April 9, 2023. And so it begins.

We had a lovely spring break in Florida. We spent most of it in the Keys, where we snorkeled and learned how to pilot boats. Two huge spotted eagle rays swam under Alyssa. We saw key deer who swim, greedy pelicans, lizards bobbing their heads for a date, lots of fish including “needle fish skipping on top of the water like a skipping stone” (Google it for a neat video), tarpons, and sharks. Then our return flight got cancelled so we spent a couple extra days on Miami, where we saw a 6-foot shark near the beach. 😳 Very exciting! It was weird to see people exit the water, and then go back in <10 min later.

Recap: our daughter Alyssa has a rare immune disorder called C3G that deposits proteins that hurt her kidneys, so we are trying to preserve her native kidneys. Other C3G patients have gone through multiple kidney transplants. Alyssa is no longer in remission, and there are limited medication options, being a rare disease. We have now started her on an investigational drug trial, which means she is a human guinea pig to test out a new medication. The medication pegcetacoplan has already been on the market for another condition, so we feel like this is a safe medication to start her on, as well as one of the first medications to actual target the earlier stage of her problem. Like diabetes and hypertension, there is no cure for this condition, so we pursue treatments for best possible outcome. She is on an immunosuppressant (MMF/Cellcept capsules) in addition to the trial medication. We wear cough masks, and I struggle with worry and anxiety about preventing infections that make her C3G more mad, but I also worry about smothering my kids with worry. I put the “mother” in “smother.”

She completed Trial Visit #3. This is the big one, where she starts the medication (or placebo, as it is a blinded trial). We spent the week in Iowa, so she can complete 2 appointments and doses throughout the week. I worried it would be intense in learning how to administer the medication via subcutaneous infusion via pump, but it was rather straightforward. Alyssa saw how small and thin the needles were and she was relieved. In fact, she was more worried about the EKG stickers than the needles, because the sticker adhesives trigger her mild allergy and she gets itchy. 🤭

TLDR (“Too Long, Don’t Read” this if you don’t want details): This next paragraph explains the trial in layman’s terms. The first half of the year is a “closed label” or “blinded trial,” meaning people don’t know if she’s getting the real medicine or the placebo, except for a select few people running the trial. Even the doctor and nurse don’t know. A placebo is a fake drug, to go through the motions of giving someone medicine. For some people, there’s something called the “placebo effect” where they think it’s helping them feel better or worse. Fascinatingly, some people’s body actually respond to placebo effect, like “ohhh my pains are going away” or something as extreme as a false pregnancy. This blinded trial is to test and document results, to prove whether or not the medication actually makes a difference compared to placebo, specifically in C3G patients. If the medication does not prove it actually makes a difference compared to placebo, then why bother selling that medication? Thankfully the USA has organizations such as the FDA (Food and Drug Administration) who set these guidelines so people can’t make false claims and sell junk to desperate sick people. Interestingly, I am not allowed to share any test results with Alyssa, such as to affect her thoughts that she may or may not be getting the medication, because they want to avoid any placebo effect. Personally, I think it’s sucks to do all the infusions for 6 months and it turns out to only be saline (salty water) placebo. But it’s a small price to pay to get the real medication for the 2nd half of the year. The 2nd half of the year is “open-label”, where everyone gets the real medication. Joining this trial is a great opportunity to get the drug for free and (in our case) even be paid to get this medication, plus get regular medical attention and monitoring of her symptoms. Is it scary to make her a trial participant (human Guinea pig)? Of course it can be. So Al and I weigh the risks versus benefits and do the best we can. Having a great medical team also helps. And if Alyssa has any serious concerns along the way, we can withdraw from the trial. Some people are uncomfortable with joining trials, which is understandable too. Knowing that pegcetacoplan has already been tested and proven safe and effective (effective means the medicine actually works better than placebo) on a similar condition helps us know that the odds are in our favor. The nurse tells Alyssa, “You are so brave. You are helping other kids in the future.”

The “subcutaneous infusion” means this. There is a pump that is programmed to slowly push 15 mL liquid (adults get more) into tubes into 2 small needles into her body. Subcutaneous means under skin, so these needles don’t even really go as deep as vaccination shots. Because it’s into the skin, the fluid makes some liquid bump under skin by the time the 30 min infusion is done, and over a few hours that fluid gets absorbed and the bump goes away. So far, she’s handling it well. There is some discomfort at site of injection that goes away a few hours later. She says that “brain freeze” (when your head hurts from too much ice cream) hurts more than the needles. 😂 And she doesn’t react to the adhesive! 👏

Overall it’s going well. She’s a bit scared of giving herself infusions, but she’s MORE scared of ME giving her infusion 😂 So she’ll be doing these infusions on her own under my supervision twice a week at home . We return to Iowa about once a month for checkups, like collecting vitals (Heart rate, blood pressure, temperature), collecting urine, blood tests, as part of the trial process.

We are blessed with a new beginning. We are also celebrating Easter, which is about Jesus and His new life. How appropriate. And so it begins, both a new chance medically and a reminder to also refresh spiritually.