An overview of the different levels of clinical trials and their significance in providing patients with chronic lymphocytic leukemia with alternatives for treatment.
PUBLISHED March 25, 2019
Nicole Lamanna, M.D.: When deciding different options for therapy there are standard approaches, such as what Danise [Hoover] got with chemoimmunotherapy. But then when we talk about alternative treatments, the good news is there are many options of therapy for patients with chronic lymphocytic leukemia [CLL]. I think that’s one take-home point I want to say, because there are lots of different options for patients. Whether it’s traditional or nontraditional, there are lots of options. So that’s good. And in fact, your physician should help guide you with those options because it may be dependent on your age, your other medical problems, your kidney function, your heart disease, pulmonary disease. What might be relevant for one patient may not be completely relevant for another. And so your physician really should help guide you with treatment approaches, in general.
Now when we talk about clinical trials, not every medical center or every doctor’s office may have access to clinical trials. So let’s talk about clinical trials in general. Danise participated in a clinical trial and we’re so happy that she did. But let’s talk about what clinical trials mean, because I think that there’s a lot of misconception about what clinical trials are.
There are different levels of clinical trials. There are what we call phase 1, phase 2, and phase 3 trials. Early trials are typically the phase 1 studies. Generally, these are trials for which we don’t know if the drug works. We don’t know what dose of the drug might be appropriate yet in humans, so we base this on animal data. This is the first time we’re testing this drug in humans, and so we don’t know the side effects. We don’t know if it works. We don’t know the right dosing yet.
Typically, these phase 1 studies are offered to patients who might have gone through every standard treatment option there is for that particular disease. Really, those are patients who’ve exploited all traditional treatments by any measure, and their disease is getting more resistant. So this is oftentimes where a phase 1 study will be introduced to a patient, if they’re eligible.
Phase 2 and phase 3 studies are more advanced clinical trials. In those we know that the drug has some efficacy in the disease. We know the dosing, we know the side effects, and now we’re looking at how much better this particular drug is compared to other treatments that are already approved for this disease. These are advanced studies. When we talk about if they are compared, a phase III study may compare a drug [with] a traditional therapy, meaning a therapy that’s already approved for that disease. You’d either receive the standard therapy or the novel therapy.
We already know that the novel therapy works. Now we’re just trying to see if it’s better than the standard therapy. There are lots of different types of phase 2 and phase 3 studies, but these are advanced and we know that a drug’s going to have activity. I think patients often feel, when they’re approached about a clinical trial, that they might be a guinea pig. That comes up all the time when we talk about this with patients. They’re nervous that they are being experimented on. I think it takes the right team and the physicians to go over what that study really means to the patient, so patients know whether they’re getting active treatments and why, or why they’re being approached about a particular study. There are differences in studies, and of course we want the best for the patients too.
For some patients it may be difficult to participate in a clinical trial. In other words, their ability to go back and forth. Trials do require a lot more out of the patient and the team. There’s a lot more work involved potentially, depending upon the study. There might be more imaging, and certainly more blood tests. So it really depends on the nature of the study, and that’s unique to each study. It’s up to the team and the physician to go over this with the patient. But certainly, this is where the advances in the disease come from.
By being a part of a clinical trial you might be receiving, we hope, the next best treatment for that disease that will hopefully lead to an approval for the drug for everybody else with CLL, or with a different disease. And it may be advancing, because then this will lead to us considering how we can learn and strategize the next best treatment options for our patients with CLL. And so, that’s where clinical trials may be appropriate for that patient.