Clinical Trials
There is a promising treatment for genetic mutations like mine, called an Antisense Oligo Nucleotide (ASO) . It's a piece of artificial DNA that suppresses the adverse action of a mutated gene.
Two firms in the USA (Ionis and Biogen) have produced an ASO that is designed to tackle the C9orf72 mutation that is the cause of my MND. There are early clinical trials running all over the world, with three centres in the UK: Sheffield, King's College and UCL.
With support from the MND Unit at the John Radcliffe, I have been angling to get on one of these trials. I was referred to Kings College, but heard nothing, then recently to the National Hospital for Neurology, part of UCL.
Yesterday I received a phone call from the lead investigator for the trial at the National Hospital for Neurology. He would like me to join Cohort 4 in their Phase 1 trial, subject to being assessed against their entry criteria.
They are running 6 cohorts with 8 people in each. The purpose of the Phase 1 trial is to assess people's tolerance for different doses, which are injected into the spinal fluid. Cohort 1 gets 10 mg, which is so low as to be equivalent to no drug at all. Each successive cohort gets an increased dose. They have to run in series, so Cohort 4 won't start until the new year.
A proportion of each cohort, maybe a quarter, will get a placebo rather than the drug. Neither patient nor doctor will know who gets which.
I'm invited to visit them in central London for an initial informal meeting in the next few weeks, which I will do. I'll find out a lot more then.
Two firms in the USA (Ionis and Biogen) have produced an ASO that is designed to tackle the C9orf72 mutation that is the cause of my MND. There are early clinical trials running all over the world, with three centres in the UK: Sheffield, King's College and UCL.
With support from the MND Unit at the John Radcliffe, I have been angling to get on one of these trials. I was referred to Kings College, but heard nothing, then recently to the National Hospital for Neurology, part of UCL.
Yesterday I received a phone call from the lead investigator for the trial at the National Hospital for Neurology. He would like me to join Cohort 4 in their Phase 1 trial, subject to being assessed against their entry criteria.
They are running 6 cohorts with 8 people in each. The purpose of the Phase 1 trial is to assess people's tolerance for different doses, which are injected into the spinal fluid. Cohort 1 gets 10 mg, which is so low as to be equivalent to no drug at all. Each successive cohort gets an increased dose. They have to run in series, so Cohort 4 won't start until the new year.
A proportion of each cohort, maybe a quarter, will get a placebo rather than the drug. Neither patient nor doctor will know who gets which.
I'm invited to visit them in central London for an initial informal meeting in the next few weeks, which I will do. I'll find out a lot more then.
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