I am so sorry to hear about your daughter with Batten. My twins have Niemann Pick Type C, it's similar to Batten.

I have been pursuing iPS cells. They can make these from a person's skin and hopefully then correct the genetic mutation and then make healthy neurons for future transplant. I have been working with MIT on it. It's a ways off before we can get these types of transplants -- our biggest hurdles will be the FDA and it's going to be a nightmare to get them to approve anything. Look at how slow it is to get drugs approved!

There are other cells besides iPS. You would have to go outside of the US for stem cell treatment for your child. It worries me a lot because there are lots of snakeoil types who want to sell us hope. I think the South Koreans are doing amazing work. But the technology is not there yet from everything I have researched.

I attended the World Stem Cell conference in Pasadena this past year -- I would get in touch with them and see what they have to say. There were two prominent doctors from Mexico in attendance doing interesting work.

I have told my Batten friends about cyclodextrin and asked your researchers to test it to see if it could interact with the liopfuscin that is stuck inside the cell. You need to look for things you can get your hands today to help your daughter. This is the strategy I am pursuing.

Again, sorry you are suffering with this horrible disease. There are many people facing the same nightmare and it's heartbreaking.