A marathon, not a sprint: Life with chronic myeloid leukemia

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(BPT) - On a Friday night in 1999, Al saw a TV commercial about a new cancer treatment that was enrolling patients in a clinical trial — a novel, targeted therapy that scientists were investigating for the treatment of chronic myeloid leukemia (CML), a cancer that develops in the blood-forming cells of the bone marrow.

Two years prior, Al had been diagnosed with CML. At that time, his treatment options were limited. Most people with this diagnosis faced a five-year survival rate below 40%.

Al called his doctor the following Monday to find out if the new treatment — a tyrosine kinase inhibitor (TKI) — might be right for him. He was accepted into the trial a few months later — an impactful step that is indicative of the future of Al’s treatment journey, and the bravery he continually displayed through every high and low.

A Long and Winding Road

The clinical trial Al enrolled in led to the FDA approval of the first TKI for the treatment of CML, and the first-ever targeted therapy acting on specific cancer cells while causing less damage to normal cells.

With the introduction of TKIs, the five-year survival rate for patients diagnosed with CML improved, increasing to over 70%, bringing the lifespan of a patient with CML closer to the lifespan of a person without cancer.

However, despite the significant positive impact on the treatment of CML, it is not uncommon for some patients to not respond to certain TKIs, or struggle with side effects, or find that their response or tolerability to a specific TKI changes over time.

After the clinical trial was complete, Al decided to stay on the treatment. However, while the treatment was easy to tolerate — giving him few side effects — Al’s doctors told him that his CML was not responding as well as it should have been and there was a risk that his CML would progress and become more severe.

Al participated in more clinical trials before finding a treatment that was right for him in his fourth trial. This treatment was for a different type of TKI known as Scemblix® (asciminib) tablets. Scemblix is approved by the FDA for adults with Philadelphia chromosome-positive CML in chronic phase (Ph+ CML-CP) who have previously been treated with two or more TKIs1. More information about Scemblix can be found at us.scemblix.com.

Scemblix targets CML differently than other TKIs. Because cancer can mutate when it gets accustomed to how a treatment works, approaching it in a new way can outsmart the mutations. Scemblix may help patients overcome the resistance and side effects patients with CML may experience with other TKIs.

In a clinical trial, Scemblix showed how well it works to counteract cancer compared with Bosulif® (bosutinib), another TKI, in patients who had previously tried two or more TKIs1. Nearly twice as many patients on Scemblix achieved major molecular response, or MMR (25% for Scemblix [40 of 157] vs. 13% for Bosulif [10 of 75]) at 24 weeks, which is an important treatment milestone in CML, meaning that there is a low amount of the BCR::ABL in blood cells1.

This efficacy trend continued at 96 weeks, almost two years after the trial started, when more patients on Scemblix achieved MMR compared to those on Bosulif (38% [59 of 157] vs. 16% [12 of 76])1.

Additionally, in this trial [one study for 3L CML], three times fewer patients treated with Scemblix needed to stop treatment due to side effects compared to those taking Bosulif (8% for Scemblix vs. 26% for Bosulif) at the Week 96 analysis1. The most common adverse reactions in patients who received Scemblix in this trial were upper respiratory tract infections, musculoskeletal pain, headache, and fatigue1.

Al’s journey to the right treatment was made possible not only by his bravery in trying new treatment options, but also by good communication with his doctor. While Scemblix may not be right for everybody, it helped Al achieve his milestones.

Want to learn more about Al’s story and Scemblix? Visit us.scemblix.com.

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.

Reference

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

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