Novel PD Gene Therapy Agent Shows Promise

A novel, investigational gene therapy, NLX-P101 led to a mean 23.1 percent improvement (8.1 points) in off-medication Unified PD Rating Scale (UPDRS) motor score at the six-month study endpoint, compared to a mean 12.7 percent (4.7 points) improvement with placebo treatment, a new study shows. Published in an online-first edition of The Lancet Neurology, the “randomized, double- blind, sham surgery-controlled trial of 45 subjects with advanced PD met its primary outcome measurement for efficacy and demonstrated that NLX-P101 gene therapy was safe and well-tolerated over the six month blinded study period.”

Study authors also say that 50 percent of subjects treated with NLX-P101 attained “previously defined moderate-to-large clinically-meaningful symptom improvements (>/= 9 points in UPDRS), compared to just 14 percent of subjects who received a sham surgical treatment (p=0.03).” There were no serious adverse events associated to the gene therapy or surgical procedure reported.

FDA Approves Banzel (rufinamide) Oral Suspension

Banzel (rufinamide) Oral Suspension, 40mg/mL will be available for the adjunctive treatment of seizures associated with Lennox-Gastaut syndrome (LGS) in children four years and older and adults, thanks to recent FDA approval. The oral suspension formulation is bioequivalent to the currently marketed Banzel tablet formulation on a milligram per milligram basis and will be available for prescription use by late March 2011. The company hopes it will be an option for patients who prefer a liquid or have difficulty taking medication in tablet form.

Intuniv (guanfacine) Extended-Release Tablets Get Go- Ahead for Use as Treatment for ADHD

Once-daily Intuniv (guanfacine) extended-release tablets received FDA approval “as adjunctive therapy to stimulants for the treatment of attention deficit hyperactivity disorder (ADHD) in children and adolescents ages 6 to 17 as part of a total treatment program,” according to Shire. The approval is rooted in results from a nine-week placebo-controlled study of Intuniv given in combination with a stimulant, in children and adolescents with ADHD.

That study was multicenter, double-blind, randomized and included 455 patients experiencing a suboptimal response to stimulant treatment for ADHD. They received a morning or evening dose of Intuniv (1mg, 2mg, 3mg, or 4mg) or placebo in combination with their prescribed dose of a stimulant. “Clinicians using the ADHD-RS-IV, which includes both hyperactive/impulsive and inattentive subscales, reported significant reductions in total scores from baseline at end point for patients receiving Intuniv and stimulant when Intuniv was dosed either in the morning or evening, compared with placebo and stimulant,” they note.

The efficacy of Intuniv for more than nine weeks has not been systematically evaluated, and physicians who elect to use Intuniv for extended periods should periodically re-evaluate its long-term usefulness for the individual patient.

Vimpat Hits 100,000

Vimpat (lacosamide, UCB) has reached 100,000 patients since its launch for use as an add-on treatment for adults with partial-onset seizures, the company announced. In recent analyses, Vimpat showed substantial reduction in the incidence of seizures for up to five years: “a consistent longterm tolerability profile, and improvement of health-related quality of life assessments on a long-term basis,” UCB says. The maximum recommended daily dose for Vimpat in the US is 400mg/day.

Amyloid-beta Clearance Gene Found

Researchers have identified a new biomarker that could help them follow how well the immune system is able to clear the brain of amyloid beta, according to a study published in the Journal of Alzheimer's Disease.

The pilot study reveals how the immune gene MGAT3–essential in clearing amyloid beta–is expressed differently in different Alzheimer patients. The hope is that it will also lead to understanding which patients will react to therapy with vitamin D3 and curcumin, a chemical found in turmeric spice, both of which were shown in earlier research by this team from UCLA to help stimulate specific immune system cells to clear amyloid beta in a laboratory test.

For their study, researchers took blood samples from 20 AD patients and 20 healthy controls and then “isolated critical immune cells from the macrophages, which are responsible for gobbling up amyloid beta and other waste products.” The immune cells were incubated overnight with amyloid beta to test the cells' ability to "turn on" MGAT3. They also tested the effect of a synthetic form of curcumin on MGAT3 expression and the absorption of amyloid beta.

The researchers identified three groups of Alzheimer patients, based on the study results:

Type 0 patients. This group had very low expression of MGAT3 and very low absorption rates of amyloid beta.

Type I patients. This group also had low expression of MGAT3 and low amyloid beta absorption rates, but the strength of the MGAT3 message and the absorption of amyloid beta increased when researchers stimulated the macrophages with synthetic curcumin.

Type II patients. This group initially had high amyloid beta absorption rates, but when scientists added synthetic curcumin, MGAT3 expression lessened and absorption was reduced.

Scientists have followed 14 of the 20 AD patients for two years, and found that those who were Type 0 had a worse two-year prognosis regarding the loss of their ability to live independently than the other types of patients.

Furthermore, 45 percent of the AD patients were Type 0 in their MGAT3 immunity expression, while just 10 percent of the healthy controls were of this patient type. Vitamin D3 and curcumin and their effects in Type 0 patients have not yet been properly investigated.

Drinking Coffee a Boon for Women

Low or no coffee consumption is associated with an increased risk of stroke in women, according to data published the March 10 electronic edition of Stroke. Researchers prospectively followed of 34,670 women with no history of cardiovascular disease or cancer at baseline in 1997. The women's coffee consumption was assessed in 1997 using a self-administered questionnaire and incident stroke cases were determined by the Swedish Hospital Discharge Registry.

During a mean follow-up of 10.4 years, the authors discovered 1,680 stroke events, including 1,310 cerebral infarctions, 154 intracerebral hemorrhages, 79 subarachnoid hemorrhages, and 137 unspecified strokes. “After adjustment for other risk factors, coffee consumption was associated with a statistically significant lower risk of total stroke, cerebral infarction, and subarachnoid hemorrhage but not intracerebral hemorrhage,” they said in the study. The multivariable relative risks of total stroke across categories of coffee consumption (<1 cup/day, 1 to 2 cups/day, 3 to 4 cups/day, and. ≥5 cups/day) were 1.00, 0.78 (95% CI, 0.66 to 0.91), 0.75 (95% CI, 0.64 to 0.88), and 0.77 (95% CI, 0.63 to 0.92), respectively.

The link between drinking coffee and cerebral infarction was not changed by smoking status, body mass index, history of diabetes or hypertension, or alcohol intake.

Complete Response Letters Provided for Amyvid, Cladribine

According to the complete response letter provided to Eli Lilly and Company and Avid Radiopharmaceuticals for their New Drug Application (NDA) for Amyvid (florbetapir F 18 injection), FDA feels the companies need to establish a reader training program for market implementation that helps to ensure reader accuracy and consistency of interpretations of existing Amyvid scans. Amyvid is a Positron Emission Tomography (PET) imaging agent under investigation for the detection of beta-amyloid plaque in the brains of living patients.

Merck also received a complete response letter regarding Cladribine tablets, seeking an improved understanding of safety risks and the overall benefit/risk profile. The company says it will request an end-of-review meeting to clarify next steps.

GBM Tumor Therapy Endorsed

Benefits of treatment with a portable, non-invasive medical device designed for continuous use throughout the day by the patient outweigh its risks when it is used as monotherapy in place of standard medical therapy for supra-tentorial glioblastoma multiforme (GBM) tumors, an FDA advisory panel voted. Neurological Devices Advisory Panel of the Medical Devices Advisory Committee voted (7 yes; 3 no; 2 abstain) in favor of the device, NovoTTF-100A, which has been shown in vitro to slow and reverse tumor cell proliferation by inhibiting mitosis.

In trials, patients treated with the NovoTTF alone achieved a comparable overall survival to patients treated with the physician's choice of the best chemotherapy. Patients treated with the NovoTTF also had higher rates of progression free survival at six months and higher tumor response rates compared to chemotherapy treated patients (21 percent vs. 15 percent and 14 percent vs. 10 percent, respectively). NovoTTF- treated patients reported better quality of life scores and fewer side effects compared to patients treated with chemotherapy.

Neurological Disease Awareness:

MS Awareness Week, sponsored by the National MS Society, took place in March, and the Multiple Sclerosis Foundation declared it “MS Awareness Month.” In addition to educating the public, raising funds for research, and increasing support to patients, these awareness campaigns offer an opportunity for neurologists to revive educational efforts and support strategies for MS patients through the coming year.
For information, visit:
The National MS Society:
The Multiple Sclerosis Foundation:

Instituted by Congress last year, April is officially designated Parkinson's Awareness Month. The National Parkinson Foundation has launched a membership drive. The Parkinson's Unity Walk is scheduled for April 16 in Central Park in New York City.
For information, visit:
American Parkinson Disease Association: Parkinson's Unity Walk:
The National Parkinson Foundation:
The Michael J. Fox Foundation for Parkinson's Research:

More resources at our website: We've compiled resources related to MS and Parkinson's online at, including special programs and prescription savings offers as well as societies and support groups. Learn about ways to support your patients or download copies to distribute.