Changing the world one clinical trial at a time

We are so grateful for the people from start to finish who worked so hard for this particular clinical trial, the people that may never know the impact their work has, aside from seeing the results on a spreadsheet. This is why we were so moved when we had the opportunity to meet the incredible people at AGC Biologics and they could see the results of their hard work face to face! I will never, ever forget such an emotional day!! All these people changing the world in one room, changing the course of someone’s life who probably isn’t even born yet, so no family has to hear “I’m sorry, there is no cure” ever again for MLD! How incredible to be a part of that!!

Per The World Health Organization – A clinical trial is a type of research that studies new tests and treatments and evaluates their effects on human health outcomes

Ella and Eli ten years ago 💗💙

When we found out about the clinical trial for MLD over 10 years ago, we didn’t know much. Thanks to an MLD mom that I was friends with on Facebook, she introduced me to another MLD mom who was in the trial in Italy. She was very informative and explained what gene therapy for MLD was like for her child, and how he was doing in that moment. It was nothing short of amazing which helped us push even more for inclusion into the trial. At the time, there were less than 10 children in the world treated with gene therapy for MLD.

After doing our research, I remember we told our doctors what we were thinking about doing and how the clinical trial was going to be our only option that we would fight for. The doctors were surprised and some not in a good way. We did this research on our own with help from a few friends who were in the pharmaceutical field. We never had a doctor at any local hospitals mention any clinical trials for MLD. I’m not sure if they didn’t know about any, or if they didn’t believe in it, but Im guessing it could be either or both. We only knew one thing, and that was that if we did not get accepted, we would give Eli and Ella the best life possible in the time that we had with them.

Unless a trial has been around a long time, you don’t know much about it and isn’t that the point of a trial, to learn if a treatment works or not?

It was over six months of doing everything we could to be accepted, which was a lot of time and that time was filled never ending prayers. We still didn’t know everything, but we believed this was the way to go. We did do our research though, and learned about the process, the risks and benefits. They all outweighed the thought of losing them so we agreed and we never looked back.

We were told that we would be followed for a long period, which ended up being 10 years since we first went to Italy. During those years, Eli and Ella received wonderful follow up. Many tests to monitor how and if the disease was progressing. It was quite a challenge at times, but in the back of our head we always believed that we/they were making a difference for the future of MLD treatments. If this trial worked and prolonged their life, then surely we could help others from suffering the ultimate fate of losing their child/children. Eli and Ella would be two of the kids to change the world, and if we continued to go to our follow ups, they could learn how the treatment has truly affected them.

We had to continue to be followed; going to Italy and sometimes getting tests done here at our local hospital, but in our eyes, this was a small price to pay for saving the life of two of our four children.

I don’t like to think back to those early days as they were hard. Living in another country, not speaking the language, being away from your support system. None of it was easy, but as I said this past week, it was all worth it. I recently read that between 20 and 30 percent of people enrolled in clinical trials drop out! I’m glad we stuck through it and we hope they gathered some good information on the disease process from Eli and Ella.

Our particular trial was different than most because of having to be in a foreign country. It takes a toll on many things, relationships, the kids in the trial, the ones at home and it changes you… how could it not? The alternative is worse and we will focus on that ALWAYS.

I have always said and always will, that when you or someone you love is diagnosed with a disease that they don’t know much about, don’t give up! Research, research and research some more. You never know what is out there and who you will meet, just like us. I would have never imagined that a story like ours, that started with us hearing that two of our kids were not going to make it, would be changed by social media, a fight like no other, and a clinical trial more than 4000 miles away in another country.

Ten years later 💙💗

The people who are crazy enough to think they can change the world, are the ones who do – Steve Jobs


~ by Rebecca on May 1, 2023.

One Response to “Changing the world one clinical trial at a time”

  1. Well said Becky! Nothing more to say except…THANK YOU ELI, ELLA and VIVIAN FAMILY! Sticking with the trial till the end undoubtedly taught the researchers many things. Hugs and prayers always!

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