This is for all the Calebs

Posted Wednesday January 25, 2012 by Melissa

Faria family

Have you realized that not all children with Hunter Syndrome can receive treatment? In some countries, if their brain has been affected by the disease or when that effect is shown (as occurs in 75-90% of boys with Hunter Syndrome), they become ineligible to receive Elaprase. When it boils down to it, the blunt argument is that because the drug is so expensive, on a national health plan in such countries as Australia and some provinces in Canada (those are the ones I know of off hand), those funds are better spent elsewhere than on only physically improving the life of a child who will become progressively more mentally handicapped and eventually die anyway.

Caleb Faria

Michelle Faria with her son Caleb, who has Hunter syndrome. Photo credit to DENNIS MANKTELOW

I am not here to debate national health policy. I understand that my child’s drug is expensive. But so are so many other drugs that are not denied because someone has mental handicaps.

I have been following the plight of the Faria family in Australia whose son was diagnosed with Hunter Syndrome at around 18 months, at least 6 months earlier than Case was diagnosed. Caleb has repeatedly been denied access to funding for Elaprase because they could see the disease had affected his brain (just like Case) and he is now over 2 years old and still without treatment.

I have not found a way to be in contact with the family, but I could not sit idly by and watch this child suffer when we sit here in the United States not only with access to Elaprase, but to the life-saving intrathecal drug as well. Although I am only one, I had to at least try to make a difference.

Here is the rub. One might understand the policy reasons for denying Elaprase when there was no hope of saving the child’s brain and thus, his life. But there is.

So here is my attempt to make a difference – my e-mail to the Health Minister of Australia explaining the reasons why this situation is unique. If you’re so inclined, feel free to use portions of this for your own letters in situations like these affecting your child or others.

———————————-

To: Tanya.Plibersek.MP@aph.gov.au

Cc: sunbury@leadernewspapers.com.au

Re: Caleb Faria

————

Hon. Tanya Plibersek,

I am writing because of my deep concern for Caleb Faria and his fight against Hunter Syndrome. My son Case is 4 1/2 years old and also suffers from Hunter Syndrome, diagnosed at the age of two years old after already suffering some developmental delay. However, as you will read, his life and cognitive abilities are now potentially being saved. We hope that for Caleb Faria as well.

Faria family

Michelle and Biju Faria with son Caleb who has Hunter Syndrome.

However, unlike Caleb, because we live in the United States, my son was able to begin receiving idursulfase almost immediately. He changed from often-sick, slow-moving, falling, and delayed child to a laughing, singing, and running child within weeks. He continued to show vast improvement in both his physical health, behavior, and cognitive skills, including reaching the ability to say 9- word sentences after 6 months on idursulfase when prior to that, he could not put two words together.

Idursulfase makes an incredible difference to a child and his family and it also provides the bridging means to possibly save his life. You see, because my son was on idursulfase and was healthy, he was able to enroll in the groundbreaking clinical trial for the intrathecal idursulfase drug.

Your government does not approve idursulfase because it does not treat the brain disease. But by withholding it, you are PREVENTING the treatment of the child’s brain disease and that treatment is finally at hand.

My son has cognitively improved and stabilized on the intrathecal drug. Although the clinical trial is currently completing a Phase I/II, a Phase III trial in which Caleb Faria could be considered were he on idursulfase could possibly begin within a year’s time. But because he is not on idursulfase, Caleb Faria will be ineligible and possibly, too physically infirm, and thus, this is a devastating death sentence for him when finally, Hunter Syndrome no longer needs to be.

I have consistently documented my child’s improvement in the clinical trial on a website, www.savingcase.com and would be happy to answer any questions that you might have. Please reconsider providing idursulfase when it is the necessary predicate for a lifesaving drug that is at hand.

Sincerely and with deepest concern,

Melissa J. Hogan

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6 Responses to “This is for all the Calebs”

  1. I believe New Zealand also has these policies as their public heath insurance plan will not cover other lysosomal drugs like that for pompe. Very sad.

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  2. Hi Melissa,
    I met Michelle last year at an MPS conference. At the time I actually gave her the link to your blog, so there is a slight chance she is reading this. I had her email address, but I can’t seem to find it. But I may be able to get it through the Australian MPS society. Would you like me to try?
    BTW, how did you hear about her?

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    • Yes, I would love to be in contact. I looked for any contact info online but could not get a bead on them. I would love to find out if there is anything else we can all do to help. As far as how I found out, I have google alerts for all things rare disease, especially MPS. Thanks Sandi!

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  3. Hi Melissa, god does work in misterious ways! I met Sandy last year and here I am hopeful to talk with you I have read the article thank you from the bottom of our heart’s for helping, you are certainly God sent. We are on a roller coaster struggle to get Caleb on the treatment. I will stand beside you together we will pave the way for other struggling families. God Bless your kind heart

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  4. I like to be of some help.
    I live in Sunbury.
    Can please Michelle/Biju Faria get in touch with me.

    Thanks
    Dr.Simonne Jameson

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