Before an experimental drug can be tested in a clinical trial, researchers must first study whether it might be safe and effective. This is determined through a rigorous preclinical research process that involves testing experimental drugs in cell models and small animal models in the lab.
Why Does Preclinical Research Matter?
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Preclinical research is an important and essential part of the clinical trial process. It cannot guarantee that an experimental drug will work, but it can help ensure that the drugs that make it to clinical trials have scientific evidence behind them.
At every stage of the preclinical research process, researchers carefully check for potential risks and side effects. Though preclinical safety testing does not guarantee that experimental drugs will be safe, it helps lower the risk for clinical trial participants. |
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The Challenges of Preclinical Research
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Turning a scientific discovery into a potential treatment that can be tested in people can take years of preclinical research, and there are many challenges along the way.
ALS is a very complex disease that manifests differently in different people. Researchers have realized that there are multiple forms of ALS, and each form may develop and progress differently. They are now studying treatments for subtypes of ALS to better understand which approaches may work best for different groups of people. |
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A big challenge for ALS preclinical research is that an experimental drug that works in cells, mice, or other models often doesn’t work in people. Researchers must carefully study how potential therapies behave in increasingly complex systems.
Another challenge for preclinical research is funding, which is often more available for early discoveries or once a drug has shown enough promise to make investment less risky. To help bridge this gap, researchers need funding for their preclinical research. They may apply for grants and/or partner with biotech companies to test potential treatments. Multiple organizations, including our partners, also help raise money to fund this essential behind-the-scenes work.
Once researchers have identified, screened, and successfully tested a drug candidate in the lab, they submit their preclinical research data to the FDA. If the FDA reviews the data and allows the process to move forward, preparations begin for Phase 1 of the clinical trial.
Another challenge for preclinical research is funding, which is often more available for early discoveries or once a drug has shown enough promise to make investment less risky. To help bridge this gap, researchers need funding for their preclinical research. They may apply for grants and/or partner with biotech companies to test potential treatments. Multiple organizations, including our partners, also help raise money to fund this essential behind-the-scenes work.
Once researchers have identified, screened, and successfully tested a drug candidate in the lab, they submit their preclinical research data to the FDA. If the FDA reviews the data and allows the process to move forward, preparations begin for Phase 1 of the clinical trial.
Moving ALS Research Forward
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For everyone currently impacted by ALS, the research process is too slow. But ALS research progress is happening—and it is accelerating. There are now more clinical trials than ever before, and more drugs are in the development pipeline.
Preclinical research and basic research in the lab continue to build a foundational understanding of ALS that moves the entire field forward. Knowledge built over decades of learning about genetic ALS played a huge role in the development and approval of tofersen (Qalsody), the first gene-targeted therapy for ALS. Researchers are currently trying to develop more gene-targeted therapies as well as drugs for people whose ALS does not have a known genetic cause. |
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Current ALS research efforts include:
- Investigating why a normally useful protein called TDP-43 stops working properly and becomes toxic in the vast majority of all ALS cases
- Identifying and validating biomarkers (measurable indicators of what is happening inside the body) that could help measure drug effectiveness in clinical trials, diagnose ALS with a blood test, and more
- Developing newer models and methods for studying ALS
Every day, researchers in labs around the world are trying to unlock the mysteries of what causes ALS, how it progresses, and how it can be slowed, stopped, and prevented. These dedicated scientists work collaboratively, share data, present at conferences, and publish findings in a collective effort to end ALS.
To help with this effort, you can provide valuable information for ALS researchers by volunteering to enroll in a clinical trial, participate in observational studies, or donate tissue.
To help with this effort, you can provide valuable information for ALS researchers by volunteering to enroll in a clinical trial, participate in observational studies, or donate tissue.
Learn More
- This What is Preclinical Research? article from ALS TDI focuses on the importance of preclinical research and the steps involved.
- This Preclinical Research webinar hosted by NEALS features three experts who discuss the role of preclinical research and why collaboration is important.
- This Drug Development Process webpage from the FDA provides an overview of the entire process from scientific discovery through drug approval.
