Today after three-pronged efforts that included a few good folks from the HD family community, clinical investigators in the HART ACR16 clinical trial and the sponsoring drug company, Neurosearch is moving forward in its application to the FDA to provide drug to North American participants after they have completed the placebo controlled trial. It goes to show that the HD World can be moved -- if we all push together.
The Story as told by Jason from his Blog:
"It's been hard for my family to watch my father's decline since the end of the ACR16 trial (now named Huntexil). While my father is stoic, it is apparent even to people outside the family that his chorea has worsened without the drug. It often seems hard or even impossible for him to focus on a conversation now.
So it was very exciting for us to receive word from Maggie through the HDAC forums that NeuroSearch is making the drug available for "Compassionate Use" in Europe and the United States for persons that have gone through the trial. This basically means that anyone that has trialed the drug can continue to receive the drug until it is made available via prescription. It is up to the pharmaceutical company to decide whether to charge for it or not, but NeuroSearch is offering it free of charge to those qualified.
To get the details on the status of this availability here in the United States I phoned Denmark very early this morning and was forwarded to the Clinical Project Leader for the Huntexil trial. After calling me back an hour later I was pleased to find that she was very gracious; she spoke to me for several minutes regarding the status of Huntexil for Compassionate Use. I was told that NeuroSearch has never gone through the American process for getting a drug certified for Compassionate Use and that apparently there were multiple ways of getting it done; they were not yet sure of which was was best for this drug. It is unfortunate news that the process has not yet begun, but I was encouraged a bit by her saying that I was the first person to have contacted her from the United States and that it was significant to her and that she would redouble her efforts on getting the drug approved here in the USA."
This is Very Good News on Several Fronts:
First and foremost Huntington's families in Europe and in North America are reporting significant improvement during the trial. And just like what happened when the first drug of this type was given to a Huntington's person in 1999, symptoms return when the drug is stopped. Importantly this suggests that this drug may be working at a significant level.
The second part of this good news is that the drug company is responding to the needs of those good Huntington's families who have participated in this study. Though NeuroSearch has initiated FDA approval to provide drug to trial participants, it is not know when the program will actually begin. But it is exciting indeed to know that this process has begun. Know that this is an expensive service to provide.
The third part of what we hope will be more good news is that this will add incentive for others to sign up for this trial -- that if successful will get this drug to all Huntington's families.
We Can Move the HD World: Right now it is Huntington families who have the power to move this drug faster. If you can, join this clinical trial; and if you can't encourage someone else!
And I'm very happy to report that a new HSG site will be opening in my region at the Evergreen Neuroscience Center. Enrollment for ACR16 starts in Seattle area is just 2 weeks. Know I'll be pushing too.