Potential Major Breakthrough in Treatment of Wet Age-Related Macular Degeneration

February 28, 2012

A single injection of a new gene therapy treatment from Avalanche Biotech could possibly stop the advance of neovascularization in the wet form of AMD for several years. Human clinical trials are currently underway.

Here is how I commented about this breakthrough in my writeup:

“If this approach is successful, it could result in a “game change” in the way wet AMD is treated. Instead of multiple injections to gain control of neovascularization, one injection at the first signs of neovascularization could stop it dead in its tracks and the effect could last, potentially, for several years.

In addition, it is conceivable that patients at risk of developing wet AMD could receive a therapeutic injection of AVA-101 to prevent the occurrence of wet AMD. This would be a definite paradigm shift in the treatment of AMD.

With the human clinical testing now underway, we should have an indication of initial results with this unique approach within about a year.

Stay tuned!”

In a nutshell, Avalanche has developed a proprietary drug delivery system, known as Ocular BioFactoryTM, that continuously secretes a therapeutic protein over an extended period following a single subretinal injection, thus avoiding the need for frequent intraocular injections of recombinant anti-VEGF protein.

To read more about this potential “game changing” breakthrough in the future treatment of wet AMD, please follow this link.

 

 



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6 Responses to “Potential Major Breakthrough in Treatment of Wet Age-Related Macular Degeneration”

  1. ari on February 28th, 2012 11:53 am

    if this is a device which is secreting drug over a long time, then it’s not gene therapy. im a little confused.

  2. Irv Arons on February 28th, 2012 12:50 pm

    Ari,

    It is not a device, but a gene therapy biologic. It is injected subretinally and the virus vector expresses its therapeutic protein that initiates a response from the retinal tissue resulting in a continuous steady-state rate level of therapeutic protein, eliminating the need for frequent re-injection of an anti-VEGF.. As noted in the writeup, based on preclinical studies, the therapeutic effect was maintained for at least 18 months and has the potential to last for several years.

    This could truly be a “game changer”!

    Irv

  3. ari on February 29th, 2012 8:47 am

    wow. impressive. thanks for the clarification.

  4. David Richardson, MD on February 29th, 2012 9:15 am

    I hate to be a cynic here with such potentially good news, but it is Merck that is behind this so expect something else to be a “game changer”: the price. I would be very surprised is this was priced below $5,000 a treatment. Now, compared to Lucentis, that might be a “deal,” but compared to Avastin it would be a “raw deal.”

  5. Irv Arons on February 29th, 2012 12:07 pm

    Dr. Richardson,

    You may be right about the price — I really have no idea what it will be — didn’t ask as it much too early in the development, but you’re wrong about Merck being behind it.

    Merck is just the supplier of the protein that is added to the virus vector, according to my understanding. Not to say that if it is successful, that Merck might become an investor.

    I still think that 1 injection (even at $5000) compared to 8-12 injections yearly (even for Avastin), might make a difference to the patient and doctor doing the treatment (fewer office visits, etc.).

    I guess we’ll just have to wait and see.

    On another subject, I have asked the company about whether they are planning on U.S, clinical trials and will pass along the information when I receive it.

    Irv Arons

  6. Irv Arons on February 29th, 2012 11:54 pm

    I heard back from the company and they will consider U.S. clinical trials provided they get positive results from the current clinical trial in Australia.

    Irv Arons

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