Only one consultation today, with Dr. Whitley, a geneticist at the University of Minnesota. Not much to say here, but he did offer an alternative to the bone marrow transplant that we are here to investigate, and that is what he and his team are calling AIM therapy, which involves using a cocktail of previously FDA-approved drugs for a different purpose, in this case (experimentally) treating GM1. Some of the research has pointed to what Armand has being something comperable to inflammation, like a bee sting — the body then sends enzymes and specialized cells to fight infection. In GM1, they’ve found that similar cells in the brain (and possibly in the liver, spleen, and bones) are always active and on alert, constantly attacking. This is what partially causes expansion of the liver and spleen, and generally bone issues in severe GM1 cases. By treating this as an inflammation case, along with another drug that inhibits development of a gangliocide precourser, they may be able to slow down progression as well as stave off neurological damage for a time. This is highly experimental, they’ve only tried it on a few cases with some promising (very very) preliminary results. One big hurdle is that one of the drugs involved is very expensive and is only approved by insurance for a particular disorder — getting approval for GM1 from Lindy’s UHC may-or-may-not be an issue. If we don’t get appoved by insurance, the cost could run around $30-40,000 just for this one medicine. Other medicines, including for instance Ibuprofen, won’t be an issue.
What we’re faced with now is the idea that his bone marrow transplant, if we do it, may-or-may-not do what we need, which is provide stem cells to release the enzyme he needs across the blood-brain barrier, helping prevent neurological damage; a second option that, as Dan Patrick says, “sounds good in theory, but find me Theory on a map”, and may-or-may-not even be approved, much less work; or just go the Christian Science route and pray that God may-or-may-not have some mercy on the Fat Man. We also may-or-may-not be able to do both the bone marrow transplant, and follow that with the drug therapy a few months after that once he’s successfully grafted (had his new bone marrow grow and his immune system ‘reboot’).
Lindy is troubled by the news of limited possibilities, and for the first time cried on my shoulder. She’s also, I think, disappointed we may-or-may-not get to go to the Mall of America, because it’s about an hour bus/train ride from where we are staying. Armand, on the other hand, discovered some toys here to play with, including a girl’s baby stroller. According to Lindy, he may-or-may-not get one for his birthday. In this case, I’m hoping to be Mayor of May Not.