Take-aways from today's Pharma talk

[Lulu54]

Here are the miscellaneous notes I jotted today during a luncheon I attended.

The Biogen Idec lunch talk I went to today was very interesting and quite a different approach than the usual tons of slides about Tysabri. It focused on what those numbers in clinical trials really mean. Of course it was directed at explaining why the Avonex number and Tysabri numbers are what they appear to be, but it really got me thinking.


Several key points which I think are close to the truth - people who do MS clinical trials fall mainly into two categories - either young and new to dx and have little to lose or advanced disease that isn't responding to tx an again has little to lose by participatin in a clinical trial. Both groups seem to do well in trials .  Perhaps This self-selection for participating in trials skews the results.

What works now for MS doesn't correlate to what used to work in the past, since the options have changed dramatically, Comparing therapies from 20 year ago to today's latest and greatest is doing an apples to oranges comparison..

Adverse events still intrigue me - my husband is doing a COPD double blind trial right now and he has been instructed to contact them about anyting and everythingl. If he has a car accident, he is supposed to let them know, just in case it relates to his drug.  Anyway, the point is even though the AE's may appear significant on the surface, we need to understand the circumstances further before assigned them specifically to a particular drug's side effect.

The presentation (Biogen slides) looked in depth at the use and AE's of Gilenya. Perhaps this was a practice run of getting ready for the BG 12 release and market blitz? But the neuro also talked about Natiluzamab (tysabri) and the PML problems associated with it. The take aways were:

PML occurs with other drugs/diseases too, but doesn't occur spontaneously. People with JC Virus negatives NEVER develop PML according to the stats.

Most interesting to me was the idea that that European incidence rate of PML is hypothesized to be significantly greater because of the cost constraints of health care. The ideas is many of these countries deny tx with the CRABs because of cost and they end up being treated with the chemotherapy drugs such as methotrexate. This early exposure to immunosuppressant drugs sets the body up to being susceptible to PML. PML in Europeans is MUCH greater than in North Americans, where the CRABS are the platform drugs for MS.

I hope you can make sense of these disjointed notes.   I am happy to entertain questions if something isn't quite clear.
Lulu

[Lulu54]

I am so sorry about all the short hand stuff -  I get tired of typing all these phrases over and over but usually try to use the whole word at least once each time for reference.

I don't usually quote from Wikipedia for obvious reasons, but this one on PML sums up answers for a lot of what you have asked....

"Progressive multifocal leukoencephalopathy (PML), also known as progressive multifocal leukoencephalitis, is a rare and usually fatal viral disease that is characterized by progressive damage (-pathy) or inflammation of the white matter (leuko-) of the brain (-encephalo-) at multiple locations (multifocal).

It occurs almost exclusively in people with severe immune deficiency, such as transplant patients on immunosuppressive medications,[1] patients receiving certain kinds of chemotherapy, patients receiving natalizumab (Tysabri)[2] for multiple sclerosis, psoriasis patients on long-term efalizumab (Raptiva)[3] or AIDS patients.

It is caused by a virus, the JC virus, which is normally present and kept under control by the immune system. Immunosuppressive drugs prevent the immune system from controlling the virus."

http://en.wikipedia.org/wiki/Progressive_multifocal_leukoencephalopathy

The estimate is about 56% of all people test JC positive.  I just go my test results back yesterday and surprisingly I test negative.  It makes Tysabri a very good choice for me and my treatment.

PML is not a demyelinating disease - it goes straight to the white matter of the brain .


Kelly, that is a great question.  I will try to remember to ask it when to opportunity comes up.  I will also try to do some research on that - there have to be research on that somewhere.  Anyone else is free to jump in and look! :-)

I have to get ready for work in a few minutes but please remind me to tell you some about the why's of PPMS research.  He did touch on that question as well.

later,
Laura

[anon07072023]

Beth, here's what I think I know:

PML - is a hard-core demyelinating disease that's somewhat similar to MS but more like MS on major steroids - and most of the time it's fatal, since there's no cure. It's caused by the JC Virus.

JC Virus - is already present in a lot of people - but once it's activated (or maybe rather, re-activated), it can cause PML.  If your immune system is REALLY low, then this could happen (ie from the use of chemotherapies).

CRABs - Copaxone, Rebif, Avonex and Betaseron

Lu, did they say what the Europeans disability rates are compared with the US?

Thanks for sharing this.
-Kelly

[maestro1959]

Hi Lulu,

Being somewhat new to this whole MS thing I am still learning some of the acronyms.  I will just list them.

Tx--treatment?
PML--??
CRABS--??
JC Virus--??
AE's--adverse effects?
BG 12--??

Do these discussions ever mention any of these drugs that may be recommended for treatment of PPMS?  Is there not someone, somewhere who has benefitted from the treatment of their PPMS with any of them?  I realize I am grasping at straws here, but knowing how different everyone's body chemistry can be why will they not at least try?

TY--thank you!.............LOL

Most sincerely,
Beth