Response from Xenon re Xen402

General Blog
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Hi Folks,

I have had a reply from Dr Paul Goldberg at Xenon concerning a question I asked about clinical trials, he sent me a very comprehensive reply around care and management for our Daughter. I thought this was extremely decent of him , here is the paragraph concerning trials etc. Apologies if this is information you may already have.

Dear David

I am sorry to hear about the terribly difficult situation with your daughter. I have had a lot of interactions with patients with erythromelalgia over the past several years and have learnt a lot about this condition. I am keen to help you where possible. Unfortunately, the drug is still an experimental drug and thus we are legally unable to provide it to Emily at this time. It is not yet approved and can only be used under very strict regulatory conditions in a clinical trial setting. The next clinical trial is at least a year away and so this option will not be of help in the near term. Furthermore, unfortunately, the drug has not yet been tested in the paediatric setting and thus Emily at this time would not be eligible for the upcoming trial.

I do want to leave you with a positive note. I know the situation looks bleak at the moment and that Emily is suffering badly. However, with persistence and repeated evaluation of the wide variety of existing medications, many patients do find some measure of relief and some achieve dramatic improvements. Furthermore, the drug we are developing is targeted at the root cause of the disease (ie a defective sodium channel) and we are very encouraged that this mode of intervention will prove highly beneficial for patients with erythromelalgia.

With kind regards

Paul Goldberg

So the next clinical trial is a year away and this was the address I sent the query to research@xenon-pharma.com. It may be worthwhile contacting Xenon to see if you are eligible for the trial. For the moment Emily has a heart ultrasound in the morning, all going well she will be having a Licocaine infusion followed up by oral mexilitine hopefully within the next few weeks.

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Thanks David for posting the Xenon letter. So sorry about your daughter. Hope all will go well with her procedures.

I was eligible for Xenon's trial and have sent in my saliva sample. Just waiting for an answer. Xenon said it could be up to 12 weeks and the answer is sent to my primary care doctor. I will post when I get my answer.

Pat in NH

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I have heard about the trials but still find it interesting to hear from this doctor - thank you Davi. But I do have a question and maybe you can answer it - how is it that a defective sodium channel is the root cause of the disease? I have never heard that before. I thought that was the cause for the increased sensation of pain and the cause of EM is the overdilation of blood vessels. Or is it that this overdilation is caused by defective sodium channels?

Hope you can find meds to relief your daughters suffering.

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That was a pretty awesome response. It's nice to know that there are doctors out there who actually care. I think too many of us experience a few bad ones. Thanks for posting. :)

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I received a DNA sampling kit from Xenon. I am sending it back to the BC office tomorrow. See what happens from there.

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Dearest David, Thanks for posting Xenon reply. ... that was so very kind of you to share information with us. Just to say that I and a few others are seeing positive changes on the sodium channel blocker therapy ie: lidocaine followed by mexiletine. Changes are subtle and doses need to be tritated slowly over months. At 3 months now I am really hopeful- its a blessing.

You and Emily are in my heart, thoughts and prayers.

Please let me know how things go

God bless

mads x