Transcript Italyx

INTERNATIONAL SYMPOSIUM ON
IBD RESEARCH
FUNDED BY PATIENTS' ORGANISATIONS
“ROADMAP”
Brussels 18 October 2012
Hotel Métropole
EVENT ORGANIZED BY EFCCA
INTRODUCTION
Trend annual increase of resources
Part of GDP (Gross Domestic Product) invested on health:
+ 1,8% on 2010
+ 2,2% on 2009
+ 3,2% on 2008
+ 3,4% on 2007
For the first time relationship between health expenditure and GDP
decreased in 2011= 7,1%
Nel 2010 = 7,2%
Source: Corte dei conti, 2011
All this despite the health sector produces the 11, 2% of GDP
Source: Confindustria
INTERNATIONAL SYMPOSIUM ON IBD RESEARCH FUNDED BY PATIENTS ORGANISATIONS – ROADMAP
Clinical Studies in Italy
Studies supported by No Profit
DESCRIPTION OF RESEARCH
AMICI is currently involved in 5
research projects:
2 projects concluded
3 projects in progress
INTERNATIONAL SYMPOSIUM ON IBD RESEARCH FUNDED BY PATIENTS ORGANISATIONS – ROADMAP
GENERAL DATA
(TIMEFRAME, INVESTED FUNDS, SOURCE OF FUNDS)
For all research projects by AMICI:
Duration / timeframe 2 years
Source of funds for research:
Organisation Funds obtained through a percentage of the taxes of citizens (5 per
mille)
Aims of study / expected outcomes:
Identify the mechanisms of action of the disease and increase the awareness.
Based on which criteria is this particular research chosen?
Projects selected by AMICI’s scientific committee (+ International Referees)
INTERNATIONAL SYMPOSIUM ON IBD RESEARCH FUNDED BY PATIENTS ORGANISATIONS – ROADMAP
FUNDS INVESTED
48.000 € PROJECT CONCLUDED
48.000 € PROJECT CONCLUDED
50.000 € PROJECT IN PROGRESS
50.000 € PROJECT IN PROGRESS
50.000 € PROJECT IN PROGRESS
246.000 €
INTERNATIONAL SYMPOSIUM ON IBD RESEARCH FUNDED BY PATIENTS ORGANISATIONS – ROADMAP
Projects Concluded
• Prospective study on Clostridium difficile
prevalence, antibiotic sensitivity, toxin production
profile, genetic polymorphisms in inflammatory
bowel disease in north Italy [48.000 €]
• The role of lymphatic system in inflammatory bowel
disease [48.000 €]
INTERNATIONAL SYMPOSIUM ON IBD RESEARCH FUNDED BY PATIENTS ORGANISATIONS – ROADMAP
Prospective study on Clostridium difficile prevalence, antibiotic
sensitivity, toxin production profile, genetic polymorphisms in
inflammatory bowel disease in north.
• What is already known and confirmed by this study:
– IBD patients are more frequently infected by toxigenic C. difficile than
healthy subjects
– In IBD patients CDI is mostly community- rather then hospital-acquired
– C difficile strains are highly sensible to both metronidazole and
ciprofloxacin
• What is added by this research:
– CDI in Italy is less frequent than in north America and northern Europe
– The risk of CDI in IBD patients is similar in UC and CD
– No risk factors for CDI have been identified in IBD patients including
antibiotics, anti-TNF, immune-suppressors
– IBD patients asymptomatic carriers of both toxigenic and non-toxigenic C
difficile have the same risk of clinical or endoscopic relapse, surgery and
therapy escalation of IBD patients without C difficile
– Toxigenic CDI does not affect the course of underling IBD also in patients
with CDI detected at IBD relapse
– Virulence of strains isolated in IBD patients is higher than in non-IBD
patients
INTERNATIONAL SYMPOSIUM ON IBD RESEARCH FUNDED BY PATIENTS ORGANISATIONS – ROADMAP
The role of lymphatic system in inflammatory
bowel disease [THERAPEUTIC IMPLICATIONS]
• Entirely new way of therapeutic approach
• Stimulating the lymphatic function may
help to drain inflammatory cells away from
the inflamed tissue and resolve tissue
edema
• Decoy receptors for inflammatory
chemokines: inflammation resolution
INTERNATIONAL SYMPOSIUM ON IBD RESEARCH FUNDED BY PATIENTS ORGANISATIONS – ROADMAP
Projects in progress [50.000 € each]
Therapeutic strategies to alter the natural
history of pediatric Crohn’s disease:
Efficacy and safety
SIRT1-based therapy as a novel approach
for inhibiting gut inflammation
Genetic predisposition and mechanisms of
intestinal fibrosis in Crohn’s disease:
Basis for therapeutic and diagnostic
INTERNATIONAL SYMPOSIUM ON IBD RESEARCH FUNDED BY PATIENTS ORGANISATIONS – ROADMAP
Therapeutic strategies to alter the natural history of
pediatric Crohn’s disease: Efficacy and safety
[Expected Results]
1. Higher rate of remission and mucosal healing in the group of patients treated with IFX
as first-line therapy (top-down group) compared to children treated with conventional
therapy, based on corticosteroids and immunosuppressors (step-up group) at the end
of the induction and maintaining phases (8, 48 weeks of the treatment phase) and at
the end of the study (24 months);
2. Lower number of hospitalization and operation in the top-down group compared to
the step-up group at the end of the study (24 months);
3. Better growth pattern, due to reduced flare-ups of the disease, in the top-down group
compared to the step-up group at the end of maintaining phase (48 weeks of the
treatment phase) and at the end of the study (24 months);
4. Good safety profile of IFX used as first-line therapy in pediatric patients with early CD
at the end of the maintaining phase (48 weeks of the treatment phase) and at the end
of the study (24 months). 4) SIRT1-based therapy as a novel approach for inhibiting gut
inflammation –associated colorectal cancer.
5. Genetic predisposition and mechanisms of intestinal fibrosis in Crohn’s disease: Basis
for therapeutic and diagnostic intervention.
INTERNATIONAL SYMPOSIUM ON IBD RESEARCH FUNDED BY PATIENTS ORGANISATIONS – ROADMAP
SIRT1-based therapy as a novel approach for
inhibiting gut inflammation [Expected Results]
Results
SIRT1 regulates negatively T cell activation, and loss of SIRT1 function
results in development of immune-mediated diseases
 SIRT1 is down-regulated in the inflamed gut of IBD patients
 The expression of SIRT1 is reduced in normal lamina propria mononuclear
cells (LPMC) by pro-inflammatory cytokines, such as TNF-a and IL-21
 Infliximab (anti-TNF-a) increases SIRT1 expression in IBD
 SIRT1 activation leads to diminished production of inflammatory cytokines
Restoring SIRT1 activity could be an useful approach to dampen
the ongoing mucosal inflammation in IBD
INTERNATIONAL SYMPOSIUM ON IBD RESEARCH FUNDED BY PATIENTS ORGANISATIONS – ROADMAP
Genetic predisposition and mechanisms of intestinal fibrosis
in Crohn’s disease: Basis for therapeutic and diagnostic
intervention [Expected Results]
Multidisciplinary approach to intestinal fibrosis will surely
more clearly elucidate the cellular and molecular mechanisms
involved; the identification of a genetic signature of MF
expression profile, and possible confirmation at mucosal
levels could probably pave the way for a genetic risk
prediction of fibrostenosing behaviour. More importantly the
validation of correlation between MRI imaging and fibrotic
score will clearly enhance the diagnostic/prognostic
evaluation of fibrosis. Finally, the clarification of early
pathogenic changes of fibrosis, together with the MF
interaction with inflammatory, angiogenic, wound healing,
and hypoxic mediators, might help for identification of
potential anti-fibrogenic compound.
INTERNATIONAL SYMPOSIUM ON IBD RESEARCH FUNDED BY PATIENTS ORGANISATIONS – ROADMAP
SUGGESTIONS FOR THE FUTURE
It is hard to find 1 a donor who give you
1.000.000 € but it is easier to find 1.000.000
donors who give you 1 € but…..
it is also hard to find a cure with a small study
but it is easier to find a cure if we invest
1.000.000 €
INTERNATIONAL SYMPOSIUM ON IBD RESEARCH FUNDED BY PATIENTS ORGANISATIONS – ROADMAP
CONCLUSION
“Healthy citizens are the
greatest asset any country can
have.” Winston Churchill
INTERNATIONAL SYMPOSIUM ON IBD RESEARCH FUNDED BY PATIENTS ORGANISATIONS – ROADMAP