ANA 2018: key highlights and news stories

Written by Sharon Salt, Editor

Find out more about the headlines from the 143rd Annual Meeting of the American Neurological Association (ANA2018; 21–23 October, Atlanta, GA, USA) below, which features top abstracts presented from the conference. Highlights include:

Gene therapy shows early signs of improvement in Parkinson’s disease

Investigators at the University of California San Francisco (CA, USA) and the University of Pittsburgh Medical Center (PA, USA) have reported on an early phase clinical trial of adenovirus-delivered gene therapy for the treatment of Parkinson’s disease that is resistant to standard medical treatment.

The presented study assessed the safety and tolerability, AADC activity and functional outcomes following administration of VY-AADC01, an AAV2 gene therapy encoding AADC.

The engineered virus was injected into the putamen in order to transfer DNA that encodes an enzyme called AADC, which enhances the synthesis of dopamine. In this preliminary study, improvements in mobility and reduction in abnormal movements were still measurable a year and a half out from treatment.

“Though the patient numbers are small and further, larger trials are required, this study provides highly encouraging support for the potential of gene therapy in Parkinson’s disease,” commented M Elizabeth Ross, Chair of the ANA’s Scientific Program Advisory Committee.

The authors concluded in their abstract that “VY-AADC01 was well tolerated in this ongoing Phase 1B study, with evidence for dose-dependent responses that are both clinically important and durable.”

Sources: Christine CW. VY-AADC01 in medically refractory Parkinson’s disease: safety and efficacy of a Phase 1B dose-ranging study 12 months and beyond. Program and Abstracts of the 143rd Annual Meeting of the American Neurological Association. Atlanta, GA, USA, 21–23 October 2018 (Abstract M300);

Chemical markers of Alzheimer’s may identify those at greatest risk

In an inherited form of Alzheimer’s disease for which the disease progression can be more accurately predicted, investigators at Washington University (MO, USA) have reported that phosphorylation on a particular amino acid of tau can be measured in patient spinal fluid, correlating with the distinct biological stages of the disease.

The researchers employed a mass spectrometry method to measure tau and phosphorylated tau peptides in CSF and assessed the rate of phosphorylation on four tau sites, and correlated these with neuroimaging measures in asymptomatic dominantly inherited Alzheimer’s disease mutation carriers.

The team believe that the phosphorylation status of the tau protein measured in the CSF appears to track distinct stages of the disease cascade in dominantly inherited Alzheimer’s disease, as measured both by estimated years to symptom onset as well as neuroimaging biomarkers that sequentially change as dementia approaches.

To conclude, the authors stated in their abstract that: “This work suggest that, in addition to neurofibrillary tau tangles, the protein tau may be involved in the Alzheimer’s disease pathological process throughout the disease process indicating the need for more work in understanding tau-directed therapeutics.”

Sources: Barthélemy N. Profiling Alzheimer disease stages in dominantly inherited Alzheimer disease using CSF tau phosphorylation isoforms: position matters. Program and Abstracts of the 143rd Annual Meeting of the American Neurological Association. Atlanta, GA, USA, 21–23 October 2018 (Abstract M150);

Stroke risk elevated up to 3 months after acute heart attack, study indicates

The weeks following a heart attack have been associated with an increased risk of stroke, but how long that increased risk lasts was unknown until now.

Researchers at Weill Cornell Medicine (NY, USA) studied the course of 1,750,000 people with acute myocardial infarction between 2008 to 2015 and selected randomly from Medicare beneficiaries aged 66 or older. After adjustment for other factors, they found that the risk of stroke was still elevated up to 3 months after acute heart attack, 2 months longer than was previously thought.

“These results will likely encourage more active and prolonged stroke prevention management of patients after a heart attack,” M Elizabeth Ross stated.

Sources: Merkler A. Duration of heightened ischemic stroke risk after acute myocardial infarction. Program and Abstracts of the 143rd Annual Meeting of the American Neurological Association. Atlanta, GA, USA, 21–23 October 2018 (Abstract M122);

Novel study reports long-term neurological effects of Ebola virus

Ebola virus causes severe brain symptoms, including seizures, meningitis, coma and stroke-like symptoms. However, the long-term effects on the brain among survivors are poorly understood.

In the presented study, investigators at the National Institutes of Health studied 20 Liberian volunteers who survived the West African Ebola epidemic in 2015, three of whom underwent intensive evaluations including brain imaging and nerve function testing.

Preliminary findings suggest that the Ebola virus disease survivors demonstrated residual effects of meningoencephalitis and small blood vessel disease presenting as persistent abnormalities on brain imaging, visual, motor and cognitive functions.

“Although more data including controls of non-Ebola sufferers matched from the same community are needed, these studies indicate that measures to protect the brain from the effects of Ebola virus disease will be critical to successful management of the acute infection,” concluded M Elizabeth Ross.

Sources: Billioux BJ. Case series of Ebola survivors from Liberia with neurological sequelae undergoing in-depth neurological evaluation at the National Institutes of Health. Program and Abstracts of the 143rd Annual Meeting of the American Neurological Association. Atlanta, GA, USA, 21–23 October 2018 (Abstract M191);