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Why we all have cause for optimism in the battle against blood cancers

Why we all have cause for optimism in the battle against blood cancers

Turn the clock back 70 years and the only way to treat cancer was to either “cut it out or burn it away with radiation”.[1] Meanwhile, any that couldn’t be surgically removed or irradiated, like blood cancers, were essentially untreatable.[1] Yet fast forward to today and we're seeing some blood cancer patients reach 10 years of remission thanks to a whole array of new, breakthrough therapies, coupled with a deeper understanding of the mechanisms of disease.[2]

Even going back as little as three years provides a different picture. Reflecting on an article by my colleague, Patrick Laroche, in 2020, I'm struck by the progress made. Since then, we've reached a point where multiple treatment options exist across the spectrum of blood cancers.[3] This not only makes a huge difference for those who have relapsed, but also provides hope for those with difficult-to-treat disease types. But progress hasn’t been limited to those patients who have experienced treatment failures; novel therapies and regimens have also led to improved patient outcomes in first-line treatment of multiple myeloma (MM) and greater flexibility for clinicians to make treatment decisions based on the unique needs of each patient.[4]

As a blood cancer community, there is a lot for us to be optimistic about in the future. Survival outcomes and quality of life for patients are improving all the time. The era of precision medicine is also ushering in new and more individualised approaches to screening, diagnosis, disease assessment and treatment decision-making. But there's no room for complacency. Collectively, we can be pleased with our achievements to date, but we must continue to strive for better and focus on one key question: what's next?

Targeting multiple myeloma

This question is crucial for all cancers. For MM, the considerable progress shouldn’t dampen our ambition to innovate and seek breakthroughs in areas of the disease where we still have a lot to learn. While treatments for solid cancers have leapt forward with precision medicine, allowing us to prescribe treatments to patients displaying specific biomarkers, MM lacks specific response predicting biomarkers that have proven successful in novel treatments for cancers such as prostate or lung.[5] In cancer, biomarkers are abnormalities or mutations found in cancer cells that can be used to detect and identify different types of cancer, predict the probable course of the disease, including its recurrence, and help guide treatment decisions by providing insights into patients likely response to a therapy.[6] The largest MM study on this topic to date identified 63 genes that recurrently mutate, initiating and/or driving disease progression.[7] Increasing our understanding of MM's unique challenges and, crucially, our ability to target these mutations, should therefore be a priority.

Likewise, we need to improve real-world representation in MM trials wherever possible, preventing the highly heterogeneous nature of patients from creating a disconnect between trial data and treatment individualisation strategies.[8] And we have to recognise the emotional, physical and mental impact of multiple treatment failures on patients, helping them better navigate their cancer journey and overcome the burden of third- to fifth-line treatments, which can often feel overwhelming.[3]

The rise (and rise) of immunotherapies

But that's enough of the 'should’ – let's get back to the 'can'. To continue using MM as an example, the development of immunotherapies represents a major milestone with the potential to change the treatment paradigm for all types of patients regardless of their age or setting (newly diagnosed or relapsed).[9]

These therapies represent pioneering science at its best — attacking cancer cells in different and multiple ways, working alongside a patient’s immune system. In short, they mean we're no longer approaching blood cancer ‘one therapy at a time’ and this opens the door to more effective treatment plans for patients, including those experiencing resistant disease or who have failed to respond to multiple previous options.[9] Identifying the patients who are most likely to respond to a particular therapy helps reduce the harm caused by unnecessary testing and treatment, having a positive impact on cost reduction and the sustainability of the healthcare system.[10]

In fact, such has been the success of immunotherapies in MM, that they're now recommended for a number of indications across transplant eligible and non-transplant eligible patients in Europe. Their introduction providing a much-needed additional option. The efficacy of these immunotherapies has been further built on with the latest generation of treatments, such as chimeric antigen receptor T (CAR-T) cells.[9]

10 years…and beyond

It's not just MM; we’ve seen significant leaps forward in other blood cancers too. All of which lay an exciting platform for future progress as we work towards a cure.

For example, a wave of innovation in B-cell malignancies, such as chronic lymphocytic leukaemia (CLL), has transformed the treatment landscape over the past decade.[11],[12] This includes the introduction of first and second-generation Bruton’s Tyrosine Kinase inhibitors (BTKi), which have shifted the standard of care for CLL from chemoimmunotherapy to highly effective targeted agents and combination therapies.[11],[12] Meanwhile, the introduction of risk factor testing and the provision of both fixed and continuous therapy options is enabling treatments to be personalised to a patient's individual needs and lifestyle.

Of course, the job's far from done. Blood cancers continue to affect millions of people worldwide and many experts still consider them to be largely incurable diseases. Yet while any cancer death is one death too many, there's little doubt that rapid advances in scientific research and development are taking us ever closer to functional cures.

In his article three years ago, Patrick finished by asking if the 2020s would be the decade when blood cancer patients and their families could enjoy longer, happier, healthier lives together. Today, I'm truly inspired to write that a growing number of them are now living many years in remission, and importantly with meaningful improvements to their quality of life compared to conventional therapeutic approaches.[2],[13] If we maintain and even accelerate the recent rate of progress, we can expect to see many more reach that milestone – and go beyond it – in the future.

[1] Dana Farber – Cancer Treatment: A Look at How It Has Evolved in 70 years. Available at: Accessed May 2023.

[2] Melenhorst JJ, Chen GM, Wang M, et al. Decade-long leukaemia remissions with persistence of CD4+ CAR T cells [published correction appears in Nature. 2022 Dec;612(7941):E22]. Nature. 2022;602(7897):503-509.

[3] Jensen C. The high cost burden of third- to fifth-line treatments for multiple myeloma: unsustainable and unaffordable. J Manag Care Spec Pharm. 2021;27(9):1321-1324.

[4] Dimopoulos et al. Developments in continuous therapy and maintenance treatment approaches for patients with newly diagnosed multiple myeloma. Blood Cancer. 2020;10(17)

[5] Pal M et al. Current advances in prognostic and diagnostic biomarkers for solid cancers: Detection techniques and future challenges. Biomedicine & Pharmacotherapy. 2022;146:112488

[6] Unlocking the potential of precision medicine in Europe. Improving cancer care through broader access to quality biomarker testing. Policy recommendations. IQN Path, European Cancer Patient Coalition and EFPIA. Feb 2021. Available at: Accessed May 2023.

[7] Walker BA, Mavrommatis K, Wardell CP, et al. Identification of novel mutational drivers reveals oncogene dependencies in multiple myeloma [published correction appears in Blood. 2018 Sep 27;132(13):1461]. Blood. 2018;132(6):587-597.

[8] Terpos E, Mikhael J, Hajek R, et al. Management of patients with multiple myeloma beyond the clinical-trial setting: understanding the balance between efficacy, safety and tolerability, and quality of life. Blood Cancer J. 2021;11(2):40.

[9] Kaplan, DA. Multiple Myeloma: Top 10 Advances in the Past 10 years. Available at: Accessed May 2023.

[10] Genomics Education Programme. Precision medicine: what is it and how will it be achieved? 2015. Available at: Accessed May 2023.

[11] Yosifov DY, Wolf C, Stilgenbauer S, Mertens D. From biology to therapy: the CLL success story. Hemasphere. 2019; 3:2.

[12] Smolewski P, Robak T. Current treatment of refractory/relapsed chronic lymphocytic leukemia: a focus on novel drugs. Acta Hematol. 2021;144(4):365-379.

[13] Bhatt et al. Relapsed/Refractory Multiple Myeloma: A Review of Available Therapies and Clinical Scenarios Encountered in Myeloma Relapse. Curr Oncol. 2023;30(2):2322-2347