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called Touch; the program was instituted when Tysabri was brought back to the market in July 2006 after having been withdrawn in 2005 because of its link to a rare and often fatal brain infection called PML.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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Biogen Idec has turned down the requests of the family and high-profile friends of a dying patient at the Mayo Clinic to provide an emergency dose of Tysabri to try to combat the man’s cancer—an unapproved use of the drug. It’s a vivid example of the ethically treacherous zone that companies must traverse when patients request access to their drugs before they are approved as safe and effective by the FDA.

The case of Frederick Baron hit the blogosphere in a open letter to Biogen CEO James Mullen posted this morning by Baron’s son, Andrew. The letter grabbed instant attention (including from TechCrunch’s Michael Arrington, who wrote a scathing post about Biogen’s refusal), in part because Andrew Baron, CEO of RocketBoom, noted that celebrities including Lance Armstrong, former President Bill Clinton, U.S. Senators Ted Kennedy and John Kerry, among others, all personally called on Biogen to clear the way for their friend to get an emergency dose of Tysabri. Frederick Baron, 61, a famous trial attorney and prominent Democratic fund-raiser, has multiple myeloma, a deadly cancer of the bone marrow; Tysabri is only approved by the FDA for the treatment of multiple sclerosis and Crohn’s disease.

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.

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.

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