Advocacy Program Urges Dystonia Patients to Make Their Mark

The recently launched “Make Your Mark” campaign supported by Merz Pharmaceuticals, celebrates the inspirational stories of people living with blepharospasm or cervical dystonia (CD, or spasmodic torticollis), and aims to raise awareness of these often debilitating conditions. The campaign features patient advocates who share their stories and offer information and encouragement to others dealing with the conditions.

Cervical dystonia and blepharospasm are relatively rare conditions that may be under- or misdiagnosed. Results of an online survey conducted by Harris Interactive on behalf of Merz Pharmaceuticals, show 55 percent of surveyed CD and blepharospasm patients, on average, reported that they experienced symptoms for one year or more before their conditions were diagnosed. Two thirds (66 percent) of surveyed patients, on average, say they were not diagnosed sooner due to a lack of knowledge about these conditions or they were misdiagnosed with another condition first.

According to William Ondo, MD, Professor of Neurology at Baylor College of Medicine in Houston, more attention to these conditions is needed in the public and medical spheres. “The first issue is identification of the disease,” Dr. Ondo stresses. Both affected individuals and physicians should also recognize that effective treatment is possible in the form of botulinum toxin therapy. Involuntary movements and pain “both can improve dramatically with appropriate treatment,” Dr. Ondo notes.

Affected individuals may not be aware that the field of treatment options has grown, Dr. Ondo says. There are now four botulinum toxin formulations available on the market, including three of the type A serotype, and there are some differences between those three formulations, he suggests. Among the type A serotype formulations is incobotulinumtoxin A (Xeomin), which is marketed by Merz. Patients who have sub-optimal response to or are dissatisfied with one therapy can attempt treatment with an alternative formulation, he says. Any clinician who does not provide botulinum toxin therapy should refer patients with focal dystonia to a neurologist who does, Dr. Ondo urges.

Any clinician who does not provide botulinum toxin therapy should refer patients with focal dystonia to a neurologist who does, Dr. Ondo urges. In addition to improvement of pain and involuntary movements, treatment may improve the quality of life of infected individuals, who report high levels of social isolation, depression, and psychological affects, he observes.

Carotid Stent Indication Expanded

The FDA has expanded the indication of RX Acculink carotid stent, allowing treatment of a new group of patients at risk of stroke due to clogged neck arteries.

In 2004, FDA approved the stent for patients with clogged carotid arteries who were at high risk of complications if they underwent carotid endarterectomy. This new action “expands the indication for use of the stent to include all patients with clogged carotid arteries who are at risk for stroke, not just those who are not good candidates for surgery,” according to the FDA.

The approval was based on a 10-year study of 2,502 patients at 119 clinical sites in the US and Canada. Patients were treated and followed for at least one year. The NIH helped to fund the study, which found that patients treated with RX Acculink had a similar combined rate of death, stroke, and myocardial infarction as those who underwent surgery.

As a condition of the approval, the manufacturer, Abbott Vascular, is required to conduct a postapproval study to follow new patients treated with RX Acculink for at least three years to confirm the results identified in the pre-market study. The post-approval study will also evaluate:

How patients age 80 years and older respond to treatment; Whether treatment success is affected by the physician's experience with the product; Whether patients who show symptoms of having a clogged carotid artery experience a different outcome than those who don't show any symptoms prior to treatment.

Lamictal XR Gets FDA Nod

The FDA approved Lamictal XR (lamotrigine) extended-release tablets for conversion to monotherapy in patients 13 years and older with partial seizures taking one anti-epileptic drug. The drug is currently approved as add-on treatment for partial seizures and primary generalized tonic-clonic seizures in patients in this age group. Approval is based on data from study LAM30055, an international, multicenter, historical control study evaluating 300mg per day and 250mg per day of Lamictal XR for conversion to monotherapy in patients 13 years and older with partial seizures taking one antiepileptic drug. “Of the 226 patients treated with Lamictal XR, eight patients reported 10 treatmentemergent serious adverse events, including one serious adverse event of rash,” according to the company, GlaxoSmithKline. Other serious adverse events were seizure related, trauma, neoplasm, upper gastrointestinal hemorrhage, pyrexia and respiratory failure. No deaths were reported during the study.

Tysabri European Label Update

Europe's Committee for Medicinal Products for Human Use has recommended a label update for Tysabri to include anti-JC virus antibody status as an additional risk factor for the MS drug.

According to a release from Elan, the addition of anti-JCV antibody status as a risk factor allows for a three-factor approach to risk stratification. Prior immunosuppressant therapy and Tysabri treatment duration are established risk factors previously included in the product labeling. This provides an opportunity to identify patients at lower or higher risk for the development of progressive multifocal leukoencephalopathy (PML).

Global turnover of Tysabri (natalizumab) climbed 20 percent to $349.4 million; at the end of March, some 58,400 patients were on therapy worldwide, according to a Pharma Times report.

Alemtuzumab Data Promising

Five-year patient data from a completed Phase II multiple sclerosis trial demonstrated that “nearly two-thirds of alemtuzumab treated patients remained free of clinically-active disease as much as four years after most patients received their last course of the investigational drug,” according to a release from Genzyme Corp., a subsidiary of Sanofi-Aventis Group.

Alemtuzumab was given to patients in two or three annual cycles of not more than five days per cycle, while high dose interferon beta-1a was given to patients three times per week, every week for three years. New study findings:

  • An estimated 65 percent of alemtuzumabtreated patients were free of clinically-active disease, compared to 27 percent of patients taking Rebif (p<0.0001). To be free of clinically-active disease, MS patients in the trial were both relapsefree and without a sustained increase in disability as measured by the Expanded Disability Status Scale (EDSS) through five years;
  • An estimated 72 percent of alemtuzumabtreated patients were relapse-free compared to 41 percent of patients taking interferon beta-1a.

Potential New AD Treatment

A new study to be released in the September 6 issue of the Journal of Alzheimer's Disease has demonstrated that oral administration of a cysteine protease inhibitor, E64d, reduced the build-up of beta-amyloid in the brains of animal models for Alzheimer's disease, and substantially improved memory deficit.

Cognitive Impairment and Preschoolers With Epilepsy

Cognitive impairment is evident early on in preschool children with epilepsy, consistent with results of similar studies in older children (Epilepsia, June 2). Age of onset of first seizure is a significant predictor of cognitive impairment according to the first study to evaluate cognitive impairment in children age three to six. The authors looked at a group consisting of 64 preschool children with active epilepsy to determine the frequency of cognitive impairment and the epilepsy-related factors contributing to the impairment. The study showed that the prevalence of epilepsy was roughly three per 1,000 children, which is consistent with prior studies. The authors based cognitive function on psychological evaluation, parental reports, and observations from daycare. “The intelligence quotient (IQ) was used to measure cognitive function; children were classified as having normal IQ (IQ over 70) or with mild (50- to 69), moderate (35 to 49), or profound (less than 34) intellectual disability,” according to a release from the authors. “In the preschool cohort, 50 percent of the children displayed normal cognitive function, 22 percent had mild cognitive impairment, and 28 percent showed moderate to severe intellectual disability.”

Medicaid Stops Paying for Certain Mistakes

Medicaid will no longer pay hospitals or doctors for about two dozen “never events,” including operations on the wrong body part and certain surgicalsite infections, the Health and Human Services Department announced. "These steps will encourage health professionals and hospitals to reduce preventable infections, and eliminate serious medical errors," Centers for Medicare and Medicaid Services Administrator Donald Berwick said in a statement.

According to the Kaiser Health Network, about 21 states have such a nonpayment policy. The 2010 federal health law, in effect, expands the ban nationwide, and this ruling gives states until July 2012 to implement it. It's believed the change will save about $35 million over the next five years.

Vermont Moving Toward Single-Payer System

Gov. Peter Shumlin signed a bill in late May laying the groundwork for the nation's first publicly financed health care system. A key section creates a state health benefits exchange, as mandated by new federal health care laws, that will offer coverage from the private sector, state-sponsored and multi-state plans, according to Reuters, and will include tax credits to make premiums affordable to the uninsured.

A five-member board that would set reimbursement rates for health care providers and streamline administration into a single, unified system would manage the exchange. Residents and small employers will be able to compare rates from the various plans and choose their coverage. The law is unlikely to go into effect until six years or more from now, according to CQ HealthBeat.

Group Finds Flaws in Medicare Payments

The National Academy of Sciences says Medicare uses imprecise, unreliable data to pay doctors and hospitals, with some getting less reimbursement money based on geographic location. The panel said the issue of paying doctors has “fundamental conceptual problems,” and the method of paying hospitals is so unrealistic that almost 40 percent of them have been reclassified into higher-paying areas, according to The New York Times. A 2007 report by the Government Accountability Office said physicians in many urban areas were likely to be underpaid and some physicians in rural areas were overpaid.

Dexpramipexole ALS Trial Underway

A Global phase III trial of dexpramipexole for ALS is underway, with enrollment of the first patient, Biogen Idec and Knopp Biosciences Announce. The EMPOWER study will evaluate the efficacy, safety, and pharmacokinetics of dexpramipexole 150mg twice daily compared to placebo. A total of 804 patients will be enrolled in the trial and randomized.

Vest Assists Mobility

A poncho-style vest designed with handles to assist in the transfer of the minimally- to non-ambulatory patient, the new SDS Transfer & Rehab Vest, is now available. It is available for use in any healthcare setting-hospitals, surgery centers, nursing homes, assisted living centers-as well as in-home use. For information: www.ChooseTheVest.com.

Online Vision Test Available

People concerned they may be visually impaired may log on to a free web service from NovaVision. The Online Neurological Vision Loss Test (www.helpforvisionloss.com/NVisionTest.html) is an interactive online test that provides immediate results, that patients are encouraged to discuss with their neurologist, ophthalmologist, optometrist, or physiatrist who can prescribe appropriate therapy, including possibly NovaVision's Vision Restoration Therapy or VRT.

AMA Presses for Healthcare Liability Reform

The Help Efficient, Accessible, Low Cost, Timely Healthcare (HEALTH) Act of 2011 has been introduced in the House of Representatives, and the AMA is campaigning for lawmakers to pass the bill (the comparable Senate bill S.218 is in committee). The bill “sets conditions for lawsuits arising from health care liability claims regarding health care goods or services or any medical product affecting interstate commerce.” Among provisions in the bill are a statute of limitations of three years after the date of manifestation of injury or one year after the claimant discovers the injury, with certain exceptions. Noneconomic damages would be capped at $250,000.

In support of the bill, the AMA cites a Harvard study showing that 40 percent of medical liability claims lack any evidence of either a medical error or patient injury. They also report that, even though 64 percent of claims against physicians that closed in 2009 were dropped, withdrawn or dismissed, each case cost an average of $26,000 to defend. For more information: www.ama assn.org/ama/pub/advocacy/getinvolved. page.