Dilemma: When 1 Drug Treats 2 Diseases

From the wallstreetjournal.com

A drug used to treat multiple sclerosis has also been shown to slow the progression of Lou Gehrig's disease in mice, a nonprofit biotechnology company plans to announce Tuesday. Now, scientists face a dilemma.

Many patients with Lou Gehrig's disease, a fatal disorder formally known as amyotrophic lateral sclerosis, may want to try the drug on their own—even though there's no evidence that it is safe or effective in patients with the disease.But scientists want patients to enroll in a clinical trial, one that specifically examines how the drug, Gilenya, works in people with ALS. They hope to launch that trial later this year.

"We want to make sure we are not doing any harm. We want to do the trials correctly and quickly," said Steven Perrin, president and chief executive officer of the ALS Therapy Development Institute, a Cambridge, Mass.-based group that is releasing preliminary findings on Gilenya.

When it comes to developing therapies for the disease, 'for ALS patients, yesterday is not fast enough,' says Steven Perrin, president and chief executive officer of the ALS Therapy Development Institute.Still, Dr. Perrin acknowledged the difficulty in asking ALS patients to hold off while a clinical trial is conducted on a drug that could potentially ease their condition. When it comes to developing therapies for the disease, "for ALS patients, yesterday is not fast enough," Dr. Perrin said.

This research dilemma is occurring with increasing frequency, as scientists and public-health officials look to already approved medicines to see if they can treat other illnesses.

Last week, a paper in the journal Science showed that skin-cancer drug bexarotene improved brain function in mice bred with Alzheimer's disease. Following publication, Alzheimer's patients asked their doctors to prescribe the drug off-label before researchers even launched a trial in people.

Doctors are allowed to prescribe approved medications off-label when they believe the patient may benefit, but many are reluctant because the drug may prove to be toxic in a different condition.There are many reasons why patients seek a drug off-label. Designing clinical trials and getting them approved by hospitals and the Food and Drug Administration takes time, and patients with deadly diseases often feel they cannot wait. Drug trials often cannot accept very ill patients, who often feel they have little to lose if they take the drug off-label. Most important, many patients object to the possibility of getting a placebo, which helps researchers determine if a drug is really working. 

Merit Cudkowicz, the co-principal investigator on the planned Gilenya trial in ALS, cited a previous effort to test a promising ALS drug. Published studies indicated that a widely used antibiotic, minocycline, appeared to slow disease progression in mice. But it took several years to enroll enough patients in a trial because so many people started taking the drug off-label, Dr. Cudkowicz said. In the end, the trial showed not only that minocycline didn't work in ALS, but that it accelerated the illness. "If we knew the drug worked, we wouldn't need the trial," said Dr. Cudkowicz, professor of neurology at Massachusetts General Hospital and Harvard Medical School.She said that Massachusetts General has started bringing in patients and their families to explain the importance of enrolling in a trial. They're also emphasizing the need for a placebo group in order to compare the drug's impact on patients' functional change and survival. But in response to patients' concerns, ALS doctors are trying to find ways to shorten the length of time on a placebo, and they sometimes promise that if the drug works, all patients in the trial will have access to the compound at a later stage .

Read the entire article on the wallstreetjournal.com