Drug Development


The drug development process takes years to complete its many steps and is costly – $1-3 billion dollars – to move a basic research idea all the way through clinical trials and FDA approval. Collaboration is essential. Researchers from all over the world in academia, government, non-profit and industry work together to move along the pathway of drug development as rapidly and efficiently as possible.

Steps in Drug Development


Basic Research: Scientists from every sector of research (government, academia, non-profit and industry) contribute to understanding the disease process, leading researchers to identify potential disease pathways as drug targets. This step is completed using a variety of experimental tools including biochemical analysis of proteins involved in the disease and model development using cell and animal models in the laboratory.

Drug Discovery: A variety of treatment approaches are in development for ALS including small molecule approaches, gene therapy approaches and antisense technologies. Researchers in industry and academia identify appropriate chemical entities based on pathways believed to be relevant to the disease. These chemical entities are selected from large screens of thousands of molecules. Those that appear to interact with the target of interest and can be identified by an appropriate read out are selected and further optimized. There are a variety of chemical properties that are required for a drug to be developed into a therapy. For ALS it is critical that the compound is able to penetrate the blood-brain barrier. Toxicity testing in cellular and animal models in the laboratory is also required. Academic and industry partnerships are extremely valuable and should be encouraged early in the development process to maximize the appropriate skills and expertise.

Preclinical: Researchers extensively test the drug to determine if it is safe enough for studies in humans, again using cell and animal models.

Clinical Development

Investigational New Drug (IND) Application is Submitted: The pharmaceutical/biotech companies provide the Food and Drug Administration (FDA) with an IND, which contains all preclinical testing results and plans for clinical testing, so that the FDA can determine if the drug is safe enough to move to human trials.

Clinical Trials: The candidate drug is assessed for safety and efficacy in three phases of clinical trials, usually beginning with tests in a small group of healthy volunteers and then moving into larger groups of patients.

New Drug Application (NDA)/ Biologics License Application (BLA) Submitted: The drug sponsor (company) submits a NDA or BLA to the FDA requesting approval to market the drug. These comprehensive applications contain the results and data analysis from the entire clinical development program and earlier preclinical testing, as well as the proposed labeling and manufacturing plans of the new drug.

FDA Review: The FDA reviews the NDA or BLA submission to determine if the drug can be approved for patients to use. They may solicit the opinion of an independent advisory committee.

FDA Approval: Following comprehensive reviews of the drug’s safety and efficacy, the FDA will either approve the drug or request additional studies. If the drug is approved, formulation, scale-up and manufacturing of the drug will get underway.

Ongoing Study of the Drug

Post-Approval Research and Monitoring: The sponsoring company monitors the drug as it is used in the larger population to capture any unexpected serious side effects. They also gather additional data, both through formal clinical studies and the collection of real-world evidence, which may reveal greater therapeutic potential in other indications, formulations, combinations, etc.

What The ALS Association is Doing

The Lawrence and Isabel Barnett Drug Development Program supports development of several different approaches to treatment of ALS including small molecule, gene therapy and stem cell approaches. The most promising initial targets for development include, but are not limited to, SOD1, C9orf72 and TDP43, as well as pathways that are involved in many neurodegenerative diseases, including inflammation. The Drug Development Contract program encourages academic groups to submit applications together with an industry partner ensuring that the appropriate expertise are in place. In many cases the industry partner does not receive funding from The Association, however they provide the academic partner with tools, expertise and resources for drug discovery.

The Association convenes workshops to bring the various stakeholders together to further drug development. For example, The ALS Association hosts a Drug Company Working Group meeting annually at the American Academy of Neurology meeting to highlight progress in the field and stimulate discussion amongst academic, government, industry and clinical partners. In addition, The Association works closely with the FDA to provide guidance to expedite clinical trials and treatments for ALS.

Why It Matters

Studies all over the world, many funded by The Association, are ongoing to develop effective treatments and cures for ALS.

Bringing an effective therapy to people living with ALS presents many technical challenges. Drug Development can take at least 10 years on average at a cost of $1-3 billion. The ALS Association is fully aware of these challenges and is working tirelessly to accelerate drug development of new treatments and ensure access to them. We strategically collaborate with the government, other non-profit organizations, pharmaceutical/biotech companies and academia to rapidly move forward research, patient care and pubic policy initiatives. People living with ALS are the center of everything we do to ensure that more effective treatments are developed.