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THE DIRECTOR |
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NFP37 anno 4 , Director's address 2000
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THE DIRECTOR |
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We survived the lowest and enjoyed the highest point
In the last twelwe months, people in the field of gene therapy have experienced extremely serious adrenaline fluctuations. Just around the last year's meeting we all experienced the cold shower of the news of the death of a patient under experimental treatment. These were followed by a very much advertised US federal scrutiny of the whole field and the subsequent investigations have revealed several episodes that prompted the American Society for Gene Therapy to issue a memorandum which strongly ostracises the conditions in which the principal investigators may have major conflicts of interests (Mol Ther 1, 384). Well dear friends, it is undisputable that greed for money and fame added to personal interests in owned company shares can move people to act in unresponible manner, by for instance promoting premature clinical experimentations or by overemphasising results or by pushing clinicians to adopt risky and dangerous clinical practices. However, it is equally undisputable that public funding is totally insufficient to cover the immense costs of clinical investigations (half a billion per registered new clinical procedure or new drug). This confines the clinical investigation to be intimately bound to private interests. For purist, this may seem a big drawback, but if this is correctly implemented it may be even a big bonus. And I say that, because private companies are used to work with an accelerated rhythm, streamlined strategies, less bureaucracy and finally more efficiently than the public systems in general. However, pitfalls like the ones revealed in the last few months in which some PI's have allegedly looked more carefully at their stock options or corporate problems than to the safety of their patients or the transparency of their reports, can pull an entire discipline into discredit and this can have serious consequences for the progress of the entire field. Even if this will finally turn out to be largely untrue (as I firmly believe) and the accused investigators shall be cleaned of the most serious charges, the damage will remain concrete. For this reason, I have entitled this year's edition "yin-yang between industry and academy in gene therapy research" and I have invited external speakers who combine a solid academic reputation with an equally solid strong industrial experience. I hope we shall have time to discuss some of these issues with those people who understand both scenarios.
So much for the high adrenaline raised by those highly mediatised bad news! The most refreshing and long expected good news came officially in April with the publication of the decisive therapeutical success of Alain Fischer (Science 288, 669 ff) . This is more than just a success of one team, it is the proof of principle for all those who dream to use gene therapy for chronic disorders, a goal that seemed still rather far-fetched before Alain's results. What we have most appreciated from this dynamic French investigator are the personal modesty and the scientific 'rigueur '. Although disclosed to insiders in specific meetings back in December 99, nothing was delivered to the mass-media before the publication of the peer-reviewed paper. A lesson for all of us of how a real scientist and scholar should act! I sincerely hope that the small-step/giant-leap of Alain Fischer will give the necessary guts to all those who started doubting, to pursue this marvelous adventure towards success that is somatic gene therapy. It is in this spirit that I wish everybody an enjoyable meeting.
TABLE 1: SOME FACTS ABOUT THE NFP37:
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GENERAL DATA |
PHASE A |
PHASE B |
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(1996-99) |
(1999-2001) |
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Submissions within the deadline |
56 |
29 |
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Granted |
19 |
21 |
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Total requested amount |
32 Mio Sfr |
9 Mio Sfr |
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Granted amount |
7.6 Mio Sfr |
6.4 Mio Sfr |
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GEOGRAPHICAL DISTRIBUTION |
PHASE A |
PHASE B |
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BASEL |
2 |
4 |
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BERN |
3 |
4 |
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FRIBOURG |
2** |
3** |
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GENEVA |
5 |
5 |
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LAUSANNE |
5 |
6 |
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ZUERICH |
5 |
5 |
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DISEASE ORIENTATION |
PHASE A |
PHASE B |
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Cancer |
8 |
10 |
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Hereditary disorders |
2 |
4 |
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Infectious diseases |
1 |
2 |
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Acquired or degenerative disorders |
2 |
9 |
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Vector development (no disease orientation) |
6 |
4 |
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RESEARCH LEVEL |
PHASE A |
PHASE B |
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Fundamental research |
10 |
7 |
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Preclinical research (animal models) |
5 |
11 |
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Clinical research phase I |
2 |
5 |
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Clinical research Phase II |
0 |
1 |
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Clinical research Phase III |
0 |
0 |
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Ethical/social aspects |
1 |
2 |
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