Al Kept Searching for the Right CML Treatment—and You Should Too

 

Learn More About Al’s CML Journey Here

“Plans are made to be broken.”

This common phrase may not have been coined with health in mind, but most anyone who lives with a chronic health condition would tell you this same story: one thing that’s consistent about the patient journey is the many ups and downs that can often leave plans significantly changed — plans as small as going to a child’s soccer game or as large as changing careers.

Life with chronic myeloid leukemia (CML) is no exception. It’s uncommon to find a smooth treatment journey that aligns with an individual’s every expectation; there are bound to be ups, such as a positive response to a new course of treatment, and there are bound to be downs, such as unmanageable side effects from treatment.

If anyone can attest to the bumpy, ever-changing road that is life with CML, it’s Al.

A Boston native who now lives in the city’s suburbs, Al is a freelance web developer. Between that work and his previous work in the IT industry, both rapidly evolving with technology, he has seen transition and transformation throughout his professional life.

His health experiences have been no different; between his CML diagnosis in 1997 and today, he has been on an odyssey that looks nothing like a straight line — zigzagging between medications, clinical trials, and running through a long and winding list of treatment options that ultimately brought him to SCEMBLIX^® (asciminib) tablets, a treatment that he feels is “just right” for him.

“The way I would talk about the journey of a CML patient: it’s a marathon, it’s not a sprint.”

Navigating across decades with CML

It was a routine blood test that brought CML into Al’s life. He got a message asking him to return to redo his labs after some unusual levels were detected. When the second tests came back, Al was referred to an oncologist, who then told him about something called chronic myeloid leukemia, a cancer that develops in the blood-forming cells of the bone marrow, which builds up and eventually invades the blood¹.

“I had heard about leukemia, but I didn’t know all these different variations. And I certainly did not think somebody who was 32 years old at the time could get it.”

Al describes the first years following his diagnosis—Philadelphia chromosome-positive CML in chronic phase (Ph+ CML-CP) — as “the dark years,” recalling that he “tried a lot of things — not [finding] a whole lot of success.”

His experience started to shift in December of 1999 when he saw a story on the news about a new drug for CML that was in a clinical trial — a tyrosine kinase inhibitor (TKI). “I remember, it was a Friday night, and Monday morning, I was on the phone with my doctor… I said, ‘How can I get on this?’”

By the following May, he was involved in the trial, which lasted a year. Around the conclusion of the trial, he found he had a match for a bone marrow transplant and his doctor presented options to him—either stay with his treatment or seek out the transplant, which could have its own risks. “I decided to stay on the treatment.”

Al recalls that the first drug was easy to tolerate, but noted there was still some resistance — “it didn’t get me all the way there.”

Though there have been significant advances in CML care over the last few decades, some people living with CML may not respond to certain treatments, may not tolerate treatment side effects, or may find their response or tolerability to a specific medication changes over time. Any of these twists and turns can result in the need to switch treatment, even if that means coming off a medicine that was once working.

A need to switch medication is an experience that is more common than not among people living with CML. While the need to switch is clear when the treatment is not working, the need to switch is not always as clear when it is driven by side effects. Approximately 55% of patients with CML who have switched therapies at least twice due to failure were intolerant to a previous treatment^2,3.

When Al did not experience a full response on the first trial, as new treatments for CML were being developed, he qualified for a second trial, which was followed by a third, and then a fourth.

In this fourth trial, Al’s physicians recommended he participate in the clinical trial of another type of CML treatment, which is known today as Scemblix^® (asciminib) tablets.  Scemblix is approved by the FDA for adults living with Philadelphia chromosome-positive CML in chronic phase who have previously been treated with 2 or more tyrosine kinase inhibitor medications.

Although his treatment journey had been a roller coaster, it was that roller coaster that ultimately brought Al to Scemblix, which worked for him.

Finding a treatment that works is a meaningful achievement in CML treatment. Individuals not only need to respond to the treatment, but they also need to find an option where the side effects are manageable and non-disruptive to their daily life, so they can be comfortable taking the therapy long-term. Al and his doctor discussed that every medicine has risks and benefits, and together, they talked through some of the most common adverse reactions of Scemblix, including upper respiratory tract infection, muscle pain, headache, and fatigue.

Scemblix is one treatment option that hopes to help CML patients keep their CML in check.  Scemblix targets CML differently than other tyrosine kinase inhibitors. Individuals with Philadelphia chromosome-positive CML have a mutation in a specific gene called BCR::ABL, which makes tyrosine kinases — a type of protein that tells leukemia cells to keep growing⁴. By blocking tyrosine kinases from a different site, Scemblix may help patients overcome the resistance they may experience with other tyrosine kinase inhibitors (TKIs)⁴.

In a clinical trial, Scemblix showed superior efficacy—how well it works to counteract cancer cells—compared to another TKI, Bosulif^® (bosutinib), in patients who had tried 2 or more TKIs. Nearly twice as many patients (25%, 40 of 157, for Scemblix vs. 13%, 10 of 75, for Bosulif) achieved major molecular response or MMR at 24 weeks — an important treatment milestone in CML in which blood tests detect a low amount of BCR::ABL in the blood cells (specifically, ≤0.1%, or 1 out of every 1000 cells has the BCR::ABL gene)⁵.

That trend continued at 96 weeks, almost two years after the trial started, when more patients (38%, 59 of 157) on Scemblix achieved MMR compared to those on Bosulif (16%, 12 of 76)⁵.

Additionally, 3 times fewer patients treated with Scemblix in clinical trials needed to stop treatment due to side effects compared to those taking Bosulif (8% for Scemblix vs. 26% for Bosulif), at the Week 96 analysis⁵.

Al’s story is extraordinary in the way it follows the gains made in CML treatment over the last 2 decades, and it is one that has been changed for the better by using Scemblix as his TKI therapy. While Scemblix may not work for all people with CML, it has helped Al manage his CML for over 6 years.

What has helped Al navigate CML

Having experienced many ups and downs over 25 years, Al said he can feel like he’s “the old guy of CML patients.” He is quick to share advice he has picked up through the winding course of his treatment.

“You may need to try different treatments before they find one that works great for you.” Speaking to those who have tried 2 or 3 other drugs already, he starts by sharing what he’s been through. “I will tell [them] about my journey [and tell them] to not give up hope… to keep an open mind about what’s possible.”

Something Al recommends, as it helped him greatly along his journey, is learning as much as one possibly can about CML and how the treatments work . “I think the more you know, the better kinds of conversations you can have with your care team,” he said. Maintaining strong, open lines of communication with a care team, including bringing up side effects as they occur to a doctor, can be an impactful way for people living with CML to improve their treatment experience. Whether your body is not responding, has stopped responding, or the side effects of your medication have become unmanageable, you can use this Doctor Discussion Guide to track recent side effects, their duration, and your BCR-ABL1 levels, which will help you better approach the conversation with your doctor.

When facing a long up-and-down odyssey, finding support is also critical. Al advocates for the importance of leaning on others, which can come through a formal support group or through friends and family, “Don’t be so proud. Don’t be embarrassed to ask for help.” For those seeking support resources, services can be found through Scemblix Connex, a patient support program available through Scemblix.com.

Recognize your own story or that of a loved one through Al’s story? If you live with CML and experience a lack of response or unmanageable side effects from current medication for Philadelphia chromosome-positive CML in chronic phase, you’re not alone.

If you would like to learn more about Scemblix and explore any of the additional patient support resources they offer, you can do so by clicking here.

APPROVED USES for SCEMBLIX^® (asciminib) Tablets
SCEMBLIX is a prescription medicine used to treat adults with:

• Philadelphia chromosome-positive chronic myeloid leukemia (Ph+ CML) in chronic phase (CP), previously treated with 2 or more tyrosine kinase inhibitors (TKIs)
• Ph+ CML in CP with the T315I mutation

It is not known if SCEMBLIX is safe and effective in children.

IMPORTANT SAFETY INFORMATION for SCEMBLIX^® (asciminib) Tablets
Before taking SCEMBLIX, tell your doctor about all your medical conditions, including if you:

• have a history of inflammation of your pancreas (pancreatitis)
• have a history of heart problems or blood clots in your arteries and veins (types of blood vessels)
• are pregnant or plan to become pregnant. SCEMBLIX can harm your unborn baby
  • Your doctor will do a pregnancy test before you start treatment with SCEMBLIX
  • Females who are able to become pregnant should use effective birth control during treatment and for 1 week after your last dose of SCEMBLIX. Talk to your doctor about birth control methods that may be right for you
  • Tell your doctor right away if you become pregnant or think you may be pregnant during treatment with SCEMBLIX
• are breastfeeding or plan to breastfeed. It is not known if SCEMBLIX passes into your breast milk. Do not breastfeed during treatment and for 1 week after your last dose of SCEMBLIX

Tell your doctor about all the medicines you take, including prescription medicines, over-the-counter medicines, vitamins, and herbal supplements. SCEMBLIX and other medicines may affect each other and cause side effects.

SCEMBLIX may cause serious side effects, including:

• Low blood cell counts: SCEMBLIX may cause low platelet counts (thrombocytopenia), low white blood cell counts (neutropenia), and low red blood cell counts (anemia). Your doctor will do blood tests to check your blood cell counts every 2 weeks for the first 3 months of treatment and then monthly or as needed during treatment with SCEMBLIX. Tell your doctor right away if you have unexpected bleeding or easy bruising, blood in your urine or stools, fever, or any signs of an infection
• Pancreas problems: SCEMBLIX may increase enzymes in your blood called amylase and lipase, which may be a sign of pancreatitis. Your doctor may do blood tests monthly or as needed during treatment with SCEMBLIX to check for problems with your pancreas. Tell your doctor right away if you have sudden stomach-area pain or discomfort, nausea, or vomiting
• High blood pressure: Your doctor may check your blood pressure and treat any high blood pressure during treatment with SCEMBLIX as needed. Tell your doctor if you develop elevated blood pressure or symptoms of high blood pressure including confusion, headaches, dizziness, chest pain, or shortness of breath
• Allergic reaction: Stop taking SCEMBLIX and get medical help right away if you get any signs or symptoms of an allergic reaction, including:

trouble breathing or swallowing swelling of the face, lips, or tongue skin rash or flushing of your skin

feeling dizzy or faint fever fast heartbeat

• Heart and blood vessel (cardiovascular) problems: SCEMBLIX may cause heart and blood vessel problems, including heart attack, stroke, blood clots, blockage of your arteries, heart failure, and abnormal heartbeat, which can be serious and may sometimes lead to death. These heart and blood vessel problems can happen in people with risk factors or a history of these problems, and/or previously treated with other TKI medicines. Your doctor may monitor you for heart and blood vessel problems and treat you as needed during treatment with SCEMBLIX. Tell your doctor or get medical help right away if you get:

shortness of breath chest pain or pressure a feeling like your heart is beating too fast or you feel abnormal heartbeats swelling in your ankles or feet dizziness weight gain

numbness or weakness on one side of your body decreased vision or loss of vision trouble talking pain in your arms, legs, back, neck, or jaw headache severe stomach-area pain

The most common side effects of SCEMBLIX include:

nose, throat, or sinus (upper respiratory tract) infections muscle, bone, or joint pain headache tiredness nausea rash diarrhea

decreased blood platelet counts, white blood cell counts, and red blood cell counts increased blood fat (triglycerides) levels increased blood creatine kinase levels increased blood liver enzyme levels increased blood pancreas enzyme (amylase and lipase) levels increased blood uric acid levels

Your doctor may change your dose, temporarily stop, or permanently stop treatment with SCEMBLIX if you have certain side effects.

SCEMBLIX may cause fertility problems in females. This may affect your ability to have a child. Talk to your doctor if this is a concern for you.

These are not all the possible side effects of SCEMBLIX. For more information, ask your doctor or pharmacist. Call your doctor for medical advice about side effects.

You are encouraged to report negative side effects of prescription drugs to the FDA. Visit www.fda.gov/medwatch, or call 1-800-FDA-1088.

Please see accompanying full Prescribing Information, including Patient Information.

References

¹American  Cancer Society. What Is Chronic Myeloid Leukemia? 2022. Available at https://www.cancer.org/cancer/chronic-myeloid-leukemia/about/what-is-cml.html.

²Giles FJ, et al. Nilotinib is active in chronic and accelerated phase chronic myeloid leukemia following failure of imatinib and dasatinib therapy. Leukemia. 2010; 24(7):1299–1301.

³Ongoren S, Eskazan AE, Suzan V, et al. Hematology. 2017;23(4):212-220.

⁴National Cancer Institute. Chronic Myelogenous Leukemia Treatment (PDQ®)–Patient Version. 2022. Available at https://www.cancer.gov/types/leukemia/patient/cml-treatment-pdq.

⁵Scemblix [prescribing information]. East Hanover, NJ: Novartis Pharmaceuticals Corp.

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