So There’s This Drug

BMN 111.

I read about it briefly when I was scouring the internet in those first few months after Declan’s birth; googling ‘achondroplasia’, in search of any and all information I could get my hands on. I didn’t really dwell on it too much; it didn’t seem pertinent at the time. Back then, I just needed to know the ins and outs of the here and now.

It’s come up in the odd discussion in POLP groups that I’m a part of. Lots of people feel very strongly about this issue one way or the other.

BioMarin Pharmaceutical has produced BMN 111. What the heck is it? Well BMN111 is a possible treatment for children born with achondroplasia.

My first thought… “well, my child isn’t sick.”

Two years ago, when I first read about this drug, it was in its infancy of research. But yesterday? Yesterday BioMarin began Phase 2 of the study and dosed the first patient (a child with achondroplasia) with BMN111. All of a sudden it’s a lot more real.

My second thought… “I can’t imagine subjecting my young child to a new drug study.”

From the Press Release:

The Phase 2 study is an open-label, sequential cohort, dose-escalation study of BMN 111 in children who are 5-14 years old. The primary objective of this study is to assess the safety and tolerability of daily subcutaneous doses of BMN 111 administered for 6 months. The secondary objectives will include an evaluation of change in annualized growth velocity, changes in absolute growth parameters, changes in body proportions and other medically relevant and functional aspects of achondroplasia, such as sleep apnea and joint range of motion.

Some more thoughts (presuming this might, someday, be a possible cure or treatment for achondroplasia)…

What’s wrong with my happy, healthy, funny, smart, most amazing little 2 year old boy?

Why would I want to change him?

Does this insinuate that there’s something wrong with him?

Why wouldn’t I want to give him the opportunity for “an easier life”? (practically speaking, a chance at living without the accommodations that come along with being short statured in this average sized world).

It would be years before we knew the long terms effects of this drug.

I’ve met amazing and inspiring adult LPs. What’s ‘wrong’ with having him grow up just the way he is?

If I could erase the potential future pain and surgeries and other issues that come along with achondroplasia, why wouldn’t I?

A little all over the map? Yep, that’s me. I really don’t know what I would do, if given the opportunity. Declan is only (just) two years old. He has a lot of growing years ahead of him. What if this drug breezes through its trials and does exactly what they think it will do? I just don’t know. It’s different than (the also controversial procedure) limb lengthening. Limb lengthening is an option (an incredibly painful option that takes years to complete) when a child is older and can play a large role in the decision making.  I’m assuming BMN111 would be administered when the child is younger, leaving the decision making to the parents.

I’d love to hear your thoughts!

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Did I say someone turned 2? Oh yes he did!

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About Tammy

I am a single mother to 2 wonderful children, ages 1 and 5. My youngest was surprisingly born with a rare genetic condition, achondroplasia, which is a type of dwarfism. This blog is a glimpse into my crazy life as I wade through figuring out dwarfism and divorce while enjoying every second of my deficient domestic bliss.
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5 Responses to So There’s This Drug

  1. Pingback: A Scary Concept | The "M" Word

  2. I would be afraid to use it on my child. Like you said, there is nothing wrong. Who knows about the possible side effects…especially long term! I understand they have to test it some how, but it seems harsh to subject children who can’t agree/disagree with what their body is going to be put through.

  3. kerri says:

    We totally agree. We received a letter wanting us to participate in this study, after much fasting and praying we declined. There are just too many unknowns with drug testing and We didn’t want Madilynn to ever think we thought there was something wrong with her. Achondroplasia is part of her and we love her that way. When she’s older she can choose, as for now she is surrounded by loving role models, both AH and LP. Thanks for sharing.

  4. traceybecker says:

    I don’t have a LP child, so I cannot begin to imagine this particular situation. But I imagine it is similar to the internal struggles that so many families living with hearing impairment or deafness of any sort encounter when they consider the cochlear implants, or surgeries for those with any level of blindness… They don’t have diseases; they’re just different. I think the hardest parts would be that this drug is EXPERIMENTAL and also that the decision is being made FOR them, as children. I hope the drug ends up having no side effects for the children who are testing it…

    Your son is so adorable. I love two year olds!

  5. Nancy says:

    Hi. Our son Brandon turned two in May. He was diagnosed with achon. at one year. In regards to the limb lengthening, I was reading a book by Andrew Soloman and in it he says that if someone had asked him when he was a teen if he could take a pill to not be gay anymore would he, he would’ve in a heart beat because being different during the teen years is one of the hardest things to go thru. He said in the book how he wouldn’t change it for the world now as an adult.
    I don’t know how much a teen could help in the decision making process for limb lengthening. I feel like they would always choose to be as “normal” as possible. It’s with age that you learn to not give a crap what other people think.
    Your son is adorable and looks so much like our own. It’s hard finding shirts that fit well with large enough neck openings isn’t it??

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