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Research for Alpha-1.

What goes on in the world of Alpha-1 research?

It takes years, even decades, to start and finish research for any new drug. Each drug goes through many stages and variations of tests ― or clinical trials. This process helps scientists find out how the drug helps treat Alpha-1 symptoms, how safe it is and how it should be given. And because Alpha-1 is so rare, it takes a long time to get enough patients in the clinical trials.

Take a look at the big picture of how new drugs get approved.


It all starts with the scientists. They look at the many ways Alpha-1 causes lung or liver damage and research different drugs that might work for one or more of those causes. After they decide which drug to study, the scientists work in the lab, often with animals at first. They understand how the drug works, how much is needed for it to work and what some of the side effects might be.

Clinical trial.

Once the drug is found to be safe in the lab, the next step is to test it in people. Each clinical trial is planned so the scientists know what to measure, what to look for and how patients respond.

Phase 1 starts with small groups of people who don’t have Alpha-1. The researchers make sure the medication is safe and find the right dose.

If the drug appears safe, Phase 2 tests larger groups of people with Alpha-1. The scientists make sure that the drug does what they thought it would do. They also learn about the drug side effects. Each test takes months to years.

In phases 3 and 4, more people with Alpha-1 are added to the study and the scientists continue to make sure the drug works safely. Any new drug has to pass all the phases before the U.S. Food and Drug Administration (FDA) reviews the drug for approval.

FDA approval.

If the drug passes all the tests, the results get sent to the FDA. The FDA carefully reviews the results and decides if the drug needs more testing or is ready for patients to take. If the drug is approved for Alpha-1, the drug company can make and sell it. Doctors can prescribe the drug and people with Alpha-1 can start using it.


As people with Alpha-1 use the drug, the FDA continues to monitor the drug for safety. Any serious adverse reactions are reported to the FDA.

Can I join a study? To learn about studies that are going on right now for Alpha-1, you can find a list on the Alpha-1 Foundation website.

If you have any questions about possible new drugs for Alpha-1, talk to your doctor. Your Alpha-1 Advocate Team is also here for you. Call us at 1-877-602-6726 when you need us.

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