Anavex Life Sciences Corp (AVXL)

[XenaLives]

Good discussion of biomarkers -

https://www.alzdiscovery.org/news-room/blog/alzheimers-biomarkers-explained

Things are changing rapidly, and many of these changes will benefit Anavex by making trials simpler and more accurate.

WHAT BIOMARKERS DO WE NEED?
One of the biggest challenges facing physicians and researchers is the lack of qualified biomarkers for Alzheimer’s disease. Response and monitoring biomarkers are needed for use in clinical trials of innovative new drugs. As new targets emerge and drugs are developed and tested in human clinical trials, researchers need to know whether the drug reaches its intended target and how it is affecting patients. Too often, researchers are left to infer results using biomarkers that measure something “downstream,” or indirectly affected by, the target. For example, patient response to a drug intended to regenerate neurons lost to disease is measured using a hippocampal volume MRI. The theory is that new neurons will inevitably result in more visible brain tissue detectable by an MRI. But the researcher can’t know exactly how the drug is affecting individual neurons in a living patient’s brain because no validated response biomarker exists (several are, however, in development).

We also need more diagnostic tools. The beta-amyloid PET scans are useful, but they aren’t definitive. And while they are designed for Alzheimer’s disease, they don’t give a complete picture of the other underlying causes of the disease. Misdiagnosis and underdiagnosis are common across neurodegenerative diseases—including Parkinson’s, ALS, Huntington’s, frontotemporal dementia, dementia with Lewy bodies, primary progressive aphasia, and more—because of the lack of accurate biomarkers. PET scans are also expensive, limiting their widespread use. Less expensive tests—including blood and CSF markers—would allow doctors to more frequently screen patients for neurodegenerative diseases. Ideally, such tests would identify a disease in its early stages, before patients develop symptoms of dementia. This would enable both prevention and treatment. To this end, we are currently funding three teams working to develop blood tests for Alzheimer’s, two working on CSF tests for types of FTD, and another working on a CSF test for microRNAs.

The ADDF supported the early development of the Amyvid™ amyloid PET scan. Recognizing the power of neuroimaging, the ADDF has continued to expand our funding in this area. In keeping with our mission to find cures for dementias other than Alzheimer’s, we are funding neuroimaging projects for FTD and chronic traumatic encephalopathy (CTE). We are also funding the development of PET ligands to detect tau and epigenetic changes in the brain, and an MRI method for detecting beta-amyloid.

Over a decade ago, Columbia University professor Dr. Richard Mayeux wrote: “Biomarkers will . . . provide a dynamic and powerful approach to understanding the spectrum of neurological disease.” We agree and add that they will also enable us to prevent and treat it.