Research is the process scientists use to explain how our bodies work and why our bodies sometimes stop working well. Research eventually helps scientists understand enough about our bodies to treat or even cure what goes wrong. There are currently dozens of studies focused on myasthenia gravis (MG).1
Current areas of focus
Research helps build understanding into the causes of and treatments for myasthenia gravis. Some current types of MG research include:1,2
- Safety and effectiveness of new drugs for myasthenia gravis
- Whether exercise improves strength, fitness, and lung function
- How well some current treatments control muscle weakness
- Comparing different ways to lower how much steroid a person takes
- How well a single IVIG treatment works to treat a myasthenic crisis when delivered under the skin versus into the bloodstream
- Whether thymectomy combined with the steroid prednisone reduces symptoms better than taking prednisone alone
Studies may focus on how well certain drugs work in people with generalized MG or refractory MG. Some studies are only available in a few locations in the United States, while others recruit patients all over the world. For the most part, clinical trials are located in major cities.
Today’s newer treatments became available after dozens of people volunteered to try a new drug. These volunteers had to meet certain requirements, then report any side effects and whether their MG symptoms got better or not.
Clinical trials
Clinical trials are studies performed under controlled conditions. The goal of a clinical trial is to gather evidence (data) to find out whether a new drug or new treatment regimen is effective for someone with a specific condition or has higher efficacy than older drugs or treatments. These studies also look to determine what side effects a new medicine or device might have.
The U.S. Food and Drug Administration (FDA) approves and oversees all clinical trials conducted in the United States. In addition, individual hospitals oversee clinical trials through institutional review boards (IRBs), which review the way a study is designed to protect patients.
Clinical trials follow a protocol (plan) that outlines who can participate (such as people over age 18 who have generalized MG), what tests the person will receive and how often, and what type of data will be collected.
Clinical trials run in 4 stages:
- Phase I – A very small group of people take a new drug for the first time to look at safety, dosage range, and side effects. Less than 25 people may be involved in a Phase I trial.
- Phase II – A larger group takes the drug to see how well it works and if it is still safe. It varies, but a phase II trial may include just 50 to 300 people.
- Phase III – At this point, the drug is thought to be safe enough to give to large groups of people. Doctors continue to look for side effects, but Phase III trials focus more on how well the drug works. Sometimes the new drug will be compared to other drugs. Several hundred to a few thousand people may be taking the drug at this point.
- Phase IV – The drug is now marketed to the public and even larger groups of people are able to take the drug. The government and drug companies gather information about how the drug works in a larger, more diverse population. They also look for new or different side effects that happen with long-term use.
MG clinical trials usually have far fewer people participate than the average because there are so few people who have the condition.
If a drug or device appears to be unsafe at any stage, the clinical trial is stopped. The researchers involved then issue an explanation of what they believe went wrong. Often, any medical care related to the trial is provided to the patient at no cost because it is experimental.
Is medical research safe?
All medical research on humans must be approved and monitored by a group of scientists and doctors called an institutional review board (IRB). IRBs must follow rules for human safety set out by the FDA. In the United States, all IRBs that work on FDA-regulated studies must register with the FDA.
IRBs and these FDA regulations exist to minimize the risks to people who participate in clinical trials. However, it is the nature of experimental drugs and procedures that there will be both predictable and unpredictable risks.
Who pays for it?
There are a few main sources of funding for research into diseases. The federal government, nonprofit organizations, universities, and pharmaceutical companies all fund MG research. In the United States, the Myasthenia Gravis Foundation of America supports several research studies and funds training for doctors.3
How to get involved
Eventually, research will help scientists find better treatments, and hopefully, a cure. Until then, joining a clinical trial lets you contribute to the MG community by sharing your unique genes and responses to drugs. To find a study near you, talk to your doctor, the government’s clinical trial registry at ClinicalTrials.gov, or join the MG Patient Registry.3
