Is The New Catena Drug a Good Treatment Option for Leber’s Hereditary Optic Neuropathy (LHON)?

November 11, 2010

In Jan.2008 my son began to have sudden blurred vision that ended with a weeklong stay in the hospital. He was eventually diagnosed with Leber’s Hereditary Optic Neuropathy (LHON), an inherited disease that has damaged his optic nerve, leaving him blind in both eyes with some peripheral vision. There is no treatment.

He is 20 and in his third year of university and doing well, using assistive software. My question is, will stem cell treatment help him and repair his optic nerve even though it has now been 2 years. Does the length of time he has been blind affect his recovery?

Also, there is a new drug called Catena (from Santhera Pharmaceuticals) that is supposed to help restore vision. With his degree of blindness, only 25% chance of some recovery of vision is possible. Is this treatment worth pursuing?

 

 



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6 Responses to “Is The New Catena Drug a Good Treatment Option for Leber’s Hereditary Optic Neuropathy (LHON)?”

  • Sarah

    Was wondering if either of you tried the Catena . My
    Mother was recently diagnosed with Lebers and is only 45. We are in search for anything that may help.

  • Jody Cunningham

    Hi
    Just reading through this article and was wondering if you tried this drug and what the results were. Am looking into seeing if there is a cure or any new promising treatment to try for I have LHON.
    thanks

  • Jim leeder

    Lhon is very different to LCA, and is caused by mitochondrial rarher than nuclear genes. Although some research is going on into gene therapy for LHON, it is still very experimental. Idebenone (brand name Catena) has been shown to give some sight recovery in some people. It is taken daily and any recovery will take months to begin. The chance of recovery will be higher for those who start taking Idebenone soon after symptoms begin, but recovery can happen years after onset.

    Santhera is applying for authorisation to market Idebenone under the name Catena , and doctors in Italy are already prescribing it to people who present with LHON. Although only effective for some patients, it is better than no treatment at all.

  • ari weitzner

    actually, Leber’s congenital amaurosis ( LCA) and Leber’s hereditary optic neuropathy (LHON) are 2 distinct diseases with very different degrees of vision loss.

  • I touched upon some of the gene therapy treatments for Leber’s Congenital Amaurosis in my recent article on gene therapy. It can be found at the very end of the article, just before my conclusions. (I don’t know enough about Leber’s to know if LCA is the same or different from LHON.)

    Here is the link: http://tinyurl.com/GeneTherapy-RP-AMD

  • Dr. Ari Weitzner

    i think i saw a piece about catena for leber’s, but i dont think it was published in a peer-reviewed ophthalmology journal yet. i quickly checked it out on google- they did a randomized, placebo-controlled study and showed a modest improvement, so i would try it out and not wait, as it looked safe.

    stem cells can only be used for eye surface disease, not retina or optic nerve. it will be many many years before we have success in retina and nerve.

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