Clinical Trial Started

Emmett started the clinical trial on Friday.  It's an oral drug called tazemetostat that Emmett will take twice a day.  They treat the drug like it's toxic chemotherapy -- we have to return all the syringes, gloves and wipes that come in contact with the drug.  It comes in a chemotherapy bag.  It's especially important that Katy not come into contact with the drug being pregnant.   Even though adults and other kids have been treated with this drug, the fact that it's still not FDA approved is a pretty big deal.  It's a little unsettling to take something that's treated like poison and give it to your son.

Emmett has to take 28.8 milliliters of the drug morning and night!  That's like having to take two tablespoons of nasty cough syrup twice a day.  He did pretty good on Friday, but since then it's gotten harder and harder to get him to take it.

The good news is that the doctors have already identified the effective dose on this drug, and that's the dose Emmett will receive.  Hopefully this means it will be more effective in treating his disease.  The initial kids treated on this drug generally didn't do well, but they started off at 280 mg/m^2.  The dose Emmett is on is 1200 mg/m^2.  They've given kids up to 1500 mg/m^2, but at that dose the kids started showing side effects (increased fatigue, nausea, blood count suppression, etc.).  So they backed the dose back down to 1200.  The higher the dose, they more they expect will cross the blood brain barrier, and that's where Emmett's disease is.  Hopefully 1200 is Emmett's golden ticket.  (Somewhat ironically, the effective dose for adults is 800 mg given twice a day....notice that it's not "/m^2".  So the kids can get higher doses of the drug than adults, depending on their surface area.  I'm not sure what they've calculated for Emmett's m^2.)


They kept us in the clinic all day Friday so they could do blood draws and run EKGs on Emmett throughout the day to make sure the drug wasn't having any negative acute effects.  Everything looked good so they released us Friday evening.  We're not scheduled to be back in the clinic until next Friday, so Saturday we drove back to Albuquerque and we'll spend a few days here before making the return trip to Denver.  We expected we would need to stay in Denver the entire time that Emmett is on this clinical trial, but it sounds like we will have some opportunities to commute back and forth.

We pray that this drug is effective against Emmett's tumor and that he'll be brave and strong enough to take it willingly.  Go Little Lion!