MS Research Roundup: August 8, 2014

MS Research Roundup collects items of interest to multiple sclerosis researchers from around the Web. Send us your tips: tips@msdiscovery.org.

Myelin Drug Pipeline

Biogen Idec is sitting pretty with its reputation as one of the most successful pharmaceutical companies in treating MS. Boosted by its latest blockbuster dimethyl fumarate (Tecfidera), the company announced $2.4 billion in revenues in its quarterly financial report in July. With this news comes anticipation of results promised later this year from a phase 2 clinical trial for the treatment of acute optic neuritis using a novel monoclonal antibody, anti-LINGO-1 or BIIB033. A second phase 2 clinical trial of the experimental drug for multiple sclerosis is still recruiting patients, with results expected in 2016. Scientists, doctors, and patients aren’t the only ones interested in the outcomes. Investors are salivating over the potential for the drug to be a “game-changer” and “substantial proof of concept for the entire remyelination thesis,” with potential to generate over $1 billion in annual sales. Gilmore O’Neill, Biogen’s VP of global MS clinical development, told the Boston Business Journal that the company was proud it was discovered internally in a “hunting program” initiated to discover a compound capable of remyelination. Meanwhile, European regulators approved Biogen’s new variation on an old drug. The pegylated interferon β-1a (Plegridy) requires less frequent injections. U.S. Food and Drug Administration approval is pending, with a decision expected later this summer. (Boston Business Journal, Boston Globe, Wall Street Journal)

Expensive With Scant Evidence

The new disease-modifying therapies for MS are not cheap, but one rationale for the high prices is the expense of conducting large clinical trials to show that the drugs work better than other medicines. Not so for H.P. Acthar Gel (repository corticotropin injection, Questcor Pharmaceuticals), an obscure injectable from the 1950s. Approved for managing symptoms in MS and for 18 other conditions, it has become one of the most expensive drugs around (now about $32,000 for each five-dose vial). Despite scant evidence of effectiveness, Medicare’s tab for the anti-inflammatory jumped 20-fold from 2008 to 2012, reaching $141.5 million, reported the New York Times and ProPublica. For MS exacerbations, high-potency corticosteroids have become the standard of therapy for far less expense, according to one evidence review. Part of the reason Questcor can keep the prices so high is that the manufacturing of ACTH is categorized as a trade secret, making it difficult for others to create a generic version. The rest of the expense comes from aggressive marketing. The top 15 prescribers of Acthar accounted for 10% of Medicare prescriptions, and the top four were paid by Questcor either as promotional speakers, researchers, or both. The number one prescriber of Acthar in Medicare in 2012 was William Shaffer, M.D., of Greeley, Colorado, who said he gives speeches that draw on his experiences as both a neurologist and a patient with multiple sclerosis. The amounts paid to practitioners have not been disclosed, but all such payments by pharmaceutical and medical device companies will be public under the Physician Payment Sunshine Act. The database is expected to go live September 30, but medical groups are asking for a delay in launch until March 31. Some private insurance companies like Aetna and Cigna have restricted access to Acthar, but it’s unclear what path—if any—Medicare will take to lower the cost. (New York Times, ProPublica, The Hill)

Progressive MS Self-Portraits

Art reflects life for Patricia Lay-Dorsey. She was diagnosed with primary progressive MS in 1988 at age 45. Before then, she had run marathons. In her self-portrait series, titled “Falling Into Place,” she documents her life simply as it is. Photos of Lay-Dorsey opening containers with her mouth sit alongside photos of her smiling in her scooter. According to a story in the Huffington Post, Lay-Dorsey does not want her photos to foster feelings of pity or admiration in the eye of the beholder. Able-bodied photographers and others sometimes focus on the challenges of disability and forget about the person with those disabilities. By setting up her own photos of her going about her daily life, Lay-Dorsey captures the essence of her everyday life better than any other photographer. (Patricia Lay-Dorsey, Huffington Post)

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