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Santhera's Catena® in Leber's Hereditary Optic Neuropathy

Santhera Pharmaceuticals Holding AG will present newest clinical data related to the results of a clinical trial of Catena® in Leber's Hereditary Optic Neuropathy (LHON). Catena® is the first drug that has been evaluated in arandomized, placebo-controlled study in LHON. Catena® might protect the retinal and optic nerve cellsand thereby delay, lessen or prevent vision loss. LHON is a rare neuropathy of the eye nerves, estimated with 20,000 patients in Europe and in the United States.

The compounds has been tested in the RHODOS study (Rescue of Hereditary Optic DiseaseOutpatient Study) and Catena® was evaluated against placebo over a six-monthtreatment period.

The principle investigator Professor Thomas Klopstock, from the University of Munich, according to a press release from Santhera commented:

"Catena® holds the promise to protect LHONpatients from deterioration of vision. The RHODOS-data establish the drug as afirst treatment option for LHON. The trial also has key importance for the wholefield of mitochondrial medicine as it shows that randomized, placebo-controlledstudies can be conducted in these rare disorders. The energy defect ofmitochondria may be tackled by drugs."

Other treatment approaches in LHON are genetical, such as RPE65 gene therapy for Leber's congenital amaurosis were results of a phase 1 dose-escalation trial have been published. ^[1]^[2]^[3]^[4]

Jan M. Keppel Hesselink, MD, PhD

Referenties

[1]: Maguire AM, High KA, Auricchio A, Wright JF, Pierce EA, Testa F, Mingozzi F, Bennicelli JL, Ying GS, Rossi S, Fulton A, Marshall KA, Banfi S, Chung DC, Morgan JI, Hauck B, Zelenaia O, Zhu X, Raffini L, Coppieters F, De Baere E, Shindler KS, Volpe NJ, Surace EM, Acerra C, Lyubarsky A, Redmond TM, Stone E, Sun J, McDonnell JW, Leroy BP, Simonelli F, Bennett J. | Age-dependent effects of RPE65 gene therapy for Leber's congenital amaurosis: a phase 1 dose-escalation trial. | Lancet. | 2009 Nov 7;374(9701):1597-605. Epub 2009 Oct 23.

[2]: Sun X, Pawlyk B, Xu X, Liu X, Bulgakov OV, Adamian M, Sandberg MA, Khani SC, Tan MH, Smith AJ, Ali RR, Li T. | Gene therapy with a promoter targeting both rods and cones rescues retinal degeneration caused by AIPL1 mutations. | Gene Ther. | 2010 Jan;17(1):117-31. Epub 2009 Aug 27.

[3]: Smith AJ, Bainbridge JW, Ali RR. | Prospects for retinal gene replacement therapy. | Trends Genet. | 2009 Apr;25(4):156-65. Epub 2009 Mar 18.

[4]: Tan MH, Smith AJ, Pawlyk B, Xu X, Liu X, Bainbridge JB, Basche M, McIntosh J, Tran HV, Nathwani A, Li T, Ali RR. | Gene therapy for retinitis pigmentosa and Leber congenital amaurosis caused by defects in AIPL1: effective rescue of mouse models of partial and complete Aipl1 deficiency using AAV2/2 and AAV2/8 vectors. | Hum Mol Genet. | 2009 Jun 15;18(12):2099-114. Epub 2009 Mar 19.