Wednesday, February 24, 2010

FTY720 TRANSFORMS ext. phase checkup at 6 mo into 2nd year

Went for my trial checkup yesterday and had great results!

I didn't quite beat my best time on the timed 500 meter walk of 11 min 46 seconds, but I sure gave it the old college try. I was limping and stiff legged by lap 12 (out of the 25 laps of the hallway that somehow equal a distance of 500 meters).

I ACED that MSFC test where you have to listen to a recording of a guy spouting numbers every 3 seconds. Your job (if you choose to accept it) is to add the 2 numbers he said together, say the sum out loud, then throw away what you just said and concentrate on the last number the guy said so you can add it to the next one he says... it goes something like this.

Guy: 3
Guy: 7
Me: 10
Guy: 5
Me: 12
Guy: 5
Me: 10
Guy: 9
Me: 14

...and so on

It can get very hairy when Recording Guy starts saying things with double digit answers. All that math can wear a girl out! And I'm not good with adding 9 to anything although logically it should be as easy as adding 10...minus 1.

At one point, in the middle of the test, I faltered and couldn't come up with 9 + 6 = 15... and while my brain was farting, he spouted another number. I quickly said "15!" and then had to remember that he was still saying numbers... I was able to blast out 3 correct answers in one massive breath and get back on track.

It was close, but I scored 100%. :D

The clinical trial nurse shared with me that she once administered the test to another trial patient who is a school teacher and she failed so miserably she broke down in tears. I can really relate to this as the first time I took the test I didn't know the *trick*. Since I devised The Trick © I haven't failed yet.

It consists of simply saying the answer necessary and "throwing it away" only to repeat over and over (in my brain) the number the guy just said... so it would go something like this:

Guy: 4
Guy: 8
Me: 12 (8 8 8 8 8 8 8 8 )
Guy: 9
Me: 17 (9 9 9 9 9 9 9 9 )

And that's all there is to it. Since concocting The Trick © I now no longer fail this test, my hair is growing in thicker, I have made a fortune on Wall Street, and my boobs are 3 times their normal size.

You're more than welcome to use
The Trick © but be forewarned: your mileage may vary.

Other highlights of my visit included the animated discussion of the new name "Gilenia" which I stated sounded like LymphopENIA or some other such medical deficit. My clinical trial nurse said to her, it brought about the image of GardENIAs.

I told her that unless they put a flower on the box, I'm going to be sorely disappointed.

I had my PFT performed by a new guy who asked me if I could teach him how to administer the test and then, after a big ol "Ha Ha!" about that... I ended up having to teach him how to administer the test.

Well, at least the part with the flute that you have to measure peak inspiration and expiration. That thing that looks like something a pothead might get some bright ideas about how to turn into a bong... It is a clear plastic cylinder with a hole in the side, open on one end and closed on the other and hooked to some plastic tubing that in turn connects you to the machine that knows everything... every little breath you take, so to speak.

The point of the test is to breathe normally, and then take a big breath and cover the hole in the "flute" with your finger for a couple seconds during peak inhale or exhale, to create resistance, thus in turn, measuring your lung function.

We kept getting the timing off and I was getting my cardboard mouth guard soaking wet and soggy. That's when I told him "you're not doing it like the other guy does it."

He perked up hoping for an answer to how to get this old stupid dog to perform his new trick.

He said "how'd he do it?"

I said "He said 'Breathe nomal, finger over the hole, deep breath in, finger off.'"

It was like a light shone down upon him from the heavens as he had his eureka! moment. You'd have thought I just gave him the secret to the universe.

Then his eyes narrowed to little, untrusting slits.

"If you KNEW that, why didn't you just DO that?"

To which my reply was "I was the one having to breathe. You can't expect me to breathe AND think at the same time, can you?"

With the test completed he walked me to the exit door, thanking me for actually teaching him something about communicating his directions to patients better.

I told him he'd be getting my bill in the mail.

Then on to the eye exam. Well actually I got it all out of order since blood draw was first, PFT second, EDSS and patient questionnaire was next, and then eye exam last, but that's the power of being an Author. I can relay it any way I like. And since I have short term memory issues, I'm not worried so much about order of events as much as I am ecstatic that I can recall any of them.

So, where was I? Oh yeah, the eye exam. There is one important reason that I remember distinctly that this was the LAST test of the day. Because I had my eyes dilated so much I had to sit in the parking lot for an additional hour in order to allow them to adjust enough to attempt a 2 hour drive at dusk in rush hour 6 lane traffic.

The eye exam was not anything to speak of really. I was in and out and given a clean bill of eye health in a relatively short time (in waiting room years).

But the part I really remember was getting the drops. 3 kinds. One to numb me for the pressure test, some yellow stuff to see what, I don't know (but it really freaks you out when you wipe your eyes with a Kleenex and then look and see fluorescent yellow stuff on it. Eeek!) and then the drops to open up the pupils.

After I got the drops, within a minute I could feel them draining into my sinuses. And within another minute my head started to hurt. Just mildly at first, but snowballing into a big old headache of peculiar intensity.

I asked the doc when she was shining that bright light in my eyes if it was normal to get a headache from the drops.

She asked "do you have a history of migraines?"

To which I said "yes", but was thinking "well DUH! Read my file, would ya??"

"Oh, well those drops can precipitate a migraine so be sure to get some sunglasses on the way out."

So I left there looking like I just came out of a 3D movie that I really hated. I had the stupid glasses but all I could do was frown.

I made it home somehow. My vision looked a lot like this only you had to add in a squint factor since it was making my headache worse:

How I managed to get hom without a huge game of bumper cars is beyond me.

I still have the headache today, but luckily my vision has returned to normal.

All in all, it was a pretty good day. I liked all my results and all the people who work with the patients in the trial are great to be around and fun to talk to. Everyone has a good sense of humor. Of course, they have to if they are going to be dealing with the likes of me. :D

The lead investigator, when I told him I would be making him my regular neuro after the trial ends responded with a big smile and quipped:

"I'll be sure to call in sick that day."

Monday, February 22, 2010

Gilenia? Are you serious? You'll always be Fingolimod to me.

This little tidbit just hit my inbox courtesy google alerts....

Novartis Oral Multiple Sclerosis Development Compound Gilenia®* (FTY720) Granted US Priority Review Status

Really??! Is that the best you could do? How the hell am I going to remember Gilenia? And I'm not crazy about the "enia" suffix. Sounds too much like a depletion of something vital to my system. Conjures up negative connotations. Sure, I realize that it does create lymphopenia which is the primary medthod of action, but come on!

I was hoping for a little more glitz and glamor. May as well just stick with Fingolimod (which I will no doubt do).

And is "Priority Review" a fancy, scientific term for Fast Tracking? Does this mean evil people at the FDA are reading my blog and rubbing their hands together and laughing their insidious "MWAHAHAHAHAHAHA!" knowing full well that the end of the review process marks the end of my Pharm Party?

Say it ain't so!

Oh yeah, I almost forgot... Here's the story that goes with that headline:

. US Food and Drug Administration grants priority review status after accepting US regulatory submission for 0.5 mg once-daily Gilenia (fingolimod)
.
US and European Union regulatory submissions completed in December 2009 for Gilenia include more than 4,000 patient years of clinical trial data

Basel, February 22, 2010 - Gilenia®* (FTY720, fingolimod) has been granted priority review status by the US Food and Drug Administration (FDA), which accepted the regulatory submission made in December 2009 for this medicine. Once-daily Gilenia (0.5 mg) has the potential to become the first approved oral therapy for the treatment of multiple sclerosis (MS).

The FDA grants priority reviews for investigational medicines that could offer significant advances beyond current treatments or where no adequate therapy exists. As a result of this designation, the standard 10-month FDA review period will be reduced to six months.

Since Gilenia involves a new active ingredient (New Molecular Entity), the FDA is likely to require an Advisory Committee meeting and evaluate the risk management program, which could result in the FDA extending its review at the end of the six-month period in June 2010.

"We welcome the decision granting priority review to Gilenia, which underscores the potential benefits of this medicine to patients," said Trevor Mundel, MD, Global Head of Development at Novartis Pharma AG. "MS is a leading cause of neurological disability in young adults, particularly in women, and this medicine has the potential to offer real advances in the care of people with MS."

Approximately 4,000 patient years of experience have been gained in MS clinical studies involving Gilenia, with some patients now in their sixth year of treatment. Data from one of the largest-ever Phase III clinical trial programs conducted in MS patients were submitted to support the US and European regulatory submissions, including results of the TRANSFORMS and FREEDOMS studies that were recently published in The New England Journal of Medicine[1],[2].

Combined data from these studies provided evidence of the efficacy of Gilenia in reducing relapses, disability progression and brain lesions in patients with the relapsing-remitting form of MS as well as safety data. Approximately 85% of patients with MS are estimated to have the relapsing form at the onset of disease[3].


Disclaimer
The foregoing release contains forward-looking statements that can be identified by terminology such as "priority review," "potential," "could," "will," "likely," or similar expressions, or by express or implied discussions regarding potential marketing approvals for Gilenia, or the potential timing of such approvals, or regarding potential future revenues from Gilenia. You should not place undue reliance on these statements. Such forward-looking statements reflect the current views of management regarding future events, and involve known and unknown risks, uncertainties and other factors that may cause actual results with Gilenia to be materially different from any future results, performance or achievements expressed or implied by such statements. There can be no guarantee that Gilenia will be approved for sale in any market, or at any particular time. Nor can there be any guarantee that Gilenia will achieve any particular levels of revenue in the future. In particular, management's expectations regarding Gilenia could be affected by, among other things, unexpected regulatory actions or delays or government regulation generally; unexpected clinical trial results, including unexpected new clinical data and unexpected additional analysis of existing clinical data; competition in general; government, industry and general public pricing pressures; the company's ability to obtain or maintain patent or other proprietary intellectual property protection; the impact that the foregoing factors could have on the values attributed to the Novartis Group's assets and liabilities as recorded in the Group's consolidated balance sheet, and other risks and factors referred to in Novartis AG's current Form 20-F on file with the US Securities and Exchange Commission. Should one or more of these risks or uncertainties materialize, or should underlying assumptions prove incorrect, actual results may vary materially from those anticipated, believed, estimated or expected. Novartis is providing the information in this press release as of this date and does not undertake any obligation to update any forward-looking statements contained in this press release as a result of new information, future events or otherwise.


About Novartis
Novartis provides healthcare solutions that address the evolving needs of patients and societies. Focused solely on healthcare, Novartis offers a diversified portfolio to best meet these needs: innovative medicines, cost-saving generic pharmaceuticals, preventive vaccines, diagnostic tools and consumer health products. Novartis is the only company with leading positions in these areas. In 2009, the Group's continuing operations achieved net sales of USD 44.3 billion, while approximately USD 7.5 billion was invested in R&D activities throughout the Group. Headquartered in Basel, Switzerland, Novartis Group companies employ approximately 100,000 full-time-equivalent associates and operate in more than 140 countries around the world. For more information, please visit http://www.novartis.com.

The brand name Gilenia has been provisionally approved by the FDA for use in connection with the product, but the product itself has not received marketing authorization or NDA approval from any regulatory authorities.

References
[1] Cohen J. et al. Oral Fingolimod vs. Intramuscular Interferon in Relapsing Multiple Sclerosis. N Eng J Med. Vol.362 No.5, Feb 4, 2010 (printed version).
[2] Kappos L, et al. Placebo-Controlled Study of Oral Fingolimod in Relapsing Multiple Sclerosis. N Eng J Med. Vol.362 No.5, Feb 4, 2010 (printed version).
[3] National Multiple Sclerosis Society website. http://www.nationalmssociety.org/about-multiple-sclerosis/what-is-ms/index.aspx. Accessed January, 2010.


Sunday, February 21, 2010

Just how long....?

...Can a person taking Fingolimod expect to be in remission?

I think about this question A LOT lately. This has been the longest period of remission in my MS *career* (it's kind of like a job since dealing with it every day sucks), and I can't help but wonder when it's going to end.

Yesterday marked the 2 year, 6 month mark since starting the trial, and the 2 year, 10 month, 16 day mark since the start of my last MS attack. To me, that is insanely fantastic. I still can't get over it and (other than the part about being broke and knowing it's NOT true), I often feel like I have won the lottery or something. Well, I am winning the MS lottery I guess.

If I were a character in some video game and the cruel kid playing forced me to choose between the More Health potion jar, or to go around finding bags of gold coins, I'm taking the health every time. It's only logical that running around gathering bags of gold coins all willy nilly without regard to your Health-o-meter getting low and turning red will only result in a premature Game Over message...

Fingolimod has restored my Health Meter back into the green zone and I just don't know when the MS monster is going to poke it's head back up in this game and bite me on the butt, sending my health meter into a downward spiral.

I read somewhere on the internet (so it must be true, right?) that FTY-720 has residual effects. Some lab rats somewhere (the furry version, not two legged) were followed after being given the drug to see how long the stuff stayed in their system and had a beneficial effect. If I recall correctly, (and don't hold me to this because I can't recall correctly why I went into the kitchen and stood in front of the pantry a few minutes ago), it seemed the length of time it had a beneficial effect depended on the length of time one was on the stuff.

It would really be cool if you could spend 3 years taking it for 6 years of coverage. Maybe this trial will drag out for 10 years (please oh please) and, when the magic Fingo carpet gets yanked out from under me, I could have 10 years of residual protection.

That's probably too much to ask. It's probably more like a guy taking off a condom and saying "the residual effects of having warn it will guarantee that you are safe from STDs and becoming pregnant, I swear!". Guys. They'll say anything won't they? (JUST kidding, okay? You men out there don't need to send me snarky comments because I made this drivel up.)

Back on topic... It used to be that, way back at the start of this trial, if I so much as sneezed I would cringe and duck and wait for the wrecking ball with the big "MS" logo on the side to smash me where I stood. I used to have pretty ferocious attacks which were precipitated by nothing more than a head cold or stressing about money. It was only natural, when I experienced these triggers, that I would brace myself for the wallop of a big old relapse.

I think it was somewhere around the beginning of the second year into the trial that I got a cold for a couple of days and realized it came and went and I didn't even worry about an MS attack.

By God, this stuff has more power than I thought! It has the power not only to leap tall buildings in a single bound, but, more importantly, to force me to feel normal again.

The average person, health meter intact and never drained by a chronic disease, cannot realize what a precious gift they possess. They run around with a green, sparkly health meter over their heads never even aware they could ever turn to yellow or red. To have "normal" health is taken for granted.

To have something like Fingolimod put this green, shiny almost-as-good-as-new health meter over my head and allow me to run around (literally!) in this big video game that is my life, to me is just incredible.

Maybe it's got something to do with the other lifestyle changes I made at around the same time as starting the study (i.e. quitting smoking) and the fingo's got nothing to do with it? Who knows. Maybe the simple act of laying down the coffin nails and not cramming something lit on fire into my mouth so I could suck the soot and ashes of it into my lungs was enough to make the MS monster say "Ahhhhhhh!" and go back into his cave and hibernate. Maybe.

But I'm not willing to give up that little pill in order to find out.

I never thought when I got into this trial that I would become so affectionately and emotionally attached to an ugly Chinese fungus, but I have. The day this all ends will be a sad one indeed.

So I'm wondering... don't they need to do a reeeeeeaaaaaaaalllllly long term extension study (say, hmmmmm, til I die maybe? <--omg I HOPE that's going to be a really long time (frantically seeking wood on which to knock!)) to see how long this stuff can keep a person in remission? Come on! Nearly 3 years with no relapse? That's gotta be some sort of record. Well, for me at least I KNOW it is. The longest I went before that was the first 38 years I lived, but that doesn't count because it was pre-diagnosis.

I propose a study called "JERI" or Just Exactly how much time Remains In which patients from the phase III trial can go between relapses? (See, Novartis, you aren't the only one who can come up with those catchy trial names! I figured out how you do it and before that I thought it was cheating to use letters out of the middle of a word for your acronym).

Anyhoo, what better person to have in this extended, last until she's dead, Phase Infinity trial than the person's whose name matches the acronym? Eh, see? Great idea, no?

In case that idea never makes it to the board room, my only other option is to hope the FDA takes their sweet time. I can't bring myself to wish that selfish wish, though, because there are TOO MANY MSers out there waiting to quit jabbing sharp metal objects into their bodies repeatedly.

Ya'll are just going to love this therapy. You pop the teensy tiny capsule, way smaller than a vitamin, and take a sip of water. The capsule floats in your mouth and when you swallow you have to check around with your tongue to make sure it's gone. You never felt it go down.

It just doesn't get any better than that.

I'm praying I can go forever without another relapse on this stuff.

That said, watch. I'll be crawling back to this computer chair tomorrow to try and bang out H-E-L-P M-E-! on the blog.

Cringing and ducking, I'm outta here... to go have a NORMAL, BORING DAY. YAY! I've won the Lottery.