From the Clinic

Lora Clawson, M.S.N., C.R.N.P., manages Johns Hopkins’ ALS clinic. She also oversees its clinical trials.

Lora Clawson, M.S.N., C.R.N.P., manages Johns Hopkins’ ALS clinic. She also oversees its clinical trials.

How do I participate in ALS research?
Traditionally, assisting with research means becoming part of a clinical trial. Trials test drugs or new treatments for safety and effectiveness. Another way to help involves surveys. Hopkins, for example, offers several surveys and maintans the ALS CARE database, a collection of facts on individual patients’ treatment and care from diagnosis onward.

A third way to participate involves donating blood or tissue for scientific study. Several years ago, for example, after researchers discovered SOD1 mutations in people with certain familial forms of ALS, many sporadic ALS patients also offered samples of their blood for gene screening. Finding that none of them had the mutations was extremely important.

What should I expect when I enroll in a trial?
You’ll be given a written consent form to go over prior to entering a study. You’ll also talk with someone who understands the trial and who can answer questions. Then, the principal investigator (PI)—the one who’s put the study together—should sit down with you and review the details. You and the PI must sign the consent before any procedures begin. You can ask questions throughout the study. And, of course, you may withdraw from a research study at any time. Participants usually don’t pay for procedures related to a trial. Also, some studies provide parking and some may pay you for your participation, something that’s always discussed before you enroll.

How do I obtain the results of a study?
After patients finish participating in a study, it still takes time for researchers to assemble and interpret data. Later, when analysis is complete, the trial center sends out a sealed letter—one with a special ID number that reveals a participant’s particular arm of the study. It also explains study results and tells how the researchers interpret them.

In trials where initial results show a drug or treatment might be helpful, participants who’d been in the non-treatment arm of a study may be invited to join the next step, called an “open-label” phase. Then they receive the experimental drug or therapy.

Does the Center have any trials directed at improving breathing?
In ALS, effective breathing and clear airways are obviously of prime importance. One ongoing trial at the Center is for buspirone, a drug that may maximize existing respiratory nerve function. “Buspar” slightly eased breathing in mice, though it didn’t prolong life. And the Center’s also planning larger-scale studies that will try to shield respiratory motor neurons from ALS’s damage. Meanwhile, trials are starting on the ABI vest, a sleeveless, vibrating jacket that pulses and loosens airway mucus.

You can submit questions by e-mailing the editor, mcentofanti@jhmi.edu. See back page for mail addresses.

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