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Illustration of 3 round B cells, a type of white blood cell involved in blood cancer treatment

Inside the development of a revolutionary treatment for blood cancers

In 2014, a Johnson & Johnson therapy gained approval for the treatment of a type of B-cell malignancy—increasing survival rates and changing the way scientists approach cancer treatment. These 8 milestone moments tell the transformational story.

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A cancer diagnosis may bring on feelings of confusion, stress and fear. Perhaps the only thing worse than the diagnosis itself is finding out that available treatment options might have toxic side effects or that overall survival and quality-of-life data are not very encouraging.

Up until a decade ago, this was the reality for people with blood cancers known as B-cell malignancies that form in a type of white blood cell called B cells. The job of B cells is to protect you from infection by making proteins called antibodies.

Chronic lymphocytic leukemia (CLL) is among the most common kind of B-cell cancer. If you have CLL, the cancerous B cells multiply faster than the healthy ones. This compromises your immune system and may leave you vulnerable to infection.

Prior to 2014, the main way to treat CLL was with chemotherapy. “Those who were younger than 65 and deemed fit enough were given a type of chemotherapy that was sometimes effective but extremely toxic,” says Mark Wildgust, Ph.D., Vice President, Global Medical Affairs, Oncology, Johnson & Johnson. “Others who were older or frailer could only be treated with a less toxic chemotherapy that had fewer side effects but didn’t work as well.”

That all changed a decade ago. In 2014, the U.S. Food and Drug Administration (FDA) approved a drug co-developed by Johnson & Johnson for the treatment of adults with CLL who have received at least one prior therapy—transforming what it means to be diagnosed with the disease.

2024 marks the 10-year anniversary of the drug’s use for CLL, and it’s a medical milestone to celebrate. The introduction of this drug “was a giant step forward,” says Sarbani Chaudhuri, Vice President, Global Head, Hematology, Johnson & Johnson. “It revolutionized outcomes in CLL so much that today, some patients are living as long as an average person their age.”

Here’s a look at just how dramatically the landscape of B-cell malignancy treatment, specifically when it comes to CLL, has changed over the years—and what that means for patients.
  • Patient with chronic lymphocytic leukemia undergoing chemotherapy treatment
    1980s–early 2000s

    Most CLL patients are treated with chemotherapy

    During this period, a diagnosis of CLL often means undergoing a harsh type of chemotherapy. In addition to the toxicity of this treatment, the prognosis for most patients is poor. In the early 1980s, the average five-year survival rate was about 54%, and by 2004 it had increased—but only by 10% to about 60%.
  • 3D rendering of chronic lymphocytic leukemia cells

    The FDA approves an immune-based therapy for CLL

    In 2010, the FDA approves an immune-based therapy for CLL. The therapy, to be taken in combination with chemotherapy, targets a receptor found on the cell surface of B cells.
  • Scientist developing new targeted therapy for chronic lymphocytic leukemia (CLL)

    Johnson & Johnson partners to develop the first BTK inhibitor

    Johnson & Johnson accelerates its commitment to unmet patient needs and joins forces with a small biotech company called Pharmacyclics to develop a new targeted therapy for B-cell cancers, including CLL. “We had seen some promising early data and recognized the transformative nature of this research,” says Wildgust.

    The compound becomes the first in a brand-new class of drugs called BTK inhibitors, which work by blocking an enzyme called Bruton’s tyrosine kinase (BTK).

    “What happens in CLL is that the B cells proliferate in an uncontrolled way,” says Wildgust. “They take over the bone marrow and stop it from making normal red blood cells as well as white blood cells, which fight infections.” By inhibiting BTK, these drugs stop abnormal B cells from dividing and replicating.
  • Approved stamp

    The first BTK inhibitor gets FDA approval

    The FDA approves the Johnson & Johnson/Pharmacyclics product, which becomes the first-ever BTK inhibitor on the market. Fast-tracked under the FDA’s accelerated approval program, this medication, an oral therapy, is one of the first medicines with the FDA’s Breakthrough Therapy Designation to garner approval.

    This BTK inhibitor is approved in the U.S. for patients with CLL who had tried at least one other therapy. It is also approved in Europe to treat adult patients with relapsed or refractory mantle cell lymphoma (MCL). In subsequent years, it earns approval as a first-line treatment for patients with CLL.

    Thanks to this new treatment option, “many patients got their life back,” says Wildgust. “The treatment is more tolerable than chemotherapy, and patients didn’t have to go into the hospital to get it. They were able to live their lives at home.”
  • Scientist in lab wearing latex gloves holding test tube and pipette

    Johnson & Johnson’s BTK inhibitor is approved to treat additional diseases

    In 2015, the FDA approves the Johnson & Johnson/Pharmacyclics BTK inhibitor for the treatment of Waldenström’s macroglobulinemia. Never before had there been an FDA-approved treatment for this disease.

    In 2017, the FDA also approves this drug for patients with chronic graft-versus-host disease (cGVHD) who are not helped by other therapies. cGVHD is not cancer, but it is a common and serious complication of allogenic stem cell transplants (which are used to treat certain cancers as well as noncancerous conditions like aplastic anemia).

    During this time period, the drug also gains approval from numerous international regulatory authorities for the treatment of CLL, helping to change the standard of care for patients living with CLL by making life with an at-home, chemotherapy-free oral therapy a reality for patients worldwide. It’s also approved in Europe either in a fixed-duration combination with another B-cell cancer therapy or as a continuous monotherapy in first-line CLL. It continues to transform the CLL treatment landscape by giving healthcare providers the freedom to personalize therapies that may help patients achieve remission and improve overall quality of life.

    Between 2019 and 2023, three other BTK inhibitors gain FDA approval and become available in the U.S.
  • Clipart of a prescription pill bottle

    WHO names Johnson & Johnson’s BTK inhibitor to its list of Essential Medicines

    This year, the World Health Organization (WHO) includes the company’s BTK inhibitor in its list of “Essential Medicines.” These medicines “are selected with due regard to disease prevalence and public health relevance, evidence of efficacy and safety and comparative cost-effectiveness,” states the WHO on the organization’s website. “They are intended to be available in functioning health systems at all times, in appropriate dosage forms, of assured quality and at prices individuals and health systems can afford.” Johnson & Johnson’s BTK inhibitor has remained on the list ever since.
  • Doctor showing a chronic lymphocytic leukemia (CLL) patient BTK inhibitors for improved survival rate

    CLL survival rates increase

    Thanks largely to the availability of BTK inhibitors, survival rates for people with CLL continue to improve. A recent study tracked patients for eight years and found that those who are treated with one specific BTK inhibator “have similar survival estimates as age-matched patients in the general population.”

    “This was absolutely revolutionary,” says Chaudhuri. “There are very few disease states where we can say a treatment has the potential to restore normal life expectancy.”
  • Johnson & Johnson scientist looking into a microscope

    Scientists work to move from treatment to cures

    While the advent of BTK inhibitors has redefined what it means to live with CLL, Johnson & Johnson scientists are continuing to innovate.

    “While we have significantly improved survival and outcome, we are working on the next generation of treatment modalities that will help us move toward cures,” says Wildgust. “Our work is not done yet.”

    “We are focused on working toward a functional cure with a finite period of treatment so patients won’t experience a lifetime of taking medication,” adds Chaudhuri. “Striving to deliver curative regimens is fundamental to our vision.”

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