scholarship grants, registering patents, etc. (on this subject, see the note “Nuovo Accordo” in “EX”, March 2004, page 10).
In the current year, almost 100,000 has already been spent to pay scholarship grants to:
- three researchers in the Gambari group (two working at the “Thalassaemia Gene Therapy Laboratory” directed by Prof. Rivella in New York and one working at Rome University TRE in the Biochemical Laboratory directed by Prof. Pier Luigi Luise);
- one of Prof. Fibach’s researchers/collaborators in Jerusalem;
- a researcher from ChittagongUniversity (Bangladesh) who is currently working at the ThalLab in Ferrara.
By the end of 2008, AVLT will have spent £ 1.513.394.440 on scientific research into thalassaemia.
We have written the sum in Lire because it gives a better idea of the enormous efforts made by a small Association (136 members this year).
The ThalLab is not an isolated research structure, as shown by the extremely important collaborations set up with the HebrewUniversity - Hadassah School of Medicine in Jerusalem, the “Thalassaemia Gene Therapy Laboratory” at the Weill Medical College of Cornell University in New York and, more recently, with ChittagongUniversity.
Moreover, the ThalLab is open to setting up new agreements with other research centres in order to develop a true network that enables thalassaemia researchers to remain in close communication.
Right from the start, AVLT had set this as a primary goal: to avoid spending money indiscriminately on unconnected projects and instead to encourage an overall framework for the research which would hopefully pool the worldwide wealth of knowledge on thalassaemia.
In the two years since this feature began, we have provided a great deal of news on the activities of the above-mentioned Laboratories, we have quoted scientific journals where significant papers on the results obtained so far have been published, we have indicated the numbers and titles of the patents registered and we have also provided “EX” readers with updates on the research activities through an interview and a report by Prof. Stefano Rivella (see “EX”, April 2003, pages 10-13 and “EX”, April-May 2004, pages 18-19) and an interview with Prof. Roberto Gambari (see “EX”, July-August 2004, pages 18-20).
In other words, we have done our duty, which is to account for how the money donated by citizens is spent and to provide news on the results of the research.
AVLT will certainly be able to meet their financial commitments because they are sure that they can also count on the future support of the Veneto citizens, to whom heartfelt thanks are due for what they have contributed so far to the fight against thalassaemia, as well as to the blood donor associations (AVIS, FIDAS, RDS) and the AGESCI who are at AVLT’s side every year in the challenging “Fundraising Campaigns”.
We do feel however that this support should also come personally from the Italian thalassaemics themselves and from their families so that these Laboratories can be further developed and in line with the expectations of the ill.

The research costs are extremely high: as well as paying the researchers (who should be increased in number), materials have to be purchased for the experiments, because the more experiments are done, the more likely it is that the research will take great leaps forward.
The research we support has reached the pre-clinical phase, as evidenced by the experiments being performed on mice in Prof. Rivella’s Laboratory. The pharmaceutical industry, as we will discuss in future features, is showing an interest in the results achieved. And we can cherish high hopes for gene therapy.
We all know that thalassaemics must first and foremost follow an appropriate daily treatment and that they must therefore constantly refer to the clinics that are treating them. But why shouldn’t they hope to radically improve the conditions of their daily life or even be cured?
They must personally cherish this hope, but they need to give it wings for it to one day come true.
Those wings mean making a great deal of money available. Thalassaemia, although very common in numerous countries, nevertheless remains a rare disease worldwide and the funds available for rare diseases are modest, particularly compared to their number. It is calculated that over 5,000 rare diseases exist in the world and around 2,200 inItaly. Where can the financial means for supporting research into so many illnesses be found? Those directly affected must not therefore simply wait for intervention by state health organisations; they themselves must promote initiatives to support scientific research. Unlike many other rare diseases, in many cases lacking even a diagnosis and a treatment, thalassaemia is very well known and thalassaemia sufferers can count on excellent clinics and a therapy which, although amidst problems and sacrifices, means they can be treated. Thalassaemics must however express a strong desire for scientific research to bring a decisive change to their lives.
This is why they too must be prepared to make a small sacrifice.
By giving up the odd pizza or some other treat, they could participate in funding the scientific research by giving


50 Euro a year,


less than fifteen cents a day, perhaps put aside every day in a moneybox or paid as a lump sum on one of those occasions (Christmas, Easter, the summer holidays and so on) when we are prepared to spend a little more.
AVLT has contributed to creating and developing a far-reaching PROJECT and they feel they have worked with reliability and commitment and that they deserve the trust of Italian thalassaemics. That is why they are asking you for your contributions. The Association’s post office account number is 11235454.
AVLT will continue along its chosen path and pursue the goals described above and will guarantee increasingly more frequent and detailed public information on its activities, both by taking advantage of the spaces generously offered by “EX” and by further improving the Internet site, already rich in information.
They will provide news in this magazine of every payment made and all donors will have the right to request any further details and make any suggestions.
Thalassaemics have the chance to look confidently to the future and they must not miss this exceptional opportunity.
Considering there are around 5,000 thalassaemics in Italy, that would mean a sum of 250,000 Euro available every year, which would also permit significant activity in the clinical research field.
In order to be successful, the appeal contained in this “letter” must be supported by all the Associations and by individual thalassaemics.
Indeed, it should also reach those who don’t read “EX”.
Anyone who would like to contribute to its diffusion can call 0425-33806 at the cost of just one unit. If you leave your number, AVLT will call you back.
A Consultative Committee could be set up with the representatives of the Associations who back this new initiative in order to decide how to spend the funds raised.
If this “open letter” meets with the success we hope, the Italian thalassaemia movement will come to the attention of the whole world not only because of the novelty of the event but also and above all as an example of assuming responsibility, for the concreteness of its activities and for the high moral value its actions will take on. That cannot fail to lead to an attitude of respect and esteem from citizens and Institutions and will make it easier for us to work with those who can help us achieve our goals. It is up to us, through all the means of modern technology, to spread the message of our commitment and seriousness everywhere.
If instead this “letter” fails to reach the hearts and intelligence of our thalassaemics and their families and is thrown into the bin like litter, it will mean that cherishing the hope of a considerable improvement in daily life or of curing thalassaemia isn’t worth the sacrifice of fifty euro a year.
We don’t want to believe that! It would mean we’re not interested in impacting on how soon thalassaemics are freed from the condition of being chronically ill and that we are resigned to entrusting this goal to fate.

Elio Zago, President AVLT

(translation by Ms Mary Hawkes)

(“EX”, year XXX, n. 7/8, November/December 2004)