In pursuit of stopping the progression of Amyotrophic Lateral Sclerosis, researchers are studying the correlation between C- reactive Protein (CRP) Levels and the disease progression rate in ALS patients. CRP acts as a protein bio-marker for systemic inflammation; higher inflammation levels are believed to contribute towards the progression of ALS.
The lead investigator in the study Christian Lunetta said: “Understanding the role of inflammation in disease progression is critical as researchers continue to work on potential treatments for ALS. While additional research is necessary, our retrospective analysis of three separate data sets will help guide the development of investigational therapies, especially those, like NP001, focused on systemic inflammation.”
The use of CRP as a biomarker and diseases progression indicator in ALS patients was affirmed in the data analysis. The drug under investigation, NP001, uses CRP levels to monitor inflammation levels. It is in a confirmatory phase 2 study level.
Amyotrophic Lateral Sclerosis also known as ALS or Lou Gehrig’s disease often leads to the loss of voluntary muscle movement and eventual death. There is no known cure for the disease and its causes remain unknown. It has been proposed that there is promising evidence that links inflammation of the neurological system with prognosis of the disease.
It is common belief in the scientific world that in people with ALS, there are increased levels of activated inflammatory macrophages that trigger a release of factors in the central nervous system that damage motor neurons. Macrophages are a type of white blood cells found at sites of inflammation of infection. During inflammation, there is a need for a regulator, like NP001, to convert in the activated macrophages back to their normal state.
With over 400,000 ALS patients worldwide and no existing cure or meaningful treatment, the need for a new and effective therapy is great. The patients need treatment therapies that will slow the prognosis or stabilise the disease and improve their quality of life.
The confirmatory second phase 2 of the drug trial will be a “randomised, double-blind, placebo-controlled multi-centre study” of NP001 in people with ALS who can prove that they have systemic inflammation. The patients will receive either NP001 or placebo over a period of six months. The investigators will then monitor the changes from the baseline.
The confirmatory phase will be conducted in 20 centres across North America and had 87 participants. The main sponsors of the studied anticipate closure of enrolment into the study by quarter two of 2017 and complete data analysis in the first quarter of 2018.
In the first phase 2 trial, the research team found that patients with higher CRP levels at the beginning were less impaired after NP0001 treatment. They were doing so much better than even the patients with normal CRP levels in the drug treatment and the placebo groups.
NP001 is showing great promise for the therapy of ALS and other neurodegenerative diseases. From the data analysis from both phase 2 studies, NP001 could be on its way to approval as an ALS drug.