Aa
Why no treatments for PPMS sufferers
Hi all
I suppose as my symptoms worsen, a bit of desperation sets in
Can anyone tell me why there are no treatments recommended for the PPMSers out there
I know we only make up 10% of the total of MS sufferers but surely that is a large enough group that would benefit from a drug trial
Or am I ignorant of the fact that there have been trials
Oh well back to my wobbly world
John
I suppose as my symptoms worsen, a bit of desperation sets in
Can anyone tell me why there are no treatments recommended for the PPMSers out there
I know we only make up 10% of the total of MS sufferers but surely that is a large enough group that would benefit from a drug trial
Or am I ignorant of the fact that there have been trials
Oh well back to my wobbly world
John
Primary progressive multiple sclerosis : current and future treatment options.
Leary SM, Thompson AJ.
Institute of Neurology, University College London, Queen Sqaure, London, UK.
Approximately 10% of patients with multiple sclerosis (MS) run a primary progressive course characterised by an accumulation of neurological deficits without relapse or remission. Designing therapeutic trials in primary progressive MS (PPMS) has presented several problems. Patient recruitment may be difficult because of the relative rarity of PPMS and historically has been hindered by the lack of specific diagnostic criteria. There has been a limited choice of validated outcome measures, although, in recent studies, the MS functional composite measure and magnetic resonance imaging measures of lesion load and atrophy have been widely used. Despite these problems, several trials have been designed specifically for PPMS, including exploratory randomised controlled trials of interferon-beta-1a and interferon-beta-1b and mitoxantrone, a phase III trial of glatiramer acetate, and an open-label study of riluzole. Patients with PPMS have also been included in randomised controlled trials of azathioprine, methotrexate, cladribine, intravenous immunoglobulin and cyclophosphamide, and open-label studies of haematopoietic stem cell transplantation and pirfenidone in progressive MS. However, no treatment has been proven definitively to modify the course of the disease. Looking to the future, therapeutic agents should aim to target the underlying pathogenic mechanisms in PPMS. As a result of the relative lack of inflammation in PPMS, neuroprotective agents that target neuronal loss directly, rather than inflammation, may be more worthwhile. However, further investigation into the pathogenic mechanisms in PPMS is required to guide the development of future therapeutic agents.
Leary SM, Thompson AJ.
Institute of Neurology, University College London, Queen Sqaure, London, UK.
Approximately 10% of patients with multiple sclerosis (MS) run a primary progressive course characterised by an accumulation of neurological deficits without relapse or remission. Designing therapeutic trials in primary progressive MS (PPMS) has presented several problems. Patient recruitment may be difficult because of the relative rarity of PPMS and historically has been hindered by the lack of specific diagnostic criteria. There has been a limited choice of validated outcome measures, although, in recent studies, the MS functional composite measure and magnetic resonance imaging measures of lesion load and atrophy have been widely used. Despite these problems, several trials have been designed specifically for PPMS, including exploratory randomised controlled trials of interferon-beta-1a and interferon-beta-1b and mitoxantrone, a phase III trial of glatiramer acetate, and an open-label study of riluzole. Patients with PPMS have also been included in randomised controlled trials of azathioprine, methotrexate, cladribine, intravenous immunoglobulin and cyclophosphamide, and open-label studies of haematopoietic stem cell transplantation and pirfenidone in progressive MS. However, no treatment has been proven definitively to modify the course of the disease. Looking to the future, therapeutic agents should aim to target the underlying pathogenic mechanisms in PPMS. As a result of the relative lack of inflammation in PPMS, neuroprotective agents that target neuronal loss directly, rather than inflammation, may be more worthwhile. However, further investigation into the pathogenic mechanisms in PPMS is required to guide the development of future therapeutic agents.
Sorry, I put that "particularly Alex" part because I thought she had started this thread. But then I just noticed ozjohn was the original poster. I didn't want to give the impression I was ignoring anyone.
You rang?
Oh boy - I don't want to give people the impression that I'm an expert on this.
But, currently Hopkins is only using HiCy for RRMS. In fact, I think their inclusion criteria is a little more strict than most, in that they require Gd-enhancing lesions seen on MRI.
I'm not sure it was ever tested for PPMS - I'd have to go back and do some digging. One of the original trials which was actually done at Stonybrook (before Hopkins became invloved with HiCy) included some SPMS patients. But, again, I'd have to go back and do some reading to find the results for the progressive patients.
Recently, I was looking through the trials at the .gov website, and this one struck me. Folks, particularly Alex might be interested:
http://www.clinicaltrials.gov/ct2/show/NCT00950248?term=multiple+sclerosis&recr=Open&lup_s=09%2F01%2F2009&lup_e=01%2F09%2F2010&rank=3
(You'll have to cut and paste into your browser.)
It talks about a drug called Idebenone, which is being studied for PPMS. One really interesting thing, is that it says this drug is similar to coenzyme Q10.
Oh boy - I don't want to give people the impression that I'm an expert on this.
But, currently Hopkins is only using HiCy for RRMS. In fact, I think their inclusion criteria is a little more strict than most, in that they require Gd-enhancing lesions seen on MRI.
I'm not sure it was ever tested for PPMS - I'd have to go back and do some digging. One of the original trials which was actually done at Stonybrook (before Hopkins became invloved with HiCy) included some SPMS patients. But, again, I'd have to go back and do some reading to find the results for the progressive patients.
Recently, I was looking through the trials at the .gov website, and this one struck me. Folks, particularly Alex might be interested:
http://www.clinicaltrials.gov/ct2/show/NCT00950248?term=multiple+sclerosis&recr=Open&lup_s=09%2F01%2F2009&lup_e=01%2F09%2F2010&rank=3
(You'll have to cut and paste into your browser.)
It talks about a drug called Idebenone, which is being studied for PPMS. One really interesting thing, is that it says this drug is similar to coenzyme Q10.
If you look back for decades over the history of MS research, one thing has been stable. No therapy has been shown to stop the progression of PPMS. All the DMDs have been studied.
Believe me there have been a lot of studies over the years.
I think that there has been some progress with the radical treatments like HiCy - High dose Cyclophosphamide with it's complete destruction of the immune system and near complete ablation of the bone marrow. This is three days of the chemo drug and all blood counts go to zero for a brief time, but they let the few remaining cells of the immune system get ahold and repopulate. It takes red cell transfusions, platelet transfusions and careful control against infection, but it is showing promise at Johns Hopkins. The procedure has potentially fatal side effects, but no deaths reported as of yet with a dozen or two people tested.
I think the HiCy procedure has been tested with PPMS and very aggressive RRMS.
Patientx ?
Quix
Believe me there have been a lot of studies over the years.
I think that there has been some progress with the radical treatments like HiCy - High dose Cyclophosphamide with it's complete destruction of the immune system and near complete ablation of the bone marrow. This is three days of the chemo drug and all blood counts go to zero for a brief time, but they let the few remaining cells of the immune system get ahold and repopulate. It takes red cell transfusions, platelet transfusions and careful control against infection, but it is showing promise at Johns Hopkins. The procedure has potentially fatal side effects, but no deaths reported as of yet with a dozen or two people tested.
I think the HiCy procedure has been tested with PPMS and very aggressive RRMS.
Patientx ?
Quix
There is a trial that is nearing completion and the doctor conducting it believes that it will yield results that lead to a drug being using for PPMS, probably next summer? There is hope, it's not over, people are working on it, so hang in there.
Bio
Bio
Alex is a quick study - her info on PPMS sure makes sense to me.
If you go to
clinicaltrials.gov
and put in primary progressive multiple sclerosis,
you will return a list of studies being done on PPMS. It is a very short list, but nonetheless a list.
be well,
Lulu
If you go to
clinicaltrials.gov
and put in primary progressive multiple sclerosis,
you will return a list of studies being done on PPMS. It is a very short list, but nonetheless a list.
be well,
Lulu
That is fantastic
I truly hope it works or at least slows down the progression
I will be thinking of you
John
I truly hope it works or at least slows down the progression
I will be thinking of you
John
John,
Just got an e-mail from my PCP she wants to try a statin for MS. A Dr. she knows was in a study. I have heard mixed things. I may try it and ldn. Who knows. I at least need hope.
Just got an e-mail from my PCP she wants to try a statin for MS. A Dr. she knows was in a study. I have heard mixed things. I may try it and ldn. Who knows. I at least need hope.
Hi John.
First of all there are no FDA approved treatments for PPMS. There is a trial coming up at Duke for PPMS drug. The DMDs usually have no effect on PPMS so the Insurance Companies use the fact they are not FDA approved for PPMS as a reaon to not pay for them.
RRMS is mostly a disease of inflammation where as PPMS is one of degeneration. In a nutshell it is hard to measure changes in PPMS so trials would take years longer than RRMS so researchers tend to go for RRMS. They measure the relapses in RRMS. Also there are more people with RRMS 85% so the drug companies make more money on RRMS drugs. RRMS drugs are some of the most lucrative drugs in history. Teva hopes to double its profits in five years and that is mostly because of Copaxone.
Keep in mind every case of PPMS is different. Hang in there,
Alex
First of all there are no FDA approved treatments for PPMS. There is a trial coming up at Duke for PPMS drug. The DMDs usually have no effect on PPMS so the Insurance Companies use the fact they are not FDA approved for PPMS as a reaon to not pay for them.
RRMS is mostly a disease of inflammation where as PPMS is one of degeneration. In a nutshell it is hard to measure changes in PPMS so trials would take years longer than RRMS so researchers tend to go for RRMS. They measure the relapses in RRMS. Also there are more people with RRMS 85% so the drug companies make more money on RRMS drugs. RRMS drugs are some of the most lucrative drugs in history. Teva hopes to double its profits in five years and that is mostly because of Copaxone.
Keep in mind every case of PPMS is different. Hang in there,
Alex
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