Potential ALS Drug Fails in Study

The AP (5/18) reports, "Teva Pharmaceutical Industries Ltd. said Monday its potential Lou Gehrig's disease treatment," talampanel, "failed to meet a key goal in a midstage study." The drug failed to meet its effectiveness goal in a study of 559 patients, although it did meet its safety goals.

AMA Morning Rounds: Lithium may not help patients with ALS

The AP (4/6, Cheng) reports, "Lithium doesn't help patients with ALS, or Lou Gehrig's disease, contrary to previous study results, new research says." For the "first trial to scientifically assess whether lithium works for Lou Gehrig's disease, doctors found it had no effect -- and stopped the study early because it seemed futile." Eighty-four "patients with Lou Gehrig's disease" were enrolled in the study, and after "nearly six months, researchers didn't see any difference: 22 of the 40 patients in the lithium group had gotten worse versus 20 of 44 patients in the placebo group." The Los Angeles Times (4/5, Maugh) "Booster Shots" blog and MedPage Today (4/5, Gever) also covered the story.

AMA Morning Rounds: FDA grants orphan drug status to potential ALS treatment.

The AP (3/10) reported, "Cytokinetics Inc. said Wednesday regulators have granted 'orphan drug' status to its potential" amyotrophic lateral sclerosis, or "Lou Gehrig's disease treatment." The company also "said it plans to start a mid-stage clinical trial in the first half of this year to test the drug's effectiveness." Reuters (3/10, Sengupta) noted that the drug is called CK-2017357.

AMA Morning Rounds: Bristol-Myers Squibb, Allergan to jointly develop drug to treat neuropathic pain

The AP (3/3) reported that "Bristol-Myers Squibb and Allergan will jointly attempt to develop a drug to treat chronic pain from tissue damage, the drugmakers said." According to the AP, "the drug is said to be ready for mid-stage human trials as a treatment for neuropathic pain, or chronic pain usually accompanied by tissue injury. It is common in people with arthritis, cancer, and nerve damage."

The Orange County Business Journal (3/3) reported that "Bristol-Myers gains the rights to develop, make and market the compound," currently known as AGN-209323, "for just about all uses except for those related to the eye, which Allergan retains." Allergan will receive "an initial payment of $40 million and potential payments of up to $373 million if the compound is successful." Reuters (3/3) and the Wall Street Journal (3/3, Becker) also covered the story.

AMA Morning Rounds: H1N1 Hits Children with Neuromuscular Disorders

USA Today (2/18, Sternberg) reports that "thousands of children and adolescents with neuromuscular disorders, asthma and other conditions...are suffering consequences of H1N1 that will linger long after the 2009/2010 swine flu pandemic ends." Although "no one knows exactly why flu has such a devastating effect on" these children, "the evidence has been mounting since late 2005. Researchers at Children's Hospital of Philadelphia "reported that, even in typical flu years, children with these ailments were six times more likely than other children to develop severe complications from flu."

Paraspinal Mapping Superior to Imaging for Diagnosing Stenosis

A recent JAMA commentary by AANEM member Dr. Andy Haig, notes that while most surgeons use imaging to diagnose stenosis and make surgical recommendations, recent studies have shed doubt on the ability of imaging to confirm a diagnosis of stenosis. In contrast, new data has shown that paraspinal mapping can reliably identify patients with clinical stenosis. Haig says there are three key steps that need to be taken:“Find and treat what is not stenosis, define and treat the effects of stenosis, and treat presumed stenosis without a definitive diagnosis. Failing all of these steps, a positive diagnosis is an important consideration before surgery,” said Haig.

“I hope this commentary can begin a shift in the way some of these topics are considered and researched. Patients don’t present with stenosis; they present with back pain. As physicians, our guidelines should reflect that we diagnose and treat patients who have symptoms that help us make a diagnosis and treatment plan,” Haig concluded.

The commentary written was published in the January 6, 2010, issue of JAMA. The article was co-author by Christy Tomkins, PhD.

AMA Morning Rounds: Early data for ALS drug candidate shows improved muscle function

The AP (12/10) reports, "Sangamo Biosciences Inc. said Wednesday that early data from a clinical trial shows its Lou Gehrig's disease drug candidate," SB-509, "improved patients' muscle function." Patients injected with the drug "were about twice as likely to have improved muscular function," according to Sangamo. Specifically, "32 percent of patients who received SB-509 had improved muscle function, compared to 17 percent of patients who took standard treatments." The company "is also testing SB-509 as a treatment for diabetic neuropathy."

AMA Morning Rounds: Findings shed light on essential tremor

In the New York Times (12/8, D7) Personal Health column, Jane Brody writes, "Recent studies have begun to unravel the mysteries of essential tremor," a condition characterized by "uncontrollable shaking...that is not due to some other condition." According to Dr. Elan D. Louis, of Columbia University, the progress is marked by "discoveries in three areas -- the brain, clinical findings, and genetics and environment." Researchers have "analyzed and compared...the brains of normal individuals" to those of patients with essential tremor, finding that "this is probably a family of diseases." Abnormal findings include "degenerative changes in the cerebellum," and "a messy arrangement of neurofilaments." Meanwhile, "two environmental toxins have been found to be elevated in tremor patients: lead and a dietary chemical called harmane that occurs naturally in plants and animals."

AMA Morning Rounds: FDA lifts clinical hold on CytRx Lou Gehrig's treatment

The AP (12/2) reported that the FDA "lifted a nearly two-year suspension on development of" CytRx Corp.'s "arimoclomol as a treatment for Lou Gehrig's disease." The agency "halted arimoclomol studies" in January 2008, "citing the need for additional analysis from previously completed animal studies with arimoclomol."

Reuters (12/2) reported that the FDA accepted CytRx's revised trial protocol to review the safety and efficacy of the experimental drug at four times the dose previously studied. The FDA has granted the drug a fast-track review and orphan drug status to treat Lou Gehrig's disease.

AMA Morning Rounds: Neuromuscular Electrical Stimulation My Help Bed-Ridden Patients

The Los Angeles Times (9/21, Stein) "Booster Shots" blog reported, "The wasting away of muscle tissue can be a serious problem for people who are hospitalized and confined to bed due to a critical illness." But, a team at Johns Hopkins discovered, through meta-analysis, that "by putting patients through neuromuscular electrical stimulation and having them do simple exercises (some with the aid of devices), muscles can be shored up, speeding recuperation and getting people back on their feet." According to the paper published in Critical Care Medicine, the group also "developed a special walker that helps severely ill patients move around more easily, and with fewer helpers."