Chronic Myeloid Leukemia (CML) Q&A with Giora Sharf

Background
Mendus’ lead candidate vididencel is in a Phase 2b trial for acute myeloid leukemia (AML) and could benefit patients with chronic myeloid leukemia (CML). Over 25 years, tyrosine kinase inhibitors (TKIs)—targeted pills that block the protein causing CML—have transformed this blood cancer from a fatal disease into a manageable chronic condition. Yet only about a quarter of patients in Western countries and about 10 percent of patients globally can stop therapy after a decade and remain cancer-free. Many still struggle with daily side effects, limited access to testing, and uncertainty about long-term outcomes.

Glossary of Key Terms
CML (Chronic Myeloid Leukemia): A type of blood cancer caused by a chromosome swap that creates the “Philadelphia chromosome.”
Philadelphia Chromosome (9/22): The abnormal chromosome that drives CML.
TKIs (Tyrosine Kinase Inhibitors): Targeted oral drugs that block the cancer-causing protein.
PCR Test: A highly sensitive blood test that detects tiny traces of leukemia.
TFR (Treatment-Free Remission): When a patient stops therapy but remains in remission under careful monitoring.

Giora, you are a co-founder of the International CML Advocates Network and have battled CML since 2000. Can you give us a quick snapshot of what CML is and an idea of which symptoms impact younger and older patients?
CML is a blood cancer caused by a genetic swap between chromosomes 9 and 22—known as the Philadelphia chromosome—that creates an abnormal protein driving uncontrolled white-blood-cell growth. When I was diagnosed twenty-five years ago, it was considered fatal. Doctors told me I might have only three or four years to live. Thanks to TKIs, life expectancy for many patients today is similar to that of the general population. For anyone newly diagnosed, that shift offers tremendous hope.
But survival isn’t the whole story. Fatigue is the most common and disruptive symptom, affecting work, family, and mental health. Patients may also experience rashes, cramps, bone pain, diarrhea, eye swelling, or even memory and concentration problems. Some drugs can cause fluid around the lungs or heart issues. Younger patients often feel side effects more intensely, which can affect their daily life and adherence to treatment.
“Fatigue is the most common and disruptive symptom.”

And do you see there being a significant difference regarding the symptoms between younger and older patients?
Yes. Younger people often seem to experience stronger side effects, even if formal studies haven’t fully proven this. They’re juggling careers, school, relationships, or starting families, and persistent fatigue or cramps can interfere with every part of life. Being told in your twenties or thirties that you’ll need medication indefinitely can be emotionally overwhelming. Some younger patients skip doses or take breaks to “feel normal,” which can jeopardize their health.
Older patients may be more consistent with treatment but can face other health problems that complicate therapy. Recognizing these differences helps doctors and support groups offer tailored advice and encouragement.
“Younger adults sometimes stop pills just to feel normal again—adherence is critical.”

Can you share a bit more detail on your personal experience managing the disease?
When I was diagnosed, the standard therapy was interferon with chemotherapy—treatments with harsh side effects. I searched online and found a patient chat group discussing a promising new drug, imatinib. I contacted Professor Andreas Hochhaus in Germany and joined the IRIS clinical trial for Gleevec or as it was named then- STI-571. At first, I was randomized to the older therapy, which was so difficult that I constantly emailed my doctor about side effects.
In February 2001, I finally switched to imatinib, and that day felt like a second birthday. I took it for fourteen years. My main issues were eye swelling and painful nighttime cramps, but the medication gave me back a normal life. After achieving a deep molecular remission—no detectable cancer even with sensitive PCR tests—my doctor encouraged me to stop. In 2014, I ended treatment and have now been in treatment-free remission for over eleven years. I still do PCR tests every six months and know that if I relapse, several effective drugs are available.
“February 14, 2001—my switch to imatinib—was like a second birthday.”

There seems to be a misperception that CML is a disease under control. Can you elaborate a bit more on that?
It’s easy to think CML is “solved” when you look only at survival rates in wealthy countries. Many doctors who treat more aggressive leukemias or lymphomas understandably focus elsewhere. But for patients, the story isn’t that simple. Side effects can limit work and relationships. Living with a controlled cancer is still emotionally heavy, and in many parts of the world, TKIs or reliable PCR testing are not guaranteed.
Quality of life matters as much as length of life. Even with modern drugs, some patients develop resistance or never reach deep remission. Until stopping therapy without relapse is reliable and global access is ensured, CML is not a finished problem.
“High survival doesn’t mean CML is solved—quality of life still lags behind.”

What needs to happen to make sure more CML patients reach treatment-free remission?
First, reliable PCR monitoring must be available everywhere. To stop therapy safely, patients need frequent testing—monthly for the first six months, then less often. In many low- and middle-income countries, these tests are too expensive or unavailable, so patients remain on treatment even when they might qualify to stop. Expanding access, as groups like the Max Foundation are doing, is crucial.
Second, we need better prediction tools. Researchers are studying factors such as how quickly leukemia levels fall and how long patients remain in remission. The more accurately we can forecast who will succeed without treatment, the more patients can attempt TFR confidently. Education and emotional support for patients considering stopping are also vital.
“Without reliable testing, many patients stay on therapy simply because it’s not safe to stop.”

And finally, could you tell us a little bit more about the CML Advocates Network that you helped co-found and what its ambition is?
The CML Advocates Network began in 2003 after Novartis invited about 25 patient advocates to Switzerland for the first CML Horizons meeting. By 2009, three of us—Jan Geissler, Jana Pelouchova, and I—took over and formed an independent non-profit in Switzerland. Today, we represent 130 patient groups in nearly 100 countries.
Our mission is to empower patient organizations, raise awareness—September 22 (9/22 is International CML Awareness Day—and ensure patient voices shape research and policy. We run CML Horizons conferences, training academies, and global surveys on adherence, stopping therapy, and quality of life. By publishing real-world evidence, we help doctors and companies understand what matters most to patients. Ultimately, we want a world where every person with CML has the knowledge, support, and treatments needed to live fully—and, one day, be cured.
“We give CML patients everywhere a voice and the tools to live fully.”

What is your message to researchers and companies developing CML therapies?
Please continue innovating. Even with TKIs, some patients develop resistance or severe side effects, and a few still need bone marrow transplants. New therapies in the pipeline give hope to those running out of options. Our community is encouraged when companies show commitment to improving treatment choices.
Every additional therapy broadens options and improves lives. Progress in CML proves what’s possible when patients, researchers, and industry collaborate. The goal isn’t just survival—it’s ensuring people with CML everywhere can live well, free from constant reminders of illness.
“Don’t stop innovating—patients still need better, safer options.”