Access to T DM-1

[phil]

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]

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 (http://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]

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]

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]

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]

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]

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]

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]

Phil, I salute you!

[1rarebird]

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]

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]

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]

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]

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]

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]

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

http://santamariatimes.com/news/local/article_cd4985e6-3bfa-11e0-a3c5-001cc4c002e0.html

['lizbeth]

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]

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]

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]

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.

[phil]

Lorraines story should air tomorrow night ( Thurs, ) ch. 5 boston, at 11. bostonchannel.com. Hope it goes national.

[Chelee]

Hi Phil,
I look forward to reading Lorraine's story. Thank goodness for the internet...this way we don't miss much. I still can't thank you two enough for all the hard work to help spread the word about T-DM1. When you have time keep us updated on Lorraine. You two take care.

Chelee

[phil]

Check outLorraines' story on bostonchannel.com, Search :
" heidke-mccartin". Lorraine was awesome, I'm so proud of her. You can see how well shes doing. Living proof that this FDA Admin. is botching up the approval process. Cant keep pace with the new drugs. And Kelley Tuthill did agreat job helping us feel at ease, and putting the story together. Lets keep up the pressure !

[Chelee]

Phil,
Excellent job getting your story across. You even managed to get John Kerry to fire off a letter. This whole T-DM1 issue is so infuriating to me. Especially for us stage IV women.

This link below will take you to Lorraine's story for those that want to see it. It's well worth it...great job!

http://www.thebostonchannel.com/health/27150551/detail.html (http://www.thebostonchannel.com/health/27150551/detail.html)

Here's to Lorraine becoming NED thanks to T-DM1.

Chelee

[phil]

Those of you who live in the DC or NY area, call your local TV, newspapers, ask for the health editors e-mail. Send them the story.

[Chelee]

Phil,
I don't live in the NY or DC area...but I still like the idea of calling my newspaper. I use to work for them yrs ago. It might be worth it to tell them about your story, along with my own troubles and opinions on this T-DM1 issue. Maybe I can get their attention? It's worth a try. Thanks for your idea.


Chelee

[schoonder]

When FDA approved Tykerb/Xeloda protocol for MBC it changed landscape of available treatments.
At that time Genentech readily and willingly discontinued T-DM1 Her2+ 3rd line phase II trial that started in 7/07 and after implementing necessary alterations to account for this change, began a new evaluation (7/08). Yes, net result for pertinent MBC patients was a program delay of one year, but those are the rules, data presented to FDA must include latest treatment options for that disease. A possible benefit from conducting this second phase II is that FDA and Genentech had opportunity to sample results for not only a longer period of time, but also examine a much larger candidate population then they originally intended for this what was hoped to become a fast-track program.
So where are Genentech and FDA now? Well from Her2+ 3rd line MBC perspective they are at a two plus year standoff, because FDA refuses to evaluate the supplied data and Genentech isn’t willing to correct shortcomings identified by FDA for this particular trial. By mid-2012 company hopes to submit a new set of data from a currently worldwide ongoing trial in 2nd line Her2+ MBC (Emilia) and by doing this they also hope to receive approval in 3rd line setting.
This sums it up between Genentech and FDA, but where does this leave the thousands of patients that currently are identified being 3rd line Her2+ MBC, the ones that are so desperately in need of new, effective and easy to tolerate drugs? It looks like that patient population will be left carrying the bag. Usually in a standoff between parties they continue to get together to get differences resolved, when that after some finite period of time fails and subject matter is deemed of sufficient importance, arbitrators are assigned to bring matters to a fair conclusion.
Well, there are no continued talks, no arbitrators, guess subject matter isn't important, let’s do nothing, pull the rug out from under these sick people, let’s not give them opportunity to benefit from a potentially efficacious drug, let’s just remove all hope. Maybe this is could be conceived as a cruel and unusual punishment, but these patients are not criminals, so the 8th amendment of the U.S. Constitution doesn’t apply to them. Case is shut.

[phil]

Thanks chelee. Anyone contacting media should mention that Gen. is keeping access from NY, Boston, the whole Northeast.
Schoonder, the FDA and Gen. are talking, taking their sweet time, but they are talking. We must keep up the pressure.

[phil]

genentech announced that they are closing the access program as of Sept. 6. Those already in it will be grandfathered in. They didnt say it, but its related to this FDA's fumbling of the development of t dm-1, a life-saving tx. A drug w/ the best stats coming out of Phase II trials , since Herceptin itself, 15 yrs before. This FDA denies approval of t dm-1 last Sept., recommending 3 more yrs of study, and approves Halaven the same month, a drug w/ much, much less impressive stats. I have nothing against halaven, but this FDA is burying its head in paperwork, refusing to see whats on tv. " Living Proof " , scores of women like Lorraine.Making us jump through more hoops, now its their new mantra , " Overall Survival ".
As research gets closer to winning the war on breast cancer , this FDA is losing it. Speak up !!

[Jackie07]

Chrisy has posted detailed information on this thread:

http://her2support.org/vbulletin/showthread.php?t=50864

[phil]

Important addendum. Gen. is adding a large new trial to try to satisfy this FDA 's new focus on " Overall Survival ",( I call it " Slow As Usual "), at 42 sites, comparing t dm-1 to other txs. None of this should have happened, if ever adrug deserved accelerated approval, it was this drug. Starting in 2010, this FDA began slowing down the process, just as this breakthrough drug was ready.
They refused to see what they had already pre-determined they would not see. Interesting that they began the process of revoking Avastins approval at the same time .
They need to be fixed from above. This is President Obamas FDA, and this Congress's FDA. Make sure you let your reps, and the Congressional " HELP" Committee know how you feel about this FDA. Once they know, then they own it. Its theirs. In an election year. 46,00 women and men will die fron breast cancer this year, and this FDA is slower, more contradictory than ever...

[schoonder]

Latest update on wider T-DM1 access.
http://assertivecancerpatient.com/2011/08/index.html

[phil]

Lorraine got a call from the research clinic in Va. They have started the process of transferring her T DM-1 Access to Dana Farber in Boston ! We are very, very excited !!
Any woman in the Access Program, traveling long distances to get T DM-1, should contact your site , to ask about transferring to a site closer to home. Make sure the site has started the transfer process. The sooner you dont have to travel , the better.
Any woman w/ Her2 illness that has progressed on taxanes, and tyk/ xel, ( or intolerable s/e w/ tyk./ xel ) can still enroll in Access by Sept 6th. After that a new trial ( Theresa) will be starting, but the start date is un-clear. And any trial will have more regs thatn Access , like a 30day wash-out vs. 2 weeks.
Contact Genentech Access Solutions w/ any ? about the above info.

[StephN]

WOW, Phil! I am jumping for joy right along with you and Lorraine. Thanks for letting us know that your long and intrepid work is paying off.

I am also so happy that the drug has done such good for your dear wife.

[phil]

Please support our petition to Dr. Sebelius, asking her to investigate this FDA's denial of Herceptin T DM-1 to Stage IV Survivors. She is this FDAs boss. Go to the link below, study up on the issue, send the word out !
http//signon.org/sign/fda-blocks-life-saving?source=c.url&r-by=60340

[schoonder]

Phil
Link didn't work for me.

[rhondalea]

The link is missing a colon. It should be:

http://signon.org/sign/fda-blocks-life-saving?source=c.url&r-by=60340

rl

[Jackie07]

Newest information posted by Brenda on the Her2 Board:

UPDATE as of today, 9/16/2011, from Genentech:

Genentech would like to provide an update on the protocol amendment to the T-DM1 patient access study (called T-PAS or TDM4884g) that may allow some patients enrolled in the study to receive T-DM1 closer to their homes. We submitted the proposed amendment to the FDA on August 8, 2011, and have received no questions or concerns to date that would require re-evaluation or revision. As a result, we are proceeding with this amendment. All of the study sites involved have been informed, and the next step is for each site to approve and implement it within their own institutions.

The T-PAS protocol amendment provides T-PAS patients the option to enroll into another, ongoing Genentech/Roche study (called the “extension study” or TDM4529g), which, in essence, doubles the number of sites and locations where T-PAS patients can receive T-DM1.. This option is only available to patients currently enrolled in T-PAS. Transferring to one of the extension study sites is optional and available to patients who have completed at least six cycles (18 weeks) of treatment, are determined stable by their doctor (i.e., not progressing and tolerating T-DM1 without major side effects) and meet other required criteria for the TDM4529g study. Current T-PAS patients interested in receiving T-DM1 at a site closer to home should contact the T-PAS study coordinator at their current site. The study coordinator can help determine if one of the extension study sites is closer and begin the transfer process to the new site. Since the amendment requires IRB approval at each of the original T-PAS and extension study sites, some sites may be able to accommodate the transfer of T-PAS patients more quickly than others.

People not enrolled in T-PAS but still interested in T-DM1 should talk to their doctor about their eligibility for the other T-DM1 studies currently enrolling patients.

__________________
Brenda

[annmask]

Hi Phil,

I find this site hard to use.....looks like maybe you added this to the existing TDM thread.....I had a hard time finding it, and tried post it myself. It still seems hard to locate and it seems that this site should have about everyone signing if they know about it!

[ElaineM]

Thanks for your efforts Phil. I signed the petition and sent the link to a few other people and asked them to consider signing it too. Even if your efforts are not successful I appreciate the effort you put into this project. You did it for your wife. You also did it for everyone else. That is great !!
By the way, Annmask it is not absolutely neccessary to share alot of details if doing so proves to be difficult or if you don't wish to share some details with everyone. Many of us don't share alot of details. You could do what I did (see below) or post the information you wish to share with us somewhere else and post a link to it in your account. Participation is what is important around here. You did just fine with your post in the other thread.
Take care everyone.

[phil]

Sorry that I didnt get the link exact. Try again : http://signon.org/sign/fda-blocks-life-saving?source=c.url&r-by=603490

[schoonder]

http://southshorexpress.com/whitman-hanson/8831-couple-crusades-for-fda-approval-of-new-cancer-drug.html
A recent WSJ article reported new FDA programs are being implemented by FDA Commissioner Margaret Hamburg to make the approval process clearer and more predictable for applicants. In particular elements in need to be improved upon were:.

1 - Timelines are long.
2 - Lack of clarity and consistency from FDA reviewers.
3 - Early dialogues between company founders and the agency will help smooth the regulatory process.
4 - New emphasis on personalized medicine, a burgeoning area that needs an updated approval strategy.
5 - Expedited drug development pathway for drugs that target threatening conditions and illnesses for which there is no great alternative already on the market.
6 - Unpredictability at the FDA.

http://blogs.wsj.com/venturecapital/2011/10/05/fda-tries-to-mend-fences-with-med-tech-start-ups-investors/?mod=google_news_blog (http://blogs.wsj.com/venturecapital/2011/10/05/fda-tries-to-mend-fences-with-med-tech-start-ups-investors/?mod=google_news_blog)
Since drug approval process is so closely tied to actual life and death issues, one would think Congressional Oversight Committee would assure immediate steps be taken to set right what now has been accepted to be a somewhat flawed approval practice, especially with personalized (targeted) medicine.
The T-DM1 refuse to file letter send to Genentech IMO was a direct consequence of a number of the shortcomings identified in that WSJ article.
There’s little to no risk involved for FDA to retract that RTF decision and to instantly start with examination of T-DM1 phase II results. Roche and partners are currently conducting THREE large, international phase III trials in 1st, 2nd and 3rd line HER2+ MBC setting. These data results when they are filed, the 2nd line study expected to complete by mid-2012, will guide FDA in further decision making.

[phil]

Thanks Schoonder. Several of you have e-mailed me , asking if Lorraine got to switch tx. to Boston . She did NOT !! Genentech decided to close access , and with it the flexible tx. plan we had been working with in Va. That plan reduced the dose , because of low platelets. The cancer shrank more, and plats , still low , stabilized over the past 3 months. Now Genetech says Lorraine must meet " new criteria " for a " new study ". We were shocked. It had been portrayed as a simple roll-over , to a " maintenance " study. And Farber , the t dm-1 trial hospital in Boston, said that since it wasnt a full-fledged trial we could get treated by our doc at MGH. But she has never handled t dm-1. Partners IRB, which owns MGH, got involved. And we are stuck between the two. First the FDA, now this ! So, we have met w/ our Congressional reps, and had to go back to Va. this week. To stay in the flexible plan that Gen. and Va. worked out.
The south shore express article you included as a link, is better on paper than on-line. It was shrunk to fit on-line.
A few in-accuracies, and the pictures should be captioned : Before T DM-1 ( on adria last Nov., w/ our youngest grand dau. ), and After T DM-1 ( last week ). Those pics are worth the whole article !!
We fight on , David fighting Three Goliaths !

[phil]

Dont forget , Sign our petition : " FDA Blocks Life-Saving Drug ", Google it !

[ElaineM]

I signed the petition when it was first published.
Have you checked on the phase 3 T DM 1Theresa trial to see if it will be availabe near you? It is listed on clinicaltrials.gov. You can also get more information from the Genetech trials information line.
All the best to you and your wife.

[radiant]

Holy Cow Phil. I am just amazed at all you do! I read the southshore express article. It's terrific.

I am very sorry to hear that after all that work, you have to go back to VA. At the same time, it's INCREDIBLE news that Lorraine is down to ONE tumor. This is phenomonal news.

Very touching to read that you've both lost your 1st spouses to cancer, and are now in these shoes. Amazing that you two keep showing the power that "one" person can make.

Hugs,
kim

[schoonder]

Phil, here are some recent excerpts plus link to opinions on FDA controversial MBC drug rulings.

"Steven Walker on October 18, 2011 5:33 AM writes...
T-DM1 did not just get dusted off. It has been in human clinical testing for a quite a while, many experts think it is already proven to be better than herceptin alone, and FDA (meaning Dr. Pazdur) refused last year to agree to an Accelerated Approval pathway, which imposed at least a 2 year delay (nd probably longer) on its availability to women with Her-2 positive breast cancer. That action by FDA was part of the same ill-considered campaign by Pazdur to eliminate Accelerated Approval. I am often perplexed by the tendency among some to automatically think that everything happening in the cancer space is about nothing but greed. Our system is far from perfect on all sides of the process, but virtually all the drugs we have that actually do effectively treat cancer arrive as the result of major efforts by drug companies, regardless of whether they were originally conceived by the drug companies or others. Women with breast cancer need drugs like T-DM1 - which does not contain Taxotere and is much less toxic. The new results are from a trial that compared treatment with T-DM1 to treatment with a combination of Herceptin and Taxotere. T-DM1 - which is much less toxic, proved to be far more effective.
Why doesn't anyone on this blog ever seem to consider the undeniable fact that sometimes (actually fairly often) the FDA is screwing up by delaying or sometimes even denying, availability of new (and old) drugs that work to patients who need them. It happens a lot more than you might think. Do some real research on what has been happening with T-DM1 and you might find yourself wondering why this drug isn't already approved by the FDA.
Incidentally, the "shelved" drug referred to in the article isn't shelved at all. Taxotere is still very much approved, still used and still quite toxic. It is exactly the kind of drug researchers and Roche are trying to improve on, and Genentech/Roche developed a combination of Herceptin and DM1 to try to do that. T-DM1 is a good drugm, a fact now known for several years from well-run clinical trials. FDA has been sitting on it, requirirng more data and refusing to fast track it - which by the way drives up the development costs by hundreds of millions of dollars - which in turn drives up the cost of the drug after approval. Please check your facts. You are simply wrong on this one. Genentech/Roche are not the problem here. The FDA (yes, Dr. Pazdur again) is the problem.
Permalink to Comment (http://pipeline.corante.com/archives/2011/10/14/avastin_false_hope_for_metastatic_breast_cancer.ph p#574848)

18. (http://pipeline.corante.com/archives/2011/10/14/avastin_false_hope_for_metastatic_breast_cancer.ph p#575183)ex-Pfizerite on October 18, 2011 2:03 PM writes...
Steven, most commentators on this board are people who work in the pharma industry and although we may seem cynical it is because we have seen to many positive phase II clinical trials that were not replicable in a powered phase III clinical trial. After a while in the industry you only believe a fully powered clinical trial and then you believe that trial only after it has been confirmed with another fully powered clinical trial. I believe that anecdotal reports are only indicative of something to study more closely via cell based assay or an animal model before even beginning to think about a clinical trial.
I also think that most of the people who read and comment on this board, while they may have had projects that they feel were delayed by the FDA or felt that the FDA had moved the goal posts, would also agree that the FDA does a very difficult job in an exemplary manner and making personal attacks on a FDA official is uncalled for.

Permalink to Comment (http://pipeline.corante.com/archives/2011/10/14/avastin_false_hope_for_metastatic_breast_cancer.ph p#575183)

19. (http://pipeline.corante.com/archives/2011/10/14/avastin_false_hope_for_metastatic_breast_cancer.ph p#576005)Steven Walker on October 19, 2011 8:09 AM writes...
To ex-Pfizerite: I am well aware that exposing the warts of our drug development system from the real patient perspective is uncomfortable for people who work in it. Sorry to crash your club, but you can expect patients and their advocates to be doing a lot more of that. From where we sit, the system is a disaster. In my response above, I was adding factual information to the discussion because the person who posted didn't spend the extra ten minutes it would have taken to understand what T-DM1 really is, and to also understand that what Roche is doing is not based on greed, but rather on exactly what we want the drug compaies to be doing - trying to bring better, less toxic treatments for serious diseases to market. In the case of T-DM1, a great many people, including many in the industry and some former high-ranking FDAers were very troubled (some were outraged) by FDA's decision not to fast track the drug and grant it Accelerated Approval based on early trial data. I am perhaps the nation's most focused and heeded expert on Pazdur's policy missteps over the last ten years, and his delaying action for T-DM1 was part of his aggressive policy enforcement efforts. This is not about a personal attack on Pazdur, it is about educating the public regarding a powerful official's misguided policies and the enormous negative effects those policies have had on patients (my first and primary concern) and progress against cancer (a very close second). Pazdur aggressively sought the role of oncology czar, and he has executed that role as an effectively unsupervised regulator within CDER, but in reality separate from the management structure of CDER. Because of these facts, confirmed to me by senior officials in the agency, Pazdur and his senior staff are in fact the cause of the policy problems, policies which he has clearly explained as being "his policies." They are not the policies of some amorphous "FDA." So how would you have us, the patient community, explain what we think the problem is? Use the term "unnamed FDA official/" For us, this is real life and death stuff happening in real time, to us. So how would you have us explain it? The problem is in fact Dr. Pazdur and his senior staff. Sorry if that huirts his feelings, but a dying cancer patient doesn't have time for beating around the bush.
Again, sorry to crash your party, but when people post things that are simply wrong, or take strong positions in the public debate that run counter to the best interests of real patients who will be directly, adversely effected in real time - expect us to speak up.
We think we are very important stakeholders in this process, and with all due respect, we are tired of being told by the traditional insiders that we should just shut up."

http://pipeline.corante.com/archives/2011/10/14/avastin_false_hope_for_metastatic_breast_cancer.ph p

[Ellie F]

Phil
What level of platelets do they accept in order to have T-DM1?
Thanks
Ellie

[phil]

For trials they accept platelets of 100, 000 or above. Normal platelts run btween 150,000 to 400,000. The new " study " Gen. is talking about is an extension, for pts who have done well on the drug, and finished trials, access. So, they allow for a low of 75,000. Lorraine has gone as high as 71,000 this month. She is , as far as we know, the only candidate for this " new study " in Boston. Its a Catch 22, initiated by Gen. and running into a breuacratic repsonse so far from Partners / MGH IRB as well.
Al this highlights the difficulties thta Stage IV pts suffer in the current access/ trial system. I agree w/ Mr. Walker above . This particular T DM-1 issue started w/ Dr. Pazdurs team. I dont feel its personal when you call it as you see it. He and his team want to change the rules of Accelerated Approval. They didnt like what they saw w/ Avastin, and T DM-1 got caught up in that .
I think T DM-1 will be their stumbling block, its only out -performing very apporved drug out there. > And every researcher in the country was shocked when this FDA denied T DM-1. Its Phase II stats of 38% " complete or significant reduction " in Stage IV tumors speaks for itself. You dont havr to be " rocket scientist " to see the meaning of those no's. Those bloggers who defend this FDA about this drug are as " sincerely deluded " as Dr. Pazdur himself. Is that personal ? I dont think so, he sincerely believes that this Acc. Approval Program needs changing. He feels he is keeping Americans safe. Probably is , but not in this case. And he forgets tht we live in a Democracy, where he should not have this type of un-checked power.
Its not personal, when you deal w/ large Govt. agencies, or Global Corporations , its Very Impersonal. They all should spend more time in the chemo rooms, that might help change the System to be more " Stage IV Survivor Freindly " . Right now its causing un-necessary suffering and death. And its not just the FDA. Doctors are silent, Big Advocacy is silent, Big Media is silent Capitol Hill is silent. So we speak up !

[Ellie F]

Thanks for the information Phil.I am full of admiration for your campaigning. As you know we here in the UK get what's approved in the States much later, hence the battle for tykerb! I sincerely wish you every success getting T-DM1 approved. The ppl who make these decisions have no understanding of what it's like to walk the path of a cancer patient and struggle to get access to these newer less toxic therapies.
Ellie

[phil]

Roche/Genentech are starting trials in Europe w/ T DM-1 . Contact Gen. for details.
Re platelets, blood counts while on t dm-1. Someone e-mailed me about sharks liver oil. Lorraine has been taking it , along w/ chlorophyll, and 1,000mg. folic acid. The sharks liver oil seems to have helped her red counts actually ris to their highest level in 5 yrs. Not 100% sure, also, may be helping her plats stay in 60,000 -70,000 range. Along w/ dose reduction to 3.0, spacing tx's out more. We didnt start these supplements until after 7 full strength doses, and plats dropping below 75,000. Who knows if we had started earlier. All part of the learning process for those of you on t dm-1 , or starting.
That was the flexible plan for Lorraine in Va., that Genentech has now tried to change. And Partners IRB in Boston is delaying.

[phil]

I promised to keep you all up to date. We continue to be stone-walled by top execs at Genentech, and forced by them to travel to Va. Thats the truth, and they continue to try to blame Partners IRB. We know different. Some of you out there in Calif. will have to try and explain this co. to us. Brilliant researchers, callous businessmen, breakthroughs and blunders. Movie material. Dr. Mukherjee ought to write a book on Gen. alone. Has anyone ? Its definitely tragic for women/men asking for a chance , at single pt access.
Dont forget this FDA, the " 1,000 lb gorilla in the back of the room ", punishing Genentech for pushing a true miracle drug... and trials drag on, 5 years ...
Back to Lorraine. Just got back from Va. yesterday. Second dose working well, tumor markers at continued all time low, 14. Thanks to generous family, friends, it cost us " only " 400 $ , cheapest yet.
Seriously, this has only increased the strain on Lorraine. When will it end ? we fight on...
Oh, those new to this site, Genentech monitors it, so keep that in mind, speak your mind. I do.

[phil]

Correction, second dose since Gen. told Lorraine she would have to meet new " criteria " for anew " study ". 13th dose over past year, 7 at 3.6 strength, 5 at 3.0. Great results, great quality of life. Stable platelets.

[phil]

We had our FDA Blocks Life-saving Cancer Drug Rally. You can see Lorraine on youtube at " Boston FDA Rally ". We will get an edited version of all 5 Speakers out soon. One of the speakers , Peter , is in the Pertuzumab/T DM-1 / taxol trial. he is doing great ! Counts dropping w/ just afew doses. Definitely worth looking into. You may or may not get pert., but you do get t dm-1. The trial w/ T DM-1 and GDC0945 also worth alook, because you get T DM-1.
The Petition was delivered to Sec. Sebelius. We would like to continue growing the pet. until we get a meeting w/ Sec. Sebelius in DC.

[phil]

FYI, " Our Her2 Cancer Struggle " ( on FB), has posted the Video of our December " FDA Blocks Life-Saving Cancer Drug Rally " on Youtube. Please watch all 4 Parts, and pass the links on to your media,advocacy and political contacts ! We Are Not Going Away !!
Heres the link to Part 1: http://www.youtube.com/watch?v=JlsrTqwUL6g
And , yes , Lorraine is still being forced to travel to Va. to get Herceptin T DM-1. Even though she has no visible disease, and has been stable at 3.0, every 3 weeks, since last July !! At the highest levels of bureaucracy, Genentech , Partners, FDA, you have folks who see only " black or white, statistics, numbers, ". These Execs have No contact w/ pts in chemo rooms ..rules are rules etc. " Thats why we must Speak , and be Heard ! This is about Saving Patients Lives !!
We dedicate the Rally Videos to all those Stage IV Her2 + FIGHTERS who never got a chance to try this miracle drug in 2011 ! Approval Must Come in 2012 ! Speak up , make the calls , and FOLLOW UP w/ more calls !!

[schoonder]

Phil,
You did a fantastic job in showing how firmly the FDA is choked up with obsolete regulations that deal with approval process of lifesaving, state-of-the-art drugs and the consequences. Of course it goes much deeper than that, first with FDA Oversight Subcommittee failing to take any action to overturn this abominal ruling and secondly, a media more interested in reporting on an assassinated nuclear scientist, or a body found on grounds belonging to the queen of England, or Gingrich calling Romney a liar and the like.
The clock is ticking, HER2+ MBC patients are dying and nothing is being done to "expedite" approval of T-DM1.

[phil]

Lorraine made it to Boston ! She did it on her own ( Really a God Thing ! ), her platelets hit 80,000 this past Monday. had a CT Tuesday - No Detectable Disease ! T DM-1 in Boston today ! After 16 trips to Va.
I will never complain about Boston Rush Hour Traffic Again ! Her story is a Testimony to Flexible Treatment for Stage IV Fighters, she got some chances, extra time, dose reduction to 3.0. And her platelets slowly responded , lows were less low than before, like a step-ladder. They "took off " 2 weeks ago, rising 19,000 in 9 days, biggest jump ever, so she could get treated , it turns out, on the 19th. Since she was a young girl, her " lucky " no. has been : 19 !
Thanks to the clinical teams at Va., Genentech , she s made it so far. For those who havent seen her go to : http://www.youtube.com/watch?v=JlsrTqwUL6g.
This FDA needs to be Investigated. Genentech needs to give single pt. access to Stage IV Fighters who have exhausted all other options ! The Drug Works ! Shame On Them ! Lorraine is Living Proof : Flexible Informed Choice Treatment Saves Stage IV Lives !

[oregon]

Hi Phil,

I hope you wife can continue to get this treatment. I'm towards the end of my Herceptin treatments, I was stage 1B, had neo-adjunct therapy. If you don't mind, I have some questions for you about Herceptin. I am trying to get educated, because with being HER2+, I feel like it's difficult to truly move on. If your wife was ever on Herceptin, what side affects did she experience? I didn't even know about the new drug TDM-1, is it suppose to be a better drug than Herceptin? If so, why? How does one even know whether or not the Herceptin is working?

Thanks for getting back with me.

[phil]

Hi Oregon :
All her2 + pts should be GREATLY encouraged by the development of Herceptin T DM-1. It is the best tx. for Her2 + since the invention of original Herceptin. Researchers have told me they think it will largely replace Herceptin over the next few yrs. Because it takes a chemo, t dm-1, directly to the cancer cells only , and blows them up ! There is no other drug like it in the world ! We just need to get This FDA to stop obstructing it ! They can't deny it forever ! ( another yr at most ).
My wife took herceptin for yrs, without s/e. Some pts have cardiac s/e. About 20% of her2 pts respond well tojust herceptin alone. The other 80 % need chemo. Herceptin T DM-1 attaches a chemo directly to herceptin, much more powerful than other chemos. It does affect platelets in about 30 % of pts, but that issue will improve when its approved, w/ more flexible dosing.
As far as knowing if herc. is working : do your regular follow-up w/ scans , tumor marker tests. And you know your body best , any symptoms , tell your doc you want a check-up. Any other ? , let me know.

[schoonder]

There's a relatively small t-dm1 phase1 trial, focusing on candidates with normal, mild and moderate impaired liver function, about to get underway in Canada, France, Italy and Spain. Trial particulars can be found at:
http://www.clinicaltrials.gov/ct2/show/NCT01513083?term=emtansine&rank=1

[phil]

Thanks schoonder, I hope our mbrs in , Canada, Europe see this ! Further thoughts from above : T DM-1 has fewer s/e overall than other chemos, no hair loss, no mouth sores, because its the first targeted chemo. Pts have more enrgy , better quality of life than old-style chemos. Go to the Rally video on youtube , " FDA Blocks Life-Saving cancer Drug rally ". Look at Lorraine in Part 4, Peter in Part 3, how strong they are . They are ' Living Proof ", and a Living Indictment of This Post -2008 FDA !