Subscribe"My sympathies go out to the Baron family.
This unfortunately is a common problem in the fight for cancer. I lost my brother to cancer and saw many of the same issues. On cause of this problem is that drug companies & researchers attempt to find cures that can be isolated and proved in formal FDA trials if they hope to get a drug approved as a treatment option (which can be worth billions ala Avastatin etc. and cost hundreds of millions to go through the process).
This applies for new drugs as well as for off-label or re-approval of an existing medication for cancer.
When CEO’s make the decision not to share drugs that show some promise with terminal cancer patients they are protecting their future options to submit these drugs as part of formal trials with controlled patient groups. Giving out the drug to terminal patients who request it, have no other options and are happy to experiment with anything that may offer hope can in the future prevent or disrupt their data resulting in problems conducting formal FDA trials. So some would say the CEO’s are doing their fiscal or moral responsibility to run the drug through the proper process that would enable it’s approval as a treatment for millions of patients rather then just the one patient showing the urgent need at that time.
(I would disagree with this, but the case could be made)
At the same time, the approach that doctors took to fighting the AIDS virus which was a series of experimental cocktails created outside of the FDA trial system created a number of treatment protocols that have made huge improvements in the life of afflicted patients. This type of process is not possible in Cancer research since even if you are participating in the FDA trial of an experimental treatment you are forbidden from combining it with other experimental treatments which would pollute their studies.
This approach to approving drugs has led to a slow down in innovation of one the front lines treatment protocols as drug companies are hesitant to led their drugs be combined & experimented on by doctors & patients who have no other hope and are more then willing to combine treatments to attempt a cocktail based treatment protocol.
Many leading cancer researchers have begun to push for a change in this process which would allow them more flexibility in combining different experimental treatments with terminal patients - but the costs & risks to the drug companies under the current FDA process is too great.
This is part of the reason why some of the best treatment protocols are being developed and advanced in Europe & around the world. Anti-angiogensis, Cox-2 inhibitors and a number of other treatment options (in the case of colorectal cancer which I was involved in researching for my brother) were all medications that had better options in Europe then in the United States and Canada.
The incremental, prove of efficacy approach of the FDA may seem like a good idea to create good data for proof of efficacy - but I would argue in the war against cancer, which continues to be a elusive and complicated disease we need to have the option to pull out all the stops and throw everything we have at it to be able to create more data about off label use of drugs, cocktails and experimental therapies in combination with the standard chemo treatments.
This is an FDA problem more so then the CEO of the drug company. They’ve created this environment and there are millions of terminal patients who are every year left in the same hopeless situation of having to beg for access to something that has shown some results in labs - but not yet made it through the arduous FDA approval process for treatment in Cancer."
“The safety and legality of the drug is highly contested. Tysabri is also know as Natalizumab, and its FDA file details a controversial history dating back four years.
According to the FDA, Tysabri was approved in 2004, and voluntarily withdrawn by manufacturer Biogen in 2005 when patients developed deadly brain infections during trials. Tysabri was approved again in January 2008, but only for Crohn's disease, and only as part of a strict program limiting risk of developing progressive multifocal leukoencephalopathy (PML), the deadly brain infection.
On August 26, 2008, two more cases of PML-- the deadly brain infection-- were uncovered in European patients using Tysabri to treat multiple sclerosis. The patients are European because use of the drug to treat multiple sclerosis is not allowed in the US.”
"Once the drug gets on market, the FDA cannot tell doctors what to do with that medication. Doctors may choose to use a drug that is not listed by the FDA for a certain condition. This is off-label use."
"Andrew - having been in the pharma industry for many years I have seen countless doctors prescribe drugs off-label for patients in need. It is common practice in oncology, psychiatry and on and on and on. Biogen had many problems with Tysabri as you probably know by now and the FDA pulled it off the market for a time for a particular indication - MS I want to say. There are NO circumstances where a representative of a drug manufacturer can 'approve' off label usage of one of their drugs but they routinely employ Medical Science Liaisons to discuss off label usage with physicians. While not 'recommending' off label usage they are able to legally speak with doctors about unusual applications and even recommend that a physican speak to another doctor that has used the drug off label. There is NO reason in the world why your dad's doctor has not (ESPECIALLY with the FDA's blessing) been speaking to other docs about using Tysabri in this case. Your dad's doc should have already administered the drug which is absolutely NO problem to acquire at ANY drug store. Please push your dad's doctor!! He/She is the one that is blocking progress for your dad at this juncture! I sincerely hope that this doctor is not fearful of liability, getting sued, getting in 'trouble' and that is why he has not proceeded.....that would be reprehensible. Good luck Andrew, my heart goes out to you and your family."
"The recommended dose of natalizumab for the treatment of relapsing-remitting multiple sclerosis is 300 mg administered intravenously every four weeks. Tysabri is administered intravenously by trained professionals at infusion centers."So, on one hand the drug is available at lower dosages (20 mg) quite readily, but larger dosages (300 mg) require participation in the TOUCH Prescribing Program with intravenous application at an approved facility.
"If the patient has been immunocompromised, giving him Tysabri would put him at extreme risk of PML, a brain virus that has a lethal history. While Biogen and Elan have developed a way to remove Tysabri from the system quickly, in an immune compromised patient, a patient that doesn’t have the natural ability to produce the antibodies necessary to check the virus, the PML would continue to develop and eventually kill the patient. Biogen and Elan are both struggling against a pr nightmare with Tysabri and PML.
On top of that, they have initiated trials specifically for multiple myeloma. Their hope is to carefully select patients who can benefit from Tysabri that also have a relatively intact immune system. Outside of the trial, if the patient dies, it will have negatively impact the trial results even though the person wasn’t in the trial under controlled conditions.
In the big picture, they may not want to risk drug development complications by providing it to a patient for whom it may have an unacceptable risk profile. If the drug works, think of the thousands of multiple myeloma patients who would suffer if development of the drug were slowed or stopped. Beyond those patients, another incidence of PML would affect the perception of Tysabri risk overall. The loss of Tysabri to patients could go well beyond the multiple myeloma indication which would be a catastrophe for Biogen, Elan and the many patients who suffer from diseases for which Tysabri helps."
"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."
"Something about this whole story is out of whack. The FDA approved clinical trials for treating multiple myeloma with Tysarbi over a month ago...
I am assuming that what Biogen determined was that his father was not suitable to be included in the clinical trials (either because he was so far progressed in his cancer or for other reasons). Given that clinical trials have to be conducted under very defined conditions, nothing unreasonable about that.
However, I don’t see why that would prevent off-label usage. It is, as others have pointed out, a widely used proceedure that doesn’t require any permission from the drug company (or for them to even know)."
“Frederick Baron’s physician hit upon the idea of treating him with Tysabri because samples of his cancer cells turned out to carry a molecular marker that Tysabri, an antibody drug, is designed to seek out and block, according to Andrew Baron’s letter. Biogen has done some animal tests that suggest Tysabri might be an effective treatment for multiple myeloma, and in fact, it 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.
In his letter, Andrew Baron wrote that FDA commissioner Andrew von Eschenbach ‘has granted special approval’ for his father to receive Tysabri, which essentially puts the ball in Biogen’s court. Under FDA rules, the company has discretion to give out the drug for such unapproved uses—what is known as ‘emergency use’ or ‘compassionate use’—but it chose not to, Aoki says. 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.
Still, I asked if this was a difficult decision, given the life and death nature of the matter. Andrew Baron’s letter says his father may have only a day or two left to live. ‘Of course, it’s a difficult decision for us. Our thoughts go out to the family. We don’t take this lightly, but we feel we have a responsibility to protect patients already on this drug who are benefiting from this drug,’ Aoki says.
When asked if legal liability is part of the company’s concern, Aoki referred back to her statement about protecting the interests of patients already on the drug.
This sort of ethical quandary has come up for other companies, most recently Plainfield, NJ-based PTC Therapeutics, which has refused to give a boy with muscular dystrophy an experimental medicine outside of the protocol of a clinical trial.”
"Biogen Idec Inc. reported late yesterday that another patient contracted a potentially fatal brain disease after taking the multiple sclerosis drug Tysabri, the third case since the biotechnology giant reintroduced the treatment two years ago. The news sent shares tumbling 12.1 percent in after-hours trading."
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posted by fungible at 8:41 AM on October 14, 2008