Today was our last appointment in Cleveland for our 1 year HPN trial medication. We have travelled there 9 times over the last year. Some Alexa and I took a flight, but most we opted to drive so that Christian could come with us. According to mapquest, door to door should take just over 5 hours, but that does not account for travelling with a preschooler who must eat at a certain time, take medication and formula at a certain time, use the bathroom frequently, etc. We never make it in less than 6 hours. Last night we drove through pretty terrible weather with little to zero visibility. It was really scary and we did not arrive until after 11:30pm. We were all exhausted to say the least.
I am excited to not have to make that drive again.
But our team at Cleveland are SO wonderful, that it really was a sad goodbye!
You see, we had MANY hurdles to overcome to get into the trial. To start off, let me explain that Alexa was 14th on the list to get into the trial. They were accepting only 8 children into this trial, so chances were slim, but by some grace we were able to get in! Since we were in quite late, arrangements had to be made very quickly. We were all set to fly off on Monday. The Thursday prior, I received a call from the coordinator informing me that they put a hold on Alexa participating in the study. The legal team at Hyperion were concerned with the logistics of a Canadian travelling to the USA to pick up medication that had not been approved and taking it back over the border. I WAS DEVESTATED!!!
However, the next morning, I received a call from Dr. McCandless, the lead investigator for the trial in Cleveland, informing me that he had spoken with Hyperion and knew what needed to be done for Alexa to participate in the trail....he made all the arrangements with our team at Sick Kids as well as with Hyperion.
It would have been very easy for him to just go to the next person on the waiting list, but he would not have it!
He was our hero!
Once in Cleveland, the entire team, from coordinator, to nurses and doctors have been nothing but fantastic with us. They went above and beyond to ensure that we were always taken care of. As many have heard, nurses have an extremely hard time getting blood access. The very first appointment we had, nurses tried about 4 times with no luck. They were no longer willing to attempt. If they couldnt get a sample, we could not participate, so Dr. McCandless arranged for a doctor from the critical care unit to come up with his portable ultrasound to ensure that we get a good sample at first attempt. Well it worked! And from then on, at every visit, Dr. Dave met us in the research ward with the doplar to do the poke.
They really are incredible!!!
So as happy as I am to now get to go to Toronto for the long term trial, I was extremely sad to say goodbye to such a wonderful team. We will forever be grateful to them for changing our lives with this great new medication!
Coincidentally, just after we signed all our papers, switched into the long term trial, transferred documents to Toronto, and got our 6 month supply of medication, came the most wonderful news!
The FDA announced this morning that they approved HPN 100 for ages 2 years +, and it will now be known as Ravicti. This is news that has been 10 years in the making! It is going to change the lives of UCD patients everywhere and am so grateful to have been a part of the process. When I heard the news, I literally had to hold back tears!
There are a lot of questions now about when it will be available to patients, etc, and that information will come out shortly I am sure, but for now we celebrate a victory for UCD patients.
Cheers to all my wonderful UCD families!