Action Alert : Biogen Denying Cancer Drug to Fred Baron

Remember Fred Baron, the financier in the midst of the John Edwards-Rielle Hunter story?

He was diagnosed last week with multiple myeloma and given a week to live. There's a drug, Tsyabri, that's an exact match for him and the FDA has okayed it and requests have been made by Lance Armstrong, Bill Clinton, John Kerry and others to provide it and the CEO of Biotech is refusing.

Read his son's letter (Andrew is the founder of Rocketboom)and do what you can to add to the pressure. The drug is readily available, it's just not prescribed for myeloma.

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    I can't find why they are denying it (5.00 / 1) (#1)
    by nycstray on Tue Oct 14, 2008 at 06:41:45 PM EST
    It makes no sense if he's going to die anyway. If it works, all the better for everyone, right?

    Biogen spokeswoman gave a brief statement (5.00 / 1) (#23)
    by Iphie on Tue Oct 14, 2008 at 07:37:29 PM EST

    (Biogen) began treating the first patient in a clinical trial of Tysabri against the condition last month, says company spokeswoman Naomi Aoki. Frederick Baron's physician is an investigator on this study and well-familiar with its rules, but Baron isn't eligible to participate in the trial because he is too sick, Aoki says.

    Biogen is unwilling to make an exception for compassionate use because

    If something went wrong in an uncontrolled setting, it might lead to further restrictions on the drug's use among existing patients with MS and Crohn's disease, she says.

    "We concluded when we re-introduced Tysabri that we cannot make it available for uses other than the FDA-approved use, or for an FDA-approved clinical trial," Aoki says.

    I hate to have to admit it (5.00 / 1) (#53)
    by Montague on Tue Oct 14, 2008 at 10:34:19 PM EST
    but there is some validity in Biogen's claims.  Far be it from me to be on the side of a pharmaceutical company, but I've spent years working in a huge public university that runs hundreds of clinical trials.  Compassionate use sounds great, and I fight for it a lot when negotiating contracts with pharmaceuticals and device manufacturers, but even when something is used off-label and everybody says they understand that it's experimental and last-ditch, you might be surprised at how many families will attempt to sue after the patient's death.  They don't necessarily limit the suit to the company, either; generally the suits extend to the hospital, the university, the physicians, and more.

    In addition, the FDA may indeed restrict the drug's use further based on tort claims like this, or on negative outcomes.  Think of, say, Vioxx, which probably helped a lot of people who now no longer have access to it.  Was it good or bad overall?  Who knows?  But the flurry of lawsuits brought it down and now some people could be suffering because of that.  We all need to accept the responsibility of making choices about the medications we take.  

    It's a no-win situation, and all sides have some culpability in what's happening.


    s.o.p. (none / 0) (#82)
    by wystler on Fri Oct 17, 2008 at 11:53:31 AM EST
    They don't necessarily limit the suit to the company, either; generally the suits extend to the hospital, the university, the physicians, and more.

    The litigating attorney opens himself up to a malpractice claim if all parties are not named as defendants.


    This is how drugs are done (5.00 / 1) (#62)
    by samtaylor2 on Tue Oct 14, 2008 at 11:14:26 PM EST
    For MM.  Giving a cancer drug for someone with end stage MM, makes no sense if there is no data for it.  There are very good reasons drugs aren't given in these situations.  I am not saying that there isn't some alternative reason, but scientific process (as well as, what might be the humane process), might be being followed.  

    Thanks (none / 0) (#26)
    by nycstray on Tue Oct 14, 2008 at 07:45:51 PM EST
    Did you catch the son's comment there? If it's true, I don't see why they can't make a last ditch effort. Too sick to participate is interesting. You could argue the need for studies to see if it could effect such an advanced case . . . . especially if it carried no risk to further studies/use.

    What it comes down to, it seems, (5.00 / 3) (#29)
    by Iphie on Tue Oct 14, 2008 at 08:13:41 PM EST
    is that Biogen doesn't really want to know if there are other side-effects of the drug and therefore wants to limit the possibility of discovering those side-effects by limiting the number of people trying the drug. Aoki basically said as much -- Biogen's worried about something going wrong, not because of Baron's life, but because it might limit future sales of the medication.

    I cannot see any reasonable justification for denying him this drug -- it is clear his family understands the dangers, it should be their decision to make.


    There's a time to stop arguing ... (none / 0) (#27)
    by robrecht on Tue Oct 14, 2008 at 07:53:27 PM EST
    First, understand that MM destroys the patient's immune system.  Do you really think it's wise to give a severely immunocompromised patient a drug that has been linked to a deadly brain infection?  Unfortunately, it sounds like Fred Baron is going to die in a few days, and no miracle drug can stop it.  I feel for the desperation of the family.

    Er, if he's certain to die within days without it (none / 0) (#36)
    by gyrfalcon on Tue Oct 14, 2008 at 09:06:29 PM EST
    and he's OK with the chance of dying from a "deadly brain infection" instead...

    Which is why I said below I would probably err (5.00 / 2) (#49)
    by robrecht on Tue Oct 14, 2008 at 10:06:06 PM EST
    on the side of recklessness ... out of sympathy for the desperation of the family.  

    If he (or more likely his family) is OK with designing such an 11th hour desperation individual clinical trial, what's the harm?

    Well, scientifically, it would clearly be wrong to enroll him in a trial that is prescriptively designed with scientific inclusion and exclusion criteria to test a null hypothesis.  That would fundamentally violate good scientific method and not advance genuine hope for finding a potentially safe and efficacious treatment.

    Would another multiple myeloma patient with a realistic hope for successful treatment be excluded from participating in the scientifically designed clinical trial?

    How many more multiple myeloma patients would demand 11th-hour treatment with a still unproven therapy.  Severely immunocompromised patients at this stage in their disease are exceedingly poor subjects for this treatment.

    Would multiple sclerosis patients ultimately be denied this successful treatment becuase of additional cases of this deadly brain infection (PML: progressive multifocal leukoencephalopathy) eventually leading to the drug being withdrawn from the market?

    Like I say, I would probably err on the side of misguided reclessness, but I would recognize this for what it is--desperation of family members.  There is no miracle cure for Fred Baron at this stage.


    That's not science... (none / 0) (#43)
    by Fabian on Tue Oct 14, 2008 at 09:38:12 PM EST
    That's not a controlled study.  

    It's not even usable data.  There's no way to determine if any results were signal or noise.  

    I get mad enough when relevant data is deliberately manipulated.  Junk data is even worse.


    He's at the Mayo Clinic (5.00 / 1) (#50)
    by nycstray on Tue Oct 14, 2008 at 10:11:32 PM EST
    they've tested his blood with the treatment and it looked positive. I would think even if it wasn't an "official" study, the Mayo Clinic might find some science in it. There could be some valuable discovery there, or not. Nothing ventured, nothing gained. Why should a CEO trump the Mayo Clinic and the possible advancement of science? Not to mention, it might buy the guy some more time on this earth with his family. Heck, if it just bought the adjustment to acceptance for the family, that's got to be worth something.

    The in vitro test data are irrelevant (none / 0) (#52)
    by robrecht on Tue Oct 14, 2008 at 10:16:08 PM EST
    in this clinical case.  I'm sure the doctors at the Mayo Clinic realize this.  This isn't Hollywood or Star Trek.

    I realize it isn't Hollywood or ST (none / 0) (#55)
    by nycstray on Tue Oct 14, 2008 at 10:34:53 PM EST
    it's just a man's life and his family's.

    Why is the test data irrelevant and why have the doctors let it get this far? Why did the FDA give the go ahead? Why can't the guy be a test subject if the negative won't effect the future of the drug (unless something is discovered that could be dangerous to others)? Shouldn't experimentation in cases like this be encouraged over discouraged? They seem to know enough about the treatment to start trials for this exact same disease. The data they collect if he tries the treatment could be information down the road.


    Lot of questions ... (5.00 / 1) (#61)
    by robrecht on Tue Oct 14, 2008 at 11:03:59 PM EST
    In vitro data are irrelevant here because Fred Baron's clinical presentation is just way too far gone.  He's got a couple of days left.  The disease has already ravaged him.

    Why have the doctors let it get this far?  I'm sure they've done everything possible for him up until now.  There's no cure, but there are very good treatments for many people when the disease is caught early enough and when the patients are responsive to treatment.

    Or perhaps did you mean, why did the doctors let this request for compassionate use get this far?  I don't know that they did.  We have a post and letter by a desperate family member.  I haven't seen any advocacy on the part of his doctors.

    Why did the FDA give the go-ahead?  Was this a verbal or written communication from Andrew von Eschenbach?  To Fred Baron's doctor or to his son?  Has anyone seen the terms of this go-ahead?  I think we can only make assumptions here.  The FDA's governs the marketing of a drug by pharmaceutical companies, it does not make individual prescribing decisions.  It's correct for them to free the company in this case, but it's not their responsibility to make the more difficult decisions.

    There's already been discoveries about the lethal effects of this drug for others.  That's why there is such a strict prescribing and surveillance program.  The drug was previously removed from the market.  An end-stage multiple myeloma patient is one of the most vulnerable imaginable patients for these potential fatal effects.  The comapnies and the multiple sclerosis patients who benefit greatly from this drug have a legitmate interest in not risking that this very valuable drug be taken away from them again.

    If Tysabri eventually proves to be helpful for multiple myeloma patients, I suspect it will be most helpful for early stages of the disease or for patients who are in remission and who are not so severely immunocompromised.  But yes, of course, this one person experiment certainly could provide valuable data, aside from the many potential benefits to the patient and family.

    Should experimentation in cases like this be encouraged rather than discouraged?  Yes and no.  One needs to balance the benefits and risks for competing interests of different patient populations.  Such decisions can be quite a burden.  Not as easy as some would have us believe.  Sympathy for desperate family members does not always trump all other concerns and interests.


    Thanks :) (5.00 / 1) (#63)
    by nycstray on Tue Oct 14, 2008 at 11:28:58 PM EST
    Yeah, I meant why did they go ahead and even test. That makes me think he is/was borderline. There just seems to be a series of events that make me wonder what if . . . .

    Sickening (5.00 / 1) (#14)
    by mmc9431 on Tue Oct 14, 2008 at 07:11:31 PM EST
    What are sorry state of affairs. Even if the drug only adds mental comfort for him and his family (that they at least tried). Where's the harm? Where's the compassion? What does this say about the society we've created?

    Amen mmc... (none / 0) (#30)
    by kdog on Tue Oct 14, 2008 at 08:24:46 PM EST
    A dying human being should have access to whatever drugs they want...period.  

    What bugs me the most is, according to his son's letter, the drugs are sitting in the pharmacy in the hospital where Baron is being treated.  It makes no sense whatsoever not to respect his decision.  I mean the guy is dying for christs sake.  

    We've created a society void of common sense...sorry state of affairs indeed.


    Devil's advocate (5.00 / 1) (#56)
    by Montague on Tue Oct 14, 2008 at 10:44:04 PM EST
    We've created a society of people who fear death.  We cannot live forever and no miracle drug is ever going to change that.  I think we need to get a healthier attitude about dying.  Nothing is going to save this man, and nothing will save the indigent patient in another hospital with the same problem but for whom John Kerry isn't making efforts.  

    There is nothing fair about our medical system - well, we knew that already.  The financial burden on the system is largely related to our fear of death and prolonging life at any cost, for every extra second.  End of life care takes 10-12% of the total health care budget.  Not that I advocate in any way government or anyone forcing someone to die or to go without medical care.  But I do feel very strongly that we must to come to terms with the idea that we WILL die - and proceed accordingly.


    Peace of mind (5.00 / 1) (#68)
    by mmc9431 on Wed Oct 15, 2008 at 07:20:06 AM EST
    It has nothing to do with living forever. It has everything to do with compassion for the family. The drug can't hurt him. It can give the family a sense of closure. Having been the caretaker for two terminally ill member of my immediate family, I can understand their reasoning. There's always that lingering doubt that there were things you could have or should have done to help the patient live or die with dignity. I just see no harm in giving him whatever drug he wants at this stage.

    I'm not saying there is harm (5.00 / 1) (#77)
    by Montague on Wed Oct 15, 2008 at 11:15:17 PM EST
    in giving the drug and I am 100% in favor of pain alleviation drugs and methods. It's just that I can understand reasons for denying it as well.  It's really an extremely complicated situation and it plays out all over the U.S. daily.  It would play out in developing nations everywhere except those nations don't have access to our drugs, usually, so it's a moot point there.

    The problem I'm getting at is that our fear of death leads us to believe we can blame others for absolutely anything bad that happens to us - i.e., our litigious society (something else missing in developing nations).  And that in turn makes it harder to continue to develop new medications.  If you think you're going to be sued, you might decide not to go into that field.  

    The flip side of the non-litigious developing nations, of course, is that big pharma often uses those nations and their impoverished people as low-risk testing centers.


    I know where you're coming from.... (5.00 / 4) (#70)
    by kdog on Wed Oct 15, 2008 at 08:30:42 AM EST
    My old man basically chose not to go on living and embraced his own death...and I respected that decision, as difficult as it was.  And I agree some of the lengths people will go to to try and dodge death are troubling to me personally.

    But I'm a freedom first guy...Baron and/or his decision makers want to try this drug, the drug is sitting on a shelf right in the same hospital...let the poor dying slob have it, even as nothing more than a last dying wish.  If he wanted a big shot of heroin to go out with a smile, I'd say he should have the right to have that too.


    Like I said, devil's advocate (none / 0) (#78)
    by Montague on Wed Oct 15, 2008 at 11:19:35 PM EST
    Actually I'm very much in line with your thoughts.  Let him have it.  Let anyone have it.  And let them have all the available morphine, heroin, anything at all.

    The devil's advocate side is saying that I understand the fear of the part of the pharmaceutical.  Drug companies are pretty "evil" to my mind, but if we want to continue to have improved healthcare, they are a necessary evil, at least in a capitalist society.  


    He bares some responsibility (none / 0) (#80)
    by Davix on Thu Oct 16, 2008 at 10:31:44 AM EST
    Fred Baron bares some responsibility for the current state of affairs he finds himself in. He made his money suing companies like Biogen when their drugs have unintended consequences. It was his own actions that makes companies gun shy.

    I'm seeing ZERO information here. (5.00 / 2) (#17)
    by Fabian on Tue Oct 14, 2008 at 07:22:26 PM EST
    I had to go to wikipedia to look up the disease.

    Multiple Myeloma doesn't give you a week to live - it's the involvement of vital organs(usually kidney failure) and the failure of bone marrow function that dooms you.  A week to live means major organ failure as a secondary or tertiary effect of the disease, not the disease itself.

    Even a "Stage 3" diagnosis gives a life expectancy of 29 months.  One week?  That implies terminal and irreversible damage to the body.  The disease isn't the problem at that point, it's the cumulative damage.

    I know people love to lash out at Big Pharma and insurance companies, but sometimes they are in the right.  I can't be sure, but this may be one of those times.

    The family will do what they need to.  Best of luck to them.

    Very true (5.00 / 1) (#21)
    by robrecht on Tue Oct 14, 2008 at 07:35:19 PM EST
    Unfortunately, I cannot imagine that Fred Baron has any hope whatsoever for survival.  I've seen people die of multiple myeloma.  If you catch it early, and if the patient responds to treatment, s/he can live for years.  For example, Geraline Ferraro has MM.  Some are eligible for stem cell treatment, bone marrow transplants, or chemo and radiation, expect many blood transfusions, but once the disease has run its course, and you're at death's door, unfortunately there's nothing more that can be done.

    I dealt with a similar issue (none / 0) (#19)
    by nycstray on Tue Oct 14, 2008 at 07:29:15 PM EST
    and the failure of bone marrow function that dooms you.

    with a cancer kitty. Red blood cells iirc. I needed a drug that's given to chemo and dialysis patients to reverse it. It helped some, but he just didn't have enough strength left to really make it.

    The Mayo Clinic ran tests, so it may be that he is still at a point of reversal.


    I feel deep sympathy (5.00 / 3) (#31)
    by Steve M on Tue Oct 14, 2008 at 08:32:12 PM EST
    but I can't help thinking that if it were my own father in this situation, I really doubt Bill Clinton and John Kerry would be writing heartfelt pleas on his behalf.

    In fact, the striking aspect of this story is that a wealthy and influential person apparently isn't able to get whatever medical treatment he desires at the drop of a hat.

    It's not just the wealthy and influential (none / 0) (#32)
    by nycstray on Tue Oct 14, 2008 at 08:36:57 PM EST
    No heartfelt pleas for Joe Blow... (none / 0) (#33)
    by kdog on Tue Oct 14, 2008 at 08:41:46 PM EST
    thats for damn sure brother...I can't help but think if it was my loved one in that spot I'd be trying to talk some sense into the pharmacist 6 floors down and appeal to their humanity...that is where the drugs are.

    That or casing the joint...


    I don't agree (none / 0) (#39)
    by befuddledvoter on Tue Oct 14, 2008 at 09:11:03 PM EST
    I live in the Boston area and I cannot emphasize enough that stature does not seem to be a deal breaker around here when it comes to medical treatment.  I hate to read comments that turn this tragedy into a class struggle.  

    Really? (5.00 / 2) (#47)
    by Steve M on Tue Oct 14, 2008 at 09:44:54 PM EST
    John Kerry writes letters on behalf of every terminally ill person in Boston?  Now that's constituent service.

    John Kerry does not have to (none / 0) (#54)
    by befuddledvoter on Tue Oct 14, 2008 at 10:34:27 PM EST
    You can walk into any prime medical facility in Boston and find the indigent pauper getting the same treatment at the same facility as the most affluent CEO.  Just visit BI/Deaconess; Mass Gen; Brigham & Womens and you would know what I mean.  These hospitals have a cadre of interpreters available in too many languages to even count.

    I am talking (5.00 / 1) (#65)
    by Steve M on Wed Oct 15, 2008 at 01:28:32 AM EST
    about the situation described in this post, not some other situation.

    If you want to tell me the indigent pauper gets the exact same attention from prominent politicians in the situation described in this post, go ahead.


    Fred (5.00 / 1) (#71)
    by txpublicdefender on Wed Oct 15, 2008 at 10:18:16 AM EST
    My thoughts are certainly with the Baron family.  I met Fred Baron when he was the keynote speaker at my law school's Public Interest Law Conference.  He spoke quite eloquently about the work he has done over the years for Trial Lawyers for Public Justice on environmental issues, especially.  He has certainly made himself a very wealthy man through his plaintiffs litigation work, but he has also been a major crusader for social justice through high-powered targeted litigation on behalf of the disadvantaged.  He will certainly be missed.

    All that said, I have read a lot about the issue of compassionate use, including a very long and interesting New Yorker article on the topic several months ago.  It is much more complicated than it is being made out by a lot of posters here, and, based on what I have read, I cannot fault the company for its decision.  It is a reasonable one considering all the public health and scientific study issues that are in play.  (FWIW, I don't think the fear of being sued by Fred's family has anything at all to do with the company's decision.  It is about the adverse data and the study protocols.)

    Again, my thoughts are with Fred and his family during this very difficult time.

    Compassionate use (5.00 / 1) (#79)
    by Montague on Wed Oct 15, 2008 at 11:23:11 PM EST
    can be instituted without enrolling the subject into the routine protocol.  But it is very true that an adverse (and anecdotal) finding after giving it to a single patient as compassionate use could cause difficulty to the drug development process.

    I do agree that that, and not litigation, is Biogen's base fear.


    This all seems a little nutty. (5.00 / 2) (#74)
    by sarcastic unnamed one on Wed Oct 15, 2008 at 02:39:38 PM EST
    Fred's going to die - very soon - whether he gets this medicine or not.

    There is essentially no chance this drug would extend his life by even one minute.

    Basically, by allowing this experimental and hopeless use of the drug, Biogen would open themselves up to considerable risk of lawsuits and having the drug's approved uses restricted even further, thereby hurting many other people who, unlike Fred, could benefit from it.

    Biogen didn't create our drug approval process and legal system, however they are required to work within them.

    asdf (none / 0) (#83)
    by wystler on Fri Oct 17, 2008 at 11:59:10 AM EST
    Basically, by allowing this experimental and hopeless use of the drug, Biogen would open themselves up to considerable risk of lawsuits ...

    most specifically, shareholder rights suits against management if there's any hitch in the approval process. my guess is that the class action usual suspects are already laying their groundwork.

    (not suggesting that there's no validity to the suits.)


    This is just mind blowing. If the drug is (none / 0) (#2)
    by Teresa on Tue Oct 14, 2008 at 06:46:53 PM EST
    readily available, why can't his doctor just diagnose him with whatever it's made for? It's common for doctors to do that. I've had it done twice for me for minor problems.

    No wonder people hate big drug companies. The FDA says yes, the CEO says no. I almost wish something bad to happen to him.

    Something bad, (none / 0) (#7)
    by BrassTacks on Tue Oct 14, 2008 at 06:57:10 PM EST
    like being sued, and losing his company, because he gave a drug to someone who he knew it wouldn't help and might even hasten their death?  Or sued and lose his company because the drug didn't work to help this man?  

    Why do you think the company is refusing to give him the drug?  Just meanies?  


    It seems to me (5.00 / 3) (#11)
    by Steve M on Tue Oct 14, 2008 at 07:04:15 PM EST
    that letting someone die is a poor strategy for avoiding lawsuits.

    Oddly it can be the reverse (5.00 / 2) (#57)
    by Montague on Tue Oct 14, 2008 at 10:50:20 PM EST
    I know you were being ironic, but in fact most lawsuits come after someone HAS taken a drug, not after being refused a drug.  He takes the drug, he dies... bam! Family brings lawsuit.  It's not that uncommon.  And the sad part is that the lawsuit can prevent further research with that drug.

    I'm not advocating for the pharmaceuticals because lord knows they care more about getting rich than anything else, yet they remain a valid participant in drug development that has helped millions upon millions of people.


    You make it sound like (none / 0) (#42)
    by Fabian on Tue Oct 14, 2008 at 09:29:09 PM EST
    they are with holding a miracle drug.

    I listened to the PBS cancer special this weekend.  Most cancer treatment is a matter of degrees.  Some treatments may cause remission.  Some may give a few years.  Some may give weeks or months.

    The results depend on the particular cancer, the extent it has spread and the overall health of the patient.

    There is no magic bullet, no miracle drug.  


    Not at all (none / 0) (#46)
    by Steve M on Tue Oct 14, 2008 at 09:43:42 PM EST
    I am merely responding to someone who made the silly argument that perhaps they are letting him die simply to ensure that they don't get sued.

    Silly (none / 0) (#37)
    by gyrfalcon on Tue Oct 14, 2008 at 09:08:13 PM EST
    A few squiggles of a pen would indemnify the company.

    What they risk instead is being sued because their refusal, or even delay, killed the man.


    Not that simple (5.00 / 1) (#58)
    by Montague on Tue Oct 14, 2008 at 10:51:53 PM EST
    Just because a person signs an indemnification clause doesn't mean it can't be struck down later in court.

    They aren't going to be sued. (none / 0) (#41)
    by Fabian on Tue Oct 14, 2008 at 09:22:40 PM EST
    The disease is chronic and terminal.  The disease will kill him no matter what the company does.

    This is one of the cancers where you try for quality of life and extension of life, but you know the cancer will kill you in the end.


    bullfeathers (none / 0) (#84)
    by wystler on Fri Oct 17, 2008 at 12:02:02 PM EST
    those squiggles would do nothing to prevent a shareholder rights class action if the treatment appears to create a hitch in the FDA approval process. there's a serious capital investment involved in bringing a new drug to market.

    they don't want to mess up their (none / 0) (#35)
    by coigue on Tue Oct 14, 2008 at 09:06:24 PM EST
    clinical trial results.

    It's that simple, I bet.


    That's what it looks like to me too. (5.00 / 1) (#66)
    by DeborahNC on Wed Oct 15, 2008 at 03:46:19 AM EST
    Is the CEO (none / 0) (#3)
    by WS on Tue Oct 14, 2008 at 06:48:32 PM EST
    afraid of legal action if he accepts the offer and it doesn't work?  They should sign something that prevents this and I'm sure Baron would gladly accept.  

    I hope Fred Baron gets the medication he needs and becomes more compassionate to those less fortunate and with less money than he does (assuming he's an ultra-winger).  

    Wasn't he a big Edwards supporter? (none / 0) (#4)
    by Teresa on Tue Oct 14, 2008 at 06:51:44 PM EST
    They've already agreed to no liability for the company. This is just outrageous.

    Oh wait (none / 0) (#5)
    by WS on Tue Oct 14, 2008 at 06:54:25 PM EST
    I read the post wrong.  I thought he financed the investigation.  

    Is there any such thing? (none / 0) (#6)
    by BrassTacks on Tue Oct 14, 2008 at 06:55:04 PM EST
    Won't they be sued, no matter what is signed?  

    That's the only reason I can imagine that they would withhold it, unless they know it might kill him even more quickly.  

    Surely he can get the drug from Canada, or Europe.  


    He could die in 24-48hrs (5.00 / 2) (#10)
    by nycstray on Tue Oct 14, 2008 at 07:00:28 PM EST
    according to one report I read. The drug would have to kill him pretty damn fast. Something tells me the family would be fine with the risk.

    Heck, they should sue for the withholding since it seems like there's enough evidence it could help. Even the FDA (aside what I think of them) said go for it.


    If the drug has never been prescribed for this (none / 0) (#9)
    by BrassTacks on Tue Oct 14, 2008 at 06:59:27 PM EST
    use, how does anyone know that it is an "exact match'?  

    They started trials last (5.00 / 1) (#12)
    by nycstray on Tue Oct 14, 2008 at 07:06:30 PM EST

    Tysabri Trial for Multiple Myeloma Gets Under Way

    Elan and Biogen Idec have started a Phase I/II clinical trial of their multiple sclerosis drug Tysabri to treat multiple myeloma, a plasma cancer.

    The first multiple-myeloma patient took Tysabri (natalizumab) Sept. 4. The open-label, two-arm study will evaluate the safety and potential anti-tumor activity of the drug in adult patients with relapsed or refractory multiple myeloma.

    In the Phase I portion of the trial, a standard dose-escalation design will be used to assess the safety and tolerability of Tysabri in as many as 12 patients. In the Phase II portion, as many as 30 patients will be randomized to the tolerated doses identified in Phase I.

    Treatment cycles will be intravenous infusions of Tysabri once every 28 days for six months. After that, patients who have had a partial or complete response may continue to receive Tysabri once every 28 days until the disease begins to progress, according to a statement from the companies.

    Doesn't look all that promising (5.00 / 1) (#20)
    by Fabian on Tue Oct 14, 2008 at 07:29:47 PM EST
    to me, more like a shot in the dark in this particular case.  Better than nothing, but just.

    The basic treatment for MM is containment.  In this case, it appears there is no possibility of containment.  Then it's just managing the damage, not the disease.


    In May of last year, (5.00 / 1) (#25)
    by MKS on Tue Oct 14, 2008 at 07:45:06 PM EST
    the FDA approved a new treatment:  autologous stem cell transplant.  Basically, healthy stem cells are taken from the bone marrow of the patient, those cells are stored, then the patient receieves heavy doses of radation that kills almost all cells in the bone marrow.  Then the healthy cells are transplanted back into the marrow....

    This treatment is the most promising treatment in years for a disease that is basically incurable.  The problem is that many patients are ineligible for the treatment because they have already received radiation treatment and their stem cells are no longer healthy, an dno healthy ones were stored.....Mean survival time from date of diagnosis (according to Mayo Clinic stats I saw last summer) has been about 2 years--and that survival rate has remained steady for decades.....

    A regular contributor here who was planning on attending the Democratic Convention has Multiple Myeloma--as does Geraldine Ferraro.  Very terrible disease....


    would you please read the links (5.00 / 1) (#13)
    by Jeralyn on Tue Oct 14, 2008 at 07:07:31 PM EST
    I posted. It's all explained there.

    Right!!!! (5.00 / 1) (#28)
    by befuddledvoter on Tue Oct 14, 2008 at 07:57:19 PM EST
    Biogen Idec is walking distance from my house in Cambridge. Here, I think it is not a matter of liability; it is a matter of statistics. Biogen wants to cherry pick their candidates and are afraid that this patient is too far gone and will spoil their data..  That is just so unfair. I will forward this to the Cambridge City Council.  Wish I knew of this earlier.  I think they do not meet again until Monday.  

    unfair, but standard practice (5.00 / 1) (#38)
    by coigue on Tue Oct 14, 2008 at 09:09:46 PM EST
    its not cherry-picking, it's using an a priori  determined criteria for choosing test subjects.

    However, there's no reason they cannot give the guy the drug outside of the study. I don't know why someone doesn't just get on a plane to Canada for the meds.  


    It sounds personal. (none / 0) (#44)
    by Fabian on Tue Oct 14, 2008 at 09:40:19 PM EST
    This is a lousy argument from a public health perspective.  However, if there's a grudge against the company - it's a grand way to pay the company back in bad publicity.

    It's a decent argument from a scientific (none / 0) (#45)
    by coigue on Tue Oct 14, 2008 at 09:43:14 PM EST
    viewpoint, and I suspect that this is where they are coming from

    You're right (5.00 / 2) (#59)
    by Montague on Tue Oct 14, 2008 at 10:54:54 PM EST
    Pharmaceuticals do like to cherry-pick the research subjects.  That's one way of looking at it.  Another way, though, is that these protocols are very carefully designed to allow the collection of rigorous data, without which the study could wind up useless.  Drug development is a delicate, wary dance between academia and big business.  

    What data? (5.00 / 1) (#64)
    by BrassTacks on Tue Oct 14, 2008 at 11:34:49 PM EST
    He's not asking to be in the trial.  

    now underway, but the data would be there nevertheless.  

    Off-label? (none / 0) (#15)
    by miwome on Tue Oct 14, 2008 at 07:18:47 PM EST
    I'm somehow failing to understand why the physician couldn't prescribe the drug off-label. I mean, I still think it's ridiculous that the CEO is stonewalling, but it still seems odd, especially with the FDA on the side of the patient.

    I wondering if it has anything (5.00 / 1) (#16)
    by nycstray on Tue Oct 14, 2008 at 07:21:38 PM EST
    to do with the patent? Thinking about Monsanto and how they are with their engineered seeds . . . ?

    Not really an easy situation (none / 0) (#18)
    by robrecht on Tue Oct 14, 2008 at 07:23:06 PM EST
    The drug has been linked to a deadly brain infection and the company is trying to abide by a very strict conditions approved by the FDA for when and how it can be used for MS patients or MM patinets who fit the FDA approved study protocol.  The company's fear is not merely that of a lawsuit from the Baron family but they're balancing the needs of one desperately terminal patient with no real hope for survival with the needs of many others who currently have FDA-approved access to this very dangerous drug.  

    If I were the CEO of Biogen I would indeed try and find a way but it's silly to pretend that this is an easy case and many would probably correctly contend that such a use is actually reckless.


    Opening the floodgates? (none / 0) (#24)
    by Fabian on Tue Oct 14, 2008 at 07:43:09 PM EST
    That's a good reason not to.  The whole point of the testing process is to find out if any potential benefits outweigh the costs.  If there are serious suspected risks, is it ethical to start allowing treatment before testing is completed?  Do we want physicians to prescribe off label when it could put patients at risk?  

    Even if everyone involved says "We won't hold the pharma company responsible this time." then what about any other times?  I could see a suit arguing that the company had essentially encouraged off label use for the sake of profits.


    Baron & Budd (none / 0) (#22)
    by MKS on Tue Oct 14, 2008 at 07:35:30 PM EST
    is probably the pre-eminent firm handling mesothelioma and other asbestos cases.....Out of Texas, they handled the mesothelioma case of Admiral Zumwalt....

    They have handled some of the biggest mesothelioma cases here in California....

    Being a tort lawyer doesn't appear to be helping Baron....Terrible tragedy

    This is one of the days I wish I worked (none / 0) (#34)
    by scribe on Tue Oct 14, 2008 at 08:44:57 PM EST
    in the D.A.'s office.  That CEO would be before a grand jury by 9 AM tomorrow.

    Every now and again, one runs into cases of medical negligence so egregious that they cry out for more sanctions than civil litigation can provide.  A colleague had one once, where the pharmicist "misread" the scrip and the labels on the packaging and wrote the instructions on the patient's scrip to take 1 a day for a month, instead of 1 a month.  That patient died, grotesquely, from the drug on day 17 or so.  But doctors are almost never prosecuted for their wrongdoing.

    Frankly, I have heard of a case where the head of the FDA personally approved off-label use.  Ever. ("off-label use" is this: there are certain conditions for which drugs are approved to be used.  Using the drug for another condition is technically illegal and is called "off-label use".  One familiar example would be prescribing hunam growth hormone to pro athletes for "growth deficiency", when everyone knows it is to help them pump themselves up.  The former use is OK, the latter is not.)

    This strikes me as either the head of the company has serious vengeance issues against the ill person personally, his friends, or Democrats.  Or, as another commenter suggested, he does not want the results of the study to be messed up with some person likely to die being included in the sample space.

    It needs be noted that the reason they are pursuing this treatment is because the drug, tested in a tube against the ill person's disease, was found to be the exact match necessary for the ill person's disease.  All the "bad" cells were captured by the drug.

    Hold on there (5.00 / 1) (#60)
    by Montague on Tue Oct 14, 2008 at 10:57:01 PM EST
    There is NOTHING "technically illegal" about prescribing off-label.  Physicians do it on a daily basis throughout the country and are at no risk whatsoever of being charged with any illegal activity.

    But it puts patients at risk. (none / 0) (#67)
    by Fabian on Wed Oct 15, 2008 at 05:32:08 AM EST
    The whole point of trials and studies is to determine that a drug is both a safe and effective treatment for whatever it is prescribed for.  There's a long standing controversy over prescribing drugs for pediatric psychiatric disorders that are only cleared for use in adults.

    (The problem?  Not enough studies because it's much more difficult to run studies on minors due to legal and ethical rules.)


    Not necessarily (5.00 / 1) (#76)
    by Montague on Wed Oct 15, 2008 at 11:07:19 PM EST
    It is VERY common to prescribe off-label.  I think most people don't realize that.  Physicians are absolutely permitted to prescribe any medication approved by the FDA, and for any indication they want.  That said, a good physician keeps up with the latest literature and trials, has a sense of how things are going and how safe a medication might be in a population not (yet) approved, and balances that against potential help to the patient.

    I have to admit I don't know all the details of the Baron case but if the drug is on the shelves of the pharmacy and not merely for use in a clinical trial (this is based on what I've read here; I don't know if it is true), then I would think his physician could simply prescribe it.


    Oops. (none / 0) (#40)
    by scribe on Tue Oct 14, 2008 at 09:12:23 PM EST
    I wrote above:  

    Frankly, I have heard of a case where the head of the FDA personally approved off-label use.  Ever.

    It should read:  

    Frankly, I have never heard of a case where the head of the FDA personally approved off-label use.  Ever.

    Changes the meaning a bit.  Sorry.


    I think Rove's telling him what to do. (none / 0) (#72)
    by sarcastic unnamed one on Wed Oct 15, 2008 at 11:44:05 AM EST
    This strikes me as either the head of the company has serious vengeance issues against the ill person personally, his friends, or Democrats.

    Where is the negligence (none / 0) (#75)
    by standingup on Wed Oct 15, 2008 at 04:07:32 PM EST
    in this case?  I don't see how anyone could make a case for negligence here.  

    The problem appears to be (none / 0) (#48)
    by Abdul Abulbul Amir on Tue Oct 14, 2008 at 10:00:20 PM EST

    The problem appears to be that the FDA regulations will not allow Fred to get the drug unless part of a trial.  Then Fred's case cannot be excluded from the trial data.  

    If the drug is effective and Fred's death delays its availabity by years, then many more people will die.  

    Under those conditions the company is doing exactly the right thing, given the FDA regulationa as they are.

    FDA said it would be outside of the (5.00 / 1) (#51)
    by nycstray on Tue Oct 14, 2008 at 10:14:07 PM EST
    study according to the son linked above in the company's statement.

    Save Fred Baron (none / 0) (#73)
    by PLPickering on Wed Oct 15, 2008 at 01:00:32 PM EST
    Fred Baron deserves a fighting chance, just as you or I would if we were in his situation. What IS the problem with attempting to save a human life, especially in light of the fact that they have the FDA's approval for Fred to receive the medication. I don't know much about medicine, or about clinical trials, but I do know that there is something incredibly wrong with a world that gives corporate beings the power to decide who lives and who dies. Where have our morals gone? Frankly, Biogen stands only to gain from a successful trial of this treatment! What a windfall it could be for them - can't you see the headlines? Fred Baron is a wonderful man. Please do everything you can to help him - he has dedicated his life to helping others. Call Biogen and beg them to save Fred...  

    Fighting Chance? (none / 0) (#81)
    by Davix on Thu Oct 16, 2008 at 11:03:13 AM EST
    You have obviously no idea how MM works. If he has been only been given days to live the cancer has done so much damage that even if the drug killed the cancer the damage would still be there and he would die anyway.

    As to your comment "...he has dedicated his life to helping others."

    You need to rephrase it to say "..he has gotten very wealthy 'helping' others."