Latest Advancements in the Treatment of Chronic Myeloid Leukemia
Jorge Cortes, MD, Georgia Cancer Center, Augusta, Georgia, discusses progress in the treatment of chronic myeloid leukemia (CML), including the introduction of tyrosine kinase inhibitors (TKIs) such as asciminib as a frontline treatment for patients with CML.
Dr Cortes stated, “Recent progress has been tremendous with new tyrosine kinase inhibitors and new mechanisms of action, but we still have a way to go. We'll continue working on that so that every patient can have the outcome that they expect and that they want.”
Transcript:
Hello, my name is Jorge Cortes. I'm the director of the Georgia Cancer Center at Augusta University in Augusta, Georgia. I've been working in CML for many years, and I've been very fortunate to see the great progress that we've made in CML, from the years where we were only doing chemotherapy with busulfan and hydroxyurea.
A major evolution that was the introduction of interferon and stem cell transplants. More recently, with the use of the tyrosine kinase inhibitors, a revolution that completely changed the nature, not only of CML, but of cancer in general where we're no longer using chemotherapy, we're using what we call targeted therapies that specifically block the proteins that cause the leukemia. That has been a great advance. It has allowed patients that have good access to treatment and good monitoring and good management to have a near normal life expectancy, which is a huge development.
However, we're not satisfied. There's a lot more that we need to do, and that is the focus for the coming years. For example, at the beginning, we didn't think that we will be able to stop therapy in any patient and that patients would be okay as long as they took their medication for the rest of their lives. We learned a few years ago, well, probably about 10 years ago, that we could stop treatment in some patients that met some specific criteria, and that has been another great advance. The problem is that that is available successfully for only about 25 to 30% of the patients. Some of them don't meet the criteria to stop. Some of them meet the criteria, but then the disease comes back. One of the bigger challenges that we have is, can we find better approaches that can allow us to have greater success so that more patients can stop therapy eventually and be free of disease, free of therapy as well, what we call treatment-free remission?
Another important focus is the quality of life. These drugs are incredibly superior to chemotherapy and incredibly superior to interferon in terms of their side effects and their tolerability. Still, patients have long-term effects that limit sometimes their ability to do their normal life, their normal work, their activities that they enjoy. Every patient is different. Some live a completely normal life, but others do have limitations. We also are focused on how can we improve the quality of life so that patients not only live longer but live better. Those are some of the main focuses on our research recently.
One of the probably major breakthroughs that we've had that we think are getting us closer to achieving bold goals is a development of the most recent tyrosine kinase inhibitor, the sixth one approved in the United States called asciminib. This is a drug that, although a tyrosine kinase inhibitor, works through a completely different mechanism, and it has shown that it is very effective in patients that have not responded well or have not tolerated well other tyrosine kinase inhibitors, with many of them, the majority achieving a good response.
It's also associated with a very good safety profile. It tends to be very well tolerated. More recently, it has actually been introduced as first-line therapy because of all these properties and showing that it can give us more patients to respond faster and deeper and also with fewer side effects so far. We are very encouraged by this new step.
We are continuing to look for additional ways to improve on all of these aspects. We are looking, for example, at combinations of therapy to see if we can take advantage of the immune system, other targets that can collaborate with asciminib or other tyrosine kinase inhibitors to further improve the probability of getting to the responses that we need to stop therapy. Combine the 2 types of tyrosine kinase inhibitors, the standard ones, and asciminib, these myristoyl pocket inhibitors. That's another approach. We are exploring all of these approaches to try to see where we can continue to improve.
There are also new drugs that are being developed and some are fairly advanced. There is a possibility that we will see a few more drugs coming into our [indiscernible], although we have many, you can never have enough because patients, some of them still don't respond or don't tolerate what we have available.
There's continued research, continued improvement, and our goal is to try to get to a final cure for all patients. With the International CML Foundation, we are putting together a consortium of international investigators, experts in every aspect of CML, from the laboratory to the clinic with a project that we call Cure CML, where that's our ambition to understand what causes the persistence of the disease and try to eliminate it for every patient possible.
We are encouraged by the progress that we've made. Recent progress has been tremendous with new tyrosine kinase inhibitors and new mechanisms of action, but we still have a way to go and we'll continue working on that so that every patient can have the outcome that they expect and that they want. Thank you very much for your attention.
