Humongous Leap Forward

By September 17, 2013November 27th, 2018Tracy's Blog

Charley is finally in a clinical trial! It is so surreal and miraculous to be participating in this historic leap forward in the fight against Duchenne. HT-100 (also known as “Halo”) is an anti-fibrotic medication that we hope will prevent and maybe even reduce fibrosis. When boys do not make dystrophin, a cascade of negative events occurs in the muscles. Fibrosis — or scar tissue — is a key factor in the domino effect. Scientists have known about halofuginone for a long time. But this medicine didn’t make it very far in previous clinical trials for other diseases. One reason is that the drug caused severe nausea and vomiting. HT-100 is a new formulation of halofuginone that involves a special coating designed to enable the drug to bypass the stomach. So far, Charley and seven other boys have been dosed with the new formulation. It is too early to report any data, but I can tell you that right after taking his first dose Charley asked if he could order some sushi!

So far, Charley has taken the medicine twice a day for a full week. Now he’s in a washout period while his bloodwork is analyzed. Once we get the “all clear,” he will go back on the drug for one full month. Then, if things continues to go well, he can stay on the medicine for six additional months. Doctors are testing whether the newly formulated medicine is safe and tolerable. They will also look for early signs of efficacy.

As exciting as it is to be in a clinical trial for a hopeful new medicine, it is also extremely taxing. Charley and I travel to the closest participating hospital, Johns Hopkins/Kennedy Krieger Institute, in Baltimore MD. He had to stay overnight in the hospital three times hooked up to an iv so his blood could be drawn multiple times over the course of 24 hours. There are very long days filled with medical imaging exams, physical therapy evaluations, blood pressure checks, and seemingly endless poking and prodding. And he is missing quite a bit of school. I’m constantly scrambling to find the cheapest and fastest route to Baltimore and back, and to make sure Charley’s brother and sister get where they need to go while I’m gone. But do not think I am complaining, not for a second! I am filled with hope and gratitude for this opportunity, even more so because this trial is all due to the work of Charley’s Fund, our long-time partner the Nash Avery Foundation, and our 17 nonprofit partners who have pitched in to get HT-100 this far along in the drug development process.

In between visits to Baltimore and before school started, we snuck in a family trip to Maine. The first day, we swam in the ocean, strolled around the quaint seaside town of Kennebunkport, then ate lobster rolls and homemade fudge. “Well that was a great day,” Charley announced after dinner. “Now I’m not thinking about the hospital anymore.” I don’t know if it is a survival technique borne from necessity or if he has always had that personality from day one. But Charley has an incredible ability to acknowledge the very tough parts of life and then put them in their place so he can enjoy the great parts. Now if I could just bottle up that attitude and sell it somehow — well I wouldn’t need to fundraise anymore!!