News Release

Renal amyloidosis worsens the Familial Mediterranean Fever prognosis

Study highlights need for specific therapy for AA amyloidosis

Peer-Reviewed Publication

Ketchum UK

Wednesday, 11th June 2003, World Congress of Nephrology, Berlin, Germany – Data presented today show that the occurrence of renal amyloidosis (RA) worsens the Familial Mediterranean Fever (FMF) prognosis, according to Dr Hedi Ben Maïz from the Department of Nephrology and Internal Medicine, Charles Nicolle Hospital, Tunisia.

The study examined 37 patients (36 Arabic and 1 Jewish) with FMF and proven RA with a mean age of 25 years, 1 year from the time of kidney biopsy. Nephrotic syndrome was observed in all 37 patients, high blood pressure in 6 patients and renal failure in 20 patients. 1

FMF, one of a number of chronic inflammatory diseases, is a recessive disorder characterised by acute attacks of fever and severe inflammation of tissues. The disease affects certain groups, mainly Sepharadic, Jews, Armenians, Turks and Arabs. RA is the most severe complication in 80% of cases, leading inevitably to chronic renal failure and to death.2,6

Mortality and morbidity were compared amongst patients treated with colchicines. The study reveals that while colchicines therapy seems to give some good results on confirmed renal amyloidosis in a small proportion of patients, amyloid deposits are associated with an overall poor prognosis.

'These data suggest that renal amyloidosis may increase mortality for patients whose health is already compromised by Familial Mediterranean Fever', says Dr Ben Maïz. 'This study highlights vital concerns about the health impact of renal amyloidosis. These data strengthen our need for a specific therapy to treat renal amyloidosis.'

Renal amyloidosis is a chronic and fatal disease marked by the deposit of amyloid fibrils in the kidney. When caused by an inflammatory disease such as FMF the deposited fibrillar material is of the Amyloid A (AA) type and the condition in this instance is known as AA amyloidosis.

AA amyloidosis can be caused by a number of chronic inflammatory conditions including rheumatoid arthritis, Crohn's disease and Familial Mediterranean Fever. It is part of a large family of diseases, called amyloidosis, which are all linked by abnormal Amyloid protein deposits including the amyloid plaques and lesions of Alzheimer's disease. Abnormal amyloid deposits linked to AA amyloidosis mainly accumulate in the liver, spleen, kidney and GI tract, leading to organ dysfunction and death in 42% of patients within 4 years of diagnosis.

There is currently no specific treatment, or treatment protocols for AA amyloidosis. Clinical management is limited to control of the underlying inflammatory disease, or to the management of organ dysfunction. The only therapeutic option in many cases is dialysis or renal transplantation for patients with end-stage renal disease. 7

'Dr Ben Maïz's study highlights the current unmet need in a specific treatment for AA amyloidosis,' says Denis Garceau, Ph.D., Vice President Drug Development of Canadian firm Neurochem, specialists in the development of therapeutics for neurological disorders and many diseases related to amyloid. 'Physicians need to consider the potential presence of AA amyloidosis, especially in at-risk patients with chronic inflammatory conditions, including FMF. A strong and international effort will soon work towards developing both therapeutic and diagnostic guidelines for AA Amyloidosis.'

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For further information, please contact:
Lise Hébert, PhDNeurochemTel: 1-514-337 4646 Mobile: 1-514-924-7394 lhebert@neurochem.com
Stephen MorganKetchumTel: 44-20-7611-3614 Mobile: 44-7771-788067 stephen.morgan@ketchum.com
Katharine ParmigianiKetchumTel: 44-20-7611-3559 Mobile: 44-7977-140259 katharine.parmigiani@ketchum.com

References:
1. Ben Maïz H, et al. Renal amyloidosis in patients with Mediterranean Fever. World Congress of Nephrology presentation (W247), Wednesday 11 June, 10:00am CET

2. Cunnane G, Whitehead A. Amyloid precursors and amyloidosis in rheumatoid arthritis. Baillières Best Pract Res Clic Rheumatol 1999;13:615-28

3. Falk R, et al. The systemic amyloidosis. N Engl J Med 1997;337:898-909

4. Mayo reference services. Communiqué September 2002:7

5. Garceau D, et al. Safety, tolerability and pharmacokinetic profile of Fibrillex ™ (anti-AA amyloid agent) in healthy and renal impaired subjects. Proceedings from the IXth International Symposium on Amyloidosis 2001, Budapest, Hungary

6. Joss N., et al. Presentation, survival and prognostic markers in AA amyloidosis. QJM 2000;93:535-42

7. Ben Maïz H, et al. Renal replacement therapy for end-stage renal disease related to AA amyloidosis. World Congress of Nephrology presentation (M655), Monday 9 June, 10:00am CET


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