3 ms·

Another use of a modified virus is to deliver an antibody to VEGF, the factor that causes macular degeneration (blindness). Prof. Elizabeth Rakoczy has been rec

by pdm55 9y ago

Another use of a modified virus is to deliver an antibody to VEGF, the factor that causes macular degeneration (blindness). Prof. Elizabeth Rakoczy has been recently awarded the 2017 CSL Florey medal for this work. http://www.aips.net.au/news-events/the-florey-medal/2017-professor-elizabeth-rakoczy/ http://www.aips.net.au/news-events/the-florey-medal/2017-pro... The video has a cartoon showing the modified virus being inserted in the retina. I think 40 patients have been successfully treated in a initial 3 year clinical trial. I found these links to abstracts of her recent publications, https://www.socrates.uwa.edu.au/Staff/StaffProfile.aspx?Person=PiroskaRakoczy https://www.socrates.uwa.edu.au/Staff/StaffProfile.aspx?Pers..., but I cannot find an open source research document. Some of her team members: http://www.scienceinpublic.com.au/floreymedal/2017-csl-florey-medal-photos http://www.scienceinpublic.com.au/floreymedal/2017-csl-flore...

pdm55 9y ago
Some figures: I got my "every second month" injection of Eylea this morning, https://www.macular.org/eylea-injection-treatment https://www.macular.org/eylea-injection-treatment. It cost $800 Australian dollars. Under our health scheme, I get $600 back. The guy beside me in the waiting room has been having injections for 17 & 1/2 years, that is, since the year 2000. He gets state-assistance to fly down 800 km to our capital city from the country. The lady across the aisle from me in the packed waiting room, has a relative drive her down from a regional town every second week, as she has macular degeneration in both eyes, with the alternate eye being treated on alternate visits. I give you these cases to illustrate that a lot of money is being presently spent on treating this disease. I have heard the incidence of macular degeneration in Australia described as an epidemic. Obviously, everyone would like a cheaper, longer-lasting treatment. That's what I am hoping the modified-virus treatment provides. The figure that jumps out of the page for me in the modified-virus report, http://www.aips.net.au/news-events/the-florey-medal/2017-professor-elizabeth-rakoczy/ http://www.aips.net.au/news-events/the-florey-medal/2017-pro..., is "This therapy has been licensed to US company Avalanche Biotechnologies Inc., which has raised over $400 million to progress the treatment through clinical trials and bring it to market." This figure of US$400 million takes my breath away. I note one of Prof. Rakoczy's clinical trials in Australia on 32 patients was done with a AU$370 thousand grant, among others, https://demo.ands.org.au/long-term-follow-gene-therapy/107896?source=undefined https://demo.ands.org.au/long-term-follow-gene-therapy/10789.... I guess the US$400 million trial will be more extensive. This means a few more years will have to go by before this comes to market (if it indeed does). I have found an open source article about Prof. Rakoczy's trial, https://www.ncbi.nlm.nih.gov/pmc/articles/PMC5161436/ https://www.ncbi.nlm.nih.gov/pmc/articles/PMC5161436/. I note they removed the vitreous humor to place the modified-virus under the retina (termed a vitrectomy), recording that "It is not yet clear if subretinal injection can be done safely without vitrectomy". They seem very pleased with the results of that phase 2 trial.

by pdm55 9y ago

pdm55 9y ago

Some figures: I got my "every second month" injection of Eylea this morning, https://www.macular.org/eylea-injection-treatment https://www.macular.org/eylea-injection-treatment. It cost $800 Australian dollars. Under our health scheme, I get $600 back. The guy beside me in the waiting room has been having injections for 17 & 1/2 years, that is, since the year 2000. He gets state-assistance to fly down 800 km to our capital city from the country. The lady across the aisle from me in the packed waiting room, has a relative drive her down from a regional town every second week, as she has macular degeneration in both eyes, with the alternate eye being treated on alternate visits. I give you these cases to illustrate that a lot of money is being presently spent on treating this disease. I have heard the incidence of macular degeneration in Australia described as an epidemic. Obviously, everyone would like a cheaper, longer-lasting treatment. That's what I am hoping the modified-virus treatment provides. The figure that jumps out of the page for me in the modified-virus report, http://www.aips.net.au/news-events/the-florey-medal/2017-professor-elizabeth-rakoczy/ http://www.aips.net.au/news-events/the-florey-medal/2017-pro..., is "This therapy has been licensed to US company Avalanche Biotechnologies Inc., which has raised over $400 million to progress the treatment through clinical trials and bring it to market." This figure of US$400 million takes my breath away. I note one of Prof. Rakoczy's clinical trials in Australia on 32 patients was done with a AU$370 thousand grant, among others, https://demo.ands.org.au/long-term-follow-gene-therapy/107896?source=undefined https://demo.ands.org.au/long-term-follow-gene-therapy/10789.... I guess the US$400 million trial will be more extensive. This means a few more years will have to go by before this comes to market (if it indeed does). I have found an open source article about Prof. Rakoczy's trial, https://www.ncbi.nlm.nih.gov/pmc/articles/PMC5161436/ https://www.ncbi.nlm.nih.gov/pmc/articles/PMC5161436/. I note they removed the vitreous humor to place the modified-virus under the retina (termed a vitrectomy), recording that "It is not yet clear if subretinal injection can be done safely without vitrectomy". They seem very pleased with the results of that phase 2 trial.