Access to T DM-1

phil · 11-30-2010, 10:42 AM

This is my first post ever. My wife is a HER2 + patient, Stage IV since diagnosis in 2006. She is alive because of the advanced treatments we received at MGH in Boston. Earlier this year we were referred to Dana Farber for a new drug , Herceptin T DM-1. We were waiting to get it, when the FDA abruptly declined to approve it. ( Sept. 1) . It is not available in the Northeast, incl. NY. It's a long story, but the phar. co., Genentech has it at 13 sites elsewhere in the country. You can call them at : 888-662-6728, 9 A.M. - 6PM., EST. We have had to struggle to get it, and have finally received it in Virginia last week. We hear great things about it. It is the first " conjugate " , where Herceptin is paired up with a " toxin " ( chemo -like substance ) . Goes like a heat-seeking missile, only to the mutated cells . Few side-effects for many. Nothing is 100 %, but the Phase I, II data is very promising. Especially when you consider the alternatives for Stage IV HER2. So far my wife experiences only fatigue, and a low-grade fever. Three women we have met, have greatly benefited from the drug as well.
I cant talk too long at this point, but I wanted to get the word out. I am interested in talking with others who would like to advocate for this drug.
I definitely would recommend thast if you haveHER2 that has progressed on regular Herceptin, or especially if you have tried Tykerb/Xeloda, and it didn't work , or the side-effects were intolerable, that you talk with your doctor about this drug.
There are 2 " on paper " ways to get a non FDA-approved drug; "single patient access" or " expanded access" , If you call Gen, ask about " expanded access". Exp. access has only a 2 week " wash-out " period. ( Because of overall mild side-effects ) . There is no single -patient access at this time.It is also available in small clinical trials at Dana, ( and elsewhere) but we can't wait 3 months or longer to get in.
If the FDA would approve it ( which they should ) , we wouldn't jump through hoops... I'll have plenty to say about the FDA later...
Thanks, and God Bless...

ElaineM · 11-30-2010, 11:39 AM

Welcome aboard Phil. Thanks for your posting and for sharing information about T DM1 with us.
It is something we have been discussing, hoping and praying for all year. Some of us were pretty upset at the FDA when it refused to approve it a couple months ago.
I posted some links about the various T DM1 clinical trials in the clinical trials section and maybe the her2 section. In any case if you want more information about T DM1 and the ongoing trials go to www.clinicaltrials.gov and do a search for T DM1. You will get a list of the various trials for T DM1.
A few of our members have taken T DM1 and are doing well. I wish your wife well. I hope T DM1 works for her.
Let us know how she is doing from time to time. Take good care of yourself and your wife. Enjoy whatever holidays you celebrate in December and January.

phil · 12-08-2010, 05:58 PM

My wife is doing wll. Today we got back our 2 week labs, and they were " excellent ", according to MGH. After just one dose ! We now have heard from over a dozen women who are doing well. One woman for almost 3 years. It doesn't work for everybody, several women had to come off.

phil · 12-16-2010, 09:34 AM

My wifes labs just get better and better, with just one dose of T DM-1. We are in Va. to get the second dose. And almost no side-effects ! Her hair is growing in fast.
I heard on another web-site that people are " excited " about " compassionate use " being expanded " soon ". People need to be clear about the FDA terminology ; As of today ther is NO single - patient access ( also called compassionate use ), and there is no expanded access in the Northeast ! I will be excited when the drug is fully - approved by the FDA . Which should have happened 3 1/2 months ago !

Sandra in GA · 12-18-2010, 07:17 AM

Phil,
Thank you so much for this post. I am so glad your wife is responding. I am sure prayers have been answered.
Sandra

Chelee · 12-18-2010, 10:10 PM

Phil,
That is is fantastic that your wife is getting such a great response from T-DM1 so quickly. I've heard nothing but very encouraging things about it. In fact when I went out to see Dr. Slamon he was excited about T-DM1. If he liked it...I knew it was going to be a really good drug for us to use

We have one Her2 sister on this board named Chrisy. She enrolled in a trial with T-DM1 almost 3 yrs ago and it has kept her NED this entire time. She was stage IV with liver mets. I can't wait till all of us that need it can access it. Last I heard T-DM1 might get approved for use this coming Fall. (I wished it was sooner then that.)

I'm thrilled to hear your wife is doing so well with very few SE's...that's great! Sure sounds like your wife is a really good responder to T-DM1. Wishing your wife the very best! Thanks for sharing the news with us.

Chelee

phil · 01-06-2011, 07:33 PM

Check out my wifes story on the front page of Jan. 5th Boston Globe. boston.com/business/healthcare/articles/2011/01/05/testing. Our struggle to get T DM-1. If noone else will speak for us, then we'll just have to do it ourselves. Wait for the FDA, until 2o12 ? No way !!
This is a breakthrough drug for criticall ill patients. More quantity of life for many, and definitely better quality of life for even more pt's.
Just like with Herceptin itself in 1998, the FDA must approve now, we can test later.

'lizbeth · 01-06-2011, 08:33 PM

Phil,

We've been waiting for you. Most of us are mystified that TDM1 was not fast tracked.

Which phase of the trial is this in? Stage 2 or Stage 3?

schoonder · 01-07-2011, 06:50 AM

Phil, I salute you!

1rarebird · 01-07-2011, 09:27 AM

Phil--Thanks so much for sharing your story and this information on TDM-1. It has given me a shot of optimism since I worry too often that I could be on the short road from early stage to IV. Sort of like waiting for the other shoe to drop. But your wife's success with this new wonder drug helps me trust that there will be excellent options for me in the future if the worst happens. Thanks again and best of luck to you and your wife.

Phil (aka bird)

phil · 01-07-2011, 05:44 PM

This is Phase III since last year ! The FDA says it must be tested against another combo, Tykerb/Xeloda, which is more toxic, and is no more effective . My wife was on it, and it was awful, her fingers split open, she would wear gloves with lotion inside, and it still hurt. GI effects, turned her palms and soles orange.
The FDA said " patients must exhaust all other options " ? My wife has ! They were just talking nonsense. It must be both a cost issue and just bureaucratic bungling. They will review in March, 2012 ! Criminal.

phil · 01-30-2011, 08:09 PM

Went to DC this week for tx. # 3. Our 8 week CT was " fantastic " according to the Va. doc ! Met with aides to Sen. Harkin, chair of HELP Comm. They oversee the FDA. We'll see if they " HELP ". I think I gave them alot to look into. I told them " The problem started with the FDA last Summer, the solution must start with them. " Genentechs limiting of access will be looked at later.
Any of you with Advocacy contacts ( the higher up the better ), send our Jan. 5th Boston Globe article to them, and my e-mail, pmccartin@comcast.net. I'd love to talk about how this FDA Administration is denying such an obviously superior drug to critically ill patients.
If they do this to one drug, they will do it with others - PI3 kinase inhibitors, PARP inhibitors, Avastin, vaccines in the near future.
We are all in this together ! Keep up the fight !

ElaineM · 01-30-2011, 09:16 PM

Thanks Phil. I salute you for going to Washington. There is a site where a person can start a petition. I don't remember the url, but I put a link to it in one of our previous discussions about T DM1 a couple months ago, so if you want to do a search on this site for T DM1 it might come up. Let us know if you start some kind of petition.
The FDA may have started the issue last summer, but the drug company is also being extra careful. It seems to be making doctors and medical groups that want to offer some of the trials to their patients jump through more hoops than they had to jump through before.
The docs and medical groups were planning to offer some of the trials here and were ready several months ago. It seems everytime we ask about it there is a new thing the docs or the medical group has to do to please either the drug company or the FDA. I will keep asking for all her2 positive people in Hawaii every chance I get.

schoonder · 01-31-2011, 07:44 AM

Phil,
Was thrilled to read that your wife in this short time span is responding so well to this relatively easy to tolerate drug and I can readily understand why many other patients having similar MBC characteristics would like to have access to this new agent.
There's something fundamentally very wrong with FDA's decision to on one data driven hand allow, yes maybe even encourage a 25 center extended trial to be conducted and on the other hand declare that this data from the 2nd phaseII trial, one that was necessitated because a recent approved Tykerb/Xeloda treatment changed trial option landscape, could not be looked at.
Drug's efficacy and tolerability and a good measure of common sense should be driving force behind FDA decisions.

phil · 02-19-2011, 07:17 AM

Lorraine and I were interviwed by Kelley Tuthill, from Boston Ch. 5 new. She will air the interview in the next week or so. She is aHER2 survivor herself.
T DM-1 continues to be " amazing " for Lorraine. All liver lesions shrinking. L. had her bile duct stent re-placed last week. That surgeon said its amazing. Hes never seen T DM-1. Said " When we put that stent in in Nov., your duct was squeezed almost shut ( 2 mm.). Now its wide open ! " After 4 doses !
Now, lets get it to Boston, Ny. etc.

schoonder · 02-19-2011, 07:39 AM

Thanks for posting Phil, here's another patient that's doing real well on the west-coast.

http://santamariatimes.com/news/loca...cc4c002e0.html

'lizbeth · 02-19-2011, 08:49 AM

I'm so encouraged to hear stories of patients doing well with this treatment. Thanks Phil for bringing attention to the success of TDM1.

Chelee · 02-19-2011, 03:54 PM

Phil,
That is such great news to hear how well Lorraine continues to do on T-DM1.

I also love hearing how amazed her surgeon was! Thanks so much for all your hard work and effort to spread the word about this wonderful drug. I am due to start chemo and have been trying so hard to get by just long enough to get on T-DM1 myself. I keep praying for breaking news that the FDA has approved it earlier then planned on. It's so upsetting to have us run thru toxic chemo's when there is more then enough hard data to show T-DM1 is safe and effective with much less side affects. It's such a hardship for so many of us to travel to these EAP locations...let alone the financial burden it causes.

schoonder, Thanks so much for sharing that link with us. Another uplifting and encouraging story about of how well T-DM1 can work for some. It sure made major changs in this women's life. To go from a wheelchair to walking around again is pretty amazing! Just got to love it.

It's stories like this that bring real hope to so many of us. Thanks again Phil for all your hard work--it's so appreciated more then you know!

Chelee

Henry · 02-26-2011, 01:24 PM

Phil,
Thank you for sharing about your T-DM1 experience. We are encouraged about the promising reports on this advanced treatment. My wife entered the trial and received her 1st treatment two days ago. Thankfully we are only 120 miles from the trial location. Our current status is Stage 4, with lung mets. (original Stage 2 diagnosis in June 2002 with recurrance in Dec 2007). The day after 1st treatment brought a little more fatigue than the Navalbine/Herceptin, and other moderate side effects (low fever, aches possibly due to the lung mets) but appears to be manageable. We hope this trial continues to be effective in the battle against BC. We welcome any suggestions in complementing the treatment or any managing side effects any may have experienced. Best Wishes

phil · 02-27-2011, 04:03 PM

I am so glad that your wife gets to try this wonderful drug. About 40% of those who try it, have great results. I think those numbers will rise once the FDA allows it to be approved. So women can get it earlier in the fight. The s/e seem to lessen as your body adjusts to the drug. For Lorraine, the s/e have gotten better as she went from round 3 to 4 and now round 5. Fatigue is less ( but still there). Round 5 fever was 100.4 , just one day. We are trying to add iron to her diet, with red meat, spinach, broccoli., etc.
L. paces herself, doing some walking, a mile every other day or so. She has a kidney stent, so she has to be careful with exercise. We went snow-shoeing yesterday, for about 45'. God bless and good luck. I pray the drug does the same wonders for your wife that it has for mine.