Why Clinical Trials Matter to Everyone

What are clinical trials and why do we need them?

Clinical trials are how we get our next generation of new cures.  Every single medical therapy — whether it's for cancer or Alzheimer's — has to be tested in humans. When your doctor says, take this once a day, twice a day, every other day, that is based on information that we got from a clinical trial. We need clinical trials to see whether or not discoveries that work in the lab actually do what they say they're going to do in people.

How do you know if a drug or vaccine is a suitable candidate for a clinical trial?

It all starts with an idea, such as how a particular receptor on a cell affects blood sugar metabolism. Researchers then design and test different molecules to interact with that receptor, potentially leading to a new drug for people with diabetes. That basic science is the key to all of the new treatments and cures that we have, and is almost universally paid for through the NIH. However, to ensure the molecule's safety and effectiveness in humans, clinical trials are necessary.

What happens during a clinical trial?

In the case of drug therapies, we do what's called a phase one trial, where we try it in humans to make sure it’s safe. We give it to a few healthy people and if they have no bad reactions to it, we go on to do what's called a phase two trial, where we test whether the drug does what it’s intended to do. Usually, in a phase two trial, some people get the drug and some people get a placebo — an inactive compound — and we compare the outcomes of both groups to learn how effective the drug is.

We work with the FDA to ensure that we're following all regulations and that we’re safely taking care of the volunteers. Once we demonstrate to the FDA that the drug is working and is still safe, we do a very large study. And at the end of the day, if it helps somebody feel better or function better or increase their chance of survival — if the benefits of taking it outweigh the risks — then the drug would get approved and you could use it in the clinic.

What kind of person volunteers to participate in a clinical trial?

Doing clinical trials absolutely makes me believe in the human spirit. What I've learned throughout my career is that humans are very kind and altruistic and want to help. I see it over and over — people going out of their way to participate in clinical trials and undergoing a lot of tests for things that might not even help them personally, but might help somebody else in the future. Without that genuine human kindness we would not have the therapies and treatments that we have with modern medicine today.

Who else makes clinical trials happen?

Everybody is involved. We have front desk staff, clinical research coordinators and clinical research nurses to facilitate whatever procedures are required for a particular visit. We have somebody to collect blood samples from participants, which are then processed by lab technicians. Clinicians, including physicians or nurse practitioners, are brought in to consult with patients and do physical exams. We have data analysts and statisticians who process all the information we collect. And then there are researchers like myself who analyze trial data and sort through whether or not the drug worked, prepare reports to the FDA or to the drug company, and write papers to communicate the results to the public.

All this is a big engine, or family, as I like to say, to make sure that we can get the best science out there to find out whether or not this new idea that started back in the lab years before actually does the thing that we want it to do.

We also have community advisors. They review the science we are planning to do and the science that is being conducted. They give us feedback on how the science will impact the community and how the community can engage around this new science, whether it be a clinical trial or new treatment.

What are some of the treatments that are now possible because of research at ACTRI?

If you can think of a disease, we probably have been studying it. And if you can think of a therapy, we probably helped get it over the finish line. Many drugs that started the clinical trial process here at the ACTRI are now used to treat everything from high blood pressure, heart failure and diabetes to Alzheimer’s, Parkinson's, ALS and cancer. During COVID, ACTRI did a lot of trials for treatments and vaccines. Every single HIV drug that has ever been used in a person was tested here at UC San Diego, we had a very big part to play in that.

How do funding cuts to NIH and other federal agencies affect clinical research?

If we're going to have the next generation of cures and treatments and new tests, we really need to continue to have support from the federal government. It takes a lot of money to get these drugs tested to make sure that they work the way they're supposed to.

ACTRI’s clinical research center is almost entirely paid for by NIH funds designated for Facilities and Administrative costs — also known as indirect costs. This provides the infrastructure so that we can see participants. It also pays for some of our staff and for all of our business and finance costs.

ACTRI also conducts studies in partnership with foundations focused on specific diseases, who support research into treatments for diabetes in kids, for example. We also work with companies that make vaccines, drugs, diagnostic tests and medical devices. But most companies don't do basic science during this process — that’s funded by the government.

Funding to the FDA is also under threat of being scaled back. I worry that with a smaller footprint, the agency might not have as much oversight around safety and efficacy as it had before. As a clinician prescribing therapy, I'm going to have more hesitancy to use some of these newer cures for which the risks and benefits in people aren’t fully known.

To learn more about volunteering for a clinical trial at ACTRI, visit https://clinicaltrials.ucsd.edu.