Recently I had a conversation with Melissa Thomas, a mom to two boys with Hunter Syndrome. Her son Nathan passed away from the effects of the disease about five years ago, and Easton, her youngest son, soon thereafter became the first child enrolled in the Phase I/II intrathecal idursulfase clinical trial. She shared her thoughts, perspective, and learning from the last almost five years of participation of the trial and its extension. We are grateful for her time in discussing these issues with all of us since her thoughts are beneficial not only to other patients, but also to clinicians, regulators, and pharma who are involved in the clinical trial process.
1. When and how did you hear about the clinical trial?
I heard about the clinical trial from our geneticist. He had been telling us about the promising things they were seeing in the animal models and knew that they would be seeking approval for a clinical trial. He would give me updates periodically during our weekly infusions. When it was evident that the trial would be starting soon, he let us know and I contacted UNC. This was around June 2009.
2. What convinced you to want Easton to participate? Did you have any reservations?
Shortly before the trial started, my older son [who also had Hunter Syndrome] passed away. I had watched him decline for years neurologically and physically. I knew that Easton’s fate would be much the same. I had to give him every chance I could. I also knew that both of my daughters are carriers for the disease and I could potentially help theirs and many other children in the future. I also knew that if it didn’t work, I had tried everything in my power to help his future.
The only reservation I had were all the what ifs, what if it didn’t work, what if there were reactions, what if he got an infection, or it caused problems with his brain.
3. I know you write a blog, not just about Easton but about your whole family, so what made you decide to write about the trial on your blog?
I started the blog in theory that I figured pharma companies might be reading it as well as potential families looking into the trial. I wanted to be able to give them, as well as my family, a first look at what was really happening. When my older son Nathan was in the Natural History study for Elaprase, and the clinical trial for Elaprase was ongoing, I would have given anything to know what was happening and how it was working, but this was before blogging and social networking. I also didn’t want my other children to feel like all I cared about was what was going on with Easton, so I tried to include them as well and make it more of a family blog. I also thought it would be a nice way for people to get to know us a little better.
4. If you look back at the beginning of your participation in the trial, what do you wish you’d known then?
I wish I would have been more assertive with my needs during the beginning. I wish I would have known about the perks of the hotel being able to store the toys. I would have liked to have a list of places created by parents of Hunter boys of places to eat, play, entertain. I don’t think doctors get what is important to us in terms of facilities and what many of our kids can and can’t handle when they are neurologically impaired by the disease.
5. How have your writings about Easton and the trial changed over time?
I started out with all of the good things that were happening, and as time went on I started to insert my needs hoping people involved with the trial might read about them and do something. I also wanted my son to be not just another number to them. I wanted them to see and know the face of who they were helping. My blog has kind of fizzled; I haven’t written anything for months, but hopefully I will update it very soon.
6. What has been the best benefit to Easton of the trial?
The best benefit for Easton has been his cognitive skills increasing. When we started the trial, he had reached a point that is typical in Hunter boys where they reach a certain level and then kind of just stop. They aren’t really losing skills, but they aren’t gaining new skills either, Easton was at this stage and the older he got, the further behind his peers he would be. Not only did his cognition stabilize, it increased!!!
7. Other than the benefits of the drug to Easton, what do you think has been the best thing about participating in a clinical trial?
The best thing about being in the trial besides the drug has been the opportunity to meet and interact with other families going through and experiencing the same things. Watching Easton make new friends that he otherwise would not have. Also, working one-on-one with Dr. Muenzer.
8. What have been any scary parts about the trial? Have you ever considered stopping?
The scariest part of the trial for me was being the first one, the not knowing how it would all work. I also had never been away from my other kids, let alone out of the state, without my husband James, so driving and getting around unknown territory was nerve wracking to say the least. I was afraid my other kids would resent me for being gone so much and missing important things in their life. I worried they would resent Easton for monopolizing my time even more than he was. I worried about my marriage and the strain this added trial would make.
I have thought with all of the problems Easton had with the device [the original port-a-cath had a number of problems – see past post], that I would like him to just be a normal boy, no more surgeries, hospitals, no doctors. I sometimes just wanted to let him live and enjoy his life without all this. Deal with nature and its natural course. Then I would look at all of his progress and have to put into perspective that these problems would pass and he would be so much better for it. With all the headaches he endures, I have remind myself that it’s only a couple of days a month and then he’s fine. Sometimes I feel drained and just want normalcy.
9. The next phase of the drug approval process for IT Idursulfase, a pivotal Phase II/III trial, has now begun. If you could give families participating in this trial three pieces of advice, what would they be?
1. Be prepared financially, the reimbursement isn’t near enough to cover your expenses.
2. Plan special one-on-one time for your other family members.
3. Make time for yourself or you’ll burn out.
10. If you could give drug companies designing clinical trials for rare disease three pieces of advice, what would they be?
Listen to the parents, they know more about things than you think. Plan for families that struggle financially, make sure their needs are being met and do things necessary to help ease the burden of them being in the trial such as pre-pay their reimbursement before they get there. This way they don’t have to worry about how they are going to do this and pay their bills. Be aware that they are using their time off work to do this. Personalize this a little bit.
11. Anything else you’d like to add about your participation in this trial or about clinical trials in general?
I would do it a hundred times over!