Dying Cancer Patient Gets Tysabri After Biogen Idec’s Earlier Denial

Xconomy Boston — 

Frederick Baron, the prominent attorney dying of bone marrow cancer, has gotten his wish for a dose of Tysabri after all. The FDA and the Mayo Clinic found a legal basis to give him the drug in an emergency situation, according to a post on Dembot by his son, Andrew.

“We have every expectation of a positive result,” said Andrew Baron, the CEO of Rocketboom, in the web posting. “We cannot thank you enough for all of your thoughts and support.”

The decision by the FDA and the Mayo Clinic caps a frenzied week in which prominent friends of Baron, including former President Bill Clinton, cyclist Lance Armstrong, and U.S. Senators Ted Kennedy and John Kerry, all urged Biogen Idec to give Baron the medicine. Biogen refused, because Tysabri is unapproved for multiple myeloma, Baron’s disease, and it said that giving him the drug outside of a controlled clinical trial would create risks that the drug might be restricted for patients who need it for multiple sclerosis and Crohn’s disease.

Despite numerous phone calls from friends of Baron, a longtime Democratic fundraiser, and other concerned citizens, Biogen never re-considered its decision to provide the drug, says company spokeswoman Naomi Aoki. Yet as pressure mounted, the FDA informed Biogen late Wednesday that it was trying to resolve Baron’s situation directly with the Mayo Clinic. Aoki couldn’t be more specific on how the drug is being provided, or who is assuming liability in case something goes wrong.

Normally, with a marketed drug like Tysabri, physicians like those at the Mayo Clinic in Rochester, MN, are free to prescribe it for any disease, or any dose, however they see fit, under what is known as “off-label” use. The situation is more complicated with Tysabri, because it is currently available only under a strict prescribing and monitoring program called Touch; the program was instituted when Tysabri was brought back to the market in July 2006 after having been withdrawn in 2005 because of its link to a rare and often fatal brain infection called PML. Tysabri is now taken by an estimated 32,000 patients worldwide with multiple sclerosis and Crohn’s disease.

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 a letter Andrew Baron posted online Monday. 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, Aoki has said. Baron’s physician is an investigator on this study and well-familiar with its rules, Aoki says.

Liability has been a key question in this case all along. Andrew Baron, in an earlier comment on this site, said the FDA had assured Biogen that it would be protected from legal liability if it gave Frederick Baron the drug. “The FDA said that whatever happened in this particular case with my father, would not be counted against them. This is similar to a case which becomes expunged,” he wrote.

An FDA spokesman, in a written response to the Pharmalot blog, said it never offered Biogen a liability shield. “The FDA has provided no such assurance to the company. Our policy is to consider all available information when evaluating the safety and effectiveness of drugs,” the spokesman told Pharmalot. “We recognize and appreciate that expanded access programs involve less-controlled use of experimental treatments than the well-controlled environment of a clinical trial, and thus we would consider information that might be obtained in this instance within that context.”

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8 responses to “Dying Cancer Patient Gets Tysabri After Biogen Idec’s Earlier Denial”

  1. Kelly Martini says:

    I sincerely hope Mr. Baron recovers fully and then sue the hell out of Jim Mullen who delayed the approval process that made him suffer more pain from MM. He should ask for $100,000,000,000 damage because his time, his son’s time, Clinton’s time, Armstrong time, Harkin’s time and the FDA Chief’s time ain’t cheap…….

    Only in America!

  2. Mitch says:

    Kelly Martini’s comments are sadly off the mark. The approval process for Tysabri is strongly controlled by the FDA. This drug was released under strict FDA mandated RiskMAP guidelines. There are many off label uses out there for which Tysabri could be used and if a company like Biogen Idec succumbed to every request the public would be slamming it for trying to make money on unproven usage of a drug. Mayo Clinic in general has been very conservative in its recommendations for the treatment of MS with all of the biological agents so it is odd that they are in the limelight to be pushing for an off label usage for Tysabri when they are basically very skeptical as an institution for usual and approved usage of the drug. Furthermore they keep arms length away from the pharmaceutical industry except in publicity cases like this.

  3. NyHack says:

    Only in America is right…. what happened to the assurances from Mr Baron’s lawyer friends and his son that the FDA would hold nothing against the company if something went wrong? I’m so glad that friends in right places can jeopardize the long term future of a drug that is currently helping patients who have few treatment options. This is a great day in the battle against evil pharma.

    I can’t imagine being the family of anyone who enrolls in the randomized part of the Phase 2 trial in MM. How do you make the case that there is a chance they won’t recieve the study drug? Why are they less deserving than a rich lawyer with famous friends?

  4. Enfermera says:

    I’m on Tysabri for my MS. I went through a lot of red tape to obtain the drug, which Mr. Baron obviously has not. Would the average Joe with multiple myeloma get this medication? Can the average Joe get politicians and celebrities to call on his behalf? Hell no he can’t! I don’t have a problem with people who desperately need the medication getting it. What I have a problem with is some receiving more equal treatment than others.

  5. Sicko says:

    “Liability has been a key question in this case all along. Andrew Baron, in an earlier comment on this site, said the FDA had assured Biogen that it would be protected from legal liability if it gave Frederick Baron the drug. “The FDA said that whatever happened in this particular case with my father, would not be counted against them. This is similar to a case which becomes expunged,” he wrote.”

    vs. “The FDA has provided no such assurance to the company”

    Lawyers lie … no surprise. How do I know they are lying? Their lips are moving …

  6. mjl2112 says:

    It’s the rich and powerful get their way. As a couple eluded to already, would the every day average person be able to get this done? No, money and powerful friends talk. I credit Biogen Idec for sticking to their decision. It may be unpopular, but you have to think of the great good of the many here. What if he dies as a direct result of a weakened immune system and then being exposed to this drug that has shown very accute side effects to folks in such a situation. Then, even the press of such, especially if Biogen had said yes, could be enough to preven those that need it from having this drug prescribed. I wish no-one to die needlessly, but should one mans life be placed so far ahead of 32,000 and growing?

  7. DW says:

    I think EVERY American has the right to the very best health care, just like Congress, The House, and President do. The sad fact is, they don’t. If a regular everyday guy is dying from cancer, they’re not going to go out of their way to insure he gets the very best health care. Do you think they would do for a regular person what they did for this man? Don’t kid yourself. If you don’t have insurance and have a catastrophic illness like cancer, then you better tuck your head between your legs and kiss your butt good-bye cause you’re headed for the grave yard.