Doctors at the University of Newcastle and in Munich are running a clinical trial of the drug Idebenone for treating people RECENTLY affected by LHON. They are looking for people affected by LHON within the last 3 months who are willing to take part in the trial.
If you live in the United Kingdom and have recently developed the symptoms of LHON then you may be eligible to take part in this trial. If you know someone who may be eligible please pass this information on to them.
The trial has alredy started recruiting people to take part in Germany and in the United Kingdom.
Details of the trial can be found at the University of Newcastle website below.
Study of possible treatments is confused because a small number of people with LHON recover their eyesight without treatment, so individual cases of recovery do not prove that a treatment works generally.
A 'Healthy' lifestyle and balanced diet rich in antioxidants (such as Vitamins A, C and E, Selenium and Zinc) can be recommended. For information on a balanced diet see the Food Standards Agency daily diet site.
Several patients have been treated with antioxidant diet supplements. See the important warning about vitamin supplements below.
Avoid possible triggers such as smoking and drinking alcohol.
Injections of Hydroxycobalamin (Vitamin B12) during the first few months after eyesight loss may help some people. It is thought that sometimes a deficiency of this vitamin may have triggered the symptoms.
A diet supplement called Co-enzyme Q10 (sometimes called Ubiquinone) and a related chemical called Idebenone have been studied. Co-enzyme Q10 is part of the group of chemicals which turn food into energy in the Mitochondria. It is not thought to be effective as a general treatment for LHON.
Researchers in Italy and Japan have tried using
Idebenone on individual patients but there have been few
general tests of it published. One Japanese study suggested that treatment
with a combination of Idebenone, Vitamin B2 and Vitamin C could speed up the process
when eyesight was recovering but did not seem to make more people recover.
Some doctors used to treat LHON with injections of steroids. These are powerful anti-inflammatory drugs which were thought to help reduce any inflammation of the optic nerves during the 'acute' phase of LHON, but again this is now not used as a general treatment.
CENTERWATCH Clinical Trial site publicises tests of treatments and is used to recruit subjects for trials. Dr. Nancy Newman is currently recruiting for a study based in North America (US and Canada), quote:
"to investigate the safety and effectiveness of an eye drop (the test drug) in preventing or reducing vision loss caused by Leber's in the second eye, once a person has suffered vision loss in the first eye."
For more details visit the CenterWatch site and search for LHON.
Gene Therapy Research for LHON
Researchers at the University of Florida have used a synthetic ND4 gene to prolong the life of experimental cells which
contain the 11778 LHON mutation. This is not a treatment for people but shows that gene therapy could
eventually be used to treat the effects of the LHON mutation.
Scientific Paper ref: Ann Neurol. 2002 Nov;52(5):534-42
Rescue of a mitochondrial deficiency causing Leber Hereditary Optic Neuropathy.
Guy J, Qi X, Pallotti F, Schon EA, Manfredi G, Carelli V, Martinuzzi A, Hauswirth WW, Lewin AS.
Department of Ophthalmology, Neuro-Opthalmology Service,
University of Florida College of Medicine, Gainesville, FL 32610, USA.
A group of researchers supported by the Foundation Fighting Blindness from Cornell University, University of Pennsylvania and the University of Florida have published
an article in 'Nature Genetics' (May 2001) describing the use of
gene therapy to treat dogs which have a form of Leber Congenital Amaurosis. The treatment involves injecting viruses carrying a 'healthy' form of the RPE65 gene into the eyes of affected dogs. After twelve weeks the treated dogs showed signs of improved retinal function.
This is an important step in reasearching the treatment of LCA, but it is not yet a treatment used for humans. It is important to realise that LCA is not the same disease as LHON. In LHON the optic nerve is damaged rather than the retina, and different, mitochondiral, genes are involved.
It is encouraging that advances are being made in the possible use of gene therapy.
OPTOBIONICS are developing an eye implant called the Artificial Silicon Retina or ASR. This is aimed at treating conditions where the retina is
damaged but the underlying nerve tissue and optic nerve are still
working. As LHON damages the nerve tissue this treatment is not thought to be useful for LHON patients. Leber's Congenital Amaurosis , a form
of Retinitis Pigmentosa, and Age-related Macular Degeneration are among
the major conditions which this treatment may eventually help.
Important Warning About Vitamin Supplements
Some vitamins are poisonous if taken in large doses. Do not take high doses of vitamins without medical supervision.
This is a drug which is being studied as a possible treatment for Alzheimer's Disease, and may be able to treat the symptoms of LHON during the acute stage. It is NOT a proven treatment for LHON, not even for Alzheimer's, but it has been used to treat some LHON patients. I am adding this information to the LHON Treatment page because of the many questions raised about Idebenone.
Idebenone is being studied as a treatment for Altzheimer's and has recently been studied as a treatment for Leber's Optic Neuropathy. When combined with vitamin therapy it appears to speed up the recovery of eyesight in some patients.
Idebenone may help the cells of the body get energy from food. It works as part of the Electron Transport Chain in a similar way to Co-enzyme Q10, but is thought to have some advantages:
Fewer rogue free radicals than Co-enzyme Q10 causing less damage to the mitochondria and cell.
More efficient conversion of the energy in food to energy available to the cell.
By-passes the Complex I/Co-Q10 pathway in the ETC by stimulating the Complex II and Complex III pathways.
Crosses the blood-brain barrier more easily than Co-enzyme Q10
It is taken daily, by mouth in tablet form.
Some web links relating to Idebenone:
