A Roller Coaster Filled With Twists and Turns: Life With Chronic Myeloid Leukemia

Lynne never thought she’d take part in a clinical trial. But after struggling to find a treatment that kept her condition in check—and didn’t give her intolerable side effects—the possibility of finding the right medication for her outweighed the perceived risks.

And finding the right treatment was critical. Lynne has Philadelphia chromosome-positive chronic myeloid leukemia in chronic phase (Ph+ CML-CP), a rare cancer that develops in the blood-forming cells of the bone marrow, which builds up and eventually invades the blood¹. If left untreated, CML, like most cancers, can be fatal.

Since her diagnosis, Lynne’s journey has been anything but a straight line—volleying between different medications and a clinical trial, making progress and experiencing setbacks, struggling to find a treatment option that was right for her.

On the 10-year anniversary of her diagnosis, the start of an important new chapter in Lynne’s treatment experience, she began a clinical trial of an investigational treatment for Ph+ CML-CP that is today known as SCEMBLIX^® (asciminib) tablets.

However, this is not where Lynne’s story starts…

The beginning of a journey filled with twists and turns

Now 58, Lynne lives in northwestern Connecticut. It’s here that she built a home and raised two adventurous children: an environmental scientist son and a snowboard instructing daughter who spends her time on both coasts of the United States.

Today, life is good. A walk-through of Lynne’s home reveals several half-knitted scarves, attestations to Lynne’s busy life and affinity for creative projects. Also plainly on display when you meet Lynne? How much family means to her, the love for her children, and a deep passion for helping others. She loves summer, the beach, and reading. 

But in 2005, when Lynne was only 39 and her children just 11 and 13, she grew sick—she was unaware that she was living with Ph+ CML-CP, and her life was about to become a roller coaster. 

When the roller coaster started to drop

After getting to the bottom of her symptoms and coming away with a CML diagnosis, Lynne began to see an oncologist. At her first appointment, Lynne learned there were only a few treatment options—including a tyrosine kinase inhibitor, or TKI. The TKI treatment had been approved a few years prior and was shown to be effective in the management of CML. She opted to start the TKI, and left feeling confident. She notes that even though she was initially nervous about the treatment she’d receive at her small-town hospital, having a “rockstar” specialist who listens to you is “the most important thing you can have.” Her complete blood count numbers balanced out within two weeks of starting treatment. The roller coaster seemed to plateau, but she still never met the treatment milestones expected.

However, even though her numbers were steady and in an acceptable range, her side effects were difficult to manage. But, thinking of her kids, she pushed through for more than 5 years, until a second TKI was approved after clinical trials and became available.

Navigating the loops of life with CML

As she began this new treatment option, Lynne experienced relief from her current side effects for the first time in years. But what she thought to be a high point on her treatment roller coaster turned out to be a loop—she faced a new set of side effects that she could not tolerate. 

A need to switch medication is an experience that is relatively common among people living with CML. While the need to switch is clear when the treatment is not working, there may be more hesitation around switching due to challenging side effects. Yet, approximately 55% of patients with CML who have switched therapies at least twice due to failure were intolerant to a previous treatment^2,3. Knowing this facet of life with CML, Lynne made the switch she needed to make, returning to the first TKI treatment after only 3 months of being on the second. 

When a third TKI treatment became available within the year, Lynne switched again and the side effects she faced lessened. Lynne remained on this treatment for 4 years. Her brother invited her on a trip to Costa Rica and, as an avid beach lover, Lynne was excited. However, as she began counting down the days until their departure, Lynne’s doctor called to tell her that her blood tests were showing that the medication was not keeping her leukemic cells in check as well as it once had—her response to the medication was changing. As her anticipated vacation grew closer, things continued to decline, and she was at risk of her CML progressing. Side effects had pushed Lynne off her first and second treatments, but her cancer was not being managed by this third one.

Eager to find a better option for her, Lynne’s doctor fought to get her into a clinical trial for a new investigational type of TKI for the treatment of CML. Before starting, her doctor explained some of the common adverse events—including upper respiratory tract infection, muscle pain, headache, and fatigue—and, on the 10-year anniversary of her diagnosis, Lynne decided she was ready to try. That clinical trial led to the FDA approval of Scemblix, a prescription medication used to treat patients with Ph+ CML-CP, previously treated with 2 or more TKIs. Scemblix targets CML differently than other TKIs. Individuals with Ph+ CML have a mutation in a 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 TKIs⁴.

Finding the right treatment option

With this treatment option today known as SCEMBLIX, Lynne has been able to manage her side effects and keep her cancer in check since 2015. She describes learning she was finally meeting her CML treatment milestones as emotional. 

In a clinical trial of patients who had tried two or more TKIs, nearly twice as many patients achieved a major molecular response (MMR) with Scemblix (25%, 40 of 157) at 24 weeks than with the TKI Bosulif^® (bosutinib) (13%, 10 of 76)⁵. At 96 weeks, almost 2 years after the start of the trial, 59 out of 157 patients (38%) on Scemblix achieved MMR compared with 12 out of 76 patients (16%) on Bosulif⁵.

The most common side effects reported (≥20%) with Scemblix in this clinical trial were nose/throat infections; pain in the muscles, bones, or joints; headache; and tiredness.

Lynne’s advice for traversing the winding road of life with CML

Today, Lynne says she feels better, although she does still experience some side effects. While there are Scemblix patients who similarly continue to have some side effects, there is encouraging data that show three times fewer patients (7% vs. 25%) treated with Scemblix in a clinical trial needed to stop treatment permanently due to side effects compared with those taking Bosulif^®5. This trend continues almost two years from the start of the trial at 96 weeks. Twenty-six percent of patients taking Bosulif (20 of 76 patients) had to stop taking their medication due to side effects compared to only 8% of those taking Scemblix (12 of 156 patients)⁵. 

Lynne wants others to know that while their journey with CML may be a “bumpy ride,” there are highs to be celebrated. There are more options available today, and the treatment landscape for CML continues to improve. Lynne is actively engaged with the CML community and hopes that by sharing her story, she can help others who face uncertainty in their own treatment journey. 

Do you recognize your own story or that of a loved one through Lynne’s story? If you live with CML and experience a lack of response or unmanageable side effects from your current medication for Ph+ CML-CP, you’re not alone. You can learn more about Scemblix and patient support resources 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.

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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