Rivastigmine--Alzheimer's drug

We're headed off to Baltimore in the morning. Chris is joining Hannah and me. He's going to take a look at Johns Hopkins, while Johns Hopkins is taking a look at Hannah.

A few weeks ago we saw an email about a new research study focusing on an Alzheimer's medication and it's efficacy in the Down syndrome population.   This caught my interest.  I've always been leery of the Alzheimer's problem.   I remember a decade ago when I first heard a research study had found amyloid plaques in EVERY Down syndrome brain they autopsied.  I don't know how many they autopsied--but I remember that the individuals who died from things other than Alzheimer's Disease (AD).   And they found these plaques from age 9 months to 40 years old.   Nine months old!   That totally freaked me out. The plaques started so early!  Clearly the 21st chromosome has an impact on neural plaques.

But, compartmentalizing my fear, I decided that by the time Hannah was endangered from AD, the medical establishment would have solved the problem.   Science knew that AD was caused by these plaques.  They knew that the DS population would be perfect to study--there would be lots of pressure from the aging senior citizens to solve AD, so there would be a big push for research, and our kids would be the beneficiaries.

Well, the cogs move slowly.    There are some AD drugs out there, that show some promise in the general Alzheimer's population.   In 2006 Duke University (and one of my favorite researchers Dr. Priya Kishnani) looked at the safety and efficacy of one such drug in adolescents with Down syndrome.  There were only 11 subjects.  But all of them showed statistically significant improvements in adaptive function, attention, memory and language.  The researchers recommended that more studies were necessary--larger, controlled studies.

It took four years since that study was published before someone picked it up and studied it again.  I know that the current study was in the works for quite a while, but it's finally coming to fruition.   And I'm SO EXCITED that Hannah gets to be a part of the solution!

Dr. George Capone, at Kennedy Krieger is leading the study.  I first heard him years ago at an NDSS conference where he was presenting as a member of the Clinical Advisory Board (Dr. Kishnani and Dr. Skotko are also on the Board, or, were..I dunno who's there now!).

This will be a double-blind study, where half the subjects will get the medication and the other half will get a placebo. There will be cognitive and medical testing three times: baseline, interim and final testing. Twenty weeks of treatment.  Forty subjects. That's still small.  But it's a start.

Hannah will have to discontinue some of her vitamin/herb protocol.   That made me happy.  It means that, even if there are no studies that show these nutritional interventions are effective, clearly Dr. Capone thinks that something is working there, or he wouldn't need them removed.    I'm already missing Hannah's ginkgo...piano practice is markedly more challenging--for her, and for my patience.

I'm going to spend the next 6 months assuming that Hannah will be getting the placebo.  We won't know.  They won't know.  After it's all done, they'll open the records and see what the results show.

Friday is the screening visit.  She'll have medical testing--bloodwork, EKG, pregnancy test (WHAT?? Yes. All women of child-bearing potential are required to have a pregnancy test.  It took me a while to swallow that.  But it is what it is:  Research.  Controlled Research.)   Hannah won't know that it's a pregnancy test.  She just wouldn't understand that at all.

We go back in 2-4 weeks for the Baseline Visit where there will be more cognitive testing.  We'll receive the medication.   10 weeks later, we go back form more medical monitoring, and 10 weeks after that we'll be done--testing testing testing.

I had our pediatrician look over the study proposal before we made our decision. She talked at length with Dr Capone. Our doctor was very interested in the study. She feels that there is very low risk, but a significant possibility of positive outcomes for Hannah (if she gets the medication). Rivastigmine works on cholinergic deficiency (remember that study about pregnant moms who took choline? The babies with Ds had less delay than those whose mothers did not supplement choline while pregnant. Interesting.).

Yes, I'd love for Hannah to have cognitive gains. But more than that, I'd love for the research to get underway and solve the Alzheimer's problem for all of our loved ones--be they aging parents or individuals with Down syndrome.
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Follow up post can be read here.