WOW, it is great to be on the other side of the treatment approvals!!!!
As of Friday, Damian has taken the experimental treatment 8 times - he has taken it every 3 days since April 22, 2022 [watch his first time taking it here!].
Everyone has been excitedly asking me: "Have you seen a difference yet???"
The answer is a solid... maybe?
What We Can Expect
Maybe this heading is misleading, because the truth is we don't know what is going to happen. We do know that one boy has taken this drug before, albeit a smaller dose amount and he was far further regressed than Damian is when he was on it. That study very importantly proved the safety of the drug, and the boy's mother told me she did see some proof of its efficacy (though it ultimately, heartbreakingly, appeared the dosage was too little and too late for her son).
This drug is intended to stop the buildup of Sphingomyelin in Damian's brain as a byproduct of inhibiting the enzyme FAAH (short for Fatty Acid Amide Hydrolase) from doing it's job. Inhibiting FAAH primarily allows certain lipids (like the cannabinoid "anandamide") to build up, but it also showed in the animal testing (lab mice with ASMD) to improve their ASMD symptoms, which is what we're really really hoping for Damian!
As far as Damian's medical team goes, it seems no one is expecting to see an immediate change - it could be 3 months or longer before we see some concrete evidence that the drug is doing something (which is why I can't wait to see if this drug is a miracle pill before raising the rest of the money we need for the next brain treatment). But the good news is that... it's POSSIBLE that something is already happening.
What I've Seen So Far
First and foremost, I haven't seen any negative side effects since Damian's first dose. No immediate bodily reactions, no weird digestive issues, and not even an indication that Damian thinks it tastes bad. That is all a HUGE relief and truly the first big hurdle. Damian has been more tired than usual, but so have I - the frequent traveling back and forth to Los Angeles (about a 40-50 minute drive one way) has taken a lot out of the both of us, so I'm pretty sure that's the only reason he's been so tired.
He's only been on this drug for a few weeks, not even a month, and yet I do think I've seen some positive differences (granted, my hope that this drug works is packaged with a ton of emotions, so take my word for what you will):
I think Damian seems to be more enabled to move. The drug isn't designed to make him stronger, but you know how I've said before that Damian seems to have more energy and drive and seems to be less mentally inhibited during a growth spurt? Well, while I don't think his muscles are stronger now than they were a few weeks ago, but he does seem to be more like he is during a growth spurt: more alert and more able to control the body he has, if that makes sense. Even Damian's physical therapist noticed multiple times this week that Damian has been exerting more physical effort into trying to crawl and stand (we typically do this in our physical therapy sessions, but for the past several months it's really been just Damian's therapist moving his limbs for him). This week, Damian has been really trying to push his muscles! He still isn't going anywhere, but just the fact that he's clenching his muscles at the right time is great to see!!
Related to that, I really think Damian's head control and body stabilization is more steady. He still tires out quickly, understandably - but today for example, I was holding him up on my lap while holding on to only his hands and Brock and I were amazed at how long he tolerated that without collapsing onto me! I'll be working on editing a video soon of all the footage I've taken over the past few weeks so hopefully you can see what I mean!
Next Steps, per the Treatment Protocol
Damian is currently taking 0.3 mg of the FAAH Inhibitor drug every 3 days. The protocol (our roadmap) for this study recommends Damian does this for 3 months, then at that point, we should increase the frequency so he's taking it every 2 days for another 3 months. Then we increase it again and will start giving it to him every day indefinitely.
Since this is an experimental study, with all the testing that is going on, the doctor may look at all the blood tests and MRIs, etc. and hear my feedback and choose to change some aspects of the protocol as we go.
He did that this week!
Part of the original protocol said that Damian needed to do frequent blood draws, about twice a week, for the sake of monitoring any changes. However, because Damian's first 7 doses had gone without a hitch, and all the basic blood tests that Damian had taken throughout the past couple weeks showed little to no changes whatsoever, the doctor said it should be fine to just do blood draws once a month until we increase the dose frequency. Not drawing a ton of blood for a month can only be good for Damian, so I am 100% in agreement on this!
AND, because the doctor is not requiring a blood draw before every dose, last week the doctor was able to send us home with a 30-dose supply so we can just give Damian his treatment AT HOME!!! This coming week we don't have ANY appointments at Children's Hospital and it's been a long time since I've been able to say that!! I'm really hoping we'll get to see "peak Damian" a bunch this week because we won't be wearing him out with all the traveling. I can't wait!
[This Instagram reel shows how I give Damian his treatment at home - THANK YOU to everyone who has shared this reel, which about 10,000 people have now already watched!!]
What Else is Coming Up?
This summer is going to be a big one - Damian has 2 MRI's coming up in a few weeks (which he will likely be sedated for), then his second-ever surgery has been scheduled for the last week in June (to place a feeding tube in his intestine - more on that soon), and we are tentatively planning to start the liver/spleen treatment Damian needs in August (Enzyme Replacement Therapy). On top of that, Damian's 3rd birthday is on May 24th (just in a couple weeks!!) and not only is that a big birthday for any toddler, let alone one fighting ASMD, but 3 is also the age that the state completely pulls funding for all the therapies Damian is receiving now and transitions resources over to the public school system - which means Damian may start going to a special needs preschool class soon. I get sick to my stomach every time I think about dropping Damian off at a curb of a big school... so we're still thinking this through.
Of course, I am more determined than ever to get the rest of our campaign funding in hand by next month!! Recent conversations with the Wylder Nation Foundation surrounding the brain treatment that is still in development just reiterated the importance of getting this funding as soon as possible, and by golly, we are GOING TO DO IT!! Thank you all so so much for continuing to share our story - our posts, Instagram reels, campaign updates, events, and even creating your own content to spread the word!!! We are SO blessed by all of you. You all have made SUCH A DIFFERENCE in this community already. Multiple children with ASMD are now in the process of getting started with the experimental treatment Damian just started, because of your help. We are so so so grateful.
More updates coming soon!!!