EPI-743 Day One

Our trip is going well, the Bay Area is beautiful!  This morning we drove to the Lucile Packard Children's Hospital at Stanford for Hudson's echocardiogram and EKG.  He did fine for it, but Jake and I were nervous about getting the results.  After his heart surgery in Houston, the surgeon and his cardiologist told us that they didn't know the reason Hudson's heart got worse so suddenly, and there is a possibility it could happen again.  So we go into echocardiograms very nervously! 

Huddy looking tired after his echo

After the tests we had a few hours before our clinic appointment with Dr. E, the doctor in charge of the trial.  We went to lunch, where Hudson busied himself sticking crayons in his ears, and then found a store where we could buy him a Stanford shirt.

Our clinic appointment with Dr. E was very informative.  He was so nice, and entered the room saying, "So this is the famous Hudson!"  At times it seemed like Hudson was not going to be accepted into the trial, at first because of aspects of his heart condition, and then because of the GI infections that he is currently still fighting.  Dr. E was worried that the absorption of the trial drug would be affected, but in the end decided to go for it, treat Hudson and hope for the best!  We are very thankful for that!

We learned today that Huddy is the 25th person in the country to be treated with this drug!  I just can't believe that, it's hard to wrap my head around it.  I also learned a lot more about the trial.  Currently the trial is in an Emergency Treatment phase, and this phase is going to end at the end of the month.  To qualify for the emergency treatment a patient has to have mitochondrial disease and be considered 90 days from end of life care.  Dr. E explained to us that Hudson's mito and especially his heart condition qualify him for this emergency treatment phase.  I want to stress that we do not feel that anything is going to happen to Hudson within 90 days, it's more about the very unpredictable nature of mito and his heart in general.  The next phase that the trial moves into next month is the placebo phase, where a number of mitochondrial disease patients with a specific subset of mito called Leigh's Disease either receive the drug or a placebo.  We honestly feel blessed and very thankful that Hudson was accepted under the current emergency treatment protocol. 

Dr. E showed us the results of the echo, which were pretty good.  It showed left atrial dilation and mild mitral valve regurgitation, but these are consistent with his post-op echos in Houston, so nothing has gotten worse.  Great news!  Dr. E did a neurologic exam and videotaped Hudson per the trial requirements, and then we were off to do labs. 

Most of the other trial participants are doing a brain spec that can look at the function of the mitochondria in the brain, and that would have taken place tomorrow.  Dr. E and the anesthesiologist feel very strongly that because of Hudson's heart we shouldn't sedate him unnecessarily though, and so they are waiving the brain spec requirement.  So we'll have tomorrow off, and then Friday morning Hudson will be dosed the first dose of the trial drug.  They will run labs and another EKG for safety purposes, and then we leave Saturday.  We will follow up with Dr. K in Houston for labs during the trial period, which is 13 weeks.  At the end of 13 weeks we will return to Stanford to run the baseline tests again, and from that point we can choose to keep Hudson on the drug or discontinue it.  I just know we'll have great results, and I'm so thankful that Hudson was chosen to take part in this.  Dr. E's excitement about the trial was very evident, and I feel confident that we'll have good things to report at the end of the trial period.  I'll update again after he gets the first dose to let you know how he did!