We traveled this past week to Houston to have our final visit for the two year clinical trial that Nathan has been participating in. It was a very quick visit as we flew in on a Thursday and came home Saturday. We were able to fit in an amazing BBQ dinner and Jay got to enjoy a Seafood dinner.
Nathan was 1 of 6 patients in the Phase I/II of the trial. Phase III would have opened it up to the patients under 17. Phase III has been put on an indefinite hold due to a funding issue.
What we found out at our visit was that the Pharmaceutical company gave their medication “stock” to the Principal Investigator. It will be dispersed between the 6 patients. When I asked how long the medication will last, we were told it was enough for 1 year for each patient. This was a little disheartening as we have been told that we would have been given medication for the duration of the Phase III Trial which at minimum would have been at least 2 years. We also found out that we have to return in 6 months to Texas to obtain more medication (we left with a 6 month supply). I really thought we were done with traveling for the trial since we had moved to the Expanded Access arm
of the trial.
I stand firm in my belief that Nathan and 5 other patients did a phenomenal thing for all the other kids to follow in the trial. They tested this drug for safety. It had not been tried before. We went through all that comes with traveling to the NIH in Maryland every six months. We had people in our homes, he had numerous blood draws, he had to change his diet. We had to log every single dose of his medication. It was not easy. I believe that these 6 patients should have first access to the medication. They have been on it and for them not to have access to it could be very detrimental.
So what all of this means is that we have access to Miglustat for 1 year. At that time we have to petition our insurance for off label use. Keep in mind this medication costs roughly $22,000 a month. Trying to get an insurance company to pay for a medication that is not FDA approved will be challenging. We have been told that the Principal Investigator will fight for and with us. He has been successful with other patients. I will say, the idea of fighting makes me sick. I have fought every other entity on behalf of Nathan and will continue to do so. I just do not want there to be any lapse in the medication and approval can take awhile. Timing will be critical.
While we have time, I ask that you pray that we will be able to get insurance to cover this with ease. We do believe that this medication has provided a plateau effect. My fear is that without the medication he will “fall off the cliff” so to speak.
This has been a long two years of unknowns and firsts, but as always Nathan is amazing! He inspires me and is truly my hero. He will never understand how much he has taught me. If only the rest of us could see the world the way Nathan does without even being able to see what a sight that would be 💜.
Nathan's Batten Battle 
Praying that his medication is covered by insurance.