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THE DIRECTOR |
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NFP37, anno 3
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THE DIRECTOR |
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Dear NFP37 grantees and other gene therapy friends,
One of the leading pontifications among the gene-sceptical circles is a sentence such as: «in spite of a long and expensive research, gene therapy has not been demonstrating convincing therapeutic effects». This is like pretending just after the moon landing in 1969, that a Mars landing program should be started. The engineers would have answered, «yes, the basic technology is here but we need a number of advances to pass the hurdles of such a long mission». Thirty years have passed and still a mission to Mars remains a major enterprise, though at closer reach. The same tech-scepticals will tell us that this costed an immense amount of funds and has not produced anything concrete. They tend to ignore that the space research has produced or contributed to generate a large panoply of daily usable spin-offs, including the notebook from which I am typing this address.
Our NFP37 gene therapy program started in 1996 and is entering now phase 2 with a number of new teams with novel ideas (see table I, below). Back in '96, the perspectives were not that great and many technical hurdles seemed either far-fetched or insurmountable. While attending recent gene therapy meetings, I had the net impression that we may be seeing the famous light at the end of the tunnel.
Thus, without playing the prophet, I am absolutely sure that in gene transfer we won't have to wait thirty years to accomplish challenging missions. Perhaps gene transfer will be different than originally thought, perhaps it will be rather coupled to cell therapy (see degenerative disorders), coupled to conventional techniques (see cancer chemotherapy), or used as a transient beneficial treatment (see transplant rejection, inflammation, etc), or mostly as a preventive tool (see vaccination), or adopt revolutionary technologies (such as chimeroplasty or artificial viruses).
The invited speakers of this year's meeting will tell it clear and loud: No matter how we handle it, gene transfer is bound to enter the routine clinics. I add that this may happen within ten years and from the perspective given me by the NFP37 program I am proud to remark how many distinguished scientists and clinicians in our country firmly believe in this challenge and are doing all the possible to be prepared for the D-Day. By coming to the annual meeting so numerous you witness the strength of this vision.
I can only say: «thank you for being there,
good job,
keep going, and good luck:
this latter, not only you need it but you deserve it!»
Sandro Rusconi
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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