Quest for an effective MS treatment stymied

Neurologist Rhonda Voskuhl has spent more than 10 years trying to get estriol to MS patients. She started with tests on mice.

My colleague Marie McCullough has a fascinating article in today’s paper about the challenges that UCLA neurologist Rhonda Voskuhl is facing trying to get an old drug tested for use as a treatment for women with multiple sclerosis.

Despite clear evidence that estriol – a form of the female hormone estrogen – was an effective treatment for MS, there is no money for a company to test a generic drug and it took Voskuhl more than a decade to get a major clinical trial going. She’s now leading a $5.6 million trial at 16 medical centers across the US, including one here at the University of Pennsylvania, but needs to raise another $20 million for a final study to get FDA approval of the treatment. So, some women with MS aren’t waiting for the studies.

Here’s what my colleague has to say about her story:

Imagine you’re a researcher and you discover an inexpensive, safe, side-effect-free drug that comes in a pill and may be effective against a dreadful, incurable disease.

Moreover, drugs already approved for that dreadful disease have significant side effects, require shots or intravenous drips, and cost so much money that uninsured patients can’t afford them.

Now imagine that you set out to get your inexpensive drug approved as a therapy for the dreadful disease – only to be stymied because your discovery won’t make you or anyone else rich.

That’s more or less the tale of neurologist Rhonda Voskuhl and her quest to get a generic drug called estriol approved as a therapy for women with multiple sclerosis, a neurological disorder that slowly destroys nerves in the brain, spinal cord and eyes. The story, in today’s Inquirer, may be an eye-opener for anyone who believes the business of drug development is about finding new therapies to battle illness.
Voskuhl, head of the Multiple Sclerosis program at the University of California, Los Angeles, has spent a decade so far testing estriol in an animal model of MS and in a small study of six women. The results were promising, so now she’s leading a $5.6 clinical trial that is recruiting 150 women at 16 medical centers, including the University of Pennsylvania.

Estriol is a form of estrogen that is made by the placenta and normally surges only during pregnancy. It has been sold as a menopausal therapy in Europe and Asia for 40 years. In the US, the Food and Drug Administration has never approved it, but it has long been available through “compounding” pharmacies that fill customized prescriptions.

Voskuhl struggled for years to cobble together money for the current trial, which is funded by the National MS Society, the National Institutes of Health, and the economic stimulus package. If the results, due in 2013, are positive, Voskuhl will need to amass an even more daunting sum, about $20 million, for the third and final trial that is required by the FDA to prove safety and effectiveness.

Here’s the rub: estriol is a generic drug. As such, its chemical composition cannot be patented. Composition patents are coveted because they guarantee exclusive marketing rights for 20 years. That’s 20 years during which a seller can set a whopping price without fear of competition because no one else is entitled to make the drug.

The bottom line is that it takes a lot of money to get an old drug approved for a new use, but it likely won’t make a lot of money. And finding a way to underwrite costly research that won’t necessarily have a big payoff is about as easy as finding a miracle cure.