Sunday, October 11, 2015

Blindness Awareness Month

This month, October, is Blindness Awareness Month.  The time we remind people to remember what Finley and other vision impaired people go through on a daily basis.

This month, Mat was able to go to the University of Michigan (where our RDH12 researcher is) with a few other parents in our group, and talk to her about where the research stands.

So I thought today I would post what he learned:

"It has been awhile since I have been able to give a full update.  Mainly because there wasn't much new to tell.  Buton Friday, Mike, Jogin, and I went to Ann Arbor and visited with Drs. Thompson and Ali.  We were able to get the best view of where the RDH12 gene therapy program sits in a while, and I will say I feel better about things since the last time I wrote the group.  As I think I mentioned last time, Dr. Thompson wanted to go back determine if it might be better use a different version of the virus to deliver the RDH12 gene.  This decision most certainly cost us precious time but Dr. Thompson felt it was necessary to ensure the gene therapy was safe.  When we saw her on Friday, she felt she has now completed this work and has a virus she feels is safe.  With that out of the way, she and Robin both agreed they were ready to move forward and finally start the work that is necessary for the clinical trial - which is the toxicology studies.  This was very reassuring to hear. Because we are now at the point that they think a clinical trial is possible, there are a couple of other important developments they shared with us. 

First is that they are starting to build a patient registry.  In other words they are trying to build a complete list of every RDH12 patient out there plus the medical records from those individuals.  In order to know that the gene therapy is working, they need to know how LCA-RDH12 normally progresses.  

They other import event coming up is that Robin Ali is going to take the RDH12 program to the board of his company and request that they take it on.  He will do this before Christmas.  If they agree (and Robin has no doubt that they will), they will help in supporting the clinical trial.  Of course the only way we can assure that clinical trial goes ahead without a hitch is for us to have the funds to support it (and we are still short of what we need) but it is will help to have the support from his company.  Not only in terms of money but even more so in people and expertise.  People that have done this successfully in the past.

Last time I wrote you, I was worried we were moving backwards but this time I can say we are moving forward again.  It is taken longer that we originally planned to get here but those critical clinical trial enabling tox studies are now happening.  I will continue to keep you up to date as new information comes in and will definitely let you know about the registry once I get the information from Dr. Thompson."

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