April 3 Unrelated blogger comments

April Comments from the Unrelated blogger link have been moved here


30 comments:

Anonymous said...
Prof. Giovanonni,

I would like to know at what point does a patient with severe symptoms of PPMS get admitted to hospital for treatment or recovery in the UK? I am familiar with a case where there might be a danger of irreparable axonal loss as treatment is being delayed (for reasons unknown to me). This patient is rapidly deteriorating with little hope of quick help. I am asking this because being German and benefiting from the German health system, Doctors in my country have asked me why treatment for this patient is being delayed. According to the little research I have done, it seems to be vital to stop axonal loss. I am looking forward to your views on this.
Gavin Giovannoni said...
Re: "I would like to know at what point does a patient with severe symptoms of PPMS get admitted to hospital for treatment or recovery in the UK?"

We rarely admit MSers to hospital in the modern era. Hospital are dangerous places, particularly in the era of severe and sometimes untreatable hospital acquired infections. The exceptions being; (1) severe and disabling relapses and the MSer is not coping at home, they would then come in for steroids and to be assessed by the rehab team; (2) severe infections; (3) to have pressure sores managed; (4) bladder and bowel surgery; (5) insertion of a balcofen pump; (6) day-case for starting fingolimod and natalizumab infusions.

Apart from these obvious exceptions MS is almost exclusively an outpatient disease. I am aware that in Germany hospitals and neurologists often only get reimbursed for inpatient care, which is why a lot of MSers get admitted in Germany. The latter is due to the perverse incentive created by reimbursement. In the NHS these incentives are minimised.

Unfortunately, we don't have any DMTs licensed for PPMS; if only we have. All we can really use with any confidence is best supportive care. We have some evidence that in young PPMSers and in those with active MRI scans that they may benefit from anti-CD20 therapy. As this is expensive and unlicensed we would have to make a special request to the MSers PCT (primary care trust) to pay for the drug.
Anonymous said...
Many thanks for your very quick reply, Prof. Giovannoni. I agree,hospitals are a very bad place to be these days. No doubt, there are perverse elements in every health system, be it in Germany, France or the UK. But I was not referring to abuse but was rather interested to see what the chances/treatments/offers are for patients with severe symptomps of PPMS in the UK. May I ask you if FINGOLIMOD (Gilenya)is being tested on patients with PPMS?
MouseDoctor said...
Yes FTY720 in Patients With Primary Progressive Multiple Sclerosis (INFORMS) Trial see the post about clinical trials a few days ago.

This study is fully recruited and is due to finish 2014.

If you check out these details in the links in the clinical trials post you can seee what is being done.
Anonymous said...
Is FINGOLIMOD therefore not available for PPMS patients who are not on a trial? Does this apply to the whole of the EU?
MouseDoctor said...
Yes that would be my belief, Prof G will know more.

It would not be licensed for PPMS yet
just RRMS.
Anonymous said...
Could you tell me more about treating PPMS with cannabinoids? What are the options and positive/negative effects? Is it something to look into?
MouseDoctor said...
At the moment sativex is available in some countries for the treatment of pain and spasticity, if you can get your PCT to fund. However in addition to symptom control there is a theorectical possibility that cannabinoids can have some beneficial effect on progression both PPMS and SPMS. There is a three year trial called CUPID that is testing a component of cannabis tetrahydrocannabinol).

This is fully recruited has been going on for the past 3 years and
is due to report the results this year

If this is positive you have the first drug for progressive MS. There are pharmaceutical versions of this called marinol.

The positive effect would be a slowing of progression and also possible symptomatic benefit and the negative effects are those associated with cannabis intoxication and cannabis use. Driving risksetc. The triilas will provide information on this.
Anonymous said...
What is the procedure: if a trial is positive, NICE has to approve the medication for the NHS? How long does approval normally take? Do you expect something in 2012?
Anonymous said...
Sativex is available on prescription in Germany since 2011. Can doctors collaborate on this across EU borders, i.e. if a German doctor prescribes the medicament, could a British neurologist do the monitoring?
MouseDoctor said...
Sativex is available in a number of countries in the EU, however within the UK things are very patchy. I understand from GW Pharma that Sativex is selling like space cakes..sorry hot cakes in Germany.

The issue is not if German doctors could prescribe and it could be monitoried in UK, it is a question of who pays for the drug. Is it work insurance, national health service, private funding. Each country has different systems

However, there is an interesting ethical issue here because there clearly are big differences in access to health care in different EU member states, should it not be universal. This is a question for the Eurozone and other Governments
MouseDoctor said...
What is the procedure if a trial is poisitive, NICE has to approve th medication for the NHS?

I'll do a post on this for tomorrow
Anonymous said...
Thank you for all yr Swift comments.
Access to Health care in the EU should be harmonized, I agree.
Payment of sativex aside, let's assume costs were legally covered through a German prescription, would it be legal for a UK neurologist to treat patient who brings along medication? I am trying to think about different approaches for unusual illnesses which are subject to complicated national legislations.
MouseDoctor said...
Not being a neurologist I can not comment with any authourity, maybe G will, but it would create issues I am sure.

You could be using the drugs outside the scope of its licence if it is not approved. Drugs can be conterfeited and so the neuro has no way of determining quality control if they do not know the source.

However it is happening all the time, I suspect because large numbers of MSers are self medicating with a variety of supplements and the neurologists would not know where the supplements come from. However I doubt that the nueor would administer the supplement
Anonymous said...
Not sure where to post this as first time. I have PPMS I amconcernerd about the relationship between Vitamin D deficiency and MS as my 11 year old daughter has had Vit D level of 14. She had difficulty walking and has now been treated with 4000u/day for four months. No follow up blood test has been done and we were told to give her over the counter vitamins which only contain 400u. The Rheumatologist said there was no link between Vit D deficiency and MS. Should we give her a higher and long term dose.
Gavin Giovannoni said...
Re: "Is FINGOLIMOD therefore not available for PPMS patients who are not on a trial? Does this apply to the whole of the EU?"

That is correct; only 50% of MSers in the trial with PPMS are getting the drug. Outside of trials would require a neurologist to prescribe it off license; this would be very difficult in the UK as the PCT/Commissioners would not pay for it.
Gavin Giovannoni said...
Re: "Payment of sativex aside, let's assume costs were legally covered through a German prescription, would it be legal for a UK neurologist to treat patient who brings along medication?"

Yes, of course! We could even make the case to the local PCT to continue the drug if it was having benefit.
Gavin Giovannoni said...
Re: "I amconcernerd about the relationship between Vitamin D deficiency and MS as my 11 year old daughter has had Vit D level of 14. She had difficulty walking and has now been treated with 4000u/day for four months. No follow up blood test has been done and we were told to give her over the counter vitamins which only contain 400u. The Rheumatologist said there was no link between Vit D deficiency and MS. Should we give her a higher and long term dose."

You should refer your daughter's rheumatologist to the literature on MS and vD. Regarding what dose to give; 400U will not be enough to make your daughter replete. She will need her levels checked and the dose increased as appropriate. There are clinical reasons not to give vD supplements therefore this decision needs to be taken with all the information on the table. I therefore can't make specific recommendations.
MouseDoctor2 said...
Anonymous.
Population and genetic studies are confirming the link between multiple sclerosis and vitamin D, says Richard Ransohoff in an article published recently in Nature ( 484,S8(12 April 2012). I suggest you refer you refer your rheumatologist to the article so that they may better educate themselves!
Helen Scott said...
Would just like to say "well done" to Prof G for his marathon efforts. Hope the legs are not too sore and a good massage was available.
Anonymous said...
Re: Sativex. Great news! Thank you very much, prof. Giovannoni. That was my thinking as well; that it could have positive reverberations on the UK decisions if German prescription helps the patient. Is Sativex an ad-on medication for PPMS which works with other main medication or could it work as THE main medication itself?
Anonymous said...
I must say, as someone with PPMS, I find it sickening how unhelpful consultants are when it comes to trying out new ideas and thoughts regarding drugs like Sativex and Fampridine.

Prof G, when you start trialling the new drugs to treat progressive MS, how long do you think it'll take for NICE to agree to give it to us? What date/ year do you have in mind?

Let's say the new trials prove fruitful, will you be monitoring progress throughout? In that case, if the drugs prove useful, can you demand that the trials be cut then short and the drugs get fasttracked?
MouseDoctor said...
Prof G can answer when he gets up he's on US time, but each drug trial is its own beast, what you think is plain sailing often isn't.

For new drugs it will be the same long process, but for some of the others we have in mind they are prescribable for say elipesy and some aspects of MS already, so one would not think that long.

The length of time depends on the trial design if you saw the video of Prof Gs new idea this can cut a seven year process down. I know this is being cagey but there is no point in giving dates that we have no way of guaranteeing

In any trial there is interim analysis notably for safety issues but if a drug is obviously working so well they can be cut short on ethical grounds.

Fast tracking is interesting tysabri was fast tracked and then PML came along..so the regulators become more caustious. There is the regulatory machinery to do this.
MouseDoctor said...
Sativex as an add on or could it work as the main medication.

Sure it could if you look at the experimental data, but in humans there is no proof so that is why trials are being undertaken now.

For symptom control I think sativex is an add-on, but this makes it harder to show effects in trials and complicates it for the pharma company also who would want it to be medicated alone
Anonymous said...
The unpleasant trolling comments could be from troublemakers or from a disturbed person.
MouseDoctor said...
Yes it is hard to say it is either
some one or it is orchestrated as a short burst, seeing if I can find out, but 3-4 times this month.

However it is sad really because it does them no good, it does us no good as it is unpleasant and wastes our time and the time of anyone getting stupid emails.

We don't have to see eye to eye, we don't have to agree, anyone on the blog knows that you can critise.

Prof G takes it better than me but he gets more practise.
MouseDoctor said...
Based on tonights (UK) or should we say this afternoons (North American) posts I would suggest a disturbed person........taking three-four minutes a post.

Unfortunately completely futile and now their 12-15 minutes of madness end up in the bin in a few seconds.
Louis Theroux’s “Extreme Love” said...
Mouse Doc, have you been watching Louis Theroux’s “Extreme Love” season on BBC 2? He explored the subject of autism and dementia in two separate documentaries. The series has been both touching and, at times, very heartbreaking. What is for sure is that neurological ailments are a serious global issue.

There are approximately a billion people in the world with some kind of neurological illness right now. Very little can be done for these people and the cost to society is overwhelming in economical terms. Furthermore, Theroux’s documentaries revealed that the people who suffer the most are immediate family members, the partners and children of sufferers.

Neurological ailments are a serious problem affecting both young and old people. There needs to be some kind of concerted thinking. Do you think that people like you, as in MS scientific researchers, can learn and share information with scientists working in the neurological field of say dementia or motor neuron disease? How closely associated are neurological disorders? What is the reality?
MouseDoctor said...
No I didn't see it maybe on catch up.

Yes we do indeed learn from other conditions, and that is what G is doing at the moment. We dont go to just MS meeting to me MS scientistists, but there are neurology meeting like the one G is attending so you learn about other diseases and then apply the knowledge to MS. You read papers of things occurring in other conditions. Some of ideas drugs for progressive MS say come from looking at motor neuron disease.

Some diseases have different causes but the end results are similar. For example a cell dies in one of essentially two ways it explodes or collapses in on itself. However there are many causes of cell death.

Good post
Gavin Giovannoni said...
Re: "what is the point of slowing down progression?"

MS is an exception; it is one of the diseases that is leading the way in terms of progress. You wouldn't think so by reading this blog.

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