MPS II Intrathecal Trial on Hold

Posted Wednesday June 15, 2011 by Melissa

Taking Time

UPDATE: As of 7/1/11, the suspension on enrollment in the intrathecal trial was lifted by the UNC Institutional Review Board.


A clinical trial is never a place to get too comfortable.

I had planned to blog today about some of the fun iPad apps that Case and some of the other boys in the clinical trial are using…. But we had some upsetting news today that has circulated quickly around the MPS community, so I don’t feel like this post is letting the cat out of the proverbial bag. I felt like I couldn’t let this pass without posting and being true to the heartache we are feeling, both for our children and those of our friends.

If you’ve been following Case’s and some of the other boys’ journeys in this MPS II intrathecal clinical trial, you’ll know that there have been some “problems” with the intrathecal port-a-cath. Not to get too detailed, but several children in this and in the MPS III trial where they are using the same device have had complications with the port, requiring replacement of the device, catheter, or both. Some repeated surgeries are not a big deal, but these are neurosurgeries, putting a catheter into the spinal fluid, so it is definitely not something to be taken lightly.

But, MPS is not something for the faint of heart either.

No one who signed up for this trial thought it would be smooth sailing onto a nice, normal life for our children. But if a parent were asked whether they would remotely consider removing their child from the trial due to these complications, I think we would all look at you not only as if you had a third eye, but as if you had wings, a tail, scales, and just flew in here from the planet Areyouabsolutelyandutterlybonkers?!?!

But for those who don’t necessarily understand this perspective and who may only see ports breaking over and over, they are concerned over safety. It is in that vein that the UNC Institutional Review Board has, for now, suspended new enrollment into the clinical trial. The boys already involved (such as Case) can continue, but no new boys can be added unless the suspension is lifted or some other solution is in place.

This is both heartbreaking for our friends who are eagerly anticipating participation in the trial, even knowing all of the risks and port issues, and concerning because there is a possibility, however slight, that this could lead to more drastic action such as halting the trial altogether. If you don’t understand the implications of that, let  me be clear that the incredible gains you’ve seen or read about in Case are only expected to continue for as long as he receives this intrathecal medication. If it were to stop, the disease would progress as it was expected to before – absent a cure or another treatment, a progressive and degenerative disease leading to an early death. Don’t mean to be cold in those words, but that is the reality.

So we ask for your prayers:

  • that the IRB would understand that the nature of a rare disease trial such as this is risk versus reward and the reward to our boys is potentially (and has shown itself to be) amazing,
  • for comfort and peace for those families now in limbo awaiting the next steps in the trial,
  • for peace for the families currently involved and continued safety and improvement in our boys.

I hope to have good news in the coming weeks that the suspension has been lifted – a decision that I think would be applauded by all affected and concerned.

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One Response to “MPS II Intrathecal Trial on Hold”

  1. Having a LP shunt that is placed in exactly the same fashion as the IT trial Ports but for hydrocephalus (drainage of CSF from the brain/spine into the peritoneal cavity) I understand the risks of these repeated surgeries all to well – the trauma they place on the spinal cord itself (scarring, compression, need for spinal cord surgery atleast in my case due to repeated shunt surgeries/replacements). BUT I also 100% understand the benefits of this trial and while im not a parent so I cant understand the heartache of the ‘not knowing’ right now I can understand what IT clinical trials mean – my prayers will be with each of the families going through this for cont’d well being and gains and for the families waiting I will pray for comfort and patience in an incrdibly diffcult time.

    Many prayers,