Clinical Trials are Fueling Medical Breakthroughs in Cancer

September 24, 2020

Clinical trials lead to new cancer treatments — and can give a patient access to effective drugs years before they’re made available to the public.

Clinical trials are essential in the development of new cancer therapies, yet many patients have misconceptions about clinical trials that may make them apprehensive to enroll in one.

On behalf of the Leukemia & Lymphoma Society, CURE® spoke with Dr. Susan M. O’Brien, a hematology oncologist at UCI Health in Orange, California, who is actively involved in clinical trials as director of the UCI Health Sue and Ralph Stern Center for Cancer Clinical Trials and Research. O’Brien discusses the importance of clinical trials for advancing the field of chronic lymphocytic leukemia (CLL) and offers advice for patients who may be interested in a trial but don’t know where to start.

CURE®: What are the different kinds of clinical trials, and why are they important?

O’Brien: Clinical trials come in a lot of different flavors. They can be trials of new agents, completely novel agents that don’t yet have FDA (Food and Drug Administration) approval. And they could be drugs that are next generation. So for example, we have a BTK (Bruton tyrosine kinase) inhibitor, but ... some company might think that they have a better BTK inhibitor. There can be trials looking at treatments for relapse disease; there (can) be trials looking at initial frontline (treatments).

I think a lot of people sometimes have the misconception and anxiety of enrolling in a clinical trial because they might be treated with a placebo. That’s very rare to see in a cancer trial. I don’t think I’ve ever seen (a CLL clinical trial) in patients who need treatment where there’s any kind of assignment to a placebo.

I think it’s important for a patient to consider a clinical trial, particularly with the novel agents if they’re available. ... Think about the fact that Imbruvica (ibrutinib) completely changed the landscape for CLL. It’s really been a total game changer. Consider how long it takes to get a drug FDA approved, and that one, by the way, went really fast. What that means is that people who are on the clinical trials actually have access to the drug years before that drug becomes FDA approved. So it can really get you access to novel, great drugs before they’re even out there for the rest of the community to use.

We also have to keep in mind that clinical trials are very important because (they’re) the only way we get new drugs. Every drug that is approved by the FDA had to be tested in usually more than one clinical trial. (The phase 1 trial determines) the appropriate dose, and the second trial looks at how well people respond. And then when we go to what we call phase 3; those are the randomized trials where a patient is randomly assigned to what’s usually considered the standard of care or to the newer drug.

There are different places along the way where we have to accumulate that trial data before it goes to the FDA for approval. That’s why people who happen to get on a great drug can have years of early access to it before it even gets approved.

Can you talk a bit more about the misconceptions that people have about clinical trials that may make them apprehensive?

I think some people have anxiety (and think), “You’re experimenting on me.” We don’t like to call it experimenting. But yes, you can think of it as the fact that you are testing out a drug that maybe has not been given to a lot of people.

If you’re (on a phase 1 trial), then yes, you are one of the first people ever to get that drug. If you’re on a phase 2 trial, you’re looking at response (to the drug): We’ve already defined the dose, we know what the dose is, we know what the (side) effects are. People did get (the drug) before you, and (it was deemed promising enough to move) forward to phase 2. By the time it gets to phase 3 — where you’re randomly comparing it to standard of care — there’s plenty of knowledge. (There’s often already publicly) presented data or published data from the prior clinical trials, so you are going into a trial to get ... a drug or combination that you (might not be able to) get outside the trial. But hopefully that’s something that will benefit you and then benefit society as a whole if (it) leads to subsequent approval of that drug.

How can patients learn more about clinical trials?

Of course the Leukemia & Lymphoma Society is a great (resource) that really helps to match up patients with trials in their area. This is one of the issues about clinical trials: It’s probably easier for patients in larger metropolitan areas to participate than it might be for someone who lives in a really rural area where they’re not that close to their medical center. They have to be able to get there.

There are certain trials where the larger centers will partner with smaller practitioners. ... In some of the cooperative groups, that hub-and-spoke model is how they operate. So for example, there are major academic centers, but then there are affiliated, much smaller centers that participate in those trials too. So cooperative group trials are sometimes easier for patients to have access to because of their design. And you don’t have to (go) to one of, say, the four centers that’s doing that trial. So that can be really helpful.

A number of different sites can be really helpful when trying to identify trials, particularly in a certain region, (because you probably don’t want) a patient who lives in Arizona (to have) to fly to New York on a weekly basis. I mean, that’s realistically not going to work. There are other trials at major centers where they have to go to the trial to enroll, but some of the treatment — particularly if it’s an oral agent that the patient takes themselves — can actually be administered at home.

So those kinds of trials, the patients do have some back and forth to the trial center, but much less than if it’s a drug that actually has to be delivered at the trial center. Some do, some don’t. There are a variety of possibilities, even for people who don’t live in highly populated metropolitan areas, to potentially (participate) in a clinical trial.


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