Overview

Biomarkers in Amyotrophic Lateral Sclerosis is focused on the biomarker identification and development in patients with Amyotrophic Lateral Sclerosis (ALS). This report focuses on the ALS space featured in the parent report, highlighting several biomarker targets under investigation and the progress that has been made in the industry. With regards to employing the use of biomarkers for Amyotrophic Lateral Sclerosis, Biomarkers in Amyotrophic Lateral Sclerosis captures market growth of biomarkers, advantages, disadvantages, and validation techniques.

Experts interviewed in this report include:

• Dr. Merit Ester Cudkowicz, Chief of Neurology at Massachusetts General Hospital
• Dr. Andreas Jeromin, CSO and President of Atlantic Biomarkers

Also available in this report is extensive survey data exclusively conducted for this report. Illustrated by 30 figures captured by an in-depth analysis, this section features insight into targets under investigation, challenges, advantages, and desired features of future diagnostic applications.

Finally, Insight Pharma Reports concludes this report with clinical trial and pipeline data featuring targets and products from over 30 companies working in Amyotrophic Lateral Sclerosis.

Executive Summary

Biomarkers have been a heavily studied topic of interest, and recently on the rise is the interest in neurodegenerative disorders, specifically Amyotrophic Lateral Sclerosis (ALS). This report covers extensive background information, highlights market growth and outlook, and features clinical trial and pipeline information.

After the introduction, Chapters 2 and 3 highlight background information relevant to the rest of this report and include definitions and elaborate examples of different types of biomarkers used in the clinic. Chapter 2 concludes with market growth, advantages of biomarkers, disadvantages of biomarkers, and validation techniques. Chapters 3 gives a brief overview of neurodegenerative disorders, also speaking to the market growth and rise in interest in biomarkers over the years.

Chapter 4 gives specifics on ALS, also known as Lou Gehrig’s disease. This chapter features definitions, symptoms, genetic markers, and current research regarding informative background information in this disease space. In this chapter, Dr. Merit Ester Cudkowicz, Chief of Neurology at Massachusetts General Hospital, discusses her progress on discovering biomarkers for therapeutic development and understanding the biology of ALS. This is further complemented by Dr. Andreas Jeromin, CSO and President of Atlantic Biomarkers. He provides insight on the diagnostic applications that biomarkers possess, and the company’s impact on healthcare.

Chapter 6 includes an elaborate survey analysis exclusively done for this report. Qualifying participants worked with neurobiomarkers, neurodiagnostics, or both. With over 30 survey figures depicting the general R&D group working in this space, this section provides information including: research demographics, targets under investigation, challenges, advantages, and desired features of future diagnostic applications.

Finally, Insight Pharma Reports also engineered a table of clinical and pipeline information related to Amyotrophic Lateral Sclerosis.

Table of Contents

Executive Summary

Chapter 1: The Focus of this Report

Chapter 2: Biomarkers and Their Clinical Utility
What are Biomarkers?
Advantages of Biomarkers
Clinical Endpoints vs. Surrogate Endpoints
Advantages of Biomarkers as Surrogate Endpoints
Disadvantages to Biomarkers as Surrogate Endpoints
How are Biomarkers Validated?

Chapter 3: Biomarkers in Neurodegenerative Disorders

Chapter 4: Amyotrophic Lateral Sclerosis
What is Amyotrophic Lateral Sclerosis?
Genetic Markers Identifying ALS
SOD1
C9orf72
Fused in Sarcoma (FUS)
TDP-43
Potential Biomarker Candidates for ALS
TDP-43
pNF-H
Cystatin C
Other Biomarkers
Interview with Dr. Merit Ester Cudkowicz
Research Background
ALS Research
Research Advantages and Challenges
ALS outlook

Chapter 5: Biomarkers for Diagnostics
Atlantic Biomarkers: Research Background
Mass-Spectrometry Assays for Biomarkers
Impact on Healthcare
Interview with Dr. Andreas Jeromin
Company Background
Neurobiomarker Research
Mass Spectrometry-Based Assays

Chapter 6:Survey Results

Chapter 7: Clinical Trials and Pipeline Information for Amyotrophic Lateral Sclerosis*

References

About Cambridge Healthtech Institute

List of Tables

Table 2.1: Clinical Applications of Biomarkers as Surrogate Endpoints

List of Figures

Figure 2.1: Growth of Interest in Biomarkers
Figure 2.2: Biomarker Presence in Diagnostic Development vs. Therapeutic Development
Figure 3.1: Comparison of Disease Categories
Figure 3.2: Growth of Interest in Biomarkers for Neurology
Figure 3.3: Growth of Interest in Biomarkers for Amyotrophic Lateral Sclerosis
Figure 6.1: How Would You Categorize Your Organization?
Figure 6.2: Which Neurodegenerative Condition are You Currently Studying?
Figure 6.3: Which Targets for Huntington's Disease (HD) are You Currently Studying?*
Figure 6.4: What is the Clinical Status of Your Target(s) for HD?*
Figure 6.5: When Do You Expect Your Targets for HD to Enter Clinical Trials?
Figure 6.6: When Do You Expect Your Target to be an Available Therapeutic for HD?
Figure 6.7: Which Targets for Amyotrophic Lateral Sclerosis (ALS) are You Currently Studying?*
Figure 6.8: What is the Clinical Status of Your Target(s) for ALS?*
Figure 6.9: When Do You Expect Your Targets for ALS to Enter Into Clinical Trials?
Figure 6.10: When Do You Expect Your Target to be an Available Therapeutic for ALS?
Figure 6.11: Which Targets for Multiple Sclerosis (MS) are You Currently Studying?*
Figure 6.12: What is the Clinical Status of Your Target(s) for MS?*
Figure 6.13: When Do You Expect Your Targets for MS to Enter Into Clinical Trials?
Figure 6.14: When Do You Expect Your Target for MS to be an Available Therapeutic?
Figure 6.15: Which Signatures for Alzheimer's Disease (AD) are You Currently Studying?*
Figure 6.16: What is the Clinical Status of Your Target(s) for AD?*
Figure 6.17: When Do You Expect Your Targets for AD to Enter Into Clinical Trials?
Figure 6.18: When Do You Expect Your Target to be an Available Therapeutic for AD?
Figure 6.19: Which Signatures for Parkinson's Disease (PD) are You Currently Studying?* 37
Figure 6.20: What is the Clinical Status of Your Target(s) for PD?*
Figure 6.21: When Do You Expect Your Targets for PD to Enter Into Clinical Trials?
Figure 6.22: When Do You Expect Your Targets for PD to be an Available Therapeutic?
Figure 6.23: How Would You Describe Your Line of Research?
Figure 6.24: With Respect to Neurodiagnostics, Which Diagnostic Tools are You Developing for Biomarker Signatures?*
Figure 6.25: With Respect to Neurodiagnostics, What are Challenges You Have Encountered with Your Diagnostic Development?*
Figure 6.26: With Respect to Biomarker Development, What Tools are You Using to Identify Biomarker Signatures?*
Figure 6.27: With Respect to Biomarker Development, What are Challenges You Have Encountered with Your Therapeutic Discovery/Development?*
Figure 6.28: With Respect to Biomarker Therapeutics, What Tools are You Using to Identify Biomarker Signatures?*
Figure 6.29: With Respect to Biomarker Therapeutics, What are Challenges You Have Encountered with Your Therapeutic Discovery/Development?*
Figure 6.30: Which Imaging Techniques do You Feel are the Most Beneficial for Studying the Effects of Neurodegenerative Diseases?*
Figure 6.31: Which Therapeutics do You Feel will be the Most Beneficial for Neurodegenerative Diseases?*