I am unsure if my killjoy colleagues have clocked just how significant the FDA’s Tofersen ALS decision is for the future of neurodegenerative disease diagnosis and treatment.
I'm 60 and perhaps 10 years into PPMS, having also had Mononucleosis (Glandular Fever) at 19. I have a daughter of 27 and son of 19 who are showing no signs of MS, thankfully. Statistically they have approx. 2-4% probability of contracting MS themselves.
The question is, should I get them screened for RIS and where should I go for that?
I don't think screening is feasible or advisable at the present time unless as part of a research programme. Until we have licensed treatments for RIS all we can do is watch and wait, which simply cause anxiety. All they need to do is keep trim and fit, don't smoke and make sure they are vitamin D replete.
It can be so stressful when we look for symptoms. But how can we absolutely not when we just want to protect them. My young people are 35,33 and 22. I’ve advised them to do their best with lifestyle choices. My eldest son got a little freaked out when he tried to get life insurance and MS was excluded based on the age of my diagnosis and my sisters diagnosis. Ive just told them if they have any concerns we follow it up. No hanging about. If they develop it they start on the hardest hitting treatment. I’d sell and downsize my house if necessary for them, to pay if needs me.
I don't know how I would live with myself if I "give" my kids this disease. Others have and somehow come to terms with it but it must be impossibly hard. As you say, at least we will be vigilant and alert to any ominous signs. I was misdiagnosed and hence diagnosed late (EDSS6.0) but the kids would stand more of a chance of an early diagnosis.
As Gavin says, we MS-ers must the best versions of ourselves that we possibly can, and that applies to our kids too. My kids have accepted taking vitamin D supplements for life without complaint and that has been achieved with the lightest of touches. I won't start quoting % probabilities, that will be cruel and they have a right to live their own lives.
Ive never been given a score. Now I don’t want one as it makes no difference or offers me no help. Also diagnosed late as many of us older MSers have been. I’m hopeful that this is not so common with younger generations. So they can carry on working and feel a valued member of society. Not scrap heaped.
I hope these new meds for MND can help many people gain some years. Any hope of a med is better than no hope. I knew someone who died 6 months from diagnosis with MND. The cruelest of illnesses and horrid way to die.
All this sounds like exciting news and a more positive outlook for those with neuro degenerative disorders. Almost certainly too late for me but hope for my grandchildren.
“when you are symptomatic with Alzheimer’s or Parkinson’s, it is really too late to make much of a difference to the natural history of the disease”. The Michael J Fox foundation has funded $1 billion of research projects into Parkinson’s disease. No amount of research funding seems to make any real difference to the treatment of neurodegenerative diseases. My dad’s friend died of MND in 1985 (4 years after dx). His neighbour died of MND last year (4 years after dx). Nothing really changes.
I think we are at a watershed with MND and AZD. The equivalent of an interferon-beta in MS moment. Treatments will emerge, but early diagnosis and treatment will be essential. This is why presymptomatic diagnosis is so important.
Neurology is always jam tomorrow. How many more Michael J Foxes, Doddie Wiers, Jacqueline du Pres do we need to see before something is done about these devastating diseases? Perhaps these neurodegenerative diseases are just too difficult to crack.
Life is just information (coded information) and a diseased life is misinformation or disinformation. Therefore it is only a matter of time before technology catches up with the mis/disinformation and recodes it. Neurodegenerative diseases are tractable problems all we need is time.
My subscription was cancelled as my Credit cards were scammed. Can I pay without saving card details as I’m struggling cognitively to keep track of subscriptions and passwords.
Very disappointing that EBV was not discussed at the AAN meeting. How is a cure going to be found without discussing EBV? Just occurred to me why Prof G said testing on mice EAE models do not work as well as testing on pwMS. Cos they do not have EBV virus in them!
Approval of tofersen is three good news: treatment itself, new class of medicine becoming established treatment and NFL stuff.
Regarding RIS if payers won't pay, would it be feasible for neurologists to go for a course of rituximab off label? I would say that there is relevant chances that if a depleted therapy is given first line and timely could avoid future MS development or worsening
This didn't work for type 1 diabetes it only delayed the onset. But worth a try. I would prefer cladribine; it works like a small molecule ant-CD20 therapy, with CNS penetration.
The problem with extending the diagnosis of MS into RIS is improving the specificity of the diagnostic criteria to make sure you don't include MS mimics or other diseases. The reason for this is so if it comes to more aggressive treatments you don't use them inappropriately.
Very thought-provoking, Gavin.
I'm 60 and perhaps 10 years into PPMS, having also had Mononucleosis (Glandular Fever) at 19. I have a daughter of 27 and son of 19 who are showing no signs of MS, thankfully. Statistically they have approx. 2-4% probability of contracting MS themselves.
The question is, should I get them screened for RIS and where should I go for that?
I don't think screening is feasible or advisable at the present time unless as part of a research programme. Until we have licensed treatments for RIS all we can do is watch and wait, which simply cause anxiety. All they need to do is keep trim and fit, don't smoke and make sure they are vitamin D replete.
It can be so stressful when we look for symptoms. But how can we absolutely not when we just want to protect them. My young people are 35,33 and 22. I’ve advised them to do their best with lifestyle choices. My eldest son got a little freaked out when he tried to get life insurance and MS was excluded based on the age of my diagnosis and my sisters diagnosis. Ive just told them if they have any concerns we follow it up. No hanging about. If they develop it they start on the hardest hitting treatment. I’d sell and downsize my house if necessary for them, to pay if needs me.
I don't know how I would live with myself if I "give" my kids this disease. Others have and somehow come to terms with it but it must be impossibly hard. As you say, at least we will be vigilant and alert to any ominous signs. I was misdiagnosed and hence diagnosed late (EDSS6.0) but the kids would stand more of a chance of an early diagnosis.
As Gavin says, we MS-ers must the best versions of ourselves that we possibly can, and that applies to our kids too. My kids have accepted taking vitamin D supplements for life without complaint and that has been achieved with the lightest of touches. I won't start quoting % probabilities, that will be cruel and they have a right to live their own lives.
Ive never been given a score. Now I don’t want one as it makes no difference or offers me no help. Also diagnosed late as many of us older MSers have been. I’m hopeful that this is not so common with younger generations. So they can carry on working and feel a valued member of society. Not scrap heaped.
I hope these new meds for MND can help many people gain some years. Any hope of a med is better than no hope. I knew someone who died 6 months from diagnosis with MND. The cruelest of illnesses and horrid way to die.
All this sounds like exciting news and a more positive outlook for those with neuro degenerative disorders. Almost certainly too late for me but hope for my grandchildren.
“when you are symptomatic with Alzheimer’s or Parkinson’s, it is really too late to make much of a difference to the natural history of the disease”. The Michael J Fox foundation has funded $1 billion of research projects into Parkinson’s disease. No amount of research funding seems to make any real difference to the treatment of neurodegenerative diseases. My dad’s friend died of MND in 1985 (4 years after dx). His neighbour died of MND last year (4 years after dx). Nothing really changes.
I think we are at a watershed with MND and AZD. The equivalent of an interferon-beta in MS moment. Treatments will emerge, but early diagnosis and treatment will be essential. This is why presymptomatic diagnosis is so important.
Neurology is always jam tomorrow. How many more Michael J Foxes, Doddie Wiers, Jacqueline du Pres do we need to see before something is done about these devastating diseases? Perhaps these neurodegenerative diseases are just too difficult to crack.
Life is just information (coded information) and a diseased life is misinformation or disinformation. Therefore it is only a matter of time before technology catches up with the mis/disinformation and recodes it. Neurodegenerative diseases are tractable problems all we need is time.
Hi! Sorry for being off topic but im kind of desperate. I paid for subscription but im having problems with it. Who can i contact for help?
Ana, everything seems fine from my side. Drop me an email if it doesn't.
My subscription was cancelled as my Credit cards were scammed. Can I pay without saving card details as I’m struggling cognitively to keep track of subscriptions and passwords.
Very disappointing that EBV was not discussed at the AAN meeting. How is a cure going to be found without discussing EBV? Just occurred to me why Prof G said testing on mice EAE models do not work as well as testing on pwMS. Cos they do not have EBV virus in them!
Approval of tofersen is three good news: treatment itself, new class of medicine becoming established treatment and NFL stuff.
Regarding RIS if payers won't pay, would it be feasible for neurologists to go for a course of rituximab off label? I would say that there is relevant chances that if a depleted therapy is given first line and timely could avoid future MS development or worsening
Rituximab for how long? Lifelong? And where do you derive the idea that depletion therapy would lead to future MS development and or worsening?
This didn't work for type 1 diabetes it only delayed the onset. But worth a try. I would prefer cladribine; it works like a small molecule ant-CD20 therapy, with CNS penetration.
One single infusion. I say the opposite: it will avoid MS development and worsening
Ok so one blast of rituximab and they're good. Do you have any literature on this?
The problem with extending the diagnosis of MS into RIS is improving the specificity of the diagnostic criteria to make sure you don't include MS mimics or other diseases. The reason for this is so if it comes to more aggressive treatments you don't use them inappropriately.