The Cure Starts Now

Translating Clinical Trials

Keith Desserich, Chairman of the Board at The Cure Starts Now, explains clinical trials – what they mean for cancer patients and how they work.

Clinical trials are set up in phases, all designed to focus on an idea. We call these preclinical, or even translational research trials. They are designed to conduct testing on cancer cells in a test tube or mouse modeling.  Ultimately you are trying to determine whether or not this idea, this concept, is actually going to work on cancer cells.

From there, if you're successful, you might end up moving into Phase 0 drug trials. These are very small groups - small numbers of patients. They're quick trials. Sometimes they may not be a real benefit to the patient, but what they're designed to do is to figure out how the drug works and test the concept of “if it will work in a test tube, will it also work in human type of trial?”

The real conventional phases and the conventional trials start with a Phase I trial. This is what we classically call clinical trials. What this is designed to do is determine whether or not the treatment is safe. It's not focused on whether or not the drug works or the concept works. It's about looking at the maximum tolerated dose, or MTD. Effectively, what they do is progress through each one of the patients with a slightly higher dose until they start to see side effects.

From there, if it continues and there is a safe option for it, they move into Phase II clinical trials. Phase II clinical trials are where we start to evaluate whether or not the treatment works. For those, we're talking about 25-100 different patients, and there may even be different treatment options with different dosages and/or combinations of drugs. This is where we're starting to see some of the results of it and determining whether or not there's a potential that we can possibly use this for future treatments against cancer.

The next phase is a Phase III clinical trial. This phase is about determining whether or not it's better than what we currently have as standard treatments. These are often randomized trials with several hundred participants. It can be spread out over many different centers - even internationally. Even the doctor sometimes doesn't know who has received it. This is what we call a double-blind sampling.

If it succeeds through Phase I, II and III, and we can show the dosage actually works, and that it's better than what's currently on the market, then the hope is that the drug can go ahead and get approval through government organizations like the FDA. If it does, even after that, sometimes we’ll even create a Phase IV trial.  This is where we go back to look at approved drugs, and thousands of different people to discern is if there's something that we didn't know. Maybe there's a side effect. Maybe there's something else that it can possibly be used to treat.

Sometimes patients and families can get frustrated just trying to understand which phase a trial is in and whether it's going to benefit them. But, one of the biggest questions that we get asked is, why has it stopped? You see this ongoing right now as we deal with the current pandemic.  There – just like in cancer research - trials are paused. Often these are paused because either they found that there was a side effect or a problem and they don't want to keep introducing it to new patients. Sometimes they find that it wasn’t related to the trial and then the trial opens back up in a few days or weeks.  Sometimes they just reach the patient number or the threshold that they were looking for in their trial framework. Ultimately, it is about safety, because no one wants to treat hundreds, or even thousands of people, without fully identifying whether the drug or treatment actually works.

Other times the reason why treatments are paused is because they may be moving to the next phase, and that's a good thing. It may take a little bit of time to ramp up or change strategies, or even get some participation, to be able to move on to the next phase.

As you go through this, it is important to know that sometimes even the doctors that are participating in the administration of the trial may not even know the results until the trial ends. They may know from their patient group, and they may have some basic understanding of it, but at the end of the day, whether a phase is open or closed really does relate back to you and helps determine whether it's a trial that you want to participate in.

To make it easier, we created a trial update finder at and geared for those families facing a diagnosis of DIPG, DMG or medulloblastoma brain cancers.  There you can actually log in and pull up any trials that are open and/or closed, and return daily results as they open and close.  And with this system, you aren’t just pulling trial data in the U.S.A. but also European, Canada, Australia and New Zealand trials.  It’s all designed to give the patient and family the power of information and the ability to discuss the latest options with their doctor.