We continue to fight to find a cure for ATRT. This is a long & complicated fight but we are making progress, albeit slowly. This is the research that you & all our generous donors have helped to support over the past few years. We couldn't do this without you.
Epizyme recently announced results of their Phase I data on patients who have INI1-deficient tumors. Recall that phase I is a dose escalation to ensure safety so most patients get a dose that is substantially below the final dose. That said, the results are quite encouraging – they even had three complete responses. This is quite unusual in a phase I trial and it is now going on to phase 2.
We know this isn't yet the cure we need but we will keep working until we get there.
To those families that are struggling daily with AT/RT, let this be your Hope.
To families that have lost their beloved child to this disease, we do this to honor your brave babies.