A Randomized, Double-Blind, Placebo-Controlled Trial of Curcumin in Leber's Hereditary Optic Neuropathy (LHON)

2014-07-24 14:20:25 | BioPortfolio


Background Leber's Hereditary Optic Neuropathy (LHON) is a maternally inherited ocular disorder associated with a mutation in mtDNA . The common manifestation is visual loss which caused by the respiratory chain enzymes complex dysfunction resulting in increased oxidative stress enzymes production.

Purpose To determine whether curcumin which is an antioxidant agent is beneficial to the patients with 11778 LHON mutation.

Material and Method Seventy patients with 11778 LHON mutation were randomly treated with oral curcumin (500 mg/day) and placebo for 1 year. The visual acuity, computerized visual field, electrophysiologic parameters and oxidative stress enzymes in plasma were compared before and after treatment at 3, 6, and 12 months interval.


Leber's hereditary optic neuropathy (LHON) is a maternally inherited disease that is characterized by a simultaneous or more common sequential bilateral loss of central vision, which typically occurs during the teenager years or early adulthood. The disease may be progressive and the patients eventually become blind. In contrast to blindness in patients caused by congenital diseases, the patients who develop blindness caused by LHON will have more trouble in their life because they previously had better vision. Although LHON is associated with mitochondrial DNA mutation at a variable nucleotide position but no certain mechanism of optic nerve injury has been found. It has been postulated that a defect in a complex of respiratory chain enzyme which is caused by mitochondrial DNA dysfunction, results in an increase of free radical substances that interfere with optic nerve function in LHON. Some antioxidant substances have been used to decrease the progression of visual impairment. However, there have been few therapeutic trials for LHON.

Curcumin, a component of tumeric, which comes from the root Curcumin longa has antioxidant, anti-inflammatory and anti-carcinogenic activities. There is some evidence which suggests that curcumin contributes to the in vitro removal of free radical gradients in thalassemic serum and to a clinical improvement of thalassemic patients. We propose a randomized controlled trial study of curcumin, which is an antioxidant substance, for LHON. This study will provide not only an insight into the therapeutic efficacy of curcumin, a kind of Thai herb, for LHON but also the development of future therapeutic strategies to prevent blindness.

Study Design

Allocation: Randomized, Control: Placebo Control, Endpoint Classification: Efficacy Study, Intervention Model: Parallel Assignment, Masking: Double Blind (Subject, Investigator), Primary Purpose: Treatment


Optic Atrophy, Hereditary, Leber




Professor Wanicha Chuenkongkaew




Mahidol University

Results (where available)

View Results


Published on BioPortfolio: 2014-07-24T14:20:25-0400

Clinical Trials [658 Associated Clinical Trials listed on BioPortfolio]

EAP_GS010_single Patient

Expanded Access Use for a single patient of Bilateral Intravitreal Injection of GS010 in a Single Subject Affected with G11778A ND4 Leber Hereditary Optic Neuropathy

Safety and Efficacy Study of Gene Therapy for The Treatment of Leber's Hereditary Optic Neuropathy

This study is meant to evaluate the safety and efficacy of rAAV2-ND4 treatment for Leber hereditary optic neuropathy with the G11778A mutation in mitochondrial DNA.

RESCUE and REVERSE Long-term Follow-up

The goal of this clinical trial is to assess the long-term safety and efficacy of GS010, a gene therapy, and assess the quality of life in subjects with LHON due to the G11778A ND4 mitocho...

Safety and Efficacy Study of rAAV2-ND4 Treatment of Leber Hereditary Optic Neuropathy (LHON)

This study is meant to assess the safety and efficacy of rAAV2-ND4 treatment of Leber hereditary optic neuropathy with 11778 LHON mutation.

Efficacy and Safety Study of Bilateral Intravitreal Injection of GS010 for the Treatment of Vision Loss up to 1 Year From Onset in LHON Due to the ND4 Mutation

The goal of this clinical trial is to assess the safety and efficacy of GS010, a gene therapy, in improving the retina functional & structural outcomes in subjects with LHON due to the G11...

PubMed Articles [1624 Associated PubMed Articles listed on BioPortfolio]

Leber hereditary optic neuropathy.

Leber hereditary optic neuropathy is characterized by bilateral, painless loss of vision in children and young adults (generally up to 25 years old). Since its first description in 1871, the understan...

Novel truncating mutation in CACNA1F in a young male patient diagnosed with optic atrophy.

Low vision in children can be accompanied by pallor of the optic disc with little or no characteristic morphologic changes of the retina. A variety of diseases can be the underlying cause, including h...

Genetic Causes and Genetic Diagnostic Testing of Inherited Optic Atrophies.

Hereditary optic atrophies are a heterogeneous group of rare degenerative disease affecting the retinal ganglion cells and their axons which form the optic nerve. With an estimated prevalence of 1 :...

Topographic Macular Microvascular Changes and Correlation with Visual Loss in Chronic Leber's Hereditary Optic Neuropathy.

To study the macular microvascular networks in patients affected by chronic Leber's hereditary optic neuropathy (LHON) using optical coherence tomography angiography (OCTA), and to quantify these chan...

Current and Emerging Treatment Modalities for Leber's Hereditary Optic Neuropathy: A Review of the Literature.

The purpose of this review is to present the current and emerging treatment alternatives for Leber's hereditary optic neuropathy (LHON), emphasizing the most recent use of idebenone and stem cells or ...

Medical and Biotech [MESH] Definitions

Hereditary conditions that feature progressive visual loss in association with optic atrophy. Relatively common forms include autosomal dominant optic atrophy (OPTIC ATROPHY, AUTOSOMAL DOMINANT) and Leber hereditary optic atrophy (OPTIC ATROPHY, HEREDITARY, LEBER).

Atrophy of the optic disk which may be congenital or acquired. This condition indicates a deficiency in the number of nerve fibers which arise in the RETINA and converge to form the OPTIC DISK; OPTIC NERVE; OPTIC CHIASM; and optic tracts. GLAUCOMA; ISCHEMIA; inflammation, a chronic elevation of intracranial pressure, toxins, optic nerve compression, and inherited conditions (see OPTIC ATROPHIES, HEREDITARY) are relatively common causes of this condition.

Dominant optic atrophy is a hereditary optic neuropathy causing decreased visual acuity, color vision deficits, a centrocecal scotoma, and optic nerve pallor (Hum. Genet. 1998; 102: 79-86). Mutations leading to this condition have been mapped to the OPA1 gene at chromosome 3q28-q29. OPA1 codes for a dynamin-related GTPase that localizes to mitochondria.

A rare degenerative inherited eye disease that appears at birth or in the first few months of life that results in a loss of vision. Not to be confused with LEBER HEREDITARY OPTIC NEUROPATHY, the disease is thought to be caused by abnormal development of PHOTORECEPTOR CELLS in the RETINA, or by the extremely premature degeneration of retinal cells.

A maternally linked genetic disorder that presents in mid-life as acute or subacute central vision loss leading to central scotoma and blindness. The disease has been associated with missense mutations in the mtDNA, in genes for Complex I, III, and IV polypeptides, that can act autonomously or in association with each other to cause the disease. (from Online Mendelian Inheritance in Man,, MIM#535000 (April 17, 2001))

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