Multiple Myeloma Clinical Trials
Top myeloma expert, Dr. Rafael Fonseca, share the basics of what clinical trials are, how they work, and why they are so important in the field of multiple myeloma.
What phases do clinical trials go through?
There are three key clinical trial groups people need to know about. There are more than that, but the popular ones to know are phases 1, 2 and 3.
Phase 1 trials are typically small clinical trials. A drug is being tested for the first time against a certain condition and sometimes for the first time in humans.
The only focus of that trial is to find safety and make sure there are no unexpected safety hazards before opening the trial up to more people. Typically, these trials will start with low doses and gradually increase.
The next step is to see if what you’re doing has any activity. That’s called a phase 2 trial. They usually are small to medium-sized. These people are given the dose that was determined to be safe in the first phase.
What they’re looking for here is what fraction of patients respond to a specific treatment. It’s pretty straightforward. Phase 1 and 2 clinical trials are done with drugs that aren’t yet available.
If a phase 2 trial is successful, there’s some hope that what you’re seeing is going to be better for the disease. That’s where you go to phase 3. That’s where they would prove that a new drug is going to be better than what’s considered standard or that it can improve patient outcomes when added to the standard.
This is considered the gold standard because in this phase, you can test rigorously whether or not something adds to the care of patients. This is where they ask questions like, “How long does it control the disease or improve survival?” It looks at all sorts of metrics.
Would you say that phase 3 trials are better to look at getting into than 1 and 2?
Not necessarily. In phase 3, you probably have the lowest risk as far as clinical trials, but you also have a less innovation, too. There have already been some advancements.
Phase 1 and 2 give you options for things that aren’t yet being considered for phase 3. There have been myeloma drugs approved on the basis of phase 2 because it’s just so obvious that patients who didn’t have any other options were responding.
In fact, that has and will be the case for some of these new immunotherapeutics. They can sometimes save individuals who would otherwise have not had any options.
Even phase 1 trials are interesting. If you go back about 40 years, people used to think of phase 1 clinical trials as, “Well, let’s just see if something works.”
Now these things are quite different. You don’t see what the low levels of responses like you would back then. They’re very well thought out.
Where should patients go if they’re interested?
I would recommend they go to SparkCures. There’s clinicaltrials.gov. That’s sort of the central source, but if you go there, you’ll notice it’s not really that easy. There are many trials listed with no way of knowing if you are eligible or not.
With SparkCures, you put in your information. You can tell them how far you’re willing to travel, and it will suggest trials you could be eligible for. It has a really neat tool that tells you about centers of support.
Let’s say you live in LA but have a family member living in Atlanta. As it turns out, Atlanta has a great myeloma center. Suddenly Atlanta is now an option because you might be able to stay with your family.
As a doctor, I can tell you that we’re always thinking, “Is there a trial that could be good for this patient?” If I don’t talk about a trial, it’s maybe because there’s nothing that would be better than what we’re going to recommend.
Trials often open up new avenues for patients to consider. These new avenues could be adding a drug to an existing course of treatment. For example, there was a trial for adding Darzalex to the standard induction treatment VRd.
With this, they were able to greatly increase patient responses. 94-percent of patients in the trial were able to reach a complete response at the end of the consolidation period. This is going to lead to more clinical trials.
Trials can also help you by bringing in drugs that aren’t yet approved but show some promise. There’s a new antibody out now from GSK. It has a warhead attached to it basically.
It has a molecule that’s going to be toxic to myeloma cells, and it seems to be working. It has some toxicities, and we’re trying to figure out how to best use it. It seems to work even when nothing else is working, and that’s only available through clinical trials right now.
Never think of trials as being exposed to random things. They’re very well thought out. More often than not, you have a high level of reassurance.