Old drug + potential new use = headache for pharma

Potential new uses for approved drugs crop up regularly. Most drug companies build their business plans around adding indications to new products; after all, the more patients who are eligible to use a drug, the more money that can be made. But new indications are usually offshoots to the old: A cancer drug is proven effective against another type of cancer, or an eye drug against another vision problem, or a blood thinner for a different cardiovascular use.

What if the potential new use were in an entirely different disease? And what if that other disease wasn't just serious, but devastating and incurable? That's what's happened in two cases over the past week. And as The Wall Street Journal reports, promising reports from animal studies are actually a pretty thorny problem.

Last week, Eisai's cancer drug Targretin showed promise at fighting one of the scariest diseases: Alzheimer's. The drug acted dramatically in mice, lowering levels of beta amyloid, a hallmark of the disease, and improving the rodents' memories. This week, the Novartis ($NVS) multiple sclerosis drug Gilenya slowed the progression of another frightening neurological problem--Lou Gehrig's disease--also in mice. In both cases, the drugs need trials in human patients not only to confirm that they'd work in people as they did in mice, but to determine dosage levels and identify potential side effects.

But patients with Alzheimer's and amyotrophic lateral sclerosis, as Lou Gehrig's disease is formally known, don't want to wait. Alzheimer's patients were calling their doctors to ask for prescriptions as soon as the Targretin study made headlines. As the WSJ notes, ALS patient advocates say "yesterday is not fast enough" for a new treatment. And U.S. law allows doctors to prescribe drugs for uses not yet approved by the FDA.

The problem is that off-label use can interfere with patient recruitment for real clinical trials. Not to mention the fact that human studies don't always confirm that therapies that worked in mice perform the same way in people. But well-designed and executed clinical trials take time, something that patients with these serious diseases don't necessarily have. "It's hard to do good quality research, and bad research hurts everyone," PatientsLikeMe's Jamie Heywood told the WSJ. "No matter what the companies do, someone will be upset."

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