August 13, 2020 at 9:09 pm #16124AmandaKeymaster
Have you volunteered for clinical trials or any kind of ALS research? What research program are you involved with? How did you find the program? What did you consider before making a decision to participate?
June 16, 2021 at 7:43 am #19550
Hi Amanda, I’m about to hear whether I’m accepted onto the first-in-human long-term (40 weeks) phase 1 trial of Biogen BIIB078 genetic therapy. It starts in the next few weeks.
On 11 June 2021 I was on holiday in a remote cottage over-looking the Isle of Iona. BBC Radio 4 morning news was on and I heard an interview with Prof Dame Pamela Shaw talking about her team’s research into exercise and ALS progression. I emailed her that morning about my ALS progression and history of extreme exercise, and my mother who died aged 49 with MND, a former sports teacher and athlete . I also mentioned my son, who carries the c9orf72 gene mutation that I carry, and is an ultra-fit runner about to run 49 peaks over 3,000 ft in seven days to raise funds for ALS research.
She replied the next day (a Saturday) offering to see us both in her clinic. She then went on to invite me onto the clinical trial and giving links to the referral process. My consultant has now agreed to refer me.
I ask why it required a chance email to a busy academic, on the day media outlets were full of the news about exercise and ALS, to learn about the trial. What was my consultant doing? What does he read? He told me several months ago he had referred me to the Sheffield Institute of Translational Neuroscience (SITraNS), of which Prof Shaw is director. This is news to her.
I considered various issues before deciding to enrol: transport (4 hrs each way), pain of repeat lumbar puncture, 1:3 chance of placebo, stopping TUDCA & Metformin which seem to have slowed progression (the former being trialled at SITraNS), my normal life (I chair a monthly council meeting, and am vice-chair of a folk festival).
But decided to go ahead on the balance of probabilities and knowing pain is usually bearable. In fact my two sons and my wife now play back to me my frequent ‘no pain; no gain’. Both sons and my wife run, cycle, climb mountains, cycle up steep hills and recall my ‘encouragement’ over the years.
I’m waiting for email confirmation and a start date.
June 16, 2021 at 8:19 am #19551AmandaKeymaster
David that is really exciting and serendipitous. It makes no sense that your doctor(s) didn’t know about the trials and refer you. I thought there was a collaborative project where all ALS research and trials was being shared with people in the field. I’ve heard GREAT things about toferson and it’s past trials. I believe, correct me if im wrong, that after the treatments you will be eligible for ongoing treatments of toferson regardless if you received the placebo or not. I hope you get the drug, and I hope it continues to be promising. Please keep us posted on how you are doing and what you think.
The lumbar puncture frightens me. I am supposed to get one done this summer for the research study. I’ve heard they aren’t bad at all, uncomfortable for about 15 minutes.
David, thank you for participating. I hope it benefits you and your health. Also please know that you are helping all pALS and you are appreciated!!!
June 16, 2021 at 12:00 pm #19552Jean-Pierre Le RouzicParticipant
> I believe, correct me if im wrong, that after the treatments you will be eligible for ongoing treatments of toferson regardless if you received the placebo or not
Amanda, you mention “TofersEn” also known as IONIS-SOD1Rx and BIIB067.
Which is for SOD1 mutation, I think that you have that mutation in your family.
David discuss of BIIB078 which is for C9orf72 dipeptide repeats.
While both BIIB078 and BIIB067 are developed in a collaboration between Biogen and Ionis Pharmaceuticals, they are two different, non interchangeable therapies.
June 18, 2021 at 8:47 am #19565
Ah, but Tofersen is the SOD1 genetic therapy. I have C9orf72 mediated fALS, BIIB078 rather than BIIB077.
But if I am in the placebo group I should still be offered the active treatment if it proves effective.
Still no confirmation I’m accepted onto the trial. Grrr.
June 18, 2021 at 11:35 am #19566Jean-Pierre Le RouzicParticipant
> BIIB078 rather than BIIB077.
David, you probably mean BIIB078 rather than BIIB067.
Biogen has 4 therapies:
– BIIB067 (tofersen), the one that Amanda had in mind.
– BIIB078 (IONIS-C9Rx), the one you are discussing David.
– BIIB100 (XPO1 inhibitor), one which may be used against TDP-43 proteopathies, so probably it will be useful to most pALS.
– BIIB105 (ATXN2 ASO) a mediator of TDP-43, so again a therapy that could help most pALS.
TDP-43 therapies could be also useful in subsets of Alzheimer and Parkinson diseases.
June 30, 2021 at 5:41 am #19724
Back from meeting the prof. Gets better: she wants me to participate in the 1b/2a trial of Wave Life Science’s WVE 004. I need to update my SVC, but I meet all criteria. I’m apparently the first person to be referred for this trial.
All this because my wife was listening to BBC Radio4 as we lay in bed.
And more serendipity: Prof Shaw lived in the flat below my sister and was in the same year at medical school as my brother-in-law. We share many acquaintances.
July 1, 2021 at 3:21 pm #19741BillParticipant
I’ve been in two drug trials. Neither reached desired endpoints, but I was glad to assist. I also was in non drug studies. One looking for bio markers for ALS and tracking my progression. That one lost its funding. The other were genetic studies searching for mutations and familial ALS. Unfortunately they did not locate the specific mutation in my family. Other of my siblings are being added to continue the search as it appears we are a rare case of multiple different rare MNDs.
My drug trials were a direct result of going to a university ALS that does drug trials. The other studies I found by researching sites such as clinical trials.gov.
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